On August 6, 2025 Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company working to develop a new class of drugs based on targeted protein degradation, reported financial results for the second quarter ended June 30, 2025, and provided a corporate update (Press release, Arvinas, AUG 6, 2025, View Source [SID1234654859]).

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"It was an eventful and exciting quarter at Arvinas, with significant clinical and regulatory progress across our pipeline of PROTAC degraders," said John Houston, Ph.D., Chairperson, Chief Executive Officer and President at Arvinas. "The recent submission of a New Drug Application to the U.S. Food and Drug Administration for vepdegestrant represents a truly significant first for Arvinas – the first PROTAC degrader to enter clinical trials and have a positive readout in a Phase 3 clinical trial, and the first ever new drug application submitted for a PROTAC. We also continued to advance our early-stage programs, presenting compelling first in human data from ARV-102, our LRRK2 degrader, and preclinical data for our BCL6 degrader, ARV-393, as well as initiating a Phase 1 clinical trial with our KRAS G12D degrader, ARV-806. We have multiple near-term clinical and regulatory milestones, and our programs offer a rich set of catalysts over the next 12 months."

2Q 2025 Business Highlights and Recent Developments

Vepdegestrant: Oral PROTAC ER degrader
As part of Arvinas’ global collaboration with Pfizer, the companies:

Submitted a New Drug Application to the U.S. Food and Drug Administration for vepdegestrant.
Presented results from the VERITAC-2 Phase 3 clinical trial in a late-breaker oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper), with simultaneous publication of results in the New England Journal of Medicine.
Pivotal Phase 3 VERITAC-2 clinical trial results demonstrated 2.9-month improvement in median progression-free survival (PFS) when compared to fulvestrant in previously treated patients with an estrogen receptor 1 mutation; the trial did not reach statistical significance in improvement in PFS in the intent-to-treat population.
Vepdegestrant was generally well tolerated, with few discontinuations and low rates of gastrointestinal-related adverse events
Added a combination cohort of vepdegestrant plus Pfizer’s KAT6 inhibitor (PF-07248144) to Pfizer’s ongoing Phase 1 clinical trial (ClinicalTrials.gov Identifier: NCT04606446).
The trial is currently evaluating Pfizer’s KAT6 inhibitor in combination with endocrine therapies following CDK4/6 inhibitor treatments; the trial is being operationalized and funded by Pfizer.
ARV-102: Oral PROTAC LRRK2 degrader

Presented single ascending dose (SAD) and multiple ascending dose (MAD) data from the ongoing Phase 1 clinical trial in healthy volunteers in an oral session at the Alzheimer’s Disease/Parkinson’s Disease (AD/PD) conference and at the International Association of Parkinsonism and Related Disorders demonstrating blood-brain barrier penetration, central and peripheral LRRK2 degradation, and reduction of pathway biomarkers:
At a single oral dose of at least 60 mg, and once daily repeated oral doses of at least 20 mg, ARV-102 achieved greater than 50% LRRK2 reduction in the cerebral spinal fluid (CSF) and greater than 90% LRRK2 reduction in the peripheral blood mononuclear cells (PBMCs), indicating substantial central and peripheral LRRK2 protein degradation.
Inhibition of Rab10 phosphorylation in PBMCs and reduction of bis(monoacylglycerol)phosphate (BMP) in urine following single doses of ARV-102, signifying downstream LRRK2 pathway engagement.
Bioavailable and brain penetrant with dose dependent exposure in the CSF.
ARV-102 was generally safe and well tolerated with no serious adverse events reported after single or multiple doses.
Completed enrollment in the SAD cohort of the ongoing Phase 1 clinical trial in patients with Parkinson’s disease.
Activated site in preparation for multiple dose cohort initiation in patients with Parkinson’s disease.

ARV-393: Oral PROTAC BCL6 degrader

Continued recruiting patients in the first-in-human Phase 1 clinical trial in patients with relapsed/refractory non-Hodgkin lymphoma (NHL) (ClinicalTrials.gov Identifier: NCT06393738).
Presented new preclinical data at the 2025 American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting and the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress:
Data presented at AACR (Free AACR Whitepaper) demonstrated that ARV-393 had broad and significant combinability with standard of care chemotherapy, standard of care biologics and investigational small molecule inhibitors targeting clinically validated oncogenic drivers of lymphoma.
Data presented at EHA (Free EHA Whitepaper) demonstrated:
Significant single-agent activity of ARV-393 in models of nodal T-follicular helper cell lymphoma, angioimmunoblastic-type (also known as AITL) and transformed follicular lymphoma.
Enhanced tumor growth inhibition, including tumor regressions, with ARV-393 in combination with small molecule inhibitors in models of aggressive diffuse large B-cell lymphoma (DLBCL).
Overall, current preclinical data suggest that ARV-393 has the potential to be an attractive combination partner for development of novel therapies for lymphoma, including chemo-free combination regimens and/or "all oral" treatment options.

