Posts Tagged neglected and rare diseases
Posted by Darshana V. Nadkarni, Ph.D. in Biotech - Medical Device - Life Science - Healthcare on February 24, 2014
EPPICon 2014 (www.eppicglobal.org) will address the important topic of developing therapeutics for rare and neglected diseases. So what are rare and neglected diseases? Rare diseases are diseases that affect a small percentage of the population, most of them are genetic, and may be present throughout a person’s life. In the United States, rare diseases are defined as those that affect fewer than 200,000 people. About 7,000 diseases have been designated as rare and as many as one in ten Americans may suffer from a rare disease. More rare diseases are discovered ongoingly. Despite the growing numbers of rare diseases, the overall numbers are small and therefore they had not been a focus of research and many do not have treatments available.
Neglected diseases have also not been a focus of great deal of research and many of these diseases also lack viable treatment options. These include tropical diseases like Tuberculosis and Malaria and affect over 1.4 B people, worldwide. Often these affect the most vulnerable populations in the developing world, who lack access to basic sanitation, healthcare, and clean water.
A panel moderated by Roopa Ramamoorthi will discuss how companies and researchers can engage and bring forward cures for these diseases. Rmamoorthi is an experienced scientist with extensive background in global health, drug development, biotechnology, bacteriology, and engineering. As an Associate Director for Partnering and Scientific Affairs, at BioVentures for Global Health, Ramamoorthi leads the efforts to match researchers with pharma and other contributors, with an aim to accelerate product development for neglected tropical diseases like TB and Malaria.
Panelists include David Swinney, CEO of iRND3 (Institute for Rare and Neglected Diseases Drug Discovery). Swinney has 20+ years of broad experience in preclinical drug discovery. He founded his current non profit, in 2010, with a mission to help discover new medicines for rare and neglected diseases. The equipment for its lab in Mountain View was donated by Roche, and three early stage drug discovery programs at iRND3 have focused on pediatric cancers and parasitic diseases.
Eric Easom is the VP of neglected diseases at Anacor Pharmaceuticals, and Vimal Srivastava, is VP or Product Development, at Ultragenyx Pharmaceuticals. Anacor is focused on discovering, developing and commercializing novel small molecule therapeutics derived from its novel boron chemistry platform. Ultragenyx is committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with an initial focus on serious and debilitating, metabolic genetic diseases. On the panel, they are likely to add the big pharma perspective regarding focusing on neglected and rare diseases.
Ponni Subbiah, MD has an extensive experience in global medical affairs and clinical development across multiple therapeutic areas in the pharmaceutical sector. Currently, in the role of Global Program Leader, Subbiah is leading drug development efforts, at PATH, a nonprofit organization, with a bold vision for improving health, worldwide. PATH seeks to blend the entrepreneurial side of the business and the scientific expertise of a research institution with on-the-ground experience of an international NGO.
Other interesting panels at EPPICon include, “Innovations in Clinical Development of Novel Agents” and “Point of Care Gold Rush – Hype versus Reality”. Besides keynotes and networking opportunity, the conference will also feature a Speed Pitch session where entrepreneurs of early stage companies in the life sciences, are invited to give a five minute pitch about their technology and receive a quick feedback from a distinguished panel of VCs.
EPPIC is a volunteer driven organization, with a mission to promote networking, entrepreneurship, and mentoring for life science professionals. All day EPPIC conference will be held at The Westin, San Francisco Airport, in Milbrae, CA, on March 29, 2014. For more details and to register for the conference, go to www.eppicglobal.org . Please note: Early bird registration is extended to March, 7. EPPICon has an excellent lineup of great speakers and panels. This is not a conference to miss for any life science industry professional. Hope to see you there.
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.