On March 13, 2025 Intensity Therapeutics, Inc. ("Intensity" or "the Company") (Nasdaq: INTS), a late-stage clinical biotechnology company focused on the discovery and development of proprietary, novel immune-based intratumoral cancer therapies designed to kill tumors and increase immune system recognition of cancers, reported 2024 year-end financial results and provides a corporate update (Press release, Intensity Therapeutics, MAR 13, 2025, View Source [SID1234651129]).

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Corporate Update

INVINCIBLE-3 Study: Phase 3 open-label, randomized study testing INT230-6 as monotherapy compared to the standard of care ("SOC") drugs in second and third line treatment for certain soft tissue sarcoma subtypes. The INVINCIBLE-3 Study is expected to enroll 333 patients and initiate sites in eight countries. This study has been authorized by the US FDA, Health Canada, the European Medicines Authority (for France, Germany, Italy, Poland and Spain), and Australia’s Therapeutics Goods Administration. The primary endpoint in the INVINCIBLE-3 Study is overall survival.

In July 2024, the Company initiated and dosed its first patient in the INVINCIBLE-3 Study. The trial is actively enrolling patients across the US, Canada, Europe and Australia. Up to 60 sarcoma-focused institutions are expected to participate from these regions. The Company has contracted 32 sites with 25 patients screened to date. The Company expects to complete enrollment in the first half of 2026.

INVINCIBLE-4 Study: Phase 2 randomized open-label, multicenter study to analyze the clinical activity, safety, and tolerability of INT230-6 given before administration of the SOC treatment in patients with early-stage, operable triple-negative breast cancer ("TNBC") and SOC alone. The primary endpoint is the change in the pathological complete response rate for the combination compared to the SOC alone. The INVINCIBLE-4 Study is expected to enroll approximately 54 patients in Switzerland and France.

In October 2024, in collaboration with The Swiss Group for Cancer Research SAKK ("SAKK"), the Company initiated and dosed its first patient in Switzerland in the INVINCIBLE-4 Study. To date, the Company has activated eight sites in Switzerland and treated several patients. The Company expects to activate additional sites in Switzerland and France in the first half of 2025 and complete enrollment by the end of the first quarter of 2026.

"In 2024, Intensity Therapeutics finalized both Phase 3 and Phase 2 protocols, engaged leading hospitals around the world, and obtained regulatory authorization to recruit patients in 9 countries to initiate treatment," stated Lewis H. Bender, Intensity Founder, President, and CEO. "Our programs were again selected for presentation at major sarcoma and breast cancer societies. Many of the best sarcoma treatment centers from the US, Canada, Europe and Australia are either participating now or in contract discussions. For our breast cancer trial, our partners at SAKK have recruited interest by the leading hospitals in Switzerland and France to participate. Physicians are screening patients at an increasing rate. We believe in the potential for our drug to have a positive impact on the lives of metastatic sarcoma and presurgical breast cancer patients around the world, who so desperately need improved alternatives to current therapies."

2024 Year End Financial Results

Research and development expenses were $10.5 million for the year ended December 31, 2024, compared to $4.8 million for the year ended December 31, 2023. The increase was primarily due to an increase of $5.6 million in the INVINCIBLE-3 Study in 2024, in which we enrolled our first patient in the third quarter of 2024, and to a lesser extent, an increase of $0.5 million in the INVINCIBLE-4 Study, in which we enrolled and dosed our first patient in the fourth quarter of 2024. These increases were partially offset by a decrease of $1.1 million in our IT-01 Study due to the completion of enrollment in this study in mid-2022 and the completion of study-related costs in 2023. Research and development also increased due to higher salary, benefits, and stock-based compensation.

General and administrative expenses were $6.1 million for the year ended December 31, 2024, compared to $3.5 million for the year ended December 31, 2023. The increase was primarily due to increased expenses related to salary, benefits and stock-based compensation, higher legal and consulting fees, and higher directors and officers insurance.

Overall, net loss was $16.3 million for the year ended December 31, 2024, compared to a net loss of $10.5 million for the year ended December 31, 2023.

As of December 31, 2024, cash and cash equivalents totaled $2.6 million.

