Friday, January 4, 2013

Targeted failure of the week. Post No 40-42. Urocidin, PRT-201 and dexpramipexole.



In the beginning of the new year we have plenty of targeted failurs.

The simpliest case is dexpramipexole – nice and worthless structure (see the picture).

File:Dexpramipexole.pngAnother case is very typical – biologicals PRT-201 (recombinant human pancreatic elastase) - PRT-201 missed the primary endpoint in a Phase II trial to improve vascular access in chronic kidney disease (CKD) patients undergoing surgery for arteriovenous fistula (AVF) creation. Specifically, PRT-201 given immediately after AVF creation missed the primary endpoint of prolonging the duration of unassisted primary patency vs. Placebo.

In general it is almost a rule that biologicals of non-hormonal mode of action are inefficient and this failure should be expected.

But the third case is extremely funny The intravesical formulation of mycobacterial cell wall-DNA complex (MCC), known as Urocidin(TM). This product was designed to provide an alternative to cystectomy for patients with bacillus Calmette-Guerin (BCG) refractory non-muscle invasive bladder cancer (NMIBC). Just think a minute about how in the world mycobacterial cell wall-DNA complex could be helpful in cancer treatment?! The imagination of the authors of the product was extremely impressive!

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