1stOncology™: Cancer Intelligence Service – Page 14402 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Verastem Expands Duvelisib Development Program to Include Peripheral T-Cell Lymphoma

On September 6, 2017 Verastem, Inc. (NASDAQ:VSTM), focused on discovering and developing drugs to improve the survival and quality of life of cancer patients, reported the expansion of its duvelisib development program to include targeting the treatment of patients with Peripheral T-Cell Lymphoma (PTCL) (Press release, Verastem, SEP 6, 2017, View Source [SID1234520381]). Duvelisib has been granted Fast Track designation by the U.S. Food & Drug Administration (FDA) for the treatment of patients with PTCL who have received at least one prior therapy. Duvelisib, Verastem’s lead drug candidate, is an oral inhibitor of phosphoinositide-3-kinase (PI3K)-delta and PI3K-gamma being investigated for the treatment of hematologic cancers, including chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL), indolent non-Hodgkin lymphoma (iNHL) and other T cell lymphomas.

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!

Development of duvelisib in PTCL is supported by compelling Phase 1 clinical data which demonstrated a 50% investigator-assessed overall response rate in 16 heavily pre-treated patients with relapsed or refractory PTCL, including 3 (19%) complete responses and 5 (31%) partial responses. Verastem intends to initiate an open-label, multicenter, Phase 2 clinical trial evaluating the efficacy and safety of duvelisib in patients with relapsed or refractory PTCL by year end 2017. Verastem expects that the Phase 2 study will be conducted in both the U.S. and Japan.
Today, the Company also announced the appointment of Brian Stuglik, RPh, to its Board of Directors. Mr. Stuglik brings to Verastem 35 years of experience in pharmaceutical and oncology commercialization in both the U.S. and international markets. He has successfully launched several multi-billion dollar brands over his career, including Gemzar, Alimta and Erbitux.

"Expansion of the duvelisib clinical development program, and the accompanying receipt of Fast Track designation from the FDA, are important steps in Verastem’s strategy to efficiently develop the potential of duvelisib in additional cancers such as T-cell malignancies," said Robert Forrester, President and Chief Executive Officer of Verastem. "PTCL is a rare and usually aggressive type of NHL where currently available therapies only provide modest benefit. We believe an oral monotherapy like duvelisib could be an important new treatment alternative for patients with T-cell Lymphomas, including PTCL, and we look forward to initiating a Phase 2 study in patients with relapsed or refractory disease by year end."

Mr. Forrester added, "Brian Stuglik is an accomplished executive with significant oncology commercialization expertise who can bring immediate value to Verastem as we now move towards commercializing duvelisib. He brings over 35 years of commercializing important novel oncology drugs together with his extensive external network of clinical thought leaders and deep industry connections. His experience will prove invaluable as we advance duvelisib toward the planned regulatory filing, and potential approval and commercialization."

Most recently, Mr. Stuglik has provided commercial and strategic consultancy services to a variety of life science companies as the founder of Proventus Health Solutions, LLC. Prior to Proventus, he served for over 30 years at Eli Lilly and Company, culminating in his role as Global Vice President and Chief Marketing Officer, Oncology Global Marketing, and advancing Lilly Oncology from a single brand and approved product to a portfolio of over 6 marketed or late-stage compounds across more than 10 cancer types. While at Lilly, Mr. Stuglik helped lead the efforts to acquire Imclone Systems and later led the integration and transition team for Lilly.

More About Fast Track Designation
The FDA defines Fast Track designation as a process designed to facilitate the development and expedite the review of drugs and biologics, to treat serious or life-threatening conditions, and to fill an unmet medical need. Specifically, Fast Track designation facilitates frequent interactions with the FDA review team, including meetings to discuss all aspects of development to support approval, and also provides the opportunity to submit sections of an NDA on a rolling basis as data become available.

About Peripheral T-Cell Lymphoma
Peripheral T-cell lymphoma (PTCL) is a rare, aggressive type of non-Hodgkin lymphoma (NHL) that develops in mature white blood cells called "T cells" and "natural killer (NK) cells"1 which circulate with the lymphatic system.2 PTCL accounts for between 10-15% of all non-Hodgkin lymphomas (NHLs) and generally affects people aged 60 years and older.1 Although there are many different subtypes of peripheral T-cell lymphoma, they often present in a similar way, with widespread, enlarged, painless lymph nodes in the neck, armpit or groin.2 There is currently no established standard of care for patients with relapsed or refractory disease.1

About the Tumor Microenvironment
The tumor microenvironment encompasses multiple tumor and non-tumor cell populations and an extracellular matrix that support cancer cell survival. This includes immunosuppressive regulatory T-cells, myeloid-derived suppressor cells, tumor-associated macrophages, cancer-associated fibroblasts, and extracellular matrix proteins that can hamper the entry and therapeutic benefit of cytotoxic T-cells and anti-cancer drugs. In addition to targeting the proliferative and survival signaling of cancer cells, Verastem’s product candidates, including duvelisib and defactinib, also target the tumor microenvironment to potentially improve response to therapy.

