Pathfinders Podcast

Biopharma Investors See Opportunities in Targeted Treatments and Gene Editing

Personalized, targeted drug therapies and novel gene editing techniques are helping researchers and biopharmas to deliver to an expanding healthcare market.

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Brian Abrahams, Gregory Renza and Luca Issi
Hosted by Joseph Coletti
Published June 3, 2024 | 5 min read
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Key Points

  • Targeted drug therapies for subtypes of disease are transformational in the oncology and cardiology spaces.
  • Gene therapy is moving more quickly from identifying root causes of disease to treating disease with mRNA, SIRNA and CRISPR cas9 techniques.
  • The addressable market for novel therapies is growing, particularly in immunological and allergic diseases from lupus to COPD.

Personalized, targeted drugs and gene therapies are still at the forefront of medical innovation, as we discovered at RBC Capital Markets annual Doctor Days meetings. These insightful forums bring together key opinion leader clinicians and researchers from various therapeutic fields with institutional investors to discuss where opportunities lie, the next big breakthroughs and the most novel drug therapies. Across areas like cancer, cardiology, immunology and more, research is narrowing its focus, looking to identify key markers for different subtypes of illness to target them more effectively and uncovering genetic roots and genetic fixes for disease.

Targeted therapies for cancer and cardiology

For some time, what’s hit the headlines in cancer research has been the more precise and personalized approaches to treating an array of cancers. Gregory Renza, senior biotechnology analyst at RBC Capital Markets, said that some of the key therapeutic modalities that have dominated the headlines this year are antibody drug conjugates and targeted radio pharmaceutical therapies.

“Radioligand therapy was [also] indicated as a significant area of interest,” he said. “And this is driven by improved real world experiences, such as with Pluvicto. And that in turn drives treatment centers and patients to want to participate in more clinical trials.

“I think biopharma is matching this enthusiasm that we are seeing and hearing on the front lines. The strategic activities over the last year on radioligand therapies have been unprecedented, and also rather validating. Acquisitions, but also some key collaborations. So these are companies working together, in addition to larger players just simply beefing up their resources. And we believe this can only accelerate that ability to reach patients.”

“The strategic activities over the last year on radioligand therapies have been unprecedented, and also rather validating.”

Gregory Renza, M.D., Senior Biotechnology Analyst

Fellow senior biotechnology analyst Luca Issi sees something similar happening in cardiology, where research is beginning to identify subtypes of cardiac disease to target.

“We've seen a lot of success in oncology because there are much better approaches to slicing and dicing patient populations and finding the right therapy for various cancer subtypes. I think cardiology is the new oncology because we now have much more sophistication in really slicing and dicing patient populations across that space, and TTR cardiomyopathy (TTR-CM) is the perfect example of this,” he said.

TTR-CM refers to a heart condition caused by abnormal protein deposits in the heart muscle and the new innovations to tackle this specific protein.

“There is a current therapy from Pfizer that generates $4 billion in revenues, and has been transformational for some patients, but now we have a lot more coming down the pike. There is this phase three trial upcoming from Alnylam. This is a highly anticipated trial that may show us that a new class of therapy called silencer therapies, which actually turn off this protein, may make an impact for patients.”

Genetics moves from root causes to cures

Gene therapy is, of course, the ultimate targeted therapy, but only if the genes behind diseases can be found and then altered. For a long time, genetic research was uncovering the root causes of disease, but doctors still lacked the tools to actually do anything about those causes. Today, Issi explained, if a disease is caused by a lack of a gene, you can actually deliver that gene with either mRNA or gene therapy. To turn off a gene, you can use siRNA or ASO (antisense oligonucleotide), and you can edit genomes using gene editing and CRISPR cas9.

“One target in cardiology that was highlighted was LP(a) (LP little a). LP(a) could basically be the next cholesterol, just as we talk about cholesterol today, maybe in the future, we're going to talk about LP(a). There are a lot of companies developing therapies for LP(a), they're trying to knock down LP(a), essentially testing the hypothesis that knocking down LP(a) can actually drive a cardiovascular benefit for patients,” he added.

However, in some cases, investors are cautious on gene therapies when existing treatments allow patients to be relatively well managed. In wet AMD (age-related macular degeneration), a subtype of the more common AMD eye disease, the current standard of care is relatively high, so investors are more hesitant. But on the medical side, the researchers are pretty excited.

“This has the potential of a one-and-done approach, this is as close as it gets to a potentially functional cure.”

Luca Issi, PhD, Senior Biotechnology Research Analyst

“This has the potential of a one-and-done approach,” said Issi, “this is as close as it gets to a potentially functional cure.”

A sizeable addressable market

Brian Abrahams, head of biotechnology research, notes that 14 million people in the U.S. suffer from COPD, over 6.5% of the adult population. But treatments really haven't changed in years, patients are still primarily treated with bronchodilators and steroids.

“[Now] doctors are really excited about some cutting edge development in the space, a different approach altogether, using biologics to tamp down some of the inflammatory pathways underlying the disease in certain patients. It's a concept that's really transformed the asthma treatment space, but hasn't really been used before in COPD,” he said.

“Doctors are really excited about using biologics to tamp down some of the inflammatory pathways underlying COPD in certain patients.”

Brian Abrahams, M.D., Head of Global Healthcare Research

As immunological and allergic ailments increase in the U.S., the potential returns for investors with novel treatments does too. Renza sees meaningful opportunities in lupus, autoimmune kidney disease and cytopenia, where over an estimated $165 billion in sales is potentially achievable. In allergy and dermatology, and other key inflammation and immunology area indications like chronic urticaria, asthma, and COPD (chronic obstructive pulmonary disease), RBC estimates about five million patients have an addressable market and a market potential of over $100 billion.

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