Vaccinex to Participate in the LifeSci Partners 10th Annual Healthcare Corporate Access Event

On January 5, 2021 Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating cancer and neurodegenerative disease through the inhibition of SEMA4D, reported that it will participate in the 10th Annual LifeSci Partners Corporate Access Event, January 6-8 and 11-14, 2021 (Press release, Vaccinex, JAN 5, 2021, View Source [SID1234573492]).

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Maurice Zauderer, Ph.D., President and CEO, will host 1×1 meetings and will present a corporate update on Wednesday, January 6th at 11am EST.

To register to listen to the presentation or to request a meeting, visit: View Source

Ultragenyx to Present at 39th Annual J.P. Morgan Virtual Healthcare Conference

On January 5, 2021 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, reported that Emil D. Kakkis, M.D., Ph.D., the company’s Chief Executive Officer and President, will present at the 39TH Annual J.P. Morgan Virtual Healthcare Conference on Tuesday, January 12, 2021 at 2:50 pm EST (Press release, Ultragenyx Pharmaceutical, JAN 5, 2021, View Source [SID1234573491]).

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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.

Marker Therapeutics Announces FDA Lifted Partial Clinical Hold on Phase 2 AML Clinical Trial

On January 5, 2021 Marker Therapeutics, Inc. (Nasdaq:MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, reported that the U.S. Food and Drug Administration (FDA) lifted the partial clinical hold on the Company’s Phase 2 trial investigating the safety and efficacy of MT-401, Marker’s lead multi-tumor-associated antigen (MultiTAA)-specific T cell product candidate for the treatment of post-transplant acute myeloid leukemia (AML) (Press release, TapImmune, JAN 5, 2021, View Source [SID1234573490]).

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"We are pleased to move forward with our Phase 2 AML trial of MT-401, which we believe may provide a safe and effective treatment option for patients with post-transplant AML over the standard of care," said Mythili Koneru, M.D., Ph.D., Chief Medical Officer of Marker Therapeutics. "During the partial clinical hold, we continued to open clinical centers to enroll patients in the first half of the safety lead-in of our Phase 2 trial. With the FDA’s decision, we will now be able to seamlessly enroll patients in the second half of the safety lead-in, as well as the remainder of the trial. We look forward to working with our clinical sites to continue enrolling patients."

The multicenter Phase 2 AML study is evaluating clinical efficacy of MT-401 in patients with AML in both the adjuvant and active disease setting, following an allogeneic stem-cell transplant. In the adjuvant setting, approximately 120 patients will be randomized 1:1 to either MT-401 at 90 days post-transplant versus standard-of-care observation, while about 40 patients with active disease will receive MT-401 as part of the single-arm group. The trial also includes a safety lead-in expected to enroll six patients.

The primary objectives of the trial are to evaluate relapse-free survival in the adjuvant group and determine the complete remission rate and duration of complete remission in active disease patients. Additional objectives include, for the adjuvant group, overall survival and graft-versus-host disease relapse-free survival while additional objectives for the active disease group include overall response rate, duration of response, progression-free survival and overall survival.

In April 2020, the FDA granted Orphan Drug designation to MT-401 for the treatment of patients with AML following allogeneic stem cell transplant.

Replimune Announces First Patient Dosed with RP3 in a Phase 1 Clinical Trial in Patients with Advanced Solid Tumors

On January 5, 2021 Replimune Group, Inc. (NASDAQ: REPL), a biotechnology company developing oncolytic immuno-gene therapies derived from its Immulytic platform, reported it has dosed the first patient in a Phase 1, open-label, dose-escalation and expansion clinical trial of RP3 both alone and in combination with anti-PD-1 therapy (Press release, Replimune, JAN 5, 2021, View Source [SID1234573489]). RP3, Replimune’s third product candidate to enter clinical development, encodes for CD40L and 4-1BBL in addition to the GALV-GP R(-) fusogenic protein encoded in RP1 and the anti-CTLA-4 molecule encoded in RP2. Expression of CD40L and 4-1BBL are intended to further stimulate an anti-tumor immune response through immune co-stimulatory pathway activation.

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Replimune’s product candidates are designed to comprehensively activate an anti-tumor immune response to a patient’s cancer through the delivery of potent immune activating signals into the tumor, such that each of the stages of immune activation are maximized. The signals provided are (i) potent antigen release and presentation, so-called immunologic "Signal 1", provided by robust tumor destruction and immunogenic cell death through the use of the high potency oncolytic backbone expressing the GALV-GP R- fusogenic protein in all of our product candidates; (ii) the inhibition of signals which block full immune activation, provided by expression of anti-CTLA-4 in both RP2 and RP3; (iii) provision of potent immune co-stimulatory signals, so-called immunologic "Signal 2", provided by expression of CD40L and 4-1BBL in RP3; and (iv) the production of inflammatory cytokines, so-called immunologic "Signal 3", also intended to be stimulated by the expression of CD40L and 4-1BBL in RP3. RP3 therefore represents the culmination of this approach to date, and is intended to build on the compelling clinical data generated with RP1 and RP2, including in immune non-responsive tumor types.

"We are pleased to be advancing our third oncolytic immune-gene therapy into the clinic," said Robert Coffin, PhD, Founder, President and Chief Research & Development Officer of Replimune. "Having demonstrated the potential of our platform through positive clinical data readouts with both RP1 and RP2 in multiple tumor types, we have armed RP3 to express additional immune-activating proteins intended to further enhance the ability of our product candidates to treat less immune-responsive tumor types. We believe that the combined properties of RP3 are unique in the field of immune oncology and demonstrate the power of our platform to generate product candidates with multiple mechanisms of action which together potently activate a patient’s immune system against their cancer. We look forward to providing initial data with RP3, along with our other clinical programs over the course of 2021."

The Phase 1, dose-escalation clinical trial is currently enrolling patients with advanced solid tumors. The clinical trial is primarily designed to assess safety and tolerability of RP3 and to determine the recommended Phase 2 dose. Following dose selection, the second part of the clinical trial is intended to dose patients with RP3 in combination with anti-PD1 therapy to further assess for both safety and initial efficacy.

Regulus Therapeutics to Present at the H.C. Wainwright Virtual BioConnect Conference

On January 5, 2021 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs ("Regulus"), reported that Jay Hagan, President and Chief Executive Officer of Regulus, will present at the H.C. Wainwright Virtual BioConnect Conference being held from January 11-14, 2021 (Press release, Regulus, JAN 5, 2021, View Source [SID1234573488]).

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A webcast of the on-demand presentation will be available beginning Monday, January 11, 2021 through the investor relations section of the Company’s website at www.regulusrx.com.