On September 19, 2024 Xspray Pharma AB (publ) (Nasdaq Stockholm: XSPRAY), a pharmaceutical company leveraging its proprietary HyNap technology to develop enhanced cancer therapies, reported significant progress following a productive meeting with the U.S. Food and Drug Administration (FDA) (Press release, Xspray, SEP 19, 2024, View Source [SID1234649584]). The company plans to resubmit its New Drug Application (NDA) for Dasynoc in Q4 2024, incorporating feedback from the Complete Response Letter (CRL) issued by FDA in July 2024. If the FDA sets a two-month review period upon resubmission, the launch of Dasynoc could be expected as early as Q1 2025.

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 FDA recommends adjustments to Dasynoc’s tablet strengths to reduce the risk of medication errors. Although these changes are minor (within normal variability), the FDA has requested new batches to be produced before submission. Xspray Pharma has already initiated production of these batches. The company will also provide further clarification on the manufacturing process to ensure full alignment with the FDA’s requirements.

Per Andersson, CEO of Xspray Pharma, commented: "We are very encouraged by the positive and collaborative discussions with the FDA. The new tablet strengths will enhance patient safety by reducing the potential for dosing errors, and we are working diligently to meet all the necessary requirements. Our production process is well underway, and we are on track to resubmit the NDA in Q4 2024."

He continued: "Xspray Pharma is particularly eager to bring Dasynoc to market because of the critical need we have identified in our research. Many patients, especially those relying on pH-altering medications like antacids, face challenges with existing cancer treatments due to inconsistent absorption. Dasynoc’s innovative pH-independent formulation directly addresses this issue, which minimizes the risks associated with fluctuating drug absorption ensuring that patients can receive consistent treatments. We believe this solution will be welcomed by healthcare providers and patients alike, who are in need of more reliable treatment options."

Upon resubmission, Xspray Pharma expects the FDA to assign a new Prescription Drug User Fee Act (PDUFA) date, with a final decision anticipated within two or six months of the resubmission, depending on the review timeline set by the FDA.

On September 19, 2024, FibroBiologics, Inc. (the "Company") reported to have entered into a Master Services Agreement (the "Agreement") with Charles River Laboratories, Inc. ("CRL") (Filing, 8-K, FibroBiologics, SEP 19, 2024, View Source [SID1234646838]).

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 Agreement governs the general terms under which CRL will provide development and manufacturing services as specified by the Company. Pursuant to the Agreement, the Company and CRL will enter into various Statements of Work to set forth the services to be performed by CRL and the payments to be made by the Company for each project (each, a "Statement of Work"). Each Statement of Work will be governed by the terms of the Agreement, unless expressly modified in such Statement of Work.

The Agreement has a term that expires on September 19, 2029 (the "Term"), unless earlier terminated by either party in accordance with the Agreement. The Company may terminate any services, Statement of Work or the Agreement upon ninety (90) days’ prior written notice. CRL may terminate the Agreement upon ninety (90) days’ prior written notice; provided all Statements of Work then in effect, and all ongoing services thereunder, will remain in effect, and the terms of the Agreement will continue to apply to them as if the Agreement remained in effect. Either party may terminate any services, Statement of Work, or the Agreement upon ninety (90) days’ prior written notice for material breach of the Agreement by the other party if such breach is not remedied within the ninety (90) day notice period or if such breach is not capable of cure within such ninety (90) day period.

The Agreement contains representations, warranties and indemnity obligations customary for agreements of this type.

The foregoing description of the Agreement does not purport to be complete and is qualified in its entirety by reference to the full text of the Agreement, which has been filed as Exhibit 10.1 to this Current Report on Form 8-K and is incorporated herein by reference.

On September 19, 2024 TME Pharma N.V. (Euronext Growth Paris: ALTME), a clinical stage biotechnology company focused on developing novel therapies for treatment of cancer by targeting the tumor microenvironment (TME), reported that an abstract highlighting NOX-A12 data from preclinical studies performed by the U.S. National Cancer Institute (NCI) has been selected for poster presentation at the 2024 Society for Neuro-Oncology (SNO) Annual Meeting, taking place in Houston, Texas, USA, November 21-24, 2024 (Press release, TME Pharma, SEP 19, 2024, View Source [SID1234646758]). NCI is part of the National Institutes of Health.

