Bcr-Abl inhibitors(Bcr-Abl tyrosine kinase inhibitors), CSF-1R antagonists(Colony stimulating factor 1 receptor antagonists), DDR1 antagonists(Discoidin domain receptor 1 antagonists)

Therapeutic Areas

NeoplasmsHemic and Lymphatic DiseasesDigestive System Disorders+ [3]

Active Indication

Aggressive-Phase Chronic Myelocytic LeukemiaAccelerated phase Philadelphia chromosome positive chronic myeloid leukemiaPhiladelphia chromosome-positive chronic myeloid leukemia in chronic phase+ [7]

Inactive Indication

Acoustic NeuromaAcral Lentiginous Malignant MelanomaAcute Lymphoblastic Leukemia+ [25]

Originator Organization

Novartis Pharma AG

Active Organization

Novartis Pharmaceuticals Corp.Novartis Pharma Schweiz AGNovartis Europharm Ltd.

+ [8]

Inactive Organization

Novartis Pharma Stein AG

Beijing Novartis Pharma Co. Ltd.Novartis Pharma AG

Drug Highest PhaseApproved

First Approval Date

CH (24 Jul 2007),

Philadelphia chromosome positive chronic myelogenous leukemia

RegulationAccelerated Approval (US), Orphan Drug (JP), Orphan Drug (US)

Structure

Molecular FormulaC28H25ClF3N7O2

InChIKeyYCBPQSYLYYBPDW-UHFFFAOYSA-N

CAS Registry923288-90-8

235

Clinical Trials associated with Nilotinib Hydrochloride

IRCT20200623047902N17 / Suspended

Bioequivalence study of Nilotinib 200mg capsule manufactured by Rooyan darou versus Tasigna 200 mg in healthy volunteers in the fasted condition

Start Date22 Oct 2024

Sponsor / Collaborator-

NCT06409936 / Not yet recruitingPhase 2IIT

Asciminib as Single Agent or in Combination With Nilotinib in the 1st-line Treatment of BCR-ABL1+ Chronic Myeloid Leukemia: a Randomized GIMEMA-GELMC Phase II Study PEARL Study: PotEntial of Asciminib in the eaRly Treatment of CML

A phase 2, interventional, randomized unblinded study will be conducted in newly diagnosed CP CML patients, to investigate the efficacy and the safety of asciminib at a dose of 80 mg QD as single agent (arm A) or 40 mg BID in combination with nilotinib 300 mg BID (arm B).
All patients in both arm A and arm B will be treated for a minimum of 2 years (core phase). If they will have achieved a DMR (MR4), or if it will be in the interest of the patient, the treatment will be continued.
During the consolidation phase (2 years) asciminib will be continued at the same dose in both arms; in the combination arm the nilotinib dose will be reduced to 300 mg daily.
The patients maintaining a stable MR4 up to the end of the fourth year will discontinue the treatment (TFR phase). The rate of TFR at 5 year (1 year after discontinuation) will be evaluated.

Start Date01 Sep 2024

Sponsor / Collaborator

Gruppo Italiano Malattie EMatologiche dell'Adulto

CTIS2024-514448-10-00 / Not yet recruiting

- BLCL-NIL-FDA-02

Start Date09 Aug 2024

Sponsor / Collaborator

Bluepharma Indústria Farmacêutica SA

100 Clinical Results associated with Nilotinib Hydrochloride

100 Translational Medicine associated with Nilotinib Hydrochloride

100 Patents (Medical) associated with Nilotinib Hydrochloride

2,656

Literatures (Medical) associated with Nilotinib Hydrochloride

01 Jan 2025·EUROPEAN JOURNAL OF HAEMATOLOGY

Choice of Frontline Tyrosine‐Kinase Inhibitor and Early Events in Very Elderly Patients With Chronic Myeloid Leukemia in Chronic Phase: A “Campus CML” Study

