Table of contents

• Trial overview
• Study populations
• Study phases and design
• Outcomes being measured
• Trial status and enrollment
• What these trials mean for patients

Trial overview

Tyra-300-B01 is being studied in two interventional cancer trials. One study is in non-muscle invasive bladder cancer, and the other is in advanced urothelial carcinoma and other solid tumors with activating FGFR3 gene alterations.^[1][2]

Both studies are open-label, which means the study team and the participants know what treatment is being given.^[1][2]

Study populations

The Phase 2 study is focused on people with non-muscle invasive bladder cancer.^[1] This is bladder cancer that has not grown into the muscle layer of the bladder wall.

The Phase 1/2 study includes people with advanced urothelial carcinoma and other solid tumors that have activating FGFR3 gene alterations.^[2] These gene alterations are changes that may be linked to cancer growth, and the study is aimed at tumors with this specific feature.

Study phases and design

The bladder cancer study is a Phase 2 trial with an enrollment of 90 participants.^[1] Phase 2 studies mainly look at whether a treatment shows signs of benefit while continuing to check safety.

The other study is a Phase 1/2 trial with 261 planned participants.^[2] Phase 1 is the early part of the study and focuses on safety and dose finding, while Phase 2 looks more closely at anti-tumor activity, which means how well the treatment works against the cancer.^[2]

The Phase 1 part of the study includes Parts A and B and measures dose-limiting toxicity during Cycle 1, which is the first 28 days of treatment.^[2] Dose-limiting toxicity means a side effect severe enough to limit how much treatment can be given safely.

Outcomes being measured

The main outcome in the Phase 2 bladder cancer study is complete response rate at 3 months.^[1] Complete response means no signs of cancer are seen based on the study’s rules.

In the Phase 1/2 study, the early phase measures the incidence of dose-limiting toxicity during the first treatment cycle.^[2] The later phase measures investigator-assessed objective response rate, which includes complete response or partial response by RECIST v1.1.^[2]

Objective response rate is the share of participants whose tumors shrink or disappear according to standard cancer trial rules.^[2] Partial response means the tumor gets smaller, but does not fully disappear.

The Phase 1/2 study also aims to find the optimal dose, the maximum tolerated dose, and the recommended Phase 2 dose of Tyra-300-B01.^[2] The recommended Phase 2 dose is the dose chosen for later testing after early results show it is suitable for further study.

Trial status and enrollment

The Phase 2 bladder cancer study is listed as Authorised and plans to enroll 90 people.^[1]

The Phase 1/2 study is listed as Suspended and plans to enroll 261 people.^[2] Suspended means the trial is not currently moving forward as planned.

What these trials mean for patients

These studies are testing Tyra-300-B01 in specific cancer groups rather than in a broad patient population.^[1][2] The focus is on whether the treatment can produce responses in bladder cancer and in tumors with activating FGFR3 gene alterations.

For patients, the most important points are the study phase, the cancer type being studied, and the main outcome being measured.^[1][2] In these trials, researchers are mainly looking at safety, dose selection, and signs that the cancer responds to treatment.