Arrowhead Pharmaceuticals is turning its financial tie-up with a promising but still experimental heart drug into 250 million dollars in immediate cash, a move that comes days after reporting phase 2 data showing the therapy successfully reduced levels of a protein linked to heart disease risk.
The data for the drug, olpasan, was reported by Amgen, which licensed the RNA therapy from Arrowhead. That deal gives Pasadena, Calif.-based Arrowhead royalties from Amgen’s sales of the drug if it reaches the market. This market is large as heart disease remains a leading cause of death, positioning oplasiran as a potential blockbuster.
Arrowhead is making good on the olpasiran promise by selling royalties to Royalty Pharma, a company that makes deals to acquire licensing rights for development-stage and commercialized drug assets. According to the financial terms announced Wednesday, the $250 million coming to Arrowhead is an upfront payment. Royalty Pharma could pay the biotech up to $160 million more depending on the drug achieving clinical, regulatory and commercial milestones.
Olpasiran is an RNA therapy that works through RNA interference (RNAi), an approach sometimes called gene silencing. The small interfering RNA in the drug stops the gene from producing a disease-causing protein. Olpasiran is designed to reduce the levels of lipoprotein (a), a type of “bad” form of cholesterol that increases the risk of cardiovascular problems, such as heart attack and stroke. The drug comes from Arrowhead’s technology platform called Targeted RNAi Molecule, or TRiM.
Amgen licenses the rights to olpasiran and a second RNAi drug in 2016, committing $56.5 million plus another $617 million related to milestones. The deal provided Arrowhead with low-double-digit sales royalties on olpassed. Amgen assumed responsibility for the drugs’ clinical development.
On Sunday, Amgen presented the results of phase 2 for olpassed during the American Heart Association Scientific Sessions Conference in Chicago. The double-blind, placebo-controlled trial included 281 adults with high levels of lipoprotein (a) and a history of atherosclerotic cardiovascular disease. Four doses of the subcutaneously injected drug were tested in this dose-finding study.
Amgen reported that more than 98% of participants who received the 75 mg dose (median dose) or higher achieved a reduction in lipoprotein (a) of 95% or more compared to placebo at week 36. The pharmaceutical company Thousand Oaks, Calif. The company said adverse reactions were similar in both arms of the study, with injection site reaction being the most common. The data was too published Sunday in the New England Journal of Medicine. Based on the encouraging Phase 2 results, Amgen plans to move to a Phase 3 cardiovascular outcomes study, which is expected to begin enrolling patients in December, the company said in investor presentation on Monday.
Although Arrowhead has given up its rights to royalties for Olpass, it can still make money from Amgen’s progress with the drug. Arrowhead is still eligible for up to $400 million in milestone payments from its original deal with Amgen — rights not included in the deal for Royalty Pharma.
“Olpasiran, which we developed and licensed to Amgen for further clinical development and commercialization, has the potential to benefit millions of patients worldwide by reducing the risk of cardiovascular disease,” said Arrowhead President and CEO Chris Anzalone in a prepared statement. “The early monetization of this potential royalty stream confirms the significant potential of olpasiran and allows us to continue to invest in our TRiM platform and our diverse and growing pipeline of RNAi therapeutic candidates.”
Arrowhead’s most advanced program is another cardiovascular drug candidate. ARO-APOC3 is designed to reduce levels of apolipoprotein C-III to treat hypertriglyceridemia, a disorder characterized by high blood levels of fats called triglycerides. The domestic pipeline includes programs for liver, lung and muscle diseases.
In addition to its partnership with Amgen, Arrowhead has placed assets in other pharmaceutical companies. A 2018 deal gave Johnson & Johnson rights to an Arrowhead RNAi drug for chronic hepatitis B infection in 2020. Arrowhead licenses Takeda Pharmaceutical rights to RNAi drug for rare liver disease. Last year, Horizon Therapeutics has acquired rights to Arrowhead’s experimental gout treatment.
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