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OMEROS’ NARSOPLIMAB PIVOTAL TRIAL DATA SHARED IN ORAL PRESENTATION AT 2021 ANNUAL CONGRESS OF THE EUROPEAN HEMATOLOGY ASSOCIATION

On June 14, 2021 Omeros Corporation (Nasdaq: OMER) reported that data on organ function improvement from its pivotal trial of narsoplimab for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) were shared during an oral presentation at the virtual edition of the 26th Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) (Press release, Omeros, JUN 14, 2021, View Source [SID1234583968]). The presentation, entitled Narsoplimab (OMS721) Treatment Contributes to Improvements in Organ Function in Adult Patients with High-Risk Transplant-Associated Thrombotic Microangiopathy, was delivered last Friday by Miguel-Angel Perales, M.D., Chief of Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center. The organ function improvement data presented underscore the potential of narsoplimab as a significant advance in the treatment of often fatal HSCT-TMA.

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The trial’s findings include:

The study population was high-risk, with 93 percent having multiple risk factors for poor outcomes, and highly reflective of "real-world" clinical practice
At baseline:
75% of patients had kidney dysfunction
57% had neurologic dysfunction
18% had pulmonary dysfunction
50% had multiple organ TMA involvement
86% had significant infection
68% had graft versus host disease (GVHD)
61% of the intent-to-treat (ITT) population (any patient receiving at least 1 dose of narsoplimab) and 74% of the per-protocol (PP) population (those patients receiving ≥ 4 weeks of dosing) responded to narsoplimab based on improvement in laboratory TMA markers (platelet count improvement and reduction in LDH levels) and clinical status (organ function or freedom from transfusion)
74% of eligible patients in the ITT population experienced improvement in organ function (67%, 50% and 100% in kidney, neurologic, or gastrointestinal function, respectively); 77% of eligible patients in the PP population experienced organ function improvement
48% of eligible patients in the ITT population and 55% in the PP population experienced freedom from transfusion
Narsoplimab was well tolerated in this very sick population
The most common adverse events were pyrexia, diarrhea, vomiting, nausea, neutropenia, fatigue, and hypokalemia, all common in HSCT
Six patients died during the core study period due to causes common in HSCT
There were no study discontinuations due to non-fatal adverse events
Detailed data and findings from the study are being submitted to a peer-reviewed scientific journal for publication.

Dr. Perales’ question-and-answer panel discussion for his presentation is scheduled for Tuesday, June 15, at 1:00-1:45 pm CEST/7:00-7:45 am EDT and will be available to registered attendees through the EHA (Free EHA Whitepaper) Congress virtual platform.

In severe cases of HSCT-TMA, mortality can exceed 90 percent and, even in those who survive, significant morbidity is common with chronic organ injury often persisting. There is no approved treatment for HSCT-TMA. A Biologics License Application for use of narsoplimab in the treatment of HSCT-TMA is under Priority Review by the US Food and Drug Administration (FDA) with a Prescription Drug User Fee Act action date of October 17, 2021.

About Hematopoietic Stem Cell Transplant-associated Thrombotic Microangiopathy

Hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) is a significant and often lethal complication of stem cell transplantation. This condition is a systemic, multifactorial disorder caused by endothelial cell damage induced by conditioning regimens, immunosuppressant therapies, infection, graft-versus-host disease, and other factors associated with stem cell transplantation. Endothelial damage, which activates the lectin pathway of complement, plays a central role in the development of HSCT-TMA. The condition occurs in both autologous and allogeneic transplants but is more common in the allogeneic population. In the United States and Europe, approximately 25,000 to 30,000 allogeneic transplants are performed annually. Recent reports in both adult and pediatric allogeneic stem cell transplant populations have found an approximately 40-percent incidence of HSCT-TMA, and high-risk features may be present in up to 80 percent of these patients. In severe cases of HSCT-TMA, mortality can exceed 90 percent and, even in those who survive, long-term renal sequalae (e.g., dialysis) are common. There is no approved therapy or standard of care for HSCT-TMA.

About Narsoplimab

Narsoplimab, also known as "OMS721," is an investigational human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), a novel pro-inflammatory protein target and the effector enzyme of the lectin pathway of complement. Importantly, inhibition of MASP-2 does not appear to interfere with the antibody-dependent classical complement activation pathway, which is a critical component of the acquired immune response to infection. Omeros controls the worldwide rights to MASP-2 and all therapeutics targeting MASP-2.

