Periodic Reporting for period 1 - MYCureX (FIRST-IN-CLASS MYC INHIBITOR: THE MAKING OF A BREAKTHROUGH CANCER THERAPY)
Okres sprawozdawczy: 2023-07-01 do 2024-04-30
Cancers are a leading cause of mortality, accounting for nearly 10 million (M) annual deaths worldwide Personalized medicine and immunotherapy have improved the prognosis for several patients, yet their applicability remains limited, often targeting redundant cell functions leading to therapy resistance. Moreover, most treatments cause undesirable side effects and high toxicity.
A critical cancer driver is MYC, a non-redundant transcription factor essential for cancer cell proliferation and survival. MYC dysregulation (~70% of cancers) is associated with poor prognosis and resistance to therapy. Despite the high interest in developing a clinical MYC inhibitor, none is currently available. MYC's classification as undruggable stems from its lack of a well-defined structure and nuclear localization, which together challenge the most cutting-edge medicinal chemistry tools.
Pancreatic ductal adenocarcinoma (PDAC) is one of the most lethal cancers with less than 10% of patients surviving beyond 5 years. In PDAC, besides controlling metabolism, proliferation and survival, MYC also controls the production of fibrosis and immune suppression, preventing access of cytotoxic drugs and immune recognition. As a result, the current therapeutic options for PDAC consist mainly of dated and ineffective chemotherapy regimen.
Peptomyc’s disruptive drugs attack cancer's engine MYC. OMO-103 is a patented, first-in-class cell-penetrating mini-protein based on Omomyc, the best MYC inhibitor to date, and the first drug to overcome the challenges of drugging MYC. In addition, OMO-103 fosters synergy with cutting-edge personalized therapies and could apply to many additional cancers where MYC is deregulated.
OMO-103 successfully completed a FIH clinical trial in advanced solid tumour patients. The drug showed excellent safety, dose-dependent target engagement and clinical activity, including a 49% tumour volume reduction and > 6 months of disease stabilisation in a metastatic PDAC patient. Moreover, a predictive and a pharmacodynamic biomarker signature were identified.
Peptomyc's goal is to reach a licensing agreement for OMO-103 with a pharmaceutical company that will complete its development and commercialization. The next step in this journey is to perform a Phase 1b CT in 1st-line PDAC patients to assess safety and efficacy in combination with the standard of care, to develop the companion diagnostic prototype and a commercial-ready manufacturing process to sustain the scale up phase of the product, all encompassed objectives within this MYCureX project.
If the results of this project are positive as expected, Peptomyc could close a licensing agreement by 2026, bringing Peptomyc to revenue-stage and allowing reinvestment in our pipeline assets at discovery stage. Beyond this economic impact, MYCureX has social impact at both the EU and global levels, by addressing the broad cancer challenge, and will contribute to position Peptomyc and EU’s leadership in healthcare, by enabling targeting notoriously undruggable and clinically valuable targets, becoming a symbol of successful innovation.
A critical cancer driver is MYC, a non-redundant transcription factor essential for cancer cell proliferation and survival. MYC dysregulation (~70% of cancers) is associated with poor prognosis and resistance to therapy. Despite the high interest in developing a clinical MYC inhibitor, none is currently available. MYC's classification as undruggable stems from its lack of a well-defined structure and nuclear localization, which together challenge the most cutting-edge medicinal chemistry tools.
Pancreatic ductal adenocarcinoma (PDAC) is one of the most lethal cancers with less than 10% of patients surviving beyond 5 years. In PDAC, besides controlling metabolism, proliferation and survival, MYC also controls the production of fibrosis and immune suppression, preventing access of cytotoxic drugs and immune recognition. As a result, the current therapeutic options for PDAC consist mainly of dated and ineffective chemotherapy regimen.
Peptomyc’s disruptive drugs attack cancer's engine MYC. OMO-103 is a patented, first-in-class cell-penetrating mini-protein based on Omomyc, the best MYC inhibitor to date, and the first drug to overcome the challenges of drugging MYC. In addition, OMO-103 fosters synergy with cutting-edge personalized therapies and could apply to many additional cancers where MYC is deregulated.
OMO-103 successfully completed a FIH clinical trial in advanced solid tumour patients. The drug showed excellent safety, dose-dependent target engagement and clinical activity, including a 49% tumour volume reduction and > 6 months of disease stabilisation in a metastatic PDAC patient. Moreover, a predictive and a pharmacodynamic biomarker signature were identified.
Peptomyc's goal is to reach a licensing agreement for OMO-103 with a pharmaceutical company that will complete its development and commercialization. The next step in this journey is to perform a Phase 1b CT in 1st-line PDAC patients to assess safety and efficacy in combination with the standard of care, to develop the companion diagnostic prototype and a commercial-ready manufacturing process to sustain the scale up phase of the product, all encompassed objectives within this MYCureX project.
