• Alcyone’s ThecaFlex is an implantable medical device under investigation for routine subcutaneous administration of therapeutics to the cerebrospinal fluid (CSF).
• The first stage of the pivotal IDE PIERRE trial has been completed.
• 10 spinal muscular atrophy (SMA) patients have undergone ThecaFlex implantation and have received nusinersen through the device.
•  No device-related adverse events, including infections, were observed in the 30 days after implantation.
• FDA has agreed to an additional 80-SMA patient, 30-center, enrollment after review of the clinical data by Data Monitoring Safety Committee of first 10 SMA patients implanted.

Lowell, Mass., July 31, 2024 / PRNewswire / Alcyone Therapeutics Inc. (Alcyone), a clinical-stage biotechnology company pioneering next-generation precision genetic therapies and precision delivery solutions of therapies for complex neurological conditions, announced today that the U.S. Food and Drug Administration (FDA) has provided approval to continue enrollment of the PIERRE study (https://clinicaltrials.gov/ct2/show/NCT05866419) to evaluate the safety and effectiveness of the ThecaFlex DRx™ subcutaneous port and intrathecal catheter system for chronic intrathecal access, CSF aspiration, and delivery of SPINRAZA® (nusinersen) in SMA patients as an alternative to repeat lumbar puncture (LP).

As previously announced, the PIERRE IDE trial was designed to enroll up to 90 SMA patients with a minimum age of 3 years old and be conducted in two stages. The first stage, consisting of 10 SMA patients enrolled, implanted, infused with nusinersen and followed for 30 days, is complete. During the first stage of the trial, all attempts to aspirate and infuse through the ThecaFlex port were successful, and no infection or device-related adverse events were observed. Based on a review of the data from the first stage, the FDA provided approval to continue enrollment of an additional 80 SMA patients in up to 30 centers across the U.S. and Europe.

Based on experience among the first 10 SMA patients enrolled in the trial, the observed duration for implantation of ThecaFlex was between one and two hours, depending on spinal complexity of the patient, with most SMA patients discharged from the hospital within 24 hours of implantation. Following implantation, the infusion procedure was typically performed in a non-specialized setting (i.e., a standard exam room) with an observed infusion procedure duration of less than 30 minutes with the actual nusinersen subcutaneous infusion time taking less than 10 minutes.

“As we approach the next stage of the PIERRE IDE study, we will continue to evaluate the safety and efficacy of the ThecaFlex system to help patients with spinal muscular atrophy receive the SPINRAZA® (nusinersen) therapy,” says Dr. Scellig Stone, M.D., Ph.D., FRCSC the Boston Children’s Hospital primary investigator for the PIERRE study. “The initial data and patient feedback are optimistic and a key first step toward developing a less invasive, more accessible procedure that may improve the overall dosing experience for SMA patients and may help optimize hospital resources.”

“We were pleased to receive the recommendation from the independent data monitoring safety committee to continue enrollment without any changes to the PIERRE study protocol as well as the FDA’s subsequent approval to continue enrollment,” said Dr. Kathrin Meyer, Ph.D., Alcyone’s Chief Scientific Officer and Head of Research & Development. “This represents a tangible step toward our goal of substantially improving treatment experience for patients requiring repeat intrathecal delivery of medications for the treatment of neurological disorders.”

Potential clinical benefits of the ThecaFlex DRx System are currently being evaluated in the PIERRE study and may include: (a) enabling routine subcutaneous administration of ASO (antisense oligonucleotide) therapies to the cerebrospinal fluid, and (b) reducing the need for anesthesia and radiation exposure compared to repeat LP for SPINRAZA infusion in SMA subjects.

“For those who currently require anesthesia and radiographic guidance for delivery of nusinersen, which exposes them to radiation, ThecaFlex could shift administration from a specialized visit to an exam room setting,” said Dr. Michael Muhonen, M.D. at the Children’s Hospital of Orange County in California. “I look forward to the continued enrollment of the study and future learnings from the patient therapy administration experience with this innovative device.”

Beyond this trial, if ThecaFlex is approved for chronic bolus intrathecal administration of drugs without indication restrictions, it may increase accessibility for people suffering from other neurological disorders that need repeat intrathecal drug delivery. It has the potential to be the first implantable device designed to enable routine subcutaneous access for the delivery of ASO therapies.

The PIERRE pivotal IDE trial is part of a collaboration between Biogen Inc. (Nasdaq: BIIB) and Alcyone previously announced in January 2023. Additional details can be found at https://clinicaltrials.gov/ct2/show/NCT05866419.

About The ThecaFlex DRx™ System
The ThecaFlex DRx System (ThecaFlex), a technology within Alcyone’s Falcon™ Delivery Platform, is an implantable intrathecal (IT) catheter, catheter fixation device, and subcutaneous port system designed to provide access to the cerebrospinal fluid (CSF) for the infusion of therapies by IT bolus administration. Lumbar puncture (LP), commonly known as a spinal tap, is the current standard of care approach to delivering therapeutics into the CSF. ThecaFlex is designed to be an alternative to LP, especially for people with a challenging spinal anatomy or those requiring multiple anesthesia and radiation exposures for repeat LPs, or for patients for whom the treating physician determines implantation of ThecaFlex is otherwise in the patient’s best interests. ThecaFlex has received a CE Mark in Europe and IDE (Investigational Device Exemption) from FDA to conduct a clinical investigation but has not yet been approved for commercial use by FDA. In addition, ThecaFlex has received Breakthrough Device Designation from FDA. For more information, visit www.alcyonetx.com.

About Alcyone Therapeutics
Alcyone Therapeutics is a clinical-stage biotechnology company pioneering next-generation precision gene-based therapies for complex neurological conditions. The company integrates innovation in neuroscience, precision dosing platforms, and manufacturing capabilities to deliver transformative therapies to patients. Alcyone leverages the synergy between FalconTM, the Company’s proprietary intrathecal precision dosing and biodistribution platform that incorporates deep knowledge of cerebrospinal fluid (CSF) dynamics, computational modeling, bioengineering, and novel gene-based therapeutics platforms developed at the Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital. This comprehensive approach allows for the optimization of central nervous system (CNS) dosing and delivery to better target the pathophysiology and anatomy specific to various neurological diseases. Alcyone’s lead programs utilize X chromosome reactivation for X-linked disorders and targets the treatment of Rett syndrome. For more information, visit www.alcyonetx.com.

About SPINRAZA® (nusinersen)
SPINRAZA is approved to treat infants, children, and adults with spinal muscular atrophy (SMA) and is approved in more than 60 countries. As a foundation of care in SMA, more than 13,000 individuals have been treated with SPINRAZA worldwide.¹
SPINRAZA is an antisense oligonucleotide (ASO) that targets an underlying cause of motor neuron loss in SMA by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.² It is administered directly into the central nervous system, where motor neurons reside, to deliver treatment where the disease starts.²

SPINRAZA has demonstrated sustained efficacy across ages and SMA types with a well-established safety profile based on data in patients treated for up to eight years, combined with unsurpassed real-world experience.³ The nusinersen clinical development program encompasses more than 10 clinical studies, which have included more than 460 individuals across a broad spectrum of patient populations, including two randomized controlled studies (ENDEAR and CHERISH). SHINE and ongoing NURTURE open-label extension studies are evaluating the long-term impact of SPINRAZA. The most common adverse events observed in clinical studies were respiratory infection, fever, constipation, headache, vomiting and back pain. Laboratory tests can monitor for renal toxicity and coagulation abnormalities, including acute severe low platelet counts, which have been observed after administration of some ASOs.

Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS). Please click here for Important Safety Information and full Prescribing Information for SPINRAZA in the U.S., or visit your respective country’s product website.

