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Lisa Suennen
Matt Wilsey, CEO of Grace Science, once said, “You may have a plan set out for your career and your family, but life doesn’t always turn out that way…you have to make the most of it…turn the lemons into lemonade.”
It is a fitting description for a person who has made several shifts on a quest to cure a rare disease.
Matt started his career in politics. He then moved to Silicon Valley, and co-founded several tech companies, including Zazzle and CardSpring.
In 2013, he was on the precipice of selling the latter company to Twitter when his daughter, Grace, was diagnosed with NGLY1 Deficiency. She was three.
NGLY1 deficiency is a complex neurological syndrome in which the patient lacks an enzyme known as N-glycanase 1 (NGLY1). NGLY1 helps the body degrade mis-folded, malfunctioning proteins, a process essential for cell homeostasis. People with NGLY1 deficiency experience many physical challenges and tend to have relatively short life spans. There is no known cure.
Matt Wilsey, co-founder and CEO, Grace Science
Matt and his wife Kristen struggled to understand how to best care for Grace and to learn all they could about the disease. The body of evidence was thin. Only five people in the world were known to have NGLY1 Deficiency at that time. Things looked grim, but Matt and Kristen didn’t give in to despair. Rather, they scoured the world for knowledge that could help them understand what could be done for Grace and others like her.
Matt made a sharp turn in his career, moving from tech to medicine. He taught himself as much as he could about the genetic disorder and related diagnostic and treatment options and sought out scientists who could help him learn about rare genetic diseases. One thing he learned early on – there was a lot for scientists to learn about the biology of NGLY1, and that new knowledge offered some new hope for patients.
With a bias towards action, Matt and Kristen poured their heart and resources into the founding of the Grace Science Foundation, a non-profit focused on funding medical research that could facilitate a cure for NGLY1, and Matt volunteered as President of the organization. While they feared they would be too late to help Grace, they wanted to ensure that other families would not experience what their family was going through.
As Matt and Kristen sought funding for the Foundation, a key challenge was convincing donors that the organization could operate at the highest level of scientific validity given the backgrounds of the founders. Many asked Matt and Kristen, who had no formal scientific training, how they could possibly drive the kind of quality medical discovery that would be necessary.
They answered those questions and successfully raised donations to support the Foundation. They spent a relatively small portion of it to find as many of the people on earth that they could who also had the condition that Grace has. By 2022, they had identified 100 individuals around the world who were living with NGLY1 Deficiency.
In 2017, when approximately 40 affected families had been identified, the Grace Science Foundation brought 21 of the families together for a conference in Palo Alto, Calif. They commiserated, shared their day-to-day experiences of living with NGLY1, and took genetic tests that could be used to the benefit of all. This was the beginning of a tight-knit rare disease community, and a valuable resource on which to build knowledge about NGLY1 Deficiency.
Many of these patients and families had never been on an airplane or left their countries or states of origin, much less interacted with others who lived with the same condition.
As the research progressed, it became clear to Matt and Kristen that the Foundation needed to partner with a biotech company to get the science translated into a treatment.
An entrepreneur by heart, Matt wanted to start his own company. He set that idea aside for a while, as he suspected that an existing for-profit enterprise would be able to develop a drug faster. He tried to convince one of the rare disease companies to collaborate but was told that the disease was too rare and that a cure was too much of a long shot. Having been denied, the team at Grace Science Foundation set their sights on starting their own company.
Along the way, Matt reached out to Carolyn Bertozzi, Professor of Chemistry and Professor of Chemical & Systems Biology and Radiology at Stanford University. Bertozzi is also an entrepreneur, having co-founded Lycia Therapeutics and Palleon Pharmaceuticals among others. The Foundation funded a few projects in her lab and she and Matt had become friendly. When she heard about the biobank he had built and met Grace, she offered up some of her post-doctoral students to help.
Carolyn Bertozzi
When Matt suggested that a more targeted approach would be to start a company together, Bertozzi got excited. Through the collaboration with Grace Science Foundation, Bertozzi had already shown in her lab that it was possible to shut down or turn off the proteasome as a means of treating cancers such as multiple myeloma.
To advance the new enterprise, incorporated in 2017 and named Grace Science, LLC, the team realized they had to open the aperture of the work to attract a broader slate of investors who had an interest in both the social mission and financial returns, not just in helping Grace and those like her.
To date, the company has raised $54 million, mostly from high-net-worth individuals and family offices who are patient; these investors are focused not just on returns, but also on the ability to make a societal impact.
Having secured the capital they needed, the company hired and partnered with an experienced team of scientists and drug development experts. They started focusing on churning out lemonade.
It wasn’t easy. Gene therapy was still a nascent approach. But it seemed the best path for their work after realizing that gene therapy had the potential advantage of addressing the missing gene and restoring function in key tissues. Small molecule development would probably take longer, and likely had too many off-target effects. So, they set off down the path of developing a gene therapy for NGLY1 deficiency.
This particular shift was especially challenging, as an entirely new animal model had to be developed to test their lead drug candidates. They found a partner in Takeda Pharmaceuticals, which helped them establish a new rat model. After Grace Science tested 39 different gene therapy constructs in human cell lines and advanced the top two candidates through preclinical tests, the company was able to demonstrate that their lead candidate was safe and effective in rats.
Grace Science now has a lead drug candidate for NGLY1 Deficiency in the GMP manufacturing process and is hopeful the FDA will soon provide a “green light” to start a clinical trial.
While this was happening, another shift was in the works – Grace Science began expanding its orientation towards becoming a multi-product company. Because the science had potential value for treating cancer, consistent with Bertozzi’s earlier work, the company is now developing a small molecule drug for cancers that that can be treated through NGLY1 inhibition. So far, they have identified eight potential malignancies that are dependent on NGLY1. Grace Science is ramping up fast on these initiatives, securing lab space, hiring a dedicated team and speaking with potential partners.
Along the way, Matt says, he learned three critical things.
First, it’s essential for a company like his to have “more than one line in the water…as many as the budget will allow.” This raises the chances of success.
He also learned the importance of finding balance between micro and macro managing. He works hard to stay close to what’s important, giving his employees autonomy to do their work, but without over-delegating. For instance, he said, he and Kristen used to speak regularly to every family in the NGLY1 community but as the numbers grew, he realized it wasn’t sustainable. They now have a genetic counseling team that helps keep in touch with their patient community. But Matt and Kristen continue to find ways to stay personally connected with the families with whom they share so much hope.
Matt says that his third key learning has been the importance of hiring people who are truly excellent and empathetic. “It’s not just A+ operators that you need, it’s A+ people,” he said.
Considering the short timeline – just eight years from starting the nonprofit foundation – a lot has happened. The foundation raised money and built a patient and scientific network. That work gave rise to a company. The drug development enterprise now has a novel product candidate on track to enter a Phase I trial in early 2023. The company knows it still has much evolution and lemonade-making ahead, but the family’s optimism for Grace and those like her grows by the day.
Academia has a tradition of doing nothing, or next to nothing, to educate and encourage young people to learn about entrepreneurship.
But whether academia wants it or not, it’s coming. Young scientists are taking matters into their own hands, organizing themselves in many major research hubs.
Nucleate, the nonprofit providing free resources to empower a new generation of biotech entrepreneurs, is announcing today it is roughly doubling its network of regional chapters from 9 to 17. The new nodes will organize young scientists in some major US research centers – Ann Arbor, Atlanta, Boulder, Chicago, Seattle and Texas. The group is also expanding internationally to the UK and Switzerland.
Soufiane Aboulhouda, PhD student, Church Lab at the Wyss Institute of Harvard University; co-founder and co-president, Nucleate
“Nucleate has created a mechanism for connecting us all together,” said Soufiane Aboulhouda, a PhD student in the George Church Lab at the Wyss Institute of Harvard University. “We’re no longer limited to the Harvard or MIT peer network. Now I interact with people from all around the world.”
What the trainees want to learn about, and interact over, is all the stuff they don’t get from the lab, or from class. How to put together a business plan to build on the science. How to start a company. How to think about IP. How to find the right service providers. How to make a pitch to raise money. How to build a network of people with complementary skills to turn an idea into a reality.
“They want to learn, and they want to learn by doing,” says Oliver Dodd, a co-founder and co-president of Nucleate. He’s also a PhD student in the Church Lab, long known for its entrepreneurial bent.
The current crop of young leaders at Nucleate have assembled educational resources online for what it takes to start a company. They’ve tracked down skilled industry mentors like Peter Barrett of Atlas Venture, former Alnylam CEO John Maraganore, Mira Chaurushiya of Westlake Village Biopartners, Cami Samuels of Venrock and Peter Kolchinsky of RA Capital. Well-known scientific entrepreneurs like George Church at Harvard, Steve Quake at Stanford and Pam Silver at Harvard are also among the advisors.
During the pandemic times, they’ve poured their energy into community organizing of students across the country, which would not have been possible before Slack and Zoom and social media. The resources they put online for their members, like so much else online, are free. Even a long-anticipated two-day in-person summit in Boston Aug. 29-30 is free to members.
About 560 grad students and postdocs are active organizers, Aboulhouda said.
Campus culture usually says to keep your head down in the science, work hard, and maybe someday you can become a principal investigator.
That’s not realistic for most people, and hasn’t been for a long time.
