On August 14, 2025 Prelude Therapeutics Incorporated (Nasdaq: PRLD), a clinical-stage precision oncology company, reported its financial results for second quarter ended June 30, 2025, and provided an update on its clinical development pipeline and other corporate developments (Press release, Prelude Therapeutics, AUG 14, 2025, View Source [SID1234655313]).

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"From the discovery of first-in-class highly selective SMARCA2 degraders through Phase 1 studies in biomarker selected population of patients, Prelude demonstrated exemplary execution of our SMARCA2 program to deliver a potentially novel treatment option for patients with aggressive cancers harboring SMARCA4 deletion," stated Kris Vaddi, Ph.D., Chief Executive Officer of Prelude. "This past quarter, we completed our Phase 1 dose escalation of PRT3789 (IV), both as monotherapy and in combination with docetaxel. With the knowledge and experience gained through PRT3789 clinical development, we were able to expeditiously advance our oral program, PRT7732, which began in the fourth quarter of 2024. I am pleased with the progress made to date with PRT7732, which is currently enrolling our seventh dose cohort of 125 mg."

Vaddi continued, "We’ve decided to pause further development of PRT3789, and focus solely on PRT7732 as our go-forward strategy for our SMARCA2 Program. While PRT3789 demonstrated initial proof of concept for the mechanism, a number of considerations – including the potential need for higher target coverage throughout the dosing interval, and capital needs to continue to advance both agents – contributed to this decision. The clinical profile observed to date with PRT7732 including oral once daily dosing, safety and tolerability, oral exposures, and >90% target degradation positions us well to explore the potential for this mechanism in SMARCA4 deleted cancers and determine the path forward for continued development by year end."

Continued Vaddi, "We’ve made significant progress across our core research and development organization, while employing disciplined capital management through resource allocation and headcount management throughout the Company. Notably, we’ve continued to advance our KAT6A degrader program, on track for IND filing in the first half of 2026, presented preclinical data on mCALR-targeted ADCs and continued progress with our partner AbCellera related to precision ADCs."

Clinical Program Updates and Upcoming Milestones

SMARCA2 Degrader Development Program

PRT3789 – A first-in-class, highly selective, intravenous SMARCA2 degrader

PRT3789 is designed to treat patients with a SMARCA4 mutation. Patients with SMARCA4-mutated cancer, a particularly aggressive form of the disease, have a very poor clinical prognosis. Approximately 10% of all non-small cell lung cancers and 5% of all cancers broadly, harbor a SMARCA4 mutation. In NSCLC, these patients tend to have poor response to standard of care chemoimmunotherapy and are largely ineligible for other targeted therapies. We believe that this represents an area of high unmet medical need.

PRT3789 has completed Phase 1 clinical development in patients with biomarker selected SMARCA4-mutated cancers. The Company anticipates providing updated data from the Phase 1 study by year-end 2025. Based on the totality of the data and available resources, the Company would only advance the program in the context of a partnership and will be focusing internal resources solely on PRT7732.

PRT7732 – A potent, highly selective and orally bioavailable SMARCA2 degrader

PRT7732 is a highly selective and orally bioavailable SMARCA2 degrader with a distinct chemical composition to PRT3789. In the fourth quarter of 2024, the Company initiated and enrolled the first patients in a phase 1 multi-dose escalation trial of PRT7732 (NCT06560645) in biomarker selected SMARCA4 mutated cancers. Enrollment continues to advance rapidly, and the Company is currently enrolling patients in the seventh dose escalation cohort (125 mg once daily). The Company expects to provide an initial first-in-human data update including PK/PD, safety and an initial look at clinical activity at biologically relevant doses by year end 2025.

Highly selective KAT6A oral degrader program

KAT6 is an emerging and recently validated target in the treatment of ER+ breast cancer and other malignancies. Prelude discovered and is developing the industry’s first – based on currently published patents and literature – highly potent, selective and orally bioavailable KAT6A selective degraders. The Company is now advancing a development candidate and remains on track to file an IND in the first half of 2026. Prelude believes that selectively degrading KAT6A has the potential for improved efficacy, tolerability and combinability with other agents relative to non-selective inhibitors of KAT6A/B. The Company recently presented preclinical data validating this hypothesis at the AACR (Free AACR Whitepaper) Annual Meeting 2025. The presentation can be found at Publications – Prelude Therapeutics.

