- Novartis will work with Alnylam Pharmaceuticals to explore whether the Cambridge, Massachusetts-based biotech company's RNA technology could be used to help restore function in failing livers.
- The partnership, which was announced Thursday and lasts three years, is aimed at identifying a potential treatment alternative to organ transplant in patients with end-stage liver disease, Novartis said. Initial research will be handled by Alnylam and, should a drug candidate be developed, would later transition to Novartis.
- The collaboration will focus on an unspecified drug target in the liver discovered by scientists at the Novartis Institutes for BioMedical Research. The partners hope to block that target with small, "interfering" RNA molecules — a technology Alnylam has spent nearly 20 years perfecting.
Novartis and Alnylam have a long history. The two companies first partnered in 2005, more than a decade before Alnylam had proven the Nobel Prize-winning science of RNA interference could be turned into safe and effective drugs.
But five years later Novartis parted ways with Alnylam, pulling back from RNAi amid a broader waning of large pharmaceutical company interest in the technology. The decision triggered layoffs at Alnylam and led to several lean years for the biotech.
Research progress and clinical trial success, however, resulted in Alnylam winning a pioneering approval of the first RNAi drug, Onpattro, for a rare hereditary disease in 2018. The company has since won Food and Drug Administration OKs for two more medicines, Givlaari and Oxlumo.
A third drug invented by Alnylam was cleared by the agency in December for treating high cholesterol. Called Leqvio, it's sold by Novartis after the Swiss pharma bought its developer The Medicines Company in 2019. Alnylam will receive royalties worth up to 20% of the drug's sales, although half of that revenue it will pay to Blackstone under a separate deal.
The research partnership struck by Novartis and Alnylam Thursday is focused far earlier in development than any of those arrangements and is potentially years away from even having a drug candidate ready for testing in humans. But it signals interest by Novartis in using RNAi in new ways.
Onpattro, Givlaari and Oxlumo all work by interfering with RNA signaling to block, directly or indirectly, production of toxic proteins associated with the rare diseases each treats. With their collaboration, Novartis and Alnylam aim to develop a medicine that helps cells regrow in damaged livers by inhibiting the target discovered by Novartis scientists.
"Building on a legacy of leadership in regenerative medicine, we have devised a restorative strategy that could potentially deliver a transformative benefit to patients with liver failure," said Jay Bradner, head of the Novartis Institutes for BioMedical Research, in the company's statement.
In an email, a NIBR spokesperson said company scientists "have been exploring this target for several years." The company envisions its approach could work to treat liver failure across "multiple indications."
Novartis is studying a drug called iscalimab in liver transplant patients and has also done research in NASH, a liver disease that can lead to cirrhosis. (GlaxoSmithKline recently inked a deal with Arrowhead Pharmaceuticals to explore whether RNAi drugs could be used to effectively treat NASH.)
For initial research, Alnylam will use tests Novartis developed that are specific to the unnamed drug target. Once a drug candidate is identified, Novartis would lead any clinical research that results.
Novartis did not disclose any financial details, nor did the company specify what options would be evaluated after the three-year research term.
Note: This story has been updated with comment from Novartis.