References:

1. Based on commercial patients, early access patients, and clinical trial participants through March 31, 2022.
2. SPINRAZA U.S. Prescribing Information available at: https://www.spinraza.com/content/dam/commercial/specialty/spinraza/caregiver/en_us/pdf/spinraza-prescribing-information.pdf. Accessed: October 2022.
3. Core Data sheet, Version 13, October 2021. SPINRAZA. Biogen Inc, Cambridge, MA.

 

Media & Investor Contact:
Chris Coletta
Alcyone Therapeutics
(978) 709-1946
ir@alcyonetx.com

–Key Additions to Executive Team as Alcyone Continues Clinical Progress–

LOWELL, Mass., May 08, 2024 / PRNewswire / – Alcyone Therapeutics, a clinical-stage biotechnology company pioneering next-generation precision genetic therapies for complex neurological conditions, today announces appointments of Norbert Riedel, Ph.D., a skilled and experienced biopharmaceutical executive, as the Chairman of its Board of Directors, and Kathrin Meyer, Ph.D., a leader in development and clinical translation of genetic therapies for the central nervous system (CNS), as Alcyone’s new Chief Scientific Officer and Head of R&D.
“I am delighted and honored to join the board of Alcyone Therapeutics,” said Dr. Riedel. “The company is developing innovative precision genetic therapies for diseases of the central nervous system and has obtained highly promising clinical results using its proprietary intrathecal drug delivery platform. I am very excited about the programs we have in development to address the significant unmet patient needs in several neurological indications.”
“Developing neurological genetic therapies demands both a highly precise biological mechanism of action and effective delivery to the central nervous system. At Alcyone, we’re uniquely positioned to merge optimized delivery and biodistribution with next-generation gene expression technology. This combination has the potential to revolutionize patient care, offering safer, more accessible, and more effective treatments. I’m thrilled to be a part of Alcyone, contributing to the advancement of therapies that can truly make a difference in the lives of patients and their families,” said Dr. Meyer.
“With a history of innovation in developing technologies for the treatment of CNS disorders, Alcyone is rapidly advancing the development of novel precision genetic therapies for X-linked diseases, including a next-generation Rett Syndrome therapy and precision delivery and biodistribution systems,” said PJ Anand, Chief Executive Officer of Alcyone Therapeutics. “Drs. Riedel and Meyer bring extraordinary skills and an immense network in the development of life-altering therapies as well as significant and relevant experience in the building of innovative companies. We are excited to welcome them to Alcyone as we embark on our next stage of growth.
Dr. Riedel has decades of leadership experience in large healthcare companies and in the biotechnology industry with a proven track record in successful company building, fundraising, and developing therapeutics across multiple indications for the CNS. His extensive industry experience includes roles as President and CEO of Aptinyx Inc., President and CEO of Naurex which was acquired by Allergan, and corporate Vice President and Chief Science and Innovation Officer at Baxter International. Dr. Riedel holds a Ph.D. and a Diploma in Biochemistry from the University of Frankfurt and serves on the boards of Jazz Pharmaceuticals, Eton Pharmaceuticals, and the Illinois Biotechnology Innovation Organization. Until its acquisition by Abbvie, he is also a member of the board of directors of Cerevel Therapeutics Holdings.
Dr. Kathrin Meyer is a pioneer in the research and clinical development of novel therapies for the CNS. Until recently, Dr Meyer was a Principal Investigator at Nationwide Children’s Hospital, Abigail Wexner Research Institute Center for Gene Therapy, and an Assistant Professor at The Ohio State University in Ohio. During Dr. Meyer’s post-doctoral research and then tenure at Nationwide Children’s, she played a key role in the successful first-in-human translation of several genetic therapies, including Zolgensma®, intrathecal Zolgensma®, Batten’s disease, Spinal Muscular Atrophy with Respiratory Distress type 1 (SMARD1) and Charcot Marie Tooth type 2 (CMT2S). She is a co-inventor of Alcyone’s miREX™ microRNA gene expression platform with a focus on Rett Syndrome and other X-linked disorders. Dr. Meyer holds a Ph.D. in the area of molecular and cellular biology from the University of Bern in Switzerland.

About Alcyone Therapeutics

Alcyone Therapeutics is a clinical-stage biotechnology company pioneering next-generation precision gene-based therapies for neurological conditions. The company integrates innovation in neuroscience and precision dosing platforms to deliver transformative therapies to patients. Alcyone leverages the synergy between Falcon™, the company’s proprietary intrathecal precision dosing and biodistribution platform and novel genetic therapeutic platforms. Falcon™ incorporates deep knowledge of cerebral spinal fluid (CSF) dynamics, computational modeling, and bioengineering. This comprehensive approach optimizes central nervous system dosing and delivery to better target the pathophysiology and anatomy specific to various neurological diseases. Alcyone’s X-reactivation genetic therapy platform was developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital (AWRI) and the University of Virginia and allows expression and upregulation of X-linked genes for the treatment of X-linked disorders including Rett Syndrome.
Alcyone has a license and collaboration agreement with a large-cap biopharmaceutical company to evaluate its novel CNS delivery device to improve patient experience and access to neurological antisense oligonucleotide (ASO) genetic therapies.
For more information, visit www.alcyonetx.com.

MEDIA & INVESTOR CONTACT:

Alcyone Therapeutics
PJ Anand, Chief Executive Officer
+1 978-709-1946
ir@alcyonetx.com

• Alcyone’s ThecaFlex DRx™ System is an FDA Breakthrough designated implantable medical device in clinical development to enable routine subcutaneous administration of therapeutics to the cerebrospinal fluid to potentially improve patient experience
• The first implantation of the ThecaFlex DRx™ System was performed at Texas Children’s Hospital in Houston, Texas by PIERRE study investigator David Bauer, MD, MPH

LOWELL, Mass., Jan. 3, 2024 /PRNewswire/ — Alcyone Therapeutics Inc. Alcyone, a biotechnology company pioneering next-generation precision genetic therapies and precision delivery solutions of therapies for complex neurological conditions, announced the first patient implanted in the PIERRE pivotal study to evaluate the safety and performance of the ThecaFlex DRx System (ThecaFlex) subcutaneous port and intrathecal catheter system for repeat intrathecal access, cerebrospinal fluid (CSF) aspiration, and delivery of SPINRAZA® (nusinersen) in spinal muscular atrophy (SMA) patients. The first PIERRE patient was implanted by study Investigator David Bauer, MD, MPH at Texas Children’s Hospital in Houston, Texas.

“The ThecaFlex device has the potential to provide a significant benefit for patients with SMA who are being treated with SPINRAZA and are resistant to lumbar punctures. It may reduce the need for serial lumbar punctures under sedation and improve the overall administration experience of SPINRAZA,” said David Bauer, MD, MPH, Principal Investigator at Texas Children’s Hospital for the PIERRE Study and professor of pediatric neurosurgery at Baylor College of Medicine.

Timothy Lotze, MD, the lead neurologist for the PIERRE study at Texas Children’s Hospital added, “Currently, most of my SMA patients treated with SPINRAZA are dosed in the interventional radiology suite due to complex spines, and many of these visits require extended time and radiation, as well as the potential need for sedation. With ThecaFlex, these patients may be able to have their therapy administered through a sub-cutaneous injection into the port in the neurology clinic, reducing the need for anesthesia or radiation.” Lotze is also a professor of pediatric neurology at Baylor.

The PIERRE pivotal study (https://clinicaltrials.gov/ct2/show/NCT05866419) will enroll up to 90 patients and will be conducted in two stages. The first stage will consist of up to 10 patients implanted in a limited number of sites in the U.S., including Texas Children’s Hospital, Boston Children’s Hospital, and Children’s Hospital of Orange County and followed for 30 days. The second stage will enroll an additional 80 patients in a broader number of sites across the U.S. and Europe.

