Presbycusis

As presented at ARO, 10 of 11 children with at least one post-treatment assessment showed notable improvements in hearing Speech and development progress followed dramatic improvements in hearing in first child treated in the trial TARRYTOWN, N.Y. , Feb. 24, 2025 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced updated data for the investigational gene therapy DB-OTO from the Phase 1/2 CHORD trial in 12 children who have profound genetic hearing loss due to variants of the otoferlin (OTOF) gene. These include 72-week results showing speech and development progress in the first child dosed at 10 months of age, as well as initial results in 11 children (aged 10 months to 16 years old) – three of whom received DB-OTO bilaterally (in both ears). The latest results were presented in an oral presentation at the Association for Research in Otolaryngology’s (ARO) 48th Annual MidWinter Meeting. “Sound is a significant part of the human experience that connects us to each other and our environment,” said Jay T. Rubinstein , M.D., Ph.D., Virginia Merrill Bloedel Professor of Otolaryngology and Bioengineering and Director, Bloedel Hearing Research Center , University of Washington School of Medicine , and a CHORD clinical trial investigator. “A year after treatment in one ear with DB-OTO, a child born profoundly deaf was able to enjoy music, engage in imaginative play and participate in bedtime reading when the cochlear implant on their other ear was removed. These seemingly small interactions are life-changing for these children as well as their families and these results continue to underscore the revolutionary promise of DB-OTO as a potential treatment for otoferlin-related hearing loss.” In the trial, 12 participants have received DB-OTO to date – of whom nine were administered an intracochlear injection in one ear and three received it bilaterally. The surgical procedure to administer DB-OTO leverages an approach similar to cochlear implantation, which enables use in young infants. As presented at ARO, 48-week results from the first participant dosed in the trial showed improvement of hearing to near-normal levels across key speech frequencies. This included hearing thresholds that were within normal limits (0.25-2.0 kHz) in most speech-relevant frequencies and corroborated with positive auditory brainstem responses (ABRs). Particularly encouraging were results from formal speech perception tests in which the child demonstrated improvement from week 48 to week 72 and correctly identified words – such as mommy, cookies and airplane – that were presented at a conversational level without any visual cues. Among the 11 participants with at least one post-treatment assessment, 10 demonstrated a notable response, with improved hearing at various decibel hearing levels (dBHL). Additionally, among five participants with 24-week assessments, three experienced improvements in average hearing thresholds to “nearly normal” (n=1; ≤40 dBHL) or normal (n=2; ≤25 dBHL) hearing levels. All ABR responses were corroborated by hearing improvements assessed by pure tone audiometry (PTA). One participant has not experienced a change from their baseline hearing at 24 weeks post-dosing. Across all 12 participants, both the surgical procedure and DB-OTO were well tolerated, and there were no adverse events or serious adverse events considered related to DB-OTO. Five of 12 participants experienced transient post-surgical vestibular adverse events (e.g., nystagmus, nausea, dizziness, vomiting), which resolved within 6 days of dosing. DB-OTO received Orphan Drug, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy designations from the U.S. Food and Drug Administration and Orphan Drug Designation was granted by the European Medicines Agency . The potential use of DB-OTO for otoferlin-related hearing loss is currently under clinical investigation, and its safety and efficacy have not been evaluated by any regulatory authority. About Otoferlin-related Hearing LossCongenital deafness (hearing loss present at birth) is a significant unmet medical need that affects approximately 1.7 out of every 1,000 children born in the U.S. and approximately half of these cases have genetic causes. However, otoferlin-related hearing loss is ultra-rare. This specific condition is caused by variants in the OTOF gene, which leads to a lack of a functional otoferlin protein that is critical for the communication between the sensory cells of the inner ear and the auditory nerve. About the CHORD TrialThe CHORD trial is an ongoing, Phase 1/2 first-in-human, multicenter, open-label trial to evaluate the