On May 12, 2021 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported that interim data from the ongoing Phase 1/2 clinical trial investigating the safety and efficacy of MB-106 CD20-targeted CAR T for high-risk B-cell non-Hodgkin lymphomas ("B-NHL") and chronic lymphocytic leukemia ("CLL") has been selected for an e-poster presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 2021 Virtual Congress ("EHA2021") (Press release, Mustang Bio, MAY 12, 2021, View Source [SID1234579818]). MB-106 is being developed in a collaboration between Mustang and Fred Hutchinson Cancer Research Center ("Fred Hutch").

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In the abstract posted today on the EHA (Free EHA Whitepaper)2021 website, Fred Hutch reported on 12 patients treated with MB-106, which underwent a major cell manufacturing modification after treating the first 7 patients as previously reported at the 62nd Annual American Society of Hematology (ASH) (Free ASH Whitepaper) meeting in 2020. The 12 patients treated under the new manufacturing process were treated at dose levels ("DL") ranging from 3.3×105 to 1×107 CAR T cells/kg, and clinical responses were observed at all DLs with no dose-limiting toxicities. Cytokine release syndrome occurred in 3 patients (25%): 2 patients with grade 1 and 1 patient with grade 2. Only 1 patient required tocilizumab and dexamethasone, and no immune effector cell-associated neurotoxicity syndrome of any grade was observed. Overall response rate ("ORR") was 92% (11/12) with a complete response ("CR") rate of 58% (7/12). In 9 patients with follicular lymphoma, ORR and CR were 89% (8/9) and 67% (6/9), respectively. The patient with CLL had a PET-negative CR and undetectable measurable residual disease in peripheral blood and bone marrow by flow cytometry (10-4) (uMRD4) on day 28. Among patients who received the highest two dose levels, DL3 (3.3×106 CAR T cells/kg; n=4) and DL4 (1×107 CAR T cells/kg; n=1), CR rate was 100% (5/5). All 7 patients who achieved a CR remain in remission at a median follow-up of 4 months. CAR T expansion was robust, with median peak blood levels of CAR+ T cells of 122 CAR+ cells/μl (range 0.27-2024), corresponding to 19% (range 0.15 – 65%) of all CD3+ cells. Updated data will be presented at EHA (Free EHA Whitepaper)2021.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "We are pleased that Fred Hutch will present interim data from the ongoing Phase 1/2 trial of MB-106 at EHA (Free EHA Whitepaper)2021. The data thus far indicate that MB-106 has a highly favorable safety profile at all dose levels when compared to commercially available CAR T cell therapies targeting CD19. We look forward to the continued progression of this CD20-targeted CAR T cell therapy program for patients with relapsed or refractory B-cell non-Hodgkin lymphomas and CLL."

Details of the MB-106 e-poster presentation are as follows:

Topic: 25. Gene therapy, cellular immunotherapy and vaccination – Clinical
Abstract Code: EP731
Title: Immunotherapy Using a 3rd Generation CD20 Targeted CAR T-Cell (MB-106) for Treatment of B-Cell Non-Hodgkin Lymphoma (B-NHL) and Chronic Lymphocytic Leukemia (CLL)
Presenter: Mazyar Shadman, M.D., M.P.H., Fred Hutchinson Cancer Research Center, Seattle, WA, USA
Date and Time: All e-posters will be available for viewing on June 11, 2021 at 09:00 Central European Summer Time (CEST) / 3 a.m. ET

A copy of the abstract can be viewed online through the EHA (Free EHA Whitepaper)2021 website at View Source

On May 12, 2021 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported an abstract presentation related to AUTO1 in relapsed / refractory (r/r) indolent B cell lymphomas (IBCL) at the European Hematology Association (EHA) (Free EHA Whitepaper) Virtual Congress to be held June 9-17, 2021 (Press release, Autolus, MAY 12, 2021, View Source [SID1234579817]).

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Title: Early safety and efficacy findings of AUTO1 (CAT19), a fast-off rate CD19 CAR, in Relapsed/Refractory Indolent B Cell Lymphomas

Presenter: Clare Roddie, MD, PhD, FRCPath, Consultant Haematologist and Honorary Senior Lecturer, Cancer Institute, University College London (UCL)

Date and Time: All e-poster presentations will be made available on the on-demand Virtual Congress platform as of Friday, June 11 at 9.00 AM CEST.

