On November 13, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), reported an enrollment update with 60% of the target number of subjects for the first planned interim unblinding of data having consented to its pivotal Phase 2B/3 "MIRACLE" study of Annamycin in combination with cytarabine for the treatment of adult patients with acute myeloid leukemia (AML) who are refractory to or relapsed (R/R) after induction therapy (R/R AML). The targeted number for the first unblinding of data is 45 subjects. Additional subjects continued to be identified by site investigators. This update is as of November 4, 2025, as identification and recruitment are ongoing. The Company expects to complete treatment of the first 45 subjects in the first quarter of 2026 with the initial unblinding of data thereafter.

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Walter Klemp, Chairman and CEO of Moleculin, commented, "We’re very encouraged by the strong momentum in recruitment and enthusiasm I’ve personally heard from investigators around Europe and the US. To date, we are seeing blinded response activity tracking within our expected range, based on historical responses of the trial arm equivalents. Although enrollment at some sites in Europe has been impacted by bed shortages, the MIRACLE study continues to progress as planned. As we move toward our first unblinding milestone, we are excited about Annamycin’s potential to fill a major gap in AML treatment. We believe we’re well on our way to determining if Annamycin has the potential to offer a much-needed, safer, and more effective option for patients facing this devastating disease."

MIRACLE Trial Progress and Next Steps

The MIRACLE study (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a Phase 2B/3, global multi-center, randomized, double-blind, placebo-controlled, adaptive designed clinical trial whereby data from the 2B (Part A) portion will be combined with the Phase 3 (Part B) portion for purposes of measuring its primary efficacy endpoint. The protocol for the MIRACLE trial 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 treated with Annamycin (190mg/m2 and 230 mg/m2) in combination with HiDAC and 15 subjects treated with just HiDAC plus placebo.

The Company expects to reach the recruitment and treatment of the first 45 subjects in the first quarter of 2026 with unblinding thereafter, in addition to the second unblinding, which is expected in the first half of 2026. This accelerated estimated timeline is due in part to the positive response the Company received in meetings during December with potential investigators regarding recruitment for the trial.

The currently enrolled subjects are from sites across five countries, providing a diverse base of subjects. With the upcoming holidays in addition to unexpected bed shortages at certain EU sites, the Company expects treatment of enrolled subjects to slow, pushing the completion of the first 45 subjects into the first quarter of 2026. Such data will be audited, locked, and reviewed prior to release. The release of the unblinded data will be thereafter.

For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756. Additionally, the clinical trial in the EU is on euclinicaltrials.eu and the reference identifier there is 2024-518359-47-00.

Annamycin, also known by its non-proprietary name of naxtarubicin, 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. Annamycin also benefits from composition of matter patent protection through 2040 with the potential to extend that protection as far as 2045. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the EMA.

(Press release, Moleculin, NOV 13, 2025, View Source [SID1234659907])

On November 13, 2025 Merck (NYSE: MRK), known as MSD outside of the United States and Canada, reported that Jannie Oosthuizen, president, Human Health U.S., and Dr. Marjorie Green, senior vice president and head of oncology, global clinical development, Merck Research Laboratories, are scheduled to participate in a fireside chat at the Jefferies Global Healthcare Conference in London on Thursday, Nov. 20, 2025, at 9:30 a.m. ET / 2:30 p.m. GMT.

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Investors, analysts, members of the media and the general public are invited to listen to a live audio webcast of the presentation at this weblink.

(Press release, Merck & Co, NOV 13, 2025, View Source [SID1234659906])

On November 13, 2025 Lantern Pharma Inc. (NASDAQ: LTRN), a clinical-stage biopharmaceutical company leveraging its proprietary RADR artificial intelligence (AI) and machine learning (ML) platform to transform the cost, pace, and timeline of oncology drug discovery and development, reported operational highlights and financial results for the third quarter 2025 ended September 30, 2025, and provided an update on its portfolio of AI-driven drug candidates and AI platform, RADR.

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"The third quarter represented a transformational period for Lantern Pharma as we announced successful enrollment completion of our LP-184 Phase 1a trial, achieving all primary endpoints with unique clinical benefit observations in multiple hard-to-treat solid tumors," said Panna Sharma, CEO & President of Lantern Pharma. "The observed 48% clinical benefit rate at or above the therapeutic dose threshold, combined with the favorable safety profile and clear biomarker signals, validates our AI-driven, precision medicine approach and positions us to advance multiple planned high-value Phase 1b/2 trials. Simultaneously, our productive FDA Type C meeting provides a clear regulatory pathway for our pediatric CNS cancer program under our subsidiary Starlight Therapeutics. Additionally, the interest generated for LP-284 at the LL&M Congress underscores the commercial potential across our pipeline. We are executing with discipline and focus as we advance toward pivotal value-creation milestones in multiple oncology indications."