Mira Chaurushiya, senior partner at Westlake Village Biopartners, said the Nucleate model reminds her of Biotech Connection Bay Area – a student-led group that’s been focused more on preparing students for careers in consulting. She’s been impressed with the combination of talent, drive, and humility from the founding Nucleate crew.
Mira Chaurushiya, senior partner, Westlake Village Biopartners
“I love it when PhD students and postdocs self-organize in a way that’s productive for the whole community,” Chaurushiya said. “Unsurprisingly, they are incredibly high achieving people. To go from a standing start to what they’ve achieved today is really impressive. I’ve found them very thoughtful and humble. There’s a sense of ‘we know what we don’t know.’”
John Maraganore added: “Nucleate is a remarkable organization of students and young entrepreneurs keen to advance new frontiers of medicine. I’m amazed with how this group has grown over the years, and I’m awed by their passion and energy. There’s so much potential for this group to continue to expand as a nexus for future leaders in our industry.”
One of the key challenges will be sustainability. Student-led groups naturally have high turnover as people graduate and get jobs. Part of the challenge will be to institutionalize some of Nucleate’s processes, and develop leadership succession plans at each chapter to keep the momentum going, Aboulhouda and Dodd said.
Diversity as a value is almost a given. Half of the active members are women or gender non-binary, one in four members are from underrepresented minority groups, and members come from 44 countries around the world. Increasing diversity is “one of our primary goals,” the organization says.
There is so much interest, Nucleate turned away a few local chapters that expressed interest in joining the model this time. The group had inquiries from students at about 30-40 campuses around the world for this round of expansion that ultimately included 9 new chapters. Nucleate wants to make sure it expands its donor base to support thoughtful expansion.
The group has hired its first full-time employee, a finance person, Aboulhouda said. The vision longer-term is to have a small professional staff that can do a lot of the day-to-day work of managing the organization, with volunteer students remaining in leadership. That way, Nucleate will maintain its street cred as a group run by students, for students. It’s important to keep a finger on the pulse, to stay close to what the students need, Dodd and Aboulhouda said.
Michael Retchin, PhD student, Memorial Sloan Kettering Cancer Center; EVP of strategy, Nucleate
Dodd and Aboulhouda both say they’ve been influenced by the experience in building Nucleate, and their “Plan A” is to work at a startup after getting their PhDs.
Michael Retchin, a PhD student at Memorial Sloan Kettering Cancer Center in New York and EVP of strategy for Nucleate, said something about the group’s influence that stuck with me for days.
The community that’s been created is providing something deep, something needed, and something missing from traditional graduate school experience. It’s both a supportive fellowship for entrepreneurs, and an outlet for personal growth.
“It’s like going from being blind to being able to see things,” Retchin said. “I can see possibilities for my own career that I didn’t see before. And no longer do I think of my career as being solely for myself. Now my career is something collective. It’s about how do we work together, how do we advance the science together in the next generation. It makes me thrilled to work on this.”
Roger Longman, chairman, Real Endpoints
More than 14,000 people attended the annual #BIO22 meeting in San Diego, meaning there are at least 14,000 versions of what happened. But unless you have the emotional intelligence of a rock, you felt the unease, if not outright panic, of an industry with a fast-evaporating cash runway.
No one really knows what to do about it. If they say they do, they’re lying.
The biotechnology industry depends on two major financing sources: investors and acquirers. The former, in general, invest in companies they hope will sooner or later be acquired; the latter in companies that create medicines of significant medical and commercial value.
One problem: whereas acquirers were willing to pay significant premiums for drugs which had proven their value in clinical trials, the acquirers apparently now want their targets to do more to prove commercial value too, often by launching and selling their drugs.
It’s a recent change. A good example: the difference between Novartis in 2019 being willing to pay $9.7 billion for The Medicines Co. and its almost-approved PCSK9 inhibitor inclisiran (now marketed as Leqvio) and Pfizer, two years later, paying $11.6 billion for BioHaven only after it had successfully launched the migraine medicine rimegepant (Nurtec). It’s why, I increasingly believe, we see more biotechs launching, or about to launch, their own drugs.
The implication for investors: they’ll have to wait longer for most of their exits; capital costs will increase; returns in too many cases will decrease. The implication for biotech C-suites: they’ll now have to get smart about something most really haven’t had to worry much about — actually selling drugs.
Earlier stage companies will need to show investors they recognize the hurdles in front of them, commercially speaking. They’ll need to show they have a sophisticated understanding of payer strategies, distribution, pricing and contracting analogs, and patient support services. Moreover, they will need to be able to crisply articulate why they will succeed in a market chockablock with competitors, whether that competition is direct or for overall budget dollars.
Later-stage companies will need management who have successfully brought products to market – and actually want to do it again. Those people aren’t common and they’re not cheap. And both early- and late-stage companies will need board members who have an intimate and contemporary — not decades-old — understanding of the commercial and reimbursement challenges products face today.
A few CEOs we met at BIO think they just need to sit tight. Trim their costs, sure – but no need to re-set the goalposts. The investment cycle, as it has before, will roll on, they believe, and valuations will rise to pre-crash levels. They may be right about valuations — though that’s not a bet I’d be willing to stake my company on. And it doesn’t mean that the valuation endpoints will be the same.
We firmly believe that the biotech industry has delivered – and will continue to deliver – extraordinary value to both society and investors. The science continues to astound. But the valuation markers have moved. While clinical and regulatory proof is still a must-have, there’s now a second hurdle: quarterly sales.
The question is how quickly, and how well, biotechs can prepare themselves to meet that challenge.
Amanda Banks, MD
On Friday night, after the Supreme Court’s decision to overturn Roe vs. Wade, my extended family gathered around my mother’s dining table. We were supposed to be there to celebrate my 18-year-old niece’s graduation from high school.
We ended up talking late into the night, discussing the implications of the Court’s majority opinion which will greatly restrict access to safe abortion services for millions of women in the US.
I found myself counseling every woman and girl of childbearing age around that table, some as young as 12, to get an IUD or implant placed now. And to stock up on Plan B, the emergency contraceptive pill, currently available over the counter. Do these things before they, too, become illegal.
We’ve seen this attack on women’s rights coming for a while, but it’s still shocking. When the Supreme Court’s draft opinion was leaked in May, I wrote about how, early in my career as a physician, I took care of a woman in the ICU who ultimately died after seeking abortion care from an unqualified provider.
There is no question that now, in a post-Roe world where abortion will be criminalized but not eliminated, there will be a sharp rise in avoidable deaths of pregnant women. This in a country where maternal death rates are already far higher than most developed nations in the world. Worldwide, 47,000 women die each year as a result of unsafe abortion and an estimated 5 million are hospitalized for the treatment of serious complications such as bleeding or infection, the International Federation of Gynecology and Obstetrics estimates.
This number is now set to rise.
The Supreme Court is showing no signs of holding back. Access to birth control could be next.
Justice Clarence Thomas, in a concurring opinion with the majority, wrote: “in future cases, we should reconsider all of this Court’s substantive due-process precedents, including Griswold, Lawrence, and Obergefell.”
Those last three names are references to landmark cases ensuring rights to contraception, gay rights, and same-sex marriage.
Let’s look at the implications of what it would mean to block access to safe, effective contraception.
The CDC estimates that between 2015–2017, 64.9%—or 46.9 million of the 72.2 million women aged 15–49 in the United States were using some form of contraception. Nearly all women use a contraceptive method in their lifetimes. The most effective methods, IUDs and implants, have a failure rate of 0.1% or lower (similar to vasectomy), making these methods far more effective and reliable than tubal ligation, birth control pills and condoms.
The majority opinion, in overturning Roe, prioritizes the rights of a potential life (the fetus) over an actual life (the pregnant woman), regardless of the quality of that actual life. By abandoning the notion of viability (24 weeks) and not explicitly addressing in the opinion the need for any exceptions to a ban on abortion, including for the health and life of the pregnant woman, one could take the Court’s logic one step further to conclude that anything that ends a potential life is therefore also illegal. The link between birth control and overturning Roe lies in how these devices and medicines work:
IUDs and commonly-used implants release progestin, either locally in the uterus, or systemically in the blood stream, respectively. They prevent pregnancy in three key ways: by thickening cervical mucous and preventing sperm from entering, by suppressing ovulation, and by endometrial thinning that prevents implantation of a fertilized egg.
And therein lies the rub: if life is defined as beginning at conception (fertilization), and anything that ends that “life” is illegal, it is easy to see how any contraceptive method that works by preventing implantation of a fertilized egg would be swept up into this net. There is a clear conceptual path to see how these birth control methods and Plan B would be outright banned in at least half the country.
As all rights currently protected under the 14th Amendment are re-examined, let’s take this framework a little further: If the existence of life is valued above the quality of life, whatever the cost or collateral damage, what is stopping these same forces from prohibiting a patient with cancer from refusing additional chemotherapy or surgery?
If that patient values quality over quantity of life, if that patient does not want to bear the toxicities of additional therapy and the attendant hospitalizations it might require, but prefers to live their remaining days at home with their family, does hospice care become illegal?
This may sound outlandish, but is it really? Half of the people in this country have just been stripped of that same right.
