GUILFORD, CT, October 2, 2023 —OrphAI Therapeutics Inc., a clinical-stage biopharmaceutical company developing novel therapeutics for rare diseases, announced today data presentations at the 22nd Annual NEALS (Northeast ALS Consortium) meeting being held October 4 - 6, 2023 in Clearwater Florida.
October 5, 2023, 11:10 a.m. ET
· Title: Results of the Phase 2a clinical trial of LAM-002A (apilimod dimesylate) inC9orf72 ALS
Presenters: Dr. Suma Babu, Sean M. Healey Center for ALS at MGHl; Peter Young, Ph.D. Chief Scientific Officer, OrphAI Therapeutics.
Location: Opal Ballroom
· Title: AIT-101 Improves Functional Deficits in a Human TDP-43 Animal Model of ALS
Presenter: Peter Young, Ph.D., Chief Scientific Officer, OrphAI Therapeutics
Location and Time: Poster Session 1, Abstract: 4, October 4, 2023, 5:15p – 7:15pET
· Title: Results of a double blind, placebo-controlled clinical trial of AIT-101(LAM-002A) in C9ORF72 ALS- A biomarker driven Phase 2a clinical trial targetingPIKfyve inhibition
Presenter: Dr. Suma Babu, Sean M. Healey Center for ALS at MGH
Location and Time: Poster Session 2, Abstract: 136, October 5, 2023, 4:30p -6:30p ET
About OrphAI Therapeutics
OrphAI Therapeutics’ mission is to transform the lives of patients with rare diseases. The company is currently developing three investigational therapies across multiple orphan indications: AIT-101, a first-in-class PIKfyve inhibitor, which recently completed a Phase 2a clinical trial in amyotrophic lateral sclerosis(ALS); LAM-001, a proprietary inhaled form of rapamycin, currently in Phase 2for bronchiolitis obliterans syndrome (BOS) and pulmonary arterial hypertension(PAH); and AIT-102, a proprietary analogue of mithramycin, in preclinical development for the treatment of SWI/SNF mutated or dysregulated tumors. To learn more, please visit: OrphAI-Therapeutics.com