IDEAYA Biosciences Announces Positive Interim Phase 2 Data for Darovasertib and Successful FDA Type C Meeting on Registrational Trial Design for Regulatory Approval in Neoadjuvant Uveal Melanoma
IDEAYA Biosciences (Nasdaq:IDYA) announced positive interim Phase 2 data for darovasertib in neoadjuvant uveal melanoma (UM) and a successful FDA Type C meeting on registrational trial design. Key highlights include:
- ~49% of patients showed >30% tumor shrinkage
- ~61% eye preservation rate for enucleation patients
- Targeting Phase 3 randomized registrational trial initiation
- FDA guidance supports eye preservation rate and time to vision loss as primary endpoints
- Potential for broad indication label in neoadjuvant UM
- Projected ~400 patients for registrational trial enrollment
- Annual incidence of primary UM in North America, Europe, and Australia is ~12k patients
The company plans to advance darovasertib to a registrational trial in neoadjuvant UM, addressing a significant unmet medical need with no current FDA-approved therapies.
IDEAYA Biosciences (Nasdaq:IDYA) ha annunciato dati positivi interinali della Fase 2 per il darovasertib nel melanoma uveale (UM) neoadiuvante e un incontro di successo con la FDA di tipo C riguardo al design dello studio registrativo. I punti salienti includono:
- ~49% dei pazienti ha mostrato una riduzione del tumore superiore al 30%
- ~61% tasso di preservazione dell'occhio per i pazienti sottoposti a enucleazione
- Obiettivo di avviare la Fase 3 dello studio registrativo randomizzato
- Le linee guida della FDA supportano il tasso di preservazione dell'occhio e il tempo fino alla perdita della vista come endpoint primari
- Potenziale per un'etichetta di indicazione ampia nel melanoma uveale neoadiuvante
- Proiezione di ~400 pazienti per l'arruolamento nello studio registrativo
- L'incidenza annuale di melanoma uveale primario in Nord America, Europa e Australia è di circa 12.000 pazienti
L'azienda prevede di portare avanti il darovasertib verso uno studio registrativo nel melanoma uveale neoadiuvante, affrontando un significativo bisogno medico non soddisfatto, poiché non esistono attualmente terapie approvate dalla FDA.
IDEAYA Biosciences (Nasdaq:IDYA) anunció datos interinos positivos de la Fase 2 para el darovasertib en melanoma uveal (UM) neoadyuvante y una reunión exitosa de tipo C con la FDA sobre el diseño del ensayo registral. Los aspectos más destacados incluyen:
- ~49% de los pacientes mostraron una reducción tumoral superior al 30%
- ~61% tasa de preservación ocular para los pacientes sometidos a enucleación
- Objetivo de iniciar el ensayo registral aleatorio de Fase 3
- La guía de la FDA apoya la tasa de preservación ocular y el tiempo hasta la pérdida de visión como endpoints primarios
- Potencial para una etiqueta de indicación amplia en UM neoadyuvante
- Proyección de ~400 pacientes para la inscripción en el ensayo registral
- La incidencia anual de UM primario en América del Norte, Europa y Australia es de aproximadamente 12,000 pacientes
La empresa planea avanzar con el darovasertib a un ensayo registral en UM neoadyuvante, abordando una necesidad médica significativa no satisfecha, ya que actualmente no hay terapias aprobadas por la FDA.
IDEAYA Biosciences (Nasdaq:IDYA)는 선행 치료(neoadjuvant) 맥락에서의 유비질 멜라노마(UM)에 대한 다로바세르티브의 2상 임상시험 긍정적인 중간 결과와 함께 등록 임상시험 설계에 관한 성공적인 FDA 타입 C 회의를 발표했습니다. 주요 내용은 다음과 같습니다:
- ~49%의 환자가 30% 이상의 종양 축소를 보였습니다.
- 안구 절제 환자에 대한 ~61%의 안구 보존율
- 3상 무작위 등록 시험 시작 목표
- FDA의 가이드라인은 안구 보존율과 시력 상실까지의 시간을 주요 종단점으로 지원합니다.
- 선행 치료 UM에서의 넓은 적응증 가능성
- 등록 시험 등록을 위한 ~400명의 환자 예상
- 북미, 유럽 및 호주에서의 원발성 UM의 연간 발병률은 약 12,000명의 환자입니다.
