Liquidia Stock

Liquid Corporation operates as a biopharmaceutical company.

The company focuses on the development, manufacturing and commercialization of products that address unmet patient needs, with current focus directed towards the treatment of pulmonary hypertension (PH). The company operates as a single entity through its two wholly owned operating subsidiaries, Liquidia Technologies and Liquidia PAH.

The company generates revenue pursuant to a Promotion Agreement between Liquidia PAH and Sandoz Inc. (Sandoz) sharing profit derived from the sale of Sandoz’s substitutable generic treprostinil injection (Treprostinil Injection) in the United States. Liquidia PAH has the exclusive rights to conduct commercial activities to encourage the appropriate use of Treprostinil Injection. The company employs a targeted sales force calling on physicians and hospital pharmacies in the treatment of pulmonary arterial hypertension (PAH), as well as key stakeholders involved in the distribution and reimbursement of Treprostinil Injection.

The company conducts research, development, and manufacturing of novel products by applying its subject matter expertise in cardiopulmonary diseases and its proprietary PRINT technology, a particle engineering platform that enables precise production of uniform drug particles designed to improve the safety, efficacy, and performance of a wide range of therapies.

The company’s lead product candidate is YUTREPIA for the treatment of PAH. YUTREPIA is an inhaled dry powder formulation of treprostinil designed with PRINT to improve the therapeutic profile of treprostinil by enhancing deep lung delivery while using a convenient, low resistance dry-powder inhaler (DPI) and by achieving higher dose levels than the labelled doses of inhaled therapies. The United States Food and Drug Administration (FDA) tentatively approved the company’s New Drug Application (NDA) for YUTREPIA for the treatment of PAH in November 2021. The FDA also confirmed that the clinical data in the NDA would support the company’s pursuit of a supplemental NDA to treat patients with pulmonary hypertension and interstitial lung disease (PH-ILD) upon the expiration of regulatory exclusivity for the nebulized form of treprostinil in March 2024.

The company’s lead investigational drug, YUTREPIA (treprostinil) inhalation powder was tentatively approved by the FDA in November 2021. YUTREPIA is an inhaled dry powder formulation of treprostinil designed to improve the therapeutic profile of treprostinil by enhancing deep lung delivery and achieving higher dose levels than current inhaled therapies while using a convenient, easy-to-use dry-powder inhaler, the RS00 Model 8 DPI. This device and its variants have beenused in at least eight marketed products globally since 2001, including Novartis’s Foradil Aerolizer for the treatment of asthma and chronic obstructive pulmonary disease (COPD).

YUTREPIA can become the prostacyclin of first choice across the disease continuum in PAH and PH-ILD because of its convenience, low-resistance device and the ability to titrate to higher doses.

The company has developed YUTREPIA under the 505(b)(2) regulatory pathway using the nebulized form of treprostinil, Tyvaso, as the reference listed drug. This regulatory pathway allows the company to rely in part on the FDA’s previous findings of efficacy and safety of Tyvaso and the active ingredient treprostinil. The company submitted the New Drug Application (NDA) for YUTREPIA in January 2020. The FDA conducted on-site pre-approval inspections of two U.S. manufacturing facilities: the company’s Morrisville, North Carolina facility and the facility of the third-party provider of encapsulation and packaging services for YUTREPIA in August 2021 and October 2021, respectively. In November 2021, the FDA issued a tentative approval of YUTREPIA which indicated that the NDA had met all the requirements for final approval but cannot yet be marketed.

The company’s NDA submission also includes results from pharmacokinetic (PK) studies in healthy volunteers indicating that the single-capsule dose of 79.5 mcg YUTREPIA provides comparable PK with nine breaths of Tyvaso (54 mcg). For reference, the target dose of Tyvaso is 9 to 12 breaths per treatment session, 4 times daily. Clinical results from the PK and pivotal studies of YUTREPIA have been presented at various international scientific meetings such as the American Thoracic Society (ATS), International Society of Heart Lung Transplantation (ISHLT), Pulmonary Vascular Research Institute (PVRI), American College of Chest Physicians (ACCP) in 2019 and 2020.

