Celcuity to Initiate NDA Submission of Gedatolisib in PIK3CA Wild-Type Cohort in HR+/HER2- Advanced Breast Cancer Under FDA’s Real-Time Oncology Review Program
Celcuity (NASDAQ:CELC) announced that the FDA has agreed to review its New Drug Application (NDA) for gedatolisib in HR+/HER2- advanced breast cancer under the Real-Time Oncology Review program. The company plans to initiate a rolling submission in September 2025, with completion targeted for Q4 2025.
The submission is based on groundbreaking Phase 3 VIKTORIA-1 trial results where the gedatolisib-triplet therapy reduced disease progression risk by 76% compared to fulvestrant alone, with a median PFS of 9.3 vs 2.0 months. The gedatolisib-doublet showed a 67% risk reduction with median PFS of 7.4 vs 2.0 months. These results represent unprecedented improvements in HR+/HER2- advanced breast cancer treatment.
Celcuity (NASDAQ:CELC) ha annunciato che la FDA ha accettato di esaminare la sua Domanda di Nuovo Farmaco (NDA) per gedatolisib nel trattamento del carcinoma mammario avanzato HR+/HER2- tramite il programma Real‑Time Oncology Review. L'azienda prevede di avviare una presentazione scaglionata a settembre 2025, con completamento previsto nel quarto trimestre 2025.
La richiesta si basa sui risultati rivoluzionari dello studio di Fase 3 VIKTORIA‑1: la terapia tripla a base di gedatolisib ha ridotto il rischio di progressione della malattia del 76% rispetto al solo fulvestrant, con una PFS mediana di 9,3 mesi contro 2,0 mesi. La combinazione doppia con gedatolisib ha mostrato una riduzione del rischio del 67%, con una PFS mediana di 7,4 vs 2,0 mesi. Questi dati rappresentano miglioramenti senza precedenti nel trattamento del carcinoma mammario avanzato HR+/HER2-.
Celcuity (NASDAQ:CELC) anunció que la FDA aceptó revisar su Solicitud de Nuevo Fármaco (NDA) para gedatolisib en cáncer de mama avanzado HR+/HER2- bajo el programa Real‑Time Oncology Review. La compañía tiene previsto iniciar una presentación escalonada en septiembre de 2025, con finalización prevista para el cuarto trimestre de 2025.
La presentación se fundamenta en los resultados innovadores del ensayo de Fase 3 VIKTORIA‑1: la terapia triple con gedatolisib redujo el riesgo de progresión de la enfermedad en un 76% frente a fulvestrant solo, con una SSP media de 9,3 frente a 2,0 meses. La doblete con gedatolisib mostró una reducción del riesgo del 67%, con SSP media de 7,4 vs 2,0 meses. Estos resultados suponen mejoras sin precedentes en el tratamiento del cáncer de mama avanzado HR+/HER2-.
Celcuity (NASDAQ:CELC)는 FDA가 Real‑Time Oncology Review 프로그램을 통해 HR+/HER2- 진행성 유방암 치료제 gedatolisib의 신약허가신청(NDA)을 검토하기로 합의했다고 발표했습니다. 회사는 2025년 9월부터 롤링 제출을 시작해 2025년 4분기 내 완료할 계획입니다.
이번 제출은 3상 VIKTORIA‑1 연구의 획기적인 결과를 근거로 합니다. gedatolisib를 포함한 3제 요법은 질환 진행 위험을 76% 감소시켰으며, 무진행생존기간(PFS) 중앙값은 9.3개월 대 2.0개월이었습니다. gedatolisib 2제 요법은 67%의 위험 감소를 보였고, PFS 중앙값은 7.4대 2.0개월이었습니다. 이 결과는 HR+/HER2- 진행성 유방암 치료에서 전례 없는 개선을 의미합니다.
