The lessons learned from the global pandemic are expected to translate to existing and emerging therapeutic areas - particularly oncology; more efficient regulatory-industry relationships; mRNA is a word we will continue to hear a lot about; and home health care is here to stay.
When our backs were against the wall, the biotech/biopharma community came together to create not one, but two vaccines currently approved for emergency use in the U.S. This has earned the industry a lot of respect. The lessons learned from the global pandemic are expected to translate to existing and emerging therapeutic areas - particularly oncology; more efficient regulatory-industry relationships; mRNA is a word we will continue to hear a lot about; and home health care is here to stay. These are just a few of the insights BioSpace discovered when we spoke with 12 executives from the biotech/biopharma space. Read on for more.
Deploying the Triangle Offense Against Cancer
NantWorks and ImmunityBio Chief Executive Officer, Patrick Soon-Shiong, believes that science has reached the point where it is ready to pit a “triangle offense” against even the toughest cancers. The South-African-American transplant surgeon, bioscientist and minority owner of the L.A. Lakers, has aligned his network of healthcare, biotech and artificial intelligence startups, NantHealth, to harness the power of the immune system and the natural killer cell to treat even the toughest cancers.
Soon-Shiong laid out the history of oncology. Then, he identified the problem:
“Really, what the tumor has done is to figure out a way to hide from the immune system, and all of our therapy has actually been helping that tumor to hide from the immune system. Whether it be high-dose chemotherapy or high dose radiation. So, from a 10 000 foot view is the realization that ‘hey, wait a minute. Maybe we need to treat the host rather than the cell or the disease. Maybe we need to modulate the host.”
Along these lines, Soon-Shiong explained that the next five years will be “an orchestration. They [T cells, B cells, NK cells] all act together in what I call the tumor microenvironment. We’ve never had the tools before to interrogate a tumor in that way. That’s the next thing you’ll be seeing. For the first time, we have the tools that can deconstruct in real-time, not the tumor itself, but the immune system surrounding the tumor.”
“[It’s] the realization that it’s not just one, but it’s a complex orchestration of the natural killer cells. I call that the triangle offense. The triangle offense is really the T cell, NK cell, and the macrophage.”
NK Cells Locked, Loaded, and Ready to Fire
“The last year was a very exciting year for cell therapy in oncology. One big thing is that not only do we see T cells work, we are now seeing that natural killer [NK] cells work for cancer treatment as well and that this approach is now coming to the main stage,” said Acepodia Chief Executive Officer, President and co-founder, Dr. Sonny Hsiao Ph.D.
His company is leveraging the novel cellular immunotherapy development platform he invented to develop novel, targeted, allogeneic cell therapies against cancer with NK cells, the body’s immune cells that specialize in recognizing and attacking cancer cells.
In the cell therapy arena, Hsiao told BioSpace that “the biggest challenge we’re trying to address as a company, or as a whole field, is to see if we can also target and kill low-expression tumor antigens on tumor cells so that we can hopefully eliminate the relapsed tumor and provide a cure for these patients.”
“The metastatic tumor will also be a good target, and the type of tumor we’re going after. It’s actually very important because that’s why patients die. Not because of the local tumor. It’s because the tumor metastasized, and those are the types of tumor that cell therapy can be of potential benefit for the patient.”
Moving on From the Amyloid Hypothesis
“I actually think neuroscience is going to heat up in 2021,” said Ben Zeskind, co-founder and CEO of Immuneering, which is using bioinformatics and computational biology to develop new drugs in this space, along with oncology and immuno-oncology.
“There’s obviously been some big challenges, particularly in Alzheimer’s the past couple of years. I think a lot of that is because the vast majority of those challenges can be traced to a single hypothesis, which I think people are ready to move on from – amyloid. I think this year, kind of the last big hope for the Amyloid hypothesis ran into serious challenges, so I think there’s going to be this whole renaissance in Alzheimer’s and neuroscience more generally.”
Referencing Immuneering’s own Alzheimer’s program, Zeskind said, “What we’ve done is to take Alzheimer’s and say: ‘what if this thing we call Alzheimer’s, this collection of memory loss, dementia, accumulation of plaques and tangles, what if it’s actually just several completely different diseases at the molecular level that all result in the same set of symptoms?”
From a wider angle, Zeskind believes that the current moment is a revitalization of the industry on the whole:
“I think it’s an opportunity for our industry. The industry’s kind of been given a second chance in the eyes of the public. I think there was a lot of negative attention on the industry, and I think the success of these vaccines will give the whole industry an opportunity to really have people give a fresh look and see a lot of the amazing work that we do, creating new medicines for patients.”
Are Biomarkers Finally Arriving?
