On January 22, 2014 Cancer Research Technology reported that an innovative screening technology that tags compounds with unique strands of DNA – like barcodes – will be used to assess up to a billion prototype drug molecules for anti-cancer activity, under a collaboration between the Institute of Cancer Research, London, Cancer Research Technology (CRT) and Denmark-based drug discovery company Nuevolution A/S (Press release, Cancer Research Technology, JAN 22, 2014, View Source [SID1234523231]).

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Researchers will use Nuevolution’s novel screening technology, Chemetics, to screen libraries of DNA-tagged compounds to identify those that act on a key protein in the stress response pathway, which has an important role in cancer cell survival and resistance to cancer treatments.

The collaboration will give scientists at The Institute of Cancer Research (ICR) access to data from screens of Nuevolution’s proprietary library of small-molecule compounds, each of which is tagged with a unique strand of DNA – marking it like a barcode. Up to a billion compounds will be assessed, with the successful hit compounds identifiable through their DNA tag. This state-of the-art screening technology allows potent drug leads to be identified quickly, accurately and from very large and complex compound mixtures.

The three-way deal between the ICR, Nuevolution and CRT, the commercial arm of Cancer Research UK, builds on a previous collaboration between CRT and Nuevolution, which aimed to identify drug leads that block the activity of several challenging cancer targets of therapeutic interest.

Under the new deal the Cancer Research UK Cancer Therapeutics Unit at the ICR and Nuevolution will collaborate to screen a key target within the stress response pathway using Nuevolution’s Chemetics technology. Researchers from the Cancer Research UK Cancer Therapeutics Unit at the ICR will provide detailed insights and scientific expertise on the specific stress pathway target as well as their extensive experience in cancer drug discovery and development. In addition, they will use their cancer biology and molecular pharmacology expertise to validate prototype drug molecules identified by the Chemetics screening.

The parties have an option to co-develop promising compounds arising from this collaboration. The agreement is open-ended and allows for the screening of additional targets if the collaboration is successful.

Professor Paul Workman, deputy chief executive of The Institute of Cancer Research, London, and director of the Cancer Research UK Cancer Therapeutics Unit said: "The stress response pathway plays a key role in allowing cancer cells to survive and to develop drug resistance – so it is increasingly being seen as an exciting source of future drug targets.

"But for some of these targets it is technically very challenging to identify prototype small molecule drugs. The new collaboration between the ICR, Cancer Research Technology and Nuevolution will allow us to screen very rapidly and efficiently for compounds that are able to bind to a key component of the stress response pathway that we have identified as especially important, and could help us to identify new drug candidates far more quickly than would otherwise be the case. By working in partnership we can accelerate the potential for patient benefit."

Thomas Franch, CSO, Nuevolution A/S said: "We are delighted to enter this project and to expand our present collaborations with ICR and CRT. The project will address a highly challenging target for which small molecule compounds is not easily identified using conventional screening methods. We hope to identify lead compounds using the Chemetics technology and look forward to moving this exciting project forward together with the world-leading team at ICR".

Dr Phil L’Huillier, Cancer Research Technology’s director of business management, said: "Our role is to build global industry-academic partnerships to bring the best technologies and expertise together to develop new treatments for cancer patients – ultimately saving more lives from the disease. This exciting international collaboration combines global expertise and resources to exploit the untapped biology of the stress response pathway.

"This work will accelerate the identification of potential new cancer drugs though an innovative approach to scan for DNA ‘barcode’ tags on promising new molecules – extending the existing relationship between Nuevolution and CRT."

On January 21, 2014 PIQUR Therapeutics AG, focused on the discovery and development of innovative anti-cancer drugs, reported that it has successfully completed the Series A financing round (Press release, PIQUR Therapeutics, JAN 21, 2014, View Source [SID1234527276]). The private placement was rapidly oversubscribed. Subscribers include existing shareholders as well as new investors from the life science industry.

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Dr. Vladimir Cmiljanovic, CEO and co-founder of PIQUR, commented: "We are delighted by the trust of investors in our company. In particular, we successfully attracted further distinguished individuals from the life science industry as investors. The successful round of funding has encouraged us in our determination to continue pursuing our corporate strategy. The funds that have been procured secure the cost of the first clinical studies with our lead compound PQR309."

