Destiny Pharma announces appointment of Chief Medical Officer

Destiny Pharma plc
(“Destiny” or “the Company”)

Destiny Pharma announces appointment of Chief Medical Officer

Dr Jesús González brings extensive experience in the clinical development of anti-infectives, as the Company prepares to initiate a Phase 2b trial for lead asset

Brighton, United Kingdom – 26 September 2018 – Destiny Pharma (AIM: DEST), a clinical stage biotechnology company focused on the development of novel antimicrobial drugs, which address the global problem of antimicrobial resistance (AMR), today announces the appointment of Jesús M González Moreno, M.D. as Chief Medical Officer (CMO) of the Company, with immediate effect.

Dr González is an infectious disease expert with more than 11 years’ experience of working within global pharmaceutical and biotechnology companies to design, coordinate and execute clinical development plans for anti-infective drug candidates. His experience spans from early and late clinical development to medical affairs and preparation for marketing authorisation submissions. Dr González most recently served as Global Clinical Development Director at TiGenix SAU in Spain, where he was responsible for the overall development strategy for Cx611, a stem cell treatment for sepsis secondary to community-acquired pneumonia, including the design and implementation of its Phase 1b/2a clinical trial.

Prior to that Dr González worked at Basilea Pharmaceutcia in Switzerland as Medical Science Liaison team leader, responsible for the global medical plan for anti-infectives. Dr González also held a number of medical director roles at AstraZeneca within their Infection division, where he contributed to the development strategy, was responsible for the coordination and implementation of Phase 2-3 trials for novel antibiotics and collaborated in regulatory fillings globally. Additionally, Dr González worked as Regional Director of Medical Affairs in Antibiotics & Antifungals for Merck&Co and MSD, responsible for the development and implementation of regional medical and scientific strategy for these anti-infectives in Europe, Middle East, Africa, and Canada.

Dr González is a licensed physician with a medical doctor degree from the Universidad Autónoma de Madrid, Spain. Dr González also received a master’s degree in Statistics and Study Design for Research in the Health Sciences from the Universidad Autónoma de Barcelona. Dr González completed his postdoctoral fellowship in emerging and re-emerging infectious diseases at the Uniformed Services University of the Health Sciences, Department of Defence, Maryland, US.

Neil Clark, Chief Executive Officer of Destiny Pharma, commented:
I am delighted to welcome Dr González to the senior management team as our new Chief Medical Officer. Jesús brings a wealth of knowledge and experience in the clinical development of anti-infective drugs, from early stage development through to regulatory submission. Dr González’ proven track record will be invaluable to Destiny Pharma as we continue to progress the development of our novel XF drugs to address antimicrobial resistance and I look forward to working closely with him.”

For further information, please contact:

Destiny Pharma plc
Neil Clark, CEO
Simon Sacerdoti, CFO
pressoffice@destinypharma.com
+44 (0)1273 704 440

FTI Consulting
Simon Conway / Victoria Foster Mitchell
destinypharma@fticonsulting.com
+44 (0) 20 3727 1000

Cantor Fitzgerald Europe (Nominated Adviser and Joint Broker)
Philip Davies / Will Goode, Corporate Finance
Andrew Keith, Healthcare Equity Sales
+44 (0)20 7894 7000

finnCap Ltd (Joint Broker)
Geoff Nash /Kate Bannatyne, Corporate Finance
Alice Lane, Corporate Broking
+44 (0)20 7220 0500

About Destiny Pharma
Destiny Pharma is an established, clinical stage, innovative biotechnology company focused on the development of novel medicines that represent a new approach to the treatment of infectious disease. These potential new medicines are being developed to address the need for new drugs for the prevention and treatment of life-threatening infections caused by antibiotic-resistant bacteria, often referred to as “superbugs”. Tackling anti-microbial resistance has become a global imperative recognised by the WHO and the United Nations, as well as the G7 and the G20 countries. For further information, please visit https://www.destinypharma.com

Directorate Change

Destiny Pharma plc
(“Destiny Pharma” or “the Company”)

Directorate Change

New Chairman Appointment

Brighton, United Kingdom – 6 September 2018 – Destiny Pharma (AIM: DEST), a clinical stage biotechnology company focused on the development of novel antimicrobial drugs, which address the global problem of antimicrobial resistance (AMR), announces that Sir Nigel Rudd is stepping down as Chairman and a member of the Board after serving eight years and having made his first investment in the Company in 2003.

