Live Blog

Moderated by Phyllis Arthur, BIO's VP of Infectious Diseases and Diagnostics Policy, this was an illuminating conversation between the people representing each facet and phase of a global pandemic and where their minds are when we talk about future strategies to protect the global population. 

Panelists:

• Julie Gerberding, MD, MPH, Executive Vice President and Chief Patient Officer, Strategic Communications, Global Public Policy, Merck
• Simba Gill, President and CEO at Evelo; and Venture Partner at Flagship Pioneering
• Otello Stampacchia, Managing Director, Omega Funds
• Ethan Weiss, Cardiologist and Associate Professor, UCSF

“This is the beginning of a new normal for us,” said Merck's Julie Gerberding. “This is not the once in a 100 years pandemic. We’re likely to see emergence of important, potentially global threats because of the nature of our world.” 

The speakers agreed that while it’s great we detected this outbreak very quickly, the containment efforts were reactive. Containment can flatten the curve and slow down spread, but there was a tremendous burden on health systems around the world and inadequate planning--so we’ve got to solve that gap.

Simba Gill and Otello Stampacchia spoke about the ways companies rapidly partnering have moved forward solutions. 

Simba Gill captured some of the great work to bring therapeutics to the various stages of addressing and treating COVID-19, including treating the virus, handling the virus, and treating the consequences of the infection.

“We need to shift from just in time to just in case,” Otello Stampacchia advised the audience as a motto for moving forward and better preparing.

Ethan Weiss pointed to San Francisco/Bay Area as an example of what happens when a community can contain, but not strategize. In the beginning, leaders convinced people to take the disease seriously and got people to buy into the commitment that if everyone hunkered down it would be short-lived, but recently trust has started to erode. Folks are wondering, “Why are bars and gyms open, but schools and playgrounds aren’t?” 

The group further discussed how this is an example of the difference early investment can make. Being able to provide resources, a national strategy, and ultimately prevention is the goal.    

The overwhelming consensus from experts on a market outlook panel: the biotech sector is on solid ground.

Sara Michelmore, Senior Vice President of MacDougall Biomedical Communications, led a discussion with:

• Janice Bourque, MBA, Managing Director, Hercules Capital
• James Healy, PHD, MD, General Partner, Sofinnova Ventures, Inc.
• David Lederman, Director, Equity Capital Markets, Chardan Capital Markets
• Jordan Saxe, Senior Managing Director, Nasdaq

Despite challenges in the rest of the economy, 2020 has been the strongest on record for biotech—record IPOs, record follow-on offerings (or FPOs), significant uptick in private investment, significant new fund formation, and strong capital formation.

In fact, IPOs alone have already raised $12 billion—and we still have a few months to go.

Nasdaq’s Jordan Saxe said the strong momentum will carry the sector well into 2021, with an expected 10 more IPOs by the end of the year on top of the 60 which have already occurred.

What is driving the success?

It’s not just about COVID—though they agreed that the pandemic has made biotech popular, sparking interest from traditional investors but also from “generalists” who do not typically invest in this space.

Other success factors include the way the IPOs are priced and the fact that these emerging companies are delivering. As Chardan’s David Lederman noted, “It helps that all the deals are pricing right, trading well. Companies are delivering on what they said they were going to do.”

Janice Bourque of Hercules Capital pointed to a maturity on the part of emerging biotechs: “What we’re seeing on both the private and public side is that these companies are  getting the funding that they need and getting it well in advance of some of the milestones they are looking to hit, so that they have runway to be able to get through.”

The risks of a rosy outlook

There are more generalists coming into the market because of increased interest due to COVID, but as James Healy of Sofinnova Ventures pointed out, “These investors may not know the risk they are underwriting.”

“I’m not sure everybody appreciates how long and how challenging it can be when companies are still pre-clinical,” agreed Bourque.

Another problem: the political environment, including the undermining of the public trust of the FDA, CDC, and NIH as well as the election that could cause apprehension in the marketplace since the marketplace likes certainty.

