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SELLAS Announces Positive Follow-Up Phase 1/2 Clinical Data for Galinpepimut-S (GPS) in Acute Myeloid Leukemia (AML)

On February 26, 2020 SELLAS Life Sciences Group, Inc. (Nasdaq: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel cancer immunotherapies for a broad range of cancer indications, reported final follow-up data for its Phase 1/2 study of GPS in patients with acute myeloid leukemia (AML) in second complete remission (CR2) (Press release, Sellas Life Sciences, FEB 26, 2020, View Source [SID1234554788]). The final data show a median overall survival (OS) of 21.0 months, at a median follow-up of 30.8 months, in patients receiving GPS therapy compared to 5.4 months in the AML CR2 patients treated with best standard care, a statistically significant difference (p-value < 0.02). Final analysis also showed that GPS therapy continued to be well-tolerated throughout the study.

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"We’re extremely pleased with this follow-up data, which show that GPS may have potential as a longer-term therapy for AML patients in CR2, an aggressive disease where the majority of patients typically relapse and have a survival rate of approximately 5 months with best standard therapy," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "The 21-month survival data observed further increases our confidence in the potential of GPS as a maintenance treatment for AML patients in CR2, the same patient population as our pivotal Phase 3 study, known as REGAL."

"These follow-up data build upon the initially published clinical results from the Phase 1/2 study of GPS in AML patients in CR2 and provide further evidence that this novel immunotherapeutic vaccine approach may improve outcomes for patients in this setting, who often harbor measurable residual disease and have a poor prognosis if they are unable to undergo allotransplant," said Javier Pinilla-Ibarz, MD, PhD, Director of Immunotherapy for Malignant Hematology at the H. Lee Moffitt Cancer Center, and principal investigator of the Phase 1/2 study. "With this persistently positive efficacy signal, low toxicity burden, and CD4+ and CD8+ T cell responses, GPS has significant potential to serve as a maintenance therapy in AML patients in CR2, a patient population at great risk of leukemic relapse."

The Company previously reported initial data from the Phase 1/2 study of GPS in AML patients in CR2 at a median follow-up of 19.3 months, showing median OS in GPS-treated patients of 16.3 months vs. 5.4 months in a patient cohort contemporaneously treated with best standard therapy (p = 0.0175). The final analysis, at a median follow-up of 30.8 months, now shows a median OS of 21 months in the GPS-treated patient cohort.

"Given these results, it is particularly exciting to be involved in the ongoing pivotal Phase 3 REGAL study of GPS in AML patients in CR2," said Hagop M. Kantarjian, MD, Professor and Chair of the Department of Leukemia at the University of Texas – MD Anderson Cancer Center, and principal investigator of the Phase 3 REGAL study. "We are working to rapidly enroll patients who meet entry criteria for this study and believe these compelling results will enhance the visibility of this novel therapy and encourage broader participation in the pivotal Phase 3 trial. I look forward to initial results from the REGAL study, as I remain supportive of GPS’s potential promise as an immunotherapeutic agent in the AML CR2 setting."

SELLAS is currently enrolling patients in the ongoing Phase 3 REGAL study, a 1:1 randomized, open-label study comparing GPS monotherapy in the maintenance setting to investigators’ choice best available treatment in AML patients who have achieved hematologic complete remission, with or without thrombocytopenia (CR2/CR2p), after second-line antileukemic therapy and who are deemed ineligible for or unable to undergo allogeneic stem-cell transplantation. The primary endpoint is the OS from the time of study entry. Secondary endpoints include leukemia-free survival, antigen-specific T-cell immune response dynamics, measurable residual disease by multigene array, and assessments of AML clonal evolution and inflammasome molecular signatures in the tumor microenvironment in bone marrow biopsy samples. SELLAS expects an interim analysis for safety and futility in the fourth quarter of 2021.

Magenta Therapeutics to Participate in Cowen Health Care Conference on Monday, March 2nd in Boston

On February 26, 2020 Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune reset to more patients, reported that the company is scheduled to participate in a fireside chat at the 40th annual Cowen Health Care Conference on Monday, March 2nd, 2020, at 4:10 p.m. ET at the Boston Marriott Copley Place (Press release, Magenta Therapeutics, FEB 26, 2020, View Source [SID1234554787]).

A live webcast of the fireside chat can be accessed on the Magenta Therapeutics website at View Source The webcast replay will be available for 90 days following the event.

GT Biopharma Announces Dosing First Patient In GTB-3550 TriKE(TM) Phase I/II Clinical Trial

On February 26, 2020 GT Biopharma, Inc. (OTCQB:GTBP)(GTBP.PA) an immuno-oncology company focused on innovative treatments based on the Company’s proprietary NK cell engager (TriKE) platform reported that the first patient has been dosed in a Phase I/II clinical trial of its anti-CD16/IL-15/anti-CD33 TriKE, GTB-3550 (Press release, GT Biopharma, FEB 26, 2020, View Source [SID1234554786]). The clinical trial is being conducted at the University of Minnesota’s Masonic Cancer Center in Minneapolis, Minnesota under the direction of Dr. Erica Warlick.