About INT230-6
INT230-6, Intensity’s lead proprietary investigational product candidate, is designed for direct intratumoral injection. INT230-6 was discovered using Intensity’s proprietary DfuseRx℠ technology platform. The drug is comprised of two proven, potent anti-cancer agents, cisplatin and vinblastine, and a penetration enhancer molecule (SHAO) that helps disperse potent cytotoxic drugs throughout tumors for diffusion into cancer cells. These agents remain in the tumor, resulting in a favorable safety profile. In addition to local disease control and direct tumor killing, INT230-6 causes a release of a bolus of neoantigens specific to the malignancy, leading to immune system engagement and systemic anti-tumor effects. Importantly, these effects are mediated without immunosuppression which often occurs with systemic chemotherapy.

On March 13, 2025 IN8bio, Inc. (Nasdaq: INAB), a clinical-stage biopharmaceutical company developing innovative gamma-delta T cell therapies, reported financial results and business highlights for the fourth quarter and full-year ended December 31, 2024 (Press release, In8bio, MAR 13, 2025, View Source [SID1234651128]).

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William Ho, Chief Executive Officer and co-founder of IN8bio, commented, "In 2024, IN8bio made significant progress advancing its pipeline of gamma-delta T cell therapies and positioning the Company for long-term success. The INB-100 program continues to demonstrate 100% long term durable response rates as of January 17, 2025, reinforcing its potential to help treat and maintain remissions in high-risk AML patients." Mr. Ho continued, "We have also disclosed our novel INB-600 platform, which we believe has the potential to reshape the T cell engager landscape by potentially improving both the durability of response and safety compared to existing CD3 targeting TCE therapies. Throughout 2025, we remain focused on driving innovation, advancing our clinical programs and delivering value to patients and our stakeholders."

Key Highlights

Announced INB-600 Platform & INB-619, a Novel Preclinical Gamma-Delta TCE

Introduced INB-619, a proprietary next generation gamma-delta TCE targeting CD19 for potential use in oncology and autoimmune diseases (March 2025)
Demonstrated potent B-cell depletion and sustained gamma-delta T cell expansion in preclinical studies, offering commercial and scientific advantages by potentially improving durability and safety over existing CD3 targeting TCE therapies
Demonstrated Additional Clinical Progress with INB-100 (Allogeneic Gamma-Delta T Cell Therapy for AML & Leukemias) for High-Risk AML

Presented new Phase 1 data showing durable remissions with 100% of treated AML patients remaining relapse-free (February 2025)
Observed favorable safety profile with no cytokine release syndrome (CRS) or neurotoxicity (ICANs) reported to date
Presented expanded clinical data at 2025 Tandem Meetings reinforcing potential of INB-100 to significantly reduce post-transplant relapse in high-risk AML patients relative to historical real-world data
Presented Continued Durable Remissions in Phase 1 Trial of INB-200 (Autologous, Drug-Resistant Gamma-Delta T Cell Therapy for Glioblastoma)

Presented updated data in a plenary oral presentation at the 2024 Society for Neuro-Oncology (SNO) Annual Meeting, showing the majority of patients exceeded their expected progression free survival based on age and tumor status
Highlighted potential long-term benefits of INB-200. As of October 18, 2024, five patients remained alive, three patients had returned to work, and one IDH-mutant, grade 4 glioma patient remained progression free for an impressive 40.5 months post treatment
Strategically Optimized Pipeline and Launched Operational Efficiency Initiatives

Paused enrollment in the Phase 2 INB-400 glioblastoma program – despite compelling data – to focus resources while exploring partnership and alternative funding opportunities
Implemented cost-saving measures to extend cash runway and prioritize pipeline development
Raised approximately $16.6 million in gross proceeds through various equity offerings, including through our at-the-market ("ATM") offering program and private placements, during 2024 through February 2025, extending the Company’s cash runway into March 2026
Upcoming Anticipated Pipeline Milestones and Important Events

INB-100: For the potential treatment of high-risk leukemias including AML

Accelerating patient enrollment in expansion cohort of ongoing Phase 1 clinical trial including the inclusion of an additional investigational center
Aim to complete enrollment into the expansion cohort in 2025. Guidance from the U.S. Food and Drug Administration (FDA) confirmed relapse-free survival (RFS) in AML patients as an acceptable primary endpoint for a potential pivotal randomized control trial
Expect to present updated clinical data in the second half of 2025
Advancing preparations for potential registrational Phase 2 trial with possible IND submission anticipated in 2026 based on receipt of additional funding
INB-200 and INB-400 (Autologous Gamma-Delta T Cell Therapy for Glioblastoma & Solid Tumors):

