- Efficacy endpoints reordered and
co-primary endpoints established -- Ongoing trial,
if successful, expected to serve as single pivotal study for BLA
submission -
Proteon Therapeutics Inc. (Nasdaq:PRTO), a company developing
novel, first-in-class therapeutics to address the medical needs of
patients with kidney and vascular diseases, today announced its
financial results for the year ended December 31, 2016, and
provided an update on the PATENCY-2 clinical trial and other recent
business highlights.
“The results from PATENCY-1 provided us critical
insights into studying vonapanitase that have allowed us to
strengthen the PATENCY-2 trial,” said Timothy Noyes, President and
Chief Executive Officer of Proteon Therapeutics. “We are encouraged
by the high degree of engagement by the FDA and appreciate their
continued guidance with respect to our ongoing Phase 3 trial.”
Clinical Trial Update for
PATENCY-2
Important changes to PATENCY-2,
the second Phase 3 clinical study of investigational vonapanitase.
After announcing top-line results from the first Phase 3 clinical
trial, PATENCY-1, in December 2016, Proteon started discussions
with the U.S. Food and Drug Administration, or FDA, regarding
changes to the PATENCY-2 trial. Following the Company’s review of
the complete data sets from the PATENCY-1 trial and discussions
with the FDA, Proteon amended the protocol for the PATENCY-2 trial
as follows:
- The protocol amendment reordered the existing endpoints for the
PATENCY-2 trial, establishing secondary patency and fistula use for
hemodialysis as co-primary endpoints.
- Secondary patency is defined as the
length of time from surgical creation until fistula abandonment
(final failure), the same definition used in PATENCY-1 in which
secondary patency served as the secondary endpoint. In PATENCY-1,
vonapanitase-treated patients experienced a 34% reduction in the
risk of secondary patency loss over one year, compared to placebo
(p=0.048). At the end of one year, 74% of vonapanitase-treated
patients maintained secondary patency, compared to 61% of
placebo-treated patients.
- Use for hemodialysis is
defined as use of the fistula for hemodialysis for at least 90 days
or, if hemodialysis was not initiated at least 90 days prior to the
patient’s last visit, for at least 30 days prior to the patient’s
last visit and in use at the patient’s last visit. This is the same
definition used in PATENCY-1. In PATENCY-1, 64% of
vonapanitase-treated patients used their fistula for hemodialysis,
compared to 44% of placebo-treated patients (p=0.006), a 45%
relative increase.
- The protocol amendment also increased the planned enrollment
for this trial from 300 to 500 patients, which provides greater
than 90% power to detect the differences observed in the PATENCY-1
trial with a p-value ≤0.05 for each of the co-primary endpoints.
- Based on the Company’s interactions with the FDA, Proteon
believes that, if the PATENCY-2 trial is successful in showing
statistical significance (p≤0.05) on each of the co-primary
endpoints, the PATENCY-2 trial together with data from previously
completed studies would provide the basis for a Biologics License
Application, or BLA, submission as a single pivotal study, in which
case no additional studies would need to be conducted.
As of February 28, 2017, Proteon had enrolled
315 patients in PATENCY-2 at approximately 40 centers in the U.S.
and Canada. Proteon expects to complete enrollment in the PATENCY-2
trial during the fourth quarter of 2017 and to report top-line data
in the fourth quarter of 2018. If the PATENCY-2 trial is
successful, Proteon expects to submit a BLA in 2019.
2016 Highlights
Enrollment continues according to plan
in PATENCY-2. PATENCY-2 is a multicenter, randomized,
double-blind, placebo-controlled study expected to enroll 500
patients in the United States and Canada with chronic kidney
disease (CKD) undergoing surgical creation of a radiocephalic
arteriovenous fistula for hemodialysis. In February 2017, the
Company achieved its guidance from early 2016 to enroll 300
patients by the end of the first quarter of 2017. Full enrollment
of 500 patients is expected in the fourth quarter of this year. The
study’s co-primary endpoints are secondary patency and fistula use
for hemodialysis, each of which demonstrated improvements in
PATENCY-1.
Topline clinical results announced in
December 2016 for PATENCY-1, the first Phase 3 clinical
study of vonapanitase. PATENCY-1 was a multicenter, randomized,
double-blind, placebo-controlled study, which enrolled 313 patients
in the United States with CKD undergoing surgical creation of a
radiocephalic fistula for hemodialysis. While the trial did not
show statistical significance for the primary efficacy endpoint,
primary unassisted patency, vonapanitase demonstrated improvements
in other efficacy endpoints, including secondary patency and
fistula use for hemodialysis.
