Hearing Loss, Sensorineural

Event Scheduled for June 26 and 27, 2024 CARLSBAD, Calif.--(BUSINESS WIRE)-- Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, and the Christopher & Dana Reeve Foundation, a non-profit organization dedicated to advancing innovative research and improving quality of life for individuals impacted by paralysis, are proud to again collaborate to present the 2nd Annual Spinal Cord Injury Investor Symposium ("2nd SCIIS"). The 2nd SCIIS builds upon a successful inaugural event held on June 29, 2023, and will connect a diverse set of stakeholders in the field of spinal cord injury (SCI). This year’s event aims to accelerate development in SCI research and treatments by bringing together companies working in the development of treatments for SCI, with regulators, key opinion leaders, persons with lived experience, patient and community advocacy organizations and the investment community, in order to discuss perspectives on current and future treatment alternatives, impact and support SCI disease awareness and clinical trial participation through the implementation of patient appropriate clinical endpoints, and importantly, broaden awareness of and investment capital into SCI. The 2nd SCIIS will be held on June 26 and 27, 2024 at the Sanford Consortium for Regenerative Medicine in La Jolla, CA. This press release features multimedia. View the full release here: "We are excited to continue our collaboration with the Reeve Foundation on this event, which provides an excellent opportunity to highlight the important work being done by for-profit and non-profit organizations alike and to broaden awareness and impact for those affected by this debilitating condition," stated Brian M. Culley, Lineage CEO. "Our aim at Lineage is to improve recovery and mobility by replacing the cells which are destroyed following a spinal cord injury. We are encouraged by the initial clinical testing which has been performed with our approach and look forward to continuing our work. In certain rare disease settings, we have seen that raising investor awareness can lead to greater investment, which can help to accelerate new treatments. This year’s symposium aims to build on the success of last year’s event and to continue to foster open and collaborative dialogue among leading experts, companies, researchers, persons with lived experience, caregivers, advocacy organizations, regulators and members of the investment community." “Real breakthroughs in SCI science require diverse stakeholders to start talking, sharing data and collaborating,” said Marco Baptista, Ph.D, Chief Scientific Officer at the Reeve Foundation. “We are focused on propelling the field forward and incentivizing high-risk, high-reward projects that can be translated from the lab into the clinic and make a real impact on quality of life. Success necessitates more collaboration among industry and academia, informed by those with lived experience. We’re excited to again bring together all those who are impacted by SCI and paralysis to explore and learn from promising science in progress.” The 2nd Annual Spinal Cord Injury Investor Symposium will be held in-person, on June 26 and 27, 2024, in the Duane Roth Auditorium at the Sanford Consortium for Regenerative Medicine in La Jolla, CA. This event will bring together both public and private companies developing new treatment options for people with spinal cord injury, alongside leaders in the spinal cord injury medical and advocacy communities. A number of important topics will be discussed, including but not limited to preclinical and clinical SCI treatment approaches and the drug development process; racial, ethnic, socioeconomic, geographic vulnerabilities within SCI care and outcomes; hearing the person's with lived experience voice and ensuring patients' perspectives, needs and priorities are captured in the drug development process; and discussions of SCI clinical endpoints and the need for new and more precise assessment tools. About Spinal Cord Injuries A spinal cord injury occurs when the spinal cord is subjected to a severe crush or contusion and frequently results in severe functional impairment, including limb paralysis, aberrant pain signaling, and loss of bladder control and other body functions. There are approximately 18,000 new spinal cord injuries annually in the U.S. The cost of a lifetime of care for a severe spinal cord injury can be as high as $5 million. About the Sanford Consortium for Regenerative Medicine The Sanford Consortium is a multi-disciplinary and collaborative community of scientists who share the goal to harness the potential of stem cells for the treatment and cure of human diseases. The Sanford Consortium marshals the intellectual resources of five world leaders in life sciences research: La Jolla Institute for Allergy & Immunology, the Salk Institute for Biological Studies, the Sanford Burnham Prebys Medical Discovery Institute, Scripps Research, and the University of California, San Diego. Each of the Collaborating