Article

Author: Loglisci, G. ; Capodanno, I. ; Galimberti, S. ; Crugnola, M. ; Sancetta, R. ; Elena, C. ; Breccia, M. ; Saglio, G. ; Abruzzese, E. ; Luciano, L. ; Attolico, I. ; Scortechini, A. R. ; Cavazzini, F. ; Malato, A. ; Tiribelli, M. ; Lunghi, F. ; Vincelli, D. I. ; Luzi, D. ; Miggiano, M. C. ; Crescenzi, S. Leonetti ; Russo, S. ; Crisà, E. ; Bucelli, C. ; Bonifacio, M. ; Murgano, P. ; Carmosino, I. ; Latagliata, R. ; Sorà, F. ; Annunziata, M. ; Caocci, G. ; Fava, C. ; Mullai, R. ; Di Veroli, A. ; Stagno, F. ; Rapezzi, D. ; Iurlo, A. ; Leporace, A. P. ; Bocchia, M. ; Binotto, G. ; Maggi, A. ; Sportoletti, P. ; Specchia, G.

ABSTRACT:

Objectives:

The study aimed to evaluate the utilization of frontline TKI therapy in a large cohort of elderly CP‐CML patients.

Methods:

A retrospective analysis was conducted on 332 CP‐CML patients aged 75 years or older among 1929 diagnosed from January 2012 to December 2019 followed at 36 participating Hematology Centers involved in the “Campus CML” project.

Results:

Among the patients analyzed, 85.8% received imatinib (IM) while 14.2% received second‐generation TKIs (2G‐TKI), 59.5% dasatinib, and 40.5% nilotinib. Most patients initiated IM at standard dose (67.3%) while 32.7% at reduced dose. A similar trend was observed with 2G‐TKIs. The cumulative incidence of permanent TKI discontinuation at 12 months was 28.4%, primarily due to primary resistance (10.1%) and extra‐hematologic toxicity (9.5%), with no significant difference between IM and 2G‐TKI groups. Following the introduction of generic IM in Italy in 2018, IM usage increased significantly compared with 2G‐TKIs.

Conclusions:

IM was in our Centers the preferred frontline therapy for older CP‐CML patients, with increasing utilization after the introduction of generic formulations. However, 2G‐TKIs are still used in a substantial proportion of patients, suggesting individualized physician assessments regarding patient suitability and expectations. Further investigation is needed to assess efficacy and safety of reduced TKI doses in this patient population.

01 Dec 2024·MOLECULAR BIOLOGY REPORTS

Investigating the impact of SMAD2 and SMAD4 downregulation in colorectal cancer and their correlation with immune markers, prognosis, and drug resistance and sensitivity

Article

Author: Peymani, Maryam ; Amani, Melika Saadat

BACKGROUND:

While many genes linked to colorectal cancer (CRC) contribute to cancer development, a thorough investigation is needed to explore crucial hub genes yet to be fully studied. A pivotal pathway in CRC is transforming growth factor-beta (TGF-β). This study aimed to assess SMAD2 and SMAD4 gene expression from this pathway.

METHODS AND RESULTS:

Counted data from the Cancer Genome Atlas (TCGA) were examined, comparing 483 tumor and 41 normal samples. Using clinical data, genes impacting overall survival (OS) were evaluated. GSE39582 was employed to confirmed the levels of genes in CRC compared to the normal samples. Additionally, employing unhealthy samples and the RT-qPCR means our outcomes was validated. Finally, PharmacoGx information were utilized to connect the levels of potential genes to drug tolerance and susceptibility. Our findings showed SMAD2 and SMAD4 levels in TGF-β signaling were more significant than other pathway genes. Our findings indicated that the protein levels of these genes were lower in malignant tissues than in healthy tissues. Results revealed a significant correlation between low levels of SMAD2 and unfavorable OS in CRC individuals. RT-qPCR results demonstrated decreased expressions of both SMAD2 and SMAD4 in cancer tissues compared to elevated levels in adjacent normal samples. Our results showed significant association between selected genes and immune cell infiltration markers such as CD8+, and B-cells. Our results indicated a potential association among the levels of SMAD2 and SMAD4 genes and tolerance and susceptibility to Nilotinib and Panobinostat drugs.

