MONTPELLIER, France--(BUSINESS WIRE)--Regulatory News:

法国蒙彼利埃——（商业新闻短讯）——监管新闻：

Sensorion (FR0012596468 – 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 disorders, today announces that it has received approval for its Clinical Trial Application (CTA) to initiate a Phase 1/2 clinical trial of SENS-501 (OTOF-GT), in France.

Sension（FR0012596468–ALSEN）是一家开创性的临床阶段生物技术公司，专门开发用于恢复、治疗和预防听力障碍领域的新型疗法，今天宣布，它已获得临床试验申请（CTA）的批准，将在法国启动SENS-501（OTOF-GT）的1/2期临床试验。

The conclusion of the Part I of the assessment report according to regulation EU 536/2014 covering France, Italy and Germany is that the conduct of the clinical trial is acceptable..

根据涵盖法国、意大利和德国的欧盟536/2014法规，评估报告第一部分的结论是，临床试验的进行是可以接受的。。

The phase 1/2 clinical trial (Audiogene) aims to evaluate the safety, tolerability, and efficacy of intra-cochlear injection of SENS-501 for the treatment of OTOF gene-mediated hearing impairment in paediatric patients aged 6 to 31 months at the time of gene therapy treatment. Targeting the first years of life, the time period when the auditory system plasticity is optimal, will maximize the chances of these young children with pre-lingual hearing loss to acquire normal speech and language.

1/2期临床试验（Audiogene）旨在评估耳蜗内注射SENS-501治疗基因治疗时6至31个月的儿童OTOF基因介导的听力障碍的安全性，耐受性和有效性。针对生命的头几年，即听觉系统可塑性最佳的时期，将最大限度地提高这些语前听力损失幼儿获得正常言语和语言的机会。

Audiogene will also assess the clinical safety, performance, and usability of the administration device system under development in partnership with EVEON. The design of the study will consist of two cohorts of 2 doses followed by an expansion cohort at the selected dose. While the safety will be the primary endpoint for the dose escalation cohort , the auditory brainstem response (ABR) will be the primary efficacy endpoint of the dose expansion cohort.

Audiogene还将评估与EVEON合作开发的管理设备系统的临床安全性、性能和可用性。该研究的设计将包括两个2剂量的队列，然后是所选剂量的扩展队列。虽然安全性将是剂量递增队列的主要终点，但听觉脑干反应（ABR）将是剂量扩展队列的主要疗效终点。

The CTA approval follows extensive preclinical studies assessing the safety and efficacy of SENS-501 and successful manufacturing of the gene therapy Drug Product for the clinical trial. Sensorion will communicate about the first patient in the course of H2 2024..

CTA批准之前进行了广泛的临床前研究，评估了SENS-501的安全性和有效性，并成功制造了用于临床试验的基因治疗药物产品。Sension将在2024年下半年与第一位患者进行沟通。。

Nawal Ouzren, Sensorion's Chief Executive Officer, said: 'Securing approval to start the phase 1/2 clinical trial for SENS-501 marks a significant milestone for Sensorion and confirms our position of a leading company in the emerging field of gene therapies for hearing loss. Hearing loss caused by mutations of the gene encoding for otoferlin is a challenging disorder and there are no approved curative treatments for children with congenital deafness, so this is a significant unmet medical need.

Sension首席执行官纳瓦尔·乌兹伦（Nawal Ouzren）表示：“获得批准开始SENS-501的1/2期临床试验，标志着Sension的一个重要里程碑，并证实了我们在新兴的听力损失基因治疗领域的领先地位。由编码otoferlin的基因突变引起的听力损失是一种具有挑战性的疾病，目前尚无针对先天性耳聋儿童的批准治疗方法，因此这是一项尚未得到满足的重大医疗需求。

We look forward to continuing to work with healthcare providers, regulatory authorities, patient groups to address unmet and underserved medical needs in the hearing space.”.

