Marco Dieci, CEO,

Holostem Terapie Avanzate

"My vision is to see a single pan-European payer for rare diseases, linked to a database of patients, each with its particular indication."

Holostem was founded in 2008 as the first biotech company fully devoted to ATMPs (advanced therapies medicinal products) based on epithelial stem cells. Could you introduce Holostem to us? Holostem was created as a spin-off from the University of Modena and Reggio Emilia, together with the partnership of Chiesi. Our concentrated efforts led to the GMP authorization of the facility and the development and registration of Holoclar, the first regenerative medicine product used in limbal stem cell deficiency (LSCD) caused by burns to the eyes. This rare disease affects about 3.3 out of 100,000 people in Europe. The market authorization was received seven years later, in 2015. Throughout this period, Chiesi used its network to commercialize the product in eight European countries, where it is still present. However, since June 2020, we officialized the transfer of marketing authorization to Holostem in order to ensure greater success of the treatments by offering an integral service with dedicated people, facilitating direct contact of our medical service with clinical centers and physicians during the critical phases of biopsy and implant. Our aim has been to provide a full-package service that supports all the stakeholders in delivering the best therapeutic treatment to patients. By September 2020, we successfully registered the product under our own brand, and this year we also made Holoclar available in the Czech Republic.

Could you help our audience better understand what defines ATMPs? The big difference ATMPs make is that they have a long-term restorative effect rather than offering a temporary solution. Based on different variables, ATMPs have an efficiency rate of 30% to 50% and are only administered only once or twice, having a life-changing impact.

What is the market size of LSCDs and what is your strategy for reaching this fragmented market? Every year, about 700 new cases of LSCD are registered in Europe, with around 50 to 100 new patients in each European county, depending on its population size. However, there are many patients who might have suffered an eye injury 20 years ago and they can have the option to see again. As an orphan drug, it is indeed challenging to commercialize the product given the different rules in different jurisdictions. My vision is to see a single pan-European payer for rare diseases, linked to a database of patients, each with its particular indication.

What is the scalability enabled by the GMP stem cell-based cultures facility? Our current facility could cover 250 patients yearly. To significantly increase our capacity, we need to build a new facility. Holostem’s business model relies on one technology platform for regenerative medicine for various applications, so the capacity expansion could also be spared for other indications like hypospadias, which has a larger patient pool. In gene therapy, epidermolysis bullosa is a condition with a devastating impact on the life of the patients and their families; there are about 7,000 cases in Europe, but we can treat only around 100 patients in the EU at the current capacity.

What is the strategy for the rest of your pipeline, including the Phase 3 treatment for the junctional epidermolysis bullosa? The Phase 3 trials will start in three countries – Italy, Germany and France. To bring this pipeline forward, we are looking for additional investors to speed up clinical trials, registration and commercialization. As one of the few biotech companies with a university on board, we’d like to bring in a third stream as either a buy-out, a venture capital fund, or institutional support who can affiliate in this private-public partnership.

How do you see the bridge between innovative ideas and commercial success in Italy’s ecosystem? In 2015, Holocar was the first drug in its field to receive market authorization, and although we have met all compliance requirements, we have not benefited from government support in the form of a cash refund or discounts for the development activities. The biotech segment in Italy abounds with know-how, yet this know-how is lent in a CMO context. Holostem is one of the few players who develops and also commercializes its products. In Italy, there is a big gap between the lab and ideas hitting the market.