On March 27, 2025 Arbutus Biopharma Corporation (Nasdaq: ABUS) ("Arbutus" or the "Company"), a clinical-stage biopharmaceutical company focused on infectious disease, reported fourth quarter and year end 2024 financial results and provided a corporate update (Press release, Arbutus Biopharma, MAR 27, 2025, View Source [SID1234651518]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"With my recent appointment as CEO of Arbutus, I am excited to lead the company into its next chapter," said Lindsay Androski, President and CEO of Arbutus. "Along with Arbutus’ new directors, my focus is on evaluating strategies to accelerate the development and potential approval of imdusiran, alongside several subject matter experts being retained to assist with this evaluation. As part of this review, we have implemented a reduction in our workforce of 57%, retaining a core team well-positioned to advance imdusiran into a Phase 2b trial, and have taken additional related steps to improve our financial and operational efficiency. We will provide an update on our pipeline and development timeline upon completion of our review of our chronic hepatitis B virus (cHBV) programs."

Ms. Androski continued, "I am also excited to welcome Tuan Nguyen to our leadership team as Chief Financial Officer. Tuan’s appointment reinforces our renewed focus on advancing our pipeline efficiently and delivering value to patients and our stakeholders. In addition, the Company announced today the departure of David Hastings, Karen Sims and Christopher Naftzger, and I would like to thank them for their valuable contributions over the past years.

We will continue to consult closely with and support our exclusive licensee Genevant Sciences to protect and defend our intellectual property, including through recently filed international lawsuits against Moderna for patent infringement in the development of its COVID-19 and RSV vaccines. We expect 2025 to be a transformative year for Arbutus."

2024 Clinical Development Milestones

Imdusiran (AB-729)

At the American Association for the Study of Liver Diseases (AASLD) – The Liver Meeting in November 2024, the Company presented new data from its IM-PROVE I Phase 2a clinical trial showing that six doses of imdusiran and 24 weeks of pegylated interferon alfa-2α (IFN), a standard-of-care immunomodulator, added to ongoing nucleos(t)ide analogue (NA) therapy led to a functional cure rate of 50% (3/6) in HBeAg-negative patients with baseline HBsAg levels less than 1000 IU/mL, and an overall functional cure rate of 25% (3/12). Those patients that achieved a functional cure also seroconverted with high anti-HBs antibody levels. The data from this trial indicated that the combination of imdusiran, IFN and NA therapy was generally safe and well-tolerated.
The Company also presented data from its IM-PROVE II Phase 2a clinical trial at AASLD showing that the addition of low dose nivolumab increased rates of HBsAg loss in cHBV patients that were first treated with imdusiran, ongoing NA therapy and Barinthus Biotherapeutics’ VTP-300, an HBV antigen-specific immunotherapy. In this clinical trial, 23% (3/13) of patients that received imdusiran, VTP-300, NA therapy and nivolumab achieved HBsAg loss by week 48. The Company is evaluating functional cure in these patients.
The Company is reviewing development plans for a Phase 2b clinical trial of imdusiran, including potential ways to accelerate the development timeline. To assist with its review, the Company is currently retaining experts in virology, hepatitis B, and in the clinical development and approval of antiviral treatments. The Company will provide a further update once its review is complete.
AB-101 (oral PD-L1 inhibitor)

AB-101-001 is a Phase 1a/1b double-blind, randomized, placebo-controlled clinical trial designed to investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single- and multiple-ascending doses of AB-101, the Company’s oral PD-L1 inhibitor, in healthy subjects and patients with cHBV.
Based on data from Part 2 of this clinical trial reported in November 2024 showing that AB-101 was generally well-tolerated with evidence of dose-dependent receptor occupancy in healthy subjects, Arbutus has moved into Part 3 which evaluates repeat doses of AB-101 for 28 days in patients with cHBV. Next steps for AB-101 will be determined upon completion of the Company’s review of its cHBV programs.
LNP Litigation

Arbutus will continue to consult closely with and support our exclusive licensee Genevant Sciences to protect and defend Arbutus’s intellectual property, which is the subject of on-going lawsuits against Moderna and Pfizer/BioNTech. The Company, together with Genevant, is seeking fair compensation for Moderna’s and Pfizer/BioNTech’s use of Arbutus’s patented LNP technology that was developed with great effort and at a great expense, and without which Moderna’s and Pfizer/BioNTech’s COVID-19 vaccines would not have been successful.
The claim construction hearing for the lawsuit against Pfizer/BioNTech occurred in December 2024. The court is expected to provide its ruling on the claim construction and issue a further scheduling order in 2025.
The jury trial in the Moderna U.S. litigation is currently scheduled for September 2025. Expert discovery continues in this lawsuit. On March 3, 2025, the Company announced that alongside Genevant Sciences, it filed five international lawsuits seeking to enforce patents protecting their innovative LNP technology across 30 countries.
Corporate Updates

In March 2025, the Company’s Board took action to reduce the Company’s workforce by 57% resulting in a total workforce after reductions of 19 employees. The Board also decided to exit the Company’s corporate headquarters in Warminster, PA, and to discontinue in-house scientific research. In connection with these actions, the Company expects to incur a one-time restructuring charge in the first quarter of 2025 of approximately $11 million to $13 million.
Also in March 2025, the Board appointed Tuan Nguyen as Chief Financial Officer effective March 28, succeeding David C. Hastings. Additionally, the Company announced the departure of J. Christopher Naftzger as General Counsel and Chief Compliance Officer and Dr. Karen Sims as Chief Medical Officer.
Tuan Nguyen has almost two decades of experience in biopharma working on small molecules and AAV gene therapies. Most recently, he served as Chief Financial Officer of Kinevant Sciences, a subsidiary of Roivant Sciences, dedicated to treating rare inflammatory and autoimmune diseases with significant unmet need. Prior to that he held various senior finance leadership roles at Adverum, Intarcia Therapeutics, Fibrogen, and UCB. He has helped raise over $2 billion in dilutive and non-dilutive capital. Mr. Nguyen earned his MBA with dual concentrations in Finance and Entrepreneurship, Innovation, & Change from Emory University. Mr. Nguyen will report directly to Lindsay Androski, President and CEO.
Financial Results

