Coronavirus: Treatments and Vaccines

Recently a science teacher said that treatment for covid with remdesivir or hydroxychloroquine is better than a vaccine. I said, treatment like that is good but a vaccine is better. She said, “no. since many people get adverse reactions from vaccines, a treatment is better”. So I decided to write this blog post. First, let us consider what are drug treatments and how they are different from vaccines.

Drug treatments: Currently over 70 companies are working on various drug treatments for novel coronavirus. Although this virus is novel, not all drugs being considered are entirely novel. For instance, like the previously considered malaria drug, hydroxychloroquine, Gilead’s drug, Remdesivir, that is in the news, is not a novel drug. Remdesivir is a broad spectrum antiviral drug that has been found to successfully prevent MERS coronavirus in rhesus monkeys. Besides antiviral drugs, there are other anti-inflammatory drugs. Scientists believe that in the end, what might work may be a sort of a cocktail or some combination of antiviral and anti-inflammatory drugs.

Drug treatments do not prevent Covid-19. These drugs are offered to patients who are already suffering from coronavirus. On the other hand, vaccines prevent people from contracting the disease in the first place.  

Drug treatments are not cures. 1) there isn’t a 100% response rate among people treated with remdesivir or any other treatment. So despite many drugs showing promise, there are a few people who will respond to a drug; many still die. 2) many who suffer from the disease but go on to recover, they still utilize huge amounts of hospital resources during their illness phase. 3) during the illness phase, these sick people also spread the virus with the possibility of making others (including healthcare professionals) sick. 4) even people who may recover with the help of these drugs from covid, may have suffered substantial damage to their lungs and that may reduce their lifespan or affect them in other ways.

Vaccines:  Vaccines prevent people from getting sick. So when people don’t get sick, they don’t spread the virus, don’t utilize healthcare resources, don’t infect healthcare professionals or others in the community, and don’t get any kind of long lasting damage to their lungs. Clearly, vaccine is a far superior way to deal with coronavirus.

Why are vaccines taking much longer than drugs for covid?

As I mentioned above, many of the drugs considered currently for treatment of covid are already in existence for some other illnesses or diseases. So there are existing drugs considered for either preventing covid deaths or preventing people from becoming sicker and helping them recover faster and ending hospital stays earlier.

Vaccines on the other hand, have to be developed. There are considerable challenges at each stage of vaccine research, development and manufacturing. First of all, pharmaceutical companies have decreased vaccine research and development in the last several years, because vaccine development and manufacturing is expensive, time consuming and offers smaller payback in terms of revenues. For each new virus and for all mutations, the vaccine has to be developed and then when the virus is not active then the vaccine is not useful. Clinical studies take place during the research and development phase. Only if the studies show the vaccine to be safe and effective then the vaccine proceeds to the next phase where it has to be manufactured in large quantities required.

During a pandemic like this one, not only do we need a vaccine quickly but the world will also need  hundreds of millions of doses for the entire world population.  Fortunately, pharma companies, scientists from all over the world, billionaires like Bill Gates and Governments and countries have stepped up to tackle this challenge, with an unprecedented spirit of collaboration.  There are measured expectations for an effective vaccine to be available within 12 to 18 months. That is an unprecedented and highly aggressive timeline. Bill Gates has offered to support manufacturing of top 7 candidates during the research and development phase. That means, even without the proof of effectiveness of any of them, he will foot the bill for manufacturing all of them, in the hope that at least a couple of them will prove to be safe and effective and the world will get required doses speedily. Meanwhile, countries like India are getting ready for manufacturing of vaccines. India has proven capability and expertise to ramp up production of vaccines, while keeping costs down. Regulatory hurdles are likely to be less severe as US FDA and other regulatory bodies have promised EUA (emergency use authorization) for drugs and vaccines that may help contain covid.

If drugs like Remdesivir work then that will help some people recover and recover faster; so let us hope that any one of these drugs alone or in combination with other drugs will reduce mortality, shorten hospital stays, and help reduce spread of covid. But ultimately we need a vaccine for covid-19 and the way in which entire world is coming together, gives much hope for availability of a speedy vaccine.

