Latest news with #PTC518


Business Wire
16-06-2025
- Business
- Business Wire
PTCT Investors Have Opportunity to Join PTC Therapeutics, Inc. Fraud Investigation with the Schall Law Firm
LOS ANGELES--(BUSINESS WIRE)-- The Schall Law Firm, a national shareholder rights litigation firm, announces that it is investigating claims on behalf of investors of PTC Therapeutics, Inc. ('PTC' or 'the Company') (NASDAQ: PTCT) for violations of the securities laws. The investigation focuses on whether the Company issued false and/or misleading statements and/or failed to disclose information pertinent to investors. PTC issued a press release on May 5, 2025, 'announc[ing] results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington's disease (HD) patients.' According to the Company, 'the study met its primary endpoint of reduction in blood Huntingtin (HTT) protein levels (p<0.0001) at Week 12 and favorable safety and tolerability.' However, analysts who reviewed the data believe that the Company would need to conduct a Phase 3 study to determine if the treatment actually slows HD. Based on this news, shares of PTC fell by more than 18.6% on the same day. If you are a shareholder who suffered a loss, click here to participate. We also encourage you to contact Brian Schall of the Schall Law Firm, 2049 Century Park East, Suite 2460, Los Angeles, CA 90067, at 310-301-3335, to discuss your rights free of charge. You can also reach us through the firm's website at or by email at bschall@ The Schall Law Firm represents investors around the world and specializes in securities class action lawsuits and shareholder rights litigation. This press release may be considered Attorney Advertising in some jurisdictions under the applicable law and rules of ethics.
Yahoo
07-05-2025
- Business
- Yahoo
Why Did PTC Therapeutics Stock Fall On Monday Despite Mid-Stage Study For Huntington's Hits Primary Goal?
PTC Therapeutics, Inc. (NASDAQ:PTCT) on Monday announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington's disease (HD) patients. Huntington's disease is a progressive neurodegenerative disorder caused by mutation of the huntingtin (HTT) gene, leading to the breakdown of brain cells. It is characterized by uncontrolled movements (chorea), cognitive decline, and psychiatric disturbances. The study met its primary endpoint of reduction in blood Huntingtin (HTT) protein levels (p<0.0001) at Week 12 and favorable safety and tolerability. In addition, the 12-month data from the Stage 2 patients are consistent with the previously reported dose-dependent lowering of HTT protein and dose-dependent trends across clinical scales. Also Read: European Commission Declines To Renew Marketing Authorization Of Translarna Duchenne Muscular Disorder 'We look forward to discussions on the next development and regulatory steps, including the potential for accelerated approval as we work to potentially bring the first disease-modifying therapy...,' said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. Results from the full 12-month cohort demonstrate dose-dependent lowering in blood HTT levels, with 23% at the 5mg dose level for both Stage 2 and 3 patients and 39% and 36% at the 10mg dose level for Stage 2 and 3 patients, respectively. For Stage 2 patients, there were dose-dependent trends of benefit on clinical scales, including the Composite Unified Huntington's Disease Rating Scale (cUHDRS) and Total Motor Score (TMS) subscale. For Stage 3 patients, trends favored the 5mg dose group relative to placebo but not the 10mg dose group, suggesting that treatment effect may differ in Stage 3 patients relative to Stage 2 patients. It seems investors are reacting to this as the stock is trading almost 20% lower during the premarket session. PTC518 showed a favorable safety and tolerability profile for all dose levels and disease stages with no treatment-related serious adverse events or neurofilament light chain protein (NfL) spikes. In addition, 24-month treatment data from the patients on whom data were shared last year (N=21) demonstrate signals of dose-dependent trends on the cUHDRS, Total Function Capacity (TFC), and Symbol Digit Modalities Test (SDMT) subscales when compared to a propensity-matched natural history cohort from the ENROLL-HD Registry. At Month 24, there was also a dose-dependent lowering of plasma NfL from a baseline of -8.9% (nominal p=0.12) for the 5mg dose level and -14% (nominal p=0.03) for the 10mg dose level.
