Forum Arrowhead Research geopend

1. de tuinman 28 november 2025 17:07

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   • de tuinman

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   Rare koers bewegingen.
2. Tom3 29 november 2025 13:10

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   Bioboyscout zit aan een waarde van $41 miljard te denken:
   
   drive.google.com/file/d/1WKVXf-pLRSjQ...
   
   Nog even volhouden dus :-)
3. Tom3 29 november 2025 15:20

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   www.novartis.com/ch-de/news/media-rel...
   
   Novartis gaat ook vol in de siRNA's.
4. Tom3 30 november 2025 20:30

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   Eindelijk meer duidelijkheid over ARO-RAGE:
   
   substack.com/home/post/p-179637260
   
   Direct Test of the Central RAGE Hypothesis
   ANALYSIS:
   This is the first human study designed to evaluate whether silencing RAGE can reduce the late asthmatic response—the downstream physiological event most tightly linked to allergen-driven inflammation.
   
   A positive result would provide the first human demonstration of the drug’s mechanistic relevance beyond biomarker reductions.
   
   Focus on Small-Airway Reactance Implies Potential Differentiation
   ANALYSIS:
   Selecting maximum area of reactance as the primary endpoint suggests attention to peripheral airway dysfunction, an area where current asthma biologics often show limited impact.
   
   If upstream RAGE silencing influences small-airway physiology, this could represent a mechanistic niche not addressed by existing therapies.
   
   Narrow, Atopic Population Used for High Signal-to-Noise
   ANALYSIS:
   Restricting enrollment to mild atopic asthma with confirmed allergen sensitivity may reflect a desire to establish a clean mechanistic signal before expanding to mixed or severe asthma populations targeted in the larger Phase 2.
   
   Extended Timeline Indicates Long-Horizon Platform Commitment
   ANALYSIS:
   With primary completion anticipated in late 2027 and full completion in 2028, Arrowhead is implicitly committing to a multi-stage, multi-year program.
   
   The pulmonary RNAi platform—including RAGE—appears to be treated as a long-term strategic asset, consistent with the layering of sequential Phase 2a ? Phase 2 ? Phase 3 development.
   
   What the New Trial Does Not Indicate
   ANALYSIS:
   It does not replace the large Phase 2 study.
   It does not offer real-world asthma control or exacerbation outcomes.
   It does not imply any change in safety outlook.
   It does not support broad efficacy claims.
   This is a mechanistic validation study, not a therapeutic efficacy trial.
   
   Conclusion
   The ARORAGE-2001 allergen-challenge study marks a meaningful evolution in the ARO-RAGE development program.
   It introduces a tightly controlled mechanism-focused step designed to test the drug’s foundational biological premise in humans: that silencing RAGE can modify allergen-induced airway inflammation and small-airway mechanics.
   The evidence indicates continued momentum.
   
   The interpretation suggests a thoughtful tightening of the development pathway—validating the mechanism first, then expanding to broader asthma populations.
5. Tom3 1 december 2025 18:51

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   Koers blijft wonderwel liggen, had een flinke dip verwacht. Voorbode voor de ALK7 resultaten?
6. Tom3 1 december 2025 19:10

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   Dit helpt ook:
   
   scr.zacks.com/news/news-details/2025/...
7. Missolapola 1 december 2025 22:26

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   Arrowhead Pharmaceuticals to Participate in Upcoming December 2025 Conferences
   December 1, 2025
   PASADENA, Calif.--(BUSINESS WIRE)--Dec. 1, 2025-- Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it is scheduled to participate in the following upcoming events:
   
   Piper Sandler 37th Annual Healthcare Conference – December 2-4, 2025
   
   Type: Fireside Chat Presentation
   Date/Time: December 3, 2025, 10:30 a.m. EST
   
   8th Annual Evercore Healthcare Conference – December 2-4, 2025
   
   Type: Fireside Chat Presentation
   Date/Time: December 4, 2025, 10:00 a.m. EST
   
   World Congress of Insulin Resistance, Diabetes and Cardiovascular Disease (WCIRDC) – December 4-6, 2025
   
   Title: Baseline Characteristics and Rationale of the SHASTA-3 and SHASTA-4 Phase 3 Studies of Plozasiran in Patients with Severe Hypertriglyceridemia
   Presenter: Norman Lepore
   
   Title: Recurrent Hospital Admissions in Acute Pancreatitis and Persistent Chylomicronemia
   Presenter: Rohit Loomba
   
   Title: A Monte Carlo Simulation Comparing TG Lowering with Plozasiran versus Olezarsan in Patients with FCS
   Presenter: Nihar Desai
   
   Title: Plozasiran Decreases the Risk of AP and may Improve QOL in FCS
   Encore Presentation
   
   Title: A Randomized Placebo Controlled Phase 3 Study of Plozasiran in Patients with FCS: Palisade 1-Year OLE
   Encore Presentation
   
