Aura Biosciences Reports Positive Phase 2 End of Study Results Evaluating Bel-sar as a First-Line Treatment for Early-Stage Choroidal Melanoma
Aura Biosciences (NASDAQ: AURA) reported positive Phase 2 end of study results for bel-sar (AU-011) in treating early-stage choroidal melanoma (CM). The study showed an 80% tumor control rate and 90% visual acuity preservation among Phase 3-eligible patients. Bel-sar demonstrated a highly favorable safety profile with no treatment-related serious adverse events. The drug achieved complete cessation of growth in responders, with a post-treatment average growth rate of 0.011 mm/yr compared to 0.351 mm/yr prior to study entry (p<0.0001). Notably, 80% of patients were at high risk for vision loss due to tumor proximity to critical eye structures. These results are significant given the poor prognosis associated with CM and the lack of approved vision-preserving therapies. Aura is currently enrolling patients in the global Phase 3 CoMpass trial for bel-sar.
Aura Biosciences (NASDAQ: AURA) ha riportato risultati positivi della fine dello studio di Fase 2 per bel-sar (AU-011) nel trattamento del melanoma coroideo (CM) in fase iniziale. Lo studio ha mostrato un tasso di controllo tumorale dell'80% e una preservazione dell'acuità visiva del 90% tra i pazienti idonei alla Fase 3. Bel-sar ha dimostrato un profilo di sicurezza altamente favorevole senza eventi avversi gravi correlati al trattamento. Il farmaco ha raggiunto una completa cessazione della crescita nei rispondenti, con un tasso medio di crescita post-trattamento di 0,011 mm/anno rispetto a 0,351 mm/anno prima dell'ingresso nello studio (p<0.0001). È importante notare che l'80% dei pazienti era ad alto rischio di perdita della vista a causa della vicinanza del tumore a strutture oculari critiche. Questi risultati sono significativi dato il scarso prognosi associata al CM e la mancanza di terapie approvate per la preservazione della vista. Aura sta attualmente arruolando pazienti nella trial globale CoMpass di Fase 3 per bel-sar.
Aura Biosciences (NASDAQ: AURA) informó resultados positivos de la fase 2 al final del estudio para bel-sar (AU-011) en el tratamiento del melanoma coroideo (CM) en etapas tempranas. El estudio mostró un tasa de control tumoral del 80% y una preservación de la agudeza visual del 90% entre los pacientes elegibles para la fase 3. Bel-sar demostró un perfil de seguridad altamente favorable sin eventos adversos graves relacionados con el tratamiento. El medicamento logró una completa cesación del crecimiento en los respondedores, con una tasa de crecimiento promedio posterior al tratamiento de 0.011 mm/año en comparación con 0.351 mm/año antes de la entrada al estudio (p<0.0001). Notablemente, el 80% de los pacientes estaban en alto riesgo de pérdida de visión debido a la proximidad del tumor a estructuras oculares críticas. Estos resultados son significativos dado el mal pronóstico asociado con el CM y la falta de terapias aprobadas que preserven la visión. Aura está actualmente inscribiendo pacientes en el ensayo global CoMpass de fase 3 para bel-sar.
Aura Biosciences (NASDAQ: AURA)는 초기 단계 맥락막 흑색종 (CM) 치료를 위한 bel-sar (AU-011) 연구의 2상 최종 결과를 발표했습니다. 이 연구는 3상 자격이 있는 환자 가운데 80%의 종양 조절률과 90%의 시력 보존률을 보였습니다. Bel-sar는 매우 유리한 안전성 프로파일을 보여주었으며, 치료와 관련된 심각한 부작용은 없었습니다. 이 약물은 반응자를 대상으로 성장이 완전히 중단되었습니다. 치료 후 평균 성장률은 0.011 mm/년으로 연구 참여 전 0.351 mm/년과 비교되었습니다 (p<0.0001). 특히, 80%의 환자가 치명적인 시각 손실 위험에 처해 있었습니다 한편, 이는 종양이 중요한 눈 구조에 가까이 위치해 있기 때문입니다. CM과 관련된 불리한 예후 및 승인된 시력 보존 치료의 부족을 고려할 때, 이러한 결과는 의미가 있습니다. Aura는 현재 bel-sar에 대한 글로벌 3상 CoMpass 시험에 환자를 모집하고 있습니다.
