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Cellectar Biosciences Announces USPTO Grants Patent Allowances for
Radiotherapeutic PDCs for A Variety of Solid Tumor Types

On December 14, 2016 Cellectar Biosciences, Inc. (Nasdaq: CLRB) (the "company"), an oncology-focused clinical stage biotechnology company, reported that the United States Patent and Trademark Office ("USPTO") has issued patent allowances covering method of use for radiotherapy with the company’s lead compound, CLR 131, as well as CLR 125, another of the company’s pipeline products using its proprietary phospholipid drug conjugate ("PDC") to deliver a radio therapeutic (Filing, 8-K, Cellectar Biosciences, DEC 14, 2016, View Source [SID1234517072]).

The allowance covers radiotherapy applications for a broad range of solid tumors, specifically: lung, adrenal, intestinal, colon, colorectal, ovarian, cervical, prostate, liver, breast, subcutaneous and pancreatic cancers, as well as melanoma, retinoblastoma, glioma, carcinosarcoma, squamous cell carcinoma and hepatocellular carcinoma. These claims are a continuation of US Patent No. 8,877,159, which provides patent protection into 2025.

"This patent allowance further expands the intellectual property protection of our lead radiotherapeutic candidate beyond the hematologic cancers we are currently researching," said Jim Caruso, president and CEO of Cellectar. "The expanded patent estate provides us and any potential partners with the option to advance CLR 131 clinical development into a wide variety of solid tumor applications."

About CLR 131
CLR 131 is an investigational compound under development for a range of hematologic malignancies. It is currently being evaluated in a Phase I clinical trial in patients with relapsed or refractory multiple myeloma. The company plans to initiate a Phase II clinical study to assess efficacy in a range of B-cell malignancies in the first quarter of 2017. Based upon pre-clinical and interim Phase I study data, treatment with CLR 131 provides a novel approach to treating hematological diseases and may provide patients with therapeutic benefits, including overall response rate (ORR), an improvement in progression-free survival (PFS) and overall quality of life. CLR 131 utilizes the company’s patented PDC tumor targeting delivery platform to deliver a cytotoxic radioisotope, iodine-131 directly to tumor cells. The FDA has granted Cellectar an orphan drug designation for CLR 131 in the treatment of multiple myeloma.

About Phospholipid Drug Conjugates (PDCs)
Cellectar’s product candidates are built upon its patented cancer cell-targeting delivery and retention platform of optimized phospholipid ether-drug conjugates (PDCs). The company deliberately designed its phospholipid ether (PLE) carrier platform to be coupled with a variety of payloads to facilitate both therapeutic and diagnostic applications. The basis for selective tumor targeting of our PDC compounds lies in the differences between the plasma membranes of cancer cells compared to those of normal cells. Cancer cell membranes are highly enriched in lipid rafts, which are glycolipoprotein microdomains of the plasma membrane of cells that contain high concentrations of cholesterol and sphingolipids, and serve to organize cell surface and intracellular signaling molecules. PDCs have been tested in over 70 different xenograft models of cancer.

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Arrowhead Reports Fiscal 2016 Year End Results

On December 14, 2016 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported financial results for its fiscal 2016 fourth quarter and year ended September 30, 2016 (Press release, Arrowhead Research Corporation, DEC 14, 2016, View Source [SID1234517070]). The company is hosting a conference call at 4:30 p.m. EST to discuss results.

Conference Call and Webcast Details

Investors may access a live audio webcast on the Company’s website at View Source For analysts that wish to participate in the conference call, please dial 855-215-6159 or 315-625-6887 and enter Conference ID 33791749.

A replay of the webcast will be available on the company’s website approximately two hours after the conclusion of the call and will remain available for 90 days. An audio replay will also be available approximately two hours after the conclusion of the call and will be available for 3 days. To access the audio replay, dial 404-537-3406 and enter Conference ID 33791749.

