BioInvent is granted a patent in Europe for the immune-oncology antibody BI-505

On March 2016 BioInvent International (BINV) reported that the European Patent Office, EPO, has decided to grant the company’s patent EP 1 960 432, relating to the immune-oncology antibody BI-505 (Press release, BioInvent, MAR 16, 2016, View Source [SID:1234509596]). BioInvent plans to shortly initiate a Phase II trial with BI-505 in patients with multiple myeloma, with the aim to prevent or significantly delay relapse of this serious form of hematologic cancer.

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The European patent for BI-505 will enter into force on 6 April 2016 and will expire in 2026. Corresponding patents have already been granted in the US, Japan, Australia, China, South Korea and Russia, among other countries.

"The extended geographical patent protection for BI-505 increases the value of our project and underlines BioInvent’s ability to develop innovative and unique antibodies against severe cancer diseases", says Michael Oredsson, CEO of BioInvent.
During 2016, the start of clinical studies for three of BioInvent’s clinical phase drug projects are planned. In addition to the study mentioned above with BI-505, clinical studies are also expected to be initiated with BI-1206 in patients with non-Hodgkin lymphoma and chronic lymphatic leukaemia, and with TB-403 for treatment of rare but serious types of cancer in children.

About BI-505
BI-505 is a human antibody against ICAM-1 developed by BioInvent, which will be clinically tested in cooperation with researchers at Penn Medicine as an immuno-oncological therapy to prevent or delay relapse in patients with multiple myeloma (a form of bone marrow cancer) undergoing stem-cell transplantation. Preclinical data indicates improved activity against myeloma when BI-505 is administered in combination with Velcade or Revlimid. BI-505’s favourable safety profile has been demonstrated in a previous phase I trial. This and the unique mechanism of action, "flagging" remaining myeloma cells for elimination by actively recruited macrophages, as well as the potential to inhibit ICAM-1 dependent survival signals between myeloma cells and tumour stroma, indicate a unique possibility of improving the therapeutic effect of stem-cell transplantation.

Celsion Announces Presentation of Preclinical Data for GEN-1 IL-12 Immunotherapy in Combination with Avastin® and Doxil® at the AACR Annual Meeting 2016

On March 16, 2016 Celsion Corporation (NASDAQ: CLSN), an oncology drug development company, reported that its preclinical data for GEN-1 in combination with Avastin and Doxil for the treatment of ovarian cancer will be presented at the upcoming AACR (Free AACR Whitepaper) Annual Meeting 2016 in New Orleans (Press release, Celsion, MAR 16, 2016, View Source [SID:1234509587]).

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The presentation will be held on Monday, April 18, 2016 (1:00 pm to 5:00 pm) and will summarize results from preclinical studies demonstrating significant synergistic anti-cancer effects when GEN-1 is combined with Avastin and Doxil, a current Standard of Care (SoC) for platinum resistant ovarian cancer patients. GEN-1 is an IL-12 DNA plasmid vector formulated into a nanoparticle with a non-viral delivery system to cause the sustained local production and secretion of the Interleukin-12 (IL-12) protein loco-regionally to the tumor site. These data will be used by the Company to support a comprehensive IND protocol filing for a Phase I/II clinical trial evaluating the combination in recurrent ovarian cancer later this year.

"The immune stimulating nature of GEN-1 in combination with Avastin and Doxil, two of the most widely used cancer therapies, makes for an ideal therapy, bolstering the anti-cancer effect beyond what has been observed when used alone," said Nicholas Borys, M.D., senior vice president and chief medical officer of Celsion. "Results from this important study are highly encouraging and suggest that when these therapies are combined, they offer the potential to significantly reduce tumor burden and disease progression in this highly aggressive cancer in patients who have failed first line platinum-based therapies."

Data is emerging on how SoC chemotherapy treatments influence the immune response in cancers that they are targeting. Chemotherapy stimulates the immune system by (i) making the dying tumor cells more visible to the body’s immune system through the release of tumor antigens; (ii) destroying the immune suppression caused by the tumor; and (iii) directly or indirectly affecting immune cells and immune modulators.

