Disc Medicine Announces Successful Type C Meeting with FDA for Bitopertin in Erythropoietic Protoporphyria (EPP) and Shares Plans for NDA Submission
Disc Medicine (NASDAQ:IRON) has received positive feedback from its Type C meeting with the FDA regarding bitopertin for Erythropoietic Protoporphyria (EPP) treatment. The company plans to submit a New Drug Application (NDA) in H2 2025 under the accelerated approval pathway, using protoporphyrin IX (PPIX) reduction as the surrogate endpoint.
The FDA meeting resulted in alignment on the APOLLO post-marketing confirmatory trial design, set to begin by mid-2025. The trial will feature co-primary endpoints: average monthly time in sunlight without pain and percent change in whole blood metal-free PPIX after 6 months of treatment. The study will be double-blind, placebo-controlled with approximately 150 patients aged 12+ randomized 1:1, including sites in the US, Canada, Europe, and Australia.
Disc Medicine (NASDAQ:IRON) ha ricevuto un feedback positivo dalla sua riunione di tipo C con la FDA riguardante il bitopertin per il trattamento dell'Eritropoietica Protoporfiria (EPP). L'azienda prevede di presentare una Nuova Richiesta di Medicinali (NDA) nel secondo semestre del 2025 attraverso la via di approvazione accelerata, utilizzando la riduzione della protoporfirina IX (PPIX) come endpoint surrogato.
La riunione con la FDA ha portato a un allineamento sul design del trial di conferma post-marketing APOLLO, che dovrebbe iniziare entro la metà del 2025. Il trial avrà come co-endpoint primari: il tempo medio mensile trascorso al sole senza dolore e la variazione percentuale della PPIX priva di metallo nel sangue intero dopo 6 mesi di trattamento. Lo studio sarà in doppio cieco, controllato con placebo, con circa 150 pazienti di età superiore ai 12 anni randomizzati 1:1, inclusi siti negli Stati Uniti, Canada, Europa e Australia.
Disc Medicine (NASDAQ:IRON) ha recibido comentarios positivos de su reunión de tipo C con la FDA sobre el bitopertin para el tratamiento de la Protoporfiria Eritropoética (EPP). La compañía planea presentar una Solicitud de Nuevo Medicamento (NDA) en la segunda mitad de 2025 bajo la vía de aprobación acelerada, utilizando la reducción de protoporfirina IX (PPIX) como un punto final sustituto.
La reunión con la FDA resultó en un acuerdo sobre el diseño del ensayo de confirmación post-comercialización APOLLO, que comenzará a mediados de 2025. El ensayo tendrá como puntos finales co-principales: el tiempo medio mensual al sol sin dolor y el cambio porcentual en la PPIX libre de metales en sangre completa después de 6 meses de tratamiento. El estudio será doble ciego, controlado con placebo, con aproximadamente 150 pacientes de 12 años o más aleatorizados 1:1, incluidos sitios en EE. UU., Canadá, Europa y Australia.
Disc Medicine (NASDAQ:IRON)는 FDA와의 C형 회의에서 Erythropoietic Protoporphyria (EPP) 치료를 위한 bitopertin에 대해 긍정적인 피드백을 받았습니다. 회사는 프로토포르피린 IX (PPIX) 감소를 대체 최종점으로 사용하여 2025년 하반기에 가속 승인 경로를 통해 새로운 의약품 신청(NDA)을 제출할 계획입니다.
FDA 회의는 2025년 중반부터 시작될 APOLLO 사후 마케팅 확인 시험 디자인에 관한 합의로 이어졌습니다. 이 시험은 두 가지 주요 최종점을 갖게 되며: 통증 없이 햇빛에 노출된 평균 월간 시간과 치료 6개월 후 전체 혈액 내 메탈 프리 PPIX의 변화 비율입니다. 이 연구는 더블 블라인드, 플라세보 대조 연구로 약 150명의 12세 이상의 환자가 1:1로 무작위 배정되며, 미국, 캐나다, 유럽 및 호주에 있는 사이트를 포함합니다.
Disc Medicine (NASDAQ:IRON) a reçu des retours positifs lors de sa réunion de type C avec la FDA concernant le bitopertin pour le traitement de la Protoporphyrie Érythropoétique (EPP). La société prévoit de soumettre une Demande de Nouveau Médicament (NDA) au second semestre 2025 selon le processus d'approbation accélérée, en utilisant la réduction de la protoporphyrine IX (PPIX) comme point de terminaison substitut.
La réunion avec la FDA a abouti à un accord sur le design de l'essai de confirmation post-commercialisation APOLLO, qui devrait débuter d'ici mi-2025. L'essai comportera deux points principaux : le temps moyen passé au soleil sans douleur par mois et le changement en pourcentage de la PPIX libre de métaux dans le sang total après 6 mois de traitement. L'étude sera en double aveugle, contrôlée par placebo, avec environ 150 patients âgés de 12 ans et plus randomisés dans un rapport de 1:1, comprenant des sites aux États-Unis, au Canada, en Europe et en Australie.
