BPC January 13 update

​Verona VRNA shares add 21% on positive mid-stage data; ​Acasti ACST slides 65% on late-stage fail

Price and Volume Movers

Verona Pharma plc (Nasdaq: VRNA) announced that it met the primary endpoint of improved lung function in its Phase 2b dose-ranging trial evaluating nebulized ensifentrine or placebo as an add-on treatment to tiotropium, in patients with moderate to severe chronic obstructive pulmonary disease (COPD). Shares closed up 21% to $7.23.

Acasti Pharma Inc. (NASDAQ: ACST) announced that its Phase 3 TRILOGY 1 trial of CaPre for the treatment of severe hypertriglyceridemia did not show a statistically significant decrease in triglycerides compared with placebo. Data from its second trial are due by mid-February. Shares tumbled to close down 65% to $0.76. Shares of Amarin Corporation plc (NASDAQ: AMRN) closed up 4% to $19.75 on the news, also aided by the announcement that AstraZeneca (NYSE: AZN) will also be closing its Phase 3 CV outcomes trial of Epanova for the treatment of mixed dyslipidaemia.

NantKwest, Inc. (NK) shares spiked in late-trading, closing up 91% to $6.80. The price action was in response to an interview on Bloomberg Television, where the CEO, Patrick Soon-Shiong, noted one complete response in a patient with metastatic pancreatic cancer following treatment with its immune-system based therapy.

Adaptimmune Therapeutics plc (NASDAQ: ADAP) shares closed up 200% to $3.99 following a trial update at the J.P.Morgan Conference. The company announced two partial responses in its Phase 1 trials in patients with liver cancer and melanoma.

Dare Bioscience, Inc. (NASDAQ: DARE) shares closed up 98% to $1.65. The company announced a license agreement with Bayer, where it may commercialize its contraceptive product, Ovaprene. The deal includes up to $310m in milestone payments. The upfront payment, which was disclosed in a SEC filing but not in the press release, is $1m.

Aquestive Therapeutics, Inc. (NASDAQ:AQST) shares fell 34% to $4.36 following news the FDA denied the company’s Citizen's Petition requesting the FDA stay approval of a New Drug Application (NDA) for Valtoco (diazepam nasal spray) submitted by Neurelis, Inc for the treatment of epilepsy cluster seizures. The FDA noted it had approved Neurelis's NDA on January 10, 2020.

-

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

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

ADVANCERS:

Liquidia Technologies, Inc. (LQDA): $5.55; +23%.

CEL-SCI Corporation (CVM): $9.34; +20%.

VBI Vaccines Inc. (VBIV): $1.91; +17%.

La Jolla Pharmaceutical Company (LJPC): $7.00; +17%.

Alpine Immune Sciences, Inc. (ALPN): $4.41; +16%.

DECLINERS:

SELLAS Life Sciences Group, Inc. (SLS): $2.95; -19%.

Urovant Sciences Ltd. (UROV): $11.51; -17%.

Salarius Pharmaceuticals, Inc. (SLRX): $3.14; -17%.

Cassava Sciences, Inc. (SAVA): $6.99; -14%.

Exact Sciences Corporation (EXAS): $90.16; -14%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ACST – Acasti Pharma Inc.
CaPre (TRILOGY 1 and 2)
Hypertriglyceridemia

Phase 3 Phase 3 TRILOGY 1 data did not meet primary endpoint - January 13, 2020. TRILOGY 2 data due on OR about August 31, 2020,
$65.6 million

ADAP – Adaptimmune Therapeutics plc
MAGE-A4 (ADP-A2M4)
Solid tumors

Phase 1 Phase 1 durability and translational data from patients with synovial sarcoma due at a medical conference 2H 2020.
$1.2 billion

AZN – Astrazeneca PLC
Lynparza + Avastin- PAOLA-1
First-line ovarian cancer

Approved FDA Approval announced May 8, 2020.
$144.8 billion

AZN – Astrazeneca PLC
Epanova
Hypertriglyceridaemia CVOT

Phase 3 Phase 3 trial to be discontinued due to low likelihood of demonstrating a benefit - January 13, 2020.
$144.8 billion

BPMC – Blueprint Medicines Corporation
Avapritinib - EXPLORER
Advanced Systemic mastocytosis (SM)

Phase 1 Phase 1 top-line data due 3Q 2020.
$4.1 billion

CHMA – Chiasma Inc.
Mycapssa - OPTIMAL
Acromegaly

Approved FDA approval announced June 26, 2020.
$264.2 million

CYCN – Cyclerion Therapeutics Inc.
IW-6463
Healthy volunteers

Phase 1 Phase 1 translational pharmacology top-line data due late-summer 2020.
$176 million

PTCT – PTC Therapeutics Inc.
PTC-AADC
AADC deficiency

BLA Filing BLA filing due 2H 2020.
$3.3 billion

RARE – Ultragenyx Pharmaceutical Inc.
Burosumab
Tumor-induced osteomalacia (TIO)

Approved FDA Approval announced June 18, 2020.
$5.2 billion

RUBY – Rubius Therapeutics Inc.
RTX-134
Phenylketonuria (PKU)

Phase 1/2 Phase 1b trial to be discontinued - noted March 12, 2020
$420 million

SLS – SELLAS Life Sciences Group Inc.
NeuVax in combination with Herceptin
Breast cancer - 1+/2+

Phase 3 Phase 3 trial planned.
$19.6 million

SRPT – Sarepta Therapeutics Inc.
Casimersen
Duchenne muscular dystrophy

NDA Filing Rolling NDA has been completed - June 26, 2020.
$12.3 billion

TENX – Tenax Therapeutics Inc.
Levosimendan
Pulmonary Hypertension associated with Heart Failure and preserved Ejection Fraction (PH-HFpEF)

Phase 2 Phase 2 top-line data released June 2, 2020. Primary endpoint (Pulmonary Capillary Wedge Pressure (PCWP) with bicycle exercise) not met. Various secondary endpoints met.
$16.3 million

VERU – Veru Inc.
Zuclomiphene citrate
Hot flashes

Phase 2 Phase 3 trial to commence late-2020.
$187.2 million