BioBoyScout
@BioBoyScout
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Independent Biotech Analyst & Patent Atty. Robert Toczycki, JD, MBA. Developer of online biotech valuation calculators. Chicago native. Opinions my own. RNAi
Joined February 2015
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See a risk-adjusted DCF (discounted cash flow) model in action, just like what Wall Street analysts use. You can control the numbers and develop your own valuation.
@BioBoyScout
's $96 Price Target for $ARWR -
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If there's ever been a
#biotech
company that's at least 5x undervalued heading into
#JPM24
it's $ARWR. Its Pulmonary
#RNAi
franchise is easily worth $10B+ today, yet WS Analysts completely hide it from their valuations. Mark this post!
#JPM2024
1/6 Here's why TD Cowen knows that home run pulmonary data from $ARWR is EXTREMELY valuable. Please read my Revelatory Research Note on ARO-MMP7 below.
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Interesting
#RNAi
company timeline. $ARWR ($3B MC) now has as many drugs in the clinic as $ALNY ($22B MC), and has been on fire with drug development the last 5 years with the latest generation of RNAi drugs. $NVO
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$ARWR - if you want to search Twitter and exclude all the nonsense discord posts, use the following search term:
$ARWR -url:discord
This will specifically exclude all posts that have a link to discord. Instantly all the garbage will disappear. You can do this for any stock.
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1/6 Here's why TD Cowen knows that home run pulmonary data from $ARWR is EXTREMELY valuable. Please read my Revelatory Research Note on ARO-MMP7 below.
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$ARWR
Up to 97% reduction in intra-hepatic Z-AAT polymer
Are you kidding me?
#RNAi
is just an incredible way to tackle diseases. This will shake some trees.
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HUGE!!!!!!!! $ARWR Completed chronic GLP tox for pulmonary candidates ARO-RAGE and ARO-MMP7. Results were highly encouraging and suggest sufficient safety margins to proceed to Phase 2 studies.
#RNAi
#BigPharmaInTheMaking
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How is
#RNAi
maturing? Looks to be coming along nicely - judge for yourself. $ARWR $ALNY $DRNA
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#RNAi
drug development takes years of experience. You're either best-in-class: $ALNY $ARWR $NVO, or you're always playing catch-up with inferior drugs that just won't compete. Interesting timeline and stats for the top 3 RNAi players (eventual top 5 BPs).
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$ARWR ARO-AAT has now been granted its generic drug name - FAZIRSIRAN. Just recently adopted by the U.S. Adopted Name Council. Now on to approval.
#RNAi
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The only
#RNAi
player in the pulmonary space, $ARWR has no competition with genetically targeted pulmonary drugs - upside is beyond massive. Targets are genetically validated, and getting knocked-down like it's nobody's business. Smart money just starting to see this. $VRTX
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Massively undervalued $ARWR up over 5% after hours after hosting its best quarterly conference call to date. High confidence after pulmonary proof of concept, a breakthrough in CNS, and possible partnership for muscle.
#RNAi
Today we are hosting a webcast and conference call at 4:30 p.m. ET to announce our financial results for the fiscal 2023 second quarter that ended March 31, 2023. More details here: $ARWR
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$ARWR ARO-HSD - these quotes sum it up pretty well: "measurements were reduced to below the lower limit of quantitation" and "well tolerated without any identified safety signals". Congrats
@ArrowheadPharma
- you've done it again.
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Don't know why this hasn't been PR'd yet, just HUGE for
#RNAi
: $ARWR's Performance Objectives, Next Gen. Goals were met and EXCEEDED! "Achieved significant target knock down in new tissue type." Must be cancer tumor and/or lung (CF) - most likely both.
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BREAKING NEWS! - Just posted $ARWR $AMGN Olpasiran phase 3 cardiovascular outcome study treating Lp(a) now posted. Start 12/21/22 with completion of Dec. 2026! 6,000 participants. Only 4 years - that's a HUGE win. Ph2 data must be spectacular.
