On January 7, 2022 NOXXON Pharma N.V. (Euronext Growth Paris: ALNOX) (Paris:ALNOX), a biotechnology company focused on improving cancer treatments by targeting the tumor microenvironment (TME), reported that the Data Safety Monitoring Board (DSMB) positively evaluated safety data from the initial four weeks of treatment of the first patient enrolled in the GLORIA clinical trial expansion arm with NOX-A12 combined with radiotherapy and bevacizumab (Press release, NOXXON, JAN 7, 2022, View Source [SID1234598421]). The DSMB concluded that it is safe and appropriate to continue recruitment of five additional remaining patients into this arm according to the study protocol. NOXXON also announced that the German Federal Institute for Drugs and Medical Devices (BfArM, Bundesinstitut für Arzneimittel und Medizinprodukte) approved the third expansion arm of the GLORIA clinical trial in which patients will receive the PD-1 immune checkpoint inhibitor pembrolizumab in combination with NOX-A12 and radiotherapy.

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Aram Mangasarian, CEO of NOXXON, commented: "The three arms in the expansion of our Phase 1/2 study of NOX-A12 are supported by the clinical data from the GLORIA trial and are designed to explore the potential for improved benefits for patients with brain tumors. The combination with the anti-PD-1 inhibitor pembrolizumab is of particular interest as we expect this combination therapy to unlock a stronger and more durable immune response against the tumor. This is based on our previous observation that NOX-A12 drives infiltration of activated cytotoxic immune cells into brain tumor tissue. We plan to use clinical data from the expansion arms to support our future pivotal glioblastoma study."

The GLORIA Phase 1/2 clinical trial evaluates the safety and efficacy of NOX-A12 combined with radiotherapy in newly diagnosed brain cancer (glioblastoma) patients with unmethylated MGMT promoter. Three expansion arms, each intending to enrol six patients, will evaluate the benefit of NOX-A12 in other therapeutic settings:

Arm A: NOX-A12 with radiotherapy in patients with complete tumor resection
Arm B: NOX-A12 with radiotherapy and bevacizumab in patients with incomplete tumor resection
Arm C: NOX-A12 with radiotherapy and pembrolizumab in patients with incomplete tumor resection.

On January 7, 2022 QIAGEN N.V. (NYSE: QGEN; Frankfurt Prime Standard: QIA) reported new additions to the growing number of applications for QIAcuity, its ultrasensitive digital PCR (dPCR) platform that has set new standards by using so-called nanoplates to process samples in two hours rather than the five hours required by other systems (Press release, Qiagen, JAN 7, 2022, View Source [SID1234598420]).

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QIAGEN has entered into two new collaborations that extend the QIAcuity ecosystem:

A collaboration with Atila BioSystems to provide non-invasive prenatal testing (NIPT) solutions to QIAGEN’s dPCR franchise. NIPT requires only a blood sample from the mother, and replaces more invasive testing methods such as amniocentesis that can endanger the fetus and mother.
A co-exclusive licensing and co-marketing agreement with German life-sciences start-up Actome GmbH extends QIAcuity’s reach beyond genomics into proteomics, enabling the quantification of proteins as well as the analyses of interactions between different proteins and between proteins and target genes.
"QIAcuity is expanding into new research fields as a cost-effective and very reliable digital PCR system," said Thomas Schweins, Senior Vice President, Head of the Life Science Business Area at QIAGEN. "The partnership with Actome extends for the first time the use of this technology to protein quantification and protein-protein interaction. Our aim is to make digital PCR and QIAcuity the new standard in PCR. This technology is significantly more robust and precise than traditional real-time PCR. In particular, it enables the quantification of rare genetic events – such as cancer mutations – with very high precision and in absolute terms, even in a high background of wildtype genes. We expect more than half of all traditional PCR applications to eventually move to digital PCR."

