Clinical trialists are always trying to come up with catchy study titles. I’m not a big fan of these acronyms, and try to avoid using them. They’re often forgettable.

But this week I saw one that stood out as especially meaningful. Yale University researchers are running a new Long COVID study. It’s called – LISTEN.

It stands for Listen to Immune, Symptom and Treatment Experiences Now.

There’s certainly interest in what’s happening biologically with so many people — potentially millions — who are reporting some symptoms of Long COVID. Lead investigators Akiko Iwasaki and Harlan Krumholz appear to have a solid plan to collect data on the many potential contributing factors to this mysterious ailment — demographic, clinical, social, and environmental factors associated with health status.

They’re collecting blood and saliva samples, and incorporating use of a health app called Kindred that is supposed to connect patients to each other’s experiences, to their own data, and to other potentially helpful studies.

This study, and others like it, could help point the way toward more effective prevention and treatment.

Equally important, though, is the sentiment around rebuilding public trust in science. We need to do a better job of listening to accomplish this goal.

It’s important that the research participants feel like they’re being listened to, that they’re being treated respectfully, and that they are part of helping solve the medical mystery. Studies like this point the way toward how to conduct rigorous scientific investigations with the kind of respectful, two-way street of engagement that participants want and deserve. We need studies like this to encourage more people to participate, and then to foster more confidence in the knowledge that results.

Many millions of people don’t trust what the scientific community has learned the past couple years about the pandemic. Especially during moments of maximum uncertainty, way too many people have turned to charlatans and other peddlers of misinformation for answers. Many people are vulnerable to these appeals because they feel like outsiders, like they’re not being listened to by The Establishment.

Some of this feeling is understandable, especially among people suffering from Long COVID, who haven’t been taken seriously enough by policymakers and the scientific community.

A little more careful listening could go a long way.

We might learn about the virus. We might also learn a bit about how to repair some of that torn social fabric. We need to do both of these things to get the scientific enterprise on a more sustainable footing, and to be better prepared for the next big thing.

 

This Week in Drug Pricing

How much do hospitals mark up the prices of cancer drugs they administer via infusion? How much do these prices vary from place to place? How often do they follow new federal rules on price transparency? Most people have no idea, and they would be shocked to learn the answers. Read about the eye-popping markups – between 120 percent and 630 percent — in in JAMA Internal Medicine.

Financings

Boston-based Sionna Therapeutics said it raised a $111 million Series B financing led by OrbiMed. The company is working on small molecules to fully restore function of the CFTR protein in cystic fibrosis patients. RA Capital, Atlas Venture and the CF Foundation also participated in the financing. Read RA Capital partner Josh Resnick’s piece on the opportunity to go beyond what Vertex has done in the past decade to transform the treatment of CF.

Somerville, Mass.-based Tessera Therapeutics said it raised more than $300 million in a Series C financing to advance its gene writing technology for drug discovery. Abu Dhabi Investment Authority; Alaska Permanent Fund Corporation; Altitude Life Science Ventures; ARTIS Ventures; Cormorant Asset Management and Tessera’s founder, Flagship Pioneering were among the investors.

Cambridge, Mass.-based Satellite Bio came out of stealth, saying it has raised $110 million in seed and Series A funds to make programmable cell therapies. aMoon Growth led the A round. Other investors include seed co-lead Lightspeed, aMoon Velocity, Polaris Partners and Polaris Innovation Fund. New Series A investors included Section 32, Catalio Capital Management and Waterman Ventures.

San Francisco-based Unlearn raised $50 million in a Series B financing. The company is working on a machine learning platform to help companies run randomized controlled trials with a higher probability of success, and with enrolling fewer patients. Insight Partners led with participation from new investor Radical Ventures, as well as all of the company’s existing investors including 8VC, DCVC, DCVC Bio and Mubadala Capital Ventures.

Denver-based Pathware raised $7 million to develop hardware and software for digital pathology, so it can go from the central lab to the point of care. UnityPoint Health Ventures and Level Eight Ventures co-led.

Emeryville, Calif.-based Octant said it raised $80 million in a Series B financing led by Catalio Capital Management. It’s using synthetic biology and chemistry to develop precision medicines. The company said it also established a Deep Mutational Scanning partnership with Bristol Myers Squibb, and hired Dean “Rick” Artis as its first Chief Scientific Officer. Feng Zhang also joined the Scientific Advisory Board.

Framingham, Mass.-based Alzheon, an Alzheimer’s drug developer, raised $50 million in a Series D financing.

Personnel File

Cambridge, Mass.-based eGenesis, an organ transplantation company, promoted Michael Curtis to CEO. He’s been president of R&D since 2020. Outgoing CEO Paul Sekhri will remain on the board of directors.

South San Francisco-based Twist Bioscience, the DNA synthesis company, promoted Tracey Mullen to senior vice president of operations, and Nimisha Srivastava to senior vice president of R&D. Patrick Weiss is stepping down as chief operating officer.

Cambridge, Mass.-based Evelo Biosciences, the developer of single-strain microbial therapies that can be given orally for inflammatory diseases, said it added John Maraganore and Tassos Gianakakos as strategic advisors. Maraganore is the former CEO of Alnylam Pharmaceuticals and Gianakakos is the former CEO of Myokardia.

Somerville, Mass.-based Finch Therapeutics, a developer of microbiome-based therapies, said it’s cutting its workforce by 20 percent.

Deals

Tarrytown, NY-based Regeneron Pharmaceuticals agreed to acquire Checkmate Pharmaceuticals, a cancer drug developer, for $10.50 a share, or $250 million. Checkmate is the developer of vidutolimod, a CpG-A oligodeoxynucleotide Toll-like receptor 9 (TLR9) agonist delivered in a virus-like particle to stimulate the innate immune system against cancer. Some market observers hoped that the acquisition might lift the biotech stock markets out of the doldrums, providing hope to the roughly 150 public companies that are now considered by investors to be essentially worthless. (See @bradloncar tweet).

New York-based Terran Biosciences said it licensed a pair of late-stage programs for Sanofi, and plans to develop them for neurological and psychiatric diseases. The assets weren’t disclosed, and neither were the financial terms.

AbbVie terminated its partnership with Sweden-based BioArctic. The companies had been working together since 2016 on antibodies directed against alpha-synuclein for Parkinson’s disease.

Waltham, Mass.-based Dragonfly Therapeutics said it expanded its research collaboration with AbbVie to work on immune-mediated diseases. AbbVie is getting the option to license multiple new candidates that use Dragonfly’s tri-specific NK cell engaging technology. Terms weren’t disclosed.

Data That Mattered

Amgen said it passed a Phase III trial with a biosimilar version of J&J’s ustekinumab (Stelara), an IL-23 antagonist for moderate to severe plaque psoriasis.

