Phase I Study with Dose-escalation and Expansion Evaluating the Safety and Efficacy of Oral Arsenic (ATO) in Low-risk Myelodysplastic Syndromes Failing Erythropoiesis Stimulating Agents and Luspatercept (or Ineligible for the Latter)

Phase I study with dose-escalation and expansion evaluating the safety and efficacy of oral Arsenic (ATO) in low-risk Myelodysplastic Syndromes having failed to Erythropoiesis Stimulating Agents and Luspatercept (or ineligible for the latter).

Start Date01 Feb 2025

Sponsor / Collaborator

Groupe Francophone des Myélodysplasies

NCT06088030 / RecruitingPhase 2IIT

Clinical Research on Efficacy and Safety of Arsenic Trioxide Combined With Chemotherapy in p53-mutated Pediatric Cancer Patients：A Prospective，Single-arm, Multi-center Study

This prospective, single-arm, multi-center clinical trial aims to explore and evaluate the efficacy and safety of arsenic trioxide combined with chemotherapy for pediatric cancer with p53 mutation.

Start Date13 Dec 2023

Sponsor / Collaborator

Sun Yat-Sen Memorial Hospital

[+1]

ChiCTR2300073941 / RecruitingPhase 2IIT

A single arm phase II clinical study of ATO combined with PD-1 inhibitor in the treatment of unresectable advanced hepatocellular carcinoma with TP53 gene mutation

Start Date31 Jul 2023

Sponsor / Collaborator-

100 Clinical Results associated with Arsenic Trioxide

100 Translational Medicine associated with Arsenic Trioxide

100 Patents (Medical) associated with Arsenic Trioxide

5,998

Literatures (Medical) associated with Arsenic Trioxide

01 Jan 2025·Journal of Ethnopharmacology

Targeting Nrf2/HO-1 signaling by crocin: Role in attenuation of arsenic trioxide-induced neurotoxicity in mice

Article

Author: Zhang, Shi ; Shi, Jing ; Li, Chen ; Zhao, Yawei ; Wang, Hefei ; Xu, Hao ; Dong, Chunhui ; Chu, Xi ; Hao, Yiwei ; Han, Xue ; Wu, Zhonglin

ETHNOPHARMACOLOGY RELEVANCESaffron is a valued herb, obtained from the stigmas of the C.sativus Linn (Iridaceae). Pharmacopoeias have described it as having a variety of actions, such as stimulant, anti-carcinogen, and anti-depressant. As a folk medicine, crocin has been reported to have anti-cardiotoxicity and anti-hepatotoxicity effects. This paper focuses on crocin, one of the bioactive molecules found in saffron that are known to have therapeutic effects. Crocin has been shown in numerous experimental studies to be beneficial in treating depression, however, there aren't many studies on its neurotoxicity.AIM OF THE STUDYApplications of arsenic trioxide (ATO) in medical settings is limited by its side effects. This study aims to examine crocin's protective effect against ATO-induced neurotoxicity and understand its potential molecular mechanism. Materialandmethods: A neurotoxicity model was created by administering ATO (4 mg/L/d). To counteract this, mice were intraperitoneally injected with crocin (100, 200 mg/kg/d). After 60 days, biochemical, histopathological, transmission electron microscopy, ELISA, and western blotting analyses were then performed.RESULTSOur results indicated that crocin decreased neuronal death and loss caused by ATO, countered oxidative stress damage, and mitigated pro-inflammatory cytokines. Mice treated with crocin also displayed positive signs of brain tissue recovery. Additionally, crocin reduced the protein expressions of NLRP1, apoptosis-associated speck-like protein containing a CARD (ASC), Caspase-1, GRP78, CHOP, and ATF4.CONCLUSIONSThis study attests that crocin can reduce ATO-induced neurotoxicity by safeguarding nerves from oxidative stress, inflammation, and apoptosis, possibly through the activation of the Nrf2/HO-1 signaling pathway.

