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Last Updated: February 17, 2025

CLINICAL TRIALS PROFILE FOR PRALSETINIB


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All Clinical Trials for Pralsetinib

Trial ID Title Status Sponsor Phase Start Date Summary
NCT03037385 ↗ Phase 1/2 Study of the Highly-selective RET Inhibitor, Pralsetinib (BLU-667), in Patients With Thyroid Cancer, Non-Small Cell Lung Cancer, and Other Advanced Solid Tumors Recruiting Blueprint Medicines Corporation Phase 1/Phase 2 2017-03-17 This is a Phase 1/2, open-label, first-in-human (FIH) study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary antineoplastic activity of pralsetinib (BLU-667) administered orally in patients with medullary thyroid cancer, RET-altered NSCLC and other RET-altered solid tumors.
NCT03037385 ↗ Phase 1/2 Study of the Highly-selective RET Inhibitor, Pralsetinib (BLU-667), in Patients With Thyroid Cancer, Non-Small Cell Lung Cancer, and Other Advanced Solid Tumors Recruiting Hoffmann-La Roche Phase 1/Phase 2 2017-03-17 This is a Phase 1/2, open-label, first-in-human (FIH) study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary antineoplastic activity of pralsetinib (BLU-667) administered orally in patients with medullary thyroid cancer, RET-altered NSCLC and other RET-altered solid tumors.
NCT04222972 ↗ A Study of Pralsetinib Versus Standard of Care for First-Line Treatment of Advanced Non-Small Cell Lung Cancer (NSCLC) Recruiting Blueprint Medicines Corporation Phase 3 2020-07-24 This is an international, randomized, open-label, Phase 3 study designed to evaluate whether the potent and selective RET inhibitor, pralsetinib, improves outcomes when compared to a platinum chemotherapy-based regimen chosen by the Investigator from a list of standard of care treatments, as measured primarily by progression free survival (PFS), for participants with RET fusion-positive metastatic NSCLC who have not previously received systemic anticancer therapy for metastatic disease. Participants who have centrally confirmed progressive disease on the control arm have the option to crossover to pralsetinib.
NCT04222972 ↗ A Study of Pralsetinib Versus Standard of Care for First-Line Treatment of Advanced Non-Small Cell Lung Cancer (NSCLC) Recruiting Hoffmann-La Roche Phase 3 2020-07-24 This is an international, randomized, open-label, Phase 3 study designed to evaluate whether the potent and selective RET inhibitor, pralsetinib, improves outcomes when compared to a platinum chemotherapy-based regimen chosen by the Investigator from a list of standard of care treatments, as measured primarily by progression free survival (PFS), for participants with RET fusion-positive metastatic NSCLC who have not previously received systemic anticancer therapy for metastatic disease. Participants who have centrally confirmed progressive disease on the control arm have the option to crossover to pralsetinib.
NCT04302025 ↗ A Study of Alectinib, Entrectinib, Vemurafenib Plus Cobimetinib, or Pralsetinib in Patients With Resectable Stages II-III Non-Small Cell Lung Cancer With ALK, ROS1, NTRK, BRAF V600, or RET Molecular Alterations Recruiting Blueprint Medicines Corporation Phase 2 2020-11-06 This trial will evaluate the efficacy and safety of targeted therapies in participants with resectable Stage IIA, IIB, IIIA, and select IIIB (T3N2) resectable and untreated non-small cell lung cancer (NSCLC) tumors with selected molecular alterations.
NCT04302025 ↗ A Study of Alectinib, Entrectinib, Vemurafenib Plus Cobimetinib, or Pralsetinib in Patients With Resectable Stages II-III Non-Small Cell Lung Cancer With ALK, ROS1, NTRK, BRAF V600, or RET Molecular Alterations Recruiting Genentech, Inc. Phase 2 2020-11-06 This trial will evaluate the efficacy and safety of targeted therapies in participants with resectable Stage IIA, IIB, IIIA, and select IIIB (T3N2) resectable and untreated non-small cell lung cancer (NSCLC) tumors with selected molecular alterations.
NCT04589845 ↗ Tumor-Agnostic Precision Immuno-Oncology and Somatic Targeting Rational for You (TAPISTRY) Platform Study Recruiting Hoffmann-La Roche Phase 2 2021-01-18 TAPISTRY is a Phase II, global, multicenter, open-label, multi-cohort study designed to evaluate the safety and efficacy of targeted therapies or immunotherapy as single agents or in rational, specified combinations in participants with unresectable, locally advanced or metastatic solid tumors determined to harbor specific oncogenic genomic alterations or who are tumor mutational burden (TMB)-high as identified by a validated next-generation sequencing (NGS) assay. Participants with solid tumors will be treated with a drug or drug regimen tailored to their NGS assay results at screening. Participants will be assigned to the appropriate cohort based on their genetic alteration(s). Treatment will be assigned on the basis of relevant oncogenotype, will have cohort-specific inclusion/exclusion criteria, and, unless otherwise specified, will continue until disease progression, loss of clinical benefit, unacceptable toxicity, participant or physician decision to discontinue, or death, whichever occurs first.
>Trial ID >Title >Status >Phase >Start Date >Summary

