Latest news with #pharmacokinetics
Business Wire
29-05-2025
- Health
- Business Wire
Drug Farm Reports Data on Safety and Pharmacokinetics from Phase 1 First-in-Human Trial of DF-003 in Healthy Volunteers
ALBANY, N.Y. & SHANGHAI--(BUSINESS WIRE)--Drug Farm, a private biotechnology company utilizing genetics and artificial intelligence technologies to discover and develop innovative, immune-modulating therapies, today announced positive results from a recently completed Phase 1 study (NCT05997641) of DF-003 in healthy volunteers. The data were presented at the American Society for Clinical Pharmacology & Therapeutics (ASCPT) meeting in Washington, DC. DF-003 is a first-in-class, immune-modulating alpha-kinase 1 (ALPK1) inhibitor that targets the root cause of the rare genetic disease, ROSAH syndrome. This randomized, placebo-controlled, double-blinded study evaluated the safety, tolerability and pharmacokinetics of DF-003 in forty-eight healthy volunteers. In the first portion of the trial, single ascending doses were orally administered in five cohorts (randomized 3:1; DF-003: placebo) ranging from 3 mg up to 150 mg. In the second portion of the trial, 50 mg of DF-003 was orally administered daily for fourteen consecutive days in one cohort (randomized 3:1; DF-003: placebo) in eight healthy volunteers. The primary objective of the study was to evaluate the safety and tolerability of DF-003, as well as its pharmacokinetic properties. Key Results Safety: DF-003 was safe at all doses tested with no serious adverse events described. Rates of treatment emergent adverse events (TEAEs) were similar between active and placebo-treated participants, and no TEAEs required dose modification or interruption. Pharmacokinetics: The pharmacokinetic profile of DF-003 was largely dose proportional and the data support additional clinical trials as a once-daily, orally administered drug. 'We are happy to share the results from our Phase 1 study that demonstrate the excellent safety of DF-003,' said Neil Solomons, MD, Head, Clinical Development at Drug Farm. 'We are also pleased that the pharmacokinetic profile of DF-003 leads to trough concentrations in blood that are consistent with efficacy in preclinical models of ROSAH syndrome and cardio-renal disease. The dose ranges explored in this Phase 1 trial will be used in our upcoming proof of concept trials in ROSAH syndrome and cardio-renal disease patient populations.' 'This is a major inflection point for Drug Farm in our quest to advance DF-003, a first-in-class, immune-modulating drug that precision targets the disease-causing mutations of ROSAH syndrome, as well as the excessive activation of the ALPK1 pathway found in cardio-renal disease,' said Henri Lichenstein, PhD, Chief Executive Officer at Drug Farm. 'There are no approved drugs for ROSAH syndrome, and we are excited about the potential of DF-003 to save sight and to ameliorate other debilitating inflammatory symptoms associated with this disease.' About DF-003 DF-003 is a proprietary, first-in-class drug developed by Drug Farm that inhibits the activity of ALPK1 and variants of ALPK1 which cause ROSAH syndrome. DF-003 has therapeutic potential for ROSAH syndrome and cardio-renal disease as the drug has shown efficacy in preclinical models of these disease indications. DF-003 has completed a Phase 1 clinical trial (NCT05997641) in normal healthy volunteers and is now accruing patients with ROSAH syndrome in a Phase 1b trial (NCT06395285). About ROSAH Syndrome ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) syndrome is a rare, autosomal dominant autoinflammatory genetic disease named according to the characteristic symptoms exhibited by affected patients (1, 2). Disease-causing mutations in ALPK1 lead to ROSAH syndrome. The most common presenting symptom is a progressive decline in visual acuity that typically begins before 20 years of age, with ophthalmologic examination often revealing optic disc elevation, uveitis, and retinal nerve degeneration (2, 3). Most ROSAH patients also exhibit inflammatory features such as non-infectious low-grade fevers, arthralgia, headaches, and persistently elevated levels of inflammatory cytokines including tumor necrosis factor-α (TNFα), interleukin-6 (IL-6), and IL-1β (3). 1. Tantravahi SK, et al. An inherited disorder with splenomegaly, cytopenias, and vision loss. Am J Med Genet A. 2012;158(3):475-81. 2. Williams LB, et al. ALPK1 missense pathogenic variant in five families leads to ROSAH syndrome, an ocular multisystem autosomal dominant disorder. Genet Med. 2019;21(9):2103-15. 