Arrowhead Pharmaceuticals (ARWR)

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Re: Arrowhead Pharmaceuticals (ARWR)

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Arrowhead Pharmaceuticals, Inc. : ARWR ,Acquired all of Roche's RNAi business in 2011, and all of Novartis' RNAi business in 2015, including all related Intellectual property. Targeted RNAi Therapeutics Company. RNAi is a way of silencing target genes, and is highly specific.





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Arrowhead Pharmaceuticals, Inc. : ARWR ,Acquired all of Roche's RNAi business in 2011, and all of Novartis' RNAi business in 2015, including all related Intellectual property. Targeted RNAi Therapeutics Company. RNAi is a way of silencing target genes, and is highly specific.

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Re: Arrowhead Pharmaceuticals (ARWR)

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Welk persbericht zou er feitelijk eerst kunnen volgen ivm nieuwe phases ?
Arrowhead Pharmaceuticals, Inc. : ARWR ,Acquired all of Roche's RNAi business in 2011, and all of Novartis' RNAi business in 2015, including all related Intellectual property. Targeted RNAi Therapeutics Company. RNAi is a way of silencing target genes, and is highly specific.

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Re: Arrowhead Pharmaceuticals (ARWR)

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alle foto's via : https://seekingalpha.com/article/440678 ... rnai-space
All credits to : Andy Jones

Dicerna Is A Reasonably Priced Play In The Attractive RNAi Space
Feb. 17, 2021 4:56 PM ETDicerna Pharmaceuticals, Inc. (DRNA)

Dicerna has a huge pipeline of both internal and partnered RNAi products, one of which already has positive Phase 3 results.
Dicerna has over $600 million in cash with more to come from its lucrative big pharma partnerships.
Despite this, Dicerna trades at a huge valuation discount to its RNAi peers, so I believe Dicerna could make an attractive long-term investment at present.
This idea was discussed in more depth with members of my private investing community, Biotech Value Investing. Get started today »
Dicerna Pharmaceuticals (DRNA) is a small biopharma in the cutting-edge RNAi space. Dicerna has yet to get a product to market, but its close competitor Alnylam has proven the market relevance of RNAi technology, and Dicerna already has some positive Phase 3 results in its lead internal product. In this article, I give an overview of RNAi technology, Dicerna's pipeline and partnerships, Dicerna's cash flow management, and my take on Dicerna's valuation. I view Dicerna as a high-quality company with lots of upside potential and relatively low long-term downside risk.

RNAi Technology
Many diseases are caused by particular proteins, and these disease-causing proteins have historically been treated with either small molecules or recombinant proteins. Some proteins cannot be treated with either of these approaches though. Small molecules rely on attaching to a binding pocket on the surface of the protein, and some proteins do not have the right binding pockets. Recombinant proteins are typically limited to extracellular proteins and cell surface interactions and thus are unable to target proteins that stay entirely within the cell.

RNA interference is actually a naturally occurring phenomenon, and the discovery of this process in the worm C. elegans won Andrew Fire and Craig Mello a Nobel Prize in 1998. Genes code for particular proteins, and then messenger RNA carry this code to the intracellular machinery responsible for manufacturing proteins. RNA interference works by intercepting the messenger RNA before it can direct the cell to make a protein, effectively silencing the underlying gene. This happens when small pieces of RNA called either microRNA or small interfering RNA (siRNA) bind to the messenger RNA and cleave the strand as well as cause the release of the enzymes that further degrade the messenger RNA. This can occur both when the microRNA or siRNA come from outside the cell in the form of a bacterial or viral product or from inside the cell, too.



Figure 1: How RNAi Works (source: Polyplus Transfection)

These small RNA strands that create interference are themselves created through the action of an enzyme called Dicer which causes double-stranded breaks to pieces of RNA that cause the formation of both microRNA and siRNA. RNAi therapeutics then mimic this natural pathway by artificially supplying an RNA strand to the cell which is acted on by the Dicer enzyme to form either the microRNA or siRNA that then interfere with particular messenger RNA inside the cell that code for a protein that is causing disease.

