Blueprint Medicines Corporation Research Articles

Avapritinib versus Placebo in Indolent Systemic Mastocytosis

BackgroundIndolent systemic mastocytosis (ISM) is a clonal mast-cell disease driven by the KIT D816V mutation. We assessed the efficacy and safety of avapritinib versus placebo, both with best supportive care, in patients with ISM.MethodsWe randomized patients with moderate to severe ISM (total symptom score [TSS] of ≥28; scores range from 0 to 110, with higher numbers indicating more severe symptoms) two to one to avapritinib 25 mg once daily (n=141) or placebo (n=71). The primary end point was mean change in TSS based on the 14-day average of patient-reported severity of 11 symptoms. Secondary end points included reductions in serum tryptase and blood KIT D816V variant allele fraction (≥50%), reductions in TSS (≥50% and ≥30%), reduction in bone marrow mast cells (≥50%), and quality of life measures.ResultsFrom baseline to week 24, avapritinib-treated patients had a decrease of 15.6 points (95% CI, −18.6 to −12.6) in TSS compared to a decrease of 9.2 points (−13.1 to −5.2) in the placebo group; P<0.003. From baseline to Week 24, 76/141 patients (54%; 45% to 62%) in the avapritinib group compared to 0/71 patients in the placebo group achieved a ≥50% reduction in serum tryptase level; P<0.001. Edema and increases in alkaline phosphatase were more common with avapritinib than placebo; there were few treatment discontinuations because of adverse events.ConclusionsIn this trial, avapritinib was superior to placebo in reducing uncontrolled symptoms and mast-cell burden in patients with ISM. The long-term safety and efficacy of this approach for patients with ISM remain the focus of the ongoing trial. (Funded by Blueprint Medicines Corporation; ClinicalTrials.gov number, NCT03731260.)

Psychometric evaluation of the Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF©) and determination of a threshold score for moderate symptoms

BackgroundThe Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF) (©Blueprint Medicines Corporation), a 12-item daily diary that assesses 11 signs and symptoms of indolent systemic mastocytosis (ISM) and smoldering systemic mastocytosis (SSM), was psychometrically evaluated among patients with ISM. Additionally, thresholds of the ISM-SAF total symptom score (TSS) to distinguish patients with moderate to severe symptoms from those with mild symptoms were evaluated.MethodsThe ISM-SAF was completed daily as an electronic diary in a prospective, observational study utilizing an online survey of patients with ISM in the United States. Descriptive statistics, psychometric analyses, and analyses to estimate ISM-SAF TSS clinical cutoff values were conducted.ResultsA total of 103 patients (81.6% female; mean age = 50.2 [± 12.6]) with a self-reported diagnosis of ISM or SSM (58 of whom also had a medically documented diagnosis) contributed to the analyses. Psychometric analysis supported the trustworthiness of the biweekly TSS, which was reliable (α > 0.8, ICC > 0.9), construct-valid, and able to distinguish among clinically distinct groups as specified by the Patient Global Impression of Severity, 12-item Short-Form Health Survey, and Mastocytosis Quality of Life Questionnaire (p < 0.01). A biweekly ISM-SAF TSS from 21 to 28 begins to distinguish the moderately to severely symptomatic ISM/SSM patients from mildly symptomatic patients.ConclusionThe biweekly TSS of ISM-SAF was reliable, construct-valid, and able to distinguish among clinically distinct groups. A cut-off value of 28 is a conservative threshold that can be used for screening purposes in future clinical studies to identify patients with at least a moderate severity of ISM symptoms.

MPN-435 Overall Survival in Patients With Systemic Mastocytosis With Associated Hematologic Neoplasm Treated With Avapritinib Versus Best Available Therapy.

