Moleculin Reports Second Quarter 2024 Financial Results and Provides Corporate Update

Rhea-AI Summary

Moleculin Biotech (Nasdaq: MBRX) reported its Q2 2024 financial results and provided a corporate update. Key highlights include:

1. Positive outcome from End of Phase 1B/2 Meeting with FDA
2. Proceeding with pivotal, adaptive Phase 3 MIRACLE trial for possible accelerated approval of Annamycin in combination with cytarabine for R/R AML treatment
3. Q2 2024 R&D expense: $4.1 million (up $0.2 million YoY)
4. Q2 2024 G&A expense: $2.1 million (down $0.4 million YoY)
5. Cash and cash equivalents: $10.8 million as of June 30, 2024
6. Filed S-1 to raise additional $12 million through equity issuance

The company expects to begin contracting MIRACLE trial sites in 2H 2024 and treat the first subject in Q1 2025.

Positive

• Positive outcome from FDA End of Phase 1B/2 Meeting, allowing progression to Phase 3 trial
• 41% of subjects in MB-106 trial achieved composite complete remission (CRc)
• 50% CR rate for 2nd line AML patients in MB-106 trial
• Annamycin has Fast Track Status and Orphan Drug Designation from FDA and EMA
• Planned $12 million equity raise to extend cash runway into Q2 2025

Negative

• R&D expenses increased by $0.2 million compared to Q2 2023
• Current cash only sufficient to fund operations into Q4 2024
• Potential dilution for existing shareholders due to planned equity issuance
• No guarantee of successful fundraising through equity issuance

Financial Analyst

Moleculin's Q2 2024 results reveal a mixed financial picture. R&D expenses increased slightly to $4.1 million, while G&A expenses decreased to $2.1 million. The company's cash position of $10.8 million is concerning, as it only provides runway until Q4 2024. The planned $12 million equity raise is important for extending operations to Q2 2025, but introduces potential dilution risks for current shareholders.

The positive FDA meeting outcome for Annamycin is promising, but investors should note that the pivotal Phase 3 MIRACLE trial timeline extends to 2028, indicating a long path to potential commercialization. While the adaptive trial design may reduce risks, it also adds complexity and potential costs. The 50% CR rate in 2nd line AML patients is encouraging but needs validation in larger studies.

Oncology Doctor

The MIRACLE trial's design is intriguing from a clinical perspective. The adaptive approach, aligning with FDA's Project Optimus, allows for dose optimization based on safety and efficacy data. This could potentially lead to a more effective and tolerable treatment regimen for R/R AML patients.

The reported 50% CR rate in 2nd line patients is promising, especially considering the poor prognosis of R/R AML. However, the small sample size (n=10) warrants caution. The median duration of response (mDOR) of 7 months is encouraging but needs further follow-up. The inclusion of 3rd line patients in MIRACLE2 could provide valuable data on Annamycin's efficacy in heavily pretreated populations, addressing a significant unmet need in AML treatment.

Market Research Analyst

Moleculin's focus on R/R AML positions it in a niche but potentially lucrative market. The global AML therapeutics market is projected to reach $3.56 billion by 2031, with a CAGR of 8.5%. R/R AML represents a significant portion of this market, with effective treatment options available.

The Fast Track and Orphan Drug designations for Annamycin could provide regulatory advantages and market exclusivity if approved. However, competition in the AML space is intense, with several big pharma companies and biotechs developing novel therapies. Moleculin's success will depend on Annamycin's efficacy data from the MIRACLE trial and its ability to differentiate from existing and emerging treatments. The company's pipeline diversity, including WP1066 for glioblastoma, provides some risk mitigation but also stretches resources.

-         Positive outcome from End of Phase 1B/2 Meeting with FDA

-         Company proceeding with a pivotal, adaptive Phase 3 clinical trial (the "MIRACLE" trial) designed for possible accelerated approval of Annamycin in combination with cytarabine for the treatment of R/R AML

-         Webcast replay of the Company's recent MIRACLE trial update available here

HOUSTON, Aug. 14, 2024 /PRNewswire/ -- Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a Phase 3 clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, today reported its financial results for the quarter ended June 30, 2024.

"I am extremely pleased with our recent clinical and regulatory achievements. Of particular note, the recent positive outcome from our end of phase 1B/2 clinical trial (EOP1B/2) meeting with the US Food and Drug Administration (FDA) combined with the encouraging Annamycin data demonstrated to date, positions us well for the next phase of development for our AML program," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "We are grateful to the FDA for their constructive feedback and I would like to congratulate our entire clinical team for their operational excellence in driving Annamycin forward as a potential treatment for relapsed or refractory acute myeloid leukemia (R/R AML) patients. We believe we are truly on the cusp of unlocking high-value potential for all stakeholders, and most importantly addressing a significant unmet need for R/R AML patients."

