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.

On February 27, 2025 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, reported operational highlights and financial results for the fourth quarter and year ended December 31, 2024 (Press release, Intellia, FEB 27, 2025, View Source [SID1234650708]).

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"We are off to an excellent start in 2025 with renewed focus and strong operational execution across our three, pivotal Phase 3 studies," said Intellia President and Chief Executive Officer John Leonard, M.D. "We are excited by the clinical data presented during the fourth quarter. Our Phase 1/2 results in HAE suggest that NTLA-2002 could represent a functional cure for patients with HAE – for the first time a patient has the potential to be both free from attacks and free from chronic therapy. Similarly, the rapid, deep and durable reductions in serum TTR demonstrated to date in our Phase 1 study of nex-z in ATTR represent a highly differentiated profile that may offer patients an opportunity to stabilize or improve their clinical readouts in an otherwise unrelenting, progressive disease."

Fourth Quarter 2024 and Recent Operational Highlights

Hereditary Angioedema (HAE)

NTLA-2002: NTLA-2002 is a wholly owned, investigational in vivo CRISPR-based therapy designed to knock out the KLKB1 gene in the liver, with the goal of lifelong control of HAE attacks after a single dose.
In January, Intellia announced the first patient was dosed with NTLA-2002 in the global Phase 3 HAELO study. The Company expects to complete enrollment in the second half of 2025.
The Company plans to submit a Biologics License Application in the second half of 2026 to support plans for a U.S. launch in 2027.
Intellia expects to present longer-term data from the ongoing Phase 1/2 study in 2025. The data will include patients in the Phase 2 portion who initially received a 25 mg dose or placebo and were subsequently given the 50 mg dose of NTLA-2002 selected for the Phase 3 study.
Transthyretin (ATTR) Amyloidosis

Nexiguran ziclumeran (nex-z, also known as NTLA-2001): Nex-z is an investigational in vivo CRISPR-based therapy designed to inactivate the TTR gene in liver cells, thereby preventing the production of transthyretin (TTR) protein for the treatment of ATTR amyloidosis. Nex-z offers the possibility of halting and reversing the disease by driving a deep, consistent and potentially lifelong reduction in TTR protein after a single dose. Nex-z has been generally well tolerated across all patients and at all dose levels tested. The most common treatment-related adverse event was an infusion reaction, which were mild or moderate; all patients were able to receive the intended dose of nex-z. Intellia leads development and commercialization of nex-z in collaboration with Regeneron.
ATTR Amyloidosis with Cardiomyopathy (ATTR-CM):
Enrollment in the pivotal Phase 3 MAGNITUDE trial is progressing ahead of our target projections and we anticipate enrollment to exceed 550 total patients by year end.
Intellia presented data from the ongoing Phase 1 study at the 2024 American Heart Association (AHA) Scientific Sessions and published the findings online in the New England Journal of Medicine. Across all patients (n=36), a single dose of nex-z led to consistently rapid, deep and sustained serum TTR reduction, regardless of baseline levels, through the latest follow-up. At month 12, the mean serum TTR reduction was 90%, and the mean absolute residual serum TTR concentration was 17 µg/mL. With 11 patients who have reached 24 months of follow-up, all patients continued to show a sustained response with no evidence of a waning effect over time. The consistently low levels of serum TTR are anticipated to reduce the rate of ongoing amyloid formation and potentially allow for amyloid clearance and improvement in cardiac function. Nex-z was generally well tolerated across all patients.
Hereditary ATTR Amyloidosis with Polyneuropathy (ATTRv-PN):
We are actively screening patients for the Phase 3 MAGNITUDE-2 study and are on track to dose the first patient in the first quarter of 2025.
Intellia presented data from the ongoing Phase 1 study in November. At month 12, patients who received a dose of 0.3 mg/kg or higher (n=33) had a mean serum TTR reduction of 91% and mean absolute residual serum TTR concentration of 20 µg/mL. For the 16 patients who reached 24 months of follow-up, there was no change to their post-dose TTR levels. It is anticipated that greater TTR reduction may lead to a greater clinical benefit in patients with ATTRv-PN. Nex-z was generally well tolerated across all patients and at all dose levels tested.
In November, Intellia announced the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) to nex-z for the treatment of ATTRv-PN.
Intellia expects to present longer-term data from both ATTR-CM and ATTRv-PN patients in the Phase 1 study in 2025. The data will include updated measures of clinical efficacy and safety.
Platform Update

