With Deciphera Pharmaceuticals Inc.’s positive top-line data in hand from the pivotal phase III study called Motion, Wall Street speculation turned to the comparative safety and regulatory odds for vimseltinib (which analysts often refer to as “vim”) in patients with tenosynovial giant cell tumor (TGCT) not amenable to surgery.
Failing to meet the primary endpoint in its confirmatory phase III Embark trial, Sarepta Therapeutics Inc.’s gene therapy, Elevidys (delandistrogene moxeparvovec), which received accelerated approval in June and was priced at $3.2 million, has one of three fates in its future, all of which are dependent on how the U.S. FDA perceives the data. Based on secondary endpoints showing statistical significance and a recent positive meeting with the agency, Sarepta could continue to market Elevidys under its current label for 4- and 5-year-old ambulatory Duchenne muscular dystrophy (DMD) patients; Sarepta is filing the postmarketing requirement needed to transition from accelerated to full approval.
With Deciphera Pharmaceuticals Inc.’s positive top-line data in hand from the pivotal phase III study called Motion, Wall Street speculation turned to the comparative safety and regulatory odds for vimseltinib (which analysts often refer to as “vim”) in patients with tenosynovial giant cell tumor (TGCT) not amenable to surgery.
The pivotal phase III study of brilaroxazine from Reviva Pharmaceuticals Holdings Inc. for adults with schizophrenia hit its primary endpoint and two key secondary endpoints, following positive phase II data in 2021. The serotonin-dopamine signaling modulator, the company’s lead candidate, is a once-daily treatment. Results from the study showed brilaroxazine demonstrated reductions in all major symptom domains for the patients and the secondary endpoints at week 4 when comparing the 50-mg dose with placebo.
Upbeat phase III findings outweighed less encouraging late-stage trial news, as big pharma provided a mixed bag of cancer findings – with one data batch to form the basis of global approval bids, as Astrazeneca plc with Daiichi Sankyo Co. Ltd. unveiled interim results from a study called Tropion-Breast01. Targeting trophoblast cell surface antigen 2, datopotamab deruxtecan (dato) hit the mark in progression-free survival for patients with hormone receptor-positive, HER2-low or negative breast cancer in the study called Tropion-Breast01.
The majority of patients with acromegaly who switched from therapy with injected somatostatin receptor ligands to Crinetics Pharmaceuticals Inc.’s once-daily, oral drug paltusotine maintained stable levels of insulin-like growth factor 1 in a phase III trial, moving the company’s first-in-class lead closer to becoming the potential new standard of care for the rare hormonal disorder.
Heterozygous familial hypercholesterolemia (HeFH) patients treated with lerodalcibep achieved a 58.6% reduction in LDL-cholesterol at week 24 and a 65% reduction at the mean of weeks 22 and 24 in the phase III trial Liberate-HeFH. The developer, Cincinnati-based Lib Therapeutics Inc., was founded in 2015 when it licensed the technology from Bristol Myers Squibb Co. but is just now emerging from stealth.
Merck & Co. Inc. is pledging major resources on its prospect in the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor space with a phase III program that together will enroll about 17,000 subjects and test oral peptide MK-0616’s effect on tackling low-density lipoprotein cholesterol.
The dent made in Bavarian Nordic A/S’ future revenues after it dropped development of late-stage respiratory syncytial virus (RSV) contender MVA-BN-RSV could be offset by potential sales of its chikungunya virus vaccine, PXVX-0317, if recent phase III immunogenicity data manage to persuade the U.S. FDA that it’s as worthy as Valneva SA’s VLA-1553, the other chikungunya vaccine racing to be first to market.
Not hitting the primary endpoint of a phase III study caused the U.S. FDA to say it would not review Biohaven Ltd.’s NDA for troriluzole to treat spinocerebellar ataxia (SCA), an ultra-rare disorder.