Moderna said its bivalent COVID vaccine candidate, designed to stimulate the immune system against mutations found in the Beta variant of concern, was able to elicit higher levels of neutralizing antibodies than the original vaccine formulation. The bivalent vaccine candidate appeared superior to the original against the Beta, Delta and Omicron variants one month after administration, the company said.

San Diego-based Arcturus Therapeutics said its self-amplifying mRNA vaccine candidate against COVID-19 delivered 55 percent vaccine efficacy against symptomatic COVID, and 95 percent efficacy against severe disease or death, in a study of 19,000 adults in Vietnam. Adverse events were similar between the vaccine and placebo groups, the company said. Arcturus said it’s planning a pivotal study of the vaccine candidate as a booster.

Ponte Vedra, Florida-based Orasis Pharmaceuticals said it met the primary and secondary endpoints of a Phase III trial that evaluated its eye drop for presbyopia – the loss of the ability to see items up close, a natural part of aging. The company said it plans to submit a New Drug Application to the FDA in the second half of 2022.

Science Policy

• Better Ventilation Would Create a Healthier Workplace, but Companies Have to Invest. NPR / Kaiser Health News. Apr. 19. (Liz Szabo)
• Now’s Not the Time to Dispense with COVID-19 Precautions. Washington Post. Apr. 20. (Lucky Tran and Oni Blackstock)

Strategy

• The Case for American Seriousness. Common Sense. Apr. 18. (Katherine Boyle)
• Nektar’s IL-2 Legacy: The Two Sides of Failure. RApport. Apr. 20. (Jake Simson & Peter Kolchinsky)

Annals of Manufacturing

Bothell, Wash.-based Seagen, the developer of antibody-drug conjugates for cancer, officially announced its plans to open a 270,000-square foot manufacturing facility in Everett, Wash. The company said the new facility will give it “greater control and flexibility over the production of its medicines to treat cancer.” I consider this to be a shrewd move for several reasons. (See my Frontpoints column, Feb. 2022, “Biotech’s Future Off the Beaten Path.”)

Novocure said it’s breaking ground on a new facility in Portsmouth, NH, which will house “a training and development center where partners from around the world can come to learn about Novocure’s Tumor Treating Fields (TTFields) cancer therapy.” NH Gov. Chris Sununu attended the ceremony.

Santa Monica, Calif.-based Kite Pharma received FDA clearance for a new cell therapy manufacturing facility in Frederick, Maryland. The company said it now has facilities in southern California, Amsterdam and Maryland. Kite said in a statement it has “the largest, dedicated in-house cell therapy manufacturing network in the world, spanning process development, vector manufacturing, clinical trial production and commercial product manufacturing.”

Science

• The Future of Early Cancer Detection. Nature Medicine. Apr. 19. (Rebecca Fitzgerald et al University of Cambridge)
• Somatic mutation rates scale with lifespan across mammals. Nature. Apr. 13. (Alex Cagan et al Wellcome Sanger Institute)
• The Challenge of Genetic Variants of Uncertain Clinical Significance. Annals of Internal Medicine. Apr. 19. (Wylie Burke et al University of Washington)
• Development of a clinical polygenic risk score assay and reporting workflow. Nature Medicine. Apr. 18. (Matthew Lebo et al Mass General Brigham Personalized Medicine)

Science of SARS-CoV-2

• The Evolution and Biology of SARS-CoV-2 Variants. Cold Spring Harbor Perspectives in Medicine. Apr. 20. (Amalio Telenti of VIR Biotechnology, Emma B. Hodcroft of University of Bern, Switzerland and David L. Robertson of MRC-University of Glasgow Center for Virus Research)
• Intramuscular AZD442 (Tixagevimab–Cilgavimab) for Prevention of Covid-19. NEJM. Apr. 20. (Myron Levin et al Provent Study Group). For people at increased risk of an inadequate response to vaccination or increased risk of exposure, AstraZeneca’s antibody cocktail (Evusheld) delivered a 77 percent relative risk reduction of coming down with symptomatic COVID, compared with placebo. Patients were followed six months. (AZ press release)
• Increased Memory B Cell Potency and Breadth After a SARS-CoV-2 mRNA Boost. Nature. Apr. 21. (Paul Bieniasz & Michel Nussenzweig et al Rockefeller University)
• Vaccine effectiveness against SARS-CoV-2 infection and COVID-19-related hospitalization with the Alpha, Delta and Omicron SARS-CoV-2 variants: a nationwide Danish cohort study. MedRxiv. Apr. 20. (Christian Hansen et al Statens Serum Institut)
• Admissions to a large tertiary care hospital and Omicron BA.1 and BA.2 SARS-CoV-2 PCR positivity: primary, contributing, or incidental COVID-19. MedRxiv. Apr. 18. (Juliette Severin et al University Medical Center Rotterdam, Netherlands)
• Hospitalizations of Children Aged 5–11 Years with Laboratory-Confirmed COVID-19 — COVID-NET, 14 States, March 2020–February 2022. CDC Morbidity and Mortality Weekly Report. Apr. 19. (Dallas Shi et al CDC)

Science Features

• Scientists Find No Benefit to Time-Restricted Eating. NYT. Apr. 20. (Gina Kolata)

A Word on Climb to Fight Cancer

Many of you saw social media announcements about my latest Climb to Fight Cancer campaign for the Fred Hutch Cancer Center.

I want to say thanks to everyone who contributed to this campaign. It was a huge success.

The 18-person team of biotech leaders reached Everest Base Camp in Nepal (elev. 17,600 feet) on Apr. 4. We raised $1.3 million for cancer research.

We enjoyed spectacular scenery in the world’s highest mountain range, the Himalayas.

We made new friendships on the trails.

We reveled in the culture of the Sherpa, the indigenous people of the Khumbu Valley famous for their strength at high altitude, and their generous spirit.

These trips are clearly resonating with many members of the biotech community. It was evident on the Kilimanjaro climb of 2019, on the Timmerman Traverse for Life Science Cares in 2021, and again on the Everest Base Camp trek of 2022.

I’m planning more expeditions. These trips mobilize the biotech community around good causes. I believe there’s tremendous potential for good work in the biotech community. I will continue finding ways to harness it.

For today, enjoy a few photos from the Everest Base Camp trek of 2022. Click on images to view at full size.

If you are interested in participating in a future expedition, or your company is interested in sponsoring one of these campaigns for cancer research or poverty relief, see me. 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.

Climbing Mt. Everest changed my life. The world’s highest mountain required digging deep — physically, mentally, emotionally.

That was four years ago.

That original climb was a success, raising $340,000 for cancer research at the Fred Hutchinson Cancer Research Center. But what came next meant more. The climb opened my mind to new ways of making a contribution, in addition to biotech journalism.

The expeditions that followed have become bigger. More impactful and more enduring.

I’m returning to the Himalayas this month. This time, it will be with a team of 18 people I recruited to trek to Everest Base Camp, elevation 17,600 feet. Before hitting the trails of Nepal, we have worked together to raise more than $1 million for cancer research at the Fred Hutch. We could exceed $1.2 million before it’s all done.