safety, tolerability and preliminary efficacy of DB-OTO in infants, children and adolescents with otoferlin variants. Currently enrolling children across sites in the U.S. , United Kingdom and Spain (<18 years of age), CHORD is being conducted in two parts. In the initial dose-escalation cohort (Part A), participants receive a single intracochlear injection of DB-OTO in one ear, and in the expansion cohort (Part B), participants receive simultaneous single intracochlear injections of DB-OTO in both ears at the selected dose from Part A. Hearing improvements were assessed by PTA and ABR. PTA is considered by auditory experts to be the gold standard measurement of hearing and is measured through behavioral responses to sound (e.g., turning head towards sound) that is emitted at different intensity levels and measured in decibels (dB). ABR corroborates these behavioral responses, serving as an objective confirmation of hearing function, and is measured through electrical brainstem responses to sound emitted at different dBs. At baseline, all participants had no behavioral (PTA) or electrophysiological (ABR) responses at maximum sound levels (≥100 dB). Additional information about the trial, including enrollment, can be obtained by contacting clinicaltrials@regeneron.com. About DB-OTO and the Regeneron Auditory ProgramDB-OTO is an investigational cell-selective, dual adeno-associated virus (AAV) vector gene therapy designed to provide durable, physiological hearing to individuals with profound, congenital hearing loss caused by variants of the OTOF gene. The treatment aims to deliver a working copy to replace the faulty OTOF gene using a modified, non-pathogenic virus that is delivered via an injection into the cochlea under general anesthesia (similar to the procedure used for cochlear implantation). In this gene therapy, the newly introduced OTOF gene is under the control of a proprietary cell-specific Myo15 promoter, which is intended to restrict expression only to inner hair cells that normally express otoferlin. In addition to OTOF, Regeneron is committed to investigating several other targets for genetic forms of hearing loss, including GJB2. About Regeneron Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous approved treatments and product candidates in development, most of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, neurological diseases, hematologic conditions, infectious diseases, and rare diseases. Regeneron pushes the boundaries of scientific discovery and accelerates drug development using our proprietary technologies, such as VelociSuite®, which produces optimized fully human antibodies and new classes of bispecific antibodies. We are shaping the next frontier of medicine with data-powered insights from the Regeneron Genetics Center® and pioneering genetic medicine platforms, enabling us to identify innovative targets and complementary approaches to potentially treat or cure diseases. For more information, please visit www.Regeneron.com or follow Regeneron on LinkedIn, Instagram, Facebook or X. Forward-Looking Statements and Use of Digital MediaThis press release includes forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Regeneron Pharmaceuticals, Inc. (“Regeneron” or the “Company”), and actual events or results may differ materially from these forward-looking statements. Words such as “anticipate,” “expect,” “intend,” “plan,” “believe,” “seek,” “estimate,” variations of such words, and similar expressions are intended to identify such forward-looking statements, although not all forward-looking statements contain these identifying words. These statements concern, and these risks and uncertainties include, among others, the nature, timing, and possible success and therapeutic applications of products marketed or otherwise commercialized by Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Products”) and product candidates being developed by Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Product Candidates”) and research and clinical programs now underway or planned, including without limitation the investigational gene therapy DB-OTO; the likelihood, timing, and scope of possible regulatory approval and commercial launch of Regeneron’s Product Candidates and new indications for Regeneron’s Products, such as DB-OTO for the treatment of otoferlin-related hearing loss; uncertainty of the utilization, market acceptance, and commercial success of Regeneron’s Products and Regeneron’s Product Candidates and the impact of studies (whether conducted by Regeneron or others and whether mandated or voluntary), including the studies discussed