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Alnylam Pharmaceuticals, inc (ALNY -0.11%)
Q3 2021 Earnings Call
Oct 29, 2021, 8:30 p.m. ET

Contents:

  • Prepared Remarks
  • Questions and Answers
  • Call Participants

Prepared Remarks:

Operator

Ladies and gentlemen, thank you for standing by, and welcome to the Alnylam Pharmaceuticals Q3 2021 Earnings Call. [Operator Instructions]

I would now like to turn the call over to the company. You may begin.

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Christine Lindenboom -- Senior Vice President, Investor Relations and Corporate Communications

Good morning. I'm Christine Lindenboom, Senior Vice President of Investor Relations and Corporate Communications at Alnylam. With me today on the phone are John Maraganore, Chief Executive Officer; Tolga Tanguler, Chief Commercial Officer; Akshay Vaishnaw, President of R&D Jeff Poulton, Chief Financial Officer; and Yvonne Greenstreet, President and Chief Operating Officer. For those of you participating via conference call, the accompanying slides can be accessed by going to the Events section of the Investors page of our website, investors.alnylam.com/events.

During today's call, as outlined on Slide two, John will provide some introductory remarks, provide some general context and discuss the planned leadership transition. Yvonne will provide some remarks on the leadership transition plans; Tolga will provide an update on our global commercial progress. Akshay will review recent clinical and preclinical updates, including the new positive top line results of the 18-month endpoints of the HELIOS-A study of Vutrisiran. Jeff will review our financials and then provide's a brief summary of upcoming milestones before opening the call to your questions.

I would like to remind you that this call contains remarks concerning Alnylam's future expectations, plans and prospects, which constitute forward-looking statements for the purposes of the safe harbor provision under the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in our most recently quarterly report on file with the SEC. In addition, any forward-looking statements represent our views only as of the date of this recording and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update such statements.

With that, I will turn the call over to John.

John Maraganore -- Chief Executive Officer

Thanks, Christine, and thank you, everyone, for joining the call today. In the third quarter and recent period, we made continued progress bringing our commercial RNAi therapeutics to patients around the world. We advanced our clinical pipeline of over a dozen potential transformative medicine programs, and we delivered on the promise of sustainable innovation, with RNAi, with our industry-leading platform and our preclinical efforts. Of course, a highlight for the period was the positive top line 18-month results from the HELIOS-A Phase III study of Vutrisiran that we announced just yesterday. And Akshay will review those results very shortly.

As we announced this morning, after much thoughtful consideration and planning, I've decided that this is the right time to begin a smooth transition of leadership at Alnylam. I've decided that this is the time for me to begin a new chapter in my career of yet again pushing the bounds of biomedical innovation by helping entrepreneurs and companies advance new modalities to improve the lives of patients. I couldn't be more pleased that Yvonne Greenstreet has agreed to help lead Alnylam in a planned transition that will occur at the end of this year. Of course, I am so honored to have the opportunity to build Alnylam over the last 19 years in RNAi therapeutics to patients around the world.

I am so proud of what we've accomplished, and I'm so thankful for the opportunity to have worked with our past and present Alnylam team to build this incredibly special company. I have no doubt that Alnylam will continue to soar in the years to come, helping patients with its transformative science and medicines.

So with that, let me turn it over to Yvonne to make some remarks as well. Yvonne?

Yvonne Greenstreet -- President and Chief Operating Officer

Thank you, John, and good morning, everyone. We've been so fortunate as Alnylam to have had your tremendous leadership, John, over these last 19 years. And I know I speak for the entire Alnylam team in thanking you for your commitment, passion and excellence. You have been the architect and the inspiration behind our success at this stage, and I'm so grateful that you've given me the opportunity to advance Alnylam in its next exciting chapter. And I couldn't be more excited about Alnylam's prospects going forward with Alnylam P5 times25.

This is our clear road map for delivering RNAi therapeutics to help patients around the world and deliver value to our shareholders. And in the coming year, we expect to bring our fifth RNAi therapeutic to market and potentially expand our TTR franchise opportunity significantly. We expect steady and continued growth of our three directly marketed products with excellence in our commercial performance while also advancing sustainable innovation from our organic product engine, including our first extrahepatic program. These are very bright days for Alnylam. And I really encourage you to join us at our upcoming R&D Day on November 19 to hear some of exciting new progress.

With that, let me now turn the call over to Tolga for a review of our commercial performance. Tolga?

Tolga Tanguler -- Chief Commercial Officer

Thank you, Yvonne, and good morning, everyone. We're pleased with our third quarter performance, particularly with ONPATTRO, but also with our two ultrarare medicines, GIVLAARI and OXLUMO, where we're still in launch phases and seeing some continued impact from the pandemic, particularly for GIVLAARI. For ONPATTRO, we achieved $120 million in global net product revenues, representing approximately 6% quarter-on-quarter growth compared with the second quarter. We ended the quarter with over 1,875 patients on commercial treatments.

In the U.S., we continue to see strength on many fronts, including 12% growth in demand and notable growth in new prescribers. We have also continued to see encouraging signs of the healthcare system reopening. As an example, we've also seen an uptick in face-to-face interactions between our field team and HCPs in the third quarter, resulting in the second highest number of Start Forms for ONPATTRO in the U.S. in any quarter since launch. Further, patient diagnosis is evolving well, with continued growth in the use of PYP scans, which is often the start of the patient journey toward hATTR polyneuropathy diagnosis.

Treatment compliance has returned to pre-COVID levels and remained stable at over 90%, a remarkable result for an infused medicine. We also have confirmed access over 98% of covered U.S. lives with no payer headwinds. Now with regard to the rest of the world, market access has been achieved in over 30 countries worldwide, and notable highlight in Q3 was achieving of pricing and reimbursement in Ireland, which is a country with endemic disease due to the T60A mutation. We're also observing a good balance of first-line use and switches from other products, including stabilizers in rest of world markets. Moving to GIVLAARI. We continue to execute on this product's launch, having achieved $32 million in global net product revenues in the third quarter, representing approximately 4% quarter-on-quarter growth compared with Q2.

As of September 30, we attained over 300 patients worldwide on commercial therapy. We observed a steady quarter-on-quarter patient growth rate of over 10%, driven primarily by geographic expansion, and we're optimistic GIVLAARI will continue to perform well over time. In the U.S., we continue to make strong progress toward establishing Value-Based Agreements with over 10 finalized to date with commercial payers. We also have confirmed access for over 94% of covered U.S. lives with no pushback or headwinds. We continue to make great progress with market access efforts outside of the U.S. with recent launches in Japan and Spain, and achievement of pricing and reimbursement in the U.K. and France.

