On November 3, 2022 MaaT Pharma (EURONEXT: MAAT – the "Company"), a French clinical-stage biotech and a pioneer in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to improving survival outcomes for patients with cancer, reported that extended results from its Early Access Program (EAP) of MaaT013 in patients with GI-aGvHD have been selected for oral presentation at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting held from December 10-13, 2022, in New-Orleans, Louisiana, U.S.A (Press release, MaaT Pharma, NOV 3, 2022, View Source [SID1234622950]). Additionally, detailed results of the Phase 1b trial of MaaT033 in patients with acute myeloid leukemia (AML) were selected for presentation in a poster session. This is the sixth year in a row that the Company’s clinical data are selected for presentation at the ASH (Free ASH Whitepaper) Conference, the world-leading event in malignant and non-malignant hematology, and the third year in a row for an oral presentation.

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In line with the conference embargo policy, MaaT Pharma will detail the presented results through a press release on Saturday, December 10, 2022. The Company will also host an investor webcast on Monday, December 12th, 2022, at 6:00pm CET (additional details will be provided at a later date).

The EAP results include data from 81 patients treated with MaaT013, with steroid-resistant or steroid-dependent aGvHD with GI involvement, who had previously failed 1 to 6 lines (median: 2) of systemic therapy; MaaT Pharma provided the MET product to hospitals under a compassionate access program in France. In parallel, MaaT013 is currently being evaluated in a pivotal open-label, single-arm Phase 3 trial in Europe (n=75) in GI-aGvHD patients refractory to corticosteroids and ruxolitinib; a first data review is expected in the first half of 2023. As of today, MaaT013 has been safely administered to more than 160 patients in Europe in clinical trials and in the Expanded Access Program in France.

Oral Presentation:

Title: Pooled Fecal Allogenic Microbiotherapy for Refractory Gastrointestinal Acute Graft-Versus-Host Disease: Results from the Early Access Program in France
Presenter: Professor Mohamad Mohty, hematology professor and Head of the Hematology and Cellular Therapy Department at the Saint-Antoine Hospital and Sorbonne University
Publication Number: 112
Session: 722. Allogeneic Transplantation: Acute and Chronic GVHD, Immune Reconstitution: Clinical Studies Exploring the Immunobiology of HCT
Session Date/Time: Saturday, December 10, 2022; 10:15am EST
Room: 252-254 (Ernest N. Morial Convention Center)
Poster:

Title: Restoration Of Gut Microbiota Diversity With Oral Pooled Fecal Microbiotherapy In Acute Myeloid Leukemia Patients After Intensive Chemotherapy: The Phase 1b CIMON Trial
Presenter: Professor Mohamad Mohty, hematology professor and Head of the Hematology and Cellular Therapy Department at the Saint-Antoine Hospital and Sorbonne University
Poster number: 2765
Session: 616. Acute Myeloid Leukemias: Investigational Therapies, Excluding Transplantation and Cellular Immunotherapies
Session Date/Time: Sunday, December 11, 2022: 6:00pm -8:00pm EST
Room: Hall D (Ernest N. Morial Convention Center)

Upcoming scientific conferences participations

November 8-10, 2022 – 9th International Human Microbiome Consortium (IHMC) Congress: Poster and oral presentation
November 9-11, 2022 – 21st Société Francophone de Greffe de Moelle et de Thérapie Cellulaire (SFGM-TC) Congress – Booth #10 – poster and oral presentation
December 10-13, 2022 - 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting: Poster and oral presentation
About MaaT013

MaaT013 is a full-ecosystem, off-the-shelf, standardized, pooled-donor, enema Microbiome Ecosystem TherapyTM for acute, hospital use. It is characterized by a consistently high diversity and richness of microbial species and the presence of ButycoreTM (group of bacterial species known to produce anti-inflammatory metabolites). MaaT013 aims to restore the symbiotic relationship between the patient’s functional gut microbiome and their immune system to correct the responsiveness and tolerance of immune functions and thus reduce steroid-resistant, gastrointestinal-predominant aGvHD. MaaT013 has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

About MaaT033

MaaT033 is an oral, full-ecosystem, off-the-shelf, standardized, pooled-donor, high-richness Microbiome Ecosystem TherapyTM. MaaT033 is designed to restore the gut ecosystem to full functionality to improve clinical outcomes as well as to control adverse events related to conventional treatments for liquid tumors. The capsule formulation facilitates administration and allows the potential to treat larger patients’ population while maintaining the high and consistent richness and diversity of microbial species, including anti-inflammatory ButycoreTM species.

