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Arrowhead Pharmaceuticals, Inc. (ARWR)
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Oppenheimer analyst Jay Olson raised the price target on Amgen (NASDAQ: AMGN) to $275.00 on the belief that Olpasiran has the potential to drive upside while noting multiple other catalysts.The analyst stated "We're especially encouraged by olpasiran, which is an siRNA that inhibits Lp(a) for ASCVD patients with an ongoing Ph2 trial. We forecast risk-adjusted (41% #$%$ estimate) olpasiran sales of $1.5B in 2030.
Chris Anzalone Q&A Q1 2021 CC on Feb 5.....
HIF-2A...We have done as much as we have could -- as we've been able to do in animal models, but as you know, translation is always a trick with oncology. And so we're just waiting to see -- we're LOOKING FORWARD TO SEEING THE DATA OVER THE NEXT COUPLE MONTHS and we'll see where we are. The animal data have been good. We feel confident that we'll see knockdown. We don't have a good idea about how much knockdown you really need to see to see a clinical benefit. But similar here, I think we would call a win 50% or so knockdown. That's a bit arbitrary, but that feels reasonable to us, and so we'll see how we stack up against that over the next few months.....later in the CC Q&A... With regard to HIF-2 alpha, I tell you, the -- since this is our first solid tumor targeting program, we're really just focused -- our primary focus here is just two things: safety and tolerability, of course, and so far that's been acceptable; and second, knockdown. And as I mentioned there, we just haven't seen data yet. Those biopsies are going to be batched, and we'll have an idea oveer the next several months what kind of knockdown it gets. But we're really focused on knockdown, not only for the drug for ARO-HIF2, but also for the franchise. If we see that we are getting consistent and good knockdown. And what does good mean, it's hard to say. But it sort of feels like if we are in the 50-or-so percent knockdown range, we feel pretty good about that. If we can hit that, then we think we've got a drug, and we think we've got a franchise. HIF-2 alpha, as you know, is well-validated target. There have been other -- there's another drug right now in development that looks quite good. And so we think that as long as we can show reasonable knockdown, there's a place for us with this drug. And then again, we see a lot of upside. If we can knock down HIF-2 alpha in solid tumors, we can knock generally, we think, a whole host of other cancer targets. And so fill in the blank, there's a number of targets that we're going to want to go after, maybe ourselves, maybe in partnership with others, against various tumors. So we're in a really interesting, really exciting spot right now, where we're just waiting for data. And if those data look positive, then I think we're off to the races....later in the CC Q&A.. My hope is that we see some good positive data in ARO-HIF2 that would enable us to bring in a good partner to start to really blow out that franchise. It doesn't mean that we won't have our own wholly owned offerings, but at some point, it would be -- we'd be certainly open to working with a broad-based oncology company with deep expertise to help us prioritize targets and such. We're not there yet. We want to generate some data first. But at some point, that probably makes some sense for us......further in the CC Q&A....Keay Thomas Nakae -- Chardan Capital Markets -- Analyst
Yeah, thanks. Chris, with respect to HIF, if your working thesis right now is that 50% knockdown could have an effect on the disease, should that prove to be too low based on your current dose levels and dose intervals? Do you believe you could go higher than that to achieve a higher knockdown if needed?
Christopher Anzalone -- President and Chief Executive Officer
Yes. We'll see. It'll be really helpful to see what the dose response looks like if, in fact, we see 50% knockdown at the highest dose we're giving. AT LEAST FROM WHAT WE'VE SEEN SO FAR, WE SEE NO SIGNS THAT WE CAN'T ESCALATE MORE. This is not -- it's not a traditional cancer trial, right, where we dose as high as we can until we get people sick and then we knock those down. We don't expect those living toxicities. And so to your point, sure, it is certainly possible that we get 50% knockdown and we still think that there's more knockdown to be had. And as long as we're not seeing dose limiting toxicities, we're happy to escalate. And like I said, in neither HIF-2 alpha nor in ENaC so far have we seen evidence that we've got a safety issue.......my overall takeaway...CA looking for 50 percent knockdown along with safety and tolerability. CA stated with 50% knockdown he believes ARWR has a drug...and also a franchise....with over 50% knockdown look for ARWR to bring in a broad based oncology partner to help blow out the franchise....CA also stated he was really interested in seeing the data over the next couple months...it has been 2 months since the Q1 CC... I think he now knows if the 50% knockdown can be achieved (this assumes safety and tolerability are met)...CA tupically has unserpromised and over delivered...so I am expecting knowdown to significantly exceed 50%...we shall soon know...I am looking forward to the sharing of data...stay long and stay strong...the intrinsic value of ARWR far exceeds the current market cap value. Am I missing anything?
