BPC October 08 update

Phase 1/2 biotech stock events to watch 4Q; Biotech week in Review

Weekly watchlist

Following last week’s preview of upcoming key Phase 3 data readouts to watch this quarter, this week we focus on Phase 1 and 2 events to watch, before looking ahead next week at upcoming PDUFA and Advisory Committee meetings. First, let’s review the week that was with notable healthcare news.

CLINICAL DATA

Eli Lilly and Company (NYSE: LLY) announced that its Phase 2 combination trial of LY-CoV555 and LY-CoV016, in patients with COVID-19, significantly reduced viral load at day 11 (p=0.011), meeting the primary endpoint. COVID-related hospitalization visits were 0.9% versus placebo 5.8%, a relative risk reduction of 84.5 percent (p=0.049). It has submitted an initial request for emergency use authorization (EUA) for LY-CoV555 monotherapy, with a request for the combination EUA due in November. Shares closed the week up 8% to $156.84.

Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) shares closed Tuesday up 18% to $18.74 on news its Phase 2 trial of mavrilimumab in giant cell arteritis (GCA) achieved both the primary and secondary efficacy endpoints.

miRagen Therapeutics, Inc. (NASDAQ: MGEN) announced Monday it will discontinue development of cobomarsen following data from its Phase 2 SOLAR clinical trial in patients with Cutaneous T-Cell Lymphoma (CTCL) which showed lack of efficacy. Shares closed the week down 21% to $0.5974.

Amgen (NASDAQ:AMGN) and Cytokinetics, Incorporated (NASDAQ:CYTK) announced data from their Phase 3 GALACTIC-HF trial of omecamtiv mecarbil in patients with heart failure with reduced ejection fraction (HFrEF), met the primary composite efficacy endpoint reducing cardiovascular (CV) death or heart failure events compared to placebo. However, it did not meet the secondary endpoint of reduction in cardiovascular (CV) death, which saw shares tumble with Cytokinetics shares closing Thursday down 42% to $16.00, while Amgen shares closed 7% lower to $240.09.

Corbus Pharmaceuticals Holdings, Inc. (NASDAQ:CRBP) announced its Phase 2b trial of lenabasum in patients with cystic fibrosis did not meet its primary endpoint. Shares fell Tuesday by 36% to $1.21.

BUYOUT RELATED

Bristol Myers Squibb (NYSE:BMY) announced Monday it will acquire MyoKardia, Inc. (NASDAQ:MYOK) for $13.1 billion, or $225 per share in cash. Shares of MyoKardia closed up 58% to $220.24.

Eidos Therapeutics, Inc. (NASDAQ:EIDX) shares closed Monday up 42% to $73.78 on news BridgeBio Pharma, Inc. (NASDAQ:BBIO) will acquire all of the outstanding common stock of Eidos it does not already own, representing approximately 36.3% of Eidos' outstanding shares.

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Phase 1/2 biotech stock events to watch 4Q 2020.

Drug Stage Catalyst Market Cap

ALT – Altimmune Inc.
T-COVID
COVID-19

Phase 1/2 Phase 1/2 data due 4Q 2020.
$368.6 million

ARCT – Arcturus Therapeutics Holdings Inc.
LUNAR-COV19
COVID-19 vaccine

Phase 1 Phase 1 initial data due 4Q 2020.
$1.2 billion

ARNA – Arena Pharmaceuticals Inc.
Etrasimod
Atopic dermatitis (AD)

Phase 2 Phase 2 data due 4Q 2020.
$4.8 billion

ARPO – Aerpio Pharmaceuticals Inc.
Razuprotafib (AKB-9778)
Open-angle glaucoma (OAG)

Phase 2 Phase 2 enrollment completion announced September 15, 2020 with top-line data due 4Q 2020.
$54.7 million

ARQT – Arcutis Biotherapeutics Inc.
ARQ-154
Scalp psoriasis

Phase 2b Phase 2b data due 4Q 2020.
$877.5 million

AVXL – Anavex Life Sciences Corp.
ANAVEX 2-73 - U.S. RS-001
Rett syndrome

Phase 2 Phase 2 top-line data due 4Q 2020.
$336.8 million

BTAI – BioXcel Therapeutics Inc.
BXCL501 (TRANQUILITY)
Agitation associated with dementia

Phase 1b Phase 1b data due 4Q 2020.
$1 billion

CALA – Calithera Biosciences Inc.
CB-839 + Cabozantinib (CANTATA)
Renal cell carcinoma

Phase 2 Phase 2 top-line data due late-4Q 2020 or early-1Q 2021.
$251.9 million

CCXI – ChemoCentryx Inc.
Avacopan -AURORA
Hidradenitis Suppurativa (HS)

Phase 2b Phase 2b data released October 28, 2020. Primary endpoint across Hurley Stages 2 and 3 not met. Phase 3 trial planned in Stage 3 patients only.
$3.6 billion

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

Phase 1/2 Phase 1/2 update due 4Q 2020.
$479.8 million

KALV – KalVista Pharmaceuticals Inc.
KVD900
Hereditary Angioedema (HAE)

Phase 2 Phase 2 data due 4Q 2020.
$286.9 million

MRTX – Mirati Therapeutics Inc.
MRTX849 - KRYSTAL
Solid tumors

Phase 1/2 Phase 1/2 updated data noted (23/51) 45% ORR in NSCLC patients - October 25, 2020.
$9 billion

RCKT – Rocket Pharmaceuticals Inc.
RP-A501
Danon disease

Phase 1 Phase 1 data due 4Q 2020.
$1.5 billion

SRRK – Scholar Rock Holding Corporation
SRK-015 - TOPAZ
Spinal muscular atrophy (SMA)

Phase 2 Phase 2 top-line data due 2Q 2021.
$1.3 billion