Akt inhibitors(Proto-oncogene proteins c-akt inhibitors), PI3K family inhibitors(Phosphatidylinositol 3-kinase family inhibitors), mTORC1 inhibitors(Mammalian target of Rapamycin (mTORC1) inhibitors)

Therapeutic Areas

NeoplasmsSkin and Musculoskeletal DiseasesUrogenital Diseases

Active Indication

Hormone receptor positive HER2 negative breast cancerMetastatic castration-resistant prostate cancerAdvanced Triple-Negative Breast Carcinoma+ [2]

Inactive Indication

Advanced cancerAdvanced Head and Neck CarcinomaAdvanced Lung Squamous Cell Carcinoma+ [14]

Originator Organization

Pfizer Inc.

Active Organization

Pfizer Inc.

Celcuity, Inc.

Inactive Organization-

License Organization

Celcuity, Inc.

Pfizer Inc.

Drug Highest PhaseApproved

First Approval Date

United States (14 Jul 2026),

Hormone receptor positive HER2 negative breast cancer

RegulationBreakthrough Therapy (United States), Fast Track (United States), Priority Review (United States)

Structure/Sequence

Molecular FormulaC32H41N9O4

InChIKeyDWZAEMINVBZMHQ-UHFFFAOYSA-N

CAS Registry1197160-78-3

Clinical Trials associated with Gedatolisib

NCT06757634

/ RecruitingPhase 3

VIKTORIA-2: A Randomized, Open-Label, Phase 3 Study Evaluating Efficacy and Safety of Gedatolisib With Endocrine Therapy and Palbociclib vs Endocrine Therapy and Ribociclib as First-Line Treatment in Patients With HR-Positive and HER2-Negative Advanced Breast Cancer

This is a Phase 3, open-label, randomized, clinical trial evaluating the efficacy and safety of gedatolisib and palbociclib plus endocrine therapy for the treatment of patients with locally advanced or metastatic HR+/HER2- advanced breast cancer.

Start Date24 Jul 2025

Sponsor / Collaborator

Celcuity, Inc.

NCT06190899

/ RecruitingPhase 1/2

A Phase 1/2, Open-Label, Randomized, Dose Finding and Dose Expansion Study of Gedatolisib in Combination With Darolutamide in Metastatic Castration-Resistant Prostate Cancer (mCRPC)

This is a Phase 1/2, open-label, randomized, dose finding and dose expansion study to evaluate the safety, preliminary efficacy, and PK of gedatolisib in combination with darolutamide in subjects with mCRPC.

Start Date01 Jan 2024

Sponsor / Collaborator

Celcuity, Inc.

NCT05501886

/ Active, not recruitingPhase 3

Phase 3, Open-Label, Randomized, Study Comparing Gedatolisib Combined With Fulvestrant & With or Without Palbociclib to Standard-of-Care Therapies in Patients With HR-Positive, HER2-Negative Advanced Breast Cancer Previously Treated With a CDK4/6 Inhibitor in Combination w/Non-Steroidal Aromatase Inhibitor Therapy

This is a Phase 3, open-label, randomized, clinical trial evaluating the efficacy and safety of gedatolisib plus fulvestrant with or without palbociclib for the treatment of patients with locally advanced or metastatic HR+/HER2- breast cancer following progression on or after CDK4/6 and aromatase inhibitor therapy.

Start Date08 Dec 2022

Sponsor / Collaborator

Celcuity, Inc.

100 Clinical Results associated with Gedatolisib

100 Translational Medicine associated with Gedatolisib

100 Patents (Medical) associated with Gedatolisib

122

Literatures (Medical) associated with Gedatolisib

01 Jul 2026·EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

Discovery of novel bis-aryl urea-linked triazine derivatives as dual PI3K/mTOR inhibitors via scaffold hopping strategy and biological activity evaluations

Article

Author: Qiao, Dan ; Zhang, Dajun ; Cheng, Zhenjie ; Zheng, Pengwu ; Xu, Shan ; Yang, Yang ; Wang, Ran ; Peng, Rujue ; Wang, Linxiao

Phosphatidylinositol 3-kinase (PI3K) and mammalian target of rapamycin (mTOR) are overexpressed in breast cancer and drive oncogenesis, rendering PI3K/mTOR inhibitors as promising therapeutic agents. However, tumor cells readily develop resistance to single-agent PI3K or mTOR inhibitors. In this study, 40 novel bis-aryl urea-linked triazine derivatives were designed and synthesized as dual PI3K/mTOR inhibitors using a scaffold hopping strategy. Their biological activities were evaluated. The results showed that J-33 was a dual inhibitor of PI3K and mTOR kinases, with IC₅₀ values of 400.5 nM and 8.2 nM, respectively, and it inhibited other tested kinases by less than 50%. The antiproliferative IC₅₀ value of J-33 against MCF-7 cells was 1.5 ± 0.2 μM. Hemolysis assays indicated that J-33 exhibited low hemolytic toxicity. Apoptosis and AO staining experiments demonstrated that J-33 induced apoptosis in MCF-7 cells in a concentration-dependent manner. Western blot analysis showed that J-33 significantly downregulated the phosphorylation level of the PI3K-AKT-mTOR pathway. Therefore, we conducted in vivo antitumor studies using a nude mouse model with MCF-7 cell xenografts. The results demonstrated that at the same dose of 75 mg/kg, J-33 exhibited a higher tumor inhibition rate (44.9%) compared to PKI-587 (43.6%). In summary, a highly potent and low-toxic dual PI3K/mTOR inhibitor was developed, which deserves further investigation.

