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Aandeel Arrowhead Pharmaceuticals OTC:ARWR, US04280A1007

  • 60,390 24 nov 2020 22:00
  • -7,810 (-11,45%) Dagrange 58,020 - 66,750
  • 3.536.392 Gem. (3M) 1,6M

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  1. forum rang 4 Hulskof 22 november 2020 00:26
    CEO over Merck/Peloton: "Mrck data is good and it further validates the hif2a target.
    We don’t have to be better than that drug to have a drug….physicians like options. I think if we can provide options then I think we have another drug on our hands. Ours is a targeted drug. The mrck peloton drug is a small molecule that goes everywhere. This rnai drug is designed to go tumors and so we may get less anemia…I don’t know if we’re dependent upon that but I believe it is a possibility. I’m hopeful we get good knockdown, and like enac data, if we get good data here, there are two things we can read on that:

    A). I think it suggests we have a drug
    B). We have a platform

    Our targeting strategy is designed to work for a variety of solid tumors, we may have a platform that can apply to various solid tumors, various gene targets and we think that is a very large opportunity we will have data we can share in the first half of next year."

    Bereidt hij ons hier voor op minder effectieve data dan Merck te bieden heeft? Of wordt het zoals altijd: underpromise and overdeliver?

    Groot vraagteken.
  2. mvdln 22 november 2020 21:05
    quote:

    Hulskof schreef op 22 november 2020 00:26:


    CEO over Merck/Peloton: "Mrck data is good and it further validates the hif2a target.
    We don’t have to be better than that drug to have a drug….physicians like options. I think if we can provide options then I think we have another drug on our hands. Ours is a targeted drug. The mrck peloton drug is a small molecule that goes everywhere. This rnai drug is designed to go tumors and so we may get less anemia…I don’t know if we’re dependent upon that but I believe it is a possibility. I’m hopeful we get good knockdown, and like enac data, if we get good data here, there are two things we can read on that:

    A). I think it suggests we have a drug
    B). We have a platform

    Our targeting strategy is designed to work for a variety of solid tumors, we may have a platform that can apply to various solid tumors, various gene targets and we think that is a very large opportunity we will have data we can share in the first half of next year."

    Bereidt hij ons hier voor op minder effectieve data dan Merck te bieden heeft? Of wordt het zoals altijd: underpromise and overdeliver?

    Groot vraagteken.

    Juist! CA heeft ervaring. Over ARO-AAT bijvoorbeeld:


    Q3/2020 earnings 05/08/2020: We will also be looking at whether the accumulated Z-AAT polymer can start to decrease after six months, which we are not expecting but would be pleasantly surprised to see. In addition, we will look at changes in inflammation and in various histologic parameters. Again, we wouldn't expect to see these measures change after only six months of treatment, but it would be a very exciting result if we were to see improvements this quickly.

    16/09/2020 interim data: Three of four patients demonstrated reductions in intra-hepatic Z-AAT polymer, with a maximum reduction of 97%.
  3. Missolapola 24 november 2020 08:05
    Arrowhead Pharmaceuticals, Inc. (ARWR) CEO Christopher Anzalone on Q4 2020 Results - Earnings Call Transcript

    Nov. 23, 2020 11:30 PM ETArrowhead Pharmaceuticals, Inc. (ARWR)
    Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR) Q4 2020 Earnings Conference Call November 23, 2020 4:30 PM ET

    Company Participants

    Vincent Anzalone - Vice President of Investor Relations

    Christopher Anzalone - President and Chief Executive Officer

    Javier San Martin - Chief Medical Officer

    Ken Myszkowski - Chief Financial Officer

    James Hassard - Chief Commercial Officer

    James Hamilton - Senior Vice President and Head of Discovery and Translational Medicine

    Conference Call Participants

    Shawn Egan - Citigroup

    Maury Raycroft - Jefferies

    Alethia Young - Cantor Fitzgerald

    Salveen Richter - Golden Sachs

    Ted Tenthoff - Piper Sandler

    Mani Foroohar - SVB Leerink

    Luca Issi - RBC Capital

    Madhu Kumar - Baird

    Mayank Mamtani - B. Riley

    Patrick Trucchio - H.C. Wainwright

    Operator

    Ladies and gentlemen, welcome to the Arrowhead Pharmaceuticals Conference Call. Throughout today's recorded presentation, all participants will be in a listen-only mode. After the presentation, there will be an opportunity to ask questions.