Organizations has substantive complementary research strengths that combined create a synergistic research enterprise, the whole of which is substantially greater than the sum of its parts. The Collaboratory has served as a focal point for stem cell science and has catalyzed increased collaboration among the stem cell research community. For more information, please visit www. . About the Christopher & Dana Reeve Foundation The Christopher & Dana Reeve Foundation is dedicated to curing spinal cord injury by funding innovative research and improving the quality of life for individuals and families impacted by paralysis. Additionally, through a cooperative agreement with the Administration for Community Living, the Reeve Foundation’s National Paralysis Resource Center (NPRC) promotes the health, well-being, and independence of people living with paralysis, providing comprehensive information, resources, and referral services assisting over 125,000 individuals and families since its launch in 2002. The Reeve Foundation is committed to elevating our community’s voices and needs to achieve greater representation and independence. We meet all 20 of the Better Business Bureau’s standards for charity accountability and hold the BBB’s Charity Seal. For more information, please visit ChristopherReeve.org or call 800-225-0292. About Lineage Cell Therapeutics, Inc. Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel or "off-the-shelf," cell therapies to address unmet medical needs. Lineage’s programs are based on its proprietary cell-based technology platform and associated development and manufacturing capabilities. From this platform, Lineage designs, develops, manufactures, and tests specialized human cells with anatomical and physiological functions similar or identical to cells found naturally in the human body. These cells are created by applying directed differentiation protocols to established, well-characterized, and self-renewing pluripotent cell lines. These protocols generate cells with characteristics associated with specific and desired developmental lineages. Cells derived from such lineages are transplanted into patients in an effort to replace or support cells that are absent or dysfunctional due to degenerative disease, aging, or traumatic injury, and to restore or augment the patient's functional activity. Lineage’s neuroscience focused pipeline currently includes: (i) OpRegen, a retinal pigment epithelial cell therapy in Phase 2a development under a worldwide collaboration with Roche and Genentech, a member of the Roche Group, for the treatment of geographic atrophy secondary to age-related macular degeneration; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of spinal cord injuries; (iii) ANP1, an auditory neuronal progenitor cell therapy for the potential treatment of auditory neuropathy; (iv) PNC1, a photoreceptor neural cell therapy for the potential treatment of vision loss due to photoreceptor dysfunction or damage; and (v) RND1, a novel hypoimmune induced pluripotent stem cell line being developed in collaboration with Eterna Therapeutics Inc. For more information, please visit or follow the company on X/Twitter @LineageCell. Forward-Looking Statements Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as "believe," “aim,” “may,” “will,” “estimate,” “continue,” “anticipate,” “design,” “intend,” “expect,” “could,” “can,” “plan,” “potential,” “predict,” “seek,” “should,” “would,” “contemplate,” “project,” “target,” “tend to,” or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to: the potential effect of the 2nd SCIIS, including accelerating development in SCI, broadening SCI disease awareness, or increasing clinical trial participation; and the ability of cell transplant therapy approaches, including OPC1, to improve recovery or allow a patient to regain more mobility than what could otherwise be expected. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineage’s actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including, but not limited to, the following risks: that positive findings in early clinical and/or nonclinical studies of a product candidate may not be predictive of success in subsequent clinical and/or nonclinical studies of that candidate; that engagement with patients and the advocacy community may not lead to improvements in patient outcomes; and that raising investor awareness may not lead to greater investment, nor accelerate new treatments; and those risks and uncertainties inherent in Lineage’s business and other risks discussed in Lineage’s filings with the Securities and Exchange Commission (SEC). Lineage’s forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading “Risk Factors” in Lineage’s periodic reports with the SEC, including Lineage’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the SEC and its other reports, which are available from the SEC’s website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

