On February 27, 2025 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for endocrine diseases and endocrine-related tumors, reported financial results for the fourth quarter and full year ended December 31, 2024 (Press release, Crinetics Pharmaceuticals, FEB 27, 2025, View Source [SID1234650699]).

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"2024 was a year of significant progress and execution across all fronts," said Scott Struthers, Ph.D., founder and chief executive officer of Crinetics. "The acceptance of our NDA for paltusotine in acromegaly was a key achievement, and under the leadership of our new chief commercial officer, Isabel Kalofonos, our commercial team is preparing for the expected launch, which will be a truly significant milestone for the company. Our ongoing clinical programs continue to yield promising results, paving the way to initiate four late-stage trials this year. We’re also advancing four new candidates toward IND filing, underscoring the power of our internal research and development engine. Our balance sheet empowers us to execute our strategic vision over the next several years, and we are excited to bring on board a new chief financial officer, Toby Schilke, to provide financial leadership during this next stage of growth. Building on the strong foundation we’ve established, 2025 is positioned to be a transformative year for Crinetics."

Full Year 2024 and Recent Highlights:

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Strengthened leadership team with key appointments across finance, commercial, medical and clinical development to strengthen organization ahead of launch. In February 2025, Crinetics appointed Tobin "Toby" Schilke as Chief Financial Officer. In December 2024, Crinetics appointed Isabel Kalofonos as Chief Commercial Officer. In April 2024, Crinetics appointed Lise Kjems, M.D., Ph.D. as Senior Vice President of Endocrinology Clinical Research, and in October 2024, appointed Bin Zhang, M.D., M.Sc. as Senior Vice President of Oncology Clinical Development. In May 2024, Crinetics appointed Robert M. Cuddihy, M.D., as Senior Vice President of Medical Affairs.
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New Drug Application (NDA) for paltusotine for the treatment of acromegaly filed and accepted for review by U.S. Food and Drug Administration (FDA). This submission was based on positive topline results from the PATHFNDR-1 trial reported in September 2023 and from the PATHFNDR-2 trial reported in March 2024.
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European Medicines Agency (EMA) granted paltusotine Orphan Drug Designation (ODD) for the treatment of acromegaly. Designation was given following a positive recommendation from the EMA Committee for Orphan Medicinal Products, highlighting the potential impact of paltusotine for acromegaly patients in the EU.
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Phase 2 open-label study of paltusotine in carcinoid syndrome reported positive results. In March 2024, Crinetics reported positive topline results; paltusotine was shown to result in rapid and sustained reductions in frequency and severity of flushing episodes and bowel movements.

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Phase 2 TouCAHn open-label study of atumelnant in congenital adrenal hyperplasia (CAH) reported positive results. In January 2025, Crinetics reported positive topline results. Atumelnant administration was shown to result in rapid, substantial and sustained statistically significant reduction in A4 levels, the key biomarker for disease control. Atumelnant was well-tolerated and treatment with atumelnant was associated with significant clinical improvements.
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Debut of novel Nonpeptide Drug Conjugate (NDC) platform. Presented data from CRN09682 at the North American Neuroendocrine Tumor Society (NANETS) Annual Meeting in November 2024.
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Development candidates nominated in multiple programs. Crinetics has identified an oral thyroid stimulating hormone (TSH) receptor antagonist development candidate for the potential treatment of Graves’ disease, including hyperthyroidism and thyroid eye disease (TED); an oral parathyroid hormone (PTH) antagonist development candidate for the treatment of hyperparathyroidism; and an SST3 agonist development candidate for the treatment of autosomal dominant polycystic kidney disease (ADPKD).
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Strengthened balance sheet. Current cash position of $1.4B is expected to support our business activities into 2029.

Key Upcoming Milestones:

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FDA Prescription Drug User Fee Act (PDUFA) target action date of September 25, 2025 for paltusotine NDA for the treatment and maintenance therapy of acromegaly.
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Enrollment of the first patient in the pivotal Phase 3 trial of paltusotine in carcinoid syndrome is anticipated in the second quarter of 2025.
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Crinetics expects to begin enrollment of patients in two pivotal studies of atumelnant in CAH: Phase 3 in adults and Phase 2b/3 in pediatrics.
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Crinetics is also planning a study of atumelnant in Cushing’s disease. Enrollment of patients is expected to begin in late 2025 or early 2026.
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Four novel IND filings expected in 2025 for the development candidates nominated in 2024.