QIAcuity disperses a sample over thousands of tiny nanoplate partitions and then simultaneously reads the reaction in each one. This enables it to quantify even the faintest signals from DNA and RNA – and now proteins – as it tests for viruses, bacteria or other disorders, including rare cancer mutations. Its precision has already established QIAcuity as a vital tool in testing wastewater for SARS-CoV-2.

Leveraging QIAGEN’s capabilities in dPCR and also sample preparation, Atila BioSystems has launched the iSAFE Non-Invasive Prenatal Test Kit for use on QIAcuity in singleton pregnancies. The Research Use Only test can detect targets that indicate possible chromosomal anomalies – trisomy 21 associated with Down Syndrome, trisomy 18 associated with Edwards Syndrome, and trisomy 13 associated with Patau Syndrome and fetal fractions. Processing on QIAcuity takes only 2.5 hours compared to five hours for rival devices, and comes with five detection channels, making it the system with the highest multiplexing capabilities for this application.

The partnership with Actome involves a co-marketing agreement and provides QIAGEN with a co-exclusive license for digital PCR applications to use Actome’s patented Protein-Interaction-Coupling (PICO)-Technology on QIAcuity as an ultrasensitive method to measure smallest amounts of proteins and the interaction between proteins as well as between proteins and genes. The PICO-Technology translates complex protein status into DNA barcodes that can be amplified and detected on QIAcuity.

As part of the agreement, QIAGEN has made an undisclosed investment in Actome. The companies will now jointly develop assays for protein quantification, which will complement genomic analysis and help establish a fuller picture of a disease. The technology has the potential to drive the discovery of new biomarkers and the development of new drugs and diagnostics. It is a step on the path to understanding the interactome, all the molecular interactions in a cell, that contribute to diseases.

On January 7, 2022 Kriya Therapeutics, Inc., a fully integrated company pioneering novel technologies and therapeutics in gene therapy, reported that it has acquired Warden Bio, a company developing novel AAV-mediated gene therapies for glycogen storage disorders (GSDs) (Press release, Kriya Therapeutics, JAN 7, 2022, View Source [SID1234598419]). Under the agreement, Kriya has obtained exclusive rights to Warden Bio’s five preclinical gene therapy programs. This acquisition serves as the foundation for Kriya’s Rare Disease Division focused on the discovery and development of gene therapies for rare diseases.

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GSDs are a group of rare disorders caused by distinct single enzyme deficiencies involved in the synthesis or breakdown of glycogen. Warden Bio was established with a goal of comprehensively addressing multiple GSDs without approved therapies based on pioneering gene therapy technologies originally developed at Duke University. Currently, approved therapies are only available for one type of GSD, Pompe disease (GSD II).

"We are incredibly enthusiastic about the compelling research that has led to the development of Warden Bio’s pioneering gene therapies for GSDs," said Shankar Ramaswamy, M.D., Co-Founder and Chief Executive Officer of Kriya. "This acquisition and the establishment of our Rare Disease Division align with our commitment to accelerating the advancement of best-in-class gene therapies. Kriya’s technology and manufacturing platforms uniquely position our company to drive meaningful progress to address a group of diseases in desperate need of new and better treatments."

Together with the acquisition, Warden Bio co-founder Kunal Kishnani is joining Kriya as President of its Rare Disease Division, where he will lead overall strategic, development, and partnership activities. Prior to founding Warden Bio, Mr. Kishnani was a member of the corporate development team at Roivant Sciences, where he was responsible for helping to evaluate and negotiate new investment opportunities and strategic alliances. Mr. Kishnani also previously served as Co-Founder and Head of Operations at Genevant, where he was instrumental in driving key milestones for the company, including its launch, establishment of business operations, and execution of multiple collaborations with major pharmaceutical companies. Mr. Kishnani has degrees in finance and accounting from Elon University.

"I am excited to join Kriya and help advance its mission of expanding the reach of gene therapy to new frontiers," said Mr. Kishnani. "Despite recent advancements in the rare disease field, many conditions remain significantly undertreated. There is an urgent need to address rare diseases such as GSDs, and we are committed to making the treatment of these patients our priority – both previously at Warden Bio and now at Kriya."

