U.S. FDA Grants Orphan Drug Designation to BCMA CAR-T Cell Therapy Co-Developed by IASO Bio and Innovent

IASO Biotherapeutics ("IASO Bio"), a clinical-stage biopharmaceutical company engaged in discovering, developing, and manufacturing innovative cell therapies and antibody products, and Innovent Biologics, Inc. ("Innovent", HKEX: 01801), today jointly announced that the Office of Orphan Products Development (OOPD) of the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to their fully human anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T-cell therapy (IASO Bio: CT103A, Innovent: IBI326) for the treatment of relapsed and/or refractory multiple myeloma (R/R MM).

This ODD will accelerate drug development and registration action of CT103A in the United States. CT103A will be eligible for certain development incentives, including FDA support for clinical studies, a waiver or reduction of registration application fee, and a seven-year U.S. market exclusivity granted upon product approval. In February 2021, CT103A was granted Breakthrough Therapy Designation by China's National Medical Products Administration (NMPA) for the treatment of R/R MM.

"FDA approval of ODD to CT103A is of great significance to patients with multiple myeloma and represents the FDA's recognition of CT103A and the clinical data provided by IASO Bio", said Dr. Wen Wang, Chief Executive Officer and Chief Medical Officer of IASO Bio. "Currently, our team is advancing the clinical development of CT103A to the four dimensions of strategy including frontline therapy, combination therapies, indication expansion, and ex-China development. We are looking forward to the launch of CT103A both in China and US as soon as possible to offer living-saving treatment option to more patients."

"This ODD of IBI326 is a milestone recognizing our effort to develop a novel anti-BCMA CAR with better efficacy and persistence," said Dr. Hui Zhou, Senior Vice President of Innovent, "It underscores the importance of bringing this therapeutic option to patients with multiple myeloma, and strongly motivates us to expedite the clinical development of IBI326.We hope to launch IBI326 as soon as possible which will bring hope to patients with multiple myeloma."

About the FDA's Orphan Drug Designation

The Orphan Drug Designation (ODD) is granted by the FDA's Office of Orphan Products Development (OOPD) to eligible drugs (including biologic agents) for the prevention, treatment, and diagnosis of rare diseases. The FDA defines rare diseases as those which affect fewer than 200,000 people in the United States. Due to the small number of patients, low market demand and huge R&D costs, pharmaceutical companies were less inclined to invest in the development of these drugs without specific policy support. In 1983 however, the United States Congress passed the Orphan Drug Act (ODA), stipulating that all drug candidates granted the ODD will have access to a series of supporting policies.

About Multiple Myeloma (MM)

Multiple myeloma is a deadly blood cancer that often infiltrates the bone marrow causing anemia, kidney failure, immune problems, and bone fractures. For multiple myeloma patients, common first-line drug treatments include proteasome inhibitors, immunomodulatory drugs, and alkylating agents. While treatment may result in remission, most patients will inevitably enter the relapsed or refractory stage as there's currently no cure. As a result, there is a significant unmet need for patients with relapsed/refractory multiple myeloma. In the United States, MM accounts for nearly 2% of new cancer cases, and more than 2% of all cancer-related deaths.

According to Frost & Sullivan, the number of new MM cases in the United States rose from 30,300 in 2016 to 32,300 in 2020 and is expected to increase to 37,800 by 2025. The total number of patients diagnosed with MM in the United States increased from 132,200 in 2016 to 144,900 in 2020 and is expected to rise to 162,300 by 2025.

In China, the number of new MM cases rose from 18,900 in 2016 to 21,100 in 2020 and is expected to increase to 24,500 by 2025. The total number of patients diagnosed with MM in China increased from 69,800 in 2016 to 113,800 in 2020 and is expected to rise to 182,200 by 2025.

About CT103A/IBI326 (BCMA CAR-T)

CT103A is an innovative therapy co-developed by IASO Bio and Innovent. Previous studies indicate subjects with relapsed/refractory multiple myeloma (R/R MM) who received high-dose BCMA-targeting CAR-T cells may achieve better remission but have worse adverse events. Moreover, once the disease progresses again, the re-infusion of CAR-T cells will not be effective. To solve this dilemma, CT103A has been developed, a lentiviral vector containing a CAR structure with a fully human scFv, CD8a hinger and transmembrane, 4-1BB co-stimulatory and CD3ζ activation domains. Based on strict selection and screening, utilizing a proprietary in-house optimization platform, the construct of the BCMA CAR-T is potent and persistent. In February 2021, CT103A was granted "Breakthrough Therapy Designation" by the NMPA for the treatment of relapsed/refractory multiple myeloma. In February 2022, CT103A was granted "Orphan Drug Designation" by the FDA for the treatment of relapsed/refractory multiple myeloma.

