Progressive Supranuclear Palsy
Milestone: Ready to start Phase 2a study (Timings subject to financing / partnering)
Dipraglurant is a metabotropic glutamate receptor subtype 5 negative allosteric modulator, or mGlu5 NAM, that we are developing for post-stroke sensorimotor recovery.According to the World Stroke Organization (www.world-stroke.org), over there are 12.2 million strokes each year globally which can lead to motor paralysis, loss of sensory function, impaired autonomic functions such as bladder/bowel control, impaired cognition leading to deficits in communication, attention and memory, and accompanied by pain. There are currently no drugs to support sensorimotor recovery and current therapies rely on retraining and physiotherapy, with rehabilitation, largely partial, taking 6 month or more. Functional recovery by stimulating network connectivity in the brain has been demonstrated post-stroke preclinically with dipraglurant which significantly restored functional control after just three days of once-daily treatment.
We are conducting additional in vivo studies with dipraglurant in animal models of stroke and subject to funding, we plan to commence a Phase 2a study. There is a large unmet need in post-stroke sensorimotor recovery, and we believe this innovative approach represents a significant commercial opportunity.
We are also investigating dipraglurant’s suitability for traumatic brain injury, or TBI, recovery, where we believe the mechanism of action would be similar to stroke recovery and where the fast onset could be advantageous.
Milestone: IND enabling studies ready to start in 2025* (Timings subject to financing / partnering)
We have selected a drug candidate to advance our own independent GABAB PAM program for the treatment of chronic cough.
This target is clinically validated with baclofen, an orthosteric agonist of GABAB, is used off label to treat chronic cough patients. However, baclofen’s use is limited by serious side-effects, short half-life and gradual loss of efficacy during chronic treatment. By more precisely targeting the GABAB receptor with a PAM we aim to have a best-in-class treatment with improved tolerability suitable for the chronic nature of this disease.
This indication has a significant unmet medical need and represents a solid commercial opportunity.Subject to funding we plan to start IND enabling studies in 2025.
Milestone: IND enabling studies started in H2 2024
Our partner, Indivior has selected a GABAB PAM drug candidate from our research program for future development in substance use disorder and will now undertake all future development of their selected compound.
Under the terms of the agreement, Addex is eligible for payment of up to USD 330 million on successful achievement of prespecified regulatory, clinical and commercial milestones as well as tiered royalties on the level of net sales from high single digits up to low double-digit.
We believe that substance use disorder is an indication with a significant commercial opportunity. Existing therapies often do not provide effective control of symptoms or have side effects that discourage adherence. Subject to regulatory approval, we believe that GABAB PAM may offer an innovative and differentiated treatment approach from existing therapies and may provide benefit to patients.
This program is currently in IND enabling studies phase.
