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.
$227.2 million

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

Phase 1/2 Phase 1/2 data to be presented at SIMD April 6-9, 2019. Phase 3 trial to be initiated 2Q 2019 with data due 1Q 2021.
$227.2 million

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

Phase 3 Phase 3 data due 2H 2019.
$198.9 million

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

Phase 2b Phase 3 planned for 1H 2019.
$198.9 million

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

Phase 3 Phase 3 data due early 2019.
$198.9 million

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

Phase 3 Phase 3 initial data due in 2H 2019.
$198.9 million

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

Phase 2 Phase 2 data released January 22, 2019. Primary endpoint not met, secondary met.
$599.5 million

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

Phase 1/2 Phase 1 data presented February 6, 2019.
$423.4 million

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

Phase 1/2 Phase 1/2 trial planned for 2019.
$423.4 million

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

Phase 3 Phase 3 trial initiation announced August 9, 2018.
$7.4 billion

BGNE – BeiGene Ltd.
Tislelizumab
Urothelial carcinoma - Bladder cancer

Phase 1/2 Phase 1/2 data from ongoing trial presented at ESMO-IO December 14, 2018. 1/17 CRs 4/17 PRs.
$7.4 billion

BSTC – BioSpecifics Technologies Corp
XIAFLEX
Uterine fibroids

Phase 1 Phase 1 data released October 31, 2018 - primary endpoint of safety and tolerability met.
$466.5 million

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

Phase 3 Phase 3 trial to be completed in 2020.
$714.2 million

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

Phase 3 Phase 3 data due 4Q 2019.
$625.6 million

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

Phase 1 Phase 1 data due 1H 2019.
$230.8 million

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

Phase 2 Phase 2 trial noted clinically meaningful data but not statistically significant. Full data due 1H 2019.
$34.7 million

CHMA – Chiasma Inc.
Mycapssa - OPTIMAL
Acromegaly

Phase 3 Phase 3 data due 3Q 2019.
$146.2 million

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

Phase 2/3 Phase 2 interim analysis due 2020 with final data 2021.
$984.2 million

COCP – Cocrystal Pharma Inc.
CC-31244
Hepatitis C

Phase 2a Phase 2a initial data released January 22, 2019. SVR12 66%.
$81.1 million

COCP – Cocrystal Pharma Inc.
CC-42344
Influenza

Phase 1 Phase 1 trial to be initiated 1H 2019.
$81.1 million

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

Phase 1 NDA filing due 1Q 2019.
$

CORT – Corcept Therapeutics Incorporated
Relacorilant
Solid tumors - cancer

Phase 1/2 Phase 2 pancreatic data due 2Q 2019.
$1.3 billion

CORT – Corcept Therapeutics Incorporated
Relacorilant plus Abraxane
Ovarian cancer

Phase 2 Phase 2 trial initiated January 2019.
$1.3 billion

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

PDUFA PDUFA date for sNDA filing June 30, 2019.
$239.9 million

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

Phase 3 Phase 3 data due late-2Q 2019.
$565.9 million

HSGX – Histogenics Corporation
NeoCart
Cartilage defects in the knee

Phase 3 Development to be suspended - noted December 21, 2018.
$11.6 million

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

Phase 2 Phase 2 pivotal trial data due 2H 2019.
$347.7 million

KZR – Kezar Life Sciences Inc.
KZR-616
Lupus

Phase 1/2 Initial Phase 1b top-line data due 2Q 2019.
$338.6 million

LLY – Eli Lilly and Company
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.
$134.3 billion

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

Phase 2 Phase 2 data noted all patients met primary endpoint.
$632.9 million

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

Phase 1 Phase 1 trial to be completed 2019.
$1.3 billion

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

Phase 2 Phase 2 data due 2020.
$1.3 billion

MTEM – Molecular Templates Inc.
MT-3724
Diffuse large B-cell lymphoma (DLBCL)

Phase 2 Phase 2 interim data due 1H 2019.
$191.6 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.
$831 million

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

Phase 2 Phase 2 additional data released March 18, 2019 - PFS 7 months, high rates of diarrhea.
$1.5 billion

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

Phase 3 Phase 3 data released December 17, 2018. PFS endpoint met but OS co-primary missed. Data to be presented 1H 2019.
$1.5 billion

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 due 1H 2019.
$1.5 billion

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

Phase 2a Phase 2a data due 1H 2019.
$152.4 million

PIRS – Pieris Pharmaceuticals Inc.
PRS-060
Healthy subjects

Phase 1 Phase 1 initial data due at upcoming meeting.
$152.4 million

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

Phase 1/2 Phase 1 data due 2019.
$152.4 million

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

Phase 3 Phase 3 dosing of first patient announced October 15, 2018.
$77.2 million

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

Phase 3 Phase 3 open label interim data released January 30, 2019 - 11/17 partial or complete responses.
$77.2 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.
$77 million

SNNA – Sienna Biopharmaceuticals Inc.
SNA-001
Acne

Phase 3 Phase 3 data released July 30, 2018 did not meet primary endpoint.
$77 million

SNNA – Sienna Biopharmaceuticals Inc.
SNA-125
Atopic dermatitis

Phase 1/2 Phase 2 trial planned.
$77 million

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

Phase 3 Phase 3 data met non-inferiority primary endpoint - February 8, 2019. Seeking partner for further advancement.
$77 million

SNNA – Sienna Biopharmaceuticals Inc.
SNA-125
Psoriasis

Phase 1/2 Phase 1/2 data released August 27, 2018.
$77 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 top-line data due 4Q 2019.
$1.1 billion

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

Phase 3 Phase 3 trial to be initiated 1Q 2019.
$227 million

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

Phase 1/2 Phase 1/2 preliminary data due 1H 2019.
$136.3 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.
$136.3 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.
$136.3 million

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

Phase 3 Phase 3 data due late-2Q 2019.
$282.7 million

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

Phase 3 Phase 3 top-line data due 2Q 2020.
$282.7 million

SVRA – Savara Inc.
Molgradex - OPTIMA
Nontuberculous mycobacteria (NTM)

Phase 2a Phase 2 top-line data due 1H 2019.
$282.7 million

TORC – resTORbio Inc.
RTB101 and Everolimus
Respiratory Tract Infections

Phase 3 Phase 3 enrolment to commence 2Q 2019 with top-line data due mid-2020.
$227.4 million

TORC – resTORbio Inc.
RTB101
Parkinson's disease

Phase 2 Phase 2 trial to be initiated 1Q 2019.
$227.4 million

UBX – Unity Biotechnology Inc.
UBX0101
Osteoarthritis

Phase 1 Phase 1 data due 2Q 2019.
$334.3 million

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

Phase 2 Phase 2 additional NAFLD data to be presented at EASL April 11-14, 2019 with Phase 2b trial to be initiated 2H 2019.
$583.1 million

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.
$1.4 billion

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

Phase 1/2 Phase 1b/2a top line data 1H 2019.
$1.4 billion