Fourth Quarter and Full Year 2024 Financial Results:

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Research and development expenses were $66.6 million and $240.2 million for the three months and full year ended December 31, 2024, compared to $45.6 million and $168.5 million for the same periods in 2023. The increases were primarily attributable to an increase in personnel costs of $12.6 million for the quarter ended December 31, 2024 and $43.4 million for the year ended December 31, 2024, and increased clinical and preclinical development activities of $5.1 million and $10.2 million for the quarter and year ended December 31, 2024, respectively.
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General and administrative expenses were $28.2 million and $99.7 million for the three months and full year months ended December 31, 2024, compared to $17.1 million and $58.1 million for the same periods in 2023. The increases were primarily driven by an increase in personnel costs of $5.4 million for the quarter ended December 31, 2024 and $23.8 million for the year ended December 31, 2024.
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Net loss for the three months ended December 31, 2024, was $80.6 million, compared to a net loss of $60.1 million for the same period in 2023. For the year ended December 31, 2024, the company’s net loss was $298.4 million compared to a net loss of $214.5 million for the year ended December 31, 2023.
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There were no revenues for the three months ended December 31, 2024 or 2023. Revenues were $1.0 million for the full year ended December 31, 2024, compared to $4.0 million for the same period in 2023. Revenues for 2024 were primarily derived from the paltusotine licensing agreement with Sanwa Kagaku Kenkyusho Co., Ltd. License revenues for 2023 were derived from licensing agreements with Sanwa Kagaku Kenkyusho Co., Ltd. and Cellular Longevity, Inc.
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Cash, cash equivalents, and investments totaled $1.4 billion as of December 31, 2024, compared to $558.6 million as of December 31, 2023. This includes gross proceeds of $350 million from the February 2024 private placement equity financing and $575 million from the October 2024 public offering. Based on current projections, Crinetics expects that its cash, cash equivalents and short-term investments will be sufficient to fund its current operating plan into 2029. For 2025, we anticipate our cash used in operations to be between $340 and $380 million.

Conference Call and Webcast Details

Management will hold a live conference call and webcast today, Thursday, February 27 at 4:30 p.m. ET. To participate, please dial 1-800-267-6316 (domestic) or 1-203-518-9783 (international) and refer to Conference ID CRNXQ4. To access the webcast, the direct link (HERE) or visit the Events section of the Crinetics website. Following the live event, the webcast will be archived on the Investor Relations section of www.crinetics.com.

On February 27, 2025 Castle Biosciences, Inc. (Nasdaq: CSTL), a company improving health through innovative tests that guide patient care, reported its financial results for the fourth quarter and year ended December 31, 2024 (Press release, Castle Biosciences, FEB 27, 2025, View Source [SID1234650689]).

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"Castle delivered an outstanding fourth quarter that rounded out an exceptional 2024, including 51% revenue growth and 36% test report volume growth compared to 2023," said Derek Maetzold, president and chief executive officer of Castle Biosciences. "I am incredibly proud of our team’s hard work in achieving these results, including achieving our previously provided 2025 revenue guidance range one year ahead of expectations.

"We are extremely pleased with the progress we made across our initiatives in 2024 in the areas of our core growth drivers, including a 17% increase in our dermatologic test reports (DecisionDx-Melanoma and DecisionDx-SCC combined) and a 130% increase in our TissueCypher Barrett’s Esophagus test reports, over 2023. We believe this was driven largely by the clinical value of our tests, strong execution on our commercial strategy and expansion of our clinical evidence base.

"Building on our momentum and successes, we expect to continue to focus on delivering operational excellence. Further, sound capital allocation remains a priority, including pursuing strategic opportunities, aimed at driving stockholder value in 2025 and beyond."

Twelve Months Ended December 31, 2024, Financial and Operational Highlights
•Revenues were $332.1 million, a 51% increase compared to $219.8 million in 2023.
•Adjusted Revenues, which exclude the effects of revenue adjustments related to tests delivered in prior periods, were $333.8 million, a 49% increase compared to $224.3 million in 2023.
•Delivered 96,071 total test reports in 2024, an increase of 36% compared to 70,429 in 2023:
◦DecisionDx-Melanoma test reports delivered in 2024 were 36,008, compared to 33,330 in 2023.
◦DecisionDx-SCC test reports delivered in 2024 were 16,348, compared to 11,442 in 2023.
◦MyPath Melanoma test reports delivered in 2024 were 3,909, compared to 3,962 MyPath Melanoma and DiffDx-Melanoma aggregate test reports in 2023.
◦TissueCypher Barrett’s Esophagus test reports delivered in 2024 were 20,956, compared to 9,100 in 2023.
◦IDgenetix test reports delivered in 2024 were 17,151, compared to 10,921 in 2023.
◦DecisionDx-UM test reports delivered in 2024 were 1,699, compared to 1,674 in 2023.
•Gross margin for 2024 was 79%, and Adjusted Gross Margin was 82%, compared to 75% and 80% respectively for the same periods in 2023.
•Net cash provided by operations was $64.9 million, compared to net cash used in operations of $5.6 million in 2023.

