BPC August 09 update

La Jolla LJPC earnings miss -25%; Earnings pipeline wrap

Price and Volume Movers

Citius Pharmaceuticals, Inc. (NASDAQ: CTXR) announced the pricing of an underwritten offering of 7,843,138 shares of its common stock and common warrants to purchase 7,843,138 shares. Shares of the nano-cap closed up 48% to $1.69 on 68x average volume after trading up as high as $3.14 earlier on the day.

La Jolla Pharmaceutical Company (Nasdaq: LJPC) shares closed down 25% to $24.46 following a disappointing 2Q earnings report where the company noted a net loss of $52.8m for the quarter, or $2.02 per share, larger than investors had expected.

Rigel Pharmaceuticals, Inc. (Nasdaq:RIGL) shares closed up 13% to $3.02 following its report of 2Q earnings. The company reported net product sales from Tavalisse of $1.8m, launched on May 29 for patients with chronic immune thrombocytopenia (ITP).

 

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

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

ADVANCERS:

Checkpoint Therapeutics, Inc. (CKPT): $3.17; +28%.

Idera Pharmaceuticals, Inc. (IDRA): $7.31; +27%.

Novavax, Inc. (NVAX): $1.47; +21%.

NantKwest, Inc. (NK): $3.27; +16%.

Acer Therapeutics Inc. (ACER): $29.82; +16%.

DECLINERS:

Portola Pharmaceuticals, Inc. (PTLA): $30.81; -18%.

Akebia Therapeutics, Inc. (AKBA): $8.22; -15%.

Spring Bank Pharmaceuticals, Inc. (SBPH): $12.30; -13%.

TRACON Pharmaceuticals, Inc. (TCON): $2.15; -12%.

Calithera Biosciences, Inc. (CALA): $4.53; -11%.


Pipeline Database Updates

Drug Stage Catalyst Market Cap

AGLE – Aeglea BioTherapeutics Inc.
Pegzilarginase (AEB1102)
Solid tumors

Phase 1 Phase 1 data presented at ESMO October 2018 noted 1/28 PRs and 8/23 SD.
$179.7 million

AGLE – Aeglea BioTherapeutics Inc.
Pegzilarginase (AEB1102)
Arginase I deficiency

Phase 1/2 Phase 1/2 repeat dose data due 1H 2019. Phase 3 trial to be initiated 2Q 2019 with data due 1Q 2021.
$179.7 million

ALDX – Aldeyra Therapeutics Inc.
ADX-102
Noninfectious anterior uveitis

Phase 3 Phase 3 data due 2H 2019.
$248.3 million

ALDX – Aldeyra Therapeutics Inc.
ADX-102
Dry eye syndrome

Phase 2b Phase 3 planned for 2019.
$248.3 million

ALDX – Aldeyra Therapeutics Inc.
Reproxalap (ADX-102)
Allergic conjunctivitis

Phase 3 Phase 3 data due early 2019.
$248.3 million

ALDX – Aldeyra Therapeutics Inc.
ADX-102
Sjögren-Larsson Syndrome (SLS)

Phase 3 Phase 3 initial data due in 2H 2019.
$248.3 million

AUPH – Aurinia Pharmaceuticals Inc
Voclosporin Ophthalmic Solution (VOS)
Dry eye syndrome

Phase 2 Phase 2 data due January 2019.
$512.9 million

AVRO – AVROBIO Inc.
AVR-RD-01
Fabry disease

Phase 1/2 Phase 2 interim update due 1Q 2019.
$584 million

AVRO – AVROBIO Inc.
AVR-RD-02
Gaucher disease

Phase 1/2 Phase 1/2 trial planned for 2019.
$584 million

BGNE – BeiGene Ltd.
Tislelizumab
Urothelial carcinoma - Bladder cancer

Phase 1/2 Phase 1/2 data from ongoing trial to be presented at ESMO-IO December 14, 2018.
$7.9 billion

BGNE – BeiGene Ltd.
Tislelizumab
Squamous non-small cell lung cancer (NSCLC)

Phase 3 Phase 3 trial initiation announced August 9, 2018.
$7.9 billion

BSTC – BioSpecifics Technologies Corp
XIAFLEX
Uterine fibroids

Phase 1 Phase 1 data released October 31, 2018 - primary endpoint of safety and tolerability met.
$445.9 million

CBAY – CymaBay Therapeutics Inc.
Seladelpar
Primary biliary cholangitis (PBC)

Phase 3 Phase 3 trial to be completed in 2020.
$514.7 million

CCXI – ChemoCentryx Inc.
CCX168 (ADVOCATE)
Associated vasculitis (AAV)

Phase 3 Phase 3 data due 4Q 2019.
$506.8 million

CERC – Cerecor Inc.
CERC-301
Neurogenic Orthostatic Hypotension (nOH)

Phase 1 Phase 1 data due 1H 2019.
$135.5 million

CFRX – ContraFect Corporation
CF-301
Serious infections caused byStaph aureus including MRSA

