Vor Biopharma Inc.’s trem-cel, a stem cell transplant designed to block the toxicity from cancer treatments, has produced some positive early stage results, including delayed relapse in patients. Phase I/IIa study data showed participants with relapsed/refractory acute myeloid leukemia benefited from trem-cel followed by treatment with Pfizer Inc.’s antibody-drug conjugate cancer fighter Mylotarg (gemtuzumab ozogamicin).
Cytokinetics Inc. CEO Robert Blum said his firm chalked “a watershed moment” during last weekend’s congress of European Society of Cardiology (ESC) in London, where further mid- and late-stage data were disclosed with the heart drug aficamten, a myosin inhibitor. South San Francisco-based Cytokinetics rolled out six presentations, including two late breakers, with four concurrent publications in medical journals.
With Novo Nordisk A/S hobbled by a complete response letter, Eli Lilly and Co. has forged ahead in developing a once-weekly type 2 diabetes treatment. Top-line data from Lilly’s phase III Qwint-1 and Qwint-3 studies of efsitora alfa showed noninferior average levels of blood glucose, A1C, compared to those using another daily basal insulin treatment.
Athira CEO Mark Litton said the firm is “evaluating all the options” after disclosing top-line results from its phase II/III Lift-AD clinical trial of fosgonimeton (fosgo), a hepatocyte growth factor-positive modulator, in patients with mild to moderate Alzheimer’s disease (AD). Javier San Martin, chief medical officer, said the company will schedule “a conversation at the right time” with the U.S. FDA.
Phase I/II results described by one analyst as “stunning” put Vaxcyte Inc. in position for a phase III trial with VAX-31, the firm’s 31-valent pneumococcal conjugate vaccine candidate designed to prevent invasive disease. San Carlos, Calif.-based Vaxcyte’s shares (NASDAQ:PCVX) closed Sept. 3 at $110.15, up $29.39, or 36.4%, on positive top-line results from the study testing the safety, tolerability and immunogenicity of the product in 1,015 healthy adults ages 50 and older.
While Dyne Therapeutics Inc.’s DYNE-251 demonstrated high levels of dystrophin expression and functional improvement in boys with Duchenne muscular dystrophy who are amenable to exon 51 skipping, investors focused on three serious adverse events related to the drug, driving shares (NASDAQ:DYNE) down by 31% on Sept. 3.
With a successful phase III study of plozasiran in hand, Arrowhead Pharmaceuticals Inc. plans to file an NDA with the U.S. FDA for treating the rare genetic disease familial chylomicronemia syndrome. While the data are strong, the company is playing catch-up to Ionis Pharmaceuticals Inc., which has a December 2024 PDUFA for its candidate, olezarsen, in the same indication.
George Medicines Ltd. has published positive data from two phase III studies showing GMRx2, a low-dose triple-drug combination treatment for hypertension, is superior to dual combinations of its components.
Sanofi SA’s brain-penetrant Bruton's tyrosine kinase (BTK) inhibitor, tolebrutinib, met the primary endpoint in the phase III Hercules trial in non-relapsing secondary progressive multiple sclerosis (nrSPMS). The first compound to show reduction in disability accumulation in MS, tolebrutinib delayed the time to onset of confirmed disability progression in people with nrSPMS, a population for which there are currently no approved therapies.
After initially reporting that Xanamem failed to meet the primary endpoint in cognitive attention in the phase IIa Xanacidd cognition and depression trial, Actinogen Medical Ltd. CEO Steven Gourlay said a new analysis shows clinically and statistically significant benefits in depression.
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