ibrx-20251231
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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 10-K
(Mark One)
For the fiscal year ended December 31, 2025
or
For the transition period from to
Commission file number: 001-37507
_____________________________________
IMMUNITYBIO, INC.
(Exact name of registrant as specified in its charter)
3530 John Hopkins CourtSan Diego, California 92121
(Address of principal executive offices) (Zip Code)
Registrant’s telephone number, including area code: (844) 696-5235
Securities registered pursuant to Section 12(b) of the Act:
Title of each class Trading Symbol(s) Name of each exchange on which registered
Common Stock, par value $0.0001 per share IBRX The Nasdaq Global Select Market
Securities registered pursuant to Section 12(g) of the Act:
None
Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. Yesþ No ̈
Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or Section 15(d) of the Act. Yes ̈Noþ
Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yesþ No ̈
Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yesþ No ̈
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.
Large accelerated filer þ Accelerated filer ̈
Non-accelerated filer ̈ Smaller reporting company ̈
Emerging growth company ̈
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ̈
Indicate by check mark whether the registrant has filed a report on and attestation to its management’s assessment of the effectiveness of its internal control over financial reporting under Section 404(b) of the Sarbanes-Oxley Act (15 U.S.C. 7262(b)) by the registered public accounting firm that prepared or issued its audit report. ☑
If securities are registered pursuant to Section 12(b) of the Act, indicate by check mark whether the financial statements of the registrant included in the filing reflect the correction of an error to previously issued financial statements. ̈
Indicate by check mark whether any of those error corrections are restatements that required a recovery analysis of incentive-based compensation received by any of the registrant’s executive officers during the relevant recovery period pursuant to §240.10D-1(b). ̈
Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Act). Yes ̈ No þ
The aggregate market value of the registrant’s voting and non-voting common equity held by non-affiliates, computed based on the closing price of shares of common stock on the Nasdaq Global Select Market on June 30, 2025 was approximately $690.2 million.
The number of shares of the registrant’s common stock outstanding as of February 19, 2026 was 1,028,111,456 (excluding 163,800 shares held by a majority-owned subsidiary of ours that are treated as treasury shares for accounting purposes).
DOCUMENTS INCORPORATED BY REFERENCE
As noted herein, the information called for by Part III of this Annual Report is incorporated by reference to specified portions of the registrant’s definitive proxy statement to be filed in conjunction with the registrant’s 2026 Annual Meeting of Stockholders, which is expected to be filed not later than 120 days after the registrant’s fiscal year ended December 31, 2025.
IMMUNITYBIO, INC.
ANNUAL REPORT ON FORM 10-K
FOR THE YEAR ENDED DECEMBER 31, 2025
TABLE OF CONTENTS
Page
PART I
Item 1. Business. 1
Item 1A. Risk Factors. 55
Item 1B. Unresolved Staff Comments. 128
Item 1C. Cybersecurity. 129
Item 2. Properties. 131
Item 3. Legal Proceedings. 131
Item 4. Mine Safety Disclosures. 134
PART II
Item 6. Reserved. 136
Item 7A. Quantitative and Qualitative Disclosures About Market Risk. 158
Item 8. Financial Statements and Supplementary Data. 160
Item 9A. Controls and Procedures. 238
Item 9B. Other Information. 241
PART III
Item 10. Directors, Executive Officers and Corporate Governance. 242
Item 11. Executive Compensation. 242
Item 14. Principal Accountant Fees and Services. 242
PART IV
Item 15. Exhibits and Financial Statement Schedules. 243
Defined Terms
Unless expressly indicated or the context required otherwise, the terms “ImmunityBio,” “the company,” “we,” “us,” and “our” in this Annual Report refer to ImmunityBio, Inc., a Delaware corporation, and, where appropriate, its subsidiaries. We have also used several other terms in this Annual Report, the consolidated financial statements and accompanying notes included herein, most of which are defined below:
Term Definition
2015 Plan ImmunityBio, Inc. 2015 Equity Incentive Plan
2025 Plan ImmunityBio, Inc. 2025 Equity Incentive Plan
3M IPC 3M Innovative Properties Company
3PL Agent logistics agent
401(k) Plan 401(k) retirement and savings plan
AAHI Access to Advanced Health Institute
ACA Affordable Care Act
ADCC antibody-dependent cellular cytotoxicity
AI artificial intelligence
AKS Anti-Kickback Statute
ALC absolute lymphocyte count
Altor Altor BioScience, LLC
America Invents Act Leahy-Smith America Invents Act
Amyris Amyris, Inc.
ANC absolute neutrophil count
Annual Report Annual Report on Form 10-K for the year ended December 31, 2025
Approved product ANKTIVA
ART anti-retroviral therapy
ASC Accounting Standards Codification
ASC 470-50 FASB ASC Topic 470-50, Debt—Modifications and Extinguishments
ASC 480 FASB ASC Topic 480, Distinguishing Liabilities from Equity
ASC 606 FASB ASC Topic 606, Revenue from Contracts with Customers
ASC 718 FASB ASC Topic 718, Compensation—Stock Compensation
ASC 808 FASB ASC Topic 808, Collaborative Arrangements
ASC 815 FASB ASC Topic 815, Derivatives and Hedging
ASC 825 FASB ASC Topic 825, Financial Instruments
ASCO American Society of Clinical Oncology
ASU Accounting Standards Update
Athenex Athenex, Inc.
ATM “at-the-market” sales agreement
ATRA American Taxpayer Relief Act of 2012
i
Term Definition
BCG Bacillus Calmette-Guérin (TICE® BCG approved for use in the U.S.)
Beike Shenzhen Beike Biotechnology Co. Ltd.
BeOne BeOne Medicines Ltd. (formerly BeiGene, Ltd.)
BIS U.S. Department of Commerce, Bureau of Industry and Security
BLA Biologics License Application
BPCIA Biologics Price Competition and Innovation Act of 2009
Brink Brink Biologics, Inc.
Cambridge Cambridge Equities, LP
CAP community-acquired pneumonia
CAR chimeric antigen receptor
CCPA California Consumer Privacy Act of 2018
CEO chief executive officer
CFO chief financial officer
cGMP current Good Manufacturing Practice
CHMP Committee for Medicinal Products for Human Use
CI confidence interval
CIO chief information officer
CIS carcinoma in situ
Clinic Immuno-Oncology Clinic, Inc.
Closing Date when used in connection with the RIPA, December 29, 2023
CMO contract manufacturing organization
CMS Centers for Medicare & Medicaid Services
Code Internal Revenue Code of 1986, as amended
CODM chief operating decision maker
CPI checkpoint inhibitor
CPRA California Privacy Rights Act
CRADA Cooperative Research and Development Agreement
CRL complete response letter
CRO contract research organization
CVR contingent value right
DGCL Delaware General Corporation Law
DSCSA Drug Supply Chain Security Act
Duley Road Duley Road, LLC
EAP Expanded Access Program
EEA European Economic Area
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Term Definition
EGFR epidermal growth factor receptor
EMA European Medicines Agency
ERM Enterprise Risk Management
EU European Union
Exchange Act Securities Exchange Act of 1934, as amended
Exyte Exyte U.S., Inc.
FASB Financial Accounting Standards Board
FCA False Claims Act
FCPA U.S. Foreign Corrupt Practices Act
FD&C Act Federal Food, Drug, and Cosmetic Act
FDA U.S. Food and Drug Administration (or the Agency)
FDASIA Food and Drug Administration Safety and Innovation Act of 2012
FIFO First In First Out inventory method
FTC Federal Trade Commission
FTO freedom-to-operate
FVO fair value option
GBM glioblastoma
GCP Good Clinical Practice
GDPR General Data Protection Regulation
GlobeImmune GlobeImmune, Inc.
