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.

On February 27, 2025 BullFrog AI Holdings, Inc. (NASDAQ: BFRG; BFRGW) ("BullFrog AI" or the "Company"), a technology-enabled drug development company using artificial intelligence (AI) and machine learning to enable the successful development of pharmaceuticals and biologics, reported its entry into a collaboration agreement with Eleison Pharmaceuticals Inc. ("Eleison"), a Phase 3 oncology company focused on novel chemotherapeutic treatments for rare cancers (Press release, Bullfrog AI, FEB 27, 2025, View Source [SID1234650693]). Under the terms of the agreement, BullFrog AI will provide access to its BullFrog Data Networks AI solution to enhance clinical trial efficiency and patient insights. Financial terms of the collaboration were not disclosed.

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"The integration of artificial intelligence in clinical trials represents a transformative shift in how pharmaceutical companies can de-risk drug development and optimize patient outcomes," said Vin Singh, CEO of BullFrog AI. "We are thrilled to partner with Eleison to apply our bfLEAP AI technology, which has the potential to refine patient selection, improve trial efficiency, and ultimately accelerate the path to market for life-saving therapies."

Through this collaboration, BullFrog AI will apply its proprietary BullFrog Data Networks solution, powered by the bfLEAP platform, to analyze clinical data from Eleison’s ongoing Phase 3 trial and previous clinical studies of glufosfamide, an investigational treatment for pancreatic cancer. The platform will evaluate the current trajectory of the trial with respect to safety signals, extract predictive biomarkers for efficacy and safety performance from prior studies to support future trial design, and provide data-driven insights to optimize Eleison’s planned clinical trials for inhaled lipid-complexed cisplatin (ILC) and dibromodulcitol (DBD). These insights are expected to streamline trial efficiency and improve decision-making for Eleison’s broader oncology pipeline.

Glufosfamide is a third-generation alkylating agent designed for greater specificity and tumor uptake, with reduced systemic toxicities and side effects. It is currently being evaluated by Eleison in a pivotal Phase 3 international randomized clinical trial, for the second-line treatment of patients with pancreatic cancer. Although pancreatic cancer is among the rarer cancer types, it is the third leading cause of death by cancer in the United States. More than 67,000 Americans and 510,000 people worldwide are diagnosed with pancreatic cancer annually. Few therapeutic options exist to treat the disease and five-year survival rates are typically less than 5%. Eleison expects to complete this ongoing Phase 3 trial in 2027.

"Our collaboration with BullFrog AI underscores our commitment to innovation in drug development," said Edwin Thomas, CEO of Eleison. "By leveraging AI-powered analytics, we aim to generate deeper insights into patient responses and safety trends, which will not only benefit our glufosfamide program but also inform the strategic direction of our broader oncology pipeline."

On February 27, 2025 Bio-Techne Corporation (NASDAQ: TECH) reported that it will present at the following investor conferences (Press release, Bio-Techne, FEB 27, 2025, View Source [SID1234650694]):

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TD Cowen 45th Annual Health Care Conference
March 4, 2025
2:30 PM EST

Leerink Partners Global Healthcare Conference
March 11, 2025
11:20 AM EDT

Barclays 27th Annual Global Healthcare Conference
March 12, 2025
10:00 AM EDT

A live webcast of the presentations can be accessed via the IR Calendar page of Bio-Techne’s Investor Relations website at View Source

On February 27, 2025 BeiGene, Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company that intends to change its name to BeOne Medicines Ltd., reported financial results and corporate updates from the fourth quarter and full year 2024 (Press release, BeiGene, FEB 27, 2025, View Source [SID1234650695]).

