Cystinosis

March 12, 2024 -- Arctic Therapeutics ehf, an Icelandic drug development company and partner Nacuity Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other diseases caused by oxidative stress, today announced approval by the European Medicines Agency (EMA) for initiation of the first clinical trial evaluating AT-001 (Nacuity’s NPI-001) for the treatment of Hereditary Cystatin C Amyloid Angiopathy (HCCAA) in Iceland. HCCAA is an ultra rare, serious condition characterized by the accumulation of amyloid in the brain's blood vessels, which can lead to cerebral hemorrhage, stroke, and progressive neurological deterioration, including dementia. Current treatment options are severely limited, focusing primarily on symptom management rather than addressing the underlying cause of the disease. AT-001 is a differentiated, purified, prescription-quality, GMP-grade N-acetylcysteine amide (NACA). “EMA's approval paves the way for a comprehensive investigation into the safety and efficacy of a novel therapeutic approach designed to halt or reverse the progression of HCCAA. Our study aims to explore the potential of AT-001 in modifying the disease's course, improving patient outcomes, and ultimately, saving lives,” said Ivar Hakonarson, CEO and co-founder of Arctic. In September 2022, Nacuity and Arctic entered into an exclusive licensing agreement and strategic partnership for the development and commercialization of Nacuity’s NPI-001 under the name AT-001 for HCCAA in Iceland and related forms of amyloid angiopathies (CAAs) worldwide. Nacuity is also currently evaluating NPI-001 in Phase 1/2 clinical trials in Australia for retinitis pigmentosa associated with Usher syndrome and cystinosis. “Our proprietary NPI-001 has potential to address the myriad diseases and conditions where oxidative stress plays a role. EMA approval for the evaluation of NPI-001 tablets for treatment of HCCAA represents most importantly, a step forward in addressing HCCAA, a life threatening and underserved rare disease, and secondly, a key pharmaceutical quality milestone for the companies in the global development of NPI-001,” said G. Michael Wall, Ph.D., SVP & CSO, Nacuity. Nacuity Pharmaceuticals, Inc. (http://www.nacuity.com/), is a clinical-stage leader in innovative treatments for oxidative stress. The company’s powerful, targeted therapies aim to attentuate oxidative tissue damage, a driver of blinding eye diseases and a broad spectrum of serious chronic conditions. Nacuity has three highly differentiated clinical programs ongoing in retinitis pigmentosa, cataracts and cystinosis with the potential to be first-of-a-kind therapies and gateways to wider applications. Nacuity has operations in Fort Worth, TX, USA, and Australia, and extensive managerial and scientific domain expertise as well as backing from Foundation Fighting Blindness (https://www.fightingblindness.org/) and its venture arm RD Fund (https://www.retinaldegenerationfund.org). Arctic Therapeutics ehf (AT) (https://www.arctictherapeutics.com/) is an Icelandic drug development company, established in 2015 as a spin-off from the US-based Center for Applied Genomics (CAG), a research center at the Children's Hospital of Philadelphia. AT leverages the transformative power of applied genomics, steering the course of drug development towards safer, more effective treatments for some of the world’s most challenging diseases. The company has operations in Iceland, USA, and multiple collaborations across Europe. The content above comes from the network. if any infringement, please contact us to modify.

