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GoedeDag
0
quote:

Tom3 schreef op 15 mei 2024 10:20:

[...]

Slimme move: de helft van de bezittingen bestaat uit een retailer CAVA. Die positie wordt met meer dan 50% winst langzaam ingeruild tegen voornamelijk biotechs. Die zijn de laatste jaren sterk achter gebleven bij de rest.
True maar het is wel speculatief biotechs. Wel gemerkt dat voor arrowhead gedacht dat de rente naar beneden gaat de koers toch wel flink hoger heeft gezet (om dan weer in de afgrond te lopen omdat inflatie toch langer duurt). Echte verlaging van de rente die eens gebeurd zal misschien de koers wat permanent hoger zetten (zonder extra nieuws van al de conferences of onderzoek).

Als ik zoveel kapitaal als een holding had zou ik ook wel kleine verhoging durven wagen in arrow. If only :)
de tuinman
0
Arrowhead Pharmaceuticals Presents New Clinical Data Showing ARO-RAGE Achieves High Level of Gene Knockdown in Patients with Asthma
Missolapola
1
Arrowhead Pharmaceuticals Presents New Phase 2 Data of Plozasiran in Patients with Mixed Hyperlipidemia
May 28, 2024
PDF Version
- Plozasiran significantly lowered triglyceride levels with commensurate reductions in APOC3, non-HDL-C, and remnant cholesterol

- Data presented at European Atherosclerosis Society 92nd Congress and simultaneously published in the New England Journal of Medicine

PASADENA, Calif.--(BUSINESS WIRE)--May 28, 2024-- Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced results from the Phase 2b double blind, randomized MUIR study of investigational plozasiran (formerly ARO-APOC3) in patients with mixed hyperlipidemia. Treatment with plozasiran in the MUIR study achieved reductions in triglyceride rich lipoproteins, a genetically validated target associated with increased risk of atherosclerotic cardiovascular disease (ASCVD)1,2. These data were presented in an oral presentation today at the European Atherosclerosis Society (EAS) 92nd Congress and simultaneously published in the New England Journal of Medicine.

“Plozasiran demonstrated potent and durable reductions of atherogenic lipoproteins, such as non-HDL-C, ApoB, and remnant cholesterol in the Phase 2 MUIR study that supports its further development in Phase 3 studies for patients with increased risk for ASCVD,” said Christie M. Ballantyne, M.D., Baylor College of Medicine, and Principal Investigator for the MUIR study. “Despite advances in treatment, patients with mixed hyperlipidemia have elevated and persistent ASCVD risk due in part to high levels of atherogenic triglyceride rich lipoproteins. The promising results from treatment with plozasiran in the MUIR study help to lay the groundwork for a more extensive study to potentially test whether plozasiran reduces ASCVD risk.”

Bruce Given, M.D., interim chief medical scientist at Arrowhead added, “Results from the MUIR study of plozasiran in patients with mixed hyperlipidemia are encouraging and we are excited to present data at EAS and publish the results today in the New England Journal of Medicine. We believe plozasiran shows promise in multiple diseases with substantial unmet need, including familial chylomicronemia syndrome, severe hypertriglyceridemia, and mixed hyperlipidemia, and we are eager to further investigate plozasiran in additional clinical studies.”

Select MUIR Results

By silencing Apolipoprotein C-III (APOC3), plozasiran significantly reduced triglycerides and atherogenic triglyceride rich lipoproteins and increased HDL, across all dose levels at Week 24 in patients with mixed dyslipidemia.

At week 24, representing trough effect after 2 quarterly doses, plozasiran treatment was associated with placebo adjusted reductions in triglycerides of -50%, -56%, and -62% (all p<0.001) at the 10, 25, and 50 mg doses, respectively. Fasting triglyceride levels were normalized (achieved levels below 150 mg/dL) in most patients (79-92%) randomized to a treatment arm. Commensurate reductions in APOC3 of -57%, -73%, and -79%, with strong positive correlations with changes in triglyceride levels were observed.

Changes in other atherogenic lipoprotein parameters were also observed. At week 24, for the quarterly doses of 10, 25, and 50 mg, the following placebo adjusted changes were observed: non-HDL-C levels -17%, -18%, and -24%; apoB levels -10%, -13%, and -19%; and remnant cholesterol levels -43%, -49%, and -48% with strong correlations with changes in triglyceride levels.