Dicerna's proprietary iteration of this is called its GalXC RNAi technology platform. Dicerna's currently-in-use GalXC RNAi's are double strands of RNA with one strand being a guide strand that locates and binds to the target mRNA and the other being a passenger strand that is complementary to the guide strand and serves to optimize both the structure and stability of the RNAi molecules. This double-stranded RNA is then bound to the simple sugar, N-acetyl-D-galactosamine (GalNAc), which itself binds to the ASGPR receptor on the surface of hepatocytes (liver cells), thus achieving a natural liver targeting for Dicerna's initial set of RNAi molecules. Because of the liver targeting, Dicerna is able to deliver its RNAi therapies through a simple subcutaneous injection.



Figure 2: Dicerna's GalXC Technology (source: Dicerna's Corporate Presentation)

Dicerna is also conducting research to expand its RNAi therapeutics into other tissue types. This involves replacing the GalNAc sugar on the end of the RNAi therapeutic molecules with a different component to target entry into cells in CNS rather than hepatic tissue as well as changing the injection site to be either intrathecal or intracisterna magna.




Figure 3: Diagram Showing Potential CNS Injections (sources: SCA Source and The Neuroscientist)

Dicerna seems to be making good progress, but no CNS therapy has made it to the clinic yet. Dicerna has been using acyl groups in place of the sugar on the end of the RNA strand and the idea is to possibly combine the acyl group with other useful targeting mechanisms like maybe a peptide to ensure that the RNAI therapeutic finds its mark. This idea has a growing amount of support in the scientific literature, with some groups referring to these combined targeting mechanisms as bioconjugation.




Figure 4: Diagram Showing Dicerna's RNAi Technology With an Acyl Group Instead of a Sugar (sources: SCA Source and The Neuroscientist)

These targeting ligands are an important area Dicerna needs to progress in to be able to keep up with its two main competitors in the RNAi space, Alnylam and Arrowhead. Alnylam also uses the GalNAc sugar in some of its therapeutics, but the company is working very actively to expand beyond hepatic targeting, most notably through a five-year research partnership with Regeneron for RNAi therapeutic discovery in various CNS and ocular disorders. Arrowhead may actually be the furthest ahead in this area and touts its proprietary TRiM platform which uses targeting ligands that the company says can already target liver, lung, tumor, muscle, and other unspecified tissues. I strongly believe Dicerna's long-term value proposition is closely tied towards continued progress in this area. Falling behind in this area is one of the bigger risks with Dicerna that I believe investors need to be aware of although their progress looks promising at present.

RNAi therapy is an important area that is starting to get an increasing amount of attention which makes Dicerna a potentially appealing long-term investment. As I've described in this section, RNAi is very akin to many gene therapies except that it looks to be much more effective than CRISPR or other gene therapies for use-cases where knocking out or silencing a gene is what's needed because RNAi doesn't have to cause a break to the cell's DNA to be effective, making it a safer and more consistent approach. RNAi therapies also are typically delivered via lipid nanoparticles which don't have the same drawbacks often associated with AAV's that are used in many gene therapies. As I'll discuss more below, we've seen big growth in Alnylam and Arrowhead stock in anticipation of the usefulness of these therapies, but Dicerna hasn't seemed to benefit much from this yet.

Dicerna's Pipeline and Strategic Focus
I'll break down both the internal and partnered pipelines in more depth below, but first I wanted to write a little about how Dicerna discusses its overall pipeline focus and strategy. Dicerna is clearly making liver-targeted therapies first, but the company has also been clear that branching out is a priority, especially with CNS (neurodegeneration and chronic pain) and cardiometabolic diseases.

Dicerna is trying to keep a substantial stake in its rare disease programs that have well-defined disease markers while partnering on more complex diseases with either multiple gene dysfunctions or higher patient populations. Thus, its internal pipeline is focused on fairly de-risked opportunities - sales will be easier because patients with these rare diseases are treated at a small number of facilities and the unmet need is high. The diseases in its internal pipeline are also well characterized and revolve around typically just one dysfunctional gene.

