Pembrolizumab

Biomarker-activated cancer drug-candidate LP-184 demonstrates encouraging efficacy signals in DNA damage repair deficient tumors with an acceptable safety and tolerability profile – meeting all primary endpoints. LP-184 demonstrated clinical benefit in multiple highly aggressive cancers with a 54% disease control rate at or above therapeutic dose levels. Multiple Phase 1b/2 clinical trials are now being planned across cancer indications and supported by 3 FDA Orphan Drug, 2 FDA Fast Track Designations and a Rare Pediatric Disease Designation. Detailed webinar with management and KOL from Fox Chase Cancer Center highlighting the drug-candidate and patient insights can be found at "Inside The Data" . DALLAS--(BUSINESS WIRE)--Lantern Pharma Inc. (NASDAQ: LTRN), a clinical-stage biotechnology company using artificial intelligence and genomics to transform oncology drug development, announced additional details and clinical insights from its completed Phase 1a dose-escalation study of LP-184 as well as highlights from its recent webinar. The clinical trial demonstrated encouraging durable disease control in 63 heavily pre-treated patients with advanced solid tumors, many of which had DNA damage repair (DDR) pathway deficiencies. The clinical trial met all primary endpoints for safety, tolerability, and established a clear recommended phase 2 dose (RP2D). Building on these encouraging Phase 1a results, Lantern is advancing an ambitious precision oncology development strategy featuring multiple biomarker‑guided Phase 1b/2 clinical trials in triple‑negative breast cancer (TNBC), glioblastoma multiforme (GBM), non‑small cell lung cancer (NSCLC), and advanced urothelial carcinoma (bladder cancer). The company and independent industry analysts estimate that the aggregate annual market opportunity for LP‑184 across these and additional targeted indications could exceed $10 billion. KEY HIGHLIGHTS: Phase 1a Trial Successfully Completed with Encouraging Efficacy Signals: 63 heavily pre-treated patients (median 3+ prior therapies) with advanced solid tumors enrolled; trial achieved 54% disease control rate in patients at or above therapeutic dose levels, demonstrating promising activity in DNA damage repair-deficient cancers Exceptional Durability in Difficult-to-Treat Cancers: Patients with stage 4 squamous lung cancer (BRCA1 mutation), thymic carcinoma (CHEK2 mutation), and gastrointestinal stromal tumor (ATM mutation) remain on treatment with 12+ to 23+ months of ongoing clinical benefit and meaningful tumor reductions after failing multiple prior therapies. Addresses Major Market Opportunity in Precision Oncology: LP-184 targets the estimated 20-25% of solid tumor patients with DDR deficiencies—representing more than 400,000 cases annually and a market potential exceeding $10 billion in annual drug sales. AI-Driven Biomarker Strategy Strengthened: Lantern's RADR ® platform identified PTGR1 as a key predictive biomarker; >87% of Phase 1a patients (recurrent, advanced solid tumors) exceeded the bioactivation threshold, with a diagnostic-ready molecular assay enabling patient selection and/or stratification. Strong Regulatory Support Accelerates Development: Five FDA designations (3 Orphan Drug, 2 Fast Track) for LP-184 across TNBC, GBM, pancreatic cancer, and rare pediatric tumors provide development advantages and potential expedited pathways to approval. Multi-Indication Phase 1b/2 Program Are Advancing: Biomarker-guided trials are being planned and advancing in markets with high patient need: triple-negative breast cancer (monotherapy + PARP inhibitor combination); drug and IO resistant, PDL-1 low, non-small cell lung cancer (NSCLC); recurrent glioblastoma (GBM) ( with +spironolactone); and advanced recurrent DDR deficient bladder cancer. PHASE 1A CLINICAL HIGHLIGHTS The Phase 1a study enrolled 63 patients with advanced solid tumors who had received a median of three prior lines of therapy. Key findings include: Established Recommended Phase 2 Dose (RP2D): 0.39 mg/kg administered intravenously on Days 1 and 8 of a 21-day cycle Favorable Safety Profile: Main adverse events included reversible transaminitis, nausea/vomiting, and thrombocytopenia—all clinically manageable and consistent with the alkylating agent class High Therapeutic Index: Therapeutic plasma concentrations achieved three dose levels below RP2D, indicating a wide therapeutic window (~2.45-fold) Encouraging Disease Control: 54% disease control rate and 8% clinical benefit rate at ≥6 months in DDR-altered tumors Biomarker Validation: More than 87% of Phase 1a patients exceeded the PTGR1 bioactivation threshold, confirming the biomarker's utility for patient selection REMARKABLE PATIENT DURABILITY SIGNALS CLINICAL POTENTIAL Several patients with DDR pathway alterations in the Phase 1a study achieved exceptional, ongoing responses despite having progressed through multiple prior standard-of-care therapies: A 62-year-old male patient with stage 4 squamous NSCLC harboring a BRCA1 alteration, who had failed radiation and durvalumab, began LP-184 treatment in December 2023 and continues on therapy with more than 23 months of clinical benefit (Cycle 34 ongoing) and 22% target lesion reduction A 50-year-old male patient with stage 4 thymic carcinoma with a CHEK2 alteration, who had progressed through four prior lines including pembrolizumab, started LP-184 in November 2023 and remains on treatment with more than 12 months of benefit (Cycle 17 ongoing) and 26% target lesion reduction A 62-year-old female patient with stage 4 gastrointestinal stromal