The investor's perspective: “I’ve been really concerned about the undermining of the public’s trust in @US_FDA, @CDCgov, @NIH,” says Janice Bourque of Hercules Capital #BIFDigitalpic.twitter.com/vAxORs978j

— I Am Biotech (@IAmBiotech) October 14, 2020

Dr. Cartier Esham, BIO’s EVP, ECS and SVP of Science and Regulatory Policy, spoke with Dr. Patrizia Cavazzoni, Acting Director of the Center for Drug Evaluation and Research (CDER) at the U.S. Food and Drug Administration (FDA).

So, how is FDA managing during the pandemic?

Following a period of adaptation, FDA has reached “a steady state when it comes to reacting to the virtual environment,” said Dr. Cavazzoni. The agency is leveraging virtual platforms to conduct meetings with sponsors.

When it comes to inspections, FDA has made “substantial adaptations,” including using information shared by trusted regulatory partners, requesting records directly from facilities in lieu of on-site drug inspections, and actively exploring alternatives for remote/live interactions.

“The new normal”

One benefit could more opportunities to meet with international regulatory partners with less travel.

“A positive outcome of this very challenging and painful experience for the world is that we may find ways to be more connected with less effort and more efficiently, which will benefit everyone’s productivity,” said Dr. Cavazzoni.

Operation Warp Speed

FDA/CDER regulates the development of therapeutics with “very clear delineation between Operation Warp Speed activities and FDA regulatory decision-making,” akin to a “firewall." FDA is interacting with OWS as they would with a co-developer of therapeutics.

How are we ensuring that these treatments being developed so quickly are safe and effective?

"The distinction between approval and Emergency Use Authorization is very important – and I might say not easily understood."

Approval of a drug product by FDA requires “substantial evidence of effectiveness” with results from "adequate and well-controlled trials,” said Dr. Cavazzoni. “This substantial evidence standard underpins the decision for approval.”

FDA has a “robust and well-proven system” for monitoring post-market safety of drugs, including through the Sentinel System and using reviews of adverse event reports.

Emergency Use Authorizations (EUAs) “strengthen our response to a public health emergency" if the product meets certain criteria, she said. The criteria includes that the product shows it may be effective and the known and potential benefits must outweigh the known and potential risks. FDA must establish conditions for emergency use to protect public health including spelling out appropriate conditions for monitoring and reporting adverse events.

What will we bring with us to the other side of this experience?

In the long term, Dr. Cavazzoni hopes the use of digital technologies and decentralized trials will help facilitate clinical trial enrollment and retention, while “optimizing or promoting clinical trial diversity.” One challenge to clinical trial diversity has been limitations on having clinical trial sites in areas where they can reach diverse populations.

“What has been really tremendous to see is how much the stakeholders across the world – industry, academia, regulatory agencies, international regulatory partners – have collaborated and are continuing to collaborate in a way that in decades [in academia and the public and private sector] I have never seen before," concluded Dr. Cavazzoni.

"And that is what is going to get us out of this really difficult situation as a nation and as a world: rallying around this problem and all working together to solve it."

The coronavirus is making life hard. It’s making clinical trials harder.

With more than 75% of clinical trials being disrupted by the virus, companies have pivoted to e-clinical tools and technologies for decentralized clinical trials. But there are questions regarding the feasibility of these trials and how they affect the data, regulatory efforts, and patients.

To share perspectives and answer these questions, Karen Carey, News Analyst at BioWorld Today, moderated a panel with:

• David Coman, CEO, Science 37
• Bill Gerhart, CEO, Respivant Sciences, Inc.
• Adam Goulburn, Partner, Lux Capital
• Rasika Kalamegham, Head, US Regulatory Policy, Genentech, A Member of the Roche Group
• Michelle Longmire, CEO, Medable
• Andrew Schachter, CEO / Founder, Axiom Real-Time Metrics

Companies have gone to extreme lengths to bring the trial to the patient. Respivant Sciences used mobile nursing to bring resources to patients’ homes, communicating through screen porches or windows, explained Gerhart.

