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Bayer East Coast Innovation Center Head reveals early strategic advantage is something pharma wants from biotech startups

chandra-ramanathan-biopharm-americaPartnering Insight recently had the pleasure of speaking with Dr. Chandra Ramanathan, Head of the East Coast Innovation Center at Bayer, whose mission is building an innovative product portfolio with key partners including academia and members of the local innovation ecosystem of biotech startups. Bayer is well known for their ability to tap into the innovation mindset. Dr. Ramanathan will be participating on the panel “What’s the next big thing on pharma’s wish list?” at BioPharm America™ taking place in Boston, MA, September 26–27. We spoke to Dr. Ramanathan to hear more about his work at the Center and to hear his strategy for tapping into innovation.

The East Coast Innovation Center at Bayer is part of broader US innovation strategy that includes the Bayer West Coast Innovation Center in San Francisco. The launch of the East Coast Innovation Center occurred in March 2016.

“Our innovation strategy for identifying and working with the most innovative startups to forge partnerships with Bayer is based on four core pillars,” said Ramanathan, described as: (more…)

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Biotech Startups: Land and conduct a successful investor meeting

biotech-startups-pitch-tips

Biotech startup companies face many challenges, but one of the most common is landing and conducting meetings with potential investors.

The following are pieces of advice from investors on what they want and how they want you to approach them. The advice comes from Sinclair Dunlop of Epidarex Capital; Carolyn Green of Pfizer Worldwide R&D, ERDI; Sam Hall of Appletree Partners; Rick Jones of Broadview Ventures; Dennis J. Purcell of Aisling Capital; Jeremy Springhorn of Flagship Ventures; and David Steinberg of PureTech from a previous edition of BioPharm America, which will be taking place again this year in Boston, September 26–27. The 2017 edition features expanded offerings and special pricing for biotech startup companies. (more…)

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The Advocate of Rare Disease Advocates: Remembering Henri Termeer

We were saddened to come to work Monday morning to discover that innovator, industry giant and Genzyme founder Henri Termeer had died. We had the pleasure of working with Termeer over the years as a speaker and sponsor of life science partnering activities. He is best known for creating Genzyme and turning that company from a small startup into a leader in bringing transformative therapies for rare diseases to patients.

Part of Termeer’s magic was the real connection he felt to his work. Speaking at Biotech Showcase™ as part of RARE In The Square event in San Francisco in January, Termeer spoke about the knock on the door one day in 2011 that changed his life. That knock was from Daniel de Boer, a 29-year-old engineer. Thanks to an introduction from a friend, de Boer met with Termeer and related to him that his young son had been diagnosed with cystic fibrosis, and Termeer committed to finding a way to help him and other patients with the disease. That meeting resulted in the formation of ProQR Therapeutics, a biotech company headquartered in Leiden, NL that is working to develop drugs to treat severe genetic disorders.   (more…)

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DIGITAL-MEDICINE-WHITEPAPER

How will digital technologies impact disease management and healthcare over the next decade?

Find out in the just-released whitepaper, “The Digital Medicine Crystal Ball: Unlocking the Future of Real-Time, Precise, Effective, Healthcare.”

The last five years have seen an unprecedented eruption in technological and health advances.

These new technologies and products—many undergoing rigorous clinical validation—will have significant direct impacts on diagnosing, preventing, monitoring or treating a disease, condition or syndrome, which in turn will transform disease management and alter business models across industries. (more…)

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Where to start: How to gain a foothold in the China biopharmaceutical market

The China pharmaceutical market has come of age. With record breaking investments in innovative companies, support from the CFDA, and new product approvals pending, China has entered a new age as a global competitor in new drug development and commercialization. China remains the second largest global economy, with USD 5.3 billion+ in VC investment in the life sciences sector in 2016. There are more market entry opportunities than ever before. (more…)

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Tissue regeneration: A vital area within regenerative medicine