Safety and Tolerability

Plozasiran demonstrated a favorable safety profile in the MUIR study. The overall rates of occurrence of treatment-emergent adverse events (TEAEs) and discontinuations were similar for plozasiran and placebo throughout the 48 weeks of observation. Observed adverse events generally reflected the comorbidities and underlying conditions of the study population. TEAEs occurring in 5 or more patients were COVID-19, worsening glycemic control, upper respiratory tract infection, urinary tract infection, headache, and bronchitis.

Arrowhead is also presenting data from the SHASTA-2 study of plozasiran and the ARCHES-2 study of zodasiran (formerly ARO-ANG3) at EAS. Details about the EAS presentations are listed below.

European Atherosclerosis Society (EAS) 92nd Congress – May 26-29, 2024

Title: PLOZASIRAN (ARO-APOC3), DECREASES APOC3 AND TRIGLYCERIDES (TG) IN PATIENTS WITH MIXED DYSLIPIDEMIA: MUIR FINAL RESULTS
Date/Time: May 28, 2024, 12:04 p.m. CEST
Presenter: Christie M Ballantyne, M.D.
Session: New Therapeutics

Title: PLOZASIRAN (ARO-APOC3), AN INVESTIGATIONAL RNAI THERAPEUTIC, DEMONSTRATES PROFOUND AND DURABLE REDUCTIONS IN APOC-3 AND TRIGLYCERIDES (TG) IN PATIENTS WITH SEVERE HYPERTRIGLYCERIDEMIA (SHTG), SHASTA-2 FINAL RESULTS
Date/Time: May 28, 2024, 2:49 p.m. CEST
Presenter: Daniel Gaudet, M.D., Ph.D.
Session: SaaG Session: The Enigmas of TG-rich Lipoproteins

Title: ZODASIRAN SILENCES HEPATIC ANGPTL3 LEADING TO DEEP AND DURABLE REDUCTIONS IN ATHEROGENIC LIPIDS AND LIPOPROTEINS IN MIXED DYSLIPIDEMIA PATIENTS: FINAL RESULTS FROM ARCHES-2, DOUBLE-BLIND PERIOD
Date/Time: May 29, 2024, 11:30 a.m. CEST
Presenter: Robert Rosenson, M.D.
Session: Late Breaker Session 2: New Therapeutic Agents
Missolapola
1
Arrowhead Pharmaceuticals Presents New Phase 2 Data of Zodasiran in Patients with Mixed Hyperlipidemia
May 29, 2024
PDF Version
- Zodasiran significantly reduced triglycerides and atherogenic triglyceride rich lipoproteins across all dose levels at Week 24

- Data presented at European Atherosclerosis Society 92nd Congress and simultaneously published in the New England Journal of Medicine

PASADENA, Calif.--(BUSINESS WIRE)--May 29, 2024-- Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced results from the Phase 2b double blind, randomized ARCHES-2 study of investigational zodasiran (formerly ARO-ANG3) in patients with mixed hyperlipidemia. Zodasiran was associated with robust and durable reductions in triglycerides, triglyceride rich lipoprotein remnants, and total atherogenic lipoproteins, including LDL-C. These data were presented in a late-breaking oral presentation today at the European Atherosclerosis Society (EAS) 92nd Congress and simultaneously published in the New England Journal of Medicine.

“Results from clinical studies of zodasiran, including those for the ARCHES-2 study in patients with mixed hyperlipidemia presented today at EAS and published in the New England Journal of Medicine, continue to support ANGPTL3 as an exciting target for an RNAi-based gene silencing strategy,” said Bruce Given, M.D., interim chief medical scientist at Arrowhead. “Genetic studies show that ANGPTL3 loss-of-function variants lead to enhanced lipoprotein lipase and endothelial lipase activity, resulting in lower concentrations of most plasma lipoproteins, including triglyceride rich lipoproteins, LDL-C, VLDL/remnant cholesterol, and HDL-C. Individuals with these variants also have demonstrated a markedly reduced risk of ASCVD and have no known adverse clinical phenotypes1-3.”

Robert Rosenson, M.D., Icahn School of Medicine at Mount Sinai, and Principal Investigator for the ARCHES-2 study added, “The potent reductions in serum lipids and lipoproteins and favorable safety profile seen in the ARCHES-2 clinical study of zodasiran suggest its potential to treat residual ASCVD risk in patients with elevated triglyceride rich lipoproteins. The genetic data around ANGPTL3 as a target are very compelling and support further Phase 3 studies to determine whether the large reductions in triglyceride rich lipoproteins observed after zodasiran treatment can replicate the genetic data and reduce ASCVD risk.”

Select ARCHES-2 Results

Zodasiran treatment was associated with dose-dependent placebo adjusted reductions in triglycerides, remnant cholesterol, LDL-C, ApoB, and Non-HDL-C across all dose levels in patients with mixed hyperlipidemia.