Cash, Cash Equivalents and Investments

As of December 31, 2024, the Company had cash, cash equivalents and investments in marketable securities of $122.6 million compared to $132.3 million as of December 31, 2023. During the year ended December 31, 2024, the Company used $64.9 million in operating activities, which was partially offset by $44.1 million of net proceeds from the issuance of common shares under its "at-the-market" (ATM) offering program and $7.5 million of proceeds from the exercise of employee stock options. The Company expects to significantly reduce its net cash burn in 2025 when compared to 2024. Given the Company’s review of its pipeline and development plans for its cHBV programs and refocused operations, the Company has terminated its ATM offering program.

Revenue

Total revenue was $6.2 million for the year ended December 31, 2024, compared to $18.1 million for the same period in 2023. The decrease of $11.9 million was due primarily to a $9.3 million decrease in revenue recognition of the upfront license fee received in 2022 from Qilu, the Company’s collaboration partner in China, Hong Kong, Macau and Taiwan, as less effort was required from the Company in 2024 compared to 2023 to support Qilu’s progress towards achieving its own imdusiran manufacturing capability. Additionally, license royalty revenues decreased $2.6 million in 2024 compared to 2023 due to a decrease in Alnylam’s sales of ONPATTRO.

Operating Expenses

Research and development expenses were $54.0 million for the year ended December 31, 2024 compared to $73.7 million for the same period in 2023. The decrease of $19.7 million was due primarily to: i) a decrease in clinical expenses related to the discontinuation of the Company’s coronavirus and AB-161 programs during the fourth quarter of 2023; ii) a decrease in research activities and preclinical study costs for AB-101 which is now in a Phase 1a/1b clinical trial; and iii) cost savings from the Company’s decision in August 2024 to streamline the organization to focus its efforts on advancing the clinical development of imdusiran and AB-101, which included ceasing all discovery efforts, discontinuing its IM-PROVE III clinical trial and reducing its workforce by 40%. These actions in August 2024 resulted in the Company incurring a one-time restructuring charge of $3.7 million in the third quarter of 2024.

General and administrative expenses were $22.1 million for the year ended December 31, 2024, compared to $22.5 million for the same period in 2023. This decrease was due primarily to decreases in employee compensation-related expenses, partially offset by an increase in litigation-related legal fees.

Net Loss

For the year ended December 31, 2024, our net loss was $69.9 million, or a loss of $0.38 per basic and diluted common share, as compared to a net loss of $72.8 million, or a loss of $0.44 per basic and diluted common share, for the year ended December 31, 2023.

Outstanding Shares

As of December 31, 2024, the Company had 190.0 million common shares issued and outstanding, as well as 16.9 million stock options and unvested restricted stock units outstanding. Roivant Sciences Ltd. owned approximately 20% of the Company’s outstanding common shares as of December 31, 2024.

UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF LOSS
(in thousands, except share and per share data)

Year Ended December 31,
2024 2023
Revenue
Collaborations and licenses $ 3,919 $ 14,274
Non-cash royalty revenue 2,252 3,867
Total revenue 6,171 18,141
Operating expenses
Research and development 54,037 73,700
General and administrative 22,108 22,475
Change in fair value of contingent consideration 2,625 69
Restructuring costs 3,720 —
Total operating expenses 82,490 96,244
Loss from operations (76,319 ) (78,103 )
Other income (loss)
Interest income 6,585 5,688
Interest expense (137 ) (459 )
Foreign exchange gain (49 ) 25
Total other income 6,399 5,254
Net loss $ (69,920 ) $ (72,849 )
Net loss per common share
Basic and diluted $ (0.38 ) $ (0.44 )
Weighted average number of common shares
Basic and diluted 185,608,874 165,960,379

UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS
(in thousands)

December 31, 2024 December 31, 2023
Cash, cash equivalents and marketable securities, current $ 122,623 $ 126,003
Accounts receivable and other current assets 4,693 6,024
Total current assets 127,316 132,027
Property and equipment, net of accumulated depreciation 3,309 4,674
Investments in marketable securities, non-current — 6,284
Right of use asset 1,048 1,416
Other non-current assets 34 —
Total assets $ 131,707 $ 144,401

Accounts payable and accrued liabilities $ 7,564 $ 10,271
Deferred license revenue, current 7,571 11,791
Lease liability, current 483 425
Total current liabilities 15,618 22,487
Liability related to sale of future royalties 4,829 6,953
Deferred license revenue, non-current 2,863 —
Contingent consideration 10,225 7,600
Lease liability, non-current 806 1,343
Total stockholders’ equity 97,366 106,018
Total liabilities and stockholders’ equity $ 131,707 $ 144,401

UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
(in thousands)

Twelve Months Ended December 31,
2024
2023
Net loss $ (69,920 ) $ (72,849 )
Non-cash items 7,899 5,146
Change in deferred license revenue (1,357 ) (10,664 )
Other changes in working capital (1,472 ) (7,569 )
Net cash used in operating activities (64,850 ) (85,936 )
Net cash provided by investing activities 22,948 50,773
Issuance of common shares pursuant to the Open Market Sale Agreement 44,123 29,852
Cash provided by other financing activities 7,873 795
Net cash provided by financing activities 51,996 30,647
Effect of foreign exchange rate changes on cash and cash equivalents (49 ) 25
Increase/(decrease) in cash and cash equivalents 10,045 (4,491 )
Cash and cash equivalents, beginning of period 26,285 30,776
Cash and cash equivalents, end of period 36,330 26,285
Investments in marketable securities 86,293 106,002
Cash, cash equivalents and marketable securities, end of period $ 122,623 $ 132,287