31 Dec 2024·Pharmaceutical Biology

Shenxiang Suhe pill improves cardiac function through modulating gut microbiota and serum metabolites in rats after acute myocardial infarction

Article

Author: Zhao, Xin ; Wang, Xiaoying ; Sun, Congying ; Wei, Xing ; Xie, Tian ; Zhu, Jiaming ; Zhang, Zhaojian ; Wang, Kaiyue ; Yan, Junyuan ; Chen, Wei ; Zhong, Xinqin

CONTEXTShenxiang Suhe pill (SXSH), a traditional Chinese medicine, is clinically effective against coronary heart disease, but the mechanism of cardiac-protective function is unclear.OBJECTIVEWe investigated the cardiac-protective mechanism of SXSH via modulating gut microbiota and metabolite profiles.MATERIALS AND METHODSSprague-Dawley (SD) male rats were randomly divided into 6 groups (n = 8): Sham, Model, SXSH (Low, 0.063 g/kg; Medium, 0.126 g/kg; High, 0.252 g/kg), and Ato (atorvastatin, 20 mg/kg). Besides the Sham group, rats were modelled with acute myocardial infarction (AMI) by ligating the anterior descending branch of the left coronary artery (LAD). After 3, 7, 14 days' administration, ultrasound, H&E staining, serum enzymic assay, 16S rRNA sequencing were conducted to investigate the SXSH efficacy. Afterwards, five groups of rats: Sham, Model, Model-ABX (AMI with antibiotics-feeding), SXSH (0.126 g/kg), SXSH-ABX were administrated for 14 days to evaluate the gut microbiota-dependent SXSH efficacy, and serum untargeted metabolomics test was performed.RESULTS0.126 g/kg of SXSH intervention for 14 days increased ejection fraction (EF, 78.22%), fractional shortening (FS, 109.07%), and aortic valve flow velocities (AV, 21.62%), reduced lesion area, and decreased serum LDH (8.49%) and CK-MB (10.79%). Meanwhile, SXSH upregulated the abundance of Muribaculaceae (199.71%), Allobaculum (1744.09%), and downregulated Lactobacillus (65.51%). The cardiac-protective effect of SXSH was disrupted by antibiotics administration. SXSH altered serum metabolites levels, such as downregulation of 2-n-tetrahydrothiophenecarboxylic acid (THTC, 1.73%), and lysophosphatidylcholine (lysoPC, 4.61%).DISCUSSION AND CONCLUSIONThe cardiac-protective effect and suggested mechanism of SXSH could provide a theoretical basis for expanding its application in clinic.

31 Dec 2024·Hematology

Prognosis influence of additional chromosome abnormalities in newly diagnosed acute promyelocytic leukemia with t(15;17)(q24;q21)

Article

Author: Wang, Lu ; Tong, Hongyan ; Liu, Lin ; Wang, Jinghan ; Xu, Huan ; Zhao, Shuqi ; Jin, Jie ; Wang, Huanping ; Huang, Jiansong

OBJECTIVESIn patients with acute promyelocytic leukemia (APL), additional chromosomal abnormalities (ACAs) are prognostic indicators. However, the clinical features of ACAs were not systematically reported in Chinese patients. Therefore, we enrolled a large cohort of APLs to demonstrate the clinical characteristics and prognostic value of ACAs.METHODS268 patients with newly diagnosed APL with t(15;17)(q24;q21) were retrospectively enrolled, and their clinical characteristics and the predictive value of ACAs were assessed between patients with the presence and absence of ACAs.RESULTSAPL patients with and without ACAs did not differ significantly in their clinical features or treatment response and clinical outcomes like overall survival (OS) and disease-free survival (DFS). It appeared to be substantially associated with worse OS in APL patients with trisomy 8, which was the most common ACA, although DFS was unaffected. Interestingly, the presence of ACAs or trisomy 8 affected OS and DFS in the subgroup of patients aged ≥60 years; by contrast, ACAs had no effect on OS or DFS in any treatment subgroup (ATRA + ATO/RIF or ATRA + ATO/RIF + CH or ATRA + CH), except for the ATRA + ATO/RIF + CH treatment subgroup, where their impact on DFS was less favorable.CONCLUSIONSOur results suggested that OS and DFS were unaffected by ACAs. Nonetheless, in the subgroup of patients older than 60, the existence of ACAs or trisomy 8 appeared to impact OS and DFS negatively. Individuals with t(15;17) alone had a higher DFS and were more susceptible to ATRA + ATO/RIF + CH than individuals with t(15;17) ACAs.

