Robust top-line phase III data showed Intra-Cellular Therapies Inc.’s marketed oral atypical antipsychotic drug, Caplyta (lumateperone), achieved statistically significant and clinically meaningful best-in-class results, potentially expanding its reach into the billion-dollar major depressive disorder (MDD) market. The company’s shares (NASDAQ:ITCI) surged by 28%, or $18.23, midday to $82.99. Caplyta, which gained U.S. FDA approval for schizophrenia in 2019 and for bipolar depression in 2021, met the primary endpoint of change from baseline at week six on the Montgomery-Asberg Depression Rating Scale total score vs. placebo, with a 4.9-point reduction, higher than improvements seen with four leading marketed products for MDD.

Edgewise posts positive phase I data in stabilizing Becker muscular dystrophy

Edgewise Therapeutics Inc. has reported positive two-year, phase I data looking at Becker muscular dystrophy patients’ ability to physically function, plus biomarker data. Adults treated with the oral, small-molecule EDG-5506 (sevasemten) saw their physical ability to lead an active daily life stabilized in the past two years, along with significant decreases in key muscle-damage biomarkers. Becker, like Duchenne muscular dystrophy, is defined by progressive muscle weakness and wasting. It is tied to mutations in the DMD gene. There are no drugs approved by the U.S. FDA for treating Becker. The rare disease typically is managed by corticosteroids, heart medication and physical therapy.

Eubiologics wins WHO prequalification for oral cholera vaccine

South Korea’s Eubiologics Co. Ltd. said it gained the World Health Organization’s (WHO) prequalification designation on April 16 for its simplified oral cholera vaccine, approved as Euvichol-S. Developed jointly with Seoul-headquartered International Vaccine Institute, Euvichol-S is a simplified formulation of Euvichol-Plus, the company’s oral cholera vaccine first WHO-approved in August 2017 as the world’s first plastic vial-packaged oral cholera vaccine. A streamlined manufacturing process that resulted in Euvichol-S will help boost vaccine production by about 40% over Euvichol-Plus, the Gangnam-gu, Seoul-based company said.

Radiopharma glioma imaging agent fast-tracked as Telix preps NDA

The U.S. FDA granted fast track designation to Telix Pharmaceutical Ltd.’s TLX101-CDx for glioma imaging as the firm prepares to file its NDA in the first half of 2024, a Telix spokesperson told BioWorld. The Melbourne-based company’s glioma imaging product, TLX-101-CDx (Pixclara, ¹⁸F-floretyrosine, ¹⁸F-FET) characterizes progressive or recurrent gliomas using positron emission tomography. Telix is in the final stages of preparing its NDA for TLX-101-CDx in this initial indication in both adult and pediatric patients. The newly awarded fast track status enables expedited review and closer consultation with the FDA during the review process.

Neurocrine advances Nxera’s NBI-1117568 in schizophrenia

Nxera Pharma Co. Ltd announced that its partner, Neurocrine Biosciences Inc., is advancing NBI-1117568 to phase II trials for treatment of schizophrenia and other neuropsychiatric disorders. An oral selective muscarinic M4 receptor agonist, NBI-1117568 successfully completed a long-term preclinical toxicity program to support safe, chronic dosing of NBI-1117568 in future clinical trials. The milestone triggered a $15 million payment to Nxera from Neurocrine.

BioWorld Insider Podcast: Biopharma execs seek to avoid pain points in AI integration

Because artificial intelligence is such a new technology it comes with a mountain of unknowns. Integrating it into a pharmaceutical company presents a unique series of challenges, as a new survey from Verix shows. The report is based on responses from directors, vice presidents and C-suite executives in sales, marketing and brand management at pharmaceutical companies in the U.S. that have more than $100 million in annual revenue. Doron Aspitz, the CEO of Verix, talked about the new findings with the BioWorld Insider podcast and the profound shift that executives face as the new technology takes over an entire industry.

Also in the news

Acorda, Aim Immunotech, Applied, Aptevo, Asher, Athira, Black Bird, Calidi, Cancervax, Chimeron, Clearside Biomedical, Curevac, Endeavor, Evergreen Theragnostics, Faron, Foghorn, Fresenius, Geneventiv, Inovio, IO, KBI, Nacuity, Neurvati, Obsidian, Ocular, Outlook, Recursion, Turnstone, Vittoria, Voyager