Follow the Money to Bio/Pharmaceutical Opportunities
Spotlight on AveXis, Inc.


Follow the Money to Bio/Pharmaceutical Opportunities highlights a newly funded bio/pharmaceutical company. This includes important insights into the featured company’s product pipeline, manufacturing and business relationships, and likely sourcing opportunities.

AveXis, Inc., develops gene therapies for the treatment of rare and orphan neurological genetic diseases. It plans to raise $234.5 million in a secondary public offering, as reported in the PharmSource Lead Sheet on June 29, 2017.

Corporate Highlights

  • AveXis was incorporated in Delaware in March 2010 under the name Biolife Cell Bank, LLC and changed its name in January 2014.
  • Corporate Headquarters: Bannockburn, IL – approximately 15,668 square feet of office and laboratory space
  • Other facilities: Richmond, VA; Brookvale, Australia; Ljubljana, Slovenia
  • Stock Exchange: NASDAQ
  • Stock Symbol: AVXS

Sourcing Opportunities

  • AveXis plans to use the proceeds to fund general corporate purposes, including manufacturing, R&D activities and ongoing clinical trials of its pipeline, which may include:
    • AVXS-101 – To initiate Phase II clinical trials:
      • For treatment of Spinal muscular atrophy type 1
      • Dosage Form: Parenteral – Liquid
      • Nature of API: Biologic – DNA – Viral vector
      • Therapeutic Areas: Muscular/Musculoskeletal; Pediatric
  • The company contracts with contract research organizations (CROs) to conduct clinical trials.
  • AveXis currently does not own or operate any manufacturing facilities and relies on third party contract manufacturers for all required raw materials, API and finished product for preclinical studies and clinical trials. The company is currently in the process of establishing their own manufacturing facility.
  • AveXis has no sales, marketing and distribution capabilities.

Business Relationships

  • In March 2014, AveXis and ReGenX Biosciences entered into an exclusive license agreement for the development and commercialization of AAV9 products for the treatment of spinal muscular atrophy. AveXis paid an initial $2 million fee and up to $12.25 million in milestone payments. As of December 31, 2016, AveXis paid a total of $2.4 million in fees and milestone payments.1

Drug Product Pipeline

Product Candidate Indication Dosage Form Status Next Anticipated Step
AVXS-101 Spinal Muscular Atrophy type 1 Parenteral – Liquid Phase I Plans to initiate Phase II clinical trials


(In $ thousands) 2016 2015
R&D Expenditures
General & Administrative Expenditures
Total Operating Expenses

1AveXis Corporation, 3/16/17 10-K,

Contact Information

AveXis, Inc. Key Officers
2275 Half Day Road, Suite 200 Sean P. Nolan, CEO
Bannockburn, IL, 60015 USA Andrew Knudten, SVP, Manufacturing & Supply Chain
Phone: +1-847-572-8280 Allan Kaspar, PhD, VP, R&D
Web: Sukumar Nagendran, MD, SVP, CMO

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Scotty is a Research Analyst at PharmSource. He holds a BS degree in Biology from George Mason University and an MPH degree from Eastern Virginia Medical School. He’s experienced in data collection, analysis and management, and has done laboratory analysis and safety program development.

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