Aura Biosciences (NASDAQ: AURA) a rapporté des résultats positifs en fin d'étude de phase 2 pour bel-sar (AU-011) dans le traitement du mélanome choroïdien (CM) à un stade précoce. L'étude a montré un taux de contrôle tumoral de 80% et une préservation de l'acuité visuelle de 90% parmi les patients éligibles pour la phase 3. Bel-sar a démontré un profil de sécurité très favorable sans événements indésirables graves liés au traitement. Le médicament a permis une cessation complète de la croissance chez les répondants, avec un taux de croissance moyen post-traitement de 0,011 mm/an contre 0,351 mm/an avant l'entrée dans l'étude (p<0,0001). Il est à noter que 80% des patients étaient à haut risque de perte de vision en raison de la proximité de la tumeur par rapport aux structures oculaires critiques. Ces résultats sont significatifs compte tenu du mauvais pronostic associé au CM et du manque de thérapies approuvées pour préserver la vision. Aura recrute actuellement des patients pour l'essai mondial de phase 3 CoMpass pour bel-sar.
Aura Biosciences (NASDAQ: AURA) berichtete über positive Endergebnisse der Phase-2-Studie für bel-sar (AU-011) zur Behandlung von frühem choroidalem Melanom (CM). Die Studie zeigte eine Tumorkontrollrate von 80% und 90% Erhalt der Sehschärfe bei Phase-3-geeigneten Patienten. Bel-sar wies ein sehr günstiges Sicherheitsprofil auf, ohne behandlungsbedingte schwere Nebenwirkungen. Das Medikament erreichte bei den Ansprechern ein vollständiges Wachstumsstillstand, mit einer durchschnittlichen Wachstumsrate von 0,011 mm/Jahr nach der Behandlung im Vergleich zu 0,351 mm/Jahr vor Studienbeginn (p<0.0001). Bemerkenswert ist, dass 80% der Patienten ein hohes Risiko für einen Sehverlust hatten, da sich der Tumor in der Nähe wichtiger Augenstrukturen befand. Diese Ergebnisse sind bedeutend, angesichts der schlechten Prognose, die mit CM verbunden ist, und des Mangels an genehmigten therapies zur Erhaltung der Sehkraft. Aura rekrutiert derzeit Patienten für die globale Phase-3-CoMpass-Studie für bel-sar.
- 80% tumor control rate achieved in Phase 3-eligible patients
- 90% visual acuity preservation in treated patients
- Highly favorable safety profile with no treatment-related serious adverse events
- Complete cessation of tumor growth in responders (p<0.0001)
- Potential to become the first vision-sparing, first-line treatment for early-stage CM
- FDA agreement on Special Protocol Assessment for Phase 3 CoMpass trial design
- None.
Insights
The Phase 2 results for bel-sar in early-stage choroidal melanoma (CM) are highly promising. The 80% tumor control rate and 90% visual acuity preservation are significant improvements over current treatments. Notably, 80% of patients were at high risk for vision loss, yet still achieved these results. This is a major advancement, considering standard radiotherapy can lead to legal blindness in up to
The safety profile is particularly impressive, with no treatment-related serious adverse events. The mild ocular side effects were mostly self-resolving or easily treated. Importantly, there were no posterior inflammation events, which are often concerning in ocular treatments.
These results could potentially revolutionize CM treatment, offering a vision-sparing option for a disease that typically forces patients to choose between treating the cancer and preserving their sight. The FDA's Special Protocol Assessment agreement for the Phase 3 trial further validates the potential of this treatment.