On November 3, 2022 Genmab A/S (Nasdaq: GMAB) reported that it is improving its 2022 financial guidance published on August 8, 2022 (Press release, Genmab, NOV 3, 2022, View Source [SID1234622949]). The improved guidance is driven primarily by the positive foreign exchange rate impact on our royalty revenue, and the continued strong performance of DARZALEX net sales.

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Genmab expects its 2022 revenue to be in the range of DKK 13,500 – 14,500 million, an increase to the previous guidance of DKK 12,000 – 13,000 million, driven primarily by the positive foreign exchange rate impact on our royalty revenue, and the continued strong performance of DARZALEX net sales. The upper end of the revenue guidance range now assumes a significant milestone associated with the potential acceptance by the U.S. Food and Drug Administration to review the Biologics License Application submission for epcoritamab. Genmab’s projected revenue for 2022 primarily consists of DARZALEX royalties. Such royalties are based on Genmab’s revised estimate of DARZALEX 2022 net sales of USD 8.0 – 8.2 billion compared to Genmab’s previous estimate of USD 7.8 – 8.2 billion.

Genmab anticipates its 2022 operating expenses to be in the range of DKK 8,000 – 8,400 million, an increase to the previous guidance of DKK 7,600 – 8,200 million, primarily driven by the negative impact of the strong U.S. Dollar. Operating expenses continue to be driven by the advancement of Genmab’s clinical programs, continued investment in research and development, as well as building Genmab’s commercial organization and broader organizational infrastructure.

Genmab now expects its 2022 operating profit to be in the range of DKK 5,100 – 6,500 million, an increase to the previous guidance of DKK 3,800 – 5,400 million, driven primarily by the items described above.

Genmab’s financial results for the first nine months of 2022 will be published on November 9, 2022.

The above expectations are based on assumptions including those described on pages 5 and 6 of the Interim Report for the first half of 2022 (Company Announcement No. 41/2022) as well as an updated USD/DKK exchange rate of 7.2, compared to the previous exchange rate of 6.8.

On November 3, 2022 Genomic Testing Cooperative (GTC) reported With the publication of a paper validating two artificial intelligence-based algorithms for assisting pathologists in molecular profiling of patients, is fully implementing the technology at all of its member laboratories (Press release, Genomic Testing Cooperative, NOV 3, 2022, View Source [SID1234622948]).

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The paper, which appeared earlier this month in the American Journal of Pathology, delved into how the algorithms performed in the differential diagnosis of both hematologic and solid tumors.

The primary algorithm, called RNAnalysis, generates probability scores to help clinicians distinguish between 45 classes of diagnostics. A second algorithm, TraceWork, goes deeper into the data whenever RNAnalysis produces similar high probability scores for more than one diagnostic class.

Corresponding author Maher Albitar, founder, CEO, and chief medical officer of GTC, said that RNAnalysis and TraceWork are "intertwined" for helping make and refine diagnoses and prognoses. Irvine, California-based GTC developed both algorithms.

The research looked at how joining targeted transcriptomes and AI — the latter from the two GTC algorithms — can improve diagnostic accuracy and patient outcomes for patients with hematologic and solid tumors.

The researchers chose targeted transcriptomes over whole transcriptomes "to improve the detection dynamic range of genes that may be expressed at low levels, which may have a significant impact on oncogenesis and cell differentiation," the researchers wrote in the American Journal of Pathology article.

On Oct. 1, GTC announced the general availability of Liquid Trace, a liquid biopsy assay that combines targeted transcriptome and cell-free DNA testing. The cooperative said that the test provides transcriptome data that is enriched by various tumor markers including CA125, CA 15-3, and CEA, making it possible to perform liquid immunoprofiling by evaluating levels of biomarkers including CD19, CD20, CD33, CD4, and CD8.

With the introduction of Liquid Trace, GTC is able to offer comprehensive cell-free RNA analysis alongside DNA analysis. RNAnalysis is compatible with this new assay.

In the paper, an independent blind test found that RNAnalysis settled on the correct top choice for diagnosis in 100 percent of acute lymphoblastic leukemia cases. Accuracy was 88 percent for acute myeloid leukemia and for chronic lymphocytic leukemia, 85 percent for diffuse large B-cell lymphoma, 82 percent for colorectal cancer, and 72 percent for follicular lymphoma, though only 49 percent for lung cancer.

TraceWork was able to distinguish between lung cancer and colorectal cancer with 97 percent sensitivity and nearly 95 percent specificity. It showed similar or better results for distinguishing between Hodgkin lymphoma and normal lymph nodes, between follicular lymphoma and diffuse large B-cell lymphoma, and between breast and ovarian cancer.

