AcelRx Announces Primary Endpoint Met in Phase 3 Non-Inferiority Study of Sublingual Sufentanil NanoTab® PCA System vs. IV PCA

  AcelRx Announces Primary Endpoint Met in Phase 3 Non-Inferiority Study of
      Sublingual Sufentanil NanoTab® PCA System vs. IV PCA Morphine for
                             Post-Operative Pain

-In this open-label study, the Sufentanil NanoTab PCA System also demonstrated
statistical superiority to IV PCA morphine for primary endpoint of Patient
Global Assessment of method of pain control

- Nurses and patients rated Sufentanil NanoTab PCA System higher for Overall
Satisfaction and Ease of Care compared to IV PCA morphine

- AcelRx to conduct conference call and webcast today, November 15, at 8:00
a.m. EST (5:00 a.m. PST) to discuss top-line results in detail

PR Newswire

REDWOOD CITY, Calif., Nov. 15, 2012

REDWOOD CITY, Calif., Nov. 15, 2012 /PRNewswire/ -- AcelRx Pharmaceuticals,
Inc. (Nasdaq: ACRX), a specialty pharmaceutical company focused on the
development and commercialization of innovative therapies for the treatment of
acute and breakthrough pain, today announced top-line data showing that the
open-label Phase 3 study of its investigational sublingual (under the tongue)
Sufentanil NanoTab PCA (patient-controlled analgesia) System met its primary
endpoint of non-inferiority in patient global assessment (PGA) with method of
pain control in comparison to intravenous (IV) PCA with morphine. Additional
analyses also showed that in this study the NanoTab System was statistically
significantly superior to IV morphine for the PGA measurement. In addition,
using validated assessment tools, nurses managing patients in the study and
the patients themselves reported that they had significantly greater Overall
Satisfaction with the NanoTab System compared to IV PCA morphine and
significantly greater Overall Ease of Care with the NanoTab System compared to
IV PCA morphine.

"With these impressive top-line results from this head-to-head clinical trial,
we have successfully completed an important step towards our New Drug
Application (NDA) submission and, dependent on completing the remaining Phase
3 trials and obtaining FDA approval, eventual commercialization of the
Sufentanil NanoTab PCA System," commented Richard King, President and CEO, of
AcelRx Pharmaceuticals, Inc. "Delivering patient-controlled analgesia in a
non-invasive, pre-programmed system that provides powerful pain control while
enhancing patient ease of care and satisfaction and nurse satisfaction
compared to current invasive delivery systems will be a major advance for
hospital care."

Utilizing a randomized, open-label, parallel-group design, this Phase 3 study
enrolled 359 adult patients at 26 U.S. sites and compared efficacy and safety
of AcelRx's investigational ARX-01 sublingual Sufentanil NanoTab PCA System
(15 mcg/dose) to the commonly used IV PCA with morphine (1 mg/dose) for the
treatment of acute post-operative pain immediately following major abdominal
or orthopedic surgery. Patients were randomized 1:1 to treatment with the
NanoTab System or IV PCA morphine and were treated for post-operative pain for
a minimum of 48 hours and up to 72 hours.

Top-line results of the Phase 3 clinical trial demonstrate that the Sufentanil
NanoTab PCA System was non-inferior (p<0.001) to IV PCA morphine for the
primary endpoint of PGA over the 48-hour study period as determined by the
combined percentage of patients with PGA ratings of "good" or "excellent"
(78.5% vs. 66.1% respectively). The assessment of non-inferiority is based on
a lower limit of -15% for the 95% confidence interval (CI) around the
difference between these percentages. Because the 95% CI was +3.2% to +21.6%
for the 48 hour PGA and therefore didn't cross the zero difference line, a
statistical analysis for superiority could be performed, which demonstrated
that for this study, the NanoTab System was statistically superior to IV PCA
morphine for the PGA endpoint (p=0.009). This statistically superior PGA was
also seen at the 24 hour and 72 hour timepoints. Additionally, the percentage
of patients rating the NanoTab System as "Excellent" was higher than those
rating IV PCA morphine as excellent (42.9% vs. 30.6%, p=0.016). Similar
percentages of NanoTab System-treated and IV PCA morphine-treated patients
dropped out of the study prematurely due to lack of efficacy (7.3% vs. 8.3%
respectively) or due to an adverse event (7.9% vs. 11.1% respectively).

