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 - Novartis announced positive results from the V-DIFFERENCE Phase IV study, demonstrating the efficacy of Leqvio (inclisiran) in lowering LDL-C levels in patients with high cholesterol who did not meet guideline-recommended targets [1][2][4] Group 1: Study Results - After 90 days of treatment, 85% of patients on Leqvio achieved their LDL-C targets compared to 31% on placebo, with statistical significance (p<0.0001) [2][3] - Significant benefits were observed as early as 30 days, with 81% of patients achieving LDL-C targets [2] - Leqvio reduced LDL-C levels by an average of 59% after 360 days, outperforming placebo by 35% (p<0.0001) [3][9] Group 2: Patient Outcomes - The study showed that patients receiving Leqvio were 43% less likely to experience muscle-related adverse events compared to those on placebo (p<0.0001) [3][7] - Improvements in pain-related quality-of-life scores were also reported among patients treated with Leqvio [3] Group 3: Clinical Significance - V-DIFFERENCE is the largest LDL-C lowering study with Leqvio to date, focusing on patient-centered outcomes [3][10] - The study included 1,770 individuals, randomized to receive either Leqvio plus LLT or placebo plus LLT [8][9] Group 4: Broader Context - Leqvio is the first and only small interfering RNA therapy approved in over 100 countries, including the US, EU, Japan, and China [5][6] - The VictORION clinical program, which includes V-DIFFERENCE, encompasses over 60,000 patients across more than 30 trials, aiming to expand evidence for LDL-C reduction [10]

Key Takeaways Sarepta Therapeutics (SRPT) announced that it has sold at least $174 million worth of its equity investment in Arrowhead Pharmaceuticals to help fund a milestone payment to the latter. Shares of Sarepta rose around 9% on Wednesday in response to the announcement. Year to date, the stock has plunged 83.2% compared with the industry's 2% decline. Image Source: Zacks Investment Research SRPT's Deal with Arrowhead SRPT Stock on Investors' Radar Sarepta has been facing a lot of heat from investors ...

Core Viewpoint - Arrowhead Pharmaceuticals reported significant misses in both GAAP revenue and earnings for Q3 FY2025, highlighting ongoing financial volatility despite progress in its clinical pipeline [1][5][6] Financial Performance - Q3 FY2025 GAAP revenue was $27.8 million, missing analyst estimates of $46.8 million by nearly 41% [5] - GAAP earnings per share for Q3 FY2025 were $(1.26), falling short of the $(0.95) forecast by $0.31 [1][5] - Operating loss for Q3 FY2025 was $(165.6 million), an improvement of 6% compared to $(176.1 million) in Q3 FY2024 [2][6] - Research and development expenses increased by 6.6% year-over-year to $162.4 million [2][6] - Total cash resources rose to $900.4 million, a 32.1% increase from $681.0 million in Q3 FY2024 [2][7] Company Overview and Strategy - Arrowhead Pharmaceuticals focuses on RNA interference (RNAi) therapeutics, targeting diseases with significant unmet needs using its TRiMTM platform [3][4] - The company is advancing its drug pipeline with an emphasis on late-stage development and regulatory approvals, while building strategic partnerships with larger pharmaceutical companies [4] Product Pipeline Progress - Plozasiran, Arrowhead's leading RNAi candidate, received NDA acceptance from the FDA for familial chylomicronemia syndrome, with a PDUFA target date of November 18, 2025 [8][11] - Phase 3 studies for severe hypertriglyceridemia are ongoing, with data expected in mid-2026 [8][11] - Zodasiran, targeting rare cholesterol disorders, has commenced its Phase 3 trial [12] - The obesity portfolio includes ARO-INHBE and ARO-ALK7, which are in Phase 1/2 studies [13] Strategic Partnerships and Milestones - Visirna, a subsidiary, entered a deal with Sanofi for rights to four cardiometabolic programs, resulting in immediate payments of $130 million and potential milestones of up to $265 million [9] - The company anticipates additional milestone payments from Sarepta Therapeutics, totaling up to $200 million by the end of 2025 [9] Commercial Readiness - Arrowhead is preparing for the potential launch of plozasiran by expanding its U.S. sales force and engaging with payers [10] - The company is focused on rare disease awareness and patient identification, crucial for the target population of plozasiran [10] Outlook and Guidance - Management indicated that Arrowhead is funded through 2028, focusing on regulatory approval for plozasiran and commercialization efforts [15] - Key near-term catalysts include pivotal SHTG data readout in mid-2026 and potential partnership milestones within the next twelve months [15]

Core Insights - Bio-Path Holdings, Inc. is a clinical-stage biotechnology company focused on RNA interference therapeutics using its DNAbilize® platform [1] - The lead candidate, prexigebersen (BP1001), is in Phase 2 trials for acute myeloid leukemia (AML) and shows potential for FDA approval [7] - Bio-Path is advancing its pipeline with multiple candidates targeting various cancers and metabolic diseases [1][7] Company Overview - Bio-Path Holdings, Inc. is based in Bellaire, Texas and specializes in RNAi therapeutics [1] - The company employs a proprietary liposomal delivery system to enhance drug stability and cellular uptake while reducing toxicity [1] Pipeline Development - Prexigebersen (BP1001) targets the Grb2 protein and is currently in Phase 2 trials for AML [1] - A modified version, BP1001-A, is in Phase 1/1b trials for solid tumors and has shown early efficacy [7] - Another candidate, BP1002, targets the Bcl-2 protein for blood cancers and solid tumors [1] - Bio-Path is preparing an IND application for BP1003, a STAT3 inhibitor for pancreatic cancer [1]