Please login to the form below

Not currently logged in

Sanofi exits R&D pact with Alnylam on rare disease

Exit also frees up $700m equity investment


Sanofi is bowing out of a five-year-old R&D alliance with Alnylam on rare disease therapies, but the partners will continue to develop the drugs that have already emerged from the programme.

The move reflects shifting investment priorities at the Paris-headquartered big pharma company, which now has a strong in-house pipeline of specialist blood disorder drugs following the twin acquisitions of Bioverativ an Ablynx last year.

Sanofi and Alnylam started working together on gene-silencing therapies in 2014, with the French drugmaker’s rare disease unit Genzyme making a $700m investment in the RNA interference specialist and claiming global rights to three drugs in Alnylam’s pipeline.

Genzyme had previously licensed rights in 2012 to Alnylam’s lead RNAI drug Onpattro (patisiran) for hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), which became the first drug in the RNAi class to be approved for marketing in the US last August.

The R&D deal was tweaked in January last year, with Sanofi licensing an RNAi drug called fitusiran for haemophilia A and B as well as other rare bleeding disorders, but returning development and commercial rights to patisiran and follow-up hATTR amyloidosis therapy vutrisiran ALNTTRsc02 to Alnylam and settling for a royalty stream for those drugs.

The latest announcement ends the R&D phase of the collaboration, which means that no additional compounds will be advanced by the partners, with the exception of one very early-stage programme in an undisclosed rare genetic disease.

Alnylam is preparing that for clinical testing, and if Sanofi exercises its option it will be responsible for any future development and commercialisation, paying royalties to Alnylam if approved.

Ending the R&D arrangement also means that Sanofi is able to free up some of the $700m equity investment in Alnylam, which had been locked up under the original terms of the 2014 deal.

Bringing the collaboration to an end means that the net yield of the agreement for Sanofi has been one late-stage candidate for haemophilia, royalties on Onpattro and potentially vutrisiran if approved, and the early-stage candidate.

Prospects for haemophilia candidate fitusiran looked a little shaky in 2017 when a fatal blood clot in one patient treated with the drug caused the FDA to suspend phase 2 and 3 trials.

The clinical hold was lifted a few weeks later however, and fitusiran is now well advanced in the phase 3 ATLAS programme, which includes three separate trials that will recruit patients with haemophilia A and B with or without inhibitors and patients receiving prophylactic, or preventive, therapy.

Haemophilia and other rare blood diseases have become key therapeutic targets for Sanofi in the wake of its acquisition of Bioverativ – which gave it long-acting clotting factor therapies Eloctate and Alprolix – and the takeover of Belgian biotech Ablynx last year to claim rights to acquired thrombotic thrombocytopenic purpura (aTTP) therapy Cablivi (caplacizumab).

The French drugmaker is also working on sutimlimab, an antibody for cold agglutinin disease in phase 3 testing, long-acting clotting factor BIVV001 for haemophilia A (phase 2) and gene-editing therapies for beta thalassemia and sickle cell disease.

Article by
Phil Taylor

8th April 2019

From: Marketing



COVID-19 Updates and Daily News

Featured jobs


Add my company
Fishawack Health

Fishawack Health is the leading global commercialization partner for the modern life science era. Established in 2001 and headquartered in...

Latest intelligence

The disparity of patient experiences with Crohn’s Disease in Europe and Japan...
Patient diversity in clinical trials
The pursuit of patient diversity in clinical trials
When all-inclusive isn’t an option, it’s an obligation...
Overcoming the digital patient recruitment challenges of connecting with patients
How many irrelevant experiences, ads, emails, direct messages have you rejected this week?...