GM1

Passage Bio Reports Fourth Quarter and Full-Year 2023 Financial Results and Provides Recent Business Highlights

Retrieved on: 
Monday, March 4, 2024

"In 2023, we achieved a significant milestone by announcing encouraging data from three patients in Cohort 1 of our FTD-GRN program.

Key Points: 
  • "In 2023, we achieved a significant milestone by announcing encouraging data from three patients in Cohort 1 of our FTD-GRN program.
  • We eagerly anticipate a catalyst-rich 2024, supported by our robust balance sheet, bringing us closer to improving patient outcomes across neurodegenerative diseases."
  • Additionally, Dose 1 of PBFT02 continued to be generally well-tolerated in patients who received an enhanced steroid regimen for immunosuppression.
  • Cash Position: Cash, cash equivalents and marketable securities were $114.3 million as of December 31, 2023, as compared to $189.6 million as of December 31, 2022.

Gain Therapeutics’ CEO Matthias Alder Issues Letter to Shareholders and Provides Operational Update

Retrieved on: 
Wednesday, January 31, 2024

In 2023, we advanced GT-02287 through preclinical development and initiated the company’s first clinical trial in September 2023 on time and on plan.

Key Points: 
  • In 2023, we advanced GT-02287 through preclinical development and initiated the company’s first clinical trial in September 2023 on time and on plan.
  • The dose escalation of the SAD phase is underway, and the MAD phase of the study is expected to begin in Q1 2024.
  • In 2023, we made several data presentations of results of our GBA1 program in preclinical models of Parkinson’s disease and Alzheimer’s disease.
  • Xavi is leaving the world of academia as a professor at the University of Barcelona and his part-time engagement with Gain to join a major pharmaceutical company.

National Tay-Sachs & Allied Diseases Association Hosts First of Its Kind Drug Development Meeting for GM2

Retrieved on: 
Thursday, February 1, 2024

BOSTON, Feb. 1, 2024 /PRNewswire/ -- National Tay-Sachs & Allied Diseases Association (NTSAD), leader in the worldwide fight to treat and cure Tay-Sachs, Canavan, GM1, and Sandhoff diseases, is hosting the first-ever, Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting for GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases) on February 15, 2024, 9:30am-3pm Eastern.

Key Points: 
  • BOSTON, Feb. 1, 2024 /PRNewswire/ -- National Tay-Sachs & Allied Diseases Association (NTSAD), leader in the worldwide fight to treat and cure Tay-Sachs, Canavan, GM1, and Sandhoff diseases, is hosting the first-ever, Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting for GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases) on February 15, 2024, 9:30am-3pm Eastern.
  • This input can inform FDA's decisions and oversight during drug development and their review of marketing applications.
  • Nearly 30 people will share their stories and experiences with infantile, juvenile, or late onset GM2, including adult patients, parents, caregivers, and bereaved family members.
  • This virtual meeting will be livestreamed via YouTube, open to the public, free to attend, but advance registration is required.

Azafaros to present data from PRONTO study in patients with GM1 and GM2 gangliosidoses at the 20th annual WORLDSymposium™

Retrieved on: 
Tuesday, January 23, 2024

As well as data from PRONTO, Azafaros will present details of its Phase 2 RAINBOW study, aimed at investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of nizubaglustat in patients with GM2 gangliosidosis or NPC.

Key Points: 
  • As well as data from PRONTO, Azafaros will present details of its Phase 2 RAINBOW study, aimed at investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of nizubaglustat in patients with GM2 gangliosidosis or NPC.
  • Title: A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO) - patients’ and caregivers’ assessments.
  • Title: A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO)- baseline clinical data.
  • Title: A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO) - evaluation of different assessments.

Passage Bio Announces Promising Initial Data From Phase 1/2 Clinical Trial of PBFT02 in FTD-GRN and Updated Strategic Priorities

Retrieved on: 
Wednesday, December 20, 2023

Additionally, the company shared updated strategic priorities aimed at further optimizing its portfolio for the treatment of neurodegenerative conditions.

Key Points: 
  • Additionally, the company shared updated strategic priorities aimed at further optimizing its portfolio for the treatment of neurodegenerative conditions.
  • "We are proud to announce initial clinical data from our upliFT-D clinical trial, which showcases the ability of PBFT02 to elevate CSF progranulin to supraphysiologic levels at the lowest tested dose, Dose 1, up to six months post-treatment.
  • The upliFT-D clinical trial evaluates PBFT02 as a single dose delivered via intra-cisterna magna (ICM) injection.
  • These strategic priorities and clinical milestones underscore Passage Bio's dedication to advancing cutting-edge, one-time genetic medicines and protecting patients and families against loss in neurodegenerative conditions.

Azafaros’ Phase 2 RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients, is now fully enrolled

Retrieved on: 
Monday, December 11, 2023

The success in enrollment is a major milestone for Azafaros’ lead asset, nizubaglustat, which is being developed to treat patients with GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC).

