Dicerna Pharmaceuticals’ research on medicines intended to stop a gene from producing a disease-causing protein has caught the eye of Roche, which is paying the company $200 million up front for global rights to its early-stage hepatitis B virus infection drug.
According to deal terms announced Thursday, Dicerna (NASDAQ: DRNA) could earn up to $1.47 billion in additional payments if the hepatitis B treatment, DCR-HBVS, achieves milestones. The Cambridge, MA-based drug developer would also receive royalties from sales if the medicine is approved and reaches the market.
The deal marks another vote of confidence in Dicerna and its technology. A year ago Eli Lilly (NYSE: LLY) committed $200 million to Dicerna in a partnership focused on developing drugs for pain, cardiovascular and metabolic diseases, and neurodegenerative disorders.
Dicerna is one of a number of companies developing drugs that employ an approach called RNA interference (RNAi). These drugs “silence” a gene before it can produce a disease-causing protein. Hepatitis B infection affects an estimated 257 million people worldwide, according to the World Health Organization. Dicerna says it aims to improve upon current hepatitis B therapies, which suppress the virus, but do not eliminate it. The company says DCR-HBVS uses RNAi to selectively knock down specific genes that play a role in creating hepatitis B messenger RNA and allow the virus to enter liver cells.
Dicerna is currently testing DCR-HBVS in a Phase 1 clinical trial. In mouse studies, Dicerna says the drug cleared nearly 100 percent of circulating hepatitis B surface antigen, a protein on the surface of the virus. Measures of that protein are used to assess whether a person with a hepatitis B infection can transmit the disease to others. Dicerna says its drug could offer a “functional cure,” in that it would keep the virus below detectable levels.
RNAi drugs are still relatively new. The Alynlam Pharmaceuticals (NASDAQ: ALNY) drug patisiran (Onpattro), approved last year, was the first RNAi medicine to receive an FDA nod. The Alnylam drug treats the nerve damage experienced by people who have the rare disease hereditary transthyretin amyloidosis.
Dicerna’s other RNAi drug partnerships include deals with Alexion Pharmaceuticals (NASDAQ: ALXN) and Boehringer Ingelheim. According to the agreement with Roche, Dicerna keeps an option to join in financing development of DCR-HBVS worldwide. Exercising that option would enhance Dicerna’s royalties and entitle the company to work with Roche to co-promote the hepatitis B drug and other products in the US.
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