On March 31, 2022 Biognosys, a leader in next-generation proteomics solutions for drug discovery and development, reported the details of its presence at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022, which will be held April 8-13, 2022, both in person in New Orleans, US and virtually (Press release, Biognosys, MAR 31, 2022, View Source [SID1234611314]). The company will be presenting a talk and five scientific posters around two of its major service platforms, TrueDiscovery and TrueTarget. Additionally, Biognosys contributed to a poster that will be presented by its collaborator, NeoGenomics.

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"The proteome contains a wealth of information about health and disease, and mass spectrometry-based proteomics has great potential in uncovering that information," commented Lukas Reiter, Ph.D., Chief Technology Officer at Biognosys. "Our AACR (Free AACR Whitepaper) data shows how Biognosys has been able to establish mass spectrometry-based proteomics technology to provide detailed and unbiased insights about the proteome. We illustrate how we’ve been able to increase efficiency and scalability at every step of the process. Moreover, we’re showcasing data we’ve generated with our collaborators that demonstrate the practical application of our platforms across all stages of research and drug development, from discovery to clinical settings."

Biognosys will also be present with a team of scientific experts at booth #885.

Talk Details

Abstract 2136: Prediction of small molecule-protein binding events for BRD4 and EGFR inhibitors using HR-LiP, a novel structural proteomics approach
Presenter: Yuehan Feng, Ph.D.
Collaborator: Cedilla Therapeutics
Platform, Technology and Application: TrueTarget, High Resolution Limited Proteolysis Mass Spectrometry (HR-LiP), Drug Target Validation
Session: Drug Design and Lead Optimization Strategies Toward Novel or Difficult-to-Drug Cancer Targets
Date and Time: Monday, April 11, 3:20 p.m. – 3:35 p.m. CDT
Poster Presentation Details

Abstract 1374: Discovery of MHC class I and class II neoantigens in lung cancer in needle biopsy tissue samples using an optimized high-throughput workflow
Presenter: Marco Tognetti, Ph.D.
Platform, Technology and Application: TrueDiscovery, Hyper Reaction Monitoring, Immunopeptidome Profiling
Session: Tumor Antigens, Antigen Presentation, and Tumor Immunity
Date and Time: Monday, April 11, 9:00 a.m. – 12:30 p.m. CDT
Abstract 3110: Identification of the phosphorylation network in PDX and corresponding organoid (PDXO) models upon targeted therapy treatment using deep phosphoproteomic analysis
Presenter: Yuehan Feng, Ph.D.
Collaborator: Crown Bioscience
Platform, Technology, and Application: TrueDiscovery, Hyper Reaction Monitoring, Phosphoproteome Profiling
Session: Patient-Derived Xenografts
Date and Time: Tuesday, April 12, 1:30 p.m. – 5:00 p.m. CDT
Abstract 2924: Target identification, selectivity profiling and mechanistic insights of a CDK9 inhibitor using complementary proteomics methods
Presenter: Yuehan Feng, Ph.D.
Collaborator: AstraZeneca plc
Platform, Technology and Application: TrueTarget, Limited Proteolysis Mass Spectrometry (LiP-MS), Drug Target Deconvolution
Session: Structural and Chemical Biology
Date and Time: Tuesday, April 12, 9:00 a.m. – 12:30 p.m. CDT
Abstract 3923: Ubiquitin ligases implicated as predictive biomarkers for poor outcome to immunotherapy in melanoma patients
Presenter: Jakob Vowinckel, Ph.D.
Collaborator: NeoGenomics Laboratories
Platform, Technology and Application: TrueDiscovery, Hyper Reaction Monitoring, Tissue Biomarker Discovery
Session: Proteomics, Signaling Networks, and Biomarker Discovery
Date and Time: Wednesday, April 13, 9:00 a.m. – 12:30 p.m. CDT
Abstract 3920: Precise solid tumor classification through unbiased quantification of proteoforms deep into tissue leakage
Presenter: Marco Tognetti, Ph.D.
Platform, Technology and Application: TrueDiscovery, Hyper Reaction Monitoring, Biofluid Biomarker Discovery
Session: Proteomics, Signaling Networks, and Biomarker Discovery
Date and Time: Wednesday, April 13, 9:00 a.m. – 12:30 p.m. CDT
NeoGenomics Poster Collaboration Details