Disc Medicine (NASDAQ:IRON) hat positives Feedback aus seinem Typ-C-Meeting mit der FDA hinsichtlich Bitopertin zur Behandlung der Erythropoetischen Protoporphyrie (EPP) erhalten. Das Unternehmen plant, im 2. Halbjahr 2025 einen Antrag auf Zulassung neuer Arzneimittel (NDA) über den beschleunigten Zulassungsweg einzureichen, wobei der Rückgang von Protoporphyrin IX (PPIX) als surrogate Endpunkt verwendet wird.
Die FDA-Sitzung führte zu einer Einigung über das Design der APOLLO-Studie zur post-marketing Bestätigung, die Mitte 2025 beginnen soll. Die Studie wird zwei primäre Endpunkte haben: die durchschnittliche monatliche Zeit im Sonnenlicht ohne Schmerzen und die prozentuale Veränderung von metal-freiem PPIX im Vollblut nach 6 Monaten Behandlung. Die Studie wird doppelblind und placebokontrolliert sein, mit insgesamt etwa 150 Patienten ab 12 Jahren, die im Verhältnis 1:1 randomisiert werden, unter Einschluss von Standorten in den USA, Kanada, Europa und Australien.
- FDA alignment achieved on PPIX reduction as surrogate endpoint for accelerated approval pathway
- Clear regulatory pathway established with NDA submission planned for H2 2025
- Agreement reached on APOLLO confirmatory trial design with FDA
- Global trial footprint across multiple regions (US, Canada, Europe, Australia)
- Full approval dependent on successful completion of post-marketing confirmatory trial
- Extended timeline with NDA submission not until H2 2025
Insights
The FDA's alignment on bitopertin's accelerated approval pathway represents a significant regulatory milestone for Disc Medicine. The agency's acceptance of protoporphyrin IX reduction as a surrogate endpoint streamlines the approval process substantially. The planned NDA submission in H2 2025 with concurrent initiation of the APOLLO confirmatory trial demonstrates an optimized regulatory strategy.
The APOLLO trial design shows sophisticated planning with dual co-primary endpoints that address both biochemical markers (PPIX reduction) and clinically meaningful outcomes (sunlight tolerance). The 150-patient randomized controlled trial is well-powered and appropriately designed to support potential conversion from accelerated to full approval. The inclusion of patients aged 12+ broadens the potential market while the global trial sites enhance enrollment feasibility.
The trial design for APOLLO demonstrates robust clinical methodology. The co-primary endpoints are particularly well-chosen - the average monthly time in sunlight without pain provides a direct measure of patient benefit, while PPIX reduction validates the mechanism of action. The 60 mg dosing selection and 6-month treatment duration are based on previous Phase 2 data, suggesting confidence in the therapeutic window.
The inclusion of secondary endpoints like phototoxic reactions, PGIC and time to prodrome will build a comprehensive efficacy profile. For investors, the alignment on trial design significantly de-risks the regulatory pathway. The strategy of initiating the confirmatory trial before potential accelerated approval demonstrates confidence in the drug's efficacy and could accelerate time to full approval.
This regulatory progress significantly enhances Disc Medicine's market position in the EPP space. With no FDA-approved treatments currently available for EPP, bitopertin could capture a valuable orphan drug market. The accelerated approval pathway could bring the treatment to market faster, providing first-mover advantage. The global trial footprint across US, Canada, Europe and Australia suggests a comprehensive commercialization strategy.
For investors, this news reduces regulatory uncertainty and provides a clear timeline to potential commercialization. The
- Pursuing accelerated approval for bitopertin in EPP with protoporphyrin IX (PPIX) reduction as the surrogate endpoint
- Planning to submit NDA under accelerated approval pathway in H2 2025 based on existing clinical data, including results from BEACON and AURORA Phase 2 trials
- Achieved regulatory alignment on APOLLO post-marketing confirmatory trial design and on track to initiate trial by mid-year 2025
- Aligned on average monthly time in light without pain during the last month of the 6-month treatment period and percent change from baseline in whole-blood metal-free PPIX after 6 months of treatment as coprimary endpoints for confirmatory trial
- Management will host a conference call on Tuesday, January 21 at 8:00 am EST.
WATERTOWN, Mass., Jan. 21, 2025 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today announced positive feedback from its Type C meeting with the U.S. Food and Drug Administration (FDA) to discuss the APOLLO post-marketing confirmatory trial for bitopertin in EPP.
“Our recent FDA interaction marks another step toward delivering a potentially life-altering therapy for EPP patients, and we appreciate the collaboration with regulators, our investigators, and the EPP patient community which has brought us to this point,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc. “Last year, we aligned with the FDA on PPIX reduction as a surrogate endpoint for potential accelerated approval of bitopertin, and we are actively pursuing that path with plans to submit an NDA in the second half of 2025. As part of that process, the Type C meeting has provided further clarity on our plans for the APOLLO post-marketing confirmatory trial, which will kick off by the middle of this year and could eventually be the basis for converting an accelerated approval, if granted, to a full approval.”
