What we think about United Therapeutics
United Therapeutics in Three Words: Practical, Innovative, Forward-Thinking
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Background
United Therapeutics is a somewhat unique biotech company. Unique in that it is more solutions driven rather than relying on one particular technology or technique. The problem it is trying to solve is prolonging the lives of people living with pulmonary arterial hypertension (PAH) and other life-threatening diseases for which there are few treatment options and currently no cure.
The company defines its approach to its product pipeline as “islands we must reach in our journey to better patient outcomes. Driven by our vision of cures, we build bridges between them and travel towards a future of individualized medicine via gene therapy and organ manufacturing.”
Pulmonary arterial hypertension is a form of pulmonary hypertension that is caused by the constriction of arterial blood vessels in the lungs. The constriction causes the increased pressure, hypertension. It also pushes back on the heart and causes right ventricle enlargement. It is a rare disease so it only affects a few hundred thousand people worldwide. As such, people with the disease had few treatment options available and a dismal prognosis and life expectancy.
That poor prognosis led Founder and CEO Martine Rothblatt to start United Therapeutics upon learning that her daughter was diagnosed with the disease. While most parents would do anything to fight for the lives of their child, Dr. Rothblatt had the means to do something about it (see below under management for more information.) And so she did.
Although the disease is rare and has varying causes, including reaction to drugs and as a symptom of other diseases, scientists understand several molecular pathways that lead to the arterial constriction. In 2002 the company, founded in 1996, had approval of its first drug, Remodulin (treprostinil.) This drug has a half-life of 45 minutes and is delivered by injection. The experience is by no means pleasant for the patients, but it does improve and prolong their lives. A year after its approval, the drug was generating $50M in revenue.
Being a company focused on solving problems for the patient, and using its therapies as bridges to outcomes on a patient’s journey, the company continues to work on reformulating its drugs to better meet the needs of patients. Being practical is how CEO Dr. Rothblatt describes the company. The company expanded its pipeline by reformulating its approved molecule for treating PAH, treprostinil, thereby improving the patient experience. It also enables the company to very efficiently bring new drugs to market and extend patent life.
The company now has five drugs on the market, three as delivery variations of treprostinil, and generated almost $450M in net sales in Q3 2021. It is continually working to improve its drug delivery mechanisms and has several variations in phase 3 clinical trials. It is also expanding to other lung diseases for which its drugs can be used. These include pulmonary hypertension (a distinct World Health Organization classification from PAH), interstitial lung disease (ILD), chronic obstructive pulmonary disease (COPD), and idiopathic pulmonary fibrosis (IPF.) This broadens the company’s addressable market and opportunity for revenue growth.
The company has applied its bridge analogy to its product pipeline. Its near, mid, and long-term products are practical steps that serve as bridges on a patient’s journey towards a cure. While its near-term pipeline is focusing on formulation improvements, its mid- and long-term solutions focus more on novel biologics that can deliver a more individualized patient experience.
The company is in early stages of developing cell-based therapies. Its SAPPHIRE Aurora-GT eNOS program is a form of gene therapy. With this treatment, the company collects endothelial progenitor cells from a patient, adds the gene for endothelial Nitric Oxide Synthase (eNOS) to those cells, amplifies the new cells, and then infuses them back into the patient. This program is currently recruiting patients for safety and efficacy studies in Canada, where the company also has a location.
The company is exploring another cell-based gene therapy that is still in the preclinical stages. This program uses mesenchymal stem cells which have reduced immunosensitivity concerns such that it can become an “off-the-shelf” product without requiring collection of patient cells. These cells are being enhanced with genetic modifications to treat pulmonary diseases or acute inflammatory diseases, such as PAH or sepsis.
The company also recognizes that its treatments to dilate arteries do not cure the disease, they only prolong the lives of patients. To truly solve the patient’s problems, they ultimately need a new pair of lungs. So the company is working on that too as part of it’s longer-term pipeline. It has three programs underway to provide manufactured organs.
The first is its Centralized Ex-Vivo Lung Evaluation Program which is focused on ex-vivo lung perfusion (EVLP). With this program, the company is able to revitalize unusable organs to make them available for transplantation. The company’s subsidiary, Lung Bioengineering is completing clinical trials for this process. As of September 2020, 150 lungs that have gone through the EVLP process have been transplanted into patients.
Another of its programs, xenotransplantation, may be somewhat familiar to you since it has been in the news recently. The company’s wholly owned subsidiary Revivicor is developing genetically modified pig organs to be used for transplant into humans. The company has shown successful transplant of a kidney and most recently of a heart. The company genetically modifies the organs to decrease the chance of rejection and to prevent the growth of pig tissue post transplant. Revivicor also gained U.S. Food and Drug Administration (FDA) approval in 2020 for Intentional Genomic Alteration (IGA) of a line of domestic pigs for food consumption and potential therapeutic purposes. The resultant animal, referred to as GalSafe Pig, no longer has alpha-gal sugars on its cells. This sugar causes allergies to some people when they eat pork and is believed to be the cause of organ rejection in xenotransplantation.
Ideally, if a lung was made from a patient’s own cells, rejection and the potential damage to organs that immunosuppressive therapy presents would not be a problem. With its eyes set on a solution to a problem, the company is working on 3D printing and manufacturing lungs as well. This preclinical product uses the cartilage from a porcine lung or a 3D printed collagen scaffold and allogeneic (neutral) or a patient’s own cells to create a lung. While it seems like manufactured organs are in the realm of science fiction, United Therapeutics and its subsidiary Lung Biotechnology are already making strides for this to become a reality.
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