Intellia continues to apply novel technologies, such as CRISPR-based gene editing technologies and lipid nanoparticle (LNP) delivery technologies, to develop in vivo and ex vivo product candidates. Treating—and potentially curing—a broad range of severe diseases requires the application of multiple technologies. With Intellia’s proprietary technology at the core of the platform, the Company continues to research and develop new gene editing and delivery technologies to expand the therapeutic opportunities, furthering progress on the frontier of genetic medicine.
Corporate Update

On January 9, 2025, the Company announced that, after a strategic review of its business, it elected to prioritize late-stage programs – NTLA-2002 for HAE and nex-z for ATTR amyloidosis – and select research investments to focus on near-term value creation. As a result, the Company discontinued NTLA-3001 and other, undisclosed programs, and is reducing its workforce by approximately 27% in 2025. The Company expects to incur charges of approximately $8.0 million for severance and other employee termination-related costs in the first quarter of 2025.
Upcoming Events

The Company will participate in the following events during the first quarter of 2025:

AAAAI/WAO Joint Congress, March 1, San Diego
TD Cowen 45th Annual Health Care Conference, March 4, Boston
Leerink 2025 Global Biopharma Conference, March 10, Miami
Barclays 27th Annual Global Healthcare Conference, March 11, Miami
Jefferies Biotech on the Beach Summit, March 12, Miami
Fourth Quarter and Full-Year 2024 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $861.7 million as of December 31, 2024, compared to $1.0 billion as of December 31, 2023. The Company’s cash position as of December 31, 2024, is expected to fund operations into the first half of 2027.
Collaboration Revenue: Collaboration revenue was $12.9 million during the fourth quarter of 2024, compared to negative $1.9 million during the fourth quarter of 2023. The $14.8 million increase was mainly driven by collaboration revenue received under the Regeneron agreements.
R&D Expenses: Research and development (R&D) expenses were $116.9 million during the fourth quarter of 2024, compared to $109.0 million during the fourth quarter of 2023. The $7.9 million increase was primarily driven by the advancement of our lead programs. Stock-based compensation expense included in R&D expenses was $24.4 million for the fourth quarter of 2024.
G&A Expenses: General and administrative (G&A) expenses were $32.4 million during the fourth quarter of 2024, compared to $29.0 million during the fourth quarter of 2023. The $3.4 million increase was primarily related to stock-based compensation. Stock-based compensation expense included in G&A expenses was $15.2 million for the fourth quarter of 2024.
Net Loss: Net loss was $128.9 million for the fourth quarter of 2024, compared to $132.2 million during the fourth quarter of 2023.
Conference Call to Discuss Fourth Quarter and Full-Year 2024 Results

The Company will discuss these results on a conference call today, Thursday, February 27 at 8 a.m. ET.
To join the call:

U.S. callers should dial 1-833-316-0545 and international callers should dial 1-412-317-5726, approximately five minutes before the call. All participants should ask to be connected to the Intellia Therapeutics conference call.
Please visit this link for a simultaneous live webcast of the call.
A replay of the call will be available through the Events and Presentations page of the Investors & Media section on Intellia’s website at intelliatx.com, beginning on February 27 at 12 p.m. ET.

