What makes a successful incubator?

According to Dr. Walshok, incubators must be characterized by:

1. Openness, a willingness to talk about failures as well as successes.
2. Talent.
3. Diversity of ideas and solutions. This includes rewarding performance.

Dr. Sharon Chan discusses the success at JLABS @ Shanghai - a no strings attached incubator where over 70% of the residents are first-time entrepreneurs. The goal of JLABS is the discovery and advancement of the best science in the world so that innovators can deliver to patients.

Current biotech industry trends

Dr. McKee notes the level of disruption COVID-19 has had on biopharma companies:

1. Companies that already had vaccine experience are able to marshal resources quickly to focus on COVID.
2. Companies with less-active infectious disease activity, but may have had immunological asset, will adapt ongoing business while contributing to COVID.
3. Small to medium companies outside infectious diseases are the most stressed.
4. Companies with less than a year of cash runaway are the most challenged and viewed as a risky investment.

Susie Harborth says the pandemic has changed science and put an emphasis on the need to collaborate. 

Rajeev Dutt discusses one of the problems AI is trying to solve - the timeline of the drug discovery process. “One of the problems that we are trying to solve is the fact that drug discovery currently takes about $2.5 billion over 12 years on average.  One of our central premises – and this is something shared by a number of pharmaceutical companies around the world – is that discovery of new drugs and therapeutics will be driven by AI,” he says. 

Susie Harborth says the companies that have been able to adapt to the work from home environment will be the ones that will move into the future and work in a "seamlessly virtual environment." 

“The companies that have been able to pivot to this #WFH and adaptation of technology to enable collaboration are going to be the ones that are going to be able to move into the future” Susie Harborth of @MissionBioCap #BIODigital pic.twitter.com/hesoSaLc3H

— I Am Biotech (@IAmBiotech) June 10, 2020

The most important elements when trying to find an R&D partner

Dr. Ted Love, CEO at Global Blood Therapeutics succinctly summarized three principles when evaluating partnership opportunities: 1) financial capability to provide capital 2) strategic advantage, particularly in a specific therapeutic area, for development or commercialization 3) tackling problems at scale.

Overcoming challenges with external partnerships

Dr. Sara Nayeem pointed to the challenges of meeting milestones and aligning on a time frame when moving a project forward. While approaches may vary, communication is critical as obstacles are encountered along the way.

The role of patient organizations in the context of partnering for rare diseases

Relationships with patient advocacy organizations are critical in the rare disease space. Companies benefit from collaborating with these organizations to establish desirable clinical outcomes for that patient population. Companies are increasingly hiring staff specifically devoted to engagement with patient organizations.

Best practices for engaging with patient organizations

“I would say early and often,” Jazz Pharmaceutical’s Robert Iannone noted.

How partnerships have changed given the pandemic

The formation of new partnerships has been difficult given the pandemic, but that hasn’t completely ceased the work that is being done. Gregory DiRusso highlighted Pfizer’s focus on working with regional guidelines in moving forward or pausing clinical trials. He also pointed out that for many clinical trials, direct data capture allows patients to circumvent appearing at sites to report making it easy for trials to continue in an era of social distancing.   

Overall Takeaway

The sharing of expertise and capabilities between internal R&D teams and external partners plays a vital role in advancing rare disease diagnosis and drug development more quickly, efficiently, and effectively – and remains a cornerstone of the industry.

This session was sponsored by Jazz Pharmaceuticals

-Jeremy Isenberg

“I do think we’re at an inflection point. You can always say that the science is getting better—that’s the nature of science, it builds upon itself," he said.

"But where we’re at now, with cell therapy, and gene therapy, immunoncology, and all that’s coming forward through gene editing opportunities, I think the pace is going to pick up – unless the politicians mess things up.”

"We're at a low point for how we're perceived," but we'll overcome that.

Fun fact: Jim loves birds.

He started birdwatching in college, and he's a board member of the National Audubon Society!

And in his next chapter, he'll focus on climate change.

“I think climate change is dire," he said.

"It’s an existential threat to the planet. Few things are: nuclear war, climate change, a horrible pandemic, far worse than this one. But climate change trumps them all, in my opinion, because it goes to the issue of inhabitability of the planet, not only by all of the species but by our species, so that’s what I want to focus on.”

