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).
Another 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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