Innovent Biologics and IASO Bio Receive FDA Orphan Drug Designation for BCMA-Targeted CAR-T Candidate IBI326/CT103A for Relapsed/Refractory Multiple Myeloma

San Francisco, USA、SUZHOU AND NANJING, China, Feb. 14, 2022 /PRNewswire/ -- Innovent Bio (HKEX: 01801), a biopharmaceutical company dedicated to the research, development, production, and commercialization of innovative drugs for the treatment of major diseases such as cancer, metabolic disorders, and autoimmune diseases, announced today with IASO Bio, a clinical-stage innovative biopharmaceutical company focused on the development and industrialization of cell therapies and antibody drugs, that the Office of Orphan Products Development (OOPD) of the U.S. Food and Drug Administration (FDA) has officially issued a written response granting orphan drug designation to their jointly developed fully human autologous B-cell Maturation Antigen (BMCA)-targeted Chimeric Antigen Receptor Autologous T-cell (CAR-T) injection (Innovent Bio's R&D code: IB1326; IASO Bio's R&D code: CT103A).Orphan Drug Designation(Orphan Drug Designation, ODD), for the treatment of relapsed/refractory multiple myeloma (R/R MM).

FDA Orphan Drug Designation can help accelerate the clinical development and registration progress of drugs in the United States. The IBI326 candidate product will be eligible for several preferential policies, including FDA support for clinical research guidance, special fee reductions, and seven years of market exclusivity in the U.S. after approval. In February 2021, IBI326 for R/R MM was included as a "Breakthrough Therapy" by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) in China.

Dr. Zhou Hui, Senior Vice President of Innovent Bio"The FDA's orphan drug designation for IBI326 marks a significant milestone in our commitment to developing a BCMA-targeted CAR-T therapy with superior efficacy and persistence. It highlights the importance of bringing this treatment option to patients with multiple myeloma and motivates us to further accelerate the clinical development and registration of IBI326. We hope this candidate product will reach the market soon, bringing hope to patients suffering from multiple myeloma."

Dr. Wang Wen, CEO and Chief Medical Officer of IASO Bio"IASO Bio stated: 'The FDA's granting of CT103A’s orphan drug designation is of great significance to patients with multiple myeloma, representing the FDA's recognition of the CT103A candidate product and the clinical data previously provided by IASO Bio. Currently, the IASO team is advancing the clinical development of CT103A in four dimensions: frontline treatment, combination therapy, autoimmune disease indications, and overseas expansion. We look forward to the early market entry of this candidate product to save more patients’ lives!'"

About FDA Orphan Drug Designation

Orphan Drug Designation (ODD) is a qualification granted by the FDA's Office of Orphan Products Development (OOPD) to drugs (including biologics) that meet the criteria for the prevention, treatment, and diagnosis of rare diseases. Orphan drugs, also known as rare disease drugs, refer to medicines used for the prevention, treatment, and diagnosis of rare diseases. The FDA has clear definitions for rare diseases, specifically those affecting fewer than 200,000 people in the United States. Due to the small patient populations, limited market demand, and high research and development costs, few pharmaceutical companies focus on the development of treatments for these conditions, hence the term "orphan drugs." In 1983, the United States enacted the Orphan Drug Act (ODA), which stipulates that any drug candidate granted orphan drug status may be eligible for a series of supportive policies.

About Multiple Myeloma

Multiple Myeloma (MM) is one of the most common blood cancers and is a malignant disease characterized by the abnormal proliferation of clonal plasma cells. For newly diagnosed multiple myeloma patients, commonly used first-line treatment drugs include proteasome inhibitors, immunomodulatory agents, and alkylating agents. For the majority of patients, commonly used first-line treatments can stabilize the condition for 3-5 years, but a small number of patients exhibit primary resistance at initial treatment, with their conditions not effectively controlled. For most initially responsive patients, after a period of disease stabilization, they will inevitably enter the relapsed or refractory stage. Therefore, there remains an unmet need for patients with relapsed/refractory multiple myeloma. In the United States, MM accounts for nearly 2% of all new cancer cases and over 2% of cancer-related deaths.

