Kiora Pharmaceuticals Receives Investigational New Drug Approval to Initiate ABACUS-2, a Phase 2 Clinical Trial of KIO-301 for the Treatment of Retinitis Pigmentosa | KPRX Stock News

Kiora Pharmaceuticals (NASDAQ: KPRX) received regulatory approval to initiate ABACUS-2, a Phase 2 clinical trial for KIO-301 in retinitis pigmentosa patients. The trial will involve 36 patients across five Australian centers, with participants randomized 2:1 to receive KIO-301 or control. The study will evaluate two dosage levels (50 and 100 micrograms) administered intravitreally every 6 weeks. KIO-301 is a photoswitch molecule designed to restore vision by targeting retinal ganglion cells, potentially benefiting patients regardless of their specific gene mutation. The trial's primary endpoints include safety and tolerability, with efficacy measured through functional vision assessments.

Kiora Pharmaceuticals (NASDAQ: KPRX) ha ricevuto l'approvazione normativa per avviare ABACUS-2, uno studio clinico di Fase 2 per KIO-301 nei pazienti con retinite pigmentosa. La sperimentazione coinvolgerà 36 pazienti in cinque centri australiani, con i partecipanti randomizzati nel rapporto 2:1 per ricevere KIO-301 o un trattamento di controllo. Lo studio valuterà due livelli di dosaggio (50 e 100 microgrammi) somministrati per via intravitreale ogni 6 settimane. KIO-301 è una molecola fotosensibile progettata per ripristinare la vista agendo sulle cellule gangliari retiniche, potenzialmente a beneficio dei pazienti indipendentemente dalla specifica mutazione genetica. Gli obiettivi primari della sperimentazione includono la sicurezza e la tollerabilità, con l'efficacia misurata attraverso valutazioni della visione funzionale.

Kiora Pharmaceuticals (NASDAQ: KPRX) recibió la aprobación regulatoria para iniciar ABACUS-2, un ensayo clínico de Fase 2 para KIO-301 en pacientes con retinitis pigmentosa. El ensayo involucrará a 36 pacientes en cinco centros australianos, con los participantes randomizados 2:1 para recibir KIO-301 o un control. El estudio evaluará dos niveles de dosificación (50 y 100 microgramos) administrados intravítreamente cada 6 semanas. KIO-301 es una molécula fotosensible diseñada para restaurar la visión al dirigirse a las células ganglionares retinianas, lo que podría beneficiar a los pacientes independientemente de su mutación genética específica. Los objetivos primarios del ensayo incluyen la seguridad y la tolerabilidad, con la eficacia medida a través de evaluaciones de la visión funcional.

Kiora Pharmaceuticals (NASDAQ: KPRX)는 망막색소변성증 환자를 위한 KIO-301에 대한 ABACUS-2 2상 임상 시험 개시를 위한 규제 승인을 받았습니다. 이 시험은 호주 내 5개 센터에서 36명의 환자를 대상으로 하며, 참가자는 KIO-301 또는 대조 약물을 받기 위해 2:1로 무작위 배정됩니다. 연구는 6주마다 안구 내 투여되는 두 가지 용량(50마이크로그램과 100마이크로그램)을 평가합니다. KIO-301은 망막 신경세포를 타겟으로 시력을 회복하도록 설계된 광스위치 분자로, 특정 유전자 변이에 관계없이 환자에게 혜택을 줄 수 있습니다. 시험의 주요 목표는 안전성과 내약성을 포함하며, 효과성은 기능적 시각 평가를 통해 측정됩니다.

Kiora Pharmaceuticals (NASDAQ: KPRX) a reçu l'approbation réglementaire pour lancer ABACUS-2, un essai clinique de phase 2 pour KIO-301 chez des patients atteints de rétinite pigmentaire. L'essai impliquera 36 patients répartis dans cinq centres australiens, les participants étant randomisés dans un rapport de 2:1 pour recevoir KIO-301 ou un traitement de contrôle. L'étude évaluera deux niveaux de dosage (50 et 100 microgrammes) administrés par voie intravitréenne toutes les 6 semaines. KIO-301 est une molécule photosensible conçue pour restaurer la vision en ciblant les cellules ganglionnaires rétiniennes, ce qui pourrait bénéficier aux patients, quel que soit leur type spécifique de mutation génétique. Les objectifs principaux de l'essai comprennent la sécurité et la tolérabilité, avec l'efficacité mesurée par des évaluations de la vision fonctionnelle.

