On November 17, 2016 Triphase Accelerator Corporation, a private drug development company dedicated to advancing novel compounds through Phase 2 proof-of-concept, reported that Celgene Corporation, through an affiliate, has acquired the company’s assets related to its proteasome inhibitor, marizomib (MRZ), which is in development for glioblastoma and relapsed and/or refractory multiple myeloma (Press release, Celgene, NOV 17, 2016, View Source [SID1234527214]).

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Under the terms of the agreement, Celgene will make an upfront payment plus additional regulatory, approval and sales milestone payments. Specific financial terms were not disclosed. "This acquisition validates the potential of marizomib based on early clinical results. Our vision is to become a leading early stage oncology drug development company, and this first opt-in by Celgene brings us a step closer to achieving that goal," said Mohit Trikha, Ph.D., chief scientific officer, Triphase Accelerator Corporation. "Just as importantly, this transaction affords us the opportunity to accelerate our efforts on advancing other assets in our pipeline."

"Consistent with our deep commitment and passion for the patients, glioblastoma is an area of significant unmet medical need, and Celgene is committed to helping these patients. We are pleased with Triphase Accelerator’s rapid and high quality work to date, and we value the exceptional collaboration we have with them to advance marizomib," said Celgene’s President of Hematology Oncology, Michael Pehl.

Going forward Celgene has full responsibility for the development of marizomib and will pay Triphase to complete the ongoing clinical studies with marizomib, including a Phase 1 study in relapsed refractory multiple myeloma, a Phase 2 study in recurrent glioma and a Phase 1 study in newly diagnosed glioma.

About Marizomib
Marizomib is a novel, brain-penetrant proteasome inhibitor, which inhibits all three proteasome subunits.

Triphase Accelerator is developing marizomib in both intravenous (IV) and oral formulations as a proteasome inhibitor for hematologic malignancies and solid tumors. The IV formulation has been evaluated in more than 300 patients in multiple clinical studies in patients with solid and hematologic malignancies, either as a single agent or in combination with dexamethasone, a histone deacetylase inhibitor, or an immunomodulatory drug.

The company is currently evaluating marizomib in a proof-of-concept clinical study in combination with bevacizumab (Avastin) in patients with Grade IV malignant glioma (glioblastoma), and has received Orphan Drug designation for marizomib in glioblastoma in the United States from the FDA. In addition, Triphase Accelerator is currently developing marizomib in combination with pomalidomide and dexamethasone in patients with relapsed and refractory multiple myeloma, and has received Orphan Drug designation for marizomib in multiple myeloma in the United States and the European Union. Triphase Accelerator is also evaluating an oral formulation in preclinical studies.

Marizomib has not been approved for any use in any country.

On November 15, 2016 Cellectar Biosciences, Inc. (Nasdaq: CLRB) (the "company"), an oncology-focused, clinical stage biotechnology company, reported it has selected INC Research (Nasdaq: INCR), a leading, global Phase I to IV contract research organization, to oversee its NCI-supported Phase II clinical trial of CLR 131 in patients with multiple myeloma and select hematologic malignancies (Filing, 8-K, Cellectar Biosciences, NOV 17, 2016, View Source [SID1234516654]). The company anticipates that its $2M NCI grant will cover approximately 50 percent of the study’s cost, and the terms of the grant allow Cellectar to pursue an additional $3M for a pivotal Phase III trial of the company’s lead radiotherapeutic compound.

Cellectar plans to leverage the results of its 80-patient, Phase II study to optimally design its pivotal trial of CLR 131 in multiple myeloma and other hematologic malignancies. The multi-armed study will include relapse/refractory patients with multiple myeloma (MM), chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), lymphoplasmacytic lymphoma (LPL), marginal zone lymphoma (MZL), mantle cell lymphoma (MCL), and potentially diffuse large B-cell lymphoma (DLBCL), who have been treated with standard therapy for their underlying malignancies. The company recently accelerated its guidance and announced plans to initiate the trial during the first quarter of 2017.

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"INC Research has outstanding experience in cancer clinical research and a strong reputation within the hematology community. With strong investigator relationships, proven operational expertise and a commitment to high-quality data, they are the ideal partner for this important trial," said Jim Caruso, president and CEO of Cellectar. "Given the accelerated initiation of our Phase II study to the first quarter of 2017 and that we will utilize as many as 15 participating sites, we can confidently plan on providing initial efficacy data in the second half of 2017."

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 preclinical 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.

