Latest news with #stemcelltherapy
National Post
2 days ago
- Business
- National Post
Pluristyx Completes Investment Round to Expand Product Portfolio and Commercial Operations
Article content SEATTLE — Pluristyx, a leading provider of induced pluripotent stem cell (iPSC) technologies and proprietary gene editing solutions, today announced the successful close of its most recent round of funding led by BioLife Solutions, the global leader in cryopreservation technologies and systems that support cell and gene therapy (CGT) manufacturing, and BroadOak Capital Partners, a specialist investor in the life science tools and services industry. Article content The newly raised capital will enable Pluristyx to expand inventory with new clinical grade cell lines incorporating Pluristyx's proprietary FailSafe® and iACT™ engineering and hypoimmune genetic edits in its best-in-class, polyclonal, synthetic RNA reprogramed iPSCs. These proprietary cells are specifically designed to support making next-generation genetically engineered, stem cell-derived therapies and support uniquely safe, effective, and scalable living medicines. The funding will also support Pluristyx's recently launched PluriForm ™ kit, which provides researchers with streamlined tools for generating organoids and other complex test systems. Article content 'We are incredibly excited for the successful close of this round and gratified to have industry leaders and peers validate our vision with their investment,' said Benjamin Fryer, PhD, CEO of Pluristyx. 'The support from our Seattle area neighbor, BioLife Solutions, and continued support from our colleagues at BroadOak Partners will allow us to manufacture, commercialize, and distribute our innovative solutions and help our customers solve critical industry challenges.' Article content Roderick de Greef, BioLife's Chairman and CEO, remarked, 'We have known and worked with the founding team at Pluristyx for many years, and have a great deal of respect for their scientific expertise in cell therapy. Their recent development of an iPSC-based biological assay for organoid manufacturing dovetails with our interest in exploring biological assays more broadly as a product portfolio adjacency.' Article content 'We are excited to continue our support of Dr. Fryer and the entire Pluristyx team,' said Daniel Friedman, Principal at BroadOak Capital Partners. 'Pluristyx is differentiated by its suite of proprietary technologies that directly address some of the most critical scientific challenges of the iPSC field.' Article content iPSCs are unique tools and building blocks for medicines. In addition to applications for innovative new toxicology, safety, and efficacy assays to replace animal models, Pluristyx's products are the ideal universal raw material to produce living medicines to cure currently untreatable diseases. This strategic investment will enable Pluristyx to be the industry leader offering the highest quality stem cells available anywhere. Article content About BioLife Solutions Article content BioLife Solutions is a leading developer and supplier of bioproduction products and services for the cell and gene therapy (CGT) and broader biopharma markets. Our expertise facilitates the commercialization of new therapies by supplying solutions that maintain the health and function of biologic materials during collection, development, storage, and distribution. For more information, please visit or follow BioLife on LinkedIn and X. Article content About BroadOak Capital Partners Article content BroadOak Capital Partners is a boutique financial institution that provides direct investment and investment banking services to companies in the life science tools, diagnostics, and biopharma services sectors. BroadOak has led or participated in investments in over 70 companies across the life sciences industry. For more information, visit Article content Pluristyx is dedicated to enabling the development of next-generation cell-based tools and therapies. PluriKit™, PluriForm™ Organoids, and PluriBank™ stem cells incorporating safety and tolerance features like FailSafe®, iACT™, and hypoimmune engineering streamline developers' path to the clinic and approval. Pluristyx's iPSC lines, backed by an FDA registered drug master file, with platform genetic engineering are available 'off-the-shelf' along with comprehensive support services to accelerate the creation of novel diagnostics, screening and safety testing, and curative treatments. For more information, visit Article content Article content Article content Article content
Yahoo
12-07-2025
- Health
- Yahoo
Stem Cell Treatment to Reverse Hearing Loss Kicking Off in Human Patients
