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.

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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:

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 mid-February 2020.
$60.9 million

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

Phase 1 Phase 1 updated data noted one partial response - January 13, 2020.
$423.8 million

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

PDUFA priority review PDUFA date under priority review 2Q 2020.
$131.7 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.
$131.7 billion

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

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

CHMA – Chiasma Inc.
Mycapssa - OPTIMAL
Acromegaly

PDUFA PDUFA date June 26, 2020.
$199.6 million

CYCN – Cyclerion Therapeutics Inc.
IW-6463
Healthy volunteers

Phase 1 Phase 1 translational pharmacology top-line data due mid-2020.
$101.1 million

PTCT – PTC Therapeutics Inc.
GT-AADC
AADC deficiency

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

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

sNDA Filing sBLA filing announced January 13, 2020.
$3.3 billion

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

Phase 1/2 Timing of data to be released once the first patient has been dosed - noted January 13, 2020.
$675.1 million

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

Phase 2b Waiting on further guidance from FDA regarding future development - likely 1Q 2020.
$15.1 million

SRPT – Sarepta Therapeutics Inc.
Casimersen
Duchenne muscular dystrophy

NDA Filing Rolling NDA commenced January 2020.
$8.5 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 due 1H 2020.
$9.5 million

VERU – Veru Inc.
Zuclomiphene citrate
Hot flashes

Phase 2 Phase 2 data released January 13, 2020. 50mg dose shown to be effective (10mg ineffective). Phase 3 trial to commence 1H 2020.
$279 million