On September 25, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat cancers and viral infections, reported that the Canadian Intellectual Property Office (CIPO) has issued a notice of allowance for Patent Application No. 3,142,510 titled, "PREPARATION OF PRELIPOSOMAL ANNAMYCIN LYOPHILIZATE." A patent from the application is expected to be issued in the coming months (Press release, Moleculin, SEP 25, 2025, https://moleculin.com/moleculin-announces-notice-of-allowance-for-canadian-patent-covering-annamycin/ [SID1234656241]).

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When issued, the patent claims will cover methods of making a preliposomal Annamycin lyophilizate with improved stability and high purity, with a base patent term currently extending until June 2040, subject to extension to account for time required to fulfill requirements for regulatory approval. Moleculin’s novel drug candidate is being positioned to become the first ever non-cardiotoxic anthracycline to be approved and is currently being developed for the treatment of acute myeloid leukemia (AML) and soft tissue sarcoma lung metastases (STS lung mets). Additional preclinical studies performed at a world-renowned cancer center indicate Annamycin may be a potential treatment for many other types of cancers. The new chemical entity uses a unique lipid-based delivery technology and has shown the potential to be used in a wide range of cancers. In addition to the newly expected Canadian patent and previously issued U.S. and allowed European patents, Moleculin has additional patent applications related to Annamycin pending in the U.S., Europe and in major jurisdictions worldwide.

Walter Klemp, Chairman and CEO of Moleculin, commented, "We remain committed to bolstering our global intellectual property portfolio for Annamycin. This Canadian patent further strengthens our current IP portfolio which includes claims to methods of making our preliposomal Annamycin, in yet another key territory. As we continue to advance our development of Annamycin as a potentially transformative therapeutic candidate for hard-to-treat tumors, building on our global patent protection remains a focus."

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 AML, in addition to Orphan Drug Designation for the treatment of STS lung mets. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory AML from the EMA.

On September 25, 2025 Kura Oncology, Inc. (Nasdaq: KURA) and Kyowa Kirin Co., Ltd. (TSE: 4151, "Kyowa Kirin") reported the Journal of Clinical Oncology published the full results from the pivotal KOMET-001 clinical trial (NCT04067336) evaluating ziftomenib, an investigational, once-daily, oral menin inhibitor, in adult patients with relapsed/refractory (R/R) NPM1-mutated (NPM1-m) acute myeloid leukemia (AML) (Press release, Kura Oncology, SEP 25, 2025, View Source [SID1234656240]). Although newly diagnosed patients with NPM1-m AML have high response rates to approved standard of care, relapses are common and survival outcomes are poor. There is currently no approved therapy to specifically target NPM1-m AML. Ziftomenib is currently under priority review by the Food and Drug Administration (FDA) for treatment of R/R NPM1-m AML.

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"Relapsed or refractory NPM1-mutated AML remains very challenging to treat, particularly after venetoclax-based therapy or transplant," said Eunice Wang, M.D., Chief of Leukemia Service, Professor of Oncology, Roswell Park Comprehensive Cancer Center, Buffalo, NY. "The manuscript describes deep responses, signals of clinical activity across relevant subgroups and a generally manageable tolerability profile, which is important in treatment of late line AML patients where accumulated toxicity can limit treatment options. The benefit-risk profile of ziftomenib is highly encouraging and, if replicated in additional treatment settings, has potential to be transformative for a large population of patients with menin pathway-driven AML."

"Publication in ASCO (Free ASCO Whitepaper)’s Journal of Clinical Oncology is an important advancement for adult patients with NPM1-m AML," said Mollie Leoni, M.D., Chief Medical Officer of Kura Oncology. "In addition to evidence of monotherapy activity, the safety and tolerability profile of ziftomenib from this trial is encouraging, marked by the absence of clinically meaningful QTc prolongation as well as low rates of both myelosuppression and treatment discontinuation. No clinically meaningful drug-drug interactions were observed, including with commonly used supportive-care medications, which may simplify co-administration in a polypharmacy setting. We continue to conduct studies in earlier line settings and in combination with multiple therapeutic agents in close collaboration with investigators, study teams and our partner Kyowa Kirin."

Summary of the published data
The publication, entitled "Ziftomenib in Relapsed or Refractory NPM1-Mutated AML", includes positive data from 92 adult patients with R/R NPM1-m AML in the phase 2 portion of the clinical trial as of the primary data cutoff date of October 28, 2024.

The KOMET-001 phase 2 trial met its primary endpoint with a complete remission with full or partial hematologic recovery (CR/CRh) rate of 22% (95% CI, 14 to 32; P=0.0058), which was significantly higher than the 12% historical standard-of-care response rate for patients with R/R NPM1-m AML. One additional response of CRh occurred after the primary analysis data cutoff resulting in a cumulative CR/CRh rate of 23% (95% CI, 15 to 33). 61% of evaluable CR/CRh responders were negative for measurable residual disease (MRD). Overall response rate (ORR) was 33% (95% CI, 23 to 43), with a median duration of overall response of 4.6 months (95% CI, 2.8 to 7.4).

Median overall survival (OS) was 6.6 months (95% CI, 3.6 to 8.6). Among ORR responders, median OS was 18.4 months (95% CI, 8.6 to not estimable) vs. 3.5 months (95% CI, 2.7 to 4.2) among non-responders. Two responders received subsequent allogeneic stem cell transplant and both resumed ziftomenib maintenance after transplant. At the time of data cutoff, nine patients (two after transplantation) remained on ziftomenib treatment. Prespecified subgroup analyses showed comparable CR/CRh rates regardless of lines of therapy, prior venetoclax exposure, or presence of co-mutations, including FLT3m or IDH1/2m.

Ziftomenib was well tolerated with a safety profile consistent with previously disclosed data. The most common grade ≥3 treatment-emergent adverse events were febrile neutropenia (26%), anemia (20%), and thrombocytopenia (20%). Differentiation syndrome occurred in 25% of patients (15% grade 3; no grade 4-5) and was manageable with protocol-defined mitigation. Three patients (3%) discontinued treatment because of ziftomenib-related adverse events.

These findings formed part of the data set used for the New Drug Application for ziftomenib as a potential treatment for patients with R/R NPM1-m AML. The FDA target action date is November 30, 2025. There is currently no FDA-approved treatment for patients with R/R NPM1-m AML.

"The publication of the investigational ziftomenib data adds important scientific context for clinicians and patients," said Takeyoshi Yamashita, Ph. D., Executive Vice President and Chief Medical Officer, Kyowa Kirin. "Together with Kura Oncology, we are committed to rigorous, globally coordinated evidence generation to support the benefit-risk profile of menin inhibition across the treatment landscape. Our shared goal is to advance development rapidly and generate the evidence needed to deliver ziftomenib to appropriate patients in need."

The publication is now available on the Journal of Clinical Oncology website and in the Scientific Manuscripts section on Kura’s website.

Ziftomenib is currently under clinical development, and its safety and efficacy have not been evaluated by any regulatory authority.

On September 25, 2025 INOVIO (NASDAQ: INO), a biotechnology company focused on developing and commercializing DNA medicines to help treat and protect people from HPV-related diseases, cancer, and infectious diseases, reported that it will present data at a number of upcoming scientific conferences, highlighting key aspects of lead product candidate INO-3107 for RRP (Press release, Inovio, SEP 25, 2025, View Source [SID1234656239]). INOVIO has begun the rolling submission of its Biologics License Application (BLA) for INO-3107, which it anticipates completing in the second half of 2025 with the goal of file acceptance by the U.S. Food and Drug Administration by the end of 2025.

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INOVIO also plans to highlight its next-generation DNA-medicine technology, including presentations on a Phase 1 clinical trial of a DNA-encoded monoclonal antibody (DMAb) product candidate and new preclinical data on DPROTs addressing Hemophilia A.

Presentations on INO-3107 for RRP:

American Academy of Otolaryngology – Head and Neck Surgery Annual Meeting
Indianapolis
Date: October 11-13
Poster Title: DNA Immunotherapy (INO-3107) Demonstrates a Durable Response for Treatment of HPV-6/11 Recurrent Respiratory Papillomatosis

World Vaccine Congress Europe
Amsterdam, NL
Date: October 13
Time: 3:45 PM CEST
Presentation Title: Leveraging the Potential of DNA Immunotherapy: Long-Term Efficacy in HPV-6 & 11 Recurrent Respiratory Papillomatosis

European Society For Medical Oncology Congress
Berlin, DE
Date: October 19
Poster Title: DNA Immunotherapy (INO-3107) in HPV-6 & 11 Recurrent Respiratory Papillomatosis – Long-Term Efficacy

37th Annual International Papillomavirus Society Conference
Bangkok, TH
Date: October 24
Time: 2:40 – 2:55 PM ICT
Presentation Title: Clinical Response to INO-3107 in RRP is Irrespective of Papilloma Microenvironment and Molecular Subtype

Date: October 25
Time: 6:54 – 7:00 PM ICT
E-Poster Title: DNA Immunotherapy (INO-3107) Induces Persistent Immune Responses Resulting in Long-term Efficacy Through Post-Treatment Year 2 for HPV 6&11 RRP

International Society for Vaccines Annual Congress
Stellenbosch, SA
Date: October 30
Time: 10:20 – 10:35 AM SAST
Presentation Title: Immunotherapy, INO-3107, is Well-Tolerated, Effective, and Elicits an Antigen-Specific T-cell Response in Adults with HPV-6 & 11 Recurrent Respiratory Papillomatosis

Presentations on Next-Generation DNA Medicine:

European Society of Gene and Cell Therapy Congress
Seville, ES
Date: October 7-10
Poster Title: Employment of the CELLECTRA in vivo gene delivery platform in a first in human (FIH) DNA encoded monoclonal antibody (DMAb) clinical trial

World Vaccine Congress Europe
Amsterdam, NL
Date: October 16
Time: 1:00 PM CET
Presentation Title: DMAb Technology: Next Generation DNA Medicine Design and Delivery

World Orphan Drug Congress
Amsterdam, NL
Date: October 29
Time: 11:10 AM CET
Presentation Title: DMAb Technology: The Transformational Potential of Next Gen DNA Medicine in Rare Disease

14th World Federation of Hemophilia Global Forum
Montreal, CA
Date: November 14
Time: 1:45 PM EST
Presentation Title: Novel FVIII Secretion Via Non-Viral Vector DNA Medicine Platform

Available abstracts will be shared on INOVIO’s website following presentations.

