A Phase 1b/2 Trial of PXS-5505 Combined With First Line Atezolizumab Plus Bevacizumab For Treating Patients With Unresectable Hepatocellular Carcinoma

This trial will assess the safety and tolerability of PXS-5505 incorporating first-line combination therapy Atezolizumab and Bevacizumab in unresectable or metastatic hepatocellular carcinoma. Phase 2 will assess the efficacy of this combination therapy in unresectable or metastatic hepatocellular carcinoma.

Start Date20 Sep 2022

Sponsor / Collaborator

University of Rochester

NCT04676529

/ CompletedPhase 1/2

A Phase 1/2a Study to Evaluate Safety, Pharmacokinetic and Pharmacodynamic Dose Escalation and Expansion Study of PXS-5505 in Patients With Primary, Postpolycythemia Vera or Post-essential Thrombocythemia Myelofibrosis

This study will be an open-label phase 1/2a study to evaluate the safety and tolerability of PXS-5505 in patients with primary, postpolycythemia vera (PV) or post-essential thrombocythemia (ET) myelofibrosis.

Start Date18 Feb 2021

Sponsor / Collaborator

Syntara Ltd.

100 Clinical Results associated with PXS-5505

100 Translational Medicine associated with PXS-5505

100 Patents (Medical) associated with PXS-5505

Literatures (Medical) associated with PXS-5505

15 May 2026·CANCER RESEARCH

LOX Inhibition Disrupts a Collagen–Integrin–MYC Axis to Suppress Progression of Invasive Lobular Carcinoma

Article

Author: Battista, Laura ; Brisken, Cathrin ; Pezot, Solène ; Okines, Alicia F.C. ; Turner, Nicholas C. ; Hughes, Flavia ; Brough, Rachel ; Lord, Christopher J. ; Zhang, Yueyun ; Iravani, Marjan ; Nicholas, Anya A. ; Ronchi, Carlos ; Zaman, Khalil ; Roumeliotis, Theodoros I. ; Isacke, Clare M. ; Yang, William W. ; Jarolimek, Wolfgang ; Kemp, Harriet ; Liu, Yanbo ; Jouny, Samuel ; Perryman, Lara ; Flaherty, Renée L. ; Stravodimou, Athina ; Ambrosini, Giovanna ; Becker, Sarah ; Fiche, Maryse ; Howard, Beatrice A. ; Sflomos, George ; Padda, Harveena ; Quinn, Hazel M. ; Choudhary, Jyoti S. ; Haider, Syed ; Marlow, Rebecca ; Ziehme, Amelie ; Sanz-Moreno, Victoria

Abstract:

Invasive lobular carcinoma (ILC) accounts for 15% of breast cancers yet lacks specific therapy because ILCs are underrepresented in clinical trials and preclinical models are lacking. In this study, we established intraductal xenograft models to test whether the clinical pan-lysyl-oxidase inhibitor PXS-5505, now in phase I/IIa trials for myelofibrosis, can exploit the collagen-rich matrix dependency in ILC created by CDH1 loss. PXS-5505 remodeled fibrillar collagen and halted tumor expansion and metastatic seeding across estrogen receptor–positive and triple-negative models without systemic toxicity. Genome-wide CRISPR screens revealed ITGAV and ITGB5 as synthetic lethal partners of CDH1, and LOX inhibition downregulated their expression, together with MYC, NF-κB, and AP-1 transcriptional programs. Collagen fiber density/alignment and MYC/AP-1 gene signatures served as pharmacodynamic readouts of drug activity. These data uncover a tractable extracellular matrix–integrin–MYC axis in ILC and nominate PXS-5505, alone or with endocrine therapy, for window-of-opportunity trials in this understudied breast cancer subtype.

Significance::

A clinical stage LOX inhibitor slows tumor progression and alters multiple molecular endpoints in invasive lobular carcinoma, providing a translatable therapeutic strategy for this frequent breast cancer subtype currently lacking specific therapies.

