Editas Medicine is developing an editing technology (CRISPR/Cas9 and TALENs) into a novel class of human therapeutics that enable precise and corrective molecular modification to treat the underlying cause of a broad range of diseases at the genetic level (Company Pipeline Editas Medicine, NOV 10, 2014, View Source [SID:1234500942]).

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(Filing, 20-F, Rosetta Genomics, NOV 9, 2014, View Source [SID:1234505276])

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On November 7, 2014 Verastem reported that a paper, titled "PYK2 Promotes Tumor Progression in Multiple Myeloma," has been published in Blood (2014 Oct 23;124(17):2675-86), a peer-reviewed medical journal published by the American Society of Hematology (ASH) (Free ASH Whitepaper) (Press release Verastem, NOV 7, 2014, View Source;p=RssLanding&cat=news&id=1987281 [SID:1234500939]).

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"These data demonstrate that PYK2 is a promising therapeutic target in multiple myeloma," said Jonathan Pachter, Ph.D., Verastem Head of Research. "These results build upon prior scientific findings demonstrating the activity of dual FAK/PYK2 inhibitors across multiple types of cancer and support the ongoing clinical development of VS-4718."

The paper describes the finding that patients with multiple myeloma have a higher expression of proline-rich tyrosine kinase 2 (PYK2), a member of the focal adhesion kinase (FAK) family, compared to healthy individuals, and that PYK2 plays a tumor-promoting role in myeloma progression. The FAK family is composed of just two members, FAK and PYK2, which are highly homologous. In the published study, it was demonstrated that inhibition of PYK2 led to reduction of myeloma tumor growth in vivo as well as decreased cell proliferation, cell cycle progression, and adhesion ability in vitro. In contrast, overexpression of PYK2 was shown to promote the malignant phenotype, as evidenced by enhanced tumor growth and reduced survival. The paper further describes how administration of Verastem’s FAK/PYK2 inhibitor, VS-4718, effectively inhibited myeloma cell growth in both in vitro and in vivo models.

"In addition to this work recently published by our collaborators at the Dana Farber Cancer Institute, we have been conducting further research and collaborating with scientific leaders to understand the potential of cancer stem cell inhibitors in hematological malignancies", continued Dr. Pachter. "We believe that inhibitors of FAK and PYK2, such as VS-4718, may be useful in the treatment of many types of cancer, particularly where there is minimal residual disease with enrichment of cancer stem cells following chemotherapy. We and our collaborators will be presenting additional research at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) in December."

"VS-4718 is currently being evaluated in a Phase 1 dose escalation clinical trial in patients with advanced solid tumors," said Robert Forrester, President and Chief Executive Officer of Verastem. "A new Phase 1 trial of VS-4718 in hematological malignancies is currently planned to begin in the first quarter of 2015."

On November 6, 2014 MacroGenics reported that pre-clinical data on MGD011, a humanized CD19 x CD3 Dual-Affinity Re-Targeting (DART) protein, will be highlighted in a poster presentation at the 56th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), to be held December 6-9, 2014 in San Francisco, CA (Press release MacroGenics, NOV 6, 2014, View Source [SID:1234501246]).

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MGD011 is designed to redirect T-cells to eliminate CD19-expressing cells found in many hematological malignancies and has been engineered to address half-life challenges posed by other programs targeting CD19 and CD3. Moreover, MGD011 and other DART molecules are manufactured using a conventional antibody platform without the complexity of having to genetically modify T cells from individual patients as required by approaches such as chimeric antigen receptor (CAR) T cells. MGD011 has a modified Fc domain, which allows for extended pharmacokinetic properties and convenient dosing at a once-a-week or longer interval. It is one of two new oncology-based DART candidates for which MacroGenics intends to initiate clinical studies in 2015.

"This represents another important milestone for our DART platform," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. "CD19-targeted therapies have generated much excitement, and, based on our pre-clinical data, we believe that MGD011 has great potential in the treatment of patients with certain types of hematological malignancies. We look forward to initiating clinical development in 2015."

On November 6, 2014 Genmab reported additional data from the interim analysis of the ofatumumab (Arzerra) Phase III study, PROLONG (OMB112517) (Press release Genmab, NOV 6, 2014, View Source [SID:1234500929]). The study evaluated ofatumumab maintenance therapy versus no further treatment (observation) in patients with a complete response (CR) or partial response (PR) after 2nd or 3rd line treatment for chronic lymphocytic leukemia (CLL). The improvement in the study’s primary endpoint, progression free survival (PFS), met the prespecified statistical significance level for the interim analysis.

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A total of 474 patients were included in the interim analysis. Patients who received ofatumumab maintenance treatment lived 13.4 months longer without their disease worsening (median PFS) than patients who received no further treatment. Median PFS was 28.6 months for the ofatumumab treatment arm and 15.2 months for the observation arm (Hazard Ratio 0.48; p<0.0001).

The amount of time until patients started their next therapy was significantly longer in the ofatumumab treatment arm than in the observation arm (median 38.0 months vs 27.4 months, Hazard Ratio 0.63; p=0.0076).

There were no unexpected safety findings. Adverse events occurred in 87% of patients in the ofatumumab treatment arm versus 75% in the observation treatment arm. In the ofatumumab treatment arm, 25% of patients experienced grade 3-4 adverse events compared to 17% in the observation arm. Grade 3-4 infections were 18% in the ofatumumab arm and 13% in the observation arm. The two most common grade 3-4 adverse events were neutropenia (22% in ofatumumab arm vs 9% in observation arm), and pneumonia (7% in ofatumumab arm vs 4% in observation arm). The death rate was similar in both arms (14%).

"The interim results of this study show that ofatumumab maintenance therapy significantly extended the amount of time the patients in the study lived without their CLL symptoms getting worse," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

These data will be presented in an oral session at the 56th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in San Francisco, California on December 6 from 12 Noon to 1:30PM PST.