ENPP1

Inozyme Pharma Reports Full Year 2023 Financial Results and Provides Business Highlights

Retrieved on: 
Tuesday, March 12, 2024
Administration, Plasma, Kidney disease, ECTS, Massachusetts General Hospital, Pyrophosphate, PD, ASN, Partnership, Pharmacokinetics, Calciphylaxis, G&A, PROPEL, Doctor of Philosophy, Chemistry, Investment, PXE, Society, Pathology, Therapy, Safety, Research and development, ESKD, ENPP1, MMSc, Natural history, Pharmaceutical industry

BOSTON, March 12, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“the Company” or “Inozyme”), 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 full year ended December 31, 2023, and provided recent business highlights.

Key Points: 
  • “We believe we are well-positioned to continue to advance INZ-701 through several anticipated value-creating milestones in the year ahead,” said Douglas A. Treco, Ph.D., CEO of Inozyme Pharma.
  • Calciphylaxis represents a devastating condition characterized by pathologic mineralization and intimal proliferation.
  • Patients will receive 1.8 mg/kg of INZ-701 once weekly, coinciding with their hemodialysis treatment, for a total of 30 days.
  • The study’s primary endpoint will assess safety and change from baseline plasma PPi concentration, with secondary endpoints including PK and PD parameters.

Rallybio Highlights Portfolio Advances and Outlines Expected Milestones in 2024

Retrieved on: 
Thursday, January 4, 2024
Research, Finance, Clinical Trials, Professional Services, Biotechnology, Health, Pharmaceutical, General Health, Science, Conference, Geographic atrophy, Food, GMG, Risk, Woman, EMA, Paratriathlon, Data, Injection site reaction, AbCellera, HPP, Antiphospholipid syndrome, European Medicines Agency, Pregnancy, Corporation, DSM-IV codes, C5, Hypophosphatasia, ENPP1, Investment, Webcast, Patient, Therapy, Pharmaceutical industry

Rallybio expects to initiate its Phase 2 dose confirmation study for RLYB212 in the second half of 2024.

Key Points: 
  • Rallybio expects to initiate its Phase 2 dose confirmation study for RLYB212 in the second half of 2024.
  • Rallybio expects to complete this manufacturing work and provide an update on the development plan for RLYB116 in the second half of 2024.
  • Rallybio and EyePoint expect to provide an update on this collaboration in the first half of 2024.
  • Rallybio and Exscientia plan to provide an update on the progress of the program in the second half of 2024.

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

Retrieved on: 
Tuesday, November 7, 2023
Generalized arterial calcification of infancy, Patient, Research, Research and development, Genomics, Clinical trial, PD, Calciphylaxis, Therapy, Biomarker, ESKD, G&A, American Society of Nephrology, Conference, Pros, Kidney disease, R, American Society for Bone and Mineral Research, Society, PXE, Columbia University School of General Studies, ENPP1, ASN, PK, Clinical Global Impression, Annual general meeting, GIC, Safety, ABCC6, Investment, Doctor of Philosophy, Administration, Nursing, Pharmaceutical industry

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

Retrieved on: 
Tuesday, October 10, 2023
Genomics England, Skeleton, Columbia University School of General Studies, Therapy, Heart, Genomics, Disease, ABCC6, Health, Blood, NHS, Gene, ENPP1, Genetic disorder, Generalized arterial calcification of infancy, Enzyme replacement therapy, Pharmaceutical industry

“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 Reports Second Quarter 2023 Financial Results and Provides Business Highlights

Retrieved on: 
Tuesday, August 8, 2023
Interim, Therapy, Marketing, Food, Cohort, European Medicines Agency, G&A, Pyrophosphate, ENPP1, EMA, Chemistry, Plasma, Congress, Investment, Administration, European Society of Endocrinology, Infant, Research and development, R, Research, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Conference, Safety, Medical imaging, Life insurance, Pharmaceutical industry, EU, Endocrinology, Patient

BOSTON, Aug. 08, 2023 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“Inozyme” or “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 second quarter ended June 30, 2023, and provided business highlights.

Key Points: 
  • Pending regulatory discussions and appropriate financial resources, the Company also plans to conduct the ENERGY-4 pivotal trial in adolescents and adults with ENPP1 Deficiency.
  • R&D expenses were $11.7 million for the quarter ended June 30, 2023, compared to $10.0 million for the prior-year period.
  • G&A expenses were $4.7 million for the quarter ended June 30, 2023, compared to $5.4 million for the prior-year period.
  • Net loss was $15.6 million, or $0.35 loss per share, for the quarter ended June 30, 2023, compared to $15.3 million, or $0.38 loss per share, for the prior-year period.

OncoNano Presents Compelling Data for ONM-501 and a Novel Tumor Specific Delivery Technology at AACR Annual Meeting 2023

Retrieved on: 
Tuesday, April 18, 2023
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Infection, AACR, CDN, PD, Ectonucleotide pyrophosphatase/phosphodiesterase 1, Nucleic acid metabolism, Therapeutic index, Cancer, American Association for Cancer Research, Lead, Toxicity, ENPP1, Research and development, Development, Translational medicine, Patient, Interleukin 12, Stimulator of interferon genes, Mouse, Interleukin, Vaccine, Research

OncoNano Medicine, Inc. presented three posters at the American Association for Cancer Research (AACR) Annual Meeting 2023.

