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Gracell Reports Data of First-in-human Clinical Trial for Universal TruUCAR™ GC027 in Relapsed or Refractory T-cell Acute Lymphoblastic Leukemia at the AACR Virtual Annual Meeting

On April 28, 2020 Gracell Biotechnologies Co., Ltd. ("Gracell"), a clinical-stage immune cell and gene therapy company, reported data of its first-in-human clinical trial for Universal TruUCAR GC027 in relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL) patients (Press release, Gracell Biotechnologies, APR 28, 2020, View Source [SID1234556719]). The data was presented in the Adoptive Cell Transfer Therapy section during the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting held on April 28.

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T-ALL, a form of acute lymphoblastic leukemia, consisting of 20-25% of adult ALL, and 12-15% of pediatric ALL, represents a hard-to-treat disease with a high unmet clinical need.[1] Outcome of relapsed and refractory T-ALL remains poor, with very limited treatment options available. Most T-ALL patients relapse within two years after multi-agent chemotherapy regimens. The study of TruUCAR GC027 in relapsed and refractory T-ALL reports early efficacy outcomes of five patients treated in this area of high unmet medical need.

The clinical investigator initial trial (IIT) intends to evaluate safety and efficacy of TruUCAR GC027, the first-in-human, universal CAR-T therapy for R/R T-ALL. As of February, the study enrolled a total of five patients with R/R T-ALL, with median prior lines of therapy 5 (range 1-9). Baseline bone marrow tumor burden was 38.2% (range 4-80.2). All patients received a single infusion of TruUCAR GC027 in one of three dose levels: 0.6*10^7cells/kg, 1.0*10^7cells/kg or 1.5*10^7cells/kg. Notably, these patients were not HLA matched, and no one accepted post-infusion hematopoietic stem cell transplantation (HSCT).

Treatment efficacy was assessed in five patients with 28 days of follow-up, of which:

Five (100%) achieved a complete remission with or without complete blood count recovery (CR/CRi);
Four (80%) achieved minimum residual disease negative complete remission (MRD-CR).
All five patients tolerated the single infusion of TruUCAR GC027 with no neurotoxicity events or acute graft-versus-host disease (aGvHD) observed. Cytokine release syndrome (CRS) presented in all patients at any grade.

"We are delighted to report the outcome on the first five patients treated with TruUCAR GC027. These promising preliminary results are encouraging and warrant further evaluation of the therapy in this area of high unmet clinical need." said Dr. Martina Sersch, CMO of Gracell.

About TruUCAR

TruUCAR is Gracell’s proprietary and patented platform technology, with selected genes being edited to avoid GvHD and immune rejection without using strong immunosuppressive drugs. In addition to T-ALL antigen, the platform technology can also be implemented for other targets of hematological malignancies.

About GC027

GC027 is an investigational, off-the-shelf CAR-T cell therapy, redirected to CD7 for the treatment of T cell malignancies. GC027 was manufactured from T cells of human leukocyte antigen (HLA) unmatched healthy donors using TruUCAR technology, which is expected to improve efficacy and reduce production time, available for off-the-shelf use in a timely manner.

About T-ALL

T – Lymphoblastic Leukemia (T-ALL) is an aggressive form of acute lymphoblastic leukemia, with a diffuse invasion of bone marrow and peripheral blood. In 2015, T-ALL affected around 876,000 people globally and resulted in 110,000 deaths worldwide. T-ALL compromises about 15%-20% of all children and adult acute lymphoblastic leukemia[2]. Current standard of care therapies for T-ALL are chemotherapy and stem cell transplantation. 40-50% of patients will experience relapse within two years following front line therapy with limited treatment options available[3][4]. Treatment of relapsed and refractory T-ALL remains a high unmet medical need.

Synthetic Biologics to Report 2020 First Quarter Operational Highlights and Financial Results on May 5, 2020

On April 28, 2020 Synthetic Biologics, Inc. (NYSE American: SYN), a diversified clinical-stage company leveraging the microbiome to develop therapeutics designed to prevent and treat gastrointestinal (GI) diseases in areas of high unmet need, reported that the Company intends to release its operational highlights and financial results for the quarter ended March 31, 2020 on Tuesday, May 5, 2020, and to host a conference call the same day at 4:30 p.m. ET (Press release, Synthetic Biologics, APR 28, 2020, View Source [SID1234556718]). The dial-in information for the call is as follows:

U.S. (toll free): 1-888-347-5280
International: +1-412-902-4280

Participants are asked to dial in 15 minutes before the start of the call to register. The call will also be webcast over the Internet at View Source." target="_blank" title="View Source." rel="nofollow">View Source An archived replay of the call will be available for approximately ninety (90) days at the same URL, View Source beginning approximately one hour after the call’s conclusion.

