Latest news with #ENPP1
Otago Daily Times
2 days ago
- Health
- Otago Daily Times
Discovery of genes relevant to disease a ‘eureka moment'
University of Otago microbiologist and immunologist Dr Tony Merriman. PHOTO: SUPPLIED Treatment for calcium pyrophosphate deposition (CPPD) disease — more commonly known as inflammatory arthritis — may soon take a major step forward following a major discovery by a University of Otago researcher. There is an unmet need for treatment of CPPD disease, which focuses on the alleviation of inflammation with non-steroidal anti-inflammatory drugs. But University of Otago microbiologist and immunologist Dr Tony Merriman has led an analysis of the genes of 550,000 Americans with the disease, while based at the University of Alabama, Birmingham. It is a first-of-its-kind genome-wide association study into the disease and the research found two genes — RNF144B and ENPP1 — that cause the debilitating calcium pyrophosphate crystal deposits in joints. He said the genes were detected both in people of European ancestry and African ancestry. "The most significant result of our research was the discovery of one of the genes, ENPP1. "The protein encoded by this gene controls the production of chemicals — adenosine monophosphate and inorganic pyrophosphate — that, together with calcium ions, lead to the formation of the CPP crystals," Dr Merriman said. Little is known about the other gene — RNF144B — aside from the possibility it is involved in inflammation. More was known about ENPP1 and of its potential importance to people with CPPD disease. Drugs targeting the protein have been developed in the treatment of infectious disease and cancer and they could now be evaluated for the treatment of CPPD disease. Dr Merriman said the discovery opened up promising new avenues for targeted prevention and treatment of CPPD disease, which were lacking at present. Co-investigator and Brigham and Women's Hospital rheumatology, inflammation and immunity division researcher Dr Sara Tedeschi said the genome-wide association with ENPP1 was "particularly exciting" as a rheumatologist because "it makes sense". "ENPP1 generates inorganic pyrophosphate, one of the components of CPP crystals. "Patients with CPPD disease are desperate for an effective treatment and trials testing ENPP1 inhibitors in CPPD disease would be of great interest." Dr Merriman said the researchers were "thrilled" about the potential impact of their discovery and the possibility of new drugs being developed for the treatment of CPPD disease. "The findings of this study produced a 'eureka moment', which can be rare in a scientist's career."
Yahoo
20-05-2025
- Business
- Yahoo
BioMarin Offers to Buy Inozyme for $270M to Boost Enzyme Therapy Biz
BioMarin Pharmaceutical BMRN announced that it has entered into a definitive agreement to acquire all outstanding shares of the clinical-stage company Inozyme Pharma INZY for $4.00 per share in cash, totaling nearly $270 million. The deal, approved by the board of directors of both companies, is expected to be closed in the third quarter of 2025. Following the completion of this acquisition, BioMarin will add Inozyme's lead asset, INZ-701, to its pipeline. This investigational enzyme replacement therapy (ERT) is currently being evaluated in a pivotal late-stage study for the treatment of a rare genetic disorder, ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) deficiency, in children. This condition affects blood vessels, soft tissues and bones, and is associated with increased cardiovascular mortality risk. Interim results from this late-stage study are expected in early 2026. If this data is positive, a regulatory approval for INZ-701 is expected in 2027, making it the first-in-disease treatment for this indication. Inozyme is also evaluating the ERT in a pivotal study for infants with ENPP1 and plans to initiate a supportive study for adolescents and adults with the disease. Inozyme is also exploring the potential of INZ-701 in separate clinical studies for two more rare disorders, namely ABCC6 Deficiency and calciphylaxis. Like ENPP1, there are currently no approved therapies for either of these disorders. Following this news on Friday, shares of Inozyme surged 178% and those of BioMarin gained about 2%. Year to date, shares of BMRN have lost 10%, while those of INZY have risen 43%. During the same timeframe, the industry has declined 7%. Image Source: Zacks Investment Research In our opinion, the deal is a strategic fit for BioMarin, which already markets five first-in-disease enzyme therapies, namely