The meeting resulted in alignment on the design of the APOLLO post-marketing confirmatory trial. Key features include:
- Co-primary endpoints of average monthly total time in sunlight without pain between 10:00 and 18:00 during the last month of the 6-month treatment period and percent change from baseline in whole blood metal-free PPIX after 6 months of treatment;
- Other measures of efficacy such as occurrence of phototoxic reactions, cumulative total pain-free time in sunlight, patient global impression of change (PGIC), and time to prodrome;
- Selection of 60 mg dose of bitopertin and 6-month treatment duration;
- Inclusion of patients aged 12+ with EPP including X-linked protoporphyria (XLP); and
- Double-blind, placebo-controlled study with ~150 patients randomized 1:1.
Disc plans to initiate the APOLLO trial by mid-2025 and will include sites in the US, Canada, Europe, and Australia. Based on guidance toward an NDA submission in H2 2025, Disc expects enrollment of the APOLLO trial to be well underway by the time of an accelerated approval, if granted.
Management will host a call to discuss these updates on Tuesday, January 21 at 8:00 am EST. Please register for the event on the Events and Presentations page of Disc’s website (https://ir.discmedicine.com/).
About Bitopertin
Bitopertin is an investigational, clinical-stage, orally administered inhibitor of glycine transporter 1 (GlyT1) that is designed to modulate heme biosynthesis. GlyT1 is a membrane transporter expressed on developing red blood cells and is required to supply sufficient glycine for heme biosynthesis and support erythropoiesis. Disc is planning to develop bitopertin as a potential treatment for a range of hematologic diseases including erythropoietic porphyrias, where it has potential to be the first disease-modifying therapy. Bitopertin has been studied in multiple clinical trials in patients with EPP, including the Phase 2 open-label BEACON trial, the Phase 2 double-blind, placebo-controlled AURORA trial, and an open-label extension HELIOS trial.
Bitopertin is an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. Disc obtained global rights to bitopertin under a license agreement from Roche in May 2021.
About Erythropoietic Protoporphyria (EPP)
Erythropoietic protoporphyria (EPP), including X-linked Protoporphyria (XLP), is a rare, debilitating and potentially life-threatening disease caused by mutations that affect heme biosynthesis, resulting in the accumulation of a toxic, photoactive intermediate called protoporphyrin IX (PPIX). This causes severe reactions when patients are exposed to sunlight, characterized by excruciating pain, edema, burning sensations and potential blistering and disfigurement. PPIX also accumulates in the hepatobiliary system and can result in complications including gallstones, cholestasis, and liver damage in 20
About Disc Medicine
Disc Medicine is a clinical-stage biopharmaceutical company committed to discovering, developing, and commercializing novel treatments for patients who suffer from serious hematologic diseases. We are building a portfolio of innovative, potentially first-in-class therapeutic candidates that aim to address a wide spectrum of hematologic diseases by targeting fundamental biological pathways of red blood cell biology, specifically heme biosynthesis and iron homeostasis. For more information, please visit www.discmedicine.com.
Disc Cautionary Statement Regarding Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, express or implied statements regarding Disc’s expectations with respect to its potential APOLLO confirmatory clinical study of bitopertin in EPP and XLP patients, including the proposed study design, the anticipated timeline, and the results thereof; and the possible regulatory path forward for bitopertin in EPP, including whether the FDA will determine that the accelerated approval pathway continues to be appropriate, the treatment of the APOLLO clinical study as a post-marketing confirmatory trial, and the timeline for a potential NDA submission and accelerated approval, if granted, and whether the NDA submission will meet the standards of accelerated approval. The use of words such as, but not limited to, “believe,” “expect,” “estimate,” “project,” “intend,” “future,” “potential,” “continue,” “may,” “might,” “plan,” “will,” “should,” “seek,” “anticipate,” or “could” or the negative of these terms and other similar words or expressions that are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Disc’s current beliefs, expectations and assumptions regarding the future of Disc’s business, future plans and strategies, clinical results and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.
Disc may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and investors should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of a number of material risks and uncertainties including but not limited to: the adequacy of Disc’s capital to support its future operations and its ability to successfully initiate and complete clinical trials; the nature, strategy and focus of Disc; the difficulty in predicting the time and cost of development of Disc’s product candidates; Disc’s plans to research, develop and commercialize its current and future product candidates; the timing of initiation of Disc’s planned preclinical studies and clinical trials; the timing of the availability of data from Disc’s clinical trials; Disc’s ability to identify additional product candidates with significant commercial potential and to expand its pipeline in hematological diseases; the timing and anticipated results of Disc’s preclinical studies and clinical trials and the risk that the results of Disc’s preclinical studies and clinical trials may not be predictive of future results in connection with future studies or clinical trials and may not support further development and marketing approval; and the other risks and uncertainties described in Disc’s filings with the Securities and Exchange Commission, including in the “Risk Factors” section of its Annual Report on Form 10-K for the year ended December 31, 2023, and in its Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2024. Any forward-looking statement speaks only as of the date on which it was made. None of Disc, nor its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law.
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