Q3 2022 Alnylam Pharmaceuticals Inc Earnings Call

October 27, 2022

Okay.

Thank you for standing by and welcome to the on Island pharmaceutical third quarter 2022 financial results Conference call. At this time, all participants are in a listen only mode.

After the Speakers' presentation there'll be a question and answer session to ask a question at that time. Please press star one one when you touched on the telephone.

Today's conference call is being recorded I would now like to turn the conference over to the company. Please go ahead.

Good morning, I'm, Christine <unk> Senior Vice President of Investor Relations and corporate Communications at an island with me today on the phone or Yvonne Greenstreet, Chief Executive Officer, <unk> <unk>, Chief Commercial officer, Oxy based on President and Jeff Poulton Chief Financial 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 investor thought on island Dotcom Flash events.

During today's call as outlined on slide two of them will offer introductory remarks and provide some general context.

So, although we'll provide an update on our global commercial progress.

Okay, well reviewed early pipeline updates and clinical progress and Jeff will review, our financials and guidance followed by a summary of upcoming milestones before we open the call to your questions.

I would like to remind you that this call will contain remarks concerning on the island's future expectations plans prospects, which constitute forward looking statements for the purposes of the safe Harbor provision under the private Securities Litigation Reform Act of 995 axon.

Actual results may differ materially from those indicated by these forward looking statements as a result of various important factors, including those discussed 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 reporting 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'd like to turn the call over to Ivan Ivan.

Thanks, Christine and thank you everyone for joining the call today.

We're very pleased with the results delivered in the third quarter, both commercially and with our pipeline.

Commercially this is the first full quarter, Hey, Brian good trend launch since U S approval back in June , which drove 30% growth in the U S for the TCR franchise compared to quarter two.

Critically the TCR franchise was a highlight as well with <unk>.

Presented positive results with the Apollo B phase III study of <unk> in patients with <unk> amyloidosis cardiomyopathy, validating the therapeutic hypothesis that.

<unk> silencing of GTR has the potential to result in a disease modifying impact on the cardiac manifestations of <unk> amyloidosis.

With a lighter, but the totality of these results and we look forward to submitting a supplemental NDA from Petro by year end.

This also gives us increased confidence in the Helios B phase III outcome study of <unk> in <unk> amyloidosis with cardiomyopathy.

Which is on track for top line results in early 2024.

Our earlier stage programs advance as well with positive results presented from the Phase III study of <unk> in patients with Iga nephropathy as well as preliminary data in patients from the ongoing phase one study of <unk> and HFC in development for the treatment of Nash.

In the coming quarters.

Spectra have more exciting updates from our pipeline.

Including results for potential biannual dose regimen.

<unk>.

Preliminary phase one results in healthy volunteers.

And ex the H and investigational <unk> therapeutic for the treatment of gout.

Completion of enrollment in the cardio <unk> phase II study of Dolby surround.

And the initiation of a phase one healthy volunteer study.

And <unk> for which we filed the Cta earlier this month.

This execution is in line with our focus on these key drivers strong items growth over the next several years.

Firstly, the potential near term expansion of our TCR franchise opportunity, where we aim to become a global leader in delivering impactful and highly differentiated medicines to patients.

Second our expansion beyond rare diseases are good prevalent disease.

And the third growth driver for the company comes from our sustainable innovation engine comprised of new platform enhancement opportunities with extra hepatic delivery and our ability to find new genetically validated targets, which could drive further pipeline expansion to 2025 and beyond.

We believe all of this positions us well to deliver on our nylon pizza fit by 'twenty five goals.

All nine of the top biotech company, developing and commercializing transformative medicines for rare and prevalent diseases for patients around the world driven by a high yielding pipeline of first and best in class product candidate from our organic product engine.

All while delivering exceptional financial results.

With that let me now turn the call over to talk us through a review of our commercial performance.

<unk>.

Thanks, Paul and good morning, everyone.

Q3 was a strong quarter for our commercial portfolio with 9% quarter over quarter growth driven by a strong launch upon which in the U S. As <unk> highlighted.

While still early we are encouraged by several signs, indicating that on which way is expanding the size of the opportunity for our <unk> franchise.

I'll have more details to share on this shortly.

The strengthening of the U S. Dollar continues to impact product revenues from our international operations as the old patio give Laurie Shlomo Q3 product sales.

Each experienced.

FX headwinds compared with the prior quarter and prior year periods.

With initial sales upon which are now reflected in our quarterly financial results moving forward, we will highlight our results for one pathway in Alberta.

On a combined franchise basis.

We saw robust growth in our TCR franchise in Q3, achieving $107 million in global net product revenues for our Petro and Im withdraw representing an 11% increase compared with the second quarter and a solid 41% growth compared with Q3 2021.

At the end of Q3 over 2500 80 patients who are on commercial on pass through all of them, which are treatment worldwide up from over 2400 patients at the end of the second quarter, representing steady 8% quarterly patient growth.

In the U S combined sales of unpopular and a butcher increased a robust 30% versus the second quarter and were primarily impacted by the following.

And 19% increase in demand growth, which was driven by the strength of the initial him with truck patient uptake more than offsetting the decrease in patients on on Petro that switch to ultra.

In total for the quarter 180 patients initiated on water treatment with over 40%, representing new patients falling island, and the balance representing patients switching from one platform.

Initial launch inventory stocking in the distribution channel increased growth an additional 11% in the quarter.

In our international markets.

Q3 product sales.

<unk>, 5% versus Q2 'twenty two despite an increase in patients on therapy as the timing of orders and partner markets and FX headwinds offset an increase.

Our global results continue to be challenged by foreign exchange headwinds with all parts of the year over year reported growth of 41% held back 11 percentage points due to the foreign exchange impact of the strengthening U S dollar.

Now I'd like to provide you with some additional color on our outlook for U S launch.

In the first four months of launch new patients start forms have doubled to an average of 60 start forms per month, excluding switches in comparison to 30 start forms per month for on petrol in the first half of this year, which we believe highlights the strength of the launch to date.

Our demand generation has been healthy and balanced between community count and centers of excellence, while about 17% of start forms have been generated from new prescribers.

We're seeing our patient base broadened to include a variety of newly and previously diagnosed patients starting on with trucks with significant enthusiasm being expressed for ultra's product profile, including quarterly subcutaneous dosing.

Meanwhile, on the access front, given our parity pricing to date, we have not faced with any significant access headwinds and have made significant progress with formulary approvals, providing smooth access to patients that are put on with her therapy.

Additionally, we are pleased with the average time from receipt of start forms to initiation of therapy kilometer patients, which is already in line with our own Petro benchmark.

To wrap up with a woodshop.

Also excited about the recent regulatory approvals in the EU and Japan for <unk> patients with Polyneuropathy and are looking forward to launches in Germany, and Japan in the fourth quarter.

Moving to our ultra rare disease franchise first.

Give laurie.

We achieved $46 million in global net product revenues in the second quarter, representing a 1% increase compared with Q2 dollars 22, and 43% growth versus Q3 dollars 21.

At the end of Q3 over 460 patients who are on commercial <unk> worldwide up from over 420 at the end of second quarter, representing 10% quarterly patient growth.

In the U S sales of <unk> increased 5%.

The second quarter, primarily due to demand growth of 7% by an increase in patients on therapy, which was modestly offset by changes in inventory stocking and gross to net sales deductions.

In our international markets.

<unk> sales decreased 6% compared with the second quarter with growth in patients on therapy more than offset by lower net pricing and FX headwinds.

Finally, the reported 43% increase in year over year global net product revenue growth of your body.

Its held back seven percentage points due to unfavorable movements in foreign exchange rates.

Moving now to our second ultra rare disease product Ochsner mall.

We achieved $16 million in global net product revenues in the third quarter, representing a 10% increase compared with the second quarter.

At the end of Q3 over 230 patients who are on commercial ochsner treatment worldwide up from over 200.

The end of the second quarter, representing 15% quarterly patient growth.

In the U S sales of ocular decreased 10% versus the second quarter as growth in patients on therapy was more than offset by decreased average patient utilization during the quarter driven by fewer patients on the monthly loading those portion of their initial treatment.

In our international business sales increased 29% compared with the second quarter due to an increase in patients on therapy and higher net realized price during the quarter, partially offset by FX headwinds.

Additionally, as we don't Puckering give laurie changes in foreign exchange rates also negatively impacted normal Q3 dollars 22 results with reported year over year growth of 10% held back by 10 percentage points due to the strengthening U S dollar.

In conclusion, we're pleased with the growth in revenues and patient demand achieved in Q3, and particularly with early signs of strong performance associated with our with the launch which we believe represents an important therapy option for <unk> patients with Polyneuropathy and an extra.

There is a growth opportunity for our TTS franchise.

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

Sure.

Thanks, Tony and good morning, everyone.

Start with our efforts in <unk> amyloidosis, where we are advancing two clinical stage product candidates <unk> and <unk>.

<unk> is currently approved in multiple markets around the world to treat the polyneuropathy associated with hereditary <unk> amyloidosis.

If youre expanding the product's label for the treatment of cardiomyopathy in both.

<unk> and wild type <unk> patients.

And we're excited to have recently presented positive results from the Apollo B Phase III study with <unk> achieved a statistically significant and clinically meaningful improvement relative to placebo in the six minute walk test at 12 months.

Every endpoint of the study.