The partnered pipeline then absorbs a lot of the higher development risk in diseases that are not as well characterized and a lot of the commercialization risk that comes with targeting larger, and often more competitive, patient populations. In a sense, the partnered pipeline is a win-win for Dicerna. The company generally bears no expense in the partnered programs and yet has been getting substantial cash milestones to support the development of its internal pipeline. Then if these programs are successful, Dicerna will make royalties that flow straight to the company's bottom-line, and in the case of its more recent partnerships, Dicerna even has an opt-in option that it can potentially exercise once its partner has already demonstrated some of the clinical viability of the product.

I really like management's thought process behind its strategic pipeline decision, and they seem to be executing well on the vision so far.

Dicerna's Internal Programs
Dicerna has a fairly modest internal pipeline at the moment with just two disclosed programs, but these two could still make a substantial impact given their rare disease nature and the type of pricing those opportunities typically command.



Figure 5: Dicerna's Internal Pipeline (source: Dicerna's Corporate Presentation)

The first, and probably biggest, of those opportunities is nedosiran which is being developed for primary hyperoxaluria. Primary hyperoxaluria comes in three forms, PH1, PH2, and PH3, which are each caused by related but different genetic mutations. These are conditions where the genetic mutations cause an overproduction of oxalate, which is a natural molecule found in plants that binds to calcium. Where the problem comes in is that the kidneys can't properly process these calcium oxalate crystals that form, and they cause problems ranging from painful kidney stones up to end stage renal failure requiring dialysis. This is obviously very serious then given that the average life expectancy of dialysis patients is just five to 10 years.

Before November, the best treatment for PH prior to the patient developing kidney failure is simply hyperhydration. Hyperhydration isn't feasible though for many infants and toddlers, so it typically requires a gastrostomy tube which is miserable for both the patient and parents. Hyperhydration is also not an adequate strategy for post-surgical patients or during other illness like diarrhea/vomiting. Dialysis is then required for more advanced patients with disease progression, sometimes up to six or seven days per week due to the sheer severity of the kidney damage.

Doctors now are sometimes trying liver/kidney combined transplants, but the surgery is not without risks and the patient would require long-term immunosuppression as well. In November, a competitor product from Alnylam, Oxlumo, was approved by the FDA and since launched, and I'll discuss this in more detail below. In a way, Oxlumo is a substantial clinical validation for nedosiran itself.

Nedosiran works by blocking production of the lactate dehydrogenase A enzyme by silencing the LDHA gene. This enzyme is required by the liver in the last step of making oxalate, so blocking it should prevent all three forms of hyperoxaluria. Nedosiran, like most RNAi therapies, is a very targeted treatment as it doesn't cause any other measurable changes in other metabolic markers as measured in the blood or urine.

Nedosiran has already received orphan drug designations from both the FDA and EMA, and the therapy has several trial results and ongoing trials at the moment. First was the PHYOX1 Phase 1 trial. This was a single-ascending-dose which ranged from 0.3 mg/kg up to 12.0 mg/kg for 18 total patients with PH1 or PH2. 14 out of those 18 made it to either normal or near normal urinary oxalate levels after a single administration of nedosiran.

The strength of these results led to the enrollment of PHYOX3, an ongoing open-label, multi-dose Phase 3 trial. PHYOX3 reported positive interim data in September 2020 at the American Society of Nephrology Annual Conference.



Figure 6: PHYOX3 Interim Data (source: Dicerna's Corporate Presentation)

As you can see, from the 60-day visit on, literally all patients saw urinary oxalate levels reduced to normal or near normal. This is a stunning improvement in a disease characterized ordinarily by extremely high urinary oxalate levels.

In addition to PHYOX3, Dicerna started two other trials: PHYOX2, a pivotal Phase 2 trial that completed enrollment in January, and PHYOX4, second pivotal trial that began earlier this month. Given the data we've already seen as well as the already approved RNAi therapeutic in this area, I view these trials as very low risk. Dicerna has said to expect an NDA submission in Q3 if data is positive, setting up a mid-2022 approval and launch potentially.

