Sponsor: University of Texas Southwestern Medical Center (other)
Phase: 2
Start date: Feb. 18, 2025
Planned enrollment: 64
Ligufalimab (AK117) is a humanized IgG4 monoclonal antibody targeting the “don’t‑eat‑me” signal CD47, developed by Akeso. Early clinical activity has been reported in higher‑risk myelodysplastic syndromes (HR‑MDS) and in treatment‑naïve acute myeloid leukemia (AML) when combined with azacitidine. In September 2025, the FDA granted orphan drug designation for AML. (ashpublications.org)
AK117 binds CD47 with high affinity and blocks CD47–SIRPα, restoring macrophage phagocytosis of tumor cells. Preclinical characterization showed AK117 does not induce red‑blood‑cell (RBC) hemagglutination and had reduced erythrophagocytosis compared with an earlier anti‑CD47 antibody, potentially due to a distinct binding conformation. In non‑human primates, AK117 produced less hematologic toxicity than the comparator. (pubmed.ncbi.nlm.nih.gov)
At the recommended phase 2 dose (RP2D; 30 mg/kg Q2W), the complete remission (CR) rate was 48.1% among 27 evaluable patients; among those with ≥3 cycles, CR 52%; ≥6 cycles, CR 68.4%. Transfusion independence for RBCs was 61.5% among initially transfusion‑dependent patients. A randomized, double‑blind, placebo‑controlled Phase 2 trial (NCT06196203) is ongoing. (ashpublications.org)
AML, treatment‑naïve, unfit (Phase 1b/2, NCT04980885; AK117 + azacitidine)
At RP2D (30 mg/kg Q2W), reported CR was 50.0% (composite CR 55.0%) among 20 response‑evaluable patients; median time to CR 1.84 months. Trial status: active, not recruiting. (onclive.com)
Solid tumors (Head and neck squamous cell carcinoma, PD‑L1–positive; Phase 2 data reported at ESMO 2024)
In HR‑MDS Phase 1b (AK117 + azacitidine), anemia occurred in 29–31% overall; grade ≥3 anemia in ~22–24%. Other common TEAEs (≥30%) included neutropenia, leukopenia, thrombocytopenia, pyrexia, lymphopenia, constipation, and vomiting. Three patients (4.2%) discontinued for TEAEs. Importantly, unlike first‑generation CD47 antibodies, AK117 did not require a priming dose. (ashpublications.org)
In AML Phase 1b/2 (AK117 + azacitidine), sponsor‑summarized safety has been described as manageable with a low incidence of anemia; detailed peer‑reviewed safety tables were not located. (akesobio.com)
Preclinical studies demonstrated no hemagglutination with AK117 and less hematotoxicity vs a benchmark anti‑CD47 antibody. (pubmed.ncbi.nlm.nih.gov)
Notes: Reported solid‑tumor efficacy comes from sponsor press at ESMO 2024; interpret with caution pending peer‑reviewed presentation/publication. (prnewswire.com)
Last updated: Oct 2025
Cadonilimab (AK104; Chinese trade name Kai Tan Ni) is a first-in-class, tetravalent bispecific antibody that targets PD‑1 and CTLA‑4. It has regulatory approvals in China for advanced cervical cancer (second line in June 2022; expanded to first line in June 2025) and for first-line gastric/GEJ adenocarcinoma (September 2024). Several phase 2–3 trials and real‑world/retrospective studies across tumor types have reported efficacy and safety results. (link.springer.com)
Gastric/GEJ adenocarcinoma (first line; randomized phase 3) - Cadonilimab + chemotherapy vs placebo + chemotherapy (N=610). Median OS 14.1 vs 11.1 months (HR 0.66; 95% CI 0.54–0.81; P<0.001); median PFS 7.0 vs 5.3 months (HR 0.53; 95% CI 0.44–0.65). ORR 65.2% vs 48.9%; median DoR 8.8 vs 4.4 months. Benefit also observed in PD‑L1 CPS ≥5 subgroup (OS 15.3 vs 10.9 months; HR 0.58). (link.springer.com)
Cervical cancer (first line; phase 2, single‑arm; COMPASSION‑13) - Cadonilimab + chemotherapy ± bevacizumab in recurrent/metastatic disease showed ORR 66.7% (A‑15), 68.8% (A‑10), and 92.3% (B‑10) among evaluable patients; phase 3 confirmatory trial ongoing (NCT04982237). (aacrjournals.org) - A separate randomized phase 3 in the same setting met its primary PFS endpoint (press release). (akesobio.com)
Non–small cell lung cancer - Phase Ib/II monotherapy in previously treated NSCLC (AK104‑202): ORR 10% in one cohort; no responses in others; the study did not meet its primary endpoint. (pubmed.ncbi.nlm.nih.gov) - Multicenter retrospective series in IO‑refractory NSCLC (n=41 evaluable for efficacy): ORR 4.3%, DCR 39.1%, median PFS ~3.5 months (108 days). (pubmed.ncbi.nlm.nih.gov)
