On March 31, 2025 MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, reported it will present new non clinical data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, taking place on April 25-30, 2025 in Chicago, Illinois (Press release, MaaT Pharma, MAR 31, 2025, View Source [SID1234651679]).
MaaT034 is the Company’s next-generation full ecosystem synthetic microbiome therapy and the first candidate from its proprietary MET-C platform. Developed using a co-culturing technology optimized for large-scale production, MaaT034 is designed to improve patient responses to immunotherapy in combination with Immune Checkpoint Inhibitors.

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The key findings, obtained from studies conducted in germ-free mice, demonstrate that MaaT034:

Effectively engrafts, thus ensuring an enduring presence of beneficial bacteria in the gut environment.
Potentiates anti-tumor effects mediated by anti-PD-1 checkpoint blockade.
Leads to the production of key microbial-derived metabolites in germ-free mice.
Improves gastrointestinal physiological functions.
In parallel, the Company is developing donor-derived microbiome therapies that are being explored in multiple clinical trials. Among these, MaaT013 has been tested in patients with acute Graft-versus-Host Disease (aGvHD) and advanced melanoma receiving immune checkpoint inhibitors (PICASSO trial).

"While the PICASSO trial builds on MaaT013’s established safety and efficacy to potentially unlock significant new market opportunities in oncology, our next-generation candidate, MaaT034 — to be featured at AACR (Free AACR Whitepaper) — is designed with an optimized microbiome composition that eliminates the need for donors and incorporates defined and reproducible immunomodulatory properties, offering enhanced therapeutic potential across a broader range of oncology indications," stated Hervé Affagard, CEO and co-founder of MaaT Pharma.

AACR Poster Presentation details:

Title: MaaT034, a new co-cultured microbiome ecosystem therapy candidate, is capable to safely colonize the gastro-intestinal tract of germ-free mice to restore a healthy gut physiology and to stimulate immunity
Abstract Number: 2209
Session Category: Immunology
Session Title: Microbiome, Inflammation, and Cancer
Session Date/ Time: Monday April 28th, 2025 – 9:00:00 AM U.S Central Time
About Picasso

PICASSO is a Phase 2a clinical trial sponsored by AP-HP and in collaboration with INRAE and Institut Gustave Roussy, evaluating MaaT013 in combination with immune checkpoint inhibitors, ipilimumab (Yervoy) and nivolumab (Opdivo). The trial is fully recruited and have enrolled 70 patients. Key study endpoints include MaaT013 safety profile and best-overall response rate vs placebo as add-on treatment to Ipilimumab + Nivolumab. Clinical Trials.gov: NCT04988841

About MaaT034

MaaT034, currently in preclinical development, is a next-generation donor-independent full ecosystem synthetic microbiome therapy, dedicated to improving patient responses to immunotherapy in combination with Immune Checkpoint Inhibitors. Developed using the Company’s co-culturing proprietary MET-C platform, MaaT034 is optimized for large-scale production in oncology. Previous presented preclinical data showed that MaaT034 produced key metabolites, recognized as promoting gut barrier restoration and modulating immune responses, such as Short-Chain Fatty Acids (SCFA), secondary bile acids, and tryptophan derivatives. These data support the role of MaaT034 in gut barrier repair and in T cell reactivation either in combination with anti-PD1 or with anti-PD-L1. By enhancing gut barrier repair and modulating immune responses, MaaT034 is expected to complement the action of these immunotherapeutic agents, potentially improving their efficacy in treating solid tumors cancer.

On March 31, 2025 Hoth Therapeutics, Inc. (NASDAQ: HOTH), a patient-focused clinical-stage biopharmaceutical company, reported that it has received an official Filing Receipt from the United States Patent and Trademark Office (USPTO) for a new patent application related to the proprietary formulation of HT-001, the Company’s lead clinical asset (Press release, Hoth Therapeutics, MAR 31, 2025, https://www.prnewswire.com/news-releases/hoth-therapeutics-receives-uspto-filing-receipt-for-ht-001-formulation-patent-application-expanding-existing-patent-coverage-302416196.html [SID1234651698]).

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This newly filed application seeks to expand the Company’s intellectual property portfolio surrounding HT-001 and builds upon Hoth’s already approved patent for the compound. The formulation-specific filing is aimed at further protecting the unique composition and delivery method of HT-001, which is being developed as a novel topical treatment to alleviate dermatologic side effects commonly experienced by cancer patients undergoing epidermal growth factor receptor (EGFR) inhibitor therapy.