“Alcyone is deeply committed to helping patients in need by providing solutions that have the potential to positively impact the administration of intra-CSF therapeutics. We are looking forward to providing a therapeutic delivery alternative designed to improve patient experience for SMA patients being treated with SPINRAZA who are resistant to lumbar punctures,” said Norbert Riedel, Ph.D., Chairman of Alcyone’s Board of Directors. He further added, “The implantation of the first patient with ThecaFlex represents an important step toward providing access to a delivery system specifically designed for repeat bolus intrathecal drug delivery to patients in the SMA community.”

ThecaFlex has the potential to be the first implantable device designed to enable routine subcutaneous access for delivery of antisense oligonucleotide (ASO) therapies. ThecaFlex has received CE Mark in Europe, and IDE approval to conduct a clinical investigation from the U.S. Food and Drug Administration (FDA) but is not approved for commercial use by the FDA. In addition, it has also received Breakthrough Device Designation from the U.S. FDA.

The PIERRE pivotal study is part of a collaboration between Biogen Inc. (Nasdaq: BIIB) and Alcyone previously announced in January 2023. Additional details on the trial can be found at https://clinicaltrials.gov/ct2/show/NCT05866419.

About The ThecaFlex DRx™ System
The ThecaFlex DRx System (ThecaFlex), a technology within Alcyone’s Falcon™ Delivery Platform, is in development as an implantable intrathecal (IT) catheter, catheter fixation device, and subcutaneous port system designed to provide access to the cerebrospinal fluid (CSF) for the infusion of therapies requiring repeat IT dosing. Lumbar puncture (LP), commonly known as a spinal tap, is the current standard of care approach to delivering therapeutics into the CSF. ThecaFlex is designed to be an alternative to LP, especially for people with challenging anatomy or for those who require multiple anesthesia and radiation exposures for repeat LPs or for patients for whom the treating physician determines implantation of ThecaFlex is otherwise in the patient’s best interests.

About SPINRAZA® (nusinersen)
SPINRAZA is a Biogen therapy that is approved in more than 60 countries to treat infants, children, and adults with spinal muscular atrophy (SMA). As a foundation of care in SMA, more than 14,000 individuals have been treated with SPINRAZA worldwide.¹ SPINRAZA is an antisense oligonucleotide (ASO) that targets the root cause of SMA by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.² It is administered directly into the central nervous system, where motor neurons reside, to deliver treatment where the disease starts.²

SPINRAZA has demonstrated sustained efficacy across ages and SMA types with a well-established safety profile based on data in patients treated up to 8 years, combined with unsurpassed real-world experience.³  The nusinersen clinical development program encompasses more than 10 clinical studies, which have included more than 460 individuals across a broad spectrum of patient populations, including two randomized controlled studies (ENDEAR and CHERISH). The SHINE and NURTURE open-label extension studies are evaluating the long-term impact of SPINRAZA. The most common adverse events observed in clinical studies were respiratory infection, fever, constipation, headache, vomiting and back pain. Laboratory tests can monitor for renal toxicity and coagulation abnormalities, including acute severe low platelet counts, which have been observed after administration of some ASOs.

Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS). Please click here for Important Safety Information and full Prescribing Information for SPINRAZA in the U.S., or visit your respective country’s product website.

About Alcyone Therapeutics
Alcyone Therapeutics is a biotechnology company pioneering next-generation precision gene-based therapies for complex neurological conditions. The company integrates innovation in neuroscience, precision dosing platforms, and manufacturing capabilities to deliver transformative therapies to patients. Alcyone leverages the synergy between Falcon™, the Company’s proprietary intrathecal precision dosing and biodistribution platform that incorporates deep knowledge of cerebral spinal fluid (CSF) dynamics, computational modeling, and bioengineering, and novel gene-based therapeutics platforms developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital (AWRI). This comprehensive approach allows for the optimization of central nervous system (CNS) dosing and delivery to better target the pathophysiology and anatomy specific to various neurological diseases. Alcyone’s lead programs utilize X chromosome reactivation for X-linked disorders and targets the treatment of Rett syndrome. For more information, visit www.alcyonetx.com.

Biogen Safe Harbor
This news release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, about the potential benefits of our collaboration with Alcyone; and the potential benefits of an implantable device designed to enable the administration of ASO therapies. These statements may be identified by words such as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “plan,” “possible,” “potential,” “will,” “would” and other words and terms of similar meaning. You should not place undue reliance on these statements.

These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including without limitation, risks relating to the occurrence of adverse safety events and/or unexpected concerns that may arise from additional data or analysis; the risk that we may not fully enroll our clinical trials, or enrollment will take longer than expected; failure to obtain regulatory approvals in other jurisdictions; risks of unexpected costs or delays; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; regulatory authorities may require additional information or further studies; product liability claims; third party collaboration risks; and the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations and financial condition. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement as well as the risk factors identified in our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements speak only as of the date of this news release. We do not undertake any obligation to publicly update any forward-looking statements.

References:

1. Based on commercial patients, early access patients, and clinical trial participants through December 31, 2022.
2. SPINRAZA U.S. Prescribing Information. Available at: https://www.spinraza.com/content/dam/commercial/specialty/spinraza/caregiver/en_us/pdf/spinraza-prescribing-information.pdf. Accessed: June 2023.
3. Core Data sheet, Version 13, October 2021. SPINRAZA. Biogen Inc., Cambridge, MA.

###

MEDIA & INVESTOR CONTACT:

Alcyone Therapeutics
Chris Coletta
+ 1 978-709-1946
ir@alcyonetx.com

• Alcyone’s ThecaFlex DRx™ System is an implantable medical device in development to enable routine subcutaneous administration of therapeutics to the cerebrospinal fluid
• First stage of the pivotal study expected to initiate in summer of 2023 for first 10 patients followed by enrollment of the remaining 80 patients in a second stage beginning in 2024

Lowell, Mass., June 27, 2023 / Alcyone Therapeutics Inc.  Alcyone, a biotechnology company pioneering next-generation precision genetic therapies and precision delivery solutions of therapies for complex neurological conditions, announced today that the U.S. Food and Drug Administration (FDA) has approved an Investigational Device Exemption (IDE) to initiate a pivotal trial of Alcyone’s ThecaFlex DRx System (ThecaFlex). The PIERRE pivotal study will evaluate the safety and performance of the ThecaFlex subcutaneous port and intrathecal catheter system for repeat intrathecal access, cerebrospinal fluid (CSF) aspiration, and delivery of SPINRAZA® (nusinersen) in spinal muscular atrophy (SMA) patients.

The PIERRE pivotal study (https://clinicaltrials.gov/ct2/show/NCT05866419) will enroll up to 90 patients and will be conducted in two stages. The first stage is expected to be initiated in the summer of 2023 in a limited number of sites in the U.S. and will consist of up to 10 patients enrolled, implanted, and followed for 30 days. The second stage will enroll an additional 80 patients in a broader number of sites across the U.S. and Europe and is expected to begin in 2024.

“Alcyone is excited to begin the process of evaluating the safety and performance of ThecaFlex in SMA patients being treated with SPINRAZA. ThecaFlex represents the culmination of a deliberate effort to design a delivery system specifically for repeat bolus intrathecal drug delivery,” said PJ Anand, Chief Executive Officer of Alcyone. “IDE approval and the impending start of the pivotal study are critical steps toward helping patients in need with a potential therapeutic delivery alternative and improved treatment experience.”

ThecaFlex has the potential to be the first implantable device designed to enable routine subcutaneous access for delivery of antisense oligonucleotide (ASO) therapies. ThecaFlex has received a CE Mark in Europe. In addition, it has also received Breakthrough Device Designation from the U.S. FDA.

