CNS drug wins orphan status

The clinical-stage drug-development company Mithridion has won orphan status from the US Food and Drug Administration (FDA) for a potential treatment for progressive supranuclear palsy (PSP), a progressive brain disease in which neurons degenerate in regions of the brain that control eye movements, balance, walking, speech, and cognition.

The phase I drug, MCD-386CR, is the lead product in Mithridion's pipeline, which is focused on serious central nervous system (CNS) disorders.

Orphan drug status, which is designed by the FDA to encourage the development of treatments for diseases affecting fewer than 200,000 Americans, carries with it a number of benefits to the drug sponsor, including tax credits, access to grant funding for clinical trials, accelerated FDA approval, seven years of marketing exclusivity after drug approval, and potential exemption from the FDA's prescription drug application fee.

"Orphan status will help us 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," said Trevor M Twose, the company's CEO.

PSP is characterised by a deficiency in the neurotransmitter acetylcholine, which is involved in functions like memory and cognition. MCD-386 is an agonist for M1-type acetylcholine muscarinic receptors designed to mimic acetylcholine in a selective way to produce therapeutic effects while minimising side effects.

The phase I single- and multi-dose clinical trials for MCD-386 tested the drug in 55 healthy volunteers, including 29 subjects who received a controlled-release tablet formulation.

"Extensive pharmacokinetic information obtained during the trials demonstrated 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," the company said.