Kiora Pharmaceuticals Receives European Orphan Medicinal Product Designation for KIO-301 for the Treatment of Inherited Retinal Dystrophies
Kiora Pharmaceuticals (NASDAQ: KPRX) has received Orphan Medicinal Product Designation from the European Medicines Agency for KIO-301, a small molecule photoswitch, to treat inherited retinal dystrophies (IRDs) including retinitis pigmentosa. This designation provides at least ten years of market exclusivity in Europe, along with other regulatory benefits. Kiora is finalizing the design of the Phase 2 trial (ABACUS-2) for KIO-301, set to begin later this year.
KIO-301 is designed to restore light-sensing capabilities to retinal ganglion cells in patients with IRDs. In January 2024, Kiora partnered with Théa Open Innovation for worldwide co-development and commercialization of KIO-301, excluding Asia. The upcoming ABACUS-2 trial will be a multi-center, double-masked, randomized, controlled, multiple-dose study focusing on vision restoration in individuals with retinitis pigmentosa.
Kiora Pharmaceuticals (NASDAQ: KPRX) ha ottenuto la designazione di Medicinale Orfano dall'Agenzia Europea per i Medicinali per KIO-301, una piccola molecola fotosensibile, per trattare le distrofie retiniche ereditarie (IRD), inclusa la retinite pigmentosa. Questa designazione garantisce almeno dieci anni di esclusiva di mercato in Europa, insieme ad altri benefici normativi. Kiora sta finalizzando il progetto del trial di Fase 2 (ABACUS-2) per KIO-301, che inizierà entro la fine dell'anno.
KIO-301 è progettato per ripristinare le capacità di rilevamento della luce nelle cellule gangliari retiniche dei pazienti con IRD. Nel gennaio 2024, Kiora ha stipulato una partnership con Théa Open Innovation per il co-sviluppo e la commercializzazione a livello mondiale di KIO-301, escludendo l'Asia. Il prossimo trial ABACUS-2 sarà uno studio multicentrico, doppio cieco, randomizzato e controllato, con dosi multiple, focalizzato sul ripristino della visione in individui con retinite pigmentosa.
Kiora Pharmaceuticals (NASDAQ: KPRX) ha recibido la designación de Producto Medicinal Huérfano de la Agencia Europea de Medicamentos para KIO-301, una pequeña molécula fotosensible, para tratar las distrofias retinianas hereditarias (IRDs) incluyendo la retinitis pigmentosa. Esta designación proporciona al menos diez años de exclusividad en el mercado en Europa, junto con otros beneficios regulatorios. Kiora está finalizando el diseño del ensayo de Fase 2 (ABACUS-2) para KIO-301, que comenzará más adelante este año.
KIO-301 está diseñado para restaurar las capacidades de detección de luz en las células ganglionares de la retina en pacientes con IRDs. En enero de 2024, Kiora se asoció con Théa Open Innovation para el co-desarrollo y comercialización mundial de KIO-301, excluyendo Asia. El próximo ensayo ABACUS-2 será un estudio multicéntrico, doble ciego, aleatorizado y controlado, con múltiples dosis, centrado en la restauración de la visión en individuos con retinitis pigmentosa.
Kiora Pharmaceuticals (NASDAQ: KPRX)는 유럽 의약품 청으로부터 유전성 망막 질환(IRD), 특히 망막색소변성이 포함된 질환을 치료하기 위해 소형 분자 포토스위치인 KIO-301에 대해 의약품 희귀질환 지정을 받았습니다. 이 지정은 유럽에서 최소 10년의 시장 독점권을 보장하며, 다른 규제 혜택도 포함됩니다. Kiora는 올해 말 시작될 2상 시험(ABACUS-2)의 디자인을 마무리하고 있습니다.
KIO-301은 IRD 환자의 망막 신경 세포에 빛 감지 기능을 복원하도록 설계되었습니다. 2024년 1월, Kiora는 아시아를 제외하고 KIO-301의 전 세계 공동 개발 및 상용화를 위해 Théa Open Innovation과 파트너십을 체결했습니다. 다가오는 ABACUS-2 시험은 망막색소변성 환자의 시력 복원에 초점을 맞춘 다기관, 이중 맹검, 무작위, 대조, 다회 투여 연구가 될 것입니다.
