Metastatic Solid Tumor

Clinical Trials Showcased at Global Gathering FORT MYERS, Fla., April 5, 2024 /PRNewswire/ -- Clinical research conducted at Florida Cancer Specialists & Research Institute, LLC (FCS) will be highlighted at the American Association for Cancer Research 2024 Annual Meeting in San Diego as scientists, clinicians and other health care professionals and stakeholders from all over the world gather to share the latest advances in cancer science and medicine. Continue Reading Over a dozen FCS abstracts will be presented at the 2024 AACR annual meeting. "Finding cancer cures requires diligence and collaboration among the greatest minds in cancer research," noted Lucio N. Gordan, MD, FCS President & Managing Physician. "Through our extensive and growing clinical trials research program, FCS is proud to be part of global efforts that are saving lives and enhancing the quality of life for patients and their families." FCS provides ongoing access to more than 160 clinical trials within 32 FCS clinics and three Drug Development Units (early-phase) across Florida, in Sarasota, Lake Mary and Lake Nona. In recent years, the majority of new cancer drugs approved for use in the U.S. were studied in clinical trials with FCS participation, prior to approval. FCS physician investigators will present findings during oral and poster sessions at the ACCR 2024 Annual Meeting. FCS abstracts at AACR 2024 Annual Meeting: 068, 075, 132, 141, 156, 163, 291, 6552, 7172, 099, 077, 2565 FCS Director of Drug Development Manish Patel, MD, co-author of nine abstracts: Interim results of the ongoing phase 1-2 clinical trial of KVA12123, an engineered IgG1 targeting VISTA, as monotherapy and in combination with pembrolizumab in patients with advanced solid tumors Phase 1 trial evaluating AC699, an orally bioavailable chimeric estrogen receptor degrader, in patients with advanced or metastatic breast cancer Phase I/IIa clinical trial (TranStar101) to evaluate the safety, tolerability and pharmacokinetics of OSEMITAMAB administered as monotherapy or in combination with nivolumab or standard of care in patients with locally advanced or metastatic solid tumors mRNA-4157 (V940) individualized neoantigen therapy (INT) + pembrolizumab (pembro) in advanced unresectable HPV- head & neck carcinoma (HNSCC): Clinical & translational analysis Phase I study evaluating AC676, an innovative BTK chimeric degrader, in patients with relapsed and refractory B-cell malignancies Phase 1 clinical trial of the farnesyl transferase inhibitor KO-2806 alone or as part of combination therapy for advanced solid tumors Phase 1/2, first-in-human study of DS-3939a in patients with advanced solid tumors: A new DXd ADC targeting TA-MUC1 Intratumoral (ITu) delivery of mRNA-2752 encoding human OX40L/IL-23/IL-36γ in combination with durvalumab induces an immunostimulatory effect within the tumor microenvironment (TME) of patients with advanced solid tumors Pharmacokinetic/biomarker (PK/BMx) analysis of the toll-like receptor 7 and 8 (TLR7/8) agonist EIK1001 in phase 1 studies in participants (Pts) with solid tumors FCS Associate Director of Drug Development Judy Wang, MD first author: NGM707 in combination with pembrolizumab in advanced or metastatic solid tumors: Preliminary results from dose escalation FCS Scientific Director of Research and Associate Professor of Medicine, Wake Forest University School of Medicine Lowell Hart, MD, FACP, first author: Vitamin D deficiency and outcomes in prostate cancer patients-A real world database Co-authors include FCS Trial Match Specialist and Medical Oncologist and Hematologist (retired) James Reeves Jr., MD, FCS Nurse Specialist Stacey Garofalo RN and members of the FCS Real-World Evidence Team, IT Director Data Science Derek Hall and Informatics Data Scientist Parth Kothiya. FCS Director of Drug Development, Lake Nona DDU, Cesar Augusto Perez, MD, co-author: Open-label phase 1 study to investigate SGN-CEACAM5C in adults with advanced solid tumors "Our clinical trials research contributes daily to the discovery of ground-breaking new drugs and therapies that are accelerating our understanding of cancer and transforming the standard of care," said FCS Director of Late-Phase Clinical Research Bradley Monk, MD. Dr. Monk is co-author of a study conducted at HonorHealth University of Arizona College of Medicine and Creighton University School of Medicine prior to being named to his leadership position at FCS earlier this year, "Tumor microenvironment (TME) biomarkers of TIGIT and PD-L1 immune checkpoint blockade in cervical cancer: An exploratory biomarker analysis from SKYSCRAPER-04 (SKY04) study." Abstracts will be published in the AACR journal, Cancer Research, following the annual meeting. The American Association for Cancer Research (AACR), the first and largest cancer research organization dedicated to accelerating the conquest of cancer, represents 58,000 members residing in 141 countries. About Florida Cancer Specialists & Research Institute, LLC: (FLCancer.com) Florida Cancer Specialists & Research Institute (FCS) offers patients access to more clinical trials than any private oncology practice in Florida. The majority of new cancer drugs recently approved for use in the U.S. were studied in clinical trials with FCS participation.