We hereby issue the following press release from CoA Therapeutics:

"CoA Therapeutics and BridgeBio Pharma present data from the Phase 1 study in healthy individuals to support the development of BBP-671 for PKAN and organic acidemias

On May 26, 2022, CoA Therapeutics and its parent company BridgeBio Pharma presented interim results from a Phase 1 study of BBP-671 in 77 healthy individuals. BBP-671, which is not approved by the FDA or any other regulatory agency, is an oral small molecule therapy that activates pantothenate kinase enzymes. BBP-671 is being developed as a potential therapy for diseases associated with coenzyme A (CoA) deficiency, including pantothenate kinase-associated neurodegeneration (PKAN), propionic acidemia and methylmalonic acidemia.
In phase 1 trials, researchers test a new potential therapy on healthy individuals to gather information on how a drug interacts with the human body. This early information is used in future clinical trials. In the study, BBP-671 was generally well tolerated by the healthy volunteers. No serious side effects occurred. The most common adverse events associated with BBP-671 administration were headache (3 subjects), neutropenia (3 subjects), abdominal pain (1 subject) and nausea (1 subject). In people who experienced neutropenia, i.e. a decrease in white blood cell counts, white blood cell counts returned to normal within 3 days of stopping BBP-671.
BBP-671 has been detected in blood and cerebrospinal fluid, the fluid that surrounds the brain, suggesting that BBP-671 can cross the blood-brain barrier and reach the brain.
In healthy people, CoA is produced in the body by pantothenate kinase enzymes, which break down pantothenate (vitamin B5) to produce CoA and acyl-CoAs, including acetyl-CoA. BBP-671 appears to increase acetyl-CoA levels and decrease pantothenate levels in the blood of healthy individuals administered BBP-671. These changes in biomarkers show for the first time in humans that BBP-671 acts as expected in the body.
Based on these data, CoA Therapeutics and its parent company BridgeBio Pharma plan to proceed with the second part of the Phase 1 clinical trial in people with propionic acidemia and methylmalonic acidemia in the second half of 2022 and to initiate a Phase 2/3 clinical trial in people with PKAN in 2023.
Further information on the ongoing Phase 1 clinical trial with BBP-671 (trial number NCT04836494) can be found at here on the ClinicalTrials.gov website. The scientific poster presented today can be found here.

About CoA Therapeutics
CoA Therapeutics was founded in 2018 with the goal of developing new therapies for difficult-to-treat and rare genetic diseases by leveraging advances in the understanding of the CoA pathway. CoA Therapeutics' approach and lead compounds are based on groundbreaking science led by Suzanne Jackowski, Charles Rock, Richard Lee and Stephen White at St. Jude Children's Research Hospital in Memphis, Tennessee. Our goal is to work with patients, patient advocates and physicians to bring effective therapies for PKAN, propionic acidemia, methylmalonic acidemia and other diseases to patients as quickly as possible. 1800 Owens Street, Suite C-1200 San Francisco, CA 94158

About BridgeBio Pharma
BridgeBio Pharma, Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded with the mission to discover, develop, test and deliver breakthrough medicines to treat patients with genetic diseases and cancers. BridgeBio's pipeline ranges from early-stage research to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com. ( ... )"

Translation with the help of Deepl.com/Translator (free version), slightly edited by AK (Hoffnungsbaum). CoA Therapeutics is responsible for the content of this press release.

The Californian company CoA Therapeutics is currently preparing a clinical trial with a new drug for PKAN patients. In order to make this study as patient-oriented as possible, CoA Therapeutics developed a survey last year in which PKAN patients and their families could make their voices heard. The company actively involved representatives of the international NBIA patient organizations in the preparations. Two representatives from Hoffnungsbaum e.V. based on their own experience as parents of PKAN children and as long-standing patient representatives, helped to prepare the survey in the best possible way and to make it known to the families. We are therefore very pleased that CoA Therapeutics is now presenting the survey results and would like to share this summary with you:

Summary of CoA Therapeutics' survey for people with PKAN

The team at CoA Therapeutics would like to thank the individuals and families affected by PKAN for their participation in the recent survey. We were very impressed by the level of community engagement with 183 responses received. We would especially like to thank our partners in patient organizations who commented on the survey and helped to disseminate it.

Key findings from the survey included information on the timing of disease onset in people with PKAN: half of respondents said that the first PKAN-related symptoms appeared before the age of 10, more than a third experienced the first symptoms at the age of 10 or later, and ten percent were not sure when the first symptoms appeared. Respondents came from many countries, most from Poland (21%), the Dominican Republic (18%), the USA (15%), Italy (9%), Spain (7%) and Germany (6%). Responses from affected families also came from 17 other countries, including France, Brazil, Canada, Cuba, Egypt, India, Iran, Jordan, Mexico, the Netherlands, New Zealand, Norway, Saudi Arabia, Slovenia, Switzerland, Ukraine and the United Kingdom.

The majority of people with PKAN reported being diagnosed before the age of 12 (59%), and most had a genetic test to confirm their PKAN diagnosis (81%). Respondents most commonly reported that problems with balance/falls, motor skills and muscles were the first PKAN symptoms they experienced.
The majority of respondents stated that they were taking several medications, with anticonvulsants, skeletal muscle relaxants and antipsychotics being mentioned most frequently. Only 22 people stated that they were not taking any medication. Approximately 1 in 4 respondents reported having a deep brain stimulation (DBS) device implanted and a similar proportion reported having a feeding tube.
When asked about the most difficult medical examinations, respondents stated that imaging procedures (e.g. X-ray, MRI, CT scans) were the most difficult, as they could not maintain the required posture, were tense and required sedation.
In an open-ended question about the top three symptoms that are most difficult/challenging for people with PKAN, dystonia (86 mentions), impact on speech (83 mentions) and difficulty eating (64 mentions) were most frequently mentioned. When asked to name the three most important symptoms that could hopefully be improved by a new treatment, the most frequently mentioned were effects on speech (87 mentions), dystonia (70 mentions) and difficulty eating and walking (47 mentions each).
About half of respondents reported having participated in a clinical trial in the past, and these individuals reported that travel and visits were the most difficult aspects of trial participation. Also, the most frequently cited ways to improve clinical trial participation were less travel/visits and better on-site visit experiences, such as more support at the clinical center, more comfortable admission conditions, and a place to rest.
Thank you again for your participation in our survey. Your experiences are invaluable to our work and directly help us as we work to develop a therapy for PKAN. As next steps, we are preparing a more detailed presentation to share with the community later this year. We at CoA Therapeutics are also committed to sharing these research findings with a broader audience to inform other scientists, clinicians and companies working to develop therapies for PKAN.

Translation from English with the support of https://www.deepl.com/translator, free version