"We can solve this problem, but only if the US leads," he said. 

But we won't lead if there are too many climate deniers in Congress.

He's created a Super Pac, Fight for Earth.

"We have to have a movement," he said. 

The science is clear. Climate change will destroy the economy, and the economic opportunities to confront it 

– Good Day BIO Editors

Commenting on the path to approval, FDA’s Dr. Marks emphasized the need for a standardized clinical protocol and a framework to streamline the development of more gene therapies.

 He also spoke to the recent impact COVID-19 has had on gene therapy reviews, reinforcing the agency’s commitment to move forward submissions as fast as possible despite hiring challenges in the current environment.

The importance of large-cap companies

Dr. Hwang noted the importance of large-cap companies, such as Novartis, Roche, Astellas, Pfizer, etc., which have been investing in independent, public gene therapy companies, such as Spark, Audentes, and Avexis in the last few years.

 Holles agreed, saying an acquisition from Astellas was like “rocket fuel” for the company. 

How gene therapy can serve patients’ needs

While many patients remain hopeful about gene therapy, Debra Miller offered a compelling patient perspective. She wants the industry to consider those who may not qualify for gene therapy clinical trials or treatments due to their age or possessing existing antibodies.

 Other panelists agreed we must look beyond gene therapy and the typical adeno-associated virus (AAV) vector delivery methods. What other methods exist, or can we create, to properly serve patient needs? 

Overcome manufacturing issues early

When considering delivery methods, companies must also consider manufacturing capabilities and the need to develop manufacturing experience early on in the process. As Miller noted, delivery is important especially in Duchenne Muscular Dystrophy as corrected genes need to be delivered to muscles all over the body, so this requires a lot of vectors to be produced.

 Overcoming manufacturing issues early is critical in establishing safety and effectiveness, both Dr. Marks and Dr. High confirmed. And most importantly, clinical success fuels better investment. 

What can the gene therapy industry learn right now?

Holles said the gene therapy industry should see the lack of diversity as an opportunity to bridge the opportunity gap and broaden the type of people recruited within the biotechnology and gene therapy industry.

 “This industry has always thrived on doing the right thing and I think we’ve got a real opportunity imposed upon us.” 

– Rose Ramseth

It's important to think about the last step, said Dr. Tsu.

We have to start thinking now about how the vaccine is going to roll out.

How is Moderna managing scaling up?

Bancel said the company did not have the budget to go so fast at this scale at the start of 2020. To build capacity, you need space, equipment, raw materials, and people.

Moderna partnered with Lonza to build capacity to produce 1 billion doses per year.The team is working on scaling up process at the same time – how much can you do in one week? They are also investing in raw materials. 

He's the most worried about raw materials, in particular, scaling up raw materials.

Raw materials matter

There is potential for a scramble and pressure on raw materials, said Dr. Hatchett – for example, there's a global glass shortage. Globally, we're not going to have oversupply of vaccine," he said. That supply could be further constrained by raw materials shortages.

"If we're going to have vaccine as quickly as possible," we need to invest in this, said Dr. Hatchett.

If you could move vaccines up by even two weeks, we'll have a positive financial impact, explained Berkley. This is why we need to invest in manufacturing now.

"The kitchen sink"

The vaccine industry is more or less running at capacity pre-COVID, said Merck's Dr. Markels. 

"What are we doing? It's the kitchen sink," he said. We're doing whatever we can to get more capacity and scale manufacturing.

You're building a factory based on what you think the vaccine is going to be, he said. But you don't quite know yet.

In the end, there probably will be unused capacity, but investing in a portfolio approach makes sense.

"It would be impossible to overinvest if ultimately you were able to support a successful candidate, or two, or three."

So, give us an update on what you're doing.

Novavax's spike protein vaccine is showing "a very strong immune response." It's a platform based on a flu product. The Phase I trial began in May, and results should come in July. 

Sanofi is working on two vaccines: one based on recombinant protein expression, and the other based on an mRNA program. In addition, they are working with GSK on adjuvants. Sanofi's also working on an anti-inflammatory drug; it doesn't appear to be effective in earlier phases, but may be effective in the later stage. 

