Sponsor: University of Texas Southwestern Medical Center (other)
Phase: 2
Start date: Feb. 18, 2025
Planned enrollment: 64
Ligufalimab (AK117) is an investigational humanized IgG4 monoclonal antibody targeting CD47, developed by Akeso, Inc. It is designed to enhance the phagocytic activity of immune cells against tumor cells by blocking the CD47-SIRPα interaction, thereby inhibiting tumor growth. Notably, AK117 does not induce hemagglutination, a common adverse effect associated with other CD47-targeting therapies, eliminating the need for a priming dose to prevent anemia. (pubmed.ncbi.nlm.nih.gov)
AK117 binds with high affinity to CD47 on tumor cells, effectively blocking its interaction with SIRPα. This blockade enhances macrophage-mediated phagocytosis of tumor cells, leading to tumor inhibition. The antibody's unique binding conformation prevents hemagglutination, distinguishing it from other CD47-targeting antibodies. (pubmed.ncbi.nlm.nih.gov)
Myelodysplastic Syndrome (MDS):
In a Phase Ib clinical trial presented at the 65th American Society of Hematology (ASH) Annual Meeting, AK117 combined with azacitidine (AZA) was evaluated in patients with newly diagnosed higher-risk MDS. Among 27 evaluable patients:
Additionally, 61.5% of patients who initially required red blood cell transfusions became transfusion-independent. (akesobio.com)
Acute Myeloid Leukemia (AML):
In the same Phase Ib study, among 20 evaluable treatment-naïve AML patients:
These results suggest that AK117 combined with AZA may offer a promising first-line treatment option for AML patients ineligible for intensive chemotherapy. (akesobio.com)
Colorectal Cancer:
A Phase II study evaluated ivonescimab (a PD-1/VEGF bispecific antibody) combined with FOLFOXIRI chemotherapy, with or without AK117, in patients with metastatic colorectal cancer. Among 39 patients:
These findings indicate that the addition of AK117 may enhance treatment efficacy in colorectal cancer. (stockwatch.com)
AK117 has demonstrated a favorable safety profile across multiple studies. In the MDS trial, anemia occurred in 29.1% of patients, a lower incidence compared to other CD47-targeting therapies. The combination of AK117 and AZA was well-tolerated, with no dose-limiting toxicities reported. (akesobio.com)
In the colorectal cancer study, the safety profile was acceptable and manageable. Serious treatment-related adverse events occurred in 22.7% of patients receiving ivonescimab plus chemotherapy and 11.1% in those receiving the combination with AK117. (stockwatch.com)
Last updated: Aug 2025
Cadonilimab (AK104; “Kai Tan Ni”) is a tetravalent, Fc-null bispecific IgG1 antibody that targets PD-1 and CTLA-4. It has been evaluated across multiple solid tumors, with most mature data in cervical cancer and gastric/gastroesophageal junction (GEJ) adenocarcinoma. In China, cadonilimab received regulatory approval in June 2022 for second-line treatment of recurrent or metastatic cervical cancer. (ncbi.nlm.nih.gov)
Cervical cancer - First-line, with chemotherapy ± bevacizumab (COMPASSION-16; randomized, double-blind, phase 3, China): - Median PFS 12.7 vs 8.1 months; HR 0.62 (95% CI 0.49–0.80), p<0.0001. - Median OS not reached vs 22.8 months; HR 0.64 (95% CI 0.48–0.86), p=0.0011. (thelancet.com) - Previously treated, monotherapy (multicenter, open-label, single-arm phase 2): - ORR 33.0% (12% CR, 21% PR) by independent review; median PFS 3.75 months; median OS 17.51 months. Among PD‑L1 CPS ≥1 tumors, ORR 43.8%. (gynecologiconcology-online.net) - First-line, phase 2 combination run-in (COMPASSION-13): - With chemotherapy (± bevacizumab), ORR 66.7–92.3% across cohorts; study designed primarily for safety with encouraging antitumor activity; supported advancement to phase 3. (aacrjournals.org)
Gastric/GEJ adenocarcinoma - Phase 1b/2 (COMPASSION-04; HER2‑negative, first-line): - Recommended phase 2 dose: 6 mg/kg Q2W. - ORR 52.1%; median DOR 13.73 months; median PFS 8.18 months; median OS 17.48 months. Benefit observed irrespective of PD‑L1 status. (nature.com) - Phase 3 (COMPASSION-15/AK104‑302; double-blind, first-line with XELOX): - Nature Medicine publication (Jan 22, 2025) reports a statistically significant OS benefit for cadonilimab + chemotherapy vs chemotherapy; trial met primary endpoint. Conference reports detailed median OS 15.0 vs 10.8 months (HR ~0.60) and median PFS 7.0 vs 5.3 months (HR 0.53), with benefit regardless of PD‑L1 expression. (nature.com, onclive.com, oncnursingnews.com)