Aura Biosciences' bel-sar shows strong potential as a first-in-class therapy for early-stage choroidal melanoma. The Phase 2 results are compelling, addressing an unmet need in a rare but serious cancer. The 80% tumor control rate and 90% visual acuity preservation in high-risk patients are particularly noteworthy.
The favorable safety profile and efficacy data position bel-sar well for the ongoing Phase 3 trial. The FDA's Special Protocol Assessment agreement is a significant regulatory milestone, potentially streamlining the path to market.
For investors, these results could signify a substantial market opportunity. With no currently approved vision-preserving therapies for CM, bel-sar could capture a significant share of this niche market. However, it's important to monitor the Phase 3 trial progress and any potential competitors in the pipeline. The company's focus on precision therapies for solid tumors could also open doors to broader applications in the future.
The Phase 2 trial design and execution for bel-sar appear robust. The study included both single and multiple ascending dose cohorts, with a total of 22 patients and a 12-month follow-up period. This approach allows for a comprehensive assessment of safety and efficacy.
The statistical significance (p<0.0001) in tumor growth rate reduction among responders is a strong indicator of efficacy. The 80% tumor control rate and 90% visual acuity preservation in Phase 3-eligible patients are impressive outcomes.
The ongoing global Phase 3 CoMpass trial, with FDA agreement under a Special Protocol Assessment, is a critical next step. This agreement suggests that if the trial meets its objectives, it could support a biologics license application. However, it's important to note that Phase 3 trials can sometimes yield different results from earlier phases. Investors should closely monitor patient enrollment, interim analyses and any protocol amendments in the Phase 3 trial.
Bel-sar Demonstrated
Aura to Host a Virtual Ocular Oncology Investor Event Featuring Key Opinion Leaders Today at 8:00 am ET
BOSTON, Sept. 12, 2024 (GLOBE NEWSWIRE) -- Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing precision therapies for solid tumors designed to preserve organ function, today announced positive Phase 2 end of study results evaluating bel-sar (AU-011) for the first-line treatment of early-stage choroidal melanoma (CM), a vision and life-threatening ocular cancer. The results were presented at The Retina Society Annual Meeting, on Thursday, September 12, 2024, in Lisbon, Portugal.
The Phase 2 study (NCT04417530) is an open-label, ascending single and repeat dose escalation trial in patients with early-stage CM (small CM and indeterminate lesions) designed to evaluate the safety, tolerability, and efficacy of up to three cycles of bel-sar treatment. The trial included both single and multiple ascending dose cohorts, with a total of 22 patients enrolled. Patients were closely monitored over a twelve-month follow-up period to assess tumor control, visual acuity preservation, and tumor growth rate.
Tumor Control and Visual Acuity Preservation
The Phase 2 results demonstrated that bel-sar achieved an
Highly Favorable Safety Profile with No Dose-Limiting Toxicities
The safety profile of bel-sar was highly favorable in all participants regardless of dose. There were no treatment-related serious adverse events (SAEs) reported. Ocular treatment-related AEs (TRAEs) were mild (Grade 1), included anterior chamber inflammation (
“Many patients with early-stage choroidal melanoma currently face the difficult choice of whether to treat the cancer and risk losing their vision in the treated eye, or delay treatment and risk the tumor progressing,” said Dr. Ivana Kim, Director of the Ocular Melanoma Center, Mass Eye and Ear / Harvard Medical School. “The Phase 2 end of study data that I presented at The Retina Society Annual Meeting showed
“We believe these Phase 2 results provide clinical evidence for bel-sar as a potential vision-sparing, first-line treatment option for patients with early-stage CM,” said Dr. Jill Hopkins, Chief Medical Officer and President of Research and Development at Aura Biosciences. “Bel-sar is potentially a first-in-class novel therapy and we are excited to continue to advance this program, which is currently enrolling patients in our ongoing global Phase 3 CoMpass trial.”