"The receipt of this Filing Receipt from the USPTO marks another critical milestone in the development of HT-001," said Robb Knie, Chief Executive Officer of Hoth Therapeutics. "Expanding our patent protection to include formulation-specific claims reinforces the strength of our IP position and our long-term commitment to patients and shareholders."

The USPTO Filing Receipt confirms that the patent application has been formally accepted and will proceed through the examination process. This development adds an important layer of protection as Hoth continues advancing HT-001 through clinical development.

On March 31, 2025 BioLineRx Ltd. (NASDAQ/TASE: BLRX), a development stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, reported its audited financial results for the year ended December 31, 2024, and provided a corporate update (Press release, BioLineRx, MAR 31, 2025, View SourceArchives/edgar/data/1498403/000117891325001124/exhibit_1.htm" target="_blank" title="View SourceArchives/edgar/data/1498403/000117891325001124/exhibit_1.htm" rel="nofollow">View Source [SID1234651661]).

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"It has been just over four months since we implemented a major strategy shift, highlighted by the transformational exclusive licensing agreement that we entered into with Ayrmid Ltd., granting it the rights to commercialize APHEXDA (motixafortide) in all non-solid-tumor indications and all territories other than Asia," said Philip Serlin, Chief Executive Officer of BioLineRx. "Since then, we implemented cost efficiencies across the Company, including the shutdown of our U.S. commercial operations, that have resulted in an approximate 70% reduction in our operating expense base, which, together with recent financings, have put us on a firm footing with a cash runway through the second half of 2026."

"As we return to our roots as a lean drug development company, with a highly validated development platform focused on oncology and rare diseases, we believe these actions help ensure that we remain nimble and capable of seizing the opportunities in front of us. Our strategy moving forward is to in-license additional assets over the next year that we can advance through clinical proof-of-concept, funded in part by milestones and royalties from our out-licensing transactions. To that end, I am pleased to report that we are evaluating numerous promising candidates. This process is methodical and steady to ensure that our due diligence is thorough as we look for new chemical entities. Based on our deep and validated experience in drug development, I believe we are well positioned to create sustained value for our shareholders. I am excited about what the future holds for our Company this year and beyond," Mr. Serlin concluded.

Corporate Updates

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Executed license agreement with Ayrmid Pharma Ltd. to develop and commercialize APHEXDAâ (motixafortide) in all indications except solid tumors, and across all territories except Asia

o
License agreement included a $10 million upfront payment, up to $87 million in potential commercial milestones, and royalties on net sales ranging from 18% to 23%

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Announced receipt of a Notice of Allowance from the U.S. Patent & Trademark Office (USPTO) for a patent, titled "Composition of BL-8040," which strengthens BioLineRx’s robust intellectual property (IP) estate and extends its patent protection on motixafortide (BL-8040) in the U.S. through December 2041

Financial Updates

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Completed two financings in past few months which raised combined gross proceeds of $19 million

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Reduced operating expense run rate by approximately 70% beginning January 1, 2025 through the APHEXDA program transfer to Ayrmid and the resulting shutdown of the Company’s U.S. commercial operations in Q424, as well as additional headcount and other operating expense reductions

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Significantly reduced outstanding debt and restructured the remainder on favorable terms to the Company

APHEXDA 2024 Performance Update

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Aphexda achieved 10 percent market share of total CXCR4 inhibitor usage in the U.S., which compares APHEXDA to branded MOZOBIL and generic plerixafor in all indications

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BioLineRx generated more than $6 million in net product sales year-to-date through the November 2024 completion of the Ayrmid out-licensing transaction

Clinical Updates

Motixafortide

Pancreatic Ductal Adenocarcinoma (mPDAC)

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Additional trial sites activated for the CheMo4METPANC Phase 2b clinical trial being led by Columbia University. Full enrollment in the randomized trial targeting 108 patients is anticipated in 2027, with a prespecified interim futility analysis planned when 40% of PFS events are observed

Sickle Cell Disease (SCD) & Gene Therapy

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First patient dosed in the multi-center Phase 1 clinical trial evaluating motixafortide for the mobilization of CD34+ hematopoietic stem cells (HSCs) used in the development of gene therapies for patients with Sickle Cell Disease (SCD). The trial is sponsored by St. Jude Children’s Research Hospital.

