Suzhou, China; August 18, 2020 -- CStone Pharmaceuticals (“CStone”; HKEX: 2616), a leading biopharmaceutical company focused on developing and commercializing innovative immuno-oncology (IO) therapies and precision medicines, today reported recent business highlights and financial results for the first half of 2020.

Dr. Frank Jiang, Chairman and Chief Executive Officer of CStone Pharmaceuticals, said, “We continued to make significant progress in our strategic transition to commercialization in the first half of 2020. We obtained one marketing approval from the regulator in Taiwan Region. In addition, we also filed two New Drug Applications (NDAs) in Chinese mainland and Taiwan Region respectively, and was granted priority review status in Chinese mainland. Furthermore, we have released highly promising data for our three core assets in their application for several key indications and have obtained several IND approvals.

Since the beginning of 2020, we’ve achieved three critical clinical and regulatory milestones, which propelling the company to near commercial stage:

• Sugemalimab (CS1001, PD-L1 antibody), one of our backbone IO products, has demonstrated superior efficacy as a first-line treatment in patients with stage IV squamous and non-squamous non-small cell lung cancer (NSCLC) in a randomized, double-blind phase III clinical trial, and it is expected to become the world's first anti-PD-L1 monoclonal antibody to be approved for the first-line treatment of squamous and non-squamous NSCLC, in combination with chemotherapy.
• Pralsetinib, another core asset in our precision therapies, has shown durable anti-tumor activity and well-tolerated safety profile when used as second-line treatment for RET fusion-positive NSCLC patients.
• Submitted NDAs for Avapritinib for the indication of PDGFRA exon 18 mutant gastrointestinal stromal tumors (GIST) in the Chinese Mainland and Taiwan. This is expected to become China’s first approved precision therapy for GIST patients harboring PDGFRA exon 18 mutation.

These significant milestones further demonstrate our robust clinical development engine as we continue to accelerate our commercialization strategy.

As of today, we have completed the building of an industry-leading commercialization core team, successfully had our two precision therapies recommended for use according to the guidelines of Chinese Society of Clinical Oncology (CSCO) and launched our first drug in China through a commercial agreement in Bo’ao Hope City, Hainan. In the meantime, our Suzhou manufacturing site also started construction in the first half of the year. It is expected to provide strong manufacturing capabilities to bolster our commercialization initiatives and set a new benchmark for the industry.

The company is working on to accelerate the progress of nearly 30 clinical trials, including 15 registrational clinical studies and 11 clinical trials on combination therapies. In parallel, we continue to engage potential partners to discuss an array of value-creating opportunities, including in-licensing, out-licensing and strategic partnerships.

In terms of upcoming milestones in the second half of 2020, we look forward to one marketing approval and the submission of three NDAs. The two NDAs we plan on filing for NSCLC, together with ongoing pivotal trials for other products in our pipeline indicated for NSCLC, have prepared us with a powerful product portfolio for lung cancer, the largest cancer type presently found in China. The company will continue to strengthen product accessibility by leveraging synergies between our industry-leading commercial team and active external strategic collaborations, in turn better serving patients in need in China and across the globe, and further maximizing our overall commercial value.”

Business Highlights

As of the date of this announcement, significant advancement has been made with respect to our product pipeline and business operations:

Late-stage Assets Progress

• Sugemalimab (CS1001, PD-L1 antibody): We have made significant progress to advance our lead immuno-oncology (“IO”) asset sugemalimab in the clinic, qualifying it as a promising anti-PD-L1 with unique advantages and significant differentiation.

In August 2020, the Phase III trial of sugemalimab met primary endpoint as first-line treatment for Stage IV squamous and non-squamous non-small cell lung cancer (“NSCLC”). We plan to submit a New Drug Application (“NDA”) to the National Medical Products Administration (“NMPA”) of the People’s Republic of China (“China”) in the second half of 2020.

Globally first anti-PD-L1 monoclonal antibody to demonstrate overwhelming efficacy as 1L treatment of Stage IV squamous and non-squamous NSCLC in a randomized, double-blind phase III trial.

Interim analysis showed that sugemalimab combined with chemotherapy had a statistically significant prolongation of progression-free survival (“PFS”), the primary endpoint of the trial, compared with placebo combined with chemotherapy, reducing the risk of disease progression or death by 50%. The median PFS was 7.8 months vs. 4.9 months in sugemalimab combined with chemotherapy and placebo combined with chemotherapy, respectively.