3. Kozycki CT, et al. Gain-of-function mutations in ALPK1 cause an NF-κB-mediated autoinflammatory disease: functional assessment, clinical phenotyping and disease course of patients with ROSAH syndrome. Ann Rheum Dis. 2022;81(10):1453-64. About Drug Farm Drug Farm is a private biotechnology Company developing innovative treatments targeting innate immunity for hepatitis B, heart and kidney diseases, and ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) syndrome. Drug Farm's unique IDInVivo platform combines breakthrough technologies in genetics and AI to discover new treatments. IDInVivo technology allows the direct assessment of gene targets in living animals with intact immune systems. Using the IDInVivo platform, Drug Farm has identified novel innate immunity pathways and targets and is now rapidly advancing multiple first-in-class drug candidates into clinical development. For more information please visit:
Associated Press
12-05-2025
- Business
- Associated Press
Circle Pharma to Present Trials in Progress Poster on CID-078, a First-in-Class Cyclin A/B Inhibitor, at the ASCO 2025 Annual Meeting
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--May 12, 2025-- Circle Pharma, a clinical-stage biopharmaceutical company advancing macrocycle therapeutics for difficult-to-treat cancers, today announced that its Trials in Progress abstract has been selected for poster presentation at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting taking place May 30 to June 3 in Chicago. The poster will showcase the design and ongoing progress of its Phase 1 study of CID-078 (NCT06577987), a first-in-class, oral macrocycle cyclin A/B RxL inhibitor. The abstract will be featured in a Poster Session focused on Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology, under the New Targets and New Technologies (non-IO) subtrack. Poster Presentation Details: Abstract Title:A phase 1 study to evaluate the safety, pharmacokinetics, and efficacy of the first-in-class cyclin A/B RxL inhibitor CID-078, an orally bioavailable, cell-permeable macrocycle First Author: Nehal Lakhani, MD, PhD, The START Center for Cancer Research, Grand Rapids, MI Abstract Number: TPS3175 Poster Board Number: 481a Session Title: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology Date & Time: June 2, 2025, from 1:30 PM – 4:30 PM CDT 'CID-078 exemplifies Circle Pharma's commitment to advancing new treatments for patients with cancer,' said Michael C. Cox, PharmD, MHSc, BCOP, SVP, and head of early development of Circle Pharma. 'CID-078 has shown strong single-agent activity in tumor models with dysregulated cell cycle pathways, where activity has been linked to specific molecular features such as RB1 mutations and high E2F pathway scores. We believe CID-078 has the potential to be a new treatment option for patients with solid tumors such as small-cell lung cancer or triple-negative breast cancer which are known to harbor these alterations, or solid tumors harboring an RB mutation identified through comprehensive genomic profiling. We are encouraged by the progress of the CID-078 phase 1 study, and we're excited to share early clinical insights at ASCO 2025.' Contributing Authors: William McKean, Ildefonso Rodriguez Rivera, Antonio Giordano, Timothy Yap, Afshin Dowlati, Vivek Subbiah, Judy Wang, Manali Bhave, Kyaw Thein, Jinshu Fang, Eric Connor, Li-Fen Liu, Peadar Cremin, Lukas Makris, Li-Pen Tsao, Lisa Kopp, Michael Cox, and Shivaani Kummar. About CID-078, Circle Pharma's Cyclin A/B RxL Inhibitor Program CID-078 is an orally bioavailable macrocycle with dual cyclin A and B RxL inhibitory activity that selectively targets tumor cells with oncogenic alterations that cause cell cycle dysregulation. In biochemical and cellular studies, Circle Pharma's cyclin A/B RxL inhibitors have been shown to potently and selectively disrupt the protein-to-protein interaction between cyclins A and B and their key substrates and modulators, including E2F (a substrate of cyclin A) and Myt1 (a modulator of cyclin B). Preclinical studies have demonstrated the ability of these cyclin A/B RxL inhibitors to cause single-agent tumor regressions in multiple in vivo models. A multi-center phase 1 clinical trial ( NCT06577987 ) is currently enrolling patients. About Circle Pharma, Inc. South San Francisco-based Circle Pharma is a clinical-stage biopharmaceutical company harnessing the power of macrocycles to develop therapies for cancer and other serious illnesses. The company's proprietary MXMO™ platform overcomes key challenges in macrocycle