I’m proud of this team and what it has accomplished for science.

It’s been a long and hard road the past two years for everyone. I can hardly imagine what it has been like for my Sherpa friends.

I will cry tears of joy and gratitude when we meet in Kathmandu. Hugs are on the first team meeting agenda.

The people of Nepal are poor in material terms, yet so generous and grateful in spirit. Going back, and giving back, is one way to make a positive contribution.

We will be meeting with researchers from the Kathmandu Cancer Center. We will be learning from people in the villages. We will enjoy the mountain scenery, savor warm cups of tea, and form meaningful relationships on the trail. More good things are bound to emerge.

Expect things to be quiet at Timmerman Report the next couple weeks. I plan to be mostly off the grid, with a few brief team updates from Nepal. By mid-April, I’ll be back.

Thanks to all the TR readers. Your outpouring of comments about the 7^th anniversary of TR were heartwarming. Many of you have contributed to my campaigns for Fred Hutch and Life Science Cares. A few of you have participated. Some of you may want to participate someday. There will be time to discuss all of that later.

Tumultuous things are happening in the world. But one thing I learned four years ago is that in difficult moments, people are capable of more than they realize. Sometimes a kind word, or a sincere expression of confidence, is all it takes to provide people with the spark.

When we commit to a cause larger than ourselves, and when we show faith in others, amazing things can result.

Let’s keep lifting up the people around us.

 

Data That Mattered

Gilead Sciences released underwhelming top-line Phase III clinical results for sacituzumab govitecan-hziy (Trodelvy), in metastatic HER2-negative breast cancer patients. The company issued a mealy-mouthed and detail-light press release which said it hit the primary endpoint of Progression-Free Survival, and that the study was powered to show a 30 percent reduction on PFS, but that only a trend toward benefit on Overall Survival was seen. These were sick patients previously treated with CDK4/6 inhibitors, but still. That’s not great news, especially for a key asset obtained in a $21 billion acquisition of Immunomedics. Baird analyst Brian Skorney summed it up well: “Although not a failure, we think this top line release indicates that the opportunity in HR+/HER2- breast cancer is likely to be constrained, as the benefit seen may not be enough to drive utilization vs. the cheap generic chemos Trodelvy was compared to in this study.” Gilead followed up its tepid clinical results by cutting 114 jobs in the former Immunomedics unit it acquired, and reallocating some resources for manufacturing in Southern California. The series of missteps at Gilead has been jarring of late, and has prompted some analysts to wonder about the state of the company under CEO Daniel O’Day. See Matt Herper’s take in STAT.

France-based Valneva, a vaccine developer, reported positive Phase III results with a single-shot, live-attenuated vaccine candidate against chikungunya virus. The data from a study of more than 4,000 subjects showed protective levels of neutralizing antibodies were observed in 98.9 percent of subjects on the vaccine after one month, and 96.3 percent of vaccine recipients after six months. The company said it plans to seek FDA approval before the end of June. If approved, this would be the first vaccine against this mosquito-borne illness that sickens millions of people around the world.

Deals

San Diego-based Illumina, the market leader in DNA sequencing, continues to struggle with antitrust regulators. This week, Reuters, citing people familiar with the matter, reported that European Union regulators are still unsatisfied and yet to approve of the company’s plan to acquire Grail for $8 billion. The acquisition would allow Illumina to expand globally into the emerging market of early cancer detection powered by DNA sequencing technology.

Cambridge, Mass.-based Voyager Therapeutics pocketed $54 million upfront from Novartis in an option agreement. The deal gives Novartis the option to license novel AAV capsids to use in gene therapies against three targets of the Central Nervous System, and potentially two more targets in the future.

San Francisco-based Cellevolve struck a partnership with Seattle Children’s Therapeutics, a venture from Seattle Children’s Hospital.

Cellevolve will bring its expertise in development and commercial strategy for cell therapies to the table, while Seattle Children’s Therapeutics brings its GMP CAR-T cell therapy manufacturing capability and lentiviral vector manufacturing capability for cell therapies. The organizations will collaborate on three pediatric cell therapy programs for malignancies of the Central Nervous System. Michael Jensen, a Seattle Children’s researcher and co-founder of Juno Therapeutics and Umoja Biopharma, has agreed to serve as chair of Cellevolve’s Scientific Advisory Board. (Listen to Cellevolve founder and CEO Derrell Porter on The Long Run podcast, Feb. 2022)

Our Shared Humanity

• ‘Something is off here’: Black biotech entrepreneurs still struggle for funding as the industry pledges to diversify. STAT. Mar. 4. (Jonathan Wosen)
• Orcas doctor to climb to Mount Everest Base Camp in campaign to raise $1m for cancer research. The Orcas Island Sounder. Mar. 9. (Colleen Smith)
• Unclouded Judgment. NEJM. Mar. 9. (Lisa Rosenbaum)

Science Features

• The Coronavirus’s Next Move. The Atlantic. Mar. 9. (Katherine Wu)
• Vaccinating Kids Has Never Been Easy. The Atlantic. Mar. 10. (Sarah Zhang)

Science

• Tumor-Infiltrating T cells. A Portrait. NEJM. Mar. 10. (David Braun and Catherine Wu)
• Distinct biological ages of organs and systems identified from a multi-omics study. Cell. Mar. 8. (Chao Nie et al BGI Shenzhen)

Science of SARS-CoV-2

• Whole genome sequencing reveals host factors underlying critical Covid-19. Nature. Mar. 7. (Mark Caulfield, Kenneth Baillie et al Genomics England)
• The changing epidemiology of SARS-CoV-2. Science. Mar. 10. (Natalie Dean et al Emory University)
• The immunology and immunopathology of COVID-19. Science. Mar. 10. (Akiko Iwasaki et al Yale University).
• Broad neutralization of SARS-CoV-2 variants by an inhalable bispecific single-domain antibody. Cell. Mar. 9. (Cheng Li et al Shanghai Institute of Infectious Disease)
• Resistance mutations after sotrovimab use. NEJM. Mar. 9. (Rebecca Rocket et al University of Sydney, Australia)
• Immunogenicity and Reactogenicity of Vaccine Boosters after Ad26.COV2.S Priming. NEJM. Mar. 10. (SWITCH Research Group)
• Boosters protect against SARS-CoV-2 infections in young adults during an Omicron-predominant period. MedRxiv. Mar. 9. (Peter Frazier et al Cornell University)

Science Policy

• We need an early warning system to prevent the next COVID surge. The Hill. Mar. 5. (Lynn Goldman and Emily Smith of George Washington University’s Milken Institute School of Public Health)
• A roadmap for living with COVID. (Trevor Bedford, Lu Borio, Rick Bright et al).
• Improving Ventilation Will Stop More Than COVID-19. Bloomberg Opinion. Mar. 8. (Lisa Jarvis)
• DeSantis aide bucks medical consensus that healthy children should get COVID vaccine. ABC News. Mar. 7. (Arielle Mitropoulos)
• “Back to normal” is not enough. Science. Mar. 10. (Christina Pagel)
• Why Test to Treat is Imperative Now. Medscape. Mar. 9. (Eric Topol, Abraham Verghese & Michael Mina)
• Taking stock as America moves into a new phase of the pandemic. The Economist. Mar. 12.

