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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]).

"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.

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]).

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.

Pulse Biosciences, Inc. Announces Redemption of Warrants

On January 5, 2021 Pulse Biosciences, Inc. (Nasdaq: PLSE) (the "Company" or "Pulse Biosciences"), a novel bioelectric medicine company progressing Nano-Pulse StimulationTM (NPS) technology, reported that on December 31, 2020, it delivered a notice of redemption to redeem all of its outstanding warrants (the "Warrants") to purchase shares of the Company’s common stock, $0.001 par value per share (the "Common Stock"), issued on June 16, 2020 in connection with the Company’s rights offering pursuant to its Registration Statement on Form S-3, as amended (File No. 333-237577), and that remain unexercised at 6:30 p.m., Eastern time, on February 5, 2021 (the "Redemption Date") for a redemption price of $0.01 per Warrant (the "Redemption Price") (Filing, 8-K, Pulse Biosciences, JAN 5, 2021, View Source [SID1234573487]).

Under the terms of the Warrants, the Company has the right to redeem all of the outstanding Warrants if the VWAP (as defined in the Warrants) exceeds $14.02 per share for ten consecutive trading days at least six months after the date that the Warrants were issued. The requirement was met for the each of the ten consecutive trading days preceding December 31, 2020. The Company had an average last reported sale price of $21.90 over this period.

The Warrants may be exercised by the holders thereof until 6:30 p.m. Eastern time on the Redemption Date at the exercise price of $7.01 per share of Common Stock. Any Warrants that remain unexercised at 6:30 p.m. Eastern time on the Redemption Date will be void and no longer exercisable, and the holders of those Warrants will be entitled to receive only the redemption price of $0.01 per Warrant. The Company received aggregate gross proceeds of $30 million from the rights offering, which was completed in June 2020 and, if all the Warrants are exercised prior to the Redemption Date, the Company will receive an additional $4.5 million of gross proceeds.

None of the Company, its board of directors or employees has made or is making any representation or recommendation to any holder of the Warrants as to whether to exercise or refrain from exercising any Warrants.

A registration statement, as amended, relating to the rights offering was previously filed with the Securities and Exchange Commission (the "SEC") and declared effective on May 8, 2020. A prospectus relating to the offering was filed with the SEC on May 14, 2020 and is available on the SEC’s website.

For a copy of the notice of redemption sent to the holders of the Warrants, please visit the Company’s investor relations website at investors.pulsebiosciences.com.

Questions concerning redemption and exercise of the Warrants can be directed to Broadridge Corporate Issuer Solutions, Inc., Attn: BCIS Re-Organization Dept., P.O. Box 1317, Brentwood, NY 11717-0718, telephone number (888) 789-8409.

No Offer or Solicitation

This press release shall not constitute an offer to sell or the solicitation of an offer to buy nor shall there be any offer of any of the Company’s securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction.