Full Press Release Details
Corporation Reports First Quarter 2025 Financial Results
and Provides Corporate Update
MORRISVILLE, N.C., May 8, 2025 -
Liquidia Corporation (NASDAQ: LQDA), a biopharmaceutical company developing innovative therapies for patients with rare cardiopulmonary
disease, today reported financial results for the first quarter ended March 31, 2025. The company will also host a webcast at 8:30
a.m. ET on May 8, 2025 to discuss its financial results and provide a corporate update.
Dr. Roger Jeffs, Liquidia's Chief Executive
Officer, said: "With the FDA's PDUFA goal date on the YUTREPIA NDA just over two weeks away, we remain focused on ensuring
that we are prepared to make YUTREPIA commercially available in the quickest time possible if granted full approval. We continue to believe
that YUTREPIA has the potential to be the prostacyclin of first choice for patients with pulmonary arterial hypertension (PAH) and pulmonary
hypertension associated with interstitial lung disease (PH-ILD)."
Awaiting FDA action on NDA for YUTREPIA (treprostinil) inhalation
On March 28, 2025, the U.S. Food and Drug
Administration (FDA) accepted Liquidia's New Drug Application (NDA) resubmission for YUTREPIA (treprostinil) inhalation
powder to treat PAH and PH-ILD as a complete Class 1 response to the previous action letter issued on August 16, 2024, which
granted tentative approval of YUTREPIA. The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of May 24, 2025, the
day after regulatory exclusivity expires for Tyvaso DPI .
Court will not hear cross-claim that challenged the amendment to
the YUTREPIA NDA to add the PH-ILD indication
On May 2, 2025, Liquidia announced that the U.S. District Court for the District of Columbia (District Court) dismissed, without prejudice,
the cross-claim filed by United Therapeutics (UTHR) that sought to challenge Liquidia's amendment to its NDA for YUTREPIA
(treprostinil) inhalation powder, which added the treatment of PH-ILD)to the proposed label for YUTREPIA. In its ruling, the District
Court determined that UTHR's claim was unripe and that UTHR had failed to plausibly allege that it has standing. UTHR has the right
to appeal the Court's ruling.
Fully enrolled Cohort A of ASCENT study
In March 2025, Liquidia completed enrollment of Cohort A of the open-label ASCENT study evaluating the tolerability and titratability
of YUTREPIA in PH-ILD, with more than 50 patients enrolled. An interim look at the dosing and tolerability profile in the first 20 patients
to complete eight weeks of treatment was consistent with observations made in the INSPIRE study of PAH patients. To date, patients in
Cohort A of ASCENT were able to titrate to doses that are three-times higher than the labelled target dose of nebulized Tyvaso, while
showing positive trends on exploratory measures of efficacy, including 6-minute walk distance. Liquidia will present additional data from
Cohort A of the ASCENT study during two poster sessions at the American Thoracic Society (ATS) 2025 International Conference on May 21,
Strengthened financial position ahead
of launch via amendment to Agreement with HealthCare Royalty
On March 17, 2025, Liquidia entered into a sixth amendment to its
agreement with HealthCare Royalty (HCR Agreement) to provide for up to an additional $100 million of financing in three tranches. The
company intends to use the proceeds to fund ongoing commercial development of YUTREPIA, continued development of YUTREPIA in other
clinical trials, including but not limited to trials for pediatric patients and trials further evaluating the use of YUTREPIA in PAH
and PH-ILD patients, clinical development of L606, a sustained-release formulation of treprostinil administered twice-daily with a next-generation
nebulizer, and for general corporate purposes.
First Quarter 2025 Financial Results
Cash and cash equivalents totaled $169.8 million as of March
31, 2025, compared to $176.5 million as of December 31, 2024.
Revenue was $3.1 million for the three months ended March 31, 2025,
compared to $3.0 million for the three months ended March 31, 2024. Revenue related primarily to the
promotion agreement with Sandoz, Inc. pursuant to which we share profits from the sale of Treprostinil Injection in the United States
(Promotion Agreement). The increase of $0.1 million was primarily due to the impact of unfavorable gross-to-net returns adjustments recorded
in the prior year offset by lower sales volumes in the current year.
Cost of revenue was $1.5 million for each of the three months ended
March 31, 2025 and 2024. Cost of revenue related to the Promotion Agreement as noted above.
