Moleculin Reports Second Quarter 2024 Financial Results and Provides Corporate Update
- 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
"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
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 inMadrid, 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
The EOP2 meeting was supported by second-line treatment results from the Company's ongoing MB-106 clinical trial. As recently reported on
Of the 10 ITT subjects for whom AnnAraC was administered in the 2nd line setting, 5 achieved a CR (50%) and 6 achieved a CRc (60%). Of the 14 subjects in the ITT evaluable population that were 2nd line or 3rd 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
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 3rd line subjects in MIRACLE2
- 2027 – Enrollment ends in 2nd line subjects
- 2028 – Final Data for 2nd 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
Summary of Financial Results for the Second Quarter 2024
Research and development (R&D) expense was
General and administrative expense was
As of
About
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
Investor Contact:
(833) 475-8247
MBRX@jtcir.com
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Unaudited Condensed Consolidated Balance Sheets |
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(in thousands) |
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Current assets: |
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Cash and cash equivalents |
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$ 10,845 |
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$ 23,550 |
Prepaid expenses and other current assets |
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2,886 |
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2,723 |
Total current assets |
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13,731 |
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26,273 |
Furniture and equipment, net |
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221 |
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272 |
Intangible assets |
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11,148 |
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11,148 |
Operating lease right-of-use asset |
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475 |
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524 |
Total assets |
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$ 25,575 |
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$ 38,217 |
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Current liabilities: |
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Accounts payable and accrued expenses and other current liabilities |
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$ 5,746 |
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$ 6,815 |
Total current liabilities |
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5,746 |
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6,815 |
Operating lease liability - long-term, net of current portion |
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420 |
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474 |
Warrant liability - long term |
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1,704 |
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4,855 |
Total liabilities |
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7,870 |
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12,144 |
Total stockholders' equity |
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17,705 |
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26,073 |
Total liabilities and stockholders' equity |
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$ 25,575 |
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$ 38,217 |
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Unaudited Condensed Consolidated Statements of Operations |
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Three Months Ended |
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Six Months Ended |
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(in thousands, except share and per share amounts) |
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2024 |
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2023 |
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2024 |
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2023 |
Revenues |
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$ - |
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$ - |
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$ - |
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$ - |
Operating expenses: |
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Research and development |
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4,090 |
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3,888 |
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8,342 |
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9,576 |
General and administrative and depreciation and amortization |
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2,095 |
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2,523 |
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4,520 |
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5,190 |
Total operating expenses |
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6,185 |
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6,411 |
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12,862 |
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14,766 |
Loss from operations |
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(6,185) |
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(6,411) |
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(12,862) |
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(14,766) |
Other income: |
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Gain from change in fair value of warrant liability |
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1,696 |
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36 |
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3,151 |
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75 |
Other income, net |
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11 |
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9 |
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22 |
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17 |
Interest income, net |
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159 |
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390 |
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400 |
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783 |
Net loss |
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(4,319) |
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(5,976) |
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(9,289) |
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(13,891) |
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Net loss per common share - basic and diluted |
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$ (1.70) |
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$ (3.02) |
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$ (3.71) |
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$ (7.13) |
Weighted average common shares outstanding - basic and diluted |
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2,543,244 |
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1,979,258 |
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2,504,709 |
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1,948,135 |
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