Full Press Release Details
Sage Therapeutics Announces Fourth Quarter and Full Year 2021 Financial Results and
Highlights Pipeline and Business Progress
Rolling NDA submission for zuranolone in MDD expected to begin in early 2022 and planned
to be completed in the second half of 2022 now supported by data from six positive clinical studies
An associated NDA submission in
PPD expected in 2023; Fast Track designation received for zuranolone in PPD
Progressing seven ongoing and planned studies across
neurology and neuropsychiatry franchises in 2022
Ended 2021 with cash balance of $1.7 billion; anticipate ending 2022 with a
cash balance of approximately $1.3 billion
Cash and cash equivalents, ongoing collaboration funding, and potential revenue,
will support operations into 2025
Conference call today at 8:00 a.m. ET
CAMBRIDGE, Mass. Feb. 24, 2022 Sage Therapeutics, Inc. (Nasdaq: SAGE), a biopharmaceutical company fearlessly leading the way to create a
world with better brain health, today reported business highlights and financial results for the fourth quarter and full year ended December 31, 2021.
2021 was a data rich year marked by important advancements in multiple disease areas across all of our brain health franchises, said Barry Greene,
chief executive officer at Sage Therapeutics. I m excited to build on this foundation, especially with the initiation of the rolling NDA submission for zuranolone in MDD planned for early this year. We believe the entirety of the
development program to date supports zuranolone s potential to address substantial unmet needs in major depressive disorder and postpartum depression and to be a differentiated treatment option for people with these brain health
Positive topline data from the WATERFALL and SHORELINE Studies announced in 2021 and multiple data presentations supporting zuranolone efficacy and
safety: Along with collaborator Biogen, Sage announced positive topline data from the WATERFALL and SHORELINE Studies in 2021. The Companies also presented multiple datasets from the LANDSCAPE and NEST clinical development programs that support
the potential efficacy and safety of zuranolone for the treatment of major depressive disorder (MDD) and postpartum depression (PPD), respectively.
Including the CORAL Study, zuranolone now has six positive clinical studies: As recently announced, results from
the Phase 3 CORAL Study in people with MDD met the trial objectives, demonstrating a rapid and statistically significant reduction in depressive symptoms in the zuranolone co-initiated with standard antidepressant arm compared to the standard
antidepressant co-initiated with
placebo arm at Day 3 and over the 2-week treatment period, achieving the primary and key secondary endpoints. In meeting its
pre-defined objectives, the CORAL Study supports the potential of zuranolone, when co-initiated with standard of care, to accelerate the benefit of depression treatment
compared to treatment with antidepressant treatments (ADTs) alone.
Planned New Drug Application (NDA) submission for zuranolone: Sage and Biogen
announced their plan to submit an NDA to the U.S. Food and Drug Association (FDA) for zuranolone in the second half of 2022, with rolling submission planned to begin in early 2022.
Topline data from PARADIGM and LUMINARY Studies with SAGE-718: SAGE-718 demonstrated improvements across multiple domains of cognition in Phase 1 and Phase 2a studies of people with cognitive impairment across several
indications, including Huntington s disease (HD), Parkinson s disease (PD) and Alzheimer s disease (AD). These findings support the Company s belief in the potential for SAGE-718 to be an
important treatment for disorders associated with cognitive dysfunction.
Topline data from the KINETIC Study with SAGE-324:
Fourth Quarter 2021 Portfolio Updates
Sage is advancing a portfolio of clinical programs featuring internally-discovered novel chemical entities with the potential to become differentiated products
designed to improve brain health by targeting the GABAA and NMDA receptor systems. Dysfunction in these systems is thought to be at the core of numerous neurological and neuropsychiatric
Depression Franchise
Sage s depression franchise features zuranolone, Sage s next-generation positive allosteric modulator (PAM) of GABAA receptors being evaluated in clinical development as a treatment for various affective disorders, and ZULRESSO (brexanolone) CIV
injection, approved by the FDA as the first treatment specifically indicated for PPD. Zuranolone has received breakthrough therapy designation from the FDA for the treatment of MDD.
