The first ever approval of a new kind of cancer immunotherapy called CAR-T is one step closer. A 10-member panel of doctors and researchers who advise the Food and Drug Administration recommended with a rare unanimous vote that the agency approve a treatment for kids and young adults with a severe form of leukemia who … Continue reading “Unanimous Advice To FDA: Approve Landmark CAR-T Cancer Therapy”
Author: Alex Lash
I've spent nearly all my working life as a journalist. I covered the rise and fall of the dot-com era in the second half of the 1990s, then switched to life sciences in the new millennium. I've written about the strategy, financing and scientific breakthroughs of biotech for The Deal, Elsevier's Start-Up, In Vivo and The Pink Sheet, and Xconomy.
Before Key Meeting, FDA Stresses Risks of Novartis CAR-T Cancer Therapy
Two days before a panel of the FDA’s outside advisors grill Novartis about its groundbreaking cell therapy under review, agency staffers released their own assessment of the product, which now goes by the name tisagenlecleucel. If approved for kids and young adults with acute lymphoblastic leukemia, or ALL, it would likely be the first so-called … Continue reading “Before Key Meeting, FDA Stresses Risks of Novartis CAR-T Cancer Therapy”
Bio Roundup: Senate Drama, A.I. Stories, Data Dives, Shkreli & More
There is no Obamacare replacement, at least not as of this writing. The Senate Republicans are fractured, with a handful of conservatives and moderates each giving a cold shoulder to their chamber’s version of healthcare reform, the Better Care Reconciliation Act. The nonpartisan Congressional Budget Office report Monday put the bill’s potential effects in stark … Continue reading “Bio Roundup: Senate Drama, A.I. Stories, Data Dives, Shkreli & More”
New Study: DNA Tests For Healthy People Have “Uncertain Value”
Genome sequencing is becoming more common for people diagnosed with cancer. Should it become part of a healthy person’s checkup, too? A new study published Monday in the Annals of Internal Medicine questions the practicality of making DNA tests standard for people who don’t have a cancer diagnosis or aren’t trying to identify a mysterious … Continue reading “New Study: DNA Tests For Healthy People Have “Uncertain Value””
Bio Roundup: Senate Trumpcare, FDA On Cancer, Alzheimer Review & More
All eyes are on the nation’s capital this week, where the Senate released its version of the American Health Care Act (now called the Better Care Reconciliation Act) and the White House worked on an executive order to address drug prices. No one knows how to pay for high-priced medicines that could bring long-term relief … Continue reading “Bio Roundup: Senate Trumpcare, FDA On Cancer, Alzheimer Review & More”
Bluebird Reports Early Results From Upgraded Gene Therapy
One of the year’s most closely watched clinical studies could lead to a landmark approval of a gene therapy and throw wide open the debate over how to pay for expensive drugs. The first drips of data have emerged. Bluebird Bio (NASDAQ: [[ticker:BLUE]]) says the first three patients—of 15 total expected—have had good results from … Continue reading “Bluebird Reports Early Results From Upgraded Gene Therapy”
Alzheimer’s Drug Pipeline Analysis: Have We Hit Peak Amyloid?
A 2014 report provided a stark frame of reference for the challenge of fighting Alzheimer’s disease, showing that 99.6 percent of all drugs in the field had failed in one way or another. Led by Jeffrey Cummings, director of the Cleveland Clinic’s Lou Ruvo Center for Brain Health, the same authors recently published a new … Continue reading “Alzheimer’s Drug Pipeline Analysis: Have We Hit Peak Amyloid?”
