BRAINSTORM CELL THERAPEUTICS INC. Q2 FY2023 earnings call

• Preparations for the FDA's Cellular Tissue and Gene Therapies Advisory Committee meeting on September 27 to review the NurOwn BLA, with a PDUFA date of December 8, 2023. • Focus on commercial preparedness, including manufacturing, commercial, and medical affairs activities to make NurOwn available to patients if approved. • Updates on the team, including promotions and new hires like Dr. Kirk Taylor as EVP and CMO, Dr. Bob Dagher as EVP and Chief Development Officer, and Nir Naor joining the board. • Discussion of biomarker data from the NurOwn Phase 3 trial, including analysis of disease co-variants and their impact on clinical outcomes, presented at the Gordon Research Conference. • Plans for a confirmatory trial for NurOwn, with input from a steering committee and consideration of FDA feedback.

Cash, cash equivalents and short-term bank deposits were approximately $0.75 million as at the end of June 2023, compared to approximately $3 million at the end of December 2022. Research and development expenses for Q2 2023 were approximately $2.8 million vs. $5.1 million in Q2 2022. General and administrative expenses for Q2 2023 were approximately $2.7 million vs. $2.5 million in Q2 2022. Net loss for Q2 2023 was approximately $5.3 million or $0.13 per share vs. a net loss of approximately $7 million or $0.19 per share in Q2 2022.

• Confidence in achieving a successful outcome from the ADCOM for NurOwn's BLA. • Intention to proceed with a confirmatory clinical trial for NurOwn, with input from the FDA and steering committee. • Continued focus on preparing for NurOwn's potential approval and commercialization.

• General risks associated with forward-looking statements, including uncertainties in regulatory reviews, clinical trial outcomes, and market acceptance.

Q: The submitted BLA for NurOwn specifically seeking full approval or accelerated approval?

A: The BLA filed is seeking full approval.

Q: Does the BLA include data collected from patients that have been treated under the Expanded Access Program and from the Israeli HE pathway?

A: Yes. Data from both programs were included in the BLA.

Q: Do you still intend to publish your biomarker manuscript?

A: Oh, absolutely. The biomarker data is a compelling part of our evidence, which provides strong support of the clinical data and we can confirm the papers on the review at a highly regarded journal.

Q: Why did you only recently decide to look at baseline characteristics of patients and make this presentation at Gordon? And did the results change substantially once you've counted the baseline characteristics?

A: This is an example of emerging science. The FDA did work exploring the importance of disease co-variants, and we prospectively specified co-variants in our efficacy analyses. The results from models with and without adjusting for co-variants were very similar, with the estimate of treatment effect having more precision.

Q: Do you intend to proceed with a confirmatory clinical trial?

A: Yes. We have definite plans to proceed with a confirmatory trial, having met with the steering committee and intending to share more after input from the FDA.

Q: Is there a reason that you're granted a different ADCOM from the ADCOM that oversaw the amyloid and Biogen drugs?

A: The designation of the advisory committee is determined by the FDA center the application is filed with. NurOwn was submitted to CBER, while others were to CDER.

Q: Have you been able to submit amendments to address the CMC items identified by the FDA in their initial RTF letter? And if so, or if not, do you anticipate any impact on the PDUFA date?

A: Yes, we have submitted amendments and the PDUFA date remains December 8, 2023.

Q: Have you been having any conversations directly with the FDA while the BLA has been under review?

A: Yes. We have regular interactions with the FDA, responding to requests for information and sharing presentations.

Q: You reported that around 25% of patients in your Phase 3 study had a baseline value below 25%. And in these patients for the decline could not be measured, because the items reached zero. You referred to this as the floor effect. Can you please expand on this floor effect? And do you have any biomarker data for these participants?

A: The ALSFRS-R has limitations as a bounded scale. We looked at biomarker data in these participants and observed significant improvements on ALS biomarkers with NurOwn versus placebo across all trial participants.

Q: BrainStorm has said that it's working on the use of its product for other indications. Investors have been hearing about this now for years, please provide some specificity with respect to what working on it means.

A: We completed a Phase 2 study evaluating NurOwn for progressive MS and have a protocol concept for Alzheimer's disease. We are prioritizing resources for the ALS program but intend to move forward with other programs as resources allow.

Q: Did BrainStorm finalize on the follow-on offering with Maxim for $7.5 million?

A: Yes. We completed the registered direct offering, raising net proceeds of approximately $7 million in July 2023.

Q: About that ADCOM, do you expect, based on your co-variant analysis of the co-variants and what you've shown around NFL, particularly in that presentation in July, do you expect the same questions that Biogen had gotten where there was the vote on NFL 9 to 0, but there was some differences in the voting, three yes, three no on the full approval, I guess, requiring a confirmatory study. Do you expect a similar question?

A: This is a different ADCOM, center, application, and mechanism of action. We'll know the specific questions closer to the ADCOM.

Q: During that ADCOM as part of the presentation, will be made more of a point on the safety aspects of NurOwn, given that it is an autologous cell therapy, it's not genetically manipulated and then it is a far safer approach apparently than other drugs or ALS, including the Biogen drug?

A: We will present the full set of data, efficacy and safety, and provide a compelling overview of all data at our presentation.