{"id":57716,"date":"2023-07-10T10:03:45","date_gmt":"2023-07-10T08:03:45","guid":{"rendered":"https:\/\/pharma-trend.com\/en\/sensorion-submits-clinical-trial-application-for-lead-gene-therapy-candidate-otof-gt-in-the-uk\/"},"modified":"2023-07-10T10:03:45","modified_gmt":"2023-07-10T08:03:45","slug":"sensorion-submits-clinical-trial-application-for-lead-gene-therapy-candidate-otof-gt-in-the-uk","status":"publish","type":"post","link":"https:\/\/pharma-trend.com\/en\/sensorion-submits-clinical-trial-application-for-lead-gene-therapy-candidate-otof-gt-in-the-uk\/","title":{"rendered":"Sensorion Submits Clinical Trial Application for Lead Gene Therapy Candidate OTOF-GT in the UK"},"content":{"rendered":"<div>\n<p>MONTPELLIER, France&#8211;(BUSINESS WIRE)&#8211;Regulatory News:<\/p>\n<p><a href=\"https:\/\/mms.businesswire.com\/media\/20230709452628\/en\/1836116\/5\/logo_sensorion_3-2-1-RGB.jpg\"><img decoding=\"async\" src=\"https:\/\/mms.businesswire.com\/media\/20230709452628\/en\/1836116\/21\/logo_sensorion_3-2-1-RGB.jpg\"><\/a><br \/><a href=\"https:\/\/mms.businesswire.com\/media\/20230709452628\/en\/1836116\/5\/logo_sensorion_3-2-1-RGB.jpg\"><img decoding=\"async\" src=\"https:\/\/mms.businesswire.com\/media\/20230709452628\/en\/1836116\/22\/logo_sensorion_3-2-1-RGB.jpg\"><\/a><\/p>\n<p>\n<b>Sensorion (FR0012596468 \u2013 ALSEN) <\/b>(Paris:ALSEN)<b> <\/b>a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders, today announced that it has submitted a first Clinical Trial Application (CTA) for OTOF-GT to the UK\u2019s Medicines and Healthcare products Regulatory Agency (MHRA).<\/p>\n<p>\nThe phase 1\/2 clinical trial (Audiogene), aims to evaluate the safety, tolerability and efficacy of intra-cochlear injection of OTOF-GT, for the treatment of otoferlin gene-mediated hearing loss in pediatric patients aged up to 31 months. The CTA submission follows extensive preclinical studies assessing the safety and efficacy of OTOF-GT and successful manufacturing of the gene therapy Drug Product for the clinical trial.<\/p>\n<p>\nSensorion\u2019s OTOF-GT dual AAV vector gene therapy development program aims to restore hearing in patients with mutations in OTOF who suffer from severe to profound sensorineural prelingual non syndromic hearing loss. Otoferlin is a protein expressed in the inner hair cells (IHC) present in the cochlea and is critical for the transmission of the signal to the auditory nerve. Otoferlin related hearing loss is responsible for up to 8% of all cases of congenital hearing loss, with around 20,000 people affected in the US and Europe<sup>1<\/sup>. OTOF-GT previously received Orphan Drug Designation from the US Food and Drug Administration (FDA)<sup>2<\/sup> and the European Medicines Agency (EMA)<sup>3<\/sup> and Rare Pediatric Disease Designation from the FDA in Q4 2022.<\/p>\n<p>\n<b>Nawal Ouzren, Chief Executive Officer of Sensorion<\/b>, commented: \u201cThis first gene therapy CTA filing is a major milestone for our OTOF-GT program and Sensorion\u2019s broader gene therapy franchise. This is the first of several planned CTA filings and we hope to commence patient recruitment soon, subject to the CTA approval. We look forward to continuing to work with clinicians, regulatory authorities and patient groups to address unmet and underserved medical needs in the intricate hearing space.