SWITZERLAND – Novartis and Alnylam have announced a collaboration to develop a targeted therapy to promote the regrowth of functional liver cells using Alnylam’s siRNA technology.

The study will center on a target discovered at the Novartis Institutes for BioMedical Research, with the ultimate goal of providing an alternative to transplantation for patients with liver failure.

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,” remarked Jay Bradner, president of the Novartis Institutes for BioMedical Research.

The executive suggested that Alnylam’s siRNA platform “is optimally suited to translate this concept to clinical investigation.”

Novartis and Alnylam have a long history of collaboration. The two companies first collaborated in 2005, more than a decade before Alnylam demonstrated that Nobel Prize-winning RNA interference science could be translated into safe and effective drugs.

However, Novartis parted ways with Alnylam five years later, withdrawing from RNAi amid a general decline in large pharmaceutical company interest in the technology. The decision resulted in layoffs at Alnylam and a string of difficult years for the biotech.

However, advancements in research and clinical trial success resulted in Alnylam receiving the first RNAi drug, Onpattro, for a rare hereditary disease in 2018. Since then, the company has received FDA approval for two more medicines, Givlaari and Oxlumo.

In December, the FDA approved Alnylam’s third drug for the treatment of high cholesterol. Novartis sells it as Leqvio after purchasing its developer, The Medicines Company, in 2019.

Alnylam will receive royalties of up to 20% of the drug’s sales, with half of that revenue going to Blackstone under a separate agreement.

Novartis and Alnylam’s research collaboration recently announced, is much earlier in development than any of those agreements, and it could be years before a drug candidate is ready for human testing. However, it indicates Novartis’s interest in exploring new applications for RNAi.

Success RNAi drugs

Onpattro, Givlaari, and Oxlumo all work by interfering with RNA signaling to prevent the production of toxic proteins associated with the rare diseases they treat, either directly or indirectly.

Novartis and Alnylam hope to create a medicine that helps cells regrow in damaged livers by inhibiting a target discovered by Novartis scientists.

Novartis is researching a drug called Iscalimab in liver transplant patients, as well as NASH, a liver disease that can lead to cirrhosis.

GlaxoSmithKline recently signed a collaboration agreement with Arrowhead Pharmaceuticals to investigate whether RNAi drugs could be used to effectively treat NASH.

Alnylam will conduct preliminary research using Novartis-developed tests that are specific to the unnamed drug target. Novartis would lead any clinical research that resulted from the identification of a drug candidate.

Novartis did not provide any financial information, nor did it specify what options would be considered after the three-year research period.

The companies stated that during the three-year research collaboration, Alnylam will develop and test potential siRNAs using Novartis’ target-specific assays.

The Swiss drugmaker will conduct additional development and clinical research once a lead candidate has been identified. The deal’s financial terms were not disclosed.

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