Clinical Pipeline Developments

LP-184: Detailed Phase 1a Results Demonstrate Clinical Proof-of-Concept & Activity

In September, Lantern announced the completion of enrollment and initial clinical results from its LP-184 Phase 1a clinical trial (NCT05933265), which successfully achieved all primary endpoints. The data across the 63 patients enrolled provided critical insights into safety, pharmacokinetics, biomarker correlations, and clinical activity that position LP-184 for advancement into targeted planned Phase 1b/2 studies.

Highlights of the Phase 1a Results:

● Clinical Benefit and Activity: The trial demonstrated clinical benefit in 48% of evaluable cancer patients treated at or above the therapeutic dose threshold. This encouraging activity was observed in heavily pretreated patients who had exhausted available standard-of-care therapies, representing proof-of-concept for LP-184’s synthetic lethal mechanism.
● Safety Profile Supports Broad Development: LP-184 demonstrated a favorable safety and tolerability profile with minimal dose-limiting toxicities. The safety data support advancement into both monotherapy and combination therapy approaches with PARP inhibitors and immunotherapy agents.

● Activity in Difficult-to-Treat Cancers: Notable clinical benefits were observed in historically challenging tumor types including glioblastoma multiforme (GBM), gastrointestinal stromal tumor (GIST), and thymic carcinoma. Several patients have continued treatment beyond enrollment completion due to ongoing clinical benefit.
● Biomarker Strategy Strengthened: A key finding from the Phase 1a trial was the observation of marked tumor reductions in patients harboring specific DNA damage repair mutations, including CHK2, ATM, BRCA1, and STK11/KEAP1 alterations. These biomarker insights directly validate the AI-driven patient stratification approach developed through Lantern’s RADR platform and support the use of genomic selection criteria in future trials.
● Pharmacokinetics Enable Dose Optimization: The trial successfully characterized LP-184’s pharmacokinetic profile and established the recommended Phase 2 dose (RP2D) of 0.39 mg/kg, providing clear dosing guidance for planned Phase 1b/2 studies across multiple indications.

Based on the Phase 1a results and biomarker insights, Lantern is advancing development plans for LP-184 in three high-value indications:

● Triple-Negative Breast Cancer (TNBC), which represents a potential annual market opportunity exceeding $4 billion.
● NSCLC with STK11/KEAP1 Co-mutations: Phase 1b/2 study in a biomarker-defined subset of patients with mutations in STK11 and/or KEAP1 genes, representing a significant unmet medical need and a potential annual market approaching $1.5 billion.
● Bladder Cancer with DNA damage repair mutations in patients who have relapsed from SOC (standard of care) therapies, which is planned to be an investigator-led study initiating in Denmark.

Comprehensive results from the LP-184 Phase 1a trial are being prepared for submission to peer-reviewed journals and presentation at major oncology conferences. To provide additional insights and expert analysis of the clinical data, Lantern will host a Key Opinion Leader (KOL)-hosted scientific webinar on November 20, 2025 at 4:30 p.m. Eastern Time featuring detailed discussion of the Phase 1a results, biomarker findings, and clinical development strategy.

FDA Type C Meeting: Clarity in Regulatory Path for Pediatric CNS Cancer Program

A major third quarter regulatory milestone was the successful completion of a Type C meeting with the U.S. Food and Drug Administration during September. This meeting provided important guidance on the regulatory pathway and trial design for Starlight Therapeutics’ – a wholly owned subsidiary of Lantern Pharma – planned pediatric clinical trial focused on CNS cancers, including Atypical Teratoid Rhabdoid Tumor (ATRT). The FDA provided constructive and supportive feedback on the proposed clinical trial structure.

Key outcomes from the Type C meeting included:

● Parallel ATRT Cohort Supported: The FDA confirmed support for a parallel cohort design specifically for ATRT patients, which will accelerate data collection in this ultra-rare pediatric population while maintaining statistical rigor.
● Combination Strategy Confirmed: The agency confirmed the potential incorporation of spironolactone as a combination agent with LP-184/STAR-001. This combination strategy is based on preclinical data demonstrating synergistic activity and RADR platform predictions of enhanced efficacy in pediatric brain tumors.
● Trial Design Alignment: The FDA provided guidance on appropriate endpoints, patient selection criteria, and safety monitoring approaches for this vulnerable pediatric population, enabling Starlight Therapeutics to finalize the clinical protocol with confidence in the regulatory pathway.

