CStone successfully hosted the first U.S. R&D Day in New York

• Among CStone's 15 cancer drug candidates, five candidates are in pivotal late-stage clinical development, with a total of 13 on-going registrational trials.
• The Company has implemented its pipeline strategy for early-stage candidates and combination therapy. With an established clinical development platform as its growth engine, CStone is at an inflection point to transition from a R&D focused organization into a commercial-stage biopharmaceutical company.
• CStone expects to achieve up to 13 milestones in 2020, including:
  - An NDA approval in Taiwan for TIBSOVO® (ivosidenib) for the treatment of relapsed or refractory acute myeloid leukemia (R/R AML), and become CStone's first commercialized product in the Greater China region.
  - A total of five NDA submissions in Mainland China and Taiwan for CS1001, an anti-PD-L1 monoclonal antibody; pralsetinib, a RET inhibitor; and AYVAKIT^TM (avapritinib), a KIT and PDGFRA inhibitor. CStone plans to release top-line data from the seven ongoing clinical trials of these three drug candidates.
• In the next two to three years, the Company expects to launch four innovative products, targeting multiple indications. CStone will leverage its unique and highly efficient R&D platform to bolster its pipeline and sustain the competitiveness of its portfolio.

SUZHOU, China, Jan. 23, 2020 /PRNewswire/ -- CStone Pharmaceuticals ("CStone" or the "Company", HKEX: 2616) announced that it successfully hosted its 2020 U.S. R&D Day in New York on January 21. CStone's management team highlighted major milestones the Company had achieved in research, clinical development, business development and commercialization strategies since its establishment. The management team also provided updates on the progress of the Company's pipeline. Leading academics and key opinion leaders from China and the United States provided insights on the latest trends in the transformation of cancer treatments in China.

Targeting five indications, CStone plans to submit five NDAs in 2020 for CS1001, an anti-PD-L1 monoclonal antibody; pralsetinib, a RET inhibitor; and AYVAKIT^TM (avapritinib), a KIT and PDGFRA inhibitor, in Mainland China and Taiwan. The Company plans to release the top-line data from seven ongoing clinical trials of these three drug candidates. CStone will focus on developing and commercializing innovative immuno-oncology therapies and precision medicines to address the unmet needs in cancer treatment in China and globally, and drive its Pipeline 2.0 strategy to accelerate its transition toward a commercial-stage biopharmaceutical company with a unique and efficient R&D platform. Moreover, the Company plans to submit NDAs for around 10 indications for four products in the next two to three years.

"China's biopharmaceutical industry is undergoing significant transformation, and CStone is at the forefront of this paradigm shift. With a strategy focused on large cancer indications in China, unmet patient needs, and combination therapies, we are currently conducting over 30 clinical trials, including 13 registrational studies, and 14 combination trials," said Dr. Frank Jiang, Chairman and CEO of CStone. "We are very pleased that Shirley Zhao has joined us as General Manager for Greater China and Head of Commercial Operations, at this inflection point in CStone's transition from clinical-stage to commercial-stage. In the next two to three years, Shirley will lead the establishment of CStone's commercialization capabilities and the launch of multiple innovative products in the Greater China region. Leveraging our unique and highly efficient early-stage clinical development platform, we will continue to strengthen our pipeline and drive biopharmaceutical innovations in China."

Event highlights:

CStone's achievements

• CStone has established a clinical development-driven business model and built a world-class clinical team with significant experience in translational medicine and clinical development. In addition, the Scientific Advisory Board comprised of four internationally renowned immuno-oncologists has substantially strengthened the Company's R&D capabilities in immunotherapies and precision medicines, further enhancing and optimizing CStone's R&D strategies and product pipeline.
• CStone has built a risk-balanced and highly competitive immunotherapy-focused pipeline. Among the Company's 15 cancer drug candidates, five are in pivotal late-stage clinical development. AYVAKIT^TM (avapritinib), developed by Blueprint Medicines, and TIBSOVO® (ivosidenib), developed by Agios Pharmaceuticals have received NDA approvals from the U.S. FDA.
  Among these trials, the clinical studies of CS1001, an anti-PD-L1 monoclonal antibody, have achieved promising clinical results with patients treated with CS1001 exceeding one thousand. In 2019, CStone released encouraging data from the trials of CS1001 in esophageal carcinoma and a number of other large cancer indications in China at major medical conferences, including the American Society of Clinical Oncology (ASCO), the Chinese Society of Clinical Oncology (CSCO), and the American Society of Hematology (ASH) annual meetings.
  The two in-licensed products of CStone's pipeline candidates that have received NDA approvals from the U.S. FDA are AYVAKIT^TM (avapritinib), developed by the partner company, Blueprint Medicines, and TIBSOVO® (ivosidenib), developed by Agios Pharmaceuticals. Moreover, Blueprint Medicine has initiated a rolling NDA submission to the U.S. FDA for pralsetinib. All three candidate drugs are being investigated in multiple registrational trials in China.
  In December, 2019, the global Phase III VOYAGER trial of avapritinib in third-line gastrointestinal stromal tumors (GIST), and the global Phase I/II ARROW study of pralsetinib in second- and later-line non-small cell lung cancer (NSCLC) have both completed their targeted enrollments of Chinese patients ahead of schedule.
• CStone developed comprehensive early-stage programs and is scaling up its combination therapy-focused pipeline. The Company has made significant progress with two of its backbone immunotherapy assets, CS1002, an anti-CTLA-4 monoclonal antibody; and CS1003, an anti-PD-1 monoclonal antibody. Both assets generated promising results as monotherapies in Phase I studies, and CS1003 has already entered a global Phase III registrational trial. In the development of combination therapies, CStone recently initiated multiple combination trials and will continue to advance these combination studies in 2020.
• Leveraging a unique and highly efficient R&D platform and an innovation ecosystem, CStone has further strengthened its Pipeline 2.0. The Company will continue to develop first-in-class and best-in-class drugs and therapies to maintain its lead in innovations.
  In 2019, CStone advanced two drug candidates in its pipeline into clinical development, and commenced a dozen preclinical research initiatives.