Yahoo
07-05-2025
- Business
- Yahoo
PTC Therapeutics price target lowered to $57 from $65 at RBC Capital
RBC Capital lowered the firm's price target on PTC Therapeutics (PTCT) to $57 from $65 and keeps an Outperform rating on the shares after data from the company's phase 2 PIVOT-HD study of PTC518 in stage 2 and stage 3 Huntington's disease patients. The firm acknowledges some possible signals for '518 in the pre-defined, mechanistically rational earlier-stage population, good safety, which is reassuring, and understandable limitations with HD heterogeneity and to clinical and biomarker assays. Still, RBC believes the entirety of the results just does not look quite as promising as the earlier cut. While it had not expected statistical significance clinical score benefits and always expected PTC would need some phase 3 results to satisfy FDA, today's more mixed data may push regulators to want more from phase 3 prior to approval, and adds risk to future studies, the firm argues. Protect Your Portfolio Against Market Uncertainty Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See the top stocks recommended by analysts >> Read More on PTCT: Disclaimer & DisclosureReport an Issue
Yahoo
05-05-2025
- Business
- Yahoo
PTC stock slides on new data for Huntington's drug
This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. Shares in PTC Therapeutics fell by double-digits Monday morning as investors parsed new clinical trial results for an important Huntington's disease drug the biotechnology company has been developing. Results from the study showed PTC's drug lowered levels of a target protein associated with Huntington's, confirming an earlier readout. According to the company, the data also suggested 'trends' in benefit on scales designed to assess clinical outcomes. PTC said it would discuss with regulators whether it could seek an accelerated approval. However, analysts expect the company will need to conduct a Phase 3 study to better establish whether the treatment is actually slowing disease. The results PTC released Monday give a fuller look at a Phase 2 study dubbed PIVOT-HD, which randomized Huntington's patients to receive either placebo or one of two doses of the company's drug, PTC518. Treatment with PTC518 hit the study's main goal, which measure blood levels of a protein called huntingtin at 12 weeks. That lowering was then maintained through one year, the updated data show. Normally, huntingtin protein plays a variety of helpful functions in the brain. But when mutated in Huntington's disease, the protein can clump together in a manner that's toxic to nerve cells. Treatments like PTC518 are designed to degrade the nucleic acid blueprints that encode for the protein, thereby lowering levels. However, linking PTC518's effect on huntingtin protein to clinical benefit is harder to assess. For patients who were in an earlier, 'Stage 2' of disease, the data showed 'dose-dependent trends of benefit' versus placebo on a key scale measuring disease progression, PTC said. Among later, 'Stage 3' patients, there were similar trends in the low dose group, but not among patients given the higher dose. PTC said this might suggest a different treatment effect in Stage 3 patients versus Stage 2 individuals. The company also presented interim, 24-month results that compared study participants to a natural history control group. There, too, PTC said the data hinted at a clinical benefit. Favorable trends may not be enough to prove PTC518 is worthy of regulatory approval, though. 'There are no mentions of any associations between mHTT lowering and clinical outcomes, which we think is the most important aspect of this data readout,' wrote Leerink Partners analyst Joseph Schwartz in a client note Monday. 'As a reminder, the [Food and Drug Administration] told [PTC] that while they were aligned with [huntingtin] lowering in principle to support an accelerated approval pathway, they wanted to see associations with [huntingtin] lowering and clinical outcomes.' Kristen Kluska, an analyst at Cantor Fitzgerald, added in a separate note that she thought 'the market reaction today seems more focused on the potential for [accelerated approval] vs. focusing on whether or not these data are de-risking.' Shares in PTC were trading down by about 18% near mid-day Monday. Recommended Reading Novartis wagers billions of dollars on PTC Huntington's drug Sign in to access your portfolio