BPC August 28 update

Phase 3 biotech stock catalysts to watch in September; Biotech week in Review

Weekly watchlist

This week we preview Phase 3 clinical readouts/analyses slated for September. Companies frequently provide timeline guidance by quarter (e.g 3Q 2020) which does make it challenging to nail down exactly which month a catalyst is due. However, with just month remaining in the third quarter we can safely say that all such catalysts will read out in September.

Next week we will focus on Phase 2 readouts.

First, let’s review the week that was with notable news. After starting the quarter full of positive news the last two weeks have seen catalysts predominantly producing downward pricing moves.

CLINICAL DATA

UroGen Pharma Ltd. (NASDAQ:URGN) shares closed Thursday down 11% to $19.86 following news its Phase 2 APOLLO clinical trial of a RTGel hydrogel formulation in combination with Botox (onabotulinumtoxinA) in patients with overactive bladder (OAB) and urinary incontinence did not meet the primary endpoint of improvement of overactive bladder symptoms.

Outlook Therapeutics, Inc. (NASDAQ:OTLK) shares closed Wednesday down 36% to $0.82 following news data from its Phase 3 NORSE 1 trial of ONS-5010, for the treatment of wet age-related macular degeneration (wet AMD), showed no statistical improvement when compared with Lucentis (ranibizumab).

Onconova Therapeutics, Inc. (NASDAQ:ONTX) announced Monday that its Phase 3 INSPIRE trial of IV rigosertib in higher-risk MDS (HR-MDS) patients, did not meet its primary endpoint of improved survival. Shares plunged to close down 66% to $0.34.

Odonate Therapeutics, Inc. (NASDAQ:ODT) shares slid to close Monday down 45% to $18.33 following the release of data from its Phase 3 CONTESSA trial of tesetaxel in patients with metastatic breast cancer (MBC). While the trial met the primary endpoint of improved Progression Free Survival (PFS) with median PFS of 9.8 months for tesetaxel plus a reduced dose of capecitabine vs 6.9 months for the approved dose of capecitabine alone, the improvement of 2.9 months was below expectations. The data also showed side effects, with cases of neutropenia, a cause for a previous clinical hold, recorded at 71.2% for tesetaxel plus capecitabine vs 8.3% for capecitabine alone.

REGULATORY

Lipocine Inc. (NASDAQ: LPCN) announced Friday that the FDA needs additional time to complete its review of the New Drug Application for Tlando for men with hypogonadism (low testosterone) and will not meet the assigned PDUFA date of August 28, 2020. A new PDUFA date was not provided but it expects the FDA to issue a decision in the coming weeks.

Tricida, Inc. (NASDAQ:TCDA) announced Monday it received a Complete Response Letter (CRL) from the FDA for its NDA for veverimer. Shares fell by 24% to $10.11 before closing the week at $10.18.

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Key Phase 3 biotech stock catalysts to watch in September:

Drug Stage Catalyst Market Cap

ACST – Acasti Pharma Inc.
CaPre (TRILOGY 1 and 2)
Hypertriglyceridemia

Phase 3 Phase 3 TRILOGY 1 data did not meet primary endpoint - January 13, 2020. TRILOGY 2 data August 31, 2020 also did not meet primary endpoint.
$21 million

ALBO – Albireo Pharma Inc.
Odevixibat (A4250) - PEDFIC- 1
Progressive familial intrahepatic cholestasis (PFIC)

Phase 3 Phase 3 top-line data met primary endpoints - September 8, 2020.
$738.7 million

ATRA – Atara Biotherapeutics Inc.
ATA 129 (tab-cel) - ALLELE
Epstein-Barr virus (EBV-PTLD) after solid organ transplant (SOT)

Phase 3 Phase 3 interim analysis due 3Q 2020.
$1.2 billion

CRBP – Corbus Pharmaceuticals Holdings Inc.
Resunab - RESOLVE-1
Systemic Sclerosis

Phase 3 Phase 3 top-line data did not meet endpoints - September 8, 2020.
$155.3 million

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

Phase 3 Phase 3 data failed to meet endpoints - August 2, 2019. Biomarker data to be presented at FSCDR meeting September 23-25, 2020,
$172.3 million

ITCI – Intra-Cellular Therapies Inc.
Lumateperone ITI-007-402
Bipolar depression

Phase 3 Phase 3 top-line data released September 9, 2020. High dose met primary endpoint but low dose missed.
$2.5 billion

MESO – Mesoblast Limited
Remestemcel-L
COVID-19

Phase 3 Phase 3 interim analysis recommended trial to continue - September 4, 2020. Enrollment to be completed 4Q 2020.
$2.1 billion

MRNS – Marinus Pharmaceuticals Inc.
Ganaxolone - Marigold
CDKL5 Deficiency Disorder (CDD)

Phase 3 Phase 3 trial met primary endpoint - September 14, 2020.
$422.8 million

MYOV – Myovant Sciences Ltd.
Relugolix
Advanced prostate cancer

Phase 3 PDUFA date under priority review December 20, 2020. Castration Resistance Free Survival data due 3Q 2020.
$2 billion

REGN – Regeneron Pharmaceuticals Inc.
REGN-COV2
COVID-19 (Anti-viral antibody)

Phase 2/3 Phase 2/3 trial has been initiated. Initial virology and biomarker data due September 2020.
$59.1 billion

SBBP – Strongbridge Biopharma plc
COR-003 (levoketoconazole) - LOGICS
Endogenous Cushing’s syndrome

Phase 3 Phase 3 top-line data met primary endpoint - September 8, 2020.
$120.1 million

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

Phase 3 Phase 3 data released September 8, 2020 showed non-inferiority versus intravenous ertapenem.
$262.2 million

TNXP – Tonix Pharmaceuticals Holding Corp.
TNX-102 SL (Tonmya)
Fibromyalgia

Phase 3 Phase 3 interim analysis due September 2020. Top-line data due 4Q 2020. Initiation of second Phase 3 trial announced September 3, 2020.
$120.8 million