“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, M.D., Ph.D., orthopedic surgeon, Boston Children’s Hospital, professor of orthopedic surgery, Harvard Medical School, and CureSMA Board member. “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.”
The PIERRE pivotal study is part of a previously announced collaboration between Biogen Inc. (Nasdaq: BIIB) and Alcyone that provides Biogen with the opportunity to leverage ThecaFlex with the goal of improving the patient treatment experience and accessibility for a broader population of people suffering from neurological disorders. The companies will jointly collaborate on clinical development, manufacture and commercialization of ThecaFlex for SPINRAZA and potentially other ASO therapies. The results of the PIERRE study to evaluate ThecaFlex with SPINRAZA in SMA may inform pathways for Biogen’s broader portfolio of investigational ASO therapies.

The PIERRE trial is named in memory of Pierre Boulas, a Biogen executive who played an essential role in establishing the collaboration with Alcyone. Additional details on the trial can be found at https://clinicaltrials.gov/ct2/show/NCT05866419.

About The ThecaFlex DRx™ System
The ThecaFlex DRx System (ThecaFlex), a technology within Alcyone’s Falcon™ Delivery Platform, is an implantable intrathecal (IT) catheter, catheter fixation device, and subcutaneous port system designed to provide access to the cerebrospinal fluid (CSF) for the infusion of therapies requiring repeat IT dosing. Lumbar puncture (LP), commonly known as a spinal tap, is the current standard of care approach to delivering therapeutics into the CSF. ThecaFlex is designed to be an alternative to LP, especially for people with challenging anatomy or for those who require multiple anesthesia and radiation exposures for repeat LPs or for patients whom the treating physician determines implantation of ThecaFlex is otherwise in the patient’s best interests.

ThecaFlex has received CE Mark in Europe, Breakthrough Device Designation and IDE approval to conduct a clinical investigation from the U.S. Food and Drug Administration (FDA), but is not approved for commercial use by the FDA. For more information, visit www.alcyonetx.com.

About Alcyone Therapeutics
Alcyone Therapeutics is a biotechnology company pioneering next-generation precision gene-based therapies for complex neurological conditions. The company integrates innovation in neuroscience, precision dosing platforms, and manufacturing capabilities to deliver transformative therapies to patients. Alcyone leverages the synergy between FalconTM, the Company’s proprietary intrathecal precision dosing and biodistribution platform that incorporates deep knowledge of cerebral spinal fluid (CSF) dynamics, computational modeling, and bioengineering, and novel gene-based therapeutics platforms developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital (AWRI). This comprehensive approach allows for the optimization of central nervous system (CNS) dosing and delivery to better target the pathophysiology and anatomy specific to various neurological diseases. Alcyone’s lead programs utilize X chromosome reactivation for X-linked disorders and targets the treatment of Rett syndrome. For more information, visit www.alcyonetx.com.

About SPINRAZA® (nusinersen)

SPINRAZA is a Biogen therapy that is approved to treat infants, children and adults with spinal muscular atrophy (SMA) and is approved in more than 60 countries. As a foundation of care in SMA, more than 13,000 individuals have been treated with SPINRAZA worldwide.¹
SPINRAZA is an antisense oligonucleotide (ASO) that targets the root cause of SMA by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.² It is administered directly into the central nervous system, where motor neurons reside, to deliver treatment where the disease starts.²

SPINRAZA has demonstrated sustained efficacy across ages and SMA types with a well-established safety profile based on data in patients treated up to 8 years, combined with unsurpassed real-world experience.³  The nusinersen clinical development program encompasses more than 10 clinical studies, which have included more than 460 individuals across a broad spectrum of patient populations, including two randomized controlled studies (ENDEAR and CHERISH). The ongoing SHINE and NURTURE open-label extension studies are evaluating the long-term impact of SPINRAZA. The most common adverse events observed in clinical studies were respiratory infection, fever, constipation, headache, vomiting and back pain. Laboratory tests can monitor for renal toxicity and coagulation abnormalities, including acute severe low platelet counts, which have been observed after administration of some ASOs.

Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS). Please click here for Important Safety Information and full Prescribing Information for SPINRAZA in the U.S., or visit your respective country’s product website.

References:

1. Based on commercial patients, early access patients, and clinical trial participants through March 31, 2022.
2. SPINRAZA U.S. Prescribing Information. Available at: https://www.spinraza.com/content/dam/commercial/specialty/spinraza/caregiver/en_us/pdf/spinraza-prescribing-information.pdf. Accessed: June 2023.
3. Core Data sheet, Version 13, October 2021. SPINRAZA. Biogen Inc., Cambridge, MA.

###

MEDIA & INVESTOR CONTACT:

Alcyone Therapeutics
Chris Coletta
+ 1 978-709-1946
ir@alcyonetx.com

• Alcyone’s ThecaFlex DRx™ System is an implantable medical device in development for intrathecal drug delivery

Cambridge, Mass. and Lowell, Mass. – JANUARY 4, 2023 – Biogen Inc. (Nasdaq: BIIB) and Alcyone Therapeutics (Alcyone) have entered into a license and collaboration agreement to develop Alcyone’s ThecaFlex DRx™ System, an implantable medical device intended for subcutaneous delivery of antisense oligonucleotide (ASO) therapies into the intrathecal space. Through this agreement, Biogen aims to leverage the ThecaFlex DRx™ System with a goal of improving the patient treatment experience and accessibility for a broader population of people suffering from neurological disorders, such as spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS).

The ThecaFlex DRxT™ System has the potential to be the first implantable device designed to enable routine subcutaneous administration of ASO therapies to the cerebrospinal fluid. The ThecaFlex DRx™ System has received a CE Mark in Europe. In addition, it has also received Breakthrough Device Designation from the U.S. Food and Drug Administration (FDA) and will require further clinical studies before it can be submitted to the FDA for review.

“We are continually listening to the neuromuscular disease community and whenever possible, adapting our work to meet their evolving needs for treatment and patient care,” said Priya Singhal, Interim Head of R&D at Biogen. “Biogen looks forward to working with Alcyone to explore the potential of this device, which we believe will provide greater flexibility to people with spinal muscular atrophy and other neurological disorders as well as their doctors in making the right treatment decisions.”

“Alcyone designed the ThecaFlex DRx™ System to be a therapeutic delivery alternative for patients with a chronic neurological condition whose current treatment requires repeat lumbar puncture,” said PJ Anand, Chief Executive Officer of Alcyone. “This agreement underscores Alcyone’s expertise in cerebrospinal fluid delivery technology which we believe will lead to an improved treatment experience for some people living with neurological conditions and their caregivers. We consider Biogen, a global leader, an ideal collaborator toward this mutual goal.”

Under the terms of the agreement, Biogen will make an upfront payment of $10 million to Alcyone for an exclusive global license to the ThecaFlex DRx™ System in SMA and ALS as well as a co-exclusive global license in an unnamed indication. Should certain development and commercial milestones be achieved, Alcyone will be eligible to receive up to $41 million in potential milestone payments. The deal also provides flexibility to expand the collaboration as additional ASO therapies progress through Biogen’s pipeline.

Biogen and Alcyone will jointly collaborate on clinical development of the ThecaFlex DRx™ System for ASO therapies, and Alcyone will be solely responsible for its manufacture and commercialization. The ThecaFlex DRx™ System will initially be evaluated with SPINRAZA® (nusinersen) in SMA, which will inform pathways for Biogen’s broader portfolio of investigational ASO therapies.

About The ThecaFlex DRx™ System
The ThecaFlex DRx™ System (ThecaFlex), a technology within Alcyone’s Falcon™ Delivery Platform, is an implantable intrathecal (IT) catheter, catheter fixation device, and subcutaneous port system designed to provide access to the cerebrospinal fluid (CSF) for the infusion of therapy by IT bolus administration. Lumbar puncture (LP), commonly known as a spinal tap, is the current standard of care approach to delivering therapeutics into the CSF. ThecaFlex is designed to be an alternative to LP, especially for people with challenging anatomy or for those who require multiple anesthesia and radiation exposures for repeat LPs.