Guest post by Patricia Reilly, M.S., Head of Intelligence Alliances and Unification, Pharma Intelligence and Nancy Dvorin, Executive Editor, In Vivo


Cell and gene therapies often get the lion’s share of news coverage in regenerative medicine as these advanced treatments make giant strides each year towards curing devastating diseases. Tissue regeneration and tissue engineering are also a vibrant and overlapping portion of the regenerative medicine sector.  Companies in this space have developed products and platforms to replace cells lost to injury, often by enhancing wound angiogenesis, blood vessel development and matrix restoration. We analyzed deal trends over the past five years, from 2012 to 2016, to see how tissue regeneration companies are faring.
(more…)

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Bruce Levine is a man you can thank for your future cell therapy

To call Bruce Levine, PhD, an innovator in the cell and gene therapy space is a gross understatement. He is known for his groundbreaking work in the development and use of synthetic immune cells (or CAR T cells) to attack advanced cancer cells. Insight’s Erin Righetti interviewed Levine to talk about his research and outlook on cell and gene therapies, and the importance of partnerships in progressing the field.

A sensational discovery

Twenty years ago, Levine, with Carl H. June, first began collaborations with a small biotech company, Cell Genesys, on a technique to genetically alter T cells to create synthetic immune cells, known as chimeric antigen receptor T (or CAR T) cells. With June, Levine had created a groundbreaking microbead technology to act as artificial substitute for the dendritic cells that give T cells the signal to attack cancer cells. This technology was the basis for the Cell Genesys collaboration and is now the primary method used by investigators to grow activated T cells.

“In the very early CAR T cell trials by several groups, there were some lab indicators that the cells persisted, that they were there, but clinical responses were minimal at best,” said Levine.  “We used microbead technology to more efficiently grow T cells and later, a disabled form of the HIV virus to deliver the CAR gene to T cells. Express of the CAR of the surface of T cells redirects and trains them to target the CD19 protein on malignant B cells and kills those cells.”

At critical junctures in the Penn team’s research, they ran out of funding. Research is expensive. Cell and gene clinical trial research even more so. “Over the last 25 years that we have been working in the field, adoptive immunotherapies have had a lot of peaks and valleys,” said Levine. “In the 1990s and early 2000s, it was seen as something boutique of ‘isn’t that quaint,’ and ‘interesting, but it’s never going to work.’ This idea of redirecting T Cells or immune cells was groundbreaking. NIH grant reviewers saw it as a totally different paradigm than they were used to, and many comments were not kind.

“Then in 2004, we applied for a grant from the Alliance for Cancer Gene Therapy (ACGT). This foundation was started by Edward and Barbara Netter, who endowed my Chair at the University of Pennsylvania,” said Levine. “Edward, who passed away six years ago from pancreatic cancer, was a graduate of Penn. They started the foundation in 2001 because their daughter-in-law, Kimberly Lawrence-Netter, was diagnosed with breast cancer and passed away from it. Edward was a polymath, interested in everything. After his daughter-in-law passed away, he heard about a seminar organized by Savio Woo, PhD, at Mount Sinai School of Medicine on gene therapy. It was then that Mr. Netter determined he would direct their Foundation’s efforts to gene therapy.”

In a 2010 trial in patients with advanced blood cancers who were infused with their own CAR T cells, pounds of leukemia then melted away over several weeks, according to Levine. “We were out of money, we had striking results and we were asking ourselves ‘What do we do? If we publish we can get more grants.’” At that point in 2011, with no funding left to continue the research, June and Levine and their Penn team published their results, sparking a media sensation as to the potential of CAR T cell therapies.

Without the renewed interest of and resulting involvement by partners, progress on these groundbreaking discoveries may have never seen the light of day, so to speak. Partnerships have played an important role in progressing this research and have boosted the progress of Levine’s recent work.

