Monthly Archives

April 2019

Protenus Announces Former Chief Privacy Officer of HHS Office of the National Coordinator to Keynote 3rd Annual PANDAS Conference in Baltimore

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BALTIMOREApril 30, 2019 /PRNewswire/ — The privacy and security of patient data continues to be increasingly important as access to this data increases and threats to this sensitive information continue to grow. At the same time, several new federal health information policies will make electronic health information about individuals more widely available to individuals and their caregivers and health care professionals. Lucia Savage, Former Chief Privacy Officer of HHS Office of the National Coordinator, will provide the opening keynote for the Privacy and Analytics (PANDAS) conference, focusing on innovation that equips privacy teams to effectively navigate the shift that occurs when organizations begin to more frequently utilize consumer tools to deliver healthcare.

The keynote, “Alexa, why do we need APIs in healthcare,” will focus on how health systems can balance advancing digital health and patient engagement while ensuring the privacy and security of patient data. Savage’s presentation will also highlight best practices healthcare organizations can start using to reduce organizational risk and better protect patient privacy.

“With CMS predicting the increased use of ‘common consumer tools’ to deliver care outside of traditional brick-and-mortar environments, I hope to bring a tech-forward, HIPAA-expert perspective to hospital privacy officers to help them navigate the shift,” stated Savage.

“The volume of health data available electronically has continued to grow with the adoption of consumer-facing applications focused on improving patient care. PANDAS provides our nation’s most innovative privacy officers a forum to address the new and evolving challenges these adoptions bring to healthcare,” said Nick Culbertson, CEO of Protenus, the founding organization of PANDAS. “To that end, we are thrilled to welcome Lucia. Her expertise and experience will provide unique insight as to how the industry can make important strides in protecting patient data as healthcare continues to innovate. We are looking forward to a thought-provoking discussion.”


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United Therapeutics Announces FDA Approval Of XPS™ And Steen Solution™ Used To Perform Centralized Ex-Vivo Lung Perfusion Services

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SILVER SPRING, Md., April 29, 2019 /PRNewswire/ — United Therapeutics Corporation (Nasdaq: UTHR) today announced that its collaborator, XVIVO Perfusion, Inc., a subsidiary of XVIVO Perfusion AB (STO: XVIVO), has received Premarket Approval (PMA) from the U.S. Food and Drug Administration (FDA) for the products XPS™ and STEEN Solution™. This approval means that STEEN Solution, XPS and the accompanying single-use articles are the only medical device products that are approved in the United States for ex-vivo lung perfusion (EVLP) of initially unacceptable donated lungs at body temperature.

In June 2018, United Therapeutics and XVIVO Perfusion announced a collaboration agreement to incorporate the use of XPS™ and STEEN Solution™ into the Silver Spring, Maryland laboratory of Lung Bioengineering Inc., a subsidiary of United Therapeutics’ public benefit corporation Lung Biotechnology PBC. Since then, Lung Bioengineering has used the XPS™ technology to offer centralized EVLP to transplant centers on a fee-for-service basis, in order to increase the supply of transplantable lungs to address needless patient deaths on the transplant waitlist.

“We are proud to be XVIVO Perfusion’s partner in offering unique centralized EVLP services to expand the supply of transplantable lungs,” said Martine Rothblatt, Ph.D., Chairman and Chief Executive Officer of United Therapeutics. “We are grateful to the FDA for approving this technology that enables otherwise discarded lungs, which would be unable to be used in furtherance of their donors’ generous intent, to instead be frequently restored to transplantable and hence life-saving condition. I feel it is a miracle of biotechnology that will benefit thousands of patients who die needlessly awaiting transplant.”

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Kite to Open Cell Therapy Manufacturing Facility in Frederick County Creating Significant Job Opportunities In Maryland

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Kite Pharma, a California-based biopharmaceutical company that develops innovative cancer immunotherapies, announced plans today to open a new biologics manufacturing facility in Frederick County that will produce innovative cell therapies for people with cancer. A Gilead company, Kite will open the new facility on a 20-acre site in the Urbana Corporate Center, with plans to create a significant number of job opportunities.

