An Italian innovative start up is preparing a project proposal for a Fast Track to Innovation. The company develops a novel formulation of dermatological cream containing tissue regenerative factors present in human platelets for the treatment of skin ulcers, including serious disorders, such as diabetic foot ulcers, pressure and leg ulcers. The start-up seeks two different partners: a contract manufacturing organization and a contract research organisation via research cooperation agreement.
The coordinator of the project is an innovative start-up, located in Milan, developer of highly effective (in terms of care-duration), accessible (in terms of distribution) and reliable (in terms of results achieved if compared with the state-of-the-art solutions) pharmaceutical cream, aimed at people affected by diabetic foot ulcers, pressure ulcers and leg ulcers. The project is addressed to validate the dermatologic cream formulation through the performance of pre-clinical and clinical tests and the development of the medical device to administer the cream. Moreover, it aims to develop regulatory and dissemination activities that will lead, at the end of the three project years, to reach the target set in the product business plan, that is the preparation of all activities necessary to transfer the production process to manufacturing companies of the pharmaceutical sector. This project has already involved 3 partners from Italy and Spain. The Italian start up is seeking additional partners to apply for the Fast Track to innovation call. The sought partners should complete the roadmap needed to enter in the healthcare market with a disruptive product. In order to complete the consortium, partners with the following profiles will be evaluated: 1. CONTRACT MANUFACTURING ORGANIZATION (CMO): to perform a controlled randomized clinical trial to obtain pre-regulatory authorization by the European Medicines Agency (EMA). The CMO will produce of the first batch of prototype state product (2000 units) to carry out the pre-clinical (200 units) and clinical studies (1800 units). 2. CONTRACT RESEARCH ORGANIZATION (CRO): to develop a pilot line of dermatological cream production complying with Good Manufacturing Practice (GMP) standards. The CRO will perform the study on the Efficacy and safety of Cord Blood Platelet-Gel (CBP-G) in patients with chronic neuropathic Diabetic Ulcers in a prospective randomized, open blinded endpoint (PROBE), phase II, multicenter study. Due to the Fast Track to Innovation programme characteristics, the company will not evaluate any Italian or Spanish candidates for this project proposal. Official deadline for the call: 19/02/2020 Deadline for expressions of interest: 10/01/2020 Estimated duration of the project: 3 years
Innovations and advantages
The innovation potential of the project regards: 1) the use of several thousands of ‘healthy and safe’ blood units, from umbilical cord and adults’ donors, available at hospitals but regrettably wasted (with significant cost and ethical concern); 2) the provision of a single-dose, home self-administered biopharmaceutical drug for the treatment of skin ulcers that will reduce hospitalization costs and improve the quality of life of a large number of patients.
Current stage of development
The project is moving to the “laboratory to market” phase having reached a level of maturity close to market at TRL6. This has been achieved through several stages: (TRL1) the pro-angiogenic and reparative role of blood platelet lysates has been demonstrated in early publications and recognized in numerous scientific papers (TRL2) completed proteomic studies on cord blood platelet lysates confirming the high pro-angiogenic feature of neonatal platelets (even higher than that of lysates obtained from adult blood platelets) (TRL3) documented clinical effectiveness of platelet gel obtained from placental blood in patients with devastating skin ulcers like those observed in epidermolysis bullosa (TRL4, TRL5) validation and laboratory testing of a lyophilization procedure for the long- term storage at room temperature of the basic material used for the preparation of the dermatological cream (TRL6) development of a cream formulation that is made by dissolving the lyophilized platelet lysate in proper solvents. An administration route has also been designed based on single use cream doses prepared at the moment of use by the patient that guarantees better shelf life of the lysate and easier administration. The cream and administration route have been evaluated in a relevant clinical environment to ensure efficacy and compatibility with clinical pathways in the treatment of chronic ulcers.
06001015 Pharmaceutical Products / Drugs
Market application codes
05003005 Drug delivery and other equipment
05007002 Pharmaceuticals/fine chemicals
Intellectual property rights
Comments, number and date of patent
IP assets consist of three patent families: 1. Italian patent application n. IT2008MI01316 (now granted). The application has been extended internationally through the PCT application WO2010007502 (A2). The patent family is currently granted in Europe (DE, ES, FR, GB, IT, PL and TR) and US. 2. International patent application WO2016193924 A2 The PCT application has been nationalised in EP, US, CN, CA, RU, IR, IN, EG, MY and SA (still pending). 3. Italian patent application n. 102019000005978
Type of partner sought
In order to complete the consortium the company is looking for: 1.CONTRACT MANUFACTURING ORGANIZATION (CMO) Characteristics: Private for profit entity working in GMP conditions (Good Manufacturing Practices standard), able to perform the liophilization of cord blood platelet lysate and formulation of single dose dermatological cream based on liophilized Cord Blood; responsable of labelling and primary and secondary packaging 2.CONTRACT RESEARCH ORGANIZATION (CRO) Characteristics: Certified For Profit European CRO; Development of protocol for the Phase 2 and 3 Randomized Clinical Multicenter Study “Efficacy and safety of Cord Blood Platelet-Gel (CBP-G) in patients with chronic neuropathic Diabetic Ulcers: a prospective randomized open blinded endpoint (PROBE); Primary outcomes: sample size comparison of dermatolgic cream with standard of care. The primary objective of this study will be to assess the incidence of complete wound closure after 12 weeks treatment with CBP-G in patients with diabetic foot ulcers. The secondary objectives of this study will be: o Time to achieve complete wound closure o Percentage reduction in total ulcer surface area at each visit o Incidence of complete wound healing at week 6 o Incidence and frequency of treatment-emergent adverse events (TEAEs) following 12 weeks of treatment o Wound-related infections o Changes in standard laboratory parameters CONTRACT MANUFACTURING ORGANIZATION (CMO): to perform a controlled randomized clinical trial to obtain pre-regulatory authorization by the European Medicines Agency (EMA). The CMO will produce of the first batch of prototype state product (2000 units) to carry out the pre-clinical (200 units) and clinical studies (1800 units). 2. CONTRACT RESEARCH ORGANIZATION (CRO): to develop a pilot line of dermatological cream production complying with Good Manufacturing Practice (GMP) standards. The CRO will perform the study on the Efficacy and safety of Cord Blood Platelet-Gel (CBP-G) in patients with chronic neuropathic Diabetic Ulcers in a prospective randomized, open blinded endpoint (PROBE), phase II, multicenter study.