On March 24, 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 cancers and viruses, reported its financial results for the fiscal year ended December 31, 2024 (Press release, Moleculin, MAR 24, 2025, View Source [SID1234651367]). As previously announced, the Company will host a conference call and live audio webcast to discuss the operational and financial results at 8:30 AM ET on Monday, March 24, 2025 (details below).

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"This last year proved to be a very productive and substantial year for Moleculin. We believe that we continue to successfully execute on activities for our MIRACLE trial, supported by a growing body of positive preliminary data and encouraging interactions with FDA and clinical sites," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "Looking ahead, we look forward to an exciting year with a number of potential value-driving milestones expected in the coming months. Importantly, with the clinical and regulatory progress made to date, we still expect to unblind preliminary data from the first 45 subjects in the second half of this year. Our team is dedicated to advancing the development of Annamycin and we look forward to providing additional updates."

Recent Highlights

Expanded global exclusivity for Annamycin with Notice of Intent to Grant for the European patent application titled, "Method of Reconstituting Liposomal Annamycin";
Received positive FDA guidance for acceleration of its registration-enabling MIRACLE trial for R/R Acute Myeloid Leukemia (AML) resulting in a smaller number of subjects in MIRACLE;
Received first country regulatory approval in Europe to begin enrolling for the MIRACLE trial;
Received US Institutional Review Board (IRB) approval for pivotal, adaptive Phase 3 clinical trial (the "MIRACLE" trial) and engaged a leading contract research organization (CRO); and,
Announced new preclinical findings demonstrating significant activity of Annamycin in Venetoclax resistant AML model.
Clinical Development Update

Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML)

The Company is currently 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") in 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 global, including sites in the US, Europe and the Middle East.

The MIRACLE study is a Phase 2B/3 clinical trial whereby data from the 2B portion will be combined with the Phase 3 portion. MIRACLE is subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, utilizes an adaptive design whereby the first 75 to 90 subjects will be randomized (1:1:1) in Part A of the trial to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, which Annamycin doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting. The amended protocol allows for the unblinding of preliminary primary efficacy data (Complete Remission or CR) and safety/tolerability of the three arms at 45 subjects, in addition to the conclusion of Part A (at 75 to 90 subjects). The first early unblinding will yield 30 subjects with Annamycin (190mg/m2 and 230/m2) and HiDAC and 15 subjects with just HiDAC. The Company expects to reach the first unblinding (45 subjects) in the second half of 2025, in addition to the second unblinding, which is expected in the first half of 2026. This accelerated estimated timeline is due to the positive response the Company received in meetings during December with potential investigators regarding recruitment for the trial.

Early activities include: Multiple first subjects in Ukraine are being screened and we expect to begin treatment by the end of March; Corresponding ethic committee approvals were received recently in Georgia and Egypt with the appropriate regulatory approvals remaining; and, multiple site evaluation and initiation visits are occurring and/or being scheduled.

For Part B of the trial, approximately 220 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin (randomized 1:1). The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative. For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756.

Expected Milestones for Annamycin AML Development Program

1Q – 3Q 2025 – Update on MIRACLE trial site selection/approvals by countries
1Q 2025 – First subject enrolled and treated in MIRACLE trial
2025 – Recruitment update for MIRACLE trial
2H 2025 – Data readout (n=45) unblinded efficacy/safety review
2H 2025 – 2026 – Impact of data readout (n=45) on regulatory pathway; Recruitment update
1H 2026 – Interim efficacy and safety data (n=~75-90) unblinded and Optimum Dose set for MIRACLE trial
2027 – Begin enrollment of 3rd line subjects in MIRACLE2
2027 – Enrollment ends in 2nd line subjects
2028 – Primary efficacy data for 2nd line subjects in MIRACLE
2028 – Begin submission of a Rolling New Drug Application (NDA) for the treatment of R/R AML for accelerated approval on primary endpoint of CR from MIRACLE
2028 – Primary efficacy data for 2nd line subjects
2028 – Rolling NDA submission begins
Soft Tissue Sarcoma (STS) Lung Metastases

As previously announced, the Company completed enrollment in the Phase 2 portion of its U.S. Phase 1B/2 clinical trial evaluating Annamycin as monotherapy for the treatment of soft tissue sarcoma lung metastases. Subjects who had stable disease at the time of study discontinuation were followed for progression free response and overall survival. The clinical study report is finalized but not yet filed and is expected to be released by the end of April 2025.

Expected Milestones for Annamycin STS Lung Mets Development Program

1H 2025 – Final MB-107 data readout
2025 – Identify next phase of development / pivotal IIT (investigator-initiated-trial) program
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).

