SP-420

• Updated list of active sites (correct as of 5 September 2024):

Source: Company correspondence (30 August 2024)

Update: 30 June 2024

• Updated list of active sites (correct as of 19 June 2024):

Source: Company correspondence (19 June 2024)

Update: 31 March 2024

• Updated list of active sites (correct as of 16 April 2024):

Source: Company correspondence (7 March and 26 April 2024)

Update: 20 December 2023

• Trial overview: A new medicine called SP-420 is being studied through an open-label, dose-escalation, dose-finding, and proof-of-concept trial (THAL-01) in adult patients with transfusion-dependent ß-thalassemia.

SP-420 is an iron chelator, taken orally in capsules three times a week (Monday, Wednesday and Friday).

No participants receive a placebo in this trial.

The THAL-01 trial will establish if SP-420 is safe, works well, and doesn’t cause problems for patients. The trial has two main goals:

• To see how much SP-420 you should take (the right dose).
• To see if SP-420 is safe and helps patients with this condition.

In this trial, more than 10 countries and about 90 patients will be involved. Patients will be divided into three groups, each with around 30 patients. Before all patients are given the medicine, the safety of the treatment will be checked for the first 6 patients of each group through tests like heart monitoring, urine testing, and blood sampling to make sure it is safe.

How long is the trial?

The THAL-01 trial will last approximately 52 weeks and includes:

• A screening period of up to 4 weeks, where it is confirmed if a patient can go into the trial or not
• A treatment period of 48 weeks

Key Eligibility Criteria

To participate in the THAL-01 trial, patients must:

• Be at least 18 years of age
• Have transfusion-dependent ß-thalassemia
• Be on a stable dose of iron chelation for at least 4 weeks before screening
• Weigh ≥35 kg at screening
• Be willing to stop current iron chelation therapy 7 days (±3 days) before the first dose of SP-420 and for the entire duration of the trial
• Have transfusion iron overload
• Have been treated and followed for at least the past 6 months in a specialized center that maintained detailed medical records, including transfusion and iron chelation histories

Potential risks to patients

All the possible side effects of this trial medicine are not known at present. Like all medicines, the trial medicine can cause side effects, although not everybody gets them. Most side effects are mild to moderate. However, some people may experience serious side effects and may require treatment. A full overview of the potential risks will be provided during the screening period. All patients in the trial will be closely monitored during the trial.

Potential benefits to patients

Direct benefit from participating in the THAL-01 trial may not be guaranteed, however, possible benefits from taking part in this trial may include:

• Improved medicine options for the treatment of patients with transfusion-dependent ß-thalassemia
• Medical assessments, trial medication, and follow-up care
• Updated list of active sites (correct as of 12 Dec):

Source: Company correspondence (12 December 2023)

Update: 30 September 2023

• No update available.

Update: 30 June 2023

• No update available.

Update: 31 March 2023

• No update available.