Novartis, which already has experience with RNA interference therapies, is now turning to this modality as a potential way to treat Parkinson’s disease. The pharmaceutical giant is licensing rights to an Arrowhead Pharmaceuticals RNAi therapy designed to inhibit production of alpha synuclein, a protein associated with Parkinson’s progression.
Cemdisiran, a small interfering RNA drug that Regeneron Pharmaceuticals licensed from Alnylam Pharmaceuticals, met the goals of its pivotal test in generalized myasthenia gravis. The rare neuromuscular disease has several new therapies, but analysts see cemdisiran as having advantages over one particular class of drugs.
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The structure of Atalanta Therapeutics’ RNA interference drugs enables them to distribute broadly and deeply into brain tissue. Lead programs for Huntington’s disease and a rare inherited form epilepsy are on track to enter the clinic this year.
Alnylam pioneered RNA interference drugs for rare disease. The biotech aims to bring RNAI to prevalent disorders and its alliance with Roche on the drug zilebesiran for hypertension better positions the company to achieve this goal.
Amgen posted positive Phase 2 data for olpasiran, further building the case for the drug's blockbuster potential in heart disease as it proceeds to a pivotal test. Arrowhead Pharmaceuticals, which originally developed olpasiran and licensed it to the big pharma company, is cashing in now by selling its royalty rights to the molecule.
The acquisition agreement comes exactly two years after the two companies began a wide-ranging alliance focused on developing RNA interference drug for metabolic disorders. The most advanced therapy from that alliance is on track to reach the clinic in 2022.
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Horizon Therapeutics is gaining global rights to an Arrowhead Pharmaceuticals drug for gout. The preclinical drug is the latest RNA interference program that Arrowhead has licensed to a larger pharma company.
Alnylam Pharmaceuticals has filed for FDA approval of vutrisiran, which could become the biotech's fourth drug that works by RNA interference. Meanwhile the company is responding to a federal subpoena seeking documents related to the company's marketing and promotion of its first RNAi drug, Onpattro.
Inclisiran, which The Medicines Co. is developing with Alnylam, prevents production of PCSK9, the same protein targeted by two drugs that were the subject of a major patent lawsuit ruling last week. The company plans regulatory filings starting later this year.
The firm announced that it closed a $22 million Series C2 round, following a $25 million Series C1 round that it had closed last June, led by Asian investors.
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