Optic Atrophy

Cash position amounts to €3.4 million as of September 30, 2024. LUMEVOQ ® drug product has passed all quality control tests; submission to ANSM supporting AAC resumption now scheduled for mid-November and first patient injection expected by end of December, with first payment expected between late December 2024 and early January 2025. Advanced discussions ongoing to secure financing until resumption of early access program (AAC). PARIS--(BUSINESS WIRE)--Regulatory News: 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 September 30, 2024, and provided a business update. Cash Position and Outlook GenSight Biologics’ cash and cash equivalents totalled €3.4 million as of September 30, 2024, compared to €6.9 million as of June 30, 2024. The Company does not have sufficient net working capital to meet its obligations over the next 12 months but only until mid-November 2024. The Company expects to begin receiving indemnities from the resumption of the Early Access Program (AAC) between late December 2024 and early January 2025 (vs. November 2024 as previously announced). This will extend the cash runway to Q4 2026. To manage the impact of the delayed resumption of AAC program and to account for the time until receipt of the first compensation payments, the Company is currently engaged in active discussions to secure financing for its activities from mid-November until it begins to receive AAC payments. GenSight Biologics has obtained waivers from its existing creditors, the European Investment Bank and Heights Capital Management, enabling the Company to pursue additional financing options. “ The small amount of bridge financing that we are trying to raise from existing and new investors will extend our cash runway to early January 2025, sufficient to overcome the delay we have experienced in the resumption of the French Early Access Program, ” noted Jan Eryk Umiastowski , Chief Financial Officer of GenSight Biologics. “ We are in an excellent position to resume the Early Access Program in the coming weeks, which should extend our cash runway to Q4 2026. We continue work on optimizing our cash management and ensuring a sustainable future for the Company .” LUMEVOQ ® Update LUMEVOQ ® drug product manufacture, which included the mixing of the 2 GMP drug substance batches produced in 2023, is now fully complete, with more than 100 vials available. The drug product has successfully passed all quality control tests required to release the product for human use. Vials are stored in France, labelled and ready to be supplied once the release is documented and the regulatory green light is obtained. GenSight Biologics is currently preparing the Good Manufacturing Practice (GMP) documentation and anticipates submitting the dossier to support the AAC resumption to the Agence Nationale de Sécurité du Médicament et des Produits de Santé (ANSM) by mid-November 2024, in accordance with the requirements formulated by the ANSM. Requests for compassionate treatment could subsequently be sent again to the ANSM by healthcare professionals. The Company anticipates a review period for these applications and will work closely with the ANSM to optimize the assessment timeline. GenSight Biologics will be ready to initiate the supply of the drug to the treatment center in mid-December, rather than in mid-November as previously announced. The first patient injections are expected by end of December 2024, as envisaged with the administrative and medical teams of the Quinze-Vingts hospital in Paris. “The successful production of a new GMP batch of LUMEVOQ ® marks a significant milestone in our journey to bring this innovative therapy once again to patients,” commented Laurence Rodriguez , Chief Executive Officer of GenSight Biologics. “ We know that many patients are waiting for this treatment. We are ready to answer any questions from the ANSM and share their commitment to treat these patients as quickly as possible.” The Company did not initiate requests for compassionate treatment in Italy. Such requests are the responsibility of Italian physicians who seek to obtain the authorization, which is only rarely granted by the Italian medicines agency AIFA ( Agenzia Italiana del Farmaco ). The requests from Italian physicians signal a high unmet medical need in Leber Hereditary Optic Neuropathy (LHON). The paid compassionate access program AAC is unique to France and has no bearing on programs in other countries. Share Information As of September 30, 2024, GenSight Biologics had 107,686,349 outstanding ordinary shares. About GenSight Biologics 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, LUMEVOQ ® (GS010; lenadogene nolparvovec), is an investigational compound and has not been registered in any country at this stage; a marketing authorization application is currently under review by the EMA for the treatment of Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease affecting primarily teens and young adults that leads to irreversible blindness. 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, including statements regarding product development prospects and financial projections. These statements do not constitute guarantees of future performance and involve risks and uncertainties. A further list and description of risks and uncertainties that could cause actual results to differ materially from those set forth in the forward-looking statements in this press release can be found in GenSight Biologics’ regulatory filings with the French Autorité des Marchés Financiers . Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements and estimates, which speak only as of the date hereof. Other than as required by applicable law, GenSight Biologics undertakes no obligation to update or revise the information contained in this press release. Contacts GenSight Biologics Chief Financial Officer Jan Eryk Umiastowski jeumiastowski@gensight-biologics.com LifeSci Advisors Investor Relations Guillaume van Renterghem gvanrenterghem@lifesciadvisors.com +41 (0)76 735 01 31

