ABL inhibitors(Abl family tyrosine kinases inhibitors), Bcr-Abl inhibitors(Bcr-Abl tyrosine kinase inhibitors), PDGFR antagonists(Platelet-derived growth factor receptor antagonists)

Therapeutic Areas

NeoplasmsImmune System DiseasesDigestive System Disorders+ [5]

Active Indication

Accelerated phase Philadelphia chromosome positive chronic myeloid leukemiaPhiladelphia chromosome positive chronic myeloid leukemia in blast crisisPhiladelphia chromosome-positive chronic myeloid leukemia in chronic phase+ [31]

Inactive Indication

Abdominal NeoplasmsAcral Lentiginous Malignant MelanomaAcute Eosinophilic Leukemia+ [169]

Originator Organization

Novartis Pharma AG

Active Organization

National Cancer InstituteAccord Healthcare SLU

Novartis Pharma KK

+ [9]

Inactive Organization

Novartis AGKoanaa Healthcare GmbH

Aerovate Therapeutics, Inc.

+ [15]

Drug Highest PhaseApproved

First Approval Date

US (10 May 2001),

Philadelphia chromosome positive chronic myelogenous leukemia

RegulationAccelerated Approval (US), Orphan Drug (US), Priority Review (CN), Orphan Drug (JP)

Structure

Molecular FormulaC30H35N7O4S

InChIKeyYLMAHDNUQAMNNX-UHFFFAOYSA-N

CAS Registry220127-57-1

637

Clinical Trials associated with Imatinib mesylate

NCT06692491 / Not yet recruitingPhase 2IIT

A Phase II Clinical Study on Precision Treatment of Rare Tumours in China Guided by Patient-Derived Tumor Organoids (PDO) and Next-Generation Sequencing (NGS)

The objective of this Phase II, open-label, multicenter, non-randomised controlled clinical trial is to guide precision treatment for patients with rare tumours based on Patient-Derived Organoids/Next-Generation Sequencing drug screening.

Start Date01 Jan 2025

Sponsor / Collaborator

Peking University Shenzhen Hospital

NCT06655246 / Not yet recruitingPhase 1

A Phase 1a/1b Study of the Safety, Pharmacokinetics, and Antitumor Activity of the Oral Menin Inhibitor Ziftomenib in Combination with Imatinib in Patients with Advanced Gastrointestinal Stromal Tumors (GIST) After Imatinib Failure

In this clinical trial, the safety, tolerability, and preliminary antitumor activity of ziftomenib in combination with imatinib will be evaluated in adults with gastrointestinal stromal tumors (GIST) who have been treated previously with imatinib.

Start Date01 Jan 2025

Sponsor / Collaborator

Kura Oncology, Inc.

CTRI/2024/11/077421 / Not yet recruitingNot ApplicableIIT

Treatment-free remission in chronic myeloid leukemia - NIL

Start Date10 Dec 2024

Sponsor / Collaborator-

100 Clinical Results associated with Imatinib mesylate

100 Translational Medicine associated with Imatinib mesylate

100 Patents (Medical) associated with Imatinib mesylate

16,701

Literatures (Medical) associated with Imatinib mesylate

01 Dec 2025·CANCER CHEMOTHERAPY AND PHARMACOLOGY

Influence of CYP2C8*3 and ABCG2 C421A genetic polymorphisms on trough concentration and molecular response of imatinib in Egyptian patients with chronic myeloid leukemia

Article

Author: Hussein, Abeer M R ; Abdel-Raheem, Mahmoud H ; Mangoura, Safwat A ; Eltyb, Hanan A ; Molla, Mohammed S

Abstract:

Purpose:

The treatment landscape for chronic myeloid leukemia (CML) has been revolutionized by the introduction of imatinib, a tyrosine kinase inhibitor, which has transformed the disease from a fatal condition into a manageable chronic illness for a substantial number of patients. Despite this, some individuals do not respond adequately to the treatment, and others may experience disease progression even with continued therapy. This study examined how CYP2C8*3 (G416A; rs11572080) and ABCG2 C421A (rs2231142) single nucleotide polymorphisms (SNPs) affect the plasma trough concentration and therapeutic response of imatinib in Egyptian CML patients.

Methods:

The study included fifty patients with chronic-phase CML, who were categorized into two groups: responders (n = 26) and non-responders (n = 24), according to their BCR-ABL1 transcription levels after 12 months of imatinib treatment. Genotyping of the CYP2C8*3 and ABCG2 C421A polymorphisms was performed using polymerase chain reaction-restriction fragment length polymorphism (PCR-RFLP), while plasma trough concentrations were determined through high-performance liquid chromatography with ultraviolet-diode array detection (HPLC-UV/DAD).

Results:

Patients with the CA genotype of ABCG2 C421A showed significantly higher mean plasma trough concentrations of imatinib (CA: 1731 ± 424.7 ng/mL; CC: 1294 ± 381.3 ng/mL; p = 0.0132) and demonstrated a better molecular response compared to those with the CC genotype (p = 0.0395).

Conclusion:

The ABCG2 C421A polymorphism significantly influenced imatinib plasma trough concentrations and molecular responses in Egyptian chronic-phase CML patients. Genotyping of this polymorphism in these patients could assist in optimizing imatinib therapy, predicting more favorable treatment outcomes, and enabling the development of more personalized treatment plans.

01 Apr 2025·RESPIRATORY PHYSIOLOGY & NEUROBIOLOGY

Effect of imatinib on lipopolysaccharide‑induced acute lung injury and endothelial dysfunction through the P38 MAPK and NF-κB signaling pathways in vivo and in vitro

Article

Author: Wang, Keruo ; Zhang, Tianyi ; Liu, Yaru ; Zong, Xiaolong ; Liang, Xue ; Yang, Hong ; Li, Duanyang ; Li, Zhenyu

BACKGROUND:

The primary purpose of this study was to demonstrate the preventive effects of imatinib (IMA) on lipopolysaccharide (LPS)-induced inflammation in a mouse model of acute lung injury (ALI) and human umbilical vascular endothelial cells.

METHODS:

LPS stimulation for 24 h induced ALI and cell inflammation. The pathological results of the lungs were evaluated using the wet/dry weight ratio, pulmonary vascular permeability measurements, and myeloperoxidase immunohistochemistry. The expression of pro-inflammatory mediators was analyzed using RT-PCR and enzyme-linked immunosorbent assay. Protein levels were analyzed using western blotting. The structure of cell junctions was detected using immunofluorescence.

