AURORA Phase III Trial with Voclosporin Has
Been Initiated
Cash of $202.1 million as of March 31, 2017,
Providing Resources to Fund Company Through 2020
AURA-LV Phase IIb Trial Met Key 48-Week
Endpoints, Achieving Highest Complete Remission Rate of Any Global
Lupus Nephritis Study
Cash of $9.7 million used for operating
activities in Q1 2017
Aurinia Pharmaceuticals Inc. (NASDAQ:AUPH) (TSX:AUP) (“Aurinia”
or the “Company”) has released its financial results for the first
quarter ended March 31, 2017. Amounts, unless specified otherwise,
are expressed in U.S. dollars.
“I am proud of the important clinical, regulatory and financial
milestones our team has successfully achieved in our first quarter
this year. We released positive 48-week results from our Phase IIb
AURA-LV (“AURA”) trial of voclosporin, which demonstrated
significantly improved complete remission rates in patients
suffering from lupus nephritis,” said Richard Glickman, Aurinia’s
CEO and Chairman of the Board. “We also believe we have a clear
path forward with regulators to develop voclosporin in major
markets and have successfully funded the Company’s initiated Phase
III lupus nephritis clinical trial (“AURORA”) and operations
through 2020. Furthermore, based on the results of our AURA trial
and regulatory feedback, we have moved diligently into our AURORA
trial with several sites initiated and currently screening
patients. Our clinical team is focused on continuing to initiate
sites with an aggressive patient recruitment program. The AURORA
trial design is consistent with that of the recently completed AURA
clinical trial. We believe that the totality of data from both the
AURORA and AURA trials will ultimately serve as the basis for a New
Drug Application (“NDA”) submission as well as regulatory
submissions in other major global markets.”
Recent operational highlights
48-Week AURA-LV Data presented in Late Breaker Presentation
at National Kidney Foundation 2017 Scientific Clinical
Meeting
On April 20, 2017 we announced additional 48-week results from
the global AURA study in lupus nephritis (“LN”) during the National
Kidney Foundation 2017 Spring Clinical Meetings in Orlando, FL. In
addition to the trial meeting its complete and partial remission
(“CR”/”PR”) endpoints at 48 weeks, all pre-specified secondary
endpoints that have been analyzed to date were also met at 48
weeks. These pre-specified endpoints include: time to CR and PR
(speed of remission); reduction in Systemic Lupus Erythematosus
Disease Activity Index or SLEDAI score; and reduction in urine
protein creatinine ratio (“UPCR”) over the 48-week treatment
period. Notably, of the patients that achieved CR at 24 weeks, in
the low-dose voclosporin group, 100% remained in CR at 48 weeks,
which demonstrates durability of clinical response. Proteinuria
levels and reduction in SLEDAI scores, which include non-renal
measures of lupus activity, also continued to significantly
separate over time versus the control group. Additional analyses
are ongoing and will be presented at future medical and scientific
meetings.
No unexpected safety signals were observed and voclosporin was
generally well-tolerated, with the nature of adverse events
consistent with what is expected of patients suffering from highly
active LN while undergoing immunomodulation therapy. In the
voclosporin arms, the renal function as measured by eGFR was stable
and not significantly different from the control arm during the
48-week treatment period. Mean blood pressure was also similar
between all treatment groups.