Abstract 1267: Dual approach using unbiased proteomics and multiplexed immunofluorescence for the detection of markers predictive for immunotherapy in melanoma patients
NeoGenomics Presenter: Anna Juncker-Jensen, Ph.D.
Biognosys co-authors: Nigel Beaton, Ph.D.; Kristina Beeler, Ph.D. Tobias Treiber, Ph.D.; Jakob Vowinckel, Ph.D.
Platform, Technology and Application: TrueDiscovery, Hyper Reaction Monitoring, Tissue Biomarker Discovery
Session: Biomarkers Predictive of Therapeutic Benefit 2
Date and Time: Monday, April 11, 9:00 a.m. – 12:30 p.m. CDT
For the most up to date information and resources about Biognosys’ presence at AACR (Free AACR Whitepaper), visit biognosys.com/accr22.

About TrueDiscovery

The Biognosys TrueDiscovery platform offers integrated proteomics solutions across the entire drug development pipeline.

TrueDiscovery is powered by Hyper Reaction Monitoring (HRM) mass spectrometry, an advanced Data Independent Acquisition (DIA)-based protein quantification technology co-invented and patented by Biognosys.

TrueDiscovery is the only platform that searches the complete proteome to quantify thousands of the most relevant proteins, including an unlimited number of proteoforms. The platform enables the deepest unbiased profiling of tissue and biofluids proteomes with unbeatable specificity on a large scale. The generated data are highly reproducible and easily transferrable to clinical assays. Studies can be performed in a GLP certified and GCP compliant environment. For more information, visit truediscovery.bio.

About TrueTarget

The Biognosys TrueTarget proteomics platform uniquely addresses the most pressing challenges in early drug discovery by identifying on- and off-targets to accelerate and de-risk drug development throughout the pipeline.

TrueTarget is powered by Limited Proteolysis Mass Spectrometry (LiP-MS), a proprietary, patented chemoproteomics technology co-developed by Biognosys.

TrueTarget is the only tool to probe structural changes across the complete proteome with peptide-level resolution, providing unique insights into compound binding and target identification. The platform enables elucidating mechanisms of action and revealing unanticipated toxicities. For more information, visit truetarget.bio

On March 31, 2022 Hummingbird Diagnostics GmbH, a leader in reading blood-based microRNAs for early disease detection and characterization, reported the publication of a study in the journal npj Precision Oncology that describes the discovery and validation of miRisk, a first-of-its-kind microRNA (miRNA) biomarker signature that offers the prospect of a blood-based companion diagnostic for immunotherapy in advanced non-small cell lung cancer (NSCLC) (Press release, Hummingbird Bioscience, MAR 31, 2022, View Source [SID1234611312]). The results of the study illustrate the development of miRisk and its prognostic value for overall survival following immunotherapy. The blood-based test holds the potential for broad applicability for the management of patients with cancer.

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Biomarkers have been shown to help identify patients who are most likely to benefit from treatment with immunotherapies, such as PD-1/PD-L1-targeted therapies. Still, the gold standard of tumor tissue PD-L1 staining, as well as other biomarkers, such as tumor mutational burden, do not always accurately predict the efficacy of these therapies in patients. Immune cell-derived miRNAs reflect physiological and pathophysiological processes and can be sampled from peripheral blood. miRNAs are short non-coding RNAs that act as molecular rheostats that exercise control over the expression of most human genes. miRNAs are relatively accessible yet reflect complex underlying biology, offering the prospect of a blood-based companion diagnostic to determine the best candidates for immunotherapy.

