#BIO2017: What’s Happening in the World of IP?

Who’s ready to hit the beach?

Wait, did I say beach? I meant the San Diego Convention Center.

….But the beach will be waiting for you once you wrap up two and a half days of IP educational programming!

Let’s take a look at what will be on the agenda for BIO’s Intellectual Property Track next week at our International Convention (not registered yet? We can solve that, just go here!)

Monday, June 19, 2017

Of Rats and Men: The Success Story of Human Therapeutic Antibodies Produced in the Omnirat
Time: 4:00 PM–5:00 PM    Date: Jun 19, 2017
Location: Room 7B, Upper Level

 

Tuesday, June 20, 2017

 

 

 

Wednesday, June 21, 2017

 

**Continuing Legal Education

Some of our Intellectual Property sessions at BIO will offer attorneys an opportunity to obtain Continuing Legal Education (CLE) credits in select states. Application for CLE credit will be submitted in California and Virginia, and attorneys will be notified if BIO receives credit approval. Attorneys needing CLE credit from other states are welcome to sign in, pick up the CLE forms and apply to their jurisdictions on their own. BIO will provide you with documentation when applying for CLE credit.

#BIO2017: A Peek at Pavilions | Sub-Saharan Africa

Breaking Buzz is BIO’s newest blog series that reaches across the globe to bring you an insider’s preview into the hottest international and partnering trends coming to San Diego for the BIO International Convention.

The Fastest-Growing Continent You Thought You Knew

The African continent is currently home to seven of the world’s top-ten fastest-growing economies; and by 2050 (just 33 years from today!) is projected to represent 26% of the global population.

Let that sink in for a minute.

There are 54 countries within the continent which spans more than 11.7 million square miles – second only to Asia both in percentage of land on earth, and population; it is home to over 1.2 billion people many of whom are now referred to as the world’s fastest-growing middle class. And get this: World Economics, a research organization that measures global economic activity, reports that the African continent has surpassed Europe and the Americas in real GDP growth when measuring by continent, from 1961 to 2015.

So what’s a continent to do with so much potential? Breaking Buzz asked Jennifer Dent, President of BIO Ventures for Global Health (BVGH) just that and she says, it all starts at the BIO International Convention.

“We brought in the first African delegation to the Convention from Nigeria in 2014. The next year, six countries came, and we had the first Africa Pavilion. Last year in San Francisco we had 16 African countries present. But this year in San Diego we will have 24 countries from the African continent and upward of 30 delegates – and for the record, these aren’t just any delegates. The delegates at the pavilion have real authority, so expect action.”

She is referring to Ministry of Health officials and advisors representing several countries, Nigeria, Kenya, Tanzania, Côte d’Ivoire; and high-level decision makers from academic, non-profit organizations, and life science companies. Collectively they represent 24 of the African continent’s 54 countries and share a common mission: to engage biotechnology, pharmaceutical, and life science companies in meaningful initiatives, programs and partnerships to impact global health.

The quality of attendee is a direct reflection of recent findings reported by the World Bank who had this to say about Africa: Fostering public and private investment in infrastructure has become a priority to the African continent, [so] with well-targeted capital investments and policies fostering competitiveness and productivity, Africa’s larger and younger work force has the potential to transform the continent.

Jennifer Dent, President BVGH

“Think about what’s important to industry” says Dent; “they’re looking for growth opportunities in markets across the globe, and as the World Bank reveals, Africa has made considerable progress in its infrastructure development and emerging market status. While these countries have grown and prospered, however, so have non-communicable diseases like cancer and diabetes gown in prevalence; these aren’t just markets for anti-malarials and other anti-infectives anymore.”

To enter the African market, Dent stresses that partnerships will be critical to obtaining the in-depth knowledge of the continent’s infrastructure, politics, and disease landscape; that’s where the Convention comes into play and why she’ll be launching a new initiative in San Diego this year.

The African Access Initiative  will be launched on Wednesday at the BIO International Convention in San Diego; it is an integrated package of programs, activities, and partnerships designed to address the primary barriers to cancer treatment in Africa. The AAI will create new business models and tap into company products to establish access to cancer medicines and biologics. The program will augment the African Organization for Research and Training in Cancer which is furthering research relating to cancers prevalent in Africa and facilitating and supporting training initiatives in oncology for health-care professionals. AAI will also support leading cancer researchers dedicated to building clinical trial networks, and determining how genetics affect the types of cancer prevalent to the area.

Two groups from Kenya, incubator Villgro Kenya, and the Kenya National Innovation Agency whose Chairman Professor Reuben Marwanga will also be present, are both working toward the development of a better ecosystem to support innovation and innovators in the country.

“That’s a lot,” says Dent enthusiastically, “and we have just scratched the surface. There are many milestones being reached in Africa, and taking it all in can be a challenge; which is why we’re presenting a one-hour media event at BIO to overview each of the 24 countries and their primary areas of therapeutics.”

