September 6, 2024 - 🧬 [nGram] Today’s Neurology Scoop: Abdera's FDA Orphan Drug, Ocuphire's Phase 3 Trial, LUCA's Groundbreaking Research


  1. Abdera Therapeutics receives FDA orphan drug designation for ABD-147
    • ABD-147 is a precision radiopharmaceutical biologic therapy targeting DLL3 on neuroendocrine tumors.
    • The FDA's Orphan Drug Designation provides incentives like tax credits, user fee exemptions, and potential seven-year market exclusivity.
    • Abdera plans to initiate a Phase 1 clinical trial in 2024 for patients with SCLC or LCNEC who previously received platinum-based therapy.
    • ABD-147 also received Fast Track designation for treating extensive stage small cell lung cancer (ES-SCLC).
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  2. Ocuphire Pharma initiates VEGA-3 phase 3 trial evaluating phentolamine ophthalmic solution 0.75% for presbyopia
    • Ocuphire Pharma has started the VEGA-3 Phase 3 trial for Phentolamine Ophthalmic Solution 0.75% to treat presbyopia.
    • The trial is a randomized, double-masked, placebo-controlled study involving 545 participants.
    • Participants will receive one drop of the solution or placebo each evening, with the primary endpoint being a 15-letter improvement in near visual acuity.
    • Top-line data from the VEGA-3 trial is expected in the first half of 2025.
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  3. Cyclo Therapeutics presents encouraging preliminary safety data from ongoing pivotal phase 3 study and substudy for the treatment of Niemann-Pick disease type C1
    • Cyclo Therapeutics presented positive preliminary data from its ongoing pivotal Phase 3 study (TransportNPC™) and substudy evaluating Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 (NPC1).
    • The TransportNPC™ study, the most comprehensive controlled pivotal study for NPC1, completed enrollment in May 2024 with 104 patients.
    • The substudy, focusing on newborns to 3-year-olds, completed enrollment with 10 patients and aims to evaluate the safety and efficacy of early intervention.
    • Topline data from the 48-week interim analysis is anticipated in H1 2025, with potential marketing applications to follow if data meets statistical significance.
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  4. LUCA Science publishes groundbreaking research in Nature Metabolism: mitochondria transfer-based therapies reduce the morbidity and mortality of Leigh syndrome
    • LUCA Science Inc., in collaboration with Osaka University and Washington University School of Medicine, published a study in Nature Metabolism.
    • The study demonstrates that mitochondria transfer-based therapies can alleviate symptoms and reduce morbidity and mortality in a Leigh Syndrome (LS) mouse model.
    • Key findings include significant improvements in LS-affected mice through wild-type bone marrow transplantation and administration of isolated mitochondria.
    • LUCA Science is advancing towards GMP development activities to bring Mitochondria oRganelle Complex Q (MRC-Q) to clinical development.
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  5. Medincell’s partner Teva provides new update on pivotal clinical phase 3 of investigational olanzapine long-acting injectable (LAI)
    • Teva Pharmaceuticals announced that approximately 99% of the targeted injections for submission have been performed.
    • Full submission safety results for Olanzapine LAI are expected to be available in H2 2024.
    • Olanzapine LAI is a once-monthly subcutaneous injection with a potentially favorable safety profile.
    • Medincell may receive up to $117 million in development and commercial milestones, plus royalties on net sales.
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  6. Evotec and X-Chem collaborate to accelerate early-stage drug discovery
    • Evotec SE and X-Chem announced a collaboration to accelerate early-stage drug discovery.
    • X-Chem’s DNA-encoded library (DEL) technology will be integrated with Evotec’s screening platforms.
    • The collaboration includes access to X-Chem’s DELflex and HITMiner solutions.
    • The partnership aims to streamline hit finding and deliver optimal drug intervention starting points.
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  7. NImmune Biopharma announces R&D collaboration with BioTherapeutics to advance inflammation and immunology precision medicines
    • NImmune Biopharma partners with BioTherapeutics to enhance precision immunology capabilities.
    • Collaboration includes access to BioTherapeutics' preclinical services, regulatory capabilities, and proprietary animal models.
    • NImmune aims to accelerate the development of immunoregulatory therapeutics for inflammatory and autoimmune diseases.
    • The partnership complements NImmune's existing R&D collaboration with NIMML Institute.
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  8. Vor Bio announces new clinical data validating approach of using shielded transplants to deliver targeted therapies
    • New clinical data from Phase 1/2 VBP101 study of relapsed/refractory AML patients receiving trem-cel followed by Mylotarg.
    • Data showed reliable engraftment, shielding from Mylotarg on-target toxicity, broadened therapeutic window, and early evidence of patient benefit.
    • 18 patients treated with trem-cel; 10 received Mylotarg as of July 19, 2024.
    • Vor Bio plans to discuss a pivotal trial design for trem-cel + Mylotarg with the FDA by year-end.
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  9. Aurinia announces first participant dosed in AUR200 single ascending dose trial
    • Aurinia Pharmaceuticals has dosed the first participant in a Phase 1a single ascending dose (SAD) study of AUR200.
    • The study will assess safety, tolerability, pharmacokinetics, and biomarker changes in healthy volunteers.
    • Data from the study is expected in the first half of 2025.
    • AUR200 targets both BAFF and APRIL, showing potential as a best-in-class therapy for autoimmune diseases.
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  10. Crestone announces positive data from phase 2 clinical trial of CRS3123 for C. difficile infections
    • Crestone reported positive topline results from the Phase 2 trial of CRS3123 in patients with Clostridioides difficile infection (CDI).
    • Clinical cure rates at day 12 were 97% for CRS3123 and 93% for vancomycin, with no clinical failures.
    • CDI recurrence rates at day 40 were significantly lower for CRS3123 (4%) compared to vancomycin (23%).
    • The National Institute of Allergy and Infectious Diseases (NIAID) has provided $4.5 million in new funding for further studies.
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