Profile: Catabasis Pharmaceuticals Inc (CATB.A)
13 Jun 2019
Catabasis Pharmaceuticals, Inc., incorporated on June 26, 2008, is a clinical-stage biopharmaceutical company. The Company is focused on the discovery, development and commercialization of therapeutics based on its Safely Metabolized And Rationally Targeted (SMART), a linker drug discovery platform. The SMART linker drug discovery platform enables to engineer product candidates that can modulate multiple targets in a disease. The Company's primary focus is on treatments for rare diseases. The Company is also developing other product candidates for the treatment of lipid disorders. The Company's CAT-1004 is a SMART linker conjugate of salicylate, a non-steroidal anti-inflammatory drug, and the omega-3 fatty acid docosahexaenoic acid (DHA), a naturally occurring unsaturated fatty acid with anti-inflammatory properties. The Company's CAT-2000 series product candidates inhibit the Sterol Regulatory Element Binding Protein (SREBP) pathway. The Company's CAT-4001 is a SMART linker conjugate of monomethyl fumarate and DHA.
The Company's CAT-1004 is the disease-modifying oral therapy for the treatment of Duchenne muscular dystrophy (DMD) that both inhibits muscle degeneration and promotes muscle regeneration, regardless of the underlying mutation. CAT-1004 is an orally administered SMART linker conjugate of salicylate and DHA, which the Company designed to enhance the activity of salicylate and DHA to inhibit the Nuclear Factor- kappaB (NF-kB) pathway at multiple points. The CAT-1004 conjugate is inactive outside the cell, and, once inside the cell, CAT-1004 is cleaved releasing DHA and salicylate simultaneously inside the same cell. The Company is conducting the MoveDMD trial, a Phase I/II clinical trial of CAT-1004 for the treatment of DMD, in over two parts.
The Company's other clinical-stage program is CAT-2000 series. The Company has applied its SMART linker drug discovery platform to engineer these product candidates as SMART linker conjugates of eicosapentaenoic acid (EPA) and nicotinic acid in order to inhibit the SREBP pathway. CAT-2003, the first generation product candidate in the CAT-2000 series, is an orally administered molecule that inhibits the SREBP pathway predominately in the intestine. CAT-2054 is a second generation product candidate in the CAT-2000 series, which is an orally administered molecule designed to inhibit the SREBP pathway in the liver. The Company is developing CAT-2054 for the treatment of serious lipid disorders, such as hypercholesterolemia. The Company is conducting preclinical studies in collaboration with academic institutions and has observed positive data in preclinical models that support the therapeutic potential of the CAT-2000 series in Nonalcoholic Steatohepatitis (NASH).
The Company's CAT-4001 is a SMART linker conjugate of monomethyl fumarate and DHA that it designed to combine the activities of monomethyl fumarate and DHA on the Nrf2 and NF-kB pathways. CAT-4001 is a small molecule designed to activate the nuclear factor erythroid 2 [NF-E2]-related factor 2 (Nrf2) pathway and inhibit the NF-kB pathway. The Company is developing CAT-4001 initially for the treatment of severe, rare neurodegenerative diseases, such as Friedreich's ataxia and Amyotrophic Lateral Sclerosis (ALS), two diseases of the central nervous system in which the Nrf2 and NF-kB pathways have been implicated. Nrf2 is a gene transcription factor, a protein that works inside of cells to control the expression of genes that control the body's response to cellular stress and oxidative damage. The Nrf2 and NF-kB pathways has been implicated in Friedreich's ataxia and ALS.
Catabasis Pharmaceuticals Inc
1 Kendall Sq Ste B14202
CAMBRIDGE MA 02139-1573