Celcuity Inc. is a clinical-stage biotechnology company at the forefront of developing targeted therapies for oncology. The company was co-founded in 2012 by Brian F. Sullivan and Dr. Lance G. Laing and is headquartered in Minnesota. Celcuity's lead therapeutic candidate is gedatolisib, a potent pan-PI3K and mTOR inhibitor. The company's unique approach and innovative technology have positioned it as a promising player in the oncology space.
Celcuity's Origin and Growth Celcuity was established with the goal of developing novel targeted therapies to address unmet needs in the treatment of solid tumors. The company's founders recognized the limitations of existing therapies and sought to create a more comprehensive solution that could overcome the challenges posed by single-target inhibitors.
In 2016, Celcuity initiated its first clinical trial, a Phase 1b study (B2151009) evaluating gedatolisib in patients with HR+/HER2- metastatic breast cancer. This open-label, multiple arm trial assessed gedatolisib in combination with palbociclib (a CDK4/6 inhibitor) and fulvestrant or letrozole. The study successfully determined the maximum tolerated dose for gedatolisib to be 180 mg administered intravenously once weekly.
In 2021, Celcuity obtained exclusive global development and commercialization rights to gedatolisib through a license agreement with Pfizer, Inc. This strategic partnership has been a key driver of the company's progress, allowing it to accelerate the clinical development of its lead asset.
In 2022, Celcuity reached another significant milestone by initiating patient enrollment in the VIKTORIA-1 Phase 3 clinical trial. This study evaluates gedatolisib plus fulvestrant with and without palbociclib as second-line treatment for patients with HR+, HER2- advanced breast cancer. The company's clinical development and operations teams demonstrated excellent execution, achieving 100% enrollment in the PIK3CA wild-type cohort of this study by September 2024.
Gedatolisib: A Differentiated Approach to PI3K-mTOR Inhibition Celcuity's lead therapeutic candidate, gedatolisib, is a potent, reversible, ATP-competitive, pan-PI3K and mTOR inhibitor. The drug's unique mechanism of action and pharmacokinetic properties set it apart from currently approved and investigational therapies that target PI3K or mTOR alone or in combination.
Gedatolisib's key advantages include: 1. Comprehensive inhibition of all Class I PI3K isoforms and both mTOR complexes, which prevents the compensatory resistance that can occur with isoform-specific PI3K or single mTOR kinase complex inhibitors. 2. More favorable tolerability profile compared to oral pan-PI3K or dual PI3K-mTOR inhibitors, which have been challenging for patients due to significant side effects. 3. Intravenous administration, which offers advantages in terms of market access and adherence compared to oral therapies.
Gedatolisib is a pan-class I isoform PI3K inhibitor with low nanomolar potency for the p110α, p110β, p110γ, and p110δ isoforms and mTORC1 and mTORC2 complexes. This mechanism of action is highly differentiated from other currently approved and investigational therapies that target PI3K or mTOR alone or together. By inhibiting all four PI3K isoforms and both mTOR complexes, gedatolisib prevents the confounding effect of isoform interaction that may occur with isoform-specific PI3K inhibitors and the confounding interaction between PI3K isoforms and mTOR.
Clinical Development and Upcoming Milestones Celcuity is currently advancing gedatolisib through multiple clinical trials:
1. VIKTORIA-1 Phase 3 trial: This ongoing study is evaluating the efficacy and safety of gedatolisib in combination with fulvestrant, with or without palbociclib, in patients with HR-positive, HER2-negative advanced breast cancer whose disease has progressed after prior CDK4/6 therapy. The study is designed to independently evaluate PIK3CA wild-type and PIK3CA mutant patient cohorts. 2. CELC-G-201 Phase 1b/2 trial: This trial is assessing the safety and efficacy of gedatolisib in combination with darolutamide in patients with metastatic castration-resistant prostate cancer. 3. VIKTORIA-2 Phase 3 trial: Celcuity plans to initiate this study in the second quarter of 2025, evaluating gedatolisib in combination with fulvestrant and a CDK4/6 inhibitor as first-line treatment for patients with HR-positive, HER2-negative advanced breast cancer who are endocrine therapy resistant.
The company has made significant progress in its clinical programs. The PIK3CA wild-type cohort in the VIKTORIA-1 study is fully enrolled, and Celcuity expects to report topline data for this cohort in late Q1 2025 or Q2 2025. For the PIK3CA mutant cohort, topline data is expected in the second half of 2025. If the results from the PIK3CA wild-type cohort are positive, Celcuity would expect to file a New Drug Application (NDA) with this data and follow up with a supplemental NDA if the results from the PIK3CA mutant cohort are also positive. The CELC-G-201 trial is ongoing, and preliminary data is expected in Q2 2025.
Financials and Liquidity As of September 30, 2024, Celcuity had approximately $264.1 million in cash, cash equivalents, and short-term investments. This strong financial position is the result of several financing activities, including a $60 million underwritten public offering in May 2024 and a $100 million debt financing in the same month.
The company's cash runway is expected to fund its current clinical development program activities through 2026. Celcuity's management team has demonstrated prudent financial management, with a focus on advancing its pipeline while maintaining a solid balance sheet.
For the most recent fiscal year (2023), Celcuity reported: Revenue: $0 Net Income: -$63,779,116 Operating Cash Flow: -$53,812,253 Free Cash Flow: -$53,909,897
For the most recent quarter (Q3 2024), the company reported: Revenue: $0 Net Income: -$29,791,789 Operating Cash Flow: -$20,561,698 Free Cash Flow: $35,084,608
The net loss for Q3 2024 increased by $11.39 million or 62% compared to Q3 2023. This was primarily due to a $10.1 million increase in research and development expenses, which were $27.59 million in Q3 2024 compared to $17.49 million in Q3 2023. The increase in R&D expenses was related to ongoing activities for the VIKTORIA-1 and CELC-G-201 trials as well as the initiation of the VIKTORIA-2 trial. General and administrative expenses also increased by $1.06 million to $2.47 million in Q3 2024.
Celcuity's liquidity position remains strong: Debt/Equity Ratio: 0.35 Cash and Available Credit Line: As of September 30, 2024, Celcuity had $12.6 million in cash and cash equivalents and $251.46 million in short-term investments. In May 2024, the company entered into an Amended and Restated Loan and Security Agreement with Innovatus Life Sciences Lending Fund I, LP and Oxford Finance LLC, providing for term loans up to $180 million. The company has drawn an initial $100 million under this agreement. Current Ratio: 10.36 Quick Ratio: 10.36
Celcuity is currently only selling in the United States market.
Risks and Challenges Despite the company's promising progress, Celcuity faces several risks and challenges common to clinical-stage biotechnology companies, including:
1. Uncertainty around the successful development, regulatory approval, and commercialization of gedatolisib. 2. Potential competition from other targeted therapies in the oncology space, including both approved and investigational treatments. 3. Reliance on third-party service providers for the conduct of clinical trials and manufacturing of the company's drug candidate. 4. Ability to attract and retain key personnel to support the company's growth and development.
Conclusion Celcuity's focus on developing innovative targeted therapies for oncology, exemplified by its lead candidate gedatolisib, positions the company as a promising player in the rapidly evolving oncology landscape. The company's robust clinical pipeline, strong financial position, and experienced management team suggest it is well-equipped to navigate the challenges inherent in the biotechnology industry. As Celcuity continues to advance its programs and reach key milestones, investors will closely watch the company's progress and its potential to address unmet needs in the treatment of solid tumors.