On May 30, 2024 Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a clinical stage biopharmaceutical company focused on developing multimodal biological immunotherapies to help patients fight cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to CAN-3110, a next generation oncolytic viral immunotherapy, for the treatment of recurrent high-grade glioma (rHGG) (Press release, Candel Therapeutics, MAY 30, 2024, View Source [SID1234643880]). Glioblastoma (GBM) is the most common and aggressive form of high-grade glioma.
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CAN-3110 was previously granted Fast Track Designation by the FDA for the treatment of rHGG. Candel is currently evaluating CAN-3110 in a multi-institutional phase 1b clinical trial in rHGG. Results from Arm A of the ongoing phase 1b clinical trial in rHGG exploring the clinical and biomarker activity of a single dose administration of CAN-3110 were published in Nature, demonstrating a strong anti-tumoral response associated with extended survival.1 The Company will present data on the feasibility and safety of multiple doses of CAN-3110 in patients with rHGG, supported by the Break Through Cancer Foundation, in a trials-in-progress poster presentation at the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting.
"Building on the momentum of the FDA’s Fast Track Designation, recently granted to this program, the Orphan Drug Designation for CAN-3110 further reinforces the potential of this therapy and underscores the urgent need for novel and effective treatments for patients with rHGG," said Paul Peter Tak, MD, PhD, FMedSci, President and Chief Executive Officer of Candel. "This designation not only reinforces our commitment to offering new hope and potential patient treatment options, but it also enables us to leverage development incentives and accelerate our efforts to evaluate new indications in the clinic. We are continuing our work in the phase 1b clinical trial of CAN-3110 and look forward to sharing further clinical updates in the second half of 2024."
E. Antonio Chiocca, M.D., Ph.D., Chair of the Department of Neurosurgery at Brigham and Women’s Hospital, Professor at Harvard Medical School, and Principal Investigator on the phase 1b clinical trial, said: "We are grateful to the FDA for recognizing the urgent need for new treatments in rHGG. Patients, and their families, affected by this disease, face immense challenges that the standard of care and conventional therapies have failed to adequately address. The early clinical data suggests that CAN-3110’s unique dual mechanism of action, combining oncolysis and immune activation, has the potential to overcome these challenges for rHGG patients."
About Orphan Drug Designation
Orphan Drug Designation is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, defined as one that affects fewer than 200,000 people in the United States. Orphan Drug Designation provides certain financial incentives to support clinical development, and the potential for up to seven years of marketing exclusivity for the product for the designated orphan indication in the United States if the product is ultimately approved for its designated indication.
About CAN-3110
CAN-3110 is a first-in-class, replication-competent herpes simplex virus-1 (HSV-1) oncolytic viral immunotherapy candidate designed with dual activity for oncolysis and immune activation in a single therapeutic. Its activity is designed to be conditional to the expression of Nestin in cancer cells. CAN-3110 is being evaluated in a phase 1b clinical trial in patients with recurrent high-grade glioma (rHGG). In October 2023, the Company announced that Nature published results from this ongoing clinical trial. CAN-3110 was well tolerated with no dose-limiting toxicity reported. In the clinical trial, the investigators observed median overall survival after a single CAN-3110 injection of more than 12 months in this therapy-resistant condition.1 The Company and academic collaborators are currently evaluating the effects of multiple CAN-3110 injections in rHGG, supported by the Break Through Cancer Foundation. CAN-3110 has previously received FDA Fast Track Designation for the treatment of rHGG.
Details on the CAN-3110 ASCO (Free ASCO Whitepaper) poster are as follows:
The trials-in-progress poster presentation will focus on cohort C of the ongoing phase 1b clinical trial of CAN-3110 in patients with rGBM, the most common form of rHGG. Previously presented data showed the ability of a single CAN-3110 injection to double median overall survival (mOS) in the rGBM population, as compared to contemporary control cohorts. Patients presenting with seropositivity to HSV1, reached mOS of 14 months, largely exceeding expected survival of 6 to 9 months or less for this population.
In cohort C, supported by the Break Through Cancer Foundation, two cohorts of 12 patients will receive up to six injections of CAN-3110 over a four-month period. Cohort C is currently exploring the safety and tolerability of CAN-3110 in patients with rGBM. Patients in cohort C are treated with up to six doses of CAN-3110 delivered by stereotactic injections on days 0, 15, 30, 60, 90 & 120, along with concomitant biopsies over the four-month treatment period.
Two sub-cohorts (1&2) of patients who will receive 1×107 pfu or 1×108 pfu per injection of CAN-3110 have been planned for six patients per cohort, using a Bayesian optimal interval (BOIN) design for dose ranging.
Six patients have accrued, completing cohort 1; no dose-limiting toxicities or severe adverse events were observed.
More than 300 core biopsies were obtained from all six patients across the planned time points.
Biopsies were processed for "-omic" analyses, including single-cell RNA sequencing, proteomics/phophoproteomic/immunopeptidomics, metabolomics, spatial transcriptomics, and cell profiling.
ASCO Presentation details are as follows:
Trials-in-Progress Poster Presentation Title: Longitudinal stereotactic injections of oncolytic immunoactivating rQNestin34.5v.2 (CAN-3110) with concomitant biopsies for "-omic" analyses in recurrent glioblastoma (GBM)
Presenter: David A. Reardon, MD, Professor of Medicine at Harvard Medical School; Clinical Director, Center for Neuro-Oncology at Dana Farber Cancer Institute
Session Title: Poster Session – Central Nervous System Tumors
Session Date/Time: Saturday, June 1, 2024; 9:00 AM – 12:00 PM CT