Replies to post #205442 on Biotech Values

[DewDiligence]

RARX’s own PR re IPO today:

http://finance.yahoo.com/news/ra-pharmaceuticals-announces-pricing-initial-110100001.html

[mcbio]

RARX -

…We are developing our lead product candidate, RA101495,…a subcutaneous…injection,…for the treatment of paroxysmal nocturnal hemoglobinuria, or PNH. PNH is a rare, chronic, life-threatening, blood disorder where red blood cells are mistakenly attacked and destroyed by the complement system. We expect to initiate our Phase 2 clinical program for RA101495 in PNH patients in the first quarter of 2017 and release data in the second half of 2017.

As with PTGX, not clear to me if this is more just a convenience story (subQ vs. IV) or if RARX also thinks their lead drug will have efficacy/safety advantages over Soliris. Per the web site, they do think more frequent subQ self-administered dosing may provide more improved disease control: http://rapharma.com/pipeline/ra101495/ .

I guess the other thing to think about here is pricing of Soliris and fact that it is currently the only approved option for PNH. Given this, presumably there is more of an opportunity for a second drug, even if there is not a meaningful advance on efficacy/safety front.