On September 4, 2019 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported the initiation of two clinical trials for IPI-549, a first-in-class, oral immuno-oncology product candidate targeting immune-suppressive tumor-associated myeloid cells through selective inhibition of phosphoinositide-3-kinase (PI3K)-gamma (Press release, Infinity Pharmaceuticals, SEP 4, 2019, View Source [SID1234539250]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Infinity initiated MARIO-3, a Phase 2 multi-arm study in collaboration with Roche/Genentech evaluating IPI-549 in combination with Tecentriq and Abraxane (nab-paclitaxel) in front-line triple negative breast cancer (TNBC) and in combination with Tecentriq. Following the results from Roche’s IMpassion130 study in which Tecentriq and Abraxane received accelerated approval in PDL1+ TNBC patients, MARIO-3 is evaluating the addition of PI-549 to this regimen in both PDL1+ and PDL1- front-line TNBC patients. MARIO-3 also includes a cohort evaluating IPI-549 in combination with Tecentriq and Avastin (bevacizumab) in front-line PDL1+ and PDL1- renal cell cancer (RCC) patients.

Additionally, Arcus Biosciences initiated a Phase 1/1b study, in collaboration with Infinity, evaluating IPI-549 in a novel combination regimen with AB298, Arcus’s dual adenosine receptor antagonist, and Doxil, a chemotherapy, in patients with advanced TNBC.

"The initiation of these two trials deepens Infinity’s commitment to bringing novel and potentially transformative regimens to patients with some of the most challenging to treat cancers," said Sam Agresta, Chief Medical Officer of Infinity Pharmaceuticals. "By partnering with leading pharmaceutical and biotech companies, Infinity is advancing IPI-549 into additional indications, earlier lines of therapy and novel combination treatment regimens with best-in-class therapies to maximize clinical potential. Our team has now successfully initiated three clinical trials this year: MARIO-275, MARIO-3 and the collaboration study with Arcus which, in total with MARIO-1, are projected to enroll approximately 500 patients. This is a tremendous accomplishment for Infinity and a testament to our ability to execute on clinical trials designed to transform the treatment landscape for those patients with significant unmet need."

About AB928

AB928 is an orally bioavailable, highly potent antagonist of the adenosine 2a and 2b receptors. The activation of these receptors by adenosine interferes with the activity of key populations of immune cells (e.g. T cells, NK cells) and inhibits their optimal anti-tumor immune response. By blocking these receptors, AB928 has the potential to reverse adenosine-induced immune suppression within the tumor microenvironment. AB928 was designed specifically for the oncology setting, with a profile that includes potent activity in the presence of high concentrations of adenosine and a minimal shift in potency due to non-specific protein binding, both essential properties to be efficacious in the tumor microenvironment. AB928 has other attractive features, including high penetration of tumor tissue. In a Phase 1 trials in healthy volunteers and oncology patients, AB928 has been shown to be well tolerated and to have pharmacokinetic and pharmacodynamic profiles consistent with a once-daily dosing regimen.

On September 3, 2019 Alector, Inc. (Nasdaq: ALEC), a clinical stage biotechnology company pioneering immuno-neurology, reported that Arnon Rosenthal, Ph.D., chief executive officer of Alector will participate in a fireside chat at the 17th Annual Morgan Stanley Global Healthcare Conference on Tuesday, Sept. 10, at 12:55 p.m. ET in New York City (Press release, Alector, SEP 3, 2019, View Source [SID1234551116]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

To access the audio portion of the fireside chat, please visit the "Events & Presentations" page within the Investors section of the Alector website at View Source A replay will be available on the Alector website for 90 days following the conference.

On September 3, 2019 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported that the journal Nature Medicine has published both pre-clinical results and clinical data from the ongoing Phase I CARPALL trial of AUTO1, demonstrating the potential of the company’s novel CAR T therapy targeting CD19 in development for the treatment of pediatric acute lymphoblastic leukemia (ALL) (Press release, Autolus, SEP 3, 2019, View Source [SID1234550686]). The paper reports that AUTO1, or CAT Chimeric Antigen Receptor T cells (CAT CAR T), utilizes a binder with a fast off rate and showed both increased proliferation/cytotoxicity in vitro and enhanced proliferative capacity and anti-tumor activity when compared to FMC63 CAR T therapies in vivo. In the Phase 1 clinical trial, 86% (n=14) of recurrent/refractory pediatric ALL patients achieved molecular complete remission after a single dose, with a median duration of remission of 7.4 months and no severe cytokine release syndrome (CRS; ≥ grade 3 or 4), in this relapsed and/or refractory patient population.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The safety profile emerging from this pediatric study is encouraging. AUTO1 was well-tolerated and we did not see severe cytokine release syndrome or neurotoxicity seen in other ALL programs," said Sara Ghorashian, PhD, Molecular and Cellular Immunology Section, UCL Great Ormond Street Institute of Child Health and a co-author of the paper. "It is very promising to see these strong remission rates and excellent CAR T cell expansion and persistence, which give us hope that AUTO1 could improve outcomes for these patients."

