CStone Pharmaceuticals Announces 2021 Interim Results
SUZHOU,China,Aug. 26,2021 --CStone Pharmaceuticals(CStone,HKEX: 2616),a leading biopharmaceutical company focused on researching,developing and commercializing innovative immuno-oncology therapies and precision medicines,today announced 2021 financial results.
"In the first half of 2021,CStone Pharmaceuticals,as a full-fledged biopharmaceutical company,has accomplished numerous milestones," said Dr. Frank Jiang,Chairman and CEO of CStone. "We successfully launched two first-in-class (FIC) oncology precision products GAVRETO® and AYVAKIT®,achieving remarkable sales in the first half of 2021. We have also secured three industry-leading new product application (NDA) approvals that enabled our launches,and we submitted four NDAs,among which two are for FIC /potential best-in-class (BIC) products,sugemalimab and ivosedinib,which we soon expect to receive approvals. In the meantime,we are pushing forward with multiple registrational trials at full speed. All these efforts demonstrate CStone's strong research development and commercialization capabilities.
Furthermore,we have received acceptance of an investigational new drug (IND) filing for CS2006,(PD-L1×4-1BB×HSA tri-specific) in mainland China and will complete IND-enabling studies of CS5001 (ROR1 ADC) for global submission,establishing a solid foundation for advancing our Pipeline 2.0 strategy. On the business development front,we will continue to deepen our strategic partnership with Pfizer,co-developing loratinib in the Greater China region,which will further enrich our pipeline. At the same time,we are working closely with EQRx to accelerate NDA submission for sugemalimab in multiple countries. Additionally,CStone was recently included in the Hang Seng Composite Index,which making the company's shares eligible for trading via Hong Kong Stock Connect. This development enhances our access to capital markets as we pursue sustainable,long-term growth.
Moving forward,we will maximize commercial potential of our products by expanding their indications and increasing their accessibility and affordability. We will also expedite a full slate of our clinical development programs,strengthening our presence in other high-prevalence cancers alongside our growing lung cancer portfolio,and developing our early-stage assets. In 2022,we are expecting more than five NDAs to be filed,and one to two INDs to be submitted for the assets with FIC/BIC/first-wave potential and global rights,harnessing the full potential of our Pipeline 2.0 portfolio. Through the above strategic measures,we believe that we can realize our mission to provide breakthrough therapies to cancer patients for better and healthier lives."
BusinessHighlights
In the first half of 2021,CStone continued the tremendous momentum of the prior year,extending a trackrecord ofperformanceasa full-fledgedbiopharmaceuticalcompany.Wedelivered six- months of solid execution,maintaining – and where possible,expediting – an ambitious agenda across the business. We further demonstrated our superior clinical development capabilities,with several programsreachingexciting milestones,includingthreeapprovalsthatledtoour first product launches. Our commercial team executed a flawless go-to-market strategy for our approved drugs,which achieved an exceptional sales ramp-up. Additionally,we advanced our pre-clinical efforts with progress on multiple first-in-class ("FIC")/best-in-class ("BIC")/first-wave ("FW") candidates in emerging therapeutic modalities and for which we hold global commercial rights. Our efforts have further distinguished our pipeline,which stands out for the distinctiveness of our molecules,balanceacrossstagesofdevelopment,growingindicationcoverage,andexpanding mix of global and Greater China commercial rights. Altogether,CStone's performance during the Reporting Period underscores our ability to fully harness the fundamental drivers of our business and brings into clearer view the full commercial and clinical value of our evolving portfolio.
Forthesix monthsendedJune30,2021and asofthedateof thisannouncement,significant progresshasbeenmadewithrespecttoourproductpipelineandbusinessoperations:
I. CommercialEffortsLeadtoSuccessfulProductLaunches
The first halfof2021wasthemostcommercially activeperiodinour history.Through wide and deep engagement with stakeholders in the healthcare community,our growing commercial team set the stage for our first commercial launches,those of GAVRETO® (pralsetinib) and AYVAKIT® (avapritinib). They engaged healthcare providers,regulators,hospitals,pharmaciesand payors,amongothergroups inthehealthcarecommunity,to provide education on our products and expand the number of patients who can access them. Asaresult,we broughttwo precision medicinestomarketwithexceptionalspeedand achieved a rapid sales ramp-up.
