IPS HEART Granted Orphan Drug Designation in the EU for Becker Muscular Dystrophy
While we are grateful to the dozens of BMD patients who have emailed us directly for information on our upcoming clinical trials, to date we are initially focused on advancing GIVI-MPCs clinically in a Phase I/II DMD clinical trial until we can secure additional partners to help us advance clinically in BMD. IPS HEART is currently under formal CDA with certain large pharmaceutical companies for a possible partnership that will also allow us to advance GIVI-MPCs clinically in BMD as we have already successfully produced GIVI-MPCs via large scale suspension based bioreactor manufacturing which can supply the large patient population in the future.
About IPS HEART/GIVI-MPCs
GIVI-MPCs derived from pluripotent stem cells form new skeletal muscle with 100% full-length human dystrophin for DMD and BMD. IPS HEART has also developed ISX-9-CPCs, a human iPSC-derived cardiac therapy for treatment of Duchenne cardiomyopathy and heart failure that develops new heart muscle.
About Becker & Duchenne Muscular Dystrophy
BMD is a muscular disease and DMD is a fatal neuromuscular disease.
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
10 minutes ago
- Yahoo
Sarepta stock faces multiple downgrades and questions of solvency amid Elevidys pause
Sarepta Therapeutics' (SRPT) stock has been hammered in the past week after the company was forced to pause shipments of its top-selling Duchenne muscular dystrophy (DMD) therapy Elevidys — throwing the future of the company into question. Elevidys netted $821 million of the company's $1.78 billion in 2024 revenue and contributed to more than 50% of the company's $513 million in second quarter revenue this year. It's the company's top-selling product. It's why seven analysts have downgraded the stock since Friday, including Bank of America's Tazeen Ahmad on Wednesday. Ahmad downgraded the stock to Underperform and revised the firm's price target to $10 from $20. The stock is currently trading at $13.90 per share and closed down more than 2% on Wednesday. Sarepta "has requested a meeting with FDA and might have more visibility on length of the pause once FDA responds to their submission for a revised label," Ahmad wrote in a note to clients. "In short, we think this could impact the Elevidys brand further, limiting uptake following highly focused media attention as well as a renewed chance of Elevidys being removed from the US market." Leerink Partners downgraded the stock last week from Outperform to Market Perform. Analyst Joseph Schwartz wrote in a note to clients on Monday that how long the pause lasts is important for future modeling. "This decision is also roughly aligned with our published SRPT model, which assumes a significant slowing of sales in the third quarter, zero sales in the fourth quarter, and a resumption of shipments in early 2026," Schwartz wrote. "We lack visibility into how long this pause may last, or what the intermediate scenarios are from here (e.g., a narrower approval)." The stock now has five Buy, 17 Hold, and four Sell ratings. Domino effect The technology to deliver the therapy in Elevidys is at the center of the issue facing the company. A 51-year-old, late-stage patient in a phase 1 trial of a different muscular dystrophy therapy, known only by its trial name, SRP-9004, died from liver toxicity last month, but Sarepta failed to disclose the death — even as it announced it was discontinuing the trial as part of a restructuring last week. That began the domino effect of downgrades and the stock sell-off as analysts questioned why the company had kept quiet. On a call with investors Friday, CEO Doug Ingram defended his reasoning for not mentioning it. "We did not discuss this matter in our call on Wednesday because it was neither material nor central to the topics at hand on Wednesday," Ingram said. In a note following the call, Leerink's Schwartz wrote, "Is This Material?!? All Credibility Lost." It's the third death linked to a gene therapy from the company this year and a reason why the FDA previously required the company to add new warning labels to Elevidys, which is part of the pause in shipments currently. But now, the company's future is in flux as its revenue stream has been paused for an undetermined amount of time. Jefferies analyst Andrew Tsai, one of the few who has maintained a Buy rating, said the company could defend the therapy as sicker and older patients have been more negatively impacted, while younger patients who are still able to move independently have benefited. "Prior to the third death, SRPT's internal stress tests suggested the DMD franchise could 'floor' at $1.4B annually into 2027, including $500M+ for Elevidys in ambulatory," Tsai wrote in a note to clients Sunday. "For Elevidys, one can point to how the deaths have occurred only in older patients." "More cost reductions is possible too," Tsai noted of how Sarepta could end up handling the hit. Anjalee Khemlani is the senior health reporter at Yahoo Finance, covering all things pharma, insurance, provider services, digital health, PBMs, and health policy and politics. That includes GLP-1s, of course. Follow Anjalee as AnjKhem on social media platforms X, LinkedIn, and Bluesky @AnjKhem. Click here for in-depth analysis of the latest health industry news and events impacting stock prices
