On April 18, 2024 Pacylex Pharmaceuticals Inc. (Pacylex) is a clinical-stage pharmaceutical company focused on the development of a new class of targeted therapies, N-myristoyltransferase inhibitors (NMTi) for the treatment of hematologic and solid tumor cancers, reported that the first patient has been dosed in a Phase 2a expansion study in patients with refractory metastatic colorectal cancer (Press release, Pacylex Pharmaceuticals, APR 18, 2024, View Source [SID1234645052]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

The Phase 1 dose escalation safety and tolerability study was conducted in 29 heavily pre-treated solid tumor and lymphoma patients who averaged 4 lines of prior drug therapy on which they relapsed or were refractory. Eight colorectal cancer patients were among those included in the study. The most common treatment related adverse events identified in the trial were mild to moderate gastrointestinal side effects which were self-limiting and occurred in a minority of patients. A recommended Phase 2 dose (RP2D) for expansion studies was established. Zelenirstat prolonged progression free and overall survival in Phase 1 solid tumor patients receiving the RP2D, compared to those receiving lower doses. Prolonged Stable Disease of 6 months or longer was observed in 57% (4/7) of the patients receiving RP2D, including a patient with metastatic colorectal cancer continuing on treatment for more than 1 year with reductions of approximately 50% in CEA (carcinoembryonic antigen) and tumor volumes.

"Given the patient benefits and excellent tolerability seen in our Phase 1 study, and particularly in our sentinel colorectal cancer patient, we are eager to see if other colorectal cancer patients receiving our Phase 2 dose will also benefit," said Dr. John Mackey, CMO of Pacylex. "People with refractory colorectal cancer desperately need new treatment options, and we are investigating whether zelenirstat can provide that."

"Adding colorectal cancer as our first solid tumor indication explores the broad potential for zelenirstat, a first-in-class oral cancer therapy, in both hematologic cancers and solid tumors," said Pacylex CEO Dr. Michael Weickert. "The importance of myristoylation to many cancer processes is underappreciated, and Pacylex is way ahead in developing this novel therapeutic class, NMT inhibitors, to treat cancer refractory to available treatments."

The Company has an ongoing Phase 2a study of patients with B-cell non-Hodgkin lymphoma. This colorectal cancer Phase 2a expansion study will enroll up to 20 subjects to assess the preliminary clinical activity and confirm the safety of zelenirstat in refractory colorectal cancer.

On April 18, 2024 EditCo Bio, Inc., a pioneer at the forefront of genome engineering innovation, reported the launch of its Knockout CD4+ T-cell Pools (Press release, EditCo Bio, APR 18, 2024, View Source [SID1234642167]). This latest addition to EditCo Bio’s product lineup marks a significant leap forward in CRISPR-mediated gene editing, providing the scientific community with an advanced tool for primary T-cell editing that surpasses the capabilities of traditional methodologies.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

With the advent of CRISPR-Cas9 technology, genetic engineering has entered a new era of precision, particularly in the realm of primary immune cell editing. T-cells, pivotal in the immune response, hold immense promise for elucidating the pathways of various diseases, including cancer and autoimmune conditions. Yet, conventional T-cell editing techniques have often fallen short, plagued by scalability issues and inconsistent outcomes.

Leveraging the power of EditCo Bio’s cutting-edge, automated platform, researchers can now bypass the challenging optimization steps and access highly functional edited T-cells ready for immediate use. This innovation not only accelerates the journey from research discovery to clinical application but also delivers several key benefits:

Rapid Results: A novel 7-day protocol harnesses EditCo Bio’s unique multi-guide technology to achieve knockout efficiencies beyond 90% in primary human CD4+ T-cells.
Dependable Performance: Post-editing, CD4+ cell pools maintain over 80% viability before cryopreservation and can expand 40-fold over 14 days, regardless of TCR stimulation.
Optimal Functionality: After cryo-recovery, these CD4+ T-cell pools exhibit remarkable functional integrity, matching control cells in IL2, IFNG, and TNFA protein production post-PMA/ionomycin stimulation. Further, cultured cells retain over 90% viability and maintain editing efficiency for at least 14 days.
"For too long, the potential of primary T-cell editing has been constrained by the technical limitations of existing methods," said Travis Maures, CSO of EditCo Bio. "With the launch of our Knockout CD4+ T-cell Pools, EditCo Bio is breaking down these barriers, offering researchers the precision, efficiency, and scalability needed to advance the frontiers of immunotherapy and cellular research while allowing them the benefit of bypassing tedious protocol optimization. This innovation is a testament to our commitment to empowering scientists worldwide, driving forward the possibilities of gene editing to address some of the most pressing challenges in research today."

