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Update on Summit and Utrophin trials

Meant to post this last week, but Summit have given an update on the trial results and next steps.

They reported some very encouraging results from the phase 1b as follows:

"After the 10 days of  dosing a reduction was observed in the enzymes creatine kinase ('CK'),  aspartate aminotransferase ('AST') and alanine aminotransferase ('ALT').  The levels of these enzymes are typically low in healthy people but, in  DMD patients, the muscle cells are weakened by the disease leading to  the accumulation of these enzymes in the blood.  When dosed with SMT  C1100, there was a reduction in CK (10/11 patients), AST (11/12  patients) and ALT (12/12 patients).  These reductions were statistically  significant compared to the pre-dose levels.  After dosing, the enzyme  levels increased towards pre-dose levels.  The lower levels of these  enzymes may indicate a reduction in muscle damage, which is consistent  with the proposed mechanism of action of our utrophin modulator"
.

However, because of low absorption they will effectively re run phase 1b (with mainly the same 12 boys) with a change in diet to check that was the issue with absorption and placebo controlled. It will however move directly from this into a phase 2 trial next summer.

So delayed but still happening and lots to 'hold' onto!

More here...Click on Half year report.

http://www.summitplc.com/investors/regulatory-news/

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Permalink Reply by Kenny Chu on September 11, 2014 at 8:17pm

So delayed my god

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Permalink Reply by David on September 12, 2014 at 5:02pm

Wow. Same reason this treatment was abandoned by Biomarin years ago!

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Permalink Reply by Steven on September 12, 2014 at 6:04pm

Any view on the CK/AST/ALT levels that were observed (even at a low absorption level). The fact it was almost all the boys, so would seem more than random? How important are CK/ASTALT as markers?