It also achieved a statistically significant and clinically meaningful improvement relative to placebo at 12 months on the Kansas City Cardiomyopathy questionnaire. The studies first secondary endpoint and a key measure of patient self reported health status and quality of life.

But the more but this one demonstrated an acceptable tolerability profile and we're very encouraged by the overall safety profile of <unk>.

Secondly, we believe these efficacy results validate the therapeutic hypothesis that R&R immediate silencing of <unk> has the potential to result in a disease modifying impact on cardiac manifestations of <unk> doses.

With these results in hand, we remain on track to submit a supplemental NDA filing for review by the FDA by the end of this year with the goal of obtaining regulatory approval in the U S.

This medicine available to patients with <unk> amyloidosis cardiomyopathy as rapidly as possible.

We're also advancing <unk>, which is delivered by quarterly subcutaneous injection now amongst <unk> to treat the polyneuropathy of <unk> doses.

<unk> was approved based on results from the Helios a phase III study.

That study includes an ongoing randomized treatment extension, where we're evaluating.

And Youll dose regimen, and we are on track to share results in late 2022.

As it on Patrick here too we are committed to expanding the label to include the treatment of cardiomyopathy inherent tree and wild type <unk> patients.

Is being accomplished with the Helios B phase III study of investigation with <unk>.

And USB, which is fully enrolled has an endpoint of all cause mortality and CV events assessed after at least 30 and up to 36 months and we're on track to share top line results in early 2024.

As was announced this morning after careful consideration we have made the decision not to conduct the optional interim analysis for Helios B.

Several factors drove us to this decision including.

The early completion of enrolment of Helios, B, which meant that any potential acceleration and the approval based on an interim analysis was minimal relative to waiting until the readout from the fully completed study in early 2024.

To our desire to conclude Helios b with the strongest possible data set including outcomes data and three further enhancement in confidence in Helios B with the recently Allstate Apollo B readout.

As such we look forward to the Helios B readout asset yield early in 2024.

We've also announced this morning, but while we remain very excited about the opportunity for <unk> to address startup fees, we do not plan to initiate a phase III study of <unk>. This year as previously planned as we evaluate the impact of the inflation reduction act from therapies being developed to move from an orphan disease.

We're continuing to consider options for the best path forward in starting up disease as we recognize the significant unmet medical need in this patient population.

Lastly, with regard to have GTR franchise, we're excited to announce that a clinical trial application has been filed for LNG carriers here for the first clinical program from our carrier platform, which aims to achieve an annual dosing regimen with highly potent and reversible effects.

We expect to initiate a phase one study in healthy volunteers by year end.

In addition to our late stage clinical programs. We believe we have also been making great progress with our early and mid stage programs.

Notable highlights this quarter was our announcement of positive results from our phase. Two study also this ran an investigational <unk> therapeutic targeting <unk> competitive complement pathway, which is in development in collaboration with Regeneron for the treatment of Iga Nephropathy Orion Jam.

And the study at week 32 treatment from this round resulted in a 37% mean reduction baseline in the 24 hour urine protein creatinine ratio relative to placebo.

Primary readout, the study and important prognostic marker of disease progression.

The results of secondary endpoints were also consistent with the therapeutic benefit of some of this ramp in <unk>.

There were no significant drug related safety signals and we believe these collective efficacy and safety data support continued clinical development of them disrupt monotherapy in patients with ICANN.

We look forward to gaining alignment with regeneron to finalize plans for phase III and sharing those updates with you in due course.

Another exciting highlight recently with the announcement of the preliminary phase one data supporting the clinical advancement of <unk>, an investigational <unk> therapeutic targeting hfc's <unk> in development for the treatment of nonalcoholic <unk> hepatitis or Nash after single dose evaluation in healthy adult volunteers on a multiple doses.

Well in HST are being studied in adult patients with Nash be prices in the first two pump be cohorts, namely 200, 400 milligrams quarterly have completed at least six months on the study the remaining cohorts are exploring a lower dose or later biopsy time points.

In the first two part B cohort and in HST was associated with robust target knockdown and numerically lower liver enzymes and biopsy derived nonalcoholic fatty liver disease activity score over six months in patients receiving <unk> and HFC relative to placebo.

The study was not powered to achieve statistical significance on these endpoints and the primary outcome measure is frequency of adverse events.

<unk> has exhibited an encouraging safety and tolerability profile to date.

Based on these results we plan to initiate a phase II study in adult patients with Nash in late 2022 and collaboration with Regeneron.

Now turning to our <unk> program, which is in development for the treatment of Alzheimer's disease and cerebral amyloid angiopathy.

<unk> is our first investigational conjugate R&R therapeutics targeting of gene expression in the central nervous system, we continue to make progress enrolling patients with early onset Alzheimer's disease and are progressing through the single ascending dose portion of the phase one study.

We announced in our press release. This morning, we now expect initial clinical data from this study in early 2023, a slight change from our previous guidance of late 'twenty two.

The range reflects the continued progression through dose escalation cohorts at a measured pace and the rigor with which we are screening for appropriate patients for this study that meet all necessary inclusion exclusion criteria.

We believe these initial clinical data with <unk> is positive.

Positive will be an important milestone for our CNS platform to show the R&R.

<unk> clinically relevant degrees of target knockdown in the CNS with a safety and dosing profile that supports further development and we look forward to reporting topline results from this study in early 2023.

These are just a few highlights from our broad and innovative pipeline driven by our underlying organic product engine that will drive sustainable innovation and represents a key growth driver for our volume in the years to come.

Presenter of the productivity of our engine, we look forward to two further Cta R&D filings for the first in human studies before year end, namely <unk> for the treatment of metabolic liver disease, including diabetes, which is in our wholly owned program <unk> for Nash in collaboration with Regeneron.

We will be discussing all of this in greater detail at our upcoming R&D day being held virtually on December 15th and I encourage you to tune in.

Let me now turn it over to Jeff to review, our financial results and upcoming milestones Jeff.

Thanks, Akshay and good morning, everyone I am pleased to be presenting a summary of the island's Q3 2022 financial results and an update on our full year guidance.

Starting with a summary of our P&L results for Q3 2022.

Total product revenues for the quarter were $232 million or 39% growth versus Q3 2021.

It is also worth noting that year over year growth in combined product revenues was held back by approximately 10% due to the foreign exchange impact of a strengthening U S dollar and given that approximately 45% of our product revenues are generated via sales in international markets.

Net revenue from collaborations for the third quarter was approximately $29 million, representing a 45% increase compared with Q3 2021, primarily due to an increase in revenue recognized in connection with our collaboration agreement with Regeneron <unk>.

Attributed to an increase in Reimbursable activities under our research services group arrangement. In addition to an increase in revenue recognized associated with partnership clinical trial activities.

Our non-GAAP R&D expenses increased 6% in the third quarter 2022 compared to the same period in 2021, primarily due to increased compensation related expenses. As a result of increased increased employee head count and an increase in development expenses, primarily associated with a ramp up in enrollment in the cardio <unk> and <unk>.

Tier two so I'll be saran phase II studies.

Offsetting by decreased clinical batches manufactured during the quarter.

Our non-GAAP SG&A expenses increased 33% in the third quarter of 2022 compared to the same period in 2021, primarily due to increased head count and other strategic investments in support of our <unk> launch and other corporate purposes.

Our non-GAAP operating loss for Q2, 2022 was $130 million, representing an $18 million improvement compared with Q2 2021, driven by strong top line growth offset by more moderate growth in operating expenses.

Finally, we ended the quarter with cash cash equivalents in marketable securities of $2 3 billion compared to $2 4 billion at the end of 2021 with the decrease primarily due to our year to date operating loss in 2022. This.

This decrease was largely offset by approximately $200 million received from employee option award exercises and approximately $135 million received from the issuance of convertible debt net of repayment of our Blackstone credit facility inclusive of prepayment premiums.

Purchased a capped call transactions and offering expenses.

Continue to believe our current cash balance is sufficient to bridge us to a self sustainable financial profile.

Now I'd like to turn to our full year 2022 financial guidance.

Starting with net product revenues, we continue to anticipate combined net product revenues for our four commercialized products will be between $870 and $930 million. Additionally, given the continued strengthening of the U S. Dollar since we issued our current guidance in April we are currently trending towards the lower half of our $870 to nine.

We are reducing our guidance for net revenue from collaborations and royalties from a range of between 175 and $225 million to a range between 100 $150 million, primarily due to the timing of reimbursable activities in our collaboration with regeneron and lower than anticipated <unk> royalties and sales milestones.

And our guidance for combined non-GAAP , R&D and SG&A expenses remains unchanged and is a range between $1 $390 million and $1 billion and $450 million.

Let me now turn from financials and discuss some key goals and upcoming milestones on back through the end of 2022.

We will continue executing on our global commercialization upon Petro and Blue truck of Lori <unk>.

Next our TCR franchise will have important updates.

With <unk>, we plan to submit the NDA for review by the FDA for the treatment of patients with <unk> amyloidosis with cardiomyopathy.

With <unk>, we plan to report results on a biannual dose regimen from Helios, a and <unk> amyloidosis patients with Polyneuropathy.

Lastly, frail and Trs <unk>, we are on track to initiate a phase one study in healthy volunteers.

And our mid and early stage portfolio. We are looking forward to milestones include completion of enrollment in the cardiac two phase III study of <unk> at or around year end.

Preliminary results expected from healthy volunteers in the phase one study of <unk> in development for patients with gout.

And submission of Cta filings for <unk> for the treatment of metabolic liver disease, including diabetes and airline Pnp for Nash.

Let me now turn it back to Christine to coordinate our Q&A session Christine.

Thank you Jeff Operator, we will now open the call for your questions today than we would like to ask you to limit yourself to one question each and then get back in the queue.

Additional questions.

Thank you again, ladies and gentlemen, if you like to ask a question. Please press star one on your Touchstone telephone.

Again to ask a question. Please press star one line.

Our first question comes from Maury Raycroft of Jefferies. Your line is open.

Hi, good morning, and thanks for taking my question.

I was wondering if you can talk about whether you've got any feedback from FDA on Apollo data and what the NDA package would contain.

Also if you could talk about confidence around your primary end point P value and whether FDA has provided any specific comments on that.

Thanks for that question, Marc I mean, clearly we're delighted with the results from our Apollo B study, where we deliver clinically meaningful.

Significant benefits across there.

Six minute walk test case.

<unk>, which are important measures actually for patients in terms of how they function and feel when we got good safety and new medical reduction. So we're very confident with the package of data that we have supporting the syndication holistically, particularly given the small size on short duration of the study.

Akshay perhaps.

In.

Our regulatory pre.

Progress.

Yeah. So thanks, Marty and thanks, a lot I mean, I think you addressed for the high level. The key positives that came out of the study.

Greenfield is that R&R.

<unk> therapeutics can have a significant impact we believe in <unk> amyloidosis, including in the cardiomyopathy.

We are proceeding to file the supplemental NDA by the end of year as we've said we're confident in this package.

<unk> gone outlined.

As the fall goes under review.

The idea was get the safety update 180 day safety update during review, so we'll be providing that of course.

And we will be very responsive to any questions. They have.

And so thats about as much as I can say at this point in time, but we've said the data across two maintains at IFA in Hff's had.

Are extremely strong and positive response is strong.

<unk> Kols and others and so we're excited to focus us on there that's great for us moving forward expeditiously. Thank you Mary next question.

Thank you.

Our next question comes from solving Richter.

It is open.

Lasalle ESB and from did the outcomes analysis of Apollo B impact your thinking on <unk> treated or placebo separation herein and how much time do you safe. Thank you.

Actually I think Thats fair.

So can you actually.

Repeat the full question I think talking about the outcomes analysis, but the lines operator.

We didn't quite catch the second half of your question could you repeat that for us sure.

Sure.

You explained the factors you considered when deciding not to conduct the Helios B interim I'm, just wondering whether the outcomes analysis of Apollo B impact your thinking on treated placebo separation and how much time, you're safe here.

That's really the question. The question is around the thinking behind not progressing the interim analysis, yes.

I mean as you as you intimate the positive data out of the portfolio be significantly increase our confidence and belief that the design and conduct the <unk>.

<unk> is the right test of the hypothesis that <unk> can help patients with <unk> cardiomyopathy. So we feel quite bottomed out there looking at all the detailed data most of which we've shared already of these international meetings, but there are others.

And so that was one major factor the second was.

We obviously want to have the best data associated with <unk> to make it a best in class product.

For patients with <unk> cardiomyopathy.

The best way to do that is to preserve alpha and not mess around with the statistics, which obviously if youre doing the interim.

The downstream consequence on how both the statistics for the final.

Readout in early 'twenty four.

And thirdly is just the speed at which everything which would be likely but.

The fact that Helios b low actually over enrolled at the speed that it did means is such a narrow timeframe between any putative interim that any potential approval thereof.

As the full readout in early 2004, and hopefully getting a great label that is serving patients with <unk> cardiomyopathy. That's.

That's great. Thank you very much the question.

Thank you one moment please.

Our next question comes from Praveen Ahmad Bank of America. Your line is open.

Hi, Good morning, guys. Thanks, so much for taking my question.

Maybe if I can just clarify whether your team has already met with FDA and from your pre NDA meeting regarding.

The Apollo being results, yet or is that still to come and then secondly, do you have any expectations about whether or not an AD com would be needed to review. This filing. It is the second indication proposed from an operating transcribed I just wanted to get your latest thoughts on that thanks.

Yes, I mean as you know we don't we don't comment on regulatory interactions I think.

Akshay cover this in another question, we're very confident with the package that we have supporting the syndication and we're moving forward as.

As planned with a submission date.

Later this year.

That's about all I can say.

On the topic, but thanks for the question.

Thank you.

Our next question comes from Eliana Merle of UBS. Your line is open.

Okay.

Hey, Thanks, so much for taking the question just another one in terms of thinking about Helios b as well as the potential uptake.

You may apathy could you help us understand how you interpret the data and <unk> combination and what this could potentially mean in terms of uptake and Dominic <unk>, our combination and the railroad commercially any.

Any initial feedback you are hearing initially I know, it's early but just curious your thoughts.

Sure.

Alright, So I think maybe kind of two parts to the question I think one I'd say it maybe start with you.

That's around.

The data in combination with <unk> and.

May be talk against some perspectives on how you see things like commercial.

Thank you Kevin.

Vis vis your question Liana.

However, this subgroup concomitant treatment right.

Just to step back.

<unk> got a large robust study that considered the real world population out there. The majority of them don't have any drug right now about 75% of the study was.

Placebo no background therapy.

At about 25% of patients had background families.

Yeah.

I refer you to our Hff's high output in both Maryland is very eloquent discussion of fly.

No it is.

Little bit of a fool's errand to use of face to cut into small subgroups.

Now, let's see.

That really are not powered the study was not designed has so we fool ourselves.

And again until tomorrow explain them discuss told us very well for the HFF side.

Yes.

<unk>.

The readout of the study I think was robust and excellent impacting patients.

Functionality via the six minute walk distance on the quality of life, there's lots of inventory consistency in the data. So this is a strong package.

And we're delighted with that I appreciate the interest of groups, that's really noticed but it was powered to readout.

Yes, so let just generally reflect those around.

The commercial.

Just to build on <unk> point, it would be premature for us to comment anything specific on without the label, but what we can tell you is if you just look at the numbers since the launch of the firm. It is the diagnosis rate went up by 10% 10 times and we certainly anticipate that to continue this is a highly devastating disease across the.

Across the U S and ex U S. There's about 250 to 300000 patients suffer from this devastating disease and what we know is those patients that are a lot of untreated patients and those patients who continued to progress on tough, but we believe.

Our upcoming Cherokees, if theyre approved will be a.

A very attractive option for physicians and obviously for patients.

Thank you so perhaps.

The medical need felt that these patients. Unfortunately next question. Please.

Thank you.

Our next question comes from Ritu <unk> of Cowen Your line is open.

Good morning, guys. Thanks for taking the question.

Ivano Akshay should we be expecting priority review on the SMB E. Just given what you and your regulatory consultancy as the unmet need in PCR cardiomyopathy and can you just also remind us when the last interaction you.

Had with SBA.

Was in the past obviously not forward forward looking and if theres been any cigna.

Significant turnover in the review team our senior members.

Review Division since then.

Thanks, Ritu so another question about China.

The regulatory.

Outlook actually in terms of.

Perfect.

Okay.

Sure.

Another question really just asking us to comment on.

Our progress with regulate transaction. So I think it's sort of a quick question, but maybe you can reiterate the point yes.

Thank <unk>. Thanks for the question as you know we don't discuss.

This goes back and forth with regulators I will reiterate what I said earlier that we're confident in the state of <unk> because we are on track to file the supplemental NDA by the end of the year and the priority review with.

The fda's decision and they have to evaluate.

The pros and cons of this therapy and the data package was submitted in.

We are eagerly awaiting that decision.

Not much more items.

Can you comment on a few key gentiva.

I think I think that's what the FDA management were not identical.

Efficient so to speak to that.

Ken.

Next question.

Thank you. Our next question comes from Jessica Fye of Jpmorgan. Your line is open.

Hey, guys. Good morning, Thanks for taking my question.

Did you have a pre NDA meeting yet or the on Petro cardiomyopathy filing or if not do you expect it to you.

Yes. Thanks for the question I mean again I think there's lots of interest around this the most important thing is that.

We obviously have lots of back and forth with regulators across all our programs.

And let me just reiterate that we will follow the supplemental NDA by the MBA program.

Based on everything we've discussed so far on this call and many other alright. Thanks for the question. Thanks. Mike next question. Please. Thank you. Our next question comes from the line of David Lebowitz of Citi. Your line is open.

Thank you very much for taking my question.

You look at the on Petro acutely ambulatory new patient start forms I noticed that the number of new patients.

Percentage uptick.

From what was announced in the first quarter of launch I think we're about a third to nearly 50% could you comment on.

What the expectations for new patients be coming to market is.

And what that might.

How that might impact the growth of the overall franchise relative to Petra would have done.

Without the presence of ample trial.

Great question.

Second piece of the performance of our GTR franchise, especially the progress of bandwidth.

With.

I'll switch. It then I think you made the point about the GTR franchise, which I'd like to underscore I think we now really have the GTR franchise, we expect to be able to.

But it's all good.

So if you want to add some color maybe how you're seeing the outlook for the GTR franchise no no absolutely Yvonne looked early signs of U S. <unk> launch is a good testament to the old royalty TR franchise future.

Our TCR franchise grew by 30% versus last quarter.

Driven mainly by almost two thirds of this growth was really demands.

