On August 7, 2025 Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) reported financial results for the second quarter of 2025, along with recent product highlights and corporate updates (Press release, Zai Laboratory, AUG 7, 2025, View Source [SID1234655002]).

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"Zai Lab is entering a pivotal period – defined by innovation, scale and strong execution," said Dr. Samantha Du, Founder, Chairperson, and CEO of Zai Lab. "We are making meaningful progress throughout our business – expanding patient impact, accelerating global innovation, and operating with financial discipline. Updated ASCO (Free ASCO Whitepaper) data for ZL-1310 (DLL3 ADC) reaffirm its best-in-class potential in second-line SCLC, and we are moving swiftly into pivotal development while exploring opportunities in first-line SCLC and other neuroendocrine carcinomas. We were also encouraged by positive data for bemarituzumab in first-line gastric cancer and by emerging preclinical results for our IL-13/IL-31 bispecific antibody in atopic dermatitis, reinforcing both our near-term commercial opportunities and the potential of our global pipeline, respectively. With multiple launches ahead, a robust pipeline, and profitability1 within reach, Zai Lab is executing on its vision to become a leading global biopharma company."

"VYVGART continues to lead our commercial momentum, with record patient utilization driven by longer treatment durations and growing adoption in the maintenance setting," said Josh Smiley, President and COO of Zai Lab. "The July update to national MG guidelines further strengthens VYVGART’s role in both acute and chronic care, and we expect momentum to accelerate in the second half. We are also preparing for several high-impact launches – including KarXT and bemarituzumab – that, together with pipeline-in-a-product assets like VYVGART and povetacicept, will fuel our next wave of growth. With a 28% year-over-year reduction in operating loss and a 37% improvement on an adjusted basis, we are on track to achieve profitability1 in the fourth quarter. Backed by a strong cash position2, a growing commercial business, and an advancing global pipeline, Zai Lab is well equipped to deliver long-term shareholder value."

Second Quarter 2025 Financial Results

•Product revenue, net was $109.1 million in the second quarter of 2025, compared to $100.1 million for the same period in 2024, representing 9% y-o-y growth, 10% y-o-y growth at constant exchange rate (CER). This increase was primarily driven by increased sales for VYVGART, XACDURO, and NUZYRA, partially offset by softer sales for ZEJULA:

–VYVGART and VYVGART Hytrulo were $26.5 million in the second quarter of 2025, compared to $18.1 million in the first quarter of 2025. Sales grew 46% quarter over quarter driven by an extension of duration of therapy and increasing market penetration.

–ZEJULA was $41.0 million in the second quarter of 2025, compared to $45.0 million for the same period in 2024. Sales were softer due to evolving competitive dynamics within the PARPi class.

–XACDURO, which was launched since the fourth quarter of 2024, was $4.6 million in the second quarter of 2025.

–NUZYRA was $14.3 million in the second quarter of 2025, compared to $12.3 million for the same period in 2024. This growth was supported by increasing market coverage and penetration.

•Research and Development (R&D) expenses were $50.6 million in the second quarter of 2025, compared to $61.6 million for the same period in 2024. The decrease reflects reduced personnel and clinical trial costs, driven by ongoing resource prioritization and efficiency efforts.

•Selling, General and Administrative expenses were $71.0 million in the second quarter of 2025, compared to $79.7 million for the same period in 2024. The decrease was primarily driven by reduced personnel costs because of resource prioritization and efficiency efforts.

•Loss from operations was $54.9 million in the second quarter of 2025, $34.2 million when adjusted to exclude certain non-cash expenses including depreciation, amortization, and share-based compensation. A reconciliation of loss from operations (GAAP) to adjusted loss from operations (non-GAAP) is included at the end of this release.

•Net loss was $40.7 million in the second quarter of 2025, or a loss per ordinary share attributable to common stockholders of $0.04 (or loss per American Depositary Share (ADS) of $0.37), compared to a net loss of $80.3 million for the same period in 2024, or a loss per ordinary share of $0.08 (or loss per ADS of $0.82). These decreases in net loss were primarily due to product revenue growing faster than net operating expenses.

