This week: Cynata’s CYP-001 receives Orphan Drug Designation and Phase III disc degeneration trial completes enrollment.
The news highlights:
Roche Holdings Inc. (Switzerland) has acquired Inception 5, a program focused on discovering regenerative therapies for multiple sclerosis. Roche previously provided research funding to Inception 5, a spin out from the University of California, San Francisco (CA, USA), to translate research into novel targets for remyelination into a small molecule program for multiple sclerosis.
Our Inception scientists once again demonstrated their ability to effectively translate foundational academic discoveries into high-quality drug candidates. This achievement resulted from access to cutting-edge academic research, a proven team of drug hunters with domain expertise, and support from our venture capital and pharma partners,” said Peppi Prasit, CEO of Inception Sciences.
CYP-001, a mesenchymal stem cell (MSC)-based therapy for the treatment of acute graft versus host disease (GvHD), has been granted Orphan Drug Designation by the FDA. CYP-001, developed by Cynata Therapeutics (Melbourne, Australia), is manufactured using Cynata’s Cymerus platform and will now receive commercially significant incentives including the potential to receive 7 years of marketing exclusivity after FDA approval, waiver of FDA fees and US tax credits.
Speaking to RegMedNet at the World Stem Cell Summit ((22—26 January, FL, USA), Cynata CEO, Ross Macdonald, explained: “Cymerus is a patented stem cell manufacturing process that avoids the problems often associated with deriving MSCs and primary tissue sources, and has the potential to generate large-scale, low-cost MSCs based on a single blood donation from one adult donor.” [Read the full interview]
Kilian Kelly, Cynata’s Vice President, Product Development, commented, “We are delighted that the FDA has seen fit to grant Orphan Drug Designation to CYP-001, in recognition of the potential of this product to address the substantial unmet need associated with GvHD. This follows on from our successful pre-IND meeting with the FDA last year, and we look forward to building our productive relationship with the FDA over the coming years.”
Mesoblast Limited (Australia) has announced that enrollment is complete on its Phase III trial of MPC-06-ID, an allogeneic mesenchymal precursor cell (MPC) product for chronic low back pain caused by degenerative disc disease. MPC-06-ID will be administered as a single intradiscal injection and is being evaluated for pain alleviation and function improvement in patients that don’t receive adequate relief from standard therapies. The trial has enrolled 404 patients at 48 centers in the USA and Australia.
Mesoblast Chief Executive, Silviu Itescu, commented, “There is an urgent need to provide an effective treatment for patients suffering from chronic low back pain due to degenerative disc disease, a population which today accounts for 50% of prescription opioid usage. If the Phase III results demonstrate durable improvement in pain and function, MPC-06-ID has the potential to make a major difference in patients with this serious medical condition.”
For more weekly cell therapy news, read previous editions of the cell therapy weekly.