On March 23, 2022 Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company pursuing an integrated therapeutic and companion diagnostic strategy for treating patients with cancer, reported financial results for the fourth quarter and full year ended December 31, 2021, the design for its pivotal Phase 3 trial for gedatolisib, an investigational pan-PI3K/mTOR inhibitor, and other recent business progress (Press release, Celcuity, MAR 23, 2022, View Source [SID1234610809]).

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The pivotal Phase 3 clinical trial, which is named VIKTORIA-1, will be an open-label, randomized study to evaluate the efficacy and safety of gedatolisib in combination with fulvestrant with or without palbociclib in adults with HR+/HER2- advanced breast cancer whose disease has progressed after prior CDK4/6 therapy in combination with an aromatase inhibitor. Prior to finalizing the Phase 3 trial design, Celcuity conducted two formal meetings with the U.S. Food and Drug Administration (FDA) to obtain their feedback.

The clinical trial will enroll subjects regardless of PIK3CA status while enabling separate evaluation of subjects according to their PIK3CA status. Subjects who meet eligibility criteria and do not have confirmed PI3KCA mutations (WT) will be randomly assigned (1:1:1) to receive a regimen of either gedatolisib, palbociclib, and fulvestrant (Arm A), gedatolisib and fulvestrant (Arm B), or fulvestrant (Arm C). Up to 351 subjects who are PIK3CA WT will be enrolled. Subjects who meet eligibility criteria and have confirmed PI3KCA mutations (MT) will be randomly assigned (1:1) to receive a regimen of either gedatolisib, palbociclib, and fulvestrant (Arm D) or alpelisib and fulvestrant (Arm E). Up to 300 subjects who are PIK3CA MT will be enrolled.

The primary endpoints for this pivotal study are progression free survival (PFS), per RECIST 1.1 criteria, as assessed by blinded independent central review (BICR). The primary PFS endpoints will be evaluated separately in subjects who are PI3KCA WT and PI3KCA MT.

This multi-center, international trial is expected to enroll up to 651 subjects at approximately 175 clinical sites across the U.S., Europe, and Asia. Celcuity expects to initiate the clinical trial in the first half of 2022.

"We are excited to finalize the trial design for our pivotal Phase 3 study following productive meetings with the FDA," said Brian Sullivan, CEO and co-founder of Celcuity. "This design, we believe, provides us with an opportunity to generate data that could support the future submission to the FDA of a New Drug Application for gedatolisib to treat a broad patient population."

"There is an urgent need for better treatment options for HR+/HER2- metastatic breast cancer patients whose disease has progressed after treatment with a CDK4/6 inhibitor and endocrine therapy," said Igor Gorbatchevsky, MD, Chief Medical Officer of Celcuity. "We are very encouraged by the clinical data for gedatolisib and determined to advance its development for patients as quickly as possible."

Fourth Quarter 2021 Business Highlights and Other Recent Developments

●Celcuity obtained formal feedback from the FDA regarding the design of its proposed Phase 3 clinical trial, and the trial design has been finalized.

●In January, the FDA granted Fast Track designation to Celcuity’s lead drug candidate, gedatolisib, for the treatment of patients with HR+/HER2- metastatic breast cancer after progression on CDK4/6 therapy.

●In November 2021, Celcuity and Pfizer entered into a Clinical Trial Collaboration and Supply Agreement. Under this agreement, Pfizer will supply Ibrance for Celcuity’s pivotal Phase 3 clinical trial for gedatolisib at no cost.

●Updated data from Celcuity’s ongoing Phase 1b clinical trial for patients with HR+/HER2- advanced breast cancer (ABC) was presented at the San Antonio Breast Cancer Symposium in December 2021. Consistent with the previously released results, the addition of gedatolisib to palbociclib and endocrine therapy demonstrated promising antitumor activity in both first and later line settings for HR+/HER2- ABC that compared favorably to published data for available therapeutic options. A significant objective response rate (ORR) was also observed in patients refractory to their last treatment or who had received two or more lines of prior treatment for advanced breast cancer. Gedatolisib was well tolerated with manageable toxicity and a low number of patients discontinued treatment due to an adverse event.

●Celcuity entered into a clinical trial collaboration agreement with the University of Rochester Wilmot Cancer Center and Puma Biotechnology in October 2021.