Moving now to OXLUMO, which is also still in the launch phase. We achieved $15 million in global net product revenues in the third quarter, which represented a decrease of approximately 9% from Q2 revenues. This decrease reflects the transition of the initial bolus of commercial patients from their monthly loading doses to their quarterly maintenance dosing. As we previously highlighted, is a dynamic expected to following launch. As of September 30, we attained over 120 patients on commercial OXLUMO treatment globally, representing over approximately 25 patient growth versus Q2. We believe this demonstrates that notwithstanding the decrease in revenues during the quarter, OXLUMO's fundamental growth remains strong.

Our market access efforts are also progressing well. Now with over 10 VBAs finalized to date with commercial payers and confirmed access for over 85% of covered U.S. lives with no payer headwinds. Geographic expansion of OXLUMO is moving along steadily with two important launches underway, in Germany and France, supported by timely execution of the transitions to commercial drugs for patients previously treated through early access programs. In the CEMEA region, we continue to be pleased by the broad utilization of OXLUMO across age groups and eGFR categories at this early stage of the launch.

In conclusion, we are very pleased with our third quarter commercial results with strong ONPATTRO performance and continued launch execution on GIVLAARI and OXLUMO.

With that, I will now turn it over to Akshay to review our recent R&D and pipeline progress. Akshay?

Akshay Vaishnaw -- President, Research and Development

Thanks, Tolga, and good morning, everyone. I'll start with our efforts in ATTR amyloidosis where we're advancing two clinical stage product candidates, patisiran and Vutrisiran. Whilst ONPATTRO is currently approved in multiple markets around the world to treat the associate hATTR amyloidosis, we're committed to expanding the product's label for the treatment of cardiomyopathy in both hereditary and wild-type ATTR amyloidosis patients.

To this end, we're conducting the APOLLO-B Phase III study, which is fully enrolled with over 300 patients and where we expect to report top line results in mid-2022. We're also advancing Vutrisiran, which is delivered by a quarterly subcutaneous injection and is also in development for ATTR amyloidosis. Here, we're conducting two Phase III studies. The first is HELIOS-A, evaluating Vutrisiran in hATTR amyloidosis patients with polyneuropathy.

Earlier this year, we presented positive nine-month results from the study, which showed that the study met its primary and secondary end points at nine months. These data form the basis for our regulatory submissions to both the FDA and EMA. The FDA has accepted the submission and has signed a PDUFA date to Vutrisiran of April 14, 2022, and the EMA has validated our MAA submission. Patient dosing in HELIOS-A continued past the nine-month mark. And yesterday, we are happy to report positive top line results from the 18-month time point of the HELIOS-A study, which I will now review. Please note that we plan to present the full 18-month results from the study at a medical congress in early 2022, and as such, we'll need to limit our discussion to the top line results only.

These results mark an important step in bringing Vutrisiran a low dose once quarterly and potentially biannual subcutaneously administered therapy to patients living with hATTR amyloidosis with polyneuropathy and as well as progressing our efforts to build an industry-leading franchise of medicines for the treatment of these patients. As a reminder, HELIOS-A is a randomized open-label study in patients with hereditary ATTR amyloidosis with polyneuropathy. The study enrolled 164 patients who were randomized 3:1 to receive Vutrisiran at a dose of 25 milligrams administered subcutaneously once every three months or patisiran administered intravenously once every three weeks at a dose of 0.3 mg per kg. The primary endpoint was measured at nine months and was the change from baseline in the mNIS+7 neuropathy impairment score as compared to the external placebo group from the APOLLO Phase III study.

We're delighted to report today that the HELIOS-A met all secondary endpoints measured at 18 months, including statistically significant improvements in progression of neuropathy as measured by the mNIS+7 score, quality of life, gate speed, nutritional status and overall disability relative to the placebo data from the APOLLO Phase III study of Vutrisiran. The final secondary endpoint, reduction in serum TTRF levels with Vutrisiran demonstrated non-inferiority relative to the within-study patisiran arm as expected. These results build on the positive nine-month Vutrisiran data we shared earlier this year and demonstrated that the reduction of neurological improvement and improvement in quality of life in patients with hATTR amyloidosis seen at nine months is maintained at 18 months.

Furthermore, at 18 months, Vutrisiran treated patients showed quantitative improvement across a variety of exploratory endpoints evaluated in both the intent-to-treat population as well as a prespecified cardiac subpopulation. Improvements were seen in a number of exploratory endpoints, including the biomarker NT-proBNP, a measure of cardiac stress and certain echocardiographic parameters relative to the external placebo group. Other echocardiographic endpoints showed trends toward improvement but did not reach significance due to the small sample size.

Finally, in a cohort of 48 patients, treatment with Vutrisiran was associated with an improvement in technetium uptake that is reduced tracer uptake in the heart relative to baseline in a majority of patients. This is the largest study conducted to date using imaging to characterize the impact of a TTR silencer on cardiac amyloid. Whilst it's important to recognize that these are exploratory data, that these patients have predominantly polyneuropathy, we believe these results are very encouraging with respect to providing potential evidence for reduced cardiac amyloid burden with Vutrisiran treatment. Vutrisiran's potential impact on cardiac manifestations of the disease is currently being studied in the HELIOS-B trial.

Let's now review the safety results during the 18-month treatment period. Vutrisiran demonstrated an encouraging safety and tolerability profile. Study discontinuations occurred in three patients or 2.5% and were due to adverse events in the Vutrisiran arm by month 18. The single new discontinuation since month nine was in a event of cardiac failure considered unrelated to study drug by the investigator. By month 18, there were two deaths, neither of which were considered related to study drug. There were two serious adverse events deemed related to Vutrisiran by the study investigator consisting of dyslipidemia and a urinary tract infection. Deaths and related SAEs all occurred by month nine and have been previously reported.

Treatment emergent adverse events occurring 10% or more of patients included four, diarrhea, pain in extremity, peripheral edema, arthralgia, dizziness, urinary tract infections with the exception of pain in extremity and arthralgia, each of these events occurred at a similar or lower rate as compared with the external placebo. Injection site reactions were reported in five patients or 4.1% and were all mild and transient. There were no hepatic safety concerns.