If the results of this project are positive as expected, Peptomyc could close a licensing agreement by 2026, bringing Peptomyc to revenue-stage and allowing reinvestment in our pipeline assets at discovery stage. Beyond this economic impact, MYCureX has social impact at both the EU and global levels, by addressing the broad cancer challenge, and will contribute to position Peptomyc and EU’s leadership in healthcare, by enabling targeting notoriously undruggable and clinically valuable targets, becoming a symbol of successful innovation.
During this first reporting period, we created an Ethics committee and performed a kick-off meeting to present the project, current activities and measures in place, and discuss any relevant aspect to ensure quality and regulatory compliance.
Additionally, as part of the of the clinical trial project management activities, Peptomyc selected a Clinical Research Organisation (CRO) to provide: Project Management, Regulatory, Monitoring, Medical Monitoring, Pharmacovigilance, Data management, Statistics, Medical Writing and Vendor Management.
The CRO conducted the pre-study/selection visits at the 4 selected clinical sites In April 2023, the clinical trial protocol was submitted to authorities via the CTIS portal of the EMA.
Patient’s data are recorded by clinical site staff in the electronic data capture system (eCRF) and source data verified by the CRO’s monitors on an ongoing basis.
On 27-Mar-2024, Amendment#1 to protocol was submitted to the authorities via CTIS portal of the EMA to increase the study population from 18 to 30 patients and 4 to 6 clinical sites and to add the overall survival follow-up of 12 months. The amendment package is now in the evaluation period (approval is expected between 30-May-2024 and 30-Jun-2024).
As a part of business activities, we continuously update our market analysis. During this 1st reporting period, we held a Scientific Advisory Board and two Development Advisory Board meetings with our KOLs to validate our clinical development plans and refine our market intelligence.
We selected the regulatory consultant for the support with Orphan drug designation and a kick-off meeting was held to start the activities.
Additionally, we deposited a new patent application covering the use of OMO-103 in autoimmune diseases and maintained the other patents in PCT or earlier stage.
Regarding the development of a companion diagnostic tool, we also initiated the analysis of biobank samples to assess the prevalence of the predictive biomarker signature in cancer patients. We performed a kick-off meeting with our AI subcontractor to align on the timelines for clinical trial samples analysis.
We also devised a communication plan with pharmas forecasting anticipated clinical data points with events. Presented a scientific communication at EACR-AACR-IACR oncology conference in Feb 2024. Following the publication of the results of our FIH clinical trial in Nature Medicine (https://rdcu.be/dGJbL) we reached out to several investors.
Additionally, as part of the of the clinical trial project management activities, Peptomyc selected a Clinical Research Organisation (CRO) to provide: Project Management, Regulatory, Monitoring, Medical Monitoring, Pharmacovigilance, Data management, Statistics, Medical Writing and Vendor Management.
The CRO conducted the pre-study/selection visits at the 4 selected clinical sites In April 2023, the clinical trial protocol was submitted to authorities via the CTIS portal of the EMA.
Patient’s data are recorded by clinical site staff in the electronic data capture system (eCRF) and source data verified by the CRO’s monitors on an ongoing basis.
On 27-Mar-2024, Amendment#1 to protocol was submitted to the authorities via CTIS portal of the EMA to increase the study population from 18 to 30 patients and 4 to 6 clinical sites and to add the overall survival follow-up of 12 months. The amendment package is now in the evaluation period (approval is expected between 30-May-2024 and 30-Jun-2024).
As a part of business activities, we continuously update our market analysis. During this 1st reporting period, we held a Scientific Advisory Board and two Development Advisory Board meetings with our KOLs to validate our clinical development plans and refine our market intelligence.
We selected the regulatory consultant for the support with Orphan drug designation and a kick-off meeting was held to start the activities.
Additionally, we deposited a new patent application covering the use of OMO-103 in autoimmune diseases and maintained the other patents in PCT or earlier stage.
Regarding the development of a companion diagnostic tool, we also initiated the analysis of biobank samples to assess the prevalence of the predictive biomarker signature in cancer patients. We performed a kick-off meeting with our AI subcontractor to align on the timelines for clinical trial samples analysis.
We also devised a communication plan with pharmas forecasting anticipated clinical data points with events. Presented a scientific communication at EACR-AACR-IACR oncology conference in Feb 2024. Following the publication of the results of our FIH clinical trial in Nature Medicine (https://rdcu.be/dGJbL) we reached out to several investors.
The reports from our Ethics experts are very positive. Both highlight the good quality of work done so far and do not foresee ethical concerns after evaluating in detail our project.
Regarding the clinical trial, two Safety Monitoring Committee (SMC) meetings have been held. The SMC#1, concluded that the combination of OMO-103 with the standard of care was safe, and Part 2 could start at 100% of the RP2D. The SMC#2 meeting, following three patients' completion of the first cycle, confirmed the safety of the higher OMO-103 dose. Thus, the current impact is that the study can be continued.
Regarding the clinical trial, two Safety Monitoring Committee (SMC) meetings have been held. The SMC#1, concluded that the combination of OMO-103 with the standard of care was safe, and Part 2 could start at 100% of the RP2D. The SMC#2 meeting, following three patients' completion of the first cycle, confirmed the safety of the higher OMO-103 dose. Thus, the current impact is that the study can be continued.