Nick Rodgers, Non-Executive Director of Destiny Pharma, will become Chairman following Sir Nigel’s departure on 31 December 2018. Nick has considerable Board experience in both public and private growth companies, particularly those in the life science sector, as well as a background as a successful corporate financier and investment banker. Nick is currently Chairman of SEHTA, one of the largest health technology networking organisations in the UK. Prior to this, he was Non-Executive Director and then Chairman of Oxford Biomedica plc, a leading gene-based biopharmaceutical company.

Sir Nigel Rudd, Chairman of Destiny Pharma, commented:
“It has been a privilege to be part of Destiny Pharma for the past 15 years. During this time, the Company has experienced transformative growth underpinned by the discovery of the XF drug platform which has the potential to address the global issue of antimicrobial resistance. I am confident in the outlook of the business, with a strong management team in place to deliver against the Company’s strategic goals.”

Neil Clark, Chief Executive Officer of Destiny Pharma, added:
“On behalf of Destiny Pharma, I would like to thank Sir Nigel for his considerable contribution to the Company which has grown significantly as a result of his extensive experience, specialised industry knowledge and track record of developing early stage companies. We wish Sir Nigel all the very best for the future. On behalf of Destiny Pharma, I also welcome Nick as Chairman and look forward to continuing to work closely with him as we advance our XF-drug platform through clinical development.”

For further information, please contact:

Destiny Pharma plc
Neil Clark, CEO
Simon Sacerdoti, CFO
pressoffice@destinypharma.com
+44 (0)1273 704 440

FTI Consulting
Simon Conway / Victoria Foster Mitchell
destinypharma@fticonsulting.com
+44 (0) 20 3727 1000

Cantor Fitzgerald Europe (Nominated Adviser and Joint Broker)
Philip Davies / Will Goode, Corporate Finance
Andrew Keith, Healthcare Equity Sales
+44 (0)20 7894 7000

finnCap Ltd (Joint Broker)
Geoff Nash /Kate Bannatyne, Corporate Finance
Alice Lane, Corporate Broking
+44 (0)20 7220 0500

About XF-73
XF-73 is a synthetic anti-microbial active against all tested Staphylococcus aureus strains, including drug resistant strains. By acting via a cell-surface mechanism it affects the bacterial membrane permeability and integrity, leading to cell death. XF-73 has already been through five successful Phase I/IIb clinical trials showing rapid antibacterial action.

XF-73 is indicated for the prevention of post-surgical staphylococcal infections. In the US, there are approximately 40 million surgeries per annum alone where the patient is at risk of a post-surgical infection. However, within this large population there are particular groups who are at an even higher risk of infection due to the nature of their surgery or the procedures and/or their specific hospital environment in which they are treated. These higher risk surgical procedures include cardiovascular, orthopaedic and other complex surgeries. Destiny Pharma estimates that this totals approximately 14 million US surgeries per year, with this figure set to rise within the context of an ageing population.

About Destiny Pharma
Destiny Pharma is an established, clinical stage, innovative biotechnology company focused on the development of novel medicines that represent a new approach to the treatment of infectious disease. These potential new medicines are being developed to address the need for new drugs for the prevention and treatment of life-threatening infections caused by antibiotic-resistant bacteria, often referred to as “superbugs”. Tackling anti-microbial resistance has become a global imperative recognised by the WHO and the United Nations, as well as the G7 and the G20 countries. For further information, please visit https://www.destinypharma.com

Destiny Pharma announces positive data from XF-73 Phase 1 skin irritation study

Destiny Pharma plc
(“Destiny Pharma” or “the Company”)

Destiny Pharma announces positive data from XF-73 Phase 1 skin irritation study

Positive data supports product opportunities for XF-73 in dermal infection indications

Data also supports planned Phase 2 clinical trial of XF-73 nasal gel for the prevention of post-surgical infection

Brighton, United Kingdom – 26 July 2018 – Destiny Pharma (AIM: DEST), a clinical stage biotechnology company focused on the development of novel antimicrobial drugs, which address the global problem of antimicrobial resistance (AMR), today announces positive data from the first of two Phase 1 safety clinical studies of XF-73, examining the drug’s potential to cause dermal irritation. XF-73 is the Company’s lead candidate for the prevention of post-surgical staphylococcal infections.

