BPC March 04 update

Ascendis ASND positive late-stage data - shares +74%; ​Nightstar NITE acquired by Biogen +66%

Price and Volume Movers

Ascendis Pharma A/S (Nasdaq: ASND) announced positive data from its Phase 3 theiGHt Trial, of once-weekly TransCon Growth Hormone (hGH) compared to a daily growth hormone (Genotropin) in children with pediatric growth hormone deficiency (GHD). The trial met its primary objective, demonstrating that TransCon hGH was superior to the daily hGH on the primary endpoint of annualized height velocity (AHV) at 52 weeks. Shares soared to close up 74% to $120.60. The company took advantage of the price surge when it announced after hours a public offering of $400m of American Depositary Shares (“ADSs”).

Nightstar Therapeutics plc (NASDAQ: NITE) shares surged to close up 66% to $25.18 on news that it will be acquired by Biogen Inc for $25.50 per share, or around $800m in cash.

Evoke Pharma, Inc. (NASDAQ: EVOK) shares slid, closing down 57% to $1.30 following receipt of a multi-disciplinary review (DR) letter from the FDA regarding its New Drug Application (NDA) for Gimoti. The letter described chemistry, clinical and clinical pharmacology concerns. The PDUFA date of April 1, 2019 remains. However, given the nature of the feedback from the FDA, an approval decision is unlikely.

MyoKardia, Inc. (Nasdaq: MYOK) shares closed up 7% to $51.22 following the release of positive 12- and 24-week safety, efficacy and biomarker data for mavacamten, from its PIONEER open-label extension (OLE) trial.

Agile Therapeutics, Inc. (Nasdaq: AGRX) shares closed up 20% to $1.11 following news of a stock purchase agreement with Perceptive Advisors, LLC, for the private placement of approximately 8.4m shares of common stock at $0.93 per share, resulting in expected gross proceeds of approximately $7.8m.

Horizon Pharma plc (Nasdaq: HZNP) announced that it is offering to sell $300m of its shares in an underwritten public offering.


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


TRACON Pharmaceuticals, Inc. (TCON): $1.29; +18%.

Sienna Biopharmaceuticals, Inc. (SNNA): $2.87; +15%.

Anavex Life Sciences Corp. (AVXL): $2.59; +14%.

Adverum Biotechnologies, Inc. (ADVM): $4.92; +12%.

NuCana plc (NCNA): $12.90; +12%.


Bio-Path Holdings, Inc. (BPTH): $5.50; -29%.

Intrexon Corporation (XON): $4.14; -18%.

Endo International plc (ENDP): $9.33; -17%.

Liquidia Technologies Inc. (LQDA): $17.41; -13%.

Xenetic Biosciences, Inc. (XBIO): $2.21; -11%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ADMS – Adamas Pharmaceuticals Inc.
GOCOVRI (amantadine)
Multiple sclerosis (MS)

Phase 3 Phase 3 data due 4Q 2019.
$144.5 million

AMRN – Amarin Corporation plc
High Triglycerides With Mixed Dyslipidemia

sNDA Filing sNDA filing announced March 28, 2019.
$5.8 billion

APLS – Apellis Pharmaceuticals Inc.
Geographic atrophy (GA) associated with age-related macular degeneration (AMD)

Phase 3 Phase 3 enrolment to be completed 1Q 2020.
$1.2 billion

ASND – Ascendis Pharma A/S
TransCon hGH
Growth hormone deficiency in children

Phase 3 Phase 3 data released March 4, 2019. Primary endpoint met. Phase 3 fliGHt trial detailed data due June 26, 2019.
$5.9 billion

AUTL – Autolus Therapeutics plc
Acute lymphoblastic leukemia (ALL)

Phase 2 Phase 2 trial to be initiated 2H 2019.
$978.7 million

AXSM – Axsome Therapeutics Inc.

Phase 3 Phase 3 top-line data due 2H 2019.
$835.6 million

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.
$379.5 million

BCRX – BioCryst Pharmaceuticals Inc.
Healthy subjects

Phase 1 Phase 1 trial to be initiated 2Q 2019 with data due 4Q 2019.
$379.5 million

BPMX – BioPharmX Corporation

Phase 2b Phase 2b data due July 2019.
$9.5 million

CYDY – CytoDyn Inc
Leronlimab (PRO 140)

BLA Filing Rolling BLA filing initiated March 18, 2019.
$139.2 million

EVOK – Evoke Pharma Inc.
Gimoti - EVK-001
Female diabetic gastroparesis

CRL CRL issued April 2, 2019.
$14.1 million

INO – Inovio Pharmaceuticals Inc.
VGX-3100 - REVEAL-2
Cervical Dysplasia

Phase 3 Phase 3 initiation announced March 4, 2019.
$328.4 million

IPCIF – Intellipharmaceutics International Inc - Ordinary Shares
Pain relief

PDUFA CRL issued September 25, 2017. NDA resubmitted with new PDUFA date of August 28, 2019.
$5.8 million

LJPC – La Jolla Pharmaceutical Company
Hereditary Hemochromatosis

Phase 2 Phase 2 data due 2H 2019.
$154.7 million

MYOK – MyoKardia Inc.
Mavacamten (MYK-461) - PIONEER-OLE
Non-obstructive Hypertrophic cardiomyopathy (HCM)

Phase 2 Phase 2 interim positive data released March 4, 2019. Data to be presented at ESC August 31-September 4, 2019.
$2.3 billion

ODT – Odonate Therapeutics Inc.
Tesetaxel - Contessa 2
Metastatic breast cancer (MBC)

Phase 2 Phase 2 trial initiation announced March 4, 2019.
$603.1 million

OTIC – Otonomy Inc.

Phase 1/2 Phase 1/2 trial initiation announced April 11, 2019 with data due 1H 2020.
$82.2 million

OTIC – Otonomy Inc.
Hidden hearing loss

Phase 1/2 Phase 1/2 trial to be initiated 3Q 2019 with data due 2H 2020.
$82.2 million

OTIC – Otonomy Inc.
Ménière's disease

Phase 3 Additional Phase 3 trial data due 1H 2020.
$82.2 million

PHAS – PhaseBio Pharmaceuticals Inc.
Reversal agent for ticagrelor

Phase 2a Phase 2a initial data due 2Q 2019.
$302.9 million

RNN – Rexahn Pharmaceuticals Inc.
RX-3117 in combination with Abraxane
First-line pancreatic cancer

Phase 2a Phase 2 completion of enrolment announced March 4, 2019. Further data due late-2019 or early 2020.
$20 million

SBBP – Strongbridge Biopharma plc
COR-003 (levoketoconazole) - SONICS
Endogenous Cushing’s syndrome

Phase 3 NDA to be filed 3Q 2020.
$161.4 million

SESN – Sesen Bio Inc.
Non-muscle invasive bladder cancer (NMIBC)

Phase 3 Phase 3 complete response rate 39% after 3 months, 15% after 12 months. Pre-BLA meeting June 6, 2019.
$159.6 million