CONCLUSION:

Reduced expression of SMAD2 and SMAD4 may be pivotal in CRC progression, impacting downstream genes unrelated to patient OS. These findings suggest a potential role for SMAD2 and SMAD4 as predictive markers for drug response in CRC patients.

01 Dec 2024·Current computer-aided drug design

Identification of Prognostic Markers and Potential Therapeutic Targets using Gene Expression Profiling and Simulation Studies in Pancreatic Cancer

Article

Author: Singh, Samvedna ; Jhinjharia, Divya ; Gupta, Himanshi ; Kaushik, Aman Chandra ; Sahi, Shakti

Background::

Pancreatic ductal adenocarcinoma (PDAC) has a 5-year relative survival rate of less than 10% making it one of the most fatal cancers. A lack of early measures of prognosis, challenges in molecular targeted therapy, ineffective adjuvant chemotherapy, and strong resistance to chemotherapy cumulatively make pancreatic cancer challenging to manage

Objective::

The present study aims to enhance understanding of the disease mechanism and its progression by identifying prognostic biomarkers, potential drug targets, and candidate drugs that can be used for therapy in pancreatic cancer.

Methods::

Gene expression profiles from the GEO database were analyzed to identify reliable prognostic markers and potential drug targets. The disease's molecular mechanism and biological pathways were studied by investigating gene ontologies, KEGG pathways, and survival analysis to understand the strong prognostic power of key DEGs. FDA-approved anti-cancer drugs were screened through cell line databases, and docking studies were performed to identify drugs with high affinity for ARNTL2 and PIK3C2A. Molecular dynamic simulations of drug targets ARNTL2 and PIK3C2A in their native state and complex with nilotinib were carried out for 100 ns to validate their therapeutic potential in PDAC.

Results::

Differentially expressed genes that are crucial regulators, including SUN1, PSMG3, PIK3C2A, SCRN1, and TRIAP1, were identified. Nilotinib as a candidate drug was screened using sensitivity analysis on CCLE and GDSC pancreatic cancer cell lines. Molecular dynamics simulations revealed the underlying mechanism of the binding of nilotinib with ARNTL2 and PIK3C2A and the dynamic perturbations. It validated nilotinib as a promising drug for pancreatic cancer.

Conclusion::

This study accounts for prognostic markers, drug targets, and repurposed anti-cancer drugs to highlight their usefulness for translational research on developing novel therapies. Our results revealed potential and prospective clinical applications in drug targets ARNTL2, EGFR, and PI3KC2A for pancreatic cancer therapy.

News (Medical) associated with Nilotinib Hydrochloride

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

03 Dec 2024

·www.globenewswire.com

Terns Pharmaceuticals Announces Positive Early Data from Phase 1 CARDINAL Trial of TERN-701 for Chronic Myeloid Leukemia

