Novo Nordisk puts $1.2B on the table for Prothena's ATTR drug, teeing up Alnylam challenge and cardiovascular expansion

Novo Nordisk has struck a $1.2 billion deal to buy Prothena’s ATTR amyloidosis program, which was stalled by the pandemic last year.

The deal will see Novo pay $100 million in upfront and near-term fees for worldwide rights to the phase 2-ready anti-amyloid immunotherapy PRX004.

Prothena moved PRX004 into phase 1 in 2018, only to terminate the study last year because of the pandemic after enrolling 21 subjects. The truncated trial generated evidence that PRX004 improves the neuropathy and cardiac systolic function of patients with the rare protein misfolding disorder, teeing Prothena up to set its sights on the initiation of a phase 2/3 study in the fourth quarter. 

Novo will now take control of further development. The Danish drugmaker plans to study PRX004 in ATTR cardiomyopathy, a form of the disease characterized by the accumulation of amyloid deposits in cardiac tissue. 

RELATED: Alnylam's next-gen ATTR amyloidosis drug hits goals in phase 3, teeing up approval filing

The therapeutic focus moves Novo into competition with Alnylam. Having won approval for Onpattro in hereditary ATTR amyloidosis, Alnylam is studying the RNAi drug in patients with cardiomyopathy. Phase 3 trial data are due in mid-2022. Alnylam is also studying vutrisiran as a treatment for ATTR amyloidosis with cardiomyopathy.

While Onpattro and vutrisiran suppress synthesis of TTR, thereby addressing the rare disease at its source, PRX004 is designed to bind to pathogenic TTR and clear amyloid that causes negative health outcomes.  

“With its innovative amyloid-depleting mechanism, PRX004 has the potential to offer a novel treatment option for ATTR cardiomyopathy—an often fatal disease with significant unmet medical need,” Marcus Schindler, Ph.D., chief scientific officer at Novo, said in a statement.

The deal furthers Novo’s effort to expand beyond diabetes and push into cardiovascular disease. This expansion led Novo to land an option to buy Staten Biotechnology for its anti-apoC3 antibody and acquire Corvidia Therapeutics for $725 million. 

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As for the benefits to Prothena, RBC Capital Markets analysts said the Novo deal will clear the way for the smaller biotech to focus on other areas of expertise that could bring value to shareholders. 

"We like the deal and see Novo Nordisks' expertise in cardiology both adding validation to the program and helping to reduce both development, regulatory, and commercial risk for PRX004," the analysts said in a Monday morning note. 

RBC said the market for ATTR amyloidosis is becoming increasingly competitive, and Alnylam isn't the only contender. Last month Intellia revealed first-in-human gene editing data on the company's candidate that exceeded expectations and generated plenty of buzz for what could become an important new area of medicine. 

ATTR amyloidosis is still a "highly lucrative rare disease market," but is better in Novo's hands, according to RBC. 

RELATED: Intellia hits a 'home run' with gene-editing results, setting up entire field for a grand slam

Prothena can now shift to the AL amyloidosis-drug birtamimab, which is currently in phase 3 testing, and two Alzheimer's disease therapies, PRX005 and PRX012. The Alzheimer's drugs could generate their own deal interest, given the renewed focus on the disease area spurred by the approval of Biogen's Aduhelm. 

Bristol Myers Squibb opted into a license to develop PRX005 last month and will pay Prothena $80 million. Prothena was named as one of the big winners from the Aduhelm drama that has brought Alzheimer's disease treatments back into the mainstream after years of setbacks and trial failures. 

Editor's Note: This article was amended at 9:30 a.m. ET on July 12, 2021, to include analyst commentary.