The positive results support the potential of XF compounds in dermal infection indications and also reinforce the target product profile of XF-73 in advance of it entering a Phase 2 clinical trial under the US Food and Drug Administration (FDA) Qualified Infectious Disease Product (QIDP) and Fast Track designations. The Phase 2 study will examine a nasal gel formulation of the drug for the prevention of post-surgical infections, which is on plan to report data in H2 2019.

The Phase 1 clinical study, completed in the US under an Investigational New Drug (IND) from the FDA, demonstrated that XF-73 in aqueous solution, at high concentrations, when applied daily for five days to intact and abraded skin (by tape stripping) has a similar irritancy potential to water under occluded (covered) conditions in a shorter version of an industry standard cumulative irritancy dermal safety study. The volunteer clinical trial was blinded and placebo controlled with pharmacokinetic sampling.

This finding on abraded skin, combined with the lack of systemic absorption, (i.e. no XF-73 was seen in blood samples taken throughout the study), also supports the profile of XF-73 (and potentially other XF drugs) as new treatment options for dermal infections.

Neil Clark, Chief Executive of Destiny Pharma, commented:

“We are pleased to successfully complete this Phase 1 study, confirming the safe profile demonstrated to date in all other XF-73 clinical studies. We are also excited that the abraded skin data supports the potential for the XF platform in treating difficult skin infections and we will now assess our drug development options in that therapeutic area.”

These study results add to the existing strong safety and efficacy dataset already established by Destiny Pharma in preparation for the Phase 2 planned to report data in H2 2019 to assess the effectiveness of XF-73 nasal gel in significantly reducing nasal Staphylococcus aureus prior to surgery compared to placebo. A second Phase 1 study with the XF-73 nasal gel formulation will be completed later in 2018 in a similar skin irritation potential safety volunteer trial. On completion of this study, XF-73 nasal gel will be ready to commence the Phase 2 trial.

Destiny Pharma will now finalise a programme to develop an XF drug targeting the prevention and/or treatment of specific dermal infections. As with all XF drugs, the potential target product profile will include a safe, rapid kill of relevant bacteria whilst generating low or no AMR due to the unique mechanism and the rapidity of the XF mode of action.

For further information, please contact:

Destiny Pharma plc
Neil Clark, CEO
Simon Sacerdoti, CFO
pressoffice@destinypharma.com
+44 (0)1273 704 440

FTI Consulting
Simon Conway / Victoria Foster Mitchell
destinypharma@fticonsulting.com
+44 (0) 20 3727 1000

Cantor Fitzgerald Europe (Nominated Adviser and Joint Broker)
Philip Davies / Will Goode, Corporate Finance
Andrew Keith, Healthcare Equity Sales
+44 (0)20 7894 7000

finnCap Ltd (Joint Broker)
Geoff Nash /Kate Bannatyne, Corporate Finance
Alice Lane, Corporate Broking
+44 (0)20 7220 0500

About XF-73

XF-73 is a synthetic anti-microbial active against all tested Staphylococcus aureus strains, including drug resistant strains. By acting via a cell-surface mechanism it affects the bacterial membrane permeability and integrity, leading to cell death. XF-73 has already been through five successful Phase I/IIb clinical trials showing rapid antibacterial action.

XF-73 is indicated for the prevention of post-surgical staphylococcal infections. In the US, there are approximately 40 million surgeries per annum alone where the patient is at risk of a post-surgical infection. However, within this large population there are particular groups who are at an even higher risk of infection due to the nature of their surgery or the procedures and/or their specific hospital environment in which they are treated. These higher risk surgical procedures include cardiovascular, orthopaedic and other complex surgeries. Destiny Pharma estimates that this totals approximately 14 million US surgeries per year, with this figure set to rise within the context of an ageing population.