Avapritinib, a selective KIT D816V inhibitor, was approved by the FDA and EMA (by EMA only after prior systemic therapy) for treating adults with advanced systemic mastocytosis (AdvSM) based on the single-arm phase 1 EXPLORER (NCT02561988) and phase 2 PATHFINDER (NCT03580655) studies. SM-AHN is the most common AdvSM subtype, however, there is no randomized controlled trial that compares the efficacy of avapritinib versus best available therapy (BAT) in this population. This study (NCT04695431) compared overall survival (OS) in patients with SM-AHN treated with avapritinib in EXPLORER and PATHFINDER versus patients treated with BAT in standard clinical practice. A global, observational, retrospective chart review study was conducted at 6 sites to collect data on SM-AHN patients treated with BAT. These data were pooled with those from EXPLORER and PATHFINDER. Patients with SM-AHN receiving BAT were identified using inclusion/ exclusion criteria similar to EXPLORER and PATHFINDER and could contribute data on multiple lines of therapy (LOTs). OS, defined as time from avapritinib or BAT initiation to death from any cause, was descriptively analyzed using the Kaplan-Meier method. Inverse probability of treatment weighting (IPTW) was used to adjust for differences in key covariates between the treatment cohorts. OS was compared between cohorts using an IPTW-weighted Cox proportional hazards model. 119 avapritinib (median age 70 years; 61% male) and 83 BAT patients (median age 71 years; 76% male) were included; 58% of avapritinib and 44% of BAT patients had received prior systemic therapy. BAT patients contributed 121 LOTs, most commonly tyrosine kinase inhibitors (60%) or cytoreductive therapies (38%). Median OS for avapritinib patients was 46.9 months (95% confidence interval [CI]: 44.9-not estimable) and 18.0 months (95% CI: 13.0-26.8) for BAT. Weighted Cox analyses showed that OS was significantly improved for patients treated with avapritinib versus BAT (hazard ratio [95% CI]: 0.42 [0.24-0.74]; P<0.001), after adjustment for key covariates. The results of this observational, retrospective analysis indicate that patients with SM-AHN who were treated with avapritinib had significantly longer OS than patients treated with BAT. This study was sponsored by Blueprint Medicines Corporation.

177 (PB057) - BLU-945 or BLU-701 as single agents versus their combination with osimertinib in EGFR L858R driven tumor models

Background: The most common activating EGFR mutations (EGFRm) in non-small cell lung cancer (NSCLC) are exon 19 deletions (ex19del) and L858R substitutions. EGFR tyrosine kinase inhibitors (TKIs) as first-line (1L) therapy have improved outcomes in EGFR-driven advanced NSCLC; however, treatment resistance eventually occurs. Patients with EFGR L858R-driven advanced NSCLC (39% of all EGFR mutations in NSCLC) have shorter progression-free survival than those with EGFR ex19del, suggesting a significant unmet need. BLU-945 and BLU-701 are investigational, reversible, selective, orally available TKIs optimized for use as singleagent or combination therapy to suppress activating and on-target resistance EGFR mutants while sparing wild-type EGFR. in vivo studies have shown antitumor activity of BLU-945 against EGFR L858R, L858R/C797S, L858R/ T790M and EGFRm/T790M/C797S mutants, and BLU-701 against EGFRm and EGFRm/C797S mutants. Combination of BLU-945 and BLU-701 has also shown in vivo activity in EGFRm/C797S-driven mutant NSCLC. Combining BLU-945 or BLU-701 with osimertinib could potentially enable superior coverage of frequent on-target resistance mechanisms as well additional potency on the primary EGFR activating mutations. Central nervous system (CNS) metastases are an ongoing challenge in NSCLC. BLU-701 has already shown preclinical CNS activity; and both BLU-945 and BLU-701 are currently being investigated for potential treatment or prevention of CNS metastases. Due to poorer outcomes in patients with EGFR L858R-driven advanced NSCLC treated with osimertinib, and the inevitability of treatment resistance, studies were conducted to evaluate the activity of BLU-945 or BLU-701 in combination with osimertinib in prolonging duration of response (DOR) in preclinical NSCLC tumor models driven by the L858R mutation. Material and Methods: The antitumor activity of BLU-945 or BLU-701, as single agents or in combination with osimertinib was evaluated in EGFR L858R-driven tumor models. Results: Administration of single-agent BLU-945 or BLU-701 resulted in tumor growth inhibition in EGFR L858R-driven PDX models. BLU-945 in combination with osimertinib resulted in prolonged DOR and survival when compared with BLU-945 or osimertinib as single agents. BLU-701 in combination with osimertinib resulted in superior tumor growth inhibition compared with BLU-701 or osimertinib as single agents. Conclusions: The in vivo antitumor activities of BLU-945 and BLU-701 as single agents suggest both BLU-945 and BLU-701 have the potential to be used as 1L therapy in patients with EGFR L858R-driven NSCLC. The superior in vivo antitumor activity of BLU-945 or BLU-701 in combination with osimertinib in prolonging the DOR or increasing tumor growth inhibition in these models may have clinical application in improving outcomes of patients with EGFR L858R-driven NSCLC in 1L settings. Conflict of interest: Other Substantive Relationships: Tavera L, Dhande A, Dineen T, Iliou M, Rouskin-Faust T, Rozsahegyi E, Conti C: Employee and/or equity holder of Blueprint Medicines Corporation.

