Spinal Cord Diseases

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SPG302 in the U.S. as part of a separate EAP protocol commented, "The notion that ALS is an untreatable disease is outdated.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

Biogen charted first-quarter sales growth thanks to the launches of Leqembi, Zurzuvae and Skyclarys. Burdened by competitive pressures for its stalwart multiple sclerosis franchise, Biogen has weathered years of sales declines. But in the first quarter, the company was able to see a glimpse of its hoped-for future with revenue growth thanks to three launches.Lately, Biogen's story can be summed up as a “a tale of two companies,” CEO Chris Viehbacher mused on a Thursday earnings call with analysts.There’s one company, a multiple sclerosis (MS)-centered drugmaker, with a portfolio that has been gradually declining, with its sales falling 11% during the first quarter. But now with 45% of Biogen's revenue coming from medicines outside of its MS franchise, “there’s a new Biogen emerging,” according to Viehbacher.The fresh start is hallmarked by Alzheimer’s disease med Leqembi, postpartum depression (PPD) treatment Zurzuvae and Friedreich’s ataxia therapy Skyclarys, all of which helped drive Biogen to a 6% revenue increase during the first quarter of 2025. Overall, the company generated $2.4 billion during the period. Eisai-partnered Leqembi is now into “serious product territory,” Viehbacher noted, growing a whopping 395% since last year’s first quarter—and 11% from the fourth quarter of last year—to $96 million in sales. The drug finally cinched a European approval in April after months of back-and-forth decisions by European officials, unlocking a key market with “an awful lot of eligible patients,” according to the CEO.It’s been a “challenging product to launch,” Viehbacher observed, but several recent advancements in administration—including an anticipated FDA decision for subcutaneous maintenance dosing—should help reduce the treatment burden.Currently, the Alzheimer’s antibody treatment market consists of Leqembi and Eli Lilly’s rival Kisunla (donanemab), which has faced many of the same launch barriers. Biogen accepts the competition, acknowledging that it will ultimately be “up to the physician and the patient” to determine which treatment to choose, Viehbacher explained.“I think the market ultimately gets split between us and donanemab,” the CEO said. “The most important thing for both Lilly and Biogen and Eisai is that we start to really expand this market.” As it stands, the companies are both only treating a “small fraction” of the patients who “desperately need treatment," the CEO said.Zurzuvae, meanwhile, has so far treated more than 10,000 women since its 2023 launch. Its sales were up 123% since last year’s first quarter to $28 million. Biogen managed to expand the number of physicians writing prescriptions by 20%, the company reported, in part by focusing on OB-GYNs as opposed to psychiatrists. The drug was the first oral medicine approved to specifically treat PPD. With Zurzuvae on the market, physicians are becoming “more proactive” about diagnosing the historically overlooked condition, Viehbacher commented.  Elsewhere, Biogen is still underway with a geographic expansion for Skyclarys, having scored recent approvals in the U.K. and Brazil. With $124 million in first-quarter sales, the drug was the biggest revenue contributor out of its three new launches. Friedreich’s ataxia is a genetic disease with European origin, leaving the Biogen team to get “very creative” in finding new U.S. patients through family trees and social media, according to the CEO.So far, around 2,400 patients are on the treatment globally; some are still on early access programs rather than commercial therapy. No concern for tariffs For many of Biogen's peers, the threat of looming pharmaceutical tariffs and changing trade policies could overshadow their future prospects. But Biogen's diversified revenue base means that some 55% of its product revenue last year came from sales outside of the U.S., which is “pretty unusual in our business,” Viehbacher said. And given that about 75% of last year’s U.S. product revenue is tied to drugs that have manufacturing operations in the U.S., Massachusetts-based Biogen is “more of a U.S.-based company” and is “quite proud of that,” the CEO noted. The drugmaker does not expect its financial outlook, which estimates a revenue decline by a mid-single-digit percentage in 2025 due to falling MS sales, to be impacted by currently announced tariffs, “even if the exemption for pharmaceuticals were to be removed,” the company said. Elsewhere in the policy vein, Biogen and Viehbacher have “not really seen” any delays in its interactions with the FDA or any “adverse effects” from key leaders who have left the agency.

