Catalent, Cerenis Initiate Phase III Trial of CER-001

Cerenis Therapeutics has advanced its leading clinical candidate, CER-001, into Phase III clinical studies across Europe and North America using Catalent Biologics’ GPEx cell line technology. The phase III TANGO trial was initiated in October 2015, to evaluate efficacy to regress atherosclerosis, and safety of CER-001 in patients with Familial Primary HypoalphAlipoproteinemia (FPHA).

Cerenis Therapeutics’ CER-001 candidate is an engineered complex of recombinant human apoA-I, the major structural protein of High-Density Lipoprotein (HDL) and phospholipids. The development of CER-001 has leveraged Catalent’s proprietary GPEx technology, which creates stable, high-yielding mammalian cell lines with high speed and efficiency. The advantages of applying GPEx technology span from early feasibility studies, to clinical manufacturing, through to commercial scale production. To date, six GPEx based antibody and protein products are approved and marketed and 34 therapeutic candidates are currently in the clinic across the world.

The TANGO trial is a multicenter, randomized, 48-week, double-blind, parallel-group, placebo-controlled study involving thirty patients from several sites across Europe, Canada, the United States and other countries based on availability of patients with this rare orphan disease.

“The partnership established with Catalent Biologics has empowered us to resolve a 30 year challenge: the production of a commercially viable HDL mimetic, by generating a proprietary CHO cell line with a high apoA-I expression via secretion meeting the demanding quality standards we require for our clinical study drugs,” said Jean-Louis Dasseux, founder and chief executive officer, Cerenis. “The Phase III TANGO trial will fully benefit from GPEx technology and we are convinced its application to the production of CER-001 batches will ensure better product safety and product quality. These features are particularly encouraging with regards to CER-001’s filing for future market approval expected in 2018.”

“GPEx technology was designed to offer clients and partners advantages over conventional cell line engineering systems, including increased flexibility and higher, more stable yields,” said Mike Riley, vice president and general manager, Catalent Biologics. “We are pleased to have worked with Cerenis to help advance their life-changing therapy into Phase III studies, and this represents another key milestone for our GPEx platform as an enabling technology.”

Catalent performs GPEx programs at its state-of-the-art biomanufacturing facility in Madison, WI, which was completed in June 2013. Designed for flexible cGMP production from 10 L up to 1,000 L, and non-GMP production up to 250 L, the site features extensive single-use technologies and unidirectional flow to maximize efficiency and safety.