Tokyo-based Daiichi Sankyo said it has treated the first patient in a clinical trial in Japan for Duchenne muscular dystrophy candidate drug DS-5141b in development with Japan's Orphan Disease Treatment Institute.Daiichi Sankyo CEO Joji Nakayama
The Phase I/II study is aimed at evaluating the safety, tolerability, efficacy, and pharmacokinetics. Duchenne affects one in 3,500 newborn males regardless of ethnic background, the company said in a press release.
Several firms have struggled to get potential Duchenne muscular dystrophy therapies past regulators in the U.S. recently, including Novato, California-based BioMarin ($BMRN) for drisapersen and Cambridge, Massachusetts-based Sarepta Therapeutics ($SRPT) for eteplirsen.
The Daiichi candidate, which uses the API, ENA oligonucleotide*2, is "a modified nucleic acid made using proprietary technology." The technology was developed with the Orphan Disease Treatment Institute and the Innovation Network Corporation of Japan and a fund managed by Mitsubishi UFJ Capital, the company said, citing a Feb. 14, 2013, release.
"Because the drug induces exon 45 skipping of a dystrophin mRNA to promote incomplete but functional dystrophin production, it is expected to be an effective treatment for DMD," Daiichi said in a statement. The company hopes to obtain manufacturing and marketing approval by the end of 2020.
Daiichi has cut staff in the U.S. and rejigged or closed operations elsewhere as it moves to replenish a portfolio with specialty care drugs following patent losses for the blood pressure drug Benicar and diabetes treatment Welchol.
Earlier this month, Japan's Ministry of Health, Labor and Welfare announced reviews of another 5 candidates for its fast-track approval process known as Sakigake, including a gene therapy backed by Daiichi and developed at the University of Tokyo's Institute of Medical Science dubbed G47Δ to treat brain tumors and in Phase II trials.
- here's the release
- and a trial guide on clinicaltrials.gov