Aldurazyme, Brineura, Naglazyme, Palynziq and Vimizim. The addition of Inozyme's lead candidate will expand BioMarin's Enzyme Therapies portfolio and further diversify its revenue stream. The deal also benefits INZY as it lacks the commercial infrastructure and expertise needed to bring a drug to market, areas where BioMarin is already well established. BioMarin has reaffirmed its full-year 2025 guidance of $3.1-$3.2 billion for sales and $4.20-$4.40 for adjusted EPS. BioMarin currently carries a Zacks Rank #3 (Hold). BioMarin Pharmaceutical Inc. price | BioMarin Pharmaceutical Inc. Quote A couple of better-ranked stocks from the industry are Adaptive Biotechnologies ADPT and Agenus AGEN, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. In the past 60 days, estimates for Adaptive Biotechnologies' 2025 loss per share have improved from 92 to 87 cents. During the same timeframe, estimates for 2026 loss per share have narrowed from 69 to 65 cents. Adaptive Biotechnologies' earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 21.38%. Shares of ADPT have surged 51% year to date. Estimates for Agenus' 2025 loss per share have narrowed from $7.05 to $2.78 over the past 60 days, and the same for 2026 loss has improved from $7.14 to $5.26. Agenus' 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 22.71%. Year to date, its shares have gained 27%. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report BioMarin Pharmaceutical Inc. (BMRN) : Free Stock Analysis Report Agenus Inc. (AGEN) : Free Stock Analysis Report Adaptive Biotechnologies Corporation (ADPT) : Free Stock Analysis Report Inozyme Pharma, Inc. (INZY) : Free Stock Analysis Report This article originally published on Zacks Investment Research ( Zacks Investment Research Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
19-05-2025
- Business
- Yahoo
BioMarin agrees to acquire Inozyme for $270m
BioMarin Pharmaceutical has agreed to acquire Inozyme Pharma for $4.00 per share in a $270m all-cash transaction. This will enhance BioMarin's enzyme therapy portfolio by adding a late-stage enzyme replacement therapy, INZ-701. Unanimously approved by both companies' boards, the transaction will close in the third quarter of 2025, subject to regulatory clearance, tender offer completion and other customary closing conditions. INZ-701 is being evaluated to treat ectonucleotide pyrophosphatase/ phosphodiesterase 1 (ENPP1) deficiency, a rare genetic condition that impacts blood vessels, bones and soft tissues. This leads to increased cardiovascular mortality risk, particularly in infants, and severe rickets and osteomalacia in the paediatric and adult population. The first Phase III pivotal trial data for the therapy in children is anticipated in early 2026. Regulatory approval is expected in 2027. BioMarin will commence a cash tender offer to purchase all outstanding shares of Inozyme common stock. The board of directors of Inozyme has unanimously recommended that its stockholders tender their shares. The tender offer's completion is contingent upon standard closing conditions, including a majority of the outstanding Inozyme shares being tendered, the Hart-Scott-Rodino Antitrust Improvements Act waiting period expiry or termination, and other customary conditions. After the tender offer, a subsidiary of BioMarin will merge with Inozyme, and the remaining shares not tendered will be converted into the right to obtain the same $4.00 per share in cash. The acquisition is not dependent on financing. Goldman Sachs & Co is the exclusive financial advisor to BioMarin, with Cooley as legal counsel. Centerview Partners is Inozyme's exclusive financial advisor, and Goodwin Procter provides legal counsel. BioMarin Pharmaceutical CEO and president Alexander Hardy stated: "This acquisition brings to BioMarin an important medicine that has the potential to be the first treatment for children and adults with ENPP1 deficiency, improving care for people living with this serious condition. 'As BioMarin continues our transformation and delivers on our corporate strategy, we will continue to evaluate external innovation alongside internal innovation.' In 2024, the US Food and Drug Administration expanded the approval of BioMarin's supplemental biologics licence application (sBLA) for Brineura, an enzyme replacement therapy, to include children under three years of age with neuronal ceroid lipofuscinosis type 2 (CLN2) disease. "BioMarin agrees to acquire Inozyme for $270m" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
Yahoo
19-05-2025
- Business
- Yahoo
BioMarin Expands Rare Disease Portfolio with $270M Inozyme Acquisition
