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Pfizer gene therapy trial for DMD misses primary goal

EditorNatashya Angelica
Published 06/13/2024, 04:56 AM
© Reuters

NEW YORK - Pfizer Inc. (NYSE: NYSE:PFE) disclosed today that its pivotal Phase 3 study of the investigational gene therapy fordadistrogene movaparvovec for Duchenne muscular dystrophy (DMD) did not meet its primary endpoint. The study, known as CIFFREO, aimed to improve motor function in boys aged 4 to 7 with DMD but failed to show a significant difference compared to a placebo.

The trial's primary goal was to assess changes in motor abilities using the North Star Ambulatory Assessment (NSAA) one year after treatment. Secondary endpoints, such as the 10-meter run/walk and time to rise from the floor, also did not exhibit significant improvements in the treatment group.

Despite the disappointing outcomes, the safety profile of fordadistrogene movaparvovec was considered manageable, with most adverse events being mild to moderate in severity. Serious adverse events related to the treatment generally responded to medical intervention.

"We are extremely disappointed that these results did not demonstrate the relative improvement in motor function that we had hoped," stated Dan Levy, MD, PhD, Development Head for Duchenne muscular dystrophy at Pfizer. He emphasized the company's commitment to sharing detailed findings from the CIFFREO trial with the medical and patient advocacy communities to inform future DMD research and treatment development.

Pfizer will continue to monitor all participants enrolled in the CIFFREO study and is currently evaluating the next steps for the gene therapy program. The company also noted that the study is on a dosing pause due to a fatal serious adverse event in a related Phase 2 trial named DAYLIGHT.

DMD is a severe genetic disorder characterized by progressive muscle degeneration, typically manifesting in early childhood and primarily affecting boys. The disease leads to loss of ambulation and impacts heart and respiratory muscles, often resulting in premature death.

Pfizer, a global biopharmaceutical company, is known for its contributions to health care, including the development of innovative medicines and vaccines. The company has stated that it does not plan to update forward-looking statements in this release as new information or future events develop.

This news article is based on a press release statement from Pfizer Inc. regarding the results of the CIFFREO trial for fordadistrogene movaparvovec.

In other recent news, Pfizer Inc. has been the subject of several significant developments. The pharmaceutical company has released encouraging data from its Phase 3 CROWN trial, indicating a significant extension in progression-free survival for patients with ALK-positive advanced non-small cell lung cancer treated with LORBRENA® compared to XALKORI®.

Moreover, Pfizer, along with its partner BioNTech (NASDAQ:BNTX), Moderna (NASDAQ:MRNA), and Novavax (NASDAQ:NVAX), have been involved in the development of updated COVID-19 vaccines showing promising results against newer subvariants. These vaccines are currently awaiting approval from the U.S. Food and Drug Administration (FDA).

On the legal front, over 70,000 lawsuits against former manufacturers of the heartburn medication Zantac, including Pfizer, are set to proceed. This follows a recent ruling by a Delaware Superior Court judge allowing expert testimony suggesting Zantac may be carcinogenic.

In addition, investment banking firm Jefferies has maintained its Buy rating and $32.00 price target for Pfizer shares, despite questions surrounding the potential impact of the HELIOS-B study on Pfizer's cardiovascular drug Tafamidis. These are among the recent developments surrounding Pfizer.

InvestingPro Insights

As Pfizer Inc. (NYSE: PFE) faces the challenge of its gene therapy for Duchenne muscular dystrophy not meeting the primary endpoint in its recent trial, investors and stakeholders may be assessing the company's financial health and market position. Here are some key metrics and insights from InvestingPro that can provide a broader context to Pfizer's situation:

InvestingPro Data shows that Pfizer has a substantial Market Cap of $156.68B, indicating its significant presence in the pharmaceutical industry. Despite recent trial setbacks, the company's Gross Profit Margin remains strong at 59.41% for the last twelve months as of Q1 2024, reflecting its ability to maintain profitability in its operations. Moreover, Pfizer offers a compelling Dividend Yield of 5.99%, which could be attractive to income-focused investors.

Among the InvestingPro Tips, it's noteworthy that Pfizer has consistently paid dividends to shareholders, maintaining dividend payments for 54 consecutive years, and has raised its dividend for 13 consecutive years. This track record underscores the company's commitment to returning value to shareholders even amidst research and development challenges. Analysts predict that the company will be profitable this year, suggesting confidence in Pfizer's financial outlook despite the recent clinical trial results.

For those interested in a deeper dive into Pfizer's financials and market analysis, there are additional InvestingPro Tips available, providing a comprehensive look at the company's performance and projections. Use the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription at InvestingPro, where you can access a total of 11 tips for Pfizer, offering valuable insights for informed investment decisions.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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