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We are a biotechnology company focused on the discovery, development
and commercialization of novel phage therapeutics. Phage therapeutics use bacteriophages, a family of viruses, to kill pathogenic
bacteria. Phages have powerful and highly selective mechanisms of action that permit them to target and kill specific bacteria.
We believe that phages represent a promising means to treat bacterial infections, especially those that have developed resistance
to current therapies, including the so-called multi-drug-resistant or "superbug" strains of bacteria.
Our goal is to be the leading developer of phage therapeutics.
We are combining our expertise in the manufacture of drug-quality bacteriophages and our proprietary approach and expertise in
identifying, characterizing and developing naturally occurring bacteriophages with that of our collaboration partners in bacteriophage
biology, synthetic biology and manufacturing, to develop second-generation bacteriophage products.
The extensive use of antibiotics since their discovery in the
1940s has resulted in drug resistance among many disease-causing bacteria. According to the U.S. Centers for Disease Control and
Prevention, or CDC, resistance to antibiotics threatens to reverse many of the key medical advances of the last half-century. Examples
of clinically important microbes that are rapidly developing resistance to available antimicrobials include bacteria that cause
skin, bone, lung and bloodstream infections (e.g., S. aureus and methicillin-resistant S. aureus, or MRSA), pneumonia
and lung infections in both community and hospital settings and cystic fibrosis patients (e.g., A. baumanii, P. aeruginosa,
and K. pneumoniae), meningitis (e.g., S. pneumonia), urinary tract and gastrointestinal infections (e.g., E. coli
and C. difficile). As phages kill bacteria in ways entirely unlike the mechanisms used by traditional antibiotics, we believe
that multi-drug resistant bacteria will be susceptible to phage therapy. Furthermore, should resistant bacteria emerge or evolve,
we believe it will remain possible to identify phages that can effectively kill these resistant bacteria.
Our lead product candidate is AB-SA01, for the treatment of
S. aureus infections, including MRSA. We also have another product candidate in earlier stage development, AB-PA01 for the
treatment of P. aeruginosa infections, and an additional discovery program, AB-CD01 for the treatment of C. difficile
We are developing our phage product candidates using a proprietary
discovery and development platform, which is designed for rapid identification, characterization and manufacturing of multiple
phage therapeutics. Each product candidate combines several carefully chosen phages, which target a specific disease-causing bacteria
such as S. aureus, P. aeruginosa, and C. difficile. We believe that the combination of our platform, our manufacturing
capability, our understanding of the regulatory and development requirements of bacteriophage therapeutics, and the clinical and
scientific expertise of our collaboration partners may enable the rapid advancement of phage therapeutics through the clinic and
the regulatory approval process.
In June 2013, we entered into a cooperative research and development
agreement, or Research and Development Agreement, with the United States Army Medical Research and Materiel Command focusing on
developing bacteriophage therapeutics to treat S. aureus, E. coli and P. aeruginosa infections. Under this
Research and Development Agreement, we completed enrollment of a Phase 1 safety study of AB-SA01 for the treatment of wounds infected
with S. aureus in July 2016. We expect to report top-line results by the end of the third quarter of 2016, with the complete
study report expected by the end of 2016.
In September 2013, we entered into a license agreement, or the
Leicester License Agreement, with the University of Leicester to develop a phage therapy to kill certain types of C. difficile.
Pursuant to the Leicester License Agreement, we may be obligated to pay the University of Leicester a single digit royalty and
an aggregate of up to 575,000 in milestone payments.
In November 2015, our Australian subsidiary, AmpliPhi Australia
Pty Ltd, entered into a clinical trial research agreement with the University of Adelaide and the Queen Elizabeth Hospital, both
of Adelaide, SA, Australia, to conduct a Phase 1 clinical trial titled "A Phase 1 Investigator Initiated Study to Evaluate
the Safety, Tolerability and Preliminary Effectiveness of AB-SA01 in Patients with Chronic Rhinosinusitis Associated with S.
aureus infection". The University of Adelaide will sponsor the clinical trial while we will supply AB-SA01 and control
the trial protocol. This clinical trial will primarily measure the safety and tolerability of AB-SA01 and will secondarily examine
the presence of S. aureus and symptoms assessed by the patient as well as by the physician using standard questionnaires
used by physicians to assess treatment efficacy. We plan to enroll nine patients, divided into three cohorts. The first cohort
received a twice daily dose of AB-SA01 for seven days. The second cohort received the same dose twice daily for 14 days. The third
cohort will receive a higher dose of AB-SA01 twice daily for 14 days. Patients will be monitored an additional 30 days following
their last day of treatment. Patient screening for this clinical trial commenced in late 2015 and the first patient was dosed in
January 2016. The first and second cohorts have been completed and the first subject in the third cohort has completed dosing.
