On June 10, 2020 Ryvu Therapeutics (WSE: RVU) reported its first quarter 2020 financial results and provided a corporate update (Press release, Ryvu Therapeutics, JUN 10, 2020, View Source [SID1234560981]).

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"Q1 2020 was an exciting and eventful time for Ryvu. We have managed to achieve several important milestones including clinical ones, such as the successful completion of Phase I for SEL24/MEN1703 in acute myeloid leukemia, as well as the receipt of an Orphan Drug Designation for SEL120 for the treatment of AML patients," commented Pawel Przewiezlikowski, Chief Executive Officer of Ryvu.

"We have also secured additional non-dilutive grant financing to support the development of our synthetic lethality program. Together with very good data from our early pipeline projects, it allows us to successfully continue our mission to discover and develop drugs that will improve the lives of cancer patients and their families," adds Przewiezlikowski.

Recent Achievements

On February 26, Ryvu signed a grant agreement for the development of targeted oncology therapies based on the synthetic lethality concept. This grant provides Ryvu with almost $8.3 million of non-dilutive financing to discover, develop and select a clinical candidate targeting cancers which had been considered in the past as largely undruggable using rational approaches. Total net value of the project amounts to $14.0 million and the anticipated project duration is until December 2023.
On March 5, 2020 Menarini Group announced the successful completion of Phase I clinical study of SEL24/MEN1703 in Acute Myeloid Leukemia, which entitled Ryvu to receive a $1.96 million milestone payment. The full data from the study will be presented as a poster "Results of the dose escalation part of DIAMOND trial (CLI24-001): First-in-human study of SEL24/MEN1703, a dual PIM/FLT3 kinase inhibitor, in patients with acute myeloid leukemia" during the Virtual 25th EHA (Free EHA Whitepaper) Congress taking place June 11-21.

Throughout the dose escalation part, SEL24/MEN1703 showed an acceptable safety profile up to the recommended dose established at 125 mg/day (14 days ON – 7 days OFF in 21-days cycles). Initial evidence of single agent efficacy was observed with 1 CR and 1 CRi in elderly patients who had exhausted standard therapeutic options. Cohort Expansion study planned in relapsed/refractory AML patients in the United States and Europe including Poland will further investigate the single agent activity and the safety profile of SEL24/MEN1703.
On March 25, 2020 the U.S. Food and Drug Administration (FDA) granted an orphan drug designation (ODD) to Ryvu’s SEL120, for the treatment of patients with acute myeloid leukemia (AML).
Important milestones in 2020, before the report date

On April 16, Galapagos NV (Euronext &NASDAQ: GLPG) and Ryvu Therapeutics S.A. (WSE: RVU) announced a collaboration focused on the discovery and development of novel small molecule drugs in inflammation. The collaboration is based on a novel target identified by Ryvu. Ryvu will contribute its biology and chemistry platform as well as related intellectual property to the program. During the joint research collaboration, Ryvu is responsible for early drug discovery and Galapagos will be responsible for all further development of the program.
On June 2, Ryvu obtained the occupancy permits for its newly built R&D Center for Innovative Drugs, meaning it has completed the construction of the facility. The Company plans to move to the new headquarters by the end of June 2020.
On June 3, NodThera, Ryvu spin-off company, secured £44.5million ($54.5million) Series B financing. NodThera was founded by Epidarex Capital and Ryvu in 2016 based on world class research on NLRP3 inflammasome conducted at Ryvu (at that time Selvita) in 2012 -2016. The company focused on the development of inflammasome inhibitors has already raised over £80.8 million (over $100 million) in three funding series. After the full completion of Series B capital increase, Ryvu will own 4.8% in NodThera.
On June 4, Ryvu signed a grant agreement for the development of targeted immuno-oncology therapy, which provides Ryvu with almost $5.6 million of non-dilutive financing to discover, develop and select a clinical candidate targeting cancers which had been considered in the past as largely undruggable using rational approaches. Total net value of the project amounts to over $8.9 million and the anticipated project duration is until December 2023.
In Q1 2020, Ryvu Therapeutics participated and presented at several investor conferences, including Solebury Trout Investor Access during JP Morgan 2020, Solebury Trout Virtual Investor Conference, BIO-Europe Spring 2020 and 32nd Annual ROTH Conference.

Ryvu First Quarter 2020, Financial Results

In the past quarter, Ryvu Therapeutics noted a 56% increase in its revenues, up to PLN 13.6 million ($3.5 million). Revenues from partnering contracts have increased from PLN 1.1 million ($0.3 million) in Q1 2019, up to PLN 7.8 million ($2.0 million) in Q1 2020.