Milestone achieved for SENS-501 gene therapy program with Clinical Trial Application approval in Europe (France as first country) Communication on first patient expected in H2 2024 GJB2 gene therapy program advancing towards Clinical Trial Applications filing expected in Q2 2025 Primary endpoint met in Phase 2a clinical trial of SENS-401 in the preservation of residual hearing following cochlear implantation Presence of SENS-401 detected in 100% of patients sampled at levels compatible with potential therapeutic efficacy Final data readout expected in Q3 2024 Continued progress for SENS-401 in Phase 2a clinical trial in the prevention of Cisplatin-Induced Ototoxicity Preliminary safety and efficacy data readout expected at the World Congress of Audiology in September 2024 Completion of two financing rounds, raising €35 million in August 2023 and €50.5 million in February 2024, enabling the company to extend its cash runway through the end of Q2 2025 MONTPELLIER, France--(BUSINESS WIRE)-- Regulatory News: Sensorion (Paris:ALSEN) (FR0012596468 – ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent hearing loss disorders, today reports full-year 2023 financial results and business update. Nawal Ouzren, Chief Executive Officer of Sensorion, said: “Over the past months, Sensorion has successfully achieved all the major milestones on its roadmap. Our pioneering gene therapy, SENS-501, is entering clinical development, and our small molecule, SENS-401, has met primary endpoint in its most advanced indication: residual hearing preservation after cochlear implantation, in a Phase 2a study. At the same time, we have succeeded in securing financing for our operations until mid-2025 with the support of international and long-term investors. Sensorion is well equipped to meet its operational objectives for 2024, to continue bringing innovative therapeutic solutions to patients in the field of hearing, and to create value for its shareholders.” Pipeline Highlights and Upcoming Milestones In 2023, Sensorion continued to develop innovative therapies to restore hearing, to treat and prevent hearing loss, and potentially transform quality of life for patients suffering from hearing loss disorders. 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 exclusive option to obtain an exclusive license to develop and commercialize drug candidates in gene therapy for the restoration of hearing. The company has made significant progress in its gene therapy pipeline with the submission and approval of clinical trial application for SENS-501 in Europe, and the candidate selection for GJB2-GT. The company has expanded its technical development capabilities over the period along with pilot-scale non-GMP (Good Manufacturing Practices) manufacturing capacity. SENS-501: Milestone achieved with Audiogene Clinical Trial Application approval in Europe. First patient communication anticipated in H2 2024 Sensorion’s SENS-501 (OTOF-GT) dual AAV vector gene therapy development product 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 Europe1. 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. In the first half of 2023, Sensorion achieved a major development milestone by completing the preclinical package, and successfully producing the GMP dual AAV SENS-501 clinical batches at 200L scale. The production will enable the Company to cover the needs for the envisioned Phase 1/2 gene therapy clinical trial. Following the positive feedback from Scientific Advice meetings with regulatory agencies EMA (European Medicines Agency), ANSM (French National Agency for Medicines and Health Products Safety) and the UK’s MHRA (Medicines and Healthcare product Regulatory Agency), Sensorion has submitted a Clinical Trial Application (CTA) to initiate a Phase 1/2 clinical trial of SENS-501 to the UK’s MHRA on July 10, 2023, and in Europe on July 19, 2023. In parallel, Sensorion submitted a Medical Device Application for the injection system. On January 19, 2024, Sensorion announced the approval to initiate the Phase 1/2 clinical trial of SENS-501, Audiogene, in Europe (France as first country). The CTA approval follows extensive preclinical studies assessing the safety and efficacy of SENS-501 and successful GMP 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. 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 at two different doses followed by an expansion cohort at the selected dose. While the safety will be the primary endpoint for the dose escalation cohorts, the auditory brainstem response (ABR) will be the primary efficacy endpoint of the dose expansion cohort. Sensorion will communicate on the first patient in the course of H2 2024 and expects the recruitment completion of the first two cohorts of patients in H1 2025. GJB2-GT: Moving forward towards Clinical Trial Applications submission in Q2 2025 Sensorion's GJB2 gene therapy program, initiated in 2021 and developed in collaboration with the Institut Pasteur, has the potential to address three pathologies 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. On April 6, 2023, Sensorion announced the candidate selection for GJB2-GT