As uptake of BioMarin’s gene therapy stalls, analysts see potential for divestment
26 Apr 2024
Phase 1Gene TherapyAcquisitionDrug Approval
BioMarin’s first quarter earnings report suggests little headway has been made in its ongoing struggle to get its haemophilia A gene therapy off the ground since receiving European regulatory clearance in mid-2022. Now, the biotech is evaluating three paths forward for Roctavian (valoctocogene roxaparvovec), including a potential divestment – which caught the eye of analysts.
The gene therapy has had to compete with Roche’s antibody Hemlibra, the standard of care in haemophilia A, which brought in over $4 billion in revenue for 2023. (See - Vital Signs: Roche outmanoeuvres another gene therapy).
In contrast, BioMarin reported $0.8 million in sales for Roctavian for the first quarter, associated with one patient treated in Italy. Last year, three patients received the gene therapy for a total revenue of $3.5 million – far short of the $50-$150 million estimate given after it won FDA approval in June of last year. (See - Spotlight On: BioMarin’s gene therapy promise falls short).
On a call with investors, CEO Alexander Hardy said uptake for Roctavian “continues to be complex,” citing barriers to reimbursement in Germany and the US. He did say the firm is moving forward with development in Japan, a market with “high potential.”
By its September 4 investor day, BioMarin plans to evaluate sales and expenditures associated with Roctavian and decide whether to continue full-throttle development of the programme. If sales begin to ramp up, the company will stay the course, and if uptake grows, but slowly, Hardy said the firm will seek a “reasonable return on investment by right-sizing the level of investment.”
But if turning a profit seems out of reach, BioMarin will remove the gene therapy from its portfolio.
“We believe a potential Roctavian divestiture would likely be perceived as a positive by most investors,” BMO Capital Markets analysts wrote in a note.
However, Hardy cautioned that divesting the gene therapy “is not our focus right now… our focus is really on establishing the opportunity” for Roctavian.
Pipeline cull
Separately in its earnings report, BioMarin disclosed it has axed four pipeline programmes, half of which were in the clinic, according to ClinicalTrials.gov.
Gene therapy BMN 331 was in a Phase I/II trial for hereditary angioedema, and BMN 255 had recently begun a Phase I study to treat non-alcoholic fatty liver disease and hyperoxaluria.
On the preclinical side, BMN 355 was an antibody intended to treat long QT syndrome, and gene therapy BMN 365 was in development for arrhythmogenic cardiomyopathy.
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