Bio Roundup: Biogen at CTAD, Sage’s Stumble, Astellas’s $3B Offer & More

Xconomy National — 

It’s been a roller-coaster year for those hoping for new drugs to treat Alzheimer’s disease, a leading cause of death with rising treatment costs and no clear answer yet as to its cause.

Clearing proteins, such as amyloid and tau, was once seen as the top contender for stopping or slowing neurodegeneration. Costly failures forced researchers to look at other targets. But the experimental Biogen (NASDAQ: BIIB) anti-amyloid treatment, aducanumab, is still alive and kicking after the company decided to submit to regulators results from additional analyses conducted after stopping a Phase 3 trial—and Thursday at the Clinical Trials on Alzheimer’s Disease (CTAD) congress, it presented those findings.

Holding conference attendees rapt, Biogen executive Samantha Budd Haeberlein explained how one study in the trial failed yet another succeeded. A subset of patients who received higher doses for longer showed better results, a finding that prompted the company’s decision to move forward with the aducanumab program, she said. While some were persuaded, others found Biogen’s explanation lacking, and called for additional testing. That call resides with the FDA. We’ll round up that story and the rest of the week’s biotech headlines below.


—The Biogen talk at CTAD, first on the agenda on the conference’s first full day, revealed details behind the company’s belief in the drug’s benefits for patients with Alzheimer’s disease. At day’s end, the company’s stock price was up 3 percent.

—A Sage Therapeutics (NASDAQ: SAGE) drug failed to beat a placebo in a Phase 3 study testing it in major depressive disorder. Shares of the company sank more than 60 percent on the news.

—Six scientists working on ways to diagnose Alzheimer’s disease early received grants from the Alzheimer’s Drug Discovery Foundation (ADDF) Diagnostics Accelerator initiative, which is funded by Bill Gates and other philanthropists.

—Acadia Pharmaceuticals (NASDAQ: ACAD) reported positive data from a Phase 3 study of its drug, pimavanserin (Nuplazid), in dementia-related psychosis. The drug was approved in 2016 to treat Parkinson’s disease psychosis.


Japan’s Astellas Pharma agreed to buy Audentes Therapeutics, a San Francisco biotech developing gene therapies for rare neuromuscular diseases, for $3 billion.

—Veracyte (NASDAQ: VCYT) paid $50 million for diagnostics rights to a NanoString (NASDAQ: NSTG) technology that analyzes the activity of genes. South San Francisco-based Veracyte says the technology will become the basis for a broader range of diagnostic tests it plans to develop and sell worldwide.

—Cyrus Biotechnology and the Broad Institute of MIT and Harvard announced a research partnership to make CRISPR-Cas9 technology safer as gene-edited therapies make their way into human trials.

—San Diego’s Neurocrine Biosciences splashed out $50 million up front for rights to work Xenon Pharmaceutical has done in drug discovery and development around epilepsy.

—Startups Atomwise and Stemonix announced a joint venture to use their respective technologies—deep learning tools and MicroOrgans for screening, respectively—together to speed up drug discovery and development targeting Rett syndrome.

—Kodiak Sciences (NASDAQ: KOD) pocketed $225 million by selling future royalty rights to its experimental eye drug, KSI-301, to Baker Bros. Advisors. Palo Alto, CA-based Kodiak plans to use its new cash for clinical testing, manufacturing, and if approved, commercialization of its drug for retinal vascular diseases.


—Fresh off two deals with Novartis earlier this year, IFM Therapeutics reloaded with $55.5 million in financing and plans to launch an incubator and a subsidiary, both of them focused on developing therapies for inflammatory diseases and cancer.

—Software startup Deep 6 AI raised $17M in Series A funding to advance its software system, which aims to use artificial intelligence tools to resolve the challenge of patient enrollment in clinical trials.

—What to invest in with $1 billion? GHO raised a record amount of funding to invest in healthcare—including the outsourced services providers supporting biotech startups.

—New biotech company Civetta Therapeutics raised $53 million in Series A financing to develop drugs that target “propeller proteins” as a way of treating cancers and other diseases.

—Less than a year after closing an $85 million Series B round of funding, Black Diamond Therapeutics this week raised another $85 million for its Series C round. The Cambridge, MA-based biotech is developing cancer drugs that target allosteric sites on a molecule.

—Limelight Bio raised $75 million to support its work developing gene therapies that could overcome limitations of current gene therapy and gene editing approaches. The company, which splits its operations between Philadelphia and Cambridge, is led by former Biogen executive Michael Ehlers.

—And now for something completely different: What’s the opposite of a funding frenzy? That’s what Veritas Genetics, a gene sequencing startup co-founded by Harvard geneticist George Church, appears to be going through. The Boston company, which at one time envisioned making gene sequencing affordable and accessible to all, said on Twitter that it was temporarily suspending US operations due to “an unexpected adverse financing situation.”


—Roche drug atezolizumab (Tecentriq) notched an additional FDA approval as a first-line treatment for non-small cell lung cancer, boosting its competitive position versus Merck (NYSE: MRK) immunotherapy pembrolizumab (Keytruda).

Sight Diagnostics received FDA marketing clearance for its OLO analyzer, which provides complete blood test results in minutes from just two drops of blood.

—Citing manufacturing issues, the FDA rejected a regenerative therapy that Enzyvant developed to address a rare immune cell deficiency. The Cambridge rare disease drug developer plans to meet with the FDA to discuss the agency’s questions.


—Cara Therapeutics (NASDAQ: CARA) reported positive Phase 2 data for its experimental treatment for pruritis, or severe itching, in patients with chronic kidney disease. A Phase 3 test of the drug is expected to begin next year.

—Axsome Therapeutics (NASDAQ: AXSM) reported that its experimental narcolepsy drug AXS-12 succeeded in a mid-stage study, and the company plans to advance the compound to Phase 3 testing.

FerGene, a company recently formed by Ferring Pharmaceuticals and Blackstone Life Sciences, reported that its its experimental gene therapy for bladder cancer met the main goal of a Phase 3 clinical trial.


—Agenus (NASDAQ: AGEN) Chief Technology Officer Alex Duncan is stepping down in January… IFM Therapeutics CEO Gary Glick is now the company’s executive chairman and Martin Seidel was promoted to the chief executive position… Repare Therapeutics tapped Steve Forte as its new chief financial officer… Curtis Ruegg was named president and CEO of Amphivena Therapeutics… Caraway Therapeutics appointed Martin Williams its CEO… Gabriel Cohn left Avrobio (NASDAQ: AVRO) to become chief medical officer of Homology Medicines… and John Northcott joined Nektar Therapeutics (NASDAQ: NKTR) as chief commercial officer.

Frank Vinluan and Melissa Fassbender contributed to this report