我们期待着继续与医疗保健提供者、监管机构、患者团体合作，以解决听力领域未得到满足和服务不足的医疗需求。”。

Professor Natalie Loundon, M.D., Director of the Center for Research in Pediatric Audiology, Pediatric Otolaryngologist and Head and Neck Surgeon, Necker Enfants Malades, AP-HP, in Paris, France, Coordinating Investigator of the Audiogene clinical study, commented: “SENS-501 represents a hope for numerous children born with deafness linked to a defect in the otoferlin gene and for whom no curative treatment exists today.

Natalie Loundon教授，医学博士，儿科听力学研究中心主任，儿科耳鼻喉科医生和头颈外科医生，AP-HP Necker Enfants Malades，法国巴黎，Audiogene临床研究协调研究员，评论说：“SENS-501代表了许多与耳蜗蛋白基因缺陷有关的耳聋儿童的希望，而这些儿童目前尚无治疗方法。

The administration of a single-injection therapy to very young children requires state-of-the-art hospital technical platform and health care givers team familiar with the handling of gene therapies. In keeping with our philosophy of supporting innovation wherever children's medical needs go unmet, we are delighted to be fully involved from the very beginning in this pioneering clinical trial.”.

对非常年幼的儿童进行单次注射治疗需要最先进的医院技术平台和熟悉基因治疗处理的医疗保健人员团队。为了与我们在儿童医疗需求未得到满足的地方支持创新的理念保持一致，我们很高兴从一开始就充分参与这项开创性的临床试验。”。

Sensorion’s SENS-501 (OTOF-GT) dual vector AAV gene therapy development program aims to restore hearing in patients with mutations in the gene coding for otoferlin protein who suffer from severe to profound sensorineural prelingual non-syndromic hearing loss. Sensorion’s lead gene therapy program has been developed as part of its collaboration focused on the genetics of hearing with the Institut Pasteur, which has been initiated in 2019..

Sension的SENS-501（OTOF-GT）双载体AAV基因治疗开发计划旨在恢复患有严重至严重感音神经性舌前非综合征性听力损失的耳铁蛋白基因突变患者的听力。Sension的领先基因治疗计划是与巴斯德研究所（Institut Pasteur）合作开发的，该项目于2019年启动，专注于听力遗传学。。

The Genetics and Physiology of the Hearing Unit of the Institut Pasteur, led by Professor Christine Petit, MD, PhD, has developed world-class expertise over the last 25 years in the molecular physiology and physiopathology of the hearing system. Recent advances, conducted alongside Saaid Safieddine, PhD, have led to the development of the gene therapy product SENS-501..

巴斯德研究所听力科的遗传学和生理学由医学博士克里斯蒂娜·佩蒂特教授领导，在过去的25年中，在听力系统的分子生理学和病理生理学方面拥有世界一流的专业知识。与Saaid Safieddine博士一起进行的最新进展导致了基因治疗产品SENS-501的开发。。

Professor Christine Petit, Professor at the Institut Pasteur and Professor Emeritus at the Collège de France, France, Winner of the Kavli Prize in 2018, commented: “This regulatory green light for the inclusion of patients in France in one of the world's first gene therapy trials in the field of hearing represents a major achievement for the teams at the Institut de l'Audition (Institut Pasteur center), and a crucial milestone in our strategic collaboration with Sensorion.

巴斯德研究所（Institut Pasteur）教授、法国法兰西学院（Collège de France）名誉教授克里斯蒂娜·佩蒂（Christine Petit）教授，2018年卡夫利奖（Kavli Prize）获得者，评论说：“这项将法国患者纳入世界上第一个听力领域基因治疗试验之一的监管绿灯，代表了海选研究所（巴斯德研究所中心）团队的一项重大成就，也是我们与感官战略合作的一个重要里程碑。

The SENS-501 program, which aims to correct the deficiency of a gene responsible for congenital deafness in order to restore hearing, is based on very solid pioneering research elucidating the role of otoferlin and the pathogenic processes elicited by otoferlin defect. Its success will pave the way for other potential therapeutic innovations, based on gene therapy in many forms of deafness and for thousands of patients.”.

