PTC Therapeutics

PTC Therapeutics submitted vatiquinone NDA to FDA for Friedreich ataxia treatment, potentially the first therapy for children with FA. Data from MOVE-FA and long-term studies show efficacy and safety in pediatric and adult patients.

Neurocrine Biosciences' new FDA-approved non-steroidal drug, Crenessity, treats classic congenital adrenal hyperplasia (CAH), offering an alternative to steroid treatments with fewer side effects. Approved for both adults and children aged 4+, it reduces ACTH and adrenal androgens, with potential for significant revenue. Neurocrine plans to disclose pricing upon launch, with projections of $150 million in 2025 and peak sales of $1.47 billion.

NCATS’ TRND program aided in developing FDA-approved gene therapy Kebilidi for AADC deficiency, a fatal genetic brain disorder. Kebilidi, delivered directly to the brain, uses an adeno-associated virus to restore the missing AADC enzyme. TRND collaborated with Agilis Biotherapeutics Inc. to conduct preclinical studies and develop production methods, paving the way for PTC Therapeutics to complete necessary studies for global patient access.

Novartis partners with PTC Therapeutics to develop PTC518, a small molecule therapy targeting the root cause of Huntington’s disease, offering potential disease-modifying treatment. The therapy is in Phase II trials, with Novartis assuming global commercialisation responsibilities upon trial completion.

NICE recommends Santhera's Agamree for DMD, available to 1,700 NHS patients aged 4+ after a price discount. Agamree, an alternative to corticosteroids, reduces side effects and maintains efficacy, offering benefits over current treatments.

Novartis entered a global license and collaboration agreement with PTC Therapeutics for PTC518, a potential first oral disease-modifying therapy for Huntington's disease. Novartis will pay $1 billion upfront and up to $1.9 billion in milestones, sharing US profits and losses 60/40 with PTC. The deal bolsters Novartis' neuroscience pipeline, focusing on neurodegenerative diseases with high unmet needs.

Alnylam Pharmaceuticals launched a Phase 1 trial for ALN-HTT02, a huntingtin-lowering drug for Huntington’s disease, using RNA interference. The trial aims to assess safety and efficacy, with recruitment currently open in the UK and Canada. Participants, aged 25-70, will receive spinal injections of ALN-HTT02 or a placebo, with the option to switch to the drug after 12 months.

Clinical trials of ALN-HTT02, an siRNA-based drug targeting Huntington's disease, have begun at UCLH, offering hope for disease modification. The trial involves 54 participants and aims to analyze the drug's safety and efficacy, with potential to transform current treatment paradigms. Collaboration between pharmaceutical companies and research institutions is crucial for advancing such therapies.

FDA placed a clinical hold on Rapport Therapeutics' RAP-219 for diabetic peripheral neuropathic pain, granted accelerated approval to PTC Therapeutics' gene therapy for AADC deficiency, cleared icobrain aria AI tool for ARIA detection in Alzheimer treatment, expanded age indication for Theranica's Nerivio Migraine device in pediatric patients, and issued a CRL for Applied Therapeutics' govorestat for classic galactosemia.

Takeda, Novartis, and Gilead announced separate licensing agreements for medications targeting hematologic cancers, tumors, and Huntington disease. Takeda will develop elritercept for anemia in MDS and myelofibrosis, Novartis secured rights to PTC518 for Huntington disease, and Gilead partnered with Tubulis to develop an ADC for solid tumors.