On October 19, 2025 Novartis reported new Pluvicto (lutetium (177Lu) vipivotide tetraxetan) data from the Phase III PSMAddition trial in a Presidential Symposium at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2025.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Pluvicto plus standard of care (SoC) (androgen receptor pathway inhibitor [ARPI] + androgen deprivation therapy [ADT]) demonstrated a statistically significant and clinically meaningful improvement in radiographic progression-free survival (rPFS), reducing the risk of radiographic progression or death by 28% (HR 0.72; 95% CI: 0.58, 0.90) versus SoC alone in patients with prostate-specific membrane antigen (PSMA)+ metastatic hormone-sensitive prostate cancer (mHSPC)1.

Results also show an early positive trend in overall survival (OS) in patients treated with Pluvicto plus SoC (HR 0.84; 95% CI: 0.63, 1.13); follow-up will continue until data are mature1. More patients achieved a complete response versus SoC alone (57.1% vs. 42.3%) and the overall response rate (ORR) was numerically higher in the Pluvicto plus SoC arm (85.3% vs. 80.8%)1. Pluvicto delayed time to progression to metastatic castration-resistant prostate cancer (mCRPC) (HR 0.70; 95% CI: 0.58, 0.84)1. The rPFS benefit was consistent across pre-specified subgroups1.

"In metastatic prostate cancer, choosing the most efficacious treatment early is crucial, even at initial diagnosis," said Scott T. Tagawa, MD, a professor of medicine at Weill Cornell Medicine and a medical oncologist at NewYork-Presbyterian/Weill Cornell Medical Center. "These findings suggest that combining 177Lu-PSMA-617 with standard of care hormonal therapy offers patients more time without disease progression, a safety profile with adverse events that are most often low grade and managed with supportive care, and an encouraging trend in overall survival."

"These results reinforce the potential for Pluvicto, a radioligand therapy that delivers treatment directly to target cells, to change how we treat metastatic prostate cancer," said Shreeram Aradhye, President, Development and Chief Medical Officer, Novartis. "With significant benefit now shown across multiple disease stages, Pluvicto is redefining the standard of care. The strength of these results reflects our deep commitment to patients with prostate cancer and our leadership in radioligand therapy."

The safety profile and tolerability of Pluvicto were consistent with its established profile in PSMAfore and VISION1,4,5. Grade ≥3 adverse events (AEs) were reported in 50.7% of patients in the Pluvicto plus SoC arm, compared to 43% on SoC alone1. The most common all-grade AEs were dry mouth, fatigue, nausea, hot flush and anemia1.

PSMAddition marks the third positive Phase III trial with Pluvicto1,4,5. Building on the significant benefit demonstrated in PSMAfore, which led to the US Food and Drug Administration (FDA) approval in pre-taxane mCRPC in March 2025, these new results strengthen the evidence base for Pluvicto and demonstrate its potential to improve outcomes in an even earlier stage of metastatic prostate cancer1,4,6. Novartis plans to submit these data to regulatory authorities before end of year.

About unmet need in mHSPC
Approximately 172,000 men are diagnosed with mHSPC each year across the US, China, Japan, France, Germany, Italy, Spain and the United Kingdom1. Most patients progress to mCRPC, typically within 20 months2,3,7,8. Progression to mCRPC is associated with significantly worse outcomes, including increased patient burden, worse quality of life and life expectancy less than two years9,10. More than 80% of patients with prostate cancer highly express the PSMA biomarker, making it a promising therapeutic target11-15.

About PSMAddition
PSMAddition (NCT04720157) is a Phase III, open-label, prospective, 1:1 randomized study comparing the efficacy and safety of Pluvicto in combination with SoC (ARPI + ADT) vs. SoC alone in adult patients with PSMA+ mHSPC16. The primary endpoint is rPFS, defined as the time to radiographic progression by PCWG3-modified RECIST V1.1 (as assessed by BIRC) or death16. The key secondary endpoint of OS is defined as time to death due to any cause16. The study remains ongoing and a total of 1,144 patients with mHSPC across 20 countries have been randomized in the trial16.

About Pluvicto (lutetium (177Lu) vipivotide tetraxetan)
Pluvicto is an intravenous RLT that combines a targeting compound (a ligand) with a therapeutic radionuclide (a radioactive particle, in this case lutetium-177)5,17. After administration into the bloodstream, Pluvicto binds to PSMA-expressing target cells, including prostate cancer cells that express PSMA, a transmembrane protein5,17. Once bound, energy emissions from the radioisotope damage the target cells and nearby cells, disrupting their ability to replicate and/or triggering cell death17.

