Axsome to Expedite Development for Major Depressive Disorder Treatment

Previously completed active-controlled ASCEND trial in MDD now considered as pivotal; sufficient with ongoing STRIDE-1 Phase 3 trial for NDA in MDD

Target randomization for STRIDE-1 Phase 3 trial reached; screening to continue to build required NDA safety database

Initiation of placebo-controlled Phase 3 trial in MDD anticipated in 2Q 2019; provides additional NDA path with ASCEND trial and builds required NDA safety database

Topline results of both STRIDE-1 Phase 3 trial in TRD and planned placebo-controlled Phase 3 trial in MDD expected in 2H 2019

AXS-05 has potential to be first oral NMDA receptor antagonist with multimodal activity for the treatment of depression

Company to host conference call today at 8:00 AM Eastern

Axsome Therapeutics, Inc.(NASDAQ: AXSM), a clinical-stage biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders, today announced that the development status and plan for AXS-05 in the treatment of major depressive disorder (MDD) and treatment resistant depression (TRD) have been expedited following a Breakthrough Therapy meeting with the U.S. Food and Drug Administration (FDA). AXS-05 is a novel, oral, investigational NMDA receptor antagonist with multimodal activity.

As part of the expedited development program, the Company’s previously completed ASCEND trial in MDD is now considered sufficient with the ongoing STRIDE-1 Phase 3 trial in TRD, if positive, to support the filing of an NDA (New Drug Application) for approval of AXS-05 for the treatment of MDD. Alternatively, Axsome may file an NDA for AXS-05 for the treatment of MDD with the completed ASCEND trial and a placebo-controlled Phase 3 trial of AXS-05 in MDD. Axsome intends to initiate this placebo-controlled Phase 3 MDD trial in the second quarter of 2019.

Axsome will host a conference call and webcast today at 8:00 AM Eastern to discuss the expedited development plan for AXS-05 in the treatment of major depressive disorder following a Breakthrough Therapy meeting with the FDA. To participate in the live conference call, please dial (844) 698-4029 (toll-free domestic) or (647) 253-8660 (international), and use the conference ID 3977129. The live webcast can be accessed on the “Webcasts & Presentations” page of the “Investors” section of the Company’s website at axsome.com. A replay of the webcast will be available for approximately 30 days following the live event.

https://www.marketscreener.com/AXSOME-THERAPEUTICS-INC-24949017/news/Axsome-Therapeutics-Announces-Expedited-Development-and-Pivotal-Status-for-AXS-05-in-the-Treatment-28542762/

Pfizer: FDA OKs Transthyretin Amyloid Cardiomyopath Med

Pfizer Inc. (NYSE:PFE) announced today that the U.S. Food and Drug Administration (FDA) has approved both VYNDAQEL^® (tafamidis meglumine) and VYNDAMAX™(tafamidis) for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. VYNDAQEL and VYNDAMAX are two oral formulations of the first-in-class transthyretin stabilizer tafamidis, and the first and only medicines approved by the FDA to treat ATTR-CM.

Transthyretin amyloid cardiomyopathy is a rare, life-threatening disease characterized by the buildup of abnormal deposits of misfolded protein called amyloid in the heart and is defined by restrictive cardiomyopathy and progressive heart failure. Previously, there were no medicines approved to treat ATTR-CM; the only available options included symptom management, and, in rare cases, heart (or heart and liver) transplant. It is estimated that the prevalence of ATTR-CM is approximately 100,000 people in the U.S. and only one to two percent of those patients are diagnosed today.

https://www.marketscreener.com/PFIZER-23365019/news/Pfizer-U-S-FDA-Approves-VYNDAQEL-and-VYNDAMAX-trade-for-Use-in-Patients-with-Transthyretin-Amyl-28542830/

Epizyme reports Q1 EPS (39c), consensus (46c)

Reports Q1 revenue $7.9M, consensus $2.16M. Reports cash, cash equivalents and marketable securities were $371.1M as of March 31, as compared to $247.9M as of March 31, 2018.
https://thefly.com/landingPageNews.php?id=2903507

ResMed upgraded to Buy from Neutral at UBS

https://thefly.com/landingPageNews.php?id=2903483

Pacira upgraded to Neutral from Underperform at Mizuho

Mizuho analyst Irina Koffler upgraded Pacira Biosciences (PCRX) to Neutral from Underperform and raised her price target for the shares to $38 from $29. The analyst says her sell thesis on the shares is longer valid due to an unexpected delay at competitor Heron Therapeutics (HRTX) and Pacira’s stronger guidance on recently acquired iovera. However, she still remains concerned about the longer-term outlook for the company.
https://thefly.com/landingPageNews.php?id=2903485

Biohaven Pharmaceutical initiated with a Buy at Goldman Sachs

Goldman Sachs analyst Paul Choi started Biohaven Pharmaceutical with a Buy rating and $92 price target. The analyst believes Biohaven will become a “more significant presence in the attractive” calcitonin gene-related peptide/migraine space than is currently priced into the shares. Rimegepant’s profile can drive above consensus sales on conservative patient numbers, Choi tells investors in a research note.

https://thefly.com/landingPageNews.php?id=2903497

Insulet upgraded to Buy from Neutral at BTIG

https://thefly.com/landingPageNews.php?id=2903449