Bio Roundup: Pfizer’s Future, CRISPR in Patients, Drug Imports & More

Xconomy National — 

[Corrected 12:55 p.m. ET. See below.] Acquisitions made Pfizer what it is today: the world’s biggest drug maker measured by revenue. But as the company maps its future, CEO Albert Bourla is breaking some of that legacy apart.

Blockbuster drugs such as Viagra, which was discovered in-house, and Lipitor, which came via acquisition, are currently sold by Upjohn, Pfizer’s off-patent drug division. This week, Pfizer announced it a deal to spin off Upjohn and merge it with generic drug maker Mylan, the developer of the EpiPen. The merger announcement came as Pfizer was finishing off another deal, the spinoff of its consumer product division, which was combined with a similar unit of GlaxoSmithKline. What’s left of Pfizer, Bourla told analysts, is a smaller company focused on developing more innovative—and more profitable—new medicines. [Paragraph corrected to state that Viagra was discovered by Pfizer.]

In other news this week, the White House announced a plan to import cheaper drugs from Canada, a cancer diagnostics firm made its biggest acquisition ever, and a biopharma executive found 15 minutes of fame on social media. Let’s get to those stories and much more in this week’s roundup.


—A year after buying Nationwide Children’s Hospital spinout Celenex and diving into gene therapy, Amicus Therapeutics (NASDAQ: FOLD) provided the first detailed look at the results with data from a gene therapy for Batten disease. The data, though early and in a handful of patients, suggest that the treatment might have a chance to stabilize the condition for patients with a particular genetic mutation.

—Xconomy profiled the story behind Casimir Trials, a startup formed by two Duchenne muscular dystrophy patient advocates—Christine McSherry and Mindy Leffler—in the wake of the controversial 2016 FDA approval of eteplirsen (Exondys 51). Casimir is using video capturing tools to develop new outcome measures for clinical trials. One of them is currently being evaluated by the FDA, a development that is emblematic of how the agency’s thinking has evolved over the past few years.

—Biogen (NASDAQ: BIIB) and partner Alkermes (NASDAQ: ALKS) reported that diroximel fumarate (Vumerity)—intended to be a safer version of the Biogen multiple sclerosis drug dimethyl fumarate (Tecfidera)—led to fewer gastrointestinal side effects than Tecfidera in a Phase 3 trial. Wall Street analysts, however, questioned Vumerity’s whether the benefit is enough to make Vumerity a big seller once generic forms of Tecfidera hit the market, which could occur as soon as next year.

—Roche once again delayed its proposed acquisition of gene therapy developer Spark Therapeutics (NASDAQ: ONCE) to give US and UK antitrust regulators more time to review the deal.

—Rare disease drug developer Ultragenyx Pharmaceutical (NASDAQ: RARE) filed for approval of triheptanoin. The drug is an experimental treatment for long-chain fatty acid oxidation disorders, a group of genetic disorders in which the body is unable to convert long-chain fatty acids into energy.

—Translate Bio (NASDAQ: TBIO) touted results from a handful of patients in a Phase 1/2 study of its experimental cystic fibrosis treatment MRT5005, the first messenger RNA therapy given to people with a genetic disease. After one dose, some patients showed early signs of improvement on a measure of lung function—additional results are expected next year. But shares nonetheless fell 11 percent as investors questioned the “variability” of the results, wrote SVB Leerink analyst Geoffrey Porges.

—An Alzheimer’s drug from AbbVie (NASDAQ: ABBV) failed a Phase 2 study in progressive supranuclear palsy, a neurodegenerative disorder.

—Victoria Gray, the first person in the US to receive a CRISPR-based gene-editing medicine, told her story to NPR. The Mississippi resident is receiving an experimental treatment for sickle cell disease developed by CRISPR Therapeutics (NASDAQ: CRSP).

—Spain-based publication El Pais reported that Spanish scientists are doing chimeric experiments in China to put human genes into monkey embryos.

—Novartis (NYSE: NVS) CEO Vas Narasimhan described on LinkedIn how he manages what he calls the “relentless pace” of his job. He leans on exercise, meditation, a vegetarian diet, and dark chocolate…and can’t resist small-batch gin.


