Liquidia to Expand PAH Treatment Efforts With RareGen Acquisition

Liquidia Technologies, a company developing an inhaled form of treprostinil for pulmonary arterial hypertension (PAH), has established an agreement to acquire the rare disease company and marketer of generic treprostinil injections, RareGen, strengthening the company’s efforts in the PAH field.

Under the agreement, the two companies will consolidate under a new holder company, Liquidia Corporation, that will serve as the successor of Liquidia Technologies. RareGen’s current directors, Paul B. Manning and Roger A. Jeffs, PhD, will join the board of directors of the new company.

“The combined company formed through this transaction aims to expand access and treatment options for PAH patients,” Manning, a current investor in Liquidia and beneficial owner of a majority of RareGen’s equity, said in a press release.

“RareGen’s commitment to, and knowledge in the PAH space will amplify Liquidia’s efforts to support this difficult-to-treat patient group,” added Manning, who is also president and CEO of PBM Capital Group, a healthcare investment firm that has RareGen in its portfolio of companies.

The vasodilating therapy treprostinil is a man-made version of the hormone prostacyclin, a natural compound that helps blood vessels relax and widen, and that prevents platelets from clumping and blocking arteries, improving blood flow. Treprostinil is also able to prevent artery wall remodeling processes and reduce the pulmonary artery narrowing that occurs in PAH patients.

In the U.S., three treprostinil-based therapies marketed by United Therapeutics are approved for PAH treatment — an injectable form called Remodulin, an inhaled formulation called Tyvaso, and the extended-release tablet form called Orenitram.

A generic version of Remodulin was launched last year in the U.S. and it is being commercialized by RareGen under an agreement with its developer Sandoz. Under the new agreement, Liquidia will assume RareGen’s responsibilities with Sandoz, and continue to market the generic treprostinil injections.

Liquidia is also developing a proprietary inhaled formulation of treprostinil, called LIQ861, which is under review by the U.S. Food and Drug Administration for the treatment of PAH.

This formulation is a dry powder that is turned into highly uniform drug particles using the company’s PRINT technology, improving treprostinil’s deep-lung delivery compared with other inhaled formulations. The particles are inhaled through a user-friendly, portable, dry powder inhaler.

The submission for approval of LIQ861 is based on positive data from three clinical trials, including the open-label INSPIRE Phase 3 trial (NCT03399604) that assessed the safety, tolerability, and pharmacokinetic properties (the movement of the therapy into, through, and out of the body) of LIQ861.

Results showed that LIQ861 is safe and well-tolerated after two months of treatment, and that it improves the ability to perform physical activities, stabilizes quality of life, and requires fewer inhalations to achieve a therapeutic equivalent of Tyvaso.

“We are excited about LIQ861 and think it will offer a more convenient, and portable treatment option for inhaled Treprostinil,” Manning said.

With the acquisition of RareGen — which has a small, targeted sales force focused on PAH — Liquidia will improve its knowledge base, customer reach, and commercial planning.

“The acquisition of RareGen creates tremendous value for Liquidia’s stockholders and, most importantly, patients,” said Neal Fowler, CEO of Liquidia. “RareGen offers a knowledgeable team and scalable sales force, coupled with rights to commercialize an important PAH product that complements Liquidia’s lead product candidate in PAH, LIQ861. The acquisition of RareGen bolsters Liquidia’s ability to advance much needed treatment options for the PAH community.”

The acquisition is expected to close by year’s end, pending approval from Liquidia’s stockholders and after the Securities and Exchange Commission validates the merger form.