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Marinus' (MRNS) Ganaxolone Gets Orphan Drug Status in US

Marinus Pharmaceuticals, Inc. MRNS announced that the FDA has granted orphan drug designation to its key candidate, ganaxolone, for the treatment of adult and pediatric patients with CDKL5 disorder. The stock moved up 3.6% on the news.

Marinus’ share price has increased 43.6% so far this year, outperforming the Zacks classified Medical-Drugs industry’s rise of 7.2% in the same time frame.

We note that orphan drug designation is granted to drugs capable of treating diseases that affect less than 200,000 people in the U.S. The status makes ganaxolone eligible for seven years of marketing exclusivity in the U.S., following an approval for CDKL5 disorder. The designation also makes the company eligible for certain other benefits, including tax credits related to clinical trial expenses, exemption from the FDA-user fee and assistance from the FDA in clinical trial design.

Currently, ganaxolone is being evaluated in a phase II study in children with CDKL5 disorder whose data is expected in upcoming months.

The study enrolled four pediatric CDKL5 patients in the cohort, who received up to 1800 mg/kg of ganaxolone per day for an average treatment of five-months. Per the data released in Jan 2017, there was a reduction in seizure frequency compared to baseline ranging from 52% to 88% in three of these patients. Two of these patients have completed six-months of treatment and have chosen to participate in the study extension. However, one discontinued the study after four-months of treatment due to lack of efficacy.

CDKL5 is a rare genetic disorder, which predominantly affects girls and is characterized by seizures and severe neuro-developmental impairment that impacts the ability to walk and talk.

Ganaxolone is a CNS-selective GABAA modulator being developed in three different dose forms (IV, capsule, and liquid) intended to maximize therapeutic reach to adult and pediatric patients in both acute and chronic care settings.

We note that the company has completed the phase I study evaluating ganaxolone in both pediatric and adult patients. It showed that the candidate is well-tolerated in patients.

Stocks to Consider

Stocks worth considering in the healthcare sector include Regeneron Pharmaceuticals, Inc. REGN, Sanofi SNY and Enzo Biochem, Inc. ENZ.

Regeneron’s earnings estimates moved up from $10.17 to $10.52 for 2017 and from $10.90 to $12.10 for 2018, over the last 60 days. The company came up with positive earnings surprises in two of the last four quarters, with an average beat of 0.45%. Its share price is up 38.9% so far this year.

Sanofi’s earnings estimates moved up from $3.00 to $3.18 for 2017 and from $3.25 to $3.30 for 2018, over the last 30 days. The company delivered positive earnings surprises in two of the last four quarters, with an average beat of 5.10%. The stock is up 22.98% so far this year.

Enzo Biochem’s loss estimates narrowed from 12 cents to 7 cents for 2017 and from 11 cents to 3 cents for 2018, over the last 30 days. The company came up with positive earnings surprises in each of the last four quarters, with an average beat of 55.83%. Its shares are up 63.5% so far this year.

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