Precision ADCs with SMARCA2/4 dual degrader payload

Prelude is developing potent SMARCA2/4 dual degraders that robustly inhibit cancer cell growth and induce cell death across multiple cancer types as payloads for precision ADCs. The Company presented the first preclinical data from its precision ADC platform at the 36th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium in October. These data demonstrated that SMARCA2/4 degrader antibody conjugates have potential for significantly better in vivo efficacy and tolerability when compared to traditional cytotoxic ADCs when tested head-to-head in xenograft models. The presentation can be found at Publications – Prelude Therapeutics.

Mutated Calreticulin (mCALR)

Mutant CALR is a neoantigen presented on the cell surface of malignant myeloid cells but not normal cells and is found in approximately 25-35% of patients with myelofibrosis (MF) and essential thrombocythemia (ET). Recently, a mCALR-targeted monoclonal antibody demonstrated robust clinical activity in high-risk ET patients. Prelude is seeking to further optimize this modality by developing mCALR-targeted precision ADCs using the Company’s proprietary degrader payloads. The Company presented the first preclinical data from this discovery effort at the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress in June. The presentation can be found at Publications – Prelude Therapeutics.

Second Quarter 2025 Financial Results 

Cash, Cash Equivalents, Restricted Cash and Marketable Securities:

Cash, cash equivalents, restricted cash and marketable securities as of June 30, 2025 were $77.3 million. The Company anticipates that its existing cash, cash equivalents, restricted cash and marketable securities will fund Prelude’s operations into the second quarter of 2026.

Research and Development (R&D) Expenses:

For the second quarter of 2025, R&D expense decreased to $25.8 million from $29.5 million for the prior year period. Included in the R&D expense for the three months ended June 30, 2025 was $2.2 million of non-cash expense related to stock-based compensation, including employee stock options, compared to $3.4 million for the three months ended June 30, 2024. Research and development expenses decreased primarily due to a decrease in expense related to our SMARCA2 clinical trials. Research and development expenses may fluctuate from period to period depending upon the stage of certain projects and the level of preclinical and clinical trial-related activities.

General and Administrative (G&A) Expenses:

For the second quarter of 2025, G&A expenses decreased to $6.4 million from $7.7 million for the prior year period. Included in general and administrative expenses for the three months ended June 30, 2025, was $1.6 million of non-cash expense related to stock-based compensation, including employee stock options, compared to $2.7 million for the three months ended June 30, 2024. The decrease in general and administrative expenses was primarily due to a decrease in stock-based compensation due to lower valuation on more recent grants due to the decrease in our stock price.

On August 14, 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 second quarter ended June 30, 2025, and provided a corporate update (Press release, Candel Therapeutics, AUG 14, 2025, View Source [SID1234655283]).

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"This quarter marked several pivotal achievements for Candel, highlighted by the FDA RMAT Designation for CAN-2409, and being selected for an oral presentation at ASCO (Free ASCO Whitepaper), reflecting the strength of the data based on our pivotal phase 3 clinical trial in localized prostate cancer," said Paul Peter Tak, M.D. Ph.D. FMedSci, President and CEO of Candel. "Further, the addition of Dr. Maha Radhakrishnan to our Board of Directors, as well as the appointment of Charles Schoch as Chief Financial Officer, further strengthens our organization as we accelerate our pre-commercialization activities and advance toward BLA submission, anticipated in Q4 2026."

Dr. Tak continued, "The positive results from our clinical trials in prostate cancer, pancreatic cancer, and non-small cell lung cancer reinforce the therapeutic potential of CAN-2409 as a novel therapy. With the proceeds from our recent registered direct offering being utilized to support pre-commercialization and launch readiness activities, and with multiple regulatory designations, we believe we are well-positioned to execute on our near-term milestones and advance toward our goal of bringing this important treatment option to patients with prostate cancer."