Continue to evaluate early clinical data for signs of efficacy and durability
Report additional findings at upcoming medical meetings in 2025
Seek additional funding sources to advance allogeneic product for glioblastoma and other solid tumors
INB-600 and INB-619 (Gamma-Delta T Cell Engager Targeting CD19 – Oncology & Autoimmune Diseases):

Present preclinical data evaluating potency, expansion potential, and safety profile at medical meetings in the second quarter of 2025
Plan to advance towards IND-enabling studies with additional funding to support future clinical development
Exploring partnership and collaboration options to accelerate development of the platform
Fourth Quarter and Full Year 2024 Financial Highlights

Research and Development (R&D) expenses: R&D expenses were $3.8 million for the three months ended December 31, 2024, compared with $4.5 million for the comparable prior year period. R&D expenses were $17.2 million for the year ended December 31, 2024, compared with $16.8 million in the prior year. The change was primarily due to increased clinical trial-related activities for the INB-100 and INB-400 programs and was partially offset by a decrease in personnel-related costs and facility-related and other expenses, in each case as a result of the Company’s pipeline prioritization announced in September 2024.
General and administrative (G&A) expenses: G&A expenses were $2.6 million for the three months ended December 31, 2024, compared with $3.1 million for the comparable prior year period. G&A expenses were $12.6 million for the year ended December 31, 2024, compared with $13.5 million in the prior year. The change was primarily due to cost savings related to director and officer insurance premiums, professional services, and personnel-related costs, partially offset by an increase in legal and consulting expenses.
Severance and related charges: Severance and related charges were $1.1 million for the year ended December 31, 2024, compared with zero in the prior year. These were one-time costs related to the September 2024 workforce reduction, including non-cash stock-based compensation expense and severance payments.
Net loss: The company reported a net loss of $6.4 million, or $0.08 per basic and diluted common share, for the three months ended December 31, 2024, compared with a net loss of $7.6 million, or $0.22 per basic and diluted common share, for the comparable prior year period. For the year ended December 31, 2024, net loss was $30.7 million, or $0.57 per basic and diluted common share, compared with a net loss of $30.0 million, or $1.00 per basic and diluted common share, for the prior year.
Cash position: As of December 31, 2024, the Company had cash of $11.1 million as of December 31, 2024, compared with $21.3 million as of December 31, 2023. Subsequently, in February 2025, the Company sold common stock under its ATM offering program and had Series C warrants exercised for aggregate net proceeds of $4.1 million.

On March 13, 2025 ImmunityBio, Inc. (NASDAQ: IBRX), a leading immunotherapy company, reported U.S. Urology Partners, one of the nation’s largest independent providers of urology and related specialty services, is one of the first providers to participate in ImmunityBio’s Expanded Access Program (EAP) for recombinant Bacillus Calmette-Guérin (rBCG) to address the current shortage of TICE BCG in the U.S (Press release, ImmunityBio, MAR 13, 2025, View Source [SID1234651127]).

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"The shortage of BCG has real consequences, especially in many rural communities across the country where supply shortages have impacted the treatment of patients with bladder cancer," said Christopher M. Pieczonka, M.D., Chief Executive Officer and Director of Clinical Research, Associated Medical Professionals, an affiliate of U.S. Urology Partners. "I’m pleased U.S. Urology is at the forefront of offering rBCG and would like to thank ImmunityBio for their initiative to seek and secure authorization from the FDA for expanded access to this vital BCG alternative."

Supply shortages of TICE BCG in the U.S. have become a significant impediment to the treatment of bladder cancer patients. In a recent Sermo survey of 100 U.S. urologists, 57 percent indicated they were unable to treat patients in the last 12 months due to a lack of access to TICE BCG. Providers who are unable to source TICE BCG are able to access rBCG through the ImmunityBio EAP.

"It is gratifying to see the rapid uptake and interest in the rBCG Expanded Access Program, which was authorized by the FDA just recently," added Dr. Patrick Soon-Shiong, Founder, Executive Chairman and Global Chief Scientific and Medical Officer of ImmunityBio. "Participation by a major national provider like U.S. Urology Partners is a testament to the critical importance of rBCG and the need for an alternative supply of this essential medicine."

"This is an incredibly exciting opportunity to expand the BCG supply to allow more patients to receive the full course of their cancer treatment, saving bladders and extending lives," said Dr. Eugene B. Cone, Co-Director of Clinical Research, Urology of Indiana, an affiliate of U.S. Urology.

