BPC December 17 update

Remaining 4Q biotech catalysts to watch; Biotech week in review

Weekly watchlist

As 2021 comes to a close our watchlist is updated to focus on upcoming catalysts before quarter-end. Next we will continue to count down to the quarter with catalysts likely to read out before year end.

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

REGULATORY UPDATES:

The FDA approved Calliditas Therapeutics AB’s (NASDAQ: CALT) TARPEYO (budesonide) to treat primary IgA nephropathy (IgAN). Shares closed the week up 35% at $23.48.

CLINICAL UPDATES:

Galera Therapeutics, Inc. (NASDAQ: GRTX) corrected previously announced top-line data for its Phase 3 ROMAN trial of avasopasem to treat RT-induced severe oral mucositis (SOM). Shares closed the week up 126% at $2.96.

Cabaletta Bio, Inc. (NASDAQ: CABA) released top-line data from its Phase 1 trial of DSG3-CAART to treat mucosal Pemphigus Vulgaris (mPV). 67% of patients (2/3) reported disease activity scores worsening within six months after infusion. Shares closed the week down 65% at $4.29.

Adagio Therapeutics, Inc. (NASDAQ: ADGI) released in vitro data reporting that ADG20 is less effective than previously thought in neutralizing COVID-19's omicron variant. Shares closed the week down 68% at $11.48.

OTHERS:

GENFIT S.A. (NASDAQ: GNFT) announced a licensing deal with Ipsen (OTC: IPSEY) to develop, manufacture, and commercialize its elafibranor for people living with Primary Biliary Cholangitis (PBC). Shares closed the week up 56% at $5.09.

Abeona Therapeutics Inc. (NASDAQ: ABEO) announced a public offering of 44,700,000 shares of its stock and warrants at a combined price of $0.39 per unit. The gross proceeds are to be $17.5 million. Shares closed the week down 52% at $0.29.

Drug Price Stage Catalyst Market Cap

ALDX – Aldeyra Therapeutics Inc.
ADX-629
Psoriasis / atopic asthma / COVID-19

$3.25
-0.22  -6%
Phase 2 Phase 2 top-line data due by 1Q 2022.
$188.8 million

ALDX – Aldeyra Therapeutics Inc.
Reproxalap (ADX-102) - (TRANQUILITY)
Dry eye syndrome

$3.25
-0.22  -6%
NDA Filing Phase 3 enrollment completed November 9, 2021. Phase 3 trial did not meet the primary endpoint, secondary endpoint was achieved, December 20, 2021. NDA submission expected mid-2022.
$188.8 million

ALLK – Allakos Inc.
Lirentelimab - (ENIGMA 2)
Eosinophilic Gastritis and/or eosinophilic duodenitis (EoD)

$6.85
-0.33  -5%
Phase 3 Phase 3 study showed 45% (181/405) of patients with moderate-to-severe unexplained gastrointestinal symptoms who underwent upper endoscopy with biopsy met the histologic criteria for eosinophilic gastritis and/or eosinophilic duodenitis (EG/EoD) noted October 25, 2021. Phase 3 data reported that the trial met its histological primary endpoint but did not meet patient reported symptomatic primary endpoint, noted December 21, 2021.
$372 million

AMGN – Amgen Inc.
OTEZLA (apremilast)
Plaque psoriasis

$227.72
-1.18  -1%
Approved Approved December 20, 2021.
$128.3 billion

ANNX – Annexon Inc.
ANX005
Huntington’s disease

$7.50
-0.38  -5%
Phase 2 Phase 2 data reported that treatment was generally well-tolerated, noted January 4, 2022. Full Phase 2 data due in 1H 2022.
$287.8 million

AQST – Aquestive Therapeutics Inc.
Libervant (diazepam)
Epileptic seizures

$2.95
-0.35  -11%
PDUFA CRL announced September 25, 2020. NDA resubmitted with new PDUFA date December 23, 2021. Letter from FDA December 21, 2021, noting PDUFA date will not be met. No further information provided.
$118.5 million

BBIO – BridgeBio Pharma Inc.
Acoramidis (AG10) - (ATTRibute-CM)
ATTR-CM

$10.30
-1.16  -10%
NDA Filing Phase 3 top-line reported that trial did not meet its primary endpoint, noted December 27, 2021. NDA filing expected in 2022. Part B data due 2023.
$1.5 billion

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

$2.05
-0.05  -2%
Phase 1 Phase 1 pivotal top-line data expected in 1Q 2022. BLA submission due in 2022.
$204.2 million

JAGX – Jaguar Health Inc.
Crofelemer (Mytesi)
Chronic Idiopathic Diarrhea in Non-HIV Patients

$0.81
-0.06  -7%
Phase 3 Phase 4 trial ongoing.
$37.2 million

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

$5.71
-0.36  -6%
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 to be presented at the Multidisciplinary Head and Neck Cancers Symposium February 24-26, 2022.
$162.4 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

TCON – TRACON Pharmaceuticals Inc.
TRC102
Mesothelioma cancer

$2.13
-0.12  -5%
Phase 2 Phase 2 data reported that 9 patients reported stable disease as best response with Grade 3 TRAEs including lymphopenia (4) and anemia (1), noted October 7, 2021.
$41.4 million

TSHA – Taysha Gene Therapies Inc.
TSHA-120
Giant axonal neuropathy (GAN)

$7.93
-0.24  -3%
Phase 1/2 Phase 1/2 data due in January 2022.
$305.1 million