Jim Greenwood, BIO President and long-time Africa advocate will open the event at 10:45 on Tuesday morning, the first full day of the Convention. Also in attendance will be the European Commission’s Deputy Head of Unit, Health Directorate Dr. Philippe Cupers who will introduce the European and Developing Countries Clinical Trials Partnership.

The event will take place in the African Pavilion where you can visit 24 countries in one hour. No frequent flyer miles earned, but hey, what an efficient way to travel.

Filed under: Events, , , , , , , , , , , ,

#BIO2017: A Peek at Pavilions | Sub-Saharan Africa

Breaking Buzz is BIO’s newest blog series that reaches across the globe to bring you an insider’s preview into the hottest international and partnering trends coming to San Diego for the BIO International Convention.

The Fastest-Growing Continent You Thought You Knew

The African continent is currently home to seven of the world’s top-ten fastest-growing economies; and by 2050 (just 33 years from today!) is projected to represent 26% of the global population.

Let that sink in for a minute.

There are 54 countries within the continent which spans more than 11.7 million square miles – second only to Asia both in percentage of land on earth, and population; it is home to over 1.2 billion people many of whom are now referred to as the world’s fastest-growing middle class. And get this: World Economics, a research organization that measures global economic activity, reports that the African continent has surpassed Europe and the Americas in real GDP growth when measuring by continent, from 1961 to 2015.

So what’s a continent to do with so much potential? Breaking Buzz asked Jennifer Dent, President of BIO Ventures for Global Health (BVGH) just that and she says, it all starts at the BIO International Convention.

“We brought in the first African delegation to the Convention from Nigeria in 2014. The next year, six countries came, and we had the first Africa Pavilion. Last year in San Francisco we had 16 African countries present. But this year in San Diego we will have 24 countries from the African continent and upward of 30 delegates – and for the record, these aren’t just any delegates. The delegates at the pavilion have real authority, so expect action.”

She is referring to Ministry of Health officials and advisors representing several countries, Nigeria, Kenya, Tanzania, Côte d’Ivoire; and high-level decision makers from academic, non-profit organizations, and life science companies. Collectively they represent 24 of the African continent’s 54 countries and share a common mission: to engage biotechnology, pharmaceutical, and life science companies in meaningful initiatives, programs and partnerships to impact global health.

The quality of attendee is a direct reflection of recent findings reported by the World Bank who had this to say about Africa: Fostering public and private investment in infrastructure has become a priority to the African continent, [so] with well-targeted capital investments and policies fostering competitiveness and productivity, Africa’s larger and younger work force has the potential to transform the continent.

Jennifer Dent, President BVGH

“Think about what’s important to industry” says Dent; “they’re looking for growth opportunities in markets across the globe, and as the World Bank reveals, Africa has made considerable progress in its infrastructure development and emerging market status. While these countries have grown and prospered, however, so have non-communicable diseases like cancer and diabetes gown in prevalence; these aren’t just markets for anti-malarials and other anti-infectives anymore.”

To enter the African market, Dent stresses that partnerships will be critical to obtaining the in-depth knowledge of the continent’s infrastructure, politics, and disease landscape; that’s where the Convention comes into play and why she’ll be launching a new initiative in San Diego this year.

The African Access Initiative  will be launched on Wednesday at the BIO International Convention in San Diego; it is an integrated package of programs, activities, and partnerships designed to address the primary barriers to cancer treatment in Africa. The AAI will create new business models and tap into company products to establish access to cancer medicines and biologics. The program will augment the African Organization for Research and Training in Cancer which is furthering research relating to cancers prevalent in Africa and facilitating and supporting training initiatives in oncology for health-care professionals. AAI will also support leading cancer researchers dedicated to building clinical trial networks, and determining how genetics affect the types of cancer prevalent to the area.

Two groups from Kenya, incubator Villgro Kenya, and the Kenya National Innovation Agency whose Chairman Professor Reuben Marwanga will also be present, are both working toward the development of a better ecosystem to support innovation and innovators in the country.

“That’s a lot,” says Dent enthusiastically, “and we have just scratched the surface. There are many milestones being reached in Africa, and taking it all in can be a challenge; which is why we’re presenting a one-hour media event at BIO to overview each of the 24 countries and their primary areas of therapeutics.”

Jim Greenwood, BIO President and long-time Africa advocate will open the event at 10:45 on Tuesday morning, the first full day of the Convention. Also in attendance will be the European Commission’s Deputy Head of Unit, Health Directorate Dr. Philippe Cupers who will introduce the European and Developing Countries Clinical Trials Partnership.

The event will take place in the African Pavilion where you can visit 24 countries in one hour. No frequent flyer miles earned, but hey, what an efficient way to travel.

Filed under: Events, , , , , , , , , , , ,

#BIO2017: A Peek at Partnering | SPARK

Breaking Buzz is BIO’s newest blog series that reaches across the globe to bring you an insider’s preview into the hottest international and partnering trends coming to San Diego for the BIO International Convention.