SENS-501计划旨在纠正导致先天性耳聋的基因缺陷，以恢复听力，该计划基于非常坚实的开创性研究，阐明了耳铁蛋白的作用以及耳铁蛋白缺陷引起的致病过程。它的成功将为其他潜在的治疗创新铺平道路，这些创新基于多种形式的耳聋和数千名患者的基因治疗。”。

Otoferlin is a protein expressed in the inner hair cells (IHC) present in the cochlea and is critical for hearing by ensuring the transmission of the acoustic signals to the auditory nerves. Otoferlin related hearing loss is responsible for up to 8% of all cases of congenital hearing loss, affecting around 20,000 people per year in the US and Europe1.

耳铁蛋白是一种在耳蜗内毛细胞（IHC）中表达的蛋白质，通过确保声音信号传递到听觉神经，对听力至关重要。与耳蜗素相关的听力损失占所有先天性听力损失病例的8%，在美国和欧洲每年影响约20000人1。

SENS-501 previously received Orphan Drug Designation from the US Food and Drug Administration (FDA)2 and the European Medicines Agency (EMA)3 and Rare Pediatric Disease Designation from the FDA in Q4 2022..

SENS-501之前曾于2022年第4季度获得美国食品和药物管理局（FDA）2和欧洲药品管理局（EMA）3的孤儿药指定和FDA的罕见儿科疾病指定。。

Sensorion presented preclinical data that indicated the potential for safe and efficient clinical translation of gene therapy for otoferlin delivered by a dual AAV vector. SENS-501 administration in a DFNB9 mouse model (OTOF-KO) showed long-term de novo expression of otoferlin in inner hair cells (IHCs) and ABR (auditory brainstem response) restoration.

Sension提供的临床前数据表明，通过双AAV载体递送的otoferlin基因疗法具有安全有效的临床翻译潜力。DFNB9小鼠模型（OTOF-KO）中的SENS-501给药显示耳铁蛋白在内毛细胞（IHC）和ABR（听觉脑干反应）恢复中长期从头表达。

Sensorion has also developed, in Non-Human Primates (NHPs), an optimal surgical procedure, similar to cochlear implantation, and an administration device system, in partnership with EVEON, which demonstrated an effective transduction rate of the targeted IHCs in NHPs. EVEON designs and manufactures custom medical devices for the preparation and delivery of drugs..

在非人灵长类动物（NHPs）中，Sension还与EVEON合作开发了一种类似于人工耳蜗植入的最佳手术程序和一种给药装置系统，该系统证明了NHPs中靶向IHC的有效转导率。EVEON设计和制造用于制备和输送药物的定制医疗设备。。

Denis Le Squer, Executive Director for the French non-profit foundation for hearing “Fondation Pour l’Audition”, added: “The launch of the Audiogene clinical trial is a major step forward for deaf children with otoferlin defects and their parents but also brings hope to people with genetic deafness.

法国非营利听力基金会“听力基金会”执行董事丹尼斯·勒·斯克尔补充说：“Audiogene临床试验的启动，对于耳蜗缺陷的聋儿及其父母来说是向前迈出的重要一步，但也为遗传性耳聋患者带来了希望。

We are very proud that our long-time support to French innovation, supported by Sensorion, and to the teams of Prof. Petit at Institut de l'Audition, center of the Institut Pasteur, and Prof. Loundon, at the Clinical Center for Research in Pediatric Audiology at AP-HP Necker hospital, translates now into a trial.”.

我们非常自豪的是，我们对法国创新的长期支持，得到了Sensorion的支持，以及对巴斯德研究所中心海选研究所Petit教授和AP-HP Necker医院儿科听力学临床研究中心Loundon教授的团队的支持，现在转化为一项试验。”。

This gene therapy for patients suffering from otoferlin deficiency has been developed in the framework of RHU AUDINNOVE, a consortium composed of Sensorion with the Necker Enfants Malades Hospital, the Institut Pasteur, and the Fondation pour l’Audition. The project is partially financed by the French National Research Agency, through the “investing for the future” program (ref: ANR-18-RHUS-0007)..

这种针对耳铁蛋白缺乏症患者的基因疗法是在RHU AUDINNOVE的框架下开发的，该联盟由内克儿童马拉德斯医院，巴斯德研究所和听觉基金会组成。该项目由法国国家研究机构通过“投资未来”计划（参考号：ANR-18-RHUS-0007）提供部分资金。。

About Sensorion

关于Sension

Sensorion 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.

