Sanofi licenses a preclinical genetic disease drug from an unknown biotech spun out of the NIH – Endpoints News

In a new deal Tues­day morn­ing, Sanofi is jump­ing in­to the an­ti­body-RNA con­ju­gate (ARC) space and el­e­vat­ing a lit­tle-known pri­vate biotech in­to the spot­light.
Sanofi is li­cens­ing a pre­clin­i­cal ARC can­di­date from Gaithers­burg, MD-based miRecule that’s de­signed to treat a ge­net­ic mus­cle dis­ease known as fa­cioscapu­lo­humer­al mus­cu­lar dy­s­tro­phy, or FSHD. Sanofi is get­ting ex­clu­sive world­wide rights for the pro­gram, known as MC-DX4, while miRecule gets an up­front and near-term mile­stones of about $30 mil­lion.
Ad­di­tion­al biobucks could bring the deal’s to­tal val­ue north of $400 mil­lion.
For Sanofi, the to­tal dol­lar fig­ure rep­re­sents a rel­a­tive­ly mod­est bet on a tech­nol­o­gy that’s sim­i­lar to an­ti­body-drug con­ju­gates but not quite the same. Rather than at­tach an an­ti­body to a drug, the FSHD pro­gram will be paired with the Big Phar­ma’s “NANOBODY” tech to cre­ate a sin­gle com­pound.
miRecule, mean­while, gets its day in the lime­light with its first li­cens­ing deal. The com­pa­ny, run by NIH vet An­tho­ny Saleh, was spun out of the agency in­to an in­cu­ba­tor in 2017 run by Bio­Health In­no­va­tion. Pri­or to Tues­day, miRecule had post­ed on­ly two press re­leas­es on its web­site: one de­tail­ing a $5.7 mil­lion seed round in May 2021, and an­oth­er re­veal­ing an FSHD foun­da­tion had do­nat­ed $1 mil­lion last week to ad­vance the pro­gram.
MC-DX4 is what miRecule de­scribes as an an­ti-DUX4 RNA ther­a­py, and the com­pa­ny not­ed in 2021 that this took the form of an an­ti­sense oligonu­cleotide. miRecule es­ti­mates there are about 40,000 pa­tients liv­ing with FSHD in the US.