Catalent & Valerion Therapeutics to Collaborate

Catalent Pharma Solutions recently announced it is to collaborate with Valerion Therapeutics, LLC to develop Valerion’s two lead product candidates. The collaboration will focus on the delivery of muscle-protein fusions, and treatment of multiple indications, in the fields of muscular dystrophies, glycogen storage diseases, myopathies, and enzyme-deficiency disorders.

Under the agreement, Catalent will provide all cell line engineering, process development and cGMP biomanufacturing activities associated with the two lead products, which utilize a novel antibody-based targeting system, capable of enhanced intracellular delivery of functional proteins, oligonucleotides, and small molecules. The project will utilize Catalent’s proprietary GPEx® technology, which creates high-expression, extremely stable mammalian cell lines with speed and efficiency, typically capable of getting drug development projects to clinic in one-third the time of traditional approaches. The advantages of applying GPEx technology span from early feasibility studies, to clinical manufacturing, to commercial-scale production.

Should initial studies prove successful, the therapies will be produced at Catalent’s state-of-the-art Madison, WI, site, a purpose built facility for mammalian cell culture development and cGMP manufacturing utilizing up to 1000-L single use bioreactors (SUBs).

“We selected Catalent as our development and manufacturing partner for this exciting project as the company has both the expertise and proprietary technologies required to bring these candidates to the clinic in the shortest possible timeframe,” said Deborah Ramsdell, CEO of Valerion. “Catalent’s cGMP facility capabilities are a great match for Valerion’s current supply requirements, and we will look to partner with them on future development and manufacturing needs as well.”

Barry Littlejohns, President of Advanced Delivery Technologies at Catalent, added “We are pleased to be working with Valerion to address the need for biologic based therapeutics for orphan genetic diseases. Valerion’s proprietary tissue targeting delivery platform has demonstrated huge potential in terms of bringing new treatments to market.”

Constructed in response to customer demand, and completed in June 2013, Catalent’s $26-million Madison biomanufacturing facility quadrupled the company’s biologics manufacturing capacity. Designed for flexible cGMP production from 10 L up to 1,000 L, and non-GMP production up to 250 L, the site features single-use technologies and unidirectional flow to maximize efficiency and safety.

Valerion Therapeutics is an emerging science-driven company focused on the development of biotherapeutics for orphan neuromuscular genetic diseases. Valerion generates unique product candidates utilizing a proprietary delivery technology platform that takes advantage of tissue targeting via a novel humanized antibody (VAL-1205) with cell-penetrating properties utilizing a tissue-localized membrane transporter family (ENTs). Because the transporter is naturally enriched in critical tissues (eg, skeletal muscle and CNS), Valerion is able to construct targeted fusion and chemical conjugation products (proteins, drugs, oligos) with functional intracellular activity; thereby, providing a novel way to treat a number of neuromuscular diseases with limited or no current therapeutic options.

Catalent Pharma Solutions is the leading global provider of advanced delivery technologies and development solutions for drugs, biologics, and consumer health products. With over 80 years serving the industry, Catalent has proven expertise in bringing more customer products to market faster, enhancing product performance and ensuring reliable clinical and commercial product supply. For more information, visit www.catalent.com.