Moderna Breakthrough Puts Immunotherapy Stocks Back in the Spotlight

Biotechnology companies can spend decades building toward a single moment.

Years of research. Hundreds of experiments. Multiple rounds of clinical testing. Relentless regulatory scrutiny. All building toward the point when scientific promise begins to translate into measurable human data.

For Moderna’s novel cancer therapy, Intismeran Autogene, one of those moments arrived on August 19.

In collaboration with Merck, Moderna announced positive Phase 3 results for its experimental melanoma treatment.ⁱ

The market reaction was immediate. Moderna shares surged 177% in a single trading session, adding roughly $45 billion to the company’s market value.ⁱⁱ It was a dramatic reassessment of what the underlying technology could be worth.

Moderna’s treatment uses mRNA technology to train the immune system to recognize mutations that are unique to an individual patient’s cancer.ⁱⁱⁱ

But the personalized nature of the treatment also highlights one of the industry’s biggest challenges:

Scalability.

Like today’s gold-standard CAR-T therapies, Moderna’s treatment is personalized. Each patient’s tumor must be sampled and analyzed to identify its unique mutations before a custom therapy is developed.

The level of precision is extraordinary. But the process can also be highly complex, time-consuming, and expensive.

Take Carvykti, a personalized CAR-T therapy that carries a nearly half-million-dollar price tag and can take more than a month to manufacture.^iv

That’s for a single patient.

And with nearly 21 million people diagnosed with cancer worldwide each year^v, therapies that can be repeated and scaled are increasingly sought after.

GT Biopharma (NASDAQ:GTBP) is taking a fundamentally different approach to this growing challenge.

Rather than manufacturing a new therapy for each patient, GT Biopharma’s TriKE® platform is designed to activate the body’s own Natural Killer cells and direct them to attack cancer cells.

If the approach proves successful in human trials, its potential could extend far beyond a single type of cancer.

A Programmable Engine for Natural Killer Cells

The human immune system already contains one of nature’s most powerful cancer-fighting weapons.

Natural Killer (NK) cells.

These specialized immune cells naturally patrol the body for abnormalities – including cancer cells.

The problem is that cancer has ways of fighting back. As tumors progress, NK cells can become exhausted. Their numbers can decline. Their activity can weaken.

And once that immune response begins to fade, cancer has an opportunity to regain control.

GT Biopharma’s TriKE® platform is designed to change that equation.

Think of it as a three-part bridge.

• One part attaches to an NK cell.
• Another attaches to a specific target on the tumor.
• And between them sits IL-15, an immune-stimulating protein.

The result is a single molecule designed to lock the immune cell and cancer cell together, while providing the necessary fuel to keep NK cells active.

Researchers have already tested the basic concept in humans. GT Biopharma’s first-generation TriKE candidate, GTB-3550, was evaluated in patients with difficult-to-treat acute myeloid leukemia and myelodysplastic syndromes.

The early Phase 1 study offered an important proof of concept.

57% of patients experienced a significant reduction in cancer-cell burden, with bone marrow blast levels falling by as much as 63.7%.^vi

Those results provided early evidence that the TriKE approach could indeed produce a biological response in humans.

But rather than simply advancing the original design, GT Biopharma took what it learned from the first-generation molecule and used it to build a more advanced version from the ground up.

Second Generation - Up to 40X More Potent

The key to TriKE’s redesign came from an unlikely source:

Camels.

More precisely, camelid nanobodies – single-domain antibody fragments derived from llamas and camels. Their size and structure gave researchers greater flexibility in engineering the upgraded platform.

In head-to-head preclinical testing, the difference was significant.

GT Biopharma’s second-generation TriKE molecules demonstrated roughly 10–40× greater potency than the first generation.^vii

Now that technology is being tested in humans. The first candidate, GTB-3650, is designed to target CD33, a marker found on certain blood cancers.

It’s currently advancing through a Phase 1 trial in patients with relapsed or refractory acute myeloid leukemia and myelodysplastic syndromes. The first four cohorts have been enrolled, and clinical updates are expected as the trial progresses.

GT Biopharma is also evaluating a second TriKE therapeutic candidate targeting solid tumors, GTB-5550, which entered Phase 1 testing in May 2026. GTB-5550 targets B7-H3, a protein expressed across a wide range of difficult-to-treat solid tumor cancers, including:

The same underlying TriKE architecture is now being tested against two very different types of cancer.

That’s where the economics of a platform can begin to look very different when compared to a single drug.

The Billion-Dollar Blueprint

One successful drug can create enormous value. But a platform capable of producing multiple drugs can change the equation entirely.

That distinction has helped drive some of the industry’s biggest acquisitions.

• Gilead Sciences paid $11.9 billion for Kite Pharma and its CAR-T cell therapy platform.^viii
• Celgene acquired Juno Therapeutics for approximately $9 billion, gaining a pipeline of engineered immunotherapies.^ix
• And in one of the largest oncology transactions in history, Pfizer paid $43 billion for Seagen and its antibody-drug conjugate technology.^x

Different technologies and different cancers – but each demonstrating the value of a platform capable of supporting multiple therapies.

GT Biopharma is still a long way from that kind of validation. Its current candidates remain in early-stage clinical development, and their safety and efficacy have not been established.

That’s what makes the next stage so important. Meaningful results could help validate TriKE as a broad therapeutic platform.

And now, GT Biopharma is integrating another technology that could accelerate how quickly that platform produces its next set of candidates.

How AI Could Change the Economics of Drug Discovery

Artificial intelligence has already transformed countless industries. Now it’s being used in one of the most expensive and failure-prone businesses in the world – drug discovery.

Developing a new medicine requires an enormous number of decisions long before a drug ever reaches a patient.

Which molecular design binds effectively? Which structure is most stable? Which candidate can be manufactured?

Answering those questions has required enormous amounts of laboratory work and rounds of experimentation. A drug development process that can take years, cost billions, and still end in failure.

Instead of testing every possible design, researchers can now use computational tools to analyze molecular sequences and structures, identify potential weaknesses, and prioritize the candidates that appear most promising before committing time and capital.

The goal isn’t to eliminate laboratory testing, but to make better decisions about what reaches the laboratory in the first place.

For an early-stage biotech, that distinction could be particularly important. Big pharmaceutical companies can fund dozens of development programs and absorb expensive failures. Smaller companies don’t have that luxury.

Every candidate consumes capital. Every failed experiment costs time. And every month spent pursuing the wrong molecule can delay the strongest candidates from moving forward.

GT Biopharma is now integrating AI directly into the discovery engine behind its pipeline.

The company is using AI-guided sequence and structural analysis to evaluate potential tumor-targeting engagers and other proteins before selecting candidates for further development.^xi

The technology is being used to examine characteristics including binding, stability, molecular structure, and manufacturability.

It can also help optimize how the different components of a TriKE molecule are arranged to direct to new targets. For a modular platform like TriKE, that could become especially valuable.

Management says the new approach has already produced a marked acceleration in discovery productivity. GT Biopharma expects the resulting efficiencies to help move multiple new development candidates toward pre-IND development in 2027.

And the potential could extend well beyond cancer, opening the door to entirely new areas of medicine.

The Next Data Could Be Pivotal

GT Biopharma has reached a critical stage of development. For the first time, its second-generation TriKE technology is being tested in humans across both blood cancers and solid tumors.

The next clinical data could begin to show whether TriKE’s early promise can translate across a broader platform.

Investors interested in following GT Biopharma’s progress can sign up below to receive the latest company updates or speak directly with Investor Relations at 212-957-2577.