The 24 and 48-week efficacy results are summarized below:
Endpoint
Treatment 24 weeks
P-value* 48 weeks
P-value* Complete Remission (CR)
23.7mg VCS BID 33%
p=.045 49%
p<.001 39.5mg VCS BID
27% p=.204 40%
p=.026 Control Arm
19% NA 24%
NA
Partial Remission (PR) 23.7mg VCS
BID 70%
p=.007 68%
p=.007 39.5mg VCS BID 66%
p=.024 72% p=.002
Control Arm 49%
NA 48% NA
Time to CR (TTCR) [median] 23.7mg VCS BID
19.7 weeks
p<.001 19.7 weeks
p<.001 39.5mg VCS BID 23.4
weeks p=.001 23.4 weeks
p<.001 Control Arm
NA NA
NA NA
Time to PR (TTPR) [median]
23.7mg VCS BID 4.1 weeks
p=.002 4.3 weeks
p=.005 39.5mg VCS BID
4.4 weeks P=.003
4.4 weeks p=.002 Control
Arm 6.6 weeks NA
6.6 weeks NA
SLEDAI Reduction
(non-renal lupus) 23.7mg VCS BID -6.3
p=.003 -7.9
p<.001 39.5mg VCS BID
-7.1 p=.003 -8.3
p<.001 Control Arm
-4.5 NA
-5.3 NA
Reduction in UPCR 23.7mg VCS
BID -3.769 mg/mg
p<.001 -3.998 mg/mg
p<.001 39.5mg VCS BID
-2.792 mg/mg p=.006
-2.993 mg/mg p=.008
Control Arm -2.216 mg/mg
NA -2.384 mg/mg NA
Note: “VCS” means voclosporin
*All p-values are vs control
Regulatory pathway forward
On April 7, 2017 we announced the outcome of discussions with
both the European Medicines Agency (EMA) and the Pharmaceutical and
Medical Devices Agency (PMDA) in Japan regarding the development of
voclosporin for the treatment of active LN. Pursuant to these
discussions, we believe that the confirmatory data that can be
generated from the AURORA trial and the recently completed AURA
trial should support regulatory submissions in the US, Europe and
Japan.
The AURORA trial will be a global 52-week double-blind, placebo
controlled study of approximately 320 patients. Patients will be
randomized 1:1: to either of 23.7mg voclosporin BID and
mycophenolate mofetil (MMF) or MMF and placebo, with both arms
receiving a stringent oral corticosteroid taper. As in AURA, the
study population will be comprised of patients with biopsy-proven
active LN who will be evaluated on the primary efficacy endpoint of
complete remission, or renal response, at 52 weeks, a composite
which includes:
- UPCR of ≤0.5mg/mg
- Normal, stable renal function (≥60
mL/min/1.73m2 or no confirmed decrease from baseline in eGFR of
>20%)
- Presence of sustained, low dose
steroids (≤10mg prednisone from week 16-24)
- No administration of rescue medications
throughout the treatment period
Key Developments in First Quarter, 2017
Completion of Public Offering
On March 20, 2017 we announced the closing of an underwritten
public offering of 25.64 million common shares. The shares were
sold at a public offering price of $6.75 per share. The gross
offering proceeds to the Company from this Offering were US$173.1
million. Expenses of the offering including underwriting
commissions and other offering expenses were $10.8 million.
AURA 48-Week Results
On March 1, 2017, we announced top-line results from the AURA
trial. At 48 weeks, the trial met the CR/PR endpoints,
demonstrating statistically significant greater CR and PR in
patients in both low dose (23.7mg of voclosporin twice daily
(p<.001)) and high dose (39.5mg twice daily (p=.026)) cohorts
versus the control group. No unexpected safety signals were
observed and there were no additional deaths in the voclosporin
treated patients; however, there were three deaths and one
malignancy reported in the control arm after completion of the
study treatment period.
Japanese Phase I Ethnic Bridging Study for
Voclosporin
On February 14, 2017, we announced results of a supportive Phase
I safety, pharmacokinetic (“PK”) and pharmacodynamics (“PD”) study
in healthy Japanese patients, which supports further development of
voclosporin in this patient population. Based on evaluations
comparing the Japanese ethno-bridging data vs. previous PK and PD
studies in non-Japanese patients, voclosporin demonstrated no
statistically significant differences in exposure with respect to
Area Under the Curve measurements. Furthermore, the PK parameters
in Japanese patients were generally consistent with previously
evaluated PK parameters in non-Japanese volunteers. There were no
unusual or unexpected safety signals in the study.
Financial Results for the First Quarter Ended March 31,
2017
As a result of completing the public offering on March 20, 2017,
Aurinia had cash, cash equivalents and short term investments of
$202.1 million as at March 31, 2017 compared to $39.6 million as at
December 31, 2016. We believe, based on our current plans, that we
have the financial resources to complete the AURORA trial and fund
operations through 2020.
Cash used in operating activities for the three months ended
March 31, 2017 was $9.7 million. Cash provided by financing
activities was $172.2 million comprised of net proceeds of $162.3
million from the public offering and $9.9 million from the exercise
of warrants and stock options during the three month period ended
March 31, 2017.
