REATA ANNOUNCES INITIATION OF PHASE 2/3 STUDY OF BARDOXOLONE METHYL IN THE
TREATMENT OF CHRONIC KIDNEY DISEASE DUE TO ALPORT SYNDROME
IRVING, Texas, February 23rd, 2017– Reata Pharmaceuticals, Inc. (NASDAQ:RETA) (“Reata” or the “Company”)
today announced the initiation of patient screening in a Phase 2/3 trial to evaluate bardoxolone methyl (“bard”) in
patients with chronic kidney disease (“CKD”) caused by Alport syndrome. The purpose of this study is to determine
the safety and efficacy of bard in Alport syndrome patients, and to determine if Alport syndrome patients experience
improvements in kidney function similar to those observed in multiple, previous trials of bard in patients with other forms
of CKD. Reata expects data from the Phase 2 portion of the trial to be available, and to decide on entering the Phase
3 portion, by year-end 2017.
The Alport Syndrome Foundation’s Executive Director Gina Parziale said, “As there are currently no FDA approved
treatments for those with Alport Syndrome, the Alport Syndrome Foundation encourages the development of therapies
that will delay or prevent the need for dialysis and transplantation. We are grateful to Reata for engaging us in this
process and for recognizing the crucial role of the patient perspective.”
"Based on our extensive clinical experience with bardoxolone methyl in patients with diabetic CKD, as well as in our
ongoing Phase 2 and Phase 3 trials for bardoxolone in other orphan diseases, we hope to demonstrate that
bardoxolone methyl can serve as a meaningful new treatment option for patients with Alport syndrome," said Warren
Huff, Reata’s Chief Executive Officer and President.
Overview of Clinical Trial Program in Alport Syndrome
The Phase 2 portion of the study is open-label and will enroll up to 30 patients from 12 to 60 years old with estimated
glomerular filtration rates (eGFR) between 30 to 90 mL/min/1.73 m2. Fifteen patients with microalbuminuria will receive
up to 20 mg of bard once daily, and 15 patients with macroalbuminuria will receive up to 30 mg of bard once daily. The
primary endpoint for the Phase 2 portion of the study is change in eGFR at week 12 compared to baseline.
The Phase 3 portion is designed to support regulatory approval of bard. This portion will be double-blind and placebocontrolled and will randomize approximately 180 patients on a 1:1 basis to once-daily, oral bard or placebo. Similar to
the Phase 2 portion of the trial, the study will assess dose escalation of bard from 5 mg to a maximum daily dose of 20
mg or 30 mg based on baseline proteinuria at randomization. The primary efficacy endpoint is the on-treatment change
from baseline in eGFR in bardoxolone methyl-treated patients relative to placebo after 48 weeks. The key secondary
endpoints will be the change from baseline in eGFR following a 4-week withdrawal of drug after one and two years of
treatment. Based on FDA guidance, if the trial is positive, the year one off-treatment data could support accelerated
approval under subpart H of the Food, Drug, and Cosmetic Act, and the year two off-treatment data could support full
approval.
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About Alport Syndrome
Alport syndrome is a rare, genetic disease caused by mutations in the genes encoding type IV collagen, a major
structural component of the glomerular basement membrane (“GBM”) in the kidney. The abnormal expression of type
IV collagen causes loss of GBM integrity, abnormal leakage of proteins such as albumin through the GBM, and
excessive reabsorption of protein in the proximal tubules of the kidney. Like other forms of CKD, excessive reabsorption
of protein in the tubules induces oxidative stress, chronic inflammation, and renal interstitial inflammation and fibrosis.
Alport syndrome affects approximately 12,000 people in the United States and 40,000 globally. Almost all patients with
Alport syndrome develop end-stage renal disease (“ESRD”), and approximately 50% of male patients require dialysis
or kidney transplant by the age of 25. There are currently no approved therapies to treat Alport syndrome.
About Bardoxolone Methyl
Bardoxolone methyl is an experimental, oral, once-daily activator of Nrf2, a transcription factor that induces molecular
pathways that promote the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress,
and inhibiting pro-inflammatory signaling. Bardoxolone methyl is currently being studied in CATALYST, a Phase 3
study for the treatment of connective tissue disease associated pulmonary arterial hypertension (CTD-PAH).
About Reata Pharmaceuticals, Inc.
Reata Pharmaceuticals, Inc., 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 methyl and
omaveloxolone) target an important transcription factor, called Nrf2, to restore mitochondrial function, reduce oxidative
stress, and resolve inflammation.
Forward-Looking Statements
This press release includes certain disclosures which 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, our ability to obtain and retain regulatory approval of our
product candidates, estimates of our expenses and our needs for additional financing, and our ability to obtain additional
financing for our product development activities and existing and future clinical trials and pre-clinical programs. 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
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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 are set forth in Reata’s filings with the
U.S. Securities and Exchange Commission, including its Registration Statement on Form S-1, as amended from time
to time, 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) 865-2219
[email protected]
http://news.reatapharma.com
Investor Relations:
The Trout Group
Lee M. Stern, CFA
(646) 378-2922
[email protected]
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chronic kidney disease due to Alport syndrome: interim results from the ATHENA study, a prospectively designed
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Aminzadeh MA, Reisman SA, Vaziri ND, et al. The synthetic triterpenoid RTA dh404 (CDDO-dhTFEA) restores
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Camer D, Yu Y, Szabo A, et al. Bardoxolone methyl prevents the development and progression of cardiac and renal
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