According to the Frost & Sullivan report:

About IBI326 (BCMA CAR-T)

IBI326 is an innovative candidate product jointly developed by Innovent Bio and IASO Bio. This candidate product uses lentivirus as a gene vector to transfect autologous T cells, and its CAR includes a fully human scFv, CD8a hinge and transmembrane domain, 4-1BB co-stimulatory domain, and CD3ζ activation domain. Based on stringent screening and comprehensive in vivo and in vitro functional evaluation, the IBI326 CAR-T candidate product demonstrates potent and rapid efficacy with remarkable persistence. In February 2021, IBI326 received Breakthrough Therapy Designation from the National Medical Products Administration (NMPA) for the treatment of relapsed or refractory multiple myeloma. In February 2022, IBI326 received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of relapsed or refractory multiple myeloma.

About Innovent Bio

"Start with trust, achieve through action." Developing high-quality biologics that are affordable for the general public is the ideal and goal of Innovent Bio. Founded in 2011, Innovent Bio is committed to the development, production, and sales of innovative drugs for the treatment of major diseases such as cancer, autoimmune disorders, and metabolic conditions. On October 31, 2018, INNOVENT BIOLOGICS(SUZHOU)CO LTD was listed on the Main Board of the Hong Kong Stock Exchange, stock code: 01801.

Since its establishment, the company has stood out among numerous biopharmaceutical companies with its innovative achievements and internationalized operating model. It has built a product chain comprising 29 novel drug candidates, covering multiple disease areas such as oncology, metabolic diseases, and autoimmune disorders. Among these, six candidates have been selected for the national "Major New Drug Development" program. The company already has six products (Sintilimab Injection, trade name: Tyvyt).^®, English trademark: TYVYT^®; Bevacizumab biosimilar, brand name: BYVASDA^®, English trademark: BYVASDA^®; Adalimumab biosimilar, brand name: SULINXIN^®, English trademark: SULINNO^®; Rituximab biosimilar, trade name: Dabohua^®, English trademark: HALPRYZA^®; pemigatinib oral inhibitor, brand name: Pemazyre^®, English trademark: PEMAZYRE^®; Olverembatinib Tablets, Trade Name: Nulrex^®) have been approved for marketing, five varieties have entered Phase III or pivotal clinical trials, and another 18 products have entered clinical trials.

Innovent Bio has assembled a team of top-tier biopharmaceutical development and industrialization professionals with international advanced standards, including numerous overseas returnees. It has also established strategic partnerships with international collaborators such as Eli Lilly and Company (USA), Adimab, Incyte, MD Anderson Cancer Center, and Hanmi Pharmaceutical (South Korea). Innovent Bio hopes to work together with everyone to enhance the development level of China's biopharmaceutical industry, fulfilling the public's need for accessible medication and their aspirations for health and well-being. For more information, please visit the company website:www.innoventbio.comOr company LinkedIn accountwww.linkedin.com/company/innovent-biologics/。

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1. This indication is for the investigational use of the drug and has not been approved in China or the United States;

2. Innovent Bio does not recommend the use of any unapproved drugs/indications.

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About IASO Bio

IASO Bio is an innovative biopharmaceutical company focused on the development and industrialization of cell therapies and antibody drugs. The company builds its innovation foundation on the development of hematological tumor cell-based drugs and antibody drugs, expanding into solid tumors and autoimmune diseases. It possesses a complete, full-process platform capability from early discovery, regulatory submission, clinical development, to commercial production, including five major technology platforms: a fully human antibody discovery platform, a high-throughput CAR-T drug optimization platform, a universal CAR technology platform, a manufacturing technology platform, and a clinical translational research platform. Currently, the company has 10 pipeline products at various stages of development. The most advanced candidate product, CT103A (a fully human BCMA chimeric antigen receptor autologous T-cell injection), is in the late stage of clinical development and was designated as a "breakthrough therapy" by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) in February 2021. Another self-developed innovative candidate product, CT120 (a fully human CD19/CD22 dual-target CAR-T cell injection), has entered the clinical research stage for indications including relapsed/refractory B-cell non-Hodgkin lymphoma (B-NHL) and relapsed/refractory acute B lymphoblastic leukemia (B-ALL), and has received FDA Orphan Drug Designation (ODD).

With a highly efficient management team, a rich product pipeline, and unique innovation in R&D and business models, IASO Bio is committed to becoming an influential innovative pharmaceutical company in the industry. It aims to bring truly clinically impactful, market-competitive innovative drugs to patients, paving new treatment paths and bringing new hope. For more information, please visit the company website:www.iasobio.comOr LinkedIn page: IASO Biotherapeutics (驯鹿医疗).

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