Research and development expenses were $7.0 million for the three months
ended March 31, 2025, compared to $10.1 million for the three months ended March 31, 2024. The decrease of $3.1 million
or 31% was primarily due to a $3.6 million decrease in personnel expenses (including stock-based compensation) due to a shift from activities
related to research and development to preparation for the potential commercialization of YUTREPIA. These decreases were offset by a $1.7
million increase in clinical expenses related to our L606 program, and a $0.4 million decrease in expenses related to our YUTREPIA research
and development activities.
General and administrative expenses were $30.1 million for the three
months ended March 31, 2025, compared to $20.2 million for the three months ended March 31, 2024. The increase of
$9.9 million or 48% was primarily due to a $8.1 million increase in personnel expenses (including
stock-based compensation) driven by higher headcount and a shift from activities related to research and development to preparation for
the potential commercialization of YUTREPIA, a $0.6 million increase in legal fees related to our ongoing
YUTREPIA-related litigation, and a $0.6 million increase in facilities and infrastructure expenses.
Total other expense, net was $2.9 million for the three months ended
March 31, 2025, compared with $1.3 million for the three months ended March 31, 2024. The increase of $1.6 million
was primarily driven by a $1.5 million increase in interest expense attributable to the higher borrowings under the HCR Agreement.
Net loss for the three months ended March 31, 2025, was $38.4
million or $0.45 per basic and diluted share, compared to a net loss of $30.1 million, or $0.40 per basic and diluted
share, for the three months ended March 31, 2024.
About YUTREPIA (treprostinil) Inhalation Powder
is an investigational, inhaled dry-powder formulation of treprostinil delivered through a convenient, low-effort, palm-sized device.
In August 2024, the FDA issued tentative approval of YUTREPIA for the PAH and PH-ILD indications. YUTREPIA was designed using Liquidia's
PRINT technology, which enables the development of drug particles that are precise and uniform in size, shape and composition, and
that are engineered for enhanced deposition in the lung following oral inhalation. Liquidia has completed INSPIRE, or Investigation
of the Safety and Pharmacology of Dry Powder Inhalation of Treprostinil, an open-label, multi-center phase 3 clinical study of YUTREPIA
in patients diagnosed with PAH who are na ve to inhaled treprostinil or who are transitioning from Tyvaso (nebulized treprostinil).
YUTREPIA is currently being studied in the ASCENT trial, an Open-Label Prospective Multicenter Study to Evaluate Safety and Tolerability
of Dry Powder Inhaled Treprostinil in Pulmonary Hypertension, to evaluate the safety and tolerability of YUTREPIA in PH-ILD patients.
YUTREPIA was previously referred to as LIQ861 in investigational studies.
About L606 (liposomal treprostinil) Inhalation
L606 is an investigational, sustained-release formulation of treprostinil
administered twice-daily with a next-generation nebulizer. The L606 suspension uses Pharmosa Biopharm's proprietary liposomal formulation
to encapsulate treprostinil which can be released slowly at a controlled rate into the lung, enhancing drug exposure over an extended
period of time. L606 is currently being evaluated in an open-label study in the United States for treatment of pulmonary arterial
hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD) with a planned global pivotal placebo-controlled
efficacy study for the treatment of PH-ILD.
About Treprostinil Injection
Injection is the first-to-file, fully substitutable generic treprostinil for parenteral administration. Treprostinil Injection contains
the same active ingredient, same strengths, same dosage form and same inactive ingredients as Remodulin (treprostinil)
and is offered to patients and physicians with the same level of service and support, but at a lower price than the branded drug. Liquidia
PAH promotes the appropriate use of Treprostinil Injection for the treatment of PAH in the United States in partnership with
its commercial partner, Sandoz, who holds the Abbreviated New Drug Application (ANDA) with the FDA.
About Pulmonary Arterial Hypertension
Pulmonary arterial hypertension (PAH) is a rare, chronic, progressive disease caused by hardening and narrowing of the pulmonary
arteries that can lead to right heart failure and eventually death. Currently, an estimated 45,000 patients are diagnosed and treated
in the United States. There is currently no cure for PAH, so the goals of existing treatments are to alleviate symptoms, maintain
or improve functional class, delay disease progression and improve quality of life.
About Pulmonary Hypertension Associated
with Interstitial Lung Disease (PH-ILD)
Pulmonary hypertension (PH) associated with interstitial lung disease (ILD) includes a diverse
collection of up to 150 different pulmonary diseases, including interstitial pulmonary fibrosis, chronic hypersensitivity pneumonitis,
connective tissue disease-related ILD, and chronic pulmonary fibrosis with emphysema (CPFE) among others. Any level of PH in ILD patients