Sarepta Targets Billion at Arrowhead to Expand Pipeline with RNA Therapeutics

Sarepta Targets Billion at Arrowhead to Expand Pipeline with RNA Therapeutics

Sarepta Targets Billion at Arrowhead to Expand Pipeline with RNA Therapeutics

Sarepta Therapeutics is investing heavily in the development of RNA therapeutics in partnership with Arrowhead Pharmaceuticals, committing 0 million initially to discover new therapies targeting rare genetic disorders affecting the muscles, central nervous system, and lungs.

The agreement includes a 5 million equity investment in Arrowhead, alongside an additional 0 million planned for disbursement over the next five years, as detailed in a press release from Tuesday. Assuming progress is made in a Phase I/II trial, Arrowhead could secure an additional 0 million in the upcoming year and has potential to receive up to billion in further milestone payments, plus ongoing royalties.

This collaboration had a notable impact on Arrowhead’s stock, which surged nearly 20%, reaching .42 on Tuesday morning.

Arrowhead anticipates that this funding will extend its financing runway to 2028, which will be crucial for advancing its development of plozasiran, aimed at treating familial chylomicronemia syndrome, with potential FDA approval as early as 2025. Additionally, Sarepta’s CEO will join Arrowhead’s board to help facilitate the transition from a clinical-focused organization to a commercial one.

On the flip side, Sarepta seeks to broaden its pipeline, which comprises gene therapies and different modalities targeting Duchenne muscular dystrophy and limb-girdle muscular dystrophies.

Among the four programs included in the agreement with Arrowhead are facioscapulohumeral muscular dystrophy (FSHD), myotonic dystrophy type 1 (DM1), idiopathic pulmonary fibrosis (IPF), and spinocerebellar ataxia type 2 (SCA2). Arrowhead’s RNA interference technology allows for the delivery of small interfering RNA to mitigate the impact of disease-related genes.

Sarepta currently markets four drugs, including Elevidys, a gene therapy for Duchenne muscular dystrophy that recently gained full approval for patients aged four and older with specific mutations in the DMD gene. Analysts previously forecasted swift uptake of Elevidys, which has a hefty cost of .2 million.

This collaboration marks Sarepta’s most significant commitment to enhancing its pipeline in recent years. Jefferies analyst Andrew Tsai noted that the market is expected to respond positively to the agreement, with Sarepta’s shares rising over 6% to 2.20 at Tuesday’s market open.

Sarepta will gain exclusive rights to at least seven siRNA programs for rare diseases that currently have few or no treatment options, which diversifies their portfolio beyond DMD and addresses the impending revenue challenge for Elevidys post-2030.

“This strategic move is astute,” Tsai commented, suggesting that it could potentially lead to significant financial returns in the future.

Sarepta is not alone in its substantial investments in siRNA technology. Earlier this year, Boehringer Ingelheim established a partnership with the Chinese firm Suzhou Ribo Life Science and its Swedish affiliate Ribocure Pharmaceuticals to create siRNA-based solutions targeting nonalcoholic or metabolic dysfunction-associated steatohepatitis, with the collaboration potentially exceeding billion in value.