PTX-100 / Prescient Therap, Kirax - LARVOL DELTA

Prescient Therapeutics…has received Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) for its lead candidate PTX-100 for the treatment of cutaneous T-cell lymphoma (CTCL), covering both mycosis fungoides and Sézary syndrome subtypes (Proactiveinvestors) - "PTX-100 is a first-in-class therapy designed to disrupt cancer growth pathways and is currently being evaluated in a Phase 2a trial in patients with relapsed or refractory CTCL."

Orphan drug • Cutaneous T-cell Lymphoma • Mycosis Fungoides • Sezary Syndrome

Phase 1 Pharmacodynamic and Pharmacokinetic Study of the Geranylgeranyltransferase I Inhibitor PTX-100 (GGTI-2418) in Patients with Advanced Malignancies (ASH 2023) - P1 | "Preliminary data from this phase 1 dose escalation study of PTX-100 indicates promising safety profile in this difficult to treat pt population. PTX-100 demonstrated clinical activity in TCL with an ORR of 40%, and a median PFS for all TCL pts of 5.3 months. PD studies demonstrated that the GGT-1 target was engaged with sustained inhibition of RAP1 GG."

Clinical • Metastases • P1 data • PK/PD data • Cutaneous T-cell Lymphoma • Hematological Disorders • Hematological Malignancies • Lymphoma • Neutropenia • Non-Hodgkin’s Lymphoma • Oncology • Peripheral T-cell Lymphoma • Solid Tumor • T Cell Non-Hodgkin Lymphoma • HRAS • KRAS • NRAS • RAP1A • RHOA

Pharmacokinetic and Dose Response Relationships Observed in the Study "a Phase 1, Open- Label Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PTX-100 in Patients with Advanced and Relapsed/Refractory T Cell Lymphoma" (ASH 2024) - P1 | "In contrast, no correlation is noted with dose or exposure, using hematology, RBC, WBC, Neutrophils, and Platelets. This evaluation provides information regarding the selection of the doses used in the clinic."

Clinical • Metastases • P1 data • PK/PD data • Hematological Malignancies • Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma

Prescient advances PTX-100 trial with EMA review and expanded sites (Proactiveinvestors) - "Prescient Therapeutics has progressed its PTX-100 clinical programme, submitting a European Medicines Agency (EMA) application with an expected decision around mid-December 2025...If approved, the company plans to open three sites in France and three in Italy."

EMA approval • Trial status • Cutaneous T-cell Lymphoma

Geranylgeranyl transferase-1 inhibitor GGTI-2417 enhances the anti-tumour efficacy of histone deacetylase inhibitors in different T-cell lymphoma lines. (PubMed, Br J Haematol) - "HH, H9 and Hut78 TCL-cell co-treatment with the HDACIs belinostat or romidepsin with the GGTI-2418 methyl ester pro-drug GGTI-2417 (GGTI) synergistically increased cell death. These findings argue that combined GGTI/HDACI exposure synergistically induces cell death and reduces HDACI resistance through an AKT-dependent mechanism in TCL cells. In view of clinical results with GGTI-2418, the HDACI/GGTI-2418 combination strategy warrants consideration in T-cell malignancies."

Journal • Hematological Malignancies • Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma

PTX-100-PD-012017: Phase 1 Study of PTX-100 in Patients With Advanced Malignancies With PTCL Expansion Cohort (clinicaltrials.gov) - P1 | N=25 | Completed | Sponsor: Prescient Therapeutics, Ltd. | Active, not recruiting ➔ Completed

Trial completion • Colorectal Cancer • Gastric Cancer • Hematological Malignancies • Lymphoma • Multiple Myeloma • Oncology • Peripheral T-cell Lymphoma

Prescient focuses on advancing lead cancer drug toward pivotal trial (MSN News) - "Prescient Therapeutics...is in phase 2a trial for its lead compound PTX-100 in patients with rare blood cancer relapsed/refractory Cutaneous T-Cell Lymphoma (r/r CTCL) with the potential for its phase 2b arm to be registrational."

Trial status • Cutaneous T-cell Lymphoma

Prescient Therapeutics Confirms First US Site for PTX-100 Phase 2a Refractory Cutaneous T-Cell Lymphoma Trial (Small Caps) - "Oncology biotech Prescient Therapeutics...has announced the completion of its first US site visit for its Phase 2a trial of PTX-100 for patients with refractory or relapsed cutaneous T-cell lymphoma (r/r CTCL). The Massey Comprehensive Cancer Centre at Virginia Commonwealth University – ranked as one of the top cancer research centres in the US – joins three Australian sites where four patients are currently enrolled, with the visit marking the start of US patient recruitment to the trial and access to PTX-100 in that country."

