No end in sight for Larimar’s hold woes

 Larimar Therapeutics is on the slide – again. Fears over the future of its Friedreich’s ataxia project CTI-1601 caused shares to fall 66% today, after the FDA yesterday failed to lift a clinical hold and requested further data. Last May the regulator halted work on CTI-1601 after several deaths in non-human primates. Today there were also concerns about Larimar’s cash position, despite assurances of funding until 2023. Larimar now risks falling further behind in the race to get a Friedreich’s product to market, a prize that could be claimed by Reata, which is set to complete a rolling NDA for its Nrf2 activator omaveloxolone by the end of this quarter, leading to a potential launch in early 2023 – although the company's credibility took a hit last year over its lead project, bardoxolone. Others are also edging closer to the finish line. PTC Therapeutics and Retrotope have projects in phase 3, with pivotal data from the former’s vatiquinone due in the second half of 2023, and Retrotope expected to report data for RT001 soon, after competing its 65-patient study in December. Even if CTI-1601 does get back into the clinic the market might already be sewn up.

The Friedreich's ataxia clinical pipeline
Project	Company	Description	Trial details
Phase 3
Omaveloxolone	Reata	Nrf2 stimulant	Rolling NDA to complete in Q1 2022
RT001	Retrotope	Deuterated polyunsaturated fatty acid	Ph3 completed Nov 2021
Vatiquinone (EPI-743)	PTC Therapeutics	15-lipoxygenase inhibitor	Move-FA completes Apr 2023
Phase 2
Leriglitazone (MIN-102)	Minoryx Therapeutics	PPAR-gamma agonist	Frames reported Dec 2020; primary endpoint "inconclusive"; development continues
Phase 1
CTI-1601	Larimar Therapeutics	Recombinant fusion protein	Ph1 MAD trial reported May 2021; on clinical hold
IXC-109	Ixchel Pharma	Monomethyl fumarate prodrug	Not in trial databases
Source: Evaluate Pharma. https://www.evaluate.com/vantage/articles/news/corporate-strategy-snippets/no-end-sight-larimars-hold-woes