RARE Daily

Sanofi Pulls the Plug on Regulus’ Candidate in Alport Syndrome

July 18, 2022

Sanofi notified Regulus Therapeutics that it was terminating the phase 2 clinical study of lademirsen (RG-012) for the treatment of the rare kidney disease Alport syndrome after it failed to meet Sanofi’s pre-defined futility criteria.

Sanofi also told the company that it is currently evaluating different opportunities with respect to RG-012. Regulus reported the development in a filing with the U.S. Securities and Exchange Commission.

Alport syndrome is a genetic condition that causes a progressive loss kidney function. The condition also causes hearing loss and eye abnormalities.

Lademirsen is a single stranded, chemically modified oligonucleotide that binds to and inhibits the function of miR-21. Initiated in September 2017, the HERA study is a global, randomized, placebo-controlled phase 2 study designed to assess the safety and tolerability of lademirsen, as well as the efficacy in reducing the decline in renal function in adult patients with Alport Syndrome.

Regulus was eligible for a $25 million milestone payment had Sanofi successfully completed the phase 2 study. It said under the amended license and collaboration agreement between the two companies, Regulus would still be eligible to receive a $25 million milestone payment should Sanofi successfully complete a phase 2 fibrosis proof of concept study.

Author: Rare Daily Staff

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