The market is relatively small for this rare disease indication, with only about 16,000 patients in US and Europe. At typical orphan disease pricing though, the total addressable market is north of $1.6 billion at least. Dicerna has said it expects peak sales ranging from $500 million to $1 billion.



Figure 7: Nedosiran Market Estimates (source: Dicerna's Corporate Presentation)

The only real competition for nedosiran is Alnylam's Oxlumo that I mentioned above, but Oxlumo only addresses the genetic mutation that causes PH1, not PH2 or PH3. This leaves a clear opportunity for nedosiran to distinguish itself. Also oddly Alnylam and Dicerna entered into a cross-licensing deal with Oxlumo and nedosiran in early 2020 where Alnylam has to pay Dicerna a mid-to-high single digit royalty on Oxlumo says and Dicerna has to pay Alnylam a low single digit royalty on nedosiran. Synlogic is also developing a therapy for hyperoxaluria, but it is a different type - enteric hyperoxaluria which is caused by intestinal disorders like Crohn's, not primary hyperoxaluria like Dicerna and Alnylam.

Dicerna's other internal program is DCR-A1AT to treat A1AT deficiency-associated liver disease. AATLD is caused by a genetic mutation that leads to production of an abnormal A1AT protein that builds up in the liver. This causes chronic liver disease up to and including cirrhosis, liver cancer, and liver failure, and it can also cause fibrotic effects in the lungs as well. DCR-A1AT works by silencing the defective gene which produces this abnormal protein.

Alynylam and Dicerna combined efforts and candidates in this space with Dicerna taking the lead in development and commercialization. Dicerna has chosen to move DCR-A1AT forward in the clinic with a Phase 2 that should get underway in the first half of this year. We should also see Phase 1 data during that same time period.

The market size is about 10x bigger than PH, with roughly 180k patients in the US and Europe. I haven't seen peak sales numbers for Dicerna's A1AT therapy specifically, but the figure I've seen thrown out for other new A1AT therapies has been $1 billion+ in sales. Alnylam has a zero-cost opt-in option for ex-US development that it can exercise post-Phase 3 results if the company so chooses, and that company choosing to opt-in would likely be a good thing given its ex-US infrastructure.

Despite the deal with Alnylam, Dicerna still has competition in this indication from Arrowhead. That company's candidate is further along, with a Phase 2/3 trial ongoing. As long as Dicerna avoids any huge setbacks though, I can see there being room in this space for both therapies.

Dicerna also has positive preclinical data in tests that extend its RNAi technology into CNS tissue. Continued development in this area will be important for the company's ability to further build-out its pipeline and distinguish itself from Alnylam and Arrowhead as I discussed above.

Dicerna's technology has still yet to generate sales for the company, whether internal or partnered, and it's not a given that it ever will. Investors in Dicerna need to consider this carefully along with the fact that if Dicerna started to see meaningful setbacks with its technology, things could snowball out of control with partnership funding drying up and large amounts of dilutive capital being needed. I don't view this as likely or I wouldn't be interested in investing in the company, but one should not be ignorant of these risks.

Dicerna has a Plethora of Partnered Programs
Dicerna's partnered pipeline is far more extensive than its internal pipeline and likely more potentially lucrative with many potential royalty and milestone payment opportunities likely to come. It's also noteworthy for the large number of deals with big pharma companies.
Arrowhead Pharmaceuticals, Inc. : ARWR ,Acquired all of Roche's RNAi business in 2011, and all of Novartis' RNAi business in 2015, including all related Intellectual property. Targeted RNAi Therapeutics Company. RNAi is a way of silencing target genes, and is highly specific.

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Re: Arrowhead Pharmaceuticals (ARWR)

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vervolg
Figure 8: Dicerna's Partnered Pipeline (source: Dicerna's Corporate Presentation)

The Boehringer Ingelheim deal happened first chronologically. This deal now features two targets with a potential NASH indication being the first. BI paid $10 million upfront and has agreed to pay an additional $191 million in potential milestones for NASH and up to $170 million in potential milestones for the second target. Dicerna will also stand to receive royalties that are high-single digit to low-double digit on the NASH product and tiered single-digit on the second target.

