Paris and Tarrytown, NY – May 5, 2020 - Topline data for a pivotal, single-arm, open-label trial for Sanofi and Regeneron’s PD-1 inhibitor Libtayo® (cemiplimab) in patients with advanced basal cell carcinoma (BCC) who had progressed on or were intolerant to prior hedgehog pathway inhibitor (HHI) therapy were announced today. Libtayo demonstrated clinically meaningful and durable responses in this group of patients for whom there are no approved treatments. Sanofi and Regeneron plan regulatory submissions in 2020.
BCC is a skin cancer and is the most common cancer worldwide, with approximately two million new cases diagnosed every year in the U.S. alone. While the vast majority of BCCs are caught early and cured with surgery or radiation, a small proportion of tumors can become advanced and penetrate deeper into surrounding tissues (locally advanced), which is more difficult to treat. Approximately 20,000 U.S. patients have advanced BCC and it is estimated that about 3,000 die each year. BCC marks the second non-melanoma skin cancer for which Libtayo has demonstrated first-in-class data and follows its initial U.S. approval in advanced cutaneous squamous cell carcinoma (CSCC) in 2018.
In the trial, the objective response rate (ORR) for patients (n=84) with locally advanced disease was 29% (95% CI: 19%-40%), with an estimated duration of response (DOR) exceeding one year in 85% of responders. The durable disease control rate (DCR —response or stable disease lasting at least 6 months) was 60% (95% CI: 48%-70%). In a preliminary analysis of patients (n=28) with metastatic disease, the ORR was 21% (95% CI: 8%-41%), with an estimated DOR exceeding one year in 83% of responders. The durable DCR was 46% (95% CI: 28%-66%). All data were assessed by an independent central review. Data are expected to continue to evolve with further follow-up across both patient groups.
“While PD-1 inhibitors have transformed the outlook for many patients with melanoma, progress for patients with non-melanoma skin cancers has not been as rapid,” said Peter C. Adamson, M.D, Global Head of Oncology Development at Sanofi. “We are continuing to address this unmet need by first bringing Libtayo to patients with advanced cutaneous squamous cell carcinoma, and now, with this second trial, as a potential therapy for patients with advanced basal cell carcinoma. These important new results further demonstrate Libtayo’s potential in patients with difficult-to-treat, non-melanoma skin cancers.”
There were no new safety signals in this trial. Among the 132 patients assessed for safety (84 locally advanced and 48 metastatic), 95% of patients experienced an adverse event (AE), 32% had a serious AE and 13% discontinued due to an AE. There were 10 deaths in the locally advanced group and nine deaths in the metastatic group; none of the deaths were considered treatment-related. Sanofi and Regeneron will present additional trial findings at an upcoming medical meeting.
“Libtayo is being investigated as a monotherapy treatment and as a foundation therapy for combinations with novel therapeutic approaches being developed by Regeneron and our collaborators,” Israel Lowy, M.D., Ph.D., Senior Vice President, Translational and Clinical Sciences, Oncology at Regeneron. “These data in advanced BCC provide the third instance where Libtayo monotherapy has demonstrated robust and clinically meaningful outcomes in advanced cancer, and follows last week’s announcement in advanced non-small cell lung cancer where the pivotal trial was stopped early for positive overall survival.”
In this ongoing global Phase 2 trial, patients received Libtayo 350 mg intravenously every three weeks for up to 93 weeks or until disease progression, unacceptable toxicity, withdrawal of consent or confirmed complete response. ORR is the primary endpoint and key secondary endpoints include overall survival, progression-free survival, duration of response, safety and quality of life.
Libtayo is being jointly developed and commercialized by Sanofi and Regeneron under a global collaboration agreement.
The use of Libtayo to treat advanced BCC is investigational and the safety and efficacy have not been evaluated by any regulatory authority.
About Libtayo
Libtayo is a fully-human monoclonal antibody targeting the immune checkpoint receptor PD-1 on T-cells. By binding to PD-1, Libtayo has been shown to block cancer cells from using the PD-1 pathway to suppress T-cell activation.
Libtayo is the first and only immunotherapy approved in the U.S., EU, and other countries for adults with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation. In the U.S., the generic name for Libtayo in its approved indication is cemiplimab-rwlc, with rwlc as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the U.S. Food and Drug Administration.
The extensive clinical program for Libtayo is focused on difficult-to-treat cancers. In skin cancer, this includes trials in adjuvant and neoadjuvant CSCC. Libtayo is also being investigated in potentially registrational Phase 3 trials in non-small lung cancer and cervical cancer, as well as in trials combining Libtayo with novel therapeutic approaches for both solid tumors and blood cancers. These potential uses are investigational, and their safety and efficacy have not been evaluated by any regulatory authority.
IMPORTANT SAFETY INFORMATION AND INDICATION FOR U.S. PATIENTS
What is Libtayo?
Libtayo is a prescription medicine used to treat people with a type of skin cancer called cutaneous squamous cell carcinoma (CSCC) that has spread or cannot be cured by surgery or radiation.
