FOLD Expands, CERU Hits A High, FDA Panel Verdict Out On Breo Ellipta

(RTTNews) - Amicus Therapeutics (FOLD) rose 33% on Thursday following positive regulatory updates related to approval pathways in the U.S. and Europe for its migalastat monotherapy for Fabry patients.

In the U.S., the company intends to submit an NDA for accelerated approval of migalastat monotherapy, and plans to schedule a pre-NDA meeting in the second half of 2015.

Amicus is also accelerating the timing of the MAA submission in Europe for full approval for migalastat monotherapy from the middle of 2015 to the second quarter of 2015.

FOLD closed Thursday's trading at $12.46, up 33.26.

Shares of Cerulean Pharma Inc. (CERU) surged more than 15% on Thursday after the company announced that its phase 1b/2 trial of CRLX101 in combination with Avastin in relapsed renal cell carcinoma met its primary endpoint.

According to the trial results, patients in the trial receiving CRLX101 plus Avastin experienced median progression free survival of 9.9 months.

A phase 2 trial of CRLX101 plus Avastin in 3rd and 4th line renal cell carcinoma is underway, and top-line primary endpoint data from this randomized trial are expected in the second quarter of 2016.

CERU closed Thursday's trading at $10.66, up 15.49%.

GlaxoSmithKline plc (GSK) and Theravance Inc. (THRX) announced that the FDA panel, which reviewed the supplemental New Drug Application for Breo Ellipta, a once-daily inhaled treatment for asthma, has voted 16 - 4 recommending its approval in adults 18 years of age and older. However, the panel voted against approval of the product for the proposed indication in 12-17 year olds stating that the efficacy data was not sufficient to demonstrate the benefit and safety in that sub-population.

A final decision by the FDA on the expanded indication of Breo Ellipta is scheduled for April 30, 2015.

Breo Ellipta is a combination of fluticasone furoate, an inhaled corticosteroid, and vilanterol, a long-acting beta2-adrenergic agonist (LABA).

Breo Ellipta was approved by the FDA in May 2013 for the long-term, once-daily, maintenance treatment of airflow obstruction and for reducing exacerbations in patients with chronic obstructive pulmonary disease (COPD), including chronic bronchitis and/or emphysema.

Heron Therapeutics Inc. (HRTX) touched a new high on Thursday following positive results from a phase 1 clinical study of HTX-011, its lead product candidate for the prevention of post-operative pain, in healthy volunteers.

Heron plans to move HTX-011 into phase 2 clinical development in the second quarter of 2015.

HRTX closed Thursday's trading at $16.15, up 7.95%.

Prothena Corp. plc (PRTA) was up more than 40% in extended trading on Thursday after the company announced that PRX002, its investigational monoclonal antibody for the potential treatment of Parkinson's disease was safe and well-tolerated in healthy volunteers in a phase 1 single ascending dose trial, meeting the primary objective of the study.

The study results demonstrated rapid and dose-dependent reduction of free serum alpha-synuclein, a protein potentially involved in the onset and progression of Parkinson's disease, said the company.

PRX002 is the focus of a worldwide collaboration between Prothena and Roche. To date, Prothena has received $45 million of the potential $600 million in total milestones through its collaboration with Roche.

PRTA closed Thursday's trading 2.63% higher at $29.23. In after hours, the stock was up 40.27% at $41.00.

PTC Therapeutics Inc. (PTCT) has entered into a collaboration with the Orphan Disease Center at the Perelman School of Medicine at the University of Pennsylvania for discovering and developing new treatments for orphan disorders.

The first collaborative initiative will focus on PTC therapeutics' drug candidate Translarna, which is under a phase 2, proof-of-concept study in patients with the lysosomal storage disease Mucopolysaccharidosis I (MPS I) due to a nonsense mutation. Initial data from this study are expected by the end of 2015.

PTCT closed Thursday's trading at $69.23, down 3.24%.

Synthetic Biologics Inc. (SYN) is all set to start a phase 2a clinical trial of SYN-004, which is being developed to protect the microbiome and prevent Clostridium difficile infection, this month.

Currently, there is no vaccine or drug approved by the FDA specifically for the prevention of C. difficile infection. 

SYN closed Thursday's trading at $2.53, up 6.30%.

Vital Therapies Inc. (VTL) is scheduled to report topline results from a phase 3 trial, evaluating its ELAD System in subjects with alcohol-induced liver decompensation in the third quarter of 2015. The company anticipates filing a Biologics License Application in the first half of 2016 if the results are positive.

A second phase 3 trial, evaluating the ELAD System in severe acute alcoholic hepatitis patients who have failed standard therapy, is also underway, and topline results from this study are expected to be released in early 2017.

VTL closed Thursday's trading at $27.37, up 0.22%.