•Net income for 2024, which includes non-cash stock-based compensation expense of $50.3 million, was $18.2 million, compared to a net loss of $57.5 million in 2023.
•Adjusted EBITDA for 2024 was $75.0 million, compared to $(4.4) million in 2023.
Cash, Cash Equivalents and Marketable Investment Securities
As of December 31, 2024, the Company’s cash, cash equivalents and marketable investment securities totaled $293.1 million.
Fourth Quarter Ended December 31, 2024, Financial and Operational Highlights
•Revenues were $86.3 million, a 31% increase compared to $66.1 million during the same period in 2023.
•Adjusted Revenues, which exclude the effects of revenue adjustments related to tests delivered in prior periods, were $85.8 million, a 22% increase compared to $70.2 million for the same period in 2023.
•Delivered 24,071 total test reports, an increase of 19% compared to 20,284 in the same period of 2023:
◦DecisionDx-Melanoma test reports delivered in the quarter were 8,672, compared to 8,591 in the fourth quarter of 2023.
◦DecisionDx-SCC test reports delivered in the quarter were 4,299, compared to 3,530 in the fourth quarter of 2023.
◦MyPath Melanoma test reports delivered in the quarter were 879, compared to 1,018 in the fourth quarter of 2023.
◦TissueCypher Barrett’s Esophagus test reports delivered in the quarter were 6,672, compared to 3,441 in the fourth quarter of 2023.
◦IDgenetix test reports delivered in the quarter were 3,125, compared to 3,299 in the fourth quarter of 2023. In late 2024, the Company made modifications to its promotional investments for IDgenetix, shifting resources to inside sales and non-personal promotion.
◦DecisionDx-UM test reports delivered in the quarter were 424, compared to 405 in the fourth quarter of 2023.
•Gross margin was 76%, and Adjusted Gross Margin was 81%, compared to 78% and 82% respectively for the same periods in 2023.
•Net cash provided by operations was $24.4 million, compared to $18.6 million for the same period in 2023.
•Net income, which includes non-cash stock-based compensation expense of $11.4 million, was $9.6 million, compared to a net loss of $2.6 million for the same period in 2023.
•Adjusted EBITDA was $21.3 million, compared to $9.4 million for the same period in 2023.
2025 Outlook
The Company anticipates generating between $280-295 million in total revenue in 2025.
Fourth Quarter and Recent Accomplishments and Highlights
Dermatology
•DecisionDx-Melanoma: Findings from a prospective, multicenter study demonstrating the significant impact of the DecisionDx-Melanoma test on sentinel lymph node biopsy (SLNB) decision-making for patients with melanoma were recently published in the World Journal of Surgical Oncology; further; no patient with a DecisionDx-Melanoma-predicted risk of SLN positivity of less than 5% who decided to have an SLNB procedure had a positive node.
•DecisionDx-Melanoma: The Company announced the publication of a new independent meta-analysis in Cancers assessing the efficacy of its DecisionDx-Melanoma test in predicting melanoma patient outcomes. The article, titled "The Prognostic Value of the 31-Gene Expression Profile Test in Cutaneous Melanoma: A Systematic Review and Meta-Analysis," concluded that DecisionDx-Melanoma consistently provides improved risk stratification over staging alone to inform personalized management strategies for patients with cutaneous melanoma (CM). This recently published meta-analysis encompassed 13 peer-reviewed publications involving thousands of patients and affirmed the powerful risk stratification provided by DecisionDx-Melanoma and its potential to significantly improve care for patients with CM. See the Company’s news release from Dec. 12, 2024, for more information.

•DecisionDx-Melanoma: The Company announced the latest data from a prospective, multicenter study exploring the impact of integrating DecisionDx-Melanoma test results into SLNB decision-making for patients recently diagnosed with melanoma. The updated findings demonstrated the power of the test’s results to accurately identify patients with a low risk of metastasis who can safely forgo SLNB, thereby reducing unnecessary SLNB procedures and the associated costs and risks of complications that accompany them. The data was presented in a poster and oral presentation at The European Congress on Dermato-Oncology (Dermato-Onco2024) recently held in Vienna, Austria. See the Company’s news release from Nov. 6, 2024, for more information.
•DecisionDx-SCC: The Company announced that its poster on DecisionDx-SCC was selected as a "Late Breakers" top five finalist for the Akamai Award, recognizing the best posters at Maui Derm Hawaii 2025. Specifically, the poster shared new data from a study involving Castle’s largest cohort of patients with cutaneous squamous cell carcinoma (SCC) to date (n=1,408). This study demonstrated improved risk stratification of patients with SCC tumors located on the head or neck when the test’s results are combined with Brigham and Women’s Hospital (BWH) staging. See the Company’s news release from Jan. 17, 2025, for more information.
Gastroenterology
•The Company announced that it received assay approval from the New York State Department of Health (NYSDOH) for its TissueCypher Barrett’s Esophagus (BE) test. With this approval, all of the tests in Castle’s dermatology, gastroenterology and ophthalmology portfolios, as well as its clinical laboratories in Phoenix and Pittsburgh, are now approved by the state of New York. See the Company’s news release from Jan. 6, 2025, for more information.
Corporate
•The Company announced that it was named a Houston Top Workplace for 2024 by the Houston Chronicle. This was the fourth consecutive year the Company was ranked among the Houston metro area’s top workplaces. Castle also earned three Culture Excellence awards in the areas of Employee Appreciation, Employee Well-Being and Professional Development. See the Company’s news release from Nov. 19, 2024, for more information.
Conference Call and Webcast Details
Castle Biosciences will hold a conference call on Thursday, Feb. 27, 2025, at 4:30 p.m. Eastern time to discuss its fourth quarter and full-year 2024 results and provide a corporate update.

