Kiora Receives Approval to Initiate KLARITY, a Phase 2 Clinical Trial of KIO-104 for the Treatment of Retinal Macular Edema
Kiora Pharmaceuticals (NASDAQ: KPRX) has received regulatory approval to begin KLARITY, a Phase 2 clinical trial for KIO-104 in treating retinal macular edema. The trial will evaluate a locally delivered small molecule as an alternative to steroids or systemic anti-inflammatory drugs.
The multi-center, open-label study will include up to 28 patients and will be conducted in two parts: Part A (Dose Optimization) will assess safety and efficacy of three injections every two weeks in 8 subjects across two cohorts (3.5 μg and 10 μg doses), while Part B (Cohort Expansion) will investigate different dosing regimens at intervals of 2 or 4 weeks.
KIO-104 works by suppressing T cell numbers and function in the eye through DHODH inhibition, targeting inflammation-related vision conditions including diabetic retinopathy, non-infectious uveitis, retinal vein occlusion, and post pseudophakic cataract surgery.
Kiora Pharmaceuticals (NASDAQ: KPRX) ha ricevuto l'approvazione normativa per avviare KLARITY, un clinical trial di Fase 2 per KIO-104 nel trattamento dell'edema maculare retinico. Lo studio valuterà un piccolo composto molecolare somministrato localmente come alternativa agli steroidi o ai farmaci antinfiammatori sistemici.
Lo studio multicentrico, in aperto, includerà fino a 28 pazienti e sarà condotto in due parti: Parte A (Ottimizzazione della Dose) valuterà la sicurezza e l'efficacia di tre iniezioni ogni due settimane in 8 soggetti suddivisi in due coorti (dosaggi da 3,5 μg e 10 μg), mentre Parte B (Espansione della Coorte) indagherà diversi regimi di dosaggio a intervalli di 2 o 4 settimane.
KIO-104 agisce sopprimendo il numero e la funzione delle cellule T nell'occhio attraverso l'inibizione di DHODH, mirando a condizioni visive legate all'infiammazione, inclusa la retinopatia diabetica, l'uveite non infettiva, l'occlusione della vena retinica e l'intervento chirurgico post-cataratta pseudofachica.
Kiora Pharmaceuticals (NASDAQ: KPRX) ha recibido la aprobación regulatoria para comenzar KLARITY, un ensayo clínico de Fase 2 para KIO-104 en el tratamiento del edema macular retinal. El ensayo evaluará una pequeña molécula administrada localmente como alternativa a los esteroides o a los medicamentos antiinflamatorios sistémicos.
El estudio multicéntrico, abierto, incluirá hasta 28 pacientes y se llevará a cabo en dos partes: Parte A (Optimización de la Dosis) evaluará la seguridad y eficacia de tres inyecciones cada dos semanas en 8 sujetos distribuidos en dos cohortes (dosis de 3.5 μg y 10 μg), mientras que la Parte B (Expansión de Cohorte) investigará diferentes regímenes de dosificación con intervalos de 2 o 4 semanas.
KIO-104 actúa suprimiendo el número y la función de las células T en el ojo a través de la inhibición de DHODH, dirigiéndose a condiciones visuales relacionadas con la inflamación, incluida la retinopatía diabética, la uveítis no infecciosa, la oclusión de la vena retiniana y la cirugía posterior a la catarata pseudofáquica.
Kiora Pharmaceuticals (NASDAQ: KPRX)는 망막 황반 부종 치료를 위한 KIO-104의 2상 임상 시험인 KLARITY를 시작하기 위한 규제 승인을 받았습니다. 이 시험은 스테로이드 또는 전신 항염증제의 대안으로 국소적으로 전달되는 소분자 화합물을 평가합니다.
이 다중 센터 공개 연구는 최대 28명의 환자를 포함하며, 두 부분으로 진행됩니다: A부분(용량 최적화)은 두 개의 코호트(3.5 μg 및 10 μg 용량)에서 8명의 피험자에게 2주마다 3회의 주사에 대한 안전성과 유효성을 평가하고, B부분(코호트 확장)은 2주 또는 4주 간격으로 다양한 용량 요법을 조사합니다.