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Oral presentation delivered at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition detailing initial results from a Phase 1 clinical trial evaluating motixafortide as monotherapy and in combination with natalizumab for CD34+ hematopoietic stem cell (HSC) mobilization for gene therapies in SCD. Sponsored by investigators at Washington University in St. Louis, the findings from this proof-of-concept study suggest motixafortide alone, and in combination with natalizumab, could support the collection of the large number of stem cells required by gene therapies for sickle cell disease within a single apheresis cycle.

Financial Results for the Year Ended December 31, 2024

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Revenues for the year ended December 31, 2024 were $28.9 million, an increase of $24.1 million, or 502.1%, compared to $4.8 million for the year ended December 31, 2023. The revenues in 2024 primarily reflect a portion of the up-front payment received, and a milestone payment achieved, under the Gloria license, which collectively amounted to $15.0 million, as well as the up-front payment received under the Ayrmid license and $6.0 million of net revenues from product sales of APHEXDA in the U.S. The revenues in 2023 (all of which were recorded in the fourth quarter of 2023) primarily reflect a portion of the up-front payment received under the Gloria license of $4.6 million, as well as $0.2 million of revenues from product sales of APHEXDA in the U.S.

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Cost of revenues for the year ended December 31, 2024 were $9.3 million, an increase of $5.6, or 151.4%, compared to $3.7 million for the year ended December 31, 2023. The cost of revenues in 2024 primarily reflects amortization of intangible assets, Biokine’s share of the up-front payment received under the Ayrmid license, sub-license fees accrued on a milestone payment recorded under the Gloria license, as well as royalties on net product sales of APHEXDA in the U.S. and cost of goods sold on product sales. The cost of revenues in 2023 primarily reflects Biokine’s share of the up-front payment received under the Gloria license and of the net sales.

•
Research and development expenses for the year ended December 31, 2024 were $9.2 million, a decrease of $3.3 million, or 26.4%, compared to $12.5 million for the year ended December 31, 2023. The decrease resulted primarily from lower expenses related to motixafortide NDA supporting activities, termination of the development of AGI-134 and a decrease in payroll and share-based compensation.

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Sales and marketing expenses for the year ended December 31, 2024 were $23.6 million, a decrease of $1.7 million, or 6.7%, compared to $25.3 million for the year ended December 31, 2023. The decrease resulted primarily from the shutdown of U.S. commercial operations in the fourth quarter of 2024 following the Ayrmid license.

•
General and administrative expenses for the year ended December 31, 2024 were $6.3 million, similar to the year ended December 31, 2023.

•
Net non-operating income amounted to $18.4 million for the year ended December 31, 2024, compared to net non-operating expenses of $10.8 million for the year ended December 31, 2023. Non-operating income for the year ended December 31, 2024 primarily relates to non-cash, fair-value adjustments of warrant liabilities on the Company’s balance sheet, as a result of changes in the Company’s share price, offset by warrant offering expenses. Non-operating expenses for the year ended December 31, 2023 primarily relate to non-cash, fair-value adjustments of warrant liabilities on the Company’s balance sheet.

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Net financial expenses amounted to $7.3 million for the year ended December 31, 2024, compared to net financial expenses of $0.1 million for the year ended December 31, 2023. Net financial expenses for both periods primarily relate to interest paid on loans, which increased in 2024 due to a one-time $4.0 million charge to interest expense in connection with the November 2024 amendment to the loan agreement with BlackRock, partially offset by investment income earned on bank deposits.

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Net loss for the year ended December 31, 2024 was $9.2 million, compared to $60.6 million for the year ended December 31, 2023.

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As of December 31, 2024, the Company had cash, cash equivalents, and short-term bank deposits of $19.6 million (approximately $29.0 million on a pro-forma basis, following the financing completed at the beginning of January 2025).

A copy of the Company’s annual report on Form 20-F for the year ended December 31, 2024 has been filed with the U.S. Securities and Exchange Commission at View Source and posted on the Company’s investor relations website at View Source Company will deliver a hard copy of its annual report, including its complete audited consolidated financial statements, free of charge, to its shareholders upon request at [email protected].

Conference Call and Webcast Information
To access the conference call, please dial +1-888-281-1167 from the U.S. or +972-3-918-0685 internationally. A live webcast and a replay of the call can be accessed through the event page on the Company’s website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast. The call replay will be available approximately two hours after completion of the live conference call. A dial-in replay of the call will be available until April 2, 2025; please dial +1-888-295-2634 from the US or +972-3-925-5904 internationally.

