GenSight Biologics Announces Preliminary Assay Indicating Successful Manufacture of Second LUMEVOQ® GMP Batch

Paris, France, November 13, 2023, 7:30 am CET – GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that a preliminary assay of the viral genome (vg) titer indicates the successful manufacture of a second batch of drug substance (DS) for LUMEVOQ^®, the Company’s gene therapy for Leber Hereditary Optic Neuropathy (LHON), compliant with Good Manufacturing Practice (GMP) standards.

The assay, while preliminary, is known to be predictive of the final result at the independent laboratory used for the official release assay. Because the batch was manufactured according to GMP manufacturing protocols, which are the required standards for commercial batches, the gene therapy from the drug substance of this batch may be eligible for use with patients after passing all quality control tests and pending discussions with regulatory bodies.

“We are delighted that the actions we took in collaboration with our manufacturing partner, including enhanced training and supervision for the manufacturing team, have contributed to these two successful batches,” commented Scott Jeffers, Chief Technology Officer of GenSight Biologics. “The streamlined batch records and work instructions, coupled with the training and our presence on the manufacturing floor for every critical operation, have significantly reduced process deviations. We are excited to take this level of execution quality into our future campaigns, so that GS010 can be made available to LHON patients who have an urgent need for a treatment.”

Preparations are underway for the third GMP batch of the campaign planned by the Company. As with the two batches successfully manufactured so far, all of which are outside the context of a validation campaign^[1], the third batch will generate more process data for a future Marketing Authorisation Application (MAA) submission, provide more experience of the manufacturing process to the operating teams, and help fulfill the immediate requirement of supplying product for the new RECOVER clinical trial and for the potential resumption of an early access program for patients in the beginning of the second quarter of 2024.

GenSight Biologics will communicate further in the next few days when the vg titer results are confirmed by the independent laboratory. Upon this confirmation, the Company would be eligible to drawdown the second tranche of €4 million from the bridge financing signed in August 2023 with Sofinnova Partners, Invus and UPMC Enterprises (the “Bridge Financing“).

The cash runway would be extended to mid-December 2023 with the drawdown of the second tranche of the Bridge Financing for an amount of €4 million. Following the drawdown of the second tranche, GenSight Biologics needs to seek other sources of debt, other non-dilutive or equity financing beyond that date in order to supplement its working capital requirements and fund its operating expenses until the resumption of the early access program in France (Autorisation d’Accès Compassionnel or AAC) expected in the beginning of the second quarter of 2024. GenSight Biologics estimates that, in addition to the second tranche of the Bridge Financing, it will need approximately €10 million to finance its activities until that date.

[1] A validation campaign, or Process Performance Qualification (PPQ) campaign, consists of at least 3 successful GMP (Good Manufacturing Practice, required standards for human use outside of a clinical trial) batches manufactured sequentially to demonstrate and document the robustness, control, consistency and reproducibility of the commercial manufacturing process at the designated commercial facility. This exercise is required only as part of a Marketing Authorisation Application with the EMA and a Biologics License Application (BLA) with the FDA.