Collaborations

12 Months Ended

Dec. 31, 2022

Organization, Consolidation and Presentation of Financial Statements [Abstract]

Collaborations

Collaborations

Sanofi

In February 2014, we and Sanofi entered into a second amended and restated collaboration and license agreement (the “Sanofi Agreement”) to discover, develop and commercialize microRNA therapeutics to focus on specific orphan disease and oncology targets. Under the terms of the Sanofi Agreement, Sanofi had opt-in rights to our clinical fibrosis program targeting miR-21 for the treatment of Alport syndrome (which rights were relinquished by Sanofi in November 2018), our preclinical program targeting miR-21 for oncology indications, and our preclinical program targeting miR-221/222 for HCC. We were responsible for developing each of these programs to proof-of-concept, at which time Sanofi had an exclusive option on each program. We were eligible to receive royalties on microRNA therapeutic products commercialized by Sanofi and would have had have the right to co-promote these products relating to our preclinical program targeting miR-221/222.

As of December 31, 2022, we were eligible to receive milestone payments related to the development and commercialization of miR-221/222 for HCC of up to $38.8 million for proof-of-concept option exercise fees (net of a $1.25 million creditable option fee), $25.0 million for clinical milestones and up to $130.0 million for regulatory and commercial milestones. In addition, we were eligible to receive royalties based on a percentage of net sales of any products from the miR-221/222 program which, in the case of sales in the United States, were in the middle of the 10% to 20% range, and, in the case of sales outside of the United States, were in the low end to the middle of the 10% to 20% range, depending upon the volume of sales.

On July 12, 2022, we received notification from Sanofi of its decision to terminate the Phase 2 clinical study of lademirsen (RG-012) for the treatment of Alport syndrome for failure to meet Sanofi’s pre-defined futility criteria. Sanofi also notified us that they were evaluating different opportunities with respect to RG-012. As of December 31, 2022, we remained eligible to receive the $25.0 million milestone in the event of successful completion of a Phase 2 fibrosis proof of concept study. As of December 31, 2022, the $25.0 million in development milestone payments (variable consideration) was fully constrained and, therefore, did not meet the criteria for revenue recognition.

On January 6, 2023, Sanofi elected to terminate the Sanofi Agreement. See Note 14.