Bio Roundup: Biogen’s Reversal, FDA Moves Fast, a CF Cocktail & More

Xconomy National — 

Earlier this year, it seemed that companies testing Alzheimer’s disease treatments based on a long-held hypothesis as to its cause—brain deposits of a protein called beta amyloid—were giving up the ghost.

Biogen (NASDAQ: BIIB) was among them. The drug maker had stopped testing of a drug developed to target amyloid plaque after a Phase 3 failure. But this week, Biogen made the surprise revelation that it would file for FDA review of the drug, aducanumab. It said further analysis of data from a broader group of patients showed improvement on several measures of the disease. Following a long line of Alzheimer’s drug failures, observers are excited about the prospect of a possible FDA approval of the Biogen drug. But the company’s announcement leaves many unanswered questions, and it remains to be seen how the regulator will view the new data.

In other news, Vertex Pharmaceuticals (NASDAQ: VRTX) got a lightning-quick FDA approval, Novartis (NYSE: NVS) splashed out cash to grab a NASH drug candidate, and a biotech that postponed its IPO plans over the summer now says it will look to go public after all. Let’s get to those stories in this week’s bio roundup.


—After talks with the FDA, Biogen revived investigational Alzheimer’s drug aducanumab and announced plans to submit the drug for approval.

Acorda Therapeutics (NASDAQ: ACOR) cut 25 percent of its staff in a corporate restructuring that keeps its focus on commercializing its Parkinson’s disease drug.

—The FDA gave the regulatory nod to Alexion Pharmaceuticals (NASDAQ: ALXN) drug ravulizumab (Ultomiris) for atypical hemolytic uremic syndrome, giving the Boston company its second FDA-approved therapy for the rare disease. Alexion is positioning ravulizumab as a successor to its blockbuster seller eculizumab (Soliris), which is nearing patent expiration.

—Seattle Genetics (NASDAQ: SGEN) plans to ask the FDA for approval of breast cancer drug tucatinib after preliminary data showed the experimental treatment hit the main goals of a pivotal study.

A  Vertex Pharmaceuticals (NASDAQ: VRTX) drug that combines two of the company’s existing drugs plus an experimental one was approved by the FDA just three months after the Boston company submitted its application to the regulator. The three-drug cocktail is expected to address 90 percent of CF patients.

—Attorneys general in four states proposed a $48 billion settlement to end claims made over the roles that drug manufacturers, distributors, and retailers played in the nationwide opioid epidemic. But Reuters reported that several states aren’t sure it’s the appropriate way to resolve the thousands of lawsuits facing Johnson & Johnson (NYSE: JNJ), AmerisourceBergen (NYSE: ABC), Cardinal Health (NYSE: CAH), McKesson (NYSE: MCK), and Teva Pharmaceutical (NYSE: TEVA).

—Stan Crooke, the founder and CEO of San Diego’s Ionis Pharmaceuticals (NASDAQ: IONS), and University of North Carolina at Chapel Hill emeritus professor Ryszard Kole are winners of the Massry Prize for their work developing new therapeutic drugs that target messenger RNA. Crooke started Ionis 30 years ago; it has since commercialized multiple RNA-based treatments, including nusinersen (Spinraza), the first FDA-approved treatment for spinal muscular atrophy.


—With technology licensed from MIT and $50 million in financing, Verseau Therapeutics launched to develop its technology for reprogramming immune cells to fight cancer.

—Novartis is paying Pliant Therapeutics $80 million up front for global rights to a preclinical small molecule that the South San Francisco biotech is developing as a potential treatment for fibrosis, the liver damage caused by nonalcoholic steatohepatitis. The deal also covers up to three additional Pliant compounds.

—RAPT Therapeutics this week revived its ambitions to join the public markets, a move that comes nearly three months after the South San Francisco biotech postponed its IPO plans.

—Phathom Pharmaceuticals raised $182 million from its upsized IPO. The Illinois-based developer of gastrointestinal disease drugs sold approximately 9.5 million shares priced at $19 each. Phathom previously planned to offer 7.9 million shares for between $18 and $20 apiece.

—Cabaletta Bio also went public, pricing its offering of 6.8 million shares at $11 apiece—well below its targeted $14 to $16 per share price range. The Philadelphia-based company plans to use the $66.2 million in IPO proceeds to finance development of its experimental cell therapies for autoimmune diseases.


—Vertex reached an agreement with the UK’s National Health Service, which will start covering two of the company’s cystic fibrosis drugs. The deal also expands access to a third Vertex CF drug to those not covered under an earlier reimbursement agreement.

—In other cystic fibrosis news, AbbVie (NYSE: ABBV) licensed a compound from the Cystic Fibrosis Foundation that could be developed into part of a combination treatment for the disease. Financial terms of the deal weren’t disclosed.

—Casebia Therapeutics, the joint venture that Bayer and CRISPR Therapeutics (NASDAQ: CRSP) formed in 2015 to develop gene-editing drugs, will become a standalone business. The revised pact calls for CRISPR Therapeutics to manage Casebia, which will focus on programs in hemophilia, ophthalmology, and autoimmune diseases. Bayer has the right to join development of two of them.

Roche’s $4.8 billion buyout of gene therapy developer Spark Therapeutics (NASDAQ: ONCE) is on its way to clearing a regulatory hurdle. The Federal Trade Commission is ready to recommend approval of the deal without requiring the companies to sell any assets, Reuters reported, citing the Capitol Forum, a company that provides investigative news and legal analysis on matters that affect market competition.


—Akeca Therapeutics (NASDAQ: AKCA) promoted Kyle Jenne to chief commercial officer… Barbara Fox was appointed CEO of Rheos MedicinesDavid Meline is retiring as chief financial officer of Amgen (NASDAQ: AMGN)… Sonde Health appointed David Liu CEO… and Rabia Ozden joined Akouos as chief development officer.

Frank Vinluan contributed to this report.