BPC February 16 update

Bluebird BLUE shares slide on halt to gene therapy sickle cell disease program -38%; ​Cortexyme CRTX partial clinical hold on Alzheimer's drug -35%

Price and Volume Movers

Cortexyme, Inc. (NASDAQ:CRTX) shares slid to closed down 35% to $30.72 on news the FDA has placed a partial clinical hold on atuzaginstat (COR388) in patients with Alzheimer's disease. As a result, the open-label extension part of its Phase 2/3 trial has stopped. Patients in the placebo-controlled randomized part of the trial will continue to be dosed, with top-line data still slated for 4Q 2021.

bluebird bio, Inc. (Nasdaq: BLUE) shares slumped to close down 38% to $28.44 on news that its Phase 1/2 (HGB-206) and Phase 3 (HGB-210) trials of LentiGlobin gene therapy for sickle cell disease (SCD) (bb1111) have been placed on a temporary suspension due to a reported Suspected Unexpected Serious Adverse Reaction (SUSAR) of acute myeloid leukemia (AML). A patient that was treated with LentiGlobin more than five years ago has been recently diagnosed with AML. The company is investigating the cause of the patient’s AML in order to determine if there is any relationship with the use of a lentiviral vector in the manufacture of its LentiGlobin gene therapy

AVROBIO, Inc. (Nasdaq: AVRO) shares fell in sympathy to the fall by bluebird, closing down 28% to $11.62. The company also has a clinical-stage gene therapy platform, with its most advanced, a lentiviral gene therapy for Fabry disease currently in Phase 2 trials.

Sesen Bio (Nasdaq: SESN) announced the FDA has accepted for filing its Biologics License Application (BLA) for Vicineum for the treatment of high-risk, BCG-unresponsive non-muscle invasive bladder cancer (NMIBC), and granted priority review with a PDUFA date of August 18, 2021. The company noted that an advisory committee meeting to discuss the BLA is not planned. Shares closed up 8% to $3.02.

Aravive, Inc. (NASDAQ:ARAV) shares closed up 15% to $8.39 on news of a registered direct offering with Eshelman Ventures, LLC to sell 2,875,000 shares of common stock at a price of $7.29 per share for gross proceeds of approximately $21m.

Galmed Pharmaceuticals Ltd. (NASDAQ:GLMD) announced a public offering of 2,197,803 of its ordinary shares for gross proceeds of approximately $10m. Shares fell 6% to $4.75.

Y-mAbs Therapeutics, Inc. (NASDAQ:YMAB) announced after hours the commencement of a registered underwritten public offering of up to $100m in shares of its common stock.

ESSA Pharma Inc. (NASDAQ:EPIX) also announced after hours that it has commenced an underwritten public offering of up to $100m of its common shares.

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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:

Diffusion Pharmaceuticals Inc. (DFFN): $1.70; +37%.

Applied Genetic Technologies Corporation (AGTC): $8.07; +31%.

Clene Inc. (CLNN): $16.30; +31%.

Timber Pharmaceuticals, Inc. (TMBR): $3.06; +30%.

Akers Biosciences, Inc. (AKER): $4.82; +29%.

DECLINERS:

Inhibrx, Inc. (INBX): $23.52; -30%.

Immunovant, Inc. (IMVT): $16.17; -27%.

Hepion Pharmaceuticals, Inc. (HEPA): $2.22; -25%.

Avenue Therapeutics, Inc. (ATXI): $5.35; -23%.

ImmunoGen, Inc. (IMGN): $8.62; -18%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

AMGN – Amgen Inc.
AMG 510 - sotorasib (CodeBreak 100)
Non-small cell lung cancer (NSCLC)

PDUFA priority review PDUFA date under priority review August 16, 2021.
$130.4 billion

ARQT – Arcutis Biotherapeutics Inc.
ARQ-154
Seborrheic dermatitis

Phase 3 Phase 3 trial to be initiated 2Q / 3Q 2021 with top-line data due 2Q / 3Q 2022.
$1.7 billion

BLUE – bluebird bio Inc.
LentiGlobin (HGB-210)
Sickle Cell Disease

Phase 3 Phase 3 trial temporarily suspended due to a reported Suspected Unexpected Serious Adverse Reaction (SUSAR) of acute myeloid leukemia (AML) - February 16, 2021.
$2.1 billion

CLNN – Clene Inc.
CNM-ZnAg
COVID-19

Phase 2 Phase 2 initiation announced February 16, 2021. Data due 2H 2021.
$819.1 million

CTXR – Citius Pharmaceuticals Inc.
Mino-Lok
Catheter related blood stream infection

Phase 3 Phase 3 interim analysis due 2Q 2021.
$254.1 million

DFFN – Diffusion Pharmaceuticals Inc.
Trans sodium crocetinate (TSC) - 100-303 COVID
COVID-19

Phase 1b Phase 1b trial has completed - no dose-limiting toxicities or serious adverse events were observed. TCOM trial to be initiated 1Q 2021.
$107.8 million

INSM – Insmed Inc.
Treprostinil Palmitil Inhalation Powder (TPIP)
Pulmonary arterial hypertension (PAH)

Phase 1 Phase 1 top-line data released February 19, 2021 - generally safe and well tolerated.
$4 billion

JAZZ – Jazz Pharmaceuticals plc
JZP-258
Idiopathic Hypersomnia

sNDA Filing sNDA filing has been completed - February 16, 2021.
$9.3 billion

MNPR – Monopar Therapeutics Inc.
Validive (VOICE)
Oral mucositis

Phase 2/3 Phase 2b/3 initiation of dosing announced February 16, 2021.
$74.6 million

NRIX – Nurix Therapeutics Inc.
NX-2127
B-cell malignancies

Phase 1 Phase 1 trial planned for 1Q 2021.
$1.4 billion

PYPD – PolyPid Ltd.
D-PLEX100 - SHIELD I
Abdominal (soft tissue) sternal surgical site infections

Phase 3 Phase 3 data due end of 2021.
$217.5 million

SESN – Sesen Bio Inc.
Vicinium
Non-muscle invasive bladder cancer (NMIBC)

PDUFA priority review PDUFA date under priority review August 18, 2021.
$405 million