Lysogene Announces first European patient treated in AAVance, Phase 2/3 Clinical Trial Investigating LYS-SAF302, a Gene Therapy for the Treatment of MPS IIIA (Sanfilippo Syndrome Type A)

Lysogene Announces first European patient treated in AAVance, Phase 2/3 Clinical Trial Investigating LYS-SAF302, a Gene Therapy for the Treatment of MPS IIIA (Sanfilippo Syndrome Type A) 150 150 Lysogene

— Phase 2/3 trial first set up in the US and treating patients since February 2019 —

— Expansion of trial, to Europe, to assess efficacy on neurodevelopmental status of MPS IIIA patients —

PARIS, France – June 11, 2019, at 10:00pm CEST – Lysogene (FR0013233475 – LYS), a pioneering biopharmaceutical company specializing in gene therapy targeting central nervous system (CNS) diseases announced today, the first European patient has been dosed in AAVance, a global Phase 2-3 clinical trial of LYS-SAF302, a gene therapy for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA). Lysogene and Sarepta Therapeutics, its commercialization partner for LYS-SAF302, announced the treatment of the first patient in this clinical trial in the US earlier this year.

AAVance is a single-arm trial evaluating the safety and effectiveness of a one-time delivery of a recombinant adeno-associated virus vector rh.10 carrying the N-sulfoglucosamine sulfohydrolase (SGSH) gene. MPS IIIA is caused by mutations in the SGSH gene, which is involved in producing an enzyme necessary for the breakdown and disposal of long chain sugar molecules. The goal of the therapy is to

show improved or stabilized neurodevelopmental status of MPS IIIA patients by allowing the brain cells

to secrete the missing enzyme. In the clinical trial, the objective is to enroll 20 patients at eight sites in the

“The first European patient dosed in the AAVance trial is an important step in addressing this relentlessly progressing neurodegenerative disease,” said Karen Aiach, Founder and Chief Executive Officer of Lysogene. “We are encouraged by the preliminary observations in AAVance to date, and are excited to bring this investigational therapy to patients in Europe.”

U.S. and Europe. More information can be found on (NCT03612869).

“MPS IIIA is a lethal neurological disease with debilitating symptoms for which there is currently no approved treatment,” said Prof Frits Wijburg, Academic Medical Center, University of Amsterdam and principal investigator for AAVance. “It is a great motivation to know that the work we are doing has the potential to make a life-changing difference for the children affected by this disease.”

“We are very pleased to have successfully administered LYS-SAF302 to the first European patient and mark this as a milestone for the field in advancing a potential one-time treatment for patients with MPS IIIA,” said Prof Michel Zerah, Hôpital Necker Enfants Malades in Paris.


MPS IIIA is a rare inherited neurodegenerative lysosomal storage disorder affecting approximately 1 in 100,000 newborns. Inherited in an autosomal recessive pattern, it is characterized by intractable behavioral problems and developmental regression resulting in early death. It is caused by mutations in the SGSH gene, which encodes an enzyme called Heparan-N-sulfamidase necessary for heparan sulfate (HS) recycling in cells. The disrupted lysosomal degradation and resulting storage of HS and glycolipids such as gangliosides leads to severe neurodegeneration. There are currently no treatment options for patients.

About LYS-SAF302

LYS-SAF302 is an AAV-mediated gene therapy, the goal of which is to replace the faulty SGSH gene with a healthy copy of the gene. LYS-SAF302 employs the AAVrh10 virus, chosen for its ability to target the central nervous system. Proof-of-concept was established in MPS IIIA pre-clinical models demonstrating strong expression, broad distribution, and the ability of the compound to correct lysosomal storage defects by producing the missing enzyme. Safety data from an IND-enabling toxicity and a biodistribution GLP study showed that, at any dose level evaluated, LYS-SAF302 was not associated with unexpected mortality, change in clinical signs, body weight, behavior or macroscopic findings in the brain. Sarepta Therapeutics holds exclusive commercial rights to LYS-SAF302 in the United States and markets outside Europe; and Lysogene maintains commercial exclusivity of LYS-SAF302 in Europe.

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 in partnership with Sarepta Therapeutics, Inc. is ongoing and a phase 1-2 clinical trial in GM1 Gangliosidosis is in preparation. Lysogene is also collaborating with an academic partner to define the strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism.

Lysogene Forward looking statement

This press release may contain certain forward-looking statements, especially on the Company’s progress of its phase 2-3 clinical trial. 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,” “objective”, “project,” “will,” “can have,” “likely,” “should,” “would,” “could” and other words and terms of similar meaning or the negative thereof. Forward-looking 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 2018 registration document (Document de référence), registered with the AMF on 29 April 2019 under number R. 19-016, and future filings and reports by the Company. Furthermore, these forward-looking statements are only as of the date of this press release. Readers are cautioned not to place undue reliance on these forward-looking statements. 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. If the Company updates one or more forward-looking statements, no inference should be drawn that it will or will not make additional updates with respect to those or other forward-looking statements.

This press release has been prepared in both French and English. In the event of any differences between the two texts, the French language version shall supersede.



Mathilde Bohin / Emmanuel Huynh NewCap

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