LONDON – Freeline Therapeutics Ltd. believes it has found the dose at which FLT-180a, its gene therapy for hemophilia B, will provide a functional cure, promoting expression of factor IX (FIX) blood clotting factor within the normal range.
Sanofi SA said an investigational Bruton's tyrosine kinase (BTK) inhibitor it licensed from Principia Biopharma Inc. in 2017 significantly reduced disease activity associated with multiple sclerosis (MS) as measured by magnetic resonance imaging, meeting the trial's primary endpoint.
An interim analysis of the phase III Recovery study of TNX-102 SL (cyclobenzaprine HCl sublingual tablets) for treating post-traumatic stress disorder (PTSD) compelled Tonix Pharmaceuticals Holding Corp. to halt enrollment in the clinical trial.
Lineage Cell Therapeutics Inc. CEO Brian Culley told BioWorld that concerns about the pace of the company’s dry age-related macular degeneration (AMD) phase I/IIa trial have been alleviated after an independent data safety monitoring board (DSMB) decreed that the study’s protocol-mandated treatment stagger can be removed.
DUBLIN – Shares in PTC Therapeutics Inc. dropped as much as 13% during premarket trading Feb. 6 on eagerly awaited 12-month data for its oral spinal muscular atrophy (SMA) drug risdiplam, which is licensed to Genentech. Although patients with type 2 or type 3 SMA demonstrated statistically significant improvements in motor function, the level of that improvement evidently disappointed some investors.
The National Institute of Allergy and Infectious Diseases has stopped a late-stage HIV vaccine study it sponsored after an interim review by the trial's independent data and safety monitoring board (DSMB) found the regimen failed to prevent HIV.
Shares of Bridgewater, N.J.-based Insmed Inc. (NASDAQ:INSM) closed at $28.88, up $8.34, or 40.6%, on positive top-line results from the global, randomized, double-blind placebo-controlled phase II study called Willow, testing INS-1007 once daily in adults with non-cystic fibrosis bronchiectasis (NCFBE).
BEIJING – China will kickstart a phase III trial Feb. 3 to determine whether patients with 2019-nCoV can be treated with Gilead Sciences Inc.’s NUC inhibitor, remdesivir, which was originally developed for Ebola, four days after a U.S. patient was said to have recovered by using the drug candidate.
Eli Lilly and Co. and Incyte Corp. are working on atopic dermatitis (AD) from several angles and finding success. The most recent example is the positive top-line results from their collaborative phase III trial of baricitinib in treating adults with moderate to severe atopic dermatitis that show the oral selective JAK inhibitor met the study’s primary and secondary endpoints.
Bayer AG and Merck & Co. Inc. took Wall Street by surprise in November with their phase III success testing vericiguat in heart failure (HF), such that the guanylate cyclase stimulator’s odds not only have improved significantly but also in a different way than imagined before.
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