tumor (GIST) harboring an ATM alteration, who had failed four prior therapies including sunitinib, initiated LP-184 in November 2023 and continues treatment with more than 12 months of benefit (Cycle 19 ongoing) and 9% tumor reduction These durability signals in heavily pre-treated, genomically-defined patient populations underscore LP-184's potential as a precision oncology therapy and support the company's biomarker-driven development strategy. AI-GUIDED PRECISION MEDICINE APPROACH Lantern's proprietary RADR® artificial intelligence platform played a central role in LP-184's development, identifying prostaglandin reductase-1 (PTGR1) overexpression and low expression of multiple DDR genes as strong predictors of LP-184 sensitivity. LP-184 functions as a prodrug that is selectively activated inside cancer cells by PTGR1, which is frequently overexpressed in tumors. Upon activation, LP-184 forms a highly reactive metabolite that breaks apart the DNA of the cancer cell and induces interstrand cross-links and double-strand breaks—damage that cannot be repaired in tumors with deficient DDR pathways, resulting in selective cancer-cell death while sparing normal cells. Critically, LP-184 demonstrates activity across both homologous recombination (HR)-deficient and nucleotide excision repair (NER)-deficient tumors, potentially addressing a broader patient population than PARP inhibitors, which primarily target HR deficiency. Preclinical data show LP-184 is active even in PARP inhibitor-resistant models, highlighting its differentiated mechanism. Lantern has developed a diagnostic-ready RT-qPCR assay for PTGR1 expression in FFPE tumor tissue, enabling patient selection for ongoing and future trials consistent with a precision oncology approach. REGULATORY MOMENTUM SUPPORTS ACCELERATED DEVELOPMENT LP-184 has received six FDA designations recognizing its potential to address serious unmet medical needs: Fast Track Designation for triple-negative breast cancer (TNBC) Fast Track Designation for glioblastoma multiforme (GBM) (granted October 2024) Orphan Drug Designation for malignant gliomas Orphan Drug Designation for pancreatic cancer Orphan & Pediatric Rare Disease Drug Designation for atypical teratoid rhabdoid tumors (ATRT) These designations provide significant regulatory and development advantages, including more frequent interactions with the FDA, rolling submission of New Drug Application (NDA) sections, and potential eligibility for Accelerated Approval and Priority Review—mechanisms designed to expedite patient access to promising therapies in areas of high unmet need. AMBITIOUS MULTI-INDICATION DEVELOPMENT PROGRAM IN DEVELOPMENT & UNDERWAY Building on the Phase 1a foundation, Lantern has initiated and is planning to initiate and enroll multiple biomarker-guided Phase 1b/2 trials and investigator-sponsored studies: Triple-Negative Breast Cancer (TNBC): Monotherapy and LP-184 plus olaparib (PARP inhibitor) combination arms in HR-deficient TNBC patients (n≈60-64 total). TNBC represents approximately 15% of all breast cancers with limited targeted therapy options and poor prognosis in the metastatic setting. Advanced Drug-Resistant, Non-Small Cell Lung Cancer (NSCLC): LP-184 plus nivolumab and ipilimumab (dual checkpoint inhibitors) in patients with KEAP1/STK11 mutations and PD-L1 <50% (n≈34)—a genomically-defined subset with poor response to immunotherapy and chemotherapy alone. Advanced Urothelial Carcinoma: LP-184 monotherapy investigator-sponsored trial in Denmark in patients with PTGR1-high expression and NER/HR pathway deficiencies (n≈27-39). Recurrent Glioblastoma (GBM): LP-184 plus spironolactone combination (n≈38-39), targeting the first potential new GBM therapy in more than 20 years. GBM affects more than 13,000 U.S. patients annually with median survival of 12-15 months and virtually no effective options at recurrence. Trial initiation expected in early 2025 under the wholly owned subsidiary, Starlight Therapeutics . Lantern is also reviewing additional development opportunities beyond these core Phase 1b/2 programs. Post-radiation pancreatic cancer represents a particularly compelling indication, as radiation therapy has been shown to upregulate tumoral PTGR1 expression, potentially enhancing LP-184's tumor-selective activation. The company is also further exploring optimized dosing schedules to maximize therapeutic impact in certain solid tumors. MANAGEMENT COMMENTARY The trial met all primary endpoints for safety and tolerability and established a clear Recommended Phase 2 Dose with a wide therapeutic window. "These are patients who had exhausted standard options, yet several remain on LP-184 with meaningful clinical benefit in challenging cancers one to two years later," said Panna Sharma, CEO of Lantern Pharma. "This durability in genomically selected, end-stage cancer patients is encouraging and directly validates the predictive power of our RADR® AI platform and the PTGR1 biomarker. With a diagnostic-ready molecular assay for PTGR1, strong enthusiasm from key opinion leaders and multiple FDA designations in hand, Lantern plans on advancing LP-184 into multiple precision Phase 1b/2 trials targeting indications with aggregate annual U.S. market potential exceeding $10 to 12 billion.” WEBINAR: INSIDE THE DATA, AN IN-DEPTH DISCUSSION OF THE LP-184 SCIENCE, CLINICAL TRIAL RESULTS, AND FUTURE DEVELOPMENT PLANS For a comprehensive review of LP-184's mechanism of action, detailed Phase 1a clinical data, patient case studies, and the company's development strategy, Lantern Pharma invites stakeholders to