Biogen CEO Blames Confusion, Misinfo For Slow Alzheimer’s Drug Launch

Stat: Biogen Admits Aduhelm Launch ‘Slower Than We Anticipated’
Biogen CEO Michel Vounatsos admitted Thursday that the launch of Aduhelm, the company’s intensely scrutinized treatment for Alzheimer’s disease, has been “slower than we anticipated,” which he blamed in part on “confusion, misinformation, and controversy” about whether the drug works and how it was approved. Only about 50 centers around the country have administered at least one dose of Aduhelm, which won Food and Drug Administration approval on June 7, Biogen said at an investor conference sponsored by the investment bank Morgan Stanley. That has led Biogen to revisit its “already very low sales target” for 2021, Vounatsos said. (Garde and Feuerstein, 9/9)

Stat: Early Data Suggest Hemophilia Drug Could Contend With Gene Therapy
Centessa, a biotech company that raised more than $300 million in an IPO earlier this year, has an early-stage treatment for hemophilia that might compete with the gene therapies nearing the market. The company’s drug, a monthly treatment injected under the skin, significantly reduced the rate of bleeding for patients with hemophilia A and B in a small study, Centessa said Thursday. In the trial, which enrolled just 23 people and was not placebo-controlled, the highest dose of Centessa’s drug led to an 88% reduction in annualized bleeding rate compared to baseline after six months. (Garde, 9/9)

Stat: FDA Rejection Of Humanigen Drug Points To Industry Challenge In Covid Fight
The Food and Drug Administration has declined to grant emergency use authorization to lenzilumab, an experimental drug developed by Humanigen, for the treatment of hospitalized patients with Covid-19, the company said Thursday. “In its letter, FDA stated that it was unable to conclude that the known and potential benefits of lenzilumab outweigh the known and potential risks of its use as a treatment for COVID-19,” Humanigen said. (Herper, 9/9)

In other pharmaceutical and biotech news —

Philadelphia Inquirer: Penn Scientists Win $3 Million Breakthrough Prize For RNA Research That Enabled COVID-19 Vaccines
Sixteen years after their research at the University of Pennsylvania paved the way for billions to be vaccinated against COVID-19, two scientists have been honored with a $3 million Breakthrough Prize. The award for Katalin Karikó and Drew Weissman, one of five such honors announced Thursday for achievements in science and math, recognizes their success in modifying the genetic molecule called messenger RNA (mRNA) so it can instruct human cells to make customized proteins. (Avril, 9/9)

Stat: For First Time, The FDA Warns A Researcher For Failing To Report Trial Results
In a little noticed warning, the Food and Drug Administration threatened to fine the principal investigator of a clinical trial for failing to submit results as required by federal law, marking the first time the agency has widened its list of targets that could face penalties for such a violation. Until now, the regulator had warned two different drug makers for failing to post study results to a federal database, ClinicalTrials.gov. Those moves came as a belated response to years of complaints that too many drug makers and universities do not follow requirements for reporting results, an issue that has riled countless researchers and transparency advocates. (Silverman, 9/9)

Stat: Digital Clinical Trials Can Improve, Accelerate Therapy Development 
Biopharmaceutical companies were conducting more than 9,000 clinical trials in the U.S. in March 2020 when the Covid-19 pandemic disrupted trial operations with broad travel restrictions and concerns about patient safety and investigative site capacity. Many clinical trials that required repeated, in-person visits to a clinic or trial site were delayed or halted. But some researchers forged ahead, combining novel tactics like mailing medications and relying on digital technologies such as transmitting photos of home-based blood pressure readings via text message to conduct trials remotely. (Anderson, 9/9)

Genomeweb: Amazon Investment Helps Mammoth Biosciences Reach $195M
CRISPR technology startup Mammoth Biosciences said on Thursday that it has raised a combined $195 million through Series C and Series D financing rounds, making it a so-called unicorn company — startups that are valued at more than $1 billion. The financing included a $45 million Series C round, which was completed in late 2020, and was led by Redmile Group and Foresite Capital. That round also included participation from previous investors, as well as Amazon. The financing also included a $150 million Series D round, which was led by Redmile Group, with participation from Foresite Capital, Senator Investment Group, Sixth Street, Greenspring Associates, Mayfield, Decheng Capital, NFX, and Plum Alley. (9/9)

Stat: Study Results Cloud The Future Of Apellis Pharma's Eye Disease Treatment
Apellis Pharmaceuticals has a good news-bad news problem involving its experimental treatment for geographic atrophy — a chronic eye disease and a leading cause of blindness in older people. Two large and identically designed Phase 3 clinical trials produced confounding results: One study showed Apellis’ drug, injected into the eye, was a success — significantly slowing the progression of the disease compared to a sham injection. But the drug failed to show a benefit in the other study, albeit narrowly. (Feuerstein, 9/9)

In updates from the Theranos trial —

San Francisco Chronicle: Elizabeth Holmes' Trial Delayed After Juror's Potential COVID Exposure
The trial of former Theranos CEO Elizabeth Holmes has been delayed just a day after opening statements took place after a juror was potentially exposed to the coronavirus, according to multiple reports. Friday’s hearing was postponed and the next trial date is Tuesday, Sept. 14. During an emergency Zoom hearing late Thursday, Judge Edward Davila said a juror may have been exposed last weekend and was awaiting a coronavirus test. The juror is vaccinated and isn’t showing symptoms. (Li, 9/9)