In our view early signs of performance really confirm cheeky beliefs. One is despite available treatments, there's a significant unmet needs and the role of a mitra.

Attractive product profile.

We will continue to increase growth that's going to be important.

And also we do have a strong foundational capabilities that will build over time across customer facing capabilities.

That will allow us to continue expand this growth. So we're really excited about this.

We're still scratching the surface in terms of diagnosis and treatment.

There are 10 to 15000 patients in the U S that suffer from this condition. We believe an <unk> product profile will be a very nice fit to the treatment regimens.

Thanks.

Next question please.

Our next question comes from the line of Gena Wang of Barclays. Your line is open.

Thank you for taking my questions.

Sorry, I'll ask Apollo B question again.

I think that because quite some questions were raised.

Our profitability thanks.

It's only a one study and then there is no outcome.

So maybe if you could refresh our memory the history of Apollo B study, so basically how steady with design.

And was it in line with the FDA.

Thanks Kenny.

Thanks Gene.

I think we've discussed much of this on other calls, but let me just reiterate Gina.

<unk>.

I think this is.

I'm, losing track a little bit faithful seven phase III study of <unk> phase III studies, we've negotiated with the FDA and other regulators around the world.

We will now work.

<unk> is in collaboration with guidance from regulators.

We haven't.

Our nylon products approved based on the excellent set of dialogues and relationships. We have an understanding how regulators are trying to guide us.

This product in this trial was no different from that and so if any credibility and confidence is derived from a product performance failure.

I'd like you to apply it here now in the specifics here when Apollo was done and we will remember the conversations those of you that were involved in.

The cardiac data the cardiac subgroup data from Apollo went very promising.

The FDA said, yes that is the case and we've discussed that before and based on that they guided us that we should do affect us study in cardiomyopathy population.

And with that conversation in collaboration with designs that you've seen in Apollo B.

Resulted and so this study was done in close collaboration with regulators and we're grateful for the guidance and we're very happy with the outcome. We're clearly we've shown the impact on patient functionality and quality of life, which are two cornerstones of the FDA.

Based on the approval of drugs safety looks very encouraging to us too.

Then finally on the mortality from you.

You will recall the four patients died on placebo Robert.

Shannon.

So feedback encouraging although it's a small number. So this package. We believe is rather strong first trial that was designed in collaboration with regulators and <unk>.

We're looking forward to filing the NDA.

Thank you.

Thank you. Our next question comes from the line of polymer piece of Stifel. Your line is open.

Hey, thanks, so much.

I was.

Really interested in your comments in the press release and in the prepared remarks on drug positioning related to the inflation reduction act and kind of broader drug pricing risks.

To that point, you just filed a cta for our once annual GTR knockdown drug how do you think about positioning that and kind of balancing star guard versus this potentially just being a nextgen and GT are cardiomyopathy and also enabling you to maintain premium pricing.

Beyond a decade in that space. Thanks, so much.

Yes, thank you for the.

The question I think.

So look the inflation reduction <unk> is a significant shift in Medicare and how the federal government regulate our industry we are.

Our support and that the new part D based on out of pocket caps that we have considerable concern.

About a couple of other aspects of.

Legislation created the first is around.

Concerning the small molecule medicines.

Faith negotiate that Medicare reimbursement rates in the nine years after approval compared to 13 years for monoclonal antibodies I think they present to us in the near term is that while the extensions of the inflation reduction from.

<unk> Medicare price negotiation for a drug that has a single orphan drug indication that allow disincentives to pursuing the approval of drugs and additional indications.

Bill digesting the legislation, but as you pointed out in the meantime, despite the pause button for <unk> phase III study and targets as.

As we consider our options for the best path forward.

It's just kind of three <unk> one is that we remain.

Sites that actually by the opportunity for an <unk> mediated ctr lowering medicine as a potential therapy in patients with startup disease. We think it's an important unmet medical needs.

And the second I think you touched on it when he talked about.

<unk> CFO .

Platform company, we really have the opportunity.

The interface and bring Optionality to how we think about the better thing.

<unk>.

Our medicines and I think.

<unk> CFO .

Another PTR lowering.

Program that that we're moving forward, which we're very excited about.

I think the other the other point that I'd like to make so I think it's important just to remind everybody that <unk> strength.

<unk> has orphan.

Yes.

Disease designation for <unk> amyloidosis.

Northeast or cardiomyopathy.

As with many other companies, we're reflecting on the implications of this relatively new that just based on how we think about our portfolio, but it's a company. We actually think we're very well positioned given our given our platform and our ability to continuously innovate around.

<unk>.

And maybe next question.

Our next question comes from the line of Gary Nachman of BMO. Your line is open.

Hi, good morning.

If you end up getting them patch B approval, let's say end of next year, but then youll be bumping up against the interest around Helios B data in early 'twenty four how much of an impact do you think that might have on your launches on Petro and CN when physicians there'll be anticipating this other important dataset I know it is.

A bit early thinking about the launches that given the timing of these I was curious to get some of your early thoughts on that thanks.

So I'll just start off by saying that the.

And the reason why we progressed.

Patrick.

And the cardiomyopathy indication, but let me just the level of unmet medical need for patients who continued to progress.

I debated and really we took the opportunity to bid on projects plus bring in near term.

New setting for these patients whilst we continue to develop.

Between <unk> and our Helios a study.

And as you pointed out we will have those results shortly at the beginning of 2024, So we think fits.

In a way able to meet the needs of patients in the near term hopefully with the approval of <unk> and CN and then.

As we progressed CESC bring forward.

And even more attractive regimen.

For these patients.

So shortly afterwards, but may be told that you can comment on how we're thinking about.

The launch of our Petro.

With with the opportunity.

Inventory strategy.

To these patients.

The median seven yes.

No I think you summarized perfectly ivana all I would add is.

There is a big unmet meet unmet needs. There are patients who are currently on available treatments that are continuing to progress at to your point, we want to make an alternative treatment available.

As quickly as possible. So we can actually meet those needs and also continue to build our capabilities for the larger indication. We're obviously very pleased with the capabilities that we built.

Okay.

Based on based on what we were actually able to demonstrate with our mutual Qian launch and as we continue to expand the indication. We believe are experienced with the <unk> is going to help us to have even a stronger launch with on road truck cm. If it's approved.

Thanks to all of that so great. So again with sort of TCR franchise approach.

So we think it will be important for patients.

Patients in our commercial performance going forward. Thanks.

Next question.

Our next question comes from Luca <unk> of RBC capital.

Oh, great. Thanks, so much for taking my question, maybe one on the pipeline if I may so maybe on ABP kind of any additional color on why the readout has been pushed out and maybe bigger picture can you level set expectations into data next year, what reduction in CSF <unk> Alpha and beta would you consider a meaningful and maybe what the earliest we could pass.

Simply see functional data for that dataset. Thanks, so much.

Great. Thanks, Thanks Luca.

A question on scale and AEP look where we're clearly excited about the potential for.

RNA therapeutics and addressing CNS disease.

This is the first one out of the gate.

We're progressing very well, but thoughtfully as I've described.

Introductory remark, but actually maybe you can you can speak to is the data that we have.

Expect to answer.

And so the <unk> study the crucial data so I think first and foremost as safety. This is our first candidate in the central nervous system.

Keen interest the study is progressing well.

We are very comfortable so far.

Even beyond that.

Obviously, there's lots of interest in the biomarker outlook. So we'll be looking for <unk> knockdown.

And the fragments various language per diluted frankly from AEP changes it knows that the CSF.

And.

In addition to that Theres going to be important PK because again, it's the first time, we've injected that joke.

The interest equally so those are the key data points in the short term that will be looking at and looking forward to reporting in early 'twenty three.

Absolutely.

As we move forward in <unk>, we are able to demonstrate impact.

Impact on patients with <unk>.

<unk>. This is of course, it opens up the opportunity for a whole host of them.

Devastating CNS disorders, so painful program process.

Okay.

We're progressing well thank.

Thank you for the question.

Question. Please.

Thank you. Our next question comes from Joseph Stringer of Needham <unk> Company. Your line is open.

Hi, Good morning, Thanks for taking my question a question on <unk> start forms and the percentage.

Source from new prescribers.

This quarter you have around 17% that was similar to 20%.

Sure from your last update you sort of see this.

And fairly stable in that 15% to 20% range going forward or how do you see that.

Going forward. Thank you.

Although thats clearly a question for you how do you see the sensitive new subscribers going forward, Yes look I mean, I am glad you highlighted that we're really excited about sort of how we are displaying those strong foundational capabilities that we've been able to build from diagnosis all the way to access support.

One of those exciting is as we see a healthy balance of physicians both from academic centers and community specialist start prescribing this medication and as you pointed out another important metric is the new prescribers, we see a steady increase of this new specialist around 20% month after months being added.

To the prescriber base.

And as a result of that we also see patients are treated a bit younger.

Sign that patients are getting diagnosed.

And obviously, they're getting downwards treatments.

Another important metric that we also see and.

We're obviously going to pay a lot of attention to this as our start forms to treatment rates.

Despite having a temporary J code, it's been similar to old patio and again, thanks to our good access support Vba's at our price parity strategy. So because of those elements. We believe we're going to continue to see a good expansion of new prescribers and net new patients for <unk>.

That's going to drive the GTR franchise to the next level.

Yes.

Super. Thank you very much I think we got time for one more question.

Thank you next.

