Anacor Pharmaceuticals Announces Positive Results From the First of Two Phase 3 Trials of Tavaborole for Onychomycosis

  Anacor Pharmaceuticals Announces Positive Results From the First of Two
  Phase 3 Trials of Tavaborole for Onychomycosis

    Anacor Will Host a Conference Call Today at 8am ET to Discuss Results

Business Wire

PALO ALTO, Calif. -- January 29, 2013

Anacor Pharmaceuticals (NASDAQ:ANAC) today announced positive preliminary
results from the first of two Phase 3 trials of tavaborole, its topical
anti-fungal for onychomycosis, a fungal infection of the nail and nail bed
that affects approximately 35 million people in the United States. Tavaborole
achieved a high degree of statistical significance on all primary and
secondary endpoints.

Primary Endpoint

  *In this first Phase 3 study (known as Study 301), 6.5% of patients treated
    with tavaborole met the primary endpoint of “complete cure” vs. 0.5% of
    patients treated with vehicle (p=0.001) at week 52. “Complete cure” is a
    composite endpoint that requires both a mycological cure and a completely
    clear nail.

Secondary Endpoints

  *Among the secondary endpoints, 26.1% of patients treated with tavaborole
    achieved a “completely clear” or “almost clear” (≤10% clinical
    involvement) nail vs. 9.3% in the vehicle-treated arm (p<0.001) at week
    52.
  *31.1% of patients treated with tavaborole achieved mycological cure vs.
    7.2% in the vehicle-treated arm (p<0.001) at week 52. Mycological cure is
    the absence of fungus as determined by a negative potassium hydroxide
    (“KOH”) examination and a negative fungal culture.
  *15.3% of patients treated with tavaborole achieved “completely clear” or
    “almost clear” nail with mycological cure vs. 1.5% in the vehicle-treated
    arm (p<0.001) at week 52.

In addition to the primary and secondary endpoints noted above, 87.0% of
patients treated with tavaborole had a negative fungal culture vs. 47.9% in
the vehicle-treated arm (p<0.001) at week 52, and 24.6% of patients treated
with tavaborole achieved “completely clear” or “almost clear” nail and
negative culture vs. 5.7% in the vehicle-treated arm (p<0.001) at week 52.

“We are pleased that tavaborole met all of the parameters outlined in our
Special Protocol Assessment with the FDA with a high degree of statistical
significance in this first Phase 3 trial. With its high rates of ‘completely
clear’ or ‘almost clear’ nails, high negative culture rates, demonstrated
safety and ease of use, we believe tavaborole could offer significant
advantages over currently approved treatments for onychomycosis,” said David
Perry, Chief Executive Officer of Anacor.

“Patients and physicians have been waiting for a safe and effective treatment
for onychomycosis. Millions of patients are treated several times a year with
debridement because it is the safest option to improve the appearance of the
nail and minimize discomfort, even though debridement doesn’t actually
eliminate the fungus. Tavaborole has the potential to offer these patients an
effective, safe and convenient therapy to treat this difficult infection and
prevent it from spreading to other toes and skin,” said Max Weisfeld, DPM.

“Tavaborole belongs to a novel class of drugs, known as oxaboroles, and should
be an exciting addition to dermatologists’ treatment options for patients who
suffer from onychomycosis,” said Boni Elewski, MD, Professor of Dermatology,
University of Alabama. “It is a challenging and frustrating disease, but
tavaborole could be a safe and effective alternative for patients who are
embarrassed by the appearance of their yellow, thickened nails and are either
unwilling or unable to take oral medications to treat this condition.”

Tavaborole Phase 3 Study 301 Design

Study 301 enrolled 594 patients in the United States and Mexico with distal
subungual onychomycosis, randomized two-to-one to receive either tavaborole,
5% solution, or vehicle. Eligible patients were at least 18 years of age (with
no upper age limit) with a clinical diagnosis of distal subungual
onychomycosis involving 20% - 60% of the total area of the target great
toenail, at least 3mm of clear nail from the proximal nail fold to the most
proximal visible mycotic border and positive mycology. Patients were
instructed to apply tavaborole solution or vehicle to the target great toenail
once daily for 48 weeks.

