-- Based on continued emergence of bempikibart
data from SIGNAL-AA Part A, including strong re-consent rates and
patient demand for continued dosing, Company initiating open-label
extension in 1H'25; SIGNAL-AA Part B remains on track for
initiation of dosing in 1H'25, with topline data expected in 1H'26
--
-- Company to discontinue renal basket trial
for ADX-097 to conserve resources and evaluate strategic options
for its tissue-targeted complement inhibitor platform --
-- Cash runway expected to extend to 2H'26
--
WALTHAM,
Mass., Feb. 10, 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 a corporate restructuring to focus on
the advancement of its bempikibart clinical development program for
the treatment of patients with alopecia areata (AA). As part of the
restructuring, the Company is discontinuing the Phase 2 renal
basket clinical trial of ADX-097 and is evaluating strategic
options for its tissue-targeted complement inhibitor platform,
inclusive of ADX-097 and early-stage assets. In combination with
other cost-saving measures including a reduction in personnel and
related expenses, the strategic restructuring is expected to extend
cash runway to the second half of 2026.
"We have conviction that bempikibart is differentiated from
existing AA therapies and has the potential to transform the
treatment paradigm for this disease. This is based on the continued
emergence of bempikibart data in alopecia areata patients with
longer-term follow-up from Part A of the SIGNAL-AA Phase 2a
clinical trial, the robust pharmacologic data, and a well-tolerated
safety profile," said Jodie Morrison, Chief Executive Officer
of Q32 Bio. "Further, based on the clinical characteristics of
bempikibart, we continue to believe there is potential utility
across additional autoimmune conditions. Our immediate next steps
will be to extend dosing of eligible patients from SIGNAL-AA Part A
in an open-label extension arm and to initiate dosing of patients
in the SIGNAL-AA Part B clinical trial in the first half of 2025,
with Part B topline data expected in the first half of 2026."
Morrison added, "ADX-097, our Phase 2 complement inhibitor, is a
clinical asset with strong potential to treat patients with
complement disorders across multiple indications through its novel
tissue-targeted approach. While this restructuring is a very
difficult decision, it is a necessary step in the context of the
evolving complement-mediated renal disease landscape and as we
prioritize our focus and capital to maximize the potential of
bempikibart. Our restructuring necessitates parting with some
valued colleagues, and I want to personally thank them for their
dedication to our mission, commitment to patients, and their many
contributions to the Company."
Bempikibart Phase 2 Development Program
Results observed to date from Part A of the SIGNAL-AA Phase 2a
clinical trial of bempikibart in patients with AA demonstrated
encouraging clinical activity, including improvement from baseline
on Severity of Alopecia Tool (SALT) score and meaningful
achievement of SALT-20 (SALT score less than or equal to 20)
response through week 36, and continued response in multiple
patients through week 55, approximately 7 months post last dose.
Results from this longer-term follow-up show continued mean SALT
reductions, despite dosing through only 24 weeks, which may be
suggestive of a remittive effect, durability of response, and a key
differentiation from currently approved therapies. Across clinical
trials, including SIGNAL-AA, bempikibart was observed to be
well-tolerated, with robust pharmacologic activity through desired
target engagement, as demonstrated by receptor occupancy, robust
changes in Th2 biomarkers, and expected changes in T-cells,
indicative of potent IL-7 and TSLP inhibition.
Data continues to be collected from a portion of patients who
re-consented to the trial, with patient data available through 55
weeks and longer follow-up expected. Based on re-consent rates and
strong patient demand for continued dosing, Q32 Bio is initiating
an open-label extension (OLE) following the same bempikibart dosing
regimen leveraged in Part A to enable longer-term follow-up of
patients. In addition to the initiation of the OLE, Q32 Bio expects
to initiate dosing in SIGNAL-AA Part B in the first half of
2025.
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. The primary efficacy endpoints include the
proportion of patients achieving a 30% or greater reduction in SALT
score and proportion of patients achieving a SALT-20 at week 36,
with follow-up through week 52. The trial is intended to enable
advancement into pivotal trials upon completion, pending review of
the results. Q32 Bio expects to report initial data from SIGNAL-AA
Part B in the first half of 2026.
Complement Inhibitor Platform
ADX-097, a Phase 2 asset, is designed to be a tissue-targeted
inhibitor of complement activation while minimizing systemic
complement blockade with potential in a broad range of indications
associated with C3d deposition including renal, autoimmune,
vascular, and skin diseases. In preclinical studies, ADX-097
distributed to affected tissues/organs and demonstrated durable
tissue pharmacokinetics (PK) and pharmacodynamics (PD). Q32 Bio has
evaluated ADX-097 in a Phase 1 clinical trial in healthy volunteers
where it observed circulating PK/PD consistent with preclinical
studies, which established in vivo integrity of ADX-097. ADX-097
was also shown to be well-tolerated and demonstrated minimal
anti-drug antibodies (ADA).
ADX-097 was discovered by Q32 Bio and is the most advanced
program of its tissue-targeted complement inhibitor platform.
Additional discovery and earlier development efforts include
ADX-096, a C3d mAb – CR1 fusion protein which demonstrated
preclinical data supportive of its use in ophthalmologic
indications, as well as other C3d mAb fusions and nanobodies
designed for tissue-targeted complement inhibition.
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 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.
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; and plans and expectations in connection
with the evaluation and execution of strategic options for our
tissue-targeted complement inhibitor platform 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 that Part B of
the SIGNAL-AA Phase 2a clinical trial, 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, we may not be successful in
evaluating and executing strategic options for our tissue-targeted
complement inhibitor platform; our expectations regarding the
sufficiency of our cash and cash equivalents to provide financial
runway through clinical milestones and into the second half of
2026, and that we may need additional funding to complete 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
View original content to download
multimedia:https://www.prnewswire.com/news-releases/q32-bio-announces-strategic-restructuring-to-focus-on-advancement-of-bempikibart-in-patients-with-alopecia-areata-302372657.html
SOURCE Q32 Bio