\u201d<\/p>\n<p>\n<b>G\u00e9raldine Honnet, Chief Medical Officer of Sensorion<\/b>, added: \u201cThere are currently no approved drug for patients with mutations of the gene encoding for otoferlin. Our goal is to transform the standard of care for OTOF newborns, by reducing dependence on cochlear implants, which would transform their quality of life. We believe that gene therapy has the potential to offer permanent solutions to patients with diseases caused by the OTOF mutation and other inner ear diseases and we are really excited to move OTOF-GT forward towards clinical development.\u201d<\/p>\n<p>\nSensorion plans to submit the CTA in Europe in the coming weeks.<\/p>\n<p>\n<b>About Sensorion<\/b><\/p>\n<p>\nSensorion is a pioneering clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat and prevent hearing loss disorders, a significant global unmet medical need.<br \/>\n<br \/>Sensorion has built a unique R&amp;D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases, enabling it to select the best targets and mechanisms of action for drug candidates.<br \/>\n<br \/>It has two gene therapy programs aimed at correcting hereditary monogenic forms of deafness, developed in the framework of its broad strategic collaboration focused on the genetics of hearing with the Institut Pasteur. OTOF-GT targets deafness caused by mutations of the gene encoding for otoferlin and GJB2-GT targets hearing loss related to mutations in <i>GJB2<\/i> gene to potentially address important hearing loss segments in adults and children. The Company is also working on the identification of biomarkers to improve diagnosis of these underserved illnesses.<br \/>\n<br \/>Sensorion\u2019s portfolio also comprises clinical-stage small molecule programs for the treatment and prevention of hearing loss disorders.<br \/>\n<br \/>Sensorion\u2019s clinical-stage portfolio includes one Phase 2 product: SENS-401 (Arazasetron) progressing in a planned Phase 2 proof of concept clinical study of SENS-401 in Cisplatin-Induced Ototoxicity (CIO) and, with partner Cochlear Limited, in a study of SENS-401 in patients scheduled for cochlear implantation. A Phase 2 study of SENS-401 was also completed in Sudden Sensorineural Hearing Loss (SSNHL) in January 2022.<br \/>\n<br \/><a target=\"_blank\" href=\"https:\/\/cts.businesswire.com\/ct\/CT?id=smartlink&amp;url=http%3A%2F%2Fwww.sensorion.com&amp;esheet=53441932&amp;newsitemid=20230709452628&amp;lan=en-US&amp;anchor=www.sensorion.com&amp;index=1&amp;md5=48d48112576a5fa20c4fcaef5d0535f1\" rel=\"nofollow noopener\" shape=\"rect\">www.sensorion.com<\/a><\/p>\n<p>\n<b>Disclaimer<\/b><\/p>\n<p>\nThis press release contains certain forward-looking statements concerning Sensorion and its business. Such forward looking statements are based on assumptions that Sensorion considers to be reasonable. However, there can be no assurance that such forward-looking statements will be verified, which statements are subject to numerous risks, including the risks set forth in the 2022 full year financial report published on March 30, 2023, and available on our website and to the development of economic conditions, financial markets and the markets in which Sensorion operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Sensorion or not currently considered material by Sensorion. The occurrence of all or part of such risks could cause actual results, financial conditions, performance or achievements of Sensorion to be materially different from such forward-looking statements. This press release and the information that it contains do not constitute an offer to sell or subscribe for, or a solicitation of an offer to purchase or subscribe for, Sensorion shares in any country. The communication of this press release in certain countries may constitute a violation of local laws and regulations. Any recipient of this press release must inform oneself of any such local restrictions and comply therewith.<\/p>\n<p>\n_______________________________<br \/>\n<br \/><sup>1<\/sup> Rodr\u00edguez-Ballesteros M, Reynoso R, Olarte M, Villamar M, Morera C, Santarelli R, Arslan E, Med\u00e1 C, Curet C, V\u00f6lter C, Sainz-Quevedo M, Castorina P, Ambrosetti U, Berrettini S, Frei K, Ted\u00edn S, Smith J, Cruz Tapia M, Cavall\u00e9 L, Gelvez N, Primignani P, G\u00f3mez-Rosas E, Mart\u00edn M, Moreno-Pelayo MA, Tamayo M, Moreno-Barral J, Moreno F, del Castillo I. A multicenter study on the prevalence and spectrum of mutations in the otoferlin gene (OTOF) in subjects with nonsyndromic hearing impairment and auditory neuropathy. Hum Mutat. 2008 Jun;29(6):823-31. doi: 10.1002\/humu.20708. PMID: 18381613.<br \/>\n<br \/><sup>2<\/sup> FDA Orphan Drug Designations and Approvals <a target=\"_blank\" href=\"https:\/\/cts.businesswire.com\/ct\/CT?id=smartlink&amp;url=https%3A%2F%2Fwww.accessdata.fda.gov%2Fscripts%2Fopdlisting%2Foopd%2FlistResult.cfm&amp;esheet=53441932&amp;newsitemid=20230709452628&amp;lan=en-US&amp;anchor=https%3A%2F%2Fwww.accessdata.fda.gov%2Fscripts%2Fopdlisting%2Foopd%2FlistResult.cfm&amp;index=2&amp;md5=5aabf36a282e92a02d6a0764ed774f47\" rel=\"nofollow noopener\" shape=\"rect\">https:\/\/www.accessdata.fda.gov\/scripts\/opdlisting\/oopd\/listResult.cfm<\/a><br \/><sup>3<\/sup> EU Community Register of orphan medicinal products <a target=\"_blank\" href=\"https:\/\/cts.businesswire.com\/ct\/CT?id=smartlink&amp;url=https%3A%2F%2Fec.europa.eu%2Fhealth%2Fdocuments%2Fcommunity-register%2Fhtml%2Fo2698.htm&amp;esheet=53441932&amp;newsitemid=20230709452628&amp;lan=en-US&amp;anchor=https%3A%2F%2Fec.europa.eu%2Fhealth%2Fdocuments%2Fcommunity-register%2Fhtml%2Fo2698.htm&amp;index=3&amp;md5=e7faaf7a6382ec40b0243dddcab3dd5c\" rel=\"nofollow noopener\" shape=\"rect\">https:\/\/ec.europa.eu\/health\/documents\/community-register\/html\/o2698.htm<\/a><\/p>\n<p> <b>Contacts<\/b> <\/p>\n<p>\n<b>Investor Relations<\/b><br \/>No\u00e9mie Djokovic, Investor Relations and Communications (Europe\/France)<br \/>\n<br \/><a target=\"_blank\" href=\"&#109;&#x61;&#105;&#x6c;t&#111;&#x3a;&#105;&#x72;&#46;&#x63;o&#110;&#x74;&#97;&#x63;t&#x40;&#x73;&#101;&#x6e;s&#x6f;r&#105;&#x6f;&#110;&#x2d;p&#x68;a&#114;&#x6d;&#97;&#x2e;c&#111;&#x6d;\" rel=\"nofollow noopener\" shape=\"rect\">&#105;&#x72;&#46;&#x63;o&#x6e;t&#97;&#x63;&#116;&#x40;s&#x65;n&#115;&#x6f;&#114;&#x69;o&#x6e;-&#112;&#x68;&#97;&#x72;m&#x61;&#46;&#99;&#x6f;&#109;<\/a><\/p>\n<p>\n<b>International Media Relations<\/b><br \/>Consilium Strategic Communications<br \/>\n<br \/>Jessica