The Serum Institute of India, ImmunityBio’s collaboration partner, is the world’s largest manufacturer of vaccines by volume. In bladder cancer clinical trials in Europe, the recombinant BCG vaccine has demonstrated potent immunogenicity with CD8+ and CD4+ T cell stimulation and improved safety compared to earlier BCG strains and formulations. Clinical research organization, Anova Enterprises, Inc. (Anova) is helping ImmunityBio manage the rBCG EAP.

"Constrained BCG supply has improved stewardship of a limited resource but has come at a high cost to our patients," said Dr. Chad Reichard, Co-Director of Clinical Research, Urology of Indiana. "rBCG represents a long overdue potential alternative but does not abrogate responsibility to ongoing diligence and discipline in BCG delivery."

About Recombinant BCG (rBCG)

BCG is a benign bacterium originally developed as a live vaccine against tuberculosis (TB). It is based on the well-known Mycobacterium bovis (M. bovis) Bacillus Calmette-Guérin (BCG) strain. It has been in use since 1921 and administered to more than 4 billion individuals worldwide. BCG given via intravesical instillation (delivery to the bladder via a catheter) has been the standard of care for patients with non-muscle invasive bladder cancer (NMIBC) since 1977. BCG induces an immune response in the bladder in proximity to the cancer cells, leading to clearance of the cancer in many patients.

Two gene modifications have been implemented in rBCG to improve its immunogenicity and safety in comparison to earlier strains and formulations of BCG. Recombinant rBCG has completed Phase 1/2 human clinical studies in Europe as an immunotherapy in patients with NMIBC. The findings from those studies demonstrate rBCG is well-tolerated when administered intravesically with a safety profile similar to placebo, and reduced rates of adverse events observed in earlier strains and formulations of BCG.

Supportive clinical data of rBCG as a TB vaccine are available from four clinical trials. Two studies in healthy adult volunteers and one Phase IIa study in healthy newborn infants were performed with rBCG. Additionally, a Phase II clinical trial was conducted with rBCG in HIV-unexposed and HIV-exposed, BCG-naïve newborn infants for clinical bridging. Clinical trials have also been conducted to assess the effect of rBCG vaccination on TB recurrence and on the susceptibility or severity of respiratory diseases during the severe acute respiratory syndrome-coronavirus 2 (SARS-CoV-2) pandemic.

BCG is one of the most widely used vaccines worldwide. However, because BCG is a biologic drug that uses benign bacteria, it is more complicated to manufacture than many other types of drugs. Serum Institute of India is the largest manufacturer of BCG vaccines in the world, while Merck & Co., based in New Jersey, currently is the only manufacturer of BCG (TICE BCG) in the U.S.

ImmunityBio has been awarded multiple patents covering the composition and methods of use for the combination of BCG plus ANKTIVA in bladder cancer (US 11,173,191 Β2; US 11,679,144 Β2; US 11,890,323 B2).

About ANKTIVA

The cytokine interleukin-15 (IL-15) plays a crucial role in the immune system by affecting the development, maintenance, and function of key immune cells—NK and CD8+ killer T cells—that are involved in killing cancer cells. By activating NK cells, ANKTIVA overcomes the tumor escape phase of clones resistant to T cells and restores memory T cell activity with resultant prolonged duration of complete response.

ANKTIVA is a first-in-class IL-15 agonist IgG1 fusion complex, consisting of an IL-15 mutant (IL-15N72D) fused with an IL-15 receptor alpha, which binds with high affinity to IL-15 receptors on NK, CD4+, and CD8+ T cells. This fusion complex of ANKTIVA mimics the natural biological properties of the membrane-bound IL-15 receptor alpha, delivering IL-15 by dendritic cells and drives the activation and proliferation of NK cells with the generation of memory killer T cells that have retained immune memory against these tumor clones. The proliferation of the trifecta of these immune killing cells and the activation of trained immune memory results in immunogenic cell death, inducing a state of equilibrium with durable complete responses. ANKTIVA has improved pharmacokinetic properties, longer persistence in lymphoid tissues, and enhanced anti-tumor activity compared to native, non-complexed IL-15 in-vivo.

ANKTIVA was approved by the FDA in 2024 for BCG-unresponsive non-muscle invasive bladder cancer CIS with or without papillary tumors. For more information, visit ImmunityBio.com (Founder’s Vision) and Anktiva.com.

On March 13, 2025 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery, development, and commercialization of drugs for the treatment of cancer, reported financial results for the year ended December 31, 2024, and provided a corporate update (Press release, Cellectar Biosciences, MAR 13, 2025, View Source [SID1234651125]).

