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.
Thank you for sticking with us this busy week! Between earnings calls, scientific conferences, advisory committee meetings and exclusive interviews, our team is bringing you news and scoops from all around. Dig in to see if you missed anything.
Eisai’s new Leqembi data
Eisai has long argued that treating patients with Leqembi earlier and longer would likely lead to more dramatic effects. This week, the company presented new data to back that hypothesis. But weaknesses in the study raised questions, Ryan Cross reports. Eisai and partner Biogen are continuing to ramp up sales of the Alzheimer’s drug (which Biogen CEO said is now “on the right path”), and Eisai said revenue is growing faster than expected in Japan.
Jim Wilson’s next move
Gene therapy pioneer Jim Wilson will leave his academic post at the University of Pennsylvania and launch two new companies, one focused on developing rare diseases and another meant to provide contract services for genetic medicines. No financial details were provided, but most of the current staff at UPenn’s Gene Therapy Program received offers to join those two companies, the university said.
Roivant dealmaker’s payday
One of the best pharma deals in recent history has now led to one of the industry’s biggest-ever executive paydays. Roivant awarded its top dealmaker Mayukh Sukhatme an $80.55 million cash bonus, according to a proxy filing, in service for his work as a key architect of Roivant’s sale of an immunology asset to Roche.
What’s next for MDMA?
It took more than two decades for MDMA to reach the FDA. In two months, the prospects of the company that promised to bring the first such therapy to market — Lykos Therapeutics — cracked after an expert panel picked their trial apart. Ahead of a major FDA decision, Max Bayer spoke to Lykos about the path ahead.
SPOTLIGHT
Biosecure lawmakers tell trade groups that China studies are a new focus
PARP biotech backed by more than five pharmas closes after nine years
Exclusive: Startup aims to tackle ‘undruggable’ neuro targets with tech from Kevan Shokat’s UCSF lab
WHAT WE’RE WATCHING NEXT WEEK
- On Tuesday, we will be hosting our Manufacturing Day, unpacking a part of the pharmaceutical business that’s increasingly under the spotlight. Panels will explore local opportunities amid global tensions, the GLP-1 compounding gray area and more. Learn more and register here.
- We’re not done with earnings yet! Expect close scrutiny on Novo Nordisk and Eli Lilly’s GLP-1 sales numbers — other notable names reporting include Bayer, Amgen and Gilead.
Q2 EARNINGS
- Vertex said about 20 patients have started its sickle cell therapy Casgevy, as it picks up momentum in branching out beyond cystic fibrosis. As part of the quarterly update, it disclosed cutting two rare disease programs. Earlier in the week, the company also announced that the FDA is set to make a decision about its non-opioid pain medicine in January.
- While Merck marked a milestone for Keytruda — which topped $7 billion in quarterly sales for the first time — its second-biggest product saw a slowdown. China’s recent effort to crack down on healthcare corruption is affecting shipments of its HPV vaccine Gardasil, Merck told investors.
- Pfizer increased its 2024 guidance by $1 billion after multiple rounds of cost cuts. For analysts, the uneventful update is a reprieve after a year of tumult.
- Takeda executives said the Japan-based pharma giant’s $900 million restructuring is in “full swing” as the pipeline has been reconfigured to focus on late-stage hopefuls. That includes taking a $140 million impairment on an epilepsy drug that recently missed in Phase 3. Execs also defended their recent Alzheimer’s pact with AC Immune and remained upbeat on its mega-bet in the TYK2 space.
- Vir’s overhaul brings an end to most virology work — and a beginning with cancer
- WuXi AppTec sees US revenue decline amid Biosecure threat
- RSV market pressure leads to slower launch for Moderna’s vaccine
- Updated: GSK unintimidated by RSV vaccine uptake challenges, Shingrix US sales drop
- Incyte drops four early-stage programs from its pipeline as part of strategic review
- US sales for Merck KGaA’s Bavencio impacted by Padcev/Keytruda momentum in bladder cancer
- Teva factored IRA’s next round of negotiations into its growth plans
DEALS
- Boehringer Ingelheim is paying up to $1.3 billion to acquire a relatively unknown immuno-oncology biotech out of La Jolla, CA. Preclinical-stage Nerio Therapeutics is working on a small molecule that inhibits an immune checkpoint that AbbVie is also targeting. It marks the latest in a series of deals Boehringer has inked to beef up its cancer drug pipeline. Kyle LaHucik interviewed Boehringer for a deeper look.
- Updated: Otsuka buys out small molecule drug discovery startup Jnana and its rare disease pill for $800M cash
- Updated: GSK and Flagship to create up to 10 vaccines, medicines across respiratory and immunology
- AbbVie closes $8.7B Cerevel deal in bid to compete with Bristol Myers in schizophrenia
- 23andMe board rejects Anne Wojcicki’s take-private plan, requests changes
CELL/GENE TX
- Pfizer will end development on its experimental Duchenne muscular dystrophy gene therapy after a Phase 3 failure last month and plans to eliminate roles at a North Carolina site that was working on the project. It’s also letting go of employees in another North Carolina facility, affecting about 210 workers total.
