New Waves in Biotech – Chinese Bioscience Association Annual Conference Review

The Chinese Bioscience Association (CBA) is a non-profit organization, with a mission to promote education, networking, and community building for life science professionals.

At its recent annual conference in San Francisco Bay Area, an impressive array of speakers discussed new trends, promises, and challenges in the biotech industry.

English: A diagram illustrating the distinction between cancer stem cell targeted and conventional cancer therapies (Photo credit: Wikipedia)

In his keynote address, Dr. Frank McCormick, Professor at UCSF and founder of Onyx Pharmaceuticals, talked about targeted cancer therapies and some of the lessons learned from Onyx.  Almost 30% of all mutated cancers happen in oncoproteins like BCR-ABL and RAS.  In 1994, Onyx initiated a 5 year collaboration with Bayer that led to $25M study for drug development, based on RAS pathway.  Unfortunately, it did not lead to drug leads.  In hindsight, “we realized that it was too early to jump into the pathway based on our feeble understanding of RAS pathway”, said McCormick.  However, Onyx has had several successes, despite the fact that finding new targets has become increasingly difficult, said McCormick.  Sorafenib, a kinase inhibitor, was approved for treatment of renal and liver cancers.  Onyx also has had success in viral therapies.  In 2009, Onyx sued Bayer, its longtime partner, and won the lawsuit.  Removal of the “change of control” clause in the original collaboration agreement, in favor of Onyx, has resulted in Onyx going from a $10M company in 1994 to $10B company in 2014.

English: Listeria monocytogenes grown on Listeria Selective Agar (Photo credit: Wikipedia)

Dr. Stephen Issacs, Chairman and Founder of Aduro Biotech talked about the importance of embracing change to keep growing.   Issacs shared his story from 1978 where his career began in psoralen photochemistry and then teaching at UC Berkeley.  In 1991, he found Cerus Corporation, a biomedical products company commercializing the Intercept Blood Systems.  He raised over $650M and negotiated a $200M partnership with Baxter International and took the company public.  He then found Aduro BioTech.  Here is my previous article on his company Aduro Biotech http://bit.ly/JqDJ3K that uses a bacterial vector, in the form of genetically modified listeria, to stimulate the immune system to fight cancers and infectious diseases.  Issacs also talked about his volunteer work in Kasigau, Kenya through his family non-profit organization, a cause that is very dear to his heart and to which he is highly dedicated.

Kayser-Fleischer ring: copper deposition in Descemet’s membrane of the cornea. These rings can be either dark brown, golden, or reddish-green, are 1 to 3 mm wide, and appear at the corneal limbus. With rare exceptions, they are diagnostic of inherited hepatolenticular degeneration—Wilson’s disease. This 32-year-old patient complained of longstanding difficulty speaking. He also had a tremor. (Photo credit: Wikipedia)

Dr. Chandler Robinson shared his incredible story of how he went from being an undergraduate researcher to becoming a CEO of Tactic Pharmaceuticals, a privately held biotech, in Chicago. Tactic acquires and develops pre-clinical and clinical stage compounds.  Somewhere in between his strenuous MD program at Stanford and pursuing his MBA in UK, Robinson acquired an abandoned drug, Decupratetm, and after couple of years, published his research in Science.  In early 2013, Decupratetm got Orphan Drug Designation from European Commission for treatment of Wilson’s Disease.  Wilson’s Disease is characterized by its disturbed copper metabolism that leads to copper accumulation in the body, resulting in severe disability or death. Tactic Pharma has acquired 3 compounds to date.

Diseases and conditions where stem cell treatment is promising or emerging. (See Wikipedia:Stem cell#Treatments). Bone marrow transplantation is, as of 2009, the only established use of stem cells. Model: Mikael Häggström. To discuss image, please see Template talk:Häggström diagrams (Photo credit: Wikipedia)

At Stanford Cardiovascular Institute (SVI), Dr. Joseph Wu and his team work with iPS cells (induced pluripotent stem cells).   Wu is recipient of several awards and his clinical activities involve adult congenital heart disease and cardiovascular imaging.  According to Wu, iPS will be a game changing platform for drug discovery research.  Familial hypertrophic cardiomyopathy (HCM) is one of the most prevalent cardiovascular disease.  At some point, we should be able take blood and make iPS cells, differentiate them into cardiac cells or other cell types, and expose them to different drugs to find out what would be the ideal drug for that patient, said Wu.  Wu said, it is important to study different ethnicities; different ethnic groups metabolize drugs differently.  CVI has received $20M grant and Wu’s team is trying to create a bank of 1000 cell lines, for drug discovery research.  (iPSCs) seem to have exciting therapeutic implications in regenerative medicine, particularly for myocardial infarction and possibly for neurodegenerative diseases, diabetes and other disorders.  However, stem cell biology is as yet incompletely understood and  some of the challenges include immunogenicity, tumorigenicity and so on.  Drugs can be tested on surrogates of patients, before giving them to the patients, said Wu.