Pralsetinib: chemical and therapeutic development with FDA authorization for the management of RET fusion-positive non-small-cell lung cancers.

Pralsetinib (PRL) is a selective Rearranged during Transfection (RET) inhibitor, developed by Blueprint Medicines Corporation for the treatment of RET fusion non-small-cell lung cancer (NSCLC), papillary thyroid cancer (PTC), and medullary thyroid carcinoma (MTC). RET is a known proto-oncogene found in NSCLC, PTC, and MTC. PRL was recently granted accelerated USFDA approval with the brand name GAVRETO™ on 4 September 2020 to treat metastatic RET fusion-positive NSCLC and was updated on 1 December 2020 with the addition of advanced and metastatic RET-altered MTC and PTC in the USA. On 19 November 2021, the European Commission granted conditional marketing authorization to PRL for use as a single agent in adult patients with RET fusion-positive advanced NSCLC. They were not previously treated with an RET inhibitor. This review article summarizes the milestones in the development of PRL, chemistry, chemical (synthesis) research and development, characterization and identification of PRL-resistant RET mutants, the structural basis of resistance to PRL, mechanism of action, pharmacokinetics, pharmacodynamic, adverse effects, and regulatory status, including ongoing clinical trial of PRL and other potential drug candidates, leading to this first approval of PRL for the treatment of various solid tumors (RET fusion NSCLC, MTC, and PTC).

Effective Control of Advance Systemic Mastocytosis with Avapritinib: Mutational Analysis from the Explorer Clinical Study

Effective Control of Advance Systemic Mastocytosis with Avapritinib: Mutational Analysis from the Explorer Clinical Study

Efficacy of Avapritinib in Patients with Advanced Systemic Mastocytosis: Hematologic and Bone Marrow Responses from the Phase 2 Open-Label, Single-Arm, Pathfinder Study

Efficacy of Avapritinib in Patients with Advanced Systemic Mastocytosis: Hematologic and Bone Marrow Responses from the Phase 2 Open-Label, Single-Arm, Pathfinder Study

Clinical Features Associated with Long-Term Ruxolitinib Exposure in Myelofibrosis and Predictive Role of Neutrophils

Clinical Features Associated with Long-Term Ruxolitinib Exposure in Myelofibrosis and Predictive Role of Neutrophils

Safety and Tolerability of Fedratinib (FEDR), an Oral Inhibitor of Janus Kinase 2 (JAK2), in Patients with Intermediate- or High-Risk Myelofibrosis (MF) Previously Treated with Ruxolitinib (RUX): Results from the Phase 3b FREEDOM Trial

Safety and Tolerability of Fedratinib (FEDR), an Oral Inhibitor of Janus Kinase 2 (JAK2), in Patients with Intermediate- or High-Risk Myelofibrosis (MF) Previously Treated with Ruxolitinib (RUX): Results from the Phase 3b FREEDOM Trial

A Randomized Open-Label, Phase 3 Study to Evaluate Imetelstat Versus Best Available Therapy (BAT) in Patients with Intermediate-2 (Int-2) or High-Risk Myelofibrosis (MF) Refractory to Janus Kinase Inhibitor (JAKi)

A Randomized Open-Label, Phase 3 Study to Evaluate Imetelstat Versus Best Available Therapy (BAT) in Patients with Intermediate-2 (Int-2) or High-Risk Myelofibrosis (MF) Refractory to Janus Kinase Inhibitor (JAKi)

Disease-Modifying Potential of BET Inhibitor Pelabresib (CPI-0610) As Demonstrated By Improvements in Bone Marrow Function and Clinical Activity in Patients with Myelofibrosis - Preliminary Data

Disease-Modifying Potential of BET Inhibitor Pelabresib (CPI-0610) As Demonstrated By Improvements in Bone Marrow Function and Clinical Activity in Patients with Myelofibrosis - Preliminary Data

A Pilot Study of the Anti-SLAMF7 Monoclonal Antibody, Elotuzumab, in Myelofibrosis

