Rare Disease-Focused BioMarin Narrows Sales Focus, Cuts Cost For Hemophilia A Gene Therapy; Lifts Annual Guidance

BioMarin Pharmaceutical Inc. BMRN announced an update on its Roctavian business to focus commercial operations on three markets: the U.S., Germany and Italy.

These markets are where the medicine is approved and reimbursed as a severe hemophilia A treatment.

The company will adjust its Roctavian efforts and reduce additional investments in development and manufacturing.

BioMarin anticipates reducing annual direct Roctavian expenses to approximately $60 million beginning in 2025. The company has already begun operationalizing the reduction of Roctavian expenses this year to achieve $60 million in expenses beginning in full-year 2025.

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As a result, the company expects Roctavian to be profitable by the end of 2025.

The company’s gene therapy manufacturing facility has been placed in “an idle state” until additional doses are needed. At this time, the company is making no further investments to reach additional markets.

BioMarin reported second-quarter adjusted EPS of 96 cents, up 78% year over year and beating the consensus of $0.35.

Sales increased 20% to $712.03 million. It beat the consensus of $662.05 million, driven by Voxzogo (dwarfism drug) and Naglazyme (drug for rare genetic metabolic disorder).

“Strong global demand for Voxzogo led to nearly 900 new patient starts in the first half of 2024, the highest in Voxzogo’s history,” said Alexander Hardy, President and CEO.

During the quarter, BioMarin treated three patients with Roctavian in the U.S. and two in Italy, generating $7 million in revenue.

BioMarin also decided to discontinue BMN 293, a preclinical gene therapy for myosin-binding protein C3 hypertrophic cardiomyopathy (thickened heart muscles).

Guidance: BioMarin expects 2024 revenues of $2.75 billion—$2.825 billion. Prior guidance hovered between $2.7 billion and $2.8 billion with the consensus of $2.74 billion.

The company raised its adjusted EPS guidance to $3.10-$3.25 from the prior guidance of $2.75-$2.95 above the consensus of $2.87.

William Blair writes, “With the impressive launch of Voxzogo, the company’s largest approved product to date, and a new CEO focused on expanding margins, we see strong potential for BioMarin to accelerate EPS growth in the coming years.”

“With a promising growth profile and a renewed focus on profitability, we view BioMarin as a core holding in the sector.”

The analyst maintains the Outperform rating.

Price Action: BMRN stock is up 7.69% at $86.66 at last check Tuesday.

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