BPC March 14 update

​Karyopharm KPTI PDUFA date 3-month extension - shares up 27% after hours

Price and Volume Movers

Karyopharm Therapeutics Inc. (Nasdaq: KPTI) announced the PDUFA date for its New Drug Application (NDA) for selinexor, for the treatment of patients with relapsed refractory multiple myeloma, has been extended by three months to July 6, 2019. Previously, an FDA Advisory Committee (ODAC) voted 8 to 5 recommending that the FDA wait for the results from its Phase 3 BOSTON trial, due late-2019/early-2020, before making a final decision regarding approval. Shares are trading up 28% to $5.62 after hours.

Evoke Pharma, Inc. (NASDAQ: EVOK) announced that it has submitted its response to the FDA multi-disciplinary review (DR) letter that was received on March 1, 2019 in association with the Gimoti New Drug Application (NDA), currently assigned a PDUFA date of April 1. The company plans to meet with the FDA on March 21, 2019 to obtain feedback on its response. Shares closed up 18% to $1.41.

Matinas BioPharma Holdings, Inc (NYSE AMER:MTNB) announced that it has commenced an underwritten public offering of its common stock. Shares are trading down 10% to $1.08 after hours.

Atossa Genetics Inc. (Nasdaq: ATOS) shares closed up 368% to $7.07 on news the FDA has permitted the use of oral Endoxifen as a post-mastectomy treatment in one breast cancer patient, under an expanded access program. The company noted in its headline that the FDA had issued approval but it should be noted that the expanded access for oral Endoxifen is restricted solely to this patient and the drug is still in IND (Investigational New Drug) phase.

CorMedix Inc. (NYSE American: CRMD) announced a one-for-five reverse stock split of its common stock, to become effective before trading opens on March 26, 2019.

Achieve Life Sciences, Inc. (Nasdaq: ACHV) shares are trading up 73% after hours to $4.23 following the release of 4Q earnings. The nano-cap company maintained previous guidance that Phase 2b data of cytisinicline in patients with smoking cessation, are slated for release in mid-2019. The company also noted data from a PK/PD trial of cytisinicline which saw after 26 days, subjects had an average 80% reduction in cigarettes smoked and 46% had stopped smoking.

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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:

Ritter Pharmaceuticals, Inc. (RTTR): $1.20; +41%.

Genprex, Inc. (GNPX): $2.15; +36%.

SCYNEXIS, Inc. (SCYX): $1.58; +25%.

Infinity Pharmaceuticals, Inc. (INFI): $1.91; +24%.

Tonix Pharmaceuticals Holding Corp. (TNXP): $3.17; +23%.

DECLINERS:

Axovant Sciences Ltd. (AXGT): $1.42; -19%.

Aquestive Therapeutics, Inc. (AQST): $6.37; -16%.

Seelos Therapeutics, Inc. (SEEL): $2.78; -16%.

Diffusion Pharmaceuticals Inc. (DFFN): $5.30; -12%.

Bio-Path Holdings, Inc. (BPTH): $27.47; -12%.

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ACHV – Achieve Life Sciences Inc.
Cytisine
Smoking cessation

Phase 2b Phase 2b data due mid-2019.
$19.7 million

ADMP – Adamis Pharmaceuticals Corporation
Higher Dose Naloxone Injection
Opioid overdose

PDUFA PDUFA date October 31, 2019.
$99.3 million

AGN – Allergan plc
Bimatoprost
Open-angle glaucoma or ocular hypertension

NDA Filing NDA filing due 2H 2019.
$48.8 billion

ALLK – Allakos Inc.
AK002
Eosinophilic Gastritis

Phase 2 Phase 2 top-line data due mid-2019.
$1.6 billion

ALLK – Allakos Inc.
AK002
Severe Allergic Conjunctivitis

Phase 1 Phase 1 data due 2Q 2019.
$1.6 billion

AVEO – AVEO Pharmaceuticals Inc.
TIVO-3 - tivozanib
Third line treatment of patients with renal cell cancer

Phase 3 Noted January 31, 2019 that FDA recommended against filing an NDA with current OS data. Additional interim OS analysis due August 2019 with further OS data 4Q 2019.
$79.2 million

AXSM – Axsome Therapeutics Inc.
AXS-05
Smoking cessation

Phase 2 Phase 2 top-line data due 2Q 2019.
$509.9 million

AXSM – Axsome Therapeutics Inc.
AXS-05 STRIDE-1
Treatment resistant depression

Phase 3 Phase 3 data due 2Q 2019.
$509.9 million

AXSM – Axsome Therapeutics Inc.
AXS-05 ADVANCE-1
Agitation in patients with Alzheimer’s disease (AD)

