Oral Dual-Target Inhibition to Change AML Management

Lead asset, SKI-001, is an oral dual-target ENL-AF9(MLLT 1/3) & FLT3 inhibitor with cellular activity across multiple AML genotypes and the ability to combine with standard-of-care agents

Dual Biomarker Suppression

Pronounced effects on HOXA9, MEIS1 and MYC, genes, including KMT2A and NPM1, up-regulated and over-expressed in several AML patients

Rapid Blast Clearance

Substantial reduction/elimination of blast cells in peripheral blood, bone marrow and spleen

Mechanistic Onset

Immediate cell-cycle arrest, differentiation of blasts and apoptosis

Durable & Differentiated Efficacy

Pre-clinical data show potential for rapid onset of action, complete and durable responses and efficacy potentially superior to competitors

Favorable Safety Profile

Improved safety & tolerability profile, compared to standard-of-care, seen in toxicology studies

~20,000

New US AML Cases per Year

~40%

Menin Inhibitor Treated Patients Relapse

Peak sales opportunity

$1-2B

34%

5-Year Survival Rate

More Advanced Than Dark Blue's DBT-3757

Acquired by Amgen for $840M

AML Treatment Market by 2028

$10B