Vaccines

Moderna announced its near-term global health strategy. It includes a commitment to advance vaccine candidates against 15 pathogens considered to be risks to global health, as determined by the World Health Organization (WHO) and Coalition for Epidemic Preparedness Innovations (CEPI). The company reached an agreement with the US government to build its first mRNA vaccine factory on the African continent in Kenya. It agreed to an open platform for researchers to gain access to its technology against emerging or neglected diseases. And the company agreed not to enforce its IP in 92 low to middle income countries.

Personnel File

Morphosys closed the Cambridge, Mass.-based R&D center it obtained through the acquisition of Constellation Pharmaceuticals. It’s consolidating work in Germany. (Endpoints News)

Cambridge, Mass.-based Alnylam Pharmaceuticals promoted Kevin Fitzgerald from senior vice president and chief scientific officer to executive vice president and chief scientific officer of research. He’s been with the RNA interference drug developer for 18 years.

Seattle-based Adaptive Biotechnologies hired Tycho Peterson as chief financial officer. He’s a former JP Morgan analyst. The company said it’s also cutting 12 percent of its workforce.

Waltham, Mass.-based Affinia Therapeutics, a developer of AAV gene therapies, said it’s added Diana Brainard to its board of directors. She’s the CEO of AlloVir.

Cambridge, Mass.-based Bluebird Bio said in a regulatory filing that Gina Consylman, the chief financial officer, is resigning on Apr. 3. Jason Cole, the chief business officer, will serve as the company’s primary financial officer. A few days earlier, in its 10-K, the company issued a warning to investors that it has doubt about its ability to continue as a “going concern” as it runs low on cash.

Financings

San Diego and Research Triangle Park, NC-based Creyon Bio came out of stealth with a $40 million Series A financing. It’s working on using AI to develop custom oligonucleotide therapies. DCVC Bio and Lux Capital co-led.

Concord, Mass.-based Adiso Therapeutics, backed by Morningside Ventures, announced its debut. The 25-person company is working on small molecules and single-strain live biotherapeutics for the treatment of ulcerative colitis and C. difficile infections.

Venrock led a $20 million Series B financing in San Francisco-based SmithRx, an aspiring new pharmacy benefits manager (PBM). The idea, according to Axios, is to use algorithms to help employers find “lower-priced medications based on pharmacy distribution, clinical management, rebates and special programs.”

Vancouver, BC-based Solve FSHD was founded with $100 million from Lululemon founder Chip Wilson, who suffers from facioscapulohumeral muscular dystrophy type 2.

Mountain View, Calif.-based DNAnexus raised $200 million in a deal led by Blackstone Growth to support its platform for using multi-omics data to advance precision medicine. Terms of the deal weren’t disclosed, even whether it’s equity, debt, or some combination of the two. Northpond Ventures, GV, Perceptive Advisors, Innovatus Capital Partners, and Foresite Capital joined the round.

Regulatory Action

Seattle-based Alpine Immune Sciences said the FDA placed a partial clinical hold after a patient died in the NEON-2 clinical trial that is evaluating its drug candidate davoceticept in combination with Merck’s pembrolizumab (Keytruda) for advanced cancers. The company said the death was attributed to cardiogenic shock. The treating physicians thought it was “likely related to immune-mediated myocarditis, or possibly infection,” Alpine said. The partial hold doesn’t affect Alpine’s NEON-1 trial, which evaluates its drug candidate as a monotherapy.   

Tweetworthy

Good news.

Not long ago, biotech leaders steered clear of commenting on the issues of the day. Politics was limited to certain vested interests like drug pricing, science funding, and FDA regulation.

Then came COVID-19 and the racial justice reckoning.

Staring these terrible things in the face, people began to think more about their roles in the workplace, and in the community. The aperture widened. We are redefining what it means to be a scientific citizen.

Now we have a Russian dictator starting a war against a sovereign and democratic Ukraine. The Russian leader is issuing threats reminiscent of the old Soviet empire. He has some of the world’s most powerful weapons – nuclear, cyber, and psychological infowar tools to destabilize Western democracies from within. He’s ruthless. Political opponents or journalists who dare to report uncomfortable truths get poisoned, jailed, or killed.

Staying neutral isn’t an option. This is a serious threat to the world order.

Citizens need to pay attention, and think carefully about what’s happening, how it affects us, and what we can do.

In a globalized, hyper-networked world, it’s not just all about Putin and Zelensky and Biden and Macron. Millions of people making millions of decisions each day can each play a part. Even if it’s as small and simple as choosing what to tweet or not tweet, what we spend money on and what we don’t, we are each contributing.

It’s heartening to see biotech leadership that understands. They don’t want to be part of a generation that said nothing and did nothing when it counts. They don’t want to appease, or worse, help support the rise of a monstrous regime.

Meg Alexander and Jeremy Levin of Ovid Therapeutics, Paul Hastings of Nkarta Therapeutics, Peter Kolchinsky of RA Capital Management, Ted Love of Global Blood Therapeutics, and John Maraganore, the former CEO of Alnylam Pharmaceuticals, co-authored a forceful piece about the Russian invasion on Feb. 26 on Medium.

The open letter called for business leaders to disengage from Russian interests in a few clear ways:

• Cease investment in Russian companies and new investment within the borders of Russia
• Reject investment from Russian funds
• Halt collaboration or service agreements with Russian companies
• Except for food and medicines, halt trade in goods with Russian companies

All of these actions should remain in effect, the authors wrote, until “the restoration of peace and democracy in a sovereign Ukraine.”

One week later, more than 680 people have signed on. Many more are spreading the word through social media. More people are feeling the pressure to use their voices, use their power, to stand up for people halfway around the world who are facing terrible attacks.

By now, many in biotech have heard of Enamine, the contract research organization in Ukraine that is a trusted partner to many companies. It’s already close to home.

If Mr. Putin is able to extend his sphere of influence further into Eastern Europe, we’ll hear about many more trusted partners who suddenly find themselves no longer so free and secure.

Government leaders have their role to play. But so do we.

We aren’t helpless and voiceless. One person alone may not make much of a difference, but when we activate our networks, when we make business decisions on moral grounds and shut off the flow of money and goods, those actions send ripple effects.

The US is the beating heart of biotech, but we don’t do this work alone. Great science bubbles up from all around the world. Contract development and manufacturing firms have something to contribute from all corners of the globe. Clinical research sites are almost everywhere. Patients are everywhere.

