Aerovate Therapeutics, Inc. (Nasdaq: AVTE), a clinical stage
biopharmaceutical company focused on developing drugs that
meaningfully improve the lives of patients with rare
cardiopulmonary disease, today announced financial results for the
year ended December 31, 2023, and recent business highlights.
“Aerovate reached important milestones in 2023
with the achievement of full enrollment of the Phase 2b portion and
the first patient enrolled in the Phase 3 portion of our IMPAHCT
trial evaluating AV-101 for patients with PAH,” said Tim Noyes,
Chief Executive Officer of Aerovate. “In 2024, we look forward to
presenting baseline characteristics of IMPAHCT at the ATS
international conference in May, as well as announcing Phase 2b
topline data in June with the full Phase 2b clinical trial results
to be presented at an upcoming scientific meeting. We remain
confident that delivering our proprietary formulation of imatinib
directly to the pulmonary vasculature via inhalation has the
potential to achieve robust efficacy with a low side effect burden
in PAH patients on two or three background therapies. As such, we
continue to believe that AV-101 has the potential to be an
important part of the future standard of care for PAH
patients.”
2023 Highlights
Enrollment Complete and Topline Data
Expected in June 2024 from Phase 2b Portion of the IMPAHCT Global
Phase 2b/Phase 3 Clinical Trial. IMPAHCT (Inhaled iMatinib
Pulmonary Arterial Hypertension Clinical Trial) is a global Phase
2b/Phase 3 trial of AV-101, our self-administered, twice daily dry
powder inhaled formulation of the antiproliferative drug imatinib
for the treatment of PAH. PAH is a devastating disease impacting
approximately 70,000 people in the United States and Europe. In
November 2023, we announced completion of enrollment at 202 adult
patients in the Phase 2b portion of the IMPAHCT trial, and we
continue to expect to provide topline data in June 2024.
First Patient in Phase 3 Portion of
IMPAHCT Enrolled in November 2023. Due to the seamless
trial design, we were able to enroll our first patient in the Phase
3 portion of the IMPAHCT trial in the same month we completed
enrollment in our Phase 2b portion of the IMPAHCT trial. Enrollment
continues with over 120 clinical sites participating in the Phase 3
portion of the trial. The timing for topline Phase 3 data and the
size of the Phase 3 portion of the trial will be determined based
upon the results from the Phase 2b portion of the trial.
Trial Data Published and Presented at
Scientific Conferences. We presented at the American
Thoracic Society (ATS) 2023 International Conference results in
nonclinical species that (i) direct delivery of imatinib to the
lungs demonstrated increased lung exposure compared with oral or IV
dosing and (ii) the type of formulation impacted lung exposure with
dry powder demonstrating greater lung exposure than suspension or
solution and greater lung exposure versus oral or intravenous
delivery.
Expanded Intellectual Property
Portfolio. We continued expanding our intellectual
property portfolio in 2023 with the issuance of two additional
patents bringing the total issued patents to six. In 2023, the
United States Patent and Trademark Office issued Patents 11,806,349
and 11,813,263 to us, and we continue prosecuting multiple
additional applications to cover our product globally.
Expanded Board of Directors with
Experience in PAH. We appointed Habib Dable and Donald
Santel to our Board of Directors. In March 2024, Mr. Dable was also
appointed as Chair of our Board of Directors, as successor to Mark
Iwicki, who remains a member of our Board. Mr. Dable is on three
public biopharmaceutical company boards and was previously Chief
Executive Officer of Acceleron Pharma Inc. at the time of its sale
to Merck & Co., Inc. Mr. Santel serves on the board of
directors of three biopharmaceutical companies and as chair of two
of them. Mr. Santel previously served as Chief Executive Officer
for CoTherix, Inc., a biopharmaceutical company focused on
developing therapies for cardiopulmonary disease, including
PAH.
2023 Financial Results
Cash, cash equivalents and short-term
investments totaled $122.4 million as of December 31, 2023,
compared to $135.2 million as of September 30, 2023. The decrease
was primarily driven by operational costs for the three-month
period ended December 31, 2023.
R&D expenses: Research and
development (R&D) expenses for the year ended December 31, 2023
were $64.2 million as compared to $38.6 million for the year ended
December 31, 2022. The increase in R&D expenses was due
primarily to clinical trial costs, manufacturing costs, and
increased headcount-related costs in 2023 as compared to 2022.
