BPC September 26 update

Marinus MRNS meets primary endpoint in late-stage trial +23%

Price and Volume Movers

Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS) shares closed up 23% to $1.69 on news that its open-label, dose-finding Phase 2 trial evaluating intravenous (IV) ganaxolone in patients with refractory status epilepticus (RSE), met the primary endpoint. Additional efficacy and safety data will be presented at the Neurocritical Care Society Annual Meeting on October 16, 2019. A Phase 2b trial is slated to commence in 1Q 2020.

Genmab A/S (Nasdaq: GMAB) and Johnson & Johnson (NYSE: JNJ) announced the FDA has approved the use of Darzalex (daratumumab) in combination with bortezomib, thalidomide and dexamethasone (VTd) as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant (ASCT).

Tetraphase Pharmaceuticals, Inc. (NASDAQ:TTPH) announced a 1-for-20 reverse stock split of its common stock will be effective from today and will begin trading on a split-adjusted basis when the market opens on September 27, 2019. Shares closed down 8% to $0.24.

Enanta Pharmaceuticals, Inc. (NASDAQ:ENTA) shares closed down 15% to $60.51 following Wednesday’s release of top-line data from its ARGON-1 Phase 2a study of EDP-305 for the treatment of non-alcoholic steatohepatitis (NASH). The trial only just met the primary endpoint, while the high-dose cohort saw 51% of patients exhibiting pruritus (itching of the skin). JP Morgan downgraded its rating of the company from Neutral to Underweight, while it also lowered its price target from $86 to $57.

Vaxart, Inc. (Nasdaq: VXRT) announced intraday the pricing of an underwritten public offering of 26,666,667 shares and common warrants to purchase up to 26,666,667 shares, at a combined effective price of $0.30. Prior to the news, shares had traded up over 35% early in the session, only to end the trading day down 3% to $0.30.

Arcturus Therapeutics (NASDAQ: ARCT) announced a registered direct offering of 850,000 shares at a purchase price of $11.50 per share, for aggregate gross proceeds of approximately $9.8m. Shares closed down 10% to $11.67.

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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:

Akero Therapeutics, Inc. (AKRO): $21.10; +10%.

IDEAYA Biosciences, Inc. (IDYA): $9.70; +10%.

Xenetic Biosciences, Inc. (XBIO): $1.40; +10%.

CEL-SCI Corporation (CVM): $9.60; +7%.

Applied Genetic Technologies Corporation (AGTC): $4.00; +6%.

DECLINERS:

Enlivex Therapeutics Ltd. (ENLV): $27.70; -24%.

BioXcel Therapeutics, Inc. (BTAI): $7.50; -24%.

PDS Biotechnology Corporation (PDSB): $3.43; -23%.

Springworks Therapeutics, Inc (SWTX): $18.80; -22%.

Surface Oncology, Inc. (SURF): $1.50; -15%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ADVM – Adverum Biotechnologies Inc.
ADVM-022
Wet age-related macular degeneration (Wet-AMD)

Phase 1 Phase 1 data February 8, 2020 - Cohort 1: 6 of 6 patients remain rescue-injection-free at median 50 weeks, with 3 patients at 52 weeks. Cohort 2 - 4 of 6 patients remain rescue-injection-free at 24 weeks at the lower dose.
$795.1 million

ALDX – Aldeyra Therapeutics Inc.
Reproxalap (ADX-102) - INVIGORATE
Allergic conjunctivitis

Phase 3 Phase 3 enrolment has commenced - January 21, 2020.
$106.2 million

CRTX – Cortexyme Inc.
COR388 - GAIN
Alzheimer’s disease

Phase 2/3 Phase 2/3 interim analysis to occur 4Q 2020. Final data due 4Q 2021.
$1.5 billion

FGEN – FibroGen Inc
Roxadustat
Chemotherapy-induced anemia (CIA)

Phase 2 Phase 2 trial initiation announced September 26, 2019.
$3.6 billion

GLMD – Galmed Pharmaceuticals Ltd.
Aramchol - ARMOR
Non-Alcoholic Steatohepatitis (NASH)

Phase 3 Phase 3 initial data due 4Q 2022.
$102.5 million

IGXT – Intelgenx Techs Corp
RIZAPORT (RHB-103)
Migraine

PDUFA PDUFA date March 26, 2020.
$39.4 million

JNJ – Johnson & Johnson
Daratumumab + VTD
Multiple myeloma - candidates for autologous stem cell transplant (ASCT)

Approved FDA Approval announced September 26, 2019.
$354.3 billion

MLND – Millendo Therapeutics Inc.
Livoletide
Prader-Willi syndrome (PWS)

Phase 2/3 Phase 2b data due early-2Q 2020.
$135.6 million

MRNS – Marinus Pharmaceuticals Inc.
Ganaxolone
Refractory status epilepticus (RSE)

Phase 2 Phase 3 trial to be initiated mid-2020.
$216.3 million

RDHL – Redhill Biopharma Ltd.
RHB-104 MAP
Crohn’s disease

Phase 3 Phase 3 data released July 30, 2018. Primary endpoint met but further trial required.
$145.7 million

ZGNX – Zogenix Inc.
FINTEPLA (ZX008)
Dravet syndrom

PDUFA priority review PDUFA date under priority review extended by three months to June 25, 2020
$1.1 billion

ZGNX – Zogenix Inc.
ZX008 - (Study 1601)
Lennox-Gastaut syndrome

Phase 3 Phase 3 data met primary endpoint with high dose, low dose secondary endpoint not met - February 6, 2020.
$1.1 billion