FDA Designates CAN-3110 as Orphan Drug for Recurrent High-Grade Glioma

Candel Therapeutics, Inc., a biopharmaceutical company specializing in multimodal biological immunotherapies for cancer treatment, announced that the U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation to its oncolytic viral immunotherapy, CAN-3110. This new designation targets CAN-3110 for the treatment of recurrent high-grade glioma (rHGG), which includes the particularly aggressive glioblastoma (GBM).

CAN-3110 had already received Fast Track Designation from the FDA for rHGG treatment. Currently, this therapy is under investigation in a phase 1b clinical trial across multiple institutions. Earlier results, published in Nature, demonstrated that a single dose of CAN-3110 elicited a strong anti-tumoral response and significantly extended survival rates in patients with rHGG.

Candel Therapeutics plans to present data on the safety and feasibility of administering multiple doses of CAN-3110 at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting. Supported by the Break Through Cancer Foundation, this presentation will provide insights into the therapy's clinical and biomarker activities.

The Orphan Drug Designation not only highlights the potential of CAN-3110 but also underscores the urgent need for innovative treatments for rHGG. This designation offers significant developmental financial incentives and could grant up to seven years of marketing exclusivity in the U.S., contingent on regulatory approval.

Paul Peter Tak, MD, PhD, President and CEO of Candel Therapeutics, expressed his enthusiasm for the recognition by the FDA, emphasizing the importance of developing new treatment options for patients suffering from rHGG. This designation allows the company to accelerate its clinical evaluation and explore new indications for CAN-3110.

Dr. E. Antonio Chiocca, Chair of the Department of Neurosurgery at Brigham and Women’s Hospital and a principal investigator for the phase 1b trial, highlighted the severe challenges faced by rHGG patients and their families. He pointed out that conventional therapies have not provided adequate solutions, but early clinical data indicate that CAN-3110’s dual mechanism of action—combining oncolysis and immune activation—may offer new hope.

Orphan Drug Designation by the FDA is reserved for drugs and biologics intended to treat rare diseases, classified as affecting fewer than 200,000 individuals in the United States. This designation offers various financial incentives to support clinical development and can provide up to seven years of market exclusivity once the product is approved for its designated indication.

CAN-3110 is a first-in-class, replication-competent herpes simplex virus-1 (HSV-1) oncolytic viral immunotherapy. It features dual activity designed to conditionally express within cancer cells, leading to both oncolysis and immune system activation. The phase 1b clinical trial of CAN-3110 has shown promising results, with the therapy being well-tolerated and demonstrating no dose-limiting toxicities. Patients experienced a median overall survival of more than 12 months following a single injection, a significant improvement in this therapy-resistant condition.

Candel’s presentation at the 2024 ASCO Annual Meeting will focus on cohort C of the ongoing phase 1b clinical trial, which explores the safety and tolerability of multiple CAN-3110 doses in patients with recurrent glioblastoma. The cohort includes two groups of 12 patients each, receiving up to six injections over four months. Thus far, no severe adverse events have been reported, and over 300 core biopsies from the patients have been collected for comprehensive "omic" analyses, including single-cell RNA sequencing and proteomics.

Candel Therapeutics continues to advance its clinical programs, utilizing its multimodal biological immunotherapy platforms to develop novel treatments for cancer. The company's lead product candidates, CAN-2409 and CAN-3110, are under investigation in various clinical trials targeting different types of cancer, with the aim of harnessing the body's immune response to combat malignancies more effectively.