REDWOOD CITY, Calif.,
Aug. 11, 2014 /PRNewswire/
-- AcelRx Pharmaceuticals, Inc. (Nasdaq: ACRX), (AcelRx), a
specialty pharmaceutical company focused on the development and
commercialization of innovative therapies for the treatment of
acute and breakthrough pain, today reported financial results for
the three and six months ended June
30, 2014. On July 25,
2014, AcelRx announced that the U.S. Food and Drug
Administration (FDA) issued a Complete Response Letter (CRL) for
the Company's new drug application (NDA) for Zalviso™, (sufentanil
sublingual tablet system). Since the receipt of the CRL,
AcelRx has been working on a reply and intends to meet with the FDA
to discuss the items contained in the CRL.
"We were disappointed with the receipt of a Complete Response
Letter for Zalviso and we look forward to meeting with the FDA to
clarify the items included in the CRL and to discuss our planned
response," stated Richard King,
president and CEO of AcelRx. "We have spoken to the FDA and plan to
meet with them by the end of September 2014. We anticipate we
can refile the Zalviso NDA before the end of 2014, pending the
outcome of the meeting with the FDA. We remain confident in
the Zalviso development program and will work closely with the FDA
to address the Agency's concerns as outlined in the CRL to ensure
that healthcare professionals and patient communities will have
access to Zalviso."
The CRL contains requests for additional information on the
Zalviso System to ensure proper use of the device. The
requests include submission of data demonstrating a reduction in
the incidence of optical system errors, changes to the Instructions
for Use for the device to address inadvertent dosing, among other
items, and submission of additional data to support the shelf life
of the product. We believe certain of these requests have
been addressed in amendments to the NDA that were submitted prior
to the receipt of the CRL but, as acknowledged by the FDA, have not
yet been reviewed by the Agency. While we anticipate that
additional bench testing and human factors testing may be required
to address certain items in the CRL, there were no requests to
conduct additional human clinical studies. However, there is no
guarantee that the information previously provided or to be
provided to the FDA will be adequate to address the issues raised
in the CRL.
Second Quarter Financial Results
Net loss for the second quarter of 2014 was $10.6 million, or $0.24 basic net loss per share, and $0.30 diluted net loss per share, compared to
$17.4 million, or $0.47 basic and diluted net loss per share, for
the second quarter of 2013. Basic net loss per share for the
three months ended June 30, 2014
includes $2.5 million in non-cash
income related to the valuation of PIPE warrants which were issued
in connection with a PIPE financing completed in June 2012. This non-cash income was deducted from
net loss in order to arrive at the numerator for the calculation of
diluted EPS and 1.0 million shares were added to the denominator
(using the treasury stock method) to reflect the dilutive effect of
the PIPE warrants. Common shares used in calculating earnings
per share were 43.3 million for basic EPS and 44.3 million for
diluted EPS for the second quarter of 2014, compared to 37.3
million for basic and diluted EPS for the second quarter of
2013.
The decrease in the net loss was primarily due to the valuation
of PIPE warrants, fluctuations for which are charged as other
income or expense, partially offset by an increase in operating
expenses.
During the second quarter of 2014, AcelRx recognized
$71,000 of deferred revenue under the
collaboration agreement with Grunenthal, which AcelRx announced in
December 2013, for the
commercialization of Zalviso in Europe and Australia. During the second quarter of 2013,
AcelRx recognized revenue of $407,000
resulting from reimbursement for work completed under a research
grant from the U.S. Army Medical Research and Materiel Command, or
USAMRMC, for development of ARX-04, a sufentanil tablet system
product candidate for the treatment of moderate-to-severe acute
pain in a range of ambulatory environments. The research grant was
completed in the fourth quarter of 2013.
Research and development expenses for the quarter ended
June 30, 2014 were $7.3 million, compared with $6.1 million for the quarter ended June 30, 2013. The increase was primarily
due to continued development work to support the FDA's review of
the Zalviso NDA.
General and administrative expenses were $5.0 million for the second quarter of 2014,
compared with $2.1 million for the
second quarter of 2013. The increase was primarily due
to market research and other pre-commercial activities in support
of potential marketing approval of Zalviso.
As discussed above, other income and expense in the second
quarter of 2014 includes $2.5 million
in non-cash income caused by a decrease in the value of the PIPE
warrants. During the second quarter of 2013, these PIPE
warrants created $8.7 million in
non-cash expense.
