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GT Biopharma Announces Potential New Indication for GTB-5550, a B7-H3-Targeted Natural Killer (NK) Cell Engager, for Multiple Myeloma

FDA clears a new IND for GTB-5550 in multiple myeloma as GT Biopharma targets later-line patients and extends cash runway into Q3 2027.

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GT Biopharma (GTBP) received FDA clearance for a new IND to study GTB-5550, a B7-H3-targeted TriKE NK cell engager, in multiple myeloma under an investigator sponsored Phase 1a/1b trial. Initiation of this trial in later-line multiple myeloma patients is contingent on timing and receipt of non-dilutive grant funding. A separate Phase 1 dose-escalation basket trial of GTB-5550 in solid tumor patients is actively enrolling, with an update expected in Q4 2026. The company reports an unaudited pro forma cash balance of approximately $11 million as of September 15, 2026, which it anticipates will fund operations into Q3 2027.

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Positive

  • FDA IND clearance for GTB-5550 in multiple myeloma under an investigator sponsored trial
  • GTB-5550 Phase 1 solid tumor basket trial is actively enrolling, with a data update anticipated in Q4 2026
  • Unaudited pro forma cash balance of ~$11 million as of September 15, 2026
  • Company expects current cash to provide runway into Q3 2027

Negative

  • Initiation of the multiple myeloma Phase 1 trial is contingent on securing non-dilutive grant funding

News Explained

Commercialization of GTB-5550 would carry a disclosed royalty obligation to the University of Minnesota if sales occur.

The proposed multiple-myeloma study would use subcutaneous GTB-5550 in a Phase 1a dose-escalation stage of up to six cohorts, followed by a Phase 1b expansion treating 25 patients at the maximum tolerated dose.

The protocol calls for at least two four-week treatment cycles, with possible continuation to four cycles for stable disease or one year for patients achieving a partial response or better.

Under the disclosed license, the University of Minnesota would be entitled to royalties if commercial sales of products using the TriKE technology, including GTB-5550, are realized.

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-25.75% vs previous close $8.63 last price 3.4x rel. volume Open Argus
Details

Market reaction after multiple myeloma IND clearance: GTBP -25.75%

$8.21 $13.51 Day Range
$15.58M Market Cap

Following this news, GTBP has declined 25.75%, reflecting a significant negative market reaction. Our momentum scanner has triggered 24 alerts so far, indicating elevated trading interest and price volatility. The stock is currently trading at $8.63. Trading volume is very high at 3.4x the average, suggesting heavy selling pressure.

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Market Context

The May 14 GTB-5550 solid-tumor first-patient dosing announcement was followed by a 40.36% 24-hour g...
Analysis

The May 14 GTB-5550 solid-tumor first-patient dosing announcement was followed by a 40.36% 24-hour gain; it established prior clinical advancement of the same program, while this announcement added a multiple-myeloma indication.

Key Figures

Pro forma cash balance: approximately $11 million Cash runway: Q3 2027 Dose cohorts: up to 6 dose cohorts +5 more
Pro forma cash balance
approximately $11 million
As of September 15, 2026
Cash runway
Q3 2027
Anticipated runway from pro forma cash balance
Dose cohorts
up to 6 dose cohorts
Phase 1a dose escalation in multiple myeloma
Expansion cohort
25 patients
Phase 1b treated at the maximum tolerated dose
Dosing frequency
3 non-consecutive days per week
Weeks 1 and 2 of the treatment schedule
Treatment cycle
4 weeks
Each treatment cycle
Minimum treatment
2 cycles
Planned minimum treatment duration
Follow-up period
12 months
Follow-up from first dose to assess PFS and OS