The open-label, dose-escalation Phase I portion of the trial will evaluate GTB-3550 in patients with CD33-expressing, high risk myelodysplastic syndromes, refractory/relapsed acute myeloid leukemia or advanced systemic mastocytosis, and will determine safety and tolerability as well as the pharmacologically active dose and maximum tolerated dose of GTB-3550. The Phase II portion of the trial is planned to further evaluate the recommended dose of GTB-3550 in this patient population.

Multiple strategies to redirect immunity have been developed in the past two decades, but they have technical hurdles, cause undesirable side-effects, or are very expensive as exemplified by the T cell therapy-based chimeric antigen receptor (CAR-T) therapies from Bristol-Myers Squibb [NYSE: BMY] and Gilead Sciences [Nasdaq: GILD]). Because TriKE is a targeted immuno-oncology protein-based therapeutic, the cost and patient delivery/administration of TriKE therapy are far superior compared to CAR-T cell therapies.

Mr. Anthony Cataldo, the Chairman and Chief Executive Officer of GT Biopharma commented "we are pleased to have dosed the first patient in our GTB-3550 clinical trial, and thank our investors for helping us achieve this important milestone." Mr. Cataldo further stated "what makes the multi-targeting TriKE unique from all the other single targeting NK cell therapies (Affimed NV [Nasdaq: AFMD], Innate Pharma SA [Nasdaq: IPHA] and Dragonfly Therapeutics [www.dragonflytx.com]) is the proprietary incorporation of IL-15 (an NK cell stimulating cytokine) directly into the core TriKE construct thereby enhancing activation, proliferation and persistence of NK cells while minimizing the toxicities associated with the systemic administration of cytokine therapy to stimulate the patient’s immune system."

About GTB-3550 Trispecific NK cell Engager (TriKE)

GTB-3550 is the Company’s first TriKE product candidate being initially developed for the treatment AML. GTB-3550 is a single-chain, tri-specific scFv recombinant fusion protein conjugate composed of the variable regions of the heavy and light chains of anti-CD16 and anti-CD33 antibodies and a modified form of IL-15. The natural killer (NK) cell stimulating cytokine human IL-15 portion of the molecule provides a self-sustaining signal that activates NK cells and enhances their ability to kill. We intend to study GTB-3550 in CD33 positive leukemias such as acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and other CD33+ hematopoietic malignancies.

Jounce Therapeutics to Present at Upcoming Investor Conferences in March

On February 26, 2020 Jounce Therapeutics, Inc. (NASDAQ: JNCE), a clinical-stage company focused on the discovery and development of novel cancer immunotherapies and predictive biomarkers, reported that Jounce management will present at two upcoming investor conferences (Press release, Jounce Therapeutics, FEB 26, 2020, View Source [SID1234554785]):

Cowen & Co. 40th Annual Health Care Conferenceat 10:00 a.m. ET on Wednesday, March 4, 2020 in Boston, MA
32nd Annual ROTH Conferenceat 1:00 p.m. PT on Tuesday, March 17, 2020 in Orange County, CA
A live webcast of each presentation will be available by visiting "Events and Presentations" in the Investors and Media section of Jounce’s website at www.jouncetx.com. A replay of each webcast will be archived for 30 days following the presentations.

Madrigal Pharmaceuticals Reports 2019 Fourth Quarter and Full Year Financial Results and Highlights

On February 26, 2020 Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL) reported its fourth quarter and full year 2019 financial results and highlights (Press release, Synta Pharmaceuticals, FEB 26, 2020, View Source [SID1234554778]):

"Madrigal made significant progress during 2019 in executing our business strategy and advancing the development of resmetirom. We initiated two Phase 3 studies in NASH: the liver biopsy endpoint study, MAESTRO-NASH, and a non-invasive study in NAFLD patients with presumed NASH, MAESTRO-NAFLD-1," stated Paul Friedman, M.D., Chief Executive Officer of Madrigal. "We filled vital organizational needs including expansion of our medical operations team and the addition of Jim Daly, who has deep commercial expertise, to our Board. Further, we believe we have sufficient financial resources to fund our two ongoing Phase 3 clinical studies."