Next question comes from Myles Minter from William Blair. Your line is open.

Hi, Thanks for squeezing me in just on absolute <unk> actually just with the recent FDA label expansion are you expecting like a significant amount of warehouse advance pay us one of the patients.

And I guess, how you're thinking about growing that franchise moving forward considering this book.

Flat over the past quarters.

Okay do you want <unk>, yes.

Look.

Can you just won is a devastating disease and we're very pleased to be able to actually provide.

Important treatments for these ultra.

Altra rare disease patients.

I see.

Been a great addition, we do see a good.

In addition to our data suite of data that we have we see our goods.

The group of patients both from pediatric and adults.

Evel as well as various.

Progressive stage of their diseases eliminate as simply another important element that will allow the prescribers to be able to prescribe this medicine.

To a broad range of patients that suffer from this ultra rare disease.

Look we've had a great quarter.

We really are baked into our financial performance and we're seeing strong growth in.

Patients on therapy across all of our customers.

So.

I'm, sorry, if I confused about performance.

So.

Thank you everyone for joining us on this call.

We made great progress in the third ultra in 2022.

Our strong commercial results, but also we're pleased with how we are advancing a diverse pipeline programs that are in development.

A lot of excitement ahead.

Catch us on deck in the coming months and we of course look forward to updating you along the way as we continue to deliver on our near and long term. So thank you everybody and have a great day.

Thank you ladies and gentlemen, this does conclude today's conference. Thank you all for participating you may now disconnect have a great day.

The conference will begin shortly to raise Johan during Q&A you can dial one one.

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Thank you for standing by and welcome to the nylon pharmaceutical third quarter 2022 financial results Conference call. At this time, all participants are in a listen only mode.

After the Speakers' presentation there'll be a question and answer session to ask a question at that time. Please press star one on your Touchtone telephone.

As a reminder, today's conference call is being recorded I would now like to turn the conference over to the company. Please go ahead.

Good morning, I'm, Christine limit them senior Vice President of Investor Relations and corporate Communications at an island with me today on the phone or Yvonne Greenstreet, Chief Executive Officer, Phil The Cannula, Chief Commercial officer, Akshay based on President and Jeff Poulton Chief Financial Officer.

For those of you participating via conference call. The accompanying slides can be accessed by going to the event section of the investors page of our website investor thought on island Dot Com flash events.

During today's call as outlined by two Yvonne will offer introductory remarks and provide some general context.

Although we'll provide an update on our global commercial progress Akshay will review early pipeline updates on clinical progress and Jeff will review, our financials and guidance followed by a summary of upcoming milestones before we open the call to your question.

I'd like to remind you that this call will contain remarks concerning <unk> future expectations plans prospects, which constitute forward looking statements for the purposes of the safe Harbor provision under the private Securities Litigation Reform Act of 995.

Actual results may differ materially from those indicated by these forward looking statements as a result, various important factors, including those discussed our most recently quarterly report on file with the SEC. In addition, any forward looking statements represent our views only as the date of this report and should not be relied upon as representing our views as of any subsequent date.

We specifically disclaim any obligation to update such statements.

That I would like to turn the call over to Ivan Ivan.

Thanks, Christine and thank you everyone for joining the call today.

We're very pleased with the results delivered in the third quarter, both commercially and with our pipeline.

Commercially this is the first full quarter, Hey, Brian Good tread launch since U S approval back in June , which drove 30% growth in the U S for the TCR franchise compared to quarter two.

Clinically the TCR franchise with a highlight as well with.

We presented positive results with the Apollo B phase III study of <unk> in patients with <unk> amyloidosis with cardiomyopathy.

Do you think the therapeutic hypothesis that.

<unk> mediated silencing of GTR has the potential to result in a disease modifying impact on the cardiac manifestations of <unk> amyloidosis.

With delighted with the totality of these results and we look forward to submitting a supplemental NDA from Petro by year end.

This also gives us increased confidence in the Helios B phase III outcome study of <unk> in <unk> amyloidosis with cardiomyopathy.

Which is on track for top line results in early 2024.

In the coming quarters.

To have more exciting updates from our pipeline.

Including results for potential biannual dose regimen.

The tree threat.

Preliminary phase one results of healthy volunteers.

XT eight an investigational <unk> therapeutic for the treatment of gout.

Completion of enrollment in the phase III study of Dolby surround and initiation of the phase one healthy volunteer study.

And <unk> for which we filed the Cta earlier this month.

This execution is in line with our focus on these key drivers for our items growth over the next several years.

Firstly, the potential near term expansion of our TCR franchise opportunity, where we aim to become a global leader in delivering impactful and highly differentiated medicines to patients.

Second our expansion beyond rare diseases with prevalence disease.

And the third growth driver for the company comes from our sustainable innovation engine comprised of new platform enhancement opportunities with extra hepatic delivery and our ability to find new genetically validated target, which could drive further pipeline expansion to 2025 and beyond.

We believe all of this positions us well to deliver on our nylon pizza fit by 'twenty five goal, making an item of top biotech company developing and commercializing transformative medicines for rare and prevalent diseases for patients around the world driven by a high yielding pipelines assessed.

And our best in class product candidate from our organic product engine, all while delivering exceptional financial results.

With that let me now turn the call over to <unk> for a review of our commercial performance.

<unk>.

Thanks, Paul and good morning, everyone.

Q3 was a strong quarter for our commercial portfolio with 9% quarter over quarter growth driven by a strong launch upon between the U S is <unk>.

<unk> highlighted.

While still early we.

We are encouraged by several signs, indicating that on which way is expanding the size of the opportunity for our <unk> franchise.

I'll have more details to share on this shortly.

The strengthening of the U S. Dollar continues to impact product revenues from our international operations as the old patio give Laurie Shlomo Q3 product sales each experienced FX.

<unk> headwinds compared with the prior quarter and prior year periods.

With initial sales of <unk>, which are now reflected in our quarterly financial results moving forward, we will highlight our results for one pathway in Alberta.

On a combined franchise basis.

We saw robust growth in Aki TR franchise in Q3, achieving $170 million in global net product revenues for our Petro and <unk>, representing an 11% increase compared with the second quarter and a solid 41% growth compared with Q3 2021.

At the end of Q3 over 2500, 80 patients who are on commercial loan portfolio all of which are treatment worldwide.

Up from over 2400 patients at the end of the second quarter, representing steady 8% quarterly patient growth.

In the U S combined sales of <unk> and <unk> increased a robust 30% versus the second quarter and were primarily impacted by the following.

And 19% increase in demand growth, which was driven by the strength of the initial patient uptake more than offsetting the decrease in patients on on Petro that switched encore trial.

In total for the quarter 180 patients initiated on water treatment with over 40%, representing new patients called island and the balance representing patients switching from one Patrick.

Initial launch inventory stocking in the distribution channel increased growth an additional 11% in the quarter.

In our international markets.

Fossil Q3 product sales decreased 5% versus Q2 'twenty two despite an increase in patients on therapy as the timing of orders and partner market and FX headwinds offset an increase.

Our global results continue to be challenged by foreign exchange headwinds with all parts of the year over year reported growth of 41%.

At 11 percentage points due to the foreign exchange impact of the strengthening U S dollar.

Now I'd like to provide you with some additional color on our outlook for U S launch.

In the first four months of launch new patients start forms have doubled to an average of 60 start forms per month, excluding switches in comparison to 30 start forms part of mothball petrol in the first half of this year.

Which we believe highlights the strength of <unk> with the launch to date.

Our demand generation has been healthy and balanced between community count as centers of excellence, while about 17% of start forms have been generated from new prescribers.

We're seeing our patient base broadened to include a variety of newly and previously diagnosed patients started going downward track with significant enthusiasm being expressed for <unk> product profile, including quarterly subcutaneous dosing.

Additionally, we are pleased with the average time from receipt of platforms to initiation of therapy kuramoto patients, which is already in line with our own Petro benchmark.

To wrap up with a woodshop.

Also excited about the recent regulatory approvals in the EU and Japan for <unk> patients with Polyneuropathy and are looking forward to launches in Germany, and Japan in the fourth quarter.

Moving to our ultra rare disease franchise first give Laurie we achieved $46 million in global net product revenues in the second quarter, representing a 1% increase compared with Q2 dollars 22, and 43% growth versus Q3 dollars 21.

At the end of Q3 over 460 patients who are on commercial <unk> treatment worldwide up from over 420 at the end of second quarter, representing 10% quarterly patient growth.

In the U S sales of <unk> increased 5% versus the second quarter, primarily due to demand growth of 7% by an increase in patients on therapy, which was modestly offset by changes in inventory.

Talking and gross to net sales deductions.

In our international markets.

<unk> sales decreased 6% compared with the second quarter with growth in patients on therapy more than offset by lower net pricing and FX headwinds.

Finally, the reported 43% increase in year over year global net product revenue growth of <unk> Bari withheld back seven percentage points due to unfavorable movements in foreign exchange rates.

Moving now to our second ultra rare disease product ox Laval.

We achieved $16 million in global net product revenues in the third quarter, representing a 10% increase compared with the second quarter.

At the end of Q3 over 230 patients who are on commercial ochsner treatment worldwide up from over 200 at the end.

End of the second quarter, representing 15% quarterly patient growth.

In the U S sales of <unk> decreased 10% versus the second quarter as growth in patients on therapy was more than offset by decreased average patient utilization during the quarter driven by fewer patients on the monthly loading those portion of great initial treatment.

