Magnet Biomedicine Inc. emerged from stealth mode and pulled down a $50 million series A round co-led by founding investor Newpath Partners alongside Arch Venture Partners. The firm is advancing molecular glue discovery by way of rational selection and design, looking past known protein-protein interactions and what Magnet calls “tangential” degradation approaches to analyze the broader protein landscape and ultimately pair targets with rationally chosen presenters in the tissue where the disease manifests.
Iveric Bio Inc.’s regulatory win Aug. 4 drew attention to the already hot eye-disease space, where intriguing new developments include the possibility of an oral therapy for Stargardt disease. Belite Bio Inc. in late July finished enrollment of a phase III study with once daily tinlarebant, a retinol binding protein 4 antagonist for Stargardt’s. Data from 90 adolescent subjects in the study called Dragon are due in mid-2024.
“We’re going to battle,” PTC Therapeutics Inc. CEO Matthew Klein said, responding to a surprise negative opinion from the EMA’s Committee for Medicinal Products for Human Use on converting the conditional marketing authorization to full status for Translarna (ataluren) in the treatment of nonsense mutation Duchenne muscular dystrophy. The opinion applies to the renewal of the existing conditional authorization, too.
Though data won’t be available for a few years, the disclosure in mid-July that Grifols SA completed enrollment in the phase III study called Sparta caused some ears to perk up in the alpha-1-antitrypsin deficiency (AATD) space, where a number of contenders are busy.
One hepatitis delta virus player (HDV) has dropped out after Eiger Biopharmaceuticals Inc.’s phase III effort with peginterferon lambda turned up safety issues, while others remain busy in a space highlighted during last November’s meeting of the American Association for the Study of Liver Diseases. Shares of Palo Alto, Calif.-based Eiger (NASDAQ:EIGR) closed Sept. 13 at 41 cents, down 28 cents, or 40.6%, on word that the company is stopping the Limt-2 study in patients with chronic HDV.
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.