In summary, Vutrisiran has demonstrated an encouraging efficacy and safety profile in HELIOS-A through 18 months. Moreover, within the realm of reproducing clinical trial data, we're delighted to see the extent to which HELIOS-A results with Vutrisiran recapitulate the APOLLO data with patisiran. As previously noted, the other Phase III Vutrisiran study is HELIOS-B, which is our ongoing Phase III cardiac outcome study with Vutrisiran in hereditary and wild-type ATTR amyloidosis patients with cardiomyopathy. We were excited in the third quarter to complete enrollment in the study with over 600 patients, well ahead of schedule due to strong enrollment demand. Beyond patisiran and Vutrisiran, the newest addition to our ATTR amyloidosis franchise is the preclinical program, ALN-TTRsc04.

Using our IKARIA platform, we've generated a new TTR targeting siRNA, ALN-TTRsc04 that we believe could support an annual dosing regimen with greater than 90% TTR knockdown. ALN-TTRsc04 is planned to enter clinical development with an IND filing at or around year-end 2022. Let's now move on to lumasiran. Our RNAi therapeutic approved late last year in the EU and U.S. as the first treatment for primary hyperoxaluria type one or PH1. In the third quarter, we were pleased to announce positive top line results from the ILLUMINATE-C Phase III study of lumasiran, and we announced today that we plan to present full results from the study at the American Society of Nephrology meeting being held next week.

We also now intend to submit supplemental regulatory filings with the FDA and EMA in late 2021, with the goal of further strengthening the labeling supporting OXLUMO. We're also excited about the potential of lumasiran for patients with recurrent renal stones and later this year, we plan to start a Phase II trial to evaluate that potential. Now in addition to our late-stage programs, we believe we're also making great progress with our early and mid-stage programs. Whilst I can't cover all these programs due to limited time today, one of the exciting parts of our story is the expansion of RNAi therapeutics beyond rare diseases into prevalent disease opportunities.

Our program for high potential is a great example. Zilebesiran, formerly known as ALN-AGT is our investigational RNAi therapeutic targeting the genetically validated target angiotensinogen in development for the treatment of hypertension. We look forward to presenting additional clinical results from the Phase I study at the American Heart Association meeting later this month, including in patients on a low-salt diet and in patients receiving co-administration with the conventional RAAS inhibitor, irbesartan. We also recently initiated our KARDIA Phase II program with zilebesiran.

The first of the two studies, KARDIA-1 is designed to evaluate the efficacy and safety of zilebesiran as a monotherapy in patients with mild-to-moderate hypertension. This study is currently enrolling patients. In late 2021, we plan on initiating KARDIA-2, which is designed to evaluate the efficacy and safety of zilebesiran as add-on therapy in patients with hypertension despite treatment with standard of care. We also continue to harness our organic product engine with the goal of achieving sustainable innovation with two to four INDs per year.

To this end, we're on track to file a CTA for ALN-XDH in development for the treatment of gout in late 2021. We're also on track to file a CTA for ALN-XDH in development for the treatment of gout in late 2021. We're also on track to file a CTA for ALN-APP in development for the treatment of Alzheimer's disease and cerebral amyloid angiopathy late 2021. We're very excited about our APP program as it's the first investigational RNAi therapeutic for our CNS platform, which was also a feature at the OTS Meeting this past month. We believe that our initial human data for ALN-APP expected next year, if positive, could open the frontiers of RNAi therapeutics for many CNS diseases and also herald with potential growth of our broader extrahepatic efforts.

Now in closing and on a personal note, John, thank you for your remarkable leadership over the last 19 years, as Alnylam built a whole new class of medicines, RNAi therapeutics. It has been my great privilege to work with you. You will leave us in a remarkably strong position as we build toward P5 times25 and beyond, my friend.

With that, I'll turn it over to Jeff. Jeff?

Jeff Poulton -- Chief Financial Officer

Thanks, Akshay, and good morning, everyone. I'm pleased to be presenting Alnylam's Q3 2021 financial results, which reflect another quarter of progress on our journey toward building a self-sustainable financial profile aligned with our P5 times25 goals. Turning to our results first for ONPATTRO. We generated $120.3 million in net revenue for the quarter representing 6% growth from the second quarter of 2021 and 46% growth compared with Q3 2020. U.S. ONPATTRO sales decreased 2% versus Q2 2021 and were impacted by the following: demand growth was 12%, consistent with Q2, primarily driven by an increase in patients on therapy and greater than 90% patient treatment compliance. Demand growth was offset by inventory destocking in Q3 compared with stocking in Q2, representing an approximate 10% headwind to reported Q3 growth and higher gross-to-net deductions, which negatively impacted reported Q3 growth by approximately 3%.

In our international markets, ONPATTRO performance remained strong with growth of 12% versus Q2 2021, primarily driven by increased patient demand broadly across markets in Europe, Canada and Japan. Turning to our results for GIVLAARI. We generated $31.8 million in net revenue in Q3 representing 4% growth compared to the second quarter of 2021 and 91% growth versus Q3 2020. U.S. GIVLAARI sales were flat versus Q2 2021 and were impacted by the following: demand growth was 9%, primarily due to an increased patients on therapy and greater than 90% patient treatment compliance. Demand growth was offset by inventory destocking in Q3 compared with stocking in Q2, negatively impacting Q3 reported growth by approximately 8%. With OXLUMO, we generated $14.9 million in net revenue in the quarter, representing a 9% decrease compared with Q2. As Tolga previously mentioned, despite patient growth of approximately 25% in the quarter, sales decreased due to the transition of the initial bolus of commercial patients from monthly loading dose to quarterly maintenance dose regimens.

Turning now to a summary of our full P&L results for the quarter with the following highlights: total combined product sales in the second quarter were $167 million, representing 68% growth versus Q3 2020. Net revenue from collaborations for the quarter was $20.1 million, a decrease from Q3 last year, primarily due to less revenue recognized from our collaboration with Vir. Our combined non-GAAP R&D and SG&A expenses for the quarter increased 12% versus Q3 2020. Key drivers of the increase continued to be additional R&D investment in advancing our early, mid and late-stage pipeline programs and increased SG&A investment to support our three commercial brands, including the launch of OXLUMO. Non-GAAP operating loss for the quarter decreased by approximately $20 million versus the same period in 2020, driven by strong top line growth and more moderate growth in operating expenses, continuing our progress toward achieving profitability. Cash, cash equivalents and marketable securities were $2.3 billion as of September 30, 2021, compared to $1.9 billion as of December 30, 2020.