Regulatory Designations and Market Opportunity:

● LP-184, which is being developed as STAR-001 by Starlight Therapeutics in CNS cancers, has received both Rare Pediatric Disease Designation and Orphan Drug Designation from the FDA for ATRT, along with additional Rare Pediatric Disease Designations for hepatoblastoma, rhabdomyosarcoma, and malignant rhabdoid tumors. These designations underscore the urgent unmet need for innovative therapies in aggressive pediatric cancers and provide potential pathways for possible priority review vouchers upon approval.
● ATRT is an ultra-rare pediatric brain tumor with the genetic hallmark of SMARCB1 gene loss or dysfunction, affecting primarily children under age 3. Current treatment options are limited and associated with significant long-term toxicities, creating substantial demand for novel targeted approaches.

LP-300 HARMONIC Trial: Enrollment and Follow-Up Progress

The Phase 2 HARMONIC trial continued patient enrollment and follow-up during the third quarter across sites in the United States, Japan, and Taiwan. The trial evaluates LP-300 in combination with standard-of-care chemotherapy (carboplatin + pemetrexed) in never-smokers with NSCLC adenocarcinoma who have progressed after tyrosine kinase inhibitor therapy.

● In late July, Lantern announced the completion of enrollment in Japan for the HARMONIC trial across five clinical sites in Japan, including the National Cancer Center Tokyo.
● During November, clinical investigators associated with the HARMONIC trial presented data from the ongoing study at the 66th Annual Meeting of the Japan Lung Cancer Society. Dr. Jonathan Dowell from UT Southwestern Medical Center presented, "A Phase II Trial of LP-300 plus Carboplatin and Pemetrexed in TKI-Progressed NSCLC Patients". Data from this presentation included the Asian and US cohorts of the study and will be further reviewed and presented in December by Lantern Pharma.

● Also, during the third quarter the company progressed with a change in clinical staffing and a transition of CRO services in Asia focused on significant cost reductions and efficiency in Taiwan.
● The study’s strategic positioning in Asia, where never-smokers represent 33-40% of NSCLC cases compared to approximately 15% in Western populations, positions Lantern for potential regional partnerships and co-development opportunities. The treatment of never-smokers with NSCLC represents an estimated $4+ billion annual market opportunity with no specifically approved therapies for this patient population.

LP-284: LL&M Congress Presentation & Future Development Plans

During October Lantern presented clinical data from its ongoing LP-284 Phase 1 trial at the 25th Annual Lymphoma, Leukemia & Myeloma (LL&M) Congress in New York City. The presentation showcased the confirmed complete metabolic response in a heavily pretreated DLBCL patient and highlighted LP-284’s novel mechanism of action and combination therapy potential.

LL&M Congress Impact and Additional Milestones:

● Presentation generated interest from biopharmaceutical companies and clinical investigators, with ongoing discussions focused on: combination therapy development with FDA-approved agents; post-immunotherapy treatment strategies; and LP-284’s mechanistic differentiation. LP-284 has demonstrated particular lethality in cells with deficient DNA damage response, a targetable vulnerability in NHL.
● Additional clinical sites being recruited with a focus on NHL and high-grade B-cell lymphoma patients to accelerate enrollment.
● Partnership and collaboration discussions advancing with emphasis on combination therapy protocols.
● Expansion into autoimmune and inflammatory indications under preclinical evaluation, leveraging LP-284’s B-cell depletion activity.

LP-284 benefits from strong intellectual property protection with composition of matter patents granted in the U.S., Europe, Japan, India, and Mexico, providing exclusivity through 2039. The drug candidate has received multiple FDA Orphan Drug Designations including for mantle cell lymphoma and high-grade B-cell lymphomas.

