SoCal's Arrow Pharm Just Snagged a $673.5 Million Heart Deal With Amgen

Published: Sep 30, 2016

SoCal's Arrowhead Pharma Snags a $673.5 Million Heart Deal With Amgen September 29, 2016
By Alex Keown, Breaking News Staff

THOUSAND OAKS, Calif. – Shares of Arrowhead Pharmaceuticals are up more than 18 percent to $8 per share in pre-market trading after Amgen bought a stake worth up to $675 million in the company to develop and commercialize RNA interference (RNAi) therapies for cardiovascular disease.

Under terms of the two deals, Pasadena, Calif.-based Arrowhead will receive $35 million in up-front payments and will then be eligible for up to $617 million in milestone payments. The company will also get an additional $21.5 million from Amgen in the form of an Amgen equity investment. Arrowhead will also be able to receive some royalties from sales.

Arrowhead is developing its RNAi ARC-LPA program. The engineered molecules are designed to reduce elevated lipoprotein(a), which is a genetically validated, independent risk factor for atherosclerotic cardiovascular disease. That is the first target for the Amgen and Arrowhead agreement. Under the second agreement, Amgen will receive an option for a worldwide and exclusive license for a RNAi therapy for an undisclosed genetically validated cardiovascular target. Under terms of both agreements, Amgen will be wholly responsible for clinical development and commercialization.

RNA interference is a natural mechanism of gene silencing. Much of the interest in RNAi is based on the fact that the RNAi mechanism operates upstream of protein production by silencing the mRNA that codes for such proteins, thereby preventing the disease-causing proteins from being made in the first place.

ARC-LPA is designed to reduce production of apolipoprotein A, a key component of lipoprotein(a), which has been genetically linked with increased risk of cardiovascular diseases, independent of cholesterol and LDL levels. ARC-LPA is Arrowhead's first drug candidate to use a subcutaneously administered delivery construct.

Christopher Anzalone, president and chief executive officer at Arrowhead, said the company’s capabilities have become “increasingly validated” as more headway is made in the field of RNA-based therapies.

"We have made great advances to our proprietary subcutaneous RNAi delivery vehicle and in RNAi trigger modification and stabilization that enable rapid development of new RNAi therapeutics across multiple disease areas," Anzalone said in a statement.

Sean Harper, Amgen’s vice president of research and development, said the collaboration with Arrowhead allows Amgen to build upon its commitment to developing various therapeutic approaches to cardiovascular disease. Amgen already has a number of heart disease-related drugs in its pipeline, including its anti-cholesterol drug Repatha and heart failure drug Crolanor.

"Arrowhead's expertise in RNAi makes them a valuable partner as we translate genetic discoveries into potential therapies that can improve health outcomes for patients," Harper said in a statement.

Both companies anticipate the finalization of the deal by the end of the fourth quarter of 2016.

Several companies have focused their efforts on developing RNAi therapies. In addition to Arrowhead, leading RNAi companies include Boston-based Alnylam Pharmaceuticals and California-based Ionis Pharmaceuticals . Alnylam has two drugs in Phase III trials—patisiran and revusiran, for transthyretin amyloidosis. Ionis most recently completed a successful Phase III trial for nusinersen, a treatment for spinal muscular atrophy. Additionally Ionis has another RNA-targeting drug in late-stage development, volanesorsen for patients with either familial chylomicronemia syndrome or familial partial lipodystrophy.

Another company investing in RNAi therapies is Moderna Therapeutics, which has 11 drugs in its pipeline. In July, Moderna and Vertex inked a deal worth more than $315 million to develop treatments for cystic fibrosis using mRNA technology.

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