Cloud-based eClinical Systems – Myths, Challenges & Opportunities

Al Gicqueau, CEO & President at Clinovo talked about Cloud-based eClinical Systems to make clinical trial process more efficient and cost-effective, at www.bio2devicegroup.org event.

Cloud has been a big buzz word, significantly impacting the economy, in the last few years.  Cloud is growing 3X faster than traditional internet infrastructure, said Gicqueau.  Worldwide public cloud services market will total to over $73 billion, by 2017.  There is also simultaneous cloud-bashing.  According to Citrix research, majority of the Americans don’t understand it and over 51% think it has something to do with the stormy weather.  Most also believe they have never used it but over 95% of us have used cloud based services.

It is therefore important to understand what constitutes cloud based services.  There are 5 essential components of cloud based services.
Self Service, On Demand: Cloud based services are available, when the consumer needs them.  Further, for the most part  they are autonomous and the user can perform the actions without going through the IT department.  They are easy to use and on-site training will increasingly become a thing of the past.  Any training required has to be available on-line and has to be very short and for the most part the service has to be intuitive and should not require training.
Broad Network Access: Cloud based services provide a broad internet access.  For instance, consider gmail.  It can be accessed through desktop, laptop, tablet, smart phone etc.  Cloud based services enable an ability to easily synchronize information over multiple devices.
Resource Pooling: Amount of traffic over the internet is rapidly growing. Because of the distributed nature of the internet, there is no single point of measurement for total internet traffic.  But it is a fact that the total global IP traffic will pass the zettabyte threshold by the end of 2015.  By the year 2017, the total internet traffic is expected to reach 5.3 zettabytes.  To put it in perspective, if the 11 oz coffee on your desk equals one gigabyte, a zettabyte will have the same volume as the great wall of China!  Cloud based services enable customers to pool their resources and save cost.

Rapid Elasticity: In the world of internet activity there are lot of peaks and valleys.  Cloud can scale based on demand peaks, without incurring penalty for the period of low traffic.
Measured Service: Cloud offers and ability to pay as you go.  People can pay for the internet infrastructure as they pay for electricity.
Some of the examples of cloud based services include, SalesForce, Netflix, Gmail and Amazon.

Clinical Data Trends

Spiraling costs have been a grave concern in healthcare.  Typically, efficiency has not been very high in the area of healthcare. Costs of clinical trials is likely to increase even more significantly, in future, on account of increasing costs of medical research and changing and tightening regulations, among other things.  Increased costs for clinical trials will push the cost of drugs higher.  On the other hand, there is strong public criticism of higher costs of medicines and there is a lot of pressure on drug companies to contain costs.  Companies have pressure to cut the middle layers and manage clinical trials on their own.  Citing CISCRP (Center for Information & Study on Clinical Research Participation) study, Gicqueau said, currently, only 6% of clinical trials are completed on time, whereas 72% run late by over a month.

Compliance issues and regulations specified in 21 CFR, part 11, prohibit use of public cloud, for clinical data.  Clinical data has to be stored in a private cloud.  Clinovo’s ClinCapture is a cost-effective EDC (electronic data capture) system.  It is tailored to specific clinical studies teams and offers intuitive navigation and one-click access to routine functions.  It reduces time for data entry.  ClinCapture is also flexible and can be customized and deployed rapidly.  “We validate our software like medical devices are validated”, said Gicqueau.

Mobile is next major trend, as more data entries are happening through tablets and smart phones.  Tables are also very useful in remote regions of the world where cell phone reception may be spotty or non existent, where information can be easily synchronized later.  Data entries can also be structured  by getting patients involved.  Data integration is another big challenge.  Everyone hopes to make sense of the data and make meaningful use of the data.  However, making sense of the data and putting it to good use remains expensive.  Gicqueau said, meaningful data integration is another promise of the cloud.

