BioMarin starts rebuilding after its downsizing spree

 BioMarin has been resetting its business with cuts in headcount and a narrowing of active programmes over the last year or so, but is now looking to the future with a takeover deal for Inozyme Pharma that will boost its presence in rare diseases.

The $270 million, all-cash deal is the first major acquisition under new chief business officer James Sabry, who joined BioMarin from Roche last year in an appointment that suggested a new period of business development was on the way.

Inozyme's $4-per-share acquisition represents a relatively small deal for BioMarin, given that the company said earlier this year it was interested in transactions in the $1 billion range. Nevertheless, it adds a phase 3 enzyme replacement therapy (ERT) candidate – INZ-701 for rare disorder ENPP1 deficiency – which is in a phase 3 trial and bolsters a key growth sector for the biotech.

BioMarin has set a target if reaching $4 billion in annual revenues in 2027 across three main business areas – ERT, gene therapies, and skeletal disorders. Its ERT portfolio includes Aldurazyme (laronidase) for mucopolysaccharidosis I (MPS I) and Naglazyme (galsulfase) for MPS VI, and INZ-701 will slot neatly into the business unit.

According to Inozyme, ENPP1 deficiency is a rare genetic disease linked to disruptions in the PPi-adenosine pathway, leading to severe, lifelong cardiovascular and musculoskeletal symptoms, and has no FDA-approved treatments. It can lead to deposits of minerals in the soft tissues – including blood vessels, organs, joints, and ligaments – while at the same time weakening bones and causing a narrowing of blood vessels.

INZ-701 addresses an enzyme deficiency in the disorder and is heading for a phase 3 readout in paediatric ENPP1 deficiency patients next year, setting up a possible launch in 2027.

"BioMarin has been deeply committed to advancing enzyme therapies for children and adults living with serious genetic conditions for more than 25 years, and today's agreement builds on our legacy," said Alexander Hardy, the company's CEO, who also joined the company from the Roche group, replacing long-serving CEO Jean-Jacques Bienaimé at the end of 2023.

"This acquisition brings to BioMarin an important medicine that has the potential to be the first treatment for children and adults with ENPP1 deficiency, improving care for people living with this serious condition."

BioMarin has been slashing staff as part of a restructuring drive that also included a scaling down of operations supporting haemophilia A gene therapy Roctavian (valoctocogene roxaparvovec) to three key markets, after a disappointing rollout, and narrowing its R&D programmes.

It remains reliant on Voxzogo (vosoritide) for skeletal disorders, including achondroplasia, which contributed $735 million in sales last year, although competition could be on the way from Ascendis Pharma's TransCon CNP (navepegritide), BridgeBio's infigratinib, and Robomic's RBM-007.

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