#RNAi
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$ARWR $JNJ Big news! Newly published patent application for "RNAi Agents for Hepatitis B Virus Infection". Filing date of Aug. 6, 2021. 214 pages - includes detailed data, as well as HBsAg seroclearance for at least one patient in the 200mg cohort.
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$ARWR BIG BIG NEWS! Fantastic efficacy and safety results for ARO-ANG3. Very comprehensive data set. Thanks for posting. Easily a $10B drug in its own right.
#RNAi
$arwr ARO-ANG3 also decreased atherogenic lipoproteins and, importantly, repeat dosing did not increase liver fat, with most subjects showing a numerical decline in liver fat content
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$ARWR ARO-C3 newly added drug nominated to its pipeline to treat various complement mediated diseases. Gotta admit it, Arrowhead's pipeline growth over these last 3.5 years has been astounding; 16
#RNAi
drugs with 9 currently in the clinic. I have three words UH MAY ZING!
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$ARWR Bravo to the new board member Dr. Hongbo Lu for acquiring 3,000 shares on the open market and setting the bar. You either believe it will deliver or you don't. I believe you made a great choice Dr. Lu - see you at $200 soon!
#RNAi
$XBI
#BigPharmaInTheMaking
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With $NVO buyout of $DRNA,
#RNAi
sector will become very much in demand. Amazing drug modality that works incredibly well, and only a few companies have mastered it - now down to just two. $ALNY $ARWR
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$ARWR BTD granted in China for JNJ-73763989 (ARO-HBV). What is required? early clinical evidence to suggest that the drug may demonstrate a substantial improvement over currently available therapies. Congrats $JNJ &
@ArrowheadPharma
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$ARWR must be seeing incredibly great interim data if they're now adding
#CysticFibrosis
patients 1.25 years into the study. Amazing!
#CF
#RNAi
$VRTX
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Congrats $ARWR - ARO-APOC3 Phase 3 for FCS now filed. Estimated start in November 2021.
#RNAi
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See a risk-adjusted DCF model in action, just like Wall Street analysts'. Control hundreds of numbers the way you like it, including sales for each individual drug to develop your own valuation.
@BioBoyScout
's conservative $107 Price Target for $ARWR -
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$ARWR Now with its 10th TRiM™ platform
#RNAi
drug (ARO-C3) posted in in less than 4 years - . Attributable to key partnerships, efficient operations, and top-notch scientists that have been at it for 15+ years.
#BigPharmaInTheMaking
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Major Congratulations
@ArrowheadPharma
$ARWR for hitting a home run on 3 major world-changing
#ASCVD
targets at
#AHA22
: Olpasiran - Lp(a), APOC3, and ANGPTL3.
#RNAi
is now the hottest drug modality in town.
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8/12/2020 -
@BioBoyScout
raises $ARWR's Price Target to $107 (from $81). Maintains Strong Buy. Conservatively calculated using a risk-adjusted DCF (discounted cash flow) model. Details, including an interactive online calculator at
https://bioboyscout/arrowhead
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While institutions are busy figuring out how much to accumulate, $ARWR is quietly making significant progress on their new 285,000 s.f. $250M lab and manufacturing facility in Verona, WI. This place is huge!
#RNAi
#BestInClass
#BigPharmaInTheMaking
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1/2 $ARWR Extrahepatic Targeting Patent Pending. Published US Patent Application for "Integrin Ligands and Uses Thereof" not going unnoticed by
@BioBoyScout
. Claims seek to protect multiple structures (claims 14-17), targeting numerous tissues (claims 31, 35),
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$ARWR MUC5AC ph 1/2 ~70% complete, RAGE ph 1/2 ~65% complete. Now likely dosing asthma patients. Looks like no issues to date. If proof-of-concept is shown I predict multiple $100+ price targets from analysts.
#RNAi
#pulmonary
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$ARWR newly issued U.S. Patent No. 10,544,418 today for "RNAi Inhibition of Alpha-ENaC Expression." Formerly owned by $NVS & $ALNY - acquired by $ARWR in $NVS IP acquisition in 2015.