GMP Good Manufacturing Practice
hAd5 human adenovirus serotype 5
haNK high-affinity NK
Hatch-Waxman Act Drug Price Competition and Patent Term Restoration Act of 1984
HCC hepatocellular carcinoma
HCW HCW Biologics, Inc.
HHS U.S. Department of Health and Human Services
HIPAA Health Insurance Portability and Accountability Act of 1996
HITECH Health Information Technology for Economic and Clinical Health Act
HIV human immunodeficiency virus
HPV human papillomavirus
IDE Investigational Device Exemption
IgDraSol IgDraSol, Inc., a subsidiary of the company
IL-15 novel interleukin 15
IND investigational new drug
Infinity Infinity SA LLC, as purchaser agent for affiliates of Oberland
iNHL indolent non-Hodgkin lymphoma
IPR&D In-process research and development
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Term Definition
iPSP initial Pediatric Study Plan
IRA Inflation Reduction Act of 2022
IRB Institutional review boards
IRS Internal Revenue Service
LMIC low- and middle-income countries
M-ceNK memory-like cytokine-enhanced NK
MAA Marketing Authorization Application
mAbs monoclonal antibodies
mCRCP metastatic castration-resistant prostate cancer
MDSC myeloid-derived suppressor cells
MENA Middle East and North Africa
MFN Most Favored Nation drug
MHC major histocompatability complex
MHC-I major histocompatability complex class I
MHC-II major histocompatability complex class II
MHRA UK Medicines and Healthcare products Regulatory Agency
Nant Capital Nant Capital, LLC
NantCell NantCell, Inc., a subsidiary of the company
NANTibody Immunotherapy NANTibody, LLC, a subsidiary of the company
NantKwest NantKwest, Inc.
NantMobile NantMobile, LLC
NantWorks NantWorks, LLC, a related party
NCCN National Comprehensive Cancer Network
NC 2015 Plan NantCell, Inc. 2015 Stock Incentive Plan
NCI National Cancer Institute
NCSC NantCancerStemCell, LLC
NDA New Drug Application
NHL non-Hodgkin lymphoma
NIAID National Institute of Allergy and Infectious Diseases
NIH National Institutes of Health
NIH Guidelines NIH Guidelines for Research Involving Recombinant DNA Molecules
NK natural killer
NMIBC non-muscle invasive bladder cancer
NOL net operating loss
NSCLC non-small cell lung cancer
OBA NIH Office of Biotechnology Activities
OBBB Act One Big Beautiful Bill Act
OECD Organisation for Economic Co-operation and Development
PCAOB Public Company Accounting Oversight Board (United States)
PD-1 programmed death receptor 1
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Term Definition
PD-L1 programmed death receptor ligand
PDMA U.S. Prescription Drug Marketing Act
PHI Protected Health Information
PHSA Public Health Service Act
PMA pre-market approval
PREA Pediatric Research Equity Act
PSA prostate-specific antigen
QMSR Quality Management System Regulation
QSR Quality System Regulation
QUILT QUantum Integrative Lifelong Trial
R&E research and experimental expenditures
r/r relapsed/ refractory
RAC Recombinant DNA Advisory Committee
rBCG recombinant BCG
RCT randomized controlled trial
RDO registered direct offering
REMS Risk Evaluation and Mitigation Strategy
RIPA Revenue Interest Purchase Agreement
Riptide Riptide Bioscience, Inc.
RMAT regenerative medicine advanced therapy
RSU restricted stock unit
RTF Refuse to File
SAFE Simple Agreement for Future Equity
Sarbanes-Oxley Sarbanes-Oxley Act of 2002
SARS-CoV-2 novel strain of the coronavirus (COVID-19)
sBLA supplemental Biologics License Application
SEC U.S. Securities and Exchange Commission
Section 404 Section 404 of the Sarbanes-Oxley Act of 2002
Securities Act Securities Act of 1933, as amended
Serum Institute Serum Institute of India Private Limited
SFDA Saudi Food and Drug Authority
Sorrento Sorrento Therapeutics, Inc.
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Term Definition
SPOA Stock Purchase and Option Agreement
SRLY separate return limitation year
TAA tumor-associated antigen
TCJA Tax Cuts and Jobs Act of 2017
Term SOFR Term Secured Overnight Financing Rate
Test Date when used in connection with the RIPA, December 31, 2029
TGF-β transforming growth factor beta
t-haNK targeted high-affinity NK (CAR-NK)
TLR toll-like receptor
TNBC triple-negative breast cancer
Tregs regulatory T cells
TriAd Triple Antigen (CEA, MUC1, Brachyury)
TTFields tumor treating fields
UK United Kingdom
UK GDPR UK Data Protection Act of 2018
U.S. United States of America
U.S. GAAP accounting principles generally accepted in the U.S.
USMCA United States-Mexico-Canada Agreement
USPTO U.S. Patent and Trademark Office
VBC Holdings VBC Holdings, LLC, a subsidiary of the company
VIE variable interest entity
VivaBioCell VivaBioCell, S.p.A., a wholly-owned subsidiary of VBC Holdings
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PART I
ITEM 1. BUSINESS.
Forward-Looking Statements
This Annual Report contains forward-looking statements within the meaning of Section 27A of the Securities Act and Section 21E of the Exchange Act that are based on our management’s beliefs and assumptions and on information currently available to our management. Forward-looking statements include, but are not limited to:
•our ability to successfully commercialize ANKTIVA globally in NMIBC, NSCLC or other indications or any future approved products in the U.S. or internationally;
•our ability to obtain incremental approvals for ANKTIVA for new indications, including, without limitation, in BCG-unresponsive NMIBC with papillary tumors and NSCLC from the FDA or clearances or approvals from international regulatory agencies for the treatment of patients with NMIBC, NSCLC or other indications;
•potential future uses and applications of ANKTIVA, including as a lymphopenia rescue agent in solid tumors or other indications, and use in cancer vaccines and across multiple tumor types;
•our ability to develop next-generation therapies and vaccines that complement, harness, and amplify the immune system to defeat cancers and infectious diseases;
•our ability to obtain additional financing to fund our operations and advance the commercialization of our approved product and the development and commercialization of our other product candidates;
•our ability to comply with the terms, conditions, covenants, restrictions, and obligations set forth in the RIPA and related transaction documents, including payment obligations and servicing the interest on our related-party promissory note and the repayment of such note, to the extent required;
•our expectations regarding the potential benefits of our Cancer BioShield platform (comprises multiple therapeutic modalities to activate immune response) and our strategy and technology;
•our ability to forecast operating results and make period-to-period comparisons predictive of future performance due to fluctuations in warrant, derivative, and fair value accounting measurement values;
•our expectations regarding the operation and effectiveness of our approved product and product candidates and related benefits;
•our ability to utilize multiple modes to induce cell death in cancers and infectious disease;
•our beliefs regarding the benefits and perceived limitations of competing approaches, and the future of competing technologies and our industry;
•our beliefs regarding the success, cost and timing of our approved product and product candidate development activities and current and future clinical trials and studies, including study design and the enrollment of patients;
•whether the NCCN will add recommending ANKTIVA for BCG-unresponsive NMIBC with papillary tumors in addition to the existing recommendation for BCG-unresponsive NMIBC CIS with or without papillary tumors after our submission on the anticipated timeline or at all;
•our expectations regarding our ability to utilize the Phase 1/2 aNK, haNK®, taNK, and NK-CAR (t-haNKTM) clinical trials data to support the development of our product candidates, including our NK-CAR (t‐haNK), MSC, and M-ceNKTM product candidates;
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•our expectations regarding the development, clinical trials timeline, application, commercialization, marketing, prospects and use generally of our product candidates, including hAd5 constructs, and PD-L1 t‐haNK and M-ceNK;
•the timing or likelihood of regulatory filings or other actions and related regulatory authority responses in the U.S. and jurisdictions outside of the U.S., including any planned meetings, IND, BLA, NDA or MAA or similar filings or pursuit of accelerated regulatory approval pathways or orphan drug status and Breakthrough Therapy, Fast Track or RMAT designations and any designation’s impact on BLA submission filing or approval timing and or approval probability;
•our ability to successfully address the May 2025 RTF letter received from the FDA for the sBLA for the BCG-unresponsive NMIBC with papillary tumors indication and the recommendation from the FDA to submit additional information in support of filing for review of the sBLA, and whether or not there is a need for the initiation or design of a new RCT, which we could ultimately be required to complete;
