Authored by James Sheppard
The Duchenne Muscular Dystrophy market has seen considerable advances in recent months, with companies Prosensa, PTC Therapeutics, Sarepta Therapeutics, Summit and considerable others announcing regular news on funding, IPOs, research, clinical and regulatory.
Prosensa, the Dutch based biotech announced that it was to raise extra funds through an initial public offering (IPO). Prosensa raised $78m from their IPO and immediately afterwards their share price rose by 54%. Prosensa is trading on the NASDAQ, the first cross-Atlantic IPO for 10 years. The news of the IPO was quickly followed by news from the FDA about Prosensa’s lead asset Drisapersen. The FDA announced that they had given Drisapersen ‘breakthrough’ status. The breakthrough status is distinct from other fast-track programmes in that it involves more intensive guidance from the agency on putting together an efficient drug development process. The achievement is yet another milestone for Prosensa in their race with Sarepta Therapeutics to develop exon skipping therapies. Prosensa were not the only company in DMD to have a successful IPO. US based biotech, PTC Therapeutics also announced that they had raised $125m from an initial offering on the NASDAQ. The money will be used in the continued development of PTC’s lead asset Ataluren.
Alongside the positive public offering news, UK based biotech Summit plc announced that it has received approximately £0.75m from the Australian DMD charity Save our Sons. Glyn Edwards, CEO of Summit said in a statement ‘Summit is delighted to enter this funding agreement with Save our Sons whose generous support will help advance the development of our lead utrophin modulator SMT C1100.’
In other positive news for the Duchenne community, US based biotech DART Therapeutics, announced the initiation of a phase IB/IIa of their DMD product HT-100. The study will enrol 30 patients and DART expects the study to be completed in 2014. Gene Williams, CEO of DART Therapeutics said in a statement, ‘Treating DMD will require a cocktail of therapies that will change over time. We believe that HT-100 could be a valuable part of this cocktail – even a mini cocktail on its own – because it demonstrates potential to address many different aspects of the disease in parallel, which could make it an exceptionally important therapy for all boys living with DMD.’
Sarepta Therapeutics, also announced its intention to seek early approval for its lead asset Eteplirsen. Eteplirsen is an exon 51 skipping drug that has just completed phase IIb clinical trials in the USA. The results were impressive and this has pushed Sarepta to submit the approval filings in the first half of 2014. Eteplirsen competes directly with GSK/Prosensa’s Drisapersen (also an exon 51 skipping drug) and the race is heating up to see who can reach the market first. It is highly likely that both GSK/Prosensa and Sarepta will have their respective exon skipping drugs under review by the FDA at the same time.
The Duchenne Muscular Dystrophy market is one of the most dynamic diseases areas within the rare diseases category. The market has moved forward considerably in recent years and the leading companies in the market are getting closer to the goal of bringing the first meaningful therapies to patients. With additional clinical data and regulatory strategies still playing out, the prospects of seeing products approved is a boost for those Duchenne patients who’ll benefit from these initial therapeutic products. However, considerable challenges still remain along the pathway toward new therapies in Duchenne.