Pseudoxanthoma elasticum

Inozyme Pharma Announces Three Poster Presentations at the American Society for Bone and Mineral Research (ASBMR) 2023 Annual Meeting

Retrieved on: 
Friday, October 6, 2023

BOSTON, Oct. 06, 2023 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“Inozyme” or the “Company”), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced that it will present three posters at the American Society for Bone and Mineral Research (ASBMR) 2023 Annual Meeting, which is being held October 13-16, 2023 in Vancouver, BC, Canada.

Key Points: 
  • BOSTON, Oct. 06, 2023 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“Inozyme” or the “Company”), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced that it will present three posters at the American Society for Bone and Mineral Research (ASBMR) 2023 Annual Meeting, which is being held October 13-16, 2023 in Vancouver, BC, Canada.
  • Details of the presentations are as follows:
    Title: A Phase 1/2 Open-Label, Multiple Ascending Dose Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Adults with ENPP1 Deficiency
    Author/Presenter: Yves Sabbagh, Ph.D. (Senior Vice President and Chief Scientific Officer, Inozyme Pharma)
    Title: A Phase 1/2 Open-Label, Multiple Ascending Dose Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 Followed by an Open-Label Long-Term Extension Period in Adults with ABCC6 Deficiency Manifesting as Pseudoxanthoma Elasticum (PXE): An Interim Analysis
    Author/Presenter: Yves Sabbagh, Ph.D. (Senior Vice President and Chief Scientific Officer, Inozyme Pharma)
    Author/Presenter: I-Ping Chen, D.D.S., Ph.D. (Associate Professor, Oral Health and Diagnostic Sciences, UConn Health)

Inozyme Pharma Reports Third Quarter 2022 Financial Results and Provides Business Updates

Retrieved on: 
Thursday, November 10, 2022

BOSTON, Nov. 10, 2022 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today reported financial results for the third quarter ended September 30, 2022 and provided recent business highlights.

Key Points: 
  • Preclinical data supporting INZ-701's potential to treat intimal proliferation was featured at the International Vascular Biology Meeting (IVBM) 2022 Annual Meeting in October.
  • Cash Position and Financial Guidance Cash, cash equivalents, and investments were $141.5 million as of September 30, 2022.
  • Research and Development (R&D) Expenses R&D expenses were$12.2 millionfor the quarter ended September 30, 2022, compared to$9.3 millionfor the prior-year period.
  • General and Administrative (G&A) Expenses G&A expenses were$4.7 millionfor the quarter ended September 30, 2022, compared to$4.9 millionfor the prior-year period.

Inozyme Pharma Announces Positive Preliminary Data from Phase 1/2 Clinical Trial of INZ-701 in Subjects with ABCC6 Deficiency (pseudoxanthoma elasticum or PXE)

Retrieved on: 
Tuesday, July 19, 2022

BOSTON, July 19, 2022 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced positive preliminary biomarker, safety, and pharmacokinetic (PK) data from the first three subjects treated in the Phase 1 portion of its ongoing Phase 1/2 clinical trial of INZ-701 in adult subjects with ABCC6 Deficiency, which presents as pseudoxanthoma elasticum (PXE) in older individuals. At the 0.2 mg/kg dose level of INZ-701, all three subjects showed rapid and significant increases in PPi levels. In preclinical models, PPi was shown to be a key predictive biomarker of therapeutic benefit in ABCC6 Deficiency.

Key Points: 
  • Preliminary PK and INZ-701 enzymatic activity remained consistent with data previously reported from the Companys ongoing Phase 1/2 trial of INZ-701 in subjects with ENPP1 Deficiency.
  • The Company plans to report topline data from the ongoing Phase 1/2 clinical trial in ABCC6 Deficiency in the first quarter of 2023.
  • INZ-701 is currently in Phase 1/2 clinical trials for the treatment of ENPP1 Deficiency and ABCC6 Deficiency.
  • INZ-701 is currently in Phase 1/2 clinical trials for the treatment of ENPP1 Deficiency and ABCC6 Deficiency.

Inozyme Pharma Announces Partnership with Rady Children’s Institute for Genomic Medicine to Advance Newborn Screening for Genetic Diseases

Retrieved on: 
Thursday, June 16, 2022

We look forward to working with Rady Childrens Institute for Genomic Medicine, and with the BeginNGS consortium, to advance the use of this promising screening technology.

Key Points: 
  • We look forward to working with Rady Childrens Institute for Genomic Medicine, and with the BeginNGS consortium, to advance the use of this promising screening technology.
  • We are thrilled at the prospect of newborn screening to assist in early identification of infants affected by ENPP1 Deficiency and ABCC6 Deficiency via Inozymes collaboration with Rady Childrens Institute for Genomic Medicine.
  • Rady Childrens Institute for Genomic Medicine is transforming pediatric critical care by advancing disease-specific healthcare for infants and children with rare disease.
  • These statements include, but are not limited to, statements relating the partnership with Rady Childrens Institute for Genomic Medicine.

Inozyme Pharma Reports First Quarter 2022 Financial Results and Provides Business Highlights

Retrieved on: 
Tuesday, May 10, 2022

BOSTON, May 10, 2022 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today reported financial results for the first quarter ended March 31, 2022 and provided recent business highlights.

