- French gene therapy developer GenSight Biologics is withdrawing an approval application for an experimental eye disease treatment in Europe after regulators challenged the data supporting its use, the company said Thursday. GenSight executives want guidance on what data would be sufficient to support a clearance.
- With cash due to run out in June, GenSight executives said Friday they are looking at “financing and strategic options including M&A opportunities.” As of March 31, the company had 8.5 million euros ($9.3 million), after burning through 2.1 million euros in the first three months of the year.
- The gene therapy, called Lumevoq, aims to treat a blindness-causing condition called Leber hereditary optic neuropathy, or LHON. But its path to approval has been affected by clinical research in which trial participants saw unexpected improvements in visual acuity in eyes that didn’t receive injections of Lumevoq.
GenSight is facing another setback and a dwindling cash pile at an unfortunate time. A market downturn has made it difficult for drug developers to raise money by selling shares. For GenSight to continue its clinical work, it will likely need more time than its current financial runway will provide. Executives are also looking at other ways of “significantly reducing its operating cash burn in 2023.”
As an acquisition target, GenSight and its $37 million market capitalization would make an inexpensive purchase. But any prospective buyer would have to take a risk on a gene therapy that has faced numerous setbacks in its path toward approval.
The clinical work completed so far points to improvements in visual acuity for those that have received the gene therapy. The main trial’s design had participants get the gene therapy in one eye and a placebo in another so researchers could evaluate the difference.
Surprisingly, however, visual acuity in patients’ untreated eyes improved, causing the study to miss its main goal. Researchers believe the viral vector used to deliver the therapy in the treated eyes may have also reached the untreated eyes.
Early last year, the Food and Drug Administration told GenSight that it would need to conduct another placebo-controlled trial before applying for approval because of the findings. GenSight didn’t disclose what the European Medicines Agency said about the data.
“Since it appears that the company has submitted all available data that could support Lumevoq's approval, that path may potentially include generating new clinical data,” Chardan analyst Daniil Gataulin wrote in an April 20 note to clients. That trial would likely take more than two years, Gataulin wrote.
In February, GenSight received an 8 million euro loan from European Investment Bank. A second tranche of that loan, worth 12 million euros, could become available later this year if GenSight meets certain milestones.