Periodic Reporting for period 1 - PEPTO1 (Feasibility study of a novel treatment for cancer based on a recombinant peptide therapy)
Reporting period: 2016-03-01 to 2016-07-31
Peptomyc S.L. is a spin-off company created at the Vall d’Hebron Institute of Oncology (VHIO) to develop a new, first-in-class, cancer therapy based on the use of innovative anti-Myc peptides. Myc is an oncogene deregulated in the majority of human cancers, making Peptomyc’s products applicable to the majority of cancer patients. The recombinant peptides are designed based on Omomyc, a Myc inhibitor benefiting from 20 years of preclinical validation as gene therapy. Omomyc possesses remarkable therapeutic impact in multiple cancer models, with limited side effects, and no emergence of resistance. The Omomyc-based peptides (Omomyc-CPP) developed by Peptomyc have unexpected cell-penetrating properties that make them excellent candidates for drug development. Three different drug products are being derived from the same drug substance, based on different administration routes, formulations and delivery devices. Peptomyc’s first mission, relative to this proposal, is to take the first drug product (OMO-101) to the completion of a successful phase I/II clinical trial, demonstrating safety and efficacy initially in patients suffering from glioblastoma (GB), and eventually develop the other variants/formulations to treat other types of cancer. Glioma is the most common malignant primary brain tumor and high grade glioma, glioblastoma multiforme (GB), is the most aggressive one. About 23.000 patients are diagnosed with GB each year (US and EU), with a dire prognosis and a median survival of only 15 months. No current therapy for GB is curative.
Upon completion of phase I/II, Peptomyc plans to reach a licensing deal with a pharmaceutical company, which will take care of the subsequent development and commercialization of OMO-101. In the feasibility study proposed here, Peptomyc’s team has elaborated and refined the business development plan of this new therapy to maximize its social and commercial potential. Such study includes:
a. Management of the IPR strategy;
b. Completion of the best CRO partners selection for [1] execution of the safety studies, [2] clinical trials coordination, [3] Pre- IND and IND applications and other regulatory requirements;
c. EMA Briefing meeting;
d. Refinement of the business development plan, including market and risk analyses, cost and market analysis for the commercialization of research tools (i.e. antibodies, biomarker kit) deriving from Peptomyc’s activities.
This feasibility study provides a strong asset to advance this unique, innovative therapy towards its commercialization not only into the GB market, but eventually in multiple types of cancer, and concludes that the innovation has strong potential of commercialization and requires additional funding in order to be developed to the level of investment readiness/market maturity.
Upon completion of phase I/II, Peptomyc plans to reach a licensing deal with a pharmaceutical company, which will take care of the subsequent development and commercialization of OMO-101. In the feasibility study proposed here, Peptomyc’s team has elaborated and refined the business development plan of this new therapy to maximize its social and commercial potential. Such study includes:
a. Management of the IPR strategy;
b. Completion of the best CRO partners selection for [1] execution of the safety studies, [2] clinical trials coordination, [3] Pre- IND and IND applications and other regulatory requirements;
c. EMA Briefing meeting;
d. Refinement of the business development plan, including market and risk analyses, cost and market analysis for the commercialization of research tools (i.e. antibodies, biomarker kit) deriving from Peptomyc’s activities.
This feasibility study provides a strong asset to advance this unique, innovative therapy towards its commercialization not only into the GB market, but eventually in multiple types of cancer, and concludes that the innovation has strong potential of commercialization and requires additional funding in order to be developed to the level of investment readiness/market maturity.
According to our proposal, we achieved:
• M1. Complete FTO analysis: This was performed by the legal firm ABG Patentes. According to their report there does not seem to exist any substantial risk of infringements of granted patent rights in Australia, Brazil, Canada, China, States Member of the Eurasian Patent Organization, States Member of the European Patent Organization, Hong Kong, Israel, India, Japan, South Korea, Mexico, Singapore, United States of America and South Africa.
• M2. Selection of the best value-for-money CROs for the [a] execution of the safety studies (PK/PD, toxicology), [b] clinical trials planning and coordination, [c] Pre- IND and IND applications and other regulatory requirements. This selection was made on the basis of teleconferences with the CROs, reviewing the various quotations obtained and taking into account peer opinions when needed.
• M3: Briefing meeting at EMA: An initial briefing meeting in order to clarify the regulatory strategy of the product has been requested with the EMA and granted for September 19th 2016. A Briefing Meeting Document has been prepared.