A biologics license application (BLA) is under priority review by the U.S. FDA for use of narsoplimab in the treatment of HSCT-TMA, and the drug is in Phase 3 clinical programs for immunoglobulin A (IgA) nephropathy and atypical hemolytic uremic syndrome (aHUS). Narsoplimab is also being evaluated for the treatment of COVID-19 as part of the I-SPY-COVID-19 platform trial sponsored by Quantum Leap Healthcare Collaborative. The FDA has granted narsoplimab breakthrough therapy designations for HSCT-TMA and for IgA nephropathy; orphan drug status for the prevention (inhibition) of complement-mediated thrombotic microangiopathies, for the treatment of HSCT-TMA and for the treatment of IgA nephropathy; and fast track designation for the treatment of patients with aHUS. The European Medicines Agency has granted orphan drug designation to narsoplimab for treatment in HSCT and for treatment of primary IgA nephropathy.

Dr. Paul Burton to Join Moderna as Chief Medical Officer

On June 14, 2021 Moderna, Inc., (Nasdaq: MRNA) a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, reported that Paul Burton, M.D., Ph.D., F.A.C.C, M.R.C.S will join the Company as Chief Medical Officer, effective July 6, 2021 (Press release, Moderna Therapeutics, JUN 14, 2021, View Source [SID1234583967]). He will serve on Moderna’s Executive Committee and report to Chief Executive Officer Stéphane Bancel.

"Paul’s extensive medical experience in the global pharmaceutical and biotech industry will be important to Moderna as we expand internationally and continue our journey as a commercial company," said Stéphane Bancel, Chief Executive Officer of Moderna. "As we work to deliver on the promise of mRNA science to create a new generation of transformative medicines for patients, Paul’s expertise across multiple therapeutic areas and his proven track record leveraging data science and digital technologies to reimagine medical engagement will be invaluable. I look forward to working with Paul and re-inventing how medical affairs should be built and run in a digital world."

Dr. Burton joins Moderna after spending sixteen years with Johnson & Johnson (NYSE: JNJ). Since March 2020, he served as Chief Global Medical Affairs Officer of Janssen Pharmaceuticals where he was responsible for Janssen’s worldwide medical affairs strategy and execution. Previously, he served as Janssen’s Vice President and Head, Cardiovascular and Metabolic (CVM) Medical Affairs. Dr. Burton also led the Johnson & Johnson collaboration with APPLE for the digital HEARTLINE study, and previously led clinical operations for all therapeutic areas across the Americas.

"Moderna’s transformative platform has the potential to improve the lives of so many people around the world," said Dr. Burton. "I am honored to take on the role of Chief Medical Officer at Moderna."

Dr. Burton has an M.D. from the University of London, is board certified in surgery and is a Member of the Royal College of Surgeons with specialist training in cardiothoracic surgery. He holds a Ph.D. in cardiovascular molecular and cellular biology from Imperial College in London and is a Fellow of the American College of Cardiology. He has published extensively in peer reviewed journals.

Earlier in 2021, the Company announced that Moderna’s Chief Medical Officer Tal Zaks, M.D., Ph.D., will be leaving the Company after six years of service.

"As Paul prepares to join us, I would like to thank Tal once again for his impact over the last six years. Tal’s guidance and contributions were important in helping Moderna move from a preclinical company to where we are today. Through his leadership in Moderna’s response to the COVID-19 pandemic, Tal’s contribution extends beyond Moderna to all of society. I have enjoyed having him as my partner and wish him all the best," said Stéphane Bancel.

Leap Therapeutics to Present at Raymond James 2021 Virtual Human Health Innovation Conference

On June 14, 2021 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, reported that Douglas E. Onsi, President and Chief Executive Officer, will present a corporate overview at the Raymond James 2021 Virtual Human Health Innovation Conference on Monday, June 21, 2021 at 1:20 p.m. Eastern Time (Press release, Leap Therapeutics, JUN 14, 2021, View Source [SID1234583966]).

The presentation will be webcast live and may be accessed on the Investors page of the company’s website at View Source, where a replay of the event will also be available for a limited time.

Allarity Therapeutics and Oncoheroes Biosciences to Partner on Pediatric Cancer Development of Dovitinib and Stenoparib

On June 14, 2021 Allarity Therapeutics A/S ("Allarity") and Oncoheroes Biosciences, Inc. ("Oncoheroes") reported that they have entered into binding term sheets for agreements under which Oncoheroes will acquire certain rights to dovitinib, a pan-targeted kinase inhibitor (pan-TKI), and stenoparib, a PARP inhibitor, and assume responsibility for their further clinical development in pediatric cancer (Press release, Allarity Therapeutics, JUN 14, 2021, View Source [SID1234583965]).