Alexion, which was a midsize biotech but has now been acquired by AstraZeneca, struck a deal to target rare diseases. Alexion paid $22 million upfront and made a $15 million equity investment in a deal that also features $105 million in developmental milestones per product plus up to $160 million in sales-related milestones. Dicerna's potential royalties on any of these products that get approved are mid-single to low-double digit, and the partnership encompasses four programs currently.

The Eli Lilly deal was next and features cardiometabolic, neurodegeneration, and pain-related targets. Lilly paid $100 million upfront and made a $100 million equity investment. The deal contemplates in excess of 10 targets and Dicerna stands to receive $350 million in milestone payments per target plus $5 million each time a target reaches proof of concept in animal models. Mid-single digit to low-double digit royalties.

Roche paid $200 million upfront for its deal for an HBV therapy as well as potential future milestones up to $1.75 billion and mid-teens royalties. One change with this deal versus the ones that came before is that Dicerna has an opt-in that would take the royalties to high-twenties to mid-thirties in exchange for co-funding.

The last deal with Novo Nordisk also carries such an opt-in clause but this time going both ways. This deal targets liver-related cardiometabolic diseases, like NASH, type 2 diabetes, and obesity, and Dicerna stands to receive mid-single digits to mid-teens royalties. Novo paid $175 million upfront, made a $50 million equity investment, and promised $25 million per year each year for the first three years covered by the deal. Dicerna has an opt-in on Novo's programs, and Novo gets an opt-in on two undisclosed rare liver disease programs that fall under this deal. Dicerna will still receive $25 million at the end of next year and has already reported having received $25 million from Novo in Q4 2020. Dicerna also announced that Novo has nominated its first drug candidate under this agreement, triggering an additional $2.5 million milestone payment.



Figure 9: Dicerna's Partnerships (source: Dicerna's Corporate Presentation)

Roche's HBV therapy is furthest along although Lilly also has one therapy in the clinic now for an undisclosed cardiometabolic indication. It will likely be middle of the decade before Dicerna starts recognizing a lot of these milestone payments and certainly before royalties start flowing in, but these can be substantial.

The milestone payments could be substantial. By my count, Dicerna stands to receive up to $6.031 billion in future milestones on just its already announced partnered programs. If the company is able to realize just a quarter of that total, it would amount to in excess of $1.5 billion in additional non-dilutive capital over the next decade. Royalties could have a similarly large impact. With the average royalty on these deals looking to be high single digits, say 8%, and the potentially lucrative indications that Dicerna's partners are targeting, it's easy to see combined sales of these 20+ programs in the multiple billions and royalties then well into the hundreds of million flowing straight to Dicerna's bottom-line.

Dicerna's Balance Sheet Shows Substantial Strength
Dicerna has a strong balance sheet at the moment, with the company reporting that it had $609.9 million in cash and equivalents at the end of Q3. The company also said in its Q3 update that it anticipated receiving about $100 million more in cash in Q4 2020 and 2021 from collaborators. That's a large amount of cash that will go a long way towards funding continued development of Dicerna's pipeline.

Q3 revenue also came in at $48.9 million and was all due to the company's various collaborations. Dicerna reported that net loss for Q3 was just $21.8 million, which looks really low because of the collaboration revenue that came in. The company's expenses were a much higher $71.8 million in Q3, so the burn rate would be nearly $300 million per year if Dicerna was entirely pre-revenue like most biopharma companies at its stage in development which shows the huge benefit of the company's partnerships.

Dicerna has issued guidance that current funding and anticipated milestone payments should fund operations into 2023 which to me looks like it factors in a burn rate somewhere in between what we saw in Q3 and that maximum potential amount. Getting into 2023 importantly would see Dicerna through a potential nedosiran approval and launch and hopefully also the expansion of the pipeline into several internal and partnered CNS disease indications.

One of the funds I closely follow, Perceptive Advisors, owns a position in Dicerna with an average cost basis of $21.70, and insiders own a decent amount of shares with the CEO and COO owning 37k and 28k shares respectively. With the vote of confidence from all the big pharma partnerships described above, I don't care as much about institutional and insider ownership in a company like Dicerna, but these still also suggest that Dicerna is more likely to still offer value than be a value trap.