ARV-806: Novel PROTAC KRAS G12D degrader

Initiated enrollment in the Phase 1 clinical trial evaluating ARV-806 in patients with solid tumors harboring KRAS G12D mutations (ClinicalTrials.gov Identifier: NCT07023731).

Corporate updates:

Announced that John Houston, Ph.D., Chairperson, Chief Executive Officer (CEO) and President at Arvinas, informed the Board of Directors of his plans to retire from his role as CEO and President following a search for, and the appointment of, a new CEO.
The Arvinas Board of Directors has begun a search for a new CEO, and Dr. Houston will remain Chairperson of Arvinas’ Board of Directors upon retiring as CEO and President.
Anticipated Upcoming Milestones and Expectations

Vepdegestrant: Oral PROTAC ER degrader
As part of Arvinas’ global collaboration with Pfizer, the companies plan to:

Continue market preparations for vepdegestrant in advance of the PDUFA action date.
Revise our vepdegestrant collaboration with Pfizer with the goal of maximizing the value of vepdegestrant.
Present patient reported outcomes data from the VERITAC-2 clinical trial evaluating vepdegestrant versus fulvestrant for previously treated patients with ESR1 mutated- ER+/HER2- advanced or metastatic breast cancer at the European Society for Medical Oncology 2025 Congress (October 2025).
Present results of the TACTIVE-N trial, a Phase 2 clinical trial of neoadjuvant vepdegestrant, in a mini oral session at European Society for Medical Oncology 2025 Congress (ClinicalTrials.gov Identifier: NCT05549505) (October 2025).
ARV-102: Oral PROTAC LRRK2 degrader

Share final data from the SAD/MAD cohorts of the Phase 1 clinical trial in healthy volunteers (2H 2025).
Share initial data from the SAD cohort of the ongoing Phase 1 clinical trial in patients with Parkinson’s disease (2H 2025).
Initiate enrollment in the multiple dose cohort of the Phase 1 clinical trial in patients with Parkinson’s disease (2H 2025); present initial data from multiple dose cohort (2026).
Initiate Phase 1b clinical trial in patients with progressive supranuclear palsy (1H 2026).
ARV-393: Oral PROTAC BCL6 degrader

Share preclinical data in combination with glofitamab in models of aggressive high grade DLBCL (2H 2025).
Share preliminary clinical data from the ongoing Phase 1 clinical trial in patients with NHL (ClinicalTrials.gov Identifier: NCT06393738) (2H 2025).
ARV-806: Novel PROTAC KRAS G12D degrader

Continue enrollment in the Phase 1 clinical trial of ARV-806 in patients with solid tumors harboring KRAS G12D mutations (ClinicalTrials.gov Identifier: NCT07023731).
Share preclinical data from the clinical stage ARV-806 program (2H 2025).
Financial Guidance
Based on its current operating plan, Arvinas believes its cash, cash equivalents, and marketable securities as of June 30, 2025, is sufficient to fund planned operating expenses and capital expenditure requirements into the second half of 2028.

Second Quarter Financial Results
Cash, Cash Equivalents, and Marketable Securities Position: As of June 30, 2025, cash, cash equivalents and marketable securities were $861.2 million, as compared with $1,039.4 million as of December 31, 2024. The decrease in cash, cash equivalents and marketable securities of $178.2 million for the six months ended June 30, 2025 was primarily related to cash used in operations of $177.0 million, the purchase of lab equipment and leasehold improvements of $1.6 million and $0.1 million of long-term debt repayments, partially offset by proceeds from the issuance of shares under the ESPP of $0.5 million.

Research and Development Expenses: Generally Accepted Accounting Principles (GAAP) Research and development (R&D) expenses were $68.6 million for the quarter ended June 30, 2025, as compared with $93.7 million for the quarter ended June 30, 2024. The decrease in research and development expenses of $25.1 million for the quarter was primarily due to a decrease in external expenses of $18.3 million and a decrease in compensation and related personnel expenses of $7.9 million, which are not allocated by program. External expenses include (i) program-specific expenses, which decreased by $15.9 million, primarily driven by decreases in our vepdegestrant (ARV-471) and luxdegalutamide (ARV-766) programs of $10.0 million and $9.5 million, respectively, partially offset by increases in our ARV-102 and ARV-806 programs of $2.1 million and $1.5 million, respectively, and (ii) our non-program specific expenses, which decreased by $2.4 million.