On September 25, 2025 Immuneering Corporation (Nasdaq: IMRX), a clinical-stage oncology company focused on keeping cancer patients alive, reported the pricing of its underwritten public offering of 18,959,914 shares of its Class A common stock at an offering price of $9.23 per share, which is equal to the last reported sale price for Immuneering’s Class A common stock on The Nasdaq Global Market on September 24, 2025 (Press release, Immuneering, SEP 25, 2025, View Source [SID1234656238]). In addition, Immuneering has granted the underwriters a 30-day option to purchase up to an additional 2,843,987 shares of Class A common stock at the public offering price, less the underwriting discounts and commissions. The gross proceeds from the public offering are expected to be approximately $175 million, before deducting underwriting discounts and commissions and offering expenses payable by Immuneering, and excluding any exercise of the underwriters’ option to purchase additional shares. In addition, Immuneering announced that Sanofi has agreed to purchase 2,708,559 shares of Immuneering’s Class A common stock, at a purchase price of $9.23 per share, in a separate private placement transaction that is expected to close concurrently with the public offering. All securities in the public offering and private placement will be offered by Immuneering. The public offering and private placement are expected to close concurrently on or about September 26, 2025, subject to customary closing conditions, and the closing of the private placement is contingent upon the closing of the public offering.

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Immuneering intends to use the net proceeds of the public offering and the private placement to advance the preclinical and clinical development of its product candidates and for working capital and other general corporate purposes.

Leerink Partners and Oppenheimer & Co. are acting as the joint bookrunners for the public offering and as placement agents in connection with the private placement.

The public offering is being made pursuant to a shelf registration statement on Form S-3, including a base prospectus, that was filed by Immuneering with the Securities and Exchange Commission (the "SEC") on August 13, 2025 and declared effective by the SEC on August 20, 2025. A prospectus supplement relating to the offering will be filed with the SEC. Copies of the prospectus supplement relating to the offering, when available, may be obtained from: Leerink Partners LLC, Attention: Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, by telephone at (800) 808-7525, ext. 6105, or by email at [email protected]; and Oppenheimer & Co. Inc., Attention: Syndicate Prospectus Department, 85 Broad Street, 26th Floor, New York, New York 10004, or by telephone at (212) 667-8055, or by e-mail at [email protected], or by visiting the EDGAR database on the SEC’s website at www.sec.gov.

The shares being sold in the Private Placement have not been and will not be registered under the Securities Act of 1933, as amended, or any state securities laws and may not be offered or sold in the United States absent registration with the SEC or an applicable exemption from such registration requirements.

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

On September 25, 2025 HiFiBiO Therapeutics, a clinical stage immune modulation biotechnology company, reported its participation in the 2025 Annual Meeting of the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper), taking place October 17-21 in Berlin, Germany (Press release, HiFiBiO Therapeutics, SEP 25, 2025, View Source [SID1234656237]).

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The company will present exciting Phase 1 clinical data on two immuno-oncology programs: the first-in-class anti-TNFR2 agonist emunkitug, and an anti-BTLA antagonist with best-in-class potential, HFB200603. The posters will report updated safety and efficacy results along with potential predictive biomarkers of clinical benefit.

HiFiBiO’s novel immunotherapies powered by AI/ML and its single-cell translational platform demonstrate the company’s deep commitment to the development of impactful therapies to address unmet medical needs.

Details of the poster presentation are as follows:

Presentation # 994P

Title: Potential Predictive Biomarkers of Clinical Benefit from Emunkitug (HFB200301) in Combination with Tislelizumab: Integrated Analysis of Tumor, Peripheral Blood, and Patient Treatment History.

Session Date: Sunday, October 19, 2025

Session Time: 12:00 – 12:45 CEST

Speaker: Desamparados Roda, MD, PhD, Hospital Clínico de Valencia

Presentation # 949P

Title: Evaluation of HVEM and PD-L1 Expression Profile in Tumors as Potential Predictive Biomarkers for HFB200603, a BTLA antagonist, as Monotherapy and in Combination with Tislelizumab.

Session Date: Sunday, October 19, 2025

Session Time: 12:00 – 12:45 CEST

Speaker: María de Miguel, MD, PhD, Hospital Universitario HM Sanchinarro