On January 4, 2019 Aura Biosciences, a leader in the development of novel targeted therapies in ocular oncology, reported that it has received written confirmation from the U.S. Food and Drug Administration (FDA) regarding agreement on the design of its Phase 3 registration trials designed to evaluate light-activated AU-011 for the treatment of patients with choroidal melanoma (Press release, Aura Biosciences, JAN 4, 2019, View Source [SID1234532454]). This written confirmation is the result of successful outcome of an "End of Phase 2" meeting with the FDA.

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!

The STARBRIGHT registration program will consist of two small identical clinical trials designed to assess the safety and efficacy of AU-011 versus sham control for the treatment of patients with small choroidal melanoma and high risk indeterminate lesions. Both trials, which will be titled STARBRIGHT1 and STARBRIGHT3, will be global, multicenter, randomized and masked, and will be conducted in parallel. The primary endpoint will be comprised of a combination of tumor control and vision preservation.

In addition to the design of the Phase 3 trials, the FDA agreed with Aura’s proposed safety database. The FDA also agreed that no further non-clinical studies are needed.

"We are pleased to have received such clear guidance from the FDA with respect to the Phase 3 STARBRIGHT program to be able to meet the scientific and regulatory requirements for marketing approval in the U.S." said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura.

The currently available treatments for choroidal melanoma come with the risk of severe vision loss, especially for patients with melanomas located close to the fovea or optic disk. The ongoing Phase 1b/2 study with light-activated AU-011 has shown that the drug was well-tolerated, with clear evidence of tumor control and preservation of visual acuity at long term follow up.

"We believe that a minimally invasive, non-radiation-based treatment option that enables early intervention while preserving vision has the potential to transform the therapeutic landscape for this difficult to treat, often deadly form of melanoma," said Cadmus Rich, M.D., Chief Medical Officer of Aura. "Overall, the meeting removed any remaining uncertainty on the regulatory path to approval and highlighted FDA’s commitment to guide Aura toward a potential first drug approved for patients with this highly unmet medical need."

About Choroidal Melanoma

Choroidal melanoma is a rare and aggressive type of eye cancer. Choroidal melanoma is the most common primary ocular tumor and develops in the uveal tract of the eye. No targeted therapies are available at present, and current radiotherapy treatments can be associated with severe visual loss and other long-term sequelae such as dry eye, glaucoma, cataracts and radiation retinopathy. The most common current treatment is plaque radiotherapy, which involves surgical placement of a radiation device on the exterior of the eye over the tumor. The alternative is enucleation, or total surgical removal of the eye. Choroidal melanoma metastasizes to the liver in about 40-50 percent of cases in the long term (source: OMF), and only 15 percent of patients whose melanoma has metastasized survive beyond five years after diagnosis (source: ACS).

About Light-Activated AU-011

AU-011 is a first-in-class targeted therapy in development for the primary treatment of choroidal melanoma. The therapy consists of proprietary viral-like particle bioconjugates (VPB) that are activated with an ophthalmic laser. The VPBs bind selectively to unique receptors on cancer cells in the eye and are derived from technology originally pioneered by Dr. John Schiller of the Center for Cancer Research at the National Cancer Institute (NCI), recipient of the 2017 Lasker-DeBakey Award. Upon activation with an ophthalmic laser, the drug rapidly and specifically disrupts the cell membrane of tumor cells while sparing key eye structures, which may allow for the potential of preserving patients’ vision and reducing other long-term complications of radiation treatment. AU-011 can be delivered using equipment commonly found in an ophthalmologist’s office and does not require a surgical procedure, pointing to a potentially less invasive, more convenient therapy for patients and physicians. AU-011 for the treatment of choroidal melanoma has been granted orphan drug and fast track designations by the U.S. Food and Drug Administration and is currently in clinical development.

On January 4, 2019 Sangamo Therapeutics, Inc. (NASDAQ: SGMO) reported that Sandy Macrae, CEO of Sangamo, will present a corporate overview at the 37th Annual J.P. Morgan Healthcare Conference on Wednesday, January 9th at 11:30 a.m. PT in San Francisco (Press release, Sangamo Therapeutics, JAN 4, 2019, View Source [SID1234532471]). Dr. Macrae’s presentation will be followed by a separate Q&A session starting at 12:00 p.m. PT.

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!

The presentation and Q&A session will be webcast live and may be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. The presentation will be archived on the Sangamo website for two weeks after the event.

On January 4, 2019 ViewRay, Inc. (Nasdaq: VRAY) reported the enrollment of the first patient in the Stereotactic MRI-guided On-table Adaptive Radiation Therapy (SMART) Trial, a multi-center, prospective clinical trial for locally advanced or borderline pancreatic cancer (Press release, ViewRay, JAN 4, 2019, View Source [SID1234532472]). The trial will explore the clinical benefits of precise, high dose radiation therapy enabled by MR-guidance combined with daily on-table adaptation in the treatment of pancreatic cancer.

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!

