Drug Development
The mid-stage disappointment in Alzheimer’s disease delivers another blow to Neuphoria Therapeutics, which in November last year was forced to launch a strategic business review after a Phase 3 trial in social anxiety disorder failed.
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A recent FDA reversal sparked new hope for patients with Huntington’s disease. Flying under the radar, Skyhawk Therapeutics revealed 12-month functional data from a midstage trial of its own candidate showing improvements on a key disease measurement scale.
Recent approvals for Corcept Therapeutics and Merck have injected momentum into the space, where GSK, Allarity Therapeutics, OSE Immunotherapies and others are advancing their own candidates.
The FDA plans to hold an advisory committee meeting to discuss Capricor Therapeutics’ application for deramiocel, which the agency rejected last July. The news surprised CEO Linda Marbán, who told BioSpace the FDA has not communicated any issues of concern with the company’s resubmitted application.
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Andembry is the first monthly Factor XIIa inhibitor for the prevention of HAE attacks and will be available to patients “before the end of June,” CSL announced.
Jefferies analysts said on Tuesday that Ventyx could leverage its mid-stage data for VTX3232 to position the oral drug candidate as a treatment for other neurodegenerative diseases, including Alzheimer’s.
Venclexta, when combined with azacitidine, elicited an overall survival benefit below 10% in patients with myelodysplastic syndromes.
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.