Second Quarter 2025 & Recent Highlights

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CAN-2409 – Prostate Cancer
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In an oral presentation at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting on June 3, 2025, in Chicago, IL, the Company reported phase 3 results from the CAN-2409 clinical trial in intermediate-to-high risk localized prostate cancer. The primary endpoint, agreed with the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA), was met, with a statistically significant improvement of 30% in disease-free survival among CAN-2409 recipients (HR 0.70, p=0.0155) when compared to placebo, both in combination with standard-of-care external beam radiation therapy. This data was supported by secondary endpoints.
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On June 3, 2025, Candel hosted a conference call featuring perspectives from leading prostate cancer specialists, John E. Sylvester, M.D., Atlantic Urology Clinics, Myrtle Beach, South Carolina, and Ronald F. Tutrone, Jr., M.D., FACS, CPI, National Medical Director of Clinical Research, United Urology Group, Towson, Maryland. Both physicians were principal investigators on the trial. The call replay can be accessed here.
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This phase 3 study was conducted under a SPA agreed with the FDA, meaning that certain 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 Company is advancing its pre-BLA readiness, including through Chemistry, Manufacturing, and Controls (CMC) activities and documentation, and preparation of clinical study reports.
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In May, the Company received RMAT Designation from the FDA for CAN-2409 for the treatment of newly diagnosed, localized prostate cancer in patients with intermediate-to-high-risk disease. The FDA previously granted Fast Track Designation for CAN-2409 for the treatment of localized primary prostate cancer.
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The Company continues to work toward a BLA submission for CAN-2409 in prostate cancer in Q4 2026.
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CAN-2409 – Non-Small Cell Lung Cancer (NSCLC)

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In March, the Company reported positive overall survival data from its phase 2a clinical trial of CAN-2409 in patients with stage III/IV NSCLC inadequately responding to ICI treatment.
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In patients with an inadequate response to ICI treatment (Cohorts 1+2, n=46), median overall survival (mOS) was 24.5 months.
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In patients with progressive disease, despite ICI treatment (Cohort 2, n=41), mOS was 21.5 months, which is markedly longer than the 9.8–11.8 months of survival reported in published literature1,2 in the same patient population receiving standard of care of docetaxel chemotherapy.
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37% of patients with progressive disease at enrollment were still alive > 24 months after CAN-2409 treatment at the time of the March 3, 2025 data cut, suggesting a long tail of survival. 14/15 patients with overall survival > 24 months and 9/9 patients with overall survival > 30 months had non-squamous NSCLC.
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In patients with non-squamous NSCLC and progressive disease despite ICI (cohort 2, n=33), observed mOS was 25.4 months after CAN-2409 treatment.
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A decrease in the size of uninjected tumors was observed in 69% of patients with multiple lesions (n=35), indicating that a local injection is associated with a systemic anti-tumor immune response.
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CAN-2409 maintained its generally favorable safety and tolerability profile throughout the extended follow-up period.
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The FDA previously granted Fast Track Designation for CAN-2409 for the treatment of NSCLC.
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CAN-2409 – Pancreatic Cancer
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In February 2025, the Company reported positive overall survival data from the randomized, controlled, phase 2a clinical trial of CAN-2409 in borderline resectable pancreatic ductal adenocarcinoma (PDAC).
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Patients who had received experimental treatment with CAN-2409 and chemoradiotherapy achieved a mOS of 31.4 months versus 12.5 months observed in the control arm treated with chemoradiotherapy.
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Notably, three out of seven patients in the CAN-2409 arm were long-term survivors and remained alive at 66.0, 63.6, and 35.8 months post-treatment, whereas only one out of six patients from the control arm was still alive at the time of data cut-off (February 20, 2025). Patients in the CAN-2409 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 FDA previously granted Orphan Drug Designation and Fast Track Designation for CAN-2409 in borderline resectable PDAC.