The first-ever human trial exploring the use of stem cell therapy to reverse hearing loss is about to be under way, after getting the go-ahead from the UK's Medicines and Healthcare products Regulatory Agency. Conducted by researchers from the University of Sheffield who formed their own spin-out company, the treatment targets sensorineural hearing loss, which is caused by physical damage to the tiny structures of the inner ear. In a nutshell, the treatment, dubbed Rincell-1, is intended to regrow damaged nerves in the cochlea and allow them to start sending signals to the brain again. "Our research into Rincell-1 has consistently shown its ability to target and restore the delicate neural structures of the inner ear," Marcelo Rivolta from the University of Sheffield, chief scientific officer at the spin-out biotech company Rinri Therapeutics, said in a statement about the work. More than a billion people worldwide are affected by some form of hearing loss, according to the WHO. Existing treatments, like cochlear implants, don't fully restore a patient's hearing, and require that the cochlea — the center piece of the inner ear that picks up sound and turns it into electrical signals — isn't significantly damaged to begin with. Key to the cochlea's function are the hair cells that line its surface, which are responsible for detecting sound. If these are severely damaged, it's game over: the hair cells are incapable of dividing to form new ones, meaning that they don't regenerate. This is called sensorineural hearing loss, and it's why your hearing inevitably worsens with age. Attempts to regrow these receptors have long been the white whale of modern medical science, and the researchers hope they've found it in Rincell-1. The treatment uses embryonic stem cells designed to grow into auditory neuron cells, which form the wiring that connects the hair cells to the brain stem. These are administered into the cochlea during the surgery to emplace the cochlear implants. Once on site, the stem cells form auditory neurons that help reconnect the out-of-commission hair cells so they can send signals again. "We are taking the approach of transplanting in cells that can become functional mature cells and restore the cytoarchitecture of the inner ear, and therefore, restore hearing," Simon Chandler, CEO of Rinri Therapeutics, told Labiotech on an episode of its "Beyond Biotech" podcast in June. The randomized trial will be conducted in the UK and will involve 20 patients who will undergo cochlear implant surgery. Half of the patients will have severe-to-profound age-related hearing loss, known as presbycusis, and the remainder will have what's known as auditory neuropathy spectrum disorder (ANSD), which is when the inner ear can detect sound but is unable to send the signals to the brain. Patients within each group will be randomly assigned to either receive a single dose of Rincell-1, or receive no dose and rely on the cochlear implant alone. On the podcast, Chandler said that the Rincell-1 treatment can be used alongside cochlear implants, but didn't rule out the possibility that it would be used on its own, too. Needless to say, it'd be a game-changer if it turns out to be effective in either scenario. More on stem cells: Diabetic Woman No Longer Needs Insulin After Single Dose of Experimental Stem Cells
Yahoo
09-07-2025
- Business
- Yahoo
Celularity & Fountain Life Announce Partnership to Deliver Stem Cell Therapies Under New Florida Law
Fountain Life operates four flagship longevity centers, including two in Florida, where a new state law effective July 1, 2025, authorizes licensed physicians to administer stem cell therapies to members with conditions in orthopedics, wound care, and pain management. FLORHAM PARK, N.J., July 09, 2025 (GLOBE NEWSWIRE) -- Celularity Inc. (Nasdaq: CELU) ('Celularity'), a regenerative and cellular medicine company focused on addressing age-related and degenerative diseases, announced today that it entered into a strategic partnership with Fountain Life, a global leader in science-backed longevity and preventative health company, to supply stem cell therapy products manufactured and distributed by Celularity. Fountain Life intends to deploy Celularity's technology in accordance with Florida Statute § 458.3245, which became effective July 1, 2025. The law authorizes qualified physicians in the state of Florida to treat patients with autologous or allogeneic adult stem cell products under specific conditions, including the treatment of orthopedic conditions, wound healing, and pain management, without requiring investigational new drug (IND) approval by the U.S. Food and Drug Administration (FDA). Fountain Life currently operates four state-of-the-art longevity centers across the United States, including Naples, and Lake Nona-Orlando, Florida and soon to open in Miami. These centers are well-positioned to implement Florida's