With no drugs approved by the U.S. FDA for treating Becker muscular dystrophy, Edgewise Therapeutics Inc. reported positive two-year, phase Ib data looking at patients’ ability to physically function, plus biomarker data.
With an IND filing in systemic lupus erythematosus expected during the third quarter of 2024, Cullinan Therapeutics Inc. changed its name from Cullinan Oncology Inc. and detailed its plans to target autoimmune diseases with CLN-978, a CD19xCD3 T-cell engager.
Robust top-line phase III data showed Intra-Cellular Therapies Inc.’s marketed oral atypical antipsychotic drug, Caplyta (lumateperone), achieved statistically significant and clinically meaningful best-in-class results, potentially expanding its reach into the billion-dollar major depressive disorder market. The company’s shares (NASDAQ:ITCI) surged to a 52-week high of $84.89 throughout the day, closing at $79.84, up 23.3%, or $15.08, on April 16.
Wall Street may not have responded as positively as Ultragenyx Pharmaceutical Inc. would have liked after the firm unveiled new data from the phase I/II study with GTX-102 for the treatment of Angelman syndrome (AS). Patients in expansion cohorts A & B treated with a set dose and regimen of the intrathecally delivered antisense oligonucleotide (ASO) showed rapid and clinically meaningful improvement across multiple domains.
Investors anxious to see data from Marinus Pharmaceuticals Inc.’s phase III Raise study testing intravenous ganaxolone in refractory status epilepticus (RSE) will have to wait a little longer, as the trial failed to meet the predefined criteria for stopping at the interim analysis, sending shares (NASDAQ:MRNS) down nearly 83%.
Enlivex Therapeutics Ltd. extolled the top-line “positive indication of effect and safety” from the phase II study of Allocetra in treating sepsis and sepsis shock, but the market took another view of the clinical trial. In an analysis of eligible adult patients from the cell therapy’s multicenter, randomized, placebo-controlled, dose-finding study of 120 enrolled patients, the company also reported low mortality rates.
With the recent removal of its amyotrophic lateral sclerosis drug from the market, Amylyx Pharmaceuticals Inc. is looking to revive investor interest with interim data from its phase II Helios study testing the same drug, AMX-0035 (sodium phenylbutyrate plus taurursodiol), in Wolfram syndrome, a rare indication in which Amylyx could be leading the charge.
Stable disease in about half the patients tested wasn’t enough for Wall Street, and shares of Vincerx Pharma Inc. (NASDAQ:VINC) nosedived by $3.72, or 78%, to close April 9 at $1.06 on the disclosure of preliminary phase I data with small-molecule drug conjugate VIP-236 in metastatic solid tumors.
With two respiratory syncytial virus (RSV) vaccines approved by the U.S. FDA in 2023 and a third nearing its May PDUFA date, decades of research has finally provided infants and older adults protection from the disruptive and sometimes deadly virus. But what about people in the middle, particularly those with certain chronic medical conditions? New York-based Pfizer Inc. rolled out phase III data April 9 showing that its approved RSV vaccine, Abrysvo (RSVpreF), met primary endpoints in adults ages 18 to 59 who were at high risk of RSV.
The U.S. FDA accepted for review Daiichi Sankyo Co. Ltd.’s and Astrazeneca plc’s BLA for datopotamab deruxtecan to treat adults with unresectable or metastatic hormone receptor-positive, HER2-negative breast cancer who have received prior systemic therapy for unresectable or metastatic disease.
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