Problem/issue addressed by the project
Lisapharma, an Italian pharmaceutical SME, aims to bring on the market Edroma, a new product based on a nanotechnology platform to treat malignant high-grade gliomas (HGG), in particular glioblastoma (GBM). GBM is the most aggressive type of brain cancer for which there are no satisfactory curative therapies.
The Edroma medicinal product is a dispersion for injection made of nanoparticles loading the active ingredient zoledronic acid, obtained by a self-assembling procedure patented by University of Naples (ITA) and exclusively licensed to Lisapharma. Compared to the current brain-cancer therapy solutions, Edroma demonstrated in animals significant improvements characterized by higher efficacy and lower toxicity.
Importance for society
Despite recent advances in treatment, the prognosis for patients with HGG is dismal; so far the most effective treatment, i.e. radiotherapy, brings an increase in median survival from 3-4 months to around 9-10.
Only modest advancements in the treatment of GBM have occurred in the past 25 years, due to two main problems: 1 ) No specific laboratory studies are helpful in diagnosing GBM. Imaging studies of the brain are essential but, in most cases, complete staging is neither practical nor possible. These tumors do not have clearly defined margins; they tend to invade locally and spread along white matter pathways, creating the appearance of multiple GBMs or multicenter gliomas on imaging studied. This makes early diagnosis so infrequent that treatment is usually unable to provide a complete cure. 2 ) Medical therapy primarily involves glucocorticosteroids, which often produce a marked improvement in neurological symptoms by themselves. Chemotherapy has been used for primary therapy as either single agent or multi-agent regimens. Results have generally been conflicting and significant systemic toxicity is possible. These methods have not so far demonstrated satisfactory results, prolonging the survival only of few months, if compared to surgery (not possible in most cases) and radiotherapy alone.
Overall objectives
The general objective of this Phase I project was the development of a detailed Business Plan for attracting investments for the Edroma nanomedicine product development, registration, industrialization and introduction on the market, which includes activities related to detailed measurable specific objectives listed below:
OBJ 1: Product validation and industrialization planning, including: i) planning of preclinical and clinical studies based on the Protocol Assistance document received by the European Medicines Agency (EMA) on approval of Edroma’s orphan designation (EU/3/16/1735), in which the detailed preclinical and clinical trials to execute are described; ii) industrialization requirements assessment and planning, for all the activities and resources required to transfer the production of the new product to the selected manufacturing plants operating in Good Manufacturing Practice (GMP) conditions.
OBJ 2: Commercialization planning and partnerships definition, including: i) market analysis to assess the existing and potential market (size and growth) of HGG cases that might be treated through the new brain-targeted pharmacological nanosystem medicine; detailed commercialization and dissemination planning, taking into account the necessity to distribute the product at international level, given the relatively low number of HGG cases, and to reach a highly specialized audience of customers, i.e. clinicians working in cancer care hospital departments, as well as distribution partners; potential commercialization agreement definition, in particular signing license & supply agreements that appears as the more realistic strategy to develop the business.
OBJ 3: Definition of the legal (contractual) and ethical framework, including: i) assessment of Ethical Issues and regulations affecting product distribution in the different national markets, e.g. in terms of evaluation of the therapeutic options by examining the “good and harm” factors, the respect of the equality rights of the patients and the service to the community; ii) definition of the legal architecture to manage contractual relationships with partners and institutions, taking into account of the related ethical issues, and of further IPR protection strategy for the product after the orphan medicine exclusivity period.
OBJ 4: Financial plan preparation, including: i) quantification of the revenues and costs based on the detailed market analysis and on the cost of clinical validation, industrialization, commercialization, production and distribution activities; ii) financing requirements and Return On Investment evaluation by forecasting the cash flow and projecting financial performance indicators on a 5 years period, and by identifying the planned sources of funding.
