SEC Filings

8-K
AVEXIS, INC. filed this Form 8-K on 11/09/2017
Entire Document
 

 

respectively. Patients in Cohort 2 continued to demonstrate improvements in motor milestones: the majority of patients (92%) achieved head control, rolling over (75%), sitting with assistance (92%) and sitting without assistance for at least 5 seconds (92%). Two patients in the cohort could crawl, pull to a stand, as well as stand and walk independently.

 

·                  All patients who were free of respiratory or feeding support on January 20, 2017, continued without the need for these elements of supportive care.

 

·                  Eleven of 12 (92%) patients were able to speak; three more patients than previously reported on April 25, 2017 at the American Academy of Neurology.

 

AveXis Initiated Additional Formal Clinical and Quality Scientific Advice Proceedings with the European Medicines Agency (EMA) and Initiated the Process to Discuss the Potential for a Conditional Registration Pathway for the European Union (EU): AveXis also held detailed Clinical and Quality Scientific Advice with a number of European National Authorities focused on the Clinical Development Plan, as well as the Manufacturing/Quality process, which will serve to facilitate review and approval  of upcoming Clinical Trial Applications.

 

AveXis Opened Its Research and Development Facility in San Diego, California: This will be the location for analytical development, discovery research and translational development work.

 

Phillip B. Donenberg Appointed as Chief Financial Officer: On October 19, the AveXis Board of Directors appointed Phillip B. Donenberg as Senior Vice President and Chief Financial Officer of the company. Formerly the Vice President, Controller at AveXis, Mr. Donenberg brings more than 25 years of corporate finance, accounting and business development experience at publicly traded biotech and specialty pharmaceutical companies to the position.

 

Upcoming Milestones

 

SMA Type 2 Trial: AveXis is in ongoing discussions with the FDA on the SMA Type 2 Trial and remains optimistic that the trial will initiate before the end of the year.

 

SMA Type 1 End-of-Phase 1 Meeting with FDA: The end-of-Phase 1 meeting, scheduled for late in the fourth quarter, will further inform next steps in the regulatory process for AVXS-101. AveXis expects to provide an update in the first quarter of 2018 upon receipt of the meeting minutes from the FDA.

 

SMA Type 1 EU Trial: AveXis incorporated EU specific Scientific Advice from the EMA into the protocol design and expects to initiate a pivotal trial of AVXS-101 in SMA Type 1 in the EU in the first half of 2018.

 

Third Quarter 2017 Financial Results

 

·                  Cash Position: As of September 30, 2017, AveXis had $374.2 million in cash and cash equivalents.

 

·                  R&D Expenses: Research and development expenses were $33.4 million for the third quarter of 2017 (which included $5.0 million of non-cash stock-based compensation expense), compared to $14.1 million for the same period in 2016 (which included $2.7 million of non-cash stock-based compensation expense), resulting in an increase of $19.3 million. The increase was primarily attributable to product manufacturing expenses and associated accelerated spending, including increased headcount in AveXis’ product manufacturing facility, as well as expenses related to the conclusion of the Phase 1 clinical trial of AVXS-101 in SMA Type 1,

 

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