during its gene therapy focused R&D Day. The candidate designed with a specific adeno-associated virus (AAV) capsid safely targets key cells in the ear that normally express GJB2. Sensorion has acquired bioreactors and has completed the non-GMP process development of the GJB2 candidate up to 50L scale. Process and analytical methods will be transferred to the CDMO selected by the Company for the GMP production of the clinical batch. Sensorion is advancing the candidate into IND/CTA enabling activities for future clinical development and anticipates the submission of the Clinical Trial Applications in Q2 2025. SENS-401: Sensorion’s small molecule for the prevention of hearing loss SENS-401(Arazasetron) is an orally available small molecule that aims to protect and preserve inner ear tissue from damage responsible for 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. Sensorion is developing SENS-401 in two Proof of Concept (POC) Phase 2a clinical trials. The first one is designed to assess SENS-401 for residual hearing preservation during cochlear implantation in partnership with Cochlear Limited (Cochlear), the global leader in implantable hearing solutions, and the second one is a trial assessing SENS-401 in Cisplatin-Induced Ototoxicity prevention (CIO) in adult patients undergoing a cisplatin-based chemotherapy. SENS-401 to prevent residual hearing loss after cochlear implantation. Milestone achieved with primary endpoint met. Final data readout planned in Q3 2024 Sensorion is developing SENS-401 in a multicentric, randomized, controlled open-label Phase 2a clinical 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 continue to receive SENS-401 for a further 42 days after implantation. In June 2023, Sensorion released preliminary data from its Phase 2a study, showing that SENS-401 was detected in the perilymph of all five adult patients treated with the product. Levels of SENS-401 were considered consistent with potential therapeutic effects after seven days of repeated oral treatment. In July 2023, the Company reported further analysis during its KOL webinar suggesting that SENS-401 treated patients demonstrated a clinically significant 21 dB improvement in the preservation of their residual hearing compared to the control group six weeks after cochlear implantation at 500 Hz. Indeed, in the SENS-401-treated group (N=5), the loss of residual hearing was only 12 dB, contrasting with a larger loss of 33 dB observed in the control group of four participants not treated with SENS-401. These findings reinforce the hypothesis that SENS-401, by crossing the labyrinthine barrier to reach the cochlear compartment, has a positive effect on the preservation of residual hearing. On February 1, 2024, Sensorion announced the completion of patient inclusion in the Phase 2a POC clinical trial, with 28 patients recruited in the study in total. Following this announcement, Sensorion communicated on March 11, 2024, that a total of 28 patients have been randomized and 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. The study is now completed, the follow-up of the last patients is still ongoing and the secondary endpoints including results on the preservation of the residual hearing will be available and analyzed later this year. The Company plans to publish the complete readout of the study in Q3 2024. SENS-401 to prevent Cisplatin Induced Ototoxicity (CIO) progressing as planned with efficacy and safety preliminary data expected in September 2024 Cisplatin and other platinum compounds are essential chemotherapeutic agents for many malignancies. Unfortunately, platinum-based therapies cause ototoxicity and hearing loss, which are permanent, irreversible and particularly harmful in about 60% of adult and pediatric patients treated. This indication represents a very significant unmet need for patients and is an attractive market with more than 500,000 patients forecast in 2025 in the G7 countries. SENS-401 is progressing in NOTOXIS, a multicenter, randomized, controlled, open-label Phase 2a clinical study, designed to evaluate the efficacy of SENS-401 to prevent CIO in adult patients with a neoplastic disease four weeks after the completion of cisplatin-based chemotherapy. The trial assesses several outcome measures, including the rate and severity of ototoxicity, the change from baseline in Pure Tone Audiometry (PTA) (dB) throughout the study and the tolerance. 