On May 16, BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced a $270 million acquisition of Inozyme Pharma, aiming to expand its rare disease research. The all-cash deal, backed by both companies' boards, is expected to close between July and September, giving BioMarin control of INZ-701, an enzyme replacement therapy targeting ENPP1 and ABCC6 deficiencies, life-threatening disorders caused by mineral buildup in soft tissues. A close-up of a hand holding a vial of biopharmaceutical drugs ready to be administered. With no FDA-approved treatments, INZ-701 aims to restore inorganic pyrophosphate levels, preventing tissue calcification. Late-stage trial results for ENPP1 deficiency are expected next year, with potential FDA approval by 2027. Analysts view the deal as a strategic expansion, complementing BioMarin Pharmaceutical Inc.'s (NASDAQ:BMRN) existing enzyme replacement therapies, including Vimizim, Naglazyme, and Palynziq. Despite a rare patient pool of around 10,000 globally, experts see commercial potential, expecting the acquisition to strengthen BioMarin's pipeline development. While some analysts praise its strong strategic fit, others believe it won't significantly impact BioMarin's overall market position. While we acknowledge the potential of BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) as an investment, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than BMRN and that has 100x upside potential, check out our report about the . READ NEXT: 20 Best AI Stocks To Buy Now and 30 Best Stocks to Buy Now According to Billionaires. Disclosure: None. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
16-05-2025
- Business
- Yahoo
BioMarin Strengthens Enzyme Therapy Business with Acquisition of Inozyme Pharma
Acquisition is Strong Strategic Fit for BioMarin, Adding INZ-701, a Phase 3 Enzyme Replacement Therapy Being Developed for Treatment of ENPP1 Deficiency First Pivotal Data Readout in Children Expected in Early 2026 with Potential Launch in 2027; Additional Clinical Programs to Expand to Patients of all Ages Potential First-in-Disease Treatment for ENPP1 Deficiency Conference Call and Webcast Scheduled Today at 8:45 a.m. ET SAN RAFAEL, Calif. and BOSTON, May 16, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) and Inozyme Pharma, Inc. (Nasdaq: INZY) announced today that BioMarin has entered into a definitive agreement to acquire Inozyme for $4.00 per share in an all-cash transaction for a total consideration of approximately $270 million. The transaction has been unanimously approved by the Boards of Directors of both companies and is expected to close in the third quarter of 2025, subject to regulatory approval, successful completion of a tender offer and other customary closing conditions. The acquisition will strengthen BioMarin's enzyme therapies portfolio, adding a late-stage enzyme replacement therapy, INZ-701, which is currently being assessed for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) Deficiency, a rare, serious and progressive genetic condition that affects blood vessels, soft tissues and bones. The condition is associated with increased cardiovascular mortality risk across all age groups, especially in infants. It is also associated with severe rickets and osteomalacia in children and adults. Data from the first Phase 3 pivotal study of INZ-701 in children is expected in early 2026, with potential regulatory approval in 2027. "BioMarin has been deeply committed to advancing enzyme therapies for children and adults living with serious genetic conditions for more than 25 years, and today's agreement builds on our legacy," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "This acquisition brings to BioMarin an important medicine that has the potential to be the first treatment for children and adults with ENPP1 Deficiency, improving care for people living with this serious condition. As BioMarin continues our transformation and delivers on our corporate strategy, we will continue to evaluate external innovation alongside internal innovation. We are in a strong financial position to bring in additional assets as we accelerate the development of medicines for patients with significant unmet need." "Today's announcement gives greater hope to patients who may benefit from INZ-701, a potentially transformative therapy that aims to address the underlying causes and systemic impacts of ENPP1 Deficiency," said Douglas A. Treco, Ph.D., Chief Executive Officer and Chairman of Inozyme. "BioMarin has paved the way over the past two and a half decades, successfully