01 Mar 2026·iScience

Matrix structure and microenvironment dynamics correlate with chemotherapy response in ovarian cancer

Article

Author: Maniati, Eleni ; Hartlebury, Joseph ; Joy, Joash D ; Bryan-Ravenscroft, Rachel C ; Manchanda, Ranjit ; Berlato, Chiara ; Marshall, John F ; Malacrida, Beatrice ; Elorbany, Samar ; Di Federico, Erica ; Jarolimek, Wolfgang ; Perryman, Lara ; Balkwill, Frances R ; Laforêts, Florian ; Kotantaki, Panoraia

Elevated extracellular matrix (ECM) associates with chemo-resistance and poor prognosis in cancer. Modifying the ECM could enhance response to chemotherapy. We measured chemotherapy-induced changes in the tumor microenvironment (TME) of two high-grade serous ovarian cancer (HGSOC) mouse models with different chemo-sensitivity. Carboplatin/paclitaxel treatment triggered dynamic transcriptional changes in immune and ECM-related pathways, and analyses of the ECM structure revealed modifications in the chemo-sensitive tumors. These changes, observed over twenty days post-chemotherapy, had relevance to HGSOC patient responses. Integrating transcriptomics with ECM structure metrics, we identified ECM targets, including lysyl oxidase (LOX), that might enhance effects of chemotherapy. Given alone or with chemotherapy, a pan-LOX inhibitor, PXS-5505, modulated fibroblast and immune cell distribution and decreased tumor stiffness in the less-responsive mouse model. Moreover, treatment with PXS-5505 before chemotherapy enhanced the therapeutic response. We conclude that pretreatment with ECM targeting agents may improve chemotherapy efficacy by altering ECM structure and immune responses.

Hepatology Communications

Pan-lysyl oxidase inhibition disrupts fibroinflammatory tumor stroma, rendering cholangiocarcinoma susceptible to chemotherapy

Article

Author: Yeh, Jen Jen ; Chávez Villa, Mariana ; Dave, Yatee A. ; De Las Casas, Luis ; Georger, Mary ; Hilty, Bailey K. ; Byrne, Matthew M. ; Burchard, Paul R. ; Marin, Chelsea ; Miller, Christine ; Jewell, Rachel ; Dale, Benjamin S. ; Linehan, David C. ; Ye, Jian ; Ruffolo, Luis I. ; Loria, Anthony ; Ullman, Nicholas A. ; Hernandez-Alejandro, Roberto ; Jarolimek, Wolfgang ; Perryman, Lara ; Belt, Brian A.

Background::

Cholangiocarcinoma (CCA) features highly desmoplastic stroma that promotes structural and functional resistance to therapy. Lysyl oxidases (LOX, LOXL1–4) catalyze collagen cross-linking, thereby increasing stromal rigidity and facilitating therapeutic resistance. Here, we evaluate the role of lysyl oxidases in stromal desmoplasia and the effects of pan-lysyl oxidase (pan-LOX) inhibition in CCA.

Methods::

Resected CCA and normal liver specimens were analyzed from archival tissues. Spontaneous and orthotopic murine models of intrahepatic CCA (iCCA) were used to assess the impact of the pan-LOX inhibitor PXS-5505 in treatment and correlative studies. The functional role of pan-LOX inhibition was interrogated through in vivo and ex vivo assays.

Results::

All 5 lysyl oxidases are upregulated in CCA and reduced lysyl oxidase expression is correlated with an improved prognosis in resected patients with CCA. Spontaneous and orthotopic murine models of intrahepatic cholangiocarcinoma upregulate all 5 lysyl oxidase isoforms. Pan-LOX inhibition reversed mechanical compression of tumor vasculature, resulting in improved chemotherapeutic penetrance and cytotoxic efficacy. The combination of chemotherapy with pan-LOX inhibition increased damage-associated molecular pattern release, which was associated with improved antitumor T-cell responses. Pan-LOX inhibition downregulated macrophage invasive signatures in vitro, rendering tumor-associated macrophages more susceptible to chemotherapy. Mice bearing orthotopic and spontaneously occurring intrahepatic cholangiocarcinoma tumors exhibited delayed tumor growth and improved survival following a combination of pan-LOX inhibition with chemotherapy.

