BPC July 08 update

Phase 3 data released by ​Sol-Gel SLGL +13% and Intra-Cellular ITCI -13%

Price and Volume Movers

Sol-Gel Technologies, Ltd. (NASDAQ: SLGL) announced positive data from its Phase 3 program evaluating Epsolay microencapsulated benzoyl peroxide cream, for the treatment of papulopustular rosacea, demonstrating statistically significant improvement in both co-primary endpoints of (1) the number of patients achieving “clear” or “almost clear” in the Investigator Global Assessment (IGA) and (2) absolute mean reduction from baseline in inflammatory lesion count. Shares closed up 13% to $10.30.

Adverum Biotechnologies, Inc. (NASDAQ: ADVM) shares closed up 23% to $14.90. The company announced interim 24-week data from its Phase 1 OPTIC trial of ADVM-022 for Neovascular (Wet) Age-Related Macular Degeneration, will be presented September 12, 2019 at the 52nd Annual Retina Society Meeting.

Intra-Cellular Therapies, Inc. (Nasdaq:ITCI) reported mixed data from its two Phase 3 trials (Study 401 and Study 404) evaluating lumateperone as monotherapy in the treatment of major depressive episodes associated with Bipolar I or Bipolar II disorder. In the global Study 404, lumateperone 42 mg met the primary endpoint for improvement in depression as measured by change from baseline versus placebo on the Montgomery-Åsberg Depression Rating Scale (MADRS) total score. In the intent-to-treat (ITT) study population, the least squares (LS) mean reduction from baseline for lumateperone was 16.7 points, versus 12.1 points for placebo (LS mean difference = 4.6 points; effect size = 0.56, p<0.001). However, in its U.S. Study 401 trial neither of the 28mg nor 42 mg doses met the primary endpoint. Lumateperone 42 mg and 28 mg demonstrated a LS mean reduction from baseline on the MADRS total score of 20.7 points and 18.9 points, respectively, versus 19.7 points on placebo. Shares closed down 13% to $11.70.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced that it plans to submit marketing applications to both the FDA and the European Medicines Agency (EMA) in 4Q 2019 for its gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A.

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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:

Oasmia Pharmaceuticals (OASM): $2.28; +13%.

Eiger BioPharmaceuticals, Inc. (EIGR): $11.41; +13%.

XOMA Corporation (XOMA): $16.44; +9%.

Soleno Therapeutics, Inc. (SLNO): $2.75; +8%.

MeiraGTx Holdings plc (MGTX): $28.92; +7%.

DECLINERS:

Xenetic Biosciences, Inc. (XBIO): $10.20; -17%.

Jaguar Health, Inc. (JAGX): $3.61; -14%.

AzurRx BioPharma, Inc. (AZRX): $1.25; -14%.

Coherus BioSciences, Inc. (CHRS): $19.71; -13%.

Melinta Therapeutics, Inc. (MLNT): $6.28; -12%.

Pipeline updates:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ADVM – Adverum Biotechnologies Inc.
ADVM-022
Wet age-related macular degeneration (Wet-AMD)

Phase 1 Phase 1 third cohort to commence enrolment in 4Q 2019. Cohort 1 52-week data and Cohort 2 24-week data due 1H 2020.
$446.1 million

AZRX – AzurRx BioPharma Inc.
MS1819-SD
Cystic fibrosis

Phase 2 Phase 2 higher dose trial to be initiated early 2020.
$18.8 million

AZRX – AzurRx BioPharma Inc.
MS1819-SD
Cystic Fibrosis Patients with Severe Exocrine Pancreatic Insufficiency

Phase 2 Phase 2 initial data due early 2020.
$18.8 million

BLCM – Bellicum Pharmaceuticals Inc.
BPX-501 Rivo-cel
Adjunct T-cell therapy administered after allogeneic HSCT - (hematopoietic stem cell transplantation) - cancer

Phase 1/2 Phase 2 pediatric top-line data met primary endpoint of event free survival - July 8, 2019.
$37 million

BMRN – BioMarin Pharmaceutical Inc.
Valoctocogene roxaparvovec (BMN 270)
Hemophilia A

BLA Filing BLA filing due 4Q 2019.
$12.4 billion

ENTA – Enanta Pharmaceuticals Inc.
EDP-514
Hepatitis B virus (HBV)

Phase 1 Phase 1 trial initiation announced July 8, 2019.
$1.2 billion

INO – Inovio Pharmaceuticals Inc.
VGX-3100
HPV-related pre-cancers - Vulvar Intraepithelial Neoplasia (VIN)

Phase 2 Phase 2 interim data due 4Q 2019.
$222.8 million

ITCI – Intra-Cellular Therapies Inc.
ITI-007 - Monotherapy (Study 401)
Bipolar depression

Phase 3 Phase 3 trial did not meet primary endpoint - July 8, 2019.
$456.8 million

ITCI – Intra-Cellular Therapies Inc.
ITI-007 - Monotherapy (Study 404)
Bipolar depression

Phase 3 Phase 3 trial met primary endpoint - July 8, 2019.
$456.8 million

OBSV – ObsEva SA
OBE2109 - PRIMROSE 1
Uterine fibroids

Phase 3 Phase 3 data due 1H 2020.
$324.8 million

OBSV – ObsEva SA
OBE2109 - PRIMROSE 2
Uterine fibroids

Phase 3 Phase 3 data due 4Q 2019.
$324.8 million

SLGL – Sol-Gel Technologies Ltd.
VERED (Epsolay)
Papulopustular rosacea

Phase 3 Phase 3 top-line data met primary endpoints - July 8, 2019.
$162.5 million

SNSS – Sunesis Pharmaceuticals Inc.
Vecabrutinib (SNS-062)
Advanced B-Cell Malignancies

Phase 1/2 Phase 1/2 next update due at ASH December 2019. Noted 300 mg Cohort is now enrolling - July 8, 2019.
$63.5 million

ZGNX – Zogenix Inc.
ZX008 - (Study 1601)
Lennox-Gastaut syndrome

Phase 3 Phase 3 data due 1Q 2020.
$1.9 billion