On November 3, 2022 Cerus Corporation (Nasdaq: CERS) reported financial results for the third quarter ended September 30, 2022 (Press release, Cerus, NOV 3, 2022, View Source [SID1234622952]).

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Recent developments and highlights include:

Third quarter 2022 total revenue of $46.3 million, reflecting a 10% increase over the third quarter of 2021. Total revenue was composed of (in thousands, except %):
Awarded $9.1 million contract by the U.S. Department of Defense (DoD) Industrial Base Analysis and Sustainment (IBAS) program for development of pathogen reduced, lyophilized cryoprecipitate ("LyoCryo") to treat bleeding due to trauma.
Received Health Canada approval for 7-day shelf life for INTERCEPT platelets.
Receipt of feedback from and close-out of all modules by our notified body, TUV, related to our INTERCEPT Red Blood Cells CE Mark submission; review by the competent authority is ongoing.
Third quarter 2022 net loss attributable to Cerus Corporation of $8.5 million, or $0.05 per basic and diluted share, reflecting an improvement of $3.9 million over the prior year period of $12.4 million, or $0.07 per basic and diluted share, led by strength of growing INTERCEPT platelet business in North America coupled with continued financial discipline.
Non-GAAP Adjusted EBITDA for the third quarter of 2022 was negative $2.7 million, compared to negative $5.6 million during the prior year period. For additional information, please see definitions and the reconciliation of this non-GAAP measure to net loss attributable to Cerus Corporation accompanying this release.
Narrowing full-year product revenue guidance to a range of $160 million to $162 million, given foreign exchange rate headwinds anticipated for the rest of the year.
Cash, cash equivalents, and short-term investments were $103.8 million at September 30, 2022.
"We continue to execute in this challenging environment on our mission to make INTERCEPT the standard of care in transfusion medicine and have progressed on our manufacturing expansion efforts," said William ‘Obi’ Greenman, Cerus’ president and chief executive officer. "Despite the 6% FX headwind, the resiliency in our business of safeguarding the global blood supply is evident in our results, even in the face of continued macroeconomic and geopolitical challenges over the balance of this year."

"We expect to see continued strong demand for INTERCEPT platelets as we close out the year, and at the same time, we see expanded adoption of our IFC product, which we believe has the potential to impact patient’s lives meaningfully. Given the global nature of our business coupled with expectations for strength of the dollar going forward, we are narrowing our 2022 product revenue guidance range to $160-162 million for the full year," Greenman continued. "Importantly, with this growth and our continued financial discipline, we have been progressing towards our goal of cashflow breakeven."

Revenue

Product revenue during the third quarter of 2022 was $39.6 million, compared to $36.1 million during the prior year period. The 10% year-over-year growth in product revenue during the quarter comes despite ongoing headwinds associated with unfavorable foreign exchange rates, primarily with respect to average EUR:USD rates, resulting in a top-line impact of 6% year-over-year. The reported revenue growth was once again led by continued demand for our platelet products in North America.

Third quarter 2022 government contract revenue was $6.8 million, compared to $6.0 million during the prior year period. Reported government contract revenue is comprised of funding associated with research and development (R&D) activities related to the INTERCEPT Blood System for Red Blood Cells and sponsored efforts related to the development of next-generation pathogen reduction technology for whole blood.

Product Gross Profit & Margin

Product gross profit for the third quarter of 2022 was $21.9 million, increasing by $3.4 million over the prior year period. Product gross margin for the third quarter of 2022 was 55.4% compared to 51.3% for the third quarter of 2021, up more than 300 basis points compared to the second quarter of 2022.

Operating Expenses

Total operating expenses for the third quarter of 2022 were $36.1 million compared to $35.6 million for the same period of the prior year. Ongoing financial discipline during the quarter continues to position the Company for reaching cashflow breakeven in the near term.

Selling, general and administrative (SG&A) expenses for the third quarter of 2022 totaled $19.9 million, compared to $20.4 million for the third quarter of 2021, reflecting ongoing leverage of the Company’s expense structure with ramping product sales.