170

News (Medical) associated with Arsenic Trioxide

13 Nov 2024

·www.prnewswire.com

75+ Key Companies Charting New Frontiers in Multiple Sclerosis Therapeutic Space | DelveInsight

Multiple sclerosis (MS) is a chronic neurological disorder affecting the central nervous system (CNS), particularly the brain, spinal cord, and optic nerves. Continuous research has led to new drug developments, particularly disease-modifying therapies (DMTs) that slow MS progression. Monoclonal antibodies, oral therapies, and infusion treatments are among the most promising options and drives the multiple sclerosis market. LAS VEGAS, Nov. 13, 2024 /PRNewswire/ -- DelveInsight's ' Multiple Sclerosis Pipeline Insight 2024 ' report provides comprehensive global coverage of pipeline multiple sclerosis therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the multiple sclerosis pipeline domain. Key Takeaways from the Multiple Sclerosis Pipeline Report DelveInsight's multiple sclerosis pipeline report depicts a robust space with 75+ active players working to develop 80+ pipeline therapies for multiple sclerosis treatment. Key multiple sclerosis companies such as Mapi Pharma, Novartis, Immunic, Biocad, Apimeds, Genentech (Roche), AB Science, Celltrion, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, BrainStorm Cell Therapeutics, NeuroSense Therapeutics, Denali Therapeutics Inc., Sanofi, MediciNova, Biogen, Tiziana Life Sciences, Antisense Therapeutics, RemeGen, Atara Biotherapeutics, Contineum Therapeutics, Eli Lilly and Company, Axsome Therapeutics, Accure therapeutics, Stem Cell Medicine Ltd., Ever Supreme Bio-Technology Co., Ltd., ImCyse, Lucid Psycheceuticals, Immune Response BioPharma, Guangzhou Lupeng Pharmaceutical, Kyowa Kirin, Nura Bio, Bristol-Myers Squibb, ImStem Biotechnology, Hoffmann-La Roche, LAPIX Therapeutics, Motric Bio, Immunophage Biotech, NervGen, FibroBiologics, Kyverna Therapeutics, GlaxoSmithKline, PolTREG, Cyxone, Biohaven Pharmaceuticals, HuniLife Biotechnology, GlobeStar Therapeutics, Anokion, Medsenic, ZyVersa Therapeutics, Alumis Inc., Axoltis Pharma, Barricade Therapeutics, BioNxt Solutions, Eikonoklastes Therapeutics, 4D Pharma, Gossamer Bio, Pasithea Therapeutics, Synaptogenix, Monte Rosa Therapeutics, Immix Biopharma, f5 Therapeutics, Autobahn Therapeutics, Biosenic, Polpharma Biologics, Find Therapeutics, TeraImmun, AptaTargets, Trethera, Aditxt, SetPoint Medical, Sarepta Therapeutics, Nucleome Therapeutics, Vaccinex Inc., and others are evaluating new multiple sclerosis drugs to improve the treatment landscape. Promising multiple sclerosis pipeline therapies such as GA Depot, Remibrutinib, Tolebrutinib, IMU 838, BCD-132, Apitox, Fenebrutinib, SAR 441344, CT-P53, CNM-Au8, Orelabrutinib, Vafidemstat, Temelimab, Elezanumab, NurOwn (MSC-NTF cells), PrimeC, SAR4433820, Ibudilast, BIIB091, Foralumab, ATL1102, RC18, ATA188, PIPE-307, Pirtobrutinib, LY3541860, Solriamfetol, Act-01, SCM-010, UMSC01, IMCY-0141, Lucid-MS, NeuroVax, LP-168, NB-4746, CC-97540, IMS-001, RO7121932, BMS-986196, LPX-TI641, MTR-601, IPG-11406, IPG-1094, NVG-291, CYMS-101, KYV-101, PIPE-791, GSK 3888130B, PTG-007, T 20K, BHV-8000, HuL001, Amethyst™, ANK 700, Arscimed, A-005, NX210c, Research programme: CAR-TREG, TASIN-1, Cladribine ODF, ET-101, MRx0002, GB7208, PAS-002, Bryostatin-1, MRT-6160, NXC-201, Research