BPC November 27 update

Vertex VRTX positive cystic fibrosis data +5%; Arsanis ASNS +255% on reverse merger

Price and Volume Movers

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) shares closed up 5% to $175.45 following its announcement of positive data from two Phase 3 trials of VX-659, tezacaftor and ivacaftor in patients with cystic fibrosis. Data from its trial of patients with one F508del mutation and one minimal function mutation showed a mean absolute improvement in ppFEVof 14 percentage points from baseline compared to placebo (p<0.0001). An improvement of 10 percentage points was noted in the trial of patients with two F508del mutations. 

Catalyst Pharmaceuticals Inc. (Nasdaq:CPRX) noted that a contractor working on a web page unintentionally allowed a live screen to be seen that showed the approval of Firdapse, which has a PDUFA date set for November 28. The website http://www.firdapse.com/ which initially displayed news of the FDA approval has now been taken down.

Zymeworks Inc. (NYSE: ZYME) announced a license agreement with BeiGene, Ltd. (Nasdaq: BGNE) for the development and commercialization of its pipeline candidates ZW25 and ZW49. In addition, Zymeworks granted BeiGene a license to its Azymetric and EFECT platforms. Zymeworks will receive total upfront payments of $40m under the ZW25 and ZW49 agreements and $20m under the platform agreement and is eligible to receive development and commercial milestone payments plus potential royalties on product sales. Shares closed up 5% to $12.00 after trading up as high as $13.80 (+20%) earlier in the session. 

Arsanis, Inc. (Nasdaq: ASNS) shares closed up 255% to $4.12 following news of a reverse merger with privately-held X4 Pharmaceuticals, Inc. Current X4 Pharmaceuticals holders will own 70% of the combined company while Arsanis holders will own the remaining 30%. To put today’s price surge into context, Arsanis is a recent IPO, listing in November 2017 at $10.00 which compares to today’s close of $4.12.

Tonix Pharmaceuticals Holding Corp. (Nasdaq:TNXP) announced that it will effect a 1-for-10 reverse stock split of its outstanding common stock. This will be effective for trading purposes as of the commencement of trading on Wednesday, November 28, 2018.

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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:

Cocrystal Pharma, Inc. (COCP): $3.27; +13%.

Sophiris Bio, Inc. (SPHS): $2.08; +12%.

Veru Inc. (VERU): $1.49; +12%.

Aileron Therapeutics, Inc. (ALRN): $2.12; +10%.

Gamida Cell Ltd. (GMDA): $14.43; +8%.

DECLINERS:

LogicBio Therapeutics, Inc. (LOGC): $13.20; -17%.

Therapix Biosciences Ltd. (TRPX): $5.77; -17%.

Aquestive Therapeutics, Inc. (AQST): $9.31; -14%.

Forward Pharma A/S (FWP): $1.05; -14%.

Amarin Corporation plc (AMRN): $16.83; -13%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ALRN – Aileron Therapeutics Inc.
ALRN-6924 and IBRANCE (palbociclib)
Solid tumors

Phase 1b Phase 1b enrolment to commence 1Q 2019.
$17.7 million

ASLN – ASLAN Pharmaceuticals Limited
Varlitinib + gemcitabine and cisplatin
Biliary tract cancer

Phase 1/2 Phase 1/2 new data to be presented at at ASCO Gastrointestinal Cancers Symposium January 18, 2019 11:30 AM.
$139.4 million

BLUE – bluebird bio Inc.
bb2121 - KarMMa
Relapsed/refractory multiple myeloma

Phase 2 Phase 2 pivotal trial completion of enrolment announced November 27, 2018. Data likely in 2019.
$5.7 billion

CYTK – Cytokinetics Incorporated
Reldesemtiv (CK-2127107) - FORTITUDE-ALS
Amyotrophic lateral sclerosis (ALS)

Phase 2 Phase 2 completion of enrolment announced November 27, 2018 with data due 1H 2019.
$421.3 million

DARE – Dare Bioscience Inc.
Sildenafil Cream, 3.6%
Female Sexual Arousal Disorder (FSAD)

