UNC13A

New findings highlight impact of UNC13A genotype on treatment response in ALS NEW YORK, April 29, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, in collaboration with leading ALS research centers, today announced the acceptance of new pharmacogenomic data for oral presentation of new findings on NurOwn® at the International Society for Cell & Gene Therapy (ISCT) 2025 Annual Meeting, to take place from May 6 - 10, 2025 in New Orleans. The data highlight the impact of the UNC13A genotype on clinical outcomes for amyotrophic lateral sclerosis (ALS) patients treated with NurOwn® (debamestrocel). The presentation was featured in the ISCT public announcement, which indicated that the data were "carefully reviewed and selected by the ISCT 2025 Planning Faculty, to explore the latest breakthroughs in the clinical translation of Mesenchymal Stem/Stromal Cells and how they will shape the future of cell therapies." "We look forward to sharing the latest scientific data on NurOwn at this year's ISCT meeting. These new results are particularly meaningful as they explore a genetic factor that may help predict treatment response. The feedback received from the ALS experts on these new data continues to be encouraging as we advance in our mission to address the unmet needs of patients with this devastating disease," said Chaim Lebovits, President and CEO of BrainStorm. Mr. Lebovits added, "Our immediate priority is to launch the Phase 3b trial of NurOwn to build on our clinical data and demonstrate its potential in early ALS. We are enjoying positive communication with the FDA, have submitted an IND amendment, and are implementing the SPA exactly as agreed with the Agency. We're fully prepared to begin the study." Dr. Bob Dagher, Executive Vice President & Chief Medical Officer at BrainStorm, commented, "These latest results offer additional scientific insights into our completed Phase 3 NurOwn trial and increase our understanding of treatment effects in patient subpopulations. These types of analysis provide important information for the development of future ALS treatments and add to the growing body of evidence supporting NurOwn®." Dr. Dagher added, "This represents a first-of-its-kind pharmacogenomic analysis from the Phase 3 trial in ALS and is among the first ALS studies to analyze how genetic differences, specifically the UNC13A genotype, affect patient response to therapy. Understanding how genetics can influence treatment response will help move us closer to truly personalized medicine for ALS." Presentation details: Title: Debamestrocel Effect on Clinical and Biomarker Endpoints by UNC13A Genotype in Phase 3 ALS Trial Lead Author Bob Dagher, MD Oral Session: Mesenchymal Stem/Stromal Cells #1 Date and time: Date & Time: Thursday, May 8, 2025 | 9:00 – 10:00 a.m. U.S. Central Time. Location: Ernest N. Morial Convention Center, 900 Convention Center Boulevard, New Orleans, Louisiana 70130 A copy of the poster will be available on the BrainStorm corporate website, at the conclusion of the ISCT meeting. About Brainstorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) is a leading developer of autologous adult stem cell therapies for debilitating neurodegenerative diseases. The company's proprietary NurOwn® platform uses autologous mesenchymal stem cells (MSCs) to produce neurotrophic factor-secreting cells (MSC-NTF cells), designed to deliver targeted biological signals that modulate neuroinflammation and promote neuroprotection. NurOwn® is BrainStorm's lead investigational therapy for amyotrophic lateral sclerosis (ALS) and has received Orphan Drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). A Phase 3 trial in ALS (NCT03280056) has been completed, and a confirmatory Phase 3b trial is set to launch under a Special Protocol Assessment (SPA) agreement with the FDA. The NurOwn clinical program has generated valuable insights into ALS disease biology, including pharmacogenomic response associated with the UNC13A genotype, biomarker data collected at seven longitudinal time points, and a comprehensive analysis of the "Floor Effect" — a critical challenge in measuring clinical outcomes in advanced ALS. BrainStorm has published its findings in multiple peer-reviewed journals. In addition to ALS, BrainStorm has completed a Phase 2 open-label multicenter trial (NCT03799718) of MSC-NTF cells in progressive multiple sclerosis (MS), supported by a grant from the National MS Society. BrainStorm is also advancing a proprietary, allogeneic exosome-based platform designed to deliver therapeutic proteins and nucleic acids. The company recently received a Notice of Allowance from the U.S. Patent and Trademark Office for a foundational patent covering its exosome technology, further strengthening BrainStorm's growing IP portfolio in this emerging area of regenerative medicine. To learn more, visit . Notice Regarding Forward-Looking Statements This press release contains "forward-looking statements" that are subject to substantial risks and uncertainties, including statements regarding meetings with the U.S. Food and Drug Administration (FDA), Special Protocol Assessment (SPA), ADCOM meeting related to NurOwn, the timing of a PDUFA action date for the BLA for NurOwn, the clinical development of NurOwn as a therapy for the treatment of ALS, the future availability of NurOwn to patients, and the future success of BrainStorm. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "plan," "potential," "predict," "project," "target," "aim," "should," "will" "would," or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on BrainStorm's current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. These potential risks and uncertainties include, without limitation, management's ability to successfully achieve its goals, BrainStorm's ability to raise additional capital, BrainStorm's ability to continue as a going concern, prospects for future regulatory approval of NurOwn, whether BrainStorm's future interactions with the FDA will have productive outcomes, and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at . These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations, and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements. CONTACT: Michael Wood Phone: +1 646-597-6983 [email protected] Logo: SOURCE BrainStorm Cell Therapeutics Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Unlearn, an AI-enabled company creating digital twins of clinical trial participants, announced a partnership to advance ALS research with biotechnology company Trace