•Cash and cash equivalents, short-term investments, and current restricted cash totaled $832.3 million as of June 30, 2025, compared to $857.3 million as of March 31, 2025.

Recent Pipeline Highlights

Below are key product updates since our last earnings release:

Oncology Pipeline

•ZL-1310 (DLL3 ADC):

–In June 2025, Zai Lab presented positive data from an ongoing, global Phase 1a/1b clinical trial of ZL-1310 for the treatment of patients with extensive-stage small cell lung cancer (ES-SCLC) at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. In second-line (2L) SCLC, objective response rate (ORR) was 67% across all dose levels (n=33) and 79% at 1.6 mg/kg dose (n=14). ZL-1310 demonstrated a well-tolerated safety profile at target doses of less than 2.0 mg/kg, with Grade ≥3 treatment-related adverse events (TRAEs) of 6%, no Grade ≥2 interstitial lung disease, and no drug discontinuations.

–In May 2025, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to ZL-1310 for treatment of ES-SCLC.

•Bemarituzumab (FGFR2b): In June 2025, Zai Lab announced the Phase 3 FORTITUDE-101 clinical trial evaluating first-line bemarituzumab plus chemotherapy (mFOLFOX6) met its primary endpoint of overall survival (OS) at a pre-specified interim analysis. At the interim analysis, bemarituzumab plus chemotherapy significantly improved OS in patients with FGFR2b overexpression compared to chemotherapy alone. The most common treatment-emergent adverse events (>25%) in patients treated with bemarituzumab plus chemotherapy were reduced visual acuity, punctate keratitis, anemia, neutropenia, nausea, corneal epithelium defect and dry eye. While ocular events were consistent with the Phase 2 experience and observed in both arms, they occurred with greater frequency and severity in the Phase 3 bemarituzumab arm. Detailed results from FORTITUDE 101 will be shared at a future medical meeting. Zai Lab plans to move rapidly toward regulatory submission in China in the second half of 2025.

•Tumor Treating Fields (TTFields): In May 2025, Zai Lab partner Novocure presented results from the Phase 3 PANOVA-3 trial for pancreatic cancer as a late-breaking oral presentation at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting. The data demonstrated that TTFields therapy, when added to standard of care, achieved an OS benefit supported by significantly improved quality of life and extended pain-free survival, a key outcome for patients with pancreatic cancer. Zai Lab participated in the study in Greater China (mainland China, Hong Kong, Macau and Taiwan, collectively) and plans to file for regulatory approval in China in the second half of 2025.

Immunology Pipeline

•Efgartigimod (FcRn): In July 2025, the China Guidelines for the Diagnosis and Treatment of Myasthenia Gravis (MG) (2025) was published, emphasizing the importance of Minimal Symptom Expression (MSE) as the primary treatment goal in MG. The guidelines have highlighted VYVGART’s ability to achieve MSE rapidly and provide durable benefit. VYVGART is now recommended for early use in mild-to-moderate and highly active patients and for sustained long-term treatment to maximize potential benefit.

•ZL-1503 (IL-13/IL-31R): In June 2025, Zai Lab announced new preclinical data highlighting the potential of ZL-1503 as a promising treatment for moderate-to-severe atopic dermatitis and other IL-13 and IL-31-driven diseases. Its favorable preclinical safety profile, prolonged half-life and durable suppression of both inflammatory and pruritogenic pathways support the continued advancement of ZL-1503 toward clinical development.

Anticipated Major Milestones in 2025 and the First Half of 2026

Upcoming Potential NMPA Submissions

•Bemarituzumab (FGFR2b) in first-line gastric cancer in the second half of 2025

•Tumor Treating Fields (TTFields) in first-line pancreatic cancer in the second half of 2025

•Efgartigimod (FcRn) for prefilled syringe in generalized myasthenia gravis (gMG) and chronic inflammatory demyelinating polyneuropathy (CIDP) in the second half of 2025

Upcoming Potential NMPA Approvals

•Xanomeline-Trospium (or KarXT) (M1/M4-agonist) in schizophrenia

•Tisotumab Vedotin (Tissue Factor ADC) in recurrent or metastatic cervical cancer following progression on or after chemotherapy

•Repotrectinib (ROS1/TRK) in NTRK+ solid tumors

Expected Clinical Developments and Data Readouts

Global Pipeline

ZL-1310 or Zocilurtatug pelitecan (DLL3 ADC)

•Second-Line ES-SCLC: Zai Lab to provide data update and to initiate a global registrational study of ZL-1310 monotherapy in the second half of 2025.