#CysticFibrosis
#RNAi
#biotech
#GameOn
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$ARWR Moving on up with an initiated Buy and $95 PT from
@UBS
. Suspect that extrahepatic PoCs will soon push it higher.
#RNAi
quickly becoming a must have in portfolios, as its proving to be one of the most effective, versatile, and safest drug modalities out there.
#ProveMeWrong
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$ARWR's HBV drug progressing strongly and rapidly - 11 different clinical trials without a word from JNJ, so as to keep the competition guessing, as each month of exclusivity adds ~$1B in Rev. BTD in China (7/19), EMEA to allow pediatric dosing (7/28). Not on analysts' radar.
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$ARWR going after asthma using an
#RNAi
nebulizer. CTA for ARO-RAGE filed this morning. No other RNAi (or ASO) lung competitor in sight. Definitely excited about this one.
#BigPharmaInTheMaking
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8/10/2021 -
@BioBoyScout
raises $ARWR's Price Target to $133 (from $107). Maintains Strong Buy. Conservatively calculated using a risk-adjusted DCF (discounted cash flow) model. Details, including an interactive online calculator at
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AMG 890, $AMGN's licensed CV drug from $ARWR now has a new name: Olpasiran. Fast Track designation granted by FDA.
#RNAi
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$ARWR ARO-RAGE now recruiting patients at Respiratory Centers, suggests no NHV safety issues and that final 2 Asthma Patient cohorts are being dosed. This is the trial with circulating biomarkers that can show proof of concept. End of study May 31st.
#RNAi
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$ARWR Pulmonary proof-of-concept will be a big event for Arrowhead and a major extrahepatic
#RNAi
scientific advancement. Analysts currently giving no value to pulmonary. What are the chances? I say very high - see key slides from Pulm. R&D day - very compelling - 30%+ upside.
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$ARWR books $120M in revenue today with the closing of the GSK deal for its NASH drug ARO-HSD. Another $30M on the way at the start of phase 2. Deal worth over $1B in milestones PLUS tiered royalties ~20%.
#RNAi
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BAM! From the NEJM, $ARWR planning a "three-group cardiovascular outcomes trial comparing zodasiran (200 mg) and plozasiran (25 mg) with matching placebo."
#RNAi
#CVOT
#Unprecedented
#BigPharmaInTheMaking
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1/3 $ARWR why no licensing deals yet? One theory is that they're keeping as much wholly-owned as possible for a massive buyout. With chronic tox results & GSK-JNJ deal completed as of last week, this now clears the way and guarantees max value.
#RNAi
$GSK $NVS $VRTX $AMGN $JNJ
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$ARWR why the big interest now? At Morgan Stanley HCC Arrowhead revealed the patient population for FCS to include those with TGs > 880+pancreatitis. This ADDS 70-100k in the US and is a VERY BIG DEAL. Even Steven Nissen loves this drug. Needless to say, $IONS is not happy.
#RNAi
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$ARWR continuing to move full steam ahead with 3 newly published US Patent Applications today covering DUX4, Skeletal Muscle Delivery, and the almighty MMP7 trigger targeting IPF. These are majorly impressive write-ups of 352, 105, and 348 pages.
#RNAi
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It took 6.5 yrs for $ALNY to get its 1st FDA approval for Onpattro®; 9-12 months earlier its valuation was between $7.4B -$12.3B with only 8 drugs in the pipeline. $ARWR's APOC3 and ANG3 are at 4.5 yrs and Fazirsiran is at 5.5 yrs with multiple approvals around the corner.
#RNAi
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$ARWR ARO-RAGE is just "crushing" RAGE in its knockdown.
#RNAi
clearly proving out to be a best-in-class modality for
#pulmonary
. With 3 current targets in the clinic having massive potential, there's no way any buyout under $15B should be accepted. $VRTX
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Here's an interesting stat. For the last 6 years $ARWR drugs have been averaging at least 2 new clinical trials for every 3 months. With
#RNAi
being the most successful genetic drug platform, this pipeline surely won't disappoint.
