Spark Therapeutics Achieves Recruitment Goal in Phase 3 Gene Therapy Clinical Study for Inherited Blindness

Spark Therapeutics Achieves Recruitment Goal in Phase 3 Gene Therapy Clinical
                        Study for Inherited Blindness

Spark CEO provides corporate updates at the J.P. Morgan Conference

PR Newswire

SAN FRANCISCO, Jan. 14, 2014

SAN FRANCISCO, Jan. 14, 2014 /PRNewswire/ -- Spark Therapeutics, a late-stage
gene therapy company, has reached its patient recruitment goal for its most
advanced clinical program, a Phase 3 study for inherited retinal dystrophies
caused by mutations in the RPE65 gene. Spark's CEO, Jeffrey D. Marrazzo, is
presenting this and other corporate updates today, at the 32nd Annual J.P.
Morgan Healthcare Conference in San Francisco. Marrazzo said Spark was able to
achieve its recruitment goal a year after the study's initiation and
anticipates filing a BLA on the final data in 2015. Currently, there are no
pharmacologic treatments for this or any form of inherited retinal dystrophy,
which ultimately causes irreversible blindness.

"The fact that we were able to enroll patients in our lead clinical program so
quickly and seamlessly reflects the critical, unmet medical need associated
with inherited retinal dystrophies as well as the deep expertise of our
founders and the hard work oftheclinical teams at The Children's Hospital of
Philadelphia and the University of Iowa," said Jeffrey D. Marrazzo,
co-founder, president and chief executive officer. "In order to realize our
vision of transforming the lives of patients in a single therapeutic dose, we
must continue to operate at the highest standard, as we have in achieving
patient recruitment for Phase 3 of our lead candidate right on the heels of
our corporate launch."

Spark's patient recruitment goal for the Phase 3 study was a minimum of 24
patients. The open-label, randomized, controlled study builds on a Phase 1/2
clinical trial in which 12 patients with RPE65-related blindness demonstrated
notable improvements in functional vision, moving in some cases from being
profoundly blind to being able to recognize faces and ambulate independently.
All school-aged patients enrolled in the trial were able to transfer from
Braille classrooms to sighted classrooms.

In addition to Spark's Phase 3 recruitment achievement, Marrazzo shared
several corporate updates at the J.P. Morgan conference, including:

  oThe company is well-positioned to be the first to obtain U.S. FDA approval
    for a gene therapy product and is preparing to launch the first in a
    series of therapies to address the significant unmet need of patients with
    inherited retinal dystrophies.
  oSpark expects to file an investigational new drug (IND) application for a
    second inherited retinal dystrophy this year.
  oThe company presented encouraging results from the first subject in its
    Phase 1/2 hemophilia B program, in which a study participant has been able
    to go without factor for six months since vector infusion.
  oTwo new team members have joined Spark: Carol Greve-Philips, senior vice
    president, business development and Joseph La Barge, senior vice president
    of business administration and general counsel. Greve-Philips previously
    served as vice president of corporate development at Genzyme; La Barge
    played a critical role as general counsel at Tengion, Inc., developing
    intellectual property strategies for first-in-class, cell-based

The company continues to enroll its Phase 1/2 trial for hemophilia B and is
advancing toward the clinic with gene therapy programs to address multiple,
fatal neurodegenerative diseases.

For more information on Spark Therapeutics please visit or
follow on Twitter @spark_tx.

About Spark Therapeutics
Spark Therapeutics is developing potentially curative, one-time gene therapy
products to transform the lives of patients and re-imagine the treatment of
debilitating diseases. Spark's lead gene therapy candidate, for RPE65-related
blindness, is currently in Phase 3 clinical trials with the potential to be
the first approved gene therapy in the U.S., and the first in a series of
therapies to address the significant unmet needs of patients living with
blindness due to inherited retinal dystrophies.

Spark's founding team includes scientists who led the movement to develop gene
therapy as a new treatment paradigm, establishing clinical proof of concept in
the eye and liver and contributing key insights to the field that have
resulted in a resurgence of industry interest in gene-based medicines. In
addition to the Phase 3 program in RPE65-related blindness, the company has a
Phase 1/2 program in hemophilia B, and preclinical programs to address
neurodegenerative diseases and other inherited retinal dystrophies and
hematologic disorders. Spark has rights to a proprietary manufacturing
platform that has an unparalleled track record of success in supporting
clinical studies across diverse therapeutic areas and routes of
administration. The company's expertise across research, clinical, regulatory
and manufacturing builds on a legacy of innovation and excellence in gene
therapy established by Spark's team while at The Children's Hospital of
Philadelphia Center for Cellular and Molecular Therapeutics. To learn more

Media Inquiries:
Jessica Rowlands

SOURCE Spark Therapeutics

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