On May 14, 2020 Omeros Corporation (Nasdaq: OMER) reported that the results of its pivotal trial of narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy will be presented at the 25th Annual Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) to be held June 11-14, 2020 (Press release, Omeros, MAY 14, 2020, View Source [SID1234558061]). This year the congress will be held virtually .

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The presentation, entitled Narsoplimab (OMS721) for the Treatment of Adult Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy, will be delivered by Alessandro Rambaldi, M.D., Professor of Hematology at the University of Milan and Head of the Hematology and Bone Marrow Transplant Unit at ASST Papa Giovanni XXIII in Bergamo, Italy. Selected by EHA (Free EHA Whitepaper) for a podium presentation, it will include efficacy data not previously presented.

About HSCT-TMA

Hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) is a significant and often lethal complication of stem cell transplants. This condition is a systemic, multifactorial disorder caused by endothelial cell damage induced by conditioning regimens, immunosuppressant therapies, infection, GvHD, 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 HSCT-TMA incidence of approximately 40 percent, 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 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.

Phase 3 clinical programs are in progress for narsoplimab in HSCT-TMA, in immunoglobulin A (IgA) nephropathy, and in atypical hemolytic uremic syndrome (aHUS). 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 May 14, 2020 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported plans to present updated clinical data for pralsetinib in RET-altered cancers and avapritinib in systemic mastocytosis (Press release, Blueprint Medicines, MAY 14, 2020, View Source [SID1234558060]).

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"The upcoming presentations reflect our commitment to bring transformative therapies to patients by selectively targeting genomic drivers of disease," said Andy Boral, M.D., Ph.D., Chief Medical Officer of Blueprint Medicines. "The data support the rapid clinical advancement of pralsetinib and avapritinib, with multiple global regulatory submissions under review or planned in 2020. These abstracts reinforce the consistent clinical activity shown by our drug candidates across broad patient populations with RET-altered cancers and systemic mastocytosis."

Updated clinical data will be presented in the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 (ASCO20) Virtual Scientific Program, at the European Academy of Allergy and Clinical Immunology (EAACI) Digital Congress and at the Virtual Edition of the 25th European Hematology Association (EHA) (Free EHA Whitepaper) (EHA25) Annual Congress. Accepted abstracts are listed below. Abstracts are expected to be available on the EAACI conference website (View Source) at the start of the congress, and are now available on the following conference websites: View Source and View Source The following presentations will be available to registered participants of each virtual meeting at its conference website.

ASCO20 Virtual Scientific Program
May 29-31, 2020

Oral Presentation

Presentation Title: Clinical activity of the RET inhibitor pralsetinib (BLU-667) in patients with RET fusion+ solid tumors
Session Title: Drug Development for Rare Mutations: The Opportunity to Unite and Conquer
Session Date & Time (Scheduled Broadcast): Sunday, May 31, 2020 from 10:30 a.m. – 12:00 p.m. ET
Abstract Number: 109

Poster Discussion Presentation

Presentation Title: Registrational dataset from the Phase I/II ARROW trial of pralsetinib (BLU-667) in patients (pts) with advanced RET fusion+ non-small cell lung cancer (NSCLC)
Presentation Date & Time (On-Demand): Friday, May 29, 2020 beginning at 8:00 a.m. ET
Session Title: Lung Cancer—Non-Small Cell Metastatic
Abstract Number: 9515

Poster Presentation

Presentation Title: AcceleRET Lung: A Phase III study of first-line pralsetinib in patients (pts) with RET-fusion+ advanced/metastatic non-small cell lung cancer (NSCLC)
Presentation Date & Time (On-Demand): Friday, May 29, 2020 beginning at 8:00 a.m. ET
Session Title: Lung Cancer—Non-Small Cell Metastatic
Abstract Number: TPS9633

EAACI Digital Congress
June 6-8, 2020

Late Breaking Oral Presentation

Presentation Title: Avapritinib reduced cutaneous symptoms and mast cell (MC) burden in patients (pts) with indolent systemic mastocytosis (ISM) in the PIONEER study
Presentation Date & Time (On-Demand): Saturday, June 6, 2020 beginning at 9:00 a.m. CEST (3:00 a.m. ET)
Session Title: Skin Diseases: What Is New?
Abstract Number: 1832

EHA25 Virtual Congress
June 11-14, 2020

Poster Presentations

Presentation Title: Avapritinib induces responses in patients (pts) with advanced systemic mastocytosis (AdvSM), regardless of prior midostaurin therapy
Presentation Date & Time (On-Demand): Friday, June 12, 2020 beginning at 8:30 a.m. CEST (2:30 a.m. ET)
Session Title: Myeloproliferative Neoplasms – Clinical
Abstract Number: EP1079

Presentation Title: Results from PIONEER: a randomized, double-blind, placebo-controlled, Phase 2 study of avapritinib in patients with indolent systemic mastocytosis
Presentation Date & Time (On-Demand): Friday, June 12, 2020 beginning at 8:30 a.m. CEST (2:30 a.m. ET)
Session Title: Myeloproliferative Neoplasms – Clinical
Abstract Number: EP1082

On May 14, 2020 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted cancer therapeutics and utilizing a cost efficient, CRO-independent product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the U.S., reported that positive data from envafolimab in a pivotal trial in China for the treatment of MSI-H/dMMR cancer will be presented by the Company’s corporate partners, 3D Medicines and Alphamab, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Virtual Scientific Program (Press release, Tracon Pharmaceuticals, MAY 14, 2020, View Source [SID1234558059]).