RESULTS:

IMA improved LPS-induced pulmonary pathological damage and reduced the lung wet/dry weight ratio and myeloperoxidase expression in the lung tissue. IMA decreased bronchoalveolar lavage fluid inflammatory cell count and the release of tumor necrosis factor-α (TNF-α), interleukin (IL)-6, and monocyte chemotactic protein 1 (MCP-1) in the blood. Pretreatment of human umbilical vascular endothelial cells with IMA significantly attenuated LPS-induced actin stress fiber formation and vascular endothelial-cadherin disruption. In addition, IMA downregulated the mRNA abundances of vascular cell adhesion molecule 1, intercellular adhesion molecule 1, IL-1β, IL-6, and tumor necrosis factor-α(TNF-α) expression. The phosphorylation of p65, nuclear factor-kappa B inhibitor alpha (IκBα), p38, extracellular signal-regulated kinase, and Jun N-terminal kinase induced by LPS were attenuated after IMA treatment in vivo and in vitro.

CONCLUSIONS:

IMA modulates the nuclear factor-kappa B and mitogen-activated protein kinase signaling pathways and the production of pro-inflammatory cytokines to prevent cellular damage due to LPS infection. These results indicate that IMA may be a potential modulator of LPS-induced ALI.

01 Feb 2025·EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

Circumventing Imatinib resistance in CML: Novel Telmisartan-based cell death modulators with improved activity and stability

Article

Author: Gust, Ronald ; Salcher, Stefan ; Gebhart, Maximilian ; Alilou, Mostafa

Drug resistance presents a significant challenge in cancer therapy, which has led to intensive research in resistance mechanisms and new therapeutic strategies. In chronic myeloid leukemia (CML), the introduction of Imatinib, the first tyrosine kinase inhibitor (TKI), drastically changed the outcome for patients. However, complete remission still cannot be achieved in a large number of patients in the long term. Therefore, there is a great interest in the design of new drugs to target TKI-resistant cancer cells. A promising approach to enhance the efficacy of Imatinib is the simultaneous application of cell death modulators derived from the Angiotensin II type 1 receptor blocker Telmisartan. The methyl ester (3a) of 4'-((2-propyl-1H-benzo[d]imidazol-1-yl)methyl)-[1,1'-biphenyl]-2-carboxylic acid (LEAD-acid (4)), which is the structural core of Telmisartan, has already been shown to abolish the resistance of Imatinib in TKI-insensitive CML cells at a concentration of 5 μM. As the ester was expected to be unstable in a biological environment, this study attempted to increase the stability through structural modifications. The methyl group was exchanged for longer (3b (ethyl), 3c (propyl), 3d (butyl) and branched (3e (isopropyl), 3f (tert-butyl)) alkyl chains as well as a phenyl (3g) and 4-phenoxyphenyl (3h) group. Furthermore, the esters were bioisosterically replaced with a respective substituted carboxamide (5a-h). The LEAD-amides (5a-h) showed high stability against esterases, while amidases cleaved only the carboxamides with short alkyl chains to a small extent. Esterases hydrolyzed the LEAD-alkylesters (3a-d) dependent on the chain length with τ_½ = 55-82 min. Esters with branched alkyl chains were stable and introduction of the aromatic rings mentoined above increased the half-life to τ_½ = 280 min and 360 min. In cell culture medium, only 3a-d degraded to 67-78 % after 72 h. However, the uptake studies showed that approximatly 80 % of the esters accumulated in the cell within the first 1-3 h of incubation. Therefore, it can be concluded that the intact LEAD-esters and LEAD-amides caused the biological effects. The compounds were non-cytotoxic and efficiently sensitized KD225 (K562-resistant) CML cells to Imatinib at a half-maximal sensitizing concentration (SC₅₀) of 1.5-2.9 μM (ester derivatives) and 1.3-11.2 μM (amide derivatives).

365

News (Medical) associated with Imatinib mesylate

17 Dec 2024

·endpts.com

How a duty to spend wisely on worker benefits could loosen PBMs' grip on drug prices

