On May 28, 2021 HCW Biologics reported that it has filed to raise up to $50 million in an IPO (Press release, HCW Biologics, MAY 28, 2021, View Source [SID1234583286]). The money will set HCW up to take two immunotherapy treatments into the clinic in solid tumors and an autoimmune disorder.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Florida-based HCW licensed rights to use its most advanced candidate, HCW9201, to Wugen, which is using the molecule in the generation of memorylike NK cell products in two phase 2 clinical trials in patients with relapsed or refractory acute myeloid leukemia. With Wugen initiating those studies with Washington University, HCW is working to get its next two drug candidates into the clinic.

The most advanced of the two wholly owned assets is HCW9218, a bifunctional fusion protein that HCW plans to take into a phase 1b/2 pancreatic cancer clinical trial by the end of the year. HCW9218 is designed to optimize the efficacy and minimize the side effects of chemotherapy by activating IL-15R signaling and trapping three TGF-β isoforms.

In its IPO paperwork, HCW said the "approach is unique from most other existing or investigational therapies," although it still expects to face competition from T-cell engagers, CAR-Ts and other drugs "that target specific tumor-associated antigens using immune cells or other cytotoxic modalities."

HCW is running IND-enabling studies of HCW9218 while carrying out earlier-stage work on its next candidate, HCW9302. The second asset is an IL2-based immunotherapeutic protein designed to treat autoimmune disorders such as alopecia areata by driving the expansion of regulatory T cells. HCW is aiming to take HCW9302 into clinical development in the first half of next year.

The biotech raised the money to reach this point across series A, B and C rounds from 2018 to 2020. HCW CEO Hing Wong and his spouse bought stock in the financing rounds, culminating in the pair holding a 51% stake in the company. Medmira Capital and DeepWork HCW Partners are the two other biggest stockholders.

On May 28, 2021 Ryvu Therapeutics (WSE: RVU), a clinical stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology, reported that its Clinical Trial Application (CTA) to commence a single-agent, open-label Phase I/II trial, investigating the safety and efficacy of RVU120 (SEL120) in patients with relapsed/refractory metastatic or advanced solid tumors in Poland, has been fully approved by the Polish Office for Registration of Medicinal Products, Medical Devices and Biocidal Products, and the respective Central Ethics Committee (Press release, Ryvu Therapeutics, MAY 28, 2021, View Source [SID1234583282]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Following the above-mentioned approvals, Ryvu Therapeutics will be able to initiate a clinical study and start enrolling patients in Poland.

The study is designed in two phases. Phase I part has the key objectives of assessing safety and tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary anti-tumor activity of RVU120 (SEL120) during dose escalating cohorts, and determination of the recommended phase II dose (RP2D), and the phase II part, subsequently will include specific tumor indications, enrolled at distinct study groups, such as Triple Negative Breast Cancer (TNBC).

"With approvals from Polish Office for Registration of Medicinal Products, Medical Devices, and Biocidal Products and Central Ethics Committee, we are making another important step in the clinical development of our flagship RVU120 (SEL120) program. We are very excited to develop RVU120 (SEL120) as a potential treatment in both hematological and solid malignancies," comments Setareh Shamsili, MD, PhD, Chief Medical Officer, and EVP at Ryvu Therapeutics.

"We are delighted that the new Phase I/II RVU120 (SEL120) study in patients with solid tumors will be conducted in Poland. Clinical Trial Applications in other European countries will be submitted over the coming months," adds Setareh Shamsili.

About RVU120 (SEL120)

RVU120 (SEL120) is a highly selective first-in-class CDK8/CDK19 inhibitor, which has demonstrated efficacy in a number of solid tumor types in in vitro and in vivo models as well as in onco-hematological malignancies. The first-in-human (FIH) phase I study with RVU120(SEL120), in relapsed or refractory AML or high-risk myelodysplastic syndromes (HRMDS), is currently enrolling patients in 5 investigational sites in USA (View Source).