A Pilot Study of the Anti-SLAMF7 Monoclonal Antibody, Elotuzumab, in Myelofibrosis

A Retrospective Head-to-Head Comparison between Pacritinib and Ruxolitinib in Patients with Myelofibrosis and Moderate to Severe Thrombocytopenia

A Retrospective Head-to-Head Comparison between Pacritinib and Ruxolitinib in Patients with Myelofibrosis and Moderate to Severe Thrombocytopenia

Pelabresib (CPI-0610) Monotherapy in Patients with Myelofibrosis - Update of Clinical and Translational Data from the Ongoing Manifest Trial

Pelabresib (CPI-0610) Monotherapy in Patients with Myelofibrosis - Update of Clinical and Translational Data from the Ongoing Manifest Trial

Advancing Personalized Care for Patients with Myeloproliferative Neoplasms (MPNs): Findings from a Phase 2 Scale-up Quality Initiative across Two Large U.S. Oncology Systems

BackgroundMyeloproliferative neoplasms (MPNs), a group of rare hematologic malignancies comprised of myelofibrosis, polycythemia vera and essential thrombocythemia, significantly impact the lives of patients with historically few effective treatment options. In this quality improvement (QI) initiative we assessed barriers to patient-centered MPN care in 2 large U.S. hospital systems.MethodsBetween 3/2021 and 5/2021, 24 hematology/oncology healthcare professionals (HCPs) from 2 large hospital systems completed surveys designed to characterize self-reported practice patterns, challenges, and barriers to collaborative MPN care. Additionally, 26 Black patients and 25 non-Black patients with MPNs completed surveys regarding their goals for treatment, barriers to care, and communication with providers. Findings from all surveys paired with patient chart data was presented to 18 HCPs from the systems in AF sessions to reflect on their own practice patterns and prioritize areas for improvement in MPN care. Participants developed team-based action plans to overcome identified challenges, including barriers in risk stratification, care coordination, and shared decision-making (SDM) for patients with MPNs. Surveys conducted before and after the small-group AF sessions evaluated changes in participants' knowledge and confidence in delivering collaborative, patient-centered MPN care.ResultsTeam-Based Surveys: HCPs identified difficulty managing their symptoms (35%) and difficulty choosing therapy that best meets their treatment preferences and goals (25%) as the most pressing challenges their patient's face in their MPN care. The most challenging issue encountered by HCPs in selecting therapies for patients with MPNs is identifying when patients are undergoing disease progression/transformation (48%). HCPs reported effects on quality of life (75%) and treatment effectiveness (65%) as the most important factors for treatment decision-making among patients with MPNs. Teams were underutilizing SDM to provide patient-centered care, citing not enough time to engage in SDM (55%) and patients' low health literacy (50%) as the largest barriers.Patient Surveys: In contrast to HCP responses, the biggest challenge faced in their MPN care reported by Black patients was lack of reliable transportation or long distance to and from my care center (46%) difficulty managing my symptoms (36%) for non-Black patients. Furthermore, Black patients with MPN identified cost of treatment (56%) and advice from loved ones (40%) as the top factors for treatment decision-making, whereas, non-Black patients cited how the treatment is taken (52%) and how well the treatment will control my symptoms (50%). All patients identified they wish they had more time to discuss goals and preferences for treatment (62% Black, 64% non-Black ) with their provider. Black patients reported their MPN care team could improve most in education about MPNs and treatment options (73%), while non-Black patients felt improvements in empathy throughout the emotional journey of managing my MPN (68%) would be most beneficial.Small-Group AF Sessions: Across the 2 oncology centers, teams participating in the AF sessions (Table 1) shared a self-reported caseload of 219 patients with MPNs per month. HCPs reported meaningful shifts in confidence in their ability to provide optimal, patient-centered care (Figure 1) and knowledge of treatment options for MPNs (Figure 2). The aspects of patient-centered care HCPs will routinely discuss in more detail with patients are patients' goal and preferences (81%), results of genetic testing (63%), and risks and benefits of treatment options (56%). To achieve these goals, 63% of HCPs committed to improve team skills in appropriate risk stratification and differentiation of therapy based on patient-centered factors followed by sharing action plans with additional clinical team members (56%).ConclusionsParticipation in this QI initiative resulted in increased confidence in hematology/oncology HCPs ability to deliver patient-centered MPN care and improve commitment to team-based collaboration. Remaining practice gaps and challenges can inform future QI programs.Study Sponsor StatementThe study reported in this abstract was funded by an independent educational grant from Incyte Corporation. The grantors had no role in the study design, execution, analysis, or reporting. [Display omitted] DisclosuresVerstovsek: CTI BioPharma: Research Funding; NS Pharma: Research Funding; Ital Pharma: Research Funding; Celgene: Consultancy, Research Funding; Roche: Research Funding; Protagonist Therapeutics: Research Funding; Promedior: Research Funding; PharmaEssentia: Research Funding; Gilead: Research Funding; Incyte Corporation: Consultancy, Research Funding; Genentech: Research Funding; Blueprint Medicines Corp: Research Funding; AstraZeneca: Research Funding; Novartis: Consultancy, Research Funding; Sierra Oncology: Consultancy, Research Funding; Constellation: Consultancy; Pragmatist: Consultancy. Mesa: Sierra Oncology: Consultancy, Research Funding; Gilead: Research Funding; Novartis: Consultancy; Celgene: Research Funding; Genentech: Research Funding; CTI: Research Funding; Abbvie: Research Funding; CTI: Research Funding; Incyte Corporation: Consultancy, Research Funding; Promedior: Research Funding; Samus: Research Funding; Constellation Pharmaceuticals: Consultancy, Research Funding; Pharma: Consultancy; AOP: Consultancy; La Jolla Pharma: Consultancy.