Program: Cytokine reduction, ABX-002, Arsenic trioxide, LL-341070, PB018, FTX 101, TI-235, TRE 515, NT-0007, NT-0002, NVG300, and others are under different phases of multiple sclerosis clinical trials. In October 2024, Immunic, Inc. reported favorable results from the non-binding interim futility analysis of its Phase 3 ENSURE trial, which is evaluating its lead candidate, vidofludimus calcium (IMU-838), a nuclear receptor-related 1 (Nurr1) activator, for the treatment of relapsing multiple sclerosis (RMS). In October 2024, Immunic, Inc. announced a positive outcome of the non-binding, interim futility analysis of its Phase III ENSURE program, investigating lead asset, nuclear receptor related 1 (Nurr1) activator, vidofludimus calcium (IMU-838), for the treatment of relapsing multiple sclerosis (RMS). Based on the outcome of the interim futility analysis, an unblinded Independent Data Monitoring Committee (IDMC) has recommended that the trials are not futile and should continue as planned. In September 2024, Roche, secured FDA approval for an injectable form of its blockbuster multiple sclerosis treatment, Ocrevus, reducing patient treatment time and potentially helping the company stay ahead of increasing competition. In September 2024, Sanofi announced the outcomes of Phase III clinical trials of its investigational oral brain-penetrant BTK inhibitor, tolebrutinib, aimed at treating multiple sclerosis (MS). These studies, namely HERCULES, GEMINI 1, and GEMINI 2, have provided mixed results, with the HERCULES trial meeting its primary endpoint while the GEMINI trials did not. The HERCULES study focused on the efficacy and safety of tolebrutinib in individuals with non-relapsing secondary progressive MS (nrSPMS) compared to a placebo. In August 2024, Abata Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ABA-101 for the treatment of patients with progressive multiple sclerosis (MS). The FDA recently cleared ABA-101's Investigational New Drug (IND) application, and initiation of a first-in-human (FIH) Phase I study is imminent. In August 2024, TG Therapeutics announced that the FDA has cleared an investigational new drug (IND) application submitted by TG Therapeutics for azercabtagene zapreleucel (azer-cel), an investigational allogeneic chimeric antigen receptor T-cell (CAR-T) therapy, for a phase 1 clinical trial in patients with progressive multiple sclerosis (MS). In January 2024, Kyverna Therapeutics, Inc. announced it received fast-track designation by the U.S. Food and Drug Administration (FDA) for its autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell product candidate, KYV-101, to be used for the treatment of multiple sclerosis (MS). Request a sample and discover the recent advances in multiple sclerosis treatment drugs @ Multiple Sclerosis Pipeline Report The multiple sclerosis pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage multiple sclerosis drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the multiple sclerosis clinical trial landscape. Multiple Sclerosis Overview Multiple sclerosis is a chronic autoimmune disorder that affects the central nervous system, leading to the deterioration or permanent damage of myelin, the protective sheath surrounding nerve fibers. While the exact cause of multiple sclerosis