회사는 다로바세르티브를 선행 치료 UM에 대한 등록 시험으로 진전시킬 계획이며, 현재 FDA 승인 치료제가 없는 중요한 unmet medical need를 해결하고자 합니다.
IDEAYA Biosciences (Nasdaq:IDYA) a annoncé des données intermédiaires positives de la phase 2 pour le darovasertib dans le mélanome uvéal (UM) néoadjuvant et une réunion réussie de type C avec la FDA concernant la conception de l'essai d'enregistrement. Les points saillants incluent :
- ~49 % des patients ont montré une réduction tumorale supérieure à 30 %
- ~61 % de taux de préservation de l'œil pour les patients soumis à une énucléation
- Objectif de lancement de l'essai d'enregistrement randomisé de la phase 3
- Les directives de la FDA soutiennent le taux de préservation de l'œil et le temps jusqu'à la perte de vision comme critères principaux
- Potentiel d'une étiquette d'indication large dans l'UM néoadjuvant
- Environ 400 patients projetés pour l'inscription à l'essai d'enregistrement
- L'incidence annuelle de l'UM primaire en Amérique du Nord, en Europe et en Australie est d'environ 12 000 patients
L'entreprise prévoit d'avancer le darovasertib vers un essai d'enregistrement dans l'UM néoadjuvant, répondant à un besoin médical majeur non satisfait, car il n'existe actuellement aucune thérapie approuvée par la FDA.
IDEAYA Biosciences (Nasdaq:IDYA) hat positive interim Ergebnisse der Phase 2 für Darovasertib bei neoadjuvantem uvealem Melanom (UM) bekannt gegeben und ein erfolgreiches FDA Type C-Meeting zum Design des Zulassungsversuchs abgehalten. Wichtige Highlights sind:
- ~49% der Patienten zeigten eine Tumorschrumpfung von >30%
- ~61% Erhaltungsrate der Augen bei enukleierten Patienten
- Ziel ist der Beginn eines randomisierten Zulassungsversuchs der Phase 3
- Die FDA-Richtlinien unterstützen die Erhaltungsquote der Augen und die Zeit bis zum Verlust der Sicht als primäre Endpunkte
- Möglichkeit einer breiten Indikationszulassung bei neoadjuvantem UM
- Geschätzte ~400 Patienten für die Einschreibung in den Zulassungsversuch
- Die jährliche Inzidenz von primärem UM in Nordamerika, Europa und Australien beträgt etwa 12.000 Patienten
Das Unternehmen plant, Darovasertib für einen Zulassungsversuch bei neoadjuvantem UM voranzubringen, um einen erheblichen ungedeckten medizinischen Bedarf zu decken, da es derzeit keine von der FDA genehmigten Therapien gibt.
- ~49% of patients demonstrated >30% tumor shrinkage in Phase 2 trials
- ~61% eye preservation rate observed for enucleation patients
- FDA guidance supports eye preservation rate and time to vision loss as primary endpoints
- Potential for broad indication label in neoadjuvant UM for low, intermediate, and high-risk patients
- Manageable adverse event profile with 11% grade 3 or higher AEs and 5% serious AE rate
- Darovasertib has FDA Fast Track designation for metastatic uveal melanoma
- Potential annual incidence of ~12,000 patients in North America, Europe, and Australia for neoadjuvant UM
- No FDA-approved therapies currently exist for primary uveal melanoma
- Registrational trial enrollment projected at ~400 patients, indicating a large and potentially costly study
- Initial readout for Event-Free-Survival (EFS) may take approximately 2 years of data maturity
- Phase 2 company-sponsored and IST neoadjuvant uveal melanoma (UM) clinical data update in 49 evaluable patients, demonstrates ~
49% of patients with >30% tumor shrinkage by product of diameters, and ~61% eye preservation rate for enucleation patients - Targeting to initiate Phase 3 randomized registrational trial in neoadjuvant UM following finalization of the clinical protocol with FDA
- Clinical endpoints supportive of full approval based on FDA guidance: Eye preservation rate as the primary endpoint for enucleation patients. Time to vision loss as the primary endpoint for plaque brachytherapy patients. No detriment to Event-Free-Survival (EFS) in the treatment arms is a secondary endpoint
- Discussions ongoing with FDA to include ORR as a potential surrogate and composite endpoint to support earlier approval scenarios
- Based on FDA meeting, potential for consideration of broad indication label in neoadjuvant UM for subjects with low, intermediate, and high risk for metastatic disease
- Pending FDA discussions, we project registrational trial enrollment will be ~400 patients
North America ,Europe , andAustralia annual incidence of primary UM is projected to be ~12k patients where there are currently no FDA-approved therapies- Investor webcast with Company management and KOL scheduled for Mon Sept 23, at 8am ET
"Darovasertib has demonstrated compelling preliminary clinical efficacy and a favorable AE profile in the neoadjuvant UM setting, with approximately
"The successful FDA Type C meeting provides darovasertib a potential registrational path in neoadjuvant UM, using primary clinical endpoints of eye preservation and time to vision loss, with no detriment to EFS in the treatment arms as a secondary endpoint. Based on the highly promising preliminary clinical efficacy and manageable safety profile observed with darovasertib and the high unmet medical need of neoadjuvant UM, we are excited to advance darovasertib rapidly to a registrational trial in this indication," said Dr. Darrin Beaupre, M.D., Ph.D., Chief Medical Officer, IDEAYA Biosciences.