If we’ve learned one thing the past couple years, it should be that the biopharma industry is a strategic asset to the US and is a beacon of hope to the world. This industry doesn’t thrive here by accident — it’s here in large part because of our historic systems of free enterprise and democracy. Because of our laws and spirit and traditions.

The world counts on the US to hold it together, to be a serious country, to maintain our resilience. We need all that to deliver groundbreaking science and lifesaving products.

America hasn’t always lived up to its founding ideals. But we continually strive to form a more perfect union. We can course correct, regain our balance. We still have the rule of law, the Bill of Rights, free elections and freedom of speech.

We should remember that it’s our job to use these gifts wisely, and to pass them on to the next generation. When we operate within the laws and norms of our system, we can be at our optimistic best. We can see far out, think big, and make long-term investments. It’s what has always drawn tremendous immigrants here, and continues to draw them here.

An authoritarian state like Russia can’t say these things.

I’m thankful that the US biotech industry has a generation of leaders who are willing to stand up for our ideals. I hope to see this good fight continue. It may require some sacrifice, whether in the form of clinical trial delays, lost deals, or higher prices for oil or gas. Or maybe worse. It might be the start of a long, hard confrontation.

Some things are more important than next quarter’s earnings, or the fleeting price of a gallon of gas.

We need to rediscover a certain steeliness in our national character. We need to rediscover an ability to focus on what’s important, and not allow ourselves to be distracted.

It will be difficult. But we have to defend a way of life that’s worth defending.  

 

Data That Mattered

Cambridge, Mass.-based Intellia Therapeutics and its partner, Regeneron Pharmaceuticals, reported follow-up data that show deep and sustained efficacy with in vivo gene editing for patients with transthyretin amyloidosis. The companies showed that this CRISPR gene editing technique could bring down serum TTR levels by 52 percent at the tiniest dose tested, and 93 percent among the 6 patients on the high dose – still a very small 1 mg/kg. The drug is getting to the liver, and it’s doing its edit safely — there were no clinically significant liver findings observed. It’s big news for the field, and sets the stage for single-infusion in vivo gene editing in many indications, because it has some practical advantages over ex vivo gene editing. Whether TTR amyloidosis will be the ideal first indication for CRISPR therapies is still an open question – I’m not sure it makes sense (TR coverage, July 2021).

Deals

San Diego and Suzhou, China-based Adagene struck partnership with Sanofi on masked monoclonal and bispecific antibody drug candidates for cancer. Sanofi is paying $17.5 million upfront, and will get the ability to advance two development candidates, with an option for two more. This partnership comes a few months after Sanofi agreed to acquire Amunix Pharmaceuticals for $1 billion upfront. Amunix was also developing masking technology to assist bispecifics against cancer.

Cambridge, Mass.-based Blueprint Medicines, the developer of precision cancer drugs, agreed to pay $20 million upfront to New York-based Proteovant Therapeutics to gain access to its targeted protein degrader drug discovery platform. The companies will work together on targets, and plan to advance two protein degrader development candidates.

AbbVie agreed to acquire Syndesi Therapeutics, a neuroscience drug developer. AbbVie is paying $130 million upfront, and getting ahold of a lead small molecule drug candidate in Phase I development to enhance synaptic efficiency. Synaptic dysfunction is believed to underlie the cognitive impairment seen in multiple neuropsychiatric and neurodegenerative disorders. If the drug pans out, Syndesi shareholders could collect another $870 million in milestone payments.

AstraZeneca’s rare disease unit, Alexion, agreed to pay $30 million upfront to Neurimmune. It’s for the right to develop a monoclonal antibody program to treat transthyretin amyloidosis cardiomyopathy.

Financings

San Francisco-based Rondo Therapeutics raised $67 million in a Series A financing co-led by Red Tree Venture Capital and Canaan Partners. The company is working on bispecific antibody T-cell engagers for solid tumors. It’s led by an entrepreneurial pair responsible for TeneoBio, a bispecifics-for-liquid-tumors company that was acquired last year by Amgen for $900 million upfront. (TR coverage).

Cambridge, Mass.-based Atlas Venture said it raised $450 million for Fund XIII. The firm didn’t announce any strategic or partner changes – just more investment in platforms and asset-centric biotech companies. Since 2015, when the biotech and tech investing sides of Atlas went their separate ways, the new biotech-only Atlas has raised over $2 billion and has created and/or invested in over 50 biotech companies.

San Francisco-based Foresite Capital said it raised $173 million to invest in companies it forms through Foresite Labs. This cash is in addition to the firm’s $969 million Fund V.

A team of eight Howard Hughes Medical Institute investigators at the University of Washington and Fred Hutchinson Cancer Research Center secured a three-year $15 million HHMI grant to work on ways to track, prevent, and treat COVID-19 as it moves into an endemic state. The team includes Jesse Bloom, Trevor Bedford, Harmit Malik and Erick Matsen at Fred Hutch and David Baker, Joseph Mougous, Jay Shendure and David Veesler at UW.

Cambridge, Mass.-based NextRNA Therapeutics secured $46.8 million in a Series A financing led by Cobro Ventures and Lightchain Capital. It’s working on medicines directed at non-coding RNA targets.

Cambridge, Mass.-based Flagship Pioneering announced the debut of Vesalius Therapeutics, with a $75 million commitment. The company is focusing on common illnesses, and using machine learning to assist with drug discovery. Vesalius says its platform uses “proprietary patient-derived experimental systems that it uses to screen and characterize drug candidates to restore circuits to healthy functioning.”

Boston-based Selux Diagnostics raised $50 million in a Series C financing. It’s working on diagnostics for infectious diseases, to help doctors better select precision antibiotics quickly.

Cambridge, Mass.-based hC Bioscience raised $24 million in a Series A financing to develop engineered tRNA therapies to address protein dysfunction. Arch Venture Partners, Takeda Ventures, and 8VC participated.

Princeton, NJ-based ArrePath said it raised $20 million in seed funding to advance its work on anti-infective drugs that will work against microbes that resist current therapies. Boehringer Ingelheim Venture Fund, Insight Partners, and Innospark Ventures co-led. Lloyd Payne, formerly EVP of anti-infectives at Evotec, joined as CEO.

London-based Epsilogen raised $41 million in a Series B financing to develop IgE antibodies for cancer. Novartis Venture Fund led.

Personnel File

Julie Gerberding will be the new CEO of the Foundation for the National Institutes of Health, starting May 16. She is currently Chief Patient Officer and Executive Vice President, Population Health & Sustainability at Merck.

South San Francisco-based Graphite Bio, a gene editing company, hired Alethia Young as chief financial officer. She was previously a biotech analyst with Cantor Fitzgerald. (TR coverage of Graphite, Sept. 2020)

Canada-based Amplitude Ventures hired Ali Tehrani, the co-founder and former CEO of Vancouver, BC-based Zymeworks, as a venture partner.