When they  published in 2011, we had no idea of the press coverage it would generate, appearing in over 800 media outlets, resulting in 3,000-4,000 inquiries from people interested in the clinical trials. Penn had to build a website to take the inquiries that is still in use. The 2011 publications led to interest from industry in licensing the technology.

“Ten to 12 companies of various sizes, all the way up to Novartis, contacted us wanting to license and develop the technology,” said Levine. “We then signed with Novartis in 2012. The partnership between the University of Pennsylvania and Novartis allowed the development of clinical trials of CART T cells in leukemia and other blood cancers in adults, and in children as well. The ripple effect of the Penn-Novartis alliance was a ‘licensing and investment frenzy’ across the US and now around the world among various biotech, research universities and hospitals.”

Jump ahead to 2017 and two companies have submitted applications this year asking the FDA to approve CAR T cells for Breakthrough Therapy designation in the treatment of cancer. Novartis has already submitted for pediatric acute lymphoid leukemia (ALL) which has been accepted by the FDA. Later in the year, Novartis will submit for lymphoma. Kite Pharma has also initially submitted for treatment of relapsed or refractory aggressive non-Hodgkin Lymphoma (NHL).

Big pharma: Learning to speak the language

To put the Novartis collaboration in perspective, everyone in the field thought big pharma would never be interested in cell and gene therapies, it broke their business model from multi, continuous dosing to a one time or couple time infusion where immune cells are detected years after infusion. The collaboration spurred attention in the field as to what other groups were doing, followed by the work at Juno, Kite Pharma and other companies picking up steam. Said Levine, at last count there were three dozen companies in the field of engineering immune cells to redirect against cancer, including T cells and NK cells.

“We have had other industry partners in the time since the early collaboration with Cell Genesys in the 1990s,” said Levine. “During my postdoctoral fellowship with Carl June, I had developed the magnetic bead technology to grow T cells, and Cell Genesys contacted us about research they were doing on chimeric antigen receptor T cells. That was the beginning of that relationship,” said Levine. “Carl June and I were still at Naval Medical Research Institute at that time. When we moved to the University of Pennsylvania  in 1999, inquiries followed from other companies about technology they wanted to get into clinical trials, including Sangamo BioSciences, to create HIV resistant cells using zinc finger nucleases in the first gene editing clinical trial in humans. We have worked with other companies including Virxsys, Adaptimmune, and Takara taking the technology they had developed into clinical trials, serving as R&D in clinical trial development and execution.”

The role of partnerships in the development of cell and gene therapies and cures came up frequently for Levine.

“Over the past 20 years and even more recently, in the last five to ten years, there has been an evolution in how translational research and research in general works,” said Levine. “Declining NIH funding, and signs of future cuts means academic investigators must rely more on philanthropy and industrial partnerships to conduct research. We have seen that at Penn, the balance between academic-initiated hypothesis research that is NIH grant funded and industrial targeted research; academia and industry need to learn to speak one another’s language to collaborate.”

Collaboration necessitates the need for creation of industry-wide standards for the commercialization of cell and gene therapies.

“Not only is it a new field but it’s a new field in complex biology,” said Levine. “Consider the traditional development of drugs and characterization of drugs, where small molecules are characterized at-will and made in big batches and delivered to patients. In cell therapy, you have a very complex entity. How do you characterize that in terms of potency and stability? That cell, especially autologous cells, varies greatly. How can you have a standard for that cell when they vary in so many parameters in different patients? Development of standards is important and difficult. We have had conversations on this with the Alliance for Regenerative Medicine (ARM), American Society of Gene & Cell Therapy (ASGCT), National Institute of Standards and Technology (NIST). One way to start is to look at instrumentation and determine reference standards, methods for counting, and methods for analysis. It’s very complex.

“The cell I develop is not likely to be the same as the cell another investigator develops if any of the many elements of sourcing and manufacture are even slightly different,” Levine said.

Making the case for early collaboration

For cell and gene researchers, there is substantial benefit in collaborating early, but how can they attract the attention and support of partners at an early, high-risk stage?