Nurses setting up the different treatments for use and managing patients in
the study reported that they had greater Overall Satisfaction (3.93 vs. 3.32
out of 5, p<0.001) and Overall Ease of Care (4.26 vs. 3.82, p=0.018) with the
Sufentanil NanoTab PCA System compared to IV PCA morphine. Likewise, patients
in the study reported that they had greater Overall Satisfaction (4.15 vs.
3.83 out of 5, p=0.003) and greater Overall Ease of Care (4.45 vs. 4.07,
p<0.001) with the NanoTab System compared to IV PCA morphine.

"Our goal at AcelRx has always been to improve upon the management of
post-operative pain using a simplified system that nurses don't have to
program," commented Pamela Palmer MD, PhD, Chief Medical Officer and AcelRx
co-founder. "The validated Patient Overall Ease of Care assessment is a
multi-dimensional evaluation that includes confidence, comfort and ease of
movement with the delivery device, along with dosing confidence, pain control
and knowledge and understanding of product use. The higher score in this
composite measure, which reflects higher scores in each of the subscales,
along with the higher nursing Overall Satisfaction ratings, and combined with
the excellent pain control demonstrated in the PGA rating validates our
efforts for developing this product for hospitalized patients" added Dr.
Palmer.

"It was impressive to observe the ease of set-up and use of the Sufentanil
NanoTab PCA System by both nurses and patients compared to our typical IV PCA
system," stated study investigator Dr. Harold Minkowitz in the Department of
Anesthesiology at Memorial-Hermann Hospital in Houston, Texas. "Observing the
rapid onset and significant level of pain relief obtained with this
non-invasive route of opioid administration over a broad range of patients and
types of surgery was remarkable."

In addition to this well-controlled supportive trial, two other Phase 3
clinical studies of the Sufentanil NanoTab PCA System are underway. In March
2012, AcelRx initiated a randomized, double-blind, placebo-controlled efficacy
and safety study comparing the NanoTab System to placebo for post-operative
pain control following major open abdominal surgery. In August 2012, AcelRx
initiated a randomized, double-blind, placebo-controlled efficacy and safety
study comparing the NanoTab System to placebo in treating post-operative pain
following major joint replacement surgery. Data from both of these pivotal
studies are expected in the first quarter of 2013. Additional information
about the Phase 3 clinical trials with the NanoTab System can be found by
visiting www.clinicaltrials.govand using the identifiers, NCT01660763,
NCT01539642 and NCT01539538.

About Post-Operative Pain
Acute pain management in the hospital, in particular post-operative analgesia,
remains a challenge for healthcare providers with up to 75% of patients
reporting inadequate pain relief following surgery. Inadequate treatment of
post-surgical pain can lead to decreased mobility, which increases the risks
for serious medical complications, including deep vein thrombosis and partial
lung collapse, potentially resulting in extended hospital stays. More than 30
million surgical procedures per year result in moderate to severe pain in the
US and EU, with an additional 27 million procedures in countries with moderate
to high per capita healthcare expenditures. The US, 5 main EU countries and
Japan represented $5.1 billion of acute pain treatment product sales in 2008.
Currently patients experiencing post-operative pain in the hospital may have
IV PCA treatment, typically utilizing morphine or hydromorphone. However,
there are deficiencies associated with the current use of IV PCA that can
negatively impact patient safety, well-being and recovery. These include
drug-related side effects associated with morphine or hydromorphone,
complications associated with IV delivery, and medication delivery errors
typically associated with misprogramming of the complex IV PCA pumps.

