July 25, 2017 3:01 PM ET


Company Overview of Valerion Therapeutics, LLC

Company Overview

Valerion Therapeutics, LLC is a biotechnology company that focuses on developing bio-therapeutics for orphan genetic diseases. It generates product candidates utilizing substrates (proteins, enzymes, oligonucleotides, etc.) to target specific tissues via an antibody with cell-penetrating properties dependent on a tissue-localized membrane transporter. The company utilizes an antibody-mediated delivery platform for the intracellular delivery of various active therapeutic payloads via a transport mechanism that is present in muscle and neurons and elevated in certain types of cancer. Valerion Therapeutics, LLC was formerly known as 4s3 Bioscience, Inc. The company was founded in 2007 and is ba...

100 Main Street

Suite 110

Concord, MA 01742

United States

Founded in 2007



Key Executives for Valerion Therapeutics, LLC

Chief Executive Officer, President and Director
Founder, Chief Scientific Officer, Secretary, Treasurer and Director
Compensation as of Fiscal Year 2017.

Valerion Therapeutics, LLC Key Developments

Valerion Therapeutics Initiates VAL-1221 Dosing in Patients with Pompe Disease

Valerion Therapeutics, announced that it has initiated dosing in a Phase 1/2 clinical trial evaluating VAL-1221 in patients with late-onset Pompe disease. VAL-1221 is a novel fusion protein that combines Valerion's antibody-mediated delivery technology with recombinant human acid alpha-glucosidase (rhGAA) to uniquely target both lysosomal and extra-lysosomal glycogen in the cytoplasm through enhanced intracellular delivery to affected tissues. By leveraging two uptake mechanisms, rather than one, VAL-1221 offers the potential for improved glycogen clearance and better patient outcomes. The Company expects to report top-line data from the Phase 1/2 study in the fourth quarter of 2017. The Phase 1/2 trial is a randomized, international, parallel active control, single- and multiple-ascending dose escalation study designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of VAL-1221 in up to 12 ambulatory and ventilator-free patients with late-onset Pompe disease previously treated with Myozyme or Lumizyme. Exploratory efficacy endpoints include the six-minute walk test, pulmonary function testing, quantitative and qualitative muscle testing and patient-reported outcomes. Following the three-month treatment period, responding patients will be eligible to roll-over to an open-label extension study. The trial will initially be conducted at Duke University Medical Center and The National Hospital for Neurology and Neurosurgery of London. VAL-1221, is an investigational fusion protein therapeutic that combines Valerion's antibody-mediated delivery technology with recombinant human acid alpha-glucosidase (rhGAA) to drive improved delivery of rhGAA to muscle. It is uniquely designed to target both lysosomal and extra-lysosomal glycogen in the cytoplasm. In cultured Pompe disease fibroblasts and in Pompe (GAA-deficient) mice, VAL-1221 was found to reduce lysosomal glycogen accumulation as effectively as current enzyme replacement therapies, and penetrated living cells independent of the mannose-6-phosphate receptor (M6PR), the mechanism of cell entry seen with current treatments that direct the ERT enzyme to the lysosome. VAL-1221 also enters cells through an equilibrative nucleoside transporter, ENT-2, and was shown to clear cytoplasmic glycogen in a hypoxia model. VAL-1221 is currently in clinical development for the treatment of patients with late-onset Pompe disease.

Valerion Therapeutics, LLC Announces Positive Results of Non-Clinical Research Study at SSIEM Annual Symposium

Valerion Therapeutics, LLC announced positive results from a non-clinical "proof of concept" pilot study for a novel humanized antibody and acid alpha glucosidase (GAA) fusion candidate for the treatment of Pompe disease. The studies performed with Valerion's proprietary product candidate [VAL-1221 (3E10Fab-GAA)] are part of a broad research collaboration between Valerion Therapeutics and Duke University. Valerion Therapeutics, which is part of the Alopexx Enterprises portfolio, is a developer of bio-therapeutics for neuromuscular genetic disorders with limited or no current treatment options. VAL-1221 demonstrated efficacy in both cultured Pompe patient fibroblasts and in Pompe (GAA-deficient) mice, according to results announced at the Society for the Study of Inborn Errors of Metabolism 2015 Annual Symposium in Lyon, France. The pilot results were presented by the principal investigator of the study, Baodong Sun, PhD, of Duke University. Importantly, VAL-1221 was observed in the study to penetrate living cells partially-independent of M6PR and not co-localize with lysosomal marker LAMP2. The results suggest that VAL-1221 is capable of entering the cytoplasm and potentially functioning outside the lysosome.

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