BPC November 21 update

Phase 3 key biotech stock catalysts left for 2019; Biotech week in Review

Weekly watchlist

Following last week’s look at notable remaining Phase 1/2 catalysts for 2019, we list 14 key Phase 3 catalysts that have readouts slated for release by the end of year.

First let’s review what was another full week of price-moving news.

Karuna Therapeutics, Inc. (Nasdaq: KRTX) announced Monday that its Phase 2 clinical trial of KarXT for the treatment of acute psychosis in patients with schizophrenia, met the primary endpoint, exhibiting a statistically significant 11.6 point mean reduction in total Positive and Negative Syndrome Scale (PANSS) score compared to placebo. Shares closed the week up 381% to $85.01.

The Medicines Company (NASDAQ: MDCO) shares closed the week up 32% to $68.55 following updated data presented at the AHA meeting from two Phase 3 trials of its cholesterol lowering drug, inclisiran. In its ORION-9 trial the company noted reductions of LDL cholesterol of 50% over 18 months compared with placebo, while the ORION-10 trial saw the reductions increase to 58%. Both trials had previously shown to meet their respective primary endpoints.

Myovant Sciences (NYSE: MYOV) announced its Phase 3 HERO trial of oral relugolix met its primary efficacy endpoint and all six key secondary endpoints in men with advanced prostate cancer. Shares closed the week up 128% to $14.09.

Athenex, Inc. (NASDAQ:ATNX) shares closed the week up 38% to $16.08 following its announcement that its abstract for the Phase 3 trial of oral paclitaxel and encequidar (oral paclitaxel) has been selected for the press program of the 2019 San Antonio Breast Cancer Symposium (SABCS) on Friday, December 13, 2019. The abstract title noted the treatment to be “the first orally administered paclitaxel shown to be superior to IV paclitaxel on confirmed response and survival with less neuropathy.

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) announced the FDA approved Givlaari (givosiran) injection for subcutaneous use for the treatment of adults with acute hepatic porphyria (AHP). Shares closed the week up 16% to $106.91.

Corcept Therapeutics Incorporated (NASDAQ: CORT) shares closed Thursday down 22% to $13.22 following news the U.S. Patent and Trademark Office (USPTO) will review the validity of a patent for Korlym, its approved drug in patients with Cushing's syndrome. The review follows a challenge made by Teva Pharmaceutical Industries Ltd.

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Phase 3 key biotech stock catalysts left for 2019:

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 3Q 2020.
$32.2 million

ALNY – Alnylam Pharmaceuticals Inc.
Lumasiran (ALN-GO1) ILLUMINATE-A
Primary Hyperoxaluria Type 1 (PH1)

NDA Filing Completion of NDA filing announced April 7, 2020.
$12.4 billion

ARDX – Ardelyx Inc.
Tenapanor
Serum phosphorus - chronic kidney disease (CKD) on dialysis

NDA Filing NDA filing due mid-2020.
$477.6 million

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.
$665.3 million

AUPH – Aurinia Pharmaceuticals Inc
Voclosporin - AURORA
Lupus

NDA Filing NDA rolling submission has commenced - noted March 16, 2020. To be completed 2Q 2020.
$1.6 billion

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

NDA Filing NDA filing due 4Q 2020.
$1.8 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.
$10.5 billion

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. However, noted October 22, 2019 that it intends to submit a BLA filing early 2020 based on data that showed the primary endpoint was met. Data released December 5, 2019 noted rate of decline in CDR-SB of 23% on high dose (p=0.0101 ); 14% on low dose (p=0.1171).
$52.4 billion

BMRN – BioMarin Pharmaceutical Inc.
Vosoritide
Achondroplasia

NDA Filing Phase 3 top-line data met primary endpoint - December 16, 2019. Regulatory filing due 3Q 2020.
$14.4 billion

CCXI – ChemoCentryx Inc.
Avacopan - CCX168 (ADVOCATE)
Associated vasculitis (AAV)

NDA Filing NDA filing due mid-2020.
$2.6 billion

OBSV – ObsEva SA
Linzagolix OBE2109 - PRIMROSE 2
Uterine fibroids

Phase 3 Phase 3 data met primary endpoint - December 9, 2019. 12 month data due 2Q 2020.
$94.5 million

SAGE – Sage Therapeutics Inc.
SAGE-217 - MOUNTAIN
Major Depressive Disorder (MDD)

Phase 3 Phase 3 data did not meet primary endpoint - December 5, 2019.
$1.5 billion

SLGL – Sol-Gel Technologies Ltd.
Twyneo
Acne

NDA Filing Phase 3 data met co-primary endpoints - December 30, 2019. NDA filing due 2H 2020.
$155.9 million

TGTX – TG Therapeutics Inc.
TG-1101 and TGR-1202 - UNITY-CLL study
Chronic Lymphocytic Leukemia (CLL) and non-Hodgkin's Lymphoma (NHL)

Phase 3 Phase 3 PFS interim analysis 2Q 2020 (noted in conference call March 3, 2020)
$1.1 billion