CDK1 inhibitors(Cyclin-dependent kinase 1 inhibitors), Class I PI3K inhibitors(PI3-kinase class I inhibitors), PLK1 inhibitors(Polo like kinase 1 inhibitors)

Therapeutic Areas

NeoplasmsSkin and Musculoskeletal DiseasesImmune System Diseases+ [3]

Active Indication

MelanomaEpidermolysis Bullosa DystrophicaSquamous Cell Carcinoma+ [2]

Inactive Indication

Acute Lymphoblastic LeukemiaAcute Myeloid LeukemiaAdvanced cancer+ [27]

Originator Organization

Traws Pharma, Inc.

Active Organization

Traws Pharma, Inc.

Bristol Myers Squibb Co.

Salzburger Landeskliniken Betriebsgesellschaft mbH

Inactive Organization

SymBio Pharmaceuticals Ltd.Alidac Pharmaceuticals Ltd.Hangzhou Hansi Biological Pharmaceutical Co., Ltd.

Drug Highest PhasePhase 2

First Approval Date-

RegulationOrphan Drug (United States)

Structure/Sequence

Molecular FormulaC21H25NNaO8S

InChIKeyIIRFVCCKEUSNLK-USRGLUTNSA-N

CAS Registry592542-60-4

Clinical Trials associated with Rigosertib Sodium

NCT05764395

/ SuspendedPhase 2IIT

A Phase II Study to Evaluate the Safety and Efficacy of Rigosertib (ON 01910) Plus Pembrolizumab in Patients With Metastatic Melanoma Refractory to Immune Checkpoint Blockade

This phase II clinical trial tests how well rigosertib plus pembrolizumab workings in treating patients with melanoma which cannot be removed by surgery (unresectable) or that has spread from where it first started (primary site) to other places in the body (metastatic), and that has not responded to previous treatment with PD-1 or PD-L1 inhibitors (refractory). Rigosertib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and may change the immune system to make immunotherapy more effective. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving rigosertib in combination with pembrolizumab may be more effective in treating patients with unresectable metastatic melanoma that has not responded to previous treatment with PD-1 or PD-L1 inhibitors than giving either drug alone.

Start Date09 May 2023

Sponsor / Collaborator

The Vanderbilt-Ingram Cancer Center

NCT04177498

/ RecruitingEarly Phase 1IIT

A Pilot, Open Study to Assess Efficacy and Safety of Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated Locally Advanced/Metastatic Squamous Cell Carcinoma

This pilot trial studies how rigsertib sodium works in treating patients with Recessive Dystrophic Epidermolysis bullosa (RDEB) with locally advanced Squamous Cell Carcinoma (SCC). Rigosertib may selectively target Epidermolysis bullosa (EB) cancer cells while leaving normal EB cells unaffected.

Start Date24 Aug 2021

Sponsor / Collaborator

Thomas Jefferson University

[+1]

NCT03786237

/ RecruitingPhase 1/2

A Phase II, Open Study to Assess Efficacy and Safety of Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated Locally Advanced/Metastatic Squamous Cell Carcinoma

Epidermolysis bullosa (EB) is a heritable skin disease characterized by marked fragility of epithelialized tissue with blistering in skin and mucous membranes following the slightest mechanical trauma. Eighty percent of all patients suffering from recessive dystrophic EB (RDEB), a subtype originating from mutations in the COL7A1 gene, develop squamous cell carcinoma (SCC). In RDEB patients SCC presents early (most patients are in their 20s or 30s) and shows a highly aggressive metastatic course which often leads to premature death at this young age.
In light of scarce data on the efficacy and safety of systemic treatment regimens for advanced SCC, the investigators propose to perform a small, "first in EB " trial of an experimental drug called rigosertib for the treatment of EB cancer. The trial will be conducted in two study centres, in London and Salzburg, and will last approximately 2.5 years with each patient recruited being in the study for 1 year. The drug is a polo-like kinase inhibitor interfering with different molecular pathways that are essential for cancer cell growth. Rigosertib was developed by Onconova Therapeutics and is currently tested in several clinical trials for a number of other cancers including myelodysplastic syndrome (a cancer of the blood). The investigators have identified that rigosertib most selectively kills EB cancer cells in vitro while leaving normal EB skin cells unaffected. This project will evaluate whether rigosertib is capable of inducing an anti-cancer response in EB patients and whether the drug is well-tolerated. Mechanisms of molecular targeting of squamous cancer cells by rigosertib will further be investigated in EB patients, also aiming at the identification of biomarkers that may allow the predictive identification of best responders.

Start Date12 Apr 2021

Sponsor / Collaborator

Salzburger Landeskliniken Betriebsgesellschaft mbH

100 Clinical Results associated with Rigosertib Sodium

100 Translational Medicine associated with Rigosertib Sodium

100 Patents (Medical) associated with Rigosertib Sodium

213

Literatures (Medical) associated with Rigosertib Sodium

01 Mar 2025·DRUG RESISTANCE UPDATES

Osmotic stress influences microtubule drug response via WNK1 kinase signaling

Article

Author: Rosa-Rosa, Juan Manuel ; de Cárcer, Guillermo ; Sanz-Gómez, Natalia ; Cambón, Aitana ; Ballesteros-Sánchez, Sandra ; Sánchez-Prieto, Ricardo ; Escribano-Cebrián, María ; Monfort-Vengut, Ana ; Sotillo, Rocío ; Martínez-López, Joaquín ; Ortigosa, Beatriz ; Ramos, Maria ; Lorenzo, Ángela Montes-San

