According to the latest official data published by the American Lung Association, the extremely debilitating and incurable progressive lung disease known as chronic obstructive pulmonary disorder (COPD) currently impacts as many as 24 million Americans (http://dtn.fm/4hUgy). The WHO estimates that by 2030, COPD will be the third leading cause of death and has ruled the disease a global epidemic. This statistic is, unfortunately, already true here in the U.S., where we have around 13.6 million cases currently diagnosed. COPD is actually a catch-all term for a host of lung diseases and associated ailments, like chronic bronchitis (increased mucus and inflammation) and emphysema (progressive alveoli damage), as well as refractory asthma and bronchiectasis (scarring/enlargement in damaged airways), to name but a few.
Naturally, because there is no cure, there is a sizable and growing treatment market for COPD, and one whose growth is being spurred on globally by an increasingly elderly population. In fact, the U.S. Department of Health and Human Services estimates that the older population in America alone will increase by some 120 percent through 2060. Compared to 2013’s ratio of about 14 percent (one out of every seven citizens), the number of COPD patients will more than double over this interval, to a total somewhere around 100 million.
Given that women are at particular mortality risk (http://dtn.fm/bogA1) due to a sizable uptick in female smokers starting around the late 1960’s, and that women have an inordinate susceptibility to COPD when compared to men, due to their smaller-on-average lungs and the fact that estrogen plays a key role in the worsening of the disease, COPD-related death is notably higher among women. The roughly 7 million women in the U.S. who are currently diagnosed with COPD are not only severely debilitated by an inability to breathe properly, they face a higher risk of having their lives cut short by this disease, which was historically considered to be more of a man’s disease. A four-fold increase in fatalities among women over the past three decades has thrown a bright spotlight on how misdiagnosed the disease is among women, but the problem still persists, meaning that the emergence of new and easy to use treatments are highly sought after by this eager demographic.
Thankfully a number of treatments have already emerged to help prevent complications, such as airflow obstruction and bronchospasm, in COPD patients, including GlaxoSmithKline’s (NYSE: GSK) Anoro Ellipta, a so-called LAMA/LABA (long-acting muscarinic antagonist/beta agonist) once-daily, which was designed to also help maintain profitability for GSK as its mainstay Seretide/Advair (salmeterol and fluticasone) product is opened up to generic competition. One of the big hurdles generics have faced, however, is the difficulty in getting the delivery mechanism, an inhaler, properly designed – a factor which has led to much slower than expected onset of generic competition for GSK. Novartis (NYSE: NVS) also made progress in this same area last year with solid results in a pivotal U.S. Phase III clinical trial of twice-daily QVA149 (indacaterol/glycopyrronium bromide) and NVA237 (glycopyrronium bromide), as well as a more recent expansion of its relationship with Qualcomm’s (NASDAQ: QCOM) Qualcomm Life subsidiary, aimed at powering Novartis’ next-gen Breezhaler delivery system for its COPD portfolio (Onbrez, Seebri and Ultibro).
According to leading business intelligence provider GBI Research, the global COPD market is estimated to be worth approximately $11.3 billion (http://dtn.fm/vJc6I) and is on-track to grow by around 38 percent over the next few years, reaching upwards of $15.6 billion on the strength of LABA/LAMA bronchodilators by 2019. One of the extremely attractive contenders amid this multibillion dollar market is clinical stage biopharma Pulmatrix (NASDAQ: PULM), which has already completed a Phase 1b in patients with moderate to severe COPD using its branded generic bronchodilator PUR0200, for which there is currently no generic competition. PUR0200 has demonstrated efficacy at a much smaller dose than the reference target and the Phase 1b also clearly documented the efficiency of what is the company’s first small molecule formulation using its iSPERSE inhaled dry powder technology (http://dtn.fm/mW252), via comparison with traditional lactose blend formulations.
