On February 13, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, reported it has received U.S. Food and Drug Administration ("FDA") feedback and guidance on its IND amendment that has allowed a reduction in the size of its Phase 3 pivotal trial protocol 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") for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML) (MB-108) (Press release, Moleculin, FEB 13, 2025, View Source [SID1234650256]). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) will be a global trial, including sites in the US, Europe and the Middle East.

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"Given the importance of our Miracle trial as a pivotal registration clinical trial, we were extremely pleased to receive detailed follow up from the FDA regarding our IND amendment to enable the trial. While all of the major aspects of the trial remain unchanged, guidance from FDA recommended an alteration to the statistical plan that will allow us to reduce the size of Part B of our trial by approximately 10%. Moreover, the nature of the feedback helps us move forward quickly to open sites in the US, in addition to the sites we are expecting to open in Europe and the Middle East. All of this supports the pursuit of an accelerated timeline for new drug approval," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin.

Mr. Klemp added, "The spirit of collaboration surrounding the Miracle trial is, frankly, inspiring. From investigating clinicians to regulatory authorities around the world, people are starting to realize the potential significance of approving the first-ever non-cardiotoxic anthracycline. Annamycin’s approval in AML alone would bring the potential to save thousands of lives every year, and the opportunity to eliminate the threat of cardiotoxicity in a wide range of tumors equates to as much as 20 times that in long term potential. It’s critical to remember that nearly half of all cancers are treated with an anthracycline and 60% of all childhood cancers. One of the things that drives us every day is our belief that no child should ever have to be subjected to a cardiotoxic anthracycline once Annamycin is approved."

The MIRACLE study, 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). This 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.

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.

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).

On February 13, 2025 Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-care biopharmaceutical company, reported its financial results for the full year 2024 (Press release, Ipsen, FEB 13, 2025, View Source [SID1234650253]).

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Extract of consolidated results4 for FY 2024 and FY 2023:4

FY 2024 FY 2023 % change

€m €m Actual CER1
Total Sales 3,400.6 3,127.5 8.7% 9.9%
Core Operating Income 1,109.4 1,001.0 10.8%
Core operating margin 32.6% 32.0% +0.6pts
Core Consolidated Net Profit 857.8 765.5 12.1%
Core earnings per share (fully diluted) €10.27 €9.15 12.3%
IFRS Operating Income 496.75 816.0 -39.1%
IFRS operating margin 14.6% 26.1% -11.5pts
IFRS Consolidated Net Profit 347.35 647.2 -46.3%
IFRS earnings per share (fully diluted) €4.155 €7.73 -46.3%
Dividend per share6 €1.407 €1.20 16.7%
Free Cash Flow 774.4 710.9 8.9%
Closing net cash 160.3 65.1 n/a
"Ipsen delivered solid results and advanced its pipeline in 2024, laying a strong foundation for sustained growth," said David Loew, Chief Executive Officer, Ipsen. "With the successful global rollout of Iqirvo and Bylvay, and the U.S. launch of Onivyde, alongside multiple business development deals adding several innovative assets, we are well positioned to execute our strategic roadmap. This year, we look forward to achieving key milestones, including the first data readout for the Long-Acting Neurotoxin (LANT), and further expand and progress our pipeline across all three therapeutic areas to bring promising new medicines to patients."

Pipeline Progress

Significant regulatory milestones were achieved in 2024, including FDA approval of Onivyde (irinotecan) for first-line pancreatic ductal adenocarcinoma (PDAC), along with accelerated U.S. approval and European approval for Iqirvo (elafibranor), respectively. Additionally, Kayfanda (odevixibat) was approved for Alagille syndrome (ALGS) in the E.U.

The company also opted-in for the CABINET Phase III study of Cabometyx (cabozantinib) in patients with advanced neuroendocrine tumors (NETs), with study results presented at the 2024 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress and published in the New England Journal of Medicine.

An IND application was filed for IPN01194, an ERK inhibitor, advancing the potential medicine into clinical development with a Phase I/IIa trial in advanced solid tumors.

Ipsen improved further the depth and breadth of its pipeline by adding five preclinical innovative therapies with global rights and new modalities, and an ex-U.S. licensing agreement with DayOne Biopharmaceuticals for the late-stage oncology asset tovorafenib, an oral RAF inhibitor for pediatric low-grade glioma.

