On February 21, 2024 Kazia Therapeutics Limited (NASDAQ: KZIA), a biotechnology company specializing in oncology, reported the early conclusion based on positive safety and promising clinical response findings observed to date of an important two-part Phase I trial (Press release, Kazia Therapeutics, FEB 21, 2024, View Source [SID1234640332]). This investigator-initiated trial evaluated the use of paxalisib (an oral PI3K/mTOR dual inhibitor) with radiation therapy for the treatment of patients with PI3K pathway mutation brain metastases from solid tumors.

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Part I of the study established the maximal tolerated dose (MTD) of paxalisib in combination with radiation therapy, while also demonstrating promising signs of clinical activity in all nine evaluable patients. Part II was a follow-on expansion cohort to further evaluate safety and efficacy of the MTD (45mg daily) combined with radiation therapy in up to 12 additional patients.

After reviewing the Part II patient data generated to date, the three lead investigators have determined that the primary endpoint of the study has been reached. In addition, the investigators continued to observe encouraging signs of clinical response in patients in the expansion cohort. Detailed findings from Part II of this study are slated for submission and presentation at a forthcoming global scientific meeting, where they will contribute to the ongoing conversation about treatment options for patients with these complex brain metastases.

Kazia’s CEO, Dr. John Friend, shared his enthusiasm: "We are extremely excited about these findings, which include not only encouraging safety data but also some promising efficacy signals for paxalisib in combination with radiation therapy. We are now preparing to engage with the Food and Drug Administration to discuss the data and seek guidance on the conduct of a pivotal registration study, with the goal of rapidly progressing paxalisib’s development to potentially provide a more effective treatment option for patients with brain metastases."

Previous research by Dr. Jonathan Yang, Director of Metastatic Disease and Developmental Therapeutics, Department of Radiation Oncology, University of Washington School of Medicine, and others, has shown that activation of the PI3K pathway is common in brain metastases. Moreover, PI3K pathway activation appears to result in tumor resistance to radiotherapy, which supports the rationale for evaluating paxalisib with radiotherapy in order to potentially sensitize the tumor cells to radiotherapy and achieve better disease control. Dr. Yang presented the Part I data at the 2022 Annual Conference on CNS Clinical Trials and Brain Metastases, jointly organized by the Society for Neuro-Oncology and the American Society for Clinical Oncology, held in Toronto, Canada.

Last year, paxalisib was awarded Fast Track Designation (FTD) on the basis of the Part I clinical data by the United States Food and Drug Administration (FDA) for the treatment of solid tumor brain metastases harboring PI3K pathway mutations in combination with radiation therapy.

Approximately 200,000 cancer patients develop brain metastases in the United States each year. Radiotherapy is the mainstay of treatment for brain metastases, and generally consists of either stereotactic radiosurgery (SRS) or whole brain radiotherapy (WBRT) or some combination thereof. The efficacy in patients who receive WBRT differs according to the type of tumor and the number and volume of brain metastases, but several recent publications cite overall response rates of 20-45%. The increasing incidence of brain metastasis and the low response rates to existing treatments underscores the need for new treatment options.

On February 21, 2024 Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) ("Innate" or the "Company") reported that members of its senior management team are scheduled to participate in the upcoming investor conference, detailed below (Press release, Innate Pharma, FEB 21, 2024, View Source [SID1234640330]).

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• Leerink Partners Global Biopharma Conference 2024
Event Date: March 11-13, 2024, Miami, Florida

On February 21, 2024 ImmunityBio (NASDAQ: IBRX), a clinical-stage immunotherapy company, reported that enrollment and initial follow-up has been completed for the safety portions of a clinical trial that is studying ImmunityBio’s investigational cancer vaccine of a tri-valent combination of antigens delivered by a second-generation Adenovirus vector (Tri-Ad5 CEA/MUC1/brachyury) together with its IL-15 superagonist N-803 for participants with Lynch syndrome (Press release, ImmunityBio, FEB 21, 2024, View Source [SID1234640328]). The study, sponsored by the National Cancer Institute, part of the National Institutes of Health, will include up to 186 participants when fully enrolled and is now open to the randomized controlled portion of the trial.