I am reminded of the story of Shirley Wheeler, the 8th child of a mother who died when she was 13 months old. Shirley was passed off to relatives by her alcoholic father, and at 16, was raped and became pregnant. She carried this pregnancy to term and delivered a baby that was raised by relatives. When she became pregnant again in her early 20s, she obtained an abortion which was illegal at the time. She was convicted on manslaughter charges in 1971 because she refused to disclose the names of clinicians who provided abortion services.
At 23, she was subject to 20 years in prison for this crime, but was instead sentenced to two years of probation by a judge in Florida. But there was a catch: she had to either “get married or go home” to North Carolina where she was born
Let that sink in for a minute. Get married or go home.
In the pre-Roe era, a sitting male judge told an adult woman that she could avoid prison by getting married or going home to her abusive family.
Why should we think the post-Roe era will be any different?
After imperfect but profound progress for women in the US over the last 50 years since Roe was passed, what kind of nation will we now allow this to become? This matters not just to the tens of millions of affected women, but to their families, healthcare in the US which is already buckling under the pressure of broken delivery systems, and to the biotech industry globally.
As leaders in life sciences, we must take action now, before it is too late:
Leverage our talent pool to create new solutions. Leaders in our industry are already talking about creating solutions to match women to needed and available services, streamline travel and obtain financial support. They may also match qualified providers and other volunteers to clinics with staffing shortages.
Make contraceptive and reproductive health devices, services and medicines free to those without the ability to pay, and more accessible to everyone. Manufacturers of IUDs, implants, Plan B emergency contraception, and medicines that safely induce abortion in the early weeks of pregnancy must partner with providers and other stakeholders to make these options available to any woman who needs them. This is particularly important if we consider the possibility of future legislation limiting Medicaid coverage for birth control and related medicines.
Implement policies to support affected women. Provide paid time off and financial support for any employee who needs to travel to obtain reproductive healthcare that is banned in the state where they live.
Withdraw investment in states where bans continue. Do not set up operations in these states. Move headquarters if necessary. Venture capitalists should add a “social impact” parameter to their investment decisions.
Contribute to equity by showing your opposition to the Supreme Court’s action, and by continuing to diversify C Suites and Boards. As an industry we’ve made strides to become more representative and diverse in our leadership. It matters now more than ever. Don’t be silent. Take a position.
I grieve for our nation in my own particular way, with my experience as a doctor, a mom, a daughter all coloring my lens. Those of us who are angry and feeling helpless will rally — together with like-minded, dedicated, incredible people across our industry — to do what we do best: solve hard problems in ways that make people’s lives better and healthier.
We’ve been knocked back, but make no mistake, this doesn’t stop here.
Luke Timmerman, founder & editor, Timmerman Report
Sometimes doing the right thing requires sacrifice.
Other times, the right thing for human health can also be a shrewd business move.
That was the intriguing result of a study on the returns sponsors can expect from decentralized trials. The study relied on data from Medable and the Tufts Center for the Study of Drug Development. Results were presented this week at the Drug Information Association meeting, and are being prepared for a peer-reviewed paper.
The early results are eye-opening.
Companies that incorporated decentralized trial technology into their protocols – things like eConsent, remote monitoring, local labs, wearables and telemedicine visits to reduce in-person trips to the clinical site – saw a 5-fold return on investment in Phase II trials, and a 13-fold return in Phase III trials.
What it means in practical terms is that a $2 million upfront investment can be expected to translate into $10 million in savings in a Phase II trial. A $3 million upfront investment should deliver $39 million in savings in Phase III.
Biotech companies ought to take notice. That’s real money. Learning how to incorporate the new tools into clinical development also promises to make it easier for patients to enroll in clinical trials, for underrepresented diverse populations to get engaged, and to ultimately make medicines more accessible for the people who need them.
Pamela Tenaerts, chief scientific officer, Medable
“We need a lot more hard, systematically researched metrics on what decentralized trial technologies do for clinical trials,” said Pam Tenaerts, chief scientific officer of Medable. “A lot of methods we use in clinical trials hardly have evidence at all on whether they work or not. If people are going to spend money on something there has to be a reason… we can see it’s not only the right thing to do for patients, it’s a good business thing to do.”
With this analysis, researchers wanted to know from an expected Net Present Valuation perspective whether the technologies are worth adopting.
The pandemic provided a forcing function. Many companies saw their trials go on hold in early 2020, and had to get serious about learning to use the new tools if they wanted to stay on budget and schedule.
The data do have some caveats. There was a small sample size, with data compiled from 54 companies using decentralized trial technologies from 2020-2022. A larger dataset, with more time for follow-up, could shed light on more nuanced questions such as which decentralized technologies provide better returns than others, Tenaerts said. It would be good to know whether a certain technology is more useful for oncology, or neurology or some other indication, she added.
Getting the data is a good first step. Seeing these technologies pay off in the real world is encouraging.
We know the problem well. Way too many people find it too difficult to enroll in trials. It takes too long. It costs too much. It filters out many low-income people and people of color who can’t take a whole day off of work for repeated clinical visits three hours from home.
Many of us have become numb to these barriers to participation. We accept them as business as usual. This status quo is like sand in the gears of biomedical progress. It slows the whole machine down, and corrodes the enterprise. We often don’t get results from clinical trials that are representative of what we can expect in the real world.
The biotech community is capable of amazing leaps ahead when pressed into action. The community can pool more data to get better answers on best practices to make clinical trials more accurate, more representative, and more easily accessible to everyone. It would be a great way to help people see we’re living in the biotech century, and everyone stands to gain.
TR has long advocated for much bigger investment in basic biomedical research, as a catalyst for biotech industry development, regional economic development, and national competitive advantage. Investing in basic research is one of the best things the federal government can do, as we saw with far-sighted US government bets on the Human Genome Project, the Internet, GPS and many more foundations of knowledge that industry was able build upon. That’s why I was excited to see the US House of Representatives vote overwhelmingly to embrace this vision of what government can do at its best to advance health and economic development.
The House voted 336-85 in favor of a bill (HR 5585) to create the Advanced Research Projects Agency-Health (ARPA-H). It’s modeled after the Defense Advanced Research Projects Agency (DARPA), which invests in bold, futuristic research ideas, rather than the more incremental approaches that require some data already, and are often favored by the more traditionally conservative study sections at the National Institutes of Health. Reps. Diana DeGette (D-Colo.) and Rep. Fred Upton (R-Mich.) worked together to champion this bill, much like they did the 21st Century Cures Act. Side note: the legislation overwhelmingly passed the House without the presence of Eric Lander, the president’s former science advisor who championed the creation of this daring new agency, but who had to resign amid reports of toxic, bullying office behavior.
Cambridge, Mass.-based DEM Biopharma raised $70 million in financing to target ‘don’t eat me’ and ‘eat me’ signals on cancer cells and macrophage cells. Longwood Fund and Alta Partners led.
Boston-based Carbon Biosciences raised $38 million in a Series A financing led by Agent Capital. The company, founded by Longwood Fund, is working on parvovirus-based delivery for gene therapies. The hope is that it will enable larger genetic payloads to be delivered to targets.
Menlo Park, Calif.-based MyOme raised $23 million in a Series B financing led by Healthcare Venture Partners. The company does clinical whole genome analysis, helping families assess risk of inherited diseases. Sequoia Capital, Foresite Capital and Founders Fund participated.
Alameda, Calif.-based Magnetic Insight, an imaging diagnostics company, raised $17 million in a Series B financing. Celesta Capital led, and existing investors 5AM Ventures and Sand Hill Angels participated.
Burnaby, British Columbia-based Xenon Pharmaceuticals raised $250 million in a public offering at $30.50 a share. Earlier in the week, it released subgroup analysis from a Phase II trial for an anti-seizure drug candidate.
Beijing-based Therorna raised $42 million in a Series A financing to develop circular RNA therapies.
Moderna reported some more encouraging news for its bivalent COVID vaccine candidate that’s designed to stimulate the immune system against the original SARS-2 virus and the Omicron variant. The bivalent vaccine boosted neutralizing antibody concentrations by 5-fold against the BA.4 and BA.5 subvariants now spreading and causing re-infections. Moderna CEO Stephane Bancel said the company plans to start supplying the bivalent vaccine in August, to give people the opportunity to get a booster vaccine before the anticipated surge in cases likely to come this fall. See data published June 22 in the New England Journal of Medicine on the Omicron subvariants.
Cambridge, Mass.-based Scholar Rock reported 24-month follow-up data for patients in a Phase II study of its drug candidate apitegromab for type 2 and 3 spinal muscular atrophy. Researchers reported sustained improvement in motor function for patients who can’t walk, and that results were a bit better at 24 months than at 12 months. Based on the Phase II results, CEO Nagesh Mahanthappa said Scholar Rock is “urgently enrolling” patients in an ongoing Phase III trial.
Carlsbad, Calif.-based Ionis Pharmaceuticals and its partner, AstraZeneca, reported on positive interim results from a Phase III study of eplontersen for patients with hereditary tranthyretin-mediated amyloidosis with polyneuropathy. The drug is an antisense medicine, given by an autoinjector, and which is designed to inhibit production of the TTR protein at the root of the disease. Researchers said the drug reduced the amount of TTR in the bloodstream, and met its goal on a neuropathy clinical measurement. Ionis and AZ plan to submit the data to regulators. The drug will be a competitor to Alnylam’s subcutaneous RNAi medicine, vitrusiran (Amvuttra). The Alnylam drug was approved by the FDA 10 days ago.