Two global licensing agreements for Antibody Drug Conjugate (ADC) in oncology with Sutro Biopharma and Foreseen Biotechnology were signed. An extension of the oncology partnership with Marengo Therapeutics to include TriSTAR, a next-generation precision T-cell engager was completed, as well as more recently, in the fourth quarter, a global licensing agreement with Biomunex for a preclinical novel T-cell engager (TCE). A collaboration with Skyhawk Therapeutics to develop RNA-modulating small molecules for rare neurological diseases was also signed during the year.

Ipsen executed several divestments in 2024, including the sale of Increlex (mecasermin injection) to Eton Pharmaceuticals and the sale of its rare pediatric disease Priority Review Voucher.

Environmental, Social and Governance

Ipsen took important steps in 2024 in delivering its ambitious sustainability strategy. The company continued to integrate sustainability across its operations. From reducing its environmental footprint to advancing patient access and fostering a strong workplace culture, the company increased its commitment to driving progress for patients, employees, communities, and the planet.

Our sustainability efforts were recognized across multiple environment initiatives. The company achieved a 45% reduction in Scopes 1 & 2 greenhouse gas emissions and a 25% reduction in Scope 3, fully in line with its 2030 targets (versus 2019 baseline). Significant efforts were made to engage suppliers and third parties in Ipsen’s sustainability roadmap including the first-ever "Ipsen Supplier Sustainability Day". Following an intensive transformation project, 99.8% of Ipsen’s global electricity now comes from renewable sources. Through the Fleet for Future project, the company continues to advance sustainable transportation, with 43% of its total company’s fleet now electric vehicles as of 2024.

We remain committed to gender balance in leadership, with women now representing 55% of the Global Leadership Team.

2025 Upcoming Milestones

Ipsen anticipates several key milestones across its portfolio in 2025, including:

Cabometyx (CABINET trial) – Regulatory decision in Europe for advanced neuroendocrine tumors (NETs), including pancreatic (pNETs) and extra-pancreatic (epNETs) neuroendocrine tumors
Tovorafenib (FIREFLY-1 trial) – Regulatory submission in Europe for pediatric low-grade glioma
Fidrisertib (FALKON trial) – Readout of the pivotal Phase IIb trial in fibrodysplasia ossificans progressiva (FOP)
LANT88(LANTIC trial) – Proof-of-concept data readout, evaluating its potential in aesthetics
These milestones reinforce Ipsen’s commitment to advancing innovative therapies and expanding treatment options for patients worldwide.

2025 Financial Guidance

Ipsen has set for FY 2025 the following financial guidance, which excludes any impact from potential late-stage (Phase III clinical development or later) business development transactions:

Total sales growth greater than 5.0%, at constant currency. Based on the average level of exchange rates in January 2025, a favorable effect on total sales of around 1% from currencies is expected.
Core operating margin greater than 30.0% of total sales, which includes additional R&D expenses from anticipated early and mid-stage external-innovation opportunities.
Guidance on total sales and core operating margin is based on accelerated sales growth of the ex-Somatuline portfolio and assumes negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe.

Consolidated financial statements

The Board of Directors approved the consolidated financial statements on 12 February 2025. The consolidated financial statements have been audited and the Statutory Auditors’ report is in the process of being published. Ipsen’s comprehensive audited financial statements will be available in due course on ipsen.com (regulated-information section).

Conference call

A conference call and webcast for investors and analysts will begin today at 1pm CET. Participants can access the call and its details by registering here; webcast details can be found here.

Calendar

Ipsen intends to publish its Q1 2025 sales on April 16th, 2025.

Notes

All financial figures are in € millions (€m), unless otherwise noted. The performance shown in this announcement covers the twelve-month period to 31 December 2024 (FY 2024) and the three-month period to 31 December 2024 (Q4 2024), compared to the twelve-month period to 31 December 2023 (FY 2023) and the three-month period to 31 December 2023 (Q4 2023), respectively, unless stated otherwise. The commentary is based on the performance in FY 2024, unless stated otherwise.