Trial status • Cutaneous T-cell Lymphoma

New Prescient leadership to drive PTX-100 development in rare blood cancer (Stockhead) - "Prescient Therapeutics...recently announced the first patient had been dosed in its phase 2a clinical trial of PTX-100 in patients with relapsed or refractory cutaneous T-cell lymphoma (CTCL)."

Trial status • Cutaneous T-cell Lymphoma

Prescient Therapeutics’ PTX-100 Progresses to Phase 2a Trials after Outstanding Phase 1b Results (Small Caps) - P2a | N=115 | NCT06854653 | Sponsor: Prescient Therapeutics, Ltd. | "Clinical-stage oncology company Prescient Therapeutics (ASX: PTX) is looking to disrupt a US$1.8 billion market on the back of outstanding Phase 1b clinical trial data for lead candidate PTX-100 to treat cutaneous T-cell lymphoma (CTCL). Now in its Phase 2a trial, the advanced drug has delivered a 64% tumour halt or shrinkage in patients with the rare but relentless cancer, with only 4% reporting serious adverse effects. This compares to incumbent therapies which have demonstrated a 36% chance of any relief and adverse effects in 98% of patients."

P2a data • Cutaneous T-cell Lymphoma

Prescient Therapeutics Launches Funding Round to Advance Cancer Therapy Trials (Kalkine Media) - "Prescient Therapeutics...has officially launched a Share Purchase Plan (SPP), aiming to raise up to A$7 million to advance its clinical pipeline. The funds are earmarked for Phase 2 trials of PTX-100, the company's flagship targeted cancer therapy. This move reinforces Prescient's strategic focus on addressing rare and hard-to-treat cancers with precision treatments."

Financing • Cutaneous T-cell Lymphoma

Prescient Therapeutics doses first patient in Phase 2a study of PTX-100 for rare blood cancer (Proactiveinvestors) - "Prescient Therapeutics Ltd has kicked off its Phase 2a clinical trial evaluating PTX-100 for relapsed or refractory Cutaneous T-Cell Lymphoma (CTCL), having dosed its first patient....'The first patient in Prescient’s Phase 2a study has been dosed at the Linear Clinical Research site in Perth'....The open-label Phase 2a study will assess two dosage levels of PTX-100 across up to 40 patients at multiple sites in Australia, the United States and Europe."

Trial status • Cutaneous T-cell Lymphoma

FDA grants Fast Track status to Prescient’s PTX-100, advancing commercialisation pathway (Proactiveinvestors) - "Prescient Therapeutics Ltd has received Fast Track Designation from the United States Food and Drug Administration (FDA) for its lead candidate PTX-100, putting the company closer to potential Accelerated Approval - a central component of its commercialisation strategy. The designation, which applies to the treatment of adults with relapsed or refractory (r/r) mycosis fungoides – the most common subtype of Cutaneous T Cell Lymphoma (CTCL) - offers Prescient several regulatory advantages. These include increased access to the FDA and the ability to submit New Drug Applications (NDAs) on a rolling basis, expediting the review process for PTX-100....A Phase 2 clinical trial in cutaneous T cell lymphoma (CTCL) is now underway, with the first study site activated and patient enrolment expected to commence imminently."

Fast track • Trial status • Cutaneous T-cell Lymphoma • Mycosis Fungoides

PTX-100-PD-012017: Phase 1 Study of PTX-100 in Patients With Advanced Malignancies With PTCL Expansion Cohort (clinicaltrials.gov) - P1 | N=25 | Active, not recruiting | Sponsor: Prescient Therapeutics, Ltd. | Recruiting ➔ Active, not recruiting | Trial completion date: Apr 2023 ➔ Jul 2025 | Trial primary completion date: Apr 2023 ➔ Jul 2025

Enrollment closed • Trial completion date • Trial primary completion date • Colorectal Cancer • Gastric Cancer • Hematological Malignancies • Lymphoma • Multiple Myeloma • Oncology • Peripheral T-cell Lymphoma