   7th Annual CNS Delivery Summit – December 9-11, 2025
   
   Title: TRiMTM platform for subcutaneous delivery of siRNA to Central Nervous System
   Date/Time: December 10, 2025, 9:30 am EST
   Presenter: Agnieszka Glebocka
   
   Presentation materials and webcast links, if available/applicable, may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.
8. visje2 2 december 2025 13:47

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   Arrowhead Pharmaceuticals Receives FDA Breakthrough Therapy Designation for Plozasiran in Severe Hypertriglyceridemia
   December 2, 2025
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   PASADENA, Calif.--(BUSINESS WIRE)--Dec. 2, 2025-- Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to investigational plozasiran as an adjunct to diet to reduce triglyceride (TG) levels in adults with severe hypertriglyceridemia (SHTG) (TG levels greater than or equal to 500 mg/dL). There are currently limited and inadequate treatment options for the millions of people globally living with SHTG.
   
   Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies on clinically significant endpoints1.
   
   Arrowhead is on schedule to complete the SHASTA-3, SHASTA-4, and MUIR-3 Phase 3 clinical studies of plozasiran in mid-2026 and intends to submit a supplemental New Drug Application (sNDA) to the FDA by year-end 2026. Arrowhead also plans to seek regulatory approval with additional global regulatory authorities thereafter.
   
   About Severe Hypertriglyceridemia
   
   Severe hypertriglyceridemia (SHTG) is characterized by triglyceride (TG) levels greater than 500 mg/dL, with the most severe form being familial chylomicronemia syndrome (FCS) where TGs typically exceed 880 mg/dL. SHTG significantly increases the risk of acute pancreatitis (AP), which can often include recurrent attacks requiring repeat hospital admissions and worsening outcomes. AP risk is proportional to the number, characteristics, and concentration of triglyceride rich lipoproteins (TRLs), particularly chylomicrons, and increases as TGs rise. Limited treatment options exist to sustainably reduce TGs below guideline directed risk thresholds.
   
   About SHASTA-3 and SHASTA-4 Phase 3 Studies
   
   SHASTA-3 (NCT06347003) and SHASTA-4 (NCT06347016) are double-blind, placebo-controlled, Phase 3 studies to evaluate the efficacy and safety of plozasiran in adults with severe hypertriglyceridemia. Between the two studies, approximately 750 participants were randomized to receive 4 doses (once every 3 months) of 25 mg plozasiran or placebo. The primary endpoint is percent change in fasting serum triglyceride levels from baseline to month 12 compared to placebo. After month 12, eligible participants will be offered an opportunity to continue in an optional open-label extension.
   
   About MUIR-3 Phase 3 Study
   
   MUIR-3 (NCT06347133) is a double-blind, placebo-controlled, Phase 3 study to evaluate the efficacy and safety of plozasiran in adults with hypertriglyceridemia (TGs greater than 150 mg/dL and less than 500 mg/dL). Approximately 1450 participants were randomized to receive 4 doses (once every 3 months) of 25 mg plozasiran or placebo. The primary endpoint is percent change in fasting serum triglyceride levels from baseline to month 12 compared to placebo.
   
   About Plozasiran
   
   Plozasiran is a first-in-class investigational RNA interference (RNAi) therapeutic designed to reduce production of apolipoprotein C-III (apoC-III) which is a component of triglyceride rich lipoproteins (TRLs) and a key regulator of triglyceride metabolism. ApoC-III increases triglyceride levels in the blood by inhibiting breakdown of TRLs by lipoprotein lipase and uptake of TRL remnants by hepatic receptors in the liver. The goal of treatment with plozasiran is to reduce the level of apoC-III, thereby reducing triglycerides and restoring lipids to more normal levels.
   
   Plozasiran is being investigated in the SHASTA-3, SHASTA-4, and SHASTA-5 Phase 3 studies in patients with severe hypertriglyceridemia and the MUIR Phase 3 study in patients with mixed hyperlipidemia. Plozasiran is approved in the U.S. as REDEMPLO as an adjunct to diet to reduce triglycerides in adults with Familial Chylomicronemia Syndrome, with additional global regulatory submissions and reviews in process.
   
   About REDEMPLO® (plozasiran)
   
   REDEMPLO (plozasiran) is approved by the U.S. Food and Drug Administration as an adjunct to diet to reduce triglycerides in adults with Familial Chylomicronemia Syndrome (FCS). REDEMPLO is an siRNA therapeutic designed to suppress the production of apoC-III, a protein produced in the liver that raises triglyceride levels by slowing their breakdown and clearance. By targeting apoC-III with sustained silencing, REDEMPLO delivers significant reductions in triglyceride levels. REDEMPLO is the first and only FDA-approved treatment studied in both genetically confirmed and clinically diagnosed patients living with FCS.
   
