TransCon® hGH Boosts TransCon® CNP Results in Kids with Achondroplasia by Week 26
Ascendis Pharma A/S (NASDAQ:ASND) is among the 13 Biotech Stocks with Huge Upside Potential. It reported interim Week 26 data from its Phase 2 COACH Trial, confirming that combining TransCon hGH (lonapegsomatropin) with TransCon CNP (navepegritide) raised annualized growth velocity (AGV) and height Z-scores in children with achondroplasia.
A close-up view of a hand manipulating a syringe while delivering TransCon CNP into a tumor.
A mean AGV of 9.14 cm/year and a +0.53 Z-score rise were attained by treatment-naïve youngsters (N=12). Children (N=9) treated with TransCon CNP in the past achieved an AGV of 8.25 cm/year with a +0.44 Z-score improvement. The majority of side effects were modest, and safety remained consistent with monotherapies.
The COACH Trial is the first to assess this combination in kids between the ages of two and eleven. While TransCon CNP is undergoing FDA priority review, TransCon hGH is approved as SKYTROFA® for juvenile growth hormone insufficiency. The treatment focuses on the FGFR3-CNP signaling imbalance that is at the heart of achondroplasia, a disorder that affects more than 250,000 people worldwide.
It is anticipated that the results of week 52 and a scheduled Phase 3 trial in Q4 2025 will further clarify efficacy and long-term benefits.
The biopharmaceutical business Ascendis Pharma A/S (NASDAQ:ASND) uses its TransCon technology platform to significantly improve patient outcomes.
While we acknowledge the potential of ASND 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: 10 High-Growth EV Stocks to Invest In and 13 Best Car Stocks to Buy in 2025.
Disclosure. None.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Business Insider
44 minutes ago
- Business Insider
KalVista says FDA will not meet PDUFA goal date for Sebetralstat NDA
KalVista (KALV) Pharmaceuticals nnounced that the U.S. Food and Drug Administration has notified the company that it will not meet the PDUFA goal date for the New Drug Application for sebetralstat, the company's investigational oral on-demand treatment for hereditary angioedema. The FDA notified the company on June 13, 2025, that the previously disclosed June 17, 2025 PDUFA goal date will not be met due to heavy workload and limited resources. The FDA indicated that it expects to deliver a decision within approximately four weeks. The FDA has not requested additional data or studies and has not raised any concerns regarding the safety, efficacy or approvability of sebetralstat. KalVista has addressed all prior information requests in a timely manner, and the company believes the only remaining item under FDA review is the finalization of the labelling. 'We are disappointed by this delay, most importantly because we know how much people living with HAE are looking forward to an oral on-demand option to treat their HAE attacks,' said Ben Palleiko, CEO of KalVista. 'At the same time, we remain confident in the near-term approval of sebetralstat. We are continuing to work closely with the FDA to support the completion of their review. Our commitment to bringing this important therapy to people living with HAE remains unwavering.'
Associated Press
an hour ago
- Associated Press
ROCKET ALERT: Bragar Eagel & Squire, P.C. Announces that a Class Action Lawsuit Has Been Filed Against Rocket Pharmaceuticals, Inc. and Encourages Investors to Contact the Firm
NEW YORK, June 13, 2025 (GLOBE NEWSWIRE) -- Bragar Eagel & Squire, P.C., a nationally recognized stockholder rights law firm, announces that a class action lawsuit has been filed against Rocket Pharmaceuticals, Inc. ('Rocket' or the 'Company') (NASDAQ:RCKT) in the United States District Court for the District of New Jersey on behalf of all persons and entities who purchased or otherwise acquired Rocket securities between February 27, 2025 to May 26, 2025, both dates inclusive (the 'Class Period'). Investors have until August 11, 2025 to apply to the Court to be appointed as lead plaintiff in the lawsuit. Click here to participate in the action. According to the complaint, defendants provided overwhelmingly positive statements to investors while, at the same time, disseminating materially false and misleading statements and/or concealing material adverse facts concerning the true state of RP-A501's safety and clinical trial protocol; notably, that Rocket knew Serious Adverse Events (SAEs), including death of participants enrolled in the study, were a risk. In particular, Rocket amended the trial's protocol to introduce a novel immunomodulatory agent to the pretreatment regimen without providing this critical update to shareholders. Such statements absent these material facts caused Plaintiff and other shareholders to purchase Rocket's securities at artificially inflated prices. On May 27, 2025, Rocket announced that the FDA placed a clinical hold on the RP-A501 Phase 2 pivotal study after at least one patient suffered a Serious Adverse Event (SAE), ultimately, death, while enrolled in the study following a substantive amendment to the protocol that the Company failed to disclose to investors at the time management made the revision. In fact, Rocket stated that, while the patient was dosed in May, the decision to amend the protocol was made 'several months' earlier. Despite this, Rocket made no attempt to alert investors or the public to the change until after the SAE occurred. Following this news, the price of Rocket's common stock declined dramatically. From a closing market price of $6.27 per share on May 23, 2025, Rocket's stock price fell to $2.33 per share on May 27, 2025, a decline of about 37% in the span of just a single trading day. If you purchased or otherwise acquired Rocket shares and suffered a loss, are a long-term stockholder, have information, would like to learn more about these claims, or have any questions concerning this announcement or your rights or interests with respect to these matters, please contact Brandon Walker or Marion Passmore by email at [email protected], telephone at (212) 355-4648, or by filling out this contact form. There is no cost or obligation to you. About Bragar Eagel & Squire, P.C.: Bragar Eagel & Squire, P.C. is a nationally recognized law firm with offices in New York, California, and South Carolina. The firm represents individual and institutional investors in commercial, securities, derivative, and other complex litigation in state and federal courts across the country. For more information about the firm, please visit Attorney advertising. Prior results do not guarantee similar outcomes. Follow us for updates on LinkedIn, X, and Facebook, and keep up with other news by following Brandon Walker, Esq. on LinkedIn and X. Contact Information: Bragar Eagel & Squire, P.C. Brandon Walker, Esq. Marion Passmore, Esq. (212) 355-4648
Yahoo
3 hours ago
- Yahoo
DRI Healthcare Trust Comments on FDA Delay for KalVista Pharmaceuticals' Sebetralstat PDUFA Goal Date Due to FDA Resourcing Constraints
- FDA decision anticipated within four weeks - TORONTO, June 13, 2025 /CNW/ - DRI Healthcare Trust (TSX: (TSX: DHT.U) (the "Trust") today announced KalVista Pharmaceuticals ("KalVista") has disclosed that it has received notice from the U.S. Food and Drug Administration ("FDA") that the FDA will not meet the June 17, 2025 PDUFA goal date for the New Drug Application (NDA) for sebetralstat, due to a heavy workload and agency resourcing issues. KalVista commented that the FDA has not requested additional data or studies and has communicated to KalVista that it anticipates delivering a decision within approximately four weeks. KalVista issued its own press release with the announcement earlier today (link). About DRI Healthcare Trust The Trust is managed by DRI Capital Inc., a pioneer in global pharmaceutical royalty monetization. Since its initial public offering in 2021, the Trust has deployed more than $1.0 billion, acquiring more than 25 royalties on 20-plus drugs, including Eylea, Orserdu, Omidria, Spinraza, Stelara, Vonjo, Zejula and Zytiga. The Trust's units are listed and trade on the Toronto Stock Exchange in Canadian dollars under the symbol " and in U.S. dollars under the symbol "DHT.U". To learn more, visit or follow us on LinkedIn. SOURCE DRI Healthcare Trust View original content to download multimedia:
