AveXis Inc. (AVXS) announced positive results from a Phase 1 trial of its drug, AVXS-101, to treat spinal muscular atrophy Type 1 disorder.

The early-stage, dose-escalating study assessed the safety and tolerability of the gene therapy in spinal muscular atrophy (SMA) patients. No new safety or tolerability related issues were identified during the study.

SMA is a genetic disorder caused by the deterioration of specialized nerve cells, called motor neurons, and the disease affects the control of muscle movement. It can lead to paralysis and difficulty in performing basic tasks like swallowing and breathing. Type 1 SMA starts in patients under the age of six months and is the most severe form of the disease. Type 2, 3 and 4 SMA afflicts patients in the higher age groups.

Drug Administering Show Positive Results

The early-stage trial had efficacy measure as duration of time from birth to an “event”, where the event was defined as either death or ventilation support needed for breathing in certain conditions and video confirmed achievement of ability to sit unassisted.

One-hundred percent of the cohort patients who were given one therapeutic dose of AVXS-101 had reached 13.6 months of age “event-free.” The result indicates better efficacy of the drug, as the expected event-free survival rate based on natural history of the disease is 25%.

Nine of nine patients reached 20 months of age event-free, against the expected event-free rate of 8% based on the natural history of the disease. The drug administering also resulted in improved head control, 180-degree rolling movements, and ability to sit with and without assistance for certain time.

Buoyed by the positive results, AveXis plans to meet with U.S. and European regulators, and plans to initiate a Phase 1 trial for assessing the drug for SMA Type 2 in the second quarter of 2017.

The drug is granted access to PRIority MEdicines (PRIME) program for SMA Type 1 by the European Medicines Agency (EMA), which allows for early and proactive support by EMA to accelerate drug development and quicker assessment of the Marketing Authorization Application.

AveXis will compete with established players like Biogen Inc. (BIIB) and Ionis Pharmaceuticals Inc. (IONS), who are jointly developing SMA therapies like nusinersen. (See also, Biogen Gets Competition for SMA Therapy.)

Following the positive results, Avexis stock spiked 14% Friday morning, to $84.50 per share. (See also, PTC Therapeutics Advances SMA Phase 2 Study.)

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