About Destiny Pharma

Destiny Pharma is an established, clinical stage, innovative biotechnology company focused on the development of novel medicines that represent a new approach to the treatment of infectious disease. These potential new medicines are being developed to address the need for new drugs for the prevention and treatment of life-threatening infections caused by antibiotic-resistant bacteria, often referred to as “superbugs”. Tackling anti-microbial resistance has become a global imperative recognised by the WHO and the United Nations, as well as the G7 and the G20 countries. For further information, please visit https://www.destinypharma.com

Forward looking statements

Certain information contained in this announcement, including any information as to the Group’s strategy, plans or future financial or operating performance, constitutes “forward-looking statements”. These forward looking statements may be identified by the use of forward-looking terminology, including the terms “believes”, “estimates”, “anticipates”, “projects”, “expects”, “intends”, “aims”, “plans”, “predicts”, “may”, “will”, “seeks” “could” “targets” “assumes” “positioned” or “should” or, in each case, their negative or other variations or comparable terminology, or by discussions of strategy, plans, objectives, goals, future events or intentions. These forward-looking statements include all matters that are not historical facts. They appear in a number of places throughout this announcement and include statements regarding the intentions, beliefs or current expectations of the Directors concerning, among other things, the Group’s results of operations, financial condition, prospects, growth, strategies and the industries in which the Group operates. The directors of the company believe that the expectations reflected in these statements are reasonable, but may be affected by a number of variables which could cause actual results or trends to differ materially. Each forward-looking statement speaks only as of the date of the particular statement. By their nature, forward-looking statements involve risks and uncertainties because they relate to events and depend on circumstances that may or may not occur in the future or are beyond the Group’s control. Forward looking statements are not guarantees of future performance. Even if the Group’s actual results of operations, financial condition and the development of the industries in which the Group operates are consistent with the forward-looking statements contained in this document, those results or developments may not be indicative of results or developments in subsequent periods.

Research collaboration with Aston University to investigate new XF-platform drug candidates

 Destiny Pharma plc
(“Destiny Pharma” or “the Company”)

Destiny Pharma announces research collaboration with Aston University to investigate new XF-platform drug candidates

Research to investigate potential antimicrobial candidates against biofilms

Brighton, United Kingdom – 17 July 2018 – Destiny Pharma (AIM: DEST), a clinical stage biotechnology company focused on the development of novel antimicrobial drugs, which address the global problem of antimicrobial resistance (AMR), today announces it has signed a three-year research collaboration agreement with Aston University. The research is intended to examine novel compounds from the Company’s XF-platform and assess their potential to prevent, control and eradicate dangerous bacteria and biofilms.

Serious infections are sometimes caused and exacerbated by biofilms where bacteria can hide and be protected from traditional anti-infective agents. XF compounds have already shown efficacy in biofilm models and this research project will explore that further and look at the mechanisms of action. The collaboration with Aston University will also look at other potential uses of the XF platform in the prevention and treatment of serious, drug resistant infections. Aston University’s Department of Life and Health Sciences has established expertise in in vitro bacterial biofilm models that will be utilised in the collaboration. Financial terms of the collaboration have not been disclosed.

Tony Worthington, Reader in Clinical Microbiology at Aston University, commented:

“The XF series of compounds have distinctive properties that could provide important advances in the treatment of biofilm-related infections. We are delighted to work with Destiny Pharma on the evaluation of these compounds.”

Neil Clark, Chief Executive Officer of Destiny Pharma, added:

“We are excited to collaborate with the expert team at Aston University and look forward to exploring the further potential of our XF-platform, especially in the treatment of infections involving biofilms. Biofilms represent a significant barrier to antimicrobial treatment and this collaboration, which is in line with our stated strategy, may identify additional clinical candidates that are safe, effective and with a significantly reduced level of antimicrobial resistance.”

Biofilms are an increasing problem that are poorly treated by current drugs as they act as a protective barrier for bacteria and have been traditionally associated with indwelling medical devices (for example, heart valves and joint replacements) and invasive medical devices (for example, catheters and endoscopes). It is now acknowledged that biofilms have a major involvement in bacterial vaginosis, urinary tract infections, middle-ear infections, gingivitis, corneal infections and more lethal diseases such as endocarditis and cystic fibrosis. More recently it has been noted that bacterial biofilms may impair cutaneous wound healing and reduce topical antibacterial efficiency in healing or treating infected skin wounds, including burns and leg ulcers.