•our ability to implement an integrated discovery ecosystem and the operation of that planned ecosystem, including being able to regularly add neoepitopes and subsequently formulate new product candidates;
•the ability and willingness of strategic collaborators to share our vision and effectively work with us to achieve our goals;
•the ability and willingness of various third parties to engage in research and development activities involving our product or product candidates, and our ability to leverage those activities;
•our ability to attract additional third-party collaborators;
•our ability to enter into clinical, regulatory and commercial arrangements internationally to accelerate the development and commercialization of ANKTIVA and our other product candidates;
•our expectations regarding the ease of administration associated with our approved product and product candidates;
•our expectations regarding patient compatibility associated with our approved product and product candidates;
•our beliefs regarding the potential markets for our approved product and product candidates and our ability to serve those markets;
•our expectations regarding the timing of enrollment and submission of our clinical trials, and protocols and timing of data read-outs related to such trials;
•our ability to produce a cytokine fusion protein, a DNA or recombinant protein vaccine, or a cell therapy;
•our beliefs regarding the potential manufacturing and distribution benefits associated with our approved product and product candidates, and our third-party CMOs’ abilities to follow cGMP standards to scale up the production of our approved product and product candidates;
•our plans regarding manufacturing and distribution of our approved product and potential future products, including the enhancement of our in-house manufacturing capabilities;
•our belief in the potential of our cytokine fusion proteins, DNA or recombinant protein vaccines, or cell therapies, and the fact that our business is based upon the success individually and collectively of these platforms;
•our belief regarding the magnitude or duration for additional clinical testing of our cytokine fusion proteins, DNA or recombinant protein vaccines, or cell therapies, along with other product candidate families;
•even if we successfully develop and commercialize specific product candidates, our ability to develop and commercialize our other product candidates either alone or in combination with other therapeutic agents;
•the ability to obtain and maintain regulatory approval of our approved product and to obtain and maintain regulatory approval of any of our other product candidates, and any related restrictions, limitations and/or warnings in the label of any approved product candidate;
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•the rate and degree of market acceptance of any approved products;
•our ability to attract and retain key personnel;
•the accuracy of our estimates regarding our future revenue, as well as our future operating expenses, capital requirements and needs for additional financing;
•our ability to obtain, maintain, protect, and enforce patent protection and other proprietary rights for our approved product and our other product candidates, and other technologies in development;
•the terms and conditions of licenses granted to us and our ability to license additional intellectual property relating to our product, product candidates and technology;
•our expectations regarding the results of market access initiatives and coverage under medical reimbursement policies;
•our expectations regarding the abilities of our international partners to drive commercialization of ANKTIVA in the EU and MENA regions;
•shelf life of ANKTIVA drug substance and drug product and availability of product supply;
•our global expansion efforts and the accuracy of our assumptions related to tariffs and government policy changes, including Most-Favored Nation Prescription Drug Pricing;
•any government shutdown or budget disruption, which could adversely affect the U.S. and global economies, and materially and adversely affect our business and/or our future BLA submissions;
•the impact on us, if any, if the CVRs held by former Altor stockholders become due and payable; and
•regulatory developments in the U.S. and foreign countries.
Forward-looking statements include statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “continues,” “goal,” “could,” “estimates,” “scheduled,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “indicate,” “projects,” “seeks,” “should,” “will,” “would,” “strategy,” and variations of such words or similar expressions. and the negatives of those terms. In addition, statements that “we believe” and similar statements reflect our beliefs and opinions on the relevant subject. Statements of past performance, efforts, or results of our preclinical and clinical trials, about which inferences or assumptions may be made, can also be forward-looking statements and are not indicative of future performance or results. These statements are based upon information available to us as of the date of this Annual Report, and although we believe such information forms a reasonable basis for such statements, such information may be limited or incomplete, and our statements should not be read to indicate that we have conducted a thorough inquiry into, or review of, all potentially available relevant information. These statements are inherently uncertain, and investors are cautioned not to unduly rely upon these statements.
This Annual Report also contains estimates, projections and other information concerning our industry, our business, and the markets for certain diseases, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions. Information that is based on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties and actual events or circumstances may differ materially from events and circumstances reflected in this information. Unless otherwise expressly stated, we obtained this industry, business, market, and other data from reports, research surveys, studies, and similar data prepared by market research firms and other third parties, industry, medical and general publications, government data, and similar sources.
Forward-looking statements involve known and unknown risks, uncertainties, and other factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. We discuss these risks in greater detail in Item 1A. “Risk Factors” of this Annual Report. Given these uncertainties, you should not place undue reliance on these forward-looking statements as a representation or warranty by us or any other person that we will achieve our objectives and plans in any specified time frame. Also, forward-looking statements represent our management’s beliefs and assumptions only as of the date of this Annual Report.
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Except as required by law, we assume no obligation to update these forward-looking statements, or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future. You should read this Annual Report completely and with the understanding that our actual future results may be materially different from what we expect.
ImmunityBio, ImmunityBio Care, ANKTIVA, ThAnktiva, haNK, taNK, ceNK, NK-92, Nant Cancer Vaccine, BioShield (and other BioShield-related trademarks), NANT 001, NANT XL, NANT 001 and Design, QUILT, Outsmart Your Disease, Smart Therapies for Difficult Diseases, NantKwest, VivaBioCell, and Infacell are trademarks or registered trademarks of ImmunityBio, Inc., its subsidiaries and affiliates.
ANKTIVA has been approved by the U.S. FDA, the UK MHRA, and K.S.A. SFDA and has been granted conditional marketing authorization from the EC (EMA) for the EU and Iceland, Liechtenstein, and Norway for use with BCG for the treatment of adult patients with BCG-unresponsive NMIBC CIS with or without papillary tumors. It has also been conditionally approved by the SFDA in Saudi Arabia for use with CPIs in metastatic NSCLC. Other than as set forth in such specific approved label, our product candidates, including N-803, are investigational agents that are restricted by federal law to investigational use only, and safety and efficacy have not been established by any agency, including the FDA.
This Annual Report contains references to our products and trademarks and products and trademarks belonging to other entities. Solely for convenience, trademarks and trade names referred to in this Annual Report, including logos, artwork, and other visual displays, may appear without the ® or TM symbols, but such references are not intended to indicate, in any way, that we will not assert, to the fullest extent under applicable law, our rights or the rights of the applicable licensor to these trademarks and trade names. We do not intend our use or display of other companies’ products or trade names or trademarks to imply a relationship with, or endorsement or sponsorship of us, by any other companies.