R&D expenses for the third quarter of 2022 were $16.2 million, compared to $15.3 million for the third quarter of 2021. In the third quarter, the increase in Company’s R&D expenses on a year-over-year basis were related to the development of next generation product and LED illuminator, partially offset by the completion of the Phase IV PIPER study in 2021.

Net Loss Attributable to Cerus Corporation

Net loss attributable to Cerus Corporation for the third quarter of 2022 was $8.5 million, or $0.05 per basic and diluted share, compared to a net loss attributable to Cerus Corporation of $12.4 million, or $0.07 per basic and diluted share, for the third quarter of 2021.

Non-GAAP Adjusted EBITDA

Non-GAAP Adjusted EBITDA for the third quarter of 2022 was negative $2.7 million, compared to non-GAAP Adjusted EBITDA of negative $5.6 million for the third quarter of 2021. For additional information, please see definitions and the reconciliation of this non-GAAP measure to net loss attributable to Cerus Corporation accompanying this release.

Balance Sheet & Cash Use

At September 30, 2022, the Company had cash, cash equivalents and short-term investments of $103.8 million, compared to $107.0 million at June 30, 2022.

As of September 30, 2022, the Company carried $55.0 million of notes due and a balance on its revolving line of credit of $14.9 million. The Company continues to have access to an additional $5 million under its revolving line of credit.

For the third quarter, net cash used in operating activities totaled $2.1 million as compared to $6.6 million during the prior year period. For the first nine months of 2022, net cash used in operating activities totaled $23.9 million as compared to $32.7 million during the prior year period. In both instances, increased product sales and their gross profit contribution, as well as strong working capital management, drove the improvements versus the prior year period.

2022 Product Revenue Guidance

The Company is narrowing its previously stated product revenue guidance range. The Company expects full-year 2022 product revenue to be in the range of $160 million to $162 million, compared to its previous guidance range of $160 million to $165 million, representing anticipated continued strong demand for our products and expectations for continued strength of the U.S. dollar versus the Euro.

Quarterly Conference Call

The Company will host a conference call at 4:30 P.M. EDT this afternoon, during which management will discuss the Company’s financial results and provide a general business overview and outlook. To listen to the live webcast, please visit the Investor Relations page of the Cerus website at View Source

A replay will be available on Cerus’ website approximately three hours after the call through November 17, 2022.

On November 3, 2022 Ichnos Sciences Inc., a global clinical-stage biotechnology company developing innovative multispecific immune cell engager antibodies in oncology, reported that four abstracts highlighting data on its pipeline assets have been selected for presentation at the upcoming 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (Press release, Ichnos Sciences, NOV 3, 2022, View Source [SID1234622951]). The meeting will be held December 10-13, 2022, in New Orleans, Louisiana.

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These abstracts discuss three assets – ISB 1342, ISB 1442 and ISB 2001 – two of which are currently in Phase 1 clinical studies in relapsed/refractory multiple myeloma. The data to be presented will highlight the potential of the company’s proprietary BEAT platform1 and its growing momentum toward providing curative therapies that may extend and improve lives.

"Our work is grounded in the notion that cure is possible and the understanding that there is room for novel thinking in the pursuit of effective, disease-centric therapies for oncology," said Cyril Konto, M.D., President and Chief Executive Officer of Ichnos Sciences. "Ichnos is focused on harnessing the power of the immune system in the fight against cancer, and we are pleased to have been selected to present data supporting this approach at the ASH (Free ASH Whitepaper) Annual Meeting."

The full ASH (Free ASH Whitepaper) 2022 Annual Meeting abstracts are available for review at: View Source
Visit the Ichnos Sciences booth (#3127) to learn more about Ichnos’ proprietary BEAT platform, expanding pipeline of oncology assets, partnership opportunities and more.

Follow Ichnos on LinkedIn for more updates throughout the conference.

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