LRMR

• Positive 25-milligram and 50-milligram data from the long-term open-label study for Friedreich's ataxia patients, showing increased tissue frataxin and clinical outcome improvements. - Safety: 7 participants had anaphylaxis, most on initial administration day; dosing regimen modified with 5-milligram test dose, antihistamines, and EpiPen for participants. - Development program: Amending open-label study protocol to include adolescents and adults not in prior studies; global Phase III study in progress, qualifying sites globally. - Clinical outcomes: Compared to FACOMS reference population, observed 2.25 point improvement in mFARS over 1 year vs. 1 point worsening in reference, and improvements in other measures.

• Targeting BLA submission in the second quarter of 2026, seeking accelerated approval. - Confident in nomlabofusp's potential as a disease-modifying therapy for Friedreich's ataxia.

The company's lead program is nomlabofusp for Friedreich's ataxia. Positive data from a long-term open-label study showed consistent directional improvements in key clinical outcome measures like mFARS, FARS ADL, 9-hole PEG test, and MFIS. Skin frataxin levels increased, with 100% of participants at 6 months achieving over 50% of healthy volunteer levels. No traditional product segment revenue contribution discussed as it's a clinical stage biotech focused on one program.

• Anaphylaxis events in 7 participants, with most occurring on initial administration; risk of allergic reactions and need to consider in context of the disease. - Uncertainties in clinical trial data interpretation, success, cost, and timing of product development activities; ability to raise capital.

Q: How does the data at 14 patients 6 months compare to historical control?

A: Can't compare at 6 months as FACOMS data is annual; best comparison at 1 year.

Q: Color on severity of anaphylaxis?

A: Treatment is standard of care (epinephrine, antihistamines, steroids), patients respond quickly.

Q: Confidence in titration dosing strategy?

A: Confident, with test dose and expert consultants; data shows patients still willing to enroll despite events.

Q: Details on 7 anaphylaxis events?

A: Most in previously dosed patients, not age-related; FDA agreed with new dosing regimen.

Q: Benefit of prophylactic antihistamines?

A: Test dose may help tie up receptors on mast cells.

Q: Rate and severity of anaphylaxis vs other ERTs?

A: Benign safety profile except anaphylaxis; differences in baseline characteristics of those who had vs didn't have anaphylaxis.

Q: Patient number data and anaphylaxis with urticaria?

A: Patient numbers related to those who received at least 1 dose; 3 urticaria cases discontinued early.

Q: mFARS data and baseline values?

A: Baselines similar to reference population; updates to be provided closer to BLA submission.

Q: Impact on confirmatory study powering?

A: Ambulatory patients have more change potential, but conservative approach to powering in Phase III.