Biogen and Stoke Therapeutics collaborate on zorevunersen for Dravet syndrome
- Biogen and Stoke Therapeutics have partnered to develop and commercialize zorevunersen for Dravet syndrome outside the US, Canada, and Mexico.
- Zorevunersen is an investigational antisense oligonucleotide targeting the SCN1A gene, with a Phase 3 study planned for 2025.
- Stoke will receive $165 million upfront, with potential milestone payments up to $385 million and royalties on sales.
- The collaboration aims to address unmet needs in Dravet syndrome, a severe genetic epilepsy with no approved disease-modifying therapies.
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Ultragenyx's UX111 gene therapy receives FDA priority review for Sanfilippo syndrome type A
- The FDA has accepted the Biologics License Application (BLA) for UX111, granting it Priority Review with a PDUFA date of August 18, 2025.
- UX111 is an AAV gene therapy aimed at treating Sanfilippo syndrome type A, a rare lysosomal storage disease affecting the brain.
- Clinical data shows UX111 significantly reduces heparan sulfate levels in cerebrospinal fluid and improves cognitive and communication scores.
- The therapy has received multiple designations, including Regenerative Medicine Advanced Therapy and Orphan Drug status in the U.S. and EU.
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Supernus announces topline results from phase 2b study in adults with treatment resistant depression
- The Phase 2b study of SPN-820 in adults with treatment-resistant depression did not show a statistically significant improvement in depressive symptoms compared to placebo.
- The primary endpoint was the change in the Montgomery-Ã…sberg Depression Rating Scale (MADRS) total score from baseline to Week 4.
- SPN-820 was well-tolerated with few adverse events, consistent with previous studies.
- Supernus plans to analyze the data further and discuss the future of the program with its development partner, Navitor Pharmaceuticals.
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Axsome Therapeutics reports fourth quarter and full year 2024 financial results and provides business update
- Axsome reported 4Q and full year 2024 net product revenue of $118.8 million and $385.7 million, marking 66% and 88% year-over-year growth.
- Symbravo was approved in the U.S. for acute migraine treatment, with commercial availability expected in four months.
- Phase 3 trials for AXS-05 in Alzheimer's agitation and AXS-12 in narcolepsy showed positive results, with NDA submissions planned for 2H 2025.
- Topline results for Phase 3 trials of solriamfetol in ADHD and MDD are expected in 1Q 2025.
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Ionis reports fourth quarter and full year 2024 financial results
- Ionis launched TRYNGOLZA for familial chylomicronemia syndrome, marking its first independent medicine launch.
- The company plans three more independent launches, including donidalorsen for hereditary angioedema in 2025.
- Ionis reported total revenue of $705 million for 2024, with operating expenses of $1,180 million.
- Cash and short-term investments stood at $2.3 billion as of December 31, 2024, supporting future launches.
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