ADDED
As of March 24, 2026, there were 24,939,948 shares of the registrant s Common Stock outstanding.
This number does not include 64,053,335 shares of Common Stock issuable upon the exercise of pre-funded warrants outstanding as of March 24, 2026 (which are immediately exercisable at an exercise price of $0.001 per share of Common Stock, subject to beneficial ownership limitations).
You should review the factors and risks and other information we describe herein and in the other reports we file from time to time with the SEC (as defined below).
BUSINESS Overview We are a clinical-stage biotechnology company focused on the discovery, research and development of innovative treatments for RASopathies, MAPK pathway-driven tumors, and other diseases, including central nervous system (CNS) disorders.
Our Therapeutic Pipeline We are advancing a pipeline of two therapeutic product candidates, with a focus on our lead product candidate, PAS-004, a next-generation macrocyclic (as defined below) Mitogen-Activated Protein Kinase ( MEK ) inhibitor, that we believe may address the limitations and liabilities associated with existing drugs with a similar mechanism of action.
Food and Drug Administration (the FDA ) are marketed for a range of diseases, including (i) certain cancers and (ii) symptomatic, inoperable NF1-associated plexiform neurofibromas ( NF1-PN ).
For NF1-PN, Koselugo (selumitinib) and Gomekli (mirdametinib) are FDA approved for adult and pediatric NF1-PN patients.
We believe that current FDA-approved MEK inhibitors have certain limitations, including known toxicities and high rates of adverse events ( AEs ) that may lead to dose interruptions and/or discontinuations and poor tolerability.
Unlike currently FDA-approved MEK inhibitors, PAS-004 features a macrocyclic structure, a characteristic that we believe improves selectivity, provides higher oral bioavailability, and offers better metabolic stability.
PAS-004 s macrocyclic design was specifically developed to improve metabolic stability and optimize its pharmacokinetic ( PK ) profile.
REMOVED
As of March 20, 2025, there were 2,705,263 shares of the registrant s Common Stock outstanding.
You should review the factors and risks and other information we describe in the reports we will file from time to time with the SEC.
iii EXPLANATORY NOTE On December 28, 2023, our board of directors ( Board ) approved a 1:20 reverse stock split (the Reverse Stock Split ) of our outstanding shares of common stock, par value $0.0001 per share ( Common Stock ), which became effective on January 2, 2024.
In connection with the Reverse Stock Split, there was a corresponding reduction in the number of authorized shares of Common Stock to 100,000,000.
Unless otherwise noted, the share and per share information in this Annual Report on Form 10-K reflects the Reverse Stock Split.
BUSINESS Overview We are a clinical-stage biotechnology company focused on the discovery, research and development of innovative treatments for central nervous system (CNS) disorders and other diseases, including RASopathies and certain cancers.
During the year ended December 31, 2023, we discontinued our support services to anti-depression clinics in the U.K.
In addition, we discontinued our clinical operations in Los Angeles, CA and disposed of the related property.
Accordingly, as of December 31, 2024, we currently have one reportable segment, Therapeutics, related to the research and development of our therapeutic product candidates.
Our Therapeutic Pipeline We are advancing a pipeline of three therapeutic product candidates, with a focus on our lead product candidate, PAS-004, a next-generation macrocyclic (as defined below) mitogen-activated protein kinase, or MEK inhibitor, that we believe may address the limitations and liabilities associated with existing drugs with a similar mechanism of action.