FDA lifted clinical hold on ARD-101 for the treatment of hyperphagia in Prader-Willi Syndrome (PWS)
Meeting with the FDA scheduled for the first quarter of 2027 to discuss the Company's proposed clinical development plan for ARD-101; Company expects to resume PWS clinical activities in 2027
Exploratory data from the terminated HERO and OLE trials showed numerically greater HQ-CT score reductions with ARD-101 than placebo, with a generally well-tolerated safety profile
$60.3 million in cash, cash equivalents and short-term investments as of September 30, 2026, supports projected operations through 2027
SAN DIEGO, Nov. 13, 2026 (GLOBE NEWSWIRE) -- Aardvark Therapeutics, Inc. (Aardvark or the Company) (Nasdaq: AARD), a clinical-stage biopharmaceutical company developing novel, small-molecule therapeutics designed to suppress hunger for the treatment of Prader-Willi Syndrome (PWS) and metabolic diseases, today reported financial results for the third quarter ended September 30, 2026, and provided business updates.
"The lifting of the clinical hold and the FDA's agreement that we may conduct an open-label trial at 200 mg twice daily are important steps toward resuming clinical development of ARD-101 in PWS," said Tien Lee, M.D., Founder and Chief Executive Officer of Aardvark. "We have developed a proposed clinical development plan and look forward to discussing it with the FDA. We remain committed to advancing ARD-101 for patients and families affected by PWS."
Pipeline Updates
-- In October 2026, the U.S. Food and Drug Administration (FDA) lifted the
clinical hold on the Company's Investigational New Drug (IND) application
for ARD-101 for the treatment of hyperphagia in PWS. As required by the
FDA, the Company conducted an electrocardiogram $(ECG)$ study in healthy
volunteers and an in vitro cardiac sodium channel (NaV1.5) study. No
prolongation of the QRS interval or the QT interval was observed at
ARD-101 doses of 200 mg or 400 mg twice daily (BID). The FDA has agreed
that Aardvark may conduct an open-label trial at 200 mg BID in patients
with PWS.
-- In October 2026, Aardvark reported exploratory data from the terminated
Phase 3 Hunger Elimination or Reduction Objective (HERO) trial and the
open-label extension (OLE) trial. The HERO trial randomized 67 of the
planned 90 participants before it was terminated early; as such, all
efficacy data are considered exploratory, observational, inconclusive and
too limited to support reliable interpretation. Highlights included:
-- Hyperphagia Questionnaire for Clinical Trials (HQ-CT) scores
decreased in both treatment groups, with numerically greater
reductions for ARD-101 than placebo at Weeks 2, 4, and 8. Due to
early termination, evaluable participants declined from 30
(ARD-101) and 31 (placebo) at Week 2 to 11 and 13, respectively,
at Week 12, limiting interpretation of the exploratory findings.
-- In the HERO intent-to-treat population with baseline HQ-CT scores
of 13 or greater, the mean improvement from baseline in the
clinician-rated Clinical Global Impression of Severity (CGI-S)
score was numerically greater with ARD-101 than with placebo at
every scheduled visit, including Week 12.
-- In the OLE cohort, participants who switched from placebo to
ARD-101 had an observed average additional reduction of 6 points
in HQ-CT score after four weeks of treatment. Among OLE
participants who received either placebo or ARD-101 in the HERO
trial, there was an average 11.8-point reduction in HQ-CT score
relative to their baseline score at the start of the HERO trial.
The OLE trial was open-label and did not include a control group.
-- ARD-101 was generally well tolerated, with most adverse events
being mild to moderate. In the HERO trial, treatment-emergent
adverse events were reported in 52% of ARD-101 participants and
50% of placebo participants, and treatment-related adverse events
were reported in 15% of ARD-101 participants and 21% of placebo
participants. One serious cardiac adverse event was observed in
the HERO trial at 800 mg BID in a participant who was also taking
pitolisant, a CYP2D6 substrate. The FDA concurred that this event
was due to a drug-drug interaction with pitolisant. ARD-101
inhibits CYP2D6, the enzyme that metabolizes pitolisant. As with
other approved drugs that inhibit CYP2D6, Aardvark believes this
risk may be able to be managed through measures such as
restricting, or adjusting the dose of, concomitant medications
that are CYP2D6 substrates.
