BPC January 04 update

​Epizyme EPZM shares surge 33% on accelerated approval plans for tazemetostat

Price and Volume Movers

Epizyme, Inc. (Nasdaq: EPZM) announced that following discussions with the FDA, it intends to seek accelerated approval of tazemetostat for patients with relapsed and/or refractory follicular lymphoma (FL). The company anticipates submitting a New Drug Application (NDA) in 4Q 2019. Shares closed up 33% to $7.94.

Nabriva Therapeutics plc (NASDAQ: NBRV) announced the FDA has accepted the New Drug Application (NDA) and granted a priority review for Contepo to treat complicated urinary tract infections (cUTIs). The PDUFA date is June 30, 2019. 

Mersana Therapeutics, Inc., (NASDAQ:MRSN) announced that it will terminate the development for XMT-1522. Shares, which were initially trading down 15% early in the session, pared back all losses to close up 3% to $4.15.

AmpliPhi Biosciences Corporation (NYSE American: APHB) shares closed up 19% to $0.27 on news of a reverse merger with privately-owned C3J TherapeuticsInc. After giving effect to a contemplated $10m financing, current C3J holders will own approximately 76% of the combined company while AmpliPhi holders will own approximately 24%.

Flex Pharma, Inc. (NASDAQ: FLKS) shares closed up 38% to $0.53 as it also announced a reverse merger with privately-owned Salarius Pharmaceuticals. Flex Pharma holders will own approximately 20% of the combined company while Salarius investors will own approximately 80%.

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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:

Achaogen, Inc. (AKAO): $1.69; +43%.

Sienna Biopharmaceuticals, Inc. (SNNA): $3.12; +27%.

Neos Therapeutics, Inc. (NEOS): $2.25; +24%.

Translate Bio, Inc. (TBIO): $8.38; +24%.

CTI BioPharma Corp. (CTIC): $1.21; +23%.

DECLINERS:

Arcus Biosciences, Inc. (RCUS): $8.95; -14%.

Avadel Pharmaceuticals plc (AVDL): $2.68; -12%.

Therapix Biosciences Ltd. (TRPX): $3.53; -8%.

Esperion Therapeutics, Inc. (ESPR): $41.73; -6%.

Adial Pharmaceuticals, Inc. (ADIL): $6.40; -5%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap
ARV-110
Castration-resistant prostate cancer (CRPC)

Phase 1 Phase 1 trial to be initiated 1Q 2019.

AMRN – Amarin Corporation plc
Vascepa REDUCE-IT outcomes trial
High Triglycerides With Mixed Dyslipidemia

Phase 3 sNDA filing due late 1Q 2019. New data due March 18, 2019 at ACC.
$5.8 billion

BPMC – Blueprint Medicines Corporation
Avapritinib BLU-285 - NAVIGATOR
PDGFRα driven Gastrointestinal stromal tumors (GIST)

Phase 1 Phase 1 additional data and NDA filing due 1H 2019.
$2.9 billion

BPMC – Blueprint Medicines Corporation
Avapritinib - Voyager
Gastrointestinal stromal tumors (GIST)

Phase 3 Phase 3 enrolment to be completed 2H 2019.
$2.9 billion

BPMC – Blueprint Medicines Corporation
Avapritinib BLU-285 - EXPLORER
Advanced Systemic mastocytosis (SM)

Phase 1 Phase 1 updated data due 1H 2019.
$2.9 billion

BPMC – Blueprint Medicines Corporation
Avapritinib BLU-285 - PATHFINDER
Advanced Systemic mastocytosis (SM)

Phase 2 Phase 2 enrolment to be completed 2H 2019.
$2.9 billion

BPMC – Blueprint Medicines Corporation
BLU-667 - ARROW
RET-altered solid tumors

Phase 1 Phase 1 updated data due 1H 2019. NDA filing due 1H 2020.
$2.9 billion

BPMC – Blueprint Medicines Corporation
Avapritinib BLU-285 - PIONEER
Indolent and smoldering systemic mastocytosis (SM)