launching five first-in-disease enzyme therapies. I'd like to thank the team at Inozyme and our partners for their outstanding work and dedication, as we pass this important potentially life-changing therapy to the leading innovator in genetically defined conditions." INZ-701 and ENPP1 DeficiencyINZ-701 is a potential first-in-class, subcutaneous enzyme replacement therapy that is being developed for infants, pediatric and adult patients with ENPP1 Deficiency across a continuum of phenotypes. In addition to the ongoing Phase 3 pivotal study in children, Inozyme is currently enrolling infants in a pivotal study, and a supportive study for adolescents and adults is being planned. In a Phase 1/2 study of adults living with ENPP1 Deficiency, INZ-701 demonstrated a favorable safety profile, with no serious adverse events attributed to INZ-701. Improvements in pyrophosphate levels, bone mineralization biomarkers and quality of life were all observed, suggesting prospect for benefit in patients. ENPP1 Deficiency is a lifelong, rare, progressive, multisystemic condition, caused by mutations in the ENPP1 gene, leading to loss of ENPP1 enzymatic activity that results in low pyrophosphate, upregulation of fibroblast growth factor-23 and phosphate wasting. The condition affects blood vessels, soft tissues and bones. It is associated with high risk of cardiovascular mortality in patients of all ages, especially infants. It is also associated with severe rickets and osteomalacia in children and adults. Patients require considerable multidisciplinary lifelong medical and surgical management of complications. Currently there are no approved therapies for ENPP1 Deficiency. Terms of the Transaction Under the terms of the merger agreement, BioMarin will promptly commence a cash tender offer to acquire all of the outstanding shares of Inozyme common stock at a price of $4.00 per share. Inozyme's Board of Directors unanimously recommends that Inozyme's stockholders tender their shares in the tender offer. The consummation of the tender offer is subject to customary closing conditions, including the tender of at least a majority of the outstanding shares of Inozyme, the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, and other customary conditions. Following the successful completion of the tender offer, a wholly owned subsidiary of BioMarin will merge with Inozyme and the outstanding Inozyme shares not tendered in the tender offer will be converted into the right to receive the same $4.00 per share in cash paid in the tender offer. The transaction is not subject to any financing condition. BioMarin also today reaffirmed previously provided full-year 2025 financial guidance, excluding the impact of the accounting treatment of this transaction in accordance with Generally Accepted Accounting Principles (GAAP) upon closing, as well as its plan to achieve 40% Non-GAAP Operating Margin in 2026. AdvisorsGoldman Sachs & Co. LLC is acting as exclusive financial advisor to BioMarin, and Cooley LLP is serving as legal counsel. Centerview Partners LLC is acting as exclusive financial advisor to Inozyme, and Goodwin Procter LLP is serving as legal counsel. Conference CallBioMarin will host a conference call and webcast to discuss the acquisition today, May 16, 2025, at 8:45 a.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at U.S./Canada Dial-in Number: 888-596-4144 Replay Dial-in Number: 800-770-2030 International Dial-in Number: 646-968-2525 Replay International Dial-in Number: 609-800-9909 Conference ID: 2239224 Conference ID: 2239224 About BioMarinBioMarin is a global biotechnology company dedicated to translating the promise of genetic discovery into medicines that make a profound impact on the life of each patient. The San Rafael, California-based company, founded in 1997, has a proven track record of innovation with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that offer new possibilities for people living with genetically defined conditions around the world. To learn more, please visit About Inozyme Inozyme Pharma is a clinical-stage biopharmaceutical company, with approximately 50 employees based in Boston. The company is dedicated to developing innovative therapeutics that target the PPi-Adenosine Pathway, a key regulator of bone health and blood vessel function. Disruptions in this pathway underlie a range of severe diseases, including ENPP1 Deficiency. Our lead investigational therapy, INZ-701, is an ENPP1 Fc fusion protein enzyme replacement therapy designed to restore pyrophosphate and adenosine levels. INZ-701 is currently in late-stage