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Wire

BioMarin Pharmaceutical Presents 3-Year Voxzogo Data, Early BMN 333 Results for Growth Disorder Treatments

BioMarin Pharmaceutical (BMRN) said Tuesday it presented new clinical data for its Voxzogo and BMN 333 treatments targeting hypochondroplasia and achondroplasia, both of which are growth-related bone conditions.An investigator-led Phase 2 extension study showed that Voxzogo led to sustained growth improvements in children with hypochondroplasia, the company said.The company intends to submit a supplemental New Drug Application to the US Food and Drug Administration during Q3 based on a separate Phase 3 study, it said.Initial Phase 1 testing of BMN 333 in healthy adults demonstrated prolonged drug exposure supporting a weekly dosing schedule for achondroplasia, BioMarin Pharmaceutical said.Patient enrollment for a Phase 2/3 study of the weekly drug began in April, with a data update from the dose-finding segment expected in 2027, the company added.Price: $55.13, Change: $-0.52, Percent Change: -0.93%

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Wire

BioMarin Pharmaceutical Reports No Functional Improvement in BMN 401 ENPP1 Deficiency Study

BioMarin Pharmaceutical's (BMRN) Phase 3 ENERGY 3 trial of BMN 401 in children with ENPP1 deficiency met one of two co-primary endpoints but did not show improvement in key clinical outcomes, the company said Monday.The study showed a statistically significant increase in plasma inorganic pyrophosphate levels but did not improve Radiographic Global Impression of Change scores, the primary measure of rickets severity.The company added that no notable trends were observed in secondary endpoints, and the therapy was generally well tolerated with no new safety concerns reported.Shares of BioMarin Pharmaceutical were down over 1% in Monday trading.Price: $51.27, Change: $-0.55, Percent Change: -1.05%

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Research

Research Alert: CFRA Keeps Buy Rating On Shares Of Biomarin Pharmaceutical Inc.

CFRA, an independent research provider, has providedwith the following research alert. Analysts at CFRA have summarized their opinion as follows:We retain our target at $75, based on our NPV analysis. We lower our 2026 EPS estimate from $5.17 to $4.96. We raise our 2027 EPS view from $5.86 to $6.69. BMRN reported a solid Q1 2026, highlighted by the recent closing of the acquisition of Amicus Therapeutics, which is set to accelerate revenue growth to a forecasted 20% Y/Y for 2026. The deal adds two high potential commercial products, Galafold for Fabry disease and Pombiliti + Opfolda for Pompe disease, to BMRN's portfolio, supporting revenue diversification, in our view. BMRN's total revenues rose to $766M in Q1 (+3% Y/Y), slightly above expectations, supported by healthy patient demand for the enzyme therapies and Voxzogo. Yet, non-GAAP EPS of $0.76 missed expectations primarily due to increased operating expenses. Looking ahead, the integration of Amicus, advancing the pipeline with key data readouts expected in Q2, and driving continued commercial success of the portfolio will be key for BMRN.

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Wire

BioMarin Pharmaceutical Q1 Revenue Beat Backed by Voxzogo Strength, Diversified Portfolio, Wedbush Says

BioMarin Pharmaceutical (BMRN) reported a Q1 beat on revenue, driven by Voxzogo and continued portfolio diversification amid emerging competition, Wedbush Securities said in a note Tuesday.Voxzogo and total revenue came in at $220 million and $766 million, compared with the consensus of $216 million and $757 million, respectively. The decline from Q4 was mainly seasonal, and the company expects a stronger H2, due to recent acquisitions of Galafold and Pombiliti/Opfolda, the firm said.The firm said the company has kept its Voxzogo guidance unchanged at $975 million to $1.025 billion, and despite rising competition, BioMarin plans to focus on execution to maintain its lead in achondroplasia using "robust" safety and efficacy data and broad early-treatment use."Recall management announced in January a goal of achieving sustained double-digit long-term total revenue compound annual growth rate into the 2030s, and we believe that BioMarin Pharmaceutical remains on track to deliver," the firm said.Wedbush Securities has an outperform rating and $94 price target on BioMarin Pharmaceutical.Price: $54.05, Change: $-1.42, Percent Change: -2.55%

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Wire

BioMarin Enters 'Transition Year' as Enzyme Portfolio Expands After Amicus Deal, Morgan Stanley Says

