Lysogene receives British MHRA passport for GM1 bacteriosis program

Regulatory news:

Lysogene (FR0013233475 – LYS) (Paris: LYS), a Phase 3 biopharmaceutical company based on a gene therapy technology platform targeting central nervous system (CNS) diseases, announced today that the UK regulatory agency MHRA (Medicines and Healthcare products Regulatory Agency) Awarded the Innovation Passport for Experimental Gene Therapy LYS-GM101 in the treatment of GM1 bacteriuria as part ofInnovative license and access path (ILAP) from the United Kingdom. The decision was made by the ILAP Steering Committee, which consists of representatives from MHRA, NICE (National Institute for Health and Care Excellence), and SMC (Scottish Pharmaceutical Consortium), AWTTC (All Wales Poison and Therapy Centre) and representatives of the ILAP patient and public reference group.

The Innovation Passport is a prerequisite for access to ILAP, and is designed to accelerate the development and access to promising innovative medicines in the UK, thereby facilitating patient access to and improvement of new medicines.

“We are very happy to receive the Innovation Passport” announce Marie Dino, Director of Regulatory Affairs and Quality Assurance at Lysogene. “Ensuring that patients with GM1 disease can benefit from LYS-GM101 is a priority for Lysogene. We will now work with MHRA and its partners to create a Targeted Development Profile (TDP) for LYS-GM101, and facilitate patient access to gene therapy.”

ILAP was established by MHRA in January 2021 and aims to facilitate the development and patient access to promising innovative medicines. The organizational path involves close collaboration with other stakeholders, including patient representatives. Other benefits of ILAP consist of a 150-day rapid assessment, an ongoing review of marketing authorization application and an ongoing risk-benefit assessment.

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About LYS-GM101

LYS-GM101 is in clinical development. P1-GM-101 (NCT04273269) is an open-label, adaptive, two-phase clinical trial conducted at four clinical sites, including one in the United Kingdom, with natural history data as a control. As part of the P1-GM-101 trial, a sub-study is being conducted on video outcomes and parent interviews, the data of which will complement the clinical end points. In addition, a set of natural history study data from interviews and video assessments of children with GM1 streptoderma taken at home by parents/caregivers was initiated in early 2020 and is ongoing (NCT04310163). LYS-GM101 has also been designated an orphan in the European Union (EU) and the United States, as well as a rare and ‘fast-track’ pediatric disease classification in the United States.

About Lysogen

Lysogene is a biopharmaceutical company that specializes in gene therapy targeting diseases of the central nervous system (CNS). The company has gained unique experience that enables it to offer CNS gene therapies to treat particle overload and other CNS genetic diseases. Lysogene is currently conducting a Phase 2/3 clinical study at MPS IIIA, in partnership with Sarepta Therapeutics, Inc. An adaptive clinical study in GM1 gangliosidosis is underway. In accordance with the agreement between Lysogene and Sarepta Therapeutics, Inc. Sarepta Therapeutics, Inc., will maintain will retain exclusive commercial rights to LYS-SAF302 in the United States and in markets outside of Europe, while Lysogene will retain exclusive commercial rights to LYS-SAF302 in Europe. Lysogene has also entered into an exclusive global licensing agreement with SATT Conectus for a gene therapy candidate in the treatment of fragile X syndrome, a genetic disease associated with autism.

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Lysogene forward-looking statements

This press release may contain forward-looking statements, particularly regarding clinical trial progress and the company’s cash flow projections. Although the Company considers its expectations to be based on reasonable assumptions, all statements other than statements of historical facts that this press release may contain regarding future events are subject to (i) change without notice, (ii) factors over which the Company has no control over. , (3) results of clinical studies, (4) increases in production costs, (5) potential claims on its products, (6) change in the nature of its agreements with Sarepta Therapeutics. These statements may include, without being exhaustive, all statements beginning with, followed by or including words or phrases such as “objective”, “believe”, “expect”, “objective”, “intend”, “could”, “expect”, “estimate”, “plan”, “project”, “should”, “may”, “may”, “should”, “could” and other words and expressions with a good meaning or used in the negative form. Forward-looking statements are subject to material risks and uncertainties beyond the Company’s control which may, from time to time, cause significant differences between the Company’s actual results, performance or achievements and those projected or expressed or implied by the above. See the data. A list and description of these risks, contingencies and uncertainties appear in the documents the company has submitted to the Autorité des Marchés Financiers (AMF) in accordance with its regulatory obligations, including the company’s 2020 general registration document, registered with the AMF on April 12, 2021 with number D.21-0296, as well as In the documents and reports that the company will publish later. Furthermore, these forward-looking statements speak only as of the date of this press release. Readers are cautioned not to place excessive reliance on these forward-looking statements. Unless required by law, the Company assumes no obligation to publicly update these forward-looking statements, nor to update the reasons why actual results may differ materially from the results expected from the forward-looking statements, including in the event new information becomes available. The Company’s update of one or more forward-looking statements does not mean that the Company will or will not make further updates to these or other forward-looking statements.

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