Biogen execs defend Alzheimer's approval as controversial Aduhelm brings $2M

Biogen Inc.'s top executives vigorously defended the conditional approval and launch of Alzheimer's disease treatment Aduhelm during a second-quarter earnings call and in an open letter.

Revenue from the controversial drug totaled $2 million in the quarter as Biogen's overall revenue declined 25% year over year to $2.8 billion in the face of generic-drug competition to multiple sclerosis mainstay Tecfidera.

The U.S. Food and Drug Administration granted Aduhelm — developed with Japanese partner Eisai Co. Ltd. — an accelerated approval June 7, making the drug the first available to treat clinical decline associated with the memory-robbing disease by reducing amyloid plaques in the brain.

The FDA revised the drug's label in early July to narrow the type of patient who is eligible for treatment.

The amyloid plaque theory remains unproven as a cause of Alzheimer's, and experts in the field have pointed to clinical trials conducted by Biogen during Aduhelm's development that showed mixed signals in certain patients. Skepticism of the drug's effectiveness has in turn drawn concerns about patient access and reimbursement.

Biogen CEO Michel Vounatsos addressed this "confusion and criticism" surrounding the approval during the July 22 earnings call, where he defended the company's interactions with regulatory authorities, which are under independent review at the request of acting FDA Commissioner Janet Woodcock.

"I want to be clear that Biogen stands behind the integrity of the review process," Vounatsos said.

The approval "has been the subject of extensive misinformation and misunderstanding," Biogen Head of Research and Development Alfred Sandrock said in an open letter published in the hours leading up to the call.

"We welcome a formal review into the interactions between the FDA and Biogen on the path to the approval of [Aduhelm]," Sandrock said. "A better understanding of the facts is good for everyone involved to assure confidence in both the therapy and the process by which it was approved as we prioritize the issues that affect patients."

Sandrock called the criticisms "a turn outside the boundaries of legitimate scientific deliberation."

Despite the controversy, Aduhelm's launch was progressing as expected, Jefferies analyst Michael Yee said in a July 22 note.

"Comments look generally positive as access is getting underway and suggest good strong early demand," said Yee, adding that the company's stock would take some time to respond in the second half of the year but would likely "climb the wall of worry as logistics and reimbursement generally fall into place and early strong demand pulls through."

Pushback from treatment sites

Some providers, such as Mount Sinai Health Systems and The Cleveland Clinic, have refused to carry the drug on the basis of the regulatory controversy. Of 900 sites originally planned to distribute Aduhelm, about 35% have completed reviews with a positive outcome, Vounatsos said on the call.

Those reviews "will likely be a key near-term focus as investors continue to attempt to gauge uptake over [the] next few quarters and determine how predictive initial sales figures may be for the drug's long-term trajectory," RBC Capital Markets analyst Brian Abrahams said in a July 22 note.

The reasons for access sites' individual decisions vary and cause "confusion in the marketplace," Biogen U.S. Organization President Alisha Alaimo said on the call.

"We are disappointed that sites that have specialized in Alzheimer's indicated they will not provide access to Aduhelm for now," Alaimo said. "We're making every attempt to get in front of these decision-makers to help them better understand the science and data, and there have been specialists and champions at these sites to see if they can reconsider their current policy."

Vounatsos also said the company appreciated the concerns around Aduhelm's $56,000 annual list price and is "committed to ensure sustainability of the system and maximizing access for patients."

"We are working with a sense of urgency to engage with the community, with payers, with [the Centers for Medicare and Medicaid Services] and with policymakers to discuss potential innovative approaches with the goal of ensuring the price does not represent access issues for patients," Vounatsos said.