Key Points: 
  • The success in enrollment is a major milestone for Azafaros’ lead asset, nizubaglustat, which is being developed to treat patients with GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC).
  • The 12-weeks study is a randomised, double-blind, placebo-controlled, multi-center trial which evaluates the safety, tolerability, pharmacokinetics, and pharmacodynamics across two doses of nizubaglustat in patients with GM2 gangliosidosis and NPC.
  • The trial is being conducted at sites across Brazil, with safety, pharmacokinetic and pharmacodynamic data from ongoing patient follow-ups expected in mid-2024.
  • We would like to thank all patients, their families, patient organisations and clinicians for their participation and support in this important research.”

Gain Therapeutics Announces Preclinical Data Showing Restoration of Enzymatic Function with Novel Allosteric Regulators in GM1 Gangliosidosis Model

Retrieved on: 
Friday, December 1, 2023

BETHESDA, Md., Dec. 01, 2023 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc., (Nasdaq: GANX), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announced the publication of preclinical data identifying a novel class of small molecule allosteric regulators that demonstrate therapeutic potential for galactosidase beta 1 (GLB1)-related lysosomal storage disorders (LSDs), including GM1 gangliosidosis. The study, “Validation of a highly sensitive HaloTag-based assay to evaluate the potency of a novel class of allosteric β-Galactosidase correctors,” was published in PLOS ONE.

Key Points: 
  • The study, “Validation of a highly sensitive HaloTag-based assay to evaluate the potency of a novel class of allosteric β-Galactosidase correctors,” was published in PLOS ONE.
  • GM1-gangliosidosis is an inherited, progressive disorder characterized by the degeneration of brain and spinal cord cells, leading to muscle weakness, skeletal abnormalities, dystonia, and vision problems.
  • Mutations in the GLB1 gene significantly reduce the activity and function of the lysosomal hydrolase enzyme β-galactosidase (β-Gal) due to protein misfolding.
  • Additionally, there are no available disease-modifying treatments for GM1 gangliosidosis, with available therapies only focused on reducing symptoms and improving quality of life.

Passage Bio Reports Third Quarter 2023 Financial Results and Provides Recent Business Highlights

Retrieved on: 
Monday, November 13, 2023

Robust balance sheet to support achievement of meaningful clinical milestones, with cash runway into Q4 2025

Key Points: 
  • Robust balance sheet to support achievement of meaningful clinical milestones, with cash runway into Q4 2025
    PHILADELPHIA, Nov. 13, 2023 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for central nervous system (CNS) disorders, today reported financial results for the third quarter ended September 30, 2023, and provided recent business highlights.
  • "Our team remains focused on execution, and this past quarter marked another stride toward achieving key milestones across our two lead clinical programs.
  • Initial data focused on safety and CSF progranulin levels from three Cohort 1 patients is expected in the fourth quarter of 2023.
  • Present initial safety and biomarker data from three Cohort 1 patients in upliFT-D clinical trial for FTD in Q4 2023.

Passage Bio Reports Second Quarter 2023 Financial Results and Provides Recent Business Highlights

Retrieved on: 
Monday, August 7, 2023

“We continued to make steady progress advancing our lead clinical programs in the second quarter and further improved our financial strength, extending our cash runway into the fourth quarter of 2025.

Key Points: 
  • “We continued to make steady progress advancing our lead clinical programs in the second quarter and further improved our financial strength, extending our cash runway into the fourth quarter of 2025.
  • Additionally, we are thrilled to have already dosed the first patient at Dose 3, demonstrating effective execution across the program.
  • In FTD, we are excited by emerging data from initial treated patients, which indicates translation from preclinical models into the clinic.
  • General and Administrative (G&A) Expenses: G&A expenses were $8.1 million for the quarter ended June 30, 2023, as compared to $13.0 million as of June 30, 2022.

Passage Bio Announces Promising Interim Clinical Data from First Eight Patients with GM1 Gangliosidosis in Imagine-1 Study

Retrieved on: 
Monday, August 7, 2023

Imagine-1 is a Phase 1/2, global, open-label, dose-escalation study of the AAVhu68 gene therapy PBGM01 delivered by intra-cisterna magna (ICM) injection in six cohorts of pediatric subjects with early and late infantile GM1 Gangliosidosis (GM1).

Key Points: 
  • Imagine-1 is a Phase 1/2, global, open-label, dose-escalation study of the AAVhu68 gene therapy PBGM01 delivered by intra-cisterna magna (ICM) injection in six cohorts of pediatric subjects with early and late infantile GM1 Gangliosidosis (GM1).
  • GM1 is a rare, fatal lysosomal storage disease in which mutations in the GLB1 gene result in very low activity of the enzyme beta-galactosidase (β-Gal).
  • "We are highly encouraged by the compelling data emerging from our Imagine-1 study, with results underscoring the potential of PBGM01 to be a transformative therapy for GM1 patients,” said William Chou, M.D., president and chief executive officer of Passage Bio.
  • The amended study protocol has been approved at several clinical trial sites, including in Brazil, Canada, Turkey and the United States.