pikva

@GothamGirl_1 $sgmo What about a deal with Royalty Pharma for money now in return for a share of royalties for Fabry? $ARWR did something similar for a share of potential royalties for Lp(a) drug still in clinical trials. $ions did a similar deal with Royalty Pharma a while ago.

$SGMO Some here have said Sangamo could not go-it-alone with Fabry but it seems they intend to do just that. So, how do they get from here to commercialization without a partner? And how do they move forward with the pain trial? And Prion?

$sgmo I’d like sgmo to take Fabry to the market by themselves instead of partnering, if they can get the funds to do it. A small number of employees would be needed. Revenue estimates seem to be about $500m per year, which would support a billion+ valuation and fund other drugs.

@BioBoyScout $IONS $ARWR Litigation cost ($20m?) nothing to IONS. iONS story of pioneer with copycat ARWR years behind stealing IP - plays well with a jury. Lawsuit is big overhang for ARWR if they want to get bought out. An acquirer will want it settled. Outcome - ARWR pays 10% royalty.

Just released a new Investor Note on $ARWR and the $IONS patent lawsuit. Bottom line: only the lawyers win. Asymmetric downside for Ionis, minimal risk for Arrowhead, plus Redemplo’s $60k pricing quietly helps Ionis in sHTG. drive.google.com/file/d/1sfGW6G…

@BiomedicalRX $IONS $ARWR The following slides regarding IONS BBB delivery are from their October 2025 Innovation Day presentation.

@BiomedicalRX $IONS $ARWR Fantastic data by ARWR. They may have leapfrogged IONS in subcutaneous to the brain. Could be a game changer. Interesting to see how IONS will respond, as I’m sure they’ll get questioned about it. IONS has said they’re close to BBB themselves.

$ARWR BIG 🔥 👏

@ArtsStocks @RNAiAnalyst Generally, to invalidate the patent, arwr will need to show that there was prior art before the ions invention that disclosed the ions invention or made it “obvious” in view of the prior art. $ions $arwr

@RNAiAnalyst So $arwr is claiming that the patent that was granted is invalid. How does that work? $ions saw this coming when they filed the patent.

$ARWR vs $IONS: thoughts on patent dispute. 1st: unlikely to be relevant since main claim refers to treating LPL deficiency...in ANIMALS. Unless you interpret humans=animals, since all other claims are dependent claims...forget about it. Overall, remarkable that such broad drug target (method) claim issued. Targets per se are not patentable, but in this case, it came as a big surprise when Ionis found in clinical trials that LPL deficient PATIENTS responded to ApoC3 knockdown. I give them full credit for that. Main risk to Arrowhead is that since discovery was based on clinical observation, later claims get issued that cover LPL patients (considerable part of FCS market). Having said that, Ionis litigation, trying to prevent a better ApoC3 drug to reach market, will not go down well with patient and physician community. In a way, great advertisement for plozasiran.

@RNAiAnalyst Column 6, lines 22-25 of the specification of the patent (US 9,593,333) specifically states that “‘Animal’ refers to human or non-human animal”. So, the claim covers humans. $IONS $ARWR

@BikeRieder It seems that $ARWR has been waiting for $IONS to validate a target before proceeding with their drug. It is possible that $ARWR infringed some $IONS patents. Anyway, I wouldn’t worry about it. $IONS likely won’t be able to stop $ARWR drugs, but may be able to extract a royalty.

$ARWR what's about that? Arrowhead Pharmaceuticals Files Complaint for Declaratory Judgment Against Ionis Pharmaceuticals ir.arrowheadpharma.com/news-releases/…

@hobielandrith @Xiphos_Trading Lots of long term $IONS holders out there. I’ve been holding since the infamous Worth Magazine cover story in 1997 titled “The Next Microsoft” in which IONS (then “ISIS” was picked as one of the contenders.

@Xiphos_Trading I’ve owned $IONS since 1993 (shortly after ipo) . Been a long time since really good news. My odd duck bio stock.

@auditor112017 The market is tired of $ions due to years of disappointment. $ions will probably need to show real revenue growth to break out of this, which should happen soon. Their presentation at J.P. Morgan shows revenue starting to grow quickly and reaching $5B+ in 2028 and beyond.

