BPC January 11 update

Biotech stock catalysts to watch for 2019; Biotech week in review

Weekly watchlist

The bulk of this week’s news flow was released on Monday with the release of a number of significant price-moving data and other news. We note the main highlights below from the week that was.

This week’s watch list is of a slightly different format - as a one-off we highlight 16 clinical data related catalysts to watch out for in 2019. Next week we will move back to focusing on events slated closer in the future, starting up with upcoming regulatory dates which will feature next week.

Axsome Therapeutics, Inc. (NASDAQ: AXSM) was the star performer of the week. Shares surged to close the week up 180% to $7.36 on news that its Phase 2 trial of AXS-05 for the treatment of major depressive disorder (MDD), met the prespecified primary endpoint and significantly improved symptoms of depression. At Week 6, AXS-05 demonstrated a 17.2 point reduction in the Montgomery-Åsberg Depression Rating Scale (MADRS) total score compared to a 12.1 point reduction for bupropion (p=0.013).

Eli Lilly and Company (NYSE: LLY) announced the acquisition of Loxo Oncology, Inc. (NASDAQ: LOXO) for $235 per share in cash, or approximately $8b. The transaction is not subject to any financing condition and is expected to close this quarter. Shares of Loxo closed Monday up 66% to $232.65.

Sage Therapeutics (NASDAQ:SAGE) posted positive news from yet another trial, with news that its Phase 3 trial of SAGE-217 in women with postpartum depression (PPD) met the primary endpoint. After two weeks of outpatient treatment, patients treated with SAGE-217 had a statistically significant improvement of 17.8 points in the Hamilton Rating Scale for Depression (HAMD-17) score, compared to 13.6 for placebo (p=0.0029). Shares rallied to close the week up 28% to $125.10.

Insmed Incorporated (NASDAQ: INSM) shares closed the week up 53% to $22.70 following news noting sales of its recently approved Arikayce are likely to have hit $9.8m in the latest quarter.

La Jolla Pharmaceutical Company (NASDAQ: LJPC) shares closed down Monday by 50% to $5.24 following revenue guidance, predominantly generated from its low blood pressure drug Giapreza, of $24-28m this year, well below analyst expectations.

16 biotech stock catalysts (clinical data) to watch for in 2019:

Drug Stage Catalyst Market Cap

ALNY – Alnylam Pharmaceuticals Inc.
Inclisiran - ORION-9

Phase 3 Phase 3 data 3Q 2019.
$7.2 billion

ARRY – Array BioPharma Inc.
Encorafenib and cetuximab - BEACON CRC
BRAF-Mutant Colorectal Cancer

Phase 3 Phase 3 interim analysis May 21, 2019 met primary ORR and OS endpoints.
$5.7 billion

ASLN – ASLAN Pharmaceuticals Limited
Varlitinib - TREETOPP
Biliary tract cancer

Phase 2/3 Phase 2/3 data due 2H 2019.
$109 million

BCRX – BioCryst Pharmaceuticals Inc.
BCX7353 - APeX-2
Reduce or eliminate attacks in HAE patients

Phase 3 Phase 3 data May 21, 2019 met primary endpoint but lower than expectations.
$379.5 million

BIIB – Biogen Inc.
Aducanumab (Aβ mAb) - EMERGE
Alzheimer’s disease

Phase 3 Phase 3 trial discontinued due to futility - unlikely to meet primary endpoint upon completion - March 21, 2019.
$45.6 billion

FGEN – FibroGen Inc
Anaemia in Chronic Kidney Disease

NDA Filing Phase 3 MACE safety data released May 9, 2019. Non-inferior in non-dialysis, no worse risk than placebo. NDA filing due September/October 2019.
$3.1 billion

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

Phase 3 Phase 3 data due 2H 2019.
$512.6 million

ICPT – Intercept Pharmaceuticals Inc.
Ocaliva (Obeticholic acid (OCA)) - REGENERATE
Adult nonalcoholic steatohepatitis (NASH) patients.

Phase 3 NDA filing due 3Q 2019.
$2.8 billion

IMGN – ImmunoGen Inc.
Mirvetuximab soravtansine - FORWARD I
Cancer - ovarian cancer and relapsed endometrial cancer

Phase 3 Phase 3 top-line data released March 1, 2019 did not meet primary endpoint. Further Phase 3 trial required (noted May 15, 2019).
$288.6 million

KPTI – Karyopharm Therapeutics Inc.
Selinexor - BOSTON
Multiple myeloma

Phase 3 Phase 3 data due end of 2019 or into 2020.
$352.4 million

KRYS – Krystal Biotech Inc.
Epidermolysis Bullosa

Phase 2 Phase 2 top-line data due 2Q 2019.
$466.5 million

MGNX – MacroGenics Inc.
Margetuximab SOPHIA study
Metastatic breast cancer

Phase 3 Phase 3 data released February 6, 2019 met primary endpoint. Detailed data due at ASCO June 4, 2019, 9:45am. BLA filing due 2H 2019.
$899.5 million

SGMO – Sangamo Therapeutics Inc.
MPS Type 2

Phase 1/2 Phase 1/2 updates during 2019.
$1.1 billion

SLDB – Solid Biosciences Inc.
Duchenne Muscular Dystrophy

Phase 1/2 Phase 1/2 negative initial data released February 7, 2019. Update May 12, 2019 noted one drug related serious adverse event.
$210 million

SRPT – Sarepta Therapeutics Inc.
SRP-5051 - PPMO
Duchenne muscular dystrophy

Phase 1/2 Phase 1 trial safety and dosing insight due by end of 2019 or 1Q 2020.
$9 billion

WVE – Wave Life Sciences Ltd.
Huntington’s disease

Phase 1/2 Phase 1b/2a top line data due by YE 2019.
$854.1 million