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"Our fourth quarter and full year results demonstrate our tremendous growth as a global oncology powerhouse, reinforced by the continued success of BRUKINSA and the development of one of the most prolific solid tumor pipelines in oncology with multiple data readouts expected this year," said John V. Oyler, Co-Founder, Chairman, and CEO at BeiGene. "BRUKINSA is now the unequivocal leader in new CLL patient starts in the U.S., holds the broadest label of any BTK inhibitor and serves as the cornerstone of our hematology franchise, showing immense promise as a backbone alongside our late stage BCL2 inhibitor, sonrotoclax, and our potential first-in-class BTK CDAC. We are also building future solid tumor franchises in breast, lung, and gastrointestinal cancers by leveraging our platforms in multi-specific antibodies, protein degraders and antibody-drug conjugates. 2025 marks an inflection point as we anticipate achieving positive GAAP operating income and operating cash flow alongside our intention to change our name to BeOne with our new NASDAQ ticker, ONC."

Fourth Quarter and Full Year 2024 Financial Snapshot

(Amounts in thousands of U.S. dollars and unaudited)

Fourth Quarter Full Year
2024 2023 % Change 2024 2023 % Change
Net product revenues $ 1,118,035 $ 630,526 77 % $ 3,779,546 $ 2,189,852 73 %
Net revenue from collaborations $ 9,789 $ 3,883 152 % $ 30,695 $ 268,927 (89) %
Total revenue $ 1,127,824 $ 634,409 78 % $ 3,810,241 $ 2,458,779 55 %
GAAP loss from operations $ (79,425) $ (383,795) (79) % $ (568,199) $ (1,207,736) (53) %
Adjusted income (loss) from operations* $ 78,603 $ (267,224) 129 % $ 45,356 $ (752,473) 106 %

* For an explanation of our use of non-GAAP financial measures refer to the "Note Regarding Use of Non-GAAP Financial Measures" section later in this press release and for a reconciliation of each non-GAAP financial measure to the most comparable GAAP measures, see the table at the end of this press release.

Key Business Updates
BRUKINSA (zanubrutinib) is an orally available, small molecule inhibitor of BTK designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared with other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease-relevant tissues. BRUKINSA has the broadest label globally of any BTK inhibitor and is the only BTK inhibitor to provide the flexibility of once or twice daily dosing. The BRUKINSA clinical development program includes approximately 7,100 patients enrolled to date in more than 30 countries and regions across more than 35 trials. BRUKINSA is approved in more than 70 markets, and more than 180,000 patients have been treated globally.

•U.S. sales of BRUKINSA totaled $616 million and $2.0 billion in the fourth quarter and full year of 2024, representing growth of 97% and 106%, respectively, over the prior-year periods, with more than 60% of the quarter over quarter demand growth coming from expanded use in chronic lymphocytic leukemia (CLL) as BRUKINSA continued to gain share as the leader in new patient starts in the U.S. in CLL and all other approved indications; BRUKINSA sales in Europe totaled $113 million and $359 million in the fourth quarter and full year 2024, representing growth of 148% and 194%, respectively, compared to the prior-year periods, driven by increased market share across all major markets, including Germany, Italy, Spain, France and the UK; and
•Entered into a patent litigation settlement agreement with MSN Pharmaceuticals, Inc. and MSN Laboratories Private Ltd. granting MSN the right to sell a generic version of BRUKINSA in the U.S. no earlier than June 15, 2037, subject to potential acceleration or extension under circumstances customary for settlement of this type.

TEVIMBRA (tislelizumab) is a uniquely designed humanized immunoglobulin G4 (IgG4) anti-programmed cell death protein 1 (PD-1) monoclonal antibody with high affinity and binding specificity against PD-1; it is designed to minimize binding to Fc-gamma (Fcγ) receptors on macrophages, helping to aid the body’s immune cells to detect and fight tumors. TEVIMBRA is the foundational asset of BeiGene’s solid tumor portfolio and has shown potential across multiple tumor types and disease settings. The TEVIMBRA clinical development program includes almost 14,000 patients enrolled to date in 35 counties and regions across 70 trials, including 21 registration-enabling studies. TEVIMBRA is approved in 45 markets, and more than 1.3 million patients have been treated globally.

•Sales of tislelizumab totaled $154 million and $621 million in the fourth quarter and full year 2024, representing growth of 20% and 16%, respectively, compared to the prior-year periods;
•Received U.S. Food and Drug Administration (FDA) approval in combination with platinum and fluoropyrimidine-based chemotherapy for the first-line treatment of unresectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma in adults whose tumors express PD-L1 (≥1); and
•Received European Commission (EC) approval in combination with chemotherapy for the first-line treatment of esophageal squamous cell carcinoma and gastric or gastroesophageal junction adenocarcinoma.