* Recognized for our research, FCS is a recipient of the national Clinical Trials Participation Award presented by the American Society of Clinical Oncology (ASCO). FCS physicians, trained in prestigious medical schools and research institutes, are consistently ranked nationally as Top Doctors by U.S. News & World Report. Celebrating its 40th year in 2024, FCS has built a national reputation for excellence that is reflected in exceptional and compassionate patient care, driven by innovative clinical research, cutting-edge technologies and advanced treatments, including targeted therapies genomic-based treatment and immunotherapy. Our highest values are embodied by our outstanding team of highly trained and dedicated physicians, clinicians and staff. *Prior to approval SOURCE Florida Cancer Specialists & Research Institute

– NKT3447 is a highly selective, orally bioavailable small molecule CDK2 inhibitor with prolonged pharmacodynamic effects and a unique mechanism of action, including downregulation of cyclin E and suppression of activating phosphorylation of CDK2 on Thr160 – WILMINGTON, Del.--(BUSINESS WIRE)-- NiKang Therapeutics Inc. (“NiKang”), a clinical stage biotech company focused on developing innovative small molecule oncology medicines to help patients with unmet medical needs, announced today the unveiling of NKT3447 at the American Association for Cancer Research (AACR) Annual Meeting 2024. NKT3447 is an orally bioavailable small molecule CDK2 inhibitor that exhibits high selectivity against CDK1 and other CDKs, prolonged pharmacodynamic effects, and a distinct mechanism of action. It downregulates cyclin E and suppresses activating phosphorylation of CDK2 on Thr160. “We are pleased to share the discovery of NKT3447, a highly selective CDK2 inhibitor with unique properties, at the AACR Annual Meeting”, said Zhenhai Gao, Ph.D., co-founder, president, and CEO of NiKang. “This is the first of three programs targeting the cell cycle that we are expecting to bring to clinic. We have strong conviction in CDK2 as an oncology target and have built an industry-leading potential best in class and/or first in class portfolio that also includes a CDK2-selective degrader and a CDK2/4 dual degrader. Despite clinical success with drugs targeting the cell cycle, it has been challenging to identify inhibitors of CDK2 which have sufficient selectivity against CDK1, and which do not cause a compensatory increase of cyclin E. We have developed a platform to bring unique and selective drugs targeting CDK2 which address these challenges to clinic. We appreciate the opportunity to share the unique properties of NKT3447 and are enthusiastic about exploring the potential of NKT3447 in treating various cancers driven by CDK2 and cyclin E.” NiKang has initiated a phase 1/1b, open-label, dose escalation and expansion study of NKT3447 as a single agent. This first-in-human study (NCT06264921) is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and clinical activity of NKT3447, administered orally, in adult patients with advanced or metastatic solid tumors. Poster Presentation Details: Title: Discovery of a selective slow-off CDK2 inhibitor NKT3447 with distinct features of suppressing CDK2, downregulating cyclin E, and achieving prolonged pathway inhibition Presenter: Ke Liu, Ph.D. Abstract Number: 5705/5 (link to abstract) Session: Pharmacologic Targeting of Cell Cycle Proteins Date/Time: 1:30 - 5:00pm PT on April 9, 2024 About NiKang Therapeutics NiKang Therapeutics is a clinical stage biotech company focused on discovering and developing innovative small molecule oncology medicines to help patients with unmet medical needs. Our target selection is driven by deep insight into disease biology and molecular pathways. Our discovery approach is informed by target structure biology and capitalizes on structure-based drug design. The successful implementation of our strategy enables us to rapidly and efficiently discover and advance proprietary drug candidates with the most desirable pharmacological features into clinical studies. We strive to bring transformative medicines to patients in need. For more information, please visit