KIO-104는 DHODH 억제를 통해 눈에서 T세포의 수와 기능을 억제하여 당뇨망막병증, 비감염성 포도막염, 망막 정맥 폐쇄 및 인공 수정체 수술 후 염증 관련 시각 질환을 타겟으로 합니다.
Kiora Pharmaceuticals (NASDAQ: KPRX) a reçu l'approbation réglementaire pour commencer KLARITY, un essai clinique de Phase 2 pour KIO-104 dans le traitement de l'œdème maculaire rétinien. L'essai évaluera une petite molécule délivrée localement comme alternative aux stéroïdes ou aux médicaments anti-inflammatoires systémiques.
Cette étude multicentrique, en ouvert, comprendra jusqu'à 28 patients et se déroulera en deux parties : Partie A (Optimisation de la Dose) évaluera la sécurité et l'efficacité de trois injections toutes les deux semaines chez 8 sujets répartis en deux cohortes (doses de 3,5 μg et 10 μg), tandis que la Partie B (Expansion des Cohortes) examinera différents schémas de dosages à des intervalles de 2 ou 4 semaines.
KIO-104 agit en supprimant le nombre et la fonction des cellules T dans l'œil par inhibition de DHODH, ciblant des maladies oculaires liées à l'inflammation, y compris la rétinopathie diabétique, l'uvéite non infectieuse, l'occlusion veineuse rétinienne et la chirurgie post-cataracte pseudophake.
Kiora Pharmaceuticals (NASDAQ: KPRX) hat die behördliche Genehmigung erhalten, KLARITY zu beginnen, eine Phase-2-Studie für KIO-104 zur Behandlung von makulärem Ödem der Netzhaut. Die Studie wird ein lokal verabreichtes kleines Molekül als Alternative zu Steroiden oder systemischen entzündungshemmenden Arzneimitteln bewerten.
Die multizentrische, offene Studie umfasst bis zu 28 Patienten und wird in zwei Teilen durchgeführt: Teil A (Dosisoptimierung) bewertet Sicherheit und Wirksamkeit von drei Injektionen alle zwei Wochen an 8 Probanden in zwei Kohorten (Dosen von 3,5 μg und 10 μg), während Teil B (Kohortenerweiterung) verschiedene Dosierungsschemata in Abständen von 2 oder 4 Wochen untersucht.
KIO-104 wirkt, indem es die Anzahl und Funktion von T-Zellen im Auge durch DHODH-Inhibition unterdrückt und damit entzündungsbedingte Sehkrankheiten wie diabetische Retinopathie, nicht-infektiöse Uveitis, Netzhautzellenverschluss und post-kataraktische Operationen anvisiert.
- Received regulatory approval for Phase 2 clinical trial
- Novel treatment approach targeting an established therapeutic pathway (DHODH inhibition)
- Potential alternative to current treatments with known limitations
- Small trial size of only 28 patients
- Open-label study design (not double-blind)
- Multiple competing indications may complicate efficacy assessment
Insights
The regulatory approval for KLARITY Phase 2 trial represents a significant milestone for Kiora Pharmaceuticals' ophthalmology program. The trial's design is particularly noteworthy for several reasons:
The study targets multiple high-value indications including diabetic retinopathy, uveitis and retinal vein occlusion - a strategic approach that could expedite market penetration across various retinal conditions. The dual-cohort design with dose optimization followed by expansion is efficiently structured to establish both optimal dosing and treatment intervals.
KIO-104's mechanism as a DHODH inhibitor is particularly compelling. This class has proven commercial success in autoimmune conditions, with drugs like teriflunomide generating substantial revenues. The local delivery approach could provide significant advantages over current standards of care:
- Reduced systemic exposure compared to oral medications
- Potentially better safety profile than steroids, which can cause cataracts and increased intraocular pressure
- Convenient administration through standard intravitreal injection
The market opportunity is substantial. Retinal diseases represent a multi-billion dollar market, with macular edema being a common pathway across multiple conditions. If successful, KIO-104 could capture significant market share by offering a novel, steroid-sparing alternative that addresses current treatment limitations.