Nektar to Announce Financial Results for the First Quarter 2020 on Thursday, May 7, 2020, After Close of U.S.-Based Financial Markets

On April 28, 2020 Nektar Therapeutics (Nasdaq: NKTR) reported that it will announce its financial results for the first quarter 2020 on Thursday, May 7, 2020, after the close of U.S.-based financial markets (Press release, Nektar Therapeutics, APR 28, 2020, View Source [SID1234556717]). Howard Robin, President and Chief Executive Officer, will host a conference call to review the results beginning at 5:00 p.m. Eastern Time/2:00 p.m. Pacific Time.

The press release and live audio-only webcast of the conference call can be accessed through a link that is posted on the home page and Investors section of the Nektar website: View Source The web broadcast of the conference call will be available for replay through Monday, June 1, 2020.

To access the conference call, follow these instructions:
Dial: (877) 881-2183 (U.S.); (970) 315-0453 (international)
Conference ID: 8288857 (Nektar Therapeutics is the host)

Thrive Earlier Detection Announces Groundbreaking Clinical Data from the First Ever Interventional Study of a Blood-Based Test to Screen and Identify Multiple Types of Cancer

On April 28, 2020 Thrive Earlier Detection Corp., a company dedicated to extending and saving lives by incorporating earlier cancer detection into routine medical care, together with Johns Hopkins University and Geisinger Health, reported data from the landmark DETECT-A study (Press release, Thrive Earlier Detection, APR 28, 2020, View Source [SID1234556711]). DETECT-A (Detecting cancers Earlier Through Elective mutation-based blood Collection and Testing) is the first ever prospective, interventional study to use a blood test to screen for multiple types of cancers in a real-world population. The study was conducted by Johns Hopkins University and Geisinger and enrolled more than 10,000 women with no prior history of cancer. The purpose was to identify multiple cancer types in asymptomatic individuals using an early version of CancerSEEK developed in 2016 ("Thrive’s blood test"). DETECT-A is the first study of a multi-cancer blood-based screening test to deliver results to physicians to manage patient care.

Cancer is the second leading cause of death in the United States with an estimated 600,000 people expected to die from the disease this year. Most of these cancers are often detected too late, and only after people start to experience symptoms. These "symptom-detected" cancers too frequently coincide with late stage, metastatic disease, and result in poor outcomes. However, when cancer is detected through screening, or are "screen-detected," the disease is often identified earlier when it can be more effectively treated, and in many cases even cured. Unfortunately, current standard-of-care screening tests, like mammography and colonoscopy, only detect less than 30% of all incident cancers. The DETECT-A study provides the first real-world evidence that we can significantly shift the paradigm from "symptom-detected" cancers to more "screen-detected" cancers via a multi-cancer blood-based test.

The key findings from this prospective, interventional study demonstrate the following:

Thrive’s blood test identified cancers in individuals without any history of the disease.
Thrive’s blood test more than doubled the number of cancers that were first "screen-detected." 25% of the women who were diagnosed with cancer were identified by current standard-of-care tests. By incorporating Thrive’s blood test, the percentage of "screen-detected" cancers increased from 25% to 52%.
Thrive’s blood test identified cancers across 10 different organs, seven of which currently have no standard-of-care screening.
Thrive’s blood test can identify cancers prior to clinically evident metastasis. 65% of the cancers identified were localized or regional.
Thrive’s blood test is additive and complementary to standard-of-care and was incorporated into routine medical care without discouraging patients from engaging in other forms of screening.
Thrive’s blood test, in combination with imaging, minimized false positive results with 99.6% specificity.
The study’s workflow (blood test plus imaging) safely guided clinical follow-up in blood test-positive participants with zero adverse events.
"This study is a seminal moment in cancer screening that advances the entire field," said Christoph Lengauer, Ph.D., co-founder and chief innovation officer of Thrive. "For the first time, a blood test was utilized in a real-world setting and was able to more than double the number of cancers first identified through screening methods. We learned that it can be both complementary to existing standard-of-care screening tools, and a significant benefit for many types of cancers like ovarian, appendix and kidney, which do not have any current screening modalities."

These data were published in Science and were presented today during the clinical plenary: Early Detection and CtDNA at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting.

Prospective Interventional Study Design

The ability to identify multiple types of cancers through a blood draw is one of the most exciting advances in cancer diagnostics; however, prospective, interventional studies like DETECT-A are imperative to understand if a test can work in the real world. To date, retrospective and observational studies of blood-based multi-cancer tests have curated samples from participants who were already known to have cancer at the time of testing. Conversely, in DETECT-A, a prospective interventional study, participants were unaware of cancer at the time of enrollment and test results were reported to physicians and guided intervention.

DETECT-A enrolled 10,006 women with no prior history of cancer in a population with high adherence to standard-of-care cancer screening tests, such as mammography and colonoscopy. All participants were enrolled through the Geisinger Health System, enabling access to electronic medical records of participating individuals and minimizing loss to follow up.