The HIF2a study is open label started in Aug. 2020. 4 months into the study in Dec, 2020 the study was expanded from 18 to 30 patients. These are severely ill patients with an Inclusion Criteria that reads:
"Histologically confirmed locally advanced or metastatic clear cell renal cell carcinoma that has progressed during or after at least two prior therapeutic regimens which must include vascular endothelial growth factor (VEGF)-targeted therapy and checkpoint inhibitor therapy or that has otherwise failed such therapies, is measurable disease per RECIST 1.1 criteria, is biopsy accessible."
You would expect a certain % of patients dosed in Aug. 2020 to pass away or remain in very foul shape by Dec. 2020, yet the program was expanded in dosing locations and patient numbers. This is a key fact.
April marks another 4 months of dosing/observations.
Will Arrowhead release top line data before the May ER CC?
Take a look at the Dec. 2020 Study Update:
History of Changes for Study: NCT04169711
I think the only real risk here, long term, is being out of the stock when the good news comes. For those thinking of selling at this point and buying back in, I want to remind you of the morning it went up $19 with one PR.
Just don’t see the stock price dropping too much lower with the upcoming catalysts. Also, CA made a great call to maintain ARWR’s rights to APOC, ANG3 and 50% of AAT. These are pure gold for ARWR. HSD will be another one. If DRNA had not licensed out most of its liver targets to Lilly, Novo and Alexion then its MC would be in the same ball park as ARWR. Both are really strong investments though and the future is very bright for both with the seemingly limitless extra hepatic targets. Long in ARWR and DRNA.
Making the case....I am very interested in putting sideline money into my existing Arrowhead investment. The next couple of months I view as Binary for this company. While I look at the current share price and acknowledge an undervalued company, I cannot bring myself to buy more stock until I get a read on the extra hepatic programs.
If the readout is growing pains will ensure like it or not the stock could easily find itself in the 30s-40s. If they readout with margins seen close to liver with great safety it easily surpasses the current high on the board.
What was guided for readouts will truly be a turning point for this Company and change it's dynamics for the next 3-5 years. The POC tethered to Tumor and Lung delivery is what will define Arrowhead as an RNAi leader and a breakout company or keep it grounded with it's peers.
HIF2a has always been my personal favorite program from a strict Science standpoint. I have great hope in the upcoming data but stay grounded in the fact Arrowhead is deep in the clinic accomplishing "firsts" for the RNAi field.
I eagerly await the morning I can buy this stock at 100 if they readout successful. Based on non existent volume of late it seems many are waiting for the same.
As a long-term investor I am more excited and nervous for this company than I have ever been. If Tumor and Lung readouts are a phenomenal success this could quite easily be a 20B company by year's end.
With or without extra hepatic programs this is an amazing portfolio for future growth, but with extra hepatic success this will be a 1 of 1 company and no longer an AMGN in the making but an AMGN awaiting recognition.
I hope you all have a great day, and MAAANNNN am I pumped for what's coming!
I am wondering if I stumbled across Arrowhead’s skeletal muscle target that is supposed to come out this summer. This is from Tao Pei's linkedin bio:
Group Vice President, Chemistry
Jun 2014 - Present6 years 11 months
Madison, Wisconsin Area
1) RNAi drug delivery platform research
TRiM platform in Liver, pulmonary, skeletal muscle
2) RNAi drug discovery
inventor of Olpasiran
3) discovery DMPK
From #1 in his bio, we know that he is/was working on the skelatal muscle program. Not really related, but #2 is pretty impressive. Now for #3. I had no idea what DMPK was, so when I googled it I found out that DMPK (DM1 Protein Kinase) is a Protein Coding gene. Diseases associated with DMPK include Myotonic Dystrophy 1 and Myotonic Dystrophy which are skeletal muscle diseases.
