Arrowhead Pharmaceuticals

Non-cystic fibrosis bronchiectasis market driven by rising prevalence, early diagnosis, and drug development. Over 15 companies, including Zambon SpA, Insmed, and AstraZeneca, are developing 15+ pipeline therapies. Key drugs in trials include Colistimethate sodium, Benralizumab, and Brensocatib.

Arrowhead Pharmaceuticals launches “We’ll Get There Soon” campaign for Familial Chylomicronemia Syndrome (FCS). Julie Klueckman, an FCS patient, shares her journey and optimism. Arrowhead received FDA Breakthrough Therapy designation for Plozasiran, a treatment for FCS. The company secured $500 million in strategic financing with Sixth Street to support commercial launch in 2025.

US Bancorp DE reduced its Arrowhead Pharmaceuticals holdings by 11.1% in Q3, while other institutions adjusted their stakes. Analysts maintain 'Moderate Buy' consensus with an average target price of $45.33. Arrowhead's stock opened at $21.89 on Friday, with a market cap of $2.72 billion. The company reported a Q2 EPS of ($1.38), missing estimates by ($0.80), and expects current year EPS of -4.31. Arrowhead Pharmaceuticals develops treatments for intractable diseases, with several products in clinical trials.

Analysts from Leerink Partners maintain Buy ratings on Vir Biotechnology (VIR) with a $18.00 target and Sell on Moderna (MRNA) with no target specified. The consensus for VIR is a Moderate Buy with a $32.67 target, while Moderna has a Moderate Buy consensus with a $97.62 target.

RNA interference (RNAi) research, despite initial challenges, has seen advancements with six approved medicines and many in clinical testing. New startups believe RNAi is ready for broader use beyond liver diseases, with innovations in delivery techniques to other tissues. Key players include City Therapeutics, Judo Bio, Switch Therapeutics, and Aro Biotherapeutics, backed by significant funding. The potential of RNAi, first discovered in the 1990s, is now seen as a new frontier in genetic medicine.

Arrowhead Pharmaceuticals launches 'We'll Get There Soon' campaign to raise awareness for Familial Chylomicronemia Syndrome (FCS), a rare disease with high triglyceride levels, aiming to inspire hope and educate the community on managing triglyceride levels below 500 mg/dL to prevent acute pancreatitis.

S.P. reports research grants and consulting fees from various pharmaceutical companies and organizations. M.C. reports consulting fees and institutional research support from multiple entities and serves on the board of Praxis. B.H. and E.G. report no competing interests.

Complement inhibitors market is growing due to the complement system's role in rare and chronic diseases. Over 40 companies are developing 50+ pipeline inhibitors, including Gefurulimab, ANX005, DNTH103, and others. Recent milestones include Q32 Bio presenting ADX-097 data at ASN Kidney Week 2024 and ReAlta Life Sciences dosing the first patient in a Phase II study of RLS-0071.

Alpha-1 antitrypsin deficiency (AATD) affects 1 in 3,500, causing liver/lung damage. Current treatment, augmentation therapy, doesn't reverse damage. Multiple companies develop candidates to prevent/reverse damage, with RNAi, RNA-editing, and base editing therapies showing promise. Vertex's AATD candidates failed, while Arrowhead, Sanofi, Beam Therapeutics, Wave Life Sciences, and Korro Bio advance their treatments. Augmentation therapy may become obsolete within 5-10 years.

Oprah Winfrey reflects on the impact of weight-loss drugs like Wegovy and Zepbound, which dominate the obesity market. The rise of GLP-1 drugs, initially for diabetes, has led to a 'silent revolution' in obesity treatment. Competition is heating up, with 16 new drugs expected by 2031. Small-cap companies like Viking Therapeutics and Terns Pharmaceuticals are emerging as key players, focusing on oral drugs and new therapeutic targets. Investors are advised to consider long-term efficacy and side effect management.