The ThecaFlex DRx™ System has received CE Mark in Europe and Breakthrough Device Designation from the U.S. Food and Drug Administration (FDA). ThecaFlex is not approved by the FDA. For more information, visit www.alcyonetx.com.

About SPINRAZA® (nusinersen)
SPINRAZA is approved to treat infants, children and adults with spinal muscular atrophy (SMA) and is approved in more than 60 countries. As a foundation of care in SMA, more than 13,000 individuals have been treated with SPINRAZA worldwide.[1]

SPINRAZA is an antisense oligonucleotide (ASO) that targets the root cause of SMA by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.2 It is administered directly into the central nervous system, where motor neurons reside, to deliver treatment where the disease starts. [2]

SPINRAZA has demonstrated sustained efficacy across ages and SMA types with a well-established safety profile based on data in patients treated up to 8 years, combined with unsurpassed real-world experience. [3] The nusinersen clinical development program encompasses more than 10 clinical studies, which have included more than 460 individuals across a broad spectrum of patient populations, including two randomized controlled studies (ENDEAR and CHERISH). The ongoing SHINE and NURTURE open-label extension studies are evaluating the long-term impact of SPINRAZA. The most common adverse events observed in clinical studies were respiratory infection, fever, constipation, headache, vomiting and back pain. Laboratory tests can monitor for renal toxicity and coagulation abnormalities, including acute severe low platelet counts, which have been observed after administration of some ASOs.

Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS). Please click here for Important Safety Information and full Prescribing Information for SPINRAZA in the U.S., or visit your respective country’s product website.

About Biogen
As pioneers in neuroscience, Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological diseases as well as related therapeutic adjacencies. One of the world’s first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Sir Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today, Biogen has a leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, and developed the first and only approved treatment to address a defining pathology of Alzheimer’s disease. Biogen is also commercializing biosimilars and focusing on advancing one of the industry’s most diversified pipelines in neuroscience that will transform the standard of care for patients in several areas of high unmet need.

We routinely post information that may be important to investors on our website at www.biogen.com. Follow us on social media – Twitter, LinkedIn, Facebook, YouTube.

About Alcyone Therapeutics
Alcyone Therapeutics is a biotechnology company pioneering next-generation precision gene-based therapies for complex neurological conditions. The company integrates innovation in neuroscience, precision dosing platforms, and manufacturing capabilities to deliver transformative therapies to patients. Alcyone leverages the synergy between Falcon™, the Company’s proprietary intrathecal precision dosing and biodistribution platform that incorporates deep knowledge of cerebral spinal fluid (CSF) dynamics, computational modeling, and bioengineering, and novel gene-based therapeutics platforms developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital (AWRI). This comprehensive approach allows for the optimization of central nervous system (CNS) dosing and delivery to better target the pathophysiology and anatomy specific to various neurological diseases. Alcyone’s lead programs utilize X chromosome reactivation for X-linked disorders and targets the treatment of Rett syndrome, and gene replacement for the treatment of IGHMBP2-related disorders including spinal muscular atrophy with respiratory distress type 1 (SMARD1) and Charcot Marie Tooth disease type 2S (CMT2S). For more information, visit www.alcyonetx.com.

Biogen Safe Harbor
This news release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, the potential benefits, safety and efficacy of the ThecaFlex DRx™ System; the clinical development program for ThecaFlex DRx™ system; our collaboration with Alcyone; the potential of our commercial business and pipeline programs and risks and uncertainties associated with drug development and commercialization. These forward-looking statements may be accompanied by words such as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “intend,” “may,” “plan,” “potential,” “possible,” “will,” “would” and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk and only a small number of research and development programs result in commercialization of a product. Results in early stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements or the scientific data presented.

These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including without limitation, uncertainty of success in the development and potential commercialization of the ThecaFlex DRx™ System; the risk that we may not fully enroll our clinical trials or enrollment will take longer than expected; unexpected concerns may arise from additional data, analysis or results obtained during our clinical trials; regulatory authorities may require additional information or further studies, or may fail or refuse to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risks of unexpected hurdles, costs or delays; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; product liability claims; and the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations and financial condition. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement, as well as the risk factors identified in our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this news release.

We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.

References:

1. Based on commercial patients, early access patients, and clinical trial participants through March 31, 2022.
2. SPINRAZA U.S. Prescribing Information. Available at: https://www.spinraza.com/content/dam/commercial/specialty/spinraza/caregiver/en_us/pdf/spinraza-prescribing-information.pdf. Accessed: October 2022.
3. Core Data sheet, Version 13, October 2021. SPINRAZA. Biogen Inc, Cambridge, MA.

###

MEDIA CONTACTS:
Biogen
Jack Cox
+ 1 210 544 7920
public.affairs@biogen.com

INVESTOR CONTACTS:
Biogen
Mike Hencke
+1 781 464 2442
IR@biogen.com

Alcyone Therapeutics
+ 1 978-709-1946
ir@alcyonetx.com

– X-reactivation gene therapy is safe and well-tolerated in Rett syndrome mouse model –
– Preclinical safety and efficacy data presented on ACTX-401, a gene replacement therapy for the treatment of IGHMBP2-related disorders, validates current Phase 1/2 study –
– Data validate productivity for AAV9 gene therapy manufacturing through platform upstream process –

LOWELL, Mass. – May 18, 2022 – Alcyone Therapeutics Inc. (“Alcyone”), a biotechnology company pioneering next-generation precision gene-based therapies for complex neurological conditions, today presents preclinical data showing the Company’s partnered novel adeno associated virus serotype 9 (AAV9) gene therapy vector ACTX-101 safely and effectively reactivates the inactive X chromosome in a Rett syndrome mouse model and is safe in a preliminary nonhuman primate study at the American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting.

Alcyone also presented data from its partnered gene therapy program for ACTX-401, a gene replacement therapy currently in a Phase 1/2 clinical study for the treatment of IGHMBP2-related disorders (IRDs), and on the Company’s internal AAV manufacturing process and platform.

“X-reactivation is a novel gene therapy approach to correcting mutations in X-linked dominant disorders such as Rett syndrome,” said Ottavio Vitolo, M.D., M.M.Sc., Chief Medical Officer and Global Head of R&D at Alcyone. “The Rett mouse model preclinical data produced thus far by Dr. Kathrin Meyer’s lab at the Abigail Wexner Research Institute at Nationwide Children’s Hospital and by Dr. Sanchita Bhatnagar, formerly with the University of Virginia, currently at the University of California Davis School of Medicine, demonstrate that activation of the silenced X chromosome has the potential to be a safe and effective approach, which mitigates the risk of MECP2 overexpression typical of gene replacement therapies.”

“Based on our growing body of preclinical data, we are pioneering a vectorized therapeutic X-reactivation platform that has the potential for clinical application to address Rett syndrome and other X-linked dominant disorders,” said PJ Anand, Chief Executive Officer of Alcyone. “Alcyone’s differentiated gene therapy combines the X-reactivation platform with Falcon^TM, our proprietary intrathecal CNS precision drug delivery and dosing technology platform, to optimally address the current challenges of treating people living with severe neurological disorders.”

Highlights from Alcyone’s 2022 ASGCT Presentations

Rett Syndrome

Oral presentation, A Novel Gene Therapy for Rett Syndrome through Reactivation of the Silent X Chromosome:

• ACTX-101 is a novel, vectorized gene therapy approach to X-reactivation for the treatment of Rett syndrome.
• Data from a mouse model of Rett and preliminary nonhuman primate data suggest that the X-reactivation gene therapy is safe, efficacious, and well-tolerated.
• Administration of the X-reactivation gene therapy improved MeCp2 expression and respiratory patterns, behavioral indicators of Rett syndrome, bodyweight, and survival time in a severe Rett syndrome mouse model.

This abstract received the Excellence in Research Award in recognition as one of the top 18 abstracts submitted to the ASGCT 25th Annual Meeting by a postdoctoral fellow or student.