Ann Lewandowski knows all about pharmacy benefit managers, or PBMs, the companies that shape the US drug market. Her job as a policy advocate at Johnson & Johnson was to tell patient and physician groups about the PBMs’ role in high drug prices. Armed with that knowledge, Lewandowski filed a potentially groundbreaking lawsuit in February. Rather than targeting the PBMs, however, she went after a big company that uses one — her own employer, Johnson & Johnson. Lewandowski charges in her lawsuit that by contracting with the PBM Express Scripts, which is part of the insurance giant Cigna, Johnson & Johnson — which fired her in April — failed in its duty to ensure reasonable drug prices for its more than 50,000 U.S. employees. By choosing an Express Scripts plan, she charged, J&J cost employees “millions of dollars in the form of higher payments for prescription drugs, higher premiums, higher deductibles, higher coinsurance, higher copays, and lower wages or limited wage growth.” Lewandowski, 40, from outside Madison, WI, relies on an expensive multiple sclerosis drug. She brought the lawsuit, she said, because she “had trouble aligning the policy positions” she reported on as a J&J employee “with the actions I experienced as a health plan user.” In recent years, the opaque business practices of PBMs have drawn fire. The Federal Trade Commission is conducting a lengthy investigation of the three biggest companies and sued them in September, accusing the firms of driving up insulin prices. Bipartisan bills in Congress would rein them in. And businesses such as Mark Cuban’s Cost Plus Drugs and smaller, “transparent PBMs” have tried to wean pharmaceutical companies and health plans from their reliance on the big PBMs. But Lewandowski’s lawsuit goes to a sensitive spot that had been overlooked until recently: language in the 2021 appropriations bill that revised the 1974 Employee Retirement Income Security Act, known as ERISA. The original law focused on stopping fraudulent retirement plans. Her lawsuit is based on congressional language specifying that the law’s requirement of prudent management covers health as well as retirement benefits. By providing workers with a health plan, employers aren’t “doing you a favor. They are holding your money and investing it in your health,” said Barak Richman, a George Washington University health law professor. In July, a similar lawsuit was filed against Wells Fargo, and more suits are in the works. PBMs demand discounts and rebates from drugmakers, which leads the manufacturers to charge higher list prices, which can drive up the price patients pay at the pharmacy. At the same time, retail pharmacies say PBMs are driving them out of business by paying them less than what the PBMs charge health plans — a practice known as spread pricing. Patients typically have no idea what they’ll pay for a drug, and neither do their employers, because many PBMs’ contracts contain nondisclosure clauses. Dissatisfaction with the status quo and fear of liability are pushing employers to switch from the “Big Three” PBMs to “transparent PBMs,” which don’t shroud their pricing and drug choice decisions. “We brought on nine Fortune 500s this year, 1.2 million patients,” said AJ Loiacono, CEO of New York City-based Capital Rx, a PBM founded in 2017. According to a recent survey , as many as half of US employers are considering switching. Cuban, in an interview with KFF Health News, said he has told hundreds of Fortune 500 executives, in one-on-one meetings and in groups, that they are overpaying on drug benefit plans skewed to fatten the wallets of big PBMs. “You’re getting ripped off,” Cuban said he tells them. “You don’t really understand the elements, and that’s costing you money and costing you wellness. And now you are going to get sued. It’s not a question of if, but a question of when.” The billionaire, who launched Mark Cuban Cost Plus Drugs in 2022 to upend the byzantine $500 billion US drug market, is convinced that the Lewandowski suit and others will end the dominance of the big PBMs, which control 80% of the business. Cost Plus Drugs charges a straight 15% markup with small processing fees for the 2,500 drugs it sells, most of them generics, co-founder Alex Oshmyansky said. Its nearly three million customers — individuals, health plans, and transparent PBMs — appear to be saving money in many cases. The big PBMs say their buying power and exclusive access to information enable them to save money for insurers, employers and patients. Critics say they are skimming up to 25% from the drug market, perhaps $100 billion a year, according to Oshmyansky. The opaque strategies and conflicts of interest, critics say, often result in the poorest, sickest patients paying the most for medications. The three PBMs amount to a “purchasing cartel,” Oshmyansky said in an interview at Cost Plus’ Dallas headquarters, once the office of broadcast.com, the internet radio company that made Cuban his first billion dollars when he sold it to Yahoo in 1999. “They buy all the drugs, they jack up the prices, and then they resell them.” Richman and Amy Monahan of the University of Minnesota argued in a journal article this year that the Department of Labor, which has previously focused its ERISA oversight on retirement benefits, should issue standards for the use of health care dollars under the law. When companies “enter into dumb contracts with insurers or PBMs, arguably they are in violation of ERISA,” Richman said. “Taking the law seriously would really require employers, who are spending half the health care dollars in the country, to spend that money in very different ways.” Some drug market experts, however, doubt the ERISA lawsuits will succeed. Complex PBM money channels “make it hard to build a case,” said Stacie Dusetzina, a professor of health policy at Vanderbilt University School of Medicine. “You might think your company is overpaying, but relative to what?” The ERISA Industry Committee, which lobbies Congress for some of the biggest US companies, is asking Congress to give PBMs the specific duty to represent their clients’ financial interests, said Melissa Bartlett, the group’s senior vice president for health policy. That could require patients to sue the PBMs rather than their employers. A few big employers are already changing their drug plans. In 2019, Connecticut became CVS’ first PBM customer to negotiate a transparent fee structure. Its contract required 100% of drug rebates be passed along to the state and eliminated spread pricing. The state decided to go further when it sought a new contract for its 214,000 employees this year, said Joshua Wojcik, director of health policy and benefits in the state comptroller’s office. Instead of discounts and rebates, it demanded the lowest net cost per employee. Of the three big PBMs, only CVS bid on the contract. It edged out a few “transparent PBMs” — a sign, in Wojcik’s view, that CVS at least doesn’t want to be left out as more customers ditch the current PBM business model. With the change, Wojcik estimates the state will save up to $70 million a year. Changing drug benefit policies at big companies takes time, said Oshmyansky of Cost Plus. Their PBM contracts last three to five years, so “you have to capture them in that one year where they are evaluating other options,” he said. PBMs pay benefit plan consultants and the brokers big companies hire to steer business their way. “We have this weird structure where multiple sclerosis, cancer patients subsidize everybody else’s drugs,” Oshmyansky said. Instead of creating a pool that spreads costs to everyone with insurance, there’s a “disproportionate burden placed on the sickest members.” Cost Plus generates the biggest savings for its customers on about 50 extraordinarily high-priced generic drugs. The poster child is imatinib, a generic cancer pill that Cost Plus sells for $13.40 for a 30-day supply, compared with the $2,500 it retails for at pharmacies. A study conducted by Dusetzina and colleagues found Medicare could save $662 million a year just by buying imatinib and six other generic cancer drugs from Cost Plus rather than through a big PBM. Ironically, though, most generic drugs are cheaper in the US than in Europe or Canada — so cheap, in fact, that they fall into shortages as companies get out of the business or stop making needed improvements to their production lines. In response, Cost Plus has started a compounding pharmacy to make common generics and soon hopes to have a sort of “private reserve” of 70 to 80 products that it can make on short notice if they go into shortage, Oshmyansky said. While the company hasn’t yet set up purchase agreements for most brand-name drugs, Oshmyansky and Cuban are hopeful. Drugmakers, through their trade group Pharmaceutical Research and Manufacturers of America, have lobbied fiercely to rein in PBMs in the past two years. At a Sept. 24 hearing at which Sen. Bernie Sanders (I-VT) grilled Novo Nordisk CEO Lars Fruergaard Jørgensen over high prices for diabetes and weight loss drugs Ozempic and Wegovy, the executive expressed support for a more transparent pricing model. “On average for our products we give 74% in rebates to PBMs” for every $1 the company charges, he said. If, instead, “we simply paid the PBMs a small fee for the limited risk and contribution they make, I think patients would be significantly better off.” Editor’s note: This story was written and edited by KFF Health News , and published in partnership with Endpoints. KFF Health News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF — the independent source for health policy research, polling, and journalism.

08 Dec 2024

·www.globenewswire.com

Longer-term data for Novartis Scemblix® reinforce superior efficacy with favorable safety and tolerability profile in adults with newly diagnosed CML