Key Pipeline Highlights

BeiGene’s portfolio strategy emphasizes rapid generation of early-stage clinical proof-of-concept data enabled by its speed- and cost-advantaged ("Fast to Proof of Concept") approach to global development operations. The Company’s in-house global research and development team, including clinical operations and development, is comprised of nearly 3,700 colleagues conducting trials across six continents and striving to ensure rigorous data quality through collaborations with regulators and investigators in over 45 countries. This strategic approach maximizes resources by channeling data-gated investments into the most promising clinically differentiated candidates quickly and de-prioritizing others. With one of the largest oncology research teams in the industry, BeiGene has demonstrated strengths in translational small molecule and biologics discovery, including three platform technologies: multi-specific antibodies, chimeric degradation activation compounds (CDACs), and antibody-drug conjugates (ADCs).

Hematology

BRUKINSA

•At the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual meeting, presented 5-year follow-up from SEQUOIA study; with adjustment for COVID-19 impact, the study demonstrated treatment with BRUKINSA reduced the risk of progression or death by 75% compared to bendamustine-rituximab in patients with treatment-naïve (TN) CLL;
•Anticipate FDA and EC approvals of BRUKINSA tablet formulation in the second half of 2025;
•Anticipate an interim analysis of progression-free survival for the Phase 3 MANGROVE study in TN mantle cell lymphoma (MCL) in the second half of 2025; and
•Anticipate completing enrollment for the relapsed/refractory (R/R) follicular lymphoma portion of the Phase 3 MAHOGANY study in the second half of 2025.

Sonrotoclax (BCL2 inhibitor)

•Planned data readouts in R/R CLL and R/R MCL Phase 2 trials and potential accelerated approval submissions in the second half of 2025;
•At ASH (Free ASH Whitepaper), presented data from the 320 mg expansion cohort of a Phase 1/1b study at a median follow-up of 1.5 years demonstrating no progression in patients with TN CLL in combination with BRUKINSA;
•More than 1,800 patients enrolled to date across the program;
•Completed enrollment in Phase 3 CELESTIAL study in TN CLL;
•Anticipate enrolling first subjects in global Phase 3 trials in R/R CLL and R/R MCL in the first half of 2025; and
•Continued enrollment in global Phase 2 trial in Waldenström’s macroglobulinemia.

BGB-16673 (BTK CDAC)  

•Continued to enroll potentially registration enabling R/R CLL Phase 2 study with data readout expected in 2026;
•More than 500 patients enrolled to date across the program;
•Anticipate initiation of Phase 3 trial in R/R CLL compared to physician’s choice in the first half of 2025; and
•Anticipate initiation of Phase 3 head-to-head trial against noncovalent BTK inhibitor pirtobrutinib in R/R CLL in the second half of 2025.

Solid Tumors

Anticipate data readouts for BGB-43395 (CDK4 inhibitor), BG-68501 (CDK2 inhibitor) and BG-C9074 (B7H4 ADC) in the first half of 2025, and internal proof-of-concept data for BG-60366 (EGFR CDAC), BGB-53038 (panKRAS inhibitor), BG-C137 (FGFR2b ADC), BGB-C354 (B7H3 ADC), and BG-C477 (CEA ADC) in the second half of 2025.