On February 13, 2025 ImmunityBio, Inc. (NASDAQ: IBRX), a leading immunity therapy company, reported the Medicines and Healthcare products Regulatory Agency (MHRA) has validated and accepted the marketing authorization application for ANKTIVA (Press release, ImmunityBio, FEB 13, 2025, View Source [SID1234650252]). The MHRA will now begin assessing the marketing authorization application (MAA) for ANKTIVA (nogapendekin alfa inbakicept-pmln) in combination with bacillus Calmette-Guérin (BCG) for the treatment of patients with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors. MHRA regulates medicines, medical devices and blood components for transfusion in the UK.

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"MHRA’s acceptance of our marketing authorization application for the UK comes less than three weeks after the European Medicines Agency accepted for review our MAA for the 30 countries covering the European Union, and just 10 months after FDA approval in the United States," said Dr. Patrick Soon-Shiong, Founder, Executive Chairman and Global Chief Scientific and Medical Officer of ImmunityBio. "We are determined to reach as many patients as possible with ANKTIVA and BCG and are encouraged by the momentum of our efforts."

About ANKTIVA

The cytokine interleukin-15 (IL-15) plays a crucial role in the immune system by affecting the development, maintenance, and function of key immune cells—NK and CD8+ killer T cells—that are involved in killing cancer cells. By activating NK cells, ANKTIVA overcomes the tumor escape phase of clones resistant to T cells and restores memory T cell activity with resultant prolonged duration of complete response.

ANKTIVA is a first-in-class IL-15 agonist IgG1 fusion complex, consisting of an IL-15 mutant (IL-15N72D) fused with an IL-15 receptor alpha, which binds with high affinity to IL-15 receptors on NK, CD4+, and CD8+ T cells. This fusion complex of ANKTIVA mimics the natural biological properties of the membrane-bound IL-15 receptor alpha, delivering IL-15 by dendritic cells and drives the activation and proliferation of NK cells with the generation of memory killer T cells that have retained immune memory against these tumor clones. The proliferation of the trifecta of these immune killing cells and the activation of trained immune memory results in immunogenic cell death, inducing a state of equilibrium with durable complete responses. ANKTIVA has improved pharmacokinetic properties, longer persistence in lymphoid tissues, and enhanced anti-tumor activity compared to native, non-complexed IL-15 in-vivo.

ANKTIVA was approved by the FDA in 2024 for BCG-unresponsive non-muscle invasive bladder cancer CIS with or without papillary tumors. For more information, visit ImmunityBio.com (Founder’s Vision) and Anktiva.com.

On February 13, 2025 IDEAYA Biosciences, Inc. (Nasdaq: IDYA), a precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported financial results for the quarter and full year ended December 31, 2024, and provided a business update (Press release, Ideaya Biosciences, FEB 13, 2025, View Source [SID1234650251]).

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"We are excited to be targeting multiple clinical data readouts and program updates across our clinical pipeline in 2025, including the darovasertib neoadjuvant UM data update in the first half and median PFS readout in HLA-A2 negative 1L MUM by year-end, update on the Werner Helicase inhibitor program IDE275 at a major medical conference in the first half, clinical combination results from the IDE397 and Trodelvy combination in MTAP-deletion UC where the Gilead collaboration has expanded to evaluate both UC and NSCLC, among others. Next, we are excited to target a clinical data update for potential first-in-class DLL3 TOP1 ADC IDE849 in SCLC in 2025, including potentially at a medical conference, and we believe the clinical profile observed supports a potential monotherapy regulatory path in SCLC," said Yujiro S. Hata, President and Chief Executive Officer, IDEAYA Biosciences.

"We had multiple positive clinical updates across our pipeline throughout the past year that were presented at major medical conferences, including compelling results such as tumor shrinkage and eye preservation for neoadjuvant darovasertib which was presented as an oral presentation at ASCO (Free ASCO Whitepaper) 2024, and evidence for monotherapy activity with IDE397 which delivered a high disease control rate and multiple confirmed RECIST 1.1 responses in MTAP-deletion lung and urothelial cancer, which was presented as a late breaker oral presentation at ENA 2024. Next, we believe we have generated important proof-of-concept preclinically and preliminary clinical data for the MAT2A and PRMT5 combination in the MTAP-deletion setting and are excited to advance IDEAYA’s wholly-owned IDE397 and IDE892 combination that we are targeting to have in the clinic in the second half of 2025," commented Darrin Beaupre, M.D., Ph.D., Chief Medical Officer, IDEAYA Biosciences. "Additionally, in the last several quarters we made significant progress in enhancing our potential first-in-class clinical pipeline with the in-licensing of the Phase 1 DLL3 TOP1i ADC IDE849 and FDA clearance of the Phase 1 trial for the Werner Helicase inhibitor IDE275. We are also advancing three programs into IND-enabling studies: our PRMT5 inhibitor IDE892, the B7H3/PTK7 ADC IDE034 and a KAT6/7 inhibitor IDE251," said Michael White, Ph.D., Chief Scientific Officer, IDEAYA Biosciences.

