Congrats to this year’s Nobel Prize winners in medicine… although, we have to ask: For the US researchers who are honored, isn’t there something crushing about a call in the middle of the night from Sweden, interrupting a dream about the perfect protein-protein interaction? Wouldn’t it be more logical for the committee to wait until … Continue reading “Bio Roundup: Nobel Prizes, Placebo Effect Rises, ICER’s Fire & More”
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.
The Placebo Effect Is Hobbling New Psychiatric Drugs. What Can Stop It?
Sixteen years ago, Kim Witczak’s husband died by his own hand, turning her world upside down. He had just begun taking an antidepressant off-label for insomnia, and she believes an undisclosed side effect of the drug drove him to suicide. Compelled to act, she became an advocate for tougher safety standards. Witczak now sits on … Continue reading “The Placebo Effect Is Hobbling New Psychiatric Drugs. What Can Stop It?”
Bio Roundup: Pelosi’s Reveal, Alder’s Deal, Biogen’s Fails & More
Boston, San Francisco, and a few other metro areas might dominate the US life sciences, but some weeks, all bio-related eyes are on the nation’s capital. Health concerns about vaping continue to mount, and the feds could get involved. Meanwhile, try to find someone in the federal government who isn’t involved in the drug-price debate. … Continue reading “Bio Roundup: Pelosi’s Reveal, Alder’s Deal, Biogen’s Fails & More”
Pelosi, Dems Unveil Price Plan: Are 25 Drugs Enough for Negotiation?
With the 2020 election just over a year away, House Speaker Nancy Pelosi (D-CA) has unveiled the Democratic Party’s answer to public discontent over high prescription drug prices. A preview of the plan was leaked last week. Today’s announcement doesn’t stray far. The new plan would have the U.S. Department of Health and Human Services … Continue reading “Pelosi, Dems Unveil Price Plan: Are 25 Drugs Enough for Negotiation?”
After First Look at House Drug Plan, Stocks Rise Slightly
The first details of the long-awaited House Democrat plan to lower drug prices leaked out Monday night, with elements that have long been anathema to the biopharma industry and its supporters in Washington. While the world digested the details Tuesday, however, biopharma investors didn’t seem fazed. Some individual companies saw shares dip, but the biopharma … Continue reading “After First Look at House Drug Plan, Stocks Rise Slightly”
Bankrupt uBiome Says Founders May Have Misled Investors
[Updated, 9/5/19, 1:17am. See below.] Less than a year after reeling in a $83 million investment round, privately held microbiome startup uBiome has filed for bankruptcy and says its founders might have misled those investors amid other potential improper business practices. The allegations of impropriety, made more dramatic by an FBI raid earlier this year, … Continue reading “Bankrupt uBiome Says Founders May Have Misled Investors”
Bio Roundup: Cholesterol Check, J&J’s Opioid Hit, AbbVie’s Flop & More
You might be camping, barbecuing, or sleeping on Monday. It’s Labor Day, after all. But in Paris, this cardiologist will be in a conference center, walking an audience through a slide deck packed with Phase 3 data for a new cholesterol-lowering drug, inclisiran. Inclisiran’s owner, the Medicines Co., jumped the presentation by a week, promising … Continue reading “Bio Roundup: Cholesterol Check, J&J’s Opioid Hit, AbbVie’s Flop & More”
New PCSK9 Cholesterol Drug Faces Tough Foe. (Hint: Not Cholesterol.)
[Updated 8/26/19, 10:30am. See below.] Remember when the new wave of expensive cholesterol-lowering drugs—known as PCSK9 inhibitors—was supposed to give the healthcare system a financial heart attack? Far from it. Four years after approval the two PCSK9 blockers on the market have yet to crack $1 billion in annual sales, combined, thanks to a gloves-off … Continue reading “New PCSK9 Cholesterol Drug Faces Tough Foe. (Hint: Not Cholesterol.)”
