On January 3, 2019 GT Medical Technologies, Inc., a company dedicated to improving the lives of patients with brain tumors, reported positive results from a clinical trial supporting the efficacy and safety profile of a new treatment, GammaTile Therapy, for patients with recurrent, previously treated brain tumors known as meningiomas (Press release, GT Medical Technologies, JAN 3, 2019, View Source [SID1234532431]). Meningiomas are the most common type of primary brain tumor. They are usually non-cancerous but can have a significant impact on patients’ lives, causing headaches, seizures, cognitive decline, and other life-threatening symptoms. Results were published in the Journal of Neurosurgery (JNS), the official journal of the American Association of Neurological Surgeons.

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In this single-arm, prospective study, 20 recurrent meningiomas were treated with adjuvant GammaTile Therapy immediately following surgery. The study found that in patients undergoing surgery followed by GammaTile, there was a statistically significant improvement in time-to-local disease progression (TTP) compared to the same patients’ prior rounds of treatment. At the time of the analysis, median TTP for tumors treated with GammaTile had not been reached because less than half of the patients had experienced tumor recurrence. Median TTP for tumors treated with GammaTile is projected to be at least 29 months (95 percent confidence interval) – nearly a year longer than results for prior rounds of treatment (18.3 months TTP, HR=0.17, p=0.02). At 18 months post-treatment, tumors had not recurred in 89 percent of patients treated with GammaTile, compared to 50 percent for prior rounds of treatment without GammaTile. Treatment with GammaTile was well tolerated.

GammaTile is an FDA-cleared, surgically targeted radiation therapy (STaRT) for patients with recurrent intracranial neoplasms (brain tumors) including primary (benign or malignant) and metastatic tumors. Placed directly at the site of the tumor cavity during the last few minutes of excision surgery, GammaTile Therapy is a new approach that immediately begins targeting residual tumor cells, before they can replicate. Designed to help protect healthy brain tissue and facilitate rapid, accurate placement during the procedure, the therapy features a bioresorbable, conformable, 3D-collagen tile and uniform radiation source.

GammaTile Therapy offers advantages over the most common treatment for patients undergoing surgery for recurrent brain tumors: a course of External Beam Radiation Therapy (EBRT), which requires daily treatments for up to six weeks. Some patients may not be candidates for EBRT. Once the disease has returned, many people with recurrent brain tumors have already received levels of radiation therapy that make the risk of additional external beam radiation outweigh the potential benefits. Additionally, those patients who are potentially candidates for EBRT typically have to wait two weeks or more for surgical wound healing before beginning treatment, giving any residual tumor cells a chance to replicate.

"In patients with recurrent meningiomas, treatment options are extremely limited. Repeat surgery may not be a good option without an effective adjuvant therapy. With GammaTile, we can now offer patients who otherwise would not have been able to receive treatment or who would likely be facing early recurrence another option – one proven to delay local meningioma tumor progression out to two years," said Peter Nakaji, M.D., co-author of the study and GT MedTech’s co-founder and director of the Neurosurgery Residency Program at Barrow Neurological Institute. "Because it is delivered directly to the tumor bed, GammaTile offers the benefits of radiation while minimizing damage to surrounding healthy tissue from EBRT, and reduces the need for patients to return for daily outpatient radiation treatments."

Because the therapy is implanted at surgery, patients treated with GammaTile Therapy require no additional trips to the hospital or clinic for radiation therapy. The therapy is targeted, so patients receive radiation only where it is needed and may receive a lower overall level of exposure of normal tissue to radiation. GammaTile Therapy can emit two-and-a-half times the radiation dose compared to the dose that can be achieved from EBRT. This dose is delivered to a localized area and is highly lethal to residual tumor cells.

Approximately 400,000 Americans are newly diagnosed with some type of brain tumor each year.1 Despite the efforts of the most skilled brain tumor specialists throughout the world, outcomes for patients with brain tumors have improved little over the past 30 years. Recurrence of brain tumors is common, and about half of all patients treated for brain tumors have their disease recur within a year.

