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BioMarin Inks $490M Deal to Buy Rare Bone Disease Treatment
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Key Takeaways
BioMarin will acquire ALE1 for up to $490M, adding a potential oral therapy for rare HPP.
The deal could broaden BioMarin's skeletal-disease portfolio beyond Voxzogo.
ALE1 may offer a more convenient option than Strensiq, the established injectable HPP treatment.
BioMarin Pharmaceutical (BMRN - Free Report) announced that it has entered into a definitive agreement to acquire clinical-stage biotech Alesta Therapeutics for up to $490 million. This includes an upfront payment of $275 million and the remaining $215 million in development and regulatory milestone payments.
Under the deal structure, BioMarin is effectively acquiring Alesta's lead clinical-stage asset, ALE1, rather than the company's entire existing portfolio. The drug is being developed in a phase I/IIa study as a potential treatment for hypophosphatasia (HPP), a rare genetic bone disease. If left untreated, HPP can lead to impaired bone and tooth mineralization, fractures, muscle weakness, fatigue and pain.
Immediately before the transaction closes, Alesta will spin out all non-ALE1 assets into a new entity, with Alesta employees transferring to the spinout. BioMarin will therefore gain ALE1 while avoiding the acquisition of the other assets and workforce associated with Alesta.
The boards of directors of both companies have approved the deal, which is expected to close in the third quarter of 2026, subject to customary closing conditions. BioMarin plans to fund the transaction with cash on hand. Although the company did not provide the financial impact of this transaction on its 2026 adjusted EPS, it expects the deal to have a modestly dilutive impact.
BioMarin to Broaden Rare Bone Disease Portfolio
The acquisition would expand BioMarin’s skeletal-disease portfolio, which is currently anchored by Voxzogo, the company’s largest product by net sales. The drug is on track to achieve blockbuster status in sales this year. Adding an HPP program would give the company exposure to another rare bone disorder and broaden its presence across the skeletal conditions market.
ALE1 is designed to target inorganic pyrophosphate (PPi), a metabolite central to HPP, with the potential to address both skeletal and broader disease manifestations through systemic correction of the underlying biology. The drug could also potentially offer a differentiated treatment approach in HPP, given its oral formulation. This could provide a more convenient alternative to the blockbuster therapy Strensiq, the established HPP treatment marketed by AstraZeneca (AZN - Free Report) through its Alexion division and administered via subcutaneous injection.
The commercial opportunity is significant, with Strensiq generating $1.05 billion in sales in the first half of 2026, up 41% year over year, driven by continued demand from HPP patients. The product’s performance highlights the established demand for HPP treatment that BioMarin could potentially address with ALE1.
BMRN Stock Performance
Shares of BioMarin were up 0.5% on Tuesday following the acquisition announcement. The deal was broadly consistent with management’s previously stated business-development strategy.
BioMarin completed the Amicus Therapeutics acquisition in April, which added two new marketed products to its portfolio — Galafold (for Fabry disease) and Pombiliti-Opfolda (a combination therapy for Pompe disease). During its second-quarter 2026 earnings call, management said the integration had strengthened and diversified its commercial portfolio, allowing the company to shift its focus toward expanding its clinical-stage pipeline. It added that BioMarin plans to pursue deals to expand its clinical-stage programs over the next 12 to 18 months.
Year to date, shares of BMRN have gained 13% compared with the industry’s 6% growth.
Image Source: Zacks Investment Research
BioMarin Bolsters Pipeline After Recent Setback
The Alesta acquisition comes at a time when BioMarin seeks to replenish its clinical-stage pipeline following a setback with BMN 401, an investigational enzyme replacement therapy (ERT). In May, BioMarin announced mixed results from the phase III ENERGY 3 study, which evaluated this therapy for a rare genetic disorder called ENPP1 deficiency in children aged one to 12 years. The study did not meet one of its two main goals and failed to show positive trends across secondary endpoints.
BMN 401 was also being developed for two other rare disorders, but the company recently announced that it is discontinuing development of the therapy across all indications.
The decision represents a significant setback for the company, which acquired the therapy through its $270 million acquisition of Inozyme Pharma last year. BMN 401, formerly known as INZ-701, was the lead asset in Inozyme’s pipeline and was already in phase III development for ENPP1 deficiency when BioMarin acquired the company.
BMRN’s Zacks Rank
BioMarin currently carries a Zacks Rank #3 (Hold).
Over the past 30 days, earnings per share (EPS) estimates for Anika Therapeutics have risen from 41 cents to $1.05 for 2026. Over the same period, EPS estimates have increased from 46 cents to 95 cents for 2027. ANIK shares have skyrocketed 124% year to date.
Anika Therapeutics missed on earnings in each of the trailing four quarters, delivering an average surprise of 950%.
Over the past 30 days, estimates for Repligen’s 2026 EPS have increased to $2.06 from $1.99. Over the same period, EPS estimates for 2027 have risen from $2.57 to $2.61. RGEN shares have gained 1% so far this year.
Repligen’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 16.80%.
