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.
Potential implications for others in the space, including Pfizer Inc. and Bridgebio Pharma Inc., became the subject of buzz after Alnylam Pharmaceuticals Inc. provided details from the Helios-B phase III study with RNAi candidate vutrisiran, in the works for ATTR amyloidosis with cardiomyopathy.
Neurocrine Biosciences Inc.’s top-line phase II data, though billed as promising, wasn’t enough to please observers on Wall Street, who stacked the data in schizophrenia with NBI-1117568 (NBI-'568) against others in the space. Shares (NASDAQ:NBIX) closed Aug. 28 at $123.76, down $28.79, or 18.9%.
About two months after Astrazeneca plc said its application for sipavibart (AZD-3152) had been accepted by the EMA for pre-exposure prophylaxis (PrEP) against COVID-19 in immunocompromised people, Invivyd Inc. unveiled positive 180-day exploratory efficacy data from the company’s ongoing Canopy phase III trial with Pemgarda (pemivibart) in the same indication – and made known less happy news from regulators on the other side of the pond.
Headlines in March about Cartesian Therapeutics Inc. reminded investors of the firm’s already-intriguing push with Descartes-08, an autologous anti-BCMA mRNA CAR T therapy, in the works for myasthenia gravis and systemic lupus erythematosus. Excitement generally is mounting around prospects for CAR T therapies in autoimmune disease, where developers aplenty are pursuing early stage opportunities.
The long struggle by Boston-based I2o Therapeutics Inc.’s business unit Intarcia Therapeutics to get long-lasting exenatide for diabetes onto the market ended with a final thumbs-down from the U.S. FDA because of safety concerns. At issue was ITCA-650, a twice-yearly implantable exenatide-device combo meant to improve glycemic control in adults with type 2 diabetes.
As Bridgebio Pharma Inc. awaits word from the U.S. FDA regarding the small-molecule transthyretin (TTR) stabilizer acoramidis for TTR-mediated amyloidosis cardiomyopathy, the firm closely followed this week’s headline about another spinout with news that the Palo Alto, Calif.-based company has signed a joint research pact with Hitgen Ltd., of Chengdu, China.
Regeneron Pharmaceuticals Inc. suffered an unsurprising hitch in the form of a complete response letter (CRL) related to linvoseltamab, a bispecific antibody designed to bridge B-cell maturation antigen on multiple myeloma cells containing CD3-expressing T cells, for relapsed/refractory multiple myeloma.
A week after the U.S. FDA spurned Lykos Therapeutics Inc.’s bid for approval of a therapy in post-traumatic stress disorder (PTSD), shares of Alzamend Neuro Inc. (NASDQ:ALZN) zoomed upward on more news in the indication with a next-generation, cocrystal lithium compound AL-001. Atlanta-based Alzamend’s stock closed Aug. 19 at $5.85, up $3.82, or 188%.