Perfecting Translation

Stanford’s translational research program, SPARK, is only 11 years old yet already has funded and supported 148 past and current projects, helped launch 24 start-ups and has had over 30 patents licensed. Spark lays claim to a 62% success rate; measured when a project enters into human clinical trials, or is licensed to a start-up or existing company.

With those kind of stats it’s no wonder Mahima Agochiya, Business Development and Program Manager for SPARK was besieged with meeting requests at last year’s BIO International Convention within BIO’s One-on-One Partnering™ system.

SPARK’s remarkable program – a partnership between Stanford and volunteers from the biotechnology, pharmaceutical, and healthcare investment industries – offers support to an average of 12-15 new projects each year.

Mahima Agochiya PhD, MBA
Business Development and Program Manager
SPARK Translational Research Program at Stanford University School of Medicine

Breaking Buzz sat down with Mahima to chat about the 69 meetings she and her colleagues attended in three days during the BIO International Convention in San Francisco, and asked about her strategy for partnering success.

We know SPARK is a Translational Medicine Program based out of Stanford School of Medicine that seeks partnerships with industry to help translate its projects, and is entering into its second year attending BIO. What kind of partner are you looking for this year?

SPARK is agnostic to indication so we are not limited to specific areas of therapeutics. We generally look for companies that are interested in early stage start-up funding or licensing; or both. The assets/ projects that we are looking to partner with this year are novel, address an unmet clinical need, and are at a stage where they ready for the next step.

There are thousands of companies in the BIO’s One-on-One ™ system representing tens of thousands of assets. How did you narrow it down to 69 meetings?

The first thing I do is add filters. We are mostly interested in companies that license at early-stage since our projects are preclinical at the most, so my go-to filters are “licensing” and “early-stage” for example. I then send out a lot of meeting requests. Perhaps it was because last year was only the first year that SPARK attended BIO, and my first year too, but I was very surprised by the number of invitations we got from other companies. It helps a lot to belong to a university that has a great reputation scientifically and I think people were curious about us, given our success rate.

Can you tell us about that success rate?

SPARK has a 62% success rate on all projects we take in. So far we’ve spun out 24 start-ups; eight licenses to biopharma; four tech transfers without license, and 31 clinical trials – 10 without license. To date we have provided education, mentorship and funding to 148 projects and hundreds of students.

Last year SPARK had 35 projects (assets) that you presented during partnering meetings at the Convention. Did any of those materialize into a licensing opportunity?

Yes! Last year resulted in at least one successful partnership that I will be able to talk about at the Convention this June, and we have ongoing negotiations with other companies we met, most of whom we’ll meet with again in San Diego. BIO provides a great opportunity to get to know new companies and share experiences with them. I think it might be hard to walk away with nothing.

What’s your advice to people new to the Partnering system?

Don’t have a script ready. Instead, open with a clear and concise statement saying exactly what you’re looking for. A half-hour isn’t long enough to delve into details, but rather to quickly ascertain if there is a viable partnering opportunity; so keep the conversation direct at opening, then very flexible. Also, provide all of your materials on a USB. SPARK’s assets are detailed, each on a single page, in one file given on a single USB drive.

Besides partnering meetings, what else is on your Convention calendar?

On Monday from 11:30 – 4:30, SPARK will team up with partners from Massachusetts, Quebec, Philadelphia, San Francisco Bay Area, Oslo, UK Golden Triangle, Catalonia, and the Paris Region to present cutting edge innovations in oncology at the 6th International Cancer Cluster Showcase. Each cluster will present their oncology pipelines in compact 20-minute presentations, so it’s the perfect opportunity to learn about the latest innovations and a great precursor for partnering meetings on Tuesday, Wednesday, and Thursday.

Why did you get an MBA eight years after getting a PhD in cancer research?

The two degrees are actually a perfect combination. After finishing my PhD I did about eight years of basic science which was very rewarding, but also a little heartbreaking because I never saw the work translated. That’s what made me do the MBA – now I get to see all the cool science and get to see them translate – at Stanford University no less. How perfect is that?

#BIO2017: A Peek at Partnering | UC Berkeley

Breaking Buzz is BIO’s newest blog series that reaches across the globe to bring you an insider’s preview into the hottest international and partnering trends coming to San Diego for the BIO International Convention.

A Day in the Life of a Partnering Powerhouse

UC Berkeley has had a whopping 600+ products commercialized from its research since it started counting in the 1980s. Its success stories range from mega blockbusters like CRISPR/CAS 9 to some of the early research and development of iplimumab. No matter how large or small, each of the hundreds of products is worthy of fulfilling the Berkeley School of Public Health’s mission: to improve population health, especially for the most vulnerable.

One big obstacle, however, is that as a non-profit, Berkeley is unable to commercialize the cool technologies being developed on campus, so they have to look at things differently than many of their research and development counterparts. Top of the list: they must find industry partners to license their IP. This takes time, instinctive focus and without a doubt, stamina.

