With gratitude and respect: a note to the CMT community from NMD Pharma
Dear members of the CMT patient and caregiver community,
We wanted to take a moment to communicate with you directly, following our recent press release announcement about ignaseclant (previously called NMD670) – and first and foremost, to say thank you.
The announcement had to do with the successful results of our first clinical trial for ignaseclant in CMT. One aspect of this study that matters deeply to us is who participated. Individuals living with many different genetic subtypes of both CMT1 and CMT2, and even a few with CMTX, took part. Seeing positive signals related to muscle strength and function across such a genetically diverse group gives us cautious scientific encouragement that this approach may one day have relevance across the broad CMT community, rather than being limited to a single subtype. While much more work remains, this was an important and meaningful starting point.
We also want to acknowledge something remarkable that happened because of you. The level of interest, engagement, and coordination across the patient community and advocacy leadership allowed this study to recruit and enroll far more quickly than expected. As a result, the trial completed nearly six months earlier than planned! That kind of progress does not happen without an informed, motivated, and well-connected community. This was a shared accomplishment, and we are deeply grateful.
At the same time, we want to also honestly acknowledge some shortcomings. During the study, some individuals experienced confusion or frustration when exploring whether they might qualify to participate, and at times communication or coordination between NMD Pharma, clinical sites, and patients fell short. We sincerely apologize for those experiences. We are taking these learnings seriously, and feedback from participants, caregivers, and the broader community is already shaping how we design and conduct future studies.
You may have read that the study showed signals suggesting improvements in muscle strength and function, and that ignaseclant was generally well tolerated. These findings support continued study, but we also want to be very clear: ignaseclant remains investigational, and early results do not guarantee success in future trials or regulatory approval. Progress in CMT research is rarely linear, and we recognize that this community has experienced both hope and disappointment in the past.
Our commitment is to move forward carefully, guided by data, clinicians, regulators (those who make sure we all “follow the rules”), and critically – all of you. We view this as a step-by-step effort, built with transparency, humility, and collaboration. What motivates us most is not a simple improvement on a test within a study, but the broader mission we share with this community: to explore new ways to safely improve and sustain muscle strength, function, and quality of life, alongside existing and future therapies.
Thank you for your trust, your engagement, and your willingness to partner with us. We remain committed to listening, learning, and keeping you informed as this work continues.
If you have any questions about our press release, you can find additional information on our website www.nmdpharma.com, email us at co*****@*******ma.com or email me directly (see my email address below).
On behalf of all of us at NMD Pharma, with gratitude and respect,
Dan Brennan
SVP, Corporate Strategy, Communications & Patient Engagement
NMD Pharma
Email: dj*@*******ma.com
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