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ASCO abstract #3021 entitled "Envafolimab (KN035) in Advanced Tumors with Mismatch-Repair Deficiency" reviewed data available on December 17, 2019 from the ongoing pivotal Phase 2 trial of envafolimab given by subcutaneous injection without an adjuvant in MSI-H/dMMR cancer. The trial enrolled 103 patients with MSI-H colorectal (CRC) or gastric cancer (GC) or with dMMR in other advanced solid tumors, in an open label format with efficacy endpoints, including the primary endpoint of confirmed objective response rate (ORR) determined by independent central review. MSI-H/dMMR status was assessed centrally for CRC and GC and locally for other tumors.

Key highlights included:

The confirmed ORR in 50 patients with CRC who failed a fluoropyrimidine, oxaliplatin and irinotecan (n=39) plus those with advanced GC who failed at least one prior systemic treatment (n=11), with at least two on-study tumor assessments, was 30% (95% CI: 18%, 45%), of whom 80% were responding with median follow-up of 7.5 months at the time of the data cutoff.
The confirmed ORR in the overall population (n=103) was 34% (95% CI: 25%, 44%), of whom 86% were responding with median follow-up of 6.7 months at the time of the data cutoff.
The confirmed ORR in 24 patients with CRC who failed a fluoropyrimidine and oxaliplatin or irinotecan (n=24) was 54% (95% CI: 33%, 74%) of whom 85% were responding at the time of the data cutoff.
Envafolimab was well tolerated with a safety profile similar to that of approved PD-(L)1 checkpoint inhibitors but without infusion related reactions.
"We are impressed by the confirmed ORR of envafolimab from its pivotal trial in Chinese patients with MSI-H/dMMR cancer, which is a genetically defined tumor type," said Charles Theuer, M.D., Ph.D., President and CEO. "We believe these data are important in assessing the potential of this novel subcutaneously administered product candidate in TRACON’s initial indications in the U.S. of undifferentiated pleomorphic sarcoma (UPS) and myxofibrosarcoma (MFS), two soft tissue sarcoma subtypes which have responded to treatment with other checkpoint inhibitors. Moreover, these data indicate that envafolimab’s activity in MSI-H cancer is similar to other checkpoint inhibitors, such as Keytruda or Opdivo but without infusion related reactions. We look forward to initiating our pivotal ENVASARC trial for envafolimab in UPS and MFS in the second half of 2020, for which we recently reached agreement on the key elements with the U.S. FDA."

The complete abstract is available at: View Source

About Envafolimab

Envafolimab is a novel, single-domain antibody against PD-L1 that is administered by subcutaneous injection without the need for an adjuvant. Envafolimab is currently dosing in Phase 1 trials in the U.S. and Japan and is being studied in China in a Phase 2 registration trial as a single agent in MSI-H/dMMR tumor patients, and in combination with gemcitabine and oxaliplatin in a Phase 3 registration trial in biliary tract cancer. 3D Medicines plans to file a BLA in China for envafolimab in 2020 based on overall response rate and duration of response in MSI-H/dMMR patients. The filing would be based on the the ongoing pivotal phase 2 trial data of envafolimab in MSI-H/dMMR cancer.

On May 14, 2020 Myovant Sciences (NYSE: MYOV), a healthcare company focused on redefining care for women and for men, reported that it will present new efficacy and cardiovascular safety data from the Phase 3 HERO study of once-daily, oral relugolix (120 mg) in men with advanced prostate cancer at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s upcoming ASCO (Free ASCO Whitepaper)20 Virtual Scientific Program, to be held May 29-31, 2020 (Press release, Myovant Sciences, MAY 14, 2020, https://investors.myovant.com/news-releases/news-release-details/myovant-sciences-present-new-data-relugolix-prostate-cancer [SID1234558058]).

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The new data will be presented by Neal Shore, M.D., medical director of the Carolina Urologic Research Center and HERO program steering committee member. Dr. Shore’s presentation (#5602), titled "HERO phase III trial: Results comparing relugolix, an oral GnRH receptor antagonist, versus leuprolide acetate for advanced prostate cancer" will be included in the oral abstract session, "Genitourinary Cancer—Prostate, Testicular, and Penile," which will be available for on-demand viewing starting at 8:00 a.m. Eastern Time / 5:00 a.m. Pacific Time on Friday, May 29, 2020.

