Latest news with #Rett
Yahoo
3 days ago
- Business
- Yahoo
Acadia Q2 Earnings Beat, Nuplazid & Daybue Sales Drive Revenue Growth
Acadia Pharmaceuticals ACAD reported second-quarter 2025 earnings of 16 cents per share, which beat the Zacks Consensus Estimate of 14 cents. In the year-ago quarter, the company had reported earnings of 20 cents per share. Acadia recorded total revenues of $264.6 million, which surpassed the Zacks Consensus Estimate of $260 million. ACAD's net product revenues comprise revenues generated from the sale of its two marketed products, Nuplazid (pimavanserin) and Daybue (trofinetide). Acadia's first drug, Nuplazid, is approved in the United States for the treatment of hallucinations and delusions associated with Parkinson's disease psychosis. ACAD's second product, Daybue, received approval in 2023 for treating Rett syndrome in adult and pediatric patients aged two years and older. The drug was launched in the United States in April 2023. Total revenues increased 9% year over year, driven by contributions from Daybue and continued growth in Nuplazid's market share. ACAD's Q2 Results in Detail Revenues from Nuplazid increased 7% year over year to $168.5 million in the second quarter of 2025, driven primarily by volume growth. Nuplazid sales beat the Zacks Consensus Estimate of $166 million as well as our model estimate of $164.8 million. Daybue recorded net product sales of $96.1 million in the reported quarter, up 14% year over year, driven by the growth in the drug's unit sales as Acadia shipped to more unique patients. Per the company, ACAD reached a new record high in the second quarter, with 987 unique patients receiving a Daybue shipment. The upward trend signals promising growth in both new patient starts and treatment persistence. The reported figure beat the Zacks Consensus Estimate of $93.7 million as well as our model estimate of $91.5 million. Year to date, shares of Acadia have rallied 29.7% compared with the industry's 0.2% growth. Image Source: Zacks Investment Research Research and development (R&D) expenses were $78 million, up 2% year over year. Selling, general and administrative (SG&A) expenses were $133.5 million, up 14% year over year. The increase can be primarily attributed to higher marketing costs to support the Nuplazid and Daybue franchises in the United States and investments for the planned expansion of the Daybue commercial team. Acadia had cash, cash equivalents and investments worth $762.0 million as of June 30, 2025, compared with $681.6 million as of March 31, 2025. ACAD Updates 2025 Financial Outlook Acadia now expects total revenues from the U.S. sales of its products to be in the range of $1.045-$1.095 billion, up from the previously guided range of $1.030-$1.095 billion. Nuplazid net product sales are now expected to be in the range of $665-$690 million in 2025, up from the previous guidance of $650-$690 million. U.S. sales of Daybue are expected to remain between $380 million and $405 million. R&D expenses in 2025 are projected to be in the range of $330-$350 million, while SG&A expenses are expected to be between $535 million and $565 million. ACAD's Key Updates A regulatory filing for trofinetide to treat Rett syndrome in adults and pediatric patients aged two years and above is currently under review in the EU. An approval in the EU is expected in the first quarter of 2026. Acadia reported completing enrollment in the phase III COMPASS PWS study of ACP-101 for Prader-Willi Syndrome (PWS). Top-line results are expected in the fourth quarter of 2025. Subject to the study's success, ACAD plans to submit a regulatory application to the FDA, seeking the approval of ACP-101 for PWS in the first quarter of 2026. During the reported quarter, the U.S. District Court in Delaware ruled in Acadia's favor concerning the '721 formulation patent for Nuplazid. The Court upheld Acadia's position on both infringement and validity in its patent litigation against Aurobindo Pharma Limited and other abbreviated new drug application filers. The judgment issued by the Delaware Court provides patent protection for the Nuplazid 34 mg capsule formulation until 2038. In June, Acadia received a favorable ruling from the U.S. Court of Appeals for the Federal Circuit, which upheld a December 2023 decision by the U.S. District Court for the District of Delaware validating the '740 composition of matter patent for Nuplazid. The ruling, stemming from Acadia's litigation against MSN, extends patent protection for the drug's composition into 2030. These are two huge wins for Acadia, collectively protecting the company's Nuplazid sales from generic erosion in the U.S. market for the Parkinson's disease indication. ACADIA Pharmaceuticals Inc. Price, Consensus and EPS Surprise ACADIA Pharmaceuticals Inc. price-consensus-eps-surprise-chart | ACADIA Pharmaceuticals Inc. Quote ACAD's Zacks Rank & Stocks to Consider Acadia currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the biotech sector are Arvinas ARVN, CorMedix CRMD and Immunocore IMCR. While CRMD and IMCR sport a Zacks Rank #1 (Strong Buy) each at present, ARVN currently carries a Zacks Rank #2 (Buy). You can see the complete list of today's Zacks #1 Rank stocks here. In the past 60 days, estimates for CorMedix's earnings per share have increased from 93 cents to 97 cents for 2025. During the same time, earnings per share estimates for 2026 have increased from $1.64 to $1.65. Year to date, shares of CRMD have rallied 47.5%. CorMedix's earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 25.82%. In the past 60 days, estimates for Immunocore's 2025 loss per share have narrowed from 86 cents to 68 cents. Loss per share estimates for 2026 have narrowed from $1.34 to $1.10 during the same period. IMCR stock has increased 8.8% year to date. Immunocore's earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 76.18%. In the past 60 days, estimates for Arvinas' 2025 loss per share have narrowed from $1.51 to $1.50. Loss per share estimates for 2026 have narrowed from $3.08 to $2.98 during the same period. ARVN stock has plunged 66.5% year to date. Arvinas' earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 82.09%. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report CorMedix Inc (CRMD) : Free Stock Analysis Report ACADIA Pharmaceuticals Inc. (ACAD) : Free Stock Analysis Report Arvinas, Inc. (ARVN) : Free Stock Analysis Report Immunocore Holdings PLC Sponsored ADR (IMCR) : Free Stock Analysis Report This article originally published on Zacks Investment Research ( Zacks Investment Research
Business Wire
5 days ago
- Business
- Business Wire
Acadia Pharmaceuticals to Participate in the Canaccord Genuity 45 th Annual Growth Conference
SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that it will participate in a fireside chat at Canaccord Genuity 45 th Annual Growth Conference on Wednesday, August 13, 2025 at 2:30 p.m. Eastern Time. A live webcast of Acadia's fireside chat will be accessible on the company's website, under the investors section and an archived recording will be available on the website for approximately one month following the presentation. About Acadia Pharmaceuticals Acadia is advancing breakthroughs in neurological and rare diseases to elevate life. Since our founding we have been working at the forefront of healthcare to bring vital solutions to people who need them most. We developed and commercialized the first and only FDA-approved drug to treat hallucinations and delusions associated with Parkinson's disease psychosis and the first and only approved drug in the United States and Canada for the treatment of Rett syndrome. Our clinical-stage development efforts are focused on Prader-Willi syndrome, Alzheimer's disease psychosis and multiple other programs targeting neuroscience and neuro-rare diseases. For more information, visit us at and follow us on LinkedIn and X.
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
