CTI-1601 is a recombinant fusion protein from Larimar Therapeutics intended to deliver human frataxin into the mitochondria of patients with Friedreich’s ataxia (FA) today announced topline data from its Phase 1 multiple ascending dose (MAD) clinical trial (n=27) evaluating CTI-1601 as a treatment for FA.

Phase 1 data show that repeated subcutaneous administration of CTI-1601 resulted in dose-dependent increases in frataxin (FXN) levels from baseline compared to placebo controls. These dose-dependent increases in frataxin levels were seen in all evaluated tissues (buccal cells, skin biopsies, and platelets) with daily dosing. The median change from baseline in frataxin levels observed for each dosing group and tissue. Source - Larimar Therapeutics.

Based on strong domain and business knowledge Thelansis Knowledge Partners has published the market insight report on Friedreich’s ataxia (FA) to provide a clear understanding on disease area background, epidemiology, current and future competitions, country specific standard of care, and the complete market forecast for 2020 to 2030. Based on Epidemiology study of Thelansis 18,660 patients are likely to be diagnosed patient in 6 Major markets (USA and EU5), out of which 16,800 patients will able to meet the treatment for the year 2021 for the same 6 major markets.

The Friedreich’s ataxia (FA) market insight report provides a comprehensive view of disease events, severity, identified and emerging biomarkers, Clinical Manifestations, progression, signs & symptoms, risk factors, pathogenesis, genetic basis and treatment guidelines with respect to different lines of therapy at country level. Comprehensive insight on patient segmentation based on age (age ≤20 age, 21 to 25 age, 26 to 39 age and 40 and more age), sex, classification based on Homozygous and Heterozygous screening cases, by Signs & Symptoms (early onset and atypical) based on comorbidities (Scoliosis and Pescavus; Hypertrophic Cardiomyopathy; Diabetes or Glucose Intolerance; and Hearing Loss or Optic Atrophy; and Others) has been provided into the epidemiology section of the study. However the current treatment (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, there are several pharmaceutical products are being approved as well as under different phases of development for the treatment. The key companies in to advance stage of developments are Reata Pharmaceuticals, PTC Therapeutics, Retrotope, Metro International Biotech, Larimar Therapeutics, Inc, Minoryx Therapeutics, Takeda, Horizon Pharma, Santhera Pharmaceuticals, Cardero Therapeutics targeting the different set of syndromes associated with Friedreich’s ataxia Looking at the current clinical development and assets progress card, it would really a great opportunity for the pharmaceutical company to build products around the treatment of FA.

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