On February 13, 2025 Neogap therapeutics reported new study published in the highly ranked scientific journal Gut examines how immune system T cells respond to tumours in patients with advanced liver cancer (HCC) (Press release, Neogap Therapeutics, FEB 13, 2025, View Source;pior-technology-identifies-targets-for-personalised-liver-cancer-immunotherapie,c4104603 [SID1234650258]). Using Neogap Therapeutics’ PIOR software platform, the researchers have identified tumour-specific mutations, which may contribute to the development of personalised immunotherapies.

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T cells are central to the body’s defence against cancer but can also contribute to an immunosuppressive environment within the tumour. The study analysed T cells from the liver, lymph nodes, and tumour tissue to identify the most reactive cells to cancer-related neoantigens—tumour-specific proteins formed through mutations that can activate the immune system. Bioinformatic analysis identified 542 potential neoantigens from seven patients, 14 of which were found to induce a strong immune response, particularly in T cells from the liver and lymph nodes.

By using PIOR, the researchers were able to identify neoantigens capable of activating T cells, which in turn can attack the tumour.

"The results demonstrate that PIOR can effectively identify neoantigens with potential for future therapeutic applications," says Associate Professor Anna Pasetto, a researcher at Karolinska Institutet and one of the lead authors of the study. "Data-driven analysis of neoantigens is an important component of the new generation of personalised cancer immunotherapies."

The findings show that T cells from the liver and lymph nodes have properties that may be particularly suitable for future immunotherapies.

"This study aligns with Neogap’s vision to develop the next generation of cell therapies," says Ola Nilsson, Head of Neoantigen Production, Development & Clinical Processing at Neogap and co-author of the study. "PIOR plays a key role in our efforts to identify the most promising neoantigens—an essential part of the development of future precision therapies."

The study provides deeper insights into how T cells respond to neoantigens.

"Our findings reinforce the idea that T cells from lymph nodes may be particularly valuable as source material for generating reactive T cells—an approach already applied in Neogap’s ongoing clinical trial in colorectal cancer, which specifically uses lymph nodes as starting material. These discoveries could have a significant impact on both research and the development of new treatments", says Ola Nilsson.

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 entered into agreements with certain holders of its existing warrants for the immediate exercise of certain outstanding warrants to purchase up to an aggregate of 5,828,570 shares of common stock of the Company originally issued in December 2023 and August 2024 all at a reduced exercise price of $1.00 per share (Press release, Moleculin, FEB 13, 2025, View Source [SID1234650257]). The shares of common stock issuable upon exercise of the outstanding warrants are registered pursuant to effective registration statements on Form S-1 (File No. 333-280951) and on Form S-1 (File No. 333-276851). The aggregate gross proceeds from the exercise of the existing warrants is expected to total approximately $5.8 million, before deducting financial advisory fees.

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Roth Capital Partners is acting as the Company’s financial advisor for this transaction.

In consideration for the immediate exercise of the warrants for cash, the Company will issue new unregistered warrants to purchase shares of common stock. The new warrants will be exercisable for an aggregate of up to 11,657,140 shares of common stock, at an exercise price of $0.75 per share and will be immediately exercisable upon issuance and for a term of five years from the issuance date.

The transaction is expected to close on or about February 14, 2025, subject to satisfaction of customary closing conditions. The Company intends to use the net proceeds from the offering for working capital and general corporate purposes.

The new warrants described above were offered in a private placement pursuant to an applicable exemption from the registration requirements of the Securities Act of 1933, as amended (the "1933 Act") and, along with the shares of common stock issuable upon their exercise, have not been registered under the 1933 Act, and may not be offered or sold in the United States absent registration with the Securities and Exchange Commission ("SEC") or an applicable exemption from such registration requirements. The Company has agreed to file a registration statement with the SEC covering the resale of the shares of common stock issuable upon exercise of the new warrants.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

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.