Hepatocellular carcinoma - Retrospective real‑world first‑line cadonilimab + lenvatinib (n=29): ORR 37.9%, DCR 82.8%, mPFS 8.1 months; mOS not reached at cutoff. A global randomized trial in PD‑(L)1–experienced HCC has been cleared to initiate. (pubmed.ncbi.nlm.nih.gov)
Early‑phase/multitumor experience - Phase 1b/2 COMPASSION‑03 established safety and antitumor activity across expansion cohorts (cervical, esophageal squamous cell carcinoma, HCC); dosing at 6 mg/kg Q2W or fixed 450 mg Q2W. (pubmed.ncbi.nlm.nih.gov)
References above provide detailed response rates, survival outcomes, AE profiles, and trial identifiers.
Last updated: Oct 2025
Goal: Evaluate the antitumor activity of the combination of ligufalimab (AK117) and cadonilimab (AK104) in patients with advanced hepatobiliary cancers that have progressed after prior anti–PD‑1/L1 therapy, with the primary endpoint of objective response rate.
Patients: Adults (≥18 years) with locally advanced or metastatic hepatocellular carcinoma (excluding fibrolamellar) or biliary tract cancers (intrahepatic/extrahepatic cholangiocarcinoma or gallbladder; combined HCC-CCA allowed in the BTC cohort). Disease must be unresectable and measurable per RECIST v1.1. Patients must have previously received and become refractory to or relapsed after anti–PD‑1/L1 therapy and have ECOG 0–1. For HCC, Child-Pugh A or B7 is allowed. Key exclusions include prior liver transplant, active autoimmune disease requiring systemic therapy, significant uncontrolled comorbidities, need for systemic immunosuppression, severe prior immune-related toxicity leading to ICI discontinuation, active CNS disease requiring immediate intervention, and known HIV infection. HBV and HCV infections are permitted with protocol-defined management.
Design: Open-label, non-randomized, phase II basket study with two parallel cohorts (HCC and BTC); approximately 64 patients total. Single-arm evaluation comparing clinical outcomes to historical benchmarks.
Treatments: Ligufalimab plus cadonilimab administered as intravenous infusions on day 1 of each 21-day cycle until disease progression, unacceptable toxicity, or withdrawal. Ligufalimab (AK117) is a humanized IgG4 monoclonal antibody targeting CD47 to block CD47–SIRPα signaling and enhance macrophage-mediated phagocytosis of tumor cells. Distinct from some CD47 antibodies, AK117 has shown no hemagglutination in preclinical/early clinical testing, potentially mitigating anemia risk and obviating a priming dose; early-phase studies across hematologic malignancies and solid tumors have demonstrated antitumor activity with a generally manageable safety profile. Cadonilimab (AK104) is a tetravalent, Fc-null bispecific antibody targeting PD‑1 and CTLA‑4 designed to intensify checkpoint blockade while reducing Fc-mediated toxicities. It has demonstrated efficacy across multiple tumors, including regulatory approval in China for cervical cancer and positive phase 3 results in gastric/GEJ cancer, with a safety profile characterized predominantly by hematologic and immune-related adverse events that are typically manageable.
Outcomes: Primary: Objective response rate by RECIST v1.1. Secondary: Overall survival, progression-free survival, and duration of response, each contextualized to historical controls; safety characterized by incidence and severity of adverse events per CTCAE v5.0. Treatment and follow-up continue up to 24 months or until progression, unacceptable toxicity, death, or discontinuation.
Burden on patient: Moderate. Patients receive IV infusions every 21 days with visits lasting approximately 4–5 hours, which entails regular travel and chair time. Standard oncologic monitoring is expected, including periodic laboratory testing, physical exams, and cross-sectional imaging per RECIST; the schedule is similar to other immunotherapy trials and not expected to require intensive pharmacokinetic sampling. Baseline archival or recent biopsy tissue is requested in certain HCC cases, which may add procedural burden if not already available. No mandated priming dose or complex inpatient monitoring is described, keeping visit intensity moderate compared with early phase studies; however, management of potential immune-related and hematologic adverse events may necessitate additional unscheduled evaluations or supportive care visits.