Scemblix demonstrated sustained superior major molecular response (MMR) vs. all investigator-selected TKIs (74.1% vs. 52%) and vs. imatinib alone (76.2% vs. 47.1%), meeting both ASC4FIRST 96-week key secondary endpoints1 Scemblix showed a clinically relevant 15.1% higher MMR rate vs. second generation (2G) TKIs (72.0% vs. 56.9%)1 96-week data extend favorable safety and tolerability profile for Scemblix vs. imatinib and 2G TKIs, with fewer grade ≥3 AEs and less than half the discontinuation rate due to AEs1 Latest results strengthen Scemblix as a standard of care following expanded indication in newly diagnosed and previously treated adult patents with Ph+ CML-CP and NCCN category 1 recommendation1-3 Basel, December 8, 2024 – Novartis today announced positive, longer-term results from the pivotal Phase III ASC4FIRST trial with Scemblix® (asciminib) showing superior major molecular response (MMR) rates at week 961. The study compared the MMR rate of Scemblix to investigator-selected standard-of-care (SoC) tyrosine kinase inhibitors (TKIs) (imatinib, nilotinib, dasatinib and bosutinib) and to imatinib alone in adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) at the week 96 evaluation, the study’s key secondary endpoints1. The longer-term results showed an increasing difference in Scemblix MMR rate vs. SoC, vs. imatinib and vs. 2G TKIs (nilotinib, dasatinib and bosutinib)1. Results were presented at the 66th American Society of Hematology Annual Meeting & Exposition (ASH)1. “These 96-week results are very encouraging for clinicians who aspire to obtain a balance of efficacy and tolerability profiles to help newly diagnosed adult CML patients achieve and maintain treatment goals,” said Jorge Cortes, M.D., Director, Georgia Cancer Center. “The sustained superior efficacy, deeper and more durable responses, and favorable safety and tolerability profile compared to standard of care TKIs continue to support the promise of Scemblix as a potentially practice-changing treatment option.” The median follow-up was 2.2 years for Scemblix and investigator-selected SoC TKIs1. Over 22% more patients treated with once-daily Scemblix achieved MMR at week 96 vs. all investigator-selected SoC TKIs, and nearly 30% more patients achieved MMR at week 96 vs. imatinib alone1. The Scemblix MMR rate was 15.1% (95% CI: 2.3, 28.0; not crossing zero) higher vs. 2G TKIs (72% vs. 56.9%)1. Patients treated with Scemblix also achieved deeper rates of molecular responses (MR4 and MR4.5) compared with investigator-selected SoC TKIs1. OverallaScemblix (n=201)vs. IS SoC TKIs (n=204) Imatinib stratumbScemblix (n=101)vs. imatinib (n=102) 2G TKI stratumcScemblix (n=100) vs. 2G TKIs (n=102) Key secondary endpoints MMR rates at week 96 74.1% vs. 52% 76.2% vs. 47.1% Secondary endpointsd MMR ratesat week 96 72% vs. 56.9% MR4at week 96 48.8% vs. 27.5% 52.5% vs. 23.5% 45% vs. 31.4% MR4.5at week 96 30.9% vs. 17.7% 35.6% vs. 11.8% 26% vs. 23.5% a All patients receiving Scemblix (n=201) or investigator-selected SoC TKIs (n=204). Treatment difference after adjusting for pre-randomization selected TKI and EUTOS long-term survival (ELTS) risk groups at baseline.b The 203 patients within the pre-randomization-selected imatinib stratum were randomized to receive either Scemblix (n=101) or imatinib (n=102). Treatment difference after adjusting for ELTS risk groups at baseline. c The 202 patients within the pre-randomization selected 2G TKIs stratum were randomized to receive either Scemblix (n=100) or 2G TKIs (n=102: nilotinib, 48%; dasatinib, 41%; bosutinib, 11%). d Secondary endpoints were not powered for statistical significance. The safety profile of Scemblix at 96-weeks was consistent with the 4-year follow-up of the Phase III ASCEMBL trial, with no new safety concerns observed to date1,2,4. Fewer grade ≥3 AEs and dose adjustments to manage AEs were reported for Scemblix, and discontinuation due to AEs was more than 50% lower for Scemblix vs. both imatinib and 2G TKIs1. The most frequent AEs (≥15%) were diarrhea, headache, fatigue, musculoskeletal pain, and rash1. Week 96 Scemblixn=200 Imatinibn=99 2G TKIsn=102 Grade ≥ AEsa 44.5% 49.5% 59.8% Discontinuation due to AEsa 5.5% 13.1% 12.7% AEs leading to dose adjustments/interruptionsa 33% 41.4% 57.8% aIn patients who experienced ≥1 adverse event. Novartis also presented today at ASH interim data from the Phase II ASC2ESCALATE dose-escalation study in both the second line (2L) and newly diagnosed Ph+ CML-CP settings5. In the analysis of 2L patients at week 24 (n=28) Scemblix demonstrated MMR rates of 42.9% and deep molecular responses (MR4 25% and MR4.5 10.7%), with a consistent safety and tolerability profile5. The most common AEs (>15%) were nausea, hypertension, and vomiting5. “Novartis’ decades-long work in CML and deep relationships within the community have informed our Scemblix clinical trial program of over 10 years, the centerpiece of our continuing drive to address ongoing unmet medical needs for people with CML,” said Jeff Legos, Executive Vice President, Global Head of Oncology Development, Novartis. “These latest findings reinforce the differentiated efficacy, safety and tolerability profile of Scemblix in newly diagnosed and previously treated adult CML patients.” Scemblix was recently granted accelerated approval in the US to treat newly diagnosed adults with Ph+ CML-CP, which together with its approval in previously treated adult patients with Ph+ CML-CP expands the population of Scemblix-eligible patients by four-fold 2. In addition, the