    I will now hand the conference call over to Vincent Anzalone, Vice President of Investor Relations for Arrowhead. Please go ahead, Vince.

    Vincent Anzalone

    Thank you, Chris. Good afternoon, everyone and thank you for joining us today to discuss Arrowhead's results for its Fiscal year ended September 30, 2020.

    With us today from management are President and CEO, Dr. Christopher Anzalone, who will provide an overview of the quarter; Dr. Javier San Martin, Chief Medical Officer, who will discuss our clinical programs; and Ken Myszkowski, our Chief Financial Officer, who will give a review of the financials. In addition, James Hassard, our Chief Commercial Officer, and Dr. James Hamilton, who is recently promoted to Senior Vice President and Head of Discovery and Translational Medicine will both be available during the Q&A session of today’s call.

    Before we begin, I would like to remind you that comments made during today’s call contain certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than statements of historical facts, including without limitation those with respect to Arrowhead's goals, plans, and strategies are forward-looking statements.

    These include statements regarding our expectations around the development, safety and efficacy of our drug candidates, projected cash runway, and expected future development and commercialization activities. These statements represent management's current expectations and are inherently uncertain, thus, actual results may differ materially. Arrowhead disclaims any intent and undertakes no duty to update any of the forward-looking statements discussed on today's call.

    You should refer to the discussions under Risk Factors in Arrowhead's annual report on Form 10-K and the company’s subsequent quarterly reports on Form 10-Q for additional matters to be considered in this regard, including risks and other considerations that could cause actual results to vary from the presently expected results expressed in today’s call.
  4. Missolapola 24 november 2020 08:12
    With that said, I'd like to turn the call over to Chris Anzalone, President and CEO of the company. Chris?

    Christopher Anzalone

    Thanks, Vince. Good afternoon everyone, and thank you for joining us today. This is our final earnings call in 2020. So in addition to discussing our progress for the quarter and plans for 2021, I would also like to speak a bit more broadly about our philosophy and model.

    We are building a different type of biotech company. We are not focused on a single therapeutic area or rather on any disease with unmet medical need that is addressable with our technology. We are not focused solely on rare diseases or rather address large and small populations.

    We do not rely solely on partners for late-stage clinical development and commercialization or rather we use partnering strategically and judiciously to enable us to build substantial value by commercializing our own drugs.

    We are fast, probably the fastest in the business from idea to the clinic and we intend to remain fast as we grow. That devotion to speed also applies to pipeline expansion. I believe we have a fastest growing pipeline in our field and we do not intend to slow that input just because we are entering later-stage clinical studies.

    We are not in the me-too product business where we only provide incremental benefit to patients rather we seek to be pioneers. We believe that everything in our clinical pipeline represents the first RNAi to each target in humans. We don’t operate like a normal pharmaceutical company. We are not burdened by analyst gating committees or rather empower our people to make decisions.

    Operationally, think of us as a start-up in $7 billion market cap clothing and we intend to continue in this nimble and creative fashion even as we become a substantially larger and more valuable company. We see this as the most effective way to build a business and most importantly to serve our patients. Every day that we can shave off the development process puts our patients one day closer to a new treatment they need. This is a powerful motivator indeed for us, because of the value of our work depends on the number of lives we touch.

    I mention all of this now because we are at a moment of transition for our company. We have created a lot of value to this point by building what we believe will soon be the largest RNAi-based clinical pipeline in biopharma. As we move into later-stage clinical studies, our focus needs to expand to include commercial planning. Ultimately, this is the reason we are in this business and we need to do that well.