Non-GAAP R&D expenses were $59.5 million for the quarter ended June 30, 2025, as compared with $83.0 million for the quarter ended June 30, 2024, excluding $0.6 million of restructuring expense for the three months ended June 30, 2025, and $8.5 million and $10.7 million of non-cash stock-based compensation expense for the three months ended June 30, 2025 and 2024, respectively. A reconciliation of GAAP to non-GAAP financial measures used in this press release can be found at the end of this press release.

General and Administrative Expenses: GAAP General and administrative (G&A) expenses were $25.3 million for the quarter ended June 30, 2025, as compared with $31.3 million for the quarter ended June 30, 2024. The decrease in general and administrative expenses of $6.0 million for the quarter was primarily due to a decrease in personnel and infrastructure-related costs of $4.8 million and professional fees of $2.2 million, partially offset by an increase in costs related to developing our commercial operations of $1.1 million.

Non-GAAP G&A expenses were $20.2 million for the quarter ended June 30, 2025, as compared with $20.4 million for the quarter ended June 30, 2024, excluding $0.4 million of restructuring expense for the quarter ended June 30, 2025, and $4.7 million and $10.9 million of non-cash stock-based compensation expense for the quarter ended June 30, 2025 and 2024, respectively. A reconciliation of GAAP to non-GAAP financial measures used in this press release can be found at the end of this press release.

Revenue: Revenue was $22.4 million for the quarter ended June 30, 2025 as compared with $76.5 million for the quarter ended June 30, 2024. Revenue for the quarter is related to the Vepdegestrant (ARV-471) Collaboration Agreement with Pfizer and the collaboration and license agreement with Pfizer. The decrease of $54.1 million was primarily due to $45.6 million of decreased revenue from the Novartis License Agreement and the Novartis Asset Agreement, both of which were entered into during the three months ended June 30, 2024 and were completed by December 31, 2024 as the technology transfer of our ongoing and planned clinical trials of luxdegalutamide (ARV-766) were transitioned to Novartis. Revenue from the Vepdegestrant (ARV-471) Collaboration Agreement with Pfizer decreased by $6.8 million related to the removal of the first-line Phase 3 combination trial with Pfizer’s novel investigational CDK4 inhibitor, atirmociclib, and the removal of the second-line Phase 3 combination trial with a CDK4/6 inhibitor from the development plan during the first quarter of 2025 and revenue from the Bayer Collaboration Agreement decreased by $1.6 million as a result of the termination of the Bayer Collaboration Agreement in August 2024.

Investor Call & Webcast Details
Arvinas will host a conference call and webcast today, August 6, 2025, at 8:00 a.m. ET to review its second quarter 2025 financial results and discuss recent corporate updates. Participants are invited to listen by going to the Events and Presentation section under the Investors page on the Arvinas website at www.arvinas.com. A replay of the webcast will be available on the Arvinas website following the completion of the event and will be archived for up to 30 days.

On August 6, 2025 Artiva Biotherapeutics, Inc. (Nasdaq: ARTV) (Artiva), a clinical-stage biotechnology company whose mission is to develop effective, safe, and accessible cell therapies for patients with devastating autoimmune diseases and cancers, reported financial results for the second quarter ended June 30, 2025, and highlighted recent progress (Press release, Artiva Biotherapeutics, AUG 6, 2025, View Source [SID1234654858]).

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"We are making meaningful progress across our ongoing clinical trials exploring AlloNK in autoimmune disease. We now have over a dozen sites enrolling across our trials in the US and have already treated over a dozen patients with AlloNK in combination with monoclonal antibodies across rheumatoid arthritis, SLE, lupus nephritis, Sjögren’s disease, and systemic sclerosis," said Fred Aslan, M.D., Chief Executive Officer of Artiva. "By the end of 2025, we look forward to sharing initial translational data supporting AlloNK’s mechanism of action, and safety data, supporting the potential of our therapy, which includes the use of cyclophosphamide and fludarabine, to be administered and managed in an outpatient setting across multiple autoimmune indications. We also look forward to announcing our lead indication by the end of 2025, setting the stage to share initial clinical response data in that indication in the first half of next year."

