The orphan drug status provides the company for marketing MCD-386CR as a treatment for PSP for seven years.
MCD-386 is a selective agonist (activator) for M1-type acetylcholine muscarinic receptors, mimicking acetylcholine in a selective way to achieve desired therapeutic effects while minimizing side effects.
The drug was well tolerated in previous trials.
Pharmacokinetic information obtained during the trials showed sustained release of drug substance from the formulation, and, together with other trial results, provides a strong foundation for further rational development of MCD-386.
Mithridion CEO Trevor Twose said orphan status will help them immeasurably to bring together the resources and support needed to evaluate MCD-386CR in this rare but important disease, and indeed in other devastating brain diseases for which there are no current therapies.
"Based on results in preclinical studies, we believe MCD-386CR potentially will help restore cognition, so vital to human functioning, and potentially could treat the underlying processes causing the degeneration of neurons in PSP," Twose said.