Kiniksa Announces U.S. Orphan Drug Designation for Mavrilimumab for the Treatment of Giant Cell Arteritis
September 15 2020 - 8:00AM
Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) (“Kiniksa”), a
biopharmaceutical company focused on discovering, acquiring,
developing and commercializing therapeutic medicines for patients
with significant unmet medical need, today announced that the
U.S. Food and Drug Administration (FDA) granted Orphan
Drug designation to mavrilimumab for the treatment of giant cell
arteritis (GCA). Mavrilimumab is a monoclonal antibody inhibitor
targeting granulocyte macrophage colony stimulating factor receptor
alpha (GM-CSFRα).
“We are pleased to announce that the FDA granted Orphan Drug
designation to mavrilimumab for the treatment of GCA,” said Sanj K.
Patel, Chief Executive Officer and Chairman of the Board of
Kiniksa. “Data from our Phase 2 study of mavrilimumab in GCA are
expected in the fourth quarter of this year.”
There will be a live webcast of Kiniksa’s presentation at the
Morgan Stanley 18th Annual Global Healthcare
Conference on Wednesday, September 16th at 2:00 p.m.
Eastern Daylight Time. The company will provide an update on key
activities relating to rilonacept, mavrilimumab, vixarelimab and
KPL-404. The presentation will be accessible through the Investors
& Media section of the company’s website
(www.investors.kiniksa.com). A replay of the webcast will be
available on Kiniksa’s website for 14 days following the
conference.
About Orphan Drug DesignationThe FDA grants
Orphan Drug designation status to products that treat rare
diseases, providing incentives to sponsors developing drugs or
biologics. The FDA defines rare diseases as those affecting fewer
than 200,000 people in the U.S. at the time of designation. Orphan
drug designation entitles a party to financial incentives such as
opportunities for grant funding towards clinical trial costs, tax
advantages and user‑fee waivers. If a product that has orphan
designation subsequently receives the first FDA approval for the
disease or condition for which it has such designation, the product
is entitled to orphan drug exclusivity, which means that the FDA
may not approve any other applications to market the same drug for
the same indication for seven years from the date of such approval,
except in limited circumstances. Additionally, orphan drug
designation waives the requirement to conduct pediatric studies for
the product in the disease it is designated.
About Giant Cell ArteritisGiant cell arteritis
is a chronic inflammatory disease of medium-to-large arteries.
Cranial giant cell arteritis typically presents with headache and
jaw claudication as well as constitutional symptoms of fever and
fatigue. Acute events can include permanent vision loss from
diminished blood flow to the eye. The large vessel form of giant
cell arteritis affects the branches of the aorta supplying the
trunk and limbs. There is currently one FDA-approved treatment for
giant cell arteritis as an adjunct to a steroid taper.
About MavrilimumabMavrilimumab is an
investigational fully-human monoclonal antibody that targets
granulocyte macrophage colony stimulating factor receptor alpha
(GM-CSFRα). Mavrilimumab was dosed in over 550 patients with
rheumatoid arthritis through Phase 2b clinical studies
in Europe and achieved prospectively-defined primary
endpoints of efficacy and safety. Kiniksa’s lead indication for
mavrilimumab is giant cell arteritis (GCA), an inflammatory disease
of medium-to-large arteries. Kiniksa is also evaluating
mavrilimumab in COVID-19 pneumonia and hyperinflammation. The FDA
granted Orphan Drug designation to mavrilimumab for GCA in
2020.
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 that are implicated
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
forward‑looking 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 mavrilimumab targets the cytokine that may be
responsible for the critical pathological steps of GCA and that the
rare disease has significant unmet need; timing of data from our
Phase 2 study of mavrilimumab in GCA; and our belief that our
product candidates all 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: our potential inability to replicate in later clinical
trials the positive final data from our earlier clinical trials or
investigator-initiated protocols or studies; impact of additional
data from us or other companies; potential undesirable side effects
caused by our product candidates; our potential inability to
demonstrate safety and efficacy to the satisfaction of applicable
regulatory authorities; our reliance on third parties to
manufacture our product candidates; drug substance and/or drug
product shortages; and our reliance on third parties to conduct
research, clinical trials, and/or certain regulatory activities for
our product candidates; the potential impact of the COVID-19
pandemic and measures taken in response to the pandemic; changes in
our operating plan and funding requirements; existing or new
competition; and our ability to attract and retain qualified
personnel.
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 May August 4, 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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