PATENCY-1 clinical results were
presented in February 2017 at the American Society of
Diagnostic and Interventional Nephrology 13th Annual Scientific
meeting. The presentation of Phase 3 results titled, “PATENCY-1:
Phase 3 Outcomes of Vonapanitase on Radiocephalic AVF Outcomes,”
was given by Timmy Lee, M.D., Associate Professor of Medicine in
the Division of Nephrology at the University of Alabama at
Birmingham and an investigator in PATENCY-1. The oral presentation,
which detailed the previously announced study results, was given
February 11, 2017.
The Company initiated enrollment in a
Phase 1 clinical study of vonapanitase in patients with peripheral
artery disease (PAD). The multicenter, randomized,
double-blind, placebo-controlled Phase 1 dose escalation study is
expected to enroll in 2017 up to 24 symptomatic PAD patients being
treated with balloon angioplasty of an artery below the knee and to
follow each patient for up to seven months. Immediately following
successful angioplasty, vonapanitase or placebo is delivered to the
arterial wall using the Mercator MedSystems Bullfrog®
Micro-Infusion Catheter. The primary outcome measure of the study
will be safety and the secondary outcome measure will be technical
feasibility of drug delivery via the catheter.
Board of Directors strengthened with the
addition of commercial and executive expertise. In the
fourth quarter of 2016, the Company appointed Paul J. Hastings as
Chairman of its Board of Directors. Mr. Hastings has over
three decades of operations experience in the biopharmaceutical
industry, including roles as Chief Executive Officer at multiple
public companies. Mr. Hastings is President and Chief Executive
Officer of OncoMed Pharmaceuticals and Chairman of its Board since
2013.
Key Milestones for 2017
- Complete enrollment of 500 patients in PATENCY-2 in the fourth
quarter of 2017.
- Enroll 24 patients in the PAD Phase 1 trial before the end of
2017.
Upcoming Events
- Presentation at the (i) 10th Congress of the Vascular Access
Society April 5-8 in Ljubljana, Slovenia (ii) National Kidney
Foundation 2017 Spring Clinical Meetings April 18-22 in Orlando, FL
and (iii) The Charing Cross Symposium (CX 2017) April 25-28 in
London, England.
- Presentations at the (i) Oppenheimer 27th Annual Healthcare
Conference March 21-22 in New York City, NY, (ii) Deutsche Bank
42nd Annual Health Care Conference May 3-4 in Boston, MA and (iii)
JMP Securities Life Science Conference June 20-21 in New York City,
NY.
Full-Year 2016 Financial
Results
Cash position: Cash, cash
equivalents and available-for-sale investments totaled $41.3
million as of December 31, 2016, compared to $65.3 million as of
December 31, 2015. The decrease was driven by operational costs for
2016.
R&D expenses: Research and
development expenses for 2016 were $18.9 million as compared to
$12.4 million for 2015. The increase in R&D expenses was due
primarily to increased expenses for manufacturing pre-validation
and validation efforts; increased external clinical expenses
related to ongoing radiocephalic AVF Phase 3 clinical trials and
our PAD Phase 1 clinical trials; and increased personnel costs.
G&A expenses: General and
administrative expenses for 2016 were $9.8 million as compared to
$8.5 million for 2015. The increase in G&A expenses was due
primarily to higher personnel costs.
Other expense: Other expense for 2016 was $14
thousand as compared to $0.7 million for 2015. Other expense in
2016 and 2015 included non-cash changes in the Swiss Franc
denominated currency the Company held as of December 31, 2016 and
2015 and the fair value associated with the forward foreign
currency contracts the Company entered into in June 2015.
Net loss: Net loss for 2016 was
$28.5 million as compared to $21.4 million for 2015. Net loss
included stock-based compensation expense of $3.3 million for 2016
and $2.2 million for 2015.
Financial guidance: The Company
expects that its cash, cash equivalents and available-for-sale
investments will be sufficient to fund its operations into the
third quarter of 2018.
Conference Call and Webcast regarding Clinical Trial
Update for PATENCY-2
Proteon is hosting a webcast and conference call today, March
16, at 8:00 a.m. ET to discuss changes to the PATENCY-2 trial. To
access the conference call, please dial (844) 263-8297 (U.S.) or
(478) 219-0006 (international) with Conference ID # 89059698. A
live, listen-only webcast will also be accessible on the Investors
& Media page of www.proteontx.com. A replay of the conference
call will be available for two weeks on the Proteon website or by
dialing (855) 859-2056 (U.S.) or (404) 537-3406 (international) and
using Conference ID # 89059698.