"As a treating physician, I have seen first-hand the need for better options for our patients. We created GammaTile as a therapy designed to be immediate, safe, predictable, and effective," said GT MedTech’s co-founder and chief technology officer David G. Brachman, M.D., lead author of the study, who previously served as chairman and medical director of Radiation Oncology at St. Joseph’s Hospital and Barrow Neurological Institute in Phoenix, Ariz. "These first published clinical data on the technology demonstrate that GammaTile is an effective therapy option that significantly delays the progression of this common brain tumor type."

GammaTile Therapy received FDA 510(k) regulatory clearance for the treatment of all types of recurrent brain tumors in July 2018. The data published in JNS are the initial results of a larger basket-design study that looked at the use of GammaTile in 108 patients with several kinds of recurrent brain tumors, including gliomas and brain metastasis.

Dr. Brachman continued, "We are encouraged by these data in meningiomas and look forward to sharing data on the use of this treatment in other aggressive brain tumor types in the near future."

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."

On January 3, 2019 Veracyte, Inc. ("Veracyte") (Nasdaq: VCYT) reported that it has entered into a long-term strategic collaboration with Johnson & Johnson Innovation LLC* and the Lung Cancer Initiative at Johnson & Johnson to advance the development and commercialization of novel diagnostic tests to detect lung cancer at its earliest stages, when the disease is most treatable (Press release, Veracyte, JAN 3, 2019, View Source [SID1234532382]). The collaboration will build upon foundational "field of injury" science — where genomic changes associated with lung cancer can be identified with a simple brushing of a person’s airway — to develop new interventions that can save lives.

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Under terms of the agreement, Veracyte and the Lung Cancer Initiative at Johnson & Johnson, whose mission is to prevent, intercept and cure lung cancer, will combine clinical study cohorts involving more than 5,000 patients with multiple years of clinical outcome data. Veracyte will contribute bronchial and nasal samples from its clinical trials, which are part of the company’s extensive lung cancer-focused biorepository. Veracyte will deploy its RNA whole-transcriptome sequencing platform, utilizing high-dimensional data and machine learning pipelines on the combined cohort, providing the most comprehensive genomic content available which the Lung Cancer Initiative at Johnson & Johnson will have access to for therapeutic purposes.

The collaboration is expected to accelerate two key lung cancer programs for Veracyte. First, the development of the first non-invasive nasal swab test for early lung cancer detection and, secondly, the commercialization of its Percepta classifier on the company’s RNA whole-transcriptome sequencing platform, which, as a result of this collaboration, is expected to launch in the first half of 2019.

"We are thrilled to team up with Johnson & Johnson and their Lung Cancer Initiative in the fight against lung cancer," said Bonnie Anderson, chairman and chief executive officer of Veracyte. "This strategic collaboration further advances Veracyte’s pioneering position in lung cancer diagnosis and underscores the promise of our field of injury science and approach. With the acceleration of our product pipeline, we believe this collaboration expands our addressable lung cancer diagnostic market to a $30 billion to $40 billion global opportunity."

Anderson continued, "We estimate the combined monetary and non-monetary value of the collaboration to Veracyte at more than $50 million. The monetary component consists of a $5 million upfront payment and the potential to earn up to a total of $15 million in future development and reimbursement milestone payments. The non-monetary value reflects the significant value of clinical cohorts accessible to Veracyte through the collaboration. Also as result of this agreement, we expect our biopharmaceutical service revenue for 2019 to increase by an estimated $5 million."

Lung cancer is the leading cause of cancer deaths worldwide. In the United States, lung cancer causes more than 154,000 lung cancer-related deaths each year – more than the next three most prevalent cancers combined. Because lung cancer is difficult to diagnose before it has metastasized, only 16 percent of cases are detected at an early stage, when the disease is more treatable. Lung cancer’s five-year survival rate is only 18 percent, much lower than that of other common cancers. Approximately 80 percent of lung cancer deaths are caused by smoking.

*Johnson & Johnson Services, Inc. is the legal entity party to the agreement.