Aura received written agreement from the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) for the design and planned analysis of the global Phase 3 CoMpass trial indicating concurrence by the FDA with the adequacy of the study, if successful, to address the objectives necessary to support Aura’s planned biologics license application submission. Aura Biosciences is focused on enhancing treatment options and improving outcomes for patients with CM and other cancers.
Aura Virtual Ocular Oncology Investor Event
Aura will host a virtual ocular oncology investor event featuring Dr. Ivana Kim, MD (Mass Eye and Ear) and Dr. Prithvi Mruthyunjaya, MD, MHS (Stanford University Byers Eye Institute) to discuss the Phase 2 end of study data on Thursday, September 12, 2024, at 8:00 am Eastern Time. To register for the event, click here. A live question and answer session will follow the formal discussion.
The live webcast of Aura’s virtual ocular oncology investor event will be available on the “Investors & Media” page under the “Events & Presentations” section of Aura’s website at https://ir.aurabiosciences.com/events-and-presentations, where a replay of the webcast will be archived for 90 days following the presentation date.
About Aura Biosciences
Aura Biosciences is a clinical-stage biotechnology company focused on developing precision therapies for solid tumors that aim to preserve organ function. Our lead candidate, bel-sar (AU-011), is currently in late-stage development for primary choroidal melanoma, and in early-stage development in other ocular oncology indications and bladder cancer. Aura Biosciences is headquartered in Boston, MA. Our mission is to grow as an innovative global oncology company that positively transforms the lives of patients.
For more information, visit aurabiosciences.com. Follow us on X (formerly Twitter) @AuraBiosciences and visit us on LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements that are not statements of historical fact may be deemed to be forward looking statements. Words such as “may,” “will,” “could,” “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions that can be used to identify forward-looking statements. These forward-looking statements include express or implied statements regarding Aura’s future expectations, plans and prospects, including, without limitation, statements regarding the therapeutic potential of bel-sar for the treatment of cancers including early-stage CM and other oncology indications; statements regarding Aura’s expectations for the Phase 3 clinical trial of bel-sar for early-stage CM; statements regarding Aura’s expectations for an improved quality of life of patients after treatment with bel-sar; statements regarding Aura’s beliefs and expectations for the urgent need for an effective local treatment in ocular and other oncology indications to preserve organ function; statements regarding Aura’s expectations for the estimated patient populations and related market opportunities for bel-sar; and the potential for regulatory approval of bel-sar.
The forward-looking statements in this press release are neither promises nor guarantees, and investors should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties and other factors, many of which are beyond Aura’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements, including, without limitation, uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; the expected timing for submissions for regulatory approval or review by governmental authorities; the risk that the results of Aura’s preclinical and clinical trials may not be predictive of future results in connection with future clinical trials; the risk that interim data from ongoing clinical trials may not be predictive of final data from completed clinical trials; the risk that governmental authorities may disagree with Aura’s clinical trial designs, even where Aura has obtained agreement with governmental authorities on the design of such trials, such as the Phase 3 SPA agreement with the FDA; whether Aura will receive regulatory approvals to conduct trials or to market products; whether Aura’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; Aura’s ongoing and planned preclinical activities; and Aura’s ability to initiate, enroll, conduct or complete ongoing and planned clinical trials. These risks, uncertainties and other factors include those risks and uncertainties described under the heading “Risk Factors” in Aura’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the United States Securities and Exchange Commission (SEC) and in subsequent filings made by Aura with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Aura disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Aura’s current expectations and speak only as of the date hereof and no representations or warranties (express or implied) are made about the accuracy of any such forward-looking statements.
Investor Contact:
Alex Dasalla
Head of Investor Relations and Corporate Communications
IR@aurabiosciences.com
Media Contact:
Kimberly Ha
KKH Advisors
kimberly.ha@kkhadvisors.com
917-291-5744
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1 Jarczak J, Karska-Basta I, Romanowska-Dixon B. Deterioration of visual acuity after brachytherapy and proton therapy of uveal melanoma, and methods of counteracting this complication based on recent publications. Medicina (Kaunas). 2023;59(6):1131.
FAQ
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