On January 13, 2025 Adaptimmune Therapeutics plc (NASDAQ: ADAP), a company working to redefine the treatment of solid tumor cancers with cell therapy, reported that letetresgene autoleucel (lete-cel), has been granted breakthrough therapy designation by the U.S. FDA for the treatment of patients with unresectable or metastatic myxoid/round cell liposarcoma (MRCLS) who have received prior anthracycline-based chemotherapy, are positive for HLA-A*02:01, HLA-A*02:05, or HLA-A*02:06, and whose tumor expresses the NY-ESO-1 antigen (Press release, Adaptimmune, JAN 13, 2025, View Source [SID1234649720]).

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More details about the Company’s sarcoma franchise, including the lete-cel clinical program and launch progress for TECELRA (afamitresgene autoleucel), the Company’s first commercial product and the first FDA approved engineered cell therapy for a solid tumor, will be provided during the Company’s presentation at the Annual J.P. Morgan Healthcare Conference, taking place in San Francisco, California, on Tuesday, January 14th, 2025, 4:30-5:10 PM PST (Webcast access here).

Breakthrough therapy designation for lete-cel in MRCLS was based on the results in this indication from the Phase II IGNYTE-ESO trial. The Company previously received breakthrough therapy designation for lete-cel for the treatment of synovial sarcoma in 2016. In the Phase II analysis, 27/64 (42%) people with synovial sarcoma or MRCLS had RECISTv1.1 responses by independent review, with 6 complete responses and 21 partial responses. The response rate was 14/34 (41%) for people with synovial sarcoma and 13/30 (43%) for people with MRCLS. The median duration of response (DoR) was 12.2 months (95% CI 6.8, 19.5). In synovial sarcoma, the median duration of response was 18.3 months (95% CI 3.3, -). In MRCLS, the median duration of response was 12.2 months (95%, CI 5.3, -). Safety findings were consistent with the known profile of lete-cel from previous data. All patients experienced treatment-emergent adverse events: cytopenias, cytokine release syndrome (CRS) and rash were the most common adverse events. Overall, toxicities were manageable, and consistent with an acceptable benefit to risk profile. Data from this trial were presented at the Connective Tissue Oncology Society (CTOS) 2024 annual meeting (Link to press release HERE; Presentation HERE).

Adrian Rawcliffe, Adaptimmune’s Chief Executive Office: "This designation by the FDA highlights the potential of lete-cel to address a critical need for new treatment options for patients with MRCLS. This is another important milestone in building out our sarcoma franchise, as we aim to bring lete-cel to market in 2026 for the treatment of synovial sarcoma and MRCLS. We look forward to initiating a rolling Biologics License Application for lete-cel later this year for the treatment of both sarcoma indications."

The breakthrough therapy designation is designed to expedite drug development and review processes. The criteria for this designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. With the designation, lete-cel will receive incentives, such as additional interaction and guidance from the FDA, the potential for a rolling submission, and potential priority review of the biologics license application, as well as other opportunities to expedite the development.

In addition to the Company’s presentation at J.P. Morgan, Adaptimmune will present the Company’s Allo-T program as a spin-out opportunity at the Biotech ShowCase and the Wuxi Global Forum 2025 Investor Roundtable. Details can be found below:

Biotech Showcase | Investor conference | Co-produced by Demy-Colton and EBD Group
Presentation at 2 p.m. PST on Tuesday, January 14th, Yosemite C, Hilton Hotel, Union Square.
WuXi Global Forum 2025 – WXPress: for WuXi news and R&D insights
Round Table Presentation at 5 p.m. PST on Tuesday, January 14th, Tower 3, Hilton Hotel, Union Square

On Jan. 13, 2025 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported highlights for the upcoming company presentation at the 43rd Annual J.P. Morgan Healthcare Conference on Thursday, January 16, 2025 at 9:00 AM PT/12:00 PM ET (Press release, Precigen, JAN 13, 2025, View Source [SID1234649719]). Participants may view details for Precigen’s company presentation at Precigen’s website in the Events & Presentations section at investors.precigen.com/events-presentations.

