Opus Genetics Receives FDA Agreement Under Special Protocol Assessment for Phase 3 Trial of APX3330 in Diabetic Retinopathy
Opus Genetics (Nasdaq: IRD) has secured FDA agreement under Special Protocol Assessment (SPA) for its Phase 3 clinical trial of oral APX3330 in treating moderate to severe non-proliferative diabetic retinopathy (NPDR). The agreement confirms that the trial design and endpoints will support a New Drug Application submission.
The primary endpoint focuses on reducing 3-step or greater worsening on the binocular diabetic retinopathy severity scale compared to placebo. Previous Phase 2 ZETA-1 trial showed APX3330's potential to slow DR progression with a favorable safety profile. The company plans to seek partners for further development while focusing on gene therapy candidates for inherited retinal diseases.
Diabetic retinopathy affects approximately 10 million patients in the US and is the leading cause of blindness in working-age adults.
Opus Genetics (Nasdaq: IRD) ha ottenuto l'approvazione della FDA tramite Special Protocol Assessment (SPA) per il suo trial clinico di fase 3 dell'oral APX3330 nel trattamento della retinopatia diabetica non proliferativa da moderata a grave (NPDR). L'accordo conferma che il design dello studio e i suoi obiettivi supportano la presentazione di una domanda di nuovo farmaco.
L'obiettivo primario si concentra sulla riduzione del peggioramento di 3 livelli o più sulla scala di gravità della retinopatia diabetica binoculare rispetto al placebo. Il precedente trial di fase 2 ZETA-1 ha mostrato il potenziale di APX3330 di rallentare la progressione della retinopatia diabetica con un profilo di sicurezza favorevole. L'azienda intende cercare partner per ulteriori sviluppi, concentrandosi su candidati per la terapia genica per malattie retiniche ereditarie.
La retinopatia diabetica colpisce circa 10 milioni di pazienti negli Stati Uniti ed è la principale causa di cecità negli adulti in età lavorativa.
Opus Genetics (Nasdaq: IRD) ha obtenido la aprobación de la FDA bajo una Evaluación de Protocolo Especial (SPA) para su ensayo clínico de fase 3 del APX3330 oral en el tratamiento de la retinopatía diabética no proliferativa de moderada a grave (NPDR). El acuerdo confirma que el diseño del ensayo y los objetivos apoyarán la presentación de una Solicitud de Nuevo Medicamento.
El objetivo primario se centra en reducir el empeoramiento de 3 pasos o más en la escala de gravedad de la retinopatía diabética binocular en comparación con el placebo. El ensayo anterior de fase 2 ZETA-1 mostró el potencial de APX3330 para ralentizar la progresión de la RD con un perfil de seguridad favorable. La empresa planea buscar socios para el desarrollo futuro mientras se enfoca en candidatos para terapias génicas para enfermedades retinianas hereditarias.
La retinopatía diabética afecta a aproximadamente 10 millones de pacientes en los EE. UU. y es la principal causa de ceguera en adultos en edad laboral.
오퍼스 제네틱스 (Nasdaq: IRD)는 경구용 APX3330를 사용하여 중등도에서 중증 비증식성 당뇨병성 망막병증(NPDR)을 치료하기 위한 3상 임상시험에 대해 특별 프로토콜 평가(SPA)에 따른 FDA의 동의를 확보했습니다. 이 동의는 시험 설계와 최종점이 신약 신청 제출을 지원한다는 것을 확인합니다.
주요 최종점은 위약과 비교해 양안 당뇨병성 망막병증 중증도 척도에서 3단계 이상의 악화를 줄이는 데 중점을 둡니다. 이전 2상 시험 ZETA-1에서는 APX3330의 DR 진행속도를 늦추는 잠재력이 긍정적인 안전성 프로필과 함께 보여졌습니다. 회사는 유전적 망막질환 치료 후보에 초점을 맞추면서 추가 개발을 위한 파트너를 찾을 계획입니다.
당뇨병성 망막병증은 미국에서 약 1천만 명의 환자에게 영향을 미치며, 경제 활동을 하는 성인에서 실명의 주요 원인입니다.
