GBT sickle cell trial hits endpoint ahead of NDA filing

fiercebiotech | June 14, 2019

Global Blood Therapeutics has shared updated data on the sickle cell disease trial it hopes will secure FDA approval of voxelotor. The proportion of patients who had a greater than 1 g/dL increase in hemoglobin is down on GBT’s prior update but still superior to placebo, resulting in the trial hitting its primary endpoint. California-based GBT shared data on the first 154 patients to complete 24 weeks of follow-up in its phase 3 shortly after FDA green lit it to file for accelerated approval late last year. The data showed 65% of patients on the higher dose of voxelotor achieved the desired rise in hemoglobin, compared to 10% of people in the placebo cohort. Those figures came from a per-protocol analysis. GBT now has 24-week data on all 274 patients enrolled in the trial of voxelotor, a stabilizer of sickle cell hemoglobin. On a per-protocol basis, 60% of patients in the higher-dose arm had a greater than 1 g/dL increase in hemoglobin, compared to 9% in the placebo cohort. The figures slipped to 51% and 7% in the intention-to-treat (ITT) analysis.

Spotlight

May is National Stroke Awareness Month. An estimated 75% of people, especially younger individuals, would not be able to recognize the early symptoms of a stroke. This is a staggering and ominous number, given that up to 80% of strokes could be prevented in the first place. National Stroke Awareness Month was initiated to educate people on how to know when they or someone close to them is having a stroke.

Spotlight

May is National Stroke Awareness Month. An estimated 75% of people, especially younger individuals, would not be able to recognize the early symptoms of a stroke. This is a staggering and ominous number, given that up to 80% of strokes could be prevented in the first place. National Stroke Awareness Month was initiated to educate people on how to know when they or someone close to them is having a stroke.

Related News

CELL AND GENE THERAPY

NeuExcell Therapeutics and Spark Therapeutics Announce Research Collaboration Agreement to Develop a Novel Gene Therapy for Huntington's Disease

NeuExcell Therapeutics | September 13, 2021

NeuExcell Therapeutics and Spark Therapeutics, a member of the Roche Group announced a gene therapy collaboration aimed at developing a safe and effective treatment for patients suffering from Huntington's Disease (HD). Under the terms of the agreement, Spark Therapeutics will receive access to NeuExcell's proprietary neuro-regenerative gene therapy platform and capabilities. NeuExcell's research team will collaborate closely with Spark Therapeutics to advance the program. Under the Option License NeuExcell is eligible to receive upfront, license fees, R&D and Sales milestone payments up to approximately $190 million plus product royalties. Under this Agreement, Spark Therapeutics has the option to license the exclusive worldwide rights of the NeuExcell's HD program. The prevailing assumption has been that mammalian adult neurons cannot be replaced, and so therapeutic approaches for brain diseases tend to focus on slowing disease progress. NeuExcell Therapeutics may have unlocked the method for regenerating neural tissue. The company's neuroregenerative gene therapy platform is built around transcription factor-based trans-differentiation technology. The platform seeks to reprogram endogenous glial cells like astrocytes, which surround neurons and are often reactive after neurons are injured or die, into functional new neurons. While neurons cannot divide to regenerate themselves, glial cells are a renewable source for generating new neurons at the site of injury, and at the scale needed to have a meaningful therapeutic impact. NeuExcell is developing adeno-associated viruses (AAVs)-based neuroregenerative gene therapy to regenerate functional new neurons at the site of the neurodegeneration. "At Spark, we understand that in order to break down barriers for people and families affected by genetic diseases, we need to work with like-minded partners that can integrate innovative technologies with our advanced proprietary AAV vector platform," said Joseph La Barge, Chief Business Officer of Spark Therapeutics. "Using our existing expertise in gene therapy development and NeuExcell's neuro-regenerative gene therapy research and capabilities, together we can progress the potential of gene therapy for patients living with Huntington's Disease." Spark Therapeutic's advanced proprietary AAV vector platform targeted to the central nervous system offers the HD research and development (R&D) program a major advantage. At the forefront of gene therapy research for more than two decades, Spark Therapeutics has extensive knowledge and capabilities in this field that it will use to bring the HD program forward. About Huntington's Disease HD is an incurable, hereditary brain disorder caused by a single defective gene on chromosome 4. As the disease affects different parts of the brain, it impacts movement, behavior, and cognition. It becomes harder to walk, think, reason, swallow, and talk. Eventually, the person will need full-time care. The complications associated with HD are usually fatal. About NeuExcell Therapeutics. NeuExcell is a privately held early-stage gene therapy company headquartered in Pennsylvania, USA. Its mission is to improve the lives of patients suffering from neuro-degenerative diseases and CNS injuries. Based upon the scientific work of Prof. Gong Chen (Co-Founder and Chief Scientific Advisor), the Company has developed a potentially disruptive neural repair technology through in vivo astrocyte-to-neuron conversion by introducing neural transcription factors through adeno-associated virus (AAV)-based gene therapy. NeuExcell's pipeline covers major neurodegenerative diseases such as Stroke, Huntington's Disease, Amyotrophic Lateral Sclerosis (ALS), Alzheimer's Disease, Parkinson's Disease, Traumatic Brain Injury, Spinal Cord Injury, and Glioma. About Spark Therapeutics At Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases. We currently have four programs in clinical trials. At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease.

Read More

CELL AND GENE THERAPY

Astellas and Dyno Therapeutics Announce Research Collaboration to Develop Next-Generation AAV Gene Therapy Vectors for Skeletal and Cardiac Muscle

Astellas Pharma Inc. and Dyno Therapeutics, Inc. | December 02, 2021

Astellas Pharma Inc. and Dyno Therapeutics, Inc. announced an option and license agreement was signed on November 23 to develop next-generation adeno-associated virus vectors for gene therapy directed to skeletal and cardiac muscle using Dyno's CapsidMap™ platform. Dyno's CapsidMap platform represents a transformative approach applying in vivo experimental data and machine learning to create novel AAV capsids – the cell-targeting protein shells of viral vectors – designed to optimize tissue targeting and immune-evading properties, in addition to improving packaging capacity and manufacturability. Unlike traditional approaches, CapsidMap is uniquely well-suited to simultaneously optimize capsids for delivery across multiple organs, with the goal of enabling more effective whole-body treatment for many diseases. With the establishment of the Astellas Gene Therapies Center of Excellence following the 2020 acquisition of Audentes Therapeutics Inc., Astellas is a leader in genetic medicines, working alongside its world-renowned partners to build a portfolio of potentially life-changing gene therapies. This research collaboration combines Dyno's AI-powered AAV vector engineering capabilities with Astellas Gene Therapies global leadership in AAV-based pipeline assets. "Through our efforts in gene therapy and the Astellas Gene Therapies Center of Excellence, Astellas strives to identify, develop and deliver transformative gene-based therapies for patients with genetic diseases who currently have few or no effective treatment options. Our principal focus is on developing adeno-associated virus delivered therapies for the treatment of well-defined serious diseases. We are dedicated to delivering novel approaches and utilizing new technologies that can deliver transformational value for patients." Naoki Okamura, Chief Strategy Officer and Chief Financial Officer, Chief Business Officer at Astellas "We are so happy to be partnering with Astellas, a world leader in developing gene therapies. Dyno and Astellas each bring unique strengths to this collaboration, together enabling more rapid creation of new therapies for patients with great unmet need," said Dyno's CEO and co-founder Eric D. Kelsic, Ph.D. "This partnership demonstrates the flexibility of Dyno's platform to precisely design the delivery properties of gene therapy vectors towards multi-organ and disease-specific profiles, applying the scientific insights we are rapidly learning across all our partnered and internal vector engineering programs using CapsidMap." Under the terms of the agreement, Dyno will design novel AAV capsids with improved functional properties for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities, including manufacturing, of gene therapy product candidates using the novel capsids. Dyno will receive an $18 million upfront payment and be eligible to receive additional payments during the research phase of the collaboration as well as clinical and sales milestone payments and royalties for any resulting products. The aggregate potential value of future milestone and royalty payments to Dyno exceeds $235 million per product and over $1.6 billion in total value. About Astellas Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+® healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into value for patients. About Astellas Gene Therapies Astellas integrated its wholly owned subsidiary, Audentes Therapeutics, Inc. as of April 1, 2021 and established "Astellas Gene Therapies" within the organization as an Astellas Center of Excellence to develop genetic medicines with the potential to deliver transformative value for patients. Based on an innovative scientific approach and industry leading internal manufacturing capability and expertise, we are currently exploring three gene therapy modalities: gene replacement, exon skipping gene therapy, and vectorized RNA knockdown and hope to also advance additional Astellas gene therapy programs toward clinical investigation. We are based in San Francisco, with manufacturing and laboratory facilities in South San Francisco and Sanford, North Carolina. About Dyno Therapeutics Dyno Therapeutics is a pioneer in applying artificial intelligence and quantitative in vivo experiments to gene therapy. The company's proprietary CapsidMap™ platform rapidly discovers and systematically optimizes Adeno-Associated Virus capsid vectors that significantly outperform current approaches for in vivo gene delivery, thereby expanding the range of diseases treatable with gene therapies. Dyno was founded in 2018 by experienced biotech entrepreneurs and leading scientists in the fields of gene therapy and machine learning. The company is located in Cambridge, Massachusetts.

Read More

MEDICAL

Entopsis Exploring Strategic Alternatives for its OpsisDx™ Platform

Berkery, Noyes & Co. | October 08, 2021

Entopsis, Inc., the developer of an unbiased approach to diagnostics using bio-molecular signatures with a focus on oncology, announced that it has engaged Berkery Noyes & Co. as its financial advisor to assist the company in pursuing strategic alternatives for their OpsisDxTM platform. OpsisDxTM is a urine-based technology platform that shifts the focus away from conventional RNA and DNA approaches, offering a highly sensitive and specific diagnostic test for the detection of early-stage cancers. Its IP-protected platform detects urine chemical signatures via a proprietary array, achieving clinically significant results through its proprietary machine learning algorithms. Validation studies on OpsisDxTM have initially focused on detecting early-stage cancers, and the platform has now expanded to detecting multiple other diseases in a highly scalable fashion. Now is the time to launch OpsisDxTM. There's greater acceptance of multi-disease testing platforms from a scientific, regulatory and business perspective; the industry as a whole has crossed the tipping point, We are seeking an established partner who shares our vision of empowering people with actionable, low costs and accurate healthcare information with an eye towards the long-term interests of Entopsis. - Obdulio Piloto, CEO of Entopsis Inc. AboutEntopsis, Inc., Entopsis utilizes a proprietary material science screening platform to develop cost-effective and impactful products.

Read More