Once in a while, an event occurs that lifts the veil on what RNAi assets are actually worth. Today, it was Dicerna selling their MINORITY (from the Dicerna 10-Q "pay mid- to high-single-digit royalties") stake, call it 8%, in Alnylam's PH1 drug, Oxlumo / Lumasiran for $180M upfront and $60M contingent. Keep in mind Dicerna has Nedosiran, which treats all three types of PH (vs. just PH1 for Alnylam's). Also keep in mind this is a small-ish orphan indication.
Now look again at the total pipelines of companies like Arrowhead and Dicerna (wholly owned AND partnered assets). Do their valuations make sense?
Buying more of both.
New, reversible CRISPR method can control gene expression while leaving underlying DNA sequence unchanged. In a paper published online in Cell on April 9, researchers describe a new gene editing technology called CRISPRoff that allows researchers to control gene expression with high specificity while leaving the sequence of the DNA unchanged. Designed by Whitehead Institute Member Jonathan Weissman, University of California San Francisco assistant professor Luke Gilbert, Weissman lab postdoc James Nuñez and collaborators, the method is stable enough to be inherited through hundreds of cell divisions, and is also fully reversible.
A quick thought to share with my YMB investor friends. Been learning some amazing things these past years on my ARWR investment and due diligence journey about the company, the science, and RNAi and I want to thank all of you who have contributed such great content to the YMB. ARWR has taught me a very sobering and humbling lesson that the best way to grow wealth quickly is to do it slowly. I’m learning that I’ve had to also grow my EQ along with my ARWR investment. Without the slow growth journey it could have crushed me or I could have lost it by making hasty or immature investor new wealth decisions. I’m guessing a number of the regular long term ARWR YMB posters might be able to relate.
“Wealth gained hastily will dwindle, but whoever gathers little by little will increase it” - Proverbs 13:11
The next 60-90 days could be our best yet. GLTAL’s, Go ARWR!
I haven’t posted in a while. I hope all are well. I started a new job and am tasked with leading a sales force that is interesting and presents challenges but I see the light for them in the tunnel.
That said. Any day without news means nothing bad has happened that they need to tell us about. It also means the clinical trials are being pushed forward and paid for by ARWR. Every dollar ARWR spends on products that are showing safety and efficacy will be returned to shareholders in many multiples as these products prove themselves in patients.
I thought to myself about a year ago that they could partner a product ever other month and be a traders/media darling or they could utilize their cash hoard and push these closer to commercialization or commercialize themselves and be an investors dream.
I like that they continue to move the pipeline forward and in a relative blink of an eye, many of us will be rewarded. Time will tell, but the risk reward in my opinion is heavily tilted to the reward side of the equation.
Know what you own
Only invest what you can afford to lose
Stay off margin
Sell if you need cash
Don’t set stop losses as you will likely get whipsawed
Hug those you love abs remember what matters.
The value is in what we don’t know.
Have fun and smile
The next 90 days should be interesting and allow the rest of the world to see the light.
Always in my opinion!
Changes to three JNJ HBV trials posted today.
Noted PENGUIN trial participants expanded n=20 goes to n=50, and
REEF 1 solidified Completion Dates from anticipated to Actual.
Search of: JNJ-73763989 | Recruiting, Not yet recruiting, Active, not recruiting, Enrolling by invitation Studies - List Results - ClinicalTrials.gov
Sanofi acquires Tidal Therapeutics, a pre-clinical mRNA platform company, for about $500m after achievement milestones.
"Know what you own, and know why you own it." — Peter Lynch
Reading the board recently it becomes clear that these are more challenging times for a few ARWR investors and regular posters to stay the course. Even though we know and agree to ARWR having rock solid safe RNAi science and best in class partnerships and an industry leading business plan, having no recent PR and constant short FUD spreaders working the YMB daily to shake loose weakened share holders wears on ones nerves. We’re herd animals. Fighting against our natural instincts are some of the hardest things we’ll ever have to do. Our selling emotional fear is twice as intense when the stock is low as compared to our buying emotional high which leads us to sell and buy at the wrong times to play it safe or follow the herd. Regarding your ARWR investment just keep in mind the following Warren Buffett quote:
"I will tell you how to become rich. Close the doors. Be fearful when others are greedy. Be greedy when others are fearful.
