Wacker Biotech Provides Active Ingredient to Treat Achondroplasia in Children for Ascendis Pharma Global Phase-2 Trial
Munich / Jena, Jan 08, 2020
Wacker Biotech has reached a major milestone in supporting Ascendis Pharma’s TransConTM CNP program. Following the success of clinical phase 1, Ascendis Pharma has completed an application in the U.S. for initiation of a global phase 2 trial for TransConTM CNP in children with achondroplasia, the most common form of dwarfism. TransConTM CNP drug substance, a transiently conjugated peptide using TransConTM technology, is being made by Wacker Biotech GmbH in Jena, Germany.
Roughly 250,000 patients worldwide suffer from achondroplasia, the most common form of dwarfism, for which the FDA (U.S. Food and Drug Administration) and EMA (European Medicines Agency) have not yet approved any therapy. Danish biotech company Ascendis Pharma is working to close this gap and meet significant medical needs by developing TransConTM CNP, which is designed to address all aspects of achondroplasia by providing continuous CNP (C-type natriuretic peptide) exposure with a once-weekly dose. Ascendis Pharma is being supported by Wacker Biotech GmbH, which Ascendis Pharma has contracted to manufacture TransConTM CNP. Following the success of clinical phase 1, an application for initiation of a global phase 2 trial for TransConTM 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 TransConTM CNP in children with achondroplasia. Wacker Biotech sees itself as a reliable partner to pharmaceutical and biotech companies for developing and producing biologics to international GMP standards,” said Dr. Susanne Leonhartsberger, managing director of Wacker Biotech GmbH, a WACKER Group subsidiary based in Jena. “We are expert in the bioengineered production of active ingredients on a small and large scale, for clinical development phases for approving a drug, as well as for supplying the commercial market afterward,” she added.
Wacker Biotech has been working with Ascendis Pharma since 2017. The process for making the candidate based on Ascendis Pharma’s proprietary TransCon technology was transferred successfully to Wacker Biotech GmbH in Jena at the start of this partnership. TransConTM 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 material both for the successful phase 1 trial and for the phase 2 trial that has now been applied for.
TransConTM CNP is a long-acting prodrug of C-type natriuretic peptide (CNP) in development for children with achondroplasia. Phase 1 results demonstrated that TransConTM 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.
It is designed to demonstrate the safety and efficacy of TransConTM CNP in children with achondroplasia.
About Wacker Biotech
Wacker Biotech GmbH and Wacker Biotech B.V. are full-service contract manufacturers of biopharmaceutical proteins based on microbial systems. The companies’ services range from molecular biology, analytical services and process development through to the GMP-compliant manufacture of clinical test samples and pharmaceutical actives, live microbial products and vaccines for the commercial market. Wacker Biotech has three GMP-compliant, FDA- and EMA-certified production plants in Jena and Halle in Germany and in Amsterdam in the Netherlands. Above all, Wacker Biotech offers proprietary technologies that satisfy market needs for cost-efficient production and maximum quality. Wacker Biotech GmbH and Wacker Biotech B.V. are wholly-owned subsidiaries of the Munich-based WACKER Group.
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