On February 27, 2025 Novocure (NASDAQ: NVCR) reported financial results for the quarter and full year ended December 31, 2024 (Press release, NovoCure, FEB 27, 2025, View Source [SID1234650731]). Novocure is a global oncology company working to extend survival in some of the most aggressive forms of cancer by developing and commercializing its innovative therapy, Tumor Treating Fields (TTFields).

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"Entering 2024, our team was focused on three objectives – grow our core business treating glioblastoma, launch in non-small cell lung cancer, and deliver on the promise of our clinical pipeline. I am proud to say we have successfully achieved all three goals," said Ashley Cordova, CEO Novocure. "2025 is a defining year for Novocure as we enter a new era with a multi-indication platform propelled by positive Phase 3 data in three indications – one FDA-approved and two advancing toward regulatory submission. We believe we are positioned to transform patient outcomes across multiple high-need oncology indications."

Financial updates for the fourth quarter and full year ended December 31, 2024:

Total net revenues for the year were $605.2 million, an increase of 19% year-over-year, primarily driven by continued launch success in France for Optune Gio for glioblastoma (GBM) and improved approval rates in the U.S., which are now reflected in our revenue baseline. 2025 net revenue growth is expected to reflect growth in Optune Gio active patients. As the GBM business reaches maturity we expect to continue to grow at a low mid-single digit rate this year.
Total net revenues for the quarter were $161.3 million, an increase of 21% year-over-year.
The U.S., Germany, France and Japan contributed $107.2 million, $17.4 million, $15.7 million and $8.5 million in quarterly net revenues, respectively, with our other active markets contributing $10.4 million.
Revenue in Greater China from Novocure’s partnership with Zai Lab totaled $2.0 million.
Improved approval rates in the U.S. resulted in $8.3 million of increased net revenue from prior period claims during the quarter, which we believe should not be considered in our 2025 baseline. This is in addition to the $14.0 million of increased revenue from prior period claims disclosed through the third quarter.
Gross margin for the quarter was 79%. In 2025, we expect our gross margins will be impacted by current and future product enhancements, such as the U.S. launch of our Head Flexible Electrode (HFE) transducer arrays for use with Optune Gio, and the launch of Optune Lua in metastatic non-small cell lung cancer (NSCLC). Our current analysis of the global tariff environment leads us to believe there should not be a material impact to margins in the short term and we are actively working to mitigate any potential impacts in the medium-to-long term.
Research, development and clinical studies expenses for the quarter were $51.2 million, a decrease of 6% from the same period in 2023. Clinical trial expenses can fluctuate quarter-to-quarter depending on the number of clinical trials actively underway, amount of clinical research organization services delivered and clinical materials procured.
Sales and marketing expenses for the quarter were $67.4 million, an increase of 14% from the same period in 2023. This primarily reflects the expansion of our NSCLC sales force as we launch in this new indication.
General and administrative expenses for the quarter were $72.5 million, an increase of 84% from the same period in 2023. This was primarily driven by $36.1 million in one-time stock-based compensation expenses related to U.S. Food and Drug Administration (FDA) approval of our metastatic NSCLC indication.
Net loss for the quarter was $65.9 million with loss per share of $0.61.
Adjusted EBITDA* for the quarter was $2.6 million, an increase of $34.1 million from the same period in 2023. This increase was primarily driven by revenue growth and operational efficiencies.
Cash, cash equivalents, and short-term investments were $959.9 million as of December 31, 2024.
Operational updates for the fourth quarter ended December 31, 2024:

As of December 31, 2024, there were 4,126 total active patients on TTFields therapy globally.
1,520 Optune Gio prescriptions for the treatment of GBM were received in the quarter, consistent with the same period in 2023. The U.S., Germany, France and Japan contributed 897; 190; 194 and 109 prescriptions, respectively, with the remaining 130 prescriptions contributed by other active markets.
As of December 31, 2024, there were 4,077 active Optune Gio patients on therapy. The U.S., Germany, France and Japan contributed 2,161; 564; 426 and 420 Optune Gio active patients, respectively, with the remaining 506 active patients contributed by other active markets.
On October 15, 2024, Optune Lua was approved by the U.S. FDA for the treatment of metastatic NSCLC concurrently with PD-1/PD-L1 inhibitors or docetaxel, in adults who have progressed on or after a platinum-based regimen. Between approval and year end, 52 Optune Lua prescriptions were received for metastatic NSCLC.
As of December 31, 2024, there were 20 active metastatic NSCLC patients on Optune Lua and 29 active malignant pleural mesothelioma (MPM) patients on Optune Lua.
Beginning in Q1 2026, Novocure intends to stop reporting new prescriptions received in period and will provide active patients on TTFields therapy by indication and by material market as the key operating statistics.
Fourth quarter and recent updates and achievements:

In October 2024, the FDA granted Breakthrough Device designation for the use of TTFields therapy for the treatment of brain metastases from NSCLC. Breakthrough Device designation provides more frequent, faster and interactive access to the FDA review team and senior management during the review process, priority review of marketing applications upon filing, and expedited review of pre-Premarket Approval Application (PMA) manufacturing and quality systems compliance inspections.
In October 2024, the FDA approved Novocure’s new HFE transducer arrays for use with Optune Gio for the treatment of adult patients with GBM.
In December 2024, the company announced the Phase 3 PANOVA-3 clinical trial met its primary endpoint, demonstrating a statistically significant improvement in overall survival for patients with unresectable, locally advanced pancreatic cancer. Novocure plans to submit the full data for presentation at an upcoming medical congress.
In December 2024, the FDA granted Breakthrough Device designation for the use of TTFields therapy for the treatment of unresectable, locally advanced pancreatic cancer.
In January 2025, the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) approved Novocure’s HFE transducer arrays for use with Optune Gio for the treatment of adult patients with GBM.
Anticipated clinical milestones:

Data from Phase 2 PANOVA-4 clinical trial in metastatic pancreatic cancer (2026)
Data from Phase 3 TRIDENT clinical trial in newly diagnosed GBM (2026)
Fourth quarter and full year 2024 financial results conference call:

Novocure will host a conference call and webcast to discuss fourth quarter and full year 2024 financial results at 8:00 a.m. EST today, Thursday, February 27, 2025. To access the conference call by phone, use the following conference call registration link, and dial-in details will be provided. To access the webcast, use the following webcast registration link.

The webcast and earnings slides presented during the webcast and the corporate presentation can be accessed live from the Investor Relations page of Novocure’s website, www.novocure.com/investor-relations, and will be available for at least 14 days following the call. Novocure has used, and intends to continue to use, its investor relations website, as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD.

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.

On February 27, 2025 MacroGenics, Inc. (Nasdaq: MGNX), a biopharmaceutical company focused on developing, manufacturing and commercializing innovative monoclonal antibody-based therapeutics for the treatment of cancer, reported that the Company’s management will participate in the following investor conferences in March (Press release, MacroGenics, FEB 27, 2025, View Source [SID1234650711]):

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TD Cowen 45th Annual Healthcare Conference (Boston). MacroGenics’ President & Chief Executive Officer, Scott Koenig, M.D., Ph.D., will participate in a fireside chat on Wednesday, March 5, 2025, at 10:30am ET. MacroGenics’ management will also participate in one-on-one meetings.

Leerink Partners 2025 Global Healthcare Conference (Miami). Dr. Koenig will participate in a fireside chat on Tuesday, March 11, 2025, at 11:20am ET. MacroGenics’ management will also participate in one-on-one meetings.

Barclays 27th Global Healthcare Conference (Miami). Dr. Koenig will participate in a fireside chat on Wednesday, March 12, 2025, at 2:00pm ET. MacroGenics’ management will also participate in one-on-one meetings.

Webcasts of the above presentations may be accessed under "Events & Presentations" in the Investor Relations section of MacroGenics’ website at View Source The Company will maintain archived replays of these webcasts on its website for 30 days.