A Phase 2 Study of PTX 100 in Patients With Relapsed/Refractory CTCL (clinicaltrials.gov) - P2 | N=115 | Recruiting | Sponsor: Prescient Therapeutics, Ltd. | Not yet recruiting ➔ Recruiting

Enrollment open • Cutaneous T-cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma

Prescient Therapeutics opens first clinical site for Phase 2a PTX-100 trial in cutaneous T-cell lymphoma (Proactiveinvestors) - "Clinical-stage oncology company Prescient Therapeutics Ltd has opened the first clinical site for its Phase 2a study of PTX-100 in patients with relapsed or refractory cutaneous T-cell lymphoma (CTCL)....The site is the first of several that Prescient plans to activate across Australia, the United States and Europe in the coming months....The Phase 2a study is designed as an open-label trial to evaluate two dosage levels of PTX-100 in some 40 patients diagnosed with CTCL, a rare and difficult-to-treat non-Hodgkin lymphoma that primarily affects the skin."

Trial status • Cutaneous T-cell Lymphoma

Prescient Therapeutics to present PTX-100 clinical data at international T-cell lymphoma forum (Proactiveinvestors) - P1 | N=24 | NCT03900442 | Sponsor: Prescient Therapeutics, Ltd. | "The Phase 1b study enrolled 19 patients and tested PTX-100 at doses of 500, 1,000 and 2,000 mg/m². The drug demonstrated a favourable safety profile, with an overall response rate of 42% among all evaluable TCL patients....Among cutaneous T-cell lymphoma (CTCL) patients specifically, six out of seven evaluable participants derived clinical benefit. Responders had a median progression-free survival exceeding 10 months, considerably beyond the typical 3.1 months observed with standard treatments such as vorinostat....These results provide the rationale for advancing PTX-100 into a Phase 2a trial in patients with relapsed and refractory CTCL, which is scheduled to commence in April 2025."

P1 data • Trial status • Cutaneous T-cell Lymphoma

A Phase 2 Study of PTX 100 in Patients with Relapsed/refractory CTCL (clinicaltrials.gov) - P2 | N=115 | Not yet recruiting | Sponsor: Prescient Therapeutics, Ltd.

Monotherapy • New P2 trial • Cutaneous T-cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma

Prescient Therapeutics welcomes US FDA allowance of Investigational New Drug application for PTX-100 Phase 2 clinical trial (Proactiveinvestors) - "Prescient Therapeutics Ltd...is trading higher after receiving a green light from the US Food and Drug Administration (FDA) to proceed with its Phase 2 clinical trial of PTX-100 — the company’s first-in-class Ras pathway inhibitor. The FDA cleared the Investigational New Drug (IND) application for the Phase 2 trial 100 — a significant milestone for Prescient — paving the way for the commencement of the Phase 2 study which will focus on relapsed and refractory cutaneous T-cell lymphomas (r/r CTCL)....Shares have jumped by as much as 53.7% in early ASX trading to A$0.063."

IND • New P2 trial • Stock price • Cutaneous T-cell Lymphoma

Prescient Therapeutics refines Phase 2 PTX-100 trial design for greater success (Proactiveinvestors) - "Clinical stage oncology company Prescient Therapeutics Ltd...had a cash and term deposit balance of $14.5 million on June 30, 2024, with spending in line with budget and has been actively engaged in the Phase 2 trial of PTX-100 in the past quarter, focusing on refining the trial design based on feedback from T-Cell Lymphoma experts, regulatory bodies, manufacturing and commercial advisors...Prescient plans to meet with the FDA with the revised trial strategy in the coming months and aims to start the Phase 2 trial before the end of the year. The company looks forward to sharing the trial design soon....Substantial Chemistry, Manufacturing and Control (CMC) activities are ongoing to support PTX-100’s development plans."

Clinical protocol • Commercial • New P2 trial • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma

Targeting the RAS upstream and downstream signaling pathway for Cancer treatment. (PubMed, Eur J Pharmacol) - "For instance, RTK inhibitors such as imatinib and afatinib selectively target these receptors, hindering ligand binding and reducing signaling initiation...Other inhibitors, like lonafarnib targeting Farnesyltransferase and GGTI 2418 targeting geranylgeranyl Transferase, disrupt post-translational modifications of proteins...Targeting downstream components with RAF inhibitors such as vemurafenib, dabrafenib, and sorafenib, along with MEK inhibitors like trametinib and binimetinib, has shown promising outcomes in treating cancers with BRAF-V600E mutations, including myeloma, colorectal, and thyroid cancers. Furthermore, inhibitors of PI3K (e.g., apitolisib, copanlisib), AKT (e.g., ipatasertib, perifosine), and mTOR (e.g., sirolimus, temsirolimus) exhibit promising efficacy against various cancers such as Invasive Breast Cancer, Lymphoma, Neoplasms, and hematological malignancies. This review offers an overview of small molecule inhibitors targeting specific proteins..."