The authors concluded that the study demonstrated the potential of combining AI with genomics in routine oncology practice and to diagnosis tumors and their cell origin, providing "solid objective data" for clinical decision making.

This is not true in every diagnostic class, however. For example, the researchers said that they need more cases and additional validation for early-stage myeloid samples including chronic leukemia. Solid tumors that had metastasized to lymph nodes were also problematic.

GTC and its partners expect that they can overcome these issues because the algorithms have been designed to be continuously trained with additional samples and new diagnostic classes.

Initially, the authors evaluated the efficacy of the AI technology in distinguishing between myelodysplastic syndrome and myeloproliferative neoplasms. They later branched out into other types of cancer, first looking at pairs of classes and then at multiple classes.

"Distinguishing between multiple classes was significantly more complex, because the specific biomarkers that are suitable for distinguishing between two diagnostic classes may not be relevant for determining the differences between these two classes and the rest of the diagnostic classes," the authors noted.

They particularly struggled in selecting biomarkers unique to certain classes, but overcame this by producing scores to rank 1,408 biomarkers on how they corresponded to each of 47 diagnostic classes. Sometimes they had to add mutation profiles to distinguish between normal bone marrow, myelodysplastic syndrome, chronic myelomonocytic leukemia, myeloproliferative neoplasms, and acute myeloid leukemia.

Generally, they noted in their paper, misdiagnosed cases were commonly misdiagnosed within the same diagnostic category.

Albitar said that the goal in developing and promulgating the algorithms is to reduce diagnostic error.

He was backed up by another author, André Goy, chief physician officer at John Theurer Cancer Center and academic chairman of oncology at Hackensack Meridian School of Medicine in Hackensack, New Jersey. Theurer Cancer Center hosts a GTC reference lab as part of a joint venture with Hackensack Meridian Health.

Hackensack Meridian School of Medicine and GTC established an oncology-focused DNA and RNA sequencing laboratory called Anthology Diagnostics in early 2021. The collaboration’s aim is to make next-generation sequencing an element of everyday patient care not only within Hackensack’s New Jersey-based network of 17 hospitals and 500 care sites but also along the entire East Coast.

Anthology Diagnostics builds upon a similar collaboration forged in May 2020 between GTC and the Theurer Cancer Center to offer NGS to patients treated within the Regional Cancer Care Associates network of physicians across New Jersey, Connecticut, and Maryland.

With the GTC algorithms, Hackensack Meridian is trying to use targeted transcriptomes to diagnose cancer patients.

Goy said that DNA and RNA profiling is more accurate as well as safer for patients and less expensive for the healthcare industry than a biopsy of a lymph node. "If we had a genome profile, we could actually tell someone this is a reactive [lymph node rather than] cancer," he said.

"I would argue that the future in oncology is much more diagnostic than therapeutic because we already have tons of treatment and we don’t necessarily know how to use them well," Goy added. "This is a win-win situation for society, for the patient more importantly, and for the [healthcare] system because we refocus resources where it matters," Goy said.

Classification based on molecular profiling also provides better information on morphology.

As an example, Goy mentioned mantle cell lymphoma, a rare form of lymphoma that Theurer Cancer Center specializes in.

He said the median survival rate of patients with high-risk abnormalities is less than a year even with aggressive chemotherapy and immunotherapy treatments, compared to 12 years for those without the same abnormalities. "The quality of molecular diagnostic features and dual RNA signature is very critical for us to stratify our patients," Goy said.

As an example of how RNAnalysis and TraceWork have changed treatment and outcomes, Goy discussed the case of a 40-year-old woman with no previous history of cancer who was diagnosed elsewhere with metastatic sarcoma.

"It’s unusual for sarcoma to present right away as a metastatic disease," Goy noted. This patient came to Hackensack, which performed DNA and RNA signatures and analysis, then determined that she had anaplastic large-cell lymphoma with an unusual morphologic presentation that might otherwise seem like metastatic sarcoma.

Knowledge like this allows clinicians to consider nonchemotherapy options that are less toxic. A year and a half later, this woman is free of cancer when doctors previously thought she had a terminal disease, Goy said.

Goy said that this kind of profiling should be a boon for the young but growing field of molecular pharmacology. "Molecular pharmacologists … actually understand the guidelines of molecular medicine and will use them to give recommendations like they do for antibiotics now," he said.

Albitar said that RNA profiling can replace immunohistochemistry and fluorescence in situ hybridization (FISH), making testing less invasive and more convenient for patients. Albitar said that GTC is "trying" to convince payors to cover RNA-seq as first-line testing in difficult-to-diagnose cases.