Current translational data suggest that RVU120 (SEL120) is particularly effective in undifferentiated AML STAT5-positive cancers. Administration of RVU120 (SEL120) in orthotopic AML patient derived xenograft models reduced tumor burden to the level undetectable in the peripheral blood, decreased splenomegaly and resulted in partial bone marrow recovery at well tolerated doses, providing therefore a strong rationale for the clinical development of RVU120 (SEL120) as an effective treatment for AML and potentially other hematological malignancies.

On March 25, 2020, the U.S. Food and Drug Administration (FDA) granted an orphan drug designation (ODD) to RVU120 (SEL120), for the treatment of patients with acute myeloid leukemia (AML).

On April, 2021 U.S. Food and Drug Administration, FDA, placed a partial clinical hold on the first in human Phase Ib, dose escalation clinical trial of RVU120 in patients with relapsed/refractory (R/R) AML and high-risk MDS. Patients who are currently taking RVU120 may continue treatment. Ryvu continues to work closely with the FDA to resolve the partial clinical hold with the objective of resuming enrollment in the study. RVU120 (SEL120) was discovered with the Ryvu Therapeutics discovery engine platform and has received support from The Leukemia & Lymphoma Society Therapy Acceleration Program (TAP), a strategic initiative to partner directly with innovative biotechnology companies and leading research institutions to accelerate the development of promising new therapies for blood cancers. More information about TAP program is available at: View Source

On May 28, 2021 Fluxion Biosciences reported the publication of a paper on ERASE-Seq liquid biopsy with molecular amplification pools (MAPs) (Press release, Fluxion Biosciences, MAY 28, 2021, View Source [SID1234583281]). The paper, "Sensitivity, specificity, and accuracy of a liquid biopsy approach utilizing molecular amplification pools", published in Nature Scientific Reports on May 24, is co-authored by researchers at Fluxion and the Hospices Civils de Lyon Cancer Institute. Data showed the ERASE-Seq/MAPs approach to have a 98.8% concordance to the benchmark droplet digital PCR (ddPCR) approaches.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Liquid biopsies offer the potential to improve treatment of cancer by providing affordable, non-invasive detection of actionable cancer mutations from a blood sample. However, the relative abundance of cancer DNA in blood is extremely low, limiting test sensitivity. Recent studies demonstrate good concordance between liquid biopsies when the cancer DNA exceeds 1% allele fraction (AF), but concordance falls off below 1% AF, where many variants are detected.

In this large-scale study, Fluxion’s ERASE-Seq approach was compared directly to benchmark ddPCR tests for EGFR variants that represent treatable biomarkers or indicate the onset of drug resistance mutations. Patient blood samples were drawn and split between the tests, allowing a direct, blinded head-to-head comparison for both sensitivity and specificity. ERASE-Seq demonstrated excellent concordance in the allele fraction of 0.05%-1%, where other sequencing-based liquid biopsies have reduced performance.

"Although ERASE-Seq is a sequencing-based test, it provides levels of sensitivity normally only associated with droplet digital PCR (ddPCR), an analytical technique that is considered the gold standard for sensitivity. ERASE-Seq can test for thousands of variants in a single sample, where ddPCR is limited to only a few variants per test, which means ERASE-Seq can assay many more actionable markers in one test. This is one of the largest liquid biopsy concordance studies ever conducted, and we’re excited to see that ERASE-Seq performs at this level," Fluxion CEO Jeff Jensen explains.

Fluxion offers Spotlight liquid biopsy panels utilizing the ERASE-Seq variant caller, with panels available for pan-cancer, myeloid, TP53, and others. Additionally, ERASE-Seq is easily incorporated with custom-designed panels based on user-selected genomic targets.