Safety

Overall, tavaborole was safe and well-tolerated across study subjects. There
were no serious adverse events related to study drug. The rate of
discontinuations as a result of adverse events was low (2.8% for tavaborole
and 1.6% for vehicle).

Development Plan

Study 301 is the first of two Phase 3 clinical trials of tavaborole in
onychomycosis. Anacor received a Special Protocol Assessment from the FDA on
all major parameters of the studies, including endpoints. Data from the second
Phase 3 clinical trial (known as Study 302) are expected in March of this
year. Study 302 is identical in design to Study 301 with sites in the United
States and Canada. Subject to the results of Study 302, Anacor plans to file
an NDA for tavaborole in the middle of this year.

Conference Call and Webcast

Anacor will host a conference call today at 8:00 a.m. ET / 5:00 a.m. PT to
discuss the results of this Phase 3 trial of tavaborole in onychomycosis. The
call can be accessed by dialing (877) 291-1367 (domestic) and (914) 495-8534
(international) five minutes prior to the start of the call. The call will
also be webcast live and can be accessed on the Events and Presentations page,
under Investors, on the company’s website at http://www.anacor.com and will be
available for three months following the call.

About Anacor Pharmaceuticals

Anacor is a biopharmaceutical company focused on discovering, developing and
commercializing novel small-molecule therapeutics derived from its boron
chemistry platform. Anacor has discovered eight compounds that are currently
in development. Its two lead product candidates are topically administered
dermatologic compounds — tavaborole, a topical antifungal for the treatment of
onychomycosis, and AN2728, a topical anti-inflammatory PDE-4 inhibitor for the
treatment of atopic dermatitis and psoriasis. In addition to its two lead
programs, Anacor has discovered three other wholly-owned clinical product
candidates — AN2718 and AN2898, which are backup compounds to tavaborole and
AN2728, respectively, and AN3365 (formerly known as GSK2251052, or GSK‘052),
a systemic antibiotic for the treatment of infections caused by Gram-negative
bacteria, which previously was licensed to GlaxoSmithKlineLLC, or GSK. GSK
will be returning all rights to the compound to us and we are considering our
options for further development, if any, of this compound. We have discovered
three other compounds that we have out-licensed for further development — two
compounds for the treatment of animal health indications that are licensed to
Eli Lilly and Company and AN5568, also referred to as SCYX-7158, for human
African trypanosomiasis (HAT, or sleeping sickness), which is licensed to
Drugs for Neglected Diseases initiative, or DNDi. We also have a pipeline of
other internally discovered topical and systemic boron-based compounds in
development. For more information, visit http://www.anacor.com.

Forward-Looking Statements

This press release may contain forward-looking statements that relate to
future events including the development of tavaborole, timing for filing of a
new drug application, or NDA, related to tavaborole and the potential
commercialization of tavaborole. These forward looking statements involve
known and unknown risks, uncertainties and other factors that could cause
actual levels of activity, performance or achievement to differ materially
from those expressed or implied by these forward-looking statements, including
risks related to whether the results of the ongoing Phase 3 302 study will be
successful and sufficient to support the planned NDA filing, risk of
unforeseen side effects and risks related to regulatory approval and
commercialization of new drug candidates. Reference should be made to Anacor’s
Annual Report on Form 10-K for the year ended December 31, 2011, filed with
the Securities and Exchange Commission under the heading “Risk Factors” and
Anacor’s subsequent Quarterly Reports on Form 10-Q for a more detailed
description of such factors. These statements reflect the views of Anacor as
of the date of this press release with respect to future events and, except as
required by law, it undertakes 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.

Contact:

Anacor Pharmaceuticals
DeDe Sheel, 650-543-7575
Director, Investor Relations and Corporate Communications
 
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