Ion homeostasis is critical for numerous cellular processes, and disturbances in ionic balance underlie diverse pathological conditions, including cancer progression. Targeting ion homeostasis is even considered as a strategy to treat cancer. However, very little is known about how ion homeostasis may influence anticancer drug response. In a genome-wide CRISPR-Cas9 resistance drug screen, we identified and validated the master osmostress regulator WNK1 kinase as a modulator of the response to the mitotic inhibitor rigosertib. Osmotic stress and WNK1 inactivation lead to an altered response not only to rigosertib treatment but also to other microtubule-related drugs, minimizing the prototypical mitotic arrest produced by these compounds. This effect is due to an alteration in microtubule stability and polymerization dynamics, likely maintained by fluctuations in intracellular molecular crowding upon WNK1 inactivation. This promotes resistance to microtubule depolymerizing compounds, and increased sensitivity to microtubule stabilizing drugs. In summary, our data proposes WNK1 osmoregulation activity as an important modulator for microtubule-associated chemotherapy response.

01 Feb 2025·LIFE SCIENCES

Retraction notice to “Rigosertib potently protects against colitis-associated intestinal fibrosis and inflammation by regulating PI3K/AKT and NF-κB signaling pathways” [Life Sci. 249 (2020) 117470]

Article

Author: Jafari, Mohieddin ; Hassanian, Seyed Mahdi ; Ahmadian, Mohammad Reza ; Barneh, Farnaz ; Ryzhikov, Mikhail ; Ferns, Gordon A ; Khazaei, Majid ; Avan, Amir ; Parizadeha, Mohammad Reza ; Rahmani, Farzad ; Giovannetti, Elisa ; Asgharzadeh, Fereshteh

01 Nov 2024·BIOORGANIC & MEDICINAL CHEMISTRY

Discovery of dual-targeted molecules based on Olaparib and Rigosertib for triple-negative breast cancer with wild-type BRCA

Article

Author: Huang, Xiaochao ; Huang, Rizhen ; Hu, Weiwei ; Liu, Zhikun ; Cao, Guoxiu ; Mao, Shining ; Yu, Chunhao ; Yang, Yong ; Dai, Lumei

PARP inhibitors (PARPis) demonstrate significant potential efficacy in the clinical treatment of BRCA-mutated triple-negative breast cancer (TNBC). However, a majority of patients with TNBC do not possess BRCA mutations, and therefore cannot benefit from PARPis. Previous studies on multi-targeted molecules derived from PARPis or disruptors of RAF-RAF pathway have offered an alternative approach to develop novel anti-TNBC agents. Hence, to broaden the application of PARP inhibitors for TNBC patients with wild-type BRCA, a series of dual-targeted molecules were constructed via integrating the key pharmacophores of Olaparib (Ola) and Rigosertib into a single entity. Subsequent studies exhibited that the resulting compounds 13a-14c obtained potential anti-proliferative activity against BRCA-defected or wild-type TNBC cells. Among them, an optimal compound 13b showed good inhibitory activity toward PARP-1, displayed approximately 34-fold higher inhibitory activity than that of Ola in MDA-MB-231 cells, and exerted multi-functional mechanisms to induce apoptosis. Moreover, 13b displayed superior antitumor efficacy (TGI, 61.3 %) than the single administration of Ola (TGI, 38.5 %), 11b (TGI, 51.8 %) or even their combined administration (TGI, 56.7 %), but did not show significant systematic toxicity. These findings suggest that 13b may serve as a potential candidate for BRCA wild-type TNBC.

News (Medical) associated with Rigosertib Sodium

06 Mar 2025

·www.prnewswire.com

TEPMETKO Continues to Strengthens its Position as a Leading METex14 Skipping NSCLC Therapy | DelveInsight