Bio Roundup: Zolgensma Fallout, Duchenne Redux, Ebola Boost & More
We learned last week that the FDA was investigating Novartis for manipulating animal data related to its $2 million-a-dose gene therapy Zolgensma. The activity took place at AveXis, the firm Novartis bought for $8.7 billion in 2018, and Novartis reportedly knew but failed to report it to the FDA before the agency approved Zolgensma in … Continue reading “Bio Roundup: Zolgensma Fallout, Duchenne Redux, Ebola Boost & More”
Bio Roundup: Michael Becker, Suzanne Eaton, Gene Therapy Moves & More
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. … Continue reading “Bio Roundup: Michael Becker, Suzanne Eaton, Gene Therapy Moves & More”
Trump Kills His Administration’s Plan to End Secret Drug Rebates
Donald Trump’s criticism of high drug prices began during the 2016 presidential campaign and continued through his presidency, perhaps most famously just before his inauguration when he said that drug companies were “getting away with murder.” This week has underlined the gap between the administration’s rhetoric and results. Most strikingly, the White House said last … Continue reading “Trump Kills His Administration’s Plan to End Secret Drug Rebates”
Bio Roundup: Merger Drama, FDA Trauma, Big IPOs, CRISPR Fights & More
It was a busy week. If we had to choose a theme, it was all about getting together. Two huge drug companies, AbbVie and Allergan, said they’d be better as one. Two more huge drug companies, Celgene and Bristol-Myers Squibb, learned they would have to leave a big product behind if they want to merge. … Continue reading “Bio Roundup: Merger Drama, FDA Trauma, Big IPOs, CRISPR Fights & More”
Sequencing Firm Adaptive’s $300M IPO Comes With Pharma Ambitions
If biotech’s historic bull run is losing steam, as some observers believe, this week is one hell of a final stampede. Adaptive Biotechnologies of Seattle just raised $300 million. BridgeBio Pharma of Palo Alto, CA, has topped it, raising $348.5 million. Both begin trading Thursday. The combined haul is a remarkable sum, more than 2 … Continue reading “Sequencing Firm Adaptive’s $300M IPO Comes With Pharma Ambitions”
Broad, Berkeley Return to Argue CRISPR Invention at Patent Office
A special board of the US Patent and Trademark Office has reignited the long-running patent fight over who invented the groundbreaking CRISPR-Cas9 gene editing system. Nearly a year ago, the matter seemed put to rest after a federal court upheld a ruling of the same patent office board. The court validated a key 2014 patent … Continue reading “Broad, Berkeley Return to Argue CRISPR Invention at Patent Office”
Bio Roundup: ASCO Wrap, Bluebird’s EU Tap, CRISPR Baby Risk & More
Welcome to conference week. That’s not an official moniker, but is there any busier time on the biopharma calendar? As the American Society of Clinical Oncology meeting wrapped up in Chicago, the annual BIO conference kicked off in Philadelphia, and a deep dive into diabetes begins today in San Francisco. With half the biopharma world, … Continue reading “Bio Roundup: ASCO Wrap, Bluebird’s EU Tap, CRISPR Baby Risk & More”
ASCO 2019: The Long Game, Targeted Pills, First-Ever Buzz & More
The American Society of Clinical Oncology meeting in Chicago has wrapped up for another year. Last week, we featured two stories that you wouldn’t find at the ASCO frenzy: Immunotherapy’s lack of progress in treating breast cancer, and one woman’s risky bet of tens of millions of dollars from her personal fortune to speed drug … Continue reading “ASCO 2019: The Long Game, Targeted Pills, First-Ever Buzz & More”
Study: Gene Edits in CRISPR Babies Could Boost Risk of Earlier Death
The Chinese scientist He Jiankui, who revealed last fall that he used CRISPR gene editing to try to make twin newborn girls immune to HIV infection, might have also given them a higher risk of death. That’s according to a new study from University of California, Berkeley data scientists who analyzed the records of more … Continue reading “Study: Gene Edits in CRISPR Babies Could Boost Risk of Earlier Death”
Can Tiny Drug Doses (and One Woman’s Fortune) Fight the Most Vicious Cancer?