Recent Business Highlights

AlloNK (also known as AB-101) Updates

•
Over a dozen clinical sites active and enrolling across two company-sponsored trials in autoimmune diseases: the Phase 2a basket clinical trial and the Phase 1/1b clinical trial in systemic lupus erythematosus (SLE) with or without lupus nephritis (LN)
•
First patient treated with AlloNK + rituximab in recently initiated global Phase 2a company-sponsored basket clinical trial for refractory rheumatoid arthritis (RA), Sjögren’s disease (SjD), idiopathic inflammatory myopathies (myositis, or IIM), and systemic sclerosis (scleroderma, or SSc)
•
Over a dozen patients treated with AlloNK + monoclonal antibody (mAb) across refractory RA, SLE, LN, SjD, and SSc in the company-sponsored trials and an investigator-initiated basket trial
Upcoming Milestones

•
By Year-End 2025: Initial safety and translational data for AlloNK + mAb across multiple autoimmune diseases from ongoing clinical trials and disclosure of lead indication for further development
o
Mechanistic and translational data for AlloNK in autoimmune diseases
o
Insights into tolerability of AlloNK + mAb, and the patient journey in community rheumatology sites, including the potential ease of use of conditioning regimens with cyclophosphamide and fludarabine
o
Disclosure of lead indication for AlloNK development in autoimmune diseases
•
1H 2026: Initial clinical response data in the lead autoimmune indication from ongoing clinical trials with longer follow-up to inform registrational strategy

Second Quarter 2025 Financial Results

•
Cash, Cash Equivalents and Investments. As of June 30, 2025, Artiva had cash, cash equivalents, and investments of $142.4 million, which is expected to fund operations into Q2 2027
•
Research and Development Expenses. Research and development expenses were $17.9 million for the three months ended June 30, 2025, compared to $12.3 million for the three months ended June 30, 2024
•
General and Administrative Expenses. General and administrative expenses were $4.9 million for the three months ended June 30, 2025, compared to $3.9 million for the three months ended June 30, 2024
•
Other Income (expense), net. Other income, net, was $1.6 million for the three months ended June 30, 2025, compared to other expense, net, of $1.7 million for the three months ended June 30, 2024
•
Net Loss. Net loss totaled $21.3 million for the three months ended June 30, 2025, as compared to net loss of $17.8 million for the three months ended June 30, 2024, with non-cash stock-based compensation expense of $1.5 million for the three months ended June 30, 2025, and June 30, 2024

On August 6, 2025 Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage, global biopharmaceutical company focused on developing differentiated molecules and combination therapies for patients with cancer, reported financial results for the second quarter ended June 30, 2025, and provided a pipeline update on its clinical-stage investigational molecules across multiple common cancers (Press release, Arcus Biosciences, AUG 6, 2025, View Source [SID1234654857]).

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"We have now presented data from over 125 patients treated with casdatifan monotherapy or casdatifan plus cabozantinib, which we believe demonstrate the potential for casdatifan to be the best-in-class HIF-2a inhibitor for clear cell RCC," said Terry Rosen, Ph.D., chief executive officer of Arcus. "We are forging ahead with speed and efficiency in our development of casdatifan across multiple ccRCC settings, including our global Phase 3 PEAK-1 trial in the IO-experienced setting and Phase 1b/3 eVOLVE-RCC02 trial in the first-line metastatic setting, the latter in collaboration with AstraZeneca. With a strong balance sheet and focused investment, we are well equipped to fund casdatifan through data for PEAK-1, which has been designed to enroll rapidly and to achieve a readout as quickly as possible."

Pipeline Highlights:

Casdatifan (HIF-2a inhibitor)
Phase 3 Program:
•PEAK-1, a Phase 3 study evaluating casdatifan + cabozantinib versus cabozantinib in IO-experienced metastatic ccRCC, has been initiated.
•eVOLVE-RCC02, a Phase 1b/3 study sponsored and operationalized by AstraZeneca, evaluating casdatifan + volrustomig, an investigational anti-PD-1/CTLA-4 bispecific antibody, in first-line (1L), metastatic ccRCC, has also been initiated. The study was designed to support initiation of the Phase 3 portion as efficiently as possible.
Recent Data Presentations:
•Initial data from the ARC-20 study of casdatifan + cabozantinib in IO-experienced patients with metastatic ccRCC were presented in an oral session at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

◦At the time of the data cutoff (DCO, March 14, 2025), treatment with casdatifan+cabozantinib showed a confirmed overall response rate (ORR) of 46% in patients who reached a minimum of 12 weeks (two scans) of follow-up, and almost all patients achieved tumor reduction.
◦At the time of DCO, the vast majority of patients remained on therapy.
◦There was no meaningful overlapping toxicity for the two drugs, and the combination had a well-tolerated safety profile with no patients discontinuing both therapies due to adverse events.
◦This cohort is evaluating the same regimen, in the same setting, as the PEAK-1 Phase 3 study.
Planned Data Readouts:
◦Fall 2025: More mature data from the ARC-20 cohorts evaluating casdatifan monotherapy in patients who had progressed on both an anti-PD-1 and a TKI.
◦2026: More mature data from the casdatifan plus cabozantinib cohort and initial data from the new ARC-20 cohorts evaluating casdatifan in the 1L and IO-experienced settings.
Domvanalimab (Fc-silent anti-TIGIT antibody) plus Zimberelimab (anti-PD-1 antibody)

