BPC April 01 update

​United Therapeutics UTHR FDA approval +15%; Emergent Biosolutions EBS falls on COVID vaccine manufacturing issues -13%

Price and Volume Movers

United Therapeutics Corporation (Nasdaq: UTHR) announced that the FDA approved Tyvaso (treprostinil) Inhalation Solution for the treatment of patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD; WHO Group 3) to improve exercise ability. Tyvaso was first approved in 2009 for the treatment of pulmonary arterial hypertension (PAH; WHO Group 1) to improve exercise ability. Shares closed up 15% to $192.34.

Johnson & Johnson (NYSE:JNJ) released a statement late-Wednesday notifying that a batch of its COVID-19 vaccine manufactured by Emergent Biosolutions did not meet the desired quality control standards. The New York Times reported that workers at the manufacturing facility accidentally mixed the ingredients from two vaccines of Johnson & Johnson and AstraZeneca, contaminating up to 15 million doses. The report noted that the error went undiscovered for days before Johnson & Johnson’s quality control checks uncovered it. Shares of Emergent BioSolutions Inc. (NYSE: EBS) fell 13% to $80.46 on the report. However, its CEO, in an interview on CNBC, noted that there was not a mix-up and the batch was pulled as it was inconsistent with their quality specifications.

AzurRx BioPharma, Inc. (NASDAQ: AZRX) shares fell 24% to $1.02. As noted yesterday, the company announced that top-line data from its Phase 2b OPTION 2 clinical trial investigating MS1819 in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (EPI) did not meet the primary efficacy endpoint.

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Major price movers (stocks priced > $1.00, volume > 50k listed on NASDAQ/NYSE):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume:

ADVANCERS:

Applied Molecular Transport Inc. (AMTI): $63.57; +44%.

Cyclerion Therapeutics, Inc. (CYCN): $3.29; +18%.

Cleveland BioLabs, Inc. (CBLI): $6.16; +17%.

Evofem Biosciences, Inc. (EVFM): $2.02; +15%.

Benitec Biopharma Inc. (BNTC): $5.81; +12%.

DECLINERS:

Lava Therapeutics B.V. (LVTX): $13.00; -14%.

BioXcel Therapeutics, Inc. (BTAI): $37.63; -13%.

CTI BioPharma Corp. (CTIC): $2.54; -13%.

Gracell Biotechnologies Inc. (GRCL): $13.46; -13%.

Atreca, Inc. (BCEL): $13.69; -11%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ALT – Altimmune Inc.
NASOSHIELD
Anthrax

Phase 1b Phase 1b data released April 1, 2021.
$479.2 million

BGNE – BeiGene Ltd.
DKN-01 and Tislelizumab ( DisTinGuish)
Gastric / Gastroesophageal Junction Cancer

Phase 2a Phase 2a initial data due 2H 2021.
$28.3 billion

BYSI – BeyondSpring Inc.
Plinabulin + TAC (Trial 106) - Protective-2
Chemotherapy-induced neutropenia (CIN)

Phase 3 NDA filing announced April 1, 2021.
$313.8 million

GILD – Gilead Sciences Inc.
Axicabtagene ciloleucel - (ZUMA-3)
Adult patients with r/r ALL - cancer

sNDA Filing sBLA filing announced April 1, 2021.
$82 billion

KDMN – Kadmon Holdings Inc.
KD025-215
Diffuse cutaneous systemic sclerosis (dcSSc)

Phase 2 Phase 2 open-label initiation announced April 1, 2021 with initial data due by end of 2021.
$628.9 million

ONTX – Onconova Therapeutics Inc.
ON 123300
Solid tumors

Phase 1 Phase 1 enrollment of second cohort announced April 1, 2021.
$194.4 million

PASG – Passage Bio Inc.
PBGM01
GM1 gangliosidosis

Phase 1/2 Phase 1/2 initiation of dosing announced April 1, 2021. Initial biomarker and safety data due mid-2021.
$879.6 million

RLMD – Relmada Therapeutics Inc.
REL-1017 (RELIANCE I)
Treatment Resistant Depression

Phase 3 Phase 3 data due 1H 2022.
$590 million

RLMD – Relmada Therapeutics Inc.
REL-1017 (RELIANCE II)
Major depressive disorder (MDD

Phase 3 Phase 3 data due 1H 2022.
$590 million

SWTX – SpringWorks Therapeutics Inc.
Nirogacestat and Teclistamab
Multiple myeloma

Phase 1b Phase 1b initiation of dosing announced April 1, 2021.
$3.4 billion

SYN – Synthetic Biologics Inc.
SYN-020
Healthy volunteers

Phase 1 Phase 1 top-line data due 3Q 2021.
$83.9 million

UTHR – United Therapeutics Corporation
Tyvaso
Pulmonary hypertension associated with interstitial lung disease (PH-ILD) (WHO Group 3)

Approved FDA approval announced April 1, 2021.
$9.1 billion