Latest news with #Rett
Business Wire
30-06-2025
- Business
- Business Wire
Neurogene Announces Registrational Trial Design for Embolden™ Study of NGN-401 Gene Therapy for Rett Syndrome
NEW YORK--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced details of Embolden™, the Company's registrational clinical trial designed to evaluate NGN-401 gene therapy for the treatment of females with Rett syndrome in participants ages 3 years and older. The Company has written agreement from the U.S. Food and Drug Administration (FDA) on key aspects of the registrational trial's design. 'We appreciate the partnership with the FDA as we aligned on the key elements of our NGN-401 registrational trial design, which will allow for rapid conversion of the current Phase 1/2 study to a pivotal trial,' said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. 'We believe that the robust endpoint design, which was informed by key opinion leaders, caregiver input and primary market research with payors, will support NGN-401's best-in-class potential and provide key differentiating data critical to the Rett syndrome community and the future commercial success of NGN-401.' 'I appreciate the multi-domain approach across the clinical manifestations of Rett syndrome, and rigor in which the primary endpoint will be evaluated, including both the clinician perspective of improvement using the CGI-I scale with Rett syndrome anchors and videos to demonstrate gains of skills that are clinically meaningful for girls and women with Rett syndrome and their families,' said Elizabeth M. Berry-Kravis, M.D., Ph.D., Professor of Pediatrics, Neurological Sciences and Biochemistry at Rush University, and principal investigator in the NGN-401 clinical trial. 'Participants treated with NGN-401 have demonstrated increased independence, with both fine and gross motor function improvement, and gained the ability to better communicate wants, needs and choices. I have been impressed with the improvements observed in participants after NGN-401 administration, which have included global improvement in signs and symptoms of Rett syndrome and gains of multi-domain developmental milestones that would not be expected to occur spontaneously in the post-regression stage of Rett syndrome. Evaluating treatment effect in participants ages 3 and above in the registrational study will provide important insights on the potential benefits of NGN-401 in younger patients early in the course of this progressive disease.' Obtained Written Agreement from FDA on the Following Key Elements of Embolden Registrational Trial Design After Discussions Under the START Program Study Design: Open-label, single arm, baseline control (i.e., participants serving as their own control); N=18 proposed, subject to feedback from the FDA. Patient Population: Females ages ≥ 3 years with Rett syndrome, which is consistent with Neurogene's analysis of the NIH-sponsored, International Rett Syndrome Foundation (IRSF) Rett syndrome natural history study that shows patients ages 3 and above rarely learn new skills/reach developmental milestones or relearn skills once lost. The FDA's written agreement endorsed the Company's analysis. Dose: 1E15 vector genomes (vg). Primary Endpoint: Responder-based composite endpoint that will assess an improvement in the Clinical Global Impression-Improvement (CGI-I) scale with Rett syndrome anchors and the gain of a developmental milestone/skill, compared to a participant's own baseline. Responders are defined as participants who: Attain a CGI-I score of ≤ 3 ('minimally improved'); and Gain any one developmental milestone/skill from a list of 28, as captured through standardized video recordings and independently verified by blinded central raters. Developmental milestones/skills were informed by a Rett syndrome caregiver survey, provided to the FDA, demonstrating that the acquisition of any single skill within the selected set is considered clinically meaningful. The primary endpoint will be evaluated at 12 months following NGN-401 administration, with feedback pending from the FDA to consider the addition of a 6-month endpoint. Neurogene has begun registrational trial initiation activities. 'It has been incredibly rewarding to see how RSRT's early investments in foundational Rett syndrome science helped lay the groundwork for what's now a promising gene therapy program approaching a registrational trial,' said Monica Coenraads, Founder and Chief Executive Officer of RSRT. 'RSRT has been a long-time champion of addressing the underlying genetic cause of Rett syndrome and Neurogene's program has the potential to do just that. We appreciate the thoughtful design of the trial that importantly incorporates the caregiver perspective on what meaningful improvement may look like. We congratulate the Neurogene team on reaching alignment with the FDA and thank the Rett community at large for their support in advancing this program. The future is bright!' 