Shares of Sarepta Therapeutics, Inc. (SRPT - Free Report) have displayed a solid uptrend so far this year. The biotech company’s stock has rallied 38.1% compared with 3.8% increase of the industry in the said period.
The company has only one marketed product in its portfolio, Exondys 51, which received accelerated approval in September 2016. The drug is approved for treating patients with Duchenne muscular dystrophy (“DMD”), a rare genetic disorder affecting children who have confirmed mutation of the DMD gene susceptible to exon 51 skipping. The company is also developing other pipeline candidates including gene therapies for treating DMD.
The company has progressed well with the commercialization of Exondys 51, development of its lead pipeline candidate and collaboration for gene therapies in 2019 so far.
Exondys 51’s Impressive Performance
The drug generated sales of $87 million in the first quarter of 2019, an increase of 34.7% year over year on the back of strong demand trends. The drug has also grown sequentially over the past few quarters. Sales of the drug in 2018 were $301 million. The Zacks Consensus Estimate for second-quarter and full-year sales stands at $91.16 million and $390 million, respectively, representing significant growth.
The launch of a Managed Access Program in Europe and North America last year is also favorably impacting sales as the drug is now available to a larger patient population. The company remains on track with its post-marketing phase III PROMOVI study evaluating Exondys 51. Data is expected in 2019 and will form the basis for continued approval of the drug.
Moreover, settlement of a global patent litigation related to exon-skipping technology used in DMD therapies with BioMarin Pharmaceutical Inc. (BMRN - Free Report) in mid-2017 removed a major overhang for Sarepta. Per the settlement terms, BioMarin has granted Sarepta global exclusive rights to its DMD patent estate for Exondys 51 and all future exon-skipping products.
Exon-Skipping Pipeline Progress
Sarepta is focused on building its DMD pipeline beyond Exondys 51 by developing other exon-skipping treatments. In fact, the company has about eight exon-skipping candidates in its pipeline, including golodirsen and casimersen, which, if approved, can treat75-80% of the DMD population.
Sarepta’s lead candidate, golodirsen, has shown improved outcome in patients in a mid-stage clinical study compared to results achieved by Exondys 51. The candidate has demonstrated 100% response rate with 10.7 times increase in mean dystrophin protein production from baseline, whereas Exondys 51 achieved nearly three times increase in DMD patients. A new drug application (“NDA”) is under review in the United States. The FDA will provide its decision related to the approval of the candidate next month.
Sarepta remains on track to submit a NDA for casimersen shortly. The company is developing casimersen in a phase III study. Interim data from the study has shown that casimersen has achieved a 100% response rate with statistically significant mean increase of dystrophin protein production compared to placebo.
Sarepta is also developing gene therapies for treating DMD and other nervous disorders. The gene therapies have shown promising results so far in treating DMD patients by restoring dystrophin production in muscles throughout the body by delivering microdystrophin via adeno-associated virus. Sarepta is evaluating its most advanced gene therapy, SRP-9001, in a phase I/II study in DMD patients.
It is collaborating with other companies and academic institutions to boost its pipeline or manufacturing capacity for gene therapy. The company’s gene therapy portfolio currently has multiple programs in pre-clinical or early stages of development targeting several muscular dystrophy disorder.
Zacks Rank & Stocks to Consider
Sarepta currently carries a Zacks Rank #3 (Hold).
A couple of better-ranked stocks in the pharma sector include Aytu Bioscience, Inc. (AYTU - Free Report) and Axovant Sciences Ltd. (AXGT - Free Report) . Both the stocks carry a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Aytu’s loss estimates have narrowed from $2.39 to $2.20 for 2019 and from $1.15 to 80 cents for 2020 over the past 60 days. The stock has rallied 133.4% so far this year.
Axovant’s loss estimates have narrowed from $7.00 to $5.71 for 2019 and from $6.48 to $3.59 for 2020 over the past 60 days.
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