Sanofi wins prestigious drug discovery award for innovative rare blood clotting disorder treatment

*  The British Pharmacological Society awards Cablivi® (caplacizumab), the first new treatment in 30 years for acquired Thrombotic Thrombocytopenic Purpura (aTTP), Drug Discovery of the Year 2020

*  TTP is a rare blood clotting disorder with 330 people known to be affected in England^[2]

READING, England, Dec. 17, 2019 /PRNewswire/ -- Sanofi's Cablivi^® (caplacizumab), the first nanobody therapy for the treatment of acquired Thrombotic Thrombocytopenic Purpura (aTTP), has been awarded the British Pharmacological Society's Drug Discovery of the Year 2020 award, at last night's annual Pharmacology conference.

The Sanofi team were commended by an international judging panel for the successful translation of caplacizumab from preclinical development through to approval as a first-in-class nanobody therapy, which the Society deemed as a pioneering example of the role of pharmacology in drug discovery and development.

Dr Tom Blackburn, Chair of the British Pharmacological Society's Industry Sub-Committee, said: "Caplacizumab represents a major innovation, not only in treating this life-threatening disorder (acquired Thrombotic Thrombocytopenic Purpura), but also as the first-ever nanobody therapy."

Recognition has also been given by Professor Marie Scully, Consultant Haematologist, University College London Hospital (UCLH) who commented: "The discovery of caplacizumab has been a huge and important milestone for our community. As a rare condition, aTTP is not well known but once identified, must be diagnosed and treated rapidly. Caplacizumab's ability to provide rapid protection from potentially fatal blood clots is vital for both people experiencing aTTP and the medical teams treating them."

Marc Moodley, Head of Sanofi Genzyme Medical, commented: "Every day, people at Sanofi work hard to ensure that our research and development efforts bring value not only to patients, but to the healthcare landscape as a whole. To have an independent and prominent UK body recognise this too, is a true honour. Receiving the Drug Discovery of the Year 2020 award is a fantastic achievement for our team, particularly as it translates into a real benefit for patients."

About Caplacizumab

Caplacizumab is the first and only specific therapy approved for the treatment of adults experiencing an episode of aTTP in conjunction with plasma exchange (PEX) and immunosuppression.^[3] Caplacizumab uses innovative nanobody technology to provide patients with aTTP rapid protection from blood clots in small blood vessels.^[3]

Caplacizumab was developed by Ablynx, which was acquired by Sanofi in 2018. The nanobody technology was originally discovered following identification that camelidae (e.g. camels and llamas) possess fully functional antibodies which consist of heavy chains only and therefore lack light chains.^[4] Due to their small size and unique structure, nanobodies are ideal building blocks which are small, robust and easily adaptable, resulting in a targeted biological therapy.^[4]

About acquired Thrombotic Thrombocytopenic Purpura (aTTP)

aTTP is a rare and life-threatening blood clotting disorder which can result in severe organ damage and death.^[5],[6] It usually presents as a medical emergency where sudden complications like heart attack and kidney failure can occur without warning.^{[6],[7],[8],[9]}

About Drug Discovery of the Year

The British Pharmacological Society established the Drug Discovery of the Year in 2012 to highlight the crucial role pharmacology plays in the development of new medicines and to reward the achievements of the teams of scientists who discover new drugs. The Society's Industry Sub-Committee selected caplacizumab from a shortlist of excellent first-in-class drugs and outstanding presentations. To find out more, visit: https://www.bps.ac.uk/membership-awards/prizes,-awards-and-grants/our-prizes/drug-discovery-of-the-year

About Sanofi UK

Sanofi UK is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.

With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.

Sanofi, Empowering Life

References

^[1]  The British Pharmacological Society. 2019. Available at: https://www.bps.ac.uk/news-events/news/articles/2019/pioneering-treatment-for-life-threatening-blood-cl Last accessed: December 2019

^[2] NHS England. Integrated Impact Assessment Report for Service Specifications. 2018. Available at: https://www.engage.england.nhs.uk/consultation/thrombocytopenic-purpura/ user_uploads/thrombotic-thrombocytopenic-purpura-impact-assessment.pdf. Last accessed: December 2019

^[3] EMA. Cablivi Summary of Product Characteristics. Available at: https://www.ema.europa.eu/en/documents/product-information/cablivi-epar-product-information_en.pdf. Last accessed: December 2019

^[4] Ablynx. Understanding Nanobodies. Available at: https://www.ablynx.com/technology-innovation/understanding-nanobodies/. Last accessed: December 2019

^[5] Knöbl P. Thrombotic thrombocytopenic purpura. Magazine of European Medical Oncology 2018; 11(3): 220–226

^[6] Joly BS, Coppo P, Veyradier. Thrombotic thrombocytopenic purpura. Blood 2017; 129:2836–2846

^[7] Yves Benhamou, et al. Cardiac troponin-I on diagnosis predicts early death and refractoriness in acquired thrombotic thrombocytopenic purpura. Journal of Thrombosis and Haemostasis 2015; 13(2):293–302

^[8] Han-Mou Tsai. The kidney in thrombotic thrombocytopenic purpura. Minerva Medical 2007; 98(6):731–747

^[9] Blombery P and Scully M. Management of thrombotic thrombocytopenic purpura: current perspectives. Journal of Blood Medicine 2014; 5:15–23