S4-41.1 – Naim Alkhouri on an NIT-based alternative path to conditional approval

S4-41.1 - Naim Alkhouri on an NIT-based alternative path to conditional approval
In this conversation, the Surfers are joined by Naim Alkhouri to discuss the recent FDA workshop for NIT development. Naim expands on ideas around an NIT-based alternative path to conditional approval as well as provides a general survey of how the workshop played out as he served as an expert panelist in Tuesday’s penultimate session.

In Season 4, Episode 41, the surfers (Jörn Schattenberg, Louise Campbell and Roger Green) review highlights from the FDA’s NIT workshop in three seperate interview sessions with guests Naim Alkhouri, Laurent Castera and Veronica Miller. Each guest participated in some form at the meeting and shares slightly differing but incredibly insightful perspectives.

This conversation covers the first part of our interview with Naim, who served as an expert panelist in Tuesday’s penultimate session. The conversation starts with Naim commending the session and noting that what drug developers and principal investigators truly are seeking is an NIT-based alternative path to conditional approval. Jörn’s response notes that the current alternative – biopsy to measure fibrosis level and NAS activity score – is imperfect, as would be an NIT approach at this time, but notes that he is not certain FDA sees the issue that way. Louise notes and commends Naim for the passion with which he made his case at the meeting. From here, Naim points out that the current system denies care or trial participation to people with clear NASH if the researcher cannot identify ballooning in the initial biopsy. Jörn suggests, and Naim agrees, that NITs need to be selected with an eye toward drug mode of action and markers. He specifically points out the value of MR technology and why he finds it superior to VCTE.

Plenty more ideas are explored as this is both a fascinating and pivotal workshop which covers a range of topics on NITs with presentations by the some of the field’s most innovative and knowledgable contributors. If you have questions or comments around the workshop, NITs, drug development or any other themes addressed in this episode, we kindly ask that you submit reviews wherever you download the discourse. Alternatively, you can write to us directly at questions@SurfingNASH.com.

Stay Safe and Surf On!

[Meeting Information from the FDA Website]

Drug development for NASH/MASH with liver fibrosis has increased substantially and there is growing interest in developing NITs to detect the presence of fibrosis, and to accurately classify different stages of fibrosis as well as cirrhosis. Candidate NITs include both blood tests (circulating biomarkers) and imaging tests. In recent years, most data regarding use of NITs have been collected from NASH/MASH clinical trials. However, there are limited published data for use of NITs across the spectrum of the affected population that would be encountered in routine clinical care, including patients without fibrosis. This workshop will assist the FDA in identifying current knowledge gaps for using NITs as diagnostic biomarkers and reasonably likely surrogates, as well as provide a framework for additional data that are needed to fill these knowledge gaps. Ultimately, the FDA seeks to learn whether expert stakeholders have evidence to indicate currently available NITs are adequate to meet the Agency evidentiary standard for assessing primary evidence of clinical efficacy. The primary focus of this workshop is “non-cirrhotic NASH/MASH population with advanced (i.e., Stage 2 or Stage 3) liver fibrosis”. The workshop will not address the use of biomarkers for treatment trials in cirrhosis due to NASH/MASH, however, the workshop will discuss identification of “progression to cirrhosis” using biomarkers.