Interactive chart at:
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$ARWR Knocking it out of the park again. Congrats on yet another successful data readout. These potent and safe, targeted
#RNAi
drugs will reshape medicine as we know it. Can't wait to see more at R&D Day.
“Conceive the inconceivable – then accomplish it.” - Jim Valvano
Today we presented interim data at the 91st
@EASCongress
on ARO-ANG3, an investigational
#RNAi
therapeutic designed to reduce the expression of ANGPTL3 in patients with
#HoFH
. A webcast link to the presentation is available on our website. $ARWR
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Boom! $ARWR Chris just confirmed at the TD Cowen fireside chat that its Plozasiran ph3 trial for FCS includes clinical definition of FCS (patient population into the thousands) - see slide in post below.
#RNAi
$ARWR why the big interest now? At Morgan Stanley HCC Arrowhead revealed the patient population for FCS to include those with TGs > 880+pancreatitis. This ADDS 70-100k in the US and is a VERY BIG DEAL. Even Steven Nissen loves this drug. Needless to say, $IONS is not happy.
#RNAi
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$ARWR If there's ever a time to add or jump in, it's probably NOW. On the cusp of many newsworthy events and milestones: AAT, HBV, HIF2, Lp(a), HSD, etc. and growing pipeline. High analyst price targets have remained stable over last year and will prob. soon get another boost.
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$ARWR For posterity purposes, just putting this out there. The Revelatory Research Note below is just for one drug - ARO-MMP7. $6B buyout would be a joke.
#RNAi
#IPF
#IdiopathicPulmonaryFibrosis
1/6 Here's why TD Cowen knows that home run pulmonary data from $ARWR is EXTREMELY valuable. Please read my Revelatory Research Note on ARO-MMP7 below.
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Dual lipid platform! Wow! Congrats to the R&D team. Yet another leap forward over the competition. $ARWR
#RNAi
#BestInClass
Our
#TRiM
platform is expanding to tackle more diseases and target a wider array of cell types. Check out our latest presentations at
@TIDESInforma
USA 2024 to discover our breakthroughs in delivering therapies to the CNS and adipose tissue.
#TIDES2024
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WOW! $ARWR $AMGN Best Lp(a) data out there. "These data demonstrated a significant reduction from baseline in Lp(a) of up to or greater than 90% at week 36 (prim. endpoint) and week 48 (end of treatment period) for the majority of doses. No new safety concerns were identified..."
$ARWR
AMGN topline data ph2 OLPASIRAN:
David M. Reese, M.D., executive vice president R&D: "We are very ENTHUSIASTIC about these results and look forward to advancing olpasiran as a potential treatment for patients with elevated Lp(a).
Phase 3 very soon!
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$ARWR Congrats on introducing yet another pulmonary drug targeting TSLP for Pulmonary Allergic Inflammation.
#RNAi
#BigPharmaInTheMaking
We are participating in multiple conferences this September. Read our press release for details here: $ARWR
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$ARWR newly issued U.S. Patent No. 10,550,391 today for "Organic Compositions to Treat Beta-ENaC-Related Diseases." Former $NVS & $ALNY IP acquired by
@ArrowheadPharma
. Treats:
#CysticFibrosis
#PHA1
#LiddlesSyndrome
#Hypertension
#Alkalosis
#Hypokalemia
.
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$ARWR let that sink in - LOL!
Interesting $ARWR tidbit: since 2018 the company developed and delivered a total of 17
#RNAi
drugs (3.4 per year). 3 were not advanced by $AMGN & $JNJ, 2 were dropped, and 12 currently remain in the clinic. Development and growth continues at a strong pace. Let that sink in.
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$ARWR $TAK Multiple outlets now reporting that Fazirsiran (aka ARO-AAT & TAK-999) for the treatment of alpha-1 antitrypsin (AAT) deficiency "... will expand the international study to additional adult patients and CHILDREN ..."
#RNAi
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For those that liked the
#RNAi
Clinic Timelines for $ALNY $ARWR and $NVO I posted yesterday, here's a bit more history you'll appreciate, including dropped programs. Takes blood, sweat, and tears to get this far. Charts can be found at under "Timeline".