Summary of Financial Results for the Full Year 2024

Research and development (R&D) expense was $17.7 million and $19.5 million for the years ended December 31, 2024 and 2023, respectively. The decrease in R&D of $1.8 million is mainly related to the $1.5 million WPD sublicense termination in 2023, which enabled the reacquisition of our intellectual property rights in certain territories, including parts of the European Union.

General and administrative (G&A) expenses were $8.8 million and $10.0 million for the years ended December 31, 2024 and 2023, respectively. The decrease in G&A of $1.2 million was mainly attributable to a decrease in regulatory and legal services, and consulting & advisory fees.

As of December 31, 2024, the Company had cash and cash equivalents of $4.3 million. The Company believes that the existing cash and cash equivalents as of December 31, 2024, along with $9.3 million in gross proceeds received as part of the February 2025 financing activities will be sufficient to fund our planned operations into the third quarter of 2025.

Conference Call and Webcast

Moleculin management will host its quarterly conference call and webcast for investors, analysts, and other interested parties on Monday, March 24, 2024 at 8:30 AM ET.

Interested participants and investors may access the conference call by dialing (877) 407-0832 (domestic) or (201) 689-8433 (international) and referencing the Moleculin Biotech Conference Call. The live audio webcast will be accessible on the Events page of the Investors section of the Moleculin website, moleculin.com, and will be archived for 90 days.

On March 24, 2025 CStone Pharmaceuticals (stock code: 2616.HK), an innovation-driven biopharmaceutical company focused on developing oncology drugs, reported that it has submitted a new indication application (NDA) for sugemalimab to the European Medicines Agency (EMA) for the treatment of patients with unresectable Stage III non-small cell lung cancer (NSCLC) who have not progressed after concurrent or sequential chemoradiotherapy (CRT) (Press release, CStone Pharmaceauticals, MAR 24, 2025, View Source [SID1234656219]). This is the second indication CStone has advanced in the European market, following its initial EMA approval in 2024 for the first-line treatment of metastatic squamous and non-squamous NSCLC. If approved, sugemalimab will address a critical unmet need in Stage III NSCLC, becoming the second PD-(L)1 antibody approved for this indication in Europe . Furthermore, sugemalimab’s dual application in both Stage III and Stage IV NSCLC will further solidify its position as a core immunotherapy for lung cancer.

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This application is based on a multicenter, randomized, double-blind Phase III clinical study called GEMSTONE-301 , which aims to evaluate the efficacy and safety of sugemalimab as consolidation therapy for patients with unresectable Stage III NSCLC after CRT. The results of the GEMSTONE-301 study have been published in The Lancet Oncology, and the results show that:

Sugemalimab can significantly prolong patients’ progression-free survival (PFS) and reduce the risk of disease progression or death by 36%;
A clear trend toward overall survival (OS) benefit, with a 56% reduction in the risk of death ;
Consistent clinical benefits were observed regardless of whether patients received concurrent or sequential chemoradiotherapy;
The safety profile was good and no new risk signals were found.
Dr. Jianxin Yang, CEO, President of R&D, and Executive Director of CStone Pharmaceuticals, said: "Following the successful approval of sugemalimab for the treatment of Stage IV NSCLC in Europe, we have been working closely with the EMA to actively expand its indication for earlier-stage lung cancer and other tumor types. Based on its outstanding efficacy and favorable safety profile, sugemalimab has the potential to address the unmet clinical needs of patients with Stage III NSCLC. Furthermore, we will continue to deepen our international strategic collaborations and work closely with regulatory agencies to increase global access to sugemalimab and ensure that this innovative therapy can benefit patients worldwide as soon as possible."

About Sugemalimab

Sugemalimab was developed by CStone Pharmaceuticals using the OmniRat transgenic animal platform licensed from US-based Ligand Corporation . This platform enables the one-stop production of fully human antibodies. As a fully human, full-length anti-PD-L1 monoclonal antibody, sugemalimab closely resembles the natural G-type immunoglobulin 4 (IgG4) response in humans, minimizing the potential risk of immunogenicity and related toxicities in patients. This offers unique advantages over similar drugs.

The European Commission (EC) and the UK Medicines and Healthcare Products Regulatory Agency (MHRA) have approved sugemalimab in combination with platinum-based chemotherapy for the first-line treatment of patients with metastatic NSCLC who do not have sensitizing EGFR mutations or genomic tumor alterations in ALK, ROS1, or RET.

