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LONDON - Biodexa Pharmaceuticals PLC (NASDAQ:BDRX), a micro-cap biopharmaceutical company with a market capitalization of $3.61 million specializing in treatments for diseases with unmet medical needs, announced today that the European Commission (EC) has awarded Orphan Drug Designation to its drug eRapa for the treatment of familial adenomatous polyposis (FAP), a rare and potentially cancerous colon condition. According to InvestingPro analysis, the company maintains a current ratio of 1.75, indicating adequate short-term liquidity despite its development-stage status.
This regulatory milestone in Europe follows a similar designation granted by the U.S. Food and Drug Administration (FDA) in 2019. The Orphan Drug Designation by the EC is based on a positive opinion from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP). Such status is intended to facilitate drug development for rare diseases, offering benefits like protocol assistance and potential fee reductions.
eRapa, an oral tablet formulation of rapamycin known for inhibiting the mTOR pathway involved in cellular growth and tumor development, is poised to enter a Phase 3 study. This pivotal trial is a double-blind, placebo-controlled study involving 168 patients across approximately 30 clinical sites in the U.S. and Europe. The U.S. part of the study will be conducted by LumaBridge, while Precision for Medicine LLC will handle the European segment.
The Phase 3 study is supported by a substantial grant from the Cancer Prevention Research Institute of Texas (CPRIT) and a matching company fund, both of which have been fully paid into escrow. InvestingPro data reveals that while the company holds more cash than debt on its balance sheet, it is quickly burning through its cash reserves - one of 13 key insights available to subscribers.
FAP is characterized by the growth of numerous polyps in the colon and rectum, typically manifesting in the mid-teens. If left untreated, it can progress to colorectal cancer. Currently, there are no approved drugs for FAP, with surgery being the standard care. The addressable market for eRapa in treating FAP is estimated to be around $7.3 billion, considering the prevalence rates and the high cost of orphan drugs in the U.S. With analysts setting a target price of $17.82, significantly above current trading levels, investors seeking detailed valuation metrics can access comprehensive analysis through InvestingPro.
Biodexa’s CEO and CFO, Stephen Stamp, expressed the company’s commitment to advancing eRapa’s development and providing access to patients in need. The Orphan Drug Designation is a crucial step in securing marketing authorization within the European Union.
This announcement is based on a press release statement from Biodexa Pharmaceuticals PLC.
In other recent news, Biodexa Pharmaceuticals has announced significant progress in its preparations for a Phase 3 trial of eRapa, a drug aimed at treating familial adenomatous polyposis (FAP). The company received Fast Track designation from the FDA and held a productive Type C meeting to finalize the Phase 3 protocol. This trial is set to involve 168 patients across approximately 30 clinical sites in the U.S. and Europe, with LumaBridge managing the U.S. component and Precision for Medicine overseeing the European segment. The study, expected to commence next quarter, is substantially funded by a $17 million grant from the Cancer Prevention Research Institute of Texas, with an additional $8.5 million contributed by Biodexa. The Phase 3 trial will be a double-blind placebo-controlled study and is crucial, given the absence of approved treatments for FAP. Biodexa’s eRapa, an oral tablet formulation of rapamycin, has shown promise in reducing polyp burden and preventing disease progression in previous trials. The company is also working on treatments for other conditions, including Non-Muscle Invasive Bladder Cancer and rare brain cancers. These developments reflect Biodexa’s ongoing efforts to address diseases with significant unmet medical needs.
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