About Duvelisib
Duvelisib is an investigational, dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, two enzymes that are known to help support the growth and survival of malignant B-cells and T-cells. PI3K signaling may lead to the proliferation of malignant B-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.3,4,5 Duvelisib is currently being evaluated in late- and mid-stage clinical trials, including DUO, a randomized, Phase 3 monotherapy study in patients with relapsed/refractory CLL/SLL,6 and DYNAMO, a single-arm, Phase 2 monotherapy study in patients with refractory iNHL.7 Both DUO and DYNAMO achieved their primary endpoints upon topline analysis of efficacy data. Duvelisib is also being evaluated for the treatment of hematologic malignancies through investigator-sponsored studies, including T-cell lymphoma.8 Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

Nordic Life Science Days in Malmö

On September 5, 2017 MonTa Biosciences reported that attends the Nordic Life Science Days in Malmö to meet with investors and pharma partners (Press release, MonTa Biosciences, SEP 5, 2017, View Source [SID1234618637]).

GT BIOPHARMA, INC, ANNOUNCES CLOSING OF OXIS INTERNATIONAL – GEORGETOWN TRANSLATIONAL PHARMACEUTICALS MERGER

On September 5, 2017 GT Biopharma Inc. reported the closing of the merger of GT Biopharma, Inc. (formerly Oxis International Inc.) (OTCQB: OXISD) and Georgetown Translational Pharmaceuticals Inc (Press release, GT Biopharma, SEP 5, 2017, View Source [SID1234539564]). The merger brings GT Biopharma, Inc a new Chief Executive Officer, a robust intellectual property portfolio, and a multi-million dollar financing that leaves the Company well-positioned to continue pursuing its pipeline of oncology and Central Nervous System (CNS) drugs.

As part of the transaction, GTP shareholders were issued 16,927,878 of GT Biopharma (OTCQB: OXISD) common stock. Also, as a requirement of Closing, GT Biopharma completed a financing of over $4.5 million and retired 100% of the company’s debt. The new funding provides capital for GT Biopharma to continue to pursue regulatory approval of the drugs in its oncology pipeline and newly acquired CNS pipeline. Completion of the merger and elimination of all debt is a key milestone accomplishment which positions GT Biopharma to pursue its strategy for acceptance to the Nasdaq Exchange.

The merger closed just days after Gilead Sciences announced it would pay $11.9 billion to acquire Kite Pharma (symbol: KITE) and its CAR-T cancer treatment, shining a spotlight on companies pursuing immunotherapy cancer treatment – including GT Biopharma’s platform targeted immunotherapy BiKE and TriKE technologies.

Anthony J. Cataldo, GT Biopharma’s Executive Chairman said, "This is a major accomplishment for our shareholders. We have now positioned the company to be a significant player in the expanding fight against cancer. In this calendar year we corrected our balance sheet, advanced our product pipeline (OXS-1550 phase 2 FDA trial), signed a partnership agreement with Altor Biosciences, Inc., chaired by Dr. Patrick Soon-Shiong, and advanced our highly valued TriKE platform, which is anticipated to initiate clinical trials in the US in the first half of 2018. GT Biopharma research partners at the University of Minnesota were awarded the NIH REACH award in 2016 to further pursue TriKE technology. This is given by the NIH for technologies judged likely to achieve commercial success. We also brought in an accomplished CEO (Kathleen Clarence-Smith, MD, PhD). She has already demonstrated at several major pharmaceutical companies and at VC-backed start-up companies (most recently Chase Pharmaceuticals) her ability to efficiently and rapidly develop drugs through the FDA process. Additionally, she brings to GT Biopharma a team of highly experienced, seasoned professionals in drug development, and a suite of products which are close to filing an application with the FDA to obtain approval (NDA) to market the products."

"I am pleased to be part of GT Biopharma. Our pipelines in oncology and CNS are bulging and our knowledge and expertise are in place. We are fully prepared to execute on our development plans to shepherd our drugs to commercialization and meet the urgent and expanding needs of patients with oncological and neurological disorders," said Dr. Kathleen Clarence-Smith.

Prior to founding GTP, Dr. Clarence-Smith co-founded Chase Pharmaceuticals Corporation in Washington D.C. and served as Chairman of the company’s Board from 2008 to 2014. Chase Pharmaceuticals was acquired by Allergan, PLC (AGN) in 2016 in a deal that, with significant up-front payment and milestones could reach $1 billion.

Dr. Clarence-Smith also held executive management positions with Sanofi, Roche, Otsuka Pharmaceutical and Prestwick Pharmaceuticals. She is co-founder and a managing member of KM Pharmaceutical Consulting in Washington, D. C.

GT Biopharma shares will trade under the symbol OXISD for before shifting to GTBP later this month.