clinical development in ENPP1 Deficiency, with the potential to expand into additional indications where deficiencies in the Pyrophosphate-Adenosine Pathway contribute to disease pathology, including ABCC6 Deficiency and calciphylaxis. Through our pioneering work, we aim to transform treatment options for patients affected by these devastating conditions. To learn more, please visit Forward-Looking Non-GAAP Financial Information As described above, today BioMarin reaffirmed its plan to achieve 40% Non-GAAP Operating Margin in 2026. BioMarin does not provide guidance for GAAP reported financial measures (other than revenue) or a reconciliation of forward-looking Non-GAAP financial measures to the most directly comparable GAAP reported financial measures because BioMarin is unable to predict with reasonable certainty the financial impact of changes resulting from its strategic portfolio and business operating model reviews; potential future asset impairments; gains and losses on investments; and other unusual gains and losses without unreasonable effort. These items are uncertain, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. As such, any reconciliations provided would imply a degree of precision that could be confusing or misleading to investors. Non-GAAP InformationNon-GAAP Operating Margin percentage is defined by BioMarin as GAAP Income from Operations, excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items, divided by GAAP Total Revenues. BioMarin regularly uses both GAAP and Non-GAAP results and expectations internally to assess its financial operating performance and evaluate key business decisions related to its principal business activities: the discovery, development, manufacturing, marketing and sale of innovative biologic therapies. Because Non-GAAP Operating Margin percentage is an important internal measurement for BioMarin, BioMarin believes that providing this information in conjunction with BioMarin's GAAP information enhances investors' and analysts' ability to meaningfully compare BioMarin's results from period to period and to its forward-looking guidance, and to identify operating trends in BioMarin's principal business. Non-GAAP financial measures are not meant to be considered in isolation or as a substitute for, or superior to comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP. Investors should note that the Non-GAAP information is not prepared under any comprehensive set of accounting rules or principles and does not reflect all of the amounts associated with BioMarin's results of operations as determined in accordance with GAAP. Investors should also note that these Non-GAAP financial measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future there may be other items that BioMarin may exclude for purposes of its Non-GAAP financial measures; likewise, BioMarin may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP financial measures. Because of the non-standardized definitions, the Non-GAAP financial measure as used by BioMarin in this press release may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies. Forward-Looking StatementsThis press release and the associated conference call contain forward-looking statements about, among other things, the proposed acquisition of Inozyme Pharma, Inc. (Inozyme) by BioMarin Pharmaceutical Inc. (BioMarin) and the business prospects of Inozyme and BioMarin, including, without limitation, statements about: the anticipated occurrence, manner and timing of the proposed tender offer and the closing of the proposed acquisition; the prospective benefits of the proposed acquisition, including expectations that it will strengthen BioMarin's enzyme therapies portfolio and be a strong strategic fit for BioMarin; Inozyme's product candidate INZ-701 and expectations regarding its ongoing development, including the potential for INZ-701 to be the first treatment for children and adults with ENPP1 Deficiency, the potential benefits of INZ-701 for patients, the anticipated timing for data from the first Phase 3 pivotal study of INZ-701 in children, the anticipated costs of developing INZ-701 and the potential regulatory approval of INZ-701 in 2027; potential revenue for INZ-701; additional INZ-701 clinical programs intended to expand to patients of all ages; the anticipated market for INZ-701; plans for an INZ-701 pivotal study for adolescents and adults; INZ-701's potential to expand into additional indications where deficiencies in the Pyrophosphate-Adenosine Pathway contribute to disease pathology; the accounting treatment of the potential acquisition