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Will Sarepta’s big gene therapy expansion set a precedent?
In a closely-watched decision, the FDA broadened the label for Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy to patients ages 4 and older. The approval goes beyond the patient population in which the company reported data for its randomized clinical trials. As the agency’s memos revealed, CBER Director Peter Marks again overruled other FDA leaders in pushing for the label expansion. And that’s prompting new questions about just how the agency will approach similar drugs in the future, Lei Lei Wu reports.
Inside Iambic’s AI cycle
Iambic Therapeutics doesn’t have the head-turning megaround money of other AI bios, but it’s built itself into a force to watch by focusing on the application of its models. Endpoints’ Andrew Dunn recently visited Iambic’s new San Diego headquarters to see a demonstration of its AI software and to talk with its leaders about how they make medicines.
Vertex, Lilly at #ADA24
Kicking off the annual scientific sessions of the American Diabetes Association, Eli Lilly spelled out how its weight loss treatment Zepbound helped at least 43% of patients with obstructive sleep apnea and obesity resolve symptoms of the breathing disorder in late-stage trials. Meanwhile, Vertex Pharmaceuticals reported a third type 1 diabetes patient going fully insulin-independent for at least a year after taking its islet cell therapy in an early-stage trial. Katherine Lewin reports from Orlando.
BioNTech’s ADC on hold
The FDA placed an early-stage BioNTech trial on partial clinical hold after researchers observed multiple deaths in studies involving an antibody-drug conjugate partnered with MediLink Therapeutics. Regulators said the ADC could expose patients to “unreasonable and significant risk.”
Roche bets on RNA editing
Roche tapped Boston startup Ascidian Therapeutics for access to a sweeping form of RNA editing that replaces large swathes of errant genetic code. The deal is centered around developing RNA exon editing for neurological diseases. Ascidian will get an initial $42 million and up to $1.8 billion in potential milestone payments.
SPOTLIGHT
R&D
- Takeda’s enzyme therapy soticlestat, which aims to balance brain cholesterol and reduce seizures, failed two Phase 3 trials in certain forms of epilepsy. The Japanese drugmaker had planned to submit the candidate to regulators by the end of this fiscal year. The news sank shares of Ovid Therapeutics, which gave full rights to the drug back to Takeda in 2021.
- AstraZeneca’s Truqap combo flops in Phase 3 triple-negative breast cancer trial
- Aerovate’s stock craters after PAH drug fails Phase 2 study
- Jazz’s stock dips after mid-stage fail in essential tremor
- Gilead’s twice-a-year injection fully prevented HIV in Phase 3 study with cisgender women
- Zealand Pharma hits early efficacy goal for amylin weight loss candidate
- Aphaia hits primary endpoint with oral glucose drug in mid-stage prediabetes trial
- Silence’s siRNA candidate for cardiovascular disease continues to deliver in Phase 2 trial
- Updated: Taysha teases early gene therapy data for Rett syndrome
- PTC touts Phase 2 biomarker data for Huntington’s drug, showing dose-dependent effect at one year
- J&J’s Sjögren’s disease drug shows promise in Phase 2, boosts prospects of FcRn assets in the autoimmune disease
- Marinus reports mixed Phase 3 results on IV seizure drug
- Intra-Cellular reports a depression win, setting up future competition with AbbVie’s Vraylar
#EHA24
- Nurix’s protein degrader overcomes resistance to BTK mutations in small leukemia study
- With new data, AstraZeneca makes the case for its BTK inhibitor in a first-line setting
- Gilead cancer drug magrolimab showed worrying trends around death
FINANCING
- Marea Therapeutics, a Third Rock Ventures-incubated startup, debuted with $190 million to advance a Novartis-licensed antibody for a genetic target for cardiometabolic diseases. Josh Lehrer, formerly of Graphite Bio, is the CEO, and Ethan Weiss is its chief scientific officer and a co-founder.
- Gilead tested an antibody many times. A small biotech salvages it, with $40M and ‘conviction’ elsewhere
- Life sciences investor Decheng Capital targets $700M for fifth fund
- Deerfield backs $81M round for biotech developing antifungal therapy with less toxicity
- iOnctura raises €80M to advance early-stage rare eye cancer treatment
- Blank check, please? Three healthcare SPACs reveal their IPO plans
DEALS
- Shortly after securing its first FDA approval and bagging $108 million from a priority review voucher sale, Day One Biopharmaceuticals said it will will dish out $55 million upfront to Shanghai-based MabCare Therapeutics for an antibody-drug conjugate.
- Tectonic enters public markets in one of few biotech reverse mergers this year
- As it deepens immunology bets, Sanofi inks small molecule pact with Belharra
PHARMA
- Bristol Myers Squibb won accelerated approval to take its KRAS inhibitor to colorectal cancer patients. Krazati will now be available for use with Eli Lilly’s Erbitux (cetuximab) for certain locally advanced or metastatic patients who have been previously treated and have a KRAS G12C mutation.
- GSK readies Blenrep’s commercial re-launch in the US, and a push in second line
- Amgen nabs new Blincyto approval for certain type of leukemia
- FDA approves endometrial cancer indications for Keytruda, Imfinzi
- Argenx wins second FDA approval for Vyvgart in autoimmune nerve disease
- AbbVie adds ulcerative colitis to Skyrizi label
FDA+
- The FDA’s biologics center director Peter Marks said the agency is considering the creation of a rare disease hub to better combine the expertise of both its drug and biologics experts, Lia DeGroot reports from the Drug Information Association’s annual meeting in San Diego.
- FTC backs plan to target more patent settlements it sees as anticompetitive
- FDA loosens side effect reporting requirements for some CAR-T therapies
- FDA to pharma companies: Using CRO Synapse’s studies ‘unacceptable’
- Merck’s 21-valent pneumococcal vaccine for adults wins FDA approval
- After two patient deaths from presumed sepsis, FDA puts partial hold on Zentalis studies
- FDA makes it easier for biosimilars to obtain interchangeability designations
- Lexicon resubmits type 1 diabetes drug to FDA five years after rejection
- International society criticizes Utah law for permitting unapproved stem cell therapies
LAW
- Eli Lilly is filing a fresh round of lawsuits against medspas, wellness centers and other entities that it claims are selling compounded versions of its tirzepatide-based drugs Mounjaro and Zepbound. The legal action is part of a broader campaign against unapproved, fake or counterfeit tirzepatide, as well as improper uses of its drugs.
- Janssen ordered to pay $150M over false claims around HIV drugs
- Teva and US government discuss settling kickback lawsuit over Copaxone
MANUFACTURING
- As a part of Takeda’s larger plan to boost production of its plasma-derived therapies, the company is allocating $230 million to expand its manufacturing site in Los Angeles.
- J&J cements footprint in Italy with €580M in investments over the next 5 years
- Corrected: Emergent sells Baltimore facility for $30M
- US, EU pledge $2B+ to help boost vaccine manufacturing in Africa
- FDA ships two Form 483s to Dr. Reddy’s for two separate sites in India
- Samsung Bio and Baxter dial up manufacturing deal to $223M
- Hikma to take over several assets from Xellia, including a US manufacturing site
HEALTH TECH