Initiation of Phase II clinical trial of OATD-01 (KITE) for the treatment of pulmonary sarcoidosis following approval from the US Food and Drug Administration (FDA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA). World's first administration of a chitotriosidase 1 inhibitor (or placebo) to a patient with active pulmonary sarcoidosis as part of the Phase II OATD-01 clinical trial (proof-of-concept in human) at a hospital in the UK. First success in mRNA platform development - inhibited translation of pathogenic proteins in a cellular assay (in vitro proof-of-concept) - achieving an important discovery milestone in the development of a breakthrough small-molecule drug platform targeting mRNA. Further advances in the Phase I clinical trial of OATD-02, the first-in-class dual arginase inhibitor being developed for the treatment of cancer, systematic dose escalation and progression to a fourth cohort of patients with solid tumours, to determine the maximum tolerated dose and recommended dose for the next phase of trials. Further development of the most advanced project in the preclinical phase - the USP7 inhibitor programme for use in anti-cancer therapies using artificial intelligence tools and models. Warsaw, April 3, 2024. - Molecure S.A. ("Molecure", WSE ticker: MOC), a biotechnology company that discovers and develops drugs to the clinical stage and leverages its globally unique expertise in medicinal chemistry and biology to explore and develop first-in-class small-molecule drugs that directly modulate protein activity and mRNA function to treat a range of incurable diseases, has published its 2023 annual report. The report is available at: “2023 was an exceptional year for Molecure as we made significant progress in the development of both clinical and preclinical programmes, particularly in the area of small-molecule drugs targeting mRNA. We initiated clinical trials of our drug candidate, which was discovered and brought into clinical trials for cancer patients by Molecure. We have made significant progress with OATD-02, a dual arginase inhibitor, and will soon begin administering the drug (at a dose of 20 mg) to a fourth cohort of patients with solid tumors. We are also seeing an increase in biomarkers suggesting a pharmacodynamic effect in the absence of significant side effects. In 2023, our focus was also on preparing for a Phase II clinical trial for OATD 01, Molecure's flagship clinical programme. We obtained FDA and then MHRA approval to initiate a Phase II clinical trial of OATD-01 in patients with pulmonary sarcoidosis. It is noteworthy that we went through the whole procedure very smoothly, which demonstrates the high quality of our research and documentation. We are very pleased to report that the first patient has started dosing in a clinical trial at a clinical site in the UK. OATD-01 is a first-in-class chitinase inhibitor developed by Molecure scientists for the treatment of sarcoidosis, which will be administered to placebo-controlled patients. We are shortly awaiting Phase II clinical trial approvals for OATD-01 from regulatory authorities in Denmark, France, Greece, Germany and Norway. Last year was also a time to continue Molecure's transformation into a cutting-edge company, applying the latest generative and predictive artificial intelligence techniques to our early drug discovery programmes, including a platform of small-molecule drugs interacting directly with mRNA targets. We achieved our first major success with Proof-of-Concept in vitro, confirming the inhibition of protein translation with compounds targeting the mRNA encoding the protein. We also adopted an approach to identifying the most promising preclinical projects, aiming to maintain a balanced portfolio of projects with strong clinical and transactional potential. The last two years have been difficult for biotech companies due to the destabilisation of the geopolitical environment, inflation and interest rates, which slowed down partnership discussions and capital raising. Despite these difficulties, thanks to investors' faith in the potential of our programmes, we successfully completed a capital round and raised approximately PLN 50 million in growth funding from investors through a share issue. Our clinical development is incurring increasing costs, but we have our own funding and awarded grants that will allow us to continue with both clinical projects and those with the best earlier stage of development until at least early 2025. In parallel, we are applying for new grants to support projects and IT and computational methods, which will allow us to accelerate the development