RADR AI Platform: Demonstrating Commercial Value and Industry Leadership

AI for Biology and Medicine Symposium: Showcasing Platform Capabilities

A key highlight of the third quarter was Lantern’s presentation at the inaugural AI for Biology and Medicine symposium at the University of North Texas on October 30, 2025. This presentation demonstrated the commercial readiness and real-world applicability of two RADR platform modules. An additional large-scale rollout of a multi-agentic system focused on addressing drug development and research needs in rare cancers that leverages Lantern’s unique approach to developing therapies and approaches in rare and orphan cancers is underway. This initiative is planned to be made public during December with broader industry rollout in early 2026.

predictBBB.ai Platform – Best-in-Class BBB Prediction:

● This ensemble machine learning model achieves 94.1% accuracy for blood-brain barrier permeability prediction and can screen 200,000 molecular candidates in under one week. Lantern’s algorithms currently hold five of the top eleven positions on the Therapeutic Data Commons Leaderboard, establishing clear technological leadership.
● The platform addresses a critical pharmaceutical challenge: only 2-6% of small-molecule drugs successfully cross the blood-brain barrier. The BBB technologies market is projected to grow from $1.4 billion in 2023 to $9.85 billion by 2032, representing significant commercial opportunity.

LBx-AI Liquid Biopsy Platform – Predictive Biomarker Discovery:

● This AI-powered liquid biopsy analysis platform has achieved 86% accuracy for predicting treatment response in non-small cell lung cancer and has demonstrated a 0.76 Pearson correlation for PD-L1 level inference from circulating tumor DNA analysis. This capability enables non-invasive patient stratification and real-time treatment monitoring.
● Lantern has entered into additional collaborations with leading research and cancer centers to further strengthen and validate this module in other cancers.

RADR Platform Impact Across Pipeline

The third quarter developments underscore RADR’s central role in Lantern’s drug development success:

● Biomarker Discovery: RADR predictions of LP-184 sensitivity in CHK2, ATM, and STK11/KEAP1-mutated cancers were validated in the Phase 1a trial, demonstrating the platform’s promise for identifying responsive patient populations.
● Combination Therapy Identification: RADR analysis identified LP-184’s synergy with PARP inhibitors and immunotherapy, as well as LP-284’s synergy with rituximab, directly informing clinical development strategies and partnership discussions.

● Development Efficiency: On average, Lantern’s newly developed AI-guided drug programs have advanced from initial insights to first-in-human trials in 2-3 years at approximately $1.0-2.5 million per program, demonstrating significant cost and time advantages over traditional development approaches.

Financial Results for Third Quarter 2025

Balance Sheet: Cash, cash equivalents, and marketable securities were approximately $12.4 million as of September 30, 2025, compared to approximately $24.0 million as of December 31, 2024. The company believes that its existing cash, cash equivalents, and marketable securities on hand as of the date of this press release will enable it to fund anticipated operating expenses and capital expenditure requirements into approximately Q3 2026.

Research and Development Expenses: R&D expenses were approximately $2.4 million for the quarter ended September 30, 2025, compared to approximately $3.7 million for the quarter ended September 30, 2024. The decrease was primarily attributable to decreases in research studies and materials of approximately $1,032,000 relating to the conduct and support of clinical trials, decreases in consulting expenses of approximately $55,000 and decreases in payroll and compensation expenses of approximately $224,000. This was partially offset by increases in licensing expenses of approximately $31,000.

General and Administrative Expenses: G&A expenses were approximately $1.9 million for the quarter ended September 30, 2025, compared to approximately $1.5 million for the quarter ended September 30, 2024. The increase was primarily attributable to increases in business development and investor relations expenditures of approximately $321,000, increases in other professional fees of approximately $57,000, and increases in patent costs of approximately $37,000.

Net Loss: Net loss was approximately $4.2 million (or $0.39 per share) for the quarter ended September 30, 2025, compared to a net loss of approximately $4.5 million (or $0.42 per share) for the quarter ended September 30, 2024.

Capitalization: As of September 30, 2025, the Company had approximately 11.0 million shares of common stock outstanding. Options to purchase approximately 1.2 million shares of common stock at a weighted average exercise price of $5.74 per share were outstanding. As of September 30, 2025 there were no warrants outstanding.

In July 2025, the Company entered into an ATM Sales Agreement ("ATM"), with ThinkEquity LLC ("ThinkEquity"), as sales agent, pursuant to which the Company may offer and sell up to $15,530,000 of its common stock from time to time, in "at-the-market" offerings to or through its sales agent. During the quarter ended September 30, 2025, we sold 212,444 shares of common stock under the ATM for the gross proceeds of $989,061. Between October 1, 2025 and the date of this press release, we have sold an additional 144,204 shares of common stock under the ATM for the gross proceeds of $634,333.