Chemotherapy treatments may be a good candidate for combination with immune-mediating therapies like GEN-1. Doxorubicin increases not only T-cell and natural killer (NK) cell production, but also B cells (anti-tumor antibodies) in ovarian cancer. IL-12 is a highly active cytokine that can induce a potent anti-cancer immunity mediated through the activation of cytotoxic T-lymphocytes and NK cells and the inhibition of immune-suppressing regulatory T-cells.

Clinical data supporting the combination of GEN-1 with SoC chemotherapies

GEN-1 has already demonstrated encouraging safety and efficacy clinical data in combination with PEGylated liposomal doxorubicin (Doxil) in patients with platinum-resistant ovarian cancer. Results from a Phase Ib clinical trial in platinum resistant ovarian cancer patients have shown that intraperitoneal delivery of GEN-1 in combination with Doxil produced an overall clinical benefit of 57.1% (PR=21.4%; SD=35.7%) in patients with measurable disease. The highest percentage of PRs were found at the highest dose level (28.6%) along with the highest percentage of patients achieving SD (57.1%) producing an overall clinical response rate (CR+PD+SD) of 86% at the highest GEN-1 dose cohort, which, despite a small study size, is highly encouraging considering the poor outcome with Doxil alone in this patient population.

GEN-1 has also produced encouraging data in combination with Avastin alone in earlier preclinical studies in a model of ovarian cancer, leading to a significant reduction in tumor burden and disease progression. The inhibition of VEGF by IL-12 through the secretion of interferon-gamma may help explain the impressive synergies between GEN-1 and SoC anti-angiogenic agents like Avastin. These findings open up an additional combination therapy of Avastin with an immune-based therapy like GEN-1.

Preclinical data supporting the combination of GEN-1 with SoC Chemotherapy + Avastin

Results from comprehensive studies confirmed remarkable, statistically significant initial GEN-1 + Avastin findings. Recent studies now show convincingly that GEN-1 when combined with Avastin and Doxil demonstrated a greater than 98% reduction in tumor burden when compared to the untreated control group. The findings represent a statistically significant reduction in tumor burden and disease progression when compared to the combination of Avastin and Doxil in a SKOV3 human cell line implanted into immunocompromised (nude) mice. Analysis of serum chemistry and hematology suggested no overt toxicities associated with the combined treatments. The preclinical data are consistent with the mechanism of action for GEN-1, which exhibits certain anti-angiogenic properties in addition to its well-characterized immunomodulatory activities.

"We believe that the synergy provided to the tumor micro-environment by GEN-1 is responsible for our remarkable preclinical findings," said Michael Tardugno, chairman, chief executive officer and president of Celsion. "Local cellular production and secretion of highly-tolerable endogenous IL-12, a multi-mechanistic anti-cancer agent, both supports doxorubicin’s immune system activating potential as well as reinforces the anti-angiogenic properties of one of the world’s most prescribed anti-cancer biologics, Avastin. We are excited to move forward into clinical trials with the potential for a ground breaking therapeutic approach."

The Company is currently enrolling patients in the OVATION Study, a Phase 1b dose escalating trial combining GEN-1 with neo-adjuvant therapies in newly diagnosed ovarian cancer patients which will provide a starting dose for the follow-on Phase 1/2 study combining GEN-1 with Avastin and Doxil. The Phase 1/2 combination trial is expected to begin in mid-2016.

About GEN-1 Immunotherapy

GEN-1, designed using Celsion’s proprietary TheraPlas platform technology, is an IL-12 DNA plasmid vector encased in a nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein. IL-12 is one of the most active cytokines for the induction of potent anti-cancer immunity acting through the induction of T-lymphocyte and natural killer (NK) cell proliferation. The Company has previously reported positive safety and encouraging Phase I results with GEN-1 given as monotherapy in patients with peritoneally metastasized ovarian cancer, and recently completed a Phase Ib trial of GEN-1 in combination with PEGylated doxorubicin in patients with platinum-resistant ovarian cancer. GEN-1 has also demonstrated preclinical activity in glioblastoma multiforme (brain cancer) and the Company plans to initiate a Phase I study in this indication in the second half of 2015.

