On September 19, 2024 Kiromic BioPharma, Inc. (OTCQB: KRBP) ("Kiromic" or the "Company") reported that the University of Arizona Cancer Center (UACC) has been activated as the fifth clinical trial site in the Deltacel-01 Phase 1 trial evaluating Deltacel (KB-GDT-01), Kiromic’s allogeneic, off-the-shelf, Gamma Delta T-cell (GDT) therapy, in patients with stage 4 metastatic non-small cell lung cancer (NSCLC) who have failed to respond to standard therapies (Press release, Kiromic, SEP 19, 2024, View Source [SID1234646744]).

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The UACC is one of the 57 NCI-Designated Comprehensive Cancer Centers in the U.S. Dr. Ricklie Ann Julian, Assistant Professor of Medicine at UACC, will serve as Principal Investigator at the site.

"We are delighted to partner with UACC, expanding our Deltacel-01 clinical trial to a fifth site. We believe we are well-positioned to enroll the next cohort of patients, and we are optimistic that we will continue to register encouraging results in the expansion phase of the study. The UACC has earned a reputation as a premier research institution actively engaged in clinical trials and delivering the highest-quality care to those with cancer. We look forward to working with their team," stated Pietro Bersani, CEO of Kiromic BioPharma.

The site initiation visit at UACC has been completed and patient enrollment is expected to begin in the coming weeks.

On September 19, 2024 Kiromic BioPharma, Inc. (OTCQB: KRBP) ("Kiromic" or the "Company") reported that the Deltacel-01 Safety Monitoring Committee (SMC) has unanimously voted in favor of proceeding with the expansion phase of the Deltacel-01 clinical trial (Press release, Kiromic, SEP 19, 2024, View Source [SID1234646745]). This trial is evaluating Deltacel (KB-GDT-01), the Company’s allogeneic, off-the-shelf, Gamma Delta T-cell (GDT) therapy, in patients with stage 4 metastatic non-small cell lung cancer (NSCLC) who have failed to respond to standard therapies.

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This recommendation by the SMC follows a favorable review of safety data from the first two cohorts, including recent results from the 40-day follow-up visit of the sixth patient in Deltacel-01, which demonstrated a positive safety and tolerability profile with no dose-limiting toxicities (DLTs) observed.

As part of the expansion phase of Deltacel-01, Kiromic will enroll approximately nine patients. Screening for new participants is expected to commence later this month. The expansion phase, or Part 2 of Deltacel-01, will further assess the effectiveness of Deltacel treatment.

"We are delighted to receive unanimous SMC approval to move forward with the expansion phase of the Deltacel-01 trial," said Pietro Bersani, Chief Executive Officer of Kiromic BioPharma. "As we enter this next phase with more activated clinical sites, we expect a solid cadence of patient enrollment. We are optimistic about the potential to further evaluate Deltacel’s impact on patient outcomes and address critical unmet needs in solid tumors."

About Deltacel-01

In Kiromic’s open-label Phase 1 clinical trial, titled "Phase 1 Trial Evaluating the Safety and Tolerability of Gamma Delta T Cell Infusions in Combination With Low Dose Radiotherapy in Subjects With Stage 4 Metastatic Non-Small Cell Lung Cancer" (NCT06069570), patients with stage 4 NSCLC will receive two intravenous infusions of Deltacel with four courses of low-dose, localized radiation over a 10-day period. The primary objective of the Deltacel-01 trial is to evaluate safety, while secondary measurements include objective response, progression-free survival, overall survival, time to progression, time to treatment response and disease control rates.

About Deltacel

Deltacel (KB-GDT-01) is an investigational gamma delta T-cell (GDT) therapy currently in the Deltacel-01 Phase 1 trial for the treatment of stage 4 metastatic NSCLC. An allogeneic product consisting of unmodified, donor-derived gamma delta T cells, Deltacel is the leading candidate in Kiromic’s GDT platform. Deltacel is designed to exploit the natural potency of GDT cells to target solid cancers, with an initial clinical focus on NSCLC, which represents about 80% to 85% of all lung cancer cases. Data from two preclinical studies demonstrated Deltacel’s favorable safety and efficacy profile when it was combined with low-dose radiation.

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.

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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 MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, reported its half year financial results for the six-month period ended June 30, 2024, and provided a business overview (Press release, MaaT Pharma, SEP 19, 2024, View Source [SID1234646746]).