Clinical Trial Conditions for Pralsetinib

Condition Name

Condition Name for Pralsetinib
Intervention Trials
Neoplasms by Site 2
Carcinoma, Bronchogenic 2
Neoplasms, Germ Cell and Embryonal 2
Head and Neck Neoplasms 2
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Condition MeSH

Condition MeSH for Pralsetinib
Intervention Trials
Carcinoma, Non-Small-Cell Lung 4
Lung Neoplasms 4
Neoplasms 3
Thoracic Neoplasms 2
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Clinical Trial Locations for Pralsetinib

Trials by Country

Trials by Country for Pralsetinib
Location Trials
United States 94
Spain 15
Italy 15
Australia 11
Korea, Republic of 5
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Trials by US State

Trials by US State for Pralsetinib
Location Trials
California 7
Texas 6
Michigan 6
Florida 5
Tennessee 4
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Clinical Trial Progress for Pralsetinib

Clinical Trial Phase

Clinical Trial Phase for Pralsetinib
Clinical Trial Phase Trials
Phase 3 3
Phase 2 3
Phase 1/Phase 2 1
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Clinical Trial Status

Clinical Trial Status for Pralsetinib
Clinical Trial Phase Trials
Recruiting 5
Not yet recruiting 2
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Clinical Trial Sponsors for Pralsetinib

Sponsor Name

Sponsor Name for Pralsetinib
Sponsor Trials
Hoffmann-La Roche 5
Blueprint Medicines Corporation 3
Genentech, Inc. 2
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Sponsor Type

Sponsor Type for Pralsetinib
Sponsor Trials
Industry 10
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Pralsetinib: A Breakthrough in RET Fusion-Positive Non-Small Cell Lung Cancer Treatment

Introduction

Pralsetinib, marketed as Gavreto, has emerged as a significant advancement in the treatment of non-small cell lung cancer (NSCLC) with rearranged during transfection (RET) gene fusions. This article provides an update on the clinical trials, market analysis, and projections for this groundbreaking drug.

Clinical Trials Overview

ARROW Trial

The pivotal Phase 1/2 ARROW trial (NCT03037385) has been instrumental in establishing the efficacy and safety of pralsetinib. This open-label, single-arm study enrolled patients with locally advanced or metastatic RET fusion-positive NSCLC. The trial demonstrated an overall response rate (ORR) of 78% in treatment-naïve patients and 63% in those previously treated with platinum-based chemotherapy, with median durations of response (DOR) of 13.4 months and 38.8 months, respectively[1][3][4].

Key Efficacy Metrics

  • Overall Response Rate (ORR): 78% in treatment-naïve patients and 63% in those previously treated with platinum-based chemotherapy[1].
  • Duration of Response (DOR): Median DOR of 13.4 months in treatment-naïve patients and 38.8 months in previously treated patients[1].
  • Intracranial Response: Pralsetinib showed robust activity against brain metastases, a critical aspect for patients with NSCLC[3].

Safety Profile

The most common adverse reactions (≥ 25%) included musculoskeletal pain, constipation, hypertension, diarrhea, fatigue, edema, pyrexia, and cough. Most treatment-related adverse events were grade 1–2, indicating a manageable safety profile[1][4].