of programmes. We are confident that pursuing our mission will open up new treatment opportunities for patients and provide a significant return on investment for shareholders. We thank you for your trust to date and encourage you to continue working with us.” - said Marcin Szumowski, CEO of Molecure S.A. Presentation for investors The Company's presentation to investors will take place on 10th April 2024, at 2 pm (CEST) in an online meeting format, under the link: Selected key developments in 2023 and up to the date of publication of the financial report Strategy Update The Company's main strategic objectives in the area of R&D and business development are: Continued intensive clinical development of two key projects: completion of a Phase II study for OATD-01 in sarcoidosis and completion of a Phase I clinical trial for OATD-02 in oncology patients, with possible expansion to additional indications and combination therapies Further development of early preclinical stage projects, including the identification of 1-2 advanced lead compounds (candidates for preclinical development) and the introduction of a further programme to the clinical trial stage Accelerate the development of a breakthrough platform of small-molecule drugs targeting mRNA, including the achievement of in vitro PoC and the selection of lead molecules Increasing the efficiency of drug discovery processes (by reducing time and cost and lowering the risk of failure) through investment in machine learning and generative artificial intelligence (GenAI) technology Conclusion of at least 1 high-value partnership agreement for at least one clinical-stage project, as well as the establishment of a number of commercial collaborations, including profit-sharing, for earlier-stage programmes Investment expenditures, among others, for the implementation of the objectives set out in the Strategy in the period from 2024 to the end of 2025 have been set at approximately PLN 150 million. Successful completion of a public offering (SPO) Molecure has successfully raised gross proceeds of approximately PLN 50m from its public share offering from existing and new shareholders The funds raised from the SPO and expected grants will be used to fund the Company's growth plans and continue to build a balanced portfolio of breakthrough therapies including first-in-class drugs, including the completion of a Phase II study of OATD-01 in sarcoidosis and the completion of a Phase I clinical trial of OATD-02 in oncology patients, with the potential to expand into additional indications and combination therapies Phase II clinical trial approvals for OATD-01 and initiation of the trial Molecure has received approval from the US Food and Drug Administration (FDA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA) to conduct a Phase II clinical trial for OATD-01 in the US and UK. The proof-of-concept Phase II study is the first to administer the drug to patients with pulmonary sarcoidosis. The world's first patient administration of chitotriosidase inhibitor 1 (CHIT1) or placebo took place in March this year at the Royal Infirmary in Edinburgh. As part of the trial, patients will take a fixed dose of 25 mg OATD-01 or placebo daily in tablet form for 12 weeks Molecure has reapplied for permission to conduct a Phase II clinical trial of OATD-01 in EU countries: Denmark, France, Greece, Germany and Norway. In the previous coordinated assessment procedure, a refusal decision was issued by the Polish Office for Registration of Medicinal Products, Medical Devices and Biocidal Products, and the effect of the refusal was that, despite receiving approval from the ethics committees in Greece, Germany, France, Denmark, Poland and Norway, it was not possible to conduct the clinical trial in the requested EU countries, as Poland was reported in the application as the rapporteur country. In the re-proceeding, Molecure proposed Denmark as a rapporteur country in the application assessment process. Phase I clinical trial OATD-02 Achieving significant progress in the clinical development of OATD-02, an oral, potent and selective first-in-class arginase (ARG1 and ARG2) inhibitor, in cancer therapies March 2023 saw the administration of OATD-02 to the first oncology patient in a Phase I clinical trial to evaluate the safety, tolerability and preliminary efficacy of OATD-02 in patients with advanced and/or metastatic solid tumors Continued systematic dose escalation and planned imminent commencement of administration in a fourth cohort of patients with solid tumors, to determine the maximum tolerated dose and recommended dose for the next phase of the study Development of the mRNA platform Molecure confirms the efficacy