BPC November 19 update

UniQure QURE hemophilia trial meets primary endpoint +8%; Eli Lilly LLY receives Covid-19 EUA

Price and Volume Movers

uniQure N.V. (NASDAQ: QURE) announced positive top-line data from its pivotal, Phase 3 HOPE-B gene therapy trial of etranacogene dezaparvovec for the treatment of patients with severe and moderately severe hemophilia B. Factor IX (FIX) activity increased rapidly after dosing from ≤2% to a mean of 37.2 percent at 26 weeks, meeting the first primary endpoint. 72% of patients (39/54) reported no bleeding events after dosing. Data will be presented at the American Society of Hematology (ASH) conference on December 8, 2020. Shares closed up 8% to $46.16.

Mesoblast Limited (NASDAQ:MESO) announced a collaboration agreement with Novartis for the development and commercialization of remestemcel-L, with an initial focus on the development of the treatment of acute respiratory distress syndrome (ARDS). Novartis will make a $50m upfront payment including $25m in equity. Shares are trading up 19% after hours to $14.05.

Aptevo Therapeutics Inc. (NASDAQ:APVO) shares closed up 29% to $45.69 following news late-Wednesday that Tang Capital Management has offered to acquire the company for $50 per share in cash.

Corcept Therapeutics Incorporated (NASDAQ:CORT) shares closed up 26% to $23.44 on news also announced after hours Wednesday that the U.S. Patent and Trademark Office upheld its patent for Korlym following a challenge from Teva Pharmaceuticals.

AVROBIO, Inc. (NASDAQ:AVRO) announced after hours that it intends to offer and sell $75m of its common stock in an underwritten public offering. Shares are trading down 6% to $16.00 after hours.

Oragenics, Inc. (NYSE:OGEN) also announced a proposed public offering. Shares, which closed up 13% during normal trading hours, have had their gains erased after hours, currently trading down 12% to $0.47.

Eli Lilly and Company (NYSE:LLY) was issued an Emergency use authorization (EUA) from the FDA for its drug baricitinib, in combination with remdesivir, for the treatment of COVID-19 in hospitalized patients requiring supplemental oxygen, invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO).


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:


22nd Century Group, Inc. (XXII): $1.50; +25%.

Liminal BioSciences Inc. (LMNL): $5.51; +25%.

Infinity Pharmaceuticals, Inc. (INFI): $2.12; +20%.

Tricida, Inc. (TCDA): $7.52; +18%.

SCYNEXIS, Inc. (SCYX): $6.68; +17%.


Genfit SA (GNFT): $4.22; -12%.

Kazia Therapeutics Limited (KZIA): $12.00; -11%.

Kintara Therapeutics, Inc. (KTRA): $1.50; -11%.

BeyondSpring Inc. (BYSI): $10.31; -11%.

MeiraGTx Holdings plc (MGTX): $12.92; -10%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ADAP – Adaptimmune Therapeutics plc
Synovial sarcoma or myxoid/round cell liposarcoma (MRCLS)

Phase 2 Phase 2 enrolment to be completed 1Q 2021.
$775.4 million

APLS – Apellis Pharmaceuticals Inc.
Pegcetacoplan (MERIDIAN)
Amyotrophic lateral sclerosis (ALS)

Phase 2 Phase 2 initiation of dosing announced November 19, 2020.
$3.6 billion

ARAV – Aravive Inc.
Platinum Resistant Ovarian Cancer (PROC)

Phase 3 Phase 3 trial to be initiated in 1Q 2021. Interim analysis due 1Q 2022.
$93.5 million

BPTH – Bio-Path Holdings Inc.
Refractory/relapsed lymphoma and chronic lymphocytic leukemia

Phase 1 Phase 1 initiation of dosing announced November 19, 2020.
$16.1 million

CDAK – Codiak BioSciences Inc.
Solid tumors

Phase 1/2 Phase 1/2 safety, biomarker and preliminary efficacy data by mid-2021.
$244.2 million

CDAK – Codiak BioSciences Inc.
Cutaneous T cell lymphoma

Phase 1 Phase 1 initial safety data due year end 2020. Biomarker and preliminary efficacy results in CTCL patients of due mid-2021.
$244.2 million

ENTX – Entera Bio Ltd.

Phase 2b Phase 2b or Phase 3 trial to be initiated in 2021 or 2022.
$22.4 million

INCY – Incyte Corporation
Baricitinib and remdesivir

Approved Emergency use authorization (EUA) issued by FDA November 19, 2020.
$18.5 billion

MESO – Mesoblast Limited
Heart failure

Phase 3 Phase 3 data due 4Q 2020.
$1.8 billion

MESO – Mesoblast Limited
Rexlemestrocel (MPC-06-ID)
Chronic low back pain

Phase 3 Phase 3 data due 4Q 2020.
$1.8 billion

NCNA – NuCana plc
NUC-7738 (NuTide:701)
Solid tumors

Phase 1 Phase 1 updated data due 2021.
$233.4 million

QURE – uniQure N.V.
AMT-061 (HOPE-B)
Hemophilia B

Phase 3 Phase 3 top-line data to be presented at ASH December 8, 2020. Trial met primary endpoint with mean Factor IX activity of 37% of normal at 26 weeks.
$2.1 billion

RDHL – Redhill Biopharma Ltd.

Phase 2/3 Phase 2/3 global trial to continue as planned following review by independent Data and Safety Monitoring Board (DSMB). Top-line data 1Q 2021.
$333.2 million

VBIV – VBI Vaccines Inc.
Recurrent glioblastoma multiforme (rGBM)

Phase 1/2 Phase 2a data presented November 19, 2020. 2/10 partial responses.
$829 million