This is great news for the company and indicates to investors the potential for PULM’s technology, considering how iSPERSE now very strikingly appears to have the potential to enable delivery of entirely new classes of compounds directly to the lungs, something not possible with traditional lactose delivery technologies. An R&D partnership with global pharmaceutical developer and generics juggernaut Mylan (NASDAQ: MYL) in Europe spells big things for Pulmatrix’s PUR0200, and the drug could become the first branded generic for what is a roughly $5 billion segment of the larger global COPD market.
Moreover, the company has a robust IP position with some 37 issued patents worldwide covering its core dry powder technology, iSPERSE (inhaled small particles easily respirable and emitted), a proprietary platform solution that hurdles many of the lagging formulation problems facing the industry today that are typically seen with other lactose blend and metered dose inhaler technologies. Because iSPERSE particles don’t require a carrier such as lactose and can be engineered to carry anywhere from less than one percent to more than eighty percent of an active pharmaceutical ingredient (API), Pulmatrix has an extremely versatile delivery technology on its hands that is suitable for a wide range of drug loading roles – from small molecules, to peptides and proteins, or even antibodies. Pulmatrix could go toe to toe and even surpass sector heavy-hitters such as AstraZeneca (NYSE: AZN) and Bayer (OTC: BAYRY) in certain areas of the COPD market with this technology, and the company’s team of engineers have a deep understanding of the iSPERSE platform that spans the implantation gamut, from feasibility to clinical manufacturing, something which makes PULM a force to be reckoned with when it comes to novel inhalation products.
Indeed, such formulation prowess is just the tip of the iceberg for PULM’s iSPERSE platform, as the technology features numerous other characteristics that set it apart from competitors when it comes to significantly improving the treatment of a whole host of pulmonary diseases, as well as opening the door to important new inhalation products with important characteristics such as reproducible, one-step, scalable manufacturing – using a unique spray drying process that offers high quality, consistent yields of end product, which are completely independent of specific API physical chemistries. When you stack this advantage up against the optimum dispersibility across a range of flow rates, noting that iSPERSE formulations nevertheless feature consistent emitted dose and particle sizes, one can see that you get an easy to churn out, reliable dosing solution, a solution which is right for all patient populations and which uniquely addresses the problem of delivery variance from patient to patient.
Additionally, because iSPERSE allows for the delivery of macromolecules and biologics such as antibodies, peptides and nucleic acids across such wide range of drug loads – and because the technology enables the creation of dual and even triple homogenous combinations of multiple drugs – this platform technology is an ideal vehicle for Pulmatrix’s future candidates, and the Pulmatrix pipeline (http://dtn.fm/4vAdS) already offers some other exciting candidates alongside its lead, such as PUR1900 and PUR1500, designed (respectively) to treat the pulmonary fungal infections that affect half or more of all cystic fibrosis patients, as well as the loss of lung tissue oxygen transport capability common in idiopathic pulmonary fibrosis.
PUR1900 is particularly interesting, as it has been shown to be both active and potent in animal models (http://dtn.fm/6HxEb), achieving high lung concentrations and low systemic exposure, and because it would be the first ever inhalable anti-fungal for cystic fibrosis. Because PUR1900 directly targets aspergillus infection – which is typical of several other conditions such as suppressed immune function among leukemia-related chemotherapy or tuberculosis, and is seen in non-invasive nose, ear and eye infections – there are tantalizing upper limits when it comes to PUR1900’s broader applicability.
With a $1.7 million NIH research grant under its belt to work on an inhaled anti-fibrotic with Celdara, Inc. (http://dtn.fm/bTLl5), as well as a strong cash position of some $22 million (http://dtn.fm/FE1f9) that should see development efforts at the company through to the middle of next year, Pulmatrix is in an enviable position, with a robust pipeline of candidates, the capital muscle, and the bedrock tech to back up its aspirations. The company has a truly impressive drug delivery/manufacturing technology in iSPERSE that should continue to produce upside moving forward and investors will want to keep a close eye on PULM for news regarding further PUR0200 developments that are on the immediate horizon.
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