On May 5, 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 viruses, reported the U.S. Patent and Trademark Office (USPTO) has granted two additional U.S. patents with claims covering Annamycin. U.S. patent number 12,257,261 titled, "Preparation of Preliposomal Annamycin Lyophilizate", has claims covering methods of making liposomal Annamycin and U.S. patent 12,257,262 titled "Method of Reconstituting Liposomal Annamycin", has claims covering methods of making liposomal Annamycin suspension (Press release, Moleculin, MAY 5, 2025, View Source [SID1234652511]). Both patents have a base patent term currently extending until June 2040, subject to adjustment for delays in prosecution and extension to account for time required to fulfill requirements for regulatory approval. Moleculin has additional patent applications related to Annamycin pending in the U.S., Europe and in major jurisdictions worldwide.

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Annamycin, Moleculin’s novel drug candidate, is being positioned to become the first anthracycline demonstrating a lack of cardiotoxicity 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.

Wally Klemp, Chairman and CEO of Moleculin, said, "We remain focused on expanding our intellectual property portfolio for Annamycin. Following the issuance of two U.S. patents in 2024, these new patents enhance the exclusivity of Annamycin, bringing to four the total number of U.S. patents related to Annamycin, in addition to the European patents granted. Based on the data seen to date and feedback we continue to receive from clinicians and patients, we are dedicated to advancing this important and much needed treatment option forward. We continue to make solid progress in our ongoing pivotal, adaptive Phase 3 MIRACLE trial and remain on track to report initial data in the second half of 2025."

Annamycin 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 acute myeloid leukemia from the European Medicines Agency (EMA).

On May 5, 2025 AGC Biologics, your friendly CDMO expert, reported a multiphase partnership with Novelty Nobility Inc., a clinical-stage biotech company based in South Korea (Press release, Novelty Nobility, MAY 5, 2025, View Source [SID1234652529]). Novelty Nobility will leverage AGC Biologics’ expertise and proprietary CHEF1 expression technology for cell line development and prepare for Phase I clinical trials via a multi-site partnership with AGC Biologics’ Copenhagen, Denmark and Chiba, Japan facilities.

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Novelty Nobility develops innovative antibody drugs, including bispecific antibodies and antibody-drug conjugates (ADCs). The company is currently developing two clinical assets in the United States in immunology and oncology, respectively.

AGC Biologics will develop the cell line and create a master cell bank (MCB) for Novelty Nobility’s first-in-class bispecific antibody drug candidate at its Copenhagen facility. After the MCB is created, the second phase of the partnership will focus on expanded process development work and GMP manufacturing preparation at the AGC Biologics Chiba site. The first stage of the project began in March.

"We are very happy with the partnership with Novelty Nobility, where we will apply our CHEF1 platform to a novel bispecific antibody, helping support the advancement of this important candidate towards clinical trials," said Kasper Møller, Chief Technical Officer and Executive Vice President, AGC Biologics. "Our cell line development expertise in Copenhagen is strategically aligned with Novelty Nobility’s objectives, and we believe we can provide a strong foundation for their therapeutic development program."

"Chiba’s partnership with our Copenhagen team gives Novelty Nobility access to world-class cell line development experts while keeping their regional connection strong," noted Susumu Zen-in, Senior Vice President and General Manager, AGC Biologics Chiba. "When clients work with us, they tap into our entire global network – we find the right expertise wherever it exists and bring teams together for a seamless experience, no matter where the work is being done. This approach is already helping us throughout the APAC region and creating wins for both Novelty Nobility and AGC Biologics."

AGC Biologics’ CHEF1 expression technology is a proven platform for cell line development, with a track record of producing stable cell lines for a variety of biologics. With five commercial products on the market and 54 distinct molecules developed, the platform delivers a reliable and efficient path to success. The cell line platform excels in handling complex molecules, providing solutions for challenging biologics that require specialized expression systems. The CHEF1 platform is also royalty-free, eliminating additional costs for clients as their products advance through clinical phases toward commercialization.

AGC Biologics offers a comprehensive suite of services for mammalian-based drug production, from cell line development to commercial manufacturing, enabling partners to accelerate their drug development timelines and bring life-changing therapies to patients. Visit www.agcbio.com/capabilities/mammalian to learn more.

On May 4, 2025 Nutshell Therapeutics ( Shanghai ) Co., LTD. reported to have received IND clearance from the FDA to initiate Phase 1 clinical trial in the United States for its NTS071, a novel small molecule allosteric reactivator targeting p53 Y220C mutation (Press release, Nutshell Therapeutics, MAY 4, 2025, View Source [SID1234652483]).

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NTS071 is an oral small molecule allosteric reactivator targeting p53 Y220C with a novel scaffold. It selectively binds to the p53 Y220C mutant protein, improving its thermal stability, thereby enhancing the mutant protein’s ability to bind with DNA and restoring its transcriptional activity as well as tumor-suppressing function.

