On October 1, 2019 Ziopharm Oncology, Inc. ("Ziopharm" or "the Company") (Nasdaq:ZIOP), reported that the U.S. Food and Drug Administration (FDA) has cleared an investigational new drug application (IND) for a phase 1 clinical trial to evaluate CD19-specific CAR-T, produced using a process termed rapid personalized manufacture (RPM), as an investigational treatment for patients with relapsed CD19+ leukemias and lymphomas (Press release, Ziopharm, OCT 1, 2019, View Source [SID1234539974]).

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 IND clearance builds upon the Company’s experience with two prior generations of immunotherapy trials using the Sleeping Beauty platform, which it believes is the most clinically-advanced non-viral approach to the genetic modification of T cells. With this third-generation trial, DNA from the Sleeping Beauty system is stably inserted into the genome of resting T cells to co-express a chimeric antigen receptor (CAR), membrane-bound IL-15 (mbIL15) and a safety switch, which is designed to reduce cost, simplify production, and preserve the therapeutic potential of the T cells.

"There are currently no effective treatment options for patients who relapse soon after allogeneic bone marrow transplantation (BMT), as evidenced by their low rate of remission and poor long-term survival. This trial expands the range of patients with CD19-expressing malignancies that can be treated using the RPM technology," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of Ziopharm. "We believe RPM is the fastest approach to manufacturing and releasing CD19-specific CAR-T, as T cells from the blood stream are genetically reprogramed with the Sleeping Beauty system and then infused within two days of gene transfer. Existing commercial T-cell products using viral-based manufacturing are costly, time consuming to make and complex to deliver. We are now positioned to not only address those issues, but also to treat a patient group that remains underserved by existing therapies."

Up to 24 patients will be enrolled to evaluate infusion of donor-derived RPM CAR-T in patients with CD19+ leukemias and lymphomas who have relapsed after allogeneic BMT. This study will be conducted at The University of Texas MD Anderson Cancer Center under an investigator-initiated trial expected to begin later this year.

Research reveals three-year survival for adults with CD19+ acute lymphoblastic leukemia after allogeneic BMT ranges from 30% to 65%.1 For patients with other CD19+ cancers, allogeneic BMT can provide a three-year survival rates between 30% to 75%.1 Few patients experience a durable remission who relapse in the months following allogeneic BMT, regardless of the treatment modality, with some having a median survival of only 2 to 3 months.2

On October 1, 2019 Stemline Therapeutics, Inc. (Nasdaq: STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported updated clinical data on the felezonexor (SL-801) Phase 1 trial in patients with advanced solid tumors at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) 2019 Annual Congress in Barcelona, Spain (Press release, Stemline Therapeutics, OCT 1, 2019, View Source [SID1234539973]). The presentation is now available on the Stemline website, www.stemline.com, under the Scientific Presentations tab.

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!

Felezonexor is a novel, oral, small molecule that reversibly inhibits Exportin-1 (XPO1), a nuclear export protein, overexpressed in a variety of solid and hematologic malignancies. XPO1 is a mediator of nuclear-cytoplasmic transport of nuclear proteins and has been associated with aggressive tumor behavior and poor prognosis. XPO1 is a clinically validated target in oncology, and the FDA recently approved an XPO1 inhibitor drug, in combination with other agents, for the treatment of certain oncology patients (with relapsed/refractory multiple myeloma). Felezonexor has demonstrated potent in vitro and in vivo preclinical activity. Interim results from the ongoing Phase 1 clinical trial reported at ESMO (Free ESMO Whitepaper) are summarized below.

Key Felezonexor Highlights from Ongoing Phase 1 Trial in Patients with Advanced Solid Tumors

Partial response (PR) achieved with single agent felezonexor in a 4th line patient with KRAS-positive, microsatellite stable (MSS) colorectal cancer (18+ weeks, ongoing)
– PR (RECIST 1.1 criteria) reported after 2 cycles of felezonexor (70mg then 65mg due to elevated creatinine), with the patient demonstrating serial reductions in the two target lesions (liver and spleen)
– Treatment with felezonexor ongoing (cycle 6); Next staging pending

Stable disease (SD) achieved in 12 patients, with 11/12 of these patients 3rd line or greater

Five patients had SD for 4 and 11 months, including 1 patient with basal cell carcinoma with SD for ~11 months
– 20% disease shrinkage noted in one patient with heavily pre-treated neuroendocrine tumor

Pharmacokinetic (PK) analyses suggest dose-dependent increases in exposure

Dosing regimen previously adjusted (Schedule B) to improve tolerability while maintaining dose intensity; 1 patient in Schedule B (n=7) experienced grade 3 weakness, the 75mg cohort has been expanded and enrollment continues

Ideal therapeutic dose and regimen not yet determined, and dose escalation ongoing

Further updates expected as Phase 1 trial continues to enroll

Ivan Bergstein, M.D., CEO of Stemline, commented, "We are very encouraged to witness evidence of clinical activity in a patient with a heavily pretreated and highly mutated solid tumor with poor prognostic features with single agent felezonexor in a regimen and dose that has potentially not yet been fully optimized. We continue to enroll patients with the goal of refining the ideal dose and schedule to take forward into a focused and abbreviated Phase 2 program, and plan to provide periodic updates as the Phase 1 progresses."