Merck hired Aileen Pangan, as vice president and therapeutic area head for immunology, global dlinical development, effective March 1. She previously worked at AbbVie.

Cambridge, Mass.-based Synlogic hired Michael Jensen as chief financial officer.

Needham, Mass.-based Candel Therapeutics, the developer of oncolytic virus therapies for cancer, hired Seshu Tyagarajan as chief technical and development officer.

Science

• The burden of rare protein-truncating genetic variants on human lifespan. Nature Aging. Mar. 3. (Jimmy Liu et al Biogen)
• Lipidomic risk scores are independent of polygenic risk scores and can predict incidence of diabetes and cardiovascular disease in a large population cohort. PLoS Biology. Mar. 3. (Chris Lauber et al Institute for Experimental Virology, Hanover, Germany)

Science of SARS-CoV-2

• Assessment of Clinical Effectiveness of BNT162b2 COVID-19 Vaccine in US Adolescents. JAMA Network Open. Mar. 3. (Carlos Oliveira et al Yale School of Medicine)
• Genome-wide analysis provides genetic evidence that ACE2 influences COVID-19 risk and yields risk scores associated with severe disease. Nature Genetics. Mar. 3 (Manuel Ferreira et al Regeneron Genetics Center)
• Recurrent SARS-CoV-2 Mutations in Immunodeficient Patients. MedRxiv. Mar. 2. (Sam Wilkinson et al University of Birmingham, UK)
• Covid-19 Vaccine Effectiveness against the Omicron (B.1.1.529) Variant. NEJM. Mar. 2. (UK Health Security Agency)
• Severe Neuro-COVID is associated with peripheral immune signatures, autoimmunity and signs of neurodegeneration: a prospective cross-sectional study. MedRxiv. Mar. 2. (Manina Etter et al University Hospital Basel)
• SARS-CoV-2 emergence very likely resulted from at least two zoonotic events. Zenodo. Feb. 26. (Jonathan Pekar et al UCSD)
• The Huanan market was the epicenter of SARS-CoV-2 emergence. Zenodo. Feb. 26. (Michael Worobey et al University of Arizona)

Science Policy

• National COVID-19 Preparedness Plan. March 2022. White House. (96-page PDF document).
• CVS, Walgreens, Walmart part of Biden’s Test-to-Treat COVID-19 program. Marketwatch. Mar. 3. (Jaimy Lee)
• Tim Kaine introduces bill to research and combat long covid, after suffering it himself. Washington Post. Mar. 2. (Meagan Flynn)

Science Features

• The Biden Administration Killed America’s Collective Pandemic Approach. The Atlantic. Mar. 2. (Katherine Wu)
• CRISPR Might Actually Live Up to Its Immense Hype. Bloomberg Opinion. Mar. 1. (Lisa Jarvis)
• Arthritis drug reduces mortality in severe COVID-19, huge clinical trial finds. Science. Mar. 3. (Kai Kupferschmidt)

Legal Corner

UC Berkeley lost the high-profile patent case over CRISPR technology, which meant it was also a loss for Intellia Therapeutics and CRISPR Therapeutics – the companies that took a license from UC Berkeley. That decision by the US Patent and Trademark Office was a victory to the Broad Institute and its licensee, Editas Medicine. What fewer may have noticed is that UC Berkeley’s patent was upheld by the European Patent Office. (San Jose Mercury News)

Regulatory Action

Janssen Pharmaceutical and its partner, Legend Biotech, won FDA clearance for cilta-cel (Carvykti), a new cell therapy for cancer directed at the BCMA antigen overexpressed in multiple myeloma patients. The treatment was approved on the basis of a Phase III trial which showed a remarkable 98 percent overall response rate among heavily pre-treated patients, including a 78 percent Complete Response rate. The new treatment will now compete with a BCMA-directed cell therapy from Bristol Myers Squibb (Abecma).

Seattle-based CTI Biopharma secured FDA approval for pacritinib (Vonjo), a JAK inhibitor, as a new treatment for high-risk primary or secondary myelofibrosis.

Newton, Mass.-based Karyopharm Therapeutics said that it plans to start a new Phase III clinical trial of its experimental drug selinexor for advanced or recurrent endometrial cancer. The decision comes after the FDA told the company that its existing Phase III study results are unlikely to be enough to support a supplemental New Drug Application, which the company had been planning to submit.

Belgium-based Celyad Oncology voluntarily placed a clinical trial on hold for its experimental CAR-T cell therapy being tested in combo with Merck’s pembrolizumab (Keytruda) after learning of two deaths with similar pulmonary findings. The company said it’s investigating the nature of the deaths and in discussions with regulators.

Somerville, Mass.-based Finch Therapeutics said the FDA placed its Phase III clinical trial for a C.difficile treatment on hold, over concerns that donor-derived microbiome therapies could potentially spread the SARS-CoV-2 virus.

The FDA issued a Clinical Hold to Gilead Sciences until it can resolve the issue with glass fragments showing up in vials of lenacapavir, a long-acting treatment being evaluated against HIV-1.

The FDA rejected an application from Plano, Tex.-based Reata Pharmaceuticals to market bardoxolone for kidney disease caused by Alport Syndrome.

Tweetworthy

Our healthcare system is way too costly, but it’s also way too complex. We need not only to make things more affordable, but more simple, if we want to improve access. The President spoke about making tests and antiviral treatments available at pharmacies. It’s showing an instinct for simple problem solving. Of course, it takes work behind the scenes to make things appear simple on the surface, as Eric Topol notes.

 

Every thriving biotech hub can trace its origins to one or two outstanding scientific institutions. But every thriving region can also trace some of its success back to community leaders.

These are people who attend boring night meetings. They aren’t household names. They’re fine with that.

These people were especially common in America after World War II. They laid down the physical infrastructure and social norms for those of us who came next.

One of these people in Seattle, where I live, was named Jim Ellis.

Ellis died three years ago at 98. He was a named partner at one of Seattle’s top law firms, Preston Gates & Ellis. He worked with William Gates Sr., Bill’s dad. The firm today is known as K&L Gates.

But that’s not why we remember Jim Ellis.

When he was a up-and-coming lawyer, Lake Washington, a freshwater jewel linked to Puget Sound and the Pacific, was full of sewage. People at the time said the water was like “split pea soup.” Various government fiefdoms and unrestrained private land developers contributed to it. It was a mess. Fingers were pointed.

Ellis got involved. Behind the scenes, he brought his energy and creativity to the task of herding the necessary cats to clean it up.

But there was more. The Seattle region was growing beyond its natural resource-based economy – logging, fishing, the seaport. Boeing was emerging. Newcomers were coming. The region needed to think about how to manage the growth intelligently.

