BPC April 08 update

Zogenix ZGNX issued Refusal to File letter -32%; PhaseBio PHAS +34% on Breakthrough Therapy designation

Price and Volume Movers

PhaseBio Pharmaceuticals, Inc. (Nasdaq: PHAS) announced after hours the FDA has granted Breakthrough Therapy designation (BTD) for PB2452, a reversal agent for the antiplatelet drug ticagrelor. Shares are trading up 34% after hours to $16.90, adding to gains during the normal trading session of 17%.

Zogenix, Inc. (NASDAQ: ZGNX) shares are trading down 32% to $35.51 after hours following news the FDA issued a rare Refusal to File (RTF) letter regarding its New Drug Application (NDA) for Fintepla (ZX008), for the treatment of seizures associated with Dravet syndrome. The FDA noted certain non-clinical trials were not submitted, while an incorrect version of a clinical dataset were submitted.

Histogenics Corporation (Nasdaq: HSGX) closed up 56% to $0.17 following news of a proposed reverse merger with Ocugen, Inc, a privately held company focused on eye diseases. Ocugen current holders will hold approximately 90% of the combined company, with current Histogenics holders retaining the remaining 10%.

SCYNEXIS, Inc. (NASDAQ:SCYX) shares closed up 10% to $1.80. The company’s pipeline includes treatment for Candida auris, an illness reported on by the New York Times over the weekend. The company will be presenting Phase 3 data from two patients with Candida auris enrolled in its CARES trial on April 13 at the European Congress of Clinical Microbiology and Infectious Diseases (ECCMID).

United Therapeutics Corporation (Nasdaq: UTHR) announced that its Phase 3 BEAT clinical trial of esuberaprost tablets in patients suffering from pulmonary arterial hypertension did not meet its primary endpoint of delayed time to first clinical worsening event. Shares closed down 6% to $113.92.

Concert Pharmaceuticals, Inc.(NASDAQ: CNCE) shares were hit during early afternoon trading on news it lost its inter partes review (IPR), with the U.S. Patent Trial and Appeal Board (PTAB) ruling that the 149 patent is not patentable. Incyte originally filed the IPR in 2017 seeking to invalidate Concert’s patent. Shares closed down 16% to $11.00.

Autolus Therapeutics plc (Nasdaq: AUTL) announced after hours a public offering of 4m American Depositary Shares (ADSs).

Zosano Pharma Corporation (Nasdaq:ZSAN) shares are trading down 20% after hours also on news of a public offering of its common stock.

Daré Bioscience, Inc. (NASDAQ:DARE) shares are trading down 17% to $1.14 after hours on news it intends to offer shares of its common stock in an underwritten public offering.

GlaxoSmithKline plc (NYSE: GSK) announced the FDA approved Dovato, a complete, once-daily, single-tablet regimen of dolutegravir and lamivudine for the treatment of HIV-1 infection.

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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:

Melinta Therapeutics, Inc. (MLNT): $4.94; +15%.

Celldex Therapeutics, Inc. (CLDX): $4.98; +15%.

CEL-SCI Corporation (CVM): $6.15; +12%.

Avadel Pharmaceuticals plc (AVDL): $1.36; +10%.

Nabriva Therapeutics plc (NBRV): $3.07; +10%.

DECLINERS:

Outlook Therapeutics, Inc. (OTLK): $4.78; -22%.

Vaxart, Inc. (VXRT): $1.49; -22%.

Kiniksa Pharmaceuticals, Ltd. (KNSA): $18.23; -11%.

Bio-Path Holdings, Inc. (BPTH): $16.55; -10%.

Fortress Biotech, Inc. (FBIO): $1.73; -9%.


Pipeline Database Updates

Drug Stage Catalyst Market Cap

AGLE – Aeglea BioTherapeutics Inc.
Pegzilarginase (AEB1102)
Arginase I deficiency

Phase 1/2 Phase 3 trial initiation announced June 3, 2019 with data due 1Q 2021. Phase 1/2 further data due September 2019.
$197.6 million

AGTC – Applied Genetic Technologies Corporation
AAV-based gene therapy
X-linked Retinitis Pigmentosa (XLRP)

Phase 1/2 Phase 1/2 dose escalation data due 3Q 2019. Expansion data due 4Q 2019.
$68.1 million

BLPH – Bellerophon Therapeutics Inc.
INOpulse delivery device
Pulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD)

Phase 2b Phase 2b cohort 2 data and pivotal cohort 3 (Phase 3) initiation due 2H 2019.
$43.4 million

BOLD – Audentes Therapeutics Inc.
AT702
Duchenne Muscular Dystrophy (DMD)

Phase 1/2 Phase 1/2 trial to be initiated 4Q 2019.
$1.6 billion

GSK – GlaxoSmithKline PLC
Dolutegravir + lamivudine
HIV

Approved FDA approval announced April 8, 2019.
$102.9 billion

NAVB – Navidea Biopharmaceuticals Inc.
NAV3-31 (TC99m-tilmanocept)
Rheumatoid arthritis

Phase 2b Phase 2b trial is enrolling.
$12.1 million

NGM – NGM Biopharmaceuticals Inc.
NGM313 / MK-3655
Non-alcoholic steatohepatitis (NASH) and Type 2 diabetes

Phase 2 Phase 2 trial to be initiated 2020.
$907.8 million

NTGN – Neon Therapeutics Inc.
NEO-PV-01 + Keytruda - NT-002
Non-small Cell Lung Cancer (NSCLC)

Phase 1b Phase 1b data due 3Q 2020.
$131.7 million

ONCY – Oncolytics Biotech Inc.
Pelareorep and Tecentriq - AWARE-1
Breast cancer

Phase 1b Phase 1b interim data due 2H 2019.
$33.3 million

QURE – uniQure N.V.
AMT-130
Huntington's disease

Phase 1/2 Phase 1/2 trial to commence 2H 2019.
$2.6 billion

SRPT – Sarepta Therapeutics Inc.
MYO-101
Duchenne muscular dystrophy - LGMD2E

Phase 1/2 Phase 1/2 data February 27, 2019 noted mean of 51% beta-sarcoglycan positive fibers.
$11.4 billion

UTHR – United Therapeutics Corporation
Esuberaprost - BEAT
Pulmonary arterial hypertension (PAH)

Phase 3 Phase 3 data released April 8, 2019 did not meet primary endpoint.
$3.3 billion

ZGNX – Zogenix Inc.
FINTEPLA (ZX008)
Dravet syndrom

NDA Filing Refusal to file Letter (RTF) issued April 8, 2019. Intends to resubmit NDA in 3Q 2019.
$2 billion