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BMRN Stock Down on Mixed Results From Rare Disease Therapy Study
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Key Takeaways
BioMarin shares fell 4% after BMN 401 missed one of two main goals in the phase III ENERGY 3 study.
BMRN reported higher plasma PPi levels, but no improvement in radiographic scores after 52 weeks.
BMN 401 also missed secondary goals tied to rickets severity, height and weight growth measures.
Shares of BioMarin Pharmaceutical (BMRN - Free Report) were down 4% on Monday after it reported results from the phase III ENERGY 3 study, which evaluated BMN 401, an investigational enzyme replacement therapy (ERT), for a rare genetic disorder called ENPP1 deficiency in children aged 1 to 12.
The study did not meet one of its two main goals.
BMRN’s Stock Performance
The mixed results were a setback for BioMarin, which added BMN 401 through its $270 million acquisition of Inozyme Pharma last year. This ERT was the lead asset in Inozyme’s pipeline, and the ENERGY 3 study was already underway at the time of the acquisition.
Year to date, the stock has lost 16% compared with the industry’s 3% fall.
Image Source: Zacks Investment Research
More on BioMarin’s ENERGY 3 Study Results
The study assessed two primary endpoints — changes in plasma inorganic pyrophosphate (PPi) and Radiographic Global Impression of Change (RGI-C) scores after 52 weeks of treatment. While treatment with BMN 401 after 52 weeks resulted in statistically significant increases in plasma PPi, there was no corresponding improvement in RGI-C scores.
An increase in plasma PPi is considered an important biomarker response because ENPP1 deficiency leads to low PPi levels, which can result in progressive damage to blood vessels, soft tissues and bones.
However, BMN 401 failed to demonstrate improvement in RGI-C scores, a key measure used to assess treatment impact in children with rickets. The lack of radiographic improvement suggests that the biomarker gains did not translate into measurable skeletal benefits during the study period.
The therapy also did not show positive trends across secondary endpoints, including Rickets Severity Score (RSS), a measure of rickets severity, and growth Z-scores evaluating height/body length and weight.
BioMarin stated that it will continue analyzing the complete ENERGY 3 dataset and engage with global regulatory authorities to determine the next steps for BMN 401’s development program. The company also plans to present detailed findings from the study at a future medical meeting.
BMRN’s Zacks Rank
The stock currently carries a Zacks Rank #3 (Hold).
Over the past 30 days, estimates for Immunocore’s 2026 loss per share have narrowed from 97 cents to 16 cents. Over the same period, estimates for 2027 have improved from a loss of 39 cents to earnings of 11 cents. IMCR shares have lost 18% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters but missed the mark on one occasion, delivering an average surprise of 46.66%.
Over the past 30 days, estimates for Indivior Pharmaceuticals’ 2026 EPS have increased from $3.10 to $3.35. Over the same period, EPS estimates for 2027 have risen to $3.69 from $3.47. INDV shares have risen 2% year to date.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 65.44%.
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BMRN Stock Down on Mixed Results From Rare Disease Therapy Study
Key Takeaways
Shares of BioMarin Pharmaceutical (BMRN - Free Report) were down 4% on Monday after it reported results from the phase III ENERGY 3 study, which evaluated BMN 401, an investigational enzyme replacement therapy (ERT), for a rare genetic disorder called ENPP1 deficiency in children aged 1 to 12.
The study did not meet one of its two main goals.
BMRN’s Stock Performance
The mixed results were a setback for BioMarin, which added BMN 401 through its $270 million acquisition of Inozyme Pharma last year. This ERT was the lead asset in Inozyme’s pipeline, and the ENERGY 3 study was already underway at the time of the acquisition.
Year to date, the stock has lost 16% compared with the industry’s 3% fall.
Image Source: Zacks Investment Research
More on BioMarin’s ENERGY 3 Study Results
The study assessed two primary endpoints — changes in plasma inorganic pyrophosphate (PPi) and Radiographic Global Impression of Change (RGI-C) scores after 52 weeks of treatment. While treatment with BMN 401 after 52 weeks resulted in statistically significant increases in plasma PPi, there was no corresponding improvement in RGI-C scores.
An increase in plasma PPi is considered an important biomarker response because ENPP1 deficiency leads to low PPi levels, which can result in progressive damage to blood vessels, soft tissues and bones.
However, BMN 401 failed to demonstrate improvement in RGI-C scores, a key measure used to assess treatment impact in children with rickets. The lack of radiographic improvement suggests that the biomarker gains did not translate into measurable skeletal benefits during the study period.
The therapy also did not show positive trends across secondary endpoints, including Rickets Severity Score (RSS), a measure of rickets severity, and growth Z-scores evaluating height/body length and weight.
BioMarin stated that it will continue analyzing the complete ENERGY 3 dataset and engage with global regulatory authorities to determine the next steps for BMN 401’s development program. The company also plans to present detailed findings from the study at a future medical meeting.
BMRN’s Zacks Rank
The stock currently carries a Zacks Rank #3 (Hold).
BioMarin Pharmaceutical Inc. Price
BioMarin Pharmaceutical Inc. price | BioMarin Pharmaceutical Inc. Quote
Key Picks Among Biotech Stocks
Some better-ranked stocks from the sector are Immunocore (IMCR - Free Report) and Indivior Pharmaceuticals (INDV - Free Report) , each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 30 days, estimates for Immunocore’s 2026 loss per share have narrowed from 97 cents to 16 cents. Over the same period, estimates for 2027 have improved from a loss of 39 cents to earnings of 11 cents. IMCR shares have lost 18% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters but missed the mark on one occasion, delivering an average surprise of 46.66%.
Over the past 30 days, estimates for Indivior Pharmaceuticals’ 2026 EPS have increased from $3.10 to $3.35. Over the same period, EPS estimates for 2027 have risen to $3.69 from $3.47. INDV shares have risen 2% year to date.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 65.44%.