Notably, the third quarter included the second $500 million payment from Blackstone for the monetization of 50% of inclisiran royalties. Lastly, turning to our full year 2021 financial guidance. We are reiterating our guidance for combined product sales, $640 million to $665 million; for net revenue from collaborations and royalties, $150 million to $200 million; and for non-GAAP combined R&D and SG&A expenses, $1.175 billion to $1.275 billion. Let me now turn to a review of upcoming milestones. We are very excited about the positive 18-month data from HELIOS-A for hATTR amyloidosis patients with polyneuropathy and plan to present the full results at a medical conference in early 2022. With zilebesiran, as Akshay mentioned, we plan to present additional data from the Phase I study at the AHA meeting later this month and plan to initiate the KARDIA-2 Phase II combination study later this year as well. From lumasiran, we plan to initiate a Phase II study for renal stone events in late 2021. This Phase II study will be important for life cycle management of OXLUMO with the potential to enable a Phase III trial to significantly expand the overall opportunity.

We also intend to submit supplemental regulatory filings with the FDA and the EMA in late 2021, based on the results from the ILLUMINATE-C study with the goal of strengthening the labeling supporting OXLUMO. Turning to cemdisiran for complement-mediated diseases. Our partner, Regeneron, plans to initiate a Phase III study of cemdisiran and pozelimab combination and myasthenia gravis. With ALN-HSD, as Akshay mentioned, we expect to report initial safety results in healthy volunteers from the Phase I study at our upcoming R&D Day. We're very excited to file our first CNS CTA for ALN-APP in late 2021 setting us up for potential initial clinical proof-of-concept data in late 2022. And as another step toward expanding our prevalent disease opportunities, we plan to advance ALN-XDH in development for the treatment of gout toward a CTA filing in late 2021.

Let me now turn it back to Christine to coordinate our Question and Answer session. Christine?

Christine Lindenboom -- Senior Vice President, Investor Relations and Corporate Communications

Thank you, Jeff. Operator, we will now open the call for your questions. [Operator Instructions]

Questions and Answers:

Operator

[Operator Instructions] Our first question comes from Maury Raycroft for Jefferies.

Maury Raycroft -- Jefferies -- Analyst

Hi, good morning everyone. Congrats on the updates and congrats on the Chief Executive Officer transition to both John and Yvonne. I look forward to our next steps. For my question for the HELIOS-A data, just wondering if you can contextualize what you have relative to APOLLO-A? And are there any new observations that could inform the APOLLO-B and HELIOS-B studies can read out? And I'm just wondering, just checking based on the data, do you plan to make any supplemental filings for Vutrisiran?

John Maraganore -- Chief Executive Officer

So Maury, let me start by just thanking you and your congratulatory comments. But the question here really goes right to Akshay. Akshay, take it away.

Akshay Vaishnaw -- President, Research and Development

Yes. Maury, obviously, the current data now with Vutrisiran only further strengthen our belief in the drug, the data look outstanding in hATTR amyloidosis with polyneuropathy. These new exploratory data with the cardiac endpoints are extremely encouraging. We're very excited about the technetium scan observations that show reduced uptake of the technetium. And in addition to the previously reported finding at nine months, which we see again an 18 months improvement, in proBNP, improvement in aspects to the echocardiogram and these new -- the technetium data, I think this all bodes well for the performance of Vutrisiran in HELIOS-B and strengthens our hypothesis that TTR lowering is an important way to address TTR-related cardiac disorders. And so our confidence in APOLLO-B, of course, also goes up significantly because of these observations. So I'll stop there and happy to take any other questions.

John Maraganore -- Chief Executive Officer

Does that answer your question, Maury?

Maury Raycroft -- Jefferies -- Analyst

Yes. And just wondering if you do plan on supplementing the Vutrisiran filing?

John Maraganore -- Chief Executive Officer

Yes. Just real quickly on that, obviously, the -- for the European filing, we will be including these 18-month data in the European filing that was agreed to by the [Technical Issues] but with the FDA, there's no needed up in that filing.

Maury Raycroft -- Jefferies -- Analyst

Got it. Okay, Thank you for taking my questions.

John Maraganore -- Chief Executive Officer

Thanks Maury.

Operator

Our next question comes from Gena Wang with Barclays.

Gena Wang -- Barclays -- Analyst

Thank you for taking my questions. Yvonne, congrats on the new position. And John, we will surely miss you. My best wishes to your next journey. And I hope our roads will cross again in the near future. So I have a two-part question on the ATTR franchise. So the first one is regarding the HELIOS-A, just wondering regarding the 30% cardio subpopulation, based on your definition on the cardio subpopulation, which NYHA class this patient will belong to? And then another short question regarding APOLLO-B. I understand that patients will be allowed to take tafamidis at some point in the study. So is there any set time restriction before allowing these patients to take a tafamidis? And this is referring to the 70% patient population that would not be on tafamadis on the baseline?

John Maraganore -- Chief Executive Officer

Yes. Thank you, Gena. Akshay, do you want to handle both questions?

Akshay Vaishnaw -- President, Research and Development

Yes. So with respect to HELIOS-A, we'll present the full data, Gena, at an upcoming medical meeting. But suffice it to say, the vast majority of patients would have New York Heart Association one or two level disease. With respect to APOLLO-B and tafamidis, Pushkal Garg is with us. Pushkal, do you want to comment on that?

Pushkal Garg -- Chief Medical Officer

Sure. Thanks, Akshay. Gena, to your question about TAF drop-ins, ethically patients can drop in or position to do that, but they're discouraged from doing that in the first 12 months. And our statistical analysis plan also accounts for the potential for drop-ins. So we feel good about the design of the study and accommodating that.

Gena Wang -- Barclays -- Analyst

Thank you.

Operator

Our next question comes from Tazeen Ahmad with Bank of America.

Tazeen Ahmad -- Bank of America -- Analyst

Good morning guys. Thank you for taking my questions. John, you've been a terrific Chief Executive Officer. We will certainly miss you, we wish you the best in your future endeavors and look forward to working with Yvonne. Just a clarification question. Are you just going to be joining the Scientific Advisory Board? Or you going to be staying on with the full Board of Directors. And then a quick question. How is enrollment progressing in the KARDIA-1 trial?

