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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.
Associated Press
10-03-2025
- Business
- Associated Press
Inozyme Pharma Reports Full Year 2024 Financial Results and Announces Strategic Prioritization of ENPP1 Deficiency Pivotal Program and Recent Business Highlights
- Cash, cash equivalents and short-term investments as of December 31, 2024, and anticipated cost savings from prioritizing ENPP1 Deficiency program, including an approximately 25% workforce reduction, expected to support operations into the first quarter of 2026 - - Enrollment complete in ENERGY 3 pivotal trial in pediatric patients with ENPP1 Deficiency; topline data expected in first quarter of 2026 - - Positive interim results from ENERGY 1 trial and Expanded Access Program evaluating INZ-701 in infants and young children with ENPP1 Deficiency showed improvements from baseline in multiple measures of disease - BOSTON, March 10, 2025 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) ('the Company' or 'Inozyme'), a clinical-stage biopharmaceutical company developing innovative therapeutics for rare diseases that affect bone health and blood vessel function, today reported financial results for the full year ended December 31, 2024, and provided recent business highlights. '2024 marked a transformative year for Inozyme as we achieved critical milestones in our ENPP1 Deficiency program that enabled us to complete enrollment in our pivotal ENERGY 3 trial and announce promising interim data in infants and young children with ENPP1 Deficiency in January 2025,' said Douglas A. Treco, Ph.D., CEO and Chairman of Inozyme Pharma. 'We are concentrating our resources on advancing INZ-701 toward potential approval in ENPP1 Deficiency. We believe this focus will allow us to most efficiently progress our lead program while building a strong foundation for future growth.' Strategic Prioritization to Extend Cash Runway As part of its recent strategic review, the Company has prioritized activities to support the planned Biologics License Application (BLA) filing for INZ-701 for the Company's lead indication, ENPP1 Deficiency. Patients with ABCC6 Deficiency being treated in the Company's long-term extension study, the Company's expanded access program, or under investigator-sponsored INDs will continue to receive treatment. Future trials in ABCC6 Deficiency and calciphylaxis will be postponed. These prioritization measures, which include a workforce reduction of approximately 25% of employees implemented in the first quarter of 2025, with the Company's cash, cash equivalents and short-term investments as of December 31, 2024, are expected to extend the Company's cash runway into the first quarter of 2026. 'The strategic refocusing of our portfolio necessitated difficult decisions, including a reduction in our workforce. These organizational changes, while challenging, are essential to extend our operational runway and maximize our ability to advance INZ-701 as a potential treatment for patients with ENPP1 Deficiency,' continued Dr. Treco. 'I want to express my deepest gratitude to every member of the Inozyme team, both those who will continue our mission and those who will be departing. Their exceptional dedication, talent, and passion have advanced our science and strengthened our commitment to patients with rare diseases. Enrollment Complete in ENERGY 3 Pivotal Trial In January 2025, the Company completed enrollment in its ENERGY 3 pivotal trial of INZ-701 in pediatric patients with ENPP1 Deficiency between the ages of one and less than 13 years across sites globally. With 27 patients enrolled, the trial's 2:1 randomized design provides >90% power to detect meaningful differences in radiographic global impression of change (RGI-C) between treatment and control groups. The Company anticipates completing the one-year dosing period for all patients by January 2026, with topline data expected in first quarter of 2026. Positive Interim Data from the ENERGY 1 Trial and Expanded Access Program (EAP) The Company recently announced positive interim data from the ENERGY 1 trial, involving three infants and the EAP, involving two infants and one 2.5-year-old child, which evaluated patients with generalized arterial calcification of infancy (GACI), a severe manifestation of ENPP1 Deficiency. Patients were treated with INZ-701 for periods of three weeks to 22 months. The data presentation can be accessed here on Inozyme's Investor Relations site. Financial Results for the Year Ended December 31, 2024 Cash Position and Financial Guidance – Cash, cash equivalents, and short-term investments were $113.1 million as of December 31, 2024. Based on its current plans and including the recent strategic prioritization, the Company anticipates its cash, cash equivalents, and short-term investments as of December 31, 2024, will enable the Company to fund cash flow requirements into the first quarter of 2026. Research and Development (R&D) Expenses – R&D expenses were $83.2 million for the year ended December 31, 2024, compared to $54.8 million for the year ended December 31, 2023. The increase of $28.4 million was driven by $14.9 million in clinical development and related consulting costs to support our ongoing clinical trials, $10.1 million in chemistry, manufacturing, and controls expense to support our ongoing clinical trials and prepare for potential commercialization, $3.9 million in personnel-related costs, including stock-based compensation expense, offset by a $0.5 million decrease in facilities administrative expense. General and Administrative (G&A) Expenses – G&A expenses were $20.8 million for the year ended December 31, 2024, remaining consistent with the $20.8 million reported for the year ended December 31, 2023. Net Loss – Net loss was $102.0 million, or $1.62 loss per share, for the year ended December 31, 2024, compared to $71.2 million, or $1.37 loss per share, for the year ended December 31, 2023. About ENPP1 Deficiency ENPP1 Deficiency is a serious and progressive rare disease that affects blood vessels, soft tissues, and bones. Individuals who present in utero or in infancy are typically diagnosed with generalized arterial calcification of infancy (GACI Type 1), with about 50% of these infants not surviving beyond six months. Children with this condition typically develop autosomal-recessive hypophosphatemic rickets type 2 (ARHR2), while adolescents and adults may develop osteomalacia, or softened bones. ARHR2 and osteomalacia cause pain and difficulty with movement. Additionally, patients may experience hearing loss, calcification in arteries and joints, and heart problems. ENPP1 Deficiency is an autosomal recessive disease and biallelic mutations are estimated to occur in approximately 1 in 64,000 pregnancies worldwide. Many individuals with just one copy of the mutated gene (monoallelic ENPP1 Deficiency) exhibit severe symptoms, suggesting that the worldwide prevalence of ENPP1 Deficiency may be much higher than current estimates. Currently, there are no approved therapies for ENPP1 Deficiency. About Inozyme Pharma Inozyme Pharma is a clinical-stage biopharmaceutical company 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 (ERT) designed to restore PPi 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 PPi-Adenosine Pathway contribute to disease pathology. Through our pioneering work, we aim to transform treatment options for patients affected by these devastating conditions. For more information, please visit or follow Inozyme on LinkedIn, X, and Facebook. Condensed Consolidated Balance Sheet (in thousands) December 31, 2024 December 31, 2023 Cash, cash equivalents and investments $ 113,087 $ 188,289 Total assets 123,182 200,847 Total liabilities 65,356 60,368 Additional paid-in-capital 445,705 426,362 Accumulated deficit (387,954) (285,930) Total stockholders' equity 57,826 140,479 Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands, except share and per share data) Year Ended December 31, 2024 2023 Operating expenses: Research and development $ 83,231 $ 54,847 General and administrative 20,799 20,798 Total operating expenses 104,030 75,645 Loss from operations (104,030) (75,645) Other income (expense): Interest income 7,666 7,837 Interest expense (5,558) (3,333) Other expense, net (102) (28) Other income, net 2,006 4,476 Net loss $ (102,024) $ (71,169) Other comprehensive income (loss): Unrealized gains on available-for-sale securities 24 264 Foreign currency translation adjustment 4 (18) Total other comprehensive income 28 246 Comprehensive loss $ (101,996) $ (70,923) Net loss attributable to common stockholders—basic and diluted $ (102,024) $ (71,169) Net loss per share attributable to common stockholders—basic and diluted $ (1.62) $ (1.37) Weighted-average common shares outstanding—basic and diluted 62,811,814 51,839,131 Cautionary Note Regarding Forward-Looking Statements Statements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute 'forward-looking statements' within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation, timing, and design of our planned clinical trials, enrollment and availability of data from clinical trials, the potential benefits of INZ-701, our regulatory strategy and our strategic prioritization and the period over which we believe that our existing cash, cash equivalents and short-term investments will be sufficient to fund our cash flow requirements. The words 'anticipate,' 'believe,' 'continue,' 'could,' 'estimate,' 'expect,' 'intend,' 'may,' 'plan,' 'potential,' 'predict,' 'project,' 'should,' 'target,' 'will,' 'would,' and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to realize the anticipated cost savings related to the strategic prioritization and workforce reduction; conduct its ongoing clinical trials of INZ-701 for ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis; enroll patients in ongoing and planned trials; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain, and protect intellectual property rights related to its product candidates; manage expenses; comply with covenants under its outstanding loan agreement; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the 'Risk Factors' section in the Company's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. Contacts Media: Biongage Communications Todd Cooper (617) 840-1637
