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BioMarin Discontinues Preclinical Drug Candidate, Advances BMN 333 Toward 2030

Vandana Singh
August 05, 2025

On Tuesday, Biomarin Pharmaceutical Inc. (NASDAQ:BMRN) reported second-quarter adjusted earnings of $1.44, up 50% year over year, beating the consensus of 85 cents and sales of $825.41 million, up 16%, beating the consensus of $760.39 million.

The sales increase was driven by strong 20% year-over-year growth in Voxzogo revenue from new patients initiating therapy across all regions. Global Voxzogo revenue growth in the quarter was led by U.S. contributions, with most new patients starting in the 0-4 year age group.

In the quarter, revenues from BioMarin’s Enzyme Therapies (Aldurazyme, Brineura, Naglazyme, Palynziq, and Vimizi) increased 15%, driven by a combination of increased patient demand in all regions and the timing of large government orders.

The increase was partially offset by lower Kuvan product revenues, which were attributed to continued generic competition due to the loss of market exclusivity.

Also Read: BioMarin Seeks Label Expansion For Palynziq After Positive Phase 3 Results In Adolescent Patients

BioMarin determined that BMN 390, a preclinical candidate for phenylketonuria (PKU), did not meet its target immunogenicity threshold for advancement. The company discontinued the program.

Guidance: The company raised its fiscal 2025 adjusted earnings guidance from $4.20 to $4.40 per share to $4.40 to $4.55, surpassing the consensus of $3.45. It also raised its 2025 sales guidance from $3.1 billion to $3.2 billion to $3.125 billion to $3.2 billion, versus the consensus of $3.14 billion.

Voxzogo’s contribution to 2025 revenues is expected to be $900 million to $935 million.

Trial Data:

Tucked in its earnings release, BioMarin announced data from a Phase 1 study in healthy volunteers with BMN 333, a long-acting C-type natriuretic peptide (CNP).

The study demonstrated area-under-the-curve (AUC) pharmacokinetic (PK) levels greater than three times the levels observed in other long-acting CNP studies.

No safety signals were noted. BioMarin is advancing plans to initiate the registration-enabling Phase 2/3 study in the first half of 2026. Should data be supportive, BMN 333 is on track for a potential 2030 launch.

William Blair wrote, “While we see BioMarin’s revenue beat and modified revenue guidance as an incremental positive, we highlight that it was driven by stronger-than-expected revenue from its enzyme therapy business, with the company lowering its expectations for 2025 Voxzogo revenue.”

Analyst Sami Corwin says BioMarin’s current valuation adequately reflects its top-line growth potential over the next 12 to 18 months.

For BMN 333 data, the analyst says initial data are highly encouraging and suggest it could be a best-in-class compound. However, it is unlikely to generate revenue until 2030.

“Outside its skeletal muscle franchise, we see the company’s enzyme therapies business unit as remaining a steady but modest revenue growth driver, but believe the entrance of competition to Palynziq in 2025 could also impact the business unit’s CAGR,” William Blair said in an investor note on Tuesday.

UBS maintains a Buy rating for BioMarin, raising the price forecast from $113 to $114.

Price Action: BMRN stock is up 5.71% at $63.75 at last check on Wednesday.

Read Next:

  • Warren Buffett’s Berkshire Hathaway Offloads $230 Million In DaVita Stock Ahead Of Q2 Earnings

Photo: Shutterstock

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