Sarepta Therapeutics will shell out potentially more than $38 million for rights to up to three preclinical CNS-targeting gene therapy candidates from Lacerta Therapeutics, in a broadening of the buyer’s pipeline of rare neuromuscular disease treatments beyond Duchenne muscular dystrophy (DMD).

Through a License, Development and Option Agreement inked by the companies, Sarepta is licensing exclusive rights to a Lacerta treatment for Pompe disease, as well as options to license two additional Lacerta candidates.

While the indications of those candidates were undisclosed by Sarepta in an announcement and a regulatory filing yesterday, Lacerta says its pipeline includes additional gene therapies for Sanfilippo Syndrome Type B; Aromatic L-amino acid decarboxylase deficiency; Neurodegenerative Proteinopathies; Spinocerebellar Ataxias (SCA); and Glioblastoma.

Lacerta has identified the Sanfilippo Syndrome Type B and SCA programs, along with the Pompe disease program, as its three lead programs.   

For the Pompe disease treatment license, Sarepta disclosed in its Form 10-Q quarterly filing, Sarepta has agreed to pay Lacerta $8 million upfront; a $30 million equity investment in Lacerta; additional payments tied to achieving development and commercialization milestones; and a high single digit royalty payable upon commercialization.

If Sarepta opts to license the two undisclosed candidates, it has agreed to make additional payments tied to development and commercialization milestones, as well as tiered high single digit royalties upon their commercialization.

Lacerta will manage the majority of preclinical development while Sarepta will lead clinical development and commercialization, Sarepta said.

‘Moves Us Forward’

“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 medicine companies in the coming few years,” Sarepta president and CEO Doug Ingram said in a statement.

Through the licensing agreement, Sarepta will also gain access to Lacerta’s capsid screening and proprietary OneBac manufacturing platform and process for the licensed products.

The deal also increases the gene therapy share of Sarepta’s pipeline to 11 candidates. Most of Sarepta’s 20-program pipeline is focused on treating DMD, as is its sole marketed therapy, Exondys 51™ (eteplirsen), for DMD patients with a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. The FDA approved Exondys 51 in 2016 despite recommendations by two advisory committees against approving the treatment and over objections from some administrators.

Last month the FDA placed a clinical hold on Sarepta’s Phase I/IIa trial of its DMD gene therapy candidate AAVrh74.MHCK7.micro-Dystrophin—a month after the company trumpeted the study’s first positive results.

Lacerta is a developer of adeno-associated virus (AAV)-based gene therapies for central nervous system and lysosomal storage diseases. Founded on technologies licensed from the University of Florida (UF), Lacerta is a resident company of UF Innovate’s Sid Martin Biotech incubator.

“Our co-founders have dedicated their careers to the development of AAV gene therapy platforms for the treatment of multiple diseases,” added Joseph Reddy, Ph.D., Lacerta’s president and CEO. “We are pleased to begin our collaboration with Sarepta Therapeutics, a gene therapy leader, as it represents a significant step in advancing Lacerta’s treatments to the clinic.”

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