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BUSINESS
OVERVIEW
ImmunityBio, Inc. (“ImmunityBio,” the “Company,” “we,” “us,” or “our”) is a biotechnology company focused on innovating, developing, and commercializing next-generation immunotherapies designed to activate the patient’s immune system and deliver durable protection against cancer and infectious diseases. Our approach harnesses both the adaptive and innate immune systems with the goal of restoring immune function and generating lasting immunological memory in patients. At the core of our strategy is the Cancer BioShieldTM platform, which is designed to stimulate critical lymphocytes, including natural killer (NK) cells, cytotoxic T cells, and memory T cells via our proprietary IL-15 superagonist, ANKTIVA® (nogapendekin alfa inbakicept). Our Cancer BioShield platform is anchored by this antibody-cytokine fusion protein and is complemented by a portfolio that includes adenovirus-vectored vaccines, allogeneic (off-the-shelf) and autologous NK-cell therapies, and additional immunomodulators intended to promote immunogenic cell death and support durable immune responses while potentially reducing reliance on high-dose chemo-radiation therapy.
ANKTIVA is our lead biologic product and a first-in-class IL-15 receptor superagonist antibody-cytokine fusion protein. We are commercializing ANKTIVA for the treatment of BCG-unresponsive NMIBC CIS with or without papillary tumors. ANKTIVA has received FDA Breakthrough Therapy designation for use in BCG-unresponsive NMIBC CIS in adult patients with or without papillary tumors.
ANKTIVA is now approved in the U.S., UK, and Saudi Arabia for BCG-unresponsive NMIBC CIS with or without papillary tumors. In February 2026, the European Commission granted conditional marketing authorization in the EU for ANKTIVA for the same indication. In addition, ANKTIVA is conditionally approved in Saudi Arabia, for use in combination with a CPI, for the treatment of adult patients with metastatic NSCLC whose disease has progressed following standard-of-care therapy. The approved labels highlight ANKTIVA’s ability to simultaneously activate NK cells, cytotoxic T cells, and memory T cells.
ANKTIVA in combination with our CAR-NK therapy (PD-L1 t-haNK) has received RMAT designation from the FDA for use in combination with standard-of-care chemotherapy/radiotherapy for the reversal of lymphopenia and treatment of multiply relapsed locally advanced or metastatic pancreatic cancer. Separately, the FDA has authorized an EAP for ANKTIVA to treat lymphopenia in adult patients with refractory or relapsed solid tumors, regardless of tumor type, who have progressed following first-line standard-of-care treatment, including chemotherapy, radiation, or immunotherapy. The EAP includes patients with solid tumors who have failed first-line therapy and have a low ALC (ALC <1,000/μL).
We are seeking to expand development of ANKTIVA in combination with current standard-of-care therapies across multiple solid and liquid tumor indications including:
•BCG-naïve NMIBC;
•BCG-unresponsive NMIBC with papillary tumors;
•Second-line NSCLC;
•First-line NSCLC;
•Glioblastoma;
•Indolent non-Hodgkin lymphoma, including Waldenström macroglobulinemia;
•Pancreatic cancer;
•Prostate cancer; and
•Ovarian cancer.
In addition, ANKTIVA has been selected by the NCI for evaluation in a cancer prevention study in patients with Lynch syndrome, and we are evaluating the combination of ANKTIVA with other agents in colorectal cancer. The company is also supporting an ongoing EAP for rBCG to help address U.S. supply constraints. We are also establishing or conducting trials in multiple myeloma, HCC, and TNBC.
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Commercial Performance
We began commercial distribution of ANKTIVA in May 2024 following FDA approval. The following table summarizes our commercial performance through December 31, 2025:
Metric Value Period
Net Product Revenue ~$113 million Full Year 2025
Lives Covered >240 million As of December 2025
Reimbursement: ANKTIVA received a permanent J-code (J9028) from CMS, effective January 1, 2025, for “Injection, nogapendekin alfa inbakicept, for intravesical use, 1 microgram.”
Regulatory Approvals
Jurisdiction Indication Status
As of February 2026, ANKTIVA has received regulatory approval in four jurisdictions spanning two therapeutic indications (bladder cancer and lung cancer). To our knowledge, based on publicly available information, the SFDA’s conditional approval of ANKTIVA in combination with CPIs for metastatic NSCLC is the first regulatory approval for an IL-15 receptor superagonist in lung cancer. The European Commission granted conditional marketing authorization for ANKTIVA with BCG in BCG-unresponsive NMIBC CIS with or without papillary tumors, which is the first approval of a therapeutic in this indication in Europe. ANKTIVA is now authorized for use in 33 countries.
SCIENTIFIC RATIONALE
The Lymphopenia Challenge in Oncology
Lymphopenia, defined as the severe depletion of cancer-killing NK and T cells, represents a critical and widely unaddressed problem in oncology. The ALC is a routine measurement in complete blood counts that carries independent prognostic weight across tumor types.
Prevalence: Based on published literature and internal estimates derived from American Cancer Society incidence data and treatment patterns, lymphopenia affects a substantial portion of U.S. cancer patients annually. Standard treatments, including radiotherapy, chemotherapy, steroids, and certain immunotherapies, can induce or worsen this condition.
Clinical Significance:
Mortality Risk of Low ALC in General Population, All Causes & Cardiovascular Disease
In 2019, Zidar et al., published in JAMA, “Association of Lymphopenia With Risk of Mortality Among Adults in the US General Population” and reported that low lymphocyte levels are associated with reduced survival in the U.S. general population. Their study highlighted that “based on these findings, patients with lymphopenia, especially those with other immunohematologic abnormalities, may have excess mortality risk; these patients are readily identifiable because tests of lymphocyte levels often occur during routine medical encounters.” Furthermore, the importance of this finding was emphasized by the following statement, “Immune dysregulation can increase the risk of infection, malignant neoplasms, and cardiovascular disease, but improved methods are needed to identify and quantify immunologic hazard in the general population.”
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Of note, the risk mortality from all causes and from heart and cerebrovascular disease consistently rises when the ALC levels fall from normal to below 2,000. These findings emphasize the need to protect the immune system and maintain ALC levels even in the general population.
The economic significance of increased mortality and reduced longevity is significant. In the Nature publication by Scott et al., entitled, “The Economic Value of Targeting Aging”, the authors concluded that “a slowdown in aging that increases life expectancy by 1 year is worth US$38 trillion, and by 10 years, US$367 trillion. Ultimately, the more progress that is made in improving how we age, the greater the value of further improvements.”
Association of Lymphopenia with Mortality in General Population
Solid lines indicate hazard ratio point estimates; dotted lines, 95% CI
Zidar DA et al., Figure 2. Association of Lymphopenia with Mortality
Zidar DA, Al-Kindi SG, Liu Y, et al. Association of lymphopenia with risk of mortality among adults in the US general population.
JAMA Network Open. 2019;2(12):e1916526. doi:10.1001/jamanetworkopen.2019.16526
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Furthermore, in the Zidar et al., study of over 31,000 Americans, a clear demonstration of survival related to lymphocyte levels is seen in this graph below. When lymphocyte levels fall between 0.3 to 1.5, the survival rate decreases.
Lymphocyte Levels Associated with Survival
Zidar et al., Supplemental Figure 1
Zidar DA, Al-Kindi SG, Liu Y, et al. Association of lymphopenia with risk of mortality among adults in the US general population.
JAMA Network Open. 2019;2(12):e1916526. doi:10.1001/jamanetworkopen.2019.16526
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Mortality Risk with Low ALC in Cancer
Additionally, the risk of death from cancer increases when the absolute lymphocyte count level falls below 2.0.
Association with Lymphopenia with Mortality in Patients with Cancer
Solid lines indicate hazard ratio point estimates; dotted lines, 95% CI
Zidar DA et al., Figure 2. Associated of Lymphopenia with Mortality
Zidar DA, Al-Kindi SG, Liu Y, et al. Association of lymphopenia with risk of mortality among adults in the US general population.