Clinovo is launching CloudClinica, next generation, cloud-based eClinical platform.  With its easy to use, pay as you go platform, CloudClinica will eliminate IT dependency and allow small companies  to manage clinical studies in a sophisticated manner, without high level programming skills.  About 30% of cost and 60% of time associated with clinical trials is about data management, and almost 80% of clinical trials are still conducted on paper, said Gicqueau.  Paper has many pitfalls.  Paper can get lost, it is inefficient, there is challenge of mis-reading someone’s handwriting, it has regulatory risks and other hidden costs.  CloudClinica is FDA compliant and it can scale.

Clinovo had revenues of over $4 million in 2013 and raised $500,000 from business angels over the last few months to execute on their business strategy.   The company is profitable, and has 30+ clients that include Gilead, Roche and others.  Clinovo is now targeting small to mid-size companies, said Gicqueau.  Current market of $2.3 billion can be rapidly growing in the coming years.  MediData and Oracle are two dominant players but are relatively more pricey.    Clinovo’s CloudClinica will fill in the gap and broaden the use of eClinical systems and will empower and bolster the biotech, pharmaceutical, and medical device companies, said Gicqueau.  The event was followed by Q&A.

2013 J P Morgan Healthcare Conference – Pharmaceutical & Biotech Company Presentations

2013 J P Morgan Healthcare Conference – Pharmaceutical & Biotech Company Presentations 

Disclaimer – Below is my best attempt to capture highlights from company presentations, at 2013 J. P. Morgan Healthcare Conference, in San Francisco, CA.  Please check details against more authentic sources, before making any financial decisions.  My writeup on the overview of the conference can be found at this link – http://bit.ly/UY1Cpk .  My article on the highlights from some of the medical device company presentations can be found at this link – http://bit.ly/WfhGmU .  Big Pharma industry historically enjoyed growth rates in double digits and 20% operating margins with growing demand and big profits.  But times are changing for big pharma with challenging healthcare environment, cost pressures, and patent expirations.  Many firms responded to initial challenges by investing heavily in next-generation drugs, often based on biotechnology.  Though promise of biotech has not panned out, many pharma companies have done restructuring, initiated serious cost containment and have nurtured the pipeline by investing in some promising molecules.  See below, highlights from some of these companies.

 

Amgen – 2011 & 2012 were solid years and advanced pipeline offers promise for 2013

Both 2011 and 2012 were solid years for Amgen, with revenue growth of 11%, said COO and CEO elect, Bob Bradway.  He credited the growth largely to the newly launched products, particularly denosumab franchise that include XGEVA and Prolia.  In first year in market, Amgen has recouped over $500M of revenues from this new franchise.  Amgen has returned 36% back to the shareholders.  Prolia accounts for $1B in sales in the US.

Besides growth in its core franchise and advanced footprint, Amgen also has exhibited strong commercial execution with solid performance in the US and international markets, said Barclay.  Sales of Neulasta grew and 40% of patients receive it during first chemo.  Enbrel is Amgen’s leading molecule in rheumatoid arthritis and psoriasis space and is steadily growing.  Sensipar, Nplate, and Vectibix also have strong and steady momentum. There was 18% growth in 2011 on these new products.

Additionally, there is a late stage pipeline emerging that looks promising.  It includes, molecules AMG 145, an antibody PCS K9 for hyperlipidemia for high cholesterol, AMG 785 antibody for post menopausal osteoporosis, AMG 827 (in collaboration with Astra Zeneca) for psoriasis, AMG 416 for secondary hypothyroidism, and several oncology molecules, including AMG 102 for gastric cancer and AMG 386 for ovarian cancer.

Amgen will continue collaborations with other significant players.  Recent acquisitions that are advancing its pipeline include Micromet, decode, and finally Mustafa Nevzat to help establish a presence in Turkey, an emerging market for Amgen.  Amgen will continue to return significant capital to shareholders and in 2013, Amgen will focus on growing revenue, operating efficiencies, advance its pipeline, and focus on ROI.