Surpass-ET Trial: A Phase 3, Open-Label, Multicenter, Randomized, Active-Controlled Study to Assess Pharmacokinetics and Compare the Efficacy, Safety, and Tolerability of P1101 Vs Anagrelide As Second Line Therapy for Essential Thrombocythemia

▪Background: Essential thrombocythemia (ET) is a subtype of chronic myeloproliferative neoplasms (MPN) characterized by thrombocytosis and disease-related symptoms, which may be difficult to manage. Patients with ET are also at higher risk of thrombosis and hemorrhage. Ideal therapeutic approaches should achieve adequate cytoreduction, reduce the risk of thrombo-hemorrhagic complications, and prevent progression to post-ET myelofibrosis (PET-MF) or secondary acute myeloid leukemia (AML). Low-dose aspirin with hydroxyurea (HU) is typically given as first-line therapy in high-risk patients. However, approximately 20-40% of ET patients become HU-intolerant or -resistant. In ET, resistance and/or intolerance portend an increased risk of thrombosis, hemorrhage, disease transformation and death. There is a paucity of prospective clinical trial data to guide management of ET patients who are HU resistant or intolerant. P1101 is a next generation monopegylated interferon (IFN) alfa-2b, developed specifically to treat MPNs, including ET.Study Design and Methods: The SURPASS-ET trial (NCT04285086) is a Phase 3, open-label, multicenter, randomized, active-controlled study to assess the efficacy, safety, tolerability, and pharmacokinetics (PK) of P1101 after 12 months of treatment compared with anagrelide as second line therapy for subjects with ET who have shown resistance or intolerance to HU. The primary endpoint is durable modified ELN composite response at 9 and 12 months from dosing. Cochran-Mantel-Haenszel test will be used for comparing the primary endpoint in the two treatment arms. The PK parameters of P1101, including (but not limited to) C min, T max, C max, and area-under-curve (AUC) will be derived using PPK analysis and the relationship between exposure and efficacy and safety endpoints will be examined using E-R analysis. Evaluation of efficacy will include clinical laboratory assessments, allelic burden measurements of CALR, JAK2, and MPL, spleen size measurements, bone marrow sampling (optional), EQ-5D-3L, and MPN Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) assessments.A total of 130 randomized subjects is planned to detect a difference of 40% (P1101) versus 15% (anagrelide) in durable ELN response rate with 90% power at alpha level = 0.05 using the chi-square test. To account for possible non-evaluability (e.g., no follow-up data), approximately 160 subjects will be randomized in this study to get 130 completed patients. Because of uncertainty in the assumptions on which the calculation of the sample size is based, an interim analysis for sample size adjustment will be implemented.Major inclusion criteria include subjects diagnosed with high-risk ET (either older than 60 years and JAK2V617F-positive at screening or having disease-related thrombosis or hemorrhage in the past), diagnosed according to the World Health Organization (WHO) 2016 criteria, documented resistance/intolerance to HU and IFN naïve or anti-P1101 binding antibody negative. Key exclusion criteria include pregnant females, significant cardiovascular disease, documented autoimmune disease and a history or presence of clinically significant depression or neurological disease.The study involves approximately 65 