Generalized arterial calcification of infancy

Inozyme Pharma Reports Third Quarter 2023 Financial Results and Provides Business Highlights

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Tuesday, November 7, 2023
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BOSTON, Nov. 07, 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 reported financial results for the third quarter ended September 30, 2023 and provided business highlights.

Key Points: 
  • Patient recruitment is underway, and the Company remains on track to report topline data in mid-2025.
  • Exploratory efficacy data reported suggested clinical benefit for ENPP1 Deficiency, including improvement in key biomarkers, patient-reported outcomes (PROs), and functional outcomes.
  • R&D Expenses were $13.3 million for the quarter ended September 30, 2023, compared to $12.2 million for the prior-year period.
  • Net loss was $16.6 million, or $0.29 loss per share, for the quarter ended September 30, 2023, compared to $16.4 million, or $0.38 loss per share, for the prior-year period.

Inozyme Pharma Highlights Inclusion of Generalized Arterial Calcification of Infancy (GACI) in Genomics England’s Generation Study of Rare Conditions

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Tuesday, October 10, 2023
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“We commend Genomics England for recognizing the crucial importance of early detection of GACI as we advance INZ-701 into clinical trials in infants.

Key Points: 
  • “We commend Genomics England for recognizing the crucial importance of early detection of GACI as we advance INZ-701 into clinical trials in infants.
  • Genomics England collaborated with NHS experts as well as scientists, healthcare professionals (HCPs), and people living with rare conditions to select 223 individual conditions for its current screening program.
  • Inozyme is developing INZ-701, an enzyme replacement therapy, for the treatment of rare disorders like GACI that impact the vasculature, soft tissue, or skeleton.
  • To learn more about Genomics England and its Generation Study, read the full announcement here .

Inozyme Pharma Announces Peer-Reviewed Publication Revealing Increased Prevalence of ENPP1 Deficiency

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Monday, December 5, 2022
Pediatrics, Master of Science, Physician, Orphanet Journal of Rare Diseases, Diagnosis, Data, Mortality, Prevalence, Osteomalacia, Hospital, Genomics, Degenerative disease, Cancer, Myocardial infarction, Stroke, Skeleton, Blood, Knowledge, Education, Hearing loss, Rickets, Disease, NIH, MBA, Research, Database, Generalized arterial calcification of infancy, Patient, Therapy, Pharmaceutical industry

BOSTON, Dec. 05, 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 announced the publication of an article titled “Estimation of ENPP1 Deficiency Genetic Prevalence Using a Comprehensive Literature Review and Population Databases” in the Orphanet Journal of Rare Diseases. Leading disease experts Carlos Ferreira, M.D., of the National Institutes of Health (NIH) and Frank Rutsch, M.D., of Münster University Children’s Hospital, together with Genomenon, an AI-driven genomics company, analyzed the latest genomics data and found the estimated prevalence of ENPP1 Deficiency to be 1 in 64,000 pregnancies, more than tripling the prior estimate1.

Key Points: 
  • “Our medical field team’s disease awareness, education, and patient identification efforts suggested that the number of patients with ENPP1 Deficiency has been vastly underestimated.
  • “Given the higher prevalence of ENPP1 Deficiency, it becomes obvious that many affected individuals remain undiagnosed.
  • “The prevalence data were gathered using a comprehensive database of genomic evidence, providing an unprecedented view into how many people are born with ENPP1 Deficiency worldwide.
  • INZ-701 is currently in Phase 1/2 clinical trials for the treatment of ENPP1 Deficiency and ABCC6 Deficiency.

New Study Shows Comprehensive Genomic Evidence is Critical for Calculating Rare Disease Prevalence

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Thursday, December 15, 2022
NIH, Degenerative disease, Therapy, Rickets, Generalized arterial calcification of infancy, Orphanet Journal of Rare Diseases, ENPP1, Doctor of Philosophy, Blood, Patient, Prevalence, Cancer, Research, Knowledge, ANN, Disease, Hearing loss, Genomics, Osteomalacia, Hospital, Discovery, Myocardial infarction, Stroke, Genetic disorder, Pharmaceutical industry, MD

ANN ARBOR, Mich., Dec. 15, 2022 /PRNewswire-PRWeb/ -- Genomenon, Inc., an AI-driven genomics company, today announced the publication of a paper in the Orphanet Journal of Rare Diseases that calculates the prevalence of ENPP1 deficiency—a disease claiming more than half of all infants within the first few months of life—to be more than triple the prior estimate. The study, conducted by the Genomenon scientific team and leading disease experts from the National Institutes of Health (NIH) and Münster University Children's Hospital, highlights a core strength of Genomenon's rigorous methodology for identifying and classifying genetic data needed to improve rare disease prevalence calculations.

Key Points: 
  • The study, conducted by the Genomenon scientific team and leading disease experts from the National Institutes of Health (NIH) and Mnster University Children's Hospital, highlights a core strength of Genomenon's rigorous methodology for identifying and classifying genetic data needed to improve rare disease prevalence calculations.
  • "Discovery of this higher prevalence rate aptly demonstrates how our exhaustive knowledgebase of expertly curated genomic evidence supports pharmaceutical companies that are pioneering development of therapies for orphan diseases."
  • Traditional methods for estimating prevalence rely on clinical data, which can be unreliable for rare diseases such as ENPP1 deficiency that are difficult to diagnose.
  • Genomenon is an AI-driven genomics company focused on making genomic information actionable for patients with rare genetic diseases and cancer.