"Immunotherapy has revolutionized the treatment of non-oncogene-driven advanced cancers, yet only approximately 30 percent of patients respond favorably to these treatments. Determining which patients will respond to these powerful therapies represents one of the greatest needs in oncology," said Bruno Steinkraus, PhD, Chief Scientific Officer of Hummingbird Diagnostics and leader of the published research. "This study illustrates the potential of how miRNAs taken from peripheral whole blood can be analyzed to predict the efficacy of PD-(L)1-targeted therapies in patients with NSCLC and improve patient outcomes. We thank all of our collaborators and patients for their vital contributions to this research."

The work, led by Hummingbird, was part of a collaboration with Priv.-Doz. Petros Christopoulos, MD, and Prof. Michael Thomas, MD, of the Thoraxklinik at Heidelberg University Hospital, as well as Prof. Martin Reck, MD, of the LungenClinic Grosshansdorf.

"Patient stratification — identifying who will benefit from treatment and who will likely fail to respond or even come to harm due to adverse events — remains challenging, and there is pressing need for more accurate biomarkers for immunotherapy response prediction," said Jochen Kohlhaas, Founder and Chief Executive Officer of Hummingbird Diagnostics. "This work could broaden our general understanding of tumor immunobiology and, if validated, offers several important opportunities to improve management and outcomes of patients with advanced NSCLC and possibly other cancers."

Based on an analysis of whole blood miRNA profiling of 3 well-characterized cohorts consisting of a total of 334 stage IV NSCLC patients, the team discovered and validated a 5-microRNA risk score (miRisk) that is prognostic of overall survival following immunotherapy of either pembrolizumab or nivolumab. In a head-to-head comparison of miRisk and the gold standard PD-L1 tumor proportion score (TPS), the authors report superior diagnostic performance of miRisk compared to PD-L1 status. Of note, the miRNA signature does not require tumor tissue and could thus be employed in an off-the-shelf manner. The identified biomarker signature was traced back to a myeloid origin, especially neutrophils and macrophages, and miRNA target prediction was performed to identify a potential direct mechanistic link to the PD-L1 signaling pathway and PD-L1 itself. One example of a potential first clinical application is the selection of PD-L1-high patients for either single agent immunotherapy or chemo-immuno combination therapy where presently both options are licensed. In addition, miRisk measurements might provide a tissue-independent way of identifying by current standards ineligible patients who may benefit from immuno-monotherapy.

The open-access article can be found online on the journal’s website: View Source

On March 31, 2022 Numab Therapeutics AG ("Numab") and Ono Pharmaceutical Co., Ltd. ("Ono") reported that Ono has exercised its option to enter into a development and license agreement for a multispecific antibody candidate that was generated through a research collaboration between the two companies initiated in 2017 (Press release, Numab, MAR 31, 2022, View Source [SID1234611311]). Under the terms of the license agreement, Ono acquires intellectual property rights and exclusive global development and commercialization rights for a multispecific antibody targeting a novel immuno-oncology target. In consideration for the discovery work and the license, Numab will receive up to CHF 258 million in research funding, upfront and milestone payments plus tiered single to double digit royalties on future sales.

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"We are proud to have advanced this novel multispecific antibody candidate against a new and very challenging immuno-oncology target to this milestone," said David Urech, Ph.D., Founder and Chief Executive Officer of Numab. "The licensing of the asset is an affirmation of our platform’s ability to create novel and unique pharmacology. We look forward to our continued partnership with Ono as we have been further collaborating on our shared mission of developing next generation immuno-oncology therapeutics for patients with cancer."

"We hope that the program candidate generated through our work with Numab will become a therapeutic option for patients with cancer who may benefit from this new treatment modality," said Toichi Takino, Senior Executive Officer / Executive Director, Discovery & Research of Ono Pharmaceutical. "Through the synergy between Numab’s multispecific antibody platform and Ono’s deep immuno-oncology expertise, we are continuously committed to fueling our diverse drug discovery efforts to advance our immuno-oncology pipeline."

In March 2020, Numab and Ono expanded their alliance by executing a second research and option agreement to discover and develop a multispecific antibody drug candidate for the treatment of various cancers.