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 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. In December 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 pro with previously reported data for patients exposed for up to 7 weeks. Recruitment is progressing at a sustained path, with eleven clinical centers open as of December 18, 2023. Sensorion’s management team will communicate preliminary efficacy and safety data of the POC Phase 2a clinical study of SENS-401 in CIO during the World Congress of Audiology, held on September 19-22, 2024, in Paris, France. Strengthening the Board of Directors and senior leadership On March 31, 2023, Sensorion appointed Khalil Barrage as interim Chairman of the Board. Mr. Barrage has served on the Board of Sensorion for over three years, supporting with several capital raises and providing leadership on the Company’s evolution towards becoming a gene therapy-focused company. He has been a managing director at Invus Group, a major investor in Sensorion, for nearly 20 years, heading its Public Equity group and serves on the Boards of Elevate Bio, Valerio Therapeutics, Orthobond and Protagenic Therapeutics. On August 3, 2023, following the completion of the private placement, Redmile Group (represented by Natalie Berner) became a board member in replacement of Bpifrance Investissement, whose permanent representative was Jean-François Morin. On December 12, Sensorion announced the appointment of CMC (Chemistry, Manufacturing and Control) expert Bernd Schmidt as Chief Technical Officer. He brings over 20 years of industry experience in the pharmaceutical sector, covering a broad range of innovative medicines at different stages in development and post launch. On January 25, 2024, Sensorion announced the nomination of Dr. Federico Mingozzi as Non-Executive Director to the Board of Directors. Mr. Mingozzi brings over 25 years of experience in gene therapy, immunology, as well as biochemistry and molecular biology in academia and industry. 2024 Outlook As of December 31, 2023, the Company had €37.0 million in cash and cash equivalents. Based on this cash position, the net proceeds from the €50.5 million financing announced on February 9, 2024, and its forecasted expenses, the company expects it will be able to finance its operations up until the end of Q2 2025. In 2024, Sensorion plans on pursuing developments across its portfolio of innovative programs with the aim of treating, restoring and preventing hearing disorders and improving the quality of life of patients suffering from hearing disorders. The Company plans to communicate about the first patient enrolled in Audiogene, its Phase 2a clinical trial of its gene therapy product, SENS-501, in H2 2024. The Company is on track to submit Clinical Trial Applications for its second gene therapy, GJB2-GT, in H1 2025, with ongoing IND/CTA-enabling preclinical activities. In parallel, SENS-401, Sensorion’s orally available small molecule, is progressing in two Phase 2a clinical trials. Final data readout is expected in Q3 2024 for its trial of SENS-401 in the prevention of residual hearing loss following cochlear implantation, and preliminary safety and efficacy are expected in September 2024 during the World Congress of Audiology for its second ongoing clinical trial of SENS-401, in the prevention of Cisplatin-Induced Ototoxicity. Expected next clinical milestones: Q3 2024 – SENS-401 in combination with cochlear implantation: Final data readout H2 2024, World Congress of Audiology – SENS-401 in Cisplatin-Induced Ototoxicity: Preliminary safety and efficacy data H2 2024 – SENS-501: First patient communication H1 2025 – SENS-501: Enrollment of the first two cohorts of Phase 1/2 Audiogene trial completed Q2 2025 – GJB2-GT: Clinical Trial Applications Submission Full Year 2023 financial highlights Cash Position Cash & Cash Equivalents amounted to €37.0 million as at December 31, 2023, compared to €26.2 million at December 31, 2022. Research And Development (R&D) Expenses R&D expenses decreased by 0.7 % from €22.9 million in 2022 to €22.8 million in 2023. General And Administrative (G&A) Expenses G&A expenses were €5.3 million for 2023, compared to €5.2 million for 2022. Net Loss Net loss was -€22.1 million for 2023, compared to -€23.2 million for 2022. Financial guidance Based on cash and cash equivalents of €37.0 million at 31 December 2023, and the net proceeds of a €50.5 million offering of new shares announced on February 9, 2024, amounting to c. €47 million, the Company has sufficient net working capital to meet its cash requirements beyond the next twelve months, i.e. until the end of Q2 2025. Financial results The annual accounts as at December 31, 2023, were prepared according to IFRS standards and approved by the Board of Directors on March 13, 2024. The simplified income statement as of December 31, 2023, is as follows: In thousands of Euros – IFRS standards 31.12.2023 31.12.2022 Operating income 5,698 5,006 Research & Development expenses -22,756 -22,925 General & Administrative expenses -5,252 -5,217 Total operating expenses -28,008 -28,142 Operating loss -22,310 -23,137 Financial result 544 -72 Corporate Income Tax -297 0 Net loss -22,063 -23,209 The simplified balance sheet at December 31, 2023, is as follows: In thousands of Euros – IFRS standards 31.12.2023 31.12.2022 Non-current Assets 3,236 3,176 Other Current Assets 6,293 9,565 Cash & cash equivalent 36,974 26,204 Total Assets 46,503 38,945 Equity 33,276 21,885 Non-current Liabilities 2,950 3,467 Current Liabilities 10,278 13,593 Total Liabilities 46,503 38,945 2023 certified accounts On March 13, 2024, the Board of Directors approved the Company’s full year results as of December 31, 2023. The Full Year Financial Report can be found on Sensorion’s website ( ) in the investor section under financial information. The full year accounts of 2023 have been subject to a limited review by the Company’s statutory auditors and an unqualified report is being issued. 