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"In 2024 the company showcased the efficacy and safety of iopofosine I 131 for the treatment of relapsed/refractory Waldenström macroglobulinemia. We recently completed a productive meeting with the FDA that established a clear regulatory pathway for the accelerated approval of this promising drug. Based upon this regulatory clarity, the quality of the CLOVER-WaM data, and a robust global market opportunity, we continue to evaluate inbound inquiries regarding a range of collaborations for iopofosine I 131, which we view as an attractive, non-dilutive funding approach." said James Caruso, president and CEO of Cellectar. "In addition, the company received clearance for an IND for our Auger-emitting radioconjugate and will be submitting an IND application for our alpha-emitting radioconjugate. By the middle of 2025 we will be prepared to advance into phase 1 clinical studies for both compounds, in triple negative breast cancer and pancreatic cancer indications, respectively."

2024 and Recent Corporate Highlights

· Finalized confirmatory study design and regulatory pathway for potential FDA accelerated approval of iopofosine I 131, the Company’s targeted radiotherapeutic candidate for the treatment of relapsed/refractory WM.

o The study will be a randomized, controlled trial of iopofosine I 131 versus a comparator arm, with 100 patients per arm.

o Two-stage approval process includes conditional accelerated approval based on a major response rate (MRR) endpoint with full approval based upon achieving a progression-free survival endpoint.

o Company expects to complete full patient enrollment within 24 months of the first patient admitted to the study.

o Total study cost is expected to be between $40M-$45M, with approximately $30M to full enrollment.

· Presented data from the Phase 2 CLOVER-WaM study in an oral session at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH 2024) in December. The oral presentation highlighted that treatment with iopofosine I 131 in patients suffering from relapsed/refractory WM demonstrated:

o overall Response Rate (ORR) was 83.6%;

o major Response Rate (MRR) was 58.2%, which exceeded the FDA agreed-upon primary endpoint of 20% MRR;

o durable efficacy in previously treated WM patients, with no current standard of care therapy;

o well tolerated with a manageable toxicity profile across broad biologic and clinical subgroups.

· An article published in the journal eBioMedicine, volume 111, 2025, 105496, ISSN 2352-3964 from a SPORE Grant-supported, investigator-led study utilizing iopofosine I 131 (also known as CLR 131) in combination with external beam radiation, reported the best overall response from 11 evaluable patients included seven participants with a complete response (63.6%), one with a partial response (9%), one with stable disease (9%), and two with disease progression (18%), further supporting iopofosine I 131’s therapeutic benefit in solid tumors.

· Continued development of CLR 121225 and CLR 121125, the Company’s pre-clinical radioconjugate assets, to support Phase 1 solid tumor studies:

o The company is prepared to initiate a Phase 1b/2a dose-finding study with CLR 121125 in triple-negative breast cancer. CLR 121125 is the company’s lead Auger-emitting (iodine-125) Phospholipid Radioconjugate (PRC) that provides the greatest precision in targeted radiotherapy as emissions only travel a few nanometers.

o The company plans to file an IND application in the first half of 2025 for CLR 121225. CLR 121225 is Cellectar’ s lead alpha-emitting (actinium-225) PRC, which has demonstrated activity in multiple solid tumor animal models, including pancreatic and colorectal cancer.

2024 Financial Highlights

· Cash and Cash Equivalents: As of December 31, 2024, the company had cash and cash equivalents of $23.3 million, compared to $9.6 million as of December 31, 2023. In 2024, Cellectar executed multiple financial transactions, including investors’ exercise of warrants in January 2024 that generated $44.1 million, and an inducement financing in July 2024, which included the exercise of existing warrants and the purchase of new warrants for an additional $19.4 million. The company believes its cash balance as of December 31, 2024, is adequate to fund its basic budgeted operations into the fourth quarter of 2025.

· Research and Development Expenses: R&D expenses for the year ended December 31, 2024, were approximately $26.1 million, compared to approximately $27.3 million for the year ended December 31, 2023. The decrease was primarily a result of the timing of expenditures for our WM Phase 2 study to support final patient visits, partially offset by the extensive analytic work necessary to prepare for a planned regulatory submission, product sourcing, manufacturing, and logistics infrastructure costs to support multi sourcing for each aspect of iopofosine I 131 production.

· General and Administrative Expenses: G&A expenses for the year ended December 31, 2024, were approximately $25.6 million, compared to approximately $11.7 million for the same period in 2023. The increase was primarily driven by costs associated with the development of infrastructure necessary to support potential commercialization, including the related marketing and personnel costs.