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$ARWR Steven Nissen excited about Plozasiran, as cardiologists need a CV drug that not only reduces remnant cholesterol, a major risk factor of coronary heart disease, but also raises good cholesterol (HDL). Data speaks for itself.
#RNAi
#MiracleHeartDrug
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@jfineman
Current investors very well know that $5.5B (or $45/share) is not enough to buy just one of $ARWR's cardio drugs, let alone the entire company with 14 drugs in the pipeline (and many more coming). Maybe $20B, but that's the absolute minimum.
#RNAi
#BigPharmaInTheMaking
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$ARWR WOW! ARO-HBV aka JNJ-3989
#RNAi
🔥🔥🔥🔥🔥🔥🔥🔥🔥🔥🔥🔥
Nearly 300 million people live with chronic hepatitis B & face daily stigma/discrimination because of their disease.
That’s why
#HepatitisCantWait
. Follow
@Hep_Alliance
and stay tuned on 11/15 to learn more on the human impact of stigma in
#hepB
& how we can end it.
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$ARWR earning 7 recent Price Target upgrades over the last few days, with a Bulge Bracket Investment Bank (Citi) topping the list at $110 (and surpassing
@BioBoyScout
's PT of $107). Brighter days must be ahead.
#RNAi
#Extrahepatic
#FirstMoverAdvantage
#PatentedTechnology
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@skathire
"But $ARWR's Anzalone questions the benefits of a one-time treatment whose safety still isn't borne out over decades vs. transient tmt options. He says the company's RNAi approach to high cholesterol and triglycerides - a once quarterly or biannual injection - is the safer bet."
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$ARWR Fantastic news, congrats Arrowhead on this deal with
@GSK
.
#RNAi
now being highly recognized by big pharma companies across the board. This is a huge win, as this drug was downplayed by many experts.
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$ARWR Well done Chris! Really like the 10 Core Values - Team Culture is essential for every successful biotech.
#RNAi
From L.A.: The growth of RNAi as a platform with the CEO of
@ArrowheadPharma
. $ARWR
Full video:
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Big news for $ARWR & $AMGN. AMG 890 designed to knock down Lp(a), significantly more than PCSK9 inhibitors, now moving on to phase 2. Affects over 20% of the population. Lp(a) linked with increased risk of CV disease.
#RNAi
#biotech
#GameOn
$arwr $Amgen Amgen has filed phase two of AMG-890 on . Estimated start date is June 10th. We should get a nice milestone payment on first dosing.
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$ARWR $TAK Great News! The SEQUOIA trial for ARO-AAT (Fazirsiran) appears to be moving on to Part B, as planned - the experimental open-label. IMO, must have seen compelling biopsy data.
#RNAi
#AAT
@AlphaFriend
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$ARWR ARO-APOC3 phase 3 study for FCS. Adding an open-label period for 8 more doses after the first 4 masked doses. Suggests data must be looking great.
#RNAi
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$ARWR just nailing pulmonary on multiple fronts: RAGE, subQ RAGE, MMP7, & TSLP. Prediction: these pulmonary drugs will easily push the MC to the $20B range prior to their approval. Analysts still not giving any value to pulmonary! Opportunity knocks.
#RNAi
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$ARWR $JNJ Slides now up: "Short interfering RNA JNJ-3989 combo therapy in chronic hepatitis B shows potent reduction ..."
HUGE - "effects were sustained in 38% of patients until Day 392 (336 days after last dose of JNJ-3989)"
#RNAi
#HBVFunctionalCure
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Congrats $ARWR for your newly issued US Patent No. 11,384,355 for Compositions and Methods for Inhibiting Gene Expression of Alpha-1 AntiTrypsin. Looking forward to forthcoming SEQUOIA results, as I'm sure it will "silence" the critics.
#RNAi
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Great news from $ARWR on ARO-AAT - Congrats
@ArrowheadPharma
.
@AlphaFriend
has to be ecstatic. Now looking for the pivot into ph. 3 - that would be a significant move.