Recent Key Developments and Upcoming Milestones

Research and Clinical Development

•
Darovasertib, a potential first-in-class Phase 2/3 PKC inhibitor for the treatment of metastatic uveal melanoma (MUM) and neoadjuvant uveal melanoma (UM).
•
MUM
•
Independent Data Monitoring Committee (IDMC) recommended move-forward dose and the completion of the Part 2a dose optimization for the potential registration-enabling trial of darovasertib and crizotinib in first line (1L) HLA-A2-negative MUM.
•
Targeting median progression free survival (PFS) readout for Phase 2/3 registration-enabling trial of the darovasertib and crizotinib combination in 1L HLA-A2-negative MUM by year-end 2025. Rapid enrollment in the trial continues with over 230 patients as of February 7, 2025.
•
Phase 2 median overall survival (OS) readout from study IDE196-001 in ~40 1L MUM patients targeted in 2025.
•
Neoadjuvant UM
•
A clinical update in over 75 patients in the Company-sponsored Phase 2 trial and regulatory update(s) is targeted for the first half of 2025, including vision data in plaque brachytherapy patients. 95 patients enrolled as of December 31, 2024 in the Company-sponsored Phase 2 trial.
•
Initiation of the Phase 3 registration-enabling trial for darovasertib in neoadjuvant UM is targeted for the first half of 2025.

Exhibit 99.1

•
IDE397, a potential first-in-class Phase 2 MAT2A Inhibitor for the treatment of MTAP-Deletion Solid Tumors.
•
Encouraging Phase 1 expansion results of IDE397 in MTAP-deletion urothelial cancer (UC) and non-small cell lung cancer (NSCLC) patients were presented as a late-breaking oral presentation at the 36th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium (ENA 2024). Additional preclinical data on the anti-tumor activity of IDE397 and in combination with the PRMT5 inhibitors AMG 193 and BMS-986504 in MTAP-deleted tumors were presented as a poster.
•
Targeting Phase 1/2 expansion in the first quarter of 2025 and clinical data update for IDE397 in combination with Trodelvy in MTAP-deletion UC in 2025. Entered into a Clinical Study Collaboration and Supply Agreement with Gilead to evaluate IDE397 in combination with Trodelvy in MTAP-deletion NSCLC.
•
IDEAYA plans to enable wholly-owned IDE397 combination with IDE892 in patients with MTAP-deletion non-small cell lung cancer (NSCLC) in the second half of 2025. IDEAYA and Amgen mutually agreed to wind down the IDE397 and AMG 193 clinical combination study in February 2025, and will not pursue dose expansion.
•
IDE849 (SHR-4849), a potential first-in-class Phase 1 DLL3 TOP1i antibody drug conjugate (ADC) targeting small cell lung cancer (SCLC) and neuroendocrine tumors (NETs).
•
IDEAYA entered into an exclusive global license agreement for IDE849 outside of Greater China with Jiangsu Hengrui Pharmaceuticals Co., Ltd. (Hengrui Pharma) in December 2024.
•
IDE849 is currently being evaluated by Hengrui Pharma in an ongoing Phase 1 trial in China in SCLC patients. In preliminary results from the trial with a data cut-off date of December 10, 2024, 8 out of 11 evaluable patients achieved partial response by RECIST 1.1, resulting in an overall response rate of ~73% (including both confirmed and unconfirmed responses, all unconfirmed responses were pending further evaluation). As of the data cut-off date, treatment related adverse events (TRAEs) were predominantly Grade 1 or 2. The most common TRAEs observed were white blood cell count decreased, anemia, neutrophil count decreased, nausea and platelet count decreased. In January 2025, Hengrui Pharma selected expansion doses for the Phase 1 trial.
•
IDEAYA plans to submit a U.S. IND for the evaluation of IDE849 as a monotherapy in SCLC in the first half of 2025.
•
Targeting to initiate the evaluation in combination with IDE161 and in NETs in the second half of 2025.
•
Clinical data update targeted in 2025.