Selected Fiscal 2016 and Recent Events

Discontinued development of ARC-520, ARC-521, and ARC-AAT in November 2016
The Company announced that it would be discontinuing these clinical programs, which utilized the intravenously administered DPCivTM, or EX1, delivery vehicle, and redeploying its resources and focus toward utilizing the Company’s new proprietary subcutaneous and extra-hepatic delivery systems
The decision to discontinue development of EX1-containing programs was based primarily on two factors:
During ongoing discussions with regulatory agencies and outside experts, it became apparent that there would be substantial delays in all clinical programs that utilize EX1, while the Company further explored the cause of deaths in a non-clinical toxicology study in non-human primates exploring doses of EX1 higher than those planned to be used in humans
The Company has made substantial advances in RNA chemistry and targeting resulting in large potency gains for subcutaneous administered and extra-hepatic RNAi-based development programs
Because of the discontinuation of its existing clinical programs, the Company also reduced its workforce by approximately 30%, while maintaining resources necessary to support current and potential partner-based programs and the Company’s pipeline
Entered into two collaboration and license agreements with Amgen
Total deal value of up to $673.5 million
Arrowhead received $56.5 million upfront:
$35 million in upfront cash payments, $21.5 million equity investment
Up to low double-digit royalties for ARO-LPA and single-digit royalties for the undisclosed target, ARO- AMG1
Amgen receives:
Exclusive license to ARO-LPA program
Option for an additional candidate against an undisclosed target, ARO- AMG1
Amgen will be wholly responsible for funding and conducting all clinical development and commercialization
Continued progress on preclinical candidates including ARO-HBV, ARO-AAT, ARO-F12, ARO-LPA, and ARO-HIF2
Regarding ARO-F12 and ARO-LPA:
Presented preclinical data at the American Heart Association’s Scientific Sessions 2016 for two development programs using Arrowhead’s proprietary subcutaneous delivery platform:
RNAi triggers against Factor 12 (F12) showed dose dependent reductions in serum F12
A statistically significant reduction (p=0.002) in thrombus weight was observed at greater than 95% F12 knockdown in a rat arterio-venous shunt model
There was no increased bleeding risk in ARO- F12-treated mice, even with greater than 99% knockdown of F12 levels
RNAi triggers against Lipoprotein (a) [Lp(a)] led to greater than 98% maximum knockdown after a single 3 mg/kg SQ dose in Transgenic mice
In an atherosclerosis model, data suggest that RNAi triggers can be effectively delivered to a fatty liver using the subcutaneous delivery platform
Regarding ARO-HIF2
Presented preclinical data showing that ARO-HIF2 inhibited renal cell carcinoma growth and promoted tumor cell death in its preclinical studies
Strengthened the Company’s balance sheet with August 2016 private offering and Amgen agreement upfront payments
In August 2016, the Company sold 7.6 million shares of Common Stock to certain institutional investors and received net proceeds of approximately $43.2 million
As part of the collaboration and license agreements as well as a Common Stock Purchase Agreement with Amgen, $14 million of the total $56.5 million upfront cash payments and equity investments were received in September 2016, and the remaining $42.5 million was received in November 2016
Continued progress on former drug candidates prior to the discontinuations
Presented preclinical and clinical data on former drug candidate ARC-AAT at the Liver Meeting
In a first-in-human clinical study, ARC-AAT was well tolerated and induced deep and durable reduction of the target AAT protein
The preclinical data suggest a possible improvement of liver health and arrest of further damage from treatment with ARC-AAT
Advanced former drug candidate ARC-521 into a Phase 1/2 study
Conducted multiple dose and combination studies of former drug candidate ARC-520
Selected Fiscal 2016 Year End Financial Results

ARROWHEAD PHARMACEUTICALS, INC.
CONSOLIDATED FINANCIAL INFORMATION

Year Ended September 30
OPERATING SUMMARY
2016 2015

REVENUE $ 158,333 $ 382,000
OPERATING EXPENSES
Research and development 41,454,452 47,267,361
Acquired in-process research and development - 10,142,786
Salaries and payroll-related costs 19,461,656 16,554,008
General and administrative expenses 9,940,737 7,931,184
Stock-based compensation 11,595,816 10,232,897
Depreciation and amortization 3,260,045 2,336,207
Impairment expense 2,050,817 -
Contingent consideration – fair value adjustments (5,862,464 ) 1,891,533
TOTAL OPERATING EXPENSES 81,901,059 96,355,976
OPERATING LOSS (81,742,726 ) (95,973,976 )
OTHER INCOME/(EXPENSE), PROVISION FOR INCOME TAXES 19,724 4,033,094
NET LOSS $ (81,723,002 ) $ (91,940,882 )

