Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) (“Kiniksa”), a
biopharmaceutical company with a pipeline of assets designed to
modulate immunological pathways across a spectrum of diseases,
today announced preliminary data from the Phase 1 clinical trial of
KPL-404 in healthy volunteers. KPL-404 is a monoclonal antibody
inhibitor of the CD40-CD40 ligand (CD40L) interaction, a central
control node of T-cell dependent, B-cell mediated humoral adaptive
immunity.
“The preliminary data from the single-ascending-dose Phase 1
study of KPL-404 are encouraging,” said Sanj K. Patel, Chief
Executive Officer and Chairman of the Board of Kiniksa. “We
believe the data generated to-date suggest that KPL-404 has the
potential to address a broad range of autoimmune diseases. We
expect final data and safety follow-up from all cohorts of the
Phase 1 study in the first half of 2021.”
The Phase 1 trial of KPL-404 is a randomized, double-blind,
placebo-controlled, single-ascending-dose, first-in-human study
that is divided into two parts: a single dose of KPL-404 0.03
mg/kg, 0.3 mg/kg, 1 mg/kg, 3 mg/kg or 10 mg/kg intravenously (IV)
and a single dose of KPL-404 1 mg/kg or 5 mg/kg subcutaneously
(SC). The primary objective is to assess the safety and
tolerability of KPL-404. Secondary endpoints include
pharmacokinetics, CD40 receptor occupancy (RO), the immune response
to the novel test antigen keyhole limpet hemocyanin (KLH) in
clinically relevant dose cohorts, and the anti-drug antibody
response.
All dose escalations occurred as per protocol with no dose
limiting safety findings. All 6 subjects dosed with KPL-404 3 mg/kg
IV showed full receptor occupancy through Day 29, which
corresponded with complete suppression of the T-cell Dependent
Antibody Response (TDAR) to KLH through Day 29. Consistent dose
relatedness was shown in the lower dose level cohorts, including
0.03 mg/kg, 0.3 mg/kg, 1 mg/kg IV and 1 mg/kg SC. Data collection
for the higher dose level cohorts, 10 mg/kg IV and 5 mg/kg SC, is
ongoing.
The data to-date support subsequent study in patients, including
potential IV or SC monthly administration. Kiniksa expects final
data and safety follow-up from all cohorts in the first half of
2021.
The CD40-CD40L interaction has been implicated in diseases such
as rheumatoid arthritis, Sjogren’s syndrome, Graves’ disease, and
systemic lupus erythematosus and in prevention of solid organ
transplant graft rejection, where external proof-of-concept has
been previously shown.
“KPL-404 is designed to inhibit CD40-CD40L interaction, a key
T-cell co-stimulatory signal pathway critical for B-cell maturation
and immunoglobulin class switching. Additionally, dysregulation of
the CD40-CD40L pathway has been implicated in multiple autoimmune
disease pathologies and has broad reaching implications beyond
humoral immunity, with its impact on dendritic cells and macrophage
activity,” said John F. Paolini, MD, PhD, Chief Medical
Officer of Kiniksa. “The preliminary Phase 1 data replicate and
underscore the preclinical data from this program, which showed
favorable pharmacokinetic and pharmacodynamic profiles, including
suppression of TDAR. The data to-date support continued clinical
development, and we look forward to further analyzing the totality
of the dataset.”
About KPL-404KPL-404 is an investigational
humanized monoclonal antibody that is designed to inhibit
CD40-CD40L interaction, a key T-cell co-stimulatory signal critical
for B-cell maturation and immunoglobulin class switching and Type 1
immune responses. Kiniksa believes disrupting the CD40-CD40L
interaction is an attractive approach for multiple autoimmune
disease pathologies such as rheumatoid arthritis, Sjogren’s
syndrome, Graves’ disease, systemic lupus erythematosus and solid
organ transplant. Kiniksa owns or controls the intellectual
property related to KPL-404.
