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PTIE Skyrockets On Remoxy Data, ASND On An Ascent, There Is HOPE For GBT

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

1. Pain Therapeutics Inc. (PTIE)

Gained 114.22% to close Tuesday's (Jan.9) trading at $9.04. The stock had touched an intraday high of $12.80.

News: The Company announced positive results from a human abuse potential study of its late-stage drug candidate, REMOXY.

REMOXY ER is a proprietary, abuse-deterrent, extended-release oral formulation of oxycodone. The proposed indication for this drug candidate is for "the management of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate.

According to the study results, in non-dependent, recreational opioid users, nasal administration of REMOXY resulted in significantly lower abuse potential compared to immediate-release (IR) oxycodone. All study subjects reported reduced 'Drug Liking' 'Take Drug Again' and 'Drug High' for REMOXY compared to oxycodone IR.

REMOXY ER was issued a Complete Response Letter by the FDA in September 2016.

Near-term Catalyst:

-- Resubmit NDA for REMOXY in Q1, 2018.

2. Global Blood Therapeutics Inc. (GBT)

Gained 19.07% to close Tuesday's trading at $47.15.

News: The Company has been granted FDA Breakthrough Therapy Designation for its investigational drug Voxelotor for treatment of sickle cell disease.

Voxelotor is being studied in a phase III trial in patients aged 12 and older with sickle cell disease, dubbed HOPE, and in phase IIa study in pediatric patients (age 6 to 17) with sickle cell disease, known as HOPE-KIDS 1.

Near-term Catalysts:

-- The Company expects to report top-line data from Part A of the Phase 3 HOPE Study in the first half of 2018.
-- Report top-line data from Part B of the Phase 3 HOPE Study in the first half of 2019.

3. Nektar Therapeutics (NKTR)

Gained 18.52% to close Tuesday's trading at $68.03.

News: The Company made a presentation at the 36th Annual J.P. Morgan Healthcare Conference in San Francisco on Tuesday, January 9, 2018.

Near-term Catalysts:

First Half of 2018

-- Initiate first Phase 1 clinical trial of NKTR-262 with NKTR-214 in cancer patients (solid tumors).
-- Initiate Phase 1/2 multiple-ascending dose trial of NKTR-358 in patients with lupus.
-- Submit NDA for NKTR-181 in chronic pain.
-- Initial data from phase II expansion part of the PIVOT study (NKTR-214 with Opdivo) in patients with melanoma, nonsmall cell lung cancer, renal cell carcinoma, bladder cancer and triple-negative breast cancer.
-- Initial data from sarcoma investigator-sponsored trial of NKTR-214 with Opdivo.

Second Half of 2018

-- Data from phase I/II PROPEL clinical trial of NKTR-214 with Tecentriq or Keytruda in patients with bladder and non-small cell lung cancer.
-- Data from first-in-human Phase 1 single-ascending dose clinical trial of NKTR-358 presented at major medical meeting.
-- Initial data from Phase 1 trial of NKTR-262 with NKTR-214.
-- IND Filing for NKTR-255 (IL-15 Receptor Agonist).

4. Abaxis Inc. (ABAX)

Gained 16.27% to close Tuesday's trading at $61.52.

News: The Company reported preliminary financial results for the third fiscal quarter ended December 31, 2017.

For the quarter ended December 31, 2017, revenue is expected to be in the range of $59.0 million to $60.0 million, well above Thomson Reuters consensus estimate of $56.42 million. The Company had reported revenue of $52.8 million in the comparable quarter ended December 31, 2016.

Upcoming Event:

Abaxis plans to announce its detailed earnings results for the third quarter of fiscal 2018 on January 25, 2018.

5. Alder Biopharmaceuticals Inc. (ALDR)

Gained 15.46% to close Tuesday's trading at $17.55. This is the second straight day of gain for the stock.

Recent event:

-- On January 8, 2018, the Company announced that its phase III trial with Eptinezumab for chronic migraine prevention, dubbed Promise 2, met primary and secondary endpoints.

Near-term Catalyst:

-- Submit BLA for Eptinezumab for chronic migraine prevention to the FDA in the second half of 2018.

6. MyoKardia Inc. (MYOK)

Gained 15.13% to close Tuesday's trading at $47.55.

News: No news

Clinical Trials & Near-term Catalysts:

The Company's lead product candidate is Mavacamten (formerly MYK-461), which is under phase II testing for symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
Data from the first cohort of the PIONEER-HCM study were reported in August 2017.
A pivotal trial of Mavacamten in symptomatic, obstructive hypertrophic cardiomyopathy, dubbed EXPLORER-HCM, is expected to be initiated in the second quarter of 2018.

The potential of Mavacamten is also being explored in patients with non-obstructive hypertrophic cardiomyopathy, and a phase II trial, dubbed MAVERICK-HCM, is expected to begin in the first quarter of 2018.

Also in the pipeline is MYK-491, being developed for the treatment of genetic dilated cardiomyopathy.

A phase I single-ascending dose trial of MYK-491 in healthy volunteers is underway, and topline data from this study are expected to be announced in early 2018.

7. Ascendis Pharma A/S (ASND)

Gained 13.47% to close Tuesday's trading at $46.00.

News: The Company provided an update on its rare disease endocrinology pipeline and presented initial phase 1 data for TransCon PTH, which is in development as a potential treatment for hypoparathyroidism (HP).

Clinical Trials & Near-term Catalysts:

The phase III trial of TransCon Growth Hormone for pediatric growth hormone deficiency, dubbed heiGHt, has randomized over 160 subjects, exceeding its target enrollment.

Top-line results from the heiGHt study are anticipated to be available in the first quarter of 2019.

Ascendis Pharma is also conducting the fliGHt Trial to evaluate TransCon Growth Hormone in patients who have previously been treated with daily growth hormone therapy as part of the pivotal TransCon Growth Hormone phase III program. Enrollment in the fliGHt Trial is expected to be completed during the third quarter of 2018.

The other investigational products in the pipeline are TransCon PTH, a long-acting prodrug of parathyroid hormone for hypoparathyroidism currently in a phase 1 trial, and TransCon CNP, a long-acting prodrug of C-type Natriuretic Peptide for achondroplasia.

The Company presented encouraging pharmacokinetic data from an ongoing phase 1 trial of TransCon PTH in healthy volunteers today. TransCon PTH may be moved directly into phase 3 development in the first quarter of 2019.

The company recently initiated the regulatory process in Australia to enable its first-in-human phase 1 trial for TransCon CNP. Dosing in the phase 1 trial in healthy volunteers is expected to begin in the second quarter of 2018, with top-line data to be announced in the fourth quarter of 2018.

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