The life science and healthcare worlds are vast. We often talk about impact in terms of millions of patients and billions of dollars. But these worlds can be small, too. It seems at times we’re never far removed from any one person, through their published papers, social media connections, friends, colleagues, or people they’ve mentored.
We had terrible news this week of the deaths of two people whose lives and work touched many people. Let’s start by pausing to thank Michael Becker, a biotech executive-turned-patient advocate who fought to raise awareness of the role of the human papillomavirus in cancers such as the one that eventually killed him. His blog post from March, in which he grapples with his own view of death, is something we’ll remember for a long time.
We also acknowledge Suzanne Eaton, a top researcher at the Max Planck Institute in Dresden, Germany. She went missing last week in Greece, and her body was found this week. Police believe she was murdered. It’s worth reading the testimonials about Eaton, a native of Oakland, CA, to see how “American scientist” only begins to describe her. As we remember Eaton and Becker, let’s also take time out to acknowledge people in our lives who are still here and deserve a fuller accounting. Each of us is a vast world.
This Week in Gene Therapy
—The race to develop a gene therapy for hemophilia accelerated this week. BioMarin Pharmaceutical (NASDAQ: BMRN) will file for US and Europe approvals of its hemophilia A gene therapy, known in shorthand as valrox, by the end of the year. BioMarin also presented the latest clinical results for valrox, but it’s unclear how long the treatment will last and how wide its reach will be.
—Valrox’s limitations have left the door open for rivals. One of them, SB-525 from Sangamo Therapeutics (NASDAQ: SGMO) and partner Pfizer (NYSE: PFE), produced early clinical results that could threaten valrox if the data hold up in further testing.
—UniQure (NASDAQ: QURE) also detailed the latest results for its hemophilia B gene therapy, which is in late-stage testing and battling a rival program from Spark Therapeutics (NASDAQ: ONCE).
Politics and Policy
—The White House said that President Trump killed the administration’s plan to eliminate secret rebates that insurance negotiators, known as pharmacy benefit managers, extract from drug companies. It’s the latest instance of Trump rhetoric about lowering drug prices not matching up with action.
—The administration also unveiled big changes to the federal rules governing kidney care, such as making home dialysis easier.
—A federal judge in Washington, DC, said the administration couldn’t force drug makers to include prices in their TV ads.
—Last Friday, Trump told reporters that he would soon issue an executive order to let Medicare buy drugs based on lower prices in other countries, an idea the administration first floated last year.
—Despite bipartisan political pressure to cut the cost of prescription drugs, biopharmaceutical companies kicked off the third quarter with price hikes on a range of branded and generic medications. Fierce Pharma has more.
Deals and Dollars
—Following in the footsteps of well-financed messenger RNA drug developer Moderna (NASDAQ: MRNA), German mRNA drugmaker BioNTech raised a $325 million Series B round, one of Europe’s largest biotech financings ever. Here’s more from Bloomberg.
—Revolution Medicines closed a $100 million in Series C financing that the Redwood City, CA, biotech will use to develop drugs targeting cancers with certain genetic mutations.
—Breakpoint Therapeutics spun out of Evotec to develop the German company’s pipeline of “DNA damage response” programs in cancer. Evotec and others provided Breakpoint with approximately $33 million in cash.
—Fulcrum Therapeutics revealed terms for its proposed IPO, targeting a sale of 4.5 million shares in the range of $16 to $18 apiece. The Cambridge, MA-based rare disease drug developer would use the proceeds to run Phase 2b tests of losmapimod, an experimental treatment for facioscapulohumeral muscular dystrophy.
—Mountain View, CA cancer drug developer IGM Biosciences closed $102M in Series C financing to advance its lead program, a bispecific antibody, into clinical testing.
—Celgene (NASDAQ: CELG) expanded its ongoing alliance with Nimbus Therapeutics, nabbing an option to license a cancer immunotherapy targeting the tough-to-drug protein HPK1.
Data and Research
—Amgen and Novartis stopped work on the experimental Alzheimer’s drug umibecestat after an early look at clinical data showed a decline in participants’ cognitive function.
—Belgium-based Celyad announced encouraging preliminary data from Phase 1 studies of two experimental CAR-T cell therapies for advanced colorectal cancer.
—Rivogenlecleucel (Rivo-cel), an experimental Bellicum Pharmacueticals (NASDAQ: BLCM) drug developed to reduce the rate of leukemia relapse following a stem cell transplant, met the main goal of a study expected to support a submission for approval in Europe.
—Over the July 4 holiday, a Russian scientist said he wants to edit human embryos with CRISPR and prevent them from inheriting a genetic form of deafness from their parents.
People on the Move
–Richard Peters, who just resigned as CEO of struggling Merrimack Pharmaceuticals, will succeed Tony Coles as the CEO of Yumanity Therapeutics … Richard Scheller, a former Genentech executive, left his role as chief scientific officer of 23andMe to become both the chair of R&D at BridgeBio Pharma (NASDAQ: BBIP) and a director at Maze Therapeutics … Sanjay Keswani left the CEO role at Rheos Medicines to become chief medical officer of Annexon Biosciences … Proclara CEO Suzanne Bruhn jumped to Tiaki Therapeutics for the chief executive job … Genentech’s Lori Friedman joined ORIC Pharmaceuticals as chief scientific officer … Tizona Therapeutics appointed Joyson Karakunnel chief medical officer … and Christi Shaw, senior vice president and president of Eli Lilly Bio-Medicines, will become CEO of Kite Pharma, a division of Gilead Sciences.
Ben Fidler and Frank Vinluan contributed to this report.
Photo courtesy of lraul06 via Creative Commons.