On September 7, 2016 Curis, Inc. (Nasdaq:CRIS), a biotechnology company focused on the development and commercialization of innovative and effective drug candidates for the treatment of cancer, reported that its collaborator, Aurigene Discovery Technologies Ltd., will receive 10.2 million shares of Curis’s common stock, priced at $2.40 per share, representing a 39% premium to the closing price on September 2, 2016, in lieu of receiving up to $24.5 million of milestone and other payments from Curis that may become due under the companies’ 2015 collaboration agreement.

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In January 2015, Curis and Aurigene established an exclusive collaboration focused on the discovery, development and commercialization of small molecule drug candidates in the fields of immuno-oncology and selected precision oncology targets. Under the collaboration agreement, Curis licensed CA-170, the first orally available, small molecule drug candidate designed to target the immune checkpoints programmed death ligand-1 (PD-L1) and V-domain Ig suppressor of T cell activation (VISTA), and a second candidate, CA-4948 that targets Interleukin-1 receptor-associated kinase 4 (IRAK4). Curis is currently evaluating CA-170 in a Phase 1 trial in patients with advanced tumors, while Aurigene is conducting IND enabling studies with CA-4948. In addition, Curis has selected a second program within the immuno-oncology field of the collaboration that is focused on orally available, small molecule antagonists that are designed to target PD-L1 and T-cell immunoglobulin and mucin domain containing protein-3 (TIM-3) immune checkpoints.

"We are pleased with the progress of our collaboration and drug candidates, and welcome Aurigene’s confidence in Curis as we further advance our pipeline of small molecule checkpoint antagonists," said Ali Fattaey, Ph.D., Curis’s president and CEO. "We are especially excited about the Phase 1 trial of CA-170, the first oral checkpoint inhibitor being taken by patients. We also expect to license a second oral immuno-oncology candidate into Curis later this year that targets PD-1 and TIM3 checkpoint pathways. We look forward to continued progress with Aurigene as we seek to discover and develop multiple first-in-class oral, small molecule checkpoint inhibitors for the treatment of patients with cancer."

"We are delighted with our collaboration that has led to the advancement of the first small molecule checkpoint inhibitor into the clinic, a PD-L1/ VISTA targeting molecule that came out of Aurigene’s discovery efforts over many years," said CSN Murthy, Aurigene’s CEO. "Our investment into Curis exhibits our belief and commitment for this program and beyond as we work with Curis to focus our collective resources to advance these exciting drug candidates."

Further information regarding the terms of the agreement with Aurigene, as documented in the associated Stock Purchase Agreement, Registration Rights Agreement, and Amendment to Collaboration Agreement, was provided by Curis in a form 8-K filed with the SEC today.

On September 7, 2016 In a paper published today in Science Translational Medicine, researchers from Fred Hutchinson Cancer Research Center reported data from an early-phase study of patients with advanced non-Hodgkin lymphoma (NHL) who received JCAR014, a Chimeric Antigen Receptor (CAR) T cell treatment, and chemotherapy (Press release, Juno, SEP 7, 2016, View Source [SID:1234514995]). CAR T cells are made from a patient’s own immune cells that are then genetically engineered to better identify and kill cancer cells.

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The paper reported the results of the first 32 patients in a dose-finding trial of JCAR014 following a round of chemotherapy, called lymphodepletion, designed to create a more favorable environment for the CAR T cells to grow in the patient’s body. Key findings of the study demonstrated the importance of the choice of lymphodepletion regimen and the effects of different doses of CAR T cells. 50 percent of the 18 patients who were evaluable for efficacy after receiving CAR T cells and chemotherapy agents fludarabine and cyclophosphamide (Cy/Flu) had a complete response, which compares favorably to the 8 percent complete response rate in patients who received JCAR014 plus cyclophosphamide-based chemotherapy without fludarabine. As previously reported, dose-limiting toxicities were observed in some patients in this dose-finding study who received the highest CAR T-cell dose. The study continues with the intermediate CAR T-cell dose.