Bothell, Wash.-based Athira Pharma said its lead drug candidate for Alzheimer’s disease, fosgonimeton, failed to hit the primary endpoint in a Phase II proof-of-concept study. The study was designed to measure a biomarker, change in ERP P300 latency.
Palo Alto, Calif.-based BridgeBio Pharma reported encouraging early data for its AAV-based gene therapy for Canavan disease, an ultra-rare and fatal disease. Researchers evaluating the first two patients saw significant decreases in a key biomarker, N-acetylaspartate (NAA), in the urine, the cerebrospinal fluid, and brain tissue as seen by Magnetic Resonance Spectroscopy scans. No serious adverse events were reported in the first two patients, the company said.
Mark Cuban’s Cost Plus Drug Company could save Medicare about $3.6 billion in one year with the discounted prices it obtained for 109 generic medicines through this February, according to researchers writing in the Annals of Internal Medicine. Since this involved a reality-TV show billionaire who’s a household name, it generated a lot of free media publicity for the Cuban Cost Plus Drug Company. There’s nothing wrong with negotiating for discounts on generic medicines, cutting out the middlemen, and making it simple and easy for people to purchase their meds at the lowest price. But I get a sinking feeling when reading articles like this in the WSJ that make Cuban look like a hero riding to the rescue of US healthcare, standing up for the little guy against those greedy monsters in the pharmaceutical industry. We should beware of billionaires who say they come to us bearing gifts for the masses, but may have something else in mind altogether. Like a Presidential campaign.
I’d feel better if this “Cost Plus Company” was run by talented operators behind the scenes, not a celebrity frontman. See my previous coverage (Frontpoints, Jan. 2022).
Newark, Calif.-based Ultima Genomics, the well-financed startup aspiring to sequence whole genomes for as little as $100, said it formed a long-term supply agreement with Madison, Wis.-based Exact Sciences. Exact, the maker of the noninvasive Cologuard screening test for colorectal cancer, agreed to invest in Ultima, and become an early-access customer for its next-gen sequencing technology. Driving down the costs of sequencing is especially important for high-volume clinical applications like cancer screening, minimal residual disease assessment, and recurrence monitoring, Exact CEO Kevin Conroy said in a statement.
Pfizer agreed to invest $95 million in France-based Valneva, and alter a few terms of its 2020 license agreement, to support the development of a vaccine for Lyme disease. Pfizer plans to start a Phase III trial of VLA15, a protein subunit vaccine candidate, in the third quarter of 2022.
Durham, NC-based Precision Biosciences secured $75 million upfront through a deal with Novartis, in which the big company will get access to its technology to enable in vivo gene editing for the treatment of beta-thalassemia and sickle cell disease. Precision quickly followed up the partnership news with a $50 million stock offering at $1.39 a share.
Boston-based Radius Health agreed to be acquired by Gurnet Point Capital and Patient Square Capital in a deal valued at about $890 million. The company’s lead drug is for reducing the risk of fractures in postmenopausal women with osteoporosis.
Waltham, Mass.-based Morphic Therapeutic, the developer of small molecule drugs against integrin targets, said that AbbVie walked away from a collaboration that began in 2018.
Belgium-based Galapagos agreed to acquire CellPoint for 125 million Euros upfront, and AboundBio for $14 million. The deals are intended to strengthen Galapagos’ cell therapy capabilities.
AbbVie secured FDA approval for risankizumab-rzaa (Skyrizi), an IL-23 inhibitor, for moderate to severe Crohn’s disease.
The FDA ordered Juul Labs to pull its e-cigarette products off the US market. The products have long been marketed to teens. It’s a rare and tough public health enforcement action on the part of the FDA under Commissioner Robert Califf. (FDA statement).
The FDA approved Merck’s 15-valent conjugate vaccine (Vaxneuvance) for pneumococcal infections in infants and children.
Novartis secured FDA clearance to sell the combo of dabrafenib and trametinib (Tafinlar + Mekinist) for adult and pediatric patients with BRAF V600E mutations, regardless of the tumor’s tissue of origin. See Novartis R&D chief Jay Bradner’s comment on how cancer is moving to targeted therapies based on precision molecular definitions such as this.
Watertown, Mass.-based Enanta Pharmaceuticals sued Pfizer for patent infringement. The case involves nirmaltrevir (Paxlovid), the antiviral treatment for COVID. Baird analyst Brian Skorney said the case appears to have some merit.
President Biden nominated Arati Prabhakar to be his science advisor. She’s an engineer and physicist by training who previously led DARPA in the Obama Administration. Her Cabinet-level position requires Senate confirmation.
Chirfi Guindo, chief marketing officer, Merck Human Health
Merck said Chirfi Guindo will lead marketing for Merck Human Health, starting July 1. He will report to CEO Rob Davis. Guindo was previously executive vice president and head of global product strategy and commercialization at Biogen.
Baltimore-based Delfi Diagnostics added Jacob van Naarden to its board of directors. He’s a senior vice president at Eli Lilly and CEO of Loxo@Lilly.
Cambridge, UK and New York-based Artios, the developer of small molecules aimed at DNA damage response, added Samantha Truex to its board of directors. She’s the CEO of Upstream Bio.
Cambridge, Mass.-based Oncorus, a viral immunotherapy company focused on cancer, added Doug Fambrough, former CEO of Dicerna Pharmaceuticals, to its board of directors.
San Diego-based Iconovir, an oncolytic virus therapy developer, added William Kaelin to its board of directors. He’s a scientist at Dana-Farber Cancer Institute and a Nobel Laureate.
Real Estate
Regeneron Pharmaceuticals broke ground on a $1.8 billion expansion of its campus in Tarrytown, New York. Gov. Kathy Hochul said in a statement the company has pledged to create 1,000 jobs at the facility over the next five years.
Takeda Pharmaceuticals signed a 15-year lease in Cambridge, Mass. on 600,000 square feet in R&D and office space. The facility is at 585 Third Street, walking distance from major research universities, startups, venture capital, mid-to-large companies, and more skilled consultants and service providers than you can shake a stick at, as my grandmother used to say.
Moderna said it’s building a new mRNA vaccine manufacturing facility in the UK, with the goal of turning out new vaccines in 100 days. The UK has also agreed to purchase 60 million doses of Moderna’s COVID vaccine for booster campaigns in 2022 and 2023. Moderna also has plans for an mRNA vaccine manufacturing facility in Kenya.
Today’s guest on The Long Run is Ivana Magovcevic-Liebisch
She is the CEO of Cambridge, Mass.-based Vigil Neuroscience.
Ivana Magovcevic-Liebisch, president and CEO, Vigil Neuroscience
The story starts with Amgen. The big biotech made a strategic decision to get out of neuroscience in 2019. That meant a couple of drug programs targeting TREM2 came up for sale.
One is a monoclonal antibody, and the other is a small molecule. They are aimed at a neuro-inflammation target on microglia cells, and it has been implicated in genome-wide association studies for rare neurological diseases, and for larger indications like Parkinson’s and Alzheimer’s.
Atlas Venture, which has a longstanding relationship with Amgen as a limited partner in its funds, sought to in-license the programs – which it thought had potential in a small, focused, asset-centric startup.
That’s where Ivana and Vigil Neuroscience enter the picture. She’s an experienced executive, with stops at Dyax, Teva, and Ipsen along the way. She’s been through some ups and downs in the early days of Vigil – venture financings, an IPO, a bearish stock market, and a partial clinical hold while the company and FDA discuss dose ranges with its lead monoclonal antibody for a rare disease called ALSP.
I saw Ivana speak at a recent conference about the importance of building a strong team and culture to navigate through a downturn. I think she has some valuable perspective for managing in these times.
Now before we get started, a word from the sponsor of The Long Run.
Calgary is home to more than 120 life sciences companies, from emerging startups to established firms. With this critical mass of research, technical talent and expertise, the city is an active hub for life sciences innovation.
Technologies homegrown in Calgary are changing the face of healthcare. Syantra is revolutionizing breast cancer detection using artificial intelligence-derived algorithms. NanoTess is harnessing the power of nanotechnology to tackle chronic wounds and skin conditions. And this is only the beginning. Calgary’s life sciences sector is projected to spend $428 million on digital transformation by 2024.
If you’re a bright mind or bright company solving global health challenges, Calgary is the place for you.
Take a closer look at why at calgarylifesciences.com
Now, please join me and Ivana on The Long Run.
Vikas Goyal, former SVP, business development, Pandion Therapeutics (now part of Merck)
UK-based Kymab made headlines back in January 2021. Sanofi agreed to acquire the antibody drug developer for $1.1 billion upfront, plus $350 million in potential milestones.
The main attraction for Sanofi was KY1005, a monoclonal antibody targeting OX40L, a regulator of the immune system. Five months before the deal, Kymab announced it hit both primary endpoints of a Phase 2a trial of patients with moderate to severe atopic dermatitis that wasn’t responding to topical corticosteroids.
Sanofi, in its official statement, mentioned its interest in an ICOS-agonist antibody in clinical development for cancer, along with Kymab’s broader capabilities in antibody drug discovery.
I spoke recently with Brandon Lewis, the former head of corporate strategy at Kymab, on the dynamics that were at work in this transaction.
Brandon Lewis, former head of corporate strategy, Kymab
Why was this deal the right thing to do?
