-- SIGNAL-AA demonstrated encouraging clinical
activity of bempikibart in patients with alopecia areata (AA),
including improvement from baseline on SALT score and meaningful
achievement of SALT-20 response --
-- SIGNAL-AD Phase 2a clinical trial
in atopic dermatitis demonstrated promising findings in Part A
but did not meet primary endpoint in Part B --
-- Across both trials, bempikibart was
observed to be safe and well tolerated; demonstrated potent IL-7
and TSLP inhibition via changes in both Th2 biomarkers and T-cells,
and desired target engagement --
-- Based on these results, Company plans to
advance bempikibart in patients with AA --
WALTHAM,
Mass., Dec. 10, 2024 /PRNewswire/ -- Q32 Bio Inc.
(Nasdaq: QTTB) ("Q32 Bio"), a clinical stage biotechnology company
focused on developing biologic therapeutics to restore immune
homeostasis, today announced topline results from the SIGNAL-AA
Phase 2a signal finding clinical trial evaluating bempikibart
(ADX-914), which identified encouraging clinical activity in
patients with alopecia areata (AA). The Company plans to expand the
SIGNAL-AA Phase 2a clinical trial and enroll additional patients
evaluating bempikibart in AA.
"We are pleased with the emerging signals observed in the
SIGNAL-AA Phase 2a clinical trial and based upon these, the
positive biomarker data and well tolerated safety profile observed
across both trials, we plan to enroll additional patients into the
SIGNAL-AA clinical trial to further explore the clinical effects of
bempikibart in this patient population. We believe bempikibart has
the potential to be an important new treatment option in a disease
needing new and safer alternatives to currently approved agents,"
said Jodie Morrison, Chief Executive Officer of Q32 Bio. "We
are disappointed that the SIGNAL-AD trial did not achieve its
primary endpoint. Based upon the findings, including the high
placebo rate, we plan to conduct a review to better understand the
results."
"Results from our analysis of SIGNAL-AA showed clinically
meaningful activity and a safety profile that we believe is
differentiated from the currently approved therapies for AA. We are
encouraged by our findings from this clinical trial, and we look
forward to advancing bempikibart as a potential treatment for AA,"
said Jason Campagna, M.D., Ph.D.,
Chief Medical Officer of Q32 Bio. "On behalf of Q32 Bio, I want to
express my gratitude to the patients, their caregivers, and
clinical trial sites that participated across both our bempikibart
Phase 2a trials."
The Company is also providing an update on the SIGNAL-AD
clinical trial in patients with atopic dermatitis (AD). Although
the Company is reporting promising findings in Part A, the trial
did not meet its primary endpoint in Part B. Q32 Bio plans to
conduct a review of the results.
Topline Results from SIGNAL-AA Phase 2a Clinical
Trial:
SIGNAL-AA 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 study
is being conducted to evaluate the efficacy and safety of
bempikibart 200 mg administered subcutaneously (SC),
every-other-week (Q2W) compared to placebo. A total of 44 patients
were enrolled in the trial with a primary endpoint of the mean
relative percent change in SALT score at 24 weeks compared with
baseline.
Following database lock, one site was excluded from the efficacy
analysis based on marked protocol violations of entry criteria.
This resulted in the removal of three placebo patients, rendering
the planned statistical analyses for the primary endpoint
inappropriate due to the reduced sample size. On a post-hoc
analysis of the remaining per-protocol population of patients with
AA (n=27), bempikibart demonstrated an improvement in hair
re-growth compared to placebo:
- At week 24: patients treated with bempikibart showed a
mean reduction in SALT score of 16% in the bempikibart group vs a
reduction of 2% in the placebo group. A Wilcoxon Rank Sum test yielded a p-value of
0.045.
- At week 24: 9% of bempikibart patients in the trial
achieved a SALT-20 (SALT score less than or equal to 20) compared
to 0% in placebo.
- At week 26: 13% of bempikibart patients achieved SALT-20
compared 0% in placebo.
Bempikibart was observed to be safe and well tolerated in the
SIGNAL-AA trial. There were no serious adverse events (SAE) or
Grade 3 or higher adverse events related to treatment.
"Advancing bempikibart in AA is supported by preclinical data
demonstrating the potential of IL-7Rα inhibition in this
disease, and now the resulting data from SIGNAL-AA demonstrated the
clinical potential of an IL-7Rα inhibitor in AA. I am
encouraged by the biomarker data that provide evidence of
biological activity, the safety profile of bempikibart, and the
clinical signal of hair regrowth in patients," said Brett King, M.D., Ph.D., of Dermatology
Physicians of Connecticut, and
former Associate Professor of Dermatology, Yale University School of Medicine. "I believe
these clinical results are promising and warrant further
advancement to expand upon these findings."
Q32 Bio plans to enroll approximately 20 additional patients in
a Part B expansion of the SIGNAL-AA Phase 2a clinical trial to
further evaluate bempikibart in AA, including a loading regimen.