About Imdusiran (AB-729) 

Imdusiran is an RNAi therapeutic specifically designed to reduce all HBV viral proteins and antigens including hepatitis B surface antigen, which is thought to be a key prerequisite to enable reawakening of a patient’s immune system to respond to the virus. Imdusiran targets hepatocytes using Arbutus’ novel covalently conjugated N-Acetylgalactosamine (GalNAc) delivery technology enabling subcutaneous delivery. In a Phase 2a clinical trial, imdusiran achieved meaningful functional cure rates in patients with cHBV when combined with pegylated interferon (IFN) alfa-2α and nucleos(t)ide analogue (NA) therapy. Clinical data generated thus far has shown imdusiran to be generally safe and well-tolerated, while also providing meaningful reductions in hepatitis B surface antigen and hepatitis B DNA. The Company is currently reevaluating plans for a Phase 2b clinical trial of imdusiran combined with IFN and NA therapy.

About AB-101 

AB-101 is an oral PD-L1 inhibitor candidate that is designed to allow for controlled checkpoint blockade while minimizing the systemic safety issues typically seen with checkpoint antibody therapies. Immune checkpoints such as PD-1/PD-L1 play an important role in the induction and maintenance of immune tolerance and in T-cell activation. Preclinical data generated thus far indicates that AB-101 mediates re-activation of exhausted HBV-specific T-cells from cHBV patients. AB-101 is currently being evaluated in a Phase 1a/1b clinical trial.

About HBV 

Hepatitis B is a potentially life-threatening liver infection caused by the hepatitis B virus (HBV). HBV can cause chronic infection which leads to a higher risk of death from cirrhosis and liver cancer. Chronic HBV infection represents a significant unmet medical need. The World Health Organization estimates that over 250 million people worldwide suffer from chronic HBV infection, while other estimates indicate that approximately 2 million people in the United States suffer from chronic HBV infection. Approximately 1.1 million people die every year from complications related to chronic HBV infection despite the availability of effective vaccines and current treatment options.

On March 27, 2024 AIM ImmunoTech Inc. (NYSE American: AIM) ("AIM" or the "Company") reported its financial results for the fourth quarter and full year 2024 and provided a business update (Press release, AIM ImmunoTech, MAR 27, 2025, View Source [SID1234651517]). The Company will host a conference call and webcast on Tuesday, April 1, 2025 at 8:30 AM ET (details below).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Our team made significant strides in advancing our development programs along with our collaborators over the course of 2024. As we look ahead, we will continue to execute our corporate and clinical strategies to further establish the foundation upon which we can secure the successful future of AIM and our drug Ampligen for the treatment of major-market unmet medical needs. We are focused on the successful execution of a number of key milestones expected over the course of the next 18 months with the goal of building long-term stockholder value," commented AIM Chief Executive Officer Thomas K. Equels.

Recent Highlights

Announced plans to conduct clinical study of the combination of Ampligen and AstraZeneca’s FluMist as an intranasal vaccine for influenza, including avian influenza (bird flu); Paul Goepfert, MD, of the University of Alabama-Birmingham, has agreed to act as the Principal Investigator for the study.
Announced Ted D. Kellner and David Chemerow as new members of AIM’s Board of Directors.
Erasmus Medical Center Safety Committee granted approval to proceed with the Phase 2 portion of the Phase 1b/2 clinical trial involving AIM’s Ampligen and AstraZeneca’s anti-PD-L1 immune checkpoint inhibitor Imfinzi (durvalumab) in the treatment of late-stage pancreatic cancer ("DURIPANC").
Commenced dosing in Phase 2 of the Phase 1b/2 DURIPANC clinical trial for treatment of late-stage pancreatic cancer.
Announced the final clinical study results for the "Study to Evaluate the Efficacy and Safety of Ampligen in Patients With Post-COVID Conditions" ("AMP-518") were published to ClinicalTrials.gov (See: NCT05592418).
Expanded patent portfolio with a U.S. compositions and methods patent for Ampligen for the treatment of endometriosis and a Netherlands compositions and methods patent covering Ampligen for use in the Post-COVID condition of fatigue.
Announced publication of data from Roswell Park Comprehensive Cancer Center’s Phase 1 study evaluating a combination therapy using Ampligen in early-stage triple-negative breast cancer (TNBC) in The Journal for ImmunoTherapy of Cancer.
Expected Upcoming, Value-Driving Milestones

Metastatic Pancreatic Ductal Adenocarcinoma

Phase 1b/2 Combining Anti-PD-L1 Immune Checkpoint Inhibitor Durvalumab with TLR-3 Agonist Ampligen in Patients with Metastatic Pancreatic Ductal Adenocarcinoma for Therapy Efficacy (DURIPANC) (NCT05927142); Funded through collaboration of AstraZeneca and Erasmus Medical Center

Q2/Q3 2026: Last patient enrolled in Phase 2
Refractory Melanoma

Phase 2 Polarized Dendritic Cell (aDC1) Based Treatment, Interferon Alpha-2, Ampligen, and Celecoxib for the Treatment of HLA-A2+ Refractory Melanoma (NCT04093323); Grant funded by National Cancer Institute

H1 2025: First patient dosed
Stage 4 Triple Negative Breast Cancer

Phase 1/2a Study of Ampligen, Celecoxib and Interferon Alpha 2b with Pembrolizumab for the Treatment of Patients with Metastatic or Unresectable Triple Negative Breast Cancer (NCT05756166); Grant funded by Merck and National Cancer Institute

Q2 2026: Expected completion of enrollment
Advanced Recurrent Ovarian Cancer

Phase 2 Systemic Immune Checkpoint Blockade and Intraperitoneal Chemo-Immunotherapy in Recurrent Ovarian Cancer (NCT03734692); Grant funded by Merck

H1 2025: Completion of study, publication of data
Summary of Financial Highlights for Fiscal Year 2024

As of December 31, 2024, AIM reported cash, cash equivalents and marketable investments of $4.0 million, compared to $13.1 million as of December 31, 2023.
Research and development expenses for the year ended December 31, 2024, were $6.2 million, compared to $10.9 million for the year ended December 31, 2023.
General and administrative expenses for the year ended December 31, 2024, were $13.7 million, compared to $21.1 million for the year ended December 31, 2023.
Please refer to the full 10-K for complete details.