    However, we also need to continue to do the things in discovery and early development that have made us successful while we build our commercial presence. On average, we expect to continue to introduce three new drug candidates into the clinical studies every year and we expect to be able to address a new cell type every 18 to 24 months.

    Think of the potential value embedded in those statements. I expect that we will have ten clinical programs by summer spanning four different cell types that could grow to 20 clinical programs spanning five or six cell types just four years from now. We expect this to drive substantial value, because we expect some of those to become products that we will commercialize ourselves and some can be partnered to fund developments and commercial endeavors.

    Our ability to rapidly grow our pipeline enables this hybrid model of establishing a limited number of partnerships in order to fund wholly-owned programs. We see this as a powerful model because it allows the rapid value creation associated with commercializing wholly-owned drugs while financing this expensive endeavor largely through non-dilutive capital from partnering programs.

    So, even as we approach and ultimately expand our commercial presence, our pipeline strategy should continue to be an important part of our value proposition. This is not only due to the group force of the large numbers of potential drugs in our clinical studies, but should also reflect our expectations of success. There are two components to this.

    First we seek to choose only well-validated targets. These are gene targets where consensus of experts agree the reducing expression will likely have positive therapeutic benefits. By focusing on this, we believe we enter clinical studies with reduced biology and target risk relative to other candidates.

    Second, the RNAi mechanism and experience with the TRiM Platform can provide this additional wind at our back as we continue to treat more patients with drug candidates built on the TRiM Platform and see consistent activity and up to the safety profile, our confidence increases that other candidates targeting different genes will also be successful.

    RNAi doesn’t care what gene is being silenced once we validate our ability to reduce expression of a given gene in a given cell type, we have confidence that we can replicate that in other gene targets. We believe this is a powerful and scalable concept that gives us confidence that a larger percentage of our candidates entering the clinic will ultimately become drugs compared to traditional small molecules.

    We hope that as we approach and become a commercial company, the market will properly value our growing pipeline. As I mentioned, it will continue to be an important part of our value proposition and we expect it to remain a substantial differentiator versus our competitors.

    With that, let’s move into an overview of the last quarter. During the last few months, our accomplishments included the following: one, we hosted a KOL seminar on ARO-ENaC, our first lung targeted candidate to treat cystic fibrosis and we initiated dosing in a Phase 1/2 clinical study.

    Two, we are into a $20 million milestone payment from Amgen following the start of a Phase 2 study of AMG 890, now called Olpasiran, which is a partnered program targeting Lp(a) to tread cardiovascular disease.

    Three, we initiated a Phase 1b study for ARO-HIF2, our first tumor targeted candidate to treat renal cell carcinoma. Four, we presented new data on Phase 1/2 studies of both of our own wholly-owned cardio metabolic candidates ARO-APOC3 and ARO-ANG3, and multiple medical needs including the European Society of Cardiology and the American Heart Association meetings and subsequently hosted KOL webinars to discuss the data and our plans for their future developments.

    Five, we presented Phase 2 data at AASLD on ARO-AAT, our candidate against liver disease associated with alpha-1 antitrypsin deficiency showing that ARO-AAT strongly reduces the production of mutant Z-AAT protein and led to improvements in multiple biomarkers of alpha-1 liver disease.

    And six, we signed an agreement with Takeda to co-develop and co-commercialize ARO-AAT, which includes $300 million upfront, $740 million in milestone payments, a 50{50 profit sharing agreement in U.S. and 20% to 25% royalties on sales outside the U.S. This is lot of progress in a short period of time and even more impressive with a backdrop of disruptions caused by COVID-19.

    Let’s take a look at the Takeda deal. It’s a good example of our selective partnering strategy. We expect ARO-AAT to benefit from Takeda’s global footprint, existing infrastructure, expertise and 18 year history in the AAT market to enable a rapid launch. If approved, ARO-AAT will join Takeda’s global commercially available products including Glassia, Aralast, Entyvio and their growing GI pipeline.
  5. Missolapola 24 november 2020 08:15
    Takeda has clearly invested and committed to these areas and has a proven track record of success. The deal is also important in terms of capital. In addition to the $300 million upfront, we have potential access to substantial capital in the near, mid, and long term with a possible stream of milestone payments, profit sharing and royalties. When added to the potential milestone payments and royalties from our partnerships with Amgen and Janssen, we feel our balance sheet is in very strong position.