A live webcast of the conference call can be accessed here: View Source or via the webcast link on the Investor Relations page of the Company’s website, View Source Please access the webcast at least 10 minutes before the conference call start time. An archive of the webcast will be available on the Company’s website until March 20, 2025.

To access the live conference call via phone, please dial 833 470 1428 from the United States, or +1 404 975 4839 internationally, at least 10 minutes prior to the start of the call, using the conference ID 944585.

There will be a brief Question & Answer session following management commentary.

On February 27, 2025 Cardiff Oncology, Inc. (Nasdaq: CRDF), a clinical-stage biotechnology company leveraging PLK1 inhibition to develop novel therapies across a range of cancers, reported financial results for the fourth quarter and full year ended December 31, 2024, and provided a business update (Press release, Cardiff Oncology, FEB 27, 2025, View Source [SID1234650690]).

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"2024 was a significant year for Cardiff Oncology as we shared positive data from the first 30 patients in our lead program in first-line RAS-mut mCRC," said Mark Erlander, Ph.D., Chief Executive Officer of Cardiff Oncology. "We were excited to report that patients on the 30mg onvansertib dose arm demonstrated a 64% response rate, compared to a 33% response rate in the control arm. In the experimental arm, we observed a dose response relationship with the 30mg dose of onvansertib demonstrating increased ORR and deeper tumor regression compared to the 20mg dose of onvansertib with similar safety profiles for both doses. We believe this correlation between the dose of onvansertib and the magnitude of therapeutic effect serves as validation that onvansertib is a biologically active drug for the treatment of cancer, which we believe may translate to additional oncology indications in our pipeline. In addition to the robust efficacy signal in the CRDF-004 trial, these data suggest that onvansertib could safely be combined with both standard of care first-line chemotherapy options for mCRC in the U.S. With our balance sheet strengthened by a $40M investment from biotech specialist investors, we now look forward to sharing additional clinical updates from CRDF-004 in H1 2025."

Upcoming expected milestones

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Additional clinical data from CRDF-004 trial expected in 1H 2025

Company highlights for the quarter ended December 31, 2024, and subsequent weeks include:

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Announced positive initial data from ongoing first-line RAS-mutated mCRC clinical trial (CRDF-004)
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Phase 2 trial is currently enrolling patients with mCRC who have a documented KRAS or NRAS mutation. Onvansertib is added to standard-of-care (SoC) consisting of FOLFIRI plus bevacizumab ("bev") or FOLFOX plus bev. Patients will be randomized to either 20mg of onvansertib plus SoC, 30mg of onvansertib plus SoC, or SoC alone.
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Patients on the 30mg onvansertib dose arm demonstrated 64% objective response rate (ORR) versus 33% ORR in the control arm.
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The 30mg dose of onvansertib demonstrated a higher ORR and deeper tumor regression compared to the 20mg dose of onvansertib (64% vs. 50%)
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Onvansertib was well tolerated at both doses.

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Raised $40 million in an oversubscribed underwritten registered direct offering
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The financing included participation from new and existing healthcare dedicated investors.
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Patent issuance from the United States Patent and Trademark Office (USPTO)
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U.S. patent No. 12,144,813 has an expected expiration date of no earlier than 2043. The patent claims cover the method of using onvansertib in combination with bev for the treatment of KRAS mutated mCRC patients who have not previously been treated with bev ("bev naïve"). This patent is supported by the unexpected benefits of the treatment in such bev naïve patients.
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Presented two posters at the San Antonio Breast Cancer Symposium
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Onvansertib demonstrated synergy in combination with paclitaxel in hormone receptor positive (HR+) breast cancer cell lines and robust antitumor activity in patient-derived xenograft (PDX) models resistant to first-line therapies. These data support that onvansertib in combination with paclitaxel represents a promising therapeutic strategy for HR+ breast cancer patients after progression on endocrine therapy and CDK4/6 inhibitors. A phase 1b/2 clinical trial is ongoing to evaluate the safety and efficacy of onvansertib in combination with paclitaxel in advanced triple negative breast cancer (NCT05383196).
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Onvansertib in combination with trastuzumab deruxtecan (T-DXd) was well tolerated, overcame T-DXd resistance, and displayed enhanced anti-tumor activity compared to monotherapies in drug resistant HR+ breast cancer patient derived xenograft (PDX) models. The combination of T-DXd with onvansertib represents a promising therapeutic strategy for HR+ breast cancer patients resistant to first-line therapies.
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Published clinical data of the combination of onvansertib with FOLFIRI and bev in second-line KRAS mutant mCRC in the peer-reviewed Journal of Clinical Oncology, the flagship publication of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)
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Phase 2 clinical trial treating patients with KRAS-mutant mCRC (NCT03829410) demonstrated that onvansertib combined with FOLFIRI and bev was well-tolerated and exhibited clinical activity in the second-line setting.
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A post hoc analysis revealed a greater clinical benefit in bev naïve patients, who demonstrated an ORR of 77% and mPFS of 14.9 months compared to an ORR of 10% and mPFS of 6.6 months in those previously exposed to bev.