Pluvicto is the only PSMA-targeted agent approved for PSMA+ mCRPC and is the first RLT to demonstrate a clinical benefit for patients with PSMA+ mHSPC in a Phase III trial1. Novartis is investigating Pluvicto in oligometastatic prostate cancer, an earlier stage of disease, in the PSMA-DC trial (NCT05939414).

(Press release, Novartis, OCT 19, 2025, View Source [SID1234656773])

On October 19, 2025 NETRIS Pharma, a clinical-stage biotechnology company pioneering novel therapies targeting Netrin-1 to overcome resistance to chemotherapy and immunotherapy, reported the presentation of the first results from its ongoing Phase 2 clinical trial IMMUNONET. The Study evaluates NP137 in combination with anti–PD-1/PD-L1 therapies with advanced solid tumors. The data will be shared in a poster session at the upcoming ESMO (Free ESMO Whitepaper) Oncology Congress 2025 in Berlin, Germany.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Resistance to Immune Checkpoint Inhibitors (ICI) remains a major challenge in the treatment of solid tumors such as non-small cell lung cancer (NSCLC) and head and neck squamous cell carcinoma (HNSCC). Netrin-1, an embryonic guidance factor re-expressed in various cancers, contributes to tumor resistance and epithelial-to- mesenchymal transition (EMT). NP137, a first-in-class monoclonal antibody targeting Netrin-1, has demonstrated the ability to inhibit EMT and modulate the tumor microenvironment, thereby potentially restoring sensitivity to immunotherapy.

IMMUNONET (NCT05605496) was designed to evaluate whether NP137 was able to (re)-sensitize solid tumors to ICI. Patients who had progressed under a prior anti–PD-1/PD-L1 were enrolled to receive NP137 as an add–on to their immunotherapy. They were enrolled in 3 distinct cohorts depending on their best response and time to progression (cohort 1, stable disease; cohort 2, primary refractory and cohort 3, secondary refractory). The study was designed as a 2-stage adaptive design. This poster reports the results of stage 1. In cohort 3 (secondary refractory) where a majority of NSCLC and HNSCC patients were enrolled, the primary endpoint of Progression-Free Rate at 12 weeks (PFR-12W) was met ahead of stage 2. These results strongly suggest the efficacy of combining NP137 with anti–PD-1/PD-L1 therapy in this difficult-to-treat population and warrant further controlled studies to confirm these findings.

Importantly, the combination of NP137 with ICI was very well tolerated. « These preliminary results are particularly encouraging » said Dr Jérome Fayette, M.D., Ph.D., Principal Investigator of the study. « Patients who have progressed after prior anti–PD-1/PD-L1 therapy represent one of the most difficult populations to treat. The encouraging efficacy and safety results observed in this study strongly support the continued clinical development of NP137. Advancing to a confirmatory randomized study will allow to further validate these findings and better define the therapeuticpotential of NP137 in combination with immune checkpoint inhibitors for patients with advanced solid tumors ».

« Observing durable disease control in this setting supports the concept that targeting Netrin-1 can re-sensitize tumors to immune checkpoint inhibition » added Dr. Sébastien Hazard, Chief Medical Officer of NETRIS Pharma. « Combining NP137 with checkpoint inhibitors may offer a new option for patients who no longer benefit from standard immunotherapy ».

Poster Details

Title: NP137 combined with anti–PD-1/PD-L1 therapy in ICI-pretreated solid tumors: Interim efficacy and safety results from the IMMUNONET study
Session: 966P
First Author: Dr. Jerome Fayette
About EIC Accelerator

The EIC Accelerator supports individual Small and Medium Enterprises (SMEs), in particular Startups and spinout companies to develop and scaleup game-changing

innovations. ImmunoNET is co-funded by the European Union. Views and opinions expressed are however those of the author(s) only and do not necessarily reflect those of the European Union or NETRIS Pharma. Neither the European Union nor NETRIS Pharma can be held responsible for them.