    This allows us to confidently move our wholly-owned programs into later-stage development and ultimately commercialization. This deal is also an ongoing process toward rationalizing our growing pipeline where we look to build commercial infrastructure in areas where we expect multiple drugs such as cardio metabolic, and pulmonary. We will look for synergy and leverage when deciding where to focus commercial build outs.

    So let’s take a look at the cardio metabolic pipeline. We presented data recently at AAJ and also held two KOL webinars to discuss these programs. Javier will discuss these specifically in a moment, but I want to talk about where we are with this program at a high level. The data across single and multiple-doses and in healthy volunteers as well as various patient populations has been very strong and highly consistent.

    In addition, ARO-APOC3 and ARO-ANG3 are showing unique profiles. What I mean by that is that we believe each drug may ultimately give cardiologists more tools to tailor their treatments to the specific lipid profiles to their patients. We also think they will fill holes in the current treatment paradigm and may potentially address lipid targets that had not been adequately addressed.

    For instance, there are more than 4 million patients in the U.S. with severe hypertriglyceridemia and given published data, we would expect the overwhelming majority of them would not reach normal triglyceride levels with currently available treatments. There are also approximately 30 million to 40 million addressable patients in the U.S. with mixed dyslipidemia, which has elevated hypertriglyceridemia and elevated LDL cholesterol.

    We have become increasingly confident that these programs – we have become increasingly confident in these programs and in our ability to move them to commercialization. We’ve also been able to move forward in our other clinical programs. ARO-HSD, our drug candidate against NASH and alcoholic hepatitis is now in the patient portion of the Phase 1/2 study.

    ARO-HIF2 our candidate against renal cell carcinoma and ARO-ENaC our candidate against cystic fibrosis are both being dosed to patients as well. Progress in ARO-HIF2 and ARO-ENaC is particularly important, because it has been our goal to gain clinical proof-of-concept and then move into a rapid pipeline expansion phase for tumor and pulmonary tissue types.
  6. Missolapola 24 november 2020 08:16
    We think we are just in the cusp of that phase now. To that end, we continue to work in parallel on multiple additional targets in tumor and pulmonary and also to expand our reach into new tissue types beyond these.

    Before I move on to 2021, I’d like to say a few words about the novel coronavirus. As with the rest of the world, we’re excited to see the interim results from some of the COVID-19 vaccines science. Multiple phase and effective vaccines will be a humanitarian triumph and we applaud the impressive work done by several companies.

    From an Arrowhead standpoint, some may ask that progress on the vaccines affects our internal program. The answer is, not really. We continue to make progress on an anti-viral approach that is designed work across different coronaviruses. The history of SARS, MERS and now the current coronavirus suggests that the world should expect some type of coronavirus outbreak approximately every seven years.

    As such, we are studying conserved regions in known coronaviruses with a goal of creating an inhalable antiviral that could be applied to future outbreaks, as well as the current virus should there be blind spots with the vaccines. We are still in early animal studies but I hope that we will have an idea about the feasibility of this approach in 2021.

    Moving to the future, there is lot you should expect from us during the final month of the year and into 2021. Our expectations include the following: one, we are on track to file a CTA for ARO-LUNG2 at the end of this year. This second program – this is a second program in our pulmonary franchise and is designed to treat COPD by pivoting an undisclosed target in pulmonary epithelia.

    Two, we are on pace to potentially have preliminary data readouts by the middle of 2021 for ARO-HSD, ARO-HIF2 and ARO-ENaC. Three, during the first half of 2021, we also intend to engage with the FDA and other regulators to discuss pivotal trial study design and endpoints for ARO-AAT.

