GenSight Biologics Reports Cash Position as of June 30, 2025
"The second quarter of 2025 marked a period of significant operational progress for GenSight Biologics."
Share
GenSight Biologics (" GenSight Biologics" or the " Company") (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 reported its cash position as of June 30, 2025.
"The second quarter of 2025 marked a period of significant operational progress for GenSight Biologics. We successfully completed the technology transfer of LUMEVOQ's upstream manufacturing process to our new partner Catalent, secured the ANSM's agreement to consider opening the AAC program in France, and closed our private placement financing in July, ' commented Jan Eryk Umiastowski, CFO of GenSight Biologics. ' We continue to actively pursue non-dilutive financing options to support our critical value-driving activities. With the AAC program launch planned for Q4 2025, we have established a clear financial pathway through H2 2026, when we expect to initiate our Phase III RECOVER trial and submit our marketing application to the MHRA of the UK."
Cash position as of June 30, 2025
GenSight Biologics' cash and cash equivalents totaled €0.3 million as of June 30, 2025, compared to €0.9 million as of March 31, 2025. With the equity-with-warrants-attached and pre-funded warrants financing announced on July 1, 2025 (approximately €3.9 million) and the anticipated collection of approximately additional €0.2 million in Research Tax Credit (CIR) in September, and based on current operations, plans, and assumptions, this balance should fund operations until early October 2025. EUR 0.7 million of the proceeds from the financing has been used for the repayment in principal on the convertible bonds held by Heights Capital through offset against their subscription.
The funds are insufficient to cover operational requirements for the next 12 months. However, the anticipated opening of the AAC program in France in Q4 2025 (targeted for October) will establish a clear cash pathway until H2 2026, when the company expects to initiate the RECOVER Phase III clinical trial and to submit the UK MHRA marketing application for LUMEVOQ ®. The company is pursuing strategic options that could generate funds for the Phase III trial and UK regulatory submission. These include:
Licensing of LUMEVOQ outside Europe and the United States
Exploring paid compassionate access programs in other countries
Release of the second tranche of the EIB loan
In the first quarter of 2026, the company will have a precise view of residual financing needs and will make a strategic decision on additional financing modalities. If necessary, GenSight Biologics may access financial markets. Options in the medium term include partnering or M&A arrangements.
Recapitulation of Recent Milestones
Regulatory Progress and Named Early Access Program (AAC)
In June 2025, GenSight achieved a significant regulatory milestone when the ANSM agreed to consider opening the LUMEVOQ ® AAC program, contingent upon approval of a dose-ranging study. The Company has submitted a preliminary study design to the agency and expects to finalize the protocol in Q3 2025. Concurrently, GenSight is working with the ANSM to establish AAC program access for patients who may not qualify for the study but could benefit from early access to LUMEVOQ ®. The AAC program is targeted to launch by Q4 2025, with an anticipated opening in October 2025.
Manufacturing Partnership and Scale-Up
In June 2025, the Company successfully completed the transfer of LUMEVOQ ® 's upstream manufacturing process to its new manufacturing partner, Catalent, Inc. The technology transfer is expected to be finalized by the end of 2025, positioning Catalent to manufacture the drug product for both the planned global Phase III RECOVER trial and regulatory submission requirements.
Number of outstanding shares
As of July 8, 2025, GenSight Biologics' number of outstanding shares was 152,717,762 ordinary shares.
Financial Agenda
The Company will report its interim H1 financial results by the end of September 2025.
About GenSight Biologics S.A.
GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics' pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics' lead product candidate, GS010 (lenadogene nolparvovec) is in Phase III in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics' product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.