Conference Call and Webcast Details

The Company will host a conference call and webcast to discuss the Company’s Q4/FY2024 operational and financial results on Tuesday, April 1, 2025 at 8:30 AM ET.

The call will be hosted by Thomas K. Equels, Chief Executive Officer of AIM. Interested participants and investors may access the conference call by dialing (877) 407-9219 (domestic) or (201) 689-8852 (international) and referencing the AIM ImmunoTech Conference Call. The webcast will be accessible on the Events page of the Investors section of the Company’s website, aimimmuno.com, and will be archived for 90 days following the live event.

On March 27, 2025 ADC Therapeutics SA (NYSE: ADCT), a commercial-stage global leader and pioneer in the field of antibody drug conjugates (ADCs), reported financial results for the fourth quarter and full year ended December 31, 2024, and provided recent operational updates (Press release, ADC Therapeutics, MAR 27, 2025, View Source [SID1234651516]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We achieved several key milestones in 2024, advancing our expansion trials with ZYNLONTA in combinations and in earlier lines of DLBCL therapy, progressing our early research solid tumor program to the IND-enabling stage and reducing operational spend while at the same time strengthening the balance sheet," said Ameet Mallik, Chief Executive Officer of ADC Therapeutics. "We closed the year by fully enrolling our confirmatory LOTIS-5 DLBCL study, reported encouraging initial data from our LOTIS-7 DLBCL study and were pleased to see promising Phase 2 IIT data reported at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting evaluating ZYNLONTA in indolent lymphomas. We are confident in our path forward and believe we are well positioned for success as we progress toward additional pivotal milestones in 2025."

Fourth Quarter 2024 Operational Updates and Upcoming Milestones

•Full enrollment achieved in LOTIS-5. Enrollment for the Phase 3 confirmatory trial evaluating ZYNLONTA in combination with rituximab in patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) was completed in December 2024. The Company expects to provide updated data before the end of 2025, once the pre-specified number of progression-free survival (PFS) events is reached.
•Encouraging initial data from LOTIS-7. The Company reported positive initial data in December 2024 from the LOTIS-7 Phase 1b open-label clinical trial evaluating the safety and efficacy of ZYNLONTA in combination with the bispecific antibody glofitamab (COLUMVI) in patients with r/r non-Hodgkin Lymphoma (NHL). The best overall response rate (ORR) among the 18 r/r DLBCL efficacy evaluable patients was 94%, and the complete response rate (CRR) was 72%. These encouraging efficacy data were observed across patients with different numbers of lines and types of prior treatments. Initial safety data on all 29 r/r NHL patients suggest the combination is generally well tolerated with no dose-limiting toxicities across all dose levels. Enrollment of 40 patients in the dose expansion is expected to be completed in the second quarter of 2025. We expect to share data on a subset of patients in the second quarter of 2025 with a fuller, more mature data update anticipated during the second half of 2025.
•Promising data from the Phase 2 investigator-initiated trials evaluating ZYNLONTA in indolent lymphomas. Updated data from the investigator-initiated trials (IITs) of ZYNLONTA were presented at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2024. Both the Phase 2 clinical trial evaluating ZYNLONTA in combination with rituximab in patients with r/r follicular lymphoma (FL) and the Phase 2 clinical trial evaluating ZYNLONTA for the treatment of r/r marginal zone lymphoma (MZL) are ongoing and being conducted at the Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine. Results from both trials as presented at ASH (Free ASH Whitepaper) and the FL trial simultaneously published in Lancet Haematology can be found here. Additional data are expected to be shared at a medical conference and/or in publication with plans to engage regulatory agencies and evaluate compendia strategies.
•Abstracts to be presented in oral and poster presentations in April at AACR (Free AACR Whitepaper) 2025. An abstract on the Company’s Claudin-6 targeting ADC was accepted for oral presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025. Abstracts on the Company’s PSMA and ASCT2-targeting ADCs were also accepted for poster presentations at the meeting.

Fourth Quarter and Full Year 2024 Financial Results

•Product Revenues: ZYNLONTA reached commercial brand profitability in 2024, generating net product revenues of $16.4 million for the fourth quarter ended December 31, 2024, and $69.3 million for the full year of 2024 as compared to $16.6 million and $69.1 million for the same periods in 2023. The quarter-over-quarter decrease is driven by lower sales volume, partially offset by a higher selling price. The year-to-date increase is primarily attributable to a higher selling price and favorability in prior period GTN sales adjustments, partially offset by lower sales volume.

•Research and Development (R&D) Expense: R&D expense was $27.1 million and $109.6 million for the fourth quarter and full year ended December 31, 2024, respectively. This compares to R&D expense of $30.3 million and $127.1 million for the same periods in 2023. The decrease during both periods is due primarily to the implementation of productivity initiatives and focused investment in prioritized development programs.

•Selling and Marketing (S&M) Expense: S&M expense was $11.3 million and $44.0 million for the fourth quarter and full year ended December 31, 2024, respectively. This compares to S&M expense of $13.9 million and $57.5 million for the same periods in 2023. The quarter-over-quarter decrease in S&M expense was primarily due to lower marketing and advertising costs, partially offset by higher share-based compensation expense. The year-to-date decrease was primarily due to lower marketing and advertising costs as well as lower wages and benefits.

•General & Administrative (G&A) Expense: G&A expense was $9.6 million and $41.9 million for the fourth quarter and full year ended December 31, 2024, respectively. This compares to G&A expense of $11.3 million and $48.4 million for the same periods in 2023. The quarter-over-quarter decrease in G&A expense was primarily related to lower professional fees. The year-to-date decrease was primarily related to lower share-based compensation expense, professional fees and insurance premiums.