In our international business sales.

Increased 29% compared with the second quarter due to an increase in patients on therapy.

And higher net realized price during the quarter, partially offset by FX headwinds.

Additionally, as with our powertrain give laurie changes in foreign exchange rates also negatively impacted normal Q3 dollars 22 results with reported year over year growth of 10% held back by 10 percentage points due to the strengthening U S dollar.

In conclusion, we're pleased with the growth in revenues and patient demand achieved in Q3, and particularly with early signs of strong performance associated with our withdrawal launch, which we believe represents an important therapy option for <unk>.

<unk> omni doses patients with Polyneuropathy.

That accelerated growth opportunity for our TTS franchise.

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

Thanks, Tom and good morning, everyone.

I'll start with our efforts in <unk> amyloidosis, where we are advancing two clinical stage product candidates.

<unk> and <unk>.

<unk> is currently approved in multiple markets around the world to treat the polyneuropathy associated with hereditary <unk> amyloidosis.

Commitment to expanding the product label for the treatment of cardiomyopathy in both.

Free and wild type <unk> patients.

But the more but <unk> demonstrated an acceptable tolerability profile and we're very encouraged by the overall safety profile of <unk>.

Collectively we believe these efficacy results validate the therapeutic hypothesis that R&R immediately balancing of GTR has the potential to result in a disease modifying impact on cardiac manifestations of <unk> doses.

With these results in hand, we remain on track to submit a supplemental NDA filing for review by the FDA by the end of this year with the goal of obtaining regulatory approval in the U S.

This medicine available to patients with <unk> amyloidosis cardiomyopathy as rapidly as possible.

We're also advancing <unk>, which is delivered by quarterly subcutaneous injection of <unk> to treat the polyneuropathy of <unk> doses.

<unk> was approved based on results from the Helios a phase III study.

That study includes an ongoing randomized treatment extension, where we're evaluating our biannual dose regimen and we are on track to share results in late 2022.

As it on pattern here too we are committed to expanding the label to include the treatment of cardiomyopathy inherent tree and wild type <unk> patients.

This is being accomplished with the Helios B phase III study of investigation with <unk>.

And USB, which is fully enrolled has an endpoint of all cause mortality and CV events assessed after at least 30 and up to 36 months.

We're on track to share top line results in early 2024.

As was announced this morning after careful consideration we have made the decision not to conduct the optional interim analysis for Helios B.

Several factors drove us to this decision including.

To our desire to conclude Helios b with the strongest possible dataset, including outcomes data and three further enhancement in confidence in Helios B with the recently positive Apollo B readout.

As such we look forward to the Helios B readout asset yield early in 2024.

We're continuing to consider options for the best path forward and start without disease as we recognize the significant unmet medical need in this patient population.

Lastly, with regard to have GTR franchise, we're excited to announce that a clinical trial application has been filed for <unk> for the first clinical program from our carrier platform, which aims to achieve an annual dosing regimen with highly potent and reversible effects.

We expect to initiate a phase one study in healthy volunteers by year end.

In addition to our late stage clinical programs. We believe we have also been making great progress with our early and mid stage programs.

Notable highlights this quarter was our announcement of positive results from our phase two study of <unk>, an investigational <unk> therapeutic targeting <unk> component of the complement pathway, which is in development in collaboration with Regeneron for the treatment of Iga nephropathy awry Gan.

In the study at week 32 treatments in this round resulted in a 37% mean reduction baseline in the 24 hour urine protein to cramp in ratio relative to placebo.

Primary readout the study in an important prognostic marker of disease progression.

The results of secondary endpoints were also consistent with the therapeutic benefit of some of this ramp Eni gap.

There were no significant drug related safety signals and we believe these collective efficacy and safety data support continued clinical development of them disrupt monotherapy in patients with IGN.

We look forward to gaining alignment with regeneron to finalize plans for phase III and sharing those updates with you in due course.

Another exciting highlight recently with the announcement of the preliminary phase one data supporting the clinical advancement of <unk>, an investigational <unk> therapeutic targeting HSV <unk> in development for the treatment of nonalcoholic <unk> hepatitis or Nash after single dose evaluation in healthy adult volunteers on a multiple doses.

Well in HST are being studied in adult patients with Nash are big crisis in the first two poppy cohorts, namely 200, 400 milligrams quarterly have completed at least six months on the study the remaining cohorts, we're exploring a lower dose or later biopsy time point.

In the first two part B cohorts and in HST was associated with robust target knockdown and numerically lower liver enzymes and biopsy derived nonalcoholic fatty liver disease activity score over six months in patients receiving <unk> and HFC relative to placebo.

The study was not powered to achieve statistical significance on these endpoint and the primary outcome measure is frequency of adverse events.

<unk> has exhibited some encouraging safety and Tolerability profile to date based on these results we plan to initiate a phase II study in adult patients with Nash in late 2022 in collaboration with Regeneron.

Now turning to our <unk> program, which is in development for the treatment of Alzheimer's disease and cerebral amyloid angiopathy.

As we announced in our press release. This morning, we now expect initial clinical data from this study in early 2023, a slight change from our previous guidance of late 'twenty two.

This change reflects the continued progression through dose escalation cohorts at a measured pace and the rigor with which we are screening for appropriate patients for this study that meet all necessary inclusion exclusion criteria.

Believe these initial clinical data with <unk>, if positive will be an important milestone for our CNS platform to show the R&R.

Chief clinically relevant degrees of target knockdown in the CNS with a safety and dosing profile that supports further development and we look forward to reporting topline results from this study in early 2023.

Representative of the productivity of our engine, we look forward to two further Cta R&D filings for the first in human studies before year end, namely <unk> for the treatment of metabolic liver disease, including diabetes, which is in our wholly owned program.

<unk> for Nash in collaboration with Regeneron.

We will be discussing all of this in greater detail at our upcoming R&D day being held virtually on December 15th and I encourage you to tune in.

With that let me now turn it over to Jeff to review, our financial results and upcoming milestones Jeff.

Thanks, Akshay and good morning, everyone I am pleased to be presenting a summary of the islands Q3, 2022 financial results and an update on our full year guidance.

Starting with a summary of our P&L results for Q3 2022.

Total product revenues for the quarter were $232 million or 39% growth versus Q3 2021.

Tribute to an increase in Reimbursable activities under our research services arrangement. In addition to an increase in revenue recognized associated with partnership clinical trial activities.

Our non-GAAP R&D expenses increased 6% in the third quarter 2022 compared to the same period in 2021, primarily due to increased compensation related expenses. As a result of increased increased employee head count and an increase in development expenses, primarily associated with the ramp up in enrollment in the cardio <unk> and <unk>.

<unk> phase III studies.

<unk> offset by decreased clinical batches manufactured during the quarter.

Purchase a capped call transactions and offering expenses.

Continue to believe our current cash balance is sufficient to bridge us to a self sustainable financial profile.

Now I'd like to turn to our full year 2022 financial guidance.

Starting with net product revenues, we continue to anticipate combined net product revenues for our core commercialized products will be between $870 and $930 million. Additionally, given the continued strengthening of the U S. Dollar since we issued our current guidance in April we are currently trending towards the lower half of our $870 to nine.

$130 million guidance range.

And our guidance for combined non-GAAP , R&D and SG&A expenses remains unchanged and is a range between $1 $390 million and $1 billion and $450 million.

Let me now turn from financials and discuss some key goals and upcoming milestones on back through the end of 2022.

We will continue executing on our global commercialization of on Petro and Blue truck give Laurie <unk>.

Next our TCR franchise will have important updates.

With <unk>, we plan to submit the NDA for review by the FDA for the treatment of patients with <unk> amyloidosis with cardiomyopathy.

With <unk>, we plan to report results on a biannual dose regimen from Helios, a and <unk> amyloidosis patients with Polyneuropathy.

Lastly for <unk>, we are on track to initiate a phase one study in healthy volunteers.

And our mid and early stage portfolio. We are looking forward to milestones that include completion of enrollment in the cardio <unk> phase II study in Dolby surround at or around year end.

Preliminary results expected from healthy volunteers in the phase one study of <unk> in development for patients with gout and.

And submission of Cta filings for <unk> for the treatment of metabolic liver disease, including diabetes and align Pnp for Nash.

Let me now turn it back to Christine to coordinate our Q&A session Christine.

Thank you Jeff Operator, we will now open the call for your questions today than we would like to ask you to limit yourself to one question each and then get back in the queue.

Additional questions.

Thank you again, ladies and gentlemen, I'd like to ask a question. Please press star one on your Touchtone telephone.

Again to ask a question. Please press star one line.

Our first question comes from Maury Raycroft of Jefferies. Your line is open.

Hi, good morning, and thanks for taking my question.

I was wondering if you can talk about whether you've got any feedback from FDA on Apollo data and what the NDA package would contain and also if you could talk about confidence around your primary end point P value and whether FDA has provided any specific comments on that.

Thanks for that question, Marc I mean, clearly we're delighted with the results from our Apollo B study, where we deliver clinically meaningful.

Significant benefits across there.

Minute walk test case.

<unk>, which are important measures actually for patients in terms of how they function and feel and we got good safety and new medical reduction that so we're very confident with the package of data that we have supporting the syndication holistically, particularly given the small size and on short duration of the study.

Akshay perhaps.

In.

Our regulatory pre.

Progress.