About IASO Biotherapeutics

IASO Bio is a clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and autoimmune diseases. Leveraging its proprietary fully-human antibody discovery platform (IMARS), high-throughput CAR T drug priority platform, and proprietary manufacturing processes, IASO Bio is developing a rich clinical-stage pipeline of multiple autologous and allogeneic CAR T and biologics product candidates. This includes a diversified portfolio of 10 novel pipeline products, including IASO's leading asset, CT103A, an innovative anti-BCMA CAR T cell therapy under pivotal study for relapsed/refractory multiple myeloma (R/R MM). CT103A received Breakthrough Therapeutic Designation by China's National Medical Products Administration (NMPA) in February 2021. In addition, the company's in-house developed fully-human CD19/CD22 dual-targeted chimeric antigen receptor (CAR) T cell therapy has entered phase I/II registrational clinical trial for the treatment of CD19/CD22-positive relapsed/refractory B-cell non-Hodgkin's lymphoma (R/R B-NHL). It was also granted Orphan Drug Designation by the U.S. Food and Drug Administration in October 2021. For more information on IASO Bio, please visit www.iasobio.com and or LinkedIn.

About Innovent Biologics

Inspired by the spirit of "Start with Integrity, Succeed through Action," Innovent's mission is to develop and commercialize high quality biopharmaceutical products that are affordable to ordinary people. Established in 2011, Innovent is committed to developing, manufacturing and commercializing high quality innovative medicines for the treatment of cancer, metabolic, autoimmune and other major diseases. On October 31, 2018, Innovent was listed on the Main Board of the Stock Exchange of Hong Kong, S.A.R., China Limited with the stock code: 01801.HK.

Since its inception, Innovent has developed a fully-integrated multi-functional platform which includes R&D, CMC (Chemistry, Manufacturing, and Controls), clinical development and commercialization capabilities. Leveraging the platform, the company has built a robust pipeline of 29 valuable assets in the fields of cancer, metabolic, autoimmune diseases and other major therapeutic areas, with 6 products, TYVYT® (sintilimab injection), BYVASDA® (bevacizumab biosimilar injection), SULINNO® (adalimumab biosimilar injection), HALPRYZA® (rituximab biosimilar injection),PEMAZYRE® (pemigatinib) and Olverematinib (BCR TKI) officially approved for marketing in China, 5 assets in Phase 3 or pivotal clinical trials, and additional 18 molecules in clinical trials.

Innovent has built an international team with expertise in cutting-edge biological drug development and commercialization. The company has also entered into strategic collaborations with Eli Lilly and Company, Roche, Adimab, Incyte, MD Anderson Cancer Center, Hanmi and other international partners. For more information, please visit: www.innoventbio.com and www.linkedin.com/company/innovent-biologics/.

Note:

TYVYT® (sintilimab injection) is not an approved product in the United States.

BYVASDA® (bevacizumab biosimilar injection), SULINNO®, and HALPRYZA® (rituximab biosimilar injection) are not approved products in the United States.

TYVYT® (sintilimab injection, Innovent)

BYVASDA® (bevacizumab biosimilar injection, Innovent)

HALPRYZA® (rituximab biosimilar injection, Innovent)

SULINNO® (adalimumab biosimilar injection, Innovent)

Pemazyre® (pemigatinib oral inhibitor, Incyte Corporation). Pemazyre® was discovered by Incyte Corporation and licensed to Innovent for development and commercialization in Mainland China, Hong Kong, S.A.R., China, Macau, S.A.R., China and Taiwan, China.

Disclaimer:

1. This indication is still under clinical study, which hasn't been approved in China and the U.S.

2. Innovent does not recommend any off-label usage.

3. For medical and healthcare professionals only.

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These forward-looking statements are based on the existing beliefs, assumptions, expectations, estimates, projections and understandings of the management of Innovent with respect to future events at the time these statements are made. These statements are not a guarantee of future developments and are subject to risks, uncertainties and other factors, some of which are beyond Innovent's control and are difficult to predict. Consequently, actual results may differ materially from information contained in the forward-looking statements as a result of future changes or developments in our business, Innovent's competitive environment and political, economic, legal and social conditions.

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Source: Nanjing Iaso Biotherapeutics Co., Ltd