    Based on the impressive data that came out of our 2002 open-label study, it appears that patients have large reductions in Z-AAT monomer, which we expected but also had improvements in other downstream markers such as polymer, globules, LFTs and others. These discussions may allow us to find a more streamlined and accelerated path to a potential approval.
  7. Missolapola 24 november 2020 08:17
    But there also maybe additional open-label data in 2021 for patients with 12 month and 18 month repeat biopsies.

    Four, we also intend to initiate multiple studies in first half of 2021 for both of our cardio metabolic programs. For ARO-ANG3 mixed dyslipidemia patients we are working on a Phase 2b dose-finding study.

    For ARO-APOC3, we are working to start three studies, a Phase 2b study – a Phase 2b dose-finding study in patients with triglycerides ranging from 150 to 499, a Phase 2b dose-finding study of patients with triglycerides over 500 and a Phase 3 study in patients with familial chylomicronemia syndrome or FCS.

    Five, in the second half of 2021, we intend to file a CTA for our first muscle targeted RNAi therapeutic. That program has moved forward very nicely and we are eager to talk more about that and what the data look like.

    Six, for our partnered program with Amgen and Janssen we can’t provide specific guidance on timing, but we continue to be pleased with their progress and look forward to additional future progress. Seven, lastly, we are working on several other undisclosed programs and we’ll likely have another CTA filed for another program in 2021.

    With that overview, I’d now like to turn the call over to Dr. Javier San Martin. Javier?

    Javier San Martin

    Thank you, Chris, and good afternoon to everybody on the call. I want to highlight data from the ARO-AAT, ARO-APOC3 and ARO-ANG3 programs since which are important readouts for each during the last quarter. Before I do that, I will give a very quick review of the earlier stage clinical program.

    ARO-HSD is our investigational candidate for the potential treatment of alcohol and non-alcohol related liver disease. The genetic validation is strong for inhibiting the target HSD17B13 in NASH cirrhosis, alcoholic hepatitis and cirrhosis patients.

    While conducting a Phase 1/2 single and multiple dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of ARO-HSD in normal healthy volunteers, as well as in patients with NASH or suspected NASH, we have complete single dose escalation in healthy volunteers and are currently enrolling the multiple dose patient portion of this study in NASH or suspected NASH patients. This study includes liver biopsy plus physical activity.

    ARO-HIF2 is designed to clear cell, renal cell carcinoma, and we are currently dosing patients in the Phase 1b dose-finding clinical study in up to 18 patients with advanced renal cell carcinoma. This study is designed to evaluate the safety of ARO-HIF2 and to determine the recommended Phase 2 dose by also assessing pharmacokinetics and preliminary efficacy based on RECIST and post-dose tumoral expression of HIF2 alpha and HIF associated genes.

    Our last early-stage clinical program is ARO-ENaC designed to treat cystic fibrosis. ARO-ENaC is in a Phase 1/2 dose escalated study to evaluate the safety, tolerability and pharmacokinetic effect of ARO-ENaC in up to 224 normal healthy volunteers and to evaluate safety, tolerability and efficacy in up to 30 patients with cystic fibrosis.

    We have dose escalated multiple times as planned in healthy volunteers and so far we are pleased with the safety and tolerability of this study. This is always an important finding for a new investigational drug and even more so for a new platform. We are now enrolling cystic fibrosis patients in the multiple dose portion of the study.
  8. Missolapola 24 november 2020 08:17
    Now I will move on to the recent data readouts. For ARO-AAT, our investigational RNAi therapeutic being developed as a treatment for the rare genetic liver disease associated with alpha-1 antitrypsin deficiency, we presented data at AASLD on 2002 open-label study. To review this study is fully enrolled with 15 participants in three cohorts.

    Four patients in cohort one received 200 mg of ARO-AAT and will have a repeat biopsy after six and 18 months of treatment. Cohort 1b is the same but patients received a 100 mg dose and eight patients in cohort 2 received 200 mg of ARO-AAT and will have repeat biopsy after 12 and 24 months of treatment.

