For companies aiming to develop new treatments for countless diseases and obstacles, crossing regulatory barriers is an important milestone. This week, the U.S. Food and Drug Administration (FDA) accepted research drug applications from a number of companies, clearing the initial obstacles for the next step of clinical supervision of these therapies.

1. Memgen

A few days ago, Memgen, headquartered in Texas, USA, obtained a license. In the future, the company will advance the cancer immunotherapy candidate MEM-288 into the clinic for the treatment of a variety of solid tumors. Memgen expects to start recruiting patients before the end of this year for the first phase I human study. This phase I trial will be an open-label, dose-escalation study. MEM-288 is an oncolytic adenovirus encoding human interferon beta transgene 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 also recruit cancer patients such as triple-negative breast cancer, pancreatic cancer, head and neck cancer, melanoma, and skin squamous cell carcinoma. In addition to patients with pancreatic cancer, trial participants will be required to receive anti-PD-1/PD-L1 treatment, patients who have advanced disease and can be injected.

2. Ikena Oncology

The US FDA approval will also prompt Boston-based Ikena to advance its TEAD inhibitor drug candidate IK-930 into the clinic. The company will evaluate the asset as a potential targeted therapy for cancer patients who exhibit genetic changes in the Hippo pathway. According to the company, IK-930 works by selectively binding TEAD and disrupting the TEAD-dependent transcription of key genes involved in cancer progression, metastasis, and treatment resistance. The phase I study will recruit patients with tumor types with high frequency of changes in the Hippo pathway, including patients with malignant mesothelioma lacking NF2 and some patients with soft tissue sarcoma with YAP/TAZ gene fusion. Ikena also plans to study the efficacy of the combination of IK-930 and other targeted drugs in the treatment of solid tumors, such as EGFR-mutated NSCLC and KRAS-mutated solid tumors.

3. Theseus Pharmaceuticals

Theseus Pharmaceuticals' main development candidate drug THE-630 has also received IND approval from the US FDA, supporting its use as a potential treatment for patients with gastrointestinal stromal tumor (GIST). THE-630 is a pan-variant inhibitor of receptor tyrosine kinase KIT. The drug candidate is designed for advanced GIST patients whose cancer conditions have developed resistance to early treatments. According to the company, the therapy “shows strong in vitro activity against all known types of KIT activation and resistance mutations in GIST.” Theseus intends to initiate a phase I/II dose escalation and expansion clinical trial, which will include For patients with advanced GIST who have previously received treatment, the study is expected to start at the end of the fourth quarter of 2021 or the first quarter of 2022.

4. Landos Biopharma

After the FDA decided to accept the IND application, Landos, Virginia, has begun to evaluate the efficacy of LBP-104 as a potential treatment for rheumatoid arthritis. LBP-104 is a LANCL2 agonist, and it has recently been supported as a potential treatment for systemic lupus erythematosus. The company has initiated phase I studies for these two indications. LABP-104 targets the anti-inflammatory receptor LANCL2, which has a new mechanism to enhance Treg function and reduce TNF production. The top-line results of the first phase of the study are expected to be announced in the first half of 2022.

5. Gannex Pharma

China Gannex obtained FDA approval to start human trials of ASC43F. ASC43F is a first-in-class fixed-dose combination (FDC) with dual targets of thyroid hormone receptor β (THR β) and farnesol X receptor (FXR), which can be used to treat non- Alcoholic steatohepatitis (NASH). ASC43F is a combination of ASC41 and ASC42, an oral liver-targeted THRβ agonist prodrug. ASC41 has confirmed in a previous phase I study that the drug can significantly reduce low-density lipoprotein cholesterol (LDL-C), triglycerides (TG) and total cholesterol in overweight and obese subjects with elevated LDL-C ( TC), and these are the key characteristics of NASH patients.

5. WuXi AppTec

WuXi AppTec, also located in China, has also been approved to start human research on ATG-101. ATG-101 is a bispecific monoclonal antibody under development that can be used as a potential treatment for metastatic/advanced solid tumors and B-cell non-Hodgkin's lymphoma. ATG-101 is a new PD-L1/4-1BB bispecific antibody. The upcoming phase I trial will evaluate the safety and tolerability of ATG-101 in patients with advanced solid tumors and NHL. The company said that ATG-101 has shown significant anti-tumor activity in tumor animal models that have progressed in anti-PD-1/L1 therapy, and has shown good safety in GLP toxicology studies.