   For more information about REDEMPLO, visit Our Medicines.
   
   IMPORTANT SAFETY INFORMATION
   
   CONTRAINDICATIONS
   
   None.
   
   ADVERSE REACTIONS
   
   Most common adverse reactions in REDEMPLO treated patients (incidence =10% of patients treated with REDEMPLO and >5% more frequently than with placebo) are hyperglycemia, headache, nausea, and injection site reaction.
9. Missolapola 2 december 2025 14:21

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   Top!
10. Hulskof 2 december 2025 18:10

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    quote:

    Missolapola schreef op 2 december 2025 14:21:

    Top!
    

    Voor wat het waard is. Arwr had het ook voor FCS maar kreeg gewoonde 10 maanden review period. Waarschijnlijk omdat er door de goedkeuring van Tryngolza (Ionis) geen unmeed need meer bestond. Datzelfde kan nu weer gebeuren, aangezien Ionis nog altijd een jaar op Arwr voorloopt.
12. Tom3 3 december 2025 21:29

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    @Hulskof, snap jij iets van de huidige koersexplosie?
13. Hulskof 4 december 2025 10:26

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    quote:

    Tom3 schreef op 3 december 2025 21:29:

    @Hulskof, snap jij iets van de huidige koersexplosie?
    

    Niet echt. Ik had na de goedkeuring een fikse terugval van de koers verwacht, had zelfs 15% van mijn positie verkocht. Maar ik zat er weer eens helemaal naast. ;-)
    
    Dirk suggereert een mogelijke deal mbt obesitas (ALK7 en INHBE).
    Op Discord lees ik soortgelijke verhalen.
    Holden lijkt dan weer zeker van een pulmonary deal.
    
    Ik weet het niet. Zelf vind ik dat de koers intussen aardig voor zichzelf is uitgelopen, maar ik verkoop voorlopig niets meer. Ik zal eerder bijkopen op dips.
14. Tom3 4 december 2025 12:18

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    Tja, hier de visie van Dirk op X:
    
    Dirk Haussecker
    @RNAiAnalyst
    $arwr does not call out their 'weight loss' drugs against inhibinE/Alk7 as critical to their future.
    
    CVD/sHTG and CNS candidates are prioritized also as it comes to capital allocation.
    
    Makes me a bit cautious about upcoming $wve readout.
    
    On the other hand, this may all be explained by $arwr partnering them. Delaying weight loss data into '1st week of January' due to claimed difficulties of coordinating travel plans (in the age of Zoom, come on!), may just be a cop out for announcing a blockbuster weight loss deal the week before JPM.
    
    Stock price also suggests possibility of deal near-term.
15. nb 4 december 2025 14:55

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    Is de koersontwikkeling ook niet raar, gezien de insider verkopen? Al zijn dat ook weer niet wereldschokkende bedragen.
16. Hulskof 4 december 2025 15:21

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    ir.arrowheadpharma.com/static-files/a...
    
    Vergelijking Olezarsen met Plozasiran in FCS. Dit zou ook wel eens een reden kunnen zijn, zeker met het oog op sHTG.
17. Hulskof 4 december 2025 15:22

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    quote:

    nb schreef op 4 december 2025 14:55:

    Is de koersontwikkeling ook niet raar, gezien de insider verkopen? Al zijn dat ook weer niet wereldschokkende bedragen.
    

    Lijkt me niet. Die zijn er elk jaar in december.
18. Hulskof 8 december 2025 15:03

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    Wave Life Sciences Announces Positive Interim Data from Phase 1 INLIGHT Trial of WVE-007 (INHBE) for Obesity; Single Dose Resulted in Improvement in Body Composition With Fat Loss Similar to GLP-1 at Three Months Without Muscle Loss
    
    ir.wavelifesciences.com/news-releases...
    
    Wave +80% voorbeurs, ARWR +10%
    
    Arrowhead loopt ongeveer 10 weken voor met INHBE (en ALK7), maar komt pas in januari met data.
19. de tuinman 8 december 2025 15:42

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    Arrowhead Pharmaceuticals Initiates Phase 1/2a Study of ARO-MAPT for the Treatment of Alzheimer’s Disease and Other Tauopathies
20. de tuinman 8 december 2025 15:43

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    quote:

    Hulskof schreef op 8 december 2025 15:03:

    Wave Life Sciences Announces Positive Interim Data from Phase 1 INLIGHT Trial of WVE-007 (INHBE) for Obesity; Single Dose Resulted in Improvement in Body Composition With Fat Loss Similar to GLP-1 at Three Months Without Muscle Loss
    
    ir.wavelifesciences.com/news-releases...
    
    Wave +80% voorbeurs, ARWR +10%
    
    Arrowhead loopt ongeveer 10 weken voor met INHBE (en ALK7), maar komt pas in januari met data.
    

    Wel mooi nieuws.