Forum Arrowhead Research geopend

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Tom3
1
Gevonden op bij ARWR -Stocktwits:

drive.google.com/file/d/1NuY48GcVWbAe...

De auteur lis helaas onbekend, zou van een shorter van CRISPR etc kunnen zijn. Het komt wel overeen met mijn visie op Crispr-Cas aandelen.
Tom3
1
Alnylam lijkt zich niet te concentreren op spierweefsel en kanker targets. Wel staan NASH, CNS (inclusief Alzheimer en Katwijkse ziekte), longen (maar geen CF) en ogen op het programma. Het kan nooit kwaad te weten wat de buren aan het doen zijn:

www.alnylam.com/wp-content/uploads/20...
Hulskof
0
quote:

Tom3 schreef op 25 april 2021 16:08:


Gevonden op bij ARWR -Stocktwits:

drive.google.com/file/d/1NuY48GcVWbAe...

De auteur lis helaas onbekend, zou van een shorter van CRISPR etc kunnen zijn. Het komt wel overeen met mijn visie op Crispr-Cas aandelen.


Goed stuk. Wist niet dat zoveel mensen niet in aanmerking komen voor Crispr-Cas. Dat lijkt de startbaan nog een heel stukje te verlengen...
Tom3
0
quote:

Hulskof schreef op 26 april 2021 10:56:


[...]

Goed stuk. Wist niet dat zoveel mensen niet in aanmerking komen voor Crispr-Cas. Dat lijkt de startbaan nog een heel stukje te verlengen...


Voor ex vivo toepassingen (bijvoorbeeld Fate 's modificaties van NK cellen en sikkelcelziekte ) valt het kennelijk mee maar voor in vivo toepassingen is er nog geen zicht op succes. Beam Therapeutics zal ook tegen dat probleem aanlopen, getuige haar laatste aankoop:

medcitynews.com/2021/02/beam-makes-12...

Beam werkt nog met LNP. Dat is door de Rnai bedrijven al lang verlaten.
Tom3
0
De reden van het verdwijnen van LNP's bij rnai wordt treffend beschreven in mijn lievelingspaper terzake:

www.ncbi.nlm.nih.gov/pmc/articles/PMC...

Ik zou wel eens willen weten waarom Beam nog steeds de LNP methode gebruikt?
Hulskof
0
quote:

de tuinman schreef op 26 april 2021 16:43:


twitter.com/ArrowheadPharma/status/13...


Ja, ze blijven maar plagen met die bewustwordingstweets. Tijd voor POC-data. :-)
mvdln
0
We zijn intussen eind april en Q2/2021 heeft waarschijnlijk nog wel een aantal PR's in het vooruitschiet. Iedere dag dat er geen komt wordt de spanning opgebouwd. De koers neemt alvast een voorschot op het verloop?

Over 1 week conference call. Zal er voor die tijd nog een PR uitgestuurd worden? Hebben ze toch iets om over te praten. Het zou zomaar kunnen.

Ik koop tussendoor wat call opties met een korte looptijd en met een hoge(re) uitoefenprijs dan de huidige koers. Op die manier profiteer ik mee moest 1 van de komende nieuwtjes de koers naar de stratosfeer sturen.
mvdln
1
AMGen Q1 2021 Earnings Call Transcript Q&A over AMG890 ( Olparisan):

Jay Olson -- Oppenheimer -- Analyst

Oh, hey, thanks for taking the question. Curious about the Phase 2 data for olpasiran that you expect in the first half of next year. Can you just talk about what signals you'll be looking for in that data in terms of your plans to design a Phase 3 study, and any potential points of differentiation from pelacarsen? Thank you.

David M. Reese -- Executive Vice President, Research and Development

Yeah. Thanks, Jay. And for those who may not know off the top of their heads what olpasiran is, this was formerly AMG 890. It's a small interfering RNA designed to lower lipoprotein A levels in patients with atherosclerotic cardiovascular disease where elevated Lp(a) may be a driver. As we noted, we completed enrollment in what's a robust Phase 2 trial that actually completed enrollment ahead of schedule. And what we'll be looking for as we unveil those data, Jay, are, first of all, longer term follow-up, meaning sufficient long-term suppression of Lp(a) levels. And our targets would be in the range of the Phase 1 data that we presented last November at the American Heart Association meeting and then, of course, additional safety data.