Additionally,the commercial team continued their efforts to expand the accessibility of assets on the market to bolster sales while also supporting the broader pipeline of late-stage assets whichareontrackforcommercializationandindicationexpansions.
Highlightsanddetailsonourfirst-halfcommercialactivityfollowbelow.
Healthcare community engagement supports successful product launches
Active engagement with healthcare community stakeholders expanded our coverage of the market to include over 400 hospitals across more than 130 cities,and deepened our ties to healthcare providers,pharmacies,patient groups and insurers. Our sales team is well on track to establish comprehensive coverage of the market in China for our drugs. They now cover hospitals that account for approximately 70-80% of relevant market of precision medicines. Additionally,they secured inclusion of our precision medicines in 20 of the major commercial and government insurance programs. Through this effort,we established a robust network to support our first two product launches and prepare the pathway for future launches.
We launched two precision medicines,reaching a broad swath of patients from the very first day. We successfully launched AYVAKIT® (avapritinib) in mainland China and Taiwan,China in May 2021 and June 2021 respectively,achieving net sales of RMB33.6 million in the first half of 2021. In June 2021,we successfully launched GAVRETO® (pralsetinib) in mainland China,achieving net sales of RMB45.8 million in the first half of 2021.
Strategiccollaborationagreementssupportproductdistribution
We established a strategic collaboration agreement with Sinopharm Group Co.,Ltd ("Sinopharm"). This enabled us to broaden hospital and pharmacy distribution coverage across mainland China for both GAVRETO® (pralsetinib) and AYVAKIT® (avapritinib). Through the collaboration with China (Shanghai) Pilot Free Trade Zone Lin Gang Special Area ("Lin Gang"),we were also able to expedite the process for market entry by clearing customs and completing the port inspection processes within four days,which is significantly earlier than expected.
We formed strategic collaboration agreements with three of the largest integrated healthcare service platforms in mainland China – Shanghai Meditrust Health Co.,Ltd.,Beijing Yuanxin Technology Group Co.,and Medbanks – to leverage each party's competitive advantages and utilize innovative healthcare payment programs to improve distribution and patient affordability of GAVRETO® (pralsetinib) and AYVAKIT® (avapritinib). These relationships will help to maximize distribution of these drugs and improve patient affordability.
Commercialeffortsexpandmarketpotentialandlaunchreadinessoflate-stage assets
WearecloselycollaboratingwithourpartnersPfizerand EQRxtoplanthe commercialization of sugemalimab in mainland China,and the global launch (outside Greater China) of sugemalimab. Our work with Pfizer has put us on track to receive new drug application ("NDA") approval for sugemalimab in mainland China this year,specifically for stage IV non-small cell lung cancer ("NSCLC"). This progress brings us materially closer to full-scale commercial launch of this drug. With EQRx,we are setting the stage for broad distribution of sugemalimab in markets that are forecast to generate approximately US$30 billion in PD-(L)1 sales in 2026 for the treatment of NSCLC,gastric and esophageal cancers: the U.S.,the U.K. and the European Union ("EU"),etc.
We took several steps topreparefor the indicationexpansion of GAVRETO® (pralsetinib) and AYVAKIT®(avapritinib),which will provide greater long- term sales growth potential. For GAVRETO®(pralsetinib),we have submitted NDAs in mainland China for RET-mutant medullary thyroid cancer ("MTC") and RET fusion-positive thyroid cancer and have been granted priority review.We also expect to submit an NDA in mainland China for the first-line treatment of RET fusion-positive NSCLC in the second half of 2021. In addition,we expect to submit NDAs in Hong Kong and Taiwan,China in the second half of 2021 for the second-line treatment of RET fusion-positive NSCLC. For AYVAKIT® (avapritinib),we have submitted an NDA in Hong Kong for PDGFRA D842V mutant gastrointestinal stromal tumor ("GIST"). Also,we are exploring possible routes toexpediteregistrationinmainlandChinaon thebackofU.S.FDA approval for the treatment of adult patients with advanced systemic mastocytosis ("SM").
Weadvancedthelaunchreadinessofivosidenib(IDH1inhibitor)andexpectto receive an NDA approval in the fourth quarter of 2021 or first quarter of 2022 for patients with relapsed or refractory acute myeloid leukemia("R/RAML").