SMARD1/CMT2S

Oral presentation, Multicenter AAV Gene Therapy Studies for SMARD1/CMT2S Establishes Safety and Efficacy in Multiple Animal Models and poster presentation, Evaluation of AAV9 Gene Therapy for SMARD1/CMT2S in Different Mouse Models Reveal Differences in Efficacy Dependent on Promoter Choice:

• ACTX-401 is a gene replacement therapy for the treatment of IGHMBP2-related disorders (IRDs) including SMARD1/CMT2S.
• Data from mouse models of IRDs and preliminary nonhuman primate data suggested that the gene replacement therapy is safe and well-tolerated.
• Administration of the gene replacement therapy improved behavioral and physiological indicators of IRDs and weight gain in a dose-dependent manner in three mouse models of the disease.
• Construct testing on multiple model systems helped identify the optimal gene therapy vector and establish dose-rationale critical in the development of clinical trials for SMARD1/CMT2S.
• The safety and efficacy data led to the successful approval of an investigational new drug (IND) application.
• The gene replacement therapy clinical Phase 1/2 study is actively enrolling patients and has been well-tolerated to date.

Manufacturing

Poster presentation, Development of an Upstream Process and Analytics for AAV Manufacturing:

• Development and optimization for dosing (vg) and potency assays are critical to ensure optimal process development and continued robustness of process and critical quality attributes (CQA).
• Development and optimization of an upstream process is critical and must meet the criteria for productivity and scalability with a line of sight to regulatory and economic challenges for commercialization.
• We have developed an upstream process that is flexible, versatile, and scalable with continuous process improvements for productivity.
• Optimization of chemistry manufacturing and controls (CMC) can accelerate a therapeutic candidate through the regulatory process and to the clinic.

About Alcyone Therapeutics

Alcyone Therapeutics is a biotechnology company pioneering next-generation precision gene-based therapies for complex neurological conditions. The company integrates innovation in neuroscience, precision dosing platforms, and manufacturing capabilities to deliver transformative therapies to patients. Alcyone leverages the synergy between Falcon^TM, the company’s proprietary intrathecal precision dosing and biodistribution platform that incorporates deep knowledge of cerebral spinal fluid (CSF) dynamics, computational modeling, and bioengineering, and four novel gene-based therapeutics platforms developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital (AWRI). This comprehensive approach allows for the optimization of central nervous system (CNS) dosing and delivery to better target the pathophysiology and anatomy specific to various neurological diseases. Alcyone’s lead programs utilize X chromosome reactivation for X-linked disorders and targets the treatment of Rett syndrome, and gene replacement for the treatment of IGHMBP2-related disorders including spinal muscular atrophy with respiratory distress type 1 (SMARD1) and Charcot Marie Tooth disease type 2S (CMT2S). For more information, visit www.alcyonetx.com.

About Alcyone’s Strategic Collaboration with the Abigail Wexner Research Institute at Nationwide Children’s Hospital

Alcyone works closely with scientists from the Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Children’s Hospital (AWRI) in Columbus, Ohio, where four discrete gene therapy platform technologies, each with novel and differentiated mechanisms of action, including X-reactivation, conventional transgene replacement, vectorized exon skipping and promoter modulation are designed, developed, and advanced towards the clinic. Through the collaboration with AWRI, Alcyone is exploring the potential for the clinical application of these therapeutics using Falcon^TM, its proprietary CNS precision drug delivery and dosing technology platform, to improve the lives of people impacted by severe neurological conditions. The research is led by Kathrin Meyer, Ph.D., and Nicolas Wein, Ph.D., Principal Investigators in the Center for Gene Therapy at AWRI. Both Dr. Meyer and Dr. Wein are members of Alcyone’s Scientific Advisory Board (SAB), with Dr. Meyer serving as Chief Scientific Advisor and Chair of Alcyone’s SAB.

Contact:
Aurora Krause
Alcyone Therapeutics
(978) 709-1946
ir@alcyonetx.com

– Abstract on Rett syndrome treatment through X-reactivation receives Excellence in Research Award –
– Updated preclinical safety and efficacy data will be presented for ACTX-401, a gene replacement therapy currently in a Phase 1/2 study for the treatment of IGHMBP2-related disorders –

LOWELL, Mass. – May 2, 2022 – Alcyone Therapeutics Inc. (“Alcyone”), a biotechnology company pioneering next-generation precision gene-based therapies for complex neurological conditions, today announced the acceptance of four abstracts at the 25th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, an event being held May 16-19, 2022, at the Walter E. Washington Convention Center in Washington, D.C. and virtually.

Accepted abstracts include preclinical data from its X-reactivation gene therapy platform, an approach to correct X-linked dominant genetic disorders by reactivating the silenced X chromosome; preclinical safety and efficacy data from its gene replacement platform, which utilizes viral vectors to deliver a functional gene to compensate for a cell’s missing or mutated gene; and improved efficiency of AAV gene therapy manufacturing through its platform upstream process, which is scalable and easily transferrable between multiple systems.

“The oral presentations will highlight the potential of Alcyone’s discrete gene therapy platforms, X-reactivation for treatment of Rett syndrome and gene replacement for IGHMBP2-related disorders, spinal muscular atrophy with respiratory distress type 1 and Charcot Marie Tooth disease type 2S, which we are advancing in partnership with the Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Children’s Hospital,” said PJ Anand, Chief Executive Officer of Alcyone Therapeutics. “We also will have a poster presentation demonstrating the scalability and multi-system transferability of our AAV production process, which is a critical piece in our gene therapy platform development.”

Anand continued, “Alcyone’s multidisciplinary approach to addressing complex neurological conditions is comprised of our partnered novel gene-based therapeutic platform combined with our proprietary CNS precision drug delivery and dosing technology platform, Falcon^TM, and leverages a scalable CMC process optimized to produce high-quality clinical material. Our three-pronged approach is designed to better address the current challenges of treating people living with severe neurological disorders.”

The presentations are listed below, and the full preliminary program is available online on the ASGCT website.

Oral Presentation: A Novel Gene Therapy for Rett Syndrome through Reactivation of the Silent X Chromosome

Oral Presentation Details: Presenting Author: Kathrin Meyer, Ph.D., Principal Investigator, Nationwide Children’s Hospital and Chief Scientific Advisor, Alcyone Session Title: Novel Therapeutic Targets to Treat CNS Disorders Session Date/Time: Wednesday, May 18, 3:45 – 5:30 p.m. ET Presentation Time: 4:45 – 5:00 p.m. ET Room: Room 202 Abstract #: 837

Samantha Powers, Ph.D., from the Center for Gene Therapy at Nationwide Children’s Hospital, received the Excellence in Research Award in recognition for presenting one of the top 18 abstracts submitted for the ASGCT 25th Annual Meeting by a postdoctoral fellow or student.

