Today is February 28, Rare Disease Day, which is focused on raising awareness about the prevalence and impact of rare diseases. As such, I’ve decided to focus this blog entry on orphan lung disease. However, I want to start by sharing something more personal: why this job fills me with inspiration and meaning.
I was born and raised in Durban, South Africa. With its long sunny beaches, lush grasslands and subtropical climate, South Africa is a nature-lovers paradise. But this beautiful backdrop stands in stark contrast to alarming statistics of a deadly disease plaguing the country – tuberculosis (TB). TB is a lung infection caused by the Mycobacterium tuberculosis bacteria, a cousin of nontuberculous mycobacteria (NTM). With a quarter of the world’s population infected with TB, it is one of the planet’s deadliest diseases and South Africa is one of the countries with the highest TB burden. It’s estimated that approximately 1% of South Africa’s 50 million people will develop active TB each year, and this incidence rate has increased by 400% over the past 15 years. As is the case with many drugs that attack the bacteria directly, there is an increasing problem of antibiotic resistance with TB treatments. Of personal interest to me, is a subset of the TB population that have multi drug-resistant and extensively drug-resistant TB.
When I think about the challenges facing South Africa, and the opportunities I have been afforded, I come back again and again to the words of Nelson Mandela:
What counts in life is not the mere fact that we lived. It is what difference we have made to the lives of others that will determine the significance of the life we lead.
Driven by an intense passion to help abate the growing worldwide issue of drug-resistant TB, I left the high-tech and healthcare IT industries and turned to biotech. Having founded and exited a number of companies, I had achieved some commercial success. But something was missing. Commercial success wasn’t the whole story for me and I needed to find a healthy balance between extrinsic and intrinsic rewards. I was looking for “work-life mission” – a role holding deep meaning for me personally. This meant combining work and personal goals into a single mission focused on transforming therapeutics for people living with lung disease.
By co-founding Savara, an orphan lung disease company, along with a group of biotech veterans with expertise in the space, I found a way to tie my business strengths and personal passion to my career. Being part of a team that could develop life-changing therapies for people living with orphan lung disease was fully aligned with my desire to make a difference. This intrinsic “calling” is what motivates me and makes me an unwavering believer in Savara’s vision of becoming THE orphan lung disease company.
You may be asking yourself, “what is an orphan lung disease?” That’s an important question because, to understand Savara’s vision, you need to understand our target market. An orphan disease is just as its name suggests: a rare and neglected disease for which there are, often, no viable therapeutic treatments.
The U.S. Food and Drug Administration (FDA) defines an orphan drug based on prevalence rates of fewer than 200,000 people in the U.S. While prevalence rates are low for each individual orphan disease, creating new, groundbreaking therapies is just as important to these smaller patient populations as it is for those living with a disease of epidemic proportion. Interestingly, the commercial opportunities in orphan diseases are often as robust as those of non-orphan diseases, with favorable protection and regulatory pathways. For example, the FDA has developed incentives to encourage orphan drug innovation. Under certain conditions and requirements, the agency can grant an “orphan drug designation” to an investigational therapy, whereby the product would have 7-years of market exclusivity, reduced regulatory filing fees, tax incentives and distinct regulatory requirements for approval, to name a few.
Why should we care about orphan lung diseases?
Despite the “orphan” nomenclature, when taken in aggregate these rare lung conditions aren’t so rare. It is estimated that there are as many as two million Americans living with a rare lung disease. While global epidemiology data on orphan lung disease aren’t readily available, you can extrapolate that prevalence rates could extend into the tens of millions of people worldwide. Within that population, we estimate that there are well over a hundred different orphan lung diseases, the majority of which result in a significant unmet medical need due to limited understanding about, or inability to diagnose, the condition as well as few (if any) approved therapies. And without research and development efforts from companies like Savara, people living with these diseases are without hope for a cure or interventional therapy. Just like someone living with cardiovascular disease or diabetes, people with a rare lung disease want to understand their illness. What causes the disease? What symptoms should I expect and what is my prognosis? What treatments are available to me?
The heterogeneity of these diseases makes it hard to advance the entire orphan lung space. Savara’s strategy is to leverage our capabilities and overlapping relationships with clinicians, regulatory divisions, and commercial platforms in the orphan lung disease field, with the ultimate goal of bringing numerous innovative therapies to market. We will achieve this through indication expansion and acquisitions that will address significant unmet need for many orphan lung diseases. Additionally, it is our hope that by advancing the scientific understanding of certain diseases, we will spark innovation and intervention in other areas of rare lung disease. As the saying goes, “a rising tide lifts all boats.”
At Savara, we are committed to fighting against orphan lung disease. We strive to create differentiated therapies for people living with these underserved, and often chronic, lung conditions. My job at Savara is not a mere vocation; it is demanding and fulfilling, deeply challenging and profoundly rewarding. After more than a decade at the company, I have found balance by working for an organization that can realize commercial success, punctuated by a critically important mission.
We invite you to regularly follow these blog posts. As we move into 2019, we’ll use this communication tool to (hopefully) inspire you and provide important information about the markets we serve. Over the coming months, please stay tuned for commentary from thought leaders in orphan lung disease, opinion pieces from Savara leadership, insights from advocates and more – with a focus on autoimmune pulmonary alveolar proteinosis (aPAP), a rare and debilitating autoimmune disease caused by the build-up of excess surfactant in the lungs. We expect top line results from IMPALA, a clinical study evaluating our lead asset, Molgradex, for the treatment of aPAP at the end of the second quarter this year. We look forward to sharing the results with you and the PAP community.
Chief Executive Officer
Savara cautions you that statements herein that are not a description of historical fact are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words referencing future events or circumstances such as “expect,” “intend,” “plan,” “anticipate,” “believe,” and “will,” among others. Such statements include, but are not limited to, statements relating to Savara’s unique opportunity to develop differentiated therapies for people suffering from devastating, and often chronic, lung conditions, our ability to deliver innovative therapies and improve patient outcomes being dependent on the achievement of our corporate objectives, the timing of the announcement of interim results from our OPTIMA study and the data included in that announcement, that Savara is entering unprecedented territory in its evolution as a company, the timing of our key clinical milestones in 2019, including data read-outs and study-starts, that one of the data read-outs could facilitate the submission of a BLA in 2020, that 2019 will be a transformative year for Savara, that these catalysts keep us on track to develop a strong portfolio of best-in-class therapeutics that address a broad range of rare lung diseases, that Savara has a chance to fundamentally improve quality of life for people living with these rare afflictions, and the belief that the best is yet to come. Savara may not actually achieve any of the matters referred to in such forward-looking statements, and you should not place undue reliance on these forward-looking statements. These forward-looking statements are based upon Savara’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with the outcome of ongoing clinical trials for our product candidates, the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient resources for Savara’s operations and to conduct or continue planned clinical development programs, the ability to obtain the necessary patient enrollment for our product candidates in a timely manner, the timing and ability of Savara to raise additional equity capital as needed to fund continued operations; the ability to successfully develop our product candidates, and the risks associated with the process of developing, obtaining regulatory approval for and commercializing drug candidates that are safe and effective for use as human therapeutics. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. For a detailed description of our risks and uncertainties, you are encouraged to review our documents filed with the SEC including our recent filings on Form 8-K, Form 10-Q and Form 10-K. You are cautioned not to place undue reliance on our forward-looking statements herein, which speak only as of February 28, 2019 which is the date on which they were made. Savara undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after such date, except as may be required by law.