Rivoceranib Phase 2 Trial in Adenoid Cystic Carcinoma Posts Final Results

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Elevar Therapeutics posted final results on ClinicalTrials.gov on July 30, 2026 for a phase 2 trial of the VEGFR-2 inhibitor rivoceranib in recurrent or metastatic adenoid cystic carcinoma reporting an investigator-assessed objective response rate of 13.8% among 80 enrolled patients despite early termination by the sponsor. The single-arm, open-label design and absence of a comparator arm limit causal inference, yet the data add a prospective multicenter dataset for this rare salivary gland malignancy.

Results were posted providing publicly available efficacy and safety data from this terminated multicenter study.

13.8%

ORR (Investigator)

80 patients

Enrolled

74.7%

12-Month OS

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AI-generated conceptual illustration; not study data.

Efficacy and Survival Signals From a Terminated Phase 2 Study

The trial enrolled 80 patients with histologically confirmed, recurrent or metastatic adenoid cystic carcinoma across multiple centers. Participants received rivoceranib 700 milligrams orally once daily in continuous 28-day cycles until disease progression, unacceptable toxicity, or withdrawal. The primary endpoint was objective response rate assessed by investigators per RECIST version 1.1, with independent central review as a key secondary endpoint. The study was designed as a single-group, open-label, multicenter interventional trial without a comparator arm.

Phase 2, open-label, single-group, multicenter, interventional; 28-day continuous cycles. 80 patients with recurrent or metastatic adenoid cystic carcinoma, actual enrollment. Intent-to-treat population, n equals 80; efficacy-evaluable subsets reported separately. Objective response rate per RECIST 1.1 by investigator assessment.

AI-generated conceptual illustration; not study data.

Investigator-assessed objective response rate reached 13.8%, with a 95% confidence interval spanning 7.1% to 23.3%. Independent central review yielded a lower rate of 8.8%, with a confidence interval of 3.6% to 17.2%, reflecting the well-known inter-reader variability in assessing response in adenoid cystic carcinoma, a tumor characterized by indolent growth patterns and mixed solid, cribriform, and tubular histologies that complicate radiographic interpretation.

Overall survival data showed a 12-month rate of 74.7% and a 24-month rate of 51.5%, though the wide confidence intervals around these estimates reflect the small sample size and the heterogeneous natural history of adenoid cystic carcinoma. Progression-free survival rates were also reported at six months, 12 months, and two years, providing a temporal profile of disease control under continuous VEGFR-2 inhibition. The trial was terminated early by the sponsor for strategic redirection rather than for safety concerns, and no participants completed the full treatment protocol. Forty-four of the enrolled patients died during the study period, a figure that reflects both the advanced disease stage at enrollment and the aggressive biology of recurrent adenoid cystic carcinoma.

Context for a Rare and Therapeutically Challenging Malignancy

Adenoid cystic carcinoma is rare, and its propensity for perineural invasion, late distant metastasis, and relentless local recurrence makes it a clinically challenging tumor. Treatment options remain limited, and rivoceranib is not FDA-approved for this disease. The rivoceranib data, while modest, contribute to a small but growing evidence base for anti-angiogenic strategies in salivary gland cancers and provide a benchmark for future investigation.

The discrepancy between investigator-assessed and centrally reviewed response rates carries methodological significance for future trial design. Adenoid cystic carcinoma frequently exhibits slow, indolent progression that can mimic stable disease on imaging, making RECIST-based response assessment inherently difficult. Future studies may benefit from incorporating volumetric analysis, functional imaging, or patient-reported symptom endpoints alongside conventional radiographic criteria to capture clinically meaningful benefit more accurately.

From a drug development perspective, the observed response rate, while below typical thresholds for accelerated approval in more common tumor types, is hypothesis-generating and requires controlled confirmation before it can be interpreted as clinical benefit. The survival data, showing that roughly three-quarters of patients were alive at one year, provide a benchmark against which future combination strategies and novel agents can be measured in prospective comparative settings.

Design Constraints and Unresolved Safety Questions

The trial limitations are substantial and must be weighed carefully against its findings. The single-arm, open-label design precludes any attribution of observed outcomes to rivoceranib specifically, as the natural history of adenoid cystic carcinoma is highly variable and selection bias in enrollment cannot be excluded. The absence of a control arm means that the reported response and survival figures cannot be compared against best supportive care or alternative systemic therapies, leaving the magnitude of any true treatment effect uncertain.

AI-generated conceptual illustration; not study data.

Early termination by the sponsor for strategic redirection introduces additional uncertainty. The decision was not driven by safety signals, but it means the study did not reach its planned enrollment or follow-up duration, and the posted results represent an incomplete dataset. No participants completed the treatment protocol, and the high mortality among enrolled patients underscores the advanced and aggressive nature of the study population. The wide confidence intervals surrounding all efficacy endpoints reflect the small sample size and limit the precision of effect estimates.

Subgroup analyses, while reported, are underpowered and should be interpreted as hypothesis-generating only. The absence of biomarker stratification, including MYB-NFIB fusion status or NOTCH mutation analysis, represents a missed opportunity to identify predictive subgroups that might derive disproportionate benefit from VEGFR-2 inhibition. Safety data, while collected, have not yet undergone peer-reviewed publication, and the full adverse event profile remains unavailable for independent scrutiny. Clinicians should await formal publication before drawing definitive conclusions about the risk-benefit balance of rivoceranib in this setting.

Future Directions and Evidence Gaps

The posted results establish a foundation for future investigation rather than a basis for clinical adoption. Several research directions emerge from the data. First, combination strategies pairing VEGFR-2 inhibition with immune checkpoint inhibitors, mTOR pathway agents, or NOTCH pathway modulators warrant preclinical and early-phase clinical evaluation, given the molecular landscape of adenoid cystic carcinoma and the limited single-agent activity observed here.

Second, the methodological challenges highlighted by the investigator-versus-central-review discrepancy argue for the development of disease-specific response criteria. International collaborative groups, including the Adenoid Cystic Carcinoma Research Foundation and the European Organisation for Research and Treatment of Cancer, are well positioned to convene expert consensus on endpoint selection and imaging protocols for future trials in this rare disease setting.

Third, the survival benchmarks reported here can inform sample size calculations and control arm assumptions for randomized studies. Prospective registries capturing treatment patterns and outcomes across multiple institutions would complement interventional trials by providing real-world context for efficacy signals observed in controlled settings. Peer-reviewed publication of the full dataset, including detailed safety analyses and patient-level outcomes, remains the essential next step before these results can be integrated into clinical evidence syntheses or guideline deliberations.

This summary draws exclusively on data posted to ClinicalTrials.gov by the study sponsor. No peer-reviewed publication of these results was available at the time of writing. All numeric values are reported as posted by the sponsor without independent verification. The trial was terminated early, and the posted dataset may not represent the complete evidence base. Readers should consult the primary registry entry for full protocol details and await peer-reviewed publication for validated interpretations.

ClinicalTrials.gov registry entry: https://clinicaltrials.gov/study/NCT04119453. API data source: https://clinicaltrials.gov/api/v2/studies/NCT04119453.

This article is not medical advice. It summarizes publicly posted clinical trial results for educational purposes. Rivoceranib is not approved for adenoid cystic carcinoma in any major regulatory jurisdiction. Treatment decisions require individualized assessment by qualified oncologists. Patients should discuss therapeutic options with their treating physicians and consider clinical trial enrollment where appropriate. No commercial relationships influenced this summary.