Q4 2021 United Therapeutics Corp Earnings Call

Martine Rothblatt: Carex has not impacted our traprosomal growth trajectory because patients... Physicians, payers, they all recognize our outstanding commitment and track record for customer service and supply chain reliability. Our continued double-digit percentage patient growth is how we get to our announced goal of 25,000 patients by 2025. And we remain right on target to meet that goal.

It's a profitable growth trajectory because patients physicians payers. They all recognize our outstanding commitment and track record for customer service and supply chain reliability.

Our continued double digit percentage patient growth is how we get to our announced goal of 25000 patients by 2025, and we remain right on target to make that call in.

Martine Rothblatt: In fact, we expect double-digit patient growth again this year and in 2022. The second real big picture item is that our organ manufacturing products made a dramatic and highly successful debut with one Tengene U-heart, two Tengene U-kidneys, and two U-thymokidneys transplanted. Of course, the focus of everybody is on our living UHeart recipient, Mr. David Bennett, Sr., so I'm very pleased to report that he is doing well at almost two months post-transplant. Specifically, his cardiovascular function at seven weeks is excellent, and there's no sign of rejection, even after a 30-day postoperative biopsy. But he has a lot of serious non-heart health challenges. He said last night...

In fact, we expect double digit patient growth again this year in 2022.

The second real Big picture item is that our Oregon manufacturing products made a dramatic and highly successful debut with 110 gene you hard to 10 gene you kidneys and to you Simon kidneys transplanted.

Of course, the focus of everybody is on our living you heart recipient Mr. David Bennett senior.

So I am very pleased to report that he is doing well at almost two months post transplant.

Specifically as cardiovascular function at seven weeks is excellent and there is no sign of rejection, even after a 30 day post operative biopsy.

Although we have a lot of non heart.

Various health challenges.

He said last night.

Martine Rothblatt: Thank you for this chance to live. And my response to Mr. Bennett was, thank you, Mr. Bennett, for giving us this chance to let countless thousands of others also live. I believe our clear-cut success is in 2021 with Xenoproducts. Coupled with our core competencies in this area, our GAL-SAFE-MEET, for example, was approved by the FDA in December 2020. And our core competencies in GMP manufacturing and product development all show that our organ manufacturing pipeline is now more credible than ever and of truly momentous potential.

Thank you for this chance to live.

And.

My response to Mr. Ben It was thank you Mr. Bennett for giving us this chance to let countless thousands of others also live.

Yeah.

I believe our clear cut successes in 2021 with Zeno products.

Coupled with our core competencies in this area are Gal a safe meet for example was approved by the FDA in December 2020.

And our core competencies and GMP manufacturing and product development all showed that our Oregon manufacturing pipeline is now more credible than ever.

And of truly momentous potential.

Okay.

Martine Rothblatt: Now, beyond these big picture headlines, there's been a great deal of progress in advancing our pharmaceutical pipeline as well. Some highlights are... First, our once-daily treatment for PAH, Relenopeg, is progressing nicely toward wrapping up its phase three enrollment. Second, our Phase 3 PERFECT trial, which is testing a first-ever treatment for COPD-related pulmonary hypertension, is steadily racking up more patient randomizations month by month toward completion of its Phase 3 trial. And our revolutionary disease-modifying effort against idiopathic pulmonary fibrosis, or IPF, which we call the Teton Trial, is doing so well in its U.S.

Now beyond these big picture headlines there has been a great deal of progress in advancing our pharmaceutical pipeline as well.

Some highlights are.

First our once daily treatment for ph for Atlanta, Peg is progressing nicely toward wrapping up its phase III enrollment.

Second our phase III perfect trial.

Which is testing a first ever treatment for COPD related pulmonary hypertension is steadily racking up more patient randomization month by month toward completion of its phase III trial.

Yeah.

Our revolutionary disease, modifying effort of idiopathic pulmonary fibrosis or I P F.

Which we call. The Teton trial is doing so well in its U S phase III enrollment that we opened up a ton two phase III trial outside the U S. So that we could hopefully offer this product worldwide.

Martine Rothblatt: Enrollment in phase III enrollment so that we opened up a Teton II Phase III trial outside the U.S. so that we could hopefully offer this product worldwide. Our super-convenient PiVaso DPI product remains well-positioned for its summer 2022 launch. The FDA did just delay its approval decision from this month to May. But that three-month detour is not important compared to the product's blockbuster revenue potential and patent life well into the 2030s. Three months delay to start on a billion dollar revenue trajectory is no big deal.

Our super convenient tie VSO DPI product remains well positioned for summer 2022 launch.

The FDA did just delay its approval decision from this month to me.

But that three months detour is not important compare it to the product's blockbuster revenue potential and patent life well into the 2000 <unk>.

Three months delay to start on the $1 billion revenue trajectory is no big deal.

Mike will now brief you on our commercial progress.