We realized that in order to give KY1005 the best chance of success we needed a committed partner with deep intellectual and financial resources focused in immunology. Sanofi presented the opportunity to collaborate. Sanofi has a great share of voice with patients and physicians plus an incredible amount of knowledge about the potential in dermatological diseases and beyond for the OX40-Ligand (OX40L) pathway. And they saw how our molecule could have application across multiple dermatology indications and certain autoimmune disorders.
At that moment in time, it was becoming increasingly clear that there were only so many R&D teams that had the wherewithal, capacity, and interest to drive the bus through this tiny little window to see what could happen for patients. And after many years with both of our lead molecules advancing on promising trajectories, we felt our lead OX40L molecule KY1005 deserved that, as did our immuno-oncology program KY1044 (an ICOS agonist antibody).
As we began to see some early open label clinical activity for KY1044 and recognizing the evolving competitive landscape in immuno-oncology in general, along with the opportunity to pursue both mono and combo therapy paths, we believed it would be hard for an emerging drug development company to do both programs properly and at pace by ourselves.
Several potential partnerships were available, offered, discussed.A few entered negotiations. Nigel Clark (Kymab’s SVP of Business Development, now CBO at Nucleome) is an incredible dealmaker with a unique business acumen. The parties varied from dermatology-specific companies, a few regional partners, as well as a few large pharma / large biotech companies that saw elements of KY1005’s potential but not the same breadth of potential that we saw. Of course, there was a range of opinions on how KY1005 should be priced, and whether to stick with dermatology or go broader.
We ultimately wanted a partner with the necessary enthusiasm, interest and knowledge of the space. And so that led us back to the folks at Sanofi. They had reviewed everything in the space. And had become category leaders in immuno-dermatology, which is classically a difficult space to gain adoption with new therapeutic interventions. Plus, dermatology practices have unique economic incentives regarding interventions. Even great drugs have to compete with things like microsurgery, Mohs, and cryotherapy. To get that share in dermatology, we knew we had to work with a committed company that had an established presence with both patients and physicians to launch another new therapy with a novel mechanism of action.
What drove the timeline for your acquisition? Why did this happen in January 2021?
In 2017, Kymab had four therapeutic areas and a large research and translational footprint of 150 people, plus a burgeoning clinical development organization. And the company had just raised its last equity financing in 2016.
ASCO 2017 had been incredibly exciting, and the world went mad for immune oncology. We had our first two presentations on our ICOS program at SITC in November 2017 where there was a lot of excitement with combination checkpoint inhibition at that moment. So strategically the plan was to raise a modest amount of capital, take KY1005 and KY1044 into Ph1 studies and get some early clinical data, with KY1044 as the lead focus. We had also recruited Dr. Sonia Quaratino as our Chief Medical Officer to lead the clinical development of both programs. She joined us from Novartis and is brilliant at translational clinical development. She is an immunologist with very strong oncology development experience.
Fast forward to ASCO 2018. The highly anticipated results for IDO landed awkwardly and theICOS data presented by others in the field was going to require a lot of additional interpretation. On Day 1 of ASCO the palpable enthusiasm in the audience quickly turned to concerns and questions . We had not yet presented any human clinical results from KY1044, but now we had to respond to the market’s collective confusion about the target and emerging questions around clinical development trials for combination therapy. This again, presented a considerable communications challenge to overcome and required a new approach to establishing KY1044’s target product profile.
So now we are in the summer of 2018 post an enthusiastic American Academy of Dermatology (AAD) in February and let’s say a more somber ASCO. We had already achieved some incredible preclinical in vivo data in acute and chronic Graft-versus-Host Disease (GvHD) models with our OX40L antibody, KY1005, in some challenging primate models run by Leslie Kean (then at Seattle Children’s now at Boston Children’s) which we published in Science Translational Medicine. In this model, we had a striking survival benefit. And when we examined the treated animals at the end of the study, it looked like we were resetting these activated T cells back to a homeostatic state. So, we realized we had an incredibly potent molecule that was doing the biology that we wanted it to do.
It is crucial to choose the most appropriate setting that will showcase a molecule’s full potential or shortcomings in the clinic. Atopic dermatitis provided this for KY1005. There was a clear biological rationale supported by a strong and growing body of research that published in 2012 identifying the presence of OX40L in atopic dermatitis lesions.
Despite the biology associated with OX40/OX40L axis there was still limited human clinical proof-of-concept for these mechanisms at that time back in 2017. Roche-Genentech had earlier published very nice data in transplant, but they moved forward in asthma where the studies did not produce the intended results. We had been evaluating the potential intervention in atopic disorders and skin diseases where the literature supported the biological rationale to target the OX40/OX40L axis. In the first quarter of 2018 the first in human study of KY1005’s safety and tolerability was nearly completed and we were incredibly optimistic. After this readout, our optimism matured to excitement as KY1005’s properties and drug-like attributes became further substantiated. Sonia and I headed to AAD in February 2018 in San Diego with this preliminary knowledge of KY1005 and a busy schedule of meetings with key opinion leaders and principal investigators, to further refine our thoughts around the Phase 2a study design in patients with atopic dermatitis given what we understood about KY1005’s proposed mechanism of action. There was a lot of excitement in the medical dermatology space.[Regeneron and Sanofi’s] Dupixent [an IL-4 and IL-13 inhibitor for atopic dermatitis] had begun to catch its stride and there were many companies presenting “small” signal seeking studies around blunting the immune response in atopic disease.
If you recall, until now, the OX40/OX40L checkpoint axis was primarily being investigated as another immuno-oncology approach and had limited success. These approaches were trying to agonize the [T-effector cells] Teffs and dampen the [T-regulatory cells] Tregs. KY1005 is designed to do the opposite by working to restore a more normal balance of these cell subsets. This is what we investigated in the acute GvHD animal models. This presented a real hurdle when educating the Street that our intervention was in autoimmune diseases not immuno-oncology and KY1044 was doing the opposite targeting ICOS pathway in immuno-oncology.
A company then named Glenmark (now Ichnos) was presenting on an open label uncontrolled data with its anti-OX40 antibody in atopic dermatitis. It was a small dataset, but it provided additional encouragement for the pathway and its clinical rationale. However, there are always risk and rewards with making apples and oranges comparisons. When interacting with external stakeholders there is never enough time to get through the whole story in most 1×1’s, and even more to actually do a proper deep dive on why you selected the optimal molecule .
Sonia and her team were working hard on the filing for the KY1005 study in atopic dermatitis patients.We were convinced KY1005 and its emerging therapeutic profile would have a meaningful benefit, there was a clear unmet need to offer better therapies than topical steroids, and we saw potential to be one of the first to market.
The classical development challenges remained, working through the atopic dermatitis clinical trial designs, dealing with inter site variability and patient recruitment. We worked hard and worked fast to get the trial done. And of course, the competitive landscape in atopic dermatitis evolved. We saw data from Anaptys, Equillium, Dermira, and others. And all of a sudden, we were 6th or 7th in line with KY1005.
We also had two new targets progressing to development candidate stage and were facing the decision to shift resources from discovery to development. Our discovery team was continuing to find cool new things to work on. And our development organization was trying to push INDs and CTAs forward.
In 2019 we had recruited a great new CEO Simon Sturge and he brought a new level of large pharma and biotech experience, along with corporate discipline and a commercial strategic focus to the Company. Biotech companies are constantly looking for funding and so we were always evaluating a variety of options, including, but not limited to, a possible IPO.
Later in the summer during a series of non-deal roadshows meeting investors in the US we were hit with a “black swan event” of the Woodford funds. It had been one of our largest investors after our 2016 Series C. And in the background we were also dealing with an ongoing patent challenge to some of our platform patents.
Drug development is incredibly hard, but there are so many other seen and unforeseen obstacles that will emerge and have to be overcome. I mean every time we got through one door . . . But the company persevered. Our investors and board remained very supportive of us continuing to build the company and refine the story. And we were executing nicely on both our lead clinical programs..
Now move forward to 2020 and our OX40L program is looking really promising. There was no question we had a competitive advantage on dosing schedule, durability of response, and on our impact on the immune axis. Meanwhile other biological mechanisms had not achieved the clinical success many were hoping for, and although the JAKs efficacy results were encouraging, long-term safety questions would need to be understood. And in 2020 we also got some favorable patent decisions in the US and UK.
So now there was no question this could be a successful drug. The question became, would a small company like Kymab turn KY1005 into a category killer? And could we keep up the pace to move forward much more aggressively? We were at a crossroads and the timing was right to work with Sanofi.
How important was the US Patent Trial and Appeal Board’s decision in all of this?
It was really our own data trailblazing the role of OX40L inhibition in atopic dermatitis that drove all of this. The patent issues were not at the core of it. I think this was a distraction that you don’t want to have as an operator and executive. It would have been a constant battle to get people to focus on what the Company is doing. If I was sitting on the other side of the table, this kind of issue is also an easy way for a partner to change their risk matrices in their models, because at the end of the day this is about getting to a number.
And just for context, David Chiswell (Executive Chairman, scientific advisor, and former CEO of Kymab) and I worked closely together during the early days of Cambridge Antibody’s corporate affairs and development during the early clinical translation of D2E7, which ultimately became Humira. At the time, the investment world was consistently telling us that “we don’t need another TNF-alpha inhibitor because we already had Enbrel and Remicade, they are effective and appear to be disease modifying”. And so we had some personal experience with how external dynamics can affect a young company. You have to follow the data and be confident in the collective work of your teams or you will get lost in the “noise”. Obviously, this is much easier said than done.