With increasing biomarker-driven cancer treatments, TEPMETKO benefits from growing adoption in precision oncology. Competition from other MET inhibitors like TABRECTA (capmatinib) exists, but TEPMETKO's once-daily dosing and efficacy profile offer differentiation. LAS VEGAS, March 6, 2025 /PRNewswire/ -- DelveInsight's " TEPMETKO Market Size, Forecast, and Market Insight Report" highlights the details around TEPMETKO, a kinase inhibitor indicated for the treatment of adult patients with metastatic NSCLC harboring MET exon 14 skipping alterations. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of TEPMETKO. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. EMD Serono's TEPMETKO (tepotinib) Overview TEPMETKO is a kinase inhibitor prescribed for adult patients with metastatic non-small cell lung cancer (NSCLC) carrying MET exon 14 skipping alterations. The active component is Tepotinib (as hydrochloride monohydrate), administered orally. Patients should be cautioned about the heightened risk of severe or fatal interstitial lung disease/pneumonitis, liver toxicity, and potential embryo-fetal toxicity, necessitating effective contraception during and shortly after treatment. Tepotinib specifically targets MET, including variants with exon 14 skipping mutations. It blocks HGF-dependent and independent MET phosphorylation, disrupting MET-driven signaling pathways. Additionally, at clinically relevant concentrations, Tepotinib inhibits melatonin 2 and imidazoline 1 receptors. In vitro studies show that it suppresses tumor cell proliferation, anchorage-independent growth, and migration of MET-dependent cancer cells. In mouse models with MET-driven tumors, including those with MET exon 14 skipping alterations, Tepotinib reduced tumor growth, sustained MET phosphorylation inhibition, and, in one case, decreased metastasis formation. The recommended dose of TEPMETKO is 450 mg taken orally once daily until disease progression or intolerable toxicity occurs. Patients should take it at the same time each day, swallowing tablets whole without chewing, crushing, or splitting them. If a dose is missed and less than eight hours remain until the next scheduled dose, patients should skip it. In cases of vomiting after taking TEPMETKO, the next dose should be taken at the usual time. Learn more about TEPMETKO projected market size for NSCLC @ TEPMETKO Market Potential Non-small cell lung cancer (NSCLC) is the most common type of lung cancer, accounting for 81% of all diagnosed cases. Early detection greatly improves outcomes, but diagnosing NSCLC and other lung cancers is challenging because their symptoms are often mistaken for common illnesses or the long-term effects of smoking. Consequently, 80% of NSCLC cases are already at advanced stages by the time they are identified, making treatment more difficult. Around 80% of EGFR mutations in NSCLC involve either exon 19 deletions or the exon 21 L858R substitution, both of which are classified as sensitizing mutations. Until the last decade, chemotherapy was the primary treatment for advanced and metastatic lung cancer. However, this changed in 2015 with the approval of the first immune checkpoint inhibitor (ICI), KEYTRUDA (pembrolizumab), as a second-line therapy for advanced cases. This was followed by TECENTRIQ (atezolizumab) in 2016. Both therapies were later approved for first-line treatment, broadening their use to a larger patient population. In 2020, the combination of OPDIVO (nivolumab) and ipilimumab also received approval as a first-line treatment for metastatic NSCLC. According to DelveInsight, the NSCLC market size across the 7MM is projected to grow from USD 30 billion in 2024, with a substantial CAGR through 2034. This growth is largely driven by the introduction of emerging therapies during the forecast period (2025–2034). Discover more about the NSCLC market in detail @ Non-small Cell Lung Cancer Market Report Emerging Competitors of TEPMETKO The NSCLC pipeline is very robust with the promising therapies such as Telisotuzumab vedotin (AbbVie), Patritumab deruxtecan (Daiichi Sankyo/AstraZeneca), Datopotamab deruxtecan (Daiichi Sankyo/AstraZeneca), Eftilagimod alpha (Immutep), BNT311/GEN1046 (acasunlimab) (Genmab), V940 (mRNA-4157) + Pembrolizumab (Moderna Therapeutics/Merck), Plinabulin + Docetaxel (BeyondSpring), Olomorasib (LY3537982) (Eli Lilly and Company), Zipalertinib (Cullinan Oncology/Taiho Pharma), Ceralasertib (AZD6738) (AstraZeneca), TEDOPI (EP-2101; IDM 2101; OSE-2101) (OSE Immuno-therapeutics), Sigvotatug vedotin (PF08046047) (Pfizer), ANKTIVA (N-803) (ImmunityBio), Aumolertinib/Almonertinib/HS-10206 (Jiangsu Hansoh Pharmaceutical), Niraparib (GSK), Savolitinib (AstraZeneca/Hutchison MediPharma), TRODELVY (Gilead Sciences), Pyrotinib (Jiangsu HengRui Medicine), Ociperlimab (BGB-A1217) (BieGene), Volrustomig (AstraZeneca), Gotistobart (BNT316/ONC-392) (OncoC4/BioNTech), Ivonescimab (AK112/SMT112) (Akeso Biopharma/Summit Therapeutics), ZYNYZ (retifanlimab/INCMGA00012) (Incyte/Macrogenics), Divarasib (GDC-6036) (Roche/Genentech), Tiragolumab (RG6058) (Roche), Sacituzumab Tirumotecan (Merck and Kelun-Biotech), JEMPERLI (dostarlimab/TSR-042) (GSK and AnaptysBio), Zongertinib (BI-1810631) (Boehringer Ingelheim), BAY 2927088 (Bayer), Serplulimab (HLX10) (Shanghai Henlius Biotech), Rilvegostomig (AZD2936) (AstraZeneca), MK-1084 (Merck, Taiho Pharmaceutical, and Astex), Domvanalimab (Arcus Biosciences and Gilead Sciences), OPDUALAG (nivolumab and relatlimab) (Bristol-Myers Squibb), Belrestotug + JEMPERLI (iTeos Therapeutics and GSK), Firmonertinib (ArriVent BioPharma), Sunvozertinib (DZD9008) (Dizal Pharmaceutical), Cobolimab (GSK), Livmoniplimab (AbbVie), Fianlimab (REGN3767) (Regeneron Pharmaceuticals), BNT327/PM8002 (Biotheus/BioNTech), HS-20117 (Hansoh BioMedical), IO102-IO103 + Pembrolizumab (IO Biotech), Naptumomab estafenatox (NeoTX Therapeutics/Active Biotech), FF-10832 (FUJIFILM Corporation), BNT116 (BioNTechSE/Regeneron Pharmaceuticals), CAN-2409 (Candel Therapeutics), Mecbotamab Vedotin (BA3011/CAB-AXL-ADC) (BioAtla), Bemcentinib (BGB 324/BGB-3234/R-428) (BerGenBio/Rigel Pharmaceuticals), DOVBLERON (taletrectinib/AB-106/IBI-344) (Nuvation Bio/Innovent Biologics/Daiichi Sankyo/Nippon Kayaku), Lifileucel (Iovance Biotherapeutics), IBI363 (Innovent Biologics), Sotevtamab (AB-16B5) (Alethia Biotherapeutics), Avutometinib (VS6766) (Verastem Oncology), Vebreltinib (APL-101) (Apollomics), LP-300 (Lantern Pharma), JNJ-90301900 (Johnson & Johnson Innovative Medicine), AMG 193 (Amgen), Luveltamab tazevibulin (Sutro Biopharma), PRT3789 (Prelude Therapeutics), Disitamab vedotin (Seagen), PADCEV (enfortumab vedotin) (Astellas Pharma), RP1 (Replimune), TIVDAK (tisotumab vedotin) (Seagen), Zanidatamab (Jazz Pharmaceuticals), Utidelone injectable (UTD1) (Beijing Biostar Pharmaceuticals), REGN5093-M114 (Regeneron Pharmaceuticals), SLC-391 (SignalChem Lifesciences), fulzerasib (GenFleet), Davutamig (REGN5093) (Regeneron Pharmaceuticals), TAS3351 (Taiho Oncology), H002 (RedCloud Bio), JIN-A02 (J INTS BIO), FWD1509 (Forward Pharma), Sabestomig (AZD7789) (AstraZeneca), Sasanlimab (Pfizer), Selvigaltin (GB1211) (Galecto Biotech), Vepafestinib Helsinn (Healthcare/Taiho Pharmaceutical), EP0031 (A400/ KL590586) (Ellipses Pharma/Kelun-Biotech), Pamvatamig (MCLA-129) (Merus), Zidesamtinib (NVL-520) (Nuvalent), NVL-655 (Nuvalent), RMC-4630 (Revolution Medicines), REQORSA (quaratusugene ozeplasmid) (Genprex), PDC*lung01 (PDC*line Pharma), Evalstotug (BA3071) (BioAtla and BeiGene), PT-112 (Promontory Therapeutics), MRT-2359 (Monte Rosa Therapeutics), GAIA-102 (GAIA BioMedicine), Rigosertib (Traws Pharma), HMBD-001 (Hummingbird Bioscience), PLB1004 (Avistone Biotechnology), ANS03 (Avistone Biotechnology), MYTX-011 (Mythic Therapeutics), A166 (Sichuan Kelun-Biotech Bio-pharmaceutical), Atamparib (BN-2397) (Ribon Therapeutics), DELTACEL (KB-GDT-01) (Kiromic BioPharma), Carotuximab (ENV-105) (Kairos Pharma), YL202 (MediLink Therapeutics), and others. To know more about the number of competing drugs in development, visit @ TEPMETKO Market Positioning Compared to Other Drugs Key Milestones of TEPMETKO In February 2024, the FDA granted traditional approval to TEPMETKO for adult patients with metastatic NSCLC harboring MET exon 14 skipping alterations. In February 2022, the EC approved TEPMETKO as monotherapy for the treatment of adult patients with advanced NSCLC harboring alterations leading to METex14 skipping who require systemic therapy following prior treatment with immunotherapy and/or platinum-based chemotherapy. In February 2021, Merck announced that the US FDA has approved TEPMETKO following priority review for the treatment of adult patients with metastatic NSCLC harboring MET exon 14 skipping alterations. This indication was approved under accelerated approval based on ORR and DOR. In October 2020, TEPMETKO received ODD for treating NSCLC with MET genomic tumor aberrations In March 2020, EMD Serono (the biopharmaceutical business of Merck KGaA), announced that the MHLW approved TEPMETKO for the treatment of patients with unresectable, advanced, or recurrent NSCLC with METex14 skipping alterations. In September 2019, the US FDA granted BTD for tepotinib in patients with metastatic NSCLC harboring METex14 skipping alterations who progressed following platinum-based cancer therapy Discover how TEPMETKO is shaping the NSCLC treatment landscape @ TEPMETKO NSCLC TEPMETKO Market Dynamics TEPMETKO competes in a growing but highly specialized market where MET-targeted therapies are gaining traction due to their efficacy in addressing MET-altered tumors. The market for MET inhibitors is expanding as precision oncology advances and molecular diagnostics become more widely available, enabling better identification of patients who would benefit from targeted treatments like TEPMETKO. A key factor shaping TEPMETKO's market dynamics is competition. It directly competes with Novartis' TABRECTA (capmatinib), another MET inhibitor approved for the same indication. While TEPMETKO offers the advantage of once-daily dosing compared to TABRECTA's twice-daily regimen, both drugs have similar efficacy profiles, making physician and patient preference an important differentiator. Additionally, broader competition from next-generation MET inhibitors and combination therapies in clinical trials could impact TEPMETKO's long-term market position. Regulatory approvals and market access play a crucial role in TEPMETKO's adoption. The drug has been approved in multiple regions, including the U.S., Europe, and Japan, but reimbursement and pricing strategies vary by market. Payer policies, along with the cost-benefit analysis of MET inhibitors compared to other targeted therapies, influence prescription trends. Furthermore, real-world evidence and post-marketing studies will be critical in demonstrating TEPMETKO's sustained effectiveness and safety, which could support expanded indications and increased market penetration. Looking ahead, the MET inhibitor market is expected to grow with advances in biomarker-driven therapy, further refining patient selection. TEPMETKO's success will depend on continued clinical development, potential label expansions, and strategic partnerships to strengthen its competitive edge. As new entrants emerge, Merck KGaA's ability to differentiate TEPMETKO through combination strategies or enhanced formulations may determine its long-term sustainability in this evolving landscape. Dive deeper to get more insight into TEPMETKO's strengths & weaknesses relative to competitors @ TEPMETKO Market Drug Report Table of Contents Related Reports Non-small Cell Lung Cancer Market Non-small Cell Lung Cancer Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key NSCLC companies including Daiichi Sankyo, AstraZeneca, Gilead Sciences, BieGene, AbbVie, Roche, Merck, Novartis, Pfizer, Takeda Pharmaceuticals, Eli Lilly, BerGenBio, GlaxoSmithKline, Duality biologics, among others. Non-small Cell Lung Cancer Pipeline Non-small Cell Lung Cancer Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key non-small cell lung cancer companies, including BridgeBio Pharma, Daiichi Sankyo, EMD Serono, Merck, BridgeBio Pharma, Abbvie, Pfizer, Eli Lilly and Company BioNTech SE, Shenzhen TargetRx, Taiho Pharmaceutical, Chong Kun Dang, Bristol Myers Squibb, Innovent Biologics, Xuanzhu Biopharmaceutical, Bayer, GeneScience Pharmaceuticals, InventisBio, Apollomics, Imugene, Ono Pharmaceutical, Pierre Fabre, Jiangsu Hengrui Medicine Co., Bristol-Myers Squibb, Surface Oncology, Inhibrx, Sinocelltech, Mirati Therapeutics, REVOLUTION Medicines, Yong Shun Technology Development, Iovance Biotherapeutics, Galecto Biotech, among others. C-MET Metastatic Non-small Cell Lung Cancer Market C-MET Metastatic Non-small Cell Lung Cancer Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key C-MET mNSCLC companies, including Novartis, Merck, EMD Serono, AbbVie, Regeneron Pharmaceuticals, Mythic Therapeutics, Apollomics, Johnson & Johnson Innovation, Haihe Biopharma, among others. C-MET Non-small Cell Lung Cancer Pipeline C-MET Non-small Cell Lung Cancer Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key C-MET non-small cell lung cancer companies, including AbbVie, Janssen Research & Development, Beijing Pearl Biotechnology Limited Liability Company, Novartis, 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

