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BridgeBio Pharma and Affiliate Venthera Announce Dosing of First Patient in Phase 1/2 Clinical Trial of BBP-681 for Venous, Lymphatic, and Venolymphatic Malformations
First-in-human Phase 1/2 trial in patients with venous malformations (VMs), lymphatic malformations (LMs), and venolymphatic malformations (VLMs) associated
About this update from Bridgebio Pharma, Inc.
[{"type":"text","content":"First-in-human Phase 1/2 trial in patients with venous malformations (VMs), lymphatic malformations (LMs), and venolymphatic malformations (VLMs) associated with PIK3CA or TEK mutationsClinical trial enrolling patients across multiple sites in the United States PALO ALTO, Calif., Feb. 02, 2021 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) and affiliate Venthera, which is focused on developing new treatment options for patients with rare vascular anomalies, today announced that the first patient has been dosed in its Phase 1/2 clinical trial of BBP-681 (also known as VT30 Topical Gel). This novel investigational therapy is designed to target the genetic drivers of venous (VM), lymphatic (LM), and venolymphatic malformations (VLM) at their source by delivering a potent PI3Kα inhibitor directly to affected tissue. The majority of these cutaneous vascular malformations are driven by somatic mutations in the PIK3CA and TEK genes. These mutations result in overactivation of the PI3K pathway and have been identified as the root cause of the vast majority of VMs, LMs, and VLMs. Because BBP-681 is applied directly to skin lesions, it may minimize complications seen with systemic treatments. Part 1 of the trial is a four-week treatment, open-label dose escalation study which will determine the dose and regimen of BBP-681 to be carried into Part 2 of the protocol. Part 2 is a 12-week treatment, randomized, placebo-controlled, double-blind, safety and exploratory efficacy study of topically administered BBP-681. People living with VMs, LMs, and VLMs have significant unmet medical needs related to pain, functional impairment, and disfigurement. Current treatment options include laser ablation, and invasive procedures such as sclerotherapy and surgical excision. BBP-681 is the first potential therapy designed specifically to address the needs of people living with cutaneous forms of these lesions. There are 117,000 people estimated to be living with cutaneous VMs, LMs, and VLMs in the United States and Europe. “We are dedicated to giving hope to patients by developing new treatment options and working to improve mobility and alleviate discomfort of people living with cutaneous VMs, LMs, and VLMs. The initiation of this study represents an important milestone for the patient community as we translate cutting-edge science into a cli...