- High and durable response rate in disease without any FDA-approved therapies
- Cemiplimab rolling BLA submission initiated per FDA Breakthrough Therapy Designation, with completed submission expected in Q1 2018
PARIS, France & TARRYTOWN, NY, USA I December 13, 2017 I Sanofi and Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced positive topline results from a pivotal Phase 2 clinical study of cemiplimab in 82 patients with advanced cutaneous squamous cell carcinoma (CSCC), the second deadliest skin cancer after melanoma.[i],[ii] Cemiplimab, an investigational human antibody targeting PD-1 (programmed cell death protein 1), demonstrated an overall response rate (ORR) of 46.3%, as determined by independent review. The median duration of response had not yet been reached at the data cut-off point (32 of 38 responses are ongoing). At the time of this analysis all patients had a minimum of 6 months of follow up.
The safety profile in this study was generally consistent with approved anti-PD-1 agents.
These pivotal data will form the basis of a rolling Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA), which has been initiated and is expected to be completed in the first quarter of 2018. A rolling BLA submission allows for portions of the regulatory application to be submitted to the FDA as they are completed.[iii] A submission to the European Medicines Agency (EMA) is also expected to be completed in the first quarter of 2018. These data confirm the positive Phase 1 clinical trial expansion cohort results reported at ASCO 2017, which led to a Breakthrough Therapy Designation for cemiplimab in advanced CSCC in September 2017.
“EMPOWER-CSCC 1 was initiated in 2016 and has enrolled rapidly, underscoring the serious unmet need in advanced CSCC,” said Elias Zerhouni, M.D., President, Global R&D, Sanofi. “We look forward to working with regulatory agencies globally to bring this important therapy to advanced CSCC patients as quickly as possible. We continue to rapidly advance a broad development program to evaluate cemiplimab both as monotherapy and combination across a number of solid tumor and blood cancers.”
The efficacy data reported today include results from 82 patients in the Phase 2 EMPOWER-CSCC 1 study. Approximately two-thirds of patients had progressed after prior systemic chemotherapy or radiation.
“For patients with CSCC that cannot be cured by surgery or radiation, there are no FDA-approved treatment options, and advanced CSCC is responsible for 3,900 to 8,800 deaths per year in the U.S.,[iv]” said Israel Lowy, MD, PhD, Vice President of Global Clinical Development and Head of Translational Science and Clinical Oncology, Regeneron. “This is the largest prospective study ever conducted in this disease, and we are pleased that many people were able to achieve deep and durable responses with cemiplimab monotherapy. The high and durable response rates seen in this study are particularly notable given that the study enrolled patients regardless of biomarker status.”
EMPOWER-CSCC 1 is a single-arm, open-label clinical trial and remains active. Enrollment is complete in the study arm of patients with metastatic CSCC receiving a 3 mg/kg dose of cemiplimab every two weeks. Enrollment continues in the remaining two study arms of patients with metastatic CSCC receiving a 350 mg flat dose of cemiplimab every three weeks and patients with locally advanced and unresectable CSCC receiving a 3 mg/kg dose of cemiplimab every two weeks.
Updated results from both the EMPOWER-CSCC 1 Phase 2 trial and the Phase 1 clinical trial will be submitted for presentation at a 2018 medical congress.
Cemiplimab is being jointly developed by Regeneron and Sanofi under a global collaboration agreement. Cemiplimab is currently under clinical development, and its safety and efficacy has not been fully evaluated by any regulatory authority.
About Cutaneous Squamous Cell Carcinoma (CSCC)
CSCC is the second most common type of skin cancer in the United States. Although CSCC has a good prognosis when caught early, it can prove especially difficult to treat when it progresses to advanced stages.[v] Patients at this stage can be disfigured due to multiple surgeries to remove CSCC tumors on the head, neck and other parts of the body.[vi] CSCC is the second deadliest skin cancer after melanoma[vii],[viii] and is responsible for the most deaths among non-melanoma skin cancer patients.6
About Sanofi
Sanofi 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.
