Neurocrine Biosciences Inc.’s positive top-line data from the phase III study called Cahtalyst in classic congenital adrenal hyperplasia (CAH) further whetted Wall Street’s appetite for soon-to-come results in the same indication from Spruce Therapeutics Inc.
Moderna Inc. has more than COVID-19 vaccines in the hopper, and the company aims to add still more oomph by way of an oncology deal with Immatics NV that could be worth more than $1.7 billion for the latter, which banks $120 million up front and stands to collect research funding as well.
Positive phase III data from Cymabay Therapeutics Inc. with seladelpar, the peroxisome proliferator-activated receptor for primary biliary cholangitis (PBC), could mean trouble for Intercept Pharmaceuticals Inc.’s second-line therapy, the farnesoid X receptor agonist Ocaliva (obeticholic acid), which Cymabay aims to replace with its compound as the preferred choice.
Wall Street’s hoped-for phase III derisking event from Insmed Inc. materialized, and shares of the firm (NASDAQ:INSM) closed Sept. 5 at $26.37, up $3.73, or 16.5%, on positive top-line results from the study called Arise with inhaled Arikayce (amikacin) in patients with newly diagnosed or recurrent nontuberculous mycobacterial lung infection by Mycobacterium avium complex (MAC) who had not started antibiotics. “We crushed it” on culture conversion with Arikayce, CEO William Lewis said. “We could not be happier about the results of this study. It exceeded all of our expectations on every front.”
Roche Holding AG’s Genentech subsidiary has broken new ground with a victory in phase III testing of the oral, anaplastic lymphoma kinase (ALK) inhibitor Alecensa (alectinib) in early stage, ALK-positive non-small-cell lung cancer (NSCLC). The drug, well known to doctors in the advanced setting, was compared with platinum-based chemotherapy and met its primary endpoint of disease-free survival at a prespecified interim analysis.
Phathom Pharmaceuticals Inc. made recent headway with its new mechanism of action in gastroesophageal reflux disease (GERD) even as further, disturbing discoveries in the space are seeing daylight. Florham Park, N.J.-based Phathom turned over to the U.S. FDA the requested six-month stability data related to reformulated vonoprazan tablets, and the numbers remained consistent with three-month data, reflecting N-nitroso-vonoprazan nitrosamine control and levels comfortably more than tenfold below the acceptable daily intake limit.
The paper published June 19 in Nature Genetics that described a genome-wide analysis to narrow down the implicated pathogenic signaling pathways and “prioritize drug targets for IgA nephropathy [IgAN]” no doubt proved of great interest to developers, plenty of which are busy in the space.
“Sometimes the market gets ahead of the science,” said Dennis Purcell, founder of Aisling Capital, pointing to the way backers of genomics firms “went nuts” in the early 2000s. “I think we’re in a position today where the science is ahead of where the market is.” The remarks by venture capital (VC) expert Purcell came during a virtual salon hosted Aug. 30 by Demy-Colton and titled “VC Trends in Healthcare Investing: Current Pulse Check.” Panelists acknowledged the currently unfavorable financing environment but sounded upbeat about the industry’s path forward.
The list of 10 part D Medicare drugs listed by the Centers for Medicare & Medicaid Services (CMS) as eligible for negotiation raised some eyebrows on Wall Street, but proved mostly in accord with what the industry expected. Under the Inflation Reduction Act, Medicare can for the first time bargain with drug companies. The back-and-forth begins this year, carrying into next year, and the agreed-upon prices will take effect in 2026.
About two weeks after chikungunya virus (CHIKV) vaccine contender Bavarian Nordic A/S provided phase III data with its prospect, rival Valneva SE rolled out positive phase III safety findings in adolescents with its single-dose candidate VLA-1553. Results from the Saint-Herblain, France-based company’s first trial in an endemic area with people previously infected with CHIKV showed the product was generally safe and well-tolerated in subjects aged 12-17 years, regardless of previous infection by the mosquito-borne disease, carried mainly by Aedes aegypti and Aedes albopictus. Immunogenicity data from the study are expected in November.