remains unclear, it is believed to involve a combination of genetic predisposition, environmental factors, and possibly viral infections. Common symptoms of multiple sclerosis include fatigue, difficulty walking, numbness or tingling in the limbs, muscle weakness, and vision problems, which can vary significantly among individuals and may evolve over time. Diagnosis typically involves a combination of neurological examinations, magnetic resonance imaging (MRI) to detect lesions in the brain and spinal cord, and sometimes lumbar puncture to analyze cerebrospinal fluid for inflammatory markers. While there is no cure for multiple sclerosis, treatment options aim to manage symptoms, reduce the frequency and severity of relapses, and slow disease progression. This may include disease-modifying therapies (DMTs), corticosteroids for acute exacerbations, and symptomatic treatments such as physical therapy, pain management, and lifestyle modifications. Early diagnosis and a tailored treatment plan are crucial for improving the quality of life for those living with multiple sclerosis. Find out more about multiple sclerosis treatment drugs @ Drugs for Multiple Sclerosis Treatment A snapshot of the Multiple Sclerosis Pipeline Drugs mentioned in the report: Learn more about the emerging multiple sclerosis pipeline therapies @ Multiple Sclerosis Clinical Trials Multiple Sclerosis Therapeutics Assessment The multiple sclerosis pipeline report proffers an integral view of the multiple sclerosis emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Multiple Sclerosis Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Immunomodulators, MHC class II gene modulators, Agammaglobulinaemia tyrosine kinase inhibitors, Dihydroorotate dehydrogenase inhibitors, Nuclear receptor subfamily 4 group A member 2 agonists, CD20 antigen inhibitors, Energy metabolism stimulants; Immunomodulators, Lysine-specific demethylase 1 inhibitors, Monoamine oxidase B inhibitors, Protein-arginine deiminase inhibitors, Immunostimulants Key Multiple Sclerosis Companies: Mapi Pharma, Novartis, Immunic, Biocad, Apimeds, Genentech (Roche), AB Science, Celltrion, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, BrainStorm Cell Therapeutics, NeuroSense Therapeutics, Denali Therapeutics Inc., Sanofi, MediciNova, Biogen, Tiziana Life Sciences, Antisense Therapeutics, RemeGen, Atara Biotherapeutics, Contineum Therapeutics, Eli Lilly and Company, Axsome Therapeutics, Accure therapeutics, Stem Cell Medicine Ltd., Ever Supreme Bio-Technology Co., Ltd., ImCyse, Lucid Psycheceuticals, Immune Response BioPharma, Guangzhou Lupeng Pharmaceutical, Kyowa Kirin, Nura Bio, Bristol-Myers Squibb, ImStem Biotechnology, Hoffmann-La Roche, LAPIX Therapeutics, Motric Bio, Immunophage Biotech, NervGen, FibroBiologics, Kyverna Therapeutics, GlaxoSmithKline, PolTREG, Cyxone, Biohaven Pharmaceuticals, HuniLife Biotechnology, GlobeStar Therapeutics, Anokion, Medsenic, ZyVersa Therapeutics, Alumis Inc., Axoltis Pharma, Barricade Therapeutics, BioNxt Solutions, Eikonoklastes Therapeutics, 4D Pharma, Gossamer Bio, Pasithea Therapeutics, Synaptogenix, Monte Rosa Therapeutics, Immix Biopharma, f5 Therapeutics, Autobahn Therapeutics, Biosenic, Polpharma Biologics, Find Therapeutics, TeraImmun, AptaTargets, Trethera, Aditxt, SetPoint Medical, Sarepta