Recent Highlights

• Hosted webcast presentation to discuss the Company's previously announced plans for its MIRACLE Phase 3 pivotal trial;
• Completed EOP1B/2 meeting with FDA and planning for pivotal, adaptive Phase 3 clinical trial of Annamycin in combination with cytarabine for the treatment of R/R AML;
• Reported additional positive efficacy findings from the Company's Phase 1B/2 (MB-106) clinical trial evaluating Annamycin in combination with Cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as AnnAraC) for the treatment of subjects with acute myeloid leukemia (AML);
• Announced abstract has been accepted for poster presentation at the EHA2024 Hybrid Congress being held in Madrid, Spain and virtually; and
• Recruitment began in an Investigator-initiated Phase 2 study evaluating WP1066 in combination with radiation therapy for the treatment of adults with glioblastoma (NU 21C06) in cooperation with the Company.

AML Clinical Development Update

The Company recently announced the positive discussion in and outcome of its End of Phase 1B/2 (EOP1B/2) meeting with the US Food and Drug Administration (FDA) supporting the advancement of Annamycin in combination with Cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as "AnnAraC") to a Phase 3 pivotal trial for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) will be a global trial, including sites in the US.

The EOP2 meeting was supported by second-line treatment results from the Company's ongoing MB-106 clinical trial. As recently reported on June 14, 2024, a total of 22 subjects (Lines 1^st-7^th) have been enrolled (the Intent-to-Treat population, ITT) and have completed efficacy evaluations with 9 subjects (41%) achieving a composite complete remission (CRc or CR/CRi), consisting of 8 (36%) subjects with complete remission (CR) and one subject with complete remission with an incomplete recovery of peripheral blood counts (CRi), following treatment with AnnAraC.

Of the 10 ITT subjects for whom AnnAraC was administered in the 2^nd line setting, 5 achieved a CR (50%) and 6 achieved a CRc (60%). Of the 14 subjects in the ITT evaluable population that were 2^nd line or 3^rd line treatment, 6 achieved a CR (43%) and 7 achieved a CRc (50%). The mDOR for the 9 subjects who achieved a CRc is approximately 7 months and climbing.

In its EOP1B/2 meeting, the Company obtained valuable input from the FDA and having resolved a number of key issues, believes that it has significantly de-risked the pathway to a potential approval. The MIRACLE study, subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, is expected to initially utilize an adaptive design whereby the first 75 subjects will be randomized to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin. At that point, the trial will be unblinded to select the optimum dose for Annamycin. For the second half of the trial, approximately 120 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin. The selection of the optimum dose will be based not only on the absence of dose limiting toxicities but also on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA's new Project Optimus initiative.

Expected Milestones for Annamycin AML Development Program

• 2H 2024 – Begin contracting with MIRACLE trial sites
• Q1 2025 – First subject treated in MIRACLE trial
• Q4 2025 – Recruitment update (n=40)
• Mid 2026 – Interim data (n=75) unblinded and Optimum Dose set for MIRACLE trial
• 2026 – Begin enrollment of 3^rd line subjects in MIRACLE2
• 2027 – Enrollment ends in 2^nd line subjects
• 2028 – Final Data for 2^nd line subjects in MIRACLE
• 2H 2028 – Begin submission of a new drug application (NDA) the treatment of R/R AML for accelerated approval on primary endpoint of CR from MIRACLE

Annamycin currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the European Medicines Agency (EMA). For more information about the ongoing MB-106 Phase 1B/2 trial, visit clinicaltrialsregister.eu and reference EudraCT 2020-005493-10 or clinicaltrials.gov and reference NCT05319587.

Summary of Financial Results for the Second Quarter 2024

Research and development (R&D) expense was $4.1 million and $3.9 million for the three months ended June 30, 2024 and 2023, respectively. The increase of $0.2 million is mainly related to sponsored research costs.

General and administrative expense was $2.1 million and $2.5 million for the three months ended June 30, 2024 and 2023, respectively. The decrease of $0.4 million is mainly related to a decrease in regulatory and legal fees.

As of June 30, 2024, the Company had cash and cash equivalents of $10.8 million and believes that the existing cash and cash equivalents as of June 30, 2024, will be sufficient to fund our planned operations into the fourth quarter of 2024. An S-1 was recently filed with the Securities and Exchange Commission indicating our intentions to raise additional cash via the issuance of equity in the amount of $12 million. We believe that our existing cash and cash equivalents as of June 30, 2024, along with the cash expected from this raise will be sufficient to fund our planned operations into the second quarter of 2025. The amount of this raise may increase or decrease. There is no guarantee that the Company will be successful in such a raise.

