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MedNess: bite-size biopharma and medtech news

5th January, 2022
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MedNess This Week
HIGHLIGHTS
 
Regulatory News

Fast Track Designation from U.S. FDA to NK Cell Therapy CYNK-001 in Development for the Treatment of AML
“The majority of patients with AML continue to have poor long-term outcomes, particularly those who suffer relapse or have measurable residual disease, necessitating development of novel therapies, including CYNK-001,” said Andrew Pecora, M.D., President of Celularity. Robert Hariri, M.D., Ph.D., Founder, Chairperson and Chief Executive Officer of Celularity added, “We believe that the unique properties of our cell source, including the ability to proliferate and maintain activity, could be the key to improving response rates and durability for patients. We are pleased to receive this fast-track designation from the FDA for AML supporting continued development of our placental-derived NK cell platform. CYNK-001 previously received orphan drug designation for malignant gliomas and fast track designation for glioblastoma multiforme.”
Trial Results
Positive topline results from the Ph 1 dose-escalating study of NanoZolid®-formulated docetaxel announced
“We are pleased to see that the study meets its primary endpoint of being safe and tolerable and that the reported adverse events are generally mild and local. The latter is most likely due to the observation of a very low systemic exposure of docetaxel which is in line with our intended local treatment regimen. In addition to meeting the primary endpoint, we also see signs of immune activation by an upregulation of key immune regulating molecules. This is indeed encouraging indicating that the NanoZolid®-formulated docetaxel could have the potential to change the immune microenvironment. We are working hard to finalize our plan forward and will very soon communicate how we intend to take the project further in clinical development.” said Nina Herne, CEO of LIDDS.
Trial/Program Status
Ph 1 Clinical Trial of NXP800 Initiated
Professor Udai Banerji, Chief Investigator of the trial and Deputy Director of Drug Development at The Institute of Cancer Research, London, and The Royal Marsden NHS Foundation Trust, said: "We have been following the pre-clinical development of NXP800, a first-in-class drug candidate discovered at the Institute of Cancer Research and now being developed by Nuvectis. We look forward to gaining an understanding on the safety, dose and schedule of administration of NXP800 to unlock its therapeutic potential in specific hard-to-treat cancers where new treatments are needed."
Dosing Milestone in Ongoing Ph 1/2 Sarcoma Trial Achieved
“Advancing to the 900 mg safety lead-in cohort in the Ewing sarcoma trial arm marks the latest milestone in what has been a productive year for our clinical programs investigating seclidemstat,” stated David Arthur, CEO of Salarius Pharmaceuticals. “During the past several months, we have almost doubled the number of active trial sites participating in the Sarcoma clinical trial. Meanwhile, enrollment continues across all patient groups, and we continue to anticipate potential data readouts in 2022.”
Collated by: Richa Tewari, PhD 
Genes and Therapy
Positive preliminary clinical safety data for CLN7
Taysha Gene Therapies and UT Southwestern (UTSW) announced positive preliminary clinical safety data for the first-generation construct in CLN7 disease. UTSW has completed the design of a next-generation construct, which is expected to further improve potency, packaging efficiency and manufacturability as well as reduce risk of immunogenicity over the first-generation construct. CLN7 disease is a rare, fatal and rapidly progressive neurodegenerative disease caused due to lysosomal dysfunction resulting from mutations in the MFSD8 gene.
Positive interim safety and biomarker data for GM1 Gangliosidosis
Passage Bio announced positive safety data for Phase 1/ 2 AAVhu68 gene therapy PBGM01, for early and late infantile GM1 Gangliosidosis (GM1) for low dose cohort of patients with late infantile GM1. GM1 is a rare, fatal lysosomal storage disease in which mutations in the GLB1 gene result in very low activity of the enzyme beta-galactosidase. PBGM01 utilizes a next-generation AAVhu68 capsid administered through the cisterna magna to deliver a functional GLB1 gene encoding beta-galactosidase to the brain and peripheral tissues of affected individuals.

Novartis to acquire Gyroscope Therapeutics
Gyroscope Therapeutics Holdings will be acquired by Novartis with an upfront payment of $800 million (£604 million) and up to $700 million (£528 million) potentially due upon the achievement of certain milestones. Novartis will gain control of the GT005 gene therapy pipeline, now is Phase 2 development, and a one-time treatment for geographic atrophy- an advanced form of dry age-related macular degeneration.
Editors' Desk
Richa Tewari, PhD
Oncology News
Shilpa Rawal, PhD
Onco I-Analyse
Arundithi Ananthanarayanan
MedNess Reviews
Debarati Banik
HealthIT
Darpan Chakraborty
Social Media Manager
IP & BioPharma News
 
Nisha Peter, PhD
Consulting Editor

 
Rinki Saha
BioPharma News
Managing Editor
Shalini Roy Choudhury
Genes and Therapy
Managing Editor
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Cover Image : iStock
Images : Twitter , Unsplash.com
Content Editors: Richa Tewari , Esha Sehanobish , Rinki Saha ,  Shilpa Rawal, PhD ,  Debarati Banik  , Divyaanka Iyer , Arundithi Ananthanarayanan and Abhinav Dey 
Concept and Design: Ananda Ghosh and Nisha Peter
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