NBIX Delivers, MNTA's Biosimilar Drug Trial Fails, No FDA Panel Meet For RARE

(RTTNews) - Today's Daily Dose brings you news about publication of AveXis' promising data of AVXS-101 in patients with spinal muscular atrophy Type 1 in NEJM; stellar Q3 results of Exelixis and Neurocrine, and failure of Momenta/Mylan's biosimilar drug trial.

Read on...

AveXis Inc.'s (AVXS) data as of August 7, 2017, from its phase I trial of AVXS-101 in patients with spinal muscular atrophy (SMA) Type 1 have been published in the New England Journal of Medicine (NEJM).

The data revealed that children who received a single dose of AVXS-101 demonstrated longer survival, superior achievement of motor milestones, and better motor function than historical cohorts. Moreover, all patients are alive, event-free and have reached at least 20 months of age. Historically, only eight percent of untreated patients with SMA Type 1 have survived event-free at 20 months of age, according to the Company.

AVXS-101 is currently being evaluated in a pivotal trial for the treatment of SMA Type 1.

AVXS closed Wednesday's trading at $99.92, down 4.39%.

Exelixis Inc. (EXEL) has delivered a strong financial performance in the third quarter ended September 30, 2017.

Net income for the recent third quarter was $81.4 million or $0.26 per share on total revenue of $152.5 million. This compared with a net loss of $11.3 million or $0.04 per share and revenue of $62.2 million for the comparable period in 2016.

Analysts polled by Thomson Reuters expected the Company to earn $0.08 per share on revenue of $104.89 million.

Near-term catalysts:

-- File sNDA for CABOMETYX in the treatment of advanced hepatocellular carcinoma patients who have been previously treated with Nexavar in the first quarter of 2018. -- FDA decision on CABOMETYX for the treatment of previously untreated advanced RCC is expected by February 15, 2018.

EXEL closed Wednesday's trading at $24.23, down 2.26%. In after-hours, the stock was up 7.30% to $26.00.

Momenta Pharmaceuticals Inc. (MNTA) and Mylan N.V.'s (MYL) M834, a proposed biosimilar of blockbuster drug ORENCIA, has failed to meet its primary pharmacokinetic endpoints in a phase I study.

The study compared the pharmacokinetics, safety and immunogenicity of M834 to US- and EU-sourced ORENCIA in normal healthy volunteers.

ORENCIA, developed by Bristol-Myers Squibb Co. (BMY), is indicated for the treatment of rheumatoid arthritis, juvenile idiopathic arthritis and psoriatic arthritis. The drug recorded global sales of $2.27 billion in 2016, up 20% over the prior year.

MNTA closed Wednesday's trading at $12.32, down 12.59%.

Shares of Neurocrine Biosciences Inc. (NBIX) were up over 18% in extending trading on Wednesday, following better-than-expected third-quarter financial results.

The net loss in the third quarter of 2017 narrowed to $11.1 million or $0.13 per share from $36.9 million or $0.43 loss per share for the same period in 2016. The net product sales from INGREZZA were $45.8 million in the recent third quarter whereas no sales were generated in the comparable year-ago period as the drug was made available for commercial distribution only on May 1, 2017.

Analysts polled by Thomson Reuters expected the Company to incur a loss of $0.49 per share on revenue of $29.86 million in Q3, 2017.

NBIX closed Wednesday's trading at $60.93, down 1.90%. In after-hours, the stock was up 18.99% to $72.50.

The FDA is not planning to hold an advisory committee meeting to discuss Ultragenyx Pharmaceutical Inc.'s (RARE) Biologics License Application for Burosumab to treat pediatric and adult patients with X-Linked Hypophosphatemia.

Burosumab is under Priority Review by the FDA, with a decision expected by April 17, 2018.

Ultragenyx, Kyowa Hakko Kirin and Kyowa Kirin International, a wholly owned subsidiary of Kyowa Hakko Kirin, have been collaborating in the development and commercialization of Burosumab globally.

RARE closed Wednesday's trading at $47.99, up 4.12%.