BPC April 26 update

Aldeyra ALDX Phase 3 data due premarket tomorrow; Voyager VYGR and ​uniQure QURE clinical holds released

Price and Volume Movers

uniQure N.V. (NASDAQ:QURE) shares closed up 8% to $34.95 on news the FDA has removed the clinical hold on its hemophilia B gene therapy program. 52-week data from their Phase 3 HOPE-B trial are due later this quarter.

Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) announced it will hold a conference call on Tuesday, April 27, 2021, at 8:00 a.m. (ET) to provide top-line results from the Phase 3 INVIGORATE trial of reproxalap in allergic conjunctivitis.

Eledon Pharmaceuticals, Inc. (NASDAQ: ELDN) announced that the FDA has requested the company provide data from non-human primates, prior to commencing its Phase 2 trial of AT-1501 in renal transplantation. Eledon expects to complete this evaluation by late-2022. The company previously guided that a Phase 2 trial would be initiated mid-2021. Shares, which closed the normal trading session up 7%, have retraced gains after hours on the news, currently trading down 6% to $10.76.

Reviva Pharmaceuticals Holdings, Inc. (NASDAQ: RVPH) shares closed up 104% to $8.70 following a press release noting it met the primary endpoint in a Phase 2 trial of RP5063 (brilaroxazine) for Acute Schizophrenia. It mentioned the Positive and Negative Syndrome Scale (PANSS) total score was reduced by 20 points, a statistically significant treatment difference from the placebo. These data, however, were first published in 2017.

Voyager Therapeutics, Inc. (Nasdaq: VYGR) announced the FDA has removed its clinical hold on its Investigational New Drug (IND) application for VY-HTT01, a gene therapy candidate for the treatment of Huntington’s disease (HD). Voyager plans to initiate VYTAL, a Phase 1/2 clinical trial of VY-HTT01, this year. Shares closed up 16% to $5.44.

Reata Pharmaceuticals, Inc. (Nasdaq: RETA) announced that the FDA accepted its New Drug Application (NDA) for bardoxolone for the treatment of patients with chronic kidney disease caused by Alport syndrome and assigned a PDUFA date of February 25, 2022. The FDA also advised that it is currently planning to hold an Advisory Committee meeting to discuss the application.

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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:

Aeterna Zentaris Inc. (AEZS): $1.14; +22%.

Ocugen, Inc. (OCGN): $11.57; +20%.

Aligos Therapeutics, Inc. (ALGS): $29.10; +19%.

Liminal BioSciences Inc. (LMNL): $4.71; +18%.

Landos Biopharma, Inc. (LABP): $11.83; +16%.

DECLINERS:

Eagle Pharmaceuticals, Inc. (EGRX): $40.98; -7%.

Codiak BioSciences, Inc. (CDAK): $17.21; -5%.

Gemini Therapeutics, Inc. (GMTX): $10.13; -5%.

Forward Pharma A/S (FWP): $7.20; -5%.

Sol-Gel Technologies Ltd. (SLGL): $13.74; -4%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ALDX – Aldeyra Therapeutics Inc.
Reproxalap (ADX-102) - (INVIGORATE)
Allergic conjunctivitis

Phase 3 Phase 3 trial met primary endpoint - April 27, 2021.
$697.3 million

AXSM – Axsome Therapeutics Inc.
Dextromethorphan-bupropion (AXS-05)
Major Depressive Disorder

PDUFA priority review PDUFA date under priority review August 22, 2021.
$2.2 billion

AZN – AstraZeneca PLC
Nirsevimab (MELODY)
Respiratory syncytial virus

Phase 3 Phase 3 trial met primary endpoint - April 26, 2021.
$146.7 billion

BTAI – BioXcel Therapeutics Inc.
BXCL501 (SERENITY I)
Agitation associated with schizophrenia and bipolar disorders

sNDA Filing sNDA filing announced March 11, 2021.
$764.8 million

ELDN – Eledon Pharmaceuticals Inc.
AT-1501
Healthy volunteers

Phase 1 Phase 1 data safety, tolerability and pharmacokinetic data to be presented at ATC meeting June 7, 2021. Phase 2 trial planned.
$118.9 million

IMAB – I-MAB
TJ301 (olamkicept)
Ulcerative colitis

Phase 2 Phase 2 trial met primary endpoint.
$4.5 billion

NLTX – Neoleukin Therapeutics Inc.
NL-201
Solid tumors

Phase 1 Phase 1 trial initiation announced May 5, 2021.
$460.7 million

QURE – uniQure N.V.
Etranacogene dezaparvovec (AMT-061) - HOPE-B
Hemophilia B

Phase 3 Phase 3 clinical hold released April 26, 2021. Top-line data due 2Q 2021.
$1.5 billion

RETA – Reata Pharmaceuticals Inc.
Bardoxolone methyl
Alport Syndrome

PDUFA PDUFA date February 25, 2022. Advisory committee meeting is expected.
$2.9 billion

VYGR – Voyager Therapeutics Inc.
VY-HTT01 (VYTAL)
Huntington’s Disease

Phase 1/2 Phase 1/2 trial to be initiated in 4Q 2021 with initial data due in 2022.
$171.3 million

ZSAN – Zosano Pharma Corporation
Qtrypta (M207)
Migraine

NDA Filing CRL announced October 21, 2020. NDA filing to be resubmitted following PK trial. PK trial to commence in June and finish 3Q 2021.
$78 million