BioMarin Beats on Q3 Earnings, Seeks to Divest Hemophilia Gene Therapy

BioMarin Pharmaceutical BMRN reported third-quarter 2025 adjusted earnings per share (EPS) of 12 cents against the Zacks Consensus Estimate of a loss of 15 cents. Quarterly earnings plummeted nearly 87% year over year, primarily due to IPR&D charges of $221 million recorded by the company in connection with the recently completed acquisition of Inozyme Pharma. Total revenues were $776 million, up 4% year over year. However, the figure missed the Zacks Consensus Estimate of $784 million. Product revenues totaled nearly $761 million, up 4% year over year on higher revenues from Voxzogo and Palynziq. This was partially offset by lower sales of Aldurazyme and Kuvan. Royalty and other revenues totaled over $15 million, up 29% year over year. Voxzogo, approved for achondroplasia, generated sales of $218 million, up 15% year over year, driven by new patients initiating the therapy across ex-U.S. territories. This growth was partially offset by unfavorable order timing fluctuations during the quarter. In the United States, uptake was stronger among children under two years of age, while adoption among older children remained slower. The company is implementing initiatives to expand treatment in this segment, which it expects will take time to show results. Owing to these factors, Voxzogo sales missed both the Zacks Consensus Estimate of $234 million and our model estimates of $231 million. Last year, the company updated its organizational structure and decided to consolidate revenues from its five products -- Aldurazyme, Brineura, Naglazyme, Palynziq and Vimizim -- under a single head, 'Enzyme Therapies.' Sales of this franchise rose 1% year over year to $516 million. Palynziq injection sales totaled $109 million in the quarter, up 20% year over year, driven by greater numbers of patients titrating to the daily-maintenance dose and strong adherence. The drug's sales beat both the Zacks Consensus Estimate and our model estimates of $103 million and $107 million, respectively. Vimizim sales rose 3% year over year to $183 million, which missed both the Zacks Consensus Estimate of $192 million and our model estimate of $188 million. Naglazyme sales declined 8% year over year to $122 million, due to unfavorable timing of large government orders in Latin America. Brineura added sales of $48 million, up 30%. Product revenues from Aldurazyme totaled $54 million, down 24% year over year, due to the unfavorable timing of order fulfillment to Sanofi SNY. BioMarin signed a collaboration agreement with Sanofi's subsidiary, Genzyme, for Aldurazyme. SNY, through Genzyme, is BMRN's sole customer for Aldurazyme. The Sanofi subsidiary is responsible for marketing and selling Aldurazyme to third parties. New gene therapy, Roctavian, generated $3 million in sales compared with $7 million in the year-ago period. In the phenylketonuria (PKU) franchise, Kuvan revenues declined 14% to $24 million due to generic competition. The drug lost U.S. market exclusivity in late 2020. BioMarin revised its financial guidance for the full year. It now expects total revenues in the range of $3.15-$3.20 billion, which reflects a $25 million increase from the lower end of the previous guidance of $3.13-$3.20 billion. The upgrade in guidance implies an increase of more than 11% year over year at the midpoint. The company expects Voxzogo to be a significant contributor to the top line, forecasting sales in the $900-$935 million band (unchanged). The drug's revenues are expected to reach their highest level in 2025 during Q4. Adjusted operating margin is now expected to be between 26% and 27%, compared with the prior expectations of 33-34%. This downward revision primarily reflects expenses incurred during the third quarter to close the Inozyme acquisition. Due to the Inozyme acquisition, BioMarin also lowered its adjusted EPS guidance, which is now expected to be in the range of $3.50-$3.60, down from the prior forecast of $4.40-$4.55. This revised guidance implies 1% growth over last year's level at the midpoint. During theearnings call BioMarin announced that it is rescinding its previously issued financial guidance for 2027, primarily due to competitive concerns around Voxzogo and the decision to divest Roctavian. The company also noted that business development remains a top strategic priority. CEO Alexander Hardy highlighted that BioMarin is actively pursuing multiple partnership and acquisition opportunities, including late-stage and commercial assets. It cited an estimated $4-$5 billion in total "firepower" available for future growth initiatives, supporting its disciplined capital allocation and business development strategy. Alongside the earnings results, BioMarin announced plans to divest Roctavian and remove it from its portfolio of marketed products. This decision is part of the company's strategy to focus on areas that align with its strategic priorities. In the meantime, BioMarin will continue to market the gene therapy across the United States, Italy and Germany, until a final decision is made. In April, BioMarin reported positive results from the late-stage PEGASUS study, which showed that adolescents with PKU treated with Palynziq achieved a statistically significant lowering in blood Phe levels. Based on this data, the company will target regulatory filings for expanded use of the drugs in adolescents in the United States and Europe later this year. BioMarin continues to advance its CANOPY clinical program, which evaluates Voxzogo for a potential second indication -- hypochondroplasia, a condition characterized by impaired bone growth. Data from this study is expected in the first half of 2026. As part of this program, BMRN is also evaluating the drug for four other short-stature pathway conditions -- idiopathic short stature, Noonan Syndrome, Turner Syndrome and SHOX deficiency. Based on the success seen with Voxzogo as a C-type natriuretic peptide (CNP) analog, BioMarin decided to advance BMN 333 -- a long-acting formulation of CNP -- as a potential treatment for multiple growth-related conditions, offering the option for less frequent dosing. The company plans to start a registration-enabling phase II/III study in the first half of 2026, aiming for a potential 2030 launch. The Inozyme acquisition added an investigational late-stage enzyme replacement therapy called BMN 401 (formerly INZ-701), which is being developed in a late-stage study to treat a rare disorder called ENPP1 deficiency in children aged 1-12. Initial results from this study are expected in the first half of 2026. BioMarin expects to report initial proof-of-concept data from an early-stage study on BMN 351, an oral therapeutic for liver disease associated with alpha-1 antitrypsin deficiency, before this year's end. EPS estimates for Alkermes' 2025 have increased from $1.81 to $1.85, while those for 2026 have risen from $1.68 to $1.70 in the past 60 days. ALKS stock has gained nearly 4% year to date. Alkermes' earnings beat estimates in two of the trailing four quarters and missed the mark on the other two occasions, delivering an average negative surprise of 1.19%. In the past 60 days, loss per share estimates for Werewolf Therapeutics' 2025 have improved from $1.88 to $1.61. Loss per share estimates for 2026 have narrowed from $1.61 to $1.36 during the same period. HOWL stock has gained over 12% year to date. Werewolf's earnings beat estimates in three of the trailing four quarters but missed the mark once, delivering an average surprise of 7.36%. Artificial intelligence has already reshaped the investment landscape, and its convergence with quantum computing could lead to the most significant wealth-building opportunities of our time. Today, you have a chance to position your portfolio at the forefront of this technological revolution. In our urgent special report, Beyond AI: The Quantum Leap in Computing Power, you'll discover the little-known stocks we believe will win the quantum computing race and deliver massive gains to early investors. Sanofi (SNY) : Free Stock Analysis Report BioMarin Pharmaceutical Inc. (BMRN) : Free Stock Analysis Report Alkermes plc (ALKS) : Free Stock Analysis Report Werewolf Therapeutics, Inc. (HOWL) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com).

BioMarin Beats on Q3 Earnings, Seeks to Divest Hemophilia Gene Therapy

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