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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
[email protected]Media:
SmithSolve
Alex Van Rees
(973) 442-1555 ext. 111
[email protected]
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BOSTON, July 13, 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 Gayle Gironda has been appointed as senior vice president of 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.
"Gayle brings a deep knowledge of life sciences, stemming from her work across large multinational pharmaceutical organizations and small biotech companies, and a passion for supporting people living with rare diseases," said Axel Bolte, MSc, MBA, co-founder, president and chief executive officer of Inozyme Pharma. "We are thrilled to welcome her to the team as we prepare to transition to a clinical-stage company with a commensurate expansion of our operational capabilities and growing talent pool. Gayle's experience will be essential as we position Inozyme for a new working environment while continuing to build our culture, which is focused on our patient communities and guides our mission of delivering first-in-class treatments for rare diseases without approved medicines."
Most recently, Ms. Gironda served as vice president, human resources, global hematology and global market access at Bristol Myers Squibb (BMS), playing a key role in integrating the Hematology Business Unit as part of the company's acquisition of Celgene. Additionally, she supported the Global Market Access, Pricing and Health Economics and Outcomes Research (HEOR) teams through post-acquisition strategy, organizational design and talent alignment.
Before joining Celgene in 2018 as vice president, human resources, global franchises, Ms. Gironda was executive director, HR, commercial operations at Alexion Pharmaceuticals. Her previous experience also includes leadership roles in human resources and operations for small-to-mid-sized companies such as Watson/Actavis, Jerini Ophthalmic, Inc. and Eyetech Pharmaceuticals.
"Inozyme's leadership is committed to a collaborative and inclusive culture in which the entire team is deeply connected to the goal of treating patients with rare diseases," Ms. Gironda said. "I'm proud to join the company during such a critical time and look forward to playing a role in its continued growth."
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
[email protected]Media:
SmithSolve
Alex Van Rees
(973) 442-1555 ext. 111
[email protected] -
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- Clinical trial initiation expected in mid-2021 –
- Preliminary safety and biomarker data expected by the end of 2021 -BOSTON, June 09, 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 acceptance of its Clinical Trial Application (CTA) from the National Agency for the Safety of Medicines and Health Products (ANSM) in France to allow initiation of its Phase 1/2 clinical trial of INZ-701, as a potential treatment for ABCC6 Deficiency. Inozyme plans to initiate its Phase 1/2 trial in mid-2021. This CTA was submitted and accepted as part of ANSM's Fast Track procedure designed to reduce processing times for clinical trial authorization requests for innovative medical products.
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. This is one of several disorders with 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.
"The acceptance of this CTA marks another important regulatory milestone for Inozyme and a key advancement for INZ-701 as a potential treatment for people living with ABCC6 Deficiency," said Axel Bolte, MSc, MBA, co-founder, president, and chief executive officer of Inozyme Pharma. "We are well-positioned to execute on our planned clinical study and the notable level of interest from the ABCC6 Deficiency community underscores the urgent need for therapeutic options. I want to express my thanks to the team at Inozyme and our external collaborators, all of whom have been instrumental in our continued progress."
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 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 in Enpp1-deficient mice. In Abcc6-deficient mice, subcutaneous administration of INZ-701 (2 and 10 mg/kg every other day for two or eight weeks) led to a dose-dependent increase in plasma PPi levels at both two and eight weeks after initiation of treatment, leading to significantly lower levels of soft tissue mineralization.
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 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 in the first half of 2021 and a separate Phase 1/2 clinical trial in patients with ABCC6 Deficiency in mid-2021.
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
[email protected]Media:
SmithSolve
Alex Van Rees
(973) 442-1555 ext. 111
[email protected] -
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– Filed Clinical Trial Application for INZ-701 for ABCC6 Deficiency in Europe; on track to initiate Phase 1/2 clinical trial in mid-2021 –
– Published peer-reviewed preclinical data supporting INZ-701 as a potential treatment for ENPP1 Deficiency in Journal of Bone and Mineral Research –
– Presented data from first-ever Burden of Illness Study for ENPP1 and ABCC6 Deficiencies at multiple medical conferences –
– Cash, cash equivalents, and investments expected to enable continued operations into the fourth quarter of 2022 –
BOSTON, May 12, 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 first quarter ended March 31, 2021 and provided recent business highlights.
