BPC May 10 update

​uniQure QURE adds 7% on further hemophilia data; Puma PBYI closes down 39% following Thursday's earnings miss

Price and Volume Movers

uniQure N.V. (NASDAQ: QURE) shares closed up 7% to $61.10 following the release of updated data from its Phase 2b trial of AMT-061, for the treatment of patients with severe and moderately severe hemophilia B. Data presented focused on Factor IX (FIX) activity in three patients for up to six months after a single administration of AMT-061. All three patients have demonstrated increasing and sustained FIX levels after the one-time administration of AMT-061, with two of the three patients now achieving FIX activity in the normal range. Mean FIX activity for the three patients at six months after administration increased to 47% (51%, 33% and 57%) of normal. An ongoing Phase 3 trial is slated to complete enrolment by the end of this year and has a current estimated primary completion date of March 2020.

Puma Biotechnology, Inc. (NASDAQ: PBYI) shares closed Friday down 39% to $18.42 following its earnings miss released late-Thursday, which noted net Nerlynx revenue for the quarter of $46m, compared to consensus of approximately $66m.

Catalyst Pharmaceuticals Inc (NASDAQ: CPRX) shares rebounded, paring back some of its recent losses, to close Friday up 16% to $3.14, but still well down on Monday’s close of $6.11. The FDA earlier in the week granted the approval of Ruzurgi to Jacobus Pharmaceutical Company, Inc. for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in patients 6 to less than 17 years of age. Catalyst Pharmaceuticals currently markets Firdapse in adults with LEMS. The company is due to report first quarter earnings on Monday.


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

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


Avedro, Inc. (AVDR): $15.69; +24%.

G1 Therapeutics, Inc. (GTHX): $25.02; +21%.

MorphoSys AG (MOR): $25.97; +18%.

Evofem Biosciences, Inc. (EVFM): $6.72; +17%.

Unity Biotechnology, Inc. (UBX): $10.21; +15%.


Verastem, Inc. (VSTM): $1.36; -28%.

Melinta Therapeutics, Inc. (MLNT): $3.45; -25%.

FibroGen, Inc. (FGEN): $36.39; -20%.

DelMar Pharmaceuticals, Inc. (DMPI): $2.88; -15%.

Gemphire Therapeutics Inc. (GEMP): $1.05; -14%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

BCLI – Brainstorm Cell Therapeutics Inc.
Amytrophic lateral sclerosis (ALS)

Phase 3 Phase 3 top-line data mid-2020.
$93.8 million

COCP – Cocrystal Pharma Inc.
Hepatitis C

Phase 2a Phase 2a initial data released January 22, 2019. 66% (SVR12). Full data to be presented in 2019.
$66.4 million

FBIO – Fortress Biotech Inc.
Severe Traumatic Brain Injury

Phase 2 Phase 2 data in adults due 2021.
$99.2 million

FBIO – Fortress Biotech Inc.
Intravenous (IV) tramadol
Postoperative pain following bunionectomy surgery

Phase 3 Phase 3 data released June 3, 2019 met primary endpoint.
$99.2 million

GALT – Galectin Therapeutics Inc.
Non-alcoholic steatohepatitis (NASH) with cirrhosis

Phase 3 Phase 3 trial to be initiated during fall of 2019 with top-line data due 4Q 2022.
$222.3 million

OCUL – Ocular Therapeutix Inc.
Glaucoma and ocular hypertension

Phase 3 Phase 3 data May 20, 2019 did not meet primary endpoint.
$227 million

PIRS – Pieris Pharmaceuticals Inc.
Anemia of chronic disease

Phase 2a Phase 2a data presented at EHA June 16, 2019.
$210.3 million

PIRS – Pieris Pharmaceuticals Inc.
HER2-positive solid tumors

Phase 1/2 Phase 1 data due 2019.
$210.3 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

STML – Stemline Therapeutics Inc.
SL-401 (Elzonris)
Cancer- adult relapsed or refractory acute myeloid leukemia (AML)

Phase 2 Phase 2 regimen expected 2H 2019.
$550.9 million

STML – Stemline Therapeutics Inc.
SL-401 (Elzonris)
Chronic myelomonocytic leukemia (CMML) - cancer

Phase 2 Phase 2 trial to be expanded to include pivotal cohort, to commence enrolment later in 2019.
$550.9 million

TGTX – TG Therapeutics Inc.
TG-1101 and TGR-1202 - UNITY-CLL study
Chronic Lymphocytic Leukemia (CLL) and non-Hodgkin's Lymphoma (NHL)

Phase 3 Phase 3 PFS data possibly due 2019.
$720.3 million

TRVN – Trevena Inc.
Oliceridine (TRV130)
Moderate to severe acute pain

CRL CRL issued November 2, 2018. Additional QT interval data required - trial to commence June, 2019.
$97.9 million

WVE – Wave Life Sciences Ltd.
Huntington’s disease

Phase 1/2 Phase 1b/2a top line data due by YE 2019.
$925.1 million

WVE – Wave Life Sciences Ltd.
Suvodirsen (WVE-210201)
Duchenne muscular dystrophy - Exon 51

Phase 1 Phase 1 interim efficacy data from open-label trial due 2H 2019. Phase 2/3 trial initiation announced June 27, 2019.
$925.1 million