In April 2020, Myovant submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration for relugolix as a potential treatment for men with advanced prostate cancer. The NDA is supported by positive results from the Phase 3 HERO study, a randomized pivotal study comparing relugolix versus leuprolide acetate.

On May 14, 2020 Advaxis, Inc. (Nasdaq: ADXS), a clinical-stage biotechnology company focused on the development and commercialization of immunotherapy products reported updated clinical and biomarker results from the monotherapy and combination arms of the Company’s ongoing Phase 1/2 study investigating ADXS-503 in patients with non-small cell lung cancer (NSCLC) (Press release, Advaxis, MAY 14, 2020, View Source [SID1234558057]). The trial is evaluating ADXS-503, alone and in combination with KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy. ADXS-503 is part of the Company’s ADXS-HOT cancer-type specific immunotherapy program which leverages Advaxis’ proprietary Lm technology platform to target hotspot mutations that commonly occur in specific cancer types as well as other proprietary, tumor-associated antigens.

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Key data updates:

Sustained clinical benefit in the combination arm, Part B, in the first two evaluable patients who had previously progressed on KEYTRUDA as last therapy whose best response while on KEYTRUDA was stable disease.
One observed partial response and one response of stable disease with 25% reduction in a target lesion, as confirmed in the radiographic scans at 16 weeks of combination therapy
Responses achieved after immediate prior progression on KEYTRUDA with previous best responses of stable disease suggests ADXS-503 may re-sensitize or enhance response to KEYTRUDA
Preliminary biomarker data from seven patients in monotherapy demonstrates:
Activation of cytotoxic and memory CD8+ and CD4+ T cells
CD8+ T cells generated in all seven patients evaluated
Antigen spreading observed in five of seven patients for epitopes not incorporated in ADXS-503
Part A monotherapy has been completed (n=7) with three out of six (50%) evaluable patients showing stable disease.
As a monotherapy, and in combination with KEYTRUDA, ADXS-503 appeared safe and well tolerated with no dose limiting toxicities at the dose-levels evaluated.
"We are thrilled to see sustained clinical benefit with ADXS-503 in combination with KEYTRUDA in these first two evaluable patients who had recently progressed on KEYTRUDA," said Ken Berlin, Chief Executive Officer of Advaxis. "Importantly these responses were achieved in patients with prior best responses that were limited to stable disease during their two years on this checkpoint inhibitor. Taken together, these results leave us increasingly confident that ADXS-503 has the potential to restore or enhance sensitivity to checkpoint inhibitors. In addition, we believe our recent biomarker data, which shows the robust and specific activation of an immune response to ADXS-503 antigens, provides important understanding and validation of the mechanism of action of ADXS-503."

Dr. Andres Gutierrez, Chief Medical Officer at Advaxis, said, "With these promising clinical, safety and immunogenicity data, we have decided to expand Part B of the study, which will continue evaluating patients that have progressed on KEYTRUDA as their last prior therapy. In addition, we will start Part C of the trial to evaluate ADXS-503 with KEYTRUDA combination therapy as a first line treatment for NSCLC patients that cannot tolerate the standard of care regimen with KEYTRUDA in combination with chemotherapy. With the recent dosing of the third patient in Part B, Dose Level 1 which completes this cohort, we anticipate beginning enrollment in the Part B expansion and Part C in early June and look forward to continued progress as we evaluate the ability of ADXS-503 to generate anti-tumor immune responses in advanced NSCLC patients with significant need for new treatment options."

The Phase 1/2 clinical trial of ADXS-503 is seeking to establish the recommended dose, safety, tolerability and clinical activity of ADXS-503 administered alone and in combination with a KEYTRUDA in approximately 50 patients with NSCLC, in at least five sites across the U.S. The two dose levels with monotherapy in Part A, (1 X108 and 5 X108 CFU) have been completed and Part B with ADXS-503 (1 X108 CFU) in combination with KEYTRUDA is currently closed to enrollment. With preliminary encouraging safety and efficacy results, the Company is planning to expand Part B to additional patients at dose level 1 (1 X108 CFU + KEYTRUDA) with the potential to proceed to dose level 2 (5 X108 CFU + KEYTRUDA) at a later date. In addition, the Company intends to expand the study to Part C, which will evaluate ADXS-503 in combination with KEYTRUDA (1 X108 CFU + KEYTRUDA) as a first line treatment for patients that are medically unfit to receive the standard of care regimen with KEYTRUDA in combination with chemotherapy.

About ADXS-HOT
ADXS-HOT is a program that leverages the Company’s proprietary Lm technology to target hotspot mutations that commonly occur in specific cancer types. ADXS-HOT drug candidates are designed to target acquired shared or "public" mutations in tumor driver genes along with other proprietary cancer-testes and oncofetal tumor-associated antigens that also commonly occur in specific cancer types. ADXS-HOT drug candidates are an off-the-shelf treatment, designed to potentially treat all patients with a specific cancer type, without the need for pretreatment biomarker testing, DNA sequencing or diagnostic testing.