United Therapeutics: Ability To Expand Tyvaso To Large IPF Market

Summary

  • Tyvaso is being explored by United Therapeutics Corporation in the TETON program (two phase 3 studies) which will determine if label expansion for idiopathic pulmonary fibrosis can be achieved.
  • Tyvaso in an ad-hoc analysis in the short 16-week INCREASE study was shown to improve FVC in PH-ILD patients with IPF.
  • It is expected that the global idiopathic pulmonary fibrosis market could reach $5.32 billion by 2028.
  • Ralinepag is being developed as an oral drug option for the treatment of patients with pulmonary arterial hypertension.
  • Looking for higher risk/reward options trading ideas? I offer this and much more at my exclusive investing ideas service, Biotech Analysis Central. Learn More »

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United Therapeutics Corporation (NASDAQ:UTHR) is a great long-term biotech to own. That's because it is already producing revenues with its drug named Tyvaso (treprostinil), which has been approved by the FDA for pulmonary arterial hypertension (PAH) and Pulmonary hypertension with interstitial lung disease (PH-ILD).

Total revenues in the most recent report increased by 5% year over year, which can be improved upon, but still good nonetheless. Where I believe it can improve on sales with Tyvaso is if it can ultimately obtain FDA approval for this drug for the treatment of patients with idiopathic pulmonary fibrosis (IPF). It is a large multi billion-dollar market and there are only a few drugs approved for this indication. While these drugs do okay for these patients, they appear to slightly slow the decline of IPF, but not actually target the underlying cause. What United intends to do is advance the TETON program, that is deploying two studies which will look at the potential for Tyvaso to become a possible disease-modifying drug for IPF.

While a major undertaking, I believe it could be a huge payoff if it succeeds. It has already initiated one phase 3 study known as TETON 1 and hopes to soon initiate the other phase 3 study TETON 2 soon enough. The point being that it hopes it can expand on the Tyvaso label to include IPF as well. It even has the ability to advance an oral drug known as Ralinepag for the treatment of patients with pulmonary arterial hypertension (PAH). There are two phase 3 studies which are being advanced for this indication which are ADVANCED CAPACITY and ADVANCED OUTCOMES.

I believe that this already a great long-term biotech to own because it is already generating revenues with the sale of Tyvaso for a few indications. The ability to possibly expand to the IPF market would be huge for the company, its investors and especially patients who desperately need a new treatment option for IPF.

Potential Label Expansion Of Tyvaso For The Treatment Of Patients With Idiopathic Pulmonary Fibrosis

The main reason why I believe it is important to look at United Therapeutics Corporation all has to do with the ability to expand the label of Tyvaso to treat patients with IPF. Idiopathic pulmonary fibrosis (IPF) is a chronic lung disease which progresses rapidly. The main issue is that it causes scar tissue to build up in the lungs, which in turn makes it hard for oxygen to reach the bloodstream in a proper manner. The term "idiopathic" means it is not known what causes the pulmonary fibrosis to occur. Thus, the name idiopathic pulmonary fibrosis. This disease typically effects the older aged population and comes with some horrible symptoms. Such symptoms that can be found with this disease are:

  • Lung cancer
  • Blood clots occurring in the lungs
  • High blood pressure evolving
  • Pneumonia.

It is a very large market opportunity to go after, should United Therapeutics succeed in the phase 3 TETON program using Tyvaso. It is expected that the global idiopathic pulmonary fibrosis market could reach $5.32 billion by 2028. This is a large multi-billion dollar market for sure and that's not all. It is a huge unmet medical need, where new treatment options are desperately needed. Why do I state this? That's because the two approved drugs which are now used to treat IPF are OFEV (nintedanib) from Boehringer Ingelheim and Esbriet (pirfenidone) from Genentech subsidiary of Roche (OTCQX:RHHBY, OTCQX:RHHBF).

Both of these drugs have been approved to slow progression of the disease, but there is room for more improvement. Especially, since there are also a lot of side effects with the two currently approved drugs noted above. Why is United Therapeutics advancing Tyvaso for IPF? The first reason I already mentioned briefly in that current treatment options modestly slow the decline (deterioration) of FVC over a period of time. What the hope with Tyvaso is to actually improve FVC. This is not just me stating this, it is what was actually observed in the short 16-week duration of the INCREASE study. Tyvaso was actually shown to improve Forced Vital Capacity (FVC) in the 16-weeks treatment period. The PH-ILD patients also had IPF and as such improvements in FVC were noticed in this INCREASE study. This is great for such a short period of time.

The second reason why it is a good idea is because of the great safety profile which was observed from this study. Speaking of the short treatment period of 16-weeks, the phase 3 TETON studies are being designed to treat IPF patients over a period of 52 weeks. If FVC was improved in such an analysis in only 16 weeks, then the hope is that a longer treatment period would mean a substantially greater increase in FVC with a longer treatment period. This is not guaranteed to occur, but if it does happen, then it would be a huge competitive advantage over the other two already regulatory approved drugs OFEV and ESBRIET. It was a post-hoc analysis which is why there should be some caution here, but still the ability to see an improvement in FVC is huge, considering that the two currently approved drugs only serve to slow decline in FVC instead. Having said all that, the first pivotal TETON 1 study of Tyvaso inhalation solution for IPF patients was initiated back in June of 2021.