About ELZONRIS
ELZONRIS (tagraxofusp-erzs), a CD123-directed cytotoxin, is approved by the U.S. Food and Drug Administration (FDA) and commercially available in the U.S. for the treatment of adult and pediatric patients, two years or older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). For full prescribing information in the U.S., visit www.ELZONRIS.com. In Europe, a marketing authorization application (MAA) is under review by the European Medicines Agency (EMA). ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).

About BPDCN
BPDCN is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, as well as other markers. For more information, please visit the BPDCN disease awareness website at www.bpdcninfo.com.

About CD123
CD123 is a cell surface target expressed on a wide range of myeloid tumors including blastic plasmacytoid dendritic cell neoplasm (BPDCN), certain myeloproliferative neoplasms (MPNs) including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML) (and potentially enriched in certain AML subsets), myelodysplastic syndrome (MDS), and chronic myeloid leukemia (CML). CD123 has also been reported on certain lymphoid malignancies including multiple myeloma (MM), acute lymphoid leukemia (ALL), hairy cell leukemia (HCL), Hodgkin’s lymphoma (HL), and certain Non-Hodgkin’s lymphomas (NHL). In addition, CD123 has been detected on some solid tumors as well as autoimmune disorders including cutaneous lupus and scleroderma.

On October 1, 2019 Replimune Group, Inc. (NASDAQ: REPL), a biotechnology company developing oncolytic immuno-gene therapies derived from its Immulytic platform, reported that Robert Coffin, Ph.D., Chief Executive Officer and Director of Replimune, will present and host one-on-one meetings at Chardan’s 3rd Annual Genetic Medicines Conference being held in New York City (Press release, Replimune, OCT 1, 2019, View Source [SID1234539972]). The Company is scheduled to present on Monday, October 7, 2019 at 4:15 PM ET.

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 October 1, 2019 Oncolytics Biotech Inc. (NASDAQ:ONCY) (TSX:ONC), currently developing pelareorep, an intravenously delivered immuno-oncolytic virus, reported the acceptance of an abstract highlighting data from the AWARE-1 study to be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) (Press release, Oncolytics Biotech, OCT 1, 2019, View Source [SID1234539971]). The 34th Annual SITC (Free SITC Whitepaper) Meeting will be held November 6-10, 2019 at the Gaylord National Hotel & Convention Center in National Harbor, Maryland.

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 abstract, A window-of-opportunity Study of pelareorep in Early Breast Cancer (AWARE-1), was authored by AWARE-1 principal investigator, Aleix Prat, et al. Dr. Prat is Head of Medical Oncology at the Hospital Clínic of Barcelona, Associate Professor of the University of Barcelona and the Head of the Translational Genomics and Targeted Therapeutics in Solid Tumors Group at August Pi i Sunyer Biomedical Research Institute (IDIBAPS) and member of Oncolytics’ Scientific Advisory Board.

Abstracts will be published on the SITC (Free SITC Whitepaper) 2019 website at 8:00 a.m. ET on Tuesday, November 5, 2019.

The poster will be presented on Friday, November 8 between 7:00 a.m. and 8:00 p.m. ET.

About Pelareorep

Pelareorep is a non-pathogenic, proprietary isolate of the unmodified reovirus: a first-in-class intravenously delivered immuno-oncolytic virus for the treatment of solid tumors and hematological malignancies. The compound induces selective tumor lysis and promotes an inflamed tumor phenotype through innate and adaptive immune responses to treat a variety of cancers and has been demonstrated to be able to escape neutralizing antibodies found in patients.

On October 1, 2019 Merck (NYSE:MRK), known as MSD outside the United States and Canada, reported that it will hold its third-quarter 2019 sales and earnings conference call with institutional investors and analysts at 8:00 a.m. EDT on Tuesday, Oct. 29 (Press release, Merck & Co, OCT 1, 2019, View Source [SID1234539970]). During the call, company executives will provide an overview of Merck’s performance for the quarter.

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!

Investors, journalists and the general public may access a live audio webcast of the call on Merck’s website at View Source A replay of the webcast, along with the sales and earnings news release and supplemental financial disclosures, will be available at www.merck.com.

Institutional investors and analysts can participate in the call by dialing (706) 758-9927 or (877) 381-5782 and using ID code number 5635157. Members of the media are invited to monitor the call by dialing (706) 758-9928 or (800) 399-7917 and using ID code number 5635157. Journalists who wish to ask questions are requested to contact a member of Merck’s Media Relations team at the conclusion of the call.