Elixirgen Therapeutics announced positive Phase 1/2 clinical trial data for EXG-34217, a novel therapy for Telomere Biology Disorders (TBDs). The treatment, involving ZSCAN4-modified autologous CD34+ hematopoietic stem cells, achieved sustained telomere elongation and improved neutrophil counts in two patients without adverse effects. The U.S. FDA has granted EXG-34217 Rare Pediatric Disease, Regenerative Medicine Advanced Therapy (RMAT), and Orphan Drug Designations.
This development is a major step forward for TBDs, a group of rare genetic disorders characterized by critically short telomeres, leading to conditions like bone marrow failure. Current treatment options are limited, with hematopoietic stem cell transplantation (HSCT) being the primary intervention, carrying significant risks. EXG-34217’s success in elongating telomeres and improving blood cell counts without the need for preconditioning regimens or immunosuppression offers a potentially safer and more effective alternative to HSCT, addressing a critical unmet medical need for this patient population.
The published data highlights the treatment’s effectiveness in two patients, one observed for 24 months and the other for five months. Both patients experienced telomere elongation in CD34+ cells. One patient saw a significant increase in absolute neutrophil count (ANC), and their lymphocyte telomere length reached the normal range for their age. The second patient, previously reliant on G-CSF injections, no longer required them after EXG-34217 treatment and experienced an improved ANC along with resolution of prior symptoms. Importantly, no adverse events related to the treatment were reported.
These initial clinical results are promising and suggest EXG-34217 could become a groundbreaking therapy for TBDs. The ongoing clinical trial, now expanded to include pediatric patients over 12, will be crucial in further validating these findings and exploring the therapy’s long-term efficacy and safety profile. This could pave the way for a much-needed new treatment paradigm for TBDs, potentially transforming the lives of patients facing bone marrow failure and other related complications.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