of small molecules in inhibiting the translation of pathogenic proteins achieving a key milestone in the development of a breakthrough platform of small-molecule drugs targeting mRNAs In vitro proof-of-concept (PoC) assay confirms inhibition of protein translation with compounds targeting the mRNA encoding the protein The PoC stage for the first molecule developed in the mRNA platform provides evidence of the effectiveness of Molecure's strategy to identify mRNA-binding compounds with therapeutic potential The success achieved in the development of the mRNA platform increases the likelihood of signing a collaboration agreement with partners working in this area Receipt of funding for a project to develop small-molecule drugs directly interacting with mRNA under the SMART pathway of the FENG programme (European Funds for a Modern Economy). The amount of funding is approximately PLN 32.5 million with a total project budget of approximately PLN 51.5 million. The project will be implemented between 2023 and 2028 Business Development In 2023, Molecure representatives held a total of 107 meetings with biotech industry representatives in Europe and the US (Basel, London, Boston, Munich), mainly with potential partners, investors and global pharmaceutical companies. OATD-01 (37 meetings), OATD-02 (25 meetings) and USP7 (23 meetings) attracted the most interest. As a result of the meetings held, the Company signed 11 non-disclosure agreements (CDAs) with interested companies. This year, representatives of the Company were present at the BIO-Europe conference in Barcelona, which took place from 18-20 March Key organisational changes in 2023 relevant to the growth and advancement of research programme Appointment of Dr Samson Fung - Chief Medical Officer to the Company's Board of Directors to support the clinical development of OATD-01 and OATD-02 Dr Zbigniew Zasłona has been promoted to Chief Scientific Officer from his previous role as VP Research Biology. Dr Zasłona remains a member of the Board of Directors of Molecure Summary of financial data in 2023 Operating income of PLN 1.3 million, up from PLN 1.6 million in 2022. Operating expenses were PLN 23.4 million, up PLN 4.8 million from 2022, due to the progressive advancement of the Company's programmes and pipeline expansion. Increase mainly due to an increase in salaries and wages, increasing early-stage research costs and third-party service costs Net loss in 2023 amounted to PLN 18.3 million compared to a net loss of PLN 15.3 million in the same period in 2022 As at 31 December 2023, Molecure had cash and cash equivalents of nearly PLN 64 million. As at the date of publication of the Annual Report (29 March 2024), the Company has approximately PLN 54 million at its disposal - the Company has a stable capital position necessary to continue to finance the Company's dynamic growth. In addition, contracted grant funding for the coming years amounts to PLN 32.5 million. The Company plans to obtain further grants for projects within the framework of the pipeline developed so far. *** About Molecure S.A. Molecure S.A. is a biotechnology company that discovers and develops drugs to the clinical stage, using its own unique expertise in medicinal chemistry and biology to search for and develop first-in-class small-molecule drugs that, through direct modulation of previously unexplored protein and RNA targets, could provide therapies for many incurable diseases. Molecure has generated a diverse portfolio of seven distinct programmes with the support of leading academic research institutions around the world, including Yale University, Rutgers University, the Flemish Institute for Biotechnology (VIB) in Ghent, the University of Michigan and the International Institute of Molecular and Cell Biology in Warsaw (MIBMiK). The most advanced drug candidate developed by Molecure is OATD-01, a first-in-class CHIT1 inhibitor for the treatment of interstitial lung diseases such as sarcoidosis and idiopathic pulmonary fibrosis, which is ready to enter Phase II clinical trials. The Phase II study in sarcoidosis patients started in Q4 2023 in the US and the UK and will also continue in the EU and Norway, after gaining the appropriate regulatory approval. The second drug candidate is OATD-02, an oral, selective, first-in-class, dual arginase inhibitor (ARG1 and ARG2) for the treatment of cancer, whose Phase I clinical trial has begun with first patient administration in Q1 2023. Molecure's headquarters and laboratories are located in Warsaw and Lodz. The company is listed on the Warsaw Stock Exchange (ticker: MOC). Detailed information can be found on: LinkedIn: Molecure | Twitter: @molecure_sa | YouTube: Molecure SA