Dicerna's Valuation Looks Very Reasonable Especially From a Comparative Perspective
As with Regenxbio, I first considered how Dicerna looked compared to its two major peers in the RNAi space. To me, Dicerna looks compelling from such a comparative standpoint.




Figure 10: Market Cap Comparison for Leading RNAi Companies (source: barchart.com)

As you can see from Figure 10, Dicerna trades for less than a quarter of its closest peer in terms of market cap, Arrowhead, and for about 10% of Alnylam's market cap. To be clear, both of these companies have pipelines that are either more advanced or already targeting a more broad range of indications, but not so much as to justify this extreme of a gap.

Analysts also seem to have a high degree of confidence that Dicerna's revenue will grow substantially in the latter half of this decade. For a company with a market cap around $2 billion, revenues over $1 billion (and as high as $2 billion in one analysts view) certainly suggest there is strong upside potential. By contrast, the industry average ratio of price to sales for a mature company in this sector is often considered to be around a four or a five.



Figure 11: Dicerna Analyst Revenue Estimates (source: Seeking Alpha)

I think Dicerna is a high-quality company that fits well with the other companies I follow. One important element of de-risking is having a strong technology platform with broad applicability, and Dicerna has that in spades. Dicerna's internal and partnered pipelines both look to contribute substantially to cash flow by mid-decade.



Figure 12: Dicerna's Upcoming Catalysts (source: Dicerna's Corporate Presentation)

Dicerna's management has also done a good job of managing cash-flow, particularly by using partnership cash to minimize the cost of developing the company's extensive pipeline. Finally, Dicerna looks undervalued, especially in comparison with its peers in the RNAi space, which suggests it could see meaningful upside in the coming years if success continues.
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Arrowhead Pharmaceuticals, Inc. : ARWR ,Acquired all of Roche's RNAi business in 2011, and all of Novartis' RNAi business in 2015, including all related Intellectual property. Targeted RNAi Therapeutics Company. RNAi is a way of silencing target genes, and is highly specific.

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Re: Arrowhead Pharmaceuticals (ARWR)

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Van YForum

Kunnen we dit jaar een BTD voor AAT bereiken?

Met het oog op de aanduiding Doorbraaktherapie verwijst klinisch significant eindpunt in het algemeen naar een eindpunt dat een effect meet op onomkeerbare morbiditeit of mortaliteit (IMM) of op symptomen die ernstige gevolgen van de ziekte vertegenwoordigen. Een klinisch significant eindpunt kan ook verwijzen naar bevindingen die een effect op IMM of ernstige symptomen suggereren, waaronder:
• Een effect op een vastgesteld surrogaat-eindpunt
• Een effect op een surrogaat-eindpunt of tussenliggend klinisch eindpunt waarvan wordt aangenomen dat het redelijkerwijs waarschijnlijk is dat het een klinisch voordeel voorspelt (d.w.z. de versnelde goedkeuringsnorm)
• Een effect op een farmacodynamische biomarker (s) die niet voldoen aan de criteria voor een aanvaardbaar surrogaat eindpunt, maar sterk suggereert dat er een potentieel klinisch betekenisvol effect is op de onderliggende ziekte
• Een significant verbeterd veiligheidsprofiel vergeleken met de beschikbare therapie (bijv. Minder dosisbeperkende toxiciteit voor een oncologisch middel), met bewijs van vergelijkbare werkzaamheid
Een geneesmiddel dat de aanduiding Breakthrough Therapy krijgt, komt in aanmerking voor het volgende:
• Alle Fast Track-aanduidingsfuncties
• Intensieve begeleiding voor een efficiënt geneesmiddelenontwikkelingsprogramma, dat al in fase 1 begint
• Organisatorische betrokkenheid met senior managers
CA Q1 2021 opmerkingen: Deze gegevens waren afkomstig van biopsieën na slechts 6 maanden behandeling. We verwachten ook biopsiegegevens over 12 maanden in de komende maand of 2 te hebben. We zijn van plan die gegevens te bespreken met de FDA en enkele wijzigingen in de onderzoeksopzet en eindpunten voor te stellen, met als doel de tijd tot een mogelijke NDA te verkorten.

antwoorden :
-Biopsieresultaten van 12 maanden komen binnen de komende 8 weken IMO en verdere verbeteringen zouden kunnen leiden tot BTD in 3Q (juli?) Wanneer FDA-gesprekken plaatsvinden volgens het UBS-rapport.