"The publication in Nature Medicine is a nice validation of our AUTO1 pre-clinical and Phase 1 clinical data," said Dr. Christian Itin, chairman and chief executive officer of Autolus. "AUTO1 CAR- T cells are designed to effectively engage leukemic cells while avoiding excessive immune stimulation. This profile results in an improved safety profile compared to current treatments, while achieving a high level of clinical activity. We are currently testing the activity of AUTO1 in adult patients who typically are even more susceptible to severe immunological adverse events than pediatric patients."

About the Studies

The Nature Medicine publication includes a discussion of both pre-clinical and clinical studies. The pre-clinical assessment included evaluation of in vitro responses of AUTO1 versus FMC63 CAR T cells (cytotoxicity, proliferation and cytokine production) as well as a comparison of their anti-tumor efficacy within a xenogeneic model of ALL.

CARPALL (NCT02443831) is a multi-center, non-randomized, open label Phase I clinical trial of heavily pre-treated patients under age 24 with high risk and relapsed CD19+ ALL. Enrolled patients had a median age of 9 years with a median of 4 lines of prior treatment. Seventeen patients were enrolled, and 14 patients received an infusion of CAR T cells. Ten of 14 patients had relapsed post allogeneic stem cell transplant. Eight patients were treated in second relapse, 5 in >second relapse and 3 had relapsed after prior blinatumomab or inotuzumab therapy. Two patients had ongoing CNS disease at enrollment.

This data set confirms that AUTO 1 induces no severe CRS (Grade 3-5). Nine patients experienced Grade 1 CRS, and 4 patients experienced Grade 2 CRS. No patients required tociluzumab or steroids. As previously reported, one patient experienced Grade 4 neurotoxicity; there were no other reports of severe neurotoxicity (Grade 3-5). Eleven patients experienced cytopenia that was not resolved by day 28 or recurring after day 28: 3 patients Grades 1-3 and 8 patients Grade 4. Two patients developed significant infections, and 1 patient died from sepsis while in molecular complete response (CR).

With a single dose of CAR T cells at 1 million cells/kg dose, 12/14 (86%) achieved molecular CR. Five patients relapsed with CD19 negative disease. Event free survival (EFS) based on morphological relapse was 67% (CI 34-86%) and 46% (CI 16-72%) and overall survival (OS) was 84% (CI 50-96%) and 63% (CI 27-85%) at 6 and 12 months, respectively.

CAR T cell expansion was observed in all responding patients (N=12), with CAR T cells comprising up to 84% of circulating T cells at the point of maximal expansion. The median persistence of CAR T was 215 days.

The median duration of remission in responding patients was 7.4 months with a median follow-up of 14 months. Five of 14 patients (37%) remain in CR with ongoing persistence of CAR T cells and associated B cell aplasia.

About AUTO1

AUTO1 is a CD19 CAR T cell investigational therapy designed to overcome the limitations in safety – while maintaining similar levels of efficacy – compared to current CD19 CAR T cell therapies. Designed to have a fast target binding off-rate to minimize excessive activation of the programmed T cells, AUTO1 may reduce toxicity and be less prone to T cell exhaustion, which could enhance persistence and improve the T cells’ abilities to engage in serial killing of target cancer cells. In 2018, Autolus signed a license agreement under which Autolus acquired global rights from UCL Business plc (UCLB), the technology-transfer company of UCL, to develop and commercialize AUTO1 for the treatment of B cell malignancies. AUTO1 is currently being evaluated in two Phase 1 studies, one in pediatric ALL and one in adult ALL.

About Pediatric Acute Lymphoblastic Leukemia (ALL)

According to the American Cancer Society, ALL is the most common cancer diagnosed in children, with approximately 3,400 new cases diagnosed in the United States each year. Pediatric ALL occurs when the bone marrow makes too many immature lymphocytes, which are a type of white blood cell. The current standard of care for pediatric ALL patients is combination chemotherapy. Although pediatric patients typically respond well to first-line treatment, 10 to 20% of total patients relapse with chemotherapy-resistant disease, leading to a significant unmet need in pediatric patients with high-risk relapsed or refractory ALL.

On September 3, 2019, AIM ImmunoTech Inc. (the "Company") reported its corporate presentation (Presentation, Hemispherx Biopharma, SEP 3, 2019, View Source [SID1234539394]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

On September 3, 2019 Cytokinetics, Incorporated (Nasdaq: CYTK) reported that Robert I. Blum, President and Chief Executive Officer, is reported to present a corporate update at the following investor conferences (Press release, Cytokinetics, SEP 3, 2019, View Source [SID1234539268]):

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

17th Annual Morgan Stanley Global Healthcare Conference on Monday, September 9, 2019 at 8:10 AM ET at the Grand Hyatt Hotel in New York City
21st Annual H. C. Wainwright Global Investment Conference on Tuesday, September 10, 2019 at 1:20 PM ET at the Lotte New York Palace Hotel in New York City
Interested parties may access the live webcast of these presentations by visiting the Investors & Media section of the Cytokinetics website at www.cytokinetics.com. The webcast replay of each presentation will be archived on the Presentations page within the Investors & Media section of Cytokinetics’ website for 90 days following the conclusion of each event.