New Ovarian Cancer Data Suggests Larger Role For Top Clovis Drug
Clovis Oncology received a short-cut approval from the FDA last year for its ovarian cancer drug that treats women with the common BRCA genetic mutation. Clovis (NASDAQ: [[ticker:CLVS]]) said Monday it now has evidence that the drug, rucaparib (Rubaca), can treat a wider swath of patients, and it will ask the FDA for approval this … Continue reading “New Ovarian Cancer Data Suggests Larger Role For Top Clovis Drug”
Amgen’s Heart Data Don’t Impress Drug-Price Evaluation Group
The drug-price evaluator that the drug companies love to hate has weighed in again, this time casting a skeptical eye on the price of a next-generation heart medicine from Amgen (NASDAQ: [[ticker:AMGN]]). The group known as ICER, which stands for Institute for Clinical and Economic Review, released a report today that said Amgen’s evolocumab (Repatha) … Continue reading “Amgen’s Heart Data Don’t Impress Drug-Price Evaluation Group”
Bio Roundup: ASCO Digested, Juliet Unveiled, PTC Slated & More
The New York Times Magazine wrote a few weeks ago about the extraordinary metabolic changes in a python after it gulps down a 50,000 calorie meal. The biomedical world was like a postprandial python this week; after extending its maw around the supersized meal of news and data from the American Society for Clinical Oncology … Continue reading “Bio Roundup: ASCO Digested, Juliet Unveiled, PTC Slated & More”
ASCO Roundup: Checkpoint Combos, Tumor Profiling, Financial Toxicity
[Note: Ben Fidler coauthored this report.] The American Society of Clinical Oncology conference is wrapping up, and the closely watched medical meeting-slash-hype machine produced its usual array of data. There were a few big business-focused “horse race” stories—one company’s stumble was another company’s gain—but much of the news consisted of mid-trial updates that companies produce … Continue reading “ASCO Roundup: Checkpoint Combos, Tumor Profiling, Financial Toxicity”
In Maine, Making Cancer DNA Tests Free—And Asking Tough Questions
Has the era of genetic oncology arrived? Last week, the biomedical world took a notable step in that direction when the FDA said Merck’s drug pembrolizumab (Keytruda), already one of the world’s most successful cancer immunotherapies, could now treat any tumor with a particular genetic fingerprint. It was the first time a drug has been … Continue reading “In Maine, Making Cancer DNA Tests Free—And Asking Tough Questions”
Bio Roundup: Skinny Cuts, Genomic Approvals, Unfrozen FDA & More
Team Trump unveiled its 2018 federal spending proposal—the so-called “skinny budget”—with health and science in the crosshairs. The NIH is looking at a 22 percent cut and the FDA 31 percent, although the administration wants to backfill the FDA loss with a huge boost in user fees, which drug and device companies pay to have … Continue reading “Bio Roundup: Skinny Cuts, Genomic Approvals, Unfrozen FDA & More”
Back To The Baseline? Ex-FDA Commish Califf Joins Verily As Advisor
In addition to a return to his old stomping grounds at Duke University, former FDA commissioner Robert Califf is going to work for Verily Life Sciences, the Silicon Valley R&D health group formerly known as Google Life Sciences. Verily published a blog post Wednesday in Califf’s name describing the vaguest outlines of his new gig. … Continue reading “Back To The Baseline? Ex-FDA Commish Califf Joins Verily As Advisor”
Synlogic and Its Modified Bacteria Take Over Dying Company, Go Public
Synlogic, which is developing modified bacteria to use as medicine, has gone public in an unconventional way. Instead of hiring Wall Street bankers to shop its shares to new investors, as most companies do for an initial public offering, Cambridge, MA-based Synlogic has merged with the shell of a former biotech company. The so-called reverse … Continue reading “Synlogic and Its Modified Bacteria Take Over Dying Company, Go Public”
Bio Roundup: Big Apple Mo’, Checkpoint Yes and No, CAR-T Death & More
“New York, New York, it’s a hell of a town. The Bronx is up, but we’re Brooklyn down.” For all you old-school Brooklyn swingers out there, we’ll take a short break from the madness in the nation’s capital and start our roundup in the greatest city in the world. You might disagree if you’ve got … Continue reading “Bio Roundup: Big Apple Mo’, Checkpoint Yes and No, CAR-T Death & More”
Former National Mental Health Chief Thomas Insel Leaving Verily
