PALO ALTO - BridgeBio Pharma Inc . (NASDAQ:BBIO), a biopharmaceutical company specializing in genetic diseases and cancers, announced today that its licensing partner, Alexion (NASDAQ:ALXN), a part of AstraZeneca (NASDAQ:AZN) Rare Disease, has reported positive results from a Phase 3 trial in Japan for the experimental drug acoramidis. The trial focused on patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a condition characterized by the buildup of abnormal protein deposits in the heart.
The study, which was open-label and single-arm, demonstrated that acoramidis was well-tolerated over a 30-month treatment period, with no mortality reported. These findings are consistent with those from the global ATTRibute-CM Phase 3 trial, where acoramidis showed a significant impact on survival rates and cardiovascular hospitalizations.
Patients in the Japan trial were evaluated on various measures, including the six-minute walk test and the Kansas City Cardiomyopathy Questionnaire Overall Summary Score, which assesses quality of life. The data from this study will be presented at an upcoming medical meeting and is intended to support a regulatory submission to Japan's health authority.
Acoramidis is an investigational, oral small molecule designed to stabilize transthyretin (TTR), a transport protein in the blood. By stabilizing TTR, acoramidis aims to prevent the formation of amyloid fibrils, which are responsible for the progression of ATTR-CM.
This news is based on a press release statement.
InvestingPro Insights
As BridgeBio Pharma Inc. (NASDAQ:BBIO) continues to make strides in the development of acoramidis, it's important for investors to keep an eye on the financial health and market performance of the company. According to InvestingPro data, BridgeBio has a market capitalization of $5.79 billion, indicating a significant presence in the biopharmaceutical sector. Despite the promising clinical trial results, analysts have flagged concerns, with expectations of a sales decline this year and no profitability anticipated within the same timeframe. This is reflected in the company's negative P/E ratio of -9.65 for the last twelve months as of Q1 2023, underscoring the challenges it faces.
On the brighter side, InvestingPro Tips reveal that BridgeBio has a strong gross profit margin of 73.54% for the last twelve months, suggesting that it effectively controls its cost of goods sold relative to sales. Additionally, the company's liquid assets exceed its short-term obligations, which could provide financial flexibility in its operations and research initiatives. With a remarkable one-year price total return of 199.55%, investors have seen significant gains, although the stock has fared poorly over the last month with a price total return of -17.65%.
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