On “Building a Pearl” – Story of Pearl Therapeutics from Concept to Acquisition by Astra Zeneca for $1.15B

Pearl Therapeutics is a huge success story in the biotech world.  At www.bio2devicegroup.org event, Dr. Sarvajna Dwivedi, co-founder of Pearl Therapeutics talked about his own professional journey and about Pearl team, technology and products, that brought the company to pearly stunning $1.15 Billion exit.  Pearl Therapeutics was acquired by Astra Zeneca in June, 2013.

Dwivedi began the talk by paying tribute to his mentors, his father, Professor Rewa Prasad Dwivedi, his first role model and a reknowed Sanskrit scholar and poet, his professor Dr. Alan Mitchell  at University of British Columbia who imparted important lessons on staying true to fundamentals and understanding the properties of nature, and Silicon Valley’s prolific inventor Mr. Mir Imran, who told him long before he needed to raise money, “don’t ever take investor’s money, unless you can treat it as your own”.  Dwivedi said he carried all those valuable lessons in his professional journey.

After his education where he worked on tablets, Dwivedi was invited to join Glaxo to work on inhalation products.  When he was concerned about making a jump from tablets to inhalation products, he was told “you know how to keep particles together, you would know how to keep them apart too”.  From Glaxo, he went on to Dura, where he worked on an electromechanical inhaler system, a complex 50 part device.  Then at Alkermes, Dwivedi put together a team which designed a device with a precessing capsule inside.  Each of these opportunities contributed to giving him a stronger grounding into fundamentals.

Eventually, his career journey brought him to Nektar Therapeutics.  Nektar was focusing on systemic conditions like diabetes, to be treated by inhalation delivery of drugs like insulin, and on anti-infective therapies for lungs.  Dwivedi and his colleagues were looking at delivering drugs uniformly into the lungs for diseases such as asthma and COPD, and especially make drug combinations with standard metered dose inhalers.  Global COPD or chronic obstructive pulmonary disease and asthma market is projected to reach $47 billion, by 2017.  These were compelling clinical problems that Dwivedi and his colleagues were trying to solve, but Nektar chose to stay focused on other opportunities.  Eventually, Dwivedi and his co-founder Adrian Smith formed Pearl Therapeutics and spun it out as a separate company.

Image via CrunchBase

Pearl Therapeutics has combined two bronchodilator drugs in one inhaler, and put it into Phase III.  Pearl has now demonstrated that it can also combine three drugs, to treat the fatal lung condition, COPD.  These drugs typically do not mix well together in a common inhaler and that leads to less than ideal distribution of the drugs in the lungs.  Pearl’s secret is its proprietary technology.  Dwivedi said the lessons he learned earlier in life regarding staying true to fundamentals and to not fight nature, were most useful in navigating these challenges.  The nature of particles is to coalesce, and therefore it’s very difficult to keep them separate for aerosolization purposes.  This can be achieved easily with liquid propellant suspensions, such as those in commonly used metered dose inhalers.  Pearl creates these suspensions with a proprietary technology utilizing specially engineered phospholipid porous particles.  When these inhalers are actuated then the porous particles go back from liquid to vapor, facilitating consistent and uniform inhalation delivery. The porous particles traverse the back of the throat easily and spread aerosols throughout the lung.  Astra Zeneca saw the tremendous promise in this technology, the product progression achieved by Pearl, the value of the product pipeline at Pearl, and the Pearl team assembled by Dwivedi and his co-founder, Smith.  AZ forked over $1 B+ and acquired Pearl, with a promise to enable Pearl to continue to operate independently.  This was very interesting talk and generated a great deal of discussion.

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.

EPPIC 2013 Annual Life Science Conference Highlights

EPPIC 2013 Annual Life Science Conference Highlights

The annual EPPIC Life Science Conference (www.eppicglobal.org) takes place on the day before the start of the J. P. Morgan Healthcare Conference, each year, and offers a forum for information, networking, and mentoring. This year, the conference took place at the Westin SF Airport Hotel on January 6, 2013 and was jointly chaired by Ramani Aiyer, EPPIC President & Principal at Shasta Bio Ventures and Geetha Rao, CEO of Springborne Life Sciences and VP of Corporate Development at Triple Ring Technologies. The Gold sponsors of the event were Global Industry Analysts http://www.strategyr.com, Camargo http://www.camargoblog.com, Merchant & Gould http://www.merchantgould.com, Morrison Foerster http://www.mofo.com, Vivo http://www.vivoventures.com, and WSGR http://www.wsgr.com. Below are the highlights from the conference.