•First-Line ES-SCLC: Zai Lab to provide data readout for dose escalation of ZL-1310 doublet in combination with atezolizumab.

•Other neuroendocrine carcinomas: Zai Lab to provide data readout from the global Phase 1/2 study in patients with selected solid tumors.

ZL-1503 (IL-13/IL-31R)

•Zai Lab to advance into a global Phase 1 study in moderate-to-severe atopic dermatitis in the second half of 2025.

ZL-6201 (LRRC15 ADC)

•Zai Lab to advance into global Phase 1 development for patients with sarcoma and potentially other LRRC15-positive solid tumors, such as breast cancer and other malignancies.

Regional Pipeline

Bemarituzumab (FGFR2b)

•Zai Lab partner Amgen to provide detailed results from the Phase 3 FORTITUDE-101 study of bemarituzumab combined with chemotherapy versus chemotherapy alone in first-line gastric cancer at an upcoming medical meeting. Zai Lab participated in the study in Greater China.

•Zai Lab partner Amgen to provide potential data readout from the Phase 1b/3 FORTITUDE-102 study of bemarituzumab plus chemotherapy and nivolumab versus chemotherapy and nivolumab in first-line gastric cancer. Zai Lab participated in the study in Greater China.

Xanomeline-Trospium (or KarXT) (M1/M4-agonist)

•Zai Lab partner Bristol Myers Squibb to provide data readout from the Phase 3 ADEPT-2 study of KarXT in Alzheimer’s Disease Psychosis in the second half of 2025. Zai Lab participated in the study in Greater China.

Efgartigimod (FcRn)

•Lupus Nephritis (LN): Zai Lab and partner argenx to provide topline results from the Phase 2 study in LN in the fourth quarter of 2025.

•Seronegative gMG: Zai Lab partner argenx to provide topline results from the global Phase 3 ADAPT-SERON study in seronegative gMG in the second half of 2025. Zai Lab participated in the study in Greater China.

•Ocular myasthenia gravis: Zai Lab partner argenx to provide topline results from the global Phase 3 ADAPT-OCULUS study in the first half of 2026. Zai Lab participated in the study in Greater China.

•Sjogren’s disease: Zai Lab to join the registrational UNITY study of efgartigimod subcutaneous given by prefilled syringe in Sjogren’s disease in Greater China in the third quarter of 2025.

Povetacicept (APRIL/BAFF)

•Primary Membranous Nephropathy (pMN): Zai Lab plans to partner with Vertex to execute the global pivotal Phase 2/3 study of povetacicept in pMN in Greater China. The study is expected to start in the second half of 2025.

•IgA Nephropathy (IgAN): Zai Lab partner Vertex will conduct an interim analysis of the global Phase 3 RAINIER study following 36 weeks of treatment with the potential to file for U.S. accelerated approval in the first half of 2026.

VRDN-003 (IGF-1R, subcutaneous)

•Zai Lab to initiate a registrational study in thyroid eye disease in Greater China in the second half of 2025.

•Zai Lab partner Viridian to provide topline results from the global registrational REVEAL-1 and REVEAL-2 studies in the first half of 2026.

Conference Call and Webcast Information

Zai Lab will host a live conference call and webcast today, August 7, 2025, at 8:00 a.m. ET (8:00 p.m. HKT). Listeners may access the live webcast by visiting the Company’s website at View Source Participants must register in advance of the conference call.

Details of registration links are as follows:

•For webcast: View Source

•For dial-in: View Source

All participants must use the link provided above to complete the online registration process in advance of the conference call. Dial-in details will be in the confirmation email which the participant will receive upon registering.

A replay will be available shortly after the call and can be accessed by visiting the Company’s website.

