BPC November 26 update

​Zafgen ZFGN clinical hold sends shares down 41%; Halozyme HALO alters late-stage endpoint; ACAD AMRN RCKT offerings

Price and Volume Movers

Zafgen, Inc. (Nasdaq:ZFGN) announced that the FDA has placed a clinical hold on the Investigational New Drug Application (IND) of ZGN-1061, currently in development for the treatment of type 2 diabetes. The FDA cited the possibility of cardiovascular (CV) safety risks for the hold.  Shares plunged to close the session down 41% to $5.41. 

Loxo Oncology (NASDAQ: LOXO) received FDA approval of larotrectinib for the treatment of adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment.

GW Pharmaceuticals plc (Nasdaq: GWPH) announced positive top-line data from its second Phase 3 trial of Epidiolex for the treatment of seizures associated with Dravet syndrome, a rare and severe form of childhood-onset epilepsy. The trial achieved the primary endpoint of reduction in convulsive seizures for both dose levels (10 mg/kg per day and 20 mg/kg per day) with high statistical significance compared to placebo. Shares closed up 3% to $127.70 after trading as high as $133 earlier in the session. 

Bristol-Myers Squibb Company (NYSE: BMY) announced that its Phase 3 CheckMate-451 trial of Opdivo and Yervoy did not meet its primary endpoint of overall survival (OS) as a maintenance therapy for patients with small cell lung cancer (SCLC) without disease progression after completion of first-line platinum-based chemotherapy. Shares are trading down 2.7% to $51.30 in the after-hours session. 

Amarin Corporation plc (NASDAQ: AMRN) announced a registered underwritten public offering of its American Depositary Shares. Shares are trading down after hours 6% to $18.23. 

ACADIA Pharmaceuticals Inc. (Nasdaq: ACAD) announced after hours a public offering of $200m of its common stock. Shares have fallen 7% to $18.03 after hours. 

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) also announced after hours that it has commenced an underwritten public offering of $50m of its common stock. 

G1 Therapeutics, Inc. (Nasdaq: GTHX) shares are trading down 12% to $35.00 after hours following the release of top-line data from its Phase 2 trial evaluating trilaciclib in combination with chemotherapy Tecentriq (atezolizumab) as a treatment of first-line small cell lung cancer (SCLC), with investors reacting to overall response rate data of trilaciclib which was lower than placebo.

There was no statistical difference between the trilaciclib and placebo groups in overall response rate (ORR) (trilaciclib 56.0%, placebo 63.5%) and median duration of response (DOR) (trilaciclib 5.2 months, placebo 4.2 months). Preliminary median progression-free survival (PFS) was 5.7 months for trilaciclib versus 5.4 months for placebo (hazard ratio 0.74, p=0.3025; less than 80% of events). 

Halozyme Therapeutics, Inc. (NASDAQ: HALO) shares are trading down 13% to $14.50 after hours following news that it has changed the primary endpoint of its Phase 3 trial, HALO-301 in patients with pancreatic cancer, to the single primary endpoint of overall survival (OS). As a result, the previously planned analysis of progression free survival (PFS) will not be conducted. 

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

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

ADVANCERS:

Tyme Technologies, Inc. (TYME): $2.80; +30%.

InVivo Therapeutics Holdings Corp. (NVIV): $1.92; +16%.

Soleno Therapeutics, Inc. (SLNO): $2.14; +12%.

Mersana Therapeutics, Inc. (MRSN): $6.00; +10%.

Verona Pharma plc (VRNA): $12.50; +9%.

DECLINERS:

Tenax Therapeutics, Inc. (TENX): $2.61; -20%.

ReShape Lifesciences Inc. (RSLS): $1.01; -19%.

Curis, Inc. (CRIS): $1.09; -13%.

LogicBio Therapeutics, Inc. (LOGC): $15.95; -11%.

Pfenex Inc. (PFNX): $4.03; -11%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

AMPE – Ampio Pharmaceuticals Inc.
Ampion
Osteoarthritis of the Knee

Phase 3 Noted August 7, 2018 that further trials required before filing BLA, confirmed December 13, 2018.
$42.7 million

APTO – Aptose Biosciences Inc.
APTO-253
Acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS)

Phase 1b Phase 1b clinical hold released June 29, 2018. First patient of re-initiation announced November 26, 2018. To be completed in 2019.
$85.3 million

ATNX – Athenex Inc.
Oraxol and pembrolizumab (Keytruda)
Solid Tumors

Phase 1/2 Phase 1/2 initiation announced November 26, 2018.
$800.7 million

BMY – Bristol-Myers Squibb Company
CM-451 – Opdivo + Yervoy
First-line Small cell lung cancer (SCLC)

Phase 3 Phase 3 trial did not meet primary endpoint - November 26, 2018.
$85.1 billion

ENTA – Enanta Pharmaceuticals Inc.
EDP-305 ARGON-1
Non-alcoholic steatohepatitis (NASH)

Phase 2 Phase 2 initial data due mid-2019.
$1.4 billion

ENTA – Enanta Pharmaceuticals Inc.
EDP-938
Respiratory Syncytial Virus

Phase 2 Phase 2 preliminary data due 3Q 2019.
$1.4 billion

EVLO – Evelo Biosciences Inc.
EDP1815
Psoriasis and Atopic Dermatitis

Phase 1b Phase 1b initiation of dosing announced November 26, 2018. Data due 2H 2019.
$361.9 million

EYEN – Eyenovia Inc.
MicroStat
Mydriasis - pupil dilation

Phase 3 Phase 3 trial initiation announced November 26, 2018 with data due 1H 2019.
$34.3 million

GTHX – G1 Therapeutics Inc.
Trilaciclib plus Tecentriq
First-line small-cell lung cancer

Phase 2 Phase 2 data released November 26, 2018. ORR trilaciclib 56.0%, placebo 63.5%.
$1.1 billion

HALO – Halozyme Therapeutics Inc.
PEGPH20 - HALO-301
Pancreatic cancer

Phase 3 Phase 3 dosing initiated March 2016. Noted November 26, 2018 new primary endpoint of overall survival and interim PFS analysis will not occur. Target number of OS events for final analysis est Aug - Nov 2019.
$2.2 billion

LOXO – Loxo Oncology Inc.
Larotrectinib (LOXO-101)
Solid tumors that harbor a TRK fusion.

Approved FDA Approval announced November 26, 2018.
$4.3 billion

ONCY – Oncolytics Biotech Inc.
Pelareorep with Keytruda
Pancreatic cancer

Phase 2 Phase 2 initiation of dosing announced November 26, 2018.
$34.5 million

SLS – SELLAS Life Sciences Group Inc.
NeuVax in combination with Herceptin
Breast cancer - HER2 3+

Phase 2 Phase 2 completion of enrolment announced November 26, 2018 with data due 4Q 2019.
$35.2 million

TYME – Tyme Technologies Inc.
SM-88
Pancreatic cancer

Phase 2 Phase 2 interim data to be presented January 18, 2019 at 11:30 AM at Gastrointestinal Cancers Symposium.
$397.6 million

ZGNX – Zogenix Inc.
ZX008 - Study 1504
Dravet syndrom

Phase 3 Phase 3 data released July 12, 2018. Primary endpoint met.
$1.7 billion