National Comprehensive Cancer Network (NCCN) updated its Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for the treatment of CML, recommending asciminib as a category 1 – preferred treatment for newly diagnosed Ph+ CML-CP and across all risk categories3. About the ASC4FIRST Phase III Clinical Trial ASC4FIRST (NCT04971226) is a Phase III, head-to-head, multi-center, open-label, randomized study of oral Scemblix® 80 mg QD vs. IS first- or second-generation TKIs (imatinib, nilotinib, dasatinib or bosutinib) in 405 adult patients with newly diagnosed Ph+ CML-CP2,6. The trial met both primary endpoints with Scemblix demonstrating superior MMR rates at week 48 vs. investigator-selected SoC TKIs (imatinib, nilotinib, dasatinib and bosutinib) (67.7% vs. 49.0%) and imatinib alone (69.3% vs. 40.2%) as well as the secondary, non-powered endpoint for the 2G TKI stratum of (66% vs 57.8%)1,6. The study remains ongoing with further efficacy and safety readouts planned. About the ASC2ESCALATE Phase II StudyASC2ESCALATE (NCT05384587) is a Phase II, multicenter, single-arm, dose-escalation study of oral Scemblix® 80 mg QD in both the second line (2L) and newly diagnosed (1L) Ph+ CML-CP settings in the US 5,7. While Scemblix is already approved across lines of therapy, this is the first prospective trial to assess asciminib in the 2L setting and a dose-escalation strategy of asciminib as 2L and 1L treatment for patients with CML-CP not meeting molecular milestones 5. The proportion of patients achieving MMR at 12 months in the 2L setting will be measured as the primary endpoint 5. The study remains ongoing and has completed enrollment with 196 patients (100 patients in 2L, 96 patients in 1L)5. About Scemblix® (asciminib) Scemblix® is the first CML treatment that works by Specifically Targeting the ABL Myristoyl Pocket (referred to as a STAMP inhibitor in scientific literature)4,8,9. Other currently approved CML treatments are TKIs that target the ATP-binding site (ATP-competitive)9. In the US, Scemblix was granted accelerated approval to treat newly diagnosed adults with Ph+ CML-CP and is also approved for previously treated adult patients with Ph+ CML-CP. Outside the US, it is approved in more than 75 countries, including the EU, to treat those who have previously been treated with two or more TKIs with Ph+ CML-CP2,10,11. In some countries, including the US, Scemblix is also approved in patients with Ph+ CML-CP with the T315I mutation2,3,10. Scemblix is being studied across multiple treatment lines for Ph+ CML-CP, both as a monotherapy and in combination2,4,6,8,10,12-24. Patient Access and Support Novartis, with its 20+ year history in CML, is committed to continuing to address areas of unmet patient need and reducing barriers to patient access and affordability that prevent patients from benefiting from innovation. Novartis Patient Support is available to help guide eligible patients through the various aspects of getting started on treatment including help understanding insurance coverage and identifying potential financial assistance options. Patients or providers can call 866-433-8000 or visit support.scemblix.com to learn more. About Novartis Commitment to CML Novartis has a long-standing scientific commitment to patients living with CML. For more than two decades, our bold science has helped transform CML from a life-limiting condition for many patients. Despite these advancements, there’s still work to be done. We continue to research ways to target the disease more selectively and to address the challenges of not reaching treatment efficacy goals, experiencing treatment resistance and/or intolerance that many patients face. Our legacy inspires our future innovation – we continue to lead the way in developing novel medicines to address serious unmet needs in CML. Our commitment also goes beyond science. Our 20+ year collaboration with the Max Foundation has provided access to Gleevec (imatinib), Tasigna (nilotinib) and now Scemblix and is delivering tremendous patient impact in low- and middle-income countries, with over 100,000 patients supported to date. Disclaimer This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “may,” “committed,” “contingent,” “lead,” “continue,” “ongoing,” “to deliver,” “allowing,” “continuing,” “commitment,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for Scemblix, or regarding potential future revenues from Scemblix. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that Scemblix will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that Scemblix will be commercially successful in the future. In particular, our expectations regarding Scemblix could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise. About Novartis Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people’s lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach more than 250 million people worldwide. Reimagine medicine with us: Visit us at https://www.novartis.com and connect with us on LinkedIn, Facebook, X/Twitter and Instagram. References Cortes JE, Hochhaus A, Hughes TP, et al. Asciminib Continues to Provide Superior Efficacy and Favorable Safety and Tolerability vs Tyrosine Kinase Inhibitors In Newly Diagnosed Chronic Myeloid Leukemia in ASC4FIRST: Week 96 Update. Presented at: 66th ASH Annual Meeting & Exposition; December 7 – 10, 2024; San Diego, CA. Scemblix (asciminib) Prescribing information. East Hanover, New Jersey, USA: Novartis Pharmaceuticals Corporation; October 2024. National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) Chronic Myeloid Leukemia Version 2.2025. November 13, 2024. Accessed November 22, 2024. https://www.nccn.org/professionals/physician_gls/pdf/cml.pdf Rea D, Mauro MJ, Boquimpani C, et al. A Phase 3, Open-Label, Randomized Study of Asciminib, a STAMP Inhibitor, vs Bosutinib in CML After 2 or more prior TKIs. Blood. 