Lipocine's low-T drug hits the mark in Phase III. Now it has to sway the FDA

Lipocine ($LPCN) is touting positive late-stage results for its testosterone replacement pill, gearing up for intense FDA scrutiny amid a sea change in how the regulator looks at so-called low-T treatments.

The drug, LPCN 1021, met its primary goal in a study on 315 patients with low testosterone, successfully getting serum hormone levels back to normal. According to Lipocine, the FDA's benchmark for measuring testosterone treatments has two demands: 75% of patients must get their T levels into the normal range, and the lower bound of a study's 95% confidence interval must exceed 65%. 1021 nailed both goals, the company said, notching an 88% rate of normalcy with a lower bound of 82%.

The biotech's shares shot up as much as 77% on the news Wednesday morning, with investors confident that 1021's FDA submission, expected in the second half of 2015, will be met with success.

What Lipocine has in positive data, however, it may lack in timing. The FDA has recently zeroed in on low-T treatments, taking a hard look at controversial evidence that testosterone therapy can lead to elevated risks of heart attack and other events. Last week, a panel of agency advisers voted 14-1 in favor of restricting such drugs to use only in specific medical conditions; currently, they're widely prescribed to men reporting low testosterone levels.

And, perhaps more relevant to Lipocine, a separate FDA panel has sided against approval for Clarus Therapeutics' oral low-T treatment, pointing out that while the drug didn't seem any more dangerous than other testosterone replacements, the fact that it's a pill may make patients more likely to take more of it, putting them at greater risk of the cardiovascular events tied to the whole class.

Lipocine's latest results are top-line only, and the safety component of the trial remains ongoing, the company said. So far, just 3% of participants have reported serious adverse events, none of which were drug-related, according to Lipocine. Otherwise, the 1021 has led to some increases in red blood cells and prostate-specific antigen in line with other testosterone replacements, the company said.

"We believe that LPCN 1021 represents a differentiated (testosterone-replacement therapy) for treating hypogonadism in men with the potential to both improve patient compliance and overcome inadvertent testosterone transference risk," Lipocine CEO Mahesh Patel said in a statement. "We look forward to reporting additional safety results from this ongoing study."

- read the results