August 28, 2024 - 🧬 [nGram] Today’s Neurology Scoop: FDA Priority for SpringWorks, Ocugen Dosing, Neurocrine Positive Data

1. FDA grants priority review to SpringWorks Therapeutics’ new drug application for mirdametinib for the treatment of adults and children with NF1-PN

   • The FDA has accepted SpringWorks Therapeutics' NDA for mirdametinib, granting it Priority Review with a PDUFA action date of February 28, 2025.
   • The European Medicines Agency has also validated the Marketing Authorization Application for mirdametinib.
   • Mirdametinib could become the first approved therapy for adults and a best-in-class therapy for children with NF1-PN.
   • The submissions include data from the Phase 2b ReNeu trial, which showed robust objective response rates and a manageable safety profile.
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2. Ocugen completes dosing in high dose cohort of phase 1/2 GARDian clinical trial for OCU410ST

   • Ocugen has completed dosing in the third cohort of its Phase 1/2 GARDian clinical trial for OCU410ST, targeting Stargardt disease.
   • Three subjects received a single subretinal injection of the highest dose (2.25×10^11 vg/mL).
   • The trial is being conducted at six leading retinal surgery centers across the U.S.
   • The GARDian trial will assess the safety and efficacy of OCU410ST in two phases, with Phase 1 being a multicenter, open-label, dose-ranging study.
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3. Neurocrine Biosciences reports positive phase 2 data for NBI-1117568 in adults with schizophrenia

   • The once-daily 20 mg dose met the primary endpoint, showing a 7.5-point improvement in PANSS total score compared to placebo at Week 6.
   • Statistically significant improvements were also observed in the Clinical Global Impression of Severity scale and Marder Factor Score for both positive and negative symptoms.
   • NBI-1117568 was generally safe and well tolerated, with adverse events similar to placebo, including somnolence, dizziness, and headache.
   • Phase 2 results support advancement to Phase 3 in early 2025.
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4. Yunovia announces IND approval by the MFDS to initiate phase 1 MAD study for the small molecule GLP-1 agonist

   • Yunovia received IND approval from the MFDS of Korea for a Phase 1 Multiple Ascending Dose (MAD) study of ID110521156.
   • The study will evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamic (PD) characteristics of the drug.
   • ID110521156 is an orally available small molecule GLP-1 agonist aimed at treating obesity and diabetes.
   • The company plans to collect PD data, including continuous glucose monitoring and body weight changes, as exploratory endpoints from a 4-week MAD study.
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5. Hyundai Bioscience to conduct phase 3 clinical trial for high-risk group of COVID-19 patients

   • Hyundai Bioscience has submitted an IND application for a Phase 3 clinical trial to the MFDS of South Korea.
   • The trial will target high-risk COVID-19 patients, including those aged 60+, with underlying conditions.
   • Xafty® has shown efficacy and safety in previous trials, particularly for high-risk patients on other medications.
   • The new trial will involve 290 participants and will be conducted as outpatient visits to expedite recruitment and reduce costs.
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6. Telix submits NDA for TLX101-CDx brain cancer imaging agent

   • Telix Pharmaceuticals has submitted a New Drug Application (NDA) to the FDA for TLX101-CDx (Pixclara), a PET agent for imaging glioma.
   • Pixclara has been granted Orphan Drug and Fast Track designations by the FDA, facilitating expedited review.
   • Currently, there is no FDA-approved targeted amino acid PET agent for brain cancer imaging in the U.S.
   • Pixclara aims to improve diagnosis and management of glioma, particularly in post-treatment settings.
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