20 Apr 2026·JOURNAL OF CLINICAL ONCOLOGY

VIKTORIA-1 Trial of Gedatolisib Plus Fulvestrant With or Without Palbociclib in Hormone Receptor–Positive/HER2−/PIK3CA Wild-Type Advanced Breast Cancer

Article

PURPOSE:

Gedatolisib potently targets all four class I PI3K isoforms and mTORC1 and mTORC2 to comprehensively block the PI3K/AKT/mTOR pathway and has shown compelling activity in early clinical trials with palbociclib and fulvestrant.

METHODS:

This phase III randomized trial (VIKTORIA-1; ClinicalTrials.gov identifier: NCT05501886 ) evaluated the efficacy of gedatolisib-based therapy, comparing gedatolisib, palbociclib, and fulvestrant (gedatolisib triplet) and gedatolisib plus fulvestrant (gedatolisib doublet) with fulvestrant monotherapy in patients with hormone receptor–positive, human epidermal growth factor receptor 2–negative (HER2−), PIK3CA wild-type (WT) advanced breast cancer. Eligible patients had disease progression during or after CDK4/6 inhibitor and aromatase inhibitor treatment. Comparison of progression-free survival as assessed by blinded independent central review for gedatolisib triplet versus fulvestrant and gedatolisib doublet versus fulvestrant was the primary objective.

RESULTS:

A total of 392 patients were randomly assigned 1:1:1. The median study follow-up was 10.1 months. The median progression-free survival was 9.3 months in the gedatolisib-triplet group, 2.0 months in the fulvestrant group (hazard ratio [HR] for progression or death, 0.24 [95% CI, 0.17 to 0.35]; P < .001), and 7.4 months in the gedatolisib-doublet group (HR, 0.33 [95% CI, 0.24 to 0.48]; P < .001 v fulvestrant). Grade ≥3 treatment-related adverse events (TRAEs) reported in the gedatolisib-triplet and gedatolisib-doublet groups, respectively, included neutropenia (62.3%, 0.8%), stomatitis (19.2%, 12.3%), rash (4.6%, 5.4%), hyperglycemia (2.3%, 2.3%), and diarrhea (1.5%, 0.8%). Study treatment discontinuation because of TRAEs was reported in 2.3% (triplet) and 3.1% (doublet) of patients.

CONCLUSION:

The addition of gedatolisib to fulvestrant, with or without palbociclib, significantly reduced the risk of disease progression or death in patients with hormone receptor–positive/HER2−, PIK3CA WT advanced breast cancer.

01 Apr 2026·THROMBOSIS AND HAEMOSTASIS

Middle-Throughput LC-MS-Based Platelet Proteomics with Minute Sample Amounts Using Semiautomated Positive Pressure FASP in 384-Well Format

Article

Author: John, Tobias ; Jurk, Kerstin ; Barkovits, Katalin ; Balkenhol, Johannes ; Heemskerk, Johan W.M. ; Klewe, Kristoffer ; Greinacher, Andreas ; Dandekar, Thomas ; Marcus, Katrin ; Loroch, Stefan ; Lerch, Jan-Paul ; Panagiotidis, Eleftherios ; Swieringa, Frauke ; Walter, Ulrich

Abstract:

Comprehensive characterization of platelets requires various functional assays and analytical techniques, including omics disciplines, each demanding a separate aliquot of the given sample. Consequently, sample material for each assay is often highly limited, necessitating the downscaling of methods to work with just a few micrograms of platelet protein.Here, we present a novel sample preparation platform for proteomics analysis using only 3 μg of purified platelet protein, corresponding to 2 × 106 platelets, which can be obtained from approximately 2 to 8 μL of blood from a healthy individual (1.5 × 105–4.5 × 105 platelets/μL) or approximately 100 μL of blood from a patient with severe thrombocytopenia (<2 × 104 platelets/µL).Using this platform, we detected a significant fraction of key players in the platelet activation cascade and, most importantly, identified 36 clinically relevant platelet disease markers even with a non-state-of-the art instrument. This makes LC-MS-based proteomics a highly attractive alternative to conventional assays, which often require milliliters of blood. Our platform transitions from our previously established 96-well proteomics workflow (PF96), which has been successfully employed in numerous platelet proteomics studies, into the 384-well format. This transition is accompanied by (1) a more than two-fold increase in sensitivity, (2) improved reproducibility, (3) a four-fold increase in throughput, allowing 1,536 samples to be processed per lab worker per week, and (4) reduced sample preparation costs.Thus, LC-MS-based platelet proteomics offers a compelling alternative to immunoaffinity assays (which depend on antibody availability and quality), as well as to genomic assays (which can only reveal genotypes). In summary, in conjunction with recent advances in LC-MS instrumentation, our platform represents a highly valuable tool for rapid phenotyping of platelets in research with extraordinary potential for future employment in companion or routine diagnostics.