Martine Rothblatt: Mike will now brief you on our commercial progress. But, in summary, for 2021, I can say we hit our patient growth and revenue growth targets, we validated three new XenoOrgan product candidates, and in so doing, we launched a whole new category of biotechnology.

But in summary for 2021 I can say.

We hit our patient growth and revenue growth targets.

We validated three new vino, Oregon product candidates.

And in so doing we've launched a whole new category of biotechnology.

Martine Rothblatt: We have seven phase three trials underway with multiple new product candidates, and most important of all, in this past year, I'd like to say we saved the life of Mr. Bennett with a 10-gene xenoheart. We saved the lives of at least 100 children stricken with neuroblastoma thanks to our unitoxin medicine. We have now saved over 200 lives of lung transplant patients by increasing the usable donor lung supply with our lung bioengineering technology.

We have seven phase III trials underway with multiple new product candidates.

And.

Most important of all in this past year.

I'd like to say, we save the life of Mr. Bennett with a <unk> heart.

Save the life the lives sorry, we save the lives of at least 100 children stricken with neuroblastoma.

Two our unit tucks in medicine.

We have now saved over 200 lives of lung transplant patients by increasing the usable donor lung supply with our lung bioengineering technology.

And we have saved or improved the lives of thousands of pulmonary hypertension patients in the past year with our remodeling remuneration <unk> and <unk> products.

Martine Rothblatt: And we have saved or improved the lives of thousands of pulmonary hypertension patients in the past year with our Remodulin, Remunity, Tyvaso, and Orenatram products, from the headline saving of one life to the kind of like prayers granted saving of a hundred children's lives to the now almost routine saving and improving of thousands of lives. I can most confidently report that UT's first year as a public benefit company has been super true to our public benefit purpose. Mike, can you lead us off now with commercial operations? Sure. Thanks, Martine. And good morning, everybody.

Yeah.

The headline saving of one life.

To the kind of like prayers granted saving of 100 children's lives.

Two the now almost routine saving and improving.

Since of lives.

I can most constantly report that <unk> first year as a public benefit company has been super true to our public benefit purpose.

Mike can you lead us off now with commercial operations.

Michael Benkowitz: As Martine said, we're very pleased to have posted double-digit revenue and patient growth for the full year of 2021, ending the year with more than 10,000 patients on one of our traprosonal therapy. This quarter, I'd like to dive into the underlying performance of our three terposterone products, Pivaso, Remodulin, and Orenatran. As usual, I'm going to focus more on our patient metrics, and I'll remind everyone that our quarterly troposynol revenue does not always exactly track exactly with quarterly underlying patient demand due to our specialty pharmacy partner ordering pattern. So I'll begin with Haraj.

Sure Thanks, Martin and good morning, everybody.

As Martin said, we're very pleased to have posted double digit revenue and patient growth for the full year of 2021, ending the year with more than 10000 patients on one of our cross sell therapies.

This quarter I'd like to dive into the underlying performance of our three to cross sell products.

So remodeling at Atlanta trend.

As usual I'm going to focus more on our patient metrics and I'll remind everyone that our quarterly to hospital revenue does not always track exactly with quarterly underlying patient demand due to our specialty pharmacy partner ordering patterns.

I'll begin with high ratio as you may recall in early 2021, we established a goal to double the number of patients on <unk> for approximately 3000, when the ph ILD indication was added to the label to 6000 by the end of 2022.

Michael Benkowitz: As you may recall, in early 2021, we established a goal to double the number of patients on Tybaso by approximately 3,000 when the PHILV indication was added to the label, to 6,000 by the end of 2022. This assumes no COVID-related impact to physician access and patient initiation of therapy and importantly recognizes that our path to doubling may not be linear. The fourth quarter was our third quarter of the PHILD launch.

Assumes no COVID-19 related impact to.

Physician access and patient initiation of therapy, and importantly recognizes that our path to.

Doubling may not be linear.

The fourth quarter was our third quarter of the ph ILD launch and then this quarter, we had the highest number of days, our referrals, which is what we call prescriptions since launch and we added close to 300 patients to archiving so active patient census.

Michael Benkowitz: And in this quarter, we had the highest number of Tyvaso referrals, which is what we call prescriptions, since launch. And we added close to 300 patients to our Tyvaso active patient census. However, our new patient starts were less than Q3, which is common in the fourth quarter, as many patients opt to wait until after the holidays to begin advanced therapy.

Our new patient starts were less in Q3, which is common in the fourth quarter as many patients opt to wait until after the holidays to begin advanced therapies, our specialty pharmacy partners are working to clear this backlog and get these patients started on therapy during the current quarter.

Michael Benkowitz: Our specialty pharmacy partners are working to clear this backlog and get these patients started on therapy during the current quarter. We continue to anticipate a CMS coverage decision regarding Medicare reimbursement for Tradeso and PHILD anytime between now and May of this year. All the necessary steps have been completed, including the close of a public comment period last year.

We continue to anticipate a CMS coverage decision regarding Medicare reimbursement for DSO in ph ILD anytime between now and May of this year.

