Pyrophosphate

Inozyme Pharma Announces Presentation at the American Society of Nephrology (ASN) Kidney Week 2023

Retrieved on: 
Thursday, October 26, 2023

BOSTON, Oct. 26, 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 an oral presentation at the American Society of Nephrology (ASN) Kidney Week 2023, which is being held November 2-5, 2023 in Philadelphia, PA.

Key Points: 
  • BOSTON, Oct. 26, 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 an oral presentation at the American Society of Nephrology (ASN) Kidney Week 2023, which is being held November 2-5, 2023 in Philadelphia, PA.
  • Details of the presentation are as follows:
    Title: Role of Plasma Inorganic Pyrophosphate in Calciphylaxis: A Prospective Study
    Calciphylaxis is a rare disorder with a high mortality rate that mostly affects patients with end stage kidney disease (ESKD).
  • The estimated incidence of calciphylaxis is at least 1,800 new patients per year in the United States and there are no approved therapies.
  • Inozyme previously presented data at the European Calcified Tissue Society Congress (ECTS) which showed that morbidity and mortality in patients with calciphylaxis were associated with low levels of PPi.

AM-Pharma Announces Positive Clinical Data From Phase 1b Study Evaluating Ilofotase Alfa in Hypophosphatasia Patients

Retrieved on: 
Wednesday, October 11, 2023

AM-Pharma B.V. today announced positive clinical results from the Phase 1b study evaluating the company’s proprietary recombinant alkaline phosphatase, ilofotase alfa, as potential enzyme replacement therapy in adult hypophosphatasia (HPP) patients.

Key Points: 
  • AM-Pharma B.V. today announced positive clinical results from the Phase 1b study evaluating the company’s proprietary recombinant alkaline phosphatase, ilofotase alfa, as potential enzyme replacement therapy in adult hypophosphatasia (HPP) patients.
  • Patients were followed for 10 days after dosing to evaluate changes in the biochemical fingerprint of HPP, safety and tolerability.
  • In addition, ilofotase alfa was well-tolerated with pharmacokinetics consistent with observations from previous clinical studies.
  • "The encouraging early results confirming dose-dependent reductions of PLP and PPi levels upon treatment with ilofotase alfa underline the compound's therapeutic potential in that indication and I'm looking forward to evaluating ilofotase alfa in next stage clinical studies."

Inozyme Pharma Announces Positive Interim Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with ENPP1 Deficiency and ABCC6 Deficiency (PXE)

Retrieved on: 
Tuesday, September 26, 2023

BOSTON, Sept. 26, 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 positive interim safety, pharmacokinetic (PK), pharmacodynamic (PD) and exploratory efficacy data from the Company’s ongoing Phase 1/2 clinical trials of INZ-701 in adults with ENPP1 Deficiency and ABCC6 Deficiency (PXE, pseudoxanthoma elasticum).

Key Points: 
  • For trial design details, please see the section entitled “INZ-701 in ENPP1 Deficiency Phase 1/2 Clinical Trial Design” below.
  • Exploratory biomarker data were collected throughout the study to provide evidence of the potential for disease modification with ongoing treatment with INZ-701.
  • For trial design details, please see the section entitled “INZ-701 in ABCC6 Deficiency Phase 1/2 Clinical Trial Design” below.
  • PK properties were consistent with those observed in the Phase 1/2 clinical trial in adults with ENPP1 Deficiency.

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

Retrieved on: 
Tuesday, August 8, 2023

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.

SHAREHOLDER ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Inozyme Pharma, Inc. - INZY

Retrieved on: 
Wednesday, August 2, 2023

NEW YORK, Aug. 02, 2023 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Inozyme Pharma, Inc. (“Inozyme” or the “Company”) (NASDAQ: INZY).

Key Points: 
  • NEW YORK, Aug. 02, 2023 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Inozyme Pharma, Inc. (“Inozyme” or the “Company”) (NASDAQ: INZY).
  • Such investors are advised to contact Robert S. Willoughby at [email protected] or 888-476-6529, ext.
  • The investigation concerns whether Inozyme and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices.
  • Founded by the late Abraham L. Pomerantz, known as the dean of the class action bar, Pomerantz pioneered the field of securities class actions.

Inozyme Pharma Announces Updates on Global Development Strategy of INZ-701 for the Treatment of ENPP1 Deficiency

Retrieved on: 
Wednesday, July 26, 2023

BOSTON, July 26, 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 a regulatory update for its global development strategy of INZ-701 for the treatment of ENPP1 Deficiency following recent meetings with the United States (U.S.) Food and Drug Administration (FDA) and the Paediatric Committee (PDCO) of the European Medicines Agency (EMA).

Key Points: 
  • The Company plans to initiate the ENERGY-3 pivotal trial, a multicenter, randomized, open label trial in pediatric patients with ENPP1 Deficiency in October 2023.
  • Enrollment criteria for the trial include a confirmed genetic diagnosis of ENPP1 Deficiency, radiographic evidence of skeletal abnormalities and low plasma PPi.
  • Discussions are ongoing with the FDA regarding the design of a potential pivotal trial of INZ-701 in infants with ENPP1 Deficiency in the U.S.
  • Inozyme will host a conference call and webcast to discuss its global development strategy for INZ-701 in patients with ENPP1 Deficiency today, July 26, 2023 at 8am ET.

Inozyme Pharma Announces Dosing of First Infant with ENPP1 Deficiency in a Phase 1b Trial of INZ-701

Retrieved on: 
Tuesday, June 27, 2023

“Initiation of the ENERGY-1 trial in infants is an important milestone as we continue to advance INZ-701 with the goal of improving the lives of patients with ENPP1 Deficiency across all age groups.

Key Points: 
  • “Initiation of the ENERGY-1 trial in infants is an important milestone as we continue to advance INZ-701 with the goal of improving the lives of patients with ENPP1 Deficiency across all age groups.
  • We are committed to a global program to identify and treat all newborns with this condition,” said Kurt Gunter, M.D., senior vice president and chief medical officer of Inozyme.
  • “Infants diagnosed with ENPP1 Deficiency face a high mortality risk in the first months of life.
  • ENERGY-1 is a Phase 1b, single arm, open label clinical trial designed to primarily assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of INZ-701 in infants with ENPP1 Deficiency.

Inozyme Pharma Announces Investor and Analyst Event and Highlights 2022 Progress

Retrieved on: 
Monday, January 9, 2023

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.

Rockwell Medical Provides Business Strategy Update and Announces Third Quarter 2022 Financial and Operational Results

Retrieved on: 
Monday, November 14, 2022

During the third quarter of 2022, Rockwell Medical evaluated its businesses and the potential for short- and long-term value.

Key Points: 
  • During the third quarter of 2022, Rockwell Medical evaluated its businesses and the potential for short- and long-term value.
  • In light of a very challenging macroeconomic environment, we are pleased with our performance for the third quarter of 2022.
  • During and subsequent to the third quarter of 2022, Rockwell undertook workforce reductions as part of its business restructuring.
  • Rockwell Medicals Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2022 will provide a full analysis of the Companys business strategy as well as its third quarter of 2022 results.

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.