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The EMA granted Orphan Designation for CAN-2409 for the treatment of pancreatic cancer in July 2025.
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Recent Corporate Events
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In June 2025, the Company completed a registered direct offering, of approximately 3.2 million shares of its common stock, to a select group of existing healthcare-focused institutional investors, executive officers, and directors of the Company, at a price per share of $4.67, resulting in gross proceeds of approximately $15 million, before deducting offering expenses payable by the Company.
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In June 2025, the Company appointed Charles Schoch as Chief Financial Officer (CFO). Mr. Schoch previously served as interim CFO of Candel. He will continue to be instrumental as the Company advances its clinical pipeline and prepares for BLA submission of CAN-2409 in localized prostate cancer, anticipated in Q4 2026.
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In June 2025, the Company appointed Maha Radhakrishnan, M.D., to its Board of Directors. Dr. Radhakrishnan has significant expertise in product development and commercialization.
Anticipated Milestones

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Additional clinical and biomarker activity data from an ongoing phase 1b clinical trial evaluating repeat doses of CAN-3110 in patients with recurrent high-grade glioma (rHGG), is expected in Q4 2025.
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Candel plans to host a virtual Research and Development event in Q4 2025.
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Submission of BLA for CAN-2409 in prostate cancer expected in Q4 2026.
Financial Results for the Second Quarter Ended June 30, 2025

Research and Development Expenses: Research and development expenses were $7.0 million for the second quarter of 2025 compared to $5.0 million for the second quarter of 2024. The increase was primarily due to an increase in manufacturing costs in support of the Company’s CAN-2409 programs, partially offset by a decrease in employee-related expenses, which was driven primarily from a reduction in stock-based compensation expense. Research and development expenses included a non-cash stock compensation expense of $0.4 million for the second quarter of 2025 compared to a non-cash stock compensation expense of $1.3 million for the second quarter of 2024.

General and Administrative Expenses: General and administrative expenses were $4.2 million for the second quarter of 2025, compared to $3.6 million for the second quarter of 2024. The increase was primarily due to an increase in commercial readiness costs as well as higher professional and consulting fees. General and administrative expenses included non-cash stock compensation expense of $0.6 million for both the second quarter of 2025 and the second quarter of 2024.

Net Loss: Net loss for the second quarter of 2025 was $4.8 million compared to a net loss of $22.2 million for the second quarter of 2024 and included net other income of $6.4 million and net other expense of $13.7 million, respectively. The decrease in net loss was primarily related to the change in the fair value of the Company’s warrant liability.

Cash Position: Cash and cash equivalents, as of June 30, 2025, were $100.7 million compared to $102.7 million as of December 31, 2024. Based on current plans and assumptions, the Company expects that its existing cash and cash equivalents will be sufficient to fund operations into Q1 2027, including the Company’s expected submission of the BLA for CAN-2409 in intermediate-to-high-risk prostate cancer to the FDA in Q4 2026.

About CAN-2409

CAN-2409 (aglatimagene besadenovec), 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 tumor. After intratumoral administration, HSV-tk enzyme activity results in conversion of prodrug (valacyclovir) into deoxyribonucleic acid (DNA)-incorporating nucleotide analogs, leading to immunogenic cell death in cells exhibiting DNA damage and proliferating cells, with subsequent release of a variety of tumor (neo)antigens in the tumor microenvironment. At the same time, the adenoviral serotype 5 capsid protein promotes inflammation through the induction of expression of pro-inflammatory cytokines, chemokines, and adhesion molecules. Together, this regimen is designed to induce an individualized and specific CD8+ T cell-mediated response against the injected tumor and uninjected distant metastases for broad anti-tumor activity, based on in situ immunization against a variety of tumor antigens. 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 standard of care, when indicated.

About CAN-3110

CAN-3110 is a first-in-class, replication-competent herpes simplex virus-1 (HSV-1) next-generation oncolytic viral immunotherapy candidate designed for 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.3 The Company and academic collaborators are currently evaluating the effects of repeat 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.

On August 14, 2025 Pyxis Oncology, Inc. (Nasdaq: PYXS), a clinical-stage company developing antibody-drug conjugate (ADC) therapeutics for difficult-to-treat cancers, reported financial results for the quarter ended June 30, 2025, and provided a business update (Press release, Pyxis Oncology, AUG 14, 2025, View Source [SID1234655314]).