Historical Context

2 past events · Latest: May 14
2 events
  1. May 14

    GTB-5550 first dosing

    24h Move
    +40.4%

    First patient dosed in Phase 1 GTB-5550 solid-tumor basket trial

  2. Aug 14

    Q2 earnings report

    24h Move
    -0.5%

    GTB-5550 solid-tumor basket trial had initiated with further updates anticipated

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

investigator sponsored trial, mtd, pfs, os
4 terms
investigator sponsored trial regulatory
"under an investigator sponsored trial (IST) protocol"
A researcher‑led clinical trial is a study initiated and run by an independent clinician or academic team rather than the company that makes the drug or device; the company may provide the product, data access, or funding but does not control the study. Investors care because these independent trials can provide extra validation, new uses, or safety information that affects a product’s market value—think of an outside expert testing a product in their own lab and reporting unbiased results.
mtd medical
"identify the maximum tolerated dose (MTD)"
Month-to-date (MTD) is a short-term performance measure that shows how a stock, portfolio, or financial metric has changed from the first day of the current month up to today. Think of it like checking a meter partway through a road trip to see whether you’re ahead or behind schedule; investors use MTD to spot early trends, monitor recent momentum, and make quick decisions before full-month results are available.
pfs medical
"determine progression-free survival (PFS) and overall survival (OS)"
Progression-free survival (PFS) is a clinical-trial measure that records how long, on average, patients live without their disease getting worse after starting a treatment. For investors, PFS acts like a stopwatch of a drug’s effectiveness: longer PFS can signal meaningful patient benefit, improve chances of regulatory approval or label strength, and raise a drug’s commercial value, while shorter or unchanged PFS can weigh on a company’s prospects.
os medical
"determine progression-free survival (PFS) and overall survival (OS)"
Overall survival (OS) measures the length of time from a defined starting point, usually treatment or trial enrollment, until death from any cause; it tells how much a therapy extends life. For investors, OS is a hard, widely accepted clinical endpoint that regulators and doctors use to judge a drug’s real-world benefit, and strong OS results can drive approvals, market adoption, and a company’s valuation — like a clear safety rating boosting confidence in a car model.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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FDA clearance of a new IND for GTB-5550 in multiple myeloma (MM) under an investigator sponsored trial (IST) protocol

Initiation of Phase 1 trial in MM patients contingent upon timing and receipt of non-dilutive grant funding

Phase 1 dose escalation basket trial with GTB-5550 in solid tumor patients continues to actively enroll, with an update anticipated in Q4 2026

Unaudited proforma cash balance as of September 15, 2026 of approximately $11 million anticipated to provide sufficient cash runway into Q3 2027

SAN FRANCISCO, CALIFORNIA, Sept. 15, 2026 (GLOBE NEWSWIRE) -- GT Biopharma, Inc. (the “Company”) (NASDAQ: GTBP), a clinical stage immuno-oncology company focused on developing innovative therapeutics based on the Company's proprietary TriKE® natural killer (NK) cell engager platform, today announced IND clearance from the FDA for GTB-5550 in multiple myeloma.

“Today’s IND to expand the potential indications for GTB-5550 highlights the diverse utility of our Trike® platform,” said Michael Breen, Executive Chairman and Chief Executive Officer of GT Biopharma. “The company remains focused on its two ongoing Phase 1 trials with GTB-5550 and GTB-3650, both of which are actively enrolling, and we look forward to the potential initiation of a third Phase 1 trial, pending receipt of non-dilutive grant funding.”

“Targeting B7-H3 remains an active area for clinical development of new anti-cancer drugs, yet most competitive efforts are focused on solid tumors”, said Dr. Aimee Merino, MD, PhD, Assistant Professor of Medicine, Division of Hematology, Oncology and Transplantation, The University of Minnesota1. “Based on our findings of high levels of B7-H3 expression within the bone marrow of newly diagnosed multiple myeloma patients, we have begun a dedicated effort for this novel approach in later-line patients, which continues to be an area of unmet need. We have been encouraged with our preclinical findings in a bone chip model that show high levels of antitumor cell activity mediated by NK cells in the presence of GTB-5550.”

The Phase 1a/1b trial with GTB-5550 will be the first-in-human trial of a B7-H3-targeted TriKE® in multiple myeloma and will use more patient-friendly subcutaneous administration intended to extend the half-life relative to prior intravenous TriKE formulations. The Phase 1a dose escalation portion will enroll 4th line and later patients with multiple myeloma and evaluate up to 6 dose cohorts to identify the maximum tolerated dose (MTD). Following dose escalation, the Phase 1b expansion cohort will enroll a total of 25 patients treated at the MTD and further evaluate safety, tolerability, and preliminary anti-tumor activity.

