FDA Approves New Use For Soliris, HSTM In Good Health, BYSI Springs A Surprise

(RTTNews) - Today's Daily Dose brings you news about Alexion's rare-disease drug, Soliris, getting FDA approval for a new indication; HealthStream's stellar Q3, 2017 financial results; stock offerings of Immune Design and uniQure; two China non-equity diluting grants awarded to BeyondSpring; EnteroMedics' name change and discontinuation of Global Blood's idiopathic pulmonary fibrosis program.

Read on...

The FDA has approved Alexion Pharmaceuticals Inc.'s (ALXN) rare-disease drug, Soliris, for the treatment of adult patients with refractory generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive.

Soliris is already approved by the FDA for the treatment of two rare blood disorders namely, paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis, and for atypical hemolytic uremic syndrome.

In the European Union, the drug is also approved for the treatment of refractory gMG in adults who are anti-AchR antibody-positive. Alexion's new drug application in Japan for Soliris as a treatment for patients with anti-AchR antibody-positive refractory gMG has been accepted for review by the Japanese Ministry of Health, Labour and Welfare (MHLW).

Soliris has received Orphan Drug Designation for the treatment of patients with MG in the U.S. and EU, and for the treatment of patients with refractory gMG in Japan.

Soliris, which costs $480,000 per patient per year, generated net product sales of $1.59 billion across the world in the first half of 2017, up 17% over the year-ago period. For full year 2017, the Company expects net product sales of Soliris to range between $3.07 billion and $3.12 billion.

ALXN closed Monday's trading at $135.20, down 2.75%.

BeyondSpring Inc. (BYSI) has received two China non-equity diluting grants - one in August 2017 from the city government of Dalian, China, and the other on Sept. 30, 2017, from the Dalian Economic Development Park.

The grants will be used to further develop the drug pipeline, the Company said.

The Company's lead drug candidate is Plinabulin, a first in class immuno-oncology agent, in a Phase 3 clinical trial in non-small cell lung cancer and in a Phase 2/3 clinical program in the prevention of chemotherapy-induced neutropenia.

A Phase 3 interim analysis is planned for both indications in the first half of 2018, and the Company also expects to receive earlier-than-expected market authorization for China.

BYSI closed Monday's trading at $32.50, up 12.11%.

BrainStorm Cell Therapeutics Inc. (BCLI) has expanded its patent portfolio to include a new US patent for its NurOwn technology for Parkinson's disease and amyotrophic lateral sclerosis.

The allowed claims cover methods of treating amyotrophic lateral sclerosis (ALS) and Parkinson's disease using mesenchymal stem cells that secrete neurotrophic factors, including glial derived neurotrophic factor (GDNF).

BCLI closed Monday's trading at $4.39, up 3.54%.

EnteroMedics Inc. (ETRM) has formally changed its name to ReShape Lifesciences Inc. (RSLS).

The new name reflects its expansion and growth into a full-scale provider of medical devices to address the continuum of care for obesity and its associated health conditions, the Company added.

ETRM closed Monday's trading at $2.00, down 2.44%.

Global Blood Therapeutics Inc. (GBT) has decided to pull the plug on its GBT440 program for the treatment of idiopathic pulmonary fibrosis as no clinically meaningful efficacy results were achieved in patients.

The decision is based on results from three proof-of-concept studies including a Phase 1 study in healthy volunteers called Basecamp and two Phase 2a studies in patients with IPF called GBT440-006 and ZEPHYR.

However, the data supports the potential of GBT440 as a disease-modifying treatment for individuals living with sickle cell disease, noted the Company.

A pivotal phase III trial of GBT440 in people with sickle cell disease, dubbed HOPE, is underway. The top-line clinical trial results from this study are expected in the first half of 2019.

GBT closed Monday's trading at $32.15, down 1.23%.

HealthStream Inc. (HSTM) has reported better-than expected financial results for the third quarter ended September 30, 2017.

Net income for the recent third quarter is $2.5 million or $0.08 per share compared to $1.2 million or $0.04 per share in the third quarter of 2016. Revenues for the third quarter of 2017 were $63.6 million, up 9% from $58.4 million in the third quarter of 2016. Analysts polled by Thomson Reuters expected the Company to earn $0.05 per share on revenue of $62.48 million.

HSTM closed Monday's trading at $24.44, up 2.09%.

Shares of Immune Design Corp. (IMDZ) plunged more than 15% in extended trading on Monday, following its plans to sell up to 16 million shares of its common stock in an underwritten public offering.

The Company intends to grant the underwriters a 30-day option to purchase up to an additional 15% of the shares of its common stock offered in the public offering.

IMDZ closed Monday's trading at $5.20, unchanged from the previous day's close. In after-hours, the stock was down 15.48% to $4.39.

Neurocrine Biosciences Inc.'s (NBIX) Valbenazine has been granted Orphan Drug Designation by the FDA for the treatment of pediatric patients with Tourette syndrome.

Valbenazine was approved by the FDA in April of this year for the treatment of tardive dyskinesia, and is marketed under the brand name Ingrezza.

In May of 2017, the Company announced that its initial Phase II Tourette syndrome T-Force GREEN study of INGREZZA did not meet its primary endpoint.

Commenting on the orphan drug designation to Valbenazine for the treatment of pediatric patients with Tourette syndrome, Chief Regulatory Officer at Neurocrine Biosciences said, "We will continue our clinical development program for Tourette's focused on pediatric patients as the first symptoms of Tourette syndrome typically present early in childhood and represent a significant challenge for these young patients."

NBIX closed Monday's trading at $58.53, down 0.85%. In after-hours, the stock was up 1.66% to $59.50.

uniQure N.V. (QURE) has commenced an underwritten public offering of 5 million of its ordinary shares. In addition, the Company intends to grant the underwriters a 30-day option to purchase up to 750,000 additional ordinary shares.

To know more about uniQure, please visit our Company Spotlight column.

QURE closed Monday's trading at $18.52, down 3.89%.