Zamaporvint

On April 5, 2024, the final results of the first-in-human study of the porcupine (PORCN) inhibitor zamaporvint (RXC004) in patients with advanced solid tumors were reported in 2024 AACR. RXC-004's R&D ProgressRXC-004 is a small molecule drug that targets the protein PORCN. RXC-004 has shown potential in treating various therapeutic areas, including neoplasms, digestive system disorders, endocrinology and metabolic diseases, and hemic and lymphatic diseases. According to the Patsnap Synapse, RXC-004 is currently in Phase 2, which is the highest phase of clinical development globally. And the clinical trial distributions for RXC-004 are primarily in the United States, United Kingdom and Australia. The key indication is Metastatic Microsatellite Stable Colorectal Carcinoma.  Detailed Clinical Result of RXC-004This non-randomized, sequential assignment, open-labeled clinical study (NCT03447470) was aimed to evacuate the efficacy and safety of RXC-004 in patients with advanced solid tumors.   In this study, module 1 investigated continuous monotherapy dosing, Module 2 investigated combination with nivolumab 480mg s.c. Q4W and Module 3 investigated intermittent monotherapy dosing (2 weeks on 1 week off). Patients received denosumab 120 mg s.c Q4W to prevent loss of bone mineral density (BMD), a known effect of Wnt inhibition. The primary objectives were safety and tolerability. Secondary objectives were PK and RECIST response. PD markers included on-treatment changes in: Wnt pathway and fibrosis biomarkers in paired skin biopsies; circulating immune subsets by flow cytometry; cytokine changes by Luminex, and changes in circulating-tumor DNA (ctDNA) levels by ddPCR. The result showed that in module 1, 25 patients (pts) with unselected advanced solid tumors (AST) received RXC004 between 0.5mg and 10mg QD. The RP2D was 2mg QD; at this dose the most common AEs were fatigue, diarrhoea, dysgeusia and decreased appetite; no grade 4/5 AEs or bone fragility events were reported whilst 1 pt had a RXC004-related grade 3 AE, 3 pts (50%) had a dose interruption and 1 had a dose reduction. The median T1/2 of RXC004 was 14.5h. 5 pts with upstream Wnt pathway activated tumors had stable disease (SD), in 1 case for 26 weeks with sustained reduction in ctDNA and no BMD loss. In Module 2, 14 patients with unselected AST received RXC004 at 1mg QD or 1.5mg QD with nivolumab. The AE profile was similar to Module 1. The RP2D was 1.5mg QD; at this dose 3 pts had a RXC004-related grade 3 AE, 6 pts (75%) had a dose interruption and 3 had a dose reduction, and 4 pts had SD. PK was similar to Module 1. In Module 3, 7 patients with upstream Wnt-activated AST received RXC004 2mg QD, 2 weeks on, 1 week off. The AE profile was similar to Modules 1 and 2. There were no RXC004-related Grade 3 AEs, 2 pts (29%) had a dose interruption and there were no dose reductions. PK and PD analyses confirmed time off target. 2 pts had SD; 1 pt with RSPO fusion small intestinal adenocarcinoma remained on treatment for 36 weeks, with sustained reduction in ctDNA and no BMD loss. It can be concluded that in patients with AST, RXC004 was safe and tolerated at doses up to 2mg QD as monotherapy and 1.5mg QD in combination with nivolumab, demonstrating differential disease control in upstream Wnt pathway activated tumours. Denosumab prevented loss of BMD. Intermittent RXC004 dosing was well tolerated with no detrimental impact on efficacy. How to Easily View the Clinical Results Using Synapse Database?If you want to know the other clinical results of popular conferences, please lick on the “Clinical Results” on the homepage of Patsnap Synapse, which provides multi-dimensional screening and filtering of drugs, indications, targets, companies, result evaluation, release date, popular conferences, etc. to help you quickly locate the data you need.  Select the clinical meeting you are interested in, such as ESMO. In the results, you can quickly locate the data you want to view by indication, phase and drug name.  A single result clearly shows important information such as registration number, phase, indication, Sponsor/Collaborator, biomarker, Trial number, dosing regimen and more.  If you would like to view more information about this result, you can go to the result detail page by clicking on the title.  Above the headings, we provide the original source of the outcome data. The basic information is supplemented with more information beyond the list, such as company, study. design, etc.  In the important Outcome Measures section, we provide both list and flowchart forms, which are convenient for you to overview the comparison group information and core indicator data.  Finally, if you need to download these results, you can conveniently check the check boxes on the left side of the list, or directly click the "Export" button to download the data for personalized analysis and file sharing. Click on the image below to embark on a brand new journey of drug discovery!