The introduction of Knockout CD4+ T-cell Pools is a strategic extension of EditCo Bio’s comprehensive Engineered Cell portfolio. It further solidifies the company’s position as a leader in cell engineering solutions that includes knockout and knock-in technologies for immortalized cells and iPSCs. EditCo Bio plans to expand the range of its edited primary cells products beyond CD4+ T-cells into other primary cell subsets that will be commercially available to researchers worldwide later this year.

For more information regarding EditCo Bio’s CD4+ T-cell Pools, visit www.EditCo.bio/contact.

On April 18, 2024 Azitra, Inc. (NYSE American: AZTR), a clinical-stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, reported it will be presenting promising new preclinical data from its platform built (or optimized) to discover treatments for serious skin diseases at three upcoming scientific and medical conferences (Press release, Azitra, APR 18, 2024, View Source [SID1234642166]):

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

ASGCT (American Society of Gene and Cell Therapy)
Baltimore, MD

Title: Engineered Staphylococcus Epidermidis as a Protein Delivery System for Treating Skin Diseases
Format: Oral presentation
Date: May 10th, 4:00pm – 4:15pm EDT

Title: Staphylococcus epidermidis Strain Expressing LEKTI-D6 (ATR12-351) for Netherton Syndrome
Format: Oral presentation
Date: May 10th, 5:15pm – 5:30pm EDT

SID (Society of Investigative Dermatology)
Dallas, TX

Title: Cutaneous delivery of LEKTI via an engineered strain of Staphylococcus epidermidis for the treatment of Netherton syndrome
Format: Oral presentation
Date: Friday May 17th, 2024 8:30 AM – 11:00 AM CDT

Title: Staphylococcus epidermidis for the topical treatment of epidermal growth factor receptor (EGFR) inhibitor-induced dermal toxicity
Format: Oral presentation
Date: Friday May 17th, 2024 8:30 AM – 11:00 AM CDT

Title: Clinical study of Netherton syndrome treated topically with a Staphylococcus epidermidis strain expressing recombinant human LEKTI-D6 (ATR12-351)
Format: Poster presentation
Date: Friday May 17th, 2024 4:00 – 6:00 PM CDT

ASCO (American Society of Clinical Oncology)
Chicago, IL

Title: Preclinical development of ATR04-484, an auxotrophic strain of Staphylococcus epidermidis, for the topical treatment of epidermal growth factor receptor (EGFR) inhibitor-induced dermal toxicity
Format: Online abstract
Date: Thursday, May 23rd, 2024 5:00 PM EDT

On April 18, 2024 Lumicell, Inc., a privately held company focused on developing innovative fluorescence-guided imaging technologies for cancerous tissue detection during surgery, reported the U.S. Food & Drug Administration (FDA) approved the company’s New Drug Application (NDA) for its LUMISIGHT (pegulicianine) optical imaging agent and its Premarket Approval (PMA) application for Lumicell Direct Visualization System (DVS), together referred to as LumiSystem (Press release, Lumicell Diagnostics, APR 18, 2024, View Source [SID1234642165]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

With 84% diagnostic accuracy, LumiSystem enables surgeons to scan the breast cavity post-lumpectomy, in real-time, to detect and resect residual cancer that may have otherwise been missed, potentially sparing some patients from second surgeries.1-2 The LumiSystem combination is indicated for fluorescence imaging in adults with breast cancer as an adjunct for the intraoperative detection of cancerous tissue within the resection cavity following removal of the primary specimen during lumpectomy surgery.

"We are immensely proud of the dual approval of LUMISIGHT and Lumicell DVS – we believe this is the first drug-device combination product approved in over a decade to have followed both of the FDA’s most stringent NDA and PMA review processes,"3 said Howard Hechler, President and Chief Operating Officer, Lumicell. "With the FDA’s approval, LumiSystem is now the first and only imaging combination product capable of detecting cancerous tissue where it matters most, inside the breast cavity."

"Breast cancer is all too common, and sadly, 1 in 8 women will develop it during their lifetime,"4 said Kelly Hunt, MD, Chair of the Department of Breast Surgical Oncology at MD Anderson Cancer Center and President of the Society of Surgical Oncology. "Our most common surgical procedure to treat these women is lumpectomy. Unfortunately, the intraoperative tools we have are limited and do not identify the extent of tumor accurately enough, making it challenging to achieve a complete tumor resection, leading to as many as 36% of patients needing a second surgery."5

"Up to 65% of the time, we do not find residual cancer in the second surgery and are left wondering if we performed an unnecessary surgery due to a false positive margin assessment or if the cancer was missed again in the second surgery,"6 said Irene Wapnir, MD, Breast Surgical Oncologist and Professor of Surgery, Stanford University School of Medicine.

