Poxel has set out a new vision focused on midphase programs in rare metabolic diseases. The new strategy hits pause on plans to start a phase 2b nonalcoholic steatohepatitis (NASH) trial and makes no mention of the next steps for phase 3 ready diabetes drug imeglimin.
France-based Poxel has been focused on imeglimin for years but has struggled to advance the oral oxidative phosphorylation blocker. Poxel identified the dose to take into phase 3 in 2014 but lacked a partner to support the trial until landing a deal with Roivant Sciences in 2018. However, Metavant, the Roivant company created to take imeglimin forward in the West, walked away late last year.
While Sumitomo Dainippon has won approval for imeglimin in Japan, Poxel’s focus is now on other assets. The new strategic direction set out by Poxel on Monday lacks details about the next steps for imeglimin in the U.S. and Europe.
Instead, the statement, and by extension Poxel’s R&D strategy, is focused on midphase clinical trials of PXL065 and PXL770. Poxel plans to start phase 2a proof-of-concept trials of deuterium-stabilized R-pioglitazone PXL065 and AMPK activator PXL770 in adrenomyeloneuropathy patients early in 2022, with data forecast to follow by the end of next year.
PXL065 is already in a phase 2 NASH trial that is expected to deliver topline data on which dose to take into a pivotal program around the third quarter of 2022. Poxel put the completion date at March 2022 when it registered the study on ClinicalTrials.gov.
Poxel had planned to start a phase 2b trial of PXL770 in NASH. However, the study is on hold pending the delivery of results from the PXL065 NASH trial and the adrenomyeloneuropathy proof-of-concept studies. The decision to wait for data from the other trials suggests it will be 2023 before PXL770 enters phase 2b, if it happens at all.
The emergence of adrenomyeloneuropathy, the largest subtype of X-linked adrenoleukodystrophy, at the center of Poxel’s R&D plans marks a significant shift. Poxel listed the indication as an additional opportunity in its June corporate presentation. Now, with the PXL770 phase 2b postponed and hopes for imeglimin outside of Japan fading, the rare spinal cord disease is one of the main opportunities.