With over 7,000 medicines in development, new treatments will continue to change patients’ lives, slowing disease progression, avoiding illness and reducing overall costs for healthcare systems. But developing a new medicine is a long, complex and risky process with no guarantees of success. Over the coming weeks, we look at a number of new medicines and the role that pharmaceutical incentives (or IP) have played in their development.

How Transgene wants to change patients’ lives by improving health outcomes

Transgene S.A., founded in December 1979, and part of the French Institut Mérieux since 1994, focuses on designing and developing therapeutic vaccines which may be game-changing for the treatment of cancer and infectious diseases. The company’s programs utilize viral vector technology with the goal of indirectly or directly killing infected or cancerous cells. Transgene is based in Strasbourg, France, and has additional operations in Lyon, as well as satellite offices in China and the U.S. The company employed about 120 staff mid-2017.

Transgene has several product candidates in preclinical and clinical development. The two lead clinical-stage products focus on the treatment of advanced non-small cell lung cancer (NSCLC) and liver cancer (hepatocellular carcinoma, HCC).

The impact of the diseases on patients, families, healthcare systems and society

Lung cancer is one of the most common malignancies worldwide. NSCLC represents approximately 85 percent or more of all lung cancers. Statistics from GLOBOCAN 2012 estimate that there were over 448,000 cases of lung cancer in Europe in 2012, and over 388,000 people there died from this disease. Lung cancer remains one of the cancer types with the worst prognosis, this underlines the unmet need in this disease.

TG4010 is Transgene’s therapeutic vaccine in clinical development for the treatment of advanced NSCLC. It is designed to help the body’s immune system identify cancerous cells carrying a protein which is present on solid tumours. In addition it leads to the production of Interleukin-2 (IL2), a cytokine that stimulates the immune system.

Hepatocellular carcinoma (HCC) is the most common form of liver cancer, estimated to account for over 80% of all liver cancers. According to GLOBOCAN 2012, there were over 63,000 new cases and over 62,000 deaths due to the disease estimated for 2012 in Europe. Currently there are few treatment options for advanced HCC patients, with only one drug, sorafenib, approved for the treatment of HCC. With a low five-year survival rate, especially for patients diagnosed at later stages of disease, and limited available therapies, new treatments are urgently needed.

Pexa-Vec (JX594/TG6006), a novel targeted approach to treat solid tumors with an oncolytic virus, is in clinical development for HCC. Pexa-Vec is an engineered oncolytic vaccinia virus armed with a GM-CSF gene that promotes an anti-tumour immune response. It is designed to selectively target and destroy cancer cells through three different mechanisms of action: the lysis (breakdown) of cancer cells through viral replication, the reduction of the blood supply to tumours through vascular disruption, and the stimulation of the body’s immune response against cancer cells.

IP rights and EU pharmaceutical incentives enable innovation for the cures of tomorrow

The EU intellectual property incentives framework has been and remains being instrumental in the development of Transgene’s therapies which are providing patients with urgently needed treatment options. Transgene retains full intellectual property (IP) rights to the technologies and platforms it developed and utilises. This strong patent protection led to clinical collaboration agreements between Transgene and Bristol-Myers Squibb, and with UC Davis for TG4010 in lung cancer, as well as to the partnership with SillaJen, Inc. and Lee’s Pharmaceutical to develop and commercialize PexaVec for the treatment of solid tumors in Europe.

The Advanced Therapy Medicinal Product (ATMP) status which is formally established for TG4010, and pending for PexaVec, has enabled Transgene to obtain regulatory guidance and scientific advice from the EMA at reduced cost.

TG4010 was accepted as a contribution to ADNA (Advanced Diagnostics for New Therapeutic Approaches), a program dedicated to personalized medicine approved by the European Commission. Development of TG4010 for NSCLC is therefore supported and partially funded by the French public agency Bpifrance.

Continued innovation fostered by incentives

Pexa-Vec has an orphan designation (OD) for HCC. The according waivers - for example the full waiver for paediatric development due to the very limited number of children with the disease - and OD incentives are very important for Transgene. They help to find investors and partners for the later-stage clinical development of its innovative therapies. Venture capital brings the freedom to operate, and financially strong partners are crucial to fund later stage clinical trials. Without it, Transgene’s novel cancer therapies would not reach the patients. A collaboration agreement with Sanofi, entered into in 2013, for the creation of a new state-of-the-art industrial platform dedicated to the production of immunotherapies, including Transgene’s therapeutic products in Lyon, France, also contributes to bringing medical innovations faster to patients in need.

The development of Transgene and the current clinical development pipeline

Unfortunately, Transgene does not formally count as an SME according to EU regulations, and therefore cannot benefit from the associated incentives. It is as a holding company owned by Institut Mérieux as a majority stakeholder. Apart from its shareholder structure Transgene has all the features of an SME company and would meet all SME eligibility criteria. The fact that Transgene cannot achieve SME status under the current rules results in a lack of opportunities to be even better supported by the EMA in the development of its products.

Despite the drawback of not being eligible for SME incentives, Transgene continues its efforts and significant investments for developing novel and innovative treatments for cancer and infectious diseases. By 2017 the company and its partners conducted 23 clinical studies with more than 800 patients for TG4010 and Pexa-Vec alone, and two more clinical trials for TG4010 are planned.

The company moreover keeps extending its innovative pipeline for the benefit of patients with cancer. TG4001 is a novel treatment in clinical development for head and neck cancer. This innovation also expects OD designation. This will increase the chance to find investors for further ramping up the research and early development operations of Transgene, and partners for the later stage clinical development of urgently needed therapies for rare solid tumours.