BPC May 03 update

BioNTech BNTX hits fresh highs as U.S. vaccine exports set to commence +11%

Price and Volume Movers

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) shares closed up 9% to $77.11 following the release of data from Part A of its Phase 2 Momentum trial of SRP-5051 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. The trial found that the 30 mg/kg monthly dose of SRP-5051 produced eight times (6.55% vs 0.86%) the dystrophin production as weekly doses of Exondys 51, its currently approved treatment for DMD.

BioNTech SE (NASDAQ: BNTX) shares closed up 11% to $208.90, another record high for the stock. The German biotech is partnered with Pfizer Inc (NYSE: PFE) for its COVID-19 vaccine. Pfizer saw its shares close up 3% to $39.85 ahead of its first-quarter earnings report on Tuesday. The White House also came out supporting their commencement of the export of U.S. manufactured doses of their vaccine, with Canada and Mexico among the first to receive doses from the U.S.

Novavax (NASDAQ:NVAX) shares, conversely, saw its shares close 18% lower to $195.12 following a Reuters report that the company plans to begin delivering its COVID-19 vaccine to the European Union towards the end of 2021. However, most of its intended 200 million doses will not be supplied until 2022.

AVROBIO, Inc. (Nasdaq: AVRO) announced that following an End-of-Phase 1 meeting regarding its candidate gene therapy Fabry disease candidate, AVR-RD-01, it can no longer pursue an accelerated approval process due to the recent full approval of Fabrazyme. It will now conduct a head-to-head registration trial versus Fabrazyme to be conducted in mid-2022. Shares closed down 19% to $9.48.

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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%.

Ocugen, Inc. (OCGN): $15.68; +24%.

Abeona Therapeutics Inc. (ABEO): $2.03; +15%.

Plus Therapeutics, Inc. (PSTV): $2.60; +14%.

ADiTx Therapeutics, Inc. (ADTX): $3.20; +12%.

DECLINERS:

Vaxart, Inc. (VXRT): $8.02; -26%.

Evelo Biosciences, Inc. (EVLO): $10.57; -20%.

Regulus Therapeutics Inc. (RGLS): $1.12; -19%.

Galectin Therapeutics Inc. (GALT): $3.78; -14%.

Sonnet BioTherapeutics Holdings, Inc. (SONN): $2.07; -13%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

AGLE – Aeglea BioTherapeutics Inc.
Pegzilarginase (AEB1102) - PEACE
Arginase I deficiency

Phase 3 Phase 3 data due 4Q 2021.
$366.2 million

AGTC – Applied Genetic Technologies Corporation
AGTC-501
X-linked Retinitis Pigmentosa (XLRP)

Phase 1/2 Phase 1/2 12-month data due at the American Academy of Ophthalmology Annual Meeting in November 13-16, 2021; 3-month interim Skyline data due 4Q 2021. Phase 2/3 (VISTA) six-month interim analysis 4Q 2022.
$164.2 million

CABA – Cabaletta Bio Inc.
DSG3-CAART
Pemphigus vulgaris

Phase 1 Phase 1 acute safety data for the second and third cohorts due 3Q and 4Q 2021, respectively.
$232.8 million

CLSD – Clearside Biomedical Inc.
XIPERE (Suprachoroidal CLS-TA)
Macular edema associated with uveitis

NDA Filing CRL received October 18, 2019. NDA resubmission announced May 3, 2021.
$136.5 million

DNLI – Denali Therapeutics Inc.
DNL310
Hunter syndrome MPS II

Phase 1/2 Phase 1/2 six-month data to be reported mid-2021.
$6.5 billion

HCM – HUTCHMED (China) Limited
Surufatinib
Neuroendocrine Tumors

NDA Filing NDA filing announced May 2, 2021.
$4 billion

ITCI – Intra-Cellular Therapies Inc.
CAPLYTA (Lumateperone)
Bipolar depression

PDUFA PDUFA date December 17, 2021.
$3 billion

LTRN – Lantern Pharma Inc.
LP-300
Non-small cell lung cancer (NSCLC)

Phase 2 Phase 2 trial planned for 3Q 2021.
$152.7 million

MRUS – Merus N.V.
MCLA-129
Solid Tumors

Phase 1/2 Phase 1/2 initiation of dosing announced May 3, 2021.
$864.4 million

NOVN – Novan Inc.
SB206 - B-SIMPLE4
Molluscum

Phase 3 Phase 3 data due 2Q 2021.
$179 million

RGLS – Regulus Therapeutics Inc.
RGLS4326
Autosomal dominant polycystic kidney disease (ADPKD)

Phase 1b Phase 1 data from second cohort due mid-2021.
$67.8 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

VXRT – Vaxart Inc.
Norovirus vaccine (VXA-NVV-104)
Norovirus vaccine

Phase 1b Phase 1b enrollment in dose-ranging trial in elderly adults has commenced - May 7, 2021. Data from booster dose due mid-2021. Phase 2 challenge study planned to start later in 2021.
$786.2 million

VXRT – Vaxart Inc.
VXA-CoV2-1
COVID-19 Vaccine

Phase 1 Phase 2 trial to commence mid-2021.
$786.2 million