On October 6, 2022 Scholar Rock (NASDAQ: SRRK), a Phase 3, clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported it will present data from DRAGON, a Phase 1 proof-of-concept study of SRK-181 in patients with advanced solid tumors (Press release, Scholar Rock, OCT 6, 2022, View Source [SID1234621796]). Safety, efficacy, and biomarker results from Part A (dose escalation) and an update on Part B (dose expansion) will be presented as a poster at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 37th Annual Meeting in Boston on November 8-12.

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"We are encouraged by the early clinical data from the ongoing DRAGON Phase 1 clinical trial and look forward to sharing these results at SITC (Free SITC Whitepaper), as well as at future medical meetings as more SRK-181 data from both Part A and B become available," said Nagesh Mahanthappa, Founding and Interim Chief Executive Officer of Scholar Rock. "With a novel mechanism of action and encouraging preclinical and clinical data to date, we believe that SRK-181 has promising potential for the treatment of a variety of cancers that are resistant to checkpoint inhibitors."

SRK-181 is an investigational selective inhibitor of latent TGFβ1 activation and is being developed with the aim of overcoming primary resistance to checkpoint therapy in advanced cancer patients. Combination therapy with selective TGFβ1 blockade may modulate the tumor microenvironment, allowing for immune cell infiltration and potential improvements in the response rate and survival time of cancer patients.

Details of the presentations are as follows:

Title: SRK-181, a latent TGFβ1 inhibitor: safety, efficacy, and biomarker results from the dose escalation portion of a phase I trial (DRAGON trial) in patients with advanced solid tumors
Presentation Type: Poster 780
Presenter: Timothy Yap, MBBS, PhD, FRCP, Medical Oncologist and Physician-Scientist; and Associate Professor, Department of Investigational Cancer Therapeutics, University of Texas MD Anderson Cancer Center
Location: Poster Hall
Date/Time: November 10-11, 9 a.m. to 9 p.m. ET

The abstract for this presentation is available on SITC (Free SITC Whitepaper)’s website: www.sitcancer.org/2022/abstracts/abstract-titles-publications

The presentation will be made available in the Publications & Posters section of Scholar Rock’s website following the conference.

For conference information, visit www.sitcancer.org/2022/program/annual-meeting

About SRK-181

SRK-181 is a selective inhibitor of TGFβ1 activation being developed to overcome primary resistance to checkpoint inhibitor therapy, such as anti-PD-(L)1 antibodies, in advanced cancer. TGFβ1 is the predominant TGFβ isoform expressed in many human tumor types. Based on analyses of various human tumors that are resistant to anti-PD-(L)1 therapy, data suggest TGFβ1 is a key contributor to the immunosuppressive tumor microenvironment, excluding and preventing entry of cytotoxic T cells into the tumor, thereby inhibiting anti-tumor immunity.(1) Scholar Rock believes SRK-181, which specifically targets the latent TGFβ1 isoform, has the potential to overcome this immune cell exclusion and induce tumor regression when administered in combination with anti-PD-(L)1 therapy while potentially avoiding toxicities associated with non-selective TGFβ inhibition. The DRAGON Phase 1 proof-of-concept clinical trial (NCT04291079) in patients with locally advanced or metastatic solid tumors is ongoing. The trial is currently enrolling and dosing patients in multiple proof of concept cohorts conducted in parallel, including urothelial carcinoma (UC), cutaneous melanoma (MEL), non-small cell lung cancer (NSCLC) and clear cell renal cell carcinoma (ccRCC). SRK-181 is an investigational product candidate and its efficacy and safety have not been established. SRK-181 has not been approved for any use by the FDA or any other regulatory agency.

On October 6, 2022 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 the first patient has been treated in its multicenter, open-label, non-randomized Phase 1/2 clinical trial evaluating the safety and efficacy of MB-106, Mustang’s first-in-class CD20-targeted, autologous CAR T cell therapy for the treatment of relapsed or refractory B-cell non-Hodgkin lymphomas ("B-NHL") and chronic lymphocytic leukemia ("CLL") (Press release, Mustang Bio, OCT 6, 2022, View Source [SID1234621794]). The patient did not experience cytokine release syndrome ("CRS") or immune effector cell-associated neurotoxicity syndrome ("ICANS"). MB-106 is being developed in a collaboration between Mustang and Fred Hutchinson Cancer Center ("Fred Hutch"). The multicenter trial under Mustang’s Investigational New Drug Application ("IND") builds upon the initial, ongoing Phase 1/2 clinical trial taking place at Fred Hutch in a single-center study under Fred Hutch’s IND.

