BPC January 07 update

Key Phase 3 catalysts for the first quarter; Biotech week in review

Weekly watchlist

With 2022 in full swing, we now look forward to a fresh batch of catalysts for the new quarter. Over the next few weeks, we highlight catalysts commencing with notable Phase 3 data readouts this week, followed by Phase 1 and 2 catalysts the week after.

First, let’s review the week that was with notable price-moving events in the healthcare sector.

REGULATORY

Genprex, Inc. (NASDAQ: GNPX) was granted Fast Track Designation (FTD) by the FDA for REQORSA in combination with Merck & Co's (NYSE: MRK) Keytruda to treat non-small cell lung cancer (NSCLC). Shares closed the week up 47% at $2.22.

CLINICAL UPDATES:

Lixte Biotechnology Holdings, Inc. (NASDAQ: LIXT) released mouse model data of LB-100, noting that treatment increased the responsiveness to immunotherapy and was associated with new antigen production and tumor infiltration of cytotoxicity in various types of cancer. Shares closed the week up 78% at $2.14.

Hoth Therapeutics, Inc. (NASDAQ: HOTH) also released Alzheimer's disease mouse model data of HT-ALZ, reporting a significant decrease in Aβ with HT-ALZ, compared to placebo. Shares closed the week up 44% at $0.95.

Gritstone bio, Inc. (NASDAQ: GRTS) reported Phase 1 data from the CORAL-BOOST study of its mRNA vaccine for COVID-19. Data from the first cohort demonstrated both a neutralizing antibody response to Spike and robust CD8+ T cell responses. Shares closed the week down 50% at $6.45.

AVROBIO, Inc. (NASDAQ: AVRO) is deprioritizing its Fabry disease program due to data showing variable engraftment patterns, and an increasingly challenging market and regulatory environment for Fabry disease. Shares closed the week down 45% at $2.12.

Avalo Therapeutics, Inc. (NASDAQ: AVTX) released data from its Phase 1b of AVTX-002 to treat Crohn's Disease and noted that they would be discontinuing development for AVTX-007 to treat multiple myeloma. Shares closed the week down 39% at $1.04.

OTHERS:

Alterity Therapeutics (NASDAQ: ATHE) was granted a new patent covering more than 80 novel compounds designed to redistribute excess iron implicated in many neurodegenerative diseases, including Parkinson's disease and Alzheimer's disease. Shares closed the week up 40% at $1.01.

Drug Price Stage Catalyst Market Cap

ARAV – Aravive Inc.
AVB-500
Platinum Resistant Ovarian Cancer (PROC)

$2.23
-0.05  -2%
Phase 3 Phase 3 initiation of dosing announced April 26, 2021. Interim analysis due 1Q 2022.
$46.9 million

ASND – Ascendis Pharma A/S
TransCon - (PaTHway)
Hypoparathyroidism

$107.97
-3.17  -3%
Phase 3 Phase 3 top-line data due 1Q 2022. NDA due in 3Q 2022.
$6.1 billion

CFRX – ContraFect Corporation
Exebacase - (DISRUPT)
Serious infections caused by Staph aureus including MRSA

$2.36
-0.08  -3%
Phase 3 Phase 3 interim futility analysis delayed due to COVID-19 until 1H 2022.
$92.8 million

CLVS – Clovis Oncology Inc.
OPDIVO (nivolumab) and RUBRACA (rucaparib) - (ATHENA)
Ovarian Cancer - First-line maintenance treatment

$2.19
-0.14  -6%
Phase 3 Phase 3 top-line monotherapy data due 1Q 2022. Combo data due 2H 2022.
$284.7 million

CYTK – Cytokinetics Incorporated
Omecamtiv mecarbil - (METEORIC-HF)
Acute heart failure

$30.30
-0.51  -2%
Phase 3 Phase 3 completion of enrollment announced June 15, 2021 with data due in early 2022.
$2.6 billion

ICPT – Intercept Pharmaceuticals Inc.
OCALIVA (obeticholic acid) - (REVERSE)
NASH patients with compensated cirrhosis

$15.41
-1.59  -9%
Phase 3 Phase 3 data due in 1Q 2022.
$455.3 million

KOD – Kodiak Sciences Inc
KSI-301 - (DAZZLE)
Wet age-related macular degeneration (AMD)

$57.80
-2.64  -4%
Phase 2/3 Phase 2/3 pivotal trial top-line data due 1Q 2022.
$3 billion

KPTI – Karyopharm Therapeutics Inc.
XPOVIO (selinexor) - (SIENDO)
Endometrial cancer

$8.28
+0.55  +7%
Phase 3 Phase 3 recruitment completed on December 2, 2021. Phase 3 top-line data due in 1Q 2022. sNDA due in 1H 2022.
$625.5 million

MDGL – Madrigal Pharmaceuticals Inc.
Resmetirom (MGL-3196) - (MAESTRO-NAFLD-1)
Non-Alcoholic Fatty Liver Disease

$63.57
-1.69  -3%
Phase 3 Phase 3 52-week top-line data reported rapid and sustained reduction in liver fat, liver volume, liver fibrosis, and atherogenic lipids. Treatment was safe and well-tolerated at 100 mg per day in patients treated for 52 weeks, noted November 12, 2021. Phase 3 blinded 52-week data due in January 2022.
$1.1 billion

NKTR – Nektar Therapeutics
Bempegaldesleukin (NKTR-214) and OPDIVO (nivolumab)
Melanoma

$10.95
-0.83  -7%
Phase 3 Phase 3 data due in 1H 2022.
$2 billion

OCUP – Ocuphire Pharma Inc.
Nyxol Eye Drops - (LYNX-1)
Dim Light or Night Vision Disturbances (DLD)

$3.09
-0.11  -3%
Phase 3 Phase 3 trial enrollment completed January 5, 2022. Phase 3 data due early 2022.
$58.2 million

PRQR – ProQR Therapeutics N.V.
Sepofarsen (QR-110) - (ILLUMINATE)
Leber's Congenital Amaurosis (LCA) - Genetic blindness

$5.07
-0.45  -8%
Phase 2/3 Phase 2/3 top-line data due 1Q 2022.
$373.7 million

SAGE – Sage Therapeutics Inc.
Zuranolone (MDD-305) - CORAL
Major Depressive Disorder

$38.21
-0.61  -2%
Phase 3 Phase 3 data due early 2022.
$2.3 billion

SRRA – Sierra Oncology Inc.
Momelotinib - (MOMENTUM)
Myelofibrosis

$16.87
-1.97  -10%
Phase 3 Phase 3 top-line data due by end of January 2022. NDA due 2Q 2022.
$254 million

SWTX – SpringWorks Therapeutics Inc.
Nirogacestat (DeFi)
Desmoid tumors

$54.24
-0.38  -1%
Phase 3 Phase 3 top-line data due early 2022.
$2.7 billion