Kite Pharma, A Gilead Company.  Car-T Cell Therapy for Cancer

“This new facility in Urbana builds on our substantial technical capabilities and rapid progress in making personalized chimeric antigen receptor T and TCR cell therapies for people with cancer. As we advance our industry-leading cell therapy pipeline and seek to help a growing number of people with cancer, expanding and investing in our manufacturing capabilities is essential,” said Tim Moore, executive vice president of technical operations at Kite. “With the Urbana site, we will have the opportunity to build and design the facility tailored to our own innovative processes and with state-of-the-art features that will enable us to meet the future needs for cell therapies.”

With this new facility, Kite will significantly expand its ability to manufacture commercially available and investigational cell therapies. The new Frederick facility will become part of Kite’s growing commercial manufacturing network that includes sites in California and the Netherlands.

Click here to read more via BioBuzz.

Autolus Therapeutics Receives FDA Orphan Drug Designation for AUTO3 for Treatment of Acute Lymphoblastic Leukemia

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LONDON, April 23, 2019 (GLOBE NEWSWIRE) — Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies for the treatment of cancer, announced that the United States Food and Drug Administration (FDA) has granted orphan drug designation to autologous enriched T-cells genetically modified with a retroviral vector to express two chimeric antigen receptors targeting CD19 and CD22 (AUTO3) for the treatment of acute lymphoblastic leukemia (ALL).

According to the National Institute of Health’s National Cancer Institute, in the United States, there will be an estimated 5,930 new cases of ALL and an estimated 1,500 related deaths in 2019. Patients are predominantly children; approximately 60% of cases occur at age < 20 years.  ALL occurs when the bone marrow makes too many immature lymphocytes, which are a type of white blood cell. Despite a high rate of response to induction chemotherapy, only 30–40% of adult patients with ALL will achieve long-term remission. Similarly, pediatric patients typically respond well to first-line treatment (combination chemotherapy) but 10 to 20% of total patients relapse with chemotherapy-resistant disease, leading to a significant unmet need in pediatric patients with high-risk relapsed or refractory ALL.

“We are pleased to receive orphan drug designation for AUTO3 for acute lymphoblastic leukemia,” said Dr. Christian Itin, chairman and chief executive officer of Autolus. “Earlier this year, we presented encouraging updated data from the AMELIA phase 1/2 trial of AUTO3 in pediatric ALL patients.  We believe that AUTO3 has the potential to be a best in class therapy in pediatric ALL by addressing antigen escape, a common cause of relapse in these patients.  AUTO3 may also provide an improved safety profile over currently marketed CAR T therapies with low levels of severe CRS and neurotoxicity observed in clinical studies.”

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GlycoMimetics Announces Enrollment of First Patient in NCI-Sponsored Phase 3 Trial of Uproleselan in AML

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  • Evaluating previously untreated newly diagnosed adults with acute myeloid leukemia (AML) who are fit for intensive chemotherapy
  • Second initiation among three late-stage uproleselan clinical trials

ROCKVILLE, Md.–(BUSINESS WIRE)–Apr. 23, 2019– GlycoMimetics, Inc. (NASDAQ: GLYC) announced today dosing of the first patient in a Phase 3 clinical trial being conducted under the auspices of a Cooperative Research and Development Agreement (CRADA) between GlycoMimetics and the National Cancer Institute (NCI), part of the National Institutes of Health. The second in a series of trials designed to evaluate uproleselan across the continuum of care in AML, this NCI-sponsored study is evaluating the addition of uproleselan to a standard cytarabine/daunorubicin regimen (7&3) in older adults with previously untreated AML who are suitable for intensive chemotherapy. A third trial, to be conducted by the European HOVON consortium, is expected to initiate later this year.

“The initiation of the NCI-sponsored trial is an important milestone for our uproleselan program, a drug candidate with the potential to address significant unmet treatment needs across the spectrum of AML,” noted Helen Thackray, M.D., FAAP, GlycoMimetics Senior Vice President, Clinical Development, and Chief Medical Officer. “Along with our global pivotal Phase 3 clinical trial testing the investigational drug in patients with relapsed/refractory acute myeloid leukemia, this trial will facilitate our growing understanding of how uproleselan may fit into the continuum of care for individuals living with AML.”

