Sanofi and Regeneron's Libtayo Shows Benefit in Basal Cell Carcinoma

Basal cell carcinoma cancer cells 

Paris-based Sanofi and Tarrytown, New York-based Regeneron Pharmaceuticals announced topline data for their pivotal Phase II trial of Libtayo (cemiplimab) in advanced basal cell carcinoma (BCC) patients that had progressed on or could not tolerate previous hedgehog pathway inhibitor (HHI) therapies. Libtayo is a PD-1 checkpoint inhibitor.

The drug showed clinically meaningful and durable responses in the patients. The companies expect to make regulatory submissions this year.

BCC is a skin cancer, the most common cancer globally. About 2 million cases are diagnosed each year in the U.S. Most are diagnosed early and cured with surgery or radiation, but those that advanced and metastasize are difficult to treat.

“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, 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.”

Libtayo is approved in the U.S., Europe and elsewhere for adults with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation. The drug program is focused on difficult-to-treat cancers.

In the clinical trial, the objective response rate (ORR) for patients with locally advanced disease was 29%, with an estimated duration of response (DOR) greater than one year in 85% of responders. Durable disease control rate (DCR), which is response or stable disease that lasts at least six months, was 60%.

Preliminary analysis of patients with metastatic disease had an ORR of 21%, an estimated DOR exceeding one year in 83% of responders, and a durable DCR of 46%.

In the trial, patients received 350 mg of the drug intravenously every three weeks for up to 93 weeks or until the disease progressed, there was unacceptable toxicity, withdrawal of consent or confirmed complete response. The ORR is the primary endpoint. Key secondary endpoints include overall survival, progression-free survival, duration of response, safety and quality of life.

“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,” said Israel Lowy, senior vice president, Translational and Clinical Sciences, Oncology at Regeneron. “These data in advanced basal cell carcinoma 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.”

Sanofi and Regeneron lag in their checkpoint inhibitor programs, with the dominant players being Merck’s Keytruda and Bristol Myers Squibb’s Opdivo.

Regeneron also reported first-quarter results today, with total revenues up 33% from $1.373 billion in the first quarter 2019 to $1.828 billion this quarter. Net income also climbed 36% from $461 million last year to $625 million this year.

The company’s revenues are driven by its Eylea, Dupixent and Libtayo in the U.S., while it continues to broaden its immuno-oncology platform. It is planning regulatory submissions for Libtayo in basal cell carcinoma but also in non-small cell lung cancer.