Key Points: 
  • Cash, cash equivalents and investments as of quarter end, together with proceeds from April 2022 offering, extends runway into the fourth quarter of 2023
    BOSTON, May 10, 2022 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today reported financial results for the first quarter ended March 31, 2022 and provided recent business highlights.
  • Inozyme plans to report topline data from the ongoing trial in the second half of 2022.
  • The Company is on track to report preliminary biomarker and safety data in the second quarter of 2022.
  • Inozyme expects to commence prospective natural history studies of both populations in the second quarter of 2022.

Inozyme Pharma Provides Update for Phase 1/2 Clinical Trials in ABCC6 Deficiency and ENPP1 Deficiency

Retrieved on: 
Tuesday, April 12, 2022

BOSTON, April 12, 2022 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced that the first patient has been dosed in its Phase 1/2 clinical trial of INZ-701 in adult patients with ABCC6 Deficiency and the second cohort in the ongoing Phase 1/2 clinical trial in ENPP1 Deficiency has been fully enrolled.

Key Points: 
  • In preclinical models of ABCC6 Deficiency and ENPP1 Deficiency, INZ-701 increased plasma pyrophosphate (PPi) levels and prevented soft tissue calcification, a key manifestation of both conditions.
  • The ongoing Phase 1/2 open-label clinical trial is expected to enroll up to nine adult patients with ABCC6 Deficiency at sites in the United States and Europe.
  • INZ-701 is currently in Phase 1/2 clinical trials for the treatment of ENPP1 Deficiency and ABCC6 Deficiency.
  • INZ-701 is currently in Phase 1/2 clinical trials for the treatment of ENPP1 Deficiency and ABCC6 Deficiency.

Inozyme Pharma Reports Full Year 2021 Financial Results and Provides Business Highlights

Retrieved on: 
Tuesday, March 15, 2022

BOSTON, March 15, 2022 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today reported financial results for the full year ended December 31, 2021, and provided recent business highlights.

Key Points: 
  • BOSTON, March 15, 2022 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today reported financial results for the full year ended December 31, 2021, and provided recent business highlights.
  • 2021 was a transformational year for Inozyme, as we continued to progress our INZ-701 program and graduated to a clinical-stage company.
  • Patient screening is underway in the Companys Phase 1/2 trial of INZ-701 in patients with ABCC6 Deficiency.
  • The Company expects to enroll its first patient in the Phase 1/2 trial in the second quarter of 2022.

Global Pseudoxanthoma Elasticum Market Insight, Epidemiology and Market Forecasts, 2019-2021 & 2022-2032 - ResearchAndMarkets.com

Retrieved on: 
Monday, March 7, 2022

This "Pseudoxanthoma Elasticum- Market Insights, Epidemiology, and Market Forecast-2032" report delivers an in-depth understanding of the Pseudoxanthoma Elasticum, historical and forecasted epidemiology as well as the Pseudoxanthoma Elasticum market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

Key Points: 
  • This "Pseudoxanthoma Elasticum- Market Insights, Epidemiology, and Market Forecast-2032" report delivers an in-depth understanding of the Pseudoxanthoma Elasticum, historical and forecasted epidemiology as well as the Pseudoxanthoma Elasticum market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.
  • The Pseudoxanthoma Elasticum market report provides current treatment practices, emerging drugs, Pseudoxanthoma Elasticum market share of the individual therapies, current and forecasted Pseudoxanthoma Elasticum market Size from 2019 to 2032 segmented by seven major markets.
  • The Pseudoxanthoma Elasticum market report gives a thorough understanding of the Pseudoxanthoma Elasticum by including details such as disease definition, symptoms, causes, pathophysiology, diagnosis and treatment.
  • The Pseudoxanthoma Elasticum epidemiology division provide insights about historical and current Pseudoxanthoma Elasticum patient pool and forecasted trend for every seven major countries.

Inozyme Announces Acceptance of First European Clinical Trial Application for Phase 1/2 Clinical Trial of INZ-701 in ABCC6 Deficiency

Retrieved on: 
Wednesday, June 9, 2021

This is one of several disorders with a significant decrease in plasma pyrophosphate (PPi) levels, a potent regulator of mineralization.

Key Points: 
  • 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.
  • ABCC6 Deficiency is a rare, severe, inherited disorder caused by mutations in the ABCC6 gene, leading to low levels of PPi.
  • 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.

Inozyme Pharma Presents Preclinical Data Suggesting Utility of INZ-701 as a Potential Treatment for ABCC6 Deficiency

Retrieved on: 
Friday, May 7, 2021

b'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.

Key Points: 
  • b'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.
  • 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.\n\xe2\x80\x9cIn patients with ABCC6 Deficiency, the reduced levels of PPi that lead to pathological mineralization suggest an overlap between ENPP1 and ABCC6 Deficiencies,\xe2\x80\x9d explained Yves Sabbagh, Ph.D., Senior Vice President and Chief Scientific Officer of Inozyme Pharma.
  • \xe2\x80\x9cThis supports the rationale for an enzyme replacement therapy aimed at raising PPi to treat these serious genetic disorders.