Drug Development
Leading companies spent $1.4 billion upfront on licensing deals and embarked on vast R&D programs. Clinical setbacks mean many companies are unlikely to ever recoup their investments.
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Here’s how companies can ensure they’re in compliance with new requirements that go into effect in August.
The medium-sized biopharma is showing off new results from dordaviprone and Zepzelca, both of which were acquired through Jazz Pharmaceuticals’ dealmaking over the last five years.
R&D spending across the global pharmaceutical sector climbed 1.5% in 2024, according to unreleased data from Evaluate Pharma.
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At 12 weeks, weight loss ranged from 2.6% to 11.3%, compared to a gain of 0.2% in the placebo group. Guggenheim analysts were also impressed by the tolerability profile.
Analysts at Jefferies give Roche and Prothena’s Phase III study just a 25% to 40% probability of success.
Analysts at Truist Securities called J&J’s CAR T readout “compelling,” noting that the efficacy figures could position the cell therapy as a formidable competitor to the current standard of care, Gilead’s Yescarta.
Sarepta’s shares crashed 41% in premarket trading Monday morning to $21.01 after the biotech reported a second death from acute liver failure, a known side effect of adeno-associated virus-based gene therapies.
AstraZeneca, Pfizer and more are leveraging the computational power of AI to better design trials, predict the potential efficacy and safety profiles of their molecules and synthesize massive multi-omic information to gain a more complete understanding of challenging cancers.
While cancelled NIH grants and regulatory uncertainty are less hospitable to clinical research in the U.S., Europe must play its cards right to attract more studies.
Guggenheim Partners called Insmed’s Phase IIb readout “impressive across the board,” with an efficacy profile that positions TPIP to be best-in-class in pulmonary arterial hypertension.
Gene therapies have ridden investor mania to huge valuations but commercialization challenges have pushed market caps to the floor. At a roundtable last week, FDA leaders promised faster approvals and broad support to the industry.
Gilead underscored its faith in the combo therapy and pledged to work with regulators to resolve the hold, which has paused five clinical trials. Gilead also stressed that the hold does not impact any other assets in its HIV pipeline.
Avidity has been given the go-ahead by the FDA to request accelerated approval of delpacibart braxlosiran—potentially the first disease-modifying treatment for facioscapulohumeral muscular dystrophy—with an application in the second half of 2026.