ARO-DM1 / Arrowhead, Sarepta Therap - LARVOL DELTA

Selected Recent Events (Businesswire) - "$100 million milestone earned when Arrowhead reached the first of two pre-specified enrollment targets and subsequent authorization to dose escalate in a Phase 1/2 clinical study of ARO-DM1, an investigational RNA interference (RNAi) therapeutic for the treatment of type 1 myotonic dystrophy (DM1), the most common adult-onset muscular dystrophy."

Commercial • Muscular Atrophy

Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy (clinicaltrials.gov) - P1/2 | N=78 | Recruiting | Sponsor: Arrowhead Pharmaceuticals | Trial completion date: Sep 2026 ➔ Dec 2026 | Trial primary completion date: Sep 2026 ➔ Dec 2026

Trial completion date • Trial primary completion date • Genetic Disorders • Muscular Dystrophy • Myotonic Dystrophy

Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy (clinicaltrials.gov) - P1/2 | N=90 | Recruiting | Sponsor: Arrowhead Pharmaceuticals | N=36 ➔ 90 | Trial completion date: Jun 2025 ➔ Sep 2026 | Trial primary completion date: Jun 2025 ➔ Sep 2026

Enrollment change • Trial completion date • Trial primary completion date • Genetic Disorders • Muscular Dystrophy • Myotonic Dystrophy

Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy (clinicaltrials.gov) - P1/2 | N=36 | Recruiting | Sponsor: Arrowhead Pharmaceuticals | Phase classification: P1 ➔ P1/2

Phase classification • Genetic Disorders • Muscular Dystrophy • Myotonic Dystrophy

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

Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy (clinicaltrials.gov) - P1 | N=36 | Recruiting | Sponsor: Arrowhead Pharmaceuticals | Trial completion date: Oct 2026 ➔ Jun 2025 | Trial primary completion date: Oct 2025 ➔ Jun 2025

Trial completion date • Trial primary completion date • Genetic Disorders • Muscular Dystrophy • Myotonic Dystrophy

Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy (clinicaltrials.gov) - P1 | N=48 | Recruiting | Sponsor: Arrowhead Pharmaceuticals | Not yet recruiting ➔ Recruiting

Enrollment open • Genetic Disorders • Muscular Dystrophy • Myotonic Dystrophy

Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy (clinicaltrials.gov) - P1 | N=48 | Not yet recruiting | Sponsor: Arrowhead Pharmaceuticals | Trial completion date: Oct 2025 ➔ Oct 2026

Trial completion date • Genetic Disorders • Muscular Dystrophy • Myotonic Dystrophy

Silencing DMPK gene by ARO-DM1, an siRNA therapeutic, for Type 1 Myotonic Dystrophy (MDA 2024) - "ARO-DM1 was intensely characterized for clinical development, and S-ARO-DM1 was selected for preclinical pharmacological studies due to its sequence complementarity to the transgene in the TREDT960i/HSA-rtTA mouse disease model of DM1, which expresses a partial human DMPK transgene with 960 CUG repeats (CUG960) in the 3’ UTR in skeletal muscle under the control of doxycycline-inducible system. The transgene expression was moderately reduced. However, the mis-splicing abnormalities of mRNA caused by transgene expression were substantially corrected."

Cataract • Genetic Disorders • Muscular Dystrophy • Myotonic Dystrophy • Ophthalmology

Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy (clinicaltrials.gov) - P1 | N=48 | Not yet recruiting | Sponsor: Arrowhead Pharmaceuticals

Trial completion date • Trial initiation date • Trial primary completion date • Genetic Disorders • Muscular Dystrophy • Myotonic Dystrophy