In the Phase 1 trial, GTB-5550 will be administered by subcutaneous (SQ) injection in the abdominal area on 3 non-consecutive days per week during Week 1 and Week 2, followed by 2 weeks of no treatment. One treatment cycle is 4 weeks in duration, and this dosing schedule is repeated for each subsequent cycle. A minimum of 2 cycles is planned, and disease reassessment (monoclonal protein and free light chain analysis) is performed on Day 28 of each cycle. Patients with stable disease may continue for up to 4 cycles total, and those with a partial response or better may continue for up to 1 year. Patients are followed for 12 months from the first dose of GTB-5550 to determine progression-free survival (PFS) and overall survival (OS).

About GT Biopharma, Inc.

GT Biopharma, Inc. is a clinical stage biopharmaceutical company focused on the development and commercialization of immuno-oncology therapeutic products based on our proprietary TriKE® NK cell engager platform. Our TriKE® platform is designed to harness and enhance the cancer killing abilities of a patient’s immune system’s natural killer cells. GT Biopharma has an exclusive worldwide license agreement with the University of Minnesota to further develop and commercialize therapies using TriKE® technology. For more information, please visit gtbiopharma.com.

Forward-Looking Statements

Certain statements in this press release may constitute "forward-looking statements" regarding future events and our future results. All statements other than statements of historical facts are statements that could be deemed to be forward-looking statements. These statements are based on current expectations, estimates, forecasts, and projections about the markets in which we operate and the beliefs and assumptions of our management. Words such as "expects," "anticipates," "targets," "goals," "projects", "intends," "plans," "believes," "seeks," "estimates," "endeavors," "strives," "may," or variations of such words, and similar expressions are intended to identify such forward-looking statements. Readers are cautioned that these forward-looking statements are subject to a number of risks, uncertainties and assumptions that are difficult to predict, estimate or verify. Therefore, actual results may differ materially and adversely from those expressed in any forward-looking statements. Such risks and uncertainties include those factors described in our most recent annual report on Form 10-K, as such may be amended or supplemented by subsequent quarterly reports on Form 10-Q, or other reports filed with the Securities and Exchange Commission. Readers are cautioned not to place undue reliance on these forward-looking statements. The forward-looking statements are made only as of the date hereof, and we undertake no obligation to publicly release the result of any revisions to these forward-looking statements. For more information, please refer to our filings with the Securities and Exchange Commission.

TriKE® is a registered trademark owned by GT Biopharma, Inc.

Investor Relations Contact:
LifeSci Advisors
Corey Davis, Ph.D.
cdavis@lifesciadvisors.com
212-915-2577

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1 The University of Minnesota, pursuant to its license agreement with GT Biopharma, is entitled to receive royalties should commercial sales of product using the TriKE technology be realized, including GTB-3650 and GTB-5550. This interest has been reviewed and managed by the University of Minnesota in accordance with its conflict of interest policies.


FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What is the design of the new Phase 1a/1b multiple myeloma trial for GTB-5550?

The Phase 1a/1b trial will be the first-in-human study of a B7-H3-targeted TriKE in multiple myeloma. Phase 1a will enroll fourth-line and later multiple myeloma patients in up to six dose cohorts to identify the maximum tolerated dose (MTD). Phase 1b will then enroll a total of 25 patients at the MTD to further evaluate safety, tolerability, and preliminary anti-tumor activity.

How will GTB-5550 be administered and what is the dosing schedule in the multiple myeloma trial?

In the multiple myeloma Phase 1 trial, GTB-5550 will be given by subcutaneous injection in the abdominal area on three non-consecutive days per week during Week 1 and Week 2, followed by two weeks without treatment. One treatment cycle lasts four weeks and this schedule is repeated for subsequent cycles.

How long can patients remain on GTB-5550 treatment and how are outcomes assessed?

A minimum of two treatment cycles is planned, with disease reassessment (monoclonal protein and free light chain analysis) on Day 28 of each cycle. Patients with stable disease may receive up to four cycles total, and those with a partial response or better may continue treatment for up to one year. Patients are followed for 12 months from first dose to assess progression-free survival and overall survival.

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