Oral Presentation: Multicenter AAV Gene Therapy Studies for SMARD1/CMT2S Establish Safety and Efficacy in Multiple Animal Models and Pave the Way for Initiation of a Phase I/II Clinical Trial

Oral Presentation Details: Presenting Author: Kathrin Meyer, Ph.D., Principal Investigator, Nationwide Children’s Hospital and Chief Scientific Advisor, Alcyone Session Title: Musculo-skeletal Diseases Session Date/Time: Monday, May 16, 10:15 a.m. – 12:00 p.m. ET Presentation Time: 11:15 – 11:30 a.m. ET Room: Salon G Abstract #: 33

Poster Presentation: Development of an Upstream Process and Analytics for AAV Manufacturing

Poster Presentation Details: Presenting Author: Desyree Jesus, Ph.D., Associate Director, CMC Analytics, Alcyone Session Title: Vector Product Engineering, Development or Manufacturing III Session date/time: Wednesday, May 18, 5:30 – 6:30 p.m. ET Room: Hall D Poster Board #: W-286 Abstract #: 1160

Poster Presentation: Evaluation of AAV9 Gene Therapy for SMARD1/CMT2S in Different Mouse Models Reveal Differences in Efficacy Dependent on Promoter Choice

Poster Presentation Details: Presenting Author: J. Andrea Sierra Delgado, M.D., M.Sc., Chief Research Associate in Dr. Kathrin Meyer’s Lab, Nationwide Children’s Hospital Session Title: Musculo-skeletal Diseases Session Date/Time: Wednesday, May 18, 5:30 – 6:30 p.m. ET Room: Hall D Poster Board #: W-198 Abstract #: 1072

About Alcyone Therapeutics

Alcyone Therapeutics is a biotechnology company pioneering next-generation precision gene-based therapies for complex neurological conditions. The Company integrates innovation in neuroscience, precision dosing platforms, and manufacturing capabilities to deliver transformative therapies to patients. Alcyone leverages the synergy between Falcon^TM, the Company’s proprietary intrathecal precision dosing and biodistribution platform that incorporates deep knowledge of cerebral spinal fluid (CSF) dynamics, computational modeling, and bioengineering, and four novel gene-based therapeutics platforms developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital (AWRI). This comprehensive approach allows for the optimization of central nervous system (CNS) dosing and delivery to better target the pathophysiology and anatomy specific to various neurological diseases. Alcyone’s lead programs utilize X-chromosome reactivation for X-linked disorders and targets the treatment of Rett syndrome, and gene replacement for the treatment of IGHMPB-2 related disorders including spinal muscular atrophy with respiratory distress type 1 (SMARD1) and Charcot Marie Tooth disease type 2S (CMT2S). For more information, visit www.alcyonetx.com.

About Alcyone’s Strategic Collaboration with the Abigail Wexner Research Institute at Nationwide Children’s Hospital Alcyone works closely with scientists from the Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Children’s Hospital (AWRI) in Columbus, Ohio, where four discrete gene therapy platform technologies, each with novel and differentiated mechanisms of action, including X-reactivation, conventional transgene replacement, vectorized exon skipping, and promoter modulation were designed, developed, and are being advanced towards the clinic. Alcyone has optioned the four programs and is funding research to explore the potential for the clinical application of these therapeutics using Falcon^TM, its proprietary CNS precision drug delivery and dosing technology platform, to improve the lives of people impacted by severe neurological conditions. The research is led by Kathrin Meyer, Ph.D., and Nicolas Wein, Ph.D., Principal Investigators in the Center for Gene Therapy at AWRI. Both Dr. Meyer and Dr. Wein sit on Alcyone’s Scientific Advisory Board (SAB), with Dr. Meyer serving as Chief Scientific Advisor and Chair of Alcyone’s SAB.

Contact:
Aurora Krause
Alcyone Therapeutics
(978) 709-1946
ir@alcyonetx.com

LOWELL, Mass., Oct. 25, 2021 – Alcyone Therapeutics (“Alcyone”), a biotechnology company developing precision therapies for neurological disorders with high unmet medical needs, today announced four key appointments to its executive leadership team, bringing significant expertise in neuroscience and genetic medicines development. Alcyone Therapeutics appointed Ottavio Vitolo, M.D., M.M.Sc., Chief Medical Officer and Global Head of R&D; Ravi Mehrotra, Ph.D., Chief Financial Officer and Head of Strategy; Susan D’Costa, Ph.D., Executive Vice President & Global Head of Technology; and Rachel Salzman, D.V.M., Executive Vice President of Portfolio, External Affairs & Development.

“I am thrilled to welcome Ottavio, Ravi, Susan and Rachel to Alcyone’s executive management team. They are all accomplished leaders who bring diverse and vast expertise in the development of novel treatments for neurological disorders, in particular precision genetic therapies. They also all share the mission to build Alcyone into a premier organization that will bring transformative medicines to patients in need of better treatment options,” said PJ Anand, Founder and Chief Executive Officer of Alcyone Therapeutics. “They will be integral to building our future as a leader in the development of central nervous system (CNS) therapies through a uniquely integrated, multi-disciplinary approach, as we leverage our Falcon^TM precision dosing technology and our multiple genetic therapy platforms.”

• Ottavio Vitolo, M.D., M.M.Sc., Chief Medical Officer and Global Head of R&D, is a neuropsychiatrist and an experienced industry executive in CNS drug development. His experience ranges from small molecules to biologics and gene therapy across rare and common neurological and psychiatric conditions. He was previously Chief Medical Officer and Head of R&D at Relmada Therapeutics and worked at Homology Medicines, Pfizer and Shire (Takeda). He is the co-founder of Vitam Therapeutics, a startup focused on small molecules for neuropsychiatric disorders. Dr. Vitolo holds an M.M.Sc. in translational medicine from Harvard University Medical School and an M.D. from University of Rome La Sapienza.
• Ravi Mehrotra, Ph.D., Chief Financial Officer and Head of Strategy, brings over two decades of leadership in biotechnology equity research and investment banking, as well as executive team operational experience. Dr. Mehrotra has held multiple leadership positions at banks, including at Evercore ISI, MTS Health Partners, Credit Suisse and Cowen. Dr. Mehrotra earned a B.S (Honors) and Ph.D. in biochemistry and molecular biology from The Wellcome Trust Center, Manchester University, UK.
• Susan D’Costa, Ph.D., Executive Vice President & Global Head of Technology, is a molecular virologist and has significant expertise in viral vector analytics process development and manufacturing. Prior to Alcyone, Dr. D’Costa held various leadership positions at Thermo Fisher Scientific, Viral Vector Services and its predecessor companies. She earned a Ph.D. in biology, specializing in molecular virology from Texas Tech University, an M.S. in biochemistry and a B.S. in microbiology/biochemistry from Mumbai University.
• Rachel Salzman, D.V.M., Executive Vice President of Portfolio, External Affairs & Development, was previously Founder, Chief Executive Officer and President at SwanBio Therapeutics, a gene therapy company, where she led fundraising, developed the company’s initial operational structure and oversaw its R&D footprint. She also served as Chief Science Officer at The Stop ALD Foundation, where she made critical contributions in driving forward the world’s first ex-vivo lentiviral gene therapy clinical trial conducted in non-HIV infected patients. Dr. Salzman has been an active leader for over 20 years in the American Society of Gene & Cell Therapy. Dr. Salzman earned her D.V.M. from Oklahoma State University College of Veterinary Medicine and B.S. in animal science from Rutgers University.

About Alcyone Therapeutics
Alcyone Therapeutics is a biotechnology company developing precision therapies for neurological disorders with high unmet medical needs. The company integrates innovation in neuroscience, precision dosing platforms and in-house manufacturing capabilities to deliver transformative therapies to patients. Alcyone leverages the synergy between Falcon^TM, the company’s proprietary intrathecal precising dosing platform that incorporates deep knowledge of CSF fluid dynamics, computational modeling and bioengineering, and multiple novel genetic therapy platforms developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital (AWRI). This comprehensive approach allows for the optimization of central nervous system (CNS) dosing and delivery to better target the pathophysiology and anatomy specific to various neurological disease areas. Alcyone’s lead programs target the treatment of Rett syndrome and IGHMBP2-Related Diseases (IRD). For more information, visit https://alcyonetx.com/, and follow Alcyone on LinkedIn and Twitter.

