Lysogene, looking for saving grace after trial flop, suspends stock trading and seeks partners

On the heels of Lysogene’s lead gene therapy candidate flunking a phase 2/3 study, the French biotech has suspended trading of its shares and is seeking potential partnerships and investors in an effort to stay afloat. 

The gene therapy company focused on central nervous system diseases has requested the opening of a safeguard proceeding, which is a legal option French companies can take in an attempt to stay alive. Safeguard proceedings allow struggling companies to be restructured at a preventive stage under supervision of the court.

In anticipation of the safeguard proceedings, in which a decision is expected during the week of Dec. 12, Lysogene has requested that the trading of its shares be suspended until the court rules.

The company is also pursuing discussions with potential partners and investors in an effort to strengthen its financial position and extend its cash runway, which currently only lasts into February 2023. Discussions are ongoing and the opening of a safeguard proceeding will provide time for potential partners and investors to come forward, or for the company to find potentially interested parties, according to a Dec. 6 company release.

Lysogene has been short on cash since at least mid-November, when its top pipeline candidate, dubbed LYS-SAF302, failed a phase 2/3 trial. The study assessed the gene therapy in patients with mucopolysaccharidosis type IIIA (also known as Sanfilippo syndrome), a neurodegenerative disease that can spur a wide range of bodily dysfunction.

Among the six youngest participants in the trial—children younger than 30 months—there was a statistically significant improvement in cognitive function compared to natural history two years after dosing, according to Lysogene. No treatment effect was observed among the remaining 12 participants, dubbed "the main cohort.” Development was measured using one of two different scales that assess neurodevelopmental and cognitive delay.

The results come almost a year after Sarepta walked away from a deal that gave it ex-Europe rights to LYS-SAF302, a collaboration that cost $15 million in upfront cash and up to $125 million in biobucks. At the time, Lysogene said the termination had to do in part with “unsuccessful discussions” regarding the transferring of commercial supply of the therapy back to Lysogene.

After the disappointing data drop, Lysogene began strategic discussions in the hopes of landing a new licensing deal. 

The only other clinical asset in Lysogene's pipeline is LYS-GM101, a treatment for the genetic neurological disorder GM1 gangliosidosis. The condition progressively destroys nerve cells in the brain and spinal cord.