INZY Inozyme Pharma Inc.
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| Drug | Stage | Catalyst Date |
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INZ-701
ABCC6 deficiency
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Phase 1/2
Phase 1/2
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INZ-701
ENPP1 deficiency
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Phase 1/2
Phase 1/2
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Latest News
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- Natural History Study shows ectopic calcification and/or cardiovascular disease could serve as distinguishing characteristics of ENPP1 Deficiency in some children with rickets -
- Preclinical data support AAV-ENPP1 gene therapy as a potential treatment for ENPP1 Deficiency -
BOSTON, Oct. 04, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced the presentation of data from the Company's ENPP1 Deficiency Natural History Study and its gene therapy program for the treatment of ENPP1 Deficiency. The data were presented at the American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting held October 1-4.
"Results from these studies deepen our understanding of ENPP1 Deficiency and underscore the urgent need for novel interventions," said Axel Bolte, MSc, MBA, Inozyme's co-founder, president, and chief executive officer. "With conventional therapies for FGF23-mediated hypophosphatemia associated with worsening calcification, identifying distinguishing characteristics of ENPP1 Deficiency is imperative for ensuring quick and proper diagnosis and treatment of patients suffering from this disease. We firmly believe that our pipeline candidates hold strong potential to play a meaningful role in the treatment of multiple underserved populations, and we look forward to commencing the Phase 1/2 clinical trials of INZ-701 in ENPP1 Deficiency and ABCC6 Deficiency in the fourth quarter."
ENPP1 Deficiency is a progressive condition that manifests as a spectrum of disease. Those who present in utero or infancy are typically diagnosed with generalized arterial calcification of infancy (GACI), which is characterized by extensive vascular calcification and neointimal proliferation (overgrowth of smooth muscle cells inside blood vessels), resulting in myocardial infarction or cardiac or multiorgan failure. The condition is lethal in an estimated 50% of affected babies. Children and adults with ENPP1 Deficiency typically experience rickets and osteomalacia (softened bones), also termed autosomal-recessive hypophosphatemic rickets type 2 (ARHR2), and can exhibit a range of signs and symptoms that can include hearing loss, arterial calcification, cardiac, and neurological involvement. There are no approved treatments for ENPP1 Deficiency.
Summary of Data Presented
Title: ENPP1-Deficient Patients Present With Both Skeletal Complications and Ectopic Calcification
Lead Author: Frank Rutsch, M.D., Muenster University Children's Hospital, Münster, Germany- This cross-sectional, retrospective study assessed data from 74 patients with confirmed ENPP1 variants. A total of 46% of patients demonstrated skeletal disease (median age of initial reporting: 4.3 years), with an estimated 90% of all patients expected to develop skeletal complications by 25 years. Cardiac disease was reported in 58% of patients with skeletal complications, as well as high rates of calcification of the aorta (68%) and other arteries (71%). GACI was not reported in 25% (9/34) of patients with skeletal complications; however, some of these individuals reported arterial calcification (n=2) or cardiac complications [heart valve defect (n=4), cardiac failure (n=2)]. Thirty patients received conventional therapy with calcitriol and phosphate. Young ENPP1-deficient patients are at risk for skeletal complications over a broad age spectrum, warranting continued monitoring well into adolescence. As skeletal manifestations have a similar phenotype to other forms of FGF23-mediated hypophosphatemia, a history of calcification or cardiovascular disease should trigger consideration of ENPP1 Deficiency as the etiology. These data highlight implications for the appropriate diagnosis and treatment of patients with ENPP1 Deficiency.
