Cancers are a leading cause of mortality, accounting for nearly 10 million (M) annual deaths worldwide, or 1 in 6 deaths. In the European Union (EU) in 2020, 2.7M people were diagnosed with the disease, and 1.3M lost their lives to it. Personalized medicine and immunotherapy have improved the prognosis for several patients. However, they apply to a small number of patients and they typically target degenerate and redundant cell functions, leading to disease recurrence and resistance to therapy. 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 (observed in ~70% of cancers) is associated with poor prognosis and resistance to therapy. Despite the high interest in developing a clinical MYC inhibitor, no such drug is currently available. Indeed, MYC has been long considered undruggable due to 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 median OS (mOS) lower than 5 months, and less than 10% of patients who survive beyond 5 years. PDAC is also one of the most MYC-dependent cancers: in PDAC, in addition to controlling metabolism, proliferation and survival, MYC also controls the production of fibrosis and immune suppression signals that prevent access of cytotoxic drugs and protect cancer cells from immune targeting and elimination. As a result, the current therapeutic options for PDAC consist mainly of dated and ineffective chemotherapy regimen. Clearly, there is an urgent need for better solutions to treat MYC-driven cancers, especially PDAC.

Peptomyc’s disruptive drugs attack cancer’s engine MYC. OMO-103 is a patented, intravenously-delivered first-in-class cell-penetrating mini-protein based on Omomyc, the best MYC inhibitor to date. OMO-103 is the first drug to overcome the challenges of drugging MYC: its mini-protein fold provides exquisite selectivity, while its cell-penetrating domain directs it to the nuclei. 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 in patients and clinical activity, including a 49% tumour volume reduction and > 6 months of disease stabilisation in a metastatic PDAC (mPDAC) patient. Moreover, machine-learning (ML)-enabled analysis identified a soluble (blood) predictive biomarker of response that could help select the patients more likely to respond to therapy, as well as a pharmacodynamic biomarker signature that allows monitoring patient’s response in real-time.

Peptomyc’s goal is to develop OMO-103 until sufficient clinical evidence triggers a license agreement for OMO-103 to a pharmaceutical company that will complete its development and commercialization. The next step in this journey is to perform a Phase 1b CT in 1^st-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 adressing 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.