• M4. Refined business plan, including market and cost analysis, funding strategy and meeting with VCs and business angels. In this context, the company has already completed a first round of seed capital funding for 1 M euros with VC (HealthEquity) and BAs in April 2016 and has raised approximately 1.5 M euros in public funding (RETOS grant from the Spanish Ministry of Science and Innovation and PAT grant from the Entrepreneurial Competitivity Agency (ACCIÓ) of the Catalan Government).
• M1. Complete FTO analysis: This was performed by the legal firm ABG Patentes. According to their report there does not seem to exist any substantial risk of infringements of granted patent rights in Australia, Brazil, Canada, China, States Member of the Eurasian Patent Organization, States Member of the European Patent Organization, Hong Kong, Israel, India, Japan, South Korea, Mexico, Singapore, United States of America and South Africa.
• M2. Selection of the best value-for-money CROs for the [a] execution of the safety studies (PK/PD, toxicology), [b] clinical trials planning and coordination, [c] Pre- IND and IND applications and other regulatory requirements. This selection was made on the basis of teleconferences with the CROs, reviewing the various quotations obtained and taking into account peer opinions when needed.
• M3: Briefing meeting at EMA: An initial briefing meeting in order to clarify the regulatory strategy of the product has been requested with the EMA and granted for September 19th 2016. A Briefing Meeting Document has been prepared.
• M4. Refined business plan, including market and cost analysis, funding strategy and meeting with VCs and business angels. In this context, the company has already completed a first round of seed capital funding for 1 M euros with VC (HealthEquity) and BAs in April 2016 and has raised approximately 1.5 M euros in public funding (RETOS grant from the Spanish Ministry of Science and Innovation and PAT grant from the Entrepreneurial Competitivity Agency (ACCIÓ) of the Catalan Government).
Compared to current therapies in oncology, OmomycCPP presents several advantages. First, in contrast to targeted therapies, Myc inhibition has proven to be effective against several types of tumors independently of their mutational status or Myc expression levels, without evidence of any resistance to it even after long term treatment. In contrast to competitor Myc inhibitors in the Pharma industry’s pipeline, OmomycCPP constitutes the most direct and specific Myc inhibitor available to date and acts against all members of the Myc family, c-, N- and L-Myc, which are usually mutated or amplified in a mutually exclusive manner in multiple types of cancers, increasing the spectrum of treatable tumors. In addition, Omomyc selectively blocks a subset of Myc functions, resulting in only mild, well-tolerated and completely reversible side effects in vivo. Moreover, in the context of glioblastoma as a first indication, OmomycCPP’s ability to efficiently reach the brain following nasal administration is a significant advantage compared to chemotherapy, small molecule approaches and nucleic acid delivery, which often have limited efficacy in CNS diseases. While personalized therapies tend to atomize the glioblastoma and oncology market by being applicable to specific subsets of the patient population, our approach has the potential to treat all cancer patients, regardless of their mutational status.
It is important to keep in mind that glioma is the most common malignant primary brain tumor and high grade glioma, glioblastoma multiforme (GB), is the most aggressive one. About 23.000 patients are diagnosed with GB each year (US and EU), with a dire prognosis and a median survival of only 15 months. No current therapy for GB is curative. Eventually, we aim at expanding Omomyc’s applications to most oncological diseases.
Thanks to this Instrument, we have managed our IPR strategy completing a Freedom-to-operate analysis (with excellent results) for the use of our product; we have selected the best CRO partners selection for safety studies, clinical trials coordination, and regulatory requirements; we have also requested and obtained an appointment with EMA for a briefing meeting in September 2016; finally, we have refined our business development plan (including market and cost analysis, and funding strategy).
It is important to keep in mind that glioma is the most common malignant primary brain tumor and high grade glioma, glioblastoma multiforme (GB), is the most aggressive one. About 23.000 patients are diagnosed with GB each year (US and EU), with a dire prognosis and a median survival of only 15 months. No current therapy for GB is curative. Eventually, we aim at expanding Omomyc’s applications to most oncological diseases.
Thanks to this Instrument, we have managed our IPR strategy completing a Freedom-to-operate analysis (with excellent results) for the use of our product; we have selected the best CRO partners selection for safety studies, clinical trials coordination, and regulatory requirements; we have also requested and obtained an appointment with EMA for a briefing meeting in September 2016; finally, we have refined our business development plan (including market and cost analysis, and funding strategy).