Sponsor: Apollomics Inc. (industry)
Phase: 2
Start date: Sept. 27, 2017
Planned enrollment: 497
APL-101 (also known as CBT-101, PLB1001, bozitinib, vebreltinib) is an orally bioavailable, highly selective small‑molecule inhibitor of the MET receptor tyrosine kinase. It has clinical data in MET‑altered cancers, most notably MET exon 14 skipping (METex14) non–small cell lung cancer (NSCLC). In China, vebreltinib received conditional approval in November 2023 for METex14 NSCLC and, in April 2024, approval for certain gliomas with PTPRZ1–MET fusion; development continues elsewhere in ongoing phase 1/2 studies. (pubmed.ncbi.nlm.nih.gov)
Vebreltinib inhibits MET kinase activity (ATP‑competitive), blocking downstream signaling of the HGF/MET axis. Preclinical work shows high potency and selectivity for MET (Ki ≈ 2.2 nM; cellular IC50 ≈ 0.52 nM) and brain penetration, with antitumor activity across MET‑dysregulated patient‑derived models. (aacrjournals.org)
MET exon 14 skipping NSCLC (KUNPENG, phase 2, single‑arm; n=52 evaluable): In the primary cohort treated with vebreltinib 200 mg twice daily, blinded independent review reported ORR 75.0% (95% CI 61.1–86.0), disease control rate 96.2%, median duration of response 16.5 months, median PFS 14.3 months, and median OS 20.3 months (3‑year OS rate 35.1%). Subgroups included ORR 77.1% in treatment‑naïve (n=35) and 70.6% in previously treated (n=17); ORR 100% in those with baseline brain metastases (n=5). (pmc.ncbi.nlm.nih.gov)
Basket cohort with non‑CNS MET fusions (SPARTA, phase 2, preliminary; n=14): Confirmed ORR 43% (1 complete response in 3rd‑line NSCLC; partial responses in NSCLC, pancreatic, and intrahepatic bile duct cancers). Median PFS 4.5 months; median OS 12.4 months; median time to response 3.7 months; median duration of response 5.6 months. (ir.apollomicsinc.com)
Glioma program: The approval in China for IDH‑mutant astrocytoma (WHO grade 4)/glioblastoma with PTPRZ1–MET fusion followed a randomized phase 2/3 effort; earlier work (phase 1) in recurrent high‑grade glioma with MET alterations demonstrated brain penetration and signals of activity. (ir.apollomicsinc.com)
Ongoing/global development: The SPARTA study (NCT03175224) is an international phase 1/2, multi‑cohort trial evaluating vebreltinib in METex14 NSCLC and MET‑dysregulated solid tumors, including cohorts exploring combinations (e.g., with EGFR TKIs for MET‑mediated resistance). A China partner study, KUNPENG (NCT04258033), provided the METex14 NSCLC data summarized above. (mycancergenome.org)
In the KUNPENG study, treatment‑related adverse events (TRAEs) were predominantly grade 1–2. The most common TRAEs (≥20% across 135 enrolled patients) were peripheral edema (56.3%), hypoalbuminemia (27.4%), hypoproteinemia (25.9%), and anemia (20.7%). No new safety signals emerged with longer follow‑up. The recommended dose used in phase 2 was 200 mg twice daily. (ascopubs.org)
Notes: Outside China, vebreltinib remains investigational as of October 7, 2025; efficacy and safety are being further defined in ongoing trials. (mycancergenome.org)
Last updated: Oct 2025
Goal: Evaluate the efficacy, safety, pharmacokinetics, and tolerability of the selective MET inhibitor vebreltinib (APL-101) as monotherapy across MET-altered malignancies and as add-on therapy to an EGFR inhibitor in EGFR-mutant NSCLC with acquired MET amplification after response to first-line EGFR inhibition.
