Anacor Pharmaceuticals Announces Positive Preliminary Results from AN2728 MUSE Study in Pediatric and Adolescent Patients with

  Anacor Pharmaceuticals Announces Positive Preliminary Results from AN2728
  MUSE Study in Pediatric and Adolescent Patients with Atopic Dermatitis

     Anacor to Host a Conference Call Today at 5pm ET to Discuss Results

Business Wire

PALO ALTO, Calif. -- November 12, 2013

Anacor Pharmaceuticals (NASDAQ:ANAC) today announced positive results from its
maximal use systemic exposure (MUSE) study of its boron-based
phosphodiesterase-4 (PDE-4) inhibitor, AN2728, in pediatric and adolescent
patients with atopic dermatitis. The results of this study demonstrate that
AN2728 Ointment, 2% appears to be safe, well-tolerated, and efficacious when
applied twice daily to patients, ages two to 18 years with atopic dermatitis
affecting a very large percentage of their body surface area.

“The results of this study are very encouraging. Atopic dermatitis primarily
affects children and has a big impact on the quality of life of patients and
their families. Atopic dermatitis is primarily treated with topical agents,
and physicians are constantly looking for alternatives to steroids. AN2728 has
the potential to become a widely used treatment,” said Adelaide A. Hebert,
M.D., Professor and Director of Pediatric Dermatology at the University of
Texas Medical School at Houston.

“This was an important study as it represents the first time we have treated
patients in the 2 – 11 year age group, an age range that encompasses most
atopic dermatitis patients,” said David Perry, Chief Executive Officer of
Anacor Pharmaceuticals. “We are pleased to see the efficacy and safety profile
demonstrated in this study, as it represents the final significant hurdle
before holding an End of Phase 2 meeting with the FDA.”

AN2728 MUSE Study

The multi-center, open label study enrolled 34 patients, ages two to 18 years
with mild-to-moderate atopic dermatitis with a mean involvement of 48% of
total body surface area (BSA). Mild-to-moderate atopic dermatitis was defined
as an Investigator Static Global Assessment (ISGA) score of 2 (“mild”) or 3
(“moderate”). The ISGA is a 5-point scale from 0 (“clear”) to 4 (“severe”).
Patients (or their caregivers) were instructed to apply AN2728 Ointment, 2%
twice daily for 28 days.

Primary endpoints included an assessment of safety and tolerability based on
the frequency and severity of systemic and local adverse events (AE’s) as well
as the pharmacokinetic profile when AN2728 Ointment, 2% is applied under
maximal use conditions to pediatric and adolescent patients with atopic
dermatitis. Secondary endpoints included descriptive assessments of
improvement in the severity and extent of disease based on the ISGA, signs and
symptoms of atopic dermatitis and treatable percent of body surface area.


  *65% of patients achieved an ISGA score of 0 (“clear”) or 1 (“almost
    clear”) after four weeks of treatment.
  *47% of patients achieved an ISGA score of 0 (“clear”) or 1 (“almost
    clear”) with a minimum 2-grade improvement after four weeks of treatment.
  *Mean pruritus (itching) scores improved by 62% after four weeks of
  *Patients demonstrated an average 78% reduction in affected body surface

Safety and Tolerability

  *AN2728 was generally safe and well-tolerated with the most common AE’s
    being application site reactions.

Pharmacokinetic Profile

  *Overall blood levels in pediatrics and adolescents were low and were
    similar to those previously observed in adults after adjusting for percent
    BSA treated.

AN2728 Development Plan

  *We are currently conducting a Thorough QTc (TQT) study (required by the
    FDA for all new chemical entities) in healthy adults to investigate the
    risk of adverse ECG effects. We expect results from this study before the
    end of 2013.
  *Subject to the results of the TQT study and an End of Phase 2 meeting with
    the FDA in the first quarter of 2014, we plan to initiate a Phase 3 study
    in atopic dermatitis in the first half of 2014.

About Atopic Dermatitis and Current Treatment Options

Atopic dermatitis is a chronic rash characterized by inflammation and itching.
In 2007, Datamonitor reported that atopic dermatitis affected approximately
40million people across the seven major pharmaceutical markets. The condition
most commonly appears in childhood, with up to 20% of children in the United
States affected, and it can persist into adulthood. Skin affected by atopic
dermatitis can often be broken from scratching which can allow bacterial or
viral access and lead to secondary infections. Current atopic dermatitis
treatments attempt to reduce inflammation and itching to maintain the
protective integrity of the skin. Antibiotics, antihistamines, topical
corticosteroids and topical immunomodulators, either as monotherapy or in
combination, are the current standard of care for atopic dermatitis. However,
these can be limited in utility due to insufficient efficacy, side effects or
safety concerns. The most recently approved novel topical treatments for
atopic dermatitis were topical immunomodulators, Protopic (tacrolimus) and
Elidel (pimecrolimus), approved in 2000 and 2001, respectively. Protopic and
Elidel achieved worldwide combined sales of over $500 million and generated
almost 5 million total prescriptions in the U.S. in 2004, prior to receiving
Black Box warnings from the FDA in early 2005.

Conference Call and Webcast

Anacor will host a conference call today at 5:00 p.m. ET / 2:00 p.m. PT to
discuss the results of this study. 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 and will be available for three months following the

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, an antifungal for the treatment of
onychomycosis, and AN2728, an 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, an antibiotic for the treatment of
infections caused by Gram-negative bacteria. Anacor has also discovered three
other compounds that have been out-licensed for further development — one is
licensed to Eli Lilly and Company for the treatment of an animal health
indication, the second compound, AN5568, also referred to as SCYX-7158, is
licensed to Drugs for Neglected Diseases initiative, or DNDi, for human
African trypanosomiasis (HAT, or sleeping sickness) and the third compound is
licensed to GlaxoSmithKline, LLC for development in tuberculosis. Anacor also
has a pipeline of other internally discovered topical and systemic boron-based
compounds in development. For more information, visit

Forward-Looking Statements

This release contains forward-looking statements, including statements
regarding our milestones and clinical plans. Our actual results may differ
materially from those indicated in these forward-looking statements due to
risks and uncertainties, including the timing of data and results from our TQT
study, the timing and outcome of an End of Phase 2 meeting with the FDA, the
timing of the initiation of a Phase 3 study for AN2728; the potential for
success of AN2728, and other matters that are described in Anacor’s Annual
Report on Form 10-K for the year ended December 31, 2012, and subsequent
Quarterly Reports on Form 10-Q filed with the Securities and Exchange
Commission, including the risk factors set forth in those filings. Investors
are cautioned not to place undue reliance on these forward-looking statements,
which speak only as of the date of this release and we undertake no obligation
to update any forward-looking statement in this press release except as
required by law.


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