Planned Data Readouts:

•An abstract describing OS data from the Phase 2 EDGE-Gastric study, evaluating domvanalimab plus zimberelimab and chemotherapy in upper GI adenocarcinomas, has been accepted for presentation at the 2025 ESMO (Free ESMO Whitepaper) Congress in October.
•The first Phase 3 OS data readout for domvanalimab plus zimberelimab will be from the ongoing Phase 3 study STAR-221, evaluating domvanalimab plus zimberelimab plus chemotherapy in PD-L1 all-comer 1L unresectable or metastatic upper GI adenocarcinomas. Results for this trial are event-driven, and OS data are expected in 2026.
Quemliclustat (small-molecule CD73 inhibitor)
•Orphan Drug Designation was granted by the U.S. Food and Drug Administration (FDA) to quemliclustat for the treatment of pancreatic cancer.
•PRISM-1, a Phase 3 trial of quemliclustat combined with gemcitabine/nab-paclitaxel versus gemcitabine/nab-paclitaxel in first-line metastatic pancreatic ductal adenocarcinoma, is enrolling rapidly, with enrollment completion expected in the third quarter of 2025, well within 12 months of study initiation.
Etrumadenant (adenosine A2a/A2b receptor antagonist)
•In March 2025, we engaged with the FDA regarding promising results from the ARC-9 study evaluating etrumadenant in third-line metastatic colorectal cancer (mCRC); although the FDA’s feedback confirmed the potential for a registrational path for this program in third-line mCRC, based on our strategic priorities, Arcus and Gilead agreed to not pursue a Phase 3 study at this time and, in June 2025, Gilead returned its license to etrumadenant to Arcus.
Early Discovery
•Arcus has several research and preclinical programs ongoing that are addressing validated oncology and inflammation targets. Arcus expects that the next development candidate to enter the clinic will be a small molecule directed against an inflammation target.

Financial Results for Second Quarter 2025:
•Cash, Cash Equivalents and Marketable Securities were $927 million as of June 30, 2025, compared to $992 million as of December 31, 2024. The decrease during the period is primarily due to the use of cash in our research and development activities partially offset by the net proceeds from our underwritten offering in February 2025 and the additional $50 million draw down under our term loan facility in June 2025. We believe our cash, cash equivalents and marketable securities, together with available facilities, will be sufficient to fund operations through the initial pivotal readouts for domvanalimab, quemliclustat and casdatifan, which include PEAK-1.

•Revenues were $160 million for the second quarter 2025, compared to $39 million for the same period in 2024. The increase in revenue was driven by a cumulative catch-up to revenue of $143 million relating to pausing future development of etrumadenant and Gilead’s related return of its license to the program. Arcus expects to recognize GAAP revenue of between $225 million and $235 million for the full year 2025, which includes the cumulative catch-up.
•Research and Development (R&D) Expenses were $139 million for the second quarter 2025, compared to $115 million for the same period in 2024. The net increase of $24 million was primarily due to increased CMC costs. We expect to incur elevated CMC costs through the third quarter of 2025. Otherwise, clinical costs for our late-stage programs were flat, as increased enrollment and start-up activities for PRISM-1 and PEAK-1 were largely offset by lower costs for STAR-221. Other increases in expense resulted from an increase in early-stage development activities, driven by Phase 2 activities for casdatifan. Non-cash stock-based compensation expense was $8 million for the second quarter 2025, compared to $10 million for the same period in 2024. For the second quarters 2025 and 2024, Arcus recognized gross reimbursements of $33 million and $40 million, respectively, for shared expenses from its collaborations. R&D expenses by quarter may fluctuate due to the timing of clinical manufacturing and standard-of-care therapeutic purchases with a corresponding impact on reimbursements. Arcus expects R&D expenses to decline commencing in the fourth quarter 2025 as costs related to the domvanalimab Phase 3 development program decrease significantly.
•General and Administrative (G&A) Expenses were $29 million for the second quarter 2025, compared to $30 million for the same period in 2024. The decrease was primarily driven by a decrease in compensation and personnel costs driven by lower stock-based compensation, partially offset by increased costs recognized in the quarter in connection with the Gilead Agreement. Non-cash stock-based compensation expense was $7 million for the second quarter 2025, compared to $10 million for the same period in 2024.
•Net income (Loss) was $— million for the second quarter 2025, compared to a $93 million net loss for the same period in 2024.

