Deregulation of the oncogene Myc is causally linked to most human cancers (1). Despite this evidence, targeting Myc has long been considered unfeasible because of potentially catastrophic side effects in normal tissues. However, we have shown that Myc inhibition displays extraordinary therapeutic benefit in various mouse models of cancer and causes only mild, well-tolerated and reversible side effects in normal tissues (2-7). For these studies, we employed a dominant negative inhibitor of Myc called Omomyc, which we designed and validated (4,8). Omomyc has so far been utilized only as a transgene and served solely as a proof of principle. However, thanks to the ERC-2013-CoG n° 617473 “Pushing Myc inhibition to the clinic”, we now have evidence that Omomyc-derived peptides have the potential to become the first clinically viable direct Myc inhibitor, whose application could extend to multiple types of cancer.
Indeed, such peptides can spontaneously enter cells and localize in their nuclei to inhibit Myc function. Moreover, the peptides can be delivered by intranasal administration directly to the brain and lung (among other tissues), where it exerts its antitumorigenic
action (Beaulieu et al., submitted). In order to reach commercial translation of this innovative anti-cancer therapy, we founded Peptomyc S.L. (December 2014). One essential early milestone for the pharmaceutical development of the Omomyc peptides is to establish the feasibility and cost estimations for its industrial manufacturing in compliance with the European Medicines Agency (EMA) and Federal Drug Administration (FDA) recommendations for the manufacturing of recombinant protein therapeutic products. Hence, in this Proof of Concept application, we proposed to reach this key milestone by:
1- Establishing the feasibility of recombinant cGMP production of Omomyc-CPP in partnership with an experienced Contract Manufacturing Organization (CMO);
2- Defining the analytical profile to confirm the molecule identity and activity for Drug Substance (DS) release;
3- Covering the Intellectual Property Rights (IPR) Protection during the national phase of the Patent Cooperation Treaty (PCT);
4- Establishing a briefing meeting with the EMA (and FDA);
5- Contacting Venture Capital firms (VC) and Business Angels (BA).
Through these aims, this PoC grant enabled us to develop a solid partnership with the CMO and others crucial stakeholders early in the pre-commercial development process, to demonstrate the feasibility and cost of production and to ensure protection of the IPR, and therefore provided key support to assess and de-risk the product for its pre-commercialization, overall rendering this project more attractive to investors for the continuation of its development.