How did the deal process go?
We were fortunate to have many options available to us in parallel. We had a broad process that started north of 40 companies. About half of those guys went to the next room. And several of those went to letters of intent that we reviewed with our advisors and investors. Across those proposals we had a version of each path forward. We had a capital markets solution that would let us do a lot on our own, a partnership proposal that was more-or-less a traditional 50/50 collaboration, and a partnership proposal that would have seen us building a global clinical organization and us taking KY1005 all the way to Ph3 ready.
But you know how it goes. In the middle of this, we got a call late one night, and suddenly teams are working through the holidays to get a deal done with Sanofi. Sanofi had been watching the Company intimately for a long time, and their scientists and clinical teams had been huge advocates for our OX40L program. And when Paul Hudson, CEO of Sanofi, took over and started doing the deals like the acquisition of Synthorx, that opened a window for us in a very different way across our ICOS and other oncology programs. And now Sanofi offered a way for us to properly resource all our programs.
Why did you take the $1.1 billion? How did you get comfortable that was the right price? Why not $700 million or $3 billion?
Ha, through many painful hours of splitting hairs and ongoing market analysis conducted by our business development and finance teams internally before the process was even kicked off. There was no question our OX40L was targeting a very large market opportunity, and out of everybody Sanofi uniquely understood the size of the market given their commercial presence.
And look, some other partners may have been getting to larger cumulative payments in their respective models, but the structures were such that the real payments may never have happened.
And on the capital markets side, we had enough investor feedback to estimate how much money we could likely raise and at what terms.
How did you manage all this activity?
We had an incredibly strong and dedicated internal team along with a deep bench of experienced advisors as is required with these endeavors. Personally, I thought we should either take it all the way on our own and raise enough money to do that or take an option that removes the risk and would not be dilutive to our existing shareholders.
As for me, I’m sitting inside the company as an external corporate development person, so I liaise with all my key stakeholders internally. And of course, looking across this incredibly productive platform, which is generating multiple leads for development candidate consideration, how can you ensure everyone gets the resources needed for each respective program and also align them with our strategic corporate development objectives and also balance with the resources actually available to you. We were still a young biotech, we didn’t have the evolved and established portfolio management and resource allocation expertise of more mature development companies.
There was a lot to manage. It was a lot like a chess game where we had keep thinking three steps ahead. Because every time we didn’t think ahead something would surprise us. It was not easy and I’m so proud of our team. Nigel only had 3.5 people in his business development team. It was only me and Anne Hyland (Kymab’s CFO) in the external facing finance function. And for a big chunk of this critical time, the CEO role was transitioning from David to Simon.
Why do a whole company sale? Why not a co-development collaboration on the atopic dermatitis program or a cancer partnership?
All these paths were considered at different times. We didn’t set out to sell the company.
This all really happened over the course of four years. When I joined Kymab, it was obvious to us that we had a great potential in our platform to generate antibodies to otherwise refractory targets and to achieve high biological function with those antibodies. Kymab had developed great tools to probe the immune system, identify antibodies, and create very focused and evolved biological libraries to design functional attributes of our antibodies in a way that had not historically been done for biologics.
We were working in infectious disease and had a collaboration with the Gates Foundation, we had a hematological focus with our BMP6 and matriptase programs (now licensed to RallyBio) and then of course we also had immunology and immuno-oncology. The breadth of our platform was so robust, that there was a real challenge as we began to grow into a clinical stage company. It’s not easy to develop relationships with PIs and have deep knowledge across all those areas.
And when we looked at the intellectual and financial capital needs to bring our two clinical programs and our growing preclinical pipeline through Ph2 proof-of-concept, it was going to require raising multiple hundreds of millions of dollars. So, in addition to a broad range of partnership pathways, we also aggressively and simultaneously explored several capital market solutions whether that was IPO in the US or locally in the UK, a mezzanine round, and even SPACs and reverse mergers. We believed it might be difficult to raise that amount of capital given our business strategy cut across so many therapeutic areas, but we were far enough along in these discussions to know the capital was available.
We were really at a crossroads. And like many companies, it started to become clear that our lead program was driving a lot of the perceived equity value. We could have put several strategic puzzle pieces together and come out with a mosaic.
With Sanofi we found a single partner with interest in immunology, oncology, hematology, infectious disease, and in antibody platforms. And through the acquisition by Sanofi, we found a business solution to help build and resource Kymab’s drugs and platform through one transaction as opposed to a less certain series of 4+ different transactions.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Many bright young people never get a chance to fulfill their potential as scientists because they never get on the usual scientific career on-ramps.
Many young people who get sick struggle because no one had the motivation or the wherewithal to develop new treatments or diagnostics.
Seattle Children’s Research Institute sees the problems, and has crafted a major initiative to tackle both at once. It’s through the Invent@SC Postdoctoral Scholars Program being announced today. It’s a program to recruit and support 50 postdoctoral researchers from underrepresented minority groups, helping them build on their scientific training to become scientific entrepreneurs and drug developers.
The 5-year, $45 million program includes a $12.5 million commitment from the Washington Research Foundation. Postdocs will get starting salaries of $65,000 a year – more than the NIH standard for living in Seattle – plus access to experienced industry mentors and technology resources. They’ll get training in inventorship, team science, and entrepreneurship that is seldom part of post-graduate education but which is essential to industry.
The program was designed with input from young minority scientists familiar with the challenges of those who come from disadvantaged backgrounds. To make it appealing to people who may not immediately gravitate to a predominantly white city like Seattle, it includes a community component with experienced lab, clinical, and industry mentors, many of whom are Black. The group’s connections also extend to the wider Seattle scientific community that includes labs at the University of Washington, Benaroya Research Institute and Fred Hutchinson Cancer Center.
Jim Olson, principal investigator, Seattle Children’s Research Institute; program director, Invent@SC
Jim Olson, a pediatric oncologist and founder of Seattle-based Presage Biosciences and Blaze Bioscience, is leading the Invent@SC program. He moved his lab from Fred Hutch to Seattle Children’s and gave up his clinical practice of 30 years to carve out more time for the initiative.
“This is a one-of-a-kind program,” Olson said. “It’s focused on pediatric therapeutics, with an emphasis on recruiting scientists from diverse backgrounds. We want to help educate the next-generation of biotech entrepreneurs.”
Most people that go to biotech companies were never trained to discover therapeutics, do milestone-driven research, or make the kind of go/no-go decisions required in industry, Olson said. He envisions Invent@SC developing a culture like that of a biotech company, with an emphasis on collaboration, and clear goals to develop new treatments for pediatric diseases.
Steve Graham, the co-chair of life sciences at Fenwick & West in Seattle, is retiring this month after a 40-year career. He’s one of the more prominent African Americans in biotech. He’s been winding down commitments.
But he said he couldn’t say no to an invitation to advise this new generation of young minority scientists.
“When I heard Jim describe this program, I said to myself that I have to do this,” Graham said. “This is something he really cares about fiercely. He’s passionate about it. It’s not a passing fancy. He’s truly aware of the issues, and his awareness extends to knowledge of what he’s not aware of. You sometimes run across people who are very, very smart, and that can sometimes translate into thinking they know everything. That tends not to be helpful in finding solutions.”
Steve Graham, co-chair, Fenwick & West Life Science Practice
Uciane Scarlett, a principal at MPM Capital in Boston, a cancer immunologist by training and a Black woman originally from Jamaica, is another of the mentors.
Scarlett said she agreed to help because Invent@SC meets a need for underrepresented scientists, it’s rigorous and milestone-driven like industry, and it’s focused on cell, gene and protein therapies that have a high chance of making an impact. (Disclosure: I’ve also agreed to serve as a mentor.)
“I am a true believer that the root cause of the limited diversity in life sciences is due to certain opportunity gaps for underrepresented groups,” Scarlett said. “Professor Olson is taking a holistic approach with the BIPOC program as he’s going above and beyond funding scholars to do research in childhood cancers — he’s also bridging a major component of the opportunity gap, and that’s access to relevant networks and mentors.”
Uciane Scarlett, principal, MPM Capital
The Seattle program isn’t the only one dedicated to launching scientific careers for members of underrepresented minority groups. The Howard Hughes Medical Institute (HHMI) last month announced the Freeman Hrabowski Scholars program. It’s starting with $1.5 billion of commitments over the next 20 years to propel the careers of 150 young scientists committed to improving diversity, equity and inclusion in science.
The original idea for the program goes back about six years, Olson said. That’s when one of the African American grad students in his lab, Eric Nealy, attended a seminar for HHMI Gilliam Fellows in the Washington DC area.
Nealy came back to the lab beaming.
“What’s going on? Did you find a new girlfriend or something?’” Olson recalled saying.
No, Nealy replied:
“I just spent a week surrounded by people who look like me who are smart and doing great biomedical research. It was the first time I felt like I belonged, like I wasn’t the only one.”
Alison Williams and Eric Nealy. Photo courtesy of Seattle Children’s.
That got Olson thinking. Could he create a more inclusive community, starting small in his own lab? He had conversations over the years with many scientists from minority groups – including Nealy, who’s now a postdoc, and Alison Williams, a research scientist in the Olson lab. Their input, along with others, helped shape the program.
