BPC April 29 update

CARA misses mid-stage trial goal -45%; Ardelyx ARDX 3-month PDUFA delay

Price and Volume Movers

Cara Therapeutics, Inc. (Nasdaq: CARA) announced its Phase 2 KARE trial of Oral Korsuva for the treatment of moderate-to-severe pruritus in mild-to-severe atopic dermatitis (AD) patients did not meet primary endpoint of the change from baseline in the weekly mean of the daily 24-hour Itch-Numeric Rating Scale (I-NRS) score at Week 12. Shares fell 45% to $14.07.

Adverum Biotechnologies, Inc. (NASDAQ:ADVM) shares slid 62% to $3.83 following its announcement Wednesday of a Suspected Unexpected Serious Adverse Reaction (SUSAR) of hypotony (decrease in ocular pressure) in its Phase 2 Infinity trial evaluating ADVM-022 gene therapy for the treatment of diabetic macular edema (DME).

Ocular Therapeutix, Inc. (NASDAQ: OCUL) shares closed up 23% to $19.16 following a sales update of Dextenza, its treatment for ocular pain. The company noted that 7,151 billable inserts were sold in March, compared with 4,582 and 4,901 in January and February, respectively.

Ardelyx, Inc. (Nasdaq: ARDX) shares are trading down 16% to $7.10 after hours following news the FDA has 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 by three months July 29, 2021. The extension follows the submission of new analyses to the FDA which constitutes a major amendment and an automatic extension of the PDUFA date.

Selecta Biosciences, Inc. (NASDAQ: SELB) shares fell 19% to $3.15. The company announced it will assume all rights from Asklepios BioPharmaceutical, Inc. (AskBio), for their gene therapy candidate MMA-101 and ImmTOR for the treatment of methylmalonic acidemia (MMA). However, due to a manufacturing issues, Selecta now expects the submission of the Investigational New Drug Application (IND) for MMA-101 and ImmTOR will be delayed until at least the fourth quarter of 2021.

Emergent BioSolutions Inc. (NYSE: EBS) shares are trading down 5% after hours to $59.99 following its report of financial results for the first quarter. Net product sales fell 7% to $137.9 million compared with the same period last year. Emergent also revised 2021 revenue forecasts (product sales + contract development and manufacturing (CDMO) services) to $1.7b-$1.9b, down from $1.95b-$2.05b.

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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:

Galera Therapeutics, Inc. (GRTX): $8.12; +23%.

Capricor Therapeutics, Inc. (CAPR): $4.54; +15%.

Enlivex Therapeutics Ltd. (ENLV): $12.10; +14%.

Design Therapeutics, Inc. (DSGN): $24.80; +9%.

Vaxart, Inc. (VXRT): $9.21; +9%.

DECLINERS:

Brooklyn ImmunoTherapeutics, Inc. (BTX): $34.22; -22%.

Galectin Therapeutics Inc. (GALT): $4.24; -17%.

Aldeyra Therapeutics, Inc. (ALDX): $12.51; -10%.

Edesa Biotech, Inc. (EDSA): $5.55; -10%.

Protalix BioTherapeutics, Inc. (PLX): $3.54; -10%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

AERI – Aerie Pharmaceuticals Inc.
AR-15512 (AVX-012) - COMET-1
Dry eye disease

Phase 2b Phase 2b top-line data due 3Q 2021.
$812.7 million

ALNY – Alnylam Pharmaceuticals Inc.
Lumasiran (ALN-GO1) ILLUMINATE-C
Impaired renal function

Phase 3 Phase 3 top-line data due mid-2021.
$16 billion

ARDX – Ardelyx Inc.
Tenapanor
Serum phosphorus - chronic kidney disease (CKD) on dialysis

PDUFA PDUFA date extended by three months to July 29, 2021.
$674.2 million

AVIR – Atea Pharmaceuticals Inc.
AT-527 (MORNINGSKY)
COVID-19

Phase 3 Phase 3 trial initiation announced April 29, 2021.
$1.9 billion

BMRN – BioMarin Pharmaceutical Inc.
Vosoritide
Achondroplasia

PDUFA PDUFA date extended by three months to November 20, 2021.
$14.1 billion

CANF – Can-Fite Biopharma Ltd Sponsored ADR (Israel)
Piclidenoson (CF101)
Psoriasis

Phase 3 Phase 3 top-line data due 4Q 2021.
$35.2 million

CARA – Cara Therapeutics Inc.
KORSUVA (CR845/difelikefalin) - KARE
Atopic dermatitis patients with moderate-to-severe pruritus

Phase 2 Phase 2 trial did not meet primary endpoint - April 29, 2021. Phase 3 trial planned for end of 2021.
$669.7 million

ELOX – Eloxx Pharmaceuticals Inc.
ELX-02
Cystic fibrosis

Phase 2 Phase 2 data due 2H 2021.
$65.6 million

GILD – Gilead Sciences Inc.
Domvanalimab (AB154) + Zimberelimab (AB122) (ARC-7)
Non-small cell lung cancer (NSCLC)

Phase 2 Phase 2 data due 2Q 2021.
$86.6 billion

IDRA – Idera Pharmaceuticals Inc.
Tilsotolimod + nivolumab and ipilimumab - ILLUMINATE 206
Squamous Cell Carcinoma of the Head and Neck (SCCHN)

Phase 2 Phase 2 data due 4Q 2021.
$52.5 million

LABP – Landos Biopharma Inc.
NX-13
Ulcerative Colitis

Phase 1b Phase 1b top-line data due 1Q 2022.
$468.2 million

PHAT – Phathom Pharmaceuticals Inc.
Vonoprazan PHALCON-HP
H. pylori

NDA Filing Phase 3 top-line data met primary endpoints - April 29, 2021. NDA filing due 4Q 2021.
$1 billion

SELB – Selecta Biosciences Inc.
MMA-101
Methylmalonic acidemia (MMA)

Phase 1 Phase 1 IND postponed until at least 4Q 2021.
$332.8 million

VRTX – Vertex Pharmaceuticals Incorporated
VX-864
Alpha-1 antitrypsin (AAT) deficiency

Phase 2 Phase 2 data due 2Q 2021.
$56 billion