Allarity is preparing for the submission, to the U.S. Food and Drug Administration (FDA), of an NDA for marketing approval for dovitinib as a treatment for RCC later this year. Earlier this year, in April, the Company submitted the premarket approval (PMA) application for use of its Dovitinib-DRP companion diagnostic to select and treat patients most likely to respond to the drug. In support of its NDA filing, and in accordance with FDA requirements, the Company is also planning a clinical trial in pediatric patients with osteosarcoma, in partnership with Oncoheroes, where the patients will be selected with the Dovitinib-DRP companion diagnostic. Allarity’s focus on pediatric osteosarcoma development is based on the results of two previously reported preclinical studies in which treatment with dovitinib, compared to control treatment (sucrose solution lacking dovitinib), increased the median survival time of mouse models of osteosarcoma by 50% and antitumor growth activity was observed for dovitinib as a single agent.

Allarity is also currently evaluating stenoparib for the treatment of advanced ovarian cancer in a Phase 2 clinical trial at the Dana-Farber Cancer Institute (Boston, MA U.S.A.) using the Stenoparib-DRP companion diagnostic to guide patient enrollment and improve therapeutic outcome. In prior clinical testing of more than 60 patients, stenoparib was well tolerated with a demonstrated acceptable safety profile. Through use of DRP patient selection, Allarity aims to provide a superior clinical benefit to ovarian cancer patients receiving stenoparib as compared to other approved PARP inhibitors. Moreover, there is increasing evidence pointing to the possible use of PARP inhibitors in the treatment of various pediatric cancers.

Steve R. Carchedi, CEO of Allarity Therapeutics, commented, "This is a promising opportunity to work with Oncoheroes Biosciences, a childhood cancer-focused organization, which will allow the two companies to build together on Allarity’s prior efforts to advance both dovitinib and stenoparib by further exploring the clinical potential of these drugs for the specific treatment of pediatric cancers. We are very pleased to partner with Oncoheroes on these pediatric cancer programs to help address historically underserved, rare childhood cancers. Our out-licensing of these pediatric development programs enables Allarity to remain focused on our top priority programs in adult cancers, while at the same time leveraging Oncoheroes’ resources, capabilities, and commitment to clinically advancing dovitinib and stenoparib in childhood cancers."

Under the terms outlined in the binding terms sheets, Oncoheroes will acquire global, exclusive rights to fund and conduct further clinical development of both dovitinib and stenoparib in pediatric cancer. The complete agreements are conditioned upon Oncoheroes allocating specific funds, within a specified timeframe, to conduct the contemplated pediatric clinical development. Under the dovitinib term sheet, Oncoheroes will take responsibility for pediatric cancer clinical development activities. Upon successful completion of the studies, Allarity will reimburse Oncoheroes for clinical development costs plus a fixed profit margin and pay an undisclosed milestone upon first regulatory approval for a pediatric cancer indication. Oncoheroes will not receive any pediatric field commercialization rights unless Allarity elects not to further develop the drug for pediatric indications. Under the stenoparib term sheet, Oncoheroes will receive commercialization rights for pediatric cancers, subject to Allarity’s first buy-back option, and Allarity will receive an undisclosed upfront license fee. If Allarity does not re-acquire the pediatric field rights, it will further receive certain clinical/regulatory milestone payments and royalties on sales of stenoparib in the pediatric cancer market from Oncoheroes. Allarity will support Oncoheroes’ pediatric clinical trials by providing clinical-grade drug inventory at cost and by facilitating DRP companion diagnostic screening of patients for each drug. Further financial terms of the term sheets were not disclosed.

Cesare Spadoni, COO & Founder of Oncoheroes Biosciences, further commented, "Oncoheroes is a mission-driven company committed to deliver more effective and safer treatments for children and adolescents with cancer. We are excited to partner with Allarity on these co-development programs. These fit perfectly with our goal of becoming the partner of choice for life sciences companies with drug candidates that have the potential to treat pediatric cancers. We are confident that this collaborative model will create powerful synergies to accelerate pediatric drug development and bring tangible benefits to younger cancer patients.

