GLM101 / Glycomine - LARVOL DELTA

A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG (clinicaltrials.gov) - P2/3 | N=50 | Recruiting | Sponsor: Glycomine, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open

24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Participants With PMM2-CDG (clinicaltrials.gov) - P2 | N=44 | Active, not recruiting | Sponsor: Glycomine, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Pediatrics • PMM2

Glycomine Announces $115 Million Series C Financing to Advance Lead Drug Candidate, GLM101, into a Phase 2b Clinical Trial for PMM2-CDG (Businesswire) - "Glycomine, Inc...announced a $115 million Series C financing to advance its lead candidate, GLM101, into a Phase 2b clinical trial. The financing was led by CTI Life Sciences Fund, funds managed by abrdn Inc., and Advent Life Sciences, alongside continued investment from existing investors, Novo Holdings, Sanofi Ventures, Abingworth, RiverVest Venture Partners, Sanderling Ventures, Chiesi Ventures, Remiges Ventures, and Asahi Kasei Ventures."

Financing • Genetic Disorders

A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants with PMM2-CDG (clinicaltrials.gov) - P2/3 | N=50 | Not yet recruiting | Sponsor: Glycomine, Inc.

New P2/3 trial

24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Participants with PMM2-CDG (clinicaltrials.gov) - P2 | N=44 | Recruiting | Sponsor: Glycomine, Inc. | Trial completion date: Apr 2025 ➔ Nov 2025 | Trial primary completion date: Apr 2025 ➔ Nov 2025

Trial completion date • Trial primary completion date • Pediatrics • PMM2

Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients (clinicaltrials.gov) - P2 | N=40 | Enrolling by invitation | Sponsor: Glycomine, Inc.

New P2 trial

Glycomine Receives FDA Fast Track Designation for GLM101 for the Treatment of PMM2-CDG (Businesswire) - "Glycomine, Inc...announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for GLM101, a mannose-1-phosphate replacement therapy in development for the treatment of patients with phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG)."

Fast track • Genetic Disorders

NEUROLOGICAL IMPROVEMENT IN THREE ADULT PATIENTS WITH PMM2-CDG AFTER TREATMENT WITH GLM101, A LIPOSOMAL MANNOSE-1 PHOSPHATE (M1P) SUBSTRATE REPLACEMENT POTENTIAL THERAPY (SSIEM 2024) - P2 | "Neurological symptoms in adults suffering PMM2-CDG importantly alter their autonomy and functionality. Treatment with GLM101 was well tolerated and resulted in clinically significant improvements in cerebellar syndrome for the three patients studied. Further longitudinal studies in a larger population are required to confirm this finding."

Clinical • Ataxia • Cardiovascular • Metabolic Disorders • Movement Disorders • Pain • PMM2

Treatment of congenital disorders of glycosylation: An overview. (PubMed, Mol Genet Metab) - "Innovative therapies, targeting both the root cause and resulting manifestations, have transitioned from the research stage to practical application. The present paper aims to provide a detailed overview of these exciting developments and the rising concepts that are used to treat these ultra-rare diseases."

Journal • Rare Diseases

In vitro treatment with liposome-encapsulated Mannose-1-phosphate restores N-glycosylation in PMM2-CDG patient-derived fibroblasts. (PubMed, Mol Genet Metab) - "In vivo characterization of GLM101 revealed its favorable pharmacokinetics, liver-targeted biodistribution, and tolerability profile with achieved systemic concentrations significantly greater than its effective in vitro potency. Taken as a whole, the results described in this report support further exploration of GLM101's safety, tolerability, and efficacy in PMM2-CDG patients."

Journal • Preclinical • Metabolic Disorders • ICAM1 • PMM2 • TNFA

Liposome-encapsulated mannose-1-phosphate therapy improves global N-glycosylation in different congenital disorders of glycosylation. (PubMed, Mol Genet Metab) - "ALG11-CDG fibroblasts also showed increase in high mannose glycopeptides upon treatment; however, the improvement was not as dramatic as the other two CDG. Overall, our findings suggest that treatment with GLM101 overcomes the genetic block in the glycosylation pathway and can be used as a potential therapy for CDG with enzymatic defects in early steps in protein N-glycosylation."

Journal • Review • ALG1 • PMM2

Characterization of the preclinical and clinical pharmacokinetics of GLM101, A liposome-encapsulated mannose-1-phosphate drug candidate for the treatment of patients with PMM2-CDG (SIMD 2024) - "These studies show that preclinical pharmacokinetics of GLM101 accurately predicted those observed in the clinic when this drug candidate was administered to adult healthy volunteers. The safety, pharmacokinetics, and pharmacodynamics of GLM101 is currently being assessed in an open label Phase 2 clinical study with adult and adolescent PMM2-CDG patients."

PK/PD data • Preclinical • PMM2

INfusion-related reactions are associated with pre-existing anti-PEG antibodies detected while assessing the immunogenicity of GLM101, a liposome-encapsulated mannose-1-phosphate drug candidate for the treatment of patient with PMM2-CDG (SIMD 2024) - "IARs are common AEs associated with PEG-containing liposomal and other lipid nanoparticle (LNP) therapies. The results described from this study suggest a correlation between the presence of pre-existing PEG-Ab and the development of IARs in healthy adult subjects receiving an IV infusion of GLM101."

Clinical • PMM2

24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Participants With PMM2-CDG (clinicaltrials.gov) - P2 | N=44 | Recruiting | Sponsor: Glycomine, Inc. | Trial completion date: Sep 2024 ➔ Apr 2025 | Trial primary completion date: Jun 2024 ➔ Apr 2025

Trial completion date • Trial primary completion date • Pediatrics • PMM2

24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Adults With PMM2-CDG (clinicaltrials.gov) - P2 | N=6 | Recruiting | Sponsor: Glycomine, Inc. | Trial completion date: Sep 2023 ➔ Sep 2024 | Trial primary completion date: Aug 2023 ➔ Jun 2024

Trial completion date • Trial primary completion date • PMM2

12-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Adults With PMM2-CDG (clinicaltrials.gov) - P2 | N=6 | Recruiting | Sponsor: Glycomine, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open

12-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Adults With PMM2-CDG (clinicaltrials.gov) - P2 | N=6 | Not yet recruiting | Sponsor: Glycomine, Inc.

New P2 trial