Akash Bakshi, Berkeley’s resident IP and industry research authority, knows all about trusting his instincts and building stamina. He singularly held 59 BIO One-on-One Partnering™ meetings last year at the BIO International Convention in San Francisco; with a remarkable 75% follow-up return – an accomplishment he says required first and foremost, persistence.

Breaking Buzz talks with the persistent partnering Phenom in a recent interview:

AKASH:  Be persistent because companies are busy. Don’t leave it to them to sort though and find your invitation. If you haven’t heard back and your gut tells you there’s a good reason to connect, persist!

BB: There are thousands of companies within the partnering system. How do you know who to pursue?

AKASH: I look for companies that overlap with our research strengths – whether in biomedical engineering, immunology, or molecular and cellular biology, companies who seek new insight in the life sciences. Those are the ones that always make great partners!

BB: Can you tell us more about what happened after the 59 meetings in San Francisco last year?

AKASH: Almost 75% of the meetings we set up last year resulted in further discussions about UC Berkeley’s Innovation Ecosystem, IP licensing, and even employment for our students. I attribute the high return rate on Berkeley’s breadth and research strengths. People understand we are a powerhouse for early-stage R&D. Industry knows we have the intellectual capital to do amazing stuff.

BB: How do you characterize a successful Partnering experience?

AKASH: Some folks might assess their success at BIO as the number of transactions that were completed; our view is that this is more about relationships. Success comes anytime we can collaborate to improve or validate early-stage findings and licensing IP; when we get feedback on potential technologies, investments and funding for our startups, and the hiring of our graduates.

The other side of the story is that we get good insight from industry about why they were NOT currently interested in the ideas we presented – it might be because regulatory hurdles are too high, or the project is too small. Regardless, it’s allowed me to give feedback to the faculty member to rethink the original applications.

BB: Who is on your radar as we approach the BIO International Convention in San Diego?

AKASH:  There are updates that I’m eager to share with some of the people I met with last year in San Francisco. But I’m just as excited about finding new partners this June in San Diego; companies that share our quest for innovation in the life sciences.

BB: Sounds like you’ll have another very busy BIO experience. How do you do it?

AKASH: This year there will be two of us, so my record of 59 meetings will hopefully double. But ultimately, I focus on the satisfaction I get from finding and developing ongoing relationships within an industry I love.

Well that, and a steady stream of Venti Caffe Lattes!

 

Same name, different company: Arena’s new CEO cuts the fat

It’s easy to see how Arena Pharmaceuticals grew to be a fragmented and strained biopharma company.

Founded in 1997, the San Diego, California startup was dedicated to the research and development of drugs targeting G-protein-coupled receptors (GPCRs).

Out of its preclinical research came a number of promising candidates. Among them; lorcaserin, a potential weight loss therapy. How could that not sell in a world battling an obesity epidemic?

Money was raised and resources were pumped into advancing lorcaserin — later sold as Belviq. Meanwhile, the R&D engine kept churning to produce more GPCRs.

As Arena struggled to balance its early-stage and late-stage assets, the clinical pipeline in the middle was being ignored, said new CEO Amit Munshi.

“The company essentially became, if you could imagine, a barbell,” Munshi said in a phone interview. “On one side you had this fantastic g-coupled-protein receptor platform and on the other side you had the build-out around Belviq.”

It was too much for the team to balance. By 2012, however, an end to the resource strain was in sight.

After a long battle with the FDA, lorcaserin was provisionally approved for sale in the United States. It was a major milestone, but as Arena’s five-year stock price demonstrates, the drug fell well short of sales expectations.

Instead of funding the preclinical and clinical pipelines, lorcaserin was costing Arena and its commercialization partner money.

Source: Seeking Alpha (03/31/2017)

Source: Seeking Alpha (03/31/2017)

Under an earlier agreement, Eisai had taken ownership of Belviq’s marketing and sales. It was required to pay Arena over 30 percent in royalties in return — enough to dash Eisai’s hopes of turning a profit, but not enough to help Arena offset the cost of its obligations, which included an $80 million Phase 4 cardiac outcomes trial.

Belviq was dragging the organization down, money was hemorrhaging from the preclinical research arm and its clinical assets were going nowhere.

Trimming the fat

That’s the situation Munshi signed up for when he took over as CEO in May 2016.

“The company was really in no man’s land 12 months ago,” he recalled. “It was a discovery research company without the capital to do discovery research. It had a bunch of products in the clinical development portfolio but without the capital or the manpower to progress those products forward. And it had Belviq on the other side, which was relatively flat in terms of sales, but we had substantial obligations.”

The more he looked at it, the more he realized the company couldn’t be everything to everybody. His job was to strip back the clutter to identify the real potential.

It wasn’t Belviq.

“We focused on three sets of activities,” Munshi said.