At week 24, representing trough effect, zodasiran treatment at 50, 100, and 200 mg on day 1 and week 12 was associated with placebo adjusted reductions in triglycerides of -51%, -57%, and -63% (all p<0.001) respectively. ANPTL3, the genetic target of zodasiran, was reduced compared with placebo by -54%, -70%, and -74%, and remnant cholesterol levels were reduced by -73%, -76%, and -82%, which strongly correlated with changes in triglyceride levels.

Changes in other atherogenic lipoprotein parameters were also observed across all three dose levels. At week 24, the following placebo adjusted changes were observed for the 200 mg dose: LDL-C -20%, ApoB -22%; Non-HDL-C -36%.

In a subset of patients with baseline liver fat fraction greater than 8%, dose-dependent liver fat reductions, measured by MRI-PDFF, were observed reaching -28% with the 200 mg dose compared with -2% with placebo.

Safety and Tolerability

Zodasiran demonstrated a favorable safety profile in patients with mixed hyperlipidemia in the ARCHES-2 study. Treatment-emergent adverse events (TEAEs) were generally balanced between treatment and placebo groups and generally reflected the comorbidities and underlying conditions of the study population. There were no clinically meaningful changes in laboratory safety evaluations, no changes in mean platelet counts, and modest changes in HbA1c.

Details about the EAS presentation is listed below.

European Atherosclerosis Society (EAS) 92nd Congress – May 26-29, 2024

Title: ZODASIRAN SILENCES HEPATIC ANGPTL3 LEADING TO DEEP AND DURABLE REDUCTIONS IN ATHEROGENIC LIPIDS AND LIPOPROTEINS IN MIXED DYSLIPIDEMIA PATIENTS: FINAL RESULTS FROM ARCHES-2, DOUBLE-BLIND PERIOD
Date/Time: May 29, 2024, 11:30 a.m. CEST
Presenter: Robert Rosenson, M.D.
Session: Late Breaker Session 2: New Therapeutic Agents
GoedeDag
0
Lijkt me goed nieuws, concurrent minder zou toch positief moeten zijn.

Ben wel niet technisch genoeg om te weten of arrowhead zijn ARO-ANG3 kans heeft om tegen dezelfde issues op te lopen als het gecancelde vupanorsen.
de tuinman
0
quote:

GoedeDag schreef op 31 mei 2024 22:42:

Lijkt me goed nieuws, concurrent minder zou toch positief moeten zijn.

Ben wel niet technisch genoeg om te weten of arrowhead zijn ARO-ANG3 kans heeft om tegen dezelfde issues op te lopen als het gecancelde vupanorsen.
Voor mij ook een vraagteken.

Het is erg stil bij de kenners. Toch zoekt Arrowhead de media.
Van X krijg ik een vaag vermoeden dat beleggers bezorgd zijn om de kas positie van Arrowhead.
Hulskof
6
@Tuinman en GoedeDag: Pfizer trok haar handen af van Vupanorsen in 2022. Het werd op X aangehaald om weer te geven hoe groot de ANGPTL3- markt is en hoe laag Arrowhead momenteel dus gewaardeerd wordt, aangezien Zodasiran (ARO-ANG3) veelbelovend is in die markt en geen last van de issues van Vupanorsen lijkt te hebben. Ik zeg bewust 'lijkt', want geen enkele outsider kan hier naar mijn idee een gedegen oordeel over vellen. Arrowhead lijkt in elk geval zeker van haar zaak, maar was dat voorheen met ARO-ENAC ook en toen kwamen daar ineens die ratten met hun overgevoelige longen. ;-) Ik bedoel maar, je weet nooit hoe eea loopt totdat het zover is.

Maar dat Arrowhead nu kiest voor één combinatie CVOT van APOC3 en ANG3 lijkt me alleszins erg positief.
Dat MS en GS hun koersdoelen verlagen maar ondertussen aandelen Arrowhead blijven bijkopen is nog positiever.
Het enige dat mij steekt (en de markt kennelijk ook) is het uitblijven van een nieuwe deal die flink wat geld op de plank brengt, want die gedane emissie is misschien leuk voor de korte termijn, maar lost de grote geldbehoefte voor genoemde CVOT niet op.
Zodra die horde genomen is, schiet de koers omhoog (is mijn voorspelling). Tot die tijd blijven we denk ik tussen de 20 en 25 hangen.
wijzerplaat
2
Arrowhead Pharmaceuticals Reports Successful Topline Results for Plozasiran from the
Pivotal Phase 3 PALISADE Study in Patients with Familial Chylomicronemia Syndrome
June 3, 2024
- Plozasiran achieved statistically significant median reductions in triglycerides up to 80% and mean reductions in APOC3 up to 94% at month 10
- Plozasiran achieved a statistically significant reduction in incidence of acute pancreatitis versus placebo
- Plozasiran is the company’s first investigational RNAi-based therapy to show clinical efficacy in a Phase 3 study
- Arrowhead plans to highlight recent data for its cardiometabolic pipeline at its June 25, 2024, Cardiometabolic event as part of the 2024 Summer
Series of R&D Webinars

