Orphalan announces data from secondary analyses of the CHELATE trial for maintenance patients with Wilson’s Disease

Orphalan announces data from secondary analyses of the CHELATE trial for maintenance patients with Wilson’s Disease




Orphalan announces data from secondary analyses of the CHELATE trial for maintenance patients with Wilson’s Disease

Orphalan announces data from secondary analyses of the CHELATE trial for maintenance patients with Wilson’s Disease

— Data from the company’s CHELATE trial to be presented today during an oral presentation at EASL’s The International Liver Congress™ 2022

Paris, France 22 June, 2022 — Orphalan SA (“Orphalan” or “the Company”), an international orphan drug development and commercialisation company, today announces that it will present data on secondary analyses of the company’s CHELATE study for the treatment of Wilson’s disease at the European Association for the Study of the Liver (EASL) International Liver Congress™ 2022 (ILC) taking place June 22 – 26, 2022 in London, United Kingdom.

The CHELATE trial was the first prospective randomised trial comparing d-Penicillamine with trientine tetrahydrochloride (TETA-4HCl) and supported Orphalan’s successful application to the United States Food and Drug Administration (FDA) for approval of Cuvrior™, an oral TETA-4HCl formulation for the treatment of adult patients with stable Wilson’s disease who are de-coppered and tolerant to penicillamine. It was the first treatment for Wilson’s disease to be approved by the FDA in over five decades.

In an oral presentation at 10am BST / 11am CEST on 24th June, Professor Michael Schilsky will present data from a secondary analysis of the CHELATE Trial. He will describe the intra-patient variability of urinary copper excretion (24-hour UCE) and compare the clinical utility of 24-hour UCE with a newly developed non-ceruloplasmin copper assay (NCC-sp) in the monitoring of Wilson’s disease patients being treated with d-Penicillamine and TETA-4HCl. The non-ceruloplasmin copper assay using liquid chromatography and ICP mass spectroscopy was developed for the Orphalan-sponsored CHELATE trial.

Orphalan will also host an industry symposium titled “Chelating Evidence: Achieving More for Patients with Wilson’s Disease” at 12:30-13:30 BST / 13:30-14:30 CEST on 24th June (Capital Suite 12) featuring Professor Anil Dhawan, Dr. Zoe Mariño, Dr. Olivier Guillaud, Professor David Cassiman and Professor Michael Schilsky. This will include an introduction from Professor Dhawan, and discussions on ‘Short- and Long-Term Treatment Aims for Wilson’s Disease’, ‘The Patient Experience: What Matters to Them’ and ‘Evidence Based Management Options in Wilson’s Disease’, followed by a Q&A session.

Dr. Naseem Amin, Chief Executive Officer at Orphalan, commented: “The CHELATE trial was pivotal in Orphalan gaining U.S. FDA approval for Cuvrior™, which we look forward to launching and commercializing in the U.S. We are committed to sharing what we’ve learned about Wilson’s disease and our treatment so that healthcare professionals can make educated choices in delivering the best possible patient care.”

The accepted abstracts for presentations are:

  • Poster presentation: Identifying clinically stable Wilson disease patients: Observations and Lessons from the CHELATE Trial

In this poster, exploratory secondary analyses of CHELATE trial dataset was performed to assess current clinical and laboratory methods of determining stability in Wilson’s disease patients receiving maintenance therapy.

Authors: Professor Michael Schilsky on behalf of CHELATE Trial investigators

Date and Time: 9am to 6pm BST on 23rd June.

  • Oral presentation: Clinical utility of non-ceruloplasmin copper (NCC) determined by copper speciation for monitoring Wilson disease therapy: comparative data analysis with 24-hour urinary copper excretion from the CHELATE trial

Author: Professor Michael Schilsky on behalf of CHELATE Trial Investigators

Date and Time: 10am BST on 24th June

-ENDS-

Notes to Editors

About Cuvrior™ (Trientine Tetrahydrochloride)

Cuvrior™ (trientine tetrahydrochloride) is an oral trientine formulation. In the U.S., trientine tetrahydrochloride has been granted Orphan Drug Designation for the treatment of Wilson’s disease excluding patients intolerant of penicillamine. It has been approved under the 505(b)(2) pathway for the treatment of adult stable Wilson’s disease patients who are successfully de-coppered and tolerant to penicillamine. The 505(b)(2) regulatory pathway is a type of New Drug Application (NDA).

About Orphalan

Orphalan is an international orphan drug development and commercialisation company. The company delivers worldwide innovative therapies for people living with orphan diseases and is a pioneer in the space. Orphalan was founded in 2011 and has launched Cuprior® across Europe with its own commercial organization. For more information visit www.orphalan.com and follow us on LinkedIn.

For more information, please contact:

Orphalan:

Tel: +33 (0)1 42 49 82 64

info@orphalan.com

Consilium Strategic Communications:

Mary-Jane Elliott, Allison Connolly, Genevieve Wilson, Alex Gunter

Tel: +44 (0) 203 709 5700

orphalan@consilium-comms.com