For further information, please contact:

Destiny Pharma plc
Neil Clark, CEO
Simon Sacerdoti, CFO
pressoffice@destinypharma.com
+44 (0)1273 704 440

Aston University
Press and Public Relations
pr@aston.ac.uk
+44 (0)121 204 4592

FTI Consulting
Simon Conway / Victoria Foster Mitchell
destinypharma@fticonsulting.com
+44 (0) 20 3727 1000

Cantor Fitzgerald Europe (Nominated Adviser and Joint Broker)
Philip Davies / Will Goode, Corporate Finance
Andrew Keith, Healthcare Equity Sales
+44 (0)20 7894 7000

finnCap Ltd (Joint Broker)
Geoff Nash /Kate Bannatyne, Corporate Finance
Alice Lane, Corporate Broking
+44 (0)20 7220 0500

About XF-73

XF-73 is a synthetic anti-microbial active against all tested Staphylococcus aureus strains, including drug resistant strains. By acting via a cell-surface mechanism it affects the bacterial membrane permeability and integrity, leading to cell death. XF-73 has already been through five successful Phase I/IIb clinical trials showing rapid antibacterial action.

XF-73 is indicated for the prevention of post-surgical staphylococcal infections. In the US, there are approximately 40 million surgeries per annum alone where the patient is at risk of a post-surgical infection. However, within this large population there are particular groups who are at an even higher risk of infection due to the nature of their surgery or the procedures and/or their specific hospital environment in which they are treated. These higher risk surgical procedures include cardiovascular, orthopaedic and other complex surgeries. Destiny Pharma estimates that this totals approximately 14 million US surgeries per year, with this figure set to rise within the context of an ageing population.

About Destiny Pharma

Destiny Pharma is an established, clinical stage, innovative biotechnology company focused on the development of novel medicines that represent a new approach to the treatment of infectious disease. These potential new medicines are being developed to address the need for new drugs for the prevention and treatment of life-threatening infections caused by antibiotic-resistant bacteria, often referred to as “superbugs”. Tackling anti-microbial resistance has become a global imperative recognised by the WHO and the United Nations, as well as the G7 and the G20 countries. For further information, please visit https://www.destinypharma.com

About Aston University

Aston University campus pictures

Founded in 1895 and a University since 1966, Aston is a long established university led by its three main beneficiaries – students, business and the professions, and our region and society. Aston University is located in Birmingham and at the heart of a vibrant city and the campus houses all the university’s academic, social and accommodation facilities for our students.  Professor Alec Cameron is the Vice Chancellor & Chief Executive.

Aston has been a leading university for graduate employment success for over 25 years and our students do extremely well in securing top jobs and careers.  Our strong relationships with industry partners mean we understand the needs of employers, which is why we are also ranked in the top 20 for graduate employability.

The School of Life & Health Sciences at Aston University pursues interdisciplinary research at the interface between laboratory and clinic. The Microbiology Research Group is part of a team of 20 principal investigators focused on a range of processes that are fundamental to health and disease: infection, inflammation, fibrosis, metabolism, tissue modeling and regeneration. The overarching aim of this group is to promote health through specific understanding of and intervention in these processes. For further information, please visit http://www.aston.ac.uk/lhs/research/centres-facilities/cell-tissue-biomedical-research/.

Forward looking statements

Certain information contained in this announcement, including any information as to the Group’s strategy, plans or future financial or operating performance, constitutes “forward-looking statements”. These forward looking statements may be identified by the use of forward-looking terminology, including the terms “believes”, “estimates”, “anticipates”, “projects”, “expects”, “intends”, “aims”, “plans”, “predicts”, “may”, “will”, “seeks” “could” “targets” “assumes” “positioned” or “should” or, in each case, their negative or other variations or comparable terminology, or by discussions of strategy, plans, objectives, goals, future events or intentions. These forward-looking statements include all matters that are not historical facts. They appear in a number of places throughout this announcement and include statements regarding the intentions, beliefs or current expectations of the Directors concerning, among other things, the Group’s results of operations, financial condition, prospects, growth, strategies and the industries in which the Group operates. The directors of the company believe that the expectations reflected in these statements are reasonable, but may be affected by a number of variables which could cause actual results or trends to differ materially. Each forward-looking statement speaks only as of the date of the particular statement. By their nature, forward-looking statements involve risks and uncertainties because they relate to events and depend on circumstances that may or may not occur in the future or are beyond the Group’s control. Forward looking statements are not guarantees of future performance. Even if the Group’s actual results of operations, financial condition and the development of the industries in which the Group operates are consistent with the forward-looking statements contained in this document, those results or developments may not be indicative of results or developments in subsequent periods.

