Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.
We’ve seen so much news this week that I almost can’t believe some stories featured below were from Monday. Be sure to read to the bottom in case you missed anything.
Top stories from #ASGCT24
Scientists, clinicians and startups congregated in Baltimore with updates on cutting-edge technologies at the American Society of Gene & Cell Therapy’s annual meeting. From Lei Lei Wu and Ryan Cross, we read about how, across two clinical trials, gene therapy restores hearing in children deaf since birth; several secretive and well-funded biotechs shared the first glimpse of data on their gene insertion techniques; Tessera Therapeutics announced it developed a lipid nanoparticle that shuttles a gene editing therapy directly to the bone marrow of mice; and more.
2032 deadline to decouple
An updated draft of the Biosecure Act would give biopharma companies until 2032 to end existing contracts with WuXi AppTec and other Chinese “companies of concern,” as Endpoints reported earlier in the week. In an interview with Endpoints’ Jared Whitlock, BIO CEO John Crawley defended the organization’s about-face on the legislation With tensions continuing to grow, House Oversight Committee Chair James Comer is also pressing the FDA to share more information on its presence in China.
Patient death in Pfizer gene therapy trial
A Duchenne muscular dystrophy patient died of cardiac arrest in a Phase 2 trial of Pfizer’s gene therapy, the pharma company reported this week. The patient received the therapy in early 2023. Pfizer is pausing dosing in a separate Phase 3 study and is working with investigators to understand what happened.
Sanofi teams with Novavax
Sanofi is handing Novavax $500 million upfront to bring the biotech’s Covid-19 vaccine into the fold, with dual goals of co-commercializing the current vaccine and developing combo vaccines that can tackle both Covid and flu. The deal, which can add up to $1.2 billion including milestones, comes as other Covid vaccine makers are reporting lower revenues and AstraZeneca, seeing “no future commercial demand,” decided to withdraw its vaccine globally.
One Feng Zhang startup buys another
In the bid to lock up patent rights to a growing suite of gene editing technologies, Arbor Biotechnologies acquired a small startup founded by MIT scientist and CRISPR pioneer Feng Zhang, Ryan Cross reports.
SPOTLIGHT
At least three Chinese biotech companies have recently begun tests of CRISPR treatments for blood, eye and liver diseases, with more on the way, Ryan Cross writes. The developments come a half-decade after a scientist’s reckless experiment with human embryos put a stop to much of China’s clinical work on CRISPR gene editing.
Exclusive: Engineered B cell therapy startup Walking Fish shuts down
Walking Fish Therapeutics, a biotech startup that attempted to create engineered B cell therapies, has shuttered. The company was within two months of requesting FDA approval to start its first clinical trial, but
WHAT WE’RE WATCHING NEXT WEEK
- For those of you in New York, Endpoints News and the Financial Times will be hosting the US Pharma and Biotech Summit on May 16, with a rich slate of panels on artificial intelligence, different approaches to the US longevity crisis, the battle over who owns the relationship with patients, the macro economy and its impact on investments and dealmaking, and more. You can also register to watch online.
- The House Oversight Committee is set to mark up the Biosecure Act on May 15. If passed, the far-reaching bill could force many US biopharma companies to cut ties with WuXi and several other Chinese life sciences contractors.
CELL/GENE TX
- Following two landmark approvals for CRISPR-based treatments for sickle cell disease in December, makers of both therapies touted progress on the rollout. Vertex said more than 25 treatment sites have been activated worldwide and “five patients already had cells collected.” Bluebird, meanwhile, said one patient has been started on its treatment among 15 across its three gene therapies.
- Exclusive: New York biotech debuts with $16M to find new ways to make solid cancer cell therapies
- UPenn and Stanford analysis finds no secondary cancer cases directly due to CAR-T
- Excision BioTherapeutics’ attempt to gene edit HIV disappoints. The company will now revamp vector: #ASGCT24
- Freeline unveils early efficacy signal for Gaucher gene therapy: #ASGCT24
- AstraZeneca expands stake in cell and gene therapy partner Cellectis
Q1 EARNINGS
- Takeda is launching a broad, multiyear restructuring, alongside a handful of pipeline cuts. One of the changes is discontinuing Phase 2 development of its CAR-NK TAK-007 therapy in blood cancer as it pivots the candidate to autoimmune disease. The drugmaker is also shuttering its San Diego research site, Nicole DeFeudis reports.
- Teva to close additional manufacturing sites as it enters next phase of ‘pivot to growth’
- Madrigal ‘encouraged’ by early launch progress of landmark NASH drug
DEALS
- Novo Nordisk is pairing up with another Flagship-founded biotech to create new obesity medications, and GLP-1 remains in the frame. The $600 million pact will tap into the “molecular mimicry” work at Metaphore Biotechnologies to make one or two obesity management therapeutics.