JAMA Network Open. 2019;2(12):e1916526. doi:10.1001/jamanetworkopen.2019.16526
Multiple peer-reviewed studies have shown that severe lymphopenia (ALC <1,000 cells/μL) is associated with a significantly increased risk of mortality in patients with solid tumors. Large retrospective analyses have shown that patients with peripheral lymphopenia experience markedly higher rates of high-grade chemotherapy toxicity and reduced overall survival, with published relative risk estimates for relapse-free survival ranging from 1.35 to 3.81-fold and for overall survival from 1.25 to 7.70-fold compared with patients maintaining adequate lymphocyte levels.
Of note, this association with lymphopenia and statistically increased mortality occurs regardless of tumor type as shown by Ménétrier-Caux C et al., In this publication entitled, “Lymphopenia in Cancer Patients and its Effects on Response to Immunotherapy: an opportunity for combination with Cytokines?”, the authors review “the current knowledge on the incidence and significance of lymphopenia in cancer patients, and discuss therapeutic strategies to restore lymphocyte numbers.”
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Treatment Gap: While oncologists have established supportive therapies such as erythropoietin-stimulating agents and granulocyte colony-stimulating factors to manage chemotherapy-induced anemia and neutropenia, respectively, no FDA-approved therapy has been specifically indicated for the treatment of lymphopenia in cancer patients.
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Interleukin-15 (IL-15) was ranked by the NIH and NCI as the number 1 cytokine with the potential to cure cancer. The findings of this working group was published in 2008 by Cheever M., entitled, “Twelve Immunotherapy Drugs that Could Cure Cancers.”
The NIH undertook decades of research to develop this IL-15 cytokine as a therapeutic and in 2015, the understanding of the shared and contrasting roles of IL-2 versus IL-15 was published by Thomas A. Waldmann, MD (NIH Distinguished Investigator) highlighting the decades long focus on IL-2 and the neglect of IL-15 for therapeutic lymphopenia correction as a consequence of both historical timing and incomplete mechanistic understanding that only recent insights in immunobiology had begun to correct.
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Biological Misunderstanding and Overlap Between IL-2 and IL-15
Pharmacological Development Based on Incorrect Assumption that IL-2 was the Key Cytokine For Cancer Therapy
Discovery and Development of IL-2 and IL-15: IL-2 was discovered in 1976 as “T-cell growth factor,” and by the 1980s, became the first cytokine to be evaluated in humans for cancer immunotherapy. High-dose recombinant IL-2 (aldesleukin) was FDA-approved in the 1990s for metastatic renal cell carcinoma (1992) and metastatic melanoma (1998) which established a precedent and clinical infrastructure around IL-2. IL-15 was only discovered in 1994, nearly two decades after IL-2 and was approved for the treatment of NMIBC in 2024.
Biological Misunderstanding and Overlap Between IL-2 and IL-15 Accounting for the Delay in Development of IL-15: IL-2 and IL-15 share two receptor subunits (IL-2/15Rβ and γc), which led early researchers to assume overlapping biological effects since the receptor expressed on the surface of T cells is commonly referred to as the IL-2 receptor. However, their distinct α-receptors (IL-2Rα vs. IL-15Rα) and modes of presentation (cis vs. trans) result in profoundly different immunological outcomes (see Figure 1 below). IL-2 primarily stimulates Tregs and not NK cells, leading to immune contraction and immune evasion. IL-15, in contrast, preferentially supports CD8+ memory T cells and NK cells while avoiding Treg expansion making it biologically superior for sustained immune responses—but this was only appreciated later through mechanistic studies. This biological misunderstanding partially explains why the treatment of lymphopenia by stimulating NK and T cells to date (2025) remained unaddressed.
Conclusion: The decades-long focus on IL-2 and neglect of IL-15 for therapeutic lymphopenia correction reflect both historical timing and incomplete mechanistic understanding that only recent developments in immunobiology have begun to correct.
With this insight, the cytokine fusion protein ANKTIVA was developed and studies in healthy volunteers demonstrated the mechanism of action of proliferating NK, CD4+ CD8+ T cells without up-regulating Tregs.
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Healthy Volunteers with ANKTIVA (IL-15 Superagonist): These findings were published in the Journal of Immunology (March 2022) showing that ANKTIVA had a greater than 20-fold longer in vivo half-life than native IL-15. Furthermore, NK cells and CD8+ T cells were stimulated with sustained increase over 2 weeks.
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Addressing Cold Tumors and Immune Evasion
Cancer cells can evade immune detection through “immune escape,” particularly by losing MHC-I expression on their cell surface. Tumors lacking MHC-I expression (“cold tumors”) cannot be recognized by T cells, which may limit the efficacy of CPI therapy. This phenomenon has been characterized in published literature as a mechanism of primary and acquired resistance to immunotherapy.
ANKTIVA’s mechanism of action addresses this challenge through several pathways:
1.NK-Cell Activation: ANKTIVA activates NK cells, which recognize and attack cells lacking MHC-I expression through “missing-self” recognition, a well-characterized immune surveillance mechanism.
2.Interferon-gamma Release: Activated NK cells secrete interferon-gamma, which, as shown in published studies, stimulates MHC-I re-expression on tumor cells.
3.Tumor Transformation: Through this mechanism, tumors may be converted from MHC-negative (“cold”) to MHC-positive (“hot”), potentially making them recognizable by T cells.
4.Memory T Cell Generation: ANKTIVA’s IL-15 agonist activity supports the establishment of memory T cells, which may provide long-term immune surveillance.
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Limitations of Current Checkpoint Inhibitor Therapy
The development of CPIs represented a major advance in cancer treatment. However, published data indicate that patient responses may be limited:
•Primary resistance: Approximately 40-60% of patients do not respond to CPI monotherapy, according to published clinical trial data.
•Acquired resistance: Many initial responders eventually experience disease progression.
•Limited efficacy in tumors lacking T cell infiltration.
•Dependence on adequate lymphocyte populations for optimal activity.
ANKTIVA is designed to address these limitations by restoring lymphocyte counts depleted by prior therapies, activating NK cells to target MHC-negative tumor cells, and generating memory T cells to support potentially durable responses. Although ANKTIVA for use in combination with CPIs has received conditional approval from the SFDA in Saudi Arabia for the treatment of adult patients with metastatic NSCLC whose disease has progressed following standard-of-care therapy, there can be no assurance that ANKTIVA will be effective in overcoming CPI-resistance or that it will receive additional regulatory approvals.See Item 1A. “Risk Factors—Our clinical trials may fail to adequately demonstrate the safety and efficacy of our product candidates, which would prevent or delay regulatory approval and commercialization of other product candidates” in this Annual Report.
OUR STRATEGY
We aim to become a leading global immunotherapy company by developing therapies to address serious unmet needs in oncology and infectious diseases. The key elements of our strategy include:
•building commercial infrastructure and expanding partnerships to support the global adoption of ANKTIVA as a backbone lymphocyte-stimulating agent. With FDA approval in April 2024, a permanent J-code (J9028) effective in January 2025, payer coverage exceeding 240 million lives in the U.S., and international approvals in the UK, EU, and Saudi Arabia, we are establishing global access pathways for patients, including new commercial partnerships with Accord Healthcare in the EU, and Cigalah Healthcare and Biopharma in the Middle East;
•accelerating product candidates generated by our Cancer BioShield platform toward potential registration in difficult to treat oncological and infectious disease indications in large market segments, including oncology-induced lymphopenia, bladder, lung and pancreatic cancers, and GBM and NHL;
•continuously refining our pipeline based on clinical data, regulatory feedback, competitive landscape, and resource allocation;
•continuing to prospect, license, and acquire technologies to complement and strengthen our Cancer BioShield platform and product candidates—whether used as single agents or in combination—in order to optimize responses of the innate and adaptive immune systems to generate cellular memory against multiple tumor types and infectious diseases; and
•cultivating new partnerships and expanding collaborations to advance ANKTIVA and our pipeline efficiently.