Compelling molecular responses starting at lowest dose level in heavily pre-treated patients with high baseline BCR-ABL transcript levels Encouraging safety profile with no dose limiting toxicities, adverse event-related treatment discontinuations, or dose reductions across three dose escalation cohorts High levels of target coverage achieved with once daily dosing at all doses Completion of dose escalation and initiation of dose expansion expected in 1H25 Additional efficacy data expected in 4Q25, including longer term major molecular response (MMR) rates Company to host webcast at 8:00 am ET today FOSTER CITY, Calif., Dec. 03, 2024 (GLOBE NEWSWIRE) -- Terns Pharmaceuticals, Inc. (“Terns” or the “Company”) (Nasdaq: TERN), a clinical-stage biopharmaceutical company developing a portfolio of small-molecule product candidates to address serious diseases, including oncology and obesity, today announced encouraging early data from the ongoing dose escalation part of the Phase 1 CARDINAL study evaluating TERN-701 in patients with relapsed/refractory chronic myeloid leukemia (CML). TERN-701 is an investigational, oral, potent, small molecule allosteric BCR-ABL inhibitor being developed for patients with CML. CARDINAL is a global, multicenter, open-label, two-part Phase 1 clinical trial to evaluate the safety, pharmacokinetics (PK), and efficacy of TERN-701 in patients with relapsed/refractory CML with or without BCR-ABL resistance mutations who were previously treated with at least one 2G tyrosine kinase inhibitor (TKI). Patients previously treated with asciminib are also eligible. “These exciting early data from our Phase 1 dose escalation cohorts clearly show TERN-701 has compelling clinical activity with a highly encouraging cumulative MMR rate of 50% at 3 months. At the first two dose levels, we see clinically meaningful molecular and hematologic responses in patients with high baseline BCR-ABL transcript levels who had poor responses on prior 2G TKIs, 3G TKIs including ponatinib, as well as asciminib,” said Emil Kuriakose MD, chief medical officer of Terns. “The emerging safety data show a profile supporting best-in-class potential with no dose limiting toxicities across three completed dose levels, no clinically meaningful changes in liver or pancreatic enzymes, and no AE-related dose reductions or discontinuations at doses that achieve plasma exposures well above target efficacious concentrations. Taken together, the clinical activity and safety data across the dose range in these heavily pre-treated patients with refractory disease support a potential wide therapeutic index that allows for high levels of target coverage with favorable safety/tolerability.” “We are thrilled to share these impactful early data from the Phase 1 CARDINAL study of TERN-701, which support its potential to be a best-in-class allosteric inhibitor for the treatment of CML,” said Amy Burroughs, chief executive officer of Terns. “In addition to the meaningful clinical data, the CARDINAL study highlights yet another example of excellent clinical and operational execution at Terns, with patients enrolled in all four dose escalation cohorts in less than a year. We are well-positioned to initiate dose expansion cohorts in the first half of 2025 and look forward to sharing additional safety and efficacy data, including longer term MMR data in late 2025.” As of the October 28, 2024 cutoff date, 15 patients were enrolled across three dose levels of 160mg (n=7), 320mg (n=5), and 400mg (n=3) of TERN-701 dosed once daily, with an overall median treatment duration of 3 months (range 0.79 - 7.5 months). Enrolled patients were heavily pretreated with a median of 4 prior TKIs (range: 1-6) and 80% having had 3 or more TKIs. 47% and 40% of patients, respectively, had previously received ponatinib and asciminib. 73% were not in MMR at baseline, with 60% having a baseline BCR-ABL transcript >1% international scale (IS). As of the data cutoff, 14 of 15 patients remain on treatment. Twelve patients were efficacy evaluable, defined as having baseline BCR-ABL transcript and at least two post-baseline BCR-ABL transcript levels (centrally assessed). All efficacy evaluable patients were in the 160mg and 320mg dose levels. Key efficacy highlights include: 88% (7/8) of patients with baseline transcript > 1% had decreases in BCR-ABL on treatment, with 7 ongoing as of data cutoffCumulative MMR rate of 50% (5/10) in non-T315i mutation patients with 3 or more months of treatment and/or MMR or better at baseline100% (4/4) of patients with MMR or better at baseline have maintained their response and remain on treatmentAdditional notable responses include: MR2 within 5 months in a 4L patient at 160mg QD with baseline transcript > 1% and suboptimal response and intolerance to asciminibMR4 (deep molecular response) within 3 months in a 5L patient treated at 320mg with baseline transcript >10% and treatment failure on bosutinib at study entry TERN-701 showed a highly encouraging safety profile across the 160mg to 400mg dose levels, with 500mg undergoing evaluation as of data cutoff. Key safety highlights: No dose limiting toxicities (DLT) through 400mg dose levelNo adverse event (AE)-related treatment discontinuations or dose reductionsNo Grade 3 or higher treatment-related AEsNo treatment related serious AEs The incidence of treatment emergent hematologic AEs was notably low in this heavily pre-treated population, with no Grade 3 or higher treatment-related cytopenias. There were no non-hematologic treatment-related AEs more than Grade 2 in severity. Finally, no clinically meaningful changes in liver and pancreatic enzymes, blood pressure and other vitals, or electrocardiogram were seen. Steady state PK data, available for the 160mg and 320mg dose levels at data cutoff, showed linear PK with dose proportional increases in exposure. Plasma protein binding-corrected Caverage for TERN-701 exceeded the in vitro IC90 for multiple mutated and non-mutated BCR-ABL variants with once daily dosing. Importantly, at 160mg and 320mg QD, TERN-701 achieved average free drug concentrations approximately 4-fold and 8-fold higher, respectively, than in vivo exposures where potent inhibition of the BCR-ABL signaling pathway in was seen in CML mouse tumor models, indicating robust pharmacodynamic effects at these clinical doses. As of December 3, 2024, the CARDINAL study has enrolled 19 patients inclusive of the 500mg cohort, with all dose escalation cohorts having