On October 1, 2019 Jounce Therapeutics, Inc. (NASDAQ: JNCE), a clinical-stage company focused on the discovery and development of novel cancer immunotherapies and predictive biomarkers, reported that safety and efficacy data for JTX-4014 and a clinical trial in progress description of the vopratelimab Phase 2 EMERGE study will be presented in two poster sessions at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 34th Annual Meeting, being held November 6-10, 2019 in National Harbor, Maryland (Press release, Jounce Therapeutics, OCT 1, 2019, View Source [SID1234539979]).

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Poster Details:

Poster Title: Phase 1 First in Human Study of Programmed Cell Death Receptor-1(PD-1) Inhibitor Monoclonal Antibody (mAb) JTX-4014 in Adult Subjects with Advanced Refractory Solid Tumor Malignancies (Safety, efficacy and recommended Phase 2 dose will be presented)
Author: Kyriakos P. Papadopoulos, M.D. Co-Director of Clinical Research, START, San Antonio, Texas
Poster Session Number: P439
Location: Poster Hall (Prince George AB)
Date and Time: Friday, November 8, 2019; 7:00am–8pm ET

Poster Title: Phase 2 Multicenter Trial of ICOS Agonist Vopratelimab and a CTLA-4 Inhibitor in PD-1/PD-L1 Inhibitor Experienced Adult Subjects with Non-Small Cell Lung Cancer or Urothelial Cancer (EMERGE) (This is a trial in progress poster only)
Author: Russell Pachynski M.D., Assistant Professor of Medicine, Division of Medical Oncology, Washington University School of Medicine, St. Louis, Missouri
Poster Session Number: P438
Location: Poster Hall (Prince George AB)
Date and Time: Saturday, November 9, 2019; 7:00am–8:30pm ET

About JTX-4014
JTX-4014 is a well-characterized fully human IgG4 monoclonal antibody designed to block binding to PD-L1 and PD-L2. Jounce is developing JTX-4014 for potential use in combination with its pipeline of future product candidates. Jounce completed enrollment in the Phase 1 clinical trial of JTX-4014.

About Vopratelimab
Jounce’s lead product candidate, vopratelimab (formerly JTX-2011), is a clinical-stage monoclonal antibody that binds to and activates ICOS, the Inducible T cell CO Stimulator, a protein on the surface of certain T cells commonly found in many solid tumors. Vopratelimab was previously assessed in the Phase 1/2 ICONIC trial and was found to be safe and well-tolerated, alone and in combination with each of the anti-PD-1 antibodies nivolumab and pembrolizumab, and ipilimumab, an antibody that binds to CTLA-4. In June 2018, Jounce reported Response Evaluation Criteria in Solid Tumors (RECIST) responses and other tumor reductions as determined by investigator assessment that were associated with an ICOS pharmacodynamic biomarker, ICOS hi CD4 T cells. In April 2019, Jounce reported data on patients in the ICONIC trial with the emergence of ICOS hi CD4 T cells who had improved progression free survival and overall survival compared to patients with ICOS lo CD4 T cells; this was based on an analysis of a subgroup of patients with multiple solid tumor types including PD-1 inhibitor naive and PD-1 inhibitor experienced patients. Vopratelimab is currently being assessed in the Phase 2 EMERGE clinical trial in combination with ipilimumab in patients with non-small cell lung cancer or urothelial cancer who have progressed on or after PD-1/PD-L1 inhibitor therapies.

On October 1, 2019 Cellectis (Paris:ALCLS) (NASDAQ:CLLS) (Euronext Growth: ALCLS – Nasdaq: CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on allogeneic gene-edited CAR T-cells (UCART), and Lonza (SWX: LONN), reported that the companies have entered into a manufacturing service agreement covering clinical manufacturing of Cellectis’ allogeneic UCART product candidates targeting hematological malignancies (Press release, Cellectis, OCT 1, 2019, View Source [SID1234539976]). Lonza is in charge of implementing Cellectis’ manufacturing processes as per current Good Manufacturing Practices (cGMP) in a way that meets the highest quality and safety standards outlined by the FDA. The manufacturing will take place at Lonza’s GMP facility in Geleen, Netherlands.

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William Monteith, Executive Vice President, Technical Operations, Cellectis:

"Working with Lonza, a world-class solutions provider with deep experience in the biotech and pharma industries increases our global capabilities and allows Cellectis to further strengthen its manufacturing expertise. This agreement not only bolsters our product supply for clinical trials, but it ensures that we are producing first-rate product candidates so that we can potentially deliver new hope to patients living with certain blood cancers."

Alberto Santagostino, Senior Vice President, Head of Cell & Gene Technologies, Lonza:

"Early-stage innovators with great science, like Cellectis, can find an ideal partner in Lonza as we bring great value in technical development and manufacturing, industrializing processes and enabling the journey to commercialization. We will draw on the experience at our cell and gene therapy center of excellence in the Netherlands, ideally equipped to support Cellectis in bringing their promising pipeline of allogeneic CAR-T therapies to people around the world in need of life-saving products."

Lonza’s supply will complement Cellectis’ ongoing collaboration and in-house manufacturing sites, IMPACT and SMART, which are currently under construction.

The manufacturing process of Cellectis’ allogeneic CAR T-cell product line, Universal CARTs or UCARTs, yields frozen, off-the-shelf, non-alloreactive engineered CAR T-cells. UCARTs are intended to be readily available CAR T-cells for a large patient population. Their production is industrialized with defined pharmaceutical release criteria.

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]).

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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.

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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.

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