Pralsetinib

Unassigned

New Medicines

Gavreto · RET-altered non-small cell lung cancer (NSCLC) following chemotherapy

Information

Gavreto
New molecular entity
Blueprint Medicines Corporation
Blueprint Medicines Corporation

Development and Regulatory status

Pre-registration (Filed)
Pre-registration (Filed)
Phase III Clinical Trials
Jul 20 · Roche plans to acquire rights to pralsetinib and gain an exclusive license to sell pralsetinib outside of the U.S. and China.[5]
Jun 20 · Currently pre-registration in EU. Has been filed using the centralised procedure [4].
Jun 19 · The company is planning to file in 2020 [2].
May 19 · Breakthrough therapy designation awarded in US for NSCLC that has progressed following platinum-based chemotherapy [1].
Apr 18 · Orphan drug designation in US awarded [1].

Category

Proto-oncogene protein C RET inhibitor
RET rearrangements have been seen in 1 to 2% of adenocarcinomas, occurring more frequently in the young and in never-smokers [3].
RET-altered non-small cell lung cancer (NSCLC) following chemotherapy
Oral

Trial or other data

Jul 20 · Data from PII Arrow trial (NCT03037385) and PIII AcceleRET trial (NCT04222972) are being used in support of licence applications. Interim results from Arrow trial were presented at ASCO 2020. A paper was submitted to NEJM. 1 - Gainor et al. Registrational Dataset from the Phase 1/2 ARROW Trial of Pralsetinib (BLU-667) in Patients with Advanced RETFusion+ Non-Small Cell Lung Cancer (NSCLC). Poster Number 9515. Presented at ASCO 2020, May 29 –June 2, 2020, Virtual Format. [4].
Jun 19 · Preliminary top line PII data looking at 35 patients with NSCLC show that 90% had "some tumour shrinkage", and 60% of patients were responders to treatment. Tumours that had spread to the brain shrank in seven out of nine patients. NSCLC is only one indication in this trial; other patients have thyroid cancer, pancreatic cancer and bile duct cancer [2].

Gavreto · Medullary advanced/metastatic RET mutant (MTC) and RET fusion-positive thyroid cancer

Information

Gavreto
New molecular entity
Blueprint Medicines Corporation
Blueprint Medicines Corporation

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Pre-registration (Filed)
Jul 20 · Blueprint Medicines announce submission of NDA to FDA for treatment of advanced RET mutant and RET fusion-positive thyroid cancers [1].

Category

Proto-oncogene protein C RET inhibitor
There are around 3,500 new thyroid cancer cases in the UK every year [3]. MTC accounts for approximately 5-8% of all thyroid cancer. Clinically, MTC is mainly sporadic (70-80%), but hereditary pattern is present in 20-30% of cases, transmitted as an autosomal dominant trait. Somatic RET mutations can be detected in tumour tissue of 40-60% of sporadic MTC patients [4].
Medullary advanced/metastatic RET mutant (MTC) and RET fusion-positive thyroid cancer
Oral

Trial or other data

Jul 20 · Estimated primary completion date of PIII ARROW study (NCT03037385) is Dec 21 [2].