NTS071 was discovered by leveraging Nutshell’s proprietary AI driven allosteric small molecule drug discovery platform ALLOSTAR. NTS071 demonstrated potential Best-in-Class preclinical properties for its target. NTS071 achieved a picomolar-level biochemical activity which is 20 folds more potent than the competitive compound PC14586. Additionally, NTS071 shows better stability in both liver microsomes and hepatocytes across different species and exhibits lower in vivo clearance rates and higher oral exposure s in preclinical PK studies across all tested species compared to PC14586. NTS071 has relatively lower plasma protein binding and higher free fraction than PC14586, which is beneficial for in vivo efficacy. NTS071 also addresses the CYP3A4 inhibition issue of PC14586, presenting lower risks for potential drug – drug interactions. NTS071 further displays a large safety window by exhibiting an overall good safety profile in non-clinical toxicology studies.

NTS071 exhibited dose-dependent in vivo anti-tumor activity in multiple CDX and PDX models harboring p53 Y220C mutation, spanning a number of different cancer types, including ovarian cancer, lung cancer, gastric cancer, breast cancer, head and neck cancer, esophageal cancer, pancreatic cancer, and bladder cancer, etc. Therefore, NTS071 has the potential to be a tumor-agnostic therapy for patients carrying p53 Y220C mutation. Compared to PC14586, NTS071 has shown significantly lower effective doses or better efficacy at the same dose level in all comparative preclinical in vivo studies, implying that NTS071 may overcome the limitation of its competitor that has a higher dose requirement, thereby potentially achieving better therapeutic effects. NTS071 is anticipated to initiate Phase 1 clinical trial in second half of 2025 and expected to benefit patients with solid tumors harboring this mutation.

p53 Y220C mutation is widespread in various solid tumors. Studies show that there are 125,000 to 150,000 new cases worldwide annually, representing a significant market potential. Enriched with years of accumulated experience with its proprietary computation-based allosteric drug development technology and breakthrough innovation capabilities at Nutshell Therapeutics, NTS071 has the potential to stand out among peer products and become the most competitive drug molecule for this target.

The NTS071 Poster presented at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) (ENA) 2024 conference can be accessed here: View Source

On May 3, 2025 TAE Life Sciences (TLS) reported the signing of a Letter of Intent to collaborate with The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James) in the development and evaluation of novel boron-based drug compounds—an essential component of Boron Neutron Capture Therapy (BNCT) (Press release, TAE Life Sciences, MAY 3, 2025, View Source [SID1234652484]).

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This partnership marks the first-of-its-kind U.S.-based academic-industry alliance dedicated to accelerating BNCT drug innovation and translational research. By optimizing boron delivery agents, TLS and OSUCCC aim to unlock BNCT’s full potential as a powerful, highly selective, and relatively non-toxic therapy and positioning it as a new modality in the next era of precision oncology.

"We are entering a pivotal moment in cancer therapy," said Dr. Arnab Chakravarti, Chair and Professor of Radiation Oncology, Klotz Family Chair of Cancer Research and Director of the Brain Tumor Program at Ohio State. "BNCT offers an unprecedented opportunity to treat malignancies that are otherwise unresponsive to conventional therapies, while sparing healthy tissue. Our laboratory is uniquely poised to lead this next wave of boron drug innovation, and we are excited to collaborate with TAE Life Sciences in accelerating this effort on a global scale."

The initial focus of the collaboration will be the preclinical evaluation of TLS’s proprietary boron-10 drugs in cellular and animal models, leveraging Ohio State’s specialized neutron source optimized for BNCT research. In parallel, the teams will pursue joint development of next-generation boron drugs for BNCT and adjacent applications—unlocking new possibilities in precision cancer treatment.

"This partnership marks a major leap forward for the future of BNCT," said Robert Hill, CEO of TAE Life Sciences. "We are deeply honored to collaborate with Dr. Chakravarti and his world-class team of research and clinical experts. Their pioneering track record of leadership and innovation will be instrumental not only in advancing the science behind novel boron drug compounds but also in paving the way toward potential regulatory approval. Together, we’re creating a dynamic force for innovation that has the potential to redefine cancer care."

Hill notes that this partnership establishes a key pillar for accelerator-based BNCT and significantly accelerate the path to breakthrough cancer therapies, positioning the United States to have a leading position in the next wave of innovation in precision oncology. Hill emphasized that the growing body of international clinical evidence—particularly from Japan, Taiwan, and Europe validates BNCT’s ability to deliver durable responses in patients with otherwise untreatable cancers. This collaboration will help position the United States in having a leading position in advanced drug development and establishing the next wave in precision medicine.