under GAAP and its potential impact on BioMarin's financial results and financial guidance; BioMarin's plans for external innovation, including BioMarin being in a strong financial position to acquire additional assets; BioMarin's ability to execute additional transactions in future quarters; statements about BioMarin's future financial performance, including the expectations of Non-GAAP Operating Margin percentage; and other statements that are not historical facts. Actual results could differ materially from those anticipated in these forward-looking statements. Except as required by law, each of BioMarin and Inozyme assume no obligation to update these forward-looking statements, whether as a result of new information, future events or otherwise. These statements, which represent each of BioMarin's and Inozyme's current expectations or beliefs concerning various future events that are subject to significant risks and uncertainties, may contain words such as "may," "will," "would," "could," "expect," "anticipate," "intend," "plan," "believe," "estimate," "project," "seek," "should," "strategy," "future," "opportunity," "potential" or other similar words and expressions indicating future results. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. Forward-looking statements reflect current beliefs and expectations; however, these statements involve inherent risks and uncertainties, including, without limitation, with respect to: consummating the proposed acquisition in the anticipated timeframe, if at all; how many of Inozyme's stockholders will tender their stock in the tender offer; the possibility that competing offers or acquisition proposals will be made; the possibility that various closing conditions for the transaction may not be satisfied or waived, including that a governmental entity may prohibit, delay, or refuse to grant approval for the consummation of the transaction (or only grant approval subject to adverse conditions or limitations); the difficulty of predicting the timing or outcome of regulatory approvals or actions, if any; the effects of the proposed acquisition (or the announcement thereof) on Inozyme's or BioMarin's stock price and/or BioMarin's or Inozyme's operating results; unknown or inestimable liabilities; the development, launch and commercialization of products and product candidates such as INZ-701, if approved; the successful completion of regulatory activities with respect to INZ-701; the parties' ability to realize the anticipated benefits of the proposed acquisition, including the possibility that the expected benefits from the proposed acquisition will not be realized or will not be realized within the expected time period and that BioMarin and Inozyme will not be integrated successfully or that such integration may be more difficult, time-consuming or costly than expected; obtaining and maintaining adequate coverage and reimbursement for BioMarin's or Inozyme's products; the time-consuming and uncertain regulatory approval process; the costly and time-consuming pharmaceutical product development process and the uncertainty of clinical success, including risks related to failure or delays in successfully initiating or completing clinical trials and assessing patients, including with respect to current and planned future clinical trials of INZ-701; global economic, financial, and healthcare system disruptions and the current and potential future negative impacts to BioMarin's or Inozyme's business operations and financial results; the sufficiency of BioMarin's or Inozyme's cash flows and capital resources; BioMarin's ability to fund the acquisition with existing cash and investments; BioMarin's evaluation of the accounting treatment of the potential acquisition and its potential impact on its financial results and financial guidance; BioMarin's or Inozyme's ability to achieve targeted or expected future financial performance and results and the uncertainty of future tax, accounting and other provisions and estimates; the effects of the transaction on relationships with key third parties, including employees, customers, suppliers, other business partners or governmental entities, including the risk that the proposed acquisition adversely affects employee retention; transaction costs; risks that the proposed acquisition disrupts current plans and operations; risks that the proposed transaction diverts management's attention from ongoing business operations; changes in Inozyme's business during the period between announcement and closing of the proposed acquisition; any legal proceedings and/or