· Other income and expense: Other income and expense, net, was approximately $7.3 million of income in 2024, as compared to approximately $3.9 million of expense in the prior year. These amounts are almost exclusively non-cash and driven by the issuance and valuation of equity securities in conjunction with financing activities. The only cash impact was interest income, which for 2024 improved to approximately $1.2 million from $0.4 million in the prior year.

· Net Loss: Net loss for the full year ending December 31, 2024, was $44.6 million or $1.22 per basic share and $1.40 per diluted share, compared with $42.8 million or $3.50 per basic and diluted share during 2023.

Conference Call & Webcast Details

Cellectar management will host a conference call and webcast today, March 13, 2024, at 8:30 AM Eastern Time to discuss these results and answer questions. Stockholders and other interested parties may participate in the conference call by dialing 1-800-717-1738. A live webcast of the conference call can be accessed in the "Events & Presentations" section of Cellectar’s website at www.cellectar.com. A recording of the webcast will be available and archived on the Company’s website for approximately 90 days.

On March 13, 2025 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 financial results for the fourth quarter and year ended December 31, 2024, and provided a corporate update (Press release, Candel Therapeutics, MAR 13, 2025, View Source [SID1234651126]).

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"Last year was transformational for Candel, driven by our team’s incredible focus and execution of the Company’s key priorities," said Paul Peter Tak, MD PhD FMedSci, President and CEO of Candel. "We delivered positive data across our platforms, including pivotal topline phase 3 data for CAN-2409 in intermediate-to-high-risk localized prostate cancer in December, which we believe shows the potential of CAN-2409, if approved, to redefine the current standard of care for patients with prostate cancer."

Dr. Tak continued, "We enter 2025 well-resourced with a clear direction and mandate. Our primary focus for the year is working toward readiness to submit CAN-2409’s BLA for prostate cancer, a key opportunity to address a very significant unmet need and opportunity for value creation. In addition, we continue to explore the efficacy of CAN-2409 in other indications, as evidenced by our recently disclosed positive final overall survival data for CAN-2409 in a randomized controlled phase 2a trial in borderline resectable pancreatic cancer. This result has triggered enabling work, which will include scientific advisory boards and engagement with the FDA, to get ready for a larger, late-stage, randomized clinical trial in this indication in the future. In addition, we anticipate final overall survival and biomarker data from our open label phase 2a clinical trial of CAN-2409 in therapy-resistant non-small cell lung cancer patients in the very near future. This study may also trigger similar enabling work. In Q4 2025, we will also provide a clinical and biomarker update from our ongoing phase 1b clinical trial evaluating multiple doses of CAN-3110 in patients with recurrent high-grade glioma. Finally, we have also made significant progress with our preclinical programs, leveraging the enLIGHTENTM Discovery Platform."

Fourth Quarter 2024 & Recent Highlights

•
CAN-2409 – Pancreatic Cancer
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Positive final survival data from the randomized controlled phase 2a clinical trial of CAN-2409 in borderline resectable pancreatic ductal adenocarcinoma (PDAC), demonstrating notable improvement in overall survival. Patients who had received experimental treatment with CAN-2409 and standard of care achieved a median overall survival of 31.4 months versus only 12.5 months observed in the control arm treated with standard of care.
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Notably, long-term survivors in the CAN-2409 arm remained alive at 66.0, 63.6, and 35.8-months post-enrollment, whereas only one patient from the control arm was still alive at the time of data cut-off (February 20, 2025). Patients in the experimental arm were stable at the time of last follow up with minimal maintenance therapy and, despite previous recurrence, experienced extended and ongoing post-progression survival, further highlighting the sustained benefit of CAN-2409, even in metastatic disease.
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The U.S. Food and Drug Administration (FDA) previously granted orphan drug designation and fast track designation for CAN-2409 in borderline resectable PDAC.
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CAN-2409 – Prostate Cancer
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In December 2024, the Company reported positive topline data from its multicenter, randomized, placebo-controlled phase 3 clinical trial evaluating CAN-2409 in intermediate-to-high-risk localized prostate cancer patients. The study met its primary endpoint by demonstrating statistically significant improvement in disease-free survival (DFS) in patients who received CAN-2409 plus valacyclovir (prodrug) combined with standard of care external beam radiation therapy (n=496) compared to standard of care alone (n=249) within the intent to treat population.