- Adaptimmune gets FDA accelerated approval for T cell receptor cell therapy in rare soft tissue cancer
- Umoja to dose first patient with in vivo CAR-T by end of this year
- Exclusive: Myeloid Therapeutics moves second in vivo cell therapy into trials, targeting liver cancer
- Galapagos pauses blood cancer CAR-T therapy trial due to one Parkinsonism event
R&D
- One of BioNTech’s first mRNA cancer vaccine candidates cleared a key mid-stage combination trial with Regeneron’s PD-1 drug, setting them up for a potential showdown with a closely watched and more advanced program from Merck and Moderna.
- Updated: Eli Lilly’s tirzepatide reduces risk of heart failure outcomes in Phase 3 study
- NewAmsterdam succeeds in the first of three key pivotal studies, providing hope to a troubled cardio class
- ALX Oncology continues its CD47 quest, revealing Phase 2 gastric cancer data
- Roche begins Phase 2 obesity trial for GLP-1/GIP agonist, striking while iron is hot
- Imunon shares up over 150% as ovarian cancer treatment extends survival in Phase 2
- MacroGenics stops further dosing in Phase 2 prostate cancer drug trial
- Ventyx axes development of TYK2 inhibitor following Phase 2 failure
- Sage changes main endpoint in Huntington’s study months ahead of readout
- AstraZeneca’s fixed-duration Calquence combo succeeds in Phase 3 blood cancer test
- Celldex says antibody drug improves irritable skin condition in Phase 2 test
FINANCING
- VC firm venBio secures $528M for ‘extremely selective’ fifth fund after recent IPOs, M&A exits
- With $144M Series B, Outpace Bio beats funding goal for solid tumor cell therapies
- As Amgen pursues GIP antagonism, a Novo Holdings-backed biotech does the same, boosting its Series A
- Exclusive: Ultragenyx secures first funds for Alzheimer’s gene therapy spinout
- Apogee and Spyre founder launches another autoimmune biotech, and it’s got $80M to start
- RNA editing startup AIRNA raises $60M for clinical trial in rare liver disease
- Rare disease biotech raises $47M Series C, plans for Phase 2 trial
PEOPLE
- Former RayzeBio CEO quickly returns with new biotech; Amgen’s global research leader steps down
- Despite recent trial success, uniQure plans to cut 65% of staff
- FibroGen to shrink US workforce by 75% following more late-stage fails
- After norovirus vaccine’s trial failure, HilleVax lays off 40% of staff
- Arbutus reduces headcount after axing HBV combo trial that features Imfinzi
PHARMA
- AstraZeneca and Daiichi Sankyo fail to win NICE recommendation for Enhertu, call for change to review process
- Kyowa Kirin divests China unit, prunes discovery and manufacturing work
- Agios’ late-stage Pyrukynd trial in kids with rare blood disorder misses primary endpoint
#AAIC24
- Iron chelator therapy unexpectedly worsened Alzheimer’s disease in clinical study
- Anavex’s new analysis of its Alzheimer’s pill shows mixed results
- Cognition Therapeutics’ stock sinks on Phase 2 Alzheimer’s data
FDA+
- Ahead of an advisory meeting around Zevra Therapeutics’ experimental treatment for Niemann-Pick disease type C, the FDA raised questions about the strength of human, mouse and cell data. But outside experts largely voted in favor of the drug, saying it’s effective based on current data.
- Filing a new drug with clinical data in 2025? FDA sets fee at $4.3M
- GSK scores expanded label for Jemperli in first-line endometrial cancer
- vTv Therapeutics’ diabetes drug program hit with clinical hold from FDA
- FDA’s ad enforcer critiques Brittany Mahomes’ paid Instagram post for Kaléo
LAW
- After the FDA shot down a second Novartis petition seeking to halt Entresto generics, the Swiss drugmaker sued the agency over its approval of a generic.
- Massachusetts’ plan for $1B in life science investment stalls in state legislature
- Judge rejects Novo Nordisk’s IRA case as pricing negotiations conclude
- Sanofi sues Sarepta, alleging that Duchenne treatment Elevidys infringes on AAV patents
- Indivior reaches $86M settlement over its alleged role in the opioid epidemic
MANUFACTURING
- Sanofi pours €1.3B into new insulin manufacturing plant in Frankfurt
- Doses of Lilly’s blockbuster GLP-1 in shortage have been pulled from FDA supply list
- Pfizer mulls facility sale; GSK penalized in South Korea for administrative breach
HEALTH TECH
- Exclusive: Devoted Health raises $112M, plots insurance markets expansion
- Bessemer VC Morgan Cheatham shares how AI healthcare startups can win in a crowded market
- Teladoc records $790M charge, withdraws outlook as mental health business loses users
- Humana is still struggling with high medical costs as new CEO Jim Rechtin takes over