Phase 2/3 Phase 2/3 top-line data due 1H 2020.
$509.9 million

AXSM – Axsome Therapeutics Inc.
AXS-12
Narcolepsy

Phase 2 Phase 2 trial initiation announced January 30, 2019 with top-line data due 2Q 2019.
$509.9 million

AXSM – Axsome Therapeutics Inc.
AXS-07 - MOMENTUM
Migraine

Phase 3 Phase 3 top-line data due 1Q 2020.
$509.9 million

BCLI – Brainstorm Cell Therapeutics Inc.
NurOwn
Progressive Multiple Sclerosis

Phase 2 Phase 2 commencement of enrolment announced March 14, 2019.
$87.6 million

BHVN – Biohaven Pharmaceutical Holding Company Ltd.
TRORILUZOLE
Spinocerebellar Ataxia (SCA)

Phase 3 Phase 3 commencement of enrolment announced March 14, 2019.
$2.3 billion

BLPH – Bellerophon Therapeutics Inc.
INOpulse delivery device
Pulmonary hypertension associated with Sarcoidosis (PH-Sarcoidosis)

Phase 2 Phase 2 data due later in 2019.
$48.9 million

BLPH – Bellerophon Therapeutics Inc.
INOpulse delivery device
Pulmonary Hypertension associated with COPD (PH-COPD)

Phase 2 Phase 2 trial complete. Evaluating next steps for program.
$48.9 million

BLPH – Bellerophon Therapeutics Inc.
INOpulse delivery device
Pulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD)

Phase 2b Phase 2b top-line data cohort 2 and 3 data due 2H 2019.
$48.9 million

BTX – BioTime Inc.
OpRegen
Dry age-related macular degeneration (AMD)

Phase 1/2 Phase 1/2 enrolment to be completed 2019. Updated data due April 30, 2019 at Ophthalmology Annual Meeting.
$211.3 million

CATB – Catabasis Pharmaceuticals Inc.
Edasalonexent (CAT-1004)
Duchenne muscular dystrophy (DMD)

Phase 3 Phase 3 initiation announced September 25, 2018 with data due 2Q 2020.
$82.3 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

CLBS – Caladrius Biosciences Inc.
CLBS14-CMD
Coronary microvascular dysfunction (CMD)

Phase 2 Phase 2 data due by the end of 2019 or early 2020.
$36.5 million

CLSN – Celsion Corporation
ThermoDox - OPTIMA
Hepatocellular carcinoma - liver cancer

Phase 3 Phase 3 interim efficacy analysis 4Q 2019.
$37.5 million

CLSN – Celsion Corporation
GEN-1 OVATION 2
Ovarian cancer

Phase 1/2 Phase 1/2 data due 4Q 2019.
$37.5 million

CNST – Constellation Pharmaceuticals Inc.
CPI-0610 and ruxolitinib
Myelofibrosis

Phase 2 Phase 2 interim data 2Q 2019.
$311.3 million

CNST – Constellation Pharmaceuticals Inc.
CPI-1205 - ProSTAR
Castration-resistant prostate cancer

Phase 1/2 Phase 2 interim data due 2H 2019. Phase 1b data April 1, 2019 at AACR.
$311.3 million

DCPH – Deciphera Pharmaceuticals Inc.
Rebastinib and paclitaxel
Solid tumors

Phase 1/2 Phase 1b/2 initial data due 2019.
$902.8 million

DCPH – Deciphera Pharmaceuticals Inc.
DCC-2618 - INVICTUS
Gastrointestinal Stromal Tumors (GIST) - fourth-line

Phase 3 Phase 3 data due mid-2019.
$902.8 million

DCPH – Deciphera Pharmaceuticals Inc.
DCC-3014
Solid tumors or hematological malignancies

Phase 1 Phase 1 additional data due 2019.
$902.8 million

EARS – Auris Medical Holding Ltd.
AM-125
Vertigo

Phase 1 Phase 2 trial in planning process - noted March 14, 2019.
$13.2 million

EARS – Auris Medical Holding Ltd.
AM-201
Healthy volunteers

Phase 1b Phase 1b trial to be initiated March 2019.
$13.2 million

ETON – Eton Pharmaceuticals Inc.
DS-200
Nutritional deficiencies

NDA Filing Rolling NDA filed March 14, 2019.
$137.3 million

EVOK – Evoke Pharma Inc.
Gimoti - EVK-001
Female diabetic gastroparesis

PDUFA PDUFA date April 1, 2019. Company noted March 4, 2019 that the FDA listed various clinical and chemistry concerns regarding the NDA. Meeting with FDA March 21, 2019.
$24.9 million