or referenced in this press release, on any of the foregoing or any potential regulatory approval of Regeneron’s Products and Regeneron’s Product Candidates (such as DB-OTO); the extent to which the results from the research and development programs conducted by Regeneron and/or its collaborators or licensees (including the studies discussed or referenced in this press release) may be replicated in other studies and/or lead to advancement of product candidates to clinical trials, therapeutic applications, or regulatory approval; the ability of Regeneron’s collaborators, licensees, suppliers, or other third parties (as applicable) to perform manufacturing, filling, finishing, packaging, labeling, distribution, and other steps related to Regeneron’s Products and Regeneron’s Product Candidates; the ability of Regeneron to manage supply chains for multiple products and product candidates; safety issues resulting from the administration of Regeneron’s Products and Regeneron’s Product Candidates (such as DB-OTO) in patients, including serious complications or side effects in connection with the use of Regeneron’s Products and Regeneron’s Product Candidates in clinical trials; determinations by regulatory and administrative governmental authorities which may delay or restrict Regeneron’s ability to continue to develop or commercialize Regeneron’s Products and Regeneron’s Product Candidates; ongoing regulatory obligations and oversight impacting Regeneron’s Products, research and clinical programs, and business, including those relating to patient privacy; the availability and extent of reimbursement of Regeneron’s Products from third-party payers, including private payer healthcare and insurance programs, health maintenance organizations, pharmacy benefit management companies, and government programs such as Medicare and Medicaid; coverage and reimbursement determinations by such payers and new policies and procedures adopted by such payers; changes in laws, regulations, and policies affecting the healthcare industry; competing drugs and product candidates that may be superior to, or more cost effective than, Regeneron’s Products and Regeneron’s Product Candidates (including biosimilar versions of Regeneron’s Products); unanticipated expenses; the costs of developing, producing, and selling products; the ability of Regeneron to meet any of its financial projections or guidance and changes to the assumptions underlying those projections or guidance; the potential for any license, collaboration, or supply agreement, including Regeneron’s agreements with Sanofi and Bayer (or their respective affiliated companies, as applicable), to be cancelled or terminated; the impact of public health outbreaks, epidemics, or pandemics on Regeneron 's business; and risks associated with litigation and other proceedings and government investigations relating to the Company and/or its operations (including the pending civil proceedings initiated or joined by the U.S. Department of Justice and the U.S. Attorney's Office for the District of Massachusetts ), risks associated with intellectual property of other parties and pending or future litigation relating thereto (including without limitation the patent litigation and other related proceedings relating to EYLEA® (aflibercept) Injection), the ultimate outcome of any such proceedings and investigations, and the impact any of the foregoing may have on Regeneron’s business, prospects, operating results, and financial condition. A more complete description of these and other material risks can be found in Regeneron’s filings with the U.S. Securities and Exchange Commission , including its Form 10-K for the year ended December 31, 2024 . Any forward-looking statements are made based on management’s current beliefs and judgment, and the reader is cautioned not to rely on any forward-looking statements made by Regeneron . Regeneron does not undertake any obligation to update (publicly or otherwise) any forward-looking statement, including without limitation any financial projection or guidance, whether as a result of new information, future events, or otherwise. Regeneron uses its media and investor relations website and social media outlets to publish important information about the Company, including information that may be deemed material to investors. Financial and other information about Regeneron is routinely posted and is accessible on Regeneron 's media and investor relations website (https://investor.regeneron.com) and its LinkedIn page (https://www.linkedin.com/company/regeneron-pharmaceuticals). Source: Regeneron Pharmaceuticals, Inc.