All of the necessary steps have been completed including the close of a public comment period last year.

Michael Benkowitz: We can't predict the timing of this coverage decision, but in the meantime, federal health care patients still have the opportunity to apply to our patient assistance program to access Tibasin therapy prior to the coverage decision if they are eligible. We anticipate three core drivers for continued type A servers over the next several quarters. I think I mentioned these on the last call.

We can't predict the timing of this coverage decision, but in the meantime, federal health care patients.

Still have the opportunity to apply to our patient assistance program to access high base of therapy prior to the coverage decision if they are eligible.

We anticipate three core drivers for continued <unk> growth over the next work.

Several quarters I think I mentioned this on the last call.

Michael Benkowitz: First, we think the CMS coverage decision and PHILD will be a driver for tribasal growth through the middle of this year. Even though federal health care patients may apply for our patient assistance program, we are hearing through the channel that many physicians are simply waiting for CMS coverage before prescribing Tybaso for their PHILD patients. Second, we continue to grow the tribasome prescriber base, particularly among ILD treaters, and expect to see continued growth of prescriptions from these physicians over the next two quarters and beyond as we continue our outreach and educational efforts in this space. Up until now, the vast majority of Tybasia prescribing has come from historical PAH.

We think the CMS coverage decision in ph ILD will be a driver for <unk> growth through the middle of this year.

The federal health care patients may apply for our patient assistance program. We are carrying through the channel that many physicians are simply waiting for CMS coverage before prescribing tie ratio and our ph ILD patients.

Second we continue to grow the base of our prescriber base, particularly among ILD treaters and expect to see continued growth in prescriptions from these positions over the next few quarters and beyond as we continue our outreach and educational efforts in this space.

Up until now the vast majority of <unk> prescribing has come from historical PAA trial.

Michael Benkowitz: So there is a lot of as yet untapped potential in this area. And finally, we view the upcoming Tyvaso DPI approval as an additional catalyst for Tyvaso growth. While we would have been thrilled with an FDA approval for Tyvaso DPI this week, as we said in our Q3 call, we've been assuming a launch by the summer of this year, so nothing's really changed in that regard. In the meantime, we are completing our launch preparations, and we will be ready to launch immediately upon approval. Moving to Oranatram, we saw yet another quarter of record patient counts in the fourth quarter.

So there is a lot of as yet untapped potential in this area.

And finally, we view the upcoming Friday, so DPI approval as an additional catalyst to five ACO growth, while we would have been thrilled with FDA approval for traditional DPI. This week as we said on our Q3 call. We've been assuming that locked by summer of this year. So nothing has really changed in that regard in the meantime, we are completing our launch preparations and we will be ready.

The launch immediately upon approval.

Moving to <unk>, we saw yet another quarter of record patient counts during the fourth quarter as we've discussed before we believe this uptick is driven by the freedom EV label expansion now that we're able to have more robust interactions with prescribers about the data.

Michael Benkowitz: As we've discussed before, we believe this uptick is driven by the FreedomEV label expansion now that we are able to have more robust interactions with prescribers about the data. And finally, we're pleased with the continued strong performance of remodulin despite the launch of a generic form of subcutaneous troprosinol last year. That generic launch has continued to play out closely with what we saw with the IV generic launch in 2019, with an initial bolus of patients, primarily those dual eligible Medicare and Medicaid patients, transitioning to the generic troprosinol, trickling down to an immaterial number of generic transitions in recent months.

And finally, we're pleased with the continued strong performance of our module and despite the launch of a generic form of subcutaneous <unk> in the last year.

That generic launch has continued to play out closely with what we saw with the IV generic launch in 2019 with an initial bolus of patients primarily those dual eligible Medicare Medicaid patients transitioning to the generic coprostanol trickling down to an immaterial number of generic transitions in recent months.

The feedback we've received autoimmunity pop has been quite positive that our early launch efforts as part of that feedback we learned at the pumps alarms and in some cases be overly sensitive.

Michael Benkowitz: The feedback we've received on our immunity pump has been quite positive in our early launch efforts. As part of that feedback, we learned that the pump's alarms can, in some cases, be overly sensitive. To be clear, the issues we're seeing with the alarms are not safety issues.

To be clear the issues, we're seeing with the alarms are not safety issues.

Michael Benkowitz: But to ensure that physicians and patients have an awesome experience with the pump, we recently paused the introduction of new patients to Immunity while we work with our partner, DECA, to optimize the alarm profile. We just completed final testing of the new alarm system. Everything is working as expected, and we will recommence our immunity rollout next week. To wrap up, we're pleased with the progress. And with Tyvaso and PHILD, we look forward to a CMS coverage decision in May or earlier.

But to ensure that physicians and patients have an awesome experience with a pump. We recently paused the introduction of new patients truly entity, while we work with our partner Deca to optimize the alarm profile.

We just completed final testing of the new alarm system everything is working as expected and we will recommence our community rollout next week.

To wrap up we're pleased with the progress.