G&A expenses: General and
administrative (G&A) expenses for the year ended December 31,
2023 were $17.2 million as compared to $14.6 million for the year
ended December 31, 2022. The increase in G&A expenses was due
primarily to increased headcount-related costs, travel and other
miscellaneous costs in 2023 as compared to 2022.
Net loss: Net loss for the year
ended December 31, 2023 was $75.5 million as compared to $51.5
million for the year ended December 31, 2022. Net loss included
stock-based compensation expense of $11.9 million and $5.5 million
for the years ended December 31, 2023 and December 31, 2022,
respectively.
Financial guidance: We expect
that our cash, cash equivalents and available-for-sale investments
will be sufficient to fund our operations into 2026, based on our
current operating plan.
About AV-101
AV-101 is an investigational, proprietary dry
powder inhaled formulation of the antiproliferative drug imatinib.
Developed specifically for pulmonary arterial hypertension (PAH),
AV-101 targets cellular hyperproliferation and resistance to
apoptosis, driven by improper signaling in cells of the distal
pulmonary arteries. By targeting the proliferation and accumulation
of cells in the arteries of the lungs, we believe AV-101 has the
potential to provide meaningful improvements for patients beyond
the capabilities of currently approved therapies. AV-101 is
designed for delivery by an easy-to-use dry powder inhaler,
directly into the lungs to maximize potential clinical benefit and
limit systemic adverse effects. Phase 1 results published in ERJ
Open Research showed that AV-101 delivered by dry powder
inhalation was generally well-tolerated by healthy adult volunteers
with no serious adverse events reported. Aerovate has completed
enrollment in the Phase 2b portion of the IMPAHCT clinical trial
and is currently enrolling patients in the Phase 3 portion to
evaluate the safety and efficacy of AV-101 in adults with PAH.
About the IMPAHCT Trial
IMPAHCT (Inhaled iMatinib Pulmonary Arterial
Hypertension Clinical Trial) is a multi-national,
placebo-controlled Phase 2b/Phase 3 trial in adults with PAH that
continuously enrolled patients as the study progresses from Phase
2b to Phase 3. The Phase 2b portion of the trial will evaluate
three doses of AV-101 over 24 weeks, compared to placebo, to
identify an optimal dose based on the primary endpoint, change in
pulmonary vascular resistance (PVR), and safety, tolerability, and
other clinical measures. The Phase 3 portion of the trial will
compare patients taking the optimal dose of AV-101, selected from
the Phase 2b data, to placebo. The primary endpoint of the Phase 3
portion of the trial will be change in six-minute walk distance
(6MWD) over 24 weeks versus placebo. More information about this
trial is available at
https://clinicaltrials.gov/ct2/show/NCT05036135https://clinicaltrials.gov/ct2/show/NCT05036135.
About Aerovate Therapeutics,
Inc.
Aerovate is a clinical-stage biopharmaceutical
company focused on developing drugs that meaningfully improve the
lives of patients with rare cardiopulmonary disease. Aerovate’s
initial focus is on advancing AV-101, its proprietary dry powder
inhaled formulation of the drug imatinib for the treatment of
patients with PAH. Learn more at aerovatetx.com or follow the
company on X (formerly known as Twitter) and LinkedIn.
Available Information
Aerovate announces material information to the
public about the Company, its products and services, and other
matters through a variety of means, including filings with the U.S.
Securities and Exchange Commission (SEC), press releases, public
conference calls, webcasts, the investor relations section of the
Company website at ir.aerovatetx.com, and the Company’s X (formerly
known as Twitter) account @AerovateTx in order to achieve broad,
non-exclusionary distribution of information to the public and for
complying with its disclosure obligations under Regulation FD.
Cautionary Note Regarding
Forward-Looking Statements
This press release contains forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995, as amended. Forward-looking statements can be
identified by words such as “anticipate,” “believe,” “continue,”
“could,” “estimate,” “expect,” “future,” “goal,” “intend,” “look
forward to,” “may,” “plan,” “potential,” “predict,” “project,”
“potential,” seek,” “strategy,” “should,” “target,” “will,” “would”
and similar expressions regarding future periods. These
forward-looking statements include, but are not limited to,
statements regarding the therapeutic potential and clinical
benefits of AV-101 in PAH; our anticipated timing for the release
of topline data from the Phase 2b portion of the IMPAHCT trial; our
expectations regarding continuing patient enrollment for the Phase
3 portion of the IMPAHCT trial; our belief that we will have
capital to fund Aerovate into 2026; our expectations regarding the
strength of our intellectual property portfolio globally; our
business plans and objectives for AV-101, including potential
regulatory submissions and approvals; the anticipated contribution
of the members of our board of directors and management team to our
operations and progress; and our growth and goals as a company.
