Received Priority Review and Acceptance for Filing of the NDA
for Omaveloxolone for Treatment of Patients with Friedreich’s
Ataxia
Provides Update from FDA Mid-Cycle Communication Meeting on
Omaveloxolone for Patients with Friedreich’s Ataxia
Announces Results of New Data and Analyses Submitted to
FDA
Conference Call With Management on August 8, 2022, at 8:30
a.m. ET
Reata Pharmaceuticals, Inc. (Nasdaq: RETA) (“Reata,” the
“Company,” “our,” “us,” or “we”), a clinical-stage
biopharmaceutical company, today announced financial results for
the second quarter of 2022 and provided an update on the Company’s
business operations and clinical development programs.
Recent Company Highlights
Omaveloxolone in Patients with Friedreich’s Ataxia
Following the announcement of the positive data from the MOXIe
Part 2 study in October 2019, the U.S. Food and Drug Administration
(“FDA”) stated that it did not have any concerns with the
reliability of the modified Friedreich’s ataxia rating scale
(“mFARS”) primary endpoint results in the MOXIe Part 2 study and
requested additional evidence of persuasiveness to support a New
Drug Application (“NDA”) filing. We then began a series of
interactions with the FDA to provide additional evidence of
effectiveness to support a single study approval. This ultimately
led to a pre-NDA meeting and subsequent NDA submission in March
2022 after FDA’s review of our Delayed-Start Analysis, which it had
requested.
In May 2022, the FDA accepted for filing our NDA for
omaveloxolone for the treatment of patients with Friedreich’s
ataxia and granted Priority Review. The FDA has granted Fast Track
Designation, Orphan Drug Designation, and Rare Pediatric Disease
Designation to omaveloxolone for the treatment of Friedreich’s
ataxia. The FDA advised us that it is planning to hold an advisory
committee meeting to discuss the application, and our application
has been assigned a Prescription Drug User Fee Act (“PDUFA”) target
action date of November 30, 2022.
We recently completed a mid-cycle communication meeting with the
FDA. The purpose of the mid-cycle communication meeting is for the
FDA to provide the sponsor with an update of the status of the NDA
review, including any issues identified. While we have not received
formal minutes from the FDA, in the preliminary agenda for, and
during, the mid-cycle communication meeting, the FDA stated that it
has not identified any new significant issues, but it continues to
have concerns regarding the strength of the efficacy evidence. The
FDA did not identify any significant clinical safety issues. The
FDA stated that the safety review is ongoing, and they are
continuing to evaluate the cardiac safety of omaveloxolone in
patients with Friedreich’s ataxia. They have not identified any
other major safety concerns at this stage of their review.
During the mid-cycle meeting, we proposed to address FDA’s
concerns in three ways. First, we presented updated results from
the Delayed-Start Analysis using a March 2022 data cut-off, which
contain new, later time points and increased numbers of patients at
later time points than the prior analysis. Second, we proposed to
submit a new propensity-matched matched analysis of MOXIe Extension
data using the largest, most robust Friedreich’s ataxia natural
history study to provide additional clinical data that could be
considered confirmatory evidence. Third, we discussed an additional
NDA amendment containing compelling mechanistic evidence in the
setting of Friedreich’s ataxia’s well-understood disease
pathophysiology, which could also serve as confirmatory evidence.
The FDA acknowledged these data and agreed that we could submit the
updated data to the NDA. We have submitted the following additional
data and analyses to the FDA.
