Biopharma firm Sarepta Therapeutics (SRPT 42.42, +8.34) trades about 24.5% higher this afternoon, to eight-month highs, after last night’s Q2 beat and guidance raise.
By all accounts, SRPT blew out street expectations for its Q2 report as a loss of $0.46 per share handily beat market expectations. Revenues were also firmly ahead of market expectations at $35.01 million.
The company’s EXONDYS 51 (eteplirsen) treatment is performing well, and as such SRPT raised revenue guidance for the full year to be between $125-130 million (up from about $95 million). Management pointed to the strong launch of EXONDYS 51 as reasoning for the guidance update, and with upcoming 4053-101 dystrophin data and potentially having up to seven experimental DMD therapies in the clinic, there seems to be a lot to smile about at SRPT right about now.
As a bit of background, EXONDYS 51 uses SRPT’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. EXONDYS 51 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. Data from clinical studies of EXONDYS 51 in a small number of DMD patients have demonstrated a consistent safety and tolerability profile. Essentially, eteplirsen is a treatment for select mutations which cause Duchenne muscular dystrophy.
The strong results come on the heels of the July 18 announcement that the company had settled an ongoing license dispute with BioMarin Pharma (BMRN 87.50, +0.27 +0.31%), giving SRPT global exclusive rights to BMRN’s DMD patent estate for EXONDYS 51 and all future exon-skipping products.
With an EU decision on track for early 2018, SRPT is shaping up for some possible strong months ahead. With last night’s guidance raise and the stellar performance from EXONDYS 51, the stocks +49% gains YTD seem justified.