regulatory actions that may be instituted related to the proposed acquisition; and other risks and uncertainties affecting BioMarin and Inozyme, including those risk factors detailed in BioMarin's and Inozyme's filings with the Securities and Exchange Commission (SEC), including, without limitation, the risk factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the fiscal quarter ended March 31, 2025 and Inozyme's Quarterly Report on Form 10-Q for the fiscal quarter ended March 31, 2025, as such risk factors may be updated by any subsequent reports, as well as the Tender Offer Statement on Schedule TO and related tender offer documents to be filed by BioMarin and its acquisition subsidiary, Incline Merger Sub, Inc., and the Solicitation/Recommendation Statement on Schedule 14D-9 to be filed by Inozyme. Stockholders of BioMarin and Inozyme are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin and Inozyme are under no obligation, and expressly disclaim any obligation, to update (publicly or otherwise) or alter any forward-looking statement, including without limitation any financial projection or guidance, whether as a result of new information, future events or otherwise. BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc. or its affiliates. Inozyme® is a registered trademark of Inozyme Pharma Inc. or its affiliates. Additional Information about the Acquisition and Where to Find ItThe tender offer for all of the outstanding shares of Inozyme described in this communication has not yet commenced. This communication is for informational purposes only and is neither an offer to purchase nor a solicitation of an offer to sell any securities, nor is it a substitute for the tender offer materials that Inozyme, BioMarin or its acquisition subsidiary will file with the SEC upon commencement of the tender offer. The solicitation and offer to tender and the offer to buy outstanding shares of Inozyme will only be made pursuant to a tender offer statement on Schedule TO, including an Offer to Purchase and related tender offer materials that BioMarin and its acquisition subsidiary, Incline Merger Sub, Inc., are expected to file with the SEC. At the time the tender offer is commenced, BioMarin and its acquisition subsidiary will file a Tender Offer Statement on Schedule TO, and Inozyme will file a Solicitation/Recommendation Statement on Schedule 14D-9 with the SEC with respect to the tender offer. THE TENDER OFFER MATERIALS (INCLUDING AN OFFER TO PURCHASE, A RELATED LETTER OF TRANSMITTAL AND CERTAIN OTHER TENDER OFFER DOCUMENTS), AS WELL AS THE SOLICITATION/RECOMMENDATION STATEMENT ON SCHEDULE 14D-9, WILL CONTAIN IMPORTANT INFORMATION ABOUT THE PROPOSED ACQUISITION AND THE PARTIES THERETO. INVESTORS AND STOCKHOLDERS OF INOZYME ARE URGED TO READ THESE DOCUMENTS CAREFULLY WHEN THEY BECOME AVAILABLE (AND EACH AS IT MAY BE AMENDED OR SUPPLEMENTED FROM TIME TO TIME) BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION THAT INVESTORS AND STOCKHOLDERS OF INOZYME SHOULD CONSIDER BEFORE MAKING ANY DECISION WITH RESPECT TO THE TENDER OFFER. The tender offer materials (including the Offer to Purchase, the related Letter of Transmittal and certain other tender offer documents), as well as the Solicitation/Recommendation Statement, will be made available to all investors and stockholders of Inozyme at no expense to them at SEC's website at Copies of the documents filed with the SEC by BioMarin will be available free of charge on BioMarin's website at Copies of the documents filed with the SEC by Inozyme will be available free of charge on Inozyme's website, or by contacting Inozyme's investor relations department at investorrelations@ The information contained in, or that can be accessed through, BioMarin's and Inozyme's websites is not a part of, or incorporated by reference herein. In addition to the Offer to Purchase, related Letter of Transmittal and certain other tender offer documents, and Solicitation/Recommendation Statement, BioMarin and Inozyme file annual, quarterly, and current reports, proxy statements and other information with the SEC. You may read any reports, statements or other information filed by BioMarin and Inozyme with the SEC for free on the SEC's website at BioMarin Contacts Traci McCarty, Investors BioMarin Pharmaceutical Inc.(415) 455-7558 Marni Kottle, MediaBioMarin Pharmaceutical Inc.(415) 218-7111 Inozyme Contacts Stefan Riley, Investors Inozyme Pharma, Inc. (617) 461-2442 Todd Cooper, MediaBiongage Communications(617) 840-1637 View original content to download multimedia: SOURCE BioMarin Pharmaceutical Inc.
