ASND On Track, EDAP On Strong Footing, Kyowa Kirin Gets It Right The Second Time

(RTTNews) - Today's Daily Dose brings you news about EDAP's strong financial results, anticipated milestones of Ascendis Pharma; FDA approval of a new add-on drug to treat "Off episodes" in Parkinson's and the clinical trial catalysts of Mirum Pharma.

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Ascendis Pharma A/S (ASND) has reported a wider loss and higher revenue for the second quarter ended June 30, 2019.

Net loss widened to €58.9 million or €1.25 per share for the second quarter of 2019 from €22.8 million or €0.55 per share for the same period in 2018.

Revenue for the recent second quarter was €3.2 million compared to €18 thousand in the same quarter of 2018. The increase reflects the recognition of revenue related to the Company's strategic investment in VISEN Pharmaceuticals.

The Company is planning to submit a Biologics License Application to the FDA in the first half of 2020 and a marketing application in Europe in the second half of 2020 for TransCon hGH.

TransCon hGH is being developed for the treatment of pediatric growth hormone deficiency or GHD.

Ascendis is working in collaboration with VISEN Pharmaceuticals to initiate a phase III trial of TransCon hGH later this year in subjects with pediatric GHD in China and plans to initiate a global program for adult GHD in 2020.

A phase II study of TransCon PTH in adults with hypoparathyroidism, dubbed PaTH Forward, was initiated last month. Top-line results from this study are anticipated in the fourth quarter of 2019.

ASND closed Wednesday's trading at $111.70, down 0.71%.

EDAP TMS SA (EDAP) has reported strong financial results for the second quarter of 2019.

Net income for the second quarter 2019 was EUR 1.4 million ($1.6 million) or earnings of EUR 0.05 per share compared to a net loss of EUR 0.8 million ($0.9 million), or a loss of EUR 0.03 per share in the year-ago period. Total revenue for the second quarter of 2019 was EUR 12.5 million ($14.0 million), a 45.1% increase compared to EUR 8.6 million ($10.2 million) in the year-ago quarter.

The Company noted that it is confident that this type of growth and consistency in its results could be maintained going forward.

EDAP closed Wednesday's trading at $3.42, up 7.55%.

The FDA has approved Japan-based Kyowa Kirin's Nourianz as an add-on treatment to levodopa/carbidopa in adults with Parkinson's disease who experience "OFF" episodes.

Parkinson's disease is a neurodegenerative brain disorder, in which during the course of the disease, the brain slowly stops producing a neurotransmitter called dopamine. As the dopamine level falls, it becomes difficult for the individual to regulate his/her movements, body and emotions. Levodopa/carbidopa is the most commonly prescribed treatment for Parkinson's disease. There are times when a patient's medications do not work well, and it causes an increase in Parkinson's disease symptoms, such as tremor and difficulty walking. These are known as "off" episodes.

In February 2008, the FDA refused to approve Nourianz and asked the Company to submit additional clinical follow-up data as well as an overall summary of nonclinical mineralization findings. The drug was approved in Japan in May 2013 and is marketed there under brand name Nouriast.

Parkinson's disease is the second-most common neurodegenerative disorder in the U.S. after Alzheimer's disease. An estimated 50,000 Americans are diagnosed with PD each year, and about one million Americans have the condition, according to the National Institutes of Health.

Mirum Pharmaceuticals Inc. (MIRM), which made its NASDAQ debut in July of this year, has a couple of catalysts to watch out for in the coming months.

The Company's lead drug candidate is Maralixibat, a novel, oral, minimally-absorbed agent, which is currently in development for the treatment of pediatric patients with progressive familial intrahepatic cholestasis [PFIC] and alagille syndrome [ALGS].

A phase III trial of Maralixibat in children with Progressive Familial Intrahepatic Cholestasis, dubbed MARCH-PFIC, is underway, with top-line data readout from the trial expected in late-2020.

The Company expects to meet with the FDA in the fourth quarter of 2019 to continue discussions regarding the adequacy of its phase IIb data to support an NDA submission for Maralixibat for pruritus associated with alagille syndrome, with an update anticipated to be provided in January 2020.

MIRM closed Wednesday's trading at $13.53, down 4.38%.