The new GSD gene therapy programs Warden Bio brings to Kriya are based on research led by the lab of Dr. Priya Kishnani, Chen Family Distinguished Professor & Division Chief for Medical Genetics at Duke University, in close collaboration with Dr. Aravind Asokan, Professor & Director of Gene Therapy at Duke University and Co-Founder of StrideBio. Over the past three decades, Dr. Kishnani has built an interdisciplinary team with world-leading GSD expertise whose efforts are most notably demonstrated by the long-standing research and clinical experience that resulted in FDA approval of alglucosidase alfa (Myozyme/Lumizyme) as the first available treatments for Pompe disease. Dr. Kishnani and Dr. Asokan will become Scientific Advisors to Kriya as the company advances its portfolio of gene therapies for GSDs and other diseases.

On January 7, 2022 Cytokinetics, Incorporated (Nasdaq: CYTK) and Royalty Pharma plc (Nasdaq: RPRX) reported that Cytokinetics has secured long-term capital from Royalty Pharma to support the potential commercialization of omecamtiv mecarbil and the further development of aficamten (Press release, Cytokinetics, JAN 7, 2022, View Source [SID1234598418]). In addition, Cytokinetics sold to Royalty Pharma royalties on future worldwide sales of aficamten .

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Omecamtiv mecarbil is an investigational, selective, small molecule cardiac myosin activator, that was granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) and was the subject of GALACTIC-HF, a positive Phase 3 clinical trial in patients with heart failure with reduced ejection fraction. Aficamten is an investigational selective, small molecule cardiac myosin inhibitor that is under investigation for hypertrophic cardiomyopathy and was granted Breakthrough Therapy Designation by the U.S. FDA.

"These corporate development transactions continue our history of monetizing our leadership in muscle biology through creative deal making and provide further optionality, capital efficiencies and financial resources to expand our cardiovascular development and commercialization programs," said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. "We are pleased to enter into these additional transactions with Royalty Pharma, underscoring our shared commitment to build an industry leading cardiovascular franchise in cardiac myosin modulation."

"We are pleased to expand our partnership with Cytokinetics," said Pablo Legorreta, Royalty Pharma’s Founder and Chief Executive Officer. "Royalty Pharma is excited to support the company at this important stage of transformation towards a commercial organization with an exciting late stage pipeline addressing unmet medical needs in multiple cardiovascular diseases."

Royalty Pharma will provide Cytokinetics long-term capital of up to $300 million to support the potential commercialization of omecamtiv mecarbil and the further development of aficamten, and other general corporate purposes. The capital is available in five tranches, including an initial tranche of $50 million upon closing and four additional tranches in the aggregate amount of $250 million upon the occurrence of certain regulatory and clinical development milestones related to omecamtiv mecarbil and aficamten. Each tranche has an interest-free and payment-free period of six calendar quarters, followed by 34 calendar quarters of installment re-payments totaling 1.9 times the amount drawn.

In addition, Royalty Pharma has purchased from Cytokinetics a royalty on aficamten of 4.5% on sales up to $1 billion and 3.5% on sales above $1 billion, subject to certain potential step-downs, in exchange for payments of up to $150 million, comprised of $50 million at closing and two additional $50 million payments, conditional upon the initiation of potential pivotal clinical trials for oHCM and nHCM, respectively.

From these transactions, Cytokinetics anticipates receipt of up to $150 million in near-term funding. Together with its proforma cash at the end of 2021 which was bolstered by transactions executed by Cytokinetics last year, this funding from Royalty Pharma enables Cytokinetics to have at least two years of cash runway based on expected 2022 expenditures, inclusive of planned commercialization activities and expanded pipeline development programs.

Cooley LLP and Morrison Foerster acted as legal advisors to Cytokinetics on the transactions. Goodwin Procter, Fenwick & West LLP, Dechert and Maiwald acted as legal advisors to Royalty Pharma.