About Vonapanitase
Vonapanitase is an investigational drug intended to improve
hemodialysis vascular access outcomes. Vonapanitase is applied in a
single administration and is currently being studied in a Phase 3
program in patients with CKD undergoing surgical creation of a
radiocephalic arteriovenous fistula for hemodialysis. Vonapanitase
has received fast track and orphan drug designations from the FDA,
and orphan medicinal product designation from the European
Commission, for hemodialysis vascular access indications. In
addition, vonapanitase may have other surgical and endovascular
applications in diseases or conditions in which vessel injury leads
to blockages in blood vessels and reduced blood flow. Proteon is
currently conducting a Phase 1 clinical trial of vonapanitase in
patients with peripheral artery disease (PAD).
About Proteon Therapeutics
Proteon Therapeutics is committed to improving the health of
patients with kidney and vascular diseases through the development
of novel, first-in-class therapeutics. Proteon's lead product
candidate, vonapanitase, is an investigational drug intended to
improve hemodialysis vascular access outcomes. Proteon is
evaluating vonapanitase in patients with CKD undergoing surgical
creation of a radiocephalic arteriovenous fistula. Proteon is
currently enrolling patients in a Phase 3 clinical trial,
PATENCY-2. Proteon is also evaluating vonapanitase in a Phase 1
clinical trial in patients with PAD. For more information, please
visit www.proteontx.com.
Cautionary Note Regarding
Forward-Looking Statements
This press release contains statements that are, or may be
deemed to be, "forward-looking statements." In some cases, these
forward-looking statements can be identified by the use of
forward-looking terminology, including the terms “estimates,”
“anticipates,” "expects,” “plans,” "intends,” “may,” or “will,” in
each case, their negatives or other variations thereon or
comparable terminology, although not all forward-looking statements
contain these words. These statements, including the number of
patients to be enrolled in and the timing of enrollment in the
Company’s ongoing and planned clinical trials of vonapanitase, when
the Company expects to report top-line data from the PATENCY-2
trial, whether and when we may submit a BLA in the United States,
whether additional studies will be necessary to support a BLA
submission as a single pivotal trial, whether and when we may be
able to successful complete drug substance validation runs, the
potential treatment of renal and vascular diseases with
vonapanitase, the effect or benefit of vonapanitase in patients
with CKD, whether vonapanitase improves fistula patency or use for
hemodialysis, the potential surgical and endovascular applications
for vonapanitase, including PAD, the sufficiency of the Company’s
cash, cash-equivalents and available-for-sale investments to fund
the Company’s operations into the third quarter of 2018, and those
relating to future events or our future financial performance or
condition, involve substantial known and unknown risks,
uncertainties and other important factors that may cause our actual
results, levels of activity, performance or achievements to differ
materially from those expressed or implied by these forward-looking
statements. These risks, uncertainties and other factors, including
whether our cash resources will be sufficient to fund our operating
expenses and capital expenditure requirements for the period
anticipated; whether data from early nonclinical or clinical
studies will be indicative of the data that will be obtained from
future clinical trials; whether vonapanitase will advance through
the clinical trial process on the anticipated timeline and warrant
submission for regulatory approval; whether such a submission would
receive approval from the U.S. Food and Drug Administration or
equivalent foreign regulatory agencies on a timely basis or at all;
and whether we can successfully commercialize and market our
product candidates, are described more fully in our Annual Report
on Form 10-K for the year ended December 31, 2016, as filed with
the Securities and Exchange Commission (“SEC”) on March 16, 2017,
and our subsequent Quarterly Reports on Form 10-Q and Current
Reports on Form 8-K, as filed with the SEC, particularly in the
sections titled “Risk Factors” and “Management's Discussion and
Analysis of Financial Condition and Results of Operations.” In
light of the significant uncertainties in our forward-looking
statements, you should not place undue reliance on these statements
or regard these statements as a representation or warranty by us or
any other person that we will achieve our objectives and plans in
any specified time frame, or at all. The forward-looking statements
contained in this press release represent our estimates and
assumptions only as of the date of this press release and, except
as required by law, we undertake no obligation to update or revise
publicly any forward-looking statements, whether as a result of new
information, future events or otherwise after the date of this
press release.