On February 27, 2025 Akeso, Inc. (9926.HK) reported the first patient has been enrolled in the Phase I clinical trial of AK138 D1 for the treatment of advanced malignancies in Australia (Press release, Akeso Biopharma, FEB 27, 2025, View Source;adc-strategy-302387166.html [SID1234650729]). AK138D1, a self-developed and differentiated HER3-targeting ADC (antibody-drug conjugate), is Akeso’s first ADC drug to enter clinical studies.

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Akeso is dedicated to transforming the global oncology treatment landscape by establishing new standards of care for cancer patients worldwide. A key element of this mission is the company’s "IO 2.0 + ADC" strategy. Akeso’s self-developed, first-in-class bispecific antibodies, cadonilimab (PD-1/CTLA-4) and ivonescimab (PD-1/VEGF), have both received regulatory approval and are now available to patients in China. Akeso is the only global biopharmaceutical company with two approved bispecific checkpoint antibodies for cancer immunotherapy.

The continued development of differentiated ADC therapies, such as AK138D1, further extend Akeso’s ability to explore the full clinical potential of its innovative in-house pipeline and to create synergistic new combination treatment options includes that multiple checkpoints and tumor targets.

Dr. Xia Yu, Founder, Chairwoman, President and CEO of Akeso said,

"The initiation of the clinical study for AK138D1 in Australia marks a pivotal moment in Akeso’s strategic advancement into next-generation ADC therapies. Building on our global prominence in bispecific antibodies and a robust pipeline of high-potential drug candidates, the development of AK138D1 and subsequent ADCs/bispecific ADCs will significantly bolster our product offering. This is part of Akeso’s continued effort to redefine standard of care in cancer treatment.

Following our achievements in bispecific antibody development, ADC therapies have emerged as a strategic priority for Akeso. Based on our extensive experience in bispecific antibody development, we are excited by the potential of our proprietary ADCs and bispecific ADCs. Our goal is to enhance drug efficacy while minimizing ADC toxicity, offering transformative treatment alternatives for patients worldwide.

Moreover, Akeso has established cutting-edge ADC research, pilot production, and manufacturing facilities. These investments position us for high-quality clinical development and for global market expansion of our ADC portfolio."

HER3 has emerged as an active target in cancer drug development and is the focus of ongoing research. Although experimental drugs targeting HER3 have not shown satisfactory antitumor effects over the past 30 years, studies indicate that HER3 is expressed or overexpressed in various malignancies, including breast cancer, ovarian cancer, lung cancer, colorectal cancer, melanoma, head and neck cancers, cervical cancer, and prostate cancer. Additionally, upregulation of HER3 and its synergistic interactions with other receptors contribute to tumor initiation, metastasis, and resistance to certain anticancer treatments, such as resistance to EGFR-targeted therapies, endocrine therapy in breast cancer, HER2-targeted therapies, and chemotherapy.

About AK138D1

Injectable AK138D1 is a HER3-targeted antibody-drug conjugate (ADC), with a fully humanized anti-HER3 IgG1 antibody, patritumab. It is conjugated to the topoisomerase I inhibitor DXd through a cleavable linker, MC-AAA (maleimide-alanine-alanine-alanine). After binding to HER3 on tumor cells, the ADC is internalized into the tumor cells, where the linker is cleaved, releasing the membrane-permeable DXd. This leads to DNA damage and subsequent cell apoptosis. Currently, Akeso has initiated patient enrollment for a Phase I dose-escalation and expansion clinical study in Australia. This study will investigate the safety, tolerability, pharmacokinetics, and preliminary efficacy of AK138D1 for the treatment of advanced malignancies.

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 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a commercial biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, reported fourth quarter and full year 2024 financial results and corporate updates (Press release, Iovance Biotherapeutics, FEB 27, 2025, View Source [SID1234650709]).