Conference Call and Webcast Information

Members of Cytokinetics’ senior management team will host a conference call and webcast today at 8:30 AM Eastern Time. The webcast can be accessed through the Investors & Media section of the Cytokinetics website at www.cytokinetics.com. The live audio of the conference call can also be accessed by telephone by dialing either (866) 999-CYTK (2985) (United States and Canada) or (706) 679-3078 (international) and typing in the passcode 5478696.

An archived replay of the webcast will be available via Cytokinetics’ website until January 21, 2022. The replay will also be available via telephone by dialing (855) 859-2056 (United States and Canada) or (404) 537-3406 (international) and typing in the passcode 5478696 from January 7, 2022 at 11:30 AM Eastern Time until January 21, 2022.

About Omecamtiv Mecarbil

Omecamtiv mecarbil is an investigational, selective, small molecule cardiac myosin activator, the first of a novel class of myotropes1 designed to directly target the contractile mechanisms of the heart, binding to and recruiting more cardiac myosin heads to interact with actin during systole. Omecamtiv mecarbil is designed to increase the number of active actin-myosin cross bridges during each cardiac cycle and consequently augment the impaired contractility that is associated with heart failure with reduced ejection fraction (HFrEF). Preclinical research has shown that omecamtiv mecarbil increases cardiac contractility without increasing intracellular myocyte calcium concentrations or myocardial oxygen consumption.2-4

The development program for omecamtiv mecarbil is assessing its potential for the treatment of HFrEF. Positive results from GALACTIC-HF, the first Phase 3 clinical trial of omecamtiv mecarbil demonstrated a statistically significant effect of treatment with omecamtiv mecarbil to reduce risk of the primary composite endpoint of cardiovascular (CV) death or heart failure events (heart failure hospitalization and other urgent treatment for heart failure) compared to placebo in patients treated with standard of care. No reduction in the secondary endpoint of time to CV death was observed. Adverse events and treatment discontinuation of study drug were balanced between treatment arms. METEORIC-HF, a second Phase 3 clinical trial of omecamtiv mecarbil is designed to evaluate the effect of treatment with omecamtiv mecarbil compared to placebo on exercise capacity. Results from METEORIC-HF are expected in early 2022.

About Aficamten

Aficamten is an investigational selective, small molecule cardiac myosin inhibitor discovered following an extensive chemical optimization program that was conducted with careful attention to therapeutic index and pharmacokinetic properties and as may translate into next-in-class potential in clinical development. Aficamten was designed to reduce the number of active actin-myosin cross bridges during each cardiac cycle and consequently suppress the myocardial hypercontractility that is associated with hypertrophic cardiomyopathy (HCM). In preclinical models, aficamten reduced myocardial contractility by binding directly to cardiac myosin at a distinct and selective allosteric binding site, thereby preventing myosin from entering a force producing state.

The development program for aficamten is assessing its potential as a treatment that improves exercise capacity and relieves symptoms in patients with HCM as well as its long-term effects on cardiac structure and function. Cytokinetics is currently conducting start-up activities for SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM), the Phase 3 clinical trial of aficamten in patients with symptomatic obstructive HCM. The company is also planning for an expanded development program that may evaluate aficamten in indications such as non-obstructive HCM and heart failure with preserved ejection fraction (HFpEF).

On January 7, 2022 Monopar Therapeutics Inc. (Nasdaq: MNPR), a clinical-stage biopharmaceutical company focused on developing proprietary therapeutics designed to extend life or improve the quality of life for cancer patients, reported that Chandler Robinson, MD, Monopar’s Chief Executive Officer, will present at the H.C. Wainwright BioConnect Conference (Press release, Monopar Therapeutics, JAN 7, 2022, https://ir.monopartx.com/news/detail/48/monopar-to-present-at-the-h-c-wainwright-bioconnect-conference [SID1234598417]). The presentation will be available for on-demand viewing starting on Monday, January 10, 2022 at 7:00am ET.

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Presentation Details:

Date: Monday, January 10, 2022

Time: 7:00am ET

Location: Virtual

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