DETECT-A utilized an early version of CancerSEEK that was developed in 2016, and analyzes 16 genes and nine proteins causatively linked to multiple types of cancer. Screening began with the analysis of a blood sample to identify potential abnormal values for at least one biomarker. Those with abnormal values were invited back for a confirmatory test to determine whether the identical biomarker was persistently abnormal and if appropriate, the individual was reviewed by a Multidisciplinary Review Committee. If a non-cancerous cause for the abnormal biomarker could not be identified, imaging was ordered. Patients with concerning imaging findings were referred to cancer specialists for further evaluation.

Study Results

Thrive’s blood test doubled the number of cancers first found through screening. Among the eligible participants, 96 women developed cancers: 26 of these were first identified by Thrive’s blood test, 24 were first identified by standard-of-care screening methods, and 46 were first identified by symptoms or other means. Thrive’s blood test detected cancers across ten different organs, including seven organs that do not have standard-of-care screening tools available. Notably, 65% of cancers detected by Thrive’s blood test were identified as local or regional disease, allowing for earlier intervention and if indicated, surgery with intent to cure. Thrive’s blood test’s sensitivity was 27.1% across all cancers and 31.1% for the seven cancers with no screening options. Importantly, Thrive’s blood test plus standard-of-care testing had a combined sensitivity of 52.1%, underscoring that a multi-cancer blood test is both a significant added benefit and complementary to standard-of-care screening tools.

Maintaining a high specificity thereby minimizing "false-positive" results is essential for a multi-cancer blood test. Screening with Thrive’s blood test alone had a 98.9% specificity, and when combined with imaging had a specificity of 99.6%. Thrive’s blood test plus imaging safely and efficiently guided clinical follow-up in blood test-positive participants with zero adverse events. Holistically, in this asymptomatic population with a cancer incidence of approximately 1%, Thrive’s blood test plus imaging resulted in a positive predictive value (PPV) of 40.6%, which is considerably higher than the PPV of existing single-cancer screening tests available today.

"Through this first-ever interventional study, the teams at Johns Hopkins University, Geisinger and Thrive have forged a new path and advanced the field of blood-based earlier cancer detection," said David J. Daly, chief executive officer of Thrive. "Thrive is now one step closer to realizing our vision of providing a comprehensive approach to cancer screening, helping to shift the paradigm so that in the future, most cancers can be identified through earlier detection when there is the greatest opportunity for cure."

Prokarium Signs an Exclusive Option Agreement with the Lausanne University Hospital (Centre Hospitalier Universitaire Vaudois – CHUV) for the Treatment of Non-Muscle Invasive Bladder Cancer (NMIBC)

On April 28, 2020 Prokarium, a private biotechnology company, focusing on genetically engineered bacteria for the development of microbial immunotherapy and vaccines, reported that it has signed an exclusive, worldwide Option Agreement with the Lausanne University Hospital – CHUV, a Switzerland-based research hospital, to acquire a license to cover the treatment of Non-Muscle Invasive Bladder Cancer (NMIBC) patients with intravesical instillations of Salmonella (Press release, Prokarium, APR 28, 2020, View Source [SID1234556709]).

Prokarium is investigating the use of engineered bacteria for the development of microbial immunotherapy for solid tumours. Their first oncology indication in preclinical development is NMIBC, which accounts for 400,000 new cases yearly worldwide and is currently lacking innovative treatments. Prokarium aims to disrupt the market with their engineered Salmonella that acts by boosting the natural anti-tumour immune response as well as through direct tumour killing.

"The standard of care of NMIBC is surgical removal of the tumour followed by up to 27 intravesical instillations of Bacillus Calmette–Guérin (BCG), which, despite the initial high response rate, has a 40-80% recurrence rate within 5 years" said Ted Fjallman, Ph.D, Chief Executive Officer, Prokarium. "Additionally, there is a significant worldwide shortage of BCG and many oncology patients are not able to receive their treatments."

Prokarium started a collaboration with a team at the Department of Urology of CHUV, led by Dr. Denise Nardelli-Haefliger, in 2019. The group, with Prof. Patrice Jichlinski and Dr. Ilaria Lucca, is running a Phase I trial in NMIBC patients investigating the intravesical administration of Salmonella Typhi Ty21a. Prokarium is working with this medical team to generate an adequate preclinical data package to file an IND.

"We are happy that thanks to the partnership with Prokarium, the results of our long-lasting immunotherapeutic research will soon be developed to benefit NMIBC patients" said the inventors Sonia Domingos-Pereira, Patrice Jichlinski and Denise Nardelli-Haefliger.

About NMIBC

Bladder cancer represents 5% of all new cancer cases in the US and accounts for 550,000 new cases yearly worldwide. The clinical staging is determined by the depth of invasion into the bladder wall and more than 70% of cases are diagnosed at an early stage, also known as Non-Muscle Invasive Bladder Cancer (NMIBC). Despite the early diagnosis, the only approved therapies are Bacillus Calmette–Guérin (BCG), which often faces shortages, and chemotherapy, both delivered intravesically. Because of its high incidence and the low number of treatment options, a huge unmet medical need remains in NMIBC.