For the first quarter ended March 31, 2017, we reported a
consolidated net loss of $51.9 million or $0.92 per common share.
This loss included a non-cash increase of $40.8 million related to
the estimated fair value quarterly adjustment of derivative warrant
liabilities at March 31, 2017. After adjusting for this non-cash
impact, the net loss from operations was $11.2 million or $0.20 per
common share.
This compared to a consolidated net loss of $4.3 million or
$0.13 per common share, which included a non-cash decrease on
revaluation of derivative warrant liability of $664,000 at March
31, 2016. After adjustment for the non-cash impact of the
revaluation, the net loss from operations for the three months
ended March 31, 2016 was $4.9 million or $0.15 per common
share.
The change in the revaluation of the derivative warrant
liabilities is primarily driven by the change in our share price.
Our share price was significantly higher at March 31, 2017 compared
to December 31, 2016 which resulted in a large fair value
adjustment. These derivative warrant liabilities will ultimately be
transferred to equity upon the exercise or expiry of these warrants
and therefore are non-cash adjustments.
We incurred net research and development expenditures of $7.3
million for the first quarter ended March 31, 2017, as compared to
$3.3 million for the same period in 2016. The increase in research
and development expenditures in 2017 reflected initiation costs,
including activities such as clinical site selections and
regulatory submissions and drug manufacturing costs related to the
AURORA trial and completion costs associated with the AURA
trial.
We incurred corporate, administration and business development
costs of $3.4 million for the first quarter ended March 31, 2017,
as compared with $1.2 million for the same period in 2016. These
costs included a non-cash stock compensation expense of $1.1
million in 2017 compared to $261,000 in 2016 primarily due to an
increase in the number of options granted in 2017 compared to the
same period in 2016.
This press release should be read in conjunction with the our
unaudited interim condensed consolidated financial statements and
the MD&A for the first quarter ended March 31, 2017 which are
accessible on Aurinia's website at www.auriniapharma.com, on SEDAR
at www.sedar.com or on EDGAR at www.sec.gov/edgar.
About AURORA
The AURORA trial is a 52-week global double-blind placebo
controlled phase III trial that will compare the efficacy of one
dose of voclosporin (23.7mg BID) or placebo added to current
standard of care of mycophenolate mofetil (MMF, also known as
CellCept®) in achieving renal response (formerly referred to as
complete remission) in patients with active LN. Both arms will also
receive corticosteroids as part of background therapy. These
corticosteroids will be stringently and aggressively tapered over
the course of the trial.
About AURA-LV
The AURA–LV trial (Aurinia Urinary Protein Reduction in Active
Lupus with Voclosporin) was a 48-week trial comparing the efficacy
of two doses of voclosporin added to current standard of care of
MMF against standard of care with placebo in achieving CR in
patients with active LN. All arms also received low doses of
corticosteroids as background therapy. 265 patients were enrolled
at centers in 20 countries worldwide. On entry to the study,
patients were required to have a diagnosis of LN according to
established diagnostic criteria (American College of Rheumatology)
and clinical and biopsy features indicative of highly active
nephritis. The 24-week primary and secondary endpoints were
released in Q3 2016 with top-line 48-week results announced in Q1
2017. The 48-week data was presented at a late-breaking
presentation at National Kidney Foundation (NKF) Spring Clinical
Meeting which took place April 18-22 in Orlando, FL.
About Voclosporin
Voclosporin, an investigational drug, is a novel and potentially
best-in-class calcineurin inhibitor (“CNI”) with clinical data in
over 2,200 patients across indications. Voclosporin is an
immunosuppressant, with a synergistic and dual mechanism of action
that has the potential to improve near- and long-term outcomes in
LN when added to standard of care (MMF). By inhibiting calcineurin,
voclosporin blocks IL-2 expression and T-cell mediated immune
responses. It is made by a modification of a single amino acid of
the cyclosporine molecule which has shown a more predictable
pharmacokinetic and pharmacodynamic relationship, an increase in
potency, an altered metabolic profile, and potential for flat
dosing. The Company anticipates that upon regulatory approval,
patent protection for voclosporin will be extended in the United
States and certain other major markets, including Europe and Japan,
until at least October 2027 under the Hatch-Waxman Act and
comparable laws in other countries.