Halozyme Doses First Patient In Phase 3 Clinical Trial Of PEGPH20 In Combination With ABRAXANE® And Gemcitabine

On March 16, 2016 Halozyme Therapeutics, Inc. (NASDAQ: HALO), a biotechnology company developing novel oncology and drug-delivery therapies, reported the first patient has been dosed in its Halo-301 | Pancreatic study, a Phase 3 clinical trial in previously untreated metastatic pancreatic cancer patients (Press release, Halozyme, MAR 16, 2016, View Source [SID:1234509581]).

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The global, randomized, double-blind, placebo controlled trial will study Halozyme’s investigational drug, PEGPH20, in combination with ABRAXANE (nab-paclitaxel) and gemcitabine in patients identified to have tumors that accumulate high levels of hyaluronan (HA). HA is a glycosaminoglycan, or chain of natural sugars that may impede access of drugs targeting the tumor. Halozyme’s PEGPH20 temporarily degrades HA and has been shown in animal models to increase the concentration of co-administered therapies in the targeted tumor.

Halozyme also announced that the investigational device exemption (IDE) submitted to the FDA last month by Halozyme’s partner Ventana Medical Systems has been approved. This IDE allows Halozyme to use the Ventana HA CDx Assay as the companion diagnostic in Halo-301 to prospectively identify HA-High patients for inclusion in the study.

"The initiation of our Phase 3 study of PEGPH20 in HA-High pancreatic cancer patients marks a key milestone for the Halozyme team and the PEGPH20 development program," said Dr. Helen Torley, President and CEO of Halozyme.

Dr. Margaret Tempero, Director of the Pancreas Center at University of California, San Francisco and one of the Principle Investigators of the study, said, "High levels of HA have been associated with a worse prognosis in certain cancers, notably pancreas cancer. We have assembled a team of expert investigators at nearly 200 global sites to further study this potential link with the hope of bringing new treatment options to an area of high unmet need."

Halo-301 has an enrollment target of 420 HA-high patients and seeks to improve progression-free survival and overall survival in the patients receiving PEGPH20 plus ABRAXANE and gemcitabine compared to those receiving placebo plus ABRAXANE and gemcitabine.

The FDA granted orphan drug designation to PEGPH20 for the treatment of pancreatic cancer and fast track designation for PEGPH20 in combination with gemcitabine and nab-paclitaxel for the treatment of metastatic pancreatic cancer. Additionally, the European Commission, acting on the recommendation from the Committee for Orphan Medicinal Products of the European Medicines Agency, designated investigational drug PEGPH20 as an orphan medicinal product for the treatment of pancreatic cancer.

8-K – Current report

On March 16, 2016 Provectus Biopharmaceuticals, Inc. (NYSE MKT: PVCT, www.pvct.com), a clinical-stage oncology and dermatology biopharmaceutical company ("Provectus" or "The Company"), reported that the protocol for its phase 3 clinical trial for PV-10 as an investigational treatment for melanoma has been amended to reflect current and evolving standards of care and applicable patient population for a global study in melanoma (Filing, 8-K, Provectus Pharmaceuticals, MAR 16, 2016, View Source [SID:1234509578]).

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Major amendments to the protocol include the addition of talimogene laherparepvec (ImlygicTM) as an option for use as comparator. The amended protocol also extends eligibility to include Stage IV M1a patients having no active nodal or distant cutaneous or subcutaneous metastatic disease. These patients have disease characteristics and prognosis similar to that of the Stage IIIB and IIIC patients that initially defined the study patient population.

In addition, the updated protocol clarifies eligibility for patients not having access to immune checkpoint inhibitors due to standard of care and those not having access to targeted therapy due to standard of care, as well as inclusion of patients who have failed targeted therapy. In the latter case, patients who have failed targeted therapy but meet study eligibility criteria have similar disease manifestations to the remaining study population but have limited treatment options.

Dr. Eric Wachter, CTO of Provectus, said, "These kinds of amendments are commonplace in phase 3 studies and serve to fine-tune the patient population and study procedures to match changing care standards for a large global study. They are the direct result of current and emerging options for these patients and have been developed with extensive input from global leading melanoma investigators. In particular, the most obvious amendment addresses approval in late October of Imlygic by the FDA as the first and only oncolytic viral therapy. As we implement the amended protocol we will assess potential impact on study timelines."