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 research was conducted at the NCI under the material transfer agreement established with TME Pharma in June 2022 to explore the effects of TME Pharma’s CXCL12 inhibitor NOX-A12 in brain tumors.1

The SNO Annual Meeting, the world’s largest neuro-oncology conference, attracts researchers and clinician scientists from over 40 countries. As a premier forum for sharing knowledge and gaining insights into the future of neuro-oncology research and treatment, the meeting spans all neuro-oncology disciplines, professions, and interests. TME Pharma will participate at the annual event as a supporting partner, with its team attending the conference and available for meetings with industry leaders.

Details of the poster presentation at the 2024 SNO Annual Meeting are as follows:

Title: Potentiating the efficacy of immune check-point inhibitors in glioblastoma by inhibition of CXCL12
Presenter: Dr. Chen Cam-El Makranz, Neuro-Oncology Research Fellow, National Cancer Institute, National Institutes of Health
Session: Poster Session, Poster number EXTH12
Time and Date: 7.30-9.30 p.m. CDT, Friday, November 22, 2024

The full abstract will be published in the SNO official journal Neuro-Oncology on Friday, November 11, 2024. Both the abstract and poster presentation will be made available on the TME Pharma website following the conference.

On September 19, 2024 Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, reported ADI-001 clinical biomarker data from the Phase 1 GLEAN trial which further reinforces the potential of ADI-001 as a best-in-class allogeneic cell therapy for autoimmune diseases (Press release, Adicet Bio, SEP 19, 2024, View Source [SID1234646757]). Notably, ADI-001 demonstrated robust tissue trafficking resulting in high levels of ADI-001, significant chimeric antigen receptor (CAR) T cell activation, and complete CD19+ B cell depletion in secondary lymphoid tissue. These data will be presented by Dr. Blake Aftab, Chief Scientific Officer, at the 9th Annual CAR-TCR Summit on Thursday, September 19, 2024 in Boston, MA.

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!

"These results clearly support the potential of ADI-001 and Adicet’s off-the-shelf gamma delta CAR T cell platform, by demonstrating robust trafficking and complete B cell depletion in tissue, while providing superior exposure of ADI-001 in secondary lymphoid tissue compared to published third-party data reported for alpha-beta CAR T therapies," said Blake Aftab, Ph.D., Chief Scientific Officer of Adicet Bio. "Together, the totality of our findings provide multiple levels of evidence highlighting the significant advantages of our approach and present a compelling opportunity for ADI-001 to extend B cell targeting into tissues, as we look to address a range of autoimmune diseases in the clinic."

A summary of the results is reported below:

ADI-001 demonstrated significant levels of CAR T cell activation and tissue exposure in lymph node biopsies in the GLEAN trial, with a mean exposure of 236,701 CAR T cells per million across all dose levels, representing a range of 27-64% of total cellular material detected by ddPCR in evaluable biopsies at the 1E9 dose, and exceeding levels previously reported for patients who received autologous alpha-beta CAR T therapies. CAR T cells detected in tissues also demonstrated a robust activation profile, based on in situ detection of granzyme B.
Recently published studies have demonstrated depletion of CD19+ plasmablasts, memory B cells and naïve B cells in peripheral blood using anti-CD20 targeted antibodies, however, these CD20-targeted antibody modalities failed to deplete B cells within secondary lymphoid tissues.
Concurrent with ADI-001 tissue trafficking and activation, complete depletion of CD19+ B cells within analyzed secondary lymphoid tissue was also observed. These results support ADI-001’s potential for achieving complete B-cell depletion in peripheral blood and within tissues.
Adicet is advancing the ADI-001 clinical program in lupus nephritis, systemic lupus erythematosus, systemic sclerosis and anti-neutrophil cytoplasmic autoantibody associated vasculitis (AAV) and expects to report initial clinical data in the first half of 2025.

Company webcast information

A listen-only webcast with an accompanying presentation by Dr. Aftab is accessible under Presentations & Events | Adicet Bio in the Investors section of Adicet Bio’s website. The archived webcast will be available for 30 days.

About the GLEAN trial

The Phase 1 GLEAN study was 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.