PIQUR has received approval from the authorities in Switzerland and UK to conduct the clinical Phase I study with PQR309. This study will assess the safety in cancer patients, and will help determine the ideal dosage. On January 2, 2014, PQR309 was administered to the first patient at the University Hospital in Basel. "For PIQUR, the very first administration of PQR309 represents an important milestone in clinical development. First results of the study are expected to be released before the end of 2014," said Prof. Dr. Richard Herrmann, Chief Medical Officer of PIQUR.

Also of crucial importance for the future development of the company is the broadening of the license agreement with the University of Basel. The license agreement with the University of Basel was previously limited to the field of cancer, and has now been extended to a broad range of promising clinical indications. "The expansion of the license agreement is very important for the progress and development of our company. We now have the unlimited freedom to assess the therapeutic benefit of PQR309 and all other research programs in other therapeutic fields, such as immunology, neurology and ophthalmology," pronounced Prof. Dr. Bernd Giese, Chairman of the Board of Directors.

To turn cancer into a manageable disease
PIQUR aims to help turn cancer into a manageable disease. Despite significant advances in the fight against cancer, there remains a high unmet medical need for therapies that not only prolong patients’ survival but also significantly improve quality of life. PIQUR targets both PI3K (phosphoinositide 3-kinase) and mTOR (mammalian target of rapamycin), two key signaling molecules that are vital to several essential biological processes, such as cell proliferation and survival, making inhibition of the target attractive for cancer therapy.

PIQUR’s differentiation is the level of innovation with its unique, proprietary fragment and scaffold libraries, as well as cellular technology platforms, and their excellent products with novel, dual-acting ‘strong PI3K plus fine-tuned mTOR’ inhibitors that address the given challenges, meeting therapeutic, tolerance and galenic needs.

On January 17, 2014 Takeda reported that the Japanese Ministry of Health, Labour and Welfare (MHLW) has approved the New Drug Application of ADCETRIS (brentuximab vedotin) for the Treatment for Patients with CD30 Positive Relapsed or Refractory Hodgkin Lymphoma (HL) or Relapsed or Refractory Anaplastic Large Cell Lymphoma (ALCL) (Press release Takeda, JAN 17, 2014, View Source [SID:1234500441]).
The application approval was based on a phase 1/2 clinical trial in Japanese patients with relapsed or refractory CD30-positive HL and patients with relapsed or refractory CD30-positive systemic ALCL, and data from two global pivotal phase 2 clinical trials. In March 2012, MHLW granted ADCETRIS orphan drug designation for the treatment of patients with HL and ALCL, and therefore was targeted for priority review in Japan.

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On January 15, 2014 Nordic Nanovector reported that the U.S. Patent and Trademark Office has issued patent no. 8628749 entitled "RADIOIMMUNOCONJUGATES AND USES THEREOF" (Press release Nordic Nanovector, JAN 15, 2014, View Source [SID:1234500625]). The issued claims cover Nordic Nanovector’s proprietary radioimmunotherapy technology including the company’s lead product candidate Betalutin. The expiry date for the patent is 2031.

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"The issuance of this patent supports our investment in Betalutin, which is currently being developed as a treatment for patients with relapsed B-cell Non-Hodgkin lymphoma," said Jan A. Alfheim, Chief Executive Officer.

"We plan to pursue additional patents to expand the global patent protection of our proprietary technology in order to derive maximum commercial value for our shareholders. We will continue to work diligently toward growing our increasing portfolio of patents while developing and commercializing our radioimmunotherapy product candidates"

On January 13, 2014 NantBioScience, subsidiary of NantWorks, LLC, a company focused on the discovery and development of innovative treatments for diseases with high unmet medical needs,reported a strategic collaboration with Celgene to advance bold research programs to benefit cancer patients in need of new therapeutic solutions (Press release, NantBioScience, JAN 13, 2014, View Source [SID:1234514808]). As part of the transaction, Celgene will pay $75 million in funding to NantBioScience as an upfront option fee and equity investment.

