On June 15, 2018 MorphoSys AG (FSE: MOR; Prime Standard Segment; TecDAX; Nasdaq: MOR) reported the presentation of clinical data from the exploratory phase 2 COSMOS trial (Press release, MorphoSys, JUN 15, 2018, View Source [SID1234527338]). The trial evaluates MorphoSys’s proprietary hemato-oncological drug candidate MOR208 in combination with the cancer drug idelalisib in patients with relapsed or refractory chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL), who progressed on or were intolerant to ibrutinib therapy. Data will be presented in a poster presentation on June 15, 2018, at the 23rd European Hematology Association (EHA) (Free EHA Whitepaper) Annual Meeting in Stockholm/Sweden. MOR208 is an investigational Fc-enhanced humanized monoclonal antibody directed against CD19 in clinical development for the treatment of B cell malignancies.

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"Patients with CLL after failure of ibrutinib therapy are in need of more therapeutic options. We are encouraged by the initial and, for the most part, still ongoing responses observed in this heavily pretreated patient population in our exploratory trial with MOR208 plus idelalisib," commented Dr. Malte Peters, Chief Development Officer of MorphoSys AG. "Overall, this shows the potential medical application of MOR208 in additional B cell malignancies. The data shows that MOR208 may be combined with other cancer drugs used in hematological malignancies, including PI3K inhibitors. We look forward to the upcoming results from the second cohort of MOR208 plus venetoclax of our ongoing COSMOS study which we expect later this year."

COSMOS is a phase 2, two-cohort, open-label, multicenter study evaluating the preliminary safety and efficacy of MOR208 combined with idelalisib (cohort A) or venetoclax (cohort B) in patients with r/r CLL/SLL previously treated with Bruton’s Tyrosine Kinase inhibitor (BTKi) ibrutinib.

Data presented at EHA (Free EHA Whitepaper) 2018 comprise preliminary safety and efficacy data on all 11 patients enrolled into cohort A (cut-off date: January 29, 2018). Patients enrolled had received a median of five prior treatment lines (range: 2-9 prior lines). Nine out of the eleven patients enrolled (82%) had discontinued prior ibrutinib treatment due to progressive disease and two patients (18%) due to toxicity.

The most common treatment-emerging adverse events (TEAEs) of grade 3 or higher were hematologic, with neutropenia observed for four patients (36%) and anemia for three patients (27%) being the most common reported events. Ten treatment-emergent serious adverse events (SAEs) were reported in five patients (45%) none of them being fatal. All except one of the six treatment-related SAEs reported for three patients (27%) were suspected to idelalisib.

According to the preliminary efficacy analysis conducted by the investigators, overall response rate (ORR) was 82%, including one complete response (CR, 9%) confirmed by bone marrow biopsy and eight partial responses (PR, 73%). In addition, two patients (18%) showed stable disease. The median observation time was 4.2 months. At the time of data-cut off, six patients continued treatment. One patient with a very good partial response according to response criteria was taken off the study to receive stem cell transplantation. Two previously responding patients had to discontinue the study due to progressive disease. Two patients (one PR, one stable disease SD) discontinued due to adverse events.

Details about the poster presentation on MOR208 at EHA (Free EHA Whitepaper) 2018:

Abstract Code: PF350

Two-cohort, phase II study in R/R CLL (COSMOS): First preliminary safety and efficacy results of MOR208 treatment in combination with idelalisib in patients who discontinued prior ibrutinib therapy

The poster will be presented during the session "Chronic lymphocytic leukemia and related disorders – Clinical" on Friday, June 15, 2018 5:30-7:00 pm CEST (11:30am-1:00pm EDT), in the poster area at the Stockholmsmässan in Stockholm.

In addition, the corresponding abstract will be on display on the E-poster screens at the conference from Friday, June 15, 2018, 9:30 am CEST (3:30 am EDT) to Sunday, June 17, 2018, 1:00 pm CEST (7:00 am EDT).

Additional information can be found at www.ehaweb.org, including the abstract.