Subgroup analyses showed clinical benefit across histology subtypes and PD-L1 expression levels.

Sugemalimab in combination with chemotherapy was well tolerated, no new safety signals were identified.

We have received an Investigational New Drug (“IND”) approval for the natural killer T-cell lymphoma (“NKTL”) pivotal trial from the United States (“U.S.”) Food and Drug Administration (“FDA”) in August 2020.

• CS1003 (PD-1 antibody)

We have initiated a global Phase III trial of CS1 0 0 3 in combination with LENVIMA® (lenvatinib), a standard-of-care tyrosine kinase inhibitor (“TKI”) in patients with advanced hepatocellular carcinoma (“HCC”) and dosed the first patient in December 2019. In July 2020, CS1003 was granted an Orphan Drug Designation (“ODD”) by U.S. FDA for HCC.

The first patient has been dosed in a Phase Ib trial of CS1003 in combination with regorafenib in Australia in December 2019.

A scientific paper describing the full characterization of CS1003 and its pre-clinical data was published on Acta Phamacologica Sinica in May 2020 (Fu et al, 2020 online).

• Pralsetinib (CS3009, RET inhibitor)

The registrational study of pralsetinib in Chinese RET fusion-positive NSCLC patients achieved the pre-defined results and we plan to submit an NDA to the NMPA in the second half of 2020.

Primary efficacy data showed deep and durable anti-tumor activity of pralsetinib in RET fusion-positive NSCLC treated with platinum-based chemotherapy. Pralsetinib was well-tolerated in the Chinese patient population. Overall, the data showed that efficacy and safety profile in Chinese patients with RET fusion-positive NSCLC were consistent with previously reported data from the global patient population in the ARROW trial.

We have also completed enrollment in China for the cohort of patients with RET mutant medullary thyroid cancer (“MTC”) who have not been previously treated with systemic therapy.

We have initiated an additional registrational cohort for first-line RET fusion-positive NSCLC with the first subject dosed in the first quarter of 2020.

We are enrolling patients in a basket trial in other tumor types.

Our partner, Blueprint Medicines Corporation (NASDAQ: BPMC) (“Blueprint Medicines”), has submitted an NDA to U.S. FDA for advanced or metastatic RET mutant MTC and RET fusion-positive thyroid cancers in the second quarter of 2020.

Blueprint Medicines announced global (excluding Greater China) collaboration with Roche to develop and commercialize pralsetinib for patients with RET-altered cancers in July 2020.

• Avapritinib (CS3007, KIT/PDGFRA inhibitor)

We have submitted an NDA to the NMPA for avapritinib for adults with unresectable or metastatic gastrointestinal stroma tumor (“GIST”) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, which was accepted in April 2020. We were granted priority review by the NMPA in July 2020.

We have submitted an NDA to Taiwan Food and Drug Administration (“TFDA”) for the same indication in March 2020.

Data presented at 2020 ASCO by us has shown that avapritinib was generally well-tolerated and had promising preliminary anti-tumor activity in Chinese GIST patients with PDGFRA D842V mutation.

Ivosidenib (CS3010, IDH1 inhibitor)

We have received an NDA approval from TFDA for ivosidenib for adult patients with relapsed or refractory acute myeloid leukemia (“R/R AML”) containing an isocitrate dehydrogenase-1 mutation (“IDH1m”), and the marketing approval is anticipated in the second half of 2020.

We are conducting two registrational trials in China: one for IDH1m R/R AML, and another for newly diagnosed IDH1m AML patients who are not eligible for intensive therapy.

We expect to submit an NDA for R/R AML in Singapore in the second half of 2020.

Early-stage Assets and Research Progress

• Novel IO combinations: With combination therapy as the core strategy and the unique advantage of leveraging our three IO backbone agents, we made significant progress on multiple combinations with assets from our internal pipeline and external partners:

CS1002 (CTLA-4 antibody) with CS1003 (PD-1 antibody): First patient dosed in dose-escalation in January 2020 and in dose-expansion in June 2020.

Sugemalimab (PD-L1 antibody) with fisogatinib (CS3008, FGFR4 inhibitor) in HCC: Phase Ib part was completed with the recommended Phase II dose (“RP2D”) declared in June 2020. The first patient was dosed in dose-expansion of the Phase II part in July 2020.

Sugemalimab (PD-L1 antibody) with donafenib: Phase I/II trial to be initiated in China.

