-- Results presented in AAD late-breaker
demonstrate bempikibart's encouraging improvement on SALT reduction
at week 24 and continued effects after dosing cessation in patients
with severe and very severe alopecia areata (AA) --
-- Durable, ongoing responses in multiple
patients through week 36 follow-up period and beyond to week 55,
despite only 24 weeks of dosing, suggestive of potential for
remittive effect; multiple inbound patient requests to re-initiate
dosing --
-- In the Phase 2a clinical trial, bempikibart
was observed to be safe and well-tolerated, with PK supporting
subcutaneous dosing and receptor occupancy data demonstrating
desired target engagement; clinical biomarkers showed changes in
Th2 biomarkers, and expected on-mechanism changes in T-cells,
indicative of potent IL-7 and TSLP inhibition --
-- Bempikibart development program remains on
track with open-label extension study to initiate in 1H'25;
SIGNAL-AA Part B remains on track for initiation of dosing in
1H'25, with topline data expected in 1H'26 --
WALTHAM,
Mass., March 8, 2025 /PRNewswire/ -- Q32 Bio
Inc. (Nasdaq: QTTB) ("Q32 Bio"), a clinical stage biotechnology
company focused on developing biologic therapeutics to restore
immune homeostasis, today announced additional results from Part A
of its SIGNAL-AA Phase 2a clinical trial of bempikibart in patients
with alopecia areata (AA) at the 2025 American Academy of
Dermatology (AAD) Meeting in Orlando,
FL. Bempikibart is a fully human anti-IL-7Rα antibody that
re-regulates adaptive immune function by blocking IL-7 and TSLP
signaling that is in development for the treatment of AA and
currently being evaluated in a Phase 2 program.
"We are excited to share the results from our late-breaker at
AAD which includes completed 36-week data and extended follow-up of
patients in long-term response who voluntarily re-consented for
further assessments based on their maintenance or further deepening
of responses after the end of the trial," said Jodie Morrison, Chief Executive Officer of Q32
Bio. "These findings demonstrate for the first time in patients the
potential of an IL-7Rα antagonist approach to deliver durable and
sustained activity and recapitulate over a decade of nonclinical
research highlighting the potential of this type of sustained
response in multiple animal disease models. Given these exciting
findings, we have committed to advancing bempikibart as a
potentially differentiated therapy for alopecia areata patients who
have had limited treatment choices and, to date, no biologic option
available."
"The responses of patients with longstanding and severe disease,
not only at 24 weeks but several weeks after treatment withdrawal,
is very provocative," said Brett
King, M.D., Ph.D., of Dermatology Physicians of Connecticut, and former Associate Professor of
Dermatology, Yale University School of
Medicine. "If the activity of bempikibart, including the
potential to induce a durable, long-term response, and
the safety profile are confirmed in upcoming clinical trials,
bempikibart has the potential to change the treatment paradigm of
alopecia areata."
Results from SIGNAL-AA Part A Phase 2a Clinical
Trial:
SIGNAL-AA Part A is a Phase 2a, randomized, double-blind,
placebo-controlled, multi-center clinical trial evaluating
bempikibart in adult patients with severe and very severe AA
(baseline Severity of Alopecia Tool (SALT) scores of 50-100)
treated over 24 weeks, with follow-up through 36 weeks. The trial
is being conducted to evaluate the efficacy and safety of
bempikibart 200 mg administered subcutaneously (SC),
every-other-week (Q2W) compared to placebo. SIGNAL-AA Part A
comprised of 41 patients in the modified intent-to-treat population
and 27 in the per protocol population, with a primary endpoint of
the mean relative percent change in SALT score at 24 weeks compared
with baseline, with follow-up in a 12-week post-treatment period
through week 36. Additional data has been collected on patients
after week 36, with follow-up on multiple patients through week 55
to date, and additional long-term follow-up ongoing.
Highlights on the per protocol basis from the 2025 AAD
late-breaking presentation include:
During the on-treatment window:
- At week 24: Patients with a SALT score of 50-100 treated with
bempikibart (n=23) showed a mean reduction in SALT score of 16% vs
a reduction of 2% in the placebo group (n=4). A Wilcoxon Rank Sum test yielded a p-value of
0.045.
