Periodic Reporting for period 2 - MYCureX (FIRST-IN-CLASS MYC INHIBITOR: THE MAKING OF A BREAKTHROUGH CANCER THERAPY)
Période du rapport: 2024-05-01 au 2025-02-28
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, and to develop 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, and to develop 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 second reporting period, we maintained contact with the MYCureX Ethics committee and performed a follow-up meeting to present how the project is advancing, current activities and measures in place, and discussed any relevant aspect to ensure quality and regulatory compliance.
As mentioned in the previous report, Amendment #1 to the protocol was submitted to the authorities on March 27, 2024, and approved on May 30, 2024. Following this, two additional amendments were submitted to the regulatory agency, Amendment #2 on July 24 and Amendment #3 on December 11, 2024, which were subsequently approved on September 9, 2024, and February 4, 2025, respectively.
The Phase Ib clinical trial is progressing as planned, with the recruitment rate meeting expectations. All patient data is being systematically recorded in the eCRF, ensuring accurate and comprehensive documentation. The CRO is actively monitoring the data, performing regular quality checks to ensure compliance with regulatory standards and protocol requirements. A protocol amendment and adjustment in the treatment regimen were required, but the trial is continuing as planned. Clinical sites are actively collecting biological samples, and the first shipments to the central laboratory have been completed. Initial pharmacokinetic (PK) and anti-drug antibody (ADA) analyses have also been conducted. Additionally, during this reporting period, two additional clinical sites have been activated, further strengthening the trial’s capacity and facilitating patient recruitment.
While these activities go beyond the scope of this project, we have also initiated an investigator-led Phase II trial in osteosarcoma and obtained an IND in December 2024 to conduct a Window of Opportunity (WoO) trial in the U.S. in PDAC patients.
As a part of business activities, we continuously update our market analysis. During this second 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.
Regarding the Orphan Drug Designation (ODD), it was submitted to both EU and US on 16th of August and 3rd of September 2024, respectively. Corresponding authorities (EMA and FDA) requested additional information on 11th and 24th of November, respectively.
Additionally, the company has ensured the maintenance of its patent portfolio through timely fee payments and compliance with regulatory requirements. Ongoing monitoring of the patent landscape continues to protect and strengthen our intellectual property position.
The communication plan with pharmaceutical partners has been updated to align with anticipated clinical data milestones. We have maintained engagement with multiple pharmaceutical companies and investors to explore potential collaborations and funding opportunities.
As mentioned in the previous report, Amendment #1 to the protocol was submitted to the authorities on March 27, 2024, and approved on May 30, 2024. Following this, two additional amendments were submitted to the regulatory agency, Amendment #2 on July 24 and Amendment #3 on December 11, 2024, which were subsequently approved on September 9, 2024, and February 4, 2025, respectively.
The Phase Ib clinical trial is progressing as planned, with the recruitment rate meeting expectations. All patient data is being systematically recorded in the eCRF, ensuring accurate and comprehensive documentation. The CRO is actively monitoring the data, performing regular quality checks to ensure compliance with regulatory standards and protocol requirements. A protocol amendment and adjustment in the treatment regimen were required, but the trial is continuing as planned. Clinical sites are actively collecting biological samples, and the first shipments to the central laboratory have been completed. Initial pharmacokinetic (PK) and anti-drug antibody (ADA) analyses have also been conducted. Additionally, during this reporting period, two additional clinical sites have been activated, further strengthening the trial’s capacity and facilitating patient recruitment.
While these activities go beyond the scope of this project, we have also initiated an investigator-led Phase II trial in osteosarcoma and obtained an IND in December 2024 to conduct a Window of Opportunity (WoO) trial in the U.S. in PDAC patients.
As a part of business activities, we continuously update our market analysis. During this second 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.
Regarding the Orphan Drug Designation (ODD), it was submitted to both EU and US on 16th of August and 3rd of September 2024, respectively. Corresponding authorities (EMA and FDA) requested additional information on 11th and 24th of November, respectively.
Additionally, the company has ensured the maintenance of its patent portfolio through timely fee payments and compliance with regulatory requirements. Ongoing monitoring of the patent landscape continues to protect and strengthen our intellectual property position.
The communication plan with pharmaceutical partners has been updated to align with anticipated clinical data milestones. We have maintained engagement with multiple pharmaceutical companies and investors to explore potential collaborations and funding opportunities.
The input from our Ethics experts is very positive and in line with the different regulatory consultants. 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, one Safety Monitoring Committee (SMC), SMC#3 meeting was held on July 2024 to review and discuss the safety data. After proposing some changes in the premedication and treatment regimen, the conclusion was to continue the OMO-103-02 trial according to the protocol of expansion part two.
Regarding the clinical trial, one Safety Monitoring Committee (SMC), SMC#3 meeting was held on July 2024 to review and discuss the safety data. After proposing some changes in the premedication and treatment regimen, the conclusion was to continue the OMO-103-02 trial according to the protocol of expansion part two.