Abstract 50P
Background
Pemigatinib is a selective FGFR inhibitor that showed highly effectiveness and tolerability in patients with cholangiocarcinoma, which has been demonstrated in Fight 202 study with an ORR of 35.5%. However, pemigatinib has not been investigated in Chinese population with cholangiocarcinoma(CCA).
Methods
Patients aged 18 years or older with recurrent or metastatic CCA that failed to at least 1 line prior systemic therapy were enrolled. In stage 1, 3 subjects were enrolled regardless of the FGFR2 status and were treated at 9mg pemigatinib. The other 31 subjects with documented FGFR2 fusion or rearrangement were enrolled in stage 2 and received 13.5 mg pemigatinib. From 2/26/2020 to 1/29/2021, all the subjects in both stages were orally given pemigatinib QD on a 2 weeks on/1 week off schedule until disease progression, unacceptable toxicity, withdrawal of consent, or physician decision. The primary end point was ORR assessed by independent radiological review committee (IRRC) per RECIST V1.1 in 31 patients enrolled in stage 2.
Results
As of Jan 29, 2021, 30 subjects in stage 2 were included in the efficacy evaluable population with 1 participant excluded due to inadequate FGFR2 aberrant frequency. Among 30 efficacy evaluable subjects, 15 of them had confirmed response assessed by IRRC, with an ORR of 50% (95% CI: 31.3%, 68.7%). With a median follow up of 5.13 months, 13 patients were still in response, the median DOR was not reached (95% CI: 3.4, NR), and the median PFS was 6.3 (95% CI: 4.9, NR) months. The DCR was 100% (95% CI: 88.4%, 100%). All 34 subjects in both stages were included for safety analysis. As of data cutoff date, each subject experienced at least 1 treatment-related adverse event (TRAE), the most common TRAEs were hyperphosphatemia (73.5%), xerostomia (55.9%) and alopecia (50.0%), and 14.7% had grade 3 or higher TRAEs. Three participants had SAEs, which were rectal polyps, abnormal liver function and bile duct infection. There was no treatment discontinuation and deaths due to TRAE.
Conclusions
Pemigatinib was highly effective and tolerable in Chinese patients with recurrent or metastatic CCA with FGFR2 fusion or rearrangement.
Clinical trial identification
NCT04256980.
Editorial acknowledgement
Shi G-M and Huang X-Y contributed equally to this work.
Legal entity responsible for the study
Innovent Biologics, Inc.
Funding
Innovent Biologics, Inc.
Disclosure
Y. Luo: Other, Personal, Employee: Innovent Biologics, Inc.. Y. Wang: Other, Personal, Employee: Innovent Biologics, Inc.. All other authors have declared no conflicts of interest.