About Lupus Nephritis (LN)
LN in an inflammation of the kidney caused by Systemic Lupus
Erythematosus (“SLE”) and represents a serious progression of SLE.
SLE is a chronic, complex and often disabling disorder and affects
more than 500,000 people in the United States (mostly women). The
disease is highly heterogeneous, affecting a wide range of organs
& tissue systems. It is estimated that as many as 60% of all
SLE patients have clinical LN requiring treatment. Unlike SLE, LN
has straightforward disease outcomes where an early response
correlates with long-term outcomes, measured by proteinuria. In
patients with LN, renal damage results in proteinuria and/or
hematuria and a decrease in renal function as evidenced by reduced
estimated glomerular filtration rate (eGFR), and increased serum
creatinine levels. LN is debilitating and costly and if poorly
controlled, LN can lead to permanent and irreversible tissue damage
within the kidney, resulting in end-stage renal disease (“ESRD”),
thus making LN a serious and potentially life-threatening
condition.
About Aurinia
Aurinia is a clinical stage biopharmaceutical company focused on
developing and commercializing therapies to treat targeted patient
populations that are suffering from serious diseases with a high
unmet medical need. The Company is currently developing
voclosporin, an investigational drug, for the treatment of LN. The
Company is headquartered in Victoria, BC and focuses its
development efforts globally.
Forward-Looking Statements
This press release contains forward-looking statements,
including statements around our analysis, assessment and
conclusions around the future development and commercial potential
of voclosporin; our belief that we have a clear path forward with
regulators to develop voclosporin in major markets; our belief that
we have fully funded our AURORA Phase III clinical trial and
operations through 2020; our belief that our prior clinical trial
results will serve as the basis for a NDA submission and regulatory
submissions in major global markets; and our expectation that
patent protection for voclosporin will be extended in the United
States and certain other major markets, including Europe and Japan,
until at least October 2027; and the timing of future clinical
trials; summary statements relating to results of the past
voclosporin trials; the timing of commencement and completion of
clinical trials; and plans and objectives of management.
It is possible that such results or conclusions may change based
on further analyses of these data. Words such as "plans,"
"intends," “may,” "will," "believe," and similar expressions are
intended to identify forward-looking statements. These
forward-looking statements are based upon Aurinia’s current
expectations. Forward-looking statements involve risks and
uncertainties. Aurinia’s actual results and the timing of events
could differ materially from those anticipated in such
forward-looking statements as a result of these risks and
uncertainties, which include, without limitation, the risk that
Aurinia’s analyses, assessment and conclusions of the results of
the future development and commercial potential of voclosporin set
forth in this release may change based on further analyses of such
data, and the risk that Aurinia’s clinical studies for voclosporin
may not lead to regulatory approval. These and other risk factors
are discussed under "Risk Factors" and elsewhere in Aurinia’s
Annual Information Form for the year ended December 31, 2016 filed
with Canadian securities authorities and available at www.sedar.com
and on Form 40-F with the U.S. Securities Exchange Commission and
available at www.sec.gov, each as updated by subsequent filings,
including filings on Form 6-K. Aurinia expressly disclaims any
obligation or undertaking to release publicly any updates or
revisions to any forward-looking statements contained herein to
reflect any change in Aurinia's expectations with regard thereto or
any change in events, conditions or circumstances on which any such
statements are based.
Such forward-looking statements involve known and unknown risks,
uncertainties, and other factors that may cause our actual results,
performance, or achievements to differ materially from any further
results, performance or achievements expressed or implied by such
forward-looking statements. Important factors that could cause such
differences include, among other things, the following:
- difficulties, delays, or failures we
may experience in the conduct of its planned AURORA clinical
trial;
- difficulties we may experience in
completing the development and commercialization of
voclosporin;
Although we believe that the expectations reflected in the
forward-looking statements are reasonable, we cannot guarantee
future results, levels of activity, performance or achievements.
These forward-looking statements are made as of the date hereof and
will only be updated in accordance with applicable law.
We seek Safe Harbor.
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Aurinia Pharmaceuticals Inc.Investors &
Media:Celia EconomidesHead of IR &
Communicationsceconomides@auriniapharma.comorChief Financial
Officer:Dennis Bourgeault, 780-643-2260780-484-4105
(fax)dbourgeault@auriniapharma.com