On March 31, 2025 Marker Therapeutics, Inc. (Nasdaq: MRKR), a clinical-stage immuno-oncology company focusing on developing next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumors, reported corporate updates and financial results for the year ended December 31, 2024 (Press release, Marker Therapeutics, MAR 31, 2025, View Source [SID1234651680]).

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"In 2024, we made substantial progress advancing MT-601, our lead multi antigen recognizing (MAR)-T cell therapy, and laid the groundwork for continued momentum in 2025," said Juan Vera, M.D., President and Chief Executive Officer of Marker Therapeutics. "Preliminary data from our Phase 1 APOLLO study showed encouraging safety and efficacy results in lymphoma patients who relapsed after anti-CD19 CAR-T cell therapy. With a 78% objective response rate and favorable safety profile, we believe MT-601 has the potential to provide a transformative treatment option for this patient population. We look forward to sharing additional insights during a webinar in the second quarter of 2025."

"We also strengthened our financial position through a strategic private placement and additional non-dilutive funding from the NIH and CPRIT. As we move further into 2025, our focus remains on cash preservation and disciplined execution to maximize the impact of our clinical programs," concluded Dr. Vera.

2024 PROGRAM UPDATES & OPERATIONAL HIGHLIGHTS

MT-601 (Lymphoma)

- MT-601, Marker’s lead MAR-T cell therapy, is being evaluated in the nationwide multicenter Phase 1 APOLLO study (clinicaltrials.gov identifier: NCT05798897) in patients with anti-CD19 CAR-T relapsed lymphoma or where CAR-T cells are not an option.

- The Company provided an update on the APOLLO study (Press Release, December 19, 2024). Key findings from the study include:

o Safety: MT-601 was well tolerated across all study participants. No immune-effector cell associated neurotoxicity syndrome (ICANS) and one case of Grade 1 cytokine release syndrome (CRS) were observed. No dose limiting toxicities (DLTs) have been reported to date.

o Efficacy: In the first dose cohort, 7 out of 9 patients achieved objective responses (78%) at first response assessment, with 4 patients demonstrating complete response (CR; 44.4%).

o Time in Follow-Up: Three patients have been followed for 6 to 12 months, with ongoing follow-up underway. All study participants are monitored closely to ensure comprehensive data collection and patient safety.

- The Company is enrolling additional study participants in the Phase 1 APOLLO trial and expects to report further data in the second half of 2025.

MT-601 (Pancreatic)

- Marker received $2 million from NIH SBIR and $9.5 million from CPRIT to support the development of MT-601 in metastatic pancreatic cancer.

- Clinical program launch is anticipated in the second half of 2025.

MT-401-OTS (Acute Myeloid Leukemia or Myelodysplastic Syndrome)

- The Company previously secured non-dilutive funding to support the clinical investigation of MT-401 as an "Off-the-Shelf" (MT-401-OTS) product in patients with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS). MT-401-OTS is manufactured from healthy donors and a cellular inventory has been established with ongoing efforts to expand.

- The Company anticipates clinical program initiation during the second half of 2025.

2024 CORPORATE HIGHLIGHTS

- Announced clinical pipeline prioritization in January 2024 to strategically focus on MT-601 in patients with lymphoma. This announcement also included program updates that highlighted the potential of the Company’s MT-401-OTS program for patients with AML (Press Release, January 8, 2024).

- The United States Adopted Names (USAN) and International Nonproprietary Names (INN) committees approved "neldaleucel" as the nonproprietary (generic) name for MT-601.

- On December 23, 2024, the Company announced a $16.1 million private placement to support the clinical advancements of the Phase 1 APOLLO study. The financing involved participation from new and existing investors, including esteemed firms such as Blue Owl, New Enterprise Associates (NEA) and Aisling Capital.

FISCAL YEAR 2024 FINANCIAL HIGHLIGHTS

Cash Position and Guidance: At December 31, 2024, Marker had cash and cash equivalents of $19.2 million. The Company believes that its existing cash and cash equivalents will fund its operating expenses into the first quarter of 2026, assuming no additional grant funds are received. We anticipate receiving additional grant funding, which we expect could extend our runway beyond Q1 2026.

R&D Expenses: Research and development expenses were $13.5 million for the year ended December 31, 2024, compared to $10.4 million for the year ended December 31, 2023.

