On November 3, 2022 PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TPEx:6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, reported that new data on ropeginterferon-alfa-2b will be presented during the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, December 10-13, 2022 (Press release, PharmaEssentia, NOV 3, 2022, View Source [SID1234623039]).

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Data to be presented at the meeting further explore the clinical profile of ropeginterferon alfa-2b in PV patients, including interim results from a regional study as well as safety and efficacy data in different risk populations. The company will also present details of the planned ECLIPSE-PV Phase 3b clinical trial, which aims to support an amended dosing regimen for ropeginterferon alfa-2b.

"The latest research to be presented at ASH (Free ASH Whitepaper) will explore new insights on the profile of ropeginterferon alfa-2b as a therapeutic option for the treatment of PV," said Raymond Urbanski, M.D., Ph.D., U.S. Head of Clinical Development and Medical Affairs. "We look forward to convening with the scientific community to discuss this latest research and our continued plans to address unmet needs in the PV community."

ASH abstract details

A Phase 3b, Single-Arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of Ropeginterferon Alfa-2b-Njft (P1101) in Adult Patients with Polycythemia Vera
Abstract 3004 – Sunday, December 11, 2022, 6:00 PM-8:00PM
Phase II, Open-Label, Multicenter, Single-Arm Study Investigating the Efficacy and Safety of Ropeginterferon Alfa-2b in Chinese Patients with Polycythemia Vera Resistant or Intolerant to Hydroxyurea (HU)
Abstract 3050 – Sunday, December 11, 2022, 6:00 PM-8:00 PM
Efficacy and Safety of Long-Term Ropeginterferon Alfa-2b Treatment in Patients with Low-Risk and High-Risk Polycythemia Vera (PV)
Abstract 4345 – Monday, December 12, 2022, 6:00 PM-8:00 PM
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About Polycythemia Vera (PV)

Polycythemia vera (PV) is a cancer originating from a disease-initiating stem cell in the bone marrow resulting in a chronic increase of red blood cells, white blood cells, and platelets. PV may result in cardiovascular complications such as thrombosis and embolism, and often transforms to secondary myelofibrosis or leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to a set of acquired mutations, the most important being a mutant form of JAK2.1

About BESREMi (ropeginterferon alfa-2b-njft)

BESREMi is an innovative monopegylated, long-acting interferon. With its unique pegylation technology, BESREMi has a long duration of activity in the body and is aimed to be administered once every two weeks (or every four weeks with hematological stability for at least one year), allowing flexible dosing that helps meet the individual needs of patients.

BESREMi has orphan drug designation for treatment of polycythemia vera (PV) in adults in the United States. The product was approved by the European Medicines Agency (EMA) in 2019, in the United States in 2021, and has also received approval in Taiwan and South Korea. The drug candidate was invented by PharmaEssentia and is manufactured in the company’s Taichung plant, which was cGMP certified by TFDA in 2017 and by EMA in January 2018. PharmaEssentia retains full global intellectual property rights for the product in all indications.

BESREMi was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic and infectious disorders.

Please see full Prescribing Information, including Boxed Warning.

On November 3, 2022 Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for cancer, reported that an abstract detailing updated safety and efficacy data from the Company’s Phase 1 study of ADI-001 for the potential treatment of relapsed or refractory B-cell Non-Hodgkin’s lymphoma (NHL) was made available as part of the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which is being held December 10-13, 2022 in New Orleans, Louisiana (Press release, Adicet Bio, NOV 3, 2022, View Source [SID1234623038]). The abstract outlines a summary of clinical data as of a July 15, 2022 data-cut date. Clinical data will be provided during a poster presentation by Sattva Neelapu, M.D., from the MD Anderson Cancer Center at the ASH (Free ASH Whitepaper) Annual Meeting on Saturday, December 10, 2022.

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"The latest data-cut from our ongoing study of ADI-001 is extremely encouraging, as we continue to see complete responses and preliminary durability coupled with a benign safety profile in heavily pre-treated patients with aggressive NHL. We look forward to providing additional clinical data from a later data-cut date during the ASH (Free ASH Whitepaper) Annual Meeting," said Francesco Galimi, M.D., Ph.D., Chief Medical Officer of Adicet Bio. "With the data generated to date coupled with the pace of enrollment in dose level 4, we remain on track to initiate a potentially pivotal program for ADI-001 in the first half of 2023."