Patients: Adults with unresectable or metastatic cancers characterized by MET dysregulation. Phase 2 cohorts include: MET exon 14 skipping NSCLC, both first line and previously treated MET inhibitor–naive; MET exon 14 NSCLC previously treated and MET inhibitor–experienced (enrollment completed); a basket of MET-amplified solid tumors excluding primary CNS tumors, including a dedicated NSCLC subgroup with MET amplification and EGFR wild-type; EGFR-mutant NSCLC with acquired MET amplification progressing on first-line EGFR inhibitor after a documented response, eligible for APL-101 add-on; a basket of MET fusion–positive solid tumors excluding primary CNS tumors; primary CNS tumors with MET alterations (fusion including PTPRZ1-MET, exon 14 skipping, or amplification); and tumors with wild-type MET but overexpression of HGF and MET. Key criteria include measurable disease, ECOG 0–1 (KPS ≥70 for CNS tumors), adequate organ and cardiac function, controlled CNS disease allowed, and no prior MET inhibitor exposure for MET inhibitor–naive cohorts.
Design: Phase 2, multicenter, nonrandomized, open-label, cohort expansion study following a completed Phase 1 lead-in. Up to 497 participants enrolled across molecularly defined disease cohorts; no comparator arm; independent radiology review committee employed for efficacy endpoints.
Treatments: APL-101 (vebreltinib) oral capsules given as monotherapy in all cohorts except the EGFR-mutant NSCLC acquired MET amplification cohort, which receives APL-101 added to ongoing standard-of-care EGFR inhibitor. Vebreltinib is a selective, brain-penetrant small-molecule MET kinase inhibitor that blocks MET phosphorylation and downstream signaling involved in proliferation, survival, invasion, and metastasis. Early clinical data suggest activity in MET exon 14–altered NSCLC with a reported objective response rate around two-thirds in Phase 1 experience, and a recommended Phase 2 dose of 200 mg twice daily. Its ability to cross the blood–brain barrier supports evaluation in CNS tumors and NSCLC with CNS involvement.
Outcomes: Primary endpoint is objective response rate per blinded independent review using RECIST v1.1 or tumor-appropriate criteria (RANO for CNS). Secondary endpoints include duration of response by IRC and investigator, investigator-assessed ORR, clinical benefit rate (CR + PR + durable SD), time to progression, progression-free survival, and overall survival at prespecified time points up to approximately 3 years.
Burden on patient: Moderate. The study is oral therapy–based and largely aligns with oncology standard-of-care schedules for imaging and safety assessments, but requires central confirmation of MET status using archival or fresh tumor tissue or liquid biopsy, which may necessitate an additional biopsy if tissue is insufficient. As a Phase 2 trial following a completed Phase 1 lead-in, intensive pharmacokinetic sampling is likely limited; however, regular clinic visits for drug dispensing, laboratory monitoring, ECGs for QTc surveillance, and periodic imaging per RECIST/RANO are expected. Patients in the add-on cohort continue EGFR inhibitor therapy alongside APL-101, increasing medication management and monitoring. Travel demands correspond to visit frequency typical for targeted therapy trials (approximately every 2–4 weeks initially, then every 8–12 weeks for imaging), with potentially higher burden for CNS tumor patients requiring neurological assessments and steroid management.
Last updated: Oct 2025
Major Inclusion Criteria:
1. Men and women 18 years of age or older.
2. 9 cohorts will be enrolled:
* Cohort A1 / Exon 14 NSCLC MET inhibitor naive in first line: Histologically or cytologically confirmed NSCLC with Exon 14 skipping mutations; all histologies; unresectable or metastatic disease (Stage 3b/4); treatment-naive subjects in first line; not received any MET inhibitor and no known MET kinase inhibitor resistance mutations
* Cohort A2 / Exon 14 NSCLC - MET inhibitor naïve: Histologically or cytologically confirmed NSCLC with Exon 14 skipping mutations; all histologies; unresectable or metastatic disease (Stage 3b/4); pretreated subjects refractory to or intolerant of standard therapies with no more than three lines of prior therapy in the unresectable or metastatic setting; not received any MET inhibitor and no known MET kinase inhibitor resistance mutations
* Cohort B / Exon 14 NSCLC MET inhibitor experienced: ENROLLMENT COMPLETED
* Cohort C / MET amplification basket tumor types excluding primary CNS tumors: Any solid tumor type regardless of histology excluding primary CNS tumors, with MET amplification; unresectable or metastatic disease, refractory to or intolerant of standard therapies, or refused standard therapies, or if therapy was unavailable or unfeasible, with no more than 3 prior lines of therapy in the unresectable or metastatic setting; not received any MET inhibitor and no known MET kinase inhibitor resistance mutations
* Cohort C1 / MET amplification and wild-type EGFR NSCLC: NSCLC regardless of histology, harboring MET amplification and wild-type EGFR; unresectable or metastatic disease, previously untreated or treated with no more than 3 prior lines of therapy in the unresectable or metastatic setting; not received any MET inhibitor and no known MET kinase inhibitor resistance mutations
* Cohort C2 / EGFR positive NSCLC with acquired MET amplification (APL-101 Add-on Therapy): Unresectable or metastatic NSCLC regardless of histology, harboring EGFR activating mutations with acquired MET-Amplification as resistance mechanism to the EGFR-I; developed resistance to first-line EGFR-inhibitor therapy after an initial response (documented PR for at least 12 weeks); radiological documentation of disease progression per RECIST on first-line EGFR inhibitor therapy; currently on an EGFR-inhibitor therapy and agrees to receive APL-101 as an add-on therapy during the study; no history of interstitial lung disease (ILD)/pneumonitis, Grade ≥3 liver toxicity or QT prolongation with EGFR-I therapy; not received any MET inhibitor and no known MET kinase inhibitor resistance mutations
* Cohort D / MET fusion basket tumor types excluding primary CNS tumors: any solid tumor type regardless of histology excluding primary CNS tumors; unresectable or metastatic disease, refractory to or intolerant of standard therapies, or refused standard therapies, or if therapy was unavailable or unfeasible, with no more than 3 prior lines of therapy in the unresectable or metastatic setting; not received any MET inhibitor and no known MET kinase inhibitor resistance mutations
* Cohort E / Primary CNS tumors with MET alterations: subjects with primary CNS tumors who meet inclusion criteria of MET dysregulations defined as single or co-occurred MET fusion including PTPRZ1-MET (ZM) fusion, MET Exon 14 skipping mutations, or MET amplification; refractory to or intolerant of standard therapies, or refused standard therapies, or if therapy was unavailable or unfeasible, with no more than 3 prior lines of therapy in the unresectable or metastatic setting; not received any MET inhibitor and no known MET kinase inhibitor resistance mutations; neurological symptoms controlled on a stable/decreasing dose of steroids for at least 2 weeks before C1D1
* Cohort F / Basket tumor types harboring wild-type MET with over-expression of HGF and MET: any solid tumor type regardless of histology harboring wild-type MET with overexpression of HGF and MET; Unresectable or metastatic disease, refractory to or intolerant of standard therapies, or refused standard therapies, or if therapy was unavailable or unfeasible, with no more than 3 prior lines of therapy in the unresectable or metastatic setting; not received any MET inhibitor and no known MET kinase inhibitor resistance mutations
3. Treated or untreated asymptomatic parenchymal CNS disease or leptomeningeal disease is allowed.
4. Presence of ≥1 measurable lesion (scan done ≤28 days of C1D1) to serve as target lesion according to relevant criteria
5. ECOG performance status of 0-1. For subjects with primary CNS tumors, KPS score ≥70.
6. Acceptable organ function
7. For all prior anticancer treatment, a duration of 30 days or 5 half-lives of the agents used, whichever is shorter, must have elapsed, and any encountered toxicity must have resolved to levels meeting all the other eligibility criteria prior to the first dose of study treatment. Palliative radiotherapy to non-target lesions should be completed within 2 weeks prior to APL-101 administration.