enrolled at least 3 patients. The study is on track to initiate dose expansion in the first half of 2025 with additional efficacy data expected in the fourth quarter of 2025, including longer term MMR rates. Company WebcastTerns will host a company webcast at 8:00 am ET today. The discussion will cover TERN-701’s Phase 1 interim data, next steps for the CARDINAL program, and TERN-701’s potential role in the CML treatment landscape. The event will be webcast live and can be accessed by visiting the investor relations section of the Company’s website at https://ir.ternspharma.com. An archived webcast will be available following the event. About CARDINALThe CARDINAL trial is an ongoing global, multicenter, open-label, two-part Phase 1 clinical trial to evaluate the safety, PK, and efficacy of TERN-701 in patients with previously treated CML. Part 1 is the dose escalation portion of the trial evaluating once-daily TERN-701 monotherapy in up to five dose cohorts in up to 60 adults with chronic phase CML with confirmed BCR-ABL and a history of treatment failure or suboptimal response to at least one second generation TKI (nilotinib, dasatinib or bosutinib). Participants who are intolerant to prior TKI treatment (including asciminib) are also allowed. The primary endpoints for Part 1 are the incidence of DLTs during the first treatment cycle, and additional measures of safety and tolerability. Secondary endpoints include TERN-701 PK and efficacy assessments, such as hematologic and molecular responses as measured by the change from baseline in BCR-ABL transcript levels. The starting dose is 160 mg QD (once-daily) with dose escalations as high as 500 mg QD and the option to explore a lower dose of 80 mg QD. Part 2 is the dose expansion portion of the trial that will enroll approximately 40 patients, randomized to once-daily treatment with one of two doses of TERN-701 to be selected based on data from Part 1. The primary endpoint of the dose expansion portion of the trial is efficacy, measured by hematologic and molecular responses. Secondary endpoints include safety, tolerability and PK. The overall objective of the CARDINAL trial is to select the optimal dose(s) of TERN-701 to move forward to a potential pivotal trial in chronic phase CML. About Terns PharmaceuticalsTerns Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing a portfolio of small-molecule product candidates to address serious diseases, including oncology and obesity. Terns’ pipeline contains three clinical stage development programs including an allosteric BCR-ABL inhibitor, a small-molecule GLP-1 receptor agonist, a THR-β agonist, and a preclinical GIPR modulator discovery effort, prioritizing a GIPR antagonist nomination candidate. For more information, please visit: www.ternspharma.com. Cautionary Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements about the Company within the meaning of the federal securities laws, including those related to expectations, timing and potential results of the clinical trials and other development activities of the Company and its partners; the potential indications to be targeted by the Company with its small-molecule product candidates; the therapeutic potential of the Company’s small-molecule product candidates; the potential for the mechanisms of action of the Company’s product candidates to be therapeutic targets for their targeted indications; the potential utility and progress of the Company’s product candidates in their targeted indications, including the clinical utility of the data from and the endpoints used in the Company’s clinical trials; the Company’s clinical development plans and activities, including the results of any interactions with regulatory authorities on its programs; the Company’s expectations regarding the profile of its product candidates, including efficacy, tolerability, safety, metabolic stability and pharmacokinetic profile and potential differentiation as compared to other products or product candidates; the Company’s plans for and ability to continue to execute on its current development strategy, including potential combinations involving multiple product candidates; the potential commercialization of the Company’s product candidates; the Company’s plans and expectations around the addition of key personnel; and the Company’s expectations with regard to its cash runway and sufficiency of its cash resources. All statements other than statements of historical facts contained in this press release, including statements regarding the Company’s strategy, future financial condition, future operations, future trial results, projected costs, prospects, plans, objectives of management and expected market growth, are forward-looking statements. In some cases, you can identify forward-looking statements by terminology such as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “design,” “due,” “estimate,” “expect,” “goal,” “intend,” “may,” “objective,” “plan,” “positioned,” “potential,” “predict,” “seek,” “should,” “target,” “will,” “would” and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology. The Company has based these forward-looking statements largely on its current expectations, estimates, forecasts and projections about future events and financial trends that it believes may affect its financial condition, results of operations, business strategy and financial needs. In light of the significant uncertainties in these forward-looking statements, you should not rely upon forward-looking statements as predictions of future events. These statements are subject to risks and uncertainties that could cause the actual results and the implementation of the Company’s plans to vary materially, including the risks associated with the initiation, cost, timing, progress, results and utility of the Company’s current and future research and development activities and preclinical studies and clinical trials. These risks are not exhaustive. For a detailed discussion of the risk factors that could affect the Company’s actual results, please refer to the risk factors identified in the Company’s SEC reports, including but not limited to its Annual Report on Form 10-K for the year ended December 31, 2023. Except as required by law, the Company undertakes no obligation to update publicly any forward-looking statements for any reason. Contacts for Terns InvestorsJustin Nginvestors@ternspharma.com MediaJenna UrbanBerry & Company Public Relationsmedia@ternspharma.com

