ADX71441 -- A New drug in Clinical Trials for CMT1A

Thomson Reuters – one of the world’s leading source of information – reports the approval to initiate a First-In-Man clinical study for CMT1A. The study will be conducted by Addex Therapeutics, a company based in Geneva, Switzerland, famous for its research on the field of small molecule drugs. Past products of the same company are being used to treat Parkinson’s disease.

The company website confirms the news, and names this new experimental drug ADX71441. Although the company head quarter is based in Switzerland, the approval to start a First-In-Man clinical trial has been issued by the regulatory authorities in the Netherlands. In fact the company plans to initiate the testing at the Centre of Human Drug Research in Leiden, The Netherlands.

The molecule drug is designed to be exquisitely selective and target (some of) the causes of Charcot-Marie-Tooth Type 1A and eventually other similar neuropathy. It expects to deliver Phase 1 top-line safety data by the end of 2013, and move to a more practical Phase 2a testing in 2014.

The doctor in charge for the project is the Italian born Sonia Poli, Ph.D., Head of Non Clinical Development at Addex. Dr Poli has obtained a doctorate in Industrial Chemistry at the University of Milan, Italy, and completed a post doctoral fellowship at the CNRS, in Paris, France. Besides her work with Charcot-Marie-Tooth, she has developed an expertise in many central nervous system disease (CNS), including Alzheimer’s disease, Parkinson’s disease, bi-polar disorders and anxiety.

Addex reported as follows on its website in October 2013:

Addex ADX71441 Dose Dependently Reduced PMP22 Expression Comparable to Baclofen in a Pre-Clinical Transgenic Model of Charcot-Marie-Tooth 1A Disease

Geneva, Switzerland, 3 October 2013 - Addex Therapeutics (SIX: ADXN), a leading company pioneering allosteric modulation-based drug discovery and development announced today that ADX71441 dose dependently reduced PMP22 expression comparable to baclofen in a preclinical transgenic model of Charcot-Marie-Tooth 1A disease (CMT1A).

ADX71441 was studied in the transgenic CMT1A rat model which displays a 1.6-fold PMP22 overexpression (mRNA level) and exhibits clinical abnormalities, such as reduced nerve conduction velocity and lower grip strength that closely mimic findings in CMT1A patients. In a 5 day comparative study with baclofen, a dose-response was successfully established for orally administered ADX71441. ADX71441 given orally once daily significantly reduced PMP22 mRNA expression at 3 mg/kg and 6 mg/kg (0.98-fold±0.49 and 0.93-fold±0.35, respectively). Baclofen given orally twice daily reduced PMP22 mRNA expression at 3mg/kg (0.91-fold±0.25). ADX71441 did not significantly reduced PMP22 mRNA expression at 1mg/kg (1.48-fold±0.26) compared to CMT vehicle group (1.6-fold±0.19).

"These findings in the CMT1A transgenic rat model which show that ADX71441 has comparable efficacy to Baclofen are very promising," Professor Michael Sereda, of the Max-Planck Institute of Experimental Medicine, Göttingen, Germany, in whose laboratories the study was performed. "These results are consistent with previous reported data which suggest that intraperitoneal application of ADX71441 could lower toxic PMP22 overexpression and potentially delay the progression of the disease. Oral application of ADX71441 may therefore offer a unique therapeutic opportunity for CMT1A patients."

I am thrilled to hear this good news! Absolutely thrilled!