The first step was divesting the research discovery platform, which was burning around $25 million in cash each year. Munshi held on to a few economic assets and spun the rest into a separate entity called Beacon Discovery, headed by Arena cofounder Dominic Behan.

The second major task was to stem the financial bleed from Belviq.

Two other weight loss drugs, Orexigen’s Contrave and Vivus’ Qysmia, were approved around the same time. All three have struggled with sales. Munshi’s solution was not to reinvigorate the franchise; he worked with the company’s board to formally step back.

“I’ve spent very little time thinking about what went wrong because it’s largely irrelevant to our story going forward,” Munshi said. 

Arena’s team began working with Eisai towards a more mutually beneficial argeement. The resulting deal allowed Arena to step back into a passive role, forfeiting its clinical obligations in return for a significant reduction in royalties, to between 9.5 and 18 percent.

Resetting the bar for royalties took some of the upfront pressure off Eisai as it struggled to find its feet. Arena, meanwhile, stood to save up to $100 million in expenses.

Starting fresh

The final piece of the puzzle was the company’s clinical pipeline. It had been neglected, but according to Munshi, held huge potential.

A new clinical development architecture needed to be built, to progress the drug candidates and to serve as the basis for a reimagined Arena Pharmaceuticals.

Arena Pharmaceuticals CEO Amit Munshi

Arena Pharmaceuticals CEO Amit Munshi

“A big part of my job is being able to convince a really strong executive team to join Arena. As soon as people see what we’re doing, see the pipeline and what’s ahead. It really turns a lot of heads,” Munshi said.

About half of the company is now dedicated to product development, compared to just a handful of people a year ago. He added a medical affairs team, pricing experts, and more clinical expertise. There was only one physician when he joined, now there’s six.

Arena’s pipeline centers around what they hope are better versions of existing drugs. The pathways, mechanisms and market potential are all well characterized and proven. With better chemistry and pharmacokinetics, Munshi believes his company has an edge.

The most advanced candidate is ralinepag (APD811), an oral therapy that selectively binds certain prostacyclin (IP) receptors. It works to widen blood vessels (vasodilation) for patients with pulmonary arterial hypertension (PAH).

Ralinepag, Mushi said, compares to Actelion’s Selexipag, an IP receptor that entered the U.S. market in January 2016. Its sales totaled 245 million Swiss francs (US$245 million) that year.

“We believe we’ve got the next generation molecule. We’ve got better pharmacokinetics and better potency that Selexipag,” he said.

A Phase 2 data readout for ralinepag is expected mid-year.

Next in line is etrasimod (APD334), an oral, next-generation therapy for autoimmune diseases, including ulcerative colitis. It works by selectively targeting S1P receptor subtypes.

“We believe etrasimod is an improved version of a drug called ozanimod,” Munshi stated, adding that the latter drug was the cornerstone of Celgene’s $7.3 billion acquisition of Receptos.

As with ozanimod, etrasimod has the potential to treat multiple indications. Arena is working on four right now. Data from several of those is expected in Q4 of 2017.

The third key compound is APD371, which binds cannabinoid 2 (CB2) receptors. It has a Phase 2 readout slated for the fourth quarter of this year in pain associated with Crohn’s disease.

“It’s important to remember that Arena really hasn’t had efficacy data on any compound in the last five-to-seven years,” Munshi explained. “And this year we expect somewhere between three and five clinical data readouts.”

That could lead to new financing and more evidence of a fresh start. Arena is slowly but surely changing the conversation.

The culture is changing too. Around 90 percent of the management team and over 50 percent of the wider crew joined after the new CEO, most in the last 180 days.

All of that begs the question: Did the team consider a name change?

“Yes, we did. It’s something I would have liked to do,” Munshi said ruefully. “However, name changes cost a lot of money and they are operationally distracting.”

It’s not just the name and ticker symbol. The company markets two products in some 25-odd countries, he noted, which would have made it a large undertaking. So they settled on a refresh of the logo and the website, for now.

“I want to focus on a few things and really do them well,” he said.

In a nutshell, that’s the new Arena.

Photo: erhui1979, Getty Images

Gore joins ViaCyte’s quest for a functional type 1 diabetes cure

A cure for type 1 diabetes has been “just around the corner” for decades now — or so patients have been told. But the moonshot mission has been rough.

ViaCyte knows this well after years 18 years of R&D. With every step of progress, a new mountain of challenges looms. The company puts its head down and troubleshoots through, with backing from private investors, pharma partners, the California Institute for Regenerative Medicine (CIRM) and the Juvenile Diabetes Research Fund (JDRF).

On Wednesday, the San Diego, California-based company announced a new partner; W. L. Gore & Associates, the multi-billion dollar manufacturer of medical and non-medical fabrics and devices, including the iconic GORE-TEX. If Gore can contribute some materials expertise, one more problem could get solved.

The partnership centers around ViaCyte’s flagship islet replacement therapy. 

