BPC January 08 update

​Axsome AXSM shares add a further 29% - two-day gain 238%

Price and Volume Movers

Axsome Therapeutics, Inc. (NASDAQ: AXSM) shares continued their surge closing up a further 29% to $8.88 for a two-day cumulative gain of 238%. The company announced Monday that it met the primary endpoint in its Phase 2 trial of AXS-05 for the treatment of major depressive disorder (MDD).

UroGen Pharma Ltd. (Nasdaq:URGN) announced top-line data from its Phase 3 OLYMPUS clinical trial of UGN-101 for the treatment of low-grade upper tract urothelial cancer (UTUC). The data showed a complete response (CR) rate of 57%. All patients exhibiting a CR remained disease free at six months. Shares closed down 6% to $51.35.

Novartis (NYSE: NVS) announced that the FDA has granted crizanlizumab (SEG101) Breakthrough Therapy designation for the prevention of vaso-occlusive crises (VOCs) in with sickle cell disease (SCD). An FDA filing of crizanlizumab is due 1H 2019.

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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:

vTv Therapeutics Inc. (VTVT): $3.12; +32%.

Checkpoint Therapeutics, Inc. (CKPT): $2.72; +16%.

BioXcel Therapeutics, Inc. (BTAI): $6.09; +14%.

Aerpio Pharmaceuticals, Inc. (ARPO): $2.40; +13%.

Constellation Pharmaceuticals, Inc. (CNST): $6.83; +13%.

DECLINERS:

AVROBIO, Inc. (AVRO): $13.04; -17%.

Achaogen, Inc. (AKAO): $1.48; -12%.

Gritstone Oncology, Inc. (GRTS): $16.44; -11%.

Leap Therapeutics, Inc. (LPTX): $2.70; -10%.

Dova Pharmaceuticals, Inc. (DOVA): $7.90; -10%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

DNLI – Denali Therapeutics Inc.
DNL747
Amyotrophic Lateral Sclerosis

Phase 1b Phase 1b commencement of dosing announced January 8, 2019. Data due 4Q 2019.
$2 billion

FCSC – Fibrocell Science Inc.
FCX-007
Recessive dystrophic epidermolysis bullosa

Phase 1/2 Phase 2 interim data due 1Q 2019 with Phase 3 trial to commence 1H 2019.
$18.4 million

FTSV – Forty Seven Inc.
5F9 + rituximab
Non-Hodgkin Lymphoma (NHL) - cancer

Phase 1/2 Phase 1b/2 data due mid-2019.
$424.7 million

FTSV – Forty Seven Inc.
5F9 + cetuximab
Colorectal cancer

Phase 1/2 Phase 1b/2 data due 4Q 2019.
$424.7 million

FTSV – Forty Seven Inc.
5F9 + Azacitidine
Acute myeloid leukemia (AML)

Phase 1 Phase 1b/2 data due mid-2019.
$424.7 million

FTSV – Forty Seven Inc.
5F9 + Avelumab
Ovarian cancer

Phase 1 Phase 1b data due 4Q 2019.
$424.7 million

HTBX – Heat Biologics Inc.
HS-110 and nivolumab (Opdivo)/Keytruda
Non-small cell lung cancer (NSCLC)

Phase 2 Phase 2 interim data to be presented at 2019 ASCO-SITC February 28, 2019 1pm PST. First-line maintenance treatment with Keytruda announced January 14, 2019.
$43.2 million

MRNA – Moderna Inc.
mRNA-4157 - KEYNOTE-603
Personalized cancer vaccine

Phase 1 Phase 2 trial planned.
$4.9 billion

MRNA – Moderna Inc.
mRNA-3704
Methylmalonic Acidemia (MMA)

Phase 1/2 Phase 1/2 trial planned.
$4.9 billion

MRNA – Moderna Inc.
mRNA-2752
Solid tumors, lymphoma

Phase 1 Phase 1 dosing has commenced - noted January 8, 2019.
$4.9 billion

NVS – Novartis AG
Crizanlizumab
Sickle cell disease

NDA Filing Regulatory filing due 1H 2019.
$203.6 billion

NVUS – Novus Therapeutics Inc.
OP0201
Acute Otitis Media

Phase 1 Phase 1 commencement of dosing announced January 8, 2019.
$27.1 million

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

Phase 3 Phase 3 data due 2020.
$990.3 million

SLDB – Solid Biosciences Inc.
SGT-001
Duchenne Muscular Dystrophy

Phase 1/2 Phase 1/2 initial data due 1Q 2019. Interim analysis 2H 2019.
$812.5 million

URGN – UroGen Pharma Ltd.
MitoGel - UGN-101
Urothelial Carcinoma

Phase 3 Phase 3 data released January 8, 2019 noted 57% CR. Initiation of rolling NDA announced December 17, 2018.
$739.6 million