On August 7, 2025 Cullinan Therapeutics, Inc. (Nasdaq: CGEM; "Cullinan"), a biopharmaceutical company focused on developing modality-agnostic targeted therapies, reported an update on recent and anticipated business highlights and announced its financial results for the second quarter ended June 30, 2025 (Press release, Cullinan Oncology, AUG 7, 2025, View Source [SID1234654971]).

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"I am proud of the team’s strong execution throughout the first half of the year as we continue to advance the CLN-978 program across three active studies in SLE, RA, and Sjögren’s disease. With the addition of velinotamig, we further solidified our leadership position in the development of T cell engagers for autoimmune diseases. With these two programs, our portfolio covers the entire breadth of the B cell compartment. Recent data presented at the European Alliance of Associations for Rheumatology (EULAR) meeting reinforced the potential of T cell engagers as disease-modifying therapies across a wide spectrum of autoimmune diseases," said Nadim Ahmed, Chief Executive Officer of Cullinan Therapeutics.

"We also continue making important progress across our oncology portfolio, recently sharing results from the pivotal Phase 2b portion of the REZILIENT1 study of zipalertinib at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting, and we look forward to sharing multiple new data sets at upcoming medical conferences. Pending discussions with the U.S. FDA, our partner Taiho plans to submit an NDA in relapsed EGFR ex20ins NSCLC later this year and expects to complete enrollment of the frontline study REZILIENT3 in the first half of 2026. We also plan to share clinical data for CLN-049, our FLT3xCD3 bispecific T cell engager, in patients with relapsed/refractory AML and MDS later this year. With $510.9 million in cash and investments and runway into 2028, we have the resources to generate multiple value-driving catalysts near term and beyond across both our immunology and oncology programs. Lastly, I am pleased to welcome Drs. Mittie Doyle and Andrew Allen to our Board of Directors. They are proven leaders with deep strategic and development expertise in immunology and oncology, respectively, and I believe their contributions will be invaluable as we continue to advance our programs. I would also like to thank Drs. Anne-Marie Martin and David Ryan for their contributions to our progress and success over the last several years."

Portfolio Highlights

Immunology

•
CLN-978 (CD19xCD3 bispecific T cell engager): Systemic lupus erythematosus, rheumatoid arthritis, and Sjögren’s disease
o
The global Phase 1 study in patients with moderate to severe SLE is enrolling in the United States, Europe, and Australia, and the Company plans to share initial safety data and B cell depletion data from Part A of the study in Q4 2025.
o
The Phase 1 study in patients with active, difficult-to-treat rheumatoid arthritis is enrolling in Europe. This company-sponsored study is being led by sites at FAU Erlangen-Nuremberg in Germany and Università Cattolica del Sacro Cuore in Italy. The Company plans to share initial data from this study during the first half of 2026.
o
The global Phase 1 study in patients with active, moderate to severe Sjögren’s disease is enrolling in the U.S. and is now also active in Europe following recent regulatory approval.

•
Velinotamig (BCMAxCD3 bispecific T cell engager): Autoimmune diseases
o
In June 2025, the Company entered into an agreement with Genrix Bio for an exclusive global (ex-Greater China) license to velinotamig. Under the agreement, Cullinan paid Genrix Bio an upfront license fee of $20 million. In the future, Genrix Bio will also be eligible to receive up to $292 million in development and regulatory milestones plus up to an additional $400 million in sales-based milestones, as well as tiered royalties on potential ex-Greater China net sales.
o
Genrix Bio plans to initiate a Phase 1 study in patients with autoimmune diseases in China by the end of 2025. Cullinan intends to use the data generated to accelerate global clinical development of the program. Following the completion of the Genrix Bio Phase 1 study, Cullinan will conduct all further development of velinotamig in autoimmune diseases.
Oncology