And so in ph ILD, we look forward to CMS coverage decision in May our earlier.

Michael Benkowitz: Likewise, we're on track to achieve our 6,000 patients on Tyvaso by the end of the year, and we're pleased with the continued momentum for Renitran. And finally, we think that our remodeling revenues will remain robust despite generic competition, especially once we recommend... The introduction of new patients to the Rheumatic Pump

Why is we're on track to achieve our 6000 patients on <unk> by the end of the year and we're pleased with the continued build out for <unk>.

Finally, we think that our modular on revenues will remain robust despite generic competition, especially once we recommence.

The introduction of new patients to the remuneration policy.

Martine Rothblatt: And with that, I'll turn the call back over to Martine. Thanks so much, Mike. I'm really excited about the Remunity breakout next week.

And with that I'll turn the call back over to Mark King.

Thanks, so much Mike Super exciting about the immunity.

Breakout next week awesome.

Operator: Operator, I understand that you probably have some people in queue, and feel free to bring them on. Thank you. At this time, I would like to remind everyone that in order to ask a question, press star and the number one on your telephone keypad. Your first question today comes from the line of Joseph Thome with Cowan & Co. Your line is now open.

So operator, I understand that you probably have some people in queue and feel free to.

Bring them on.

Thank you at this time I would like to remind everyone in order to ask a question Press Star then the number one on your telephone keypad.

Your first question today comes from the line of James with Cowen Inc. Cowen and co. Your line is now open.

Martine Rothblatt: Good morning, thank you for taking my question and congratulations on the progress. Maybe just on the Tybazo DPI request by the FDA, are you able to provide a little bit more information as to the type of information that you submitted, and then in terms of the safety profile you've seen with Tybazo DPI to date, some high-level thoughts there, especially as it relates to the potential for bronchospasm? Thank you. Yeah, thanks, Joe. We've really had some, you know, absolutely great experiences with the Tyveso DPI to date, and we have not experienced those, those bronchospasm side effects that you referred to.

Hey, good morning. Thank you for taking my question and congrats on the progress maybe just on the timing of the DPI request by the FDA are you able to provide a little bit more information as to the type of information that you submitted and then in terms of the safety profile of <unk> to date.

High level thoughts, there, especially as it relates to potential for bunkers.

Thank you.

Yes, Thanks, Joe.

We've really had some absolutely great experience with the Taipei, So DPI to date and we have not experienced those.

Banco spasm.

Side effects.

That you referred to.

Martine Rothblatt: So we really don't believe that that's going to be an issue at all. Then, There is some more information in the press release. If you look down toward the second or so page of the press release, there will be a little bit more information there about the submission and what was submitted and not submitted. So I think it's all really quite routine, and the FDA is doing their job as best that they can.

So we really don't believe that that's going to be an issue at all.

The.

[noise].

The there is some more information in the press release, if you look down toward the second or so page of the press release, you see a little bit more information there about the submission and will be submitted not submitted.

So I think it's all really quite routine and the FDA doing their job as best that they can as I mentioned in my introductory remarks.

Martine Rothblatt: As I mentioned in my introductory remarks, and as Mike emphasized in his remarks, three months is really meaningless in the context of this product's life. This product has a very long way to go. It is an amazing product. I mean, it's so convenient and easy for patients to use. We have the great results of the BREEZE trial. It's going to end up being used, I'm confident, by thousands and thousands, and over the course of its life, like, 10,000 more or less patients. It's just an amazing product.

As Mike emphasized in his remarks three.

Three months is really meaningless in the context of this product slides. This product has a very long patent life. It is an amazing product I mean, it's so convenient and easy for patients to use.

We had the great results of the Breeze trial, it's going to end up being used on <unk>.

<unk> bye bye, thousands and thousands and in over the course of its life.

10000, more or more patients just an amazing product.

It will get out there everybody just has to make sure that like all the boxes are checked and the ISO dotted and the t's are crossed and.

As Mike said, we feel confident with the launch by the time frames that we've <unk>.

Previously guided everyone to.

Alright, Joe Thanks for the question and operator, you can bring up the next one.

Martine Rothblatt: So it'll get out there, you know; everybody just has to make sure that all the boxes are checked and the I's are dotted and the T's are crossed. And as Mike said, we feel confident with the launch within the time frame that we've previously guided everyone to. All right, Joe, thanks for the question, and operator, you can bring up the next one. Your next question today comes from the line of Eun Yang with Jeffreys. Your line is now open. Thank you. Another question on DPI.

Your next question today comes from the line of Jim Yang with Jefferies. Your line is now open.

Thank you another question on BPI.

It's approved.

Does it need to go with.

As soon as the approval process.

<unk> ph ILD indication for Medicare coverage.

Martine Rothblatt: Once it's approved, does it need to go through a CMS approval process similar to the PHR indication for Medicare coverage? Oh, Barry, thank you. It's so nice to hear your voice this morning. You know, we have a lot of experts on our call, but I think Mike Benkowitz would be the best person to respond to your question. Mike, if that's okay.