Darovasertib is a potent and selective protein kinase C (PKC) inhibitor being developed to broadly address primary and metastatic UM. Darovasertib is currently being evaluated in four ongoing clinical trials, two of which are in collaboration with Pfizer. The darovasertib and crizotinib combination in metastatic uveal melanoma (MUM) has FDA Fast Track designation.
IDE196-009 (NCT05907954) is a company-sponsored Phase 2 trial evaluating darovasertib as neoadjuvant treatment for UM prior to primary interventional treatment of enucleation or radiation therapy, and as adjuvant therapy following the primary treatment. In addition, there is an investigator-sponsored trial (IST), NCT05187884, evaluating darovasertib as a neoadjuvant UM treatment.
Phase 2 Clinical Data Update – Darovasertib in Neoadjuvant UM
The company observed further evidence of encouraging clinical activity from an ongoing company-sponsored Phase 2 trial (NCT05907954) evaluating darovasertib in neoadjuvant uveal melanoma. The data cut-off date is August 15th, 2024, with an enrollment cut-off of May 13th, 2024, for the Phase 2 company-sponsored trial. Collectively, these clinical efficacy data from the Phase 2 company-sponsored and IST further substantiate clinical proof of concept for the use of darovasertib in the neoadjuvant uveal melanoma setting.
Clinical data update highlights include:
- 31 enucleation and 18 plaque brachytherapy evaluable UM patients treated with darovasertib neoadjuvant therapy in Phase 2 company-sponsored and IST trials
- ~
59% (29 of 49) of patients with >20% ocular tumor shrinkage by product of diameters - ~
49% (24 of 49) of patients with >30% ocular tumor shrinkage by product of diameters - ~
61% (19 of 31) eye preservation rate observed - Evidence of visual preservation observed by reducing the amount of radiation associated with plaque brachytherapy
- Manageable AE profile observed from Phase 2 company-sponsored trial (n=38), including
11% grade 3 or higher AEs, and5% serious AE rate. The discontinuation rate observed was3% . The most common AEs observed included diarrhea, nausea, vomiting and fatigue.
IDEAYA's ocular oncology advisory board recommended product of diameters for tumor measurement to determine overall response rate (ORR) criteria in ocular melanoma. In IDEAYA's ongoing Phase 2 clinical study with darovasertib, a >
FDA Guidance in Type C Meeting supports Initiation of Potential Registrational Trial
IDEAYA is targeting to initiate a potential registration-enabling Phase 3 randomized clinical trial in neoadjuvant UM patients following finalization of the clinical protocol with the FDA. The randomized Phase 3 clinical trial design incorporates guidance and feedback from the
In the Phase 3 clinical trial, we currently project approximately 400 patients will be randomized for treatment with darovasertib in the treatment arm or the control arm, with potential modifications pending further FDA feedback. Based on the currently targeted clinical trial design, there will be 2 cohorts enrolled: 1) enucleation eligible UM patients, 2) plaque brachytherapy eligible UM patients. For the enucleation cohort, the randomization will be with or without darovasertib as neoadjuvant therapy. For the plaque brachytherapy cohort, the randomization will be darovasertib followed by plaque brachytherapy versus plaque brachytherapy alone.