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Frederick Vogt, Ph.D., J.D., Interim President and Chief Executive Officer of Iovance, stated, "In 2024, we successfully drove strong early adoption for our U.S. commercial launch of Amtagvi for patients with previously treated advanced melanoma. Strong demand and growth are continuing and on track to accelerate for both Amtagvi and Proleukin in 2025 and beyond in the U.S. and globally. Our top commercial priorities are to drive broader adoption and utilization, increase patient referrals, add large community practices to our authorized treatment center (ATC) network, expand the U.S. market, and secure regulatory approvals in three new markets outside the U.S. I am confident that Iovance is well positioned to remain the global leader in innovating, developing, and delivering current and future generations of TIL cell therapy for patients with cancer."

Fourth Quarter and Full Year 2024 Financial Results, Corporate Guidance, and Updates

Product Revenue and Guidance

Fourth Quarter 2024 Total Product Revenue: Iovance recognized total revenue of $73.7 million from sales of Amtagvi and Proleukin during the fourth quarter ended December 31, 2024.
Amtagvi Revenue: Product revenue was $48.7 million from U.S. Amtagvi sales in the fourth quarter of 2024, reflecting strong adoption with increasing demand. Amtagvi revenue is recognized upon patient infusion.
Proleukin Revenue: Product revenue also included $25.0 million in Proleukin sales in the fourth quarter of 2024. Proleukin is used in the Amtagvi treatment regimen and other commercial, clinical, manufacturing, and research settings, which provide additional revenue. Proleukin revenue is generally recognized upon delivery to distributors and ATCs.
Full Year 2024 Total Product Revenue: Total product revenue was $164.1 million and achieved the high end of the company’s guidance range of $160 to $165 million for the full year 2024. Full year product revenue included the first three quarters of sales following the U.S. launch of Amtagvi on February 20, 2024. The full year 2024 product revenue for Amtagvi and Proleukin was $103.6 million and $60.5 million, respectively.
Significant Amtagvi Growth Potential at Approximately 70 ATCs in 2025: Amongst current ATCs, 76% completed tumor resections, 64% infused one or more patients, and 13% infused more than 10 patients, highlighting significant growth potential at existing ATCs.
Full Year 2025 Total Product Revenue Guidance: Iovance is reaffirming total product revenue guidance within the range of $450 to $475 million for 2025, the first full calendar year of Amtagvi sales. Amtagvi adoption is on track to continue accelerating throughout 2025 with broader utilization, higher demand, and growth in community referrals. Iovance also expects significant growth in total product revenue for full year 2026, and beyond.
Gross margins are expected to increase over time and remain on track to surpass 70% over the next several years. In line with anticipated growth in Amtagvi demand, Proleukin revenue is also expected to increase significantly in 2025 and beyond.
Full Year 2025 Expense Guidance: Cash burn for full year 2025 is expected to be under $300 million, including completion of construction of the Iovance Cell Therapy Center (iCTC) manufacturing expansion.
Cash Position: As of February 26, 2025, Iovance had cash, cash equivalents, investments, and restricted cash of approximately $422 million. The current cash position and anticipated product revenue are expected to be sufficient to fund current and planned operations, including manufacturing expansion, into the second half of 2026.
Amtagvi (Lifileucel) U.S. Launch Highlights in Advanced Melanoma

The U.S. FDA approved Amtagvi (lifileucel) on February 16, 2024, as the first treatment option for patients with advanced melanoma after anti-PD-1 and targeted therapy. Amtagvi is the first FDA-approved T cell therapy for a solid tumor indication.
Approximately 70 U.S. ATCs are active across 32 states and 95% of addressable patients live within 200 miles of an ATC. Additional U.S. ATCs will be added steadily throughout 2025, focusing on quality ATCs with a high volume of eligible patients, including large community practice ATCs.
Community referral activities are increasing throughout the U.S. to drive additional patient volume to these ATCs. Large community practices are currently onboarding, creating a new and significant opportunity for more patients to receive Amtagvi after frontline therapy.
Manufacturing turnaround time is aligning with launch expectations of approximately 34 days from inbound to return shipment to ATCs. Efforts are underway to shorten the turnaround time in 2025. The commercial manufacturing experience remains consistent with prior clinical experience.
Amtagvi is a preferred second-line or subsequent therapy in the National Comprehensive Cancer Network guidelines for treatment of cutaneous melanoma.
Reimbursement remains successful, with an average financial clearance time of about three weeks.
Approximately 75% of Amtagvi patients are covered by private payers. To date, payers or plans covering more than 250 million lives have added Amtagvi to policies since its launch.
Launch Expansion into New Markets

Amtagvi has the potential to address more than 20,000 patients annually with previously treated advanced melanoma across the U.S. and initial global markets with significant populations of previously treated advanced melanoma patients.1
Regulatory dossiers are under review, submitted, or planned across multiple international markets for lifileucel for the treatment of adult patients with unresectable or metastatic melanoma after anti-PD-1 and targeted therapy. If approved, lifileucel will be the first and only approved therapy in this treatment setting in all markets.
A marketing authorization application (MAA) was submitted to the Medicines and Healthcare products Regulatory Agency in the United Kingdom for potential approval in the first half of 2025.
A new drug submission (NDS) to Health Canada was accepted for a prioritized 200-day review process through the Notice of Compliance with Conditions (NOC/c) policy for potential approval in mid-2025.
An MAA for all EU member states was accepted for review by the European Medicines Agency for potential approval in the second half of 2025.
Named patient programs are planned in the UK, France, Germany, Canada, Switzerland, and Australia in 2025 to provide reimbursed access to treatment prior to approval or final pricing and are also expected to provide initial revenue from these markets.
Additional regulatory submissions remain on track in 2025 and 2026, including Australia in the first half of 2025 and Switzerland in the second half of 2025.
A total of 15 active ATCs are targeted by year-end to support initial launch markets outside the U.S.
Recent Iovance TIL Cell Therapy Pipeline Highlights

Lifileucel in Frontline Advanced Melanoma
Strong momentum continues with global site activation and patient enrollment in the registrational TILVANCE-301 trial, which is intended to support accelerated and full U.S. approvals of Amtagvi in combination with pembrolizumab in frontline advanced melanoma, as well as full approval of Amtagvi in post-anti-PD-1 melanoma.
Lifileucel in Previously Treated Advanced Non-Small Cell Lung Cancer (NSCLC)
Iovance expects to share additional data in the second half of 2025 from the IOV-LUN-202 registrational Phase 2 trial in post-anti-PD-1 NSCLC.
The IOV-LUN-202 trial is intended to support a potential accelerated approval of lifileucel in post-anti-PD-1 NSCLC in the U.S., with an anticipated regulatory decision in 2027. The FDA previously provided positive regulatory feedback on the proposed potency matrix in NSCLC and the IOV-LUN-202 clinical trial design.
Enrollment in IOV-LUN-202 continues with high demand at clinical sites in the U.S., Canada, and Europe with additional site activations underway in new regions with strong track records for enrollment in NSCLC studies.
Lifileucel in Frontline Advanced NSCLC
Iovance is pursuing a frontline therapy strategy to integrate lifileucel plus pembrolizumab following chemotherapy for patients with EGFR wild type NSCLC, representing most patients with an unmet medical need in this setting. This regimen will be investigated in a new Cohort 3D in IOV-COM-202 trial. Cohort 3D results will inform a registrational and confirmatory trial design in frontline advanced NSCLC.
Lifileucel in Endometrial Cancer
Iovance is actively enrolling in the IOV-END-201 Phase 2 trial which is investigating lifileucel for advanced endometrial cancer patients who have progressed after platinum-based chemotherapy and anti-PD-1 therapy regardless of mismatch repair (MMR) status.
IOV-END-201 is supported by preclinical and manufacturing success data, as well as positive feedback from gynecological oncology experts. Initial results from IOV-END-201 are expected in the second half of 2025. There are no currently approved therapies for endometrial cancer following frontline post-anti-PD-1 therapy and chemotherapy, representing a significant opportunity for TIL cell therapy to address an additional unmet medical need in the post-anti-PD-1 treatment setting.

