Celcuity (NASDAQ:CELC) Chief Executive Officer Brian Sullivan outlined the company’s clinical and commercial priorities for its lead asset, gedatolisib, during a presentation at Leerink Partners’ Global Healthcare Conference. Sullivan said the company originally began as a platform effort focused on isolating and quantifying the activity of live patient tumor cells to help identify patients most likely to respond to targeted therapies. That work led Celcuity into the PI3K pathway, where the company acquired and began developing gedatolisib, a pan-PI3K/mTOR inhibitor, in 2021.
Focus on PI3K/mTOR inhibition in HR-positive breast cancer
Sullivan framed gedatolisib’s development strategy around what he described as the central importance of the PI3K/mTOR (“PAM”) pathway in hormone receptor (HR)-positive breast cancer, including in patients without PIK3CA mutations. He said the company believes the pathway has been “suboptimally targeted to date,” with prior approaches delivering modest efficacy in subsets of patients and facing safety constraints.
Second-line results highlighted in wild-type population
Sullivan reviewed Celcuity’s previously disclosed phase III results in the second-line setting in patients he characterized as “wild types” (lacking PIK3CA mutations). He said the company reported two primary endpoints comparing:
- Gedatolisib + palbociclib + fulvestrant versus fulvestrant
- Gedatolisib + fulvestrant (doublet) versus fulvestrant
For the triplet regimen versus fulvestrant, Sullivan said the company reported a median progression-free survival (PFS) of 9.3 months compared with 2 months, representing a 7.3-month difference. He also cited a hazard ratio of 0.24, which he described as unusually favorable versus historical breast cancer results. He added that the doublet regimen also showed “very significant activity,” and said that absent the triplet, the doublet would have been the best reported second-line results.
According to Sullivan, these findings support Celcuity’s view that comprehensive inhibition of the pathway can deliver meaningful benefit regardless of PIK3CA mutational status.
Upcoming mutant-cohort readout and expectations
Looking ahead, Sullivan said Celcuity expects to report data from the PIK3CA-mutant cohort “end of this quarter, sometime next quarter.” He pointed to early phase Ib data that he said showed a median PFS of about 14.5 months in a mutant cohort, and referenced an additional analysis he said showed a 19-month median PFS in a small sample of patients with mutations, while cautioning that the sample size was limited.
He also discussed the competitive context for statistical and clinical relevance, stating that for Celcuity’s study, 10 months median PFS would be statistically significant based on prior results for a control regimen he identified as alpelisib plus fulvestrant. Sullivan added that he views capivasertib plus fulvestrant as the “true standard of care today” in PIK3CA-mutant patients, and said that regimen reported roughly 5.5 months median PFS in the setting he was discussing. Even a modestly positive study could be clinically meaningful, he argued, given that comparison.
Frontline development strategy and endocrine-resistant subgroup
Sullivan also described an ongoing phase III trial in HR-positive patients who are treatment-naïve for metastatic disease but considered endocrine resistant due to early recurrence on or shortly after adjuvant endocrine therapy. He characterized this as a difficult population and said current standard-of-care outcomes are around 7 months median PFS. Celcuity has been conducting a safety run-in evaluating gedatolisib with ribociclib and expects to provide results and any study design updates “in the near term.”
He contrasted this with the endocrine-sensitive frontline population—patients who recur long after adjuvant therapy or present with de novo metastatic disease—where he said CDK4/6 plus letrozole can yield about 25 months median PFS, implying longer trials. Sullivan referenced earlier data in treatment-naïve patients from a prior study, citing 48 months median PFS and a 79% objective response rate, and said those results support potentially launching a study in that setting.
Commercial readiness, IV administration, and ex-U.S. plans
On commercialization, Sullivan said the company has largely built out its organization except for the sales force. He said teams in market access, marketing, commercial operations, and medical affairs are in place, and that sales leadership and regional management have been established, with the sales force expected to be hired beginning in the second quarter. He also said Celcuity is targeting an approval decision around a mid-July PDUFA date.
While the company has not disclosed planned sales force size, Sullivan said other breast cancer-focused launches often involve around 90 representatives. He argued that intravenous (IV) administration should not materially limit uptake because most breast cancer practices already use infusion centers and many key breast cancer drugs are IV. He also noted that infused drugs are typically reimbursed under the medical benefit, which he suggested can be less challenging than oral reimbursement pathways. Sullivan said commercial research has been favorable, indicating physician preference for gedatolisib compared with alternatives and limited impact from IV administration.
Beyond breast cancer, Sullivan discussed an early-phase prostate cancer study combining gedatolisib with darolutamide, an androgen receptor inhibitor owned by Bayer. He said early results at two dose levels were favorable, and that safety was “better than we expected,” adding that men appear to have a different safety profile than women receiving gedatolisib. The company plans to evaluate an additional dose and expects results by the end of the year.
Sullivan said Celcuity expects patent term exclusivity through 2042 based on a dosing patent tied to a three-weeks-on, one-week-off schedule, with other patents of shorter duration. He said a subcutaneous formulation is not viewed as necessary for second-line or endocrine-resistant populations, but could be relevant for endocrine-sensitive patients with potentially long treatment durations; however, he did not provide details on development status.
Internationally, Sullivan said Celcuity intends to launch in the U.S. directly and does not plan to market ex-U.S. itself. He said the company has had discussions with potential partners but has held off on advancing a deal until mutant-cohort data are available. He added that Celcuity is preparing groundwork for a European marketing authorization application (MAA) and has aligned with Japanese regulators on additional data requirements, with the aim of progressing approvals in major ex-U.S. markets while exploring partnering options.
About Celcuity (NASDAQ:CELC)
Celcuity, Inc is a clinical-stage biotechnology company specializing in precision oncology diagnostics. The company develops and commercializes predictive biomarker assays designed to identify which patients are most likely to benefit from targeted cancer therapies. By integrating functional profiling of tumor cells with molecular analyses, Celcuity seeks to optimize treatment selection and improve outcomes for patients with solid tumors.
Celcuity’s proprietary platform evaluates tumor cell sensitivity to various therapeutic agents using ex vivo assays that measure DNA damage response and other critical pathways.
