Arrowhead Pharmaceuticals

Arrowhead Pharmaceuticals advances RNAi therapeutics, focusing on TRiM platform and late-stage cardio-metabolic franchise. Key developments include NDA submission for plozasiran to treat FCS and promising Phase 2 data for mixed hyperlipidemia and sHTG. Analysts predict potential approval in 2025, with price targets ranging from $23 to $80 per share.

Arrowhead Pharmaceuticals seeks NZ clearance for Phase I/IIa trial of ARO-ALK7, an RNAi therapy targeting obesity by suppressing ACVR1C gene in fat cells. The trial aims to recruit 90 obese adults to assess tolerability, safety, and pharmacodynamics. Part I will evaluate ARO-ALK7 as a standalone therapy, while Part II will test it with tirzepatide. Arrowhead's TRiM platform supports ARO-ALK7 development, and the company also has ARO-INHBE for obesity treatment.

Sarepta Therapeutics, known for its focus on Duchenne muscular dystrophy, made a $500 million upfront deal with Arrowhead Pharmaceuticals, investing $325 million and up to $10 billion in milestones. The deal aims to diversify Sarepta's pipeline with RNA drugs and expand its portfolio into new disease areas. Despite Elevidys failing a confirmatory trial, Sarepta secured full FDA approval and a label expansion, generating $181 million in Q3. The deal reflects Sarepta's ability to secure regulatory success and diversify its portfolio, potentially offering new options for patients with rare diseases.

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Increased R&D in myotonic dystrophy drives market growth, attracting funding and partnerships. DelveInsight's 'Myotonic Dystrophy Pipeline Insight 2024' reports on 20+ companies developing 22+ therapies, including AOC 1001, Tideglusib, and PGN EDODM1. Key events include FDA designations for ATX-01 and DYNE-101, and Fast Track designation for PGN-EDODM1.

Sarepta Therapeutics announced a deal with Arrowhead Pharmaceuticals, acquiring four clinical-stage treatments and three preclinical assets for $500 million upfront and $325 million in shares. Arrowhead will complete Phase 1/2 trials before handing over development to Sarepta, which also gains rights to nominate six additional drug targets. The deal is seen as complementary to Sarepta's genetic medicine focus and aims to address concerns over its thin pipeline.

Sarepta Therapeutics and Arrowhead Pharmaceuticals collaborate on RNAi treatments for rare genetic diseases affecting muscles, CNS, and lungs. The agreement includes exclusive rights to Arrowhead’s clinical-stage programs and leverages Arrowhead’s TRiM platform. Sarepta will invest $500 million upfront and $325 million in Arrowhead stock, with additional milestone payments and royalties. Doug Ingram, Sarepta’s CEO, will join Arrowhead’s board. The partnership also includes a discovery collaboration for additional targets.

Arrowhead Pharmaceuticals and Sarepta Therapeutics form a partnership to develop treatments for rare genetic diseases, with Arrowhead receiving $825m, potential future milestone payments up to $10bn, and royalties on commercial sales. The collaboration targets diseases affecting muscles, lungs, and the central nervous system.

Arrowhead Pharmaceuticals reported FY2024 results, submitting its first NDA for plozasiran to the FDA, with a potential 2025 launch. Key financial moves include a $825 million licensing agreement with Sarepta and a $500 million strategic financing with Sixth Street, extending the cash runway to 2028. The company initiated pivotal Phase 3 studies for plozasiran and expanded its therapeutic pipeline, aiming for long-term growth and financial sustainability.