August 9, 2024 - 🧬 [nGram] Today’s Neurology Scoop: Parkinson's Trial, Orphan Drug Designation, AI-Generated Candidate


  1. PhotoPharmics enrolls first 100 participants in groundbreaking Parkinson's disease trial
    • PhotoPharmics has enrolled the first 100 subjects in its Light for PD clinical trial, aiming for a total of 300 participants.
    • The trial is a randomized, double-blind, placebo-controlled study evaluating the Celeste light therapy device for Parkinson's disease.
    • Participants use the Celeste device for one hour daily, with primary endpoints including improvements in quality of life, motor function, sleep quality, mood, and cognition.
    • The trial's at-home, telemedicine-based format enhances accessibility, allowing broader participation, including individuals in remote areas.
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  2. Actio Biosciences receives orphan drug and rare pediatric disease designations for ABS-0871 for the treatment of Charcot-Marie-Tooth disease 2C
    • Actio Biosciences' ABS-0871, a TRPV4 inhibitor, has been granted orphan drug and rare pediatric disease designations by the FDA.
    • These designations are for the treatment of TRPV4+ Charcot-Marie-Tooth disease subtype 2C (CMT2C), a rare and debilitating disease.
    • Orphan drug designation provides benefits like tax credits, FDA fee waivers, and seven years of market exclusivity upon approval.
    • Rare pediatric disease designation awards a priority review voucher, which can be redeemed, transferred, or sold.
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  3. Recursion provides business updates and reports second quarter 2024 financial results
    • Recursion entered into a definitive agreement to combine with Exscientia, enhancing its clinical pipeline and precision chemistry capabilities.
    • The combined company expects approximately 10 clinical trial readouts over the next 18 months.
    • The first neuroscience phenomap under Recursion’s collaboration with Roche and Genentech has been optioned, triggering a $30 million payment.
    • Recursion reported a net loss of $97.5 million for Q2 2024, with cash and cash equivalents totaling $474.3 million as of June 30, 2024.
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  4. Biohaven reports second quarter 2024 financial results and recent business developments
    • Cash, cash equivalents, marketable securities, and restricted cash totaled approximately $440 million as of June 30, 2024.
    • BHV-1300, a lead investigational degrader, showed dose-dependent and rapid IgG reductions in an ongoing Phase 1 trial with no serious adverse events reported.
    • Biohaven's beta-1 adrenergic receptor (β1AR) autoantibody targeting MoDE, BHV-1600, granted INTERACT meeting with FDA in 2H 2024.
    • Advancing multiple programs including 5 Phase 2/3 trials with BHV-7000 in epilepsy and mood disorders, and Phase 2 study in acute migraine with BHV-2100 in 2H 2024.
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  5. DeepCure announces selection of first AI-generated candidate DC-9476 for autoimmune diseases
    • DeepCure has selected its first development candidate, DC-9476, a third-generation BRD inhibitor targeting the BD2 domain of Brd4.
    • DC-9476 is expected to enter clinical trials in 2025 and has shown promising efficacy in preclinical models of rheumatoid arthritis and Still’s disease.
    • The candidate was discovered using DeepCure’s proprietary AI platform, which integrates advanced ML and physics-based tools.
    • DC-9476 has an excellent safety profile and did not cause thrombocytopenia, unlike earlier generations of non-selective inhibitors.
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  6. ANEW Medical pursues neurological approach to treating weight loss and eating disorders
    • ANEW Medical has entered a worldwide licensing agreement with Teleost Biopharmaceuticals and the University of Arizona.
    • The company plans to develop therapeutics using proprietary peptides that bind and activate melanocortin receptors in the brain.
    • These peptides can be administered orally, potentially making them more affordable than current treatments like semaglutide.
    • The targeted eating disorders include obesity, cachexia, and other related conditions.
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