Patients Push FDA For Approval of Heavily Shorted Biotech's Drug

(Bloomberg) -- When Lindsey Sutton was five weeks old, a heel prick test revealed blood that was Pepto Bismol pink.

It was the first sign that she had inherited a rare disease known as familial chylomicronemia syndrome, or FCS. Because the disorder leads to the buildup of fat in the blood, what followed for the now-28 year-old Sutton was a lifetime on an extreme low-fat diet limited to roughly a tablespoon of olive oil a day and a string of hospitalizations from one of the disease’s most debilitating consequences, pancreatitis.

Since starting on Akcea Therapeutics Inc.’s experimental therapy Waylivra three years ago, Sutton has not been hospitalized or had pancreatitis. Other symptoms of the disease -- pain and fatigue -- dramatically improved and she felt normal for the first time in her life. Despite the improvements, Waylivra failed to secure U.S. Food and Drug Administration approval in August, pitting patients like Sutton against the agency.

A side effect of the drug, a drop in platelets, is part of the reason the FDA decided not to approve it. The agency wants more data on how platelets can be managed, Akcea’s Chief Executive Officer Paula Soteropoulos told Bloomberg. The FDA also had questions on a new proposed dosing regimen that had not been in use at the time of the study. According to Sutton, her platelets have always fluctuated and were not enough of an issue to dissuade her from taking Waylivra.

Soteropoulos hopes results from an open label extension study for Waylivra with another six months of data, as well as updated results from a “compassionate use” program, where patients take the drug outside of a clinical trial because their disease is so serious, will be enough to satisfy regulators when the company meets with them.

The question is when. “The sooner the better,” Soteropoulos said. The company is hoping for a meeting before the year ends.

For its part, the FDA does not comment on pending drug applications but a spokeswoman said in an emailed statement that “engaging with patient advocates is of the utmost importance” and “the FDA carefully considers patient input as it fulfills its role in the drug development process.”

The FDA’s hesitation, as well as competitive concerns over another therapy in the company’s pipeline, have led Akcea to become one of the most shorted biotech stocks this year with short interest representing 70 percent of float, according to data compiled by Bloomberg.

Shares of Akcea, which is majority-owned by Ionis Pharmaceuticals Inc, rose 0.4 percent at 11:15 a.m. in New York. An agency decision on approval for Akcea’s other lead program, Tegsedi, is expected by Friday.

Sutton is an active advocate for disease sufferers and heads up the FCS Foundation along with Melissa Goetz, whose daughter has the disease. A petition to the FDA has already amassed over 12,000 signatures, more than double the estimated 5,000 people diagnosed globally.

This has led some investors to draw parallels to another drug that eventually won accelerated approval after patients and their families faced off with the FDA following a contentious advisory committee meeting: Sarepta Therapeutics Inc’s Exondys 51.

“When we started this trial, we didn’t know as much about the serious consequences of this disease outside of pancreatitis, so we didn’t measure other factors of quality of life,” Soteropoulos said. “The only way that can be conveyed is through the patient voice.”

While FCS patients and advocates, like the Duchenne Muscular Dystrophy community before them, are vocalizing their distress at the FDA’s decision, that may be where the similarity ends.

Akcea’s drug has results that show it works and while patients’ voices may help, at the end of the day, “we have to answer to the questions on the data,” Soteropoulos said. Patients, meanwhile, will have to wait to try the treatment until after the FDA meets with Akcea to discuss a path forward.

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