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) targets deafness caused by mutations of the gene encoding for otoferlin and is currently developed in a Phase 1/2 clinical study, 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 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. 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 financial 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, Reynoso R, Olarte M, Villamar M, Morera C, Santarelli R, Arslan E, Medá C, Curet C, Völter C, Sainz-Quevedo M, Castorina P, Ambrosetti U, Berrettini S, Frei K, Tedín S, Smith J, Cruz Tapia M, Cavallé L, Gelvez N, Primignani P, Gómez-Rosas E, Martín 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. View source version on businesswire.com: Contacts Investor Relations Noémie Djokovic, Investor Relations and Communications Associate ir.contact@sensorion-pharma.com Press Relations Ulysse Communication Pierre-Louis Germain / +33 (0)6 64 79 97 51 plgermain@ulysse-communication.com Bruno Arabian / +33(0)6 87 88 47 26 barabian@ulysse-communication.com Source: Sensorion View this news release online at:

March 12, 2024 -- Arctic Therapeutics ehf, an Icelandic drug development company and partner Nacuity Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other diseases caused by oxidative stress, today announced approval by the European Medicines Agency (EMA) for initiation of the first clinical trial evaluating AT-001 (Nacuity’s NPI-001) for the treatment of Hereditary Cystatin C Amyloid Angiopathy (HCCAA) in Iceland. HCCAA is an ultra rare, serious condition characterized by the accumulation of amyloid in the brain's blood vessels, which can lead to cerebral hemorrhage, stroke, and progressive neurological deterioration, including dementia. Current treatment options are severely limited, focusing primarily on symptom management rather than addressing the underlying cause of the disease. AT-001 is a differentiated, purified, prescription-quality, GMP-grade N-acetylcysteine amide (NACA). “EMA's approval paves the way for a comprehensive investigation into the safety and efficacy of a novel therapeutic approach designed to halt or reverse the progression of HCCAA. Our study aims to explore the potential of AT-001 in modifying the disease's course, improving patient outcomes, and ultimately, saving lives,” said Ivar Hakonarson, CEO and co-founder of Arctic. In September 2022, Nacuity and Arctic entered into an exclusive licensing agreement and strategic partnership for the development and commercialization of Nacuity’s NPI-001 under the name AT-001 for HCCAA in Iceland and related forms of amyloid angiopathies (CAAs) worldwide. Nacuity is also currently evaluating NPI-001 in Phase 1/2 clinical trials in Australia for retinitis pigmentosa associated with Usher syndrome and cystinosis. “Our proprietary NPI-001 has potential to address the myriad diseases and conditions where oxidative stress plays a role. EMA approval for the evaluation of NPI-001 tablets for treatment of HCCAA represents most importantly, a step forward in addressing HCCAA, a life threatening and underserved rare disease, and secondly, a key pharmaceutical quality milestone for the companies in the global development of NPI-001,” said G. Michael Wall, Ph.D., SVP & CSO, Nacuity. Nacuity Pharmaceuticals, Inc. (http://www.nacuity.com/), is a clinical-stage leader in innovative treatments for oxidative stress. The company’s powerful, targeted therapies aim to attentuate oxidative tissue damage, a driver of blinding eye diseases and a broad spectrum of serious chronic conditions. Nacuity has three highly differentiated clinical programs ongoing in retinitis pigmentosa, cataracts and cystinosis with the potential to be first-of-a-kind therapies and gateways to wider applications. Nacuity has operations in Fort Worth, TX, USA, and Australia, and extensive managerial and scientific domain expertise as well as backing from Foundation Fighting Blindness (https://www.fightingblindness.org/) and its venture arm RD Fund (https://www.retinaldegenerationfund.org). Arctic Therapeutics ehf (AT) (https://www.arctictherapeutics.com/) is an Icelandic drug development company, established in 2015 as a spin-off from the US-based Center for Applied Genomics (CAG), a research center at the Children's Hospital of Philadelphia. AT leverages the transformative power of applied genomics, steering the course of drug development towards safer, more effective treatments for some of the world’s most challenging diseases. The company has operations in Iceland, USA, and multiple collaborations across Europe. The content above comes from the network. if any infringement, please contact us to modify.