Cerevance Chief Executive Officer and CNS drug discovery veteran, Brad Margus, is excited that biomarkers appear to be coming into their own.
“There are some exciting things happening in biomarkers at last. People have been talking about a biomarker strategy for both development and translational development, for a long time. But, at conferences, people speak on them but they don’t have that much to say,” Margus said. “In the last year or two, finally, we’re getting some biomarkers…especially in brain diseases, where you really need them because you can’t biopsy a tumor.”
Margus also expounded upon the Alzheimer’s disease hypothesis mentioned earlier by Immunering’s Ben Zeskind:
“When you’re looking at the Parkinson’s population, there are five or six different causes in there. Same with Alzheimer’s. People have done all kinds of things in genetics about genes that increase your risk and contribute to you getting Alzheimer’s. But we don’t know if it’s a lot of different diseases, and they all end up with dementia, and with certain things – plaques, tangles in your brain – or if it’s a few things that cause it. So, what you’re desperate for are some markers, that regardless of what’s causing your Alzheimer’s, still cause that protein level to change.”
A Whole New ‘Science Matters’ Perspective
“I think most of what will happen in 2021 is an absolute direct result of the things the world has gone through in 2020. I think it’s taught us three things that are very important that we need to continue to be focused on in the ensuing year. The three things I think of would be: nimble, socially responsible, and real science matters,” said John Temperato, CEO of 9 Meters Biopharma.
“If you had a technology applicable to [COVID-19], you had to do the right thing. So, I think that’s an example of being socially responsible as well. And then, always, ‘science matters.’ I think the days of me-too and being the 20th product or the 30th therapeutic in a given therapeutic category, where you’re making it iteratively better, I think those days are gone. I think the industry has come a long way, especially the last couple of years, where most organizations have moved away from a spec pharma mentality.”
Biotech is Mining for mRNA
“The biggest thing that is really happening in the biotech industry…there is a new rich source of drugs. There is a goldmine, called mRNA, that some diggers have been digging for 30 years, finding a very small part of it,” said Yochi Slonim, co-founder and Chief Executive Officer of Anima Biotech, which is advancing ‘Translation Control Therapeutics,’ the first and only platform for the discovery of small molecule drugs that control mRNA translation.
In a boone for Slonim’s research focus, the year 2020 has made this molecule a household name.
Slonim gave a detailed explanation of the interplay of mRNA, then said,
“Nobody cared, until very recently. Now, you ask every person in the world from the age of 80 down to 8, what is mRNA? They tell you, ‘that’s a drug that saves us from the coronavirus.’ A big thing that is happening is that a rich new source of drugs is coming to the mainstream. Now, it comes to the mainstream in terms of the public understanding that it exists. But it will also come into the mainstream the harder way, which is to actually deliver new drugs that are based on that technology. Regardless of the vaccine, there are two big movements of companies that are setting their destiny to find drugs around mRNA. One is the RNAi guys, the injections, and the other one is companies like Anima, that are trying to do that with small molecules.”
2020 Was Educational for Industry & Regulators
The past year has offered up many lessons for humanity. Vu L. Truong, Founder and Chief Executive Officer of Aridis Pharmaceuticals, believes that the COVID-19 pandemic has been a lesson in efficiency for industry and regulators alike.
“…clearly the regulators, FDA and EMEA this year have gotten a taste of how efficient they can be in terms of approving products, so I see this trend continuing. That is, faster approval of drugs in general. Not just pandemic-related drugs. I think, learning how to work closely with the industry partners to monitor the progress of clinical trials, and getting various regulatory submissions in quickly and efficiently to get drugs that are important, in oncology for example, “said Truong.
“I think that COVID-19 also is going to put a spotlight on infectious diseases more so in 2021. I think there’s the recognition that vaccines can have substantial value, and that hasn’t always been the case. And not just pandemic-related vaccines, just all kinds of vaccines for infectious diseases. But also maybe even in cancer vaccines,” he added.
And who can overlook the rapid transition to telehealth and remote diagnostics?
“The industry has gotten to learn, all the physicians are starting to learn how to be productive with remote diagnostic, remote treatment, and how to make the healthcare delivery process more efficient…I think remote diagnostic and treatment, and telehealth. I think that’s also going to dominate in 2021,” said Vu, whose company is developing AR-711, an inhaled, self-administered, at-home treatment for mild-to-moderate SARS-CoV-2 infections.
Will the Science of Aging Reversal Come into its Own in 2021?
“The biggest risk factor for all these diseases that we want to treat, cancer, heart disease, neurodegenerative disease, the biggest risk factor is aging,” said Dr. Thomas Rando, co-founder of Fountain Therapeutics, a biopharmaceutical company focused on reversing aging to treat disease.
