BPC May 11 update

​MyoKardia MYOK releases positive late-stage data +59% while Genfit GNFT Phase 3 NASH trial disappoints -50%

Price and Volume Movers

MyoKardia, Inc. (Nasdaq: MYOK) shares surged to close up 59% to $96.90 on news its Phase 3 pivotal EXPLORER-HCM clinical trial of mavacamten for the treatment of patients with symptomatic, obstructive hypertrophic cardiomyopathy (HCM), met the primary and all secondary endpoints. The company also announced after hours its has commenced a public offering to sell $450m in shares of its common stock.

Cytokinetics, Incorporated (NASDAQ: CYTK) shares also traded higher in sympathy to the move by MyoKardia, closing up 33% to $20.99. Data from its trial of CK-274 in HCM are due in 2H 2020.

GENFIT (Nasdaq: GNFT) shares are trading down 50% to $10.90 after hours following news its Phase 3 RESOLVE-IT trial of elafibranor in adults with nonalcoholic steatohepatitis (NASH) did not meet the predefined primary endpoint of NASH resolution without worsening of fibrosis.

CymaBay Therapeutics, Inc. (NASDAQ:CBAY) announced an independent review of its Phase 2b trial of seladelpar in patients with NASH showed no seladelpar-induced liver injury in the study, and as a result they also supported the lifting of the clinical hold and re-initiation of clinical development which was initially terminated in November 2019. Shares are trading up 109% to $3.85 after hours.

Novavax, Inc. (NASDAQ: NVAX) shares closed up 31% to $24.50 and are trading up a further 38% after hours. The company announced the Coalition for Epidemic Preparedness Innovations (CEPI) will invest up to $384m of additional funding to advance clinical development of NVX-CoV2373, Novavax’ coronavirus vaccine candidate against SARS-CoV-2. A Phase 1 trial is slated to commence this month with initial data due in July.

Akebia Therapeutics, Inc. (Nasdaq: AKBA) announced after hours the commencement of a public offering of $100m of shares of its common stock. Shares are trading down 6% to $12.10 after closing 7% higher during normal trading hours.

Sutro Biopharma, Inc. (NASDAQ:STRO) shares are trading down 16% to $8.50 after hours after it also announced a proposed underwritten public offering.

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Major price movers (stocks priced > $1.00, volume > 50k):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume:

ADVANCERS:

MacroGenics, Inc. (MGNX): $26.32; +29%.

Amneal Pharmaceuticals, Inc. (AMRX): $4.56; +28%.

Wave Life Sciences Ltd. (WVE): $10.11; +27%.

Arcturus Therapeutics Ltd. (ARCT): $48.45; +24%.

Eiger BioPharmaceuticals, Inc. (EIGR): $13.04; +23%.

DECLINERS:

Recro Pharma, Inc. (REPH): $5.08; -40%.

Cellectar Biosciences, Inc. (CLRB): $1.47; -15%.

AMAG Pharmaceuticals, Inc. (AMAG): $8.23; -11%.

Infinity Pharmaceuticals, Inc. (INFI): $1.01; -9%.

LogicBio Therapeutics, Inc. (LOGC): $6.78; 7%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ACER – Acer Therapeutics Inc.
Emetine
COVID-19

Phase 2/3 Phase 2/3 trial to be initiated 3Q 2020.
$31.5 million

AIMT – Aimmune Therapeutics Inc.
AR 201
Egg Allergy

Phase 2 Phase 2 enrolment has closed due to cost cutting measures. Data due 1H 2021.
$1.1 billion

ALRN – Aileron Therapeutics Inc.
ALRN-6924
Small Cell Lung Cancer (SCLC)

Phase 1/2 Phase 1b/2 interim data due June 2020, with final data readout 4Q 2020.
$47.1 million

BLUE – bluebird bio Inc.
LentiGlobin - HGB-206 (EHA)
Sickle disease

Phase 1/2 Phase 1/2 updated data due at EHA June 11-14, 2020.
$3.5 billion

BPTH – Bio-Path Holdings Inc.
INOpulse Inhaled Nitric Oxide
COVID-19

Phase 3 Phase 3 trial initiation announced May 11, 2020.
$20.4 million

CBAY – CymaBay Therapeutics Inc.
Seladelpar
Non-alcoholic steatohepatitis (NASH)

Phase 2b Clinical development of Seladelpar discontinued - November 25, 2019. Noted May 11, 2020 that seladelpar did not cause drug-induced liver injury as noted in November 2019.
$255.2 million

CPRX – Catalyst Pharmaceuticals Inc.
Firdapse
MuSK-antibody positive myasthenia gravis (MuSK-MG)

Phase 3 Phase 3 data due 3Q 2020.
$445.7 million

CRBP – Corbus Pharmaceuticals Holdings Inc.
Resunab - RESOLVE-1
Systemic Sclerosis

Phase 3 Phase 3 top-line data due summer 2020.
$540.8 million

GNFT – GENFIT S.A.
Elafibranor - RESOLVE-IT
Non-alcoholic steatohepatitis (NASH)

Phase 3 Phase 3 data did not meet primary endpoint - May 11, 2020.
$202.7 million

MTNB – Matinas Biopharma Holdings Inc.
MAT2203 (EnACT)
Cryptococcal meningitis

Phase 1/2 Phase 1/2 announcement of progression from first cohort to the second cohort due 2H 2020. Full data due 2H 2021.
$177.7 million

MYOK – MyoKardia Inc.
Mavacamten (MYK-461) - EXPLORER-HCM
Hypertrophic cardiomyopathy (HCM)

Phase 3 Phase 3 data met endpoints - May 11, 2020.
$4.8 billion

NVAX – Novavax Inc.
NVX-CoV2373
Coronavirus COVID-19 vaccine

Phase 1/2 Phase 1 trial initiation announced May 25, 2020 with preliminary immunogenicity and safety results expected in July 2020.
$2.7 billion

OMER – Omeros Corporation
OMS721
Hematopoietic stem cell-associated TMA (HSCT-TMA)

BLA Filing Rolling BLA filing to be completed 3Q 2020.
$811.1 million

ZYNE – Zynerba Pharmaceuticals Inc.
Zygel ZYN002
22q11.2 Deletion Syndrome

Phase 2 Phase 2 data due 3Q 2020.
$131.2 million