Journal • Review • Breast Cancer • Colorectal Cancer • Endocrine Cancer • Hematological Disorders • Hematological Malignancies • Lung Cancer • Lymphoma • Multiple Myeloma • Non Small Cell Lung Cancer • Oncology • Solid Tumor • Thyroid Gland Carcinoma • BRAF • PIK3CA • PTEN

Prescient Therapeutics to present PTX-100 results at T-Cell Lymphoma Forum in California (Proactiveinvestors) - P1 | N=24 | NCT03900442 | Sponsor: Prescient Therapeutics, Ltd. | "Clinical-stage oncology company Prescient Therapeutics Ltd...has been invited to present PTX-100 Phase 1b results at the 15th Annual T-Cell Lymphoma Forum (TCLF) to be held in California from June 6 to 8...The Phase 1b study of PTX-100, which targeted patients with relapsed and refractory T-cell lymphomas (r/r TCLs), has met its primary goals of demonstrating safety and determining both pharmacokinetics...Additionally, the study has shown promising preliminary efficacy in r/r TCL patients, surpassing the typical results expected from standard treatments, with an overall response rate of 45%....'Prescient is committed to advancing into a Phase 2 trial in r/r TCL, with the study aiming to commence in Q3 2024.'"

New P2 trial • P1 data • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma

Prescient Therapeutics to present PTX-100 results at 5th World Congress of Cutaneous Lymphomas (Proactiveinvestors) - P1b | N=24 | NCT03900442 | Sponsor: Prescient Therapeutics, Ltd. | "Prescient Therapeutics Ltd...will present results of its Phase 1b study of PTX-100 in T-cell lymphomas during a poster presentation at the 5th World Congress of Cutaneous Lymphomas (WCCL) to be held in Pasadena, California, from April 11-13, 2024...Prescient’s Phase 1b trial of PTX-100 in TCLs has concluded enrolment, although two patients remain on study. Since the last update, an additional patient with CTCL — the last patient enrolled to the study — has experienced a clinical response (partial response)....'It is also timely to meet with key opinion leaders in the field ahead of our planned Phase 2 study this year.'"

Enrollment closed • New P2 trial • P1 data • Cutaneous T-cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

Prescient Therapeutics on cusp of value creation, says Pitt Street Research (Proactiveinvestors) - "In valuing Prescient Therapeutics Ltd...at A11.6 cents per share in its base case and A16.3 cents in its bull case, Pitt Street Research said the ASX-listed oncology drug developer was set for major milestones in the coming months and was, therefore, on the cusp of value creation...In a research report, Pitt Street said the company had several platforms and products in its portfolio, led by PTX-100, which was targeting T-Cell lymphoma...Pitt Street said that PTX-100 was closer to creating value for the company after noting in its initiation report last November that this was the most advanced asset the company had....Using a ‘Sum of the Parts’ rationale with 2 risk-adjusted DCFs for PTX-100 and CellPryme, Pitt Street has valued Prescient at A11.6 cents per share in its base case and A16.3 cents per share in its bull case."

Commercial • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma

Prescient Therapeutics (ASX: PTX) advances PTX-100 to pivotal Phase 2 study (Kalkine Media) - "PTX-100 of Prescient Therapeutics Limited...is in spotlight as it is entering a new stage. The company highlighted that PTX is on the verge of a major inflexion point with the beginning of a PTX-100 Phase 2 study...After finalising Phase 1 study, the company intends to undertake Phase 2 study in r/r TCL. For this purpose, it has engaged global KOLs (key opinion leaders) and regulatory consultants to design the study. The aim is to open study around mid 2024...A manufacturing campaign is in progress to supply this study. Finalisation of manufacturing and drug delivery is expected to happen in 2Q 2024. Moreover, protocol drafting, and quality systems and controls are underway....The company informed that FDA meeting is expected in 1Q/2Q 2024."

Commercial • FDA event • New P2 trial • Trial status • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma