BPC December 20 update

10 Biotech stock catalysts to watch over the holiday period; Biotech week in Review

Weekly watchlist

Administrative note:

Happy Holidays to all readers of BioPharmCatalyst. The weekly edition will be taking a one-week break, with expected news flow to be light from Tuesday. The next edition will be on Saturday January 4.

-

The pre-Christmas expected rush of data came through as expected last week, yet a few key data releases and one FDA approval event remain on the calendar. Given that Tuesday is a half trading day and Wednesday is closed for Christmas Day, if data are to be released one would expect this to arrive on Monday or following the week of Christmas, with many offices likely to be closed. However, it should be noted that companies are also known to miss previously stated timelines so it would also not surprise if some companies do not meet their previously stated timelines.

We list below TEN key catalysts slated for release by the end of the year while also reviewing major price movers from the week that was.

Axsome Therapeutics, Inc. (NASDAQ: AXSM) shares closed the week up 115% to $100.67 following news its Phase 3 Gemini trial of AXS-05 met the primary endpoint in patients with major depressive disorder (MDD).

Wave Life Sciences Ltd. (Nasdaq: WVE) announced that it will discontinue development of suvodirsen for patients with Duchenne muscular dystrophy (DMD) who have mutations amenable to exon 51 skipping, based on its interim analysis of the Phase 1 open-label extension (OLE) trial. Shares closed the week down 59% to $15.24.

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) announced that following its pre-New Drug Application (NDA) meeting with the FDA of maralixibat for the treatment of pruritus associated with Alagille syndrome, it intends to file a rolling NDA in 3Q 2020. Shares closed the week up 188% to $23.57.

Novartis (NYSE: NVS) announced that it did not meet the primary endpoint in its Phase 3 Luster trials of its DP2 antagonist, fevipiprant (QAW039), in patients with asthma. Gossamer Bio Inc (NASDAQ: GOSS) which also has a DP2 antagonist, GB-001, saw its shares fall in response to the data, closing down the week 37% to $15.88.

Adamas Pharmaceuticals, Inc. (Nasdaq: ADMS) shares closed the week down 44% to $3.71 on news that it will not initiate its second Phase 3 trial of ADS-5102 in multiple sclerosis (MS) patients, due to data from its first trial which met the primary endpoint at the high dose but missed at the lower dose.

Proteostasis Therapeutics, Inc. (NASDAQ: PTI) shares closed the week down 71% to $1.25 following the release of disappointing data from its Phase 2 trial in F508del homozygous and heterozygous cystic fibrosis patients.

Minerva Neurosciences, Inc. (NASDAQ: NERV) shares fell 16% to $5.98 following its announcement its Phase 2b trial of MIN-117 in adult patients suffering from moderate to severe major depressive disorder (MDD) failed to meet its primary and key secondary endpoints.

Epizyme, Inc. (Nasdaq: EPZM) shares closed the week up 20% to $20.68 following news the FDA Oncologic Drugs Advisory Committee (ODAC) voted 11-0 in favor of the benefit-risk profile of tazemetostat as a treatment for patients with metastatic or locally advanced epithelioid sarcoma (ES) not eligible for curative surgery. The PDUFA date for a final decision is January 23, 2020.

Catalyst Biosciences, Inc. (Nasdaq: CBIO +66%) and Selecta Biosciences, Inc. (NASDAQ: SELB +45%) shares rallied Thursday following license agreements with Biogen Inc. (Nasdaq: BIIB) and Asklepios BioPharmaceutical, Inc, respectively.

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

APLT – Applied Therapeutics Inc.
AT-007
Galactosemia

Phase 2 Phase 2 data January 8, 2019 noted significant plasma galactitol reduction of ~50% (p<0.01). Full data to be presented at the Society for Inherited Metabolic Disorders Annual Meeting, April 26 – 29, 2020.
$904.4 million

AXSM – Axsome Therapeutics Inc.
AXS-07 - MOMENTUM
Migraine

NDA Filing Phase 3 trial met co-primary endpoints - December 30, 2019. NDA filing due 2H 2020.
$3.2 billion

BHVN – Biohaven Pharmaceutical Holding Company Ltd.
Rimegepant
Migraine - preventative

Phase 3 Phase 3 top-line data due early-1Q 2020.
$2.9 billion

ELOX – Eloxx Pharmaceuticals Inc.
ELX-02
Cystinosis

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

ITCI – Intra-Cellular Therapies Inc.
Lumateperone
Schizophrenia

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

PGNX – Progenics Pharmaceuticals Inc.
PyL
Prostate cancer

Phase 3 Phase 3 data met primary endpoint - December 23, 2019.
$406.2 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