ABIONYX Pharma, a next-generation biopharmaceutical company developing innovative therapies for sepsis and critical care based on a proprietary apoA-I technology platform, has announced a strategic manufacturing partnership with OLON, a global CDMO. The CER-001 manufacturing partnership is intended to support the next stage of development and commercialization of CER-001, while establishing capabilities that could support future commercial supply, subject to regulatory approval.
The initial work will involve the production of two validation batches of CER-001. These batches represent an important manufacturing milestone within ABIONYX’s regulatory strategy for LCAT deficiency and are also intended to help establish the industrial capabilities required for potential market supply. Manufacturing activities will take place across two OLON sites in France.
ABIONYX will be involved at two key stages of the process:
- The upstream cell-culture production of recombinant human apoA-I
- After purification, the complexation of apoA-I with phospholipids to create CER-001, followed by Fill & Finish
The CER-001 manufacturing partnership therefore extends beyond the immediate production of validation batches. ABIONYX and OLON are establishing a manufacturing framework intended to support the product through potential market entry and subsequent commercial scale-up.
Full-length apoA-I adds manufacturing complexity
CER-001 presents a distinctive manufacturing challenge because it uses full-length recombinant human apoA-I rather than approaches based on short apoA-I-derived peptides. The protein is a 243-amino-acid protein that is subsequently assembled with specific phospholipids to form a biomimetic HDL particle. Producing the protein represents only one part of the manufacturing process. CER-001 also requires the reproducible combination of apoA-I and phospholipids into a homogeneous and stable biological particle with the required pharmaceutical characteristics. This manufacturing complexity is closely associated with the design of CER-001.
By retaining full-length apoA-I within an HDL-like particle, CER-001 is designed to reproduce key structural and functional properties of naturally occurring nascent HDL and enable biological activity throughout the circulation. Over the course of development, ABIONYX has established considerable manufacturing experience around CER-001.
Its process, intellectual property and accumulated manufacturing know-how form an important part of the asset’s differentiation and create a meaningful technological barrier to replication. The CER-001 manufacturing partnership with OLON is intended to build on that experience by establishing a robust, reproducible and scalable manufacturing process suitable for potential commercial supply.
Validation batches set the foundation for scale-up
The immediate focus of the collaboration is the successful manufacture of the two validation batches. However, the longer-term objective is to ensure that manufacturing infrastructure and capabilities needed to supply the market are established if CER-001 receives regulatory approval. With OLON, ABIONYX is moving beyond manufacturing CER-001 solely for clinical development and beginning to put in place the infrastructure needed for potential commercialization.
The validation batches provide the immediate manufacturing milestone, while the broader strategic objective is to develop a scalable manufacturing platform that can support CER-001 from a potential first market entry in LCAT deficiency to a substantially broader opportunity in sepsis.
Cyrille TUPIN, Chief Executive Officer of ABIONYX Pharma, said, “CER-001 is a demanding biologic to manufacture. We produce the full-length, 243-amino-acid human apoA-I protein and then assemble it with specific phospholipids into a biomimetic HDL particle. Building a reliable and scalable process around that complexity has taken years of development and know-how. Thanks to OLON, we are now putting that manufacturing capability in place for the next stage of CER-001: first through our regulatory strategy in LCAT deficiency and, in parallel, through Phase 2b development in sepsis.”
One platform supports LCAT deficiency and sepsis programs
Andrea Conforto, M&S VP CDMO Biotech OLON, said, “Our work with ABIONYX brings together several areas of OLON’s biomanufacturing expertise, from the upstream production of a full-length recombinant protein, the complexation of apoA-I with phospholipids and Fill & Finish. The validation batches represent an important first milestone, but the manufacturing strategy has been designed from the outset with the longer-term requirements of CER-001 in mind. The program reflects our ability to manage complex biologics manufacturing processes and to support innovative products as they progress from clinical development towards potential commercial supply.”
The manufacturing investment also forms part of ABIONYX’s wider strategy for CER-001. In LCAT deficiency, an ultra-rare genetic disease, the company is pursuing a regulatory pathway intended to provide CER-001 with a potential first route to market. The two validation batches under the OLON program are an important step in preparing the manufacturing package supporting that strategy.
At the same time, ABIONYX is advancing CER-001 into Phase 2b development in sepsis, where it is evaluating the potential of the same full-length apoA-I biologic in a much larger patient population with significant unmet medical need. While the two programs differ in scale and development pathway, they use the same underlying product and manufacturing platform. The industrial capabilities being established for LCAT deficiency can therefore support the longer-term development of CER-001, including its development in sepsis. For ABIONYX, the strategy is to pursue a potential first market entry for CER-001 through the rare-disease pathway while continuing to develop the same proprietary biologic in sepsis, where the potential patient population is substantially larger.