Barry Thank you Yung from nice to hear your voice this morning.

I think we have a lot of experts and I call. It, but I think Mike bank of which would be the best person to respond to your question, Mike If thats okay.

Michael Benkowitz: Sure, I'm happy to take that question. So there is a CMS approval process that will need to happen, a review process that will need to happen with Tyvaso DPI, but it's different than what we're experiencing with PHILD. So, for PHILD, I'm sorry, yes, for PHILD.

Sure I'm happy to happy to take that question.

So there is a CMS approval process that will need to happen.

Of your process that will need to happen with Televisa DPI, but it's different than what we're experiencing with ph ILD. So for ph ILD I'm, sorry, it yet for <unk>.

Michael Benkowitz: So, you know, right now PHILD is approved for use with our TD300 nebulizer, and it's covered in the medical benefit or Part B side of Medicare. The DPI will be a pharmacy benefit product, so it will be covered in Part D. So, the process is a little different. So, while Medicare is reviewing the data, reviewing the product for coverage, patients can still access federal healthcare. The information needs to be confirmed with the Therapist's affidavit, held up with the DPI.

So.

Right now Pete ph ILD is approved for use with our TD 300 <unk>.

And it's covered in the medical benefit or part D side, because boy side of Medicare.

The DPI will be a pharmacy benefit product so covered in part D. So the process is a little different so so so while Medicare.

Is reviewing.

Reviewing the data reviewing the product.

For coverage.

Patients can still access federal health care patients can still access the DPI. The physician simply has to go through an appeal process.

To doing this.

To do this often with products that are on Medicare.

Formulary and it's just an extra step they have to go through and then it gets approved so we won't see this we won't see the same issue, we're seeing with ph ILD, where we have this.

[noise] holdup.

With the BPI those those will continue through with doctors like I said doing this at the additional step of appealing.

Michael Benkowitz: They will continue to go through with doctors, like I said, doing this additional step of appealing. Perfect. Thank you so much, Mike. Operator, who would be our next question? Your next question today comes from the line of Jessica Fye with KPMorgan. Your line is now open. Hey there, good morning.

Perfect. Thank you so much Mike.

Operator, who would be our next questioner.

Our next question today comes from the line of Jessica Fye with Jpmorgan. Your line is now open.

Martine Rothblatt: Thanks for taking my question. I'm curious what proportion of Tybaso patients are currently on free drugs and what are the next clinical or manufacturing milestones we should look out for in the organ transplant business? And what results, in your mind, would justify approving the potential investment in the first GMP or GMP-like manufacturing facility for those products?

Hi, there good morning, Thanks for taking my question.

What proportion of patients are currently on free drug and what are the next clinical manufacturing milestones, we should look at where in the organ transplant business.

Results in your mind would justify an eating.

The potential investment in the fresh <unk> light manufacturing facilities for those products.

Creek shaft, so happy to hear you on the call we think of Hubris link the two.

Martine Rothblatt: Great, Jeff. So, happy to hear you on the call. We think of you as like one of the KOLs, Pharmaceutical Analyst, so it's always exciting to hear your voice here. I think the first part of your question is probably best answered by Mike, and then Mike, if you bounce it back over to me, I'll then talk about the Xeno aspects of Jeff's question.

Kols pharmaceuticals, and with so it's always exciting to hear your voice here.

Think the first part of your question. This company best answered by Mike and then.

Mike If you bounce it back over to me.

I'll then talk about the casino aspects of Justice question.

Michael Benkowitz: Sure. This question has come up, I think, a couple of times over the last year or so, as a baseline. There's always a small percentage, you know, I would say kind of a low, low to mid single-digit percentage of patients that are in our patient assistance program at any point in time with the waiting for CMS coverage to get on PHILD. That has continued to increase. I think on the last call I reported that it was getting up into the higher single-digits.

Sure.

This question has come up I think a couple of times over the last year, so as a baseline.

There's always a small percentage I would say kind of low low to mid single digit.

<unk> patients.

And our patient assistant program at any point in time.

With that what's the.

I just need to wait for the CMS coverage decision on ph ILD and that has continued to increase and so I think I'm on the last call I reported that was getting up into the higher single digits.

Martine Rothblatt: It's about to cross over into double-digits, and we've now crossed over into double-digits. It's a double-digit percentage of patients, but it's on the low end of double-digit percentage. That's great, Mike. Jeff, it's really interesting, the launch of these new Zeno products, and we have on our call, in fact, Dr. Peterson. She is our Senior Vice President for Product Development, and she will be leading the effort now to take those products through their clinical development process. We also have on the call Pat Poisson, who is our Executive Vice President for Technical Operations.

About the crossover into double digits without crossover into double digits, yes.

It's double digit percentage of patients, but it's on the low end of double digit percentage.

Perfect.

Yep Yep that's.

That's great Mike.

