IONS Scripts History, PTLA Gets FDA Date, Strongbridge Slumps, NEOT On Watch

(RTTNews) - Shares of Ionis Pharmaceuticals Inc. (IONS) were up over 8% in extended trading on Friday, following FDA approval of SPINRAZA, an antisense drug, for the treatment of spinal muscular atrophy in pediatric and adult patients.

SPINRAZA, known generically as nusinersen, becomes the first and only treatment approved in the U.S. for SMA.

Spinal Muscular Atrophy, or SMA, is a rare neuromuscular disease, affecting the nerve cells in the lower part of the brain and spinal cord that are responsible for neuro-muscular function, and mobility becomes too difficult for children making them confined to a wheel chair.

SPINRAZA is licensed to Biogen Idec (BIIB), which is responsible for future development, manufacturing, and commercialization.

IONS closed Friday's trading at $53.41, up 3.53%. In after-hours, the stock was up 8.31% to $57.85.

Neothetics Inc. (NEOT) has initiated a phase II proof of concept trial of LIPO-202 for the reduction of submental subcutaneous fat.

The trial is expected to enroll a total of 150 patients, and they will receive up to 30 subcutaneous injections of LIPO-202 or placebo once a week for eight weeks. The top-line data from this trial are expected in late second quarter 2017.

NEOT closed Friday's trading at $1.15, up 19.79%.

Shares of Portola Pharmaceuticals Inc. (PTLA) spiked over 33% on Friday on its New Drug Application for Betrixaban being granted priority review, with a decision date set for June 24, 2017.

The company is seeking approval of Betrixaban, an oral, once-daily Factor Xa inhibitor anticoagulant, for extended-duration prophylaxis of venous thromboembolism (VTE) in acute medically ill patients with risk factors for VTE.

Additionally, the Marketing Authorization Application for Betrixaban is under review by the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) under a standard 210-day review period.

If approved, Betrixaban has the potential to be the first anticoagulant approved for in-hospital and extended-duration VTE prophylaxis in high-risk acute medically ill patients.

PTLA closed Friday's trading at $25.06, up 33.80%.

Strongbridge Biopharma plc. (SBBP) slumped more than 30% on Friday, following a $35 million equity financing, wherein it will issue 14 million ordinary shares to the investors at a price of $2.50 per ordinary share as well as warrants to purchase 7 million shares.

The warrants are exercisable at a price of $2.50 per share and expire five years from the date of issuance. The company expects the closing of the private placement to occur on or about December 28, 2016, subject to satisfaction of customary closing conditions.

The company noted that recruitment in its phase III clinical trial of COR-003 for the treatment of endogenous Cushing's syndrome, dubbed SONICS, has been slower than anticipated. Accordingly, the study is anticipated to be fully enrolled in Q2 2017, with top-line data for the primary efficacy analysis available in Q1 2018.

In a separate press release, the company announced that it has acquired the U.S. rights to KEVEYIS from a subsidiary of Taro Pharmaceutical Industries Ltd. KEVEYIS was approved by the FDA in August 2015 to treat hyperkalemic, hypokalemic, and related variants of Primary Periodic Paralysis, which is a group of rare hereditary disorders that causes potentially severe episodes of muscle weakness and/or paralysis.

SBBP closed Friday's trading at $2.50, down 30.56%.