Merck Announces Phase 3 Trial Initiation for Bomedemstat, an Investigational Candidate for the Treatment of Certain Patients With Essential Thrombocythemia
Merck (NYSE: MRK) has initiated Shorespan-007, a pivotal Phase 3 clinical trial for bomedemstat, an investigational oral LSD1 inhibitor, for treating essential thrombocythemia (ET) patients who haven't received cytoreductive therapy. This trial, comparing bomedemstat to the standard chemotherapy hydroxyurea, will enroll about 300 patients globally. The primary endpoint is durable clinicohematologic response rate.
Bomedemstat is also being studied in Shorespan-006, another Phase 3 trial for ET patients intolerant to hydroxyurea. It has received FDA Orphan Drug and Fast Track Designations for ET and myelofibrosis treatment. Merck recently presented updated data from the Phase 2b Shorespan-003 trial at the ASH Annual Meeting in December 2023.
Merck (NYSE: MRK) ha avviato Shorespan-007, uno studio clinico cruciale di Fase 3 per bomedemstat, un inibitore orale di LSD1 in fase di sperimentazione, per trattare i pazienti affetti da trombocitemia essenziale (ET) che non hanno ricevuto terapia citoreductiva. Questo studio, che confronta bomedemstat con la chemioterapia standard idrossiurea, recluterà circa 300 pazienti a livello globale. L'obiettivo primario è il tasso di risposta clinico-ematologico duraturo.
Bomedemstat è anche studiato in Shorespan-006, un altro trial di Fase 3 per pazienti ET intolleranti all'idrossiurea. Ha ricevuto le designazioni di Farmaco Orfano e Fast Track dalla FDA per il trattamento di ET e mielofibrosi. Merck ha recentemente presentato dati aggiornati dallo studio di Fase 2b Shorespan-003 durante l'Assemblea Annuale ASH nel dicembre 2023.
Merck (NYSE: MRK) ha iniciado Shorespan-007, un ensayo clínico pivotal de Fase 3 para bomedemstat, un inhibidor oral de LSD1 en investigación, para tratar a pacientes con trombocitemia esencial (ET) que no han recibido terapia citoreductora. Este ensayo, que compara bomedemstat con la quimioterapia estándar hidroxicarbamida, inscribirá a aproximadamente 300 pacientes a nivel mundial. El objetivo primario es la tasa de respuesta clinico-hematológica duradera.
Bomedemstat también se está estudiando en Shorespan-006, otro ensayo de Fase 3 para pacientes ET intolerantes a la hidroxicarbamida. Ha recibido designaciones de Medicamento Huérfano y Vía Rápida de la FDA para el tratamiento de ET y mielofibrosis. Merck presentó recientemente datos actualizados del ensayo de Fase 2b Shorespan-003 en la Reunión Anual ASH en diciembre de 2023.
머크(Merck, NYSE: MRK)는 bomedemstat의 중요 3상 임상 시험인 Shorespan-007을 시작했습니다. 이는 세포 감량 요법을 받지 않은 본태성 혈소판 증가증(ET) 환자를 치료하기 위한 경구용 LSD1 억제제입니다. 이 시험은 bomedemstat과 표준 화학 요법인 하이드록시에야를 비교하며, 전 세계에서 약 300명의 환자를 모집할 예정입니다. 주요 목표는 지속적인 임상 혈액학적 반응률입니다.
bomedemstat은 하이드록시에야에 내성이 있는 ET 환자를 위한 또 다른 3상 시험인 Shorespan-006에서도 연구되고 있습니다. 이는 ET 및 골수 섬유증 치료를 위한 FDA의 희귀의약품 및 패스트 트랙 지정을 받았습니다. 머크는 최근 2023년 12월 ASH 연례 회의에서 2b상 Shorespan-003 시험의 업데이트된 데이터를 발표했습니다.