118

News (Medical) associated with Gedatolisib

16 Jul 2026

·www.fredhutch.org

FDA approves new breast cancer drug based on VIKTORIA-1 study results

Watch out, metastatic ER+/HER2- breast cancer. There’s a new targeted agent in town. On Tuesday, the U.S. Food and Drug Administration approved the drug gedatolisib, a multi-targeted kinase inhibitor capable of squelching numerous parts of a key signaling pathway often co-opted by cancer to drive tumor growth and treatment resistance in patients. Made by the Minneapolis-based biotech firm Celcuity, the new drug ― dubbed Revtorpyk ― is designed to be used in combination with the anti-hormone drug fulvestrant, with or without a targeted CDK4/6 inhibitor, in patients with estrogen-receptor-positive or ER+/HER2-negative locally advanced or metastatic breast cancer, or mBC. The ER+/HER2- subtype is found in 60% to 70% of all mBC patients. Sara Hurvitz, MD, senior vice president and director of Fred Hutch Cancer Center’s Clinical Research Division, led the multicenter VIKTORIA-1 trial, which generated buzz when she shared results from Study 1 at the 2025 European Society for Medical Oncology (ESMO) meeting, recently published in the Journal of Clinical Oncology. Hurvitz shared trial results of VIKTORIA-1 Study 2 at the annual American Society of Clinical Oncology (ASCO) meeting last month. Built as two parallel studies under one giant umbrella, VIKTORIA-1 was launched to evaluate the new agent’s potential in inhibiting multiple parts of the PAM, or PI3K/AKT/mTOR, pathway. Trial participants in Study 1, which enrolled patients without a mutation in the gene for PI3K, were separated into three groups. Patients given the “doublet” treatment received gedatolisib with fulvestrant; those given the “triplet” treatment received gedatolisib and fulvestrant plus the targeted CDK4/6 inhibitor palbociclib, or Ibrance. Another arm of the trial had patients just take fulvestrant alone. All participants receiving the new agent showed significantly improved progression-free survival compared with standard of care. At the time, Hurvitz called the finding “statistically significant and clinically meaningful” and a “potential new standard of care.” Now that the FDA has granted approval, that new standard is underway. Subscribe to Fred Hutch News You will receive a monthly newsletter from us about our innovative research and compassionate care as well as updates on events and other helpful tools and tips. You may unsubscribe at any time. “The exciting thing about this study is really for the first time we’ve shown that an inhibitor that targets multiple parts of the PAM pathway and completely shuts it down is tolerable for patients,” said Hurvitz, who holds the Smith Family Endowed Chair in Women’s Health. “To my knowledge, there hasn't been a drug that’s made it through late phase that hits so many components of the pathway.” What’s so special about this pathway? The PAM, or PI3K/AKT/mTOR, pathway is sort of a master control center, one of 10 tiny intracellular signaling networks that regulate vital cellular functions like growth, proliferation, survival and metabolism. Unfortunately, it’s also a pathway that commonly goes sideways in cancer, driving tumor progression and treatment resistance through overactive PI3K, AKT or mTOR proteins. Half of all solid tumors are driven by overactivation or dysregulation of the PAM pathway. Hurvitz, who called it “a really complex pathway,” said it’s been implicated in resistance to CDK4/6 inhibitors and endocrine or anti-hormone therapy, both pillars of ER+/HER2- breast cancer treatment. “We have multiple inhibitors that hit one part of that pathway,” Hurvitz said. “And the benefit of those inhibitors has been muted. They help for a little bit, then resistance develops. And there have been attempts in the past to hit multiple parts of the pathway to prevent resistance from developing. But those were too toxic.” Gedatolisib, she said, hits multiple targets within the PAM pathway ― and does so without putting patients through a slew of debilitating side effects. “It’s less toxic than the agents we have available,” she said. “And it’s also demonstrated improved efficacy compared to the standard of care.” Currently, approved PI3K inhibitors are used in breast cancers with a PIK3CA mutation. But gedatolisib, she said, works whether the patient’s tumor has a PIK3CA mutation or not. “That makes it quite unique,” Hurvitz said. “We only have one other drug that targets this pathway (everolimus, which inhibits mTOR) that doesn’t require that mutation for efficacy.” According to the FDA, gedatolisib (aka Revtorpyk) is approved for patients without the PIK3CA mutation, so many more patients will be eligible to receive and potentially benefit from the new drug. “A PIK3CA mutation is a somatic mutation that occurs in about 40% of patients with ER+ metastatic breast cancer,” she said. “It is usually not acquired; if it’s there at the beginning, it'll be there throughout the disease. For 60% of patients not to be eligible for a therapy because it requires a mutation is kind of a big miss.” The newly approved agent is designed for ER+/HER2- locally advanced or metastatic breast cancer patients whose disease has progressed after being on at least one line of endocrine therapy in the metastatic setting. Patients with ER+/HER2- mBC traditionally receive treatments designed to target three signaling pathways shown to drive cancer growth. The estrogen-fueled pathway is targeted by anti-hormone agents (think Tamoxifen, Anastrozole or Fulvestrant) and the cyclin D1-CDK4/6-dependent pathway, which drives cell division, is targeted by a handful of CDK4/6 inhibitors such as Ibrance, Kisqali and Verzenio. The complex PI3K/AKT/mTOR or PAM pathway, however, is currently targeted only by drugs that inhibit a single part of it, such as Piqray, a PI3K inhibitor; Truqap, an AKT inhibitor and Afinitor, which blocks the mTOR complex. The unique design of VIKTORIA-1 allowed researchers to tease out how the new drug would respond in a number of different scenarios, including how it would perform with more than one therapy (endocrine plus CDK4/6 inhibitors) and how it would perform in those who had an unmutated or “wild type” PIK3CA gene. Additional VIKTORIA trials will see how the new drug performs with other CDK4/6 inhibitors and anti-hormone drugs, as well as how it performs in patients who have not received previous treatment. Hurvitz was equally enthusiastic about the results of VIKTORIA-1 Study 2. This study, which enrolled patients with a PIK3CA tumor mutation, showed the gedatolisib triplet and doublet regimens significantly improved median progression-free survival compared to fulvestrant/Piqray. “FDA approval may soon be expanded to these patients as well,” she said. “Both of the gedatolisib arms showed a substantially better outcome than just using fulvestrant alone. That was in Study 1. And then in Study 2, where it was just patients with PIK3CA tumor mutations, the triplet and doublet performed very similarly to one another. And they both did better than fulvestrant with alpelisib [Piqray], which is the standard PI3K inhibitor that was in the study.” That’s the current standard of care for many metastatic breast cancer patients with ER+/HER2- tumors. And the new drug performed better. “This drug hits all four isoforms of PI3 kinase class one and mTORC1 and mTORC2,” Hurvitz said. “We were all surprised when we saw the Phase 1 data. We’re like, ‘Wow, this looks really active, and it looks really tolerable,’ but you never know if in a larger patient population, it’s going to continue to hold water.” Luckily for patients, the trial results did hold water. VIKTORIA-1 Study 1 trial participants who received gedatolisib nearly doubled the amount of time they went without any kind of cancer progression. Those on the triplet regimen went 9.3 months before progression and those on the doublet regimen went 7.4 months. The control group, which received fulvestrant alone, had a median progression-free survival of two months. And there were far fewer side effects. Hurvitz said drugs that inhibit parts of the PI3K pathway often cause high blood sugar or hyperglycemia, because PI3K is involved in the way we metabolize glucose in the body. “It's an on-target toxicity we expect to see,” she said. “We also see diarrhea and rash in a lot of patients. And what was sort of surprising to all of us was that we didn’t really see diarrhea. Around 1.5% of patients had it, but it was mild, grade 1 or 2 compared to approximately 40% all-grade diarrhea which is what you see with alpelisib.” Hyperglycemia was also very low, she said, experienced by only 2.3% of participants, and there was no grade 3 hyperglycemia in the gedatolisib-fulvestrant arm at all. In contrast, she said, 55% to 60% of patients on alpelisib have hyperglycemia. “The one side effect we saw that’s important to be aware of with this drug was stomatitis or mouth sores,” she said. “They occurred at a higher rate, so we did mandate in these studies that patients receive steroid mouthwash four times a day. They just swish and spit.” Gedatolisib is an intravenous infusion designed to be given three times a month in combination with fulvestrant, an intramuscular injection given once a month, with or without Ibrance, a pill taken daily for three weeks followed by one week off. According to Celcuity, the drug should be launched by October. ― Dr. Sara Hurvitz, breast cancer oncologist and senior vice president and director of Fred Hutch’s Clinical Research Division “Big, big change, big benefit,” Hurvitz said with regard to what this would mean for patients with metastatic breast cancer, which is treatable but still not curable. “For patients with ER+/HER2- locally advanced or metastatic breast cancer, there is an urgent need for new treatment options that can meaningfully increase the likelihood of survival without disease progression or death,” Hurvitz said in the Celcuity news release. “With the approval of Revtorpyk, oncologists now have an effective new treatment option for these patients.” Hurvitz went on to praise the altruistic patients who participated in VIKTORIA-1 and other clinical trials designed to create more effective, less-toxic therapies. “It’s a time for tremendous hope with the amount of progress we’re making in breast oncology,” she said. “And the discoveries we’re making are in partnership with our patients, who are our colleagues. Without their willingness to volunteer and be part of a clinical trial, none of the progress made in the last five, 10, 20 years would have been made.” “Clinical participation can sometimes help the clinical trial participant, but not always,” she said. “But it does allow science to move forward. It’s just a wonderful example of altruism.”