Last updated: Oct 2025
Inclusion Criteria:
1. Histological confirmation of specific disease -Cohort A (HCC): Patient must have a diagnosis confirmed by histology or clinically by the American Association for the Study of Liver Diseases (AASLD) criteria in patients with cirrhosis. Known fibrolamellar HCC will be excluded.
* Cohort B (BTC, biliary tract cancers): Patients must have histologically confirmed biliary tract cancer (including intrahepatic cholangiocarcinoma, extrahepatic cholangiocarcinoma, and gall bladder cancers). Patients with combined HCC-cholangiocarcinoma may be enrolled in Cohort B.
2. Locally advanced or metastatic disease
* Patients with locally advanced or metastatic disease must have disease deemed not amenable to surgical and/or locoregional therapies or patients who have progressed following surgical and/or locoregional therapies.
* Measurable disease, as defined as lesions that can accurately be measured in at least one dimension according to RECIST version 1.1 at least 1 cm with contrast enhanced dynamic imaging (magnetic resonance imaging or computed tomography).
3. Refractory to or relapsed after prior anti-PD-1/L1 antibody therapy. May have received anti-PD-1/L1 monotherapy or combination therapy as any line of therapy including in the neoadjuvant or adjuvant setting. Patients who discontinued prior immune checkpoint inhibitor treatment due to a high-grade toxicity (Grade 4) are not eligible.
4. For patients in cohort A who do not have a clinical diagnosis of HCC according to the AASLD criteria, formalin-fixed, paraffin-embedded (FFPE) tumor diagnostic tissue samples must have been obtained within 4 years from the time of consent. Baseline tissue will be requested any time after consent. It is strongly recommended that tissue is obtained from standard-of-care biopsies confirming progression of disease on prior therapy so that the patient has not received any intervening systemic anti-cancer treatment from the time that the baseline tissue was obtained.
5. Prior locoregional therapy is allowed provided the following are met: 1) at least 2 weeks since prior locoregional therapy including surgical resection, chemoembolization, radiotherapy, or ablation; 2) target lesion has increased in size ≥25% since the cessation of locoregional therapy or the target lesion was not treated with locoregional therapy. Patients treated with palliative radiotherapy for symptoms will be eligible as long as the target lesion is not the treated lesion and radiotherapy will be completed at least 2 weeks prior to study drug administration.
6. Age ≥ 18 years
7. Child-Pugh Score A or B7 (only applicable for Cohort A)
8. ECOG Performance score of 0-1
9. Adequate organ and marrow function (without chronic, ongoing growth factor support or transfusion in the last 2 weeks) as defined below:
-Platelet count ≥ 50,000/mm3
-Hgb ≥ 9 g/dl
-Absolute neutrophil ≥ 1,000 cells/mm3
-Total bilirubin ≤ 3 mg/ml (This will not apply to subjects with Gilbert's syndrome who have persistent or recurrent hyperbilirubinemia that is predominantly unconjugated in the absence of hemolysis, or patients with hyperbilirubinemia secondary to distal malignant obstruction where endoscopic, surgical, or percutaneous bypass/stenting has been attempted. Such patients may be enrolled based in consultation with the principal investigator)
-INR ≤ 2
-AST, ALT ≤ 5 times ULN
* Calculated creatinine clearance (CrCl) ≥ 40 mL/min. CrCl can be calculated using the Cockroft-Gault method.
* Albumin ≥ 2.0 g/dl
10. All men, as well as women of child-bearing potential, defined as not surgically sterilized and between menarche and 1-year post menopause, must agree to use highly effective contraception methods (hormonal or barrier method of birth control or abstinence) 4 weeks prior to study entry, for the duration of study participation, and for 120 days after the last dose of ligufalimab or cadonilimab.
Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
A female of child-bearing potential is any woman (regardless of sexual orientation, marital status, having undergone a tubal ligation, or remaining celibate by choice) who meets the following criteria:
* Has not undergone a hysterectomy or bilateral oophorectomy; or
* Has not been naturally postmenopausal for at least 12 consecutive months (i.e., has had menses at any time in the preceding 12 consecutive months).