Merck (NYSE: MRK) a lancé Shorespan-007, un essai clinique pivot de Phase 3 pour bomedemstat, un inhibiteur oral de LSD1 en cours d'étude, destiné à traiter des patients atteints de thrombocytémie essentielle (ET) n'ayant pas reçu de thérapie cytoréductrice. Cet essai, qui compare bomedemstat à la chimiothérapie standard hydroxyurée, devrait recruter environ 300 patients à l'échelle mondiale. L'objectif principal est le taux de réponse clinico-hématologique durable.
Bomedemstat est également étudié dans Shorespan-006, un autre essai de Phase 3 pour les patients ET intolérants à l'hydroxyurée. Il a reçu des désignations de médicament orphelin et de voie rapide de la FDA pour le traitement de l'ET et de la myélofibrose. Merck a récemment présenté des données mises à jour de l'essai de Phase 2b Shorespan-003 lors de la réunion annuelle de l'ASH en décembre 2023.
Merck (NYSE: MRK) hat Shorespan-007, eine entscheidende Phase-3-Studie für bomedemstat, einen experimentellen oralen LSD1-Inhibitor, initiiert, um Patienten mit essentieller Thrombozythämie (ET), die keine zytoreduktive Therapie erhalten haben, zu behandeln. Diese Studie, die bomedemstat mit der Standardchemotherapie Hydroxyurea vergleicht, wird weltweit etwa 300 Patienten einschließen. Der primäre Endpunkt ist die anhaltende klinisch-hematologische Ansprechrate.
Bomedemstat wird auch in Shorespan-006, einer weiteren Phase-3-Studie für ET-Patienten, die Hydroxyurea nicht vertragen, untersucht. Es hat von der FDA die Auszeichnungen Orphan Drug und Fast Track für die Behandlung von ET und Myelofibrose erhalten. Merck hat kürzlich aktualisierte Daten aus der Phase-2b-Studie Shorespan-003 auf dem ASH-Jahrestreffen im Dezember 2023 präsentiert.
- Initiation of Phase 3 clinical trial for bomedemstat in essential thrombocythemia
- Bomedemstat has FDA Orphan Drug and Fast Track Designations for ET and myelofibrosis
- Ongoing multiple Phase 2 clinical trials for bomedemstat in various myeloproliferative neoplasms
- None.
Insights
The initiation of Merck's Phase 3 Shorespan-007 trial for bomedemstat in essential thrombocythemia (ET) patients is a significant step forward in addressing an unmet medical need. This LSD1 inhibitor represents a novel approach to treating ET, a condition that has seen little innovation in decades. The trial's design, comparing bomedemstat to the standard-of-care hydroxyurea, with a primary endpoint of durable clinicohematologic response rate, is robust and clinically relevant.
Importantly, the inclusion of patient-reported outcomes in the secondary endpoints, such as fatigue scores and symptom assessments, demonstrates a focus on quality of life improvements. The Fast Track and Orphan Drug designations from the FDA underscore the potential impact of this therapy. With a target enrollment of 300 patients, this global study could provide compelling evidence for a new first-line treatment option in ET, potentially shifting the treatment paradigm in myeloproliferative neoplasms.
Merck's advancement of bomedemstat into Phase 3 trials for essential thrombocythemia is a promising development in the field of myeloproliferative neoplasms (MPNs). As an orally available LSD1 inhibitor, bomedemstat offers a novel mechanism of action that could address the limitations of current cytoreductive therapies. The Shorespan-007 trial's focus on treatment-naïve patients is particularly noteworthy, as it may establish bomedemstat as a potential first-line therapy.
The inclusion of event-free survival and disease progression rate as secondary endpoints will provide important data on the long-term efficacy and safety of bomedemstat. Additionally, the concurrent Phase 3 Shorespan-006 trial in hydroxyurea-resistant or intolerant patients demonstrates a comprehensive approach to addressing various patient populations within ET. If successful, these trials could lead to a significant expansion of treatment options for MPN patients, potentially improving both disease control and quality of life.