GlycoMimetics is collaborating with both the NCI and the Alliance for Clinical Trials in Oncology to conduct the trial, which is led by Geoffrey Uy, M.D., Associate Professor of Medicine, Bone Marrow Transplantation and Leukemia, Washington University School of Medicine in St. Louis. The primary endpoint will be overall survival, with a planned interim analysis based on event-free survival (EFS) after the first 250 patients have been enrolled in the study. More information on this clinical trial can be found at

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GlycoMimetics Announces Publication of Nature Cell Biology Paper Supporting Recently Announced Clinical Trial of GMI-1359

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Data shows that E-selectin is key to tumor growth and metastasis to bone and provides further support for upcoming clinical trial in patients with metastatic breast cancer

ROCKVILLE, Md.–(BUSINESS WIRE)–Apr 22, 2019–GlycoMimetics, Inc. (NASDAQ: GLYC) today announced the publication of a paper in Nature Cell Biology that describes how tumor cells engage specific stromal components, most notably E-selectin, for propagation and outgrowth. 1 The paper provides further scientific support for the clinical trial in breast cancer patients with bone metastasis that was recently announced by GlycoMimetics.

Specifically, Esposito et. al. identify an E-selectin ligand expressed on tumor cells that is necessary for inducing mesenchymal-epithelial transition (MET) and that drives metastatic progression within the bone marrow microenvironment. Of note, in preclinical animal models of human breast cancer, inhibition of E-selectin with GlycoMimetics’ compound uproleselan (GMI-1271) prevented bone metastases progression and significantly attenuated bone metastases-associated bone degradation, resulting in a significant survival advantage in treated tumor-bearing mice. Previously published work also demonstrates a complimentary role for CXCR4. Together these observations support the testing of GMI-1359, GlycoMimetics’ dual-function antagonist, which targets both mechanisms.

“The scientific rationale for potential uses of GMI-1359 in oncology indications continues to build,” said John L. Magnani, PhD, Chief Scientific Officer of GlycoMimetics. “This most recent paper contributes additional understanding to the critical role of E-selectin and to the potential uses of compounds that target this mechanism in cancer, in particular in cancers that metastasize to bone.”

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Viela Bio Receives U.S. FDA Breakthrough Therapy Designation for Inebilizumab for Treatment of Neuromyelitis Optica Spectrum Disorder

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Viela Bio (PRNewsfoto/Viela Bio)

Gaithersburg, MD—April 18, 2019 – Viela Bio today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for the Company’s anti-CD19 monoclonal antibody inebilizumab, an investigational monotherapy for neuromyelitis optica spectrum disorder (NMOSD). NMOSD is a rare, life-threatening autoimmune disease affecting the central nervous system.

“The Breakthrough Therapy Designation for inebilizumab is based on results from the largest monotherapy study ever conducted in NMOSD,” said Jorn Drappa, M.D., Ph.D., Chief Medical Officer and Head of Research & Development at Viela Bio. “Inebilizumab is a humanized monoclonal antibody designed to bind with high affinity to CD19 and deplete a broad range of B cells, including autoantibody-secreting plasmablasts and CD19-expressing plasma cells. We continue our efforts to bring inebilizumab to patients suffering from this devastating disease for which there are currently no approved medicines.”

Breakthrough Therapy Designation is designed to expedite the development and regulatory review of medicines intended to treat a serious condition that have shown encouraging early clinical results which may demonstrate substantial improvement on a clinically significant endpoint over available medicines. The designation for inebilizumab is based on data from the pivotal N-MOmentum study evaluating inebilizumab as monotherapy.

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REGENXBIO Appoints Dr. Steve Pakola as Chief Medical Officer

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ROCKVILLE, Md.April 17, 2019 /PRNewswire/ — REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV®Technology Platform, today announced the appointment of Steve Pakola, M.D., as its Senior Vice President and Chief Medical Officer. Dr. Pakola will report to Kenneth T. Mills, REGENXBIO’s President and Chief Executive Officer.

