OrphAI Therapeutics Announces Oral Presentation and Posters at the 22nd Annual NEALS Conference

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.

Oral Presentation:

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


Poster Presentations:

·        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