Type 1 diabetes (aka juvenile diabetes) is an autoimmune disease. It accounts for around 5 percent of all diabetes cases. Some 95 percent are caused by so-called adult-onset diabetes, which is a longer-term metabolic disorder.

While genetic and environmental factors are believed to play a role, the ultimate trigger for type 1 diabetes is not known. At some point, the immune system incorrectly recognizes beta cells in the pancreas as foreign or threatening. It begins systematically destroying them and with that, the ability of the body to produce insulin.

Insulin is the key that allows glucose to enter cells. Without insulin, glucose builds up in the bloodstream while cells are effectively being starved. For decades, patients have replaced the missing insulin with a synthetic version. Yet dosing is problematic. Too little insulin and the blood glucose levels rise; too little and the patient becomes hypoglycemic — a potentially fatal condition if the individual isn’t revived with a readily available source of sugar.

Patients must manage this life or death balancing act for the remainder of their days.

ViaCyte hopes to remove the uncertainty with its PEC-Encap combination product. It comprises PEC-01 pancreatic progenitor (stem cell precursor) cells packaged in an immune-protective device called the Encaptra Cell Delivery System. 

Once implanted under the skin, the PEC-01 cells further mature into insulin-producing ‘islets’ — beta cells included.  Blood can flow in and out of the device, allowing the cells to register blood glucose in real time and produce an appropriate amount of insulin in response. 

To preserve the biological package, ViaCyte has been fine-tuning its transplant device, which CEO Paul Laikind likens to a tea bag.

“In the case of PEC-Encap it’s a semi-permeable membrane that is really the active component of the device,” Laikind said in a phone interview. “That membrane is sized such that oxygen, nutrients, glucose, and even proteins can move freely back and forth across the membrane surface. But it is also sized such that cells cannot. So none of the immune cells of the host are able to penetrate and communicate with the cells inside, so to speak, or directly access the cells in the device. And none of the cells inside the device are able to exit.”

That separation is critical for staving off the two-pronged immune response waiting to neutralize the cells.

Once triggered, the immune system of diabetes patients will forever be geared towards destroying beta cells. That’s the autoimmune component. The second issue is the natural immune rejection that occurs when cells or tissues containing foreign DNA are introduced into a transplant patient. ViaCyte uses stem cells derived from an embryo, which triggers this kind of reaction.

Finding a balance between cell preservation and efficacy is hard. According to Laikind, ViaCyte had progressed into Phase 1/2 trials with the PEC-Encap, but after treating around 20 patients it identified an issue with vascularization.

The membrane doesn’t allow the components of blood vessels to pass through — they’re too big. Yet the cells need nutrients and oxygen to survive.

“When it engrafts, what we need is that vascular network to set down on the outside of the device,” Laikind said.

That’s why Gore’s expertise in medical devices is needed.

Another product being forwarded by the ViaCyte is a straight transplant of insulin-producing cells. The patient would need to take immune-suppressing medications indefinitely to protect the introduced cells. According to Laikind, the side effect profile of this approach is better suited to patients who struggle to maintain stable blood glucose levels or for the 10-40 percent of patients who have hypoglycemia unawareness. In the latter cases, the individuals are unaware their blood sugar is dropping and thus, can’t halt their descent into hypoglycemia.

Beyond ViaCyte, other companies are attempting to break into the field.

Novo Nordisk, the world’s largest manufacturer of diabetes drugs, has signaled its intent to move into stem cell therapies. Another company, Semma Therapeutics, is eying up clinical trials, having developed a method for efficiently producing beta cells en masse.

Both are several steps behind ViaCyte, which is a blessing and a curse for the San Diego team. The company has raised millions as the flag bearer for a lasting therapy for type 1 diabetes. But it is also the first to encounter unforeseen challenges in the field. It’s those challenges that continue to stall any chance of a cure. For now, at least.

Photo: Hero Images, Getty Images

A lot to GAIN: Zavante awaits pivotal data on retro-antibiotic

When the current drugs stop working and there’s a shortage of anything new, what options does the medical community have to combat antibiotic resistance?

Circa 2010, the answer was ‘not a lot.’

It was that dire lack of options that led to a bipartisan initiative dubbed the Generating Antibiotics Incentives Now Act (GAIN Act), signed into effect in 2012. That year, the CDC estimates some 23,000 Americans died from antibiotic-resistant infections.

While the situation remains critical, the GAIN Act is working, said Ted Schroeder, founder and CEO of Zavante Therapeutics.

“The overall intent was to broaden the number of products that were available to U.S. patients,” Schroeder said in a phone interview.

That means new drugs and old drugs that could hold untapped potential.

San Diego, California-based Zavante is chasing the latter. With the passing of the GAIN Act, Cofounder and CSO Evelyn Ellis-Grosse picked up U.S. rights to an old antibiotic known as fosfomycin.