EARNINGS PER SHARE (BASIC AND DILUTED): $ (1.34 ) $ (1.60 )
WEIGHTED AVERAGE SHARES OUTSTANDING 61,050,880 57,358,442

FINANCIAL POSITION SUMMARY
September 30,
2016 2015
CASH AND CASH EQUIVALENTS 85,366,448 81,214,354
SHORT-TERM INVESTMENTS - 17,539,902
TOTAL CASH RESOURCES (CASH, CASH EQUIVALENTS AND INVESTMENTS) 85,366,448 98,754,256
OTHER ASSETS 42,810,057 33,513,658
TOTAL ASSETS 128,176,505 132,267,914
TOTAL LIABILITIES 33,152,246 22,646,280
TOTAL STOCKHOLDERS’ EQUITY 95,024,259 109,621,634
TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY 128,176,505 132,267,914

SHARES OUTSTANDING 69,746,685 59,544,677
PROFORMA SHARES OUTSTANDING (INCLUDING CONVERSION OF PREFERRED SHARES) 72,417,675 62,215,667

Genocea Announces Research Collaboration with Checkmate Pharmaceuticals in Advanced Melanoma

On December 14, 2016 Genocea Biosciences, Inc. (NASDAQ:GNCA), a company developing T cell-directed vaccines and immunotherapies, reported a research collaboration with Checkmate Pharmaceuticals, Inc. (Checkmate). The goal of the collaboration is to characterize the T cell responses of patients in the ongoing Checkmate Phase 1b clinical trial and to identify antigens associated with protective T cell responses.

ATLAS, Genocea’s proprietary rapid antigen identification screening system, will be used to profile the T cell responses of approximately 20 patients enrolled in Checkmate’s ongoing Phase 1b clinical trial of CMP-001 in combination with the checkpoint inhibitor pembrolizumab to a library of tumor-associated antigens common to patients with advanced melanoma. The T cell response signatures of those patients who respond to CMP-001 / pembrolizumab combination therapy will be compared to the signatures of those who do not respond, thereby potentially identifying antigen-based signatures associated with positive or negative patient outcomes.

"This collaboration with Checkmate is an exciting opportunity to apply ATLAS’s unique ability to comprehensively profile patients’ T cell responses to a cancer," said Jessica Baker Flechtner, Ph.D., chief scientific officer at Genocea. "Understanding how T cell responses are associated with different clinical outcomes together with the dynamics of epitope spread, where effective tumor killing leads to broader immune responses against new antigens in the tumor, is fundamental to the design of new cancer vaccines and the selection of patients most likely to benefit from different cancer treatments."

Genocea’s proprietary ATLAS technology re-creates a patient’s individual T cell immune responses to cancer ex vivo. This means ATLAS can potentially identify – not just guess or predict – targets to which patient T cells are responding to kill a tumor. It may also allow ATLAS to distinguish between antigen candidates that stimulate productive T cell responses and those that are irrelevant or are associated with inhibitory responses. It may also enable the future development of a non-invasive assay to determine patients suited for therapy.

"We look forward to collaborating with Genocea to profile the T cell responses of patients enrolled in our current Phase 1b CMP-001 / pembrolizumab clinical study," said Art Krieg, CEO of Checkmate. "We believe the ability of ATLAS to differentiate between responsive and refractory patients in this trial could inform both patient selection and clinical trial design going forward."

About ATLAS
ATLAS is a first of its kind proprietary rapid antigen identification screening system designed to find targets of protective T cell responses. The technology solves challenges to date associated with finding targets of T cell responses. ATLAS can examine T cell responses from large, diverse human populations, and comprehensively screen every potential antigen from a pathogen or target indication in a rapid, high-throughput manner, taking weeks versus years to find relevant antigens. Because targets identified by ATLAS are based on actual human immune responses to all potential antigens, with no guesswork or predictions, by the time these candidates reach clinical trials there may be a greater likelihood of success in clinical development. This approach provides the ability to identify smarter targets for use in developing vaccines and immunotherapies to treat infectious disease, cancer and autoimmunity.