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Each of the three vaccines in Tri-Ad5 targets different proteins associated with precancer and cancer cells. The vaccine combination is studying whether activation of dendritic cells and training the immune system to recognize those proteins will destroy the precancer cells before the cancer occurs. The IL-15 superagonist N-803 is designed to enhance the effects of the vaccines by increasing proliferation and activation of natural killer (NK) and T cells, thereby increasing the potential for cancer prevention in study participants.

"We are pleased to be selected to participate in this important and innovative cancer prevention study, one that could provide insights into how the immune system could be harnessed to prevent cancer in individuals with hereditary risk," said Patrick Soon-Shiong, M.D., Executive Chairman and Global Chief Scientific and Medical Officer at ImmunityBio. "With an estimated 5 to 10 percent of cancers inherited, understanding mechanisms that might prevent or delay their onset could potentially change the prospects for tens of thousands of people annually."

Lynch syndrome (also called hereditary non-polyposis colorectal cancer or HNPCC) is one of the most common hereditary cancer syndromes occurring in 1 in every 300 Americans.2 Not only can people with Lynch syndrome develop colorectal cancer 20 years before the average age of diagnosis for this cancer, they are also at an increased risk of developing multiple types of other cancers, including endometrial, stomach, ovarian, pancreas, ureter and renal pelvis, biliary tract, brain, and small intestinal cancers. Colorectal cancer is the second-deadliest cancer type in the U.S., and approximately 3% to 5% of the 153,000 cases of colorectal cancer annually are thought to be due to Lynch Syndrome, as are 2% to 3% of all cases of endometrial cancer.3

"We are encouraged by how rapidly this study has been able to enroll participants," said Asad Umar, D.V.M., Ph.D., a senior advisor to the Director for Translational Research in NCI’s Division of Cancer Prevention (DCP) and a scientific lead for the trial. "It is a strong indication of an unmet need and of the willingness of participants to help science make new discoveries in the area of cancer prevention."

To learn more about this study, please visit View Source

For patients interested in enrolling in this study, please contact NCI’s toll-free number 1-800-4-Cancer (1-800-422-6237) (TTY: 1-800-332-8615) and/or the website: View Source and/or [email protected].

ImmunityBio’s Tri-Ad5 Vaccines and N-803 are investigational. Safety and efficacy of these investigational agents have not been established by any Health Authority, including the FDA.

About ImmunityBio’s Tri-Ad5 Vaccines

ImmunityBio’s Tri-Ad5 vaccines target three tumor-associated antigens: brachyury, carcinoembryonic antigen (CEA), and mucin-1 (MUC1). Pre-clinical studies have demonstrated Tri-Ad5 vaccines elicit cytotoxic T cell-mediated tumor cell death and the establishment of memory T cells, and thus may provide protection against the growth and metastasis of cancer. Tri-Ad5 vaccines utilize a second-generation replication-defective human adenovirus serotype 5 (Ad5) vector with viral genes deleted to allow for production of the antigen and a vigorous immune response, without generating a host response to the vector and with the ability to overcome previous adenovirus immunity in cancer patients. Notably, in a phase 1 NCI trial, Tri-Ad5 generated antigen-specific T cells to MUC1, CEA, and/or brachyury in all 10 patients with no evidence of antigenic competition. The safety of multiple ImmunityBio product candidates utilizing the Ad5 technology has been demonstrated in phase 1 and 2 clinical trials for cancers across several tumor types.

On February 21, 2024 Immix Biopharma, Inc. (Nasdaq: IMMX), a clinical-stage biopharmaceutical company trailblazing cell therapies in autoimmune disease, reported its 12-month progress update including shareholder letter (Press release, Immix Biopharma, FEB 21, 2024, View Source [SID1234640326]).

