Drug Development

Regulus ordered to remain in limbo as FDA refuses to lift hold, demanding more data on lead drug

Regulus is still in the FDA’s doghouse. Regulators have stiff armed the biotech, refusing to lift a clinical hold on the hep C drug RG-101 and blowing far past the company’s promise to get everything wrapped and hopefully back on track in Q4 of last year.

The agency put the drug on hold 6 months ago after a second case of jaundice developed among the patients taking the therapy. And now it will have to wait until Q4 of this year to get more data that the FDA wants to see from ongoing studies – a hefty delay for a questionable lead therapy.

Shares of Regulus dropped 26% in after-market trading Friday as the stock plumbs new depths. And then on Monday the stock ended the day down 42%.

Analysts were already wondering how Regulus $RGLS can still make a mark in hep C after Gilead, AbbVie and Merck have all plowed ahead with a new wave of therapies that can effectively cure the disease. The only challenges left now are coming up with shorter, less expensive treatment regimens.

RG-101 targets microRNA-122, which the virus needs to replicate.

Now, after assessing the biotech’s response to its queries, regulators want to see data from clinical and preclinical studies, which aren’t likely to arrive until the last three months of 2017. And the FDA also wants “additional expert review of liver safety data in light of the proposed mechanism of hyperbilirubinemia.”

“While we are disappointed that the clinical hold was not lifted at this time, we plan to continue to work with the FDA to address their additional requests as we seek the removal of the clinical hold,” said Dr. Timothy Wright, chief R&D Officer of Regulus. Company execs promised just weeks after the hold was announced that it would be able to answer all the agency’s questions with an eye to getting restarted before the end of the year.

A hold like this, with no clear end in sight, is a biotech’s worst nightmare. The company announced just a week before the hold – which blitzed its stock – that it had lined up $30 million in debt.


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