drug development, enabling the creation of intrinsically cell-permeable and orally bioavailable therapies for historically undruggable targets. Circle Pharma's pipeline is focused on targeting cyclins, key regulators of the cell cycle that drive many cancers. Its lead program, CID-078, a cyclin A/B-RxL inhibitor, is in a Phase 1 clinical trial (NCT06577987) for patients with advanced solid tumors. To learn more about Circle Pharma, please visit View source version on CONTACT: Media Contact: Roslyn Patterson Phone:650.825.4099 Email:[email protected] KEYWORD: UNITED STATES NORTH AMERICA CALIFORNIA ILLINOIS INDUSTRY KEYWORD: ONCOLOGY PROFESSIONAL SERVICES HEALTH VENTURE CAPITAL CLINICAL TRIALS PHARMACEUTICAL BIOTECHNOLOGY SOURCE: Circle Pharma Copyright Business Wire 2025. PUB: 05/12/2025 07:05 AM/DISC: 05/12/2025 07:05 AM
Business Wire
12-05-2025
- Business
- Business Wire
Circle Pharma to Present Trials in Progress Poster on CID-078, a First-in-Class Cyclin A/B Inhibitor, at the ASCO 2025 Annual Meeting
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Circle Pharma, a clinical-stage biopharmaceutical company advancing macrocycle therapeutics for difficult-to-treat cancers, today announced that its Trials in Progress abstract has been selected for poster presentation at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting taking place May 30 to June 3 in Chicago. The poster will showcase the design and ongoing progress of its Phase 1 study of CID-078 (NCT06577987), a first-in-class, oral macrocycle cyclin A/B RxL inhibitor. 'CID-078 exemplifies Circle Pharma's commitment to advancing new treatments for patients with cancer,' said Michael C. Cox, PharmD, MHSc, BCOP, SVP, and head of early development of Circle Pharma. Share The abstract will be featured in a Poster Session focused on Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology, under the New Targets and New Technologies (non-IO) subtrack. Poster Presentation Details: Abstract Title: A phase 1 study to evaluate the safety, pharmacokinetics, and efficacy of the first-in-class cyclin A/B RxL inhibitor CID-078, an orally bioavailable, cell-permeable macrocycle First Author: Nehal Lakhani, MD, PhD, The START Center for Cancer Research, Grand Rapids, MI Abstract Number: TPS3175 Poster Board Number: 481a Session Title: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology Date & Time: June 2, 2025, from 1:30 PM – 4:30 PM CDT 'CID-078 exemplifies Circle Pharma's commitment to advancing new treatments for patients with cancer,' said Michael C. Cox, PharmD, MHSc, BCOP, SVP, and head of early development of Circle Pharma. 'CID-078 has shown strong single-agent activity in tumor models with dysregulated cell cycle pathways, where activity has been linked to specific molecular features such as RB1 mutations and high E2F pathway scores. We believe CID-078 has the potential to be a new treatment option for patients with solid tumors such as small-cell lung cancer or triple-negative breast cancer which are known to harbor these alterations, or solid tumors harboring an RB mutation identified through comprehensive genomic profiling. We are encouraged by the progress of the CID-078 phase 1 study, and we're excited to share early clinical insights at ASCO 2025.' Contributing Authors: William McKean, Ildefonso Rodriguez Rivera, Antonio Giordano, Timothy Yap, Afshin Dowlati, Vivek Subbiah, Judy Wang, Manali Bhave, Kyaw Thein, Jinshu Fang, Eric Connor, Li-Fen Liu, Peadar Cremin, Lukas Makris, Li-Pen Tsao, Lisa Kopp, Michael Cox, and Shivaani Kummar. About CID-078, Circle Pharma's Cyclin A/B RxL Inhibitor Program CID-078 is an orally bioavailable macrocycle with dual cyclin A and B RxL inhibitory activity that selectively targets tumor cells with oncogenic alterations that cause cell cycle dysregulation. In biochemical and cellular studies, Circle Pharma's cyclin A/B RxL inhibitors have been shown to potently and selectively disrupt the protein-to-protein interaction between cyclins A and B and their key substrates and modulators, including E2F (a substrate of cyclin A) and Myt1 (a modulator of cyclin B). Preclinical studies have demonstrated the ability of these cyclin A/B RxL inhibitors to cause single-agent tumor regressions in multiple in vivo models. A multi-center phase 1 clinical trial (NCT06577987) is currently enrolling patients. About Circle Pharma, Inc. South San Francisco-based Circle Pharma is a clinical-stage biopharmaceutical company harnessing the power of macrocycles to develop therapies for cancer and other serious illnesses. The company's proprietary MXMO™ platform overcomes key challenges in macrocycle drug development, enabling the creation of intrinsically cell-permeable and orally bioavailable therapies for historically undruggable targets. Circle Pharma's pipeline is focused on targeting cyclins, key regulators of the cell cycle that drive many cancers. Its lead program, CID-078, a cyclin A/B-RxL inhibitor, is in a Phase 1 clinical trial (NCT06577987) for patients with advanced solid tumors. To learn more about Circle Pharma, please visit
