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Sarepta Announces Agreement With Lysogene for Gene Therapy

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Sarepta Therapeutics, Inc. (SRPT - Free Report) announced that it has signed a license agreement with French gene therapy focused company, Lysogene, related to the development of the latter’s central nervous system (“CNS”) targeting candidate, LYS-SAF302.

LYS-SAF302 is an AAV-mediated gene therapy, which is being evaluated in a phase II/III study for the treatment of Mucopolysaccharidosis type IIIA (MPS IIIA).

Per the terms of the deal, Sarepta will own exclusive commercial rights to LYS-SAF302 in the United States and other global territories outside Europe. Lysogene will retain the European commercial rights to the candidate. Lysogene will be responsible for the completion of the pivotal study, which is expected to start by 2018-end. The deal also states that Sarepta will be responsible for manufacturing of LYS-SAF302 as well as supply it to Lysogene. Sarepta will also get certain option rights to an additional gene therapy candidate targeting CNS.

Sarepta will pay Lysogene $15 million upon closing of the transaction and has committed payments of $26 million and $19 million in cash for 2018 and 2019, respectively. The company will also purchase $2.5 million worth of Lysogene equity. Under the terms of the deal, Sarepta is expected to pay a total of $125 million to Lysogene in potential milestone payments along with royalties on sales.

With this agreement, Sarepta expands its gene therapy portfolio to include 14 programs including the additional gene therapy candidate in this deal.

MPS IIIA is a rare inherited disorder, which may cause early death due to intractable behavioral problems and developmental regression. Per the press release, the neurodegenerative disease affects approximately one newborn baby in 100000 births and has no treatment options available. A successful development is likely to boost sales for Sarepta.

Meanwhile, Sarepta’s sole marketed drug, Exondys 51, has demonstrated impressive growth in the past few quarters on the back of strong demand among Duchenne muscular dystrophy (“DMD”) patients. The company’s lead pipeline candidate, golodirsen, is also being developed for DMD. The candidate has shown better efficacy than Exondys 51 in clinical study and a new drug application is expected to be submitted by the end of this year.

The company is also developing gene therapy for treating DMD. However, there are several other pharma companies developing gene therapies for treating DMD, which includes Pfizer (PFE - Free Report) , Solid Biosciences (SLDB - Free Report) and Scholar Rock (SRRK - Free Report) .

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