Retrospective analysis of precise, high-dose MR-guided radiation therapy delivered using adaptive dose planning has shown promising results with locally advanced pancreatic cancer, suggesting the potential for improving overall survival relative to patients receiving lower radiation doses, without increasing the rate of serious gastrointestinal toxicity. The compelling nature of the retrospective data prompted the SMART trial, aimed at investigating in a controlled, prospective manner, the robustness of this outcome and tracking quality of life over a 5-year trial period.

"High-definition MR and daily treatment plan adaptation allow us to deliver ablative radiation doses safely to pancreatic cancer patients for the first time ever," said Parag Parikh, M.D., co-PI of the study and Director of GI Radiation Oncology and MR-Guided Radiation Therapy at the Henry Ford Cancer Institute in Detroit. "Through the SMART trial, we will build upon the promising experience from UCLA, Washington University, Amsterdam UMC, University of Miami and University of Wisconsin by further exploring MRIdian’s impact on associated toxicity, local control and patient outcomes in pancreatic cancer at multiple institutions around the world."

The SMART trial is the first prospective, multi-institutional study to deliver ablative doses of radiation to pancreatic cancer patients. It aims to enroll 133 participants with borderline resectable or inoperable locally advanced pancreatic cancer. In the single-arm study, participants will receive radiation therapy at a dose of 50 Gray in 5 fractions (treatment sessions) using ViewRay’s MRIdian. On-table adaptive re-planning will be used when clinically indicated. In all patients, real-time MRI imaging will be used throughout treatment delivery to monitor the target location and control the radiation beam as necessary.

The trial’s primary outcome measure is grade 3 or higher gastrointestinal toxicity in the first 90 days after treatment, with secondary measures including overall survival at two years, distant progression-free survival at six months, and changes in patient-reported quality of life.

"Early evidence on the use of MRIdian to treat locally advanced pancreatic cancer suggests the potential for significantly prolonged survival and lower toxicity rates. Through this rigorously designed study, we hope to further validate the long-term benefits of treatment on the MRIdian," said Scott Drake, President and Chief Executive Officer of ViewRay. "We believe MRIdian’s unique combination of real-time visualization, automated beam control, and daily on-table treatment adaptation has the potential to become the standard of care in radiation oncology."

Along with Parag Parikh, M.D. from Henry Ford Cancer Institute, the trial is led by Percy Lee, M.D. from the University of California Los Angeles.

For more information on the SMART trial, please visit View Source

About the SMART Trial

The SMART trial is designed to enroll 133 participants with borderline resectable or inoperable locally advanced pancreatic cancer. Patients must be 18 years and older and have documented non-metastatic disease after 3 months of systemic therapy. Radiation therapy will be delivered using ViewRay’s MRIdian at a prescribed dose of 50 Gray (Gy) in 5 fractions. Each participant will be aligned in the treatment system with MRI image-guidance. On-table adaptive re-planning will be used when clinically indicated. In all patients, real-time MRI imaging will be used throughout treatment delivery to monitor the target location and control the radiation beam as necessary. The primary outcome measure of the study is grade 3 or higher gastrointestinal toxicity in the first 90 days after treatment. Secondary measures include overall survival at two years, distant progression-free survival at six months and changes in patient reported quality of life (pre-treatment to 12 months post-treatment and for longer periods up to five years).

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.

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!

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."

On January 3, 2019 Bristol-Myers Squibb (NYSE:BMY) reported EPS guidance for full-year 2019 (Press release, Bristol-Myers Squibb, JAN 3, 2019, View Source [SID1234532386]).

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!

Bristol-Myers Squibb is providing its 2019 GAAP EPS guidance range at $3.75 to $3.85 and non-GAAP EPS guidance range at $4.10 to $4.20. Key 2019 GAAP and non-GAAP guidance assumptions include the combined dilution of $0.09 from the UPSA divestiture and U.S. Pension liabilities transactions.

The EPS guidance for 2019 excludes the impact of the Celgene acquisition or any potential future strategic acquisitions and divestitures, and any specified items that have not yet been identified and quantified. The non-GAAP 2019 EPS guidance also excludes other specified items as discussed under "Use of Non-GAAP Financial Information."

Bristol-Myers Squibb will provide full line-item guidance for when the company reports its results for the fourth quarter 2018 on January 24, 2019.

Bristol-Myers Squibb is providing its 2019 GAAP EPS guidance range at $3.75 to $3.85 and non-GAAP EPS guidance range at $4.10 to $4.20. Key 2019 GAAP and non-GAAP guidance assumptions include the combined dilution of $0.09 from the UPSA divestiture and U.S. Pension liabilities transactions.

The EPS guidance for 2019 excludes the impact of the Celgene acquisition or any potential future strategic acquisitions and divestitures, and any specified items that have not yet been identified and quantified. The non-GAAP 2019 EPS guidance also excludes other specified items as discussed under "Use of Non-GAAP Financial Information."

Bristol-Myers Squibb will provide full line-item guidance for when the company reports its results for the fourth quarter 2018 on January 24, 2019.