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"We are invigorated by the progress we’re making, particularly in our ongoing clinical trials, where we’re seeing that MICVO’s unique mechanism as an extracellular targeted ADC has the potential to transform the treatment of advanced solid tumors," said Lara S. Sullivan, M.D., President, Chief Executive Officer and Chief Medical Officer of Pyxis Oncology. "The encouraging clinical and preclinical data observed to date suggest that MICVO may offer a differentiated treatment approach as both a monotherapy and in combination with pembrolizumab for HNSCC and other advanced solid tumors. We look forward to evaluating the emerging clinical data as we continue to advance this novel ADC."

Pipeline Updates

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Pyxis Oncology anticipates having preliminary data from the Part 2 monotherapy expansion cohorts of the ongoing Phase 1 clinical trial evaluating MICVO in 2L and 3L R/M HNSCC patients who have received prior platinum and PD-1 inhibitor therapy in the second half of 2025. Preliminary data from the trial in 2L and 3L R/M HNSCC patients who have received prior EGFRi and PD-1 inhibitor therapy are anticipated in the first half of 2026. R/M HNSCC continues to be an area of high medical need despite potential improvements in treatment options.

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Pyxis Oncology anticipates having preliminary data from the Phase 1/2 combination study of MICVO in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab), in R/M HNSCC and other advanced solid tumors in the second half of 2025.

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In July 2025, Pyxis Oncology received a $2.8 million ($3 million milestone payment, less $0.2 million of tax in China) milestone payment from Simcere Pharmaceutical Group Limited ("Simcere") for the approval of suvemcitug (BD0801) in China by the National Medical Products Administration. The Company is eligible to receive mid to high single-digit percentage royalties on net sales of suvemcitug in China, under the terms of the Company’s license and collaboration agreement. In addition to suvemcitug, Pyxis Oncology retains rights to two other antibodies in development by Apexigen’s licensees, discovered through the APXiMAB platform.

Second Quarter 2025 Financial Results

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As of June 30, 2025, Pyxis Oncology had cash and cash equivalents, including restricted cash, and short-term investments, of $90.4 million. The Company believes that its current cash, cash equivalents, restricted cash and short-term investments will be sufficient to fund its operations into the second half of 2026.

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Revenues for the quarter ended June 30, 2025 were $2.8 million, compared to $0 for the quarter ended June 30, 2024. During the quarter, we recognized $2.8 million of milestone revenue ($3 million of milestone less $0.2 million of tax in China) related to regulatory approval of suvemcitug in China. The regulatory milestone was pursuant to an out-licensing and collaboration agreement between our subsidiary company, Apexigen, and Simcere, for the development and commercialization of suvemcitug for oncology in China.

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Research and development expenses were $17.1 million for the quarter ended June 30, 2025, compared to $14.0 million for the quarter ended June 30, 2024. The increase in expenses of $3.1 million was due to increased manufacturing of drug product and drug substance and clinical trial-related expenses for monotherapy and combination therapy of MICVO aggregating to $3.8 million, partially offset by reduction in expenses related to PYX-106 by $1.1 million as the clinical development of PYX-106-101 was paused in December 2024.

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General and administrative expenses were $5.4 million for the quarter ended June 30, 2025, compared to $6.1 million for the quarter ended June 30, 2024. The decrease was primarily due to lower corporate insurance costs, lower facilities costs and decrease in legal, professional, and consulting fees.

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Net loss was $18.4 million, or ($0.30) per common share, for the quarter ended June 30, 2025, compared to $17.3 million, or ($0.29) per common share, for the quarter ended June 30, 2024. Excluding non-cash stock-based compensation expense, the net loss for the quarter ended June 30, 2025 was $15.3 million, compared to net loss of $14.4 million for the quarter ended June 30, 2024.

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As of August 13, 2025, the outstanding number of shares of Common Stock of Pyxis Oncology was 62,018,135.

On August 14, 2025 CPTx, a biotechnology company developing in vivo genetic medicines built with programmable single-stranded DNA (ssDNA), reported the QUIET-CAR collaborative project with NanoCell Therapeutics has received a Eurostars Grant from the European Union through the Horizon Europe program and Eureka Network (Press release, CPTx Bio, AUG 14, 2025, View Source [SID1234655419]). The focus of the QUIET-CAR project is development of targeted lipid nanoparticles carrying novel immune-silent ssDNA for in vivo CAR T therapy.