MONTPELLIER, France--( BUSINESS WIRE )--Regulatory News: 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 loss disorders, today reports its half-year results as of June 30, 2024, as well as its outlook for the remainder of 2024. Nawal Ouzren, Chief Executive Officer of Sensorion, said : “ Over the first half of 2024, Sensorion has made steady positive progress, reaching several key milestones in its development plan. Most notably, with SENS-501, our most advanced gene therapy program, we have made significant regulatory and clinical advances. After receiving a green light from European regulators in January 2024, I am thrilled to announce that the first patient has been recruited to the study and injected. We look forward to updating you on the progress with this program in the coming months. We have also continued to advance our Phase 2a clinical programs for SENS-401. Firstly, we unveiled new positive efficacy data for SENS-401 in the prevention of residual hearing loss after cochlear implantation, with full data to be presented at the World Congress of Audiology, later this month. Secondly, we have continued to recruit adult patients to the NOTOXIS study in Cisplatin-Induced Ototoxicity. On the financial front, the steady execution of our clinical development roadmap has attracted new investment into Sensorion, with new recognised specialist international healthcare investors taking part in the two private placements completed last February and April, for a total amount of over €65 million. With these additional resources, we are well-funded to the end of 2025. Overall, I am delighted with the advances we are making, and I look forward to sharing future updates over the course of 2024, which will be rich in terms of clinical data on our lead programs .” Pipeline Highlights and Upcoming Milestones During the first half of 2024, Sensorion continued to develop innovative therapies to restore hearing, treat and prevent hearing loss, to potentially transform the patients’ quality of hearing. Gene Therapies for Hereditary Monogenic Hearing Loss Sensorion continues to advance its gene therapy programs, developed as part of its collaboration with the Institut Pasteur, initiated in 2019 and extended for an additional 5-year period in January 2024. The framework agreement for a research partnership grants Sensorion an option to obtain an exclusive license to develop and commercialize gene therapy drug candidates for the restoration of hearing. The company has notably made progress in its lead gene therapy program, SENS-501, with a first patient recruited and injected. SENS-501 (OTOF-GT): several milestones achieved with Clinical Trial Application (CTA) approval in Europe and First Patient Included (FPI) and injected. FPI initial safety data communication planned at the WCA on September 20, 2024 Sensorion’s SENS-501 (OTOF-GT) dual AAV vector gene therapy development candidate aims at restoring hearing in patients with mutations in OTOF gene who suffer from severe to profound sensorineural prelingual non syndromic hearing loss. The 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 1 . Sensorion’s lead gene therapy program, SENS-501, has been developed as part of its collaboration focused on the genetics of hearing with the Institut Pasteur which was initiated in 2019 and extended for an additional 5-year period in January 2024. On January 19, 2024, Sensorion announced the approval to initiate the Phase 1/2 clinical trial of SENS-501, Audiogene, in Europe with France as first country. 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. 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. The design of the study consists of two cohorts of two 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 will also assess the clinical safety, performance, and usability of the administration device system developed by Sensorion. On September 11, 2024, Sensorion received the European Medicines Agency decision agreeing on a Pediatric Investigation Plan (PIP) for SENS-501. A PIP is a development plan aimed at ensuring that the necessary data are obtained to support the marketing authorization of a medicine in the pediatric population in the European Union. All applications for marketing authorization for new medicines require the results of studies as described in an agreed PIP. Sensorion will communicate initial safety data about the first patient at the WCA, on September 20, 2024, and is on track for the recruitment of the first cohort by end of 2024. The recruitment completion of the two first cohorts of patients is expected in H1 2025. OTOCONEX, the Company’s Natural History Study to document the natural course of disease progression in otoferlin deficiency patients and in children with hearing loss related to GJB2 mutations, is running