On August 6, 2025 Arbutus Biopharma Corporation (Nasdaq: ABUS) ("Arbutus" or the "Company"), a clinical-stage biopharmaceutical company focused on infectious disease, reported second quarter 2025 financial results and provided a corporate update (Press release, Arbutus Biopharma, AUG 6, 2025, View Source [SID1234654856]).

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"We delivered a strong quarter, marked by positive quarterly earnings resulting from the conclusion of our Greater China partnership with Qilu," said Lindsay Androski, President and CEO of Arbutus. "Once again holding global rights for imdusiran, and launching a late-stage clinically focused Scientific Advisory Board, were two important steps taken this quarter in our quest to drive long-term value through our chronic hepatitis B virus (cHBV) programs."

"I am excited to announce that Dr. Harry Janssen of Erasmus MC in Rotterdam has joined our Scientific Advisory Board, bringing his unparalleled knowledge and experience in late-stage clinical trials in cHBV," Ms. Androski continued. "Today, the Company also congratulates Anuj Hasija on his appointment as Vice President, Global Commercial Strategy for Type 1 Diabetes at Vertex. Anuj has stepped down from the Arbutus Board in order to focus exclusively on this new role, and on behalf of the Company and the entire Board, I thank Anuj for his service. We are also excited to welcome Dr. Roger Sawhney as the newest member of our Board. Dr. Sawhney has enjoyed a distinguished career spanning senior executive roles in biotech, pharma and investing, and also brings extensive public company board experience."

LNP Litigation

Arbutus continues 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.
In the Moderna U.S. litigation, fact discovery has been completed, and expert discovery is concluding. The summary judgment phase of the case began in July 2025 and a jury trial is scheduled to be held in March 2026. Additionally, in July 2025, the case was reassigned to a different judge in the same court. In March 2025, the Company, alongside Genevant Sciences, filed five international lawsuits against Moderna and its affiliates seeking to enforce patents protecting the Company’s patented LNP technology across 30 countries. The first major hearings in the international lawsuits are expected in the first half of calendar year 2026.
The claim construction hearing for the lawsuit against Pfizer/BioNTech occurred in December 2024. The court has not provided guidance for the timing of its ruling in the claim construction hearing, which could potentially come in 2025.

Corporate Updates

In June 2025, the Company and Qilu Pharmaceutical mutually agreed to conclude the strategic partnership for the development, manufacturing, and commercialization of imdusiran in Greater China. The Company now once again holds global rights for its lead compound, imdusiran.
Dr. Harry L.A. Janssen, MD, PhD, joined the Company’s Scientific Advisory Board effective August 1, 2025, increasing the membership to six global experts in cHBV treatment. Dr. Janssen is a Professor of Hepatology and the Chair of the Department of Gastroenterology and Hepatology at Erasmus MC, Rotterdam, The Netherlands.
Effective August 4, 2025, Anuj Hasija resigned from the Company’s Board of Directors due to his transition to a full-time executive role that precludes his participation on the Arbutus and other boards of directors.
Filling the vacancy on our Board of Directors, Dr. Roger Sawhney, MD, joined the Board effective August 4, 2025. Dr. Sawhney is a seasoned executive and board member with extensive experience across biotechnology, pharmaceuticals, healthcare technology, and investment sectors. He holds an MD from Harvard Medical School and a BA in Economics from Stanford University.

Financial Results

Cash, Cash Equivalents and Investments

As of June 30, 2025, the Company had cash, cash equivalents and investments in marketable securities of $98.1 million compared to $122.6 million as of December 31, 2024. During the six months ended June 30, 2025, the Company used $29.1 million in operating activities, which included one-time payments related to our restructuring efforts. This was partially offset by $3.1 million of proceeds from the exercise of employee stock options.

Revenue

Total revenue was $10.7 million for the quarter ended June 30, 2025, compared to $1.7 million for the same period in 2024. The increase of $9.0 million was primarily due to the recognition of all previously-deferred revenue as a result of the conclusion of the Company’s strategic partnership with Qilu Pharmaceutical, partially offset by a decrease in license royalty revenues due to a decline in Alnylam’s sales of ONPATTRO.

Operating Expenses

Research and development expenses were $5.5 million for the quarter ended June 30, 2025 compared to $15.6 million for the same period in 2024. The decrease of $10.1 million was due primarily to 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 the Company’s workforce.