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"Our recent BLA submission for PRGN-2012 in RRP puts our commercial readiness activities in high gear and we are well underway to ensure full readiness in anticipation of a potential launch in the second half of this year. We have made tremendous progress in the past six months on payer and prescriber analysis, clarifying the market opportunity, building out our commercial and distribution infrastructure, and other activities to ensure we fully coordinate the payor, provider, and patient journeys for launch," said Phil Tennant, Chief Commercial Officer of Precigen. "Our updated analysis of the market using claims data and electronic health records underscores the significant potential for PRGN-2012 in RRP with approximately 27,000 adult patients now identified in the US and we expect more than 125,000 patients ex-US. We are confident that we will be ready to commercialize in the US as soon as we receive approval so that patients can gain access to this game-changing treatment."

"With 100% manufacturing success rate demonstrated to date, we anticipate our in-house commercial drug substance cGMP manufacturing facility will be ready to meet the projected demand to support potential commercial launch in the second half of this year," said Rutul Shah, Chief Operating Officer of Precigen.

"We continue to de-risk our PRGN-2012 asset and now that we have submitted the BLA in RRP, we are a step closer to completing our transition to a commercial-stage company. As we start 2025, we are laser focused on working with the FDA and advancing commercial readiness efforts in anticipation of a potential launch in the second half of this year. In addition, we are preparing for submissions to other health authorities in our prioritized global markets. Beyond RRP, we see immense potential for the AdenoVerse platform in other HPV6/11-driven indications, such as genital warts, and HPV16/18-driven indications, such as cervical cancer and head and neck cancers," said Helen Sabzevari, PhD, President and CEO of Precigen. "As communicated previously, we plan to pursue strategic partnerships to advance our UltraCAR-T programs, which deliver autologous, antigen-specific CAR-T cells overnight to a patient at the patient’s medical center. In conjunction, we are preparing for an end of Phase 1b meeting with the FDA for PRGN-3006 to share results for this highly promising program, including new clinical biomarker data that may further enable patient stratification and positively impact efficacy."

Realizing Precigen’s Commercial Vision for PRGN-2012 AdenoVerse Gene Therapy in RRP

PRGN-2012 (INN: zopapogene imadenovec†) is an investigational off-the-shelf AdenoVerse gene therapy designed to elicit immune responses directed against cells infected with human papillomavirus (HPV) 6 or HPV 11 for the treatment of recurrent respiratory papillomatosis (RRP), a rare and devastating chronic disease for which the current standard-of-care is repeated surgeries. If approved, PRGN-2012 has the potential to be the first US Food and Drug Administration (FDA)-approved therapeutic for the treatment of RRP.
PRGN-2012 received Breakthrough Therapy Designation from the FDA. PRGN-2012 also received Orphan Drug Designation from the FDA and Orphan Drug Designation from the European Commission.
In December 2024, the Company announced that it had completed the submission of a biologics license application (BLA) with a request for priority review for PRGN-2012 for the treatment of adults with RRP to the FDA. The submission is in the initial 60 day review period, during which time the FDA will decide whether to accept the BLA for further review and set the Prescription Drug User Fee Act (PDUFA) action date. The BLA included a request for priority review, which, if granted, would reduce the review timeline from the standard 10-month to a priority 6-month review from the date the submission is accepted by the FDA.
The BLA, under an accelerated approval pathway, is supported by data from the Phase 1/2 pivotal study in which more than 50% of patients achieved Complete Response and more than 85% of patients had a decrease in surgical interventions in the year after PRGN-2012 treatment compared to the year prior to treatment. PRGN-2012 was well-tolerated with no dose-limiting toxicities and no treatment-related adverse events greater than Grade 2.
The confirmatory clinical trial of PRGN-2012 was initiated and is enrolling patients in accordance with the guidance from the FDA to initiate the study prior to submission of the BLA.
The Company continues to rapidly advance its commercial and manufacturing readiness campaign in anticipation of a potential 2025 launch.
Maximizing the Potential of PRGN-2009 AdenoVerse Gene Therapy in HPV-associated Cancers

