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Larimar Therapeutics Larimar Therapeutics Reports Second Quarter 2021 Operating and Financial Results August 12 , 2021 - Reported positive proof - of - concept and dose response data from Phase 1 program evaluating CTI - 1601 in patients with Friedreich's ataxia ( FA ) - Subcutaneous injections of CTI - 1601 at doses of 50 mg or 100 mg resulted in frataxin levels in buccal cells of FA patients that were at or in excess of those that would be expected in phenotypically normal heterozygous carriers - Phase 1 safety data indicate that repeated subcutaneous injections of CTI - 1601 were generally well tolerated at doses up to 100 mg administered daily for 13 days - Completed dosing in July in 180 - day non - human primate toxicology study designed to support extended dosing of CTI - 1601 - Cash and investments of $ 70.6 million as of June 30 , 2021 - Closed $ 20 million equity financing on July 2 , 2021 BALA CYNWYD , Pa . , Aug. 12 , 2021 ( GLOBE NEWSWIRE ) -- Larimar Therapeutics , Inc. ( " Larimar ” ) ( Nasdaq : LRMR ) , a clinical - stage biotechnology company focused on developing treatments for complex rare diseases , today reported its second quarter and year to date June 30 , 2021 operating and financial results . " We finished the second quarter in a strong financial position and with a compelling clinical data set that demonstrates proof - of - concept for CTI - 1601 , which to our knowledge is the only clinical - stage candidate designed to address the root cause of Friedreich's ataxia , " said Carole Ben - Maimon , MD , President and Chief Executive Officer of Larimar . " These positive Phase 1 data along with non - clinical pharmacology data demonstrate proof- of - concept , and CTI - 1601's differentiated mechanism of action helped us to earn a PRIME designation from the European Medicines Agency , providing us with valuable regulatory benefits and important external validation . Looking forward , we continue to collect and analyze data from our 180 - day non - human primate toxicology study and remain confident that there is a path forward through the resolution of the CTI - 1601 clinical hold and towards the initiation of our Jive open - label extension and pediatric multiple ascending dose trials . " Second Quarter 2021 Highlights • In May 2021 , Larimar reported positive topline data from its Phase 1 Friedreich's ataxia ( FA ) program after completing dosing of the single ascending dose ( SAD ) trial in December , 2020 and of the multiple ascending dose ( MAD ) trial in March , 2021. Data from these trials demonstrate proof - of - concept by showing that daily subcutaneous injections of CTI - 1601 for up to 13 days resulted in dose - dependent increases in frataxin levels from baseline compared to placebo in all evaluated tissues ( buccal cells , skin , and platelets ) . Frataxin levels achieved in peripheral tissues ( buccal cells ) following daily 50 mg and 100 mg subcutaneous injections of CTI - 1601 were at or in excess of frataxin levels that would be expected in phenotypically normal heterozygous carriers . There were no serious adverse events ( SAEs ) , associated with either the MAD or SAD trials . • In May 2021 , Larimar received European Medicines Agency ( EMA ) Priority Medicines ( PRIME ) designation for CTI - 1601 in FA . Through PRIME , the EMA offers early and proactive support to medicine developers to optimize the generation of robust data on a medicine's benefits and risks and enables accelerated assessment of medicines applications so that these medicines can reach patients earlier . Larimar's PRIME designation was based on pre - clinical data as well as tolerability data from the CTI - 1601 Phase 1 program in patients with FA . • On May 25 , 2021 the United States Food and Drug Administration ( FDA ) placed a clinical hold on the CTI - 1601 clinical program following the Company's notification to the FDA of mortalities which occurred at the highest dose levels in an ongoing 180 - day non - human primate ( NHP ) toxicology study , which is designed to support extended dosing of patients with CTI - 1601 . In the clinical hold letter , the FDA stated that it needs a full study report from the ongoing NHP study and Larimar may not initiate additional clinical trials until the company has submitted the report and received notification from the FDA that additional clinical trials may commence . At the time of the notice , the Company had no interventional clinical trials with patients enrolled or enrolling . • In July 2021 , the Company completed dosing in the 180 - day NHP toxicology study discussed above . The Company is currently collecting and analyzing data from the study . While there is no way to predict the FDA's response or whether they will require additional data or testing before lifting the clinical hold on CTI - 1601 in full or in part , the Company expects to initiate its Jive open - label extension and pediatric MAD trials in the first half of next year ,