– Alcyone’s proprietary CNS precision delivery and dosing platform enables unprecedented control and versatility for CNS biodistribution and gene therapy development –
– RTW Investments, LP contributes $23 million in funding and will join Alcyone’s Board of Directors –
– Alcyone’s pipeline currently utilizes 4 gene therapy platforms and 12 AAV gene therapy programs targeting severe CNS disorders; lead program based upon novel “Pipeline in a Product” X-Reactivation platform, with Rett syndrome as initial indication –
– Collaboration with Abigail Wexner Research Institute at Nationwide Children’s Hospital (AWRI) leverages Alcyone’s delivery platform and AWRI’s research and technologies to advance broad pipeline –

LOWELL, Mass., June 9, 2021 – Alcyone Therapeutics (“Alcyone”), a biotechnology company pioneering next-generation precision central nervous system (CNS) therapies to improve the lives of patients and families impacted by severe neurological conditions, today announced its launch and $23 million in funding from funds affiliated with RTW Investments, LP (“RTW”). Piratip Pratumsuwan, Managing Director at RTW, will join Alcyone’s Board of Directors.

Alcyone’s optioned pipeline currently includes 12 adeno-associated virus (AAV) gene therapy programs for severe CNS disorders. Alcyone’s four gene therapy platform technologies include X-reactivation, conventional transgene replacement, vectorized exon skipping and promotor modulation.

Through a collaboration, Alcyone’s proprietary, next-generation CNS delivery platform and product development, manufacturing and commercialization capabilities will unite with four gene therapy technologies and discovery, research and early development capabilities developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital (AWRI). At AWRI, the collaborations are led by Kathrin Meyer, Ph.D., and Nicolas Wein, Ph.D., both Principal Investigators in the Center for Gene Therapy at AWRI. They are investigating and employing the use of gene therapies for the prevention and treatment of certain human diseases. Dr. Meyer is Chair of Alcyone’s Scientific Advisory Board.

“Our mission at Alcyone Therapeutics is to provide life-changing therapies for children and their families impacted by severe neurological conditions,” said PJ Anand, Founder, President and Chief Executive Officer of Alcyone Therapeutics. “Our next-generation precision delivery platform shows strong potential in overcoming the fundamental challenge in CNS therapy development. Coupled with multiple cutting-edge gene therapy technologies and research approaches at the Abigail Wexner Research Institute at Nationwide Children’s Hospital, we aim to transform the efficiency of CNS therapeutics. As we advance our broad pipeline towards the clinic, we are grateful to the investors who have supported us and pleased to have the financial and strategic support of RTW.”

Alcyone’s precision CNS delivery technology platform allows for unprecedented control and versatility for biodistribution of genetic medicines to CNS regions of interest, including deep brain regions and spare off-target areas. Alcyone’s CNS precision delivery can improve the efficiency of CNS gene therapy significantly impacting safety and efficacy, and the number of vectors needed, for an effective gene therapy.

Mr. Pratumsuwan commented, “We are excited by the potential for Alcyone’s CNS precision delivery platform to generate a pipeline of drug candidates aimed at improving the lives of patients affected by neurological disease. I look forward to working with Alcyone’s Board and team of experienced gene therapy leaders as they advance towards the clinic.”

About Alcyone Therapeutics

Alcyone Therapeutics is a biotechnology company pioneering next-generation precision central nervous system (CNS) therapies to improve the lives of patients and families impacted by severe neurological conditions. Alcyone Therapeutics is advancing a pipeline of adeno-associated virus (AAV) gene therapy programs that leverage its proprietary, next-generation CNS delivery technology platform. Alcyone Therapeutics’ lead programs target the treatment of Rett syndrome and spinal muscular atrophy with respiratory distress type 1 (SMARD1). For more information, please visit https://alcyonetx.com/.

About RTW Investments, LP (RTW)

RTW Investments, LP (“RTW”) is a New York-based, global, full life-cycle investment firm that focuses on identifying transformational and disruptive innovations across the biopharmaceutical and medical technologies sectors. As a leading partner of industry and academia, RTW combines deep scientific expertise with a solution-oriented investment approach to support emerging medical therapies and the companies and/or academics developing them.

For further information about RTW, please visit www.RTWfunds.com.

LOWELL, Mass., March 11, 2019 – Alcyone Lifesciences, Inc., a leader in advanced technologies to treat complex neurological and oncological conditions through the company’s proprietary advanced precision delivery platforms, announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Device Designation for its ThecaFlex DRx™ System.

To qualify for the FDA’s Breakthrough Device Program, the medical device must provide more effective diagnosis or treatment of a life-threatening or irreversible debilitating condition, and either represent a breakthrough technology, have no approved or cleared alternatives, offer significant advantages over existing alternatives, or be in the best interest of patients.  Alcyone provided the Agency with information to support that the ThecaFlex DRx System meets all of the Breakthrough Device criteria.

The proposed indication for use reviewed by the FDA for the ThecaFlex DRx System includes, “The ThecaFlex DRx System consists of an implantable subcutaneous port and intrathecal catheter used to provide access for cerebrospinal fluid (CSF) aspiration and infusion of therapies approved for intrathecal bolus administration. The ThecaFlex DRx System is indicated for patients 3 or more years of age requiring chronic bolus intrathecal treatment for life threatening, debilitating central nervous system (CNS) disorders such as neurodegenerative diseases and CNS malignancies, who are precluded from lumbar puncture (LP) due to spinal anomalies or who are resistant to repeated LP for therapy administration and CSF aspiration required for therapy administration or monitoring. A patient resistant to repeated LP is defined as one with respiratory issues or other comorbidities who is at an increased risk for complications due to the need for repeat anesthesia and imaging radiation exposure to safely perform an LP, and patients for whom the treating physician determines implantation of the ThecaFlex DRx System is otherwise in the patient’s best interests.”

Richard S. Finkel, M.D., division chief, Division of Neurology, Department of Pediatrics at Nemours Children’s Hospital in Orlando FL remarked, “the Breakthrough Device Designation for the ThecaFlex DRx System is an important milestone in the advancement of therapy enabling technologies, to provide potentially safer and more effective treatment of patients with debilitating neurodegenerative diseases such as SMA and Huntington Disease.  Intrathecally delivered therapies are often challenging to administer, and this novel delivery system will make such drugs more readily accessible to broader patient populations.”

The FDA’s Breakthrough Devices Program is designed to facilitate the development and expedite the regulatory reviews of these devices by providing a pathway for more frequent interactions with the FDA’s experts to identify areas of agreement in a timely way.  Elsa Chi Abruzzo, vice president of regulatory, clinical, and quality affairs at Alcyone Lifesciences commented, “the Alcyone Team and our clinical investigators are very pleased that as a Breakthrough Device, the ThecaFlex DRx System will be eligible for prioritized regulatory reviews as we prepare to file an Investigational Device Exemption (IDE) clinical trial submission with the FDA this year to evaluate the safety and efficacy of the device in patients with Spinal Muscular Atrophy (SMA).”

Brian Snyder, M.D., Ph.D., orthopedic surgeon, Boston Children’s Hospital and professor of orthopedic surgery, Harvard Medical School added, “patients who require chronic intrathecal administration of therapy needing repeat sedation with anesthesia or radiographic imaging to facilitate access would benefit from this single surgical insertion of a device that obviates the need for repeat anesthetic or extra radiation exposure.”

The FDA issued a Drug Safety Communication on April 27, 2017 regarding a new label warning stating that exposure to general anesthetic and sedation drugs over multiple procedures may negatively affect brain development in children younger than three years.  “Though currently the ThecaFlex DRx System is designed for patients three years of age and older who tend to have more complex spinal anatomy preventing LP or who generally have more anxiety regarding LP than younger children, Alcyone is planning to miniaturize the ThecaFlex DRx System to also benefit some of these younger patients who may not be able to tolerate LP without sedation,” added PJ Anand, chief executive officer for Alcyone Lifesciences.

About Alcyone Lifesciences, Inc.
Alcyone Lifesciences, based in Lowell, Massachusetts, is a privately-held therapeutic device company and a leader in transforming disease modifying therapies for rare and orphan disorders through Advanced Precision Delivery Platforms.  For more information, please visit www.alcyonels.com

Contact
Alcyone Life Sciences, Inc.
PJ Anand
Chief Executive Officer
978-709-1946
info@alcyonels.com