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 due 4Q 2020.
$239.4 million

COCP – Cocrystal Pharma Inc.
Hepatitis C

Phase 2a Phase 2a updated data presented AASLD November 10, 2019.
$48 million

FBIO – Fortress Biotech Inc.
Severe Traumatic Brain Injury

Phase 2 Phase 2 data in adults due 2021.
$249.1 million

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

PDUFA PDUFA date October 10, 2020.
$249.1 million

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

Phase 3 Phase 3 trial to be initiated 2Q 2020 with top-line data due 4Q 2022.
$163.1 million

OCUL – Ocular Therapeutix Inc.
Glaucoma and ocular hypertension

Phase 3 Phase 3 data May 20, 2019 did not meet primary endpoint.
$434.4 million

PIRS – Pieris Pharmaceuticals Inc.
Anemia of chronic disease

Phase 2a Phase 2a data presented at EHA June 16, 2019.
$182.1 million

PIRS – Pieris Pharmaceuticals Inc.
HER2-positive solid tumors

Phase 1/2 Phase 1 complete data to be presented 2H 2020.
$182.1 million

QURE – uniQure N.V.
Hemophilia B

Phase 2b Phase 2b one-year data presented at EAHAD February 7, 2020.
$2.8 billion

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

Phase 2 Phase 2 data due 2020.
$638 million

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

Phase 2 Phase 2 update due 4Q 2020 / 1Q 2021.
$638 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 primary endpoint met. Trial to be stopped due to superior efficacy - May 5, 2020.
$2.1 billion

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

PDUFA PDUFA date August 7, 2020.
$140.4 million

WVE – Wave Life Sciences Ltd.
Huntington’s disease

Phase 1/2 Phase 1b/2a top line data released December 30, 2019 noted statistically significant reduction of 12.4% (p<0.05) in mutant huntingtin (mHTT) protein but no difference in total HTT (tHTT) protein. 32 mg data due 2H 2020.
$336 million

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

Phase 2/3 Phase 2/3 development discontinued - December 2020.
$336 million