Conclusions::

CCA upregulates all 5 lysyl oxidase isoforms, and pan-LOX inhibition reverses tumor-induced mechanical forces associated with chemotherapy resistance to improve chemotherapeutic efficacy and reprogram antitumor immune responses. Thus, combination therapy with pan-LOX inhibition represents an innovative therapeutic strategy in CCA.

News (Medical) associated with PXS-5505

03 Sep 2025

·www.prnewswire.com

Myelofibrosis Market is Going to Grow at a CAGR of 9% in the Next Ten Years (2025-2034) | DelveInsight

The launch of emerging therapies like BESREMi (PharmaEssentia and AOP Orphan Pharmaceuticals), INCB057643 (Incyte), XPOVIO (Karyopharm Therapeutics), RYTELO (Geron), REBLOZYL (Bristol Myers Squibb), Navtemadlin (Kartos Therapeutics), Pelabresib (Novartis), and others is going to shift the myelofibrosis market. LAS VEGAS, Sept. 3, 2025 /PRNewswire/ -- DelveInsight's Myelofibrosis Market Insights report includes a comprehensive understanding of current treatment practices, emerging myelofibrosis drugs, market share of individual therapies, and current and forecasted myelofibrosis market size from 2020 to 2034, segmented into leading markets (the US, EU4, UK, and Japan). Myelofibrosis Market Summary The total myelofibrosis treatment market size in the leading markets (the US, EU4, UK, and Japan) was USD 2.2 billion in 2024. The United States accounts for the largest market size of myelofibrosis, in comparison to EU4 (Germany, Italy, France, and Spain) and the UK, and Japan. Based on DelveInsight's assessment in 2024, the 7MM had approximately 56K patient pool (prevalent cases) of myelofibrosis. Key myelofibrosis companies, including PharmaEssentia, AOP Orphan Pharmaceuticals, Incyte, Karyopharm Therapeutics, Geron, Bristol Myers Squibb, Kartos Therapeutics, Novartis, Merck, Telios Pharma, Ryvu Therapeutics, Sumitomo Pharma, Syntara, Disc Medicine, Menarini Group, and others, are actively working on innovative myelofibrosis drugs. Some of the key myelofibrosis therapies in clinical trials include BESREMi (ropeginterferon alfa-2b-njft/P-1101), INCB057643, XPOVIO (NEXPOVIO/selinexor/KPT-330), RYTELO (imetelstat), REBLOZYL (luspatercept/ACE-536), Navtemadlin (KRT-232), Pelabresib (DAK539), Bomedemstat (IMG-7289/MK-3543), TL-895, RVU120 ( SEL-120), TP-3654 (nuvisertib), SNT-5505 (PXS-5505), DISC-0974, ELZONRIS (tagraxofusp/SL-401), and others. These novel myelofibrosis therapies are anticipated to enter the myelofibrosis market in the forecast period and are expected to change the market. By 2034, among all the therapies, the highest revenue is expected to be generated by OJJAARA/OMJJARA. Discover which myelofibrosis therapies are expected to grab the largest market share @ Myelofibrosis Market Report Key Factors Driving the Growth of the Myelofibrosis Market Novel Therapies Drive Myelofibrosis Treatment Advancements Currently, four JAK inhibitors have been approved by the US FDA for the treatment of myelofibrosis, including JAKAFI (ruxolitinib), INREBIC (fedratinib), VONJO (pacritinib), and OJJAARA (momelotinib). No drug therapy can cure myelofibrosis. As myelofibrosis primarily affects older adults, stem cell transplantation is not a treatment option for most myelofibrosis patients. The launch of another JAK inhibitor, BESREMi, will further change the dynamics of the myelofibrosis market. Rich clinical pipeline and emerging non-JAK approaches driving the myelofibrosis landscape Beyond JAK inhibitors, active clinical development includes BET inhibitors (Incyte's INCB057643; Novartis' Pelabresib), XPO1 inhibitor (Karyopharm Therapeutics' XPOVIO), Telomerase inhibitor (Geron's RYTELO), MDM2 protein inhibitor (Kartos Therapeutics' Navtemadlin), Tyrosine kinase inhibitors (Telios Pharma's TL-895), PIM1 kinase inhibitor (Sumitomo Pharma's TP-3654), LOX