Full Year 2024 Financial Results:

Liquidity, cash burn, and cash runway

As of December 31, 2024, Cardiff Oncology had approximately $91.7 million in cash, cash equivalents, and short-term investments.

Net cash used in operating activities for the full year 2024 was approximately $37.7 million, an increase of approximately $6.8 million from $30.9 million for the same period in 2023.

Based on its current expectations and projections, the Company believes its current cash resources are sufficient to fund its operations into Q1 2027.

Operating results

Total operating expenses were approximately $49.3 million for the full year ended December 31, 2024, an increase of $3.4 million from $45.9 million for the same period in 2023. The increase in operating expenses was primarily due to costs associated with clinical programs and outside service costs related to the development of our lead drug candidate, onvansertib, and higher salaries and staff costs primarily due to increased headcount and stock-based compensation for additional grants to employees.

Conference Call and Webcast

Cardiff Oncology will host a corresponding conference call and live webcast today at 4:30 p.m. ET/1:30 p.m. PT. Individuals interested in listening to the live conference call may do so by using the webcast link in the "Investors" section of the company’s website at www.cardiffoncology.com. A webcast replay will be available in the investor relations section on the company’s website following the completion of the call.

On February 27, 2025 Lisata Therapeutics, Inc. (Nasdaq: LSTA) ("Lisata" or the "Company"), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, reported a business update and annonced financial results for the twelve months ended December 31, 2024 (Press release, Lisata Therapeutics, FEB 27, 2025, View Source [SID1234650691]).

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"Throughout 2024 and now into early 2025, we continue to advance our development portfolio centered around our novel product candidate, certepetide," stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Lisata. "Following the encouraging preliminary results from the ASCEND and iLSTA trials reported at this year’s ASCO (Free ASCO Whitepaper)-GI Symposium, we remain committed to exploring the broad application of certepetide’s unique mechanism of action. Our development portfolio encompasses multiple clinical and preclinical trials evaluating certepetide for the treatment of various solid tumors, including pancreatic cancer, cholangiocarcinoma, glioblastoma, colon cancer, appendiceal cancer, and melanoma. In addition, we are exploring certepetide’s versatility in non-cancerous settings such as endometriosis. For Lisata, we expect 2025 to be a data-rich year and we look forward to sharing key developments as they become available."

Development Portfolio Highlights

Certepetide as a treatment for solid tumors in combination with other anti-cancer agents

Certepetide (formerly LSTA1) is an internalizing RGD, or iRGD, (arginylglycylaspartic acid) cyclic peptide designed to selectively activate the C-end rule active transport mechanism in a tumor specific manner, resulting in systemically co-administered anti-cancer agents more efficiently penetrating and accumulating in the tumor. Additionally, certepetide has been shown to modify the tumor microenvironment, diminishing its immunosuppressive nature, enhancing cytotoxic T cell concentration and inhibiting the metastatic cascade. Lisata and its collaborators have amassed significant non-clinical data demonstrating enhanced delivery of various existing and emerging anti-cancer therapies, including chemotherapies, immunotherapies, and RNA-based therapeutics. To date, certepetide has also demonstrated favorable safety, tolerability, and clinical activity in completed and ongoing clinical trials designed to demonstrate its ability to enhance the effectiveness of standard-of-care (SoC) chemotherapy for pancreatic cancer as well as the combination of chemotherapy and immunotherapy in a variety of solid tumors. Certepetide has been awarded Fast Track designation (U.S.) and Orphan Drug Designation for pancreatic cancer (U.S. and E.U.) as well as Orphan Drug Designation for glioma, osteosarcoma, and cholangiocarcinoma (U.S.). Additionally, certepetide has received Rare Pediatric Disease Designation for osteosarcoma (U.S.). Currently, certepetide is the subject of multiple ongoing or planned clinical studies being conducted globally across several solid tumor types in combination with a variety of anti-cancer regimens, including:

•ASCEND: Phase 2b double-blind, randomized (2:1 ratio), placebo-controlled trial evaluating two dosing regimens of certepetide in combination with SoC chemotherapy (gemcitabine/nab-paclitaxel) in patients with previously untreated metastatic pancreatic ductal adenocarcinoma (mPDAC). The trial is being conducted across 25 sites in Australia and New Zealand led by the Australasian Gastro-Intestinal Trials Group (AGITG) and coordinated by the National Health and Medical Research Council Clinical Trial Centre at the University of Sydney. Cohort A, with 95 patients receiving a single intravenous (IV) dose of certepetide 3.2 mg/kg or placebo in combination with SoC, completed enrollment in the third quarter of 2023. Preliminary Cohort A data presented at the 2025 ASCO (Free ASCO Whitepaper)-GI Symposium showed a positive trend in overall survival, including four complete responses in the certepetide-treated group compared to none in the placebo

treated group. Data from Cohort B, with 63 patients receiving two IV doses of certepetide 3.2 mg/kg or placebo administered 4 hours apart in combination with SoC, is expected in the coming months with a final analysis of both cohorts available thereafter. The exact timing is dependent on accumulating the requisite number of endpoint events in Cohort B and is not something that can be accurately predicted.