Highways needed to be expanded. Parks, trails, public swimming pools and youth centers needed to be built and upgraded. It was going to cost money. Ellis mobilized community support for a series of bond initiatives in the 1950s and 1960s that were collectively known as “Forward Thrust.”

All of this was happening in tension with the natural splendor of the Cascades. Millions of acres of forest needed to be preserved for wildlife habitat, for outdoor recreation, for clean air and water, and to preserve natural beauty. The competing interests between economic growth and environmental preservation needed to be held in balance.

Ellis thought about common interests, the common good. By the early 1990s, he put it all together with his greatest achievement — the Mountains to Sound Greenway. It’s 1.5 million acres of preserved land between Ellensburg, on the east side of the Cascades, stretching to Seattle in the west.

I first saw it as a 21-year-old kid from Wisconsin. It was Memorial Day weekend of 1997. I was excited to start a summer reporting internship at one of the nation’s great regional newspapers – The Seattle Times. I had driven my rusty Pontiac 2,000 miles across the Great Plains, long stretches of Montana, and the arid Columbia River basin.

Then came the mountains. They were covered in Evergreen trees. Alpine lakes shimmered.

From Snoqualmie Pass to Seattle, for 50 miles, it kept going. No strip malls or tacky billboards. People lived in the suburbs east of Seattle, the foothills of the Cascades. But the trees were everywhere, swallowing you up. Nature felt big. Individual people felt small.

This seemed like a great place to live, to explore, to build a career.

For years, I knew nothing about the history of the Mountains to Sound Greenway. Maybe a decade ago, I learned for the first time about Jim Ellis. There was no statue to the man. When he died, I looked for more information, but there wasn’t much. Recently, I found this touching tribute published in 2021 by the Trust for Public Lands.

It’s hard to imagine what Ellis was up against in his day. Think of the vision and tenacity it must have taken with all the federal, state, and private landowning interests. A lot of people with different viewpoints needed to rally around a shared vision.

Ellis wasn’t in it for money or ego. He never ran for public office.  

One secret to his success was his philosophy on how to spend his time. He spoke of a one-third/one-third/one-third way of life. One-third was for professional work, one-third for family, and one-third for community.

Where did this philosophy and drive come from? It’s hard to say for sure. But Ellis’ brother died in World War II in 1945. That, according to the Trust for Public Lands, lit a fire in him to honor his brother’s memory. I also have a hunch that the community work, and the family time, helped energize him and make him even more effective professionally. It could have been a virtuous cycle.

Ellis was on my mind last weekend, when I took a small group of biotech people on a hike up Mailbox Peak. It’s a 4,800-foot peak near North Bend, smack in the middle of the Mountains to Sound Greenway.

Around Noon, there were about 20 people on the summit when my small group arrived. Most were in their 20s and 30s. The skies were sunny and clear. You could see snow-capped peaks more than 100 miles away.

 

Two young people we met on the hike happened to be from the biotech community. A postdoc on my team, Aleena Arakaki, wasn’t surprised. It’s part of what draws young scientists to the Fred Hutchinson Cancer Research Center – the chance to occasionally get away, to clear the head, get a little exercise, maybe get a beer afterwards with lab friends and colleagues.

Seattle’s biotech community exists because of decades of public investment in science at the University of Washington and Fred Hutch. But it also thrives today because we have such amazing quality of life in the Northwest that continually attracts people from around the world.

For that, we can thank unsung heroes like Jim Ellis.

This also makes me wonder: Who are the people doing similar things in Boston, San Francisco, San Diego, Philadelphia, Los Angeles, Chicago, Raleigh-Durham, New York, New Jersey and elsewhere?

Who laid down the critical scaffolding that made it possible for those regions to thrive?

Who’s continuing this work today?

There are people out there doing this hard and thankless work to strengthen our communities. Let’s show a little respect. Maybe get involved personally. It’s meaningful work, and it can be lasting work.

 

Financings

New York-based Kallyope, the developer of treatments based on the science of the gut-brain axis, raised $236 million in a Series D financing. Mubadala Investment Company and The Column Group co-led.

Cambridge, Mass.-based Third Harmonic Bio came out of stealth mode, announcing it has closed a $105 million Series B financing and raised a total of $155 million since inception. General Atlantic and BVF Partners co-led the B round, and Atlas Venture and OrbiMed led the A round. The company is in clinical development with an oral KIT inhibitor for treatment of severe allergy and inflammation. Natalie Holles, formerly of Audentes Therapeutics, is the CEO.

Pasadena, Calif.-based Terray Therapeutics raised $60 million in a Series A financing to advance its work on AI drug discovery. Madrona Venture Group led.

Redwood City, Calif.-based Synthego, the genome engineering company, raised $200 million in a Series E financing. Perceptive Advisors led.

The Mark Foundation for Cancer Research announced a new $500 million funding commitment from founder Alex Knaster. The foundation also announced Ray DuBois has joined as executive chairman of the board.

South San Francisco-based Walking Fish Therapeutics, a developer of B cell therapies, closed an expanded $73 million Series A financing co-led by Northpond Ventures and First Spark Ventures.

South San Francisco-based Electra Therapeutics raised $84 million in a Series B financing co-led by Westlake Village Biopartners and OrbiMed. It’s developing antibodies against signal regulatory proteins.

South San Francisco-based Twist Bioscience, the DNA synthesis company, raised $269 million in a stock offering at $55 a share.

Deals

Cambridge, Mass.-based Remix Therapeutics secured $45 million upfront through a partnership with Janssen Pharmaceuticals. The companies will work together on small molecules designed to work by reprogramming RNA processing. (TR coverage, Dec. 2020)

Watertown, Mass.-based Neumora Therapeutics obtained an exclusive worldwide license to an M4 muscarinic receptor allosteric modulating drug from Vanderbilt University’s Warren Center for Neuroscience Drug Discovery. The company is getting two novel series of compounds in late preclinical development, which it says has the potential for a better safety profile and once-daily dosing. Terms weren’t disclosed. (TR coverage of Neumora, Oct. 2021).

Waltham, Mass.-based ImmunoGen pocketed $13 million upfront through a deal with Eli Lilly to develop antibody-drug conjugates for cancer.

The Scientific Enterprise

• The Dilemma of Brilliant Jerks. The Bulwark. Feb. 11. (David Shaywitz)
• Sometimes Science Takes a Village. Whitehead Institute. Feb. 16. (Greta Friar)
• Recapturing Our Resilience. LifeSciVC. Feb. 16. (Ankit Mahadevia)
• Pfizer joined the Open Targets Consortium, a group focused on drug target identification, prioritization, and validation. EMBL’s European Bioinformatics Institute, the Wellcome Sanger Institute, GSK, Bristol Myers Squibb, and Sanofi are current members. Feb. 14. (Pfizer statement)

Vaccine Equity

• Vaccine Makers Must Help the World Prepare for the Next Pandemic. Bloomberg Opinion. Feb. 14. (Lisa Jarvis)
• Health Canada authorized the Novavax protein-plus-adjuvant vaccine for COVID-19. Novavax touted the efficacy of its vaccine, which we already knew, but said nothing in its press release about how many doses it can and will ship to Canada. (See Frontpoints, Feb. 3, 2022, “The World Waits for Novavax.”
• Moderna announced it’s building up Asian commercial subsidiaries in Malaysia, Taiwan, Singapore and Hong Kong, as well as European commercial presence in Belgium, Denmark, the Netherlands, Norway, Poland, and Sweden.

