Four days after Gemphire saw its shares $GEMP crushed by news of an FDA demand for additional safety data on its only drug before advancing it into Phase III, the biotech is back with more bad, terrible, absolutely no good news.
A Phase IIa study for gemcabene in pediatric patients with non-alcoholic fatty liver disease, or NAFLD, has been scrapped after investigators determined that the first three patients got worse after being dosed. And another mid-stage study is under close review to see if the biotech needs to shutter it as well.
Gemphire’s shares, which lost half their value earlier in the week, dropped another 44% on Friday. For the week, shares are down a catastrophic 76%.
Investigators hurried to close the NAFLD study after spotting a spike in serum alanine transaminase (ALT) and “an increase in liver fat content, as measured by MRI-PDF.”
Their conditions, in short, were getting worse rather than better, “which the investigator believed was likely due to the drug.” And now researchers are also going to stop dosing more patients in a mid-stage study of familial partial lipodystrophy so they can determine if the same thing is occurring in other groups.
Gemphire does what it always does about safety questions. The company cited stats showing that none of the 1,200 patients dosed with the old Pfizer drug have experienced a serious adverse event.
The biotech got the drug a few years ago from Pfizer, which had given up on it. Gemcabene was the subject of a partial hold back in 2014, preventing its use in studies lasting longer than 6 months due to potential toxicity issues — and Gemphire had been looking to get that lifted by presenting data from 2-year rodent safety studies.
Instead, earlier this week the company noted that the FDA was concerned by evidence of tumors in the rodents, which triggered a fresh demand for more data and a lengthy one-year delay for Phase III. The NASH and NAFLD work was their fallback effort during the revamp for the lead program.
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