Upcoming Milestones and Corporate Developments

Looking ahead to the fourth quarter of 2025 and early 2026, Lantern expects several key value-creation catalysts:

Immediate Near-Term (Q4 2025):

● November 20, 2025 at 4:30 p.m. ET: KOL hosted scientific webinar on LP-184 Phase 1a detailed results and clinical development strategy.
● December 2025: LP-300 further patient follow-up and clinical data.
● Q4 2025: Continued commercial developments for AI platform modules, including the multi-agentic system for rare cancer drug development.

Early 2026 Catalysts:

● Q1 2026: Planned Pediatric CNS cancer trial initiation through Starlight Therapeutics subsidiary (IND amendment submission)
● Q1 2026: Planned initiation of LP-184 Phase 1b/2 trials in TNBC and NSCLC (subject to funding)
● H1 2026: Investigator-led bladder cancer trial initiation in Denmark
● 2026: Additional HARMONIC trial data readouts and potential partnership announcements
● Scale up of AI platform commercial efforts
● Preparation for potential capital formation activities to support clinical advancement

Conference Call Information

Lantern Pharma will host a conference call and webcast to discuss third quarter 2025 financial results and business updates on Thursday, November 13, 2025 at 9:00 a.m. Eastern Time.

To participate in the conference call, please register at the Zoom webcast link. A replay of the earnings call webcast will be available after the call on the investor relations section of Lantern’s website at ir.lanternpharma.com.

KOL-Hosted LP-184 Scientific Webinar: In addition to the earnings call, Lantern will host a scientific webinar featuring key opinion leader analysis of the LP-184 Phase 1a results on November 20, 2025 at 4:30 p.m. Eastern Time. Please register at the Zoom webcast link.

(Press release, Lantern Pharma, NOV 13, 2025, View Source [SID1234659905])

On November 13, 2025 LabGenius Therapeutics ("LabGenius"), a drug discovery company combining artificial intelligence (AI) and high-throughput experimentation to advance next-generation multispecific antibodies for solid tumours, reported a scientific poster will be presented at the ESMO (Free ESMO Whitepaper) Immuno-Oncology Congress 2025, being held December 10 – 12, 2025, at the Queen Elizabeth II Centre in London, United Kingdom. LabGenius’ presentation will debut the pre-clinical in vivo efficacy (>90% tumour growth inhibition) and tolerability data for their lead asset, a highly tumour selective bispecific T-cell engager (TCE).

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Poster Presentation Details

Title Novel Selectivity-Enhanced Bispecific T-cell Engager Utilises Avidity to Overcome On-target, Off-tumour Toxicity
Date and time Wednesday, December 10, 2025, 08:00 (GMT)
Poster number 309P
Location The Churchill Room, Queen Elizabeth II Centre, London
Bispecific TCE Overview

For the selected target, LabGenius has developed a selectivity-enhanced TCE. The company’s lead optimisation platform, EVA, was used to discover and concomitantly optimise a TCE with improved killing selectivity, potency, efficacy, and manufacturability. The biological mechanism underlying this enhanced selectivity is based on the principle of avidity. By harnessing avidity-driven selectivity, the optimised TCE can distinguish between healthy and diseased cells based on differential tumour-associated antigen expression.

(Press release, LabGenius Therapeutics, NOV 13, 2025, View Source [SID1234659904])

On November 13, 2025 Kura Oncology, Inc. (Nasdaq: KURA) and Kyowa Kirin Co., Ltd. (TSE: 4151) reported the U.S. Food and Drug Administration (FDA) has granted full approval of KOMZIFTI (ziftomenib) for adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible NPM1 mutation who have no satisfactory alternative treatment options. KOMZIFTI is the first and only once-daily, oral menin inhibitor approved for R/R NPM1-mutated (NPM1-m) AML, a devastating blood cancer with limited treatment options.

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"KOMZIFTI combines compelling efficacy, a favorable safety profile, compatibility with concomitant medications, and convenient once-daily oral administration in a population with few effective treatment options. These features highlight KOMZIFTI’s potential to serve as the menin inhibitor of choice in its approved indication," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "Together with our partner, Kyowa Kirin, we remain committed to advancing development of KOMZIFTI across the treatment continuum for AML, where its best-in-class profile offers potential for even greater impact in combination regimens and earlier lines of therapy. We are fully prepared to launch KOMZIFTI today and deliver this new medicine to patients in need."