#RNAi
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BRAVO! Chris Anzalone has made it clear, $ARWR does NOT intend to license ARO-APOC3, it intends to keep it in-house and wholly-owned through CV outcome studies, thereby greatly increasing value.
#RNAi
#BigPharmaInTheMaking
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While $ARWR may now be sporting an impressive, and growing pipeline, one cannot overlook JNJ-3989 (licensed with JNJ/Janssen). This has been hush-hush for quite a while - suspect news is on the near horizon.
#HBVCure
$ARWR's HBV drug progressing strongly and rapidly - 11 different clinical trials without a word from JNJ, so as to keep the competition guessing, as each month of exclusivity adds ~$1B in Rev. BTD in China (7/19), EMEA to allow pediatric dosing (7/28). Not on analysts' radar.
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$ARWR newly issued U.S. Patent No. 11,884,920 today for "Alpha-1 Antitrypsin (AAT) RNAi Agents, Compositions Including AAT RNAi Agents, and Method of Use."
#Fazirsiran
$TAK
#RNAi
#BigPharmaInTheMaking
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$ARWR just told BP that 4 of its cardiometabolic drugs are now off the table in China; very strongly suggests that it will keep them wholly-owned in the US. Innovative growth by keeping control in China along with a massive boost in valuation.
#BigPharmaInTheMaking
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YOU BE THE ANALYST! Announcing the debut of . Online biotech valuation calculators for $ARWR, $ALNY and $DRNA. Only one of its kind - first ever online biotech valuation. Comments appreciated.
#RNAi
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$ARWR WOW!!!
- Up to 97% reduction in intra-hepatic Z-AAT polymer
- Up to 95% reduction in intra-hepatic total Z-AAT burden
- Up to 66% and 58% reduction in circulating ALT and GGT levels respectively
- Up to 26% improvement in FibroScan values
#RNAi
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Wondering about $ARWR? Check out
@bioboyscout
's conservative $107 PT at and see what he sees. Feel free to run your own numbers to see what valuation you come up with.
#RNAi
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@VerveTx
@WSJ
@david_wainer
@WSJheard
I recently read the FDA Draft Guidance on Human Gene Editing. Its mission is to ensure safety when using a permanent, irreversible gene altering drug. Should not be applied when other options exist.
See thread:
$VERV $ALNY $ARWR
@david_wainer
@WSJ
David, check out this thread. Risks are huge, as you cannot reverse gene editing. If safer (temporary) alternatives exist that accomplish the same task, then I doubt FDA would approve based on one shot only. Check out draft guidance and ethics concerns.
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$IONS's Vupanorsen issues are 100% modality-related. ANGPTL3 is genetically validated. Not the first time ASO's have had ALT/AST issues.
#RNAi
is King! Proof: $ARWR's ARO-ANG3 reduces TG and Non HDL-C SIGNIFICANTLY (favorable safety & tolerability profile), Vupanorsen does not:
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$ARWR Actual current state of Analyst price targets (extremely bullish). Only see it going up from here due to continued pipeline growth and expansion. Great to see approval to begin building its new $250M+ R&D lab and manufacturing facility in Verona. Congrats
@ArrowheadPharma
.
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$ARWR ARO-AAT Pivotal Phase 2/3 trial design and dosing schedule all on one chart, as best interpreted, estimated, and compiled by
@bioboyscout
from known information. Shows pivot point where FDA may allow filing for approval as early as mid 2020.
#Genius
#RNAi
#biotech
#GameOn
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$ARWR New Open Label phase 2 trial for ARO-ANG3 filed today for HoFH. Expecting a parallel closed pivotal trial to follow shortly.
#RNAi
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Congrats
@ArrowheadPharma
- just amazing data on Olpasiran! $ARWR delivers again, this time with a CV drug grand slam. $AMGN must be thrilled with their partner choice. Looking forward to ARO-APOC3 and ARO-ANG3 tomorrow to complete the trifecta. Easily the talk of
#AHA22
#RNAi
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