“That’s a really good question,” said Levine. “I wish we knew all the answers. It’s simply a different audience. For an NIH review, there’s a defined format and investigators are well versed in how to put their proposal together.” Even so, many times researchers don’t know who the panel reviewing is or how well their proposals will be received. There have been new developments in philanthropic research funding in the last 10 to 15 years, as well. “Philanthropies are now asking for return on their investment with regard to rights on their IP,” said Levine. “This throws another wrinkle into the midst. If a company wants to license, they have to consider that throwback to the licensing stake.”

So, how should cell and gene companies pitch? “You start with ‘how is your research getting out there?’ and is it being published?” said Levine. “You have to determine if the backbone behind the IP and knowhow is attractive to licensing, and then get assistance in packaging your pitch to outside companies.”

Another critical but basic parameter is whether or not the research has been published. As evidenced from previous trends, there is a bandwagon effect when a concept or certain terminology gains popularity. Just look at the hype in recent years that culminated around stem cells, then nanotech, to name a couple. A partnership proposal can benefit from using popular or trending terminology, not in a sensational way, but in an intelligent way.

“The problem with a hot term is it can be hijacked. For example, so called stem cell clinics that skirt FDA oversight in pitching to patients and charging them for every indication under the sun where there commonly isn’t a scientific rationale,” said Levine. “You want to get attention for what you’re doing but researchers and partners need to do due diligence on each other.”

Back in 2010, Levine, June and the Penn team conducted initial trials of CAR T cells that targeted CD19 in three patients with advanced cases of chronic lymphocytic leukemia (CLL). Levine admitted that having partners at that very early stage would have benefited his work.

“We had some funding through the Alliance for Cancer Gene Therapy and the Leukemia and Lymphoma Society that took us to a certain point at a certain speed. We hoped, and it turned out to be case, that we had something extraordinarily promising,” said Levine. “Had we had more funding earlier it would have developed more rapidly. We are excited and grateful for our alliance with Novartis for the opportunity to develop that.

The impact of patient advocacy

Cell and gene researchers are increasingly working with patients and patient advocacy organizations to reach a global community to advance their research and open doors at the drug policy level.

“The involvement of patient families and related groups is extraordinary,” said Levine.

Levine related a story of the first child treated with CAR T, Emily Whitehead, which was reported in the New York Times and other popular news, as well as being the subject of a 2012 documentary, “Fire With Fire” filmed by Ross Kauffman, an Oscar-winning director. Whitehead became a celebrity patient, appearing on Stand Up 2 Cancer and featured at the April 2016 opening of Napster and Facebook billionaire Sean Parker’s Parker Institute for Cancer Immunotherapy, as well as meeting President Obama and Vice President Biden, among others.

“When the New York Times (NYT) story first appeared in 2012, the staff at NYT said to the Whitehead family ‘Beware of the tsunami.’ The Whiteheads were very quickly contacted by media outlets and other patients around the world,” said Levine. Shortly thereafter, they founded a philanthropic foundation, the Emily Whitehead Foundation – Activate the Cure, which has funded the work of June and Levine and collaborators at the Children’s Hospital of Philadelphia in CAR T cells.

“This is an example of a girl who had failed all available therapies, who did not have long to live, whose parents enrolled her in a trial, she became a complete responder, and they are all now paying it forward in so many tangible ways in support of other patients. They are heroes,” said Levine.

Many patients desire to meet scientists who develop their cells and conversely, the scientists get to know their patients. Levine and his group frequently work with patient groups, foundations and advocacy organizations such as ARM. One of Levine’s former patients is on the Lymphoma & Leukemia Society board advocating for future therapies. Advocacy has a strong impact on top level decision makers, such as the NIH-backed Cancer Moonshot program which involves external experts from industry and advocacy.  “When President Obama announced Cancer Moonshot, our labs were the first stop Vice President Biden made after the announcement,” said Levine. “He was here over two hours, and returned again just six weeks ago.”

