ADDED
As of March 17, 2026, the registrant had 103,882,937 shares of Common Stock, $0.001 par value per share, outstanding.
You should understand that the following important factors could affect our future results and could cause those results or other outcomes to differ materially from those expressed or implied in our forward-looking statements: uncertainties in obtaining successful non-clinical or clinical results that reliably and meaningfully demonstrate safety, tolerability and efficacy profiles that are satisfactory to the U.S.
As of December 31, 2025, we had $136.9 million of cash, cash equivalents and marketable securities.
Together with the $107.6 million in net proceeds from our recently completed February 2026 public offering, we expect this will fund operations into the second quarter of 2027.
We are targeting to submit a BLA to the FDA in June 2026 for marketing approval of nomlabofusp using the accelerated approval pathway; however, there can be no assurance that the FDA will approve our BLA submission for accelerated approval.
Moreover, if FDA grants accelerated approval of nomlabofusp, there is no assurance that the FDA ultimately will grant traditional approval or that the accelerated approval will not be withdrawn.
and foreign laws, regulations, and rules, contractual obligations, industry standards, policies and other obligations related to data privacy and security laws.
Our actual or perceived failure to comply with such obligations could lead to regulatory investigations or actions; litigation; fines and penalties; disruptions of our business operations; reputational harm; loss of revenue or profits; and other adverse business consequences.
Our lead product candidate, nomlabofusp, is a subcutaneously administered, recombinant fusion protein intended to deliver frataxin ( FXN ), an essential protein, to the mitochondria of patients with Friedreich's ataxia ( FA ).
As of December 31, 2025, we had cash, cash equivalents, and marketable securities of $136.9 million, which, together with the net proceeds of $107.6 million from our February 2026 public offering of common stock, we anticipate will fund operations into the second quarter of 2027.
REMOVED
As of March 20, 2025, the registrant had 64,027,892 shares of Common Stock, $0.001 par value per share, outstanding.
As of December 31, 2024, we have $183.5 million of cash, cash equivalents and marketable securities which we expect will fund operations into the second quarter of 2026.
and foreign laws, regulations, and rules, contractual obligations, industry standards, policies and other obligations related to data privacy and security laws, Our actual or perceived failure to comply with such obligations could lead to regulatory investigations or actions; litigation; fines and penalties; disruptions of our business operations; reputational harm; loss of revenue or profits; and other adverse business consequences.
Our lead product candidate, nomlabofusp (nomlabofusp is the International Nonproprietary Name ("INN") and the United States Adopted Name ("USAN") for CTI-1601), is a subcutaneously administered, recombinant fusion protein intended to deliver tissue frataxin ("FXN"), an essential protein, to the mitochondria of patients with Friedreich's ataxia ( FA ).
As of December 31, 2024, we had cash, cash equivalents and marketable securities of $183.5 million, which we anticipate will fund operations into the second quarter of 2026.
In May 2021, we reported positive top-line data from our Phase 1 FA program after completing dosing of the single ascending dose ("SAD") trial in December 2020 and of the multiple ascending dose ("MAD") trial in March 2021.
Data from these trials demonstrated proof-of-concept by showing that daily subcutaneous injections of nomlabofusp for up to 13 days resulted in dose-dependent increases in FXN levels from baseline compared to placebo in all evaluated tissues (buccal cells, skin, and platelets).
There were no serious adverse events associated with either the MAD or SAD trials.
In May 2023, we reported preliminary unblinded top-line data from the 25 mg cohort of our Phase 2 four-week, placebo-controlled, dose exploration trial of nomlabofusp in FA patients.
Data from the cohort indicated nomlabofusp was generally well tolerated and showed increases in FXN levels from baseline compared to placebo in all evaluated tissues (skin and buccal cells) at day 14.