Georgetown’s CNS pipeline includes Pain Brake for the treatment of chronic neuropathic pain and a drug candidate GTP-004 for the treatment of myasthenia gravis, a rare muscular disease. The only approved drug for this disease (pyridostigmine) carries significant GI side effects, limiting its achievable efficacy. GTP-004 combines pyridostigmine with another approved treatment. The goal is to reduce side effects, providing greater safety, and to improve efficacy over existing protocols involving treatment with pyridostigmine.

A third drug candidate, GTP-011, is a treatment for motion sickness. This is a repurposed version of an existing drug. It was designed to be as effective as the scopolamine patch, but to have fewer side effects, especially fewer memory problems. Reducing or even eliminating memory disturbances is particularly important in elderly patients.

About GT Biopharma, Inc.: GT Biopharma, Inc (formerly known as Oxis International, Inc.) is an immuno-oncology focused company developing innovative drugs focused on the treatment of cancer and other unmet medical needs. Oxis’ lead drug candidate, OXS-1550 (DT2219ARL) is a novel bispecific scFv recombinant fusion protein-drug conjugate composed of the variable regions of the heavy and light chains of anti-CD19 and anti-CD22 antibodies and a diphtheria toxin as its cytotoxic drug payload. OXS-1550 targets and binds to cancer cells expressing the CD19 receptor or CD22 receptor or both receptors. When OXS-1550 binds to cancer cells they internalize the drug and are killed due to the cytotoxic payload. OXS-1550 has demonstrated encouraging results in early human clinical trials in patients with relapsed/refractory B-cell lymphoma or leukemia. OXS-3550 TriKE technology was developed by researchers at the University of Minnesota Masonic Cancer Center. As demonstrated in non-clinical models, this targeted immunotherapy directs NK cells to kill cancer cells while diminishing drug-related toxicity, and is anticipated to be to NK cells what CAR-T is to T-cells.

10-K – Annual report [Section 13 and 15(d), not S-K Item 405]

(Filing, 10-K, MEI Pharma, SEP 5, 2017, View Source [SID1234520389])

MEI Pharma Announces Exclusive License Agreement with Presage Biosciences
for Voruciclib, An Oral, Selective CDK Inhib

September 5, 2017 — MEI Pharma, Inc. (Nasdaq: MEIP), an oncology company focused on the clinical development of novel therapies for cancer, reported that it has entered into a license agreement with Presage Biosciences, Inc. for voruciclib, a clinical-stage, oral and selective cyclin-dependent kinase (CDK) inhibitor. Under the terms of the agreement, MEI Pharma receives exclusive worldwide rights to develop, manufacture and commercialize voruciclib. In exchange, Presage will receive near-term payments of $2.9 million and additional potential payments of up to $181 million upon the achievement of certain development, regulatory and commercial milestones. Presage will also receive mid-single-digit tiered royalties on the net sales of any product successfully developed.
"We are very excited by this opportunity to add voruciclib to our growing pipeline of clinical-stage oncology drug candidates," said Daniel P. Gold, Ph.D., President and Chief Executive Officer of MEI Pharma. "Voruciclib is a selective CDK inhibitor, a class of drugs that has recently demonstrated significant clinical and commercial value, and is differentiated by its potent inhibition of CDK9. This is an attractive asset that comes with an established clinical safety profile, along with compelling pre-clinical data showing suppression of MCL1, a known mechanism of resistance to BCL2 inhibitors, and synergy with the FDA-approved BCL2 inhibitor venetoclax. We believe this provides a clear and efficient clinical development path forward in combination with venetoclax. We appreciate that Presage put their trust in us to execute this plan and we are eager to get started."
Voruciclib (formerly P1446A) has been tested in more than 70 patients in multiple Phase 1 studies and has been associated with manageable side effects consistent with other drugs in its class, including nausea, vomiting and diarrhea. In pre-clinical studies, voruciclib alone induces cell death in multiple patient-derived chronic lymphocytic leukemia (CLL) samples . In addition, voruciclib shows dose-dependent suppression of MCL1 at concentrations achievable with doses that appeared to be generally well tolerated in the Phase 1 studies. Studies have shown that MCL1 is an established resistance mechanism to the B-cell lymphoma 2 (BCL2) inhibitor venetoclax (marketed as Venclexta) .
"Voruciclib is a promising drug candidate with the potential to overcome mechanisms of drug resistance and significantly improve patient outcomes," said David Johnson, Chairman of Presage. "The management team at MEI Pharma has a proven track record in oncology therapeutic development and we believe they have the clinical, regulatory and CMC expertise to maximize the value of this asset. This transaction also enables us to focus our attention on identifying and advancing additional drug candidates and combinations using our powerful CIVO intratumoral microdosing platform.
There are currently two CDK inhibitors approved by the U.S. Food and Drug Administration, palbociclib (marketed as Ibrance) and ribociclib (marketed as Kisqali), both oral, selective CDK 4/6 inhibitors approved for the treatment of hormone receptor-positive, human epidermal growth factor receptor 2 (HER2)-negative breast cancer in combination with hormonal therapy. A third, abemaciclib, was recently granted priority review by the FDA.