'Our partnership with Neurogene reflects a shared commitment to accelerating meaningful treatments for Rett syndrome,' said Laura Hameed, Chief Executive Officer of the International Rett Syndrome Foundation (IRSF). 'The use of IRSF's Natural History Study data to support the NGN-401 gene therapy registrational trial design is a powerful example of how families accelerate and streamline therapeutic development. We are grateful to the families that contributed to the NHS, and to the families participating in this trial whose time and commitment are essential to evaluating gene therapy for Rett syndrome.' Phase 1/2 Trial Data Support Registrational Trial Design Previously disclosed clinical data from the Phase 1/2 study* support the registrational trial design. Data showed improvements in Rett syndrome assessments with an aggregate of 23 developmental milestones/skills acquired in the first four participants. Specifically, the four participants: Each achieved a clinically meaningful rating of 2, or 'much improved,' on the CGI-I; and Collectively achieved 23 developmental milestones/skills in the core clinical domains of Rett syndrome – hand function/fine motor, communication/language, and ambulation/gross motor, including: Participant 1 gained 10 skills across multiple domains by 15 months post-dose; Participant 2 gained 8 skills across multiple domains by 12 months post-dose; Participant 3 gained 3 skills across multiple domains by 9 months post-dose; and Participant 4 gained 2 skills by 3 months post-dose. *Efficacy data from the first four participants; as of data cut-off date of October 17, 2024 Phase 1/2 Trial Enrollment and Update Neurogene previously shared that five additional participants had been dosed in the Phase 1/2 portion of the NGN-401 clinical trial during the first half of 2025. A total of 10 participants have received the 1E15 vg dose. There has been no evidence of hemophagocytic lymphohistiocytosis (HLH) / hyperinflammatory syndrome in any participant at the 1E15 vg dose level. Neurogene remains on track to share updated clinical efficacy and safety data in the second half of 2025. Cash Runway Update As of March 31, 2025, Neurogene had cash, cash equivalents, and investments of $292.6 million. The Company's current budget reflects a reallocation of capital from pipeline development to support pivotal trial activities for NGN-401. Neurogene now expects its existing resources will be sufficient to fund operations into early 2028. About Neurogene The mission of Neurogene is to treat devastating neurological diseases to improve the lives of patients and families impacted by these rare diseases. Neurogene is developing novel approaches and treatments to address the limitations of conventional gene therapy in central nervous system disorders. This includes selecting a delivery approach to maximize distribution to target tissues and designing products to maximize potency and purity for an optimized efficacy and safety profile. The Company's novel and proprietary EXACT™ transgene regulation platform technology allows for the delivery of therapeutic levels while limiting transgene toxicity associated with conventional gene therapy. Neurogene has constructed a state-of-the-art gene therapy manufacturing facility in Houston, Texas. CGMP production of NGN-401 was conducted in this facility and will support pivotal clinical development activities. For more information, visit About NGN-401 NGN-401 is an investigational AAV9 gene therapy being developed as a one-time treatment for Rett syndrome. It is the first clinical candidate to deliver the full-length human MECP2 gene under the control of Neurogene's EXACT™ transgene regulation technology. EXACT technology is an important advancement in gene therapy for Rett syndrome, specifically because the disorder requires a treatment approach that enables targeted levels of MECP2 transgene expression without causing overexpression-related toxic effects associated with conventional gene therapy. NGN-401 was selected by the U.S. Food and Drug Administration (FDA) for its START Pilot Program and has also received Regenerative Medicine Advance Therapy (RMAT) designation, orphan drug designation, Fast Track designation and rare pediatric designation from the FDA. Neurogene was previously granted an INTERACT meeting with the FDA regarding the EXACT technology. NGN-401 also received Priority Medicines (PRIME) designation, orphan designation and advanced therapy medicinal product designation from the European Medicines Agency (EMA) and the Innovative Licensing and Application Pathway (ILAP) designation from the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA). Cautionary Note