Title: Treatment with an AAV vector expressing ENPP1-Fc prevents ectopic tissue calcification and restores bone parameters in Enpp1-deficient mice
Presenter: Yves Sabbagh, Ph.D., Inozyme Pharma- This dose-response study assessed the pharmacological effects of AAV-ENPP1-Fc, an adeno-associated virus vector expressing a modified human ENPP1-Fc under a tissue-specific promoter, when administered to murine mouse models of ENPP1 Deficiency. Clinically relevant endpoints, including plasma PPi levels, tissue calcium content assessed by a colorimetric assay, and bone parameters, were measured after 10 weeks. At 12 weeks of age, vehicle-treated mutant mice showed profound defects in long bones, including lower trabecular number and thickness and thinner cortical bone in femora, and clear signs of rickets in the growth plate. One single intravenous injection of AAV-ENPP1-Fc resulted in a robust and durable increase in plasma ENPP1 activity for the duration of the study. AAV-ENPP1-Fc administration also led to a dose-dependent increase in plasma PPi levels and prevention of soft tissue calcification. Treatment at high dose prevented pathological calcification in all the tested organs and restored bone parameters. Findings demonstrate the potential of AAV-ENPP1-Fc gene therapy to treat ENPP1 Deficiency.
About INZ-701
INZ-701 is an ENPP1 enzyme replacement therapy (ERT) in development for the treatment of mineralization disorders of the circulatory system, bones, and kidneys. In preclinical studies, the experimental therapy has shown potential to generate plasma pyrophosphate (PPi) and to restore it to appropriate physiological levels, thereby preventing calcification in the vasculature and kidneys, while at the same time correcting bone abnormalities. Inozyme is developing INZ-701 for certain rare, life-threatening, and devastating genetic disorders such as ENPP1 Deficiency and ABCC6 Deficiency in which PPi levels are below the normal physiological levels.
About Inozyme Pharma
Inozyme Pharma, Inc. (NASDAQ:INZY), is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies.
Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.
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 and timing of our clinical trials, the initiation and timing of our natural history study, our research and development programs, the availability of preclinical study and clinical trial data, the timing of our regulatory applications and the period over which we believe that our existing cash, cash equivalents and investments will be sufficient to fund our operating expenses. 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 initiate its planned Phase 1/2 clinical trials of INZ-701 for ENPP1 Deficiency and ABCC6 Deficiency; 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; 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
Investors:
Inozyme Pharma
Stefan Riley, Director of Investor Relations
stefan.riley@inozyme.comMedia:
SmithSolve
Alex Van Rees
(973) 442-1555 ext. 111
Alex.vanrees@smithsolve.com
- This cross-sectional, retrospective study assessed data from 74 patients with confirmed ENPP1 variants. A total of 46% of patients demonstrated skeletal disease (median age of initial reporting: 4.3 years), with an estimated 90% of all patients expected to develop skeletal complications by 25 years. Cardiac disease was reported in 58% of patients with skeletal complications, as well as high rates of calcification of the aorta (68%) and other arteries (71%). GACI was not reported in 25% (9/34) of patients with skeletal complications; however, some of these individuals reported arterial calcification (n=2) or cardiac complications [heart valve defect (n=4), cardiac failure (n=2)]. Thirty patients received conventional therapy with calcitriol and phosphate. Young ENPP1-deficient patients are at risk for skeletal complications over a broad age spectrum, warranting continued monitoring well into adolescence. As skeletal manifestations have a similar phenotype to other forms of FGF23-mediated hypophosphatemia, a history of calcification or cardiovascular disease should trigger consideration of ENPP1 Deficiency as the etiology. These data highlight implications for the appropriate diagnosis and treatment of patients with ENPP1 Deficiency.
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BOSTON, Sept. 22, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases of abnormal mineralization, today announced Axel Bolte, MSc, MBA, the company's co-founder, president, and chief executive officer, will present at the 2021 Cantor Virtual Global Healthcare Conference on Wednesday, September 29, at 1:20 p.m. ET.
The presentation will be webcast live and can be accessed from the Investors & News section of Inozyme's website under Events. An archived replay of the webcast will be available for up to 60 days following the event.
About Inozyme Pharma
Inozyme Pharma, Inc. (NASDAQ:INZY), is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies.Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.
