Navigation Links
FDA Grants Orphan Drug Designation for Mithridion's MCD-386CR to Treat Progressive Supranuclear Palsy
Date:5/8/2011

MADISON, Wis., May 9, 2011 /PRNewswire/ -- Mithridion, Inc., a privately-owned clinical stage drug development company focusing on serious Central Nervous System (CNS) disorders, announces that the U.S. Food and Drug Administration (FDA) has awarded Orphan Drug designation for MCD-386CR, its lead drug candidate, for the treatment of Progressive Supranuclear Palsy (PSP).

Orphan status entitles Mithridion to seven years of market exclusivity upon approval of MCD-386 for treating PSP, and to apply for grant funding to contribute to clinical trial costs, tax credits, and a waiver of certain FDA fees.  Orphan status is intended to encourage the development of drugs for diseases affecting fewer than 200,000 persons in the USA.

PSP is a progressive brain disease in which neurons degenerate in regions of the brain vital for eye movements, balance, walking, speech, and cognition.  Cognitive impairment typically involves slowed thinking, and difficulties with reasoning, planning, and shifting between tasks, caused by dysfunction of the brain's executive functions.  This has been called "dysexecutive  syndrome."

"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, Ph.D., the company's Chief Executive. "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."

As a part of its recently-articulated strategy, Mithridion is actively exploring opportunities to develop its drug candidates in niche market opportunities in serious CNS or brain disorders, in addition to more common diseases, such as Alzheimer's disease and schizophrenia.  Several promising disease targets in addition to PSP have been identified and are currently being evaluated.  The company believes that it will be able to create additional high value and strong entry barriers through this strategy, which it intends to fund using a combination of equity capital and non-dilutive funding.  Furthermore, the company believes that it can reduce clinical trial costs and improve its ability to demonstrate proof-of-efficacy for its candidate drugs by selecting for clinical trials neurological disorders, such as PSP, with more "pure play" pathogenesis or symptoms, and by using novel biomarkers and neuropsychological tests specific for particular domains of cognition, such as executive functions.

The announcement coincides with the 6th Annual Neurotech Investing and Partnering conference being held at the St. Regis Hotel in San Francisco, CA, on May 9-10, 2011, at which Trevor Twose will be available to discuss this and other developments at Mithridion.

MCD-386 is a highly selective agonist (activator) for M1-type acetylcholine muscarinic receptors, effectively mimicking acetylcholine in a selective way to achieve desired therapeutic effects while minimizing side effects.  Acetylcholine is a chemical 'neurotransmitter' vitally involved in maintaining brain health and in many brain functions such as memory, cognition, executive functions, and attention.  MCD-386 was designed to stimulate these and other key brain functions that have been compromised by the deficit of acetylcholine that is a characteristic feature of PSP.  

MCD-386 has completed Phase I single- and multi-dose clinical trials in 55 healthy volunteers, including 29 subjects who received a controlled release tablet formulation.  MCD-386CR has so far proven to be well-tolerated at the doses tested.  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 development of MCD-386 was supported in part by NIH SBIR grant AG20454 from the National Institute of Aging, and the NIH RAID Program. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institute of Aging, or the National Institutes of Health.


'/>"/>
SOURCE Mithridion, Inc.
Copyright©2010 PR Newswire.
All rights reserved

Related medicine technology :

1. US FDA Grants Orphan Drug Status to Immtechs Pafuramidine for Treatment of African Sleeping Sickness
2. Chinese SFDA Grants Immtech Fast Track Status
3. Ethics Group Asks USAID Inspector General to Investigate Legality of Grants to Soros Groups
4. NTMir Announces Research Grants for Epidemiology and Household Water as Source of Infection
5. FDA Grants Approval to Extend U.S. Enrollment and Clinical Trial
6. FDA Grants Priority Review for Daiichi Sankyo, Lilly Drug, prasugrel
7. FDA Grants Priority Review for ACIPHEX(R) (rabeprazole sodium) sNDA for Short-Term Treatment of GERD in Adolescents
8. FDA Grants Priority Review for PROMACTA(R) (eltrombopag)
9. FDA Grants NeoVistas Request to Expand CABERNET Trial to 30 Sites in the U.S.
10. FDA Grants Market Clearance for the LipiScan(TM) Coronary Imaging System Developed by InfraReDx, Inc. - A Novel Spectroscopy System for the Identification of Lipid Core Containing Plaques of Interest in the Coronary Arteries
11. FDA Grants Pediatric Exclusivity for TOPAMAX(R), Extending the Marketing Exclusivity by Six Months
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:2/12/2016)... February 12, 2016 ... vermerkt)   http://www.sedar.com ) und ... abrufbar.    --> http://www.sedar.com ... http://www.telestatherapeutics.com abrufbar.    --> ... heute seinen Konzernabschluss des zweiten Quartals ...
(Date:2/12/2016)...  Memorial Hermann Health System has teamed up with ... bring a one-of-a-kind experience to pediatric patients at ... as 360-degree video and Google Cardboard, Howard was able ... giving the patients and their families an unexpected, and ... on video . Memorial Hermann IRONMAN ...
(Date:2/12/2016)... , Feb. 12, 2016  Eli Lilly and Company ... Court decided the Alimta® (pemetrexed disodium) vitamin regimen patent would ... the UK, France , Italy ... to dilute the product only with dextrose solution.  ... 2015, the UK Court of Appeal held that Lilly,s patent ...
Breaking Medicine Technology:
(Date:2/13/2016)... ... , ... Valentine’s Day is nearly upon us, and most singles could probably ... flawless hair, and a sparkling personality are all well and good, but if somebody ... home with Rover. (Actually, man’s best friend might not even want to be near ...
(Date:2/13/2016)... ... February 13, 2016 , ... In its newly ... vein visualization technology should be used to ensure patient safety when placing an ... INS Standards mandate the use of vein visualization technology in patients with difficult ...
(Date:2/12/2016)... ... 12, 2016 , ... According to an article published February 4th ... significant portion of hernia repairs throughout the United States. Commenting on this article, Beverly ... that this trend has not only been expected, but it seems to be a ...
(Date:2/12/2016)... ... February 12, 2016 , ... CDRH Enforcement Trends: , Back to the Future , ... , As Winston Churchill said, “Those who don’t learn from history are doomed to ... expect when they come knocking this year. But that takes time. , Take a ...
(Date:2/12/2016)... ... February 12, 2016 , ... T.E.N., a ... closed for the ISE Southeast Awards 2016. Finalists and winners of the ISE® ... Southeast Executive Forum and Awards Gala on March 15, 2016 at the Westin ...
Breaking Medicine News(10 mins):