Celcuity (NASDAQ:CELC) a annoncé que la FDA a accepté d'examiner sa demande d'autorisation de mise sur le marché (NDA) pour le gedatolisib dans le cancer du sein avancé HR+/HER2- dans le cadre du programme Real‑Time Oncology Review. La société prévoit de lancer une soumission progressive en septembre 2025, avec un achèvement visé au T4 2025.
La soumission repose sur les résultats révolutionnaires de l'essai de phase 3 VIKTORIA‑1 : la thérapie triplette avec gedatolisib a réduit le risque de progression de la maladie de 76% par rapport au fulvestrant seul, avec une SSP médiane de 9,3 vs 2,0 mois. La doublette gedatolisib a montré une réduction du risque de 67%, avec une SSP médiane de 7,4 vs 2,0 mois. Ces résultats représentent des améliorations sans précédent dans le traitement du cancer du sein avancé HR+/HER2-.
Celcuity (NASDAQ:CELC) gab bekannt, dass die FDA zugestimmt hat, den Antrag auf Zulassung eines neuen Arzneimittels (NDA) für gedatolisib bei fortgeschrittenem HR+/HER2- Brustkrebs im Rahmen des Real‑Time Oncology Review‑Programms zu prüfen. Das Unternehmen plant, im September 2025 mit einer gestaffelten Einreichung zu beginnen und diese im 4. Quartal 2025 abzuschließen.
Die Einreichung stützt sich auf die bahnbrechenden Ergebnisse der Phase‑3‑Studie VIKTORIA‑1: Die gedatolisib‑Tripletttherapie verringerte das Risiko eines Krankheitsfortschreitens um 76% gegenüber Fulvestrant allein, mit einer medianen PFS von 9,3 vs. 2,0 Monaten. Das gedatolisib‑Duett zeigte eine Risikoreduzierung von 67% bei einer medianen PFS von 7,4 vs. 2,0 Monaten. Diese Befunde stellen beispiellose Verbesserungen in der Behandlung von fortgeschrittenem HR+/HER2- Brustkrebs dar.
- FDA agreed to review gedatolisib under expedited Real-Time Oncology Review program
- Gedatolisib-triplet showed unprecedented 76% reduction in disease progression risk
- 7.3 months improvement in median PFS with gedatolisib-triplet therapy
- Drug previously received both Breakthrough Therapy and Fast Track designations
- None.
Insights
Celcuity's gedatolisib NDA acceptance under FDA's accelerated review program represents a significant advancement for HR+/HER2- breast cancer treatment with unprecedented efficacy data.
Celcuity's announcement represents a significant regulatory milestone for gedatolisib in HR+/HER2- advanced breast cancer treatment. The FDA's acceptance of their NDA under the Real-Time Oncology Review (RTOR) program is particularly noteworthy as this pathway is reserved for drugs showing substantial improvements over existing therapies. This expedited review process could potentially bring gedatolisib to market sooner than through standard channels.
The clinical data supporting this application is remarkably strong. The Phase 3 VIKTORIA-1 trial results in the PIK3CA wild-type cohort demonstrated unprecedented efficacy:
- The gedatolisib-triplet (combined with fulvestrant and palbociclib) reduced disease progression or death risk by
76% (hazard ratio: 0.24) - Median progression-free survival (PFS) improved from 2.0 months with fulvestrant alone to 9.3 months with the triplet regimen
- Even the gedatolisib-doublet (with fulvestrant) showed impressive results with
67% risk reduction (hazard ratio: 0.33) and median PFS of 7.4 months
These hazard ratios represent some of the most substantial improvements ever seen in HR+/HER2- advanced breast cancer trials. For context, most successful trials in this space typically show hazard ratios in the 0.5-0.7 range, making gedatolisib's 0.24 truly exceptional.
The drug's earlier Breakthrough Therapy and Fast Track designations further underscore its potential significance. For patients with HR+/HER2- advanced breast cancer who have progressed after CDK4/6 inhibitor treatment, therapeutic options remain limited, highlighting the unmet medical need gedatolisib addresses.
With submission initiation in September and completion targeted for Q4 2025, Celcuity is positioning gedatolisib for potential approval in 2026, assuming standard FDA review timelines following a complete submission.