sites across the US, Taiwan, Japan, China, Hong Kong, Singapore, S. Korea, Canada, and Europe. To-date 55 patients (54 Asians, 1 Caucasian) have been randomized. The mean and median age at recruitment was 58.9 years (SD: 14.34) and 63 years (range 21 to 80 years) respectively. Twenty-seven men (49.1%) and twenty-eight women (50.9%) were recruited. Forty-two subjects (76.4%) had a TSS < 20. The study is being overseen by a Data Safety Monitoring Board (DSMB). [Display omitted] DisclosuresMesa: Gilead: Research Funding; Promedior: Research Funding; AOP: Consultancy; Incyte Corporation: Consultancy, Research Funding; Abbvie: Research Funding; Genentech: Research Funding; La Jolla Pharma: Consultancy; Novartis: Consultancy; Sierra Oncology: Consultancy, Research Funding; Samus: Research Funding; Pharma: Consultancy; CTI: Research Funding; Celgene: Research Funding; Constellation Pharmaceuticals: Consultancy, Research Funding; CTI: Research Funding. Komatsu: Fujifilm Wako Pure Chemical Corporation: Research Funding; Fuso Pharmaceutical Industries, Ltd.: Research Funding; Japan Tobacco Inc.: Consultancy; Otsuka Pharmaceutical Co. Ltd: Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis Pharma KK: Consultancy, Research Funding, Speakers Bureau; Shire Japan KK: Consultancy, Research Funding, Speakers Bureau; PharmaEssentia Japan KK: Consultancy, Current Employment, Research Funding; Chugai Pharmaceutical Co., Ltd.: Research Funding; Kyowa Hakko Kirin Co., Ltd.: Research Funding. Sato: PharmaEssentia Japan KK: Current Employment. Qin: PharmaEssentia Corp.: Current Employment. Urbanski: PharmaEssentia Corporation: Current Employment. Shih: PharmaEssentia Corporation: Consultancy. Zagrijtschuk: PharmaEssentia U.S.A. Corp.: Current Employment. Zimmerman: PharmaEssentia Corporation: Current Employment. Verstovsek: Gilead: Research Funding; Protagonist Therapeutics: Research Funding; NS Pharma: Research Funding; Incyte Corporation: Consultancy, Research Funding; PharmaEssentia: Research Funding; Ital Pharma: Research Funding; CTI BioPharma: Research Funding; Blueprint Medicines Corp: Research Funding; AstraZeneca: Research Funding; Promedior: Research Funding; Genentech: Research Funding; Celgene: Consultancy, Research Funding; Roche: Research Funding; Novartis: Consultancy, Research Funding; Sierra Oncology: Consultancy, Research Funding; Constellation: Consultancy; Pragmatist: Consultancy.

A Phase 1/2 Study of Single Agent Tagraxofusp, a First-in-Class CD123-Targeted Therapy, in Patients with Myelofibrosis That Is Relapsed/Refractory Following JAK Inhibitor Therapy

A Phase 1/2 Study of Single Agent Tagraxofusp, a First-in-Class CD123-Targeted Therapy, in Patients with Myelofibrosis That Is Relapsed/Refractory Following JAK Inhibitor Therapy

A Randomized, Phase 3 Trial of Fedratinib Versus Best Available Therapy in Patients with Intermediate-2 or High-Risk Myelofibrosis Previously Treated with Ruxolitinib (FREEDOM2)

A Randomized, Phase 3 Trial of Fedratinib Versus Best Available Therapy in Patients with Intermediate-2 or High-Risk Myelofibrosis Previously Treated with Ruxolitinib (FREEDOM2)

A Phase 3 Study of the Hepcidin Mimetic Rusfertide (PTG-300) in Patients with Polycythemia Vera

A Phase 3 Study of the Hepcidin Mimetic Rusfertide (PTG-300) in Patients with Polycythemia Vera

General Characteristics and Association of Myeloid Neoplasm with Thyroid Cancer

General Characteristics and Association of Myeloid Neoplasm with Thyroid Cancer