Regulatory Approvals

FDA Approval

On August 9, 2023, the FDA granted regular approval to pralsetinib for adult patients with metastatic RET fusion-positive NSCLC, following an initial accelerated approval on September 4, 2020. This conversion to regular approval was based on additional follow-up data that confirmed the durability of response[1].

European Commission Approval

The European Commission granted a conditional marketing authorization for pralsetinib in November 2021 for adult patients with RET fusion-positive advanced NSCLC who had not previously received a RET inhibitor. This decision was supported by data from the ARROW trial[5].

Market Analysis

Market Potential

Pralsetinib has significant market potential given the unmet need for effective treatments in RET fusion-positive NSCLC. The precision medicine approach, targeting a specific genetic mutation, positions pralsetinib as a valuable option for a subset of NSCLC patients.

Competitive Landscape

The NSCLC market is highly competitive, with several targeted therapies and immunotherapies available. However, pralsetinib's specificity for RET fusions and its demonstrated efficacy and safety profile differentiate it from other treatments. The drug's approval in both the U.S. and Europe further solidifies its market position[1][5].

Patient Population

RET fusions are present in approximately 1-2% of NSCLC cases, which, although a small percentage, still represents a significant number of patients globally. The approval of pralsetinib provides these patients with a targeted and effective treatment option that was previously lacking[3].

Ongoing and Future Clinical Trials

AcceleRET Lung Trial

The Phase 3 AcceleRET Lung trial (NCT04222972) is an international, open-label, randomized study comparing pralsetinib to standard of care (platinum-based chemotherapy with or without pembrolizumab) in first-line treatment of advanced or metastatic RET fusion-positive NSCLC. This trial aims to evaluate progression-free survival (PFS), overall survival (OS), ORR, and DOR, among other endpoints[3][4].

Market Projections

Revenue Growth

Given the strong clinical data and regulatory approvals, pralsetinib is expected to see significant revenue growth. The drug's ability to provide durable responses and its approval in major markets position it for substantial market penetration.

Expansion into New Indications

Future clinical trials may explore the use of pralsetinib in other RET fusion-positive cancers, potentially expanding its market reach beyond NSCLC.

Key Takeaways

  • Clinical Efficacy: Pralsetinib has demonstrated high ORR and durable DOR in RET fusion-positive NSCLC patients.
  • Regulatory Approvals: Approved by the FDA and European Commission, solidifying its market presence.
  • Market Potential: Significant market potential due to unmet needs in targeted therapy for RET fusion-positive NSCLC.
  • Ongoing Trials: Phase 3 AcceleRET Lung trial to further establish pralsetinib's efficacy in first-line treatment.

FAQs

What is pralsetinib and how does it work?

Pralsetinib is a potent and highly selective once-daily oral inhibitor of RET fusions and mutations, designed to target and inhibit the RET protein, which is involved in the growth and spread of certain cancers.

What are the common side effects of pralsetinib?

Common adverse reactions include musculoskeletal pain, constipation, hypertension, diarrhea, fatigue, edema, pyrexia, and cough.

How is pralsetinib administered?

Pralsetinib is taken orally once daily at a dose of 400 mg, on an empty stomach (no food intake for at least 2 hours before and at least 1 hour after taking the drug).

What is the significance of the ARROW trial in the approval of pralsetinib?

The ARROW trial provided pivotal data on the efficacy and safety of pralsetinib, demonstrating high ORR and durable DOR, which supported its regulatory approvals.

What are the future clinical trials for pralsetinib?

The Phase 3 AcceleRET Lung trial is ongoing, comparing pralsetinib to standard of care in first-line treatment of advanced or metastatic RET fusion-positive NSCLC.

Sources

  1. FDA Approves Pralsetinib for Non-Small Cell Lung Cancer with RET Gene Fusions. FDA.
  2. Pralsetinib (Gavreto). Canada's Drug Agency.
  3. Blueprint Medicines Announces Top-line Data for Pralsetinib and Initiates Rolling NDA Submission to FDA. PR Newswire.
  4. AcceleRET Lung: A Phase 3 Study of First-Line Pralsetinib in Patients with RET Fusion–Positive NSCLC. ASCO.
  5. Pralsetinib Approved in Europe for RET Fusion+ Advanced NSCLC. Onclive.

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