FDA Approves Multiple Investigational New Drug Applications


Bridging regulatory hurdles is an essential milestone for companies aiming to develop new treatments for a myriad of diseases and disorders. This week, the U.S. Food and Drug Administration (FDA) accepted Investigational New Drug Applications for multiple companies, clearing the way for clinical development.

Memgen – Texas-based Memgen received clearance to advance MEM-288, the company’s wholly-owned cancer immunotherapy candidate for the treating multiple solid tumors into the clinic. Memgen expects to begin screening patients by the end of the year for a Phase I first-in-human study.

The Phase I trial will be an open-label, dose-escalation study designed to evaluate the safety, tolerability, biologic activity, and anti-tumor effects of MEM-288, an oncolytic adenovirus encoding transgenes for human interferon beta (IFNß) and the company’s proprietary recombinant chimeric CD40 ligand. According to the company, in addition to non-small cell lung cancer (NSCLC), the trial may enroll patients with triple-negative breast cancer, pancreatic cancer, head and neck cancer, melanoma, cutaneous squamous-cell carcinoma, and Merkel cell carcinoma. Except for pancreatic cancer patients, the trial participants will be patients whose disease has progressed following treatment with anti-PD-1/PD-L1 therapy and who have tumor lesions accessible for injection.

Ikena Oncology – Boston-based Ikena will advance its TEAD inhibitor candidate, IK-930 into the clinic. The company will assess the asset as a potential targeted therapy for cancer patients that exhibit genetic alterations across the Hippo pathway. According to the company, IK-930 is designed to selectively bind TEAD and to disrupt TEAD-dependent transcription of key genes involved in cancer progression, metastases, and therapeutic resistance.

The Phase I study will include patients with tumor types with a high frequency of Hippo pathway alterations, including NF2-deficient malignant mesothelioma and some soft tissue sarcomas with YAP/TAZ genetic fusions. Ikena also plans to explore combinations of IK-930 with other targeted agents to treat solid tumors, such as EGFR-mutant NSCLC and KRAS-mutant solid tumors.

Theseus Pharmaceuticals – Also based in Massachusetts, Theseus received IND clearance for THE-630, the company’s lead development candidate, as a potential treatment for patients with gastrointestinal stromal tumors (GIST). THE-630 is a pan-variant inhibitor of the receptor tyrosine kinase KIT. The drug candidate is designed for patients with advanced GIST whose cancer has developed resistance to earlier lines of therapy.

According to the company, THE-630 “exhibits potent in vitro activity against all known classes of KIT activating and resistance mutations in GIST.”

Theseus intends to initiate a Phase I/II dose-escalation and expansion clinical trial that will include patients with previously treated advanced GIST. The study is expected to begin late in the fourth quarter of 2021 or in the first quarter of 2022.

Landos Biopharma – Virginia-based Landos has begun to assess LABP-104 as a potential treatment for rheumatoid arthritis following the FDA decision to accept the IND application. LABP-104 is a LANCL2 agonist. It was also recently cleared as a potential treatment for systemic lupus erythematosus. The company has initiated a Phase I study for both indications.

LABP-104 targets the anti-inflammatory receptor LANCL2, a novel mechanism for enhancing Treg function and decreasing TNF production. Topline results from the Phase I study are expected in the first half of 2022.

Gannex Pharma – China’s Gannex was cleared by the FDA to begin human trials of ASC43F, a first-in-class, fixed-dose combination (FDC) with dual targets of thyroid hormone receptor beta (THRβ) and farnesoid X receptor (FXR) for the treatment of non-alcoholic steatohepatitis (NASH).

ASC43F, a combination of ASC41 and ASC42, is an oral hepatic targeting THRβ agonist prodrug. Previous Phase I studies of ASC41 showed the drug can significantly reduce low-density lipoprotein cholesterol (LDL-C), triglyceride (TG), and total cholesterol (TC) in overweight and obese subjects with elevated LDL-C. Those are key characteristics of NASH.

Antegene Corporation – Also based in China, Antegene was cleared to begin human studies of ATG-101, a bi-specific monoclonal antibody in development as a potential treatment for metastatic/advanced solid tumors and B-cell non-Hodgkin’s lymphoma.

ATG-101 is a novel PD-L1/4-1BB bi-specific antibody. The Phase I trial will evaluate the safety and tolerability of ATG-101 in patients with advanced solid tumors and NHL. ATG-101 demonstrated significant anti-tumor activity in animal models of tumors that progressed on anti-PD-1/L1 treatment and showed a favorable safety profile in GLP toxicology studies, the company said.

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