Reata Pharmaceuticals, Inc. Receives Orphan Drug Designation for Bardoxolone Methyl for the Treatment of Autosomal Dominant P...
June 05 2019 - 6:00PM
Reata Pharmaceuticals, Inc. (Nasdaq: RETA), a clinical-stage
biopharmaceutical company, today announced the United States Food
and Drug Administration (FDA) has granted orphan drug designation
to bardoxolone methyl (bardoxolone) for the treatment of autosomal
dominant polycystic kidney disease (ADPKD).
ADPKD is the most common inherited form of
kidney disease affecting approximately 140,000 patients in the
United States. It is characterized by the development of
pathologic fluid-filled cysts throughout the kidneys, which leads
to organ enlargement and chronic kidney disease (CKD).
Despite standard of care treatment, approximately 50% of these
patients will progress to end-stage kidney disease and require
dialysis or a kidney transplant by 60 years of age.
“Obtaining orphan drug designation for
bardoxolone for the treatment of ADPKD is an important milestone
for Reata. This is the third orphan drug designation obtained
for bardoxolone in the United States for the treatment of diseases
characterized by mitochondrial dysfunction and inflammation, and it
is the second designation for the treatment of patients with rare
forms of CKD,” said Warren Huff, Reata’s Chief Executive Officer
and President. “We believe that, if approved, bardoxolone may
prove to be a meaningful new treatment option for patients with
ADPKD.”
Last year, Reata released positive data from the
ADPKD cohort of the PHOENIX Phase 2 study of bardoxolone in four
rare forms of CKD. After 12 weeks of bardoxolone treatment,
we observed a mean increase from baseline in estimated glomerular
filtration rate (eGFR) of 9.3 mL/min/1.73 m2 (n=31;
p<0.0001). Notably, 96% of patients who reached Week 12
demonstrated an improvement in eGFR. The observed improvement
represents a recovery of approximately two years of average eGFR
loss. Based on these encouraging results, Reata launched
FALCON, an international, double-blind, placebo-controlled,
parallel group, Phase 3 study of bardoxolone in approximately 300
patients with ADPKD. The primary efficacy endpoint, which may
support accelerated approval under Subpart H, is the change from
baseline in eGFR compared to placebo after 48 weeks of treatment
followed by a 4-week drug withdrawal period.
Orphan drug status is granted to treatments for
diseases that affect fewer than 200,000 people in the United States
and provides specific incentives for the development of therapies
intended for the treatment, diagnosis, or prevention of rare
diseases. Such designation will provide Reata with certain
development incentives, including tax credits for clinical testing,
exemption from a prescription drug user fee, and seven years of
market exclusivity.
About Bardoxolone
Bardoxolone is an experimental, oral, once-daily
activator of Nrf2, a transcription factor that induces molecular
pathways that promote restoration of mitochondrial function,
reduction of oxidative stress, and inhibition of pro-inflammatory
signaling. The FDA has granted orphan drug designation to
bardoxolone for the treatment of Alport syndrome, ADPKD, and
pulmonary arterial hypertension. The European Commission has
granted orphan drug designation to bardoxolone for the treatment of
Alport syndrome. In addition to FALCON, bardoxolone is
currently being studied in CARDINAL, a Phase 3 study for the
treatment of Alport syndrome, CATALYST, a Phase 3 study for the
treatment of connective tissue disease-associated pulmonary
arterial hypertension, and AYAME, a Phase 3 study for the treatment
of diabetic kidney disease in Japan. AYAME is being conducted
by our licensee Kyowa Hakko Kirin Co., Ltd.
About Reata Pharmaceuticals,
Inc.
Reata is a clinical-stage biopharmaceutical
company that develops novel therapeutics for patients with serious
or life-threatening diseases by targeting molecular pathways
involved in the regulation of cellular metabolism and inflammation.
Reata’s two most advanced clinical candidates, bardoxolone
and omaveloxolone, target the important transcription factor Nrf2
that promotes restoration of mitochondrial function, reduction of
oxidative stress, and inhibition of pro-inflammatory
signaling. Bardoxolone and omaveloxolone are
investigational drugs, and their safety and efficacy have not been
established by any agency.
Forward-Looking Statements
This press release includes certain disclosures
that contain “forward-looking statements,” including, without
limitation, statements regarding the success, cost and timing of
our product development activities and clinical trials, our plans
to research, develop and commercialize our product candidates, and
our ability to obtain and retain regulatory approval of our product
candidates. You can identify forward-looking statements
because they contain words such as “believes,” “will,” “may,”
“aims,” “plans,” and “expects.” Forward-looking statements
are based on Reata’s current expectations and assumptions.
Because forward-looking statements relate to the future, they are
subject to inherent uncertainties, risks, and changes in
circumstances that may differ materially from those contemplated by
the forward-looking statements, which are neither statements of
historical fact nor guarantees or assurances of future
performance. Important factors that could cause actual
results to differ materially from those in the forward-looking
statements include, but are not limited to, (i) the timing, costs,
conduct, and outcome of our clinical trials and future preclinical
studies and clinical trials, including the timing of the initiation
and availability of data from such trials; (ii) the timing and
likelihood of regulatory filings and approvals for our product
candidates; (iii) the potential market size and the size of the
patient populations for our product candidates, if approved for
commercial use, and the market opportunities for our product
candidates; and (iv) other factors set forth in Reata’s filings
with the U.S. Securities and Exchange Commission, including its
Annual Report on Form 10-K, under the caption “Risk Factors.”
The forward-looking statements speak only as of the date made and,
other than as required by law, we undertake no obligation to
publicly update or revise any forward-looking statements, whether
as a result of new information, future events, or otherwise.
Contact:Reata Pharmaceuticals,
Inc.(972) 865-2219https://www.reatapharma.com
Investors:Vinny JindalVice President,
Strategy(469) 374-8721ir@reatapharma.com
Media:Matt Middleman, M.D.LifeSci Public
Relations(646)
627-8384matt.middleman@lifescipublicrelations.com
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