    At AASLD, we reported on six months results from cohort 1. We think that data strongly suggests that ARO-AAT is doing what is designed to do, which is reduce the production of the misfolded mutant Z-AAT protein. The result also indicate that the liver may have the ability to clear out accumulated Z-AAT and begin to heal itself faster than anticipated.

    Importantly, we saw the following: 86% to 93% of reduction in terms AAT protein, all patients demonstrated greater than 80% reduction in liver Z-AAT monomers, three of four patients had decrease in liver globule involvement and three of four patients demonstrated reduction in Z-AAT polymers with a range of 68% to 97%. All patients showed AAT reduction ranging from 36% to 66%.

    So we think that these are all positive indications of a strong pharmacodynamic response and improvement in liver health following just three doses of ARO-AAT.

    As Chris mentioned, we are currently preparing to engage with the FDA and other regulatory agencies in the first half of 2021 to discuss areas where the ARO-AAT program may potentially be streamlined and accelerated.

    Let’s now move to our cardio metabolic program, ARO-APOC3 and ARO-ANG3. We held two KOL webinars last week to discuss the data and the progress in some days. We were fortunate to have Dr. Christie Ballantyne from Baylor College of Medicine and Dr. Ira Goldberg from the NYU School of Medicine showing us and provided a perspective on valuable insights. Replays of these events are available on the Investor section of our website for those who missed the live presentation.

    Let’s talk about ARO-APOC3, our candidate our candidate targeting apolipoprotein C3, being developed as a potential treatment for patients with hypertriglyceridemia. The current clinical study is in Phase 1/2 single and multiple dose study to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic effects of ARO-APOC3.

    It is a single dose and multiple dose portion of the study in adult healthy volunteers and a multi-dose proportional patients with hypertriglyceridemia including at cohort and growing that 220 Chylomicronemia patients.

    The America Heart Association will report this data from the multiple dose patient portion of this study. The results were very interested and highly encouraging to us as we prepare to be in the next stage of evaluation for the program in the first half of next year.
  9. Missolapola 24 november 2020 08:18
    In patients with hypertriglyceridemia, ARO-APOC3 treatment resulted in robust, and sustained reduction in triglycerides and non-HDL cholesterol with increase in HDL cholesterol. Specifically, we have said maximum reduction of 80% to 99% in APOC3, maximum in reduction of 74% to 92% in TG or triglyceride and 39% to 62% reduction in non-HDL cholesterol and maximum in increase of 95% to 115% in HDL cholesterol.

    In patients with Chylomicronemia, 50 mg ARO-APOC3 achieved similar levels of reduction of APOC3 and changes in key related parameters, also maximum in reduction of 98% in APOC3, maximum in reduction of 88% in TG and 59% in non-HDL cholesterol and a maximal increase of 120% in HDL cholesterol.

    Importantly, the effect of ARO-APOC3 will maintain for greater than plus weeks post regardless of the patient population. We think this indicates that once locally or less frequent dosing might be possible.

    With other wholly-owned cardio metabolic candidates it’s ARO-ANG3 targeting angiopoietin like protein 3, or ANGPTL3, and is being developed as a potential treatment for patients with mixed dyslipidemia. The current clinical study is a Phase 1/2 single and multiple dose study to evaluate safety, tolerability, pharmacokinetic, and pharmacodynamic effects.

    At the American Heart Association, and the recent webinar, we also presented data on the multiple dose patient portion of the ARO-ANG3 study. As with ARO-APOC3, we were thrilled with the clinical results and move forward with the program with a lot of confidence in its potential to help patients with mixed dyslipidemia. The data showed that in heterozygous familial hypercholesterolemia and patients with non-FH and non-FH patients ARO-ANG3 resulted in mean reduction of 78% to 90% of ANGPTL3, reduction of 29% to 47% in targeted cells, 29% to 75% in LDL cholesterol and a reduction of 71% to 75% on non-HDL cholesterol.