PRGN-2009 Phase 2 clinical trials under a cost-effective cooperative research and development agreement (CRADA) with the National Cancer Institute (NCI) in recurrent/metastatic cervical cancer and in newly diagnosed HPV-associated oropharyngeal cancer are ongoing.
Enrollment was paused in the cervical cancer Phase 2 clinical trial at non-NCI sites as part of strategic reprioritization activities in 2024.
Maximizing the Value of the UltraCAR-T Platform through Strategic Partnerships

Enrollment was completed for the Phase 1b trial for PRGN-3006, which received Fast Track designation from the FDA for the treatment of relapsed or refractory (r/r) acute myeloid leukemia (AML).
Based on the results of correlative studies of the patient samples from the Phase 1/1b study, the Company has identified clinical biomarkers that correlate to objective responses after PRGN-3006 treatment in r/r AML patients. This advancement may further enable patient stratification and positively impact efficacy.
The Company is preparing for an end of Phase 1b meeting with the FDA to discuss results and next steps.
The Company plans to focus on strategic partnership opportunities to advance this promising UltraCAR-T program in AML.
*Cash on-hand is preliminary and unaudited and reflects preliminary financial information as of December 31, 2024. In preparing this information, the Company’s actual financial position as of December 31, 2024 has not yet been finalized by management or reviewed or audited by the Company’s independent registered public accounting firm. This information is also not a comprehensive statement of financial position or results of operations as of or for the year-ended December 31, 2024. Subsequent information or events may lead to material differences between the foregoing preliminary financial information and those reported in the Company’s subsequent SEC filings. Accordingly, investors should not place undue reliance on this preliminary financial information.

†zopapogene imadenovec is the international nonproprietary name (INN) for the investigational therapeutic known as PRGN-2012. Zopapogene imadenovec has not been approved by any health authority in any country for any indication.

On January 13, 2025 Autolus Therapeutics plc (Nasdaq: AUTL), a commercial stage biopharmaceutical company developing, manufacturing and delivering next-generation T cell therapies to patients, reported business updates and an overview of 2025 milestones (Press release, Autolus, JAN 13, 2025, View Source [SID1234649718]).

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"We enter 2025 with good momentum following the recent U.S. Food and Drug Administration (FDA) approval of AUCATZYL (obecabtagene autoleucel, or "obe-cel") and publication of the FELIX trial data in the New England Journal of Medicine. In addition, the inclusion of AUCATZYL in the NCCN Guidelines, noting the lack of REMS program associated with our therapy, provides further validation of the demonstrated clinical benefit, favorable safety profile and the potential impact of the therapy for patients in need," said Dr. Christian Itin, Chief Executive Officer of Autolus. "We are executing to plan with the commercial launch and are encouraged to enter the year with 24 authorized treatment centers out of the 30 we anticipated by end of Q1."

"With the AUCATZYL launch on track, we look forward to updating the market on additional clinical pipeline programs that we believe will drive future growth," Dr. Itin continued. "In the second half of 2024, we undertook an R&D portfolio review to evaluate the highest priority areas for strategic investment. In our upcoming R&D investor event we will provide an outlook and plan to share initial data from the ongoing CARLYSLE Phase 1 trial in systemic lupus erythematosus (SLE)."

AUCATZYL was approved by the FDA on November 8, 2024. Autolus expects to complete authorization of the first 30 treatment centers, covering approximately 60% of the identified target patient population, by the end of January 2025. The Company expects to complete authorization of 60 treatment centers, covering approximately 90% of the target patient population, by the end of 2025.