Bristol Myers Squibb – will its promising portfolio deliver in 2013?

While 2012 was a year of transition, BMS is now poised to deliver by driving growth of key brands, executing on new product launches, and driving late stage pipeline said, CEO, Lamberto Andreotti.

BMS’s diversified portfolio includes key brands Eliquis, Yervoy, forxiga, Orencia, Sprycel and so on.  Acquisition of Eliquis for stroke prevention and systemic embolism for Afib, offered a significant global opportunity and it was launched in Europe, US, Canada, and Japan.  BMS has a broad portfolio targeting diabetes, in partnership with Astra Zeneca and it includes Amylin, onglyza, forxiga, and Byetta.  A robust late stage portfolio targeting HCV includes some promising molecules.  Finally, immuno-oncology continues to remain an exciting portfolio.  Yervoy is showing encouraging 5 year survival data for melanoma patients, Nivolumab is in broad stage III program, and Elotuzumab phast III studies are looking promising, said Andreotti.

BMS is committed to 3% increase in dividend for 2013 versus 2012, has completed initial $3B share repurchase program and has announced additional $3B share repurchase program, and will continue to focus on business development through licensing, acquisition, and partnerships.  BMS is well poised to deliver with improved decision making, more efficient operations, and greater collaboration, said Andreotti.

Merck – Exciting Pipeline & Plans to Expand Geographic Footprint

Celebrating first year as a combined company, following the merger of Schering-Plough and Merck, the company is well poised to outperform the broader healthcare market, said Ken Frazier, President & CEO of Merck.  Its broad portfolio includes market leading medicines and vaccines, Merck has expanded geographic footprint in key markets, and has a strong exciting late stage pipeline.

While sales force was reduced in the US by 10%, in emerging markets like China, Brazil, and Russia, Merck was increasing the presence.  Despite 2012 being a year of maximum disruption, with blending sales forces, training reps on new products etc., the company maintained top line growth with 7 of the top 10 products growing at a steady pace, said Frazier.  The current late stage pipeline includes 55% legacy Merck compounds and 45% legacy Shering-Plough compounds, affirming the scientific productivity of both organizations, said Frazier.  Growth strategy going forward includes plan for geographic expansion in Japan and emerging markets, delivering on the pipeline, and expanding broader portfolio of business that include Merck BioVentures and animal health business. Some significant brands include, Simponi which is launched in 18 countries, with great success.  There are five new drug approvals including Dulera in the US, and Brinavess, Daxas, Elonva and Sycrest in Europe.

While strongly focusing on internal innovation, Merck has also signed 46 significant outside deals with external partners including most recently the SmartCells deal in diabetes.  Merck has over 20 drugs in Phase III incuding Vorapaxar, Tredaptive, Anacetrapib, and Odanacatib.  Merck will continue to remain committed to cardiovascular space, with currently over 100,000 patients in outcome studies and though costly now, the eventual aim is that these will lead to drugs that will reduce cardiovascular events, heart attacks, and strokes.  Vorapaxar is the largest study with 40,000 patiens and Tredaptive has 25,000 patients. While Merck is number two pharmaceutical company, globally, it is number five in emerging markets and going forward, plans to expand presence in these markets from current 18% total sales to 25% by 2013, said Frazier.  The plan is to grow the current portfolio in oral antibiotics and vaccines, as well as in women’s health.  Additionally, given the rise in chronic diseases, Merck plans to grow in respiratory, cardiac, and diabetes space.  Januvia is already number one oral diabetic product in emerging markets.   Merck acknowledges that countries like China are surrounded by strong science and innovation in those markets can be a huge contributor.  Merck aims to grow through value creating partnerships in those markets that would help in issues like pricing, reimbursement, market access, and low cost manufacturing.