Kiora Pharmaceuticals (NASDAQ: KPRX) a reçu la désignation de médicament orphelin de l'Agence européenne des médicaments pour KIO-301, une petite molécule photosensible, pour traiter les dystrophies rétiniennes héréditaires (IRD) y compris la rétinite pigmentaire. Cette désignation offre au moins dix ans d'exclusivité sur le marché en Europe, ainsi que d'autres avantages réglementaires. Kiora finalise le design de l'essai de phase 2 (ABACUS-2) pour KIO-301, qui devrait commencer plus tard cette année.
KIO-301 est conçu pour restaurer les capacités de détection de la lumière des cellules ganglionnaires de la rétine chez les patients atteints d'IRD. En janvier 2024, Kiora a conclu un partenariat avec Théa Open Innovation pour le co-développement et la commercialisation à l'échelle mondiale de KIO-301, à l'exclusion de l'Asie. L'essai ABACUS-2 à venir sera une étude multicentrique, en double aveugle, randomisée, contrôlée et à doses multiples, axée sur la restauration de la vision chez les individus atteints de rétinite pigmentaire.
Kiora Pharmaceuticals (NASDAQ: KPRX) hat von der Europäischen Arzneimittel-Agentur die Orphan-Medizinprodukt-Zulassung für KIO-301, ein kleines Molekül-Photoswitch, zur Behandlung von erblichen Netzhauterkrankungen (IRD) einschließlich der Retinitis pigmentosa erhalten. Diese Zulassung gewährt mindestens zehn Jahre Marktexklusivität in Europa sowie weitere regulatorische Vorteile. Kiora finalisiert derzeit das Design der Phase-2-Studie (ABACUS-2) für KIO-301, die noch in diesem Jahr beginnen soll.
KIO-301 ist darauf ausgelegt, die Lichterkennungsfähigkeiten der retinalen Ganglienzellen bei Patienten mit IRD wiederherzustellen. Im Januar 2024 hat Kiora eine Partnerschaft mit Théa Open Innovation für die weltweite Co-Entwicklung und Kommerzialisierung von KIO-301, mit Ausnahme Asiens, geschlossen. Die bevorstehende ABACUS-2-Studie wird eine multizentrische, doppelblinde, randomisierte, kontrollierte Studie mit mehreren Dosen sein, die sich auf die Wiederherstellung des Sehvermögens bei Personen mit Retinitis pigmentosa konzentriert.
- Received Orphan Medicinal Product Designation from EMA for KIO-301
- At least ten years of market exclusivity in Europe
- Phase 2 trial (ABACUS-2) for KIO-301 to begin later this year
- Worldwide co-development and commercialization agreement with Théa Open Innovation
- None.
This news from Kiora Pharmaceuticals is indeed significant for the company and the field of inherited retinal dystrophies (IRDs). The European Orphan Medicinal Product Designation for KIO-301 is a major milestone that could accelerate the drug's development and eventual market access.
The designation covers a broad range of IRDs, including retinitis pigmentosa (RP) and choroideremia, which are characterized by the degeneration of rod photoreceptors. This is important because:
- There are currently no approved therapeutics for RP, highlighting the unmet medical need.
- The designation provides 10 years of market exclusivity in Europe, a significant competitive advantage.
- It offers regulatory and financial benefits, including reduced fees and scientific advice from the EMA.
KIO-301's mechanism of action as a small molecule photoswitch is innovative. It aims to confer light-sensing capabilities to retinal ganglion cells after photoreceptor degeneration, offering a gene-mutation agnostic approach. This could potentially address a wider patient population compared to gene-specific therapies.
The partnership with Théa Open Innovation for co-development and commercialization adds credibility and resources to the project. The upcoming ABACUS-2 Phase 2 trial will be important in demonstrating KIO-301's efficacy and safety.
While promising, investors should note that the drug is still in early clinical stages and success in Phase 2 trials is not guaranteed. However, the orphan designation significantly de-risks the regulatory pathway and enhances the commercial potential if approved.
Kiora Pharmaceuticals' announcement of receiving European Orphan Medicinal Product Designation for KIO-301 is a significant positive development for the company's pipeline and potential market position.
Key financial implications include:
- Market Exclusivity: The 10-year market exclusivity in Europe, if KIO-301 is approved, provides a substantial competitive moat and potential for premium pricing.