MINNEAPOLIS, Aug. 27, 2025 (GLOBE NEWSWIRE) -- Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company pursuing development of targeted therapies for oncology, today announced the U.S. Food and Drug Administration (“FDA”) agreed to accept its New Drug Application (“NDA”) for gedatolisib in HR+/HER2- advanced breast cancer (“ABC”) for review under the Real-Time Oncology Review (“RTOR”) program, which facilitates earlier submission of topline efficacy and safety results, prior to the submission of the complete application, to support an earlier start to the FDA’s evaluation of the application. Celcuity is expected to initiate in September a rolling submission to the FDA of its NDA for gedatolisib, based on topline 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.
“On the heels of announcing positive pivotal data last month, we are pleased that the FDA agreed to review our NDA application for gedatolisib under the RTOR program,” said Brian Sullivan, CEO and co-founder of Celcuity. “Gedatolisib previously received both Breakthrough Therapy and Fast Track designations based on our promising preliminary clinical data. The FDA’s decision further highlights the urgent need for more efficacious therapies than those currently available for patients with HR+, HER2- advanced breast cancer who have received prior treatment with a CDK4/6 inhibitor. We look forward to working with the FDA to complete the review of our NDA for gedatolisib.”
The NDA submission is based on the positive topline results for the PIK3CA wild-type cohort of the Phase 3 VIKTORIA-1 trial. 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
About RTOR
The FDA established the RTOR program to facilitate a more efficient review process for drugs to ensure that safe and effective treatments are available to patients as early as possible, while improving review quality and engaging in early iterative communication with the applicant. To be considered for RTOR, submissions should demonstrate the following: 1) clinical evidence from adequate and well-controlled investigation that indicates the drug may demonstrate substantial improvement on a clinically relevant endpoint over available therapies; 2) easily interpreted clinical trial endpoints; 3) no aspect of the submission is likely to require a longer review time. Additional information about RTOR can be found at: https://www.fda.gov/about-fda/oncology-center-excellence/real-time-oncology-review-pilot-program.
About Celcuity
Celcuity is a clinical-stage biotechnology company pursuing development of targeted therapies for treatment of multiple solid tumor indications. The company's lead therapeutic candidate is 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- ABC has completed enrollment of and reported topline data for the PIK3CA wild-type cohort, and is currently enrolling patients for the PIK3CA mutant cohort. 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. A Phase 3 clinical trial, VIKTORIA-2, evaluating gedatolisib plus a CDK4/6 inhibitor and fulvestrant as first-line treatment for patients with HR+/HER2- ABC is currently enrolling patients. 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 www.celcuity.com. 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 our clinical trials; our interpretation of topline clinical trial data; the ability of our data to support the filing of an NDA with the FDA; our expectations regarding the timing of and our ability to obtain FDA approval to commercialize gedatolisib under the RTOR pilot program; and other expectations with respect to gedatolisib. 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 our topline results are based on a preliminary analysis of key 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 our planned NDA for gedatolisib; and our ability to obtain and maintain regulatory approvals to commercialize gedatolisib. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2024, as such risks may be updated in our 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 we undertake no obligation to revise or update this press release to reflect events or circumstances after the date hereof.
View source version of release on GlobeNewswire.com
Contacts:
Celcuity Inc.
Brian Sullivan, bsullivan@celcuity.com
Vicky Hahne, vhahne@celcuity.com
(763) 392-0123
ICR Healthcare
Patti Bank, patti.bank@icrhealthcare.com
(415) 513-1284
FAQ
What are the key results of Celcuity's Phase 3 VIKTORIA-1 trial for gedatolisib?
When will Celcuity (CELC) submit its NDA for gedatolisib to the FDA?
What special FDA designations has Celcuity's gedatolisib received?
What type of breast cancer is Celcuity's gedatolisib targeting?
What is the significance of FDA's Real-Time Oncology Review for Celcuity's gedatolisib?