8. Adequate cardiac function
9. Women of child-bearing potential must have a negative serum or Beta-hCG at screening or evidence of surgical sterility or evidence of post-menopausal status
10. No planned major surgery within 4 weeks of first dose of APL-101
11. Expected survival (life expectancy) ≥ 3 months from C1D1
12. Provision of sample; e.g. archival or a fresh tumor biopsy sample (if safe and feasible) either from the primary or a metastatic site) or liquid biopsy sample (if tumor tissue is insufficient or lacking, and approved by the sponsor) is required for prospective central lab confirmation for study entry (subjects with previously confirmed molecular status by the Sponsor designated central lab or FDA approved NGS based MET testing may be exempted, subjected to Sponsor approval.
Major Exclusion Criteria:
1. Hypersensitivity to APL-101, excipients of the drug product, or other components of the study treatment regimen.
2. Known actionable mutation/gene rearrangement of EGFR (except for NSCLC subjects in Cohort C and C-2), ALK, ROS1, RET, NTRK, KRAS, and BRAF.
3. Use or intended use of any other investigational product, including herbal medications, through Study Treatment Termination.
4. Active uncontrolled systemic bacterial, viral, or fungal infection or clinically significant, active disease process, which in the opinion of the investigator makes the risk: benefit unfavorable for the participation of the trial.
5. Life-threatening illness, significant organ system dysfunction or comorbid conditions, or other reasons that, in the investigator's opinion, could compromise the subject's safety or the integrity of the study outcomes, or interfere with the absorption or metabolism of APL-101.
6. Unstable angina or myocardial infarction within 1 year prior to first dose of APL-101, symptomatic or unstable arrhythmia requiring medical therapy, history of congenital prolonged QT syndrome, prolonged QT interval corrected by Fridericia formula (QTcF) at screening, or concurrent treatment with a medication that is a known risk for prolonging the QT interval. Chronic controlled atrial fibrillation is not excluded.
7. Historical seropositive results consistent with active infection for hepatitis C virus (HCV) or hepatitis B virus (HBV) with high viral loads not actively managed with antiviral therapy and human immunodeficiency virus (HIV) positive subjects who are not clinically stable or controlled on their medication (asymptomatic subjects with CD4+ T-cell (CD4+) counts ≥ 350 cells/μL and have not had an opportunistic infection within the past 12 months prior to first dose of APL-101 would be eligible for study entry. If history is unclear, relevant test(s) at Screening will be required to confirm eligibility.
8. Known significant mental illness or other conditions such as active alcohol or other substance abuse that, in the opinion of the investigator, predisposes the subject to high risk of noncompliance with the protocol treatment or assessments.
9. Unable to swallow orally administered medication whole.
10. Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter drug absorption
11. Women who are breastfeeding
12. History of another malignancy within 3 years prior to C1D1. A subject with the following malignancies is allowed if considered cured or unlikely to recur within 3 years:
1. Carcinoma of the skin without melanomatous features.
2. Curatively treated cervical carcinoma in situ.
3. Bladder tumors considered superficial such as noninvasive (T1a) and carcinoma in situ (T1s), thyroid papillary cancer with prior treatment, prostate cancer which has been surgically or medically treated and not likely to recur within 3 years.
13. Subjects who are unable or unwilling to discontinue excluded medications (drugs with known QTc risk and known strong cytochrome P450 \[CYP\]3A4 inducer and/or strong inhibitors) for at least 5 half-lives prior to first dose of study drug. Subjects may qualify if such medication(s) can be safely replaced with alternate medications with less risk of drug-drug interaction.
14. Subjects with active COVID-19 infection.
15. Symptomatic and/or neurologically unstable CNS metastases, or who require an increase in steroid dose to control CNS disease. Subjects who have been receiving a stable steroid dose for at least 2 weeks prior to C1D1 may be allowed.
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