BrainStorm Cell Therapeutics Inc. will present new biomarker data suggesting that ALS patients may benefit from longer-term treatment with debamestrocel (NurOwn®). The Company will share the data with an international audience of patient advocacy groups, physicians, research organizations, industry representatives, key thought leaders and decision makers dedicated to ALS research at The 3rd Annual ALS Drug Development Summit, to take place May 21 to 23, 2024 in Boston MA.Stacy Lindborg PhD will deliver a presentation on new biomarker data from the NurOwn Expanded Access Program (EAP) along with data from the Phase 3 trial. Highlights A fixed sample of participants in the Phase 3 NurOwn trial, who met eligibility criteria, had the opportunity to enroll in an FDA approved Expanded Access Program (EAP).

The EAP was conducted over two periods of 28 weeks each, during which participants could receive a total of 6 doses of NurOwn, 3 doses of NurOwn in each period. All participants in the EAP received NurOwn, including participants randomized to placebo in the Phase 3 trial. 13 Cerebrospinal fluid (CSF) samples were drawn during Phase 3 trial and the EAP.

Levels of neurofilament light chain (NfL) in CSF were monitored during Phase 3 and subsequent EAP periods. NfL is an important biomarker marker in ALS, which measures neurodegeneration and neural cell death. NfL values has been shown through published studies to be associated with clinical progression, and treatment driven reductions in NfL were the basis for a recent ALS drug approval.

Recently published NfL data from the Phase 3 trial showed that participants treated with NurOwn had an 11% decline from baseline in NfL. Participants randomized to placebo had NfL values similar to baseline across the trial. (Lindborg et al.

Muscle and Nerve 2024). 10 trial participants who completed Phase 3 were enrolled in the EAP. 8 participants completed EAP period 1, and 6 completed EAP period 2. The participants in the EAP had lower NfL values at Phase 3 baseline as compared to the entire Phase 3 population.

The new EAP data to be presented at the ALS Summit showed that, for participants randomized to NurOwn, there was a 4% decrease from baseline in NfL in Phase 3 and a 27% and 36% decrease from baseline, at the ends of Period 1 and Period 2 of the EAP, respectively. These results suggest continual benefit from extended treatment of NurOwn. For participants randomized to placebo and subsequently treated with NurOwn in the EAP, there was a 37% increase in NfL from baseline to study end in Phase 3. Following treatment with NurOwn in the EAP, the same patients had a 17% increase in NfL from baseline during Period 1 and a 5% decrease from baseline in NfL during Period 2. BrainStorm hopes to confirm these results in the planned Phase 3b trial of NurOwn.