- Cost Savings: Reduced or waived regulatory fees can significantly lower development costs, important for a small-cap biotech like Kiora (NASDAQ: KPRX).
- Accelerated Development: EMA scientific advice and protocol assistance could potentially speed up the development process, reducing time-to-market and associated costs.
The partnership with Théa Open Innovation for worldwide co-development and commercialization (excluding Asia) is another strong positive. It provides:
- Additional resources and expertise for clinical development
- Potential for milestone payments and shared commercialization revenues
- Validation of KIO-301's potential from an established ophthalmic company
Investors should note that while this news is encouraging, Kiora is still a clinical-stage company with no approved products. The success of the upcoming ABACUS-2 Phase 2 trial will be important for the company's valuation. Additionally, the exclusion of Asia from the Théa deal leaves open questions about Kiora's strategy for that significant market.
Overall, this designation strengthens Kiora's position in the competitive landscape of IRD treatments and could potentially lead to increased investor interest and partnership opportunities.
Encinitas, California--(Newsfile Corp. - July 30, 2024) - Kiora Pharmaceuticals (NASDAQ: KPRX) ("Kiora" or the "Company") today announced it has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) for the treatment of a group of inherited retinal diseases (IRDs) that include retinitis pigmentosa (RP), choroideremia and more. The broad designation covers KIO-301, a small molecule photoswitch, for the treatment of non-syndromic rod-dominant retinal dystrophies. This classification of dystrophies, in simplified terms, refers to rare diseases from underlying genetic mutations resulting in predominantly and primarily the degeneration of rod photoreceptors, the cells responsible almost entirely for night vision as well as peripheral and non-color vision. In RP, which today has no approved therapeutics, rods degenerate first followed by degeneration of cone photoreceptors, the cells responsible for color and central vision. This degenerative process causes partial or severe loss of vision and may result in complete blindness. There are approximately 92 million rods in the human eye compared to approximately 6 million cones.
"This designation provides Kiora and our development and commercialization partner, Théa Open Innovation, with at least ten years of market exclusivity, exclusive of patent protection, in Europe. This is in addition to other regulatory and market access benefits," said Eric Daniels, M.D., Chief Development Officer of Kiora Pharmaceuticals. "With Orphan Drug Designation already granted in the U.S., KIO-301 is well positioned for expediated regulatory guidance, review, and market exclusivity in two major marketplaces. We are finalizing the design and planning to initiate the Phase 2 trial of KIO-301 (ABACUS-2) later this year."
In addition to market exclusivity, additional benefits of Orphan Medicinal Product Designation includes:
A centralized process for EU market approval;
Reduced or waived fees for regulatory activities; and
EMA scientific advice and protocol assistance to optimize trial design.
The European Union provides Orphan Medicinal Product Designation to incentivize drug developers to bring products for rare diseases to market that may otherwise not be developed. The designation is granted after a critical review and only if a drug candidate meets specific criteria, which incudes but is not limited to the following:
The investigational therapy is medically plausible and intended to treat a serious or life-threatening disease; and
The condition affects no more than 5 in 10,000 people in the EU.
Kiora's upcoming ABACUS-2 trial of KIO-301 for vision restoration in individuals with RP, will be a multi-center, double-masked, randomized, controlled, multiple-dose study. KIO-301 is a small molecule photoswitch designed to selectively confer light-sensing capabilities to retinal ganglion cells following the degeneration of photoreceptors in inherited retinal diseases like retinitis pigmentosa. This represents a novel, gene-mutation agnostic, non-cell or gene therapy approach to addressing vision loss in IRDs.
In January 2024, Kiora, along with Théa Open Innovation (TOI), a sister company of the global ophthalmic specialty company Laboratoires Théa (Théa), agreed to an exclusive worldwide co-development and commercialization agreement, excluding Asia, for KIO-301 in the treatment of retinal diseases.
About Kiora Pharmaceuticals
Kiora Pharmaceuticals is a clinical-stage biotechnology company developing and commercializing products for the treatment of orphan 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 posterior non-infectious uveitis. 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.
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 May 10, 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:
Investors
investors@kiorapharma.com
Media
kiora@crowepr.com
To view the source version of this press release, please visit https://www.newsfilecorp.com/release/218152
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