ACELYRIN, INC. (Nasdaq: SLRN), a late-stage clinical biopharma
company focused on accelerating the development and delivery of
transformative medicines in immunology, today announced positive
proof-of-concept data from an ongoing Phase 1/2 trial of
lonigutamab in thyroid eye disease (TED). Lonigutamab is a
subcutaneously (SC) delivered humanized IgG1 monoclonal antibody
targeting the insulin-like growth factor-1 receptor (IGF-1R), a
validated mechanism of action for the treatment for TED.
In the Phase 1/2 trial, lonigutamab demonstrated rapid
improvements in proptosis and clinical activity score (CAS) at the
first measurement – within three weeks after the first subcutaneous
dose.
“These results are the first reported clinical data for the
anti-IGF-1R mechanism delivered subcutaneously and demonstrating
clinical benefit in thyroid eye disease patients. The data support
our hypothesis that lonigutamab has the potential to optimize
benefit-risk by enabling longer-term subcutaneous dosing to
increase depth and durability of clinical response while attempting
to limit safety liabilities by avoiding the high maximal
concentrations resulting from IV administration, while maintaining
optimal therapeutic levels,” said Shao-Lee Lin, MD, PhD, Founder
and CEO of ACELYRIN. “Tackling thyroid eye disease has special
meaning for our team, and we are thankful to the patients and
investigators who have partnered with us. We are delighted to have
achieved proof of concept for lonigutamab in TED and intend to
advance clinical development with the potential to move patients
toward resolution of their disease.”
Lonigutamab Phase 1/2 Study ResultsThis
multi-center, dose-ranging Phase 1/2 clinical trial is evaluating
the safety and efficacy of lonigutamab dosed in TED patients.
Cohort 1 was placebo-controlled with six patients receiving
lonigutamab and two receiving placebo. Cohort 2 is open label with
data available from six patients at six weeks.
Endpoint(% of patients achieving response at 6
weeks) |
|
Cohort 1 |
|
Cohort 2 |
|
Placebo(n=2) |
Lonigutamab 40mg Q3W(n=6) |
|
Lonigutamab 50mg Loading, 25mg
QW(n=6) |
Proptosis Response(≥ 2 millimeter reduction in proptosis from
baselinei) |
|
0% |
|
50% |
|
|
67% |
|
CAS Response(≥ 2 point reduction in CASi) |
|
0% |
|
100% |
|
|
83% |
|
Diplopia Response(> 1 Bahn-Gorman improvementi,ii) |
|
0% |
|
25% |
|
|
40% |
|
i Clinically meaningful results across each measurementii
Teprotumumab Smith, et al NEJM 2017 21% placebo-adjusted rate at 6
weeks; Douglas, et al NEJM 2020 39% placebo-adjusted rate at 24
weeks
Overall, lonigutamab has been well-tolerated across our clinical
experience to date. There have been no reports of hyperglycemia or
hearing impairment and no serious adverse events.
“It is very encouraging to see the results of subcutaneous
administration of an anti-IGF-1R therapy. The data shown suggest
that there is a clinically meaningful response in patients as early
as 3 weeks after a single subcutaneous dose of lonigutamab. In
addition, it appears that the safety profile of medication through
the subcutaneous route may be favorable when compared to standard
of care,” said Shoaib Ugradar, MD, Department of Orbital and
Oculoplastic Surgery, private practice, Beverly Hills, California.
“It is important to note that this is preliminary data in a small
group, however the positive results are highly promising. Given the
growing body of evidence that suggests thyroid eye disease may have
long-term sequelae, the convenience of a subcutaneous administered
medication with a potentially favorable side effect profile becomes
critical.”
A presentation of these data can be found on the “Events &
Presentations” section of the ACELYRIN website via this link
ACELYRIN.com. Further data from this ongoing trial will be
presented at future scientific meetings.
Next StepsWith proof of concept achieved in
Cohort 1, and Cohort 2 further validating these results, a Phase
2b/3 trial is planned to be initiated in the second half of 2024,
designed to be the first of two registrational trials in TED.
Given the close proximity of the recent data announcements for
izokibep and lonigutamab and today’s conference call, ACELYRIN will
forego hosting a fiscal year 2023 earnings call. The company will
instead announce its financial results in a press release and file
the related 10-K report no later than April 1,
2024.Conference Call InformationACELYRIN will host
a conference call and webcast today, March 20, 2024, at 8:30 a.m.
ET to review these positive clinical data. A live webcast of the
conference call can be accessed in the “Events & Presentations”
section of ACELYRIN’s website at ACELYRIN.com. A recording of the
webcast will be available approximately two hours after the event,
and will be archived on the Company’s website for approximately 30
days.