II. NumerousClinicalSuccessesSupportaMaturePipeline
Wemadesubstantialprogressduringthefirsthalfof2021toestablishamaturepipelineof late-stageFICassetsacrossvariousoncologytherapeuticareasandindications,expanding our total potential addressable market. We secured three NDA approvals to support our pralsetinib and avapritinib launches. We submitted four NDA filings covering a third asset,ivosidenib,as wellas indication andgeographicexpansionsfor pralsetinib,avapritinib and sugemalimab. We also significantly stepped up the volume of planned readouts and presentationsrelativetoprioryears.
Ofparticularsignificance,weannouncedseveralpositivedevelopmentswithsugemalimab that demonstrate its broad applicability and safety as a treatment for both stage III and IV NSCLC,including in an "all-comers" setting,which can give it a unique and potentially enduringmarketniche.
Detailsfollowbelow.
Sugemalimab(CS1001,PD-L1antibody)
InMay2021,thephaseIIItrialofsugemalimabinpatientswithstageIIINSCLC as monotherapy in the maintenance setting following concurrent or sequential chemoradiotherapy met its primary endpoint. This innovative trial design reflects real-worldclinicalpracticesand demonstrates sugemalimab'sdistinctabilityto cover a much broader patient population among PD-(L)1 treatments. We submitted an NDA for this indication to the NMPA in August 2021.
The final PFS analysis of the phase IIItrialfor stageIVsquamousand non-squamous NSCLCshowedthat sugemalimabcombined with chemotherapy as first-line treatment contributed to prolonged PFS and encouraging overall survival. Our NDA for this indication was accepted by the NMPA in November 2020 and we expect to receive the NDA approval by the end of 2021. In addition,we are working closely with EQRx on regulatory discussions for new drug applications for the two indications of stage III and stage IV NSCLC in multiple countries,including the U.S.
Pralsetinib(CS3009,RETinhibitor)
On March 24,2021,we received an NDA approval from the NMPA for the treatment of patients with RET fusion-positive NSCLC previously treated with platinum-based chemotherapy.
InApril2021,theNMPAacceptedtheNDAwithPriorityReviewDesignationfor the treatment of patients with advanced or metastatic RET-mutant MTC and RET fusion-positivethyroidcancer
Avapritinib(CS3007,KIT/PDGFRAinhibitor)
On March 31,we received an NDA approval from the NMPA for the treatment of adults withunresectableormetastaticGISTharboring a PDGFRA exon 18 mutation,including PDGFRA D842V mutations.
On April 29,we received the NDA approval license from Taiwan Food and Drug Administration ("TFDA")throughan acceleratedapprovalpathway for adults with unresectable or metastatic GIST harboring a PDGFRA D842V mutations.
In May 2021,we received the acceptanceofthe NDAfromHongKong Departmentof Health("HK DoH")foradultswithunresectable ormetastatic GIST harboring a PDGFRA D842V mutations. We expect a decision on the NDA in the second half of 2022.
Ivosidenib(CS3010,IDH1inhibitor)
Theregistrationaltrialofivosidenibinpatientswithrelapsedorrefractoryacute myeloid leukemia ("R/R AML") with an isocitrate dehydrogenase 1 ("IDH1") mutationmetthepre-specifiedendpoints.IvosidenibisthefirstIDH1inhibitorin China that has demonstrated efficacy and sustained remission in patients with R/R AML.
In August 2021,the NMPA accepted the NDA for the treatment of adults with R/R AML with a susceptible IDH1 mutation and granted priority review. We expect to receive the NDA approval around the end of 2021 or the first quarter of 2022.
In August 2021,our partner,Servier,released positive topline data from the global phase III study of ivosidenib in combination with azacitidine in patients with previously untreatedIDH1 mutantacutemyeloidleukemia.Thestudyrecently halted further enrollment due to compelling efficacy data. We expect to file an NDA for this indication with the NMPA in 2022.
III. Strategic Relationships Advance Outlook for Late-Stage Assets and Bolster Development Pipeline
Wecontinuetogrowanddeepenourrelationshipswithkeyglobalstrategicpartners,Pfizer andEQRx.
