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Developing New Products

Overview: California accounts for a fifth of the biomedical pipeline in the U.S.

CaliforniaRepresenting 21 percent of the total biomedical pipeline in the U.S., California has more than 1,460 entities in the pipeline from discovery to marketed products. The total biomedical pipeline in the U.S. is 6,904 entities.

CaliforniaIn 2012, California companies were responsible for nine of the FDA’s 39 approved new molecular entities (NME), or about 23 percent. In total, 63 products were approved, 208 were marketed and 28 were in registration.

Treatment Area #
Infectious 9
Cancer 7
Dermatology 5
Musculoskeletal 4
Ophthalmic 4
Cardiovascular 3
Endocrine / Metabolic 3
Neurology 3
Genitourinary 2
Other 2
Autoimmune 1
Gastrointestinal 1

A NCE or NME is a drug that is without precedent among regulated and approved drug products and is an indication of innovation in the development of new drugs and therapeutic biological products.

The top four disease areas of drug development for the California life science industry are cancer, neurology, infectious disease and endocrine and metabolic disorders, followed by autoimmune diseases and disorders and cardiovascular disease. More than 70 percent of the drug discovery and development efforts are in these six areas and correlates to the prevalence of diseases and disorders affecting patients in the U.S.

The top four diseases that affected Americans in 2010 are heart disease, obesity, cancer and infectious disease.4 More than 80 different types of autoimmune disorders collectively represent the most prevalent diseases in the developed world. A focus on neurology is essential as the population of the U.S. and the world ages. In 2012, the NIH spent an estimated $5.8 billion to support research in brain disorders, neurodegeneration and Alzheimer’s disease. Cancer research was just more than $5.4 billion. Read more on NIH Funding

In 2012, two medical products were approved that represented distinct examples of innovation in the industry. The first is Kalydeco, the breakthrough drug for cystic fibrosis created by a forward-thinking collaboration between Aurora, which was acquired by Vertex, and the Cystic Fibrosis Foundation. Read More. The second is the 510(k) clearance of an ingestible digital sensor from Proteus Digital Health. The ingestible sensor and feedback system provides a patient’s physiologic response to a drug and actual use of the drug. This information is designed to enable physicians to make more informed prescribing decisions and enable patients to sustain positive health habits. In this case, the FDA had to create an entirely new regulatory class based upon Proteus’s invention.

Proteus Digital Health“Our goal at Proteus is to enable consumers to realize much more therapeutic value from the drugs that they are already using. What many studies show is that prescribing can be based on little evidence and patients often lack the information and motivation required to follow through on a doctor’s orders. These challenges result in hundreds of billions of dollars in excess costs and a lot more sickness than if medicines were prescribed and used accurately.”

Andrew Thompson, Co-Founder and CEO, Proteus Digital Health

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Disease and translation focused nonprofits drive new treatments to clinic and patients

Disease and translation focused non-profit organizations that have taken an active role in therapeutic research, discovery and development made important breakthroughs in 2012. Also known as “venture philanthropy,” they function as organizing centers, bringing together academic and clinical research, biotech companies and patients around a particular disease.

One of the most important roles they play is working with all stakeholders to create and validate a robust preclinical path, including developing and analyzing potential biomarkers for clinical studies. Their efforts reduce the barriers for a company to try to develop new therapies for diseases that have either historically been difficult for drug discovery and development or have not garnered enough attention from the industry as a whole.

The Cystic Fibrosis Foundation’s (CFF) and CFF Therapeutics (CFFT) support and collaboration with Vertex Pharmaceuticals’ research facility in San Diego had a major win this year with the FDA approval of Kalydeco, the first drug to treat the underlying cause of cystic fibrosis. The CFF not only provided significant funding with a $75 million investment, it also provided key scientific and clinical support throughout the development process. The CFF has a long-running history. Established in 1955, CFF-supported scientists discovered the defective gene that causes cystic fibrosis. Today, CFF is supporting more than 30 drugs in development or use by patients, including the breakthrough Kalydeco.

aTyr“The collaboration is exemplary because it started at the concept stage. CFF/CFFT invested in an idea. The opportunity for a collaboration like this to happen again will depend on someone taking a risk on concept.”

Melissa Ashlock, M.D., (Former) Vice President of Drug Discovery, CFF, (Current) Vice President, External Scientific Alliances and Human Genetics, aTyr Pharma

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In 2002, Scott Johnson, who was diagnosed with multiple sclerosis in 1976, formed the Santa Clara, Calif.-based Myelin Repair Foundation (MRF) to help overcome the barriers to translating new research discoveries into treatments or even cures for patients. To date, the MRF has raised $45 million to support research efforts that share data and collaborate among MRF supported scientists, rather than scientists competing with each other to advance new treatments. This year, MRF-supported research has entered two clinical trials, one at the Cleveland Clinic and another at the University of Hamburg in Germany to examine potential multiple sclerosis therapeutics.

Myelin Repair Foundation“Our goal is to ensure a clear pathway into clinical trials to benefit patients waiting for new multiple sclerosis treatments. Through our collaborative research approach which greatly accelerates the entire therapeutics development process, we are well on way to achieve our goal to develop a new myelin repair drug to patients by 2019.”

