Motor Neuron Disease

FDA Authorizes Expanded Access Program (EAP) to Help Provide Access to SPG302 for 200 ALS Individuals Ineligible for Clinical Study EAP Supports Real-world Data Collection Concurrent to Ongoing Clinical Studies of SPG302 LOS ANGELES, May 5, 2025 /PRNewswire/ -- Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients worldwide, today announced that it has received notification from the Food and Drug Administration (FDA) to begin its Expanded Access Program (EAP) for SPG302 in the United States (U.S.) for people living with Amyotrophic Lateral Sclerosis (ALS) that meet program eligibility criteria. SPG302 is being developed as the first synaptic regenerative approach to treating ALS, with the potential to slow or reverse declines in cognitive and motor function. Spinogenix CEO and Founder Dr. Stella Sarraf stated, "FDA clearance to launch this new EAP supports its awareness of SPG302's safety and potential efficacy, and the need for ALS patients to have access to novel treatments. This EAP reflects our dedication to giving hope to people living with ALS and their families, providing treatment to those who do not qualify for, or may be unable to participate in, our ongoing clinical trial. Innovation is urgently needed to tackle ALS, a disease with devastating impact, and we are committed to ensuring that no patients who may benefit are left behind." EAPs are designed to allow individuals with serious or life-threatening conditions to access investigational treatments outside of a clinical trial that are not yet approved by the FDA. Dan Doctoroff, founder and chairman of Target ALS, and the first individual to receive SPG302 in the U.S. as part of a separate EAP protocol commented, "The notion that ALS is an untreatable disease is outdated. My own experience with SPG302, the first synaptic regenerative therapy in clinical development, has shown that partial stabilization of the disease is a possibility. I am thrilled that the FDA has recognized the value of allowing Spinogenix to expand access to SPG302, allowing others battling ALS to have a similar beneficial experience." In addition to serving the ALS community by providing access to an innovative investigational therapy, the EAP enables the collection of real-world data on SPG302 safety and efficacy that can support its clinical development. The FDA previously granted SPG302 Orphan Drug Designation for the treatment of ALS. Spinogenix recently completed a Phase 2 ALS trial in Australia, and patients have been offered a continuation of treatment in an open label extension. Additional information on the trial (NCT05882695) and open label extension (NCT06903286) may be found on ClinicalTrials.gov. About SPG302 SPG302 is a once-a-day pill being developed as a regenerative treatment for neurodegenerative and neuropsychiatric diseases with the unique ability to restore synapses, the key connections between neurons that allow people to think, plan, remember, and control movement. The synaptic regenerative activity of SPG302 represents a first-in-class approach to treating these diseases and has the potential to reverse declines in cognitive, respiratory, and motor function. SPG302 has been granted U.S. FDA Orphan Drug Designation for the treatment of ALS and has received preclinical support from the U.S. National Institutes of Health and the Department of Defense. Spinogenix has completed a Phase 1/2 study in Australia (NCT05882695) assessing the safety, pharmacokinetics and pharmacodynamics of once-daily dosing of SPG302 in healthy volunteers and up to 48 weeks of treatment in ALS patients. Additional information on the global clinical trials evaluating SPG302 for the treatment of schizophrenia and Alzheimer's disease can be found on ClinicalTrials.gov (NCT06442462 and NCT06427668). About Expanded Access to SPG302 Access to SPG302 is available in the U.S. for certain adults with ALS who meet eligibility criteria for participation in the FDA-authorized Expanded Access Program for SPG302. More information about the U.S. EAP can be requested through [email protected]. About Spinogenix Current treatments for neurodegenerative, neuropsychiatric and neurodevelopmental conditions primarily focus on slowing disease progression or minimizing symptoms, leaving many without hope for improvement. Spinogenix is aiming to transform the treatment of these conditions through its pioneering first-in-class and paradigm-shifting synaptic regenerative and synaptic corrective therapeutics designed to restore depleted synapses and reverse synaptic degeneration and dysfunction – offering patients and their families a new reality of hope. Spinogenix is developing two novel therapeutics: SPG302, which triggers neurons to produce new glutamatergic synapses and restore cognitive, motor, and other functions in ALS, Alzheimer's disease, schizophrenia and other diseases; and SPG601, which works at the synaptic level to correct specific dysfunctions in Fragile X Syndrome (FXS) that underlie many core symptoms. The company has received FDA Orphan Drug designation for both ALS and FXS, as well as FDA Fast Track designation for FXS. More information on Spinogenix can be found at or follow us on LinkedIn. Media Relations Nechama Rosengarten FINN Partners [email protected] Investor Relations Dan Albosta Spinogenix, Inc. [email protected] SOURCE Spinogenix WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