On September 19, 2024 researchers from City of Hope, one of the largest and most advanced cancer research and treatment organizations in the U.S., and The University of Texas M.D. Anderson Cancer Center, reported safety and efficacy results from a Phase 1 trial that featured a personalized vaccine to treat lymphoplasmacytic lymphoma, a rare and slow-growing type of blood cancer, according to a study published recently in Nature Communications (Press release, City of Hope, SEP 19, 2024, View Source [SID1234646756]).

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 current approach to lymphoplasmacytic lymphoma care is active surveillance of a patient’s possible symptoms. There is a median time of 3.5 years from diagnosis to progression of symptoms — such as fever, night sweats, weight loss and fatigue — that require chemotherapy.

"By doing an early intervention with the vaccine, we nearly doubled the disease-free progression time to an average of just under seven years," said Larry Kwak, M.D., Ph.D., director of City of Hope’s Toni Stephenson Lymphoma Center within the Hematologic Malignancies Research Institute, who developed the vaccine and is the study’s corresponding author. "In addition to being effective, the vaccine appears to be safe. It didn’t have any of the harsh side effects associated with other types of common cancer treatments."

Toxicity among trial participants was also limited, Dr. Kwak said. That’s because the vaccine uses patient-specific biologic components called tumor neoantigens that can help the body mount an immune response to a particular tumor type.

The clinical trial, led by M.D. Anderson, enrolled nine patients, who were able to tolerate the therapy without negative side effects. After a median follow up of 7.5 years, all of the patients had stable disease and more than half had not progressed to a symptomatic state.

"Using sophisticated technology called single-cell sequencing, we could see that these personalized vaccines activated T cells in the tumor microenvironment, which help destroy tumor cells," said Dr. Kwak, who is also the deputy director of City of Hope’s comprehensive cancer center and the Dr. Michael Friedman Professor in Translational Medicine. "Furthermore, we saw that the tumor cells rely on signaling from myeloid cells to survive, which we didn’t know before, and the vaccine reduced that protumoral signaling, too."

According to Dr. Kwak, disease progression was halted and one patient had a minor reduction in tumor shrinkage. The research teams believe this is because two different subtypes of cells give rise to tumors in lymphoplasmacytic lymphoma — mature B cells and plasma cells — and the vaccine only had an effect on the B cell population.

"The takeaway from that observation is that when we do the next phase clinical trial, we will want to combine the vaccine with another agent that does have more of a direct effect on plasma cells, like a monoclonal antibody," he said.

Dr. Kwak and the research team are working on the next generation of the vaccine for a possible clinical trial that may take place at City of Hope. As part of a Sponsored Research Agreement with Renhaim Inc., Dr. Kwak and team plan to adapt the vaccine to an mRNA platform, which has become a new frontier for vaccine production since the original was developed using DNA nearly a decade ago. The team is also exploring additional therapies to pair with the neoantigen for more vaccine efficacy. Dr. Kwak is a paid consultant with Renhaim Inc.

The neoantigen vaccine research builds upon three decades of investigations by Dr. Kwak, who is a world-renowned physician and pioneering scientist in immunology and cancer vaccines. Dr. Kwak was named one of TIME magazine’s "100 Most Influential People" in 2010 for his work in cancer immunology.

In 2011, Dr. Kwak and colleagues published research in the Journal of Clinical Oncology on the first iteration of the neoantigen vaccine. It was a protein-based vaccine for follicular lymphoma that garnered positive results.

"It was actually one of the first positive cancer vaccine positive trials in the field, but 15 years ago, no drug company was interested in talking with us about a personalized vaccine," Dr. Kwak said. He noted that the "one drug for one patient" model was a tough sell until CAR T cell therapies set a precedent for individualized cancer medicines. "Now they’re much more open to it. I think the future of cancer vaccines is really in this kind of setting, where we’ve shown the effectiveness of early intervention as a way to prolong and maybe even prevent progression to symptomatic disease."

The research reported in the Nature Communications paper, "Personalized neoantigen vaccines as early intervention in untreated patients with lymphoplasmacytic lymphoma: a non-randomized phase 1 trial," was supported by the National Cancer Institute and the International Waldenstrom’s Macroglobulinemia Foundation.