Galena Biopharma Announces the Phase 2 Clinical Trial of NeuVax™ (nelipepimut-S) in Ductal Carcinoma in Situ (DCIS) is Open for Enrollment and Screening Patients

On December 14, 2016 Galena Biopharma, Inc. (NASDAQ:GALE), a biopharmaceutical company committed to the development and commercialization of hematology and oncology therapeutics that address unmet medical needs, reported that the NeuVax (nelipepimut-S) Phase 2 clinical trial entitled VADIS: Phase 2 trial of the Nelipepimut-S Peptide VAccine in Women with DCIS of the Breast is now open for enrollment and screening patients (Press release, Galena Biopharma, DEC 14, 2016, View Source [SID1234517077]). The trial is being run in collaboration with the National Cancer Institute (NCI) and The University of Texas MD Anderson Cancer Center Phase I and II Chemoprevention Consortium.

Ductal Carcinoma in Situ (DCIS) is a noninvasive cancer in which abnormal cells are found in the lining of the breast duct and have not spread outside the milk duct to invade other parts of the breast. In some cases, DCIS may become invasive cancer and spread to other tissues; currently it is extremely challenging to identify which lesions may convert to invasive cancer.1 DCIS is the most common type of breast cancer and the rate at which DCIS is diagnosed has increased dramatically in recent years as a result of increased cancer screening.1

"The Phase 2 VADIS trial will broaden the development footprint for NeuVax into this new indication as we assess its safety and immunologic efficacy in patients with DCIS," said Mark W. Schwartz, Ph.D., President and Chief Executive Officer. "The challenge with DCIS is that the exact course of the disease cannot be determined and consequently the outcomes for patients may be undefined. This study will evaluate NeuVax in its capacity to generate the immune response, including tumor-infiltrating lymphocytes (TILs), that could potentially prevent the progression of the disease. Positive study results from the VADIS trial could provide potential evidence for NeuVax as a first of its kind treatment for the primary prevention of invasive breast cancer, thereby sparing significant numbers of women from disfiguring surgery and radiation treatments. We are grateful to be working on this innovative initiative with the NCI and the MD Anderson Cancer Center, two of the finest oncology institutions in the country."

Elizabeth A. Mittendorf, M.D., Ph.D., Associate Professor, Department of Breast Surgical Oncology, The University of Texas MD Anderson Cancer Center and the Principal Investigator of the VADIS trial, added, "We are pleased to collaborate with the NCI and Galena on the development of NeuVax in DCIS which affects more than 60,000 women a year. In breast cancer, DCIS may be a precursor to invasive disease, and it is possible that vaccination before tumor cells become genetically unstable and begin dividing rapidly could prevent evolution to malignancy. NeuVax is currently the most advanced breast cancer vaccine in the clinic and we think its broad immune response not only to the specific antigen targeted by the drug, but also to other antigens, speaks to why NeuVax could be an ideal therapeutic candidate in this indication. By vaccinating patients in early-phase disease, we may be giving them long-lasting immunity to protect them down the line."

The MD Anderson Consortium is funded through the Division of Cancer Prevention at the NCI, which will provide financial and administrative support for the trial. Galena will provide NeuVax, as well as some financial and administrative support.

Source: 1Pang, J-MB, et al, Histopathology 2016.

About VADIS

VADIS (Phase 2 trial of the Nelipepimut-S Peptide VAccine in Women with DCIS of the Breast) is a Phase 2 trial to evaluate women diagnosed with DCIS who are HLA-A2 positive, who express HER2 at IHC 1+, 2+, or 3+ levels, and who are pre or post menopausal. Patients will be randomized to one of two arms (n=48): NeuVax plus GM-CSF (n=32 randomized / 27 evaluable) or GM-CSF alone (n=16 randomized / 13 evaluable). The primary endpoint is immunologic and will evaluate the effect of the NeuVax vaccine on NeuVax -specific cytotoxic T lymphocytes to determine whether long-lasting immunity is induced. Secondary endpoints include evaluation of toxicity, In vivo immune response (delayed type hypersensitivity reaction (DTH)), epitope spreading, T-cell functional capacity, and histologic response measured by degree of lymphocyte infiltration, proliferation, and apoptosis.

The trial design via poster presentation is available on the Company’s website here. Additional information on the trial can be found on the ClinicalTrials.gov site here (identifier: NCT02636582).