In patients that received Cy/Flu lymphodepletion and the intermediate dose of JCAR014, the data showed a promising early efficacy and side effect profile. Specifically:
Overall Response rate: 82 percent (9/11)
Complete Response rate: 64 percent (7/11)
Severe Cytokine Release Syndrome: 9 percent (1/11)
Severe neurotoxicity: 18 percent (2/11)

JCAR014’s hallmark is its use of a one-to-one ratio of helper (CD4+) and killer (CD8+) CAR T cells, which join forces to kill tumor cells that produce CD19, a molecule found on the surface of many blood cancer cells, including lymphoma and leukemia. By controlling the mixture of T cells that patients receive, the researchers can see relationships between cell doses and patient outcomes that were previously elusive. The data also suggest that with a defined one-to-one composition of cells, efficacy of treatment is increased, while toxic side effects are minimized.

"With the defined composition treatment, we are able to get more reproducible data about the effects of the cells – both the beneficial impact against the cancer and any side effects to the patient," said Fred Hutch clinical researcher Dr. Stan Riddell, one of the senior authors of the paper, along with Dr. David Maloney. "We are then able to adjust the dose to improve what we call the therapeutic index – impact against the tumor, with lower toxicity to the patient."

"This study shows that at the right dose of CAR T cells and lymphodepletion, we can achieve very good response rates for NHL patients who have no other treatment options," said Dr. Cameron Turtle, an immunotherapy researcher at Fred Hutch and one of the study leaders.

For Juno Therapeutics (NASDAQ: JUNO), these insights from the JCAR014 study are key to its development of JCAR017, a similar product candidate for the treatment of CD19 positive blood cancers. Like JCAR014, JCAR017 uses a one-to-one ratio of helper and killer CAR T cells, and the company believes it has the potential to be a "best-in-class" treatment for non-Hodgkin lymphoma, chronic lymphocytic leukemia, and adult and pediatric acute lymphoblastic leukemia. JCAR017 is currently in a phase I, multi-center study.

"We are encouraged by the efficacy and duration of response that we are seeing with defined composition CAR T treatment in patients with lymphoma and other B-cell malignancies," said Mark J. Gilbert, Juno’s Chief Medical Officer. "We hope that the insights from JCAR014 will make it possible to bring more life-saving treatments to more patients with blood cancers."

In addition to Fred Hutch researchers, the study team also included scientists from Juno and the University of Washington. Juno provided one of the trial’s sources of funding, along with the National Institutes of Health, Washington state’s Life Science Discovery Fund and the Bezos Family Foundation.

On September 7, 2016 Selexis SA, a pioneering life sciences company and a global leader in mammalian (suspension-adapted CHO-K1) cell line generation, reported the signing of a license agreement that provides Pieris Pharmaceuticals with access to Selexis SUREtechnology Platform and SURE CHO-M Cell Line to advance the development of PRS-343, Pieris’ lead bispecific immuno-oncology drug candidate (Press release, Selexis, SEP 7, 2016, View Source [SID:1234514986]). PRS-343 is a CD137/HER2 bispecific that is designed to promote CD137 clustering by bridging CD137-positive T cells with HER2-positive tumor cells, thereby providing a potent costimulatory signal to tumor antigen-specific T cells.

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"Through the innovation behind Selexis’ technology, our dedicated researchers are driven to provide companies such as Pieris with high performance, cost-effective solutions for development of their own innovative biological drug candidates," said Marco Bocci, PhD, DPharm, Selexis vice president, licensing and business development. "Earlier this year, Pieris announced encouraging in vivo proof of concept data for PRS-343, a bispecific protein produced with the Selexis SUREtechnology Platform and cell line that is expected to enter the clinic in the first half of 2017. We look forward to the continued progression of this important and unique drug candidate."

Selexis’ proprietary SUREtechnology Platform facilitates the rapid, stable, and cost-effective production of virtually any recombinant protein and provides seamless integration of the biologics development continuum, spanning discovery to commercialization.