Opus Genetics (Nasdaq: IRD) a obtenu l'accord de la FDA par le biais de l'Évaluation de Protocole Spécial (SPA) pour son essai clinique de phase 3 du APX3330 oral dans le traitement de la rétinopathie diabétique non proliférative modérée à sévère (NPDR). Cet accord confirme que le design de l'essai et les résultats attendus soutiennent la soumission d'une demande de nouveau médicament.
L'objectif principal vise à réduire l'aggravation de 3 étapes ou plus sur l'échelle de gravité de la rétinopathie diabétique binoculaire par rapport au placebo. Le précédent essai de phase 2 ZETA-1 a montré le potentiel de l'APX3330 à ralentir la progression de la RD avec un profil de sécurité favorable. L'entreprise prévoit de rechercher des partenaires pour le développement futur tout en se concentrant sur des candidats pour des thérapies géniques destinées aux maladies héréditaires de la rétine.
La rétinopathie diabétique touche environ 10 millions de patients aux États-Unis et constitue la principale cause de cécité chez les adultes en âge de travailler.
Opus Genetics (Nasdaq: IRD) hat die Zustimmung der FDA im Rahmen der Special Protocol Assessment (SPA) für seine Phase-3-Studie mit oralem APX3330 zur Behandlung von moderater bis schwerer nicht-proliferativer diabetischer Retinopathie (NPDR) erhalten. Die Vereinbarung bestätigt, dass das Studiendesign und die Endpunkte die Einreichung eines Antrags auf Zulassung eines neuen Arzneimittels unterstützen.
Der primäre Endpunkt konzentriert sich auf die Reduzierung einer Verschlechterung um 3 Stufen oder mehr auf der Binokularen Skala der Schwere der diabetischen Retinopathie im Vergleich zu Placebo. Die vorherige Phase-2-Studie ZETA-1 zeigte das Potenzial von APX3330, das Fortschreiten der DR mit einem günstigen Sicherheitsprofil zu verlangsamen. Das Unternehmen plant, Partner für die weitere Entwicklung zu suchen, während der Fokus auf Gentherapiekandidaten für erbliche Netzhauterkrankungen gerichtet ist.
Diabetische Retinopathie betrifft ungefähr 10 Millionen Patienten in den USA und ist die Hauptursache für Erblindung bei erwerbsfähigen Erwachsenen.
- FDA agreement on SPA for Phase 3 trial design reduces regulatory risk
- Previous Phase 2 trial demonstrated favorable safety profile
- Large market opportunity with 10 million potential patients in US
- Treatment addresses leading cause of blindness in working-age adults
- Company seeking external partner for further development due to resource constraints
- Shifting focus away from APX3330 to gene therapy candidates
Insights
The FDA's Special Protocol Assessment agreement for APX3330's Phase 3 trial represents a significant regulatory milestone that substantially de-risks the development pathway. The agreed-upon primary endpoint - reduction in 3-step worsening on the DRSS score - aligns with established clinical standards and previous successful diabetic retinopathy drug approvals. The 10 million patient US market opportunity for diabetic retinopathy presents substantial commercial potential.
The company's strategy to seek a development partner for APX3330 while focusing on their gene therapy pipeline is pragmatic given their market cap of
For investors, this positions Opus for a potential value-creating partnership deal while reducing regulatory uncertainty. However, the small market cap suggests significant execution risks remain.
APX3330's oral administration represents a potentially disruptive approach in diabetic retinopathy treatment. Current standard therapies like anti-VEGF injections require frequent intravitreal administration, creating significant treatment burden. An oral option that could prevent disease progression would be transformative for both patients and clinicians.
The focus on moderate to severe non-proliferative diabetic retinopathy (NPDR) is strategically sound - this represents the critical window where intervention can prevent progression to vision-threatening complications. The Phase 2 data suggesting APX3330 can slow or prevent DR progression, combined with a favorable safety profile, indicates promising clinical potential.
The binocular DRSS endpoint is particularly relevant as it captures real-world visual function better than single-eye measurements. This patient-centric approach could support strong market adoption if the Phase 3 trial succeeds.