across Europe and plays an important role in identifying patients as early as possible. GJB2-GT: progressing CTA/IND-enabling studies towards Clinical Trial Applications Submission in H2 2025 Sensorion's GJB2 gene therapy program, developed in collaboration with the Institut Pasteur, has the potential to address three forms of hearing loss related to GJB2 mutations: early onset of presbycusis in adults, progressive forms of hearing loss in children, and pediatric congenital deafness. Although the types of GJB2 mutations in children and adults may differ, gene therapy offers potential solutions for both populations. On April 6, 2023, Sensorion announced the candidate selection for GJB2-GT during its R&D Day focusing on gene therapy. The candidate is designed with a specific adeno-associated virus (AAV) capsid and includes specific regulatory sequences to safely target key cells in the ear that normally express GJB2 and avoids ototoxicity. Sensorion has developed the non-GMP process development of the GJB2 candidate up to 50L scale. Process and analytical methods are under transfer to the CDMO for the GMP production of the clinical batch. Sensorion is advancing the candidate into CTA/IND-enabling activities for anticipated submission in H2 2025. The Company plans on providing updates on preclinical activities related to GJB2-GT including additional Proof-of-Concept data at the European Society of Cell & Gene Therapy (ESGCT), taking place on October 22-25, 2025, Rome, Italy. SENS-401 Sensorion is developing SENS-401 (Arazasetron) in three indications and two Proof of Concept Phase 2a clinical trials are ongoing, including a study designed to assess SENS-401 for residual hearing preservation during cochlear implantation in partnership with Cochlear Limited; the final results of which will be reported on September 20, 2024, during the World Congress of Audiology. The second study is a proof-of-concept trial in Cisplatin-Induced Ototoxicity (CIO). SENS-401 is an orally available small molecule that aims to protect and preserve inner ear tissue from damage responsible of hearing impairment. SENS-401 has been granted Orphan Drug Designation by the EMA in Europe for the treatment of sudden sensorineural hearing loss (SSNHL), and by the FDA in the U.S. for the prevention of platinum-induced ototoxicity in pediatric population. Milestones achieved for SENS-401 in the prevention of residual hearing loss after cochlear implantation with primary endpoint achieved and positive secondary efficacy endpoints data. Full final data readout to be presented at the WCA, on September 20, 2024 Sensorion has advanced its small molecule SENS-401 in a multicentric, randomized, controlled open-label Phase 2a trial aimed at evaluating the presence of SENS-401 in the cochlea (perilymph) after 7 days of twice-daily oral administration in adult patients prior to cochlear implantation due to moderately severe to profound hearing impairment. Patients start treatment with SENS-401 7 days before implantation and continue to receive SENS-401 for a further 42 days. On February 1, 2024, Sensorion announced the completion of patient inclusion in the Phase 2a POC clinical trial. Following this announcement, Sensorion communicated in March 2024, that a total of 25 patients have been implanted with a cochlear implant: 16 in the treated arm and 9 in the control non-treated arm. The presence of SENS-401 in the perilymph at a level compatible with potential therapeutic efficacy has been confirmed in 100% of the patients sampled, 7 days after the start of the treatment, confirming that the primary endpoint was met. On July 13, 2024, Professor Stephen O'Leary, M.D., Ph.D., the study principal investigator, presented new medical data and analysis of results from the SENS-401 Phase 2a clinical trial at the 17th International Conference on Cochlear Implants and Other Implantable Technologies (CI2024) in Vancouver, Canada. The study design included a number of secondary endpoints, notably the change of hearing threshold from baseline to the end of the treatment period in the implanted ear at several frequencies. Study entry criteria required patients to have a pure tone audiometry (PTA) threshold of 80 dB or better (i.e., ≤80 dB) at 500 Hz, defined as indicating a minimal level of residual hearing. The descriptive results show that the administration of SENS-401 reduced hearing loss following cochlear implantation. Six weeks post cochlear implantation (corresponding to end of SENS-401 treatment), the data indicate that the mean hearing loss induced by the surgery at 500 Hz is 19 dB for patients treated with SENS-401 (N=16) compared to 32 dB for control group of SENS-401 untreated patients (N=8). Similar clinically meaningful difference is observed for the mean of the three