New Data Published in JAMA Oncology Demonstrate Prognostic Utility of Veracyte’s Decipher Prostate Genomic Classifier in Locally Advanced Prostate Cancer

On June 14, 2021 Veracyte, Inc. (Nasdaq: VCYT) reported new data demonstrating the prognostic utility of the company’s Decipher Prostate genomic classifier among men with non-metastatic castration-resistant prostate cancer (nmCRPC) have been published online in JAMA Oncology (Press release, Veracyte, JUN 14, 2021, View Source [SID1234583964]). The findings, from a retrospective analysis of patients in the Phase 3 SPARTAN study, suggest that the Decipher test can help identify those patients most likely to benefit from treatment with apalutamide, a second-generation androgen receptor signaling inhibitor (ARSi), in addition to androgen-deprivation therapy (ADT).

"These results suggest that the Decipher Prostate test may be a helpful tool to identify those patients who would benefit from early treatment intensification with androgen receptor inhibitors," said Elai Davicioni, Ph.D., Veracyte’s senior vice president, Scientific and Clinical Operations, Urologic Cancers. "As the first clinical evaluation and demonstration of the Decipher test’s utility in the nmCRPC setting, this study adds meaningfully to prior evidence demonstrating the test’s ability to help inform treatment decisions and improve patient outcomes in multiple prostate cancer settings."

SPARTAN is a multicenter, international, randomized, double-blind, placebo-controlled, Phase 3 trial that investigated the efficacy of adding apalutamide to androgen-deprivation therapy (ADT) in comparison with ADT plus placebo among men with nmCRPC. Results from the trial suggest that the addition of apalutamide to ADT significantly improved metastasis free survival (MFS) and other secondary endpoints in these men.

To understand the molecular characteristics driving the SPARTAN study clinical outcomes, researchers used the Decipher genomic classifier to perform gene expression profiling on archived primary tumor samples from a subset of 233 patients enrolled in the trial. The test stratified patient tumors into Decipher high- and low-to-average-risk groups for metastasis and into basal and luminal subtypes.

The newly published results suggest that Decipher test scores and basal-luminal subtype may be biomarkers of response to apalutamide plus ADT in the nmCRPC setting, and that patients whose tumors were classified as Decipher high-risk or luminal subtype derive the greatest benefit from apalutamide therapy.

Specifically, while study results indicate an MFS improvement among all nmCRPC patients who received apalutamide plus ADT, the 116 patients in the subset who had Decipher high-risk scores exhibited the greatest improvement in MFS (HR 0.21; 95% CI, o.11-0.40; P<0.001) and progression-free survival 2 (PFS2; HR, 0.39; 95% CI, 0.23-0.67; P = 0.001) vs. placebo plus ADT. Notably, the addition of apalutamide to ADT improved the MFS percentage among the Decipher high-risk patients to a level similar to the percentage among patients classified as Decipher low-to-average-risk.

Also, among the apalutamide plus ADT group, those patients whose tumors had the luminal subtype (n=81) experienced a significantly longer MFS compared to those with the basal subtype (n=152; median MFS not reached; HR, 0.40; 95% CI, 0.38-1.60; P=0.50).

Researchers noted that Decipher testing was conducted on samples taken from ADT-naïve patients an average of 6.7 years prior to their enrollment in the SPARTAN study. This suggests that the molecular signatures in initial diagnostic samples taken from primary tumors can be informative for making treatment decisions years later when the cancer has locally advanced.

"These findings are an important addition to our growing understanding about how best to manage patients across the long trajectory of prostate cancer," said Felix Y. Feng, MD, professor of Radiation Oncology, Urology, and Medicine, and vice chair for Translational Research, Department of Radiation Oncology at the University of California, San Francisco, who is a SPARTAN study investigator and the paper’s primary author. "They suggest that genomic testing provides useful information to guide treatment decisions that may improve outcomes among men with locally advanced disease, a population for which we’ve previously lacked genomic biomarkers."

The Decipher Prostate genomic classifier is currently being investigated in seven National Cancer Institute-sponsored, Phase 3, prospective, randomized controlled clinical trials; 13 Phase 2/3 prospective trials; and more than 20 retrospective studies of Phase 3 randomized controlled trials. Many of these trials require Decipher Prostate testing for study inclusion.

About Decipher Prostate

Decipher Prostate (Decipher Prostate Biopsy and Decipher Prostate RP) is a 22-gene, whole-transcriptome-developed genomic test intended to help inform treatment decisions for men with localized prostate cancer at initial diagnosis and after surgical removal of the prostate. The test reports the Decipher Score, which prognosticates a patient’s risk of metastasis within five years and provides risk estimates of prostate cancer-specific outcomes. Decipher Prostate can help guide physicians to better select the appropriate therapy for a specific patient, which in turn can result in improved patient outcomes.