Royalty Pharma has secured a stake in another transthyretin-mediated (ATTR) amyloidosis drug, this time paying $125 million upfront to Neurimmune for a cut of the sales of its AstraZeneca-partnered med.
AstraZeneca licensed the candidate, called cliramitug or NI006, from Neurimmune in 2022. That deal gave the Swiss biotech $30 million upfront, with the potential for up to $730 million in development, regulatory and commercial milestones, as well as “low-to-mid teen royalties on net sales” should cliramitug make it to market.
Royalty has its eyes on those payments. The firm, the world’s largest buyer of biopharma royalties, has agreed to pay $125 million upfront, followed by another $125 million in the first quarter of next year, in exchange for royalties of 3% to 4% on worldwide sales of cliramitug. The deal also includes up to $175 million tied to clinical and regulatory milestones, meaning Neurimmune could eventually receive $425 million in total.
Since Neurimmune’s 2022 deal with AstraZeneca, the Switzerland-based biotech has read out data from a phase 1 study of cliramitug in transthyretin amyloid cardiomyopathy (ATTR-CM) that it claimed showed “substantial” reductions in cardiac amyloid deposition at higher doses. AstraZeneca’s Alexion rare disease unit has also taken the therapy into an ongoing phase 3 trial, which is due to read out in 2028.
Cliramitug targets misfolded transthyretin, the protein at the heart of ATTR. Changes to the structure of the protein impair its ability to bind to other molecules, leading to deposits in the heart or the nerves.
The resulting cardiomyopathy and polyneuropathy, known respectively as ATTR-CM and transthyretin amyloid polyneuropathy (ATTR-PN), are currently treated with the likes of Pfizer’s Vyndaqel and Vyndamax, Alnylam’s Onpattro and Amvuttra, and AstraZeneca’s Wainua.
Wainua ran into trouble earlier this month when the Ionis-partnered antisense oligonucleotide (ASO) inhibitor, approved to treat the polyneuropathy of ATTR-PN, failed a phase 3 study for ATTR-CM. The surprise result raised doubts about AstraZeneca’s ATTR strategy, although Jefferies analysts noted at the time that the pharma still has a shot with cliramitug.
“The depleter is designed to directly address amyloid burden, offering potential to reverse disease biology rather than delay progression,” the analysts noted. “Early-stage burden has shown reductions in amyloid burden and cardiac biomarkers, although [cliramitug’s] clinical value will depend on demonstrating meaningful cardiovascular outcome benefits in the ongoing phase 3 trial.”
Royalty Pharma clearly believes in the drug’s potential. The company entered the ATTR space eight months ago, when it paid $310 million upfront for a 1% royalty on Amvuttra sales over the next nine years.
The ATTR-CM market grew over 40% last year to pass the $7 billion sales mark, the company noted.
Royalty Pharma CEO Pablo Legorreta pointed to cliramitug’s novel mechanism, encouraging clinical results and commercial potential in a market where treatment gaps remain.
“Cliramitug is our second recent investment in this indication and has the potential to transform the ATTR-CM disease course,” Legorreta said. “This therapy further bolsters our development-stage pipeline, and we believe it will become a valuable contributor to our portfolio over the long term.”
Neurimmune CEO Roger Nitsch said the biotech would use the proceeds to advance its internal pipeline, led by a phase 2 SOD1-targeting therapy for amyotrophic lateral sclerosis, while retaining most of its royalty and milestone interests in cliramitug.