View the Zacks #1 Rank List
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BioMarin Inks $490M Deal to Buy Rare Bone Disease Treatment
Key Takeaways
BioMarin Pharmaceutical (BMRN - Free Report) announced that it has entered into a definitive agreement to acquire clinical-stage biotech Alesta Therapeutics for up to $490 million. This includes an upfront payment of $275 million and the remaining $215 million in development and regulatory milestone payments.
Under the deal structure, BioMarin is effectively acquiring Alesta's lead clinical-stage asset, ALE1, rather than the company's entire existing portfolio. The drug is being developed in a phase I/IIa study as a potential treatment for hypophosphatasia (HPP), a rare genetic bone disease. If left untreated, HPP can lead to impaired bone and tooth mineralization, fractures, muscle weakness, fatigue and pain.
Immediately before the transaction closes, Alesta will spin out all non-ALE1 assets into a new entity, with Alesta employees transferring to the spinout. BioMarin will therefore gain ALE1 while avoiding the acquisition of the other assets and workforce associated with Alesta.
The boards of directors of both companies have approved the deal, which is expected to close in the third quarter of 2026, subject to customary closing conditions. BioMarin plans to fund the transaction with cash on hand. Although the company did not provide the financial impact of this transaction on its 2026 adjusted EPS, it expects the deal to have a modestly dilutive impact.
BioMarin to Broaden Rare Bone Disease Portfolio
The acquisition would expand BioMarin’s skeletal-disease portfolio, which is currently anchored by Voxzogo, the company’s largest product by net sales. The drug is on track to achieve blockbuster status in sales this year. Adding an HPP program would give the company exposure to another rare bone disorder and broaden its presence across the skeletal conditions market.
ALE1 is designed to target inorganic pyrophosphate (PPi), a metabolite central to HPP, with the potential to address both skeletal and broader disease manifestations through systemic correction of the underlying biology. The drug could also potentially offer a differentiated treatment approach in HPP, given its oral formulation. This could provide a more convenient alternative to the blockbuster therapy Strensiq, the established HPP treatment marketed by AstraZeneca (AZN - Free Report) through its Alexion division and administered via subcutaneous injection.
The commercial opportunity is significant, with Strensiq generating $1.05 billion in sales in the first half of 2026, up 41% year over year, driven by continued demand from HPP patients. The product’s performance highlights the established demand for HPP treatment that BioMarin could potentially address with ALE1.
BMRN Stock Performance
Shares of BioMarin were up 0.5% on Tuesday following the acquisition announcement. The deal was broadly consistent with management’s previously stated business-development strategy.
BioMarin completed the Amicus Therapeutics acquisition in April, which added two new marketed products to its portfolio — Galafold (for Fabry disease) and Pombiliti-Opfolda (a combination therapy for Pompe disease). During its second-quarter 2026 earnings call, management said the integration had strengthened and diversified its commercial portfolio, allowing the company to shift its focus toward expanding its clinical-stage pipeline. It added that BioMarin plans to pursue deals to expand its clinical-stage programs over the next 12 to 18 months.
Year to date, shares of BMRN have gained 13% compared with the industry’s 6% growth.
Image Source: Zacks Investment Research
BioMarin Bolsters Pipeline After Recent Setback
The Alesta acquisition comes at a time when BioMarin seeks to replenish its clinical-stage pipeline following a setback with BMN 401, an investigational enzyme replacement therapy (ERT). In May, BioMarin announced mixed results from the phase III ENERGY 3 study, which evaluated this therapy for a rare genetic disorder called ENPP1 deficiency in children aged one to 12 years. The study did not meet one of its two main goals and failed to show positive trends across secondary endpoints.
BMN 401 was also being developed for two other rare disorders, but the company recently announced that it is discontinuing development of the therapy across all indications.
The decision represents a significant setback for the company, which acquired the therapy through its $270 million acquisition of Inozyme Pharma last year. BMN 401, formerly known as INZ-701, was the lead asset in Inozyme’s pipeline and was already in phase III development for ENPP1 deficiency when BioMarin acquired the company.
BMRN’s Zacks Rank
BioMarin currently carries a Zacks Rank #3 (Hold).
BioMarin Pharmaceutical Inc. Price
BioMarin Pharmaceutical Inc. price | BioMarin Pharmaceutical Inc. Quote
Our Key Picks Among Biotech Stocks
Some better-ranked stocks in the biotech sector are Anika Therapeutics (ANIK - Free Report) and Repligen Corporation (RGEN - 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, earnings per share (EPS) estimates for Anika Therapeutics have risen from 41 cents to $1.05 for 2026. Over the same period, EPS estimates have increased from 46 cents to 95 cents for 2027. ANIK shares have skyrocketed 124% year to date.
Anika Therapeutics missed on earnings in each of the trailing four quarters, delivering an average surprise of 950%.
Over the past 30 days, estimates for Repligen’s 2026 EPS have increased to $2.06 from $1.99. Over the same period, EPS estimates for 2027 have risen from $2.57 to $2.61. RGEN shares have gained 1% so far this year.
Repligen’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 16.80%.