Yeah. So thanks, Maury and frankly, a lot I mean, I think you addressed with a high level. The key positives that came out of the study that.

Greenfield.

R&R therapeutics can have a significant impact we believe in <unk> amyloidosis, including in the cardiomyopathy.

We are proceeding to file the supplemental NDA by the end of year as we've said we're confident in this package.

<unk> gone outlined.

As the fall goes under review.

The idea was get the safety update 180 day safety update during review so we'll be providing that of course, and we will be very responsive to any questions. They have.

And so that's about as much as I can say at this point in time, but we've shown the data across two meetings at IFA in Hff's.

At extremely strong and positive response is strong.

Investigators kols and others and so we're excited focus F&B.

So moving forward expeditiously. Thank you Mary next question.

Thank you.

Our next question comes from solving Richter.

Line is open.

So that is helping me USB and from did the outcomes analysis of Apollo B impact your thinking on treated or placebo separation herein and how much time do you safe. Thank you.

Actually I think Thats fair.

So can you.

Repeat the full question I think something about the outcomes analysis, but the lines are operating and we didn't quite catch the second half of your question could you repeat that for us sure.

Sure could.

Could you explain the factors you considered when deciding not to conduct the Helios B interim I'm, just wondering whether the outcomes analysis of Apollo B impact your thinking on treated placebo separation and how much time, you're safe here.

Between the questions around the thinking behind not.

Progressing the interim analysis, yes.

I mean as you as you intimate the positive data out of the portfolio be significantly increase our confidence and belief that the design and conduct the <unk>.

<unk> is the right test of the hypothesis.

Arne can help patients with <unk> cardiomyopathy. So we feel quite buttoned up is looking at all the detailed data most of which we've shared already international meetings, but there are others.

And so that was one major factor the second was.

We obviously want to have the best data associated with <unk> to make it a best in class product.

<unk> for patients with <unk> cardiomyopathy.

The best way to do that is to preserve alpha and not mess around with the statistics, which obviously if youre doing the interim.

Downstream consequence on how on both the statistics for the final.

Readout in early 'twenty four.

And thirdly is just the speed at which everything is new which we're delighted by that.

The fact that Helios b.

Actually over enrolled of speed that it did means is such a narrow timeframe between any putative interim at any potential approval thereof.

As the full readout in early 2004, and hopefully getting a great label in serving patients with <unk> cardiomyopathy.

That's great. Thanks, Jay Thank you very much my question.

Thank you one moment please.

Our next question comes from Praveen Ahmad of Bank of America. Your line is open.

Hi, Good morning, guys. Thanks, so much for taking my question.

Maybe can you just clarify whether your team has already met with FDA from your pre NDA meeting regarding <unk>.

Paula Deen results, yet or is that still to come and then secondly, do you have any expectations about whether or not an AD com would be needed to review. This filing. It is the second indication proposed aren't already transcribed I just wanted to get your latest thoughts on that thanks.

Yes, Thanks, Athena I mean as you know we don't we don't comment on regulatory interactions I think.

Akshay covered this in another question, we're very confident with the package that we have supporting the syndication and we're moving forward.

As planned.

With a submission later.

Later this year.

That's about all I can say.

On the topic, but thanks for the question.

Thank you.

Our next question comes from Eliana Merle of UBS. Your line is open.

Okay.

Hey, Thanks, so much for taking the question just another one in terms of thinking about Helios b.

<unk> uptake in cardiomyopathy could you help us understand how you interpret the data and <unk> combination and what this could potentially mean in terms of uptake and state Department has progressed, our combination and the railroad commercially any initial feedback you are hearing initially I know it's early but.

Just curious your thoughts.

Right. So I think maybe kind of two parts of the question I think one I'd say it may be start off with you.

With respect us around.

In a data in combination with <unk>.

Let me talk against some perspectives on how you see things in the commercial.

Okay.

Thank you Kevin.

Vis vis your question Liana.

So it's a fabulous subgroup concomitant treatment.

Just to step back.

<unk> got a large robust study that considered the real world population out there. The majority of them don't have any drug right now about 75% of the study was on placebo had no background therapy.

25% of patients had background <unk>.

<unk>.

I refer you to our Hff's say output in both Maryland is very eloquent discussion of fly.

It's a little bit of a fool's errand just to use a face to cut into small subgroups.

Let's see.

That really are not powered but we will not designed.

We fool ourselves.

And again, though Tamara explained in physical stores is very well on the HFF side.

So.

Neutral.

Readout of the study I think was robust and excellent impacting patients.

<unk> solid TV, the six minute walk distance and the quality of life lottery.

Lots of internal consistency of the data. So this is a strong package.

We're delighted with that I appreciate the interest subgroups, that's really know what the study was powered to readout.

Yes, I would just generally is.

It's around two.

Commercial.

Just to build on Akshay is point it would be premature for us to comment anything specific on without the label, but what we can tell you is if you just look at the numbers since the launch of the firm. It is the diagnosis rate went up by 10% 10 times and we certainly anticipate that to continue this is a highly devastating disease across the USA.

Across the USA.

Ex U S. Theres about 250 to 300000 patients suffer from this devastating disease and what we know is those patients that are a lot of untreated patients and those patients who continue to progress on tough so we believe.

Our.

<unk> therapies, if theyre approved will be a.

Very attractive option, both for physicians and obviously for patients.

Thank you so perhaps.

Medical needs filled that these patients. Unfortunately next question. Please.

Thank you.

Next question comes from Ritu morale of Cowen Your line is open.

Good morning, guys. Thanks for taking the question.

Ivano Akshay should we be expecting priority review on the SMB.

Just given what you and your regulatory consultancy.

That need in <unk>.

TCR cardiomyopathy and can you just also remind us.

Then the last.

Interaction you've had with SBA.

Was.

In the past obviously not forward.

Forward looking.

If theres been any.

Significant turnover in the review team our senior members.

The review Division since then.

Thanks for the question about.

The regulatory.

<unk> say in terms of expectations.

Okay.

<unk>.

Another question really just asking us to comment on.

Our progress with regulatory interactions.

It's sort of a quick question, but maybe you can reiterate the point yes.

Thank <unk>. Thanks for the question as you know we've done this.

This goes back and forth with regulators I will reiterate what I said earlier that we're confident in this data package. We are on track to file the supplemental NDA by the end of the year and the priority review will that be.

The fda's decision and they have to evaluate.

The pros and cons of this therapy and the data package was submitted in.

<unk> will eagerly await that decision, but there's not much more I can enrich company comment.

Gentiva.

I think I think that's what the FDA.

Not really in a position.

To speak to that.

Kim.

Next question.

Thank you. Our next question comes from Jessica Fye of Jpmorgan. Your line is open.

Hey, guys. Good morning, Thanks for taking my question.

Did you have a pre NDA meeting yet or the on Petro cardiomyopathy filing or if not do you expect it to.

Yes. Thanks for the question I mean again I think there's lots of interest around this the most important thing is that.

Obviously, you have lots of back and forth with regulators across all our programs.

And let me just reiterate that we will follow the supplemental NDA by the MBA program.

Based on everything we've discussed by following this call and many other alright. Thanks for the question. Thanks. Mike next question. Please. Thank you. Our next question comes from the line of.

David Lebowitz of Citi. Your line is open.

Thank you very much for taking my question when.

When you look at the on Petro acutely ambulatory new patients start forms I noticed that the number of new patients.

Percentage uptick.

What was announced in the first quarter of launch I think lead umbrella third to nearly 50% could you comment on.

What the expectations for new patients be coming to market is and what that might.

How that might impact the growth of the overall franchise.

Relative to Petra would have done.

Without the presence of ample trial.

Yes, no thats a great question I mean, we're particularly pleased with the performance of our GTR franchise, especially the progress of bandwidth.

In the U S and we're seeing a great great dynamic.

With.

Approximately half of new patients in.

Our switches and I think you made the point about the GTR franchise, which I'd like to underscore I think we now really have the GTR franchise, and we expect to be able to.

Grow the pn market.

Encourage earlier treatment given the very attractive regimen of.

Ivanka Trump, but yes, it's all you want to add some color maybe how you're seeing the outlook for the GTR franchise.

Absolutely Yvonne looked early signs of U S. <unk> launch is a good testament to the old royalty TR franchise future.

Our TCR franchise grew by 30% versus last quarter.

Driven mainly by almost two thirds of this growth was really demands.

In our view early signs of performance really confirm cheeky beliefs. One is despite available treatments, there's a significant unmet needs and the role of a mitra.

Attractive product profile.

We will continue to increase growth that's going to be important and also we do.

A strong foundational capabilities that will build over time.

Customer facing capabilities.

That will allow us to continue expand this growth. So we're really excited about this the we're still scratching the surface in terms of diagnosis and treatment.

There are 10 to 15000 patients in the U S that suffer from this condition. We believe on with trust product profile will be a very nice fit to that treatment regimens.

Thanks Peter.

Next question please.

Our next question comes from the line of Gena Wang of Barclays. Your line is open.

Thank you for taking my questions.

Sorry, I'll ask Apollo B question again.

I think that because there are quite some questions were raised.

Profitability.

It's only a one study and then there is no outcome.

So maybe if you could refresh our memory the history of Apollo B study, so basically how steady with design.

And was it in line with the FDA.

Actually I think yes, clearly fee.

Thanks Gene.

I think we've discussed much of this on other.

Cold, but let me just reiterate Gina.