•Net Loss: Net loss for the quarter ended December 31, 2024, was $30.7 million, or a net loss of $0.29 per basic and diluted share, as compared to net loss of $85.0 million, or a net loss of $1.03 per basic and diluted share for the same period in 2023. Net loss for the full year ended December 31, 2024, was $157.8 million, or a net loss of $1.62 per basic and diluted share, as compared to net loss of $240.1 million, or a net loss of $2.94 per basic and diluted share for the full year ended December 31, 2023. The decrease in net loss during both periods is primarily attributable to lower income tax expense and lower operating expenses.

•Adjusted Net Loss: Adjusted net loss, which is a non-GAAP financial measure, was $26.5 million, or an adjusted net loss of $0.25 per basic and diluted share for the quarter ended December 31, 2024, as compared to an adjusted net loss of $79.5 million, or $0.97 per basic and diluted share, for the same period in 2023. Adjusted net loss for the full year ended December 31, 2024, was $111.4 million, or an adjusted net loss of $1.15 per basic and diluted share, as compared to net loss of $185.7 million, or an adjusted net loss of $2.27 per basic and diluted share for the full year ended December 31, 2023. The decrease in adjusted net loss during both periods is primarily attributable to lower income tax expense and lower operating expenses.

•Cash and cash equivalents: As of December 31, 2024, cash and cash equivalents were $250.9 million, compared to $278.6 million as of December 31, 2023. In May 2024 the Company completed an underwritten offering resulting in net proceeds of approximately $97.4 million, extending the expected cash runway into the second half of 2026.

Conference Call Details

ADC Therapeutics management will host a conference call and live audio webcast to discuss fourth quarter and full year 2024 financial results and provide a company update today at 8:30 a.m. Eastern Time. To access the conference call, please register here. The participant toll-free dial-in number is 1-800-836-8184 for North America and Canada. A live webcast of the call will be available under "Events & Presentations" in the Investors section of the ADC Therapeutics website at ir.adctherapeutics.com. The archived webcast will be available for 30 days following the call.

About ZYNLONTA

ZYNLONTA is a CD19-directed antibody drug conjugate (ADC). Once bound to a CD19-expressing cell, ZYNLONTA is internalized by the cell, where enzymes release a pyrrolobenzodiazepine (PBD) payload. The potent payload binds to DNA minor groove with little distortion, remaining less visible to DNA repair mechanisms. This ultimately results in cell cycle arrest and tumor cell death.

The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have approved ZYNLONTA (loncastuximab tesirine-lpyl) for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (NOS), DLBCL arising from low-grade lymphoma and also high-grade B-cell lymphoma. The trial included a broad spectrum of heavily pre-treated patients (median three prior lines of therapy) with difficult-to-treat disease, including patients who did not respond to first-line therapy, patients refractory to all prior lines of therapy, patients with double/triple hit genetics and patients who had stem cell transplant and CAR-T therapy prior to their treatment with ZYNLONTA. This indication is approved by the FDA under accelerated approval and in the European Union under conditional approval based on overall response rate and continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Please see full prescribing information including important safety information about ZYNLONTA at www.ZYNLONTA.com.

ZYNLONTA is also being evaluated as a therapeutic option in combination studies in other B-cell malignancies and earlier lines of therapy.

On March 26, 2025 Bayer and Suzhou Puhe BioPharma Co.,Ltd., a clinical-stage biotechnology company, reported that they have entered into a global license agreement for Puhe BioPharma’s oral, small molecule PRMT5 inhibitor that selectively targets MTAP-deleted tumors (Press release, Bayer, MAR 26, 2025, View Source [SID1234655501]). Under the agreement, Bayer obtains an exclusive worldwide license to develop, manufacture and commercialize the MTA-cooperative PRMT5 inhibitor. Bayer has enrolled the first participant in a Phase I first-in-human dose escalation study investigating MTA-cooperative PRMT5 inhibitor under the development name BAY 3713372 for the treatment of MTAP-deleted solid tumors.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are looking forward to explore the potential of the PRMT5 inhibitor, which could improve outcomes for patients with MTAP-deleted tumors who often have a poor prognosis," said Juergen Eckhardt, M.D., Head of Business Development and Licensing at Bayer’s Pharmaceuticals Division. "The highly selective targeting of cancer cells while sparing healthy cells, based on the innovative mechanism of action is very promising. This will support our mission to build one of the most transformative and differentiated precision oncology pipelines in the industry."

"We see great potential in MTA-cooperative PRMT5 inhibitors in treating MTAP-deleted tumors. Our MTA-cooperative PRMT5 inhibitor, PH020, now named BAY 3713372, has demonstrated competitive selectivity for PRMT5 bound to MTA and activity in preclinical studies, as well as brain penetration capabilities," said Yongqi Guo, CEO of Puhe BioPharma. "We are excited to partner with Bayer, a global leader in the field of life sciences, to advance our PRMT5 inhibitor into the clinic. Together with Bayer, we look forward to bringing this therapeutic option to patients worldwide."

PRMT5 (protein arginine methyltransferase 5) and a specific gene called MTAP (metabolic enzyme 5’-deoxy-5′-methylthioadenosine phosphorylase) play important roles in cell metabolism and are critical for cell survival. MTAP deletions, which occur in approximately 10 to 30 percent of all cancers1, lead to elevated levels of MTA in tumor cells and BAY 3713372 is designed to bind the PRMT5-MTA complex thus specifically exploiting tumor vulnerability.

"Loss of the MTAP gene occurs in a variety of tumor types, including those with few treatment options and poor prognosis, such as pancreatic cancer and glioblastoma," said Dominik Ruettinger, M.D., Ph.D., Global Head of Research and Early Development for Oncology at Bayer’s Pharmaceuticals Division. "We look forward to advancing this program as we are driven by our ambition to develop innovative medicines that improve and extend the lives of cancer patients we serve, focusing on areas of high unmet medical need."