Year-to-Date Financial Results
For the six months ended June 30,
2014, AcelRx reported a net loss of $20.2 million, or $0.47 basic net loss per share and $0.50 diluted net loss per share, compared to
$30.2 million, or $0.81 basic and diluted net loss per share for
the same period in 2013. Basic net loss per share for the six
months ended June 30, 2014 includes
$1.8 million in non-cash income
related to the valuation of PIPE warrants, which was deducted from
net loss in order to arrive at the numerator for the calculation of
diluted EPS and 0.5 million shares were added to the denominator
(using the treasury stock method) to reflect the dilutive effect of
the PIPE warrants. Basic net loss per share for the six
months ended June 30, 2013 includes
$10.4 million in non-cash expense
related to the valuation of PIPE warrants. Common shares used in
calculating earnings per share were 43.3 million for basic EPS and
43.8 million for diluted EPS for the six months ended June 30, 2014, compared to 37.2 million for basic
and diluted EPS for the same period in 2013.
Research and development expenses for the six months ended
June 30, 2014 were $12.0 million, compared to $15.4 million for the six months ended
June 30, 2013. The decrease
over the six months ended June 30,
2014, was primarily due to a high level of activity
associated with Phase 3 clinical studies of Zalviso in the first
half of 2013. General and administrative expenses were
$9.0 million for the six months of
2014, compared with $4.3 million for
the six months ended June 30, 2013.
The increase was primarily due to market research and other
pre-commercial activities in anticipation of marketing approval of
Zalviso.
As of June 30, 2014, AcelRx had
cash, cash equivalents and investments of $92.3 million, compared to $92.9 million at March 31,
2014 and $103.7 million at
December 31, 2013. The decrease
in cash during the year was driven by cash used in operations,
primarily offset by the $10.0 million
draw down of the second tranche of the loan and security agreement
with Hercules, in June 2014.
Financial Outlook
We reiterate and issue financial guidance for the year, as
follows:
- Research and development expenses are expected to be in the
range of $27 to $29 million for the
year and relatively flat quarter over quarter.
- General and administrative expenses, including
pre-commercialization expenses, are expected to be in the range of
$21 to $23 million for the year and
relatively flat quarter over quarter.
- Total operating expenses for 2014 are anticipated to be in the
range of $48 to $52 million.
- Estimated cash, cash equivalents and investment balances at
December 31, 2014 of at least
$65 million.
Corporate Update and Review of Recent Accomplishments
- On July 7, 2014, and pursuant to
the license and supply agreement with Grunenthal Group, AcelRx and
Grunenthal Group announced that Grunenthal submitted a Marketing
Authorization Application (MAA) to the European Medicines Authority
for Zalviso for the management of moderate-to-severe acute pain in
adult patients in a medically supervised environment. Under the
terms of the license agreement, AcelRx will receive a cash payment
of $5 million for the MAA submission
in the third quarter of 2014. AcelRx is eligible to receive an
additional $15 million milestone
payment upon the approval of the MAA. After approval by EMA, AcelRx
is eligible to receive approximately $200
million in additional milestone payments, based upon
successful regulatory and product development efforts ($28.5 million) and net sales target achievements
($171.5 million). Grunenthal will
also make tiered royalty, supply and trademark fee payments in the
mid-teens up to the twenty percent range, on net sales of Zalviso
in the Grunenthal territory (EU, Australia and certain other countries).
- In June 2014, AcelRx completed a
pharmacokinetic study in support of the ARX-04 development program.
In this study of healthy volunteers, it was shown that two
sublingual administrations of a Zalviso 15mcg sufentanil tablet
dosed 20 minutes apart were equivalent to one sublingual
administration of an ARX-04 30mcg sufentanil tablet. The
significance of this study is that we believe we can include
approximately 300 patients from the Zalviso clinical program in the
ARX-04 safety database. The ARX-04 safety database required by the
FDA is 500 patients and, therefore, we believe this demonstration
of dose equivalency will allow for a significant reduction in the
number of patients necessary to enroll in the ARX-04 Phase 3
clinical program. We intend to initiate our Phase 3 clinical study
for ARX-04 before the end of 2014.