Creating an inclusive and supportive community in this case means more than recruiting a couple of Black scientists. It means casting a wider net to attract scientists from indigenous groups, or others from underrepresented backgrounds. It could include people who grew up in foster care, were homeless, identify as LGBTQIA, or are the part of the first generation in their family to go to college.
The goal will be to recruit an average of 10 postdocs per year for five years. The program is seeking applications this month, and will accept them on a rolling basis, Olson said.
Applicants will be evaluated not just on the traditional accomplishments valued in academia. Some of those accomplishments get accumulated after doors are opened for people who have multi-generational connections to science, Olson said. The Invent@SC program, aware of traditional structural disadvantages for young people who don’t have those advantages, will seek candidates based more on potential, motivation, curiosity, creativity and commitment to gaining new knowledge, Olson said.
Once candidates are selected, the next crucial step will be in fostering the sense of support and belonging. The program is holding a launch event tomorrow in Seattle as a first step. It’s establishing a social fabric that’s hard to measure, but undeniably important in advancing scientific careers where failure is so common.
“Too often, you find people of color, or women, who find themselves in positions where they could thrive but they don’t thrive because there’s no one there to understand their position,” Graham said. “As a result, they don’t get the right kind of moral support.”
Seattle Children’s, like other institutions in the Seattle scientific community, has an entrepreneurial track record. Its faculty have contributed to Juno Therapeutics, Be Biopharma, and Umoja Biopharma, among other recent startups. It has valuable resources for scientific entrepreneurs, including an 11,000-square-foot GMP facility for making novel cell therapies, and a 4,700-square-foot VectorWorks GMP facility for manufacturing product candidates for early phase clinical trials.
Olson emphasized the combination of the resources of Seattle Children’s, and the people who are curious and hungry to make good use of them. The tools of cell therapy, gene therapy and advanced biologic capabilities at Seattle Children’s are unique among its peer institutions, he said. Those tools can be brought to bear on research into pediatric autoimmunity, mental health and infectious diseases.
Olson, 59, said underrepresented minorities have made major contributions in his lab over the years. That includes the “tumor paint” work that led to the founding of Seattle-based Blaze Bioscience.
The Invent@SC program is an attempt to build on that type of success, and to do it in a more intentional and intensely focused way.
“When I see people coming into my lab with unrealized potential and all of a sudden they discover tumor paint and start a company, or help come up with technology for Presage, they sometimes look back and can’t believe what they accomplished,” Olson said. “I saw something special in these people. Sometimes it was a chip on the shoulder. Resilience. Or natural curiosity. All I did was create an environment where they could flourish. I get so much joy out of that.”
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Today’s guest on The Long Run is Rahul Singhvi.
Rahul is the co-founder and CEO of Resilience.
The company made a splash in the fall of 2020, when it debuted with an $800 million Series A financing. Bob Nelsen of ARCH Venture Partners led the deal. The company has now raised a couple more rounds that add up to more than $2 billion.
Rahul Singhvi, co-founder and CEO, Resilience
Nelsen, a previous guest on The Long Run, saw what many others saw – a global supply chain for manufacturing that was suddenly vulnerable to disruptions from a pandemic, and, more recently, from war. He and other investors came together to found Resilience as a domestic manufacturing response to this vulnerability. They sought to build more capacity in the US and Canada for advanced biologics, and gene and cell therapies, with a network of high-tech facilities.
Rahul came to this moment with a wealth of pertinent experience. He had spent 25 years of his career working on vaccine manufacturing.
He’s been busy the past two years at this startup, acquiring existing biotech manufacturing facilities and re-tooling them. He and his team have been forming partnerships with large and small companies that need to manufacture advanced products, and could use help with sharpening their processes and technology for manufacturing at scale.
The vision of what Resilience is doing has become more clear, at least to an outside observer like me. I’ve written a fair bit about the opportunity for more domestic biotech manufacturing, for national security reasons, for national high-tech competitiveness, for regional economic development, for creating high-quality manufacturing jobs, and for creating stable businesses.
Rahul also points to a technology industry analogy, in which more specialized, sophisticated partners work together in an ecosystem, rather than each individual company trying to own every piece of the value chain. It’s an example of horizontal scaling that has worked in tech, and could now work for biotech.
Now before we get started, a word from the sponsor of The Long Run.
Calgary is home to more than 120 life sciences companies, from emerging startups to established firms. With this critical mass of research, technical talent and expertise, the city is an active hub for life sciences innovation.
Technologies homegrown in Calgary are changing the face of healthcare. Syantra is revolutionizing breast cancer detection using artificial intelligence-derived algorithms. NanoTess is harnessing the power of nanotechnology to tackle chronic wounds and skin conditions. And this is only the beginning. Calgary’s life sciences sector is projected to spend $428 million on digital transformation by 2024.
If you’re a bright mind or bright company solving global health challenges, Calgary is the place for you.
Take a closer look at why at calgarylifesciences.com
Now, please join me and Rahul Singhvi on The Long Run.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Luke Timmerman, founder & editor, Timmerman Report
TR subscribers: I’m traveling more, and looking forward to seeing more of you in person.
I’ll be at the BIO Convention in San Diego, June 13-15. They have plenty of outdoor patio space overlooking the ocean at the San Diego Convention Center. Let’s chat.
While at BIO meeting people and gathering ideas, I’m also moderating a panel on improving diversity, equity and inclusion.
Time: 1:45-2:45 pm
Date: June 15
Place: Upper Level, Session Room 5A, San Diego Convention Center
Panelists:
Emerging Frontiers in Oncology: I’ll be in Cambridge, Mass. June 30 to moderate at the Emerging Frontiers in Oncology meeting. It’s a half-day forum at the MassBio event center at Technology Square, followed by a networking reception at Catalyst.
Speakers include:
The event is organized by Uciane Scarlett of MPM Capital, as a fundraiser for the Timmerman Traverse for Life Science Cares. For tickets, ask Uciane (pronounced YOU-shuh-nee) uscarlett@mpmcapital.com.
Timmerman Traverse for Life Science Cares: My team of 20 biotech executives and investors is gearing up for a 20-mile hike of the Presidential Traverse in the White Mountains of New Hampshire Sept. 11-14. The team goal is on pace to hit the goal of $800,000 to fight poverty in Boston, San Francisco, San Diego and Philadelphia. SVB, SmartLabs and Goodwin are among the top sponsors.
Highlights of the Timmerman Traverse, 2021
This is a phenomenal way to give back to the wider communities where biotech operates, and there will be opportunities to mix and mingle with the stellar members of this team. To see who’s hiking and support someone you know, click here. For sponsorship opportunities, see Emily Stanford at LSC emily@lifesciencecares.org.
Kilimanjaro 2023. One way I seek to build community in biotech is by creating diverse and tight-knit teams for my philanthropic expeditions.
I’m now recruiting a team of 27 people to join me on the next Kilimanjaro Climb to Fight Cancer. It’s scheduled for Feb. 9-20, 2023. The goal is to raise $1.5 million for cancer research. Please let me know if you, or someone you know, might be a good candidate for this group expedition to the highest peak in Africa. I’m seeking biotech entrepreneurs and investors who have a thirst for adventure, are physically fit, and willing to raise a minimum of $50,000 for cancer research.
To request an invitation: luke@timmermanreport.com.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
David Shaywitz
It only took a pandemic, but well-being has at last emphatically arrived in the corporate world.
Before the pandemic, wellness was often viewed as just another item on the HR benefit menu. Some companies offered limited reimbursement for fitness classes, or subsidized access to select health and wellness apps.
But in the turbulent wake of COVID-19, well-being is increasingly viewed as worthy of C-suite attention, and recognized as critical to company success.
As we collectively navigate our way towards improved well-being, we might consider two recent books that address the topic through two very different lenses.
Several years ago, Seth Stephens-Davidowitz, a data scientist and New York Times columnist, wrote about what data science had to say about human behavior. His book, Everybody Lies, was a revealing and engaging read, as I discussed here.
Seth Stephens-Davidowitz, data scientist, author, and New York Times columnist.
Stephens-Davidowitz has now returned with Don’t Trust Your Gut, a delightful, quirky romp through the data science literature on self-improvement.
Why self-improvement? Predictably, because Stephens-Davidowitz followed the data. He analyzed the reaction to his last book and noticed that readers told him they were most interested in the use of analytics to help others, particularly those less fortunate.
Yet when he looked at the actual data (the passages underlined most in the Kindle version), he discovered readers were, in fact, most interested in themselves, and he aims to oblige. (This distinction between stated and revealed preference is a key theme of his last book, and a frequent topic of this column – eg here — since the ability to discern the difference is often critical to entrepreneurial success.)
Stephens-Davidowitz starts by demonstrating how data science can be used to study human relationships by mining dating sites, which turn out to contain a treasure trove of information. Some of the insights prove modestly surprising: for example, among those individuals seeking heterosexual relationships, tall men were especially prized, but tall women were not.
At the same time, Stephens-Davidowitz writes, data scientists – despite extensive effort — have been utterly unable to predict whether two people will actually be happy in a relationship. The top lesson drawn by a researcher who exhaustively examined a dataset of 11,196 couples? “How unpredictable relationships seem to be.”