Sension是一家开创性的临床阶段生物技术公司，专门开发新疗法，以恢复，治疗和预防听力损失障碍，这是一项重要的全球未满足的医疗需求。

Sensorion has built a unique R&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. 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.

Sension建立了一个独特的研发技术平台，以扩大其对内耳相关疾病的病理生理学和病因学的理解，使其能够为候选药物选择最佳的靶标和作用机制。它有两个基因治疗计划，旨在纠正遗传性单基因形式的耳聋，这是在其与巴斯德研究所（InstitutPasteur）的广泛战略合作框架内开发的，重点是听力遗传学。

SENS-501 (OTOF-GT) targets deafness caused by mutations of the gene encoding for otoferlin and GJB2-GT targets hearing loss related to mutations in GJB2 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..

SENS-501（OTOF-GT）靶向由编码otoferlin的基因突变引起的耳聋，GJB2-GT靶向与GJB2基因突变相关的听力损失，以潜在地解决成人和儿童的重要听力损失部分。该公司还致力于鉴定生物标志物，以改善对这些服务不足疾病的诊断。。

Sensorion’s portfolio also comprises clinical-stage small molecule programs for the treatment and prevention of hearing loss disorders. Sensorion’s 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.

Sension的投资组合还包括用于治疗和预防听力损失障碍的临床阶段小分子计划。Sension的临床阶段产品组合包括一个第二阶段产品：SENS-401（Arazasetron），正在计划的第二阶段SENS-401在顺铂诱导的耳毒性（CIO）中的概念验证临床研究中取得进展，并且与合作伙伴Cochlear Limited合作，在计划进行人工耳蜗植入的患者中进行SENS-401的研究。

A Phase 2 study of SENS-401 was also completed in Sudden Sensorineural Hearing Loss (SSNHL) in January 2022..

SENS-401的第二阶段研究也于2022年1月在突发性感音神经性听力损失（SSNHL）中完成。。

www.sensorion.com

www.sensorion.com

Label: SENSORION

标签：SENSORION

ISIN: FR0012596468

ISIN:FR0012596468

Mnemonic: ALSEN

助记符：ALSEN

Disclaimer

免责声明

This 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.

本新闻稿包含有关Sension及其业务的某些前瞻性声明。此类前瞻性陈述基于Sension认为合理的假设。然而，无法保证此类前瞻性报表会得到验证，这些报表会面临许多风险，包括2023年3月30日发布的2022年全年财务报告中规定的风险，这些风险可在我们的网站上查阅，也无法保证经济状况、金融市场和Sension运营所在市场的发展。

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.

本新闻稿中包含的前瞻性声明也会面临Sension尚不知道或Sension目前认为不重要的风险。所有或部分此类风险的发生可能导致实际结果、财务状况、业绩或感知成就与此类前瞻性报表存在重大差异。

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.

本新闻稿及其包含的信息不构成任何国家出售或认购Sension股份的要约，也不构成任何国家购买或认购Sension股份的要约。在某些国家传播本新闻稿可能违反当地法律法规。

Any recipient of this press release must inform oneself of any such local restrictions and comply therewith..

本新闻稿的任何接收者必须告知自己任何此类当地限制并遵守。。

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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..

一项关于非综合征性听力障碍和听神经病患者耳铁蛋白基因（OTOF）突变发生率和频谱的多中心研究。嗡嗡声突变。2008年6月；29（6）：823-31。doi:10.1002/humu.20708。PMID:18381613。。

2 FDA Orphan Drug Designations and Approvals https://www.accessdata.fda.gov/scripts/opdlisting/oopd/listResult.cfm

2 FDA孤儿药名称和批准https://www.accessdata.fda.gov/scripts/opdlisting/oopd/listResult.cfm

3 EU Community Register of orphan medicinal products https://ec.europa.eu/health/documents/community-register/html/o2698.htm

3欧盟孤儿药品共同体注册https://ec.europa.eu/health/documents/community-register/html/o2698.htm