Regarding Forward-Looking Statements Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: trial designs, clinical development plans and timing for NGN-401, including elements of the registrational clinical study trial design subject to final approval of the FDA, such as the proposed number of participants in the Embolden trial and the addition of a six month endpoint, and timing of the conversion of the NGN-401 Phase 1/2 clinical trial to a registrational clinical trial; expected future interactions with or positions of the FDA; the safety, tolerability and efficacy of NGN-401; the potential for success of the Embolden registrational clinical trial for NGN-401 for the treatment of Rett syndrome; expected timing for additional interim data from the Company's NGN-401 Phase 1/2 trial for Rett Syndrome; the effectiveness of the monitoring and treatment protocol for HLH in Neurogene's Phase 1/2 clinical trial of NGN-401; and the time period over which existing cash resources may be sufficient to fund the Company's operations. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as 'may,' 'will,' 'should,' 'would,' 'expect,' 'anticipate,' 'plan,' 'likely,' 'believe,' 'estimate,' 'project,' 'intend,' 'on track,' and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene's control. Such risks, uncertainties and assumptions include, among other things, uncertainties regarding interactions with and feedback received from the FDA staff responsible for approving the design of our registrational trial and the risks and uncertainties identified under the heading "Risk Factors" included in Neurogene's Annual Report on Form 10-K for the year ended December 31, 2024, filed with the Securities and Exchange Commission (SEC) on March 24, 2025, Neurogene's Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, filed with the SEC on May 9, 2025, and other filings that the Company has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.
Yahoo
24-06-2025
- Business
- Yahoo
Jefferies Maintains a Buy on Taysha Gene Therapies (TSHA) With an $8 PT
Taysha Gene Therapies, Inc. (NASDAQ:TSHA) is one of the 13 Best Long-Term Penny Stocks to Buy According to Analysts. In a report released on June 11, Maury Raycroft from Jefferies maintained a Buy rating on Taysha Gene Therapies, Inc. (NASDAQ:TSHA) with a price target of $8.00. The analyst based the rating on the company's promising potential, stating that its Rett gene therapy program is exhibiting notable progress. Updated clinical data suggests continuous patient benefits over time, especially in the case of high-dose treatments. A laboratory technician preparing a gene therapy sample in a sterile environment. Raycroft stated that although they are conservatively evaluated, the analysis of milestone gains points towards significant improvements in patient outcomes. Another factor supporting the Buy rating for Taysha Gene Therapies, Inc. (NASDAQ:TSHA) is the deep reduction in the R-MBA rating scale, which the analyst considers a positive surprise reflecting the effect of the therapy on a granular and well-developed scale. Caregivers are also showing considerable interest in taking part in gene therapy trials, which, according to Raycroft, supports the potential for quick enrollment in pivotal studies. Taysha Gene Therapies, Inc. (NASDAQ:TSHA) is a clinical-stage biotechnology company that develops and commercializes adeno-associated viruses (AAV) based gene therapies to treat monogenic diseases of the central nervous system. The company is also involved in the development of multiple gene therapy platforms, including AAV9 Discovery, Novel Capsid, and AAV Redosing. While we acknowledge the potential of TSHA as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: The Best and Worst Dow Stocks for the Next 12 Months and 10 Unstoppable Stocks That Could Double Your Money. Disclosure: None. Error 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
24-06-2025
- Business
- Yahoo
Jefferies Maintains a Buy on Taysha Gene Therapies (TSHA) With an $8 PT
Taysha Gene Therapies, Inc. (NASDAQ:TSHA) is one of the 13 Best Long-Term Penny Stocks to Buy According to Analysts. In a report released on June 11, Maury Raycroft from Jefferies maintained a Buy rating on Taysha Gene Therapies, Inc. (NASDAQ:TSHA) with a price target of $8.00. The analyst based the rating on the company's promising potential, stating that its Rett gene therapy program is exhibiting notable progress. Updated clinical data suggests continuous patient benefits over time, especially in the case of high-dose treatments. A laboratory technician preparing a gene therapy sample in a sterile environment. Raycroft stated that although they are conservatively evaluated, the analysis of milestone gains points towards significant improvements in patient outcomes. Another factor supporting