Neuroscience, which creates genomic therapies for neurodegenerative diseases. Unlearn, founded in 2017, uses machine learning to create digital twins of clinical trial participants before being randomized in a controlled trial. The digital twin can provide researchers insight into participants' health outcomes. The company pitches its technology as a way to run smaller clinical trials more quickly since researchers can find fewer participants for the control group. Additionally, Unlearn offers a Digital Twin Generator (DTG) for ALS, a machine learning model trained on more than 13,000 longitudinal clinical records from the ALS-focused digital research platform APST, the PRO-ACT database for ALS research and the Northeast ALS Consortium (NEALS). Digital twin generators predict how an individual may progress under the standard of care or in the placebo group in a clinical trial, creating a "digital twin" for each trial participant based on their data. Trace Neuroscience will use Unlearn's DTG ALS and its Unlearn Platform for the biotech company's Phase 1/2 clinical trial, which targets the UNC13A protein, a genetic target directly linked to ALS progression. Unlearn's offerings will be used to simulate ALS disease progression and evaluate the relationship between clinical endpoints, disease status at baseline and biomarkers over time. Trace will also use Unlearn's platform to inform clinical trial protocol decisions. "This collaboration brings together two powerful approaches—AI and genomic medicine—to rethink how ALS trials are designed," Dr. Eric Green, cofounder and CEO of Trace Neuroscience, said in a statement. "Working with Unlearn to mine their extensive, well-curated database through the use of the ALS DTG will enable us to explore smarter designs and make confident and informed decisions as we plan our Phase 1/2 trial. Ultimately, these insights can help us to move faster for people living with ALS who are waiting for new treatment options." THE LARGER TREND Last year, Unlearn secured $50 million in Series C funding, bringing its total raise to more than $130 million. In 2022, the company scored $50 million in a Series B financing round two years after garnering $15 million in Series A funding. In December, Unlearn partnered with German-based APST Research (APST) to integrate data from over 8,000 participants in an APST longitudinal study into its DTG ALS. The dataset includes clinical data, biomarker analyses, common ALS clinical assessments and patient self-assessments. Unlearn announced a partnership last year with biotechnology company ProJenX, which would use Unlearn's DTG ALS to produce digital twins of clinical trial participants living with ALS. The digital twins would act as a placebo for trial participants dosed with prosetin—a brain-penetrant, MAP4 kinase inhibitor—in ProJenX's Phase 1 clinical trial. In 2023, Unlearn announced a partnership with clinical-stage biotechnology company QurAlis Corporation to speed up QurAlis' ALS-focused clinical program using Unlearn's genAI technology. Other companies using digital twin technology include Singaporean startup Mesh Bio, which uses digital twins to help manage rising cases of chronic diseases, particularly in Southeast Asia. Twin Health, a digital metabolic care company, offers its Whole Body Digital Twin, an AI-backed model aimed at providing individualized nutrition, sleep and activity guidance to help people prevent and reverse metabolic diseases like Type 2 diabetes.

Maze Therapeutics has submitted its pitch for an initial public offering after disclosing a $115 million Series D, forging a neuroscience spinoff and running into a dealmaking hurdle with the FTC. The Bay Area startup revealed its plans Tuesday night to trade on the Nasdaq as $MAZE . It has yet to disclose how much it could seek in the proposed offering. The startup’s IPO pitch follows a relatively dismal multiyear stretch for biopharma public debuts. Making its IPO desires known in the lead-up to next week’s annual JP Morgan Healthcare Conference will likely drive attention and be the center of conversations as industry insiders hope for more dealmaking and a more bustling public market. The 121-employee Maze had $149 million in cash and equivalents at the end of September, according to its prospectus. Third Rock Ventures, which launched Maze in 2019, is its biggest stockholder at 20%. ARCH owns nearly 12%, Matrix Capital holds about 7%, and Foresite and GV each have nearly 7% as well, according to the IPO paperwork. Its Nasdaq debut would arrive about eight months after Shionogi signed a deal for Maze’s Pompe disease asset, which is ready for Phase 2. The Japan-based partner is paying $150 million upfront and an undisclosed amount in biobucks for the small molecule. It was a quick turnaround for Maze, which was left blindsided by Sanofi’s decision to pull out of a licensing pact for the drug in late 2023 in reaction to scrutiny from the Federal Trade Commission, which had said it would move to block the deal. After the Sanofi deal ended, Maze received “a lot of inbound interest,” Maze CEO Jason Coloma told Endpoints News on the sidelines of JPM last January. Coloma said Maze would “have a better sort of guidance” on its next financing steps, including a potential IPO, once it inked a deal with a new Pompe partner. Beyond Pompe, the biotech is testing an oral small molecule, dubbed MZE829, as a potential treatment for APOL1 kidney disease. It entered Phase 2 in November, with data slated for the first quarter of 2026. Another small molecule, codenamed MZE782 for chronic kidney disease, entered Phase 1 in September and will have initial data in the second half of this year. Maze spent $61 million on research and development through the first nine months of 2024, only about $3.5 million more than the same period in 2023, according to its S-1 filing. The biotech recently shipped its UNC13A-directed antisense oligonucleotide to Trace Neuroscience, which emerged with $101 million in November to take on amyotrophic lateral sclerosis. The biotech has had mixed success with joint ventures in the past. Its ophthalmology-focused partner Broadwing Bio has been quiet in recent years, and its cardiovascular-centered JV with BridgeBio, named Contour Therapeutics, has since been unwound, Coloma previously told Endpoints . Maze’s founders include Verve Therapeutics CEO Sek Kathiresan, the Whitehead Institute’s Jonathan Weissman, Stanford’s Aaron Gitler, Harvard Medical School’s Stephen Elledge, and the Broad Institute’s Mark Daly.