following frequencies (250, 500 and 750 Hz) with 16 dB in the SENS-401 treated group compared to 31 dB in the control group. These good descriptive results remained clinically meaningful over time and up to the last study visit fourteen weeks after cochlear implantation and confirm the key role of SENS-401 in preserving residual hearing. The Company plans on communicating the results of the final analysis at the WCA, held in Paris, France, on September 19-22, 2024. SENS-401 in Cisplatin-Induced Ototoxicity progresses with patient recruitment progressing at a steady pace and positive Data Safety Monitoring Board (DSMB) recommendation to pursue the study Cisplatin and other platinum compounds are essential chemotherapeutic agents for many malignancies. Unfortunately, platinum-based therapies cause ototoxicity, or hearing loss, which is permanent, irreversible and particularly harmful to 50-60% of adult patients and 90% of pediatric patients who survive cancer. The NOTOXIS Proof-of-Concept (POC) Phase 2a trial is a multicenter, randomized, controlled, open-label study designed to assess the efficacy of SENS-401 in preventing cisplatin-induced ototoxicity in adult patients with neoplastic disease, four weeks after completion of cisplatin-based chemotherapy. The trial assesses several endpoints, including the rate and severity of ototoxicity, changes in pure tone audiometry (PTA) (dB) throughout the study compared to before cisplatin treatment, and tolerability. Eligible participants are randomized on Day 1 to either Arm A or Arm B in ratio 1:1. In Arm A, patients receive 43.5mg of oral SENS-401 one week before the start of the chemotherapy, continues throughout the entire chemotherapy duration, and extends for up to four weeks post-chemotherapy. This study is conducted in comparison to a control group of patients receiving chemotherapy alone, Arm B. The patients entering the study are to receive high doses of cisplatin, exceeding 70mg/m2 per treatment cycle and totaling at least 210 mg/m2 over the course of their chemotherapy regimen. On December 18, 2023, Sensorion announced that over one-third of the required study population had been recruited and that preliminary safety data for patients exposed to a daily dose of 43.5 mg SENS-401 administered b.i.d. for up to 11 weeks indicated a favorable profile. On July 23, 2024, Sensorion announced that the independent Data Safety Monitoring Board (DSMB) had undertaken a review of the safety data for the patients participating in the NOTOXIS POC Phase 2a clinical trial. The DSMB recommended the continuation of the study and confirmed the absence of any concern as to the safety of SENS-401 when administered in adult patients receiving a daily dose of 43.5 mg, administered twice daily, over a period of up to 23 weeks. The patient enrolment continues to progress at a steady pace, in 13 clinical centers open to date. Sensorion will report preliminary safety and efficacy data of the Phase 2a POC clinical trial of SENS-401 CIO at the WCA, to be held on September 19-22, 2024, in Paris, France. Expected future milestones and estimated timelines : September 2024 – SENS-401 in combination with cochlear implantation: Final data readout September 2024 – SENS-401 in Cisplatin-Induced Ototoxicity: Preliminary safety and efficacy data September 2024 – SENS-501: FPI initial safety data October 2024 (ESGCT) – GJB2 -GT: Update on additional Proof-of-Concept efficacy and safety data H2 2024 – SENS-501: Enrollment completion of the first cohort in Audiogene H1 2025 – SENS-501: Enrollment completion of the second cohort in Audiogene H1 2025 – SENS-401 in Cisplatin-Induced Ototoxicity: Patient recruitment completion H2 2025 – GJB2 -GT: Clinical Trial Applications filing First-half 2024 financial highlights Cash Position Cash & Cash Equivalents amounted to 87.3 million as of June 30, 2024, compared to €37.0 million as of December 31, 2023. Research And Development (R&D) Expenses R&D expenses increased by 19% from €12.3 million as of June 30, 2023, to €14.7m as of June 30, 2024, primarily due to an increase in gene therapy programs with the start of GJB2-GT manufacturing. General And Administrative (G&A) Expenses G&A expenses were €3.8 million for half-year 2024, compared to €2.6 million for the same period in 2023. Net Loss Net loss was -€13,9 million for half-year 2024, compared to €12.3 million for half-year 2023. Financial guidance Based on its forecasted expenses and a cash position of €87.3 million as of June 30, 2024, the Company expects to be able to fund its operations until end of 2025. Financial structure The half-year accounts on June 30, 2024, drawn up according to IFRS standards, were approved by the Board of Directors on September 17, 2024. The simplified income statement as of June 30, 2024, is as