Fri, 20 May 2022

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) 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 was observed for each dosing group and tissue. Source-Larimar Therapeutics.

Friedreichs Ataxia .

Based on the Thelansis market insight report on Friedreich's ataxia (FA) which provides a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2020 to 2030.

The prevalence and incidence cases of Friedreich's ataxia varies worldwide considering, ranging from 0.97 to 2.5 cases per 100,000 in the USA and approximately 1.2 to 2.37 cases per 100,000 population in Europe. Germany is the leading country among EU region in terms of total diagnosed and prevalent cases of Friedreich's ataxia.

Based on the Epidemiology study of Thelansis 18,660 patients are likely to be diagnosed patients in 6 Major Markets (the USA and EU5), out of which 17,150 patients will able to meet the treatment for the year 2022 for the same 6 major markets.

The Friedreich's ataxia (FA) market insight report provides a comprehensive insight on patient segmentation based on age, sex, Genotype, phenotype, Signs & Symptoms (LOFA & VLOFA) (Atypical & typical), Comorbid conditions (Cardiac problems, Visual problems, Auditory problems, Speech disorder, Swallowing disorder, Cognitive disorder, Deformities and fracture, Neurogenic bladder), severity (Mild, Moderate & Severe) has been provided into the epidemiology (Incidence and Prevalence) section of the Friedreich's Ataxia and its treatment in the 8 MM countries, covering the United States, EU5 (Germany, Spain, France, Italy, UK), Japan, and China

KOL database for PMR

In terms of pharmacologic therapies, Several drug candidates are in Phase I/II/ III stages of development by various companies. Key assets among these are, Omaveloxolone; Reata Pharm, MIN-102; Minoryx Therapeutics, SLS-005; Seelos Therapeutics, Epicatechin; Cardero Therapeutics, Horizon Pharma; Interferon ?-1b, RT001; Retrotope, Inc., debenone; Santhera Pharmaceuticals, EPI-743; Edison Pharmaceuticals, VP20629; Shire, LuAA24493; Lundbeck A/C, Among these key assets, some of the assets are being designated as orphan drugs from EMA and US FDA.

About Thelansis:

Thelansis is specialized in pharmaceutical market research and market Insight Report Company, published reports across the therapeutic area which includes both rare / ultra-rare and mainstream indication. Over the period of time, we have built a strong repository of 6,000+ Bio-pharma reports which essentially covers Epidemiology study and Market forecasting based on the KOL opinions. Competitive intelligence and track of trial results throughout the phases of development executed by a team of a mix of Scientific and Business backgrounds. As an organization, the major focus is to provide real-world data evidence and market insight to pharmaceutical companies for their decision-making.

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