Levine will be participating in Cell & Gene Exchange, a partnering forum focused on the regulatory, business, and patient advocate communities in cell and gene therapy sectors taking place May 22–23 in Washington, DC.

Partnering is the essential link to progress

“This is the first event of this kind [Cell & Gene exchange],” said Levine. “The idea is that this is no longer a small percolating field; it’s now evolved to the point where we have new considerations for therapies that are going to approved and commercialized. It is real.

“There has been a cascade effect and the number of companies that have started in the field has increased exponentially these last few years,” said Levine. “It is important to have a forum where experts in the field can hear from new companies. Producing CAR T cells require a complex and multi-step process, and all along the way you have ancillary reagents, technology, logistics, and so many pieces that go into it. The ability to partner is important to advance the field and promote understanding that this is a unique technology. Partnering is essential and required, it’s not optional.”

“I’ve been involved with ARM for a number of years, as we work toward scale out of CAR T cell manufacturing,” said Levine. “I am making an effort to free up more of my time to be locally, nationally and globally engaged in advancement of cell and gene therapies. For me, personally, the timing is right, and with the recent filings for Novartis and Kite, there will be even more awareness of this field, and we are looking to ARM to raise awareness in Washington for these new therapies with regard to policies and regulation.”

At the end of the day, Levine attributes his inspiration to the people who will benefit from future therapies and cures. “It’s the patients. No question about it,” said Levine. “As a PhD, the ability to do work that is not only interesting but has application to new therapies, that is the ultimate reward; to be able to meet patients who have been impacted by this work. Just to have the opportunity to have been mentored by Carl June and now to be part of a large team starting in research all the way to translation to clinical labs, to see that large team of dozens of experts coming together to get therapies to the clinic and to see the patients has been very rewarding. I think we all feel very fortunate to be in this space at this time, and we owe a great debt of gratitude to the patients whose families enroll in these trials without whom we would not be able to develop new future therapies.”

Meet Bruce Levine at Cell & Gene Exchange to hear more about exciting developments in the cell and gene sector, and meet with leaders from patient advocacy groups, industry, academia and government to explore partnership opportunities, exchange ideas, and network with the goal of advancing the development of potentially curative therapies for life-threatening diseases.

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Health tech innovators pitch their business ideas to expert panel at BIO-Europe Spring®

The buzz steadily grew as the Health Tech Competition got underway on the last day of BIO-Europe Spring® March 22 in Barcelona. As delegates filed into the competition room, the energy was palpable.

What new trends would this preselected group of competitors bring to the global health tech stage? Which seed company would be awarded first prize for its innovation?

Moderator Xavier Olba of Biocat, the organization that coordinates and promotes the healthcare and life sciences sector in Catalonia,  introduced the expert panel of judges who would determine the final outcome:

Ignasi Costas, Partner, Head of Innovation and Entrepreneurship, Head of International Relations, RCD

Jorge Juan Fernández, Head of Academics, Moebio

Frederic Llordachs, Co-Founder, Partner and Global Business Development Manager, Doctoralia Internet SL

Montserrat Vendrell, General Partner, Alta Life Sciences

People who know about the health tech industry most likely understand that the location of this competition made perfect sense. Barcelona is home to the Spanish Federation of Healthcare Technology Companies (FENIN), a multi-sector federation made up of manufacturing, import and distribution companies and  healthcare technology and product associations that supply the vast majority of Spanish healthcare institutions. The city is also home base for Moebio, a collaboration between Biocat, Stanford University, La Caixa and Barcelona Activa focused on developing health entrepreneurs in Barcelona.

In addition, Barcelona has been the scene for million-funding rounds in digital health companies such as Galgo Medical and ClinicPoint, in addition to interesting mergers and acquisitions operations such as the DocPlanner fusion with Catalan Doctoralia.

