Sarepta Expands Gene Therapy Pipeline in $30 Million Equity Deal with Lacerta

drug pipeline

Cambridge, Mass.-based Sarepta Therapeutics expanded its gene therapy pipeline up to 11 in an equity deal with Lacerta Therapeutics. Investing $30 million into Alachua, Florida-based Lacerta Therapeutics, Sarepta gained access to the company’s AAV-based CNS-targeted gene therapies.

Lacerta focuses on AAV-based gene therapy for CNS diseases and lysosomal storage disorders. It was founded on technology licensed from the University of Florida. As part of the deal, Sarepta gains the company’s expertise in the area, access to Lacerta’s capsid screening and proprietary OneBac manufacturing platform and process for the licensed products and adds three CNS-focused gene therapy programs.

Sarepta, best known for its Duchene muscular dystrophy (DMD) drug Exondys 51, has 20 programs, with eight of them—now 11—gene therapy products.

In terms of the three CNS gene therapy assets, Lacerta will continue to handle most of the preclinical development. Sarepta will lead clinical development and commercialization. Although financial details weren’t released, Lacerta will be eligible for development and sales-based milestones and single-digit royalties on any net sales.

“Today’s investment with Lacerta bolsters Sarepta’s position as a leader in precision genetic medicine and moves us forward on our mission to deliver life-enhancing therapies to those living with underserved diseases and in so doing to become one of the most meaningful global genetic medicines companies in the coming few years,” said Doug Ingram, Sarepta’s president and chief executive officer, in a statement.

Although the companies’ release does not specify which three specific programs Sarepta is picking up, Lacerta has six programs split between CNS disorders and lysosomal storage diseases. They include Pompe disease, Sanfilippo Syndrome Type B, aromatic L-amino acid decarboxylase deficiency, neurodegenerative proteinopathies, spinocerebellar ataxias, and glioblastoma. One of the programs Sarepta is picking up is for Pompe disease.

At the end of July, the U.S. Food and Drug Administration (FDA) placed Sarepta’s Phase I/IIa clinical trial for DMD Micro-Dystrophin Gene Therapy on clinical hold because of trace amounts of DNA fragments were found in the third-party supplied plasmids used in the trial. Although they are likely harmless, they were research-grade plasmids as opposed to “clinical-grade plasmids.” Although Sarepta has submitted an action plan to correct the decision and expects dosing to continue as originally planned by the end of the year, the hold has yet to be lifted.

Sarepta also released its second-quarter financial statement yesterday. It reported total revenues of $73.529 million for the quarter, up dramatically from $35.011 million in the same period in 2017. Operating losses, however, for the quarter were $103,427. The company holds about $950 million in cash, cash equivalents, and investments. R&D expenses for the quarter were $122.8 million.

“We are very pleased to report another strong quarter, marrying our vision to become one of the most meaningful precision genetic medicine companies globally with a strong focus on execution against both our near- and long-term range goals,” Ingram stated. “Our robust quarter sales for Exondys 51, increasing patients on therapy and strong adherence and compliance speaks to the benefits of Exondys 51 and to our ability as a fully integrated, commercial-stage genetic medicine company to support our therapies in the community and deliver on our commitments.”

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