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Agreements

Date: 2018-10-15

Type of information: Licensing agreement

Compound: LYS-SAF302

Company: Sarepta Therapeutics (USA - MA) Lysogene (France)

Therapeutic area: Rare diseases - Genetic diseases

Type agreement: licensing

Action mechanism:

  • gene therapy. 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 CNS. 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.

Disease: mucopolysaccharidosis type IIIA (Sanfilippo B syndrome)

Details:

  • • On October 15, 2018, Sarepta Therapeutics announced it has signed a license agreement with Lysogene for the development of a gene therapy, LYS-SAF302, to treat mucopolysaccharidosis type IIIA (MPS IIIA). Under the terms of this agreement, Sarepta will also have certain option rights to an additional CNS-targeted gene therapy candidate.
  • Under the terms of the license, Lysogene shall be responsible for completion of the pivotal trial, which is set to commence in the fourth quarter of 2018. Sarepta shall have exclusive commercial rights to LYS-SAF302 in the United States and all territories outside of Europe, and Lysogene will retain exclusive commercial rights to LYS-SAF302 in Europe. Sarepta will be responsible for global manufacturing of LYS-SAF302 and will supply Lysogene for its territory.
 

Financial terms:

  • In 2018, Sarepta will make committed cash payments to Lysogene totaling $26 million (€22 million) plus the purchase of Lysogene equity for $2.5 million (€2.2 million). In 2019, Sarepta will pay Lysogene up to an additional $19 million (€16 million).
  • Payments from Sarepta to Lysogene for all items, if all milestones are met, would total approximately $125 million (€108 million) plus royalties. The financial terms of the agreement significantly extend Lysogene’s cash runway, enabling the company to continue the development of its other assets.
  • As part of this partnership, Sarepta is subscribing to an equity investment of $2.5 million (€2.2 million) at a 30% premium to the 5-day volume-weighted average share price, through the issuance of 950,606 ordinary shares, with the same rights as existing shares, under a capital increase with cancellation of preemptive rights under article L.225-138 of the French Commercial Code and the 22d resolution of Lysogene’s Extraordinary General Meeting in June 2018. The issuance of these shares will result in dilution of 7.1% for existing Lysogene shareholders. The issuance of these shares does not require the publication of a prospectus submitted to the visa of the Autorité des Marchés Financiers in accordance with article 211-3 of the AMF General Regulations. Lysogene intends to apply the funds raised for general corporate purposes including the further development of LYS-GM101 for GM1 gangliosidosis.

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