Orphan Drug Weekly. Tau protein targets antibodies, and innovative therapies for pancreatic cancer are shortlisted.

Last week the FDA awarded six orphan drug qualifications, including Tauprotein monoclonal antibody therapy for rare brain diseases and "old-fashioned" therapy for pancreatic cancer, and today the drug Mingkang content team will take stockdrug: MPrecisionMed:P the research and development company of Msulfur
us acidfor the treatment of diseases: Pulmonary arterial hypertensionprofile: imatinib mesylate for imatinib, a small molecular inhibitor of tyrosine, an anti-tumor activityThe drug inhibits the activity of BCR-ABL, PDGFR, and c-KIT protein kinasesThese protein kinase-mediated signaling pathways are thought to be associated with the progression of pulmonary hypertensionImartinib has been used in clinical trials as an additional therapy for patients with pulmonary hypertensiondrug: Huperzine A (SPN-817)research and development: Supernus Pharmaceuticalstreatment of: Lennox-Gastaut syndrome Profile : Lennox-Gastaut syndrome (LGS) is a severe epileptic encephalopathy, accounting for about 1% to 2% of all epilepsy patients, mostly in childhood 65% to 75% of LGS patients have identifiable factors such as hereditary, structural and metabolic Huperzine A is a small molecule inhibitor of acetylcholinesterase, representing a new anticonvulsive mechanism The drug, which easily passes through the blood-brain barrier, increases levels of acetylcholine in the cerebral cortex and causes elevated levels of gamma-aminobutyric acid (GABA) in the cerebral cortex region, which can act as a suppression of seizures Its activity has been validated in preclinical models of partial seizures and Dravet syndrome drug :P NT001 research and development company :P inteon Therapeutics treatment of disease : introduction to the of nuclear paralysis : sexual cerebral palsy (PSP) is a rare brain disease that affects movement, balance and gait control, speech, swallowing, mood and behavior Symptoms usually appear in the patient's 60s, slightly more men than in women Pinteon Therapeutics developed PNT001 as a monoclonal antibody that can be precisely targeted and bindtod to the cis-pT231 epitope of the Tau protein Tau proteins can be folded into different conformations Cis-p-tau is one of these highly neurotoxic conformations The spread of cis-p-tau and cis-p-tau and neurofibrosy of acute release is considered to be a potential driver of neuronal degeneration observed in traumatic brain injury (TBI), chronic traumatic encephalopathy (CTE), Alzheimer's disease (AD) and ongoing nuclear paralysis (PSP) this highly toxic cis-p-tau conformation exposes a binding site called cis-pT321d Preclinical data show that when antibodies bind to the epitopic, they block the acute and long-term effects of toxic tau and have the potential to improve the quality of life in patients with neurodegenerative diseases Drug : Geranylgeranylacetone Research and Development : RNR BioMedical Treatment of Disease : Prevention
of Liver Failure after liver resection
: Geranylgerineusetone is an alcoholic polyoprene compound with a virocytic acid skeleton that has been developed as an oral anti-ulcer drug in Japan It has the ability to protect the gastric mucous membranes from various stress-inducing injuries and is also a powerful inducing agent of the heat shock protein (HSPs) Heat shock proteins are induced by various environmental or physiological stresss (e.g heat, hypoxia, ischemia, and infection) within cells and play a key role in providing cell protection Last week, the U.S FDA just issued its orphan drug for the prevention of acute radiation syndrome drug : proglumide research and development agency : Georgetown University treatment of disease : Pancreatic cancer profile : proglumide is an oral active gallbladder constrictor (CCK)-A/B receptor antagonists The drug can selectively block the role of CCK, has the ability to inhibit gastric fluid secretion and protect the gastric duodenum mucosa, but also has anti-epileptic and antioxidant activity The drug, once developed to treat stomach ulcers, has been tested for safety in humans Researchers at Georgetown University found in animal models that blocking CCK receptors reduces the growth of pancreatic cancer tumors, reduces fibrosis in the tumor microenvironment, and alters immune cell immersion to make tumors more sensitive to other therapies They plan to conduct Phase 1 clinical trials to explore the safety and dose of the drug as an anti-fibrosis treatment for pancreatic cancer patients drug : tacrolimus research and development : TFF Pharmaceuticals treating disease : Prevention of lung transplant rejection
Profile : The drug is an inhaled dry powder for his croms, an immunosuppressive drug for solid organ transplants Developed with TFF's thin-film freezing platform, TFF's process produces "brittle matrix particles" with low heap density (bulk density), high surface area and typical amorphous form, allowing particles to oversaturate contact target sites (e.g lung tissue) to improve the solubility and absorption of the drug The company plans to begin a phase 1 clinical study in Australia this month on the word dose of the oxmoth inhaler, with multiple incremental dose studies starting in the third quarter of this year.