BPC July 12 update

Biotech stock clinical data catalysts slated for this quarter

Weekly watchlist

Last week we highlighted regulatory events slated for the current quarter, including notable PDUFA and Advisory Committee meeting dates. This week we focus on key clinical readouts, with an emphasis on small/mid cap late-stage catalysts.

First, let's review the week that was:

Sol-Gel Technologies, Ltd. (NASDAQ: SLGL) announced Monday that its Phase 3 trial evaluating Epsolay microencapsulated benzoyl peroxide cream, for the treatment of papulopustular rosacea, met both co-primary endpoints. Shares closed the week up 15% to $10.48.

Hookipa Pharma Inc. (NASDAQ: HOOK) shares closed Friday up 23% to $8.21 on news its Investigational New Drug (IND) Application for a Phase 1/2 clinical trial of HB-201, for the treatment of Human Papilloma Virus (HPV)-positive cancers has been cleared by the FDA. Hookipa intends to start the trial in 2H 2019.

Intra-Cellular Therapies, Inc. (Nasdaq:ITCI) announced Monday mixed data from its two Phase 3 trials 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. However, in its U.S. Study 401 trial neither of the 28mg nor 42mg doses met the primary endpoint. Shares closed the week down 17% to $11.19.

Novartis (NYSE: NVS) and Amgen Inc. (NASDAQ: AMGN) announced their decision to discontinue the Phase 2/3 trial of the BACE1 inhibitor CNP520 (umibecestat) for Alzheimer’s prevention. The decision followed a review of data that identified worsening in some measures of cognitive function.

Illumina, Inc. (NASDAQ: ILMN) shares closed Friday down 16% to $305.05 following the release of updated 2019 revenue guidance. The company noted it expects to report second quarter revenue of $835m, approximately $50m less than expected.

Amneal Pharmaceuticals, Inc. (NYSE: AMRX) shares also closed lower on a cut in profit forecasts, with expected earnings falling from initial expectations of around $600m-$650m for 2019 to $425m-$475m. Shares closed the week down 44% to $3.70.

Eton Pharmaceuticals, Inc (Nasdaq: ETON) shares closed the after-hours session on Friday down 17% to $6.91 on news that the FDA provided Eton’s partner with a Complete Response Letter relating to the application for EM-100.

Johnson & Johnson (NYSE: JNJ) shares closed down 4% to $134.30 on reports by Bloomberg that the U.S. Justice Department is pursuing a criminal investigation into whether the company lied to the public about the possible cancer risks of its talcum powder.

Biotech stock clinical data catalysts slated for this quarter:

Drug Stage Catalyst Market Cap

ACAD – ACADIA Pharmaceuticals Inc.
Pimavanserin - Enhance
Adjunctive treatment of schizophrenia

Phase 3 Phase 3 data due mid-2019.
$3.8 billion

ALKS – Alkermes plc
Diroximel fumarate (BIIB098) - Head-to-head trial versus Tecfidera -
Relapsing Remitting Multiple Sclerosis (RRMS)

Phase 3 Phase 3 top-line data due mid-2019.
$3.7 billion

ALLK – Allakos Inc.
AK002
Eosinophilic Gastritis

Phase 2 Phase 2 top-line data due mid-2019.
$1.5 billion

ALNY – Alnylam Pharmaceuticals Inc.
Inclisiran - ORION-9
Hypercholesterolemia

Phase 3 Phase 3 data 3Q 2019.
$7.8 billion

ATNX – Athenex Inc.
Oraxol
Metastatic breast cancer

Phase 3 Phase 3 top-line data due August 2019.
$1.5 billion

CHMA – Chiasma Inc.
Mycapssa - OPTIMAL
Acromegaly

Phase 3 Phase 3 data due mid-3Q 2019.
$201.1 million

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

Phase 3 Phase 3 data due mid-2019.
$833.7 million

ENTA – Enanta Pharmaceuticals Inc.
EDP-305 ARGON-1
Non-alcoholic steatohepatitis (NASH)

Phase 2 Phase 2 initial data due 3Q 2019.
$1.6 billion

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

Phase 3 Phase 3 data due 2H 2019 (possibly July/August)
$542.4 million

MRNS – Marinus Pharmaceuticals Inc.
Ganaxolone - Magnolia
Postpartum depression

Phase 2 Data due first half of 3Q 2019.
$229.1 million

MYOV – Myovant Sciences Ltd.
Relugolix - LIBERTY 1 and LIBERTY 2
Menstrual bleeding associated with uterine fibroids

Phase 3 Phase 3 LIBERTY 1 top-line data met primary endpoint - May 14, 2019. LIBERTY 2 data due 3Q 2019.
$784.2 million

RARE – Ultragenyx Pharmaceutical Inc.
DTX301
Ornithine Transcarbamylase (OTC) Deficiency

Phase 1/2 Phase 1/2 third cohort data due mid-2019.
$3.4 billion

RTRX – Retrophin Inc.
RE-024 fosmetpantotenate
Pantothenate kinase-associated neurodegeneration (PKAN)

Phase 3 Phase 3 top-line data due 3Q 2019.
$869.8 million

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
POMC deficiency obesity

Phase 3 Phase 3 initial data due 3Q 2019.
$713.7 million

SAGE – Sage Therapeutics Inc.
SAGE-217 - ARCHWAY
Bipolar depression

Phase 2 Phase 2 Part A data due July 2019.
$9.4 billion