(Press release, Netris Pharma, OCT 19, 2025, View Source [SID1234656772])

On October 19, 2025 The European Organisation for Research and Treatment of Cancer (EORTC) reported that the EORTC 1333/PEACE-3 trial has reached its final overall survival (OS) endpoint at the time of the final database lock on September 19, 2025.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The EORTC 1333/PEACE-3 study is a randomized, open-label, multicentre phase III trial conducted in collaboration with Clinical Trial Ireland (CTI), the Canadian Urological Oncology Group (CUOG), the Latin American Cooperative Oncology Group (LACOG), and the French group GETUG/UNICANCER. A total of 446 asymptomatic or mildly symptomatic patients (Brief Pain Inventory score < 4) with metastatic castration-resistant prostate cancer (mCRPC) and ≥2 bone metastases were randomized 1:1 to receive either enzalutamide (160 mg daily) alone or enzalutamide plus six intravenous injections of radium-223 (55 kBq/kg every four weeks).

In the primary analysis, published in Annals of Oncology, the addition of six cycles of radium-223 to enzalutamide significantly improved the primary endpoint radiological progression-free survival (rPFS) from 16.4 months (95% CI 13.8–19.2) to 19.4 months (95% CI 17.1–25.3) (hazard ratio [HR] 0.69; 95% CI 0.54–0.87; p = 0.0009)¹, and an interim analysis at 80% of events that suggested an OS advantage for combining enzalutamide and Ra223.

The final overall survival analysis now confirms that the addition of six cycles of radium-223 to enzalutamide significantly prolongs overall survival, thereby reinforcing the findings of the interim analysis reported at the time of the primary publication.

Comprehensive results will be presented soon.

This trial is supported by an investigator driven clinical trial agreement from Bayer HealthCare Pharmaceuticals Inc. and Astellas Pharma Europe.

(Press release, EORTC, OCT 19, 2025, View Source [SID1234656768])

On October 19, 2025 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, reported new late-breaking data from its pivotal Phase 3 ROSELLA trial of relacorilant plus nab-paclitaxel in patients with platinum-resistant ovarian cancer at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2025 Annual Meeting. The data demonstrated a progression-free survival (PFS) benefit for patients who experienced disease progression while on or after taking a PARP inhibitor (PARPi), a patient population with particularly poor prognosis. The presentation slides can be found here. The company also announced expansion of the Phase 2 BELLA trial in a poster session at ESMO (Free ESMO Whitepaper), found here.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

New relacorilant data presented at ESMO (Free ESMO Whitepaper) demonstrated a consistent benefit in PARPi subgroups to overcome chemotherapy resistance. Relacorilant plus nab-paclitaxel showed a PFS benefit in patients with prior PARPi treatment (hazard ratio: 0.60; p-value: 0.0035) and in patients whose disease progressed while on a PARPi (hazard ratio: 0.56; p-value: 0.0046), with a median PFS of 7.36 months for both subgroups. Relacorilant plus nab-paclitaxel was well-tolerated in the PARPi subgroups, consistent with its known safety profile. Importantly, the type, frequency and severity of adverse events in the combination arms were comparable to those in the nab-paclitaxel monotherapy arms. Relacorilant conferred its benefit without increasing the safety burden of the patients who received it.

"These new ROSELLA data substantiate the significant benefit of relacorilant plus nab-paclitaxel in platinum-resistant ovarian cancer, an extremely difficult-to-treat cancer," said Domenica Lorusso, M.D., Ph.D., Director of the Gynaecological Oncology Unit at Humanitas Hospital San Pio X, Milan, and Full Professor of Obstetrics and Gynaecology, Humanitas University, Rozzano, European Network of Gynaecological Oncological Trial groups Principal Investigator in the ROSELLA trial and ESMO (Free ESMO Whitepaper) presenter. "These findings are especially promising for patients with an exceptionally poor prognosis and necessitate further research of relacorilant and its potential benefit in earlier treatment lines of gynecological cancers."

Corcept also announced the expansion of the recently initiated Phase 2 BELLA trial to three study arms to evaluate the efficacy and safety of: (i) relacorilant plus nab-paclitaxel and bevacizumab to treat patients with platinum-resistant ovarian cancer; (ii) relacorilant plus nab-paclitaxel and bevacizumab to treat patients with platinum-sensitive ovarian cancer whose disease progressed while on a PARPi and (iii) relacorilant plus nab-paclitaxel to treat patients with endometrial cancer who had received one or two prior lines of therapy. Initial results are expected in late 2026.