Zuranolone is being evaluated as a potential rapid-acting, once-daily, two-week treatment for MDD and PPD in the
LANDSCAPE and NEST clinical development programs, respectively. Sage and Biogen plan to submit an NDA to the FDA for zuranolone in the second half of 2022, with rolling submission planned to begin in early 2022. The initial NDA submission will seek
approval of zuranolone for the treatment of MDD with an associated NDA filing for PPD anticipated in the first half of 2023 pending completion and results from the SKYLARK Study. The decision to submit the application follows discussions with the
FDA, including a pre-NDA meeting.
In February 2022, Sage and Biogen announced that the CORAL Study in people with
MDD met the trial objectives, demonstrating a rapid and statistically significant reduction in depressive symptoms in the zuranolone co-initiated with standard ADT arm compared to the standard antidepressant co-initiated with placebo arm at Day 3 and over the 2-week treatment period, achieving the primary and key secondary endpoints. This significance was demonstrated at the first
measured time point, Day 3, with zuranolone 50 mg co-initiated with an open-label standard of care ADT as assessed by change from baseline in the 17-item Hamilton Rating
Scale for Depression (HAMD-17) compared to ADT co-initiated with placebo. The CORAL Study also met its key secondary endpoint, with zuranolone co-initiated with a standard of care ADT demonstrating a statistically significant improvement in depressive symptoms compared to ADT co-initiated with placebo, over the 2-week treatment period. Zuranolone was generally well-tolerated, and no new safety signals attributable to zuranolone were identified. In meeting its pre-defined objectives,
the CORAL Study supports the potential of zuranolone, when co-initiated with standard of care, to accelerate the benefit of depression treatment compared to treatment with ADTs alone.
Additionally, Sage today announced that the FDA granted Fast Track Designation to zuranolone for development in PPD. This designation is granted to drug
candidates that treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs.
The Company expects to achieve
the following milestones across its depression franchise in 2022, with plans to share additional analyses throughout the year:
Neuropsychiatry Franchise
Sage s neuropsychiatry
franchise features SAGE-718, the Company s first-in-class NMDA receptor PAM and lead neuropsychiatric drug candidate, in
development as a potential oral therapy for cognitive disorders associated with NMDA receptor dysfunction, potentially including HD, PD and AD. SAGE-718 received Fast Track Designation from the FDA for
development of SAGE-718 as a potential treatment for HD.
SAGE-718 is currently being studied in the ongoing Phase 2 DIMENSION
Study, a double-blind placebo-controlled study in people with early to moderate HD cognitive impairment that is designed to evaluate the efficacy of once-daily dosed SAGE-718 over three months.
The Company expects to initiate the following studies in the neuropsychiatry franchise in 2022:
Sage also plans to share additional analyses from studies
completed with SAGE-718 to date throughout 2022.
Additionally, the Company today announced its decision to
discontinue development of SAGE-904 following results from the Phase 1 clinical program that showed it did not achieve the company s target product profile for further development.
Sage s neurology franchise
features SAGE-324 and SAGE-689. SAGE-324, a next-generation PAM of
GABAA receptors and Sage s lead neurology program, is in development as a potential oral therapy for neurological conditions, such as ET, epilepsy and PD. SAGE-689 is an intramuscular GABAA receptor PAM in development as a potential therapy for disorders associated with acute GABA hypofunction.
Sage and its collaborator, Biogen, are currently enrolling people in the Phase 2b KINETIC 2 placebo-controlled study of
SAGE-324 in ET following positive results from the KINETIC Study presented in 2021. The KINETIC 2 Study is a Phase 2b dose-ranging study with the primary goal of defining the dose and frequency with a good
tolerability profile and a dosing schedule to maintain plasma concentrations of SAGE-324 that translate into sustained tremor symptom control in treating ET. KINETIC 2 will utilize evening dosing.