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"CPTx is committed to bringing in vivo CAR T therapies to patients through our proprietary pipeline development programs and now also through the QUIET-CAR project with NanoCell," said Hendrik Dietz, CEO of CPTx. "The QUIET-CAR project represents another innovative approach to CAR T, and we look forward to advancing it together. Being selected from so many high-quality submissions underscores the promise of the project and the strength of our technology."

Eurostars, part of the European Partnership on Innovative SMEs and supported by Horizon Europe, is a funding initiative to accelerate transnational innovation. With participation from 37 countries, the program selects only the most promising technological breakthroughs, evaluated by independent experts. The selection of the CPTx – NanoCell project from more than 120 proposals reflects QUIET-CAR’s strong scientific and clinical potential across oncology and autoimmune diseases. NanoCell Therapeutics is a privately held biotechnology company pioneering transformative in vivo cell engineering through its non-viral, DNA-based gene therapy platform, primarily targeting oncology and autoimmune diseases.

On August 14, 2025 Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company pursuing development of targeted therapies for oncology, reported financial results for the second quarter ended June 30, 2025 and other recent business developments (Press release, Celcuity, AUG 14, 2025, View Source [SID1234655284]).

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"We have had an eventful past few months at Celcuity. Last month, we announced positive topline data from the PIK3CA wild-type cohort of the pivotal Phase 3 VIKTORIA-1 clinical trial, which showed unprecedented reduction in risk of disease progression or death and incremental improvement in progression free survival in patients with HR+/HER2- advanced breast cancer," said Brian Sullivan, CEO and co-founder of Celcuity. "We believe topline data for both gedatolisib regimens from VIKTORIA-1 are potentially practice-changing. We are on track to submit the New Drug Application for gedatolisib, based on data from the PIK3CA wild-type cohort, later this year and to report topline data from the PIK3CA mutant cohort in the fourth quarter of 2025. Additionally, with our recent financing and our growing body of clinical data, we believe we are in a unique position to advance multiple potential blockbuster indications in breast and prostate cancer."

Second Quarter 2025 Business Highlights and Other Recent Developments

● In July 2025, the Company announced positive topline data for both primary endpoints of the PIK3CA wild-type cohort of the Phase 3 VIKTORIA-1 clinical trial that evaluated gedatolisib plus fulvestrant with and without palbociclib versus fulvestrant in patients with HR+/HER2- ABC whose disease had progressed on or after prior treatment with a CDK4/6 inhibitor. The efficacy results established several new milestones in the history of drug development for HR+/HER2- ABC.

○ The gedatolisib triplet (gedatolisib, fulvestrant and palbociclib) reduced the risk of disease progression or death by 76% compared to fulvestrant based on a hazard ratio ("HR") of 0.24. The median PFS was 9.3 months with the gedatolisib triplet versus 2.0 months with fulvestrant, an incremental improvement of 7.3 months.

○ The gedatolisib doublet (gedatolisib and fulvestrant) reduced the risk of disease progression or death by 67% compared to fulvestrant based on a hazard ratio of 0.33. The median PFS was 7.4 months with the gedatolisib doublet versus 2.0 months with fulvestrant, an incremental improvement of 5.4 months.

○ Both gedatolisib regimens showed lower rates of hyperglycemia and stomatitis and the rate of discontinuation of all treatment due to a treatment-related adverse event ("AE"), was lower than was reported in a Phase 1b study in this patient population.

○ Full data from the PIK3CA wild-type cohort of the VIKTORIA-1 clinical trial will be presented at an upcoming medical conference later this year.

○ Celcuity expects to submit an NDA to the FDA in the fourth quarter of 2025 for gedatolisib based on data from the PIK3CA wild-type cohort.

○ Enrollment is ongoing in the PIK3CA mutant cohort of the VIKTORIA-1 trial and remains on track to report topline data by the end of 2025.

● In July 2025, the first patient was dosed in VIKTORIA-2, a Phase 3 clinical trial evaluating gedatolisib plus a CDK4/6 inhibitor and fulvestrant as a first-line treatment for patients with HR+/HER2- ABC who are endocrine therapy resistant.