2021;138(21):2031-2041. doi:10.1182/blood.2020009984 Atallah EL, Levy MY, Koller P, et al. Efficacy and Safety of Asciminib in Chronic Myeloid Leukemia in Chronic Phase (CML-CP): Interim Results from the Phase 2 ASC2ESCALATE Trial in the Cohort of Patients (Pts) after 1 Prior Tyrosine Kinase Inhibitor (TKI). Presented at: 66th ASH Annual Meeting & Exposition; December 7 – 10, 2024; San Diego, CA. A Study of Oral Asciminib Versus Other TKIs in Adult Patients With Newly Diagnosed Ph+ CML-CP (ASC4FIRST). ClinicalTrials.gov identifier: NCT04971226. Updated March 25, 2024. Accessed March 26, 2024. https://clinicaltrials.gov/study/NCT04971226 Asciminib Monotherapy, With Dose Escalation, for 2nd and 1st Line Chronic Myelogenous Leukemia (ASC2ESCALATE) ClinicalTrials.gov identifier: NCT05384587. Updated October 30, 2024. Accessed November 21, 2024. https://clinicaltrials.gov/study/NCT05384587 Cortes JE, Hughes TP, Mauro MJ, et al. Asciminib, a First-in-Class STAMP Inhibitor, Provides Durable Molecular Response in Patients (pts) with Chronic Myeloid Leukemia (CML) Harboring the T315I Mutation: Primary Efficacy and Safety Results from a Phase 1 Trial. Oral presentation at: ASH Annual Meeting; Dec. 7, 2020. Schoepfer J, Jahnke W, Berellini G, et al. Discovery of Asciminib (ABL001), an Allosteric Inhibitor of the Tyrosine Kinase Activity of BCR-ABL1. J Med Chem. 2018;61(18):8120-8135. doi:10.1021/acs.jmedchem.8b01040 Novartis data on file. Scemblix. EMA Summary of Product Characteristics. Novartis Europharm Limited; 2022. Wylie AA, Schoepfer J, Jahnke W, et al. The allosteric inhibitor ABL001 enables dual targeting of BCR–ABL1. Nature. 2017;543(7647):733-737. doi:10.1038/nature21702   Hughes TP, Mauro MJ, Cortes JE, et al. Asciminib in Chronic Myeloid Leukemia after ABL Kinase Inhibitor Failure. N Engl J Med. 2019; 381(24):2315-2326. doi:10.1056/NEJMoa1902328     Hughes TP, et al. Expanded Phase 1 Study of ABL001, a Potent, Allosteric Inhibitor of BCR-ABL, Reveals Significant and Durable Responses in Patients with CML-Chronic Phase with Failure of Prior TKI Therapy. Presented at: ASH Annual Meeting & Exposition; Dec. 5, 2016.   Ottmann OG, Alimena G, DeAngelo DJ, et al. ABL001, a Potent, Allosteric Inhibitor of BCR-ABL, Exhibits Safety and Promising Single- Agent Activity in a Phase I Study of Patients with CML with Failure of Prior TKI Therapy. Blood. 2015;126(23):138. doi:10.1182/blood.V126.23.138.138   Mauro MJ, Kim DW, Cortes J, et al. Combination of Asciminib Plus Nilotinib (NIL) or Dasatinib (DAS) in Patients (PTS) with Chronic Myeloid Leukemia (CML): Results from a Phase 1 Study. Presented at: EHA Annual Meeting; June 15, 2019.   Cortes JE, Lang F, Kim DW, et al. Combination Therapy Using Asciminib Plus Imatinib (IMA) in Patients (PTS) with Chronic Myeloid Leukemia (CML): Results from a Phase 1 Study. Presented at: EHA Annual Meeting; June 15, 2019.   Manley PW, Barys L, Cowan-Jacob SW. The specificity of asciminib, a potential treatment for chronic myeloid leukemia, as a myristate-pocket binding ABL inhibitor and analysis of its interactions with mutant forms of BCR-ABL1 kinase. Leuk Res. 2020;98:106458. doi:10.1016/j.leukres.2020.106458   Study of Efficacy of CML-CP Patients Treated with ABL001 Versus Bosutinib, Previously Treated With 2 or More TKIs. ClinicalTrials.gov identifier: NCT03106779. Updated February 7, 2024. Accessed April 3, 2024. https://clinicaltrials.gov/ct2/show/NCT03106779   Asciminib in Monotherapy for Chronic Myeloid Leukemia in Chronic Phase (CML-CP) With and WithoutT315I Mutation (AIM4CML). ClinicalTrials.gov identifier: NCT04666259. Updated September 7, 2023. Accessed April 3, 2024. https://clinicaltrials.gov/ct2/show/NCT04666259   Study of Efficacy And Safety Of Asciminib In Combination With Imatinib In Patients With Chronic Myeloid Leukemia In Chronic Phase (CML-CP). ClinicalTrials.gov identifier: NCT03578367. Updated March 22, 2024. Accessed April 3, 2024. https://clinicaltrials.gov/ct2/show/NCT03578367   Study of Efficacy and Safety of CML-CP Patients Treated With Asciminib Versus Best Available Therapy, Previously Treated With 2 or More Tyrosine Kinase Inhibitors. ClinicalTrials.gov identifier: NCT04795427. Updated October 19, 2023. Accessed April 3, 2024. https://clinicaltrials.gov/ct2/show/NCT04795427   A Phase I Study of Oral ABL001 in Patients With CML or Ph+ ALL. ClinicalTrials.gov identifier: NCT02081378. Updated March 18, 2024. Accessed April 3, 2024. https://clinicaltrials.gov/ct2/show/NCT02081378   Asciminib Treatment Optimization in ≥ 3rd Line CML-CP. ClinicalTrials.gov identifier: NCT04948333. Updated February 28, 2024. Accessed April 3, 2024. https://clinicaltrials.gov/ct2/show/NCT04948333  # # # Novartis Media RelationsE-mail: media.relations@novartis.com Central North America Anja von Treskow +41 79 392 9697 Michael Meo +1 862 274 5414 Anna Schäfers +41 79 801 7267 Switzerland Satoshi Sugimoto +41 79 619 2035 Novartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: investor.relations@novartis.com Central North America Isabella Zinck +41 61 324 7188 Sloan Simpson +1 862 345 4440 Nicole Zinsli-Somm +41 61 324 3809 Jonathan Graham +1 201 602 9921 Imke Kappes +41 61 324 8269 Parag Mahanti +1973 876 4912

