On March 23, 2023 Erasca, Inc. (Nasdaq: ERAS), a clinical-stage precision oncology company singularly focused on discovering, developing, and commercializing therapies for patients with RAS/MAPK pathway-driven cancers, reported financial results for the fiscal quarter and full year ended December 31, 2022, and provided business updates (Press release, Erasca, MAR 23, 2023, View Source [SID1234629241]).

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"Erasca had a strong year in 2022, culminating in our licensing of exclusive worldwide rights for naporafenib, a pan-RAF inhibitor that has demonstrated preliminary clinical proof of concept data in multiple indications and has strong synergy across our pipeline, as well as our concurrent $100 million equity financing," said Jonathan E. Lim, M.D., Erasca’s chairman, CEO, and co-founder. "In addition, we achieved several important clinical milestones, including disclosing preliminary monotherapy safety and efficacy data for our ERK1/2 inhibitor ERAS-007 and SHP2 inhibitor ERAS-601, dosing the first patient with our CNS-penetrant EGFR inhibitor ERAS-801 in THUNDERBBOLT-1, and dosing the first patient with the combination of ERAS-007 plus ERAS-601 in the MAPKlamp sub-study of HERKULES-1."

Dr. Lim continued, "In 2023, we expect data readouts for ERAS-007, ERAS-601, and ERAS 801. In addition, based on our strategic decision to focus more resources on accelerating SEACRAFT-1 for naporafenib and promising sub-studies for ERAS-007 and ERAS-601, we now anticipate SEACRAFT-1 combination data between the second and fourth quarters of 2024, dose expansion data for HERKULES-3 in BRAF-mutated colorectal cancer between the second half of 2023 and the first half of 2024, and dose expansion data for FLAGSHP-1 in HPV-negative head and neck squamous cell carcinoma in the first half of 2024."

Research and Development (R&D) Highlights

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Four Poster Presentations at ENA Symposium: In October 2022, Erasca presented four poster presentations supporting the continued development of ERK1/2 inhibitor ERAS-007, SHP2 inhibitor ERAS-601, CNS-penetrant EGFR inhibitor ERAS-801, and KRAS G12D inhibitor program ERAS-4
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Announced Exclusive Worldwide License Agreement for Naporafenib: In December 2022, Erasca announced our exclusive worldwide license agreement with Novartis for naporafenib, a
pivotal-ready pan-RAF inhibitor with first-in-class and best-in-class potential in NRAS-mutated (NRASm) melanoma, RAS Q61X tissue agnostic solid tumors, and other RAS/MAPK pathway-driven tumors
•
Received FDA Clearance of IND Application for ERAS-3490: In December 2022, the U.S. Food and Drug Administration (FDA) cleared an investigational new drug (IND) application for ERAS-3490 (CNS-penetrant KRAS G12C inhibitor) in KRAS G12C-mutated solid tumors
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Dosed First Patient in MAPKlamp Sub-study of HERKULES-1: In December 2022, Erasca dosed the first patient in the MAPKlamp sub-study of HERKULES-1, a Phase 1b trial evaluating ERK1/2 inhibitor ERAS-007 in combination with SHP2 inhibitor ERAS-601 (together, Erasca’s first MAPKlamp) in patients with RAS/MAPK pathway-altered solid tumors

Corporate Highlights

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Entered into a CTCSA with Pfizer for ERAS-007 Combination: In October 2022, Erasca announced a clinical trial collaboration and supply agreement (CTCSA) with Pfizer Inc. (Pfizer) in which Pfizer will provide its CDK4/6 inhibitor palbociclib (IBRANCE) at no cost to Erasca in connection with a clinical proof-of-concept trial evaluating ERAS-007 in combination with palbociclib for the treatment of patients with KRAS-mutated (KRASm) or NRASm colorectal cancer (CRC) and KRASm pancreatic ductal adenocarcinoma cancer (PDAC) as part of the ongoing Phase 1b/2 HERKULES-3 master protocol in patients with gastrointestinal (GI) malignancies
•
Entered into a CTCSA with Pierre Fabre for ERAS-007 Combination: In November 2022, Erasca announced a CTCSA with Pierre Fabre in which Pierre Fabre will provide its BRAF inhibitor encorafenib (BRAFTOVI) in certain territories outside of the United States at no cost to Erasca in connection with a clinical proof-of-concept trial evaluating ERAS-007 in combination with encorafenib and cetuximab for the treatment of patients with BRAF V600E-mutant metastatic CRC. This combination is being investigated as part of the ongoing Phase 1b/2 HERKULES-3 master protocol in patients with GI malignancies
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Strengthened Executive Leadership: In November 2022, Erasca promoted Minli Xie, Ph.D., to Senior Vice President of Pharmaceutical Development and Operations
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Pricing of an Underwritten Offering for $100 million: In December 2022, Erasca announced the completion of an underwritten offering of 15,384,616 shares of its common stock at a price of $6.50 per share