● In July 2025, the US Patent and Trademark Office issued a new patent for gedatolisib covering the dosing regimen used in the VIKTORIA-1 clinical trial, extending the patent exclusivity in the U.S. into 2042.

● In July 2025, the Company conducted a concurrent public offering of 2.750% convertible senior notes due 2031, common stock and pre-funded warrants. The net proceeds from the offerings were $286.5 million, after deducting underwriting discounts and commissions and the Company’s estimated offering expenses.

● In June 2025, the Phase 1b portion of the CELC-G-201 study reported positive topline data, and additional preliminary results will be presented at a medical conference later this year.

○ As of the May 30, 2025 data cut-off, the preliminary efficacy and safety analyses for the combined arms showed the six-month radiographic progression free survival rate was 66%, no patients discontinued treatment due to a treatment-related AE, and no dose reductions were required with gedatolisib or darolutamide.

● In June 2025, the Company presented key efficacy and safety results from an investigator-sponsored Phase 2 clinical trial in 44 patients with HER2+/ PIK3CA mutated mBC, who were treated with gedatolisib plus standard doses of trastuzumab-pkrb, at the American Society of Clinical Oncologists meeting, which showed the objective response rate among all patients enrolled was 43%.

Second Quarter 2025 Financial Results

Unless otherwise stated, all comparisons are for the second quarter ended June 30, 2025, compared to the second quarter ended June 30, 2024.

Total operating expenses were $44.0 million for the second quarter of 2025, compared to $24.3 million for the second quarter of 2024.

Research and development ("R&D") expenses were $40.2 million for the second quarter of 2025, compared to $22.5 million for the prior-year period. Of the approximately $17.7 million increase in R&D expenses, $6.6 million was related to increased employee and consulting expenses, $6.1 million was related to increased research and development costs primarily attributable to activities supporting our ongoing clinical trials, and $5.0 million is related to an anticipated development milestone payment under the license agreement with Pfizer.

General and administrative ("G&A") expenses were $3.8 million for the second quarter of 2025, compared to $1.8 million for the prior-year period. Of the approximately $2.0 million increase in general and administrative expenses, $1.6 million was related to increased employee and consulting expenses. The remaining $0.4 million of the $2.0 million increase resulted from professional fees, expanding infrastructure and other administrative expenses.

Net loss for the second quarter of 2025 was $45.3 million, or $1.04 loss per share, compared to a net loss of $23.7 million, or $0.62 loss per share, for the second quarter of 2024. Non-GAAP adjusted net loss for the second quarter of 2025 was $40.5 million, or $0.93 loss per share, compared to non-GAAP adjusted net loss of $22.2 million, or $0.58 loss per share, for the second quarter of 2024. Non-GAAP adjusted net loss excludes stock-based compensation expense, non-cash interest expense, and non-cash interest income. Because these items have no impact on Celcuity’s cash position, management believes non-GAAP adjusted net loss better enables Celcuity to focus on cash used in operations. For a reconciliation of financial measures calculated in accordance with generally accepted accounting principles in the United States ("GAAP") to non-GAAP financial measures, please see the financial tables at the end of this press release.

Net cash used in operating activities for the second quarter of 2025 was $36.2 million, compared to $18.1 million for the second quarter of 2024.

At June 30, 2025, Celcuity reported cash, cash equivalents and short-term investments of $168.4 million. However, on a proforma basis, taking into account the net proceeds of the Celcuity’s financing activities in the third quarter, cash, cash equivalents, and short-term investments as of the end of Q2 2025 was $455 million, which, we believe, when including drawdowns on our debt facility, will fund operations through 2027.

Webcast and Conference Call Information

The Celcuity management team will host a webcast/conference call at 4:30 p.m. ET today to discuss the second quarter 2025 financial results and provide a corporate update. To participate in the teleconference, domestic callers should dial 1-800-717-1738 and international callers should dial 1-646-307-1865. A live webcast presentation can also be accessed using this weblink: View Source;tp_key=047c56f7b8. A replay of the webcast will be available on the Celcuity website following the live event.