NPM1 mutations are among the most common founder mutations in AML, occurring in approximately 30% of cases. Historically, approximately 20% of patients with NPM1-m AML do not respond to front-line therapy. Of those who do respond, 70% will relapse within 3 years, most within 12 months. Early relapse and declining survival with each recurrence underscore the urgent need for treatment approaches that deliver lasting remission.

Approval is supported by the pivotal KOMET-001 trial (NCT04067336), which evaluated KOMZIFTI’s safety and efficacy in 112 R/R NPM1-m AML patients. The rate of complete remission (CR) plus CR with partial hematologic recovery (CRh) was 21.4% (95% CI: 14.2, 30.2). The median duration of CR+CRh was 5.0 months (95% CI: 1.9, 8.1) and the median time to first response in patients who achieved a CR or CRh was 2.7 months (range: 0.9 to 15 months). 88% of patients who achieved CR or CRh did so within 6 months of initiating KOMZIFTI. These data from the Prescribing Information are generally consistent with findings recently published in the Journal of Clinical Oncology.1

"KOMZIFTI addresses a critical need for adult patients with R/R NPM1-m AML, many of whom are older and unable to tolerate intensive chemotherapy or transplant," said Eunice Wang, M.D., Chief of the Leukemia Service and Professor of Oncology at Roswell Park Comprehensive Cancer Center. "The clinical data demonstrate deep and durable responses with a manageable safety profile, including no drug-drug interactions and no Boxed Warnings for QTc prolongation or Torsades de Pointes – key advantages for patients on multiple concurrent medications. This approval equips physicians with a new oral therapy to integrate into care and improve outcomes for this vulnerable patient population."

The most common adverse reactions (≥20%), including laboratory abnormalities, were aspartate aminotransferase increased, infection without an identified pathogen, potassium decreased, albumin decreased, alanine aminotransferase increased, sodium decreased, creatinine increased, alkaline phosphatase increased, hemorrhage, diarrhea, nausea, fatigue, edema, bacterial infection, musculoskeletal pain, bilirubin increased, potassium increased, differentiation syndrome, pruritus, febrile neutropenia, and transaminases increased. KOMZIFTI includes a Boxed Warning for differentiation syndrome, a well-studied mechanism-based risk in drugs that restore differentiation. Absence of clinically meaningful drug-drug interactions can ease the use of KOMZIFTI with concomitant therapies, including those that cause QTc interval prolongation. QTc interval prolongation was ≤ Grade 3 in 12% of patients and no Grade 4 or Grade 5 QTc interval prolongation was reported. QTc interval prolongation of any cause occurred in 10% of the 70 patients 65 years of age or older.

"The approval of KOMZIFTI underscores our commitment to advancing precision medicines to address the genetic drivers of disease in hematology and oncology," said Takeyoshi Yamashita, Ph.D., Executive Vice President and Chief Medical Officer of Kyowa Kirin. "In AML, where many patients face severe disease progression and limited treatment options, the evolution toward targeted therapies such as KOMZIFTI represents a major step forward and offers potential to transform existing standards of care. We are proud to partner with Kura Oncology in bringing this important therapy to patients and their families."

In November 2024, Kura Oncology and Kyowa Kirin entered into a global strategic collaboration to develop and commercialize KOMZIFTI. The collaboration builds on Kyowa Kirin’s leadership and expertise in hematologic malignancies. Under the terms of the collaboration, Kura leads development, regulatory and commercial strategy in the U.S. and is responsible for manufacturing KOMZIFTI. The companies will jointly perform certain commercialization activities in accordance with a co-created U.S. territory commercialization plan. Outside the U.S., Kyowa Kirin leads development, regulatory and commercial strategy and is responsible for commercializing KOMZIFTI.

As part of its commitment to helping patients access KOMZIFTI, Kura has established a support program, Kura RxKonnect, to minimize barriers to access and reimbursement for appropriate patients prescribed KOMZIFTI. Kura RxKonnect is available 8:00 am – 8:00 pm ET by phone at (844) KuraPSP (844-587-2777) or online at www.KuraRxKonnect.com.

Conference Call and Webcast
Kura will host a conference call and webcast featuring Company management and guest speaker Eunice Wang, M.D., Chief of the Leukemia Service and Professor of Oncology at Roswell Park Comprehensive Cancer Center, at 12:30 pm ET / 9:30 am PT on November 13, 2025. The live webcast and replay will be available on the Company’s website at www.kuraoncology.com under the Investors tab in the Events and Presentations section.