Key Upcoming Anticipated Milestones

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SEACRAFT-1: Phase 1b trial for naporafenib in RAS Q61X tissue agnostic solid tumors
o
Dosing of the first patient in second half of 2023
o
Initial Phase 1b combination data between the second and fourth quarters of 2024
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SEACRAFT-2: Randomized pivotal Phase 3 trial for naporafenib in NRASm melanoma
o
Dosing of the first patient in first half of 2024
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HERKULES-1: Phase 1b trial for ERAS-007 plus ERAS-601 in patients with advanced solid tumors
o
Initial Phase 1b combination data expected in first half of 2024
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HERKULES-2: Phase 1b trial for ERAS-007 in patients with advanced non-small cell lung cancer (NSCLC)
o
Initial Phase 1b combination data in first half of 2023
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HERKULES-3: Phase 1b trial for ERAS-007 in patients with GI malignancies
o
Initial Phase 1b combination data in RAS- and RAF-mutated GI malignancies in first half of 2023
o
Phase 1b combination expansion data in BRAF-mutated CRC between the second half of 2023 and the first half of 2024
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FLAGSHP-1: Phase 1b trial for ERAS-601 in patients with advanced solid tumors
o
Initial Phase 1b combination data in first half of 2023
o
Phase 1b combination data in human papillomavirus (HPV)-negative advanced head and neck squamous cell carcinoma (HNSCC) in first half of 2024
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THUNDERBBOLT-1: Phase 1 trial for ERAS-801 in patients with recurrent glioblastoma multiforme (GBM)
o
Initial Phase 1 data in recurrent GBM in second half of 2023
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AURORAS-1: Phase 1 trial for ERAS-3490 in patients with KRAS G12Cm NSCLC
o
Initial Phase 1 data in 2024

Fourth Quarter and Full Year 2022 Financial Results

Cash Position: Cash, cash equivalents, and marketable securities were $435.6 million as of December 31, 2022, compared to $459.2 million as of December 31, 2021. During 2022, Erasca completed a $100 million underwritten offering, raising net proceeds of $94.9 million after deducting underwriting discounts, commissions, and other offering expenses. Erasca expects its current cash, cash equivalents, and marketable securities balance to fund operations into the second half of 2025.

Research and Development (R&D) Expenses: R&D expenses were $29.4 million for the quarter ended December 31, 2022, compared to $24.1 million for the quarter ended December 31, 2021. The increase was primarily driven by expenses incurred in connection with clinical trials, preclinical studies, discovery activities, facilities-related expenses and depreciation, and personnel costs, including stock-based compensation. The quarter ended December 31, 2022 also included $100.0 million of in-process R&D expenses related to the $20.0 million upfront payment and issuance of shares of our common stock to Novartis. R&D expenses were $112.5 million for the full year ended December 31, 2022, compared to $73.9 million for the full year ended December 31, 2021. The full years ended December 31, 2022 and 2021 also included $102.0 million and $10.8 million, respectively, of in-process R&D expenses related to upfront and milestone payments and stock issuances under certain of our asset acquisition and license agreements.

General and Administrative (G&A) Expenses: G&A expenses were $8.7 million for the quarter ended December 31, 2022, compared to $6.9 million for the quarter ended December 31, 2021. The increase was primarily driven by personnel costs, including stock-based compensation, legal fees, and facilities and related costs. G&A expenses were $33.0 million for the full year ended December 31, 2022, compared to $22.6 million for the full year ended December 31, 2021. The full year ended December 31, 2021 also included $17.5 million of additional G&A expense for the common shares issued to the Erasca Foundation in conjunction with Erasca’s IPO.