inhibitor (Syntara's SNT-5505), and others, raising market potential by promising new label expansions, second-line options, and higher-value therapies should any prove to be disease-modifying. An aging population & increasing incidence with better survival Myelofibrosis primarily affects older adults; population aging increases the absolute number of patients. As treatments improve symptoms and (in some cases) survival, prevalent patient pools grow, supporting longer-term market demand. In the US in 2024, the 70+ years of age group had the highest number of cases, accounting for approximately 60% of the total prevalent cases, while the ≤39 years of age group accounted for just ~2%. Diagnostic & genomic testing improvements are driving myelofibrosis patient pool, leading to the growth of myelofibrosis market Wider use of molecular profiling (JAK2, CALR, MPL, and others) enables earlier and more accurate MF diagnosis and better patient stratification for targeted therapies. This both expands the diagnosed population and helps match patients to appropriate, often higher-value, treatments. Myelofibrosis Market Analysis JAK inhibitors have become the cornerstone of treatment for patients with myelofibrosis, offering significant benefits such as spleen reduction, symptom relief, and improved quality of life, which can also extend survival in those with advanced disease. All approved JAK inhibitors primarily target JAK2, particularly the wild-type form, but they differ in their activity against other JAK family members. For instance, JAKAFI myelofibrosis inhibits both JAK1 and JAK2; INREBIC selectively inhibits JAK2 while sparing JAK1 and also affects FLT3 and other targets; VONJO myelofibrosis inhibits JAK2 while sparing JAK1 but additionally impacts FLT3, IRAK, and ACVR1; and OJJAARA, approved through a different pathway, targets JAK1/JAK2 and ACVR1, mainly for myelofibrosis patients with anemia. These varying mechanisms lead to distinct patient outcomes. JAKAFI myelofibrosis continues to see strong demand and is expected to grow further, maintaining its position as the standard of care in myelofibrosis. Myelofibrosis will remain the largest segment of JAKAFI's patient population until polycythemia vera cases eventually increase. However, market growth may be limited due to patent expirations of key therapies, with JAKAFI patents set to expire in 2027 for Novartis and 2028 for Incyte, presenting potential opportunities for competitors. In response, Incyte is exploring combination trials with novel drugs to extend JAKAFI's therapeutic lifespan. Learn more about the treatment options for myelofibrosis @ Myelofibrosis Therapy The myelofibrosis treatment landscape offers significant opportunities for innovation, focusing on several key areas: Treating Lower-Risk Patients: Developing safe and effective therapies for patients with lower-risk myelofibrosis, allowing for earlier and more favorable interventions. Enhancing First-Line Treatments: Improving existing first-line therapies for intermediate- to high-risk patients through novel drugs or combination strategies. Managing Cytopenia: Developing targeted therapies to address cytopenia, a significant unmet need in patient care. Myelofibrosis Competitive Landscape The myelofibrosis clinical trial pipeline includes several drugs in mid- and late-stage development that are expected to enter the market during the forecast period. The emerging landscape offers a diverse range of therapeutic alternatives for treatment, including XPOVIO (Karyopharm Therapeutics), Elitracet (Takeda/Keros Therapeutics), Navtemadlin (Kartos Therapeutics), Pelabresib (Novartis), INCB057643 (Incyte), Bomedemstat (Merck), and others, all of which are used in various lines of treatment. The expected launch of these therapies is expected to have a