Conference Call and Webcast Information

Veracyte will host a conference call and simultaneous webcast today at 8:30 a.m. ET to discuss its collaboration with Johnson & Johnson Innovation and the Lung Cancer Initiative at Johnson & Johnson. The call and webcast may be accessed as follows:

Webcast:
View Source

Dial-in number (U.S.): (855) 541-0980
International Number (Canada only): (970) 315-0440
Conference ID: 5295859
The webcast should be accessed 10 minutes prior to the conference call start time. A replay of the webcast will be available for one year following the conclusion of the live broadcast and will be accessible on the company’s website at View Source.

On January 3, 2019 Tarveda Therapeutics, Inc., a clinical stage biopharmaceutical company discovering and developing a new class of potent and selective miniature drug conjugates (Pentarins) for the treatment of patients with a wide range of solid tumors reported that Drew Fromkin, President and Chief Executive Officer, reported that it will present at the 11th Annual Biotech Showcase, occurring January 7-9, 2019 at the Hilton Union Square in San Francisco (Press release, Tarveda Therapeutics, JAN 3, 2019, View Source [SID1234532416]).

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The presentation will take place at 2:30pm Pacific Time on Monday, January 7 in Franciscan – D.

In the presentation, Mr. Fromkin will provide an overview of the Company’s two clinical programs including PEN-221, which is currently in clinical evaluation for the treatment of patients with somatostatin receptor 2 (SSTR2) positive neuroendocrine tumors and PEN-866, the first miniature drug conjugate from Tarveda’s HSP90 binding conjugate platform, which is being developed for the treatment of patients with solid tumors including but not limited to small cell lung cancer, pancreatic cancer and sarcomas.

On January 3, 2019 1ST Biotherapeutics, Inc., a preclinical-stage biotechnology company focused on neurodegenerative diseases, immuno-oncology, and orphan diseases, and twoXAR, Inc., an artificial intelligence (AI)-driven biopharmaceutical company, reported an agreement to jointly discover and develop novel, efficacious treatments to address unmet medical needs in glioblastoma multiforme ("glioblastoma") (Press release, 1ST Bio Therapeutics, JAN 3, 2019, View Source [SID1234532432]).

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Under the agreement, twoXAR will use its proprietary AI technology to identify a set of drug candidates with the potential to slow, stop, or reverse the progression of glioblastoma. twoXAR and 1ST Biotherapeutics will select candidates from this set to test in preclinical efficacy models of glioblastoma. Following identification of one or more candidates based on those evaluated, 1ST Biotherapeutics will use its team’s expertise in drug development to optimize candidates and finalize the creation of novel, efficacious treatments. Further details of the agreement were not disclosed.

Glioblastoma is the most common and lethal primary malignant brain tumor with a median survival of approximately 15 months1. It is characterized by an aggressive tumor that infiltrates various portions of the brain and is associated with rapid onset of neurological symptoms, including nausea, vomiting, severe headaches, cognitive issues, and seizures2. Current treatment options consist of surgical resection, radiation, chemotherapy, and angiogenesis inhibitors2. However, most glioblastoma patients experience disease relapse despite these aggressive therapies. It is estimated that 25,000 people will be diagnosed with glioblastoma in 20181.

"1ST Biotherapeutics is focused on efficiently building a pipeline of first-in-class therapeutic candidates with high likelihood of clinical success," said Jamie Jae Eun Kim, CEO of 1ST Biotherapeutics. "The twoXAR team has a track record of rapidly identifying testable novel treatments that can lead to first-in-class therapeutics. This collaboration is an opportunity to combine twoXAR’s AI-driven drug discovery approach and the 1ST Biotherapeutics team’s expertise in chemistry and pharmacology to discover and develop effective molecular therapeutics for glioblastoma patients."

"We are pleased to collaborate with 1ST Biotherapeutics, because we share common goals of efficiently discovering and developing novel therapeutics for diseases with high unmet medical need, such as glioblastoma," said Andrew A. Radin, Co-Founder and CEO of twoXAR. "The 1ST Biotherapeutics team’s deep medicinal chemistry and drug development experience in CNS and oncology diseases provides a strong complement to twoXAR’s data-driven discovery approach."