About Regeneron
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for nearly 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to six FDA-approved treatments and over a dozen product candidates in development, all of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye disease, heart disease, allergic and inflammatory diseases, pain, cancer, infectious diseases and rare diseases.
Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite® antibody technologies and ambitious initiatives such as the Regeneron Genetics Center, one of the largest genetics sequencing efforts in the world
[i] Siegel R, et al. Cancer Statistics, 2017. CA Cancer J Clin. 2017;67:7-30. doi: 10.3322/caac.21387
[ii] Burton KA, et al. Cutaneous Squamous Cell Carcinoma: A Review of High-Risk and Metastatic Disease. Am J Clin Dermatol. 2016;17:491-508. doi:10.1007/s40257-016-0207-3.
[iii] U.S. Food and Drug Administration. Guidance for Industry Expedited Programs for Serious Conditions – Drugs and Biologics. Available at :
http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm358301.pdf. Last Accessed: December 11, 2017.
[iv] Karia PS, Han J, Schmults CD. Cutaneous squamous cell carcinoma: estimated incidence of disease, nodal metastasis, and deaths from disease in the United States, 2012. J Am Acad Dermatol. 2013 Jun;68(6):957-66. doi: 10.1016/j.jaad.2012.11.037. Epub 2013 Feb 1.
[v] Medscape. Cutaneous Squamous Cell Carcinoma. Available at: http://emedicine.medscape.com/article/1965430-overview. Last Accessed: Last Accessed: December 11, 2017.
[vi] Weinberg AS, Ogle CA, Shim EK. Metastatic cutaneous squamous cell carcinoma: an update. Dermatol Surg. 2007;33:885-899.
[vii] Siegel R, et al. Cancer Statistics, 2017. CA Cancer J Clin. 2017;67:7-30. doi: 10.3322/caac.21387
[viii] Burton KA, et al. Cutaneous Squamous Cell Carcinoma: A Review of High-Risk and Metastatic Disease. Am J Clin Dermatol. 2016;17:491-508. doi:10.1007/s40257-016-0207-3.
SOURCE: Sanofi
Post Views: 23
- High and durable response rate in disease without any FDA-approved therapies
- Cemiplimab rolling BLA submission initiated per FDA Breakthrough Therapy Designation, with completed submission expected in Q1 2018
PARIS, France & TARRYTOWN, NY, USA I December 13, 2017 I Sanofi and Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced positive topline results from a pivotal Phase 2 clinical study of cemiplimab in 82 patients with advanced cutaneous squamous cell carcinoma (CSCC), the second deadliest skin cancer after melanoma.[i],[ii] Cemiplimab, an investigational human antibody targeting PD-1 (programmed cell death protein 1), demonstrated an overall response rate (ORR) of 46.3%, as determined by independent review. The median duration of response had not yet been reached at the data cut-off point (32 of 38 responses are ongoing). At the time of this analysis all patients had a minimum of 6 months of follow up.
The safety profile in this study was generally consistent with approved anti-PD-1 agents.
These pivotal data will form the basis of a rolling Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA), which has been initiated and is expected to be completed in the first quarter of 2018. A rolling BLA submission allows for portions of the regulatory application to be submitted to the FDA as they are completed.[iii] A submission to the European Medicines Agency (EMA) is also expected to be completed in the first quarter of 2018. These data confirm the positive Phase 1 clinical trial expansion cohort results reported at ASCO 2017, which led to a Breakthrough Therapy Designation for cemiplimab in advanced CSCC in September 2017.
“EMPOWER-CSCC 1 was initiated in 2016 and has enrolled rapidly, underscoring the serious unmet need in advanced CSCC,” said Elias Zerhouni, M.D., President, Global R&D, Sanofi. “We look forward to working with regulatory agencies globally to bring this important therapy to advanced CSCC patients as quickly as possible. We continue to rapidly advance a broad development program to evaluate cemiplimab both as monotherapy and combination across a number of solid tumor and blood cancers.”
The efficacy data reported today include results from 82 patients in the Phase 2 EMPOWER-CSCC 1 study. Approximately two-thirds of patients had progressed after prior systemic chemotherapy or radiation.