Becky Taub, M.D., CMO and President, Research & Development of Madrigal added, "According to plan, we initiated our Phase 3 MAESTRO-NASH clinical study in the first quarter, and our Phase 3 MAESTRO-NAFLD-1 study in the fourth quarter. Both Phase 3 studies are on track to complete enrollment this year for the 52 week readout by the end of 2021. In addition, MAESTRO-NAFLD-1 includes an open label active treatment arm that will provide data on lipids and non-invasive NASH biomarkers in 2020. We were also pleased with the publication of our successful Phase 2 NASH study in The Lancet in 2019. We continue to believe resmetirom has the potential to resolve NASH and reduce liver fibrosis while decreasing cardiovascular risk, through reduction of levels of multiple atherogenic lipids including LDL-C and triglycerides, and through the reduction of inflammatory fat in the liver. Realization of this potential could provide an important new therapy that delivers benefit to patients across the spectrum of early and late-stage NASH."

Financial Results for the Three Months and Twelve Months Ended December 31, 2019

As of December 31, 2019, Madrigal had cash, cash equivalents and marketable securities of $439.0 million, compared to $483.7 million at December 31, 2018. The decrease in cash and marketable securities resulted primarily from cash used in operations of $41.6 million.

Operating expenses were $30.0 million and $95.0 million, respectively, for the three month and twelve month periods ended December 31, 2019, compared to $14.5 million and $40.7 million in the comparable prior year periods.

Research and development expenses for the three month and twelve month periods ended December 31, 2019 were $24.9 million and $72.3 million, respectively, compared to $8.9 million and $25.4 million in the comparable prior year periods. The increases are primarily attributable to increases in clinical costs resulting from initiation of our Phase 3 studies, and personnel costs, including non-cash stock compensation.

General and administrative expenses for the three month and twelve month periods ended December 31, 2019 were $5.0 million and $22.6 million, respectively, compared to $5.6 million and $15.3 million in the comparable prior year periods. The decrease in general and administrative expenses for the latest three month period was due primarily to lower stock compensation expense, the effect of which was partially offset by higher personnel costs. The increase in general and administrative expenses for the latest twelve month period was due primarily to higher stock compensation, and personnel costs.

Interest income for the three month and twelve month periods ended December 31, 2019 was $2.2 million and $11.0 million, respectively, as compared to $3.0 million and $7.7 million in the comparable prior year periods. The decrease in interest income for the latest three month period was due primarily to lower average principal balances in our investment accounts in 2019, and lower interest rates. The increase in interest income for 2019 was due primarily to higher average principal balances in our investment accounts, the effects of which were partially offset by lower interest rates.

About resmetirom (MGL-3196)

Among its many functions in the human body, thyroid hormone, through activation of its beta receptor, plays a central role in controlling lipid metabolism, impacting a range of health parameters from levels of serum cholesterol and triglycerides to the pathological buildup of fat in the liver. Attempts to exploit this pathway for therapeutic purposes in cardio-metabolic and liver diseases have been hampered by the lack of selectivity of older compounds for the thyroid hormone receptor (THR)-ß, chemically-related toxicities and undesirable distribution in the body.

Madrigal recognized that greater selectivity for thyroid hormone receptor (THR)-ß and liver targeting might overcome these challenges and deliver the full therapeutic potential of THR-ß agonism. Madrigal believes that resmetirom is the first orally administered, small-molecule, liver-directed, truly ß-selective THR agonist.

Based on the positive Phase 2 clinical study results in patients with NASH (Phase 2 NASH 36-Week Results Press Release), Madrigal initiated a Phase 3 multinational, double-blind, randomized, placebo-controlled study of resmetirom in patients with non-alcoholic steatohepatitis (NASH) and fibrosis to resolve NASH and reduce progression to cirrhosis and/or hepatic decompensation (Phase 3 MAESTRO-NASH Initiation Press Release and ClinicalTrials.gov NCT03900429). Additionally, in both the NASH Phase 2 study, and a second positive Phase 2 clinical study in patients with heterozygous familial hypercholesterolemia (Phase 2 HeFH Results Press Release Phase 2 HeFH Results Press Release), significant reductions in multiple atherogenic lipids were observed. Based on the foregoing positive results, Madrigal also initiated MAESTRO-NAFLD-1, a 52-week, double-blind, placebo controlled Phase 3 clinical study in patients with biopsy-confirmed or presumed NASH (Phase 3 MAESTRO-NAFLD-1 Initiation Press Release and ClinicalTrials.gov NCT04197479). Key MAESTRO-NAFLD-1 endpoints are safety, including safety biomarkers, LDL cholesterol, lipid biomarkers, and fibrosis biomarkers. Except for serial liver biopsies, the study protocol is similar to the MAESTRO-NASH study and includes key secondary lipid, MRI-PDFF and NASH biomarker endpoints. In addition, MAESTRO-NAFLD-1 includes an open label arm in which up to 100 patients will be dosed with 100 mg resmetirom. The MAESTRO -NAFLD-1 study will help support the adequacy of the safety database at the time of NDA submission for subpart H approval for treatment of NASH in patients with F2 or F3 fibrosis (MAESTRO-NASH, NASH resolution surrogate endpoint).