General and administrative expenses were $3.3 million for the quarter ended June 30, 2025, compared to $7.5 million for the same period in 2024. This decrease was due primarily to cost cutting efforts by the Company, which drove reductions in litigation-related legal fees and employee compensation-related expenses.

Restructuring costs in the quarter ended June 30, 2025 were $0.2 million, and all remaining restructuring-related payments are expected to be made in the second half of 2025.

Net Income/Loss

For the quarter ended June 30, 2025, the Company’s net income was $2.5 million, or income of $0.01 per basic and diluted common share, as compared to a net loss of $19.8 million, or a loss of $0.11 per basic and diluted common share, for the quarter ended June 30, 2024.

Outstanding Shares

As of June 30, 2025, the Company had 191.6 million common shares issued and outstanding, as well as 15.2 million stock options and unvested restricted stock units outstanding.

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

Three Months Ended June 30, Six Months Ended June 30,
2025 2024 2025 2024
Revenue
Collaborations and licenses $ 10,213 $ 1,155 $ 11,529 $ 2,094
Non-cash royalty revenue 526 571 974 1,164
Total Revenue 10,739 1,726 12,503 3,258
Operating expenses
Research and development 5,498 15,551 14,457 30,954
General and administrative 3,328 7,547 9,160 12,859
Change in fair value of contingent consideration 260 211 559 391
Restructuring costs 165 — 12,538 —
Total operating expenses 9,251 23,309 36,714 44,204
Gain (loss) from operations 1,488 (21,583 ) (24,211 ) (40,946 )
Other income
Interest income 1,042 1,829 2,239 3,374
Interest expense (28 ) (34 ) (56 ) (78 )
Foreign exchange gain (loss) 21 (8 ) 25 (21 )
Total other income 1,035 1,787 2,208 3,275
Income tax expense — — — —
Net income (loss) $ 2,523 $ (19,796 ) $ (22,003 ) $ (37,671 )
Net income (loss) per common share
Basic $ 0.01 $ (0.11 ) $ (0.12 ) $ (0.21 )
Diluted $ 0.01 $ (0.11 ) $ (0.12 ) $ (0.21 )
Weighted average number of common shares
Basic 191,551,282 188,041,489 191,130,631 181,842,519
Diluted 192,399,733 188,041,489 191,130,631 181,842,519

UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS
(in thousands)

June 30, 2025 December 31, 2024
Cash, cash equivalents and marketable securities, current $ 98,088 $ 122,623
Accounts receivable and other current assets 5,031 4,693
Total current assets 103,119 127,316
Property and equipment, net of accumulated depreciation and impairment 148 3,309
Right of use asset — 1,048
Other non-current assets — 34
Total assets $ 103,267 $ 131,707

Accounts payable and accrued liabilities $ 4,508 $ 7,564
Deferred license revenue, current — 7,571
Lease liability, current 514 483
Total current liabilities 5,022 15,618
Liability related to sale of future royalties 3,910 4,829
Deferred license revenue, non-current — 2,863
Contingent consideration 10,784 10,225
Lease liability, non-current 575 806
Total stockholders’ equity 82,976 97,366
Total liabilities and stockholders’ equity $ 103,267 $ 131,707

UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
(in thousands)

Six Months Ended June 30,
2025 2024
Net loss $ (22,003 ) $ (37,671 )
Non-cash items 5,834 3,973
Change in deferred license revenue (10,434 ) (757 )
Other changes in working capital (2,537 ) 656
Net cash used in operating activities (29,140 ) (33,799 )
Net cash provided by investing activities 26,960 21,523
Issuance of common shares pursuant to the Open Market Sale Agreement — 44,124
Cash provided by other financing activities 3,237 4,676
Net cash provided by financing activities 3,237 48,800
Effect of foreign exchange rate changes on cash and cash equivalents 25 (21 )
Increase in cash and cash equivalents 1,082 36,503
Cash and cash equivalents, beginning of period 36,330 26,285
Cash and cash equivalents, end of period 37,412 62,788
Investments in marketable securities 60,676 85,725
Cash, cash equivalents and marketable securities, end of period $ 98,088 $ 148,513

About Imdusiran (AB-729)  

Imdusiran is an RNAi therapeutic specifically designed to reduce all hepatitis B viral proteins and antigens including HBsAg, which is thought to be a key prerequisite to enable reawakening of a patient’s immune system to control the virus. Imdusiran targets hepatocytes using Arbutus’ novel covalently conjugated N-Acetylgalactosamine (GalNAc) delivery technology enabling subcutaneous delivery. To date, Arbutus has reported a total of eight patients with cHBV who have achieved a functional cure following treatment with imdusiran and NA therapy in combination with either IFN or low dose nivolumab plus an immunotherapeutic. Clinical data generated thus far has shown imdusiran to be generally safe and well-tolerated, while also providing meaningful reductions in HBsAg and hepatitis B virus DNA.