The company is considering conducting other clinical trials to generate additional data to support the use of YUTREPIA, including a clinical trial in pediatric patients. The company conducted a clinical study, known as LTI 201, at certain investigational sites in France and Germany to characterize the hemodynamic dose-response relationship to YUTREPIA. In December 2020, the company decided to terminate the study earlier than planned due to challenges related to the COVID-19 pandemic.

Remodulin is treprostinil administered through continuous intravenous and subcutaneous infusion, as approved by the FDA in 2002 and 2004, and marketed by United Therapeutics. Patients must use external pumps manufactured by third parties to deliver Remodulin. Smiths Medical ASD, Inc. (Smiths Medical) manufactured the pumps used by most patients in the United States to administer Remodulin, including the CADD-MS 3 pump used to deliver subcutaneous Remodulin, and the CADD-Legacy pump to deliver intravenous Remodulin. An estimated 3,000 patients are treated annually with parenteral, infused treprostinil split between the two routes of administration.

In August 2018, Sandoz partnered with Liquidia PAH (then known as RareGen) on an exclusive basis to market and commercialize its generic Treprostinil Injection, which was subsequently launched as the first-to-file, fully-substitutable generic treprostinil for parenteral administration in March 2019. Liquidia PAH promotes the appropriate use of Treprostinil Injection for the treatment of PAH in the United States and works jointly with Sandoz on commercial strategy for the product. Sandoz retains all rights in and to Treprostinil Injection. As the Abbreviated New Drug Application (ANDA) holder, Sandoz maintains responsibility for compliance with FDA regulatory and healthcare laws including any regulatory communications with the FDA or any other regulatory authorities.

The company’s proprietary PRINT particle engineering technology allows it to engineer and manufacture highly uniform drug particles with precise control over the size, three-dimensional geometric shape and chemical composition of the particles. Treprostinil Injection is available for intravenous and subcutaneous administration at the same specialty pharmacies that dispense the brand name medicine.

When first launched in April 2019, Treprostinil Injection was only available for intravenous administration. The cartridges required to operate the CADD-MS 3 pump for subcutaneous administration were not available to patients using Treprostinil Injection due to restrictions imposed by other companies. On May 21, 2021, Liquidia PAH’s manufacturing partner, Chengdu Shifeng Medical Technologies LTD (Chengdu) began selling the RG 3ml Medication Cartridge, which may be used to supply Treprostinil Injection to PAH patients with the CADD-MS 3 pump manufactured by Smiths Medical.

Manufacturing and Supply

The company depends on third-party suppliers for commercial inventory and clinical supplies, including active pharmaceutical ingredients which are used in its product candidates. The company relies on a sole supplier, LGM Pharma, LLC, for treprostinil, the active pharmaceutical ingredient of YUTREPIA, and the company relies on a sole supplier, Plastiape S.p.A (Plastiape). The company also relies on a sole supplier, Lonza Tampa LLC, for encapsulation and packaging services.

Collaboration and Licensing Agreements

Sandoz Promotion Agreement

Liquidia PAH entered into a Promotion Agreement with Sandoz on August 1, 2018, as amended on May 8, 2020, September 4, 2020, November 18, 2022, and March 10, 2023, which engaged Liquidia PAH on an exclusive basis to promote the appropriate use of Sandoz’s treprostinil, Treprostinil Injection, referred to as the Product in the Promotion Agreement, for the treatment of PAH in the United States, including its commonwealths, territories, possessions and military bases. Liquidia PAH works jointly with Sandoz on commercial strategy for Treprostinil Injection and has responsibility for identifying, manufacturing and developing medical devices, including pumps and cartridges, that may be used to administer the Product. Sandoz retains all rights in and to the Product. Sandoz is the holder of the ANDA for the Product. As the ANDA holder, Sandoz maintains responsibility for compliance with FDA regulatory and healthcare laws including any regulatory communications with the FDA or any other regulatory authorities.