Globe and Mail
25-02-2025
- Business
- Globe and Mail
Advanced Merkel Cell Carcinoma Clinical Trials and Studies: EMA, PDMA, FDA Approvals, Mechanism of Action, ROA, NDA, IND, and Companies
DelveInsight's, 'Advanced Merkel cell carcinoma Pipeline Insight' report provides comprehensive insights about 20+ companies and 20+ pipeline drugs in Advanced Merkel cell carcinoma pipeline landscape. It covers the Advanced Merkel Cell Carcinoma pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Advanced Merkel Cell Carcinoma pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space. Explore our latest breakthroughs in Advanced Merkel Cell Carcinoma Research. Learn more about our innovative pipeline today! @ Advanced Merkel Cell Carcinoma Pipeline Outlook Key Takeaways from the Advanced Merkel Cell Carcinoma Pipeline Report In February 2025:- Sensei Biotherapeutics Inc.:- Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of SNS-101, a novel anti VISTA IgG1 monoclonal antibody as monotherapy or in combination with cemiplimab in patients with advanced solid tumors. In January 2025:- University of Washington:- This phase II trial tests how well a combination of three immunotherapy drugs work for patients with Merkel cell carcinoma that has spread to lymph nodes and/or distant parts of the body and cannot be treated with surgery (advanced or metastatic MCC) and grew despite prior PD-(L)1 therapy. The three drugs INCMGA00012 (retifanlimab, anti-PD-1), INCAGN02385 (tuparstobart, anti-LAG-3), and INCAGN02390 (verzistobart, anti-TIM-3) are monoclonal antibodies given periodically via IV to reactivate the body's immune system to attack the cancer. In January 2025:- Marengo Therapeutics Inc.:- This Phase 1/2 study consists of two parts: Phase 1 Dose Escalation and Phase 2 Dose Expansion. In Phase 1 Dose Escalation, STAR0602 will be administered intravenously in participants with advanced solid tumors to assess safety/tolerability profile of STAR0602 and to determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D) of STAR0602. In Phase 2 Dose Expansion, STAR0602 at RP2D will be administered to participants with advanced, antigen-rich solid tumors to further evaluate safety and assess preliminary clinical activity of STAR0602. Clinical activity will be evaluated by objective tumor response rate (ORR), duration of response (DOR), disease control rate (DCR), and progression free survival (PFS). DelveInsight's Advanced Merkel Cell Carcinoma Pipeline report depicts a robust space with 20+ active players working to develop 20+ pipeline therapies for Advanced Merkel Cell Carcinoma treatment. The leading Advanced Merkel Cell Carcinoma Companies such as Xencor, Inc., Incyte Corporation, Millennium Pharmaceuticals, Inc., Exelixis, Bristol-Myers Squibb, bluebird bio, Affini-T Therapeutics, ImaginAb, Inc., BioInvent International AB, Ocellaris Pharma, Inc., Sotio a.s., Morphogenesis, Inc., Checkpoint Therapeutics, Inc., and others. Promising Advanced Merkel Cell Carcinoma Therapies such as Avelumab, BT-001, GI-101, Pembrolizumab (KEYTRUDA®), Lenvatinib, OC-001, and others. Stay informed about the cutting-edge advancements in Advanced Merkel Cell Carcinoma treatments. Download for updates and be a part of the revolution in cancer care @ Advanced Merkel Cell Carcinoma Clinical Trials Assessment Advanced Merkel cell carcinoma Emerging Drugs Profile Tidutamab: Xencor, Inc. Tidutamab (previously XmAb18087) is a bispecific antibody that engages the immune system against tumors by binding to somatostatin receptor 2 (SSTR2) and CD3. Xencor's XmAb® Bispecific Fc Domain serves as the scaffold for these two antigen binding domains and confers long circulating half-life, stability and ease of manufacture on tidutamab. Engagement of CD3 by tidutamab activates T cells for highly potent and targeted killing of SSTR2-expressing tumor cells. Cabozantinib: Exelixis In the U.S., CABOMETYX tablets are approved for the treatment of patients with advanced renal cell carcinoma (RCC); for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib; for