Faron's pivotal trial outline is starting to become clearer

Translation: Original published in Finnish on 8/19/2025 at 8:05 am EEST.

Faron announced on Monday a consultation with the US authority (Food and Drug Administration). This is typically a meeting and discussion held with the authority at the end of clinical Phase II (so-called End-of-Phase II Meeting). Compared to the FDA guidance received a year ago, the upcoming pivotal marketing authorization study will focus on frontline MDS, with second-line (r/r MDS) remaining in the background. Another change compared to the previous plan relates to the dosage phase to be implemented first in the upcoming study, which will somewhat increase the total duration of the study and the number of patients compared to the previous outlook. We will review our view and forecasts no later than in connection with the H1 report to be published next Wednesday.

FDA's feedback shaped the design of the upcoming pivotal trial in some respects

According to the release, the FDA advised Faron to conduct the pivotal study in high-risk MDS patients in the frontline. This refers to patients who receive treatment for their disease for the first time (treatment-naive). A change from before is that, if the results support it, possible accelerated approval can be sought for frontline MDS, but not, in our understanding, for second-line r/r MDS based on this study. Previously, an r/r MDS application was on the roadmap as a possibility before a frontline application, according to comments made by the FDA last summer. The planned patient count was not commented on in the release, which is an interesting follow-up question ahead of the upcoming H1 report.

The Phase II/III trial first determines the appropriate dosage

A new piece of information that also emerged was the trial's opening dose-response phase. This means that initially, the most effective dose (1 vs. 3 mg/kg) will be determined, after which the study will be continued seamlessly with the most effective dose. This was somewhat of a surprise to us, as the appropriate dosage was already sought in the previous study phase. In practice, the new dosage study may prolong the study's completion timeline and somewhat increase the required number of patients compared to the previous outlook.

Primary endpoints are complete responses and overall survival

Information was also obtained on the criteria used in the study. The interim readout will use complete responses (CR and CReq) according to the IWG2023 criteria as the primary endpoint. These are strong responses that best correlate with overall survival and can therefore be used when evaluating a potential accelerated marketing authorization application. In our view, Faron originally proposed using so-called cCR responses (composite complete remission) as the primary endpoint. According to FDA guidance, cCR can be used as a secondary endpoint, i.e., to support primary efficacy data. In our view, the criteria used or the precise response types are not of great significance to Faron or the practical implementation of the study. We believe that overall survival (OS) will be the key variable determining final marketing authorization in the later stage of the study. Decisive results to this end will only be available at the final phase of the trial.

We expect further information in connection with the H1 report

Faron's H1 report will be released on Wednesday, August 27, when the company is also expected to host a webcast. We expect the company to clarify information regarding the meeting held, and particularly the more detailed execution and timeline of the planned future trial. We will review our forecasts and view in more detail in connection with the H1 update.