The Selexis SURE CHO-M Cell Line is a proprietary high-performance mammalian cell line that is derived from CHO-K1 cells and used for the production of therapeutic recombinant proteins and monoclonal antibodies (mAbs). The growth and production properties of the Selexis SURE CHO-M Cell Line are well defined, and the feed strategy has been optimized, allowing for faster and more efficient scale-up to bioreactors. Therapeutics that are generated using Selexis SURE CHO-M cells are in both clinical trials and marketed products.

On September 7, 2016 Propanc Health Group Corporation (OTCQB: PPCH) ("Propanc" or "the Company"), an emerging healthcare company focusing on development of new and proprietary treatments for cancer patients suffering from solid tumors such as pancreatic, ovarian and colorectal cancers, reported it has submitted an application for Orphan Medicinal Product Designation (OMPD) to the European Medicines Agency (EMA) for PRP, a solution for intravenous administration of pancreatic proenzymes trypsinogen and chymotrypsinogen (Press release, Propanc, SEP 7, 2016, View Source [SID:1234514985]). The proposed orphan drug indication for PRP is for the treatment of pancreatic cancer, responsible for 338,000 cancer diagnoses and 331,000 deaths worldwide in 2012, and a major unmet medical issue.

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"This is a critical step for Propanc and its plans for developing PRP as a treatment solution for aggressive, fast spreading solid tumors. Pancreatic cancer is an area where there is an urgent need for viable solutions and we remain determined to bring to market what could become a targeted and safer treatment approach, which we hope will meaningfully extend patient lives," said James Nathanielsz, Propanc’s Chief Executive Officer. "We will work hard with the authorities to provide a strong rationale for why PRP qualifies for orphan medicinal product designation, which will provide exciting potential benefits to fast track the development process and provide attractive benefits for up to ten years when we achieve market approval."

The European Union (EU) grants Orphan Medicinal Product Designation (OMPD) status to products which treat rare diseases, providing a range of incentives to sponsors developing drugs or biologics. To stimulate the research and development of orphan drugs, in 2000, the EU introduced new legislation with the aim of providing incentives for the development of orphan and other medicinal products for rare disorders. Today, companies with an orphan designation for a medicinal product benefit from incentives such as fee waivers, a 10 year market exclusivity period post authorization for designated products; scientific assistance for marketing authorizations, and the possibility of a Community marketing authorization. The EMA grants OMPD to products that meet one of the following designation criteria:

the life-threatening or debilitating nature of the condition;
the medical plausibility of the proposed orphan indication;
that the prevalence of the condition in the European Union is not more than five in 10,000; or that it is unlikely that marketing the medicinal product in the European Union, without incentives, would generate sufficient return to justify the necessary investment;
that no satisfactory method of diagnosis prevention or treatment exists, or if such a method exists, that the medicinal product will be of significant benefit to those affected by the condition.
The rationale for developing PRP, a formulation of the pancreatic proenzymes trypsinogen and chymotrypsinogen for intravenous administration, in the proposed indication pancreatic cancer, is based on a set of in-vitro studies on cancer stem cells generated from pancreatic cancer cell lines as well as xenograft and syngeneic mouse models of pancreatic cancer. In summary, these data indicate that the dramatic reduction of cellular markers associated with the process of epithelial-mesenchymal transition (EMT) as a consequence of PRP treatment could not only reverse the EMT process with the implication to stop tumor progression and metastasis, but also seem to suppress the development of cancer stem cells (CSCs). Consequently, these results are strong indicators of the therapeutic potential of PRP that could be categorized as an anti-CSC therapeutic drug.

Preliminary early clinical data on the treatment of four patients with pancreatic cancer have been obtained with a rectal formulation of pancreatic proenzymes trypsinogen and chymotrypsinogen in the context of a UK "Specials" License treatment, administered by Dr Julian Kenyon, at the Dove Clinic Center for Integrated Medicine, back in the mid 2000’s. Together, these data support the medical plausibility of the proposed indication and a distinctive benefit-safety profile of PRP for the treatment of pancreatic cancer.