Worldwide, digital health companies earned revenues of USD 16 million during 2015 and 2016. This financial success has boosted the sector to 40 digital health companies, and 200 medical technology companies, of which 94 develop innovative medical devices.

Additional startup accelerators MWC, G4A, and Health U are fueling the international fire for innovation in eHealth, medical devices, wearables and other healthcare applied technologies.

Representatives and companies participating in the Health Tech Startup Competition included:

  1. Ray G. Butler, CEO/Software R&D Manager, Butler Scientifics Product5: AutoDiscovery is an intelligent automated exploratory data analysis software that helps unveiling complex relationships hidden in the data files of your scientific experiments and clinical studies. http://www.butlerscientifics.com
  2. Laszlo Bax, Co-Founder and CEO, Braingaze Braingaze, founded in 2013, develops next-generation Mind Tracking Solutions that predict and identify personal behavior for clinical and commercial applications. http://www.braingaze.com
  3. Miquel A Bru, Co-Founder, Made of Genes One genome. Endless possibilities. The first marketplace of applications based on your genome. A new dimension in personalization. https://madeofgenes.com
  4. Francesc Martinez, Marketing Manager, Airmony By TBIOM Airmony is an innovative medical device which helps you improve your sleep. It’s a portable and autonomous CPAP, which impulses air to provide assisted ventilation to treat Obstructive Sleep Apnea Syndrome (OSAS). http://www.yourairmony.com
  5. Dr. Markus Wilhelms, CEO, MOWOOT The non-drug, non-invasive and no side effects solution for chronic constipation. https://www.mowoot.com
  6. Ignasi Heras, CEO, Transplant Biomedicals Transplant Biomedicals is a European company based in Barcelona aimed at developing novel medical strategies to improve organ, tissue and cellular preservation to overall enhance patients’ wellness. https://www.transplantbiomed.com/
  7. Xavier Raurich Fabregas, COO, MJN Neuroserveis We are a science research and engineering team developing high-technology innovative devices to predict epileptic seizures. http://mjn.cat
  8. Jordina Arcal, Co-Founder, HealthApp HealthApp was founded to improve medical assistance and the patient recovery process. By developing gamified apps, HealthApp engages patients in therapy and connects with healthcare professionals. HealthApp provides real time data and analysis to adjust treatment, actions and decisions for better care.  http://bcnhealthapp.com
  9. Xavier Guillem, CEO, Adan Medical Innovation Digital Epinephrine Management: The concept was born from daily clinical interactions between physicians and patients, based on their discomfort with current management of epinephrine auto-injectors, and with a continued effort has become a reality. http://www.adanmi.com
  10. Josep M. Escuer/Nuria Noguera, Managing Director/R&D+i Director, Qrem We offer the first lab-in-a-box device to perform a natural cure for osteoarthritis (OA) reducing 75% of the pain for more than 1 year. http://www.qrem-regenerative.com

“The HealthTech Startup Competition brought together life science executives and digital health and medical technology innovators from all over the world, with the majority of startup companies representing the BioRegion of Catalonia,” said Dr. Albert Barberà, CEO of Biocat. “This life science segment has the opportunity for tremendous growth. I am sure the participants in the competition were deeply impressed with the BIO-Europe Spring attendees and enjoyed accessing such an international conference and coming face-to-face with delegates from all over the world.”

Transplant Biomedicals, Mowoot and Braingaze won first, second and third, respectively, in the competition. Transplant Biomedicals won EUR 5,000, four hours of mentoring from Alta Life Sciences and 10 hours of legal advice from RCD.

The Health Tech Competition was sponsored by Biocat, Catalonia Trade and Investment, Alta Life Sciences, and Rousaud Costas Duran (RCD) SLP.

Click here for more blog coverage of the activities held during BIO-Europe Spring.

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Report findings provide insight into technological innovations and trends in drug development

Knect365 Life Sciences has released a report offering unique insights into the awareness, perceptions and implementation of technological innovations in the drug development industry.

