Uniqure NV rang the bell with a best-case scenario in the pivotal phase I/II study with AMT-130 for the treatment of Huntington’s disease, and shares of the Amsterdam-based firm (NASDAQ:QURE) closed Sept. 24 at $47.50, up $33.84, or 248%. The study met its prespecified primary endpoint, with high-dose AMT-130 turning up a statistically significant slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating Scale at 36 months compared to a propensity score-matched external control.
The second day’s meeting of the U.S. CDC’s Advisory Committee for Immunization Practices (ACIP) took up guidelines related to COVID-19 vaccines, of which an outspoken skeptic is Health and Human Services Secretary Robert Kennedy – who in June fired all 17 members of ACIP and replaced them with names more to his liking.
Pfizer Inc. bounced back in a big way from a GLP-1 trip-up this spring by making known its plan to take over what Metsera Inc. CEO Whit Bernard has called the “scale-obsessed” obesity player that he steers. Pfizer has agreed to pay $47.50 each for all of Metsera’s outstanding shares.
The second day’s meeting of the U.S. CDC’s Advisory Committee for Immunization Practices (ACIP) took up guidelines related to COVID-19 vaccines, of which an outspoken skeptic is Health and Human Services Secretary Robert Kennedy – who in June fired all 17 members of ACIP and replaced them with names more to his liking.
As Wall Street awaits phase IIb data from Kala Bio Inc. with KPI-012 in persistent corneal epithelial defect, odds are being weighed for that candidate – and others in development – against the lone approved treatment used in a market already worth more than $1 billion.
“People have some inability to focus on [Regeneron Pharmaceuticals Inc.’s] pipeline,” which stands as “the most prolific in the industry, I would dare to say,” CEO Leonard Schleifer remarked during the Morgan Stanley health care conference Sept. 8. Most recently, Regeneron bragged on two prospects. The ultra-rare disease fibrodysplasia ossificans progressiva (FOP) took center stage Sept. 17 with news that the phase III Optima trial testing fully human monoclonal antibody garetosmab met its primary endpoint. Separately, Regeneron provided updated analyses of the phase II Courage trial that tested new pairings of GLP-1 receptor agonist semaglutide plus the anti-GDF8/anti-myostatin compound trevogrumab, with or without garetosmab, in obesity.
Although cagrilintide from Novo Nordisk A/S proved less than impressive when paired with GLP-1 agonist semaglutide in a single drug known as Cagrisema, the dual long-acting amylin analogue and calcitonin receptor agonist is looking good as a monotherapy in a subanalysis of the phase III Redefine-1 trial.
Activity in some clinical parameters is leading Atyr Pharma Inc. to plan a sit-down with regulators, even though the tRNA synthetase-derived drug efzofitimod missed its primary endpoint of steroid tapering in the phase III Efzo-Fit study against the interstitial lung disease pulmonary sarcoidosis (PS). CEO Sanjay Shukla said his firm maintains “a high degree of conviction” regarding the candidate. “But I want to make sure we have the best briefing book possible” before consulting with the U.S. FDA, which will happen as soon as possible.
Maze Therapeutics Inc. CEO Jason Coloma said the latest phase I results with MZE-782, a prospect for phenylketonuria (PKU) as well as chronic kidney disease (CKD), bolster the firm’s “conviction to move into phase II” next year in both indications.
Positive phase I results in small-cell lung cancer (SCLC) from Ideaya Biosciences Inc. and partner Hengrui Pharmaceuticals Co. Ltd. with IDE-849, a potentially first-in-class antibody-drug conjugate targeting delta-like ligand 3 (DLL3) and topoisomerase 1, sharpened interest in the DLL3 mechanism, which gave Zai Lab Ltd. a phase Ia/Ib win this year in extensive-stage (ES) SCLC.