Redx to receive $10 million upfront; potential for up to $870 million in development, regulatory and sales milestone payments in addition to royalties on future net salesPreclinical program expands Jazz's pipeline of targeted oncology therapies DUBLIN and ALDERLEY PARK, United Kingdom, Feb. 7, 2024 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) and Redx Pharma plc (AIM: REDX) today announced that the companies have signed a definitive agreement under which Jazz will acquire Redx's KRAS (Kirsten rat sarcoma virus) inhibitor program. Jazz and Redx will collaborate to advance candidates through IND-enabling studies; Jazz will be responsible for all clinical development, regulatory, manufacturing and commercialization activities. Continue Reading "KRAS is a well-validated oncology target and there remains a high unmet need for innovation in this area based on challenges in developing molecules to target specific KRAS mutations. Redx has discovered a number of preclinical KRAS candidates and we plan to leverage our collective oncology development expertise to identify and advance the most promising molecules toward the clinic," said Robert Iannone, M.D., M.S.C.E., executive vice president, global head of research and development of Jazz Pharmaceuticals. "This transaction further expands our early-stage oncology pipeline, and we are excited to explore novel approaches to improving treatment options for cancer patients." Lisa Anson, Chief Executive Officer of Redx, commented: "Once again, our distinguished expertise in medicinal chemistry has been recognized and we are pleased to secure another strategic transaction with Jazz, with whom we have a track record of collaboration. This agreement will allow us to collaborate on the advancement of novel KRAS inhibitors and we look forward to supporting Jazz in progressing these candidates through IND-enabling studies. Partnerships remain a key pillar of our corporate strategy, allowing us to advance what we believe are differentiated molecules in areas of high unmet need, while also creating long-term shareholder value through non-dilutive funding, with the upfront milestone payment from this agreement extending our current cash runway into 2025." Transaction Terms Under the terms of the agreement, Jazz will make an upfront payment to Redx of $10 million for all rights, patents, title and interest relating to Redx's proprietary KRAS inhibitor program, which includes G12D selective and pan-KRAS molecules. Redx is eligible to receive up to $870 million in development, regulatory and commercial milestone payments from Jazz, with the next milestone being an IND clearance from this program from the U.S. Food and Drug Administration. Redx is also eligible for tiered, mid-single digit percentage royalties based on any future net sales. As part of a separate collaboration agreement, signed in parallel, Jazz will pay Redx to perform research and preclinical development activities with the goal of completing IND-enabling studies for both KRAS profiles. About KRAS Inhibitors Rat sarcoma virus (RAS) is the most frequently mutated oncogene across different cancer types, with KRAS mutations accounting for approximately 85% of these mutated oncogenes. Therefore, KRAS inhibitors targeting multiple commonly occurring mutations may offer a treatment option for large segments of colorectal, pancreatic and lung cancer patients who currently have limited treatment options. Developing orally bioavailable agents that generate optimized target coverage is a key opportunity for the next wave of KRAS-targeting agents that act beyond the G12C mutation. About Jazz Pharmaceuticals plc Jazz Pharmaceuticals plc (NASDAQ: JAZZ) is a global biopharmaceutical company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing life-changing medicines for people with serious diseases—often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines and novel product candidates, from early- to late-stage development, in neuroscience and oncology. Within these therapeutic areas, we are identifying new options for patients by actively exploring small molecules and biologics, and through innovative delivery technologies and cannabinoid science. Jazz is headquartered in Dublin, Ireland and has employees around the globe, serving patients in nearly 75 countries. Please visit for more information. About Redx Pharma Plc Redx Pharma (AIM: REDX) is a clinical-stage biotechnology company focused on the discovery and development of novel, small molecule, targeted therapeutics for the treatment of fibrotic disease, cancer and the emerging area of cancer-associated fibrosis. Redx aims to progress its programmes to clinical proof of concept before evaluating options for further development and potential value creation. The Company's lead fibrosis product candidate, the selective ROCK2 inhibitor, zelasudil (RXC007), is in development for interstitial lung disease and is undergoing a Phase 2a trial for idiopathic pulmonary fibrosis (IPF) with topline data expected in H1 2024. The Company's second fibrosis candidate, RXC008, a GI-targeted ROCK inhibitor for the treatment of fibrostenotic Crohn's disease, is progressing towards the clinic with a Clinical Trial Application (CTA) submitted during Q4 2023.Redx's lead oncology product candidate, the Porcupine inhibitor RXC004, being developed as a targeted treatment for Wnt-ligand dependent cancers, is expected to report anti-PD-1 combination Phase 2 data during the first half of 2024, following which Redx will seek a partner for ongoing development. The Company has a strong track record of discovering new drug candidates through its core strengths in medicinal chemistry and translational science, enabling the Company to discover and develop differentiated therapeutics against biologically or clinically validated targets. The Company's accomplishments are evidenced not only by its wholly-owned clinical-stage product candidates and discovery pipeline, but also by its strategic transactions, which includes the sale of pirtobrutinib (RXC005, LOXO-305), the only non-covalent or reversible BTK inhibitor now approved by the US FDA, and transactions with both AstraZeneca and Jazz Pharmaceuticals. To subscribe to Email Alerts from Redx, please visit: . Jazz Investor Contact: Andrea N. Flynn, Ph.D. Vice President, Head, Investor Relations Jazz Pharmaceuticals plc [email protected] Ireland +353 1 634 3211 U.S. +1 650 496 2717 Jazz Media Contact: Kristin Bhavnani Head of Global Corporate Communications Jazz Pharmaceuticals plc [email protected] Ireland +353 1 637 2141 U.S. +1 215 867 4948 Redx Contacts: Caitlin Pearson Head of Communications Redx Pharma UK +44 (0)1625 469 918 Matt Davis/ Adam Dawes SPARK Advisory Partners (Nominated Adviser) UK +44 (0)203 368 3550 Claes Spång/ Satheesh Nadarajah/ David Wilson WG Partners LLP (Joint Broker) UK +44 (0)203 705 9330 Rupert Dearden/Freddy Crossley/Emma Earl Panmure Gordon (UK) Limited (Joint Broker) UK +44 (0)207 886 2500 Simon Conway/Ciara Martin FTI Consulting UK +44 (0)203 727 1000 SOURCE Jazz Pharmaceuticals plc