I think this is.

I'm, losing track a little bit faithful seven phase III study of <unk> phase III studies, we've negotiated with the FDA and other regulators around the world.

We will now work.

Is in collaboration with guidance from regulators.

We have.

For our nylon products improved based on the excellent set of dialogues and relationships. We have an understanding how regulators are trying to guide us. This product in this trial was no different from that and so if any credibility and confidence is derived from a product performance failure.

Likely to apply it here now in the specifics here when Apollo was done and we will remember the conversations those of you that were involved then.

The cardiac data the cardiac subgroup data from Apollo we are very promising.

And the FDA said, yes that is the case.

We've discussed that before and based on that they guided us that we should do affect this study in cardiomyopathy population.

And with that conversation in collaboration with designs that you've seen Apollo b.

Results of it and so this study was done in close collaboration with regulators and we're grateful for the guidance and we're very happy with the outcome. We're clearly we've shown the impact on patient functionality and quality of life, which are two cornerstones of the FDA.

Based on the approval of drugs safety looks very encouraging to us too.

And then finally on the mortality front.

You will recall the four patients died.

Placebo rather.

Okay.

Panel placebo, so that's encouraging although it's a small number. So this package. We believe is rather strong first trial that was designed in collaboration with regulators and <unk>.

We're looking forward to filing the NDA.

Thank you.

Thank you. Our next question comes from the line of polymer piece of Stifel. Your line is open.

Hey, thanks, so much.

I was.

Really interested in your comments in the press release and in the prepared remarks on drug positioning related to the inflation reduction act and kind of broader drug pricing risks.

To that point, you just filed a cta for our once annual GTR knockdown drug how do you think about positioning that and kind of balancing star guard versus this potentially just being a nextgen in GTR cardiomyopathy, and also enabling you to maintain premium pricing.

Beyond a decade in that space. Thanks, so much.

Yes, thank you for the.

The question I think.

So look the inflation reduction is a significant shift in Medicare and how the federal government regulate our industry.

Our support and that's the new part D patient out of pocket caps that we have considerable concern.

About a couple of other aspects of.

Legislation created the first is around.

Concerning the small molecule medicines.

Faith negotiate that Medicare reimbursement rates in the nine years after approval <unk> four monoclonal antibodies I think they present to us in the near term is that while the extensions of the arbitration reduction effects from Medicare price negotiation for a drug that has a single orphan drug indication that added.

Incentives for pursuing the approval of drugs and additional indications.

We're still digesting the legislation, but it points out in the meantime, despite the pause button for three phase III study in stock.

As we consider our options for the best possible I'd.

I'd like to just kind of notes at 331 is that we remain.

Excited actually by the opportunity for an RNA <unk> ctr lowering medicine as a potential therapy in patients with <unk> disease, and we think it's an important unmet medical need and the <unk>.

Second I think you touched on it when he talked about GTR CFO as a platform company, we really have the opportunity.

Continuously innovate and create optionality for how we think about developing.

<unk>.

Our medicines and I think <unk>.

<unk> CFO .

Another.

PTR lowering.

Program that that we're moving forward, which we're very excited about.

I think the other the other point that I'd like to make which I think is quite important just to remind everybody that <unk> strength.

Actually has orphan.

<unk>.

Disease designation for <unk> amyloidosis.

No equity or cardiomyopathy.

As with many other companies, we're reflecting on the implications of this relatively new legislation on how we think about our portfolio, but as a company. We actually think we're very well positioned given our given our platform and our ability to continuously innovate around.

Roundup metros.

And maybe next question.

Our next question comes from the line of Gary Nachman of BMO. Your line is open.

Hi, good morning.

If you end up getting the <unk> patch I'll be approval, let's say end of next year, but then youll be bumping up against the interest around Helios B data in early 'twenty four how much of an impact do you think that might have on your launch of on Petro and CN when physicians will be anticipating this other important dataset I know it's.

So a bit early thinking about the launches that given the timing of these that I was curious to get some of your early thoughts on that thanks.

I'll just start off by saying that the domains.

The reason why we progressed on patrol in the cardiomyopathy indication, let me just the level of unmet medical need for patients who continued to progress.

Despite debatable.

Really we took the opportunity to grow twice.

Twice during a near term submission.

Whilst we continue to develop.

The <unk> study.

And as you pointed out we will have those results shortly at the beginning of 2024, So we think fits.

In a way able to meet the needs of patients in the near term hopefully with the approval of patch on CN and then.

As we progressed ESP, bringing forward.

And even more attractive regimen.

For these patients.

Shortly afterwards, but may be told that you can comment on how we're thinking about.

The launch of <unk>.

With with the opportunity of bringing inventory strategy.

These patients in the in the medium term, yes, no I think you summarized perfectly Ivana all I would add is.

There is a big unmet meet unmet needs. There are patients who are currently on available treatments that are continuing to progress at pure point, we want to make an alternative treatment available.

Quickly as possible so we can actually.

Meet those needs and also continuing to build our capabilities for the larger indication. We're obviously very pleased with the capabilities that we built.

Yeah.

Based on based on what we have what we're actually able to demonstrate with our mutual Qian launch and as we continue to expand the indication. We believe are experienced with the <unk> is going to help us to have even a stronger launch with on road truck cm. If it's approved.

Thanks to all of that so great. So again to sort of teach our franchise approach.

We think it will be important.

In Ah patients in our commercial performance going forward.

Next question.

Our next question comes from Luca <unk> of RBC capital.

Sibley see functional data for that dataset. Thanks, so much.

Great. Thanks, Thank you.

A question on ATP look where we're clearly excited about the potential for.

RNA therapeutics and addressing CNS disease businesses. This is the first one out of the gate.

We're progressing very well, but as I say describe them as <unk>.

Introductory remark, but actually maybe you can you can speak to that.

Data that we have.

To answer the.

After the after the ongoing study the crucial data. So I think first and foremost safety. This is our first candidate in the central nervous system.

So keen interest the study is progressing well so we are very comfortable so far.

Even beyond that.

This leaves us lots of interest in the biomarker outlook. So we'll be looking for <unk> knockdown.

The fragrance various language per diluted frankly, both from AEP changes it knows that the CSF.

And.

No.

In addition to that there is going to be important PK because again the success, but we've injected that joke.

The interest equally so.

Those are the key data points in the short term that will be looking at and looking forward to reporting in early 'twenty three.

Absolutely.

As we move forward in <unk>, we were able to demonstrate.

Impact on patients with <unk>.

Early onset Alzheimer's disease course of opens up the opportunity for a whole host of them.

Devastating CNS disorders, so painful program for us.

Okay.

We're progressing well thank.

Thank you for the question.

Question. Please.

Thank you. Our next question comes from Joseph Stringer of Needham <unk> Company. Your line is open.

Hi, Good morning, Thanks for taking my question a question on <unk> start forms and the percentage.

Sourced from new prescribers.

This quarter you have around 17% that was similar to 20%.

Sure from your last update you sort of see this.

And a fairly stable in that 15% to 20% range going forward or how do you see that.

Going forward. Thank you.

Although thats clearly a question for you had these <unk> sensitive new prescribers going toward yes look I mean, I am glad you highlighted that we're really excited about sort of how we are displaying those strong foundational capabilities that we've been able to build from diagnosis all the way to access support.

To the prescriber base.

And as a result of that we also see patients are treated a bit younger assigned that patients are getting diagnosed and obviously theyre getting downwards treatments.

Another important metric that we also see.

We're obviously going to pay a lot of attention to this is our start forms to treatment rates.

Having a temporary J code, it's been similar to old patio and again, thanks to our good access support Vba's at our price parity strategy.

So because of those elements. We believe we're going to continue to see a good expansion of new prescribers and net new patients for <unk>, that's going to drive the GTR franchise.

To the next level.

Right.

Super. Thank you very much I think we got time for one more question.

Thank you. Our next question comes from Myles Minter from William Blair. Your line is open.

Hi, Thanks for squeezing me in just on absolute <unk> actually just with the recent FDA label expansion are you expecting like a significant amount of warehouse advance payouts on patients.

And I guess, how you're thinking about growing that franchise moving forward considering at book.

Flat over the past quarters.

Okay do you want <unk>, yes.

Look.

Can you just won is a devastating disease and we're very pleased to be able to actually provides.

Important treatments for these ultra.

Altra rare disease patients.

H C.

Been a great addition, we do see a good.

In addition to our data suite of data that we have we see a goods.

The group of patients both from pediatric and as adults.

Evel as well as various.

Progressive stage of their diseases eliminate C is simply another important element that will allow the prescribers to be able to prescribe this medicine.

To a broad range of patients that suffer from this ultra rare disease.

Look we've had a great quarter.

We really are baked into our financial performance and we're seeing strong growth in <unk>.

Patients on therapy across all of our questions.

So.

I'm, sorry, if I'm pleased with our performance.

So.

Thank you everyone for joining us on this call.

And a great progress in the third quarter of 2022 talks about our strong commercial results, but also we're pleased with how we are advancing a diverse pipeline programs that are in development.

A lot of excitement ahead.

Catalysts on deck in the coming months and we of course look forward to updating you along the way as we continue to deliver on our near and long term. So thank you everybody and have a great day.

Thank you ladies and gentlemen, this does conclude today's conference. Thank you all participating you may now disconnect have a great day.

Q3 2022 Alnylam Pharmaceuticals Inc Earnings Call

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