potential partner, page-5

OK, here’s what I think Sillazze is keen to point out. Please correct me if I’m wrong Sillazze.

Genzyme was acquired by French pharma Sanofi in 2011 with Sanofi’s intention to make Genzyme its global centre for excellence in rare diseases. Genzyme is based in the Greater Boston area of Massachusetts.

Sanofi and Genzyme are also joined in a corporate venture capital arm known as Sanofi-Genzyme BioVentures (SGBV). SGBV has to date invested in several biotechs in the rare disease space, including an investment in 2015 in Ovid Therapeutics, which has an oral drug in early stage development for Fragile-X.

In 2015, Sillazze brought our attention to a development and option licence deal that Sanofi had signed earlier that year with gene therapy startup, Voyager (which is also based in Massachusetts). Voyager’s R & D programs were at Phase 1 stage or earlier and included therapies for Parkinson’s disease, Friedrich's ataxia and Huntington’s disease. Under the terms of the 2015 deal, Sanofi agreed to Voyager retaining US rights to the Parkinson’s disease and Friedrich’s ataxia therapies.

What has now caught Sillazze’s attention is an announcement on October 30 that Sanofi has opted out of Voyager’s lead Parkinson’s program, which was soon to enter a pivotal Phase 2/3 trial. Voyager claimed that Sanofi walked because it wasn’t given US rights. However analysts raised a red flag over the exit, querying what had changed since the original deal was struck. The only explanation given by Sanofi was “strategic reasons”.

As well as obviously being interested in therapies for Fragile-X, Parkinson’s disease and ataxia, Sanofi Genzyme also has a division specifically devoted to MS. It currently has two approved therapies in this franchise, one oral and the other injectable, but wishes to add to this. Just last week, Sanofi announced an $805 million deal to license a pre-clinical multiple sclerosis drug from Principia Biopharma (a Californian-based private biotech). The deal allowed for a $40 million upfront with $765 million in potential milestones.

As previously discussed, Sanofi Genzyme has also identified Rett Syndrome as one of the rare disease indications for which it is seeking and advancing projects.

Sanofi Genzyme has flagged that it is interested in acquiring more rare disease assets. In an interview last year, the CEO of Genzyme CEO said the company was surveying the field of rare disease-focused biotech companies, but declined to name names and the CEO of Sanofi said that the company was interested in deals big and small to counteract creeping competition and sluggish sales.

Would Sanofi Genzyme be interested in Neuren?

Trofinetide has demonstrated efficacy in Phase 2 trials of Rett Syndrome and Fragile-X and Neuren has stated that it is commencing development of trofinetide for Fragile X‐associated tremor/ataxia syndrome before the end of this year. Also worth considering is the fact that Neuren’s second drug, NNZ-2591, has to date demonstrated efficacy in pre‐clinical models including Parkinson’s disease, Fragile X syndrome, and MS.

What I think Sillazze is saying is that he is keeping a very close eye on Sanofi Genzyme!