Ever the sabremetrician, Stephens-Davidowitz points out what he sees as the grand arbitrage opportunity here: since “good romantic partners are difficult to predict with data” but “desired romantic partners are easy to predict with data,” the winning move, he suggests, he to pursue the “Youkilis of love” (Kevin Youkilis was a famously undervalued baseball player because he didn’t look the part). He advises daters to “focus more of their attention on targets who the rest of the data market ignores, even though they are just as likely to be great romantic partners.”
Having shown us the approach, Stephens-Davidowitz applies this data-driven methodology to a range of subjects, from the impact of parenting (which may matter less than we think) to the role of genetics in sports success (genes are evidently far more determinative in basketball and track-and-field than in skiing and weightlifting) to activities that make us happier than we think (going to museums, exercising, gardening) to the importance for artists to aggressively maximize their opportunity to be discovered, rather than venturing forth timidly and in effect “pre-rejecting” themselves.
Perhaps my favorite section focused on entrepreneurship, and the lessons learned from researchers analyzing massive datasets. For instance, a team analyzing every business created in the United States between 2007 and 2014 found that of the 2.7 million entrepreneurs represented, the average age of founders is about 42. They also learned that older people “succeed at creating highly profitable businesses more often than their younger peers” — a trend that continues “at least until the age of sixty.” In other words, the young tech geniuses the media often focus on are not representative – even in technology, where the average age of a founder of a highly profitable company was 42.3 years old.
Similarly, while we’re often told that industry outsiders have the independence to develop the most profitable startups, Stephens-Davidowitz cites research demonstrating that in reality,
“There is an enormous ‘Insider’s Advantage’ in entrepreneurship. Entrepreneurs are roughly twice as likely to build an extremely successful company if they previously worked in the field in which they started the company. The advantage gets larger the more directly the previous experience is related to the business.”
(Readers will note parallels between some of the findings Stephen-Davidowitz reports and those uncovered by VC and author Ali Tamaseb in Super Founders, as I’ve discussed.)
While not the definitive account of any of the topics it touches on, Don’t Trust Your Gut demonstrates the unreasonable power of empirical evaluation of extremely large datasets, including in the context of wellness, and reminds us of the many ways we can be deceived by selective sampling.
Johann Hari is a journalist and writer who, like many of us, was struck by the outsized role technology – social media in particular – plays in our lives. In Stolen Focus, he explores the origins and dangers of this contemporary menace.
Hari starts with an anecdote – familiar to most parents – of watching his teenage godson Adam become increasing absorbed into the social media on his smartphone, to the point where he seemed to enter an almost dissociated state of scrolling and staring blankly at the screen. Eager to engage Adam in a real world experience – and because Adam had once been captivated by all things Elvis – Hari took his godson to Graceland, having secured from Adam, in advance, the promise of abstaining from technology once they got there.
Johann Hari, author, “Stolen Focus.”
Yet, the moment they arrive at Graceland, Hari and Adam are each handed….an iPad and headphones, for a self-guided tour of the mansion.
But the most striking part, as Hari describes it, was when they arrived outside the famous “jungle room” (supposedly Elvis’s favorite place in the compound), Hari notices the middle-aged couple in front of him captivated by the ability to switch between images of the left and right side of the jungle room simply by flicking their fingers from side to side on the iPad.
Flabbergasted, Hari points out to the couple that they can achieve the same effect by looking up from their iPads and looking at the actual room in front of them. We’ve become so entranced by our technology, he realizes, that we instinctively are drawn to the image of the jungle room even when the room itself exists before us.
Hari explains how we’ve become so addicted to our screens: the business model for tech companies like Google and Facebook means that their revenue is maximized by increasing our time and engagement on their platforms. Consequently, he says, the smartest engineers in the world leverage the most powerful AI and savviest user experience expertise to escalate our addiction.
Hari also discusses why the constant beckoning of our smartphone apps is so destructive: it steals our ability to deeply and consistently focus. (Here, he echoes themes from Cal Newport’s Deep Work, which inspired me to quit Twitter in 2019.) It’s not that responding to a notification itself is so time consuming, but rather that it takes a shockingly long time – more than 20 minutes, he reports – to regain focus afterwards. We tend to be closer to our best selves when we can deeply focus, and when we can’t, everything is worse. We may pride ourselves on our ability to multitask, he says, but in splitting our attention, we’re sabotaging ourselves, our work, and our happiness.
Hari writes that workplaces interested in worker well-being might do well to rely less on advocating wellness practices like meditation (which Hari says he personally enjoys and values), and more on creating intrinsically better environments for workers. Essentially, he argues that instead of putting a band aid on the problem, companies should seek to provide greater opportunity to focus at work (i.e. fewer gratuitous distractions) and to relax at home (i.e. protected time and “right to disconnect” – now a legal protection in France).
While the book seemed to lose its way and go off the rails about halfway through (I couldn’t make it through to the end), the idea that we’re physically addicted – by design – to our apps is a crucial point worth amplifying.
When I thought about how I was interacting with my phone, and the number of times I reflexively checked it, I resolved to try to establish some new habits.
For one, I re-quit Twitter (I had started up again in the context of Omicron) – with immediate satisfaction and minimal regret (sorry, Elon). I also now stow my phone when I drive, so I’m not even tempted to interact with it. This took a little adjusting, but I really appreciate this new habit as well.
I like the idea of seeing wellness through the lens of discrete, modifiable habits, as Hari alludes to and a number of authors have suggested; this seems like a pragmatic way of productively changing behavior. The same science that has informed the development of smartphone apps that steal our focus and detract from our happiness can also be used to instantiate healthy habits.
In fact, it’s exactly the same science: many engineers designing today’s technology trained in B.J. Fogg’s legendary “persuasive technology lab” at Stanford (now known as the “behavioral design lab”). Fogg is also the author of the Tiny Habits, aimed at helping us use small changes to drive meaningful behavior change. (Good Habits Bad Habits, by USC professor Wendy Wood, is also highly recommended.)
Of course, not all apps are harmful, and some can even be a force for good. For example, I’ve been pleasantly surprised by impact of a corporate wellness initiative that simply involves signing up for an app that collects your daily steps from whatever device you select. You sign up in teams of four, and strive to average 7500 steps/day. Participants are encouraged to share photos of their walks with other participants through the app.
Silly as it sounds, I’ve really appreciated this experience – it’s been lovely seeing where my colleagues are walking, and I find I’m getting out much more than I used to; I’m especially likely now to use short blocks of downtime as an opportunity to get in some additional steps.
Finally, I wanted to highlight a particularly positive recent experience I had attending an external two-day retreat with an exceptional group of biomedical leaders, innovators, entrepreneurs, and investors. The opportunity to engage with other colleagues in a relaxed setting — on hikes and over meals, in person and without masks — was extraordinary.
The experience reminded me how much we’ve missed this human connection over the last two years. It also emphasized how elevating and essential physical exercise and human connection are – not just for our health, but also for our happiness.
Today’s guest on The Long Run is Chris Gibson.
Chris the co-founder and CEO of Salt Lake City-based Recursion.
Chris Gibson, co-founder and CEO, Recursion
The company was founded in 2013 when Chris was working in the lab of Dean Li at the University of Utah.
The idea at Recursion is to do what it calls “industrialized drug discovery.” It combines some of the common tools of drug discovery – CRISPR, synthetic biology, automated lab tools, high-powered computing, and Artificial Intelligence / Machine Learning to hopefully churn out more drug candidates faster, and with a higher probability of success. The company has a long history of phenotypic screening, and leans on that expertise, combined with the new tools, to create maps of biology that are supposed to help scientists predict how any two tested genes or molecules might interact with each other.
Recursion raised $436 million in an IPO in April 2021, which looks pretty well-timed based on the state of the biotech stock market today. It has a couple of partnerships – with Bayer and with Roche / Genentech. The Bayer deal is focused on treatments for fibrotic diseases, while the Roche / Genentech deal includes oncology and neuroscience indications.
Chris is a next-generation founder and industry leader who isn’t afraid to buck convention, and has a lot of interesting things to say about technology, about company building, and about creating a thriving ecosystem outside of the main industry hubs.
Now before we get started, a word from the sponsors of The Long Run.
Calgary is home to more than 120 life sciences companies, from emerging startups to established firms. With this critical mass of research, technical talent and expertise, the city is an active hub for life sciences innovation.
Technologies homegrown in Calgary are changing the face of healthcare. Syantra is revolutionizing breast cancer detection using artificial intelligence-derived algorithms. NanoTess is harnessing the power of nanotechnology to tackle chronic wounds and skin conditions. And this is only the beginning. Calgary’s life sciences sector is projected to spend $428 million on digital transformation by 2024.
If you’re a bright mind or bright company solving global health challenges, Calgary is the place for you.
Take a closer look at why at calgarylifesciences.com
Alpenglow Biosciences sheds new light on pharmaceuticals with AI-powered, 3D spatial biology. Pathology is an essential component of drug development, yet it is stuck in archaic times by looking through 2D slides. Alpenglow has developed an end-to-end drug development solution with proprietary 3D imaging, cloud processing, and AI analysis to digitize entire 3D tissues, providing 250 times more data and deeper insights. Learn how Alpenglow can illuminate your path to breakthrough results at alpenglowbiosciences.com.
Now, please join me and Chris Gibson on The Long Run.