"We closed 2020 with the clearance of our IND for INZ-701 by the FDA and our CTA by the MHRA of the UK, both for ENPP1 Deficiency. Since then, we have continued to execute on our plan to deliver a potential therapeutic option for patients with ENPP1 and ABCC6 Deficiencies," said Axel Bolte, MSc, MBA, co-founder, president, and chief executive officer of Inozyme Pharma. "We recently reported key findings from the Burden of Illness Study in patients with both ENPP1 Deficiency and ABCC6 Deficiency. Notably, we found patients in different age groups are impacted by these deficiencies in distinct ways, illuminating an age-based shift that reflects the progression of these debilitating genetic diseases. We expect site activation in the United States for our Phase 1/2 clinical trial site for ENPP1 Deficiency in June 2021 and enrollment of the first patient shortly thereafter. Subject to receiving regulatory clearance, we also expect to initiate our planned Phase 1/2 clinical trial of INZ-701 for ABCC6 Deficiency in mid-2021."
Mr. Bolte continued, "Our current cash position allows us to appropriately resource each of the key functions necessary to execute on our goals, and is expected to enable our operations into the fourth quarter of 2022."
Recent Business Highlights
- Filed Clinical Trial Application for INZ-701 for ABCC6 Deficiency in Europe – The Company expects to initiate a Phase 1/2 clinical trial in mid-2021 and to provide preliminary safety and biomarker data by the end of 2021.
- Published data supporting INZ-701 as a potential treatment for ENPP1 Deficiency – Peer-reviewed article in the Journal of Bone and Mineral Research shows INZ-701 increased plasma pyrophosphate (PPi) levels, improved disease markers, and decreased mortality in an Enpp1-deficient mouse model. Preclinical findings support increase of PPi levels as a predictive marker of therapeutic benefit.
- Data presented on utility of INZ-701 as a potential treatment for ABCC6 Deficiency – The Company and research collaborators from Thomas Jefferson University presented data from a preclinical study examining INZ-701 for the potential treatment for ABCC6 Deficiency/Pseudoxanthoma elasticum (PXE) at multiple medical conferences.
- Data presented from Burden of Illness Study for ENPP1 and ABCC6 Deficiencies – The Company presented data from the first-ever burden of illness study in patients with ENPP1 and ABCC6 Deficiencies at multiple medical conferences.
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
- June 2021: Site activation for Phase 1/2 clinical trial
- Mid-2021: Enrollment for Phase 1/2 clinical trial
- Mid-2021: Initiate prospective natural history study
- H2 2021: Report preliminary safety and biomarker data from Phase 1/2 clinical trial
- ABCC6 Deficiency
- Mid-2021: Site activation and enrollment for Phase 1/2 clinical trial
- By the End of 2021: Report preliminary safety and biomarker data from Phase 1/2 clinical trial
Financial Results for the Quarter Ended March 31, 2021
- Cash Position and Financial Guidance – Cash, cash equivalents, and investments were $147.6 million as of March 31, 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 $6.6 million for the quarter ended March 31, 2021, compared to $6.4 million for the quarter ended March 31, 2020. The increase was primarily due to costs associated with preclinical studies and clinical preparation activities with the Company's contract research organization and increased salaries and employee-related costs due to the growth in the number of R&D employees.
- General and Administrative (G&A) Expenses – G&A expenses were $4.4 million for the quarter ended March 31, 2021, compared to $1.5 million for the quarter ended March 31, 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 $11.1 million, or $0.47 loss per share, for the quarter ended March 31, 2021, compared to $7.7 million, or $6.42 loss per share, for the quarter ended March 31, 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)
March 31, 2021 December 31, 2020 Cash, cash equivalents and investments $ 147,634 $ 159,896 Total assets 158,915 169,363 Total liabilities 10,026 11,260 Additional paid-in-capital 251,001 249,175 Accumulated deficit (102,126 ) (91,076 ) Total stockholders' equity 148,889 158,103 Condensed Consolidated Statements of Operations and Comprehensive Loss
(Unaudited)(in thousands, except share and per share data)
Three Months Ended March 31, 2021 2020 Operating expenses: Research and development $ 6,603 $ 6,406 General and administrative 4,369 1,500 Total operating expenses 10,972 7,906 Loss from operations (10,972 ) (7,906 ) Other income (expense): Interest income 63 171 Other expenses (141 ) (3 ) Other income (expense), net (78 ) 168 Net loss $ (11,050 ) $ (7,738 ) Other comprehensive income: Unrealized gains on available-for-sale securities 10 23 Total other comprehensive income 10 23 Comprehensive loss $ (11,040 ) $ (7,715 ) Net loss attributable to common stockholders—basic and diluted $ (11,050 ) $ (7,738 ) Net loss per share attributable to common stockholders—basic and diluted $ (0.47 ) $ (6.42 ) Weighted-average common shares outstanding—basic and diluted 23,429,507 1,205,346
ContactsInvestors:
Inozyme Pharma
Stefan Riley, Director of Investor Relations
[email protected]Media:
Alex Van Rees, SmithSolve
(973) 442-1555 ext. 111
[email protected] -
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BOSTON, May 07, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today presented preclinical data suggesting the utility of its lead clinical development candidate, INZ-701, as a potential treatment for ABCC6 Deficiency. The data, presented at the virtual European Calcified Tissue Society Annual Congress (ECTS, May 6-8), are the first to show that an enzyme replacement therapy (ERT) increased plasma pyrophosphate (PPi) levels and reduced calcification in an animal model of ABCC6 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. This is one of several disorders with significant decrease in plasma 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.