When the brain goes bad, modern medicine is often powerless to help. That’s the case for Alzheimer’s disease, where drug after drug has failed, and the only approved treatments are marginally helpful at best. The same seems increasingly clear for the most common and aggressive type of brain cancer, glioblastoma multiforme. Its origins are mysterious, … Continue reading “Can Tiny Drug Doses (and One Woman’s Fortune) Fight the Most Vicious Cancer?”
To Catch Early Cancer, and Rivals, Thrive Banks on Hopkins Blood Test
The earlier cancer is diagnosed, the better the odds of survival, and the race is on to detect the earliest traces of the disease from an otherwise healthy person’s blood sample. A new entrant in that race has emerged today. Thrive Earlier Detection has raised $110 million in financing from biotech investors Third Rock Ventures … Continue reading “To Catch Early Cancer, and Rivals, Thrive Banks on Hopkins Blood Test”
With Sliver of Data, BioMarin to Seek OK for Hemophilia Gene Therapy
A gene therapy for hemophilia could be on the market by the end of next year, according to its developer BioMarin Pharmaceutical, if regulators agree that a tiny amount of data show enough promise. San Rafael, CA-based BioMarin (NASDAQ: [[ticker:BMRN]]) said this morning that a single dose of the therapy, valoctocogene roxaparvovec—known in shorthand as … Continue reading “With Sliver of Data, BioMarin to Seek OK for Hemophilia Gene Therapy”
Bio Roundup: Zolgensma Watch, Brain Cancer Blues, Peloton Payout & More
Patients, doctors, family members, and drug-price watchdogs continued to wait for the expected approval of Zolgensma, a Novartis (NYSE: [[ticker:NVS]]) gene therapy for the rare inherited disease spinal muscular atrophy. Originally developed at a children’s hospital in Ohio, Zolgensma will be a litmus test for the nascent field. If approved this week or next, it … Continue reading “Bio Roundup: Zolgensma Watch, Brain Cancer Blues, Peloton Payout & More”
Verve Has Top Cardio-Geneticist, $58.5M to Aim CRISPR at Heart Attacks
A new startup wants to edit out the world’s biggest killer. Prominent cardiologist and geneticist Sekar Kathiresan is heading up a new company, Verve Therapeutics, that will develop gene-altering medicine to prevent heart attacks. Heart disease is the leading cause of death in the US and worldwide. “Imagine an injection, once in life, that safely … Continue reading “Verve Has Top Cardio-Geneticist, $58.5M to Aim CRISPR at Heart Attacks”
LunaDNA, Vivid & More: The Xconomy Awards San Diego Startup Finalists
The finalists in the Startup category of San Diego’s Xconomy Awards (and other categories) show how much data security and privacy have become not just top public concerns but also the priorities of a growing number of companies. Other Startup finalists are taking various approaches to making precision medicine a reality for more patients. Here … Continue reading “LunaDNA, Vivid & More: The Xconomy Awards San Diego Startup Finalists”
Bio Roundup: Head and Spine Previews, Biogen Moves, IPO Dreams & More
America continues to suffer from healthcare headaches. Our convoluted private-public insurance system was under scrutiny this week from Democrats, holding a “Medicare for All” hearing in the House, and from Republicans, as several GOP-led states and the Trump administration asked a federal court to sweep away Obamacare. Several companies also want to reduce headaches. This … Continue reading “Bio Roundup: Head and Spine Previews, Biogen Moves, IPO Dreams & More”
Can We Afford to Be Cured? A Conversation With ICER’s Steve Pearson
[Corrected, 4/23/19, 7:10 p.m. See below.] New cell and gene therapies bring the possibility of cures once hardly imaginable. But the potential cures could also cost millions of dollars, like Novartis (NYSE: [[ticker:NVS]]), the owner of the gene therapy Zolgensma, has suggested in advance of an imminent FDA approval decision. As public backlash against high … Continue reading “Can We Afford to Be Cured? A Conversation With ICER’s Steve Pearson”
Bio Roundup: EASL Does It, Alnylam’s BFF, Gene Therapy Deals & More