About Relapsed or Refractory Multiple Myeloma
Multiple myeloma is the second most common blood or hematologic cancer with approximately 30,000 new cases in the United States every year. It affects a specific type of blood cells known as plasma cells. Plasma cells are white blood cells that produce antibodies to help fight infections. While treatable for a time, multiple myeloma is incurable and almost all patients will relapse or the cancer will become resistant/refractory to current therapies.

On November 16, 2016 Therapeutic Proteins International (TPI) reported that it will change its name to Adello Biologics, LLC, effective immediately (Press release, Therapeutic Proteins International, NOV 16, 2016, View Source;s%20Interest-,Therapeutic%20Proteins%20International%20Renames%20Company%20to%20Adello%20Biologics%2C%20LLC%3B%20Relocates,Headquarters%20to%20Piscataway%2C%20New%20Jersey&text=CHICAGO%2C%20Nov.,Biologics%2C%20LLC%2C%20effective%20immediately [SID1234563291]). The name change accompanies the move of its corporate headquarters to Piscataway, New Jersey. Along with all corporate functions, the 50,000 sq. ft. facility will house the company’s new R&D lab. The company plans to complete the move by the end of the year.

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"The new name aligns with our company’s vision; To become the trusted choice of high-quality, affordable biosimilars for patients worldwide," said Dr. Peter Moesta, Chief Executive Officer of Adello. "We are fully focused on developing a robust portfolio of biosimilars, and our move to New Jersey is an effort to further enhance our R&D bench strength, drawing on the scientific talent pool in the area."

The Chicago site will remain the company’s key manufacturing facility in the U.S. with around 100 employees supporting operations. Adello is currently actively recruiting positions for the R&D function in Piscataway. With capacity for 70 scientists, the group will continue to be led by Chief Scientific Officer, Dr. Michael Washabaugh.

On November 16, 2016 PharmaCyte Biotech (OTCQB: PMCB) reported that it has arrived at the door of U.S. FDA and awaits a pre-IND meeting with the agency (Press release, PharmaCyte Biotech, NOV 16, 2016, View Source [SID1234516661]). After years of surrounding its signature live-cell encapsulation technology, Cell-in-a-Box, with some of the brightest minds in pancreatic cancer and fine-tuning its therapy and clinical trial design, the small biotech has reached the final test before it can begin its pivotal clinical trial in advanced pancreatic cancer.

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PharmaCyte made two recent announcements that would be big for any company, but for a small biotech, the announcements are big news for shareholders who have been patiently waiting. First, it announced that oncologist, Dr. Manuel Hidalgo, who is the Chief of the Division of Hematology-Oncology at Harvard Medical School’s Beth Israel Deaconess Medical Center, has accepted the role of Principal Investigator for PharmaCyte’s planned clinical trial. And the company followed up that news with the announcement that it has requested a pre-IND meeting with the FDA for its upcoming pancreatic cancer clinical trial.

In PharmaCyte’s clinical trial, Dr. Hidalgo will once again be teamed up with renowned pancreatic cancer expert Dr. Daniel Von Hoff. Dr. Von Hoff is the Chief Development Officer at Translational Drug Development (TD2), the nation’s premiere oncology CRO and the company responsible for organizing and conducting PharmaCyte’s clinical trial.

Dr. Von Hoff and Dr. Hidalgo worked together on the clinical trials that brought the industry what is now the gold standard and the FDA approved treatment for advanced pancreatic cancer, Abraxane plus gemcitabine.

PharmaCyte has already named a handful of clinical trial sites that are being considered which include the Mayo Clinic in Scottsdale, Arizona, the Beth Israel Deaconess Cancer Center in Boston, the Dana-Farber Cancer Institute in Boston, the Baylor Cancer Center in Dallas, the City of Hope Cancer Center in Los Angeles and sites in Germany and Spain.

Creating an "Artificial Liver" to Target Pancreatic Cancer

PharmaCyte’s pancreatic cancer therapy is made up of pinhead-sized, porous capsules that are filled with thousands of genetically modified cells that act as a type of "artificial liver."

PharmaCyte’s Cell-in-a-Box is not a drug delivery system. There are no drugs encapsulated inside the porous capsules for any of its treatments. Instead, the capsules are filled with about 10,000 live cells that are capable of converting an inactive chemotherapy drug (ifosfamide) into its active cancer-killing form — just as the enzyme system in a patient’s liver would normally do.