- At week 26: Patients with severe and very severe disease
treated with bempikibart showed a mean reduction in SALT score of
18% in the bempikibart group vs a reduction of 2% in the placebo
group.
- At week 24: 9% of bempikibart patients with severe and very
severe disease achieved a SALT score less than or equal to 20
compared to 0% in placebo.
- At week 26: 14% of bempikibart patients with severe and very
severe disease achieved a SALT score less than or equal to 20
compared to 0% in placebo.
- In the subset of patients with severe disease (baseline SALT
50-95):
- At week 24: patients treated with bempikibart (n=15)
showed a mean reduction in SALT score of 25% improving to 27% at
week 26.
- At week 24: 13% of bempikibart patients achieved a
achieved a SALT score less than or equal to 20, compared to 0% in
placebo, improving to 21% at week 26 vs 0% in the placebo
group.
During the post-treatment follow-up period:
Despite only 24 weeks of treatment across bempikibart treated
patients, a deepening response, as measured by mean SALT
improvement, was observed following dosing cessation (week 24)
through the post-treatment follow-up period (week 36), a paradigm
believed to be associated with IL-7 on-mechanism modulation of
rebalancing T effector memory cells and T regulatory function.
- At week 36, across patients treated with bempikibart, a mean
reduction in SALT score of 20% was observed. In the subset of
patients with severe disease, at week 36, the mean reduction in
SALT score was 28%.
Additional post-treatment data collection remains ongoing,
including longer-term follow-up of patients following the
completion of the trial (post 36 weeks). Outreach was made to
patients regarding the post-treatment experience and patients
willing to participate were re-consented.
- Amongst patients responding to outreach that completed the
treatment period and showed a SALT response during the trial
(n=12), all achieved maintenance of response or further hair growth
in the post treatment period (post 24 weeks), including after the
end of the trial (post 36 weeks).
- All 12 were confirmed by SALT assessment by the investigator,
with a median follow-up of 41 weeks to date (17 weeks post last
treatment) with additional follow-up ongoing.
- Of these, seven patients (7/12) showed additional hair growth
by SALT assessment post-treatment, with median follow-up of 44
weeks to date (20 weeks post last treatment) with additional
follow-up ongoing.
- At week 55: two patients demonstrated improved and ongoing
responses approximately seven months following dosing cessation,
supporting the potential for remittive effect and durability of
response with bempikibart.
Bempikibart demonstrated a well-tolerated safety and
tolerability profile, with no Grade 3 or higher adverse events
related to treatment. Further, no related viral infections were
reported in the bempikibart group.
In addition, in the Phase 2a clinical trial, bempikibart at
200mg Q2W SC demonstrated favorable pharmacokinetics (PK) and
target engagement as demonstrated by substantial reductions in
biomarkers of Th2 and expected modulation of T-cells. The reduction
in Th2 biomarkers included TARC, IgE and eosinophils.
CD3+ T-cells were reduced as expected with target
engagement and IL-7Rα blockade. Q32 Bio believes these results
demonstrate that bempikibart is a potent inhibitor of both TSLP and
IL-7.
"In addition to the meaningful mean SALT reductions through week
24, we observed deepening responses throughout the follow-up period
through week 36 and longer, despite dosing only through 24 weeks,
including two patients with continued response at week 55,
approximately seven months following dosing cessation," said
Jason Campagna, M.D., Ph.D., Chief
Medical Officer of Q32 Bio. "Our development program for
bempikibart is designed to expand on these results, first with our
open-label extension allowing for longer term dosing and follow-up,
and second, with SIGNAL-AA Part B which introduces a loading dosing
regimen, longer dosing period and longer follow up. SIGNAL-AA Part
B is intended to support advancement into pivotal trials upon
completion, pending review of the results."
Bempikibart Phase 2 Development Program:
Q32 Bio is advancing a comprehensive development program
evaluating bempikibart in AA. Based on re-consent rates for
continued follow-up and strong interest from SIGNAL-AA Part A
patients to re-initiate dosing, Q32 Bio plans to initiate an
open-label extension (OLE) following the same bempikibart dosing
regimen leveraged in Part A to enable longer-term follow-up of
patients. Initiation of the OLE remains on track for the first half
of 2025.