Net Loss: Net loss was $135.3 million for the quarter ended December 31, 2022, inclusive of the $100.0 million of in-process R&D expenses recorded in connection with the Novartis license agreement, compared to $30.5 million for the quarter ended December 31, 2021. For the full year ended December

31, 2022, Erasca reported a net loss of $242.8 million, inclusive of the $100.0 million of in-process R&D expenses recorded in connection with the Novartis license agreement, or $(1.99) per basic and diluted share, compared to a net loss of $122.8 million, inclusive of the $17.5 million in expense recorded for the common shares issued to the Erasca Foundation, or $(1.85) per basic and diluted share, for the full year ended December 31, 2021.

On March 23, 2023 Equillium, Inc. (Nasdaq: EQ), a clinical-stage biotechnology company leveraging a deep understanding of immunobiology to develop novel therapeutics to treat severe autoimmune and inflammatory disorders with high unmet medical need, reported financial results for the fourth quarter and full year 2022 and provided corporate and clinical development updates (Press release, Equillium, MAR 23, 2023, View Source [SID1234629240]).

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"Equillium achieved several significant objectives during 2022, and in the process transformed into a company with a newly diversified pipeline including two wholly-owned, first-in-class clinical-stage assets, a proprietary drug discovery platform and a strong balance sheet expected to fund our development programs through multiple key milestones," said Bruce Steel, chief executive officer at Equillium. "We acquired Bioniz Therapeutics in February last year expanding our pipeline with two multi-cytokine inhibitors, EQ101 and EQ102, that we have since advanced into a Phase 2 clinical study in subjects with alopecia areata and a first-in-human clinical study in healthy volunteers to be followed by patients with celiac disease, respectively. We plan to announce data from both studies in the second half of this year. Based on the initiation of EQUATOR, our pivotal study of itolizumab in first-line acute graft-versus-host disease, and positive interim data from the EQUALISE study of itolizumab in patients with lupus nephritis, we secured a strategic partnership with Ono Pharmaceutical under which they purchased an exclusive option to acquire our rights for the development and commercialization of itolizumab. The partnership came with payments to Equillium totaling $38.6 million during December 2022, funding of itolizumab research and development costs during the option period, and potential future option exercise and milestone payments totaling approximately $139 million1. Based on our strong cash balance and significantly reduced operating burn resulting from the Ono partnership, we expect to be able to fund operations into 2025, and potentially beyond if Ono exercises its option. We look forward to upcoming data from the EQ101 and EQ102 development programs and continuing to advance itolizumab under our Ono partnership."

2022 Corporate Highlights:

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Acquired Bioniz Therapeutics, adding a proprietary product discovery platform and significantly expanding the company’s pipeline of novel immunomodulatory drug candidates, including two first-in-class clinical-stage assets: EQ101, a tri-specific inhibitor of IL-2, IL-9 and IL-15, and EQ102, a bi-specific inhibitor of IL-15 and IL-21.

•
Entered into an option and asset purchase agreement with Ono Pharmaceutical for the exclusive option to acquire our development and commercialization rights to itolizumab. Equillium received an upfront payment of $26.4 million and is also eligible to receive up to approximately $139 million1 in option exercise and payments for achieving certain development and commercialization milestones. Equillium will be responsible for conducting all research and development of itolizumab, which will be funded by Ono on a quarterly basis commencing July 1, 2022 through the option period.

•
Appointed Barbara Troupin, M.D., to Equillium’s board of directors.

1
Option exercise payment is denominated in Japanese yen (5 billion) and subject to currency exchange rates at the time of payment (U.S. dollar amount estimated above is based on the exchange rate as quoted by MUFG Bank, Ltd. on March 16, 2023). R&D funding and milestone payments are denominated in U.S. dollars.

