Oct 28, 2024

Posted on DelveInsight’s, “Spinocerebellar Ataxias Pipeline Insight 2024” report provides comprehensive insights about 10+ companies and 12+ pipeline drugs in Spinocerebellar Ataxias pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space. Explore our latest breakthroughs in Spinocerebellar Ataxias research. Learn more about our innovative pipeline today! @ Spinocerebellar Ataxias Pipeline Outlook Key Takeaways from the Spinocerebellar Ataxias Pipeline Report In October 2024:- Quince Therapeutics S.p.A.- This is an international, multi-center, randomized, prospective, double-blind, placebo-controlled, Phase 3 study, designed to assess the effect of EryDex (dexamethasone sodium phosphate [DSP] in autologous erythrocytes), administered by intravenous (IV) infusion once every 28 days, on neurological symptoms of patients with Ataxia Telangectasia (A-T). DelveInsight’s Spinocerebellar Ataxias pipeline report depicts a robust space with 10+ active players working to develop 12+ pipeline therapies for Spinocerebellar Ataxias treatment. The leading Spinocerebellar Ataxias Companies such as Lexeo Therapeutics, Vico Therapeutics, PTC Therapeutics, Minoryx Therapeutics, IntraBio, Acasti Pharma, Design Therapeutics, and others. Promising Spinocerebellar Ataxias Therapies such as IB1001, Dexamethasone sodium phosphate, VO659, troriluzole, and others Stay informed about the cutting-edge advancements in Spinocerebellar Ataxias treatments. Download for updates and be a part of the revolution in cancer care @ Spinocerebellar Ataxias Clinical Trials Assessment Vatiquinone: PTC Therapeutics PTC is developing vatiquinone, a potential treatment for Friedreich ataxia based on our Bio-e platform. Vatiquinone is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme that is a key regulator of the energetic and oxidative stress pathways that are disrupted in Friedreich ataxia. Inhibition of 15-LO helps to alleviate the consequences of mitochondrial dysfunction and oxidative stress, ultimately preventing ferroptosis and aiding neuronal survival. Vatiquinone has been evaluated in a number of clinical studies and has demonstrated an impact on mortality risk and a number of neurological and neuromuscular disease symptoms. Currently the drug is in Phase III stage of its clinical trial for the treatment of spinocerebellar ataxias. Leriglitazone: Minoryx Therapeutics Leriglitazone (MIN-102) is Minoryx’s novel orally bioavailable and selective PPAR gamma agonist with a potential first-in-class and best-in-class profile for CNS diseases. It has demonstrated brain penetration and a favorable safety profile. It showed robust preclinical proof-of-concept in animal models of multiple diseases by modulating pathways leading to mitochondrial dysfunction, oxidative stress, neuroinflammation, demyelination and axonal degeneration. In clinical trials, leriglitazone showed clinical benefit both for X-ALD and Friedreich’s Ataxia patients. Leriglitazone has been granted orphan drug status for X-ALD from the FDA and the EMA and fast track and rare pediatric disease designation from the FDA for the treatment of X-ALD. Currently, the drug is in Phase II stage of its clinical trial for the treatment of spinocerebellar ataxias. DT-216: Design Therapeutics DT-216 by Design Therapeutics is a therapeutic candidate under clinical development for the treatment of Friedreich Ataxia. It comprises synthetic transcription elongation factor 1 (Syn-TEF1) and targets frataxin. The drug is administered intravenously and is part of Design Therapeutics’ efforts to develop small-molecule genomic drugs for degenerative genetic disorders. DT-216 has shown promising results in increasing frataxin mRNA levels, with the potential to restore frataxin protein expression, which is crucial in addressing the clinical manifestations of Friedreich Ataxia. Currently the drug is in Phase I stage of its clinical trial. Learn more about Spinocerebellar Ataxias Drugs opportunities in our groundbreaking Spinocerebellar Ataxias Research and development projects @ Spinocerebellar Ataxias Unmet Needs Discover the latest advancements in Spinocerebellar Ataxias treatment by visiting our website. Stay informed about how we’re transforming the future of oncology @ Spinocerebellar Ataxias Market Drivers and Barriers, and Future Perspectives Coverage- Global Spinocerebellar Ataxias Companies- Lexeo Therapeutics, Vico Therapeutics, PTC Therapeutics, Minoryx Therapeutics, IntraBio, Acasti Pharma, Design Therapeutics, and others. Spinocerebellar Ataxias Therapies- IB1001, Dexamethasone sodium phosphate, VO659, troriluzole, and others Spinocerebellar Ataxias Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination Spinocerebellar Ataxias Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III For a detailed overview of our latest research findings and future plans, read the full details of Spinocerebellar Ataxias Pipeline on our website @ Spinocerebellar Ataxias Emerging Drugs and Companies