On May 18, 2023 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead asset mitazalimab for the treatment of pancreatic cancer (Press release, Alligator Bioscience, MAY 18, 2023, View Source [SID1234631836]).

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Mitazalimab is a monoclonal antibody targeting CD40 with the potential to sensitize tumors to chemotherapy and induce immune mediated tumor killing by activating dendritic cells, B cells, and macrophages. Mitazalimab is currently being evaluated in OPTIMIZE-1, a Phase 2 open-label, multi-center study to assess its safety and efficacy in combination with chemotherapy, mFOLFIRINOX, in previously untreated patients with metastatic pancreatic ductal adenocarcinoma (NCT04888312).

In January 2023, Alligator announced strong interim results from OPTIMIZE-1, in which mitazalimab combined with mFOLFIRINOX demonstrated an objective response rate (ORR) of 52% in 23 evaluable patients, as per the Response Evaluation Criteria in Solid Tumors (RECIST 1.1). In comparison, a similar patient population treated only with FOLFIRINOX reported an ORR of around 32%[1]. Disease control rate, the proportion of patients with objective response or stabilization of disease, was more than 90%. In April 2023, Alligator announced that OPTIMIZE-1 had been fully enrolled.

"This designation is a key milestone for our lead asset mitazalimab, which is producing outstanding clinical results in its Phase 2 trial in pancreatic cancer," said Søren Bregenholt, CEO of Alligator Bioscience. "Orphan designation confers significant benefits in the form of cost savings during development and marketing exclusivity following approval, and we are very pleased to see the potential of mitazalimab being recognized with the award of this designation."

Orphan designation is granted by the FDA to a drug or biological product to prevent, diagnose or treat a rare disease or condition, and it qualifies sponsors for various incentives, including seven years of market exclusivity after approval, exemption from user fees and a tax credit of qualified clinical trials.

The orphan designation for mitazalimab and the positive interim results from OPTIMIZE-1 will be a key component of discussions with regulatory authorities regarding subsequent clinical development and approval pathway for mitazalimab in pancreatic cancer. Additional interim data from OPTIMIZE-1, including Progression Free Survival, are due in mid-2023 and full top-line data are expected in the beginning of Q1 2024.

On May 18, 2023 Imugene Limited (ASX: IMU), a clinical stage immuno- oncology company, reported that its onCARlytics technology, in combination with Eureka Therapeutics, Inc.’s ARTEMIS cell receptor platform, has presented preclinical data at the American Society of Gene and Cell Therapy’s Annual Meeting (ASGCT) (Free ASGCT Whitepaper) demonstrating enhanced anti-tumour activity in vivo against hepatocellular carcinoma (liver cancer) tumours (Press release, Imugene, MAY 18, 2023, View Source [SID1234631817]).

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The data, presented as a poster presentation at the ASGCT (Free ASGCT Whitepaper) conference held in Los Angeles, titled ‘Effective combination immunotherapy using onCARlytics and ARTEMIS CD19 T cells against hepatocellular carcinoma’, investigates the combination in the most common type of primary liver cancer and sixth most common cancer worldwide.

Hepatocellular carcinoma (HCC) occurs most often in people with chronic liver diseases, such as cirrhosis caused by hepatitis B or hepatitis C infection.

Currently, there are few systemic therapies available for patients with advanced disease in addition to the traditional treatments including ablation, surgical resection, and liver transplantation. CD19-targeting chimeric antigen receptor (CAR) T cell therapy has demonstrated impressive clinical outcomes in blood cancers, but translating this therapy to solid-tumour cancers has met various challenges, including the immunosuppressive microenvironment, on-target off-tumour toxicity, and antigen heterogeneity. To date, CAR T cell therapies against HCC have shown nominal efficacy in clinical trials. Therefore, development of novel and innovative therapeutic approaches against HCC is needed to overcome the challenges and improve clinical outcomes.

onCARlytics in combination with ARTEMIS T cells potentially provide a solution. ARTEMIS T cells differentiate from CAR T cells with lower CRS risks, better tumour infiltration, and higher T cell persistence in pre-clinical studies, making them ideal cell therapy candidates for solid tumours.