Next Generation TIL Pipeline

PD-1 Inactivated TIL Cell Therapy (IOV-4001): The Phase 2 efficacy portion of the IOV-GM1-201 trial in previously treated advanced melanoma and NSCLC continues to enroll rapidly. Iovance utilizes the TALEN technology licensed from Cellectis to develop other investigational gene-edited TIL cell therapies with multiple knockout targets to potentially improve efficacy.
Next Generation Interleukin-2 (IL-2) for TIL Treatment Regimen: A Phase 1/2 clinical trial was initiated to investigate IOV-3001, a second-generation, modified IL-2 analog for use in the TIL therapy treatment regimen. Non-human primate and IND-enabling studies of IOV-3001 demonstrated the potential for improved safety with strong effector T cell expansion.
Next Generation, Cytokine-Tethered TIL Therapy: IND-enabling studies are proceeding for IOV-5001, a genetically engineered, inducible, and tethered interleukin-12 (IL-12) TIL cell therapy. A clinical trial of a prior generation IL-12 TIL therapy at the National Cancer Institute showed improved efficacy with low cell doses, without the use of IL-2, and provides the rationale for modifications in IOV-5001 to enhance TIL efficacy while optimizing safety. In preclinical studies, IOV-5001 drove superior antitumor activity in a simulated tumor microenvironment. Iovance plans to submit an IND in 2025 to support clinical development for multiple indications.
Manufacturing Capacity Expansion

The iCTC, and an FDA-approved legacy contract manufacturer, currently have capacity to treat several thousand patients annually.
The company completed annual scheduled maintenance at the iCTC and successfully restarted full production.
Expansion is currently underway for the iCTC campus to supply TIL cell therapies to more than 5,000 patients annually in the next few years.
Iovance is also developing a manufacturing network to address more than 10,000 patients annually.
Corporate Updates

Dan Kirby joined Iovance’s Executive Leadership Team in the newly created role of Chief Commercial Officer in February 2025.
Raj Puri, M.D., Ph.D., was promoted to the newly created role of Chief Regulatory Officer in November 2024.
Iovance currently owns more than 250 granted or allowed U.S. and international patents and patent rights for Amtagvi and other TIL-related technologies that are expected to provide Amtagvi with exclusivity through at least 2042. This patent portfolio covers TIL compositions and methods of treatment and manufacturing in a broad range of cancers, with Gen 2 patent rights expected to provide exclusivity for Amtagvi into 2038 and additional patent rights, including methods of treating melanoma and compositions and methods for potency assays, expected to provide exclusivity into 2039 and 2042, respectively. Iovance also owns an industry-leading patent portfolio covering TIL products produced with genetic engineering, using core biopsies and peripheral blood as starting material, and using combinations of TIL products with checkpoint inhibitors, as well as Iovance’s proprietary IovanceCares system. More information on Iovance’s patent portfolio is available on the Intellectual Property page on www.iovance.com.
Fourth Quarter and Full Year 2024 Financial Results

As of February 26, 2025, Iovance’s cash position is approximately $422 million. The current cash position and anticipated product revenue are expected to be sufficient to fund current and planned operations into the second half of 2026.

Net loss for the fourth quarter of 2024 was $78.6 million, or $0.26 per share, compared to a net loss of $116.4 million, or $0.45 per share, for the fourth quarter of 2023. Net loss for the full year 2024 was $372.2 million, or $1.28 per share, compared to a net loss of $444.0 million, or $1.89 per share, for the full year 2023.