John Maraganore -- Chief Executive Officer

Yes. Thank you, Tazeen. Thanks for your very warm comments. And you're going to love working with Yvonne. She is nothing short of terrific. I wouldn't be staying on the Scientific Advisory Board because I cannot do anything other than love the science of Alnylam and can't wait to continue to be involved with the great science and progress that we're making here in the company. As you can imagine, being on boards for a former Chief Executive Officer is always a complicated thing. And I just think it's a lot better for Yvonne if I'm not on the board, and I just focus on the science which I'll do and I'll torture Akshay instead, leave Yvonne in good place. But no, it's a decision that I made because I think it's very difficult for former Chief Executive Officers to stay on boards. And obviously, I will participate in the company on the Scientific Advisory Board. But Akshay, do you want to comment on KARDIA?

Akshay Vaishnaw -- President, Research and Development

Yes, KARDIA-1 enrollment is going well. Generally speaking, Tazeen, we let the trial progress and we have clear line of sight on the last patient in before we give further guidance. But it's going very well. I mean this is hypertension as also there are 1.2 billion patients around the world. So it's an eminently enrollable study and it is going well.

Yvonne Greenstreet -- President and Chief Operating Officer

I mean, John, if I could just add that I'm looking forward to actually what is going to be quite a sort of reasonable transition period here. I'm not actually taking on the role as Chief Executive Officer until January one next year, and John will be staying on as an advisor for the three-or-so months following that period. So we're going to be seeing quite often of each other, still I think take a period of time.

John Maraganore -- Chief Executive Officer

I think so. Thanks, Tazeen. Does that answer your question? Questions?

Tazeen Ahmad -- Bank of America -- Analyst

Yes. Thank you, guys.

John Maraganore -- Chief Executive Officer

Thank you.

Operator

Our next question comes from Ted Tenthoff with Piper Sandler.

Ted Tenthoff -- Piper Sandler -- Analyst

Great, thanks. My sincere congratulations too, I wish you nothing but personal and professional success. It's such a pleasure for a long time. And Yvonne, congrats. It's a really exciting transit that's important for the company. So I wanted to ask a little bit on pipelines in terms of AAP? And maybe, again, that's just still too much summer from the upcoming R&D day. But can you give us a little bit more insight into how you anticipate developing on that asset with Regeneron?

John Maraganore -- Chief Executive Officer

So Ted, you were coming in and out, which asset with Regeneron specifically?

Ted Tenthoff -- Piper Sandler -- Analyst

ALN-APP.

John Maraganore -- Chief Executive Officer

APP, fantastic. Okay. Yes. No, I mean, let me just start by saying one of the really exciting next frontiers for Alnylam is the broader extrahepatic delivery opportunities. And you'll hear a lot about that at our upcoming R&D Day, and I think you're going to be really pleased with what you hear. And of course, the program that's pushing that frontier is ALN-APP, which targets amyloid precursor protein. And we are really excited that the IND should be filed or CTA in this case will be filed by the end of the year and we do expect to have human data next year from that program, which will be really important because if we can reproduce the 70%, 80% lowering of APP and Abeta fragments that we have observed so durably in the primate that would be a major milestone for the entire field, and frankly, for the treatment of neurodegenerative diseases in the future. So we really look forward to that. Now specifically on the development plans, maybe I can point it over to Pushkal to maybe make some high-level comments. Pushkal?

Pushkal Garg -- Chief Medical Officer

Sure. Thanks, John. So with regard to ALN-APP, as Akshay mentioned in his comments, we're going to be filing the IND later this year to take that molecule forward. It has -- we think it has broad applicability across -- if it meets its proof of concept, as John highlighted, in Alzheimer's disease as well as another very, very disabling and fatal condition, cerebral amyloid angiopathy, both of which APP has been strongly implicated in. The initial study that we're planning to do will be opening the IND and CTA in early onset Alzheimer's disease, which is a well-characterized population of patients with severe unmet need. And then based on the results there, where we will be looking initially for safety, tolerability as well as pharmacologic activity, we'll be able to branch into a broader Phase II and III program in those two diseases. So that's our plan.

John Maraganore -- Chief Executive Officer

Does that answer your question, Ted?

Ted Tenthoff -- Piper Sandler -- Analyst

Sure, John. Thank you.

John Maraganore -- Chief Executive Officer

Thank you.

Operator

Our next question comes from Salveen Richter with Goldman Sachs.

Salveen Richter -- Goldman Sachs -- Analyst

Good morning and thank you for taking my questions. John, it has been a pleasure working with you. You'll be missed here and Yvonne, congratulations on the new role. In terms of APOLLO-B and this tafamidis drop-in here, how do you account for this in the statistical plan, just given that patients still do that the option to pursue it, notwithstanding discouragement? And then secondly, on the CNS platform, maybe help us understand just where you stand in the ability to deliver to that tissue?

John Maraganore -- Chief Executive Officer

Yes. Well, let me have Pushkal answer the APOLLO-B question and then Akshay can answer the APP question. But let me just start, Salveen, first of all, by thanking you for your comments at the beginning, of course, but also just reminding you that, obviously, in the design both the HELIOS-B and APOLLO-B study. We come at this with enormous experience in doing clinical studies in ATTR amyloidosis. And we come at this, of course, with a very solid track record, as you know, from our overall clinical development team and designing very robust, typically highly overpowered studies, I might say, as you can note from our traditional P values. So with that as background, Pushkal, do you want to answer Salveen's specific question?

Pushkal Garg -- Chief Medical Officer

Sure. Salveen, I think, I understand why you're asking your question, again, as John highlighted, as we designed the study, we were obviously very cognizant of the availability of tafamidis and the potential for drop-in. There was an allowance for a certain amount of baseline tafamidis use on entry into the study as we talked about, and we modeled out a variety of parameters. The studies do allow for patients to drop in on TAF, particularly guiding after the first year. And as you'll recall, in APOLLO-B, the primary endpoint is a 12-month endpoint of six-minute walk test. The other thing to remember is that as we think about the geographic deployment of the study, we all sort of aware of where TAF is available to patients. And so through an operational -- through the way we operationally manage the study, we can also have some control about the availability of TAF and the potential risks around drop-in. And then also statistically, we assumed some very conservative assumptions, as John highlighted, we're very thoughtful about how we design these studies. And so we considered very conservative assumptions in terms of drop-in and made sure that our powering considerations were robust to even the worst-case scenario. So we feel very, very comfortable with that. And then finally, our analysis plan also takes into account the potential for drop-ins, and there are analytic methods where we can, for example, sensor drop-ins and look at analysis with and without drop-ins, etc. And I'll just highlight that we monitor this very closely, as does our DMC, and we feel very, very confident and good that the numbers are -- continue to be low, and we're monitoring that, and we feel very comfortable with the overall conduct of the study.

