Menerba (MF101) / Bionovo - LARVOL DELTA

A phase 1/2a trial of amsulostat, a novel pan-lysyl oxidase inhibitor, in patients with advanced myelofibrosis as an add-on to ruxolitinib treatment for up to 52 weeks (ASH 2025) - P1/2 | "In addition to mediating extracellularstructural effects, amsulostat also reduces intracellular proto-oncogene growth factor signaling.PXS5505-MF-101 (NCT04676529) is a multi-center Phase 1/2a study of amsulostat in MF patients (pts). The majority oftreatment emergent AEs were mild, 63/84 (75%) ≤ Grade 2 (data cut-off 5th May 2025).Efficacy and safety from the completed 52-week study will be presented at the conference.ConclusionThe complete data from the AOP will provide insight into the safety profile of amsulostat in combinationwith RUX and also whether longer durations of amsulostat provide additional efficacy benefits. Thisevidence will be used to plan a randomized controlled confirmatory trial of amsulostat in the treatmentof MF."

Clinical • Metastases • P1/2 data • Fibrosis • Hematological Disorders • Immunology • Myelofibrosis • Thrombocytopenia • LOX

Multicenter, Open-Label Phase 1/2a Study of Pxs-5505 and Ruxolitinib in Patients with Primary, Post-Polycythemia Vera (PV) or Post-Essential Thrombocythemia (ET) Myelofibrosis (ASH 2024) - P1/2 | "PXS5505-MF-101 is an ongoing phase 1/2a study in patients (pts) with intermediate or high-risk MF (NCT04676529). Updated efficacy and safety data will be presented at the conference including 12-week data from all pts. Conclusion : The results from this trial using a novel combination of PXS-5505 and RUX will add to the existing safety profile of PXS-5505 and provide preliminary indicators of efficacy to help inform future investigations of PXS-5505 in pts with MF."

Clinical • P1/2 data • Tumor mutational burden • Fibrosis • Hematological Disorders • Immunology • Myelofibrosis • Myeloproliferative Neoplasm • Thrombocytopenia • Thrombocytosis • ASXL1 • IDH1 • IDH2 • SRSF2 • TMB • U2AF1

PXS5505-MF-101: A Phase 1/2a Study to Evaluate Safety, Pharmacokinetics and Pharmacodynamics of Pxs-5505 in Patients with Primary, Post-Polycythemia Vera or Post-Essential Thrombocythemia Myelofibrosis (ASH 2023) - P1/2 | "Background: Myelofibrosis (MF) is characterized by a progressive increase in extracellular matrix in the bone marrow (BM) associated with decreased production of hematopoietic cells. PXS-5505 has been well tolerated with no dose limiting toxicity or serious TRAEs. PD results indicate excellent LOX inhibition at the 200mg BID level. Further, there are preliminary indications of disease modification characterized by stable/improved blood counts and an improvement in collagen fibrosis."

Clinical • P1/2 data • PK/PD data • Anemia • Cardiovascular • Dermatology • Febrile Neutropenia • Fibrosis • Hematological Disorders • Immunology • Infectious Disease • Myelofibrosis • Myeloproliferative Neoplasm • Myocardial Infarction • Neutropenia • Septic Shock • Thrombocytopenia • Thrombocytosis • Urticaria • LOX

A PHASE 1/2A TRIAL OF PXS-5505, A NOVEL PAN-LYSYL OXIDASE INHIBITOR, IN PATIENTS WITH ADVANCED MYELOFIBROSIS (EHA 2025) - P1/2 | "PXS-5505 is a novel pan-lysyl oxidase inhibitor designed to exert an anti-fibrotic mode of action by preventing the cross-linking of collagen and elastin.PXS5505-MF-101 (NCT04676529) is a multi-center phase 1/2a study of PXS-5505 in MF patients (pts) which included a dose escalation phase (DEP), a cohort expansion phase (CEP) and an Add-on phase (AOP)...In the ongoing AOP, PXS-5505 200 mg BID is given in addition to ruxolitinib (RUX) over 52 weeks to assess safety and tolerability and determine the impact of PXS-5505 on disease-related parameters... Data from this current AOP using a combination of PXS-5505 and RUX in advanced MF will provide insight into the existing safety profile of PXS-5505 and help identify further indicators of efficacy, including when used in addition to RUX. This evidence will be used to inform clinical and regulatory discussions on the further development of PXS-5505 in MF."

