Onconic Therapeutics’ Nesuparib receives FDA orphan drug designation for small cell lung 바카라사이트 가입

[by Kang, In Hyo] Onconic Therapeutics' next-generation anti바카라사이트 가입 drug candidate, nesuparib, has further strengthened its clinical prominence, this time in the indication of small cell lung 바카라사이트 가입.

Onconic Therapeutics announced on February 24 that its next-generation dual-target synthetic lethal anti바카라사이트 가입 candidate, nesuparib, has been granted orphan drug designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of small cell lung 바카라사이트 가입 (SCLC).

Nesuparib previously received orphan drug designation from the FDA for pancreatic 바카라사이트 가입 in 2021 and for gastric 바카라사이트 가입 last year. With the recent addition of SCLC as an orphan-designated indication, its potential as a pan-tumor anti바카라사이트 가입 therapy has been further reinforced.

SCLC is widely recognized as a highly refractory malignancy characterized by rapid growth, early metastasis, and a high rate of recurrence. Treatment options remain limited, particularly in relapses following first-line treatment, and achieving durable long-term survival continues to pose a significant clinical challenge, creating a substantial medical need for novel therapies with differentiated mechanisms of action.

The FDA's Orphan 바카라사이트 가입 Designation is granted following a regulatory review of the requirements of the U.S. Rare Diseases Act, which evaluates criteria such as the scientific rationale supporting therapeutic potential and the corresponding necessity for development in rare diseases. Orphan-designated products represent a significant portion of newly approved 바카라사이트 가입s by the FDA, accounting for about half of all new 바카라사이트 가입 approvals. The designation confers seven years of market exclusivity upon approval and is widely regarded as a strategic tool in the global 바카라사이트 가입 development process.

Nesuparib is a dual-acting synthetic lethality-based anti바카라사이트 가입 agent designed to simultaneously inhibit poly ADP-ribose polymerase (PARP) and tankyrase (TNKS). It distinguishes itself for being a dual-targeting anti바카라사이트 가입 drug candidate different from existing PARP inhibitors.

PARP is a critical enzyme involved in the repair of intracellular DNA single-strand breaks. Its inhibition induces selective apoptosis in cancer cells characterized by high genomic instability. TNKS, which regulates the Wnt/β-catenin and Hippo signaling pathways, is known to play a central role in tumor proliferation, phenotypic plasticity, metastasis, and the development of treatment resistance.

The company explains that 바카라사이트 가입 has been engineered to concurrently suppress DNA damage repair mechanisms and tumor growth/ adaptive signaling pathways by simultaneously inhibiting both molecular axes.

Small cell lung 바카라사이트 가입 (SCLC) is characterized by a high initial response rate to standard chemotherapy, but the majority of patients experience rapid relapse. Following recurrence, effective treatment options are limited, making it a highly refractory malignancy. At the molecular level in particular, SCLC is marked by extreme genomic instability and a pronounced dependence on replication stress, largely attributable to the loss of TP53 and RB1 genes. These characteristics render SCLC as a 바카라사이트 가입 with high biological relevance for therapeutic strategies targeting the DNA damage response (DDR).

Although emerging treatment approaches, such as combination therapies incorporating immune checkpoint inhibitors, lurbinectedin, and DLL3 (Delta Like Ligand 3)-targeting immune cell activators, are currently under investigation, chemotherapy remains the standard of care, and treatment options capable of producing durable responses following relapse remain limited. Accordingly, there is a substantial unmet medical need for novel combination approaches or next-generation targeted therapies that concurrently address replication stress and tumor adaptive signaling. In this context, the company indicated that the successful development of 바카라사이트 가입 has garnered considerable attention.

In addition, Onconic Therapeutics is currently developing nesuparib in a Phase 2 pancreatic 바카라사이트 가입 study, a Phase 2 ovarian 바카라사이트 가입 study in combination with Celltrion's Vegzelma, an investigator-initiated Phase 2 trial in endometrial 바카라사이트 가입 in combination with the PD-1 antibody Keytruda, and a Phase 1b/2 study in gastric 바카라사이트 가입.

Nesuparib is raising high expectations as a potential pan-tumor anti바카라사이트 가입 drug in indications where treatment options have historically been lacking. The company underscored that the simultaneous advancement of four indications into Phase 2 clinical trials, coupled with the favorable safety and tolerability profile demonstrated in earlier Phase 1 trials, has contributed to growing interest in the global market. Onconic Therapeutics stated that it will continue to evaluate the potential of nesuparib with a focus on 바카라사이트 가입s characterized by high dependence on replication stress and DNA damage response (DDR), with the aim of further expanding its clinical indications.

"Recent reports indicate that small cell lung cancer (SCLC) exhibits increased activation of the Hippo and Wnt signaling pathways, along with a marked dependence on the DNA damage response during treatment. Nesuparib has received favorable feedback from the FDA regarding its potential to simultaneously inhibit two critical cancer cell survival axes, PARP and TNKS. We expect that this mechanism will offer a novel therapeutic option for patients with relapsed and treatment-resistant SCLC," an Onconic Therapeutics official said.