FDA Grants Orphan Drug Designation for CAN-3110 for the Treatment of Recurrent High-Grade Glioma
The FDA has granted Orphan Drug Designation to CAN-3110, Candel Therapeutics' oncolytic viral immunotherapy for treating recurrent high-grade glioma (rHGG). This designation offers developmental incentives and up to 7 years of U.S. market exclusivity upon approval. Previously, CAN-3110 received Fast Track Designation for rHGG. The company is conducting a multi-institutional Phase 1b clinical trial, with initial results indicating strong anti-tumoral responses and extended survival. Candel will present further data on CAN-3110's feasibility and safety at the 2024 ASCO Annual Meeting. CEO Paul Peter Tak emphasized the potential of CAN-3110 and the company's commitment to developing effective treatments for rHGG.
- FDA Orphan Drug Designation provides developmental financial incentives and up to 7 years of marketing exclusivity if approved.
- CAN-3110 previously received Fast Track Designation for the treatment of rHGG, indicating regulatory support.
- Phase 1b clinical trial results published in Nature showed strong anti-tumoral response and extended survival with CAN-3110.
- Upcoming presentation at the 2024 ASCO Annual Meeting will feature data on the feasibility and safety of multiple doses of CAN-3110.
- The efficacy and safety of CAN-3110 are still under investigation, with results yet to be fully confirmed.
- Potential for competition and market challenges remains if CAN-3110 does not meet efficacy or safety benchmarks.
- Financial and developmental incentives are contingent upon FDA approval, which is not guaranteed.
Insights
The FDA granting Orphan Drug Designation to CAN-3110 is a significant milestone for Candel Therapeutics. This designation offers developmental financial incentives and potential marketing exclusivity for up to 7 years in the United States if approved. Recurrent high-grade glioma (rHGG) is a particularly challenging form of brain cancer, with limited effective treatment options. This designation underscores the FDA's recognition of the urgency for new therapies in this area.
The phase 1b clinical trial data published in Nature showed a strong anti-tumoral response, which is promising. The key here is the dual mechanism of action - combining oncolysis and immune activation, which could provide a novel approach to treating rHGG. Investors should watch for further clinical updates in the second half of 2024 as these will be critical in assessing the future potential of CAN-3110.
From a financial perspective, the Orphan Drug Designation can be a game-changer for Candel Therapeutics. This status not only provides financial incentives such as tax credits for clinical testing, waiver of FDA fees and potential grant funding but also significantly enhances the company's ability to secure exclusive marketing rights for up to 7 years. These benefits could improve the company's financial stability and investor confidence, assuming successful trial outcomes.
Furthermore, the enhancement of the company's pipeline with a potentially effective treatment for a high-need condition could attract more investment and partnerships. However, investors should be aware that the actual financial impact will be contingent upon successful clinical trial outcomes and subsequent FDA approval. The next phase of clinical data in the second half of 2024 will be important for making more definitive financial projections.
- FDA Orphan Designation provides CAN-3110 certain developmental financial incentives, with potential for up to 7 years of marketing exclusivity in the United States, if approved
- CAN-3110 phase 1b data on the feasibility and safety of multiple doses of CAN-3110 will be featured in poster presentation at 2024 ASCO Annual Meeting
NEEDHAM, Mass., May 30, 2024 (GLOBE NEWSWIRE) -- 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, today announced 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). Glioblastoma (GBM) is the most common and aggressive form of high-grade glioma.
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 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 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 1x107 pfu or 1x108 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
About Candel Therapeutics
Candel is a clinical stage biopharmaceutical company focused on developing off-the-shelf multimodal biological immunotherapies that elicit an individualized, systemic anti-tumor immune response to help patients fight cancer. Candel has established two clinical stage multimodal biological immunotherapy platforms based on novel, genetically modified adenovirus and herpes simplex virus (HSV) gene constructs, respectively. CAN-2409 is the lead product candidate from the adenovirus platform and is currently in ongoing clinical trials in non-small cell lung cancer (NSCLC) (phase 2), borderline resectable PDAC (phase 2), and localized, non-metastatic prostate cancer (phase 2 and phase 3). CAN-3110 is the lead product candidate from the HSV platform and is currently in an ongoing phase 1b clinical trial in rHGG. Finally, Candel’s enLIGHTEN™ Discovery Platform is a systematic, iterative HSV-based discovery platform leveraging human biology and advanced analytics to create new viral immunotherapies for solid tumors.
For more information about Candel, visit: www.candeltx.com
Forward-Looking Statements
This press release includes certain disclosures that contain “forward-looking statements,” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, express or implied statements regarding the timing and advancement of development programs, including the timing and availability of additional data, key data readout milestones, and expectations regarding the therapeutic benefit of the Company’s programs, including the potential for CAN-3110 to extend survival of patients with rHGG; and expectations regarding the potential benefits conferred by Orphan Drug Designation and Fast Track Designation. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those risks and uncertainties related to the timing and advancement of development programs; the Company’s ability to continue as a going concern; expectations regarding the therapeutics benefit of the Company’s programs; that final data from the Company’s pre-clinical studies and completed clinical trials may differ materially from reported interim data from ongoing studies and trials; the Company’s ability to efficiently discover and develop product candidates; the Company’s ability to obtain and maintain regulatory approval of product candidates; the Company’s ability to maintain its intellectual property; the implementation of the Company’s business model, including strategic plans for the Company’s business and product candidates, and other risks identified in the Company’s filings, with the U.S. Securities and Exchange Commission (SEC) including the Company’s most recent Quarterly Report on Form 10-Q filed with the SEC, and subsequent filings with the SEC. The Company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. The Company disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions, or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the Company’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.
Investor Contact:
Theodore Jenkins
VP, Investor Relations and Business Development
Candel Therapeutics, Inc
tjenkins@candeltx.com
Media Contact:
Kyle Evans
ICR Westwicke
CandelPR@westwicke.com
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[1] Ling AL, et al. Nature. 2023;623(7985):157-166.
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