Johnson & Johnson, which has experience in vaccines against Zika, Ebola, and HIV,  has a vaccine ready for animal testing, with human trials expected later this year. They're also working on extensive manufacturing upscaling.

On the federal government side, FDA is expediting development and availability of a vaccine. "It's a once-in-a-lifetime event," in terms of the speed at which everyone is trying to work to stave off a second wave. 

"Everyone will need to work together," said Dr. Marks. "This is really a global issue where we're all connected." Until it's wiped out, we'll all have issues.

What does global collaboration look like?

"Everybody recognizes that as long as this virus persists anywhere in the world, we all remain vulnerable," said Dr. Hamburg.

"Science is a global enterprise and we need to bring together the best and brightest minds, wherever they are, across borders and across sectors."

Collaboration with companies and regulators is critical to make a difference, said Dr. Nabel.

How can we ensure we have adequately sized, well-designed trials against an unpredictable virus?

We're confronted with challenges in terms of where, when, and the numbers, said Dr. Stoffels. We need between 30,000-100,000 people to get to a clear answer on efficacy.

We need collaboration between industry and government on a global basis to get these vaccines tested, he continued.

Perceptions that we have moved too quickly will make us vulnerable to anti-vaccine rhetoric, said Dr. Hamburg. This is why good trials matter.

Don't forget the supply chain!

A lot of these products will use a lot of the same essential ingredients that have never needed to be scaled in this way before, said Dr. Nabel.

– Samantha Sault

“I’m very heartened by the fact that the industry has stepped to the plate—very much differently than what we saw with SARS.”

“The industry is not stupid—they figured it out!”

He expects “more than one winner in the vaccine field, because we will need vaccines for the entire world—billions and billions of doses.” The same with therapeutics—multiple different kinds of therapeutics, from direct antivirals to medicines that treat hyper-inflammatory response.

The federal government has partnered with biopharmas to “de-risk” involvement in manufacturing the doses before completion of trials, financing production of doses so it’s ready. They’re also giving companies access to clinical trial sites built in the past.

We've never seen this extent of collaboration between industry and federal government, he added.

What's the latest knowledge on how COVID attacks the human body?

We're still learning about that, he said.

"I thought that HIV was a complicated disease," he continued. "It's really simple compared to what's going on with COVID-19." There's a huge range in terms of the severity of coronavirus symptoms.

"We're at almost the beginning of understanding" COVID-19, especially the long-term effects. We don't have any idea what will happen to people who recover, because we've only been studying this disease for four months.

The African American population "is getting hit with a double whammy"

Dr. Fauci talked about why COVID-19 disproportionately affects African Americans. COVID-19 shines "a bright light" on health disparities in the United States. 

Watch him talk about why COVID-19 disproportionately affects African Americans, and what we can do about it:

So, how does Dr. Fauci relax?

"The one thing I've made sure I do is to get some sleep." 

"I unwind mostly by exercise," he added. He's been running with a mask on!

Last question: What was his wife's reaction to having Brad Pitt play him on Saturday Night Live?

"If you start taking that kind of stuff seriously...you're really in trouble," he laughed.

"I'm not as good looking as Brad Pitt and no matter what you do to me, I'm never going to be as good looking as Brad Pitt."

Now there's one thing we disagree with Dr. Fauci on...

– Samantha Sault

The ER nurse turned COVID patient

An ER nurse for 21 years and a COVID-19 survivor, Meghan Harpole talked about how her experience as a nurse helped her advocate for herself when she was a patient and also for her son when showed symptoms of the virus.

After 20 days of symptoms, she received treatment at the hospital where she works.

“The saddest part about it was I was just so happy to be around people because I hadn’t been around people in 20 days—to talk to people who touched me.” The personal connection helped her feel safe, and relieved some of the stress and anxiety she felt while she had the virus.

While she was able to advocate for herself as a patient, she worries other COVID-19 patients may not be able to speak up as much as she did, given that most COVID-19 patients are alone in hospitals.

Meghan Harpole speaks about her ability to advocate for herself when she had #COVID19. #BIODigital #Healthcare pic.twitter.com/Nhxj2cLUPd

— I Am Biotech (@IAmBiotech) June 9, 2020

Why doctors need to listen to patients

Ashley Valentine’s brother suffers from sickle cell disease. “With chronic conditions, invisible pain is really subject to whether that provider believes you. Or not.”

