Sarepta Therapeutics Inc. (NASDAQ:SRPT) is trying to bounce back after imploding early in the year over underwhelming clinical results on an ongoing gene therapy program. Amid the sell-off, management has come out fighting, reiterating that the company is increasingly advancing its multi-platform portfolio, having already achieved a string of milestones.
Clinical Trials Milestones
According to the Chief Executive Officer, Doug Ingram, a number of milestones have been achieved on the gene therapy and RNA platform. For starters, the company has already secured regulatory approval for AMONDYS 45.
Monday's becomes the third internally developed RNA therapy approved in the U.S. to treat Duchenne Muscular dystrophy. The fact that the company can now offer treatment to 8% of the Duchenne patients is an important milestone that cannot be taken for granted.
Likewise, Sarepta Therapeutics expects AMONDYS 45 to contribute to the steady growth of RNA product revenue going forward. The remarks come on the biotechnology company reporting fourth-quarter sales of $122.6 million, representing a 23% year-over-year increase. Full-year sales were up by about 20% to $455.9 million.
Amid the sales growth, Sarepta Therapeutics came out with a net loss of $2.40 a share against $2 a share expected. It marked the first time in four quarters that the company has failed to beat consensus estimates.
Q4 Milestones
In addition to solid financial results, Sarepta Therapeutics also achieved significant milestones in developing its pipeline. In January, the company reported positive clinical trials for two key value-driving assets in the development of Duchenne.
In addition, the company entered into a research collaboration with Genevant Sciences for lipid nanoparticles. Under the terms of the agreement, Genevant is to design and collaborate with the biotechnology company to develop muscle-targeted lipid nanoparticles that are to be applied on gene editing targets.
Sarepta owns rights to an exclusive license to the LNP technology in use for up to four neuromuscular indications.