Celcuity CEO Details Gedatolisib NDA, July PDUFA and Launch Plans at TD Cowen Conference

Key Points

• Celcuity’s NDA for gedatolisib has been accepted with a PDUFA date in July and Real‑Time Oncology Review plus Breakthrough Therapy designations, and management expects an approval decision around the PDUFA date while planning a potential supplemental NDA for mutant‑cohort data to seek expedited review again.
• The company has been building commercial infrastructure for roughly two years, expects to onboard sales reps in Q2, and will initially target patients with PIK3CA wild‑type disease (≈60% of patients), estimating ~37,000 second‑line eligible patients and a potential >$10 billion peak breast‑cancer opportunity if expanded to first‑line use.
• Phase 3 and additional cohort readouts are imminent with a mutant‑cohort statistical success benchmark of about a 10‑month median PFS, and gedatolisib shows a favorable tolerability profile with less than 10% overall hyperglycemia (2% Grade 3) versus ~80% for alpelisib, though stomatitis is higher but reportedly manageable.

Celcuity NASDAQ: CELC CEO and co-founder Brian Sullivan outlined the company’s clinical programs, regulatory progress, and commercial launch preparations for gedatolisib during a fireside chat at TD Cowen’s 46th Annual Healthcare Conference. Sullivan described Celcuity as a clinical-stage oncology company focused on therapies that target the PI3K/AKT/mTOR (PAM) pathway, which he called “one of the most important pathways in oncology.”

Clinical and regulatory update

Sullivan said Celcuity currently has three trials underway. The company’s lead program is a Phase 3 trial evaluating gedatolisib in combination with palbociclib and fulvestrant in women who have progressed after prior CDK inhibitor therapy. He also highlighted a first-line study using the same combination in treatment-naïve women with endocrine-resistant metastatic breast cancer. A third, earlier-stage study is evaluating gedatolisib in combination with an androgen receptor inhibitor in men with castration-resistant prostate cancer.

On the regulatory front, Sullivan discussed the company’s NDA being accepted with a PDUFA date in July, and the FDA’s Real-Time Oncology Review (RTOR) designation. He said RTOR is generally used for drugs the FDA believes have the potential to change the standard of care and noted that Celcuity already had Breakthrough Therapy designation based on earlier-phase results. He explained that RTOR allows the company to submit portions of the application ahead of the formal final submission, and said Celcuity began providing datasets to the FDA within a month of having the data and continued on a rolling basis.

Sullivan said Celcuity has a priority review and expects an approval decision “along the line of at the PDUFA date,” adding that RTOR’s timeline advantages tend to be more meaningful for supplemental NDAs. He pointed to upcoming “mutant data” the company expects to report “end of this quarter, early next, or sometime next quarter,” and said that if the results are positive, Celcuity would plan to file a supplemental NDA and seek RTOR again, potentially enabling a shorter approval timeline.

Asked about the broader FDA environment, Sullivan said he has not seen changes in the quality, timing, or type of interactions with the agency. He said Celcuity has been working with the same FDA group for about five years, described the relationship as collaborative, and stated he does not expect an anomalous outcome driven by external factors.

Commercial launch preparations

Sullivan said launch planning began roughly two years ago with the hiring of a chief commercial officer, followed by building out a senior commercial team and a “company-wide launch plan” that spans commercial infrastructure, IT systems, safety reporting readiness, HR, and finance. He said commercial hiring was largely completed by the end of last year except for the sales force, though regional sales managers were hired earlier. Celcuity has determined the size of the sales team and identified most of the candidates it expects to hire, with sales representatives expected to come onboard in the second quarter.

In discussing early launch dynamics, Sullivan described three “fairly discrete” segments in the market and said the initial focus would be on patients with PIK3CA wild-type status, which he estimated at roughly 60% of patients. He added that data for a mutant cohort is expected to be public prior to launch, and that positive results could provide momentum heading into commercialization.

Data expectations and market segmentation

Regarding timing for the mutant cohort readout, Sullivan said Celcuity has not changed its guidance and expressed confidence that the data will be available in the timeframe previously provided.

