MeiraGTx Holdings Plc

Results from the first four children to be treated have been detailed in a research paper. Credit: Tom Merton via Getty Images. MeiraGTx is advancing its investigational gene therapy toward expedited approval in the UK after 11 children who were born blind from birth gained significant vision improvements following treatment. The AAV-AIPL1 therapy is designed to address severe sight impairment caused by mutations in the AIPL1 gene. This condition is a form of retinal dystrophy that leads to severe visual impairment from birth, with affected individuals typically only able to distinguish between light and dark. The gene therapy involves injecting functional copies of the AIPL1 gene into the retina using an adeno-associated viral (AAV) vector, aiming to restore retinal cell function and prevent degeneration. Results from four of the 11 children were published in The Lancet on 21 February. The first four children treated were aged one to three years with severe retinal dystrophy linked to mutations in the AIPL1 gene. Each child received the therapy in one eye through subretinal injection. Outcome measures included visual acuity assessments, functional vision evaluations, visual evoked potentials, and retinal structure imaging. Before treatment, the children’s visual acuity was limited to light perception. At an average follow-up of three and a half years post-treatment, the treated eyes showed significant improvement, with visual acuity improving. In contrast, the untreated eyes’ visual acuity deteriorated to unmeasurable levels. Additionally, objective tests confirmed enhanced visual function and electrophysiological assessments indicated increased visual cortex activity specific to the treated eyes. Imaging revealed better preservation of retinal structure in treated eyes compared to untreated ones. MeiraGTx’s CEO Alexandria Forbes said: “These improvements extended outside the meaningful effects on vision and result in life-changing benefits in all areas of development including communication, behaviour, schooling, mood, psychological benefits and social integration.” Following these positive outcomes, a second cohort of seven children received treatment in both eyes. All 11 children treated with AAV-AIPL1 have so far exhibited meaningful visual improvements. One child experienced cystoid macular oedema in the treated eye, which partially improved over time and did not hinder the overall visual gains. No other significant safety concerns have been reported. In light of these findings, MeiraGTx has engaged in discussions with the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) and plans to submit AAV-AIPL1 for expedited approval. The company is also in talks with the US Food and Drug Administration (FDA) to explore accelerated approval pathways in the US. If approved, AAV-AIPL1 would be eligible for a priority review voucher (PRV). While gene therapies hold promise for rare diseases, commercialisation remains a challenge. High development costs, complex manufacturing processes, and limited patient populations often deter investment. Earlier this month, Italian charity Telethon Foundation submitted a marketing authorisation application (MAA) to the European Medicines Agency (EMA) for a gene therapy targeting Wiskott-Aldrich syndrome after the withdrawal of its commercial partner. This marks the second time the foundation has stepped in to support an abandoned gene therapy, following its efforts with simoladagene autotemcel for ADA-SCID. The foundation claims to be the first charity worldwide to take responsibility for drug production and distribution, highlighting the financial and logistical hurdles in bringing gene therapies to market. The incidence of AIPL1-associated severe retinal dystrophy is estimated to be around one in every million live births. During a call with investors, Forbes stated that the company is exploring all options to ensure global access to AAV-AIPL1, noting “strategic interest” in the therapy. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Optimise your cell therapy process: a guide to cell thawing Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. 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These are “strong clinical data with clear evidence of efficacy in a huge unmet need pediatric population,” analysts wrote.\n Eleven children who were legally blind at birth all gained visual acuity after receiving MeiraGTx\'s investigational gene therapy, data that are prompting the biotech to seek accelerated approval in AIPL1-associated severe retinal dystrophy.Results from four of the 11 children were published in The Lancet Feb. 21.Visual acuity—measured with a child-friendly, game-like touchscreen test—in treated eyes improved after four or more weeks, while the acuity in untreated eyes became unmeasurable, according to the published results.The effects of treatment “extended outside the meaningful effects on vision and result in life-changing benefits in all areas of development including communication, behavior, schooling, mood, psychological benefits and social integration,” MeiraGTx\'s CEO Alexandria Forbes, Ph.D., said in the biotech\'s accompanying release. One child developed cystoid macular edema, a build-up of fluid, in their treated eye due to the therapy, researchers wrote in the article, which partially improved over time and didn’t prevent the child’s vision from improving. There were no other safety concerns, the scientists said.Evercore ISI analysts described the findings as “strong clinical data with clear evidence of efficacy in a huge unmet need pediatric population.”  “As should be expected from a subretinal approach, safety was clean,” the analysts added in a Feb. 21 note.After positive conversations with the U.K.’s medicines watchdog, the firm now plans to submit the gene therapy, called rAAV8.hRKp.AIPL1, for expedited approval in that country, according to the release.MeiraGTx, which is headquartered in New York and has R&D and manufacturing in England, has also been in discussions with the FDA on how to pursue accelerated approval in the U.S., the company added.The therapy uses an adeno-associated virus to deliver functional copies of the AIPL1 gene and is injected once directly into the eye.The study began with four children between the ages of one and three who were born with severe retinal dystrophy associated with mutated copies of the AIPL1 gene. These children were each treated in one eye. After the researchers saw the treatment was safe and seemed to be working, they gave the gene therapy in both eyes of a further seven children.

Plus, news about MeiraGTx, Hookipa and Poolbeg Pharma: A pair of $500M share buybacks: Exelixis and Neurocrine Biosciences each laid out plans to buy back half a billion dollars worth of their shares. The California drugmakers are not new to the share repurchase trend . Neurocrine completed a $300 million program earlier this month, and Exelixis is currently completing an earlier announced $500 million program. — Kyle LaHucik Cosette Pharmaceuticals buys Mayne Pharma Group: The roughly $430 million acquisition is expected to close in the second quarter. The deal bolsters Cosette’s dermatology and women’s health units and brings its total number of branded products to 12. Cosette is known for marketing Vyleesi, a treatment for low sexual desire in women. — Ayisha Sharma MeiraGTx to ask UK authorities for approval of gene therapy for genetic blindness: The biotech shared detailed results for 11 children in the UK with an ultra-rare form of genetic blindness who all saw their vision improve after receiving the gene therapy. It now plans to ask the MHRA for approval based on these data, and it is also speaking with the FDA to “discuss a path forward for expedited approval in the United States.” — Lei Lei Wu Hookipa calls off acquisition talks with Poolbeg Pharma: Poolbeg said it was “surprised and disappointed” by Hookipa’s decision, but will consider other strategies to maximize shareholder value. The company’s UK-listed shares fell by around 22% on Friday morning. Hookipa first announced its plans to buy Poolbeg in January. — Ayisha Sharma