BPC May 13 update

​Bristol Myers BMY issued Refusal to File letter; ASCO abstracts released ALLO +41%; ARVN -29%; MGNX -15%

Price and Volume Movers

Bristol Myers Squibb (NYSE: BMY) and bluebird bio, Inc. (Nasdaq: BLUE) announced that the companies received a Refusal to File letter from the FDA regarding the Biologics License Application (BLA) for idecabtagene vicleucel (ide-cel; bb2121) for patients with heavily pre-treated relapsed and refractory multiple myeloma. The FDA noted that the Chemistry, Manufacturing and Control (CMC) module of the BLA requires further detail to complete the review. Bristol Myers is planning to resubmit the BLA no later than the end of July 2020.

Abstracts for the ASCO 2020 Virtual Meeting were released after hours today. Notable movers following the respective releases are listed below:

Arvinas, Inc. (NASDAQ: ARVN) shares are trading down 29% after hours to $36.25 following its abstract release from its Phase 1 trial of ARV-110 in men with metastatic castration-resistant prostate cancer. 2/15 confirmed PSA responses were noted, one of which was associated with tumor reduction. Further data will be released at the meeting on May 29.

Allogene Therapeutics, Inc. (NASDAQ: ALLO) announced data from its Phase 1 dose escalation ALPHA study of ALLO-501 in relapsed/refractory non-Hodgkin lymphoma (NHL), which showed an overall response rate (ORR) of 78% with three complete responses (CR) and four partial responses (PR) out of 11 patients. Shares are trading up 41% after hours to $43.80.

MacroGenics, Inc. (NASDAQ: MGNX) shares, which traded at around $7 less than two weeks ago, pulled back Wednesday following its recent surge. Data from the abstract release from its Phase 1 trial of margetuximab and MGD013 in advanced HER2-positive tumors, showed objective responses were observed in 6 of 14 (43%) evaluable patients. Four have been confirmed, with tumor-shrinkage observed in other patients. Shares are trading down 15% to $22.61.

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Major price movers (stocks priced > $1.00, volume > 50k):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume:

ADVANCERS:

vTv Therapeutics Inc. (VTVT): $3.19; +25%.

OncoSec Medical Incorporated (ONCS): $2.03; +21%.

Translate Bio, Inc. (TBIO): $15.67; +15%.

Brickell Biotech, Inc. (BBI): $1.41; +13%.

Veru Inc. (VERU): $3.97; +12%.

DECLINERS:

Osmotica Pharmaceuticals plc (OSMT): $3.86; -16%.

Capricor Therapeutics, Inc. (CAPR): $6.66; -16%.

Gossamer Bio, Inc. (GOSS): $13.48; -15%.

Avadel Pharmaceuticals plc (AVDL): $7.77; -15%.

Cleveland BioLabs, Inc. (CBLI): $1.90; -12%.


Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

AKRO – Akero Therapeutics Inc.
AKR-001
Nonalcoholic steatohepatitis (NASH)

Phase 2a Phase 2a dose groups all met primary endpoint - March 31, 2020. Top-line safety/tolerability, laboratory measures and paired biopsy data due 2Q 2020.
$728.9 million

ALNA – Allena Pharmaceuticals Inc.
Reloxaliase ALLN-177 (URIROX-2)
Enteric Hyperoxaluria

Phase 3 Phase 3 interim analysis at the first sample size reassessment (SSR) due 1Q 2022 and top-line data 3Q 2022.
$41.6 million

BLUE – bluebird bio Inc.
ide-cel bb2121 - KarMMa
Relapsed/refractory multiple myeloma

BLA Filing Refusal-to-file letter received May 13, 2020. BLA to be refiled no later than July 2020.
$3.5 billion

EQ – Equillium Inc.
EQ001 - itolizumab
Acute graft-versus-host disease

Phase 1b Phase 1b top-line data due 2H 2020.
$61 million

ETNB – 89bio Inc.
BIO89-100
Nonalcoholic steatohepatitis (NASH)

Phase 1/2 Phase 1b/2 trial top-line data due 2H 2020.
$355.3 million

EYEN – Eyenovia Inc.
MicroStat
Mydriasis - pupil dilation

NDA Filing NDA filing due 2020.
$57.2 million

FULC – Fulcrum Therapeutics Inc.
Losmapimod
Facioscapulohumeral muscular dystrophy (FSHD)

Phase 2b Phase 2b interim data due 3Q 2020.
$465.4 million

IMUX – Immunic Inc.
IMU-838 CALVID-1
COVID-19

Phase 2 Phase 2 dosing to commence June 2020 with data due later in 2020.
$153.4 million

NGM – NGM Biopharmaceuticals Inc.
NGM282 - ALPINE 2/3
Non-alcoholic steatohepatitis (NASH)

Phase 2b Phase 2b top-line data due 1H 2021.
$1.3 billion

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
POMC deficiency obesity / Leptin Receptor Deficiency Obesity

PDUFA priority review PDUFA date under priority review November 27, 2020.
$854.9 million

TCON – TRACON Pharmaceuticals Inc.
Envafolimab
Sarcomas

Phase 3 Phase 3 trial to commence 2H 2020 with interim data due 2021 and final data in 2022.
$12.7 million

TLC – Taiwan Liposome Company Ltd.
TLC590
Postsurgical pain

Phase 2 Phase 2 data released May 31, 2020 - statistically significant pain relief over both placebo (p<0.001) and bupivacaine (p=0.0188) from 0 to 24 hours. The differences in AUC0-72hrs did not reach statistical significance.
$182.1 million

VIE – Viela Bio Inc.
Inebilizumab
Myasthenia gravis

Phase 3 Phase 3 trial to be initiated mid-2020.
$2.4 billion

VNDA – Vanda Pharmaceuticals Inc.
HETLIOZ (tasimelteon)
Smith-Magenis Syndrome

sNDA Filing Refusal-to-file letter issued by the FDA for sNDA filing - March 13, 2020. sNDA to be refiled.
$639.3 million