In December 2024, the National Comprehensive Cancer Network (NCCN) added AUCATZYL (obecabtagene autoleucel) to its Clinical Practice Guidelines in Oncology (NCCN Guidelines) for the treatment of adult patients with relapsed/refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL). The NCCN is a not-for-profit alliance of 30 leading cancer centers devoted to patient care, research, and education. The NCCN Guidelines are a comprehensive set of guidelines detailing the sequential management decisions and interventions that currently apply to 97% of cancers affecting patients in the U.S and are intended to ensure that all patients receive preventive, diagnostic, treatment, and supportive services that reflect the latest evidence in oncological patient care.

Obe-cel is also under regulatory review in both the European Union and the United Kingdom, with marketing authorization submissions accepted by the European Medicines Agency (EMA) in April 2024, and the UK Medicines and Healthcare products Regulatory Agency (MHRA) in August 2024. Assuming precedent regulatory timelines, Autolus expects potential marketing approvals from the MHRA and EMA in the second half of 2025.

2025 Events & Milestones

Obe-cel in autoimmune disease: initial data from SLE Phase 1 study Q1 2025
R&D Investor Event, New York City April 23, 2025
Initial data from PY01 trial of obe-cel in pediatric ALL H2 2025
MHRA & EMA approvals for obe-cel in ALL H2 2025
SLE Phase 1 trial presentation at medical conference H2 2025
Additional details regarding the R&D investor event, including registration link, will be communicated in the near future.

On January 13, 2025 Moderna, Inc. (NASDAQ:MRNA) reported business updates and progress across its pipeline of mRNA medicines (Press release, Moderna Therapeutics, JAN 13, 2025, View Source [SID1234649717]). Moderna enters 2025 with a focus on a prioritized portfolio addressing respiratory viruses, rare diseases, oncology, and latent and other viruses where there is unmet need.

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"In 2024, we achieved $3.0 – 3.1 billion in product sales, approval of our RSV vaccine and continued to adapt our COVID-19 business for the endemic setting. At the same time, we reduced our cash operating cost by over 25 percent compared to 2023 and aim to reduce 2025 cash costs by $1 billion with a plan for an additional $500 million cost savings in 2026," said Stéphane Bancel, Chief Executive Officer of Moderna. "We remain focused on our three strategic priorities: driving sales growth, delivering up to 10 product approvals over the next three years, and reducing costs across our business."

The Company’s presentation will take place on Monday, January 13, 2025, at 3:45 p.m. PT/6:45 p.m. ET at the 43rd Annual J.P. Morgan Healthcare Conference. A live webcast of both the presentation and the question-and-answer session will be available under "Events and Presentations" in the investor section of Moderna’s website at investors.modernatx.com. A replay of the webcast will be archived on Moderna’s website for at least 30 days following the presentation.

Summary of Financial Updates

2024 financial updates: Moderna achieved 2024 product sales in the range of approximately $3.0 to 3.1 billion (unaudited), comprising $1.7 billion in the U.S. and $1.3 to 1.4 billion in Rest of World. This includes more than $3.0 billion in Spikevax sales and minimal sales from mRESVIA. Cash, cash equivalents and investments at year-end 2024 were approximately $9.5 billion. Full financial details will be reported in connection with the Company’s earnings call on February 14, 2025.

2025 financial framework: Moderna is accelerating and expanding its previous cost efficiency and prioritization programs and now projects cash cost reductions of $1.0 billion (including cost of sales, research and development, and selling, general & administrative expense) in 2025. The Company expects an additional $0.5 billion in potential cash cost savings in 2026. Moderna now projects $1.5 billion to 2.5 billion in revenue in 2025, mostly in the second half of the year, primarily due to Spikevax and mRESVIA vaccine sales. The Company expects to end 2025 with cash and investments of approximately $6.0 billion.