Thomas Insel, the former National Institute of Mental Health director, has left Verily Life Sciences after less than two years. With much fanfare but few details about his plans, Insel jumped in 2015 to South San Francisco-based Verily, formerly known as Google Life Sciences. In an announcement this morning, Verily said it would continue working on … Continue reading “Former National Mental Health Chief Thomas Insel Leaving Verily”
Kite CAR-T Death: An Unwelcome Mystery as FDA Mulls Approval
In a lengthy interview in January, Kite Pharma CEO Arie Belldegrun was adamant that his company’s experimental cell therapy was different from one competitor, Juno Therapeutics, because unlike with Juno, no Kite patient had died from cerebral edema, or massive swelling in the brain. Belldegrun can no longer make that claim. Kite (NASDAQ: [[ticker:KITE]]) reported … Continue reading “Kite CAR-T Death: An Unwelcome Mystery as FDA Mulls Approval”
If Budget Holds, NIH Precision Study Could Be Flush As Volunteers Arrive
Until this week, many U.S. government science agencies and programs seemed destined for a big haircut, plus the loss of a limb or two. That included the Precision Medicine Initiative, an ambitious long-term study with the goal of recruiting 1 million Americans to volunteer health data. But in staving off a government shutdown, Congress pushed … Continue reading “If Budget Holds, NIH Precision Study Could Be Flush As Volunteers Arrive”
CA Stem Cell Agency Chief Randy Mills to Leave After Three Years
[Updated 5/2/17, 7:30 p.m. ET. See below.] The head of California’s stem-cell agency is stepping down after three years on the job. C. Randall Mills, known as Randy, is leaving the agency to run a nonprofit bone marrow donor matching program as of July 1, according to CIRM. Mills joined CIRM, which stands for California … Continue reading “CA Stem Cell Agency Chief Randy Mills to Leave After Three Years”
Magenta Nabs More Cash, Licenses Drug To Boost Transplant Pipeline
Magenta Therapeutics said today it has doubled its money with a $50 million Series B round led by GV, formerly Google Ventures. The Cambridge, MA-based startup spun out of Harvard University last year with nearly $50 million in launch money to develop improved bone marrow transplants. Magenta has also licensed a drug from Novartis that … Continue reading “Magenta Nabs More Cash, Licenses Drug To Boost Transplant Pipeline”
Nominations Open for the Inaugural Xconomy Awards
Like so much of the life sciences world, Xconomy has its roots in Boston and Cambridge. When the annual Biotech Week Boston convenes this September, we’ll be there, too, with our Life Science Disruptors forum, but also something new: The inaugural Xconomy Awards, highlighting some of the most influential people, startups, and other organizations in … Continue reading “Nominations Open for the Inaugural Xconomy Awards”
Bio Roundup: Trumpcare Flails, Spinraza Sales, Batten OK & More
To judge the first 100 days of a new administration is, as many pundits have pointed out, an odd vestige of history, more convenient than significant. Which is why we’ll start this week’s roundup with our 99-day evaluation of the Trump administration—at least when it comes to healthcare and the life sciences. In healthcare, nothing … Continue reading “Bio Roundup: Trumpcare Flails, Spinraza Sales, Batten OK & More”
Pursuing Larger Rivals, NGM Bio Tries To Make Mark On Fatty Livers
[Corrected 4/25/17, 12:30 p.m. See below.] About 7,000 liver transplants occur in the U.S. each year. A condition that few people can pronounce, and no one has a treatment for, has become a main reason for those transplants. That’s why several rich, powerful drug companies are in the final expensive stage of testing new drugs … Continue reading “Pursuing Larger Rivals, NGM Bio Tries To Make Mark On Fatty Livers”
Verily’s Baseline: Homemade Wearable and Limited Data Sharing
After three years of planning, a long-range health study from the biotech group at the former Google has begun. Verily, the life sciences arm of Google’s parent company Alphabet, is funding what’s being called the “Baseline” study, which aims to collect extensive health data from at least 10,000 participants across four years. The idea is … Continue reading “Verily’s Baseline: Homemade Wearable and Limited Data Sharing”
After Medivation Sale, ex-CEO David Hung Lands Top Spot At Axovant
