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

On February 21, 2024 Gritstone bio, Inc. (Nasdaq: GRTS), a clinical-stage biotechnology company working to develop the world’s most potent vaccines, reported that management will be participating in the following investor and scientific conferences (Press release, Gritstone Bio, FEB 21, 2024, View Source [SID1234640324]):

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2024 BIO CEO & Investor Conference (Panel)
Panel Title: Reviewing Vaccine Pipeline Breakthroughs and Barriers
Date and Time: Monday, February 26, 2024 at 2:00pm ET
Location: New York, NY

IO360 Summit (Panel)
Panel Title: How to use AI to Leverage Data from Patients in the Clinic to Iterate Next Gen Immunotherapies
Date and Time: Tuesday, February 27, 2024 at 2:30pm ET
Location: New York, NY

44th Annual TD Cowen Healthcare Conference (Panel)
Panel Title: Novel I-O Corporate Panel
Date and Time: Monday, March 4, 2024 at 10:30am ET
Location: Boston, MA

World Vaccine Congress 2024 (Panel and Presentation)
Panel Title: Exploring the Future Prospects of Cancer Vaccines
Panel Date and Time: Monday, April 3, 2024 at 9:10am ET
Location: Washington, DC

Relevant events will be webcast live and available via View Source Archived replays will be accessible for 30 days following each event.

On February 21, 2024 Exact Sciences Corp. (Nasdaq: EXAS), a leading provider of cancer screening and diagnostic tests, reported that the company generated revenue of $647 million for the fourth quarter of 2023 and $2.50 billion for the full year of 2023, both ended Dec. 31, 2023 (Press release, Exact Sciences, FEB 21, 2024, View Source [SID1234640323]).

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"The Exact Sciences team advanced our mission to help eradicate cancer by testing a record number of patients with Cologuard and Oncotype DX in the fourth quarter, leading to strong financial results," said Kevin Conroy, Chairman and CEO of Exact Sciences. "Over the past decade, we have built a world-class team and unrivaled platform that is fueling predictable, profitable growth and powering the next wave of innovative cancer diagnostics to make a profound difference in the fight against cancer."

Fourth quarter 2023 financial results

For the three-month period ended Dec. 31, 2023, as compared to the same period of 2022 (where applicable):

Total revenue was $646.9 million, an increase of 17 percent, or 18 percent on a core revenue basis
Screening revenue was $486.7 million, an increase of 21 percent
Precision Oncology revenue was $160.2 million, an increase of 12 percent, or 11 percent on a core revenue basis
Gross margin including amortization of acquired intangible assets was 70 percent, and non-GAAP gross margin excluding amortization of acquired intangible assets was 73 percent
Net loss was $49.8 million, or $0.27 per share, compared to a net loss of $127.7 million, or $0.72 per share
EBITDA was $11.7 million and adjusted EBITDA was $49.7 million
Cash provided by operating activities was $69.5 million and free cash flow was $34.6 million
Cash, cash equivalents, and marketable securities were $777.6 million at the end of the quarter
Screening primarily includes laboratory service revenue from Cologuard tests and PreventionGenetics. Precision Oncology includes laboratory service revenue from global Oncotype DX and therapy selection tests.

2024 revenue outlook

The company anticipates revenue of $2.810-$2.850 billion during 2024, assuming:

Screening revenue of $2.155-$2.175 billion, and
Precision Oncology revenue of $655-$675 million
Non-GAAP disclosure

