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Modiblast MB101 program passes BfArM scientific advice and pre-CTA meeting without objections

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])

OmniNano Pharmaceuticals Awarded $2.7 Million SEED Product Development Research Grant from Cancer Prevention and Research Institute of Texas (CPRIT)

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.

"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.

Biomissile closed Series B fundraising of ￥200M (CNY) to advance its UDABTM and UDAB-MTM therapeutic pipeline

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.

The news in Chinese can be found here: View Source

Panolos, a new anti-cancer drug ‘PB101’ begins human trials

On May 17, 2023 Panolos Biosciences reported that the company has initiated the first human clinical trials for its anti-cancer drug, ‘PB101’, which targets all variants of vascular endothelial growth factor (VEGF) (Press release, Panolos Bioscience, MAY 17, 2023, View Source [SID1234633692]). This event marks a significant milestone as no drug has so far been introduced that entirely blocks placental growth factor (PlGF) within the VEGF family. The move is expected to revolutionize the field of cancer treatment. The Ministry of Food and Drug Safety (MFDS) has given the go-ahead for the phase 1 clinical trial plan (IND) for ‘PB101’ as of May 15. This comes after Panolos submitted an IND to the MFDS in the latter part of last year.

Cancer cells generate abnormal blood vessels around them by secreting angiogenic factors to derive the necessary nutrients for survival. VEGF-A (vascular endothelial generating factor A) is one such angiogenic factor that facilitates angiogenesis by binding with VEGF receptors. However, unstable blood vessels around cancer cells can undermine the effectiveness of intravenous anti-cancer drugs. Strategies like angiogenesis inhibition, as seen with Roche’s ‘Avastin’, have been formulated. Nonetheless, Avastin, while blocking VEGF-A, crucial for placental formation, could induce the expression of Placental Growth Factor (PlGF) in cancer cells, indicating a potential adverse prognosis for cancer patients.

‘PB101’, a new anti-cancer drug candidate, seeks to enhance treatment efficacy by inhibiting all types of VEGF, including VEGF-A and PlGF. This drug was developed by Panolos in collaboration with numerous experts including Professor Hong Jae Chon and Chan Kim of the Department of Hematology and Oncology at CHA University Bundang Medical Center.

The company reveals that ‘PB101’ is a protein about two-thirds the size of a typical antibody and is capable of binding to both VEGF-A and PlGF. It contains fragments of the Fc (Fragment crystallizable) region, allowing it to remain in the body for an extended duration. The drug primarily comprises two sections, including the VEGF receptor 1 (VEGFR1), which can yield therapeutic effects. Panolos anticipates that ‘PB101’ will set itself apart due to its higher PlGF binding capability compared to existing drugs.

The phase 1 clinical trial approved by the MFDS is a dosage extension and expansion study. It aims to assess the safety, tolerability, pharmacokinetic/pharmacodynamic properties, and preliminary anti-tumor activity of PB101 in patients with advanced solid cancer. The study is being conducted at three locations: the CHA University Bundang Medical Center, The Catholic University of Korea Seoul St. Mary’s Hospital, and the Seoul National University Bundang Hospital.

Siren Biotechnology Unveils Preclinical Data for Universal AAV Immuno-Gene Therapy™ for Cancer, a Novel Modality that Combines AAV Gene Therapy and Cytokine Immunotherapy

On May 17, 2023 Siren Biotechnology reported the presentation of preclinical data demonstrating the effect of a novel treatment for cancer, Universal AAV Immuno-Gene Therapy, which combines AAV gene therapy and cytokine immunotherapy into a single modality (Press release, Siren Biotechnology, MAY 17, 2023, View Source [SID1234632787]). The oral presentation titled, "AAV immuno-gene therapy delivers vectorized cytokines to effectively treat high-grade gliomas," will be given by renowned AAV gene therapy expert and Siren’s Founder and Chief Executive Officer, Nicole Paulk, PhD, at 3:45pm PT at the ASGCT (Free ASGCT Whitepaper) 26th Annual Meeting, taking place in Los Angeles, May 16-20th. Dr. Paulk will describe how this precise and highly targeted approach represents a novel method for destroying tumor cells and eliciting anti-tumor immunity. Link here to review Siren’s ASGCT (Free ASGCT Whitepaper) abstract and here to review the company’s launch press release also issued this morning.

The Siren team conducted several experiments to evaluate the safety and efficacy of an immuno-gene therapy that utilizes AAV9 vectors expressing 12 different engineered immunomodulatory interferon (IFN) cytokine payloads comprising IFNɑ1, IFNβ, IFNƔ, and combinations thereof.

In primary human high-grade glioma (HGG) organoids, Siren’s AAV immuno-gene therapies rapidly and selectively reduced tumor size. In comparison, phosphate buffered saline, dimethyl sulfoxide diluent, and AAV9-GFP (green fluorescent protein) had no effect on tumor or healthy cerebral cells in HGG organoids, and HGG tumor cells grew uncontrollably. Temozolomide chemotherapy, the current standard of care for HGG, significantly decimated healthy cerebral cells and only slightly delayed HGG tumor growth.
In vivo data demonstrated that AAV immuno-gene therapies effectively reduced tumor burden in three HGG mouse models that received intratumoral administration of the cytokine payloads via convection-enhanced delivery (CED). Treatment with AAV immuno-gene therapies resulted in tumor regression and significantly prolonged survival in human GBM6 xenografts (P<0.2-0.001 and 31 – 60% complete responses (CRs)); mouse GL261 allografts (P<0.0009); and human patient-derived xenografts (P<0.04; 30% CR); n=450.
An evaluation of tumor-bearing mouse brains demonstrated marked tumor changes following treatment with AAV immuno-gene therapies. Within 48 hours following treatment, local intratumoral expression of Siren’s engineered IFNs had already led to widespread tumor cell apoptosis, and activation of brain-resident macrophages known as microglia, a class of antigen-presenting immune cells that specialize in clearing necrotic and apoptotic cells. By Day 7, there was reproducible tumor eradication, no evidence of residual proliferating cells, no remaining engineered cytokine payload expression, and only residual microglial activity.
Subsequent single cell sequencing on syngeneic mouse brain tumors following AAV immuno-gene therapy treatment showed that tumor cells exhibited a significant upregulation of genes linked to IFN responses, as well as other classic immune response genes. These responses were specific to IFN payload expression and not AAV.
"We’re excited to present these potentially transformative data as we simultaneously introduce Universal AAV Immuno-Gene Therapy and Siren Biotechnology," said Dr. Paulk. "This novel approach represents a new, big, bold idea to fight cancer by combining the potential of AAV gene therapy and cytokine immunotherapy into a single modality. Our vision is for Universal AAV Immuno-Gene Therapy to become the standard of care for any solid tumor cancer. Bolstered by the results shared today at ASGCT (Free ASGCT Whitepaper), we’ve started our vision-led journey with an initial focus on brain and eye cancers. Attempts to treat these cancers with systemic drugs have largely failed. The unmet need for these patients is dire, and our opportunity to help is massive."