Destiny Pharma notes FDA statement on their focus to enable new approaches to fight AMR

Brighton, United Kingdom – 14 June 2018 – Destiny Pharma (AIM: DEST), a clinical stage biotechnology company focused on the development of novel anti-microbial drugs, which address the global problem of anti-microbial resistance (AMR), notes the FDA Commissioner, Scott Gottlieb M.D.’s further announcements this week outlining the regulator’s support of new incentives for companies developing novel anti-infectives through both financial reimbursement and further streamlined clinical trial requirements.

Neil Clark, Chief Executive Officer of Destiny Pharma commented:

“We welcome the FDA’s continued support for companies investing in the development of much needed novel anti-infectives to address the global issue of antimicrobial resistance. Destiny Pharma is well positioned to benefit from such initiatives as it continues the clinical development of its lead drug candidate, XF-73, for the prevention of post-surgical infections.”

The full FDA press release is below

June 12th 2018 Statement from FDA Commissioner Scott Gottlieb, M.D., on FDA’s efforts to foster discovery and development of new tools to fight antimicrobial-resistant infections

The increase in serious antimicrobial drug resistant infections is a critical public health concern and a growing threat to patients. According to our colleagues at the Centers for Disease Control and Prevention, each year in the U.S. at least 2 million people become infected with bacteria that are resistant to antibiotics and 23,000 people die each year as a direct result of these infections.

As more and more bacteria grow resistant to currently available antibiotics, we must tackle the issue on all fronts and seek new approaches to this persistent and potentially deadly problem. This means helping to ensure good antibiotic stewardship and use in appropriate clinical scenarios. It also means spurring the development of new antibiotics, for instance, by pursuing novel incentive models for developers that take into consideration the challenging economic and usage dynamics of these products.

Despite the growing incidence of these resistant strains of bacteria, there has unfortunately been an overall decline in antibiotic drug research driven largely by the significant obstacles to developing innovations in this category. This is especially true when it comes to developing new antibiotics that work through novel mechanisms that can evade existing patterns of resistance.

Consider patients who are hospitalized with life-threatening infections and need immediate treatment, but the severity and symptoms of the acute illness, such as delirium, may make obtaining informed consent from patients and performing other trial enrollment procedures difficult. This is just one example of the many challenges to conducting effective clinical trials in patients with serious bacterial diseases.

Complicating matters further, many patients with serious infections may have already tried several currently available antibiotics before a clinical trial is considered. If patients have already received substantial treatment for their infection before enrolling in a clinical trial, this exposure to other treatments can make it more difficult to isolate the effects of an investigational drug.

These are some of the scientific challenges. But there are also economic impediments.

Developing new drugs is a costly endeavor. But the current reimbursement model, where drugs are reimbursed based on each episode of their use, presents incentives that run contrary to effective stewardship over new antibiotics that might be highly effective against very rare and dangerous pathogens. When such drugs become available, we try to use them sparingly, lest pathogens become over-exposed to a new mechanism of attack and develop resistance to it. So, providers have imposed understandable restrictions on the use of such drugs. While this represents responsible stewardship, it also means that a novel antibiotic may have a very limited market. If product developers know that they will not be able to recoup their investments, there may be reduced incentive to invest the significant money needed to discover and develop such a drug.

The global effort to educate patients and providers about the importance of reducing the unnecessary and over-use of antibiotics is a crucially important means to slowing the rate of resistance. However, we must acknowledge that these essential antimicrobial stewardship programs do have an impact – as they should and aim to do – on the use of antibiotics and thus the amount of product sold. And as consequence, we must find ways to spur development and incentivize innovators. The FDA and other federal agencies are taking new steps to address each of these challenges, including new efforts to address the need for better economic incentives.

One of those steps is implementing the set of special incentives that Congress created for antibacterial and antifungal drugs that treat serious or life-threatening infections. This includes the qualified infectious disease product (QIDP) designation. Under this program, new drug applications that are designated as QIDP can receive fast track designation, priority review designation and a possible five-year extension of any exclusivity that the application qualifies for upon approval.

Even with these incentives, we recognize that challenges remain. As such, we continue to work with Congress, our partners at other agencies and the scientific community to find additional ways to create incentives for the development of novel antimicrobial drugs and strengthen the research and development pipeline.

One idea that we’re currently discussing with other agencies such as the Centers for Medicare and Medicaid Services (CMS) would involve changing the model for reimbursement of certain new, anti-microbial drugs that meet critical, public health needs – principally their ability to effectively target dangerous, multi-drug resistant infections.

