BBIO - BridgeBio Pharma's Breakthrough Potential In ATTR-CM Treatment Market

2023-07-17 14:56:32 ET

Summary

• BridgeBio Pharma, Inc. has reported encouraging results from its Phase 3 ATTRibute-CM study of acoramidis, a potential treatment for Transthyretin amyloid cardiomyopathy.
• The company's financial health is robust, with cash and equivalents increasing to $467.0 million, and operating costs and expenses decreasing to $128.0 million due to cost streamlining and reprioritized R&D programs.
• If approved, acoramidis could cater to a significant patient population previously untreated with disease-modifying therapy, potentially disrupting the current treatment landscape and competing with Pfizer's tafamidis.

Introduction

BridgeBio Pharma, Inc. ( BBIO ), a biopharmaceutical company founded in 2015, has been on a relentless mission to develop revolutionary medicines for genetically-driven cancers and genetic diseases. Their commitment to advancing genome sequencing and molecular biology research has led to practical, life-changing treatments. Over the years, BridgeBio Pharma has submitted 15 Investigational New Drug applications and gained two FDA approvals.

BridgeBio Pharma concluded a Phase 3 clinical trial for a potential treatment for Transthyretin ((TTR)) amyloidosis, known as acoramidis. Despite not meeting its primary endpoint in Part A, the drug displayed encouraging secondary results, demonstrating potential benefits on a 30-month primary endpoint.

Recent developments: Today, BridgeBio Pharma disclosed the latest findings from the Phase 3 ATTRibute-CM registrational trial of acoramidis, focusing on transthyretin amyloid cardiomyopathy (ATTR-CM).

Q1 2023 Earnings

Before we look at the data and its implications, let's review the firm's most recent quarterly report. As of Q1 2023 , BridgeBio Pharma's cash, cash equivalents, marketable securities, and restricted cash marginally increased to $467.0 million. This increase primarily resulted from net proceeds from a follow-on public offering and common stock issuance. The company reported a decrease in operating costs and expenses of $47.4 million, dropping to $128.0 million compared to Q1 2022. This reduction was mainly due to a decrease in research, development, and other expenses resulting from reprioritized R&D programs, lower selling, general, and administrative expenses due to cost streamlining, and a decrease in restructuring, impairment, and related charges. The company also reported a slight decrease in stock-based compensation expenses. All of these factors suggest that BridgeBio is continually exploring restructuring alternatives for improved operational efficiency and cost savings.

Clinical Trial Results and FDA Approval Process

BridgeBio Pharma reported encouraging results from its Phase 3 ATTRibute-CM study, evaluating the efficacy and safety of acoramidis, a potent oral small molecule stabilizer of transthyretin (TTR). This mechanism of action is designed to maximize TTR stabilization, a strategy believed to offer enhanced benefits to ATTR patients. The study findings revealed that acoramidis led to a significant 1.8 Win Ratio improvement in the primary endpoint, prioritizing all-cause mortality, cardiovascular-related hospitalization frequency, changes in NT-proBNP levels, and changes in 6-minute walk distance. The treatment group showed an 81% survival rate, a 25% relative risk reduction, and a 50% statistically significant reduction in cardiovascular-related hospitalizations.

Notably, exploratory post-hoc analysis demonstrated that acoramidis outperformed tafamidis by causing a 42% greater increase in serum TTR levels and a 92% improvement in median NT-proBNP. No safety issues were reported. Buoyed by these results, BridgeBio plans to submit an NDA to the FDA in late 2023, with regulatory filings in other markets expected in 2024.

Evolving Treatment Landscape for ATTR-CM

Due to the rapid progress in non-invasive diagnostic methods, the treatment options for ATTR-CM are significantly expanding. As the awareness of this disease grows, the estimated number of diagnosed ATTR-CM patients in the United States has risen from fewer than "5,000 in 2019 to over 30,000 in 2021," according to BridgeBio's estimates . This increase in diagnoses indicates a growing demand for therapeutic interventions targeting ATTR. If approved, acoramidis has the potential to cater to a significant patient population that has not been previously treated with disease-modifying therapy, establishing itself as a prominent contender in the ATTR-CM treatment market.

Existing Treatments and Market Competition

Having received FDA approval in 2019, Tafamidis , developed by Pfizer ( PFE ), is currently the preferred treatment for ATTR cardiomyopathy. Similar to acoramidis, this medication works by stabilizing transthyretin tetramers, potentially reducing the production of TTR amyloid. In 2022, Pfizer's tafamidis generated $2.4 billion in sales . However, other approaches such as liver transplantation and symptomatic management are also utilized. The emergence of small molecule stabilizers like Tafamidis and the potential inclusion of acoramidis represents a promising advancement in the treatment landscape for ATTR cardiomyopathy.

My Analysis & Recommendation

A thorough analysis of BridgeBio Pharma's current scenario reveals a company poised for substantial growth. The promising secondary results from the Phase 3 clinical trials for acoramidis, alongside the company's robust financial health and focus on operational efficiency, paint an optimistic picture for investors.

The growing number of diagnosed ATTR-CM patients in the U.S. indicates a burgeoning market for ATTR therapeutic interventions, including potential treatments like acoramidis. If approved, acoramidis could disrupt the current treatment landscape and compete effectively with Pfizer's tafamidis.

Given these factors, it is prudent for investors to anticipate the FDA's decision on acoramidis. Approval could trigger a rise in BridgeBio's stock value, solidifying the company's position as a significant player in the pharmaceutical industry. However, a rejection could have detrimental effects, although the company's strong financial health and diverse drug pipeline could provide a buffer.

Considering the potential growth and opportunities, my recommendation is a 'Buy' for BridgeBio Pharma. This recommendation is contingent upon close monitoring of the FDA's decision on acoramidis and awareness of the inherent risks in the pharmaceutical industry. Investors should also track the evolving competition with Pfizer's tafamidis, which could affect BridgeBio's growth trajectory in the coming months and years.

Risks to Thesis

When the facts change, I change my mind.

As with any investment, recommending a "buy" for BridgeBio Pharma does come with its share of risks. Here are some key points to consider:

• Regulatory Approval : One of the significant risks is the FDA's approval of acoramidis. While the Phase 3 trial results have been promising, the drug's approval is not guaranteed. Any delay or denial could negatively impact the company's stock price.

• Market Adoption: If acoramidis gets approved, its successful adoption in the market is another risk factor. The drug will be entering a market with established competitors like Pfizer's tafamidis, and the company's ability to gain a substantial market share is not guaranteed.

• R&D Outcomes: BridgeBio Pharma's future profitability largely depends on its R&D pipeline. Therefore, any setbacks in the company's other research and development programs could impact its overall performance.

• Financial Health: While the company's current financial health appears robust, unforeseen circumstances such as a downturn in the economy, increased operational costs, or a slowdown in investment could pose risks.

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