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The data showed a 30% reduction in the risk for prostate cancer recurrence or death due to any cause for the CAN-2409 treatment arm compared to placebo control arm (p=0.0155), and 80.4% pathological complete responses in 2-year post-treatment biopsies after CAN-2409 administration compared to 63.6% in the control arm (p=0.0015). The safety profile of CAN-2409 was generally consistent with previous studies, with no new safety signals identified.
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This study was conducted under a Special Protocol Assessment (SPA) agreed with the FDA, meaning that safety and efficacy data generated from this study could be sufficient for the Company to seek regulatory approval for CAN-2409 in this indication.
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The FDA previously granted fast track designation for CAN-2409 for the treatment of localized primary prostate cancer.
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CAN-3110 – Recurrent High-Grade Glioma
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Presented updated clinical and biomarker activity data from cohort C of a phase 1b clinical trial during the 6th Annual International Oncolytic Virotherapy Conference (IOVC) in October 2024. The Principal Investigator reported ongoing improved survival compared to historical controls, with 3 out of 6 patients still alive after more than one year (12.2, 13.0 and 18.7 months, respectively) after initiation of experimental treatment with repeated administrations of CAN-3110.
•
The FDA granted fast track designation and orphan drug designation to CAN-3110 for the treatment of rHGG in February and May 2024, respectively.
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CAN-3110 – Melanoma
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Presented preclinical results on the therapeutic potential of CAN-3110 in the Ras-Raf pathway altered melanoma model at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2024 Annual Meeting. CAN-3110 exhibited potent, tumor-specific cytotoxicity in human and murine melanoma cell lines with varied CDKN2A pathway alterations and Nestin expression. In vivo mouse studies showed dose-dependent inhibition of tumor growth, with regression observed in a subset (3 of 8) of tumor bearing animals treated with a high dose of CAN-3110. CAN-3110 treatment was well-tolerated in melanoma preclinical mouse models based on body weight and histopathological analysis following intra-tumoral administration.
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enLIGHTEN Discovery Platform
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Presented data on a new multimodal viral therapeutic candidate encoding IL-12 and IL-15 during IOVC 2024. Data showed the ability of the asset to induce expansion and activation of natural killer and CD8+ T cell populations, resulting in significant tumor growth inhibition and tumor regression in two different models.
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Recent Corporate Events
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In December 2024, the Company completed an underwritten public offering of 12,000,001 shares of its common stock (inclusive of the exercise of the underwriters’ option to purchase additional shares in full) at a price to the public of $6.00 per share, and pre-funded warrants to purchase up to 3,333,333 shares of its common stock at a price to the public of $5.99 per warrant with an exercise price of $0.01 per pre-funded warrant, resulting in net proceeds of approximately $85.9 million. The offering closed on December 16, 2024.

Anticipated Milestones

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Final overall survival data from phase 2a clinical trial evaluating CAN-2409 in patients with NSCLC and an inadequate response to immune checkpoint inhibitors, expected in Q1 2025.

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Biomarker and initial overall survival data from ongoing phase 1b clinical trial evaluating multiple doses of CAN-3110 in patients with rHGG, expected in Q4 2025.
Financial Results for the Year and Fourth Quarter Ended December 31, 2024

Research and Development Expenses: Research and development expenses were $4.8 million for the fourth quarter of 2024 compared to $7.3 million for the fourth quarter of 2023, and $19.3 million for the full year 2024 compared to $24.5 million for the full year 2023. The decrease was primarily due to lower regulatory, manufacturing and clinical trial costs in support of the Company’s CAN-2409 programs and lower payroll-related expenses following the corporate restructuring in the fourth quarter of 2023. Research and development expenses included a non-cash stock compensation expense of $0.8 million and $3.3 million for the fourth quarter and full year of 2024, respectively, as compared to a non-cash stock compensation expense of $0.5 million and $1.3 million for the fourth quarter and full year of 2023.