On February 27, 2025 Immune-Onc Therapeutics, Inc. ("Immune-Onc"), a private, clinical-stage biopharmaceutical company advancing novel therapies in immunology and oncology by targeting myeloid cell inhibitory receptors, reported that the first patient has been dosed with IO-108 in a global clinical collaboration with Roche (Press release, Immune-Onc Therapeutics, FEB 27, 2025, View Source [SID1234650732]). The Phase 1b/2 trial is designed to evaluate IO-108, a first-in-class antibody targeting LILRB2 (also known as ILT4), in combination with Roche’s atezolizumab and bevacizumab as a potential first-line treatment for patients with locally advanced or metastatic and/or unresectable hepatocellular carcinoma (HCC).

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"IO-108 has demonstrated promising clinical efficacy as a single agent and has a well-established safety profile to combine with different standard of care regimens. Dosing the first patient in this trial represents a significant advancement for Immune-Onc and, more importantly, for people battling advanced liver cancer," said Charlene Liao, Ph.D., chief executive officer of Immune-Onc. "We are excited to work with Roche to investigate how this combination therapy with IO-108 could enhance the current standard of care and offer improved outcomes for those who urgently need new therapeutic options."

The atezolizumab and bevacizumab combination is the first cancer immunotherapy regimen approved by the U.S. Food and Drug Administration for HCC and is the standard of care recommended by the National Comprehensive Cancer Network. By modulating myeloid cells in the tumor environment, IO-108 may reduce immune suppression, thereby enhancing the overall anti-tumor response and the potential to improve patients’ clinical outcomes.

The trial, sponsored by Roche, is expected to enroll 40 patients across 25 sites worldwide in the IO-108-containing arm. This arm will be compared to an active control arm of the atezolizumab and bevacizumab combination.

ABOUT THE RANDOMIZED CLINICAL TRIAL

The Phase 1b/2 global, randomized HCC study is part of Roche’s Morpheus-Liver program. It will evaluate IO-108 in combination with atezolizumab and bevacizumab versus atezolizumab and bevacizumab, the standard of care in patients with locally advanced or metastatic and/or unresectable HCC with no prior systemic treatment.

Initially, 40 patients will be enrolled across 25 sites worldwide in the IO-108 triplet combination, which will be compared to an active control arm of the atezolizumab and bevacizumab doublet combination. The study’s primary endpoint is objective response rate, and key secondary endpoints include progression-free survival and overall survival.

Under the terms of the clinical collaboration agreement, Roche will manage the study operations, and Immune-Onc will supply IO-108 to support the trial while retaining global rights to IO-108.

Tecentriq (atezolizumab) and Avastin (bevacizumab) are registered trademarks of Genentech, a member of the Roche Group.

Learn more about the trial here.

ABOUT HEPATOCELLULAR CARCINOMA

According to the American Cancer Society1, more than 800,000 people are diagnosed with liver cancer each year worldwide. It is also one of the leading causes of cancer deaths worldwide, accounting for more than 700,000 deaths each year. The number of people diagnosed is predicted to rise, with the incidence of liver cancer increasing by 55.0% and the number of deaths increasing by 56.4% between 2020 and 20402. Hepatocellular carcinoma is the most common form of liver cancer in the United States, making up almost 90% of cases3. Nine out of ten cases of HCC are caused by chronic liver disease, which includes chronic hepatitis B and C infection, non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), alcohol-related liver disease (ALD), and cirrhosis resulting from these conditions4.

ABOUT IO-108

IO-108 is a fully human IgG4 monoclonal antibody with high affinity and specificity towards the myeloid checkpoint, LILRB2 (also known as ILT4). It blocks the interaction of LILRB2 with multiple ligands involved in cancer-associated immune suppression, including HLA-G, ANGPTLs, SEMA4A, and CD1d. Clinical data from the U.S. Phase 1 dose escalation study of IO-108 (NCT05054348) was selected as an oral presentation at the 2023 American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting and published in the Journal for ImmunoTherapy of Cancer in 2024, which demonstrated a favorable safety profile and encouraging clinical benefit utilizing IO-108 as a monotherapy and in combination with anti-PD-1 across multiple tumor types. A global, randomized Phase 1b/2 study is underway to evaluate IO-108 in combination with atezolizumab and bevacizumab as a potential first-line therapy for hepatocellular carcinoma (HCC).

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.