Positive Neoadjuvant Libtayo® (cemiplimab) Monotherapy Data in Resectable Cutaneous Squamous Cell Carcinoma Presented at ESMO and Published in NEJM

63% combined pathologic response rate for neoadjuvant Libtayo monotherapy in stage II to IV resectable CSCC per primary analysis of a confirmatory Phase 2 trial

TARRYTOWN, N.Y., Sept. 12, 2022 /PRNewswire/ — Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced positive clinical data for an investigational regimen of PD-1 inhibitor Libtayo^® (cemiplimab) as neoadjuvant monotherapy in stage II to IV resectable cutaneous squamous cell carcinoma (CSCC). The data, from the primary analysis of a confirmatory Phase 2 trial, were presented in an oral session at the European Society for Medical Oncology (ESMO) Congress 2022 in Paris and concurrently published in the New England Journal of Medicine (NEJM).

“Regeneron conducted the first pivotal trial of Libtayo in advanced cutaneous squamous cell carcinoma that was unlikely to be curable by surgery or radiation, and we are now studying the utility of Libtayo in earlier stages of this disease. In addition to our neoadjuvant trial, a global Phase 3 trial of Libtayo is ongoing in the adjuvant setting after surgery and radiation for patients at heightened risk for recurrence,” said Israel Lowy, M.D., Ph.D., Senior Vice President, Translational and Clinical Sciences, Oncology at Regeneron. “In the neoadjuvant trial in stage II to IV cutaneous squamous cell carcinoma presented at ESMO and published in NEJM, Libtayo demonstrated greater than 60% response rates per both pathologic and imaging measures. Libtayo may therefore have expanded utility in earlier stages of cutaneous squamous cell carcinoma as a monotherapy to potentially help avoid surgery, and as part of a combination approach with surgery to facilitate function-preserving resections and minimize disfigurement.”

In the confirmatory, multicenter, single-arm Phase 2 trial, 79 patients received up to four fixed doses of Libtayo every 3 weeks prior to surgery, with 62 receiving all 4 doses and 70 undergoing surgery. Patients experienced the following efficacy:

• 63.3% combined pathologic response rate (50 of 79 patients), with 50.6% (40 patients) achieving the primary endpoint of complete pathologic response (0% viable tumor, excluding a null hypothesis of 25%) and 12.7% (10 patients) experiencing a major pathologic response (>0% and ≤10% viable tumor cells) by independent pathologic review.
• 68% objective response rate (ORR) (54 of 79 patients; 5 complete responses [CR] and 49 partial responses [PR]) per local imaging review.

Adverse events (AE) of any grade occurred in 87% of patients, with 17% considered serious. The most common AE was fatigue (n=24). AEs that were ≥grade 3 occurred in 18% of patients. The treatment discontinuation rate due to AEs was 1%, and there was one death due to worsening congestive heart failure considered related to treatment. There were no new Libtayo safety signals.

Patient follow-up in the trial is ongoing to assess disease-free survival. A separate global Phase 3 trial investigating Libtayo in the adjuvant CSCC setting for patients at heightened risk (i.e., due to involvement of multiple lymph nodes, extension of cancer through the lymph node capsule, perineural invasion) for recurrence is enrolling patients.

The potential use of Libtayo described above is investigational, and its safety and efficacy has not been evaluated by any regulatory authority for this indication.

About Regeneron in Oncology
At Regeneron, we’re applying more than three decades of scientific innovation with the goal of developing paradigm-changing therapies for patients with cancer. Our oncology portfolio is built around two foundational approaches – our approved PD-1 inhibitor Libtayo and investigational bispecific antibodies – which are being evaluated both as monotherapies and in combination with emerging therapeutic modalities. Together, they provide us with unique combinatorial flexibility to develop potentially synergistic treatments for a wide range of solid tumors and blood cancers.

If you are interested in learning more about our clinical trials, please contact us (clinicaltrials@regeneron.com or 844-734-6643) or visit our clinical trials website.

About Libtayo
Libtayo is a fully human monoclonal antibody targeting the immune checkpoint receptor PD-1 on T cells and was invented using Regeneron’s proprietary VelocImmune technology. By binding to PD-1, Libtayo has been shown to block cancer cells from using the PD-1 pathway to suppress T-cell activation. In the U.S. and other countries Libtayo is indicated in certain patients with advanced basal cell carcinoma (BCC), advanced cutaneous squamous cell carcinoma (CSCC) and advanced non-small cell lung cancer (NSCLC), as well as in advanced cervical cancer in Canada and Brazil. As of July 1, 2022, Libtayo is developed and marketed globally by Regeneron. 

In the U.S., the generic name for Libtayo in its approved indications 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 (FDA). Outside of the U.S. the generic name of Libtayo in its approved indication is cemiplimab.

The extensive clinical program for Libtayo is focused on difficult-to-treat cancers. Libtayo is currently being investigated in trials as a monotherapy, as well as in combination with either conventional or novel therapeutic approaches for other solid tumors and blood cancers. These potential uses are investigational, and their safety and efficacy have not been evaluated by any regulatory authority.

U.S. FDA-approved Indications Libtayo is a prescription medicine used to treat people with:

• A type of skin cancer called advanced CSCC that has spread or cannot be cured by surgery or radiation.
• A type of skin cancer called BCC:
• • That cannot be removed by surgery (locally advanced BCC) and have received treatment with a hedgehog pathway inhibitor (HHI), or cannot receive treatment with an HHI.
  • That has spread (metastatic BCC) and have received treatment with an HHI, or cannot receive treatment with an HHI. This use is approved based on how many patients responded to treatment and how long they responded. Studies are ongoing to provide additional information about clinical benefit.
• A type of lung cancer called NSCLC. Libtayo may be used as your first treatment when your lung cancer has not spread outside your chest (locally advanced lung cancer) and you cannot have surgery or chemotherapy with radiation, or your lung cancer has spread to other areas of your body (metastatic lung cancer), and your tumor tests positive for high “PD-L1” and your tumor does not have an abnormal “EGFR”, “ALK “or “ROS1” gene.

It is not known if Libtayo is safe and effective in children.

About Regeneron’s VelocImmune Technology
Regeneron’s VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron’s co-Founder, President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite^® technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create approximately one in five of all original, FDA-approved or authorized fully human monoclonal antibodies. This includes REGEN-COV^® (casirivimab and imdevimab), Dupixent^® (dupilumab), Libtayo^® (cemiplimab-rwlc), Praluent^® (alirocumab), Kevzara^® (sarilumab), Evkeeza^® (evinacumab-dgnb) and Inmazeb™ (atoltivimab, maftivimab and odesivimab-ebgn).