    In high triglyceride patients, ARO-ANG3 resulted in mean reduction of 83% for ANGPTL3, 75% for triglyceride and 56% for non-HDL cholesterol. As with APOC3, the effect of ARO-ANG3 was maintained for greater than 12 weeks both post dose regardless of patient population. We believe this indicates that once quarterly or less frequent dosing might be possible.

    These results provide further support that the RNAi mechanism in general and more specifically developed within our three platforms tend to perform very consistently regardless of the gene type. So far we have experienced very good translation of preclinical data to human clinical data with respect to safety, tolerability and activity.

    This gives us great confidence in each new program we develop even at very early stages. The next steps are to study in – and notable clinical trials with the inhibition of respective gene targets lead to the desired clinical benefit in a specific patient population.

    As Chris mentioned earlier, we try to select target at the discovery stage with generally well understood biology and strong support from human genetic studies. This provides us with even more confidence that while reducing risk to this possible and maximizing our probability of success.
  10. Missolapola 24 november 2020 08:19
    I will now turn the call over to Ken Myszkowski, Arrowhead’s Chief Financial Officer. Ken?

    Ken Myszkowski

    Thank you, Javier, and good afternoon, everyone. As we reported today, our net loss for fiscal 2020 was $84.6 million or $0.84 per share based on 100.7 million weighted average diluted shares outstanding. This compares with net income of $68 million or $0.69 per share based on $98.6 million weighted average diluted shares outstanding for fiscal 2019.

    Revenue for fiscal 2020 was $88 million, compared to $168.8 million for fiscal 2019. Revenue in both periods primarily relates to the recognition of a portion of the upfront payments and milestones from our license and collaboration agreements with Janssen.

    Revenue from the Janssen agreement is being recognized based on our estimate of the proportion of effort expended toward fulfilling our performance obligations, primarily, overseeing the completion of the Phase 1/2 HBV clinical trial. We expect the remaining $19 million of deferred revenue to be recognized in the first half of fiscal 2021.Any additional milestones achieved with Janssen or Amgen will be additive to this projection.

    In addition, current period revenue also included the $20 million milestone payment we received from Amgen on the initiation of their Phase 2 clinical trial for AMG 890, which is now referred to as Olpasiran.

    Total operating expenses for the year ended September 30, 2020 were $181.1 million, compared to $107.6 million for the year ended September 30, 2019. This increase is primarily due to increased non-cash stock compensation expense. Stock compensation expense has increased because the valuation of new stock option and restricted stock award grants has increased with the growth of our stock price.

    Additionally, stock compensation expense increased due to the timing of achievement of certain performance-based awards in each period. The increase in total operating expenses was also driven by increased clinical trial costs as our pipeline of clinical candidates has increased and increased personnel costs in both R&D and G&A as our headcount continues to grow.

    Net cash used by operating activities during in fiscal 2020 was $95.4 million, compared with net cash provided by operating activities of $173 million in fiscal 2019. The operating cash generated in fiscal 2019 reflects the $175 million upfront payments and two $25 million milestone payments received from Janssen offset by cash used for operations.

    Turning to our balance sheet. Our cash and investments balances totaled $453 million at September 30, 2020, compared to $302.9 million at September 30, 2019. The increase in our cash and investments was primarily due to the December 2019 equity financing we completed, which generated $250.5 million in net cash proceeds to the company.

    In addition to the cash and investments assets discussed, as of September 30, 2020, we also anticipate receiving $300 million from the upfront payment from Takeda by the end of this calendar year or shortly thereafter.

    Similar to our agreement with Janssen, we anticipate recognizing this upfront payment as revenue over the course of completing our performance obligations within the deal, which primarily consists of managing the ongoing ARO-AAT clinical studies and providing certain manufacturing services.

    Looking ahead to 2021, we expect our full year cash burn to be in the range of $200 million to $250 million. This increase is due to growth throughout the company. Our program costs are expected to increase as ARO-ANG3 and ARO-APOC3 begin larger Phase 2 clinical trials and our newer programs continue to advance; our headcount increase in 20202 and we expect continued increases in 2021, which drives increases in payroll, related facility costs, discovery R&D and G&A expenses. Our common shares outstanding at September 30, 2020 were $102.4 million.
  11. Missolapola 24 november 2020 08:20
    With that brief overview, I will now turn the call back to Chris.