●Enrollment in the FACT-1 and FACT-2 trials evaluating CELsignia selected patients who have early-stage HR+/HER2- breast cancer was impacted by COVID-19 related delays during the fourth quarter and early 2022. Hospitalizations of patients with COVID-19 increased dramatically during this period which led hospitals to reduce clinical trial related activities. Interim results are now expected to be available in the first half of 2023.

Fourth Quarter and Full Year 2021 Financial Results

Unless otherwise stated, all comparisons are for the fourth quarter and full year ended December 31, 2021, compared to the fourth quarter and full year ended December 31, 2020.

Total operating expenses were $6.3 million for the fourth quarter of 2021, compared to $2.6 million for the fourth quarter of 2020. Operating expenses for the full year 2021 were $28.4 million, compared to $9.6 million for the full year 2020.

Research and development (R&D) expenses were $5.5 million for the fourth quarter of 2021, compared to $2.1 million for the fourth quarter of 2020. R&D expenses for the full year 2021 were $25.8 million, compared to $7.7 million for the prior year. The approximately $18.1 million increase during the full year 2021, compared to the full year 2020, resulted primarily from a $10.0 million upfront license fee related to the execution of the Pfizer license agreement, which included $5.0 million of non-cash expense for issuance of common stock. The remaining $8.1 million increase primarily resulted from expenses initially associated with the transfer of gedatolisib-related activities from Pfizer to Celcuity and subsequently the continued support and development of gedatolisib.

General and administrative (G&A) expenses were $0.8 million for the fourth quarter of 2021, compared to $0.4 million for the fourth quarter of 2020. G&A expenses for the full year 2021 were $2.6 million, compared to $1.9 million for the prior year. The approximately $0.7 million increase during the full year 2021, compared to the full year 2020, resulted primarily from a $0.4 million increase in compensation related expenses, including approximately $0.3 million of non-cash stock-based compensation.

Net loss for the fourth quarter of 2021 was $6.8 million, or $0.45 per share, compared to a net loss of $2.6 million, or $0.25 per share for 2020. Net loss for the full year 2021 was $29.6 million, or $2.21 per share, compared to $9.5 million, or $0.92 per share in 2020. Non-GAAP adjusted net loss for the fourth quarter of 2021 was $5.6 million, or $0.37 per share, compared to non-GAAP adjusted net loss of $2.1 million, or $0.21 per share, for the fourth quarter of 2020. Non-GAAP adjusted net loss for the full year 2021 was $21.4 million, or $1.60 per share, compared to non-GAAP adjusted net loss of $7.7 million, or $0.75 per share, for 2020. Non-GAAP adjusted net loss excludes stock-based compensation expense, issuance of common stock and non-cash interest. Because these items have no impact on Celcuity’s cash position, management believes non-GAAP adjusted net loss better enables Celcuity to focus on cash used in operations. For a reconciliation of financial measures calculated in accordance with generally accepted accounting principles (GAAP) in the United States to non-GAAP financial measures, please see the financial tables at the end of this news release.

Net cash used in operating activities for the fourth quarter of 2021 was $6.1 million, compared to $2.1 million for the fourth quarter of 2020.

At December 31, 2021, Celcuity had cash and cash equivalents of $84.3 million, compared to cash and cash equivalents of $11.6 million at December 31, 2020.

Anticipated Milestones

Celcuity expects to achieve the following potential milestones over the next twelve months

●Provide an update on lifecycle development priorities for gedatolisib in the first half of 2022.
●Initiate two Phase 2 clinical trials to evaluate gedatolisib in HR+/HER2- breast cancer patients selected with a CELsignia PI3K Pathway Test.
●Obtain interim results from the FACT-1 and FACT-2 trials in the first half of 2023.

Webcast and Conference Call Information

The Celcuity management team will host a webcast/conference call at 4:30 p.m. ET today to discuss the fourth quarter and full year 2021 financial results and provide a corporate update. To participate in the teleconference, domestic callers should dial (877) 407-0784 and international callers should dial (201) 689-8560. A live webcast presentation can also be accessed using this weblink: View Source;tp_key=d9da71b7fd. A replay of the webcast will be available on the Celcuity website following the live event.