Phase 1Clinical Result

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 Nilotinib Hydrochloride

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KEGG	Wiki	ATC	Drug Bank
D06413	Nilotinib Hydrochloride	L01XE08	DB04868

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Aggressive-Phase Chronic Myelocytic Leukemia	US	Novartis Pharmaceuticals Corp.	22 Jan 2014
Accelerated phase Philadelphia chromosome positive chronic myeloid leukemia	US	Novartis Pharmaceuticals Corp.	29 Oct 2007
Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase	US	Novartis Pharmaceuticals Corp.	29 Oct 2007
Philadelphia chromosome positive chronic myelogenous leukemia	CH	Novartis Pharma Schweiz AG	24 Jul 2007

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Chronic phase chronic myeloid leukemia	NDA/BLA	EU	Novartis Europharm Ltd.	27 Jun 2024
Blast Phase Chronic Granulocytic Leukemia	Phase 3	CO	Novartis Pharmaceuticals Corp.	01 Jan 2009
Blast Phase Chronic Granulocytic Leukemia	Phase 3	VE	Novartis Pharmaceuticals Corp.	01 Jan 2009
Cerebral Palsy	Phase 3	KR	Novartis AG	01 Jan 2009
Metastatic Gastrointestinal Stromal Tumor	Phase 3	DE	Novartis Pharmaceuticals Corp.	01 Nov 2008
Metastatic Gastrointestinal Stromal Tumor	Phase 3	IT	Novartis Pharmaceuticals Corp.	01 Nov 2008
Gastrointestinal Stromal Tumors	Phase 3	US	Novartis Pharmaceuticals Corp.	01 Mar 2007
Gastrointestinal Stromal Tumors	Phase 3	AU	Novartis Pharmaceuticals Corp.	01 Mar 2007
Gastrointestinal Stromal Tumors	Phase 3	AT	Novartis Pharmaceuticals Corp.	01 Mar 2007
Gastrointestinal Stromal Tumors	Phase 3	CA	Novartis Pharmaceuticals Corp.	01 Mar 2007