Phase 2 Phase 2 data due 4Q 2018.
$165.2 million

CHMA – Chiasma Inc.
Mycapssa - OPTIMAL
Acromegaly

Phase 3 Phase 3 completion of enrollment announced October 1, 2018 with data due September 2019.
$90.6 million

CMTA – Clementia Pharmaceuticals Inc.
Palovarotene
Multiple Osteochondroma (MO)

Phase 2/3 Phase 2 interim analysis due 2020 with final data 2021.
$431.1 million

COCP – Cocrystal Pharma Inc.
CC-31244
Hepatitis C

Phase 2a Phase 2a initial data due December 2018.
$93.4 million

COCP – Cocrystal Pharma Inc.
CC-42344
Influenza

Phase 1 Phase 1 trial to be initiated 1H 2019.
$93.4 million

CORI – Corium International Inc.
Corplex Donepezil
Alzheimer's disease

Phase 1 NDA filing due 1Q 2019.
$

CORT – Corcept Therapeutics Incorporated
Relacorilant plus Abraxane
Ovarian cancer

Phase 2 Phase 2 trial to commence later in 2018.
$1.9 billion

CORT – Corcept Therapeutics Incorporated
Relacorilant
Solid tumors - cancer

Phase 1/2 Phase 2 pancreatic data due by end of 2018.
$1.9 billion

DOVA – Dova Pharmaceuticals Inc.
Avatrombopag
Immune Thrombocytopenic Purpura (ITP)

PDUFA PDUFA date for sNDA filing June 30, 2019.
$417.7 million

GLYC – GlycoMimetics Inc.
GMI-1070 (rivipansel)
Vaso-occlusive crisis of sickle cell disease

Phase 3 Phase 3 data due 2Q 2019.
$445.6 million

HSGX – Histogenics Corporation
NeoCart
Cartilage defects in the knee

Phase 3 Phase 3 data released September 5, 2018. Primary endpoint not met. Update regarding possibility of filing BLA expected by end 2018 or early 2019.
$34 million

KDMN – Kadmon Holdings Inc.
KD025 - ROCKstar (KD025-213)
Chronic graft-versus-host disease (cGVHD)

Phase 2 Phase 2 pivotal trial initiation announced October 25, 2018.
$279.4 million

KZR – Kezar Life Sciences Inc.
KZR-616
Lupus

Phase 1/2 Initial Phase 1b top-line data due 1H 2019.
$572.9 million

LOXO – Loxo Oncology Inc.
LOXO-292
RET-fusion non-small cell lung cancer (NSCLC), medullary thyroid cancer (MTC) and other tumors

Phase 1 Phase 1/2 updated data noted ORR of 59% - October 6, 2018.
$4.3 billion

LOXO – Loxo Oncology Inc.
LOXO-195
NTRK fusion cancers

Phase 1/2 Phase 1 updated data due 1H 2019.
$4.3 billion

MGTA – Magenta Therapeutics Inc.
MGTA-456
Inherited Metabolic Disorders (IMD)

Phase 2 Phase 2 data noted all patients met primary endpoint.
$229.4 million

MNTA – Momenta Pharmaceuticals Inc.
M230 (CSL730)
Healthy volunteers

Phase 1 Phase 1 trial to be completed 2019.
$895.3 million

MNTA – Momenta Pharmaceuticals Inc.
M281
generalized myasthenia gravis (gMG)

Phase 2 Phase 2 trial to be initiated 4Q 2018.
$895.3 million

MTEM – Molecular Templates Inc.
MT-3724

Phase 1b Phase 2 trial to commence 1Q 2019.
$166.4 million

OMER – Omeros Corporation
OMS721
IgA nephropathy

Phase 3 Phase 3 trial enrollment initiated February 2018. Phase 2 data released October 1, 2018 showed only marginal improvement compared with placebo.
$651.3 million

PBYI – Puma Biotechnology Inc
Neratinib SUMMIT
HER2 non-amplified breast cancer that has a HER2 mutation

Phase 2 Phase 2 data released December 6, 2018 noted ORR 30%; 9% CR.
$861.1 million

PBYI – Puma Biotechnology Inc
Neratinib monotherapy with high dose loperamide prophylaxis - CONTROL
Extended adjuvant treatment of early stage HER2-positive breast cancer

Phase 2 Phase 2 additional data December 6, 2018. Noted grade 3 diarrhea occurred in 40% of patients compared with 2% for placebo.
$861.1 million

PBYI – Puma Biotechnology Inc
Neratinib
Third-line HER2-positive metastatic breast cancer

Phase 3 Phase 3 data due 4Q 2018 or 1H 2019.
$861.1 million

PIRS – Pieris Pharmaceuticals Inc.
PRS-343
HER2-positive solid tumors

Phase 1/2 Phase 1 data due 1H 2019.
$158.1 million

PIRS – Pieris Pharmaceuticals Inc.
PRS-080
Anemia of chronic disease

Phase 2a Phase 2a data due 1H 2019.
$158.1 million

PIRS – Pieris Pharmaceuticals Inc.
PRS-060
Healthy subjects

Phase 1 Phase 1 initial data due at upcoming meeting.
$158.1 million

SCYX – SCYNEXIS Inc.
SCY-078 - oral - CARES
Candida Auris

Phase 3 Phase 3 dosing of first patient announced October 15, 2018.
$23.5 million

SCYX – SCYNEXIS Inc.
SCY-078 - oral (FURI)
Invasive candidiasis

Phase 3 Phase 3 open label trial commenced 3Q 2017. Preliminary data review 4Q 2018.
$23.5 million

SNNA – Sienna Biopharmaceuticals Inc.
SNA-001
Reduction of light-pigmented hair

Phase 3 Phase 3 data due late January/early February 2019.
$73.6 million

SNNA – Sienna Biopharmaceuticals Inc.
SNA-120
Pruritus associated with psoriasis

Phase 2b Phase 2b trial did not meet primary endpoint. Phase 3 trial to be initiated 2H 2019.
$73.6 million

SNNA – Sienna Biopharmaceuticals Inc.
SNA-001
Acne

Phase 3 Phase 3 data released July 30, 2018 did not meet primary endpoint. Data from separate trial due late January/early February 2019.
$73.6 million

SNNA – Sienna Biopharmaceuticals Inc.
SNA-125
Atopic dermatitis

Phase 1/2 Phase 1/2 commencement of dosing announced March 12, 2018 with top-line data due 4Q 2018.
$73.6 million

SNNA – Sienna Biopharmaceuticals Inc.
SNA-125
Psoriasis

Phase 1/2 Phase 1/2 data released August 27, 2018.
$73.6 million

SPPI – Spectrum Pharmaceuticals Inc.
Poziotinib
Non-small cell lung cancer (NSCLC) with exon 20 insertion mutation in EGFR or HER2

Phase 2 Phase 2 abstract noted ORR 58%; PFS 5.6 months.
$1.2 billion

SPRO – Spero Therapeutics Inc.
SPR994
Complicated urinary tract infections (cUTI)

Phase 1 Phase 3 trial to be initiated year-end 2018.
$115.3 million

SRRA – Sierra Oncology Inc.
SRA737 and niraparib
Castration-resistant prostate cancer (mCRPC)

Phase 1/2 Phase 1b/2 trial to be initiated 4Q 2018.
$104.1 million

SRRA – Sierra Oncology Inc.
SRA737 + Low-Dose Gemcitabine
Solid Tumors

Phase 1/2 Phase 1/2 preliminary data due 1H 2019.
$104.1 million

SRRA – Sierra Oncology Inc.
SRA737
Advanced Solid Tumors or Non-Hodgkin's Lymphoma (NHL)

Phase 1/2 Phase 1/2 preliminary data due 1H 2019.
$104.1 million

SVRA – Savara Inc.
Molgradex
Nontuberculous mycobacteria (NTM)

Phase 2a Phase 2a interim data due 4Q 2018 top-line data due 2Q 2019.
$261.4 million

SVRA – Savara Inc.
AeroVanc - AVAIL
MRSA in cystic fibrosis

Phase 3 Phase 3 data due 2Q 2019.
$261.4 million

SVRA – Savara Inc.
Molgradex - IMPALA
Autoimmune pulmonary alveolar proteinosis (PAP)

Phase 3 Phase 3 data due 2Q 2019.
$261.4 million

TBIO – Translate Bio Inc.
MRT5005
Cystic fibrosis

Phase 1/2 Phase 1/2 trial to be initiated year-end 2018.
$369.7 million

TORC – resTORbio Inc.
RTB101 and Everolimus
Respiratory Tract Infections

Phase 2b Phase 2b data released July 25, 2018. Mixed data. One daily treatment met primary endpoint. Twice daily and combo failed. Phase 3 trial to be initiated 1H 2019.
$345.6 million

TORC – resTORbio Inc.
RTB101
Parkinson's disease

Phase 2 Phase 2 trial to be initiated 1Q 2019.
$345.6 million

UBX – Unity Biotechnology Inc.
UBX0101
Osteoarthritis

Phase 1 Phase 1 data due 2Q 2019.
$673.4 million

VKTX – Viking Therapeutics Inc.
VK2809
Glycogen Storage Disease

Phase 1 Phase 1 trial to commence 3Q 2018.
$642.4 million

VKTX – Viking Therapeutics Inc.
VK2809
Hypercholesterolemia and Non-Alcoholic Fatty Liver Disease

Phase 2 Phase 2 data released September 18, 2018 met primary and secondary endpoints. Data presented at AASLD November 12, 2018 noted 67% of patients saw >50% liver fat content.
$642.4 million

WVE – Wave Life Sciences Ltd.
WVE-120102
Huntington’s disease

Phase 1/2 Phase 1b/2a top line data 1H 2019.
$1.5 billion

WVE – Wave Life Sciences Ltd.
WVE-210201
Duchenne muscular dystrophy - Exon 51

Phase 1 Phase 1 interim efficacy data from open-label trial due 2H 2019. Phase 2/3 trial to be initiated 2019.
$1.5 billion