ir.arrowheadpharma.com/node/19141/pdf
de tuinman
0
De eerste fase 3 uitslag. Eindelijk kan Arrowhead de markt op. Ik heb bergrepen dat dit nog een kleine markt is, klopt dat? En dat er meer fase 3 uitslagen nodig zijn om een serieuze omzet te halen. (Blockbuster?)
Hulskof
1
Driven by an older, more diverse population, along with a significant increase in risk factors including high blood pressure and obesity, total costs related to cardiovascular disease (CVD) conditions are likely to triple by 2050, according to projections from the American Heart Association. At least 6 in 10 U.S. adults (61%), more than 184 million people, are expected to have some type of CVD within the next 30 years, reflecting a disease prevalence that will have a $1.8 trillion price tag in direct and indirect costs. The new data comes from two new presidential advisories published today in the journal Circulation—Forecasting the Burden of Cardiovascular Disease and Stroke in the United States Through 2050: Prevalence of Risk Factors and Disease and Forecasting the Economic Burden of Cardiovascular Disease and Stroke in the United States through 2050. The companion papers build upon prior work by the Association to assess projections of the future cardiovascular disease prevalence and subsequent economic burden based on the current landscape.
(Looks like a tremendous opportunity for new technologies like RNAi to make an impact….)

Hou de komende jaren maar een mooi aantal stukjes Arrowhead aan...
Hulskof
0
quote:

de tuinman schreef op 3 juni 2024 17:05:

De eerste fase 3 uitslag. Eindelijk kan Arrowhead de markt op. Ik heb bergrepen dat dit nog een kleine markt is, klopt dat? En dat er meer fase 3 uitslagen nodig zijn om een serieuze omzet te halen. (Blockbuster?)
Hoe het exact zit is onduidelijk. Zal vd FDA afhangen.
Hulskof
1
Holden (de schreeuwlelijk op Discord die evenwel zinnige dingen zegt) schaalt het longprogramma vooralsnog laag in, net als de markt trouwens. Een hoop gedoe rond Feno in Rage...

Some noise exists around the company's decision to move forward with a RAGE p2 w/o complete p1 feno cohort data. BG explained it as feno is ireelevant as an endpont relative to what ARWR is therapeutically investigating. I call bullshit to that for three reasons. 1) Vince tepidly suggested almost as a afterthought that feno patients were hard to enroll bc the ARWR trial was competing for patients. Prospective patients have options an I suspect there are trials that are further along that have more established safety profiles AND some indication of efficacy. It's generally not the patient that comes up with the trial selection but the doc. So this has me concerned that a better more advanced competing product exists that might be better suited for the feno patient population. 2) Bruce suggested that feno may (IMO or may not) have anything to do with what they are trying to investigate/study for possible treatment. 3) This is a study design screwup. Why investigate something that is quite possibly not a primary outcome and maybe not even a secondary outcome? This designed was absolutely conceived of by scientists under JSM and the study was approved by JSM and unfortunately likely JH. I strongly suspect that BG is now treating this as a cut to the chase asthma trial and NOT a CF trial or something (feno) that would have been investigated by big pharma for no reason other than to fill out the scientific portfolio with minimal, if any, commercial value. I HOPE that feno has no immediately relevant commericial value and that this foolish pursuit can be hung on JSM. It is, however, another dipshit move either directed by or allowed by CA that bloated costs and slowed progress. All, of course, IMO

The Street is giving D-I-K to ARWR's lung program as it should bc, thus far, the lung program has been some place between a failure or a science project that has produced a modest amount of data that are inconclusive AND a trial that has only served to confuse the investment community rather than bolster confidence. And they have done a preposterously shitty job discussing that screwup, effectively trying to sweep it under the rug. Normally I (and unfortunately the Street) would have given them a pass on this but they are already deep in the hole in the lung program credibility trough. As far as I am concerned, wrt the lung franchise, they are sucking wind. (pun intended).
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