Clinical ResultDrug ApprovalASCOPhase 1

15 Jul 2026

·www.biospace.com

Celcuity Announces FDA Approval of REVTORPYK™ (gedatolisib) for the Treatment of HR+/HER2-, PIK3CA Wild-Type Locally Advanced or Metastatic Breast Cancer

REVTORPYK (gedatolisib) REVTORPYK (gedatolisib) vial and box REVTORPYK is the first and only FDA-approved therapy that inhibits all class I PI3K isoforms (α, β, δ, γ) and mTOR complexes mTORC1 and mTORC2 In the Phase 3 VIKTORIA-1 trial, REVTORPYK combined with palbociclib and fulvestrant and REVTORPYK combined with fulvestrant reduced the risk of disease progression or death by 76% and 67%, respectively, compared to fulvestrant among patients with PIK3CA wild-type advanced or metastatic breast cancer Celcuity to host webcast and conference call today Tuesday, July 14, 2026, at 5:30 p.m. EDT MINNEAPOLIS, July 14, 2026 (GLOBE NEWSWIRE) -- Celcuity Inc. (Nasdaq: CELC), a biotechnology company focused on developing and commercializing targeted therapies for multiple solid tumor indications, today announced that the U.S. Food and Drug Administration (“FDA”) approved REVTORPYK™ (gedatolisib) for the treatment of patients with hormone receptor positive (“HR+”), human epidermal growth factor receptor 2 negative (“HER2-”), locally advanced or metastatic breast cancer without a PIK3CA mutation detected following progression on or after treatment with at least one line of endocrine therapy in the metastatic setting. REVTORPYK is the only inhibitor of class I PI3K isoforms (α, β, δ, γ) and mTOR complexes mTORC1 and mTORC2 to receive FDA approval. “The PI3K/AKT/mTOR, or PAM, pathway is one of the most important targets in cancer, but comprehensively inhibiting it has stymied researchers and drug developers for nearly two decades,” said Brian Sullivan, CEO and co-founder of Celcuity. “REVTORPYK addressed this 20-year challenge by becoming the first pan-PI3K, mTORC1/2 inhibitor approved by the FDA. We are thankful for the opportunity to make this important new therapy available to patients with HR+/HER2- locally advanced or metastatic breast cancer.” HR+/HER2- breast cancer is the most common subtype of breast cancer, accounting for approximately 70% of all breast cancers. 1 Among this breast cancer subtype, approximately 60% have PIK3CA wild-type disease. 2 “For patients with HR+/HER2- locally advanced or metastatic breast cancer, there is an urgent need for new treatment options that can meaningfully increase the likelihood of survival without disease progression or death,” said Sara Hurvitz, MD, Senior Vice President, Clinical Research Division, Fred Hutchinson Cancer Center, Smith Family Endowed Chair in Women’s Health and Professor and Head, Division of Hematology and Oncology, University of Washington, School of Medicine and co-principal investigator for the VIKTORIA-1 trial. “With the approval of REVTORPYK, oncologists now have an effective new treatment option for these patients.” The approval of REVTORPYK is based on positive clinical results from the PIK3CA wild-type cohort of the Phase 3 VIKTORIA-1 trial, an open-label, global, randomized clinical trial evaluating the efficacy and safety of REVTORPYK plus fulvestrant, with or without palbociclib, for the treatment of patients with locally advanced or metastatic HR+/HER2- breast cancer following progression on or after CDK4/6 therapy and an aromatase inhibitor. In the VIKTORIA-1 trial, median progression free survival (“PFS”) with the REVTORPYK triplet (REVTORPYK plus palbociclib and fulvestrant) 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 REVTORPYK triplet was 32% compared to 1% with fulvestrant and the median duration of response (“DOR”) was 17.5 months. For the REVTORPYK doublet (REVTORPYK plus fulvestrant), 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 REVTORPYK doublet was 28% and the median DOR was 12.0 months. The median DOR was not determinable for fulvestrant because there was only one objective response. “Today’s approval of REVTORPYK addresses a significant unmet need for the thousands of patients affected each year by HR+/HER2-, PIK3CA wild-type locally advanced or metastatic breast cancer whose disease has progressed after endocrine therapy,” said Igor Gorbatchevsky, MD, Chief Medical Officer of Celcuity. “We are deeply grateful to the patients and their caregivers, investigators and clinical study teams, and Celcuity team members who made this advancement possible.” Celcuity anticipates commercial launch in late Q3 2026. Based on the company’s commitment to ensuring broad, affordable, and unrestricted patient access to REVTORPYK, we have designed a comprehensive patient support program. To make REVTORPYK available to patients prior to commercial launch, those who are eligible will be able to enroll in Celcuity’s expanded access program. Celcuity plans to submit in Q3 2026 a supplemental New Drug Application (“sNDA”) to the FDA for REVTORPYK for the treatment of HR+/HER2-, PIK3CA mutated, locally advanced or metastatic breast cancer, following at least one line of endocrine therapy based on results from the mutant cohort of the Phase 3 VIKTORIA-1 trial. Results for this study were recently presented in a late-breaking abstract oral session at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting. Following the sNDA submission, Celcuity intends to submit VIKTORIA-1 data for marketing authorization of gedatolisib to other regulatory authorities around the world. REVTORPYK is also being studied in the ongoing Phase 3 VIKTORIA-2 clinical trial incorporating two independent studies evaluating two separate patient cohorts with HR+/HER2- locally advanced or metastatic breast cancer who are treatment-naive in the advanced setting. Webcast and Conference Call Information The Celcuity management team will host a live webcast and conference call on Tuesday, July 14, 2026, at 5:30 p.m. EDT / 4:30 p.m. CDT to discuss the FDA approval of REVTORPYK. 