May 10Day,CStone Pharmaceuticals, an innovative biopharmaceutical company listed on the Hong Kong Stock Exchange(02616.HK) First-in-classPrecision Medicine DrugsIDH1 InhibitorTIBSOVO®（Ivosidenib Tablets）ObtainEU Commission Approves Two Indications: TIBSOVO®In combination with azacitidine for the treatment of patients carrying isocitrate dehydrogenase-1 (IDH1) R132 MutationNewly diagnosed acute myeloid leukemia (AML) unsuitable for standard induction chemotherapyAML) adult patients;And TIBSOVO®Patients with locally advanced or metastatic disease who have received at least one prior systemic therapy before monotherapy.IDH1 R132 mutationCholangiocarcinomaAdult Patients。
Notably, TIBSOVO®Is the first and only approved in EuropeIDH1Targeted therapy, for which the European Commission has previously granted orphan drug designation, in recognition of Toshuwo.® Compared to other available treatments for cholangiocarcinomaAndAML PatientsBrought bySignificantBenefitAdvantages.Based on excellent clinical performance, Toshuwo®Has also been approved for marketing in the United States and China.
IDH1Mutated patients' needs unmet
Ivosidenib Fills the Market Gap
Public information shows,IDH1 mutationACholangiocarcinoma with ML and IDH1 mutations belongs to the refractory and difficult-to-cure types of cancer.。AML is the most common type of acute leukemia in adults, with high malignancy and rapid progression, in EuropeMore than20,000 new cases，75The two-year survival rate of people over a certain age is lower than10％; Cholangiocarcinoma is often associated with a history of cirrhosis or liver infections in Europe.Approximately every year10,000 new cases，The five-year survival rate of patientsExtremely Low。IDH1 Mutation in AMLAndThe main driver of disease progression in cholangiocarcinoma, thisTwo typesThe disease is usually diagnosed at a late stage, thus representing an unmet medical need.AndTIBSOVO®New targetedTherapy，Due toIts mechanism of action is different from traditional chemotherapy,Gradually FormingForCan prolong the life of patientsSurvival period andImprove itsTreatment options for quality of life.
This EU approval of TIBSOVO®The AML indication is based onAGILE Study, this study is a globalPhase IIIMulticenter, double-blind, randomized, placebo-controlled clinical trial, which has been published in The New England Journal of Medicine. The results showed that, compared with the placebo plus azacitidine group, inIDH1MutatedIn AML patients, TIBSOVO®Combined with azacitidine can significantly prolong patients' event-free survival (EFS) and overall survival (OS), and withStatistically significant.TIBSOVO®The median of the combination of Azacitidine and placebo with Azacitidine groupsOS were24.0 months and 7.9 months. In addition, the study also met all key secondary endpoints, including complete remission (CR rate, OS, complete remission with partial hematological recovery (CRh) rate, and objective response rate (ORR).TIBSOVO®Combination with Azacitidine Shows Good Safety。The study demonstrated that TIBSOVO®Combined with Azacitidine for the Treatment of Newly DiagnosedEfficacy in IDH1-Mutated AML Patientsand safePlan.
EU Approves TIBSOVO®CholangiocarcinomaIndicationsThenYesBased onClarIDHyThe study, which is the first and only one targetingPreviously treated and carryingRandomized Phase III Trial in IDH1-Mutant CholangiocarcinomaResearch. Primary endpointThe analysis shows,Compared withInPlaceboGroup, Toshuwo®Significantly prolong the progression-free survival (PFS) assessed by the Independent Review Committee (PFS）, and hasStatisticsMeaning。TIBSOVO®GroupAnd placeboGroupMedianPFS were 2.7 months and 1.4 months, respectively. InTIBSOVO®Treatment GroupIn China,6 Months and 12Months ofPFS rates were32%And22%，Whereas the placebo groupThePatientAverageIn6 monthsAppear internallyDisease progression or death.
Promote TuoShuWo®Multiple Indications Approved
Committed to enhancing accessibility and affordability
It is estimated that currently in China, there areThere are approximately 45,000 patients with IDH1-mutated tumors, including AML, glioma, cholangiocarcinoma, relapsed/refractory myelodysplastic syndrome, and chondrosarcoma, etc.。TIBSOVO®Approved by the China National Medical Products Administration for the treatment ofAdult patients with relapsed or refractory AML harboring IDH1 mutations, In addition,CStone Pharmaceuticals is in discussions with the Center for Drug Evaluation of the National Medical Products Administration regarding Toshuwo.®For first-line treatment of newly diagnosedDiscussion on the Registration Pathway for IDH1-Mutated AML Patients and Cholangiocarcinoma Patients. At the same time, several other clinical studies are ongoing worldwide, including those for the treatment of patients carryingAdult patients with IDH1-mutant relapsed or refractory myelodysplastic syndromes (MDS), advanced glioma, etc.
Currently, TIBSOVO®Approved in the United StatesIn combination with azacitidine or as monotherapy forNewly diagnosed IDH1-mutant AML patients aged 75 years or older, or those with comorbidities who are ineligible for intensive induction chemotherapy, as well as patients with relapsed or refractory IDH1-mutant AML treated with single-agent therapy.And,TIBSOVO®Single drug hasIn the United StatesObtainBatch forTreated, locally advanced or metastaticThePatients with IDH1-mutant cholangiocarcinoma. In addition, CStone Pharmaceuticals plansWillSubmission of TIBSOVO in Hong Kong, Taiwan, and Singapore®New drug marketing application.
CStone Pharmaceuticals has been striving to enhance TIBSOVO®Accessibility and affordability of precision treatment drugs such as these. Currently,TIBSOVO®InIn ChinaMain Target Hospitals andAvailable in 100% of DTP (Direct-to-Patient Pharmacies); in the top 200 target hospitals, the genetic testing rate for potential IDH1 mutation patients has reached approximately 75%. Through collaboration with the National Pathology Quality Control Center, both the standardization of the testing process and the accuracy of testing have been improved; TIBSOVO.®Included in接近100 major commercial and government insurance programs, covering tens of millions of people.In addition, CStone Pharmaceuticals through"Vorun Hope - Patient Assistance Program"Assistance Medicines , reduce the burden of the disease on patients' families and society, and improve patients' quality of life.