Scott Johnson, CEO, President and Founder, Myelin Repair Foundation

The hands-on role of these foundations differs from traditional disease foundations, which tend to focus on promising projects and trials. The models of CFF and MRF are actually closer to biotech as they are active in building and shaping the capabilities required to achieve the translation of research advancements to the clinic. The further breakthrough CFF and MRF have achieved is creating collaborative communities, where all involved from scientists to physicians are all willing to share ideas and work together to go into the unknown and find solutions many had thought impossible.

“We have a community, a board and our scientists, that are willing to go into areas that there is a lot of unknown and a lot of risk. We have been able to create a collaborative community. This does not happen in all diseases. The ability to share ideas is the key to innovation.”

Robert J. Beall, Ph.D., President and CEO, Cystic Fibrosis Foundation

New biology is essential for better health care and medical products that fill unmet medical needs

New and emerging areas of biology as well as cross-pollination among industries are critical to making positive impacts on health care and filling unmet medical needs.

Regulus Therapeutics“The discovery of new biology is essential to foster increased innovation in the life science industry. Our industry needs to focus considerable scientific and financial resources to uncover innovative, new biology that can be used to treat diseases with high unmet medical need or treat diseases with new, more patient-friendly regimens to improve cure rates.”

Kleanthis Xanthopoulos, Ph.D., President and CEO, Regulus Therapeutics

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The deeper understanding of disease through “omics” technologies (genomics, proteomics, metabolomics, etc.) is driving personalized medicine, which holds the promise for not only providing the best health care to patients but also reducing health care costs from ineffective treatments. Furthermore, the entire field of orphan diseases requires new sciences and understandings to discover and develop new medicines.

aTyr Pharma“We need new biology to solve difficulty problems not solvable in a timely fashion by existing tools alone. New science can help you understand a way of looking at diseases, medicines, drug discovery. New biology is a bonus because it can give you a new approach to a certain single disease and open your eyes to understanding at other diseases as well.”

Melissa Ashlock, M.D., Vice President, External Scientific Alliances and Human Genetics, aTyr Pharma

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In medical practice today, we are seeing the fruits of years of basic research into the molecular and cellular characterization of cancer to deliver personalized medicine. The research understandings of cancer biomarkers have brought major advancements in oncology, enabling the development of new chemotherapies that are targeted to specific mutations in cancer cells. These targeted therapies can be less toxic and more effective than conventional chemotherapies that nonspecifically affect dividing cells.

Aragon“Drug development will be mostly driven by molecular drivers of cancer instead of targeting DNA in general with cytotoxic chemotherapy. Combination therapy will be used to target mechanisms of tumor resistance. The ultimate goal is that cancer patients will receive more personalized and efficacious cancer treatment with lower toxicity and better quality of life.”

Rich Heyman, Ph.D., President and CEO, Aragon Pharmaceuticals

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In addition, companies such as Genomic Health and Epic Sciences, have advanced technologies for new diagnostics to enable doctors to tailor therapies to a patient’s specific cancer.

“Blood tests that monitor and characterize circulating tumor cells (CTCs) can be used to diagnose cancer, monitor the course of treatment, and to match each patient to the drug most likely to work for them. Certain drugs may initially work for a patient but then they stop working because his or her tumor changes and is different compared to when the patient had a first biopsy to diagnose the tumor. Counting and characterizing CTCs can provide the real time status of disease and can be used to direct the right drug to the right patient at the right time.”

David Nelson, Ph.D., President and CEO, Epic Sciences

From Proteus’ ingestible sensors to Genomic Health’s cancer diagnostics, the industry is just beginning to see the products in what will become the wide-ranging field of personalized or “precision” medicine. Advances in the “omics” fields as well as cross-pollination with the technology industry will create improvements in the diagnosis and treatment of disease.

Illumina“Genomics is beginning to inform diagnosis and treatment for patients. This is particularly true for cancer, where whole genome sequencing is being used to identify variants that can drive disease progression, as well as to select therapies based on these findings. As we become better able to leverage genomic data in routine patient care, this will transform diagnostics as we know it today.”

Jay Flatley, President and CEO, Illumina

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Beyond science, life science companies are constantly innovating and charting new paths

The hurdles and risks to developing new therapeutics, diagnostics and medical devices are many. Not only do life sciences entrepreneurs have the inherent hurdle of ensuring the science “works,” they also face a more difficult landscape for financing, regulatory approval and reimbursement. Whereas companies once thought of innovation simply as making a better drug, device or diagnostic, nowadays investors, regulators and payers are demanding evidence of value within the larger context of the health care system.

Gilead“We face a number of challenges developing new and improved therapies for serious unmet medical needs, including diseases that are impacting entire communities here in California and around the world. Innovation can be found in all aspects of our business – in creating a new drug, improving a manufacturing process, simplifying how treatment is delivered.”

John Martin, Ph.D., Chairman and CEO, Gilead Sciences

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Medivation“Infectious diseases or cancer often try to get around established therapeutic strategies to inhibit their growth by finding alternative pathways to escape eradication. So just as viruses or cancers innovate to find novel escape pathways, the life sciences industry has to innovate to keep up with or get ahead of these diseases.”

David Hung, M.D., Co-Founder and CEO, Medivation

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Veracyte“The industry is fueled by innovation – from new technologies to innovative clinical trials to the emergence of drugs and diagnostics that offer the promise of better patient care at reduced costs. Our industry’s very existence depends on our collective ability to rapidly and continuously innovate in all areas of business.”

Bonnie Anderson, Co-Founder and CEO, Veracyte

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