M7824 is an investigational bifunctional immunotherapy that is designed to combine a TGF-β trap with the anti-PD-L1 mechanism in one fusion protein. M7824 is designed to combine co-localized blocking of the two immunosuppressive pathways – targeting both pathways aims to control tumor growth by potentially restoring and enhancing anti-tumor responses. M7824 is currently in Phase I studies for solid tumors, as well as a trial to investigate M7824 compared with pembrolizumab as a first-line treatment in patients with PD-L1 expressing advanced NSCLC. The multicenter, randomized, open-label, controlled study is evaluating the safety and efficacy of M7824 versus pembrolizumab as a monotherapy treatment.
About the FDA Orphan Designation
FDA orphan drug designation is granted to drugs intended to treat rare diseases or disorders that affect fewer than 200,000 people in the US, or those that affect more than 200,000 people, but are unlikely to recover the costs of developing and marketing the drug. Orphan drug designation by the FDA qualifies the sponsor for incentives provided for in the Orphan Drug Act, which can include protocol assistance for clinical trials, prescription drug user fee waivers, tax incentives and seven years of market exclusivity. The granting of an orphan drug designation does not alter the standard regulatory requirement to establish the safety and effectiveness of a drug through adequate and well-controlled studies to support approval. The orphan drug designation for M7824 applies only to BTC.
About Biliary Tract Cancer (BTC)
BTC is a collective term for a group of rare and aggressive gastrointestinal cancers, including intrahepatic cholangiocarcinoma (iCC), extrahepatic cholangiocarcinoma (eCC), and gallbladder carcinoma (GBC).1 Surgery is the only curative treatment, but most patients present with advanced disease and therefore have a limited survival.1 Approximately 186,000 cases of BTC are estimated to occur annually world-wide.2 However, incidence of BTC varies in different parts of the world: the incidence of cholangiocarcinomas is rising in the Western world, with reports of up to 2 in 100,0004. By contrast, in Asian countries, the incidence is much higher.4 GBC also has an incidence of 2 in 100,000, but is much more prevalent in parts of South America.4 Collectively these cancers present late in the majority of patients and long-term outcomes for resectable patients are poor with median survival in the advanced setting less than 1 year.1,3
1. Blair A B et al. Immunotherapy as a treatment for biliary tract cancers: A review of approaches with an eye to the future. Current Problems in Cancer (2018) https://www.sciencedirect.com/science/article/pii/S0147027217301083?via%3Dihub Accessed November 2018
2. Naghavi M et al. The Global Burden of Cancer 2013. JAMA Oncol. 2015 July 1; 1(4): 505–527. doi:10.1001/jamaoncol.2015.0735.
3. Hezel AF et al. Genetics of biliary tract cancers and emerging targeted therapies. J Clin Oncol 2010;28:3531–40. http://dx.doi.org/10.1200/JCO.2009.27.4787 Accessed November 2018
4. Goldstein D et al. New molecular and immunotherapeutic approaches in biliary cancer. ESMO Open (2017). Published online 2017 Mar 27. doi: https://dx.doi.org/10.1136%2Fesmoopen-2016-000152 Accessed November 2018
5. Lamarca A, et al. Ann Oncol. 2014;25(12):2328–2338.
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About Merck KGaA, Darmstadt, Germany
Merck KGaA, Darmstadt, Germany, a vibrant science and technology company, operates across healthcare, life science and performance materials. Around 51,000 employees work to make a positive difference to millions of people’s lives every day by creating more joyful and sustainable ways to live. From advancing gene editing technologies and discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices – the company is everywhere. In 2017, Merck KGaA, Darmstadt, Germany, generated sales of € 15.3 billion in 66 countries.
The company holds the global rights to the name and trademark “Merck” internationally. The only exceptions are the United States and Canada, where the business sectors of Merck KGaA, Darmstadt, Germany, operate as EMD Serono in healthcare, MilliporeSigma in life science, and EMD Performance Materials. Since its founding in 1668, scientific exploration and responsible entrepreneurship have been key to the company’s technological and scientific advances. To this day, the founding family remains the majority owner of the publicly listed company. For more information about Merck KGaA, Darmstadt, Germany, visit www.emdgroup.com.