Sanofi bets against haemophilia gene therapy

Trust Sanofi to dismiss a hot new therapy approach. Biomarin’s gene therapy last month made a splash in haemophilia, but the French group’s monster $11.6bn takeout of Bioverativ today looks like a bet on traditional factor replacements remaining a standard treatment for this blood disease for some time to come.

And this is no opportunistic acquisition: the price tag values Bioverativ at a 64% premium to its Friday close, and well over double the level at which it was spun out of Biogen less than a year ago. As such, Biogen can congratulate itself on having created an easily accessible, must-have asset.

Such a large mark-up to Bioverativ’s open market value suggests desperation on the part of Sanofi to make up for a considerable R&D shortfall, as well as the possible presence of more than one company interested in buying the Biogen spin-out. An established haemophilia player like Shire is one possible example.

But Shire is already well represented in factor-replacement therapies, though Bioverativ insists that its own marketed products, Eloctate for haemophilia A and Alprolix for haemophilia B, are an advance on Shire’s franchise by virtue of having prolonged circulation in the body. And Novo Nordisk’s recent attempt to buy Ablynx suggests an interest in rare blood disorders in general.


That said, the amount Sanofi is willing to pay could raise eyebrows, especially among gene therapy advocates; Bioverativ’s gene therapy presence is limited to preclinical assets in haemophilia A and B, partnered with the San Raffaele Telethon Institute, and a deal with Sangamo over ST-400 for beta-thalassemia and sickle cell disease.

Last month Biomarin’s valoctocogene roxaparvovec gene therapy shook up the Ash meeting with data suggesting that it could cure haemophilia A (Ash 2017 – Biomarin breaks away in haemophilia A gene therapy chase, December 9, 2017). But for Sanofi the proven safety of factor replacements has won the day, versus the largely unproven profile – not to mention pricing uncertainty – of gene therapies.

The French company is also betting against one of the hottest new entrants in haemophilia – Roche’s bispecific haemophilia A MAb Hemlibra, which might have potential in inhibitor and non-inhibitor patients alike (Roche’s latest Hemlibra win leaves no safe Haven for Shire, November 20, 2017).

On an analyst call today Sanofi insisted that it had extensively analysed haemophilia trends, and that its assessment adequately reflected competitive threats – including that of Hemlibra’s potential approval in the non-inhibitor population. Executives said they were convinced factor replacement would remain a standard of care for many years.

This is not to say that Sanofi has rejected all ground-breaking approaches; just two weeks ago it restructured a deal with Alnylam, giving it global rights to fitusiran, a phase III anti-thrombin III RNAi therapeutic in development for haemophilia A and B.

On the call Sanofi played up Bioverativ’s potential beyond the marketed factor replacements, with a pipeline including next-generation haemophilia A and B projects, assets against cold agglutinin disease, and the Sangamo deal. Sangamo was up 6% in early trade, while Swedish Orphan Biovitrum, which has rights to Eloctate in certain ex-US markets outside the US, was up 12%.

Biogen vindicated

Whatever risks Sanofi has opened itself up to in doing this deal, the move represents a fantastic outcome for Biogen, and vindicates the decision to deem its blood disorder business non-core and split this off as Bioverativ.

That transaction had been completed in February 2017, having been structured as a tax-free distribution of all of Bioverativ’s share capital to Biogen investors. It is not immediately clear how the takeout by Sanofi will affect the tax status of the distribution; Bivoerativ had not answered a question about this as EP Vantage went to press.

And spare a thought for the deal bankers, who in the space of two weeks have celebrated Celgene’s $1.1bn swoop on Impact Biomedicines, the $11.6bn Bioverativ deal, and today’s takeout of Juno by Celgene for $9bn. After a year that largely underwhelmed in terms of biopharma M&A, 2018 is already off to a roaring start.

To contact the writer of this story email Jacob Plieth in London at [email protected] or follow @JacobPlieth on Twitter

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