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

Retrieved on: 
Thursday, November 10, 2022
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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 First Self-Administration of INZ-701 in Ongoing ENPP1 Deficiency Phase 1/2 Clinical Trial

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Thursday, November 3, 2022
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Inozyme is facilitating self-administration in the Phase 2 extension portion of the clinical trial after patients complete the 32-day dose evaluation period in the clinic.

Key Points: 
  • Inozyme is facilitating self-administration in the Phase 2 extension portion of the clinical trial after patients complete the 32-day dose evaluation period in the clinic.
  • The ongoing Phase 1/2 open-label clinical trial is expected to enroll up to nine adult patients with ENPP1 Deficiency at sites in North America 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 Announces Publication of Comprehensive ENPP1 Variant Database

Retrieved on: 
Monday, October 31, 2022
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BOSTON, Oct. 31, 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 announced the publication of an article titled, “ENPP1 Deficiency: A clinical update on the relevance of individual variants using a locus-specific database,” in Human Mutation. The Company partnered with leading disease experts and Genomenon, an AI-driven genomics company, to perform a comprehensive review of the current knowledge on clinical and genetic findings of ENPP1 Deficiency (GACI, generalized arterial calcification of infancy or ARHR2, autosomal recessive hypophosphatemic rickets type 2) and produce a comprehensive variant database.

Key Points: 
  • The Company partnered with leading disease experts and Genomenon , an AI-driven genomics company, to perform a comprehensive review of the current knowledge on clinical and genetic findings of ENPP1 Deficiency (GACI, generalized arterial calcification of infancy or ARHR2, autosomal recessive hypophosphatemic rickets type 2) and produce a comprehensive variant database.
  • These data shared by our collaborators suggest a larger population with ENPP1 Deficiency than previously understood.
  • The publication also showed that there was no clear genotype-phenotype correlation and that people with heterozygous mutations may also benefit from a therapeutic option.
  • Genomenon has worked with Inozyme for the past year to produce a comprehensive variant landscape for ENPP1 Deficiency, said Mark Kiel, M.D., Ph.D., chief science officer and co-founder of Genomenon.

New Study Presents Novel Solution to Increasing Diagnostic Yield and Accelerating Diagnosis of Rare Disease

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Monday, October 31, 2022
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ANN ARBOR, Mich. , Oct. 31, 2022   /PRNewswire-PRWeb/ -- Genomenon, Inc., an AI-driven genomics company, announced the publication of a paper in Human Mutation summarizing current knowledge of clinical and genetic findings in patients with ENPP1 Deficiency. The study, completed in collaboration with Inozyme Pharma, is the largest ever gathered, and demonstrates the necessity of comprehensive genomic data for accelerating the diagnosis of rare disease.

Key Points: 
  • The study, completed in collaboration with Inozyme Pharma, is the largest ever gathered, and demonstrates the necessity of comprehensive genomic data for accelerating the diagnosis of rare disease.
  • The data has been made available to doctors, researchers, and clinicians through Genomenon's Mastermind Genomic Search Engine with the purpose of increasing awareness and diagnosis of the disease and related clinical trials.
  • Inozyme Pharma, Inc. (Nasdaq: INZY) is a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases impacting the vasculature, soft tissue, and skeleton.
  • INZ-701 is currently in Phase 1/2 clinical trials for the treatment of ENPP1 Deficiency and ABCC6 Deficiency.

AVS Announces Multi-Site Enrollment of First Patients in POWER PAD I Clinical Trial and Appointment of William H. Kucheman to Board of Directors

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Thursday, September 15, 2022
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AVS is only the second company to conduct a first-in-human study in the rapidly growing intravascular lithotripsy space.

Key Points: 
  • AVS is only the second company to conduct a first-in-human study in the rapidly growing intravascular lithotripsy space.
  • AVS is the first company to develop pulsatile intravascular lithotripsy, a therapy that is delivered through a balloon-based platform called PULSE.
  • We are excited by this first-in-human trial milestone we have achieved, added Mark Toland, President and Chief Executive Officer of AVS.
  • Kucheman added, I am delighted to join the AVS board and look forward to helping patients with limited treatment options.

Shockwave Medical and Genesis MedTech Obtain Regulatory Approval in China for Intravascular Lithotripsy

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Monday, May 23, 2022
People, Consensus, Vascular disease, Moyamoya disease, U.S. Securities and Exchange Commission, Organizational culture, Private Securities Litigation Reform Act, Plaque, SEC, Risk, Annual report, Physician, Liberation Army, IVL, Calcium, Incidence, Generalized arterial calcification of infancy, COVID-19, Marketing, Research, Shock wave, Medtech, Animation, Growth, Cardiovascular disease, GLOBE, Hospital, Partnership, Ageing, Security (finance), Population, Zhongshan Hospital, Calcification, Patient, Genesis, NASDAQ, Treatment, Forward-looking statement, NMPA, M5, Union, Technology, Coronary artery disease, C2, Fudan University, Diagnosis, Pharmaceutical industry

"Shockwave IVL technology is an innovative solution for treating coronary artery calcification, especially when the calcium is deep in the artery.

Key Points: 
  • "Shockwave IVL technology is an innovative solution for treating coronary artery calcification, especially when the calcium is deep in the artery.
  • Professor Fu said: "Shockwave IVL provides a powerful solution to calcified lesions that could not be effectively treated in the past.
  • Genesis MedTech is a perfect partner for Shockwave in China and this accomplishment underscores the value of their established infrastructure and local relationships.
  • Genesis MedTech Group covers the entire industry value chain of research and development, production, quality management, supply chain, marketing, and sales.