Kiora Pharmaceuticals (NASDAQ: KPRX) erhielt die behördliche Genehmigung zum Start von ABACUS-2, einer Phase-2-Studie für KIO-301 bei Patienten mit Retinitis pigmentosa. Die Studie wird 36 Patienten in fünf australischen Zentren umfassen, wobei die Teilnehmer im Verhältnis 2:1 randomisiert KIO-301 oder ein Kontrollpräparat erhalten. Die Untersuchung wird zwei Dosisstufen (50 und 100 Mikrogramm) testen, die alle 6 Wochen intravitreally verabreicht werden. KIO-301 ist ein Lichtschalter-Molekül, das entwickelt wurde, um das Sehvermögen durch die gezielte Ansprache von retinalen Ganglienzellen wiederherzustellen, was potenziell Patienten unabhängig von ihrer spezifischen Genmutation zugutekommt. Die Hauptziele der Studie umfassen Sicherheit und Verträglichkeit, während die Wirksamkeit durch funktionelle Sehbeurteilungen gemessen wird.

This Phase 2 trial approval for KIO-301 represents a significant milestone in addressing the unmet medical need in retinitis pigmentosa treatment. The 36-patient study incorporates several important design elements that strengthen its potential success:

The trial's focus on functional vision outcomes aligns with regulatory requirements for approval. KIO-301's mechanism as a photoswitch targeting retinal ganglion cells could potentially benefit patients regardless of genetic mutation - a key differentiator in the market. However, investors should note that meaningful results won't be available until well into 2024, given the endpoint validation work needed before first patient dosing.

With a micro-cap market value of just $11.7M, this regulatory approval could significantly impact Kiora's valuation. The partnership with Théa Open Innovation provides important validation and support for the development program. The addressable market is substantial, considering retinitis pigmentosa affects approximately 1 in 4,000 people globally. The gene-agnostic approach could potentially capture the entire patient population, unlike gene-specific therapies that target only specific mutations. The validation of novel endpoints, while extending the timeline, reduces regulatory risk and could accelerate the path to market through a potential single Phase 3 trial strategy for both US and European approval.

Encinitas, California--(Newsfile Corp. - October 29, 2024) - Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX) ("Kiora" or the "Company") today announced it received regulatory approval to initiate a Phase 2 clinical trial to investigate KIO-301 for vision restoration in patients with retinitis pigmentosa. The ABACUS-2 trial will be a 36 patient, multi-center, double-masked, randomized, controlled, multiple dose study enrolling patients with ultra-low vision or no light perception regardless of their underlying gene mutation associated with retinitis pigmentosa. Dosing of the first patient with KIO-301 is expected to begin next year following validation of novel functional vision endpoints. These functional assessments may serve as approvable primary endpoints in subsequent registration studies in the United States, Europe and other major regions.

"There are unfortunately no approved therapies for patients with retinitis pigmentosa," said Eric Daniels, M.D., Chief Development Officer at Kiora. "This study represents a significant step toward addressing this challenge. Prior to submission for approval, we engaged with European and US regulators to incorporate their expectations and guidance for approvable endpoints. Consistent with historical approval in other inherited retinal disease, both regulatory bodies emphasized the need to measure a therapy's effect on everyday functional vision. For this reason, we are investing time upfront to validate the functional endpoints for ABACUS-2, increasing our likelihood of success in a potential single Phase 3 trial for market approval in the US and Europe. This validation work is being performed, in collaboration with our partner Théa Open Innovation, with the support of the Choroideremia Research Foundation as part of a grant to design a standard endpoint for investigational therapies of inherited retinal diseases."

Kiora has identified and is now contracting with trial sites and clinical investigators at major inherited retinal disease reference centers across Australia. Patients will be randomized 2:1 to receive KIO-301 intravitreally or control. Treatment will be delivered to both eyes. Participants will be randomized to receive either a high dose (100 micrograms) or low dose (50 micrograms) of KIO-301 and after trial participants receive three consecutive doses (6 weeks apart), they will be followed for three months. Following this phase of the study, patients in the control arm may elect to cross-over into the active arm. Primary endpoints will consist of safety and tolerability, and key efficacy assessments include: functional vision; visual acuity as measured by the Berkeley Rudimentary Vision Test; visual fields as measured by perimetry, and a validated ultra-low vision quality-of-life questionnaire. The trial will be conducted across five centers within Australia.