Currently, the China National Medical Products Administration (NMPA) has approved five indications for Sugemalimab (Zegemet ) :

First-line treatment in combination with chemotherapy for metastatic non-squamous NSCLC and metastatic squamous NSCLC without EGFR or ALK gene mutations;
For the treatment of patients with unresectable, stage III NSCLC who have not experienced disease progression after concurrent or sequential chemoradiotherapy;
Treatment of patients with relapsed or refractory extranodal NK/T-cell lymphoma;
Combined with fluorouracil and platinum chemotherapy drugs as the first-line treatment for patients with unresectable locally advanced, recurrent or metastatic esophageal squamous cell carcinoma;
In combination with fluorouracil-containing and platinum-based chemotherapy, it is used for the first-line treatment of unresectable locally advanced or metastatic gastric and gastroesophageal junction adenocarcinoma expressing PD-L1 CPS ≥ 5.

On March 24, 2025 Onconetix, Inc., (Nasdaq: ONCO) ("Onconetix" or the "Company"), (formerly Blue Water Biotech, Inc. (BWV)), a cancer diagnostics company focused on the research, development and commercialization of innovative solutions for oncology, reported that new clinical data of Proclarix was presented on March 23, 2025 during the 2025 European Association of Urology (EAU) congress, which data further demonstrates the strong clinical performance of Proclarix in a Danish cohort (Press release, Onconetix, MAR 24, 2025, View Source [SID1234651368]).

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The primary approach for early detection of prostate cancer is through testing serum levels of prostate-specific antigen (PSA). However, given the limited cancer specificity of PSA, Proclarix is addressing the urgent need for improved diagnosis by lowering the burden of potential over-detection of clinically insignificant prostate cancer resulting in unnecessary biopsies.

In the study presented, Proclarix was evaluated in prospectively collected samples from 808 patients with suspected prostate cancer. In the challenging subpopulation including 371 patients with an enlarged prostate and thus frequently presenting elevated PSA levels leading to false-positive results, a negative Proclarix test resulted in a probability of 5% or less for clinically significant cancer outperforming other diagnostic tools like %fPSA (14%, p=0.028) and the ERSPC (European Randomised Study of Screening for Prostate Cancer) risk calculator (20%, p=0.026). Proclarix avoided most biopsies (22%) and missing the least significant cancers (3 out of 101 patients). In the extended population including 654 patients with a PSA level of 2-20 ng/ml, the clinical performance of Proclarix was confirmed with a sensitivity of 96% and a significantly higher (p<0.001) specificity compared to both %fPSA and ERSPC risk calculator.

Primary investigator and presenter of the study, Ahmed H. Zedan, MD, PhD from Lillebaelt Hospital – University Hospital of Southern Denmark said: "Proclarix can be safely used to reduce performed biopsies by ruling out patients with clinically insignificant or no prostate cancer and by minimizing the risk of missing clinically significant cancer".

About Proclarix

Proclarix is CE-certified under In Vitro Diagnostic Regulation ("IVDR") and indicated for prostate cancer diagnosis in patients with normal digital rectal exam (DRE), enlarged prostate volume and elevated levels of PSA at 2-10 ng/ml. Proclarix is a risk score combining in-vitro assays for the quantitative detection of biomarkers with a proprietary algorithm to assess a patient’s risk of having clinically significant prostate cancer. Detection of prostate cancer-related biomarkers in blood serum using the Proclarix risk score has been demonstrated in multiple clinical studies to be a reliable indicator of the presence of clinically significant prostate cancer. Proclarix is included in both the European (EAU) and American (AUA) guidelines.

On March 24, 2025 Artiva Biotherapeutics, Inc. (Nasdaq: ARTV) (Artiva), a clinical-stage biotechnology company whose mission is to develop effective, safe, and accessible cell therapies for patients with devastating autoimmune diseases and cancers, reported financial results for the full year ended December 31, 2024, and highlighted recent progress (Press release, Artiva Biotherapeutics, MAR 24, 2025, View Source [SID1234651394]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"2024 was a transformational year for Artiva including initiating dosing of AlloNK in patients with autoimmune disease across our trials, a successful initial public offering strengthening our balance sheet, and expanding key leadership across the organization with expertise in cell therapy and autoimmune disease," said Fred Aslan, M.D., CEO of Artiva. "We look forward to sharing initial data from our AlloNK program in autoimmune disease this year. We also look forward to sharing updated clinical data from our non-Hodgkin’s lymphoma (NHL) trial with AlloNK which continues to mature as one of the strongest data sets for the allogeneic field, demonstrating deep B-cell depletion, continued durability of response, and the compatibility of our treatment regimen with outpatient administration."

