BPC June 14 update

ArQule ARQL shares rally 30% on promising early-stage leukemia data

Price and Volume Movers

ArQule, Inc. (Nasdaq: ARQL) shares rallied following the release of preliminary data from its Phase 1 trial of ARQ 531 in patients with relapsed or refractory hematologic malignancies at the 2019 European Hematology Association (EHA) meeting. The company noted an overall response rate (ORR) of 66% (4 responses in 6 evaluable patients) was observed in heavily pretreated relapsed or refractory chronic lymphocytic leukemia (CLL) patients. Shares closed up 30% to $8.20.

Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) announced positive data from its Phase 1 trial of REGN1979 in patients with relapsed or refractory (R/R) B-cell non-Hodgkin lymphoma (B-NHL). The trial exhibited a 93% overall response (13 of 14 patients) and 71% complete response rates (10 of 14 patients) in follicular lymphoma patients. In diffuse large B-cell lymphoma (DLBCL) patients a 57% overall response rate (4 of 7 patients) was noted.

Global Blood Therapeutics, Inc. (GBT) (Nasdaq: GBT) announced data from its Phase 3 HOPE trial of voxelotor in patients ages 12 and older with sickle cell disease (SCD). 51.1% of patients on the highest dose exhibited a hemoglobin response in the intention-to-treat (ITT) analyses, compared with 6.5% for placebo and 59.5% in per-protocol (PP) analyses (9.2% placebo), an improvement that was statistically significant. However, while there were numerically fewer pain vaso-occlusive crises (VOCs), the difference for this key secondary endpoint was not statistically significant, which appears to be the reason behind today’s selloff, with shares closing down 6% to $53.98.

Ampio Pharmaceuticals, Inc. (NYSE MKT: AMPE) shares closed up 11% to $0.70 on news that the FDA issued a special protocol assessment (SPA) for its upcoming Phase 3 trial of Ampion in patients with osteoarthritis of the knee. However, shares have currently lost 26% to $0.51 after hours on news it intends to sell shares of in an underwritten public offering.

Enanta Pharmaceuticals, Inc. (NASDAQ:ENTA) announced top-line data from its Phase 2a trial of EDP-938 in healthy adults infected with respiratory syncytial virus (RSV). The trial achieved highly statistically significant (p<0.001) reductions in viral load and in resolution of clinical symptoms compared to placebo. A Phase 2b trial will be initiated by the end of this year. Shares, however, closed Friday lower 12% to $85.81.

SELLAS Life Sciences Group, Inc. (Nasdaq: SLS) announced the pricing of an underwritten public offering of 26,367,200 shares, with accompanying warrants, at a combined price of $0.15 per share. Shares closed down 57% to $0.16.


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

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


Outlook Therapeutics, Inc. (OTLK): $2.32; +21%.

Avadel Pharmaceuticals plc (AVDL): $3.19; +20%.

KemPharm, Inc. (KMPH): $1.59; +11%.

Verastem, Inc. (VSTM): $1.40; +10%.

Aeterna Zentaris Inc. (AEZS): $3.02; +10%.


Novus Therapeutics, Inc. (NVUS): $1.05; -20%.

NovaBay Pharmaceuticals, Inc. (NBY): $1.66; -20%.

Seres Therapeutics, Inc. (MCRB): $2.38; -19%.

Unum Therapeutics Inc. (UMRX): $2.43; -14%.

Applied Therapeutics, Inc. (APLT): $8.55; -12%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

AMPE – Ampio Pharmaceuticals Inc.
Osteoarthritis of the Knee

Phase 3 As of July 24, 2019 enrolment has reached approximately 30%.
$66.6 million

ARQL – ArQule Inc.
ARQ 531
B-cell malignancies

Phase 1 Phase 1 presentation at ASH December 9, 2019 at 6pm EST.
$1.2 billion

BGNE – BeiGene Ltd.
Zanubrutinib (BGB-3111) - ASPEN
Waldenström’s macroglobulinemia

Phase 3 Phase 3 top-line data due 4Q 2019.
$11.4 billion

BGNE – BeiGene Ltd.
Zanubrutinib (BGB-3111)
Waldenström’s macroglobulinemia

Phase 1 Phase 1 updated data at EHA June 2019 noted 92% ORR.
$11.4 billion

BLUE – bluebird bio Inc.
LentiGlobin - HGB-207 Northstar-2
non-β0/β0 transfusion-dependent thalassemia (TDT)

Phase 3 Phase 3 presentation of data at ASH December 9, 2019, 6pm.
$4.4 billion

BPMC – Blueprint Medicines Corporation
Avapritinib BLU-285
PDGFRα driven Gastrointestinal stromal tumors (GIST) - 4th line

PDUFA PDUFA date under priority review February 14, 2020. Likely to be extended due to requested data from Voyager trial (2Q 2020).
$3.9 billion

ENTA – Enanta Pharmaceuticals Inc.
Respiratory Syncytial Virus

Phase 2b Phase 2b trial has been initiated - noted November 21, 2019.
$1.3 billion

GBT – Global Blood Therapeutics Inc.
Sickle cell disease

Approved FDA Approval announced November 25, 2019.
$4.8 billion

GTHX – G1 Therapeutics Inc.
Small-cell lung cancer

Phase 2 Phase 2 patient-reported outcomes (PRO) presented at MASCC meeting June 21, 2019.
$860.6 million

KRYS – Krystal Biotech Inc.
Transglutaminase-1 Deficient Autosomal Recessive Congenital Ichthyosis

Phase 1/2 Phase 1/2 interim data due 1H 2020.
$1.1 billion

KURA – Kura Oncology Inc.
Relapsed or refractory peripheral T-cell lymphoma

Phase 2 Phase 2 data due at ASH December 8, 2019 at 1:15pm ET.
$707.4 million

REGN – Regeneron Pharmaceuticals Inc.
Follicular Lymphoma

Phase 1 Phase 1 updated data due at ASH December 9, 2019, 4PM.
$40.9 billion