BPC May 08 update

Intercept ICPT stock offering -5% AH; Catalyst CPRX shares continue to slide -24%

Price and Volume Movers

Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) shares are trading down 5% to $83.00 on news it is commencing an underwritten public offering of $200m of its common stock and $200m of convertible senior notes.

AstraZeneca (NYSE: AZN) and Daiichi Sankyo Company, Limited announced positive top-line results for their pivotal Phase 2 DESTINY-Breast01 trial of trastuzumab deruxtecan (DS-8201) in patients with HER2-positive, unresectable and/or metastatic breast cancer previously treated with trastuzumab emtansine. The trial met its primary objective response rate endpoint. A Biologics License Application (BLA) is on track to be filed with the FDA in 2H 2019.

Catalyst Pharmaceuticals Inc (NASDAQ: CPRX) shares continued their slide, falling a further 24% to $2.79, adding to Tuesday’s loss of 40%. The price action follows news released late-Monday that the FDA granted approval of Ruzurgi to Jacobus Pharmaceutical Company, Inc. for the treatment of Lambert-Eaton myasthenic syndrome (LEMS), resulting in a potential competitor to Catalyst Pharmaceuticals.

Novavax, Inc. (Nasdaq: NVAX) announced it will effect a 1-for-20 reverse stock split on May 10, 2019.

Provention Bio, Inc. (Nasdaq: PRVB) announced preliminary top-line data from its Phase 1b PULSE study of PRV-300 in patients with moderate-to-severe ulcerative colitis (UC). The trial met the primary safety and tolerability endpoint but did not meet efficacy and other secondary endpoints. Shares are trading down 15% to $3.48 after hours.

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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:

Evofem Biosciences, Inc. (EVFM): $5.55; +25%.

ARCA biopharma, Inc. (ABIO): $15.10; +20%.

AVROBIO, Inc. (AVRO): $21.08; +13%.

Auris Medical Holding AG (EARS): $5.37; +13%.

PDS Biotechnology Corporation (PDSB): $6.75; +13%.

DECLINERS:

Equillium, Inc. (EQ): $6.08; -21%.

Aduro BioTech, Inc. (ADRO): $3.48; -21%.

Aquestive Therapeutics, Inc. (AQST): $4.52; -17%.

Cerus Corporation (CERS): $5.73; -13%.

Infinity Pharmaceuticals, Inc. (INFI): $1.50; -12%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ALNA – Allena Pharmaceuticals Inc.
Reloxaliase ALLN-177 (URIROX-1)
Enteric Hyperoxaluria

Phase 3 Data from first Phase 3 trial due 2H 2019.
$103.5 million

APLS – Apellis Pharmaceuticals Inc.
APL-2 subcutaneous PEGASUS
Paroxysmal Nocturnal Hemoglobinuria (PNH)

Phase 3 Phase 3 data due December 2019.
$1.7 billion

AQST – Aquestive Therapeutics Inc.
Libervant - AQST-203
Epileptic seizures

NDA Filing Rolling NDA filing has commenced - noted June 4, 2019. To be completed 4Q 2019.
$103.6 million

AVDL – Avadel Pharmaceuticals plc
FT 218
Excessive daytime sleepiness and cataplexy in patients suffering from narcolepsy

Phase 3 Phase 3 enrolment to be completed 2H 2020.
$81.8 million

AZN – Astrazeneca PLC
Trastuzumab deruxtecan - - DESTINY-Breast01
Third line HER2+ breast cancer

BLA Filing Phase 2 data met primary endpoint - May 8, 2019. BLA filing due 2H 2019.
$105.5 billion

BCRX – BioCryst Pharmaceuticals Inc.
BCX7353 - APeX-2
Reduce or eliminate attacks in HAE patients

Phase 3 Phase 3 data May 21, 2019 met primary endpoint but lower than expectations.
$397.1 million

CORV – Correvio Pharma Corp.
Brinavess
Atrial fibrillation (AF)

NDA Filing NDA resubmission announced June 24, 2019.
$77.8 million

FOLD – Amicus Therapeutics Inc.
CLN6 Gene Therapy
Batten disease

Phase 1/2 Phase 1/2 preliminary data due 3Q 2019.
$3.2 billion

ITCI – Intra-Cellular Therapies Inc.
ITI-007 - Monotherapy (Study 401)
Bipolar depression

Phase 3 Phase 3 trial did not meet primary endpoint - July 8, 2019.
$629.6 million

MRNA – Moderna Inc.
mRNA-1777
RSV vaccine

Phase 1 Development has been paused - May 8, 2019.
$4.7 billion

PRQR – ProQR Therapeutics N.V.
QR-421a - STELLAR
Usher Syndrome Type 2

Phase 1/2 Presentation of data made July 13, 2019.
$348.1 million

PRVB – Provention Bio Inc.
PRV-300
Ulcerative Colitis

Phase 1b Phase 1b top-line data May 8, 2019 noted primary safety and tolerability endpoint met but efficacy and other secondary endpoints were not met.
$478.6 million
ARV-110
Castration-resistant prostate cancer (CRPC)

Phase 1 Phase 1 initial data due 2H 2019.

ESPR – Esperion Therapeutics Inc.
Bempedoic Acid/ Ezetimibe (1002-058)
LDL-C and type 2 diabetes

Phase 2 Phase 2 data due 2H 2019.
$1.2 billion