Financial terms are not disclosed.

About BAY 3713372 (MTA-cooperative PRMT5 inhibitor)
BAY 3713372 is an investigational agent and has not been approved by any health authority for use in any country, for any indication. It is an oral, potent selective MTA-cooperative PRMT5 inhibitor being evaluated as a potential new targeted treatment option for patients with MTAP-deleted solid tumors. BAY 3713372 has unique characteristics including brain penetrance, which allows targeting of central nervous system (CNS) metastases and primary brain tumors.

Protein arginine N-methyltransferase 5 (PRMT5) plays a crucial role in modifying proteins that control the cell cycle. The metabolic enzyme 5’-deoxy-5′-methylthioadenosine phosphorylase (MTAP) is involved in the methionine salvage pathway which recycles methionine from methylthioadenosine (MTA).This targeted approach takes advantage of the unique relationship between MTA and PRMT5, creating a specific vulnerability that can be exploited to effectively treat MTAP deficient cancer cells.

About the clinical phase I study
BAY 3713372 is investigated in a global Phase I first-in-human dose escalation study in patients with MTAP-deleted solid tumors. The clinical program aims to evaluate the safety, tolerability and pharmacokinetics, pharmacodynamics and preliminary clinical activity of BAY 3713372, a novel cancer cell specific PRMT5 inhibitor.

On March 26, 2024 Alphamab Oncology reported financial results for the full year ended December 31, 2024 and highlighted recent business progress (Press release, Alphamab, MAR 26, 2025, View Source,amounted%20to%20RMB%20159.46%20million. [SID1234654359]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Financial Summary

● For the year ended December 31, 2024, we recorded total revenue of RMB 640.08 million, a 192.58% year-on-year increase. Meanwhile, product revenue (attributed to the Company) amounted to RMB 159.46 million.

● For the year ended December 31, 2024, our R&D expenditure amounted to RMB 404.15 million, basically unchanged as compared with prior year.

● For the year ended December 31, 2024, we reached profitability for the first time on an annual basis, recording profit for the year of RMB 166.34 million.

● We have a healthy financial position, with cash reserves of RMB 1,571.47 million as of December 31, 2024.

Business Highlights

With multiple proprietary platforms, the Company has established a globally competitive and differentiated pipeline that includes antibody-drug conjugation (ADC), single domain antibody, and bispecific antibodies. Envafolimab, the world’s first subcutaneously injectable PD-(L)1 inhibitor, was approved by Chinese authorities in 2021, providing a safer and more convenient tumor immunotherapy for patients. Multiple assets are in phase III or pivotal clinical trials, and several other bispecific ADC new drug candidates are in preclinical development.

Commercial Product

KN035 (Envafolimab)

KN035, an innovative anti-tumor immunotherapy drug, is the first subcutaneously injectable PD-(L)1 inhibitor worldwide, the first immunotherapy drug aimed at cross-tumor indications in China and the first domestically produced PD-L1 drug. KN035 offers advantages in effectiveness, safety, convenience and compliance, particularly suitable for frail, elderly patients and those with adverse reactions to intravenous infusions, while significantly reducing the use of healthcare resources.

Events during the Reporting Period

● In January 2024, Envafolimab obtained the market approval by the Pharmaceutical Administration Bureau of the Macau Special Administrative Region of the People’s Republic of China for the treatment of non-metastatic advanced microsatellite instability-high (MSI-H) or non-mismatch-repair deficiency (dMMR) advanced solid tumors.

● In January 2024, Alphamab Oncology and 3D Medicines entered into a license agreement with Glenmark Pharmaceuticals Ltd. (Glenmark) for the subcutaneous injection PD-L1 antibody drug KN035, pursuant to which, Glenmark was granted exclusive licensing interests in clinical development and commercialization of oncology indications of KN035 (the "Field") in India, Asia Pacific (except Singapore, Thailand, Malaysia), the Middle East and Africa, Russia, Commonwealth of Independent States and Latin America (the "Territory"). Glenmark shall bear its own costs and expenses related to the development and commercialization of KN035 in the Field in the Territory. Jiangsu Alphamab retains the exclusive right to produce KN035 for any purpose either inside or outside the Territory.

● In March 2024, Envafolimab was included in the 2024 edition of the "Chinese Expert Consensus on the Use of Immune Checkpoint Inhibitors in Perioperative Treatment of Advanced Gastric Cancer" published by the Gastric Cancer Committee of the Chinese Anti-Cancer Association. Envafolimab has been highly recommended by 16 latest domestic authoritative guidelines and consensus recommendations in 2024.

● In August 2024, Envafolimab was granted breakthrough therapy designation by the Center for Drug Evaluation (CDE) of the National Medical Products Administration of China (NMPA) for the treatment of patients with unresectable or metastatic solid tumors with high tumor mutation burden (TMB-H) who have failed prior standard treatment and no satisfactory alternative treatment.

● In September 2024, the supplementary application for production site, scale and process changes of KN035 successfully passed the GMP compliance inspection, and we received Pharmaceutical GMP Compliance Inspection Certificate in December 2024.

Clinical Product Pipeline

KN026

KN026 is a HER2 heterodimeric bispecific antibody (BsAb) that can simultaneously bind two non-overlapping epitopes of HER2, leading to HER2 signal blockade. KN026 has demonstrated better tumor inhibition in HER2-positive tumor cell lines compared with Trastuzumab and Pertuzumab in combination. Additionally, KN026 has also shown inhibitory effect on tumor cells with Trastuzumab-resistant cell lines. The results of multiple clinical studies in different stages showed that KN026 has significant anti-tumor activities, even in heavily pretreated patients with HER2-positive BC and GC, including those with prior anti-HER2 treatment. KN026 in combination with chemotherapy for the treatment of patients with HER2-positive GC (including GEJ) who have failed first-line standard treatment was granted breakthrough therapy designation by the Center for Drug Evaluation (CDE) of the National Medical Products Administration of China (NMPA).