Data highlights as of the July 15, 2022 data-cut date included in the ASH (Free ASH Whitepaper) abstract were as follows:

Of the nine evaluable patients, three patients were treated at each of the three dose levels: dose level 1 (DL1; 30 million CAR+ cells), dose level 2 (DL2; 100 million CAR+ cells), and dose level 3 (DL3; 300 million CAR+ cells). Two patients at DL3 were re-dosed with a second course of ADI-001, per protocol. Six of nine were male (67%) and the median age was 62 years (range 45-75). There were eight patients with large B-cell lymphoma (LBCL) and one with mantle cell lymphoma. Of the eight patients with LBCL, five had diffuse-large B-cell lymphoma (DLBCL), two had high-grade B-cell lymphoma (HGBCL) with double/triple hit, and one had HGBCL not otherwise specified. Indolent lymphomas, such as follicular lymphoma, are currently excluded from the study.
Overall, the patients were heavily pretreated with a median number of prior therapies of four (range 2-5), and had a poor prognostic outlook as indicated by the median International Prognostic Index (IPI) score of four (range 2-4); the median tumor burden was 2,974 (150-7,919) mm2, and 89% (8/9) had stage III/IV disease.
The best overall response rate (ORR) and complete response rate (CR) was 78% (7/9). For the four patients who had prior autologous CD19 CAR T therapies, the ORR and CR rate was 100% (4/4). As of the data-cut date, of the seven patients who had achieved CR, two patients progressed, one died of unrelated causes while in complete remission and four were still in CR and in active follow up, with a range of follow-up time between 1.2 and 8.8 months.
CAR+ gamma delta T cell kinetics in the peripheral blood increased in a dose-dependent manner with peak cell expansion occurring between Days seven and 10 at DL3 based on flow cytometry.
Of the nine patients, there were no ≥ Grade 3 Cytokine Release Syndrome (CRS) or Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS) events. Two patients developed CRS: one Grade 1 and one Grade 2. At the time of the ASH (Free ASH Whitepaper) abstract submission, there were three reported related serious adverse events: Grade 2 CRS, Grade 1 ICANS and Grade 3 adenoviraemia. There was no reported graft versus host disease or protocol-defined dose-limiting toxicity events.
Response data were evaluated per Lugano 2014 criteria by independent radiographic review.
The full abstract is available online on the ASH (Free ASH Whitepaper) website.

Clinical data will be presented during a poster presentation by Sattva Neelapu, M.D. at the ASH (Free ASH Whitepaper) Annual Meeting on Saturday, December 10, 2022 from 5:30 – 7:30 p.m. CT. Adicet will provide data from a more recent data-cut date from the Phase 1 study in a press release and company webcast on Sunday, December 11, 2022 at 8:00 a.m. CT/ 9:00 a.m. ET.

Details of the poster presentation are as follows:

Session Title: 704. Cellular Immunotherapies: Early Phase and Investigational Therapies: Poster I
Title: A Phase 1 Study of ADI-001: Anti-CD20 CAR-Engineered Allogeneic Gamma Delta1 (γδ) T Cells in Adults with B-Cell Malignancies
Presenting Author: Sattva Neelapu, M.D., MD Anderson Cancer Center
Date/Time: Saturday, December 10, 2022 from 5:30 – 7:30 p.m. CT.

Adicet ADI-001 Webcast Information

The Company will host a webcast event on Sunday, December 11, 2022, at 8:00 a.m. CT/ 9:00 a.m. ET to discuss recent data from its ongoing Phase 1 clinical study of ADI-001 in relapsed or refractory aggressive B-cell NHL.

About ADI-001

ADI-001 is an investigational allogeneic gamma delta CAR T cell therapy being developed as a potential treatment for relapsed or refractory B-cell NHL. ADI-001 targets malignant B-cells via an anti-CD20 CAR and via the gamma delta innate and T cell endogenous cytotoxicity receptors. Gamma delta T cells engineered with an anti-CD20 CAR have demonstrated potent antitumor activity in preclinical models, leading to long-term control of tumor growth. In April 2022, ADI-001 was granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) for the potential treatment of relapsed or refractory B-cell NHL.

About the GLEAN Study

This Phase 1 study is an open-label, multi-center study of ADI-001 enrolling adults diagnosed with B-cell malignancies who have either relapsed, or are refractory to at least two prior regimens. The primary objectives of the study are to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of ADI-001, and to determine optimal dosing as a monotherapy. The study is expected to enroll approximately 75 patients. For more information about the clinical study design, please visit www.clinicaltrials.gov (NCT04735471).

On November 3, 2022 Ajax Therapeutics, Inc., a biopharmaceutical company applying computational chemistry and structure-based technologies to develop novel, selective small molecules for myeloproliferative neoplasms (MPNs), reported two abstracts have been accepted for oral and poster presentations at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, to be held December 10-13, 2022 in New Orleans (Press release, Ajax Therapeutics, NOV 3, 2022, View Source [SID1234623037]). The presentations highlight preclinical data on Ajax’s next-generation JAK2 inhibitors for the treatment of MPNs.