Phase 2 Phase 2b trial to be initiated in 2019 with data due 4Q 2020.
$8.6 million

EVLO – Evelo Biosciences Inc.
EDP1503 and KEYTRUDA (pembrolizumab)
Colorectal cancer; Triple-negative breast cancer

Phase 1/2 Phase 1/2 trial to be initiated 1H 2019.
$361.9 million

EVLO – Evelo Biosciences Inc.
EDP1503
Melanoma

Phase 2a Phase 2a trial to be initiated 4Q 2018 with initial data due 2H 2019.
$361.9 million

GWPH – GW Pharmaceuticals Plc
GWP42006 (CBDV)
Autism

Phase 2
$3.7 billion

GWPH – GW Pharmaceuticals Plc
Epidiolex
Tuberous Sclerosis Complex

Phase 3 Phase 3 data due 1H 2019.
$3.7 billion

GWPH – GW Pharmaceuticals Plc
GWP42006 (CBDV)
Rett syndrome

Phase 2 Phase 2 open-label trial to commence 1Q 2019.
$3.7 billion

NCNA – NuCana plc
NUC-1031 (Acelarin)
Front-Line Treatment of Advanced Biliary Tract Cancer

Phase 3 Phase 3 trial to be initiated by YE 2018.
$533 million

NCNA – NuCana plc
NUC-3373
Solid tumors

Phase 1 Phase 1 additional data due 2019.
$533 million

NCNA – NuCana plc
NUC-1031 (Acelarin)
Pancreatic cancer

Phase 3 Phase 3 ongoing.
$533 million

NCNA – NuCana plc
NUC-1031 (Acelarin)
Platinum-sensitive ovarian cancer

Phase 2 Phase 2 interim data due 2019.
$533 million

NCNA – NuCana plc
NUC-3373
Colorectal Cancer

Phase 1 Phase 1 initial data due 2019.
$533 million

NCNA – NuCana plc
NUC-7738
Solid tumors

Phase 1 Phase 1 trial to be initiated YE 2018 with data due 2019.
$533 million

NVUS – Novus Therapeutics Inc.
OP0201
Otitis media

Phase 1 Phase 1 initiation of dosing announced November 27, 2018 with data due 1H 2019.
$28.3 million

PHAS – PhaseBio Pharmaceuticals Inc.
PB1046
Pulmonary arterial hypertension (PAH)

Phase 2b Phase 2b dosing of first patient announced November 27, 2018 with data due 1H 2020.
$100.9 million

PRTK – Paratek Pharmaceuticals Inc.
Omadacycline
Urinary tract infections

Phase 2 Phase 2 data due 2H 2019.
$193.2 million

PRTK – Paratek Pharmaceuticals Inc.
Omadacycline
Complicated urinary tract infections (cUTI)

Phase 2 Phase 2 dosing announced November 27, 2018 with data due 2H 2019.
$193.2 million

PTGX – Protagonist Therapeutics Inc.
PN-10943
Inflammatory bowel disease (IBD)

Phase 1 Phase 1 trial initiation announced December 13, 2018 with data due 1H 2019.
$166.6 million

RCKT – Rocket Pharmaceuticals Inc.
RP-L102
Fanconi Anemia (FA)

Phase 1 Clinical trials to commence early 2019.
$610.3 million

TBPH – Theravance Biopharma Inc.
TD-8236
Asthma

Phase 1 Phase 1 dosing announced November 27, 2018.
$1.5 billion

VRTX – Vertex Pharmaceuticals Incorporated
VX-659 in combination with tezacaftor and ivacaftor
Cystic Fibrosis Who Have Two Copies of the F508del Mutation

Phase 3 Phase 3 positive data released November 27, 2018. 10% improvement in ppFEV1 cf placebo.
$43.5 billion

VRTX – Vertex Pharmaceuticals Incorporated
VX-659 in combination with tezacaftor and ivacaftor
Cystic fibrosis (CF) who have one F508del mutation and one minimal function mutation (F508del/Min)

Phase 3 Phase 3 positive data released November 27, 2018. 14% improvement in ppFEV1 cf placebo.
$43.5 billion