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"Building on the positive data for MaaT013 in April 2024 presented at the EBMT Congress, and the success of our recent fundraising, we have dedicated the first half of 2024 to pursuing recruitment and preparing for the topline results of our ongoing Phase 3 clinical trial for MaaT013. This trial is designed to address the urgent unmet medical need of patients with acute graft-versus-host disease not responding to current treatments. Currently, patients requiring third-line treatment options face an 85% mortality rate within one year. Furthermore, by extending our cash runway by an additional quarter, we are well-positioned to deliver on our short-term milestones," stated Siân Crouzet, Chief Financial Officer of MaaT Pharma.

Pipeline Highlights

MET-N platform

MaaT013

In hemato-oncology:
In March 2024, the Company announced the launch of a retrospective multicenter trial called CHRONOS in Europe. Its primary objective is to provide the Company efficacy data for 3rd-line therapies for aGvHD patients not receiving MaaT013 or any microbiome intervention. This retrospective study does not impact cash projections as funding is already secured.
In April 2024, at the 50th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT), the Company presented promising extended survival data from the Early Access Program in Europe, involving 140 patients with steroid-refractory (SR) or steroid-dependent (SD) acute graft-versus-host disease with gastrointestinal involvement (GI-aGvHD) treated with MaaT013. Data highlighted a high response rate (Complete Response and Very Good Partial Response) to MaaT013, demonstrating a clear reduction in disease burden and improved Overall Survival (OS) at 18 months compared to published data.
In immuno-oncology:
In March 2024, the Company informed on the completion of patient recruitment for the Phase 2a randomized clinical trial (NCT04988841) (PICASSO) sponsored by AP-HP and in collaboration with INRAE and Institut Gustave Roussy, evaluating MaaT013 in combination with immune checkpoint inhibitors (ICI), ipilimumab (Yervoy) and nivolumab (Opdivo), in metastatic melanoma patients. A total of 70 patients have been enrolled since April 2022. The Company provided its MaaT013 drug candidate and placebo and will contribute to the microbiome profiling of patients using its proprietary gutPrint AI research engine. As previously announced, data readout is expected in Q4 2024/Q1 2025.
MaaT033

In hemato-oncology:
In May 2024, the Company announced its participation in the IMMUNOLIFE RHU program, a consortium including academic partners, such as Institut Gustave Roussy (IGR), a world-renowned center in the field of cancer treatment, and biotech companies. MaaT033, an oral, pooled fecal microbiotherapy, developed by MaaT Pharma will be tested as a concomitant treatment to cemiplimab (Regeneron), an anti-PD1 therapy, to assess the potential increase in response rate in patients having received antibiotics. This randomized multicenter Phase 2 clinical trial will include advanced non-small cell lung cancer (NSCLC) patients. The related costs for MaaT Pharma are limited to clinical product supply in line with previous cash projections. The trial is expected to start in H1 2025.
As a post-period event, in July 2024, the Company announced first DSMB positive review of the Phase 2b trial PHOEBUS and recommended continuation of the trial without modification. The DSMB concluded that the safety profile was acceptable and the treatment well-tolerated. The trial is an international, multi-center, randomized, double-blind, testing MaaT033, in patient receiving HSCT, an oral freeze-dried formulation against placebo, set to be conducted in up to 56 clinical investigation sites and is expected to enroll 387 patients (NCT05762211).
Recruitment for PHOEBUS trial is ongoing in France, Germany, Spain, and Belgium, with the trial already approved in the Netherlands and the United Kingdom. Upcoming milestones include a second safety assessment by the DSMB expected in early Q1 2025. The interim analysis following the recruitment of 60 patients, is expected in H1 2025, instead of H2 2024. This slight delay is due to the strategic option taken by the Company’s management in early 2024 to prioritize resources for the Phase 3 ARES trial and open new trial sites in countries outside France and Germany in a more sedate manner than originally planned.
In neurodegenerative diseases:
In February 2024, the Company announced a positive review by the DSMB on the Phase 1 clinical trial (IASO) evaluating MaaT033 in Amyotrophic Lateral Sclerosis (ALS) for the first 8 patients. The DSMB recommended that the trial proceed without modification.
In May 2024, the Company announced the completion of patient recruitment for IASO.

MET-C platform

MaaT034

In combination with immune checkpoint inhibitors in solid tumors
In April 2024, the Company presentednew in vitro data characterizing the metabolites produced by MaaT034 and their impact on immune modulation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2024 in San Diego, California. MaaT034, the first product from MaaT Pharma’s MET-C platform, is a ground-breaking full ecosystem synthetic microbiota product being developed for patients with solid tumors to improve responses to immunotherapy in combination with an ICI treatment, which represents a potentially large market. In today’s challenging economic environment, the Company has prioritized resources to focus on MaaT013, specifically preparing marketing authorization activities in Europe and the upcoming Phase 3 topline results in Europe. This approach aimed at optimizing both short-term validation and clinical validation, has resulted in a deferral of activities related to MaaT034. Thus, clinical activities for MaaT034 are now expected to begin in 2026 and not 2025, as previously announced.

