Posts Tagged repurposing
Posted by Darshana V. Nadkarni, Ph.D. in Biotech - Medical Device - Life Science - Healthcare on February 5, 2014
Mahendra Shah, Partner with Vivo Ventures & Ravi Mistry, President of EPPICGLOBAL (www.eppicglobal.org) talked about how to take a biotech company from idea to launch, at www.bio2devicegroup.org event. Here is some of the comments made by Shah and Mistry.
Entrepreneurs starting a biotech company must start with a disease target, said Shah. First and foremost there should have a good understanding of the disease and at which stage in the disease progression is the biotech seeking to interfere. Second, one should have a good understanding of the market opportunity and about existing treatments. Entrepreneurs need to ask the hard question regarding “clinical meaningfulness”; and whether or not the possible treatment developed by the biotech will lead to significant enhancement in patient care. Payers will not pay for little improvements, said Shah. Companies need to identify gaps in the treatment, and find a niche market. Patients need to be identified for who the prescribed treatments do not work well and that could be a niche market for entrepreneurs, said Shah. Shah said that during his career, he found such niche markets through repurposing, taking old drugs and finding new indications. He also cautioned about the importance of ensuring secure Intellectual Property to protect the proposed solution. With currently available internet tools and database resources, that is not hard to do. Even speed browsing on Google can give you a quick idea of who owns the IP and if there are ways to get around it, said Shah. Biotechs based on developing Orphan drugs are a great opportunity for a new startup, because once approved, you cannot have competition for up to 7 years in the US, and up to 10 years in Europe.
In terms of drug development, one needs to also get clarity on development pathway to determine whether it will require 10 thousand patients or few hundred, since that can make a huge difference in the amount of financial capital that will be required to bring the product to market. Companies need to identify how quickly one can progress to phase 2 and bring it to a meaningful endpoint. Also it is important to get an understanding if the endpoint is subjective or objective. A subjective endpoint will have significant placebo effect and will require much higher number of patients, said Shah. In order to do successful fund raising, companies need to have approximate idea of time and costs of bringing the product opportunity to a meaningful endpoint. Companies also need to have clarity on the various product development milestones and inflection points. Finally, an experienced Team is extremely important to bring the idea to fruition. However in this, two members of the team are particularly important; the CEO and the Medical Director. Other members of the management team can be hired as consultants but these two team members of the team need to be identified early and brought on board and should be people one can trust and those who feel passionate about the technology and who buy into it.
A biotechnology company requires significant money for its operations, so it is important to make sure that the first time investors have deep pockets, very sane advice from Shah. He told the budding entrepreneurs to be careful and make sure they have synergy with the investors. “Some of the investors’ money is not good, it will give you ulcers”, warned Shah, since money can have many strings attached to its return on investment. Referring to reimbursement, Shah emphasized that the payer is very important and entrepreneurs should do a quick survey of physicians and Key Opinion and Thought Leaders and get a clear idea about existing and current modes of treatment and who will be paying for the new product opportunity.
Mistry shared some statistics on activity in the life science space. 2013 has been a banner year for life science IPOs. Out of a total of 82 total IPOs in 2013, 46 were in the life science industry. Life science industry also enjoyed a substantial investment of dollars. The increase was certainly more significant in biotechnology, while medical device industry actually saw fewer dollars invested. Mistry talked about how to prepare for an M&A exit and provided some advice to entrepreneurs to keep diligent documentation with respect to company’s IP. He also commented that when it comes to negotiation, there is no “one size fits all” and best deals can be made if entrepreneurs remain can remain flexible throughout the process.
At the start of the talk Mistry put in a plug for EPPICGlobal and their upcoming annual conference, on March 29, 2014. The conference has an exciting lineup of speakers and panels addressing neglected and rare diseases, point of care diagnostics, and innovations in clinical development of novel agents. The talk ended with Shah sharing information on exciting speed pitch session, at the EPPIC conference, where entrepreneurs will have an opportunity to pitch their company to a panel of VCs and receive real-time feedback.
I would encourage my readers to attend the EPPICon 2014 conference, and avail of the opportunity to listen to various industry renowned speakers. To register for the conference and/or register for the speed pitch, please go to www.eppicglobal.org.
Posted by Darshana V. Nadkarni, Ph.D. in Biotech - Medical Device - Life Science - Healthcare on January 13, 2013
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.