patients with advanced RCC as a first-line treatment in combination with nivolumab; and for adult and pediatric patients 12 years of age and older with locally advanced or metastatic differentiated thyroid cancer (DTC) that has progressed following prior vascular endothelial growth factor receptor (VEGFR)-targeted therapy and who are radioactive iodine-refractory or ineligible. CABOMETYX tablets have also received regulatory approvals in the European Union and additional countries and regions worldwide. In 2016, Exelixis granted Ipsen Pharma SAS exclusive rights for the commercialization and further clinical development of cabozantinib outside of the U.S. and Japan. In 2017, Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited for the commercialization and further clinical development of cabozantinib for all future indications in Japan. Exelixis holds the exclusive rights to develop and commercialize cabozantinib in the the drug is in Phase II stage of Clinical trial evaluation for the treatment of Advanced Merkel cell carcinoma. The Advanced Merkel Cell Carcinoma pipeline report provides insights into The report provides detailed insights about companies that are developing therapies for the treatment of Advanced Merkel Cell Carcinoma with aggregate therapies developed by each company for the same. It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Advanced Merkel Cell Carcinoma Treatment. Advanced Merkel Cell Carcinoma Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects. Advanced Merkel Cell Carcinoma Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type. Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Advanced Merkel Cell Carcinoma market. Learn more about Advanced Merkel Cell Carcinoma Drugs Opportunities in our groundbreaking Advanced Merkel Cell Carcinoma Research and development projects @ Advanced Merkel Cell Carcinoma Unmet Needs Advanced Merkel Cell Carcinoma Companies Xencor, Inc., Incyte Corporation, Millennium Pharmaceuticals, Inc., Exelixis, Bristol-Myers Squibb, bluebird bio, Affini-T Therapeutics, ImaginAb, Inc., BioInvent International AB, Ocellaris Pharma, Inc., Sotio a.s., Morphogenesis, Inc., Checkpoint Therapeutics, Inc., and others. Advanced Merkel cell carcinoma pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration Oral Parenteral intravenous Subcutaneous Topical. Advanced Merkel Cell Carcinoma Products have been categorized under various Molecule types such as Monoclonal Antibody Peptides Polymer Small molecule Gene therapy Discover the latest advancements in Advanced Merkel Cell Carcinoma treatment by visiting our website. Stay informed about how we're transforming the future of oncology @ Advanced Merkel Cell Carcinoma Market Drivers and Barriers, and Future Perspectives Scope of the Advanced Merkel Cell Carcinoma Pipeline Report Coverage- Global Advanced Merkel Cell Carcinoma Companies- Xencor, Inc., Incyte Corporation, Millennium Pharmaceuticals, Inc., Exelixis, Bristol-Myers Squibb, bluebird bio, Affini-T Therapeutics, ImaginAb, Inc., BioInvent International AB, Ocellaris Pharma, Inc., Sotio a.s., Morphogenesis, Inc., Checkpoint Therapeutics, Inc., and others. Advanced Merkel Cell Carcinoma Therapies- Avelumab, BT-001, GI-101, Pembrolizumab (KEYTRUDA®), Lenvatinib, OC-001, and others. Advanced Merkel Cell Carcinoma Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination Advanced Merkel Cell Carcinoma Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III For a detailed overview of our latest research findings and future plans, read the full details of Advanced Merkel Cell Carcinoma Pipeline on our website @ Advanced Merkel Cell Carcinoma Emerging Drugs and Companies Table of Content Introduction Executive Summary Advanced Merkel cell carcinoma: Overview Pipeline Therapeutics Therapeutic Assessment Advanced Merkel cell carcinoma – DelveInsight's Analytical Perspective Mid Stage Products (Phase II) Cabozantinib: Exelixis Drug profiles in the detailed report….. Early Stage Products (Phase I) IFx-Hu2.0: Morphogenesis Drug profiles in the detailed report….. Inactive Products Advanced Merkel cell carcinoma Key Companies Advanced Merkel cell carcinoma Key Products Advanced Merkel cell carcinoma- Unmet Needs Advanced Merkel cell carcinoma- Market Drivers and Barriers Advanced Merkel cell carcinoma- Future Perspectives and Conclusion Advanced Merkel cell carcinoma Analyst Views Advanced Merkel cell carcinoma Key Companies Appendix About Us DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve. 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