Alynlam, Dicerna Partner on RNAi Therapeutics for Liver Disease

Alnylam and Dicerna recently announced the development and commercialization of investigational RNAi therapeutics for the treatment of alpha-1 liver disease.

The pharmaceutical companies have also completed a cross-license of their respective intellectual property for Alnylam’s lumasiran and Dicerna’s nedosiran investigational programs for the treatment for primary hyperoxauria (PH). 

This completion will boost Alnylam’s and Dicerna’s ability to bring orphan product candidates to the healthcare market and puts the focus on patient-centered care, community first, and ensures freedom for both companies to operate their individual RNAi therapeutic programs. 

“We are excited to bring our two leading RNAi therapeutics companies together in our efforts to advance potentially transformative medicines for the treatment of two rare diseases with significant unmet medical need,” John Maraganore, PhD, chief executive officer of Alnylam, said in the announcement. Specifically, the new agreements allow for Alnylam and Dicerna to join forces in areas of common interest, namely alpha-1 liver disease and primary hyperoxaluria.” 

The partnership comes two years after Alnylam entered into a settlement agreement with Dicerna resolving trade secret misappropriation claims against Dicerna. Under the agreement, Dicerna paid $25 million in cash and stock to Alnylam.

The Alnylam and Dicerna partnership is important to grow and strengthen the pharmaceutical supply chain. Their most recent collaboration will boost treatments for patients with liver disease. Dicerna has the experience and resources to lead the development and commercialization, while Alnylam retains an ex-US commercialization option, the announcement stated.

RNAi therapeutics offer promising treatment for liver disease. The therapeutics are able to manipulate the expression levels of specific genes in liver pathology to effective treat liver disease, according to a recent study on the different RNAi therapeutics for the condition.

Under the development and commercialization agreement, Alnylam’s ATL-AAT02 and Dicerna’s DCR-A1AT – both investigational RNAi therapeutics in Phase 1/2 development – will be explored for the treatment of liver disease, the announcement stated. 

Dicerna will assume responsibility for both ALN-AAT02 and DCR-A1At at its cost and may choose one or more medicines for future clinical development. Alnylam may opt-in to commercialize the selected candidate in countries outside the US, where commercialized infrastructure was already in place. 

The announcement also noted that Dicerna would retain worldwide rights to commercialized candidates in exchange for milestones and royalties payable to Alnylam.

“These agreements between Alnylam and Dicerna represent biopharma collaboration at its best, unifying the strengths of two leaders in RNAi innovation to rally behind the common goal of delivering much-needed new therapies to patients with rare diseases,” said Douglas M. Fambrough, PhD, president and chief executive officer of Dicerna. 

“By joining our efforts in alpha-1 liver disease, we believe we can be more strongly assured of bringing forward the therapy with the greatest potential to benefit patients. At the same time, our agreement related to lumasiran and nedosiran clears a path for each company to offer a new and differentiated treatment to patients with PH.”

In a separate agreement, Alnulam and Dicerna also recently granted each other a non-exclusive cross-license to their individual intellectual property related to their PH treatment investigational programs to ensure each organization is able to develop and commercialize its respective product candidate. 

Alnylam has control over lumasiran, which targets glycolate oxidase, for the treatment of PH type 1. Meanwhile, Dicerna’s nedosiran targets lactate dehydrogenase A to treat PH types 1, 2, and 3. 

Lumasiran previously showed positive Phase 3 results in the ILLUMINATE-A study and most recently it is the focus of a rolling new drug application with the FDA. Nedosiran is currently being evaluated in the PHYOX clinical development programs in patients with PH.