An Open Label, Single Cohort Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered Via the ThecaFlex DRx™ System (PIERRE-PK)

In this PIERRE-PK study, researchers will learn how the body processes nusinersen when it is given through the ThecaFlex DRx™ System, compared to when nusinersen is given by lumbar puncture (LP). The ThecaFlex DRx system is an investigational implantable medical device developed by Alcyone Therapeutics, Inc. It consists of a catheter, which is a flexible tube, connected to a port which is placed under the skin. Alcyone Therapeutics, Inc. has an ongoing study called PIERRE to test the ThecaFlex DRx system. Participants with spinal muscular atrophy (SMA) in the PIERRE study may be enrolled in the PIERRE-PK study.
The main objective of the PIERRE-PK study is to learn how the body processes nusinersen when given by the ThecaFlex DRx system compared to a lumbar puncture. The main questions researchers want to answer are:
* What is the highest amount of nusinersen found in the blood after dosing?
* How much nusinersen is found in the blood over the first 24 hours after dosing?
The PIERRE-PK study will be done as follows:
* Participants will be screened to check if they can join the study. The screening period will be up to 30 days for this study and may overlap with the PIERRE study.
* Participants will receive a dose of nusinersen by lumbar puncture.
* The ThecaFlex DRx system will be implanted after the lumbar puncture, as part of the PIERRE study.
* Participants will receive a dose of nusinersen by the ThecaFlex DRx system, as part of the PIERRE study.
* Researchers will take blood samples before and after each dose. The last blood sample will be taken 24 hours after the dose.
* The total study duration for each participant in the PIERRE-PK study will be approximately 5 months. This period will overlap with the participant's first 5 months in the PIERRE study.

开始日期2025-01-16

申办/合作机构

Biogen, Inc.

[+1]

NCT05866419

/ RecruitingN/A

Safety and Performance of the ThecaFlex DRx™ System Port and Catheter for Chronic Intrathecal Access, Cerebrospinal Fluid (CSF) Aspiration, and DElivery of Nusinersen in Spinal Muscular Atrophy (SMA) Patients Resistant to Lumbar PunctuRE Trial (PIERRE)

The primary objective of the clinical investigation is to demonstrate successful clinical use of the ThecaFlex DRx™ System in delivering nusinersen in subjects with spinal muscular atrophy (SMA).
All enrolled subjects will undergo implantation of the investigational device (ThecaFlex DRx™ System) and will be followed for 12 months after receiving the implant. The 12-month data will be used to assess the primary endpoint support a Pre-Market Approval (PMA) application.

开始日期2023-11-27

申办/合作机构

Alcyone Therapeutics, Inc.

[+1]

NCT03770572

/ Active, not recruiting临床1/2期

Phase I/IIa Gene Transfer Clinical Trial for Juvenile Neuronal Ceroid Lipofuscinosis, Delivering the CLN3 Gene by Self-Complementary AAV9

This is a phase 1/2, open-label, single dose, dose-escalation clinical trial to evaluate the safety and efficacy of CLN-301 (previous NCH Code: scAAV9.P546.CLN3) delivered intrathecally into the lumbar spinal cord region of subjects with CLN3 Batten disease.

开始日期2018-11-13

申办/合作机构

Alcyone Therapeutics, Inc.

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项与 Alcyone Therapeutics, Inc. 相关的文献（医药）

2025-07-01·JOURNAL OF APPLIED PHYSIOLOGY

Normal variation in brain volumetrics, CSF dynamics, and ocular structures from magnetic resonance images of healthy participants over two years

Article

作者: Kramer, Larry A. ; Carter, Katrina J. ; Laurie, Steven S. ; Warthen, Katherine G. ; Young, Millennia ; Macias, Brandon R. ; Feng, Ching Mei ; Sater, Stuart H. ; Hasan, Khader M. ; Martin, Bryn A.

We report estimated thresholds of normal variability from methodological and physiological sources in brain and ocular outcomes in healthy participants. Outcomes that exceed these thresholds have less than a 5% probability of occurring due to these sources of variation. One of the present findings suggests that about 50% of the spaceflight-induced increase in lateral ventricular volume exceeds contributions of expected sources of measurement variability.

2014-05-01·Journal of neuroscience methods4区 · 医学

In vivo performance of a microfabricated catheter for intraparenchymal delivery

4区 · 医学

Article

作者: Olbricht, William L ; Anand, P J ; Mata, Jaime ; Fleisher, Adam S ; Brady, Martin L ; Singh, Deep ; Raghavan, Raghu ; Broaddus, William C

BACKGROUND:

Convection-enhanced delivery (CED) is currently the only effective clinical technique to deliver biological therapeutic agents that would otherwise not cross the blood-brain barrier. Despite the promise of CED, several technical problems have limited its effectiveness.

NEW METHOD:

Brain infusions into a large mammal (pig) were performed with a catheter that was fabricated using micro-electro-mechanical systems (MEMS) technology (Olbricht et al., 2010). The performance of the catheter was evaluated for infusions at increasing infusion rates. Magnetic resonance (MR) images were acquired in real time to examine the distribution of infused tracers in the parenchyma.

