QURE In The Pink, ARNA Heartens Investors, ELOX Up Over 40% This Month

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The following are some of today's top gainers in the pharma/biotech sector.

1. uniQure N.V. (QURE)

Gained 35.66% to close Thursday's (Nov.15) trading at $30.93.

News: The Company announced promising results from its phase IIb dose-confirmation study of AMT-061, an investigational gene therapy, for the treatment of patients with severe and moderately severe hemophilia B.

Individuals with hemophilia B have insufficient levels of a blood protein called Factor IX.

The study enrolled three patients with severe hemophilia, and they received a single intravenous infusion of AMT-061. The data reported today shows that therapeutic levels of Factor IX (FIX) activity have been achieved and sustained in all three patients at six weeks after a single administration of AMT-061.

Patients in the study will be followed for 52 weeks to assess FIX activity, bleeding rates and usage of FIX replacement therapy, and will be monitored for five years to evaluate the safety of AMT-061, according to the Company.

2. Translate Bio Inc. (TBIO)

Gained 24.03% to close Thursday's trading at $7.33.

News: No news

Anticipated Milestones:

-- Initiate multiple-ascending dose portion of a phase I/II clinical of MRT5005 for cystic fibrosis by year end 2018.
-- Initiate phase I/II clinical trial of MRT5201 in ornithine transcarbamylase (OTC) deficiency in the first half of 2019.
-- Identify lead preclinical candidates for additional lung and liver disease targets.

3. Arena Pharmaceuticals Inc. (ARNA)

Gained 21.62% to close Thursday's trading at $39.55.

News: Arena Pharma and United Therapeutics Corp.(UTHR) have entered into a global license agreement for Arena's phase III drug candidate, Ralinepag, in development for the treatment of pulmonary arterial hypertension (PAH).

The agreement entitles Arena to receive $800 million upfront, low double-digit tiered royalties, plus up to $400 million in milestone payments. United Therapeutics will receive exclusive, worldwide rights to Ralinepag.

4. Onconova Therapeutics Inc. (ONTX)

Gained 14.86% to close Thursday's trading at $4.25. A one-for-fifteen reverse stock split of its common stock was implemented on September 26, 2018.

News: No news

Pipeline & Near-term Catalysts:

-- A phase III pivotal trial with intravenous Rigosertib for high-risk second-line patients with Myelodysplastic Syndromes, dubbed INSPIRE, is underway. Top-line data from this study is expected in the second half of 2019.
-- Updated efficacy and safety data from phase II combination trial of oral Rigosertib with injectable Azacitidine is scheduled for presentation at the upcoming 60th American Society of Hematology meeting on December 1, 2018.

5. Eloxx Pharmaceuticals Inc. (ELOX)

Gained 13.42% to close Thursday's trading at $16.99.

News: No news

Near-term Catalysts:

-- Report top line data from phase II clinical trial ELX-02 in cystic fibrosis in 2019.
-- Report top line data from phase II clinical trial ELX-02 in cystinosis in 2019.

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