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. cHBV infection represents a significant unmet medical need. The World Health Organization estimates that over 250 million people worldwide suffer from cHBV infection, while other estimates indicate that approximately 2 million people in the United States suffer from cHBV infection. Approximately 1.1 million people die every year from complications related to cHBV infection despite the availability of effective vaccines and current treatment options.

On August 6, 2025 Aptose Biosciences Inc. ("Aptose" or the "Company") (OTC: APTOF, TSX: APS), a clinical-stage precision oncology company developing the tuspetinib (TUS)-based triple drug frontline therapy to treat patients with newly diagnosed AML, reported that the Cohort Safety Review Committee (CSRC) monitoring Aptose’s Phase 1/2 TUSCANY trial of TUS in combination with standard of care dosing of venetoclax and azacitidine (TUS+VEN+AZA triplet) has approved escalating from 120 mg TUS dose to 160 mg TUS dose based on its favorable review of safety and efficacy data from patients in the first three cohorts (40 mg, 80 mg, and 120 mg TUS dose levels) of the trial (Press release, Aptose Biosciences, AUG 6, 2025, View Source [SID1234654855]). Enrollment is open for dosing subjects at the 160 mg TUS dose level.

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Aptose also announced that it has received an additional advance of US$1.1M from Hanmi Pharmaceutical Co. Ltd. ("Hanmi"), as part of a US$8.5M loan facility agreement with Hanmi (the "Loan Agreement") announced prior on June 20, 2025 (press release here: View Source). To date, Aptose has received an aggregate of US$5.6M under the Loan Agreement.

The TUS+VEN+AZA triplet is being developed as a one-of-a-kind, safe and mutation agnostic frontline therapy to treat large, mutationally diverse populations of newly diagnosed AML patients who are ineligible to receive induction chemotherapy. Aptose reports that at the 120 mg TUS dose level, as with the prior reported 40 mg and 80 mg dose cohorts, no significant safety concerns or dose limiting toxicities (DLTs) have been reported in the TUSCANY trial, including no prolonged myelosuppression in Cycle 1 of subjects in remission.

All patients treated in the 120 mg dose cohort remain on study while enrollment is open for the 160 mg dose cohort. Aptose previously reported that the first two dose cohorts have demonstrated safety, CRs, and minimal residual disease (MRD) negativity across patients with diverse mutations in an oral presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress (EHA 2025) in June (press release here: View Source).

"Data from three cohorts, with a 40, 80 or 120 mg dose of TUS in the TUS+VEN+AZA triplet, continue to build a strong case for TUS as a therapeutic option for some of the most difficult to treat AML populations," said Rafael Bejar, M.D., Ph.D., Chief Medical Officer of Aptose. "In particular, patients with adverse biallelic TP53 or FLT3-ITD mutations, and those without FLT3 mutations, were able to safely achieve complete remissions with MRD negativity. After review of the most recent safety and efficacy data, our CSRC recommended that we continue to escalate dosing."

TUSCANY: TUS+VEN+AZA Triplet Phase 1/2 Study

The tuspetinib-based TUS+VEN+AZA triplet therapy is being advanced in the TUSCANY Phase 1/2 trial with the goal of creating an improved frontline therapy for newly diagnosed AML patients that is active across diverse AML populations, durable, and well tolerated. Earlier APTIVATE trials of TUS as a single agent and in combination as TUS+VEN demonstrated favorable safety and broad activity in diverse relapsed or refractory (R/R) AML populations that went beyond the more prognostically favorable NPM1 and IDH mutant subgroups. Indeed, responses were also noted in R/R AML patients with highly adverse TP53 and RAS mutations, and those with mutated or unmutated (wildtype) FLT3 genes.

The TUSCANY triplet Phase 1/2 study, being conducted at 10 leading U.S. clinical sites by elite clinical investigators, is designed to test various doses and schedules of TUS in combination with standard dosing of AZA and VEN for patients with AML who are ineligible to receive induction chemotherapy. A convenient, once daily oral agent, TUS is being administered in 28-day cycles. Multiple U.S. sites are enrolling in the TUSCANY trial with anticipated enrollment of 18-24 patients by late 2025. Data will be released as it becomes available.

More information on the TUSCANY Phase 1/2 study can be found on www.clinicaltrials.gov (here: View Source).