ANKTIVA: OUR APPROVED PRODUCT
Product Description and Mechanism
ANKTIVA is a first-in-class IL-15 receptor superagonist antibody-cytokine fusion protein. The FDA approved ANKTIVA with BCG in April 2024 for the treatment of adult patients with BCG-unresponsive NMIBC CIS with or without papillary tumors.
ANKTIVA was approved with a label indicating an immunological mechanism of action that proliferates and activates NK cells, CD8+ T cells, and memory T cells without the proliferation of immunosuppressive Tregs.
The cytokine IL-15 supports the immune system by influencing the development, maintenance, and function of NK cells and CD8+ killer T cells. ANKTIVA consists of an IL-15 mutant (IL-15N72D) fused with an IL-15Rα , which binds with high affinity to IL-15 receptors on NK, CD4+, and CD8+ T cells.
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Pharmacokinetic and Pharmacodynamic Properties
QUILT-1004 (Healthy Volunteers): A Phase 1 study in 14 healthy adult volunteers evaluated the pharmacokinetic, safety, and immunologic profile of ANKTIVA. Subjects received two subcutaneous doses (10 μg/kg followed 24 days later by 20 μg/kg) and were monitored for nine consecutive days after each dose.
Key findings from this study:
•Serum half-life of approximately 20 hours (compared to <1 hour for recombinant IL-15)
•Peak concentrations occurring 10-15 hours post-dose
•Well-tolerated, with the most common adverse events being mild injection-site reactions, chills, and pyrexia
•No grade 3 or higher or serious adverse events reported
•Greater than 3-fold increase in absolute NK cell count persisting for 24 days or more
•22-fold rise in proliferating NK cells (Ki-67 staining)
•27-fold increase in CD8+ T cells
•11-fold rise in CD4+ T cells
•No expansion of immunosuppressive Tregs
•Mean ALC exceeding 1,200 cells/μL in the majority of participants
These pharmacodynamic outcomes in healthy volunteers provide mechanistic support for the lymphopenia-reversal observed in the Phase 2 QUILT-3.055 NSCLC study and other clinical programs.
In January 2026, we announced results from two studies in 151 patients spanning first-, second-, and later-line disease in NSCLC, showing that ANKTIVA demonstrated statistically significant immune restoration and a consistent association between lymphocyte recovery and improved survival in checkpoint-experienced patients.
•In second- and later-line patients, responders (defined as achievement or maintenance of an on-treatment ALC ≥ 1.0 × 103/μL based on a mean on treatment ALC ≥ 1.0 × 103/μL) experienced significantly longer overall survival compared with non-responders (median overall survival 16.2 vs 11.8 months; hazard ratio 0.52; p=0.0369), directly linking immune restoration to clinical benefit.
•Patients achieving higher immune competence (ALC ≥1.2 ×103 cells/μL) demonstrated additional survival benefit, with median overall survival of 21.1 months (hazard ratio 0.33; p=0.0009), independent of PD-L1 status, exceeding historical overall survival of 7-9 months with standard-of-care chemotherapy.
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OUR CANCER BIOSHIELD PLATFORM
Our Cancer BioShield platform comprises multiple therapeutic modalities designed to activate both innate and adaptive immune responses:
Platform Components Addressing the Immune System
Platform Component Key Modalities
Cytokine Fusion Proteins
Cytokine fusion proteins, such as ANKTIVA, represent a novel class of biologics that improve immune responses by enhancing the therapeutic potential of cytokines and promoting lymphocyte infiltration at a site of disease. The cytokine IL-15 plays a crucial role in the immune system by affecting the development, maintenance, and function of key immune cells—NK and CD8+ killer T cells—that are involved in killing cancer cells. ANKTIVA is a first-in-class IL-15 receptor superagonist IgG1 fusion complex, consisting of an IL-15 mutant (IL-15N72D) fused with an IL-15Rα, which binds with high affinity to IL-15 receptors on
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NK, CD4+, and CD8+ T cells. This fusion complex of ANKTIVA, which confers stability and longer half-life than recombinant or native IL-15, mimics the natural biological properties of the membrane-bound IL-15Rα, delivering IL-15 by dendritic cells and drives the activation and proliferation of NK cells with the generation of memory killer T cells that have retained immune memory against these tumor clones. By activating NK cells, ANKTIVA overcomes the tumor escape phase of clones resistant to T cells without stimulating immunosuppressive Tregs and restores memory T cell activity with resultant prolonged duration of complete response. Further, by stimulating the release of interferon-γ, ANKTIVA restores MHC-I expression, making more tumor cells targets for T cell killing. As evidenced by its ability to increase lymphocyte counts in healthy adults in Phase 1 testing, ANKTIVA also has the potential to rescue lymphopenia, which is associated with poor prognosis in cancer before treatment or as a consequence of chemo or radiation therapy.
ANKTIVA’s mechanisms-of-action make it an ideal ‘backbone’ for combination therapy with the company’s platforms, such as second-generation hAd5 vaccines, off-the-shelf CAR-engineered NK cells, and M-ceNK cells, as well as other therapeutics including BCG, targeted antibodies, and CPIs.
Leveraging ANKTIVA’s success, we are developing multi-functional cytokine fusion proteins targeting TGF-ß, PD-L1, CD16, CD20, and comprising IL-12, IL-15, and IL-21, amongst others, to further enhance NK and T cell activation directed to the tumor microenvironment or virally-infected cells and to modulate the systemic and local immune response to accelerate immunogenic cell death.
DNA and Vaccine Vectors
We have developed and/or acquired rights to multiple vaccine delivery technologies for oncology to deliver common TAAs, and neoepitopes (expressed only by cancer cells) and for infectious diseases to target key viruses. These technologies can deliver DNA and protein subunits to induce B and T cell memory through activation of both CD4+ and CD8+ T cells along with antibody (humoral) responses.
Adenovirus is a well-established vector that can be utilized as a vaccine platform to stimulate the immune system, however there is risk for a treated person to develop anti-adenovirus immunity. Our second generation hAd5 vector has unique deletions in the early 1 (E1), early 2 (E2b), and early 3 (E3) regions (hAd5 [E1-, E2b-, E3-]), which allows it to be effective in the presence of pre-existing adenovirus immunity and lowers the risk of generating de novo vector-directed immunity. We have developed several hAd5 candidates that have been evaluated in multiple clinical trials as potential vaccines for and treatments of certain cancers and infectious diseases. Importantly, these product candidates have shown an ability to overcome previous adenovirus immunity in preclinical models and in cancer patients. In oncology, we are clinically evaluating hAd5 candidates in combination with ANKTIVA to yield immunological immunity in colon cancer (hAd5 TAAs CEA, MUC1, Brachyury; collectively the TriAd) and prostate cancer (hAd5 PSA), and as a single agent in HPV-associated cancers (hAd5 TAA [E6/E7]).
Cell Therapies
We believe that our NK cell-based platforms are some of the most advanced at the clinical-stage of development in oncology. NK cells are a type of cytotoxic lymphocyte critical to the innate immune system–they play a key role targeting tumor and virally-infected cells. Activated NK cells secrete several key cytokines (IFN-γ, TNF-α, GM‐CSF) and chemokines that can modulate the function of other innate and adaptive immune cells. Our NK platform works through innate, antibody-mediated, and CAR-directed killing mechanisms.
