Pulmonary hypertension is a condition in which there is high blood pressure in the arteries going from the heart to the lungs. The number of patients in the US is at least in the hundreds of thousands, according to the American Thoracic Society, but even a rough estimate is difficult to achieve because so many patients never get properly diagnosed. About 200,000 hospitalizations each year are due primarily to pulmonary hypertension, with at least 15,000 deaths ascribed.

Pulmonary arterial hypertension (PAH)—a specific type of hypertension in which the disease process occurs in the actual arteries—generally affects young and otherwise healthy individuals. The average age of diagnosis is 36 years, with three-year survival at only about 50%.

We’ve been following a company called Liquidia Technologies (proposed Nasdaq: LQDA) that has developed a particle engineering platform to enable precision production of uniform drug particles designed to improve the safety, efficacy and performance of a wide range of therapies. Liquidia is developing two product candidates from its own pipeline: LIQ861, in Phase 3 for the treatment of PAH, and LIQ865, for which the company has completed a Phase 1 study for the treatment of local post-operative pain.

Liquidia filed last week for a $50 million IPO via Jefferies and Cowen. The company’s largest investors include NEA, Canaan, Xeraya, The Bill & Melinda Gates Foundation and Morningside Ventures.

Source: Liquidia Technologies

Liquidia’s particle engineering platform is called PRINT—”Particle Replication In Non-wetting Templates”—and combines a proprietary particle molding technology with a modular, roll-to-roll GMP manufacturing process. The technology incorporates production practices from the semi-conductor and materials industries. The company asserts that its PRINT process has multiple benefits:

  • Applying precise particle design to target and design desirable pharmacological benefits into product candidates
  • Supporting development across a wide range of therapeutic areas, molecule types and routes of administrations
  • Improving medicines to meet evolving patient care and disease management needs
  • Leveraging modular, scalable and cost-effective manufacturing

LIQ861 is a dry powder formulation of treprostinil, a vasodilator marketed by United Therapeutics UTHR, that’s designed to improve the drug’s therapeutic profile by enhancing deep-lung delivery and achieving higher dose levels than the current inhaled versions. Liquidia has applied its PRINT technology to deliver LIQ861 through a disposable dry powder inhaler.

Decision Resources Group estimated that in 2016 that more than 50% of patients with PAH in the US were prescribed treprostinil across its three routes of administration (oral, inhaled and parenteral infusion), generating about $1.2 billion in revenue, about one-third of the $3.7 billion US market for PAH drug therapies.

LIQ865 is designed to deliver sustained-release particles of bupivacaine, a non-opioid anesthetic, to treat local post-operative pain for three to five days through a single administration.

Liquidia estimates that there were over 40 million surgeries in the company’s target market of orthopedic and soft tissue surgeries performed in the US in 2016. According to IQVIA, the global market for local anesthetics was approximately $776 million in 2016.

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