Dr. Pakola joins REGENXBIO from Aerpio Pharmaceuticals, Inc., where he was Chief Medical Officer. He has held key leadership roles in preclinical and clinical development, regulatory affairs and medical affairs. While Dr. Pakola’s therapeutic area experience encompasses multiple indications, his predominant focus has been the development of treatments for retinal disorders, including clinical development experience in diabetic retinopathy, diabetic macular edema and age-related macular degeneration (AMD). He was the lead inventor and program lead for the Jetrea®(ocriplasmin) program, from inception in 2002 through the therapy’s United States and European regulatory submissions in 2012.

“In this role, Steve will leverage his rich industry experience and expertise in the discovery and development of novel treatments for retinal disease,” said Mr. Mills. “Steve’s background as an innovator in drug development is particularly relevant as we leverage our breakthrough science to advance five current proprietary therapeutic programs that focus on retinal, neurodegenerative and metabolic diseases. We are excited to welcome Steve to the team.”

“I am thrilled to join REGENXBIO at this time of exciting momentum for the company as it advances the development of gene therapies,” said Dr. Pakola. “REGENXBIO’s NAV Technology Platform has the potential to transform the course of treatment for patients with rare diseases as well as in broader applications such as wet AMD. I look forward to working with my colleagues, investigators and the regulatory authorities to bring new and innovative treatments to patients.”

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GlaxoSmithKline CEO talks pharmaceutical industry and leadership

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Emma Walmsley, the CEO of GlaxoSmithKline, discussed the pharmaceutical industry and the role of leadership at the Stanford Medicine Dean’s Lecture on Tuesday. GlaxoSmithKline, commonly known as GSK, is a British-based multinational pharmaceutical company — one of the world’s largest.

Walmsley became CEO of GlaxoSmithKline in 2017, marking her as the first woman to run a major pharmaceutical company. Since taking the helm, Walmsley has instituted significant change in GlaxoSmithKline’s approach to research and development. These include a greater focus on the immune system, further investments into advanced technologies and increased utilization of genetic information and techniques.

“There has never been a more exciting time in terms of the advances in biology and technology, and how the two of them coming together could be absolutely thrilling and impactful to patients around the world,” Walmsley said.

Throughout the talk, Walmsley highlighted GSK’s new business strategies, acquisitions and trajectory, as well as the state of the pharmaceutical industry. Her educational background in Classics and her professional background in consumer products, she explained, is often untraditional in the scientific sector, yet it offers her a unique perspective as chief executive.

Walmsley also discussed the complexities involved in her role as head of a large pharmaceutical company. Throughout her tenure as CEO, she has often had to weigh tough business decisions, putting aside her personal biases and fears in favor of what is directly beneficial for the company.

Read more via the Staford Daily.

Emergent BioSolutions Announces Interim Results From Phase 2 Study Evaluating CHIKV-VLP, Chikungunya Virus Vaccine Candidate

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GAITHERSBURG, Md., April 16, 2019 (GLOBE NEWSWIRE) — Emergent BioSolutions Inc. (NYSE:EBS) today announced results from the interim analysis of its Phase 2 clinical study evaluating the safety and immunogenicity of the company’s chikungunya virus virus-like particle (CHIKV-VLP) vaccine candidate across a series of dosing regimens. The interim analysis has shown that with a single dose administered, up to 98% of study participants produced a neutralizing antibody response against the chikungunya virus (CHIKV) by Day 7. Further, the immune response was shown to be persistent through the six-month visit, including in the one-dose regimen.

“Emergent is highly encouraged about our vaccine candidate as the interim data suggest that a single dose of the vaccine was able to generate a positive immune response, which persisted through the study participants’ visits at six months,” said Abbey Jenkins, senior vice president and vaccines and anti-infectives business unit head. “Chikungunya virus infection represents a significant unmet medical need – having no vaccine or treatment available – despite its emergence as a global threat because of the highly debilitating nature of the associated disease and unprecedented magnitude of its spread.1 We look forward to completing the data set analysis and finalizing our development plan, which could allow for initiation of a pivotal trial next year.”

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