-- Following the lifting of the clinical hold, Aardvark requested a meeting
with the FDA, which has been scheduled for the first quarter of 2027, to
discuss its proposed clinical development plan for ARD-101 in PWS. The
Company intends to provide further details on the development pathway
following FDA feedback. The Company expects to resume PWS clinical
activities in 2027, subject to alignment with the FDA.
Corporate Update
-- In October 2026, Aardvark appointed David Zhang, Ph.D., MBA, to its Board
of Directors as an independent director. Dr. Zhang brings more than 25
years of experience in clinical strategy, biostatistics and regulatory
submissions, including oversight of the pivotal trial design, readout and
New Drug Application for CAMZYOS(R) (mavacamten) at MyoKardia. He
currently serves as Chief Strategy Officer of Abivax S.A.
Third Quarter 2026 Financial Highlights
-- Cash Position: As of September 30, 2026, Aardvark had cash, cash
equivalents and short-term investments of $60.3 million, compared to
$73.9 million as of June 30, 2026. Based on current operating plans,
Aardvark believes that its existing cash, cash equivalents and short-term
investments will be sufficient to fund projected operations through 2027.
-- Research & Development (R&D): R&D expenses for the third quarter of 2026
were $15.7 million, compared to $13.7 million for the third quarter of
2025. The $1.9 million increase for the three months ended September 30,
2026 as compared to the three months ended September 30, 2025 resulted
primarily from costs associated with the FDA-required cardiac safety
study of ARD-101 and higher personnel-related costs, partially offset by
lower clinical trial costs following the termination of the HERO and OLE
trials.
-- General & Administrative (G&A): G&A expenses were $4.0 million for each
of the third quarters of 2026 and 2025.
-- Net Loss: Aardvark reported a net loss of $19.2 million for the third
quarter of 2026, compared to $16.3 million for the third quarter of 2025.
About Aardvark Therapeutics, Inc.
Aardvark is a clinical-stage biopharmaceutical company developing novel, small-molecule therapeutics designed to suppress hunger for the treatment of Prader-Willi Syndrome and metabolic diseases. Hunger, which is the discomfort from not having eaten recently, is a distinct neural signaling pathway separate from appetite, the reward-seeking desire for food. Our programs explore therapeutic applications in hunger-associated indications and potential complementary uses with anti-appetite therapies. For more information, visit www.aardvarktherapeutics.com.
Forward-Looking Statements
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements." These statements include, but are not limited to, statements concerning: Aardvark's business strategy, product candidates, the path forward for ARD-101, including the anticipated resumption of dosing of ARD-101 in patients with PWS and anticipated doses to be used in future trials, the timing, design and outcome of the planned open-label trial, the anticipated timing of Aardvark's meeting with the FDA, the timing for resuming clinical activities, and Aardvark's planned engagement with the FDA regarding its Phase 3 program and the potential for any future Phase 3 trial to support a future marketing application or regulatory approval, as well as plans and objectives of management for future operations. The words, without limitation, "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these or similar identifying words. Forward-looking statements in this press release also include statements regarding Aardvark's anticipated cash runway; Aardvark's belief that risks related to drug-drug interactions may be able to be managed through concomitant medication restrictions or dose adjustments; the interpretation and potential implications of exploratory data from the HERO and OLE trials; the potential of ARD-101 as a treatment option for patients with PWS; and Aardvark's future plans for its PWS program. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the risk that the FDA may not agree with Aardvark's proposed Phase 3 program or may require additional studies; the risk that the timing of Aardvark's meeting with the FDA may be delayed; the timing of resuming dosing and activating clinical sites; uncertainties related to potential delays in the commencement, enrollment and completion of clinical
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