Therapeutics, Nucleome Therapeutics, Vaccinex Inc., and others are evaluating new multiple sclerosis drugs to improve the treatment landscape. Key Multiple Sclerosis Pipeline Therapies:GA Depot, Remibrutinib, Tolebrutinib, IMU 838, BCD-132, Apitox, Fenebrutinib, SAR 441344, CT-P53, CNM-Au8, Orelabrutinib, Vafidemstat, Temelimab, Elezanumab, NurOwn (MSC-NTF cells), PrimeC, SAR4433820, Ibudilast, BIIB091, Foralumab, ATL1102, RC18, ATA188, PIPE-307, Pirtobrutinib, LY3541860, Solriamfetol, Act-01, SCM-010, UMSC01, IMCY-0141, Lucid-MS, NeuroVax, LP-168, NB-4746, CC-97540, IMS-001, RO7121932, BMS-986196, LPX-TI641, MTR-601, IPG-11406, IPG-1094, NVG-291, CYMS-101, KYV-101, PIPE-791, GSK 3888130B, PTG-007, T 20K, BHV-8000, HuL001, Amethyst™, ANK 700, Arscimed, A-005, NX210c, Research programme: CAR-TREG, TASIN-1, Cladribine ODF, ET-101, MRx0002, GB7208, PAS-002, Bryostatin-1, MRT-6160, NXC-201, Research Program: Cytokine reduction, ABX-002, Arsenic trioxide, LL-341070, PB018, FTX 101, TI-235, TRE 515, NT-0007, NT-0002, NVG300, and others are under different phases of multiple sclerosis clinical trials. Dive deep into rich insights for new drugs for multiple sclerosis treatment, visit @ Multiple Sclerosis Drugs Table of Contents For further information on the multiple sclerosis pipeline therapeutics, reach out @ Multiple Sclerosis Treatment Drugs Related Reports Multiple Sclerosis Market Multiple Sclerosis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key multiple sclerosis companies including Immune Response BioPharma, Inc., Clene Nanomedicine, Genzyme, ImStem Biotechnology, Rho, Inc., Bristol-Myers Squibb, TG Therapeutics, Inc., Hoffmann-La Roche, Atara Biotherapeutics, Immunic AG, Celgene, Anokion SA, Sanofi, Actelion, Biogen, Supernus Pharmaceuticals, Inc., GeNeuro SA, HuniLife Biotechnology, Inc., Emerald Health Pharmaceuticals, RemeGen Co., Ltd., Antisense Therapeutics, Biocad, AB Science, Genentech, Novartis, Pipeline Therapeutics, ASLAN Pharmaceuticals, GlaxoSmithKline, Lucid Psycheceuticals, Voronoi, Hanmi Pharmaceutical, Mapi Pharma, f5 Therapeutics, Autobahn Therapeutics, AstraZeneca, ZyVersa Therapeutics, RedHill Biopharma, Gossamer Bio, Sarepta Therapeutics, BrainStorm Cell Limited, CytoDyn, Pear Therapeutics, Solstice Neurosciences, ProJenX, among others. Multiple Sclerosis Epidemiology Forecast Multiple Sclerosis Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted multiple sclerosis epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Relapsing Multiple Sclerosis Pipeline Relapsing Multiple Sclerosis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key relapsing multiple sclerosis companies, including ASLAN Pharmaceuticals, GlaxoSmithKline, Lucid Psycheceuticals, Voronoi, Hanmi Pharmaceutical, among others. Relapsing-Remitting Multiple Sclerosis Pipeline Relapsing-Remitting Multiple Sclerosis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key RRMS companies, including TG Therapeutics, Novartis, Sanofi, Cinnagen, Polpharma Biologics, Immunic, Mapi Pharma, Biocad, Apimeds, Genentech (Roche), Merck, Immune Response BioPharma, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, Emerald Health Pharmaceuticals, GlaxoSmithKline, RedHill Biopharma, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Clinical ResultFast TrackCell TherapyImmunotherapy