Drug ApprovalImmunotherapyPriority Review

14 Nov 2024

·www.globenewswire.com

Traws Pharma Provides Business Update and Reports Q3 2024 Financial Results

COVID: Phase 1 pharmacokinetic profile supports the potential for ratutrelvir to be dosed as a once-a-day, single drug, 10-day treatment course, without ritonavir, and with a lower likelihood of clinical rebound and good overall tolerabilityFlu: Phase 1 pharmacokinetic profile supports the potential for tivoxavir marboxil to be a one-time treatment for flu, including pandemic and avian flu, with good overall tolerability NEWTOWN, Pa., Nov. 14, 2024 (GLOBE NEWSWIRE) -- Traws Pharma, Inc. (“Traws” or “Traws Pharma”), a clinical stage biopharmaceutical company developing oral small molecules for respiratory viral diseases, today outlined recent business updates and reported unaudited financial results for the third quarter ended September 30, 2024. “Traws is making excellent progress with its clinical pipeline. Phase 1 data for each of the antiviral programs for COVID and influenza are particularly exciting,” said Werner Cautreels, PhD, Chief Executive Officer of Traws Pharma. “Based on our recent Phase 1 pharmacokinetic results, we believe that ratutrelvir has the potential to be used as a monotherapy to treat COVID, without ritonavir, with a lower likelihood of clinical rebound. In addition, recent Phase 1 pharmacokinetic data suggest that tivoxavir marboxil could be a single-dose treatment for influenza, including potential pandemic settings such as avian flu. These distinct characteristics support the potential for each agent to be a differentiated, best-in-class treatment and progress to Phase 2 studies expected to commence in 2025.“ Upcoming Milestones Tivoxavir marboxil: Flu: an oral influenza cap-dependent endonuclease, highly conserved across flu strains, including avian flu. Intended as a one dose treatment or prevention of seasonal and pandemic flu Initiation of Phase 2 clinical proof of concept study in community-acquired setting Ratutrelvir: COVID: an oral Mpro/3CL protease inhibitor, without the need for a CYP-inhibitor such as ritonavir. Intended as a once-a-day, single-dose, 10-day antiviral regimen for the treatment of COVID Initiation of Phase 2 clinical proof of concept study in community-acquired setting Recent Developments Antiviral programs advancing as potential best-in-class agents, poised to begin Phase 2 COVID: Phase 1 data affirms ratutrelvir’s potential to be dosed without ritonavir, with lower rebound risk: Data from the Phase 1 healthy volunteer study, with once-a-day ratutrelvir dosing for 10 days showed no treatment related adverse events and demonstrated consistent plasma drug levels in the predicted therapeutic window. The study showed that ratutrelvir achieved plasma concentrations that were consistently above the EC90 against a panel of SARS-COV-2 viruses, without the need for ritonavir co-administration, which can be a source of drug-drug interactions and severe side effects. Pharmacokinetic data also suggest that ratutrelvir may have a reduced likelihood of clinical rebound, helped to define the dose for Phase 2 studies. Previous preclinical testing in animal models showed that levels of ratutrelvir in the lung were higher than in plasma. Taken together, the data further suggest that ratutrelvir has the potential to be a once-a-day, single-dose, 10-day antiviral therapy for COVID and support Phase 2 studies.Flu: Phase 1 data support tivoaxavir marboxil’s potential to treat flu with a single dose, with potential for pandemic/avian flu: Data from the Phase 1 healthy volunteer study, with one-time tivoxavir marboxil dosing showed good overall tolerability and a pharmacokinetic profile that appears to support potential use as a one-time treatment for flu, including pandemic flu. The study showed that a single dose of tivoxavir maintained plasma drug levels above the EC90 for more than five days and helped to define a Phase 2 dose. Prior preclinical studies have shown that tivoxavir has broad activity against drug resistant viruses and highly pathogenic strains such as avian flu. In addition, preclinical data showed that a single dose of tivoxavir has more than 15X higher accumulation in the lung compared to plasma. The combined dataset support further development of tivoxavir marboxil as a potential treatment of community-acquired influenza or for use in case of an avian flu outbreak or pandemic, with further potential to prevent virus spread in households and congregant settings. Additional Updates: Luba Greenwood, J.D. added to the Board: Ms. Greenwood brings a rich depth of experience as a Board Member, Investor, Strategic Advisor and Company Executive through her industry roles, including as a board member for public life science companies across a range of therapeutic areas. In addition, Traws continues ongoing investigator-sponsored trial driven (IST) development of narazaciclib and rigosertib. Financial Results: Cash, cash equivalents and short-term investments: As of September 30, 2024, the Company had cash, cash equivalents, and short-term investments of approximately $5.4 million, compared to cash, cash equivalents, and short-term investments of approximately $20.8 million at December 31, 2023. Research and development (R&D) expense for the three months ended September 30, 2024, totaled $5.1 million, compared to $2.5 million for the comparable period in 2023. This increase was primarily attributable to the initiation and completion of our Phase 1 study for TRX100 in Australia, partially offset by the reductions in research and development costs attributable to the Australian tax incentive