Contacts
Investors:
Inozyme Pharma
Stefan Riley, Director of Investor Relations
stefan.riley@inozyme.comMedia:
SmithSolve
Alex Van Rees
(973) 442-1555 ext. 111
alex.vanrees@smithsolve.com
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- Received Orphan Drug Designation from the European Medicines Agency for INZ-701 for the treatment of ABCC6 Deficiency –
– Clinical Trial Application in Europe accepted for Phase 1/2 trial of INZ-701 in ABCC6 Deficiency –
– Investigational New Drug application in U.S. accepted for Phase 1/2 trial of INZ-701 in ABCC6 Deficiency –
- Expect to enroll patients in Phase 1/2 clinical trials in ENPP1 Deficiency and ABCC6 Deficiency in Q4 2021 and report preliminary biomarker and safety data in the first half of 2022 –
- Cash, cash equivalents, and investments expected to support continued operations into the fourth quarter of 2022 –
BOSTON, Aug. 11, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today reported financial results for the second quarter ended June 30, 2021, and provided recent business highlights.
"We achieved several important milestones during the second quarter of 2021. We expect to initiate our clinical trials in ENPP1 Deficiency and ABCC6 Deficiency in the fourth quarter of this year and then to report preliminary biomarker and safety data in the first half of 2022," said Axel Bolte, MSc, MBA, Inozyme's co-founder, president, and chief executive officer. "We continue to make progress towards our mission of bringing hope to patients and their families who are living with ENPP1 Deficiency and ABCC6 Deficiency. In addition, we added several talented members to our leadership team who will contribute to this next phase of our growth."
Recent Business Highlights
- Clinical Trial Application (CTA) in Europe and Investigational New Drug (IND) application in U.S. for ABCC6 Deficiency accepted – The Company expects to enroll patients in a Phase 1/2 clinical trial in the fourth quarter of 2021 and report preliminary biomarker and safety data in the first half of 2022.
- Published data from Natural History Study of ENPP1 and ABCC6 Deficiencies – Peer-reviewed article in the Journal of Bone and Mineral Research shows early mortality risk in GACI patients despite attempts to treat with bisphosphonates, high prevalence of rickets almost exclusive to ENPP1 Deficiency, and a spectrum of heterogenous calcification and multiple organ complications with both ENPP1 and ABCC6 variants, which suggests an overlapping pathology.
- Appointed Gayle Gironda as senior vice president, human resources – Ms. Gironda is a human resources leader with more than 20 years of experience in organizational design, talent recruitment, performance culture, planning and leadership development.
- Appointed David Thompson, M.A., M.S., Ph.D. as chief development officer – Dr. Thompson has more than 30 years of experience designing and leading research and development programs focused on bone disorders and phosphate regulation. He previously served as Inozyme's senior vice president and chief scientific officer from 2018 to 2020 and was responsible for scientific research as the Company built its proprietary pipeline of investigational therapies, beginning with INZ-701.
Upcoming Anticipated Milestones
The Company also announced the following anticipated milestones for the INZ-701 clinical development program, subject to COVID-19-related restrictions:
- ENPP1 Deficiency
- Q4 2021: Start enrollment for Phase 1/2 clinical trial
- Q1 2022: Initiate prospective natural history study
- H1 2022: Report preliminary safety and biomarker data from Phase 1/2 clinical trial
- ABCC6 Deficiency
- Q4 2021: Start enrollment for Phase 1/2 clinical trial
- H1 2022: Report preliminary safety and biomarker data from Phase 1/2 clinical trial
Financial Results for the Quarter Ended Jun 30, 2021
- Cash Position and Financial Guidance – Cash, cash equivalents, and investments were $137.5 million as of June 30, 2021. Based on its current plans, the Company expects that its existing cash, cash equivalents, and investments will be sufficient to enable funding of its operating expenses and capital expenditure requirements into the fourth quarter of 2022.
- Research and Development (R&D) Expenses – R&D expenses were $8.2 million for the quarter ended June 30, 2021, compared to $7.9 million for the quarter ended June 30, 2020. The increase was primarily due to increased salaries, employee-related costs, and stock-based compensation expense due to the growth in the number of R&D employees. These increases were partially offset by a decrease in preclinical toxicology studies in support of our IND filing for INZ-701, and lower manufacturing costs based on the timing of production runs.
- General and Administrative (G&A) Expenses – G&A expenses were $4.4 million for the quarter ended June 30, 2021, compared to $1.7 million for the quarter ended June 30, 2020. The increase was primarily due to the growth in the number of G&A employees, an increase in legal fees related to new contracts and operations as a public company and generally higher fees in areas such as audit, tax, and information technology to support the Company's growth.