The discovery of fosfomycin dates back to 1969 and it has been used at low doses in regions outside the U.S. for several decades since. It is also sold locally as an oral formulation called Monurol, though Schroeder noted that its bioavailability is low.

In an era of antibiotic resistance, where’s the logic in going back to an older generation of drugs?

Schroeder cites two reasons. The first is the unusually benign safety profile of fosfomycin. It allows Zavante to take a “modernized approach to dosing,” upping the dose and administering it intravenously, he said. It was part of the reason Ellis-Grosse sought out the U.S. rights.

A second distinguishing feature is the drug’s one-of-a-kind “primitive” mechanism of action, which could help extend the life of the drug.

“It doesn’t need a specific binding protein, so the opportunity for cells to evolve around the antibiotic and create a new pathway for resistance is dramatically lower for this antibiotic than it is for the other classes,” he explained.

At a basic level, the drug is also effective against a broad spectrum of Gram-negative and Gram-positive activity, including activity against multi-drug resistant (MDR) pathogens associated with life-threatening infections.

For it to make economic sense, a variety of incentives had to come together for Zavante to launch and raise a $45 million Series A the following year.

Its formulation of fosfomycin, called ZTI-01, was designated a Qualified Infectious Disease Product (QIDP), part of the GAIN Act program. QIDP creates a path for important antimicrobials to receive priority review, fast track designation, and a five-year extension of patent exclusivity.

The latter benefit was critical. Prior to the GAIN Act, the antibiotic would have received just three years of patent exclusivity through the Hatch-Waxman Act of 1984.

“This antibiotic would not even be a discussion if it weren’t for the GAIN Act,” Schroeder said. “With three years of Hatch-Waxman as your only regulatory exclusivity, you couldn’t afford to invest $45 plus million to bring the product to the market here. You just don’t have enough time to make a return on that investment.”

Given a version of this drug is already approved in the United States, ZTI-01 qualifies for a 505(b)(2) regulatory pathway. Existing data and studies can be used as supporting evidence. It allowed Zavante to move directly from Phase 1 through to a pivotal Phase 3 trial in hard-to-treat urinary tract infections. The data readout is expected soon.

All going well, the drug could reach the market by the end of 2018. Once approved, there are many, many possible areas to expand into, Schroeder said.

“This isn’t the answer to the problem, but it’s a significant step forward,” he noted, of the push to get ZTI-01 to the United States to help fight antibiotic resistance here.

Photo: Andrew Brookes, Getty Images

Dexcom will reap benefit as CMS posts criteria for Medicare coverage of CGMs

The Centers for Medicare and Medicaid Services has released guidelines on Medicare coverage for continuous glucose monitors, and one medtech company stands to make a whole lot of money from the change.

San Diego-based DexCom is the only manufacturer of a continuous glucose monitor (CGM) defined as therapeutic, meaning patients can make treatment decisions using the device. The DexCom G5 Mobile System provides the glucose level, the direction and rate of change, and uses built-in customizable alarms to alert wearers when their glucose is too low or too high. Low glucose levels can induce seizures, loss of consciousness, coma and death.

DexCom, diabetes professional societies, endocrinologists, the American Diabetes Association and the Juvenile Diabetes Research Foundation (JDRF) have been working on this with CMS for years, according to DexCom CEO Kevin Sayer.

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“It does expand our patient base to a group of people who really need this technology,” Sayer said in a phone interview. “It’s more than a nice business opportunity. It’s going to be a life-changing experience for these people. It will be something that will be fun to be part of.”

CMS announced its decision to cover the monitors for Medicare patients in January, and this week said that patients who have either Type 1 or Type 2 diabetes and who must intensively manage their insulin will be able to obtain reimbursement for the devices.

To get the coverage, beneficiaries must meet four criteria, according to CMS:

  • Have a diagnosis of Type 1 or Type 2 diabetes;
  • Have used a home blood glucose monitor and performing four or more blood sugar tests per day;
  • Take multiple daily injections of insulin or use a continuous subcutaneous insulin infusion pump;
  • Have a treatment regimen that requires them to make frequent adjustments based on their blood sugar test results.

“These definitions would apply to almost everyone with Type 1 diabetes,” JDRF senior vice president for advocacy and policy Cynthia Rice said in a phone interview. “It might not apply to everyone with Type 2 diabetes.”

JDRF raises money for research and awareness of Type 1 diabetes, which is usually diagnosed in children and young adults. People aged 65 and older who have Type 1 diabetes have usually been suffering from it for decades, and are more susceptible to low blood sugar because their bodies are less able to detect it, Rice explained.

“Lows can happen at night or during the day,” Rice said. “But at night, one of the advantages of the alarm is that other family members can hear it.”

DexCom began ramping up its production following CMS’ January decision. The company plans to open a second factory, in Arizona, in 2018, Sayer said. About 75 percent of the 200,000 patients who use the G5 are based in the United States and most are covered by commercial insurance, he added. Sales have grown from $66 million in 2011 to $571 million in 2016.

