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Arimoclomol maleate is in a phase III clinical trials by Orphazyme for the treatment of Niemann-Pick disease type C (NP-C). It is also in phase II clinical studies for the treatment of amyotrophic lateral sclerosis (ALS).
Arimoclomol(INN; originally codenamedBRX-345, which is a citrate salt formulation ofBRX-220) is an experimental drug developed byCytRx Corporation, a biopharmaceutical company based inLos Angeles, California. In 2011 the worldwide rights to arimoclomol were bought by Danish biotech company Orphazyme ApS.[1]TheEuropean Medicines Agency(EMA) andU.S. Food & Drug Administration(FDA) granted orphan drug designation to arimoclomol as a potential treatment forNiemann-Picktype C in 2014 and 2015 respectively.[2][3]
Fig. 1Structures of (±)-bimoclomol (1) and (R)-(+)-arimoclomol…
Indication:Ovarian cancer;Breast cancer;Non small cell lung cancer (NSCLC)лурбинектединلوربينيكتيدين芦比替定(1R,1’R,2’R,3’R,11’S,12’S,14’R)-5′,12′-Dihydroxy-6,6′-dimethoxy-7′,21′,30′-trimethyl-27′-oxo-2,3,4,9-tetrahydrospiro[β-carboline-1,26′-[17,19,28]trioxa[24]thia[13,30]diazaheptacyclo[12.9.6.13,11…2,13.04,9.015,23.016,20]triaconta[4,6,8,15,20,22]hexaen]-22′-yl acetate[ACD/IUPAC Name]2CN60TN6ZS497871-47-3[RN]9397
Lurbinectedin is in phase III clinical development for the treatment of platinum refractory/resistant ovarian cancer.
Phase II clinical trials are also ongoing for several oncology indications: non-small cell lung cancer, breast cancer, small cell lung cancer, head and neck carcinoma, neuroendocrine tumors, biliary tract carcinoma, endometrial carcinoma, germ cell tumors and Ewing’s family of tumors.
Lurbinectedin, sold under the brand nameZepzelca, is a medication for the treatment of adults with metastatic small cell lung cancer (SCLC) with disease progression on or after…
Ark Biosciences , under license from Roche , is developing AK-3280, an antifibrotic agent, for the potential oral treatment of IPF. In July 2018, Ark intended to further clinical development of the drug, for IPF. In June 2019, a phase I trial was planned in Sweden.
Originator Genentech
Mechanism of Action Undefined mechanism
Phase I Interstitial lung diseases
19 Jun 2019Ark Biosciences plans a phase I trial for Idiopathic pulmonary fibrosis (In volunteers) in Sweden (PO, Tablet), in August 2019 , (NCT03990688)
28 Sep 2018GDC 3280 is still in phase I trials for Interstitial lung diseases (Genentech pipeline, September 2018)
28 Jun 2018No recent reports of development identified for phase-I development in Fibrosis(In…
SRT-1720, also known as CAY10559 and is a drug developed by Sirtris Pharmaceuticals intended as a small-molecule activator of the sirtuin subtype SIRT1. It has similar activity in the body to the known SIRT1 activator resveratrol, but is 1000x more potent. In animal studies it was found to improve insulin sensitivity and lower plasma glucose levels in fat, muscle and liver tissue, and increased mitochondrial and metabolic function. A study of SRT1720 conducted by the National Institute on Aging found that the drug may extend the lifespan…
Percent Composition: C 45.38%, H 4.23%, N 23.52%, O 26.87%
Literature References: Prepn: DE352980 (1922 to E. Merck); Frdl.14, 1320; S. M. Ride et al.,Pharmazie32, 672 (1977). Prepn of salts: J. Baisse, Bull. Soc. Chim. Fr.1949, 769; M. Milletti, F. Virgili, Chimica6, 394 (1951), C.A.46, 8615h (1952). GC determn in urine: J. Zuidema, H. Hilbers, J. Chromatogr.182, 445 (1980). HPLC determn in serum and pharmacokinetics: S. Sved et al.,Biopharm. Drug Dispos.2, 177 (1981).