SRP-1005 / Sarepta Therapeutics, Arrowhead Pharmaceuticals - LARVOL DELTA

Sarepta Therapeutics...announced that Medsafe, the New Zealand Medicines and Medical Devices Safety Authority, has granted approval for its clinical trial application (CTA) for Study SRP-1005-101, also known as INSIGHTT. (Sarepta Therap Press Release) - "Sarepta expects to initiate this first-in-human clinical trial of SRP-1005 (formerly ARO-HTT) in the second quarter of 2026....INSIGHTT is a Phase 1, multi-center, dose escalation study that will evaluate the safety and tolerability of subcutaneous dosing of SRP-1005 in approximately 24 participants. SRP-1005 leverages an advanced TfR1 (transferrin receptor protein 1) approach that uses monovalent fragment antigen binding (fAb) designed for efficient delivery to the central nervous system."

First-in-human • New P1 trial • Huntington's Disease

Sarepta Therapeutics…announced the submission of its clinical trial application (CTA) for Study SRP-1005-101, also known as INSIGHTT, to Medsafe, the New Zealand Medicines and Medical Devices Safety Authority. (Businesswire) - "Pending approval, Sarepta expects to initiate this first-in-human clinical trial of SRP-1005 (formerly ARO-HTT) in the second quarter of 2026....INSIGHTT is a Phase 1, multi-center, dose escalation study that will evaluate the safety and tolerability of subcutaneous dosing of SRP-1005 in approximately 24 participants."

First-in-human • New P1 trial • Huntington's Disease

Arrowhead Pharmaceuticals Announces Closing of Global License and Collaboration Agreement with Sarepta Therapeutics (Businesswire) - "Arrowhead Pharmaceuticals, Inc...today announced that the global licensing and collaboration agreement with Sarepta Therapeutics (NASDAQ: SRPT) announced on November 26, 2024, has now closed. Closing of the transaction was subject to the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions...Upon closing, Arrowhead receives a $500 million upfront payment and $325 million through the purchase by Sarepta of Arrowhead common stock priced at $27.25 per share. Arrowhead will also receive $250 million to be paid in annual installments of $50 million over 5 years...ARO-HTT for patients Huntington’s disease expected to be CTA-ready in 2025; ARO-ATXN1 for patients with spinocerebellar ataxia 1 (SCA1) expected to be CTA-ready in 2026; ARO-ATXN3 for patients with spinocerebellar ataxia 3 (SCA3) expected to be CTA-ready in 2026."

IND • Licensing / partnership • Ataxia • Huntington's Disease • Idiopathic Pulmonary Fibrosis • Muscular Dystrophy • Myotonic Dystrophy

Arrowhead Pharmaceuticals Announces Global License and Collaboration Agreement with Sarepta Therapeutics for Multiple Clinical and Preclinical Programs (Businesswire) - "Arrowhead Pharmaceuticals, Inc...today announced a global licensing and collaboration agreement with Sarepta Therapeutics...Upon closing, Arrowhead will receive $825 million, consisting of $500 million cash and $325 million as an equity investment priced at a 35% premium. Arrowhead also receives $250 million to be paid in equal installments over five years and is eligible to receive an additional $300 million in near-term payments, which Arrowhead is on track to achieve during the next 12 months. Additionally, Arrowhead is eligible to receive royalties on commercial sales and up to approximately $10 billion in future potential milestone payments. The agreement covers multiple clinical and preclinical programs in rare, genetic diseases of the muscle, central nervous system (CNS), and the lungs...Summary of Programs under License and Collaboration Agreement...ARO-HTT for patients Huntington’s disease expected to be CTA-ready in 2025."

Licensing / partnership • Huntington's Disease