About Moleculin Biotech, Inc.

Moleculin Biotech, Inc. is a Phase 3 clinical stage pharmaceutical company advancing a pipeline of therapeutic candidates addressing hard-to-treat tumors and viruses. The Company's lead program, Annamycin, is a next-generation anthracycline designed to avoid multidrug resistance mechanisms and to eliminate the cardiotoxicity common with currently prescribed anthracyclines. Annamycin is currently in development for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases.

The Company is initiating the MIRACLE (Moleculin R/R AML AnnAraC Clinical Evaluation) Trial (MB-108), a pivotal, adaptive design Phase 3 trial evaluating Annamycin in combination with cytarabine, together referred to as AnnAraC, for the treatment of relapsed or refractory acute myeloid leukemia. Following a successful Phase 1B/2 study (MB-106), with input from the FDA, the Company believes it has substantially de-risked the development pathway towards a potential approval for Annamycin for the treatment of AML. This study is subject to appropriate future filings with potential additional feedback from the FDA and their foreign equivalents.

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. Moleculin is also engaged in the development of a portfolio of antimetabolites, including WP1122 for the potential treatment of viruses, as well as certain cancer indications. 

For more information about the Company, please visit www.moleculin.com and connect on X, 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, the Company's forecasted cash burn rate (including its estimate of cash sufficient to meet its projected operating requirements) and the achievement of the expected milestones set forth above. 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

Moleculin Biotech, Inc.
Unaudited Condensed Consolidated Balance Sheets
(in thousands)						June 30, 2024		December 31, 2023
Current assets:
Cash and cash equivalents						$                          10,845		$                          23,550
Prepaid expenses and other current assets						2,886		2,723
Total current assets						13,731		26,273
Furniture and equipment, net						221		272
Intangible assets						11,148		11,148
Operating lease right-of-use asset						475		524
Total assets						$                          25,575		$                          38,217
Current liabilities:
Accounts payable and accrued expenses and other current liabilities						$                             5,746		$                            6,815
Total current liabilities						5,746		6,815
Operating lease liability - long-term, net of current portion						420		474
Warrant liability - long term						1,704		4,855
Total liabilities						7,870		12,144
Total stockholders' equity						17,705		26,073
Total liabilities and stockholders' equity						$                          25,575		$                          38,217
Unaudited Condensed Consolidated Statements of Operations
		Three Months Ended June 30,				Six Months Ended June 30,
(in thousands, except share and per share amounts)		2024		2023		2024		2023
Revenues		$                             -		$                             -		$                                     -		$                                     -
Operating expenses:
Research and development		4,090		3,888		8,342		9,576
General and administrative and depreciation and amortization		2,095		2,523		4,520		5,190
Total operating expenses		6,185		6,411		12,862		14,766
Loss from operations		(6,185)		(6,411)		(12,862)		(14,766)
Other income:
Gain from change in fair value of warrant liability		1,696		36		3,151		75
Other income, net		11		9		22		17
Interest income, net		159		390		400		783
Net loss		(4,319)		(5,976)		(9,289)		(13,891)
Net loss per common share - basic and diluted		$                     (1.70)		$                     (3.02)		$                             (3.71)		$                             (7.13)
Weighted average common shares outstanding - basic and diluted		2,543,244		1,979,258		2,504,709		1,948,135

 

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SOURCE Moleculin Biotech, Inc.

FAQ

What is the MIRACLE trial for Moleculin Biotech (MBRX)?

The MIRACLE trial is Moleculin's planned pivotal, adaptive Phase 3 clinical trial designed for possible accelerated approval of Annamycin in combination with cytarabine for the treatment of relapsed or refractory acute myeloid leukemia (R/R AML).

When does Moleculin (MBRX) expect to begin the MIRACLE trial?

Moleculin expects to begin contracting MIRACLE trial sites in the second half of 2024 and treat the first subject in Q1 2025.

What were Moleculin's (MBRX) key financial results for Q2 2024?

In Q2 2024, Moleculin reported R&D expenses of $4.1 million, G&A expenses of $2.1 million, and cash and cash equivalents of $10.8 million as of June 30, 2024.

How long can Moleculin (MBRX) fund its operations with current cash?

Moleculin's existing cash and cash equivalents as of June 30, 2024, are sufficient to fund planned operations into the fourth quarter of 2024. With the planned $12 million equity raise, they expect to extend the runway into the second quarter of 2025.