BridgeBio Pharma, Inc. (NASDAQ:BBIO) ranks among the best biotech stocks to buy. UBS reaffirmed its Buy rating for BridgeBio Pharma, Inc. (NASDAQ:BBIO) on September 22, citing the company’s Attruby medication launch. The firm is optimistic about Attruby’s launch trajectory, citing a high number of new patient diagnoses. According to UBS, Attruby has the potential to become a major product that generates over $4 billion in long-term revenue, which is bolstered by growing data showing how it differs from r ...

Company Overview - Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) is a leading biotech company specializing in RNA interference (RNAi) therapeutics, focusing on treatments for rare and prevalent diseases, particularly transthyretin amyloidosis (ATTR) [1] Financial Performance - In Q2 2025, Amvuttra sales reached $492 million, exceeding the consensus estimate of $350 million, driven by expanded use for ATTR cardiomyopathy [2] - The company raised its 2025 revenue guidance for Amvuttra and Onpattro to $2.18–$2.28 billion, with total projected net revenues between $2.65–$2.8 billion [2] - Alnylam's market valuation has surpassed $50 billion, with shares increasing over 15% recently [2] Product Insights - Amvuttra's subcutaneous administration may improve patient adherence compared to oral competitors, priced at $476,000 annually for cardiomyopathy, and is increasingly recognized as a first-line therapy [3] - Commercial and Medicare payers are approving Amvuttra treatment without requiring prior use of competitor products, supporting sustainable growth in the U.S. market [3] Pipeline Development - Alnylam is advancing its pipeline, including RNAi therapeutics for cardiovascular diseases, such as zilebesiran for hypertension [4] - The company presented 12-month HELIOS-B Phase 3 data demonstrating sustained benefits of Amvuttra in treating ATTR cardiomyopathy [4] - Alnylam joined the Alliance for Genomic Discovery in September 2025 to leverage large-scale genomic datasets for accelerating target discovery and innovation in gene silencing therapies [4]

Core Insights - The PGIM Jennison Health Sciences Fund's second-quarter 2025 investor letter highlights significant volatility in equities, with the S&P 1500 Health Care Index declining by 6.9%, underperforming the S&P 500's 10.9% return [1] - Health care providers, life sciences tools, biotechnology, and pharmaceuticals underperformed the Index, while healthcare technology and medtech sectors showed gains [1] - Alnylam Pharmaceuticals, Inc. is emphasized as a key stock, with a one-month return of 1.34% and a 52-week gain of 66.94%, closing at $458.37 per share with a market capitalization of $60.083 billion [2][3] Company Analysis - Alnylam Pharmaceuticals focuses on RNA-based therapies, particularly RNA interference (RNAi), with a strong emphasis on treating conditions like Transthyretin Amyloidosis (ATTR) [3] - The company’s drug Amvuttra represents a novel approach in treating ATTR cardiomyopathy, offering a silencing mechanism that significantly reduces misfolded protein production, contrasting with existing treatments that stabilize the protein [3] - Early feedback on Amvuttra's launch has been positive, with expectations for continued strength in performance due to underdiagnosis of rare diseases and an expanding market as more treatments enter [3] - Alnylam has a robust pipeline across various therapeutic areas, indicating potential for becoming a multi-franchise company by the end of the decade, a growth opportunity not yet reflected in its stock price [3]

Financial Data and Key Metrics Changes - The company is preparing for the PDUFA date of September 22 for apategromab in SMA, indicating a significant milestone in its financial outlook [11] - The management has emphasized a strong cash position, with a runway extending into early 2027, allowing for strategic investments without immediate equity issuance [42][44] Business Line Data and Key Metrics Changes - The company has seen robust clinical development for ipilimumab in SMA, with a Phase III SAFIRE trial enrolling 188 patients, demonstrating clinically meaningful benefits [12][13] - The obesity program is set to share Phase II data from the EMBRAZE trial, focusing on the preservation of lean mass in patients receiving tirzepatide [28][29] Market Data and Key Metrics Changes - The company is under review with the FDA and the European Medicines Agency, with plans for a substantial launch in the U.S. and thoughtful expansion into Europe and Asia Pacific [25][26] - There are approximately 35,000 patients worldwide who have received at least one SMN targeted therapy, highlighting a significant market opportunity for the company's products [26][40] Company Strategy and Development Direction - The company aims to scale its growth into global commercialization, focusing on the approval of ipilimumab in multiple countries [3][25] - The strategy includes leveraging existing assets while exploring new indications for ipilimumab and SRK-439 in rare neuromuscular disorders [9][38] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of ipilimumab to reverse the trend of motor function loss in SMA patients, positioning it as a new standard of care [15][19] - The company is committed to making a meaningful difference for patients and ensuring that no patient is left behind in accessing their therapies [27][44] Other Important Information - The company is considering the implications of pricing strategies based on the rarity and severity of diseases like SMA, as well as the clinical benefits demonstrated [39][40] - The management is cautious about the investment in cardiometabolic and obesity indications, focusing instead on rare severe neuromuscular disorders [33][44] Q&A Session Summary Question: What are the expectations for the label regarding apategromab in SMA? - Management expects a broad label based on the robust data from the Phase III SAFIRE trial and the consistency of results across patient subgroups [12][13] Question: How does the drug fit into the existing treatment paradigm for SMA? - The company believes ipilimumab can provide significant benefits beyond existing SMN targeted therapies, particularly for patients who have started to lose motor function again [14][15] Question: What is the company's strategy for the obesity program? - The company plans to present Phase II data from the EMBRAZE trial, focusing on the preservation of lean mass in patients receiving tirzepatide [28][29] Question: How does the company view the regulatory pathway for obesity treatments? - Management acknowledges the evolving view of the FDA and the need to demonstrate both weight loss and preservation of lean mass in future trials [32][33] Question: What is the current cash position and runway for the company? - The company has a strong cash position with a runway extending into early 2027, allowing for strategic investments without immediate equity issuance [42][44]

Core Insights - Alnylam Pharmaceuticals has received European Commission approval for the label expansion of its RNAi therapeutic Amvuttra to treat wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy [1][9] - Year-to-date, Alnylam's stock has increased by 29.1%, significantly outperforming the industry, which has seen a decline of 0.9% [2] Approval Details - The European Commission's approval was based on positive results from the late-stage HELIOS-B study, where Amvuttra met all 10 pre-specified primary and secondary endpoints [3][9] - The HELIOS-B trial demonstrated statistically significant reductions in all-cause mortality and recurrent cardiovascular events, along with major improvements in functional capacity, health status, and heart failure symptoms [4][9] Competitive Advantage - Amvuttra is administered quarterly via subcutaneous injection, providing an advantage over existing therapies that require daily oral dosing [5] - The drug's novel RNAi mechanism allows for rapid knockdown of transthyretin (TTR), addressing the disease at its source [6] Market Impact - The approval in the EU follows similar approvals in the United States and Brazil, expanding Amvuttra's global footprint [6] - Alnylam reported better-than-expected first-quarter 2025 results, with revenue growth driven by increasing Amvuttra sales due to heightened patient demand [10][11]