new legal framework and deliver innovative, physician-directed regenerative cellular therapies to eligible patients. Celularity is a pioneer in the field of cell therapies sourced ethically from the postpartum placenta. Its pipeline includes human placental-derived stems cells and mesenchymal stem cell-like product candidates, supported by extensive clinical datasets across multiple therapeutic areas aligned with the use cases permitted under the new Florida statute. Celularity maintains a substantial inventory of investigational cell therapies, which are immediately available for physician-directed use, at its FDA-registered, accredited GMP facility in Florham Park, New Jersey. This collaboration aligns with Celularity's mission to expand access to regenerative cellular therapies and provides a regulated framework for real-world deployment of its placental-based technologies. 'Celularity's ability today to supply clinical-grade, ethically-sourced stem cells ensures 'Day One' readiness under the new Florida law to partners like Fountain Life,' said Robert J. Hariri, M.D., Ph.D., CEO and Chairman. 'We applaud Florida's proactive, data-driven approach to advancing investigational stem cell therapies and improving patient outcomes, for which Fountain Life, a leading science-backed longevity center, powered by AI, is an ideal partner.' Hariri added, 'Celularity is unique in our ability to also deliver placental-derived biomaterials for many of the indications targeted in the Florida legislation and we are committed to meeting the high standards laid out in this important new healthcare law.' 'Cellular medicine is at the core of Fountain Life's mission to transform healthcare and extend human longevity,' said Dr. William Kapp, CEO and co-founder of Fountain Life. 'We apply the most rigorous standards to every regenerative therapy we offer, ensuring it meets the highest benchmarks for safety, efficacy, and scalability. Florida's stringent criteria for stem cell sourcing align perfectly with our uncompromising commitment to clinical excellence. We chose to partner with Celularity because of their proven ability to meet and exceed those standards, providing our members access to the most vetted, advanced cellular therapeutics.' About Celularity Celularity Inc. (Nasdaq: CELU) is a regenerative and cellular medicine company developing and commercializing advanced biomaterial products and cryopreserved allogeneic cell therapies, all derived from the postpartum placenta. Celularity believes that by harnessing the placenta's unique biology and ready availability, it can develop therapeutic solutions that address significant unmet global needs for effective, accessible, and affordable therapies. For more information about Celularity and its cutting-edge regenerative medicine solutions, please visit Forward Looking StatementsCertain statements in this press release are 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, without limitation, statements regarding: (i) our future sales or sales growth; (ii) our expectations for future financial results, including levels of net sales; (iii) our expectations regarding new products including our 510K products; and (iv) future demand for our products. All statements other than statements of historical facts are 'forward-looking statements,' including those relating to future events. In some cases, you can identify forward-looking statements by terminology such as 'anticipate,' 'believe,' 'can,' 'could,' 'continue,' 'expect,' 'improving,' 'may,' 'observed,' 'potential,' 'promise,' 'should,' and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances). Forward-looking statements are based on Celularity's current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks, and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Many factors could cause actual results to differ materially from those described in these forward-looking statements, including those risk factors set forth under the caption 'Risk Factors' in Celularity's annual report on Form 10-K and Form 10-K/A for the year ended December 31, 2024 filed with the Securities and Exchange Commission (SEC) on May 8, 2025 and May 21, 2025, respectively, and other filings with the SEC. If any of these risks materialize or underlying assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. There may be additional risks that Celularity does not presently know, or that Celularity currently believes are immaterial, that could also cause actual results to differ from those contained in the forward-looking statements. In addition, these forward-looking statements reflect Celularity's current expectations, plans, or forecasts of future events and views as of the date of this communication. Subsequent events and developments could cause assessments to