About CD19 and MOR208
CD19 is broadly and homogeneously expressed across different B cell malignancies including DLBCL and CLL. CD19 has been reported to enhance B cell receptor (BCR) signaling, which is assumed important for B cell survival, making CD19 a potential target in B cell malignancies.
MOR208 (previously Xmab(R)5574) is an investigational humanized Fc-engineered monoclonal antibody directed against CD19. Fc-modification of MOR208 is intended to lead to a significant potentiation of antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP), thus aiming to improve a key mechanism of tumor cell killing. MOR208 has been observed in preclinical models to induce direct apoptosis by binding to CD19, which is assumed to be a crucial component for B cell receptor (BCR) signaling.
MorphoSys is clinically investigating MOR208 as a therapeutic option in B cell malignancies in a number of ongoing combination trials. An open-label phase 2 combination trial (L-MIND study) is investigating the safety and efficacy of MOR208 in combination with lenalidomide in patients with relapsed/refractory DLBCL who are not eligible for high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT). Based on interim data from L-MIND, in October 2017 the FDA granted Breakthrough Therapy Designation for MOR208 plus lenalidomide in this patient population. The pivotal phase 2/3 B-MIND study is designed to investigate MOR208 in combination with the chemotherapeutic agent bendamustine in patients with relapsed/refractory DLBCL who are not eligible for high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT) in comparison to the combination of the anti-CD20 antibody rituximab plus bendamustine. In addition, MOR208 is currently being investigated in patients with relapsed/refractory CLL/SLL after discontinuation of a prior Bruton tyrosine kinase (BTK) inhibitor therapy (e.g. ibrutinib) in combination with idelalisib or venetoclax.

On June 14, 2018 Aptose Biosciences Inc. (NASDAQ:APTO) (TSX:APS) reported that they have entered into a license agreement with CrystalGenomics, Inc. (KOSDAQ:083790) for China rights to CG-806 (including People’s Republic of China, Hong Kong and Macau) (Press release, Aptose Biosciences, JUN 14, 2018, View Source;p=RssLanding&cat=news&id=2354544 [SID1234527312]). Aptose will now own worldwide rights (excluding Korea) to develop and commercialize CG-806, a first-in-class, highly potent oral small molecule being developed for acute myeloid leukemia (AML), B-cell malignancies and other hematologic malignancies.

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Under the agreement, CrystalGenomics will receive an upfront payment of US $3 million and is eligible for development, regulatory and commercial-based milestones, as well as single-digit royalties on product sales in China. Total deal value for the China territory, including the upfront payment, is up to US $125 million.

On May 7, 2018, Aptose exercised its option to obtain the exclusive license from CrystalGenomics to develop and commercialize CG-806 worldwide outside of China and Korea. This new agreement extends that license agreement to include China.

"Licensing rights to CG-806 to include the China territory was a strategic decision," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer of Aptose. "Our preclinical work with CG-806 has demonstrated its superior activity to other FLT3 inhibitors on AML patient samples, its superior ability to kill B-cell malignancy patient samples relative to ibrutinib, and a favorable safety profile. We believe that CG-806 has the potential to serve as a transformational agent for multiple hematologic cancers, including AML, CLL and others."

"We are pleased to continue our relationship with the Aptose team, which recognized the exciting potential of CG-806 very early in its development," said Joong Myung Cho, Ph.D., Chairman and Chief Executive Officer of CrystalGenomics. "They have been laser focused on IND-enabling studies of CG-806, and we look forward to seeing CG-806 enter the clinic."

About CG-806

CG-806 is an oral, first-in-class pan-FLT3/pan-BTK multi-kinase inhibitor that represents a potential best-in-class therapeutic for patients with AML.

Aptose has been conducting Investigational New Drug (IND) enabling studies with CG-806, as well as numerous preclinical studies. When tested against fresh bone marrow samples from patients with AML, CG-806 demonstrated superior potency and range of activity relative to all other FLT3 inhibitors evaluated. Likewise, CG-806 demonstrated superiority over ibrutinib when tested against samples from CLL patients. The superior potency and breadth of activity against patient-derived hematologic malignancy cells is due to the ability of CG-806 to target wild type (WT) and all known mutant forms of FLT3 and BTK, and to suppress multiple signaling pathways that can rescue hematologic cancers from other agents. Once-daily oral dosing of CG-806 in murine xenograft models of human hematologic malignancies demonstrated tumor eradication in the absence of observable toxicity, and dose range finding studies have shown CG-806 to have a robust safety profile. Aptose expects to submit an IND in late 2018 and initiate clinical trials immediately thereafter.

