Elixirgen's EXG-34217 Shows Promising Telomere Elongation Results In Phase 1/2 Clinical Trial

Elixirgen Therapeutics, Inc., a clinical-stage biotechnology company focused on rare disease treatment, today announced the publication of very encouraging early clinical data from an active Phase 1/2 trial (NCT04211714), data that indicates successful and sustained telomere elongation for the first time in multiple EXG-34217-treated patients with a telomere biology disorder (TBD). Absolutely no treatment-related safety concerns were observed during the 24-month period. Furthermore, no treatment-related safety concerns were identified during the additional 5-month period after the EXG-34217 infusion. 

Kasiani C. Myers, MD, Professor, Department of Pediatrics, Cincinnati Children’s Hospital Medical Center, and principal investigator in the study, said in a statement, “The data published today in NEJM Evidence demonstrates sustained telomere elongation in blood cells in patients with TBDs without toxicity, an outcome that has not been achieved by other treatments for those with this disorder. In addition, EXG-34217 administration did not require a preconditioning regimen or immunosuppression typically required for other treatment regimens, which is critical in this radiation- and chemotherapy-sensitive population.

She further mentioned, “ There is a significant unmet need in this space since the only option for patients with TBDs who develop bone marrow failure is hematopoietic stem cell transplantation (HSCT), which can result in life-threatening complications. We look forward to enrolling additional patients, now including pediatric patients over 12 years of age, in the trial.”

Aki Ko, chief executive officer of Elixirgen Therapeutics, said, “ZSCAN4 was originally identified and characterized by our chief scientific officer, Minoru Ko, MD, PhD, at National Institute on Aging (NIA), one of the Institutes of the National Institutes of Health (NIH), and we are excited to share initial results of the first clinical trial providing ZSCAN4 to patients. This unique gene plays a critical role in several key genomic functions, including telomere extension, to restore the integrity and potency of stem cells. EXG-34217 harnesses the power of this protein and allows us to provide a potential new treatment option for TBDs. This therapy is genotype and mutation-independent since the elongation of telomeres by ZSCAN4 is telomerase-independent. We’re pleased with this new data and look forward to continuing to work with Dr. Myers and the team at Cincinnati Children’s to advance the trial. We are deeply appreciative of the patients who have participated.”