"In patients with ABCC6 Deficiency, the reduced levels of PPi that lead to pathological mineralization suggest an overlap between ENPP1 and ABCC6 Deficiencies," explained Yves Sabbagh, Ph.D., Senior Vice President and Chief Scientific Officer of Inozyme Pharma. "This supports the rationale for an enzyme replacement therapy aimed at raising PPi to treat these serious genetic disorders. The data show that INZ-701 increased plasma PPi levels and prevented abnormal calcification in an ABCC6-deficient mouse model, demonstrating its potential for treating patients with PXE, a chronic form of ABCC6 Deficiency with no approved therapeutic options."
This study was performed in collaboration with Thomas Jefferson University. Subcutaneous administration of INZ-701 (2 and 10 mg/kg every other day for two or eight weeks) was initiated in ABCC6-deficient mice at five to six weeks of age, the time where initiation of ectopic mineralization in this model is observed. INZ-701 led to a dose-dependent increase in plasma PPi levels at both two and eight weeks after initiation of treatment, leading to significantly lower levels of soft tissue mineralization. Histopathologic examination of tissue biopsies from vehicle-treated mice revealed extensive mineralization in the muzzle skin containing vibrissae, a biomarker of the mineralization process in this model. Compared to vehicle-treated mice, a quantitative calcium assay demonstrated that the amount of calcium in muzzle skin biopsies was reduced by 68% and 74% in mice receiving INZ-701 at dose levels of 2 and 10 mg/kg, respectively (p < 0.01).
"It is encouraging to see an ENPP1 enzyme replacement having an effect on tissue calcification in this PXE animal model. The patients suffering from this disease currently have no treatment options and collaborating with Inozyme to use our in-house expertise on this disease with their drug discovery efforts is exciting," said Jouni Uitto, M.D., Ph.D., Professor and Chair of Dermatology and Cutaneous Biology, and Biochemistry and Molecular Biology, at Thomas Jefferson University. "This study will help Inozyme further characterize the therapeutic potential of INZ-701 in PXE and other manifestations of ABCC6 Deficiency, which may offer hope to patient communities that have been waiting many years for a viable treatment option."
About ABCC6 Deficiency and Pseudoxanthoma Elasticum (PXE)
ABCC6 Deficiency is a rare, inherited disorder caused by mutations in the ABCC6 gene, resulting in decreased or absent activity of the ABCC6 protein. A systemic and progressively debilitating condition estimated to affect more than 67,000 individuals worldwide, ABCC6 Deficiency leads to low levels of pyrophosphate (PPi) and is associated with pathological mineralization in blood vessels and soft tissues throughout the body. These effects can result in devastating medical problems including blindness, life-threatening cardiovascular complications, and skin calcification.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. Individuals with PXE often have abnormalities in the eyes, such as changes in the pigmented cells of the retina; angioid streaks (tiny cracks in Bruch's membrane, the inner layer of the retina); and choroidal vascularization (bleeding and scarring of the retina), possibly leading to vision loss. Patients with PXE may also exhibit yellowish bumps, papules, on the neck, underarms, and other areas of the skin which becomes leathery and sagging. The skin findings indicate a general, systemic, pathological process of soft tissue calcification.
About INZ-701
INZ-701 is an ENPP1 enzyme replacement therapy 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 in the first half of 2021 and a separate Phase 1/2 clinical trial in patients with ABCC6 Deficiency in mid-2021.
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.
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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
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