While early stage, the trial's comprehensive endpoints examining safety, efficacy and pharmacokinetics will provide important data for future development and potential partnering discussions. The focus on both anatomical (edema reduction) and functional (visual acuity) outcomes aligns with regulatory requirements and clinical practice needs.
Encinitas, California--(Newsfile Corp. - February 11, 2025) - Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX) ("Kiora" or the "Company") today announced it received regulatory approval to initiate KLARITY, a Phase 2 clinical trial to investigate KIO-104 in patients with retinal macular edema, a condition where build-up of fluid behind part of the retina can be associated with adverse vision changes. KIO-104 is a potent, locally delivered small molecule being developed as an alternative to steroids or systemic anti-inflammatory drugs, both of which have known shortcomings.
KLARITY is a multi-center, open label study in up to 28 patients. The primary objective is to evaluate safety and tolerability of repeated doses of KIO-104 administered by standard intravitreal injection. Secondary endpoints will evaluate the magnitude of macula edema reduction, improvement in visual acuity and the systemic pharmacokinetic profile of KIO-104. The study will enroll patients with macular edema secondary to one of the following diseases: diabetic retinopathy, non-infectious uveitis, retinal vein occlusion, or post pseudophakic cataract surgery. The study will be performed in two parts as follows:
- Part A (Dose Optimization) will assess the safety and efficacy of three injections administered once every two weeks in 8 subjects. Cohort 1 of will receive 3.5 μg doses of KIO-104 while Cohort 2 will receive 10 μg doses of KIO-104.
- Part B (Cohort Expansion) will investigate different dosing regimens (2-week versus 4-week intervals) in the remaining subjects at a dose selected from Part A.
"Our aim is to further demonstrate the therapeutic potential of KIO-104. The need for a local, steroid-sparing approach to treating conditions associated with retinal inflammation, including macular edema, remains at the forefront of clinical retinal research. We believe KIO-104 can fulfill that role and provide meaningful benefit to patients suffering from reduced vision due to macular edema," said Eric J. Daniels, MD, MBA, Chief Development Officer at Kiora. "Our drug is differentiated by its ease of delivery and the clinically validated immunomodulatory pathway upon which it acts. The class of compounds, DHODH inhibitors, are disease modifying, clinically effective and commercially successful as systemic treatments for multiple sclerosis and rheumatoid arthritis. We are now looking to expand the potential of this therapeutic class for use in inflammatory conditions of the eye."
KIO-104 works by suppressing T cell numbers and function in the eye responsible for driving inflammation. Specifically, KIO-104 inhibits the mitochondrial enzyme, DHODH, which plays a crucial role in the synthesis of key building blocks of DNA and RNA. Through this nucleotide starvation, T cell replication is significantly impaired. In addition, these building blocks serve as key co-factors required for multiple T cell functions, including producing pro-inflammatory cytokines.
About Kiora Pharmaceuticals
Kiora Pharmaceuticals is a clinical-stage biotechnology company developing advanced therapies for retinal disease. We target critical pathways underlying retinal diseases using innovative small molecules to slow, stop, or restore vision loss. 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 (DHODH).
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 X 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, the ability to timely complete planned initiatives for 2024, including phase 2 clinical development of KIO-301 and KIO-104, the potential for KIO-301 to be the first treatment options for patients with inherited degenerative diseases like RP, Kiora's plans to further fund development of KIO-104, the potential for KIO-104 to reduce inflammation, the timing of topline results from clinical trials of KIO-104, the potential for KIO-104 to apply to other retinal inflammatory diseases, the anticipated readout dates for Kiora's clinical trials and their likelihood of success, and expected trends for research and development and general and administrative spending in 2024. These statements involve risks and uncertainties that may cause results to differ materially from the statements set forth in this press release 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 November 8, 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.
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Contact:
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