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Clinically meaningful visual acuity improvements were confirmed in a real-world setting: mean on-graph visual acuity 9 months post-injection compared to off-graph mean at baseline.
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Eyes of patients treated bilaterally showed better improvement than those treated unilaterally: +23 ETDRS letters vs. +18 ETDRS letters, higher response rate (63% vs. 58%), similar favorable Safety profile
Paris, March 6, 2024–(BUSINESS WIRE)–Regulatory news:
GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharmaceutical company focused on the development and commercialization of innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, announced today that Presented recent and promising real-world data.Program (EAP) confirms the benefits of LUMEVOQ® In patients with Leber’s hereditary optic neuropathy ND4 mutation(ND4-LHON), as observed in clinical trials.
One year after treatment, bilaterally injected patient eyes experienced an average increase from nadir1 Best-corrected visual acuity (BCVA) corresponds to +23 ETDRS letters, which is greater than the average increase of 18 letters for patients who injected only one eye. In both groups, clinical improvement exceeded the traditional definition of clinical meaning, in which an increase of 15 letters is considered meaningful. The safety profile of gene therapy continues to be favorable and comparable between bilaterally and unilaterally treated patients.
The results will be presented at the 2024 annual meeting of the North American Neuro-Ophthalmology Society (NANOS) and later this year at other major medical conferences in Europe and the United States. Dr. Chiara La Morgia, MD; Researchers from IRCCS Bologna Institute of Neuroscience and the University of Bologna, Italy, presented the results at NANOS and commented:As more and more data become available, it is very reassuring for clinicians that we continue to see a consistent picture of significant visual improvement in most patients treated with lenadogen norparvovec. is. The evidence is becoming more robust and applies to patients with severe blinding diseases such as LHON.. ”
These updated analyzes benefit from a more robust sample than the initial view of data released in March 2023.2. In particular, improvements after 1 year of treatment more clearly demonstrate the upward trajectory of mean visual acuity (see Figure 1).
Responder analysis one year after treatment showed that the majority of patients had improved vision. Clinically meaningful levels of BCVA improvement (≥0.3 LogMAR or +15 ETDRS letters) were reached in 63.2% of patient eyes that received bilateral injections. 57.9% of eyes of patients with unilateral injection. Nine months after injection, mean visual acuity was comparable to on-chart visual acuity compared to off-chart mean visual acuity at baseline.
The safety results obtained with EAP are consistent with those observed in clinical studies and demonstrate a favorable safety profile for lenadogen norparvovec.In particular, intraocular inflammatory events reported in LUMEVOQ®– Treated eyes were comparable in frequency, intensity, and location to those observed in clinical studies.
”Seeing these results, the GenSight team is focused on restoring early access to eligible patients by the third quarter of this year.,” Said Lawrence Rodriguez, CEO of GenSight. ”We are working on all options to bring LUMEVOQ to patients as quickly as possible, as there is accumulating evidence in clinical trials and real-world settings that access to LUMEVOQ can benefit LHON patients.. ”
Lumevoke® (GS010; lenadogene nolparvovec) is available through EAP in some countries upon unilateral request from clinicians and if early access use is permitted by local regulations in those countries. became. We aim to resume access early in France. Automation of Compassion (AAC) Program for Q3 2024.
About GenSight Biologics
GenSight Biologics SA is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics’ pipeline leverages his two core technology platforms, mitochondrial targeting sequencing (MTS) and optogenetics, to help preserve or restore vision for patients suffering from blinding retinal diseases. GenSight Biologics’ product candidate uses a gene therapy-based approach and is designed to be administered in a single treatment to each eye via intravitreal injection, providing patients with sustainable visual function recovery. To do.
About Leber’s hereditary optic neuropathy (LHON)
Leber’s hereditary optic neuropathy (LHON) is a rare, maternally inherited mitochondrial genetic disease characterized by degeneration of retinal ganglion cells, causing brutal and irreversible vision loss leading to legal blindness, affecting primarily adolescents and Young adults are affected. LHON is associated with sudden, painless loss of central vision.cent eyes, 2n.d. Eye problems occur one after another. It is a symmetrical disease with insufficient recovery of visual function. In 97% of subjects, vision loss occurs bilaterally within a year from the onset of vision loss, and in 25% of cases vision loss occurs in both eyes simultaneously.
About Lumevoke® (GS010; lenadogen norparbovec)
Lumevoke® (GS010; lenadogene nolparvovec) targets Leber’s hereditary optic neuropathy (LHON) by leveraging the mitochondrial targeting sequence (MTS) proprietary technology platform derived from research conducted at the Institute of Vision in Paris. This platform allows the use of AAV vectors (adeno-associated viruses) to specifically address defects within mitochondria. The gene of interest is introduced into cells and expressed to produce a functional protein. This protein is delivered to mitochondria via a specific nucleotide sequence to restore missing or deficient mitochondrial function. “LUMEVOQ” was approved by the European Medicines Agency (EMA) as the invention name for GS010 (lenadogen norparvovec) in October 2018. LUMEVOQ® (GS010; lenadogen norparvovec) is not registered in any country at this time.
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1 Nadir = lowest visual acuity observed between baseline (treatment time) and time point of interest. |
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2 “GenSight Biologics Announces Presentation of LUMEVOQ® Efficacy and Safety Data from Early Access Program for ND4-LHON Patients at NANOS 2023,” Press Release, March 15, 2023 |
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