Pharma Pioneer

FDA Approves DepYmed's Phase 1 Trial for Rett Syndrome Drug DPM-1003

19 May 2024
2 min read

DepYmed Inc., a pharmaceutical firm specializing in the development of innovative treatments for a variety of diseases, has announced that it has obtained approval from the U.S. Food and Drug Administration (FDA) to begin phase 1 clinical trials for DPM-1003, a drug intended for the treatment of Rett Syndrome. The company, which is focused on targeting the protein tyrosine phosphatase (PTP) enzyme family, has achieved a significant milestone with this clearance, marking its transition to a clinical-stage entity.
Andreas Grill, CEO of DepYmed, highlighted the importance of this development, stating that it could pave the way for the creation of new treatments for a spectrum of diseases where PTP inhibition could be beneficial. These include Rett Syndrome, cancer, metabolic disorders, obesity, and Alzheimer's Disease. The company is eager to evaluate the safety and efficacy of DPM-1003 and to advance its development for Rett Syndrome and other ongoing projects.
DPM-1003 is a novel drug candidate that targets PTP1B, a key enzyme involved in the regulation of cell signaling pathways disrupted in numerous diseases. DepYmed has pioneered the development of orally bioavailable drugs that inhibit PTP1B, with one of its compounds demonstrating potential in preclinical Rett Syndrome models. The drug has received Orphan Drug status and conditional designation under the FDA's Rare Pediatric Disease program.
Rett Syndrome is a neurodevelopmental disorder predominantly affecting females, characterized by a regression in developmental skills and the onset of various symptoms such as autistic behaviors, breathing issues, and seizures. The syndrome is often linked to mutations in the MECP2 gene, which regulates PTP1B expression.
DepYmed, Inc., headquartered in New York, was established to leverage the scientific insights from the Tonks lab at Cold Spring Harbor Laboratory regarding PTP1B's role in human diseases. The company is developing a range of small molecule inhibitors targeting PTP1B for therapeutic use in Rett Syndrome, cancer, metabolic diseases, obesity, and Alzheimer's Disease. Additionally, DepYmed is working on a new class of copper-chelating molecules for diseases like Wilson disease and certain cancers, in collaboration with Cold Spring Harbor Laboratory. 

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!

图形用户界面, 文本, 应用程序, 聊天或短信

描述已自动生成

Recce Pharma Concludes Phase I/II Dosing for RECCE® 327 in UTI and Urosepsis Trial
Pharma Pioneer
3 min read
Recce Pharma Concludes Phase I/II Dosing for RECCE® 327 in UTI and Urosepsis Trial
19 May 2024
Recce Pharmaceuticals has recently completed a new phase of clinical trials for its lead drug candidate, RECCE® 327.
Read →
Promising Early Results: Deltacel-01 Trial Stabilizes Lung Tumor and Clears Brain Metastases in Patient Two
Pharma Pioneer
3 min read
Promising Early Results: Deltacel-01 Trial Stabilizes Lung Tumor and Clears Brain Metastases in Patient Two
19 May 2024
The trial is assessing the efficacy of Deltacel™ (KB-GDT-01), an allogeneic, readily available Gamma Delta T-cell (GDT) therapy for patients with stage 4 metastatic non-small cell lung cancer (NSCLC).
Read →
Jasper Initiates SPOTLIGHT Trial with First Patient Dosed for CIndU Treatment with Briquilimab
Pharma Pioneer
2 min read
Jasper Initiates SPOTLIGHT Trial with First Patient Dosed for CIndU Treatment with Briquilimab
19 May 2024
Biotechnology firm Jasper Therapeutics has initiated its Phase 1b/2a clinical trial, for the treatment of chronic inducible urticaria (CIndU) using a novel antibody, briquilimab.
Read →
Celularity Seeks FDA Orphan Drug Status for PDA-002 to Treat FSHD
Pharma Pioneer
2 min read
Celularity Seeks FDA Orphan Drug Status for PDA-002 to Treat FSHD
19 May 2024
Celularity Inc. has submitted an application to the U.S. Food and Drug Administration (FDA) for orphan drug designation for PDA-002, its placental-derived cell therapy.
Read →
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.