change. Accordingly, forward-looking statements should not be relied upon as representing Celularity's views as of any subsequent date, and Celularity undertakes no obligation to update forward-looking statements contained herein, whether because of any new information, future events, changed circumstances or otherwise, except as otherwise required by law. Carlos RamirezSenior Vice President, Celularity About Fountain Life Fountain Life is the leading science-backed longevity company, committed to proactive and data-driven global healthcare. Founded in 2019, it is the world's first fully integrated destination for proactive health and longevity—combining advanced AI diagnostics, cutting-edge therapeutics, restorative therapies, and Zori AI, its proprietary health intelligence engine. Through continuous optimization, Fountain Life helps members live longer, healthier lives. Built to shift healthcare from reactive to preventive, Fountain Life offers scalable access through its tiered membership model: CORE, APEX, and EPIC. Fountain Life is redefining the future of medicine. Discover more at Isabel CosciaChief Marketing Officer, Fountain in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
07-07-2025
- Business
- Yahoo
Celularity Hails New Florida Law Opening Patient Access to Stem Cell Therapies
New law goes into effect on July 1st authorizing Florida physicians to provide stem cell therapies to their patients for orthopedics, wound care, and pain management FLORHAM PARK, N.J., July 01, 2025 (GLOBE NEWSWIRE) -- Celularity Inc. (Nasdaq: CELU) ('Celularity' or the 'Company'), a regenerative and cellular medicine company, today hailed a new Florida law taking effect today that opens the door to physicians' use of investigational stem cell therapies in the state in orthopedics, wound care, and pain management. 'Today, a groundbreaking new law takes effect in Florida authorizing physicians to provide stem cell therapies to their patients subject to strict requirements ensuring patient safety and ethical standards,' said Robert J. Hariri, M.D., Ph.D., Celularity CEO and Chairman. 'Celularity is fully prepared to supply the ethically sourced stem cells which enable these treatments, and we look forward to working with Florida physicians to make these innovative therapies available to patients.' The new law authorizes Florida physicians to provide stem cell therapies that have not yet been approved by the US Food and Drug Administration, or FDA, and imposes strict limits on the permissible sources of the stem cells used, which must be manufactured, stored and retrieved in facilities that are FDA registered and certified or accredited by approved organizations. Approved facilities must comply with Good Manufacturing Practices, which are FDA-established standards that mandate strict controls on facilities, processes, and quality control. Additionally, physicians must obtain patient informed consent prior to administering treatments authorized under the new law. 'We applaud Florida's intention to advance medical treatments and improve patient outcomes with this balanced approach to fostering medical innovation while upholding ethical standards,' said Dr. Hariri. 'Celularity is the long-standing leader in the field of ethically sourced stem cell therapies with extensive knowledge and real world data obtained from multiple clinical trials, including wound care and other therapeutic targets authorized under the new Florida law.' In 2001, Celularity's predecessor organization, Anthrogenesis Corporation (later Celgene Cellular Therapeutics) announced a groundbreaking discovery of a method to recover a then-novel kind of stem cell from the postpartum human placenta. President George W. Bush acknowledged this transformational discovery around ethically sourced stem cells in his landmark August 2001 speech on stem cell research. 'We believe Florida's physician-focused approach is a model for other states, insofar as physicians are ideally positioned to assess which therapeutic approach is in a patient's best interest, including investigational stem cell therapies,' said Dr. Hariri, noting the Florida Medical Association's statement that it 'was extensively involved in the crafting of this legislation and sought to ensure that a proper balance was struck between protecting the public from bogus therapies and preventing physician overregulation.' Celularity operates a purpose-built cell therapy manufacturing and storage/retrieval facility with hundreds of units of placenta-derived stem cells in inventory for use in investigational therapies including those authorized under the new Florida law. Celularity's facility is FDA registered and certified/accredited as required by the new Florida law. 