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BioMarin Posts '27 Biz View, Outlines Growth Plans for Next 10 Years

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At its investor day event on Wednesday, BioMarin (BMRN - Free Report) unveiled its long-term plans for growth, including potential drug approvals and revenue guidance over the next 10 years.

Management also updated the company’s organizational structure, splitting its existing business into three separate segments — skeletal conditions, enzyme therapies and Roctavian. Enzyme therapies include Aldurazyme, Brineura, Naglazyme, Palynziq and Vimizim.

BMRN Boasts $5B+ Revenue Opportunity From Voxzogo

Management expects Voxzogo, which is included in the skeletal conditions segment, to be a key driver of the company’s revenues. The drug, which was approved by the FDA in 2021, is currently the only medication approved to treat achondroplasia, the most common form of dwarfism.

By 2031, BioMarin aims to secure regulatory approvals for Voxzogo across five more indications — hypochondroplasia, idiopathic short stature, Noonan Syndrome, Turner Syndrome and SHOX deficiency. If approved across all these indications, management believes that it will increase the drug’s total addressable patient population from 24,000 in the current indication to nearly 420,000 across all six indications.

The company also provided an idea of the timeline by which it expects to have secured the regulatory label expansion for the drug in the above indications. It expects to secure the label for hypochondroplasia in 2027, followed by an approval for idiopathic short stature condition in 2030. It expects to get approval for the remaining indications by 2031.

BioMarin’s Updates on Pipeline & Regulatory Filings

The company is looking to launch 11 products by 2034, which includes two launches by 2027. To achieve these targets, management had undertaken a strategic portfolio review of all R&D programs. Based on the strategic review, BMRN decided to prioritize the development of multiple new pipeline candidates.

These include BMN 333 (for multiple growth disorders), BMN 349 (for liver disease associated with alpha-1 antitrypsin deficiency), BMN 351 (for Duchenne muscular dystrophy), BMN 370 (for von Willebrand Disease) and BMN 390 (for phenylketonuria [PKU]).

Management is also targeting regulatory filings for the PKU drug Palynziq, seeking label expansions in the adolescent age group in the United States by late 2025 and in ex-U.S. territories by early 2026.

Year to date, BioMarin’s stock has lost 6.8% compared with the industry’s 0.9% fall.

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Financial Updates

For 2024

BioMarin reiterated its financial guidance for 2024 — management expects total revenues in the range of $2.75-$2.83 billion and adjusted earnings per share to be in the range of $3.10-$3.25. Adjusted operating margin is expected to be between 26% and 27%.

Beyond 2024

Based on the anticipated new product launches, management issued fresh guidance for 2027 and also outlined growth plans through 2034.

The company expects sales in the Enzyme Therapies segment to grow annually at a high-single-digit percentage through 2034.

It expects to generate $4 billion in total revenues by 2027, up from the $2.4 billion generated in 2023. It also expects sales to register an annual growth rate in the mid-teens percentage through 2034.

BioMarin also expects its adjusted operating margin to be around 40% by 2026. Beyond 2026, management intends to keep this margin in the “low-to-mid 40%’s” range in the long term.

Management expects to generate more than $1.25 billion in operating cash flow by 2027.

 

BMRN’s Zacks Rank

BioMarin currently carries a Zacks Rank #3 (Hold).

Our Key Picks Among Biotech Stocks

Some better-ranked stocks include Arcturus Therapeutics (ARCT - Free Report) , Bioventus (BVS - Free Report) and Fulcrum Therapeutics (FULC - Free Report) , each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

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Earnings of Arcturus Therapeutics beat estimates in each of the last four quarters. Arcturus delivered a four-quarter average earnings surprise of 56.73%.

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BVS’ earnings beat estimates in three of the last four quarters and missed the mark on one occasion. Bioventus delivered a four-quarter average earnings surprise of 102.86%.

In the past 60 days, estimates for Fulcrum Therapeutics’ 2024 loss per share have improved from $1.24 to 48 cents. Estimates for 2025 have improved from $1.71 to $1.51 during the same period. Year to date, Fulcrum Therapeutics’ shares have increased 22.2%.

Earnings of Fulcrum Therapeutics beat estimates in each of the last four quarters. Fulcrum delivered a four-quarter average earnings surprise of 393.18%.

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