Phase 2 Phase 2 initial data due 2H 2019.
$2.9 billion

BPMC – Blueprint Medicines Corporation
BLU-554
Advanced hepatocellular carcinoma (HCC)

Phase 1 Phase 1 China enrolment to commence mid-2019.
$2.9 billion

EPZM – Epizyme Inc.
Tazemetostat
Epithelioid Sarcoma

NDA Filing NDA filing planned for 2Q 2019.
$713.3 million

EPZM – Epizyme Inc.
Tazemetostat
Follicular lymphoma

Phase 2 NDA filing due 4Q 2019. Phase 2 updated data due mid-2019.
$713.3 million

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

Phase 1/2 Phase 1/2 trial initiation announced January 4, 2019 with initial data due 1H 2020.
$280.5 million

INSM – Insmed Inc.
INS1007
Bronchiectasis

Phase 2 Phase 2 enrolment to be completed mid-2019.
$1.8 billion

MESO – Mesoblast Limited
MSC-100-IV
acute graft versus host disease (aGVHD) in children

BLA Filing BLA filing due 1Q 2019.
$461.7 million

MESO – Mesoblast Limited
MPC-150-IM - Class 2/3
Heart failure

Phase 3 Phase 3 enrolment to be completed 1Q 2019.
$461.7 million

MRSN – Mersana Therapeutics Inc.
XMT1522
HER2 - breast cancer

Phase 1 Development to be discontinued - noted January 4, 2019.
$81.4 million

MRSN – Mersana Therapeutics Inc.
XMT-1536
Ovarian Cancer, Non Small Cell Lung Cancer

Phase 1 Phase 1 data due 1H 2019.
$81.4 million

NBRV – Nabriva Therapeutics plc
Contepo
Complicated urinary tract infections (cUTI)

PDUFA priority review PDUFA date under priority review April 30, 2019.
$124.6 million

OVID – Ovid Therapeutics Inc.
TAK-935/OV935 - ARCADE
CDKL5 Deficiency

Phase 1/2 Phase 1b/2a enrolment to be completed 2H 2019.
$81.6 million

OVID – Ovid Therapeutics Inc.
OV101 (gaboxadol)
Fragile X syndrome

Phase 2 Phase 2 top-line data due 2H 2019.
$81.6 million

PGNX – Progenics Pharmaceuticals Inc.
1095
Prostate Cancer

Phase 2 Phase 2 trial to be initiated 2Q 2019.
$396.5 million

RARE – Ultragenyx Pharmaceutical Inc.
DTX401
GSD1

Phase 1/2 Phase 1/2 positive low dose cohort data released January 4, 2019. Data from second cohort due mid-2019.
$2.5 billion

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
Alstrom Syndrome and Bardet-Biedl Syndrome

Phase 3 Phase 3 data due 2020.
$990.3 million

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
POMC Heterozygous Deficiency Obesity, Alstrom Syndrome, POMC Epigenetic Disorders

Phase 2 Phase 2 updated data due 1Q 2019.
$990.3 million

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
POMC deficiency obesity

Phase 3 Phase 3 initial data due 3Q 2019.
$990.3 million

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
Leptin Receptor Deficiency Obesity

Phase 3 Phase 3 initial data due 3Q 2019.
$990.3 million

TCON – TRACON Pharmaceuticals Inc.
TRC105 + Opdivo
Non-small cell lung cancer (NSCLC)

Phase 1/2 Phase 1 data to be presented at IASLC 2019 February 20-23, 2019.
$28.3 million

TCON – TRACON Pharmaceuticals Inc.
DE-122 and Lucentis - AVANTE
Wet age-related macular degeneration (Wet-AMD)

Phase 2 Phase 2 data due mid-2019.
$28.3 million