Author: [email protected]

CRISPR/Cas9 & TALENs

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!


ArQule Announces Positive Top-Line Results of NIH-Sponsored Phase 2 Trial of Tivantinib in Prostate Cancer

On November 10, 2014 ArQule reported positive top-line results from a randomized, double-blind, placebo-controlled Phase 2 clinical trial of tivantinib as a single agent in metastatic prostate cancer (Press release ArQule, NOV 10, 2014, View Source [SID:1234500944]). This trial (clinicaltrials.gov identifier NCT01519414) was conducted under a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute’s Cancer Therapy Evaluation Program (CTEP) (“A Randomized, Double-Blind, Placebo-Controlled Phase 2 Study of ARQ 197 (tivantinib) in Men with Asymptomatic or Minimally Symptomatic Metastatic Castrate Resistant Prostate Cancer,” NCI Protocol # 8986). The principal investigator was J. Paul Monk, M.D. of The Ohio State University, Arthur G. James Cancer Hospital and Solove Research Institute.

In this trial, 78 patients were randomized 2 to 1 to receive either tivantinib as a single agent or placebo. During a pre-planned analysis, it was found that the trial met its primary endpoint of improving median progression-free survival (PFS) with tivantinib alone as compared to placebo. The results were highly statistically significant. Safety data were consistent with those observed in other trials of tivantinib.

The results of the trial are the subject of ongoing analyses and will be submitted by the investigators for presentation at a future medical conference. As final data emerge from this trial, ArQule and its partner, Daiichi Sankyo Company, Limited, will discuss with the National Institutes of Health (NIH) the potential for additional trials in this indication.

“We are pleased that these significant findings from the NIH trial in prostate cancer add to the body of clinical evidence of the anti-cancer activity of tivantinib across multiple tumor types,” said Paolo Pucci, chief executive officer of ArQule.

“Our CRADA reflects our shared commitment with the NCI to continue to explore the clinical potential of tivantinib in tumor types beyond the compound’s lead indication, hepatocellular carcinoma (HCC), currently in Phase 3 clinical trials,” said Mr. Pucci.

Uncontrolled, signal generation data from additional NIH-sponsored trials with tivantinib in breast cancer and multiple myeloma did not meet their primary endpoint of response rate. As a result, the Company does not plan to prioritize development in these indications at this time.

Genmab Announces New Phase III Combination Study of Daratumumab in Frontline Multiple Myeloma

On November 10, 2014 Genmab reported that the French Intergroup of Myeloma (IFM) in collaboration with Dutch-Belgian Cooperative Trial Group for Hematology Oncology (HOVON) and Janssen Biotech, Inc. (Janssen) plans to start an additional Phase III study of daratumumab in frontline multiple myeloma (Press release Genmab, NOV 10, 2014, View Source [SID:1234500941]). The study (MMY3006) will compare daratumumab in combination with bortezomib, thalidomide and dexamethasone (VTD) to bortezomib, thalidomide and dexamethasone alone as front line treatment for patients who are candidates for stem cell transplantation (SCT). The study is planned to start in Q2 2015. The first Phase III study in front line multiple myeloma was announced in July and is expected to start towards the end of this year, with the two other Phase III front line studies to start next year. In total today’s news is the fifth daratumumab Phase III study to be announced.

“The daratumumab development plan is progressing at a very fast pace and we are very pleased to announce yet another Phase III study. The expansive development program covers different stages of multiple myeloma, and this will be the third Phase III study to enroll patients newly diagnosed with the disease,” said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

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

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!


Scientific Data on PYK2 Inhibition by VS-4718 in Multiple Myeloma Published in the Journal Blood

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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."