Disc Medicine Announces Successful End of Phase 2 Meeting with FDA for Bitopertin in Erythropoietic Protoporphyria (EPP), Including Potential for Accelerated Approval
Disc Medicine (NASDAQ:IRON) announced positive feedback from its end-of-Phase 2 meeting with the FDA regarding bitopertin for Erythropoietic Protoporphyria (EPP) treatment. The FDA agreed with all proposed study parameters for the APOLLO trial, including a primary endpoint measuring average monthly sunlight time without pain after 6 months of treatment. The company received potential for accelerated approval using PPIX reduction as a surrogate endpoint. The APOLLO trial, planned for mid-2025, will be a randomized, double-blind, placebo-controlled study using a 60 mg dose of bitopertin in EPP and XLP patients aged 12 and older.
Disc Medicine (NASDAQ:IRON) ha annunciato un feedback positivo dall'incontro di fine Fase 2 con la FDA riguardo al bitopertin per il trattamento della Protoporfiria Eritropoietica (EPP). La FDA ha accettato tutti i parametri di studio proposti per il trial APOLLO, inclusi un endpoint primario che misura il tempo medio mensile di esposizione al sole senza dolore dopo 6 mesi di trattamento. L'azienda ha ricevuto la possibilità di approvazione accelerata utilizzando la riduzione di PPIX come endpoint surrogato. Il trial APOLLO, previsto per metà 2025, sarà uno studio randomizzato, in doppio cieco e controllato con placebo, utilizzando una dose di 60 mg di bitopertin in pazienti con EPP e XLP di età pari o superiore ai 12 anni.
Disc Medicine (NASDAQ:IRON) anunció comentarios positivos de su reunión de fin de Fase 2 con la FDA sobre el bitopertin para el tratamiento de la Protoporfiria Eritropoietica (EPP). La FDA estuvo de acuerdo con todos los parámetros de estudio propuestos para el ensayo APOLLO, incluyendo un resultado primario que mide el tiempo promedio mensual de exposición al sol sin dolor después de 6 meses de tratamiento. La compañía recibió la posibilidad de aprobación acelerada utilizando la reducción de PPIX como un endpoint de sustitución. El ensayo APOLLO, que está planeado para mediados de 2025, será un estudio aleatorizado, doble ciego y controlado con placebo que utilizará una dosis de 60 mg de bitopertin en pacientes con EPP y XLP de 12 años o más.
Disc Medicine (NASDAQ:IRON)는 Erythropoietic Protoporphyria (EPP) 치료를 위한 bitopertin에 대한 FDA와의 Phase 2 종료 회의에서 긍정적인 피드백을 발표했습니다. FDA는 치료 6개월 후 통증 없이 평균 월간 햇빛 노출 시간을 측정하는 주요 연구 목표를 포함하여 APOLLO 시험에 대한 모든 제안된 연구 매개변수에 동의했습니다. 회사는 PPIX 감소를 대체 지표로 사용하여 가속 승인 가능성을 받았습니다. 2025년 중반에 계획된 APOLLO 시험은 EPP 및 XLP 환자에게 12세 이상의 환자에게 60mg의 bitopertin 용량을 사용하는 무작위 이중 맹검 위약 대조 연구가 될 것입니다.
Disc Medicine (NASDAQ:IRON) a annoncé un retour positif de sa réunion de fin de Phase 2 avec la FDA concernant le bitopertin pour le traitement de la Protoporphyrie Érythropoïétique (EPP). La FDA a accepté tous les paramètres d'étude proposés pour l'essai APOLLO, y compris un critère principal mesurant le temps moyen d'exposition au soleil sans douleur après 6 mois de traitement. L'entreprise a reçu la possibilité d'une approbation accélérée en utilisant la réduction de PPIX comme critère substitut. L'essai APOLLO, prévu pour mi-2025, sera une étude randomisée, en double aveugle, contrôlée par placebo, utilisant une dose de 60 mg de bitopertin chez des patients atteints d'EPP et de XLP âgés de 12 ans et plus.
Disc Medicine (NASDAQ:IRON) hat positives Feedback von seinem Abschlussgespräch der Phase 2 mit der FDA bezüglich Bitopertin zur Behandlung der Erythropoetischen Protoporphyrie (EPP) erhalten. Die FDA stimmte allen vorgeschlagenen Studienparametern für die APOLLO-Studie zu, einschließlich eines primären Endpunkts, der die durchschnittliche monatliche Sonnenlichteinstrahlung ohne Schmerzen nach 6 Monaten Behandlung misst. Das Unternehmen erhielt die Möglichkeit zur beschleunigten Zulassung, indem eine PPIX-Reduktion als Surrogatendpunkt verwendet wird. Die APOLLO-Studie, die für Mitte 2025 geplant ist, wird eine randomisierte, doppelblinde, placebokontrollierte Studie sein, die eine Dosis von 60 mg Bitopertin bei EPP- und XLP-Patienten ab einem Alter von 12 Jahren verwendet.