•BOLSTER: Phase 2a double-blind, placebo-controlled, multi-center, randomized trial in the U.S. evaluating certepetide in combination with SoC chemotherapy in first- and second-line cholangiocarcinoma (CCA). The Company achieved complete enrollment in first-line CCA nearly six months ahead of plan, accelerating anticipated topline data readout to mid-2025. Based on this rapid enrollment rate and the pressing need to improve treatment outcomes in patients that have progressed after first-line CCA treatment, a second cohort has been added to the BOLSTER trial evaluating certepetide in combination with SoC in subjects with second-line CCA. In September 2024, Lisata announced first patient treated in the second-line CCA cohort, with enrollment completion targeted for later this year.

•CENDIFOX: Phase 1b/2a open-label trial in the U.S. evaluating certepetide in combination with neoadjuvant FOLFIRINOX based therapies in pancreatic, colon and appendiceal cancers. In December 2024, the Company announced enrollment completion in all three cohorts. The single-center study, conducted solely at the University of Kansas Cancer Center, was designed with a 3-cycle run-in period to ensure patients met specific criteria before receiving treatment. Of the 66 patients enrolled, 50 patients met the criteria and were treated with certepetide across three cohorts, including 24 with resectable or borderline resectable pancreatic cancer, 15 with high-grade colon or appendiceal cancer and peritoneal metastasis, and 11 with oligometastatic colon cancer. The trial will provide Lisata with valuable pre- and post-treatment tumor tissue data for immune profiling, along with long-term patient outcome information. CENDIFOX data are expected in the coming months; however, given this is an investigator-initiated study, the exact timing is not in Lisata’s control. The trial is funded by the University of Kansas Cancer Center and Lisata is supplying certepetide.
•Qilu Pharmaceutical, the licensee of certepetide in the Greater China territory, is currently evaluating certepetide in combination with gemcitabine and nab-paclitaxel as a treatment for first-line mPDAC. During the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting, Qilu Pharmaceutical presented an abstract sharing preliminary data from the study which corroborated previously reported findings from the Phase 1b/2a trial of certepetide plus gemcitabine and nab-paclitaxel conducted in Australia in patients with first-line mPDAC. Qilu has completed enrollment in its Phase 2 trial and data are expected in the coming months.
•iLSTA: Phase 1b/2a randomized, single-blind, single-center, safety and pharmacodynamic trial in Australia, funded by WARPNINE Inc., is evaluating certepetide in combination with SoC chemotherapy (nab-paclitaxel and gemcitabine) plus SoC immunotherapy (durvalumab) versus SoC alone in patients with locally advanced non-resectable PDAC. An interim analysis of the iLSTA trial, presented at the 2025 ASCO (Free ASCO Whitepaper) GI Symposium, showed preliminary results from the first 17 of the 30 targeted patients, corroborating preclinical data that certepetide enhances the effectiveness of immunotherapy. With 27 of the 30 patients enrolled, enrollment remains on track to be completed by the first half of 2025.
•A Lisata-funded Phase 2a, double-blind, placebo-controlled, randomized, proof-of-concept study evaluating certepetide in combination with SoC temozolomide versus temozolomide alone in patients with newly diagnosed glioblastoma multiforme (GBM) is being conducted across multiple sites in Estonia and Latvia and is planned to also include a site in Lithuania. The study is targeted to enroll 30 patients with a randomization of 2:1 in favor of the certepetide treatment group. Enrollment completion is targeted for the second half of 2025.

•FORTIFIDE: Phase 1b/2a, double-blind, placebo-controlled, three-arm, randomized study in the U.S. evaluating the safety, tolerability, and efficacy of a 4-hour continuous infusion of certepetide in combination with SoC in subjects with first-line mPDAC. As part of this study, Lisata has engaged Haystack Oncology to use its MRD technology to measure circulating tumor DNA levels at multiple timepoints in patients throughout the study as an exploratory endpoint for analyzing the early therapeutic effect of certepetide. The Company expects to enroll the first patient in the study in the first half of 2025. However, in parallel, management is investigating a potentially faster and more cost-effective approach to achieving the study objective, which may become the preferred strategy.
Lisata has entered into multiple research collaborations, including a sponsored research agreement with the University of Cincinnati to assess certepetide in combination with bevacizumab (a VEGF inhibitor) in a preclinical murine model for the treatment of endometriosis. Lisata is also partnering with Valo Therapeutics (ValoTx) to investigate the benefits of combining certepetide with ValoTx’s platform technology, PeptiCRAd, an oncolytic virus, and a checkpoint inhibitor in a preclinical murine model for the treatment of melanoma.