follows: In thousands of Euros – IFRS standards 30.06.2024 30.06.2023 Operating income 3,332 2,647 Research & Development expenses 14,660 12,271 General & Administrative expenses 3,791 2,572 Total operating expenses 18,451 14,844 Operating loss -15,119 -12,196 Financial result 1,323 130 Pre-tax current income -13,796 -12,066 Corporate Income Tax -98 -222 Net loss -13,895 -12,288 The simplified balance sheet as of June 30, 2024, is as follows: In thousands of Euros – IFRS standards 30.06.2024 31.12.2023 Non-current Assets 3,536 3,236 Other Current Assets 10,649 6,293 Cash & cash equivalent 87,344 36,974 Total Assets 101,529 46,503 Equity 83,462 33,276 Non-current Liabilities 3,897 2,950 Current Liabilities 14,110 10,278 Total Liabilities 101,529 46,503 First-Half 2024 certified accounts On September 17, 2024, the Board of Directors approved the Company’s first half-year results as of June 30, 2024. The Company’s statutory auditors have completed their limited review and are in the process of issuing an unqualified report, which shall be available in the coming days. About Sensorion 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. 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. SENS-501 (OTOF-GT) currently being developed in a Phase 1/2 clinical trial, 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. 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. A Phase 2 study of SENS-401 was also completed in Sudden Sensorineural Hearing Loss (SSNHL) in January 2022. www.sensorion.com Label: SENSORION ISIN: FR0012596468 Mnemonic: ALSEN Disclaimer 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 2023 full year report published on March 14, 2024, 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. ____________________ 1 Rodríguez-Ballesteros M, et al., 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.

MONDAY, July 1, 2024 (HealthDay news) -- A 14-year study of testicular cancer survivors suggests that a chemotherapy drug could greatly raise patients' long-term odds for hearing loss . The drug in question, cisplatin , has been a mainstay of cancer chemotherapy for decades. It's often used to fight a range of cancers, including including bladder, lung, neck and testicular tumors. Knowing that cisplatin might impact people's hearing could help guide treatment, said study co-author Dr. Robert Frisina . He's chair of the department of medical engineering at the University of South Florida (USF). “This research gives oncologists the information they need to explore alternative treatment plans that could reduce the long-term side effects," he said in a USF news release. Such changes might include "altering the dosages and timing of the cisplatin in the treatment, when that could be an appropriate option,” Frisina explained. The findings were published recently in the journal JAMA Oncology . The study was led by Dr. Victoria Sanchez , an associate professor of otolaryngology head & neck surgery at USF. Over an average of 14 years, her team tracked the health of 100 men who'd been treated for and survived testicular cancers. The researchers found that most (78%) of the men who received cisplatin-based chemotherapy did have some trouble with their hearing by the end of the follow-up period. Their hearing loss causing "significant difficulties in everyday listening situations, negatively impacting their quality of life," according to the USF news release. The extent of the hearing loss rose along with the dose of cisplatin. That was especially true for folks with other risk factors, such as high blood pressure and heart disease. How might cisplatin affect hearing? According to Sanchez' team, the drug is administered intravenously and permeates much of the body. However, the ears may have particular difficulty in filtering out cisplatin, allowing it to gather there. Once in place within the ear, cisplatin may trigger inflammation and the elimination of sensory cells the ears need to code sound, the USF team explained. The result could be hearing loss that lasts long after chemotherapy ends. Sanchez said this all supports the notion that cancer patients get routine hearing tests. “Most patients still do not get their hearing tested prior to, during or after chemotherapy," she noted. "Our study highlights the need for regular auditory evaluations to manage and mitigate long-term hearing damage.” More follow-up of the men in the study is planned. "It will be critically important to follow these patients for life. Their current median age is only 48 years, and eventually they will enter the years at which age-related hearing loss also begins to develop,” said study senior author Dr. Lois Travis . She's professor of cancer research at Indiana University School of Medicine. Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.