It is not known if Libtayo is safe and effective in children.
What is the most important information I should know about Libtayo?
Libtayo is a medicine that may treat a type of skin cancer by working with your immune system. Libtayo can cause your immune system to attack normal organs and tissues in any area of your body and can affect the way they work. These problems can sometimes become severe or life-threatening and can lead to death. These problems may happen anytime during treatment or even after your treatment has ended.
Call or see your healthcare provider right away if you develop any symptoms of the following problems or these symptoms get worse:
- Lung problems (pneumonitis). Signs and symptoms of pneumonitis may include new or worsening cough, shortness of breath, and chest pain.
- Intestinal problems (colitis) that can lead to tears or holes in your intestine. Signs and symptoms of colitis may include diarrhea (loose stools) or more frequent bowel movements than usual; stools that are black, tarry, sticky or that have blood or mucus; and severe stomach-area (abdomen) pain or tenderness.
- Liver problems (hepatitis). Signs and symptoms of hepatitis may include yellowing of your skin or the whites of your eyes, severe nausea or vomiting, pain on the right side of your stomach area (abdomen), drowsiness, dark urine (tea colored), bleeding or bruising more easily than normal, and feeling less hungry than usual.
- Hormone gland problems (especially the adrenal glands, pituitary, thyroid and pancreas). Signs and symptoms that your hormone glands are not working properly may include headaches that will not go away or unusual headaches, rapid heartbeat, increased sweating, extreme tiredness, weight gain or weight loss, dizziness or fainting, feeling more hungry or thirsty than usual, hair loss, feeling cold, constipation, deeper voice, very low blood pressure, urinating more often than usual, nausea or vomiting, stomach-area (abdomen) pain, and changes in mood or behavior, such as decreased sex drive, irritability, or forgetfulness.
- Kidney problems, including nephritis and kidney failure. Signs of these problems may include decrease in your amount of urine, blood in your urine, swelling in your ankles, and loss of appetite.
- Skin problems. Signs of these problems may include rash, itching, skin blistering, and painful sores or ulcers in the mouth, nose, throat, or genital area.
- Problems in other organs. Signs of these problems may include headache, tiredness or weakness, sleepiness, changes in heartbeat (such as beating fast, seeming to skip a beat, or a pounding sensation), confusion, fever, muscle weakness, balance problems, nausea, vomiting, stiff neck, memory problems, seizures (encephalitis), swollen lymph nodes, rash or tender lumps on skin, cough, shortness of breath, vision changes, or eye pain (sarcoidosis), seeing or hearing things that are not there (hallucinations), severe muscle weakness, low red blood cells (anemia), bruises on the skin or bleeding, and changes in eyesight.
- Rejection of a transplanted organ. Your doctor should tell you what signs and symptoms you should report and monitor you, depending on the type of organ transplant that you have had.
- Infusion (IV) reactions that can sometimes be severe and life-threatening. Signs of these problems may include chills or shaking, itching or rash, flushing, shortness of breath or wheezing, dizziness, fever, feeling of passing out, back or neck pain, and facial swelling.
Getting medical treatment right away may help keep these problems from becoming more serious.
Your healthcare provider will check you for these problems during your treatment with Libtayo. Your healthcare provider may treat you with corticosteroid or hormone replacement medicines. Your healthcare provider may delay or completely stop treatment if you have severe side effects.
Before you receive Libtayo, tell your healthcare provider about all your medical conditions, including if you:
- have immune system problems such as Crohn’s disease, ulcerative colitis, or lupus;
- have had an organ transplant;
- have lung or breathing problems;
- have liver or kidney problems;
- have diabetes;
- are pregnant or plan to become pregnant; Libtayo can harm your unborn baby.
Females who are able to become pregnant:
- Your healthcare provider will give you a pregnancy test before you start treatment.
- You should use an effective method of birth control during your treatment and for at least 4 months after your last dose of Libtayo. Talk with your healthcare provider about birth control methods that you can use during this time.
- Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with Libtayo.
- are breastfeeding or plan to breastfeed. It is not known if Libtayo passes into your breast milk. Do not breastfeed during treatment and for at least 4 months after the last dose of Libtayo.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
The most common side effects of Libtayo include tiredness, rash, and diarrhea. These are not all the possible side effects of Libtayo. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. You may also report side effects to Regeneron Pharmaceuticals and Sanofi at 1-877-542-8296.
For more information, please see full Prescribing Information, including Medication Guide.
About Regeneron Pharmaceuticals, Inc.
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for over 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to seven FDA-approved treatments and numerous product candidates in development, all of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, pain, infectious diseases and rare diseases.
Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite® technologies, such as VelocImmune which uses unique genetically-humanized mice to produce optimized fully-human antibodies and bispecific antibodies, and through ambitious research initiatives such as the Regeneron Genetics Center, which is conducting one of the largest genetics sequencing efforts in the world.
For additional information about the company, please visit www.regeneron.com or follow @Regeneron on Twitter.
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.
Sanofi, Empowering Life
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