2022 Clinical Highlights:

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Initiated a multicenter, Phase 2 open-label, proof-of-concept clinical study of EQ101 in adult subjects with at least 35% scalp hair loss due to alopecia areata. Approximately 30 subjects will be enrolled in the study where they will be dosed intravenously once weekly for 24 weeks. Subcutaneous formulation development of EQ101 is ongoing and expected to be ready for subsequent clinical studies.

•
Initiated a Phase 1 first-in-human randomized, double-blind, placebo-controlled clinical study of EQ102 administered subcutaneously in single ascending dose (SAD) and multiple ascending dose (MAD) cohorts in up to 64 healthy volunteers.

•
Initiated EQUATOR, a pivotal Phase 3 randomized, double-blind clinical study of up to 200 patients to assess the efficacy and safety of itolizumab versus placebo as a first-line therapy for acute graft-versus-host disease (aGVHD) in combination with corticosteroids.

•
Announced positive interim results from the Type B portion of the EQUALISE study of itolizumab in subjects with lupus nephritis

Anticipated Upcoming Milestones:

•
EQ101: Phase 2 clinical study in subjects with alopecia areata – initial data anticipated in 2H 2023, topline data anticipated in mid-2024

•
EQ102: Phase 1 first-in-human study in healthy volunteers and subjects with celiac disease – SAD/MAD data anticipated in 2H 2023, celiac disease patient data anticipated in 2024

•
Itolizumab: EQUALISE lupus nephritis topline data anticipated in 1H 2024, EQUATOR aGVHD interim review anticipated in 2024

Fourth Quarter and Full Year 2022 Financial Results

Revenue for the fourth quarter and full year of 2022 was $15.8 million and was derived from the company’s asset purchase agreement with Ono. There was no revenue recognized in the year ended December 31, 2021.

Research and development (R&D) expenses for the fourth quarter of 2022 were $8.5 million, compared with $7.5 million for the same period in 2021. For the full year of 2022, R&D expenses were $37.5 million, compared with $26.4 million for the full year of 2021. The year-over-year increase in R&D expenses was driven by start-up costs related to our Phase 3 EQUATOR clinical study and to a lesser extent start-up costs related to our EQ101 and EQ102 clinical studies, an increase in non-clinical research expenses and employee compensation and benefits, offset by a greater estimated Australian R&D Tax Incentive benefit and lower costs associated with our other itolizumab clinical studies.

General and administrative (G&A) expenses for the fourth quarter of 2022 were $5.2 million, compared with $2.8 million for the same period in 2021. For the full year of 2022, G&A expenses were $17.2 million, compared with $11.4 million for the full year of 2021. The year-over-year increase was primarily driven by increased legal expenses related to business development activities, greater headcount and consulting expenses, the non-cash write-off of issuance costs related to certain financings where future proceeds were unlikely, and greater overhead expenses.

Net Income for the fourth quarter of 2022 was $2.8 million, or $0.08 per basic share and diluted share, compared with a net loss of $10.6 million, or $(0.36) per basic and diluted share for the same period in 2021. Net loss for the full year of 2022 was $62.4 million, or $(1.85) per basic and diluted share, compared with a net loss of $39.1 million, or $(1.36) per basic and diluted share for the full year of 2021. The increase in net loss for the full year of 2022 compared to the full year of 2021 was driven primarily by greater operating expenses, including a non-cash in-process R&D expense related to the acquisition of Bioniz in 2022, partially offset by revenue recognized in the fourth quarter of 2022 related to the Ono partnership.

Cash, cash equivalents and short-term investments totaled $71.0 million as of December 31, 2022, compared to $80.7 million as of December 31, 2021. Cash provided by operating activities in the fourth quarter of 2022 was $27.7 million. Non-GAAP Adjusted Cash Used in Operations in the fourth quarter of 2022 was $5.2 million, which excludes the one-time upfront payment from Ono and is further adjusted by incorporating only the development funding received from Ono pertaining to itolizumab development costs in the fourth quarter of 2022. Equillium believes that its cash, cash equivalents and short-term investments will be sufficient to fund its currently planned operations into 2025.

