Despite numerous marketed anti-seizure medications in the U.S., patients with epilepsy continue to experience breakthrough seizures and tolerability issues compounded with drug interactions and side effects, leaving room for companies like Praxis Precision Medicines Inc. to fill treatment gaps. The Boston-based company presented phase IIa proof-of-concept data March 26 showing that in an evaluation of photo paroxysmal response, its once-daily oral small-molecule treatment, PRAX-628, resulted in a complete response in three epilepsy patients receiving 45 mg of the drug and four patients receiving 15 mg for a combined complete response rate of 88%. Read More
Updated results from Stoke Therapeutics Inc.’s studies of antisense oligonucleotide STK-001 showed a reduced convulsion seizure frequency in those ages 2 to 18 with Dravet syndrome. The new data, which also showed improved cognition and behavior in participants, substantially boosted the company’s stock value and caused analysts to applaud. Read More
Clarity Pharmaceuticals Ltd. raised AU$121 million (US$79.29 million) through a private placement that will fund the company’s radiopharma pipeline comprising targeted copper therapies out to early 2026. Read More
The fast-shifting obesity space gained more clinical results as Viking Therapeutics Inc. shared data from its phase I, multiple ascending-dose trial with oral VK-2735, a dual agonist of the glucagon-like peptide 1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) receptors. Read More
Nuvation Bio Inc. said on March 25 it will acquire Anheart Therapeutics Ltd., a U.S.- and China-based precision oncology company, in an all-stock transaction, adding Anheart’s ROS1-inhibiting lung cancer drug, taletrectinib (AB-106), to its pipeline. Read More
Regenerative medicine company Mesoblast Ltd. saw its stock shoot up 45% on the news that the U.S. FDA is satisfied with the additional data submitted from the company’s phase III study for remestemcel-L for treatment of adults with steroid-refractory acute graft-vs.-host disease (SR-aGVHD) to support filing a BLA in pediatric patients with SR-aGVHD. Read More
Europe may still await its first disease-modifying Alzheimer’s drug after the EMA postponed its decision on Leqembi (lecanemab, Biogen Inc./Eisai Co. Ltd) on March 22, but leading members of the World Dementia Council were in an optimistic mood when they convened in London four days later. Read More
While some states are beginning to double down on the prices they pay for prescription drugs, the state of Colorado is taking it to a whole new level with its Prescription Drug Affordability Review Board that was empowered to set maximum prices of prescription drugs it considers “unaffordable.” Read More
The U.S. Department of Health and Human Services (HHS) was quite vocal in its statement regarding a recent hospital cybersecurity breach, but HHS recently suffered an undisclosed data breach that cost $7.5 million in taxpayer monies. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Apogee, Astria, Axsome, Beyondspring, Cassava, Invivyd, Matinas, Syros. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Abbvie, Advicenne, Astrazeneca, Bluejay, Daiichi Sankyo, Esperion, Invivyd, Regeneron, Tonix. Read More
