GenScript ProBio and Neoletix signed a letter of intent on clinical and commercial production of recombinant human coagulation factor VIII

On June 1st 2021, GenScript ProBio and Neoletix (Beijing) Biotechnology Co., Ltd. signed a letter of intent on cooperation for clinical and commercial production of human coagulation factor VIII. GenScript ProBio successfully completed delivery of 200L GMP recombinant coagulation factor VIII (rhFVIII) for Neoletix on February 7, 2021. Unlike antibodies, coagulation factor VIII poses a great challenge to large-scale production and pharmacological research due to its poor protein stability, low expression levels, and the demand on development of special detection and analysis methods. GenScript ProBio successfully delivered the project with its internationally-recognized development platform and rich experience in difficult molecule development, such as recombinant proteins and tri-antibodies, and achieved further cooperation intent on clinical production and commercial production.

In May 2019, Neoletix and GenScript ProBio entered into a cooperation agreement in Nanjing, in which GenScript ProBio was entrusted with CMC for Neoletix's leading product – rhFVIII -covering from single cell cloning, cell culture process and analytical method development to GMP production.

Hemophilia A and B are two most common inherited bleeding disorders in which blood does not clot properly due to a lack of required blood-clotting proteins (factor VIII and IX).  Hemophilia A is most commonly treated using FVIII replacement therapy, in which FVIII can be derived either from human blood or produced recombinantly. Due to a tight hFVIII supply and its extremely high cost, most patients with Hemophilia A in China are not able to be treated by prophylaxis. We believe that this unmet medical need would be addressed very successfully with our novel product.

Neoletix's rhFVIII technology has obtained patents in China and the U.S., as well as a certification under PCT (Patent Cooperation Treaty). The expression system and production process developed by Neoletix based on its proprietary rhFVIII technology is characterized by a very high expression level and stable process control. Powered by GenScript ProBio, the production process has been further improved. By our novel process, recombinant FVIII expression level has been increased significantly to levels unachievable by other commercial vendors, while maintaining the highest quality of product. This will make the drug generally more available and considerably more affordable on a commercial scale.

Neoletix plans to start rFVIII clinical trials in Q4 2021 and launch the product after obtaining CFDA approval. GenScript ProBio's world-renowned GMP production capability can provide large-scale production services for biological macromolecules from clinical phase through commercialization. GenScript ProBio's quality system complies with the requirements of laws and regulations in China, the United States and Europe, and follows internationally-leading design concepts - all of these advantages form a true "zero-crossover, unidirectional flow" plant, which will help Neoletix to enter into clinical trials and rapidly achieve commercial production.

"GenScript ProBio and Neoletix have achieved further cooperation intent on clinical and commercial production of rhFVIII, and it is our pleasure to accompany our customers to grow together," said Dr. Brian Min, CEO of GenScript ProBio, "Since China has a large number of Hemophilia patients, the human coagulation factor VIII is short in supply. We are looking forward to, together with Neoletix, help change this dilemma for patients with our robust and complete quality management system and professional quality management team and bring benefits for hemophilia patients as early as we can."

"We appreciate GenScript ProBio's efforts in providing sustained support to Neoletix's recombinant FVIII clinical and commercial production with its international-acclaimed platform and professional team," said Dr. Chester Li, CEO of Neoletix. "High FVIII production coupled with reduced low manufacturing costs, will make our rhFVIII product much more accessible, which in turn will fundamentally reshape the treatment and prevention landscape for patients with hemophilia A and greatly improve their quality of life. We are looking forward to further cooperation with GenScript in bringing benefits for patients as early as possible."

About Neoletix (Beijing) Biotechnology Co., Ltd.

As an international innovation-oriented biopharmaceutical company, Neoletix (Beijing) Biotechnology Co., Ltd. has set up research centers in the United States and Beijing Economic-Technological Development Area, China. Neoletix has specialized in the development of new drug therapies for hemostasis, antithrombosis and rare diseases for many years, especially in such fields as treatment of hereditary or acquired bleeding disorders (such as hemophilia A and hemophilia B), acute traumatic bleeding (such as trauma or surgery), and thrombotic diseases (such as deep vein thrombosis and stroke) with traditional or new coagulation factors. At the same time, Neoletix is also engaged in immune targeted therapy for tumor. Multiple ongoing anti-tumor drugs developed based on targeted nano-antibody platforms can produce targeted effects on a variety of malignant tumors, including unlabeled malignant tumor cells, uncovering a new approach to tumor treatment.

About GenScript ProBio

GenScript ProBio is the biologics CDMO segment of GenScript, proactively providing end-to-end service from drug discovery to commercialization with proactive strategies, professional solutions and efficient processes in antibody drug and gene and cell therapy to accelerate drug development for customers.

GenScript ProBio's innovative solutions for antibody drug development include antibody drug discovery (hybridoma, antibody library, fully human transgenic mice, bispecific antibodies technologies, single b cell screening technology), antibody engineering (antibody humanization, affinity maturation, Fc Engineering) and antibody characterization (analytics and bioassays). In terms of biologics development service, GenScript ProBio has built a regulatory-compliant platform, from stable cell line development, host cell license, process development, analytical development to clinical manufacturing services, providing fed-batch and perfusion process to accelerate IND process and high-quality material for clinical trials. GenScript ProBio has successfully delivered multiple CMC and GMP manufacturing projects.

GenScript ProBio's total gene and cell therapy solution covers CMC of plasmid and virus for IND filing as well as clinical manufacturing and commercial manufacturing. Our quality management systems ensure phase appropriate compliance, data integrity and traceability.

Toward the mission of "Innovation through Collaboration", GenScript ProBio is committed to helping customers shorten the timeline for the development of biological drugs from discovery to commercialization, significantly lowering R&D costs and building a healthier future.