•
Zipalertinib (EGFR ex20ins inhibitor), collaboration with Taiho Oncology: EGFR ex20ins NSCLC
o
In June 2025, results from the pivotal Phase 2b portion of REZILIENT1 in patients with EGFR ex20ins NSCLC who have received prior therapy were shared at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting and published simultaneously in the Journal of Clinical Oncology. Cullinan plans to share updated efficacy and safety data in patients previously treated with amivantamab during a mini oral abstract session at the IASLC 2025 WCLC.
o
Pending discussions with the U.S. Food and Drug Administration, Taiho plans to submit an NDA in relapsed EGFR ex20ins NSCLC by the end of 2025. Taiho expects to complete enrollment of the pivotal study REZILIENT3 in 1L EGFR ex20ins NSCLC in the first half of 2026.
o
Taiho plans to share initial data from the REZILIENT2 cohort exploring zipalertinib in patients with uncommon EGFR mutations during a mini oral abstract session at the IASLC 2025 WCLC. Taiho also plans to share initial data from the REZILIENT2 cohort exploring zipalertinib in patients with active brain metastases at the ESMO (Free ESMO Whitepaper) Congress 2025.

•
CLN-049 (FLT3xCD3 bispecific T cell engager): Acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS)
o
Enrollment continues in the Phase 1 study in patients with relapsed/refractory AML or MDS and the company plans to share clinical data from this study in Q4 2025.
o
Enrollment also continues in the Phase 1 study in patients with measurable minimal residual disease in AML.
•
CLN-619 (Anti-MICA/MICB monoclonal antibody): NSCLC and multiple myeloma
o
Enrollment continues in the Phase 1 expansion cohorts in patients with NSCLC and the Phase 1 study in patients with relapsed/refractory multiple myeloma.
•
CLN-617 (IL-2 and IL-12 cytokine fusion protein): Solid tumors
o
Enrollment continues in the Phase 1 study in patients with advanced solid tumors.
Corporate Updates

•
Mittie Doyle, M.D., and Andrew Allen, M.D., Ph.D., were appointed to the Board of Directors, effective August 7, 2025. Both board directors bring significant leadership experience, with Dr. Doyle having extensive immunology clinical development expertise, and Dr. Allen with extensive oncology clinical development experience. Anne-Marie Martin, Ph.D., and David Ryan, M.D., will resign from Cullinan’s Board of Directors effective August 7, 2025.
Second Quarter 2025 Financial Results

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Cash Position: Cash, cash equivalents, short- and long-term investments, and interest receivable were $510.9 million as of June 30, 2025. Cullinan continues to expect its cash resources to provide runway into 2028 based on its current operating plan.
•
R&D Expenses: Research and development expenses were $61.0 million for the second quarter of 2025, compared to $36.3 million for the same period in 2024.
•
G&A Expenses: General and administrative expenses were $14.8 million for the second quarter of 2025, compared to $13.8 million for the same period in 2024.
•
Net Loss: Net loss attributable to Cullinan was $70.1 million for the second quarter of 2025, compared to $42.0 million for the same period in 2024.

On August 7, 2025 Janux Therapeutics, Inc. (Nasdaq: JANX) (Janux), a clinical-stage biopharmaceutical company developing a broad pipeline of novel immunotherapies by applying its proprietary technologies to its Tumor Activated T Cell Engager (TRACTr), Tumor Activated Immunomodulator (TRACIr), and Adaptive Immune Response Modulator (ARM) platforms, reported financial results for the second quarter ended June 30, 2025, and provided a business update (Press release, Janux Therapeutics, AUG 7, 2025, View Source [SID1234654987]).

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"The recent expansion of our TRACTr, TRACIr, and ARM development programs displays our ability to enact a strategy that attempts to both maximize the benefit and value of our current clinical programs, including JANX007, while continuing to advance other differentiated candidates in oncology and autoimmune disease," said David Campbell, Ph.D., President and CEO of Janux. "We look forward to additional clinical data from JANX007 and JANX008 expected in the second half of 2025."

RECENT BUSINESS HIGHLIGHTS AND FUTURE MILESTONES:

•
R&D Day highlighted pipeline progress and novel bispecific platform in autoimmune disease.
In July 2025, Janux management presented multiple product candidates identified from its preclinical pipeline to move towards clinical trials.