REYKJAVIK, Iceland and FORT WORTH, Texas, March 12, 2024 (GLOBE NEWSWIRE) -- Arctic Therapeutics ehf, an Icelandic drug development company and partner Nacuity Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other diseases caused by oxidative stress, today announced approval by the European Medicines Agency (EMA) for initiation of the first clinical trial evaluating AT-001 (Nacuity’s NPI-001) for the treatment of Hereditary Cystatin C Amyloid Angiopathy (HCCAA) in Iceland. HCCAA is an ultra rare, serious condition characterized by the accumulation of amyloid in the brain's blood vessels, which can lead to cerebral hemorrhage, stroke, and progressive neurological deterioration, including dementia. Current treatment options are severely limited, focusing primarily on symptom management rather than addressing the underlying cause of the disease. AT-001 is a differentiated, purified, prescription-quality, GMP-grade N-acetylcysteine amide (NACA). “EMA's approval paves the way for a comprehensive investigation into the safety and efficacy of a novel therapeutic approach designed to halt or reverse the progression of HCCAA. Our study aims to explore the potential of AT-001 in modifying the disease's course, improving patient outcomes, and ultimately, saving lives,” said Ivar Hakonarson, CEO and co-founder of Arctic. In September 2022, Nacuity and Arctic entered into an exclusive licensing agreement and strategic partnership for the development and commercialization of Nacuity’s NPI-001 under the name AT-001 for HCCAA in Iceland and related forms of amyloid angiopathies (CAAs) worldwide. Nacuity is also currently evaluating NPI-001 in Phase 1/2 clinical trials in Australia for retinitis pigmentosa associated with Usher syndrome and cystinosis. “Our proprietary NPI-001 has potential to address the myriad diseases and conditions where oxidative stress plays a role. EMA approval for the evaluation of NPI-001 tablets for treatment of HCCAA represents most importantly, a step forward in addressing HCCAA, a life threatening and underserved rare disease, and secondly, a key pharmaceutical quality milestone for the companies in the global development of NPI-001,” said G. Michael Wall, Ph.D., SVP & CSO, Nacuity. About Nacuity Pharmaceuticals, Inc. Nacuity Pharmaceuticals, Inc. ( ), is a clinical-stage leader in innovative treatments for oxidative stress. The company’s powerful, targeted therapies aim to attentuate oxidative tissue damage, a driver of blinding eye diseases and a broad spectrum of serious chronic conditions. Nacuity has three highly differentiated clinical programs ongoing in retinitis pigmentosa, cataracts and cystinosis with the potential to be first-of-a-kind therapies and gateways to wider applications. Nacuity has operations in Fort Worth, TX, USA, and Australia, and extensive managerial and scientific domain expertise as well as backing from Foundation Fighting Blindness ( ) and its venture arm RD Fund ( ). About Arctic Therapeutics ehf Arctic Therapeutics ehf (AT) ( ) is an Icelandic drug development company, established in 2015 as a spin-off from the US-based Center for Applied Genomics (CAG), a research center at the Children's Hospital of Philadelphia. AT leverages the transformative power of applied genomics, steering the course of drug development towards safer, more effective treatments for some of the world’s most challenging diseases. The company has operations in Iceland, USA, and multiple collaborations across Europe. Nacuity Media Contact Julia Clements Tel: 267.626.1085 jclements@6degreespr.com Arctic Media Contact Gulli Arnason gulli@arctictherapeutics.com

The cell and gene therapy market is forecasted to be worth $81bn by 2029, as per GlobalData’s sales and forecast database. Image Credit: JHVEPhoto / Shutterstock. Novartis has signed a collaboration and capsid license agreement with Voyager Therapeutics to develop gene therapies for Huntington’s disease and spinal muscular atrophy (SMA). Voyager will receive $100m in upfront payment, with $20m in equity. The US-based company will also be in line to receive up to $1.2bn in milestone-based payments and tiered royalties on global net sales, as per a 2 January press release. Following the news, Voyager stock was up by 24.8% when the market opened on 2 January, compared to the market close on 29 December 2023. The company’s market cap stands at $395.96m today (3 January), with the funds from the current deal expected to extend the company’s runway into mid-2026. The partnership leverages Voyager’s TRACER technology – tropism redirection of adeno-associated virus (AAV) by cell-type-specific expression of ribonucleic acid (RNA). The TRACER capsid discovery platform allows for the discovery of AAV capsids, which can cross the blood-brain barrier and target the central nervous system while de-targeting the liver and dorsal root ganglia. The companies have collaborated in the past, with Novartis signing a licensing option agreement with Voyager in March 2022. The agreement allowed Novartis access to Voyager’s technology to develop AAV capsids for use against diseases that affect the central nervous system and the option for two additional targets. Under the new deal, Novartis will have target-exclusive access to Voyager’s TRACER capsids related to SMA. It will also be responsible for the development and commercialisation of the SMA gene therapy. Novartis’ current portfolio contains an SMA gene therapy, Zolgensma (onasemnogene abeparvovec). The therapy was approved by the US Food and Drug Administration (FDA) for treating type 1 SMA in 2019. Following deal closure, Novartis will also gain global rights to Voyager’s AAV gene therapy for Huntington’s. Novartis will bear responsibility for the clinical development and commercialisation of the gene therapy whilst Voyager will be responsible for preclinical advancement. Novartis has invested in expanding its gene therapy portfolio. In May 2023, it acquired AVROBIO’s haematopoietic stem cell (HSC) gene therapy programme , designed to treat cystinosis. In July 2023, Novartis acquired San Diego-based preclinical stage biotech DTx Pharma for $1bn. DTx Pharma specialises in small interfering RNA (siRNA) therapies for central nervous system and neuromuscular indications. The cell and gene therapy market is forecasted to be worth $81bn by 2029, as per GlobalData’s sales and forecast database. GlobalData is the parent company of Pharmaceutical Technology . Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. By Cytiva Thematic By downloading this case study, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy