In the span of 16 hours, two giant biotech developments – The Boston Globe

Were in a new era of biotech, Alfred Sandrock, Biogens head of research and development and chief medical officer, said of both developments in a telephone interview. We have better drug targets validated by human genetics.

Many questions remain, particularly about Biogens stunning reversal. Still, the news involving two of the states biggest biotechs raised the hopes of families anddoctors desperate for drugs to treat the diseases.

Biogens announcement on Tuesday was the bigger bombshell. The firm has been working for years on the experimental drug aducanumab for Alzheimers. That form of dementia afflicts more than 5 million Americans, and no drugs significantly slow its ravages.

The hunt for an effective treatment has eluded some of the worlds biggest drug companies. According to an industry report, there were 146 failed attempts to develop medicines to treat and potentially prevent Alzheimers in the past two decades and only four new drugs approved to treat symptoms.

On March 21, Biogen said it was abandoning tests on aducanumab based on the recommendations of an independent monitoring board entrusted to protect patients in the study.

Biogen and its Japanese partner Eisai said they halted two late-stage clinical trials after concluding that the compound was unlikely to benefit patients.

The news sent Biogen stock into a free fall, and the company lost a staggering $18 billion the day of the announcement. With the setback, Biogen joined the long list of drug firms that had struck out on Alzheimers treatments.

It also appeared to mark the demise of a series of experimental Alzheimers drugs that targeted a protein in the brain called beta amyloid, all of which had failed.

But Michel Vounatsos, Biogens CEO, and Sandrock said the aducanumab story didnt end in March.

Biogen, in consultation with a team from the FDA, conducted a new analysis of a larger data set from the late-stage clinical trials that were halted, they said. The new analysis included additional data that became available after the prior analysis showed the study had no chance of success.

The new data showed that aducanumab was pharmacologically and clinically active in higher doses in reducing brain amyloid and in reducing clinical decline. Sandrock said the earlier data contained only about half the patients who had enrolled in the clinical trial and focused on people who had received smaller doses.

We didnt quite appreciate how important it was to get to a very high dose, he said.

On Monday, a dozen Biogen scientists and statisticians and two representatives of Eisai met for two hours with eight FDA neurology officials at the agencys Maryland headquarters, said Sandrock, who attended.

We shared with them the entire data set, and we were told it was reasonable to submit an application for approval of aducanumab, Sandrock said. Biogen expects to make the submission early next year.

Several analysts expressed deep skepticism that the FDA will OK the drug despite the new analysis.

Brian Skorney, an analyst with Baird, issued a report headlined If You Torture the Data Long Enough, It Will Confess to Anything.

Biogens decision to file for regulatory for aducanumab in Alzheimers, after halting the study for futility, is understandable given the addressable market, he wrote. However we do not share theirenthusiasm for the interpretation of the results and believe approval would rely on regulators with minimal capacity for critical analysis.

PiperJaffray analysts said in another report that aducanumabs return from the dead was a complete shocker and that they werent sure what to make of the drugs prospects.

However, Dr. Brent Forester, chief of geriatric psychiatry at McLean Hospital and a principal investigator in the aducanumab trial at his hospital, was delighted by the news.

Forester said six patients with early symptoms of Alzheimers received the drug. While its difficult to say whether it worked, he said, the patients seemed stable.

There was never any indication that Biogen would pull the plug because it wasnt working, he said.

Debbie Rosenkrantz, a 66-year-old retired clinical social worker in Cambridge, said she took the medicine in the trial for about eight months. She felt it halted the decline in her memory.

I actually felt like it was working, and then to be told that it wasnt working was kind of disappointing to me, she said. This was a nice turnaround.

The revival of aducanumab Tuesday sent Biogens stock up, and it closed up more than 26 percent on the Nasdaq. That helped the firm recoup some of the billions of dollars in value it lost when Biogen said it had pulled the plug on the compound.

Mondays announcement about Vertexs cystic fibrosis drug approval was widely expected but the speed of the government ruling caught the industry by surprise. The FDA had set a goal of deciding whether to approve the drug by March 2020.

Vertex already has three cystic fibrosis drugs on the market. Trikafta, the new combination drug, is expected to benefit up to 90 percent of the 27,000 CF patients in the country, according to the FDA.

The FDAs acting commissioner, Ned Sharpless, said after the drug won approval that the agency has been striving to speed the development of therapies for complex diseases.

Like Vertexs other drugs for cystic fibrosis, Trikafta has a jaw-dropping list price: $311,000 a year.

Joseph J. ODonnell, a Boston corporate tycoon whose son, Joey, died of cystic fibrosis in 1986 at the age of 12, was stunned to learn about the approval while on vacation.

ODonnell has helped to raise hundreds of millions of dollars for the nonprofit Cystic Fibrosis Foundation, much of which has gone to Vertex for research.

He said he and his wife, Kathy, were on a cruise ship near Athens on Monday night when they heard the news in a phone call from their daughter, Kate ODonnell.

The ODonnells celebrated over dinner with six other couples with whom they took the cruise, all of them contributors to the fund.

I knew this was going to happen sooner or later because all the results [of clinical trials] were so consistently good, ODonnell, 75, said in a phone interview. But I never dreamed it would happen before the first of the year.

Jonathan Saltzman can be reached at jsaltzman@globe.com

Read more from the original source:
In the span of 16 hours, two giant biotech developments - The Boston Globe