As of the data cut-off date of February 18, 2021, 10 r/r IBCL patients had received AUTO1 and nine patients were evaluable. AUTO1 demonstrated a tolerable safety profile in adult patients with r/r low grade B-cell lymphoma despite high disease burden. Early data shows 100% complete remission rates and excellent CAR engraftment and expansion. Grade 1 CRS was reported in 4 patients and Grade 2 CRS in 1 patient. No Immune effector cell-associated neurotoxicity syndrome (ICANS) was observed on study. At a median of 3.1 months (range 1-5.6m), 8/9 patients are in ongoing remission. One patient died in complete remission at month 5.6 of COVID-19.

On May 12, 2021 Leap Therapeutics (Nasdaq: LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, and Flagship Biosciences, the leader in data-centric pathology and tissue analysis, reported that they have developed an image analysis RNAscope assay that is being used successfully for prospective patient enrollment in a clinical trial (Press release, Leap Therapeutics, MAY 12, 2021, View Source [SID1234579816]). To the companies’ knowledge, this is the first example of an RNAscope assay using a digital image analysis solution for patient enrollment. The findings were published in an article, "Validation of a DKK1 RNAscope chromogenic in situ hybridization assay for gastric and gastroesophageal junction adenocarcinoma tumors," on Monday, May 10 in Scientific Reports. It can be viewed at View Source

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Co-authored by a collaborative team from Leap Therapeutics, Flagship Biosciences, and Athenaeum Pathology Consulting, the article highlights how Leap is identifying patients likely to benefit from DKN-01, its anti-Dickkopf-1 (DKK1) antibody, and Flagship’s ability to validate and implement complex biomarker assays using digital image analysis. Leap and Flagship’s work centered on the development and validation of a DKK1 RNAscope chromogenic in situ hybridization (CISH) assay and digital image analysis solution. DKK1 is a secreted modulator of Wnt signaling that is frequently overexpressed in tumors and associated with a poor prognosis for patients. DKN-01 is a humanized monoclonal therapeutic antibody that binds to and blocks the activity of DKK1 and has demonstrated clinical activity in gastric/gastroesophageal junction (G/GEJ) adenocarcinoma patients with elevated tumoral expression of DKK1 RNA. Leap and Flagship have validated the DKK1 RNAscope assay and accompanying digital image analysis solution as specific, sensitive, accurate, and reproducible according to Clinical Laboratory Improvement Amendments (CLIA) guidelines, and their work is currently being applied to prospectively identify G/GEJ patients with elevated tumoral expression of DKK1 for treatment with a DKN-01 plus tislelizumab combination (Leap Therapeutics; NCT04363801). Additionally, the companies are using the DKK1 RNAscope assay and digital image analysis solution for a retrospective analysis of G/GEJ patients treated with DKN-01 in combination with tislelizumab and chemotherapy.

Biomarkers are increasingly prominent in drug development and are often applied in clinical trial design to determine the patients who are most likely to benefit from a specific therapeutic or treatment regimen. To use a biomarker strategy to prospectively identify patients, the biomarker test must be robust and precise. Because of the challenges in manual semi-quantification of RNAscope tissue staining, Leap and Flagship developed the novel digital image analysis algorithm that identifies tumor cells and quantifies DKK1 signal.

"A potential challenge with RNAscope is manual scoring, which requires a pathologist to score the tissue at a high magnification to visualize and count the number of stained dots that appear in tumor cells," said Michael Kagey, Ph.D., Senior Director of Translational Medicine at Leap Therapeutics. "Since this can lead to slow, inaccurate, and non-reproducible scoring, we worked with Flagship to develop a digital algorithm. The digital algorithm reduces pathologist time, potential variability from manual scoring and allows us to reliably screen for patients who may benefit from our therapy."

In addition to the prospective screening of patients, the work is also further advancing the companion diagnostic development of the RNAscope assay and could generally be used as a guide for the validation of RNAscope CISH assays with digital image quantification.

"Our technology, combined with our scientific process and pathology oversight allows you to quickly find the right patients and get to your endpoints more rapidly," said Trevor Johnson, CEO of Flagship Biosciences. "Image analysis also provides a superior return on investment by saving time and helping get drugs to market more quickly. Our ability to take a completely novel pathology approach and turn it into a CLIA-validated diagnostic test to help our clients is extremely gratifying."

"Our clinical development has been enhanced by the rapid, prospective patient screening that has happened as a result of using the DKK1 RNAscope CISH assay and digital image analysis solution," said Douglas E. Onsi, President and CEO of Leap Therapeutics. "As a result of the partnership between Leap and Flagship, the patients who we believe will best benefit from our drug are now being included in our trial. We look forward to further collaborating with Flagship Biosciences."

