Bio Roundup: Duchenne Race, Warren v. Gottlieb, A $599 Genome & More

Xconomy National — 

As we pause to celebrate the 4th of July, another birthday is top of mind: A third baby whose embryonic DNA was edited by Chinese scientist He Jiankui. As Xconomy reported in November, He hinted about a second pregnancy after his infamous revelation of twins altered with CRISPR gene editing tools. That second pregnancy—a third CRISPR’d child—might already have arrived, MIT Technology Review reports.

As the news of ever-cheaper genome sequencing highlights, however, the genie could be out of the bottle. The means to tinker with DNA are simply too available and too easy to use. We have to lean on our fellow humans’ good ethical sense. One person who might be lacking it is former FDA commissioner Scott Gottlieb—at least according to Sen. Elizabeth Warren (D-MA), a leading presidential candidate who ripped Gottlieb this week for joining Pfizer’s board just two months after leaving the commissioner’s chair.

Elsewhere, we’ve got the latest on some pre-holiday drug approvals, a few closely watched gene therapies, and much more. Let’s fire up the roundup.


—Democratic presidential candidate Elizabeth Warren (D-MA) called on former FDA commissioner Scott Gottlieb to resign from his new board seat at Pfizer (NYSE: PFE), saying the move “smacks of corruption.” Gottlieb immediately took to CNBC to defend himself.

—Meanwhile, Gottlieb’s colleagues Alex Brill and Benedic Ippolito at the American Enterprise Institute rebutted a recent column by drug pricing researcher Peter Bach and colleagues, who argued that biosimilars are not lowering the prices of biologic drugs. AEI countered that biosimilars have, in fact, helped cut net prices.

—A new study from a New York healthcare firm used “real-world” data to try to replicate a randomized controlled diabetes study, and the results have caused a stir among top clinicians. As Stat reports, it’s part of an FDA program to explore the inclusion of medical records and other real-world data in medical studies.


—Pfizer presented data for its Duchenne muscular dystrophy gene therapy at a conference in Orlando. While cross-trial comparisons come with caveats, Wall Street analysts, citing safety concerns, deemed Pfizer’s results inferior to those compiled by Sarepta Therapeutics (NASDAQ: SRPT) thus far. Nonetheless Pfizer will begin late-stage testing next year, following closely behind Sarepta.

—Biogen (NASDAQ: BIIB) continued to defend its spinal muscular atrophy drug nusinersen (Spinraza) against the newly approved gene therapy for SMA, Zolgensma, from Novartis (NYSE: NVS). Biogen provided positive data this week from pre-symptomatic infants, while some payers have been playing hardball with Novartis on Zolgensma, FiercePharma reports. Here’s more on the coming commercial battle between Spinraza and Zolgensma.

—The Roche oral flu medicine baloxavir marboxil (Xofluza) was comparable to generic oseltamivir (Tamiflu) in a Phase 3 study in kids between 1 and 12 years old. Xofluza only needs to be taken once; Tamiflu is given twice daily for five days.

—A combination therapy from Pfizer for infants with persistent pulmonary hypertension—a regimen that included a formulation of sildenafil (Viagra)—failed a Phase 3 trial.


MIT Technology Review is reporting that a third “CRISPR baby” might have been born already, based on an estimate by a leading bioethicist who had been in close contact with He Jiankui, the Chinese scientist in charge of the experiments. After He revealed in November that gene-edited twins had been born, he said there was another pregnancy.

—Boston-based Veritas Genetics was the first company to offer genome sequencing for less than $1,000 and this week dropped the price to $599. Here’s more from OneZero on the efforts over the years to provide DNA sequencing directly to patients at lower and lower prices.

—More than four books about the creation and implications of CRISPR gene editing technology are in development, MIT Technology Review reports.


—After a delay, the FDA granted accelerated approval to selinexor (Xpovio), from Karyopharm Therapeutics (NASDAQ: KPTI), for multiple myeloma patients who have failed at least four treatments. The agency did not wait for data from an ongoing Phase 3 study but told Karyopharm those data must  “confirm” the approval. Karyopharm shares climbed 36 percent.

—Galapagos NV (NASDAQ: GLPG) and partner Gilead Sciences (NASDAQ: GILD) can file for FDA approval of their rheumatoid arthritis drug filgotinib before the end of the year, without waiting for the results of a key safety study first. If approved, filgotinib would compete with several so-called JAK inhibitors, including one from AbbVie (NYSE: ABBV) that could come to market this year.

—Amarin (NASDAQ: AMRN) boosted financial projections for its prescription grade fish oil pill Vascepa and announced plans to double its sales force. Amarin awaits an FDA decision by Sept. 28 to broaden Vascepa’s label to include data showing it can cut the risk of heart disease. Amarin shares climbed 16 percent.

—Citing safety concerns,  the FDA for the second time suspended testing of ACTR087, an Unum Therapeutics (NASDAQ: UNUM) experimental cell therapy for non-Hodgkin’s lymphoma. Unum has already deprioritized the program in favor of a new lymphoma cell therapy, but shares nonetheless fell 17 percent.

—Europe’s Committee for Medicinal Products for Human Use voted not to approve Amgen’s (NASDAQ: AMGN) osteoporosis drug Evenity, which the FDA green-lighted in April with a safety warning.

—Alnylam Pharmaceuticals (NASDAQ: ALNY) filed an approval application in Europe for givosiran, a medicine for acute hepatic porphyria that could be the second-ever marketed medicine using RNA interference. Alnylam has already filed for U.S. approval.

—Following its latest clinical setback, Merrimack Pharmaceuticals (NASDAQ: MACK) is scrapping R&D, selling off drug programs, cutting jobs, and shortening its board of directors. CEO Richard Peters and CFO Jean Franchi have both resigned.


—Century Therapeutics launched with $250 million in funding, the latest startup to emerge from an alliance between Versant Ventures and Bayer. The company is developing allogeneic, or “off the shelf” cell therapies for cancer. Here’s more from the Wall Street Journal.

—In the race to develop treatments for the fatty liver disease nonalcoholic steatohepatitis, Boehringer Ingelheim grabbed rights to an experimental drug from South Korean biotech Yuhan. BI is paying Yuhan $40 million in upfront and near-term milestones.


—Homology Medicines chief operating officer Sam Rasty was named CEO of Platelet BioGenesis… Eric Sievers, formerly of Trillium Therapeutics, is the new chief medical officer of BioAlta.

Alex Lash contributed to this report.