"The ROSELLA data give us confidence to expand the BELLA trial to include patients with ovarian cancer whose disease is platinum sensitive (an earlier stage of tumor progression), as well as patients with endometrial cancer," said Bill Guyer, PharmD, Corcept’s Chief Development Officer. "With the FDA’s recent acceptance of our New Drug Application for relacorilant in platinum-resistant ovarian cancer and the expansion of our BELLA trial, we are closer than ever to bringing new and vital treatment to patients in need. We are grateful to all the patients and investigators for participating in our ongoing trials."

Corcept previously announced that ROSELLA met its primary endpoint of improved PFS, with no need for biomarker selection. Patients who received relacorilant in addition to nab-paclitaxel chemotherapy experienced a 30 percent reduction in the risk of disease progression compared to patients who received nab-paclitaxel monotherapy (hazard ratio: 0.70; p-value: 0.0076). An interim analysis of overall survival (OS) showed that the addition of relacorilant reduced the risk of death by 31 percent, substantially lengthening patients’ lives (hazard ratio: 0.69; p-value: 0.0121).

The ROSELLA trial is being conducted in collaboration with The GOG Foundation, Inc. (GOG-F), the European Network of Gynaecological Oncological Trial groups (ENGOT), the Asia-Pacific Gynecologic Oncology Trials Group (APGOT), the Latin American Cooperative Oncology Group (LACOG) and the Australia New Zealand Gynaecological Oncology Group (ANZGOG).

About Relacorilant

Relacorilant, an oral therapy, is a selective glucocorticoid receptor (GR) antagonist that modulates cortisol activity by binding to the GR but not to the body’s other hormone receptors. Corcept is developing relacorilant in ovarian cancer and a variety of other serious disorders, including endogenous hypercortisolism and prostate cancer. Relacorilant is proprietary to Corcept and is protected by composition of matter, method of use and other patents. It has been designated an orphan drug by the FDA and the European Commission (EC) for the treatment of hypercortisolism and by the EC for the treatment of ovarian cancer. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) date of December 30, 2025 for relacorilant as a treatment for patients with hypercortisolism, and a PDUFA date of July 11, 2026 for relacorilant as a treatment for patients with platinum-resistant ovarian cancer. Corcept also recently submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for relacorilant to treat patients with platinum-resistant ovarian cancer.

About Cortisol’s Role in Oncology

Cortisol plays a role in tumor growth through several mechanisms. It helps solid tumors resist chemotherapy by inhibiting cellular apoptosis — the tumor-killing effect chemotherapy is meant to stimulate. In some cancers, cortisol promotes tumor growth by activating oncogenes in the cells to which it binds. Cortisol also suppresses the body’s immune response, which weakens its ability to fight all diseases, including cancer.

About Ovarian Cancer

Ovarian cancer is the fifth most common cause of cancer death in women. Patients whose disease returns six months after receiving platinum-containing therapy have "platinum-sensitive" disease and those with disease progression of less than six months have "platinum-resistant" disease. There are few treatment options for these women. Approximately 20,000 women with platinum-resistant disease and 13,000 women with platinum-sensitive disease are candidates to start a new therapy each year in the United States, with at least an equal number in Europe.

(Press release, Corcept Therapeutics, OCT 19, 2025, https://ir.corcept.com/news-releases/news-release-details/corcept-presents-esmo-2025-late-breaker-relacorilant [SID1234656765])

On October 18, 2025 Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company pursuing development of targeted therapies for oncology, reported detailed efficacy and safety results from the PIK3CA wild-type ("WT") cohort of the Phase 3 VIKTORIA-1 clinical trial of gedatolisib, an investigational pan-PI3K/mTORC1/2 inhibitor, in adults with hormone receptor positive ("HR+"), human epidermal growth factor receptor 2 negative ("HER2-"), PIK3CA WT, advanced breast cancer ("ABC"), following progression on, or after, treatment with a CDK4/6 inhibitor and an aromatase inhibitor. As previously announced, the gedatolisib triplet demonstrated a statistically significant and clinically meaningful improvement in median progression-free survival ("PFS") versus fulvestrant, reducing the risk of disease progression or death by 76%. The gedatolisib doublet reduced the risk of progression or death by 67% versus fulvestrant.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The detailed study results were presented at a late breaking oral presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress today, Saturday, October 18 at 4:25 a.m. ET/10:25 a.m. CEST.