'Celularity is positioned right now to supply the stem cells necessary to fulfill the objectives of the new Florida law and we look forward to working with Florida physicians to ensure their patients have access to stem cell therapies,' said Dr. Hariri. About Celularity Celularity Inc. (Nasdaq: CELU) is a regenerative and cellular medicine company developing and commercializing advanced biomaterial products and allogeneic, cryopreserved, placental-derived cell therapies, all derived from the postpartum placenta. Celularity believes that by harnessing the placenta's unique biology and ready availability, it can develop therapeutic solutions that address significant unmet global needs for effective, accessible, and affordable therapies. For more information about Celularity and its cutting-edge regenerative medicine solutions, please visit Forward Looking StatementsCertain statements in this press release are 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, without limitation, statements regarding: (i) our future sales or sales growth; (ii) our expectations for future financial results, including levels of net sales; (iii) our expectations regarding new products including our 510K products; and (iv) future demand for our products. All statements other than statements of historical facts are 'forward-looking statements,' including those relating to future events. In some cases, you can identify forward-looking statements by terminology such as 'anticipate,' 'believe,' 'can,' 'could,' 'continue,' 'expect,' 'improving,' 'may,' 'observed,' 'potential,' 'promise,' 'should,' and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances). Forward-looking statements are based on Celularity's current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks, and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Many factors could cause actual results to differ materially from those described in these forward-looking statements, including those risk factors set forth under the caption 'Risk Factors' in Celularity's annual report on Form 10-K and Form 10-K/A for the year ended December 31, 2024 filed with the Securities and Exchange Commission (SEC) on May 8, 2025 and May 21, 2025, respectively, and other filings with the SEC. If any of these risks materialize or underlying assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. There may be additional risks that Celularity does not presently know, or that Celularity currently believes are immaterial, that could also cause actual results to differ from those contained in the forward-looking statements. In addition, these forward-looking statements reflect Celularity's current expectations, plans, or forecasts of future events and views as of the date of this communication. Subsequent events and developments could cause assessments to change. Accordingly, forward-looking statements should not be relied upon as representing Celularity's views as of any subsequent date, and Celularity undertakes no obligation to update forward-looking statements contained herein, whether because of any new information, future events, changed circumstances or otherwise, except as otherwise required by law. Carlos RamirezSenior Vice President, Celularity in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Associated Press
07-07-2025
- Health
- Associated Press
ALS Community Files Citizens' Petition asking FDA to Approve NurOwn Stem Cell Therapy
New and Unprecedented Survival, Respiratory, and Biomarker Data Prove that NurOwn Helps People with ALS Live Longer and Live Stronger BREMERTON, WA / ACCESS Newswire / July 7, 2025 / On July 4, 1939, Lou Gehrig delivered his iconic 'luckiest man' speech, announcing his retirement from the New York Yankees. On that day, ALS ended his Hall of Fame career. Less than two years later, ALS ended his life at just 37 years old. In the last 86 years, the lethal outcome has not changed. ALS is a cruel, paralyzing and 100% fatal disease. But today, the ALS community has hope. A coalition of ALS patients and family members has filed a Citizens' Petition with the FDA, requesting the approval of NurOwn, a neurotrophically-enhanced stem cell therapy. Backed by a decade of real-world data from the NurOwn trials and Expanded Access Program (EAP), the 309-page Citizens' Petition details the unprecedented survival, respiratory, and biomarker data for the FDA's consideration. The new evidence is supported by testimony from top ALS neurologists who were the trial's principal investigators, and the 'totality of the evidence' from the Phase 3 trial. And, it aligns with real-world evidence where trial participants (now-unblinded) and their treating neurologists have proclaimed that NurOwn improves how people with ALS 'feel, function and survive.' NurOwn: A Revolutionary Approach to ALS Treatment Developed by BrainStorm Cell Therapeutics, NurOwn combines the