In addition to the company’s financial results determined in accordance with U.S. GAAP, the company provides non-GAAP measures that it determines to be useful in evaluating its operating performance and liquidity. The company presents EBITDA, adjusted EBITDA, non-GAAP gross margin, non-GAAP gross profit, core revenue, and free cash flow. EBITDA and adjusted EBITDA consist of net loss after adjustment for those items shown in the table below. The company defines non-GAAP gross profit and non-GAAP gross margin as GAAP gross profit and GAAP gross margin, respectively, excluding amortization of acquired intangible assets. The amortization of acquisition-related intangible assets used in the calculation of non-GAAP gross profit and non-GAAP gross margin pertain only to the amortization associated with developed technology acquired and recorded through purchase accounting transactions. The amortization of these intangible assets will recur in future periods until such intangible assets have been fully amortized. Core revenue is calculated to adjust for recent acquisitions and divestitures, COVID-19 testing revenue and foreign currency exchange rate fluctuations. To exclude the impact of change in foreign currency exchange rates from the prior period under comparison, the Company converts the current period non-U.S. dollar denominated revenue using the prior year comparative period exchange rates. The company considers free cash flow to be a liquidity measure and is calculated as net cash used in or provided by operating activities, reduced by purchases of property, plant and equipment. Management believes that presentation of non-GAAP financial measures provides useful supplemental information to investors and facilitates the analysis of the company’s core operating results and comparison of operating results across reporting periods. The company uses this non-GAAP financial information to establish budgets, manage the company’s business, and set incentive and compensation arrangements. The company believes free cash flow provides useful information to management and investors since it measures our ability to generate cash from business operations. Non-GAAP financial information, when taken collectively, may be helpful to investors because it provides consistency and comparability with past financial performance. However, non-GAAP financial information is presented for supplemental information purposes only, has limitations as an analytical tool and should not be considered in isolation or as a substitute for financial information presented in accordance with U.S. GAAP. For example, non-GAAP gross margin and non-GAAP gross profit exclude the amortization of acquired intangible assets although such measures include the revenue associated with the acquisitions. Additionally, adjusted EBITDA excludes a number of expense items that are included in net loss. As a result, positive adjusted EBITDA may be achieved while a significant net loss persists. For a reconciliation of these non-GAAP measures to GAAP, see below "EBITDA and Adjusted EBITDA Reconciliations", "Non-GAAP Gross Profit and Non-GAAP Gross Margin Reconciliations", "Reconciliation of Core Revenue", and "Condensed Consolidated Statements of Cash Flows and Reconciliation of Free Cash Flow". The company presents certain forward-looking statements about the company’s future financial performance that include non-GAAP measures. These non-GAAP measures include adjustments like stock-based compensation, acquisition and integration costs including gains and losses on contingent consideration that are difficult to predict for future periods because the nature of the adjustments pertain to events that have not yet occurred. Additionally, management does not forecast many of the excluded items for internal use. Information reconciling forward-looking non-GAAP measures to U.S. GAAP measures is therefore not available without unreasonable effort and is not provided. The occurrence, timing, and amount of any of the items excluded from GAAP to calculate non-GAAP could significantly impact the company’s GAAP results.

Fourth quarter conference call & webcast

Company management will host a conference call and webcast on Wednesday, February 21, 2024, at 5 p.m. ET to discuss fourth quarter and full year 2023 results. The webcast will be available at exactsciences.com. Domestic callers should dial 888-330-2384 and international callers should dial +1-240-789-2701. The access code for both domestic and international callers is 4437608. A replay of the webcast will be available at exactsciences.com. The webcast, conference call, and replay are open to all interested parties.

About Cologuard

The Cologuard test was approved by the FDA in August 2014, and results from Exact Sciences’ prospective 90-site, point-in-time, 10,000-patient pivotal trial were published in the New England Journal of Medicine in March 2014. The Cologuard test is included in the American Cancer Society’s (2018) colorectal cancer screening guidelines and the recommendations of the U.S. Preventive Services Task Force (2021) and National Comprehensive Cancer Network (2016). The Cologuard test is indicated to screen adults 45 years of age and older who are at average risk for colorectal cancer by detecting certain DNA markers and blood in the stool. Do not use the Cologuard test if you have had precancer, have inflammatory bowel disease and certain hereditary syndromes, or have a personal or family history of colorectal cancer. The Cologuard test is not a replacement for colonoscopy in high risk patients. The Cologuard test performance in adults ages 45-49 is estimated based on a large clinical study of patients 50 and older. The Cologuard test performance in repeat testing has not been evaluated.

The Cologuard test result should be interpreted with caution. A positive test result does not confirm the presence of cancer. Patients with a positive test result should be referred for colonoscopy. A negative test result does not confirm the absence of cancer. Patients with a negative test result should discuss with their doctor when they need to be tested again. Medicare and most major insurers cover the Cologuard test. For more information about the Cologuard test, visit cologuardtest.com. Rx only.