Conference Call
AcelRx will conduct a conference call
and webcast today, August 11, at
4:30 p.m. Eastern time (1:30 p.m. Pacific time) to discuss its financial
results and program updates. To listen to the conference
call, dial in approximately ten minutes before the scheduled call
to (800) 860-2442 for domestic callers, (866) 605-3852 for Canadian
callers, or (412) 858-4600 for international callers. Those
interested in listening to the conference call live via the
Internet may do so by visiting the Investors section of the
company's website at www.acelrx.com and selecting the Webcast link
for the Q2 2014 earnings conference call. A webcast replay
will be available on the AcelRx website for 90 days following the
call by visiting the Investors section of the company's website at
www.acelrx.com.
About Zalviso™
Zalviso is an investigational
pre-programmed, non-invasive system to allow hospital patients with
moderate-to-severe acute pain to self-dose with sublingual
sufentanil tablets to manage their pain. Zalviso consists of
sufentanil tablets delivered by the Zalviso System, a needle-free,
handheld, patient-administered, pain management system (together,
"Zalviso"). Zalviso is designed to help address certain
problems associated with post-operative intravenous
patient-controlled analgesia, by
offering:
- A high therapeutic index opioid: Zalviso uses sufentanil, an
opioid that has a high therapeutic index. The therapeutic index is
the ratio of the effective dose versus the lethal or toxic dose. In
animal studies, the therapeutic index for sufentanil was
approximately 100 times larger than fentanyl and 300 times larger
than morphine.
- A non-invasive route of delivery: Zalviso utilizes a sufentanil
tablet which allows for a sublingual (under the tongue) route of
delivery. Sufentanil is highly lipophilic which provides for rapid
absorption in the fatty cells (or mucosal tissue) found under the
tongue and for rapid transit across the blood-brain barrier to bind
the mu-opioid receptors in the brain. The sublingual delivery used
by Zalviso provides rapid onset of analgesia. The sublingual
delivery system also eliminates the risk of IV-related analgesic
gaps and IV complications, such as catheter-related infections. In
addition, because patients do not require direct connection to an
IV PCA infusion pump through IV tubing, Zalviso allows for ease of
patient mobility.
- A pre-programmed PCA solution: Zalviso allows patients to
self-dose sufentanil sublingual tablets via a pre-programmed,
secure system designed to eliminate the risk of programming
errors.
About AcelRx Pharmaceuticals, Inc.
AcelRx
Pharmaceuticals, Inc. is a specialty pharmaceutical company focused
on the development and commercialization of innovative therapies
for the treatment of acute and breakthrough pain. AcelRx's
lead product candidate, Zalviso, is designed to improve the
management of moderate-to-severe acute pain in adult patients in
the hospital setting by utilizing a high therapeutic index opioid,
through a non-invasive delivery route via a pre-programmed,
patient-controlled analgesia device. AcelRx has announced positive
results from each of the three completed Phase 3 clinical trials
for Zalviso, and has submitted an NDA to the FDA seeking approval
for Zalviso in the treatment of moderate-to-severe acute pain in
adult patients in the hospital setting and on July 25th, received a Complete Response Letter
from the FDA. AcelRx plans to initiate a Phase 3 clinical
trial for ARX-04, a product candidate for the treatment of
moderate-to-severe acute pain in a medically supervised setting, by
the end of 2014. The Company has two additional pain treatment
product candidates, ARX-02 and ARX-03, which have completed Phase 2
clinical development. For additional information about
AcelRx's clinical programs, please visit www.acelrx.com.
Forward-Looking Statements
This press release
contains forward-looking statements, including, but not limited to,
statements related to the Company's Zalviso NDA and the Complete
Response Letter (CRL), our plans to address the issues raised in
the CRL, our anticipated resubmission of the Zalviso NDA to the
FDA, including the scope of the resubmission and the timing of the
resubmission and FDA review time, planned initiation of the Phase 3
clinical trial for ARX-04, approval of marketing authorization of
Zalviso in Europe, and the
therapeutic potential of AcelRx's product candidates, including
Zalviso. These forward-looking statements are based on AcelRx's
current expectations and inherently involve significant risks and
uncertainties. AcelRx'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, risks related to:
AcelRx's ability to receive regulatory approval for Zalviso; any
delays or inability to obtain and maintain regulatory approval of
its product candidates, including Zalviso, in the United States and Europe; AcelRx's ability to build an effective
commercial organization; its ability to obtain sufficient financing
to commercialize Zalviso and proceed with clinical development of
ARX-04; the success, cost and timing of all product development
activities and clinical trials, including the planned Phase 3
ARX-04 trial; the market potential for its product candidates; and
other risks detailed in the "Risk Factors" and elsewhere in
AcelRx's U.S. Securities and Exchange Commission filings and
reports, including its Quarterly Report on Form 10-Q filed with the
SEC on May 8, 2014. AcelRx undertakes
no duty or obligation to update any forward-looking statements
contained in this release as a result of new information, future
events or changes in its expectations.
