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Alcyone Presents Preclinical Data on Gene Therapy Programs with The Center for Gene Therapy at The Abigail Wexner Research Institute (AWRI) at ASGCT 2022

– 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 FalconTM, 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 presentationA 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 presentationMulticenter 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 presentationDevelopment 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 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 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 FalconTM, 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

Alcyone Announces Two Oral Presentations on its Gene Therapy Platforms at The 25th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

– 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, FalconTM, 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 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 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 FalconTM, 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

Alcyone Therapeutics Strengthens Executive Team with New Senior Leadership Appointments

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 FalconTM 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 FalconTM, 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 Therapeutics Launches to Advance Next-Generation Gene Therapies for CNS Disorders with $23 Million in Funding Contribution from RTW Investments

– 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.

Alcyone Lifesciences Receives Breakthrough Device Designation from U.S. FDA for the ThecaFlex DRx™ System

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