Clinical ResultPhase 2Phase 3Drug ApprovalASH

28 Nov 2024

·www.prnewswire.com

New Indication of Olverembatinib Included into China 2024 National Reimbursement Drug List

ROCKVILLE, Md. and SUZHOU, China, Nov. 28, 2024 /PRNewswire/ -- Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in discovering, developing and commercializing therapies to address global unmet medical needs primarily for malignancies, today announced that the new indication - adult patients with chronic-phase chronic myeloid leukemia (CML-CP) resistant and/or intolerant of first-and second-generation tyrosine kinase inhibitors (TKIs) - of its novel drug, olverembatinib (HQP1351), has been included into the China 2024 National Reimbursement Drug List (NRDL), effective January 1, 2025. This indication was added to the NRDL through the simple contract renewal process. The previously included indication of adult patients with CML-CP or accelerated-phase CML (CML-AP) harboring the T315I mutation will maintain its reimbursable status on the NRDL. Olverembatinib is a novel drug developed by Ascentage Pharma with support from the National Major New Drug Development Program in China and the first third-generation BCR-ABL inhibitor approved by the China National Medical Products Administration (NMPA). As a novel drug, olverembatinib can effectively target BCR-ABL and a spectrum of BCR-ABL mutants, including the T315I mutation. In November 2021, olverembatinib received its first approval in China, thus became the first therapy approved for the treatment of T315I mutant CML in the country. In November 2023, olverembatinib was approved for the treatment of adult patients with CML-CP resistant and/or intolerant of first-and second-generation TKIs. Olverembatinib is being jointly commercialized in China by Ascentage Pharma and Innovent Biologics. CML is a hematologic malignancy of the white blood cells. The introduction of BCR-ABL TKIs have significantly improved the management of CML. However, TKI resistance is still a global challenge for CML treatment. 20%-40% of patients fail to achieve desired treatment outcome due to resistance or intolerance to TKIs1-3, thus leading to disease progression or even death. The successful renewal and the inclusion of additional indication of olverembatinib in the NRDL is expected to greatly improve the drug's accessibility, bringing benefit to more and a broader population of patients with CML in China. "We are glad that the new indication of olverembatinib has been included into the NRDL through the simple contract renewal process," said Dr. Dajun Yang, Chairman and CEO of Ascentage Pharma. "This decision reaffirms the importance of olverembatinib as an innovative drug that has the potential to fulfill the urgent unmet needs and treatment gaps in CML. With its new indication included into the NRDL at the third anniversary of the initial approval in China, olverembatinib is positioned to bring renewed hope to more patients with CML, thereby helping countless families and communities. Moving forward, we will work actively to ensure the rapid rollout of the expanded insurance coverage. We are confident these efforts will make olverembatinib more accessible and affordable, ultimately allowing more patients and their families to benefit from this novel therapeutic as soon as possible." References O'Brien SG, Guilhot F, Larson R, et al. Imatinib compared with interferon and low-dose cytarabine for newly diagnosed chronic-phase chronic myeloid leukemia. Engl J Med. 2003 Mar 13;348(11):994-1004. Jabbour E, Kantarjian H. Chronic myeloid leukemia: 2014 update on diagnosis, monitoring, and management. Am J Hematol. 2014 May;89(5):547-56. Larson R, Hochhaus A, Hughes T, et al. Nilotinib vs imatinib in patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase: ENESTnd 3-year follow-up. Leukemia. 2012 Oct;26(10):2197-203. * Olverembatinib is an investigational drug that has not been approved for any indication outside China. About Olverembatinib Developed by Ascentage Pharma, the novel drug olverembatinib is an orally-available third-generation tyrosine kinase inhibitor (TKI), and the first China-approved third-generation BCR-ABL inhibitor that can effectively target BCR-ABL and a spectrum of BCR-ABL mutants, including the T315I mutation. Olverembatinib, developed with support from the National Major New Drug Development Program in China, has also been granted Priority Review designations and Breakthrough Therapy designations by the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA). At present, olverembatinib has been approved in China for the treatment of adult patients with TKI-resistant chronic-phase chronic myeloid leukemia (CML-CP) or accelerated-phase CML (CML-AP) harboring the T315I mutation; and adult patients with CML-CP resistant and/or intolerant of first-and second-generation TKIs. In addition to CML, olverembatinib has also shown potential clinical benefits to patients with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). A global registrational Phase III study of olverembatinib in newly diagnosed patients with Ph+ ALL is currently underway, potentially paving the way for olverembatinib become the first China-approved TKI for the treatment of patients with Ph+ ALL in the first-line setting. In the area of solid tumors, olverembatinib is being evaluated in a global registrational Phase III study for the treatment of succinate dehydrogenase- (SDH-) deficient gastrointestinal stromal tumor (GIST) and has already been granted a Breakthrough Therapy designation by the China CDE. As a novel drug, olverembatinib has received widespread interest from the global hematology community. The clinical results of olverembatinib have been selected for oral presentations at the American Society of Hematology (ASH) Annual Meetings for seven consecutive years. To date, olverembatinib has been granted four Orphan Drug Designations and a Fast Track Designation by the US Food and Drug Administration (FDA), and an Orphan Designation by the EMA of the EU. In February 2024, olverembatinib was cleared by the US FDA to enter a global registrational Phase III study. In July 2021, Ascentage Pharma (6855.HK) and Innovent Biologics (1801.HK) reached the agreement regarding the joint development and commercialization of olverembatinib in China. In June 2024, Ascentage Pharma and Takeda, a multinational pharmaceutical company, entered into an Exclusive Option Agreement for olverembatinib. If exercised, the Option would allow Takeda to license global rights to develop and commercialize olverembatinib in all territories outside of, among others, the mainland China, Hong Kong China, Macau China, and Taiwan China. About Ascentage Pharma Ascentage Pharma (6855.HK) is a global, integrated biopharmaceutical company engaged in discovering, developing and commercializing therapies to address global unmet medical needs primarily in malignancies. On October 28, 2019, Ascentage Pharma was listed on the Main Board of the Stock Exchange of Hong Kong Limited with the stock code 6855.HK. The company has built a rich pipeline of innovative drug candidates that includes novel, highly potent Bcl-2 and dual Bcl-2/Bcl-xL inhibitors, as well as candidates aimed at IAP and MDM2-p53 pathways, and next-generation TKIs. Ascentage Pharma is also the only company in the world with active clinical programs targeting all three known classes of key apoptosis regulators. The company has conducted more than 40 clinical trials in the US, Australia, Europe, and China, including 13 registrational studies (completed/ ongoing/planned). Olverembatinib, the company's first lead asset developed for the treatment of drug-resistant chronic myeloid leukemia (CML) and the company's first approved product in China, has been granted Priority Review Designations and Breakthrough Therapy Designations by the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA). To date, the drug had been included into the China National Reimbursement Drug List (NRDL). Furthermore, olverembatinib has been granted Orphan Drug Designations (ODDs) and a Fast Track Designation (FTD) by the US FDA, and an Orphan Designation by the EMA of the EU. To date, Ascentage Pharma has obtained a total of 16 ODDs from the US FDA and 1 Orphan Designation from the EMA of the EU for 4 of the company's investigational drug candidates. Leveraging its robust R&D capabilities, Ascentage Pharma has built a portfolio of global intellectual property rights and entered into global partnerships and other relationships with numerous leading biotechnology and pharmaceutical companies such as Takeda, AstraZeneca, Merck, Pfizer and Innovent; and research and development relationships with leading research institutions such as Dana-Farber Cancer Institute, Mayo Clinic, National Cancer Institute and the University of Michigan. The company has built a talented team with a wealth of global experience in the discovery and development of innovative drugs and fully functional commercial manufacturing and Sales & Marketing teams. One pivotal aim of Ascentage Pharma is to continuously strengthen its R&D capabilities and accelerate its clinical development programs, in order to fulfil its mission of addressing unmet clinical needs in China and around the world for the benefit of more patients. Forward-Looking Statements 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, Ascentage Pharma undertakes 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 and expectations or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions and expectations may alter in light of future development. SOURCE Ascentage Pharma WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Orphan DrugPhase 3Fast TrackBreakthrough TherapyDrug Approval

100 Deals associated with Imatinib mesylate

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KEGG	Wiki	ATC	Drug Bank
D01441	Imatinib mesylate	L01XE01	DB00619

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Accelerated phase Philadelphia chromosome positive chronic myeloid leukemia	US	Shorla Pharma Ltd.Startup	22 Nov 2024
Philadelphia chromosome positive chronic myeloid leukemia in blast crisis	US	Shorla Pharma Ltd.Startup	22 Nov 2024
Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase	US	Shorla Pharma Ltd.Startup	22 Nov 2024
Metastatic Gastrointestinal Stromal Tumor	EU	Accord Healthcare SLU	30 Jun 2013
Metastatic Gastrointestinal Stromal Tumor	IS	Accord Healthcare SLU	30 Jun 2013
Metastatic Gastrointestinal Stromal Tumor	LI	Accord Healthcare SLU	30 Jun 2013
Metastatic Gastrointestinal Stromal Tumor	NO	Accord Healthcare SLU	30 Jun 2013
Myeloproliferative Disorders	EU	Teva Pharmaceuticals Europe BV	07 Jan 2013
Myeloproliferative Disorders	IS	Teva Pharmaceuticals Europe BV	07 Jan 2013
Myeloproliferative Disorders	LI	Teva Pharmaceuticals Europe BV	07 Jan 2013
Myeloproliferative Disorders	NO	Teva Pharmaceuticals Europe BV	07 Jan 2013
Ph-Like Acute Lymphoblastic Leukemia	EU	Teva Pharmaceuticals Europe BV	07 Jan 2013
Ph-Like Acute Lymphoblastic Leukemia	IS	Teva Pharmaceuticals Europe BV	07 Jan 2013
Ph-Like Acute Lymphoblastic Leukemia	LI	Teva Pharmaceuticals Europe BV	07 Jan 2013
Ph-Like Acute Lymphoblastic Leukemia	NO	Teva Pharmaceuticals Europe BV	07 Jan 2013
Philadelphia Chromosome Positive Leukemia	EU	Teva Pharmaceuticals Europe BV	07 Jan 2013
Philadelphia Chromosome Positive Leukemia	IS	Teva Pharmaceuticals Europe BV	07 Jan 2013
Philadelphia Chromosome Positive Leukemia	LI	Teva Pharmaceuticals Europe BV	07 Jan 2013
Philadelphia Chromosome Positive Leukemia	NO	Teva Pharmaceuticals Europe BV	07 Jan 2013
Acute Lymphoblastic Leukemia	US	Novartis Pharmaceuticals Corp.	19 Oct 2006