Exhibit 99.1

•
IDE275 (GSK959), a potential first-in-class and best-in-class Phase 1 Werner Helicase inhibitor for the treatment of high microsatellite instability (MSI-High) tumors.
•
Received FDA IND clearance for a Phase 1 trial in MSI-High tumors and earned a $7.0 million milestone payment from GSK.
•
Data highlighting IDE275’s differentiated potential best-in-class profile will be presented with GSK at a medical conference in the first half of 2025.
•
IDE161, a potential first-in-class Phase 1 PARG inhibitor for the treatment of solid tumors.
•
Selected initial Phase 1/2 expansion dose for IDE161 monotherapy in endometrial cancer, based on AE profile and preliminary clinical efficacy observed. Phase 1 monotherapy dose optimization is ongoing to confirm a move-forward Phase 2 expansion dose.
•
First patient dosed in the Phase 1 trial evaluating IDE161 in combination with KEYTRUDA in patients with MSI-High and microsatellite stable (MSS) endometrial cancer. Targeting Phase 1 expansion in 2025.
•
Highlighted preclinical data of IDE161 and antibody drug conjugate (ADC) combination rationale at a poster presentation at ENA 2024.
•
Targeting clinical combination(s) of IDE161 with TOP1i ADCs in solid tumors in 2025.
•
IDE705 (GSK101), a potential first-in-class Phase 1 Pol Theta Helicase Inhibitor in combination with PARP inhibitor for the treatment of HRD solid tumors.
•
Targeting Phase 2 expansion in HRD solid tumors, which would trigger a potential $10 million milestone payment from GSK.
•
Announced three development candidates for the treatment of solid tumors:
•
IDE892, a potential best-in-class MTA-cooperative PRMT5 inhibitor to enable wholly-owned combination with IDE397. IND filing targeted for mid-year 2025.
•
IDE034, a potential first-in-class B7H3/PTK7 TOP1i bispecific ADC with combination potential with IDE161. IND filing targeted for the second half of 2025.
•
IDE251, a potential first-in-class KAT6/7 dual inhibitor development candidate with combination opportunities with multiple programs in the Company’s pipeline. IND filing targeted for the second half of 2025.

Corporate Development

•
Appointed Joshua Bleharski, Ph.D., as Chief Financial Officer. Dr. Bleharski to join IDEAYA from J.P. Morgan, serving most recently as Managing Director and Global Co-Head of Biopharma in the Healthcare Investment Banking group. Josh spent nearly 17 years at J.P. Morgan advising clients in the biopharma sector on capital markets transactions, corporate strategy and other investment banking services representing more than $65 billion of value for biotechnology companies worldwide.
•
Appointed Stu Dorman as Chief Commercial Officer. Mr. Dorman brings over 20 years of global commercialization experience in oncology with prominent biopharmaceutical companies such as Gilead Sciences and Bristol Myers Squibb, where he led multiple commercial launches for products including Trodelvy and Opdivo.

Financial Results

As of December 31, 2024, IDEAYA had cash, cash equivalents and marketable securities of approximately $1.1 billion, compared to $632.6 million as of December 31, 2023. The increase was primarily driven from $379.9 million in net proceeds from the sale of common stock shares through at-the-market financings during the year, $283.8 million from an underwritten public offering of common stock and pre-funded warrants to purchase common stock completed in July 2024, partially offset by net cash used in operations. IDEAYA believes that these funds will be sufficient to fund its planned operations into at least 2028.

Subsequent to the reporting period for the year ended December 31, 2024, the Company generated net proceeds of approximately $25.1 million from the sale of shares of its common stock through at-the-market (ATM) offerings in January 2025.

Collaboration revenue for the three months ended December 31, 2024 totaled $7.0 million compared to $3.9 million for the three months ended December 31, 2023. Collaboration revenue was recognized for a $7.0 million milestone payment from GSK that was earned for the IND clearance of IDE275 (GSK959) in October 2024.