restorative potential of autologous mesenchymal stem cells with the regenerative power of neurotrophic factors, which are like 'Miracle-Gro' for dying motor neurons. NurOwn uses a patient's own stem cells that work like a FedEx truck, delivering nano-packages of neurotrophic factors and immunomodulatory cytokines directly to damaged motor neurons. The results are profound. Within days, trial participants reported halting of symptoms like fasciculations, cramping and clonus; and some improvements in function. With additional doses, the EAP data confirm NurOwn's ability to slow lethal ALS progression, improve function, restore breathing, and extend survival - offering a lifeline to those battling this 100% fatal and paralyzing disease. Unprecedented Survival and Respiratory Data Survival data have long been the gold standard for FDA approvals; and as Commissioner Makary has emphasized: 'gold standard science and common sense' will guide this FDA's decisions. To that end, Petitioners have submitted survival data derived from their own real-world evidence over the past decade. These survival data are unprecedented in ALS clinical trial history. (See Petition's Emergent Fact section C at pg 19-33). At the FDA Advisory Committee meeting for NurOwn in 2023, Dr. Anthony Windebank of Mayo presented the clinical trial data and shared his expert opinion about the progression-free survival that he and other experienced trial investigators had witnessed - unprecedented in their prolific 40+ year neurology practices: 'I think this data is compelling & it should be approved…. While not everyone responds to the treatment, there are clearly a significant number who do. I have clearly seen some people stabilize in a way that I have never seen in any other trial. In fact, in the small number of people who participated in EAP and received 6-9 treatments, there were people who stabilized while on NurOwn in the trial. In the interval before they were in the EAP - which was over a year or more in some cases - these participants deteriorated, then again stabilized in the additional [EAP] treatment period. There were some who IMPROVED their score. Other investigators who have been working 'hands on' with the participants in the trial have seen similar responses....' Dr. Windebank's testimony underscores the unprecedented impact of NurOwn on people with ALS. And the NurOwn survival data is buttressed by other compelling efficacy data also detailed in the Citizen's Petition: Real-World Evidence and Patient Experiences Our Citizens' Petition also leverages real-world evidence (RWE) and real-world data (RWD) from the EAP and Right to Try - consistent with the Congressional intent of the 21st Century Cures Act. Multiple trial participants testified, submitted Public Comments and shared their RWE, which aligns with the type of efficacy evidence specified in the ALS Guidance Document and 21st Century Cures. At the time of the advisory committee meeting in 2023, many trial participants reported tangible improvements in how they felt and functioned, and hence, an improved quality of life. (See sections H & I, pgs. 91-127). Their testimony was supported by video evidence documenting those improvements and by the opinions from multiple treating neurologists outside the clinical trial. For example, neuromuscular specialist Dr. Danielle Geraldi-Samara submitted a Public Comment to the FDA about what she observed in many of her patients participating in the NurOwn Phase 3 trial and EAP: 'The real world evidence could not be more striking. I have known patients nearly immobile who gained some functionality in their gait, patients with severe dysarthria become intelligible, patients who could not manage the fine motor skill needed to button or zipper, finally able to dress independently. I have patients with solid plateaus [in ALSFRS-R scores] over the course of a year.' Her clinical observations of progression-free survival after the NurOwn trial mirror those of Dr. Windebank and the other investigators during the trial and EAP. Now that the Phase 3 trial has been unblinded, multiple trial participants have confirmation that NurOwn halted their lethal progression and helped some people regain function. Our lived patient experiences now have both validation and vindication. When people are becoming paralyzed, it's common sense that we know when a therapy helps us function. Our lived patient experiences aren't anecdotal hyperbole; they are evidence. And as Commissioner Makary recently said at the Gene and Cell Therapy Forum, there is value in learning from 'n of 1" cases. Combined, the EAP 'n of 10" and the right to try 'n of 1" illustrate compelling and consistent, dose-dependent evidence of efficacy. Reinforcing the efficacy data, Navy pilot Matt Bellina shared the RWE and