Biotech Values

[DewDiligence]

Musings on RVNC interim phase-2 data in CD (refer to slide set in #msg-127195330):

Although these data come from a small, open-label, proof-of-concept trial, I think it’s fair to say that RT002 clearly works in cervical dystonia, and it appears to have a somewhat longer duration of action than Botox. All told, today’s data are excellent, but not quite a home run because patients in the second and third cohorts have not yet been observed for the full 24 weeks.

The lowest-dose cohort (n=12; 174U average dose [range: 100-200U]) produced stellar efficacy and duration of action (see slides #8 and #9), so there is probably no good reason for RVNC to go to a higher dose in the next CD trial. The second cohort (n=12; 229U average dose [range: 200-300U]) was part of the initial study design, but the third cohort (n=11; average dose 323U [range: 300-450U]) was added on the fly.

Why was the third cohort added? The only answer that makes sense, IMO, is that the FDA wanted a higher-dose cohort to establish a robust safety margin for the lower doses. As far as I can tell, there is no other rationale that justifies delaying the completion of the PoC trial—and hence the start of the next trial in CD—by about six months.

Fortunately, the safety data presented today are fine for all three cohorts—with the proviso that patients in the second cohort have been followed for 16 weeks of the 24-week total and patients in the third cohort have been followed for 4 weeks of the 24-week total.

In short, I have no serious concerns about RT002’s safety and efficacy in cervical dystonia, and RT002 should be able to more than hold its own against Botox and any other botulinum toxin. I fully expect CD to be the first non-cosmetic indication in which RT002 is approved for marketing.