Today, a brief rundown of news from PTC Therapeutics and Novartis, as well as updates from Amylyx Pharmaceuticals and Intercept Pharmaceuticals that you may have missed.Europes drug regulator is standing firm in its decision not to renew the marketing permit for Translarna, a treatment developed by PTC Therapeutics for a rare muscle-wasting disease. The European Medicines Agency initially authorized Translarna in 2014, with the condition PTC gather more data to show the drug is effective in certain cases of Duchenne muscular dystrophy. But after four reviews of the evidence now available, the EMA concluded Translarnas effectiveness still has yet to be confirmed. The agency will send its verdict to the European Commission, which will make the final decision on the drugs status for EU countries. Jacob BellNovartis is paying China-based Chengdu Baiyu Pharmaceutical $70 million for global licensing rights to a single small molecule cancer drug. The deal, which includes up to $1.1 billion in additional payments based on achievement of development and commercial milestones, doesnt specify any biological or disease targets for the drug. Chengdu Baiyu said it is has “focused research on the core pharmacological components of Ginkgo biloba including development of botanical drugs. It has six cancer-related experimental drugs in its pipeline. Jonathan GardnerShares of Amylyx Pharmaceuticals oscillated Thursday with the release of data for one the companys two main drugs. Amylyx said the drug, known as AMX0035, succeeded in a small study of patients with Wolfram Syndrome, a rare, inherited condition that damages organs like the eyes, brain and pancreas. The company now plans to meet with stakeholders, including the Food and Drug Administration, for input on Phase 3 testing, and expects to provide updates on that program next year. AMX0035 had been cleared as a treatment for ALS, but was pulled from the market in April after an important follow-on trial found it wasnt substantially better than a placebo at treating the nerve-destroying disorder. Jacob BellThe Food and Drug Administration has delayed a decision on full approval of Intercept Pharmaceuticals Ocaliva in primary biliary cholangitis, a condition for which the Alfasigma subsidiary won accelerated approval in 2016. The FDA delayed its Oct. 15 decision deadline, but didnt provide a new action date, Intercept said in a statement. The delay follows a negative vote by a panel of independent FDA advisers on Sept. 13. That committee concluded confirmatory trials intended to justify full approval didnt provide enough evidence Ocaliva prevented liver transplants and death in people with the rare disorder. Ocaliva is Intercepts only drug, and generated $286 million in sales in 2022, Intercepts last full year as an independent company. Jonathan GardnerThe Australian agency that regulates pharmaceuticals has decided not to clear for market Leqembi, the closely watched Alzheimers disease drug from Eisai and Biogen. In a statement Wednesday, the Therapeutic Goods Administration said that, based on the available evidence, Leqembis safety risks outweigh its potential benefits. Eisai plans on asking the agency to reconsider its decision. While its approved in the U.S. for people in an early stage of Alzheimers, Leqembi has been met with resistance in Europe and, now, Australia, where estimates hold there are more than 400,000 people with dementia. Jacob BellAmicus has staved off competition for its biggest seller, Fabry disease drug Galafold, until 2037 under the terms of a settlement with Teva Pharmaceuticals. Amicus granted Teva a license to begin selling a generic version of Galafold on Jan. 30 of that year, and the two sides have agreed to drop all federal litigation over Galafold patents. The company continues to fight a second generic manufacturer, Aurobindo, in court. A stay is in place on litigation with a third generic manufacturer, Lupin. Jonathan Gardner '

Amylyx is trying to move forward with its effort to turn its former ALS drug into a treatment for other conditions, and data released Thursday build on a small interim dataset from earlier this year that showed signs of improving outcomes in a rare genetic disease. Wolfram syndrome usually manifests in childhood and patients experience symptoms typically seen in diabetes (high blood sugar, high urine production) that may progress to blindness and deafness. The Phase 2 study of Amylyx’s drug, AMX0035, measured whether patients’ average levels of an amino acid called C-peptide increased after 24 weeks. C-peptide response rates are traditionally used to determine the health of diabetes patients’ pancreatic cells and how well they function, according to the NIH . In order to look at how patients responded to AMX0035, Amylyx asked 12 patients to take a “mixed meal tolerance test,” in which patients ingest a “standardized liquid meal” in the mornings with their C-peptide responses measured over time. Usually, C-peptide responses peak after 30 minutes in non-diabetic individuals, but Wolfram syndrome patients’ levels peak more slowly, after about 120 minutes. After 12 patients took AMX0035 for 24 weeks, the drug improved C-peptide responses at the 120-minute mark by an average of 3.8 minutes by ng/mL compared to baseline levels. Response rates continued to improve at 36 and 48 weeks, Amylyx said. But one of the patients did not meet the trial’s inclusion and exclusion criteria, possessing just one of the two alleles necessary to categorize the disease. Amylyx also presented an 11-patient dataset removing this individual, showing improved C-peptide responses at the 120-minute mark of an average of 20.2 minutes by ng/mL compared to baseline levels. In an investor call Thursday afternoon, co-CEO Josh Cohen said the company would look to implement stricter genetic screening measures in a Phase 3 trial to prevent this error from happening again. The company is continuing to discuss what a study might look like, including whether it will seek accelerated approval, and will reveal more details next year. AMX0035 was previously approved to treat ALS under the brand name Relyvrio. The drug eventually failed a Phase 3 study and forced Amylyx to voluntarily withdraw it from the market. That failure caused a huge plunge in the company’s stock and led to layoffs. Shares $AMLX were up as much as 7% Thursday.