Operational costs related in majority to the research and development expenditures, remain at a stable level and amounted to PLN 18.6 million ($4.7 million), as compared to PLN 18.4 million ($4.9 million) in same quarter last year. Operational loss has decreased and amounted to PLN 5.0 million ($1.3 million), compared to PLN 9.7 million ($2.6 million) in 1Q 2019.

On June 5, 2020, Ryvu Therapeutics held PLN 48.2 million ($11.6 million) in cash, cash equivalents, and short-term investments.

On June 10, 2020 BryoLogyx Inc. reported it has entered into two agreements with Neurotrope, Inc. (Nasdaq: NTRP) to acquire Neurotrope’s preclinical data package and drug product for use of bryostatin-1 in an immuno-oncology application, and to supply Neurotrope with synthetic bryostatin-1 for use in clinical trials and commercialization for the treatment of Alzheimer’s disease and other neurodegenerative diseases. Neurotrope has been developing bryostatin-1 under a Cooperative Research and Development Agreement ("CRADA") with the National Cancer Institute (NCI) (Press release, BryoLogyx, JUN 10, 2020, View Source [SID1234560980]). Specific financial terms were not disclosed.

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"BryoLogyx is capitalizing on recent advances demonstrating that bryostatin-1 has great promise in amplifying the immune response to cancer immunotherapies by multiple mechanisms, including increasing tumor antigen expression," said Thomas M. Loarie, CEO of BryoLogyx. "These two agreements with Neurotrope will accelerate our drive to clinical trials to demonstrate proof-of-concept in patients." The agreements should position BryoLogyx to begin clinical trials with the NCI in late 2020.

Under the first agreement, Neurotrope will transfer to BryoLogyx the right to develop bryostatin­‑1 for the potential treatment of CD22+ B-cell acute lymphoblastic leukemia (ALL). Relapsed ALL in CD22 CAR-T treated patients has been associated with reduced CD22 antigen density. Bryostatin-1 has been shown to increase CD22 expression levels in leukemia patients, and in a mouse model, resulted in improved treatment response and durability. Neurotrope will also transfer to BryoLogyx the Investigational New Drug ("IND") application that is in development. BryoLogyx will be responsible for the IND going forward and will pay a nominal fee on gross revenues generated by the commercial sale of bryostatin-1 product sold by BryoLogyx for the treatment of ALL.

In return, under a supply agreement, BryoLogyx will supply Neurotrope with specified amounts of synthetic, GMP-grade bryostatin-1 for manufacture of drug product to be used in clinical trials for treating Alzheimer’s and other neurological diseases. Bryostatin-1 will be chemically synthesized by Albany Molecular Research Inc. (AMRI) in collaboration with BryoLogyx.

"AMRI is privileged to be selected for this complex commercial synthesis, the first ever to be conducted for bryostatin-1," said Christopher Conway, AMRI President. "Bryostatin-1 is based from a rare marine resource; our ability to provide GMP-level material for a clinical studies program should greatly reduce the cost, and minimize related supply chain risks, for this promising therapeutic."

BryoLogyx is currently the sole global source of synthetic bryostatin-1. The Company has invested heavily to scale the patented bryostatin-1 synthesis technology developed by Dr. Paul Wender at Stanford University and licensed by the Company from Stanford. This technology holds great promise for development of commercial scale quantities of bryostatin-1 at a cost much less than the cost of extracting natural bryostatin-1 from marine sources, which has proven prohibitive for commercial development.

On June 10, 2020 bioAffinity Technologies, a privately held biotech company, reported its test validation trial for CyPath Lung resulted in specificity of 88% and sensitivity of 82%, similar to far more invasive procedures commonly used to diagnose lung cancer (Press release, BioAffinity Technologies, JUN 10, 2020, View Source [SID1234560979]). Overall accuracy of CyPath Lung was 87%.

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"It is important to note that our clinical trial compared two very similar and therefore difficult to distinguish cohorts – those at high risk with and without cancer – in contrast to the trials of some other lung cancer diagnostics that compare healthy individuals to cancer patients," said bioAffinity President and Chief Executive Officer Maria Zannes. "In an earlier study using our porphyrin marker to label sputum on microscope slides, we saw 100% accuracy in distinguishing sputum from healthy patients from those diagnosed with lung cancer. While impressive, the true test of a lung cancer diagnostic is its ability to distinguish those at high risk with and without cancer, which CyPath Lung has shown it can do. We are extremely pleased with our trial results."

CyPath Lung is a flow cytometric test to aid in the diagnosis of lung cancer. Patients collect sputum samples non-invasively at home, a particular benefit during the COVID-19 pandemic. The sample is shipped overnight to the laboratory for processing. Sample data is acquired by flow cytometry, a technique that can count, sort and profile individual cells with remarkable speed. Using an automated analysis with pre-set parameters, CyPath Lung profiles the lung environment including the presence of cancer-associated cells. Data acquisition and physician reports can be generated in minutes.

