Endogena Therapeutics Receives US FDA Fast Track Designation for EA-2353 for the Treatment of Retinitis Pigmentosa

San Francisco, USA; Toronto, Canada; Zürich, Switzerland, February 6, 2023

Endogena Therapeutics Inc., a clinical-stage biotech company focused on the
development of endogenous regenerative medicines, announced today that the US
Food and Drug Administration (FDA) has designated the investigation of EA-2353 for
the treatment of retinitis pigmentosa (RP) as a Fast Track development
program. Fast Track is a process designed to enable patients to benefit earlier from
important new drugs for serious conditions.

EA-2353 takes a novel, small-molecule approach and selectively activates
endogenous retinal stem and progenitor cells, which differentiate into photoreceptors
and can potentially preserve or restore visual function. This gene-independent
treatment approach has significant advantages in RP, which has multiple genetic
causes. EA-2353 was granted orphan drug designation by the US FDA in May 2021.

RP is a serious and debilitating condition. It consists of a group of inherited diseases
causing slow and progressive retinal degeneration and loss of vision, for which there
is currently no treatment for most patients. It is a leading cause of inherited
blindness, with an estimated 1.5 million people worldwide presently affected.

Endogena Therapeutics is currently conducting a phase 1/2a dose-escalation study
in collaboration with our lead investigator, Mark Pennesi, MD, PhD, Professor of
Ophthalmology at the Casey Eye Institute in Oregon, USA, to examine the safety,
tolerability and preliminary efficacy of EA-2353 administered by intravitreal injection
in patients with RP (ClinicalTrials.gov identifier: NCT05392751). A total of 14 patients
with RP due to any pathologic genetic mutation are being recruited across up to six
sites in the USA, and the first patient was dosed in July 2022.

This Fast Track designation will enable Endogena Therapeutics to have more
frequent communications with the US FDA on the development of EA-2353 and
allow more rapid regulatory review of the future new drug application.

Matthias Steger, PhD, MBA, CEO of Endogena, said: “This acknowledgement by
the FDA of the potential of EA-2353 for RP gives hope for patients living with this
devastating degenerative disease. It is a significant milestone for our company, our
investors, and gives recognition to our dedicated team at Endogena, who have been
working for the past six years to reach this point.”

Endogena’s artificial intelligence-driven drug discovery platform, combined with
cutting edge knowledge of molecular pathways that regulate retinal stem cells and
retinal pigment epithelial cells, provides a potential new treatment paradigm to tackle
degenerative conditions related to aging and genetic disorders. Beyond EA-2353,
other products in Endogena’s pipeline include a treatment for dry age-related
macular degeneration (AMD), which is approaching IND-enabling studies, and earlier
programs in idiopathic pulmonary fibrosis (IPF) and hematopoietic recovery.

About Endogena

Endogena Therapeutics Inc. is a clinical-stage biotech company that discovers and
develops first-in-class endogenous regenerative medicines to repair and regenerate
tissues and organs. Its approach has the potential to change the way degenerative
conditions related to aging and genetic disorders are treated. The concept is based
on selective regulation of endogenous adult stem- and progenitor cells for controlled
tissue repair by small molecules. Endogena’s most advanced programs target
degenerative diseases of the eye, including retinitis pigmentosa and geographic
atrophy (secondary to AMD). Endogena is registered in San Francisco, USA,
headquartered in Zuerich, Switzerland, and holds a research facility at JLABS in
Toronto Canada.

For more information, please visit www.endogena.com

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See our YouTube channel for a video explaining how EA-2353 works

For more information on the clinical trials, please visit www.clinicaltrials.gov
(Reference: NCT05392751)

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