BPC December 13 update

Biotech stock catalysts to watch for the upcoming week; Biotech week in Review

Weekly watchlist

Only two trading weeks remain in 2019, yet an abnormally high number of catalysts still remain on the calendar for the year. Historically, newsflow is quiet from Christmas with companies and agencies taking their holidays. Given the fact that Christmas Eve lies on a Tuesday (half-trading day) suggests that the majority of announcements will be released by Monday December 23. In other words, the upcoming week is setting up to be full of biotech catalysts. We list some of the key catalysts to watch below.

First let’s review price-moving events from the week that was.

Amarin Corporation plc (NASDAQ: AMRN) received approval late-Friday from the FDA for the use of Vascepa (icosapent ethyl) as a secondary therapy to reduce the risk of cardiovascular events among adults with elevated triglyceride levels (a type of fat in the blood). The decision came ahead of the PDUFA date of December 28, 2019.

Sarepta Therapeutics, Inc. (NASDAQ: SRPT) shares closed Friday up 31% to $132.05 on news that the FDA approved Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy (DMD) patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping.

Assertio Therapeutics, Inc. (NASDAQ: ASRT) shares closed the week up 68% to $1.19 following its announcement that privately-held Alvogen will purchase its product Gralise (gabapentin) for the treatment of pain after shingles. Assertio will receive $75m in cash upon closing, expected in early January 2020.

Correvio Pharma Corp. (NASDAQ:CORV) announced that the FDA Cardiovascular and Renal Drugs Advisory Committee (CRDAC) voted that the benefit-risk profile of Brinavess, under review for the rapid conversion of recent onset atrial fibrillation (AF), was not adequate to support approval. The PDUFA date for a final decision by the FDA for the New Drug Application is December 24, 2019. Shares closed the week down 70% to $0.3950.

Synthorx, Inc. (NASDAQ:THOR) announced it will be acquired by Sanofi for $68 per share in cash, which represents an aggregate equity value of approximately $2.5b. Shares closed the week up 172% to $68.03.

ArQule, Inc. (NASDAQ:ARQL) also announced that it will be acquired by Merck (NYSE:MRK), for $20 per share in cash for an approximate total equity value of $2.7b. Shares closed Monday up 104% to $19.70.

Forty Seven, Inc. (NASDAQ: FTSV) was the standout performer from the ASH conference, with shares closing the week up 148% to $35.75 following the release of data from its Phase 1b trial of magrolimab in combination with azacitidine for the treatment of myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). Fate Therapeutics (NASDAQ: FATE), another major mover at ASH, saw its shares close the week up 37% to $18.79 following the release of data from its Phase 1 trial of FT516 in patients with AML.

XBiotech Inc (NASDAQ: XBIT) shares closed Monday up 75% to $19.50 following news that Johnson & Johnson (NYSE: JNJ) will acquire rights to bermekimab, in Phase 2 development for the treatment of atopic dermatitis and hidradenitis suppurativa. XBiotech will receive $750m and up to $600m in potential milestone payments.


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 due mid-February 2020.
$60.9 million

ADMS – Adamas Pharmaceuticals Inc.
GOCOVRI (amantadine)
Multiple sclerosis (MS)

Phase 3 Phase 3 data met primary endpoint at one of two doses. Secondary endpoint in walking impairment not met. Second trial will not be initiated as a result of data - December 17, 2019.
$167.4 million

AXSM – Axsome Therapeutics Inc.
AXS-05 -GEMINI
Major Depressive Disorder

NDA Filing Phase 3 data released December 16, 2019 met primary endpoint. NDA filing due 2H 2020.
$3.2 billion

BGNE – BeiGene Ltd.
Zanubrutinib (BGB-3111) - ASPEN
Waldenström’s macroglobulinemia

Phase 3 Phase 3 top-line data did not meet primary endpoint - December 16, 2019.
$13 billion

BMRN – BioMarin Pharmaceutical Inc.
Vosoritide
Achondroplasia

Phase 3 Phase 3 top-line data met primary endpoint - December 16, 2019.
$15.4 billion

ELOX – Eloxx Pharmaceuticals Inc.
ELX-02
Cystinosis

Phase 2 Phase 2 data released January 14, 2020.
$180.4 million

EPZM – Epizyme Inc.
Tazemetostat
Epithelioid Sarcoma

Approved FDA approval announced January 23, 2020.
$2.6 billion

FIXX – Homology Medicines Inc.
HMI-102
Phenylketonuria (PKU)

Phase 1/2 Phase 1/2 initial data released December 17, 2019.
$725.5 million

ITCI – Intra-Cellular Therapies Inc.
Lumateperone
Schizophrenia

Approved FDA Approval announced December 23, 2019.
$1.4 billion

PTI – Proteostasis Therapeutics Inc.
PTI-428 + PTI-801 + PTI-808
Cystic fibrosis

Phase 2 Phase 2 negative data released December 17, 2019. Mean changes in ppFEV1 were not statistically significant in the heterozygous population. Phase 3 trial to be initiated in 2020.
$95.6 million

SLDB – Solid Biosciences Inc.
SGT-001
Duchenne Muscular Dystrophy

Phase 1/2 Phase 1/2 trial placed on clinical hold - November 12, 2019. Still in place as of December 18, 2019.
$168.3 million

SLGL – Sol-Gel Technologies Ltd.
Twyneo
Acne

NDA Filing Phase 3 data met co-primary endpoints - December 30, 2019. NDA filing due 2H 2020.
$273.8 million

SPPI – Spectrum Pharmaceuticals Inc.
Poziotinib - Zenith20
Non-small cell lung cancer (NSCLC) with exon 20 insertion mutation in EGFR or HER2

Phase 2 Phase 2 primary endpoint from Cohort 1 was not met - December 26, 2019. Cohort 2 data due mid-2020 with Cohort 3 data due 2020.
$323.7 million

WVE – Wave Life Sciences Ltd.
WVE-120102
Huntington’s disease

Phase 1/2 Phase 1b/2a top line data released December 30, 2019 noted statistically significant reduction of 12.4% (p<0.05) in mutant huntingtin (mHTT) protein but no difference in total HTT (tHTT) protein and lower than competition.
$268.1 million