And of course, we are exploring different dose levels as is pretty much standard in a program like this. And so this would be in part for dose selection for Phase 3 going forward. We are very actively engaged already in Phase 3 planning and what the design of that trial may look like. Based on the data that we've seen to date, one of our goals may be relatively infrequent dosing given the duration of effect that we observed in the Phase I trial, and that's one thing that we'll be taking a close look at as well in Phase II.
holenbeer
2
Post van BioBoyScout op Yahoo:

Valuation dilemma. I believe that Arrowhead is coming to a cross-roads where valuing the company "fairly" will become very difficult. If Arrowhead can show that it can successfully target diseases extrahepatically in multiple tissues, the world is truly their oyster - more so than even I can fully imagine, or even begin to understand what the "fair" method of valuation really is.

While biotechs generally get valued by the drugs that are in their pipeline based on the diseases they're able to treat, and what kind of sales they can generate from that particular patient population. Those sales are then risk-adjusted and then also further adjusted to determine their present value. This is a fairly simple, straight-forward process to come up with a fair valuation. However, the tables are turned on this process when you have a tweakable technology that has literally of hundreds, if not thousands, of applications in areas that no other drugs can come close to in effectiveness, particularly when you can go outside of the liver to other tissues. An RNAi knockdown chart is just not in the same ballpark as a knockdown chart using other modalities. This is clearly a game changer, and as I said earlier, the world becomes your oyster.

So how do you value such a biotech? Do you just continue to value the drug pipeline as you normally do? That's one approach, however, everyone now knows that there are PLENTY of new targets coming, and it's just a matter of time. Why? because the drug development process for an RNAi drug is a fairly straight-forward process, and that process is the TRiM platform - just insert a genetically validated target, run your bioinformatics to determine your targets, test your targets, apply your appropriate targeting ligand, and poof, you have a super effective drug that will just blow away any other modalities out there.

So what does this mean? It means that even though you may not have a pipeline of 100 RNAi drugs today, you have the very solid potential of developing a pipeline in the near future that has a very high chance of being able to produce, and I hate to say it, well over a trillion dollars in sales. That is exactly why, if extrahepatic truly works, Arrowhead scientists should be multi-millionaires.

So that begs the question, HOW DO YOU VALUE SUCH A COMPANY that has a flexible platform to hit all sorts of other targets? It HAS to have a valuation beyond the existing portfolio, as you know that it's the goose that's laying golden eggs. It's easy to throw any number out there for many reasons, i.e. $1B, $5B, $20B, $30B, or even $50B or $100B, as you know that this platform will end up delivering and provide a fantastic ROI. The drug candidates are there, the need for more effective drugs in other tissues is there, and the money to pay for these drugs is also there.

So that's the valuation dilemma. How do you fairly value the TRiM platform once extrahepatic proves out? I have plenty of ideas, and it will be interesting to see how analysts move forward. I do think it would help if the company laid out a 15-30 year plan of what is truly possible, as that would help investors understand the amount of potential future revenue that's at hand. That would also help value TRiM. Chris wasn't kidding when he was using the word franchise to describe all the various different tissues and targets they can go after. I think he knows he's sitting on a gold mine, and he just needs some time to see it through.
Hulskof
0
Nu nog proof of concept buiten de lever om bovenstaand verhaal te bevestigen. De klok tikt...
wijzerplaat
0
Ik haal dit ook graag nog eens aan:

twitter.com/BikeRieder/status/1386905...

31 Post's on drug candidates
->HIF2=4
->ENaC=9
->AAT=6
->APOC3 (FCCS)=6
->APOC3 (HTG)=4
->ANG3=1
->HSD non -need for combo with partner
4 other post's
Comparison DRNA
64 Post's
->40 other
->24 (22 on 1 hyperoxa. cand.)
Do we have something good for patients?


Alsook nog eens zijn poster:
pbs.twimg.com/media/EpRrlNRW8AErEx_?f...


