Nothing to do with NEU's shareprice; rather, another example of...

Nothing to do with NEU's shareprice; rather, another example of a rare disease licensing deal....

Leading rare disease pharma, Sanofi, has entered into an exclusive ex-US licensing agreement with Fulcrum Therapeutics for losmapimod, an asset for which Phase 3 results are due in Q4 this year. Fulcrum will retain full US rights and the parties will share future global development costs 50:50.

The Fulcrum CEO said that the agreement would "leverage Sanofi’s exceptional global commercial capabilities and established infrastructure in key markets around the world" while allowing Fulcrum to concentrate on the commercialization of losmapinod in the US.

Sanofi paid an upfront of US$80m. There is a further US$975m in milestones and tiered royalties commencing in the low-teens in play. Losmapimod, which Fulcrum in-licensed from GSK, is an oral small molecule drug being developed for the treatment of the rare, muscle-wasting disorder, facioscapulohumeral muscular dystrophy (FSHD).

Losmapimod could become the first approved therapy for FSHD, a disease with an estimated prevalence of 1 in 8,000- 15,000. Roche also has a drug in development for FHSD, which has just completed Phase 2 enrolment, and Avidity and Arrowhead Pharmaceuticals currently have candidates in Phase 1/2 studies.

Skyclarys has been mentioned as a guide to likely pricing if the drug successfully makes it to market, although the patient population in FSHD is larger than Friedreich’s ataxia.

Success for losmapinod in Phase 3 is not a sure thing.  The drug missed the primary endpoint in a Phase 2b trial in FSHD in 2021, although the company decided to forge ahead on the basis of potential improvements seen in prespecified structural and functional outcomes and patient-reported global impression of change compared with placebo.

Losmapimod also previously failed a Phase 3 trial in acute coronary syndrome and a mid-stage study in chronic obstructive pulmonary disease.

Fulcrum Therapeutics Enters into a Collaboration and (globenewswire.com)

Sanofi pays Fulcrum $80M for rights to phase 3 rare disease drug (fiercebiotech.com)

Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trial - The Lancet Neurology