Selected Financial
Data
|
(in thousands, except
per share data)
|
(unaudited)
|
|
|
|
|
|
|
|
|
|
Three Months
Ended
|
|
Six Months
Ended
|
|
June
30,
|
|
June
30,
|
|
2014
|
|
2013
|
|
2014
|
|
2013
|
Statement of
Comprehensive Loss Data
|
|
|
|
|
|
|
|
Revenue:
|
|
|
|
|
|
|
|
Collaboration
agreement
|
$
71
|
|
$
-
|
|
$ 166
|
|
$ -
|
Research
grant
|
-
|
|
407
|
|
-
|
|
1,347
|
Total
revenue
|
71
|
|
407
|
|
166
|
|
1,347
|
|
|
|
|
|
|
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
Research and
development (1)
|
7,284
|
|
6,108
|
|
11,995
|
|
15,426
|
General and
administrative (1)
|
5,047
|
|
2,070
|
|
8,972
|
|
4,261
|
Total operating
expenses
|
12,331
|
|
8,178
|
|
20,967
|
|
19,687
|
Loss from
operations
|
(12,260)
|
|
(7,771)
|
|
(20,801)
|
|
(18,340)
|
|
|
|
|
|
|
|
|
Interest
expense
|
(530)
|
|
(403)
|
|
(1,002)
|
|
(857)
|
Interest income and
other income (expense), net(2)
|
2,215
|
|
(9,273)
|
|
1,597
|
|
(11,012)
|
Net loss
|
$ (10,575)
|
|
$
(17,447)
|
|
$(20,206)
|
|
$(30,209)
|
|
|
|
|
|
|
|
|
Basic net loss per
common share
|
$
(0.24)
|
|
$
(0.47)
|
|
$ (0.47)
|
|
$ (0.81)
|
|
|
|
|
|
|
|
|
Shares used in
computing basic net loss per common share
|
43,333
|
|
37,262
|
|
43,262
|
|
37,198
|
|
|
|
|
|
|
|
|
Diluted net loss per
common share
|
$
(0.30)
|
|
$
(0.47)
|
|
$ (0.50)
|
|
$ (0.81)
|
|
|
|
|
|
|
|
|
Shares used in
computing diluted net loss per common share
|
44,310
|
|
37,262
|
|
43,774
|
|
37,198
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
(1)
Includes the following non-cash, stock-based compensation
expense:
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development
|
$
560
|
|
$
411
|
|
$ 1,039
|
|
$ 766
|
General and administrative
|
334
|
|
391
|
|
830
|
|
793
|
Total
|
$
894
|
|
$
802
|
|
$ 1,869
|
|
$ 1,559
|
|
|
|
|
|
|
|
|
(2) Interest income
and other income (expense) includes $2.5 million and $1.8 million
in non-cash income for the three and six months ended June 30,
2014, respectively, and $8.7 million and $10.4 million in non-cash
charges during the three and six months ended June 30, 2013,
respectively, related to warrants issued in connection with a
private placement equity financing, completed in June
2012.
|
|
|
|
|
|
|
|
|
|
June 30,
2014
|
|
December 31,
2013
|
|
|
|
|
Selected Balance
Sheet Data
|
|
|
|
|
|
|
|
Cash, cash
equivalents and investments
|
$ 92,349
|
|
$
103,663
|
|
|
|
|
Total
assets
|
100,374
|
|
110,031
|
|
|
|
|
Total
liabilities
|
44,825
|
|
36,872
|
|
|
|
|
Total stockholders'
equity
|
55,549
|
|
73,159
|
|
|
|
|
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SOURCE AcelRx Pharmaceuticals, Inc.