P.iddybull
1
Arrowhead Announces Improvement in Fibrosis after ARO-AAT Treatment in Patients with Alpha-1 Liver Disease
Apr 28, 2021 at 7:30 AM EDT
PDF Version
PASADENA, Calif.--(BUSINESS WIRE)--Apr. 28, 2021-- Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced positive interim 48-week liver biopsy results from the AROAAT2002 study, an open-label Phase 2 clinical study of ARO-AAT, the company’s second generation investigational RNA interference (RNAi) therapeutic being co-developed with Takeda Pharmaceutical Company Limited ("Takeda”) as a treatment for the rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD). The results demonstrate that ARO-AAT treatment led to a consistent and substantial reduction in intra-hepatic mutant AAT protein (Z-AAT), both Z-AAT monomer and Z-AAT polymer; a consistent decrease in histological globule burden; improvements in fibrosis; and, improvements in other relevant biomarkers of liver health. Arrowhead intends to present additional interim AROAAT2002 data at an upcoming medical congress, pending abstract acceptance.

After 48 weeks of treatment with investigational ARO-AAT in cohort 2 (n=5) of the AROAAT2002 study, the following results were observed:

Four of the five patients achieved a 1 or greater stage improvement in Metavir fibrosis stage, with no worsening of fibrosis in the fifth patient
All five patients demonstrated reductions in histological globule assessment scores
Total intra-hepatic Z-AAT decreased by 77-97%
After only 24 weeks of treatment with investigational ARO-AAT in cohort 1 (n=4) of the AROAAT2002 study, the following results were observed:

Two of the four patients achieved a 1 or greater stage improvement in Metavir fibrosis stage, with no worsening of fibrosis in the other two patients
The two patients who improved fibrosis stages during treatment were both deemed cirrhotic (F4) when the study began
All four patients demonstrated reductions in histological globule assessment scores
As previously reported, total intra-hepatic Z-AAT decreased by 72-95%
Javier San Martin, M.D., chief medical officer at Arrowhead, said: “The results from treatment with investigational ARO-AAT in the Phase 2 AROAAT2002 open-label study continue to impress us. We believe the pharmacodynamic effect in alpha-1 patients is clear and consistent. The results from 24 and 48 weeks of treatment also indicate that when production of the Z-AAT protein is inhibited, the liver has the ability to clear the accumulated mutant protein and begin the fibrosis regression process earlier and more efficiently than we anticipated, even in patients with severe liver disease. We look forward to sharing more details about these exciting results at an upcoming medical congress. We also intend to use these and other results to inform further interactions with regulatory authorities to pursue opportunities for potential accelerated approval pathways, if appropriate. Our collaboration with Takeda on the ARO-AAT program has been highly productive, and we continue to see them as the ideal partner as the program advances towards patients in need of new therapies for alpha-1 liver disease.”

In October 2020, Arrowhead and Takeda announced a collaboration and licensing agreement to develop ARO-AAT. Under the terms of the agreement, Arrowhead and Takeda will co-develop ARO-AAT which, if approved, will be co-commercialized in the United States under a 50/50 profit-sharing structure. Outside the U.S., Takeda will lead the global commercialization strategy and receive an exclusive license to commercialize ARO-AAT with Arrowhead eligible to receive tiered royalties of 20-25% on net sales. Arrowhead received an upfront payment of $300 million and is eligible to receive potential development, regulatory and commercial milestones of up to $740 million.

AROAAT2002 (NCT03946449) is a pilot open-label, multi-dose, Phase 2 study to assess the response to ARO-AAT in 16 patients with AATD associated liver disease and baseline liver fibrosis. All eligible participants receive a pre-dose biopsy and an end of study biopsy. Treated participants will also be offered the opportunity to continue treatment in an open-label extension (OLE). Including the OLE, interim assessments will be made after 6 months, 12 months, 18 months, and 24 months of treatment with ARO-AAT.
Hulskof
1
Ik denk dat de waardering vanuit de markt voor AAT pas komt als Vertex aankondigt gefaald te hebben. Nog even geduld...
P.iddybull
1
Deze PB is inderdaad een opwarmertje voor wat nog volgen gaat.
NBI op -1,2 % momenteel,speelt ook wat mee.
Tom3
0
quote:

holenbeer schreef op 28 april 2021 08:30:


Post van BioBoyScout op Yahoo:

Valuation dilemma. I believe that Arrowhead is coming to a cross-roads where valuing the company "fairly" will become very difficult. If Arrowhead can show that it can successfully target diseases extrahepatically in multiple tissues, the world is truly their oyster - more so than even I can fully imagine, or even begin to understand what the "fair" method of valuation really is.