With Pfizer,we are preparing sugemalimab for full-scale commercial launch for stage IV NSCLC in mainland China. We are partnering with them in discussions with regulators and working together closely to establish connections with and educate other important healthcare community stakeholders.Theseefforts areintendedtosetthestage for broadandrapid market adoption and sales ramp-up of sugemalimab upon commercial launch.
In addition,we broadened our relationship with Pfizer in the first half of the year with the agreement to co-develop Pfizer's late-stage oncology asset lorlatinib insecond line c-ros oncogene 1 ("ROS1")-positive NSCLC in Greater China. This type of collaboration was envisioned in the original partnership that we announced last year. It is a significant developmentbothclinically and in terms of ourrelationship withPfizer.The planfor lorlatinib is toassess if thisagent canprovide benefits tothe relapsed ROS1-positive advanced NSCLC after crizotinib,which if positive would add a new therapeutic approach to our lung cancer line-up. This program also bolsters the foundation of our relationship with a global biopharmaceutical leader and sets us up for future collaboration with them.
WithEQRx,we haveinitiateddiscussionswith stakeholdersinkeyglobal markets–the U.S.,the U.K.,and the EU – around the registration of sugemalimab for NSCLC indications. Relevant discussions are ongoing. We are collaborating with EQRx to explore the feasibility of extending the range of covered indications for this drug,including gastric cancer and esophageal cancer. In addition,we are working with EQRx to expand a phase III study of CS1003 in HCC in the U.S. and major EU markets.
IV. Pipeline2.0EffortsHarnessFullPotentialofNext-GenCandidates
We have begun to realize the benefits of the revamp of our research capabilities in order to advanceourdevelopmentofBICandFICassetswithglobalcommercialrights.Weexpect thisefforttoenhanceourinternalsourcesofinnovation,generateasustainedsupplyofone totwoinvestigationalnewdrug("IND")application(s)peryear,andsupportdevelopmentof agloballydistinctiveanddifferentiatedpipeline.
We are maintaining our near-term Pipeline 2.0 focus on two emerging therapeutic modalities: antibody-drug conjugates ("ADC") and multi-specific biologics. In the first half of 2021,we madesubstantialprogressadvancingtwosuchassetsintotheclinicalstagethisyear:
CS2006 (NM21-1480,PD-L1×4-1BB×HSA tri-specific molecule): The dose escalation is ongoing and includes sites in the U.S. and Taiwan,China. We have completed dose level 4 enrollment in the U.S. and dose level 5 enrollment is ongoing. We submitted an IND application to the NMPA and received the IND acceptance in July 2021.
CS5001 (LCB71,ROR1 ADC): The IND-enabling activities are ongoing and are expected to be completed with an IND/CTA submitted in the U.S./Australia thereafter by the end of 2021.
InadditiontoCS2006andCS5001,wearefurtherdevelopingadditionalFIC/BIC/FWassets for which we hold global commercial rights,including two multi-specific biologics and one ADC.
V. ExpandingCapitalMarketsAccess
Duetothestrongperformanceinoursharesduringthe12monthsasofJune2021,ourstock hasbeenincludedintheHangSengCompositeIndexanditisexpectedtobeincludedin theHongKongStockConnectimminently.Thisdevelopmentissignificantinthatitcan foster greater trading in our shares,more efficient price discovery and additional liquidity for investors.
Financial Highlights
For the six months ended June 30,our revenue reached RMB79.4 million,primarily attributable to sales of the Company's pharmaceutical products (avapritinib and pralsetinib). The revenue of avapritinib and pralsetinib reached RMB33.6 million and RMB45.8 million respectively.
Excluding the share-based payment expenses,the research and development expenses,the administrative,selling and marketing expenses and the loss for the period were respectively RMB444.8 million,RMB214.3 million and RMB632.5 million.
As of June 30,our time deposits and cash and cash equivalents were RMB2,447.2 million.
2021Interim Results Presentation Information
The Company will host a live webcast for 2021 interim result presentation at 10am HKT,August 27th,please find the access information as below.
https://goldmansachs.zoom.us/webinar/register/WN_mn1nvi0iSGWRde3dRGFNPw
Meeting ID: 939 2219 3743
Passcode: 778867