RESULTS:

Both backflow and the distribution of CED of infusates into a variety of cytoarchitectures in porcine brain were quantified. Concentration profiles were determined for several MR contrast reagents as well as a fluorescent dye that are the sizes of small molecules, therapeutic proteins and an adeno-associated virus (AAV). The reagents can serve as surrogates for assessing the convective distribution of active molecules. Infusion rates up to 20μL/min were attained without evidence of backflow along the catheter.

COMPARISON WITH EXISTING METHODS:

The device performed well in terms of both backflow and infusion, superior to that of many studies reported in the literature on other catheters. All infused molecules had comparable ratios of distribution to infusion volumes.

CONCLUSIONS:

The catheter described in this report appears able to target tissue structures with precision, deliver therapeutics at high infusion rates, and resist backflow that can compromise the efficacy of CED therapy. The technology allows development of "smart" catheters for future applications.

Frontiers in neuroimaging

Investigation of Human Intrathecal Solute Transport Dynamics Using a Novel in vitro Cerebrospinal Fluid System Analog

Article

作者: Burla, Goutham Kumar Reddy ; Martin, Bryn A ; Bowen, Mayumi ; Shrestha, Dev ; Seiner, Akari ; Horvath, Joshua D

Background:

Understanding the relationship between cerebrospinal fluid (CSF) dynamics and intrathecal drug delivery (ITDD) injection parameters is essential to improve treatment of central nervous system (CNS) disorders.

Methods:

An anatomically detailed in vitro model of the complete CSF system was constructed. Patient-specific cardiac- and respiratory-induced CSF oscillations were input to the model in the subarachnoid space and within the ventricles. CSF production was input at the lateral ventricles and CSF absorption at the superior sagittal sinus. A model small molecule simulated drug product containing fluorescein was imaged within the system over a period of 3-h post-lumbar ITDD injections and used to quantify the impact of (a) bolus injection volume and rate, (b) post-injection flush volume, rate, and timing, (c) injection location, and (d) type of injection device. For each experiment, neuraxial distribution of fluorescein in terms of spatial temporal concentration, area-under-the-curve (AUC), and percent of injected dose (%ID) to the brain was quantified at a time point 3-h post-injection.

Results:

For all experiments conducted with ITDD administration in the lumbar spine, %ID to the brain did not exceed 11.6% at a time point 3-h post-injection. Addition of a 12 mL flush slightly increased solute transport to the brain up to +3.9%ID compared to without a flush (p < 0.01). Implantation of a lumbar catheter with the tip at an equivalent location to the lumbar placed needle, but with rostral tip orientation, resulted in a small improvement of 1.5%ID to the brain (p < 0.05). An increase of bolus volume from 5 to 20 mL improved solute transport to the brain from 5.0 to 6.3%ID, but this improvement was not statistically significant. Increasing bolus injection rate from 5 to 13.3 mL/min lacked improvement of solute transport to the brain, with a value of 6.3 compared to 5.7%ID.

Conclusion:

The in vitro modeling approach allowed precisely controlled and repeatable parametric investigation of ITDD injection protocols and devices. In combination, the results predict that parametric changes in lumbar spine ITDD-injection related parameters and devices can alter %ID to the brain and be tuned to optimize therapeutic benefit to CNS targets.

