WACKER BIOTECH Delivers Active Ingredient to Treat Dwarfism in Children

Wacker Biotech has reached a milestone in its support of Ascendis Pharma’s TransCon™ CNP program. Following the success of clinical phase 1, Ascendis Pharma has filed an application in the USA for initiation of a global phase 2 trial for TransCon™ CNP in children with achondroplasia, the most common form of dwarfism. A peptide conjugated with TransCon™ technology, the pharmaceutical is manufactured by Wacker Biotech GmbH in Jena, central Germany. Roughly 250,000 patients worldwide suffer from achondroplasia, for which neither the FDA (U.S. Food and Drug Administration) nor the EMA (European Medicines Agency) has approved a therapy yet. Danish biotech company Ascendis Pharma is working to close this gap and meet significant medical needs by developing TransCon™ CNP. The pharmaceutical is designed to treat all aspects of achondroplasia with a weekly dose. Ascendis Pharma is being supported by Wacker Biotech GmbH, which Ascendis has contracted to manufacture the active ingredient TransCon™ CNP. Following the success of clinical phase 1, an application for initiation of a global phase 2 trial for TransCon™ CNP in children with achondroplasia has been filed at the FDA.

“We are delighted to be able to support Ascendis Pharma in supplying the clinical trials for TransCon™ CNP in children with achondroplasia. Wacker Biotech GmbH sees itself as a reliable partner to pharmaceutical manufacturers and biotech companies in the development and production of biologics to international GMP standards,” explained Dr. Susanne Leonhartsberger, who became president of WACKER BIOSOLUTIONS on April 1, 2020. “We are experts in bioengineering techniques for producing active ingredients on small and large scales – for clinical development phases for approving a drug and for supplying the commercial market afterward.”

Wacker Biotech has been cooperating with Ascendis Pharma since 2017. The process for making the active candidate based on Ascendis Pharma’s proprietary TransCon™ technology was successfully transferred to Jena-based Wacker Biotech GmbH at the start of this partnership. The active TransCon™ CNP is manufactured there in compliance with pharmaceutical GMP (Good Manufacturing Practice) requirements to ensure the high quality necessary for the approval of clinical trials or for market approval by the FDA or EMA. Wacker Biotech made the materials for both the successfully completed phase 1 trial and the phase 2 trial for which an application has been filed.

TransCon™ CNP is a sustained-release C-type natriuretic peptide (CNP) prodrug in development for children with achondroplasia. Phase 1 results demonstrated that TransCon™ CNP provided continuous exposure to CNP with a pharmacokinetic profile designed to maximize efficacy with once-weekly dosing. The ACCOMPLISH trial that has now been applied for is a global, phase 2, randomized, double-blind, placebo-controlled trial that will enroll approximately 60 children, aged 2 to 10, with achondroplasia. The trial was designed to prove the safety and efficacy of TransCon™ CNP in the treatment of dwarfism in children.