The Company will defer enrollment into the planned Phase 2 clinical
trial of ADX-097 in ANCA-Associated Vasculitis (AAV), previously
expected to begin in 2025, to focus efforts on continued enrollment
in the ongoing bempikibart AA and ADX-097 renal basket Phase 2
clinical trials.
Topline Results from SIGNAL-AD Phase 2a Clinical
Trial:
SIGNAL-AD is a two-part Phase 2a, randomized, double-blind,
placebo-controlled, multi-center clinical trial evaluating
bempikibart in adult patients with persistent, moderate-to-severe
AD. Part A (n=15) was conducted to evaluate safety,
pharmacokinetics (PK), and to enable dose selection for Part B of
the clinical trial. Part A was randomized 2:1 between bempikibart
and placebo in each of two dose cohorts of 2mg/kg or 3mg/kg Q2W SC
for 12 weeks.
In Part A, at week 14, improvement in average EASI score from
baseline was 58% in patients treated with 2mg/kg Q2W SC and 84% in
patients treated at 3mg/kg Q2W SC, and 72% on a pooled basis,
compared to 38% in patients treated with placebo.
In Part B, which evaluated both efficacy and safety of
bempikibart compared to placebo, patients were enrolled 1:1 in the
bempikibart 200 mg Q2W SC (n=52) and placebo (n=54) arms for 12
weeks of treatment. The primary endpoint is the mean percent change
from baseline to week 14 in the Eczema Area and Severity Index
(EASI) score. At week 14, data from Part B demonstrated that
patients treated with bempikibart showed a 74% improvement in
average EASI from baseline, compared to 76% for the placebo group
(p= not statistically significant). Results of the primary endpoint
were generally consistent when stratified for pre-specified
baseline entry criteria. Bempikibart was observed to be safe and
well tolerated in the SIGNAL-AD trial. There were no serious
adverse events (SAE) or Grade 3 or higher adverse events related to
treatment. Q32 Bio plans to conduct a review of the SIGNAL-AD
results.
Biomarker Results in SIGNAL-AD and SIGNAL-AA:
Across SIGNAL-AD and SIGNAL-AA, bempikibart at 200mg Q2W SC
demonstrated favorable PK and target engagement as demonstrated by
substantial reductions in biomarkers of Th2 and Th1. These results
include:
- A reduction in Th2 biomarkers, including TARC, IgE and
eosinophils, which was consistent with the type of biomarker impact
previously observed with other agents that have demonstrated
utility in atopic dermatitis, such as IL-4Rα, IL-13 and OX40
ligand-targeted agents.
- An expected modulation of T-cells, with a 20-30%
reduction, consistent 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 signaling as evidenced by
robust changes in both Th2 biomarkers and T-cells. The Company
believes the mechanism of action of bempikibart has the potential
to be active in other Th2 and Th1 driven diseases, including
asthma, COPD, ulcerative colitis (UC), rheumatoid arthritis (RA),
celiac disease, multiple sclerosis (MS) and others.
"These impressive biomarker data represent a meaningful
advancement in the clinical understanding of how inhibition of
IL-7Rα can be leveraged to treat autoimmune and inflammatory
diseases," said Shelia Violette,
Ph.D., Co-Founder and Chief Scientific Officer of Q32 Bio. "Based
upon its observed mechanism of action, bempikibart continues to
show strong potential as an IL-7Rα inhibitor to treat AA and other
diseases."
The Company has published an updated investor presentation with
additional details regarding the bempikibart update for review by
interested parties. The updated presentation can be
found on the company website at www.Q32Bio.com
under Investors & Media.
About Q32 Bio
Q32 Bio is a clinical stage biotechnology company developing
biologic therapeutics targeting potent regulators of the innate and
adaptive immune systems to re-balance immunity in autoimmune and
inflammatory diseases. Q32 Bio's lead programs, focused on the IL-7
/ TSLP receptor pathways and complement system, address immune
dysregulation to help patients take back control of their
lives.
Q32 Bio's program for adaptive immunity, bempikibart (ADX-914),
is a fully human anti-IL-7Rα antibody that re-regulates adaptive
immune function for the treatment of autoimmune diseases being
evaluated 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. Q32 Bio's program for innate immunity, ADX-097, being
evaluated in a Phase 2 program, is based on a novel platform
enabling tissue-targeted regulation of the complement system
without long-term systemic blockade – a key differentiator versus
current complement therapeutics.
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 including
biomarker data, potential efficacy and potential benefits of
bempikibart; plans and expectations for Part B of the SIGNAL-AA
Phase 2a clinical trial are forward-looking statements, 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, future clinical studies, including the Part B of
the SIGNAL-AA Phase 2a clinical trial, may not be completed in a
timely manner or at all, might be more costly than expected or
might not yield anticipated results, the Company may need
additional funding to complete its clinical studies, which may not
be available on favorable terms or at all, 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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