Key Points: 
  • OncoNano Medicine, Inc. presented three posters at the American Association for Cancer Research (AACR) Annual Meeting 2023.
  • The posters detail positive nonclinical data for ONM-501, the Company’s dual-activating STING (STimulator of INterferon Genes) agonist and lead therapeutic development candidate, formulated with the company’s OMNI™ polymer technology as well as positive data for encapsulated bispecific antibody and cytokine using ON-BOARD™ tumor specific delivery technology.
  • This novel mechanism provides potent anti-tumor efficacy as a monotherapy and in combination with anti-PD1 in multiple preclinical mouse models with both “hot” and “cold” tumor microenvironments.
  • The promising data that we presented at AACR 2023 suggest our technology may provide a solution to overcome the clinical application limitations of these highly potent protein therapeutics.”

Rallybio Reports Fourth Quarter and Full Year 2022 Financial Results

Retrieved on: 
Monday, March 6, 2023
Professional Services, Health, Finance, General Health, Clinical Trials, Pharmaceutical, Biotechnology, Research and development, Geographic atrophy, Retirement, Corporation, DSM-IV codes, G&A, Therapy, Administration, Acquisition, AbCellera, Research, General Administration of Sport of China, HPP, Woman, ENPP1, Paratriathlon, Rare, Patient, Risk, Pharmacokinetics, Hypophosphatasia, Disease, Hematology, Male, Quarter, Pregnancy, R, CFO, Female, CPA, Safety, Pharmaceutical industry, Vaccine, Cryptocurrency, IND

In the fourth quarter of 2022, we initiated a multiple ascending dose Phase 1 study of RLYB116, our inhibitor of complement component 5 (C5), and we look forward to sharing initial data from this study in the fourth quarter of 2023.

Key Points: 
  • In the fourth quarter of 2022, we initiated a multiple ascending dose Phase 1 study of RLYB116, our inhibitor of complement component 5 (C5), and we look forward to sharing initial data from this study in the fourth quarter of 2023.
  • The Company expects results from this cohort of subjects in the fourth quarter of 2023.
  • Rallybio initiated dosing in the first multiple ascending dose cohort of a Phase 1 study of RLYB116 in the fourth quarter of 2022.
  • Fourth Quarter and Full Year 2021 Financial Results:
    Research & Development (R&D) Expenses: R&D expenses were $10.8 million for the fourth quarter of 2022, compared to $6.1 million for the same period in 2021.

Inozyme Pharma Announces Investor and Analyst Event and Highlights 2022 Progress

Retrieved on: 
Monday, January 9, 2023
Pyrophosphate, Orphanet Journal of Rare Diseases, PXE, Degenerative disease, Caregiver, Master of Science, Mutation, PLOS One, PLOS, Dermatology, Western literature, Safety, Precision medicine, Hospital, ABCC6, Biology, Diagnosis, Therapy, Patient, Prevalence, Human Mutation, Research, Disease, Publication, ENPP1, Natural history study, Whole genome sequencing, Genetic distance, MBA, Infant, NIH, Dietary supplement, Pharmaceutical industry

BOSTON, Jan. 09, 2023 (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 that it will share topline pharmacokinetic, pharmacodynamic (PK/PD) and safety data from the ongoing Phase 1/2 clinical trials of INZ-701 in ENPP1 Deficiency and ABCC6 Deficiency at a virtual Investor and Analyst Event on Thursday, Feb. 16, 2023.

Key Points: 
  • We also saw promising PPi elevation in our ABCC6 Deficiency trial, with a rapid initial increase at the lowest dose of INZ-701.
  • The Company will share topline pharmacokinetic, pharmacodynamic (PK/PD) and safety data from ongoing Phase 1/2 trials of INZ-701 in ENPP1 Deficiency and ABCC6 Deficiency.
  • The event will also feature presentations from members of the Inozyme management team, as well as from key opinion leaders in ENPP1 Deficiency and ABCC6 Deficiency.
  • The webcast will be accessible through the Investor Relations section of Inozyme’s website under events and will be available for a limited time following the event.

Rallybio Highlights Portfolio Advances and Outlines Expected Milestones in 2023

Retrieved on: 
Thursday, January 5, 2023
Professional Services, Health, Finance, Clinical Trials, General Health, Biotechnology, C5, AbCellera, Pharmacokinetics, Hypophosphatasia, Risk, Corporation, ENPP1, Disease, Patient, Pregnancy, Degenerative disease, Safety, HPP, Therapy, Webcast, IND, Conference, Woman, Pharmaceutical industry

We also significantly expanded our earlier-stage pipeline through strategic business development efforts.”

Key Points: 
  • We also significantly expanded our earlier-stage pipeline through strategic business development efforts.”
    Dr. Mackay continued, “We are excited to build on this progress in 2023.
  • Rallybio also continues to advance the development of RLYB114, formulated for intravitreal injection, for the treatment of ophthalmic disorders.
  • In vivo efficacy data are expected in the second half of 2023.
  • In December 2022, Rallybio announced a strategic alliance with AbCellera to discover, develop, and commercialize novel antibody-based therapeutics for rare diseases.

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

Retrieved on: 
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.