FDA Pipeline: Applications Accepted in Gynecologic Cancers, Targeted Therapies


Recently, the U.S. Food and Drug Administration (FDA) accepted a new drug application for niraparib in the treatment of certain pretreated gynecologic cancers; granted Fast Track designation to a T-cell product; accepted investigational new drug applications for a myelopreservation agent and an engineered toxin body; and cleared a scalp cooling system for the prevention of chemotherapy-associated hair loss.

FDA Accepts sNDA, Grants Priority Review to Niraparib for the Treatment of Gynecologic Cancers

The FDA accepted a supplemental new drug application (sNDA) for niraparib and granted the application Priority Review. The sNDA supports a potential new indication for the treatment of patients with advanced ovarian, fallopian tube, or primary peritoneal cancer who have been treated with three or more prior chemotherapy regimens, whose cancer is associated with either a BRCA mutation or a homologous recombination deficiency (HRD), and whose disease has progressed more than 6 months after the last platinum-based chemotherapy. Niraparib is a poly (ADP-ribose) polymerase inhibitor.

The sNDA is supported by data from the QUADRA trial. Data from QUADRA was recently published by Moore et al in The Lancet Oncology. QUADRA is a large multicenter, open-label, single-arm, phase II study that evaluated the safety and activity of niraparib in adult patients with relapsed, high-grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who were treated with three or more previous chemotherapy regimens. Patients received oral niraparib at 300 mg once daily continuously until disease progression. The primary objective was the proportion of patients achieving an investigator-assessed confirmed overall response in patients with HRD-positive tumors (including patients with and without BRCA mutations) sensitive to their last platinum-based therapy.

FDA Grants Fast Track Designation for TRQ-1501

The FDA granted Fast Track designation for TRQ-1501 (Deep IL-15 Primed T Cells). The Fast Track designation is for the treatment of relapsed or refractory solid tumors and lymphomas that express any of five tumor-associated antigens: PRAME, WT-1, SSX2, Survivin, and NY-ESO-1.

TRQ-1501 is an investigational immune cell therapy produced from a patient’s own T cells, which are primed to target multiple tumor-associated antigens and loaded with Deep IL-15 (a multimer of IL-15 cytokine) anchored to the T cells’ surface. A phase I/II clinical trial of TRQ-1501 in solid cancers and lymphoma is currently enrolling and will evaluate TRQ-1501 both as a single agent and in combination with pembrolizumab.

FDA Accepts IND Application for ALRN-6924 as a Myelopreservation Agent in Patients With p53-Mutated Cancer Treated With Chemotherapy

The FDA accepted an investigational new drug (IND) application for ALRN-6924 as a myelopreservation agent in patients with p53-mutated cancers treated with chemotherapy.

ALRN-6924 is a first-in-class, stabilized cell-permeating alpha-helical peptide that mimics the p53 tumor-suppressor protein to disrupt its interactions with its endogenous inhibitors, MDMX and MDM2. ALRN-6924 is currently being evaluated in multiple clinical trials for the treatment of a variety of cancers, including cancers with MDM2-amplified tumors. 

FDA Accepts IND Application for TAK-169, an Engineered Toxin Body Targeting CD38

The FDA accepted an IND application for TAK-169, an engineered toxin body targeting CD38. TAK-169 consists of a single-chain variable fragment with affinity for CD38, fused to the enzymatically active deimmunized Shiga-like toxin-A subunit (SLTA). The agent specifically binds and kills CD38-expressing cells in a manner consistent with SLTA-mediated cellular cytotoxicity.

TAK-169 has been specifically designed to avoid competition with and to overcome the primary mechanisms of tumor resistance to daratumumab and other monoclonal antibodies targeting CD38. It has been shown to be active in the presence of daratumumab. As such, TAK-169 may have the potential to be combined with approved CD38-targeted therapies.

An open-label phase I dose escalation and expansion study in patients with relapsed or refractory multiple myeloma is being planned.

Dignitana Receives FDA Clearance for DigniCap Delta Scalp-Cooling System

The FDA has cleared DigniCap Delta for use by U.S. health-care providers. The new device is the fourth generation of The DigniCap Scalp-Cooling System, and is indicated to prevent chemotherapy-induced hair loss in patients with solid tumors.

DigniCap Delta uses solid-state cooling for precise temperature control in an advanced and redesigned model of the 2015 FDA-cleared DigniCap Scalp-Cooling System. Other features of the new system include:

  • Single-patient use: The new cap system provides each patient with a flexible cooling wrap and an adjustable thermal cap designed to optimize scalp-cooling outcomes and minimize clinic storage needs
  • Smaller size: The new device is 54% smaller than the previous unit
  • Reduced nursing time: An intuitive interface potentially offers up to an 80% reduction in nursing time per patient infusion vs the existing device.

DigniCap Delta is now available to medical centers in the United States, and delivery to customers will begin in July. The current DigniCap device is already in use in 30 states in the United States and in 37 markets globally.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.