On August 14, 2018 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium" or "the Company"), reported that it will conduct a conference call on Wednesday, August 15, 2018 at 9:00 AM ET to provide an update on the Pivotal Phase 3 SIERRA Trial (Study of Iomab-B in Elderly Relapsed/Refractory AML) of Iomab-B (Press release, Actinium Pharmaceuticals, AUG 14, 2018, View Source [SID1234528958]). Actinium recently announced that the SIERRA trial had reached twenty-five percent patient enrollment and that the independent Data Monitoring Committee (DMC) would conduct a formal analysis, which has now occurred. Post this event, members of Actinium’s management team are hosting this call to provide an update on the SIERRA trial.

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Conference Call Details

Date: Wednesday, August 15, 2018
Time: 9:00 AM ET
Registration Link: View Source
Toll-Free Dial-in: (855) 427-0225
Dial-in: (718) 865-8336
Conference ID: 4831

"Based on the DMC’s unanimous recommendation, we are pleased that the ongoing SIERRA trial will continue as planned," said Dr. Mark Berger, Chief Medical Officer of Actinium. "This is an important milestone for Iomab-B since it is the first formal safety evaluation of the trial. We note that no Iomab-B safety concerns were raised. With new insights from the data available from the trial thus far as well as feedback from the trial sites, we will make certain protocol revisions to further expand salvage regimens in the control arm. We’ll also be making it easier for patients on the Conventional Care arm who have disease progression to access Iomab-B treatment. In addition, we will be simplifying certain data collection requirements. These improvements coupled with our deeper understanding of referral patterns and other outreach efforts, are anticipated to enable the recently strengthened SIERRA clinical team to complete the trial as quickly as possible with the goal of bringing Iomab-B to a patient population with a significant unmet need."

Sandesh Seth, Actinium’s Chairman and CEO added, "Iomab-B is a very compelling drug candidate that has been studied in over 500 patients in multiple hematologic malignancies including AML, myelodysplastic syndrome, lymphoma and multiple myeloma and is intended to facilitate a potentially curative bone marrow transplant. Iomab-B was developed by the Fred Hutchinson Cancer Research Center in collaboration with the National Cancer Institute and has been studied extensively by leading bone marrow transplant physicians. We are incredibly proud of the pedigree of Iomab-B and motivated by its potential to address unmet medical needs as a targeted conditioning agent in multiple hematologic diseases. We believe that Iomab-B via the SIERRA trial can be the linchpin for developing the leading franchise in targeted conditioning with an emphasis on improving bone marrow transplant access and outcomes."

About Iomab-B

Iomab-B, Actinium’s lead targeted conditioning product candidate, is currently being studied in a 150-patient, multicenter pivotal Phase 3 clinical trial in patients with relapsed or refractory acute myeloid leukemia who are age 55 and above. This pivotal Phase 3 study is called the SIERRA Trial (Study of Iomab-B in Elderly Relapsed/Refractory AML). Upon approval, Iomab-B is intended to prepare and condition patients for a bone marrow transplant which is often considered the only potential cure for patients with certain blood-borne cancers and blood disorders. Iomab-B targets cells that express CD45, an antigen widely expressed in the hematopoietic system on all leukemic and lymphomic (white blood cells), bone marrow cells and cancer stem cells with the monoclonal antibody, BC8 or apamistamab, labeled with the radioisotope, iodine-131. By carrying iodine-131 directly to the bone marrow in a targeted manner, Actinium believes Iomab-B will avoid the side effects that conventional treatments such as chemotherapy and radiation has on most healthy tissues while effectively killing the patient’s cancer and marrow cells potentially enabling more bone marrow transplants with better outcomes through targeted conditioning. In a Phase 2 clinical study in 68 patients with advanced AML or high-risk myelodysplastic syndrome (MDS) age 50 and older, who typically would not be transplant candidates, were able to receive a transplant after being conditioned with Iomab-B and the study resulted in significantly improved transplant success and survival. Iomab-B was developed at the Fred Hutchinson Cancer Research Center where it has been studied in almost 500 patients in a number of Phase 1 and Phase 2 clinical trials across a variety of blood cancer indications with promising results. The studies included patients with acute myeloid leukemia (AML), chronic myeloid leukemia (CML), acute lymphoblastic leukemia (ALL), chronic lymphocytic leukemia (CLL), Hodgkin’s disease (HD), Non-Hodgkin lymphomas (NHL) and multiple myeloma (MM). Iomab-B has been granted Orphan Drug Designation for relapsed or refractory AML in patients 55 and above by the U.S. Food and Drug Administration and the European Medicines Agency.