- Net Loss – Net loss was $12.5 million, or $0.53 loss per share, for the quarter ended June 30, 2021, compared to $9.5 million, or $7.57 loss per share, for the quarter ended June 30, 2020.
About Inozyme Pharma
Inozyme Pharma, Inc. (NASDAQ:INZY), is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies.
Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.
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 and timing of our clinical trials, the initiation and timing of our natural history study, our research and development programs, the availability of preclinical study and clinical trial data, the timing of our regulatory applications and the period over which we believe that our existing cash, cash equivalents and investments will be sufficient to fund our operating expenses. 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 initiate its planned Phase 1/2 clinical trials of INZ-701 for ENPP1 Deficiency and ABCC6 Deficiency; 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; 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.
Condensed Consolidated Balance Sheet Data
(Unaudited)(in thousands)
June 30,
2021December 31,
2020Cash, cash equivalents and investments $ 137,464 $ 159,896 Total assets 148,259 169,363 Total liabilities 9,985 11,260 Additional paid-in-capital 252,920 249,175 Accumulated deficit (114,666 ) (91,076 ) Total stockholders' equity 138,274 158,103 Condensed Consolidated Statements of Operations and Comprehensive Loss
(Unaudited)(in thousands, except share and per share data)
Three Months Ended June 30, 2021 2020 Operating expenses: Research and development $ 8,220 $ 7,877 General and administrative 4,435 1,671 Total operating expenses 12,655 9,548 Loss from operations (12,655 ) (9,548 ) Other income (expense): Interest income 58 71 Other income (expense) 57 4 Other income (expense), net 115 75 Net loss $ (12,540 ) $ (9,473 ) Other comprehensive income (loss): Unrealized gains (losses) on available-for-sale securities 6 (15 ) Total other comprehensive income (loss) 6 (15 ) Comprehensive loss $ (12,534 ) $ (9,488 ) Net loss attributable to common stockholders—basic and diluted $ (12,540 ) $ (9,473 ) Net loss per share attributable to common stockholders—basic and diluted $ (0.53 ) $ (7.57 ) Weighted-average common shares outstanding—basic and diluted 23,490,591 1,251,244 Six Months Ended June 30, 2021 2020 Operating expenses: Research and development $ 14,823 $ 14,283 General and administrative 8,804 3,171 Total operating expenses 23,627 17,454 Loss from operations (23,627 ) (17,454 ) Other income (expense): Interest income 121 242 Other income (expense) (84 ) 1 Other income (expense), net 37 243 Net loss $ (23,590 ) $ (17,211 ) Other comprehensive income (loss): Unrealized gains (losses) on available-for-sale securities 16 8 Total other comprehensive income (loss) 16 8 Comprehensive loss $ (23,574 ) $ (17,203 ) Net loss attributable to common stockholders—basic and diluted $ (23,590 ) $ (17,211 ) Net loss per share attributable to common stockholders—basic and diluted $ (1.01 ) $ (14.01 ) Weighted-average common shares outstanding—basic and diluted 23,460,218 1,228,296 Contacts
Investors:
Inozyme Pharma
Stefan Riley, Director of Investor Relations
Stefan.riley@inozyme.comMedia:
Alex Van Rees, SmithSolve
(973) 442-1555 ext. 111
Alex.vanrees@smithsolve.com
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BOSTON, Aug. 04, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced Axel Bolte, MSc, MBA, the company's co-founder, president, and chief executive officer, will participate in a panel at the 2021 Wedbush PacGrow Healthcare Virtual Conference on Wednesday, Aug. 11. Members of the Inozyme management team will also host investor meetings during the conference.
2021 Wedbush PacGrow Healthcare Virtual Conference
- Panel: UltraOrphan – When You're One in a Million
- Date: Wednesday, Aug. 11
- Time: 2:55-3:25 p.m. ET
About Inozyme Pharma
Inozyme Pharma (NASDAQ:INZY) is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies.Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.