Hodgson\/Sue Stuart\/Isabelle Abdou<br \/>\n<br \/>+44 7561 424788<br \/>\n<br \/><a target=\"_blank\" href=\"&#x6d;a&#x69;&#108;&#x74;&#111;:&#x53;&#101;&#x6e;&#115;o&#x72;&#105;&#x6f;&#110;&#64;&#x63;o&#x6e;&#115;&#x69;&#x6c;i&#x75;&#109;&#x2d;&#99;o&#x6d;&#109;&#x73;&#46;c&#x6f;m\" rel=\"nofollow noopener\" shape=\"rect\">Sensorio&#110;&#64;&#99;&#111;&#110;&#115;&#105;&#108;&#105;&#117;&#x6d;&#x2d;&#x63;&#x6f;&#x6d;&#x6d;&#x73;&#x2e;&#x63;&#x6f;&#x6d;<\/a><\/p>\n<p>\n<b>Ulysse Communication<\/b><br \/>Pierre-Louis Germain \/ 00 33 (0)6 64 79 97 51<br \/>\n<br \/><a target=\"_blank\" href=\"&#x6d;&#97;&#x69;&#108;t&#x6f;&#58;p&#x6c;&#103;&#x65;&#x72;m&#x61;&#105;n&#x40;&#117;&#x6c;&#x79;s&#x73;&#101;-&#x63;&#111;m&#x6d;&#117;&#x6e;&#105;c&#x61;&#116;i&#x6f;&#110;&#x2e;&#x63;o&#x6d;\" rel=\"nofollow noopener\" shape=\"rect\">&#x70;l&#x67;&#101;r&#x6d;&#97;&#x69;&#x6e;&#64;&#x75;&#108;y&#x73;&#115;&#x65;&#45;c&#x6f;&#109;m&#x75;&#110;&#x69;&#99;a&#x74;&#105;&#x6f;&#x6e;&#46;&#x63;&#111;m<\/a><\/p>\n<p>\nBruno Arabian \/ 00 00(0)6 87 88 47 26<br \/>\n<br \/><a target=\"_blank\" href=\"&#109;a&#105;l&#116;o&#x3a;b&#x61;r&#x61;b&#x69;a&#x6e;&#64;&#x75;l&#x79;s&#x73;e&#x2d;c&#x6f;&#109;&#x6d;&#117;&#x6e;&#105;&#x63;&#97;&#x74;&#105;&#x6f;&#110;&#x2e;&#99;&#x6f;&#109;\" rel=\"nofollow noopener\" shape=\"rect\">&#x62;&#x61;&#114;&#97;&#98;i&#x61;&#x6e;&#x40;&#117;&#108;ys&#x73;&#x65;&#x2d;&#99;&#111;m&#x6d;&#x75;&#x6e;&#105;&#99;&#97;t&#x69;&#x6f;&#x6e;&#46;&#99;om<\/a><\/p>\n<\/div>\n","protected":false},"excerpt":{"rendered":"<p>MONTPELLIER, France&#8211;(BUSINESS WIRE)&#8211;Regulatory News: Sensorion (FR0012596468 \u2013 ALSEN) (Paris:ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders, today announced that it has submitted a first Clinical Trial Application (CTA) for OTOF-GT to the UK\u2019s Medicines and Healthcare products &#8230; <span class=\"more\"><a class=\"more-link\" href=\"https:\/\/pharma-trend.com\/en\/sensorion-submits-clinical-trial-application-for-lead-gene-therapy-candidate-otof-gt-in-the-uk\/\">[Read more&#8230;]<\/a><\/span><\/p>\n","protected":false},"author":4,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[13],"tags":[],"class_list":{"0":"entry","1":"post","2":"publish","3":"author-business","4":"post-57716","6":"format-standard","7":"category-industry"},"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.5 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Sensorion Submits Clinical Trial Application for Lead Gene Therapy Candidate OTOF-GT in the UK - Pharma Trend<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/pharma-trend.com\/en\/sensorion-submits-clinical-trial-application-for-lead-gene-therapy-candidate-otof-gt-in-the-uk\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Sensorion Submits Clinical Trial Application for Lead Gene Therapy Candidate OTOF-GT in the UK - Pharma Trend\" \/>\n<meta property=\"og:description\" content=\"MONTPELLIER, France&#8211;(BUSINESS WIRE)&#8211;Regulatory News: Sensorion (FR0012596468 \u2013 ALSEN) (Paris:ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders, today announced that it has submitted a first Clinical Trial Application (CTA) for OTOF-GT to the UK\u2019s Medicines and Healthcare products ... 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