The main thing to note is that this is a U.S. study. United Therapeutics is in the process of launching a second phase 3 study known as TETON 2 coming up. It will be similar to TETON 1, but a huge difference is that it will be conducted outside of the United States.

Ralinepag Could Offer Huge Improvement For Pulmonary Arterial Hypertension Patients

Even though Tyvaso was approved for the treatment of patients with pulmonary hypertension (PH) and pulmonary arterial hypertension (PAH), United Therapeutics is advancing an oral drug for PAH. Pfizer (PFE) completed its acquisition of Arena Pharmaceuticals for $6.7 billion back in March of 2022. However, before such an acquisition took place, United Therapeutics bought rights to Ralinepag for an upfront payment of $800 million to Arena at that time. Thus, the reason why United now has rights to advance this drug for PAH. Ralinepag is a once daily oral receptor agonist drug which is currently being explored in two phase 3 studies. These two phase 3 studies are ADVANCE CAPACITY and ADVANCE OUTCOMES. In terms of the ADVANCED OUTCOMES study, it is a randomized, placebo-controlled, double-blind study which is evaluating the use of oral Ralinepag in patients with PAH.

The other late-stage study, known as ADVANCE OUTCOMES, is a study being done to determine if Ralinepag can have a huge impact on exercise capacity for patients with PAH. Why the need to advance Ralinepag for PAH? There are a few reasons actually. The first reason is the ability to give patients an oral drug option for starters. The second reason is because in nonclinical trials and proof of concept phase 2, Ralinepag demonstrated superior and improved pharmacology over UPTRAVI (selexipag) in PAH. UPTRAVI is marketed by Johnson & Johnson (JNJ) and was approved for the treatment of patients with PAH to delay disease progression and reduce risk of hospitalization. These two ongoing phase 3 studies will ultimately determine how well Ralinepag does in treating patients with PAH.

Financials

According to the 10-Q SEC Filing, United Therapeutics had $3.8 billion in cash as of June 30, 2022. It generated total revenues of $466.9 million in Q2 of 2022 which was a 5% year over year increase compared to the same quarter in the year prior. It added a good number of patients on Tyvaso, about 500 during the quarter to be specific.

Not only that, but it also had other advancements as well during the same time period. It obtained FDA approval of Tyvaso DPI for PAH and Pulmonary hypertension with interstitial lung disease (PH-ILD). What DPI stands for is "dry inhalation powder" which is the first treatment of its type for both of these indications. It offers a more convenient portable option for these patients. That's because the inhaler is so small it fits in the palm of a patient's hand.

It also obtained Medicare coverage for Tyvaso for the treatment of patients with PH-ILD as well. With all these recent events, it believes it can obtain about 6,000 patients on Tyvaso by the end of 2022.

Risks To Business

There are several risk factors that investors should be aware of before investing in United Therapeutics. The first risk involves the potential revenues that it could generate with the sales of its products. Even though it generated a 5% increase in revenues of $466.9 million in Q2, doesn't mean this percentage trend will continue for the remaining quarters. Especially, if the economy starts to see a major downward trend.

The second risk relates to the TETON study program. While Tyvaso was determined in an ad-hoc analysis to improve forced vital capacity (FVC) in patients with PH-ILD plus IPF, doesn't mean the same results will be obtained when the final results are released. Still, I think it's a huge positive that Tyvaso in such an analysis was able to improve FVC and not just slow the decline of it.

The final risk relates to Ralinepag itself, which is being developed for the treatment of patients with PAH. The hope is that it achieves successful results, in both the ADVANCE CAPACITY and ADVANCE OUTCOMES studies. There is no guarantee that either or both studies will be successful. Even if the primary endpoints are met in each of these late-stage studies, the drug will have to prove to be equal to or superior to other drugs like UPTRAVI for PAH.

Conclusion

The final conclusion is that United Therapeutics is a great long-term pharmaceutical company to own. That's because it already has FDA approved drugs and is generating revenues for them. In Q2 of 2022, treprostinil-based products (Tyvaso, Remodulin and Orenitram) grew by $42.2 million year over year.

It remains to be seen if this trend continues, but I believe that it has a good shot in doing so. Especially following the label expansion of Tyvaso to treat patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD).

Where I believe this company can increase in value is if it can eventually expand the label of Tyvaso to include the treatment of patients with IPF. Again, the IPF market is very large and the two currently approved drugs slow the decline of FVC, but don't actually improve it. Tyvaso has the opportunity to possibly improve FVC for this patient population. If this can be proven in the two phase 3 TETON studies, then not only do I believe that the label expansion for Tyvaso will happen, but that it will overtake the other two drugs currently marketed for IPF.

Based on the possible label expansion of Tyvaso for IPF, plus the ability to advance oral Ralinepag for the treatment of patients with PAH, these are the reasons why I believe United Therapeutics is a great long-term company to own.

This article is published by Terry Chrisomalis, who runs the Biotech Analysis Central pharmaceutical service on Seeking Alpha Marketplace. If you like what you read here and would like to subscribe to, I'm currently offering a two-week free trial period for subscribers to take advantage of. My service offers a deep-dive analysis of many pharmaceutical companies. The Biotech Analysis Central SA marketplace is $49 per month, but for those who sign up for the yearly plan will be able to take advantage of a 33.50% discount price of $399 per year.

This article was written by

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