In November 2024, Lisata entered into an Exclusive License and Collaboration Agreement with Kuva Labs, Inc. ("Kuva"), in which Lisata granted Kuva an exclusive license to explore the synergistic potential of certepetide as a targeting and delivery agent for Kuva’s NanoMark imaging technology in solid tumors. Under the agreement, Kuva will assume full responsibility for research, development, and commercialization costs, while Lisata will be responsible for supplying certepetide pursuant to a Clinical Supply Agreement. As consideration for the license, the Company is to receive a $1.0 million upfront license fee and is eligible for certain development and commercial milestone payments of up to $19.0 million, as well as a single-digit percentage royalty on net sales.

Full Year 2024 Financial Highlights

For the year ended December 31, 2024, revenue totaled $1.0 million in connection with an upfront license fee related to the Exclusive License and Collaboration Agreement with Kuva Labs, Inc. The Company did not have any revenue for the year ended December 31, 2023.

For the year ended December 31, 2024, operating expenses totaled $23.4 million compared to $25.7 million for the year ended December 31, 2023, representing a decrease of $2.3 million or 8.9%.
Research and development expenses were approximately $11.3 million for the year ended December 31, 2024, compared to $12.7 million for the year ended December 31, 2023, representing a decrease of approximately $1.4 million, or 11.0%. This was primarily due to a reduction in expenses associated with the Phase 2b ASCEND trial which completed enrollment in the prior year, lower spend on chemistry, manufacturing and controls, and lower equity expense.
General and administrative expenses were approximately $12.1 million for the year ended December 31, 2024, compared to $13.0 for the year ended December 31, 2023, representing a decrease of approximately $0.9 million or 6.9%. This was primarily due to one-off related severance costs in the prior year associated with the elimination of the Chief Business Officer position on May 1, 2023, a reduction in equity expenses, a decrease in directors’ and officers’ insurance premiums, and a reduction in spend on legal fees partially offset by one-off settlement related costs and an increase in consulting expenses.

Overall, net losses were $20.0 million and $20.8 million for the years ended December 31, 2024 and 2023, respectively.

Balance Sheet Highlights

As of December 31, 2024, Lisata had cash, cash equivalents, and marketable securities of approximately $31.2 million. Based on its existing and planned activities, the Company believes available funds will support current operations into the second quarter of 2026.

Net Operating Loss Sale
Earlier this year Lisata received $0.9 million in non-dilutive funding as an approved participant of the Technology Business Tax Certificate Transfer Program (the "Program") sponsored by the New Jersey Economic Development Authority (NJEDA). The Program enables qualifying New Jersey-based biotechnology or technology companies to sell a percentage of their New Jersey net operating losses and research and development tax credits to unrelated qualifying corporations with a lifetime cap on the tax benefit sales of $20.0 million. To date, under the Program, the Company has sold $19.6 million in tax benefits for net proceeds of $18.4 million.
Conference Call Information
Lisata will hold a live conference call today, February 27, 2025, at 4:30 p.m. Eastern Time to discuss financial results, provide a business update, and answer questions.
Those wishing to participate must register for the conference call by way of the following link: CLICK HERE TO REGISTER. Registered participants will receive an email containing conference call details with dial-in options. To avoid delays, the Company encourages participants to dial into the conference call 15 minutes ahead of the scheduled start time.
A live webcast of the call will also be accessible under the Investors & News section of Lisata’s website and will be available for replay beginning two hours after the conclusion of the call for 12 months.

On February 27, 2025 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science, reported financial results for the year ended December 31, 2024, as well as business updates (Press release, C4 Therapeutics, FEB 27, 2025, View Source [SID1234650692]).

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"In 2024, C4T made significant progress across our degrader portfolio and our discovery collaborations. We shared clinical data from our two lead programs, where cemsidomide demonstrated a potential best-in-class profile, and CFT1946 demonstrated proof of mechanism and early signs of anti-tumor activity, and a third program, CFT8919, entered clinical development in Greater China. We also delivered two development candidates to Biogen and initiated a new discovery collaboration with Merck KGaA, Darmstadt, Germany," said Andrew Hirsch, president and chief executive officer of C4 Therapeutics. "Entering 2025, we continue to advance these clinical programs and operationalize the next phase of cemsidomide development to enable patient dosing in early 2026. We remain focused on unlocking value of cemsidomide and generating data from the Phase 1 trials of CFT1946 and CFT8919 to inform future development strategies for these programs."