About KOMZIFTI
KOMZIFTI (ziftomenib) is an oral menin inhibitor approved for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible NPM1 mutation who have no satisfactory alternative treatment options.

KOMZIFTI is in development for the front-line treatment of AML harboring NPM1 mutations, KMT2A translocations and FLT3 mutations, with the potential to be combined with approved therapies and benefit a broad spectrum of patients earlier in their disease course.

IMPORTANT SAFETY INFORMATION FOR KOMZIFTI FROM THE U.S. PRESCRIBING INFORMATION

Boxed WARNING: DIFFERENTIATION SYNDROME

Differentiation syndrome, which can be fatal, has occurred with KOMZIFTI. Signs and symptoms may include fever, joint pain, hypotension, hypoxia, dyspnea, rapid weight gain or peripheral edema, pleural or pericardial effusions, pulmonary infiltrates, acute kidney injury, and rashes. If differentiation syndrome is suspected, interrupt KOMZIFTI, and initiate oral or intravenous corticosteroids with hemodynamic and laboratory monitoring until symptom resolution; resume KOMZIFTI upon symptom improvement.

WARNINGS AND PRECAUTIONS

Differentiation Syndrome

KOMZIFTI can cause fatal or life-threatening differentiation syndrome (DS). DS is associated with rapid proliferation and differentiation of myeloid cells. Symptoms of DS, including those seen in patients treated with KOMZIFTI, may include fever, hypoxia, joint pain, hypotension, dyspnea, rapid weight gain or peripheral edema, pleural or pericardial effusions, acute kidney injury, and rashes.

In the clinical trial, DS occurred in 29 (26%) of 112 patients with R/R AML with an NPM1 mutation who were treated with KOMZIFTI at the recommended dosage. DS was Grade 3 in 13% and fatal in two patients. In broader evaluation of all patients with any genetic form of AML treated with KOMZIFTI monotherapy in clinical trials, DS occurred in 25% of patients. Four fatal cases of DS occurred out of 39 patients with KMT2A-rearranged AML treated with KOMZIFTI. KOMZIFTI is not approved for use in patients with KMT2A-rearranged AML.

In the 112 patients with an NPM1 mutation, DS was observed with and without concomitant hyperleukocytosis, in as early as 3 days and up to 46 days after KOMZIFTI initiation. The median time to onset was 15 days. Two patients experienced more than one DS event. Treatment was interrupted and resumed in 15 (13%) patients, while it was permanently discontinued in 2 (2%) patients.

Prior to starting treatment with KOMZIFTI, reduce the WBC counts to less than 25 x 10⁹/L. If DS is suspected, interrupt KOMZIFTI, initiate oral or intravenous corticosteroids (e.g., dexamethasone 10 mg every 12 hours) for a minimum of 3 days with hemodynamic and laboratory monitoring. Resume treatment with KOMZIFTI at the same dose level when signs and symptoms improve and are Grade 2 or lower. Taper corticosteroids over a minimum of 3 days after adequate control or resolution of symptoms. Symptoms of DS may recur with premature discontinuation of corticosteroid treatment.

QTc Interval Prolongation

KOMZIFTI can cause QTc interval prolongation. In the clinical trial, QTc interval prolongation was reported as an adverse reaction in 12% of 112 patients treated with KOMZIFTI at the recommended dosage for R/R AML with an NPM1 mutation. QTc interval prolongation was Grade 3 in 8% of patients. The heart-rate corrected QT interval (using Fridericia’s method) (QTcF) was greater than 500 msec in 9% of patients, and the increase from baseline QTcF was greater than 60 msec in 12% of patients. KOMZIFTI dose reduction was required for 1% of patients due to QTc interval prolongation. QTc prolongation occurred in 14% of the 42 patients less than 65 years of age and in 10% of the 70 patients 65 years of age or older.

Correct electrolyte abnormalities, including hypokalemia and hypomagnesemia, prior to treatment with KOMZIFTI. Perform an ECG prior to initiation of treatment with KOMZIFTI, and do not initiate KOMZIFTI in patients with QTcF > 480 msec. Perform an ECG at least once weekly for the first four weeks on treatment, and at least monthly thereafter. Interrupt KOMZIFTI if the QTc interval is > 500 ms or the change from baseline is > 60 ms (Grade 3). In patients with congenital long QTc syndrome, congestive heart failure, electrolyte abnormalities, or those who are taking medications known to prolong the QTc interval, more frequent ECG monitoring may be necessary. Concomitant use of KOMZIFTI with drugs known to prolong the QTc interval may increase the risk of QTc interval prolongation, result in a greater increase in the QTc interval and adverse reactions associated with QTc interval prolongation, including Torsades de Pointes, other serious arrhythmias, and sudden death.