On May 2, 2025 Genmab A/S (Nasdaq: GMAB) reported its intention to submit in the first half of 2025 a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) for subcutaneous epcoritamab, a bispecific antibody being investigated in combination with rituximab and lenalidomide (R2) for the treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma (FL), following at least one prior systemic therapy (Press release, Genmab, MAY 2, 2025, View Source [SID1234652444]).

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The decision to submit the sBLA is supported by positive topline results from the Phase 3 EPCORE FL-1 trial evaluating epcoritamab plus R2 versus R2 alone in adult patients with R/R FL. Based on an interim analysis conducted by an Independent Data Monitoring Committee (IDMC) review, the study met one of its dual primary endpoints of ORR (Complete Response plus Partial Response, p-value < 0.0001). The safety profile of epcoritamab plus R2 in adult patients with R/R FL was consistent with the known safety profiles of the individual regimens (epcoritamab and R2) and as presented in the U.S. prescribing information for epcoritamab. No new safety signals were observed. The full results will be submitted later this year for presentation at an upcoming medical congress and discussed with global regulatory authorities.

"We are pleased with the strength of the data that allows us to submit a supplemental Biologics License Application in accordance with the U.S. FDA’s Project Frontrunner, which supports our commitment to advance novel medicines to patients who need them. The interim topline results demonstrate the potential of this investigational epcoritamab combination regimen to treat relapsed or refractory follicular lymphoma patients," said Jan van de Winkel, Ph.D., Chief Executive Officer, Genmab. "This milestone represents our commitment to the ongoing development of epcoritamab, with our partner AbbVie, and we look forward to seeing the full results from the study."

Use of epcoritamab plus R2 in R/R FL is not approved in the U.S., in the EU or in any other territory. The safety and efficacy of epcoritamab for use as a combination therapy in FL have not been established. Epcoritamab is currently approved by the FDA under Accelerated Approval as a monotherapy for the treatment of adults with R/R FL after two or more lines of systemic therapy.

About Follicular Lymphoma (FL)
FL is typically an indolent (or slow-growing) form of non-Hodgkin’s lymphoma (NHL) that arises from B-lymphocytes and is the second most common form of NHL accounting for 20-30 percent of all cases.i About 15,000 people develop FL each year in the U.S.ii and it is considered incurable with current standard of care therapies.iii Patients often relapse and, with each relapse the remission and time to next treatment is shorter.iv Over time, transformation to diffuse large B-cell lymphoma (DLBCL), an aggressive form of NHL associated with poor survival outcomes, can occur in more than 25 percent of FL patients.v

About the EPCORE FL-1 Trial
EPCORE FL-1 (NCT05409066) is a Phase 3 open-label interventional trial to evaluate the safety and efficacy of epcoritamab plus rituximab and lenalidomide (R2) versus R2 alone in patients with relapsed/refractory (R/R) follicular lymphoma (FL). The dual primary endpoints are ORR and progression-free survival assessed by independent review committee (IRC) per Lugano criteria.

About Epcoritamab
Epcoritamab is an IgG1-bispecific antibody created using Genmab’s proprietary DuoBody technology and administered subcutaneously. Genmab’s DuoBody-CD3 technology is designed to direct cytotoxic T cells selectively to elicit an immune response toward target cell types. Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B cells and induces T-cell-mediated killing of CD20+ cells.vi

Epcoritamab (approved under the brand name EPKINLY in the U.S. and Japan, and TEPKINLY in the EU) has received regulatory approval in certain lymphoma indications in several territories. Epcoritamab is being co-developed by Genmab and AbbVie as part of the companies’ oncology collaboration. The companies will share commercial responsibilities in the U.S. and Japan, with AbbVie responsible for further global commercialization. Both companies will pursue additional international regulatory approvals for the investigational R/R FL indication and additional approvals for the R/R DLBCL indication.

Genmab and AbbVie continue to evaluate the use of epcoritamab as a monotherapy, and in combination, across lines of therapy in a range of hematologic malignancies. This includes five ongoing Phase 3, open-label, randomized trials including a trial evaluating epcoritamab as a monotherapy in patients with R/R DLBCL compared to investigators choice chemotherapy (NCT04628494), a trial evaluating epcoritamab in combination with R-CHOP in adult patients with newly diagnosed DLBCL (NCT05578976), a trial evaluating epcoritamab in combination with rituximab and lenalidomide (R2) in patients with R/R FL (NCT05409066), a trial evaluating epcoritamab in combination with rituximab and lenalidomide (R2) compared to chemoimmunotherapy in patients with previously untreated FL (NCT06191744), and a trial evaluating epcoritamab in combination with lenalidomide compared to chemotherapy infusion in patients with R/R DLBCL (NCT06508658). The safety and efficacy of epcoritamab has not been established for these investigational uses. Please visit www.clinicaltrials.gov for more information.