BPC November 24 update

Key Phase 1 and 2 catalysts left for the quarter; Biotech week in review

Weekly watchlist

This week we revisit our Phase 1 and 2 catalysts from our fourth quarter watchlist. Next week we will highlight regulatory events remaining for the quarter.

First, however, let’s review the week that was with price-moving events.

REGULATORY UPDATES:

Longeveron Inc. (NASDAQ: LGVN) received Rare Pediatric Disease (RPD) designation from the FDA for its drug Lomecel-B to treat Hypoplastic Left Heart Syndrome (HLHS). Shares closed the week up 386% at $42.3.

NRx Pharmaceuticals, Inc. (NASDAQ: NRXP) announced that the FDA denied Breakthrough Therapy Designation (BTD) for aviptadil. The FDA already granted priority and rolling review as part of the Fast Track Designation awarded in July 2020. Shares closed the week down 14% at $4.50.

CLINICAL UPDATES:

Aptevo Therapeutics Inc. (NASDAQ: APVO) released data from its Phase 1b expansion trial of APVO436 to treat acute myeloid leukemia (AML). The data noted that in Cohort 1, a high-risk AML patient achieved complete remission after treatment. Shares closed the week up 61% at $11.95.

OTHER:

Qualigen Therapeutics, Inc. (NASDAQ: QLGN) shares closed the week up 75% at $1.75, following a Zach’s Small Cap report noting a $9 price target for the company.

Outlook Therapeutics, Inc. (NASDAQ: OTLK) announced that, due to demand, the underwriter has agreed to increase the size of the previously announced public offering and purchase 40,000,000 shares at $1.25 per share. The gross proceeds are expected to be $50 million. Shares closed the week down 18% at $1.27.

Vallon Pharmaceuticals (NASDAQ: VLON) shares traded up after HC Wainwright & Co. initiated coverage with a Buy rating and $12 price target. Shares closed the week up 15% at $5.39.

Gracell Biotechnologies Inc. (NASDAQ: GRCL) announced that members of its senior management team will use personal funds to purchase up to a maximum of $2 million of American depositary shares (ADSs) within the next three months. Shares closed the week down 18% at $7.09.

Drug Price Stage Catalyst Market Cap

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

$5.46
-0.35  -6%
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 trial complete enrollment, top-line results expected December 2021.
$427.7 million

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

$5.46
-0.35  -6%
Phase 2b Phase 2b enrollment completed September 23, 2021 with top-line data due December 2021.
$427.7 million

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

$2.83
-0.15  -5%
Phase 1 Phase 1 pivotal top-line data due by year-end 2021. BLA submission due in 2022.
$282 million

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

$1.38
-0.02  -1%
Phase 1/2 Phase 1/2 initiation of dosing announced July 13, 2021, with initial data due 1Q 2022 to early 2Q 2022.
$79.3 million

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

$1.90
-0.16  -8%
Phase 1/2 Phase 1b/2 initial data due 2H 2021.
$205.5 million

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

$10.31
+0.66  +7%
Phase 2 Phase 2 achieved its preliminary safety benchmark in its first 12 patients September 20, 2021. Phase 2 completion of enrollment announced October 4, 2021. Phase 2 initial data due 4Q 2021 or 1Q 2022.
$293.2 million

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

$4.52
-0.16  -3%
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 expected to be presented in an ASH poster presentation and at the Investor Day on December 16, 2021.
$111.7 million