CMT-SORD Clinical Trial Update Promising Results from the INSPIRE Study on Govorestat!
We’re excited to share new results from HNF TRIAD Partner, Applied Therapeutics for the INSPIRE Phase 2/3 clinical trial of Govorestat (AT-007) for CMT-SORD presented at the Peripheral Nerve Society Annual Meeting on May 18, 2025.
“This is a pivotal moment for the CMT-SORD community. For the first time, we’re seeing a treatment that not only slows progression but truly improves patients’ lives. We are incredibly proud to support this groundbreaking work through HNF’s TRIAD program and remain committed to accelerating access to treatment for all CMT patients.” Allison Moore, CEO, Hereditary Neuropathy Foundation
Key Takeaways for Our CMT Community:
- Slowed Disease Progression: MRI scans at 24 months show slowed progression in patients taking govorestat.
- Sustained Improvements: Patients continued to show improved function and well-being through 18 and 24 months of treatment.
- Patient-Reported Benefits: A significant boost in CMT-Health Index (CMT-HI) scores shows patients are feeling better and moving better.
- Blood Sorbitol Levels Reduced: Govorestat significantly lowered toxic sorbitol levels in the blood—a key issue in CMT-SORD.
- Safe & Tolerated: The treatment remained generally safe with no major differences in side effects compared to placebo.
What’s CMT-SORD?
CMT-SORD is a rare and newly identified form of CMT, discovered in 2020. It’s caused by mutations in the SORD gene, leading to a buildup of sorbitol in the body. This can damage peripheral nerves and affect mobility, strength, and daily function.
What’s Govorestat?
Govorestat is an orphan drug designed to lower sorbitol levels and protect nerves. The INSPIRE trial tested its effect on symptoms, walking ability, and patient quality of life in people with CMT-SORD.
Trial Highlights
- Participants: 56 people with CMT-SORD
- Design: Randomized, placebo-controlled, double-blind study
- Duration: Up to 24 months of treatment
- Results: Improvements in walking, mobility, and daily life activities
What’s Next?
- 90% of patients have chosen to stay on Govorestat through the Open-Label Extension study
- Applied Therapeutics is planning to submit a New Drug Application (NDA) to the FDA later this year
- The CMT community is one step closer to a potential treatment for CMT-SORD
Learn More:
Visit www.appliedtherapeutics.com
Know someone with CMT Type 2 who hasn’t had genetic testing? They could have CMT-SORD. Share this news and ask HNF about getting tested.
Together, we’re moving closer to real treatments for CMT.
The Hereditary Neuropathy Foundation
www.hnf-cure.org | Join the GRIN registry to support research
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