On February 19, 2019 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, reported an agreement with Editas Medicine, Inc., a leading genome editing company, to evaluate the potential use of Editas Medicine’s CRISPR technology to edit NAM-NK cells, which are natural killer cells that have been expanded using Gamida Cell’s proprietary nicotinamide-based, or NAM, technology (Press release, Gamida Cell, FEB 19, 2019, View Source [SID1234533505]). Through this agreement, the companies aim to discover optimized NAM-NK cells that could be used to improve the treatment of hematologic malignancies (blood cancers) and solid tumors.

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"We are encouraged by the early data generated in the Phase 1 study of NAM-NK as an investigational therapy for patients with non-Hodgkin lymphoma and multiple myeloma, and we are pleased to have the opportunity to accelerate and broaden our NAM-NK research efforts through this agreement," stated Julian Adams, Ph.D., chief executive officer of Gamida Cell. "By leveraging the collective expertise of the Gamida Cell and Editas Medicine teams, we hope to enhance the efficacy of NAM-NK cells through CRISPR editing and potentially bring life-changing immunotherapy treatments to patients."

"Natural killer cells are increasingly recognized as a potential breakthrough approach to treating various cancers. This agreement with Gamida Cell enables us to combine our industry-leading genome editing platform with Gamida Cell’s proprietary NAM-NK cells in an effort to develop best-in-class cellular medicines," said Charles Albright, Ph.D., chief scientific officer of Editas Medicine.

Under the terms of the agreement, Gamida Cell and Editas Medicine will engage in joint research to evaluate unnamed targets by combining Gamida Cell’s proprietary NAM-based cell expansion technology with Editas Medicine’s CRISPR technology. The research initiative is focused on exploring the potential to edit NAM-NK cells to further optimize their tumor-killing properties, and compare the function of the edited and unedited cells in inducing NK cell tumor clearance.

About NAM-NK
Gamida Cell applied the capabilities of its NAM-based cell expansion technology to highly functional NK cells to develop NAM-NK, an innate immunotherapy for the treatment of hematologic and solid tumors in combination with standard-of-care antibody therapies. NAM-NK addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. NAM-NK is in Phase 1 development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.1

NAM-NK is an investigational therapy, and its safety and efficacy has not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On February 19, 2019 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. based pharmaceutical company with a platform to develop and accelerate the launch of pharmaceutical products and innovative therapeutics in China, U.S., and throughout the world, reported that the National Medical Products Administration (NMPA) has approved the Company’s Clinical Trial Application (CTA) to allow for a confirmatory clinical trial to evaluate the efficacy and safety of ibritumomab tiuxetan injection (ZEVALIN) (Press release, CASI Pharmaceuticals, FEB 19, 2019, View Source [SID1234533487]). Ibritumomab tiuxetan (ZEVALIN) is a U.S. Food and Drug Administration (FDA)-approved product indicated for the treatment of patients with relapsed or refractory, low-grade or follicular B-cell non-Hodgkin’s lymphoma (NHL) and is currently marketed in the U.S. by Spectrum Pharmaceuticals, Inc. (Spectrum). The Company previously acquired greater China rights to ZEVALIN from Spectrum and intends to launch ZEVALIN in China as soon as the confirmatory trial is completed and marketing approval is received from the NMPA.

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The Company currently is addressing certain requirements provided by the Center for Drug Evaluation (CDE), a part of the NMPA; upon satisfying those requirements, the Company will start the ZEVALIN clinical development program in China.

Ibritumomab tiuxetan (ZEVALIN) is also indicated for the treatment of patients with previously untreated follicular non-Hodgkin’s Lymphoma who achieve a partial or complete response to first-line chemotherapy. Ibritumomab tiuxetan (ZEVALIN) builds on the combined therapeutic effects of a monoclonal antibody directed against the CD20 antigen, augmented with a beta-emitting radioisotope. Rituximab and rituximab biosimilars target the same CD20 antigen.

Wei-Wu He, Ph.D., the Company’s Executive Chairman commented, "We are extremely pleased with NMPA’s approval to start the confirmatory clinical trial for ZEVALIN in China. This is another important milestone for CASI as the approval further demonstrates our team’s regulatory strength in working with the NMPA and the effective cross-border operations of our teams in the U.S. and China."

On February 18, 2019 Clarity Pharmaceuticals, a radiopharmaceutical company focused on the treatment of serious disease, is reported that it has raised a total of $8,965,959.75 (Press release, Clarity Pharmaceuticals, FEB 18, 2019, View Source [SID1234533370]). This includes the previously announced strategic investment of $5,000,002.75 from GenesisCare, the largest private provider of cancer treatment in Australia and Europe, and an additional $3,965,957.00 from the undertaking of a Rights Issue to Clarity’s existing shareholders.