A major conference devoted to liver disease kicked off with updates from the race for approval of the world’s first drug to treat a form of advanced liver disease called nonalcoholic steatohepatitis, or NASH. The gene therapy field continued to attract venture cash and industry interest, and it was a busy week in Washington for … Continue reading “Bio Roundup: EASL Does It, Alnylam’s BFF, Gene Therapy Deals & More”
Intercept Touts a Smaller Piece of Its NASH Trial at Liver Conference
Intercept Pharmaceuticals (NASDAQ: [[ticker:ICPT]]) said today it was on track to ask US and European regulators this year to approve what could be the first drug for an advanced liver disease that poor diet and exercise habits have turned into a quiet epidemic. The disease is called nonalcoholic steatohepatitis, or NASH, and as the name … Continue reading “Intercept Touts a Smaller Piece of Its NASH Trial at Liver Conference”
Report: Biogen, Novartis Should Drop Prices of Billon-Dollar SMA Drugs
A new report from a U.S. drug-pricing watchdog takes issue with the high cost of the only approved treatment for spinal muscular atrophy (SMA), a rare genetic disease that affects young children and in some cases leads to a quick death. The report, from the Boston-based Institute for Clinical and Economic Review (ICER), is loaded … Continue reading “Report: Biogen, Novartis Should Drop Prices of Billon-Dollar SMA Drugs”
Analysts: Proteostasis Data Are No Boost vs. Vertex in Cystic Fibrosis
As with cancer and HIV infection, progress in treatment for the genetic disorder cystic fibrosis has come with ever-more sophisticated cocktails of drugs. Proteostasis Therapeutics (NASDAQ: [[ticker:PTI]]) is one of the firms trying to catch up to cystic fibrosis leader Vertex Pharmaceuticals (NASDAQ: [[ticker:VRTX]]), but data it reported this morning did not satisfy investors, who … Continue reading “Analysts: Proteostasis Data Are No Boost vs. Vertex in Cystic Fibrosis”
Bio Roundup: Sage Postpartum Help, Biogen Bids Adu, Heart Beats & More
One group of people in dire need of medical relief got good news this week. The first drug for postpartum depression was approved. With its complicated logistics, side effects, and potential high cost, it won’t be for everyone who experiences the condition—1 of every 9 U.S. women giving birth. But at least it’s an option. … Continue reading “Bio Roundup: Sage Postpartum Help, Biogen Bids Adu, Heart Beats & More”
Drug-Price Watchdog ICER Dives into Trump-Fueled Pharma Rebate Debate
[Updated 3/13/19, 12:20pm. See below.] With public and political winds blowing in the same direction, significant reform of the complicated U.S. drug-pricing system seems ever more likely. One part of the system that the Trump administration wants to overhaul are the secret rebates that drug makers, insurers, and middlemen negotiate behind closed doors. Replacements for … Continue reading “Drug-Price Watchdog ICER Dives into Trump-Fueled Pharma Rebate Debate”
Bio Roundup: Gottlieb Exits, Bristol Defends, Biogen Buys & More
In early 2017, Scott Gottlieb was considered the most moderate of the potential candidates to head the Food and Drug Administration, which is the most powerful regulator of medical products in the world. It wasn’t a high bar to clear. Libertarians who had questioned some of the basic premises of the FDA were in the … Continue reading “Bio Roundup: Gottlieb Exits, Bristol Defends, Biogen Buys & More”
Two Years and Done: FDA Commissioner Gottlieb To Resign
Two months after insisting on Twitter that he wasn’t going anywhere, Food and Drug Administration Commissioner Scott Gottlieb announced his resignation today. He leaves a record of health and medical regulation that was more active than critics who spoke out against his nomination in early 2017 might have expected. Gottlieb’s remit extended beyond drugs, of … Continue reading “Two Years and Done: FDA Commissioner Gottlieb To Resign”
Zafgen Taps Biogen Exec Priya Singhal as New R&D Chief