On May 12, 2021 Checkpoint Therapeutics, Inc. ("Checkpoint") (NASDAQ: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported the completion of enrollment for the metastatic cutaneous squamous cell carcinoma ("cSCC") cohort in its registration-enabling clinical trial of anti-PD-L1 antibody, cosibelimab (Press release, Checkpoint Therapeutics, MAY 12, 2021, View Source [SID1234579815]).

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In January 2020, Checkpoint announced that the U.S. Food and Drug Administration had confirmed the registration submission pathway for cosibelimab in metastatic cSCC based on the ongoing clinical trial, which has a target enrollment of approximately 75 patients and a primary efficacy endpoint of confirmed objective response rate assessed by independent central review. Top-line results are expected in the fourth quarter of 2021 and, upon a successful outcome, Checkpoint intends to submit a Biologics License Application ("BLA") for cosibelimab in the first half of 2022, followed shortly thereafter by a Marketing Authorization Application submission in Europe. Additionally, Checkpoint continues to enroll a registration-enabling cohort of patients with locally advanced cSCC and anticipates that this second indication will also be included in the planned BLA and MAA submissions next year.

James F. Oliviero, President and Chief Executive Officer of Checkpoint, stated, "We are pleased to report the completion of enrollment for our metastatic cSCC cohort, with over 75 patients enrolled, which we expect will enable a readout of top-line results in the fourth quarter of this year." Mr. Oliviero continued, "Based on the interim data presented last year at the European Society for Medical Oncology ("ESMO") Virtual Congress 2020 and the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) ("SITC") 35th Anniversary Annual Meeting, we believe cosibelimab has the potential to be a best-in-class anti-PD-L1 antibody, which we intend to commercialize at a substantially lower price in comparison to currently marketed anti-PD-(L)1 therapies. With a compelling safety and efficacy profile, as well as our market disruptive pricing strategy, we believe cosibelimab can achieve meaningful and rapid market share in the $25 billion and growing PD-(L)1 class."

About Cutaneous Squamous Cell Carcinoma
cSCC is the second most common human cancer in the United States, with an estimated annual incidence of 700,000 cases. While most cases are localized tumors amenable to curative resection, approximately 8% of patients will experience a local recurrence, 5% of patients will develop nodal metastases, and an estimated 2% of patients will die from their disease. Ten-year survival rates are less than 20% for patients with regional lymph-node involvement. For those patients who develop distant metastases, the median survival time is estimated to be less than two years. In addition to being a life-threatening disease, cSCC causes significant functional morbidities and cosmetic deformities based on tumors commonly arising in the head and neck region and invading blood vessels, nerves and vital organs such as the eye or ear.

About Cosibelimab
Cosibelimab (formerly referred to as CK-301) is a potential best-in-class, high affinity, fully-human monoclonal antibody of IgG1 subtype that directly binds to programmed death ligand-1 ("PD-L1") and blocks the PD-L1 interaction with the programmed death receptor-1 ("PD-1") and B7.1 receptors. Cosibelimab’s primary mechanism of action is based on the inhibition of the interaction between PD-L1 and its receptors PD-1 and B7.1, which removes the suppressive effects of PD-L1 on anti-tumor CD8+ T-cells to restore the cytotoxic T cell response. Cosibelimab is potentially differentiated from the currently marketed PD-1 and PD-L1 antibodies through sustained >99% target tumor occupancy to reactivate an antitumor immune response and the additional benefit of a functional Fc domain capable of inducing antibody-dependent cell-mediated cytotoxicity ("ADCC") for potential enhanced efficacy in certain tumor types.

On May 12, 2021 Adamis Pharmaceuticals Corporation (NASDAQ: ADMP), a biopharmaceutical company developing and commercializing specialty products for respiratory disease, allergy and opioid overdose, reported that it will host an investor conference call on Monday, May 17, 2021 at 2 p.m. Pacific Time to discuss its financial and operating results for the first quarter of 2021 as well as provide a business update (Press release, Adamis Pharmaceuticals, MAY 12, 2021, View Source [SID1234579814]). The company’s press release concerning its first quarter 2021 financial results will be available after 1 p.m. Pacific Time on May 17, 2021 and on its website at www.adamispharmaceuticals.com, and the company also expects to file its quarterly report on Form 10-Q for the quarter ended March 31, 2021 on that date.

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Dennis J. Carlo, Ph.D., President and CEO of Adamis, will host the call along with other members of the management team. The call is open to the public and will provide an update on recent developments, events that have taken place during the quarter, and certain target milestones and goals for future periods. Forward-looking statements concerning expectations regarding future company performance may be made during the conference call.

A live audio webcast of the conference call will also be available via this link – View Source, with a replay available shortly after the live event.