Genentech jumped through hoops to get medicine to a patient stuck vacationing in Brazil so no dose was missed, said Kalamegham.

If you have a solution for one patient, you have a solution for one patient. Making sure the patient’s needs are met is the number one priority, says @Bill_Gerhart of @respivant#BIFDigital

— I Am Biotech (@IAmBiotech) October 14, 2020

Technology can help overcome some of the barriers companies are facing. It’s important to think about all aspects of the trial, said Coman, from a central hub to operate to a workflow to execute.

And being able to use technology throughout the patient journey is critical, added Longmire.

Investments in digital health businesses have risen significantly this year, said Goulburn. Biotech is a large industry spending money in clinical trials, creating innovation to problems that need solutions, and technology is the main driver.

But at the end of the day, decentralized trials are about serving the patients’ needs.

The patient is the focus in decentralized clinical trials during #COVID and beyond, says Rasika Kalamegham of @genentech. #BIFDigitalpic.twitter.com/xkZg6VZaxv

— I Am Biotech (@IAmBiotech) October 14, 2020

“There’s a disparity between the U.S. population and what it looks like for the pipeline. That disparity is increased when you look at who works in this industry and then it’s increased again when you look at venture,” said Brady Huggett, Senior Editor at Nature Publishing Group.

He moderated this expert panel featuring:

• Susan Angele, Senior Advisor, KPMG LLP
• Dr. Grace Colon, President & CEO, InCarda Therapeutics Inc.
• Jacqueline Grant, Principal, Abingworth
• Sonia Gupta, Managing Director, Goldman Sachs & Co.
• Andrew Schwab, Founder and Managing Partner, 5AM Ventures

How can we attract good, diverse candidates?

Panelists agreed on the need to reach out to people at a young age, even as young as high school.

“We need to think about this biotech ecosystem as a true ecosystem,” said Goldman Sachs’ Sonia Gupta. “It’s our responsibility as leaders in the field to ensure that we are promoting diversity from the highest levels across all of those segments of the ecosystem in order to bring people in and train them.”

“It’s on the onus of us as large influential institutions to set goals,” she continued. Large companies need to use their resources to find and nurture that talent.

Small companies can do this, too, said InCarda Therapeutics' Dr. Grace Colon. In addition to hiring diverse candidates, developing an "atmosphere of inclusivity" is important, too.

"What matters more than the size of the company is whether people at the top view it as a priority," added KPMG's Susan Angele.

Networks matter.

To improve the diversity of boards, you have to "make sure venture capital firms aren't all white men," said 5AM Ventures' Andrew Schwab.

Abingworth's Jackie Grant discussed further:

Our U.S. election outlook panel moderated by Bloomberg Intelligence's Brian Rye continued with a discussion on drug pricing policy, which everyone brought up in their comments.

Mike Mattoon, BIO’s VP of Federal Government Relations, said President Trump’s “most-favored nation” drug pricing scheme is similar to H.R. 3, House Democrats’ drug pricing bill, which the president’s own economic advisers said would lead to fewer drugs due to less investment in R&D.

“Basing pricing and reimbursement on, in some cases, the lowest price paid in certain countries” could lead to what Mattoon called a “downward spiral” as it would create added risk for investors trying to fund research that’s already risky.

We need to limit out-of-pocket costs and incentivize patients to take the right drugs that they need, said GlycoMimetics' Rachel King.

“Price controls don’t work to incentivize innovation,” she continued:

The issue of drug pricing for COVID-19 medicines will be a big issue in a Republican or Democratic government, added FTI Consulting's Sonja Nesbit.

We have the data on how many new biotech companies are formed in Europe vs. the U.S., said Samara BioCapital’s Dr. Srinivas Akkaraju. Europe has great science, but it doesn’t translate into new drugs and new companies due to price controls.

“I do worry if we take a draconian stance on drug pricing, it will have a chilling effect on innovation,” he continued.