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang said, "The first clinical trial under Mustang’s IND is an important milestone in the ongoing development and evaluation of MB-106. Data presented at several prestigious medical meetings earlier this year from the initial, ongoing Phase 1/2 clinical trial at Fred Hutch show that MB-106 continues to demonstrate high efficacy and a favorable safety profile across patients with a wide range of hematologic malignancies. We look forward to providing updates on our multicenter MB-106 clinical trial as it progresses and anticipate reporting efficacy data in the fourth quarter of this year."

Interim data from 28 patients treated in the initial, ongoing Phase 1/2 investigator-sponsored clinical trial at Fred Hutch continue to support MB-106 as a viable CAR T cell therapy for B-NHLs and CLL. As of September 9, 2022, the interim data show:

An overall response rate of 96% and complete response ("CR") rate of 75% in a wide range of hematologic malignancies including follicular lymphoma ("FL"), CLL, diffuse large B-cell lymphoma, and Waldenstrom macroglobulinemia
Twelve patients have experienced CR for more than 12 months (10 ongoing); four patients with CR for more than two years and the longest patient with CR is at 33 months
Six patients with partial response ("PR") improved to CR and all remain in ongoing CR
All three patients previously treated with CD19 CAR T cell therapy have responded to treatment with MB-106
A favorable safety profile for MB-106 as an outpatient therapy remains with no CRS or ICANS ≥ Grade 3
CAR-T persistence results in deepening responses following initial 28-day assessments
"We are excited to broaden the evaluation of MB-106 with this multicenter clinical trial under Mustang’s IND. To date, the data from the initial, ongoing clinical trial at Fred Hutch continue to demonstrate a high rate of complete and durable responses," said Mazyar Shadman, M.D., M.P.H., Study Chair, Associate Professor and physician at Fred Hutch and University of Washington. "In addition, MB-106 has shown potential to treat patients in an outpatient setting and provide another immunotherapy option for patients treated previously with CD19-directed CAR T cell therapy."

About Mustang’s Multicenter MB-106 Phase 1/2 clinical trial
The six-center Phase 1/2 clinical trial is a three-arm study targeting CLL and B-NHL including FL, diffuse large B-cell lymphoma and mantle cell lymphoma. Included in the eligibility criteria are patients who have relapsed after treatment with CD19 CAR-T cell therapy. Additionally, the FL arm will evaluate other indolent histologies including Waldenstrom macroglobulinemia, a rare type of B-NHL for which the U.S. Food and Drug Administration recently granted MB-106 Orphan Drug Designation. Since the Mustang-sponsored multicenter clinical trial is using the same lentiviral vector as the Fred Hutch-sponsored single-center trial, the FDA has allowed dose escalation to begin at a higher dose than what was originally conducted at Fred Hutch.

An estimated 287 patients are anticipated to be enrolled in the trial. All patients must have evidence of CD20 expression in both phases of the clinical trial. In Phase 1, escalating MB-106 dose levels will be tested independently in each arm using a 3+3 design. Patients will be enrolled in one of three arms, based on their primary diagnosis.

A total of up to 18 patients are anticipated to be treated in each Phase 1 arm, including six patients at the maximum tolerated dose, prior to proceeding to the Phase 2 portion of the study for each respective arm, where a total of up to 71 patients will participate in each independent arm. Safety of each dose level will be reviewed for each arm until the maximum tolerated dose has been reached and the recommended Phase 2 dose ("RP2D") has been established for each arm. An assessment of the safety and tolerability of the dose will be made by the Safety Review Committee based on the data from the 28-day dose-limiting toxicity observation period.

In Phase 2, specific arms of relapsed or refractory CD20-positive B-cell NHL or CLL patients will be treated with MB-106 at the respective RP2D for each arm. Each arm will initially include up to 20 patients. Based on the results of the interim analysis, up to an additional 51 patients may be added to each of the arms.

Additional information about the trial can be found on clinicaltrials.gov using the identifier NCT05360238.