Corporate updates

In March 2024, the Company announced the appointment of Jonathan Chriqui, PharmD, as Chief Business Officer and member of the executive management team. Jonathan Chriqui will be responsible for MaaT Pharma’s business development and partnering strategies.
In May 2024, the Company announced the successful completion of its offering of 18.2 million euros. The net proceeds from the Primary Offering were €17.3 million.
In May 2024, the Company announced the production of MaaT013 batches for clinical supply in the US, while advancing the readiness phase to initiate clinical activities. This includes ongoing discussions with prominent US clinicians in stem cell transplantation.
In June 2024, the Company announced the appointment of Gianfranco Pittari, M.D., Ph.D., as Chief Medical Officer. Dr. Pittari brings over 15 years of international experience in clinical research and drug development in hematology, oncology, and immunology. The Company also welcomed Carole Ifi, as head of Regulatory Affairs. Former Senior director, global regulatory lead at UCB, Carole Ifi brings over 25 years of expertise in regulatory affairs and will be instrumental in leading MaaT013 submission.

On September 18, 2024 GlyTherix Ltd (GlyTherix) an Australian targeted radiotherapy company specializing in developing antibody radiopharmaceuticals for solid tumors reported a new global clinical supply agreement with Wisconsin-based SHINE Technologies, a pioneer in next-generation fusion-based technology and North America’s largest producer of non-carrier added lutetium-177 (n.c.a. Lu-177) chloride (Press release, Glytherix, SEP 18, 2024, View Source [SID1234646729]). SHINE will supply its n.c.a. Lu-177 chloride, Ilumira, for use in GlyTherix’s clinical trials focused on innovative treatments for aggressive and invasive cancers.

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As GlyTherix advances its clinical trials using the medical radioisotope Lu-177, it is building a global supplier network with proximity to major global markets, capable of consistently delivering high-quality, n.c.a. Lu-177 to patients. SHINE will supply n.c.a. Lu-177 for use in GlyTherix’s clinical trials with a particular focus on the expansive U.S. market.

GlyTherix’s radiotherapy combines Lu-177 with a molecule targeting Glypican-1, a protein in aggressive cancers, to deliver localized radiation while sparing healthy tissue. Their 177Lu-DOTA-Miltuximab will enter Phase Ib trials in early 2025.

GlyTherix will use 177Lu-DOTA-Miltuximab targeting tumors expressing Glypican-1 in its planned Australian Phase Ib therapeutic dose escalation trial scheduled to commence early 2025. Glypican-1 is an attractive tumor target that occurs in several aggressive and invasive cancers including prostate, pancreatic, bladder, lung, glioblastoma and ovarian cancer.

Dr. Brad Walsh, GlyTherix’s Chief Executive Officer said, "SHINE’s investment in high-quality isotope production places them at the forefront of the radiopharmaceutical supply chain with particular strength in servicing the U.S. market. This supply agreement for the medical radioisotope Lu-177 adds to GlyTherix’s global clinical supplier network, which also includes a clinical supply agreement with the Australian Nuclear Science and Technology Organization (ANSTO)".

Greg Piefer, SHINE founder and CEO added, "Our partnership with GlyTherix, a true pioneer in targeted radiotherapy, represents an important step in advancing next-generation cancer treatments. GlyTherix’s innovative approach has the potential to transform cancer care for patients with some of the most challenging solid tumors. By providing a reliable supply of high-quality Ilumira, we’re proud to support their groundbreaking work that could offer new hope to patients with limited treatment options."

SHINE’s Ilumira is produced in the company’s Cassiopeia facility in Janesville, Wisconsin – the largest of its kind in North America. With an initial production capacity of 100,000 doses per year and the potential for expansion to 200,000 doses annually, SHINE is well-positioned to meet the growing demand for Lu-177 in cancer therapies.

This partnership with GlyTherix marks another significant milestone in SHINE’s mission to harness nuclear technology for human health. By providing a reliable, high-quality supply of Ilumira for innovative clinical trials, SHINE is playing a crucial role in advancing targeted radiotherapy. As SHINE continues to expand its production capabilities and pursue vertical integration in Lu-177 supply, collaborations like this have the potential to transform cancer treatment worldwide, offering new hope to patients facing aggressive and hard-to-treat cancers.