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Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) is a leading clinical-stage biotechnology company dedicated to developing innovative medicines that address intractable diseases through gene silencing. The company utilizes an extensive array of RNA chemistries and efficient delivery systems to harness the power of RNA interference (RNAi) mechanisms. This approach allows for the rapid, deep, and durable knockdown of target genes, thereby reducing the production of specific proteins that cause diseases.
Arrowhead's groundbreaking therapies are at the forefront of genetic-based treatments, presenting potential life-changing solutions for patients. Notable among their technologies is the Targeted RNAi Molecule (TRiMTM) platform. This platform employs ligand-mediated delivery for tissue-specific targeting while maintaining structural simplicity. The TRiMTM platform is built upon over a decade of research in actively targeted drug delivery, offering advantages such as simplified manufacturing processes and enhanced specificity.
The company's diverse portfolio includes promising treatments for conditions such as Hepatitis B, Alpha-1 Antitrypsin Deficiency, thrombosis and angioedema, clear-cell and renal cell carcinoma, and cardiovascular diseases. Arrowhead is continuously engaged in advancing its clinical programs and forging strategic partnerships to bring its novel therapies to market.
With headquarters in Pasadena, California, Arrowhead Pharmaceuticals remains committed to transforming the landscape of genetic medicine and improving patient outcomes worldwide. Their dedication to scientific innovation and patient care positions them as a pivotal player in the biotechnology sector.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) recently announced interim results from its ongoing Phase 1/2 study of ARO-RAGE, an RNA interference therapeutic aimed at treating inflammatory pulmonary diseases like asthma. The study reported a mean maximum reduction of 80% in serum soluble RAGE (sRAGE) levels after two doses, with some patients experiencing reductions of up to 90%. The pharmacologic effect lasted at least 6 weeks following the 92 mg dose. No serious adverse events were reported, indicating promising safety and tolerability. Data from the study will be further presented at the upcoming R&D Day on June 1, 2023. This represents a significant step for Arrowhead's therapeutic pipeline, showcasing the potential of its proprietary TRiM platform.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) will host a webcast and conference call on May 2, 2023, at 4:30 p.m. ET to discuss its financial results for the fiscal 2023 second quarter ended March 31, 2023. Investors can access the live webcast on the Company's website, with a replay available two hours after the call. The company is known for developing RNA interference-based medicines targeting intractable diseases through innovative gene-silencing technologies. Details of the call can be found on their investor relations page.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) will host a Research & Development Day on June 1, 2023, at 9 a.m. ET in New York City. The event will focus on the company's aim to expand its RNAi therapeutics pipeline to 20 clinical products by 2025. Key advancements include the expanded TRiM platform for central nervous system (CNS) delivery, particularly the ARO-SOD1 candidate, targeting ALS linked to SOD1 mutations, with a CTA filing expected by Q3 2023. The R&D Day will feature expert presentations on cardiometabolic and pulmonary diseases, and updates on various clinical programs, including ARO-APOC3 and ARO-ANG3. Attendance is limited, and registration is required.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) announced the dosing of the first patient in Takeda’s Phase 3 REDWOOD study for fazirsiran (TAK-999/ARO-AAT), targeting alpha-1 antitrypsin deficiency-associated liver disease (AATD-LD). This event unlocks a $40 million milestone payment from Takeda to Arrowhead. Fazirsiran, an investigational RNA interference therapy, aims to reduce the production of the harmful Z-AAT protein. The study, involving 160 patients, evaluates the drug's efficacy and safety. Fazirsiran has previously received Breakthrough and Orphan Drug Designations from the FDA.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) has achieved a $30 million milestone from GSK (LSE/NYSE: GSK) following the initiation of GSK4532990's Phase 2b trial for treating non-alcoholic steatohepatitis (NASH). This investigational RNA interference therapeutic was licensed to GSK in November 2021, with Arrowhead retaining rights for Greater China. The trial, known as HORIZON (NCT05583344), will assess the drug's efficacy and safety in up to 246 participants with pre-cirrhotic NASH. GSK4532990 targets HSD17B13, a gene linked to metabolic protection against NASH.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) announced on March 29, 2023, that its Compensation Committee approved inducement grants for 31 new employees, totaling up to 46,050 restricted stock units. These grants, which incentivize new hires, are outside the company’s stockholder-approved equity incentive plans and will vest over four years. Arrowhead specializes in developing RNA interference-based therapeutics that target intractable diseases by silencing specific genes. This strategic move aims to attract talent to enhance its research and development efforts in RNAi-based medicines.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) has received Fast Track designation from the FDA for its investigational therapy ARO-APOC3, aimed at reducing triglycerides in patients with familial chylomicronemia syndrome (FCS). This designation facilitates a faster development process and offers benefits such as priority review and accelerated approval. ARO-APOC3 targets apolipoprotein C-III (APOC3) and is currently being assessed in multiple clinical trials, including Phase 3 and Phase 2 studies for FCS and other conditions. There are currently no approved therapies for FCS, underscoring the significance of this treatment.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) is set to participate in the respiratory/infections panel discussion at the Cowen 43rd Annual Health Care Conference on March 8, 2023, at 10:30 a.m. ET, in Boston. The event will be accessible via a webcast on their Investors page. Arrowhead specializes in developing RNA interference therapeutics targeting intractable diseases, utilizing innovative RNA chemistries for gene silencing.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) announced interim results from Part 1 of the AROC3-1001 Phase 1/2 clinical study of ARO-C3, an RNA interference therapeutic aimed at reducing complement component 3 (C3) in patients with various complement-mediated diseases. The interim results showed a mean reduction of 88% in serum C3 and 91% in AH50 at the highest dose. The study indicated safety and tolerability, with no significant adverse events reported. Dosing for Part 2 is expected to start in the first half of 2023. The company aims to address unmet medical needs in conditions like IgA nephropathy and C3 glomerulopathy.
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