Two months after getting hammered on the failure of its lead late-stage study for a rare type of seizures, Sage Therapeutics is claiming a victory in a pair of Phase III studies for the same drug in postpartum depression.
Sage reported this morning that both of its late-stage studies for brexanolone (SAGE-547) for major postpartum depression successfully edged out a placebo — but failed to register the big improvement over a sugar pill that was seen in Phase II. And the biotech $SAGE says that it will use the data to back an FDA submission for their drug — which requires a 60-hour infusion — next year.
Sage’s shares spiked in pre-market trading on Thursday and then waffled for a but. By mid-day, though, the stock was up 51% as investors bought into the upbeat tone.
The biotech recruited 226 patients for these two studies for an ailment that afflicts huge numbers of women each year. Typically a disease like PPD would involve large numbers of patients in search of two positive outcomes, but Sage believes it’s right on track to break new ground and score a major OK.
The main goal of both studies was a significant reduction in depression scores 60 hours after treatment. And on that level the drug scored a 17.7-point mean reduction for the high dose and a 19.9-point improvement for the low dose in the first study for severe PPD compared to 14 points in the placebo arm. In study two there was a 14.2-point vs 12-point difference in the moderate PPD group.
In Phase II, researchers reported a 12.2-point spread between the drug and the placebo, leaving Sage defending a significantly reduced margin of improvement.
Researchers also noted that the drug effect lasted through 30 days in the first study, but did not mark a statistically significant impact after a month in the second study for moderate PPD, which could raise a red flag on durability.
That Phase II comparison may be a bit of a letdown, concedes Leerink’s Paul Matteis, but a win in Phase III is a major plus for Sage, which he believes is headed for an approval. Getting an oral version, he adds, would be a tremendous boost.
Nonetheless, the positive phase III PPD results are a technology validating event for SAGE who is seeking to recapitulate the mechanism of brenaxolone in an oral formulation (SAGE-217) across an array of CNS disorder. In the backdrop of a planned NDA filing for brenaxolone, oral data in major depressive disorder represent the next major event in 4Q.
There was at least one serious adverse event associated with the drug, which was not explained in the company’s statement. But Sage says the safety profile overall was comparable to the placebo arm. More data will be released at an scientific conference.
Sage has mustered fierce support as well as plenty of critics for its R&D strategy, using small studies to highlight the potential of a drug. And in this case, PPD represents the kind of major market opportunity likely to require a significant amount of data to win over regulators.
Last September the biotech reported that the drug did no better than a sugar pill in treating super-refractory status epilepticus.
Now we’ll see how the FDA feels about all of this.
Sage CEO Jeff Jonas touted the results as a game changer:
We believe the data represent an unprecedented opportunity in the development of treatments for PPD, and may serve as the catalyst for a paradigm shift in how the disease is approached and, if approved, may change how PPD is treated.
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