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Clarity is now opening the capital raise to external investors for the remaining $1,034,045.75 to complete a total of $10,000,005.50.

The money raised will fund further development of Cu-64 SARTATE and Cu-67 SARTATE, which are next generation, highly targeted, theranostic pharmaceuticals with increased specificity and in vivo stability. Cu-64 SARTATE allows for diagnostic PET imaging, as well as the potential for personalised dosimetry. Cu-67 SARTATE is a potential therapeutic product which could enable treatment for cancers that express the somatostatin receptor 2 (SSTR-2). Clarity commenced its first therapy trial in July 2018 using SARTATE in a serious brain cancer called meningioma and have now reached 50% of our patient recruitment target. This trial of Cu-67 SARTATE will help to pave the way for the use of Cu-67 SARTATE in this patient group as well as using this data as a basis to help expand other indications that express SSTR-2, such as neuroblastoma, an aggressive children’s cancer, and other cancers. Clarity will also continue the development of Cu-64 SARTATE as a diagnostic product for neuroendocrine tumours (NETs) following a successful first-in-human study, completed in 2016.

The capital raising will also fund the development of two other assets in Clarity’s pipeline: a prostate specific membrane antigen (PSMA) targeting product for the diagnosis and treatment of prostate cancer; and a Bombesin (BBN) product that targets the gastrin-releasing peptide receptor, which is present in a number of serious cancers including lung, ovarian, prostate, and breast.

Dr Alan Taylor, Executive Chairman of Clarity Pharmaceuticals, commented, "We are excited to see such strong support from our existing shareholders in this Rights Issue as well as increased interest from a number of strategic companies. This gives us great confidence in our technology, and to continue growing and developing our Company, its capabilities and pipeline in the future. We are also looking forward to welcoming a small number of new investors to our Company given now is the time when the radiopharmaceutical market is gaining significant traction with a number of multibillion-dollar acquisitions in the last few years."

On February 18, 2019 Exicure, Inc. (OTCQB:XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) constructs, reported that its CEO, Dr. David Giljohann, will present at SMi’s RNA Therapeutics 2019 Conference being held February 20-21, 2019 at the Copthorne Tara Hotel in London, UK (Press release, Exicure, FEB 18, 2019, View Source;p=RssLanding&cat=news&id=2387769 [SID1234533371]). Dr. Giljohann’s presentation titled "Spherical Nucleic Acids: Clinical Progress" will occur on Wednesday, February 20, 2019 at 9:10 am GMT.

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On February 18, 2019 Xspray Pharma AB reported the results of a clinical Phase I pilot study with its HyNap-Sora drug candidate (Press release, Xspray, FEB 18, 2019, View Source [SID1234649548]). The study examined HyNap-Sora’s bioavailability compared to the sorafenib cancer drug, currently marketed as Nexavar for the treatment of unresectable hepatocellular carcinoma and advanced renal cell carcinoma. Both investigated HyNap compositions demonstrated significantly increased bioavailability. The variability between subjects in AUC and Cmax was reduced to half compared to Nexavar for one of the HyNap-Sora formulations.
In the completed Phase I clinical trial in 14 healthy subjects, bioavailability of two different formulations of HyNap-Sora was assessed in comparison with Nexavar. The results are positive and the study achieved its primary purpose to show increased bioavailability compared to the originator product. In addition, and as shown in earlier studies with Xspray’s lead product candidate, HyNap-Dasa, the study also showed reduced between subject variability.

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"Sorafenib has a very different pharmacokinetic profile than our product candidates based on nilotinib and dasatinib. Therefore, I’m very pleased that we achieved the primary purpose of this study with sorafenib which enables continuation of the development program. It confirms and strengthens our belief that HyNap formulations of PKIs have the potential to improve both present and upcoming products with poor solubility, improving efficacy and safety enhancing patients’ quality of life during this type of therapy," says Per Andersson, CEO of Xspray.

The clinical results in summary:

• The HyNap formulations were administered at half the dose compared to Nexavar to account for expected increase in bioavailability

• Area under the curve (AUC) was approximately 80 % for the HyNap-Sora formulations compared to Nexavar, indicating that the bioavailability of Sorafenib from HyNap-Sora was 1.6-fold higher

• The maximum concentration of Sorafenib in plasma (Cmax) was approximately 95 % compared to Nexavar.

• The variability between subjects in AUC and Cmax was reduced to half compared to Nexavar for one of the HyNap-Sora formulations.

Sorafenib is a compound with low aqueous solubility and low bioavailability from the sorafenib tablet (Nexavar). The present study demonstrates that the HyNap technology can increase the extent of absorption of sorafenib and reduce between subject variability by improving the solubility.