August 27, 2024 - 🧬 [nGram] Today’s Neurology Scoop: Ocugen's Phase 3 Trial, Invivyd's COVID-19 Breakthrough, RedHill's Orphan Drug

1. Ocugen receives Health Canada approval for phase 3 trial of OCU400 gene therapy

   • Health Canada has approved Ocugen to initiate the Phase 3 liMeliGhT clinical trial for OCU400 in Canada.
   • The trial will enroll up to 50 subjects across 5 sites, running parallel to the U.S. FDA trial.
   • OCU400 targets retinitis pigmentosa (RP) and aims to provide a gene-agnostic treatment option.
   • The primary endpoint is the change in functional vision, measured by the Luminance Dependent Navigation Assessment (LDNA).
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2. Clearside Biomedical completes final participant visit in ODYSSEY Phase 2b trial of CLS-AX in wet AMD

   • CLS-AX is a highly potent tyrosine kinase inhibitor delivered suprachoroidally using Clearside’s proprietary SCS Microinjector.
   • Topline data expected to be reported during the week of October 7, 2024.
   • ODYSSEY is a 36-week, randomized, double-masked, parallel-group, active-controlled, multi-center Phase 2b clinical trial in participants with wet AMD.
   • The trial involves 60 participants randomized to either CLS-AX or aflibercept with a 2:1 ratio.
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3. Invivyd announces pemgarda demonstrated 84% relative risk reduction in symptomatic COVID-19

   • PEMGARDA (pemivibart) showed an 84% relative risk reduction in symptomatic COVID-19 compared to placebo in the CANOPY Phase 3 trial.
   • In immunocompetent participants, the rate of symptomatic COVID-19 was 1.9% with pemivibart versus 11.9% with placebo.
   • In immunocompromised participants, pemivibart demonstrated a 3% rate of confirmed symptomatic COVID-19.
   • The safety profile of pemivibart was consistent with previous CANOPY trial data, with common adverse events including viral infections and infusion-related reactions.
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4. RedHill's opaganib granted orphan drug designation by the FDA for childhood cancer, neuroblastoma

   • The FDA has granted orphan drug designation to RedHill Biopharma's opaganib for the treatment of neuroblastoma, a rare childhood cancer.
   • Orphan drug designation provides seven years of marketing exclusivity upon approval, along with potential benefits like accelerated development, grant funding, and tax credits.
   • Neuroblastoma is the most common infancy malignancy, accounting for 10% of all childhood cancer cases in the U.S. and 15% of pediatric cancer-related deaths.
   • This is the second orphan drug designation for opaganib in oncology, following its previous designation for cholangiocarcinoma (bile duct cancer).
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5. First patient dosed with LIXTE’s LB-100 in new clinical trial to treat colorectal cancer

   • LIXTE Biotechnology Holdings has dosed the first patient in a new clinical trial for metastatic colorectal cancer.
   • The trial is in collaboration with the Netherlands Cancer Institute (NKI) and supported by Roche.
   • LIXTE is providing its lead compound, LB-100, while Roche is providing atezolizumab (Tecentriq).
   • The trial aims to bring immunotherapy to approximately 85% of colorectal cancer patients who have not responded to traditional treatments.
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6. Find Therapeutics announces FDA clearance of IND application for a Phase 1 study of FTX-101 for the treatment of chronic optic neuropathy

   • FDA has cleared Find Therapeutics' IND application for FTX-101.
   • The Phase 1 study will assess the safety, tolerability, and pharmacokinetics of FTX-101.
   • The study will be a single ascending dose (SAD) and multiple ascending dose (MAD) trial.
   • Enrollment is expected to include up to 80 participants, commencing in Q4 2024.
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7. Addex and Indivior select clinical candidates from GABAB positive allosteric modulator research collaboration

   • Addex Therapeutics and Indivior have selected clinical candidates from their GABAB PAM research collaboration.
   • Indivior will develop a compound for substance use disorder, with potential payments to Addex up to USD 330 million.
   • Addex will advance its own GABAB PAM program for chronic cough into IND enabling studies.
   • GABAB PAMs are expected to deliver efficacy with fewer adverse effects compared to traditional agonists.
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8. Study results from phase 1 studies with exidavnemab published in The Journal of Clinical Pharmacology

   • BioArctic AB announced phase 1 study results for exidavnemab, published in The Journal of Clinical Pharmacology.
   • Exidavnemab is a monoclonal antibody targeting aggregated forms of α-synuclein, aimed at treating Parkinson's disease.
   • Two phase 1 studies assessed safety, tolerability, and pharmacokinetics in 98 healthy volunteers.
   • Results showed exidavnemab was well-tolerated with a half-life of approximately 30 days, supporting further clinical development.
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9. Amarna Therapeutics receives constructive feedback from FDA INTERACT meeting for AM510 gene therapy development targeting type 1 diabetes

   • Amarna Therapeutics completed an FDA INTERACT meeting on July 31, 2024, for their AM510 gene therapy targeting Type 1 Diabetes.
   • The meeting provided extensive feedback on the manufacturing and controls (CMC) program, nonclinical program, and safety aspects of the clinical trial design.
   • The FDA's insights will help refine the approach to ensure the safety and efficacy of AM510 for T1D patients.
   • Amarna plans to engage with the FDA in a pre-IND meeting within the next 2 years.
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