Under the Promotion Agreement, Sandoz retains responsibility for: the specifications, manufacture and supply, distribution and future development of treprostinil; regulatory submission and interactions with the FDA pertaining to treprostinil, including maintaining all necessary regulatory approvals; reporting to the FDA or other regulatory authorities on matters relating to manufacturing, sale or promotion, such as any safety events involving treprostinil; internally reviewing and, as it determines appropriate, approving promotional materials developed by Liquidia PAH, and making submissions to the FDA’s Office of Prescription Drug Promotion; handling safety activities including adverse event reporting, and initiating and managing any recalls of treprostinil.

The University of North Carolina at Chapel Hill

In December 2008, the company entered into the Amended and Restated License Agreement with The University of North Carolina at Chapel Hill (UNC) for the use of certain patent rights and technology relating to initial innovations of its PRINT technology (the UNC License). Under the terms of the UNC License, the company has an exclusive license to such patent rights and technology for its drug products. The UNC License grants the company the right to grant sublicenses to the technology, as well as control the litigation of any infringement claim instituted by or against the company in respect of the licensed patent rights.

Aerie Pharmaceuticals

The company has exclusively licensed its PRINT technology to Aerie Pharmaceuticals, Inc., which in 2017 acquired most of the assets of Envisia Therapeutics, Inc., an entity which it formed for broad usage in the design and commercialization of small molecule and biologic ophthalmic therapies. In November 2022, Alcon completed its acquisition of Aerie Pharmaceuticals to helps bolster Alcon’s presence in the ophthalmic pharmaceutical space and as a result, retains Aerie’s direct license to the use of PRINT.

GlaxoSmithKline

In June 2012, the company entered into an Inhaled Collaboration and Option Agreement (the GSK ICO Agreement) with GSK to collaborate on research regarding the application of its PRINT technology to specified inhaled therapies. Pursuant to the GSK ICO Agreement, the company granted GSK exclusive options and licenses to further develop and commercialize such inhaled therapies using its PRINT technology. In September 2015, GSK exercised its option to obtain an exclusive, worldwide license to certain of the company’s know-how and patents relating to its PRINT technology for the purpose of developing inhaled therapeutics.

In June 2019, the company and GSK executed an amendment to the collaboration agreement providing it with rights to develop and commercialize three specified molecular entities for application in inhaled programs using its PRINT technology at its sole expense. This amendment also provides a mechanism for the company to acquire rights to develop and commercialize further molecular entities for inhaled applications.

In January 2020, the company notified GSK of its intent to terminate the GSK ICO Agreement based upon GSK’s lack of continued performance under the original agreement, which constitutes a material breach of the agreement.

Research and Development Expenses

The company’s research and development expenses were $19.4 million for the year ended December 31, 2022.

Intellectual Property

The company is the owner or exclusive licensee of patents and applications relating to its proprietary technology platform and its product candidates and is pursuing additional patent protection for these and for its other product candidates and technology developments.

The company has a total of 145 patents and pending patent applications in its patent portfolio which protect its PRINT technology and drug products in development. As of December 31, 2022, the company was the sole owner of 16 patents in the United States and 41 patents in foreign jurisdictions, as well as 12 additional pending patent applications, including provisional patent applications, in the United States, Europe, Japan and other jurisdictions. In addition to the patents and patent applications owned solely by the company, its patent portfolio also includes 72 patents and 3 patent applications licensed from third parties. As of December 31, 2022, the company had an exclusive, worldwide license from UNC to 19 U.S. patents and 52 foreign patents, as well as three additional patent applications in the United States or selected foreign jurisdictions. Five of the patents in the portfolio licensed from UNC are jointly owned by the company. The company also jointly owns one patent application with Glaxosmithkline Intellectual Property (No. 2) Limited. YUTREPIA is specifically protected by 15 issued patents in the United States, the longest-lived of which will expire in 2037.

The company holds multiple U.S. trademark registrations and has numerous pending trademark applications. Issuance of a federally registered trademark creates a rebuttable presumption of ownership of the mark; however, it is subject to challenge by others claiming first use in the mark in some or all the areas in which it is used.

History

Liquidia Corporation was founded in 2004. The company was incorporated in Delaware in 2004.