Tragara Pharmaceuticals Appoints Scott Megaffin as Chief Executive Officer and Changes Name to Adastra Pharmaceuticals

On January 3, 2019 Tragara Pharmaceuticals Inc. reported the appointment of Scott Megaffin as Chief Executive Officer and a member of the Board of Directors effective immediately (Press release, Tragara Pharmaceuticals, JAN 3, 2019, View Source [SID1234539282]). Megaffin brings extensive leadership experience in clinical stage companies in oncology, specialty and critical care. He replaces Tom Estok, who will remain active with the company as an advisor and continue in his role on the Board of Directors.

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The addition of Megaffin comes at the time of a strategic pivot for the company that includes a change of name to Adastra Pharmaceuticals Inc. As part of this strategy, Adastra has expanded its operations beyond San Diego with the opening of an office in Princeton, N.J. This is consistent with the Adastra strategy to grow and attract new pharmaceutical talent in the New Jersey area. Adastra, which is derived from the Latin phrase "to the stars," is a name that reflects the boundless motivation and dedication of each team member to the development of best-in-class therapeutics for patients with cancer.

"Scott has the right experience to drive forward all of the Adastra strategic imperatives and continued program advancement of TG02," said Dennis Podlesak, Adastra Chairman and Partner of Domain Associates. "The Board would like to acknowledge and thank Tom for his vision and dedication in getting us to this point. We have advanced TG02 in clinical studies in collaboration with healthcare providers in the United States and Europe for adult patients diagnosed with glioblastoma multiforme (GBM). Furthermore, we have prepared for the start of an exploratory study in pediatric patients with diffuse intrinsic pontine glioma (DIPG). Between now and the first quarter of next year, Adastra will have a series of clinical milestones and data read-outs that we expect will enable the company to explore multiple development and business paths forward."

Prior to joining the company, Megaffin was President of Churchill Pharmaceuticals LLC, where he successfully marshalled the company through a transaction that resulted in the sale of company assets before an FDA approval, while simultaneously preparing the company for its own possible commercialization activities. Before this, he held numerous global strategic and operational positions of increasing responsibility with Onconova Therapeutics Inc., Cephalon Inc., Adolor Corp., Yamounchi and Bristol-Myers Squibb Co. He has led six global product launches and clinical development programs in a variety of therapeutic categories, including oncology, hematology, virology, critical care, anti-infectives, pain and inflammation. Megaffin earned a B.S. in Biology from Pittsburg State University.

TG02 is a highly differentiated orally delivered inhibitor of cyclin-dependent kinase 9 (CDK9) with high penetration of the blood brain barrier, overcoming a major challenge in GBM drug development. TG02 exerts inhibitory effects on RNA Polymerase II phosphorylation, leading to depletion of the key cancer cell survival protein, Myc, thus committing cancer cells to apoptosis. TG02 has demonstrated broad anti-tumor activity across cell lines and shows a positive synergistic effect when added to standard of care treatments for GBM. Currently, the National Cancer Institute (NCI) and European Organisation for Research and Treatment of Cancer (EORTC) are enrolling patients in trials of TG02 in GBM. In addition, Adastra will initiate a study of TG02 in DIPG in the second quarter of this year.

Megaffin added, "I am very happy to take on this role at Adastra during this transformational year for the company. The TG02 trials in GBM are progressing nicely, and we are entering into the start of the DIPG study in the pediatric patient population. When evaluating the opportunity to join the company, I reviewed the data in hand for TG02 and became excited by its potential for the benefit of patients. GBM and DIPG are both devastating brain cancers and represent high unmet treatment needs. TG02 presents Adastra with regulatory opportunities of rapid advancement into registrational studies in the near future. We look forward to reporting our progress in 2019 as we focus sharply on clinical programs as well as exploring possible pipeline expansion opportunities."

Deciphera Pharmaceuticals Initiates a Phase 1b/2 Clinical Trial of Rebastinib in Combination with Carboplatin in Patients with Advanced or Metastatic Solid Tumors

On January 3, 2019 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH), a clinical-stage biopharmaceutical company focused on addressing key mechanisms of tumor drug resistance, reported that it has initiated an open-label, multicenter, Phase 1b/2 study of rebastinib, the Company’s investigational small molecule switch control inhibitor of TIE2 kinase, in combination with carboplatin in patients with advanced or metastatic solid tumors (Press release, Deciphera Pharmaceuticals, JAN 3, 2019, View Source [SID1234532385]).

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"We are extremely pleased with the recent progress made on our rebastinib program, including today’s announcement that we have initiated a second Phase 1b/2 clinical study of rebastinib in combination with chemotherapy," said Michael D. Taylor, Ph.D., President and Chief Executive Officer of Deciphera. "This second clinical study will evaluate rebastinib in combination with carboplatin, a treatment regimen that, based on preclinical observations, we believe has the potential to be an important new approach to treating patients with advanced solid tumors."