Gilead – In addition to targeting Hep C, also continues to grow & remain a Leader in HIV Treatment

2012 has been a busy year for Gilead, said Chairman and CEO, John Martin.  Gilead’s current commercial portfolio includes HIV, liver, respiratory, cardiovascular and other areas.  The biggest part of revenue generation has been HIV.  Truvada, the single regimen pill is steadily growing and is made available in low and middle income countries at steeply discounted prices or through generic licensing partners.  In 2012, Gilead launched single table regimens, Atripla and Completera for HIV, distributed through join ventures with Merck and Janssen Therapeutics.  Also in 2011, Gilead extended licenses to Indian partners to grant them future rights to produce generic versions of single table regimen, Stribild.  Stribild was approved by US FDA, in August, 2012 and approval in Europe is expected in 2013.

Gilead’s pipeline includes Tenofovir, delivered directly to lymph nodes and showing greater efficacy, Sofosbuvir for Hep C, and Simtuzumab targeting Ideopathic Pulmonary Fibrosis.  While much of the focus in 2012 had been on Gilead’s Hep C pipeline, it seems the company’s HIV franchise may emerge as a strong growth driver, than previously anticipated.  The number of people in developing countries receiving Gilead antiretroviral therapy has increased from less than 30,000 in 2006 to over 3 million in 2012 and one-third of people treated for HIV in developing countries receive Gilead medicines.  

Eli Lilly – Poised to deliver with 13 molecules in Phase 3, and 20 molecules in phase 2

Chairman, President, and CO John Lechleiter shared that Lilly’s total revenue is over $20B, with gross margin of revenue, approximately 78%, $3 billion in net income with at least $4 billion in operating cash flow.  Lilly will continue to stay the course and implement the strategy that includes, replenishing and advancing the pipeline, driving growth in countercyclical growth areas, and increasing productivity to fund R&D.

In 2008, Lilly made the largest acquisition in Lilly’s history, by acquiring ImClone, to advance its oncology pipeline.  In 2012, Lilly launched Amyvid in US, got approval for Erbitux and Jentadueto.  Cymbalta and Zyprexa are approved for new indications in Japan and Cialis is approved in Europe for once daily use for BPH.    Some of the setbacks included slower emerging market growth, due to patent expirations.   Growth is expected from dulaglutide in collaboration with Boehringer Ingelheim to target diabetes, launch of Tradjenta in 30+ markets, and from Lilly’s animal health division Elanco’s acquisition of Janssen Pharmaceuticals animal health business.  Lilly is poised to deliver with rich pipeline that includes 13 molecules in Phase 3, and 20 molecules in phase 2, said Lechleiter.

Glaxo Smith Kline –  Refocused on Science & Revamped R&D Engine

Patrick Vallance, President of Pharmaceutical R&D opened the presentation  with assurances that GSK has re-engineered its drug discovery organization, has built a late stage pipeline, restructured its commercial and manufacturing to support the pipeline and will deliver value.  Given where GSK was a year ago, there seemed to be a marked progress in terms of its late stage pipeline.

Six new drugs that completed phase III studies in 2012 include dabrafenib and trametinib in oncology, albiglutide targeting diabetes, dolutegravir targeting HIV and Relvar and UMEC/VI for respiratory diseases.  In oncology, combination therapy with both molecules is indicating more complete blockade of critical pathway and ability to prevent or delay emergence of resistance.  For type II diabetes, albiglutide, first once-weekly fully humanized GLP-1RA regimen that can be administered with a pen device, is indicating opportunity to delay use of basal insulin and no weight gain.  For HIV, current trials indicate dolutegravir to be statistically superior to Atripla at week 48.  GSK has a broad portfolio targeting respiratory diseases.  Relvar/Breo for asthma and COPD, appears well tolerated and efficacious at lower doses and shows significant improvement in lung functions compared to FF or FP.  UMEC/VI for COPD, once daily, is potentially first in class in the US and indicates statistically significant improvement over placebo.  GSK has more advanced portfolio in respiratory disease space.  GSK has reengineered to deliver sustainable pipeline flow with visible multiple waves of pipeline delivery, said Vallance.  It seems that GSK has refocused on science and has revamped its R&D engine.  Will it deliver?