Contacts
Investors:
Inozyme Pharma
Stefan Riley, Director of Investor Relations
Stefan.riley@inozyme.comMedia:
SmithSolve
Alex Van Rees
(973) 442-1555 ext. 111
Alex.vanrees@smithsolve.com -
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BOSTON, July 21, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to INZ-701 for the treatment of ABCC6 Deficiency. INZ-701, an investigational enzyme replacement therapy (ERT), was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and the EMA for the treatment of ENPP1 Deficiency.
ABCC6 Deficiency is a rare, inherited disorder that can present as generalized arterial calcification of infancy (GACI) type 2 in infants and as pseudoxanthoma elasticum (PXE) in children and adults. It is one of several disorders that features a significant decrease in plasma pyrophosphate (PPi) levels, a potent regulator of mineralization. In patients with ABCC6 Deficiency, the abnormal calcification caused by low PPi can result in vision loss and life-threatening cardiovascular complications, among other morbidities. There is no approved treatment for ABCC6 Deficiency.
"Obtaining Orphan Drug Designation from the EMA for the treatment of ABCC6 Deficiency marks another important step toward providing relief for patients who currently have no treatment options and positions INZ-701 to become the first-ever available therapy for this condition," said Axel Bolte, MSc, MA, co-founder, president, and chief executive officer of Inozyme.
Orphan Drug Designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products (COMP). To qualify, a therapeutic candidate must be intended to treat a serious condition that affects fewer than five in 10,000 people in the EU, and there must be sufficient data to suggest the candidate may produce clinically relevant outcomes. The designation could allow for drug development incentives, including clinical protocol assistance, access to the centralized authorization procedure, reduced regulatory fees, and a ten-year period of market exclusivity in the EU after product approval.
About INZ-701
INZ-701 is an ENPP1 enzyme replacement therapy (ERT) in development for the treatment of mineralization disorders of the circulatory system, bones, and kidneys. In preclinical studies, the experimental therapy has shown potential to generate plasma pyrophosphate (PPi) and to restore it to appropriate physiological levels, thereby preventing calcification in the vasculature and kidneys, while at the same time correcting bone abnormalities. Inozyme is developing INZ-701 for certain rare, life-threatening, and devastating genetic disorders such as ENPP1 Deficiency and ABCC6 Deficiency in which PPi levels are below the normal physiological levels.Inozyme is preparing to initiate a Phase 1/2 clinical trial in patients with ENPP1 Deficiency and a separate Phase 1/2 clinical trial in patients with ABCC6 Deficiency in mid-2021.
About ABCC6 Deficiency
ABCC6 Deficiency is a rare, severe, inherited disorder caused by mutations in the ABCC6 gene, leading to low levels of PPi. PPi is essential for preventing harmful soft tissue calcification and regulating bone mineralization. ABCC6 Deficiency is a systemic and progressively debilitating condition estimated to affect more than 67,000 individuals worldwide. The condition is characterized by pathological mineralization in blood vessels and soft tissues throughout the body that can drive devastating medical problems.Some infants with ABCC6 Deficiency are diagnosed with generalized arterial calcification of infancy (GACI) type 2, a vascular condition that resembles GACI type 1, the acute infantile form of ENPP1 Deficiency. In older patients, ABCC6 Deficiency presents as pseudoxanthoma elasticum (PXE), a rare, inherited disorder in which individuals develop calcification of soft connective tissues, including in the eyes, cardiovascular system, and skin. There is no approved treatment for ABCC6 Deficiency.
About Inozyme Pharma
Inozyme Pharma (NASDAQ:INZY) is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies.Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.
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 potential of our lead product candidate, INZ-701, the initiation and timing of our future clinical trials and our research and development programs. 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 initiate its planned Phase 1/2 clinical trials of INZ-701 for ENPP1 Deficiency and ABCC6 Deficiency; 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; 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, 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
Investors:
Inozyme Pharma
Stefan Riley, Director of Investor Relations
Stefan.riley@inozyme.comMedia:
SmithSolve
Alex Van Rees
(973) 442-1555 ext. 111
Alex.vanrees@smithsolve.com