June 10, 2014
Makindus, Inc. a specialty pharmaceutical company, received orphan drug designation for their lead product MI-100 from the U.S.Food and Drug Administration Office of Orphan Products Development for the treatment of Stargardt’s Disease. “The FDA’s approval of our orphan drug designation application for MI-100 to treat Stargardt’s disease is a key milestone for the company and we look forward to initiating our Phase 3 confirmatory trial early next year,” stated Manohar Katakam, President and CEO of Makindus. Makindus acquired all intellectual property and associated clinical data supporting multiple ocular indications through a licensing deal that was closed in 2013.
According to Kim Dukes, Makindus’ Chief Business Officer, “Stargardt’s is a progressive, debilitating disease with no currently approved treatments or methods to halt progression. We are thrilled with the approved orphan designation request and are looking forward to sharing details about the program with potential strategic partners at BIO 2014 in San Diego, June 23-27. We believe our product is an excellent fit for companies focusing on rare/ specialty /orphan /ophthalmic diseases.”
Orphan drug designation provides certain exclusivity benefits, tax credits for certain research and a waiver of the New Drug Application (NDA) user fee. Approximately 1:9,000 individuals suffer from Stargardt’s disease in the U.S. where there is no currently approved treatment.
About Makindus, Inc.
Makindus is a specialty pharmaceutical company currently developing MI-100, a legacy, Phase 3â€“ready molecule for Stargardt’s disease with 50 years of safety history in humans at significantly lower dose than that of the marketed product. Stargardt’s is a genetic disease caused by mutations in the ABCA4 gene. Age of onset is typically 8-12 years with significant variability in the speed of disease progression. There is currently no treatment for the disease nor any way to slow its progression. Makindus is seeking to license MI-100 to a strategic partner or obtain equity funding to progress the program to NDA filing in November 2017.
For more information, visit www.makindus.com