BioMarin Pharmaceutical (BMRN) is entering a "transition year" in 2026, prioritizing growth acceleration and market share gains as its expanded enzyme portfolio scales following the acquisition of Amicus Therapeutics (FOLD), Morgan Stanley said in a report Tuesday.In its Q1 results, BioMarin raised its fiscal 2026 revenue guidance to $3.825 billion to $3.925 billion, driven by contributions from newly added therapies Galafold and Pombiliti plus Opfolda, but lowered its non-GAAP earnings per share outlook to $4.85 to $5.05 due to integration-related costs and higher interest expenses, associated with the transaction, the report said.BioMarin is focused on scaling its expanded enzyme portfolio and driving commercial execution, with its management expected to outline a detailed roadmap in Q2 covering "long-term revenue potential," peak sales and cost synergies, the investment bank said.Earnings are expected to be weighted toward the second half of 2026, with the Amicus deal seen by the management as "slightly dilutive" this year but "accretive" within 12 months and more meaningful in start of 2027, the report said.The "combined enzyme portfolio" is expected to support durable growth and help offset rising competition for Voxzogo, while pipeline developments and new launches, including Palynziq expansion and BMN-333, could further support long-term growth, according to the report.Morgan Stanley has an overweight rating on BioMarin Pharmaceutical and lowered its price target to $119 from $120.Price: $54.26, Change: $-1.20, Percent Change: -2.16%

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Research

Research Alert: Bmrn Delivers Mixed Q1 Results As Amicus Deal Closes And Pipeline Advances

CFRA, an independent research provider, has providedwith the following research alert. Analysts at CFRA have summarized their opinion as follows:BioMarin delivered mixed Q1 2026 results with revenue of $766M (+3% Y/Y), beating consensus by $14M, but Non-GAAP EPS of $0.76 vs $1.13 (-33% Y/Y) missed consensus of $0.91. The quarter was impacted by a $31M NAGLAZYME manufacturing charge and Amicus acquisition costs. The $4.8B Amicus acquisition closed in April, adding GALAFOLD and POMBILITI + OPFOLDA to expand BioMarin's rare disease portfolio. Management raised 2026 revenue guidance to $3.825B-$3.925B, representing 20% Y/Y growth at the midpoint. Multiple pipeline catalysts are expected in 2026, including BMN 401 Phase 3 results for ENPP1 deficiency and VOXZOGO Phase 3 data for hypochondroplasia in Q2, both potentially supporting regulatory submissions in 2H 2026. BMRN expects more than 55% of 2026 revenues in the second half, driving back-end-loaded EPS recognition, in our view. With $2B in cash and $221M Q1 operating cash flow, BioMarin maintains substantial financial flexibility, in our opinion.

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Wire

BioMarin Seen Building Broader Rare-Disease Platform After Amicus Deal, Morgan Stanley Says

BioMarin Pharmaceutical (BMRN) is emerging as a broader rare-disease platform as its acquisition of Amicus Therapeutics (FOLD) adds two approved medicines and reduces reliance on Voxzogo, Morgan Stanley said Monday in a report.The Amicus deal reshapes BioMarin's outlook by adding Galafold and Pombiliti with Opfolda, products that fit the company's global commercial network, the report said. The therapies are expected to generate more than $1.3 billion in revenue by 2030, Morgan Stanley said.The acquisition is projected to accelerate revenue growth immediately after closing and to be accretive to non-GAAP earnings within 12 months, with "substantial accretion" beginning in 2027, the report said. BioMarin closed its $4.8 billion purchase of Amicus on Monday.Morgan Stanley resumed coverage on BioMarin stock with an overweight rating and a $120 price target.Price: $54.08, Change: $+0.90, Percent Change: +1.68%

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Wire

BioMarin Pharmaceutical Closes $4.8 Billion Acquisition of Amicus Therapeutics

BioMarin Pharmaceutical (BMRN) said Monday that it has completed its earlier stated acquisition of Amicus Therapeutics (FOLD) for $4.8 billion.The deal adds Galafold, an oral treatment for Fabry disease, and Pombiliti, Opfolda combination for Pompe disease to BioMarin's portfolio, the company said.BioMarin also said it now has US rights to investigational therapy DMX-200 for the treatment of focal segmental glomerulosclerosis.Shares of the company were up more than 1% in recent Monday trading.Price: $53.60, Change: $+0.42, Percent Change: +0.79%

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Research

Morgan Stanley Resumes BioMarin Pharmaceutical at Overweight With $120 Price Target

BioMarin Pharmaceutical (BMRN) has an average rating of overweight and mean price target of $90.17, according to analysts polled by FactSet.(covers equity, commodity and economic research from major banks and research firms in North America, Asia and Europe. Research providers may contact us here: https://www..com/contact-us)

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