Thinly traded Principia Biopharma (PRNB +2.8%) is up out of the gate this morning in response to positive preliminary data from a Phase 1/2 clinical trial evaluating lead candidate PRN1008 for the treatment of immune thrombocytopenia purpura (ITP), a condition characterized by easy bruising and a bleeding tendency due to low blood platelets.
26 participants have been enrolled to date. After a
median duration of treatment of almost 13 weeks, 39% (n=10/26) achieved
the primary efficacy endpoint of at least two consecutive platelet
counts of at least 50K/µL at least five days apart and platelet count
increases of at least 20K/µL from baseline without needing rescue
medication. Preliminary results across all doses in patients who
completed at least 12 weeks of treatment showed a response rate greater
than 50% on both both primary endpoints (the other one is safety).
Updated data will be presented at ASH in early December.
PRN1008 is an optimized Bruton’s tyrosine kinase
(BTK) inhibitor that the company says enables rapid reversibility of
effects on the immune system which makes it suitable for chronic
therapy. It is also being developed to treat pemphigus.
https://seekingalpha.com/news/3505761-principia-bio-3-percent-positive-prn1008-data
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