Astellas and Sangamo Therapeutics announce capsid license agreement to deliver genomic medicines for neurological diseases
- Astellas gains rights to use Sangamo's STAC-BBB capsid for up to five neurological disease targets.
- Sangamo will receive a $20 million upfront fee and could earn up to $1.3 billion in milestone payments.
- The STAC-BBB capsid has shown effective blood-brain barrier penetration in nonhuman primates.
- Astellas will handle research, development, and commercialization of the gene therapy products.
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Mesoblast’s Ryoncil is the first U.S. FDA-approved mesenchymal stromal cell therapy
- Ryoncil (remestemcel-L) is the first FDA-approved mesenchymal stromal cell (MSC) therapy in the U.S.
- Approved for treating steroid-refractory acute graft versus host disease (SR-aGvHD) in children aged 2 months and older.
- In a Phase 3 trial, 70% of children with high severity SR-aGvHD achieved an overall response by Day 28.
- Ryoncil's immunomodulatory effects suggest potential for other inflammatory conditions.
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Genentech’s phase IIb study of prasinezumab suggests possible benefit in early-stage Parkinson’s disease
- The Phase IIb PADOVA study involved 586 participants with early-stage Parkinson’s disease, treated for at least 18 months.
- Prasinezumab missed the primary endpoint of time to confirmed motor progression but showed potential efficacy with a HR=0.84.
- A more pronounced effect was observed in participants treated with levodopa, with a HR=0.79.
- The study will continue with open-label extensions to further explore the effects, and full results will be presented at an upcoming medical meeting.
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Insitro receives $25 million in milestone payments from Bristol Myers Squibb for ALS target discovery
- Insitro received $25 million from Bristol Myers Squibb for achieving discovery milestones and selecting a novel ALS target.
- The collaboration, initiated in 2020, aims to discover new therapies for ALS, a disease with no modifying treatments.
- Insitro's platform uses machine learning to identify and validate novel gene targets for ALS.
- Bristol Myers Squibb will advance the program through clinical development, with potential for further target selections.
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Praxis Precision Medicines receives rare pediatric disease designation for relutrigine in Dravet syndrome
- The FDA granted Rare Pediatric Disease Designation (RPDD) for relutrigine in Dravet syndrome.
- This is the third RPDD for relutrigine, adding to those for SCN2A and SCN8A DEEs.
- Praxis plans to initiate the EMERALD trial, inclusive of Dravet syndrome, in the first half of 2025.
- The designation could lead to a priority review voucher, expediting future drug application reviews.
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Vertex announces results from phase 2 study of suzetrigine for the treatment of painful lumbosacral radiculopathy
- Vertex's Phase 2 study of suzetrigine met its primary endpoint, showing a significant reduction in pain for patients with lumbosacral radiculopathy.
- The study involved 218 patients and demonstrated a mean change in pain intensity of -2.02 on the numeric pain rating scale.
- Suzetrigine was well tolerated, with a lower incidence of adverse events compared to the placebo group.
- Vertex plans to advance suzetrigine into pivotal development for lumbosacral radiculopathy, incorporating trial design innovations to manage placebo response.
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Genethon announces 2024 milestones in gene therapy
- Genethon has released positive safety and efficacy results from the Phase 1/2 study of GNT0004 for Duchenne muscular dystrophy.
- The independent data monitoring committee has approved the progression to Phase 3 trials, expected to start in Europe in 2025.
- A Phase 2 trial of imlifidase, in partnership with Hansa Biopharma, aims to address anti-AAV antibodies in Crigler-Najjar syndrome patients.
- Genethon has published research on AI-designed capsids for gene therapy vectors in Nature Communications.
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Phase 3 trial results of brexpiprazole and sertraline for PTSD published
- Otsuka and Lundbeck's Phase 3 trial results for brexpiprazole with sertraline in PTSD treatment published in JAMA Psychiatry.
- The trial showed significant improvement in PTSD symptoms with brexpiprazole and sertraline compared to sertraline and placebo.
- The trial was a randomized, double-blind, active-controlled study with 416 adult participants.
- Next steps include working with the FDA to bring this combination therapy to healthcare professionals.
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Bioxodes meets enrollment milestone in Phase 2a intracerebral hemorrhagic stroke trial
- Bioxodes has enrolled the first 16 of 32 patients in its Phase 2a trial for BIOX-101, targeting intracerebral hemorrhagic stroke.
- The trial is open-label, randomized, and controlled, with an interim analysis triggered by the treatment of the first cohort.
- BIOX-101, derived from tick saliva, aims to prevent secondary brain injuries post-stroke without increasing bleeding risk.
- Bioxodes has filed for orphan drug status in the U.S. and Europe, with potential regulatory responses expected in early 2025.
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