"We successfully tested our automated analysis program and found it to be fast and very robust in predicting what is cancer and what is high risk," Zannes said. "Physician reports can be generated immediately after flow cytometry acquires sample data in approximately 20 minutes per sample. Looking beyond lung cancer, we believe our automated platform can be successfully applied to cancer diagnostic tests for several other cancers, which will improve overall survival rates for patients through early diagnosis and treatment."

CyPath Lung’s automated flow cytometry analysis reveals four distinct predictors that distinguish the sputum collected from high-risk individuals from samples collected from patients with lung cancer. The Company’s proprietary porphyrin compound preferentially binds to cancer and cancer-related cells. Confirming results of an earlier clinical trial, the test validation trial found cells labeled by the porphyrin molecule are a significant predictor in the test’s automated analysis for lung cancer.

"CyPath Lung reveals the lung environment by profiling cell populations in sputum, meaning that the test analyzes whole cells and cell populations to find predictors of lung cancer in a sample that comes directly from the lung," explained Executive Vice President and Chief Medical and Science Officer Vivienne Rebel, MD, PhD. "Our technology has advantages over genomic approaches to diagnosing earlier stage lung cancers that search for circulating tumor cells or cell fragments in a much more dilutive blood sample."

The test validation trial compared sputum samples collected from individuals at high risk for lung cancer with sputum samples collected from lung cancer patients. Individuals at high risk for lung cancer have smoked the equivalent of one pack of cigarettes a day for 30 years or more, have not quit smoking in the past 15 years and are 55-80 years of age. Trial participants who had lung cancer collected their sputum sample prior to tissue biopsy that confirmed the cancer diagnosis. Trial results are based on analysis of 150 samples, including samples from 122 high-risk individuals and 28 people diagnosed with lung cancer.

The U.S. Preventive Services Task Force recommends that smokers and former smokers at high risk for lung cancer undergo annual screening by low dose computed tomography (LDCT). Screening by LDCT has been proven to detect lung cancer at earlier stages when it can be more successfully treated. The National Lung Cancer Screening Trial (NLCST) of more than 53,000 participants resulted in a 20% decrease in lung cancer–specific mortality when LDCT screening is performed in high-risk patients. However, screening by LDCT had a low 3.8% positive predictive value (PPV) which raises the risk of unnecessary, invasive, and costly procedures for those who test positive. In the NLCST, for every 100 people who received a positive LDCT, less than four of those individuals actually had lung cancer.

In the CyPath Lung validation trial, the non-invasive test showed a positive predictive value (PPV) of 62% and a 95% negative predictive value (NPV). Using CyPath Lung after a positive LDCT screen has the potential to significantly improve PPV compared to LDCT alone in the high-risk population. Early diagnosis of lung cancer followed by treatment can significantly increase survival. Studies have shown early diagnosis of lung cancer can increase the 10-year survival rate of the disease to 88% from the present overall 5-year survival rate of 19%.

"CyPath Lung proves to be a highly accurate test for the early detection of lung cancer," Zannes said. "Our test for lung cancer has high specificity and sensitivity, in contrast to other tests that claim high sensitivity and very low specificity, or vice versa."

CyPath Lung has been licensed by Precision Pathology Services, a CAP/CLIA laboratory in San Antonio, TX. Precision Pathology Services anticipates certification and sale of CyPath Lung in 2020 as a Laboratory Developed Test (LDT). Following its certification as an LDT, physicians will order CyPath Lung for their patients at high risk for lung cancer and who receive a positive screening result or otherwise are suspected of having the disease.

On June 10, 2020 Genetron Holdings Limited ("Genetron Health"), a China-based precision oncology company that covers full-cycle cancer care, reported that it has entered into a strategic partnership agreement with Thermo Fisher Scientific ("Thermo Fisher") (Press release, Genetron Health Technologies, JUN 10, 2020, View Source [SID1234560978]). With plans to build on the sequencer Genetron S5 (Registration Number 20193220820), the partnership aims to enhance innovation, commercialization and promotion of next-generation sequencing (NGS) platforms in the field of molecular cancer diagnosis in China’s public hospitals.

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The partnership will introduce the Company’s medium-throughput NGS system Genetron S5 to public hospitals across China, promoting the application and development of Genetron S5 in more fields, such as reproductive health, genetic disease, pathological microorganism testing, and other independent clinical lab testing and scientific research.

"Genetron Health is committed to leading and empowering the cancer diagnostics and treatment industry," said Wang Sizhen, Co-Founder and CEO of Genetron Health. "We have been working with Thermo Fisher, one of our world-class partners, to create open, flexible, convenient and accurate molecular diagnostic products, and to offer a comprehensive one-stop solution. Our products and solutions are designed to empower medical institutions continuously improve their diagnostic and treatment capabilities, bringing more accurate and speedy services to the public."