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. About REVTORPYK (gedatolisib) REVTORPYK is a kinase inhibitor of class I PI3K isoforms (α, β, δ, γ) and mTOR complexes mTORC1 and mTORC2, resulting in downstream inhibition of multiple effectors, including AKT. 3.4.5 REVTORPYK is in development for the first-line treatment of HR+/HER2- locally advanced or metastatic breast cancer and for the second-line treatment of metastatic castration resistant prostate cancer. Indication Statement REVTORPYK (gedatolisib) is a kinase inhibitor indicated in combination with fulvestrant, with or without palbociclib, for the treatment of adult patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative locally advanced or metastatic breast cancer without a PIK3CA mutation detected following progression on or after treatment with at least one line of endocrine therapy in the metastatic setting. IMPORTANT SAFETY INFORMATION WARNINGS AND PRECAUTIONS Stomatitis: REVTORPYK can cause severe stomatitis, including ulcers and oral mucositis. Stomatitis occurred in 72% of patients treated with REVTORPYK with fulvestrant and palbociclib, including Grade 3 events in 22% of patients. Stomatitis occurred in 58% of patients treated with REVTORPYK with fulvestrant, including Grade 3 events in 12% of patients. Initiate a steroid-containing, alcohol-free mouthwash prior to starting treatment with REVTORPYK and continue prophylactically during treatment. Monitor patients for signs and symptoms of stomatitis. Withhold, reduce dose, or permanently discontinue REVTORPYK based on severity. Dermatologic Adverse Reactions: REVTORPYK can cause severe rash. Rash occurred in 30% of patients treated with REVTORPYK in combination with fulvestrant and palbociclib, including Grade 3 events in 6% of patients. Rash occurred in 40% of patients treated with REVTORPYK with fulvestrant, including 5% of patients with Grade 3 events. Monitor patients for rash and infectious sequelae. Instruct patients to limit sun exposure during REVTORPYK treatment. Withhold, reduce dose, or permanently discontinue REVTORPYK based on severity. Hyperglycemia: REVTORPYK can cause severe hyperglycemia. Monitor fasting glucose prior to initiating treatment with REVTORPYK and periodically during treatment. Monitor HbA1c level if clinically indicated. Increased fasting glucose occurred in 46% of patients receiving REVTORPYK in combination with fulvestrant and palbociclib (Grade 3: 0.9%) and in 57% of patients receiving REVTORPYK in combination with fulvestrant (Grade 3: 1.8%). The safety of REVTORPYK has not been established in patients with Type 1 or uncontrolled Type 2 diabetes mellitus. Patients with well-controlled Type 2 diabetes may require intensified antihyperglycemic therapy and close monitoring of fasting glucose. Manage hyperglycemia with antihyperglycemic medications as clinically indicated. Evaluate fastingblood glucose and HbA1c levels prior to starting and at regular intervals during treatment. Withhold, reduce dose, or permanently discontinue REVTORPYK based on severity. Embryo-Fetal Toxicity: Based on its mechanism of action, REVTORPYK can cause fetal harm when administered to a pregnant woman. Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with REVTORPYK and for 2 weeks after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment and for 2 weeks after the last dose. Advise women not to breastfeed during treatment with REVTORPYK and for 2 weeks after the last dose. When REVTORPYK is used in combination, advise patients to use effective contraception during treatment and for the longest post-treatment duration recommended in the Prescribing Information of any of the individual products. Verify the pregnancy status of females of reproductive potential prior to initiating treatment. ADVERSE REACTIONS REVTORPYK in Combination with Fulvestrant and Palbociclib: The most common (≥20%) adverse reactions, including laboratory abnormalities when given in combination with fulvestrant and palbociclib were decreased white blood cells, decreased neutrophils, decreased hemoglobin, decreased lymphocytes, stomatitis, nausea, decreased platelets, increased fasting glucose, fatigue, vomiting, rash, constipation, diarrhea, increased alanine aminotransferase (ALT), increased aspartate aminotransferase (AST), musculoskeletal pain, decreased sodium, and increased eosinophils. REVTORPYK in Combination with Fulvestrant: The most common (≥20%) adverse reactions, including laboratory abnormalities when given in combination with fulvestrant were stomatitis, glucose increased, eosinophils increased, hemoglobin decreased, nausea, rash, ALT increased, fatigue, musculoskeletal pain, lymphocytes decreased, vomiting, AST increased, pruritus, and diarrhea. USE IN SPECIFIC POPULATION Lactation: Advise women not to breastfeed during treatment with REVTORPYK and for 2 weeks after the last dose. When used in combination, advise patients not to breastfeed during treatment and for the longest post-treatment duration recommended in the Prescribing Information of any of the individual products. Infertility: Advise females and males of reproductive potential that REVTORPYK may impair fertility. Please see full Prescribing Information , including Patient Information , for REVTORPYK. You may report side effects related to Celcuity products to Celcuity Medical