Use of Non-GAAP Financial Measures (Unaudited)

In this release, we use the metric of Adjusted Cash Used in Operations, which is a non-GAAP financial measure and is not calculated in accordance with generally accepted accounting principles in the United States (GAAP). Adjusted Cash Used in Operations reflects adjustments to net cash provided by (used in) operating activities to exclude the effects of any one-time payments from Ono and quarterly development funding received in but unrelated to the period, and add any quarterly development funding amounts receivable related to development costs in the period.

We believe Adjusted Cash Used in Operations is a useful metric to investors as a supplement to GAAP measures in the assessment of our operating cash burn because it removes the effects of any one-time payments, which are not indicative of our ongoing cash flow from operations, and it provides better matching of the timing of itolizumab development funding payments with the associated itolizumab development costs. However, Adjusted Cash Used in Operations may fluctuate significantly from quarter to quarter, and the estimate provided for one quarter should not be assumed to be representative of other quarters. In addition, this non-GAAP financial measure may be different from non-GAAP financial measures used by other companies, even when the same or similarly titled terms are used to identify such measures, limiting their usefulness for comparative purposes.

This non-GAAP financial measure is not meant to be considered in isolation or used as a substitute for net cash provided by (used in) operating activities reported in accordance with GAAP; should be considered in conjunction with our financial information presented in accordance with GAAP; has no standardized meaning prescribed by GAAP; is unaudited; and is not prepared under any comprehensive set of accounting rules or principles. In addition, from time to time in the future, there may be other items that we may exclude for purposes of this non-GAAP financial measure, and we may in the future cease to exclude items that we have historically excluded for purposes of this non-GAAP financial measure. Likewise, we may determine to modify the nature of adjustments to arrive at this non-GAAP financial measure. Because of the non-standardized definitions of non-GAAP financial measures, the non-GAAP financial measure as used by us in this press release and the accompanying reconciliation table has limits in its usefulness to investors and may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies. Accordingly, investors should not place undue reliance on non-GAAP financial measures. Reconciliations of this non-GAAP financial measure to the most directly comparable GAAP financial measure are presented in the table at the end of this release.

About Multi-Cytokine Platform and EQ101 & EQ102

Our proprietary multi-cytokine platform generates rationally designed composite peptides that selectively block key cytokines at the shared receptor level targeting pathogenic cytokine redundancies and synergies while preserving non-pathogenic signaling. This approach is expected to avoid the broad immuno-suppression and off-target safety liabilities that may be associated with other therapeutic classes, such as Janus kinase inhibitors. Many immune-mediated diseases are driven by the same combination of dysregulated cytokines, and we believe identifying the key cytokines for these diseases will allow us to target and develop customized treatment strategies for multiple autoimmune and inflammatory diseases.

Current platform assets include EQ101, a first-in-class, selective, tri-specific inhibitor of IL-2, IL-9 and IL-15, and EQ102, a first-in-class, selective, bi-specific inhibitor of IL-15 and IL-21.

About Itolizumab

Itolizumab is a clinical-stage, first-in-class anti-CD6 monoclonal antibody that selectively targets the CD6-ALCAM signaling pathway to selectively downregulate pathogenic T effector cells while preserving T regulatory cells critical for maintaining a balanced immune response. This pathway plays a central role in modulating the activity and trafficking of T cells that drive a number of immuno-inflammatory diseases.

On March 23, 2023 Cyteir Therapeutics, Inc. ("Cyteir") (Nasdaq: CYT), a clinical stage oncology company, reported financial results for the fourth quarter and full year ended December 31, 2022 and provided an update on recent operational highlights (Press release, Cyteir Therapeutics, MAR 23, 2023, View Source [SID1234629239]).

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"We continue to be encouraged by the early clinical activity of CYT-0851 in ovarian cancer and are committed to bringing CYT-0851 to patients," said Markus Renschler, MD, President and Chief Executive Officer of Cyteir. "Our significant cash runway gives us the resources to focus our development on CYT-0851 and into a potentially registrational trial as early as the second half of 2024."