Some payors in some states are working with GTC to disseminate this knowledge, according to Goy, but it is not a universal sentiment. "Insurance [companies] sometimes have a short-term vision and don’t see the benefit in the long run," he said.

Reposted with permission from GenomeWeb. Initially publication: October 31, 2022. By Neil Versel

On November 3, 2022 Perrigo Company plc (NYSE: PRGO), a leading global provider of Consumer Self-Care Products, reported that its Board of Directors declared a quarterly dividend of $0.26 per share, payable on December 20, 2022, to shareholders of record on December 2, 2022 (Press release, Perrigo Company, NOV 3, 2022, View Source [SID1234622947]).

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On November 3, 2022 Elevation Oncology, Inc. (Nasdaq: ELEV), a clinical stage biopharmaceutical company focused on the development of precision oncology products for patients with genomically defined cancers, reported financial results for the quarter ended September 30, 2022, and highlighted recent progress (Press release, Elevation Oncology, NOV 3, 2022, https://elevationoncology.com/elevation-oncology-reports-third-quarter-2022-financial-results-and-highlights-recent-company-progress/?utm_source=rss&utm_medium=rss&utm_campaign=elevation-oncology-reports-third-quarter-2022-financial-results-and-highlights-recent-company-progress [SID1234622946]).

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"Our newly expanded pipeline provides an opportunity to continue building an industry leading precision oncology company for patients with genomically defined cancers and significant unmet medical needs. We remain focused on execution, as we continue enrolling and treating patients in the ongoing Phase 2 CRESTONE study evaluating seribantumab," said Shawn M. Leland, PharmD, RPh, Founder and Chief Executive Officer of Elevation Oncology. "We are also committed to bringing EO-3021, our differentiated ADC targeting Claudin18.2 into the clinic and plan to initiate a Phase 1 clinical trial in the US in patients with solid tumors in 2023."

Recent Progress and Highlights

Seribantumab

On track to report additional interim data from Cohort 1 of the CRESTONE study in the first half of 2023. CRESTONE is a Phase 2 potential registration-enabling study evaluating seribantumab in patients with solid tumors harboring NRG1 gene fusions. Initial data presented at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting showed positive clinical proof-of-concept data supporting the potential of seribantumab to induce deep and durable benefit for patients.
EO-3021

Phase 1 clinical trial in the US to evaluate EO-3021 in solid tumors expected to initiate in 2023. In July 2022, Elevation Oncology expanded its pipeline through the licensing of EO-3021, a differentiated, clinical-stage antibody-drug conjugate (ADC) that binds to Claudin18.2 and delivers a cytotoxic payload to eliminate tumor cells. Claudin18.2 is a clinically validated oncology target expressed in several solid tumor types including many gastrointestinal cancers such as gastric, gastroesophageal junction, and pancreatic cancer. Our collaboration partner, CSPC Pharmaceutical Group (HKEX: 01093) is currently conducting a Phase 1, dose escalation clinical trial in China in patients with solid tumors.
Expected Upcoming Milestones and Operational Objectives

Additional interim seribantumab data from the Phase 2 CRESTONE study are expected in the first half of 2023
Phase 1 clinical trial in the US to evaluate EO-3021 expected to initiate in 2023
Topline data for seribantumab from the Phase 2 CRESTONE study results are expected in 2024
Ongoing target evaluation and continued execution of the Company’s strategy for future pipeline expansion
Third Quarter 2022 Financial Results

As of September 30, 2022, the Company had cash, cash equivalents and marketable securities totaling $107.9 million, compared to $122.5 million as of June 30, 2022.

Research and development expenses for the third quarter 2022 were $34.3 million, compared to $9.3 million for the third quarter 2021. The increase in R&D expense was primarily related to a one-time upfront license fee of $27.0 million paid to CSPC Megalith Biopharmaceutical Co., Ltd., a subsidiary of CSPC Pharmaceutical Group Limited, for the exclusive rights to develop and commercialize EO-3021 outside of Greater China.

General and administrative expenses for the third quarter 2022 were $4.2 million, compared to $3.0 million for the third quarter 2021. The increase in G&A expense was primarily related to personnel costs, professional services and other administrative costs.

Net loss for the third quarter 2022 was $38.8 million, compared to $12.3 million for the third quarter 2021.

Financial Outlook

Elevation Oncology expects its existing cash, cash equivalents and marketable securities as of September 30, 2022, will be sufficient to fund its current operations into 2024.