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASH2024	Not Applicable	Philadelphia chromosome positive chronic myelogenous leukemia	-	Nilotinib	dydisrqpvn(zwonshknpb) = increased creatinine and thyroid disease in flumatinib group sxesnyyqqo (adochruekt ) View more	-	09 Dec 2024
				Dasatinib
ASH2024	Not Applicable	Philadelphia chromosome positive chronic myelogenous leukemia	-	Imatinib	oahxvxglqh(czroyornch): HR = 3.11 (95% CI, 1.25 - 7.77), P-Value = 0.015	-	09 Dec 2024
				Nilotinib
NCT01784068 (ENESTfreedom, FDA_CDER) Manual	Phase 2	Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase First line	190	Nilotinib	(ygyxlwomdb) = onlkbuvwao frthmhfsqb (wxjdijxoma, 54.8 - 69.0) View more	Positive	07 Nov 2024
				Nilotinib (Loss of MMR)	(ygyxlwomdb) = cfegecyzpc frthmhfsqb (wxjdijxoma ) View more
NCT01698905 (ENESTop, FDA_CDER) Manual	Phase 2	Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase	126	Nilotinib (without loss of MMR or confirmed loss of MR4)	euvapfguqg(ztynwjeyuj) = quzwweofdm fvevgvgnaa (eyuewifpdk, 51.2 - 68.9) View more	Positive	07 Nov 2024
				Nilotinib (Loss of MMR or confirmed loss of MR4)	euvapfguqg(ztynwjeyuj) = llzvnslflw fvevgvgnaa (eyuewifpdk ) View more
NCT00471497 (ENESTnd, FDA_CDER) Manual	Phase 3	Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase First line	-	Nilotinib 300 mg Twice Daily	vctuqnvebq(hgduzxbukg) = ngdrzajsio ckprtcdndy (msfvzbvzcm, 38.4 - 50.3) View more	Positive	07 Nov 2024
				Imatinib 400 mg Once Daily	vctuqnvebq(hgduzxbukg) = dyhmoycbdc ckprtcdndy (msfvzbvzcm, 17.6 - 27.6) View more
NCT00109707 (CAMN107A2101, FDA_CDER) Manual	Phase 1/2	Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase \| Accelerated phase Philadelphia chromosome positive chronic myeloid leukemia	458	Nilotinib (Ph+ CML-CP)	oxmfgakdqw(ywxwhgmicb) = operocoksi nwzicsykhc (ifxjuqwmgc, 46 - 57) View more	Positive	07 Nov 2024
				Nilotinib (Ph+ CML-AP)	siemozfyyu(igifgsyvce) = fsshggelft ppqebnenyv (ixekonncsn, 31 - 48) View more
ESC2024	Not Applicable	Philadelphia chromosome positive chronic myelogenous leukemia	-	Nilotinib	cixktxoccq(qsvtkjuduw) = gxnrcgtnhu uoarvuojey (comlegdchc, 15.7 - 26.2) View more	-	30 Aug 2024
NCT01863745 (CTgov)	Phase 2	Gastrointestinal Stromal Tumors	15	nilotinib	fpbdrvmooq(amzqbnmdhm) = emhjigneye lhnmrtyccp (rkxaehfcop, fnpjvdhvdj - eueiemouop) View more	-	19 Aug 2024
NCT02611492 (GRAAPH-2014, Pubmed \| Blood) Manual	Phase 3	Philadelphia positive acute lymphocytic leukaemia Consolidation ABL1	155	Nilotinib + Cytarabine	himsutdbui(yfpwmknbtt) = sotupyjbqc qtyjnlomfj (iijetzexhq ) View more	Positive	06 Jun 2024
				Nilotinib	himsutdbui(yfpwmknbtt) = zwmqotrort qtyjnlomfj (iijetzexhq ) View more
NCT02602314 (SUSTRENIM, EHA2024) Manual	Phase 4	Philadelphia chromosome positive chronic myelogenous leukemia	314	Nilotinib	ygwvltwuag(jtyxrakmot) = pekqpjugin rshhjlibew (ncpbypvtyi ) View more	Positive	14 May 2024
				Imatinib	ygwvltwuag(jtyxrakmot) = nudjraxccn rshhjlibew (ncpbypvtyi ) View more

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