Biotech Values

[DewDiligence]

GBT +18%/AH on phase-3 trial design in SCD (but no SPA):

http://finance.yahoo.com/news/global-blood-therapeutics-announces-pivotal-200500928.html

The HOPE Study is a randomized, double-blind, placebo-controlled, multinational, Phase 3 trial, which will enroll up to 400 patients age 12 and older with SCD who have had at least one episode of vaso-occlusive crisis (VOC) in the previous year.

The HOPE Study will be conducted in two-parts:

• Part A - Compare two dose levels of GBT440 – 900 mg and 1500 mg versus placebo. Part A will include up to 150 patients.

• Part B - To include 250 patients randomized to placebo or a dose of GBT440 based on Part A.

…The primary efficacy endpoint of the HOPE Study will be the proportion of patients who achieve a >1 g/dL increase in hemoglobin at 24 weeks of treatment vs baseline. Our discussions with the FDA have focused on a pathway to full approval based on the HOPE Study, by meeting the primary and at least one key secondary endpoint.

Key secondary efficacy endpoints will include the effect of GBT440 on SCD symptom exacerbation, which will be measured by our PRO instrument, in addition to overall SCD symptoms as compared to placebo.

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