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On November 3, 2022 Asher Biotherapeutics, Inc. (Asher Bio), a biotechnology company focused on developing therapies to precisely engage specific immune cells to fight cancer, chronic viral infection and autoimmune disease, reported that it will present preclinical data showing that cis-targeted IL-2 or IL-21 cytokine fusion molecules selectively augment CAR-Ts in vitro and enhance in vivo anti-tumor activity and survival at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held in New Orleans, Louisiana, December 10-13, 2022 (Press release, Asher Biotherapeutics, NOV 3, 2022, View Source [SID1234623036]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Asher Bio’s cis-targeted cytokine fusions consist of a targeting antibody directed against a tag expressed on the CAR-T surface that is co-expressed with the CAR and a cytokine mutein with attenuated binding to its cognate cytokine receptor.

On November 3, 2022 Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapy candidates for patients with hematological and solid cancers and other serious diseases, reported an oral presentation of a real-world analysis comparing the effectiveness of omidubicel to other allo-HCT donor sources from the Center for International Blood and Marrow Transplant Research (CIBMTR) database (Press release, Gamida Cell, NOV 3, 2022, View Source [SID1234623035]). These data are being presented at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which is being held in New Orleans from December 10-13, 2022.

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"These data reinforce the clinical relevance of the rapid time to neutrophil engraftment observed in patients transplanted with omidubicel, and support the potential use of omidubicel in patients with hematologic malignancies requiring transplant. We are diligently preparing to bring this important therapy to patients upon potential FDA approval."

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"Data from our phase 3 study compared the safety and efficacy of omidubicel to standard cord blood. We are delighted to have the opportunity, in a podium presentation at ASH (Free ASH Whitepaper), to share the work we have done with CIBMTR, exploring their expansive real-world database and performing the first comparative efficacy analyses between omidubicel and other donor sources for patients undergoing allogeneic stem cell transplant," said Ronit Simantov, M.D., Chief Medical and Scientific Officer of Gamida Cell. "These data reinforce the clinical relevance of the rapid time to neutrophil engraftment observed in patients transplanted with omidubicel, and support the potential use of omidubicel in patients with hematologic malignancies requiring transplant. We are diligently preparing to bring this important therapy to patients upon potential FDA approval."

Details about the ASH (Free ASH Whitepaper) presentation are as follows:

Title: Clinical Outcomes Following Allogeneic Hematopoietic Cell Transplantation with Omidubicel or Other Donor Sources in Patients with Hematologic Malignancies: Comparison of Clinical Trial Results to Center for International Blood and Marrow Transplant Research Database Controls
Session Title: 732. Allogeneic Transplantation: Disease Response and Comparative Treatment Studies: Clinical Outcome: Real World Studies Based on Database Analyses
Lead Author: Smitha Sivaraman, PhD.
Time: Saturday, December 10, 2022, 2:00 p.m. – 3:30 p.m. EST (session time) and 2:30 p.m. EST (presentation time)
Location: Ernest N. Morial Convention Center, 391 – 392

A prospective cohort analysis study to compare the effectiveness of omidubicel versus other allo-HCT donor sources (MUD, MMUD and haploidentical) used in clinical practice is being presented. The study compared data from the omidubicel (n=52) and control arms (n=56) of the phase 3 study with a cohort of similar patients derived from the CIBMTR database (n = 807) who had undergone transplant with matched unrelated donors, mismatched unrelated donors, or haploidentical donors. The analysis showed that omidubicel was associated with more rapid neutrophil recovery (median: 10 days) compared to all other donor sources (median 15-20 days; p<0.001). While platelet recovery took longer in the omidubicel cohort, rates of severe acute and chronic graft versus host disease (GVHD) were comparable. Importantly, analyses of non-relapse mortality, disease-free survival, and overall survival showed similar results among all donor sources. While the phase 3 study compared omidubicel to standard cord blood, these real-world data reinforce the clinical utility of omidubicel as a graft source in patients in need of an allogeneic stem cell transplant.

About Omidubicel

Omidubicel is an advanced cell therapy candidate developed as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant for patients with blood cancers. Omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment in comparison to standard umbilical cord blood in an international, multi-center, randomized Phase 3 study (NCT0273029) in patients with hematologic malignancies undergoing allogeneic bone marrow transplant. The Phase 3 study also showed reduced time to platelet engraftment, reduced infections, and fewer days of hospitalization. One-year post-transplant data showed sustained clinical benefits with omidubicel as demonstrated by significant reduction in infectious complications as well as reduced non-relapse mortality and no significant increase in relapse rates nor increases in graft-versus-host-disease (GvHD) rates. Omidubicel is the first stem cell transplant donor source to receive Breakthrough Therapy Designation from the FDA and has also received Orphan Drug Designation in the US and EU.

The BLA for omidubicel has been assigned a Prescription Drug User Fee Act (PDUFA) target action date of January 30, 2023. If approved, omidubicel will be the first allogeneic advanced stem cell therapy donor source for patients with blood cancers in need of a stem cell transplant.

Omidubicel is an investigational stem cell therapy candidate, and its safety and efficacy have not been established by the FDA or any other health authority. For more information about omidubicel, please visit View Source

About NAM Technology

Our NAM-enabling technology is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (nicotinamide), we can expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.