On August 13, 2018 Cellectar Biosciences (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to CLR 131, the company’s lead Phospholipid Drug Conjugate (PDC) product candidate, for the treatment of Ewing’s sarcoma, a rare pediatric cancer (Press release, Cellectar Biosciences, AUG 13, 2018, View Source [SID1234528817]).

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"We are delighted to announce receipt of our third RPDD from the FDA, which underscores Cellectar’s commitment to rare pediatric cancers. There is a critical need to develop new therapies to fight deadly childhood cancers such as Ewing’s sarcoma, and CLR 131 has shown early promise in this arena," said John Friend, M.D., chief medical officer of Cellectar Biosciences. "This designation, combined with our receipt of FDA Orphan Drug Designation for Ewing’s sarcoma last month, will help support our efforts to optimize the drug development path in this indication and, if successful, enable this new therapeutic candidate is made available to patients as rapidly as possible."

Since March 2018 the FDA has granted RPDDs to CLR 131 for the treatment of three separate rare disease indications including neuroblastoma, rhabdomyosarcoma and now Ewing’s sarcoma. Should CLR 131 be approved by the FDA in any of these indications, the RPDD may enable Cellectar to receive a priority review voucher. Priority review vouchers can be used by the sponsor to receive priority review designation for a future NDA or BLA submission, which could reduce the FDA review time from twelve months to eight months. Currently, these vouchers can also be transferred or sold to another entity. Since the beginning of 2017, six priority review vouchers were sold for between $80 million and $150 million each.

The FDA grants RPDD for diseases that primarily affect children from birth to age 18, and affect fewer than 200,000 persons in the U.S. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

Cellectar plans to evaluate CLR 131 in a Phase 1 clinical study for the treatment of pediatric patients with Ewing’s sarcoma, rhabdomyosarcoma, osteosarcoma, neuroblastoma, high-grade glioma and lymphomas. Cellectar has received clearance from the FDA for an accelerated Phase 1 trial designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of CLR 131 in pediatric patients with these cancer types. Further details about the trial can be found at clinicaltrials.gov using the identifier number NCT03478462.

About Ewing’s Sarcoma

Ewing’s sarcoma is the second most common bone malignancy among children and adolescents. According to a study published in the Journal of Hematology/Oncology, the incidence is about 3 cases per 1 million per year in children younger than age 20. Despite the favorable prognosis, an American Cancer Society study showed that approximately 30-40% of patients develop metastases or local recurrence, and the long-term survival rate for refractory or recurrent disease is only 22-24%. The relapsed and refractory statistics underscore the need for new treatment options.

About CLR 131

CLR 131 is Cellectar’s investigational radioiodinated PDC therapy that exploits the tumor-targeting properties of the company’s proprietary phospholipid ether (PLE) and PLE analogs to selectively deliver radiation to malignant tumor cells, thus minimizing radiation exposure to normal tissues. CLR 131, is in a Phase 2 clinical study in relapsed or refractory (R/R) MM and a range of B-cell malignancies and a Phase 1 clinical study in patients with (R/R) MM exploring fractionated dosing. The company is currently initiating a Phase 1 study with CLR 131 in pediatric solid tumors and lymphoma and is planning a second Phase 1 study in combination with external beam radiation for head and neck cancer.