Science Policy

• Pandemics disable people — the history lesson that policymakers ignore. Nature. Feb. 16. (Laura Spinney)
• Long-Haul COVID Cases Could Spike after Latest Wave. Scientific American. Feb. 3 (Melinda Wenner Moyer)
• Americans are tired of the pandemic. But disease experts preach caution — and endure a ‘kill the messenger’ moment. Washington Post. Feb. 17. (Joel Achenbach)
• The Moral Danger of Declaring the Pandemic Over Too Soon. NYT. Feb. 17. (Gregg Gonsalves)

Our Shared Humanity

Health Affairs came out with a series of articles on Racism & Health. There is a lot here to read and think about carefully. This issue deserves some quiet reading time over the weekend. (Health Affairs)

The Millions of People Stuck in Pandemic Limbo. What Does Society Owe Immunocompromised People? The Atlantic. Feb. 16. (Ed Yong)

The Internet is fundamental to life in the 21^st century. It’s hard to communicate with the healthcare system, find out where vaccines are available, make an appointment, etc. without it. And yet in America, a lot of people don’t have easy access to the Internet, and that fact makes it harder for them to access healthcare. Read more about it in “Advancing Diversity, Equity, and Inclusion Through Web Accessibility” by Mona McCarthy, Senior Director of Data and Technology, and Warner Santiago, Senior Director of DEI and Workforce Development at MassBio.

Data That Mattered

Cambridge, Mass.-based Sage Therapeutics and Biogen reported that a Phase III trial of zuranolone hit its primary and secondary endpoints in a study of patents with major depressive disorder. The effect kicked in quickly, after three days, and lasted through the two-week treatment period.

Regulatory Action

Eli Lilly won FDA Emergency Use Authorization for bebtelovimab, as a new therapeutic neutralizing antibody against SARS-CoV-2, including the Omicron variant. It’s for patients ages 12 and up, for the treatment of mild to moderate COVID, for patients who are at high risk.

Cambridge, Mass.-based Agios Pharmaceuticals won FDA approval for mitapivat (Pyrukynd) as a treatment for hemolytic anemia in adults with pyruvate kinase deficiency.

Somerset, NJ-based Legend Biotech said the FDA placed its Phase I trial of an autologous CAR-T cell therapy for T-cell lymphoma on clinical hold. The first patient dosed in the trial experienced low CD4+ T cells, and the company notified the FDA before the hold. The patient hasn’t suffered a serious adverse event, the company said.

A Less Known Infectious Threat

The State of Innovation in Antibacterial Therapeutics. BIO Industry Analysis. Feb. 2022. (David Thomas and Chad Wessel)

Science

• Safety and target engagement of an oral small-molecule sequestrant in adolescents with autism spectrum disorder: an open-label phase 1b/2a trial. Nature Medicine. Feb. 14. (Stewart Campbell et al Axial Therapeutics)
• Whole genome sequencing for the diagnosis of neurological repeat expansion disorders in the UK: a retrospective diagnostic accuracy and prospective clinical validation study. The Lancet Neurology. March 2022. (Kristina Ibanez et al Genomics England)
• EDP-938, a Respiratory Syncytial Virus Inhibitor, in a Human Virus Challenge. New England Journal of Medicine. Feb. 17. (Alaa Ahmad et al Enanta Pharmaceuticals)
• Enhancing neural markers of attention in children with ADHD using a digital therapeutic. PLoS One. Dec. 31, 2021. (Courtney Gallen et al UCSF

Science of SARS-CoV-2

• The oral protease inhibitor (Pfizer’s Paxlovid) protects Syrian hamsters against infection with SARS-CoV-2 variants of concern. Nature Communications. Feb. 15. (Rana Abdelnabi et al Rega Institute for Medical Research, Belgium.)
• Virological characteristics of SARS-CoV-2 BA.2 variant. BioRxiv. Feb. 15. (Daichi Yamasoba et al University of Tokyo)
• Virome characterization of game animals in China reveals a spectrum of emerging pathogens. Cell. Feb. 15. (Wan-Ting He et al Nanjing Agricultural University, China)
• Multistate Outbreak of SARS-CoV-2 B.1.1.529 (Omicron) Variant Infections Among Persons in a Social Network Attending a Convention — New York City, November 18–December 20, 2021. CDC Morbidity and Mortality Weekly Report. Feb. 18. (Sarah Smith-Jeffcoat et al CDC)
• Waning 2-Dose and 3-Dose Effectiveness of mRNA Vaccines Against COVID-19–Associated Emergency Department and Urgent Care Encounters and Hospitalizations Among Adults During Periods of Delta and Omicron Variant Predominance — VISION Network, 10 States, August 2021–January 2022. CDC Morbidity and Mortality Weekly Report. Feb. 11. (Jill Ferdinands et al CDC)

Science Features

The Calorie Counter. Evolutionary anthropologist Herman Pontzer busts myths about how humans burn calories—and why. Science. Feb. 17. (Ann Gibbons)

We’re Entering the Control Phase of the Pandemic. The Atlantic. Feb. 17. (Katherine Wu)

Personnel File

The US Senate voted 50-46 to confirm Robert Califf as FDA commissioner. The acting commissioner, Janet Woodcock, will stay on as principal deputy commissioner. There’s not a lot here worth analyzing. It’s simple. An experienced, visionary and utterly non-controversial nominee to run this critical public health agency was delayed three months for no good reason.

The White House, days after Eric Lander resigned as science advisor, said former NIH director Francis Collins will step in as science advisor to the President. Alondra Nelson will perform the job of director of the Office of Science & Technology Policy, stepping up from her current role as deputy director for science and society at OSTP. These two have important work to do in continuing to shepherd along the reboot of the Cancer Moonshot, and the creation of the Advanced Research Projects Agency for Health (ARPA-H).

San Francisco-based Vertex Ventures HC hired Christine Brennan as a managing director. She is based in Cambridge, Mass. and was previously a partner with MRL Ventures Fund.

New York-based Stablix hired Tony Kingsley as CEO. He previously did a brief stint at Scholar Rock.

Brett Giroir, former assistant secretary of Health and Human Services, was hired as CEO of Atlanta-based Altesa Biosciences. The company is working on antiviral drugs against common respiratory viruses such as rhinovirus and parainfluenza, and global viral threats like Dengue fever and Yellow Fever.