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"The past 12 months have seen landmark achievements for Immix Biopharma, cementing our position as a leading cell therapy company in autoimmune disease. We successfully received U.S. FDA investigational new drug clearance for NXC-201. NXC-201 is the only CAR-T in AL Amyloidosis and is expanding into additional autoimmune indications, a $25 billion combined annual market segment, where limited treatments are available today. At the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 2023, we presented clinical data from additional NXC-201 patients enrolled in our ongoing NEXICART-1 clinical trial, totaling 73 NXC-201 patients dosed to-date, demonstrating 100% and 95% overall response rates in relapsed/refractory AL Amyloidosis patients and relapsed/refractory multiple myeloma patients, respectively. We received U.S. FDA Orphan Drug Designation in both AL Amyloidosis and multiple myeloma, as well as EU Orphan Drug Designation for AL Amyloidosis. NXC-201 is the first and only ‘Single-Day CRS’ CAR-T, a critical advancement for autoimmune diseases like AL Amyloidosis," said Ilya Rachman, MD PhD CEO Immix Biopharma. Gabriel Morris, CFO Immix Biopharma, added, "In the next 12 months, we plan to continue our approximately quarterly clinical data updates, presenting at premier academic forums, finalize the selection of our next autoimmune indication, and to dose U.S. patients with NXC-201. We are in an exciting position as the premier autoimmune CAR-T company with a robust clinical dataset. Top-tier U.S. clinical sites are currently being activated for NXC-201 dosing."

In the last 12 months, Immix Biopharma achieved:

Clinical

Dosed additional NXC-201 patients: totaling 10 relapsed/refractory AL Amyloidosis patients and 63 relapsed/refractory multiple myeloma patients, presented at the 65th annual ASH (Free ASH Whitepaper) conference in San Diego, CA
Regulatory

Received U.S. Food and Drug Administration (FDA) clearance of Investigational New Drug (IND) for NXC-201 treatment of U.S. patients
Received FDA Orphan Drug Designation (ODD) for the treatment of AL Amyloidosis
Received FDA Orphan Drug Designation (ODD) for the treatment of multiple myeloma
Received EU Orphan Drug Designation (ODD) for the treatment of AL Amyloidosis
Expanded Scientific Advisory Board Membership

Heather Landau, MD – Memorial Sloan Kettering Amyloidosis Program Director
Michaela Liedtke, MD – Stanford Medicine Cancer Center Hematology Program Lead and Co-Director Stanford Amyloid Center
Suzanne Lentzsch, MD – Director of the Multiple Myeloma and Amyloidosis Program at the College of Physicians and Surgeons of Columbia University and at New York Presbyterian Hospital
Marko Radic, PhD – Autoimmune CAR-T Pioneer and Associate Professor at the University of Tennessee Health Science Center
Vaishali Sanchorawala, MD – Skinner Professor of Amyloidosis Research in the Department of Medicine at Boston University Chobanian & Avedisian School of Medicine, and Director of the Amyloidosis Center at Boston Medical Center (BMC) and Boston University
Expanded Team

Dr. Gerhard Bauer, head of cell therapy manufacturing, with more than two decades of experience in design, manufacturing, and operation of GMP cell therapy manufacturing facilities and numerous investigational new drug (IND) applications to FDA
David Marks, MBBS, PhD, chief medical officer, cell therapy, who was appointed by regulators as a clinical expert in 2 CAR-T regulatory approvals: KYMRIAH and TECARTUS
Henry A. McKinnell, former Pfizer CEO; Mary Sue Coleman, former Johnson & Johnson Director; Jeffrey Cooper, former BioMarin CFO; Edward Borkowski, former Mylan CFO, who along with existing director Helen Adams have participated in close to $20 billion in U.S. pharmaceutical mergers & acquisitions
Additional team members across business development, regulatory, clinical and research & development staff
NXC-201 AL Amyloidosis Publications and Presentations

