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Sarepta Therapeutics (Nasdaq: SRPT) | Company Profile, History, Competitors


Sarepta Therapeutics (NASDAQ: SRPT) develops RNA-based therapeutics for rare and infectious diseases.  It was founded in 1980 and headquartered in Cambridge, MA.

Sarepta focuses on treatments related to Duchenne Muscular Dystrophy (DMD). Their leading drug candidate Exondys 51 is undergoing marketing and is a therapeutic for patients with DMD disease.

Key Product Portfolio (2017)

 Brand     Status      Description
 EXONDYS 51 Marketing DMD amenable to exon-51 skipping
 SRP-053 Phase 3 DMD amenable to exon-53 skipping
 SRP-045 Phase 3 DMD amenable to exon-45 skipping
 Ezutromid Phase 2  DMD
 PPMO Preclinical DMD amenable to exon skipping
 Microdystrophin Gene Therapy Preclinical DMD
 GALGT2  Preclinical DMD

Exondys 51 Drug Candidate

Exondys 51 is a lead drug for Sarepta. Exondys 51 is an IV-administered antisense RNA oligomer that enables exon-51 skipping from dhystrophin mRNA transcripts in patients with Duchenne Muscular Dystrophy (DMD).

The number of DMD patients globally is hard to pinpoint. Large studies have suggested that DMD occurs in 1/5000 newborn births. Survival estimates had been historically limited to mid-20s, but now is in mid-30s due to modern care.


Sarepta's approved drugs may not ramp up as quickly as expected. Drugs undergoing clinical trials may not receive approval.


Douglas Ingram Esq. (CEO): Douglas Ingram serves as CEO since 2017. Prior, he was CEO of Chase Pharmaceuticals. He has also held positions as President of Allergan and EVP Europe & Africa at Allergan. He as also an attorney at Gibson Dunn & Crutcher LLP.

Sandesh Mahatme (CFO): Sandesh Mahatme serves as CFO since 2012. Prior, he was VP Corporate Strategy at Celgene and Senior Counsel at Pfizer.

Gilmore O'Neill (CMO): Gilmore O'Neill serves as Chief Medical Officer since 2018. Prior, Dr. O'Neill was a late clinical researcher at Biogen and was an instructor at Harvard Medical School. Dr. O'Neill graduated with a Bachelor of Medicine from University College Dublin and a Masters of Medical Sciences from Harvard University. Dr. O'Neill is the recipient of the Lefler Fellowship in the Department of Neurobiology at Harvard Medical School. He is the author of numerous publication on multiple sclerosis and has served as a neurology peer reviewer for medical literature.


 $ in millions    2013    2014 2015 2016 2017
 Revenues $14 $10 $1 $5 $155 
 Gross Profit$14 $10 $1 $5 $147 
 Operating Profit ($90)($134) ($220) ($267)  ($171)
 Net Income ($112)($136)($220) ($267) ($51)
 EPS ($3.31) ($3.39)($5.20)($5.49)($0.86)