Cytarabine

New patents will extend coverage to major markets outside the U.S., expanding upon existing U.S. claims Affirms expectations for an initial data readout of the phase 3 MIRACLE trial for the second half of 2025 HOUSTON, March 6, 2025 — Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, today announced it has received a Notice of Intent to Grant for the European patent application titled, “Method of Reconstituting Liposomal Annamycin“. The grant is subject to payment of fees and completion of final amendments and formalities. Such grant will enhance the global exclusivity of Annamycin with the potential to be a next generation, non-cardiotoxic treatment for certain cancers. When issued, the patent claims will cover methods of making liposomal Annamycin suspension as well as the resulting compositions for use in the treatment of cancers, with a base patent term currently extending until June 2040, subject to extension to account for time required to fulfill requirements for regulatory approval. Moleculin’s novel drug candidate is being positioned to become the first ever non-cardiotoxic anthracycline to be approved and is currently being developed for the treatment of acute myeloid leukemia (AML) and soft tissue sarcoma lung metastases (STS lung mets). Additional preclinical studies performed at a world-renowned cancer center indicate Annamycin may be a potential treatment for many more other types of cancers. The new chemical entity uses a unique lipid-based delivery technology and has shown the potential to be used in a wide range of cancers. In addition to the expected European patent and previously issued U.S. patents, Moleculin has additional patent applications related to Annamycin pending in the U.S., Europe and in major jurisdictions worldwide. Wally Klemp, Chairman and CEO of Moleculin, said “Acknowledgment by the European Patent Office of the innovation underlying Annamycin is an important milestone for Moleculin, underscoring the importance and proprietary nature of the innovation that makes this next generation anthracycline possible. We expect Europe to be an important market for Annamycin and look forward to making an important new treatment available to patients in this region. This enhancing of our exclusivity for Annamycin is exciting when coupled with our continued expectation for an initial data readout for the MIRACLE trial in the second half of 2025.” 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 pathogenic 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 timing of the achievements of each of the milestones in this press release. Moleculin will require significant additional financing, for which the Company has no commitments, in order to conduct its clinical trials as described in this press release, and the milestones described in this press release assume the Company’s ability to secure such financing on a timely basis. 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, LLCJenene Thomas(908) 824-0775MBRX@jtcir.com View original content to download multimedia:https://www.prnewswire.com/news-releases/moleculin-announces-additional-annamycin-patent-allowances-to-enhance-global-exclusivity-302393600.html SOURCE Moleculin Biotech, Inc.

Last year was a rough one for cell and gene therapy companies. The expensive treatments faced stiff competition from more accessible options in crowded indications — prompting a broad industry migration out of oncology and into the autoimmune space, particularly for cell therapy developers (see – Vital Signs: The four big ideas that flopped in 2024).Unlike several of its peers — including Adicet Bio, IGM Biosciences, Caribou Biosciences and Nkarta — IN8bio did not change disease course last year with its allogeneic gamma-delta T cell therapy. While the biotech did pause its clinical-stage glioblastoma programmes to conserve cash, its lead candidate, INB-100, remains in Phase I testing for acute myeloid leukaemia (see – Spotlight On: Five reasons lupus research momentum is mounting).But now, after taking the necessary time to select the right candidate with a thought-out clinical and commercial strategy, IN8bio is ready to make its move into autoimmunity. Taking the TCE tackThe biotech unveiled a gamma-delta T cell engager (TCE) platform on Monday, as well as the first candidate to be built with the new technology: INB-619, a CD19-targeted TCE that could help treat leukaemias, lymphomas, and yes, autoimmune diseases. "When everybody else moved with cellular therapy into the autoimmune space, we didn't do that," IN8bio CEO William Ho told FirstWord. "We took our time, used our knowledge about biology, used our knowledge about diseases and gamma-delta T-cell biology, to create something where we think we can have some real advantages."Some of the cell therapy firms that jumped into the autoimmune space last year are already facing headwinds. Kyverna Therapeutics raised the second-largest biotech IPO last year on the strength of its inflammatory disease–directed CAR-T, but has since lost much of its valuation. A data disclosure in June for KYV-101 suggested some risk of recurrence following treatment — potentially undercutting one of the major purported advantages of cell therapy in settings like lupus (see – Vital Signs: Biotech's best (and worst) in class for 2024).In addition, while cell therapies make sense for patients who are dying of cancer, the treatments — which must be given at certified treatment centres, carry the risk of serious toxicities and can only be administered after lymphodepleting regimens — may not be attractive options to patients with autoimmune conditions like lupus. "When you look at the literature, a very large population of autoimmune patients are women between the ages of 20 and 45 — women of maternal age," Ho said. Because the lymphodepleting agent cytarabine can cause permanent ovarian damage, he speculated that only women who are very sick with an autoimmune disease would be willing to try a cell therapy.With those treatment barriers in mind, IN8bio decided that its gamma-delta T cell therapy wasn't the right horse to bet on for autoimmunity — but lucky for the biotech, it had something else in the works that was better suited for the disease space."It was somewhat fortuitous that we had developed this internal programme," Ho said of IN8bio's TCE. "By going to an engager — by using our knowledge and know-how about the gamma-delta T cell — we created a potentially off-the-shelf therapy that requires no lymphodepletion, that can be re-dosed, that drives deep B-cell depletion."The gamma-delta advantage?Like many B-cell depleting agents, IN8bio's candidate targets CD19. But rather than binding the T-cell antigen CD3 like most TCEs, INB-619's other arm engages with an undisclosed gamma-delta T-cell receptor.  The hope, Ho said, is that by avoiding CD3 engagement, the programme will lead to lower IL-6 secretion, and in turn, a lower risk of two of the most severe side effects associated with CAR-Ts and TCEs: cytokine release syndrome and immune effector cell–associated neurotoxicity syndrome.Some other potential advantages of INB-619 over traditional TCEs is its ability to drive high levels of T cell expansion and activate both major gamma-delta T cell subsets, which could help make the treatment more durable. IN8bio is evaluating the programme in preclinical studies, and is looking for a partner to move INB-619 into IND-enabling trials.