Elpida Therapeutics and CureCMT4J Announce FDA Clearance of IND Application for ELP-02, an AAV9 Gene Therapy for CMT4J
Los Angeles, CA and Ipswich, MA – Dec 19, 2024 – Elpida Therapeutics, a leader in gene therapy for ultra-rare diseases, and CureCMT4J, a nonprofit dedicated to advancing treatments for Charcot-Marie-Tooth disease type 4J (CMT4J), are excited to announce that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for ELP-02, an intrathecal AAV-9 gene therapy developed for the treatment of CMT4J, an ultra-rare progressive neurodegenerative disorder.
This milestone paves the way for the initiation of a first-in-human Phase 1/2 clinical trial, designed to evaluate the safety, tolerability, and preliminary efficacy of ELP-02 in individuals living with CMT4J. CureCMT4J spearheaded early efforts years ago, providing essential funding and leadership that laid the foundation for today’s progress. The program is now supported by a grant from the California Institute for Regenerative Medicine (CIRM) and is being advanced in collaboration with the Accelerating Medicines Partnership® (AMP®) Bespoke Gene Therapy Consortium (BGTC) led by the Foundation for the National Institutes of Health (FNIH).
"We are thrilled to advance ELP-02 into clinical development. This IND clearance marks a monumental step toward providing a transformative therapeutic option for individuals and families affected by CMT4J," said Terry Pirovolakis, Founder and CEO of Elpida Therapeutics. "It reflects our commitment to addressing the unmet needs of ultra-rare disease communities and the groundbreaking potential of our platform."
CMT4J is caused by mutations in the FIG4 gene, leading to the loss of myelin in peripheral nerves and axonal degeneration, resulting in muscle weakness and atrophy, sensory loss, and progressive nerve dysfunction. With no approved treatments available, the novel gene therapy approach delivers a functional copy of the FIG4 gene directly to affected cells, aiming to halt or potentially reverse disease progression.
The Phase 1/2 clinical trial will involve a multicenter, open-label design, focusing initially on safety in a small cohort of patients. Patient enrollment is expected to begin in 2025.
“Elpida’s IND clearance is a positive step forward in finding treatment solutions for CMT4J patients and their families,” said Rosa Canet-Avilés, PhD, Chief Science Officer (CSO) at CIRM. “CIRM remains committed to supporting therapies that target rare diseases and address the critical unmet needs of these patients.”
“The BGTC congratulates the Elpida team on their successful IND clearance for ELP-02, and look forward to further progress towards the clinical trial for CMT4J” said Dr. P.J. Brooks, BGTC co-chair. This program underscores Elpida Therapeutics’ mission to develop innovative treatments for ultra-rare diseases. The IND clearance reflects the culmination of years of work on behalf of CureCMT4J, as well as collaborative efforts with researchers, industry, and regulatory agencies, highlighting the importance of partnerships in advancing therapies that address critical unmet needs.
About Elpida Therapeutics
Elpida Therapeutics is dedicated to delivering life-saving therapies for ultra-rare diseases. Founded to tackle the unmet medical needs of children living with rare conditions, Elpida works to rapidly advance its therapeutic programs in partnership with leading researchers and institutions around the globe. [www.elpidatx.com]
About CureCMT4J
Founded by Jocelyn Duff, CureCMT4J is a nonprofit organization leading the charge to bring treatments to people with CMT4J and FIG4-related disorders. Through research funding, advocacy, and community support, CureCMT4J is dedicated to advancing treatments for CMT4J, offering hope to families impacted by this ultra-rare disease. [www.curecmt4j.org]
About the California Institute for Regenerative Medicine (CIRM)
The California Institute for Regenerative Medicine (CIRM) is a funding agency established by Californians to accelerate regenerative medicine research to deliver treatments for patients with unmet medical needs. Established in 2004 through the passage of Proposition 71, CIRM was initially funded with $3 billion from the state of California to support ongoing research, and in 2020, was funded again with another $5.5 billion through Proposition 14 to continue the Agency’s important work.
CIRM has provided billions in funding to support stem cell, genetic research, and development programs in its portfolio. Through the Agency’s research, infrastructure, and education programs, CIRM aims to transform the field of regenerative medicine, stimulate economic growth, and improve the lives of diverse communities throughout the state. For more information go to [cirm.ca.gov].
About the Foundation for the National Institutes of Health
The Foundation for the National Institutes of Health (FNIH) builds public-private partnerships that connect leading biomedical scientists at the National Institutes of Health (NIH) with their counterparts in life sciences companies, academia, patient organizations, foundations, and regulatory agencies (including the Food and Drug Administration and European Medicines Agency). Through team science, the FNIH solves complex health challenges and accelerates breakthroughs for patients, regardless of who they are or what health threats they face. The FNIH contributes to the development of new therapies, diagnostics, and potential cures; advances global health and equity in care; and celebrates and helps train the next generations of scientists. Established by Congress in 1990 to support the mission of the NIH, the FNIH is a not-for-profit 501(c)(3) charitable organization. For more information about the FNIH, please visit [fnih.org].