August 12, 2024 - 🧬 [nGram] Today’s Neurology Scoop: NurExone's ExoPTEN, n-Lorem in Nature Medicine, Neurotech Patent

1. NurExone's ExoPTEN loaded with siRNA demonstrates ability to target injury and inflammation sites

   • NurExone Biologic Inc. announced new data for its ExoPTEN nanodrug, marking a significant step towards commercial-grade manufacturing.
   • The study assessed ExoPTEN loaded with siRNA produced by a new GMP-compliant CRO partner, focusing on its ability to target inflammation and injury sites.
   • ExoPTEN was administered to rats after spinal cord compression injury, showing exceptional homing capacity to the injured spinal cord.
   • The results validate the quality of the siRNA produced by the GMP partner and the use of NurExone’s exosomes as a drug delivery system.
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2. Positive clinical observations from n-Lorem treated, KIF1A nano-rare patient published in Nature Medicine

   • n-Lorem published a case study in Nature Medicine detailing the treatment of a nano-rare patient with a personalized ASO medicine.
   • The patient, Susannah, showed significant improvements in seizure frequency, gait, speech, and overall quality of life.
   • Before treatment, Susannah experienced up to 290 seizures a day, which reduced to less than 30 a week post-treatment.
   • The patient has been on ASO treatment for 20 months with a favorable safety and tolerability profile.
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3. Neurotech innovation achieves patent approval and featured cover story in prominent industry journal

   • Longeviti Neuro Solutions received a patent for its ClearFit® prosthetic translucent cranial implant.
   • The implant integrates brain-computer interface (BCI), brain mapping, and neurosonography.
   • Science Translational Medicine featured the implant in a cover story, highlighting its research and applications.
   • The implant allows for real-time, non-invasive brain monitoring and improved surgical precision.
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4. Lykos Therapeutics receives complete response letter for midomafetamine capsules for PTSD

   • The FDA issued a complete response letter (CRL) for Lykos Therapeutics' new drug application (NDA) for midomafetamine capsules for PTSD.
   • The FDA requested an additional Phase 3 trial to further study the safety and efficacy of midomafetamine.
   • Lykos plans to request a meeting with the FDA to discuss the decision and recommendations for resubmission.
   • The company remains committed to developing midomafetamine-assisted therapy and will explore all regulatory pathways to bring the treatment to market.
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5. MAPS receives FDA complete response letter for MDMA-assisted therapy for PTSD

   • The FDA has issued a complete response letter delaying the approval of MDMA-assisted therapy for PTSD.
   • MAPS has been advocating for MDMA-assisted therapy for over 38 years and remains committed to its development.
   • The organization focuses on advancing research, changing drug policy, and shaping culture through education and events.
   • MAPS continues to support psychedelic and marijuana research, particularly in areas heavily impacted by trauma.
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6. Neurogene reports second quarter 2024 financial results and highlights recent updates

   • NGN-401 gene therapy for Rett syndrome received RMAT designation and was selected for the FDA's START Pilot Program.
   • First patient dosed in high-dose Cohort 2 in May; early favorable safety profile reported.
   • Interim efficacy data from low-dose cohort expected in Q4 2024; additional data from both cohorts expected in 2025.
   • Completed enrollment in NGN-101 gene therapy trial for CLN5 Batten Disease; interim data and regulatory update expected in Q1 2025.
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