While biotechs generally get valued by the drugs that are in their pipeline based on the diseases they're able to treat, and what kind of sales they can generate from that particular patient population. Those sales are then risk-adjusted and then also further adjusted to determine their present value. This is a fairly simple, straight-forward process to come up with a fair valuation. However, the tables are turned on this process when you have a tweakable technology that has literally of hundreds, if not thousands, of applications in areas that no other drugs can come close to in effectiveness, particularly when you can go outside of the liver to other tissues. An RNAi knockdown chart is just not in the same ballpark as a knockdown chart using other modalities. This is clearly a game changer, and as I said earlier, the world becomes your oyster.

So how do you value such a biotech? Do you just continue to value the drug pipeline as you normally do? That's one approach, however, everyone now knows that there are PLENTY of new targets coming, and it's just a matter of time. Why? because the drug development process for an RNAi drug is a fairly straight-forward process, and that process is the TRiM platform - just insert a genetically validated target, run your bioinformatics to determine your targets, test your targets, apply your appropriate targeting ligand, and poof, you have a super effective drug that will just blow away any other modalities out there.

So what does this mean? It means that even though you may not have a pipeline of 100 RNAi drugs today, you have the very solid potential of developing a pipeline in the near future that has a very high chance of being able to produce, and I hate to say it, well over a trillion dollars in sales. That is exactly why, if extrahepatic truly works, Arrowhead scientists should be multi-millionaires.

So that begs the question, HOW DO YOU VALUE SUCH A COMPANY that has a flexible platform to hit all sorts of other targets? It HAS to have a valuation beyond the existing portfolio, as you know that it's the goose that's laying golden eggs. It's easy to throw any number out there for many reasons, i.e. $1B, $5B, $20B, $30B, or even $50B or $100B, as you know that this platform will end up delivering and provide a fantastic ROI. The drug candidates are there, the need for more effective drugs in other tissues is there, and the money to pay for these drugs is also there.

So that's the valuation dilemma. How do you fairly value the TRiM platform once extrahepatic proves out? I have plenty of ideas, and it will be interesting to see how analysts move forward. I do think it would help if the company laid out a 15-30 year plan of what is truly possible, as that would help investors understand the amount of potential future revenue that's at hand. That would also help value TRiM. Chris wasn't kidding when he was using the word franchise to describe all the various different tissues and targets they can go after. I think he knows he's sitting on a gold mine, and he just needs some time to see it through.



Om deze vooruitzichten heb ik me al een tijdje geleden voorgenomen dat Arrowhead zo ongeveer het laatste aandeel is dat ik ga verkopen. Tot heden zijn er geen veiligheidsissues gemeld met de niet -lever gerelateerde rnai siRNA's. Rnai heeft vanaf het TRIM platform bewezen dat de pre-klinische resultaten een goede voorspeller is gebleken. De beurskoers is daarbij naar mijn idee nog geheel gebaseerd op de lever gerelateerde indicaties terwijl al jaren bekend is dar rnai een veel bredere toepassing heeft. Wat wil je nog meer?









Tom3
0
Zelfs Dirk schrijft weer positief over Arrowhead:

twitter.com/RNAiAnalyst/status/138741...

Zou hij weer aandeelhouder zijn geworden nadat hij zijn Novavax positie (met dikke winst) heeft verkocht?
mvdln
0
quote:

Tom3 schreef op 29 april 2021 15:11:


Zelfs Dirk schrijft weer positief over Arrowhead:

twitter.com/RNAiAnalyst/status/138741...

Zou hij weer aandeelhouder zijn geworden nadat hij zijn Novavax positie (met dikke winst) heeft verkocht?

Ik heb gisteren mijn positie in Novavax ook mooi winstgevend gesloten! Nu nog PHIO...

Wie weet, als je jezelf RNAiAnalyst noemt dan kan ARWR bezwaarlijk ontbreken, toch?
nelis h
0
nog even geduld met Vertex VX-864 in AAT

---

Patients enrolled in the Phase 2 proof-of-concept study for the Z-AAT corrector, VX-864, have completed the 28-day dosing period. The study includes a 28-day safety follow-up period which is ongoing, and results are expected in the second quarter of 2021.
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