Recent Updates to the CYT-0851 Clinical Program

•
In January, Cyteir reported encouraging preliminary clinical activity in the Phase 1 dose escalation cohorts with CYT-0851, an investigational oral monocarboxylate transporter inhibitor, in combination with capecitabine for the treatment of advanced ovarian cancer. Cyteir announced the prioritization of development of CYT-0851 in combination with capecitabine in advanced ovarian cancer and deferred development of additional indications with this combination. Cyteir plans to enroll up to an additional nine patients with advanced ovarian cancer in the capecitabine combination at the 400 mg CYT-0851 dose level. If supported by the data and regulatory feedback, Cyteir intends to pursue development and potential registration of CYT-0851 in combination with capecitabine as an all-oral treatment for platinum resistant ovarian cancer. Preliminary data on the combination with capecitabine are expected to be disclosed in mid-2023.
•
Enrollment in the Phase 1 dose escalation cohorts of CYT-0851 in combination with gemcitabine in solid tumors continues. This combination is currently being evaluated at 300 mg of CYT-0851 in combination with gemcitabine, and if deemed tolerable, will advance to 400 mg of CYT-0851 in combination with gemcitabine. Preliminary data from the Phase 1 dose escalation cohorts of CYT-0851 in combination with gemcitabine are expected to be disclosed in mid-2023.

Recent Business Updates

•
In conjunction with the prioritization of the clinical plan for CYT-0851, Cyteir also ceased all discovery projects focused on identifying inhibitors of DNA damage repair. Cyteir is pursuing out licensing of its preclinical pipeline

Fourth Quarter and Full Year 2022 Financial Results

Cash and cash equivalents: Cash and cash equivalents as of December 31, 2022 were $147.1 million, which are expected to fund planned operations into 2026.

Research and development (R&D) expenses: R&D expenses were $7.5 million for the fourth quarter of 2022 versus $8.3 million for the same period in 2021 and $34.6 million for the full year 2022 versus $31.0 million for full year 2021. The year-over-year increase in R&D spending was due primarily to increased headcount and research activity. The decrease in fourth quarter 2022 R&D spending versus 2021 was due to lower clinical trial costs.

General and administrative (G&A) expenses: G&A expenses were $2.6 million for the fourth quarter of 2022 compared to $3.6 million for the same period in 2021 and $13.5 million for the full year 2022 compared to $11.3 million for full year 2021. The year-over-year increase in full year 2022 G&A expenses was primarily due to employee-related costs, as well as other administrative expenses associated with company growth and operating as a public company. The decrease in fourth quarter 2022 G&A spending versus 2021 was due to lower employee-related costs and other administrative expenses.

Net loss: Net loss was $8.8 million, or $0.25 per share, in the fourth quarter of 2022 compared to $11.8 million, or $0.34 per share, for the same period in 2021. For the full year 2022, net loss was $46.1 million, or $1.31 per share compared to $42.1 million, or $2.16 per share for full year 2021.

On March 23, 2023 Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company focused on development of targeted therapies for oncology, reported financial results for the fourth quarter and full year ended December 31, 2022 and other recent business developments (Press release, Celcuity, MAR 23, 2023, View Source [SID1234629238]).

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"We achieved a number of critical milestones in the fourth quarter. Most importantly, we dosed the first patient in our Phase 3 VIKTORIA-1 trial of gedatolisib in advanced breast cancer. This milestone, in turn, triggered the closing of our $100 million PIPE financing and a $20 million drawdown on our term loan," said Brian Sullivan, CEO and Co-Founder of Celcuity. "These financings significantly strengthen our balance sheet and are expected to provide the capital we need to fund operations through 2025

Fourth Quarter 2022 Business Highlights and Other Recent Developments

● In December, the first patient was dosed in the Phase 3 VIKTORIA-1 clinical trial. VIKTORIA-1 is evaluating the safety and efficacy of Celcuity’s lead drug product candidate, gedatolisib, an investigational pan-PI3K/mTOR inhibitor, in combination with fulvestrant with or without palbociclib in adults with HR+/HER2- advanced breast cancer whose disease progressed while receiving prior CDK4/6 therapy. Patient enrollment is currently in progress. Gedatolisib was previously granted Breakthrough Therapy designation in July 2022 from the U.S. Food and Drug Administration for the treatment of patients with HR+/HER2- advanced breast cancer whose disease progressed during treatment with a CDK4/6 therapy and a non-steroidal aromatase inhibitor.