On the heels of positive Phase 2 data for its lead product, an experimental type 2 diabetes treatment, Zafgen (NASDAQ: [[ticker:ZFGN]]) has hired Priya Singhal as head of R&D. Singhal was most recently a senior VP at Biogen. Zafgen is trying to bounce back from the 2016 decision to abandon its then-lead drug candidate beloranib, … Continue reading “Zafgen Taps Biogen Exec Priya Singhal as New R&D Chief”
With $191M Haul, Maze Therapeutics Takes Aim at Genetic Diseases
People who share the same disease-causing genetic mutation can have a wide range of severity in their disease. Some don’t get sick at all. Environment can play a role, but with ever-more genetic data available, scientists are unraveling how much other genes can counteract the effect of the main disease-causing gene, too. Two high-profile biotech … Continue reading “With $191M Haul, Maze Therapeutics Takes Aim at Genetic Diseases”
Pharma CEOs to Senate: We Will Lower Drug Prices if Rebates Go Away
Seven top pharmaceutical executives gathered today in Washington, DC, for a Senate hearing on drug prices, bringing face-to-face two groups held in the lowest esteem possible by the American public. Top executives from seven companies—AbbVie, AstraZeneca, Bristol-Myers Squibb, Merck, Johnson & Johnson, Pfizer, and Sanofi, which represent a combined $1.1 trillion in market value—stuck to … Continue reading “Pharma CEOs to Senate: We Will Lower Drug Prices if Rebates Go Away”
Bio Roundup: Trump’s Scalps, Policy Pressure, NASH Crash & More
While politicians continue to debate what kind of healthcare system is best for the American people, the mostly U.S.-based pharmaceutical industry is trying to convince those same politicians—and anyone else who will listen—that what’s good for the industry is also good for America. Forcing drug prices lower, industry argues, would be bad for our health, … Continue reading “Bio Roundup: Trump’s Scalps, Policy Pressure, NASH Crash & More”
With Low Expectations, Gilead’s Top NASH Drug Fails 1st Major Test
Gilead Sciences (NASDAQ: [[ticker:GILD]]) said after market close Monday that its most advanced candidate to treat the fatty liver disease known as NASH failed the first of two Phase 3 trials. NASH, or nonalcoholic steatohepatitis, is a quickly growing condition, thanks to the obesity epidemic. It causes liver inflammation and scarring, and when it progresses … Continue reading “With Low Expectations, Gilead’s Top NASH Drug Fails 1st Major Test”
Abeona Therapeutics CEO João Siffert Sheds “Interim” Title
After three months as the interim CEO of gene-therapy developer Abeona Therapeutics (NASDAQ: [[ticker:ABEO]]), João Siffert will drop the interim part of his title. Siffert took over when previous CEO Carsten Thiel was suddenly ousted for what the company called “personal misconduct.” Thiel had only been in the position for seven months. Siffert joined Abeona … Continue reading “Abeona Therapeutics CEO João Siffert Sheds “Interim” Title”
In Landmark Gene-Editing Study, Sangamo Reports Little Benefit
The first U.S. trial of a gene-editing medicine in humans isn’t going well. Sangamo Therapeutics (NASDAQ: [[ticker:SGMO]]) reported Phase 1 results this morning for its treatment for Hunter syndrome, a rare genetic disease. While relatively safe, the treatment, known as SB-913, did not seem to help patients much, based on measurements of proteins in their … Continue reading “In Landmark Gene-Editing Study, Sangamo Reports Little Benefit”
New CRISPR Fight: Old Friends Intellia, Caribou in License Dispute
An epic legal battle over CRISPR-Cas9 genome editing ended last year. But another one, smaller in scope and involving two long-time CRISPR allies, has quietly begun. Last October, Intellia Therapeutics (NASDAQ: [[ticker:NTLA]]) began an arbitration proceeding against Caribou Biosciences, alleging in a recent regulatory filing that its longstanding partner had broken the terms of a … Continue reading “New CRISPR Fight: Old Friends Intellia, Caribou in License Dispute”
Bio Roundup: Bosley’s Editas Exit, Bridge Bucks, CRISPR Crime & More