• Numab collaboration: In March 2020, our partner, Numab Therapeutics AG (“Numab”), filed an IND application for NM21-1480 (PD-L1×4-1BB×HSA tri-specific molecule) to the U.S. FDA and received “may proceed” letter in April 2020. The IND has been approved by U.S. FDA in June 2020. The first patient dosing of NM21-1480 was completed in July 2020. We have received an IND approval for NM21-1480 from TFDA in August 2020.
• Other early assets development

CS3002 (CDK4/6 inhibitor): The first patient was dosed in Australia in January 2020 in a phase I trial of CS3002 as a single agent for the treatment of patients with solid tumors in Australia and China. In February 2020, we received IND approval from NMPA for the treatment of patients with solid tumors.

CS3005 (A2aR antagonist): The first patient was dosed in Australia in January 2020 in a phase I trial of CS3005 as a single agent for the treatment of patients with solid tumors in Australia and China. In May 2020, we received IND approval from NMPA for the treatment of patients with solid tumors.

In June 2020, we released the pre-clinical data of sugemalimab (PD-L1), CS3002 (CDK4/6) and CS3003 (HDAC6), in the E-poster presentation session at the 2020 American Association for Cancer Research (“AACR”) virtual annual meeting II.

Manufacturing Facility

• The construction of the state-of-the-art manufacturing facility in Suzhou has been commenced in the first half of 2020 and is proceeding on schedule.

Commercial Progress

• We are preparing for the launch of avapritinib, pralsetinib and sugemalimab in 2021 in mainland China with a well-established local commercial operation. In Taiwan, we expect to launch ivosidenib by the end of 2020 and avapritinib in 2021. Our commercial team is on track to achieve the Company’s goal of transitioning from R&D to commercial stage in 2020, with focus on strategy development, commercial capability build-up, launch readiness preparation and branding establishment.
• During the six months ended June 30, 2020, several seasoned commercial functional leaders including the general managers of Taiwan and Hong Kong, as well as the head of Sales, Marketing, Medical Affairs and Market Access, all with over 15 years of working experience in pharmaceutical industry at different multinational corporations have onboarded to drive commercialization readiness. A solid foundation of commercial capability was set up, and we are ready to build a powerful and effective commercial team for successful launches of 4 products in Greater China in 2020 and 2021.
• We have actively participated in activities of influential local cancer society, such as Chinese Society of Clinical Oncology (“CSCO”), China Anti-Cancer Association (“CACA”) and Chinese Thoracic Oncology Group (“CTONG”), to increase company and brand awareness. Moreover, ivosidenib (IDH1 inhibitor) and avapritinib (KIT/PDGFRA inhibitor) have been successfully included into CSCO guideline.
• With online digital education programs and well-established publication platforms, we are continuously increasing the share of voice for key opinion leaders (“KOL”) engagement, education of healthcare professionals (“HCP”) on disease, precision medicines and diagnostics, laying a solid foundation for prelaunch readiness. In addition, we are continuously working on the market access and network establishment. For example, we have signed the first commercial agreement for Hainan Bo’ao early access program to address the unmet needs for patients in China, laying a solid foundation for prelaunch readiness.

Business Development

• We keep engaging potential partners for multiple partnership opportunities that will accelerate our value creation, including in-licensing, out-licensing and strategic partnership.
• In March 2020, we amended the agreement with Agios Pharmaceuticals, Inc. (NASDAQ: AGIO) (“Agios”), to extend our territory beyond greater China to Singapore to develop and commercialize ivosidenib.

Financial Highlights

For the six months ended June 30, 2020, the research and development expenses and the administrative and selling expenses amounted to RMB470.4 million and RMB100.3 million respectively. While the loss excluding the effect of the fair value changes of the conversion feature of preferred shares and share-based payment expenses amounted to RMB508.5 million. As of June 30, 2020, our time deposits and cash and cash equivalents were RMB2,123.8 million.

Interim Results Presentation Information

The Company will host a live webcast for 2020 interim result presentation at 10am HKT, August 19, 2020, please find the access information as below.

If you are connecting outside Mainland China

Audience URL:

https://morganstanley.webcasts.com/starthere.jsp?ei=1353977&tp_key=0d201dc5a5

If you are connecting within Mainland China

Audience URL:

https://morganstanley.gmwebcasts.cn/starthere.jsp?ei=1353977&tp_key=0d201dc5a5

About CStone Pharmaceutical

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.

Forward-looking Statement

The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development.