“For patients with CSCC that cannot be cured by surgery or radiation, there are no FDA-approved treatment options, and advanced CSCC is responsible for 3,900 to 8,800 deaths per year in the U.S.,[iv]” said Israel Lowy, MD, PhD, Vice President of Global Clinical Development and Head of Translational Science and Clinical Oncology, Regeneron. “This is the largest prospective study ever conducted in this disease, and we are pleased that many people were able to achieve deep and durable responses with cemiplimab monotherapy. The high and durable response rates seen in this study are particularly notable given that the study enrolled patients regardless of biomarker status.”
EMPOWER-CSCC 1 is a single-arm, open-label clinical trial and remains active. Enrollment is complete in the study arm of patients with metastatic CSCC receiving a 3 mg/kg dose of cemiplimab every two weeks. Enrollment continues in the remaining two study arms of patients with metastatic CSCC receiving a 350 mg flat dose of cemiplimab every three weeks and patients with locally advanced and unresectable CSCC receiving a 3 mg/kg dose of cemiplimab every two weeks.
Updated results from both the EMPOWER-CSCC 1 Phase 2 trial and the Phase 1 clinical trial will be submitted for presentation at a 2018 medical congress.
Cemiplimab is being jointly developed by Regeneron and Sanofi under a global collaboration agreement. Cemiplimab is currently under clinical development, and its safety and efficacy has not been fully evaluated by any regulatory authority.
About Cutaneous Squamous Cell Carcinoma (CSCC)
CSCC is the second most common type of skin cancer in the United States. Although CSCC has a good prognosis when caught early, it can prove especially difficult to treat when it progresses to advanced stages.[v] Patients at this stage can be disfigured due to multiple surgeries to remove CSCC tumors on the head, neck and other parts of the body.[vi] CSCC is the second deadliest skin cancer after melanoma[vii],[viii] and is responsible for the most deaths among non-melanoma skin cancer patients.6
About Sanofi
Sanofi 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.
About Regeneron
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for nearly 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to six FDA-approved treatments and over a dozen product candidates in development, all of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye disease, heart disease, allergic and inflammatory diseases, pain, cancer, infectious diseases and rare diseases.
Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite® antibody technologies and ambitious initiatives such as the Regeneron Genetics Center, one of the largest genetics sequencing efforts in the world
[i] Siegel R, et al. Cancer Statistics, 2017. CA Cancer J Clin. 2017;67:7-30. doi: 10.3322/caac.21387
[ii] Burton KA, et al. Cutaneous Squamous Cell Carcinoma: A Review of High-Risk and Metastatic Disease. Am J Clin Dermatol. 2016;17:491-508. doi:10.1007/s40257-016-0207-3.
[iii] U.S. Food and Drug Administration. Guidance for Industry Expedited Programs for Serious Conditions – Drugs and Biologics. Available at :
http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm358301.pdf. Last Accessed: December 11, 2017.
[iv] Karia PS, Han J, Schmults CD. Cutaneous squamous cell carcinoma: estimated incidence of disease, nodal metastasis, and deaths from disease in the United States, 2012. J Am Acad Dermatol. 2013 Jun;68(6):957-66. doi: 10.1016/j.jaad.2012.11.037. Epub 2013 Feb 1.
[v] Medscape. Cutaneous Squamous Cell Carcinoma. Available at: http://emedicine.medscape.com/article/1965430-overview. Last Accessed: Last Accessed: December 11, 2017.
[vi] Weinberg AS, Ogle CA, Shim EK. Metastatic cutaneous squamous cell carcinoma: an update. Dermatol Surg. 2007;33:885-899.
[vii] Siegel R, et al. Cancer Statistics, 2017. CA Cancer J Clin. 2017;67:7-30. doi: 10.3322/caac.21387
[viii] Burton KA, et al. Cutaneous Squamous Cell Carcinoma: A Review of High-Risk and Metastatic Disease. Am J Clin Dermatol. 2016;17:491-508. doi:10.1007/s40257-016-0207-3.
SOURCE: Sanofi
Post Views: 23