Axovant Sciences, with a high-profile Alzheimer’s treatment in Phase 3 studies, has brought on David Hung as CEO. Hung was founder, president, and CEO of San Francisco-based Medivation, which Pfizer (NYSE: [[ticker:PFE]]) bought for $14 billion last year, in large part to gain control of the prostate cancer drug enzalutamide (Xtandi). Medivation’s Marion McCourt will … Continue reading “After Medivation Sale, ex-CEO David Hung Lands Top Spot At Axovant”
Bio Roundup: Gottlieb Talks Vax, Acorda’s Axe, Adams, 23andMe & More
In the nation’s capital, healthcare and life sciences were on center stage. Or stages, actually. On Capitol Hill, FDA commissioner nominee Scott Gottlieb, the drug industry’s favorite pick, told senators his extensive industry ties wouldn’t cloud his judgment or create conflicts. At a downtown convention center, Joe Biden slammed the Trump administration’s budget-cutting plans. And … Continue reading “Bio Roundup: Gottlieb Talks Vax, Acorda’s Axe, Adams, 23andMe & More”
AACR Review: Biden Fired Up, Incyte Doubles Up, BMS Up And Down
The American Association for Cancer Research held its annual meeting this week in Washington, DC, a convenient venue for former Vice President Joe Biden to deliver a broadside against the Trump administration’s proposed budget cuts in science funding. For Biden, it’s personal. In the final year of the Obama administration, he championed a push for … Continue reading “AACR Review: Biden Fired Up, Incyte Doubles Up, BMS Up And Down”
FDA Nominee Scott Gottlieb To Anti-Vaccine Crowd: Get Over It
In what passes for a short hearing these days in Washington, DC, the Trump White House’s pick to run the Food and Drug Administration, Scott Gottlieb, spent less than three hours fielding questions from a Senate committee Wednesday morning. Amid predictable splits between Republican praise for his past industry experience and Democrat fire over potential … Continue reading “FDA Nominee Scott Gottlieb To Anti-Vaccine Crowd: Get Over It”
FDA OKs Eczema Injection; Owners Regeneron, Sanofi Set $37K Price
For the more severe cases of eczema—inflamed skin that can range from annoying dry patches to painful swelling, oozing, and cracking—there’s a new injectable medicine on the market that will cost tens of thousands of dollars a year. The FDA approved today dupliumab (Dupixent) for adults whose condition cannot be controlled with topical creams. The … Continue reading “FDA OKs Eczema Injection; Owners Regeneron, Sanofi Set $37K Price”
Amid Gender Gap Talk, Mentor Network Emerges For Women In Health, Bio
It’s been more than a year since an infamous party with hired models in cocktail dresses captured the biotech community’s attention at the 2016 J.P. Morgan conference. There has been plenty of talk since about closing biotech’s notable gender gap. At this year’s J.P Morgan conference, for example, a group of 100 life science executives … Continue reading “Amid Gender Gap Talk, Mentor Network Emerges For Women In Health, Bio”
Bio Roundup: Trump Budget, FDA Chief, CAR-T Qs, CRISPR Cash, & More
If it wasn’t clear that the Trump administration disdained most types of scientific inquiry and practice, the White House’s proposed budget drove the point home. The top-line figures: a nearly 20 percent cut in the National Institutes of Health, and a 30 percent cut in the Environmental Protection Agency. These and other cuts to programs … Continue reading “Bio Roundup: Trump Budget, FDA Chief, CAR-T Qs, CRISPR Cash, & More”
CRISPR Organs? eGenesis Raises $38M For Pig-To-Human Transplants
One of the companies trying to revive the once-failed notion of xenotransplantation—transplanting animal organs or tissues into humans—has raised a significant round of cash. The $38 million Series A round announced by Cambridge, MA-based firm eGenesis comes as the field of human-compatible organs grown in animals, while still years from real products, is back in … Continue reading “CRISPR Organs? eGenesis Raises $38M For Pig-To-Human Transplants”
Former Gates Investor, Advisor Unveil $200M Biomatics Health Fund
After years of investing Bill Gates’s money and giving him scientific advice, two of the software billionaire’s associates have launched their own $200 million healthcare fund. Some of biotech’s highest-profile bets are already in their portfolio. Based in Seattle, Biomatics Capital Partners has been an open secret for some time thanks to its own regulatory … Continue reading “Former Gates Investor, Advisor Unveil $200M Biomatics Health Fund”
Possible Cures. Mystery Deaths. Daunting Costs. Can CAR-T Be Tamed?