further positive impact on the market. INCB57643 is an orally administered small molecule. Bromodomain and extra-terminal (BET) proteins act as epigenetic readers that control the expression of key oncoproteins implicated in the development of myelofibrosis and other hematologic malignancies, including B-lymphoma-2, nuclear factor kappa, and c-Myc. In a prior Phase I/II clinical trial, the oral BET inhibitor INCB057643, tested both as a monotherapy and in combination with ruxolitinib, demonstrated favorable tolerability and promising clinical activity in patients with advanced cancers. In its Q2 2025 financial report, the company announced that the combination of INCB057643 with ruxolitinib and INCB57643 (a JAK1/JAK2 and BET inhibitor) is currently being evaluated in a Phase II trial for myelofibrosis. RYTELO (imetelstat) is an investigational telomerase inhibitor that targets telomerase, selectively eliminating malignant stem and progenitor cells in the bone marrow, which drive diseases like myelodysplastic syndromes (MDS) and myelofibrosis. By blocking the proliferation of these malignant cells, imetelstat supports the recovery of healthy bone marrow and blood cell production and has shown disease-modifying effects and clinical benefits in Phase III trials for myelofibrosis. This mechanism sets imetelstat apart from other approved or investigational therapies for these blood cancers. In January 2025, Geron reported achieving 75% enrollment in the Phase III IMpactMF trial, which is comparing imetelstat to Best Available Therapy (BAT) in intermediate-2 or high-risk myelofibrosis patients who have relapsed or are refractory to JAK inhibitor treatment. Elritercept is a late-stage investigational activin inhibitor aimed at treating anemia associated with hematologic malignancies, including MDS and myelofibrosis. It is currently undergoing Phase II evaluation in patients with myelofibrosis. In December 2024, Keros Therapeutics presented updated data from this ongoing Phase II trial at ASH 2024. Additionally, in December 2024, Takeda announced an exclusive licensing agreement with Keros to further develop, manufacture, and commercialize elritercept globally, excluding mainland China, Hong Kong, and Macau. The anticipated launch of these emerging myelofibrosis therapies are poised to transform the myelofibrosis market landscape in the coming years. As these cutting-edge myelofibrosis therapies continue to mature and gain regulatory approval, they are expected to reshape the myelofibrosis market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. To know more about new myelofibrosis treatment, visit @ Myelofibrosis Treatment Market Recent Developments in the Myelofibrosis Market In July 2025, Incyte announced that the Phase I data in patients with myelofibrosis as monotherapy and in combination with ruxolitinib are anticipated in the second half of 2025. In June 2025, QIAGEN and Incyte announced a new global collaboration to develop a novel diagnostic panel to support Incyte's extensive portfolio of investigational therapies for patients with myeloproliferative neoplasms (MPNs), including Incyte's monoclonal antibody INCA033989. In January 2025, Karyopharm Therapeutics stated that it expects to report top-line results from the Phase III SENTRY trial in the second half of 2025, which could represent a potentially transformative opportunity to establish a new treatment paradigm in myelofibrosis. What is Myelofibrosis? Myelofibrosis is a rare blood cancer marked by the accumulation of scar tissue, or 'fibrosis', in the bone marrow. This excess scar tissue prevents the bone marrow from producing enough healthy blood cells. It belongs to a group of blood cancers called 'myeloproliferative neoplasms (MPNs)', in which the blood cells produced by the bone marrow grow and