Forward-Looking Statements
This press release contains forward-looking statements. All statements, other than statements of historical facts, included in this press release are forward-looking statements. These forward-looking statements include, but are not limited to, statements regarding the completion expected proceeds and anticipated use of proceeds of the Private Placement; the anticipated cash runway of the Company; and future expectations, plans, and prospects of the Company. Words such as 'anticipates,' 'believes,' 'expects,' 'intends,' 'projects,' and 'future' or similar expressions are intended to identify forward-looking statements. These forward-looking statements are subject to the inherent uncertainties in predicting future results and conditions and no assurance can be given that the proposed securities offering discussed above will be consummated on the terms described or at all. Completion of the proposed Private Placement and the terms thereof are subject to numerous factors, many of which are beyond the control of the Company, including, without limitation, market conditions, failure of customary closing conditions and the risk factors and other matters set forth in the filings the Company makes with the AMF from time to time. The Company expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise, except as may be required by law.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Associated Press
2 days ago
- Associated Press
Artelo Biosciences Receives Favorable UK MHRA Guidance for a Phase 1 Trial of ART12.11, the Company's Proprietary CBD:TMP Cocrystal Being Developed for the Treatment of Anxiety and Depression
SOLANA BEACH, Calif., Aug. 01, 2025 (GLOBE NEWSWIRE) -- Artelo Biosciences, Inc. ('Artelo' or the 'Company') (Nasdaq: ARTL), a clinical-stage pharmaceutical company focused on modulating lipid-signaling pathways to develop treatments for people living with cancer, pain, dermatologic, or neurological conditions, today announced that it has received written scientific advice from the UK's Medicines and Healthcare products Regulatory Agency (MHRA) on the nonclinical development and first-in-human (FIH) clinical study plans for ART12.11, a novel cocrystal of Cannabidiol (CBD) and Tetramethylpyrazine (TMP). The MHRA agreed that reliance on the substantial body of historic nonclinical and clinical evidence for CBD, alongside legacy data for TMP, provides a scientifically justified basis for a streamlined clinical trial application (CTA)-enabling nonclinical development plan of the cocrystal combination to support the proposed FIH study, with clear guidance for achieving the agreed-upon FIH study design for ART12.11. The agency also affirmed that the proposed first-in-human (FIH) study design—a single-dose, multi-formulation crossover study—was methodologically sound for characterizing ART12.11's pharmacokinetic profile. Importantly, the MHRA offered constructive and specific guidance for completing the data package supporting the agreed-upon Phase 1 trial. Additionally, the agency proposed that ART12.11 may be a candidate for the Innovative Licensing and Access Pathway (ILAP). ILAP offers a unique opportunity to accelerate the development and patient access of promising new therapies through early and sustained collaboration with the MHRA, National Health Service, and health technology assessment bodies. Given ART12.11's novel mechanism and potential to address unmet needs in anxiety and depression, Artelo believes the program aligns well with ILAP's criteria and will evaluate a formal application to enter the pathway in the coming months. 'It's gratifying to receive this positive feedback and actionable recommendations from the MHRA, which provides a clear path forward as we prepare to initiate clinical studies with ART12.11,' said Dr. Andrew Yates, Chief Scientific Officer at Artelo. 'The recommendation to explore ILAP reinforces the proposition of ART12.11 as a novel drug with the potential to transform the treatment landscape for anxiety and depression.' Multiple nonclinical studies with ART12.11 have shown its promising profile compared to an antidepressant or CBD alone. The Company recently announced positive preclinical results in a depression model comparing ART12.11 with sertraline (Zoloft), a selective serotonin reuptake inhibitor (SSRI), where Artelo's patented CBD:TMP cocrystal demonstrated efficacy on par with sertraline and showed superior cognitive restoration compared to the leading SSRI. Additionally, in a rodent model of stress-induced anxiety and depression, where ART12.11 was compared to CBD dosed at 300% the amount of CBD contained in the oral tablet of ART12.11, the CBD:TMP cocrystal demonstrated efficacy where CBD alone did not. 'The clear regulatory assurance from the regulatory authority in the UK is expected to reduce expenses for our ART12.11 program,' added Gregory Gorgas, President & CEO at Artelo. 'We are especially pleased with the potential for an accelerated development strategy which could greatly accelerate our progress with ART12.11 and could provide for a longer period of market exclusivity as our patents are valid in 20 countries through the end of 2038. Over the next few months we look forward to finalizing our preparations to enter the clinic with ART12.11 early next year.' About ART12.11 ART12.11 is Artelo's wholly owned, proprietary cocrystal composition of cannabidiol (CBD) and tetramethylpyrazine (TMP). Isolated as a single crystalline form, ART12.11 has exhibited better pharmacokinetics and improved efficacy compared to other forms of CBD in nonclinical studies. Greatly enhanced pharmaceutical properties, including physicochemical, pharmacokinetic, and pharmacodynamic advantages have been observed with ART12.11. Artelo believes a more consistent and improved bioavailability profile in a solid dosage form may ultimately lead to increased safety and efficacy in humans, thus making ART12.11 a preferred CBD pharmaceutical composition. The U.S. issued composition of matter patent for ART12.11 is enforceable until December 10, 2038, and has now been granted or validated in 19 additional countries. About Artelo Biosciences Artelo Biosciences, Inc. is a clinical stage pharmaceutical company dedicated to the development and commercialization of proprietary therapeutics that modulate lipid-signaling pathways. Artelo is advancing a portfolio of broadly applicable product candidates designed to address significant unmet needs in multiple diseases and conditions, including anorexia, cancer, anxiety, dermatologic conditions, pain, and inflammation. Led by proven biopharmaceutical executives collaborating with highly respected researchers and technology experts, the Company applies leading edge scientific, regulatory, and commercial discipline to develop high-impact therapies. More information is available at and Twitter: @ArteloBio. Forward Looking Statements Investor Relations Contact: Crescendo Communications, LLC Tel: 212-671-1020 Email: [email protected]