Agreement Reached on Primary Endpoint and Phase 3 Trial Design
Oral APX3330 is a Late-Stage Clinical Asset Available for Partnering
FARMINGTON HILLS, Mich., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage ophthalmic biotechnology company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and small-molecule drugs to treat other ophthalmologic disorders, today announced that it has reached agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA) for a Phase 3 clinical trial evaluating oral APX3330 for the treatment of moderate to severe non-proliferative diabetic retinopathy (NPDR).
The SPA agreement reflects that the proposed Phase 3 trial design, endpoints, and planned analyses will be adequate to support a New Drug Application (NDA) submission for treatment of NPDR, subject to a successful outcome of the trial and review of all data in the NDA. The agreed primary endpoint is a reduction in 3-step or greater worsening on the binocular diabetic retinopathy severity scale (DRSS) score, compared to placebo. In the previous Phase 2 ZETA-1 trial, oral APX3330 showed the potential to slow or prevent clinically meaningful progression of DR and demonstrated a favorable safety profile.
“This SPA agreement reflects our alignment with the FDA on the design of a Phase 3 trial for APX3330 and is a testament to the team’s developmental and regulatory acumen,” said George Magrath, M.D., Chief Executive Officer of Opus Genetics. "If successful in Phase 3 and subsequently approved, APX3330 has the potential to be a transformative treatment option for patients with NPDR. We believe that having this SPA in place will help de-risk certain regulatory aspects of this program. Our intention is to seek a partner for APX3330 to fund further development, as we focus our resources on advancing our gene therapy candidates for IRDs.”
Diabetic retinopathy is a progressive eye disease which results in vision impairment and blindness among adults with diabetes. It is the leading cause of blindness in working age adults and impacts approximately 10 million patients in the US.
About APX3330
APX3330 is a first-in-class, small molecule, oral inhibitor of the transcription factor regulator Ref-1 (reduction-oxidation effector factor-1). With a novel, multimodal mechanism of action, APX3330 modulates the downstream pathways regulated by Ref-1 – which involve angiogenesis (VEGF), oxidative stress (Nrf2) and inflammation (NFkB) – to restore homeostatic levels of angiogenenic, oxidative stress and inflammatory factors that are implicated across several ocular diseases, including DR, diabetic macular edema (DME), and age-related macular degeneration (AMD). APX3330 has shown a favorable tolerability profile in 12 clinical trials conducted in healthy, hepatitis, cancer, and diabetic subjects.
About Opus Genetics
Opus Genetics is a clinical-stage ophthalmic biotechnology company developing gene therapies to treat patients with inherited retinal diseases (IRDs) and other ophthalmologic disorders. The pipeline includes adeno-associated virus (AAV)-based investigational gene therapies that address mutations in genes that cause different forms of bestrophinopathy, Leber congenital amaurosis (LCA) and retinitis pigmentosa. The company’s most advanced gene therapy program is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein and is currently being evaluated in a Phase 1/2 open-label, dose-escalation trial, with encouraging early data. BEST1 investigational gene therapy is designed to address mutations in the BEST1 gene, which is associated with retinal degeneration; a Phase 1/2 study is expected to be initiated in 2025. The pipeline also includes Phentolamine Ophthalmic Solution
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements concerning expectations regarding the potential of APX3330 to be a treatment option for patients with DR and future development of APX3330 with a partner.
These forward-looking statements relate to us, our business prospects and our results of operations and are subject to certain risks and uncertainties posed by many factors and events that could cause our actual business, prospects and results of operations to differ materially from those anticipated by such forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those described under the heading “Risk Factors” included in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 and in our other filings with the U.S. Securities and Exchange Commission. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. In some cases, you can identify forward-looking statements by the following words: “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “aim,” “may,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. We undertake no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise.