"During lumpectomy surgery, surgeons still struggle to identify and remove all of the tumor during the first operation,"7 said Barbara Smith, MD, PhD, Director of the Breast Program at Massachusetts General Hospital and Professor of Surgery at Harvard Medical School. "With LumiSystem, we will now have a technology that is clinically proven to achieve a more complete cancer resection during lumpectomy that could help some patients avoid a second surgery."2

The safety of the system was established using data from more than 700 breast cancer patients across five clinical studies at top academic and regional community cancer centers across the U.S. The most common side effects with LUMISIGHT are hypersensitivity and an abnormal color in urine. LUMISIGHT may cause serious hypersensitivity reactions, including anaphylaxis. Results of the Investigation of Novel Surgical Imaging for Tumor Excision (INSITE) pivotal trial, used to support the efficacy of the system, were published in NEJM Evidence.

"We want to thank our clinical investigators and the hundreds of women who participated in our breast program for LUMISIGHT and Lumicell DVS," said Jorge Ferrer, Chief Scientific Officer, Lumicell. "Due to your important contributions, LUMISIGHT and Lumicell DVS are now approved and will be available in the United States shortly."

Please visit www.LumiSystem.com to learn more about LUMISIGHT and Lumicell DVS.

On April 18, 2024 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a leader in the development of Antibody Radiation Conjugates (ARCs) and other targeted radiotherapies, reported that results from the Phase 3 SIERRA trial of Iomab-B were presented in an oral presentation at the 50th Annual European Bone Marrow Transplant Society Meeting (EBMT) held in Glasgow, Scotland on April 14-17 (Press release, Actinium Pharmaceuticals, APR 18, 2024, View Source [SID1234642164]). The results showed that an Iomab-B led bone marrow transplant (BMT) results in higher rates of remissions and durable Complete Remission (dCR), which is the primary endpoint of the SIERRA trial, as well as significant improvement in overall survival in TP53 positive patients. Iomab-B is a targeted radiotherapeutic comprised of an anti-CD45 monoclonal antibody with the Iodine-131 radioisotope payload. The Phase 3 SIERRA trial enrolled 153 patients age 55 and above with active relapsed or refractory acute myeloid leukemia (AML) and compared outcomes of patients receiving Iomab-B BMT to those of patients receiving physician’s choice of care in the control arm. In total, 24% (37/153) of the patients enrolled on SIERRA had a TP53 mutation, which is associated with limited treatment options and poor outcomes.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Data highlighted in the ASH (Free ASH Whitepaper) oral presentation, which can be accessed on the investor relations page of Actinium’s website, included:

Response rates by TP53 Mutation Status:

Iomab-B & Crossover

Control Arm

TP53 Positive

N=27

N=10

CR

55.56% (15/27)

0 %

dCR

14.81% (4/27)

0 %

TP53 Wildtype

N=93

N=23

CR

58.06% (54/93)

17.39% (4/23)

dCR

16.13% (15/93)

0 %

Overall Survival in Patients with a TP53 Mutation:

Iomab-B & Crossover

Control Arm

Median OS

5.49 months

1.66 months

Number of Patients

27

10

Hazard Ratio

0.23

p-value

0.0002

Median OS was 6.37 months in TP53 negative patients receiving Iomab-B and 5.72 months for TP53 positive patients demonstrating Iomab-B’s ability to overcome TP53 gene mutations.

Dr. Hannah Choe, Assistant Professor of Medicine at Ohio State University and SIERRA trial investigator, commented, "TP53 mutations are associated with very poor outcomes due to resistance to anti-leukemic therapies with patients rarely offered access to potentially curative transplantation. The SIERRA trial showed that Iomab-B was well tolerated and can enable unprecedented access to transplant in this patient population and induce high complete remission rates despite active, relapsed/refractory disease and a TP53 mutation. These results were very well received at EBMT and demonstrate the novelty and safety of a CD45-directed antibody radiation conjugate. More importantly, we see that these response rates translated into improved overall survival, overcoming the increased risk associated with TP53 mutation while no other viable treatment options exist. We are excited for Iomab-B’s potential use and safety for disease control in patients with a TP53 mutation."

About the EBMT Annual Meeting

The Annual Meeting of the EBMT is attended by more than 5,500 participants, including physicians, nurses, data managers, statisticians, quality managers, cell therapists, paediatricians, pharmacists, psychologists, psychiatrists and psychoanalysts, transplant coordinators, lab scientists, trainees, and patients. This important congress ensures and encourages dialogues and information exchange, education and scientific productivity.

The full annual meeting program is available online at:

View Source