Embryo-Fetal Toxicity

Based on findings in animals and its mechanism of action, KOMZIFTI can cause embryo-fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to the fetus. Advise females of reproductive potential to use effective contraception during treatment with KOMZIFTI and for 6 months after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with KOMZIFTI and for 3 months after the last dose.

ADVERSE REACTIONS

Fatal adverse reactions occurred in 4 (4%) patients who received KOMZIFTI, including 2 with differentiation syndrome, 1 with infection, and 1 with sudden death. Serious adverse reactions were reported in 79% of patients who received KOMZIFTI. Serious adverse reactions occurring in ≥ 5% of patients included infection without an identified pathogen (29%), febrile neutropenia (18%), bacterial infection (16%), differentiation syndrome (16%), and dyspnea (6%).

Dosage interruption of KOMZIFTI due to an adverse reaction occurred in 54% of patients. Adverse reactions that required dose interruption in ≥ 2% of patients included infection without an identified pathogen (15%), differentiation syndrome (13%), febrile neutropenia (5%), pyrexia (4%), electrocardiogram QT prolonged (4%), leukocytosis (4%), bacterial infection (3%), cardiac failure (2%), cholecystitis (2%), diarrhea (2%), pruritus (2%), and thrombosis (2%). Dose reduction of KOMZIFTI due to an adverse reaction occurred in 4% of patients. Permanent discontinuation of KOMZIFTI due to an adverse reaction occurred in 21% of patients. Adverse reactions that required permanent discontinuation of KOMZIFTI in ≥ 2% of patients were infection without an identified pathogen (8%), bacterial infection (4%), cardiac arrest (2%), and differentiation syndrome (2%).

Most common (≥ 20%) adverse reactions, including laboratory abnormalities, were aspartate aminotransferase increased (53%), infection without an identified pathogen (52%), potassium decreased (52%), albumin decreased (51%), alanine aminotransferase increased (50%), sodium decreased (49%), creatinine increased (45%), alkaline phosphatase increased (41%), hemorrhage (38%), diarrhea (36%), nausea (35%), fatigue (34%), edema (30%), bacterial infection (28%), musculoskeletal pain (28%), bilirubin increased (27%), potassium increased (26%), differentiation syndrome (26%), pruritus (23%), febrile neutropenia (22%), and transaminases increased (21%).

DRUG INTERACTIONS

Drug interactions may occur when KOMZIFTI is concomitantly used with:

Strong or Moderate CYP3A4 Inhibitors: Monitor patients more frequently for KOMZIFTI-associated adverse reactions.
Strong or Moderate CYP3A4 Inducers: Avoid concomitant use of KOMZIFTI.
Gastric Acid Reducing Agents: Avoid concomitant use of KOMZIFTI with proton pump inhibitors (PPIs), H2 receptor antagonists (H2RAs), or locally acting antacids. If concomitant use with H2RAs or locally acting antacids cannot be avoided, modify KOMZIFTI administration time.
Take KOMZIFTI 2 hours before or 10 hours after administration of an H2 receptor antagonist.
Take KOMZIFTI 2 hours before or 2 hours after administration of a locally acting antacid.
Drugs that Prolong the QTc Interval: Avoid concomitant use of KOMZIFTI. If concomitant use cannot be avoided, obtain ECGs when initiating, during concomitant use, and as clinically indicated. Interrupt KOMZIFTI if the QTc interval is > 500 ms or the change from baseline is > 60 ms.
USE IN SPECIFIC POPULATIONS

Pregnancy: Based on findings in animals and its mechanism of action, KOMZIFTI can cause embryo-fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Verify pregnancy status in females of reproductive potential prior to starting KOMZIFTI.

Lactation: Because of the potential for adverse reactions in the breastfed child, advise women not to breastfeed during treatment with KOMZIFTI and for 2 weeks after the last dose.

Infertility: Based on findings in animals, KOMZIFTI may impair fertility in females and males of reproductive potential.

Please see full Prescribing Information, including Boxed WARNING.

NPM1, nucleophosmin 1, KMT2A, lysine methyltransferase 2A, FLT3, Fms-like tyrosine kinase 3

(Press release, Kura Oncology, NOV 13, 2025, View Source [SID1234659903])