function abnormally. When myelofibrosis arises independently, without being caused by another bone marrow disorder, it is referred to as primary myelofibrosis. In other cases, it can develop from another MPN, such as polycythemia vera or essential thrombocythemia. When this occurs, it is called secondary myelofibrosis, sometimes specifically termed post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis. Myelofibrosis Epidemiology Segmentation The myelofibrosis epidemiology section provides insights into the historical and current myelofibrosis patient pool and forecasted trends for the leading markets (the US, EU4, UK, and Japan). It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The myelofibrosis market report proffers epidemiological analysis for the study period 2020–2034 in the leading markets (the US, EU4, UK, and Japan) segmented into: Total Prevalent Cases of Myelofibrosis Type-specific Cases of Myelofibrosis Myelofibrosis Cases Based on Risk Stratification Age-specific Prevalent Cases of Myelofibrosis Myelofibrosis Cases Based on Molecular Alterations Download the report to understand which factors are driving myelofibrosis epidemiology trends @ Myelofibrosis Treatment Drugs Scope of the Myelofibrosis Market Report Myelofibrosis Therapeutic Assessment: Myelofibrosis current marketed and emerging therapies Myelofibrosis Market Dynamics: Conjoint Analysis of Emerging Myelofibrosis Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Myelofibrosis Market Unmet Needs, KOL's views, Analyst's views, Myelofibrosis Market Access and Reimbursement Discover more about myelofibrosis drugs in development @ Myelofibrosis Clinical Trials Table of Contents Related Reports JAK Inhibitors Market JAK Inhibitors Market Size, Target Population, Competitive Landscape, and Market Forecast – 2034 report delivers an in-depth understanding of the market trends, market drivers, market barriers, and key JAK inhibitors companies, including Pfizer, AbbVie, Galapagos, Sierra Oncology, Theravance Biopharma, Dizal Pharmaceutical, Aclaris Therapeutics, Celon Pharma, Incyte Corporation, Gilead Sciences, Reistone Biopharma, Jiangsu Hengrui Medicine Co., MaxiNovel Pharmaceuticals, among others. Myelofibrosis Clinical Trial Analysis Myelofibrosis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key myelofibrosis companies, including Geron Corporation, Merck, Ryvu Therapeutics SA, Morphic Therapeutic, iOnctura, Pharmaxis, Nippon Shinyaku, Active Biotech, Incyte Corporation, Sumitomo Pharma America, Inc., Cellenkos, Disc Medicine, among others. Polycythemia Vera Market Polycythemia Vera Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key polycythemia vera companies, including Incyte, Novartis, PharmaEssentia, AOP Orphan Pharmaceuticals, Protagonist Therapeutics, Merck (Imago BioSciences), Italfarmaco, Ionis Pharmaceutical, Silence Therapeutics, Perseus Proteomics, AbbVie, Johnson & Johnson Innovative Medicine, Mabwell (Shanghai) Bioscience, Disc Medicine, GluBio Therapeutics, among others. Multiple Myeloma Market Multiple Myeloma Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key multiple myeloma companies, including Sanofi, Karyopharm Therapeutics, AbbVie, Takeda Pharmaceutical, Celgene, Bristol-Myers Squibb, RAPA Therapeutics, Pfizer, Array Biopharma, Cellectar Biosciences, BioLineRx, Celgene, Aduro Biotech, ExCellThera, Janssen Pharmaceutical, Precision BioSciences, Takeda, Glenmark (Ichnos Sciences SA), Poseida Therapeutics, Molecular Partners AG, Chipscreen Biosciences, AbbVie, Genentech (Roche), Janssen Biotech, Nanjing Legend Biotech, Merck Sharp & Dohme Corp., 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 . Connect with us on LinkedIn | Facebook | Twitter Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2License out/inPhase 1Drug ApprovalClinical Result