Business Wire
11 minutes ago
- Business Wire
Chimera Investment Corporation Sets Date for Second Quarter 2025 Earnings Release and Conference Call
NEW YORK--(BUSINESS WIRE)--Chimera Investment Corporation (NYSE: CIM) (the "Company") announced today that it will release its financial results for the second quarter ended June 30, 2025, on Wednesday, August 6, 2025, prior to 7:00 a.m. Eastern Daylight Time (EDT). The Company will host a conference call and live audio webcast to discuss the results on Wednesday, August 6, 2025, at 8:30 a.m. EDT. Call-in Number: U.S. Toll Free: (866) 604-1613 International: (201) 689-7810 Webcast: Conference Call Replay: U.S. Toll Free: (877) 660-6853 International: (201) 612-7415 Conference ID: 13754918 A replay of this call can be accessed through Wednesday, August 20, 2025. If you would like to be added to the e-mail distribution list, please visit click on News & Events, and complete the email notification form. About Chimera Investment Corporation Chimera is a publicly traded real estate investment trust, or REIT, that is primarily engaged in the business of investing for itself and for unrelated third parties through its investment management and advisory services in a diversified portfolio of real estate assets, including residential mortgage loans, Non-Agency RMBS, Agency RMBS, business purpose and investor loans, including RTLs, MSRs, and other real estate-related assets such as Agency CMBS, junior liens and HELOCs, equity appreciation rights, and reverse mortgages. Please visit for additional information about the Company.
Business Wire
11 minutes ago
- Business Wire
CoStar Group Names Grant Montgomery as National Director of Multifamily Analytics
ARLINGTON, Va.--(BUSINESS WIRE)--CoStar Group, Inc. (NASDAQ: CSGP), a leading provider of online real estate marketplaces, information, analytics, and 3D digital twin technology in the property markets, announced today the appointment of Grant Montgomery as National Director of Multifamily Analytics. The hire will augment the company's already powerful analytics across the U.S. multifamily sector, adding value for CoStar Group clients and the commercial real estate industry as a whole. In his new role, Montgomery will serve as CoStar Group's industry-facing representative for the multifamily sector, delivering the company's data-driven perspective and outlook on the market. As CoStar Group's subject matter expert on all multifamily themes, trends, and topics, Montgomery will use his extensive knowledge to uphold CoStar Group's commitment to providing quality analytics and insights for clients and the industry. 'We are thrilled to have Grant, a demonstrated, multi-faceted researcher, leader and strategic advisor, join the team,' said Galina Alexeenko, Vice President, CoStar Group Market Analytics. 'Grant's vast experience in real estate research and operations, coupled with CoStar's unparalleled multifamily data and analytics, will provide our clients with insights into the multifamily market's key trends and developments. We welcome this outstanding addition to CoStar Group's analytics team.' Montgomery joins CoStar Group with more than 25 years of real estate research and consulting experience. Previously serving as Vice President of Research for Elme Communities, Montgomery led the development of strategic quantitative market analysis that supported the company's transformation into a multifamily REIT, directly influencing portfolio strategies. Prior to his time at Elme, he served as Senior Vice President and Apartment Practice Director at Delta Associates, delivering critical insights into market conditions to developers, lenders, and public agencies. Montgomery is a frequent speaker at industry events on multifamily market conditions. About CoStar Group CoStar Group (NASDAQ: CSGP) is a global leader in commercial real estate information, analytics, online marketplaces, and 3D digital twin technology. Founded in 1986, CoStar Group is dedicated to digitizing the world's real estate, empowering all people to discover properties, insights, and connections that improve their businesses and lives. CoStar Group's major brands include CoStar, a leading global provider of commercial real estate data, analytics, and news; LoopNet, the most trafficked commercial real estate marketplace; the leading platform for apartment rentals; and the fastest-growing residential real estate marketplace. CoStar Group's industry-leading brands also include Matterport, a leading spatial data company whose platform turns buildings into data to make every space more valuable and accessible, STR, a global leader in hospitality data and benchmarking, Ten-X, an online platform for commercial real estate auctions and negotiated bids and OnTheMarket, a leading residential property portal in the United Kingdom. CoStar Group's websites attracted over 141 million average monthly unique visitors in the second quarter of 2025, serving clients around the world. Headquartered in Arlington, Virginia, CoStar Group is committed to transforming the real estate industry through innovative technology and comprehensive market intelligence. From time to time, we plan to utilize our corporate website as a channel of distribution for material company information. For more information, visit