In addition, Q32 Bio is advancing bempikibart in the Part B
portion of the SIGNAL-AA Phase 2a clinical trial. SIGNAL-AA Part B
is an open-label clinical trial, dosing patients with bempikibart
for 36 weeks, with follow-up out to 52 weeks, in approximately 20
evaluable patients with severe or very severe AA. Dosing will
include an initial loading regimen of 200mg of bempikibart dosed
weekly over four weeks, followed by a maintenance dose of 200mg
every-other-week over a 32-week period for a total of 36 weeks.
Efficacy will be evaluated on the basis of mean percentage change
from baseline in SALT scores as well as the proportion of subjects
achieving various relative and absolute SALT improvements.at week
36, with follow-up through week 52. The trial is intended to
support advancement into pivotal trials upon completion, pending
review of the results. Q32 Bio expects to initiate Part B in the
first half of 2025 and report topline data in the first half of
2026.
A copy of the AAD late-breaking presentation is available on the
Presentations and Publications page of Q32 Bio's website.
About Q32 Bio
Q32 Bio is a clinical stage biotechnology company whose science
targets potent regulators of the adaptive immune system to
re-balance immunity in autoimmune and inflammatory diseases.
Q32 Bio is advancing bempikibart (ADX-914), a fully human
anti-IL-7Rα antibody that re-regulates adaptive immune function for
the treatment of autoimmune diseases, in a Phase 2 program. The
IL-7 and TSLP pathways have been genetically and biologically
implicated in driving several T cell-mediated pathological
processes in numerous autoimmune diseases.
For more information, visit www.Q32Bio.com.
Availability of Other Information About Q32 Bio
Investors and others should note that we communicate with our
investors and the public using our company website www.Q32Bio.com,
including, but not limited to, company disclosures, investor
presentations and FAQs, Securities and Exchange Commission filings,
press releases, public conference call transcripts and webcast
transcripts, as well as on X (formerly Twitter) and LinkedIn. The
information that we post on our website or on X or LinkedIn could
be deemed to be material information. As a result, we encourage
investors, the media and others interested to review the
information that we post there on a regular basis. The contents of
our website or social media shall not be deemed incorporated by
reference in any filing under the Securities Act of 1933, as
amended.
Forward-Looking Statements
This communication contains forward-looking statements within
the meaning of the U.S. Private Securities Litigation Reform Act of
1995, as amended, and other federal securities laws. Any statements
contained herein which do not describe historical facts, including,
among others, our beliefs, observations, expectations and
assumptions regarding the topline data from the SIGNAL-AA Phase 2a
and the safety, tolerability, clinical activity, potential efficacy
and potential benefits of bempikibart; which involve risks and
uncertainties that could cause actual results to differ materially
from those discussed in such forward-looking statements.
Forward-looking statements are based on management's current
beliefs and assumptions, which are subject to risks and
uncertainties and are not guarantees of future performance. Such
risks and uncertainties include, among others, the risk that
additional data, or the results of ongoing data analyses, may not
support our current beliefs and expectations for bempikibart,
including with respect to the durability of clinical responses,
future clinical studies, including that the OLE may not be
initiated by the first half of 2025 and that Part B of the
SIGNAL-AA Phase 2a clinical trial may not be initiated by the first
half of 2025, may not be completed by the first half of 2026 or at
all, might be more costly than expected or might not yield
anticipated results, and such other risks and uncertainties
identified in the Company's periodic, current and other filings
with the U.S. Securities and Exchange Commission, including its
Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 and any subsequent filings
with the Commission, which are available at the SEC's website at
www.sec.gov. Any such risks and uncertainties could materially and
adversely affect the Company's results of operations and its cash
flows, which would, in turn, have a significant and adverse impact
on the Company's stock price. We caution you not to place undue
reliance on any forward-looking statements, which speak only as of
the date they are made. The Company disclaims 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.
Contacts:
Investors: Brendan Burns
Media: Sarah Sutton
Argot Partners
212.600.1902
Q32Bio@argotpartners.com
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