receivable. General and administrative (G&A) expense for the three months ended September 30, 2024, totaled $3.5 million, compared to $2.7 million for the comparable period in 2023. This increase was primarily attributable to a $1.2 million increase in professional and consulting fees associated with seeking strategic alternatives for our investors. This increase was offset by a $0.7 million decrease in public company costs. Net loss: The net loss for the three months ended September 30, 2024 was $8.5 million, or $8.81 per basic and diluted common share. This compares with a net loss of $4.7 million, or $5.64 per basic and diluted common share, for the same period in 2023. Traws had 3,025,554 shares outstandings as of November 11, 2024. The shares outstanding reflect a 25:1 reverse split, effective as of September 20, 2024. About Traws Pharma, Inc.Traws Pharma is a clinical stage biopharmaceutical company developing potential oral small molecule therapies for the treatment of respiratory viral diseases and cancer. The viral respiratory disease program includes two oral, novel, Phase 2-ready, potentially best-in-class, small molecule drug candidates: tivoxavir marboxil, in development for flu and pandemic flu, targeting the influenza cap-dependent endonuclease (CEN); and ratutrelvir, in development as a COVID treatment, targeting the Mpro (3CL protease), without the need for co-administration of ritonavir. In the cancer program, Traws is utilizing a partnering strategy, supported by investigator sponsored studies, to advance two novel proprietary multi-kinase inhibitors, narazaciclib, targeting CDK4+, and rigosertib, targeting cell cycle proteins including PLK-1. Traws is committed to delivering novel compounds for unmet medical needs using state-of-the-art drug development technology. With a focus on product safety and a commitment to patients in need or that are specifically vulnerable, we aim to build solutions for important medical challenges and alleviate the burden of viral infections. Forward-Looking StatementsSome of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995, and involve risks and uncertainties including statements regarding the Company, its business and product candidates, including the potential opportunity, benefits and the regulatory plans for tivoxavir marboxil and ratutrelvir, as well as narazaciclib and rigosertib. The Company has attempted to identify forward-looking statements by terminology including “believes”, “estimates”, “anticipates”, “expects”, “plans”, “intends”, “may”, “could”, “might”, “will”, “should”, “supports”, “preliminary”, “encouraging”, “approximately” or other words that convey uncertainty of future events or outcomes. Although Traws believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including the success and timing of Traws’ clinical trials, collaborations, market conditions and those discussed under the heading “Risk Factors” in Traws’ filings with the U.S. Securities and Exchange Commission and assumes the Company is successful in its fundraising activities. Any forward-looking statements contained in this release speak only as of its date. Traws undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events. Traws Pharma Contact:Mark GuerinTraws Pharma, Inc.267-759-3680www.trawspharma.com Investor Contact:Bruce Mackle LifeSci Advisors, LLC646-889-1200bmackle@lifesciadvisors.com Traws Pharma, Inc.Condensed Consolidated Balance Sheets(unaudited) September 30, December 31, 2024 2023Assets Current assets: Cash and cash equivalents$5,410,000 $20,821,000 Tax incentive and other receivables 2,121,000 18,000 Prepaid expenses and other current assets 1,392,000 1,821,000 Total current assets 8,923,000 22,660,000 Property and equipment, net 12,000 22,000 Other non-current assets 1,000 1,000 Total assets$8,936,000 $22,683,000 Liabilities and stockholders’ (deficit) equity Current liabilities: Accounts payable$5,472,000 $5,619,000 Accrued expenses and other current liabilities 2,650,000 3,375,000 Deferred revenue 226,000 226,000 Total current liabilities 8,348,000 9,220,000 Deferred revenue, non-current 2,621,000 2,791,000 Total liabilities 10,969,000 12,011,000 Commitments and contingencies Stockholders’ (deficit) equity: Series C Preferred stock, $0.01 par value, 5,000,000 shares authorized, 7,440 shares issued and outstanding at September 30, 2024 and no shares issued and outstanding at December 31, 2023 — — Common stock, $0.01 par value, 250,000,000 shares authorized, 3,025,554 and 840,251 shares issued and outstanding at September 30, 2024 and December 31, 2023, respectively 30,000 9,000 Additional paid in capital 617,202,000 493,317,000 Accumulated deficit (619,232,000) (482,631,000)Accumulated other comprehensive loss (33,000) (23,000)Total stockholders’ (deficit) equity (2,033,000) 10,672,000 Total liabilities and stockholders’ (deficit) equity$8,936,000 $22,683,000 Traws Pharma, Inc.Condensed Consolidated Statements of Operations (unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023Revenue$57,000 $57,000 $170,000 $170,000 Operating expenses: Acquired in-process research and development — — 117,464,000 — Research and development 5,113,000 2,460,000 10,989,000 8,996,000 General and administrative 3,480,000 2,686,000 8,813,000 7,010,000 Total operating expenses 8,593,000 5,146,000 137,266,000 16,006,000 Loss from operations (8,536,000) (5,089,000) (137,096,000) (15,836,000)Other income, net 61,000 350,000 495,000 1,072,000 Net loss$(8,475,000) $(4,739,000) $(136,601,000) $(14,764,000)Net loss per share, basic and diluted$(8.81) $(5.64) $(130.87) $(17.59)Basic and diluted weighted average shares outstanding 961,530 840,117 1,043,781 839,243