These forward-looking statements are based upon our current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including, without limitation:
- Our ability to successfully integrate the business of former Opus Genetics Inc. and manage our expanded combined product pipeline;
- Our ability to develop and obtain regulatory approval for newly acquired gene therapies to treat inherited retinal diseases;
- Our ability to obtain and maintain orphan drug designation or rare pediatric disease designation for our current and future product candidates;
- The success and timing of regulatory submissions and pre-clinical and clinical trials, including enrollment and data readouts;
- Regulatory requirements or developments;
- Changes to or unanticipated events in connection with clinical trial designs and regulatory pathways;
- Delays or difficulties in the enrollment of patients in clinical trials;
- Substantial competition;
- Rapid technological change;
- Our development of sales and marketing infrastructure;
- Future revenue losses and profitability;
- Changes in capital resource requirements;
- Risks related to our inability to obtain sufficient additional capital to continue to advance our product candidates and our preclinical programs;
- Domestic and worldwide legislative, regulatory, political and economic developments;
- Our dependency on key personnel;
- Changes in market opportunities and acceptance;
- Reliance on third parties to conduct our clinical trials and supply and manufacture drug supplies;
- Future, potential product liability and securities litigation;
- System failures, unplanned events, or cyber incidents;
- The substantial number of shares subject to potential issuance associated with our equity line of credit arrangement;
- Risks that our licensing or partnership arrangements may not facilitate the commercialization or market acceptance of our product candidates;
- Future fluctuations in the market price of our common stock;
- The success and timing of commercialization of any of our product candidates;
- Obtaining and maintaining our intellectual property rights; and
- The success of mergers and acquisitions.
The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive. Readers are urged to carefully review and consider the various disclosures made by us in this report and in our other reports filed with the Securities and Exchange Commission that advise interested parties of the risks and factors that may affect our business. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements concerning expectations regarding our cash runway, data from and future enrollment for our clinical trials, our pipeline of additional indications, expectations of potential growth, and our expectations regarding integration following the acquisition of Opus Genetics, including with respect to the combination of their portfolio of clinical assets into our existing portfolio and our combined focus on gene therapy treatment.
These forward-looking statements relate to us, our business prospects and our results of operations and are subject to certain risks and uncertainties posed by many factors and events that could cause our actual business, prospects and results of operations to differ materially from those anticipated by such forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those described under the heading “Risk Factors” included in Ocuphire’s Annual Report on Form 10-K. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this report. In some cases, you can identify forward-looking statements by the following words: “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “aim,” “may,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. We undertake no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise.
These forward-looking statements are based upon our current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including, without limitation:
- The success and timing of regulatory submissions and pre-clinical and clinical trials, including enrollment and data readouts;
- Regulatory requirements or developments;
- Changes to or unanticipated events in connection with clinical trial designs and regulatory pathways;
- Delays or difficulties in the enrollment of patients in clinical trials;
- Substantial competition and rapid technological change;
- Our development of sales and marketing infrastructure;
- Future revenue losses and profitability;
- Our relatively short operating history;
- Changes in capital resource requirements;
- Risks related to our inability to obtain sufficient additional capital to continue to advance our product candidates and our preclinical programs;
- Domestic and worldwide legislative, regulatory, political and economic developments;
- Employee misconduct;
- Reliance on third parties;
- Future, potential product liability and securities litigation;
- System failures, unplanned events, or cyber incidents;
- The substantial number of shares subject to potential issuance associated with our equity line of credit arrangement;
- Risks that our partnership or other licensing arrangements, may not facilitate the commercialization or market acceptance of our product candidates;
- Future fluctuations in the market price of our common stock;
- Our ability to realize the expected benefits of the acquisition of Opus Genetics;
- Our ability to execute clinical programs for gene therapies successfully and changes in expected commercial value we predict from the development of gene therapies;
- The success and timing of commercialization of any of our product candidates; and
- Obtaining and maintaining our intellectual property rights.
The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive. Readers are urged to carefully review and consider the various disclosures made by us in this report and in our other reports filed with the Securities and Exchange Commission that advise interested parties of the risks and factors that may affect our business. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.
Contacts
Corporate | Investor Relations |
Nirav Jhaveri CFO ir@opusgtx.com | Corey Davis, Ph.D. LifeSci Advisors cdavis@lifesciadvisors.com |
FAQ
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