SAGE-689 remains in Phase 1 development.
The Company expects to achieve the following milestones across its neurology franchise in 2022:
Sage also plans to share additional analyses from studies completed with SAGE-324 to date
progressing its early development programs with IND-enabling work underway for SAGE-319 and SAGE-421.
ANTICIPATED 2022 MILESTONES
FINANCIAL RESULTS FOR THE FOURTH QUARTER AND FULL YEAR 2021
Conference Call Information
Sage will host a conference call and webcast today, Thursday, February 24, at 8:00 a.m. ET to discuss its fourth quarter and full
year 2021 financial results and recent corporate updates. The live webcast can be accessed on the investor page of Sage s website at investor.sagerx.com. A replay of the webcast will be available on Sage s website approximately two
hours after the completion of the event and will be archived for up to 30 days.
About Sage Therapeutics
Sage Therapeutics is a biopharmaceutical company fearlessly leading the way to create a world with better brain health. Our mission is to pioneer solutions to
deliver life-changing brain health medicines, so every person can thrive. For more information, please visit. www.sagerx.com.
Various statements in this release concern Sage s future expectations, plans and prospects, including without limitation our
statements regarding: plans for an NDA filing and associated submission for zuranolone in MDD and PPD, and the potential timing of such submissions; our belief in the adequacy of the data we plan to include in the zuranolone NDA; the potential for
FDA acceptance of an NDA for zuranolone; our belief in the regulatory filing pathways for zuranolone; the potential profile and benefit of zuranolone in MDD and PPD; the potential for regulatory approval and commencement of commercialization of
zuranolone; other planned next steps for the program; anticipated timelines for reporting clinical trial results, commencement of trials, and initiation of new activities; our plans for advancement of our pipeline; our belief in the potential
profile and benefit of our product candidates; potential indications for our product candidates; the potential for success of our programs, and the opportunity to help patients in various indications; the mission and goals for our business; and our
expectations with respect to 2022 year-end cash, no receipt of milestones from collaborations in 2022 and funding of future operations. These statements constitute forward-looking statements as that term is
defined in the Private Securities Litigation Reform Act of 1995. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond our
control, which could cause actual results to differ materially from those contemplated in these forward-looking statements, including the risks that: we may experience delays or unexpected hurdles in our efforts to submit an NDA for zuranolone and
we may not be able to submit the NDA on the timelines we expect or at all; the FDA may find inadequacies and deficiencies in our NDA for zuranolone, including in the data we submit, despite prior discussions, and may decide not to accept the NDA for
filing; even if the FDA accepts the NDA for filing, the FDA may find that the data
included in the NDA are not sufficient for approval and may not approve the NDA; the FDA may decide that the design, conduct or results of our completed and ongoing clinical trials for
zuranolone, even if positive, are not sufficient for approval in MDD or PPD and may require additional trials or data which may significantly delay and put at risk our efforts to obtain approval and may not be successful; the FDA may not meet
expected review timelines for our NDA; other decisions or actions of the FDA or other regulatory agencies may affect our efforts with respect to zuranolone and our plans, progress or results; we may experience negative results in the ongoing SKYLARK
Study in PPD that negatively affect our ability to file an NDA for approval of zuranolone or results of ongoing or future studies may impact our ability to obtain approval of zuranolone or impair the potential profile of zuranolone; success in
earlier clinical trials of any of our product candidates may not be repeated or observed in ongoing or future studies, and ongoing and future clinical trials may not meet their primary or key secondary endpoints which may substantially impair
development; unexpected concerns may arise from additional data, analysis or results from any of our completed studies; we may encounter adverse events at any stage of development that negatively impact further development or that require additional
nonclinical and clinical work which may not yield positive results; we may encounter delays in initiation, conduct or completion of our ongoing and planned clinical trials, including as a result of slower than expected site initiation or enrollment,