ABIONYX Pharma and OLON partner for CER-001 biologic manufacturing

ABIONYX Pharma and OLON have entered into a strategic manufacturing partnership to prepare CER-001 for regulatory approval and commercial supply. CER-001 is a distinctive manufacturing challenge because it is a full-length recombinant human apoA-I, a 243-amino-acid protein designed to form a biomimetic HDL particle. The partnership includes 2 validation batches for the LCAT regulatory pathway and for the commercial production of CER-001 in sepsis. The agreement with OLON sets up an integrated manufacturing framework intended to support CER-001 from regulatory validation through potential commercial supply.

ABIONYX Pharma, FR0012616852 — ABNX — eligible for the PEA PME, said the collaboration with the global CDMO is aimed at supporting the next stage of development and commercialization of CER-001. The collaboration will first focus on production. The partnership covers two validation batches of CER-001, which ABIONYX says is a key step in its regulatory strategy for LCAT deficiency. It is also meant to build the industrial capability needed for future commercial supply, subject to regulatory approval.

Work under the agreement will be split across two OLON sites in France. ABIONYX will be involved in two critical stages: upstream cell-culture production of recombinant human apoA-I and, after purification, the complexation of apoA-I with phospholipids to form CER-001, followed by Fill & Finish. ABIONYX and OLON are setting up a manufacturing framework intended to support CER-001 through potential market entry and later commercial scale-up.

ABIONYX said CER-001 presents a distinctive manufacturing challenge because, unlike short apoA-I-derived peptides, it uses full-length recombinant human apoA-I, a 243-amino-acid protein, which is then assembled with specific phospholipids to form a biomimetic HDL particle. Manufacturing CER-001 requires the reproducible assembly of apoA-I and phospholipids into a homogeneous, stable biological particle with the required pharmaceutical characteristics. That challenge is tied to the design of CER-001, which retains full-length apoA-I in an HDL-like particle.

CER-001 is designed to reproduce key structural and functional properties of naturally occurring nascent HDL and to support biological activity throughout the circulation. ABIONYX says it has built substantial manufacturing experience with CER-001 during its development. The company said its process, together with its intellectual property and accumulated manufacturing know-how, is an important part of the asset’s differentiation and creates a technological barrier to replication. The partnership with OLON is meant to turn that experience into a robust, reproducible and scalable manufacturing process for potential commercial supply.

The immediate aim of the collaboration is to successfully manufacture the two validation batches. The longer-term objective is to ensure that, if CER-001 receives ABIONYX said the manufacturing infrastructure and capabilities needed to supply the market are already in place, following a regulatory approval. Through the partnership, ABIONYX is extending CER-001 production beyond clinical development and starting to build the infrastructure needed for possible commercialization. The two validation batches are described as the immediate milestone.

The company said the next strategic step is a scalable manufacturing platform that could support CER-001 from a potential first market entry in LCAT deficiency to a broader opportunity in sepsis. Chief Executive Officer Cyrille TUPIN said CER-001 is difficult to manufacture, adding that the company makes the full-length, 243-amino-acid human apoA-I protein and assembles it with specific phospholipids into a biomimetic HDL particle.

TUPIN said building a reliable and scalable process around that complexity has taken years of development and know-how, and that OLON is now helping put that manufacturing capability in place for the next stage of CER-001, first through the regulatory strategy in LCAT deficiency and in parallel through Phase 2b development in sepsis. Conforto, M&S VP CDMO Biotech at OLON, said the collaboration with ABIONYX combines several parts of OLON’s biomanufacturing expertise, including upstream production of a full-length recombinant protein, complexation of apoA-I with phospholipids and Fill & Finish.

He added that the validation batches are an important first milestone, while the manufacturing strategy was designed from the outset with CER-001’s longer-term requirements in mind. The program, he said, shows OLON’s ability to handle complex biologics manufacturing and support innovative products as they move from clinical development toward potential commercial supply. The manufacturing platform also supports ABIONYX’s wider strategy for CER-001. In LCAT deficiency, an ultra-rare genetic disease, ABIONYX is pursuing a regulatory pathway aimed at giving CER-001 a potential first route to market.

The two validation batches to be produced under the OLON program are a key step in preparing the manufacturing package for that strategy. ABIONYX is also advancing CER-001 into Phase 2b development in sepsis. The company is assessing the same full-length apoA-I biologic in a much larger patient population with a significant unmet medical need. The two programs differ in scale and development route, but they use the same underlying product and manufacturing platform.

That setup lets the industrial capabilities being built for LCAT deficiency also support the longer-term development of CER-001, including in sepsis. ABIONYX said it plans to use the rare-disease route for a potential first market entry for CER-001 while advancing the same proprietary biologic in sepsis, where the potential patient population is substantially larger. OLON is an Italian company focused on the development and production of active pharmaceutical ingredients, with expertise in chemical synthesis and biological processes. Based in Rodano, near Milan, Italy, OLON operates 14 manufacturing sites and 14 R&D centers worldwide, and has over 2,800 employees.

It said it provides end-to-end support from early development to commercial manufacturing. ABIONYX Pharma says it is a next-generation biopharmaceutical company focused on sepsis and critical care, developing biotherapies for serious diseases that it says currently have no effective treatments. The company says its proprietary apoA-I-based technology platform is used to design biopharmaceuticals and next-generation HDL vectors targeting immune-inflammatory dysregulation linked to sepsis and other severe diseases.

ABIONYX Pharma said it is relying on scientific expertise, a differentiated pipeline and an expanding global clinical network as it seeks to redefine therapeutic standards for sepsis and establish itself as a major player in intensive care. The document includes a disclaimer that some statements may be forward-looking, and says such statements can generally, but not always, be identified by words including believes, expects, should, anticipates, intends to, estimates, is expected to, will continue to, may, is likely to, forecasts or similar expressions, including variations, negative forms or comparable terms.

Forward-looking statements are not guarantees of future performance and involve known and unknown risks, uncertainties and other factors. ABIONYX Pharma said its actual operating results, financial condition and industry development may differ materially from those indicated or implied by the forward-looking statements in this document. The company added that even if its operating results and financial condition, and developments in the industry in which it operates, are consistent with the forward-looking statements, those results or developments are not necessarily indicative of subsequent periods.

ABIONYX Pharma said it does not undertake to publicly update or revise any forward-looking statements made in this document, whether due to new information, future events or otherwise.