DURHAM, N.C.--(BUSINESS WIRE)--May 1, 2025-- Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies, including gene elimination, gene insertion, and gene excision programs, today announced that five abstracts detailing Precision’s wholly owned or partnered programs have been accepted for presentation at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting being held May 13-17, 2025, in New Orleans, Louisiana. "We are pleased to present new data at ASGCT from multiple programs across our in vivo gene editing pipeline, highlighting the potentially unmatched breadth and therapeutic potential of our ARCUS gene editing technology,” said Michael Amoroso, President and Chief Executive Officer at Precision BioSciences. “Clinically, we have made progress advancing our lead program, PBGENE-HBV, which is a novel gene editing approach being evaluated in a first-in-human study for chronic hepatitis B, and we look forward to sharing initial safety data from the first two dose cohorts at ASGCT. Additionally, we continue to be excited by the promising clinical results generated thus far by our partner iECURE for ECUR-506 in OTC deficiency, which utilizes an ARCUS nuclease and will also be presenting data at this conference." “We are also excited to share promising preclinical results from two muscle programs, PBGENE-3243 program for m.3243-associated mitochondrial disease and PBGENE-DMD for the treatment of Duchenne Muscular Dystrophy (DMD),” Mr. Amoroso continued. “Collectively, these presentations highlight the progress we are making with ARCUS by deploying a variety of gene edit types, including gene elimination, gene insertion and gene excision across a range of complex genetic diseases as we seek to develop durable, curative treatments for patients." Clinical Program Presentations: Title: Initial Safety Data From ELIMINATE-B, the First Clinical Trial of a Gene Editing Treatment for Chronic Hepatitis B Oral Presentation Session: Gene Therapy Trials - In-Vivo Gene Therapy Modification Date and Time: Friday, May 16, 2025, 4:00 PM CT Location: Room 393-396 Title: ARCUS in vivo OTC, Large Gene Insertion (clinical stage partnered program) Oral Presentation Session: Advances in Genome Editing: Novel Large DNA Insertion Technologies and Their Potential Towards Curative Therapies Date and Time: Wednesday, May 14, 2025, 8:00 AM CT Location: NOLA Theater B Preclinical Program Presentations: Title: Excision of the C9orf72 Hexanucleotide Repeat Expansion Using a Dual-ARCUS Gene Editing Approach Reduces Neurotoxic RNA Foci and Dipeptides in an In Vivo Model of ALS Poster Session: Poster Reception Date and Time: Tuesday, May 13, 2025, 6:00 PM - 7:30 PM CT Location: Poster Hall I2 Title: Systemic Delivery of a Mitochondria-Targeting ARCUS Gene Editing Nuclease by AAV Eliminates Mutant Mitochondrial DNA, Demonstrating Therapeutically Meaningful Heteroplasmy Shifts In Vivo Oral Presentation Session: Gene Editing: New Tools and Technology Advances Date and Time: Wednesday, May 14, 2025, 3:45 PM CT Location: NOLA Theater A Title: ARCUS-Mediated Gene Editing Excision of Exons 45-55 of the Human Dystrophin Gene using PBGENE-DMD Leads to Functional Dystrophin Protein and Durable Restoration of Skeletal Muscle-Function In Vivo for the Treatment of Duchenne Muscular Dystrophy Poster Session: Poster Reception Date and Time: Wednesday, May 14, 2025, 5:30 PM - 7:00 PM CT Location: Poster Hall I2 The abstracts are now publicly accessible through the ASGCT website here. About Precision BioSciences, Inc. Precision BioSciences, Inc. is a clinical stage gene editing company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform that differs from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics may enable ARCUS nucleases to drive more intended, defined therapeutic outcomes. Using ARCUS, the Company’s pipeline is comprised of in vivo gene editing candidates designed to deliver lasting cures for the broadest range of genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit www.precisionbiosciences.com. The ARCUS® platform is being used to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion (inserting DNA into gene to cause expression/add function), elimination (removing a genome e.g. viral DNA or mutant mitochondrial DNA), and excision (removing a large portion of a defective gene by delivering two ARCUS nucleases in a single AAV such as in the DMD program). Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the therapeutic potential of ARCUS compared to other gene editing technologies; clinical development and expected safety, efficacy and benefit of our wholly owned gene editing product candidates PBGENE-HBV, PBGENE-DMD, PBGENE-3243, and PBGENE-CNS programs and those being advanced by our collaborators and partners including ECUR-506; our gene editing approaches including editing efficiency, and the suitability of ARCUS nucleases for gene insertion, gene elimination and gene excision and differentiation from other gene editing approaches; the expected timing of regulatory processes and clinical operations for our wholly-owned and partnered product candidates, including filings, studies, enrollment and clinical data; the translatability of preclinical data to development of durable, curative treatments for patients; the initial safety and antiviral activity observed in the ELIMINATE-B clinical trial; plans to provide ongoing updates on the PBGENE-HBV study; anticipated timing of clinical data; clinical efficacy and safety data in the OTC-HOPE clinical trial; ability of an ARCUS nuclease to eliminate mutant mitochondrial DNA and achieve therapeutically meaningful heteroplasmy shifts in vivo; the therapeutic potential of PBGENE-DMD and advancement through preclinical studies; and the potential of proof of concept for ARCUS-mediated excision of the C9orf72 gene HRE and further development of PBGENE-CNS for ALS. In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “appear,” “approach,” “believe,” “contemplate,” “could,” “designed,” “encouraged”, “estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “promising,” “pursue,” “should,” “strive,” “suggest,” “target,” “will,” “would,” or the negative thereof and similar words and expressions. Forward-looking statements are based on management’s current expectations, beliefs and assumptions and on information currently available to us. These statements are neither promises nor guarantees, but involve number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to become profitable; our ability to procure sufficient funding to advance our programs; risks associated with raising additional capital and requirements under our current debt instruments and effects of restrictions thereunder; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities, preclinical studies and clinical trials; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators’ ability to identify, develop and commercialize product candidates; potential product liability lawsuits and penalties against us or our collaborators related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators’ development of product candidates; our or our collaborators’ or other licensees’ ability to advance product candidates into, and successfully design, implement and complete, clinical or field trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; delays or difficulties in our and our collaborators’ ability to enroll patients; changes in interim “top-line” and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; our ability to obtain orphan drug designation or fast track designation for our product candidates or to realize the expected benefits of these designations; our or our collaborators’ ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; the rate and degree of market acceptance of any of our product candidates; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate executives and personnel; effects of system failures and security breaches; insurance expenses and exposure to uninsured liabilities; effects of tax rules; effects of any pandemic, epidemic, or outbreak of an infectious disease; the success of our existing collaboration agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events; effects of sustained inflation, supply chain disruptions and major central bank policy actions; market and economic conditions; risks related to ownership of our common stock, including fluctuations in our stock price; our ability to meet the requirements of and maintain listing of our common stock on NASDAQ or other public stock exchanges; and other important factors discussed under the caption “Risk Factors” in our Annual Report on Form 10-K for the annual period ended December 31, 2024, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC’s website at www.sec.gov and the Investors page of our website under SEC Filings at investor.precisionbiosciences.com. All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. View source version on businesswire.com: https://www.businesswire.com/news/home/20250501903476/en/ Investor and Media Contact: Naresh Tanna Vice President of Investor Relations naresh.tanna@precisionbiosciences.com Source: Precision BioSciences, Inc.

