PMM2-CDG

Glycomine Announces Encouraging Efficacy Data from Ongoing Phase 2 Clinical Study in PMM2-CDG

Retrieved on: 
Monday, March 4, 2024

This compares favorably to prior results reported with acetazolamide which demonstrated an average improvement of 6 points after 25 weeks in adult and pediatric patients (n=25).

Key Points: 
  • This compares favorably to prior results reported with acetazolamide which demonstrated an average improvement of 6 points after 25 weeks in adult and pediatric patients (n=25).
  • “We are very encouraged by the initial results we have seen and delighted to be part of this important study.”
    The GLM101-002 Phase 2 clinical study has enrolled 10 adult patients in the U.S. and Spain (ClinicalTrials.gov Identifier: NCT05549219).
  • Study participants have received GLM101 at either 10 mg/kg (n=3), 20 mg/kg (n=3), or 30 mg/kg (n=4) for up to 24 weeks.
  • Four adolescent patients have initiated treatment, and the study is planned to be extended into younger PMM2-CDG patients in the coming months.

Glycomine Announces First Pediatric Patient Dosed in Phase 2 Clinical Study of GLM101, a Potential Treatment for PMM2-CDG

Retrieved on: 
Monday, December 11, 2023

Glycomine, Inc. , a clinical-stage biotechnology company focused on developing new therapies for orphan diseases, announced the first pediatric patient has been dosed in a multi-center open-label Phase 2 study of GLM101, a mannose-1-phosphate replacement therapy, in patients with phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG).

Key Points: 
  • Glycomine, Inc. , a clinical-stage biotechnology company focused on developing new therapies for orphan diseases, announced the first pediatric patient has been dosed in a multi-center open-label Phase 2 study of GLM101, a mannose-1-phosphate replacement therapy, in patients with phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG).
  • “The initiation of dosing in pediatrics is an important milestone for the company,” said Steven Axon, CEO of Glycomine.
  • “We are happy to be a part of an effort that has the potential to make a positive impact on PMM2-CDG patients and the community.”
    Following a dose range finding study that enrolled 10 adult PMM2-CDG patients, this Phase 2 study in pediatric patients with a confirmed diagnosis of PMM2-CDG will be an open-label, 24-week treatment with GLM101 (ClinicalTrials.gov Identifier: NCT05549219 ).
  • The purpose of the study is to evaluate potential pharmacodynamic activity and clinical efficacy, as well as safety, tolerability, and pharmacokinetics of GLM101.

Glycomine Presents at ASHG 2023 Data from Ongoing Natural History Study with Insight into Genetic Mutations and Biomarkers for PMM2-CDG

Retrieved on: 
Thursday, November 2, 2023

Glycomine, Inc. , a biotechnology company focused on developing new therapies for orphan diseases, announced the presentation of summary findings from its ongoing natural history study at the American Society for Human Genetics (ASHG) 2023 Annual Meeting taking place in Washington, DC.

Key Points: 
  • Glycomine, Inc. , a biotechnology company focused on developing new therapies for orphan diseases, announced the presentation of summary findings from its ongoing natural history study at the American Society for Human Genetics (ASHG) 2023 Annual Meeting taking place in Washington, DC.
  • The findings included genotype characterization, combined with the progression of key biomarkers, over two to four years.
  • The effort has resulted in the largest single dataset in patients with PMM2-CDG from a prospective natural history study to date.
  • PB1696) is “Ongoing Natural History Study in Phosphomannomutase 2 Congenital Disorder of Glycosylation (PMM2-CDG): Clinical and Basic Investigations.”

Glycomine Appoints Dr. Rose Marino Chief Medical Officer

Retrieved on: 
Thursday, October 12, 2023

Glycomine, Inc. , a clinical-stage biotechnology company focused on developing new therapies for serious orphan diseases, announced the appointment of Rose Marino, M.D.

Key Points: 
  • Glycomine, Inc. , a clinical-stage biotechnology company focused on developing new therapies for serious orphan diseases, announced the appointment of Rose Marino, M.D.
  • as Chief Medical Officer.
  • Dr. Marino was the medical lead for the palovarotene clinical development program for fibrodysplasia ossificans progressiva, overseeing the pivotal program through final FDA approval.
  • Dr. Marino completed her residency in pediatrics at Loyola University Medical Center, and received an M.D.

Data from Glycomine’s Ongoing Natural History Study Provides Insight into Clinical Measures and Biomarkers for PMM2-CDG

Retrieved on: 
Wednesday, August 30, 2023

The findings included baseline values of key coagulation, liver, metabolic, and endocrine measurements and their evolution over two to four years.

Key Points: 
  • The findings included baseline values of key coagulation, liver, metabolic, and endocrine measurements and their evolution over two to four years.
  • “We are grateful for the time and commitment contributed by the patients and their caregivers and appreciative of the investigators and clinical sites conducting the study,” said Steven Axon, CEO of Glycomine.
  • Every patient currently enrolled has completed more than two years in the study.
  • The effort has resulted in the largest single dataset in patients with PMM2-CDG from a prospective natural history study to date.

