FDA Grants Fast Track Designation to Pidnarulex in BRCA1/2, PALB2+ Ovarian and Breast Cancer


Fast track designation was granted by the FDA for pidnarulex in patients with BRCA1/2 or PALB2 mutations with ovarian or breast cancer.

The FDA has granted fast track designation to pidnarulex (CX-5461) for the treatment of ovarian and breast cancer with BRCA1/2, PALB2, or other homologues recombination deficiency (HRD) mutations, according to a press release from developer Senhwa Biosciences.

In previous phase 1 study, pidnarulex yielded clinically significant and lasting benefit for those with BRCA1/2- and PALB2-mutant disease, and who were resistant other platinum-based therapies. Currently, a phase 1b trial (NCT04890613) is being conducted to determine an acceptable dose for patients with solid tumors such as breast, ovarian, pancreatic, and prostate cancer, with specific mutations such as BRCA2 and PALB2 for solid tumors and BRCA1 and other HR gene mutations for those with ovarian cancer.

"We are excited to receive fast track designation and look forward to working closely with the US FDA to accelerate the development of pidnarulex, aiming to bring a meaningful treatment to patients with breast and ovarian cancer whose tumors have BRCA1/2, PALB2, or other HRD mutations," Mei-Hui Kuo, acting chief executive officer at Senhwa Biosciences, said in a press release.

A total of 52 patients are estimated to be enrolled in the non-randomized study. Patients will either be included in the main cohort for those with histologically confirmed solid tumors with germline BRCA2 or PALB2 mutations, or the exploratory cohort for those with ovarian cancer and pathogenic BRCA1 and/or other HRD mutations.

Sixteen patients from the main cohort and 10 from the exploratory cohort will be given pidnarulex at 250 mg/m2 for 60 minutes via intravenous infusion on day 1 and 8 of each cycle. Investigators will add an additional 16 and 10 patients to the respective arms upon review of the data and the safety profile of pidnarulex. These patients will receive pidnarulex at 325 mg/m2 for 60 minutes intravenously on day 1 and 8 of each cycle.

The primary end point of this study will be to determine the recommended phase 2 dose of pidnarulex. This will be assessed by a safety review every 4 weeks from the first patient’s enrollment until all patients have been enrolled and evaluated for toxicity up to 24 months. Secondary end points included adverse effects, objective response rate, and patient reported outcomes.

Eligibility criteria in the main cohort included confirmed malignancy of the pancreas, prostate, breast, or ovary; documented evidence of germline mutations; and measurable disease via RECIST 1.1 criteria. In the exploratory cohort patients needed to have confirmed ovarian, fallopian tube, or primary peritoneal cancer; documented evidence of germline mutation; measurable disease via RECISTS 1.1 criteria; and have either platinum sensitive with no evidence of disease progression and/or be platinum resistant within 6 months of the last dose of platinum-based chemotherapy.

Additional inclusion criteria for all patients required an ECOG score of 2 or less within 24 days of registration, radiographically documented disease progression within 28 days of registration, and adequate bone marrow, renal, and hepatic function per local laboratory ranges within 14 days of registration.


Senhwa’s pidnarulex received US FDA fast track designation for the treatment of solid tumors with BRCA1/2, PALB2, and other HR gene mutations. News Release. SenhwaBiosciences. January 25, 2022. Accessed January 28, 2022. https://yhoo.it/3s0CNAU

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