18 Oct 2023

·www.proactiveinvestors.com.au

Pharmaxis finalises sale of MBU to Arna Pharma; shifts focus to blood-related cancer treatments

Pharmaxis Ltd (ASX:PXS, OTC:PMXSF) has completed the sale of its mannitol respiratory business (MBU) to Arna Pharma Pty Ltd. On October 3, 2023, PXS announced it would sell MBU as part of a major company restructure to focus on developing drugs to treat blood-related cancers. The restructure involves the creation of clinical stage drug development company, Syntara as well as the sale of MBU, along with changes to the company’s board of directors. Pharmaxis CEO Gary Phillips said at the time: “Over the last few years Pharmaxis has built a commanding position in lysyl oxidase biology and chemistry research. “We have collaborated with leading clinicians and scientists worldwide and forged through the early-stage studies that have given us a clinical pipeline with great potential and resulted in multiple Nature publications. “Building on this heritage and the proven capability of our discovery and development teams, the restructure announced today and the creation of Syntara enables us to focus and accelerate our clinical development programs.” Arna Pharma, an Australian pharmaceutical company involved in a global healthcare alliance, has taken charge of the day-to-day operations of the Medical Business Unit (MBU), which includes Bronchitol® and Aridol®. As per the sale agreement terms, Arna Pharma will compensate Pharmaxis for the lion's share of residual expenses incurred during the transition period, set to conclude in May 2024. The divestiture of MBU and the subsequent restructuring within Pharmaxis are anticipated to slash annual core costs by more than 60%, amounting to a saving of over US$14 million annually. Pharmaxis continues to develop its drug PXS-5505, aimed at treating myelofibrosis, a bone marrow cancer that causes a build-up of scar tissue leading to diminished production of red and white blood cells and platelets. The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to PXS-5505 for the treatment of myelofibrosis and has approved an Investigational Drug Application (IND) to proceed with a phase 1c/2 clinical trial. Recruitment for the trial began in the first quarter of 2021, with final interim data reported in July 2023. In addition to PXS-5505, Pharmaxis is also developing other drug candidates from its amine oxidase chemistry platform. These candidates target various fibrotic diseases, including kidney fibrosis, Non-alcoholic Steatohepatitis (NASH), pulmonary fibrosis and cardiac fibrosis. The platform is also focused on fibrotic scarring resulting from burns and other trauma, along with additional inflammatory diseases. Specifically, PXS-4728 is under study in collaboration with Parkinson's UK as a leading SSAO/MAOB inhibitor, intended to treat sleep disorders and slow the progression of neurodegenerative diseases such as Parkinson's by reducing neuroinflammation.

Orphan DrugClinical Study

29 Aug 2023

·www.sciencedaily.com

Drug that targets scar-like tissue in tumors shows promise for aggressive pancreatic cancer

Early laboratory results in mice show the drug PXS-5505 increases survival when combined with chemotherapy in the treatment of pancreatic ductal adenocarcinomas. Findings from the Garvan Institute of Medical Research reveal a new Australian drug that targets scar-like 'fibrotic' tissue within tumours shows promise for treating pancreatic ductal adenocarcinoma, one of the most aggressive forms of pancreatic cancer with a five-year survival rate of less than 10%. The research in mouse models showed that when given in combination with chemotherapy, the drug PXS-5505 increased survival time by more than 35%, compared to chemotherapy treatment alone. "The preclinical validation of this first-in-class anti-fibrotic drug marks a major milestone in our quest to overcome the significant challenges in treating pancreatic cancer and brings hope to patients and their families," says Associate Professor Thomas Cox, head of the Matrix & Metastasis Lab at Garvan and senior author of the study, published in the journal Nature Cancer. Potential to increase cancer survival Pancreatic cancer is often diagnosed at an advanced stage, which means that chemotherapy is often the only treatment option available. Many pancreatic cancers develop chemotherapy resistance soon after treatment starts, which contributes to the poor survival of patients. Part of this resistance is driven by tumour fibrosis -- the formation of a mesh of scar tissue-like collagen -- within and around pancreatic tumours that in turn reduces the effectiveness of chemotherapy drugs. The new drug PXS-5505, developed by Sydney-based pharmaceutical research company Pharmaxis (ASX: PXS) and currently in Phase II clinical trials for the treatment of bone marrow cancer, works by blocking a family of enzymes that are critical for the deposition of collagen into the fibrotic tissue around tumours. In collaboration with Pharmaxis, Garvan researchers found that the drug significantly reduced fibrosis in pancreatic tumours in mouse models. The combination therapy also substantially reduced the spread of the cancer to other organs, such as the liver, by 45%. "PXS-5505 returns the tumour microenvironment to a more 'normal' state by reducing fibrosis and decreasing tumour stiffness," explains Dr Jessica Chitty, Senior Research Officer at Garvan and first author of the study. "This allows chemotherapy drugs to penetrate the tumours more easily, work more effectively, and destroy more cancer cells." "PXS-5505 shows real potential to improve chemotherapy for patients," says Associate Professor Cox. "We are now in the process of progressing this work toward clinical trials that will evaluate this promising drug combination approach for pancreatic cancer patients." "Pharmaxis has already seen very promising early results in a Phase II trial with patients that have the bone marrow cancer myelofibrosis," commented Gary Phillips, CEO of Pharmaxis. "This groundbreaking research stems from a long collaboration with the team of high calibre researchers at the Garvan Institute and provides exciting new evidence that PXS-5505 may also have a role as a therapy to improve the effect of current chemotherapy drugs in solid tumours like pancreatic cancer and extending the life of patients."

