BPC February 08 update

​uniQure QURE +10% following positive hemophilia data

Price and Volume Movers

uniQure N.V. (NASDAQ: QURE) shares closed up 10% to $36.49 following the release of positive updated data from its ongoing Phase 2b trial of AMT-061 for the treatment of patients with severe and moderately severe hemophilia B. The data showed that levels of Factor IX (FIX) activity continue to be sustained in all three patients up to sixteen weeks after a single administration of AMT-061. Mean FIX activity increased to 38% of normal at 12 weeks after treatment.   

Celldex Therapeutics, Inc. (NASDAQ:CLDX) announced a one-for-fifteen reverse stock split of its common stock became effective today and will trade on a split-adjusted basis from Monday. Shares closed down 31% to $0.39. 

Pulmatrix, Inc. (NASDAQ: PULM) shares closed down 33% to $0.90 following news of yet a further direct offering of 1,706,484 shares at a price of $1.465 per share for gross proceeds of approximately $2.5m. Warrants to purchase up to 1,706,484 shares were also attached to the offering. 

Sienna Biopharmaceuticals, Inc. (NASDAQ:SNNA) announced that its pivotal trial of SNA-001, a topical pre-treatment to standard laser hair removal, met the primary endpoint of non-inferiority in hair reduction (-17.5 percent with SNA-001+Laser compared to -1.1 percent with vehicle+Laser following six treatment sessions). The company noted however that it will prioritise its focus on Phase 3 development of SNA-120, for the treatment of psoriasis and pruritus, and will seek a partner for further development of SNA-001. Shares traded up as much as 29% early in the session, before paring back gains to end the day up 5% to $2.84.

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

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


Eyenovia, Inc. (EYEN): $6.30; +28%.

Mustang Bio, Inc. (MBIO): $5.61; +26%.

NantKwest, Inc. (NK): $1.35; +26%.

Selecta Biosciences, Inc. (SELB): $2.09; +19%.

Arrowhead Pharmaceuticals, Inc. (ARWR): $15.66; +18%.


OHR Pharmaceutical, Inc. (OHRP): $2.14; -23%.

Gemphire Therapeutics Inc. (GEMP): $1.06; -17%.

Alpine Immune Sciences, Inc. (ALPN): $5.50; -15%.

Seattle Genetics, Inc. (SGEN): $65.02; -12%.

Avadel Pharmaceuticals plc (AVDL): $2.26; -11%.


Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

CBIO – Catalyst Biosciences Inc.
Marzeptacog alfa

Phase 2 Phase 2 final data released July 7, 2019. Primary endpoint met.
$100.4 million

IMGN – ImmunoGen Inc.
Mirvetuximab soravtansine
Cancer - ovarian cancer and relapsed endometrial cancer

Phase 3 Phase 3 top-line data released March 1, 2019 did not meet primary endpoint. Further Phase 3 trial to be initiated later in 2019 with data due 1H 2022.
$370.8 million

IMGN – ImmunoGen Inc.
Mirvetuximab soravtansine - FORWARD II
Ovarian cancer and relapsed endometrial cancer

Phase 1/2 Phase 1b/2 presentation at ASCO June 1, 2019.
$370.8 million

IMGN – ImmunoGen Inc.
Acute Myeloid Leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN)

Phase 1 Phase 1 updated data due 2019.
$370.8 million

QURE – uniQure N.V.
Hemophilia B

Phase 2b Phase 2b nine month data presented at ISTH July 5, 2019. FIX activity up to 54% of normal, with mean of 45% of normal.
$2.6 billion

SNNA – Sienna Biopharmaceuticals Inc.
Reduction of light-pigmented hair

Phase 3 Phase 3 data met non-inferiority primary endpoint - February 8, 2019. Seeking partner for further advancement.
$24.3 million