|
Proteon Therapeutics, Inc. |
|
|
Consolidated Balance Sheet
Data |
|
|
(In thousands) |
|
|
|
|
|
|
|
|
|
|
|
December 31, |
|
|
|
|
|
2016 |
|
|
|
2015 |
|
|
|
|
|
|
|
|
|
|
Cash,
cash equivalents and available-for-sale investments |
|
$ |
41,317 |
|
|
$ |
65,263 |
|
|
|
|
|
|
|
|
|
|
Prepaid
expenses and other current assets |
|
|
1,438 |
|
|
|
1,345 |
|
|
|
|
|
|
|
|
|
|
Property
and equipment, net and other non-current assets |
|
|
765 |
|
|
|
930 |
|
|
|
|
|
|
|
|
|
|
Total
assets |
|
$ |
43,520 |
|
|
$ |
67,538 |
|
|
|
|
|
|
|
|
|
|
Accounts payable and
accrued expenses |
|
$ |
5,079 |
|
|
$ |
3,596 |
|
|
|
|
|
|
|
|
|
|
Other liabilities |
|
|
- |
|
|
|
537 |
|
|
|
|
|
|
|
|
|
|
Common
stock and additional paid-in-capital |
|
|
198,218 |
|
|
|
194,667 |
|
|
|
|
|
|
|
|
|
|
Accumulated deficit and accumulated other comprehensive loss |
|
|
(159,777 |
) |
|
|
(131,262 |
) |
|
|
|
|
|
|
|
|
|
Total liabilities and stockholders’ deficit |
|
$ |
43,520 |
|
|
$ |
67,538 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Proteon Therapeutics, Inc. |
|
Condensed Consolidated Statements of
Operations |
|
(in thousands, except share and per share
data) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Year Ended December 31, |
|
|
|
|
2016 |
|
|
|
2015 |
|
|
|
2014 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Revenue |
|
$ |
- |
|
|
$ |
- |
|
|
$ |
2,948 |
|
|
Operating expenses: |
|
|
|
|
|
|
|
Research
and development |
|
|
18,869 |
|
|
|
12,381 |
|
|
|
6,432 |
|
|
General
and administrative |
|
|
9,836 |
|
|
|
8,489 |
|
|
|
4,096 |
|
|
Total
operating expenses |
|
|
28,705 |
|
|
|
20,870 |
|
|
|
10,528 |
|
|
Loss
from operations |
|
|
(28,705 |
) |
|
|
(20,870 |
) |
|
|
(7,580 |
) |
|
Other
income (expense): |
|
|
|
|
|
|
|
Interest
income (expense) |
|
|
193 |
|
|
|
144 |
|
|
|
(833 |
) |
|
Other
(expense) income |
|
|
(14 |
) |
|
|
(651 |
) |
|
|
5,071 |
|
|
Total other (expense) income |
|
|
179 |
|
|
|
(507 |
) |
|
|
4,238 |
|
|
Net
loss |
|
$ |
(28,526 |
) |
|
$ |
(21,377 |
) |
|
$ |
(3,342 |
) |
|
Net loss
per share attributable to common stockholders - basic and
diluted |
|
$ |
(1.72 |
) |
|
$ |
(1.30 |
) |
|
$ |
(3.16 |
) |
|
Weighted-average common shares outstanding - basic and diluted |
|
|
16,561,799 |
|
|
|
16,464,123 |
|
|
|
3,064,507 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Supplemental disclosure of stock-based compensation
expense and loss from currency forward
contracts: |
|
Included in operating expenses, above, are the following
amounts for non-cash stock based compensation expense: |
|
Research
and development |
|
$ |
1,114 |
|
|
$ |
650 |
|
|
$ |
114 |
|
|
General
and administrative |
|
|
2,229 |
|
|
|
1,514 |
|
|
|
345 |
|
|
Total |
|
$ |
3,343 |
|
|
$ |
2,164 |
|
|
$ |
459 |
|
|
|
|
|
|
|
|
|
|
Included in other expense, above, are the following amounts
from forward foreign currency contracts: |
|
|
|
Realized
losses from forward foreign currency contracts |
|
$ |
(61 |
) |
|
$ |
(52 |
) |
|
$ |
- |
|
|
Unrealized losses from forward foreign currency contracts |
|
|
127 |
|
|
|
(537 |
) |
|
|
- |
|
|
Total |
|
$ |
66 |
|
|
$ |
(589 |
) |
|
$ |
- |
|
|
|
|
|
|
|
|
|
Investor ContactGeorge Eldridge, Proteon
Therapeutics, Senior Vice President and Chief Financial
Officer781-890-0102geldridge@proteontherapeutics.com
Media ContactAnn Stanesa, Ten
Bridge
Communications617-230-0347proteon@tenbridgecommunications.com
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