Spinal muscular atrophy treatment Spinraza (nusinersen) became Biogen’s best-selling drug in Q1 2025. Image credit: Shutterstock/ Tada Images. Biogen has unveiled Q1 2025 revenues above estimates, spurred by strong sales of rare disease assets amid a sales dropoff from its multiple sclerosis business. Quarterly revenues for the neurological disease drugmaker were $2.43bn, above analyst group William Blair’s estimates of $2.3bn.  The Q1 sales performance represents a 6% increase compared to the same period in 2024, with Biogen ramping up commercial duties for existing drugs and buying assets during that time. One of those assets is Skyclarys, a drug picked up in a $7.3bn acquisition of Reata Pharmaceuticals in September 2023. The treatment for the neurodegenerative disorder Friedreich’s ataxia generated revenue of $124m in Q1, a value that had already taken into account the impact seen in US sales due to Medicare discount dynamics. Approved in the US, Europe, and UK, amongst others, Skyclarys has a high growth ceiling. GlobalData’s Pharma Intelligence Centre forecasts sales of $1.3bn by 2031. GlobalData is the parent company of Pharmaceutical Technology . Skyclarys is one of the drugs spurring a change of the guard in Biogen’s pipeline. The company is well known for its host of multiple sclerosis treatments, such as Vumerity (diroximel fumarate), Tysabri (natalizumab), and Avonex (interferon beta-1a). But, sales for these treatments have declined over the years as biosimilars entered the market. The company’s multiple sclerosis business shrank 11% in Q1 2025 compared to the same period last year. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence “Biogen is really, a tale of two companies in my view. There’s one company which has been a multiple sclerosis company, and that portfolio, as you all know, has been gradually declining,” said Biogen’s CEO Chris Vibacher in a Q1 earnings call. “But there’s a new Biogen emerging. And when I look at the rare disease business […] we actually have a commercial portfolio that we’re actively promoting that’s now gotten to be about 45% of our product revenue. And those products mostly have a very long runway to continue to grow,” Vibacher added. Alongside Skyclarys, Biogen’s rare disease business is headed by spinal muscular atrophy treatment Spinraza (nusinersen). Spinraza generated global Q1 2025 sales of $423.9m, up from $341.3m in Q1 2024. This increase, coupled with the decline of Tysabri, meant Spinraza was Biogen’s best-selling drug in Q1 2025. Leqembi’s “modest growth” Another key part of Biogen’s business model is its Alzheimer’s segment, now solely headed by Leqembi (lecanemab) after the company discontinued Aduhelm (aducanumab) at the start of 2024. Biogen splits revenue from Leqembi with co-developer Eisai equally, the latter reporting Q1 sales of $96m for the drug. The US, where Leqembi got a full approval in July 2023, accounted for $52m of those sales. Biogen has already secured key milestones for Leqembi this year. In January, the US Food and Drug Administration (FDA) approved maintenance dosing of Leqembi, a move expected to expand its market share. Then in April, the Leqembi was authorised for use in the European Union (EU). Biogen said the Q1 sales for Leqembi represented “continued sequential growth.” William Blair analyst Myles R. Minter said in a research note that: “Skyclarys had a strong quarter, but the commercial focus remains on Leqembi, which has seen only modest growth, in our view.” In the mergers and acquisitions space, Biogen failed a $469m takeover bid of Sage Therapeutics in January.