If the market believes that $ARWR Plozasiran is superior than $ions Olezarsen, why is $arwr is still trading near its low? It is likely due to the overall weakness of the biotech sector and short sellers are taking advantage of it. $xbi

@wallstlobo $IONS is probably 5 years ahead of LLY. LPa space is definitely getting competitive, but IONS is years ahead of everyone and will definitely get a significant market share in advance that may not want to switch if they are having success.

$IONS Looks like the market woke up to LLY's LPa oral drug bye bye pelacarsen

@GeneInvesting @Biotechbull2 You can’t undue gene therapy if side effects occur. With $IONS, you could just stop the injections. $IONS

@Biotechbull2 It will have an incredibly broad label, just wait and see for HAE. Patients of all ages etc. For a patient who is 20, you could get one treatment from $NTLA, or 720+ from $IONS over the next 60 years. I’d be much more worried about side effects from 720 injections than 1 CRISPR

Dear $IONS lover, That is probably the worst example I’ve ever seen. Many feel horrible the first day after receiving the vaccine, and even I had chills. I’d sleep WAY better at night without worrying about having more attacks, and having to constantly make certain my medication was covered by my insurance. You are essentially cured with $NTLA, and with $IONS you are still diseased and require chronic treatment. 1/3 of the patients will have a clear choice with $NTLA. Out of the other 2/3 you will find many who prefer not requiring chronic treatment every month for the rest of their lives. You can get more gene altering when you get a sunburn. ☀️ Yours truly, Gene Investing

@TomSilver39 The weak data based on a rushed phase3 gives a huge opening to $IONS. IONS planned a longer, more thorough P3 based on changes in the patient population and the use of Tafamedis.$ALNY $IONS

How does the $ARWR data compare to $ions? finance.yahoo.com/news/arrowhead…

@MattWallace888 @TaraBull808 The Castellows

This all girl band is quickly rising up the charts. What do you notice about them?

$ALNY changing their Helios-B trial analysis late in the game. $ions made a decision long ago to expand their PH3 in ATTR-CM to include many subgroups, including mono and combined with tafamedis. $ions just gained an edge.

@auditor112017 @mRNAdisrupts Bayer returned F11 to $ions to instead focus on their own F11 drug, asundexian. But, as recently reported, asundexian failed a p3 trial. Maybe Bayer re-licenses F11 from $ions?

@mRNAdisrupts $ions Factor X1 could have been the 1st to market. Now, it seems like a me-too drug. Very bad decision to partner with #bayer knowing they're working on a competing drug. Will this drug ever get to the market?

#Bayer $ions $azn $lly $pfe $gsk #Roche $tak $jnj healio.com/news/cardiolog…

@RNAiAnalyst With all the data that’s been coming out at #AHA2023, $IONS antisense is looking very competitive with APOC3 $ARWR and LPA $SLN at therapeutic doses. Plus, IONS has a multi year lead. I reduced some $ARWR today, and added some $IONS. $IONS also very competitive in TTR $ALNY.

Old-fashioned RNAi! $LLY $ARWR $ALNY $SLN

@auditor112017 @mRNAdisrupts I think the only thing holding $ions back is the believe that rnai is better than antisense. $Ions liver drugs are being chased by rnai ($arwr), and $ALNY and $Arwr are getting into neuro. But, I think that antisense is doing well in this competition.

@mRNAdisrupts Analysts need to increase their valuation of $ions CNS pipeline. They now have blockbuster Spinraza for SMA and an approved ALS (SOD1) drug, and positive results from their TAU drug and now Angelman syndrome. This shows that their CNS platform works.

A majority of patients showed improvement in overall functioning on the #AngelmanSyndrome Clinical Global Improvement Change (SAS-CGI-C) scale, which evaluates clinicians' impressions across 9 key functional areas. $ions $biib #neurology #Anglemans #geneticdisease #genetherapy

@RNAiAnalyst In P2, I believe $ions reported 62% trig reduction for 50 mg monthly in hypertrig. P3 is 80 mg monthly, which probably has similar trig reduction as $arwr.

$ARWR ApoC3-related AHA abstracts highlights: severe, > 500mg/dL hypertrig group: -66-74% trig reduction (depending on dose; 2 doses) mixed dyslipidemia, 150-500mg/dL: -52-64% trig reduction Main apparent drawback/AE: worsening of glycemic control. This is what I've been saying: how does $VERV $CRSP intend to manage irreversible glycemic control AEs after genome editing? #RNAi/ASOs are reversible.