On May 28, 2021 QIAGEN N.V. (NYSE: QGEN; Frankfurt Prime Standard: QIA) reported the launch of an expanded scope of companion diagnostic (CDx) claims for the therascreen KRAS RGQ PCR Kit (therascreen KRAS Kit) after it received U.S. regulatory approval as a companion diagnostic to aid in the identification of non-small cell lung cancer (NSCLC) patients that may be eligible for treatment with LUMAKRASTM (sotorasib), a newly approved therapy developed and marketed by Amgen Inc. (AMGN) (Press release, Qiagen, MAY 28, 2021, View Source [SID1234583280]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The therascreen KRAS Kit is the first companion diagnostic test to obtain premarket approval from the U.S. Food and Drug Administration (FDA) for use to identify the KRAS G12C mutation in samples of NSCLC tumour tissue. KRAS is one of the most frequently occurring mutation in this form of cancer, and is estimated to be present in up to 13% of cases of the disease. Until now KRAS G12C has not been actionable, and in fact had only previously been linked with resistance to therapies. The real-time qualitative PCR kit is used with the Rotor-Gene Q MDx instrument, a member of the modular QIAsymphony family of automation solutions, and builds upon QIAGEN’s nine years of experience in KRAS CDx test development and commercialization.

"We are pleased to announce this significant expansion in the scope of FDA-approved CDx claims for the therascreen KRAS Kit" said Jean-Pascal Viola, Senior Vice President and Head of QIAGEN’s Molecular Diagnostics Business Area. "This new approval further expands our market-leading therascreen portfolio of companion diagnostic tests, and illustrates our determination to support the delivery of the latest innovations in precision healthcare to patients with NSCLC, for whom every new treatment option is extremely welcome."

"With advances in precision medicine, biomarker testing is critical for patients with non-small cell lung cancer because it informs treatment options during the course of their disease. It is important that patients and their healthcare providers know that KRAS G12C is now an actionable mutation and start testing for it," said Darryl Sleep, M.D., chief medical officer and senior vice president of Global Medical at Amgen. "With the approval of QIAGEN’s companion diagnostic for LUMAKRAS, patients and clinicians will have more options and flexibility for biomarker testing."

Up to 13% of NSCLC-patients may have KRAS G12C positive tumours and hence be potentially eligible for treatment with LUMAKRASTM. To accelerate identification of these patients, following the FDA approval of this test QIAGEN is making testing of NSCLC tumour tissue samples with the therascreen KRAS Kit available immediately at leading laboratories across the U.S, through QIAGEN’s Day-One Lab Readiness program for Precision Medicine.

QIAGEN’s therascreen KRAS Kit was used to support the CodeBreaK 100 clinical trial of sotorasib and the expansion of the Kit’s CDx claims to include identification of the KRAS G12C mutation in NSCLC samples has been co-approved with LUMAKRAS by the FDA. The Amgen drug is a new inhibitor of the G12C-mutated form of the KRAS (Kirsten rat sarcoma) protein, and is the first-in-class drug approved for treatment of this form of cancer. Further details about the Kit are available at www.qiagen.com/KRAS.

QIAGEN’s Day-One Lab Readiness program builds on the FDA’s modernized regulatory approach to benefit patients by accelerating the launch of advanced diagnostics. An updated list of U.S. laboratories offering testing of NSCLC samples for the KRAS G12C mutation using the therascreen KRAS test is available at www.qiagen.com/KRAS-lab-finder.

QIAGEN is a pioneer in Precision Medicine and the global leader in collaborations with pharmaceutical and biotechnology companies to co-develop companion diagnostics, which detect clinically relevant genetic abnormalities to provide insights that guide clinical decision-making in diseases such as cancer. QIAGEN has an unmatched depth and breadth of technologies from next-generation sequencing (NGS) to polymerase chain reaction (PCR) for companion diagnostic development. QIAGEN now has ten PCR based companion diagnostic indications that are FDA approved, including therascreen EGFR for non-small cell lung cancer, therascreen KRAS for colorectal cancer, therascreen FGFR for urothelial cancer, therascreen PIK3CA for breast cancer based on tissue or plasma samples and the therascreen BRAF kit for colorectal cancer.

Currently, QIAGEN is working under master collaboration agreements with more than 25 companies to develop and commercialize companion diagnostic tests for their drug candidates – a deep pipeline of potential future products to advance Precision Medicine for the benefit of patients. The therascreen KRAS Kit co-approval with LUMAKRASTM marks the tenth FDA approval of a therapy partnered with a QIAGEN companion diagnostic assay.