the Buy rating for Taysha Gene Therapies, Inc. (NASDAQ:TSHA) is the deep reduction in the R-MBA rating scale, which the analyst considers a positive surprise reflecting the effect of the therapy on a granular and well-developed scale. Caregivers are also showing considerable interest in taking part in gene therapy trials, which, according to Raycroft, supports the potential for quick enrollment in pivotal studies. Taysha Gene Therapies, Inc. (NASDAQ:TSHA) is a clinical-stage biotechnology company that develops and commercializes adeno-associated viruses (AAV) based gene therapies to treat monogenic diseases of the central nervous system. The company is also involved in the development of multiple gene therapy platforms, including AAV9 Discovery, Novel Capsid, and AAV Redosing. While we acknowledge the potential of TSHA as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: The Best and Worst Dow Stocks for the Next 12 Months and 10 Unstoppable Stocks That Could Double Your Money. Disclosure: None. Sign in to access your portfolio
Daily Mirror
21-06-2025
- Health
- Daily Mirror
Parents worried over dreaded 8-words are saved from 'apocalyptic life'
Simon and Helen's daughter Ruby-Mae has severe complex needs. They say without Noah's Ark Children's Hospice, which faces rising costs, that life would be 'apocalyptic'. In 2023, Simon Morley and his partner Helen Bolger heard the words no parent wants to hear. "It was a scary moment when one of Ruby's epilepsy consultants told us, 'your daughter would benefit from the children's hospice'," Simon, 46, says. "But from the moment we walked through that door at Noah's Ark, it was such positivity and professionalism – it was an amazing set-up. We are part of the furniture now." Simon and Helen's seven-year-old daughter Ruby-Mae was diagnosed with Rett syndrome, a rare genetic neurological and developmental disorder, at the age of two-and-a-half. She has severe complex needs, uses a wheelchair and needs an eye-gaze device to communicate, as she is non-verbal. For her family, life would now be unthinkable without the hospice. "Boy, has this hospice changed all our lives," Simon, who has put his micro-brewery business on pause to become Ruby's primary carer. "Noah's Ark is like the oxygen mask on a plane, it helps us to then help Ruby. It is wrap-around care for the whole family. Ruby absolutely loves it. Without The Ark being there, her life would be so diminished, she would not be the happy girl she is. Our lives would be apocalyptic without Noah's Ark." Noah's Ark Children's Hospice has been caring for children since 2006 but this year found itself facing severe financial challenges as an unintended consequence of National Insurance rises introduced by the Treasury. There are over 50 children's hospices and 200 adult hospices in the UK, providing palliative care and end-of-life support to over 300,000 people a year. "The NI increase is expected to cost Noah's Ark approximately £100,000 per year" says CEO, Sophie Andrews OBE. "Despite this, we are committed to continuing our care for children with life-limiting and life-threatening conditions." Noah's Ark Children's Hospice says that no services are being immediately impacted. "However, this unbudgeted cost places additional strain on our ability to grow and scale our services to reach more babies, children, and young people, and their families." In 2024/25 it cost £7.1million to run the hospice – which provides paediatric palliative care via specialist nurses and carers, creative therapies, a specialist play team, and a Holiday Lodge in Mersea Island, Essex – services which already rely heavily on voluntary income. Katie and Tom Morsbach's daughter Leia is six-years-old. A big sister to brothers Robin and Dylan, Leia has mitochondrial disease and severe epilepsy. Katie and Tom are opera singers. Tom donated one of his kidneys to Leia. In 2019, the Great Ormond Street Palliative Care Team referred the family to Noah's Ark Children's Hospice. "The MRI came back at GOSH, and it was pretty bad." Katie, 37, says. "The palliative care team was in the room with us pretty much straight away, and we were handed leaflets for the hospice. It's a grieving process. You have all these hopes and dreams for your children and in one fell swoop it goes out of the window. "Leia is amazing, she's always smiling, she loves life. She has been through a lot – she's had dozens of operations and spent months at a time in hospital. She has taught us to value things in life and to make the most of the present day. We were told she had just months to live but she's beaten all expectations. "In the beginning we felt isolated, it was just awful, I couldn't even talk to my best friend or family because they didn't understand. My issues were different to other first-time mums, so chatting to parents