Summary of Late-Stage Pipeline Milestones

Respiratory vaccines:

Next-generation COVID-19 vaccine: Moderna shared positive Phase 3 vaccine efficacy and immunogenicity data for its next-generation COVID-19 vaccine (mRNA-1283) at its 2024 R&D Day event. The Company has filed for regulatory approval of mRNA-1283 using a priority review voucher. The FDA has accepted Moderna’s Biologics License Application (BLA) for mRNA-1283 and has assigned a Prescription Drug User Fee Act (PDUFA) goal date of May 31, 2025.

Respiratory syncytial virus (RSV) vaccine: Moderna received regulatory approval of its RSV vaccine mRESVIA (mRNA-1345) for adults aged 60 years and older in 2024. The Company shared positive Phase 3 data for mRNA-1345 in high-risk adults aged 18-59 at its 2024 R&D Day event and has since filed with the FDA for regulatory approval using a priority review voucher.

Seasonal flu/COVID vaccine: Moderna shared positive Phase 3 immunogenicity data for its flu/COVID combination vaccine (mRNA-1083) for adults aged 50 years and older at its 2024 R&D Day event. The Company has filed with the FDA for regulatory approval of mRNA-1083.

Seasonal flu vaccine: The Company has initiated a two-season Phase 3 efficacy study (P304) for its seasonal flu vaccine (mRNA-1010), which has demonstrated consistently acceptable safety and tolerability across three previous Phase 3 trials. The Company anticipates efficacy data from the study in 2025 if sufficient cases are accrued in the first season; otherwise, the study will continue to a second season.

Latent and other vaccines:

Cytomegalovirus (CMV) vaccine: The pivotal Phase 3 study of Moderna’s CMV vaccine candidate (mRNA-1647) is fully enrolled and accruing cases, evaluating its efficacy, safety and immunogenicity in the prevention of primary infection in women of childbearing age. The Data Safety Monitoring Board (DSMB) met to review the initial study data and has informed the Company that the criterion for early efficacy was not met. The DSMB recommended that the study continue as planned. The Company remains blinded and anticipates final efficacy data from the study in 2025.

Norovirus vaccine: Moderna’s trivalent vaccine candidate for the prevention of norovirus (mRNA-1403) has advanced into a two-season pivotal Phase 3 randomized clinical trial evaluating its efficacy, safety and immunogenicity. The Company anticipates efficacy data from the study in 2025 if sufficient cases are accrued in the first season; otherwise, the study will continue to a second season.

Oncology therapeutics:

Individualized Neoantigen Therapy (INT): Moderna continues to demonstrate the potential clinical benefit of its individualized neoantigen therapy (INT) (mRNA-4157). Moderna and Merck rapidly expanded clinical studies to additional tumor types and the Phase 3 clinical trial for adjuvant melanoma completed enrollment in 2024.

Rare disease therapeutics:

Propionic acidemia (PA) therapeutic: In an ongoing Phase 1/2 study designed to evaluate safety and pharmacology in trial participants with PA, Moderna’s investigational therapeutic (mRNA-3927) has been generally well-tolerated to date with no events meeting protocol-defined dose-limiting toxicity criteria. Early results suggest potential decreases in annualized metabolic decompensation event (MDE) frequency compared to pre-treatment, and the majority of patients have elected to continue on the open label extension study. The Company began generating registrational trial data in 2024.


Methylmalonic acidemia (MMA) therapeutic: Moderna’s investigational therapeutic for MMA (mRNA-3705) has been selected by the FDA for the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program. The FDA and Moderna have agreed on the pivotal study design. The Company expects to start a registrational study in the first half of 2025.

Key 2025 Investor and Analyst Event Dates

Fourth Quarter and Fiscal Year 2024 Earnings Call: February 14, 2025

Analyst Day: November 20, 2025

On January 13, 2025 Arbutus Biopharma Corporation (Nasdaq: ABUS) ("Arbutus" or the "Company"), a clinical-stage biopharmaceutical company leveraging its extensive virology expertise to develop a functional cure for people with chronic hepatitis B virus (cHBV) infection, reported its 2025 corporate objectives and provided a financial update (Press release, Arbutus Biopharma, JAN 13, 2025, View Source [SID1234649716]).

