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 did not meet primary endpoint - July 22, 2019.
$6.6 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 met primary endpoint - July 30, 2019.
$3.3 billion

ALLK – Allakos Inc.
AK002
Eosinophilic Gastritis

Phase 2 Phase 2 top-line data met primary and secondary endpoints - August 5, 2019. Extension data due 1H 2020. Phase 3 trial to commence 1Q 2020.
$3 billion

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

Phase 3 Phase 3 data met all endpoints - September 25, 2019. Data November 18, 2019 noted LDL-C reductions of 50%.
$13.2 billion

ATNX – Athenex Inc.
Oraxol
Metastatic breast cancer

NDA Filing Phase 3 data noted PFS (p=0.077) 9.3 months vs 8.3 months. OS (p=0.035) 27.9 months vs 16.9 months. ORR 40.4% vs 25.6%. Meeting with FDA April 2020 with NDA to be filed shortly after.
$945.6 million

BMY – Bristol-Myers Squibb Company
CM-227 – Opdivo + Yervoy
First-line Non-small cell lung cancer (NSCLC)

PDUFA priority review PDUFA date under priority review May 15, 2020.
$133.3 billion

CHMA – Chiasma Inc.
Mycapssa - OPTIMAL
Acromegaly

PDUFA PDUFA date June 26, 2020.
$187 million

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

PDUFA priority review PDUFA date under priority review August 13, 2020.
$2.7 billion

ENTA – Enanta Pharmaceuticals Inc.
EDP-305 ARGON-2
Non-alcoholic steatohepatitis (NASH)

Phase 2 Phase 2b trial to be initiated early-2Q 2020.
$1 billion

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.
$157 million

MRNS – Marinus Pharmaceuticals Inc.
Ganaxolone - Magnolia
Postpartum depression

Phase 2 Phase 2 negative data released July 23, 2019.
$216.3 million

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

NDA Filing NDA filing due April 2020.
$924.8 million

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

Phase 1/2 Phase 1/2 prophylactic steroid cohort data due 2H 2020.
$3.2 billion

RTRX – Retrophin Inc.
Fosmetpantotenate
Pantothenate kinase-associated neurodegeneration (PKAN)

Phase 3 Phase 3 top-line data did not meet endpoints - August 22, 2019.
$668.1 million

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
POMC deficiency obesity

NDA Filing NDA filing due 1Q 2020.
$838.1 million

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

Phase 2 Phase 2 Part A data July 24, 2019 noted MADRS score of 15.5 points at Day 15 (n=23; p<0.0001) - secondary endpoint.
$2.4 billion