Lung Cancer 

•Tarlatamab (AMG757, DLL3xCD3 BiTE): anticipate data readout from Phase 3 study in second-line small cell lung cancer in the first half of 2025;
•Advan-TIG-302 (TIGIT antibody): anticipate interim data readout from Phase 3 study in first-line PD(L)1-high non small cell lung cancer in the second half of 2025;
•BG-60366 (EGFR CDAC): entered into the clinic in the fourth quarter of 2024; differentiated degrader mechanism to completely abolish EGFR signaling; highly potent across osimertinib-sensitive and resistant EGFR mutations; strong preclinical efficacy data with oral and daily dosing;
•BG-89894 (MAT2A inhibitor): entered dose escalation in fourth quarter of 2024; potential best-in-class characteristics with superior potency and brain penetration; strong synergy between PRMT5i and MAT2Ai in preclinical models;
•BGB-58067 (MTA-cooperative PRMT5 inhibitor): entered into the clinic in the beginning of January 2025; best-in-class potential with high potency, selectivity, and brain penetrability; and
•BG-T187 (EGFR x MET trispecific antibody): initiated dose escalation in fourth quarter of 2024; differentiated MET biparatopic design with optimal MET inhibitory activity to pursue best-in-class opportunity.

Breast and Gynecologic Cancers

•BGB-43395 (CDK4 inhibitor): continued dose escalation in monotherapy and in combination with fulvestrant and letrozole in the anticipated efficacious dose range; more than 180 patients enrolled to date and proof-of-concept expected in the first half of 2025; planning underway for Phase 3 trial in second-line HR+/HER2- metastatic breast cancer in combination with endocrine therapy ; and

•BG-68501 (CDK2 inhibitor) and BG-C9074 (B7H4 ADC): continued monotherapy dose escalation; more than 50 patients and more than 70 patients enrolled to date, respectively.
 
Gastrointestinal Cancers 

•Zanidatamab (HER2 bispecific antibody) in combination with tislelizumab and chemotherapy: anticipate primary PFS data readout from Phase 3 study in first-line HER2-positive gastroesophageal adenocarcinoma in the second half of 2025; and
•NMEs advanced into the clinic in the fourth quarter of 2024:
•BGB-53038 (panKRAS inhibitor): highly potent and selective with broad activity against KRAS mutations in multiple tumor types; limits toxicity by sparing other RAS proteins; KRAS mutations are present in 19 percent of cancers; and

•BG-C137 (FGFR2b ADC): potential first-in-class ADC for a validated target in upper gastrointestinal and breast cancers; potential superior efficacy compared to leading monoclonal antibody in both high- and medium-expression models.
Inflammation and Immunology

BGB-45035 (IRAK4 CDAC): currently in dose escalation in both SAD and MAD cohorts with more than 130 subjects enrolled; potent and selective degrader that targets both kinase and scaffold functions of IRAK4 for complete target degradation; Phase 2 study planned in 2025; proof-of-concept for tissue IRAK4 degradation in the second half of 2025.

Corporate Updates

•Announced intent to change the Company’s name to BeOne Medicines, pending shareholder approval; the new name reflects the Company’s commitment to develop innovative medicines to eliminate cancer by partnering with the global community to serve as many patients as possible;
•Announced a global licensing agreement with CSPC Zhongqi Pharmaceutical Technology (Shijiazhuang) Co., Ltd. for SYH2039 (BG-89894), a novel MAT2A inhibitor being explored for solid tumors as monotherapy and in combination with BGB-58067 (MTA-cooperative PRMT5 inhibitor);
•Changed the Company’s Nasdaq stock ticker from "BGNE" to "ONC"; and
•Hosted an investor webinar on December 16, 2024, highlighting key data from the hematology franchise from the ASH (Free ASH Whitepaper) 2024 and the 2024 San Antonio Breast Cancer Symposium and presented at the 2025 J.P. Morgan Healthcare Conference on January 13, 2025. Replays and materials can be found at the Investor Events and Presentations section of the Company’s website.

Fourth Quarter and Full Year 2024 Financial Highlights

Revenue for the fourth quarter and full year 2024 was $1.1 billion and $3.8 billion, respectively, compared to $634 million and $2.5 billion in the prior-year periods driven primarily by growth in BRUKINSA product sales in the U.S. and Europe.

Product Revenue totaled $1.1 billion and $3.8 billion for the fourth quarter and full year 2024, respectively, compared to $631 million and $2.2 billion in the prior-year periods. The increase in product revenue was primarily attributable to increased sales of BRUKINSA. For the quarter and full year 2024, the U.S. was the Company’s largest market, with product revenue of $616 million and $2.0 billion, respectively, compared to $313 million and $946 million, respectively, in the prior-year periods. U.S. sales were also positively impacted in the fourth quarter of 2024 by seasonality and the timing of customer order patterns of approximately $30 million. In addition to BRUKINSA revenue growth, product revenues were positively impacted by growth from in-licensed products from Amgen and tislelizumab.