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Building on the nab (nanoparticle albumin-bound) technology platform, NantBioScience will create a pipeline of nab-based molecules. As part of the collaboration, Celgene will license two nab product candidates to NantBioScience, both of which had previously received Investigational New Drug (IND) approval. The first product candidate (NTB-011) is a nanoparticle albumin-bound formulation of a novel colchicine dimer with cytotoxic and vascular disrupting properties. The second product candidate (NTB-010) is a nanoparticle albumin-bound formulation of the geldanomycin analogue, 17-AAG, a potent HSP90 inhibitor, which is planned to be studied in patients with a variety of hematological and solid tumors. Phase I clinical trials for these product candidates are being planned for initiation in 2014 – 2015.

The objective of NantBioScience is to innovate drug development by testing molecularly targeted drugs, based on the molecular profile of the patient’s tumor, independent of the cancer’s anatomical type. With capabilities of next generation sequencing and targeted proteomics, each cancer may now be viewed as a series of rare diseases. These comprehensive "omic" analytic tools and "big data" generated from supercomputing have been previously untapped on the scale now available in the field of drug development. NantBioScience and NantWorks are uniquely positioned to develop molecularly designed drugs in this era of genomics and proteomics, by identifying patients and their tumor signature at the most granular cellular, DNA and protein levels. Patients entering clinical trials would be identified after a comprehensive "omic" analysis from tissue to cell to DNA to RNA to protein to peptide to drug, and tested based on this molecular profile to maximize clinical outcome and minimize side effects.

"We intend to make obsolete the standard method of clinical trial design of ‘trial and error’ and replace it with a level of quantitative predictability based on both the genomic and proteomic profile performed a priori. We also intend to make obsolete the common understanding that cancer treatments, developed under the age old dogma of ‘maximum tolerated dose,’ may work but only by wreaking terrible side effects, bringing patients to the brink of death," said NantWorks founder, Dr. Patrick Soon-Shiong. "Celgene has been a strong steward for Abraxane which is now approved for metastatic breast, lung and pancreatic cancer. This new partnership will enable us to aggressively advance our drug pipeline and put us one step closer to developing – and then delivering – molecular designed cancer treatments for patients to receive the right care at the right time."

"We have invested over $100 Million in the pursuit of this platform to date and are very excited to have Celgene as our partner in this pursuit," he said.

"Celgene is excited to team up again with Dr. Patrick Soon-Shiong, the creator of Abraxane and the founder of the nab technology platform. We are committed to his vision of molecularly driven personalized medicine and to collaborating with NantBioScience at the forefront of this era where genomic- and proteomic- based solutions may provide the path to a cure for cancers," said Bob Hugin, Chairman and Chief Executive Officer of Celgene Corporation.

In addition to the two nab product candidates, NantBioScience has a broad R&D program to discover new compounds that are specifically targeted at tumor signaling pathways in patients with specific genetic mutations. A novel inhibitor of oncogenic KRAS is showing promise in early development studies and will advance into a program of IND enabling studies in 2014. Normally a proto-oncogene, KRAS, when mutated to an oncogene, is transformed into the driver of tumorigenesis in pancreatic cancer amongst many other cancers and molecular profiles.

NantBioScience’s pursuit of targeted therapies includes the discovery and development of drugs that remediate the activities of the often mutated tumor suppressor, p53. Because the loss of p53 functionality is a driver for the development of over 50% of all cancers and because p53 is responsible for maintaining the integrity of the cellular genome, pharmaceuticals targeting cells harboring p53 mutations are highly coveted. NantBioScience plans to initiate IND-enabling studies on its lead p53 remediating compound, for which NantBioScience is targeting a first-in-man study in 2015.

NantBioScience’s pipeline also contains novel potent multi-kinase inhibitors. These compounds are entering into a program of IND-enabling studies, with first-in-man studies planned for 2016. NantBioScience’s kinase inhibitor program is further buoyed by its library of >4,000 multi-kinase inhibitors which are currently under investigation for a variety of indications and molecular profiles.

As part of the collaboration, Celgene will receive an option to license a certain number of product candidates developed by NantBioScience, including the two nab product candidates to be licensed to NantBioScience. The options may be exercised by Celgene through completion of Phase I clinical studies.

The transaction is subject to customary closing conditions, including the expiration or termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, and is expected to close in the first quarter of 2014.