BPC June 28 update

Biotech stock catalysts slated for July or early August

Weekly watchlist

With the first half of the 2019 trading year at a close, attention now focuses on catalysts slated for the third quarter, with a particular emphasis on those likely to occur by mid-August at the latest. We have pulled out such key catalysts based on the premise that events due for “mid-2019” occur during this period.

First, let’s review the week that was:

Zogenix, Inc. (NASDAQ: ZGNX) shares closed the week up 18% to $47.78 on news that it intends to resubmit its New Drug Application (NDA) for Fintepla (ZX008, fenfluramine) for the treatment of seizures associated with Dravet syndrome in 3Q 2019. The company was previously issued a Refusal to file Letter (RTF) in April 2019.

Dova Pharmaceuticals, Inc. (NASDAQ: DOVA) shares closed the week up 58% to $14.10 following its announcement the FDA approved a supplemental New Drug Application (sNDA) that expands the use of Doptelet (avatrombopag) to include the treatment of thrombocytopenia in adults with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Aclaris Therapeutics, Inc. (NASDAQ: ACRS) announced Wednesday that its Phase 2 clinical trial of ATI-502 (AA-201 Topical), in patients with alopecia areata (AA), did not meet its primary and secondary endpoints. Shares closed the week down 52% to 2.19.

Acer Therapeutics Inc. (Nasdaq: ACER) shares slumped to close the week down 79% to $3.90 on news that it received a Complete Response Letter (CRL) from the FDA regarding its New Drug Application (NDA) for Edsivo for the treatment of vascular Ehlers-Danlos syndrome (vEDS). The FDA requested a further clinical trial is required in order to resubmit the NDA.

AbbVie Inc. (NYSE: ABBV) announced that it intends to acquire Allergan plc (NYSE: AGN) in a cash and stock transaction. Allergan shareholders will receive 0.8660 AbbVie shares and $120.30 in cash for each Allergan share, for a total consideration of $188.24 per share. Shares of Allergan closed the week up 28% to $167.43, while AbbVie shares closed down 7% to $72.72.

Krystal Biotech, Inc. (Nasdaq: KRYS) announced positive data from its Phase 2 trial of KB103 in patients with severe generalized recessive dystrophic epidermolysis bullosa (RDEB). Five out of six wounds (four recurring and two chronic) treated with KB103 fully closed during the trial. The company also announced an underwritten public offering of $100m of its common stock. Shares closed the week up 38% to $40.27.

Minerva Neurosciences, Inc. (NASDAQ: NERV) shares surged to close the week up 33% to $5.63 on news that its Phase 2b trial of seltorexant (MIN-202) in patients with insomnia disorder demonstrated highly statistically significant (p ≤0.001) and clinically meaningful improvement on LPS (latency to persistent sleep) at Night 1, the primary endpoint of the study.

Conatus Pharmaceuticals Inc. (Nasdaq:CNAT) shares slumped to close the week down 72% to $0.26 following news that top-line results from its ENCORE-LF clinical trial of emricasan in NASH cirrhosis, did not meet its primary endpoint.

Sarepta Therapeutics, Inc. (NASDAQ: SRPT) shares rallied to close Friday up 17% to $151.95 following the release of initial Phase 1b clinical data from Pfizer Inc. (NYSE:PFE) regarding its Phase 1b trial of PF-06939926, in patients with Duchenne muscular dystrophy (DMD), which noted one of the six participants developed a rapid antibody response which resulted in acute kidney injury, hemolysis, and reduced platelet count.

Biotech stock catalysts slated for July or early August:

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

ANAB – AnaptysBio Inc.
ANB019
Generalized pustular psoriasis

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

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

Phase 3 Phase 3 data due mid-2019.
$832.3 million

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

Phase 3 Phase 3 data due 2H 2019 (possibly July/August)
$550.1 million

ITCI – Intra-Cellular Therapies Inc.
Lumateperone
Schizophrenia

PDUFA PDUFA date September 27, 2019. Advisory Committee Meeting July 31, 2019.
$638.5 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.
$638.5 million

KPTI – Karyopharm Therapeutics Inc.
Selinexor
Quadruple Refractory Multiple Myeloma

Approved FDA Approval announced July 3, 2019. Continued approval may be contingent upon data from its Phase 3 Boston trial (data due late-2019/early 2020).
$499.1 million

MRKR – Marker Therapeutics Inc.
MultiTAA-Specific T Cells
Pancreatic cancer

Phase 1 Phase 1/2 data to be presented July 20, 2019 at AACR.
$413.9 million

MRNS – Marinus Pharmaceuticals Inc.
Ganaxolone - Magnolia
Postpartum depression

Phase 2 Data due first half of 3Q 2019.
$228.6 million

NTEC – Intec Pharma Ltd.
AP-CDLD
Advanced Parkinson's Disease

Phase 3 Phase 3 data due July/August 2019.
$114.9 million

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

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

SGMO – Sangamo Therapeutics Inc.
SB-525
Hemophilia A

Phase 1/2 Phase 1/2 data due presented ISTH July 5, 2019 noted first two patients achieved normal, sustained Factor VIII levels.
$1.4 billion

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

Phase 3 Phase 3 top-line data met primary endpoints - July 8, 2019.
$192.7 million