—The Merck immunotherapy pembrolizumab (NASDAQ: MRK), when combined with chemotherapy, beat chemo alone in a Phase 3 trial in triple-negative breast cancer. Keytruda was used in the “neoadjuvant” setting, before surgery to remove a tumor. The news marked a step forward for immunotherapy in breast cancer, which as Xconomy highlighted earlier this year, has proved a difficult challenge for a variety of reasons.

—Separately, Merck also picked up a new FDA approval for Keytruda in throat cancer.

—Darolutamide (Nubeqa), a drug developed by Bayer and partner Orion, won FDA approval as a treatment for castration-resistant prostate cancer that has not spread to other parts of the body.

—In its quarterly report Amgen (NASDAQ: AMGN) said its cutting-edge treatment AMG510 has made progress in two more types of cancer. The Thousand Oaks, CA-based company reported earlier this year that the drug shrank lung tumors. It was the first drug targeting the cancer gene KRAS to deliver clinical data.

—More quarterly news: Gilead Sciences (NASDAQ: GILD) said its CAR-T cell therapy Yescarta tallied $216 million in 2019 sales through two quarters, double the sales of the other commercial CAR-T, Novartis’s Kymriah. Xconomy reported earlier this year on the commercial obstacles these drugs face. Reuters reported this week that sales are low because many lymphoma patients are opting instead to enter clinical studies of experimental medicines.


—Pfizer (NYSE: PFE) reached a stock deal to spin out Upjohn, its off-patent drug division, and merge it with generic drug maker Mylan (NASDAQ: MYL).

—Exact Sciences (NASDAQ: EXAS) paid $2.8 billion to buy Genomics Health (NASDAQ: GHDX), its largest acquisition ever, expanding the company’s suite of cancer diagnostics.

—European venture capital firm Wellington Partners raised approximately $233 million for a new life science fund expected to invest in 15 to 20 startups primarily in Europe, but also in North America and Asia.

—Vida Ventures closed a $600 million fund for more investments in life science companies.

—South San Francisco-based RAPT Therapeutics, an early-stage developer of drugs for cancer and inflammatory diseases, postponed its IPO, according to Renaissance Capital.


—The Trump administration announced a preliminary framework for importing cheaper drugs from Canada and other countries, but observers noted that loopholes in the proposals leave out many of the most expensive medicines.

—Democratic presidential candidate Sen. Kamala Harris (D-CA) unveiled her healthcare plan, which includes a 10-year transition to a form of Medicare for all that would retain private insurance as an option.

—The Tuesday and Wednesday night debates over healthcare were detailed, “ferocious,” and at times confusing, with Harris’s proposal a major focus, prompting Senate minority leader Chuck Schumer (D-NY) to warn candidates to avoid a “circular firing squad.” Coverage questions dominated, leaving other healthcare issues like the opioid crisis nearly unmentioned.

—Meanwhile, the Kaiser Family Foundation updated its national poll on Medicare and healthcare access.


—This week we named Third Rock Ventures co-founder and partner Mark Levin the winner of our 2019 Lifetime Achievement Award in Boston. Xconomy also profiled Levin’s life, career, and impact on Boston’s life sciences ecosystem. Levin will receive the award and give an acceptance speech at our annual Xconomy Awards gala on Sept. 12.


–Verb Surgical named Kurt Azarbarzin its new CEO… Arvinas (NASDAQ ARVN) appointed Ronald Peck chief medical officer… Yi Larson left Goldman Sachs (NYSE: [[ticker:]]) to become chief financial officer of Turning Point Therapeutics (NASDAQ: TPTX)… Harvard’s Willam Blake joined Sherlock Biosciences as chief technology officer… Verastem Oncology (NASDAQ: VSTM) appointed board member Brian Stuglik to serve as CEOMark Avagliano left Pfizer to become chief business officer of G1 Therapeutics (NASDAQ: GTHX)… Dan Chevallard joined Viracta Therapeutics as CFO… Frontier Medicines hired Kevin Webster as chief scientific officer and Johannes Hermann as chief technology officer… and Mehdi Gasmi, president and chief scientific officer of Adverum Biotechnologies (NASDAQ: ADVM), will retire in September.

Ben Fidler and Alex Lash contributed to this report. 

Photo by Flickr user Matthew Rutledge via a Creative Commons license