In a week of big personnel moves, none was more surprising than Editas Medicine CEO Katrine Bosley’s departure. After guiding the CRISPR-Cas9 genome-editing startup from scientific breakthrough toward the development of a potential cutting-edge medicine, slogging victoriously through a legendary patent fight, and reaching the cusp of an historic clinical trial, the well-traveled Bosley is … Continue reading “Bio Roundup: Bosley’s Editas Exit, Bridge Bucks, CRISPR Crime & More”
FDA’s Gottlieb Adds Confusion to Aimmune’s Claims of Shutdown Delay
The first biotech company to cite the partial government shutdown as a reason for serious delay was Aimmune Therapeutics (NASDAQ: [[ticker:AIMT]]), as Xconomy reported Tuesday. The announcement, however, soon raised more questions than answers. Aimmune reported in a regulatory filing Tuesday that the FDA would not start reviewing its peanut-allergy treatment, AR101, because of the … Continue reading “FDA’s Gottlieb Adds Confusion to Aimmune’s Claims of Shutdown Delay”
Notes from the JPM19 Vortex: Price Apocalypse, Celgene Fallout & More
[Editor’s note: Ben Fidler and Sarah de Crescenzo coauthored this report.] It is here, and then it is gone. But by the time everyone staggers to the airport or back home to sleep in their own beds, the annual few days of the J.P. Morgan Healthcare Conference and everything that mushrooms around it seem just … Continue reading “Notes from the JPM19 Vortex: Price Apocalypse, Celgene Fallout & More”
Bio Roundup: $74B for Celgene, Two CAR-T Tales, Ready for SF & More
In 2018, my Exome colleagues and I published hundreds of stories about health, medicine, the biopharma industry, government policy, and more. You’ll find a few of our favorite stories in this review of some of the year’s best from across the Xconomy network. Topics ranged from the worries over CRISPR genome editing and Wild West … Continue reading “Bio Roundup: $74B for Celgene, Two CAR-T Tales, Ready for SF & More”
For CAR-T Cancer Fighters in the Real World, Two Roads Diverge
[Corrected, 1/4/19, 3:55pm ET. See below.] A generation ago, cancer treatments made from a patient’s own living immune cells would have been science fiction. Now they’re here. The first two products, approved in 2017 and known in shorthand as CAR-T, have brought some people with otherwise untreatable blood cancers back from the brink of death. … Continue reading “For CAR-T Cancer Fighters in the Real World, Two Roads Diverge”
Bio Roundup: 2019 Trials, Pilgrim Price Chat, Pre-Xmas Sales & More
It’s beginning to look a lot like, well, the last roundup of the year, with plenty of news to pack in before our holiday breather. Whew. Before you fly off, or drive, or simply unplug, check out our preview of what could be next year’s top clinical studies; the tumult in big pharma’s on again-off … Continue reading “Bio Roundup: 2019 Trials, Pilgrim Price Chat, Pre-Xmas Sales & More”
“JK Told Me He Was Planning This”: A CRISPR Baby Q&A with Matt Porteus
Last week, Chinese researcher He Jiankui stunned the world with his claim to have created the world’s first gene-edited humans. He said he had used CRISPR-Cas9 to change the DNA of the embryos of twin girls, to make them immune to HIV infection, then implanted them in their mother’s womb. He announced on video the … Continue reading ““JK Told Me He Was Planning This”: A CRISPR Baby Q&A with Matt Porteus”
Bio Roundup: CRISPR Babies, Blood Diseases, Big Cancer Nod & More
This was one of those weeks when the world seemed to slip into a new era with no going back. A Chinese researcher, He Jiankui, claimed he helped bring to life two genetically engineered babies, twin girls with a gene disabled to make them immune to HIV infection. Even though the technology He used—in vitro … Continue reading “Bio Roundup: CRISPR Babies, Blood Diseases, Big Cancer Nod & More”
Under Fire, He Jiankui Says He’s “Proud” to Help Make CRISPR’d Babies
[Updated, 11/28/18, 12:20pm ET. See below.] Is there another CRISPR baby on the way? In his first public appearance since his stunning claim, released on YouTube Sunday, that he helped bring the first genome-edited humans into the world, He Jiankui, a genomics researcher at Southern University of Science and Technology of China in Shenzhen, said … Continue reading “Under Fire, He Jiankui Says He’s “Proud” to Help Make CRISPR’d Babies”