-Natuurlijk kan een belangrijke bepalende factor voor de Takeda-deal zijn gebaseerd op uw BTD-mogelijkheid. Ze streven zeker naar die FDA-aanduiding

Credits to J.
Could we achieve a BTD for AAT this year?
For purposes of Breakthrough Therapy designation, clinically significant endpoint generally refers to an endpoint that measures an effect on irreversible morbidity or mortality (IMM) or on symptoms that represent serious consequences of the disease. A clinically significant endpoint can also refer to findings that suggest an effect on IMM or serious symptoms, including:
• An effect on an established surrogate endpoint
• An effect on a surrogate endpoint or intermediate clinical endpoint considered reasonably likely to predict a clinical benefit (i.e., the accelerated approval standard)
• An effect on a pharmacodynamic biomarker(s) that does not meet criteria for an acceptable surrogate endpoint, but strongly suggests the potential for a clinically meaningful effect on the underlying disease
• A significantly improved safety profile compared to available therapy (e.g., less dose-limiting toxicity for an oncology agent), with evidence of similar efficacy
A drug that receives Breakthrough Therapy designation is eligible for the following:
• All Fast Track designation features
• Intensive guidance on an efficient drug development program, beginning as early as Phase 1
• Organizational commitment involving senior managers
CA Q1 2021 comments: These data were from biopsies after only 6 months of treatment. We also expect to have 12-month biopsy data over the next month or 2. We plan on discussing those data with the FDA and proposing some changes to the study design and endpoints, with a goal of shortening the time to a potential NDA.
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Arrowhead Pharmaceuticals, Inc. : ARWR ,Acquired all of Roche's RNAi business in 2011, and all of Novartis' RNAi business in 2015, including all related Intellectual property. Targeted RNAi Therapeutics Company. RNAi is a way of silencing target genes, and is highly specific.

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Arrowhead Pharmaceuticals, Inc. : ARWR ,Acquired all of Roche's RNAi business in 2011, and all of Novartis' RNAi business in 2015, including all related Intellectual property. Targeted RNAi Therapeutics Company. RNAi is a way of silencing target genes, and is highly specific.

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Re: Arrowhead Pharmaceuticals (ARWR)

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Erg knap animatiefilmpje van onze collega's



https://www.youtube.com/watch?v=lIGPNBs ... e=emb_logo


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meer op YT : https://www.youtube.com/watch?v=cK-OGB1_ELE
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Arrowhead Pharmaceuticals, Inc. : ARWR ,Acquired all of Roche's RNAi business in 2011, and all of Novartis' RNAi business in 2015, including all related Intellectual property. Targeted RNAi Therapeutics Company. RNAi is a way of silencing target genes, and is highly specific.

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Re: Arrowhead Pharmaceuticals (ARWR)

Bericht door piddybull »

https://www.streetinsider.com/Analyst+C ... 86711.html

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Arrowhead Pharmaceuticals, Inc. : ARWR ,Acquired all of Roche's RNAi business in 2011, and all of Novartis' RNAi business in 2015, including all related Intellectual property. Targeted RNAi Therapeutics Company. RNAi is a way of silencing target genes, and is highly specific.

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piddybull
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Re: Arrowhead Pharmaceuticals (ARWR)

Bericht door piddybull »

WEEK 7 CHART 2

Week + 1,49 %
YTD + 16,66 %
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Arrowhead Pharmaceuticals, Inc. : ARWR ,Acquired all of Roche's RNAi business in 2011, and all of Novartis' RNAi business in 2015, including all related Intellectual property. Targeted RNAi Therapeutics Company. RNAi is a way of silencing target genes, and is highly specific.


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