The ASGCT (Free ASGCT Whitepaper) event is now in its 26 th year and attracts a range of professionals in the area of gene and cell therapy, who observe new scientific research and technologies alongside peers in the industry. It is being held 16-20 May 2023 at the Los Angeles Convention Center, CA, USA.

The poster presentation can be viewed on the Imugene website.

On May 17, 2023 Modiblast GmbH, a preclinical-stage biopharmaceutical company developing novel immunomodulatory cancer therapies, reported that its lead development program MB101 has passed a recent scientific advice and pre-CTA meeting with the Bundesinstitut für Arzneimittel und Medizinprodukte (BfArM) – Germany’s Federal Institute for Drugs and Medical Devices – without objections. Achieving this milestone significantly increases the chances to get final regulatory approval and expedites the transition of the Company’s lead development program into a planned Phase 1/2 clinical trial in acute myeloid leukemia (AML) and Myelodysplastic syndromes (MDS).

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"Today’s news is a significant step forward for Modiblast and paves the way for a swift transition into clinical-stage development once the necessary funding can be secured. The meeting outcome validates that Modiblast has addressed all safety aspects sufficiently to initiate a clinical study, that our preclinical data package is considered adequate, and that the study design is well substantiated. I want to thank all our collaborators and clinical partners that have contributed to achieving this milestone," commented Prof. Dr. rer. nat. Helga Schmetzer, Managing Director and Founder of Modiblast.

Modiblast’s therapeutic approach combines two crucial factors to turn ‘foe’ into ‘friend’. A myeloid differentiation factor fosters the creation of dendritic cell progenitor cells and hematological recovery. The patented combination with a danger signal and maturation factor drives the formation of dendritic cells (DCs). Both signals combined trigger the reprogramming of cancer blast into dendritic cells of leukemic origin, or DCleu, and boost the creation of healthy DC. Our first program, MB101, aims for a fixed-dose combination of granulocyte-macrophage colony stimulation factor (GM-CSF), and prostaglandin E1 (PGE1) as a ‘danger signal’.

(Press release, Modiblast, MAY 17, 2023, https://www.modiblast.com/modiblast-mb101-program-passes-bfarm-scientific-advice-and-pre-cta-meeting-without-objections/ [SID1234662144])

On May 17, 2023 OmniNano Pharmaceuticals LLC, a developmental stage start-up company, reported that it has been awarded $2.7 million from the Cancer Prevention and Research Institute of Texas (CPRIT) to develop ONP-001, OmniNano’s lead drug candidate, for treating pancreatic ductal adenocarcinoma (PDAC) (Press release, OmniNano Pharmaceuticals, MAY 17, 2023, View Source [SID1234656610]). 1 Pancreatic cancer is a devastating disease with a 5-year survival rate of just 12.5% and an overall median survival for PDAC of less than 12 months.2 Currently, no effective targeted therapy or immunotherapy is available for PDAC.

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"We are very excited to be awarded this SEED product development research grant. It validates the value of the patented platform technology and comprehensive lab and animal works already performed," says Guorong Ma, PhD, Chief Executive of OmniNano. "We are very appreciative to CPRIT and its review committee for recognizing our technology and proposed IND-enabling studies. This grant is critical for us to speed up our developmental work and facilitate further private investment. We are eager to advance our product, ONP-001, to the clinical trial phase to improve outcomes and extend life in pancreatic cancer patients, who are in desperate need of effective treatment."

ONP-001 consists of novel polymeric micelles encapsulating two naturally occurring drug compounds: one is an FDA-approved chemotherapeutic drug, and the other is capable of depleting cancer stem cells and modulating tumor stroma to overcome drug resistance, enhance drug delivery efficiency, and display significant antitumor activity on preclinical PDAC models.

On May 17, 2023 Biomissile, a biotech company developing fully human domain antibody (UDABTM) and multi-specific antibody (UDAB-MTM) reported the successful completion of a ￥200M (CNY) Series B fundraising (Press release, Biomissile, MAY 17, 2023, View Source;article_category_id=1&article_id=16 [SID1234644276]). Proceeds from the financing will be used to advance the company’s UDAB-MTM based therapeutic pipeline, and further extend its scientific and technical leadership. Series B financing was led by Kequan Fund, along with continued support by existing investor, Fosun Health Capital.

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The news in Chinese can be found here: View Source