On March 23, 2015 MEI Pharma reported top-line data from a randomized Phase II clinical study of its investigational drug candidate Pracinostat in combination with azacitidine in patients with previously untreated intermediate-2 or high-risk myelodysplastic syndrome (MDS) (Press release, MEI Pharma, MAR 23, 2015, View Source [SID:1234502473]). The double-blind, placebo-controlled study enrolled a total of 102 patients, randomized one-to-one, at 19 sites in the U.S.

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!

According to the top-line data, the combination of Pracinostat and azacitidine showed no difference in the rate of complete remission (CR), the study’s primary endpoint, compared to azacitidine alone. Data from event-driven endpoints, including duration of response, event and progression free survival and overall survival, are immature and will require longer follow-up in order to achieve meaningful conclusions. There were no new or unexpected toxicities observed in the study. Fatigue, gastrointestinal toxicities and myelosuppresion occurred more frequently in the combination group and resulted in a higher rate of drug discontinuations compared to azacitidine alone. The Company expects to present full results of the study at a scientific meeting later this year.

"Our goal when we initiated this study was to build on prior data and rigorously assess the clinical benefit of Pracinostat in combination with azacitidine in MDS," said Daniel P. Gold, Ph.D., President and Chief Executive Officer of MEI Pharma. "While we are disappointed with these top-line response data, we are diligently analyzing the entire data set as well as subsets from this study. Specifically, we are trying to fully assess the impacts of discontinuations on clinically important efficacy outcomes, including duration of response, event and progression free survival and overall survival. These findings will be important to inform the future development path for Pracinostat."

At the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting In December 2014, the company reported significant clinical activity from 33 evaluable patients in an open-label, single-arm Phase II study of Pracinostat and azacitidine in elderly patients with newly diagnosed acute myeloid leukemia (AML). Further follow-up indicates that the response rate and overall survival of these patients continued to increase. To date, 12 patients have been on study for more than six months, including five who have surpassed one year. Data from all 50 patients enrolled in this study have been submitted for presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress in June 2015.

"AML represents another important component of our Pracinostat development strategy," continued Dr. Gold. "We remain encouraged by the durable responses and long-term tolerability observed in our ongoing Phase II study and will continue to monitor these patients closely to get a better estimate of the survival benefit. However, we do not intend to initiate any further studies of Pracinostat and azacitidine until we have gained a more complete understanding of the totality of clinical data surrounding the combination. We expect to be in a position to share more information regarding these findings and future development plans for Pracinostat later this year."

About Pracinostat

Pracinostat is an oral histone deacetylase (HDAC) inhibitor that has been tested in a number of Phase I and Phase II clinical studies in advanced hematologic disorders and solid tumor indications. Pracinostat has been generally well tolerated in more than 300 patients, with manageable side effects often associated with drugs of this class, notably fatigue. Pracinostat has exhibited pharmacokinetic properties in these studies that compare favorably to other oral HDAC inhibitors, including Zolinza (vorinostat) and Farydak (panobinostat).

MEI Pharma owns exclusive worldwide rights to Pracinostat.

On March 23, 2015 GenVec reported that it has formed a collaboration with TheraBiologics to develop cancer therapeutics leveraging both GenVec’s proprietary gene delivery platform and TheraBiologics’ proprietary neural stem cell (NSC) technology (Press release, GenVec, MAR 23, 2015, View Source [SID:1234502469]).

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!

In exchange for an economic participation in the products being developed under the collaboration, GenVec will contribute technology, know-how, vector construction, and technical and regulatory support to the program. TheraBiologics will be responsible for all other development costs.

The initial goal of this collaboration is to generate a next-generation product candidate to TheraBiologics’ TBX02. TBX02 utilizes an established NSC line engineered to express a modified carboxylesterase enzyme that converts the prodrug CPT-11 (Irinotecan) to the potent chemotherapy agent SN-38, which is 1000x more toxic to cancer cells than CPT-11. GenVec’s proprietary adenovectors will be used to provide important manufacturing and potency advantages to other adenovirus-derived vectors and will utilize well-defined production methodologies that have already produced clinical trial material for over 3,000 study patients.

"NSCs offer an unprecedented advantage of therapeutic specificity over conventional cancer treatments as a result of their inherent tumor-tropic properties," stated TheraBiologics founder and chief scientific officer, Karen S. Aboody, MD. "The NSCs selectively target invasive cancer sites resulting in tumor-localized chemotherapy production, sparing the rest of the body from toxic side effects. GenVec’s technology provides us with a well-validated process for modifying these cells to reach their full therapeutic potential."

"TheraBiologics has demonstrated the promise of NSC-mediated approaches to treat cancer and we believe that GenVec’s technology will greatly facilitate the development of these novel treatments," stated Douglas J. Swirsky, GenVec’s president and chief executive officer. "This collaboration is in line with our goal of building a pipeline of exciting opportunities that cost-effectively leverage our technology."

On March 23 Takeda and ImmunoGen reported that Takeda has licensed exclusive rights to use ImmunoGen’s ADC technology – including ImmunoGen’s new DNA-acting IGN payload agents – to develop and commercialize targeted anticancer therapeutics to up to two undisclosed targets (Press release, ImmunoGen, MAR 23, 2015, View Source [SID:1234502463]). The agreement also provides Takeda with the option to take a license for a third target for an additional upfront fee.

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!

ImmunoGen will receive $20 million upfront and – for each target – is eligible to receive milestone payments potentially totaling up to $210 million plus royalties on the commercial net sales of any resulting ADC products. Takeda is responsible for the development, manufacturing and marketing of any ADC products resulting from this agreement.

"Takeda shares our commitment to developing novel anticancer therapies that meaningfully improve the lives of patients, and we look forward to collaborating with them to create important new ADC product candidates," commented Daniel Junius, ImmunoGen President and CEO.

"ADC technology is a critically important tool in addressing unmet needs in oncology," said Christopher Claiborne, Ph.D., Head of the Oncology Drug Discovery Unit at Takeda. "By partnering with ImmunoGen, we are able to leverage this important technology in Takeda’s R&D program and bring novel agents through the clinic."

Takeda signed an agreement with ImmunoGen through its wholly owned subsidiary, Millennium Pharmaceuticals, Inc.

ImmunoGen is not updating its guidance for its 2015 fiscal year at this time.