Because the chemotherapy drug ifosfamide is a prodrug or an inactive drug, it can travel all over the body and have no effect whatsoever until it is activated in the liver. PharmaCyte’s therapy essentially moves the "normal" conversion site of that inactive drug (the patient’s liver) closer to the cancerous tumor by using Cell-in-a-Box capsules and the live cells inside them to do the job of the patient’s liver or to act as an "artificial liver."

How Does PharmaCyte Biotech Do It

The encapsulated live cells (Cell-in-a-Box capsules) are placed as close to the patient’s cancerous tumor as possible. Once implanted, ifosfamide, the aforementioned chemotherapy drug that needs to be activated in the body, is given to the patient intravenously at one-third the normal dose. The ifosfamide is then carried by the circulatory system to where the encapsulated cells have been placed.

When the ifosfamide, which is normally activated in the liver, comes in contact with the encapsulated live cells in the Cell-in-a-Box capsules, the chemotherapy drug is activated into its cancer-killing form right at the site of the cancer. This is "targeted chemotherapy" in the truest sense, and the company’s therapy has proven effective and safe to use in past clinical trials.

Chemotherapy with No Side Effects

The obvious question is why move the conversion site of the chemotherapy drug at all. Well, there are actually a number of reasons to move the activation site closer to the tumor. We’ll start with the chemotherapy drug itself.

Ifosfamide, when activated, has a very short half-life (time before it decays and no longer offers any effect), so by using the cells inside the Cell-in-a-Box capsules to activate the drug at the site of the tumor, ifosfamide can immediately be the most effective when it’s the most potent before dying off minutes later.

Without a treatment like PharmaCyte’s, ifosfamide would be given to the patient intravenously and then activated "normally" in the liver, the activated drug would then affect tissues and organs other than the pancreas, and by the time it reached the pancreas, it undoubtedly would have lost much of its effectiveness. So, to be effective against a pancreatic tumor when the Cell-in-a-Box capsules are not used, a large dose of the drug has to be administered.

Using ifosfamide in such large doses has proven to be damaging for tissues and organs including the patient’s liver, and because the activated drug would come in contact with such other organs and good cells throughout the body on its way to the pancreas, the side effects would be intolerable; in fact, this is known to be the case.

By moving the conversion site as close to the tumor as possible, PharmaCyte is able to give a much smaller dose of the chemotherapy drug (one-third the normal dose), which patient’s are able to tolerate, and because of the smaller dose, the treatment can be administered without any side effects from the chemotherapy.

Next Stop FDA Clinical Trial

With a list of oncologists and clinicians that reads like a who’s who now in place to lead PharmaCyte’s clinical trial, the company is now awaiting a pre-IND meeting with the FDA. After submitting questions to the FDA as part of a pre-IND meeting request where aspects of the content of the Investigational New Drug (IND) application itself (CMC section, clinical trial description, etc.) will be discussed, PharmaCyte is ready to fully engage with the FDA on its way to receiving the final approval it needs to begin its planned clinical trial in pancreatic cancer.

Once PharmaCyte navigates the pre-IND process and files its IND application, then the FDA will have 30 days to make comments, and if no comments are made, then PharmaCyte is effectively "approved" to begin its pivotal clinical trial.

On November 16, 2016 Heat Biologics, Inc. (Nasdaq:HTBX), a leader in the development of gp96-based immunotherapies that are designed activate a patient’s immune system to fight cancer, reported that it will present a poster reporting topline results, including primary endpoint data, from the Phase II trial evaluating HS-410 (vesigenurtacel-L) in combination with standard of care, Bacillus Calmette-Guérin (BCG), in the treatment of non-muscle invasive bladder cancer (NMIBC) at the Society of Urology Annual Meeting, in San Antonio, TX, on Wednesday, Nov 30th (Press release, Heat Biologics, NOV 16, 2016, View Source [SID1234516626]). The poster will be presented by study principal investigator, Gary Steinberg, MD, The Bruce and Beth White Family Professor of Surgery and Director of Urologic Oncology at The University of Chicago Medical Center.

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Poster Presentation Details:

Title: Top-Line Results from Vesigenurtacel-L (HS-410) in Combination with BCG from a Randomized, Blinded Phase 2 Trial in Patients with Non-Muscle Invasive Bladder Cancer (NMIBC)

Date and Time: November 30, 2016, 5:30-7:15PM EST (4:30-6:15PM CST)

Poster Number: 21

The abstract can be viewed in the searchable online program at: View Source