Martine Rothblatt: And then the people underneath him who, right now, are responsible for manufacturing our biological products, such as Unituxin, they will be responsible for manufacturing this new type of biological, the xenografts, be they the U-heart, the U-kidney, or the euthymokidney. So all of that is very much moving into the clinical development phase, and it's super exciting doing things like, you know, identifying the clinical trial centers, identifying the clinical investigators, putting together the necessary protocol, having meetings with the FDA to get buy-in for the protocol and clinical development.

Jess it's it's really interesting the launch of these new products and.

Martine Rothblatt: Our biostatisticians are already computing the necessary clinical trial parameters, so everybody is kind of rowing their oars in the same direction toward those clinical development and gating milestones for those products. So I think you're going to see all of that in the near term. And in between, you may hear of certain kinds of expanded access opportunities that arise, such as with the case of Mr. Bennett, where there's a possibility for a leading surgeon at a leading hospital to uniquely save somebody's life through the unique advantages of these xenografts. So I think you'll see some of that as well. In the case of Mr. Bennett, there were no other alternatives.

We have in our call in fact Doctor Peterson. She is our senior Vice President for product development and she will be leading the effort now to take those products through their clinical development process. We also have on the call Pat <unk>, who is our executive VP for Teck.

Martine Rothblatt: And here he is, almost two months out, with no sign of rejection. I just could not be more proud of all of the efforts of our group at Revivacor, how they were able to carefully splice in all of these different 10 genes to make that heart, in the words of Dr. Griffith, the transplant surgeon. He keeps telling me, Martine, this heart is a rock star. And this is the guy who's like, you know, did the second Jarvik heart. I mean, he really knows his stuff.

Cooperations and then people underneath him who right now are responsible for manufacturing on a biologic or.

Our biologic product such as unit Tux, and they will be responsible for manufacturing this new type of biologic the.

<unk> kidney or that you find mckechnie.

So all of that is very much moving into the clinical development phase.

And it's super exciting doing the things like identifying the clinical trial centers identified the clinical investigators.

Putting together the necessary protocol, having the.

The meetings with the FDA to get buy in to the protocol and the clinical development our biostatistician.

We're already computing the necessary clinical trial parameters.

So everybody is kind of rolling their oars in.

In the same direction toward those clinical development and gauging milestones for the.

For those products, so I think you're going to see all of that in the in the near term.

<unk>.

In between you me here.

Certain kind of expanded access opportunities that arise such as with.

The case of <unk>.

Mr Bennett.

Where there is a possibility for a <unk>.

Leading surgeon at a leading hospital.

<unk>.

To uniquely save somebody's life through the <unk>.

The unique advantages of these graphs.

Youll see some of that as well in the case of Mr. Bennett.

No other alternatives and here is almost two months out no sign of rejection I just could not be more proud of all of the efforts of our group at <unk>.

How they were able to.

Carefully.

Place in all of these different 10 genes to make that heart in the words of Dr. Griffith.

Transplant surgeon.

He keeps telling me Martine. This heart is a rock star and this is the Guy who is like did the second Jarvik heart I mean, he really knows his stuff.

Martine Rothblatt: And in the words of, for example, Dr. Montgomery at NYU, he is just so impressed with the, you know, it's hard to get a little bit graphic here, but like the urine produced by the xenokidney, super, super impressed with it. Dr. Locke, the head of transplantation down at UAB, she's, you know, placed her xenokidneys into Mr. Parson She's done another similar type of xenokidney transplant and in both cases, she is just, you know, super, super pleased with the outcome.

And in the words of for example, Doctor.

Summary at NYU. He is just so impressed with the.

It's hard to get a little bit graphic here, but like the euro and produced by the by the.

Zeno kidney Super Super impressed with that Dr.

Doctor lock.

The head of.

Transplant down at UAB.

She's placed her kidneys into.

Mr. Parson was one of the.

The Seaton donors orthotopic positions for the kidneys she's done another.

Similar.

Type of Zeno kidney transplant and in both cases is just super Super pleased with the outcome.

Martine Rothblatt: So this xenotransplantation train has definitely, definitely just left the station. And I think you're going to see some, you know, really exciting ungating of the clinical development and production spending, and capital expansion opportunities right up ahead. Operator, it looks like we have one more question in line. Yes, your next question comes from the line of Andres Argyrides with Wedbush Securities. Your line is now open.

So this casino transplantation train has definitely definitely definitely just left the station and I think youre going to see some really exciting on gating of the clinical development and production spending.

Capital expansion opportunities right up ahead.

Operator, it looks like we have one more question online.

Yes. Your next question comes from the line of Andre.

<unk> with Wedbush Securities. Your line is now open.

Martine Rothblatt: Good morning, and thanks for taking our question. Before I get into questions, just congratulations on the quarter and all the life-saving progress that you've made. It's a true testament to your dedication to it.

Good morning, and thanks for taking a question.

Before I get into the questions and congrats on the quarter and all of the lifesaving.

Progress that you've made it.