On May 28, 2021 Guardant Health, Inc. (Nasdaq: GH) reported that the U.S. Food and Drug Administration (FDA) has approved the Guardant360 CDx test as the first and only liquid biopsy companion diagnostic for tumor mutation profiling, or comprehensive genomic profiling, to identify patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) who harbor the KRAS G12C mutation and may benefit from LUMAKRAS (sotorasib), an FDA-approved KRASG12C inhibitor developed and manufactured by Amgen (Press release, Guardant Health, MAY 28, 2021, View Source [SID1234583279]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Guardant360 CDx FDA approval was based on clinical validation data from the CodeBreaK 100 trial evaluating sotorasib in patients with locally advanced or metastatic NSCLC. Patients identified with the KRAS G12C mutation using the Guardant360 CDx demonstrated an objective response rate consistent with those identified using traditional tissue-based biomarker testing.

Lung cancer is the leading cause of cancer death in the U.S.1 and NSCLC accounts for approximately 84 percent of all lung cancers.2 It is estimated that 66 percent of patients with NSCLC have advanced or metastatic disease at initial diagnosis,3 and two out of three with lung adenocarcinoma harbor a driver mutation.4 Clinical guidelines recommend comprehensive genomic profiling at diagnosis, for all patients with advanced NSCLC, to evaluate whether they have one of the growing list of actionable and emerging biomarkers with associated treatment options.5-7 KRAS G12C is one of the most common driver mutations in NSCLC, occurring in 13 percent of patients, and until now, FDA-approved targeted therapy options did not exist.4,8

"The approval of LUMAKRAS represents a significant medical advancement for patients with advanced non-small cell lung cancer who harbor the KRAS G12C mutation because it is the first and only targeted therapy now available to them," said Darryl Sleep, M.D., Amgen chief medical officer and senior vice president of Medical Affairs "However, patients can only benefit from targeted therapies, or personalized treatments, if they are tested for biomarkers. Today’s FDA approval of Guardant360 CDx, offers an important development in biomarker testing by providing a high-quality, blood-based testing option for patients."

"In the CodeBreaK 100 phase 2 clinical trial, which was the basis for the FDA approval, sotorasib demonstrated compelling efficacy and tolerability in patients with KRAS G12C-mutated non-small cell lung cancer. This approval represents a historic milestone for patients with this mutation," said Vamsidhar Velcheti, M.D., director of thoracic oncology at NYU Langone Health Perlmutter Cancer Center. "This new targeted therapy, reinforces once again why comprehensive biomarker testing at diagnosis is critical. Having additional options, including the availability of a blood-based testing option, such as the Guardant360 CDx, will help to more quickly identify the patients who may benefit and help guide treatment decisions."

"This groundbreaking new therapy from Amgen, LUMAKRAS, underscores the importance of incorporating comprehensive genomic profiling in routine clinical practice to ensure all patients are evaluated for KRAS G12C and the growing list of other actionable mutations that can be treated with targeted therapies shown to significantly improve clinical outcomes," said Helmy Eltoukhy, Guardant Health CEO. "By offering an FDA-approved companion diagnostic that can quickly deliver comprehensive results from a simple blood test, clinicians can have greater confidence using the test, and patients benefit from less invasive testing and shorter wait times to see whether they are eligible for a targeted therapy such as LUMAKRAS."

For oncologists, the FDA-approved Guardant360 CDx provides comprehensive genomic results from a simple blood draw in seven days, helping them move beyond the limitations of tissue biopsies to rapidly obtain clinically relevant information in time to match patients to the optimal personalized treatment. Guardant360 CDx covers all genes recommended by the National Comprehensive Cancer Network, including those most relevant to clinical care and NSCLC treatment guidelines.

Since being introduced, the Guardant360 test has become widely accepted for blood-based comprehensive genomic profiling with more than 200 peer-reviewed publications. It has been trusted by more than 9,000 oncologists, with more than 150,000 tests performed to date, and is broadly covered by Medicare and many private payers, representing over 200 million lives.