Larimar Therapeutics Announces Positive Topline Phase 1 Clinical Trial Data of CTI-1601 in Friedreich ataxia patients.

Larimar Therapeutics, Inc., a clinical-stage biotechnology company focused on developing treatments for Friedreich ataxia and other complex rare diseases, announced today topline data from its Phase 1 multiple ascending dose (MAD) clinical trial (n=27) evaluating CTI-1601 as a FA treatment. Over 13 days, FA patients were given subcutaneous injections of CTI-1601 or a placebo at increasing doses and frequencies.

Publish Date: 11-05-2021   Source: Larimar Therapeutics, Inc.

Friedreich ataxia is a rare inherited disease that affects the nervous system and causes movement issues. It usually starts in childhood and progresses to ataxia or a loss of muscle coordination. About 25% of people with FA have an atypical form in which signs and symptoms begin after age 25. People of European, North African, or Middle Eastern ancestry are susceptible to this condition. It is uncommonly found in other ethnic groups. In most cases, it is a recessively inherited disorder caused by a GAA repeat expansion mutation in the first intron of the FXN gene, resulting in primarily reduced frataxin protein expression. Deficiency of frataxin in cells leads to decreased activities of the mitochondrial iron-sulfur cluster–containing enzymes, accumulation of iron in the mitochondrial matrix, increased sensitivity to oxidative stress, and impaired adenosine triphosphate (ATP) production.

• The incidence of Friedreich ataxia in the USA ranges between 2.5 to 3.7 cases per 100,000 population.

However, the current Friedreich ataxia treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (Leriglitazone, Epicatechin, Omaveloxolone, RT001, EPI-743, JOT101, and Others), market share, market uptake, attribute analysis has been provided under the market outlook section of the study covering 6 MM countries; the United States, EU5 (Germany, Spain, France, Italy, UK).

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for Friedreich ataxia treatment. The key companies in the advanced development stage are Reata Pharmaceuticals, PTC Therapeutics, Retrotope, Metro International Biotech, Larimar Therapeutics, Inc, Minoryx Therapeutics, etc.. targeting the different sets of syndromes associated with FA.

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Friedreich ataxia to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2021 to 2032.