Phase 2Clinical ResultPhase 1

100 Deals associated with PXS-5505

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Polycythemia Vera	Phase 2	United States	Syntara Ltd.	18 Feb 2021
Polycythemia Vera	Phase 2	Australia	Syntara Ltd.	18 Feb 2021
Polycythemia Vera	Phase 2	South Korea	Syntara Ltd.	18 Feb 2021
Polycythemia Vera	Phase 2	Taiwan Province	Syntara Ltd.	18 Feb 2021
Post-essential thrombocythemia myelofibrosis	Phase 2	United States	Syntara Ltd.	18 Feb 2021
Post-essential thrombocythemia myelofibrosis	Phase 2	Australia	Syntara Ltd.	18 Feb 2021
Post-essential thrombocythemia myelofibrosis	Phase 2	South Korea	Syntara Ltd.	18 Feb 2021
Post-essential thrombocythemia myelofibrosis	Phase 2	Taiwan Province	Syntara Ltd.	18 Feb 2021
Post-polycythemia vera myelofibrosis	Phase 2	United States	Syntara Ltd.	18 Feb 2021
Post-polycythemia vera myelofibrosis	Phase 2	Australia	Syntara Ltd.	18 Feb 2021

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04676529 (ASH2024) Manual	Phase 1/2	Post-essential thrombocythemia myelofibrosis \| Post-polycythemia vera myelofibrosis	15	ruxolitinib + PXS-5505	-	Positive	09 Dec 2024
NCT04676529 (PXS5505-MF-101, ASH2023)	Phase 1/2	Post-essential thrombocythemia myelofibrosis	21	PXS-5505	ousjpamjbo(atdwfnadvx) = vomiting, urticaria, purpura, hypomagnesaemia, platelet count decreased, anemia cvyotzoyzh (ylghfqijvr ) View more	-	10 Dec 2023
NCT04676529 \| ACTRN12619000332123 (PXS5505-MF-101, ASH 12022 \| ASH 22022) Manual	Phase 1/2	Polycythemia Vera \| Post-essential thrombocythemia myelofibrosis	5	PXS-5505	bzelfoiaku(rkwefumdcc) = bjuorfgxcd isxqoddfaz (tswfxyyjjn ) View more	Positive	15 Nov 2022
NCT04676529 (Corporate Publications) Manual	Phase 2	Primary Myelofibrosis	15	PXS-5505	hluhsohooj(iheemmnpkf) = bdlaygyagc qibreclfee (pjwztcprha ) View more	Positive	19 Oct 2022

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Hiro LS Researcher

The AI Search Engine Built to Accelerate Biopharma Decisions

Search across billion-scale life sciences data to uncover signals, validate evidence, and act with confidence.

Ask any biopharma research question→

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, Patsnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Discover Synapse Data Servers

Synapse data is now integrated into the PatSnap LS Model Context Protocol (MCP) service. Customize your LLM agent now using our MCP server!

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis