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

By | News | No Comments

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.”

Click here to read the entire press release.

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

By | News | No Comments

  • 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 www.clinicaltrials.gov.

Click here for the entire press release.

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

By | News | No Comments

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.”

Click here to read the entire release.

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

By | News | No Comments

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.

Click here to read the entire release.

REGENXBIO Appoints Dr. Steve Pakola as Chief Medical Officer

By | News | No Comments

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.”

Click here to read more of the press release.

GlaxoSmithKline CEO talks pharmaceutical industry and leadership

By | News | No Comments

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

By | News | No Comments

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.”

Click here to read the entire press release.

Catalent to acquire gene therapy leader Paragon Bioservices, Inc. for $1.2 billion

By | News | No Comments

Will provide new expertise and capabilities in one of the fastest-growing areas of healthcare, positioning Catalent for accelerated long-term growth

SOMERSET, N.J. and BALTIMORE, M.D. – April 15, 2019 – Catalent, Inc. (NYSE: CTLT), the leading global diversified provider of advanced delivery technologies and development solutions for drugs, biologics and consumer health products, and Paragon Bioservices, Inc., a leading viral vector development and manufacturing partner for gene therapies, today announced they have entered into a definitive agreement under which Catalent will acquire Paragon for $1.2 billion.

“Paragon’s unparalleled expertise in the rapidly growing market of gene therapy manufacturing will be a transformative addition to our business that we believe will accelerate our long-term growth. Paragon brings to Catalent a complementary capability that will fundamentally enhance our biologics business and our end-to-end integrated biopharmaceutical solutions for customers,” said John Chiminski, Catalent’s Chair & Chief Executive Officer. “We look forward to working with Paragon’s incredibly talented team and world-class customers to complete the significant ongoing investments into expanded state-of-the-art facilities and deliver revolutionary, lifesaving treatments to patients.”

Paragon’s differentiated scientific, development and manufacturing capabilities have positioned it to capitalize on strong industry tailwinds in the potentially $40 billion addressable market for gene therapy. Paragon brings specialized expertise in adeno-associated virus (AAV) vectors, the most commonly used delivery system for gene therapy, as well as unique capabilities in GMP plasmids and lentivirus vectors.

For over 25 years, Paragon has partnered with some of the world’s best biotech and pharma companies to develop and manufacture products based on transformative technologies, including AAV and other gene therapies, next-generation vaccines, oncology immunotherapies (oncolytic viruses and CAR-T cell therapies), therapeutic proteins, and other complex biologics.

Click here to read the entire press release.

Floreo Inc. Wins BioHealth Capital Region 4th Annual Crab Trap Competition

By | News | No Comments

– Winning company focused on learning solutions for those with Autism.

Other finalists presented innovative technologies related to Multiple Sclerosis, Spine Alignment, TBI and Oncolytic Immunotherapeutics 

Competition sponsored by Wilson Sonsini Goodrich & Rosati held during 2019 BioHealth Capital Region Forum –

Floreo 2019 crab trap image

Judges of the 2019 Crab Trap competition present Floreo Inc. CEO and Co-Founder, Vijay Ravindran their award 

ROCKVILLE, MARYLAND, April 9, 2018 – Floreo Inc., an early stage startup founded in 2016 which is leveraging the power of virtual reality to deliver immersive, fun, and affordable lessons for children and adults with autism spectrum disorder (ASD), was chosen from five finalists as the firm with the most commercial potential at the 4th Annual Crab Trap Competition. This year’s judges included industry leaders John Rubin of JP Morgan Private Bank, Charles J. Andres of Wilson Sonsino Goodrich & Rosati’s, Raghav Bhargava of New Enterprise Associates (NEA), Ethel Rubin of NIH/BHI, John Trainer of AstraZeneca, and Norm Marcus, MD. Of Sanderling Ventures.

The Judges were impressed with Floreo’s CEO and Co-Founder, Vijay Ravindran, presentation of his company’s technology which was inspired by his autistic son’s reaction to virtual reality. Floreo’s library of lessions are currently used in schools, therapy, and homes while providing a supervising adult the opportunity to monitor and track the learner’s progress. Floreo joins previous Crab Trap winners Galen Robotics (2018), LifeSprout (2017) and Sonavex (2016).

In addition to Floreo, the judges and 2019 BioHealth Capital Forum audience viewed presentations by  four other finalists – BeCareLinkBRAINBox SolutionsSpine Align, and Unleash Immuno Oncolytics. “Every year the Crab Trap competition has brought some of the most exciting science and technology to the BioHealth Capital Region Forum. Most people know somebody who has been impacted by challenges of Autism. The technology that Floreo has developed and is continuing to improve makes them a very worthy award winner.” said Richard Bendis, BHI President & CEO.  “The congratulate all the companies and presenters on their progress to date and wish them great success in the future.  The judges made clear this was a particularly competitive group of finalists.”

Founded by BioHealth Innovation, Inc. (BHI) and MedImmune (now AstraZeneca) in 2016, the Crab Trap Competition focuses on companies in therapeutics, diagnostics, medical devices, healthcare services, e-health, mobile health, electronic medical records, health informatics, and biohealth cyber security. The grand prize this year is $10,000 plus $10,000 worth of legal consultive services provided by Crab Trap Sponsor Wilson Sonsini.

About the BioHealth Capital Region

The BioHealth Capital Region (BHCR) consisting of Maryland, Virginia, and Washington, DC is the prominent BioHealth cluster in the Mid-Atlantic. The BHCR encompasses more than 800 life sciences companies, over 70 federal labs and world-class academic and research institutions. According to Genetic Engineering News in 2018, the BHCR is ranked 4th in Top Biopharma Clusters in the country. The BHCR brand represents collaboration among leading stakeholders with a collective goal to make the BHCR a top 3 biohealth cluster by 2023. http://www.biohealthcapital.com/

###

Media contacts:
Richard Bendis
Phone: 301-637-6439, e-mail: rbendis@biohealthinnovation.org

Join our Mailing List