Day two of the J.P Morgan Healthcare Conference rolled on with positive data from Sling Therapeutics Inc. that is leading the company to a phase III study in treating thyroid eye disease. The privately held company posted top-line efficacy and safety results from a phase IIb/III study of its lead candidate, linsitinib, which hits its primary endpoint with statistical significance at the twice-daily, 150-mg oral dose.
The U.S. FDA posted a draft guidance that would revisit a 2014 final guidance on sex-specific data drawn from medical device clinical trials, expanding the scope to include considerations of gender.
As 2025 makes its debut, one of the topics gaining momentum in health care is the diagnosis and potential treatment of neurodegenerative diseases, a scourge that threatens to overwhelm society and drain treatment budgets around the world. One company looking to find solutions to the oncoming neurodegenerative disease tsunami is privately held Sunbird Bio Inc., which is developing blood-based diagnostic platform for neurological diseases like Alzheimer’s and Parkinson’s disease.
The 2025 edition of Clarivate’s Drugs to Watch features 11 candidates or approved therapeutics that may well revolutionize treatments or become blockbusters. The 12th annual report has a strong track record. Twelve of the 13 drugs from the 2024 Drugs to Watch report have been approved and launched.
Shares of Phio Pharmaceuticals Corp. soared 291% Jan. 13 on news that two patients with cutaneous squamous cell carcinoma had a complete response following treatment with the company’s Intasyl siRNA gene silencing candidate PH-762.
The U.S. FDA posted a draft guidance that would revisit a 2014 final guidance on sex-specific data drawn from medical device clinical trials, expanding the scope to include considerations of gender.
Dyne Therapeutics Inc. is eyeing accelerated approval for its myotonic dystrophy type 1 treatment after reviewing new results from a phase I/II study. DYNE-101, an oligonucleotide antisense and DMPK gene modulator, produced results on disease biomarkers that included DMPK and splicing correction, disease progression reversal on several functional endpoints and a favorable safety profile. The accelerated approval submission could come in the first half of 2026.
Though Pfizer Inc.’s work on a PD-1-targeting antibody has trailed far behind that of its fellow big pharmas, the company could be the first to market in treatment-naïve, high-risk non-muscle invasive bladder cancer on the back of solid phase III data showing subcutaneously administered sasanlimab hit its endpoint of event-free survival. Pfizer anticipates meeting with regulatory agencies to discuss potential filings for what could be the first new treatment for that NMIBC population in decades.
Top-line data from Maat Pharma SA’s phase III study of MaaT-013, an enema microbiome ecosystem therapy for acute graft-vs.-host disease, hit its primary endpoint. The milestone has prompted the company to think about filing an MAA submission for the treatment in Europe sometime in the middle of 2025, which is earlier than it had anticipated.
Yuhan Corp., Oscotec Inc. and Genosco Inc. were three companies with Asian ties to reap benefit from Johnson & Johnson’s stellar top-line Mariposa study results, wherein J&J’s Rybrevant (amivantamab-vmjw) and Lazcluze (lazertinib) combo regimen beat out Astrazeneca plc’s standard of care in non-small-cell lung cancer (NSCLC).
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