On June 14, 2018 CTI BioPharma Corp. (CTI BioPharma) (NASDAQ: CTIC) reported that management will present at the JMP Securities 2018 Life Sciences Conference in New York, NY on Thursday, June 21, 2018 at 1:00 PM ET (Press release, CTI BioPharma, JUN 14, 2018, View Source;p=RssLanding&cat=news&id=2354543 [SID1234527313]).

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The presentation will be webcast live and available for replay from the Investors section of CTI BioPharma’s website at www.ctibiopharma.com.

On June 14, 2018 Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel oncology therapeutics, reported that ELZONRISTM (tagraxofusp; SL-401) will be the subject of three clinical presentations, including an oral presentation on the pivotal BPDCN program (Press release, Stemline Therapeutics, JUN 14, 2018, View Source [SID1234527314]). Updated data from the ongoing Phase 2 trial in chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF) will also be presented. Presentations will be delivered tomorrow, Friday, June 15th at the 23rdCongress of the European Hematology Association (EHA) (Free EHA Whitepaper) in Stockholm, Sweden.

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Details on the presentations are listed below. Presentations will be available on the Stemline website (www.stemline.com), Scientific Presentations tab, after their delivery.

Results of Pivotal Phase 2 Trial of SL-401 in Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm

Abstract: S116
Session: Miscellaneous Treatments in AML
Presenter: Naveen Pemmaraju, MD; MD Anderson Cancer Center
Oral Presentation: Friday, June 15; 11:45 – 12:00 CEST (5:45 AM – 6:00 AM ET)
Location: Room A4
Results from Ongoing Phase 1/2 Trial of SL-401 in Patients with Intermediate or High Risk Relapsed/Refractory Myelofibrosis

Abstract: PF618
Session: Myeloproliferative neoplasms – Clinical
Poster Presentation: Friday, June 15; 17:30 – 19:00 CEST (11:30 AM – 1 PM ET)
Location: Poster Area
Results from Ongoing Phase 1/2 Trial of SL-401 in Patients with Relapsed/Refractory CMML

Abstract: PF626
Session: Myeloproliferative neoplasms – Clinical
Poster Presentation: Friday, June 15; 17:30 – 19:00 CEST (11:30 AM – 1 PM ET)
Location: Poster Area
About ELZONRISTM (tagraxofusp; SL-401)
ELZONRISTM (tagraxofusp; SL-401) is a novel targeted therapy directed to CD123, a cell surface receptor expressed on a range of malignancies. ELZONRIS has successfully completed a pivotal trial in blastic plasmacytoid dendritic cell neoplasm (BPDCN), an indication for which it was granted Breakthrough Therapy Designation (BTD). A rolling Biologics License Application (BLA) submission is underway. ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).

On June 14, 2018 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a clinical stage biotechnology company focused on developing targeted cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported that it has successfully completed the comprehensive characterization of its proprietary cell clone known as 22P1G (Press release, PharmaCyte Biotech, JUN 14, 2018, View Source [SID1234527315]).

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The 22P1G cells constitute the cells in the Master Cell Bank (MCB) that were prepared and tested by PharmaCyte’s contractor, Eurofins Lancaster Laboratories. The cells from the MCB will serve as the active pharmaceutical ingredient (API) in the company’s Cell-in-a Box capsules that will be used (together with low doses of the cancer prodrug ifosfamide) for the treatment of locally advanced, non-metastatic, inoperable pancreatic cancer (LAPC) in its planned clinical trial.

The comprehensive characterization studies include long-term stability of the cells, and stability of the potency of the cells as a therapeutic. All studies performed are required by the U.S. Food and Drug Administration (FDA).

PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, elaborated on the significance of the studies saying, "PharmaCyte is complying with all of the FDA guidelines and recommendations for all cell tests and other recent studies with the 22P1G cells. Successful completion of these studies was a pre-requisite for the approval by the FDA for us to conduct a clinical trial in patients with LAPC. Our treatment is primarily dependent upon genetically engineered live-human cells that produce a particularly potent cytochrome P450 enzyme that can activate the chemotherapy prodrug ifosfamide (clone 22P1G cells).

"With each individual batch, these cells must be stable for the long term, and the properties of the 22P1G cells must remain consistent from batch to batch. The newly completed studies provide evidence that both requirements have been met. Our pancreatic cancer treatment utilizes 22P1G cells that have been encapsulated using the Cell-in-a-Box technology. For treatment of LAPC patients, the capsules containing the cells are implanted near the pancreatic tumor so that a high local concentration of the cancer-killing ifosfamide metabolite is produced near the tumor."