RWD contained in his VA medical records in his blog and on social media. Matt too experienced unprecedented clinically meaningful improvements after receiving 7 doses of NurOwn via Right to Try. Although he was a slow progressor, diagnosed in 2011, Matt's ALS had progressed significantly. He was choking on food, using NIV to breathe at night; had little use of his hands; and could not stand without assistance. His data are informative, supporting evidence of efficacy because he is the only person in the US who received 6 consecutive doses; because he was the only 'slow progressor' to receive NurOwn; and because his baseline score was 21/48 on the ALS Functional rating scale. Matt's large magnitude, dose-dependent improvement in function was immediate and obvious. (See section J at pgs 128-133). Matt has video documenting him standing out of a wheelchair unassisted - the first time in two years. He stopped choking on food. He i mproved his functional score by 6 points. His FVC stabilized and he stopped using NIV to breathe for more than 4 years. NurOwn interrupted Matt's lethal trajectory to death. Commissioner Makary has repeatedly offered that the FDA, under President Trump, 'believes in both the spirit and the letter of right to try. " Thus, Petitioners hope that this FDA will consider and believe the RWE from the very veteran for whom President Trump's Right to Try law was named. Totality of Evidence Methodology for Rare Diseases To determine if a therapy can meet the approval threshold of 'substantial evidence,' the FDA asks if a therapy improves how people 'feel, function or survive.' Regulators look principally at the trial's primary endpoint at one fixed point in time at the end of the trial. But in heterogeneous rare diseases with small populations and short placebo-controlled trials, efficacy signals can be missed. Hence, it's much more likely to result in a Type II statistical error: delaying or denying approval of a drug that does work. In a terminal disease like ALS, Type II errors cause ongoing paralysis and death. Thus, the Citizens' Petition reasserts the propriety of the FDA's use of the 'totality of evidence' statistical methodology to assess NurOwn's efficacy. This approach - widely accepted in oncology for evaluating therapies in heterogeneous, rare populations - strengthens the case for NurOwn's approval by highlighting the consistent benefits in the subgroup of ALS patients earlier in ALS progression (akin to a drug working on stage I and II cancer patients). When including the trial population with the most advanced ALS (akin to stage III/IV cancer), the trial did not meet its endpoints. But when looking at the patients earlier in ALS progression, NurOwn met statistical significance. Using the 'totality of the evidence' methodology, renowned biostatistician and Wilkes Award winner, Dr. Lee-Jen Wei of Harvard / Dana Farber analyzed the multiple trial endpoints, across multiple functional scale domains, at multiple time points throughout the 28-week trial. He testified at the Advisory Committee meeting that these p-values were: 0.045, 0.021, 0.007 and 0.005; thus providing more supporting evidence of NurOwn's efficacy. Meeting FDA Approval Thresholds The Citizens' Petition asserts that NurOwn achieves the statutory thresholds for multiple FDA approval pathways: 1. Traditional Approval NurOwn's survival data, including the five-year survival, TFS, PFS and OS, meet the 'substantial evidence' threshold of one well-controlled trial plus supporting evidence. This conclusion aligns with the FDA's recognition that survival data are the gold standard in FDA approvals. Thus the diversity and magnitude of NurOwn's survival outcomes fulfills both the 'quality' and 'quantity' requirements of 'substantial evidence.' (See Emergent Fact section C, pgs. 19-33). 2. Accelerated Approval NurOwn meets the 'reasonable likelihood' threshold for accelerated approval. The survival data from the 'n of 10" EAP are 'reasonably likely to predict' a favorable impact on irreversible mortality of the 32,000 people with ALS. This survival data far surpasses survival data supporting the accelerated approval of many cancer therapies. (See comparison at Memorandum section I, pgs. 191-209 and Exs. A & B). NurOwn's respiratory data, including delays in time-to-tracheostomy, time-to-NIV, and improved FVC, are also reasonably likely to predict a favorable impact on mortality. (See Emergent Fact section D, pgs. 34-43 and Memorandum II.C pgs. 219-222). NurOwn's CSF biomarker data are also reasonably likely to predict a 'clinically meaningful' effect. NurOwn caused statistically significant changes in first-in-class CSF biomarkers - regardless of disease severity and only in the NurOwn treatment arm. Of the 45 pre-specified biomarkers tested, 23 had