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This week had everything: highly anticipated trial readouts, Supreme Court decisions, groundbreaking gene editing research and some Biogen gossip. Note that next week, due to the July 4 holiday, we will only be publishing our three daily newsletters Monday through Wednesday. We’re off on the Fourth and will be sending one newsletter on Friday around 11:30 a.m. Eastern time.
Alnylam’s big Phase 3 win
Alnylam cleared a closely-watched, make-or-break Phase 3 trial for vutrisiran, which could become the first RNA silencing therapy on the market for ATTR amyloidosis with cardiomyopathy. The drug, which is already approved as Amvuttra for another manifestation of ATTR amyloidosis, reduced the risk of death, heart-related hospital visits and hospitalizations by 28% compared to placebo.
LGBTQ+ leaders charting paths
Continuing a cherished Endpoints tradition for a third year, we are celebrating Pride month with our annual feature on LGBTQ+ leaders in biopharma. This year’s 14 honorees are a multifaceted group of people who are directly creating new treatments (including one who hopes to find a drug for Alzheimer’s, which his husband might one day get) or are involved in the broader biotech ecosystem that makes this fascinating industry turn on all cylinders every day.
CRISPR and beyond
There are two stories you need to know in the gene editing space, and Ryan Cross has got you covered on both: Intellia presented a small trial suggesting that its experimental CRISPR therapy could be safe to readminister, opening the door to treating conditions that might require more than one dose for maximum benefit; while scientists led by Patrick Hsu at the Arc Institute in Palo Alto have unveiled a new form of gene editing that can add, remove or flip large stretches of DNA — all without the help of CRISPR.
FDA implications after Chevron overturned
The Supreme Court on Friday overturned a longstanding precedent where courts were directed to defer to the expertise of government agencies in ambiguous areas of the law. The decision is consequential for the FDA, which experts said will likely see delays in issuing regulations, particularly if lower courts come to different conclusions about the agency’s authority.
A $372M AI idea
While the bulk of AI-focused biotechs try to discover drugs and push the boundaries of biology, Formation Bio is focused on the unglamorous tasks that make drug development a slow, costly slog. It now has the cash to put those efficiency goals to the test after raising a $372 million Series D led by a16z with the goal of deepening its own pipeline through a flurry of dealmaking.
Biogen plans to spend up to $8B
Adam Keeney, Biogen’s head of business development, told Endpoints in an exclusive interview that the company is “actively monitoring” the M&A landscape despite pulling off a $7.3 billion buyout of Reata last year. The comments come after CEO Chris Viehbacher said in April that acquisitions would be limited for the rest of 2024.
SPOTLIGHT
Lykos’ pain is others’ gain as psychedelic biotechs take notes from adcomm vote
STARTUPS
- Ex-Meta scientists launched a startup in hopes of harnessing the power of evolution — without the millennia of waiting around. Dubbed EvolutionaryScale, the company is debuting with a $142M seed round and a next-gen AI model for protein design to rival AlphaFold.
- New cancer biotech emerges from Stanford research, with help of Nobel laureate Carolyn Bertozzi
- Can mRNA be harnessed for gene insertion? Moderna’s former chief medical officer has $82M to find out
- To develop better cell therapies for solid tumors, a new startup plans to test hundreds of them at once
FINANCING
- Alumis aimed for this year’s third-largest biotech IPO at $274 million, but underperformed those ambitions by pricing at the low end of its range. The company, which is developing a TYK2 drug and hopes to compete with BMS’ Sotyktu and Takeda’s TAK-279, ended up raising $250 million and fell more than 15% on its first day of trading Friday.
- Grail prepares for public debut, and challenges of going independent, after spinoff from Illumina
- Alexis Borisy and Zach Weinberg’s Curie.Bio secures $380M to support portfolio’s Series A rounds
- Zealand rides obesity wave with $1B raise to advance amylin drug and look for a partner
- Longevity investors look to add $200M to the table with new growth fund
- Schizophrenia biotech LB Pharmaceuticals eyes near-term IPO filing — source
- Commercial-stage blood cancer biotech files for US dual listing
DEALS
- Maze spun out its ALS antisense program into a secretive new company after its proposed licensing deal with Sanofi was blocked by the FTC. The drug is in preclinical development, and is focused on the UNC13A gene, which is associated with the disease.
- Amylyx, searching for post-ALS future, licenses a GLP-1 drug
- Haleon to sell nicotine replacement therapy business to Dr. Reddy’s for $630M
- AbbVie buys Third Rock’s Celsius for $250M to expand IBD pipeline
#ADA24
- Preclinical research in metabolic disease is drawing more investor interest at the American Diabetes Association’s annual meeting than in past years. Gilead, Viking and Biohaven detailed promising preclinical research in obesity and NASH, Katherine Lewin wrote.