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"We enter 2025 with solid financial footing and strong momentum in achieving our mission of developing a functional cure for cHBV, a disease that affects more than 250 million people worldwide and is a leading cause of liver cancer," said Michael J. McElhaugh, Interim President and Chief Executive Officer of Arbutus. "The data we reported late last year from our IM-PROVE I Phase 2a clinical trial showed a meaningful functional cure rate and immune activation in cHBV patients that were treated with our RNAi therapeutic, imdusiran, interferon and nucleos(t)ide analogue (NA) therapy. These data support our belief that imdusiran is differentiated from other RNAi therapeutics in development for HBV. Therefore, we plan to initiate a Phase 2b clinical trial combining imdusiran, interferon and NA therapy in the first half of 2025."

Imdusiran (RNAi therapeutic)

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 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 combination of imdusiran, IFN and NA therapy was generally safe and well-tolerated.
Based on this data, the Company is planning to initiate a placebo-controlled Phase 2b clinical trial with this treatment regimen in the first half of 2025. Subject to regulatory approval, the clinical trial is anticipated to enroll approximately 170 HBeAg-negative cHBV patients with baseline HBsAg ≤1000 IU/mL. Additional details will be provided by the Company after regulatory approval.
The Company also presented data from its IM-PROVE II Phase 2a clinical trial 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. 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 and anticipates reporting data in the first half of 2025.
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 (PK), and pharmacodynamics (PD) 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, Arbutus has moved into Part 3 which evaluates repeat doses of AB-101 for 28 days in patients with cHBV. Data from the 10 mg cohort is expected in the first half of 2025. Next steps for AB-101 will be determined after Arbutus evaluates data from Part 3 of this clinical trial.
LNP Litigation

Arbutus will continue to protect and defend its intellectual property, which is the subject of the on-going lawsuits against Moderna and Pfizer/BioNTech. The Company is seeking fair compensation for Moderna’s and Pfizer/BioNTech’s use of its patented LNP technology that was developed with great effort and at a great expense, 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 on December 18, 2024. The court is expected to provide its ruling on the claim construction and issue the scheduling order in the first half of 2025.
The Moderna trial date is scheduled for September 24, 2025, and is subject to the court’s availability. Expert reports and expert depositions continue in this lawsuit.
Financial Update:

The Company had cash, cash equivalents and investments in marketable securities totaling approximately $123 million as of December 31, 2024 (unaudited).
The Company expects to significantly reduce its net cash burn in 2025 when compared to 2024. Net cash burn is expected to range from $47 to $50 million in 2025 versus a 2024 net cash burn of approximately $65 million (unaudited). The Company believes its cash, cash equivalents, investments in marketable securities and anticipated contractual milestones from Qilu Pharmaceutical, its strategic partner in Greater China, are sufficient to fund its operations through the first quarter of 2028. This includes fully funding the imdusiran Phase 2b clinical trial.
The preliminary cash, cash equivalents and investments as of December 31, 2024 and the estimated 2024 net cash burn were calculated prior to the completion of an audit by Arbutus’ independent registered public accounting firm and are therefore subject to adjustment.
With its current cash balance and anticipated 2025 net cash burn, the Company does not anticipate utilizing its "at-the-market" program (ATM) this year.
About Imdusiran

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. In the first half of 2025, the Company is planning to initiate a Phase 2b clinical trial of imdusiran combined with IFN and NA therapy.

About AB-101

AB-101 is our oral PD-L1 inhibitor candidate that we believe will 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. We believe AB-101, when used in combination with other approved and investigational agents, could potentially lead to a functional cure in patients chronically infected with HBV. 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.