A boy participating in the phase II Daylight study of Duchenne muscular dystrophy (DMD) “has passed away suddenly,” according to Pfizer Inc. The participant had received fordadistrogene movaparvovec, a mini-dystrophin gene therapy, in early 2023. The fatal serious adverse event was reported May 3 as a cardiac arrest, Pfizer told BioWorld. Pfizer, together with the independent external data monitoring committee, is reviewing the data to understand the potential cause, the company added.
Homerun success of Novo Nordisk A/S’ semaglutide, which recently became the U.S.’s biggest blockbuster drug, is serving as an “inflection point” for obesity therapeutics and fueling the drive for new and improved therapies, speakers said at Bio Korea 2024 on May 8.
Less than a week ago, executives at Lyra Therapeutics Inc. were looking ahead to “imminent” data from its first phase III study in chronic rhinosinusitis (CRS), testing drug-device candidate LYR-210, a drug-device candidate largely expected to fill a much-needed gap in CRS treatment. On Monday, May 6, they were announcing plans to preserve cash in the wake of the failed Enlighten 1 study, which raised doubts as to the feasibility of the company’s CRS programs, which also include the similarly designed candidate LYR-220.
Not long after Ocular Therapeutix Inc. unveiled positive phase I data in non-proliferative diabetic retinopathy with Axpaxli (axitinib intravitreal implant), Eyepoint Pharmaceuticals Inc. offered less-sunny phase II data from the Pavia trial testing Duravyu (vorolanib intravitreal insert), previously known as EYP-1901, in the same indication.
Top-line data from Glycomimetics Inc.’s pivotal phase III study of uproleselan in 388 patients with relapsed/refractory acute myeloid leukemia missed its primary endpoint. The data crippled the stock, which closed at the low point of its 10-year existence.
The Australian government is investing AU$1.89 billion (US$1.25 billion) in what it is calling a “once-in-a generation transformation” of health and medical research in Australia with $1.4 billion tagged for new research for the Medical Research Future Fund.
Shares of Aeon Biopharma Inc. (NYSE:AEON) closed May 3 down $1.66, or 49%, finishing at $1.70 on word that the planned interim analysis of phase II data with ABP-450 (prabotulinumtoxinA) for preventing chronic migraine showed that the compound did not meet the primary endpoint. The Irvine, Calif.-based firm said it has “immediately commenced cash preservation measures and will review all strategic options.”
In the wake of Novo Nordisk’s semaglutide nabbing the title of the U.S.’s biggest blockbuster drug, it’s little surprise that Amgen Inc.’s obesity candidate, Maritide (maridebart cafraglutide), hogged the stage during the firm’s first-quarter earnings call after market close May 2, with company executives touting promising phase II data and a differentiated profile, sending shares of Thousand Oaks, Calif.-based Amgen (NASDAQ:AMGN) up nearly 12% to close May 3 at $311.29.
Achieving proof of concept and a substantial clinical benefit with its PARP inhibitor stenoparib in advanced recurrent ovarian cancer, Allarity Therapeutics Inc. stopped a phase II trial and is preparing for a registrational study for what is now the company’s only internal drug candidate.
While Nrx Pharmaceuticals Inc.’s oral antidepressant NRX-101 failed, producing a not statistically significant 33% reduction in suicidality along with a 70% reduction in the symptoms of akathisia, the company said it plans to forge ahead and conduct a registrational study.