Market Overview

Lysogene Announces FDA approval of IND Application to Initiate Phase 2-3 Clinical Trial in MPS IIIA

  • Novel, one-time, investigational gene therapy treatment for MPS
    IIIA focused toward preventing neurocognitive decline
  • LYS-SAF302 product manufacture released to support the demands of
    phase 2-3 clinical trial
  • Implementation of the clinical trial depends on the strengthening
    of the Company's financial capabilities, which is currently being

Regulatory News:

Lysogene (Paris:LYS) (FR0013233475 – LYS), a pioneering
biopharmaceutical company specializing in gene therapy targeting central
nervous system (CNS) diseases, announced today that the U.S. Food and
Drug Administration (FDA) has granted an Investigational New Drug (IND)
clearance to proceed in the U.S. with its international, Phase 2-3
(AAVance) clinical trial of LYS-SAF302 for the treatment of
Mucopolysaccharidosis Type IIIA (MPS IIIA).

Lysogene has also submitted Clinical Trial Applications (CTA) in Europe.

"The IND clearance of the Phase 2-3 study of LYS-SAF302 in MPS IIIA
represents a major milestone for the clinical trial planned in four
leading U.S. sites and four European sites," said Karen Aiach, Founder
and Chief Executive Officer. "The same design, implementation and
operational conduct of the study has also been approved by the European
Medicines Agency Pediatric Committee (PDCO), thus allowing a robust,
rigorous and consistent multi-national approach."

"Following standing interactions with the FDA, we are pleased to
announce full alignment with the Agency on the non-clinical, clinical,
regulatory and manufacturing dimensions of our Phase 2-3 study in the
MPS IIIA," added Sophie Olivier, M.D., Chief Medical Officer. "Also, the
past months, were important from a manufacturing perspective resulting
in the successful production of LYS-SAF302 and product release to
support the demands of the phase 2-3 clinical trial."

LYS-SAF302 Pediatric Investigation Plan (PIP) received approval from the
PDCO earlier this year. Additionally, LYS-SAF302 has received Orphan
Drug Designation from the FDA and EMA as well as Rare Pediatric Disease
Designation from the FDA. Leading international gene therapy and MPS
centers plan to participate in the clinical trial (

As previously announced, the company is currently looking for funding
opportunities in order to extend its cash runway and launch the MPS IIIA
clinical trial, notably through a strategic partnership or transaction,
or potential sale of one or several products.

Lysogene's approach

MPS IIIA is a lethal neurological disease with debilitating symptoms for
which there is currently no approved treatment. CNS manifestations
predominate, in particular intellectual disability, progressive loss of
acquired skills, severe sleep and behavior disorder.

LYS-SAF302 is an rAAV vector serotype rh.10 carrying the gene coding for
SGSH. This in vivo gene therapy offers the possibility of a one-time
treatment by inserting a healthy copy of the SGSH gene and allowing the
body to start making the missing enzyme, therefore slowing or halting
disease progression. Lysogene's gene therapy is delivered directly to
the CNS during a neurosurgical procedure. By delivering the missing SGSH
gene, Lysogene believes MPS IIIA patients will be provided a permanent
source of functional enzyme in the brain that reverses phenotypic
abnormalities of CNS cells.

LYS-SAF302 is Lysogene's fully optimized second generation gene therapy
program for MPS IIIA. The data build upon its completed five-year Phase
1-2 clinical study with LYS-SAF301. Furthermore, the selection of an
optimally efficacious dose and of a specific cannula to deliver
LYS-SAF302 directly to the CNS, where it is most needed, reinforce
Lysogene's belief that LYS-SAF302 could potentially show substantial
improved efficacy. Lysogene holds an exclusive worldwide license from
REGENXBIO for the use of AAVrh.10 in MPS IIIA.

About Lysogene

Lysogene is a gene therapy company focused on the treatment of orphan
diseases of the central nervous system (CNS). The company has built a
unique capability to enable a safe and effective delivery of gene
therapies to the CNS to treat lysosomal diseases and other genetic
disorders of the CNS. A pivotal clinical trial in MPS IIIA and a phase
1-2 clinical trial in GM1 Gangliosidosis are in preparation, while we
are currently collaborating with a major partner to define the strategy
of development for the treatment of Fragile X syndrome, a genetic
disease related to autism.

Forward looking statement

This press release may contain certain forward-looking statements.
Although the Company believes its expectations are based on reasonable
assumptions, all statements other than statements of historical fact
included in this press release about future events are subject to (i)
change without notice, (ii) factors beyond the Company's control and
(iii) the financial capabilities of the Company. These statements may
include, without limitation, any statements preceded by, followed by or
including words such as "target," "believe," "expect," "aim," "intend,"
"may," "anticipate," "estimate," "plan," "project," "will," "can have,"
"likely," "should," "would," "could" and other words and terms of
similar meaning or the negative thereof. Forwardlooking statements are
subject to inherent risks and uncertainties beyond the Company's control
that could cause the Company's actual results, performance or
achievements to be materially different from the expected results,
performance or achievements expressed or implied by such forward-looking
statements. A further list and description of these risks, uncertainties
and other risks can be found in the Company's regulatory filings with
the French Autorité des Marchés Financiers, including in the 2017
registration document (Document de référence), registered with the
French Markets Authorities on June 4, 2018, under number R. 18-047, and
future filings and reports by the Company. Except as required by law,
the Company assumes no obligation to update these forward-looking
statements publicly, or to update the reasons actual results could
differ materially from those anticipated in the forward-looking
statements, even if new information becomes available in the future.

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