Information at 1-877-4-CELCUITY (1-877-423-5284) or to FDA at 1-800-FDA-1088 or . © 2026 Celcuity Inc. All rights reserved. REVTORPYK and its logo are trademarks of Celcuity, Inc. US-G1B-26-023407/26 About Celcuity Celcuity is a biotechnology company focused on developing and commercializing targeted therapies for the treatment of multiple solid tumor indications. The company's first FDA-approved product is REVTORPYK (gedatolisib), a potent, pan-PI3K and mTORC1/2 inhibitor that comprehensively blockades the PAM pathway. Its mechanism of action and pharmacokinetic properties are differentiated from other currently approved and investigational therapies that target PI3Kα, AKT or mTORC1 alone or together. A Phase 3 clinical trial, VIKTORIA-1, evaluating gedatolisib in combination with fulvestrant with or without palbociclib in patients with HR+/HER2- locally advanced or metastatic breast cancer (“ABC”), supported FDA approval of REVTORPYK for use in patients without a PIK3CA mutation detected. Results for the PIK3CA mutant cohort of VIKTORIA-1 have been released. VIKTORIA-2 is an ongoing Phase 3 clinical trial incorporating two independent studies, Study 1 and Study 2, in two separate cohorts of patients with ABC who are treatment-naive in the advanced setting. Study 1 is evaluating gedatolisib plus palbociclib plus fulvestrant as first-line treatment for patients with endocrine-resistant HR+/HER2- ABC. Study 2 is evaluating gedatolisib plus palbociclib plus letrozole as first-line treatment for patients with endocrine- sensitive HR+/HER2- ABC. A Phase 1/2 clinical trial, CELC-G-201, evaluating gedatolisib in combination with darolutamide in patients with metastatic castration-resistant prostate cancer, is ongoing. More detailed information about Celcuity’s active clinical trials can be found at ClinicalTrials.gov . Celcuity is headquartered in Minneapolis. Further information about Celcuity can be found at . Follow us on LinkedIn and X . Forward Looking Statements This press release contains statements that constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements relating to the potential therapeutic benefits of gedatolisib; the size, design and timing of the Company’s clinical trials; the Company’s interpretation of clinical trial data; the status and timing of the submission, and the FDA’s review, of the Company’s sNDA for gedatolisib, and for making comparable filings with other regulatory authorities outside the U.S.; the market opportunity for gedatolisib; the Company’s expectations regarding the timing of and its ability to commercialize gedatolisib; the Company’s strategy, marketing and commercialization plans, including the benefits of strategic decisions regarding studies and trials; other expectations with respect to gedatolisib, including subcutaneous formulations to support potential future indications for gedatolisib regimens; the Company’s anticipated use of cash; and the strength of its balance sheet. Words such as, but not limited to, “look forward to,” “believe,” “expect,” “anticipate,” “estimate,” “intend,” "confidence," "encouraged," “potential,” “plan,” “targets,” “likely,” “may,” “will,” “would,” “should” and “could,” and similar expressions or words identify forward-looking statements. The forward-looking statements included in this press release are based on management's current expectations and beliefs which are subject to a number of risks, uncertainties and factors, including that the Company’s topline clinical results are based on an ongoing analysis of efficacy and safety data and such data may change following a more comprehensive review of the data related to the clinical trial; unforeseen delays in the Company’s clinical trials or the submission, and FDA’s review of, its sNDA for gedatolisib; the Company’s ability to obtain regulatory approval of its sNDA and maintain regulatory approvals to commercialize gedatolisib, and the market acceptance of gedatolisib; the development of therapies and tools competitive with gedatolisib; and the Company’s ability to access capital upon favorable terms. In addition, all forward-looking statements are subject to other risks detailed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025, as such risks may be updated in its subsequent filings with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by these cautionary statements, and the Company undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof. References: National Cancer Institute. Surveillance, Epidemiology and End Results Program (Accessed July 2025). Anderson, E. et al. A Systematic Review of the Prevalence and Diagnostic Workup of PIK3CA Mutations in HR+/HER2– Metastatic Breast Cancer, Int J Breast Cancer. 2020 Jun 20;2020:3759179. Venkatesan, A. M., et al. Bis(morpholino-1,3,5-triazine) derivatives: potent adenosine 5'-triphosphate competitive phosphatidylinositol-3-kinase/mammalian target of rapamycin inhibitors: discovery of compound 26 (PKI-587), a highly efficacious dual inhibitor. J Med Chem, 2010;53(6), 2636-2645. Mallon, R., et al. Antitumor efficacy of PKI-587, a highly potent dual PI3K/mTOR kinase inhibitor. Clin Cancer Res, 2011;17(10), 3193-3203. Rossetti, S., et al. Gedatolisib shows superior potency and efficacy versus single-node PI3K/AKT/mTOR inhibitors in breast cancer models. NPJ Breast Cancer, 2024;10(1), 40. Contacts: For Investors: Brian Sullivan, bsullivan@celcuity.com Vicky Hahne, vhahne@celcuity.com (763) 392-0123 Jodi Sievers, jsievers@celcuity.com (415) 494-9924 For Media: Sam Brown LLC Laura Morgan, lauramorgan@sambrown.com (951) 333-9110 A photo accompanying this announcement is available at

