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DXR On Fire, IONS Ups Stake In AKCA, HAIR Grows, FLKS To Report Data This Month

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

1. Daxor Corp. (DXR)

Gained 71.16% to close Thursday's (Mar.15) trading at $13.35. This is the second straight day of double-digit gain for the stock.

The Company manufactures and markets FDA-cleared BVA-100 blood volume analyzer, which is used in conjunction with a single-use diagnostic kit.

Recent news:

-- On Mar.14, 2018, the Company announced new data from its study showing that patient care individualized by its Precision Blood Volume Analysis (BVA) reduces heart failure readmissions by 56% and mortality by over 80%.

2. Akcea Therapeutics Inc. (AKCA)

Gained 38.35% to close Thursday's trading at $28.14.

News: Akcea has gained worldwide rights to Inotersen and IONIS-TTR-L Rx from Ionis Pharmaceuticals Inc. (IONS).

Inotersen is an investigational drug for the treatment of patients with hereditary TTR amyloidosis (hATTR). It is currently under regulatory review for marketing authorization in the U.S. and EU. The FDA's decision on Inotersen is expected by July 6, 2018.

IONIS-TTR-L Rx, now known as AKCEA-TTR-LRx, is being developed for hereditary and wild-type forms of ATTR. AKCEA-TTR-LRx is planned to enter clinical development this year.

As part of the deal, Ionis will purchase $200 million of Akcea common stock priced by reference to a recent trading average, and Ionis' ownership in Akcea will increase by 7%, from 68% to 75%, totaling 64,114,545 shares.

The transaction is potentially worth up to approximately $1.7 billion to Ionis plus profit sharing payments.

3. Flex Pharma Inc. (FLKS)

Gained 27.34% to close Thursday's trading at $7.36.

Flex Pharma is engaged in developing innovative and proprietary treatments for cramps and spasticity associated with the severe neurological diseases of amyotrophic lateral sclerosis, multiple sclerosis and peripheral neuropathies such as Charcot-Marie-Tooth (CMT).

The Company's lead drug candidate is FLX-787.

Clinical Trials & Near-term Catalysts:

-- Top-line results from a phase II placebo-controlled, cross-over study testing FLX-787 in patients who suffer from spasticity, cramps and spasms as a consequence of multiple sclerosis, which is ongoing in Australia, are expected to be reported at the end of March.
-- A phase II trial evaluating FLX-787 in the United States in Charcot-Marie-Tooth patients who suffer from painful, debilitating cramps, dubbed COMMIT, is underway. Top line data from this study is expected in early 2019.
-- A phase II trial evaluating FLX-787 in the US in patients with motor neuron disease patients who suffer from painful, debilitating cramps, known as COMMEND, is ongoing - with top line results expected by early 2019.

4. Adaptimmune Therapeutics plc (ADAP)

Gained 18.78% to close Thursday's trading at $10.94.

News: The Company announced compelling clinical data from its NY-ESO Program, which is partnered with GSK.

There have been three partial responses (two confirmed and one to be confirmed), and one stable disease in the first four patients dosed with NY-ESO SPEAR T-cells in myxoid/round cell liposarcoma (MRCLS).

You can find more about ADAP in our Company Spotlight column.

5. ArQule Inc. (ARQL)

Gained 15.56% to close Thursday's trading at $2.08.

News: No news

Clinical Trials:

-- ARQ 087 (Derazantinib), under phase III trial in Intrahepatic Cholangiocarcinoma (iCCA), is ongoing.
-- ARQ 531, proposed for heme malignancies, is under phase 1a/b testing.
-- Miransertib (ARQ 092), for Proteus syndrome, is under Phase I/II trial.
-- ARQ 751, for solid tumors, is under Phase 1a study.

Near-term Catalysts:

-- Data from a phase 1b trial for Miransertib in combination with Anastrozole in PIK3CA or AKT1-mutant endometrial and ovarian cancers will be presented on April 15, 2018.
-- Results of a phase I dose escalation study of ARQ 751 in adult subjects with advanced solid tumors will be presented on April 15, 2018.

6. Presbia PLC (LENS)

Gained 15.47% to close Thursday's trading at $3.06.

News: No news

Pipeline:

The Company's lead product is Presbia Flexivue Microlens, a revolutionary optical lens implant for treating presbyopia, the age-related loss of near vision. The Presbia Flexivue Microlens is currently approved for sale in 42 countries around the world, and has also received CE Mark approval in Europe. It is not approved in the U.S. yet.

A staged pivotal U.S. clinical trial for the Presbia Flexivue Microlens commenced in 2014.

7. Restoration Robotics Inc. (HAIR)

Gained 15.23% to close Thursday's trading at $6.81.

News: No news

Restoration Robotics is a medical device company developing and commercializing the ARTAS Robotic Hair Restoration System.

Recent event:

On March 5, 2018, the Company reported financial results for the fourth quarter and full year ended December 31, 2017.

Fourth quarter revenue was $5.9 million, up 13% year-over-year, and full year revenue was $21.3 million, up 37% year-over-year.

8. RXi Pharmaceuticals Corp. (RXII)

Gained 12.09% to close Thursday's trading at $5.10.

News: No news

Clinical Trials & Near-term Catalysts:

-- The lead compound is RXI-109, which successfully completed a phase II trial for dermal scarring last December.

RXI-109 is also being tested in a phase 1/2 study in an ophthalmology indication namely in patients with advanced, wet age-related macular degeneration and associated retinal scarring. Readouts from this trial are expected this month.

-- Also in the pipeline are Samcyprone, which is being evaluated in a phase IIa clinical trial in subjects with at least one cutaneous, plantar or periungual wart, and RXI-231, a cosmetic ingredient in a proprietary gel formulation designed to aid in the reduction of pigmentation to improve skin appearance.

The Company expects to share full read-outs from the Phase 2a clinical trial of Samcyprone for the treatment of cutaneous warts in Q1-Q2 2018 time period.

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