Sarepta Therapeutics, Inc. ( SRPT Quick Quote SRPT - Free Report) announced that it has signed a research collaboration and option agreement with privately-held Genevant Sciences for gaining access to the latter’s lipid nanoparticle (LNP) platform to be applied to Sarepta’s gene editing targets.
Per the agreement, Sarepta will assess and develop muscle-targeted LNPs, which will be used with its precision genetic medicine candidates, in collaboration with Genevant during early-stage development. The company will use the technology to develop more precise genetic medicines in its targeted segment, Duchenne muscular dystrophy.
Sarepta will have an option to gain rights to an exclusive license to Genevant’s LNP technology for up to four neuromuscular indications. Sarepta will pay Genevant approximately $50 million in near-term payments and will also pay undisclosed amounts on achieving certain development, regulatory and commercial milestones. Genevant is also eligible for tiered royalties on any future net sales from products developed under the agreement.
In the past year, the company’s shares have decreased 33% against the
industry‘s rise of 8.4%.
Sarepta is committed to develop advanced precision genetic medicine across multiple modalities to improve the utility and benefit of gene-based medicines. The company is developing several gene therapies targeting muscular dystrophies including DMD and central nervous system disorders.
Meanwhile, the company suffered a
significant setback earlier this month when it announced that its gene therapy candidate, SRP-9001, did not achieve the statistical significance for the primary functional endpoint in an early-stage study. However, the study met its primary biological endpoint of micro-dystrophin protein expression.
The candidate holds potential and attracted Swiss pharma giant
Roche ( RHHBY Quick Quote RHHBY - Free Report) to sign a collaboration deal with Sarepta in 2019 related to commercialization of SRP-9001 in ex-U.S. markets.
Meanwhile, we note that there are several other companies developing gene therapies for treating DMD including
Solid Biosciences ( SLDB Quick Quote SLDB - Free Report) , Audentes Therapeutics, and Pfizer ( PFE Quick Quote PFE - Free Report) . Successful development of gene therapies for DMD will increase competition in the field.
Apart from the gene therapy, Sarepta’s exon-skipping commercial portfolio of two DMD drugs (Exondys 51 and Vyondys 53) is doing well. In the first nine months of 2020, net product revenues were up 18.7% year over year to $333.2 million on the back of strong demand. The trend is expected to have continued in the fourth quarter. A regulatory application seeking approval for the third DMD drug (casimersen) is under review in the United States. A decision is expected next month.
The company is also developing next-generation exon-skipping candidate, SRP-5051, for the treatment of DMD. Last month, the company
announced data from a phase II study, which demonstrated proof-of-concept for the candidate. Zacks Rank
Sarepta currently carries a Zacks Rank #3 (Hold). You can see
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