BPC October 08 update

Key Phase 1/2 catalysts for the fourth quarter; Biotech week in review

Weekly watchlist

We continue our preview of fourth-quarter biotech catalysts to watch with 15 notable Phase 1 and 2 trial readouts. Next week we will follow with a preview of regulatory events such as PDUFA dates and Advisory Committee meetings to look forward to as the fourth quarter comes underway.

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

REGULATORY UPDATES:

Allogene Therapeutics, Inc. (NASDAQ: ALLO) announced Thursday that the FDA placed a clinical hold on Allogene’s AlloCAR T trials after a chromosomal abnormality was reported in a patient in its Phase 1 ALPHA2 study to treat large B-cell lymphoma. Shares closed the week down 46% at $13.13.

ChemoCentryx, Inc. (NASDAQ: CCXI) announced that the FDA approved TAVNEOS (avacopan) to treat ANCA-associated vasculitis (AAV). Shares closed the week up 109% at $38.41.

CLINICAL UPDATES:

Merus N.V. (NASDAQ: MRUS) released data Thursday from its Phase 1 trial of MCLA-158 to treat solid tumors. Merus reported tumor reductions in all patients (7), with three partial responses (PR) and one complete response (CR). Shares closed the week up 29% at $28.10.

Prelude Therapeutics Inc. (NASDAQ: PRLD) shares fell this week following the release of data from two Phase 1 trials to treat tumors. Its PRT543 trial noted one CR and five stable diseases (SD); PRT811 data displayed one CR, one SD, and one PR. Shares closed the week down 50% at $15.87.

OTHER

Voyager Therapeutics (NASDAQ: VYGR) announced a license option agreement with Pfizer (NYSE: PFE) for tracer AAV capsids to enable neurologic and cardiovascular gene therapy programs. Voyager received a $30 million upfront payment with milestones up to $600 million. Shares closed the week up 113% at $5.55.

Osmotica Pharmaceuticals plc (NASDAQ: OSMT) announced a public offering this week of common shares at $2.50 per share with an accompanying warrant. The gross proceeds are to be $35 million. Osmotica also entered into a note purchase agreement for up to $100 million. Shares closed the week down 38% at $1.91.

Drug Price Stage Catalyst Market Cap

ACHL – Achilles Therapeutics plc
ATL001 - THETIS
Melanoma

$6.68
-0.64  -9%
Phase 1/2 Phase 1/2 update 4Q 2021.
$271.2 million

AMTI – Applied Molecular Transport Inc.
AMT-101 combo with with anti-TNFα (MARKET)
Ulcerative colitis

$22.89
+0.15  +1%
Phase 2 Phase 2 top-line data due January 2022.
$879.5 million

BLU – BELLUS Health Inc.
BLU-5937 (SOOTHE)
Chronic cough

$6.16
+0.07  +1%
Phase 2b Phase 2b administrative interim analysis released September 13, 2021- at least one dose of BLU-5937 met the predefined probability threshold for a clinically meaningful reduction in placebo-adjusted 24-hour cough frequency; Limited taste-related adverse events were observed, consistent with previous BLU-5937 trials. No serious adverse events were reported. Phase 2b trail complete enrollment, top-line results expected December 2021.
$482.6 million

BLU – BELLUS Health Inc.
BLU-5937 (BLUEPRINT)
Chronic pruritus associated with atopic dermatitis

$6.16
+0.07  +1%
Phase 2b Phase 2b enrollment completed September 23, 2021 with top-line data due December 2021.
$482.6 million

CLNN – Clene Inc.
CNM-Au8 (RESCUE-ALS)
Amyotrophic Lateral Sclerosis

$5.74
-0.35  -6%
Phase 2 Phase 2 Australia study data due 4Q 2021, study completed September 9, 2021.
$353 million

CMPS – COMPASS Pathways Plc
Psilocybin
Treatment Resistant Depression

$37.84
+2.52  +7%
Phase 2b Phase 2b data due by end of 2021.
$1.6 billion

CMRX – Chimerix Inc.
ONC201
H3 K27M-mutant Glioma

$5.31
-0.39  -7%
Phase 2 Phase 2 blinded independent central review due 4Q 2021.
$458 million

FBIO – Fortress Biotech Inc.
Cosibelimab (CK-301)
Cutaneous squamous cell carcinoma (CSCC)

$2.98
-0.06  -2%
Phase 1 Phase 1 pivotal top-line data due 4Q 2021.
$301.7 million

GNCA – Genocea Biosciences Inc.
GEN-011 (TITAN study)
Various tumors

$1.70
-0.04  -2%
Phase 1/2 Phase 1/2 initiation of dosing announced July 13, 2021, with initial data due late-4Q 2021 or 1Q 2022.
$96.9 million

IFRX – InflaRx N.V.
Vilobelimab (IFX-1) - (IXCHANGE)
ANCA-associated Vasculitis (AAV)

$2.94
+0.16  +6%
Phase 2 Phase 2 data due by the end of 2021.
$129.9 million

MREO – Mereo BioPharma Group plc
Etigilimab (OMP-313M32)
Solid tumors

$2.15
-0.03  -1%
Phase 1/2 Phase 1b/2 initial data due 2H 2021.
$232 million

OCUL – Ocular Therapeutix Inc.
OTX-CSI
Dry eye disease (DED)

$6.93
-3.90  -36%
Phase 2 Phase 2 data did not show separation between the OTX-CSI treated subjects and the vehicle treated subjects for the primary endpoint of increased tear production, noted October 22, 2021.
$531.1 million

PDSB – PDS Biotechnology Corporation
PDS0101 (VERSATILE-002)
Head and neck cancer

$11.64
+0.06  +1%
Phase 2 Phase 2 achieved its preliminary safety benchmark in its first 12 patients September 20, 2021. Initial data due 4Q 2021 / 1Q 2022. Phase 2 completion of enrollment announced October 4, 2021.
$330.9 million

QURE – uniQure N.V.
AMT-130
Huntington's disease

$28.37
+0.13  +0%
Phase 1/2 Phase 1/2 trial DSMB review and initial imaging and biomarker data due before the end of 2021.
$1.3 billion

XFOR – X4 Pharmaceuticals Inc.
Mavorixafor and IMBRUVICA (ibrutinib)
Waldenström macroglobulinemia

$4.40
-0.55  -11%
Phase 1b Phase 1b initial data released at EHA meeting June 11, 2021. At 6 months, patients achieved median IgM level reductions of 60%-75%. Further data due 4Q 2021.
$109 million