- Danish drugmaker Novo Nordisk is expanding further in researching drugs for the heart, announcing Monday a deal to buy an experimental rare disease treatment from Dublin-based biotech company Prothena.
- Novo will pay Prothena $100 million in the near term for rights to a drug called PRX004, which Prothena has been developing for the progressive protein disorder ATTR amyloidosis. All told, the deal could be worth as much as $1.2 billion for Prothena, should clinical development progress and the drug win approval.
- Novo plans to focus on developing PRX004 for a type of ATTR amyloidosis that affects the heart. Prothena has completed a Phase 1 safety test of the drug in hereditary ATTR amyloidosis, but designed the drug to work for what's known as the "wild-type," or non-hereditary, form of the disease, too.
Novo is best known for the insulins and other diabetes medicines its scientists spent years developing and refining. Deals for drugs invented outside its laboratories are relatively rare, as are agreements focused on diseases other than the metabolic condition.
But Novo has shown an interest in expanding its research into blood disease and cardiovascular treatments, mounting a failed bid for the Belgian drugmaker Ablynx in 2018 and, more recently, acquiring Corvidia Therapeutics and its antibody drug for heart disease.
The deal for Prothena's PRX004, though a bit further afield, appears to fit into that pattern, giving Novo a mid-stage therapy for a disease that's receiving more and more attention from drugmakers.
ATTR amyloidosis is caused by the toxic build-up of a misfolded form of a protein called transthyretin. The disease is progressive and typically manifests in symptoms tied to damage to nerves or to the heart. PRX004, an antibody drug, is designed to clear our those deposits in people who have the disease due to mutations in a gene that are either inherited or acquired.
Preclinical studies showed PRX004 helped clear plaques formed through the protein buildup, and a Phase 1 study of 21 patients showed the drug to be safe and "well-tolerated" across several doses, according to Prothena.
Under the deal, Novo will acquire a subsidiary of Prothena working on amyloidosis research, gaining full rights to PRX004 in the process. The Danish pharma will pay $60 million upfront and an additional $40 million when a Phase 2 study of the drug begins, wrote Kennen Mackay, an analyst at RBC Capital Markets, in a research note after speaking with Prothena executives.
ATTR amyloidosis is the focus of several other drugmakers, too, including Alnylam Therapeutics, Ionis Pharmaceuticals, Pfizer and Intellia Therapeutics. Alnylam, Ionis and Pfizer all have approved medicines on the market, although Alnylam's and Ionis' are cleared for use only in patients with nerve damage.
Intellia, which is partnered with Regeneron, recently unveiled promising results for its treatment, which uses CRISPR gene editing to turn off production of the toxic protein. The treatment, called NTLA-2001, is designed for use in patients with the less-common hereditary form of ATTR amyloidosis.
For Prothena, the deal with Novo is its second in the past three weeks. In late June, the biotech agreed to license rights to an experimental Alzheimer's treatment to Bristol Myers Squibb in exchange for $80 million upfront and the chance to earn as much as $2 billion more in milestone payments.