Recent Business Highlights

Corporate and Financial Updates

•
Expanded Board of Directors: In January 2025, Artiva appointed Dan Baker, M.D., as an independent member of its Board of Directors. Dr. Baker brings over two decades of drug development experience in the pharmaceutical industry. He is currently the interim Chief Development Officer of Cue Biopharma, Inc., and previously held a 19-year tenure at Johnson & Johnson (Janssen/Centocor) most recently as the Vice President of Immunology R&D.
•
Bolstered Key Development Leadership: Artiva appointed key leadership with cell therapy and autoimmune expertise across the development organization, including Benjamin Dewees as Senior Vice President (SVP), Regulatory Affairs, David Moriarty, Ph.D., as SVP, Clinical Operations, and Feng Xu as SVP, Biometrics. Collectively, the leadership team brings experience developing therapies targeting autoimmune indications including systemic lupus erythematosus (SLE), lupus nephritis (LN), rheumatoid arthritis (RA) and Sjogren’s disease from their tenures at companies such as Kyverna Therapeutics, Inc., Horizon Therapeutics plc and IGM Biosciences, Inc.
Upcoming Milestones

•
Initial data for AlloNK (also known as AB-101) on autoimmune indications from at least one of the following trials expected in H1 2025:
o
Artiva Sponsored Trial in SLE/ LN: Ongoing Phase 1/1b trial evaluating AlloNK in combination with rituximab or obinutuzumab in patients with SLE with or without LN.
o
Ongoing IIT Basket Trial: Investigator-initiated basket trial (IIT) assessing the safety, tolerability, and clinical activity of AlloNK plus rituximab in patients with RA, pemphigus vulgaris, granulomatosis with polyangiitis/microscopic polyangiitis, and SLE. The trial is being conducted by Integral Rheumatology & Immunology Specialists, a community rheumatology clinic.
•
Updated clinical data from the Phase 1/2 trial exploring AlloNK + rituximab in patients with relapsed/refractory B-cell NHL showing continued durability of response to be presented at a medical conference in 2025

Full Year 2024 Financial Results

•
Cash, Cash Equivalents and Investments. As of December 31, 2024, Artiva had cash, cash equivalents, and investments of $185.4 million. This includes $179.0 million in gross proceeds from Artiva’s completed initial public offering in July 2024 in which it sold 14,920,000 shares of its common stock, including partial exercise of the overallotment option. Existing cash, cash equivalents, and investments as of December 31, 2024, are expected to fund operations at least through the end of 2026.
•
Collaboration Revenue. Collaboration revenue was zero for the year ended December 31, 2024, compared to $32.9 million for the year ended December 31, 2023.
•
Research and Development Expenses. Research and development expenses were $50.3 million for each of the years ended December 31, 2024 and 2023.
•
General and Administrative Expenses. General and administrative expenses were $17.2 million for the year ended December 31, 2024, compared to $13.9 million for the year ended December 31, 2023.
•
Other Income, net. Other income, net, was $1.9 million for the year ended December 31, 2024, compared to other income, net, of $2.0 million for the year ended December 31, 2023.
•
Net Loss. Net loss totaled $65.4 million for the year ended December 31, 2024, as compared to net income of $28.7 million for the year ended December 31, 2023, with non-cash stock-based compensation expense of $7.0 million and $7.1 million for the years ended December 31, 2024 and 2023, respectively.

On March 24, 2025 Pasithea Therapeutics Corp. (NASDAQ: KTTA) ("Pasithea" or the "Company"), a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic MEK inhibitor, for the treatment of neurofibromatosis type 1 (NF1) and other cancer indications, reported its Vice President of Business Development, Mathew Lazarus, will be presenting at the 2025 Cancer Advocacy Group of Louisiana ("CAGLA") NeauxCancer Conference taking place on March 27-29, 2025 at the The Roosevelt Hotel in New Orleans (Press release, Pasithea Therapeutics, MAR 24, 2025, View Source [SID1234651369]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Mr. Lazarus’s presentation will provide an overview of Pasithea’s next generation macrocyclic MEK inihibitor, PAS-004, including interim safety, tolerability and pharmacokinetic (PK) data from the Company’s ongoing open-label dose escalation Phase 1 study in advanced cancer patients with MAPK pathway-driven advanced tumors. He will discuss the Company’s latest progress in clinical development and how Pasithea is advancing its next-generation molecule to transform cancer and NF1 treatment.

Details of the presentation are as follows:

Event: 2025 CAGLA NeauxCancer Conference
Date: March 28, 2025
Time: 11:00 AM CDT
Location: The Roosevelt New Orleans
Webcast: CAGLA 2025 Livestreams

The CAGLA NeauxCancer Conference is a premier gathering of leading oncology researchers, industry executives, and medical professionals focused on groundbreaking developments in cancer treatment and care.

For more information about the conference, visit View Source