About ARX-01, the Sufentanil NanoTab PCA System

ARX-01 is an investigational pre-programmed, non-invasive, handheld system
that allows post-operative patients to self-dose with sublingual Sufentanil
NanoTabs to manage their post-operative pain. The ARX-01 System is designed
to address the limitations of IV PCA by offering:

  oA high therapeutic index opioid: ARX-01 uses the high therapeutic index
    opioid sufentanil; it offers post-operative pain patients the potential
    for effective patient-controlled analgesia with a low incidence of
    drug-related side effects.
  oA non-invasive route of delivery: The sublingual route of delivery used in
    ARX-01 provides rapid onset of analgesia, therefore eliminating the risk
    of IV-related analgesic gaps and IV complications, such as
    catheter-related infections. In addition, because patients are not
    tethered to IV tubing and a pump for pain relief, ARX-01 allows for ease
    of patient mobility.
  oA simple, pre-programmed PCA solution: ARX-01 is a pre-programmed PCA
    System designed to eliminate the risk of pump programming errors.

Conference Call

The conference call and webcast will be held today, Thursday, November 15,
2012 at 8:00 a.m. Eastern Time (5:00 a.m. Pacific Time) to discuss the Phase 3
top-line results. 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 Investor Relations section of the company's
website at www.acelrx.com.

A webcast replay will be available on the AcelRx website for 90 days following
the call by visiting the Investor Relations section of the company's website
at www.acelrx.com

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,
the ARX-01 Sufentanil NanoTab PCA System, which is currently in Phase 3
clinical development, is designed to solve the problems associated with
post-operative intravenous patient-controlled analgesia which has been shown
to cause harm to patients following surgery because of the side effects of
morphine, the invasive IV route of delivery and the inherent potential for
programming and delivery errors associated with the complexity of infusion
pumps. AcelRx has two additional product candidates which have completed
Phase 2 clinical development: ARX-02 for the treatment of cancer breakthrough
pain, and ARX-03 for mild sedation, anxiety reduction and pain relief for
patients undergoing painful procedures in a physician's office. AcelRx has
initiated a Phase 2 study for a fourth product candidate, ARX-04, a sufentanil
formulation for the treatment of moderate-to-severe acute pain, funded through
a grant from the U.S. Army Medical Research and Materiel Command, or USAMRMC.
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 process and timing of anticipated future
clinical development of AcelRx Pharmaceuticals' product candidates, including
the release ARX-01 top-line clinical trial data, the release and anticipated
timing of additional ARX-01 clinical trial data, the potential filing of an
NDA for the ARX-01 and the timing thereof, therapeutic and commercial
potential of ARX-01 and the anticipated timing and therapeutic and commercial
potential of other AcelRx Pharmaceuticals' product candidates. These
forward-looking statements are based on AcelRx Pharmaceuticals' current
expectations and inherently involve significant risks and uncertainties.
AcelRx Pharmaceuticals' 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: the ability of AcelRx Pharmaceuticals to successfully
complete the clinical trials for ARX-01, that fact that subsequent analyses of
the full data set may lead to different (including less favorable)
interpretations of the results than the analyses conducted to date or may
identify important implications of the study that are not reflected in these
statements, or be subject to differing interpretations by the regulatory
agencies; the success, cost and timing of all product development activities
and clinical trials; the uncertain clinical development process, including the
risk that clinical trials, have an effective design, enroll a sufficient
number of patients, or be completed on schedule, if at all; any delays or
inability to obtain regulatory approval of its product candidates in the
United States and Europe; its ability to obtain adequate clinical supplies of
the drug and device components of its product candidates; its ability to
attract funding partners or collaborators with development, regulatory and
commercialization expertise; its ability to obtain sufficient financing to
complete development and registration of its product candidates in the United
States and Europe; its ability to obtain and maintain regulatory approvals of
its product candidates in the United States and Europe; the market potential
for its product candidates; the accuracy of AcelRx Pharmaceuticals' estimates
regarding expenses, capital requirements and needs for financing; and other
risks detailed in the "Risk Factors" and elsewhere in AcelRx Pharmaceuticals'
U.S. Securities and Exchange Commission filings and reports, including its
Quarterly Report on Form 10-Q for the three months ended September 30, 2012.
AcelRx Pharmaceuticals 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.

SOURCE AcelRx Pharmaceuticals, Inc.

Website: http://www.acelrx.com
Contact: Jim Welch, Chief Financial Officer, +1-650-216-3511.
jwelch@acelrx.com; Lee A. Davies, Makovsky, +1-212-508-9651,
ldavies@makovsky.com
 
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