    Christopher Anzalone

    Thanks, Ken. There is no question we’ve had a productive recent period. The more clinical experience we gain with the TRiM system, the more confident we become that we are on the path to providing physicians with potentially transformational therapies that may make a big difference in the lives of millions of patients. That’s important and gratifying.

    We also feel confident that we have the right strategy. We believe the combination of focusing on well validated targets, our speed from idea to the clinic, our ability to address a new cell type every 18 to 24 months, the rapid nature of our pipeline expansion, and our selective partnering model together provide our shareholders with the potential for rapid value creation.

    So, what’s next for us? We think our initial commercial focus on cardio metabolic and pulmonary will allow us to build out the necessary infrastructure over the coming years in a focused and effective manner, but also in a way that is ever conscious of capital efficiency. That has been an area of hallmark and we intend for that to continue.

    And lastly, we are eager to gain clinical validation for our TRiM platform stability to target tissues outside the liver. This includes lung, tumor, muscle and other tissue types that we have not yet disclosed. RNAi works well in the podocytes we know that but our goal has never been simply to address liver-based disease, rather we have always worked to bring RNAi wherever it is needed and we are on the brink of demonstrating that right now.

    We are indeed building a different kind of biotech company and we look forward to continuing to share our progress.
  12. Missolapola 24 november 2020 08:21
    Thanks again for joining us today. I would now like to open the call to your questions. Operator?

    Question-And-Answer Session

    Operator

    [Operator Instructions] And our first question comes from the line of Shawn Egan with Citi. Your line is now open.

    Shawn Egan

    Hi guys. Thank you for the update today and for taking my questions. As investors start to do work for 2021, can you may be set expectations for ENaC program in regards to what data endpoints there will be. I know FEV1 has come up a few times and I guess what level of benefit there would give you confidence? And you guys also plan to test for ENaC knockdown or are there any other approaches you can use to validate proof-of-concept?

    Christopher Anzalone

    Sure. Javier, would you like to address that?

    Javier San Martin

    Yes. So, let me start with the comment about by when we are going to have data next year, 2021 and it’s likely to be either at the end of the first half or maybe early of the Q3 of 2021. With regard to what we think will be success. As you know, one of the key efficacy endpoint is FEV1 and we haven’t studied in alpha hopefully we’ll be able to see a change from baseline in the range of anywhere between, I would say, 5% and 15%.

    The first of that was approved in this disease was an increase in FEV1 of approximately 5%, the more proven dose approved as you know is more in the range of 10% or 13%. So we are looking for that proof-of-concept. Seen a movement in FEV1 in a Phase 1 study I think will be continued success in our mind.

    Christopher Anzalone

    And let me also expand upon that. So, when we look at our FEV1 results, we are not comparing those two TRIKAFTA. At least our initial patient populations are going after will be those patients who are not eligible for TRIKAFTA either they are known patients and so they have no CFTR to correct or they are refractory to TRIKAFTA or the like.

    So, the bar here is really nothing, right. These patients have very limited therapeutic options. And so, I think that if we can show a 5% improvement in FEV1, that’s a big thing for those patients and I think that suggests that we got to drop.

    Shawn Egan

    Great. And then, maybe one follow-up question on the ARO-ENaC program. Like when you consider the physical barriers present in CF patients, I guess what gives you confidence that the drug will get to the target epithelial cells?

    Javier San Martin

    Well. So, of course, we don't have preclinical data in this model. So we cannot answer the question definitively. One of the things that we're doing to prevent that to happen is the approach to dosing that we will dose – we are dosing patients with three consecutive days, every three weeks, so that allow us to hopefully see that the drug reach out to the anatomical areas in the land where it’s necessary.

    So, I think that is one of the things that we are doing to really trying to maximize that the dose will reach the targeted tissue.
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