at the hospice is a relief, it's good for your mental health. Sometimes you feel like you're going crazy, but you're not, you're just dealing with things that are really difficult. We have a hospice family link worker, who I can call up for a chat." Katie, who runs a singing and drama school, adds: "When we first heard the word, 'hospice', it sounded horrible, but when Leia attended her first music therapy session, it felt so positive – it isn't what I imagined a hospice to be. Noah's Ark supports my whole family. It is not a luxury, the hospice is a lifeline, they've helped us in good and difficult times. I think people are shocked when they hear it costs something like £18k a day to run, but if you think about the breadth of services they run, not just at the hospice, but in homes too. "I've made lots of friends, we attend mum's evening, dad night's out, my boys have made friends with other siblings. If I'm totally honest, Noah's Ark is the most important place with the most important people for us. We moved house to be closer. They give us the opportunity to create amazing memories. I've been able to leave Leia there, when I gave birth to the boys, because they can provide the specialist care Leia needs. A good few times a year, we use their respite service." Ruby's dad Simon says he and his partner Helen, 44, a commissioner for health and social care services in London, can't imagine life without Noah's Ark Children's Hospice. "Due to Ruby's severe complex needs, we're really limited on what we can do," he says. "So, we spend a lot of time here, she absolutely loves it. "They have an amazing playground that allows you to wheel the wheelchairs onto the swing, they host events, there is a play team and soft play. One of Ruby's highlights was playing on the trampoline with an Arsenal player, and she's played a bit of football with Tottenham Hotspur's women's team. Noah's Ark has a huge bank of great volunteers who come into the home, it allows me to wash up, put the laundry on and cook. The only time we feel safe leaving Ruby is with Noah's Ark or at school." A government spokesperson said: "The government recognises the incredible work that children and young people's hospices do across the country. We are investing £100 million to improve hospices' facilities and a further £26 million specifically for children's hospices this year, the biggest investment in hospices in a generation. We are also working to make sure the palliative and end of life care sector is sustainable in the long term. The decision to increase employer National Insurance contributions allowed us to increase investment in health and care by £26 billion this year." Helen says the hospice is invaluable. "We use the respite service, Ruby loves it, and for us that two-day weekend is so valuable, it allows us to be us and not carers. We spent 24 hours just sleeping and recovering, we can enjoy a meal out or the cinema and feel normal. Without the hospice we would be a wreck, and miserable." She adds: "Ruby is such a happy, sociable little girl, who loves joking around. Her care is exhausting, there is no let-up, but her smile makes it all worth-while. Before Noah's Ark, all we could see was a negative future. Now we're happy again, we smile."
Yahoo
18-06-2025
- Business
- Yahoo
Craig-Hallum bullish on Neurogene, initiates with a Buy
As previously reported, Craig-Hallum analyst Albert Lowe initiated coverage of Neurogene (NGNE) with a Buy rating and $50 price target. The firm notes the company is developing NGN-401, an AAV gene therapy for Rett syndrome, a rare and devastating neurodevelopmental disease that impacts girls beginning in infancy and is characterized by developmental regression and symptoms that require lifelong care. Craig-Hallum believes Neurogene is undervalued and that NGN-401 has an elegant design that gives it the upper hand over Taysha's (TSHA) competing program TSHA-102. Looking ahead, the firm thinks NGN-401 is well-positioned to show efficacy advantages over TSHA-102 in the second half of 2025 clinical data update and be the leading gene therapy for Rett. This represents a significant commercial opportunity with pricing of over $2M in a U.S. population of about 6k patients, and Albert models WW NGN-401 sales reaching $1B in 2033, including U.S. sales of about $700M. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See today's best-performing stocks on TipRanks >> Read More on NGNE: Disclaimer & DisclosureReport an Issue Neurogene initiated with a Buy at Craig-Hallum Neurogene Approves Amendments at Annual Stockholders Meeting Neurogene price target raised to $26 from $22 at BMO Capital Neurogene Highlights Gene Therapy Advancements in New Update Neurogene Reports Q1 2025 Financial Progress and Updates Error 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