Inhaler (Photo credit: Wikipedia)

Dr. Moninder Hora, SVP at Nektar Therapeutics talked about Nektar’s pipeline and technologies.  Nektar’s technology is based on polymer conjugation platform, said Hora.  Nektar has a robust pipeline of novel therapeutics and Nektar also partners with top biopharmaceutical companies to bring new products to market.  Nektar’s pipeline of late stage molecules include Amaikacin, Ciprofloxacin, Fovista, BAX 858, and NKTR-102.  Amaikacin Inhale is delivered directly to lungs to treat gram-negative ventricular pneumonia.  NKTR-102 is first PEGylated small molecule, first in its class, to treat breast cancer.  It has passed safety and efficacy markers and is showing to significantly improve overall survival and endpoints will be announced next year.  Nektar is also getting positive results from some of its early stage molecules.  Recently Nektar presented positive preclinical data for NKTR-214 for immunotherapy treatment.  Nektar is developing pain management portfolio and recently got fast track designation for NKTR-181, a new oral opioid analgesic molecule for the treatment of moderate to severe chronic pain.  NKTR has filed for regulatory approval in EU for MOVENTIG, comprising of once only daily oral tablet, to treat opioid induced constipation.  Hora concluded saying that Nektar has very robust pipeline of highly promising molecules.

English: Example transdermal patches. On left is a ‘reservoir’ type, on the right a ‘matrix’ version. Both contain exactly the same level of the same active ingredient. (Photo credit: Wikipedia)

Dr. Zander Strange, VP of Bus Dev. and Corp. Dev. at Zosano Pharma shared about their products based on novel transdermal delivery technology.  Zosano’s rapid onset system allows drug delivery to occur, almost painlessly.  The drug is delivered close to the capillary bed, allowing it to quickly dissolve.  The short-wear-time patch consists of microneedles coated with Zosano’s proprietary formulation of an existing drug, attached to an adhesive patch.  Therapeutic effect is expected to occur within 30 minutes or less, in an easy, pain free administration.  Zosano products are in dry formulation, thus vastly improving product stability and long shelf life, in addition to allowing for easy travel and storage at room temperature.

CBA annual conference was packed with impressive lineup of speakers, interspersed with plenty of networking opportunities.  For additional information, please go to www.cbasf.org .      

PS – EPPIC Digital Health event http://bit.ly/1uwQ6af is today at 6 pm in Palo Alto. Please register ASAP at http://www.eppicglobal.org .

Lessons for Starting a Medical Device Company in Silicon Valley

Mr. Michael Allen, a serial entrepreneur, (previously in executive roles at Xlumena, Metrika, and Chemtrack), and currently CEO of Vascular Designs, a targeted drug delivery company, talked about “starting a medical device company in Silicon Valley”, at www.bio2devicegroup.org .

 

Financing is one of the major early challenges that entrepreneurs face.  While it begins with a good idea, they also need to obtain some seed financing, and they need a short 2 page summary.  Their business plan should be an outline that includes some financial projections, some data about market and market need, information about regulatory path and reimbursement issues.  Advocates, typically medical experts, are also needed.  With these pieces in place, they would be ready to go for institutional financing and begin to meet the investors.

 

The VC model has changed, said Allen.  Traditional VC model used to focus around a goal with very high end value, in the range of $250 M where 10’s of millions would be invested.  It was built around the idea of large management teams in the early phase and a ready sales force in anticipation of the launch, that often never happens.  Many VCs who adhered to this model, are out of business.  The current model is to focus on a goal to achieve a moderate exit.  Team is scaled in proportion to the need, sales force is maintained at small level until product and market are demonstrated.  It is assumed that delays are inevitable and cash is king.

 

Allen talked about his experience and learnings from leading ChemTrak from seed stage to IPO exit, with 6X return.  ChemTrak was a diagnostic company, with a single use diagnostic kit to measure Total and HDL cholesterol and other non-instrumented quantitative tests.  He also shared lessons in leading Metrika, another diagnostic company that was sold to Bayer, with 1.5X return plus ongoing royalty deal.  Metrika’s several products included A1C test, giving patients with diabetes on the spot feedback on their A1C number and e.p.t. pregnancy test.   They had some good patents, but mixing electronics, optics, and chemistry was very challenging, said Allen.  Allen’s next company Xlumena, is medical device company with products aimed to advance image guided endoscopic therapy.  These products allow minimally invasive alternatives for diseases of the organs that surround the GI tract.  Summarizing some of the learnings, Allen said, CEO is responsible for everything, no matter who makes decisions.

 

Allen’s current company, Vascular Designs offers a new treatment option for people suffering from life-threatening illnesses like cancer, through targeted drug delivery, as opposed to systemic approach.  Through transfemoral catheter, drugs can be delivered directly into the tumor cells and block the blood supply.  It allows for high concentration of drugs delivered, while at the same time, reduces systemic exposure, thus minimizing caustic side effects.  This procedure can be performed as an outpatient procedure, takes only about an hour, and nothing is permanently implanted.  For success of a venture, Allen stressed, it is very important to hire the right people.  He discussed hiring process he follows and also emphasized the importance of spending time and writing a good job description.  The talk was followed by Q&A.