Glycomine Announces First Patient Dosed in Phase 2 Clinical Study of GLM101, a Potential Treatment for PMM2-CDG

Retrieved on: 
Monday, January 9, 2023

Glycomine, Inc. , a clinical-stage biotechnology company focused on developing new therapies for orphan diseases, today announced that the first patient has been dosed in a multi-center open-label Phase 2 study (ClinicalTrials.gov Identifier: NCT05549219 ) of GLM101, a targeted mannose-1-phosphate replacement therapy, in adult patients with phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG).

Key Points: 
  • Glycomine, Inc. , a clinical-stage biotechnology company focused on developing new therapies for orphan diseases, today announced that the first patient has been dosed in a multi-center open-label Phase 2 study (ClinicalTrials.gov Identifier: NCT05549219 ) of GLM101, a targeted mannose-1-phosphate replacement therapy, in adult patients with phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG).
  • “The initiation of this Phase 2 study for our lead clinical program is an important milestone for the company.
  • GLM101 is the first potential disease-modifying therapy to be evaluated in PMM2-CDG,” said Steven Axon, CEO of Glycomine.
  • Eva Morava-Kozicz, M.D., Ph.D. , Mayo Clinic, is the Principal Investigator of the Phase 2 study.

Applied Therapeutics Reports Third Quarter 2022 Financial Results

Retrieved on: 
Wednesday, November 9, 2022

NEW YORK, Nov. 09, 2022 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (NASDAQ: APLT) (the Company), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today reported financial results for the third quarter ended September 30, 2022.

Key Points: 
  • NEW YORK, Nov. 09, 2022 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (NASDAQ: APLT) (the Company), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today reported financial results for the third quarter ended September 30, 2022.
  • In the third quarter, we made significant progress across all three of our late-stage programs, said Shoshana Shendelman, PhD, Founder, CEO and Chair of the Board of Applied Therapeutics.
  • In October 2022, the Company announced full enrollment in the Phase 3 registrational ARISE-HF trial studying AT-001 in patients with DbCM.
  • Applied Therapeutics is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need.

Applied Therapeutics Announces Multiple Data Presentations at the American Heart Association Scientific Sessions November 5-7, 2022

Retrieved on: 
Monday, October 31, 2022

AT-001 has been previously studied in a Phase 1/2 study in approximately 120 patients with type 2 diabetes, a subset of which had DbCM.

Key Points: 
  • AT-001 has been previously studied in a Phase 1/2 study in approximately 120 patients with type 2 diabetes, a subset of which had DbCM.
  • Applied Therapeutics is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need.
  • The Company is also developing AT-001, a novel potent ARI, for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart.
  • In light of the significant uncertainties in these forward-looking statements, you should not rely upon forward-looking statements as predictions of future events.

Applied Therapeutics Announces Full Enrollment in the Registrational Phase 3 ARISE-HF Trial of AT-001 in Diabetic Cardiomyopathy

Retrieved on: 
Tuesday, October 25, 2022

The ARISE-HF study is a randomized double-blind placebo-controlled Phase 3 registrational trial.

Key Points: 
  • The ARISE-HF study is a randomized double-blind placebo-controlled Phase 3 registrational trial.
  • The ARISE-HF trial also includes an embedded sub-study in patients with both DbCM and Diabetic Peripheral Neuropathy evaluating the impact of AT-001 on neuropathy progression.
  • Aldose Reductase is a well validated molecular target, and over-activation of this enzyme is known to result in many diabetic complications, including Diabetic Cardiomyopathy and Diabetic Peripheral Neuropathy.
  • AT-001 is an investigational oral, novel, potent Aldose Reductase inhibitor in Phase 3 clinical development for the treatment of Diabetic Cardiomyopathy.

Applied Therapeutics Announces Positive Data Trend in AT-007 ACTION-Galactosemia Kids Pediatric Trial; Trial Will Continue to 18 Months in Blinded Format

Retrieved on: 
Thursday, October 6, 2022

NEW YORK, Oct. 06, 2022 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today announced that the ACTION-Galactosemia Kids Phase 3 trial has demonstrated a trend in clinical benefit favoring AT-007 vs. placebo. The study is designed to evaluate the impact of AT-007 vs. placebo on clinical outcomes in children with Classic Galactosemia, with a review of safety and efficacy every 6 months by a firewalled Data Monitoring Committee (DMC) until the study reaches statistical significance. Review of the data at 12 months of treatment by the DMC indicated that while the study primary endpoint has not yet reached statistical significance, a trend exists favoring AT-007 vs. placebo. The clinical benefit at this early time point was most pronounced in patients with significant deficits in clinical performance at baseline. Safety data demonstrated that AT-007 continues to be safe and well tolerated. The study will proceed in blinded format to the next review at 18 months of treatment. In the meantime, the Company will meet with the EMA to discuss potential submission of an MAA based on existing data for conditional approval.

Key Points: 
  • The clinical benefit at this early time point was most pronounced in patients with significant deficits in clinical performance at baseline.
  • The study will proceed in blinded format to the next review at 18 months of treatment.
  • In the meantime, the Company will meet with the EMA to discuss potential submission of an MAA based on existing data for conditional approval.
  • The data to date in the ACTION-Galactosemia Kids study provides hope for patients and families living with this disease.