Beacon-BTC is a global Phase III clinical trial evaluating the efficacy and safety of nanvuranlat as a second-line treatment for advanced biliary tract cancer.

In Part A, the dose and dosing regimen to be used in the subsequent stage of development will be selected. In Part B, the selected nanvuranlat dose and regimen will be compared with Physician’s Best Choice (PBC).

In June 2026, J-Pharma announced the enrollment and randomization of the first patient, and the study is currently underway.

Treatment options remain limited for patients with biliary tract cancer whose disease progresses following first-line treatment.

Based on clinical study results and other findings to date, J-Pharma is continuing the development of nanvuranlat as a second-line treatment.

It means that the trial has moved beyond the planning and registration stage and into actual patient enrollment and assignment to treatment groups.

However, no conclusions about the trial outcome, efficacy, safety, or the likelihood of regulatory approval can be drawn from this milestone alone.

In Part A, four treatment groups will be compared: three nanvuranlat groups using different doses and dosing regimens, and one Physician's Best Choice (PBC) group. The results will be used to select the nanvuranlat dose and regimen for Part B.

In Part B, the selected nanvuranlat dose and regimen will be compared with PBC to evaluate efficacy and safety.

An investigator-initiated clinical trial is a clinical trial planned and conducted primarily by physicians or medical institutions rather than by a pharmaceutical company.

This study (JON-2404-B) is being conducted primarily by the Cancer Institute Hospital of JFCR. Under an agreement with the hospital, J-Pharma provides support for the smooth conduct of the trial.

In July 2026, J-Pharma announced that the first patient had been dosed.

The study is registered on jRCT as an investigator-initiated clinical trial with a status of “Recruiting,” and has now moved into the patient enrollment and dosing stage.

Beacon-BTC is a global Phase III clinical trial comparing nanvuranlat monotherapy with Physician’s Best Choice (PBC) as a second-line treatment for biliary tract cancer.

In contrast, this investigator-initiated trial evaluates nanvuranlat in combination with durvalumab in patients with biliary tract cancer who are transitioning to maintenance therapy with an immune checkpoint inhibitor following first-line treatment. The trial primarily assesses the safety and tolerability of this combination therapy.

The two trials differ in their treatment settings, clinical development phases, and objectives.

According to publicly available information on jRCT, Part A evaluates the safety and tolerability of nanvuranlat monotherapy, while Part B evaluates the safety and tolerability of nanvuranlat in combination with durvalumab.

The primary endpoints include the types and incidence of dose-limiting toxicities (DLTs). Efficacy-related measures, including progression-free survival (PFS), response rate, and overall survival (OS), are also evaluated as secondary endpoints.

Treatment regimens that include immune checkpoint inhibitors are now standard in the first-line treatment of biliary tract cancer.

LAT1 inhibition by nanvuranlat may affect the tumor immune microenvironment, and nonclinical studies have suggested enhanced antitumor effects when nanvuranlat is combined with an immune checkpoint inhibitor.

Based on these findings, the study evaluates the safety, tolerability, and other parameters of adding nanvuranlat to maintenance therapy with an immune checkpoint inhibitor.

However, results from nonclinical studies alone cannot be used to determine whether the combination will be clinically effective.

This trial explores the potential to develop nanvuranlat for biliary tract cancer beyond the second-line treatment setting. The findings may help inform future development strategies.

JPH034 is a LAT1 inhibitor being developed by J-Pharma for central nervous system disorders. Nonclinical studies in monkeys have demonstrated high brain penetration, and J-Pharma is pursuing its development for central nervous system disorders, including multiple sclerosis.

No. The ongoing Phase I trial in the United States is an early-stage clinical trial involving healthy volunteers. It is designed to collect data on safety, tolerability, pharmacokinetics, and other parameters, rather than evaluate the efficacy of JPH034 in treating multiple sclerosis.

The study primarily evaluates safety, tolerability, pharmacokinetics, and related parameters. These data will help inform dose selection and the design of subsequent clinical trials.

Like nanvuranlat, JPH034 is being developed for the global market.

J-Pharma has announced plans to collect safety, tolerability, and pharmacokinetic data through its Phase I clinical trial in the United States, with oversight from the U.S. Food and Drug Administration (FDA). These data will support the planning of Phase II and subsequent clinical trials in the United States and Europe.

Both JPH034 and nanvuranlat are LAT1 inhibitors, but they differ in the diseases they target and how they are distributed in the body.
Nanvuranlat reaches high concentrations in bile and is being developed primarily for cancer.
JPH034, in contrast, has high brain penetration and is being developed for central nervous system disorders.

J-Pharma plans to use the safety, tolerability, pharmacokinetic, and other data obtained from the Phase I trial to guide its plans for Phase II and subsequent clinical development in the United States and Europe.

As part of its efforts toward global development and commercialization, J-Pharma signed a non-binding licensing term sheet with a European biotechnology company in September 2026. The Company will continue discussions with the aim of entering into a definitive agreement.

When considering potential partners for the future development and commercialization of JPH034, J-Pharma believes it is important to evaluate several factors, including financial terms, alignment of development strategies, expertise in the target therapeutic area, development and commercialization capabilities, and the priority a potential partner would place on JPH034.

As part of its efforts toward global development and commercialization, J-Pharma signed a non-binding licensing term sheet with a European biotechnology company in September 2026. J-Pharma will continue discussions with the aim of entering into a definitive agreement.

J-Pharma will disclose information regarding the agreement terms as appropriate, to the extent permitted under contractual confidentiality obligations and agreements with the counterparty.

Any matters that may materially affect investment decisions will be disclosed in a timely and appropriate manner in accordance with the Financial Instruments and Exchange Act and the Tokyo Stock Exchange's timely disclosure rules and other applicable regulations.

J-Pharma will consider appropriate financing options based on several factors, including the development progress of its pipeline products, including JPH034, anticipated research and development funding requirements, available cash reserves, and opportunities to secure external funding.

Any financing decisions will also take into careful consideration their potential impact on shareholder value. If J-Pharma decides to undertake a specific financing transaction that requires disclosure, it will make an announcement in a timely and appropriate manner.