•
A PSMA-TRACIr designed to be combined with potentially best-in-treatment asset, JANX007, and provide CD28 co-stimulation to further differentiate depth and durability of patient responses.
•
A TROP2-TRACTr added a first-in-class opportunity targeting multiple solid tumors with preclinical data supporting differentiated safety and efficacy potential.
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A CD19-ARM displayed rapid, deep and durable B-cell depletion in periphery and tissues with a prolonged memory B cell reset while maintaining a large safety window in non-human primates.

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JANX007 continues to enroll in the first-in-human Phase 1 clinical trial in mCRPC (NCT05519449).

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JANX008 continues to enroll in the first-in-human Phase 1 clinical trial in advanced or metastatic solid tumors (NCT05783622).

•
Clinical milestone payment of $10 million from Merck recently triggered by first patient dosed in the lead collaboration program under the companies’ 2020 Research Collaboration and Exclusive License Agreement.

Additional data from JANX007 and JANX008 will be presented at future Janux events in the second half of 2025.

SECOND QUARTER 2025 FINANCIAL RESULTS:

•
Cash and cash equivalents and short-term investments: As of June 30, 2025, Janux reported cash and cash equivalents and short-term investments of $996.0 million, compared to $1.03 billion at December 31, 2024.

•
Research and development expenses: Research and development expenses for the quarter ended June 30, 2025 were $34.7 million, compared to $14.9 million for the comparable period in 2024.

•
General and administrative expenses: General and administrative expenses for the quarter ended June 30, 2025 were $10.5 million, compared to $7.8 million for the comparable period in 2024.

•
Net loss: For the quarter ended June 30, 2025, Janux reported a net loss of $33.9 million, compared to a net loss of $6.0 million for the comparable period in 2024.

Janux’s TRACTr, TRACIr and ARM Pipeline

Janux’s first clinical candidate, JANX007, is a TRACTr that targets prostate-specific membrane antigen (PSMA) and is being investigated in a Phase 1 clinical trial in adult patients with mCRPC. Janux’s second clinical candidate, JANX008, is a TRACTr that targets epidermal growth factor receptor (EGFR) and is being studied in a Phase 1 clinical trial for the treatment of multiple solid cancers including colorectal carcinoma, squamous cell carcinoma of the head and neck, non-small cell lung cancer, renal cell carcinoma, small cell lung cancer, pancreatic ductal adenocarcinoma and triple-negative breast cancer. Janux is also advancing additional CD3-based TRACTr and CD28-based TRACIr programs for future clinical development, including a PSMA-TRACIr for use in combination with our PSMA-TRACTr JANX007, and a TROP2-TRACTr for the treatment of TROP2+ solid tumors. Janux is advancing its first ARM platform program candidate, a CD19-ARM for the potential treatment of autoimmune diseases toward clinical trials. Janux is also generating a number of additional TRACTr, TRACIr and ARM programs for potential future development.

On August 7, 2025 aTyr Pharma, Inc. (Nasdaq: ATYR) ("aTyr" or the "Company"), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, reported second quarter 2025 results and provided a corporate update (Press release, aTyr Pharma, AUG 7, 2025, View Source [SID1234655006]).

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"With the recent completion of the last patient visit in our Phase 3 EFZO-FIT study of efzofitimod in pulmonary sarcoidosis, a major form of interstitial lung disease (ILD), we are on track to report topline data in mid-September," said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. "This upcoming readout represents a major inflection point for aTyr, our clinical program for efzofitimod in ILD, and the broader sarcoidosis community, and we look forward to sharing the results."

Second Quarter 2025 and Subsequent Period Highlights

Completed the last patient visit in the global pivotal Phase 3 EFZO-FIT study to evaluate the efficacy and safety of efzofitimod in patients with pulmonary sarcoidosis. Topline data from the study are expected in mid-September 2025. This is a randomized, double-blind, placebo-controlled, 52-week study consisting of three parallel cohorts randomized equally to either 3.0 mg/kg or 5.0 mg/kg of efzofitimod or placebo administered intravenously monthly for a total of 12 doses. The study enrolled 268 patients with pulmonary sarcoidosis across 85 centers in nine countries. The trial design incorporates a forced steroid taper. The primary endpoint of the study is steroid reduction measured as the absolute change from baseline to week 48. Secondary endpoints include measures of sarcoidosis symptoms and lung function. Patients who complete the study and wish to receive treatment with efzofitimod outside of the clinical trial are eligible to participate in an Individual Patient Expanded Access Program.

