BPC April 30 update

Sarepta SRPT Duchenne muscular dystrophy data set to be released Monday

Price and Volume Movers

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) announced it will release data from the 30 mg/kg arm of its Phase 2 MOMENTUM trial, a multiple-ascending dose clinical trial of SRP-5051 for the treatment of Duchenne muscular dystrophy, on Monday May 3, 2021. A conference call will be held at 8:30am ET.

Ardelyx, Inc. (Nasdaq: ARDX) shares closed down 13% to $7.31 following news released Thursday that the FDA extended the PDUFA date for its New Drug Application (NDA) of tenapanor for the control of serum phosphorus in adult patients with chronic kidney disease (CKD) on dialysis. The new PDUFA date is July 29, 2021. New information supplied to the FDA by Ardelyx constituted a major amendment resulting the in the extension.

Vaxart, Inc. (NASDAQ:VXRT) shares closed up 17% to $10.78. The company is due to hold a webinar discussing T-cell response data from its Phase 1 trial of its COVID-19 vaccine tablet candidate on Monday, May 3, 2021 at 12:00 p.m. Eastern Time.

AstraZeneca (NASDAQ:AZN) shares closed up 3% to $53.07 following its release of first-quarter earnings. The company reported revenues of $7.23 billion for the quarter, an increase of 15% and above consensus estimates of $7.1 billion.

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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:

Brooklyn ImmunoTherapeutics, Inc. (BTX): $49.80; +46%.

Sonnet BioTherapeutics Holdings, Inc. (SONN): $2.37; +18%.

Vascular Biogenics Ltd. (VBLT): $2.26; +14%.

Cyclo Therapeutics, Inc. (CYTH): $9.27; +14%.

Checkmate Pharmaceuticals, Inc. (CMPI): $7.24; +13%.

DECLINERS:

Keros Therapeutics, Inc. (KROS): $58.80; -16%.

ALX Oncology Holdings Inc. (ALXO): $62.66; -13%.

MacroGenics, Inc. (MGNX): $32.36; -9%.

Sana Biotechnology, Inc. (SANA): $21.50; -8%.

Cara Therapeutics, Inc. (CARA): $12.95; -8%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ABBV – AbbVie Inc.
Upadacitinib
Ulcerative Colitis (induction/maintenance)

Phase 3 Phase 3 data due 2H 2021.
$206.5 billion

ALXN – Alexion Pharmaceuticals Inc.
ALXN2075 (cerdulatinib)
Refractory non-Hodgkin lymphoma and chronic lymphocytic leukemia

Phase 1/2 Phase 1/2a data due 2Q 2021.
$38.6 billion

ALXN – Alexion Pharmaceuticals Inc.
ALXN2050
Paroxysmal nocturnal hemoglobinuria (PNH)

Phase 2 Phase 2 trial has been re-initiated. Top-line data due 1H 2022.
$38.6 billion

ALXN – Alexion Pharmaceuticals Inc.
ALXN1840 (WTX101)
Wilson disease

Phase 3 Phase 3 top-line data due 3Q 2021.
$38.6 billion

ALXN – Alexion Pharmaceuticals Inc.
ULTOMIRIS (ravulizumab-cwvz)
Generalized myasthenia gravis (gMG)

Phase 3 Phase 3 top-line data due 2H 2021.
$38.6 billion

ALXN – Alexion Pharmaceuticals Inc.
ULTOMIRIS (ravulizumab) - CHAMPION-ALS
Amyotrophic lateral sclerosis (ALS)

Phase 3 Phase 3 top-line data due 1H 2022.
$38.6 billion

ALXN – Alexion Pharmaceuticals Inc.
ULTOMIRIS
Neuromyelitis Optica Spectrum Disorder (NMOSD)

Phase 3 Phase 3 data top-line data due 2H 2022.
$38.6 billion

ALXN – Alexion Pharmaceuticals Inc.
ALXN1720
Healthy volunteers

Phase 1 Phase 1 data due 2H 2021.
$38.6 billion

BMY – Bristol-Myers Squibb Company
CM-274 Opdivo (nivolumab)
Bladder cancer

PDUFA priority review PDUFA date under priority review September 3, 2021.
$146.2 billion

BYSI – BeyondSpring Inc.
Plinabulin (Trial 103) - DUBLIN-3
Non small cell lung cancer - refractory

Phase 3 Phase 3 top-line overall survival data due mid-2021.
$295.8 million

FBIO – Fortress Biotech Inc.
CAEL‐101
AL amyloidosis

Phase 3 Phase 3 top-line data due 2H 2022. Phase 2 safety and tolerability data to be presented at EHA June 11, 2021.
$344.3 million

MREO – Mereo BioPharma Group plc
Etigilimab
Clear Cell Ovarian Cancer

Phase 1/2 Phase 1/2 trial planned.
$317.9 million

SRPT – Sarepta Therapeutics Inc.
SRP-5051 (MOMENTUM)
Duchenne muscular dystrophy

Phase 2 Phase 2 30 mg/kg arm data presented May 3, 2021 - mean exon skipping of 10.79% and mean dystrophin expression of 6.55%.
$6 billion