On August 13, 2018 ArQule, Inc. (Nasdaq:ARQL), reported the publication of preclinical study data for ARQ 531, the Company’s rationally-designed, reversible inhibitor of both wild type and C481S-mutant Bruton’s tyrosine kinase (BTK) (Press release, ArQule, AUG 13, 2018, View Source [SID1234532694]). The studies, published in Cancer Discovery, were conducted in collaboration with researchers at The Ohio State University. Data from these studies demonstrated efficacy in in vitro and in vivo hematologic malignancy models that recapitulate the most common mechanisms of resistance to irreversible BTK inhibitors, including ibrutinib.

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Highlights from the manuscript (link here) include:

Differentiated Crystal Structure and Biochemical Profile

The crystal structure of ARQ 531 bound to BTK elucidates the mechanism of BTK inhibition that is not dependent on the specific amino acid residue at position 481 (eg. C or S)
Recombinant BTK biochemical assays of ARQ 531 and ibrutinib show similar inhibition for wild type BTK, however ibrutinib has dramatically lower inhibition, binding affinity and residence time for mutant BTK
"Relapsed and refractory patients that develop resistance to ibrutinib have poor outcomes and limited treatment options," said Brian Schwartz, M.D., Chief Medical Officer and Head of R&D at ArQule. "ARQ 531 was rationally-designed and selected to address this unmet need by inhibiting both wild type and mutant BTK. The published crystal structure and biochemistry clearly demonstrate the mechanism by which ARQ 531 maintains binding and inhibition of mutant BTK in conditions where ibrutinib cannot."

Established Activity in Multiple Cellular and Murine Models of Hematological Malignancies

Exhibited dose dependent toxicity in human primary CLL cells (mutant and wild type)
Inhibited CLL cell migration in vitro
Established superiority to ibrutinib in an engraftment murine model of CLL
Showed activity against other B-cell signaling pathways
Demonstrated efficacy in a murine model of Richter’s transformation
John Byrd, M.D., the Warren Brown Chair of Leukemia Research at The Ohio State University stated, "The inhibition profile of ARQ 531 may confer distinct advantages over ibrutinib, potentially expanding the patient population beyond those with a C481S mutation who may benefit from treatment. Targeting multiple kinases in the B cell activation pathway may provide more durable responses to treatment while also delaying the emergence of treatment resistance. Jennifer Woyach, M.D., Associate Professor of Medicine at The Ohio State University, added, "I am particularly encouraged by the CLL mouse model data which established the superior efficacy of ARQ 531 compared to ibrutinib and the efficacy of ARQ 531 in the model of Richter’s transformation as this is an extremely aggressive disease with very few treatment options."

On August 13, 2018 Unum Therapeutics Inc. (NASDAQ: UMRX), a clinical-stage biopharmaceutical company focused on the development of cellular immunotherapies based on its novel, universal Antibody-Coupled T Cell Receptor (ACTR) technology platform, reported that an investigational new drug (IND) application is now active for ACTR T cells in combination with trastuzumab for the treatment of patients with HER2+ advanced cancers (Press release, Unum Therapeutics, AUG 13, 2018, View Source [SID1234528936]). This represents the first solid tumor product candidate based on Unum’s novel, universal ACTR technology, and the fourth clinical trial program for the Company.

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"We are very happy to reach this important milestone for patients and for Unum," said Chuck Wilson, Chief Executive Officer of Unum. "ACTR represents a promising novel technology that can be used to target different tumor types and it’s exciting to expand its application to target solid tumors. We are committed to developing ACTR for patients with HER2+ advanced cancers who need better treatment options."

Under this IND, Unum is preparing to initiate a multi-center Phase I trial, called ATTCK-34-01, by the end of 2018 in patients with HER2+ advanced cancers. ATTCK-34-01 is designed as a dose escalation study where both the ACTR T cell drug product and trastuzumab doses are escalated in order to define the safety, tolerability, and anti-tumor activity of the combination. Expansion at the recommended Phase 2 dose is planned.