When discussing expectations for the control arm and comparative benchmarks, Sullivan cited external studies, including BYLieve and EPIK-B5, as informing assumptions about performance in similar populations. He also referenced reported results from capivasertib in a similar setting, including a median progression-free survival (PFS) of 5.5 months and a hazard ratio he characterized as roughly similar to alpelisib. Sullivan said Celcuity believes it is unlikely the control arm will overperform meaningfully relative to those historical benchmarks.

Sullivan said the statistical benchmark for success in Celcuity’s mutant analysis would be “roughly 10 months or so” median PFS for statistical significance, which he noted would be only modestly above what the company previously reported in the wild-type population. He said Celcuity views achieving that threshold as “highly probable,” and framed clinical significance in terms of comparing a potential 10-month outcome to capivasertib’s 5.5 months. He also referenced earlier Phase 1B results presented at ESMO in a similar patient population, noting that wild-type median PFS in that study was 9.1 months versus 9.3 months in the Phase 3 wild-type population, and that the mutant portion of the Phase 1B study showed 14.6 months. Sullivan said that earlier dataset supports optimism that the mutant Phase 3 result could exceed the 10-month level, while emphasizing that the Phase 3 outcome remains unknown until readout.

He also cautioned that early-phase PFS comparisons can be influenced by population mix, particularly the proportion of patients with bone-only disease versus measurable disease. Sullivan said bone-only patients can show materially better PFS outcomes in endocrine-therapy studies, referencing PALOMA-3 and noting Celcuity observed a similar pattern in a small internal sample.

Safety and quality-of-life discussion

Sullivan said one point that surprised clinicians positively was the low rate of hyperglycemia associated with gedatolisib, describing hyperglycemia as a common challenge with drugs in this pathway. He cited Celcuity’s reported hyperglycemia as less than 10% overall with 2% Grade 3, and contrasted that with alpelisib, which he said induces hyperglycemia in about 80% of patients.

He acknowledged stomatitis was higher than expected, but said it tends to appear early, improves within a couple of weeks, and that Grade 3 stomatitis is uncommon by the end of the second cycle across the overall population. Sullivan pointed to patient-reported outcomes presented at San Antonio, saying the company saw no degradation in a quality-of-life score over an eight-cycle follow-up period. He also cited a 2% discontinuation rate and said investigators have reported patients stating they “don’t feel like” they are on a cancer drug.

Sullivan attributed the tolerability profile in part to gedatolisib’s dosing schedule—three times a month—arguing that less frequent high-peak exposure differs from daily oral dosing that can contribute to chronic treatment burden. He said the company does not expect safety differences between mutant and wild-type groups.

Frontline opportunity and longer-term potential

On the first-line setting, Sullivan differentiated between early breast cancer adjuvant therapy and metastatic disease, arguing that adjuvant regimens would have limited downstream impact on advanced breast cancer for many years. He said approximately 60,000 women per year present with endocrine-sensitive metastatic disease, while another group of roughly 35,000 present with endocrine-resistant metastatic disease—patients who progress during adjuvant endocrine therapy or within 12 months. He said Celcuity’s ongoing VIKTORIA-2 study is focused on the endocrine-resistant first-line group, noting that current standard-of-care CDK4/6 plus fulvestrant yields about seven months median PFS in that population.

Sullivan also referenced an early-phase dataset in endocrine-sensitive patients, citing a reported 48-month median PFS and an 80% response rate in a 41-patient single-arm study, compared with around 25 months median PFS for standard CDK4/6 plus letrozole. He said the findings support the idea that the PAM pathway is intrinsically relevant in the disease.

In response to a question about what may be underappreciated about Celcuity, Sullivan pointed to the potential size of the eligible population and said the company estimates about 37,000 patients could be eligible in the second-line setting. He added that, assuming successful progression into first-line use, the company sees “$10 billion plus” in potential peak revenue from breast cancer alone, characterizing the opportunity as driven by targeting one of what he called the “three critical disease drivers.”

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.

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