On March 31, 2022 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported that the European Medicines Agency (EMA) has granted obe-cel, Autolus’ leading CAR T clinical candidate, Orphan Medical Product Designation for treatment of acute lymphoblastic leukemia (ALL) patients (Press release, Autolus, MAR 31, 2022, View Source [SID1234611309]).

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"This regulatory designation is an important milestone toward addressing the urgent, unmet need in ALL patients, and follows the Orphan Drug Designation previously granted in 2019 by the U.S. Food and Drug Administration (FDA) for obe-cel," said Dr. Christian Itin, Chief Executive Officer of Autolus. "Recruitment is ongoing in the Phase 2 portion of the pivotal study of obe-cel and we look forward to announcing first Phase 2 data this year."

Orphan Medical Product Designation is granted by the EMA Committee for Orphan Medicinal Products to medicines intended for the treatment, diagnosis or prevention of a disease that is life-threatening or chronically debilitating that affects fewer than 5 in 10,000 people in the EU under the Orphan Regulation (Regulation (EC) No 141/2000). To attain Orphan Designation, it must be justified that a medicinal product could potentially be of significant benefit compared to the existing authorized medicinal products for those affected by the condition. Benefits of the designation include fee reductions for centralized activities including; applications for marketing authorization, inspections and scientific advice. A key benefit is ten years of market exclusivity in the EU following marketing approval by the EMA.

On March 31, 2022 Savara Inc. (Nasdaq: SVRA), an orphan lung disease company, reported financial results for the fourth quarter and full year ending December 31, 2021 and provided a business update (Press release, Savara, MAR 31, 2022, View Source [SID1234611306]).

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"The company underwent a significant amount of positive change in 2021. We streamlined the pipeline to focus solely on the molgramostim development program, strengthened our management team with key hires in clinical, regulatory, and CMC and improved our financial position with a $130M equity raise," said Matt Pauls, Chair and CEO, Savara. "With a cash position of ~$161M at the end of 2021, and a track record of fiscal discipline, we believe we are funded through 2025 – which is well beyond the anticipated top line read-out of IMPALA-2, our pivotal Phase 3 clinical trial in aPAP. We continue to advance the IMPALA-2 trial with most of the approximately 50 clinical trial sites now activated, and despite the ongoing COVID-19 pandemic and current geopolitical issues impacting parts of Europe, we reaffirm our guidance of top line data by the end of 2Q 2024."

Fourth Quarter Financial Results (Unaudited)

Savara’s net loss for the fourth quarter of 2021 was $11.3 million, or $(0.07) per share, compared with a net loss of $13.7 million, or $(0.23) per share, for the fourth quarter of 2020.

Research and development expenses were $7.6 million for the fourth quarter of 2021, compared with $10.2 million for the fourth quarter of 2020.

General and administrative expenses for the fourth quarter of 2021 and 2020 were $3.0 million and $2.8 million, respectively.

As of December 31, 2021, the Company had cash, cash equivalents and short-term investments of $161.2 million.

Fiscal Year 2021 Financial Results

The Company’s net loss for the year ended December 31, 2021 was $43.0 million, or $(0.32) per share, compared with a net loss of $49.6 million, or $(0.84) per share for the year ended December 31, 2020.

Research and development expenses decreased $6.0 million, or 17.3%, to $29.0 million for the year ended December 31, 2021 from $35.0 million for the year ended December 31, 2020. The decrease was largely attributable to $5.4 million of acquisition costs for the inhaled ciprofloxacin product candidate (in 2020) and a $6.9 million decrease in Chemistry, Manufacturing, and Controls (CMC) and clinical operations activities associated with the wind down of the inhaled vancomycin study. This was partially offset by a $6.4 million increase in costs associated with the startup and progression of the IMPALA-2 trial.

General and administrative expenses decreased $1.9 million, or 13.4%, to $12.4 million for the year ended December 31, 2021 from $14.3 million for the year ended December 31, 2020. The decrease was primarily due to the recognition of a one-time non-recurring charge of $0.8 million for non-cash stock-based compensation and approximately $1.5 million of paid and accrued severance payments to former members of the Company’s executive management during the year ended December 31, 2020.