On August 13, 2018 Checkpoint Therapeutics, Inc. ("Checkpoint") (NASDAQ: CKPT), a clinical-stage immuno-oncology biopharmaceutical company focused on the acquisition, development and commercialization of novel treatments for patients with solid tumor cancers, reported that preliminary safety and efficacy data from a Phase 1/2 clinical trial of CK101 (also known as RX518), a third-generation epidermal growth factor receptor ("EGFR")
tyrosine kinase inhibitor ("TKI") being evaluated in advanced non-small cell lung cancer, has been selected for a late-breaking oral presentation at the International Association for the Study of Lung Cancer (IASLC) 19th World Conference on Lung Cancer, to be held September 23-26, 2018, in Toronto (Press release, Checkpoint Therapeutics, AUG 13, 2018, View Source [SID1234528977]).

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"We are thrilled to announce that preliminary data from the Phase 1/2 trial of our novel thirdgeneration EGFR inhibitor has been selected for a late-breaking oral presentation at the World Conference on Lung Cancer. This marks the first clinical data to be reported by Checkpoint, an important clinical and corporate milestone," said James F. Oliviero, President and Chief Executive Officer of Checkpoint Therapeutics. "Third-generation EGFR inhibitors are highly selective and have the potential to demonstrate improved safety and tolerability versus earlier-generation
therapies. There is currently only one third-generation EGFR inhibitor approved and we believe CK-101 could be second to market potentially with a differentiated safety profile."
Details of the presentation are as follows:

Title: CK-101 (RX518), a Third Generation Mutant-Selective Inhibitor of EGFR in NSCLC: Results of an Ongoing Phase I/II Trial
Date: Monday, Sept. 24, 2018
Session: Novel Therapies in ROS1, HER2 and EGFR
Presenter: Melissa L. Johnson, M.D., Associate Director, Lung Cancer Research, Sarah Cannon Research Institute at Tennessee Oncology, PLLC, Nashville, Tenn.
The full abstract will be posted on the conference website on Wednesday, Sept. 5, 2018, at 5 p.m. ET. For additional information, please visit: View Source Checkpoint holds an exclusive worldwide license (except with respect to certain Asian countries) to CK‐101, which it acquired from NeuPharma, Inc. in 2015.

About CK-101
CK-101 (also known as RX518) is an oral, third-generation, irreversible kinase inhibitor against selective mutations in the EGFR gene. Activating mutations in the tyrosine kinase domain of EGFR, such as L858R and exon 19 deletion, are found in approximately 20 percent of patients with advanced non-small cell lung cancer ("NSCLC").
Compared to chemotherapy, first-generation EGFR inhibitors significantly improved objective response rate and progression-free survival in previously untreated NSCLC patients carrying EGFR mutations. However, tumor progression could develop due to resistance mutations, often within months of treatment with first-generation EGFR inhibitors. The EGFR T790M "gatekeeper" mutation is the most common resistance mutation found in patients treated with first-generation EGFR inhibitors. The mutation decreases the affinity of first-generation inhibitors to EGFR kinase domain, rendering the drugs ineffective. Second-generation EGFR inhibitors have improved
potency against the T790M mutation, but have not provided meaningful benefits in NSCLC patients due to toxicity from also inhibiting wild-type EGFR. Third-generation EGFR inhibitors are designed to be highly selective against both EGFR-TKI-sensitizing and resistance mutations, with minimal activity on wild-type EGFR, thereby improving tolerability and safety profiles. Checkpoint Therapeutics is developing CK-101 for the treatment of NSCLC patients carrying the
susceptible EGFR mutations. These include the EGFR T790M mutation in second-line NSCLC patients, as well as the EGFR L858R and exon 19 deletion mutations in first-line NSCLC patients.