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Mixed phenotype acute leukemia	Phase 3	US	National Cancer Institute	08 Aug 2017
Mixed phenotype acute leukemia	Phase 3	AU	National Cancer Institute	08 Aug 2017
Mixed phenotype acute leukemia	Phase 3	AT	National Cancer Institute	08 Aug 2017
Mixed phenotype acute leukemia	Phase 3	BE	National Cancer Institute	08 Aug 2017
Mixed phenotype acute leukemia	Phase 3	CA	National Cancer Institute	08 Aug 2017
Mixed phenotype acute leukemia	Phase 3	CL	National Cancer Institute	08 Aug 2017
Mixed phenotype acute leukemia	Phase 3	CZ	National Cancer Institute	08 Aug 2017
Mixed phenotype acute leukemia	Phase 3	FI	National Cancer Institute	08 Aug 2017
Mixed phenotype acute leukemia	Phase 3	FR	National Cancer Institute	08 Aug 2017
Mixed phenotype acute leukemia	Phase 3	DE	National Cancer Institute	08 Aug 2017

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03578367 (ASC4MORE, Pubmed \| J Hematol Oncol)	Phase 2	Chronic phase chronic myeloid leukemia Add-on	84	Asciminib 40 mg QD add-on to Imatinib 400 mg QD	gmgjhlndip(wbqxzxehpz) = lqrufilqxh vbvdfebytt (crusvazygv ) View more	Positive	18 Dec 2024
				Asciminib 60 mg QD add-on to Imatinib 400 mg QD	gmgjhlndip(wbqxzxehpz) = qlpwpodkmm vbvdfebytt (crusvazygv ) View more
ASH2024	Not Applicable	Philadelphia positive acute lymphocytic leukaemia	-	Imatinib + low intensity chemotherapy	lxgmyulblf(jkhgjnhuau) = dabjgfjhds lovokdofvo (efzbenyjnp )	-	09 Dec 2024
				Imatinib (Allogeneic SCT)	lxgmyulblf(jkhgjnhuau) = krrmvvdleb lovokdofvo (efzbenyjnp )
ASH2024	Not Applicable	Philadelphia chromosome positive chronic myelogenous leukemia	-	Imatinib	xicombnkmk(jaicwnqyrj): HR = 3.11 (95% CI, 1.25 - 7.77), P-Value = 0.015	-	09 Dec 2024
				Nilotinib
NCT04070443 (TIPI, ASH2024)	Phase 2	Philadelphia chromosome positive chronic myelogenous leukemia	-	Ponatinib 30 mg QD	vfcqgmctns(eqhfqnpwyr) = 2 hematologic (grade 4 neutropenia at M6) ofhdrbqamw (pwimqzlhcy )	Positive	08 Dec 2024
				Imatinib 400 mg QD
ASH2024	Not Applicable	-	-	Imatinib (TKI-Sensitive (TKI-S) patients)	fqxmnfvvrx(nenhuamcvq) = fnsfrdtbil kbfwyjmyns (mpayinchtk ) View more	-	08 Dec 2024
				Imatinib (TKI-Resistant (TKI-R) patients)	fqxmnfvvrx(nenhuamcvq) = mehwhzyzjt kbfwyjmyns (mpayinchtk ) View more
FDA_CDER Manual	Phase 2	Chronic phase chronic myeloid leukemia First line	51	Imatinib 340 mg/m2/day (pediatric patients)	ovqfthurxa(erivpbddyr) = lqlzkjxslm lckiwxrfyh (lwpfqlkooz ) View more	Positive	22 Nov 2024
FDA_CDER Manual	Phase 2	Myelodysplastic-Myeloproliferative Diseases	31	Imatinib	xepixgglne(vnsvnknkfa) = appkaafwgo zcerlngwes (bccwzazwqw ) View more	Positive	22 Nov 2024
				Imatinib (Chromosome 5 translocation)	xepixgglne(vnsvnknkfa) = ypzinybqmp zcerlngwes (bccwzazwqw ) View more
FDA_CDER Manual	Phase 2	Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase \| Philadelphia chromosome positive chronic myeloid leukemia in blast crisis \| Accelerated phase Philadelphia chromosome positive chronic myeloid leukemia	-	imatinib (Chronic phase IFN failure)	iqvvosegjt(gdblhpzakt) = gqdqdvfvyd bsvghatstb (vlegwajnvo, 92.3−96.3) View more	Positive	22 Nov 2024
				imatinib (accelerated phase)	iqvvosegjt(gdblhpzakt) = yselmzszls bsvghatstb (vlegwajnvo, 64.8−76.8) View more
FDA_CDER Manual	Not Applicable	Unresectable Gastrointestinal Stromal Tumor	713	Imatinib 400 mg/day	bjewrrgsiu(msodwkwhsa) = ewhpttoytd rxnhgauvlx (rampuumpqf ) View more	Positive	22 Nov 2024
				Placebo	bjewrrgsiu(msodwkwhsa) = bqwzhypyoo rxnhgauvlx (rampuumpqf ) View more
FDA_CDER Manual	Phase 3	Gastrointestinal Stromal Tumors KIT Positive	397	Imatinib 400 mg/day for 12 months	heeijpmszy(fihaysgqht) = mszxgdwtir axkitrpabw (vncbzcpsxu ) View more	Positive	22 Nov 2024
				Imatinib 400 mg/day for 36 months	heeijpmszy(fihaysgqht) = olsvxaciuu axkitrpabw (vncbzcpsxu ) View more

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