- FDA agreement on all proposed study parameters for APOLLO trial
- Potential accelerated approval pathway using existing data
- Single trial may be sufficient for registration
- Clear regulatory path forward established
- Trial initiation not until mid-2025
- Additional FDA meeting required to finalize APOLLO trial details
Insights
The successful FDA End-of-Phase 2 meeting for bitopertin marks a significant milestone for Disc Medicine's EPP program. The agreement on utilizing PPIX reduction as a surrogate endpoint for accelerated approval could substantially expedite the drug's path to market. The APOLLO trial design, with its focus on sunlight exposure without pain, addresses a critical quality-of-life metric for EPP patients.
The inclusion of patients aged 12+ and the expansion to XLP broadens the potential patient population. The 60 mg dose selection and 6-month treatment duration are supported by previous data, suggesting a well-defined therapeutic window. The possibility of accelerated approval based on existing data indicates strong preliminary efficacy signals and could significantly reduce time to market, providing earlier access for patients with treatment options.
This regulatory breakthrough significantly de-risks Disc Medicine's lead program and could accelerate revenue generation. The potential for accelerated approval based on existing data could reduce development costs and time to market by several years. With a market cap of
The agreement on trial design parameters reduces regulatory uncertainty and provides clear development milestones through mid-2025. The single pivotal trial requirement, rather than multiple studies, represents significant cost savings and faster path to commercialization. EPP's orphan disease status and treatment options position bitopertin for premium pricing if approved, potentially leading to strong market penetration.
- Alignment with the FDA across all proposed study parameters, providing a clear development path to registration
- Agreement on proposed primary endpoint of average monthly time in sunlight during the last month following a 6-month treatment period
- Potential for accelerated approval based on existing data and utilizing reduction of PPIX as a surrogate endpoint
- Plan to initiate APOLLO trial, a 6-month study of a 60 mg dose of bitopertin in EPP and XLP patients ages 12+ by mid-2025
- Management will host a conference call on Monday, November 4 at 8:00 am EST
WATERTOWN, Mass., Nov. 04, 2024 (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 end-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA), supporting the regulatory path forward for bitopertin in EPP.
“We are thrilled with the outcome of our end-of-Phase 2 meeting with the FDA, which provides us with a clear development path forward for bitopertin. Importantly, the FDA agreed with all attributes of our study design, including a primary endpoint we feel is statistically robust and would fully capture the potential benefit of bitopertin in EPP,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc. “We’re particularly excited by the potential to file under the Accelerated Approval Program based on our existing data and use of PPIX reduction as a surrogate endpoint. This is a testament to the significant unmet need in EPP and the strength of the bitopertin data package, and we look forward to engaging further with the FDA on this pathway.”
The meeting resulted in agreement on all proposed attributes of the APOLLO study, which the company plans to initiate by mid-2025, including the:
- Sufficiency of a single, randomized, double-blind, placebo-controlled trial;
- Primary endpoint of average monthly total time in sunlight without pain during the last month following 6 months of treatment;
- Additional measures such as change in PPIX, occurrence of phototoxic reactions, cumulative total pain-free time in sunlight, and patient global impression of change (PGIC);
- Selection of 60 mg dose of bitopertin and 6-month treatment duration; and
- Inclusion of patients aged 12+ with EPP, including X-linked protoporphyria (XLP).
In addition, the FDA also agreed with the potential for reduction of PPIX to serve as a surrogate endpoint to support an accelerated approval. Under this pathway, Disc would have the potential to submit an NDA based on the existing data package and the APOLLO trial would serve as a confirmatory trial. Disc will be meeting with the FDA to finalize the details of APOLLO and plans to provide an update in Q1 2025 on this discussion as well as timing for NDA filing under an accelerated pathway.
Management will host a call to discuss these updates on Monday, November 4 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) and X-linked Protoporphyria (XLP)
Erythropoietic protoporphyria (EPP) and X-linked Protoporphyria (XLP) are rare, debilitating and potentially life-threatening diseases 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 clinical study of bitopertin in EPP and XLP patients, including the proposed study parameters, the anticipated timeline, and the results thereof; and the possible regulatory path forward for bitopertin in EPP, including the potential to seek approval under the Accelerated Approval pathway and conduct a confirmatory trial, and the timeline of related discussions with the FDA. 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 our Annual Report on Form 10-K for the year ended December 31, 2023, and in subsequent Quarterly Reports on Form 10-Q. 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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