Major milestones expected in 2020 include:

• One New Drug Application approval
  TIBSOVO^®, for the treatment of R/R AML, is expected to receive an NDA approval in Taiwan and become CStone's first commercialized product in the Greater China region.
• 5 NDA submissions
  CStone plans to submit five new drug applications in Mainland China and Taiwan, for CS1001, pralsetinib, and avapritinib.
• Seven clinical trial data releases
  CStone plans to release top-line data from seven clinical trials of CS1001 (an anti-PD-L1 monoclonal antibody), pralsetinib, and avapritinib, and six of these trials are registrational studies.

Development strategies for 2020

• To consolidate resources to focus on the delivery of the 13 milestones.
• CStone will build a commercial organization for Greater China equipped with core commercialization capabilities to enable the Company's successful transition to the commercial-stage.
• CStone will continue to seek strategic collaborations with key players, to drive the future growth of the company.
• Leveraging its internal R&D, CStone will transition into its Pipeline 2.0 and drive the sustained growth of the Company. In the next three to five years, CStone will add first-in-class and best-in-class multi-specific monoclonal antibodies and molecular backbone assets to its pipeline. The Company plans to further explore tumor microenvironment modulators, cancer vaccines, new pathway inhibitors, and innovative combination approaches that could potentially bolster the effect of anti-PD-(L)1 antibodies.

New product launches

• In the next two to three years, CStone plans to launch four of its drug candidates, including avapritinib, pralsetinib, ivosidenib and the anti-PD-L1 monoclonal antibody CS1001, in the Greater China region, targeting a total of 10 cancer indications.

Key messages from KOL speakers

• Dr. Paul Bunn, MD, former President of American Society of Clinical Oncology (ASCO) and Distinguished Professor at University of Colorado Cancer Center, commented that latest clinical data of the RET inhibitor pralsetinib were very encouraging. The trial has already completed the patient target enrollment, which included naïve patients and patients who have received platinum-based chemotherapy. He expects this precision medicine will benefit cancer patients in the future.
• Dr. Eytan Stein, MD, Professor at the Memorial Sloan Kettering Cancer Center, commented that compared to current standard chemotherapies, ivosidenib could bring significant clinical benefits to AML patients and its high-selectivity could broaden clinical benefits to cancer patients with an IDH1-mutation.
• Dr. Yilong Wu, MD, President of the Chinese Thoracic Oncology Group (C-TONG), past president of Chinese Society of Clinical Oncology (CSCO), tenured professor of Guangdong Provincial People's Hospital and Guangdong Lung Cancer Research Institute, emphasized that CStone has initiated three registrational lung cancer trials and the Company will play an important role in the lung cancer field in China.
  - CS1001 is currently being investigated in the Phase III GEMSTONE-301 study in patients with locally advanced or unresectable NSCLC, and it could potentially benefit a wide range of patients. Considering the clinical practices in China, the trial also included patients who had received either concomitant or sequential radiochemotherapy. In addition, CS1001 is also being investigated in a Phase III trial in first-line Stage IV NSCLC.
  - The study of pralsetinib in RET-fusion NSCLC has demonstrated promising clinical utility. Due to the persistent high incident rate of lung cancers and the lack of any effective treatment for RET-mutant cancers, pralsetinib has the potential of effectively addressing the current treatment gap in RET-fusion NSCLC in China.
• Dr. Jian Li, MD, Professor at Beijing Cancer Hospital and Chairman-elect of the Chinese Society of Clinical Oncology's (CSCO) Committee on GIST, acknowledged avapritinib's therapeutic promise in the treatment of GIST in China. Avapritinib is a first-in-class  precision medicine approved by the U.S. FDA for the treatment of GIST, and there is currently no effective treatment for PDGFRA D842V mutant GIST in China. Available clinical data of avapritinib in PDGFRA exon 18 mutant GIST (including PDGFRA D842V mutations) have demonstrated compelling antitumor activity and a more tolerable safety profile. The drug could significantly benefit GIST patients who are in need of effective treatment options.
• Dr. Weiping Zou, MD, PhD, Professor at University of Michigan, Director of the Center of Excellence for Cancer Immunology and Immunotherapy, and Chair of the American Association of Cancer Research's (AACR) Cancer Immunology Working Group (CIMM), commented that CStone has made significant progress in the past three years, and he will likely to further collaborate with the Company in selecting Class I drug targets for future development and he looks forward to facilitating CStone's transition to Pipeline 2.0.

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About CStone

CStone Pharmaceuticals (HKEX:2616) is a biopharmaceutical company focused on developing and commercializing innovative immuno-oncology and precision medicines to address the unmet medical needs of cancer patients in China and worldwide. Established in 2015, CStone has assembled a world-class management team with extensive experience in innovative drug development, clinical research, and commercialization. The company has built an oncology-focused pipeline of 15 drug candidates with a strategic emphasis on immuno-oncology combination therapies. Currently, 5 late-stage candidates are at pivotal trials. With an experienced team, a rich pipeline, a robust clinical development-driven business model and substantial funding, CStone's vision is to become globally recognized as a leading Chinese biopharmaceutical company by bringing innovative oncology therapies to cancer patients worldwide.

For more information about CStone Pharmaceuticals, please visit: www.cstonepharma.com

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SOURCE CStone Pharmaceuticals