11\. Women of child-bearing potential must have a negative serum pregnancy test at screening.
12\. Subjects are eligible to enroll if they have non-viral-HCC, or if they have HBV-HCC, or HCV-HCC defined as follows:
1\) HBV-HCC: Hepatitis B subjects will be allowed if they meet the following criteria: On antiviral therapy for HBV. Subjects who are anti-HBc (+), negative for HBsAg, negative for anti-HBs, and have an HBV viral load under 100 IU/mL do not require HBV anti-viral prophylaxis.
2\) HCV-HCC: Active or resolved HCV infection as evidenced by detectable HCV RNA or antibody. Patients who have failed HCV therapy as evidenced by detectable HCV RNA will be eligible. Subjects with chronic infection by HCV who are treated (successfully or treatment failure) or untreated are allowed on study.
13\. Ability to understand and the willingness to sign a written informed consent.
14\. Willing and able to comply with the requirements and restrictions in this protocol.
15\. Patients who have received the vector, protein subunit, or nucleic acid COVID-19 vaccines are eligible to enroll.
Exclusion Criteria:
1. Prior liver transplant.
2. Known human immunodeficiency virus (HIV) positive (testing not required).
3. Use of any live vaccines against infectious diseases within 28 days of first dose of study drug administration.
4. History of trauma or major surgery within 28 days prior to the first dose of study drug administration. (Tumor biopsy or placement of central venous access catheter (eg, port or similar) is not considered a major surgical procedure).
5. Underlying medical conditions that, in the investigator's opinion, will make the administration of study drugs hazardous, including but not limited to:
* Interstitial lung disease, including history of interstitial lung disease or non infectious pneumonitis (lymphangitic spread of cancer is not disqualifying),
* Active viral, bacterial, or fungal infections requiring parenteral treatment within 14 days of the initiation of study drugs,
* Clinically significant cardiovascular disease,
* A condition that may obscure the interpretation of toxicity determination or AEs,
* History of prior solid-organ transplantation.
6. Hypersensitivity to IV contrast; not suitable for pre-medication.
7. Any active autoimmune disease or a documented history of autoimmune disease or syndrome that required systemic treatment in the past 2 years (ie, with use of disease-modifying agents, corticosteroids, or immunosuppressive drugs), except for vitiligo or resolved childhood asthma/atopy.
* Replacement therapy (eg, thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency) is not considered a form of systemic treatment.
* Participants with asthma who require intermittent use of bronchodilators, inhaled corticosteroids, or local corticosteroid injections will not be excluded from this study. Participants on chronic systemic corticosteroids will be excluded from the study.
8. Known history of active bacillus tuberculosis.
9. Subjects with a condition requiring systemic treatment with either corticosteroids (\> 10 mg/day prednisone equivalent) or other immunosuppressive medications within 14 days of study administration. Inhaled or topical steroids and adrenal replacement doses ≤10 mg/day prednisone equivalents are permitted in the absence of autoimmune disease.
10. Patients who discontinued prior immune checkpoint inhibitor treatment due to Grade ≥ 3 or Grade 2 serious toxicity (i.e., pneumonitis, uveitis, neurological symptoms, cardiac toxicity, etc.) immune-related adverse events.
11. Known severe hypersensitivity reactions to monoclonal antibodies (≥Grade 3).
12. Prior malignancy that required systemic treatment within the previous 2 years except for locally curable cancers that have been apparently cured, such as basal or squamous cell skin cancer, superficial bladder cancer, or carcinoma in situ of the cervix, breast, or prostate cancer.
13. Prisoners or subjects who are involuntarily incarcerated.
14. If a participant has symptomatic or clinically active brain metastases including leptomeningeal disease, they must be excluded if:
* Has evidence of progression by neurologic symptoms
* Has metastatic brain lesions that require immediate intervention.
* Has carcinomatous meningitis, regardless of clinical stability
15. Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after contraception and until the termination of gestation, confirmed by a positive hCG laboratory test.
16. Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.
17. Has significant dementia or other mental condition that precludes the participant's ability to consent to the study.
18. Use of other investigational drugs (drugs not marketed for any indication) within 28 days or 5 half-lives (whichever is longer) of first dose of study drugs.
19. Known hypersensitivity to recombinant proteins, or any excipient contained in the study drug formulations.
Dallas, Texas, 75390, United States
[email protected] / 214-648-7097
Status: Recruiting