Trial of implantable device to help deliver Spinraza soon to start
ThecaFlex DRx aims to make repeat therapy use easier, more convenient
The U.S. Food and Drug Administration (FDA) has given Alcyone Therapeutics the go-ahead to start a clinical trial testing ThecaFlex DRx, its implantable system for repeat use of Spinraza (nusinersen) to treat people with spinal muscular atrophy (SMA).
The pivotal trial, called PIERRE (NCT05866419), will be conducted in two parts. Initially, up to 10 patients, ages 3 and older, will be enrolled at sites in the U.S. — likely starting in the next month or two — and implanted with the device, then followed for 30 days. If findings are positive, another 80 patients will be enrolled at sites across the U.S. and Europe in 2024, given the implant and evaluated.
All trial participants will be followed for 12 months after receiving the implant.
Enrollment of SMA patients prescribed Spinraza likely to begin this summer
“Alcyone is excited to begin the process of evaluating the safety and performance of ThecaFlex in SMA patients being treated with Spinraza,” PJ Anand, CEO of Alcyone Therapeutics, said in a company press release.
The FDA’s clearance and pending trial start “are critical steps toward helping patients in need with a potential therapeutic delivery alternative and improved treatment experience,” Anand said.
Spinraza, the first therapy approved for SMA, works like other approved SMA treatments to boost production of the SMN protein whose deficiency causes the disease. Spinraza is administered via an injection into the spinal canal, known as an intrathecal injection.
The ThecaFlex DRx system is designed to act as a subcutaneous port and intrathecal catheter system — a catheter and fixation device — for repeat intrathecal access. In other words, the system aims to provide a ready access point that clinicians can use to access the fluid inside the spinal cord, whether to administer Spinraza or to sample that fluid through a spinal tap.
The goal is to make administering the therapy easier and more convenient for patients.
“Many of my SMA patients have complex spinal anatomy or scoliosis and may benefit from receiving Spinraza through an alternative administration approach,” said Brian Snyder, MD, PhD, a professor of orthopedic surgery at Harvard Medical School and board member of Cure SMA.
“ThecaFlex has the potential to drastically improve the treatment experience for these individuals and represents an important alternative approach to the standard lumbar puncture procedure,” Snyder added.
The PIERRE trial is being run in collaboration with Spinraza’s maker, Biogen, under an agreement between the company and Alcyone. The trial is named in memory of Pierre Boulas, a Biogen executive who played a major role in establishing this agreement.
“ThecaFlex represents the culmination of a deliberate effort to design a delivery system specifically for repeat bolus intrathecal drug delivery,” Anand said.