- Sarepta Therapeutics reached a licensing deal with Lacerta Therapeutics for three gene therapy programs, expanding its pipeline to include 11 such programs, the company announced Wednesday afternoon.
- Sarepta gains the exclusive rights to Lacerta's candidate for Pompe disease along with options for two others, as well as access to capsid screening and a manufacturing platform. Lacerta is a gene therapy company founded as a spin-out by a handful of University of Florida researchers. Its treatments target the central nervous system.
- The Cambridge, Massachusetts-based biopharma will pay $30 million upfront along with development and sales-based milestones and single-digit royalties on net sales. For the three treatments in the agreement, Lacerta will lead pre-clinical development, while Sarepta will manage clinical development and commercialization.
The deal will help Sarepta flesh out its gene therapy goals, manifested in lofty ambitions by its leaders. On the earnings call, CEO Doug Ingram said the deal is a step forward in becoming "one of the most meaningful global genetic medicine companies in the coming few years."
This year, it's been doing that via deal-making. In May, Sarepta inked an agreement with Myonexus Therapeutics, paying $60 million upfront for five gene therapy programs in limb-girdle muscular dystrophy.
Ingram commented further on the company's expanding research portfolio in response to an analyst question on the call.
"We're going to continue to build on what we know, as we move out in concentric circles of focus," he said. "Moving to CNS is a very logical next step. We're not limiting ourselves in how far that takes us from a therapeutic area perspective."
Sarepta will face steep competition going into Pompe disease with its preclinical gene therapy program.
Pompe disease is an inherited lysosomal storage disorder that progressively weakens patients' muscles, robbing them over time of their ability to walk, hold objects or even complete critical functions such as swallowing.
Additionally, analysts also expect pre-clinical data from Spark Therapeutics' candidate this fall. Audentes Therapeutics is planning to file its Investigational New Drug application in the first half of 2019.
Joseph Schwartz, an equity analyst for Leerink, noted the Lacerta deal "offers another avenue for growth in the long run." Sarepta may need that new growth area, as progress in its micro-dystrophin gene therapy program has slowed under a July 25 clinical hold by the Food and Drug Administration.
While a hold is not unusual in the space — Solid Biosciences got through its own hold on DMD testing earlier this year — it has nonetheless set back research progress. Ingram said he expects to send a full reply to the FDA before the end of August.
Meanwhile, the company has leaned on Exondys 51 (eteplirsen) as its sole commercialized product. That gene therapy for Duchenne muscular dystrophy has enjoyed sizable sales growth since the FDA's controversial approval in September 2016 despite a lack of clear-cut efficacy data.
Exondys 51 posted $73.5 million in sales from April through June, a 110% increase from last year's second quarter. Analysts believe it is right on track to hit the company's expectation of $295 million to $305 million for yearly sales. Company officials said on the call they continue to look into an international roll-out, especially in European markets.
Investors and analysts seemed satisfied with the company's overall progress, as share prices shot up roughly 8% Thursday morning.