● The dosing of the first patient in the VIKTORIA-1 clinical trial triggered the closing of a $100 million private placement and drawdown of a $20 million term loan tranche in December. Proceeds from the private placement, combined with the debt facility and the company’s current cash, cash equivalents and marketable securities, are expected to be sufficient to fund Celcuity’s current operating plan through 2025.

● In December 2022, Celcuity presented updated efficacy and safety results from a Phase 1b trial evaluating gedatolisib during a Spotlight Poster Discussion at the 2022 San Antonio Breast Cancer Symposium (SABCS). The presentation reported that patient sub-groups with and without PIK3CA mutations achieved comparable efficacy in the four expansion arms of the Phase 1b study. Additionally, median progression free survival of 42.3 months was reported for patients who were treatment naïve in the advanced setting, which compares favorably to published data for current standard-of-care regimens for this patient population.

● In February 2023, the company presented data from preclinical studies evaluating gedatolisib and other PI3K/AKT/mTOR inhibitors in prostate cancer cell lines at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium. The presentation demonstrated gedatolisib’s superior potency and efficacy across different prostate cancer cell lines relative to other PI3K/AKT/mTOR inhibitors regardless of PTEN or PI3K status.

● In March 2023, an abstract reporting data from preclinical studies evaluating gedatolisib and other PI3K/AKT/mTOR inhibitors in gynecological cancer lines was published in advance of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2023 to be held April 14-19, 2023. A poster will be presented at the meeting on April 18, 2023 in Orlando, Florida.

● Enrollment is ongoing in the FACT-1 and FACT-2 trials for CELsignia selected patients who have early-stage HR+/HER2- breast cancer with interim results expected in the second half of 2023.

Fourth Quarter and Full Year 2022 Financial Results

Unless otherwise stated, all comparisons are for the fourth quarter and full year ended December 31, 2022, compared to the fourth quarter and full year ended December 31, 2021.

Total operating expenses were $11.6 million for the fourth quarter of 2022, compared to $6.3 million for the fourth quarter of 2021. Operating expenses for the full year 2022 were $39.4 million, compared to $28.4 million for the full year 2021.

Research and development (R&D) expenses were $10.6 million for the fourth quarter of 2022, compared to $5.5 million for the prior-year period. The increase was primarily the result of activities supporting the initiation of the VIKTORIA-1 pivotal trial.

R&D expenses for the full year 2022 were $35.3 million, compared to $25.8 million for the prior year. The increase in R&D expenses included a $10.0 million reduction in gedatolisib licensing related expenses. This reduction was offset by increases in other research and development expenses, which included employee and consulting expenses, increased expenses for existing clinical trials and for activities supporting the initiation of the VIKTORIA-1 pivotal trial.

General and administrative (G&A) expenses were $1.0 million for the fourth quarter of 2022, compared to $0.8 million for the prior-year period. The increase in G&A expenses arose primarily from non-cash stock-based compensation

G&A expenses for the full year 2022 were $4.1 million, compared to $2.6 million for the prior year. The increase arose primarily from non-cash stock-based compensation.

Net loss for the fourth quarter of 2022 was $11.6 million, or $0.69 loss per share, compared to a net loss of $6.8 million, or $0.45 loss per share, for the fourth quarter of 2021. Net loss for the full year 2022 was $40.4 million, or $2.64 loss per share, compared to a net loss of $29.6 million, or $2.21 loss per share, in 2021. Non-GAAP adjusted net loss for the fourth quarter of 2022 was $10.2 million, or $0.60 loss per share, compared to non-GAAP adjusted net loss of $5.6 million, or $0.37 loss per share, for the fourth quarter of 2021. Non-GAAP adjusted net loss for the full year 2022 was $34.9 million, or $2.26 per share, compared to non-GAAP adjusted net loss of $21.4 million, or $1.60 per share, for 2021. 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 in the United States (GAAP) to non-GAAP financial measures, please see the financial tables at the end of this press release

Net cash used in operating activities for the fourth quarter of 2022 was $9.5 million, compared to $6.1 million for the fourth quarter of 2021. Net cash used in operating activities for the full year 2022 was $36.0 million, compared to $20.3 million for the full year 2021

At December 31, 2022, Celcuity had cash, cash equivalents and short-term investments of $168.6 million

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 2022 financial results and provide a corporate update. To participate in the teleconference, domestic callers should dial 1-877-407-0784 and international callers should dial 1-201-689-8560. A live webcast presentation can also be accessed using this weblink: View Source;tp_key=796153253b. A replay of the webcast will be available on the Celcuity website following the live event.