FOURTH QUARTER 2024 HIGHLIGHTS AND RECENT ACHIEVEMENTS

Cemsidomide:

Multiple Myeloma (MM)

In December 2024, at the Annual Society of Hematology (ASH) (Free ASH Whitepaper) meeting, presented data from the ongoing dose escalation trial with cemsidomide in combination with dexamethasone. As of the data cutoff date of October 11, 2024, the 75 µg once daily (QD) dose level achieved an overall response rate (ORR) of 36 percent. Cemsidomide was well-tolerated across all dose levels.
The maximum tolerated dose has not yet been reached. Patients are enrolling at the 100 µg QD dose level.
Non-Hodgkin’s Lymphoma (NHL)

In December 2024, at ASH (Free ASH Whitepaper), presented data from the ongoing cemsidomide monotherapy dose escalation trial. As of the data cutoff date of October 11, 2024, cemsidomide demonstrated an ORR of 38 percent across all subtypes and doses studied. In peripheral T-cell lymphoma (PTCL), cemsidomide achieved an ORR of 44 percent and a 25 percent complete metabolic response rate.
The maximum tolerated dose has not yet been reached. Patients are enrolling at the 87.5 µg QD dose level.
CFT1946:

In October 2024, at the Targeted Protein Degradation Summit, presented new preclinical data demonstrating CFT1946 crosses the blood-brain barrier, with Kpuu values of 0.34-0.88, an important feature as a portion of patients with BRAF V600 mutant solid tumors develop brain metastases.
Continued to enroll patients with BRAF V600 mutations in the ongoing Phase 1/2 trial across multiple cohorts including monotherapy in melanoma, in combination with trametinib in melanoma and in combination with cetuximab in colorectal cancer (CRC).
CFT8919:

In November 2024, Betta Pharmaceuticals, with C4T support, initiated the Phase 1 clinical trial of CFT8919 in Greater China.
CORPORATE UPDATE:

Continued to evolve the Board of Directors with the appointment of biotechnology executive, Steve Hoerter, who has over three decades of executive management, commercial and board experience in oncology.
KEY UPCOMING MILESTONES

Cemsidomide:

Complete Phase 1 dose escalation and present data in MM and NHL in the second half of 2025.
Open expansion cohort(s) in PTCL as part of the current Phase 1/2 trial in the second half of 2025.
Enable initiation of the next phase of clinical development for MM and PTCL, with new studies expected to initiate in early 2026.
CFT1946:

Complete monotherapy Phase 1 dose escalation in BRAF V600 mutant solid tumors in the first half of 2025.
Present Phase 1 data in the second half of 2025, which will include: (1) CFT1946 monotherapy in BRAF V600 mutant solid tumors, (2) CFT1946 monotherapy expansion in melanoma and (3) CFT1946 in combination with cetuximab in CRC.
UPCOMING INVESTOR EVENTS

March 3, 2025 at 9:10 am ET: Management will be present at TD Cowen 44th Annual Healthcare Conference taking place March 3 – 5, 2025 in Boston, MA.
March 10, 2025: Management will participate in the Leerink Partners Global Healthcare Conference taking place March 9 – 12, 2025 in Miami, FL.
FOURTH QUARTER AND FULL YEAR 2024 FINANCIAL RESULTS

Revenue: Total revenue for the fourth quarter and full year ended December 31, 2024 was $5.2 million and $35.6 million, respectively, compared to $3.3 million and $20.8 million for the prior year periods. The increase in revenue was primarily due to new collaborations with Merck KGaA, Darmstadt, Germany (MKDG) and Merck, as well as revenue related to our ongoing collaboration with Betta Pharmaceuticals. Total revenue for the full year 2024 reflects revenue recognized under our collaborations with Biogen, Betta Pharmaceuticals, Merck, MKDG and Roche, and total revenue recognized for the full year 2023 reflects revenue recognized under collaboration agreements with Biogen, Roche and Calico.

Research and Development (R&D) Expense: R&D expense for the fourth quarter and full year ended December 31, 2024 was $32.5 million and $110.6 million, respectively, compared to $30.4 million and $117.7 million for the prior year periods. The increase in R&D expense for the fourth quarter was primarily related to clinical trial expense as cemsidomide and CFT1946 continue to advance. The decrease in R&D expense for the full year was primarily due to reduced headcount and external services resulting from restructuring activities that occurred in January 2024.

General and Administrative (G&A) Expense: G&A expense for the fourth quarter and full year ended December 31, 2024 was $10.4 million and $42.1 million, respectively, remaining relatively flat compared to $10.3 million and $42.1 million for the prior year periods.

Net Loss and Net Loss per Share: Net loss for the fourth quarter and full year ended December 31, 2024 was $34.6 million and $105.3 million, respectively, compared to $34.8 million and $132.5 million for the prior year periods. Net loss per share for the fourth quarter and full year ended December 31, 2024 was $0.49 and $1.52, respectively, compared to $0.68 and $2.67 for the prior year periods.

Cash Position and Financial Guidance: Cash, cash equivalents and marketable securities as of December 31, 2024 were $267.3 million, compared to $281.7 million as of December 31, 2023. The reduction in cash, cash equivalents and marketable securities during 2024 was primarily the result of $65.2 million of cash used in operating activities (net of $16 million received in milestone payments from Biogen), partially offset by $24.4 million in proceeds from the sale of shares of our common stock through our "at-the-market" offering arrangement and $20 million received under the Betta stock purchase agreement, all of which were previously disclosed. The company expects that its cash, cash equivalents and marketable securities as of December 31, 2024 will enable the company to fund its operating plan into 2027.