Plenary Talk – Carlos Olguin

The conference opened with plenary remarks by Carlos Olguin, Head of Bio/Nanotechnology/Programmable Matter Group at Autodesk and was moderated by John Cumbers, Head of Synthetic Biology & Resource Utilization in Space at NASA Ames. If you are wondering what has Autodesk to do with life sciences, the talk was an eye opener about interesting things happening on the boundary between bio-nano technology with 3D visualization, simulation, and design. Autodesk professionals bring design and engineering knowledge and look at ways to program matter, through scan, modify, and print pattern, said Olguin. Scan in the case of molecular biology is akin to sequencing DNA, modify refers to modeling, simulation, and optimization and print alludes to synthesizing the DNA. Looking beyond molecular biology, design and engineering in manufacturing as a whole may shift from being top down to self assembled, from being inert to being alive, from being generic to personalized, and from being product oriented to being service oriented, said Olguin. The talk was a clear indication that interesting, cutting edge stuff is happening on the boundaries between disciplines and that is where we will see some breakthrough advances take place in the years to come.

Personalized Medicine Panel

Personalized Medicine Panel was moderated by Sara Kenkare-Mitra, SVP of Development Sciences at Genentech. Brian Naughton, Founding Scientist, 23 and Me, Eric Sasso, VP of Medical and Scientific Affairs at Crescendo Bioscience, and Deborah Kilpatrick, Chief Commercial Officer at CardioDx discussed the implications of efforts in personalized medicine for chronic diseases.

Crescendo Bioscience is a molecular diagnostics laboratory focused on rheumatology. Currently,  assessment of rheumatoid arthritis disease activity depends on subjective clinical indicators and is a time consuming process. Crescendo is developing quantitative, objective, biology-based tests intended to provide rheumatologists with deeper clinical insights, said Naughton. The blood test, commercially available since  2010, integrates the concentrations of 12 serum proteins associated with rheumatoid arthritis disease activity into a single objective score to help physicians make more informed treatment decisions.

CardioDx offers Corus CAD blood test that can quickly and safely identify symptomatic patients unlikely to have obstructive coronary artery disease (CAD). However, getting physicians to think differently about using these tests versus imaging based tools, is a challenge, and “we are asking them to trust biology and not just an image”, said Kilpatrick. Currently, 75% of its commercial use is by primary care physicians and about 25% by cardiologists. Cardio Dx works the referral channel from an advocacy standpoint. An additional benefit observed by CardioDx is that after the test, the compliance to statins among patients is dramatically improved. Thus the test leads to a behavior change that would not otherwise occur.

23 and Me has an exciting story and it will again be covered in greater detail in my JPM post. The exciting development is in dramatic reduction of cost. Naughton shared that now it is within the reach of everyone and for $99, any consumer can get all sorts of information about their genetics, including increased risks for diseases like AMD, Alzheimer’s and others The panelists discussed their strategies for broadening the use of their tests with partnership opportunities and for other indications. Hearing about advances in personalized medicine, beyond oncology, was exciting.

Panel on Repurposing of Drugs

Mahendra Shah, partner with Vivo Ventures, moderated the panel that focused on repurposing of drugs. While new drugs cost upwards of $1.1B to develop and bring to market, and take over 10 years, once approved, there is no guarantee that they would be commercially successful, said Shah. The knowledge accumulated, can be analyzed to see the effectiveness of old drug for new usage. NIH is also planning to make an extensive database of old drugs available, to enable scientists to look for new indications.

Ken Phelps, CEO of Camargo Pharmaceutical Services, talked about the regulatory pathway in repurposing of drugs. Camargo specializes in FDA’s 505(b)(2) drug approval process. In 2011, more 505(b)(2) NDAs were approved than 505(b)(1) compounds, said Phelps. Sharing the history of 505(b)(2), Phelps emphasized that currently over 3000 drugs are still on the market that were introduced prior to 1962, when they were not required to be shown to be efficacious, and they are for taking. There are also compounds with new chemical entities and new indications that are available. He observed that it is important to identify compelling commercial differentiation and then take the knowledge available in the public domain, think outside the box, and use it effectively.

Warren Cooper, President at Coalescence was CEO of Prism Pharmaceuticals, a venture backed company that he led from inception to sale of the company to Baxter in 2011. According to Cooper, in repurposing a drug, same criteria should be used as NCE; identification of well defined clinical need, market value, and willingness to adopt and obtain reimbursement, technical feasibility, ability to protect from generics for meaningful period of time, and positive financial justification. What is different from NCE is that compound has a history and sometimes that is helpful and sometimes a hindrance. The deeper the history, the higher the challenge it may cause in the regulatory pathway of repurposing. Cooper discussed the example of injectable amiodarone that was originally developed for use in cardiac arrhythmias and was transferred at Prism Phrama, into NEXTERONE, from IV to ready to use pre-mixed product. The regulatory challenges required rewriting of the label and negotiations with FDA on labeling.