Whatever happens with drug pricing or the Affordable Care Act or anything else…

We have to make sure “vaccines and therapies are distributed equitably so that we don’t have communities that are being left out in the cold,” said Mattoon.

The job of distributing the vaccine and therapies for COVID will be falling upon the next administration. These will be “massive issues” and “the pressure will be to find money for those,” he continued.

With 20 days to go until Election Day, we brought together a panel of experts to talk about what’s ahead for health care policy – and make their predictions.

Moderated by Brian Rye, Senior Health Care Policy Analyst at Bloomberg Intelligence, the panel featured:

• Dr. Srinivas Akkaraju, Founder and Managing General Partner at Samsara BioCapital
• Justin Field, SVP, Government Affairs at the National Venture Capital Association (NVCA)
• Rachel King, Founder & CEO of GlycoMimetics, Inc.
• Mike Mattoon, BIO's Vice President of Federal Government Relations
• Sonja Nesbit, Managing Director Government Affairs at FTI Consulting, Inc.

As we learned in 2016, anything can happen, said BIO’s Mike Mattoon—and we may not know the outcome for a few days or weeks after November 3.

While it’s not a done deal, right now, he thinks it’s increasingly likely that Democrats will win the White House, regain the Senate, and retain control of the House.

Want a deep dive into the election? Check out the Biotech Votes 2020 Election Preview Report.

How does the election affect the industry’s policy priorities?

“There is a broad recognition that our industry is literally stepping up to save the world, so how that dynamic plays into our traditional parochial battles around drug pricing and other things I think is really yet to be seen," said Mattoon.

GlycoMimetics’ Rachel King highlights two concerns from the company perspective:

1. Maintaining public confidence in the safety and efficacy of our products. We’ve always been able to count on the U.S. Food and Drug Administration’s “stringent, science-based, excellent review” as the gold standard, but now we’re seeing “politicization of vaccine programs.”
2. Ensuring affordable access to our products, particularly ensuring politics doesn’t “undermine investment.”

The drug pricing conversation could “be further heightened…once when we get to a vaccine,” said FTI Consulting’s Sonja Nesbit.

The investor perspective

Despite all the uncertainty, we’ve seen an “influx of deal activity," said Samsara BioCapital’s Dr. Srinivas Akkaraju.  

A Biden administration could reduce uncertainty but “add some pressures on the health side,” he continued. “Every time there’s some potential pressure on drug pricing negotiations, that had a real impact on valuations in biotech and it does cause a little bit of slowing.” While he thinks we’ll have to “grapple with drug pricing and what it means for valuations,” he sees a Biden administration as “a net-positive” for the industry, especially because of what it will mean for policy regarding science.

NVCA’s Justin Field says immigration is a big challenge with the Trump administration, but the capital gains tax policy and “how that impacts decisions to invest” is a concern about the Biden administration.

Brad Loncar is an investor who believes that some of the most exciting advances in the coming decades will be in biology and biotech—and investing in the advancement of medicine will allow more people to participate in that. 

“To be able to invest in something that helps people is one of the most rewarding things that you can do,” he says.

Watch Brad tell his story:

Visit www.bio.org/iambio to learn more and share your story!

Here's what we'll be covering today:

12:00 PM ET/9:00 AM PT - U.S. Election Outlook: Presidential & Congressional Scenario Planning for Biopharma Priorities

1:00 PM ET/10:00 AM PT - Achieving Corporate Diversity & Inclusion Goals through Investment & Policy Choices

2:00 PM ET/11:00 AM PT - Conducting Remote Clinical Trials Productively During a Pandemic

4:00 PM ET/1:00 PM PT - Fireside Chat: Patrizia Cavazzoni, MD, Deputy Director for Operations, Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA)

5:00 PM ET/2:00 PM PT - Market Outlook: Solid Ground or Thin Ice?

7:00 PM ET/4:00 PM PT - Treating Infectious Diseases: Lessons from COVID-19 and Avoiding Future Pandemics