Results from March 2022 Data Cut-Off of
MOXIe Extension
Results of the updated Delayed-Start Analysis from the March
2022 data cut-off demonstrated that the between-group difference in
mFARS observed at the end of the placebo-controlled MOXIe Part 2
treatment period (LS mean difference = -2.17 ± 1.09) was preserved
at MOXIe Extension Week 72 in the delayed-start period (LS mean
difference = -2.91 ± 1.44). Consistent with a persistent treatment
effect on disease, the upper limit of the 90% Confidence Interval
(“CI”) for the difference estimate was less than zero (-0.09),
meeting the threshold for demonstrating significant evidence of
non-inferiority. Additionally, the between group difference in
mFARS was maintained at Extension Week 96, 120, and 144 (LS mean
difference = -2.19 ± 1.38, -2.74 ± 1.26, and -2.58 ± 1.47
respectively), and the threshold for non-inferiority was met at
Extension Week 120 with an upper limit of the 90% CI of -0.106.
Post Hoc Propensity-Matched Analysis of
MOXIe Extension
We recently completed a post hoc analysis comparing the mFARS
progression of omaveloxolone-treated patients in the open-label
MOXIe Extension trial to the progression of propensity
score-matched untreated patients in the largest natural history
study of Friedreich’s ataxia, Clinical Outcome Measures in
Friedreich’s ataxia (“FA-COMS”). All patients enrolled in the MOXIe
Extension study with at least one post-baseline assessment (n=136)
were matched one to one with patients from the FA-COMS study
(n=136) using five baseline characteristics, or covariates,
including sex, baseline age, age of Friedreich’s ataxia onset,
baseline mFARS score, and baseline gait score, which have been
demonstrated to be predictive of disease progression. Demographics
and baseline characteristics were highly comparable between MOXIe
Extension patients and the matched FA-COMS external control
group.
In the Primary Pooled Population (n=136 per group), patients in
the matched FA-COMS group progressed 6.61 mFARS points at Year 3,
whereas patients treated with omaveloxolone in MOXIe Extension
progressed 3.00 points for a difference of -3.61 mFARS points
(nominal p=0.0001). In this analysis, progression in mFARS was 55%
slower in MOXIe Extension patients treated with omaveloxolone
compared to matched untreated patients in the FA-COMS study.
Mechanistic Validation of Nrf2 Target
Biomarkers in Friedreich’s Ataxia
We have provided additional pharmacodynamic information to the
FDA including an integrated and detailed presentation of the
disease pathophysiology of Friedreich’s ataxia, a review of the
available pharmacodynamic data, justification of the relevance of
these data in Friedreich’s ataxia and an explanation of the
relationship between the mechanistic data and the observed
biomarker and clinical treatment effects in patients treated with
omaveloxolone. Substantial evidence demonstrates that Nrf2 levels
and activity are suppressed in cells from patients with
Friedreich’s ataxia and in preclinical animal models of the
disease. Omaveloxolone restores Nrf2 levels and increases the
expression of Nrf2 target genes, including those that encode
ferritin and gamma-glutamyl transferase (“GGT”), in nonclinical
models. Treatment with omaveloxolone in MOXIe Part 1 resulted in
dose-dependent increases in Nrf2 activity, as assessed by serum
ferritin and GGT levels. Data from MOXIe Part 2 showed an
association between omaveloxolone-induced Nrf2 activity and
measures of neurological function, with larger increases in Nrf2
target levels associated with larger improvements in mFARS
scores.
"We look forward to continuing to work with FDA on its review of
our NDA for omaveloxolone for the treatment of patients with
Friedreich’s ataxia, a rare, genetic, debilitating, and
degenerative neuromuscular disorder with no approved therapies,”
said Warren Huff, Reata’s Chief Executive Officer. “We have
submitted these additional data and analyses to the FDA and are
continuing to prepare for the upcoming Advisory Committee
meeting.”
Bardoxolone Methyl in Patients with Chronic Kidney Disease
Caused by Alport Syndrome
We received a Complete Response Letter (“CRL”) from the FDA in
February 2022 with respect to its review of our NDA for bardoxolone
methyl (“bardoxolone”) in the treatment of patients with chronic
kidney disease caused by Alport syndrome. The CRL indicated the FDA
cannot approve the NDA in its present form. We have recently
requested a Type C meeting to discuss the program and continue to
work with the FDA to confirm our next steps on our Alport syndrome
program.