Jaisim Shah, Board Director at Igdrasol, discussed the key drivers for drug repurposing that include, reduction in cycle time, lower cost, better success rate, and quicker ROI for investors. Drug repurposing sales are up, to $20 B this year, said Shah. In 2012, as Chief Business Officer of Elevation Pharma, Shah led the sale of the company to Sunovian for upto $430 million, contingency based deal. The company took glycoyrrolate, a generic drug, approved decades ago by the FDA, and reformulated the compound for use with a nebulizer for chronic obstructive pulmonary disease (COPD), which includes emphysema and chronic bronchitis. It is found to be a well tolerated, tasteless, and stable formulation.

Panel on Technology Innovations that Shorten Therapeutic Product Development Life-Cycles

Moderated by Mohan Srinivasan, Director at Bristol-Myers Squibb, the panel discussed diverse and new technology adaptations aimed at compressing lifecycle and boosting success rates for drug development. Currently, for each new therapeutic, the product life-cycle ranges between 9 to 15 years and costs over a billion dollars. Can this be significantly shortened by adapting technology advancements in other sectors?

Chunlin Wang is Senior Scientist & Director of Informatics at Stanford Genome Technology Center. In last several years, Wang has been analyzing sequencing data generated by 454 FLX, I1-lumina GAIIx, HiSeq, MiSq, Ion Torrent and Pacific Bioscience platforms for various projects including drug-resistant mutation detection, whole genome sequencing, immune repertoire sequencing, high resolution high-throughput HLA genotyping and new virus discoveries. Using novel sequencing approaches to speed up drug development is one of the best ways to reduce costs, said Wang.

Scott Turner is EVP of R&D at Kinemed Inc. focused on the field of stable isotope research. Turner is leading the research into novel in vivo applications of stable isotopes in diseases. With its patented isotopic tracer technology, Kinemed enables its clients to track key chemical pathways within living systems and look at bio-chemical processes that are targets of drugs. Focusing on study in intact organism provides integrated systems biology information underlying the root causes of disease, said Turner.

Nikesh Kotecha is CEO of Cytobank Inc., a company focused on providing scientific, informatics and software solutions for cytometry. Cytobank focuses on analyzing single cell technologies and has created Cytobank, a cloud-computing platform for flow cytometry data. The Cytobank project grew out of Kotecha’s focus in Dr. Garry Nolan’s lab at Stanford University in developing a diagnostic for juvenile myelomonocytic leukemia and on analysis methods for single cell assays. Thus they take an interdisciplinary approach to address biological and clinical problems to help companies bring together their laboratory bench work and informatics needs. Cytobank is taking this beyond flow cytometry to mass cytometry, for identifying biomarkers, useful in personalized medicine, said Kotecha.

Pradeep Fernandes, co-founder and President of Cellworks Group, was most recently VP and GM of Synthesis Solutions at Cadence Design Systems, following its very successful $120M acquisition of Get2Chip in 2003.  Currently, a great deal of data is being generated.  Software is a tool that can be leveraged to manage and visualize large data, extract trends, generate insightful data and eventually predict information from large data sets, said Fernandes.  CellWorks takes in vivo and in vitro data and clinical trends and tries to build dynamic models that simulate disease phenotypes in computers and can be used to perform mechanistic studies.  This information is used to predict effects of new therapy and gain new biological insights regarding toxicity and biological efficacy of drugs.  CellWorks has an internal pipeline of therapies and is in collaboration with Astra Zeneca, Genentech and other companies interested in driving selection of therapies.

Speed Pitch, Accent on Youth, Speed Networking

In Speed Pitch, early stage life sciences companies were given the stage for a quick 5-minute pitch about their idea, technology, or company. The companies presented to the entire audience and obtained quick feedback from the distinguished VC panel and gained exposure to showcase to various potential collaborators, partners, and investors. Selected presentations are highlighted on the EPPIC website and other promotional material.

The Youth Panel was An Eppic Annual Conference First. This panel gave an opportunity to budding high school and college age scientists to showcase their outstanding research and later ask questions of each other’s research and future goals etc. The purpose of this panel was to inspire excellence among young people but these exceptionally bright, charming youngsters inspired the audience and left everyone spell bound.

Finally the Speed Networking event gave an opportunity to meet conference attendees in quick three-minute introduction and card exchange rounds.

This was a great conference that provided wonderful networking opportunity and was followed by more relaxed networking and wine reception.