John Maraganore -- Chief Executive Officer

Great. Thank you, Pushkal. Akshay, the question on APP?

Akshay Vaishnaw -- President, Research and Development

Yes. So we're very excited about CNS delivery approaches we have in hand, we shared at OTS that we've got a novel conjugate that's a lipid-based system, C16. And we've shown reproducibly, both in small animals, rodents and in nonhuman primates that we get widespread delivery in the central nervous system with potent knockdown, 80% or greater durable knockdown of up to a year and against multiple targets. And these targets we've looked at cell-specific markets, so we know we get knockdown in all the major cell types in the nervous system, the neuron, the ascites microglial cells. And anatomically, the [Indecipherable] looks very encouraging indeed with knockdown in deep brain structures, which I know has been a challenge for other modalities, cortical structures, cerebella structures in the spinal cord. So with that kind of preclinical pharmacology package, one has to be excited, and therefore the ALN-APP program, which we discussed just a few minutes ago, really is in the vanguard of what's to come, and we look forward to exciting data from that program in 2022 with biomarker lockdown, hopefully.

Salveen Richter -- Goldman Sachs -- Analyst

Thank you.

Operator

Our next question comes from Paul Matteis with Stifel.

Paul Matteis -- Stifel -- Analyst

Great. Thanks so much. And I'll echo what everyone else said, John, about working with you and Yvonne, congratulations. I wanted to ask a few more questions just about the transition and why now. And I guess one thing that I noticed going back to the last quarter press release was that Mike Bonney was named Executive Chair really recently and it had to do with legal and compliance functions. And I guess I wanted to just kind of understand, is there anything beyond what was disclosed here? As it just relates to kind of John, your vision strategically, the board's vision strategically? Was there -- I guess, really just flat out, was there any kind of disagreement at all on where Alnylam is going? Or is this purely just voluntary? And do you feel like it's the right time? And I guess if you do feel like it's really just the right time, why not wait until after APOLLO-B, given how big of a value-creating event that could be for Alnylam?

John Maraganore -- Chief Executive Officer

Paul, thanks for the question and your kind words at the beginning. And let me try to be really clear. This was my decision. I want to begin a new chapter. I've been running this company for 19 years, came here when there were six employees as the founding Chief Executive Officer, $17.5 million post-money valuation and took it to $25 billion, and obviously, 1,600 employees in 20 countries around the world. And four approved medicines. So after a 19-year tenure like that, it's not unreasonable to say, "Okay, things are really good. Things are going great, might want to not be a running Chief Executive Officer for the next 19 years." And in terms of timing, yes, I could have waited until next year, I could have waited until two years. I could have waited until 30 years, but I'd probably be dead. So at some point, you just got to make the call about when to make a transition. And I couldn't be more excited about where the company is right now. And I couldn't be more excited about the fact that Yvonne is here as my successor. I mean if I waited for a year and Yvonne got lured to go someplace else, I'd have no successor. So I have to keep kicking the can down the road. So the bottom line is this is the right time. There's never a great time, of course, but I think this is the right time. And in terms of value creation, I'm still a major shareholder of the company. And I want to stay a major shareholder of the company. And I'm obviously going to benefit from the very, in my view, very likely outcome of APOLLO-B, which I think will be a very defining event for the company. Does that answer your question? I don't know, Yvonne, if you want to say something?

Yvonne Greenstreet -- President and Chief Operating Officer

Well, I just want to add that John and I created the P5 times25 goals together. And as you all know, this lays out the very bold vision that we have at the company, delivering transformative medicines to patients around the world with both rare and more prevalent diseases from our amazing product engine as well as delivering excellent financial performance. And we put the strategy together. And going forward, I'm fully committed to progressing the strategy. I think it serves us well as a road map to guide us through the next chapter. And I kind of plan to execute fully against this plan without any loss of momentum.

Paul Matteis -- Stifel -- Analyst

If I can just ask one follow-up. Given that Barry left a year ago and John, now you're deciding it's time. Yvonne, again, congrats, do you expect the rest of the management team to remain in place?

John Maraganore -- Chief Executive Officer

I mean, absolutely, Paul. I mean, we have a very committed team of leaders here in the company. And I have no reason to believe that any changes are going to happen. But we also have a really strong bench. We have a strong group of people that are right underneath these leaders that we have right now. And so we are in a very, very strong position as a result of all that. And I have every confidence that this company will continue to perform unbelievably well for the future. No doubt about it in my mind.

Paul Matteis -- Stifel -- Analyst

Great. Thank you John.

John Maraganore -- Chief Executive Officer

Thanks Paul.

Operator

Our next question comes from Ritu Baral from Cowen.

Ritu Baral -- Cowen -- Analyst

Thanks for taking my question everyone. Yvonne, congrats, really looking forward to working with you, and John, we'll miss you, but that Lyric Opera offer still stands on my part.

John Maraganore -- Chief Executive Officer

All right. I'm in. I'm in.

Ritu Baral -- Cowen -- Analyst

All right. I wanted to ask without getting you in trouble with maybe ACC, I wanted to ask about the potential technetium data that we're going to be seeing out of HELIOS-A next year and especially how that could be important for differentiation and marketing potentially going forward. I guess, one, can you walk us through like the units of that scan and what's a meaningful burden reduction? And are we going to get that same data of APOLLO-B and HELIOS-B?

John Maraganore -- Chief Executive Officer

Okay. Great. So let me have Akshay answer. You asked the question about differentiation as well. So I'm going to have Tolga way out of that, part of it. Let me start though with Akshay commenting on the -- without getting into trouble with ACC if that's where we present it. So Akshay, go ahead.

Akshay Vaishnaw -- President, Research and Development

Yes, Ritu, we will have to wait for the full data to share in more detail. But at a high level, what I would say is, this is a -- there's the metric evaluation tool where you look at the extent of tracer uptake into the heart and what we've done in a significant number of -- actually, the largest number of patients in the study of this kind of ever looked at pre and post-treatment, and we're very encouraged by the fact that the majority show reduction in tracer uptake with technetium. Now that's not a clinically approvable endpoint today. What we know scientifically is that we're encouraged by this because the data would suggest that this could be potential evidence for reduction in cardiac amyloid and that, of course, would be very exciting. So we'll share the full data set with you in due course. But the data can only be supportive and encouraging of what's to come in APOLLO-B and HELIOS-B, and our overall belief that reduction of TTR levels with our drugs, patisiran and lumasiran hopefully, will convert significant clinical benefit for patients with ATTR cardiomyopathy.