Second Quarter Financial Highlights
Cash and Cash Equivalents
On June 30, 2022, we had cash and cash equivalents and
marketable securities of $481.5 million, as compared to $590.3
million on December 31, 2021.
GAAP and Non-GAAP Research and Development (“R&D”)
Expenses
R&D expenses according to generally accepted accounting
principles in the U.S. (“GAAP”) were $39.3 million for the second
quarter of 2022, as compared to $40.1 million for the same period
of the year prior.
Non-GAAP R&D expenses were $33.0 million for the second
quarter of 2022, as compared to $34.8 million, for the same period
of the year prior.1
GAAP and Non-GAAP General and Administrative (“G&A”)
Expenses
GAAP G&A expenses were $25.1 million for the second quarter
of 2022, as compared to $22.0 million, for the same period of the
year prior.
Non-GAAP G&A expenses were $17.6 million for the second
quarter of 2022, as compared to $14.0 million for the same period
of the year prior.1
GAAP and Non-GAAP Net Loss
The GAAP net loss for the second quarter of 2022, was $73.6
million, or $2.02 per share, on both a basic and diluted basis, as
compared to a GAAP net loss of $72.7 million, or $2.00 per share,
on both a basic and diluted basis, for the same period of the year
prior.
[1] See “Non-GAAP Financial Measures”
below for a description of non-GAAP financial measures and a
reconciliation between GAAP and non-GAAP R&D expenses, GAAP and
non-GAAP G&A expenses, and GAAP and non-GAAP net loss,
respectively, appearing later in the press release.
The non-GAAP net loss for second quarter of 2022, was $49.4
million, or $1.36 per share on both a basic and diluted basis, as
compared to a non-GAAP net loss of $48.0 million, or $1.32 per
share, on both a basic and diluted basis, for the same period of
the year prior.1
Cash Guidance
The Company reaffirms its existing cash & cash equivalents
and marketable debt securities will be sufficient to enable it to
fund operations through the end of 2024.
Non-GAAP Financial Measures
This press release contains non-GAAP financial measures,
including non-GAAP R&D expenses, non-GAAP G&A expenses,
non-GAAP operating expenses, non-GAAP net loss and non-GAAP net
loss per common share – basic and diluted. These measures are not
in accordance with, or an alternative to, GAAP, and may be
different from non-GAAP financial measures used by other
companies.
The Company defines non-GAAP R&D expenses as GAAP R&D
expenses, which exclude stock-based compensation expense; non-GAAP
G&A expenses as GAAP G&A expenses, which exclude
stock-based compensation expense; non-GAAP operating expenses as
GAAP operating expenses, which exclude stock-based compensation
expense; non-GAAP net loss as GAAP net loss, which excludes
stock-based compensation expense and non-cash interest expense from
liability related to sale of future royalties; and non-GAAP net
loss per common share – basic and diluted as GAAP net loss per
common share – basic and diluted, which excludes stock-based
compensation expense and non-cash interest expense from liability
related to sale of future royalties. The Company has excluded the
impact of stock-based compensation expense, which may fluctuate
from period to period based on factors including the variability
associated with performance-based grants of stock options and
restricted stock units and changes in the Company’s stock price,
which impact the fair value of these awards. The Company has
excluded the impact of accreted non-cash interest expense from
liability related to sale of future royalties as it may be
calculated differently from, and therefore may not be comparable
to, peer companies who also provide non-GAAP disclosures. The
Company has excluded the impact of stock-based compensation expense
and non-cash interest expense from liability related to sale of
future royalties because the Company believes its impact makes it
difficult to compare its results to prior periods and anticipated
future periods.