On March 23, 2023 BridgeBio Pharma, Inc., (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, reported that the first patient with non-small cell lung cancer (NSCLC) has been dosed in its Phase 1/2 clinical trial of BBP-398, an investigational SHP2 inhibitor, with Bristol Myers Squibb’s OPDIVO (nivolumab) in advanced solid tumors with KRAS mutations (NCT05375084) (Press release, BridgeBio, MAR 23, 2023, View Source [SID1234629237]).

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KRAS mutations occur in approximately 27% of NSCLC cases and approximately 17% of malignant solid tumors. By combining SHP2 inhibition with KRAS inhibition in patients, there is potential to prevent oncogenesis and overactive cellular proliferation.

"SHP2 has been shown to be a key regulator of both tumor and immune cell signaling. In KRAS mutant tumors, SHP2 promotes survival, proliferation and decreased immunogenicity by driving the active form of KRAS, while in immune cells, it associates with PD-1 which leads to immunosuppression in the tumor microenvironment," said Eli Wallace, Ph.D., chief scientific officer of oncology at BridgeBio. "By partnering with Bristol Myers Squibb on this trial, we hope to show that targeting PD-1 with a two-prong approach can unlock the potent benefits of immunotherapy against this cancer and provide new treatment options for patients who need them."

The Phase 1 study will include a dose escalation period followed by dose expansion, and is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of BBP-398 in combination with OPDIVO. Both the dose escalation and expansion periods will enroll patients who were unresponsive to standard of care with advanced NSCLC with a KRAS mutation. The dose expansion period will evaluate the antitumor activity of BBP-398 in combination with OPDIVO.

In May 2022, BridgeBio entered into an exclusive license agreement with Bristol Myers Squibb to develop and commercialize BBP-398 in oncology worldwide, except for in mainland China and other Asian markets. These territories are part of BridgeBio’s separate strategic collaboration with LianBio announced in 2020. The 2022 agreement with Bristol Myers Squibb expands upon the earlier agreement between the companies signed in 2021 to investigate the combination of BBP-398 with OPDIVO (nivolumab) in patients with advanced solid tumors with KRAS mutations.

Additionally, BridgeBio has a non-exclusive clinical collaboration with Amgen to evaluate the combination of BBP-398 with LUMAKRAS in patients with advanced solid tumors with the KRASG12C mutation.

BBP-398, as a monotherapy or in combination with other targeted therapies, could potentially be a promising therapy for patients with KRAS mutations. Initial Phase 1 data from the ongoing BBP-398 trial is expected in 2023.

OPDIVO (nivolumab) is a trademark of Bristol-Myers Squibb Company.

About BBP-398
BBP-398 is a SHP2 inhibitor that is being developed for difficult-to-treat cancers and was founded through a collaboration with The University of Texas MD Anderson Cancer Center’s Therapeutics Discovery division. SHP2 is a protein-tyrosine phosphatase that links growth factor, cytokine and integrin signaling with the downstream RAS/ERK MAPK pathway to regulate cellular proliferation and survival. In May 2022, BridgeBio entered an exclusive license with Bristol Myers Squibb to develop and commercialize BBP-398, a potentially best-in-class SHP2 inhibitor. Additionally, BridgeBio has a strategic collaboration with LianBio for clinical development and commercialization of BBP-398 in combination with various agents in solid tumors such as non-small cell lung cancer, colorectal and pancreatic cancer, in mainland China and other Asian markets and clinical collaborations; with Bristol Myers Squibb for combination with OPDIVO (nivolumab) in patients with advanced solid tumors with KRAS mutations; and with Amgen for combination with LUMAKRAS (sotorasib), Amgen’s KRASG12C inhibitor, in patients with advanced solid tumors with KRASG12C mutations.