GLTAL’s Go ARWR!
Did anyone discuss HSD farther ahead on the pipeline chart than HIF2a or ENAC? I have a feeling that HSD has a high probability of a partnership with much larger upfront and then similar revenue sharing as the LPa Takeda deal. Each deal has gotten better as they progress their science and we know multiple BP's were interested in them targeting HSD. That could be the next major shot accross the bow. Or about a 100 other things.
Has anyone comped the enterprise value immediately post $58 Goldman / Jefferies offering with the current EV? (shares then outstanding x $58/share minus cash vs. current shares outstanding x $63.50 / share minus cash). Compared the pipeline from that November 2019 offering prospectus w/ the pipeline as it stands now? What about XBI where it was then vs. where it is now?
I have, and if you're anything like me, you've probably reached the same conclusion:
1. The business is trading at about the same valuation as it was 18 months ago despite significant pipeline progress and growth, another 18 months of pristine safety records, balance sheet derisking, PoC in AAT / Takeda deal, etc. etc. etc.
2. The XBI is a red herring.
Admittedly, I was a bit annoyed with all the "buy the dip" talk when the dip was $85 ---> $83.50. Didn't think long time investors should have been pumping / pounding the table near all time highs. But now I'm not seeing much of that talk, when, ironically, this is when you should be telling your friends and family to start buying.
Will it go lower? Maybe. Do you want to nitpick over a couple points when the long term story is better than ever?
Someone picked up 100 of the Sept 21/135 calls today. Let's hope it's a good investment.
Ionis Enac data of 4.5% FEV1 improvement on top of existing CF treatment is a promising sign for our EH targets and I hope we continue to outclass ASOs with RNAi like we did in hepatics.
New franchises set both in Pulmonary and Cancer will certainly take us to a whole new level in our quest to BP status. I was in ARWR primarily for HBV and now that has become a bonus goal with our best in class wholly owned cardio duo drugs (~20bn potential in 3-5 years). But I can't ignore HBV and do believe JNJ will know internally how the 1st 2 REEF performs by end of Q3 this year. I am most interested in NCT04129554 the triple combo 12 monthly dosing in e- patients.
JNJ should know whether the triple cocktail works by end of Q3 2021 after 1 full year post dose. We have yet gone beyond 5 dose in past ARC human trials so this Q4 2021 could be a big surprise to our pipeline. Imagine J&J initiating P3 enrollment to 10k HBV patients worldwide and how that will translate to our share price but I believe they would likely reach a compromise with ARWR before then. I mean you would if you were in JNJ's shoes. You can't ignore the facts that ARWR potentially has the best in class cardio drugs, probably a break into VRTX's monopoly with CF, and a whole non-toxic franchise in Cancer drugs and other breakthrough cell types. Recall CA's conference where he actually convince the audience his company is in better positioned to be acquired as they have not mass partnered their entire pipeline like their close counterparts have.
I am excited about our future but at the same time, a bit disappointed that the powers would present a check big enough and cannot be refused by CA. Whatever that number is, hopefully it surpasses Moderna's peaked MC valuation (70bn due from Covid craze) to be content with the offer; however, that will probably be unlikely, and I bet $35bn is a more acceptable low end offer given we are still 3-6 years away in maturing our pipeline and commercialization.
just more hallucinations from the drought of news but I do hope we get a $1-2 bn upfront cash on one more of our drugs and put this potential steal in the backburner. Will it be HIF2a, HSD, or CF that will be sacrificed for our freedom?
Vertex is guided to report data this quarter (2Q) for VX-864, its small molecule corrector, for A1AT patients. If the data is weak, then it will help both DRNA and ARWR given their respective A1AT candidates. On the other hand, if VX 864 data looks good then this clinical candidate will potentially correct the misfolded protein for A1AT patients and address both the lung and liver concerns for A1AT patients. Keep your eyes open for this PR.
I know there hasn't been a heck of a lot to talk about, but I still miss seeing posts from BioBoyScout & Dr. Cheddar. Hopefully soon.
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