About KiniksaKiniksa is a
biopharmaceutical company focused on discovering, acquiring,
developing and commercializing therapeutic medicines for patients
suffering from debilitating diseases with significant unmet medical
need. Kiniksa’s clinical-stage product candidates, rilonacept,
mavrilimumab, vixarelimab and KPL-404, are based on strong biologic
rationale or validated mechanisms, target underserved conditions
and offer the potential for differentiation. These pipeline assets
are designed to modulate immunological pathways across a spectrum
of diseases. For more information, please
visit www.kiniksa.com.
Forward-Looking StatementsThis press release
contains forward-looking statements within the meaning of the
Private Securities Litigation Reform Act of 1995. In some cases,
you can identify forward looking statements by terms such as “may,”
“will,” “should,” “expect,” “plan,” “anticipate,” “could,”
“intend,” “target,” “project,” “contemplate,” “believe,”
“estimate,” “predict,” “potential” or “continue” or the negative of
these terms or other similar expressions, although not all
forwardlooking statements contain these identifying words. All
statements contained in this press release that do not relate to
matters of historical fact should be considered forward-looking
statements, including without limitation, statements regarding: our
belief that KPL-404 has the potential to address a broad range of
autoimmune diseases; our belief that the data to-date from our
Phase 1 clinical trial of KPL-404 in healthy volunteers support
continued clinical development; expected timing of final data and
safety follow-up from all cohorts of the Phase 1 trial in the first
half of 2021; our beliefs about the mechanisms of action of our
product candidates and potential impact of their approach,
including our belief that KPL-404’s disruption of the CD40-CD40L
interaction is an attractive approach for multiple autoimmune
disease pathologies; and our belief that all of our product
candidates offer the potential for differentiation.
These forward-looking statements are based on management’s
current expectations. These statements are neither promises nor
guarantees, but involve known and unknown risks, uncertainties and
other important factors that may cause our actual results,
performance or achievements to be materially different from any
future results, performance or achievements expressed or implied by
the forward-looking statements, including without limitation, the
following: delays or difficulty in completing our Phase 1 clinical
trial of KPL-404 in healthy volunteers; potential for changes
between final data and any preliminary, interim, top-line or other
data from the Phase 1 clinical trial; our potential inability to
replicate in later clinical trials the positive final data from our
earlier clinical trials or studies; impact of additional data from
us or other companies, including the potential for our data to
produce negative, inconclusive or commercially uncompetitive
results; potential undesirable side effects caused by KPL-404; our
reliance on third parties to manufacture our product candidates;
drug substance and/or drug product shortages; our reliance on third
parties to conduct research, clinical trials, and/or certain
regulatory activities for our product candidates, including for
KPL-404; potential complications in coordinating requirements,
regulations and guidelines of regulatory authorities across
jurisdictions for our clinical trials, including for the Phase 1
clinical trial; the potential impact of the COVID-19 pandemic and
measures taken in response to the pandemic on our business and
operations as well as the business and operations of our
manufacturers, CROs upon whom we rely to conduct our clinical
trials, and other third parties with whom we conduct business or
otherwise engage, including the FDA and other regulatory
authorities; changes in our operating plan and funding
requirements; and existing or new competition.
These and other important factors discussed under the caption
“Risk Factors” in our Quarterly Report on Form 10-Q filed with
the Securities and Exchange Commission (“SEC”) on
November 5, 2020 and our other reports subsequently filed with
the SEC could cause actual results to differ materially
from those indicated by the forward-looking statements made in this
press release. Any such forward-looking statements represent
management’s estimates as of the date of this press release. While
we may elect to update such forward-looking statements at some
point in the future, we disclaim any obligation to do so, even if
subsequent events cause our views to change. These forward-looking
statements should not be relied upon as representing our views as
of any date subsequent to the date of this press release.
Every Second Counts!™
Kiniksa Investor and Media
ContactMark Ragosa(781)
430-8289mragosa@kiniksa.com
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