Sarepta Therapeutics to Present at the 42nd Annual J.P. Morgan Healthcare Conference

On January 2, 2024 Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, reported that senior management will present at the 42nd Annual J.P. Morgan Healthcare Conference in San Francisco, Calif. on Monday, Jan. 8, 2024 at 12:00 p.m. E.T. / 9:00 a.m. P.T (Press release, Sarepta Therapeutics, JAN 2, 2024, View Source [SID1234638880]). Following the presentation there will be a Q&A session starting at 12:20 p.m. E.T. / 9:20 a.m. P.T.

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The presentation will be webcast live under the Events & Presentations section of the investor relations section of Sarepta’s website at View Source and will be archived there following the presentation for 90 days. Please connect to Sarepta’s website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.

Leap Therapeutics Announces Completion of Enrollment in Randomized Controlled Part C of the DisTinGuish Study of DKN-01 for the Treatment of Gastric Cancer Patients

On January 2, 2024 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, reported that enrollment has been completed in the randomized controlled Part C of the DisTinGuish study evaluating DKN-01, Leap’s anti-Dickkopf-1 (DKK1) antibody, in combination with tislelizumab, BeiGene’s anti-PD-1 antibody, and chemotherapy in patients with advanced gastroesophageal junction and gastric cancer (Press release, Leap Therapeutics, JAN 2, 2024, View Source [SID1234638879]).

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"The completion of enrollment in Part C of the DisTinGuish study is an important milestone and continues to underscore the high level of interest from both patients and investigators in DKN-01," said Cynthia Sirard, M.D., Chief Medical Officer of Leap. "Long-term follow-up data from Part A of the study clearly demonstrated the potential to induce durable responses and clinically meaningful survival outcomes in first-line advanced gastroesophageal junction and gastric cancer patients. We expect Part C to further validate the potential of DKN-01 and tislelizumab combination therapy and look forward to having initial data from Part C over the course of this year."

Part C of the DisTinGuish study (NCT0436380) is a Phase 2, randomized, open-label, multicenter study of DKN-01 in combination with tislelizumab and chemotherapy in first-line patients with advanced gastroesophageal adenocarcinoma. Part C enrolled 170 first-line, HER2-negative patients. Patients were randomized 1:1 to evaluate DKN-01 in combination with tislelizumab and standard of care (SOC) chemotherapy, compared to tislelizumab and SOC chemotherapy alone. The primary objective is progression-free survival (PFS) in DKK1-high and in all patients. Secondary objectives of Part C include overall survival and objective response rate as measured by RECIST v1.1 in DKK1-high and in all patients.

INmune Bio Announces First Patient Dosed in a Phase 1/2 Study of INKmune™ in Patients with Metastatic Castration-Resistant Prostate Cancer

On January 2, 2024 INmune Bio, Inc. (NASDAQ: INMB) (the "Company"), a clinical-stage immunology company focused on developing treatments that harness the patient’s innate immune system to fight disease, reported to have dosed the first patient in the Phase I/II trial in men with metastatic castration-resistant prostate cancer (mCRPC) on December 27, 2023 (Press release, INmune Bio, JAN 2, 2024, View Source [SID1234638875]). INKmune is a biologic therapy given as out-patient therapy without the need for pre-medication or cytokine support.

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"Results of immunotherapy trials for mCRPC have been disappointing," said Professor Matt Rettig, Professor of Medicine and Urology, Medical Director of the Prostate Cancer Program at the David Geffen School of Medicine at UCLA and member of the Jonsson Comprehensive Cancer Center. "After many failures using T-cell focused immunotherapy approaches, targeting NK cells — which are abundant in the prostate cancer tumor microenvironment — is a promising and novel strategy. I am optimistic about the therapeutic potential of INKmune, an off-the-shelf innate immune therapy." Professor Rettig was deeply involved in the design of the clinical trial. Dr. Rettig’s statements should not be construed as endorsement by the University of California.

CaRe PC is an open label Phase I/II trial that will test up to three doses of INKmune in men with mCRPC. INKmune is given as out-patient therapy via intravenous infusion three times in the first two weeks of treatment (days 1, 8 and 15). The patient is followed for six months with careful study of immunologic and anti-cancer responses to INKmune treatment. Immune responses include changes in numbers of tumor killing memory-like NK cells in the patient’s blood and how long these specialized NK cells remain in the circulation. Anti-tumor responses will be monitored by following the level of prostatic surface antigen (PSA) in the blood, as well as using Artificial Intelligence (AI) to quantify the number and size of metastatic lesions using piflufolastat F 18 – a PSMA (prostate-specific membrane antigen) imaging agent developed by Lantheus, and by measuring circulating tumor DNA (ctDNA) in the blood. As many as 30 patients will receive one of three levels of dose of INKmune (low, medium, high).

"There are two key elements for successful immunotherapy. There must be immune cells in the tumor and the drug must convert those immune cells into cancer killing cells," said Prof. Mark Lowdell Ph.D., CSO of INmune Bio and inventor of INKmune. "Prostate cancer has many resting NK cells, and we believe INKmune will convert those resting NK cells into memory-like NK cells that can attack the tumor."

The study uses a novel modified Bayesian design. The sequential Phase I dose escalation portion will be followed by a Phase II trial that will simultaneously enroll patients in all dosing cohorts. Once the Phase I portion is complete, the doses that are safe will be tested simultaneously in the Phase II portion of the trial. Up to 10 patients will be enrolled at each dose level. There are two primary goals of the trial. The first is to demonstrate the safety of INKmune in the patient population (men with mCRPC). The second is to determine which dose of INKmune should be used in a blinded, randomized registration trial. Determining the best dose of INKmune to use in future clinical trials will depend on a combination of immunologic and anti-tumor responses seen in the men treated with INKmune therapy.

Ultragenyx to Present at the 42nd Annual J.P. Morgan Healthcare Conference

On January 2, 2024 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, reported that Emil D. Kakkis, M.D., Ph.D., the company’s chief executive officer and president, will present at the 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024, at 3:00 PM PT (Press release, Ultragenyx Pharmaceutical, JAN 2, 2024, View Source [SID1234638873]).

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The live and archived webcast of the presentation will be accessible from the company’s website at View Source The replay of the webcast will be available for 30 days.

Syndax Announces Presentation at 42nd Annual J.P. Morgan Healthcare Conference

On January 2, 2024 Syndax Pharmaceuticals (Nasdaq: SNDX), a clinical-stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that Michael A. Metzger, Chief Executive Officer of Syndax, will present at the 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024 at 5:15 p.m. PT/ 8:15 p.m. ET (Press release, Syndax, JAN 2, 2024, View Source [SID1234638872]).

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A live webcast of the fireside chat can be accessed from the Investor section of the Company’s website at www.syndax.com, where a replay of the event will also be available for a limited time.