Baxter – Diverse Core Portfolio, Robust R&D Pipeline, Targeted Acquisitions – (stock worth watching)

Baxter CFO, Robert Hombach began the presentation with a reminder that this $38 billion healthcare company is one of the most diversified companies presenting at this conference.  Baxter focuses on acute and critical care business and on treatment of chronic diseases.  Emerging markets account for 20% of sales.  Baxter has a diverse core portfolio, a robust R&D pipeline, and engages in targeted acquisitions.

BioScience accounts for $6.1 billion business and represents vaccines, bio surgery and hemophilia.  Baxter is advancing pipeline of novel recombinant proteins.  Bio surgery business continues to be double digit growth driver.  Advate, a targeted therapy for hemophilia A, which affects approximately 1 in 5,000 males, is also a strong growth driver, showing to be very safe and efficacious and remains a gold standard of therapy. Investors believe Advate sales will continue higher, on account of FDA’s recent approved a higher dosage strength of the drug, in 2012. The higher dose allows patients to be treated only once every three days, which is a first in the hemophilia market and gives Baxter a sustainable advantage.  Medical products business account for $7.8 billion in sales in 2011.  Its key renal business accounted for 32%.  Baxter acquired Swedish-based Gambro, a kidney and liver dialysis company, for $4 billion, in late 2012, to integrate end stage renal market and enhance worldwide reach.

Baxter’s late stage product pipeline in 2012, includes 18 molecules in phase 3.  SubQ self-administration therapy of Gamnagard Liquid for Primary Immunodeficiency is showing favorable tolerability pipeline including low infusion site reaction and is advancing label indications.  A possible future blockbuster drug, Baxter has multiple data releases of Gamnagard for Alzheimer’s disease this year.  There is a probability that Baxter receives FDA approval for Gammagard Liquid in mild/moderate Alzheimer’s in coming years.  Baxter’s expanding portfolio to new therapeutic areas include, CD34+ stem cells as a possible treatment for chronic myocardial ischemia, anti-MIF antibody targeting MIF protein that influences tumor growth, and Rigosertib, a novel targeted anti-cancer compound.  Baxter has partnered with Momenta Pharmaceuticals, in a $452 million deal, to tap into the biosimilars market.   Baxter is continuing the innovation in home dialysis, with over 30 new products in the pipeline and has potential to contribute up to $250 million to its top line.  This is one of the richest pipeline in Baxter’s history, said Hombach.

Abbott – New Diversified Healthcare Company with Focus on Nutrition, Established Pharma, and Medical Devices

Abbott is a large cap diversified healthcare company and is now separate from new company, abbvie, a large cap biopharmaceutical company, said CEO, Miles White.  The new Abbott Laboratories is strongly position with $5.9 billion sales in medical devices and $5.4 billion in sales in established pharma market, in 2011.  Diagnostics accounted for an additional $4.2 billion in sales and nutrition accounted for $6 billion in sales, in 2011.  There is an increasing emerging market presence with sales targeted to approach 50%, by 2015.

Abbott enjoyed leadership position in large markets in several industry sectors.  In diagnostics, Abbott hold number 1 place in immunoassay diagnostics and in blood screening and has a leading point of care platform.  In nutrition, it is number 1 in adult and pediatric nutrition and is a leading science based nutrition company.  In medical devices, Abbott is number 1 in DES, BMS, BVS, and number 1 in Lasik and number 2 in cataract, said White.  Diabetes and vision care is driving growth in emerging markets.   Abbott also has robust vascular pipeline and in 2012, launched Absorb, a bioresorbable vascular scaffold for treatment of CAD, in Europe, Asia, and Latin America.  In established pharmaceuticals, Abbott is a leader in branded generics, with a broad portfolio that includes, 500+ branded generics, a strong development pipeline, and 60% presence in emerging markets.

AbbVie – Will other products pick up slack from Humira’s patent expiration for more agile new company?