13 Nov 2024

·endpts.com

Syros' shares plummet as blood cancer drug fails again

Syros Pharmaceuticals’ share price nosedived after its last drug candidate flunked a trial in hematological malignancies for the second time in recent months. In a 190-participant Phase 3 trial, the biotech’s tamibarotene plus azacitidine led to a 23.8% complete response rate in people with newly diagnosed higher-risk myelodysplastic syndromes and RARA gene overexpression versus 18.8% for azacitidine plus placebo. The result was not statistically significant with a p-value of 0.2084, according to a Tuesday release . Syros’ share price $SYRS was down 80% premarket Wednesday. The trial failure triggers a default event under the terms of Syros’ secured loan facility with Oxford Finance. The biotech plans to stop the trial, analyze the data and “evaluate the next steps,” CEO Conley Chee said in the release. The study flop comes just three months after an August disappointment for a tamibarotene triplet regimen in a Phase 2 study of newly diagnosed, unfit patients with acute myeloid leukemia and RARA gene overexpression. Tamibarotene is a retinoid acid receptor alpha agonist. “Tamibarotene’s demise brings to an end Syros’ last clinical program,” TD Cowen analysts said in a Wednesday note, adding the company is likely to end all development of the drug. The biotech previously stopped work on two other clinical candidates, SY-2101 and SY-5609, although it said on an earnings call last month it is looking for licensing opportunities for the latter. The company reported $58.3 million in cash and equivalents as of the end of September.

Phase 3Phase 2Clinical Trial Failure

13 Nov 2024

·firstwordpharma.com

Another study failure puts brakes on Syros’ tamibarotene in blood cancers

After flunking a mid-stage study in front-line acute myeloid leukaemia three months back, Syros Pharmaceuticals’ oral retinoic acid receptor alpha (RARα) agonist, tamibarotene, has hit yet another and a potentially terminal roadblock. This time, the investigational therapy in combination with azacitidine failed to meet the primary endpoint of a late-stage study involving patients with newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS) harbouring RARA gene overexpression – triggering a loan default for the company with Oxford Finance LLC.“We are deeply disappointed by this outcome, particularly for the HR-MDS patients who are seeking a new treatment option for this challenging disease,” said CEO Conley Chee, adding that while Syros will terminate the trial, it will continue to review exisiting data and chart out a further course of action.The Phase III SELECT-MDS-1 study assessed the efficacy and safety of tamibarotene plus azacitidine versus placebo plus azacitidine in RARA-overexpressed patients with HR-MDS, the main goal being the complete response (CR) rate in the first 190 participants. Results showed the tamibarotene plus azacitidine arm demonstrated a CR rate of 23.8% in the initial 190 patients, compared with the placebo plus azacitidine arm’s 18.8%, with no statistically significant difference between the groups. Meanwhile, the safety analysis of all enrolled 245 patients showed the tamibarotene combination was generally well-tolerated, with an adverse event profile comparable to previous studies.Facing financial troubles in the wake of failed deals with Incyte and Pfizer in 2023, the company cut 35% of its workforce last October and discontinued most clinical programmes, including SY-2101 for acute promyelocytic leukaemia, to focus on its lead asset, tamibarotene.

Phase 3Clinical ResultFinancial Statement

100 Deals associated with Arsenic Trioxide

External Link

KEGG	Wiki	ATC	Drug Bank
D02106	Arsenic Trioxide	L01XX27	DB01169

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Acute Promyelocytic Leukemia	US	Cephalon, Inc.	25 Sep 2000

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Acute Myeloid Leukemia	Phase 2	CA	Cephalon, Inc.	01 Oct 2007
Acute Myeloid Leukemia	Phase 2	US	Cephalon, Inc.	01 Oct 2007
Plasma cell myeloma refractory	Phase 2	US	CTI BioPharma Corp.	01 Feb 2001
Relapse multiple myeloma	Phase 2	US	CTI BioPharma Corp.	01 Feb 2001
Multiple Myeloma	Phase 2	US	CTI BioPharma Corp.	01 Jan 2001
Multiple Myeloma	Phase 2	US	CTI BioPharma Corp.	01 Jan 2001
Plasmacytoma	Phase 2	US	CTI BioPharma Corp.	01 Jan 2001
Chronic graft-versus-host disease	Phase 2	-	BioSenic SA	-
Scleroderma, Systemic	Phase 2	-	Bone Therapeutics SA	-
Systemic Lupus Erythematosus	Phase 2	-	Bone Therapeutics SA	-

