Addex ADX71441 Included in the NINDS Anticonvulsant Screening Program

Addex ADX71441 Included in the NINDS Anticonvulsant Screening Program

Geneva, Switzerland, 25 November 2015
 – Addex Therapeutics (SIX: ADXN), a leading company pioneering allosteric modulation-based drug discovery and development announced today entering into an agreement with the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH), enabling the Company to submit ADX71441, a GABAB receptor positive allosteric modulator (PAM) to the Anticonvulsant Screening Program (ASP) for evaluation in preclinical models as a potential treatment for epilepsy.

Established in 1975 to stimulate the discovery of new drug therapies for epilepsy, the ASP has evaluated  more than 32,000 compounds submitted from more than 600 participants world-wide and has contributed to 10 marketed anti-seizure drugs.

Alterations of the GABAergic system play a major role in epilepsy. Malfunctioning of the GABAA system has long been known as important in controlling seizures, however an important role of the GABAB receptor in epilepsy has been emerging more recently and the involvement of GABAB receptors in controlling seizures has been reported in various models of epilepsy.  GABAB receptor PAMs have a similar pharmacological profile to the orthosteric agonist, baclofen, while showing a more favorable tolerability profile and they offer a unique therapeutic opportunity as they potentiate only activated receptors. ADX71441 is a GABAB PAM discovered and developed by Addex which has completed preclinical development and is ready to enter Phase I clinical evaluation. 

"The role of GABAB receptors in epilepsy is increasingly recognized as important and the allosteric mechanism of action of ADX71441 represents a significant opportunity to drug this receptor in a more refined manor than currently available drugs." explained Sonia Poli, CSO at Addex”

“The inclusion of ADX71441 in the NINDS ASP will provide Addex with invaluable information for the further development of ADX71441,” commented Tim Dyer, CEO at Addex. “The opportunity to participate in the NINDS ASP is another example of us executing our strategy to advance our pipeline through collaborations with cutting edge academic and governmental research groups.”

About GABAB Activation and ADX71441 
Activation of gamma-aminobutyric acid subtype B (GABAB) receptor, a Family C class of GPCR, is clinically & commercially validated. Generic GABAB receptor agonist, baclofen, is marketed for spasticity and some spinal cord injuries, and French health authorities have approved its use for the treatment of alcoholism on a "case by case" basis. Baclofen is also used off label for a number of other indications including overactive bladder (OAB), but its utility is limited due to variety of side effects and rapid clearance. ADX71441 is a potent selective positive allosteric modulator (PAM) which potentiates GABA responses at the GABAB receptor. ADX71441 is a novel, first-in-class, oral, small molecules that has demonstrated excellent preclinical efficacy and tolerability in several rodent models of pain, anxiety, addiction and OAB and have also proven efficacy in a genetic model of Charcot-Marie-Tooth Type 1A disease (CMT1A). ADX71441 differs from the generic drug baclofen in that it is a positive allosteric modulator rather than an orthosteric agonist at the GABAB receptor. ADX71441 only acts when the natural ligand (GABA) activates the receptor, and therefore respecting the physiological cycle of activation. It has been proposed that PAMs produce less adverse effects and lead to less tolerance than direct agonists. Addex has published positive data with ADX71441 in two models of alcohol abuse in mice, the ethanol binge-like drinking, drinking-in-the-dark (DID) and a model of long-term, excessive drinking, intermittent access to alcohol (IAA).

About Epilepsy
Epilepsy represents a group of neurological diseases characterized by epileptic seizures which tend to recur, with no apparent immediate underlying cause. In epilepsy, the normal pattern of neuronal activity becomes disturbed, causing strange sensations, emotions, and behavior or sometimes convulsions, muscle spasms, and loss of consciousness. The epilepsies have many possible causes and there are several types of seizures. The cause of most cases of epilepsy is unknown, although some people develop epilepsy as the result of brain injury, stroke, brain tumor, and substance use disorders. Anything that disturbs the normal pattern of neuron activity—from illness to brain damage to abnormal brain development—can lead to seizures. Seizures can be controlled with modern medicines in about 70% of cases with some drugs being more effective for specific types of seizures. In those whose seizures do not respond to medication, then surgery, neurostimulation, or dietary changes may be considered. About 1% of people worldwide (65 million) have epilepsy and nearly 80% of cases occur in developing countries. 

About Addex Therapeutics
Addex Therapeutics ( is a biopharmaceutical company focused on the development of novel, orally available, small molecule allosteric modulators for neurological disorders. Addex lead drug candidate, dipraglurant (mGluR5 negative allosteric modulator or NAM) has successfully completed a Phase IIa POC in Parkinson’s disease levodopa-induced dyskinesia (PD-LID), and is being prepared to enter Phase IIa for PD-LID. In parallel, dipraglurant’s therapeutic use in dystonia and treatment resistant depression is being investigated. Addex second clinical program, ADX71149 (mGluR2 positive allosteric modulator or PAM) is being developed in collaboration with Janssen Pharmaceuticals, Inc. Addex also has several preclinical programs including: ADX71441 (GABAB receptor PAM) which has received regulatory approval to start Phase I and is being investigated for therapeutic use in Charcot-Marie-Tooth (Type 1A) disease, alcohol use disorder and nicotine dependence; mGluR4PAM for drug abuse and dependence, Parkinson’s disease and other neurodegenerative diseases; mGluR2NAM for treatment resistant depression and cognitive deficits; mGluR7NAM for psychosomatic disorders, TrkBPAM for neurodegenerative disorders; and GLP1PAM for type 2 diabetes. Allosteric modulators are an emerging class of small molecule drugs which have the potential to be more specific and confer significant therapeutic advantages over conventional "orthosteric" small molecule or biological drugs. Addex allosteric modulator drug discovery platform targets receptors and other proteins that are recognized as essential for therapeutic intervention – the Addex pipeline was generated from this pioneering allosteric modulator drug discovery platform.

About the NINDS Anticonvulsant Screening Program
The ASP service is provided at no cost, and participants include academic institutions, pharmaceutical and biotechnology companies, and other organizations. The ASP participant agreement signed by NINDS and the participant delineates protection of intellectual property and confidentiality. NINDS ASP staff communicates with participants to plan testing and, based on outcome, determine next steps. In response to recent program reviews, the ASP is focusing on identifying new treatments to address the unmet medical needs in epilepsy, including treatment-resistant epilepsy and disease prevention. For more information, go to

Press Contact:

Tim Dyer
Chief Executive Officer
Addex Therapeutics
Telephone: +41 22 884 15 61
Email: pr(at)

Disclaimer / Forward-looking statements: This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Addex Therapeutics Ltd. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements.