Regulus hopes for quick turnaround on FDA hold on hep C drug

Small cap Regulus Therapeutics ($RGLS) saw its shares nosedive last month when the FDA put a clinical trial hold on its leading hep C candidate RG-101--but the company said this morning it hopes to get all of its ducks in a row for the FDA to lift the hold by the start of Q4.

The U.S. regulator put the trial of the drug on hold over safety concerns, coming after a second case of jaundice emerged in a Phase I trial after the patient received a single dose of RG-101. The drug is being developed with Regulus’ partner GlaxoSmithKline ($GSK).

It was given an oral notice from the FDA exactly a month ago, but today said it had been given a detailed sheet of tasks to do before the regulator can lift the hold.

These include detailed safety data analysis from preclinical and clinical studies; exploration of potential mechanisms of hepatoxicity in nonclinical models; review and input from independent hepatotoxicity experts; additional PK data from the U.S. Phase I study; and a risk/benefit assessment for the proposed therapeutic regimens containing RG-101. 

Although a long list, this is essentially a data-gathering exercise, and does not include the need to conduct any further trials. Regulus was upbeat about the task ahead, saying it “anticipates submitting the necessary information by early Q4 2016.”

A month after these new data have been submitted, the FDA should let the biotech know whether it will lift the hold, or requires more info. The drug is currently undergoing Phase I/II trials.

"We are working expeditiously to resolve the issues outlined in the letter and anticipate a decision from the FDA in the fourth quarter," said Paul Grint, president and CEO of Regulus.

"Timelines of on-going studies have not been impacted by the clinical hold, and we remain on track to deliver follow-up results from ongoing RG-101 studies at upcoming relevant scientific meetings later this year."

RG-101 works as a GalNAc-conjugated anti-miR targeting microRNA-122 for the treatment of chronic hepatitis C. It is however looking to get into a crowded space, with Gilead’s ($GILD) megablockbusters Sovaldi and Harvoni dominating the market for the past three years with their impressive cure rates, with new therapies from Bristol-Myers Squibb ($BMY), Merck ($MRK) and AbbVie ($ABBV) all vying for space.

Regulus is also working on RG-012, an anti-miR targeting microRNA-21 for the treatment of Alport syndrome--a life-threatening kidney disease driven by genetic mutations with no approved therapy.

The California biotech finished the day up by more than 16%, but traded down after hours by nearly 2% after the company posted the update at 16:10 ET. The biotech as of yesterday had a market cap of just $220 million. 

- check out the release

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