John Maraganore -- Chief Executive Officer

And Tolga, do you want to comment a little bit. Sorry, go ahead, Ritu.

Ritu Baral -- Cowen -- Analyst

Akshay, will those two trials also generate technetium cohort data?

Akshay Vaishnaw -- President, Research and Development

Yes. The technetium is an optional assessment in that. Not all patients will have it, and of course, the availability will vary at sites around the world. But yes, there'll be some technetium data. More importantly, I think in those studies, Ritu will be the clinical endpoints, of course, right, which six-minute-walk distance for APOLLO-B, and mortality and hospitalization for Helios-B.

John Maraganore -- Chief Executive Officer

And Tolga, do you want to comment a little bit on Ritu's question around the potential differentiation of these data as it relates to the competitive landscape and so forth?

Tolga Tanguler -- Chief Commercial Officer

Sure. I mean, first of all, obviously, we are very pleased with the ONPATTRO performance and how we've been able to expand our prescriber base and increase our Start Forms. Now there will be patients and physicians who are an waiting and see category. And I believe with a combination of efficacy safety and once quarterly in the future and possibly semiannual subcutaneous treatment regimen, along with -- coupled with the strong commercial capabilities that we already built. I believe this product will enable us to further expand the franchise that we already established, especially in markets where increased convenience, for instance, in Japan, we have limited home infusion availability. The switch dynamic will certainly increase as well as in the EU, where increased convenience could accelerate to switch dynamic from a stabilizer to increase our category share of first line versus other treatment options

Yvonne Greenstreet -- President and Chief Operating Officer

And I'd just like to add one point on that just to emphasize that our commercial organization has done a fantastic job, but we have something like 1,875 patients that are currently on commercial treatment. We have an opportunity that's much, much larger than that. If you look at the patients that would be appropriate for ONPATTRO, and hopefully, shortly Vutrisiran. You're looking at over 30,000 patients. So we really are at the beginning of this journey that I think, going to just continue to pick up steam as time goes on.

Ritu Baral -- Cowen -- Analyst

Great, thank you Yvonne.

Operator

Our next question comes from Anupam Rama with JPMorgan.

Anupam Rama -- JPMorgan -- Analyst

Hey guys! Thank you for taking the question. John, sad to see you go, but it's been really cool to see everything you built, man and wishing you the best. Yvonne, congrats and look forward to seeing you at the conference in January. On patisiran and APOLLO-B, more of a market research question, what does your market research say about a knockdown agent within every three-week IV regimen in a six-minute-walk distance endpoint? How does that kind of fit in versus tafamidis, if particularly in wild-type cardiomyopathy, where we know they have hospitalizations and mortality type of data?

John Maraganore -- Chief Executive Officer

Thanks, Anupam, and I will miss you in January, but I'll perhaps be the audience watching Yvonne. But getting back to your question on differentiation, and specifically the question on how does it all play out, I'll let Tolga answer in just a minute. But let me just start by saying that obviously, if we generate positive results out of APOLLO-B and if the drug is approved for the wild-type and hereditary ATTR cardiomyopathy segment, those are, of course, important ifs. We think that the profile of a TTR silencer, the mechanism of action of a TTR silencer will be a valued part of the treatment options that are available for patients with this disease. And we certainly believe, based on market research we've done that Tolga can comment on, that, that type of availability certainly for patients that may be progressing in their ongoing treatment with a stabilizer drug if they're on a stabilizer drug. That type of treatment option may be important for the patients, again, assuming positive APOLLO-B data and positive approval. So that's at least the foundational aspect of it. Tolga, do you want to add anything more to that as well?

Tolga Tanguler -- Chief Commercial Officer

No, John. I mean, essentially, what we shared earlier as well based on some of the market research studies we've done, we've certainly seen the early indicators of cardiomyopathy or cardiac data, secondary endpoints tend to be seen very favorably by the cardiologist if the product is obviously approved. And obviously, given the profile of the product, they being a quarterly subcutaneous injection clearly addresses the adherence issue, which, again, would be another important indicator.

John Maraganore -- Chief Executive Officer

Yes. Pushkal, do you want to comment?

Pushkal Garg -- Chief Medical Officer

Sure. I think it's an important question. I think maybe just a couple of points to reiterate around ONPATTRO, I mean, and the profile of tafamidis I think it's important to recognize that patients on tafamidis even coming out of the ATTR-ACT study continue to decline in terms of their six-minute walk test. And so we think there's an opportunity, again, based on the data we've seen more broadly with silencing to potentially have a very differentiated clinical profile. The other point around this, and we're starting to see some evidence of that, while there haven't been head-to-head studies, it was interesting, for example, that there was a recent abstract presented at the EU, TTR meeting that looked at technetium scans in patients given the silencer and patients given tafamidis. And Akshay mentioned early on that we've seen evidence of improvements or reduced uptake on technetium scanning with a silencer. And in this case with Vutrisiran and HELIOS-A in that study, they looked at patients on ONPATTRO and saw similar results. And interestingly, in that say, with tafamidis patients and stabilize their patients that they did not see those kinds of improvements. So again, a small study -- academic study. But I think as we start to look at the totality of data that's emerging on our class of silencers versus stabilizers, we are starting to see differentiation. And we'll see then when we get the results from APOLLO-B how those materialize, clinical trial data.

Akshay Vaishnaw -- President, Research and Development

By the way, just to keep the record straight, I was referring to HELIOS-B, not APOLLO-B. So sorry about the subcutaneous one.

John Maraganore -- Chief Executive Officer

Does that answer your question, Anupam.

Anupam Rama -- JPMorgan -- Analyst

Yes. Thank you so much for taking the question.

John Maraganore -- Chief Executive Officer

Thank you.

Operator

Our next question comes from Alethia Young with Cantor.

Alethia Young -- Cantor -- Analyst

Hey guys! Thanks for taking my question. Yvonne, you've been killing it over there. So keep on killing there, congrats. And John, you guys always have a special place in my heart, and it's been great working with you and one of the first companies I ever covered. So all the best, man. So I guess I want to talk a little bit about like big picture, like you guys have a lot of things going on. I'm obviously now starting to branch down to many different indications. So when you look across like the next three to five years, I mean, some of these start to come potentially to fruition, are you looking to kind of build deeper commercial organizations that do more and diversify? Or are you kind of thinking about different other strategic ways and things to do things?

John Maraganore -- Chief Executive Officer

Well, it's a terrific question, Alethia. And you've always been such a great covering analyst, so it's wonderful to have been one of the first companies you've covered. But I think your questions are really terrific one for Yvonne. So I going to just pass it right over to her.