Medscape
4 days ago
- Medscape
MHRA Approves First Targeted Drug for SOD1-ALS
The Medicines and Healthcare products Regulatory Agency (MHRA) has approved tofersen (Qalsody, Biogen) for treating amyotrophic lateral sclerosis (ALS) in adults with mutations in the superoxide dismutase 1 (SOD1) gene, also known as SOD1-ALS. The approval makes tofersen the first targeted therapy in the UK against this rare form of motor neurone disease (MND) with a genetic basis. A Rare but Aggressive Condition SOD1-ALS causes the body to produce a misfolded, toxic version of the SOD1 protein. This triggers motor neuron degeneration in the brain and spinal cord. Patients experience progressive muscle weakness, atrophy, respiratory decline, and eventual paralysis and death. SOD1 mutations are responsible for approximately 2% of all ALS cases globally. Over 200 distinct mutations in SOD1 have been identified, each contributing to a wide range of disease progression rates. This variability highlights the importance of developing targeted therapies for SOD1-ALS. How Tofersen Works Tofersen is an antisense oligonucleotide that binds to SOD1 mRNA, promoting its degradation through an RNase H-mediated mechanism. This reduces the production of the toxic SOD1 protein. The drug is administered via intrathecal injection through a lumbar puncture at scheduled intervals, under the supervision of trained healthcare professionals. Success in Clinical Trials The approval was supported by data from the phase 3 VALOR study and its open-label extension (OLE). In the VALOR trial, 108 adults with SOD1-ALS were treated with either intrathecal tofersen (n=72) or placebo (n=36) for 28 weeks. The primary endpoint, the ALSFRS-R score — which assesses bulbar, motor, and respiratory functions — showed a positive trend with tofersen, although the difference was not significant. However, tofersen significantly reduced biomarkers of neurodegeneration, including cerebrospinal fluid (CSF) SOD1 protein levels and plasma neurofilament light chain (NfL) concentrations. By week 28, plasma NfL levels decreased by 55% in the tofersen group, compared to a 12% increase with placebo. Clinical trends suggested improvements in respiratory function, muscle strength, and quality of life. These effects were more pronounced with earlier treatment and during the OLE phase. Safety and Tolerability The most common side effects in tofersen-treated patients included: Back, muscle, and joint pain Fatigue Fever Raised CSF protein levels or white blood cell counts These reactions were generally mild to moderate in severity. Further information, including the Patient Information Leaflet and Summary of Product Characteristics, will be available on the MHRA website within 7 days of approval.
Business Wire
6 days ago
- Business Wire
Genethon to Launch Pivotal Trial in Europe of GNT0004 a Low-Dose Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy
PARIS--(BUSINESS WIRE)--Genethon, a worldwide pioneer and leader in research and development in gene therapy for rare genetic diseases, has received approvals from regulatory authorities, MHRA and EMA*, to begin pivotal Phase 3 clinical trials in France and the UK of its gene therapy, GNT0004, for Duchenne muscular dystrophy (DMD). Genethon CEO Frederic Revah observed, 'We are delighted to be able to continue these trials and are determined to bring GNT0004 to market for young patients and their families who are waiting for a therapeutic solution. This marks a decisive step forward for our gene therapy program for DMD, which began in 2021 and has demonstrated extremely promising results in the first children treated in the Phase1/2 portion of our Phase1/2/3 study.' Dr. Revah added, 'In addition to the very positive results in patients treated in the early phases, one of the strengths of our product is the dose selected for the pivotal phase, which is lower than those used in other gene therapy trials for DMD. Approvals of our Phase 3 trials reflect the regulatory authorities' confidence in GNT0004 as well as the work accomplished by our teams.' The Phase 3 authorizations in Europe are based on the results of Phase 1/2 studies showing good tolerance of GNT0004 as well as efficacy in terms of microdystrophin expression, creatine phosphokinase (CPK) reduction, and motor function. Patients showed prolonged improvement or stabilization of motor functions and significant persistent reduction in CPK, a key marker of muscle damage. The Phase 3 double blind trials will begin in August and September in the UK and France using a single intravenous injection of GNT0004, which contains an optimized hMD1 transgene, a shortened (3x10¹³ vg/kg microdystrophin) but functional version of the gene encoding dystrophin in an AAV8 vector associated with transient immunological prophylactic treatment. The vector is designed to express itself in muscle tissue and the heart thanks to a Spc5-12 promoter sequence specific to these tissues. A total of 64 boys aged 6 to 10 with DMD who have retained their walking ability will be enrolled. * French ANSM as reporting member state for the EMA About Duchenne muscular dystrophy Duchenne muscular dystrophy is a rare progressive genetic disease that affects all the muscles in the body and mainly boys (1 in 5,000). It is caused by abnormalities in the gene responsible for the production of dystrophin, a structural protein essential for the stability of muscle fiber membranes and their metabolism. The absence of dystrophin leads to progressive degeneration of the skeletal and cardiac muscles, loss of walking and respiratory capacity, progressive heart failure, and death between the ages of 20 and 40. About Genethon A pioneer in the discovery and development of gene therapies for rare diseases, Genethon is a non-profit laboratory created by the AFM-Telethon. A first gene therapy drug, to which Genethon contributed, has been approved for marketing for spinal muscular atrophy. With more than 240 scientists and experts, Genethon's goal is to develop innovative therapies that change the lives of patients suffering from rare genetic diseases. Thirteen gene therapy products developed by Genethon or to which Genethon has contributed are currently undergoing clinical trials for diseases of the liver, blood, immune system, muscles, and eyes. Seven other products are in preparation for clinical trials over the next five years.