Because management believes certain items, such as stock-based
compensation expense and non-cash interest expense from liability
related to sales of future royalties, can distort the trends
associated with the Company’s ongoing performance, the following
measures are often provided, excluding special items, and utilized
by the Company’s management, analysts, and investors to enhance
consistency and comparability of year-over-year results, as well as
to industry trends, and to provide a basis for evaluating operating
results in future periods: non-GAAP net loss; non-GAAP net loss per
common share – basic and diluted; non-GAAP R&D expenses;
non-GAAP G&A expenses; and non-GAAP operating expenses.
The Company believes the presentation of these non-GAAP
financial measures provides useful information to management and
investors regarding the Company’s financial condition and results
of operations. When GAAP financial measures are viewed in
conjunction with these non-GAAP financial measures, investors are
provided with a more meaningful understanding of the Company’s
ongoing operating performance and are better able to compare the
Company’s performance between periods. In addition, these non-GAAP
financial measures are among those indicators the Company uses as a
basis for evaluating performance, allocating resources, and
planning and forecasting future periods. These non-GAAP financial
measures are not intended to be considered in isolation or as a
substitute for GAAP financial measures. A reconciliation between
these non-GAAP measures and the most directly comparable GAAP
measures is provided later in this press release.
Conference Call Information
Reata’s management will host a conference call on August 8,
2022, at 8:30 am ET. The conference call will be accessible by
dialing (844) 200-6205 (toll-free domestic) or (929) 526-1599
(international) using access code 964090. The webcast link is
https://event.choruscall.com/mediaframe/webcast.html?webcastid=ai4Jk1V6.
Second quarter 2022 financial results to be discussed during the
call will be included in an earnings press release that will be
available on the Company’s website shortly before the call at
https://www.reatapharma.com/investors/ and will be available for 12
months after the call. The audio recording and webcast of the
conference call will be accessible for at least 90 days after the
event at https://www.reatapharma.com/investors/.
About Reata
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, omaveloxolone and
bardoxolone, target the important transcription factor Nrf2 that
promotes the resolution of inflammation by restoring mitochondrial
function, reducing oxidative stress, and inhibiting
pro-inflammatory signaling. Omaveloxolone and bardoxolone 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, our plans to
submit regulatory filings, 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,” “model,” 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) whether regulatory authorities determine that
additional trials or data are necessary in order to obtain
approval; (iv) 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 (v) other factors set forth in Reata’s filings with
the U.S. Securities and Exchange Commission, including its Annual
Report on Form 10-K for the fiscal year ended December 31, 2021,
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.
Three Months Ended
Six Months Ended
June 30
June 30
2022
2021
2022
2021
Consolidated Statements of
Operations
(unaudited)
(in thousands, except share
and per share data)
Collaboration revenue
License and milestone
$
754
$
803
$
1,648
$
1,598
Other revenue
8
1,418
29
1,568
Total collaboration revenue
762
2,221
1,677
3,166
Expenses
Research and development
39,331
40,066
79,136
74,946
General and administrative
25,143
21,998
49,984
42,703
Depreciation
273
287
581
561
Total expenses
64,747
62,351
129,701
118,210