- Chiesi bets $160M on Gossamer Bio’s make-or-break late-stage PAH drug
- After FTC scrutiny of Sanofi deal, Maze finds a new Pompe partner in Shionogi
- Illumina takes a step toward spinning off Grail, though keeps option open for sale
PEOPLE
- Sanofi’s CSO and global head of research Frank Nestle is leaving the company after taking over both positions in 2020. He was previously the French pharma’s head of immunology and inflammation research therapeutic area, and his exit marks Sanofi’s second major departure this year.
- Acelyrin’s founding CEO steps down, company scraps earnings call
- Pfizer hires Citi analyst Baum to oversee portfolio strategy
- Ginkgo to cut spending, lay off staff after earnings disappointment
- AGC Biologics to cut close to 4% of staffers ‘to adjust business priorities’
- Marinus lays off 20% of workforce, cuts costs to stay afloat
- Lykos shores up commercial team for MDMA drug; Former J&J CEO Alex Gorsky finds his next gig
STARTUPS
- LabCentral helped create 6,300 jobs and 19 IPOs. Amid a slump, demand for lab space has slowed
- Flagship commits $50M to new startup that scours viral proteins for new drugs
FINANCING
- Eccogene, a Boston and Shanghai biotech working on an oral GLP-1 candidate with AstraZeneca, is considering an initial public offering early next year, Kyle LaHucik reports. The company is also gearing up for multiple mid-stage tests in the liver condition NASH.
- Lonnie Moulder’s Zenas closes a $200M Series C to broaden lead drug’s autoimmune potential
- Aardvark nabs $85M to target hunger, not appetite, as it eyes 2026 NDA filing
- Infectious disease biotech Bluejay raises $182M for hepatitis trials
- Attovia reels in $105M from crossover investors to run multiple trials of I&I biologics
- Danish biotech nabs Novo Holdings support in seed round for complement platform
- Memo Therapeutics refuels with $22M to expand trial for infection-fighting antibody in kidney transplant recipients
- Verona inks financing deal worth up to $650M ahead of COPD decision
R&D
- ‘Bigger, faster, better’: Google DeepMind and Isomorphic Labs unveiled the newest version of AlphaFold, taking its flagship AI model far beyond predicting the structure of a single protein.
- CytomX and Amgen’s “masked” T cell engager showed mixed efficacy signals in an early trial in advanced solid tumors, sending the biotech’s shares down even as analysts saw the data in a more positive light.
- J&J reports latest data on Balversa-based ‘pretzel’ therapy for bladder cancer
- Teva’s antipsychotic asset passes Phase 3 test in schizophrenia
- MacroGenics investigates three patient deaths in Phase 2 prostate cancer drug trial
- GlycoMimetics’ AML drug flunks Phase 3 trial, quashing filing plans as stock sinks
- Rapt Therapeutics winds down Phase 2 studies of lead candidate in wake of clinical holds
- ADC Therapeutics reveals topline Zynlonta data, $105M stock offering
PHARMA
- The FDA delayed the PDUFA date for Moderna’s RSV vaccine. The delay likely won’t push the decision past the end of May and analysts largely still expect the vaccine to be approved. What comes after, Max Gelman writes, is likely to be a tougher, longer climb.
- Industry groups critique new guidance for Medicare drug price negotiations in 2027
- House Republicans put pressure on ASPR’s management of the national drug stockpile
- Senators question Chamber of Commerce’s motives around march-in rights
- Ahead of likely approval, ICER finds Geron’s imetelstat is not cost-effective at $250,000 annually
FDA+
- US lawmakers are seeking more information on government response to the H5N1 bird flu. Senate HELP Committee ranking member Bill Cassidy called on the Biden administration to do a better job of communicating its findings on the recent cattle outbreak, while the Senate Appropriations Committee questioned FDA Commissioner Rob Califf on preparation against the virus.
- White House unveils long-awaited policy on overseeing research of concern
- Lilly gets June adcomm for Alzheimer’s drug donanemab, following earlier delay
- Remodeling REMS: FDA kicks off guidance series with draft on new framework
- Corrected: Experts to decide within a year whether to recommend Duchenne muscular dystrophy for newborn screening
- House panel marks up bill to require PBMs to use a flat fee in Medicare
CHINA
- Charles River touts first-quarter manufacturing revenue, says China bill is ‘net positive’
- Innovent says next-gen GLP-1 drug from Lilly beats top diabetes med in Phase 3 China study
- BeiGene earmarks $74M to build fourth facility in China
HEALTH TECH
- For the first time in April, biosimilars started to gain a larger share of the Humira market — and it’s largely because Humira came off the formulary of CVS Health’s pharmacy benefit manager Caremark. Lydia Ramsey Pflanzer takes us inside the decision in an interviews with a Caremark executive.
- As digital health unicorns are pressured to do more with less, Transcarent gets a fresh $126M
- How insurance upstart Alignment kept medical costs in check while Medicare Advantage plans struggled
- Oscar is winding down its small employer health plans with Cigna
- Employers are pulling back from providing mental health benefits, Talkspace warns
DON’T MISS