Key highlights of the target registrational trial design in neoadjuvant UM, based on FDA guidance and information provided in IDEAYA's FDA briefing book:
- Eye preservation rate is the primary endpoint for enucleation UM patients. Time to vision loss is the primary endpoint for plaque brachytherapy UM patients
- No detriment to Event-Free-Survival (EFS) in the treatment arms is a secondary endpoint
- Discussions ongoing with FDA to include ORR as a potential surrogate and composite endpoint to support earlier approval scenarios. The FDA briefing book notes a >
20% ocular tumor shrinkage by product of diameters correlates to clinical benefit in the ongoing Phase 2 clinical study, including eye sparing for enucleation UM patients and visual preservation for plaque brachytherapy UM patients - Primary endpoint of % eye preservation rate: The FDA briefing book notes objective to exceed lower bound of
10% eye preservation rate with a95% confidence interval - The registrational study will enroll UM patients with high risk for metastatic disease. Based on our preliminary projections, we anticipate the no detriment to EFS in the treatment arms for this high-risk population will take approximately 2-years of data maturity to initial readout
- Based on FDA meeting, potential for consideration of broad indication label in neoadjuvant UM for subjects with low, intermediate and high risk for metastatic disease
- 300mg BID darovasertib noted in FDA briefing book as the move-forward dose for registrational trial
Addressable Patient Population in neoadjuvant UM
Neoadjuvant UM represents a significant expansion opportunity for darovasertib – with a potential annual incidence of approximately 12,000 patients aggregate in
IDEAYA owns or controls all commercial rights in darovasertib, including in MUM and in UM, subject to certain economic obligations pursuant to its exclusive, worldwide license with Novartis.
IDEAYA Investor Webcast and Conference Call
IDEAYA will host an investor webcast and conference call today, Monday, September 23, 2024 at 8:00 am ET, to present the darovasertib Phase 2 clinical efficacy and tolerability data, and the potential registrational clinical trial design based on guidance and feedback from the recent FDA Type C meeting.
Presenters at the investor webcast and conference call will include Dr. Carol L. Shields, M.D., Chief of the Ocular Oncology Service at Wills Eye Hospital and Professor of Ophthalmology at Thomas Jefferson University. IDEAYA management, Yujiro S. Hata, Chief Executive Officer and President of IDEAYA Biosciences, and Darrin Beaupre, M.D., Ph.D., Chief Medical Officer of IDEAYA Biosciences, will also serve as presenters.
IDEAYA's darovasertib investor webcast presentation, as well as an updated corporate presentation, will be available on the company's website, at its Investor Relations portal (https://ir.ideayabio.com/) in advance of the investor webcast presentation today, Monday, September 23, 2024, at approximately 8:00 am ET.
About IDEAYA Biosciences
IDEAYA is a precision medicine oncology company committed to the discovery and development of targeted therapeutics for patient populations selected using molecular diagnostics. IDEAYA's approach integrates capabilities in identifying and validating translational biomarkers with drug discovery to select patient populations most likely to benefit from its targeted therapies. IDEAYA is applying its research and drug discovery capabilities to synthetic lethality – which represents an emerging class of precision medicine targets.
IDEAYA's updated corporate presentation is available on its website, at its Investor Relations page: https://ir.ideayabio.com/.
Forward-Looking Statements
This press release contains forward-looking statements, including, but not limited to, statements related to (i) the timing of FDA regulatory guidance and a potential registration-enabling Phase 3 randomized clinical trial in neoadjuvant UM patients, (ii) the potential therapeutic benefits of IDEAYA therapeutics, (iii) the translation of preliminary clinical trial results into future clinical trial results, and (iv) the estimate of patient populations. Such forward-looking statements involve substantial risks and uncertainties that could cause IDEAYA's preclinical and clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the drug development process, including IDEAYA's programs' early stage of development, the process of designing and conducting preclinical and clinical trials, the regulatory approval processes, the timing of regulatory filings, the challenges associated with manufacturing drug products, IDEAYA's ability to successfully establish, protect and defend its intellectual property, and other matters that could affect the sufficiency of existing cash to fund operations. IDEAYA undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of IDEAYA in general, see IDEAYA's Annual Report on Form 10-K dated February 20, 2024 and any current and periodic reports filed with the
Investor and Media Contact
IDEAYA Biosciences
Andres Ruiz Briseno
SVP, Head of Finance and Investor Relations
investor@ideayabio.com
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FAQ
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