statistically significant changes and 15/23 had p-values ≤0.001. These CSF biomarkers provide objective biological evidence of target engagement across pathways of neuroinflammation, neurodegeneration, and neuroprotection. (See Petition Facts section M, pgs. 156-166 and Memorandum section II.C pgs. 219-222). Additionally, Brainstorm Cell has shared neurofilament light biomarker data in a poster presentation at the 2024 NEALS conference. As the FDA has acknowledged, as ALS progression advances, harmful NfL increases, reflecting more diseased and dying motor neurons. At the end of the Phase 3 trial, there was a 9.4% delta between the NurOwn and placebo arm (p=.037). But in those 10 from EAP who were earlier in progression at the start of the Phase 3 trial, the delta between the NurOwn and placebo arms was more apparent. At the end of Phase 3, the 4/10 on placebo had a 37% increase in harmful NfL whereas the 6/10 on NurOwn had a 4% decrease in NfL. With the additional dosing in EAP, the 4/10 in the placebo-crossover group finally experienced a 5% decline in harmful NfL, whereas people on NurOwn maintained a 36% decrease from baseline. Not surprisingly, those who received the most doses of NurOwn and received it earliest in ALS progression had the largest magnitude functional changes and as well as the largest decrease in NfL levels - with two people who received 9 total doses having a decrease of ≥60% in harmful NfL levels. (See table in section II.C.2.a on page 222). As such, the changes in CSF biomarkers are reasonably likely to predict a clinically meaningful benefit, and thus, the third way that NurOwn can meet the threshold for accelerated approval. 3. Conditional Approval NurOwn aligns with Commissioner Makary's proposed 'plausible mechanism of action' threshold for conditional approval. Both stem cell technology and neurotrophic factors are plausible mechanisms of action in ALS; and NurOwn's CSF biomarker data confirms biological plausibility. (See Petition Fact section N, pgs. 173-176 and Memorandum section II.G at pgs. 234-241). A Call for De Novo Review and Expedited Action The Citizens' Petition requests a de novo review by the FDA. The Center for Biologics Evaluation and Research (CBER) has not ever considered the EAP survival, respiratory, or biomarker data, nor has it considered the Right to Try data from Navy pilot Matt Bellina, nor the unblinded and now corroborated RWE/RWD from people who have benefitted from NurOwn since 2011. The Petitioners also request that CBER use the Commissioner's new Priority Voucher to expedite review. The Citizens' Petition also proposes that FDA consider the far-reaching benefits of a Phase 4 post-marketing study, including a biorepository and natural history/exposome database, which aligns with the FDA Priorities outlined by Doctors Makary and Prasad. A Historic Moment for the ALS Community At the recent 2025 Gene and Cell Therapy Forum, Secretary Kennedy shared that the FDA will do everything it can to 'accelerate approvals for rare diseases.' And in their Joint OpEd for JAMA Viewpoints, Commissioner Makary and Director Prasad said the FDA is committed to 'rapidly usher to market new products with transformational potential.' In furtherance of that commitment, Director Prasad told the rare disease community that the FDA will: The Citizens' Petition argues that NurOwn has more than transformational potential. Rather, the survival and respiratory data, along with 8 years of RWE, demonstrate its already transformational impact on people living with ALS. Thus, the ALS community calls on the FDA to approve NurOwn, honoring its commitment to marry 'gold standard science and common sense.' ALS is stealing decades from our lifespans. Just as the FDA acts with urgency for people with terminal cancer, the Citizens' Petition asks the FDA to act with the same urgency as ALS is killing our motor neurons. Please don't let another generation of people with ALS die waiting when we know a stem cell therapy can help us live. About ALS ALS is a 100% fatal, heterogeneous, rare neurodegenerative disease. As motor neurons die, the brain can no longer communicate with the voluntary muscles, which slowly become paralyzed. For reasons researchers don't fully understand, ALS impacts only the motor neurons, not the sensory neurons. Thus, people with ALS still feel cramping, sensations, fasciculations and pain, but they can't move to respond to them. Ultimately, people lose the ability to walk, talk, move, eat, drink, swallow, and eventually, breathe. About the Petitioners The Petitioners are a coalition of people who received NurOwn and others with ALS who could not. We are committed to advancing research, treatment access, and policy changes for ALS. Petitioners: Contact: SOURCE: NurOwn Citizen's Petition press release
