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.

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

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

ADVANCERS:

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%.

DECLINERS:

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.
NurOwn
Amytrophic lateral sclerosis (ALS)

Phase 3 Phase 3 top-line data mid-2020.
$111.2 million

COCP – Cocrystal Pharma Inc.
CC-31244
Hepatitis C

Phase 2a Phase 2a initial data released January 22, 2019. 66% (SVR12). Full data to be presented in 2019.
$78.7 million

FBIO – Fortress Biotech Inc.
CEVA101
Severe Traumatic Brain Injury

Phase 2 Phase 2 data in adults due 2020.
$126.5 million

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

Phase 3 Phase 3 data due mid-2019
$126.5 million

GALT – Galectin Therapeutics Inc.
GR-MD-02
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.
$206.1 million

OCUL – Ocular Therapeutix Inc.
OTX-TP
Glaucoma and ocular hypertension

Phase 3 Phase 3 data May 20, 2019 did not meet primary endpoint.
$111.8 million

PIRS – Pieris Pharmaceuticals Inc.
PRS-080
Anemia of chronic disease

Phase 2a Phase 2a data to be presented at EHA June 16, 2019.
$180.5 million

PIRS – Pieris Pharmaceuticals Inc.
PRS-343
HER2-positive solid tumors

Phase 1/2 Phase 1 data due 2019.
$180.5 million

QURE – uniQure N.V.
AMT-061
Hemophilia B

Phase 2b Phase 2b 26-week follow-up data presented at HTRS May 10, 2019. Noted Mean FIX of 47% of normal.
$2.4 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.
$659.6 million

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

Phase 2 Presentation at ASCO June 3, 2019, 8am CT.
$659.6 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.
$635.8 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.
$121 million

WVE – Wave Life Sciences Ltd.
WVE-120102
Huntington’s disease

Phase 1/2 Phase 1b/2a top line data due by YE 2019.
$858.2 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 to be initiated July 2019.
$858.2 million