G&A Expenses: General and administrative expenses were $4.2 million for the year ended December 31, 2024, compared to $7.5 million for the year ended December 31, 2023.

Net Loss: Marker reported a net loss of $10.7 million for the year ended December 31, 2024, compared to a net loss of $8.2 million for the year ended December 31, 2023.

About MAR-T cells

The multi-antigen recognizing (MAR) T cell platform (formerly known as multiTAA-specific T cells) is a novel, non-genetically modified cell therapy approach that selectively expands tumor-specific T cells from a patient’s/donor’s blood capable of recognizing a broad range of tumor antigens. Unlike other T cell therapies, MAR-T cells allow the recognition of hundreds of different epitopes within up to six tumor-specific antigens, thereby reducing the possibility of tumor escape. Since MAR-T cells are not genetically engineered, Marker believes that its product candidates will be easier and less expensive to manufacture, with an improved safety profile compared to current engineered T cell approaches, and may provide patients with meaningful clinical benefits.

On March 31, 2025 IDEAYA Biosciences, Inc. (NASDAQ: IDYA), a precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation (BTD) for darovasertib, a potential first-in-class protein kinase C (PKC) inhibitor, for the neoadjuvant treatment of adult patients with primary uveal melanoma (UM) for whom enucleation has been recommended (Press release, Ideaya Biosciences, MAR 31, 2025, View Source [SID1234651699]).

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"We are pleased to receive FDA Breakthrough Therapy designation as we prepare to advance neoadjuvant darovasertib into a potential Phase 3 registrational trial in patients with primary UM. This designation highlights the potential of monotherapy darovasertib in a patient population with significant unmet medical need where there are currently no FDA-approved systemic therapies," said Dr. Darrin Beaupre, M.D., Ph.D., Chief Medical Officer of IDEAYA Biosciences. "We are targeting to present the updated Phase 2 clinical data in neoadjuvant UM that was provided as part of the BTD application at multiple medical conferences in 2025," said Yujiro S. Hata, President and Chief Executive Officer, IDEAYA Biosciences.

This U.S. FDA BTD designation, follows the Fast Track designation granted by the U.S. FDA for evaluation of darovasertib in combination with crizotinib in adult patients being treated for metastatic uveal melanoma (MUM), where a Phase 2/3 registration-enabling trial of the darovasertib and crizotinib combination in 1L HLA-A2-negative MUM is ongoing. Darovasertib has also been designated as an Orphan Drug by the U.S. FDA in UM, including in MUM, entitling IDEAYA to certain potential tax credits, exemptions from user fees, and statutory marketing exclusivity.

The BTD application was supported by updated interim clinical data from an ongoing Phase 2 open-label trial (NCT05907954) evaluating darovasertib monotherapy in the neoadjuvant setting for localized UM. IDEAYA presented interim clinical data demonstrating an 82% ocular tumor shrinkage rate and a 61% eye preservation rate in UM patients in September 2024 (press release), and neoadjuvant UM data was also presented as an oral presentation at ASCO (Free ASCO Whitepaper) 2024. Updated clinical data in neoadjuvant UM, including efficacy, safety, radiation reduction, eye preservation, and vision preservation / improvement on treatment, were submitted as part of the BTD application that we plan to present at medical conferences in 2025. Multiple clinical data updates in neoadjuvant UM and MUM, including median overall survival (mOS) from the Phase 2 study (IDE196-001), are targeted to be presented at medical conferences in mid-year 2025 and the second half of 2025. A median progression free survival (mPFS) readout for the Phase 2/3 registration-enabling trial of the darovasertib and crizotinib combination in 1L HLA-A2-negative MUM is targeted by year-end 2025. The Company also intends to initiate a Phase 3 randomized registrational trial in neoadjuvant UM in the first half of 2025.

A potential Phase 3 registrational study would evaluate neoadjuvant darovasertib in primary UM patients who are eligible for enucleation (Cohort 1) or plaque brachytherapy (Cohort 2). Neoadjuvant UM has a projected annual incidence for North America, Europe, and Australia of approximately 12,000 patients, and is a high unmet medical with no FDA approved systemic therapies.

BTD is designed to expedite the development and regulatory review of promising therapies for serious or life-threatening conditions where preliminary clinical evidence suggests substantial improvement over existing treatments. The designation facilitates more intensive FDA guidance, cross-disciplinary collaboration, and eligibility for rolling submission and priority review.