Larimar Therapeutics reports Q3 EPS (24c), consensus (37c)

“Our nomlabofusp program continues to advance, with the potential to be the first frataxin protein replacement therapy for patients with FA. All sites are activated with ongoing enrollment in our OLE study evaluating the long-term safety, PK and frataxin levels in patients with FA following daily subcutaneous administration. In mid-December, we plan to provide a development program update that will include available safety, PK, and frataxin data from patients receiving 25 mg of nomlabofusp daily for up to 180 days in our OLE study. We expect to also provide an update on enrollment,” said Carole Ben-Maimon, CEO. “In November, we look forward to presenting at the International Congress for Ataxia Research meeting new data on results from our completed dose exploration study including exploratory gene and lipid expression results following nomlabofusp treatment. In addition, we will also be presenting two posters with data from patients participating in our Phase 1 single ascending dose and multiple ascending dose studies and Phase 2 dose exploration study. One poster will provide patient data on baseline disease characteristics and baseline tissue frataxin levels, and the other will present the relationship between dose, PK and tissue frataxin levels using modeling and simulation. We remain on track to initiate a PK run-in study in adolescent patients with FA in the fourth quarter of this year which is the first step towards evaluating nomlabofusp treatment in pediatric patients. On the regulatory front, we were pleased to recently receive Innovative Licensing and Access Pathway (ILAP) designation from the Medicines and Healthcare Products Regulatory Agency which aims to facilitate patient access by accelerating time to market in the U.K. We also held our first meetings with the Food and Drug Administration as part of the Support for Clinical Trials Advancing Rare Disease Therapeutics pilot program and appreciate the dialogue and interaction designed to help advance our development program. In parallel, we began work to further understand the continued areas of unmet need in the FA therapeutic landscape from the perspective of physicians, payers, and most importantly, patients. We will use this information to develop our commercial approach and to refine our market entry strategy. Finally, we continue scaling up our manufacturing efforts and collecting required data to support a potential accelerated approval path. Our targeted BLA submission remains on track for the second half of 2025.”