Yvonne Greenstreet -- President and Chief Operating Officer

Yes. No, that's great. I think we're in a really strong position where we put in place to continue to execute. If we look at the opportunities that are ahead of us, they're going to be focused around the cardiac market for the next few years. Obviously, hopefully, we get great data out of APOLLO-B and HELIOS-B. And then we look forward to zilebesiran for hypertension, which we believe is going to completely reimagine the treatment of hypertension. I think we're going to be able to build on this footprint from a commercial perspective within the company. But clearly, as we go forward with all the opportunity we have, what that gives us is tremendous optionality but thinking about how we want to build the business. We're just incredibly fortunate to be standing on the foundation that John has built over the last 19 years, and I couldn't be more excited about we have in front of us.

John Maraganore -- Chief Executive Officer

Does that answer your question, Alethia?

Alethia Young -- Cantor -- Analyst

It does. And congrats, guys.

John Maraganore -- Chief Executive Officer

Thank you.

Operator

Our next question comes from David Lebowitz with Morgan Stanley.

David Lebowitz -- Morgan Stanley -- Analyst

Hi, thank you very much for taking my question. Yvonne, congratulations on the new role. And John, it's been great working with you over the years. A quick question on the data from the Vutrisiran trial. Would you be able to compare the safety profiles of ONPATTRO, Vutrisiran and revusiran the discontinued therapy and as far as similarities and differences across the platforms?

John Maraganore -- Chief Executive Officer

Well, let me give some context and then maybe, Akshay, you can comment on it. I mean revusiran, of course, is a completely different animal, early generation STC, GalNAc conjugate, poorly stable, not very stable, required extremely high doses and turned out to be not well-tolerated in the frail patient population. And obviously, that program was discontinued. I think the big difference between the subcu delivered Vutrisiran versus the IV-infused ONPATTRO really comes down to the type of administration that's involved. If you look at subcu injection, we see a low incidence of mild-to-moderate, generally always reversible type of injection site reactions with subcu delivered Vutrisiran. With intravenously administered ONPATTRO, we see a low incidence, roughly 50-or-so percent of infusion-related reactions, which are not uncommon with IV infused drugs as well. And so I think that's really the biggest difference between them. Of course, Vutrisiran also does not require pre-medication, which is a nice feature around Vutrisiran as well. And so in general, I think those are the main differences. Akshay, do you want to comment any further?

Akshay Vaishnaw -- President, Research and Development

No, I think you covered it well, John. I would just add that the safety profile of Vutrisiran looks extremely encouraging on its face and comparing to the placebo data from APOLLO. And just as one marker of that, I would say, in a three-study discontinuations, in a study of this length with over 120, 130 patients on Vutrisiran is pretty remarkable. I mean these are very frail, sick patients. And with the encouraging efficacy data we've discussed today. I think that all adds to a very encouraging benefit/risk profile. Of course, we'll submit these data to regulators and they have the final judgment, but we're certainly very encouraged by the overall profile of this drug.

John Maraganore -- Chief Executive Officer

Yes. And I would just add to wrap it up, that that obviously both Vutrisiran and ONPATTRO, well, in the case of ONPATTRO, we also have post-marketing data, and we are encouraged by the very consistent post-marketing data as it relates to the study results that we saw in APOLLO. But then in terms of ongoing clinical studies for both patisiran and Vutrisiran in the cardiomyopathy study, the APOLLO-B and HELIOS-B studies, we have data safety monitoring boards that review the unblinded safety data quarterly. And so far, so good in terms of any externally support for continuing to move forward. So that's the landscape, David. We've learned a lot over the years in terms of how to make these molecules better and better. And I think knock on wood, so far so good in terms of how that's played out.

David Lebowitz -- Morgan Stanley -- Analyst

If we jump over to OXLUMO, I know that the -- you were talking about the bolus patients at the beginning, who had the loading dose led to the, I guess, the downtick from last quarter. How should we view that factor going forward from this point, as far as mapping out our run rates going forward for the drug?

John Maraganore -- Chief Executive Officer

Maybe, Tolga and Jeff can take that on. Tolga, do you want to start?

Tolga Tanguler -- Chief Commercial Officer

I mean, look, at the end of the day, we were able to grow our patient basis over 5% and added, now we're at 120 patients. Majority of the patients that we've been able to build over time has been coming from either naive patients where you do see this starting dose impact, and as well as early access program patients that already were getting their starting those prior to the program that before they were commercial. The way I would really think about this is, as we are expanding geographically, we would still see some level of this impact to a degree based on the markets that we're expanding as well as our continuous performance in the U.S. where we didn't really have a lot of early access program patients. So this dynamic will continue, but maybe not to the magnitude that we currently see at this stage. Jeff, do you have anything else to add?

Jeff Poulton -- Chief Financial Officer

The one thing I'd add just on top of that is, I think it's the scale of the opportunity grows, some of the noise that you see from these dynamics will dissipate.

David Lebowitz -- Morgan Stanley -- Analyst

Okay. Thank you very much for taking my questions.

Operator

I will now turn the call back to the company for closing comments.

John Maraganore -- Chief Executive Officer

All right. Well, thank you, everyone, for joining on this call. This is my last one at Alnylam. I want to thank all of you for the support over the last 19 years. We've built a remarkable company that's absolutely here to last. And our next chapter has so much promise for patients and our broader stakeholders. Thank you. Have a great day.

Operator

[Operator Closing Remarks]

Duration: 64 minutes

Call participants:

Christine Lindenboom -- Senior Vice President, Investor Relations and Corporate Communications

John Maraganore -- Chief Executive Officer

Yvonne Greenstreet -- President and Chief Operating Officer

Tolga Tanguler -- Chief Commercial Officer

Akshay Vaishnaw -- President, Research and Development

Jeff Poulton -- Chief Financial Officer

Pushkal Garg -- Chief Medical Officer

Maury Raycroft -- Jefferies -- Analyst

Gena Wang -- Barclays -- Analyst

Tazeen Ahmad -- Bank of America -- Analyst

Ted Tenthoff -- Piper Sandler -- Analyst

Salveen Richter -- Goldman Sachs -- Analyst

Paul Matteis -- Stifel -- Analyst

Ritu Baral -- Cowen -- Analyst

Anupam Rama -- JPMorgan -- Analyst

Alethia Young -- Cantor -- Analyst

David Lebowitz -- Morgan Stanley -- Analyst

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