Gross Margin as a percentage of global product sales for the fourth quarter and full year 2024 was 85.6% and 84.3%, respectively, compared to 83.2% and 82.7% in the prior-year periods on a GAAP basis. The gross margin percentage increased in both the quarter-over-quarter and year-over-year periods due to a proportionally higher sales mix of global BRUKINSA compared to other products in our portfolio, partially offset by the impact of accelerated depreciation expense of $16 million and $33 million, respectively, for the fourth quarter and full year 2024 resulting from the move to more efficient, larger scale production lines for tislelizumab. On an adjusted basis, which does not include the accelerated depreciation, gross margin as a percentage of product sales increased to 87.4% and 85.5% for the fourth quarter and full year 2024, respectively, compared to 83.7% and 83.2%, respectively, in the prior-year periods.

Operating Expenses
The following table summarizes operating expenses for the fourth quarter 2024 and 2023, respectively:
GAAP Non-GAAP
(in thousands, except percentages) Q4 2024 Q4 2023 % Change Q4 2024 Q4 2023 % Change
Research and development $ 542,012 $ 493,987 10 % $ 474,874 $ 437,383 9 %
Selling, general and administrative $ 504,677 $ 418,385 21 % $ 433,059 $ 361,435 20 %
Total operating expenses $ 1,046,689 $ 912,372 15 % $ 907,933 $ 798,818 14 %

The following table summarizes operating expenses for the full year 2024 and 2023, respectively:
GAAP Non-GAAP
(in thousands, except percentages) FY 2024 FY 2023 % Change FY 2024 FY 2023 % Change
Research and development $ 1,953,295 $ 1,778,594 10 % $ 1,668,368 $ 1,558,960 7 %
Selling, general and administrative $ 1,831,056 $ 1,508,001 21 % $ 1,549,864 $ 1,284,689 21 %
Total operating expenses $ 3,784,351 $ 3,286,595 15 % $ 3,218,232 $ 2,843,649 13 %

Research and Development (R&D) Expenses increased for the fourth quarter and full year 2024 compared to the prior-year periods on both a GAAP and adjusted basis primarily due to advancing preclinical programs into the clinic and early clinical programs into late stage. Upfront fees and milestone payments related to in-process R&D for in-licensed assets totaled $63 million and $114 million in the fourth quarter and full year 2024, respectively, compared to $31.8 million and $46.8 million in the prior-year periods.

Selling, General and Administrative (SG&A) Expenses increased for the fourth quarter and full year 2024 compared to the prior-year periods on both a GAAP and adjusted basis due to continued investment in the global commercial launch of BRUKINSA primarily in the U.S. and Europe. SG&A expenses as a percentage of product sales were 45% and 48% for the fourth quarter and full year 2024, respectively, compared to 66% and 69% in the prior-year periods.

Net Loss
GAAP net loss improved for the fourth quarter and full year 2024, as compared to the prior-year periods, primarily attributable to reduced operating losses.
For the fourth quarter of 2024, net loss per share was $0.11 per share and $1.43 per American Depositary Share (ADS), compared to $0.27 per share and $3.53 per ADS in the prior-year period. Net loss for full year 2024 was $0.47 per share and $6.12 per ADS, compared to $0.65 per share and $8.45 per ADS in the prior-year period.
Cash Provided by Operations for the fourth quarter 2024 was $75 million, an increase of $297 million over the prior-year period. For full year 2024 cash used in operations was $141 million, a decrease of $1.0 billion from the prior year period. The improvement in operating cash flows in the period was primarily driven by improved GAAP operating loss and non-GAAP operating income.
For further details on BeiGene’s 2024 Financial Statements, please see BeiGene’s Annual Report on Form 10-K for fiscal year 2024 filed with the U.S. Securities and Exchange Commission.