Many patients with peripheral nerve diseases do not have a sufficient regenerative response because of genetic inheritance, infections or chronic disease, leading them to lifelong pain and disability.
Based on positive phase III study data, Applied Therapeutics Inc. plans to take its CNS-penetrant aldose reductase inhibitor to the U.S. FDA to talk about an NDA for treating the rare disease sorbitol dehydrogenase (SORD) deficiency. Interim data from 12 months of treatment showed govorestat (AT-007) hit the study’s primary endpoints along with several key secondary endpoints. The double-blind, placebo-controlled registrational study of patients ages 16 to 55 is ongoing, with another 12 months of data yet to come. SORD, a hereditary axonal neuropathy created by sorbitol dehydrogenase gene mutations, affects about 3,300 people in the U.S. and about 4,000 in Europe, according to Applied Therapeutics.
South Korea’s Chong Kun Dang Pharmaceuticals Corp. said that it struck a deal potentially worth $1.3 billion with Swiss pharma giant Novartis AG for its CKD-510 candidate for neurological and cardiovascular diseases, propelling its shares upward 26.11% by market closing of Nov. 6. With the “largest ever” deal in its history, shares of the Seoul-based pharmaceutical (KOSPI:185750) on the Korea Exchange rose by 26.11%, or ₩26,500, closing at ₩128,000 ($98.70).
South Korea’s Chong Kun Dang Pharmaceuticals Corp. said that it struck a deal potentially worth $1.3 billion with Swiss pharma giant Novartis AG for its CKD-510 candidate for neurological and cardiovascular diseases, propelling its shares upward 26.11% by market closing of Nov. 6. With the “largest ever” deal in its history, shares of the Seoul-based pharmaceutical (KOSPI:185750) on the Korea Exchange rose by 26.11%, or ₩26,500, closing at ₩128,000 ($98.70).
At the 2023 Annual Congress of the European Academy of Neurology, Mary Reilly described the relationship between bench and bedside as “a continuous circle of translation,” with each cycle beginning with patients and their needs.
It seems unlikely that American poet and civil rights activist Maya Angelou spent much time thinking about translational research. But two quotes of hers capture the essence of the interplay between bench and bedside: “I did then what I knew how to do. Now that I know better, I do better” and “I’ve learned that I still have a lot to learn.” At the 2023 Annual Congress of the European Academy of Neurology, Mary Reilly described the relationship between bench and bedside as “a continuous circle of translation,” with each cycle beginning with patients and their needs.
Vanda Pharmaceuticals Inc.'s antisense oligonucleotide (ASO) VCA-894A has been awarded orphan drug designation by the FDA for the treatment of Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), caused by cryptic splice site variants within IGHMBP2.
Charcot-Marie-Tooth disease (CMT) is a group of neuropathies characterized by sensory and motor dysfunction that progress with aging. It is considered that about 60% of the axonal forms of the disease, such as CMT2, remain genetically undiagnosed.
Oryzon Genomics SA has nominated ORY-4001, a selective histone deacetylase 6 (HDAC6) inhibitor, as a clinical development candidate for the treatment of certain neurological diseases, including Charcot-Marie-Tooth (CMT), amyotrophic lateral sclerosis (ALS) and others.
