Bio Roundup: Alzheimer’s Fail, Drug Price Plan, Gene Therapies & More

Xconomy National — 

Pharmaceutical companies typically raise prescription drug prices at the start of each year and this year was no different. The increases were the latest reminder that, despite President Trump’s fiery campaign rhetoric, the administration had done little to actually lower drug prices. But a new plan aimed at the secretive rebate deals between pharma companies and pharmacy benefit managers could change things.

Insurers, or their intermediaries, negotiate with pharma companies for pricing discounts. But rather than passing the savings on to patients, the companies typically pocket the difference. The Department of Health and Human Services on Thursday unveiled a plan that would allow drug makers to pass discounts on to consumers, but forbid the companies from giving rebates to the middlemen in pricing negotiations. The proposal would apply to beneficiaries of Medicare and Medicaid, but HHS officials told The Washington Post that they expect these changes would also be adopted by employer health plans.

Indicative of the ongoing spat between biopharma and PBMs over blame for high drug prices, their respective lobbying groups had disparate views of the plan. The Pharmaceutical Care Management Association, which represents PBMs, warned it would raise costs for Medicare beneficiaries. Pharma’s lobbying group, the Pharmaceutical Researach and Manufacturers of America, said it would lower patients’ out-of-pocket costs, according to the Wall Street Journal. Time will tell, but the plan could take effect next year.

In other news this week, yet another Alzheimer’s disease drug failed its final test, a major pharmaceutical company dove deeper into gene therapy, and a new biotech emerged in a new area of cell biology. Let’s get to those stories and more in this week’s news roundup.


—The U.S. House of Representatives, now controlled by Democrats who have pledged to investigate the drug industry, and the Senate held simultaneous hearings Tuesday on drug prices. Members of both parties took the pharma industry to task; Sen. Chuck Grassley (R-IA) said he would investigate the high prices of insulin, in particular.

— Senators Bill Cassidy (R-LA) and Mark Warner (D-VA) introduced the Patient Affordability Value and Efficiency Act, a bill meant to lower existing legal barriers to value-based agreements—the type of pay-for-performance drug pricing models that gene therapies will likely rely on.

—Novartis (NYSE: NVS) is considering installment payment plans to cover the likely multi-million dollar cost of Zolgensma, the spinal muscular atrophy gene therapy expected to win FDA approval this year. Here’s more from Bloomberg.


—Johnson & Johnson (NYSE: JNJ), meanwhile, signaled plans to grow a franchise in gene therapy, paying New York-based MeiraGTx (NASDAQ: MGTX) $100 million to grab rights to multiple experimental gene therapies for genetic eye diseases and boost its manufacturing capabilities. Meira shares climbed 28 percent.

—J&J’s apalutamide (Erleada) succeeded in a Phase 3 study in men with a form of metastatic prostate cancer. The drug is part of J&J’s plan bid to build on the success of its other prostate cancer treatment abiraterone (Zytiga). Here’s more from Vantage.

—Crenezumab, a drug from Roche and partner AC Immune, became the latest in a long line of therapies to fail a big test in Alzheimer’s disease. It’s the latest blow to the “amyloid hypothesis” that many believe to explain the cause of the memory robbing disorder, but the theory lives on—a similar drug from Biogen (NASDAQ: BIIB) could produce data this year or next.

—The FDA rejected a Sunovion Pharmaceuticals Parkinson’s disease drug, giving Acorda Therapeutics (NASDAQ: ACOR) a chance to enter the market with a similar, rival Parkinson’s treatment without a direct competitor.

—Ravulizumab (Ultomiris), from Alexion Pharmaceuticals (NASDAQ: ALXN), met the main goal of a Phase 3 study in patients with hemolytic uremic syndrome, a rare disease that leads to progressive organ damage. Alexion will file for FDA approval in the first half of the year.

—Alexion also nabbed an option to license CAEL-101, a drug for immunoglobulin light chain amyloidosis, from privately held Caelum Biosciences and acquired a minority stake in the company.

—Trevena (NASDAQ: TRVN) said it reached an agreement with the FDA on a path forward for its pain drug oliceridine. The FDA rejected the treatment last year.

—TAK-003, a vaccine from Takeda Pharmaceutical (NYSE: TAK) for Dengue fever, hit its main goal in the first part of a Phase 3 trial. The vaccine needs to succeed in a longer-term part 2 of the study before Takeda files for approval, however.

—Pfizer (NYSE: PFE) and Eli Lilly (NYSE: LLY) reported that their antibody pain drug, tanezumab, beat a placebo in two of the three main goals of a second Phase 3 study in osteoarthritis. The drug succeeded in an earlier Phase 3 test.

—Shares of Oncocyte (NYSE: OCX) rose more than 260 percent to $6.84 apiece after it published positive results for its liquid biopsy test. Shares have since retreated to $4.81 apiece as of late Thursday.


—Neurocrine Biosciences (NASDAQ: NBIX) paid Voyager Therapeutics (NASDAQ: VYGR) and made a $50 million equity investment in the Cambridge, MA, company to get rights to a group of experimental gene therapies for neurological diseases—among them a Parkinson’s disease treatment beginning late-stage testing.

Lyndra Therapeutics raised $55 million in Series B financing for mid-stage clinical tests of its extended-release drug delivery technology.

Dewpoint Therapeutics emerged from stealth with $60 million to develop drugs in an emerging realm of science focusing on protein droplets in cells that are linked to disease, including cancer and neurodegeneration.

—Aldeyra Therapeutics (NASDAQ: ALDX) paid $10 million in stock to buy Healio Vision, a privately held biotech with a drug for the eye disease proliferative vitreoretinopathy on the verge of Phase 3 testing.

—Cancer drug developer Agenus (NASDAQ: AGEN) revealed plans to offer digital securities that provide investors the opportunity to put their money behind a single biopharmaceutical product. Agenus says it aims to raise up to $100 million in mid-February and it will provide additional details at that time.

—In more conventional financing moves, Gossamer Bio now plans enter the public markets via a standard IPO. When the SEC was shuttered during the partial government shutdown, the San Diego biotech had said it would go public through a rarely used rule that has less regulatory oversight.


—Aduro Biotech (NASDAQ: ADRO) laid off more than a third of its staff as the Berkeley, CA, biotech focuses on its clinical-stage cancer immunotherapies, including one partnered with Novartis (NYSE: NVS).

—Mehrdad Mobasher left Genentech for the chief medical officer post at Corvus Pharmaceuticals (NASDAQ: CRVS) … former Sanofi (NYSE: SNY) executive Sheldon Koenig joined Portola Pharmaceuticals (NASDAQ: PTLA) as chief commercial officer … and Aleksandra Rizo left Celgene (NASDAQ: CELG) for the chief medical officer job at Geron (NASDAQ: GERN).

Ben Fidler and Alex Lash contributed to this report.

Photo by Flickr user Chris Potter via a Creative Commons license