- Rare disease drugmaker Sarepta released limited portions of fourth quarter financial results at the J.P. Morgan Healthcare Conference on Tuesday, highlighting relatively strong returns for its most talked about treatment.
- Exondys 51 (eteplirsen), a drug for patients with Duchenne muscular dystrophy (DMD), brought in $5.4 million during the quarter, with 250 patients on the drug since its approval in late September.
- DMD affects mostly boys and arises from a genetic mutation that negatively affects the body's production of dystrophin, a protein vital to muscle development. While estimates vary regarding its patient population, the Centers for Disease Control approximated that in 2009, the disease affected 16 out of every 100,000 males.
Given the controversy surrounding the Food and Drug Administration's approval of Exondys 51, which is the first and only marketed treatment for DMD, as well as the small patient population the drug targets, its understandable why Sarepta was eager to tout sales results.
The announcement and disclosure in the company's 8-K reinforced investor confidence. The Cambridge, MA-based biopharma saw its stock rise 20% to $37.89 per share by close of market on Tuesday.
In a presentation delivered at the J.P. Morgan Healthcare Conference, Sarepta outlined the next goals for the drug. The company plans to gain approval in Europe and "other jurisdictions," then pursue other different methods for treating DMD.
Exondys 51, as its name suggests, works on a section of the gene that codes for dystrophin, called exon 51.While DMD arises in about 13% of patients due to mutations in that piece of DNA alone, targeting other sections where mutations sprout up can also combat the disease. As such, Sarepta is looking at developing treatments that target exons 45 and 53, which are found along with exon 51 mutations in 29% of all DMD patients, according to the company's presentation.
Sarepta also revealed it has nearly $330 million in cash and cash equivalents, according an 8-K filing with the Securities and Exchange Commission. What's more, the company announced two agreements — one for licensing, another for research — with Nationwide Children's Hospital directed at gene therapies.