- Alexion, the rare disease drug division of AstraZeneca, will pay $30 million to Switzerland's Neurimmune for rights to an antibody drug developed by the biotech for treating a rare and fatal condition that leads to progressive heart failure.
- Through the deal, announced by the companies Friday, Alexion will gain access to Neurimmune's therapy for transthyretin amyloid cardiomyopathy. Neurimmune's drug, dubbed NI006, is designed to remove amyloid deposits in the heart and is currently in early clinical testing.
- Neurimmune could receive as much as $730 million should NI006 hit certain, unspecified development, regulatory and commercial milestones. The biotech will finish out the ongoing Phase 1b trial, after which Alexion will be responsible for further clinical development.
Transthyretin amyloidosis, a disease caused by the build-up of misfolded protein in the heart, nerves and other organs, has become a common target for rare disease drug developers. Three medicines from Alnylam Pharmaceuticals, Ionis Pharmaceuticals and Pfizer are already approved to treat forms of the disease, while other potential treatments are being advanced by Intellia Therapeutics and Prothena. (Another experimental drug from Bridgebio recently failed a Phase 3 trial, though testing remains ongoing.)
AstraZeneca has already shown interest in developing drugs for the condition, reaching a deal last month for a successor treatment from Ionis that's designed to treat the disease in both the heart and in the nerves. Alexion, which AstraZeneca bought in December 2020, also owns rights in Japan to Bridgebio's treatment and had partnered on a drug for a different type of amyloidosis known as light chain, too.
The latest deal with Neurimmune will cost AstraZeneca less upfront than its licensing agreement with Ionis, but adds a treatment that works in a different way and is aimed specifically at transthyretin amyloidosis of the heart.
"There is a significant unmet medical need for patients with various types and levels of severity of amyloidosis that may require multiple mechanisms of action to address those needs," AstraZeneca said in a statement on the Neurimmune deal.
ATTR with cardiomyopathy is thought to be more prevalent than the neuropathic form of the disease, and therefore is viewed as a larger market by drugmakers. ATTR can have genetic causes or occur naturally; both Alnylam's and Ionis' drugs are approved for the hereditary form.
Neurimmune's drug is meant to clear deposits of TTR protein that are misfolded for genetic reasons as well as natural causes like aging.
Based in Zurich, Neurimmune is probably best known for its invention of the antibody aducanumab, which it licensed to Biogen and was recently approved by the Food and Drug Administration for the treatment of Alzheimer's disease. The biotech is also co-developing an ALS drug with Eli Lilly and is working with Biogen on another Alzheimer's treatment.