Revenue was $73.7 million for the fourth quarter of 2024 and consisted of product revenue from Amtagvi and Proleukin sales. Iovance recognized $48.7 million in revenue from Amtagvi infusions that were completed during the fourth quarter of 2024 and $25.0 million in global revenue for Proleukin. An additional $0.5 million in cash was received in the fourth quarter of 2024 for Amtagvi sales that will be recognized as revenue in the first quarter of 2025. Revenue for the fourth quarter of 2023 was $0.5 million for global sales of Proleukin.

Revenue for the full year 2024 was $164.1 million and reflected product revenue of $103.6 million from Amtagvi and $60.5 million from Proleukin. Revenue for the prior full year period 2023 was $1.2 million for global sales of Proleukin which Iovance began to recognize during the three-month period ended June 30, 2023.

The increases in revenue in the fourth quarter and full year 2024 over the prior year periods were primarily attributable to the U.S. launch of Amtagvi, including revenue recognized for Amtagvi, as well as significant growth in U.S. Proleukin revenue for use in the Amtagvi treatment regimen and global Proleukin sales.

Cost of sales includes inventory, overhead and related cash and non-cash expenses that are directly associated with sales of Amtagvi and Proleukin, as well as manufacturing costs for Amtagvi. Cost of sales for the three months ended December 31, 2024 was $45.5 million, primarily attributed to $9.1 million in period costs associated with patient drop off and manufacturing success rates, $5.9 million for non-cash amortization expense for intangible assets and fair value mark up of inventory, and $6.0 million in royalties payable on product sales. Cost of sales for the three months ended December 31, 2023 was $4.4 million, primarily related to non-cash amortization for intangible assets.

Cost of sales for the full year 2024 was $124.0 million, primarily related to $26.3 million in certain costs associated with patient drop off and manufacturing success rates, $26.2 million in non-cash amortization expense for intangible assets and fair value mark-up of inventory, and $14.2 million royalties payable on product sales. Cost of sales for the full year 2023 was $10.8 million, primarily related to non-cash amortization for intangible assets.

Increases in cost of sales in the fourth quarter and full year 2024 over the prior year periods were primarily attributable to the initiation of product sales, commercial manufacturing, and related cash and non-cash expenses tied to the U.S. launch of Amtagvi that began during the first quarter of 2024.

Research and development expenses were $72.2 million for the fourth quarter of 2024, a decrease of $15.3 million compared to $87.5 million for the fourth quarter of 2023. Research and development expenses were $282.3 million for the full year 2024, a decrease of $61.8 million compared to $344.1 million for the full year 2023.

The decreases in research and development expenses in the fourth quarter over the prior year period were primarily attributable to the transition of Amtagvi to commercial manufacturing. This decrease was partially offset by increases in headcount and related costs, including stock-based compensation, and clinical trial costs. The decrease in research and development expenses in the full year 2024 over the prior full year period was primarily attributable to the transition of Amtagvi to commercial manufacturing and lower clinical costs. These decreases were partially offset by increases in headcount and related costs, including stock-based compensation and lab and consumable costs.

Selling, general and administrative expenses were $42.5 million for the fourth quarter of 2024, an increase of $12.6 million compared to $29.9 million for the same period ended December 31, 2023. Selling, general and administrative expenses were $153.0 million for the full year 2024, an increase of $46.1 million compared to $106.9 million for the prior full year period.

The increase in selling, general and administrative expenses in the fourth quarter and full year 2024 compared to the prior year periods was primarily attributable to increases in headcount and related costs, including stock-based compensation, to support the growth in the overall business and related corporate infrastructure, as well as legal costs and costs incurred to support the commercialization of Amtagvi and Proleukin.

For additional information, please see the Company’s Selected Consolidated Balance Sheets and Statements of Operations below.

Webcast and Conference Call

Management will host a conference call and live audio webcast to discuss these results and provide a corporate update today at 4:30 p.m. ET. To listen to the live or archived audio webcast, please register at View Source The live and archived webcast can be accessed in the Investors section of the Company’s website, IR.Iovance.com, for one year.