“I think that that will continue to be a real focus. Can we develop…almost a preventative therapeutic approach? There’s been a lot of movement over the last several years in terms of therapeutics targeted for the basic biology of aging, and I think that’s something that has just continued to gain steam increasingly over the last 5-10 years for sure, and even more so over the last few years I think that’s a big shift, and I think it’s moving from the academic world into the biotech and pharma world.”
In the treatment space, Rando believes that the overall viewpoint is changing for “a couple of reasons. Really, a lot of the drugs that we use in clinical practice are actually preventative medicines. So, it’s not that different. I think, number one, they’ve come to recognize that we already do this, disease by disease. Another thing is, there’s been so much investment in biotech in this area, that I think they’re just realizing there’s something here, and no one wants to miss the boat.”
The Grounding of Business Travel
“I believe that the year 2021 is going to be the year of vaccinations,” said Valneva President and CEO, Thomas Lingelbach. He is a 30-year veteran of the vaccine industry.
“I believe that, hopefully, access to vaccines will help us globally tackle what is a large problem with COVID-19 right now, but it will also do something else. It will change the prospect toward vaccination in general. I believe that even the most skeptical people will have understood that from a health economic perspective, that prophylaxis is always cheaper than therapy, and there is added value in investing more in infectious diseases and diseases that could have the potential to come up with the next pandemic threat. I believe that many, many vaccine preventable diseases will be tackled that haven’t been addressed thus far, so I think this will be a shift.”
Lingelbach, currently focused predominantly on traveler’s vaccines with Valneva, had unique insight into the rebound of that sector.
“I believe that the business travel side of things is going to change quite drastically. I don’t think that people, for business, will travel in the way people used to travel in the past. I’m expecting some continuity of new ways of working, interacting in our digital world. I think that we’re going to see probably the single largest shift in the way of working in 2021, once we have gotten back to a new normality.”
The Growing Sense That We’ve Learned Our Lesson
“Anything with infectious disease has been kind of out-of-favor for a long time. And I think new ways to deal with emerging epidemics or variations or mutations of the current epidemic I think will be well perceived by the marketplace. Because it’s not going to stop with COVID,” said Jeff Wolf, Founder and Chief Executive Officer of Heat Biologics, a biopharma company focused on activating and modulating the immune system against cancer and infectious disease.
“I think new approaches that can help us prepare better for the next epidemic and for other infectious diseases, we’ll be well prepared. In dealing with COVID, we’ve learned a lot, how to deal with the infectious disease space, so I think this could be something that really takes off in 2021.”
Wolf added, “I think oncology can make a comeback, with different therapeutic regimens kind of working their way through in oncology. New ways to deal with cancer could be a big thing. New approaches that complement things like checkpoint inhibitors and co-stimulators and other approaches that are out there.”
Returning to Normalcy with Epigenetic Regulation
“I think the obvious one is dealing with the pandemic first. But I do believe that with the vaccines coming forward, that that will be something that we as a society and as a healthcare system are able to get under control. I don’t know whether it’ll be by the middle of the year or by the third quarter, but at some point in time, enough people will be vaccinated that life will start to get back more towards normal,” said Dr. James Brown, President and Chief Executive Officer of DURECT Corporation, a biopharma company developing medicines based on its epigenetic regulator, DUR-928.
Brown explained why he thinks the epigenome, an umbrella for all the chemical modifications to DNA and histone proteins that regulate the expression of genes within the genome, will gain traction.
“As you move toward the future, it’s what we’re doing at DURECT, and it’s about understanding the epigenome and its place in disease,” said Brown. “I think that’s a very important piece because we do have experience in medicine now with the epigenome in trying to help deal with cancer and kill cancer cells effectively by disrupting the epigenome. But to be able to look to regulate it, set it back toward normalcy, that’s a whole different realm.”
The Future of the Cancer Prevention Space
Innovative and unobtrusive tests in the cancer prevention space are much needed, and Israeli-based Check-Cap is developing one in the area of colorectal cancer screening.
“The future of cancer prevention? If you look at the potential solutions that exist in the market, those include structural tests such as colonoscopy, such as capsule endoscopy. The other screening tests are mainly biomarker tests that include FIT…and liquid biopsies. Those are the most popular that are being either developed or existing. I do believe that in order to keep up the momentum, there is a need to identify precancerous polyps because that’s the easiest way to maintain health,” said Check Cap Chief Executive Officer, Alex Ovadia.
Ovadia believes that screening for disease is another area that may be moving toward the home care space:
“I think that another factor where we might have a very big advantage would be the home care solutions. Especially these days, when you look at the coronavirus, I think that people would like to get treated and or diagnosed, and/or screened at home as much as possible.”
