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.


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:


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%.


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.
Agitation associated with schizophrenia and bipolar disorders

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

ELDN – Eledon Pharmaceuticals Inc.
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

TJ301 (olamkicept)
Ulcerative colitis

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

NLTX – Neoleukin Therapeutics Inc.
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.
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)

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