15 Jul 2026

·www.biopharmadive.com

Celcuity gains FDA approval for closely watched breast cancer drug

Celcuity has scored its first regulatory approval, winning on Tuesday a U.S. clearance for a closely watched breast cancer treatment itll sell as Revtorpyk.The Food and Drug Administration on Tuesday cleared Revtorpykfor use in two- and three-drug combinations involving hormone therapy and Pfizers Ibrance in certain people with a common form of advanced breast cancer. Specifically, Revtorpyk is available for use in the second-line setting among people with ER-positive, HER2-negative breast cancer, but dont have a so-called PIK3CA mutation. Celcuity expects to begin selling the therapy late in the third quarter.Revtorpyks clearance was based upon data from part of a study called VIKTORIA-1. In that trial, a regimen of Revtorpyk, the hormone treatment fulvestrant and Ibrance held tumors in check for a median of 9.3 months, compared to 2 months for those receiving fulvestrant alone. A doublet of Revtorpyk and Ibrance delayed tumor progression for a median of 7.3 months.In a note to clients Tuesday, Jefferies analyst Maury Raycroft wrote that Revtorpyks approval gives Celcuity a differentiated foothold in a setting where competition has largely centered around patients with PIK3CA mutations.Celcuity has also noted how Revtorpyk was associated with lower patient discontinuation ratesdue to treatment-related adverse events than what was observed in testing of other approved drug combinations for this patient population.However, company shares fell by nearly 20% on Wednesday following the news and a subsequent conference call with analysts. The drugs expected launch timing surprised investors given prior commentary on launch readiness, wrote Leerink Partners analyst Andrew Berens.Revtorpyks prescribing information also included higher discontinuation rates than expected, as the numbers were different than what the company disclosed at a medical meeting last year. In speaking with experts, Berens noted how real-world experience will be crucial to shaping clinician and patient perception of Revtorpyk.Still, others rallied to Celcuitys defense. In a Tuesday research note, Citizens JMP Securities analyst Silvan Turkcan pointed out that Revtorpyk was approved a few days ahead of schedule, a “positive indication“ for its clinical results. Those findings, combined with what's currently a “fragmented“ treatment landscape, leave the company with a “strong opportunity,“ Turkcan wrote.Raycroft, meanwhile, wrote that Celcuitys strong data, manageable safety compared to other approved treatments and more recent success in patients with PIK3CA mutations support a credible path to bigger sales.The company plans to submit a supplemental application in the third quarter to expand use.Celcuity hasnt yet revealed Revtorpyks cost, but said it will price the treatment at a premium compared to current therapies.Editors note: This story has been updated with additional analyst commentary. '

Drug ApprovalClinical Result

100 Deals associated with Gedatolisib

External Link

KEGG	Wiki	ATC	Drug Bank
D10635	Gedatolisib	-	DB11896

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hormone receptor positive HER2 negative breast cancer	United States	Celcuity, Inc.	14 Jul 2026