Phase 1Financial StatementClinical ResultPhase 2Executive Change

12 Oct 2024

·www.globenewswire.com

Traws Pharma Announces Positive Topline Phase 1 Data for Flu Candidate, Tivoxavir Marboxil

Investigational agent in development as a one dose treatment or prevention of seasonal and pandemic influenza A dose ranging, Phase 1 study in healthy volunteers demonstrated positive tolerability results and plasma levels in the predicted therapeutic window, enabling selection of Phase 2 dose Preclinical data showed potent inhibition of drug-resistant and bird influenza viruses Phase 2 study expected to begin in H1 2025 Improved therapy is an important need for both seasonal and pandemic flu Oct. 08, 2024 -- Traws Pharma, Inc. (NASDAQ: TRAW) (“Traws Pharma”, “Traws” or “the Company”), a clinical-stage biopharmaceutical company developing oral small molecule therapies for the treatment of respiratory viral diseases, today announced positive topline Phase 1 safety and pharmacokinetic results for its investigational one-dose influenza (flu) therapy, tivoxavir marboxil (tivoxavir). Tivoxavir was designed as a potential best-in-class inhibitor of the highly-conserved influenza protein, CAP-dependent endonuclease (CEN), intended for use across a broad range of flu viruses. “Topline data from the Phase 1 healthy volunteer study showed good overall tolerability and a pharmacokinetic profile that appears to support tivoxavir’s potential use as a one-time treatment for flu, including pandemic flu. We are especially pleased with data showing that a single dose of tivoxavir maintained plasma drug levels above the EC90 for greater than 5 days1,” said Werner Cautreels, PhD, Chief Executive Officer of Traws Pharma. “In addition, preclinical data show that tivoxavir is a potent inhibitor of drug-resistant influenza and bird flu viruses1. Together, these initial results suggest that tivoxavir has the potential to be developed as a best-in-class agent for influenza. With these data, we plan to advance the program to a Phase 2 study in H1 2025.” “Influenza is a substantial public health burden in the US2, with a disproportionate impact on older adults and vulnerable populations. Data from the last influenza season showed that flu-related hospitalizations3 and mortality4 were highest, by approximately three times, among people 65 years of age or older5. Recent bird flu outbreaks, and the risk that this highly pathogenic virus (H5N1) poses for evolving into a pandemic outbreak6, also signal the need for new antiviral treatments,” said Robert R. Redfield, MD, Chief Medical Officer for Traws Pharma and former Director of the U.S. Centers for Disease Control and Prevention (CDC). “Our enthusiasm for tivoxavir is based on the data announced today and the need for novel therapeutics, especially as valuable resources in case of an avian flu outbreak or a pandemic, and for potential use to prevent virus spread in households and congregant settings.” “We selected CEN as the target for our flu program because it is conserved among human and avian influenza viruses, including the highly pathogenic H5N1. As a result, we believe tivoxavir has the potential to suppress a wide range of viruses. We are very pleased that tivoxavir demonstrated broad activity in laboratory studies against drug-resistant viruses and highly pathogenic strains such as avian flu1. In addition, we believe that preferential uptake in the lung is another important feature of a candidate agent. Preclinical data indicate that a single dose of tivoxavir has more than 15X higher accumulation in the lung compared to plasma1,” said C. David Pauza, PhD, Chief Science Officer for Traws Pharma. “Positive Phase 1 data showing that tivoxavir achieved plasma blood levels in healthy subjects that are consistently above the EC90 and within the predicted therapeutic window for more than five days lead to the identification of our Phase 2 dose, supporting further development of the program.” The Phase 1 trial was a randomized, double-blind, placebo-controlled study to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ascending doses of one-time tivoxavir marboxil treatment in healthy, influenza-negative, adult volunteers. At the identified Phase 2 dose, no treatment related adverse events were reported during the Phase 1 study. Topline data from this study showed that a single dose of tivoxavir marboxil maintained plasma drug levels consistently above the EC90 for more than five days and within the predicted therapeutic window. Preclinical studies showed that tivoxavir marboxil demonstrated potent inhibition of drug-resistant influenza viruses, as well as potent inhibition of highly pathogenic bird flu viruses1. Seasonal influenza is estimated to represent a multi-billion antiviral market opportunity, largely driven by global health organizations, practice guidelines and government tenders1, with upside potential from pandemic flu outbreaks. Tivoxavir marboxil (also known as 27-5116 or TRX-100) was designed as an inhibitor of the highly conserved influenza protein, CAP-dependent endonuclease (CEN). It has demonstrated potent in vitro activity against a range of influenza strains, including the highly pathogenic avian flu, in preclinical studies. The drug candidate’s Phase 1 pharmacokinetic (PK) profile in healthy subjects, including the ability to achieve plasma levels that are consistently above the EC90 (as determined in laboratory studies), for more than five days and within the predicted therapeutic window, may enable a single dose treatment regimen. These data, combined with good overall tolerability results in healthy subjects, support further development of tivoxavir marboxil as a one-time treatment for influenza. Traws Pharma is a clinical stage biopharmaceutical company developing potential oral small molecule therapies for the treatment of respiratory viral diseases and cancer. The viral respiratory disease program includes two oral, novel, Phase 1, potentially best-in-class, small molecule drug candidates: tivoxavir marboxil, in development for flu and pandemic flu, targeting the influenza cap-dependent endonuclease (CEN); and ratutrelvir, in development as a COVID treatment, targeting the Mpro (3CL protease), without the need for co-administration of ritonavir. In the cancer program, Traws is utilizing a partnering strategy, supported by investigator sponsored studies, to advance two novel proprietary multi-kinase inhibitors, narazaciclib, targeting CDK4+, and rigosertib, targeting cell cycle proteins including PLK-1. Traws is committed to delivering novel compounds for unmet medical needs using state-of-the-art drug development technology. With a focus on product safety and a commitment to patients in need or that are specifically vulnerable, we aim to build solutions for important medical challenges and alleviate the burden of viral infections. 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Clinical Result