About the Phase 1/2 TrialThe Phase 1/2 clinical
trial (NCT05683496) is a multi-center trial evaluating the safety
and efficacy of lonigutamab dosed subcutaneously in three cohorts
of patients with active thyroid eye disease (TED). Cohort 1 is
placebo-controlled testing lonigutamab 40mg every three weeks (Q3W)
through six weeks, cohort 2 is open label testing a 50mg loading
dose followed by 25mg every week (QW), and cohort 3 is testing
every four weeks (Q4W) dosing.
For more information about the Phase 1/2 trial, please visit
www.clinicaltrials.gov.
About Thyroid Eye DiseaseThyroid Eye Disease
(TED) is a vision-threatening autoimmune disease in which there is
both inflammation and expansion of the tissues behind the eye,
resulting in eye bulging, known as proptosis, and the subsequent
inability to close the eyelids. Double vision, or diplopia, can
occur, as well as the potential for compression of the retinal
nerve, which can lead to blindness. Thus, TED is a progressive,
chronic inflammatory disease where longer-term treatment has the
potential to improve depth and durability of response. More than
100,000 people in the United States are estimated to suffer from
TED.
About Lonigutamab (anti-IGF-1R)Lonigutamab is a
humanized IgG1 monoclonal antibody targeting the IGF-1 receptor and
is delivered subcutaneously. Relative to standard of care,
lonigutamab binds to a distinct epitope, which results in
internalization of the receptor within minutes, and in preclinical
binding and functional laboratory assays, it has been shown to be
75-fold more potent. The characteristics of lonigutamab that enable
subcutaneous delivery also enable the potential for longer-term
dosing, which we believe can improve depth and durability of
clinical response. Based on our preclinical and pharmacodynamic
data from our completed single ascending dose study with
lonigutamab, we can optimize the therapeutic window utilizing the
SC route of administration. The characteristics of lonigutamab also
allow the potential to minimize exposures relative to IV therapy.
IGF-1 is neuroprotective to cochlear cells of the inner ear and
serves to repair damage that can occur over time. We hypothesize
that high concentrations of anti-IGF-1R due to Cmax from IV
administration can penetrate the blood-labyrinth barrier and
interfere with this normal function. Lonigutamab originated from
Pierre Fabre Laboratories, a French pharmaceutical group.
About ACELYRIN, INC.ACELYRIN, INC. (Nasdaq:
SLRN) is a Los Angeles area-based late-stage clinical biopharma
company – with additional operations in the San Francisco Bay area
– focused on providing patients life-changing new treatment options
by identifying, acquiring, and accelerating the development and
commercialization of transformative medicines. ACELYRIN has two
programs in late-stage clinical development. Izokibep is a next
generation inhibitor of IL-17A in Phase 3 development for the
treatment of psoriatic arthritis, hidradenitis suppurativa and
uveitis. Lonigutamab is a subcutaneously delivered monoclonal
antibody targeting IGF-1R being investigated for the treatment of
TED.
For more information about ACELYRIN, visit us at
www.acelyrin.com or follow us on LinkedIn and X.
Forward Looking StatementsThis press release
contains forward-looking statements including, but not limited to,
statements related to ACELYRIN’s ability to accelerate the
development and delivery of transformative medicines; the
advancement of ACELYRIN’s product candidate lonigutamab and
its therapeutic potential, including its ability to offer
clinically meaningful, differentiated benefits for TED patients
that may improve over time, move patients towards disease
resolution and limit safety liability versus other treatment
options; anticipated development activities including the planned
initiation of a Phase 2b/3 trial in thyroid eye disease and the
ability for such trial to serve as the first of two registrational
studies in TED; and other statements that are not historical fact.
These forward-looking statements are based on ACELYRIN’s current
plans, objectives and projections, and are inherently subject to
risks and uncertainties that may cause ACELYRIN’s actual results to
materially differ from those anticipated in such forward-looking
statements. Such risks and uncertainties include, without
limitation, those associated with the successful completion of
development and regulatory activities with respect to ACELYRIN’s
product candidates, the risk that future results could differ
materially and adversely from early clinical data and other risks
and uncertainties affecting ACELYRIN including those described from
time to time under the caption “Risk Factors” and elsewhere in
ACELYRIN’s current and future reports filed with the Securities and
Exchange Commission, including its Quarterly Report on Form 10-Q
for the quarter ended September 30, 2023. Forward-looking
statements contained in this press release are made as of this
date, and ACELYRIN undertakes no duty to update such information
except as required under applicable law.
ACELYRIN, INC.
Contact:investors@acelyrin.commedia@acelyrin.com
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