- Altimmune says ‘double G’ drug has even better lean mass preservation data in obesity trial update
- Fractyl pitches potential of GLP-1 gene therapy, but pushes clinical trials back to 2025
R&D
- In an early-stage clinical trial with a handful of patients, Lyell Immunopharma’s experimental CAR-T therapy for solid tumor cancers showed hints of efficacy but also faced significant safety concerns, including one death.
- J&J details Phase 3 success of autoimmune disease drug, touting broader range than argenx’s Vyvgart
- Novo Nordisk takes $816M writedown after Phase 3 fail in chronic kidney disease
- Wave Life Sciences touts Huntington’s disease win in Phase 1b/2a trial
- Merck KGaA’s bet on head and neck cancer drug from Debiopharm ends in Phase 3 failure
- Arrowhead shelves one cardiometabolic drug candidate, focuses on another
- Phase 3 breast cancer failure leads to staff cuts, stock drop at G1 Therapeutics
- AstraZeneca’s Imfinzi aces Phase 3 test in bladder cancer, fails another in lung cancer
- J&J’s Tremfya fails giant cell arteritis study, where Roche and AbbVie have succeeded
- Novo reveals more hemophilia A data showing superiority in annualized bleeding rates
- Savara’s rare lung disease treatment passes Phase 3
- Lykos takes another hit as ICER lays out ‘insufficient’ evidence for MDMA-based treatment
PEOPLE
- Ginkgo to cut at least 35% of staff in multi-year restructuring plan
- Q&A: Vertex’s pain research chief on moving beyond opioids, AI and how pharma acquisitions thrive or fail
PHARMA
- After Moderna and Sanofi, Eli Lilly became the latest pharma to tap OpenAI for help. Lilly said generative AI will “accelerate the discovery of novel antimicrobials and the development of custom, purpose-built technologies in the battle against drug-resistant pathogens,” but it didn’t elaborate on how.
- Supreme Court narrowly rejects Purdue bankruptcy plan, says it gives Sacklers too much protection
- CDC advisors expand RSV vaccine recommendations, namely for adults 75 and older
- Why BARDA is handing out hundreds of millions of dollars to long-shot biotechs for Covid
- Moderna’s updated RSV vaccine disappoints with only 50% efficacy
- Grail pioneered multi-cancer tests. But the company’s rough public debut shows the industry could be in trouble
- What Coherus’ $40M sale of its Humira biosimilar says about the crowded market
- UK taps Pfizer over hometown GSK to supply RSV vaccine doses
- HHS reveals 64 drugs up for Medicare inflation rebates next quarter
- Teva to pay $750M to resolve Israel tax disputes
- Amgen calls Colorado’s price controls on Enbrel ‘unconstitutional’
- Korea-based Bridge Biotherapeutics closes Boston office
- Optum Rx may enter biosimilar market to match competitors CVS, Express Scripts
MANUFACTURING
- Novo Nordisk is further boosting manufacturing in the US for its obesity drugs as it injects $4.1 billion for a second fill-finish facility in Clayton, NC. But the Danish company is pulling back elsewhere, canceling plans for a new build in Ireland that was also intended to bolster GLP-1 production, Anna Brown reports.
- EMA calls for member states to lend a hand to ease GLP-1 supply bottlenecks
- GSK’s manufacturing sites in Singapore will use 100% renewable energy by 2025
- ROVI teases acquisition offers for its CDMO unit potentially worth €2B to €3B
FDA+
- Regulators rejected Daiichi Sankyo’s HER3 antibody-drug conjugate patritumab deruxtecan. It’s also a blow to Merck, which had signed on to jointly develop and commercialize the ADC for about $1.5 billion upfront last October. The pair are working on multiple programs together.
- FDA rejects Rocket Pharma’s ultra-rare immune disease gene therapy over manufacturing
- FDA again rejects AbbVie’s continuous infusion therapy for Parkinson’s
- FDA publishes long-awaited guidance on clinical trial diversity
- FDA approves new COPD drug from Verona Pharma
- House committee presses FDA on signs of inconsistent inspections in China and India
- Q&A: FDA medical policy chief talks decentralized trial guidance, improving diversity
- AbbVie and Genmab score accelerated follicular lymphoma nod for Epkinly
- Merck calls ACIP’s pneumococcal recommendation a ‘missed opportunity,’ advocates for expanding age group
- House panel advances bill to allow Medicare to cover obesity drugs
- CMS spells out how states can join new cell and gene therapy access pilot
HEALTH TECH
- Digital health funding flattens out, PitchBook report shows
- Exclusive: Cityblock Health investors buy employee shares in tender offer
- One Medical CEO-turned-VC wants startups that are ‘disruptive from within the ecosystem’
DON’T MISS