ASH – 65th Annual American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (AL Amyloidosis) – Dec 10, 2023 Oral Presentation
ASH – 65th Annual American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition – (AL Amyloidosis) – 2023 Abstract
IMS – International Myeloma Society – 20th Annual Meeting – Oral Presentation (AL Amyloidosis) – Sep 27-30, 2023 Oral Presentation
ASGCT – American Society of Gene & Cell Therapy – 29th Annual Meeting – May 19, 2023 Oral Presentation (Late-Breaking)
ASGCT – American Society of Gene & Cell Therapy – 29th Annual Meeting – May 19, 2023 Late-Breaking Abstract
NXC-201 Multiple Myeloma Publications and Presentations

ASH – 65th Annual American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition – (Multiple Myeloma) – Dec 11, 2023 Poster Presentation
ASH – 65th Annual American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition – (Multiple Myeloma) – 2023 Abstract
IMS – International Myeloma Society – 20th Annual Meeting – (Multiple Myeloma) – Sep 27-30, 2023 Poster Presentation
European Society for Blood and Marrow Transplantation 49th Annual Meeting – 58 Patient Clinical Data Poster Presentation – Apr 23-26, 2023 Poster Presentation
5th European CAR T-cell Meeting – 42 Patient Clinical Data Poster Presentation – Feb 9-11, 2023 Poster Presentation
Publication: Asherie N, Kfir-Erenfeld S, Avni B, Assayag M, Dubnikov T, Zalcman N, Lebel E, Zimran E, Shaulov A, Pick M, Cohen Y, Avivi I, Cohen C, Gatt ME, Grisariu S, Stepensky P. Development and manufacture of novel locally produced anti-BCMA CAR T cells for the treatment of relapsed/refractory multiple myeloma: results from a phase I clinical trial. Haematologica. 2023 Jul 1;108(7):1827-1839. doi: 10.3324/haematol.2022.281628. PMID: 36200421; PMCID: PMC10316256.
Editorial Publication: Sjöstrand M, Sadelain M. Driving CARs to new places: locally produced BCMA CAR T cells to treat multiple myeloma. Haematologica. 2023 Jul 1;108(7):1721-1723. doi: 10.3324/haematol.2022.282053. PMID: 36794501; PMCID: PMC10316265.
The above publications and presentations are available on the "publications" section of the ImmixBio website.

Investor Events

Presented at the Bank of America Healthcare Trailblazers Conference
Presented at the JMP Securities Hematology and Oncology Summit
Hosted a Key Opinion Leader (KOL) event discussing NXC-201 for the treatment of relapsed/refractory AL Amyloidosis (November 29 2023)

On February 21, 2024 Halia Therapeutics, a clinical-stage biopharmaceutical company pioneering a novel class of small molecule medications designed to combat inflammation, reported that David J. Bearss, Ph.D., President and CEO of Halia Therapeutics, will present at the BIO CEO & Investor Conference on Tuesday, February 27 (Press release, Halia Therapeutics, FEB 21, 2024, View Source [SID1234640325]).

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Dr. Bearss’ presentation will highlight the recent company developments on its novel pipeline of therapeutics designed to improve patients’ lives with chronic inflammatory disorders and neurodegenerative diseases, including the initiation of 2 Phase II clinical trials evaluating its lead asset, HT-6184, a selective and orally bioavailable first-in-class inhibitor of NLRP3/NEK7 inflammasome. The presentation will include recent business updates and anticipated milestones.

Details about the presentation are as follows:

Presenter: David J. Bearss, Ph.D., President and CEO of Halia Therapeutics
Date: Tuesday, February 27, 2024, from 9:30 a.m. to 9:45 a.m. EST
Location: Plymouth, 6th floor, Marriott Marquis, New York, NY
Registration: Here

Dr. Bearss and Jeff Burton, CFO, will be available for one-on-one investor meetings with registered conference attendees. Meetings can be scheduled via the BIO One-on-One Partnering system: View Source