Clinical • Metastases • P1/2 data • Fibrosis • Hematological Disorders • Immunology • Myelofibrosis • Thrombocytopenia • ASXL1 • IDH1 • IDH2 • JAK2 • LOX • SRSF2 • U2AF1

A phase I / IIa trial of PXS-5505, a novel pan-lysyl oxidase inhibitor, in advanced myelofibrosis. (PubMed, Haematologica) - "PXS5505-MF-101 is a multi-center phase 1/2a study of PXS-5505 in MF patients which included a dose escalation phase (DEP) and a cohort expansion phase (CEP). Over the 24-week treatment period preliminary indications of clinical efficacy, including a reduction in BM collagen, were evident. Overall, these data support continued investigation of PXS-5505."

Journal • P1/2 data • Fibrosis • Hematological Disorders • Immunology • Myelofibrosis • Thrombocytopenia • LOX

Menarche-a journey into womanhood: age at menarche and health-related outcomes in East Asians. (PubMed, Hum Reprod) - "Our study provides insights into the genetic architecture of AAM and its health-related outcomes in East Asians, highlighting causal links between BMI/T2D and earlier AAM, which may suggest potential prevention strategies for early puberty."

Journal • Diabetes • Glaucoma • Gynecology • Metabolic Disorders • Ophthalmology • Osteoporosis • Rheumatology • Solid Tumor • Type 2 Diabetes Mellitus • Uterine Leiomyoma • Women's Health

Phase 1/2a Study to Evaluate Safety, Pharmacokinetic and Pharmacodynamic Dose Escalation and Expansion Study of Pxs-5505 in Patients with Primary, Post-Polycythemia Vera or Post-Essential Thrombocythemia Myelofibrosis (ASH 2022) - P1/2 | "PXS5505-MF-101 is an ongoing, multi-center, open-label phase 1/2a study evaluating the safety and tolerability of PXS-5505 in patients with primary, post-polycythemia vera (PV) or post-essential thrombocythemia (ET) myelofibrosis (NCT04676529)...Eligible patients must have PMF or post-ET/PV MF of intermediate/high risk, relapsed/refractory, intolerant or not eligible to available JAK inhibitors (ruxolitinib, febratinib), including those with severe thrombocytopenia, and requiring therapy for symptomatic disease... These findings indicate that excellent targeted LOX and LOXL2 inhibition is being achieved at the 200mg BID level which has been selected as the recommended dose for the currently ongoing expansion phase with no dose limiting toxicity seen."

Clinical • P1/2 data • PK/PD data • Acute Myelogenous Leukemia • Hematological Disorders • Immunology • Myelofibrosis • Myeloproliferative Neoplasm • Polycythemia Vera • Thrombocytopenia • Thrombocytosis • LOX

Molecular cloning and expression analysis of two key genes, HDS and HDR, in the MEP pathway in Pyropia haitanensis. (PubMed, Sci Rep) - "...In this study, we reported the isolation and characterization of full-length HDS (MF101802) and HDR (MF159558) from Pyropia haitanensis...Additionally, the expression levels could be influenced by light, temperature and salinity and induced by methyl jasmonate (MJ) and salicylic acid (SA). This study contributed to our in-depth understanding of the roles of PhHDS and PhHDR in terpenoid biosynthesis in Pyropia haitanensis and the regulation of the two genes by external environments."

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