Phase II
Roivant Sciences will wind down operations for Hemavant, which was formed two years ago to advance RVT-2001 as a potential first-in-class treatment for transfusion-dependent anemia in patients with lower-risk myelodysplastic syndromes.
Following promising Phase IIb studies, Takeda will advance its oral ORX2 agonist TAK-861 into Phase III studies for narcolepsy type 1, while nixing the candidate’s development in narcolepsy type 2.
The monoclonal antibody Nipocalimab, which J&J acquired from Momenta Therapeutics, met its primary endpoints in mid-stage and late-stage trials in myasthenia gravis and Sjögren’s disease.
Topline results from a mid-stage study show that 4D Molecular Therapeutics’ investigational gene therapy cut annual rates of Eylea injections by 85% and 89% for the low and high doses, respectively.
With one disease-modifying therapy already reaching patients and another expected to soon, several biopharma companies anticipate key data for novel assets in the coming 12 months.
Biotech companies are exploring multiple targets in the fight against neuroinflammation. Experts say these projects could yield the next breakthrough in treating Alzheimer’s disease.
Gene therapy company Sarepta Therapeutics provided additional data for SRP-5051 in treating Duchenne muscular dystrophy patients who are amenable to exon 51 skipping.
In a Phase I/II trial, an 11-year-old boy gradually regained hearing—eventually reaching normal range for some frequencies at 30 days—following treatment with Eli Lilly’s dual adeno-associated viral vector-delivered gene therapy.
From ALS to depression to Huntington’s disease, many neuroscience-focused companies are anticipating key data over the next few months.
Anavex Life Sciences’ stock dropped by more than a third in value on Tuesday after results for a Phase II/III Rett syndrome study failed to impress investors.
PRESS RELEASES