True Testament to your dedication to it so.

Martine Rothblatt: So, you know, we appreciate that too. So, just continuing on the transplantation, could you maybe give us a little bit more color around how you think or what the next kind of major milestone would be in these programs? Is it six months out? And then, you know, looking at DPI approval, how are you looking?

We appreciate that so just continuing on the trend.

Thanks, Jason.

Could you maybe give us a little bit more.

Color around how you think or what.

The next kind of major milestone would be.

And these programs at six months out and then.

Looking at.

BPI approval how are you looking at.

The <unk>.

<unk> contributing to.

Or the potential amongst other contributing to.

Growth of patients on <unk> this year and next year as well. Thank you.

Martine Rothblatt: That launch is contributing to the growth of patients on type A, so this year and next year as well. Thank you. Thank you so much, Andreas, for the questions, and thank you for the recognition of us doing our best with our public benefit purpose. Mike, could I call on you again to answer first the DPI-related question, and then I'll follow up with the Zeno-related question.

Thank you so much Andreas for the questions and thank you for the recognition of us doing our best with our public benefit purpose.

Mike if I could call on you again too.

To answer first the DPI related question and then I'll follow up with the <unk> related question.

Michael Benkowitz: Transcripts provided by Transcription Outsourcing, LLC. As I said in my opening remarks, you know, we'll hear back from the FDA in May, assuming that it gets approval, and we're ready to go, ready to launch. We have everything in place and ready to go. We'll have inventory, plenty of inventory. We actually had our launch meeting with our sales team this week, so they're prepped and ready to go. And so we'll be able to really, you know, press play on our launch once we hear back from the FDA. And, you know, we do think that there are opportunities in both PHILD, as I mentioned, to accelerate growth there, as well as in PAH.

Sure so on the <unk> launch.

As I said in my opening remarks.

We'll hear back from the FDA.

Assuming approval and are ready to go ready to launch.

We have everything in place and ready to go.

Well the inventory plenty of inventory.

We actually had our launch meeting with our sales team. This week, so they're prepped and ready to go and so we'll be able to really.

Press press play on our launch.

Once we hear back from the FDA and we do think that there is.

Opportunities in both our ph ILD as I mentioned.

To accelerate growth there as well as PAA. So there's really I think a lot of excitement and enthusiasm around the type of cdti.

Martine Rothblatt: So, you know, there's really, I think, a lot of excitement and enthusiasm around the Tyvasa DPI, and I think it really is going to create an opportunity for us to expand the number of patients that we're able to help with process cyclin therapy in PAH, as well as PHILD, as I said. So we look at that as being a significant contributor to our goal of 25,000 patients by the end of 2025 As Martine said, it should be a blockbuster drug. That's certainly how we view it and how we're planning to approach it from a launch perspective. Thanks so much, Mike.

And I think it really is going to create an opportunity for us to expand the number of patients that we're able to help with process psychotherapy NPA.

N P H as well as ph ILD as I said, so we look at that as being a significant contributor.

Two our March 25000 patients by the end of 2025.

As Martine said.

It should be a blockbuster drug that that's certainly how we view it and how were planning to approach it from a loss perspective.

Martine Rothblatt: Andreas, with regard to the next thing that, you know, you might hear about, it's, I think that, you know, there is a pretty good likelihood that there will be some sort of announcement, and again, knock on wood and, you know, cross my fingers that Mr. David Bennett, Sr. continues to do well at the 90-day and the six-month mark. And the reason I call those out is when the FDA looks at clinical transplantation milestones, they put a lot of emphasis on sort of like two weeks, 30 days, 60 days, 90 days, you know, and, you know, maybe one year.

Thanks, so much Mike.

Entrees with regard to the next thing that you might hear about it.

I think that there is a pretty good likelihood that there will be.

Some sort of an announcement and again knock on wood cross my fingers that.

Mr. David <unk> senior continues to do well at like the 90090, <unk> and the six months Mark and the reason I called those out.

Is when the FDA looks at.

Clinical transplantation milestones they put a lot of emphasis on sort of like two weeks 30 days 60 days 90 days.

And maybe one year.

Martine Rothblatt: And we know this because one of our phase three trials, for example, which relates to the over 200 lives we've saved with our lung bioengineering technology, relates to getting clinical FDA approval for our second lung bioengineering technology, which is wrapping up its last couple of patients in its phase three trial. And with this type of technology, which is, The FDA wants to see that the patients who are on our lung bioengineering technology do as well at two weeks, four weeks, three months, six months, et cetera, as the patients who don't have the advantage of the lung bioengineering technology.

And we know this because one of our phase III trials for example, which relates to the over 200 lives we've saved with our bioengineering technology really.

It relates to getting our clinical FTA approval for our second <unk>.

Bioengineering technology, which is in its ramping up its last couple of patients of its phase III trial.

And with this type of technology, which is.

Kind of techno speak it's called ex vivo lung perfusion technology.