Two subjects remain to be dosed in the final cohort and we expect to report data from this first clinical trial in the second half
of 2016. We are planning a Phase 2 trial in chronic rhinosinusitis patients, to commence in the second half of 2017.
In January 2016, we entered into an Asset Purchase Agreement
with Novolytics Ltd., which we refer to as the Novolytics Purchase Agreement, to purchase certain tangible and intangible assets.
Pursuant to the Novolytics Purchase Agreement, we acquired all rights, title and interest to two families of patents. The first
patent family is titled "Anti-bacterial compositions" and has been granted in Australia and China with prosecution
pending in the United States and other countries. The second patent family is titled "Novel bacteriophages" and the
prosecution is pending in the United States and other countries. We also received clinical isolates for S. aureus which
will bolster our libraries of clinically relevant strains. Additionally, we received know-how relating to certain formulation processes.
We also have access to all previous dialogue between Novolytics and various regulatory organizations including the United Kingdom
Medicines and Healthcare Products Regulatory Agency, or MHRA.
The Need for New Anti-Infective Therapies
The rapid and continuous emergence of antibiotic-resistant bacteria
has become a global crisis. Despite this crisis, the number of novel anti-infective therapies currently in development is at historically-low
levels. The CDC estimates that more than two million people in the United States acquire an antibiotic-resistant infection each
year and more than 23,000 of these prove fatal. It is estimated that 50% of hospital-acquired infections are resistant to first-line
anti-infective therapies. The cumulative annual cost for treating resistant bacterial infections in the United States alone is
estimated to be $20 billion, while the global antibiotics market opportunity was estimated to be $40.3 billion in 2015.
The CDC's latest report on the matter, Antibiotic Resistance
Threats in the United States, 2013, notes that there are "potentially catastrophic consequences of inaction" and
ranks C. difficile as belonging to the highest tier of threat, or "Urgent Threats." Despite the potential market
opportunity, only two New Drug Applications, or NDAs, for antibacterial drugs were approved by the FDA between 2010 and 2012 compared
to 18 in the period between 1980 and 1984. One of the primary recommendations of the CDC is the development of new antimicrobials
to diversify treatment options.
AB-SA01: Infections Caused by S. aureus
By screening our proprietary library of phage samples against
a panel of S. aureus bacteria, collected from around the world, we have selected a phage product candidate mix that has
demonstrated, in in vitro studies, greater than 92% efficacy with high overlap against a global diversity panel that includes
some of the most virulent isolates of S. aureus, including MRSA isolates. The three phage constituents of AB-SA01 were selected
for their ability to target the greatest number of bacterial isolates in the collection and maximal complementation. Complementation,
defined as the percentage of S. aureus isolates susceptible to more than one phage, is emphasized in product selection to
reduce risk of the emergence of bacterial resistance.
In connection with our Research and Development Agreement with
the U.S. Army Medical Research and Materiel Command, we are developing AB-SA01 to treat acute and chronic infections caused by
S. aureus, including infections caused by MRSA strains of the same bacterium. MRSA infections are one of the most common
causes of hospital-acquired (nosocomial) infections. The CDC estimates that more than 850,000 patients were treated for S. aureus
infections of the skin or soft tissue in 2013 and, due to failure of first line treatment, more than 50% of these patients required
a second-line treatment and approximately 35% of them required a third-line treatment. Global Data estimates the market for MRSA
infection treatments alone was more than $2.7 billion in 2007. This market is forecasted to grow to more than $3.5 billion by 2019.
Also in connection with our Research and Development Agreement
with the U.S. Army, we submitted a pre-IND briefing package to the FDA to obtain their feedback on our Chemistry, Manufacturing
and Controls, or CMC, program and plans for our first human clinical trial of AB-SA01 for the treatment of S. aureus infections
of wound and skin. The FDA concurred with our plan for progressing this bacteriophage product candidate into clinical trials, specifically
agreeing with the proposed manufacturing process and product specifications and not requiring non-clinical toxicology data to initiate
our first Phase 1 clinical trial. We initiated the Phase 1 clinical trial in May 2016 and completed enrollment in July 2016. We
expect the complete study report to be available before the end of 2016.
In December 2015, we opened a clinical trial at the University
of Adelaide Queen Elizabeth Hospital to evaluate the safety and preliminary efficacy of AB-SA01 in chronic rhinosinusitis patients
infected with S. aureus. The first patient in this clinical trial was dosed in January 2016, and we have continued to dose