On September 22, 2021 The Charcot–Marie–Tooth Association (CMTA), the largest philanthropic funder of CMT research worldwide, and Addex Therapeutics (SIX: ADXN and Nasdaq: ADXN), a clinical-stage pharmaceutical company pioneering allosteric modulation-based drug discovery and development, reported a collaboration to investigate a potential therapy for CMT type 1A (CMT1A), the most common subtype of the disease, which affects approximately 1.5 million people (Press release, Addex Therapeutics, SEP 22, 2021, View Source [SID1234590196]).
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The primary goal of the collaboration is to evaluate the benefit of Addex’s proprietary positive allosteric modulator’s (PAM’s) targeting the gamma-aminobutyric acid subtype B (GABAB) receptor in rodent models of CMT1A. The GABAB receptor has previously been shown to be instrumental in controlling the overexpression of Peripheral Myelin Protein-22 (PMP22) in a rat model of CMT1A. Elevated PMP22 is closely associated with the disabling peripheral neuropathy that accompanies CMT1A.
The CMTA’s Strategy to Accelerate Research (STAR) connects leading CMT clinicians and researchers with pharmaceutical partners committed to developing treatments and a cure for CMT. Strategic alliances with pharmaceutical partners like Addex Therapeutics support drug development efforts to deliver therapies to CMT patients.
"We are excited to establish this partnership with Addex Therapeutics as they work to advance their PAM’s to treat CMT1A," said CMTA’s CEO Amy Gray. "Strategic research partnerships with companies like Addex is a central part of our strategy to accelerate the development of treatments for the CMT community. Since launching STAR 12 years ago, the CMTA has been able to establish research partnerships with almost 40 pharmaceutical and biotech companies, and leading research labs around the world. We share Addex’s enthusiasm and passion for developing life-changing treatments for patients."
The research alliance with Addex will include joint study planning aimed at the chronic dosing of select GABAB PAM’s in rodent models of CMT1A, followed by detailed assessments aimed at measuring the improvement of key outcomes. These outcome measures include biomarkers, motor function, electrophysiology and peripheral nerve histology. This is made possible through the CMTA’s preclinical testing alliance: www.cmtausa.org/our-research/for-researchers/cmta-preclinical-testing-network/
"By combining the expertise of both the Charcot-Marie Tooth Association with our proven track record in the discovery and development of allosteric modulators, we aim to further understand how targeting GABAB with a positive allosteric modulator could benefit patients with CMT1A," said Tim Dyer, CEO of Addex Therapeutics. "As we move towards IND enabling studies, we look forward to working with the CMTA to build scientific evidence supporting our approach to tackle this tremendous unmet need."