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASH2024	Not Applicable	Acute Promyelocytic Leukemia	-	Standard-dose ATO (10mg IV daily for 28 days)	xdijalmlci(sgglmzktqn) = Patients receiving ATO treatment have a high rate of adverse events in both groups, reducing the total ATO dosage at induction treatment may reduce the adverse event rate and help patients achieve the same long-term survival. xlynkptvzg (qluzfoslbg )	-	09 Dec 2024
				Low-dose ATO (10mg IV daily for less than 28 days)
EHA2024	Not Applicable	Acute Promyelocytic Leukemia First line	-	ATO-based chemotherapy-free regimens	nttxsnmzkm(bbeeintdku) = ewnxeyyxfw ceikvdldlb (yoqdqnuasc, 0.06‒0.47)	Negative	14 May 2024
				ATO-containing chemotherapy-based regimens	nttxsnmzkm(bbeeintdku) = nrvtxfzxvo ceikvdldlb (yoqdqnuasc, 0.70‒1.43)
ASH2023	Not Applicable	Acute Promyelocytic Leukemia	-	Oral-Arsenic Trioxide	(dcphffasbx) = hwgpjtyrak pkqbfbnrdj (bwoykcllkx ) View more	-	09 Dec 2023
				All-Trans Retinoic Acid	(dcphffasbx) = bamklmfwsz pkqbfbnrdj (bwoykcllkx ) View more
ASH2023	Not Applicable	Acute Promyelocytic Leukemia	135	ATRA-ATO	(bynzpggofh) = jxjxtgptqq zbtmjnvxym (nryopiqlla ) View more	Positive	09 Dec 2023
				ATRA+chemotherapy	(bynzpggofh) = koqwsoprhm zbtmjnvxym (nryopiqlla ) View more
AML-103 (SOHO2023)	Not Applicable	Acute Promyelocytic Leukemia Consolidation \| Maintenance	12	ATRA+ATO	hikvlrgmjs(anheickyxk) = yagmttpncj rlvxzekjaa (vbxozlaxuq )	Positive	01 Sep 2023
NCT00045565 (ASCO2023)	Phase 1	Glioblastoma	30	Arsenic Trioxide once weekly	qacdkwabri(norfykjcmp) = Major dose limiting toxicities were increased aspartate and alanine aminotransferases and one case of hyperkalemia jmdbkcvqww (rsevhfxkzo )	-	31 May 2023
				Arsenic Trioxide twice weekly
NCT02966301 (Pubmed) Manual	Phase 2	Chronic graft-versus-host disease First line	21	Corticosteroids+Arsenic Trioxide	(qzhrzrcvfm) = bqhqtuszrk eyogckdttt (mytnlhqzcs, 50.9 - 91.3) View more	Positive	10 Jul 2022
NCT02966301 (GvHD-ATO, CTgov)	Phase 2	Chronic graft-versus-host disease	21	Arsenic Trioxide Injectable Solution	uvdrmsyevl(ckniwxmnjg) = npylycbdbq vpufwkhtrx (aygdrpyhjz, yamxzwkfqq - hxedulcfpy) View more	-	09 May 2022
NCT01404949 (CTgov)	Phase 2	Acute Promyelocytic Leukemia	17	Tretinoin+Arsenic Trioxide	dkmczcsfgz(zdgrxlvdso) = thxpprkurz dkbkgghtcq (hamntgmjdu, xbskleqwop - coghperxid) View more	-	30 Nov 2021
NCT02339740 (AAML1331, Pubmed \| JAMA Oncol)	Phase 3	Acute Promyelocytic Leukemia	154	ATRA and arsenic trioxide-based regimen	(jecxhrbnqg) = hlmrbbvtkb ezamfrecpd (chdyungqbp )	Positive	11 Nov 2021
				Chemotherapy	(jecxhrbnqg) = jbhznesrjp ezamfrecpd (chdyungqbp )

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