Off-The-Shelf Targeted CAR-NK Cells
Key attributes of our CAR-NK assets include:
•cytotoxicity and cytokine-directed cell expansion
•absence of key inhibitory receptors to maintain killing function
•ADCC-targeted killing through high-affinity CD16 (Jochems 2016) – allowing engagement with IgG1-type antibodies (e.g., BAVENCIO®, trastuzumab, etc.)
•CAR receptors such as PD-L1 and CD-19
◦PD-L1 CAR-NK (PD-L1 t-haNK): potent antitumor effects against MDSCs and overcome T cell escape in multiple types of resistant tumors.
◦CD19 CAR-NK (CD19 t-haNK): robust ADCC antitumor activity against cancerous B cells and with potential applications for patients with r/r NHL.
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Our clinical programs including our CAR-NK product candidates have been initiated with promising early readouts in a range of oncology indications:
•PD-L1 CAR-NK
◦TNBC (Phase 1)
◦Pancreatic cancer QUILT-88 (Phase 2)
◦GBM QUILT-3.078 (Phase 2)
•CD19 t-haNK (Phase 1 trials)
◦r/r NHL QUILT-3.092
◦r/r selected CD19+ and CD20+ NHL QUILT-106
ANKTIVA + PD-L1 CAR-NK (t-haNK) has received FDA RMAT designation for use in combination with standard-of-care chemotherapy/radiotherapy for the reversal of lymphopenia and treatment of multiply relapsed locally advanced or metastatic pancreatic cancer.
Autologous and Allogeneic M-ceNK (World Bank of Natural Killer Cells)
NK persistence and function can be enhanced with cytokine stimulation. Our M-ceNK cells are generated from lymphocytes collected from donors that are then pre-activated ex-vivo by exposure to interleukins -12 (IL-12), -15 (N-803) and -18 (IL-18), which results in differentiation and acquisition of enhanced responses to cytokine re-stimulation. The apheresis processing and cell manufacturing activities for these studies are currently performed by trained personnel using controlled, manual processes designed to ensure product quality and consistency. M-ceNK have increased antitumor characteristics, including enhanced IFN-γ production and cytotoxicity against leukemic cell lines. M-ceNK cells are further distinguished by their unique cell-surface marker profile and their highly desirable feature of immune-memory, marked by their pronounced anti-cancer activity for weeks to months in duration, which has made these cells a research focus for more than a decade.We have developed a unique ability to generate a portfolio of distinct M-ceNK cell products through the application of our proprietary technology and cytokines. Also, we can manufacture these cell products for clinical delivery using our proprietary methods and overall expertise in scale manufacturing of NK cell-based products.
We have initiated or completed a series of trials evaluating the safety and feasibility of the technology including:
•NK2022 & NK2023 (Cancer Patients & Healthy Donors): 64 subjects completed apheresis and M-ceNK manufacturing cell therapy process development for robotic training and future AI robot manufacturing. Cells cryopreserved and stored.
•QUILT-3.076: Safety Phase 1 of apheresis and M-ceNK and ANKTIVA completed: Solid tumor trial with apheresis followed by M-ceNK and ANKTIVA that enrolled 10 patients treated with autologous M-ceNK infusion
•QUILT-105 (Healthy Volunteers): Apheresis followed by ANKTIVA stimulation prior to second apheresis
We believe our studies have demonstrated that M-ceNK cells can be reliably manufactured, cryopreserved, and administered with acceptable safety and tolerability profiles and evidence of immune activation, supporting continued clinical development. We continue to improve the workflow and manufacturing techniques and intend to incorporate increased artificial intelligence and fully robotic automation over time to support scalability in our cellular therapies as the program advances.
Platform Components Addressing the Immune System
As of December 31, 2025, key elements of our pipeline include clinical-stage programs in the following indications:
•Bladder Cancer: BCG-unresponsive NMIBC CIS with or without papillary tumors (approved), BCG-unresponsive papillary, BCG-naïve NMIBC
•Lung Cancer: Progressing, metastatic NSCLC in combination with CPIs (conditional approval in Saudi Arabia)
•Lymphopenia: Tumor-agnostic treatment of chemotherapy/radiation-induced lymphopenia (EAP authorized)
•Pancreatic Cancer: Advanced/metastatic disease (RMAT designation)
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•Glioblastoma: Recurrent GBM (registration trial)
•Non-Hodgkin Lymphoma: Waldenström macroglobulinemia and other CD19+/CD20+ lymphomas
•Colorectal Cancer/Lynch Syndrome: Cancer prevention in high-risk patients (NCI-sponsored)
•Prostate Cancer: High-risk prostate cancer
•Ovarian Cancer: Platinum-resistant ovarian cancer
•Infectious Diseases: HIV, Long COVID, universal nucleocapsid vaccine
•Apheresis Platform: M-ceNK with cryopreservation and storage
PIPELINE SUMMARY
Product Candidate Indication Phase Status
ANKTIVA + BCG BCG-naïve NMIBC Phase 2B 85% enrolled
ANKTIVA + BCG BCG-unresponsive papillary sBLA Resubmission plan
ANKTIVA + PD-L1 CAR-NK Recurrent GBM Phase 2/3 Registration trial
ANKTIVA + PD-L1 CAR-NK Pancreatic cancer Phase 2 RMAT designation
CD19 CAR-NK + rituximab NHL Phase 1 QUILT-106
ANKTIVA Lymphopenia (solid tumors) Expanded Access FDA EAP authorized
rBCG (Serum Institute) NMIBC Expanded Access FDA EAP authorized
M-ceNK Solid tumors Phase 1 Completed
Ad-HPV HPV Phase 2 Initiated and enrolling
CLINICAL DEVELOPMENT PROGRAMS
Bladder Cancer
According to the American Cancer Society, bladder cancer is the sixth most diagnosed cancer in the U.S., with an estimated 84,530 new cases and 17,870 deaths expected in 2026. NMIBC represents approximately 75% of all bladder cancers. Radical cystectomy is a treatment option for unresponsive NMIBC; however, the procedure significantly impacts quality of life.
Regulatory Designations: Breakthrough Therapy (BCG-unresponsive NMIBC CIS with or without papillary tumors); Fast Track (BCG-unresponsive NMIBC with papillary tumors, BCG-naive NMIBC with CIS).
Seminal patents covering intravesical administration of BCG and ANKTIVA have been issued with term coverage until 2035.
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BCG-Unresponsive NMIBC CIS (Cohort A) QUILT-3.032
Trial Design: Phase 2/3 open-label multi-center trial. Patients received BCG with ANKTIVA weekly for six consecutive weeks during induction, followed by three weekly maintenance instillations at three-month intervals for up to 12 months and then at month 18. Patients with no disease or low-grade Ta disease at months 24, 30, and 36 were eligible for continued BCG with ANKTIVA treatment (3 weekly instillations), at the principal investigators’ discretion.
Primary Endpoint: Complete response rate at any time of 30% or greater with a lower bound of 95% CI of 20% or greater. Complete response, or the disappearance of measurable disease in response to treatment, is evaluated at three months or six months following initial administration of BCG plus ANKTIVA (and every three months thereafter until 24 months). This endpoint would be achieved once at least 24 of the 80 patients in the trial achieve a complete response.