100 Deals associated with Rigosertib Sodium

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D10155	Rigosertib Sodium	-	DB12146

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Indication	Highest Phase	Country/Location	Organization	Date
Metastatic Pancreatic Cancer	Phase 3	United States	Traws Pharma, Inc.	01 May 2011
Metastatic Pancreatic Cancer	Phase 3	Hungary	Traws Pharma, Inc.	01 May 2011
Metastatic Pancreatic Cancer	Phase 3	India	Traws Pharma, Inc.	01 May 2011
Metastatic Pancreatic Cancer	Phase 3	Russia	Traws Pharma, Inc.	01 May 2011
Metastatic Pancreatic Cancer	Phase 3	Ukraine	Traws Pharma, Inc.	01 May 2011
Pancreatic adenocarcinoma metastatic	Phase 3	United States	Traws Pharma, Inc.	01 May 2011
Pancreatic adenocarcinoma metastatic	Phase 3	Hungary	Traws Pharma, Inc.	01 May 2011
Pancreatic adenocarcinoma metastatic	Phase 3	India	Traws Pharma, Inc.	01 May 2011
Pancreatic adenocarcinoma metastatic	Phase 3	Russia	Traws Pharma, Inc.	01 May 2011
Pancreatic adenocarcinoma metastatic	Phase 3	Ukraine	Traws Pharma, Inc.	01 May 2011

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04263090 (Corporate Publications) Manual	Phase 1/2	KRAS mutant Non-small Cell Lung Cancer KRAS Mutation	19	Rigosertib+nivolumab	aqswrywuck(gonzjnlmfm) = wwjewpuapv wbfaawamzh (sxfiqmhzil ) View more	Positive	12 Sep 2022
NCT04263090 (ESMO2022)	Phase 1/2	KRAS mutant Non-small Cell Lung Cancer Second line	18	Rigosertib+Nivolumab	qzoejtijgu(hciteqggzj) = dysuria/hematuria (65%) qlatnkmfhk (sbwqnmtuoa ) View more	Positive	10 Sep 2022
NCT04263090 (GlobeNewswire) Manual	Phase 1/2	RAS positive Non-Small Cell Lung Cancer KRAS Positive	12	Rigosertib Sodium+Nivolumab	udnkmdbbef(fgpfbqouxw) = zdogepjanb cxqqqrdrkv (cmycotcdld ) View more	Positive	22 Sep 2021
NCT02730884 (CTgov)	Phase 2	Anemia \| Mycosis Fungoides \| Myelofibrosis ... View more	3	Questionnaire+Rigosertib	zoxfmqfhdy = ccqykgqngl vogticwsxl (tgghedukze, udeycipisb - soeiivxcev) View more	-	07 Aug 2020
NCT01926587 (Pubmed \| Leuk Res) Manual	Phase 1	Acute Myeloid Leukemia \| Myelodysplastic Syndromes	18	azacitidine+Rigosertib	kwsjqokjpi(kgfhrrqowz) = aaxejnhkxb gmyysjpxln (xngbsahjoq ) View more	Positive	01 Jul 2020
ASH 12019 \| ASH 22019 Manual	Phase 2	Myelodysplastic Syndromes First line	-	Azacitidine+rigosertib	fgfmbvizfe(jknbdkeegh) = 26% [urinary tract pain (2), and 1 each for urinary retention, hematuria, hydronephrosis, osteolysis, cerebral haemorrhage, WBC count decreased, neutrophil count decreased, & abd pain] hviflabygb (nvbwzoltzf )	Positive	13 Nov 2019
EHA2019	Phase 1/2	High Risk Myelodysplastic Syndrome	-	Oral rigosertib + Azacitidine	glwgzxoezi(ikvvwpqkpj) = dysuria bmwckkuzjx (kjmwqvshjd ) View more	-	15 Jun 2019
NCT00854646 (Pubmed) Manual	Phase 1/2	Acute Myeloid Leukemia \| Myelodysplastic Syndromes	22	rigosertib	usgofmuhyd(rdilomxklk) = fatigue, diarrhea, pyrexia, dyspnea, insomnia, and anemia. vkxxvgcoms (veuaaygilr )	Positive	01 Jan 2018
EHA2017	Phase 1/2	Myelodysplastic Syndromes	-	Rigosertib+Azacitidine	vcvxuamaqf(dqhmddgfyn) = ziqaravqaz rcfjowjekf (sjftgojdur )	-	18 May 2017
EHA2017	Phase 1/2	Myelodysplastic Syndromes	-	Rigosertib+Vorinostat	wnypzobego(cgothqpuck) = tsskzgkibp agktsntbbw (bcxhkuwgxr )	-	18 May 2017
EHA2017	Phase 1	Refractory Myelodysplastic Syndrome \| Recurrent Myelodysplastic Syndrome	9	Rigosertib 560 mg BID	iemvtgynkh(fzxiryqmxw) = One case of grade 3 neutropenia developed in the 280 mg BID arm bzxexkudbh (sprcjmqafj ) View more	-	18 May 2017

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