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.

Clinical Genomics Appoints Betsy Hanna CEO

On January 3, 2019 Clinical Genomics, a leading provider of cancer diagnostic solutions including liquid biopsy tests, reported the appointment of Betsy Hanna as president and chief executive officer (Press release, Clinical Genomics, JAN 3, 2019, View Source [SID1234532434]).

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Hanna will also join the Clinical Genomics Board of Directors.

Hanna succeeds Dr. Lawrence LaPointe as CEO, who after a decade building Clinical Genomics will transition to the new role of chief innovation officer, responsible for developing solutions to improve healthcare leading clinical trials, R&D and scientific developments. He will continue to serve as an advisor to the board.

The appointments are effective immediately.

Clinical Genomics’ board chairman Mary Padbury noted, "Both Betsy and Larry bring enormous experience in their new roles. Betsy was most recently our chief commercial officer and has a 20-year career in healthcare turning products into commercial successes. Larry, as a co-founder and an accomplished scientist, will continue to lead our innovation in this rapidly evolving field."

Ms. Hanna noted, "Clinical Genomics has been successful in science and product development. Our challenge now is to accelerate commercial growth with COLVERA, a blood diagnostic for colorectal cancer (CRC) recurrence, and InSure ONE, both now in the market. Our goal is to also expand future applications in screening."

Background: Betsy Hanna

Betsy Hanna is a healthcare and biotech leader. Most recently she was Clinical Genomics’ chief commercial officer with responsibility for developing and executing go-to-market strategies.

Ms. Hanna has two decades of healthcare industry experience including strategic marketing, U.S. and international sales management, and product launch. She joined Clinical Genomics from Origin, Inc., a clinical-stage biotech company where she was the president and chief operating officer.

Prior to Origin, Ms. Hanna had increasing responsibility during eight years at Johnson & Johnson. At J&J diagnostics, she was vice president, strategic marketing and led the global launch of two instrument platforms.

At J&J diagnostics, Hanna also served as vice president Europe, Mideast and Africa and, for J&J Vision Care, was vice president of strategic marketing and a member of the global management board.

Ms. Hanna has a BS in Chemical Engineering from the University of Illinois and an MBA from Harvard Business School.

Background: Lawrence LaPointe

Larry LaPointe, PhD, has more than two decades building healthcare companies to transform cancer testing, including most recently as co-founder of Clinical Genomics. He previously served as CTO and general manager of Enterix Inc, a cancer screening company he also co-founded.

Dr. LaPointe is a well-published research scientist and holds numerous patents in biomarker discovery, cancer detection and molecular diagnostics. He has a BS in Biology from Florida State University and a PhD in Bioinformatics from Flinders University, Australia.

Background: Clinical Genomics

Clinical Genomics is dedicated to improving patient outcomes through early detection of colorectal cancer.

COLVERA is a proprietary liquid biopsy blood test that identifies circulating tumor DNA for the detection of residual disease and early detection of colorectal cancer recurrence.
Clinical Genomics products span the spectrum of colorectal cancer testing from screening to post-treatment monitoring. InSure ONE is a one-sample fecal immunochemical test used to detect lower GI bleeding for screening in healthy adults.

Dynavax to Present at 37th Annual J.P. Morgan Healthcare Conference

On January 3, 2019 Dynavax Technologies Corporation (NASDAQ: DVAX), a fully-integrated biopharmaceutical company focused on discovering and developing novel vaccines and immuno-oncology therapeutics, reported that Eddie Gray, Chief Executive Officer, will present at the 37th Annual J.P. Morgan Healthcare Conference on Thursday, January 10, at 9 am PT (Press release, Dynavax Technologies, JAN 3, 2019, View Source [SID1234532433]).

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The presentation will be webcast and may be accessed at the "Events & Presentations" section of the Company’s website at View Source

1ST Biotherapeutics and twoXAR Form Co-Development Collaboration to Discover and Develop Treatments for Glioblastoma

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."

GT Medical Technologies Announces Publication of Data Showing Company’s Targeted Therapy is Effective for Treating Recurrences of Common Type of Brain Tumor

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."