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.

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"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.

On June 14, 2021 Veracyte reported that $600 million to buy Decipher Biosciences and its genomic prostate cancer test (Press release, Veracyte, JUN 14, 2021, View Source [SID1234583986]). Now, that diagnostic has racked up new data showing it can help identify patients most likely to benefit from specific drugs.

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Veracyte picked up urologic cancer specialist Decipher—itself once known as GenomeDx—in March in a cash deal to expand its diagnostic portfolio to seven of the 10 most common tumor types.

Its prostate test is already included in national cancer treatment guidelines, while its kidney and bladder cancer tests are in development, with the latter expected to launch later this year.

The new data—taken from a retrospective analysis of a phase 3 study of men with non-metastatic, castration-resistant prostate cancer, or nmCRPC, and published in JAMA Oncology—showed the test could use genomic data to judge which patients are most likely to see gains from early treatment with Janssen’s Erleada (apalutamide) alongside androgen-deprivation therapy.

RELATED: Veracyte drops $600M to acquire cancer tester Decipher Biosciences

The study is "the first clinical evaluation and demonstration of the Decipher test’s utility in the nmCRPC setting," said Veracyte’s senior VP for scientific and clinical operations in urologic cancers, Elai Davicioni, who added that the data helps demonstrate the test’s usefulness in multiple types of prostate cancer.

Previously, the study showed that Erleada combined with ADT could extend overall survival by 14 months and cut the risk of death by 22%. It also showed improvements in metastasis-free survival and other secondary endpoints. Annually, nmCRPC is estimated to affect about 60,000 men in the U.S.

The Decipher diagnostic relies on gene expression profiling to split patients into groups with low, average and high risks of metastasis, as well as by their tumor’s genetic subtype—with those with higher risks seeing the greatest benefits from Erleada therapy.

RELATED: J&J won’t pursue Erleada, Zytiga prostate cancer combo after trial results disappoint

The results "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," said the paper’s primary author, Felix Feng, vice chair for translational research in the department of radiation oncology at the University of California, San Francisco.

In addition, the molecular signature can help guide treatment decisions for years after an initial diagnosis, once the cancer has advanced locally, the company said.

RELATED: ASCO (Free ASCO Whitepaper): New data set up Veracyte’s nasal swab lung cancer test for launch later this year

The Decipher genomic classifier is currently being tested in seven randomized phase 3 prostate cancer trials sponsored by the National Cancer Institute, plus 13 more phase 2/3 trials, as well as more than 20 retrospective studies of phase 3 data, according to Veracyte.

On June 14, 2021 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported that management will participate in three virtual investor conferences including SVB Leerink CybeRx Series: "Liver Disease Day" Conference taking place June 17, 2021, the Raymond James Human Health Innovation Conference taking place June 22, 2021 and the Piper Sandler "EASL Takeaway Day" taking place June 28, 2021 (Press release, CymaBay Therapeutics, JUN 14, 2021, View Source [SID1234583952]).

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SVB Leerink CybeRx Series: "Liver Disease Day" Conference
Date: Thursday, June 17
Time: 1-on-1 meetings only

Raymond James Human Health Innovation Conference
Date: Monday, June 21
Time: 9:20 am Eastern Time
Panel Discussion: "Itching for New Therapies in Cholestatic Liver Disease"

Piper Sandler "EASL Takeaway Day"
Date: Monday, June 28
Time: 3:00 pm Eastern Time / Fireside Chat
Webcast: View Source

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.

On June 14, 2021 Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, reported that two poster presentations of new clinical data and analyses related to imetelstat, the Company’s first-in-class telomerase inhibitor, are now available on Geron’s website as well as to participants of the EHA (Free EHA Whitepaper)2021 Virtual Congress (Press release, Geron, JUN 14, 2021, View Source [SID1234583987]).

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"These poster presentations further support imetelstat’s differentiated approach to potentially target the malignant stem and progenitor cells in the bone marrow by inhibiting telomerase activity," said Aleksandra Rizo, M.D., Ph.D., Geron’s Chief Medical Officer. "Through this novel mechanism of action, imetelstat has the potential to alter the course of MDS and MF which distinguishes it from other treatments currently approved or in development. We look forward to confirming these results in our ongoing Phase 3 clinical trials, IMerge Phase 3 in lower risk MDS and IMpactMF in refractory MF."

Title: Efficacy of Imetelstat is Independent of Molecular Subtypes in Heavily Transfused Non-Del(5q) Lower Risk MDS (LR-MDS) Relapsed/Refractory (R/R) to Erythropoiesis Stimulating Agents (ESA)
Poster Code: EP910

New data and analyses were presented on the clinical efficacy of imetelstat in molecularly defined subtypes based on cytogenetic and mutation profiles for patients in the IMerge Phase 2 clinical trial. As reported at previous EHA (Free EHA Whitepaper) meetings, meaningful and durable transfusion independence were observed in patients from IMerge Phase 2, including transfusion-free periods greater than one year, as well as substantial increases in hemoglobin. The current presentation reported clinical responses across different cytogenetic and molecularly defined categories whereby responses were independent of mutation status or number of mutations. These data support the unique telomerase inhibition mechanism of action of imetelstat and the potential to target the malignant stem and progenitor cells of the underlying disease.

Title: Imetelstat Demonstrates an Acceptable Safety Profile in Myeloid Malignancies
Poster Code: EP1106

Safety data from the Phase 2 IMbark and IMerge trials were further analyzed to understand the characteristics of hematologic and non-hematologic adverse events. These analyses highlighted that the imetelstat-related cytopenias are short, reversable and with limited clinical consequence when managed with the dose modification guidelines in the protocols. These data are further evidence for the on-target effect of imetelstat based on the selective reduction of malignant cells in the bone marrow through telomerase inhibition resulting in the observed meaningful clinical benefits for patients in the Phase 2 trials.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Data from Phase 2 clinical trials provide strong evidence that imetelstat targets telomerase to inhibit the uncontrolled proliferation of malignant stem and progenitor cells in hematologic myeloid malignancies resulting in malignant cell apoptosis and potential disease-modifying activity. Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for both the treatment of patients with non-del(5q) lower risk MDS who are refractory or resistant to an erythropoiesis-stimulating agent and for patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus kinase (JAK) inhibitor treatment.