Silver Spring’s United Therapeutics Corp. (NASDAQ: UTHR) has garnered global attention for its trailblazing work in organ transplantation — and it’s not stopping now.
That’s the message UT CEO Martine Rothblatt shared on a fourth-quarter earnings call Thursday, calling 2021 “the most exciting year in our company’s history.”
The R&D picture
That’s for a few reasons including xenotransplantation, or the process of transplanting organs between species. The company’s organ manufacturing products “made a dramatic and highly successful debut,” Rothblatt said on the call, noting the recipient of a pig’s heart from subsidiary Revivicor Inc. is still alive with “excellent” cardiovascular function nearly two months following surgery.
That heart transplant followed the prior transplant of a pig kidney into a brain-dead patient whose family had granted consent for the experiment at New York University Langone Health. Such successes “show our organ manufacturing pipeline is now more credible than ever and of truly momentous potential,” Rothblatt said.
The biotech is moving into the clinical development phase of this work, identifying trial sites, tapping investigators and meeting with regulators regarding protocols, Rothblatt said. It could also see “expanded access opportunities” for transplant patients with no other alternatives in unique cases, she said.
The company has said it expects breakthroughs in xenotransplantation, regenerative medicine and 3D bioprinting technology this year, as it builds a large clinical-quality organ facility near Revivicor's headquarters in Blacksburg to support clinical trials for pig heart and kidney programs.
United Therapeutics is also advancing several pharmaceutical products through the clinic, including seven phase 3 trials now underway. The pipeline includes Ralinepag, a once-daily treatment for pulmonary arterial hypertension, and Tyvaso, an existing drug for PAH, which UT is testing in patients with PAH-associated chronic obstructive pulmonary disease, and in patients with idiopathic pulmonary fibrosis in the U.S. and abroad.
Not all good news
But the company also reported setbacks, including a delay to a potential approval for Tyvaso DPI, an inhaler that dispenses a dry powder form of PAH drug treprostinil. The company had sought the Food and Drug Administration’s green light in April to take the inhaler to market as a treatment for PAH and PAH associated with interstitial lung disease, after buying a voucher to speed up the regulatory process and earning priority review for it.
But after the FDA issued a complete response letter withholding its nod in October, UT received a letter from the agency seeking more information about the product’s safety. That request stemmed from a citizen’s petition — a request made by someone else to change how a drug is marketed, among other things — submitted in July regarding the safety of an inactive substance in Tyvaso DPI. The company responded and the FDA extended its review deadline from February 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,” Rothblatt said Thursday, adding the product “remains well-positioned for its summer 2022 launch.” The company also reported that it still expects to achieve its goal of 6,000 patients using Tyvaso by the end of 2022 after expanding its use.
UT has had other recent challenges. A year after kicking off commercial sales of its Remunity Pump, the company has paused its rollout to new patients as it works with partner DEKA Research and Development Corp. to improve the product’s alarm — issues that “do not raise safety concerns” but that it will address “to ensure a continued positive experience with the pump,” UT said in filings with the Securities and Exchange Commission. “We are conducting final testing of the pump’s alarms before we continue with our broader launch.”
UT also disclosed that in February it cut bait on an oral version of Orenitram called OreniPro because of similarities to Orenitram’s dosing schedule, according to SEC filings. That's after it stopped work on an Implantable System for Remodulin in June 2021 with Medtronic Inc. because of “the evolution of treatment options and paradigms in PAH over the past few years, and the anticipated efforts required to satisfy the FDA’s conditions of approval,” the company wrote in its federal filings. Also in 2021, UT discontinued Trevyent a few years after acquiring the disposable pump system.
Commercial success despite generic competition
Still, UT’s commercial success has translated to its income statement. The company grew its revenue and number of patients using its products by double-digit percentages in 2021 from the prior year, despite generic competition that continues to threaten the business, Rothblatt said on Thursday’s call. The drugmaker remains on track to meet its goal of reaching 25,000 patients with its therapies by the end of 2025, she said, adding that it expects double-digit patient growth again in 2022.
Revenue increased to $1.69 billion in 2021 from $1.48 billion in 2020. That growth was aided by a $134.2 million boost in 2021 from sales of the company’s three treprostinil products: Tyvaso ($607.5 million), Remodulin ($513.7 million) and Orenitram ($306.1 million). The company also reported in its SEC filings that patients in the U.S. that use these therapies reached an all-time high in the fourth quarter.
United Therapeutics, which counted about 965 employees at the end of 2021, changed its legal status in October to operate as a public benefit corporation.
