odiparcil (IVA336) / Inventiva - LARVOL DELTA

Mucopolysaccharidosis VI: Therapeutic strategies and perspectives. (PubMed, Mol Genet Metab) - "Additionally, substrate reduction therapy with odiparcil, immunomodulation, and stop codon read-through therapies have been explored in MPS VI. Future directions in MPS VI should include targeting cellular alterations, such as mitochondrial dysfunction, exploring cartilage-targeting alternatives, and implementing pharmacological chaperones. This manuscript highlights recent progress and emerging strategies for treating MPS VI."

Journal • Review • Bone Marrow Transplantation • Gene Therapies • Immunology • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases • Transplantation

Reduction of lysosome abundance and GAG accumulation after odiparcil treatment in MPS I and MPS VI models. (PubMed, Mol Genet Metab Rep) - "Furthermore, odiparcil was shown to be effective in reducing CS, DS, and HS concentrations in liver and eye, as representative organs, in MPS VI and MPS I mice treated with 3 doses of odiparcil over 3 and 9 months, respectively. In conclusion, our data demonstrates odiparcil efficiently reduced lysosome abundance and tissue GAG concentrations in in vitro and in vivo models of MPS VI and MPS I and has potential as a treatment for these disorders."

Journal • Hurler Syndrome

Elucidating the functional impact of G137V and G144R variants in Maroteaux Lamy's Syndrome by Molecular Dynamics Simulation. (PubMed, Mol Divers) - "The native along with G137V and G144R structures were fixed as the receptors and subjected to Molecular docking with the small molecule Odiparcil to analyze the binding efficiency and the varied interactions of the receptors towards the drug...The overall study indicates that the drug has similar therapeutic towards the native and variant based on the higher binding affinity and also the complexes show stability with an average of 0.2 nm RMS value. This can aid in the future development therapeutics for the Maroteaux Lamy syndrome."

Journal • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

Oral Treatment for Mucopolysaccharidosis VI: Outcomes of the First Phase IIa Study with Odiparcil. (PubMed, J Inherit Metab Dis) - "This study confirmed the mechanism of action and established the safety of odiparcil with clinical beneficial effects after only a short treatment duration in an advanced stage of disease. Further assessment of odiparcil in younger patients is needed."

Clinical • Journal • P2a data • Cardiovascular • Lysosomal Storage Diseases • Metabolic Disorders • Ophthalmology • Pain • Rare Diseases

MPS VI associated ocular phenotypes in an MPS VI murine model and the therapeutic effects of odiparcil treatment. (PubMed, Mol Genet Metab) - "Analysis of GAG types accumulating in the MPS VI Arsb eyes demonstrated major contribution of DS and CS, with some increase in heparan sulphate (HS) as well and all were reduced with odiparcil treatment. Taken together, we further reveal the potential of odiparcil to be an effective therapy for eye phenotypes associated with MPS VI disease."

Journal • Preclinical • Lysosomal Storage Diseases • Metabolic Disorders • Ophthalmology • Rare Diseases

Odiparcil, a potential glycosaminoglycans clearance therapy in mucopolysaccharidosis VI-Evidence from in vitro and in vivo models. (PubMed, PLoS One) - "Our data demonstrate that odiparcil effectively diverts the synthesis of cellular glycosaminoglycans into secreted soluble species and this effect can be used for reducing cellular and tissue GAG accumulation in MPS VI models. Therefore, our data reveal the potential of odiparcil as an oral GAG clearance therapy for MPS VI patients."

Journal • Preclinical • Gene Therapies • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases • Transplantation

iMProveS: A Study in MPS VI to Assess Safety and Efficacy of Odiparcil (clinicaltrials.gov) - P2a; N=24; Recruiting; Sponsor: Inventiva Pharma; Not yet recruiting ➔ Recruiting

Enrollment open • Biosimilar • Gene Therapies

Current and Emerging Direct Oral Anticoagulants: State-of-the-Art. (PubMed, Semin Thromb Hemost) - "Several drugs (apixaban [BMS-562247], dabigatran [BIBR953], edoxaban [DU-1766], rivaroxaban [BAY 59-7939]) have already received widespread approval by national or supranational medicinal agencies. This narrative article provides a state-of-the-art for these and for several other DOACs at different stages of clinical evaluation (betrixaban, darexaban, eribaxaban, letaxaban, nokxaban), and certain others whose development has been discontinued (AZD-0837, fidexaban, LY517717, odiparcil, otamixaban, TTP889, and ximelagatran)...These factors contribute to challenging the minds of physicians, who may find difficulty navigating their way through multiple indications, different pharmacological profiles, various side effects, and specific drug-to-drug interactions. Such considerations also burden laboratory professionals, who may face organizational and economic challenges in developing and/or implementing multiple assays to assess the pharmacodynamics (effect on coagulation) or..."

Journal

iMProveS: A Study in MPS VI to Assess Safety and Efficacy of Odiparcil (clinicaltrials.gov) - P2a; N=20; Completed; Sponsor: Inventiva Pharma; Recruiting ➔ Completed; Trial completion date: Mar 2020 ➔ Oct 2019

Clinical • Trial completion • Trial completion date

"#ivaa odiparcil phase 2a results By end of 2019! The comeback of this company. And Lanifibranor in h1 2020 (NASH)" (@niekjes1)

P2a data

"$VRP COPD ph2a success for the DPI formulation of RPL554, $APLHA consolidates after Tasly bond conversion+some insider sales, $IVA RPDD for odiparcil, $OSE new IO bi-spec platform, and a bunch of earnings. Also a 2nd HIV patient got in abnormally LT remission after an HSCT." (@BiotechRadar)

Clinical • P2 data • P2a data