HARP – Harpoon Therapeutics Inc.
HPN424
Metastatic castration-resistant prostate cancer

Phase 1 Phase 1 preliminary data due 2H 2019.
$243.4 million

HARP – Harpoon Therapeutics Inc.
HPN536
Ovarian cancer

Phase 1 Phase 1 trial to be initiated 1H 2019.
$243.4 million

HARP – Harpoon Therapeutics Inc.
HPN217
Multiple myeloma

Phase 1 Phase 1 trial to be initiated 2H 2019.
$243.4 million

INFI – Infinity Pharmaceuticals Inc.
IPI-549 + Nivolumab (MARIO-275)
Urothelial cancer

Phase 2 Phase 2 trial to be initiated 2Q 2019.
$99.6 million

INFI – Infinity Pharmaceuticals Inc.
IPI-549 + Tecentriq and Abraxane (MARIO-3)
Front-line triple negative breast cancer (TNBC)

Phase 2 Phase 2 trial to be initiated 2H 2019.
$99.6 million

INFI – Infinity Pharmaceuticals Inc.
IPI-549 + Tecentriq and Avastin
Front-line renal cell cancer (RCC)

Phase 2 Phase 2 trial to be initiated 2H 2019.
$99.6 million

KALV – KalVista Pharmaceuticals Inc.
KVD824

Phase 1 Phase 1 update due mid-2019.
$495.3 million

KALV – KalVista Pharmaceuticals Inc.
KVD001
Diabetic macular edema (DME)

Phase 2 Phase 2 completion due 2H 2019.
$495.3 million

KPTI – Karyopharm Therapeutics Inc.
Selinexor
Quadruple Refractory Multiple Myeloma

PDUFA priority review PDUFA date under priority review extended by three months to July 6, 2019. Advisory Committee February 26, 2019 voted 8-5 recommending that approval decision is held off until after the release of data from BOSTON trial, late-2019/2020.
$335.9 million

MLND – Millendo Therapeutics Inc.
Livoletide
Prader-Willi syndrome (PWS)

Phase 2/3 Phase 2b initiation announced March 19, 2019 with data due 1H 2020.
$183.1 million

MLND – Millendo Therapeutics Inc.
Nevanimibe
Congenital adrenal hyperplasia (CAH)

Phase 2b Phase 2b data due 1H 2020.
$183.1 million

PGNX – Progenics Pharmaceuticals Inc.
PyL
Prostate cancer

Phase 3 Phase 3 data due early 2020.
$361.8 million

PGNX – Progenics Pharmaceuticals Inc.
1095
Prostate Cancer

Phase 2 Phase 2 trial to be initiated 2Q 2019.
$361.8 million

RMTI – Rockwell Medical Inc.
IV Triferic
Anemia

NDA Filing NDA filing due 2Q 2019.
$330 million

SCYX – SCYNEXIS Inc.
SCY-078 - ibrexafungerp
Recuurent Vulvovaginal candidiasis (VVC)

Phase 3 Phase 3 trial to commence 1H 2019.
$77.2 million

SCYX – SCYNEXIS Inc.
SCY-078 - oral ibrexafungerp - VANISH
Vulvovaginal candidiasis (VVC)

Phase 3 Phase 3 data due 1H 2020.
$77.2 million

SLNO – Soleno Therapeutics Inc.
Diazoxide Choline Controlled-Release (DCCR) - DESTINY PWS
Prader-Willi syndrome (PWS)

Phase 3 Phase 3 data due late-2019.
$74.6 million

SPRO – Spero Therapeutics Inc.
SPR206
Healthy Volunteers

Phase 1 Phase 1 top-line data due 2H 2019.
$227 million

SPRO – Spero Therapeutics Inc.
SPR720
Healthy Volunteers

Phase 1 Phase 1 data due 2H 2019.
$227 million

TTPH – Tetraphase Pharmaceuticals Inc.
TP-6076

Phase 1 Phase 1 bronchopulmonary disposition trial to be completed 2H 2019.
$65 million

XFOR – X4 Pharmaceuticals Inc.
X4P-001
WHIM syndrome

Phase 2 Phase 3 trial to be initiated 1H 2019.
$132.7 million