Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
FGF401 · 1 trial · 2 indications
A dose-limiting toxicity was defined as an adverse event or abnormal laboratory value assessed as unrelated to disease, disease progression, inter-current illness, or concomitant medications that occurred within the evaluation period of DLTs and met any of the criteria listed. The estimation of the maximum tolerated dose (MTD)/recommended phase 2 dose (RP2D) of the treatment was based upon the estimation of the probability of DLT during the evaluation period for subjects in the dose determining set (DDS). A subject with multiple occurrences of a DLT under one treatment is counted only once in the AE category for that treatment. A subject with multiple DLTs within a primary system organ class is counted only once in the total row.
TTP is defined as the date of start treatment to the date of event defined as the first documented progression or death due to underlying cancer. Method used was Kaplan-Meier analysis. Group 1: HCC subjects form Asian countries; Group 2: HCC subjects form non-Asian countries
ORR is defined as the percentage of patients with a best overall response of CR or PR (RECIST v1.1). FGF401 single agent-Phase II part - Group 3 (non-HCC, other solid tumors).
| Arm | Type | Description |
|---|---|---|
| Phase I: FGF401 50 mg fasted | EXPERIMENTAL | Participants received single agent FGF401 50 mg while fasted |
| Phase I: FGF401 80 mg fasted | EXPERIMENTAL | Participants received single agent FGF401 80 mg while fasted |
| Phase I: FGF401 80 mg fed | EXPERIMENTAL | Participants received single agent FGF401 80 mg while fed |
| Phase I: FGF401 120 mg fasted | EXPERIMENTAL | Participants received single agent FGF401 120 mg while fasted |
| Phase I: FGF401 120 mg fed | EXPERIMENTAL | Participants received single agent FGF401 120 mg while fed |
| Phase I: FGF401 150 mg fasted | EXPERIMENTAL | Participants received single agent FGF401 150 mg while fasted |
| Phase I: FGF401 80 mg + PDR001 300 mg | EXPERIMENTAL | Participants received FGF401 80 mg in combination with PDR001 300 mg while fasted |
| Phase I: FGF401 120 mg + PDR001 300 mg | EXPERIMENTAL | Participants received FGF401 120 mg in combination with PDR001 300 mg while fasted |
| Phase II: Group 1 - FGF401 120 mg QD | EXPERIMENTAL | Group 1 was comprised of HCC participants from Asian countries who received single agent FGF401 120 mg QD while fasted |
| Phase II: Group 2 - FGF401 120 mg QD | EXPERIMENTAL | Group 2 was comprised of HCC participants from non-Asian countries who took single agent FGF401 120 mg QD while fasted |
| Phase II: Group 3 - FGF401 120 mg QD | EXPERIMENTAL | Group 3 was comprised of participants with other solid malignancies regardless of geography who took single agent FGF401 120 mg QD while fasted |
| Name | Type | Description |
|---|---|---|
| FGF401 | DRUG | FGF401 is a FGFR4 inhibitor. |
| PDR001 | BIOLOGICAL | PDR001 is a humanized anti-PD1 IgG4 antibody that blocks the binding of PD-L1 and PD-L2 |
Inclusion Criteria: 1. ECOG Performance Status ≤ 1 2. Presence of at least one measurable lesion according to RECIST v1.1. c-i) FGF401 single agent-Phase I and Phase II, Group 3: Patients with HCC or advanced solid tumors, who have progressed despite standard therapy or are intolerant of standard t...
FGF401 is an investigational small molecule being studied for the treatment of hepatocellular carcinoma (HCC), the most common form of primary liver cancer. It has also been evaluated in other solid tumors characterized by FGFR4 and KLB expression. FGF401 is not approved for any indication and remains in clinical development.
FGF401 targets FGFR4, a receptor tyrosine kinase. It is an FGFR4 inhibitor, meaning it is designed to block signaling through this receptor. FGFR4 signaling has been studied in liver cancer biology, and FGF401 was developed to selectively inhibit this target in tumors expressing FGFR4 and the co-receptor KLB.
FGF401 is being developed by Novartis AG, which trades on the New York Stock Exchange under the ticker NVS. Novartis sponsored the clinical trial evaluating FGF401 in hepatocellular carcinoma and other solid tumors characterized by FGFR4 and KLB expression.
FGF401 is in Phase 1 clinical development. It has not received FDA approval and is investigational. The Phase 1 trial evaluating FGF401 in hepatocellular carcinoma and solid tumors has been completed, and no active trials are currently listed for this asset.
FGF401 has been studied in one clinical trial, NCT02325739, titled "FGF401 in HCC and Solid Tumors Characterized by Positive FGFR4 and KLB Expression." This completed Phase 1 trial enrolled 172 participants across the United States, China, France, Germany, Hong Kong, Italy, Japan, Singapore, South Korea, Spain, and Taiwan.