Results (published in NEJM Evidence, November 2022):
•Complete Response Rate: 71% (58 of 82 patients; 95% CI: 59.6%, 80.3%)
•Median Duration of Complete Response: 26.6 months (95% CI: 9.9, upper bound not reached)
•24-Month Cystectomy Avoidance: 91.4% in patients with complete response; 84.1% in all patients
•24-Month Disease-Specific Survival: 100%
•Safety: 1% treatment-related serious adverse events; 0% immune-related serious adverse events
Updated Results (December 2024): Full enrollment of 100 patients completed. Complete response rate of 71% maintained. Durable responses extending to 54 months. This data supported the EMA’s conditional marketing authorization that was granted in February 2026.
Regulatory Status:
In April 2024, the FDA approved ANKTIVA with BCG for the treatment of adult patients with BCG-unresponsive NMIBC CIS with or without papillary tumors (after we had resubmitted our BLA in May 2023 after an initial CRL related to deficiencies associated with the FDA’s pre-license inspection of our third-party CMOs, among others). In July 2025, ANKTIVA received approval from the MHRA for the UK and in January 2026 received accelerated approval from the SFDA in Saudi Arabia in the same indication. In February 2026, the European Commission granted conditional marketing authorization in the EU for ANKTIVA in combination with BCG for adult patients with BCG-unresponsive NMIBC CIS with or without papillary tumors. We are required to comply with certain post-marketing commitments, including completion of our QUILT-3.032 clinical trial and annual reporting for up to four years, with a final report submission to the FDA by the end of 2029.
BCG-Unresponsive NMIBC With Papillary Tumors (Cohort B), QUILT-3.032
Trial Design: Phase 2 open-label multi-center trial. Patients received BCG plus ANKTIVA weekly for six consecutive weeks during induction. The patients also receive additional treatment including three weekly maintenance instillations every three months for up to 12 months and then every nine months for up to 24 months
Primary Endpoint: 12-month disease-free survival rate greater than or equal to 30% and the lower bound of the 95% CI must be greater than or equal to 20% for success. To meet the primary endpoint, 24 out of 80 patients must be disease free at 12 months.
Results (published in The Journal of Urology, Chang et al., January 2026):
•12-Month Disease-Free Survival: 58.2% (95% CI: 46.6%, 68.2%)
•36-Month Disease-Specific Survival: 96.0% (median not reached)
•36-Month Progression-Free Survival: 83.1%
•12-Month Cystectomy-Free Survival: 92.2%
•36-Month Cystectomy-Free Survival: 81.8%
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Regulatory Status: At a Type B meeting in December 2025, the company presented an overview of the clinical status of its papillary disease program, including more than five years of follow-up data supporting the papillary indication. Based on these discussions, the FDA recommended the company provide certain additional information for its consideration to support a potential filing and review of the sBLA initially submitted in 2025 for the papillary indication. The company submitted the requested information to the Agency in February 2026. There can be no assurance that we will be successful in our efforts to resolve the May 2025 RTF letter with the FDA and/or that our sBLA, or any newly submitted sBLA or BLA, will be accepted for filing and review in this indication in a timely manner, or at all, without the need to conduct a new RCT prior to submission, or that we will ultimately receive approval even if it is accepted for filing.
As presented at ASCO 2022, the combination of BCG plus ANKTIVA (as measured in BCG-unresponsive NMIBC patients, Cohorts A and B combined from QUILT-3.032) was well-tolerated with 1% treatment-related serious adverse events, 0% immune-related serious adverse events, and 100% bladder cancer-specific overall survival at 24 months. Low-grade treatment-related adverse events include dysuria (22%), pollakiuria (20%), hematuria (17%), fatigue (16%), and urgency (12%), and all other treatment-related adverse events were seen at 7% or less.
BCG-Naïve NMIBC with CIS, QUILT-2.005
As discussed above, ANKTIVA received Fast Track designation from the FDA for the treatment of BCG-naïve NMIBC with CIS.
Trial Design: Phase 2B blinded, randomized, two-cohort trial of intravesical BCG plus ANKTIVA versus BCG alone in BCG-naive patients with high-grade NMIBC.
Enrollment: Cohort A (CIS): 366 patients planned; Cohort B (papillary): 230 patients planned. As of January 2026, enrollment is 85% complete in Cohort A, with full enrollment expected in Q2 2026.
Historical Data (Phase 1B, N=9 since 2014): 100% complete response rate. 8-year follow-up: all 6 evaluable patients remain disease-free with bladder preservation over a median survival period of 8.8 years.
Interim Analysis (FDA-requested, N=43): 84% complete response rate at nine months. Statistically significant longer duration of complete response with ANKTIVA in combination with BCG compared to BCG alone.
Timeline: Pivotal data readout expected in the second half of 2026. BLA submission target set for late 2026 to early 2027.
Recombinant BCG Supply Solution
In May 2024, we announced an exclusive global arrangement with Serum Institute to supply us with rBCG for use in combination with ANKTIVA. This addresses a significant supply challenge: TICE BCG (Merck) has been the sole supplier of BCG product in the U.S. since 2012, and rationing has been ongoing since 2019. According to published surveys, 57% of U.S. urologists report being unable to treat patients due to the BCG shortage, affecting an estimated 35,000 patients annually.
Regulatory Progress: In February 2025, the FDA authorized an EAP for rBCG. In Q1 2025, shipments commenced under the EAP. The company continues to work with the FDA to solve this 13-year shortage.
Lung Cancer
According to the American Cancer Society, lung cancer is the second most common cancer in the U.S., with an estimated 229,410 new cases and 124,990 deaths in 2026. NSCLC accounts for approximately 83% of all lung cancer diagnoses.
QUILT-3.055: Second-Line and Beyond NSCLC
Trial Design: Single-arm Phase 2B multi-cohort basket trial of ANKTIVA and CPI combinations in patients who were actively progressing on prior PD-1/PD-L1 CPI therapy.
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Results (presented at IASLC World Conference on Lung Cancer, September 2024 and January 2026):
•Patients enrolled: 86 evaluable patients in pooled analysis
•Median overall survival: 14.1 months (95% CI: 11.7, 17.4)
•Published Historical Comparator (any therapy post-CPI, Freeman et al., 2020): 6.1 months
•Published Historical Comparator (docetaxel + CYRAMZA, Brueckl et al., 2021): 7.5 months
•Maximum overall survival: 58 months
•Patients achieving ALC >=1.2 x 103 cells/μL: 80% (69/86)
•Median overall survival in patients with ALC >=1.2 x 103 cells/μL : 15.8 months (95% CI: 12.6, 21.9)
•Median overall survival in patients with ALC <1.2 : 11.5 months (95% CI: 4.2, 13.3); p=0.0057
Among 147 patients enrolled in QUILT-3.055, the common ANKTIVA attributed grade 1 and 2 adverse events included: injection-site reaction (84%), chills (44%), fatigue (30%), pyrexia (28%), nausea (16%), injection site erythema (14%), injection site pruritus (14%), injection site pain (12%), influenza like illness (12%), and decreased appetite (10%). A total of 38 grade 3 or higher adverse events attributed to ANKTIVA have been reported among 24 patients (16%) in the trial as of January 2026. All reported grade 3 or higher adverse events occurred at a frequency of 2% or less including: fatigue, injection site reaction, chills, injection site pain, injection site illness, injection site erythema, injection site pruritus, aspartate aminotransferase increased, alanine aminotransferase increased, blood alkaline phosphatase increased, lymphocyte count decreased, weight decreased, anaemia, diarrhoea, colitis, ileus, small intestinal obstruction, pneumonitis, respiratory failure, rash maculo-papular, deep vein thrombosis, hypovolaemic shock, pericardial effusion, tricuspid valve incompetence, and injection site cellulitis.
QUILT-2.023: First-Line NSCLC
Trial Design: Randomized Phase 3 trial to evaluate ANKTIVA in combination with CPIs versus other CPI combinations in the first-line setting for NSCLC