Under such an approach, instead of paying for drugs that meet a narrow set of critical, public health criteria on a per use basis – for each prescription that’s written, as is done now – one might move instead to a licensing model. Under such a model, the acute care institutions that are most likely to prescribe these medicines would pay a fixed licensing fee for access to the drug, which would offer them the right to use a certain number of annual doses. This is similar to the way that software often gets reimbursed, where institutions pay a licensing fee for a fixed number of installations. We have been speaking with our counterparts at CMS as to whether such an approach is feasible, whether it can be formulated as a demonstration, and as a demonstration, whether it would have the intended public health benefits.

These concepts are still being developed and we look forward to greater public engagement around these ideas. Adapting this licensing payment model to drugs that target dangerous, antimicrobial resistant organisms can help achieve two important public heath goals. First, such a model would create a natural market for drugs that meet certain public health criteria, by providing a predictable return on investment and revenue stream through more foreseeable licensing fees. Second, it would put the institutions fully in charge of stewardship of these important medicines. Once they purchase the ability to access a drug, they would be stewards of its use up to a certain number of annual doses, which could be tied to the number of beds an institution has or its likelihood of encountering certain organisms.

This reimbursement model would address some of the investment challenges associated with the market for potent antimicrobials that target multidrug resistant organisms. These are drugs that we want to have available to us, but that we should keep in reserve and hope that we seldom have to use them. It is my belief that a licensing model might offer an effective “pull incentive” that attempts to create a predictable market for antimicrobial drugs that would meet a narrow set of critical, public health criteria.

We are currently discussing these ideas as part of the FDA’s broader policy work in this area. We plan to release more information soon. Such an approach potentially de-links the return on investment on an important antimicrobial drug from the volume of that drug that’s used. This would achieve an important public health purpose since these are drugs that we would want to hold in reserve.

We’re also taking other new steps to help advance development of improved antimicrobial drugs, through measures that make the development process more predictable and efficient.

Towards these ends, another new program is the Limited Population Pathway for Antibacterial and Antifungal Drugs, or LPAD pathway; established by Congress under the 21st Century Cures Act. The FDA believes this program will advance development and approval of antibacterial drugs to treat serious or life-threatening infections in limited populations of patients with unmet needs. Today FDA issued a draft guidance to assist in the development of drugs using this additional, important pathway.

This draft guidance, when finalized, will support drug development by describing the criteria, processes and other general considerations for drugs approved under the LPAD pathway. In reviewing an application submitted under the LPAD pathway, the FDA will consider the severity, rarity or prevalence of the infection that the drug is intended to treat. The agency will also consider the availability or lack of alternative treatment in the limited population.

The guidance will also assist companies in developing labeling, including prescribing information, patient labeling and carton/container labeling, to inform the medical community that the drug was approved under the LPAD pathway based on a benefit-risk assessment in a limited population.

We’ve already had meaningful, early interest by innovators in potentially developing drugs under this new pathway. We expect that development programs for drugs eligible for approval under the LPAD pathway will follow streamlined approaches to clinical development. This may involve smaller, shorter or fewer clinical trials. However, the LPAD pathway still requires these drug products meet the FDA’s approval standard for safety and effectiveness. Early and frequent communications between the FDA and drug companies interested in pursuing approval under the LPAD pathway for their products can help reduce overall product development times.

While we hope our work will help advance the development of new antibacterial and antifungal drugs, this isn’t a problem that can be addressed by our agency alone. We’re collaborating with agency partners, the broader scientific and policy community, and medical product sponsors to address scientific challenges. Bacteria will continue to evolve. They will continue to chip away at the usefulness of available treatments – including the medicines that we have relied on for years.

Many of the existing antibiotics are simply old. They were screened out of nature where they resided in soil for centuries, engaging in a natural battle with various bugs.

More judicious use of antibiotics in health care and agriculture settings can help slow the rate at which bacteria become resistant to antibiotics. But even with prudent use, we will need to continuously encourage the development of new therapeutic options to keep pace with these challenges. The steps we’re taking, including the issuance of today’s draft guidance on the LPAD pathway and the discussion of new incentive models with our partners at other agencies, are some of the additional steps we’re taking towards strengthening the fragile antibacterial drug pipeline, as part of the larger effort to combat antibiotic-resistant bacteria.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency is also responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.