A Phase II, Single Arm Study of BGJ398 in Patients With Advanced Cholangiocarcinoma

Adult patients with histologically or cytologically confirmed advanced or metastatic

cholangiocarcinoma with FGFR2 gene fusions or translocations or other FGFR genetic

alterations have been enrolled. Subjects must have received at least one prior regimen

containing gemcitabine with or without cisplatin for advanced/metastatic disease.

Subjects should have had evidence of progressive disease following their prior regimen or

if prior treatment was discontinued due to toxicity must have continued evidence of

measurable disease. Up to approximately 160 adult patients over age 18, both male and

female were planned for enrollment.

Three cohorts of subjects comprise the study population:

Cohort 1: subjects with FGFR2 gene fusions (ie, fusions or rearrangements [formerly

translocations]).

Cohort 2: subjects with FGFR genetic alterations other than FGFR2 gene fusions or

rearrangements.

Cohort 3: subjects with FGFR2 gene fusions or rearrangements who have received a prior

FGFR inhibitor.

All subjects received oral BGJ398 (infigratinib), once-daily, on a three weeks on (21

days), one week off (7 days) schedule. One treatment cycle consists of 28 days.

Notes:

Cohort 1 was pre-specified as the primary analysis population. Results of these analyses

were previously disclosed (posted 22 June 2022). There were no additional efficacy or

safety endpoints to assess in Cohort 1 after primary completion (01 March 2021).

Cohorts 2 and 3 were added at protocol amendment (PA) 4 to support only exploratory

efficacy objectives of the study. These cohorts were ongoing the time of primary

completion (01 March 2021). After interim review of the data from these cohorts (as

permitted by the protocol) only limited efficacy was observed and the sponsor terminated

the study early. Therefore, a formal efficacy analysis was not performed for Cohorts 2

and 3. However, baseline characteristics and safety data were analyzed.