FDA advisers snub Regeneron drug for gout

Posted under Arcalyst, Blog, Companies, Diagnostics, FDA, Funding, gout, Medical Devices, Medical Supply, Pharmaceuticals, Regeneron, Regeneron Pharmaceuticals, Regulatory, Startups, Universities, Videos by rmcbride

Regeneron ($REGN) lost a bid to gain a key endorsement for using the biotech group's drug Arcalyst to combat gout flare-ups. An FDA advisory committee rejected the drug in an 11-0 vote against approval as experts harbored concerns about the lack of safety data and the limited time period of 16 weeks patients took the drug in studies. 

The FDA will weigh the panel's advice in the agency's review of the drug, with a decision targeted by July 30, Reuters reported. Tarrytown, NY-based Regeneron is seeking approval of the injected therapy, which is already on the market for a rare genetic disorder, to treat a much larger population of patients during a period of time in which they are open to gout flare-ups. Yet members of the advisory committee were concerned that the drug could be used for longer periods, and there's a lack of data to show that the treatment is safe for prolonged usage.  

Safety risks were highlighted in the FDA staff briefing documents on Regeneron's application last week, which noted that Arcalyst could boost the risk of infections and malignancies in patients while imparting only a modest benefit. And non-agency experts sounded equally unsatisfied with the body of evidence the company has provided to prove the safety and efficacy of the drug for gout patients.

"A lot of the patients where we envision using this drug are going to take it for longer than 16 weeks. It's hard for me to allow patients to be exposed [without longer safety data]," said panel member Dr. David Blumenthal, of Case Western Reserve University School of Medicine, as quoted by Reuters.

- check out the Reuters article
- see the PharmaTimes report

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FDA notes cancer cases linked to Regeneron gout drug

Posted under Arcalyst, Blog, Companies, Diagnostics, FDA, Funding, gout, Medical Devices, Medical Supply, Pharmaceuticals, Pipeline, Regeneron, Regeneron Pharmaceuticals, Regulatory, Startups, Universities, Videos by rmcbride

Regeneron Pharmaceuticals' ($REGN) Arcalyst heads to an FDA panel next week as a new treatment for gout flare-ups after a mixed review of the drug in FDA staff documents released Friday. The drug developer is seeking an expanded approval of the drug, previously OK'd for combating rare genetic auto-inflammatory conditions, for use in certain patients with the much more common ailment.

The FDA staff review questioned whether the risks of the treatment overwhelm the benefits of the drug in fighting gout flare-ups, saying that the drug provided only a "small" benefit and pointing out that 6 patients studied developed malignancies, Bloomberg reported. Regeneron wants approval for Arcalyst to treat gout patients during a 16-week period when they are open to attacks from the disease and initiating uric-acid lowering treatment.

"From an efficacy standpoint, it will be important to address whether 16 weeks provide for an adequate duration for flare prophylaxis during initiation of (uric acid-lowering therapy)," the FDA staff said in review documents, as quoted by Reuters. The news service also reported that reviewers noted that the immune-suppression of the biologic could be linked with the cancer cases, but they were uncertain whether the relatively short duration of treatment on the drug could cause malignancies.

Regeneron's shares were down 1.37% to $132.08 as of 11:57 a.m. ET today.

The additional approval for Regeneron's drug could significantly grow revenue from sales of the product, which were just $20 million last year but could jump to more than 10 times that total by 2015 with the gout indication, Bloomberg reported, citing data and analyst estimates.

- see Bloomberg's article
- and Reuters' coverage

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Sanofi, Regeneron report stellar LDL drug data as race heats up

Posted under Amgen, Blog, cholesterol, Companies, Diagnostics, Funding, Lipitor, Medical Devices, Medical Supply, Pharmaceuticals, Regeneron Pharmaceuticals, Sanofi, Startups, Universities, Videos by John Carroll

With an eye to keeping its lead on Amgen ($AMGN), Sanofi ($SNY) and Regeneron ($REGN) today noted that their rival PCSK9 inhibitor triggered a significant drop in LDL levels among patients with a high level of the "bad" kind of cholesterol. Responding to the promising Phase II data, Sanofi R&D chief Elias Zerhouni vowed to pivot into a late-stage program in the second quarter.

The key numbers for Sanofi were recorded in two groups among the 183 patients recruited for the study. In one group patients taking SAR236553/REGN727 in a subcutaneous injection twice a month registered a 72% mean drop in LDL compared to a 5% drop in the placebo arm. A separate cohort taking the twice-monthly treatment along with 80 mg of Lipitor saw a 73% reduction compared to an 18% drop for the Lipitor-only arm.

"I've not seen anything like this in all my days of research," says James McKenney, CEO of National Clinical Research and the lead investigator, who has been studying drugs for three decades. "This is so impressive."

The mid-stage data reflected an improvement over early-phase results, adds McKenney. In an interview with FierceBiotech he added that the longer you give the drug to a patient, the more efficacious it becomes. And one of the central objectives of this study was to zero in on the best dosage and frequency of treatment. 

"No one else has finished the Phase II," he added. "We're the first ones out."

"There are a lot of implications for people who receive gold standard statins and either don't respond in a way that gives them a vigorous reduction or can't tolerate high doses, experiencing muscle pain and weakness," added McKenny. "That's the group that is an obvious low-hanging fruit that needs additional help. And this product based on what we found can give them a remarkable and dramatic additional lowering" of LDL. Later, he added, investigators will be able to assess the potential health benefits for the broader population.

Just how big that market might be triggered some chatter on Twitter over the weekend, with at least one analyst raising concerns that only a small subpopulation of patients could benefit from LDL drugs like the ones Amgen and Sanofi/Regeneron are investigating. But in a recent note, Jefferies analyst Biren Amin reportedly noted estimates that 77 million U.S. adults have high levels of LDL, with an estimated 3.4 million high-risk patients that could benefit most from a treatment like this.

According to their release, serious adverse events occurred in one patient receiving placebo and three patients in the active treatment arms, including a patient on active treatment who experienced a skin rash diagnosed as leukocytoclastic vasculitis.

"Based on this finding and the results of our Phase II trials, Sanofi and Regeneron plan to initiate the SAR236553/REGN727 Phase III program in the second quarter," says Zerhouni.

- here's the press release

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Developers spotlight huge potential of new class of cholesterol drugs

Developers spotlight huge potential of new class of cholesterol drugs

Posted under Amgen, Blog, cholesterol, cholesterol drug, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, Merck, Pharmaceuticals, Regeneron Pharmaceuticals, Startups, Universities, Videos by John Carroll

Previewing the upcoming meeting of the American College of Cardiology meeting in Chicago, Reuters goes in-depth on a new class of potential blockbusters that will muscle its way into the R&D spotlight. The drugs inhibit the PCSK9 protein, offering a fresh approach to dramatically reducing levels of "bad" cholesterol.

At the top of the list of contenders is Regeneron Pharmaceuticals' ($REGN) REGN 727, which reduced LDL up to 65% in clinical trials. On Sunday, Amgen ($AMGN) will produce early-stage data on its anti-PCSK9 program, called AMG 145. And Reuters adds that Merck ($MRK) and others will muscle rivals in the field for attention as well.

Mani Mohindru, an analyst with Think Equity, tells Reuters that she "can't believe the cardiologists won't be in awe of the LDL reductions."

The developers certainly seem to be in awe of the potential of a new class of drugs that could be offered to millions of patients. "PCSK9 is one of the most exciting targets in cardiovascular drug development today," said Michael Severino, Amgen's chief medical officer.

- here's the story from Reuters

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