"In a short window since entering our partnership with Kiora, tremendous progress has been made on advancing KIO-301 toward ABACUS-2," said Dr. Céline Olmiere, Head of Théa Open Innovation. "What makes KIO-301 compelling is that it appears, based on the Phase 1b data, to have potential for meaningful vision restoration. Further, because of its unique mechanism of action, it has the potential to work across all 150-plus underlying gene mutations associated with retinitis pigmentosa and other inherited retinal diseases."

KIO-301 is a small molecule that acts as a light-sensitive photoswitch that has the potential to return vision to patients living with reduced sight due to inherited retinal diseases. The novel compound is activated in the presence of light and deactivated in the absence of light. Because hundreds of gene mutations underlie inherited retinal diseases like retinitis pigmentosa, there is an important need for therapies like KIO-301 that have the potential to act in a gene mutation agnostic manner.

KIO-301 targets and enters specialized cells of the retina (retinal ganglion cells or RGCs) that are located 'downstream' of degenerated rods and cones, the cells normally responsible for converting light to vision. KIO-301, when inside RGCs, is activated by visible light and confers light-sensing capabilities by altering the flow of ions in and out of the cell, thus giving RGCs the ability to facilitate visual processing. This is a reversible process allowing turning on and off the RGCs in the presence and absence of light.

About Kiora PharmaceuticalsKiora Pharmaceuticals is a clinical-stage biotechnology company developing and commercializing products for the treatment of retinal diseases. KIO-301 is being developed for the treatment of retinitis pigmentosa, choroideremia, and Stargardt disease. It is a molecular photoswitch that has the potential to restore vision in patients with inherited and/or age-related retinal degeneration. KIO-104 is being developed for the treatment of retinal inflammation. It is a next-generation, non-steroidal, immuno-modulatory, and small-molecule inhibitor of dihydroorotate dehydrogenase.

In addition to news releases and SEC filings, we expect to post information on our website (www.kiorapharma.com) and social media accounts that could be relevant to investors. We encourage investors to follow us on Twitter and LinkedIn as well as to visit our website and/or subscribe to email alerts.

About Théa Open Innovation

Théa Open Innovation (TOI) is a sister company of Théa, the leading independent European pharmaceutical company specialized in the research, development, and commercialization of eye care products. TOI's mission is to establish partnerships with startups, biotech companies, and universities to help bring the most cutting-edge eye care products to the market.

For more information, visit www.theaopeninnovation.com

Forward-Looking Statements

Some of the statements in this press release are "forward-looking" and are made pursuant to the safe harbor provision of the Private Securities Litigation Reform Act of 1995. These "forward-looking" statements include statements relating to, among other things, Kiora's ability to execute on development and commercialization efforts and other regulatory or marketing approval efforts pertaining to Kiora's development-stage products, including KIO-104 and KIO-301, as well as the success thereof, with such approvals or success may not be obtained or achieved on a timely basis or at all, the sufficiency of existing cash on hand to fund operations for specific periods, the projected cash runway, and Kiora's plans to further fund development of KIO-104. These statements involve risks and uncertainties that may cause results to differ materially from the statements set forth in this press release, including, among other things, the ability to satisfy the closing conditions related to the offering ,the ability to conduct clinical trials on a timely basis, market and other conditions and certain risk factors described under the heading "Risk Factors" contained in Kiora's Annual Report on Form 10-K filed with the SEC on March 25, 2024 or described in Kiora's other public filings including on Form 10-Q filed with the SEC on August 9, 2024. Kiora's results may also be affected by factors of which Kiora is not currently aware. The forward-looking statements in this press release speak only as of the date of this press release. Kiora expressly disclaims any obligation or undertaking to release publicly any updates or revisions to such statements to reflect any change in its expectations with regard thereto or any changes in the events, conditions, or circumstances on which any such statement is based, except as required by law.

Contacts:

[email protected]

To view the source version of this press release, please visit https://www.newsfilecorp.com/release/228101

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