Branaplam
- Generic Name
- Branaplam
- Brand Names
- -
- Drug Type
- Small Molecule
- Chemical Formula
- C22H27N5O2
- CAS Number
- 1562338-42-4
- Unique Ingredient Identifier
- P12R69543A
- Background
Branaplam is under investigation in clinical trial NCT02268552 (An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)).
- Indication
用于治疗亨廷顿氏病（HD，是一种罕见的遗传性神经退行性疾病）。
- Associated Conditions
- -
- Associated Therapies
- -
Novartis Tries Again in Huntington's, Putting Up $1B to Partner on a PTC Therapeutics Drug
Novartis pays $1 billion upfront to license PTC518, a Huntington's disease drug, with potential for $1.9 billion in milestone payments and royalties. PTC retains 40% of U.S. profits and leads Phase 2 completion and FDA discussions. The deal is expected to close in Q1 2025.
Novartis, PTC Ink Up-to-$2.9B Huntington's Disease Collaboration
Novartis partners with PTC Therapeutics for up-to-$2.9 billion to develop PTC518 for Huntington's disease, with $1 billion upfront and potential $1.9 billion in milestones and royalties. PTC518 showed dose-dependent reductions in mutant Huntingtin protein levels and potential for accelerated FDA approval.
Huntington's disease: a therapeutic field on a bumpy ride
Huntington's disease treatments face challenges due to rarity and trial failures, but recent milestones like Wave's WVE-003, Roche-Ionis' tominersen, uniQure's AMT-130, and PTC Therapeutics' PTC518 offer hope. Prilenia's pridopidine is under EMA review, while Annexon's ANX005 and Sage's dalzanemdor faced setbacks. Despite these, cautious optimism remains for effective treatments.
Huntington Study Group (HSG) Conference 2024 – Day 1
HSG 2024 in Cincinnati features updates on HD research, including MyHDStory, KINECT-HD, huntingtin-lowering debates, drug delivery, CRISPR gene editing, and innovative HD therapeutics from various companies.
Drug Development Updates
Stay informed with timely notifications on clinical trials, regulatory changes, and research advancements related to this medication.
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