General and Administrative Expenses: General and administrative expenses were $3.3 million for the fourth quarter of 2024 compared to $3.1 million for the fourth quarter of 2023, and $14.1 million for the full year 2024 compared to $13.9 million for the full year 2023. The increase was primarily due to higher professional and consulting fees, which were partially offset by decreased payroll-related expenses following the corporate restructuring in the fourth quarter of 2023. General and administrative expenses included non-cash stock compensation expense of $0.4 million and $2.0 million for the fourth quarter and full year of 2024, respectively, as compared to a non-cash stock compensation expense of $0.5 million and $1.7 million for the fourth quarter and full year of 2023.Net Loss: Net loss for the fourth quarter of 2024 was $14.1 million compared to a net loss of $11.1 million for the fourth quarter of 2023, and included net other expense of $5.9 million and $0.8 million, respectively, related primarily to the change in the fair value of the Company’s warrant liability. Net loss for the full year 2024 was $55.2 million compared to a net loss of $37.9 million for the full year 2023 and included net other expense of $21.8 million and net other income of $0.5 million, respectively. The change from net other income in 2023 to net other expense in 2024 primarily related to the change in the fair value of the Company’s warrant liability.

Cash Position: Cash and cash equivalents as of December 31, 2024 were $102.7 million, as compared to $35.4 million as of December 31, 2023. Based on current plans and assumptions, the Company expects that its existing cash and cash equivalents will be sufficient to fund its current operating plan into the first quarter of 2027.

About CAN-2409

CAN-2409, Candel’s most advanced multimodal biological immunotherapy candidate, is an investigational, off-the-shelf, replication-defective adenovirus designed to deliver the herpes simplex virus thymidine kinase (HSV-tk) gene to a patient’s specific tumor and induce an individualized, systemic immune response against the tumor. HSV-tk is an enzyme that locally converts orally administered valacyclovir into a toxic metabolite that kills nearby cancer cells. Together, this regimen is designed to induce an individualized and specific CD8+ T cell-mediated response against the injected tumor and un-injected distant metastases for broad anti-tumor activity, based on in situ vaccination against a variety of tumor antigens. Because of its versatility, CAN-2409 has the potential to treat a broad range of solid tumors. Encouraging monotherapy activity as well as combination activity with standard of care radiotherapy, surgery, chemotherapy, and immune checkpoint inhibitors have previously been shown in several preclinical and clinical settings. More than 1,000 patients have been dosed with CAN-2409 with a favorable tolerability profile to date, supporting the potential for combination with other therapeutic strategies.

Currently, Candel is evaluating CAN-2409 in NSCLC and borderline resectable PDAC, in ongoing clinical trials, and has recently completed a successful phase 3 clinical trial in localized prostate cancer. CAN-2409 plus prodrug (valacyclovir) has been granted fast track designation by the FDA for the treatment of PDAC, stage III/IV NSCLC in patients who are resistant to first line PD-(L)1 inhibitor therapy and who do not have activating molecular driver mutations or have progressed on directed molecular therapy, and localized primary prostate cancer. Candel’s pivotal phase 3 clinical trial in prostate cancer was conducted under a SPA agreed with the FDA. The FDA has also granted orphan drug designation to CAN-2409 for the treatment of PDAC.

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. CAN-3110 is being evaluated in a phase 1b clinical trial in patients with 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 improved median overall survival compared to historical controls after a single CAN-3110 injection 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 and orphan drug designation for the treatment of rHGG.

About the enLIGHTEN Discovery Platform

The enLIGHTEN Discovery Platform is a systematic, iterative herpes simplex virus (HSV)-based discovery platform leveraging human biology and advanced analytics to create new multimodal biological immunotherapies for solid tumors. The enLIGHTEN Discovery Platform has been designed to deconvolute the characteristics of the tumor microenvironment related to clinical outcomes. These characteristics are rapidly translated into optimized multi-gene payloads of tumor modulators that can be delivered to the tumor microenvironment for specific indications, disease stages, and rationally designed therapeutic combinations. In 2022, the Company announced a discovery partnership with the University of Pennsylvania Center for Cellular Immunotherapies to create new viral immunotherapies that could enhance the efficacy of chimeric antigen receptor T cell (CAR-T) therapy in solid tumors. During the SITC (Free SITC Whitepaper) 2023 Annual Meeting and the 2023 IOVC meeting, Candel presented encouraging data on the first candidate from this platform, Alpha 201-macro-1, which was designed to interfere with the CD47/SIRP1α pathway, in mouse models of breast cancer and lung cancer. During the AACR (Free AACR Whitepaper) Annual Meeting 2024, Candel presented preclinical data, unveiling the second candidate from the enLIGHTEN Discovery Platform, a first-in-class multimodal immunotherapy candidate to induce tertiary lymphoid structures, being developed as a novel therapeutic for solid tumors. Candel presented data at the 2024 IOVC meeting. The presentation focused on a multimodal viral therapeutic candidate encoding IL-12 and IL-15, the latest asset from the platform.