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Metastatic castration-resistant prostate cancer	Phase 2	United States	Celcuity, Inc.	01 Jan 2024
Metastatic castration-resistant prostate cancer	Phase 2	France	Celcuity, Inc.	01 Jan 2024
Metastatic castration-resistant prostate cancer	Phase 2	Spain	Celcuity, Inc.	01 Jan 2024
Metastatic castration-resistant prostate cancer	Phase 2	United Kingdom	Celcuity, Inc.	01 Jan 2024
Advanced Triple-Negative Breast Carcinoma	Phase 2	United States	Pfizer Inc.	17 Apr 2019
HER2-negative breast cancer	Phase 2	United States	Pfizer Inc.	17 Apr 2019
Triple Negative Breast Cancer	Phase 2	United States	Pfizer Inc.	17 Apr 2019
Breast cancer recurrent	Phase 2	United States	Pfizer Inc.	26 Feb 2019
Early Stage Breast Carcinoma	Phase 2	United States	Pfizer Inc.	26 Feb 2019
metastatic non-small cell lung cancer	Phase 2	United States	Pfizer Inc.	25 Sep 2017

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05501886 (VIKTORIA-1, Pubmed \| J Clin Oncol)	Phase 3	PIK3CA mutation/HR-positive/HER2-negative Breast Cancer hormone receptor-positive \| HER2− \| PIK3CA wild-type	392	Gedatolisib + Palbociclib + Fulvestrant	zmqlbrnwic(gvysydybvo) = pfqrfuugne qlnbvabemd (dbwdaasoeo ) View more	Positive	20 Apr 2026
				Gedatolisib + Fulvestrant	zmqlbrnwic(gvysydybvo) = xwfjohtitd qlnbvabemd (dbwdaasoeo ) View more
NCT05501886 (VIKTORIA-1, J Clin Oncol \| Biospace) Manual	Phase 3	PIK3CA mutation/HR-positive/HER2-negative Breast Cancer Hormone Receptor–Positive \| HER2− \| PIK3CA Wild-Type	392	gedatolisib+palbociclib+fulvestrant	vyvommheol(yeyxgglnjv) = eqgdwkyqnk awykzbvmjn (yzptxstxvr ) Met View more	Positive	09 Mar 2026
				gedatolisib+fulvestrant	vyvommheol(yeyxgglnjv) = lzsaeqvhsd awykzbvmjn (yzptxstxvr ) Met View more
NCT06190899 (ASCO_GU2026)	Phase 1/2	Metastatic castration-resistant prostate cancer	56	Gedatolisib 120 mg + Darolutamide	uuooqevvvg(yclsowkhua) = lsowyxayde vlqbcdmmmq (zpxmrfgrdm ) View more	Positive	26 Feb 2026
				Gedatolisib 180 mg + Darolutamide	sjnqfndgxl(dwwemystph) = hvkfgosuef rtohfjvgdh (zelpldlojy ) View more
VIKTORIA-1 (ESMO2025)	Phase 3	Advanced breast cancer Second line HR+ \| HER2- \| PIK3CA wild-type	392	Gedatolisib + Palbociclib + Fulvestrant	rybkfhcduy(ycpfjfvkpt) = eqnfugwlxa sporwunhuh (gjwrdefrzb ) View more	Positive	17 Oct 2025
				Gedatolisib + Fulvestrant	rybkfhcduy(ycpfjfvkpt) = eambfzxsyk sporwunhuh (gjwrdefrzb ) View more
Pubmed \| Clin Cancer Res Manual	Phase 1	Hormone receptor positive HER2 negative breast cancer First line HR+/HER2−	41	Gedatolisib + Palbociclib + Letrozole	xwtgyzmhfm(pybtddojrb) = uyxzyyvdqg pilloutakc (pbemygulbc ) View more	Positive	20 Aug 2025
NCT05501886 (VIKTORIA-1, PipelineReview \| NEWS) Manual	Phase 3	Hormone receptor positive HER2 negative breast cancer HER2 Negative \| HR Positive \| PIK3CA Gene Mutation Negative	-	Gedatolisib Gedatolisib + palbociclib + fulvestrant	pvwjgweljl(jblwvhmqrv) = ztabzmnyzb tqoykiqdiq (qevggykeyz ) View more	Positive	28 Jul 2025
				Gedatolisib Gedatolisib + fulvestrant	pvwjgweljl(jblwvhmqrv) = pkwywhmdst tqoykiqdiq (qevggykeyz )
NCT03911973 (Pubmed \| Breast Cancer Res Treat) Manual	Phase 1/2	Advanced Triple-Negative Breast Carcinoma \| BRCA mutation positive Breast Cancer HER2 Negative \| ER Negative \| PR Negative ... View more	-	Gedatolisib + Talazoparib	rdzxvjdmih(tbmvkjgbli) = ohswuwrekm syayyxccke (ekcazmwljk ) View more	Negative	05 Jun 2025
NCT06190899 (CELC-G-201, GlobeNewswire \| Company_Website)	Phase 1	Castration-Resistant Prostatic Cancer \| Metastatic castration-resistant prostate cancer Second line \| Third line --	38	Gedatolisib+Darolutamide	ahdwqbiiwi(qpqdpxumsd) = uawlxecfsv nsrsurbocw (xfzuwuytmc )	Positive	01 Jun 2025
NCT06757634 (VIKTORIA-2, AACR2025)	Phase 3	Hormone receptor positive HER2 negative breast cancer First line PIK3CA	-	Gedatolisib + CDK4/6 inhibitor + Fulvestrant	vmrcdvryfs(casoghpbdb) = expected mPFS is 25 to 28 months xzbcdscpcd (dtlmzhsugs ) View more	Positive	29 Apr 2025
NCT02684032 (Pubmed)	Phase 1	Hormone receptor positive HER2 negative breast cancer Second line hormone receptor-positive \| HER2-negative	103	Gedatolisib 180 mg	grtzakwary(idnofstvxr) = Grade 3-4 hyperglycaemia was reported in six (6%) participants meydrpawte (senqefrrxb ) View more	-	01 Apr 2024

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