Other income (expense), net
(9,571
)
(13,223
)
(19,343
)
(25,780
)
Loss before taxes on income
(73,556
)
(73,353
)
(147,367
)
(140,824
)
Benefit from (provision for) taxes on
income
1
653
(30
)
669
Net loss
$
(73,555
)
$
(72,700
)
$
(147,397
)
$
(140,155
)
Net loss per share—basic and diluted
$
(2.02
)
$
(2.00
)
$
(4.04
)
$
(3.87
)
Weighted-average number of common shares
used in net loss per share basic and diluted
36,467,802
36,299,735
36,440,364
36,251,948
As of
As of
June 30, 2022
December 31, 2021
(unaudited)
(in thousands)
Condensed Consolidated Balance Sheet
Data
Cash and cash equivalents and marketable
debt securities
$
481,471
$
590,258
Working capital
444,885
542,481
Operating lease right-of-use assets
129,159
126,777
Total assets
631,549
735,016
Liability related to sale of future
royalties, net
382,290
362,142
Operating lease liabilities
142,512
136,033
Deferred revenue
-
1,648
Accumulated deficit
(1,403,028
)
(1,255,631
)
Total stockholders’ equity
$
68,636
$
185,989
Reconciliation of GAAP to Non-GAAP
Financial Measures
The following table presents
reconciliations of non-GAAP financial measures to the most directly
comparable GAAP financial measures (in thousands, except for per
share data):
Three Months Ended
Six Months Ended
June 30
June 30
2022
2021
2022
2021
Reconciliation of GAAP to Non-GAAP
Research and development:
(unaudited)
GAAP Research and development
$
39,331
$
40,066
$
79,136
$
74,946
Less: Stock-based compensation expense
(6,344
)
(5,263
)
(13,951
)
(12,071
)
Non-GAAP Research and development
$
32,987
$
34,803
$
65,185
$
62,875
Reconciliation of GAAP to Non-GAAP
General and administrative:
GAAP General and administrative
$
25,143
$
21,998
$
49,984
$
42,703
Less: Stock-based compensation expense
(7,520
)
(7,981
)
(15,357
)
(15,852
)
Non-GAAP General and administrative
$
17,623
$
14,017
$
34,627
$
26,851
Reconciliation of GAAP to Non-GAAP
Operating expenses:
GAAP Operating expense
$
64,747
$
62,351
$
129,701
$
118,210
Less: Stock-based compensation expense
(13,864
)
(13,244
)
(29,308
)
(27,923
)
Non-GAAP Operating expense
$
50,883
$
49,107
$
100,393
$
90,287
Reconciliation of GAAP to Non-GAAP Net
loss:
GAAP Net loss
$
(73,555
)
$
(72,700
)
$
(147,397
)
$
(140,155
)
Add: Stock-based compensation expense
13,864
13,244
29,308
27,923
Add: Non-cash interest expense from
liability related to sale of future royalties
10,277
11,429
20,148
22,354
Non-GAAP Net loss
$
(49,414
)
$
(48,027
)
$
(97,941
)
$
(89,878
)
Reconciliation of GAAP to Non-GAAP Net
loss per common share-basic and diluted:
GAAP Net loss per common share-basic and
diluted
$
(2.02
)
$
(2.00
)
$
(4.04
)
$
(3.87
)
Add: Stock-based compensation expense
0.38
0.36
0.80
0.77
Add: Non-cash interest expense from
liability related to sale of future royalties
0.28
0.32
0.55
0.62
Non-GAAP Net loss per common share-basic
and diluted
$
(1.36
)
$
(1.32
)
$
(2.69
)
$
(2.48
)
Three Months Ended
June 30, 2022
March 31, 2022
December 31, 2021
September 30, 2021
Reconciliation of GAAP to Non-GAAP
Operating expenses
(unaudited)
GAAP Operating expenses
$
64,747
$
64,953
$
72,503
$
65,486
Less: Stock-based compensation expense
(13,864
)
(15,444
)
(15,226
)
(13,657
)
Non - GAAP Operating expenses
$
50,883
$
49,509
$
57,277
$
51,829
Reconciliation of GAAP to Non-GAAP Net
loss
GAAP Net loss
$
(73,555
)
$
(73,842
)
$
(85,385
)
$
(71,846
)
Add: Stock-based compensation expense
13,864
15,444
15,226
13,657
Add: Non-cash interest expense from
liability related to sale of future royalties
10,277
9,871
12,376
11,958
Non-GAAP Net loss
$
(49,414
)
$
(48,527
)
$
(57,783
)
$
(46,231
)
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Reata Pharmaceuticals, Inc. (972) 865-2219
https://www.reatapharma.com/
Investor Relations & Media Relations: John Hunter
ir@reatapharma.com Wendy Segal media@reatapharma.com
https://www.reatapharma.com/contact-us/
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