Research highlights AAV manufacturing optimization via Lexeo’s Sf9-baculovirus processNEW YORK, May 01, 2025 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company dedicated to pioneering novel treatments for cardiovascular diseases, announced today that new data supporting its AAV manufacturing approach will be presented at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) taking place May 13-17, 2025 in New Orleans, LA. “The data being presented at ASGCT underscore the strength of our industry-leading capabilities in AAV manufacturing with high yield and high quality,” said José Manuel Otero, Chief Technical Officer of Lexeo Therapeutics. “Scientists at Lexeo have optimized a manufacturing platform that can maintain purity and potency while significantly improving scalability of production and reducing cost. Ultimately this platform will enable Lexeo to deliver more efficiently against key milestones in our clinical-stage gene therapy programs and will help to accelerate our mission to bring potentially transformative therapies to patients as quickly as possible.” Presentation Details: Title: Improving VP1 Ratios Impact on CQAs in rh10 AAV Manufactured through Sf9 PlatformPresenter: Elena Bianchetti, Lexeo TherapeuticsDate/Time: Tuesday, May 13, 6:00 – 7:30 p.m. CSTSession Title: Tuesday Poster ReceptionAbstract Number: 948 Title: Development of a Novel High-Yielding Scalable Sf9-Baculovirus Platform to Produce Quality AAV at 200L ScalePresenter: Eric Lin, Lexeo TherapeuticsDate/Time: Thursday, May 15, 5:30 – 7:00 p.m. CSTSession Title: Thursday Poster ReceptionAbstract Number: 1972 About Lexeo TherapeuticsLexeo Therapeutics is a New York City-based, clinical stage genetic medicine company dedicated to reshaping heart health by applying pioneering science to fundamentally change how cardiovascular diseases are treated. The Company is advancing a portfolio of therapeutic candidates that take aim at the underlying genetic causes of conditions, including LX2006 for the treatment of Friedreich ataxia (FA) cardiomyopathy, LX2020 for the treatment of plakophilin-2 (PKP2) arrhythmogenic cardiomyopathy, and others in devastating diseases with high unmet need. Cautionary Note Regarding Forward-Looking StatementsCertain statements in this press release may constitute “forward-looking statements” within the meaning of the federal securities laws, including, but not limited to, Lexeo’s expectations and plans regarding its current product candidates and programs, and the timing and likelihood of potential regulatory approval. Words such as “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “expect,” “believe,” “design,” “estimate,” “predict,” “potential,” “develop,” “plan” or the negative of these terms, and similar expressions, or statements regarding intent, belief, or current expectations, are forward-looking statements. While Lexeo believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements. These forward-looking statements are based upon current information available to the company as well as certain estimates and assumptions and are subject to various risks and uncertainties (including, without limitation, those set forth in Lexeo’s filings with the U.S. Securities and Exchange Commission (SEC)), many of which are beyond the company’s control and subject to change. Actual results could be materially different from those indicated by such forward-looking statements as a result of many factors, including but not limited to: risks and uncertainties related to global macroeconomic conditions and related volatility; expectations regarding the initiation, progress, and expected results of Lexeo’s preclinical studies, clinical trials and research and development programs; the unpredictable relationship between preclinical study results and clinical study results; delays in submission of regulatory filings or failure to receive regulatory approval; liquidity and capital resources; and other risks and uncertainties identified in Lexeo’s Annual Report on Form 10-K for the annual period ended December 31, 2024, filed with the SEC on March 24, 2025 and subsequent future filings Lexeo may make with the SEC. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Lexeo claims the protection of the Safe Harbor contained in the Private Securities Litigation Reform Act of 1995 for forward-looking statements. Lexeo expressly disclaims any obligation to update or alter any statements whether as a result of new information, future events or otherwise, except as required by law. Media Response:Media@lexeotx.com