Melpida / CureSPG50, Elpida Therap - LARVOL DELTA

A Unique Model for Accelerating Drug Development and Clinical Trials towards Drug Approvals in Rare Pediatric CNS disorders (ASGCT 2025) - "Our lead clinical-stage program for SPG50 disease has progressed through clinical phases in half the time of traditional pipelines, and our lead product MELPIDA currently at Phase 3 trials. Our strategy at Elpida Therapeutics exemplifies a novel and highly effective approach to drug development in rare CNS disorders where there is a limited patient population and a significant unmet medical need. This model not only promises to shorten timelines but also aims to make the drug development process more patient-centric, cost-effective, and scalable for the future of precision medicine in rare diseases."

Clinical • Late-breaking abstract • CNS Disorders • Pediatrics • Rare Diseases

Phase 3, Open-label Study to Assess the Efficacy and Safety of a Single Lumbar Intrathecal Administration of MELPIDA in Individuals with Hereditary Spastic Paraplegia Type 50 (SPG50) Versus Matched Prospective Concurrent Controls. (clinicaltrials.gov) - P3 | N=24 | Not yet recruiting | Sponsor: Elpida Therapeutics SPC

New P3 trial • Genetic Disorders

Administration of MELPIDA to Determine the Safety and Efficacy for Patients With Spastic Paraplegia Type 50 (clinicaltrials.gov) - P1 | N=1 | Active, not recruiting | Sponsor: The Hospital for Sick Children

New P1 trial • Genetic Disorders

Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt) (clinicaltrials.gov) - P1/2 | N=2 | Recruiting | Sponsor: University of Texas Southwestern Medical Center | Not yet recruiting ➔ Recruiting

Enrollment open • Genetic Disorders • Movement Disorders • Muscle Spasticity

Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt) (clinicaltrials.gov) - P1/2 | N=2 | Not yet recruiting | Sponsor: University of Texas Southwestern Medical Center | Initiation date: Oct 2022 ➔ Jan 2023

Trial initiation date • Genetic Disorders • Movement Disorders • Muscle Spasticity

Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt) (clinicaltrials.gov) - P1/2 | N=2 | Not yet recruiting | Sponsor: University of Texas Southwestern Medical Center

New P1/2 trial • Genetic Disorders • Movement Disorders • Muscle Spasticity