Medicare will reimburse for CGMs at a rate of $250 to $275 per month or about $3,000 per year, which covers rental of a durable component and purchase of disposable accessories, Sayer noted. The other major manufacturer of CGMs, Medtronic, does not have the proper FDA labeling for to obtain Medicare coverage. The company did not respond to requests for comment.

Private insurers began covering CGMs in 2008 and 2009, according to Rice.

“It took a long time to get CMS here, but it’s wonderful that they have taken this step,” Rice said. “We’re really pleased with their decision in January, and what happened yesterday really makes it real, makes it ready to implement. So many people will benefit from having access to this important technology.”

Photo: Jirsak, Getty Images

#BIO2017: A Peek at Partnering | Protalix Biotherapeutics’ Bill Taylor

Breaking Buzz is BIO’s newest blog series that reaches across the globe to bring you an insider’s preview into the hottest international and partnering trends coming to San Diego for the BIO International Convention. 

Breaking Buzz Sits Down with Partnering King

BIO’s One-on-One Partnering™ opens April 10 and it’s not too early to register, set up a profile and start requesting and accepting meetings before schedules get filled. BIO will host a Webinar on April 5 to explain everything you need to know about the partnering system and offer tips to ensure a successful BIO 2017.

Recently we connected with Industry veteran Bill Taylor, one of BIO’s most prolific partnering delegates, having single-handedly scheduled and attended 55 formal meetings, over just three days, during last year’s International Convention in San Francisco. This impressive number of well-executed meetings wasn’t beginner’s luck. Mr. Taylor first attended BIO when he was with Pharmacia in 2001 and since then has racked up a lot of meetings, many of which resulted in more meetings. We can all agree that with more meetings often come lasting partnerships and potential product commercialization agreements.

Mr. Taylor, now Vice President, Business Development for Protalix Biotherapeutics, sat down with Breaking Buzz to share some of his strategies on how to pull off a great number of effective meetings in a meager amount of time.

BB:  What’s the single most important task when it comes to scheduling partnering meetings?

TAYLOR: It will seem obvious, but the most important thing I’ve learned is to DO YOUR HOMEWORK. I usually start researching companies within three months of the Convention because otherwise it can feel like diving into a mosh pit of 20,000 people. The online instructions are a great place to start. A close second most important task though, is to make sure you populate your profile really well and use well-chosen key words to highlight your company interests and offerings, not just name and location.

Bill Taylor, VP Business Development, Protalix Biotherapeutics

BB:  What is the most important part of a company profile?

TAYLOR: In the Protalix profile I talk about what we’re doing and what our pipeline is, which allows other companies looking for opportunities to send me an invite. You’ve got to recognize that your company profile on the system is a two-way street in that respect. Our profile will read something like this:

Protalix is a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by our proprietary ProCellEx® plant cell-based protein expression system. Our pipeline consists of proprietary, potentially clinically superior versions of recombinant therapeutic proteins that target established pharmaceutical markets with a focus on rare diseases.

BB:  How many people do you think should attend a partnering meeting from each company?

TAYLOR:  Two to three, tops. One or two from the company, and myself. That said, sometimes companies want to bring their science and technical experts to do some drill-down for their initial diligence questions. Knowing this in advance will save time.

BB:  What should a company expect to achieve during partnering meetings?

TAYLOR: Like most things you get what you put into it. So, if I’ve done my homework, I expect to walk away with well-grounded conversations that cover the full width of our pipeline; conversations where I can articulate our value propositions, while conveying the commercial opportunities that our products represent. BIO is unique because it provides a very broad band forum in which to meet up and engage with a large number of companies across a vast range of geographies.

BB:  Should a company lock-down their schedule, or keep it flexible?

TAYLOR: I find that there’s no lack of people pinging me with lots and lots of invites, so I have to be highly disciplined about my scheduling strategy, while remaining open-minded to unforeseen possibilities. The challenge is to get your scheduled meetings locked down early, but it’s equally important to have some safety valve time-slots available to ensure a meeting can actually occur if it comes in on a last-minute blitz.

BB:  This will be your 16th consecutive year attending the BIO International Convention. What keeps you coming back?

TAYLOR: BIO is a lot like Homecoming. There may be nearly 20,000 people but we have a lot in common; we’re a collegial group that live and survive by the networks we’ve created over the years. Even waiting for a coffee — or a beer at the end of the day — you’ll strike up a conversation with someone who may end up as a great resource or they might be someone you’ve met at a past event.  It’s a place for former colleagues to re-connect, meet up with old contacts and make new connections. In that respect it really does feel like Homecoming.

BB: Does that make you the BIO International Convention Homecoming King?

TAYLOR: I guess so. You should see my convention badge collection!  After all these years, I’m still just a science geek!

Athena Staton serves as executive editor for BIO Buzz, the Official Show Daily for the BIO International Convention, and BIO’s Breaking Buzz blog series.