Merck's initiation of the Phase 3 Shorespan-007 trial for bomedemstat in essential thrombocythemia (ET) represents a strategic move in the rare disease market. With ET affecting approximately 1 in 100,000 people, bomedemstat's potential approval could secure Merck a significant position in this niche but lucrative market. The drug's multiple designations, including Orphan Drug and Fast Track, may accelerate its path to market and provide extended exclusivity, enhancing its commercial potential.
Investors should note that success in rare diseases can lead to high-margin products due to premium pricing. However, the extended timeline of Phase 3 trials means that any revenue impact is likely several years away. The company's commitment to myeloproliferative neoplasms, evidenced by multiple ongoing trials, suggests a long-term strategy to build a strong portfolio in hematology-oncology. This diversification could help offset potential losses from patent expirations in other areas, contributing to Merck's future growth prospects.
The initiation of a second Phase 3 clinical trial for bomedemstat demonstrates company’s commitment to advancing research in myeloproliferative neoplasms (MPNs)
“The standard of care in essential thrombocythemia has remained unchanged for decades, and patients are in need of new options that have the potential to not only improve disease control, but also improve their quality of life,” said Dr. Gregory Lubiniecki, vice president, global clinical development, Merck Research Laboratories. “We are rapidly advancing our clinical development programs with the goal of helping to address these unmet needs and bring more options to patients living with myeloproliferative neoplasms.”
Shorespan-007 is a Phase 3, randomized, double-blind, active comparator-controlled clinical trial (NCT06456346) evaluating bomedemstat compared to the current standard of care chemotherapy, hydroxyurea, for treatment of patients with ET who have previously not received cytoreductive therapy. The trial will enroll approximately 300 patients globally. The primary endpoint of the study is durable clinicohematologic response rate (CHR). Key secondary endpoints include Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF v4.0) individual fatigue symptom item score, Patient-reported Outcomes Measurement Information System (PROMIS) Fatigue SF-7a total fatigue score and MFSAF v4.0 total symptom score. Additional secondary endpoints include duration of hematologic remission, event-free survival and disease progression rate.
As previously announced, in addition to Shorespan-007, bomedemstat is also being investigated in Shorespan-006, a Phase 3, global, randomized, open-label, active comparator-controlled clinical trial (NCT06079879), evaluating bomedemstat compared to best available therapy as treatment in approximately 300 patients with ET who have an inadequate response to or are intolerant of hydroxyurea.
Bomedemstat has
About essential thrombocythemia
Essential thrombocythemia is a chronic, rare blood disorder that is the most common type of myeloproliferative neoplasm. Essential thrombocythemia is most often caused by genetic mutations that cause the bone marrow to produce too many platelets, which can obstruct blood flow and cause a stroke, heart attack or pulmonary embolism. The disease is more common in women than men and in people ages 50 to 70. It is estimated that fewer than 200,000 people in the
About bomedemstat
Bomedemstat (MK-3543) is an investigational small molecule, irreversible LSD1 inhibitor being developed by Merck. LSD1 regulates the proliferation of hematopoietic stem cells, playing an essential role in cell differentiation and maturation. Bomedemstat is being evaluated in a wide range of MPNs, including ET, MF and PV.
Merck’s focus on cancer
Every day, we follow the science as we work to discover innovations that can help patients, no matter what stage of cancer they have. As a leading oncology company, we are pursuing research where scientific opportunity and medical need converge, underpinned by our diverse pipeline of more than 25 novel mechanisms. With one of the largest clinical development programs across more than 30 tumor types, we strive to advance breakthrough science that will shape the future of oncology. By addressing barriers to clinical trial participation, screening and treatment, we work with urgency to reduce disparities and help ensure patients have access to high-quality cancer care. Our unwavering commitment is what will bring us closer to our goal of bringing life to more patients with cancer. For more information, visit https://www.merck.com/research/oncology.
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