AbbVie is the new spinoff company, from Abbott Laboratories, focused on “research-based pharmaceuticals.”   It is a global biopharma company, with focus and agility of a biotech, said CFO, Bill Chase.  AbbVie has revenues of about $18 billion and Humira, a prescription drug for rheumatoid arthritis, is its best asset.   Humira is a huge product, generating about $9 billion in sales.  There is a concern with Humira’s pending patent expiration in 2016, but Chase outlined some future plans.  AbbVie will focus on achieving Humira’s full potential through new indications, maximizing product portfolio, advancing pipeline, and leveraging global footprint.

Humira has recently been approved for ulcerative colitis.  Beyond Humira, other growth brands and durable performers include, Andro Gel, leading in testosterone replacement, Lupron, Synagis1, Creon, Synthroid, Kaletra, Norvir and Zemplar.  Humira faces stiff competition, in addition to patent expiration.  Will AbbVie’s other products easily take up the slack?

Bausch & Lomb – Only Global Company Focused on Eye Care Focused on Future Growth & New Products

B&L is the only global company found on caring for the eyes, said, President & CEO Brent Saunders.  B&L is an iconic brand with 160 year history.  The company manufactures and markets eye health products that include contact lenses and lens care products for Astigmatism, Presbyopia, Nearsighted/Farsighted, and cataract patients.  The company also provides intraocular lenses and delivery systems, ophthalmic surgical devices and instruments, and ophthalmic pharmaceuticals; vision shaping treatment products; dry eye products; allergy/redness relief products; eye wash products; eye vitamins and so on.  Three important worldwide trends, the aging population, higher diabetes prevalence, and emerging middle class more interested in eye care, will lead to increased demand, said Saunders.

At B&L, 80% of business is in cash market.  Some of the upcoming B&L products to watch are, BioTrue, one day lenses made from a next generation, bioinspired material called HyperGel, Victus Fentosecond Laser platform for enhanced performance across cataract and corneal procedures, and enVista, new glistening free, hydrophobic acrylic IOLs.  What is in its future?  Will there be a buyer willing to offer multi-billion dollar deal or will this private equity firm turn to an initial public offering (IPO)?  Following the company presentation, Saunders mentioned that the company is focused on future growth and also “aspiring to return to public markets”.

Cerulean Pharma – Private Company with Nanoparticle Based, Dynamic, Targeted Drug Delivery Platform

Cerulean Pharma develops and markets novel, intelligently designed, nanoparticle based technology to target tumor agents, in fight against cancer, said CEO Oliver Fetzer.  Tumor is targeted via leaky vasculature, used at entry portals into the tumor tissue.  Cerulean nanoparticles are are small enough to penetrate these tumor blood vessels but are too large to enter health tissue.  These nanoparticles are transported through the tumor tissue up into tumor cells and then the drug is gradually released within tumor cells. The drug is covalently bound and dynamically releases within tumor cells and the nanoparticles eventually disintegrate over time.

While chemo works well in getting the tumor cells, it also harms the healthy tissue.  History of oncology is somewhat gloomy.  With surgery, radiation, combination therapy, chemo, personalized medicine, immunotherapy etc., 5 year survival is still only 50%.  This kind of dynamic tumor targeting has two important benefits.  By focusing on tumors and sparring healthy tissue from unwanted exposure, Cerulean nanopharmaceuticals leads to limited side effects, while also enhancing therapeutic results.  The linker that attached the drug to the nanoparticle is selected to provide optimal intra-tumor drug release, by gradually breaking apart inside the tumor and releasing the drug payload over time.  The current trials indicate significantly lower toxicity and higher effectiveness with drugs with high adverse profile, like Docetaxel, Erlotinib, and Pemetrexed.  Cerulean’s own small molecule oncology product pipeline in trials, includes CRLX101, CRLX301, and siRNA delivery platform.  Cerulean develops products with partners by marrying their molecules, marketed or in development, with their nanopharmaceutical platform.