It’s a struggle that comic-book fans know well. Ordinary people, bestowed with super powers from a spider bite or gamma rays, struggle to harness their own abilities. T cells, key soldiers of the immune system, are wrestling with new-found super powers, too. Certain T cells hunt down invasive viruses, bacteria, and the body’s own bad … Continue reading “Possible Cures. Mystery Deaths. Daunting Costs. Can CAR-T Be Tamed?”
Bio Roundup: Healthcare Is Hard, “Slow” FDA, Drugging RNA & More
In an address to Congress this week, President Donald Trump outlined many of his near-term goals, including an overhaul of the Affordable Care Act, also known as Obamacare, even as public opinion has begun to shift in favor of the law. Trump toned down his typical blunderbuss rhetoric, but he had choice words for the … Continue reading “Bio Roundup: Healthcare Is Hard, “Slow” FDA, Drugging RNA & More”
After Trial Deaths, Juno Pivots and Scraps Lead CAR-T Therapy
Juno Therapeutics said today it would discontinue its experimental CAR-T cell product known as JCAR015. It was once Juno’s top candidate as the company raced rivals to be the first to win approval of a new kind of cancer treatment called CAR-T. The decision was not unexpected. Juno (NASDAQ: [[ticker:JUNO]]) hoped that a 110-person clinical … Continue reading “After Trial Deaths, Juno Pivots and Scraps Lead CAR-T Therapy”
With Eye On FDA, Kite Says CAR-T For Lymphoma Holds Up After 6 Months
Kite Pharma released more data this morning that it hopes will lead this year to the first commercial approval of a cutting-edge cancer treatment known as CAR-T therapy. The Santa Monica, CA, firm will soon ask the FDA for approval to use its CAR-T therapy, known as axicabtagene ciloleucel, to treat patients with desperate cases … Continue reading “With Eye On FDA, Kite Says CAR-T For Lymphoma Holds Up After 6 Months”
Bio Roundup: Trump Backlash, Verdine Plans, Sarepta Sale & More
A short week makes for a shorter roundup. As we’ve come to expect, however, there was no dearth of health and life-sciences news from Washington, D.C. A bipartisan group of Congress members who oversee health policy countered President Donald Trump’s encouragement of vaccine skeptics; the real test will be the upcoming budget’s funding of Centers … Continue reading “Bio Roundup: Trump Backlash, Verdine Plans, Sarepta Sale & More”
Patent Judges’ CRISPR Ruling For Broad Leaves Daylight For Berkeley
The U.S. Patent and Trademark Office has made a decision in the patent fight over ownership of the landmark CRISPR-Cas9 gene editing technology. The ruling is in favor of the Broad Institute of MIT and Harvard, whose 2014 patents were challenged by a group led by the University of California, Berkeley (UC). But the ruling … Continue reading “Patent Judges’ CRISPR Ruling For Broad Leaves Daylight For Berkeley”
Merck Alzheimer Drug Is Latest To Fail In A Big Study
Another Alzheimer’s drug has failed a major test. Merck (NYSE: MRK) reported late Tuesday that its pill verubecestat was not helping people with significant Alzheimer’s symptoms. The drug maker announced that it halted its EPOCH study early because an oversight group said there was “virtually no chance of finding a positive clinical effect.” Verubecestat is … Continue reading “Merck Alzheimer Drug Is Latest To Fail In A Big Study”
Bio Roundup: Travel Ban Block, Gilead Shock, Dean Kamen Talk & More
The biotech community pushed back against President Donald Trump’s order to block travelers and refugees this week, and not much later, a federal court did, too. Patient advocates have scored big wins with recent approvals of rare disease treatments, but they’re not as happy with insurers’ coverage decisions or with Trump’s promises of massive FDA … Continue reading “Bio Roundup: Travel Ban Block, Gilead Shock, Dean Kamen Talk & More”
Often Advocates Of Faster FDA, Patient Groups Wary Of Trump Deregulation