Announced interim data from the ongoing Phase 2 EFZO-CONNECT study to evaluate the efficacy, safety and tolerability of efzofitimod in patients with limited or diffuse systemic sclerosis (SSc, or scleroderma)-related ILD (SSc-ILD). This proof-of-concept study is a randomized, double-blind, placebo-controlled, 28-week study consisting of three parallel cohorts randomized 2:2:1 to either 270 mg or 450 mg of efzofitimod or placebo administered intravenously monthly for a total of six doses. Enrollment in the study is ongoing, and the study intends to enroll up to 25 patients at multiple centers in the United States. The interim analysis evaluated skin assessments and serum biomarkers at baseline and week 12 for efzofitimod and placebo patients. Eight patients were evaluated, including five with diffuse and three with limited SSc-ILD. Key findings for efzofitimod-treated patients to date included:

Stable or improved modified Rodnan Skin Score (mRSS), a key measure of skin fibrosis, for all patients and an improvement of 4 points or greater for three out of four efzofitimod-treated patients with diffuse SSc-ILD, where the minimal clinically important difference (MCID) is a 4 to 6 point improvement at 12 months

Preliminary signals of improvement for inflammatory biomarkers including interferon gamma (IFN-γ) and monocyte chemoattractant protein-1 (MCP-1) and disease biomarkers Krebs von den Lungen-6 (KL-6) and surfactant protein-D (SP-D)

Efzofitimod was generally safe and well tolerated at all doses, with no treatment related serious adverse events

Advanced ATYR0101 to investigational new drug (IND) candidate stage for pulmonary fibrosis. ATYR0101 is a fusion protein derived from a proprietary extracellular domain of aspartyl-tRNA synthetase (DARS) that binds to latent transforming growth factor beta binding protein 1 (LTBP-1) to induce cell death of myofibroblasts, which are key cells responsible for driving the progression of fibrosis. The Company anticipates filing an IND application in the second half of 2026.

Preclinical data generated to date demonstrating ATYR0101’s unique anti-fibrotic mechanism through LTBP-1 were presented in an oral presentation at the American Thoracic Society 2025 Respiratory Innovation Summit

Announced that the Company was added to the Russell 2000 Index and broad market Russell 3000 Index. These additions were a part of the 2025 Russell U.S. Indexes annual reconstitution.

Second Quarter 2025 Financial Highlights and Cash Position

Cash & Investment Position: Cash, cash equivalents, restricted cash and available-for-sale investments as of June 30, 2025, were $83.2 million. Subsequent to the end of the second quarter 2025, the Company raised approximately $30.7 million in gross proceeds from its at-the-market (ATM) offering with Jefferies LLC. The Company believes its cash runway will be sufficient to fund its operations for a period of one year following the Phase 3 EFZO-FIT readout.

R&D Expenses: Research and development expenses were $15.4 million for the second quarter 2025, which consisted primarily of clinical trial costs for the Phase 3 EFZO-FIT and Phase 2 EFZO-CONNECT studies, manufacturing costs for the efzofitimod program and research and development costs for the efzofitimod and discovery programs.

G&A Expenses: General and administrative expenses were $4.9 million for the second quarter 2025.

About Efzofitimod

Efzofitimod is a first-in-class biologic immunomodulator in clinical development for the treatment of interstitial lung disease (ILD), a group of immune-mediated disorders that can cause inflammation and fibrosis, or scarring, of the lungs. Efzofitimod is a tRNA synthetase derived therapy that selectively modulates activated myeloid cells through neuropilin-2 to resolve inflammation without immune suppression and potentially prevent the progression of fibrosis. aTyr is currently investigating efzofitimod in the global Phase 3 EFZO-FIT study in patients with pulmonary sarcoidosis, a major form of ILD, and in the Phase 2 EFZO-CONNECT study in patients with systemic sclerosis (SSc, or scleroderma)-related ILD. These forms of ILD have limited therapeutic options and there is a need for safer and more effective, disease-modifying treatments that improve outcomes.

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.