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Roche said the Phase II results help build the case for advancing CT-388 into late-stage testing, which is set to get underway this quarter.
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Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging field.
From opening new therapeutic mechanisms to repairing neuronal damage, investigational molecules from Ventyx Therapeutics, AC Immune, Gain Therapeutics and more could shape the future of Parkinson’s disease treatment.
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The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
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The multi-billion deal, in which Eli Lilly will acquire Scorpion Therapeutics’ STX-478 program, is a shot in the arm for PI3K treatments, which have had a mixed history over the past few years.
Passage Bio’s workforce reduction could affect about 32 people, leaving the company with 26 employees as it continues evaluating a treatment for frontotemporal dementia with granulin mutations.
On the heels of an FDA approval for its monoclonal antibody Bizengri, Merus will generate three novel cancer-targeting antibodies that it will pass over to Biohaven to link into antibody drug-conjugates.
Biogen’s proposed acquisition comes after two difficult years of regulatory and clinical challenges, during which shares of Sage Therapeutics have fallen by more than 90%.
Among Intra-Cellular’s neuropsychiatric assets is Caplyta, a pill approved for schizophrenia and bipolar depression and proposed for major depressive disorder.
Gilead’s investment will let it assess the therapeutic potential of targeting STAT6, a transcription factor involved in IL-4 and IL-13 signaling, which in turn are known inflammatory targets.
Emraclidine was the centerpiece of AbbVie’s $8.7 billion acquisition of Cerevel in December 2023 but failed two mid-stage trials. Tavapadon, meanwhile, has been a more rewarding asset for the pharma, clearing three Phase III Parkinson’s studies in 2024.
Metsera will use its IPO proceeds to fund the Phase III development of its injectable, ultra-long-acting GLP-1 therapy MET-097i, which last week achieved 11.3% weight loss in a Phase IIa study.
The first major deal of JPM 2025 will give GSK a promising small molecule drug for gastrointestinal stromal tumors.
Benefiting from technological and conceptual groundwork and positive early data, gene therapies are advancing in the clinic for cardiovascular diseases including congestive heart failure, chronic refractory angina and cardiomyopathy.