" Thermo Fisher’s memorandum of understanding with Genetron in this field will help further our companies’ work together to develop IVD solutions for the fast-growing Chinese market," said Tony Acciarito, president of Thermo Fisher Scientific China "We look forward to generating significant customer value and promoting precision medicine to improve public health throughout China."

Genetron Health has received National Medical Products Administration (NMPA) approval for clinical application of seven IVD products, which cover three major technology platforms: the NGS, dPCR and qPCR. The NGS platform, in particular, provides flexible combinations of products with low, medium and high-throughput. Genetron Health also offers overall R&D and commercialization solutions for in vitro diagnostic technologies available on multiple technology platforms and in various application scenarios, by fully exploring and tapping into the unique strength of each technology platform.

About Genetron S5

Genetron S5 platform approved by NMPA on November 1, 2019, is a medium-throughput NGS system that enables simple targeted sequencing workflows at an affordable price, without compromising on performance or reliability. Genetron S5 platform offers several throughput options, which provide the flexibility to scale from small to large projects, enabling multiple targeted sequencing applications on a single system. Based on the design, Genetron S5 is particularly suitable for hospitals as it offers more practical solutions and greater scalability.

On June 10, 2020 Menarini Group, a privately held Italian pharmaceutical and diagnostics company, reported that is has successfully completed the acquisition of Stemline Therapeutics Inc., a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics (Nasdaq: STML), for an aggregate cash consideration up to $677 million on a fully diluted basis (Press release, Menarini, JUN 10, 2020, View Source [SID1234560977]).

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The transaction, which was announced on 4 May 2020, strengthens Menarini’s oncology portfolio with the addition of both commercial and clinical-stage assets. Menarini acquired Stemline for an upfront payment of $11.50 per share in cash and one non-tradeable Contingent Value Right (CVR) that will entitle each holder to an additional $1.00 per share in cash upon completion of the first sale of ELZONRIS in any EU5 country after European Commission approval.

Stemline launched ELZONRIS for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adult and pediatric patients, two years or older, following the approval by the United States Food and Drug Administration in December 2018. ELZONRIS is a novel targeted therapy directed to the interleukin-3 (IL-3) receptor-α (CD123), a target present on a wide range of malignancies. In parallel, Stemline has been evaluating ELZONRIS in clinical trials in additional indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), acute myeloid leukemia (AML), and others. Stemline’s additional pipeline candidates include felezonexor (SL-801) (XPO1 inhibitor; Phase 1 in advanced solid tumor patients ongoing) and SL-1001 (RET kinase inhibitor, IND-enabling studies ongoing).

Elcin Barker Ergun, CEO of Menarini Group, commented, "We are very excited to complete the acquisition of Stemline and to welcome their accomplished team to Menarini. The addition of ELZONRIS, which has potential to treat many other malignancies, as well as the other attractive pipeline assets augments our research and development capabilities and will accelerate our efforts to deliver novel oncology therapeutics to patients in need."

About ELZONRIS

ELZONRIS (tagraxofusp), a targeted therapy directed to CD123, is approved by the U.S. Food and Drug Administration (FDA) and commercially available in the U.S. for the treatment of adult and pediatric patients, two years or older, with BPDCN. For full prescribing information in the U.S., visit www.ELZONRIS.com. In Europe, a marketing authorization application (MAA) is under review by the European Medicines Agency (EMA).

ELZONRIS is also being evaluated in additional clinical trials in other CD123+ indications, including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), acute myeloid leukemia (AML), and others are planned including a CD123+ all-comers trial.

About CD123

CD123 is a cell surface target expressed on a wide range of malignancies including blastic plasmacytoid dendritic cell neoplasm (BPDCN), certain myeloproliferative neoplasms (MPNs) including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML) (and potentially enriched in certain AML subsets), myelodysplastic syndrome (MDS), and chronic myeloid leukemia (CML). CD123 has also been reported on multiple myeloma (MM), acute lymphoid leukemia (ALL), hairy cell leukemia (HCL), Hodgkin’s lymphoma (HL), and certain Non-Hodgkin’s lymphomas (NHL). In addition, CD123+ cells have been detected in the tumor microenvironment of several solid tumors as well as in certain autoimmune disorders including cutaneous lupus and scleroderma.

About BPDCN

BPDCN, formerly blastic NK-cell lymphoma, is an aggressive hematologic malignancy, often with cutaneous manifestations, with historically poor outcomes. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, as well as other markers. The World Health Organization (WHO) termed this disease "BPDCN" in 2008; previous names included blastic NK cell lymphoma and agranular CD4+/CD56+ hematodermic neoplasm. For more information, please visit the BPDCN disease awareness website at www.bpdcninfo.com.