Pharmaceutical Business review

Gene therapy firm AveXis secures $65m in financing

The proceeds will be used to advance the company’s ongoing spinal muscular atrophy (SMA) clinical program and expand key operational capabilities.

The Class D financing was led by funds and accounts managed by T. Rowe Price Associates, a new investor in AveXis.

Existing investors, including Deerfield Management, Roche Venture Fund and Venrock, also participated in the financing, as did new participants, including Janus Capital Management, Adage Capital Management, RA Capital Management, QVT Financial, Rock Springs Capital Management, Foresite Capital Management, RTW Investments, and Boxer Capital of Tavistock Life Sciences.

AveXis president and CEO Sean Nolan said: "We are pleased to have such a robust syndicate of well-respected investors, led by T. Rowe Price funds and accounts, join with our existing investors in this financing. This points to the broad enthusiasm surrounding our SMA clinical program and the progress we have made to date.

"We are now well-positioned to continue development of our novel gene therapy for patients suffering from SMA Type 1, a rare genetic disease and the most common genetic cause of infant mortality."

AveXis has an ongoing Phase 1 dose-ranging clinical trial utilizing scAAV9.CB.SMN in patients with SMA Type 1. This gene transfer therapy introduces fully-functioning copies of a survival motor neuron (SMN) gene.

The copied gene is intended to supplement the body’s production of SMN protein using an adeno-associated viral vector that carries the SMN gene. Following this treatment, the company believes that the cell machinery could then continually produce SMN protein from the new copies of the SMN gene.

T. Rowe Price Health Sciences Fund portfolio manager Taymour Tamaddon said: "SMA is a devastating genetic disease with no approved treatments.

"We believe the AveXis program holds great promise for patients suffering from SMA and we are pleased to support the company."

Cooley LLP served as legal advisor to the company and Jefferies LLC acted as financial advisor to the company for the transaction.