In the trial, median PFS with the gedatolisib triplet was 9.3 months versus 2.0 months with fulvestrant, an incremental improvement of 7.3 months (HR=0.24; 95% CI: 0.17-0.35; p<0.0001). The objective response rate ("ORR") of the gedatolisib triplet was 31.5% compared to 1% with fulvestrant and the median duration of response ("DOR") was 17.5 months. For the gedatolisib doublet, the median PFS was 7.4 months versus 2.0 months with fulvestrant, an incremental improvement of 5.4 months (HR=0.33; 95% CI: 0.24-0.48; p<0.0001). The ORR of the gedatolisib doublet was 28.3% and the median DOR was 12.0 months. The median DOR was not determinable for fulvestrant because there was only one objective response.

The topline efficacy data from the VIKTORIA-1 PIK3CA WT cohort established several new milestones in the history of drug development for HR+/HER2- ABC:

● The hazard ratios for the gedatolisib triplet and doublet are more favorable than have ever been reported by any Phase 3 trial for patients with HR+/HER2- ABC.

● The 7.3- and 5.4-months incremental improvements in median PFS for the gedatolisib triplet and gedatolisib doublet over fulvestrant, respectively, are higher than have ever been reported by any Phase 3 trial for patients with HR+/HER2- ABC receiving at least their second line of an endocrine therapy-based regimen.
● Gedatolisib is the first inhibitor targeting the PI3K/AKT/mTOR ("PAM") pathway to demonstrate positive Phase 3 results in patients with HR+/HER2-/PIK3CA WT ABC whose disease progressed on or after treatment with a CDK4/6 inhibitor.
● The median DOR and incremental ORR improvement relative to control for the gedatolisib triplet and doublet are the highest reported for an endocrine therapy-based regimen in 2L HR+/HER2- ABC.

The median PFS benefit of the gedatolisib triplet and doublet compared to fulvestrant was consistent across subgroups with the gedatolisib triplet showing higher clinical benefit in nearly all subgroups compared to the gedatolisib doublet, particularly for patients who were pre/perimenopausal, endocrine therapy resistant, or had visceral metastases. For patients enrolled in the United States and Canada, median PFS was 19.3 months (HR=0.13; 90% CI: 0.07-0.29) for the gedatolisib triplet and 14.9 months (HR=0.35; 90% CI: 0.17-0.76) for the gedatolisib doublet.

Sara Hurvitz, MD, Senior Vice President, Clinical Research Division, Fred Hutchinson Cancer Center, Smith Family Endowed Chair in Women’s Health, Professor and Head, Division of Hematology and Oncology, University of Washington, Department of Medicine and co-principal investigator for the trial, said: "VIKTORIA-1 is the first study to demonstrate a statistically significant and clinically meaningful improvement in median PFS with inhibition of the PI3K/AKT/mTOR pathway in patients with PIK3CA wild-type disease, all of whom previously received a CDK4/6 inhibitor. With these results, the gedatolisib regimens represent a new potential standard of care for patients with HR+, HER2-negative, PIK3CA wild-type advanced breast cancer whose disease progressed on or after treatment with a CDK4/6 inhibitor."

The gedatolisib triplet and doublet were generally well tolerated in the trial with mostly low-grade treatment-related adverse events ("TRAEs"). The most common grade 3 TRAEs for the gedatolisib triplet, gedatolisib doublet, and fulvestrant groups included neutropenia (52.3%, 0%, and 0.8% of patients, respectively); stomatitis (19.2%, 12.3%, and 0%) rash (4.6%, 5.4%, and 0%); and hyperglycemia (2.3%, 2.3%, and 0%). The primary grade 4 TRAEs for the gedatolisib triplet and gedatolisib doublet groups were neutropenia (10.0% and 0.8%, respectively), leukopenia (0.8% in the gedatolisib triplet group) and pneumonitis (0.8% in gedatolisib doublet group). TRAEs led to the discontinuation of study treatment in 2.3% of patients in the gedatolisib triplet group, 3.1% in the gedatolisib doublet group, and 0% in the fulvestrant group.

Overall survival, a key secondary endpoint in VIKTORIA-1, while immature at the time of the analysis, with less than one-half of the required number of events having occurred, showed promising trends for both the gedatolisib triplet and doublet.

