BPC September 27 update

17 Phase 3 biotech stock catalysts slated for 4Q 2019

Weekly watchlist

With just one trading day left in the third quarter, eyes now turn toward the final quarter of 2019, which promises to be full of price-moving catalysts. This week we highlight Phase 3 data readouts slated for the final quarter, which will be followed over the next two weeks by Phase 1/2 readouts to watch and regulatory (PDUFA/Advisory Committee) dates.

First, let’s review the week that was.

Avadel Pharmaceuticals plc (NASDAQ:AVDL) announced Monday that the FDA has agreed to lowering the enrolment in a Phase 3 trial of once-nightly sodium oxybate, FT218, in the treatment of excessive daytime sleepiness and cataplexy in patients suffering from narcolepsy. Enrolment is now expected to be completed by the end of 2019, compared with recent guidance of 2H 2020. Top-line data are due in 2Q 2020. Shares closed the week up 26% to $4.25.

Enanta Pharmaceuticals, Inc. (NASDAQ:ENTA) announced Wednesday data from its Phase 2a trial of EDP-305 for the treatment of non-alcoholic steatohepatitis (NASH). The trial only just met the primary endpoint (p=0.049) and the high-dose cohort saw 51% of patients exhibiting pruritus (itching of the skin). Shares closed the week down 16% to $60.28.

The Medicines Company (NASDAQ: MDCO) announced that it met primary and secondary endpoints in both its Phase 3 ORION-9 trial of inclisiran in patients with Heterozygous Familial Hypercholesterolemia (HeFH) and its ORION-10 trial in patients with atherosclerotic cardiovascular disease (ASCVD).

AzurRx BioPharma Inc (NASDAQ: AZRX) shares closed the week down 42% to $0.58 following negative data from its Phase 2 clinical trial of MS1819 for the treatment of exocrine pancreatic insufficiency in cystic fibrosis (CF).

Akcea Therapeutics, Inc. (NASDAQ: AKCA) shares closed Monday down 20% to $18.04 on news of the departures of Paula Soteropoulos, chief executive officer, Sarah Boyce, president, and Jeff Goldberg, chief operating officer.

Concert Pharmaceuticals, Inc. (NASDAQ:CNCE) shares closed Friday down 37% to $6.25 following news from its partner, Avanir Pharmaceuticals, that its Phase 3 trial of AVP-786 for the treatment of moderate-to-severe agitation in patients with Alzheimer’s dementia did not meet its primary and key secondary endpoints.

Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS) shares closed Thursday up 23% to $1.69 on news that its open-label, dose-finding Phase 2 trial evaluating intravenous (IV) ganaxolone in patients with refractory status epilepticus (RSE), met the primary endpoint. Shares pulled back Friday to close the week at $1.52.

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15 Phase 3 biotech stocks catalysts slated for 4Q 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.
$67.3 million

ALNA – Allena Pharmaceuticals Inc.
Reloxaliase ALLN-177 (URIROX-1)
Enteric Hyperoxaluria

Phase 3 Phase 3 trial met primary endpoint, key secondary endpoint missed - November 7, 2019.
$46.7 million

APLS – Apellis Pharmaceuticals Inc.
Pegcetacoplan (APL-2) PEGASUS
Paroxysmal Nocturnal Hemoglobinuria (PNH)

NDA Filing NDA filing due 2H 2020.
$2.4 billion

AUPH – Aurinia Pharmaceuticals Inc
Voclosporin - AURORA
Lupus

NDA Filing NDA rolling submission has completed - May 26, 2020.
$1.7 billion

AXSM – Axsome Therapeutics Inc.
AXS-05 STRIDE-1
Treatment resistant depression

Phase 3 Phase 3 data did not meet primary endpoint. Secondary endpoints met. Second Phase 3 trial to commence 3Q 2020.
$3 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.
$15.6 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.
$22.9 billion

CARA – Cara Therapeutics Inc.
KORSUVA (CR845/difelikefalin) oral
Stage 3-4 Chronic Kidney Disease-Associated Pruritus

Phase 2 Phase 2 data met primary endpoint but secondary missed - December 3, 2019. Phase 3 trial to be initated 2H 2020.
$825.5 million

HALO – Halozyme Therapeutics Inc.
HALO-301
Pancreatic cancer

Phase 3 Phase 3 data November 4, 2019 did not meet primary endpoint.
$3.8 billion

INCY – Incyte Corporation
Itacitinib - GRAVITAS-301
Treatment-naïve acute GVHD

Phase 3 Phase 3 trial did not meet primary endpoint - January 2, 2020.
$23.4 billion

KALA – Kala Pharmaceuticals Inc.
KPI‑121 0.25% STRIDE 3
Dry eye disease

Phase 3 Phase 3 data met primary endpoint - March 9, 2020.
$584.5 million

KPTI – Karyopharm Therapeutics Inc.
Selinexor - BOSTON
Multiple myeloma

Phase 3 sNDA filing announced May 20, 2020.
$1.4 billion

OPK – Opko Health Inc.
Somatrogon (hGH-CTP)
Children - growth hormone deficiency

Phase 3 Phase 3 data met primary endpoint.
$2.7 billion

RETA – Reata Pharmaceuticals Inc.
Bardoxolone methyl - CARDINAL
Chronic Kidney Disease Caused by Alport Syndrome

Phase 2/3 Phase 3 trial met primary endpoint - November 11, 2019.
$5.2 billion

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

Phase 3 Phase 3 data did not meet primary endpoint - December 5, 2019.
$2.3 billion

SLGL – Sol-Gel Technologies Ltd.
Twyneo
Acne

NDA Filing NDA filing due 2H 2020.
$185.2 million

SUPN – Supernus Pharmaceuticals Inc.
SPN-810
Impulsive Aggression in ADHD

Phase 3 Phase 3 trial did not meet primary endpoint - November 5, 2019.
$1.3 billion