The FDA wants to see that the patients who are on our lung bioengineering technology do as well at two weeks four weeks three months six months et cetera.

As the patients who don't have the advantage of the lung bioengineering technology.

Martine Rothblatt: So that same kind of milestone metrics, I think, are going to apply in the xenocontacts. And our goal would be to show that our xenograft patients, of course, separately for kidneys and hearts, as well at two weeks, four weeks, three months, six months, and a year, as would patients who are otherwise similarly matched but have allografts. Now, you know, depending on the organ, the slope is pretty severe. You know, even with something that has been done for decades, like aloha transplants, you're looking at, you know, 80 to 90% survival rate for one year, which, you know, depending on whether you're looking at the glass half full or half empty, it's like, amazing, these lives were saved. But, you know, oh, my God, can't we do better and not lose, you know, 10 to 20% of the people?

So that same kind of.

Milestone metrics I think are going to apply them to zeno context, and our goal would be to show that our Zeno <unk> patients.

Martine Rothblatt: And for things like lungs, for example, the curve is worse. For kidneys, it's better. Another advantage with kidneys is that you can always, you know, back up to have the patient on dialysis. So that's, you know, that's an advantage. A huge advantage for Zeno is that there is pretty much consensus agreement among experts in the field that people who have rejected a previous kidney transplant and have a lot of reactive antibodies in their blood, that those do not cross-react with Zeno antibodies.

Of course separately for kidneys and Hearts.

Two as well at two weeks four weeks.

Three months six months and the year as wood patients who are otherwise similarly matched but have.

Allografts.

Now depending on the Oregon, the slope is pretty severe.

Even with something that has been done for decades like alloy heart transplants.

Youre looking at.

80% to 90% survival at one year, which.

Depending if you're looking at the glass half full or half empty. It's amazing the flex were saved but Oh, my God can we do better and not lose 10% to 20% of the people.

And for things like lungs. For example, unfortunately, the curve is worse for kidney sits better.

Another advantage with kidneys as you can always back up to half the patient on dialysis. So that's that's an advantage a huge advantage for Zeno is that there is pretty much consensus agreement around the experts in the field.

People, who are who have rejected a previous kidney transplant.

And have a lot of reactive.

Hey bodies in there in their blood that those do not cross react with Zeno antigens. So a person who has already had a kidney transplant would have.

So a person who has already had a kidney transplant would have a, you know, much, much tougher declined to try to get another kidney transplant but would not likely have the same aggressive reaction to another allotransplant as, I mean, to another xenotransplant as they would have had a very aggressive reaction to another allotransplant. So that's a kind of favorable patient category. There are also about 80% of the people who are on dialysis in the U.S. do not even qualify to be on the kidney transplant list.

Much much tougher.

Climb to try to get another kidney transplant, but would not likely have the same aggressive reaction to another allo transplant.

As to another to Athena transplant as they would have had a very aggressive reaction to another allo transplant. So that's a kind of a favorable patient category. There is also about 80% of the people who are on dialysis in the U S do not even qualify to be on the kidney transplant.

So that's another, you know, huge patient population. Then there are patients who have been on dialysis for so long that they have actually, you know, pretty much run out of access ports in their bodies for dialysis. And that becomes increasingly problematic for those patients.

Another huge patient population.

And there are patients who have been on dialysis for so long that they have actually you know pretty much run out of access.

Ports in their body.

For dialysis and that becomes increasingly problematic for those patients.

So, there are just many, many different opportunities to insert xenotransplantation into the transplant paradigm, and we are going to do it, and we're going to follow the same clinical milestones that you've seen the FDA previously require, such as two weeks, 30 days, 60 days, 90 days, et cetera, as for allotransplantation. Well, I see that we're at the half hour mark here. So thank you, Andreas, very much for your questions. Thank you, everybody.

There are just so many many different opportunities to.

To ensure it does you know transplantation into the transplant paradigm.

And.

We're going to do it and we're going to follow the same clinical milestones that you've seen.

The FDA previously require such as two weeks 30 days 60 days 90 days, etc. As for Allo transplantation.

Well I see that we were at the half hour market.

Sure Mark here.

So thank you Andreas very much for your questions. Thank everybody operator, or thank you Dean and do you want me to turn it back to you or to the operator.

Operator, or thank you, Dean. And do you want me to turn it back to you or to the operator? Operator, it's all yours. Thank you for participating in today's United Therapeutics Corporation earnings webcast. A rebroadcast of this webcast will be available for replay for one week by visiting the events and presentation section of the United Therapeutics Investor Relations website at ir.unither.com. Thank you. You may now disconnect.

Yeah.

Yeah.

I guess.

The operator, okay.

Operator, it's all yours.

Thank you for participating in today's United Therapeutics Corporation earnings webcast, a rebroadcast of this webcast will be available for replay for one week by visiting the events and presentations section of the United therapist.

<unk> Investor Relations website at IR, Scott, you and I T H E R Dot com. Thank.

Thank you you may now disconnect.

[music].

Yeah.