About Ductal Carcinoma in Situ

Ductal Carcinoma in Situ (DUK-tul KAR-sih-NOH-muh in SY-too), or DCIS, is defined by the NCI as a noninvasive condition in which abnormal cells are found in the lining of a breast duct, and is the most common type of breast cancer. The abnormal cells have not spread outside the duct to other tissues in the breast. In some cases, DCIS may become invasive cancer and spread to other tissues; currently there is no way to know which lesions could become invasive. Current treatment options for DCIS include breast-conserving surgery and radiation therapy with or without tamoxifen, breast-conserving surgery without radiation therapy, or total mastectomy with or without tamoxifen. According to the American Cancer Society, in 2015 there were over 60,000 diagnoses of ductal carcinoma in situ.

About NeuVax (nelipepimut-S)

NeuVax (nelipepimut-S) is a first-in-class, HER2-directed cancer immunotherapy under evaluation to prevent breast cancer recurrence after standard of care treatment in the adjuvant setting. It is the immunodominant peptide derived from the extracellular domain of the HER2 protein, a well-established target for therapeutic intervention in breast carcinoma. The nelipepimut-S sequence stimulates specific CD8+ cytotoxic T lymphocytes (CTLs) following binding to specific HLA molecules on antigen presenting cells (APCs). These activated specific CTLs recognize, neutralize and destroy, through cell lysis, HER2 expressing cancer cells, including occult cancer cells and micrometastatic foci. The nelipepimut-S immune response can also generate CTLs to other immunogenic peptides through inter- and intra-antigenic epitope spreading. In clinical studies, NeuVax is combined with recombinant granulocyte macrophage-colony stimulating factor (GM-CSF).

NeuVax is also currently in two breast cancer studies in combination with trastuzumab (Herceptin; Genentech/Roche): a Phase 2b trial in node positive and triple negative HER2 IHC 1+/2+ (clinicaltrials.gov identifier: NCT01570036); and, a Phase 2 trial in high risk, node positive or negative HER2 IHC 3+ patients (clinicaltrials.gov identifier: NCT02297698). A Phase 2 clinical trial is planned in patients with gastric cancer.

DelMar Pharmaceuticals Receives Notice of Allowance from the US Patent and Trademark Office for Key Patent for VAL-083

On December 14, 2016 DelMar Pharmaceuticals, Inc. (NASDAQ: DMPI) ("DelMar" and the "Company"), a biopharmaceutical company focused on the development and commercialization of new cancer therapies, reported that it received a Notice of Allowance from the U.S. Patent and Trademark Office (USPTO) for United States Patent Application Serial No. 13/933,844 entitled, "Improved Analytical Methods for Analyzing and Determining Impurities in Dianhydrogalactitol (Press release, DelMar Pharmaceuticals, DEC 14, 2016, View Source [SID1234517069])."

The patent has also been granted in Australia and is being prosecuted in other international jurisdictions as part of more than 100 patent filings from thirteen patent families representing claims covering VAL-083 until 2036.

"This patent provides DelMar with ownership of essential manufacturing controls required under good manufacturing practice (GMP) guidelines," said Jeffrey Bacha, chairman and CEO of DelMar Pharmaceuticals. "This is an important component of our strategy to establish a strong international patent portfolio as the foundation to support the global development of VAL-083 in multiple cancer indications."

DelMar is preparing to advance VAL-083 into a pivotal Phase III clinical trial in the United States as a potential treatment for refractory glioblastoma multiforme (GBM), the most common and aggressive form of brain cancer.

The Company has also recently presented data and outlined plans for expansion of VAL-083 into new indications, including newly diagnosed GBM, ovarian and lung cancer. DelMar’s most recent abstract entitled "Assessment of Dianhydrogalactitol (VAL-083) in the Treatment of Relapsed or Refractory Non-Small Cell Lung Cancer" was presented at the 17th World Congress on Lung Cancer in Vienna, Austria on December 7, 2016.

Further details regarding DelMar’s scientific presentations can be found at View Source

The USPTO issues a Notice of Allowance after it makes a determination that a claimed invention is novel and nonobvious in light of all known technology in existence, and that a patent should be granted from such an application. Based on the timing of this Notice of Allowance, DelMar Pharmaceuticals expects the forthcoming US patent to be issued in early 2017.