SAGE Therapeutics Announces First Quarter 2017 Financial Results And Provides Pipeline Update

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sage Therapeutics, Inc. (NASDAQ:SAGE), a clinical-stage biopharmaceutical company developing novel medicines to treat life-altering central nervous system (CNS) disorders, today reported business highlights and financial results for the first quarter ended March 31, 2017.

“We continue to make good progress in building Sage into a leading CNS company with the potential to deliver differentiated medicines for a variety of central nervous system disorders. We are focused on closing the innovation gap in areas of brain disorders where more breakthroughs for patients are needed,” said Jeff Jonas, M.D., Chief Executive Officer of Sage. “We are now nearing completion of enrollment in our lead Phase 3 program in super-refractory status epilepticus (SRSE), and remain focused on completing Phase 3 clinical development of brexanolone in both SRSE and postpartum depression (PPD) in 2017. Further, we continue to advance our growing pipeline of novel product candidates, including our lead oral compound, SAGE-217, in four clinical programs in both mood and movement disorders.”

Brexanolone in SRSE - Phase 3 STATUS Trial Update

Sage is nearing completion of enrollment in the Phase 3 STATUS Trial, the first ever global, randomized, double-blind, placebo-controlled trial in SRSE. The Company expects to report top-line results from the STATUS Trial in the third quarter of 2017 after enrollment of an anticipated 126 evaluable patients and completion of all study follow-up periods and data analysis. Sage expects top-line results to include the primary endpoint, safety and tolerability, and select secondary endpoints, including open-label retreatment arm response and the Clinical Global Impression scale.

Top-Line Results from Part A of SAGE-217 Phase 2 in Parkinson’s Disease

Sage today announced top-line results from the Part A open-label portion of a Phase 2 clinical trial of SAGE-217 in Parkinson's disease:

• SAGE-217 was studied in an exploratory open-label trial designed to understand the safety, tolerability, pharmacokinetics and activity of SAGE-217 in a cohort of 12 Parkinson’s disease patients in order to determine whether there is a suitable activity signal to justify further development, and if so, to help develop the methodology needed to expedite progress into further Phase 2 testing.
• Twelve Parkinson’s disease patients with moderate disease severity (Hoehn and Yahr stage 2 or 3) who were on stable doses of the anti-Parkinsonian agent levodopa/carbidopa prior to the study were evaluated. As an exploratory study, the Part A phase enrolled an all-comer population that was not enriched based on tremor severity or for any other particular Parkinson’s disease symptom complex. Patients were withdrawn from maintenance therapy and administered their baseline medicine (levodopa/carbidopa morning dose only) for three days, followed by a single morning dose of SAGE-217 administered as a monotherapy for the subsequent four days.
• For the overall population in the trial, levodopa/carbidopa activity was primarily focused on the motor symptoms of bradykinesia and rigidity, while SAGE-217 activity as a monotherapy was primarily focused on tremor symptoms.
• In the five subjects with overt tremor (tremor score over five at baseline), an approximate 20-30% improvement in tremor symptoms was observed on the four days of SAGE-217 open-label treatment, as assessed by change in the Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Part III tremor score. This improvement in tremor score during the SAGE-217 dosing phase was longer-lasting than the effect on tremor observed in these subjects during the levodopa/carbidopa-only phase.
• Administration of SAGE-217 during the day was found to be generally well-tolerated with no serious adverse events or discontinuations reported. Similar to findings in the Phase 1 clinical program, the most common adverse events were sedation and somnolence (occurring 2 to 4 hours post dose). While dosing was initiated at the 30 mg per day maximum tolerated dose established in the Phase 1 program, the majority of patients were down-titrated to 10 to 20 mg of SAGE-217 per day.
• Based on the signal of activity in reducing Parkinsonian tremor in these patients, Sage plans to proceed to an open-label Part B study evaluating SAGE-217 as an adjunctive treatment to anti-Parkinsonian agents in tremor-predominant patients, and intends to further evaluate non-motor symptoms of Parkinson’s disease, including depression, anxiety, cognition and sleep.

Pipeline Update

Sage is advancing a portfolio of novel central nervous system (CNS) product candidates targeting the GABA and NMDA receptor systems. Dysfunction in these systems is known to be at the core of numerous psychiatric and neurological disorders. Sage is pursuing a data-driven approach to CNS drug development by employing efficient human proof-of-concept studies both to uncover activity signals and to help understand future trial methodology, before investing in larger clinical programs.

• Brexanolone (SAGE-547): Sage is currently developing brexanolone in separate Phase 3 clinical programs as an acute interventional treatment for super-refractory status epilepticus (SRSE) and postpartum depression (PPD). Brexanolone is Sage’s proprietary intravenous (IV) formulation of allopregnanolone, a naturally occurring neuroactive steroid that acts as a synaptic and extrasynaptic modulator of the GABA_A receptor.