Research and development (R&D) expenses for the three months ended December 31, 2024, totaled $140.2 million compared to $38.8 million for the three months ended December 31, 2023. The increase was primarily due to a $75.0 million upfront payment under the license agreement for IDE849 with Hengrui Pharma, higher clinical trial expenses to support the portfolio growth and personnel-related expenses.

General and administrative (G&A) expenses for the three months ended December 31, 2024 totaled $11.0 million compared to $7.1 million for the three months ended December 31, 2023. The increase was primarily due to higher personnel-related expenses, higher consulting and legal fees.

The net loss for the three months ended December 31, 2024, was $130.3 million compared to the net loss of $34.0 million for the three months ended December 31, 2023. Total stock compensation expense for the three months ended December 31, 2024, was $9.5 million compared to $4.8 million for the same period in 2023.

The net loss for the year ended December 31, 2024 was $274.5 million compared to $113.0 million for the same period in 2023. Total stock compensation expense for the year ended December 31, 2024, was $34.7 million compared to $18.5 million for the same period in 2023.

On February 13, 2025 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that its research collaborators at a major cancer research center in Houston, Texas have published a new study in eLife titled, "NPRL2 gene therapy induces effective antitumor immunity in KRAS/STK11 mutant anti-PD1 resistant metastatic non-small cell lung cancer (NSCLC) in a humanized mouse model (Press release, Genprex, FEB 13, 2025, View Source [SID1234650250])."

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"We are thrilled by the continued advancements of our academic partners on the NPRL2 gene therapy, which supports the therapeutic potential of our non-viral Oncoprex Delivery System to deliver the NPRL2 tumor suppressor gene therapy," said Ryan Confer, President and Chief Executive Officer at Genprex. "These data indicate the single agent potential of NPRL2 in lung cancer. They also demonstrate that the Oncoprex Delivery System used in Reqorsa Gene Therapy with the TUSC2 tumor suppressor gene is a platform and can be used with other tumor suppressor genes, such as NPRL2. We are pleased that these positive data also support the therapeutic potential of NPRL2 gene therapy in NSCLC and positions Genprex for the expansion of our clinical development pipeline."

The studies used the Company’s non-viral ONCOPREX Delivery System in KRAS/STK11 double mutant anti-PD1 resistant metastatic NSCLC xenografts in humanized mouse models. The ONCOPREX Delivery System is a novel non-viral approach utilizing lipid nanoparticles to deliver tumor suppressor genes that have been deleted during the course of cancer development. The platform allows for the intravenous delivery of various tumor suppressor genes to achieve a therapeutic effect without the risk of toxicity often associated with viral delivery systems.

In the published study, humanized mice were treated with NPRL2 gene therapy, immunotherapy pembrolizumab (Keytruda), or the combination. A dramatic antitumor effect was mediated by NPRL2 treatment alone, whereas pembrolizumab alone was ineffective, and the combination added little to NPRL2 treatment alone.

A significant antitumor effect was also found in non-humanized NSG mice, although the antitumor effect was greater in humanized mice, suggesting that the effects of NPRL2 gene therapy are achieved through the immune system. This is consistent with a more detailed analysis showing that NPRL2 gene therapy induces antitumor activity against KRAS/STK11 mutant anti-PD1 resistant tumors through dendritic cell mediated antigen presentation and cytotoxic immune cell activation. KRAS/STK11 mutant tumors are particularly resistant to treatment, and demonstrating efficacy in this setting suggests that a wide variety of lung cancers could potentially be targeted. This potentially would include the approximately 30% of NSCLCs that have KRAS mutations.

About Reqorsa Gene Therapy

REQORSA (quaratusugene ozeplasmid) consists of a plasmid containing the TUSC2 gene encapsulated in non-viral lipid-based nanoparticles in a lipoplex form (the Company’s Oncoprex Delivery System), which has a positive charge. REQORSA is injected intravenously and specifically targets cancer cells. REQORSA is designed to deliver the functioning TUSC2 gene to negatively charged cancer cells while minimizing uptake by normal tissue. Laboratory studies conducted at MD Anderson show that the uptake of TUSC2 in tumor cells in vitro after REQORSA treatment was 10 to 33 times the uptake in normal cells.