Part 1 (3+3 dose escalation) of this two part study is designed to evaluate the safety, tolerability and pharmacokinetics of 50 mg and 100 mg rebastinib twice daily (BID) when administered in combination with carboplatin, and to determine the recommended phase 2 dose (RP2D) of rebastinib in combination with carboplatin, in patients with advanced or metastatic solid tumors that are refractory to standard therapies. In part 2, the safety, tolerability and efficacy of the RP2D of rebastinib in combination with carboplatin administered once every 3 weeks will be assessed across multiple disease cohorts, including: breast cancer, ovarian cancer, and mesothelioma. This trial is expected to enroll up to 117 patients in total, with approximately 18 patients in part 1 and up to 99 patients in part 2. For more information about the clinical trial design please visit www.clinicaltrials.gov (NCT03717415).

"The biological mechanisms by which tumors co-opt the surrounding microenvironment to grow, survive and become more invasive, are becoming better understood. Recent research indicates that TIE2 kinase is involved in multiple mechanisms favoring a pro-tumoral microenvironment, including the regulation of a population of immunosuppressive macrophages, promotion of tumor angiogenesis, and function of perivascular pumps that lead to tumor cell recruitment and metastasis," said Daniel L. Flynn, Ph.D., Chief Scientific Officer of Deciphera. "In addition, use of chemotherapeutic agents are believed to enhance the recruitment of these macrophages, leading to increased tumor vascularization and dissemination. In preclinical studies rebastinib has been shown to block these unintended effects of chemotherapy, providing rationale for the potential combination of rebastinib with chemotherapy."

Preclinical information on the role of TIE2 kinase was published in Molecular Cancer Therapeutics, 2017; 16: 2486-2501 and Science Translational Medicine, July 5, 2017; 9: eaan0026.
(www.deciphera.com/science/presentation-publications/)

About Rebastinib
Rebastinib is an investigational, orally administered, potent and selective inhibitor of the TIE2 kinase, the receptor for angiopoietins, an important family of vascular growth factors in the tumor microenvironment that also activate pro-tumoral TIE2 expressing macrophages. In a Phase 1 clinical study, biomarker data have demonstrated rebastinib-induced increases in the TIE2 ligand angiopoietin 2, secondary to TIE2 inhibition. Rebastinib is currently being evaluated in a Phase 1b/2 clinical study in combination with paclitaxel (NCT03601897), in a Phase 1b/2 clinical study in combination with carboplatin (NCT03717415), and in an investigator sponsored Phase 1b trial in patients with metastatic breast cancer in combination with paclitaxel or eribulin (NCT02824575).

Verastem Oncology to Present at the 37th Annual J.P. Morgan Healthcare Conference

On January 3, 2019 Verastem, Inc. (Nasdaq:VSTM) (Verastem Oncology or the Company), focused on developing and commercializing medicines to improve the survival and quality of life of cancer patients, reported that the Company will present at the 37th Annual J.P. Morgan Healthcare Conference on Thursday, January 10, 2019 at 11:00 a.m. PST in San Francisco, CA (Press release, Verastem, JAN 3, 2019, View Source;p=irol-newsArticle&ID=2381972 [SID1234532403]).

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A live webcast of the presentation will be available on the investors section of the Company’s website at www.verastem.com. An archived presentation will be available for 90 days.

Personal Genome Diagnostics Collaborates with Merck on Global Clinical Trial Evaluating Response to Dual Biomarker Directed Precision Oncology Combination Therapy

On January 3, 2019 Personal Genome Diagnostics Inc. (PGDx), a leader in cancer genomics, reported that its 500+ gene pan-cancer tumor profiling tissue assay is being used in Merck’s Phase 2 precision oncology KeyImPaCT study of biomarker-directed, pembrolizumab-based combination therapy for advanced non-small-cell lung cancer (NSCLC) (KEYNOTE-495) (Press release, Personal Genome Diagnostics, JAN 3, 2019, View Source [SID1234532419]). This study will investigate the utility of biomarker-based triage for study participants with previously untreated advanced NSCLC. The PGDx elioTM tissue complete assay is being used during the enrollment process for the analysis of tumor mutational burden (TMB) status.

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Study participants within groups defined by a biomarker-based classifier (gene expression profile [GEP] and TMB) will be randomized to receive pembrolizumab in combination with MK-4280 or lenvatinib. The study will involve more than 70 clinical sites worldwide.

"We are proud to collaborate with Merck on this trial. Merck is a leader in immune oncology and we view their selection of PGDx as a testament to our capabilities around TMB," said Doug Ward, Chief Executive Officer at PGDx. "With the kitted approach for the PGDx elio portfolio, we aim to expand patient access to comprehensive, local genomic testing and reduce turn-around time for results that inform critical treatment decisions for patients worldwide."

PGDx provides genomic solutions from biomarker discovery to companion diagnostic development through its CAP/CLIA certified laboratory and is developing a portfolio of regulated tissue-based and liquid biopsy genomic products to enable local next-generation sequencing (NGS) testing in laboratories worldwide.

Ultragenyx to Present at J.P. Morgan Healthcare Conference

On January 3, 2019 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, reported that Emil D. Kakkis, M.D., Ph.D., the company’s Chief Executive Officer and President, will present at the 37TH Annual J.P. Morgan Healthcare Conference on Tuesday, January 8, 2019 at 4:00 pm PT in San Francisco (Press release, Ultragenyx Pharmaceutical, JAN 3, 2019, View Source [SID1234532435]).

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The live and archived webcast of the company presentations will be accessible from the company’s website at View Source The replay of the webcast will be available for 90 days.