Events during the Reporting Period

● The phase III clinical trial of KN026 combined with chemotherapy as second-line and later-line treatment of HER2-positive GC/GEJ is undergoing smoothly.

● The phase III clinical trial of KN026 combined with albumin-bound Docetaxel HB1801 as first-line treatment of HER2-positive recurrent and metastatic BC is undergoing smoothly.

● In October 2024, a phase III clinical trial of KN026 in combination with albumin-bound Docetaxel HB1801 was approved by CDE as neoadjuvant treatment for HER2-positive early or locally advanced BC, and the first patient was successfully dosed in December 2024.

Events after the Reporting Period

● In January 2025, the results of phase II clinical trial of KN026 in combination with docetaxel as first-line treatment for HER2-positive recurrent or metastatic BC were published in Cancer Communications.

● In March 2025, the results of phase II clinical trial of KN026 in combination with KN046 for the treatment of advanced HER2-positive solid tumors (excluding BC) were published in Signal Transduction and Targeted Therapy.

JSKN003

JSKN003 is a bispecific ADC developed based on KN026 using the proprietary Glycan-specific conjugation platform. JSKN003 can bind HER2 on the surface of tumor cells and release topoisomerase I inhibitors (TOPIi) through cellular endocytosis, thereby exerting anti-tumor effects. Compared with its ADC counterparts, JSKN003 demonstrated better serum stability and stronger bystander effect, which effectively expands the therapeutic window. Results of multiple clinical studies at various stages of JSKN003 in China and Australia have demonstrated favorable safety profile, with promising efficacy of JSKN003 in heavily pretreated patients with advanced solid tumors, especially in patients with platinum-resistant ovarian cancer (PROC), HER2-expressing BC, or high HER2-expressing solid tumors.

Events during the Reporting Period

● In April 2024, the results of the dose-escalation stage of the phase I clinical trial of JSKN003 conducted in Australia were presented at the 2024 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, which demonstrated favorable tolerability and safety profile of JSKN003 in patients with advanced/metastatic solid tumors who received prior multi-line treatment. The occurrence of hematologic toxicity was very low among common treatment-related adverse events (TRAEs) of all grades and no TRAE led to death or treatment discontinuation. The results also demonstrated encouraging preliminary anti-tumor activity.

● In June 2024, data from a phase I clinical trial of JSKN003 conducted in China were presented for the first time at the 2024 Annual Meeting of American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper). The results showed that the ORR was 51.1% in all efficacy evaluable patients across HER2 low and HER2 positive populations, of which 28 patients who received prior anti-HER2 the ORR was 57.1%, and 21 patients who received prior anti-HER2 ADC the ORR was 57.1%. The results still showed efficacy signals in patients with previous anti-HER2 ADC treatment.

● In September 2024, clinical data from two studies on JSKN003 for the treatment of PROC and HER2-positive (IHC 3+) advanced solid tumors (excluding BC) were presented at the 2024 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress. The results have demonstrated favorable tolerability and safety profile, with promising efficacy of JSKN003 in heavily pretreated patients with PROC or HER2-positive solid tumors.

● In September 2024, the Company entered a licensing agreement with JMT-Bio Technology Co., Ltd. (JMT-Bio), a wholly-owned subsidiary of CSPC Pharmaceutical Group Co., Ltd. (CSPC) (stock code: 1093.HK), pursuant to which, JMT-Bio was granted the exclusive license and sublicense rights to develop, sell, offer for sale and commercialize JSKN003, for the treatment of tumor-related indications (the "Field") in mainland China (excluding Hong Kong, Macau or Taiwan) (the "Territory") and become the sole marketing authorization holder for JSKN003 for the Field in the Territory. Alphamab retains the sole right to supply JSKN003. According to the licensing agreement, Alphamab is entitled to receive upfront payment and milestone payments of up to RMB3.08 billion in total, and a double-digit percentage of royalties on net product sales of JSKN003.

● In December 2024, JSKN003 received approval from CDE to initiate a phase III clinical trial to compare the efficacy of JSKN003 versus investigator-selected chemotherapy for the treatment of platinum-resistant recurrent epithelial ovarian cancer, primary peritoneal cancer, or fallopian tube cancer, and the first patient was successfully dosed in February 2025.

● The phase III clinical trial of JSKN003 for the treatment of unresectable locally advanced or metastatic HER2-low expressing BC is undergoing smoothly.

● Multiple exploratory phase II clinical studies of JSKN003 are currently being conducted.

Events after the Reporting Period

● In February 2025, JSKN003 received approval from CDE to initiate a phase III clinical trial to compare the efficacy and safety of JSKN003 versus trastuzumab emtansine (T-DM1) for the treatment of HER2-positive advanced BC, and the first patient was successfully dosed in the same month.

● In March 2025, JSKN003 was granted breakthrough therapy designation by CDE. The designation is for the treatment of platinum-resistant recurrent epithelial ovarian cancer, primary peritoneal cancer, or fallopian tube cancer, not restricted to HER2 expression levels.

JSKN016

JSKN016 is a bispecific ADC simultaneously targeting HER3 (Human epidermal growth factor receptor 3) and TROP2 (Trophoblast cell surface antigen 2), which is developed with proprietary single-domain antibody platform and Glycan-specific conjugation platform. TROP2 and HER3 are overexpressed in multiple solid tumors. JSKN016 exerts tumor cell-killing activity through dual binding to both antigens, coupled with endocytosis and bystander effects, demonstrating superior efficacy compared to monospecific TROP2 or HER3 ADCs. Furthermore, JSKN016 shows enhanced ability to overcome drug resistance caused by tumor heterogeneity.

