BPC June 10 update

​Vertex VRTX disappoints with key mid-stage data -13% AH; Orphazyme ORPH soars 302% on speculative trading

Price and Volume Movers

Orphazyme A/S (NASDAQ:ORPH) shares saw extreme volatility closing up 302% to $21.00 after reaching an intra-day high of $77.77 (+1,387%). No news was released from the company. The company is awaiting a decision from the FDA for its regulatory application of Arimoclomol for Niemann-Pick disease Type C with a PDUFA (FDA approval) date scheduled for June 17. CytRx Corporation (CYTR), which is entitled to milestone payments should the drug be approved, saw its shares rally to close 55% higher to $3.10.

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) shares are trading down 13% to $188.00 after hours following the release of data from its much-awaited Phase 2 trial of VX-864 in people with alpha-1 antitrypsin deficiency (AATD). The company noted that while the trial met the primary endpoint, the treatment effect is unlikely to translate into substantial clinical benefit. As a result, Vertex will not advance VX-864 into late-stage development.

Progenity, Inc. (NASDAQ:PROG) announced a private placement for the sale of units consisting of shares of common stock and warrants to purchase common stock, for gross proceeds of $40 million. Shares fell 7% to $2.49.

Ocugen, Inc. (NASDAQ: OCGN) shares fell 28% to $6.69 following news that it will no longer file a COVID-19 vaccine Emergency Use Authorization (EUA) for COVAXIN, partnered with Bharat Biotech. Instead, on the recommendation from the FDA, it will go through the longer and traditional approval process by submitting a Biologics License Application (BLA).

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Other 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:

Galecto, Inc. (GLTO): $8.00; +60%.

Axcella Health Inc. (AXLA): $5.13; +49%.

Heat Biologics, Inc. (HTBX): $9.60; +40%.

Graybug Vision, Inc. (GRAY): $5.53; +22%.

Terns Pharmaceuticals, Inc. (TERN): $19.34; +18%.

DECLINERS:

Regulus Therapeutics Inc. (RGLS): $1.05; -17%.

Jaguar Health, Inc. (JAGX): $1.91; -13%.

ZIOPHARM Oncology, Inc. (ZIOP): $2.90; -11%.

Aeterna Zentaris Inc. (AEZS): $1.02; -9%.

Inovio Pharmaceuticals, Inc. (INO): $9.15; -9%.

Pipeline updates below:

Pipeline Database Updates

Drug Price Stage Catalyst Market Cap

CASI – CASI Pharmaceuticals Inc.
CID-103
Multiple myeloma

$1.51
+0.04  +3%
Phase 1 Phase 1 initiation of dosing announced June 10, 2021.
$211.1 million

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

$18.34
-0.52  -3%
Phase 2b Phase 2b trial initiation announced June 10, 2021. Phase 1b/2a topline data from the paired-biopsy, open-label histology cohort due by end of 2021.
$367.9 million

GKOS – Glaukos Corporation
iDose TR
Glaucoma

$84.79
+0.91  +1%
Phase 3 Phase 3 completion of enrollment announced June 11, 2021. NDA filing due 2022.
$3.9 billion

IBRX – ImmunityBio Inc.
Anktiva (N-803)
HIV

$13.66
-0.11  -1%
Phase 2 Phase 2 trial initiation announced June 10, 2021.
$5.2 billion

OCGN – Ocugen, Inc.
COVAXIN
COVID-19 vaccine

$7.41
+0.01  +0%
Phase 3 Phase 3 interim data from partner Bharat Biotech noted efficacy rate of 78% - April 21, 2021. BLA filing to be submitted instead of FDA Emergency Use Authorization Application - June 10, 2021.
$1.5 billion

PGEN – Precigen Inc.
AG019 ActoBiotics
Type 1 diabetes

$6.30
+0.03  +0%
Phase 1/2 Primary endpoint assessing safety and tolerability met for both Phase 1b monotherapy and Phase 2a combination therapy portions of study - June 10, 2021.
$1.3 billion

TYME – Tyme Technologies Inc.
SM-88 (Tyme-88-Panc)
Pancreatic cancer

$1.27
0.00  0%
Phase 2/3 Phase 2/3 enrollment to be terminated and trial will be closed down. Company citing slower than expected enrollment for the decision - June 10, 2021.
$218.7 million

TYME – Tyme Technologies Inc.
SM-88 (HopES)
Ewing’s sarcoma

$1.27
0.00  0%
Phase 2 Phase 2 enrollment to be completed 1H 2022.
$218.7 million

TYME – Tyme Technologies Inc.
SM-88 and Gemcitabine (PanCAN_Precision Promise)
Pancreatic Cancer

$1.27
0.00  0%
Phase 2/3 Phase 3 advancement determination decision 2H 2022.
$218.7 million

TYME – Tyme Technologies Inc.
SM-88 (OASIS)
HR+/HER2- breast cancer

$1.27
0.00  0%
Phase 2 Phase 2 trial initiation announced June 10, 2021. Trial status update 1H 2023.
$218.7 million

VERU – Veru Inc.
VERU-100
Prostate cancer

$8.51
+0.03  +0%
Phase 2 Phase 2 commencement of enrollment announced June 10, 2021. To be completed 2H 2021.
$678.1 million

VRTX – Vertex Pharmaceuticals Incorporated
VX-864
Alpha-1 antitrypsin (AAT) deficiency

$188.87
+1.17  +1%
Phase 2 Phase 2 data released June 10, 2021. Primary endpoint met but insufficient clinical benefit to advance program.
$48.9 billion