Patients need to educate themselves, too: “You have to know more about the diseases than the providers, than the insurance companies, than the legislators. You, ultimately, are the expert of your disease.”

“You are the expert of your disease,” Ashley Valentine of @SickCells talking about #patient advocacy at #BIODigital. #Healthcare pic.twitter.com/EWr4VnIM35

— I Am Biotech (@IAmBiotech) June 9, 2020

– Nicole Chardavoyne

It is through dialogue and collaboration, particularly leveraging public-private partnership, that we will be best prepared for the second-wave of this and future pandemics.

Despite the varying healthcare systems around the world, all systems are being tested and forced to adapt. It's a bit of a health care revolution.

Australian Health Minister Greg Hunt noted that this urgent health crisis also pushed world governments to move and act quickly in a way that didn’t seem necessarily possible. 

It makes us wonder why our governments don’t take such quick action on everything!

Israel’s Innovation Authority & Chief Scientist of the Ministry of Economy and Industry shared how the pandemic has taken its toll on tech in his country, including making investors far more reserved.

But just because some investors are zipping up their wallets, doesn’t mean there’s no hope. As we’ve seen all over, evolutions in telehealth, AI, and data technologies are critical to improving access for patients.

Amgen's Christophe Bourdon said that the industry is committed to investing right now because we know a second wave is coming.

“We know it’s going to come and we cannot be taken by surprise,” so that means continuing with research, testing, and leveraging private-public partnerships.

Ultimately, this pandemic is an opportunity to share what science is about with the public. Fostering science at the grassroots level will positively shape this ecosystem and shape outcomes for patients. There will be disappointments along the way, but given recent efforts, we are encouraged about the future.

This session was sponsored by: Amgen

- Kara Nelson and Cornelia Poku

The COVID-19 pipeline

The COVID-19 pipeline is strong, with 550 drug programs in 20 weeks. Visit www.bio.org/covidpipelinetracker to learn more. 

The US has over 50% of total COVID-19 drug programs (280) followed by China (39) and Switzerland (26). Of these, 60% are pre-clinical/discovery phase—basically, just a press release with no evidence.

Amazingly, however, there are 216 clinical-stage compounds (40%).

We’re seeing a lot of repurposing: 93% of therapeutics and 80% of antivirals/antibodies are repurposed, but just 3% of vaccines are repurposed.

“In terms of the number of shots on goal, this bodes very well…for success in getting something to the market,” said Thomas.

Check out the full report here.

Want more data? Visit www.bio.org/iareports for all BIO Industry Analysis reports.

It's Day 2 of BIO Digital. Here's what we're covering today:

11 AM ET/8 AM PT – State of Emerging Therapeutic Company Investment, Deal-Making, Pipeline

11:30 AM ET/8:30 AM PT - International Plenary: How Are Global Leaders Collaborating to Combat the Pandemic? (Sponsored by Amgen)

1 PM ET/10 AM PT – Understanding the Voices of Patients: Unique Perspectives on Healthcare

2 PM ET/11 AM PT - LIVE PLENARY: Winning the War on COVID through Vaccines feat. Dr. Anthony Fauci (Sponsored by Johnson & Johnson)

4:30 PM ET/1:30 PM PT - Realizing the Promise of Gene Therapies for Patients Around the World (Sponsored by Pfizer Inc.)

4:30 PM ET/1:30 PM PT - Realizing the Promise of Gene Therapies for Patients Around the World (Sponsored by Pfizer Inc.)

5:45 PM ET/2:45 PM PT - Fireside Chat with the Hon. Jim Greenwood and Dr. Michelle McMurry-Heath

6:30 PM ET/3:30 PM PT - Advancing Rare Disease Diagnosis and Drug Development Through Partnership (Sponsored by Jazz Pharmaceuticals)

7:30 PM ET/4:30 PM PT - Fireside Chat with Dave Ricks, Chairman and CEO of Eli Lilly (Sponsored by Eli Lilly)

7:30 PM ET/4:30 PM PT - How an Innovation Ecosystem Enables Growth During Times of Crisis and Beyond (Sponsored by Shonan iPark)