Arrowhead Pharmaceuticals Closes Global License and Collaboration with Sarepta Therapeutics
Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced the successful closing of its global licensing and collaboration agreement with Sarepta Therapeutics (NASDAQ: SRPT). The agreement, initially announced on November 26, 2024, has now closed after meeting all necessary conditions, including the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, along with other customary closing conditions.
Summary of Financial Terms
Upon the transaction’s closing, Arrowhead Pharmaceuticals will receive a substantial upfront payment of $500 million from Sarepta. In addition, Sarepta has purchased Arrowhead common stock at $27.25 per share, totaling $325 million. Over the next five years, Arrowhead will also receive $250 million in annual installments of $50 million each.
Moreover, Arrowhead has the potential to earn $300 million in near-term payments associated with the continued enrollment of certain cohorts in the Phase 1/2 study of ARO-DM1, a promising treatment currently under development. These milestone payments are expected to be achieved within the next 12 months.
The agreement also allows Arrowhead to receive development milestone payments ranging between $110 million and $410 million per program, as well as sales milestone payments ranging from $500 million to $700 million per program. Additionally, Arrowhead will receive tiered royalties on commercial sales of each program, potentially up to the low double digits.
Programs Under License and Collaboration
This collaboration covers several of Arrowhead’s leading gene-silencing programs, each targeting significant unmet medical needs.
Clinical-Stage Programs:
ARO-DUX4 – Designed to target the DUX4 gene, which encodes the DUX4 protein linked to facioscapulohumeral muscular dystrophy type 1 (FSHD). The program is currently enrolling patients in a Phase 1/2 clinical study.
ARO-DM1 – Aiming to reduce the expression of the dystrophia myotonica protein kinase (DMPK) gene, which causes type 1 myotonic dystrophy (DM1). This program is also in a Phase 1/2 clinical study.
ARO-MMP7 – Designed to target matrix metalloproteinase 7 (MMP7), potentially providing a treatment for idiopathic pulmonary fibrosis. This program is in a Phase 1/2 clinical study.
ARO-ATXN2 – Aims to silence the ATXN2 gene, which produces a toxic protein associated with spinocerebellar ataxia type 2 (SCA2). This program is currently in Phase 1/2 and open for enrollment.
Preclinical-Stage Programs:
ARO-HTT – Targeting Huntington’s disease, expected to be ready for clinical trials in 2025.
ARO-ATXN1 – Aiming at spinocerebellar ataxia type 1 (SCA1), with clinical trials expected to begin in 2026.
ARO-ATXN3 – Targeting spinocerebellar ataxia type 3 (SCA3), with clinical trials expected to start in 2026.
Discovery Stage:
Under the agreement, Sarepta has the option to propose up to six new CNS or muscle targets for which Arrowhead will conduct discovery and preclinical development. Sarepta will receive exclusive licenses for these new programs and will be responsible for their clinical development and commercialization.
Drug Manufacturing
Arrowhead will also handle the manufacturing of the clinical drug supply for all programs resulting from the collaboration, as well as commercial drug product for the four programs currently in clinical trials.
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals is a leader in the development of medicines aimed at treating intractable diseases by silencing the genes responsible for these conditions. The company leverages a broad portfolio of RNA chemistries and efficient delivery methods to develop innovative therapies that induce rapid, deep, and durable gene silencing through RNA interference (RNAi).
RNA interference is a natural process within living cells that inhibits the expression of specific genes, preventing the production of harmful proteins. By utilizing this mechanism, Arrowhead’s RNAi-based therapeutics offer a promising way to treat diseases at the genetic level, addressing the root causes of many chronic and genetic conditions.
The collaboration with Sarepta Therapeutics represents a significant step forward for Arrowhead Pharmaceuticals, further expanding its reach in the gene therapy space and strengthening its pipeline of innovative, RNAi-based treatments.
Through this partnership, Arrowhead is poised to continue advancing its groundbreaking therapies, addressing high-need diseases with the potential to transform the treatment landscape for patients worldwide.
