Moleculin Announces Dosing of First Subject in Italy for Phase 1/2 Trial Evaluating Annamycin in Combination with Cytarabine (Ara-C) for the Treatment of Acute Myeloid Leukemia (AML)

Rhea-AI Summary

Moleculin Biotech, Inc. (NASDAQ: MBRX) announced the treatment of the first subject in Italy as part of its Phase 1/2 trial, MB-106, evaluating Annamycin combined with Cytarabine for refractory acute myeloid leukemia (AML). The Phase 1/2 trial builds on earlier successful trials, with the MB-105 study showing an 80% overall response rate. Annamycin is designed to avoid cardiotoxicity and demonstrate higher efficacy against chemotherapy-resistant cancers. The company aims to expand recruitment and has received Fast Track and Orphan Drug Designation from the FDA for Annamycin. The final clinical study report is expected to be published soon.

Positive

• Commencement of Phase 1/2 trial in Italy is a key milestone for MBRX.
• 80% overall response rate reported in previous MB-105 study for relapsed/refractory AML.
• Annamycin shows potential to improve median overall survival compared to Cytarabine alone.

Negative

• None.

HOUSTON, March 1, 2023 /PRNewswire/ -- Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, today announced the first subject has been treated in Italy in the Company's Phase 1/2 trial evaluating Annamycin in combination with Cytarabine (Ara-C) for the treatment of subjects with AML who are refractory to or relapsed after induction therapy (MB-106).

"We continue to be highly encouraged by the data demonstrated from the successfully concluded Annamycin single agent trials and the no evidence of cardiotoxicity seen to date. We remain very optimistic in Annamycin's potential to be an important treatment for refractory or relapsed AML in combination with Cytarabine. We are pleased to commence subject dosing in Italy and are continuing our efforts to bring additional sites online and build momentum in enrollment.  We are committed to driving the development of Annamycin for the treatment of AML and look forward to continuing our evaluations of Annamycin in combination with Ara-C," commented Walter Klemp, Chairman and CEO of Moleculin.

Annamycin is the Company's next-generation anthracycline that has been designed to be non-cardiotoxic and has been shown in animal models to accumulate in the lungs at up to 30-fold the level of doxorubicin (a commonly prescribed anthracycline), as well as demonstrating the ability to avoid the multidrug resistance mechanisms that typically limit the efficacy of doxorubicin and other currently prescribed anthracyclines. Annamycin is currently in development for the treatment of relapsed or refractory AML and STS lung metastases and the Company believes it may have the potential to treat additional indications.

The Phase 1/2 MB-106 trial is an open label trial that builds on the safety and dosage data from the two successfully concluded single agent Annamycin AML Phase 1 trials, MB-104 and MB-105, conducted in the U.S. and Europe, respectively. Highlights from the final data demonstrated in the MB-105 study include:

• MB-105 demonstrated 80% overall response rate (ORR) in final cohort for treatment of relapsed/refractory acute myeloid leukemia (R/R AML).
• Final reported results showed one CRi (complete response with incomplete recovery of peripheral blood count) and three PRs (Partial Response).
• The responses in the MB-105 trial were seen in subjects 62-73 years of age and the CRi was from a subject 65 years of age.

The clinical study report for MB-105 has been finalized and the Company expects to publish detailed data in a paper or publication in the near future.

Data so far have resulted from treatment with Annamycin as a single agent, however, additional preclinical animal data from sponsored research demonstrated Annamycin in combination with Cytarabine had a 68% improvement in the median overall survival (OS) compared to Annamycin as a single agent and a 241% increase in OS compared to Cytarabine alone.¹

Annamycin currently has Fast Track Status and Orphan Drug Designation from the US Food and Drug Administration (FDA) for the treatment of STS lung metastases and the treatment of relapsed or refractory AML. For more information about the MB-106 Phase 1/2 trial, visit clinicaltrialsregister.eu and reference EudraCT 2020-005493-10 or clinicaltrials.gov and reference NCT05319587.

About Moleculin Biotech, Inc.

Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on the development of a broad portfolio of drug candidates for the treatment of highly resistant tumors and viruses. The Company's lead program, Annamycin is a next-generation anthracycline designed to avoid multidrug resistance mechanisms with little to no cardiotoxicity. Annamycin is currently in development for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases.

Additionally, the Company is developing WP1066, an Immune/Transcription Modulator capable of inhibiting p-STAT3 and other oncogenic transcription factors while also stimulating a natural immune response, targeting brain tumors, pancreatic and other cancers, and WP1220, an analog to WP1066, for the topical treatment of cutaneous T-cell lymphoma. Moleculin is also engaged in the development of a portfolio of antimetabolites, including WP1122 for the potential treatment of COVID-19 and other viruses, as well as cancer indications including brain tumors, pancreatic and other cancers.

For more information about the Company, please visit www.moleculin.com and connect on Twitter, LinkedIn and Facebook.

Forward-Looking Statements

Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties. Forward-looking statements in this press release include, without limitation, whether the results of Moleculin's preclinical animal models can be replicated in human trials and Moleculin's ability to expand its MB-106 clinical trial using Annamycin in combination with Cytarabine for the treatment of R/R AML into additional sites in Poland and other countries in Europe to potentially improve recruitment rates. Although Moleculin believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. Moleculin has attempted to identify forward-looking statements by terminology including 'believes,' 'estimates,' 'anticipates,' 'expects,' 'plans,' 'projects,' 'intends,' 'potential,' 'may,' 'could,' 'might,' 'will,' 'should,' 'approximately' or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including those discussed under Item 1A. "Risk Factors" in our most recently filed Form 10-K filed with the Securities and Exchange Commission ("SEC") and updated from time to time in our Form 10-Q filings and in our other public filings with the SEC. Any forward-looking statements contained in this release speak only as of its date. We undertake no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.

Investor Contact:

JTC Team, LLC
Jenene Thomas
(833) 475-8247
MBRX@jtcir.com

DC:82440151.1

¹ Zal T, Zielinski R, Grela K, Cardenas-Zuniga R, Skora S, Fokt I, Zal A, Andreeff M, Gil L Shephard R, Priebe W, High Efficacy of Liposomal Annamycin (L-ANN) in Combination with Cytarabine in Syngeneic p53-Null AML Mouse Model, Blood (2020) 136 (Supplement 1): 6-7. https://doi.org/10.1182/blood-2020-143344

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SOURCE Moleculin Biotech, Inc.

FAQ

What is the purpose of Moleculin's Phase 1/2 trial MB-106?

The Phase 1/2 trial MB-106 aims to evaluate Annamycin in combination with Cytarabine for treating patients with refractory or relapsed acute myeloid leukemia (AML).

What were the results of the previous MB-105 study?

The MB-105 study showed an 80% overall response rate, including one complete response with incomplete recovery and three partial responses in relapsed/refractory AML patients.

How does Annamycin differ from traditional anthracyclines?

Annamycin is designed to be non-cardiotoxic and can bypass multidrug resistance, showing significantly higher accumulation in lungs compared to doxorubicin.

What designations has Annamycin received from the FDA?

Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for both relapsed or refractory AML and soft tissue sarcoma lung metastases.

When can we expect the detailed data from the MB-105 study?

The detailed clinical study report for the MB-105 study is expected to be published soon.