, Inc. (NASDAQ:ARWR) on Tuesday shared interim results from two Phase 1/2a trials of ARO-INHBE and ARO-ALK7, the company’s investigational RNA interference (RNAi) therapeutics for obesity.Preliminary results showed meaningful reductions in multiple key measures, including visceral fat, total fat, and liver fat.What Is ARO-INHBE and ARO-ALK7 Therapy?Unlike current GLP-1 drugs (like Wegovy or Zepbound) that primarily reduce appetite, these candidates aim to change how the body stores and burns fat by silencin ...

Alnylam Pharmaceuticals is set to invest $250m in the expansion of its facility in Norton, Massachusetts, US, to meet the growing global demand for RNA interference (RNAi) therapeutics. This is claimed to become the first fully dedicated small-interfering RNA (siRNA) enzymatic-ligation manufacturing facility. The facility expansion is intended to increase capacity, lower production expenses, and enable support for future medicine launches from the company’s development pipeline. As part of this, the co ...

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]

Core Insights - Sarepta Therapeutics (SRPT) has sold at least $174 million worth of its equity investment in Arrowhead Pharmaceuticals to fund a $100 million milestone payment related to the phase I/II study of SRP-1003 for treating myotonic dystrophy type 1 (DM1) [1][4][5] - Following the announcement, Sarepta's shares rose approximately 9%, although the stock has declined 83.2% year-to-date compared to a 2% decline in the industry [1][4] Company Developments - Sarepta entered an exclusive global licensing and collaboration agreement with Arrowhead Pharmaceuticals in February 2025, acquiring rights to four investigational RNA interference therapeutics [3] - The decision to sell equity in Arrowhead was driven by a milestone payment obligation due to the successful early enrollment and safety review of SRP-1003 [4][6] - Sarepta has sold 9.26 million shares of Arrowhead in a private sale and expects to receive at least $174 million in cash proceeds [5] Financial and Operational Challenges - The company is facing scrutiny from investors due to three patient deaths linked to its gene therapy treatments, which were attributed to acute liver failure [6][7] - Following these incidents, Sarepta suspended dosing of Elevidys for non-ambulatory patients but has since resumed shipments after FDA clearance for ambulatory patients [8][9] - To address safety concerns, Sarepta is developing a new protocol for Elevidys administration in non-ambulatory patients and plans to submit findings to the FDA [9] Strategic Restructuring - Last month, Sarepta initiated a restructuring plan aimed at saving nearly $400 million annually starting in 2026, which includes laying off 36% of its workforce [10] - The company is now focusing on its siRNA pipeline acquired from Arrowhead Pharmaceuticals as part of its strategic shift [10]

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]