Events during the Reporting Period

● In March 2024, JSKN016 received approval from CDE to initiate a phase I clinical trial for the treatment of advanced malignant solid tumors and the first patient was successfully dosed in May 2024. JSKN016 has demonstrated preliminary antitumor activity and a favorable safety profile across multiple solid tumor types, supporting its advancement to the next phase of clinical study.

Events after the Reporting Period

● The phase II clinical trial of JSKN016 monotherapy in multiple subgroups of lung cancer to evaluate efficacy, safety, and dose optimization is currently being conducted.

● The cohort expansion phase clinical trial of JSKN016 monotherapy for the treatment of non-HER2-positive BC is currently being conducted.

● In March 2025, IND application for JSKN016 combined with chemotherapy/immunotherapy /tyrosine kinase inhibitors (TKI) as first-line and later-line treatment of multiple subgroups of non-small cell lung cancer (NSCLC) was approved by CDE.

● In March 2025, IND application for JSKN016 combined with chemotherapy/immunotherapy as first-line and later-line treatment of multiple subgroups of non-HER2-positive BC was approved by CDE.

JSKN033

JSKN033 is a proprietary high-concentration co-formulation consisting of ADC and immune checkpoint inhibitor, independently developed by the Company, which can be administered subcutaneously. JSKN033 is the world’s first subcutaneous injectable ADC for clinical trials. By combining immunotherapy (KN035) and ADC (JSKN003), JSKN033 is anticipated to significantly enhance efficacy while leading to improved safety and convenience.

Events during the Reporting Period

● In March 2024, the first patient has been successfully dosed in Australia in the phase I/II clinical study of JSKN033 for the treatment of HER2-expressing advanced or metastatic solid tumors. The dose escalation phase has been completed until now.

● In November 2024, the research updates of the first-in-human phase I/II clinical trial of JSKN033 for the treatment of HER2-expressing advanced or metastatic solid tumors, have been presented for the first time as a poster in the Late-Breaking Abstract session at the 39th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper). JSKN033 presented a favorable safety profile and encouraging anti-cancer activity in heavily treated patients.

● In December 2024, JSKN033 received approval from CDE to initiate a phase I/II clinical trial in Chinese patients with advanced metastatic malignant tumors and the first patient was successfully dosed in January 2025. This study has previously been included in the "Pilot Program for Optimizing the Review and Approval of Clinical Trials for Innovative Drugs".

KN046

KN046, a bispecific antibody (BsAb) immune checkpoint inhibitor, composed of a fusion of CTLA-4 and PD-L1 single domain antibody, engineered to target the tumor microenvironment with high PD-L1 expression. Multiple clinical trials of KN046 at different stages covering different types of tumors including non-small cell lung cancer (NSCLC) have been conducted in China, the United States and Australia.

Events during the Reporting Period

● In February 2024, the results of the phase II clinical trial of KN046 in combination with nab-paclitaxel as first-line treatment for advanced triple-negative breast cancer were published in Nature Communications.

● In March 2024, the results of the phase II clinical trial of KN046 in combination with chemotherapy as first-line treatment for metastatic NSCLC were published in Cell Reports Medicine.

● In August 2024, the results of phase Ib clinical trial of KN046 in combination with chemotherapy and palliative radiotherapy as first-line treatment for advanced esophageal squamous cell carcinoma were published in Cancer Immunology, Immunotherapy.

● In September 2024, we completed OS final analysis for a phase III clinical trial of KN046 for the treatment of advanced squamous NSCLC (sq NSCLC).

● In December 2024, the results of the phase II clinical trial of KN046 in combination with Axitinib as first-line treatment for advanced NSCLC were presented at the European Society for Medical Oncology Immuno-Oncology (ESMO IO) Congress 2024.

Events after the Reporting Period

● In February 2025, the results of phase II clinical trial of KN046 in combination with lenvatinib for the treatment of advanced unresectable or metastatic hepatocellular carcinoma were published in Nature Communications.

Early-stage R&D

Leveraging proprietary platform technologies including glycan-specific conjugation, linker-payload, dual-payload conjugation, and bispecific antibodies, the Company has developed globally competitive new drugs such as ADCs and bispecific antibodies. The bispecific ADCs (BADC) and dual-drug conjugates (BADDC) developed by the Company, not only improve tumor targeting but also address heterogeneity and resistance issues, offering new strategies for cancer treatment. Currently multiple innovative bispecific ADC new drug candidates are in preclinical development and will be advanced into clinical trials sequentially.

● In June 2024, Jiangsu Alphamab entered into a research and collaboration agreement with ArriVent BioPharma, Inc. to use Jiangsu Alphamab’s proprietary linker-payload (Alphatecan) and glycan-specific conjugation platforms to discover and develop novel bispecific ADCs.

Manufacturing Facilities

The Company’s manufacturing facility was constructed in compliance with Good Manufacturing Practice (GMP) standards of the National Medical Products Administration of China (NMPA), the U.S. Food and Drug Administration (FDA), and the European Medicines Agency (EMA). The production lines are equipped with world-class equipment capable of meeting the large-scale manufacturing needs for a variety of biologics, including ADCs, both in the clinical and commercial stages. In 2024, the Company further expanded its production capacity by constructing the new suite dedicated to ADC drugs, which will be put into operation soon.

Other Highlights

● In November 2024, the Company was granted "2024 Top 100 Chinese Pharmaceutical Innovative Enterprises"and"2024 China Pharmaceutical Innovative Enterprise Bispecific Antibody Track Top5"by Healthcare Executive, a specialized magazine focusing on the pharmaceutical industry.

● In November 2024, the Company was granted "2024 Top 100 Brand Influence of Chinese Pharmaceutical Enterprise" by the China Health Culture Association, the China Hospital Association, and the National Health Commission of the People’s Republic of China Health TV Channel (CHTV) at the 2024 Healthy China Communication Conference.

For more information, please refer to the Company’s Annual Results Announcement for the Year Ended December 31, 2024 published on the Hong Kong Stock Exchange and the Company’s official website.