项与 Alcyone Therapeutics, Inc. 相关的新闻（医药）

2025-10-01

·BioPharmaDive

A new biotech aims to get RNA drugs into the brain

The latest biotechnology company hoping to get genetic medicines into the brain has now launched, with backing from more than half a dozen investment firms.Aerska, a Dublin-based startup whose name comes from a Gaelic proverb, on Wednesday announced it has raised $21 million in seed funding.Age1, Backed VC and Speedinvest co-led the funding round, while other early-stage investors like Blueyard, Norrsken VC and Ada Ventures participated.Aerskas research focuses on RNA interference, a drugmaking technique that treats illnesses by blocking the genetic information the body uses to create harmful or undesirable proteins. Specifically, Aerska is trying to silence genes that drive brain diseases. Its an ambitious goal. So far, RNA interference technology has been most successful when directed at disease targets in the liver.The brain, meanwhile, is encased in a protective shield that keeps out larger molecules, including genetic medicines.Over the past decade or so, a new class of biotechs has spawned boasting technologies designed to surmount that tricky blood-brain barrier.“One, Aliada Therapeutics, formed in 2021 with seed funding from RA Capital Management and the venture arm of Johnson & Johnson. Built around a J&J technology, Aliada operated for about three years before AbbVie swooped in and bought it for $1.4 billion.AbbVies acquisition added to an already hot area of dealmaking.Eisai, the Japan-based pharmaceutical firm, in April of last year agreed to work with longtime partner BioArctic on a preclinical drug made with the latters patented BrainTransporter platform. Then, a few months later, Roche licensed a technology from Sangamo Therapeutics.Even more recently, GSK wagered billions of dollars on barrier-crossing tools from ABL Bio while Biogen purchased Alcyone Therapeutics for a technology meant to conveniently send RNA drugs known as antisense oligonucleotides to the brainThose deals followed other, similar pacts between Biogen and Denali Therapeutics and Eli Lilly and Entos Pharmaceuticals.Neurological diseases remain one of the greatest challenges in medicine, with limited options to alter the course of disease, said Jack OMeara, Aerskas CEO and one of its three co-founders, in a statement. The companys aim, OMeara added, is to enable precise, durable gene silencing in the CNS or central nervous system by integrating brain shuttles with RNA therapeutics.Another Aerska co-founder,Stuart Milstein, helped lead brain delivery efforts at Alnylam Pharmaceuticals, a pioneering company in the RNA interference field. The biotechs research head, Mike Perkinton, previously led discovery work and served as an executive director in AstraZenecas neuroscience division.Aerskas antibody-oligo conjugateplatform is meant to deliver RNA interfering medicines throughout the brain, where they can be taken in by neurons and subdue certain genes.Additionally, the company is investing in data science to advance a precision medicine strategy in neurology, with an initial focal point being genetic forms of Alzheimers disease and Parkinsons.Delivery across the blood-brain barrier remains the bottleneck for genetic medicines in neurology, said Alex Brunicki, a partner at Backed VC and an Aerska board member. Aerskas platform therefore positions the company to be at the forefront of CNS therapeutics. '

并购寡核苷酸

2025-09-23

·PMLiVE

Biogen expands portfolio in deal with Alcyone Therapeutics

Biogen is set to acquire US-based Alcyone Therapeutics. Biogen has an existing partnership with Alcyone, and both companies are developing a device that has the potential to improve how medications are given to patients with neurological disorders. The device, ThecaFlex DRx, is an implantable subcutaneous port and catheter device and it is being investigated for the intrathecal delivery of antisense oligonucleotides (ASOs). The device is designed to provide an alternative to repeat lumbar punctures in chronic intrathecal administration of medicines, which could improve patient experience and accessibility for people with neurologic disorders. The ThecaFlex DRx System is initially being evaluated for use with SPINRAZA (nusinersen) and Biogen will drive end-to-end development, manufacturing and commercialisation of the device. SPINRAZA is use to treat paediatric and adult patients with spinal muscular atrophy (SMA). Biogen plans to introduce the new drug delivery system for SPINRAZA in early 2028, contingent upon the successful completion of clinical trials and regulatory approval. Nicole Murphy, Head of Pharmaceutical Operations and Technology at Biogen said: “We believe the acquisition of Alcyone Therapeutics offers a strategic opportunity to both expand the company’s capabilities and enhance the value proposition of our medicines by offering a meaningful, patient-centred solution.” PJ Anand, Founder, President, and Chief Executive Officer of Alcyone Therapeutics commented: “With ThecaFlex DRx, following our productive partnership with Biogen, we now have the opportunity to further deliver what could be the first truly patient-centred, chronic intrathecal delivery option for these important therapies.” Biogen will pay US-based Alcyone Therapeutics an upfront cash payment of $85M plus certain milestones payable related to the development and regulatory approval of ThecaFlex DRx with nusinersen and additional pipeline products, securing all rights to ThecaFlex Drx. Once the deal is concluded, Alcyone employees will become part of Biogen’s product delivery solutions team.

寡核苷酸并购

2025-09-19

·Firstwordpharma

Biogen snaps up Alcyone and its intrathecal delivery platform for $85M upfront

Biogen entered into a definitive agreement to acquire Massachusetts-based Alcyone Therapeutics, securing full rights to the latter’s investigational intrathecal drug delivery system.The buyout centres on ThecaFlex DRx, an implantable subcutaneous port and catheter device being evaluated for intrathecal delivery of antisense oligonucleotides (ASOs). The system is designed to provide an alternative to repeated lumbar punctures in chronic intrathecal medicine administration, thereby enhancing patient experience and accessibility.ThecaFlex DRx is currently being assessed with Biogen's SMN2-directed ASO Spinraza (nusinersen) in patients with spinal muscular atrophy as part of an earlier collaboration between the companies in 2023. The drugmaker plans to introduce the new delivery system for Spinraza in early 2028, subject to trial completion and regulatory approval.“Following our productive partnership with Biogen, we now have the opportunity to further deliver what could be the first truly patient-centred, chronic intrathecal delivery option for these important therapies,” said PJ Anand, chief executive of Alcyone.Under terms of the transaction announced Thursday, Alcyone will receive an upfront payment of $85 million plus potential milestone payments tied to the development and approval of ThecaFlex DRx with Spinraza and other pipeline assets. Additionally, Alcyone's remaining assets, including its Falcon precision intra-cerebrospinal fluid drug transport modelling, and intraparenchymal delivery technologies, will be divested into a new independent entity — Neela Therapeutics.

并购寡核苷酸临床研究

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