view the recent "Inside The Data" webinar featuring management and a Key Opinion Leader from Fox Chase Cancer Center. The webinar provides in-depth scientific context and clinical insights that complement this announcement and is available on Lantern Pharma's YouTube channel at: https://youtu.be/yyAaQZvMx6I?si=nRvjbW9wWiVLtVrp About LP-184 LP-184 is a next-generation acylfulvene that is synthetically lethal and designed to selectively target solid tumors with DNA damage repair pathway deficiencies. As a prodrug activated by the enzyme PTGR1, LP-184 induces irreparable DNA damage in cancer cells while sparing normal tissue. The compound has demonstrated nanomolar potency in preclinical models and encouraging durability in clinical studies in heavily pre-treated patients. LP-184 has received FDA Fast Track Designation for TNBC and GBM, and Orphan Drug Designation for malignant gliomas, pancreatic cancer, and ATRT. About Lantern Pharma Lantern Pharma (NASDAQ: LTRN) is a clinical-stage biotechnology company using artificial intelligence, machine learning, and genomic data to streamline oncology drug development and bring precision therapies to patients who need them. The company's proprietary RADR® AI platform integrates billions of data points to identify biomarkers, predict drug response, and design smarter clinical trials. Lantern's clinical-stage pipeline includes LP-184 (alkylating agent), LP-300 (PTPN11 inhibitor), and LP-284 (PKC-beta inhibitor), each targeting genomically-defined patient populations. For more information, visit www.lanternpharma.com. Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These forward-looking statements include, among other things, statements relating to: future events or our future financial performance; the potential advantages of our RADR® platform in identifying drug candidates and patient populations that are likely to respond to a drug candidate; our strategic plans to advance the development of our drug candidates and antibody drug conjugate (ADC) development program; estimates regarding the development timing for our drug candidates and ADC development program; expectations and estimates regarding clinical trial timing and patient enrollment; our research and development efforts of our internal drug discovery programs and the utilization of our RADR® platform to streamline the drug development process; our intention to leverage artificial intelligence, machine learning and genomic data to streamline and transform the pace, risk and cost of oncology drug discovery and development and to identify patient populations that would likely respond to a drug candidate; estimates regarding patient populations, potential markets and potential market sizes; sales estimates for our drug candidates and our plans to discover and develop drug candidates and to maximize their commercial potential by advancing such drug candidates ourselves or in collaboration with others. Any statements that are not statements of historical fact (including, without limitation, statements that use words such as “anticipate,” “believe,” “contemplate,” “could,” “estimate,” “expect,” “intend,” “seek,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “target,” “model,” “objective,” “aim,” “upcoming,” “should,” “will,” “would,” or the negative of these words or other similar expressions) should be considered forward-looking statements. There are a number of important factors that could cause our actual results to differ materially from those indicated by the forward-looking statements, such as (i) the risk that we may not be able to secure sufficient future funding when needed and as required to advance and support our existing and planned clinical trials and operations, (ii) the risk that observations in preclinical studies and early or preliminary observations in clinical studies do not ensure that later observations, studies and development will be consistent or successful, (iii) the risk that our research and the research of our collaborators may not be successful, (iv) the risk that we may not be successful in licensing potential candidates or in completing potential partnerships and collaborations, (v) the risk that none of our product candidates has received FDA marketing approval, and we may not be able to successfully initiate, conduct, or conclude clinical testing for or obtain marketing approval for our product candidates, (vi) the risk that no drug product based on our proprietary RADR® AI platform has received FDA marketing approval or otherwise been incorporated into a commercial product, and (vii) those other factors set forth in the Risk Factors section in our Annual Report on Form 10-K for the year ended December 31, 2024, filed with the Securities and Exchange Commission on March 27, 2025. You may access our Annual Report on Form 10-K for the year ended December 31, 2024 under the investor SEC filings tab of our website at http://www.lanternpharma.com/ or on the SEC’s website at http://www.sec.gov/. Given these risks and uncertainties, we can give no assurances that our forward-looking statements will prove to be accurate, or that any other results or events projected or contemplated by our forward-looking statements will in fact occur, and we caution investors not to place undue reliance on these statements. All forward-looking statements in this press release represent our judgment as of the date hereof, and, except as otherwise required by law, we disclaim any obligation to update any forward-looking statements to conform the statement to actual results or changes in our expectations.