About MB-106 (CD20-targeted autologous CAR T Cell Therapy)
CD20 is a membrane-embedded surface molecule which plays a role in the differentiation of B-cells into plasma cells. The CAR T was developed by Mustang’s research collaborator, Fred Hutch, in the laboratories of the late Oliver Press, M.D., Ph.D., and Brian Till, M.D., Associate Professor in the Clinical Research Division at Fred Hutch, and was exclusively licensed to Mustang in 2017. The lentiviral vector drug substance used to transduce patients’ cells to create the MB-106 drug product produced at Fred Hutch has been optimized as a third-generation CAR derived from a fully human antibody. MB-106 is currently in a Phase 1/2 open-label, dose-escalation trial at Fred Hutch in patients with B-NHLs and CLL. The same lentiviral vector drug substance produced at Fred Hutch will be used to transduce patients’ cells to create the MB-106 drug product produced at Mustang Bio’s Worcester, MA, cell processing facility for administration in the multicenter Phase 1/2 clinical trial under Mustang Bio’s IND. It should be noted that Mustang Bio has introduced minor improvements to its cell processing to facilitate eventual commercial launch of the product. In addition, prior to commercial launch, Mustang Bio will replace the Fred Hutch lentiviral vector drug substance with vector produced at a commercial manufacturer. Additional information on these trials can be found at View Source using the identifier NCT05360238 for the Mustang multicenter trial and NCT03277729 for the ongoing trial at Fred Hutch.

On October 6, 2022 Horizon Therapeutics plc (Nasdaq: HZNP) reported that it will release its third-quarter 2022 financial results on Wednesday, Nov. 2, 2022 (Press release, Horizon Therapeutics, OCT 6, 2022, View Source [SID1234621793]). Following the announcement, Horizon’s management will host a live webcast at 8 a.m. Eastern Time to review the Company’s financial and operating results.

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The live webcast may be accessed at View Source Please connect to the Company’s website at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. A replay will be available approximately two hours after the live webcast.

On October 6, 2022 Pulse Biosciences, Inc. (Nasdaq: PLSE), a novel bioelectric medicine company commercializing the CellFX System powered by Nano-Pulse Stimulation (NPS) technology, reported positive clinical data from an FDA approved Investigational Device Exempt treat and resect study on the use of NPS for low-risk basal cell carcinoma (BCC) lesions (Press release, Pulse Biosciences, OCT 6, 2022, View Source [SID1234621792]). An oral video presentation on the favorable results of performing the CellFX procedure for low-risk superficial and nodular BCC lesion clearance will be delivered at the 2022 American Society for Dermatologic Surgery (ASDS) Annual Meeting, taking place on October 6-10, 2022 in Denver, Colorado.

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"It is exciting and encouraging to see complete clearance of basal cell carcinoma lesions using NPS technology," said Amy Ross, MD, medical director and founder of PHDermatology of Tampa, Florida, and study investigator. "The results shown in this study gives me a high degree of confidence in NPS technology as an effective and cosmetically acceptable treatment, and potential alternative to the current standard of care for these types of BCC lesions."

Excisional surgery using electrodesiccation and curettage (ED&C) or Mohs micrographic surgery are the current standards of care for BCC lesions, both of which inevitably lead to scarring. Basal Cell Carcinoma is the most common form of skin cancer, accounting for 67% of all skin cancers and affecting more than 3.6 million patients annually. Nonsurgical NPS technology can potentially be a disruptive treatment option, due to its nonthermal energy to clear lesions with lower likelihood of scar formation.

"Showcasing this exciting new clinical data to dermatology thought leaders at ASDS is a major step towards demonstrating the applicability of NPS technology beyond benign dermatologic conditions," said Kevin Danahy, President and Chief Executive Officer of Pulse Biosciences. "The ongoing body of clinical evidence in dermatology continues to prove that NPS technology is safe and effective and produces excellent outcomes."

Oral Video Presentation:
Title: Nano-Pulse Stimulation (NPS) Technology for Treatment of Low-Risk Basal Cell Carcinoma (BCC) – Feasibility Study Using a Treat and Resect Design

Presented by: Dr. Amy Ross, Tampa, FL
Co-Authors: Amy S. Ross, MD; Todd Schlesinger, MD; Christopher Harmon, MD; Ronald Moy, MD; Thomas E. Rohrer, MD; Darius R. Mehregan, MD, PhD; Lauren M. Johnston, BS; William A. Knape, BS

Location: The video presentation will be available within the Education Session Room to those attending the 2022 American Society for Dermatologic Surgery (ASDS) Annual Meeting during open exhibit hours.

On October 6, 2022 Orion reported that it will publish Interim Report for January–September 2022 on Thursday, 20 October 2022 at approximately 12.00 noon EEST (Press release, Orion , OCT 6, 2022, View Source [SID1234621791]). The report and related presentation material will be available on the company’s website at www.orion.fi/en/investors after publishing.

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Webcast and conference call

A webcast and a conference call for analysts, investors and media will be held on Thursday, 20 October 2022 at 13.30 EEST. The event will be held only online and by conference call.

A link to the live webcast will be available on Orion’s website at www.orion.fi/en/investors. A recording of the event will be available on the website later the same day.