For further information about the study, please visit View Source

Blueprint Medicines Announces Worldwide Collaboration to Accelerate and Expand its Development of Novel Medicines in the Field of Cancer Immunotherapy

On March 15, 2016 Blueprint Medicines Corporation (NASDAQ: BPMC), a leader in discovering and developing highly selective kinase medicines for patients with genomically defined diseases, reported that it has entered into a worldwide collaboration and exclusive license agreement with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. (collectively, Roche) for the discovery, development and commercialization of up to five small molecule therapeutics targeting kinases believed to be important in cancer immunotherapy (Press release, Blueprint Medicines, MAR 15, 2016, View Source [SID1234556115]).

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Under the terms of the agreement, Blueprint Medicines will receive an upfront cash payment of $45 million and will be eligible to receive up to an additional approximately $965 million in contingent option fees and milestone payments related to specified research, preclinical, clinical, regulatory and sales-based milestones across all five potential programs. Of the total contingent payments, up to approximately $215 million are for option fees and milestone payments for research, preclinical and clinical development events prior to licensing across all five potential programs. In addition, the agreement provides for specified royalties and cost sharing, which are described in more detail below.

Immunokinases are intracellular targets known to regulate numerous aspects of immune response and represent an important opportunity for potentially innovative approaches to enhance the immune system’s ability to recognize and eradicate tumor cells. To date, cancer immunotherapies have demonstrated important clinical benefits. However, most cancer immunotherapies have focused on antibodies or combinations with existing approved therapies and have not yet targeted immunokinases with small molecules. This collaboration seeks to develop new mechanisms of modulating the tumor immune response by targeting immunokinases with the goal of enhancing response rates and broadening the utility of using cancer immunotherapies to treat additional cancer types.

"We believe Blueprint Medicines’ proprietary drug discovery platform and expertise in immunokinases, combined with our proven ability to move quickly through drug discovery, is a perfect complement to Roche’s expertise with cancer immunotherapy biology and in developing and commercializing innovative therapies," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "Under this collaboration, Blueprint Medicines will lead preclinical research and development through Phase 1 proof of concept for all five programs and retain U.S. commercial rights for two programs. We believe this highly collaborative relationship will enable us to accelerate our efforts in the emerging field of cancer immunotherapy and to continue building a leading biotechnology company."

The collaboration provides for the worldwide development and commercialization of immunokinases in the field of cancer immunotherapy for up to five small molecule drug candidates as single products or possibly in combination with Roche’s portfolio of therapeutics. Roche’s rights are structured as an option, triggered upon achievement of Phase I proof-of-concept, for an exclusive license to each drug candidate developed under the collaboration. Blueprint Medicines will be primarily responsible for preclinical research and conduct of clinical development for each program prior to any exercise of Roche’s option for such program. If Roche exercises an option for a program, Roche will be responsible for subsequent global development for that program through registrational clinical trials. For up to three of the five programs, if Roche exercises its option, Roche will receive worldwide commercialization rights for the licensed product. For up to two of the five programs, if Roche exercises its option, Blueprint Medicines will retain commercialization rights in the United States for the licensed product, and Roche will receive commercialization rights outside of the United States for such licensed product. Blueprint Medicines will also retain worldwide rights to any drug candidates for which Roche elects not to exercise the applicable option.

For any licensed product for which Roche retains worldwide commercialization rights, Blueprint Medicines will be eligible to receive tiered royalties ranging from low double-digits to high-teens on future net sales of the licensed product. For any licensed product for which Blueprint Medicines retains commercialization rights in the United States, Blueprint Medicines and Roche will be eligible to receive tiered royalties ranging from mid-single-digits to low double-digits on future net sales in the other party’s respective territories in which it commercializes the licensed product. Blueprint Medicines and Roche will share the costs of Phase 1 development for each collaboration target. In addition, Roche will be responsible for post-Phase 1 development costs for each licensed product for which it retains global commercialization rights, and Blueprint Medicines and Roche will share post-Phase 1 development costs for each licensed product for which Blueprint Medicines retains commercialization rights in the United States.