In the biomedical world, perhaps the biggest question looming over President Donald Trump’s upcoming pick for Food and Drug Administration commissioner is how drastically that person will help roll back FDA regulations. Trump said at last week’s meeting in Washington D.C., with pharmaceutical executives that he wanted to slash FDA rules by 75 to 80 … Continue reading “Often Advocates Of Faster FDA, Patient Groups Wary Of Trump Deregulation”
Kentucky Biotech CEO’s Message For McConnell: I’ll Be Muslim, Too
President Trump’s order to bar refugees and immigrants from seven Muslim-majority countries is on temporary hold, and a court hearing later today could eventually force a ruling by the U.S. Supreme Court. One of the latest biotech executives to speak out against the ban is in the backyard of Mitch McConnell (R-KY), the top Republican … Continue reading “Kentucky Biotech CEO’s Message For McConnell: I’ll Be Muslim, Too”
Drug-Hunting Startup From Scripps Attracts Celgene Alum, $50M Backing
A team of decorated academic chemists in San Diego have been working since 2014 on a biotech startup that aims to find new drugs faster. The company now has a lot of cash in the bank and a well-known biopharma scientist to guide it. Vividion Therapeutics is launching with $50 million pledged from investment firms … Continue reading “Drug-Hunting Startup From Scripps Attracts Celgene Alum, $50M Backing”
Still No Official Word From Biopharma Groups About Trump Travel Ban
Four days after President Donald Trump issued an order to temporarily ban U.S. entry for people from seven Muslim-majority countries—and for all refugees—the drug industry’s two main lobbying groups have remained silent, even while individual executives in the industry have voiced opposition. The main life science trade group in California has also stayed on the … Continue reading “Still No Official Word From Biopharma Groups About Trump Travel Ban”
Report: Biotech VCs Perpetuate Boardroom Gender Gap
If power in the business world is centered in the boardroom, women in biotech have a long way to go to get their fair share—and the venture community deserves much of the blame. That’s according to British recruitment firm Liftstream and its new study of biotech companies that went public in the recent boom years. … Continue reading “Report: Biotech VCs Perpetuate Boardroom Gender Gap”
Ex-FDA Commish Califf Gives Props to Patient Groups—With Caveats
The nation’s former Food and Drug Administration commissioner Robert Califf, appearing for the first time since he resigned last week, told a Silicon Valley crowd this morning that the FDA will have to be flexible in coming years, allowing greater patient input into drug evaluation and leaning on outside watchdogs for help weeding out “imposters.” … Continue reading “Ex-FDA Commish Califf Gives Props to Patient Groups—With Caveats”
Bio Roundup: Tom Price, Patient Conflicts, Orphan Games & More
The new president of the United States, Donald Trump, is being sworn in today amid a storm of questions about the plans that he, and those who might work for him, have in store for American healthcare and medicine. Trump’s nominee to run the $1 trillion Health and Human Services Department, Tom Price, answered—or didn’t … Continue reading “Bio Roundup: Tom Price, Patient Conflicts, Orphan Games & More”
Dems Grill HHS Nominee Price About Trump Comments, Stock Holdings
[Note: This report was co-authored by deputy biotech editor Ben Fidler.] Tom Price, the man who could oversee an overhaul of the U.S. healthcare system, answered often pointed questions this morning from U.S. senators about his views, his plans for Obamacare, and personal financial transactions. Price, an orthopedic surgeon by training, is President-elect Donald Trump’s … Continue reading “Dems Grill HHS Nominee Price About Trump Comments, Stock Holdings”