Based on a survey of their global database and 261 responses representing pharma, CROs and service providers split across North America, Europe and Asia, the report focuses on big data, cloud technology for EMRs, mHealth and patient-centricity.

The fascinating results provide a ‘state of the industry’ look at where these technologies are and where they are going, allowing professionals in the health sciences industry to better anticipate changes in their workplace.

The study found the primary benefit of big data to be increased efficiencies with the primary concern being cost of implementation. Big data practices are currently being utilized by nearly half of the industry, yet respondents in this survey indicate the quality of training received is only average (3.07 on a 5.0 scale).

Greater patient engagement and more successful patient recruitment are the top two benefits from implementation of patient-centric practices, yet cost is cited as the primary hindrance and/or concern regarding implementation. The most common ways to measure patient-centricity are patient engagement metrics and qualitative patient feedback.

Cloud technology for EMRs and mHealth technologies are still growing in their adoption by the industry, and it seems that data security concerns is the top obstacle to overcome. Respondents indicate that cloud technology for EMRs will have the greatest effect on increasing efficiencies while mHealth technologies allow real-time data acquisition and increased patient compliance.

Click here to download the report 

 

Innovative technologies and startup biotech companies are more important than ever to big pharma as their primary fuel source for drug and device pipelines. They are also a core focus of BioPharm America™, the leading US-based global innovation partnering event, September 26–27, 2017 in Boston. Learn more.

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Using Partnerships, Hua Medicine Brings Novel Diabetes Drug to Phase III

Li Chen, PhD, co-founded Hua Medicine (Shanghai) six years ago with Ge Li, PhD, founder of WuXi AppTec. The company soon in-licensed global rights to a GKA treatment for type 2 diabetes from Roche. Late last year, Hua announced positive results from the candidate’s proof-of-concept Phase II trial. The company also raised $50 million in additional capital during 2016 and has started US clinical development of the GKA candidate. Dr. Chen, who was previously CSO at Roche’s Shanghai R&D Center, talks about Hua Medicine and the contribution of the ChinaBio® Partnering Forum to China’s drug development ecosystem. The 2017 ChinaBio® Partnering Forum will be held May 31-June 1 in Zhuhai, China.

ChinaBio: Dr. Chen, Hua’s lead candidate is an oral GKA treatment for diabetes. It recently completed a Phase II trial in China. Can you tell us more about where you are with the candidate?

Dr. Chen: We expect our GKA candidate will be a first-in-class and, based on data from the Phase II trial, probably best-in-class oral type 2 diabetes treatment. The Phase II data is excellent, and we will be starting the Phase III trial later in 2017.  

ChinaBio: What’s next for the GKA drug after Phase III and, hopefully, approval?

Dr. Chen: Hua will be responsible for China commercialization and marketing. After approval, there are still a lot of things to do. We will need some way to identify patients with type 2 diabetes and then find an approach to them. Outside of China, we plan to co-develop and co-promote the drug there.

ChinaBio: You are also conducting clinical trials in the US.

Dr. Chen: Yes. The US is a very different market. In the US, the diabetic population takes metformin as a first-line treatment. So we administered our GKA treatment as an adjunct to metformin and the results were excellent. Because most China type 2 diabetics are treatment naive, we tested the drug as a monotherapy there. We saw excellent results in both countries.

ChinaBio: How does the ChinaBio® Partnering Forum fit into your strategy?

Dr. Chen: The most important role for the ChinaBio® Partnering Forum is to bring people together and allow our industry to develop. Most importantly, it brings investors together with innovators, entrepreneurs. For us, we are a small company, with about 30 to 40 people. We need a large community of 500 to 600 people in the medical and scientific communities to support our clinical research. The ChinaBio® Partnering Forum makes the community aware of us and helps us work with the larger ecosystem. It brings people together and provides an environment that allows the industry, all aspects of the industry, to develop.

ChinaBio: Are you in the hunt for additional drug candidates?