Esophageal squamous cell carcinoma (ESCC) - Ongoing investigator-initiated phase 2 (first-line cadonilimab + taxane/cisplatin): - Updated ASCO abstracts report ORR 81–90% and median PFS ~7 months; data remain early and single-arm. (ascopubs.org)
Notes - Doses and regimens varied by study (e.g., 6 mg/kg Q2W in phase 1b/2 gastric; 10 mg/kg Q3W in several phase 2/3 cervical or gastric studies). Consult individual trial publications for protocol-specific dosing. (nature.com, thelancet.com)
Last updated: Sep 2025
Goal: Evaluate antitumor activity of ligufalimab (AK117) plus cadonilimab (AK104) in patients with advanced hepatobiliary cancers that have progressed after prior anti–PD‑1/PD‑L1 therapy, with a primary focus on objective response rate.
Patients: Adults (≥18 years) with unresectable locally advanced or metastatic hepatocellular carcinoma (excluding fibrolamellar; Child-Pugh A or B7 allowed) or biliary tract cancers (intrahepatic/extrahepatic cholangiocarcinoma, gallbladder; combined HCC-CCA allowed) with measurable disease by RECIST 1.1 and ECOG 0–1. All must be refractory to or relapsed after prior anti–PD‑1/PD‑L1 therapy and not discontinued that therapy for high-grade immune toxicity. Key exclusions include prior liver transplant, active autoimmune disease requiring systemic therapy, uncontrolled infections, significant cardiopulmonary comorbidity, active CNS/leptomeningeal disease requiring immediate intervention, and need for systemic immunosuppression. HBV- and HCV-associated HCC permitted with antiviral management per protocol.
Design: Open-label, non-randomized, phase II basket study with two parallel cohorts (HCC and biliary tract cancers). Planned enrollment is 64. Primary endpoint is assessed against historical controls; no comparator arm.
Treatments: Ligufalimab (AK117) plus cadonilimab (AK104) administered as intravenous infusions on day 1 of each 21-day cycle until progression or unacceptable toxicity. Ligufalimab is a humanized IgG4 monoclonal antibody targeting CD47, blocking the CD47–SIRPα “don’t eat me” signal to enhance macrophage-mediated phagocytosis. Unlike earlier CD47 agents, ligufalimab has been engineered to avoid hemagglutination and typically does not require priming; early trials in hematologic malignancies and solid tumors have shown encouraging response rates with a manageable anemia signal. Cadonilimab is a tetravalent Fc-null bispecific antibody targeting PD-1 and CTLA-4 to deliver dual checkpoint blockade with reduced Fc-mediated toxicities; it has demonstrated efficacy signals across multiple solid tumors and regulatory approval in cervical cancer in China, with randomized data showing improved survival when combined with chemotherapy. The biological rationale for the combination leverages innate (anti-CD47) and adaptive (PD-1/CTLA-4) immune activation in ICI-exposed disease.
Outcomes: Primary: best objective response rate by RECIST v1.1. Secondary: overall survival, progression-free survival, and duration of response compared with historical controls, and safety characterized by incidence and severity of adverse events per NCI CTCAE v5.0. Treatment continues up to 24 months or until progression, toxicity, or discontinuation.
Burden on patient: Moderate. Patients receive IV infusions every 21 days with clinic visits lasting approximately 4–5 hours, requiring regular travel. Standard oncologic monitoring is expected, including periodic labs and cross-sectional imaging per RECIST; no intensive pharmacokinetic schedules are described. Baseline archival or recent tumor tissue is requested, and optional or clinically indicated biopsies may occur, but mandated serial research biopsies are not specified. Overall, visit frequency and assessments are comparable to other immunotherapy trials without added high-frequency PK draws, but infusion-based therapy and imaging confer greater burden than oral regimens.
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