Any forward-looking statements in this press
release are based on management’s current expectations and beliefs
and are subject to a number of risks, uncertainties and important
factors that may cause actual events or results to differ
materially from those expressed or implied by any forward-looking
statements contained in this press release, including, without
limitation, those risks and uncertainties related to the
therapeutic potential and clinical benefits of AV-101; the timing
associated with patient enrollment, initiation, delivery of drug
supply and continuation of our Phase 2b/Phase 3 trial of AV-101 in
PAH patients; clinical trials, operations and goals; positive
results from a clinical study may not necessarily be predictive of
the results of future or ongoing clinical studies; regulatory
developments in the United States and foreign countries; as well as
those risks and uncertainties set forth more fully under the
caption “Risk Factors” in our most recent Annual Report on Form
10-K filed with the SEC and subsequent filings with the SEC. We
caution you not to place undue reliance on any forward-looking
statements, which speak only as of the date they are made. We
disclaim any obligation to publicly update or revise any such
statements to reflect any change in expectations or in events,
conditions or circumstances on which any such statements may be
based, or that may affect the likelihood that actual results will
differ from those set forth in the forward-looking statements. Any
forward-looking statements contained in this press release
represent our views only as of the date hereof and should not be
relied upon as representing its views as of any subsequent
date.
|
Aerovate Therapeutics, Inc. |
Condensed Consolidated Balance Sheets |
(Unaudited) |
(in thousands) |
|
|
|
December 31, |
|
December 31, |
|
|
2023 |
|
2022 |
Assets |
|
|
|
|
|
|
|
|
Cash, cash equivalents and available-for-sale securities |
|
$ |
122,439 |
|
|
$ |
129,220 |
|
Other assets |
|
|
4,979 |
|
|
|
6,081 |
|
Total assets |
|
|
127,418 |
|
|
|
135,301 |
|
|
|
|
|
|
|
|
|
|
Liabilities and
Stockholders' Equity |
|
|
|
|
|
|
|
|
Accounts payable and accrued and other current liabilities |
|
$ |
17,161 |
|
|
$ |
7,397 |
|
Other liabilities |
|
|
745 |
|
|
|
1,161 |
|
Total liabilities |
|
|
17,906 |
|
|
|
8,558 |
|
Total stockholders’
equity |
|
|
109,512 |
|
|
|
126,743 |
|
Total liabilities and
stockholders' equity |
|
$ |
127,418 |
|
|
$ |
135,301 |
|
Aerovate Therapeutics, Inc. |
Condensed Consolidated Statements of Operations and
Comprehensive Loss |
(Unaudited) |
(in thousands, except share and per share amounts) |
|
|
|
Three Months Ended December 31, |
|
Year Ended December 31, |
|
|
2023 |
|
2022 |
|
2023 |
|
2022 |
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
$ |
17,813 |
|
|
$ |
12,221 |
|
|
$ |
64,219 |
|
|
$ |
38,622 |
|
General and administrative |
|
|
4,253 |
|
|
|
3,631 |
|
|
|
17,190 |
|
|
|
14,615 |
|
Total operating expenses (1) |
|
|
22,066 |
|
|
|
15,852 |
|
|
|
81,409 |
|
|
|
53,237 |
|
Loss from operations |
|
|
(22,066 |
) |
|
|
(15,852 |
) |
|
|
(81,409 |
) |
|
|
(53,237 |
) |
Total other income |
|
|
1,709 |
|
|
|
856 |
|
|
|
5,944 |
|
|
|
1,751 |
|
Provision for income
taxes |
|
|
56 |
|
|
|
25 |
|
|
|
56 |
|
|
|
25 |
|
Net loss |
|
$ |
(20,413 |
) |
|
$ |
(15,021 |
) |
|
$ |
(75,521 |
) |
|
$ |
(51,511 |
) |
Net loss per share, basic and
diluted |
|
$ |
(0.74 |
) |
|
$ |
(0.61 |
) |
|
$ |
(2.87 |
) |
|
$ |
(2.10 |
) |
Weighted-average shares of
common stock outstanding, basic and diluted |
|
|
27,695,184 |
|
|
|
24,610,723 |
|
|
|
26,331,630 |
|
|
|
24,472,104 |
|
|
(1) Non-cash charges were $3.3 million and $11.9 million, and $1.7
million and $5.5 million for the three and twelve months ended
December 31, 2023 and 2022, respectively. |
|
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