Dr. Chen: Our strategy is that, while we continue to develop and then launch our diabetes drug in China, we are talking to pharma partners and we open discussions about their potential portfolio molecules that Hua could license. In the future we would like to become a company that offers China market operations with partnerships in other regions. We could also co-promote products together outside of China.

There are several discussions underway currently. This is very exciting.

ChinaBio: Why does Hua adopt the China marketing/Ex-China co-promotion model?

Dr. Chen: Hua will do well marketing our own drugs in the China market because of our expertise and influence. Then, while we are working with partners outside of China for our drug development, we might also seek to license China marketing rights for their products. Using this model, both parties will be leveraging their strengths and partnering to benefit patients and the companies.

ChinaBio: What kind of partnering do you do in China?

Dr. Chen: We use a very innovative model here in China. We call it Collaborative Innovation. Hua has a strategic partnership with WuXi AppTec and its affiliated companies in the pre-clinical space, clinical development and contract manufacturing. Also we are working very closely with global CROs like TigerMed and dMed and a few others, working on clinical development and preparing for pharmacovigilance, monitoring and preparing for NDA approval. We have been working with over 30 partners in the areas of early discovery early development and commercialization/ manufacturing and then clinical development. Obviously, Hua will develop marketing skills through a partnership-based relationship.

ChinaBio: How did the GKA candidate perform in the China Phase II trial?

Dr. Chen: In the Phase II test, 268 diabetes patients received the treatment for three months on several different regimens. The efficacy was really great, lowering average HbA1c levels by 1.2%. The results were very significant with a p value of 0.01. Side effects were fairly benign — relatively little hypoglycemia for example — and in line with other oral type 2 diabetes treatments. It’s a good drug. Ours is the first successful trial of a GKA drug. Earlier versions caused hypoglycemia and often lost efficacy fairly quickly.

ChinaBio: When do you expect to put your GKA diabetes drug on the US market?

Dr. Chen: We have a plan for getting our partners ready in 2018-2019 and that’s where we will engage the US NDA trials. And then probably, it will take a few years to get through the trial and the drug will be ready to be launched around 2022-2023.

We want to help patients and the current drug is not very effective. There have been a lot of drugs brought to market since insulin was developed 100 years ago. Insulin makes sure that people don’t die from diabetes. But look at the disease: it’s increasing. The current drugs do not cure people. New patients are diagnosed while the older patients are still there. Fundamentally, the community has not been able to target the cause of the disease, but they work to simply lower the blood glucose level to acceptable levels. Their drugs target symptoms, not the cause of the disease. That’s where our drug is different. It targets the underlying cause and that’s why the clinical benefit is so big [Ed. — Hua’s 4th-generation GKA targets a key enzyme (GK) that acts as a glucose sensor and regulates carbohydrate metabolism. It was designed to avoid the side-effects of previous generation drugs]. We can talk more about that as we complete our Phase III trials.

ChinaBio: We talked, back when you were just starting Hua, and you said it was the perfect time to start a China innovative drug company. Were you right about that?

Dr. Chen: Yes, I think so. Basically at that time we said we wanted to be the most innovative biotech company in China and be able to offer first-in-class, best-in-class drugs for patients worldwide in exciting therapeutic areas. We just did that.

And the other part I said was that in China the regulatory and financial environment would improve, and that has also taken place. In the last few years, the CFDA has put new policies in place encouraging innovation and enforcing quality of marketed drugs and clinical trials. They are bringing the whole process of drug discovery and innovation level up to the level of the US and Europe. I think that’s a major step forward and Hua benefits from that.

Also, also the investment community is very much involved in drug development. In 2016 a huge amount of investment was made primarily to China innovative drug companies, mostly coming from China-based venture and PE funds. Significant progress I’d have to say, and it seems like what we predicted six years ago is coming true.

ChinaBio: Dr. Chen, congratulations on Hua’s success so far and thank for your time.

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