Igor Gorbatchevsky, MD, Chief Medical Officer of Celcuity, said: "We are very excited that treatment with gedatolisib combined with fulvestrant with or without palbociclib was well-tolerated by the VIKTORIA-1 patients and that only a few patients discontinued treatment due to an adverse event. This safety profile combined with the 7.3 and 5.4-months incremental improvement in median PFS relative to fulvestrant for the gedatolisib regimens, offer potentially paradigm shifting results for patients with HR-positive, HER2-negative, PIK3CA wild-type advanced breast cancer."

Celcuity initiated a rolling New Drug Application ("NDA") submission in conjunction with the U.S. Food and Drug Administration’s ("FDA") Real-Time Oncology Review program, based on data from the PIK3CA wild-type cohort of the Phase 3 VIKTORIA-1 clinical trial. Completion of the NDA submission is targeted for the fourth quarter of 2025. The PIK3CA mutant cohort of the Phase 3 VIKTORIA-1 trial is 100% enrolled and is expected to report topline data for this cohort in late Q1 2026 or during Q2 2026.

Webcast and Conference Call Information

The Celcuity management team will host a webcast/conference call on Monday, October 20, 2025, at 8:00 a.m. ET to discuss the additional results from the Phase 3 VIKTORIA-1 trial. Those who would like to participate may access the live webcast here or register in advance for the teleconference here. A replay of the webcast will be available on the Celcuity website following the live event.

Notes

HR+/HER2- Breast cancer

Breast cancer is the second most common cancer and one of the leading causes of cancer-related deaths worldwide.1 More than two million breast cancer cases were diagnosed globally in 2022.1 While survival rates are high for those diagnosed with early breast cancer, approximately 30% of patients who are diagnosed with or who progress to metastatic disease are expected to live five years after their diagnosis.2 HR+/HER2- breast cancer is the most common subtype of breast cancer, accounting for approximately 70% of all breast cancers.

Three interconnected signaling pathways, estrogen, cyclin D1-CDK4/6, and PI3K/AKT/mTOR (PAM), are primary oncogenic drivers of HR+, HER2- breast cancer.3 Therapies inhibiting these pathways are approved and used in various combinations for advanced breast cancer. Currently approved inhibitors of the PAM pathway for breast cancer target a single PAM pathway component, such as PI3Kα, AKT, or mTORC1.4,5,6,7 However, resistance to CDK4/6 inhibitors and current endocrine therapies develops in many patients with advanced disease.8 Optimizing the inhibition of the PAM pathway is an active area of focus for breast cancer research.

VIKTORIA-1

VIKTORIA-1 is a Phase 3 open-label, randomized clinical trial to evaluate the efficacy and safety of gedatolisib in combination with fulvestrant with or without palbociclib in adults with HR+/HER2- ABC whose disease progressed on or after prior CDK4/6 therapy in combination with an aromatase inhibitor. The clinical trial is fully enrolled. The trial enrolled subjects regardless of PIK3CA status while enabling separate evaluation of subjects according to their PIK3CA status. Subjects who met eligibility criteria and did not have confirmed PI3KCA mutations (WT) were randomly assigned (1:1:1) to receive a regimen of either gedatolisib, palbociclib, and fulvestrant, gedatolisib and fulvestrant, or fulvestrant. Subjects who met eligibility criteria and had confirmed PI3KCA mutations (MT) were randomly assigned (3:3:1) to receive a regimen of either the gedatolisib triplet, alpelisib and fulvestrant, or the gedatolisib doublet.

Gedatolisib

Gedatolisib is an investigational, multi-target PAM inhibitor that potently targets all four class I PI3K isoforms, mTORC1, and mTORC2 to induce comprehensive blockade of the PAM pathway.9,10,11 As a multi-target PAM inhibitor, gedatolisib’s mechanism of action is highly differentiated from currently approved single-target inhibitors of the PAM pathway.11 Inhibition of only a single PAM component gives tumors an escape mechanism through cross-activation of the uninhibited targets. Gedatolisib’s comprehensive PAM pathway inhibition ensures full suppression of PAM activity by eliminating adaptive resistance cross-activation that occurs with single-target inhibitors. Unlike single-target inhibitors of the PAM pathway, gedatolisib has demonstrated equal potency and comparable cytotoxicity in PIK3CA-mutant and wild-type breast tumor cells in nonclinical studies and early clinical data.

(Press release, Celcuity, OCT 18, 2025, View Source [SID1234656812])