The cholesterol brawl is heating up again. A pair of next-generation cholesterol-lowering drugs known as PCSK9 inhibitors came to market in 2015 and have since struggled to gain traction. Now Ann Arbor, MI-based biotech Esperion Therapeutics (NASDAQ: [[ticker:ESPR]]) thinks it can do with a once-a-day pill called bempedoic acid what the PCSK9 blockers have not: … Continue reading “Esperion Touts Low Cholesterol Drug Price, But Not All Data are Ready”
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.
Helix Raises $200M to Build Its DNA “App Store” Beyond the Family Tree
Thanks to advertising blitzes from companies like Ancestry.com and 23andMe, Joe and Jane Consumer might know that their genetic blueprint and $100 or so can dig up insights about their genealogy. San Carlos, CA-based startup Helix is banking on that budding curiosity to provide a market for all kinds of consumer apps fueled by a … Continue reading “Helix Raises $200M to Build Its DNA “App Store” Beyond the Family Tree”
Bio Roundup: Trump’s Budget, Biogen’s AD Reveal, Hugin’s Run & More
Another week of White House chaos and American gun violence. Amid it all, we tried to parse what the Trump administration’s 2019 budget proposal meant for the biomedical and life-science worlds. There was talk of tackling high drug prices in the budget document, but nothing to give Medicare direct negotiating power with drug companies or … Continue reading “Bio Roundup: Trump’s Budget, Biogen’s AD Reveal, Hugin’s Run & More”
Bristol-Myers Bets Nearly $2B on Nektar for an Immunotherapy Boost
When it comes to spending cash on potential combinations for its cancer immunotherapy drugs, Bristol-Myers Squibb (NYSE: [[ticker:BMY]]) isn’t afraid of the b-word: billions. The New York-based pharma firm is paying Nektar Therapeutics (NASDAQ: [[ticker:NKTR]]) of San Francisco $1 billion in cash and buying another $850 million in shares, all to grab partial rights to … Continue reading “Bristol-Myers Bets Nearly $2B on Nektar for an Immunotherapy Boost”
Trump Budget Touts Lower Drug Prices, But No Medicare Negotiation
As president-elect, Donald Trump in January 2017 slammed drug companies for “getting away with murder” and said he supported Medicare negotiation. He vowed during last month’s State of the Union speech that drug prices “will come down.” Today, his administration’s fiscal 2019 budget proposal laid out what it calls a “comprehensive” strategy to lower drug … Continue reading “Trump Budget Touts Lower Drug Prices, But No Medicare Negotiation”
Medical and Patient Groups Warn Congress Against “Right to Try”
Dozens of medical organizations and patient advocacy groups are pushing back against so-called “right-to-try” legislation that passed the Senate in August and is now under consideration in the House. An open letter sent today to House leaders is the latest opposition to the Republican-led push to give desperate patients access to experimental drugs without FDA … Continue reading “Medical and Patient Groups Warn Congress Against “Right to Try””
Bellicum Reveals Brain Injuries, FDA Halt on Lead T-Cell Product
Bellicum Pharmaceuticals (NASDAQ: [[ticker:BLCM]]) of Houston divulged Wednesday that its lead T-cell therapy program BPX-501 has been put on hold by the FDA. The treatment, for certain people undergoing bone marrow transplant, uses two components: off-the-shelf T cells to replace those killed in the transplant process, and a pill that acts as a “kill switch” … Continue reading “Bellicum Reveals Brain Injuries, FDA Halt on Lead T-Cell Product”
Bio Roundup: Spark v. ICER, A Solid Revelation, T Cell Deals & More
The United States has a new Health and Human Services chief who is expected to weigh in on the country’s drug-pricing debate. But how Alex Azar, a former pharma executive at Eli Lilly (NYSE: [[ticker:LLY]]), plans to throw his weight around remains to be seen. One of the latest exhibits in the debate is an … Continue reading “Bio Roundup: Spark v. ICER, A Solid Revelation, T Cell Deals & More”
CRISPR Recovery? “Furor” Briefly Snips $500M From Companies’ Values
Is the J.P. Morgan CRISPR rollercoaster ride over? As rain fell heavily in San Francisco Monday, the first day of the massive healthcare conference, biotech stocks did, too. At least three of the companies getting drenched could blame a research paper that came out the previous Friday, January 5, after the markets closed. In fact, … Continue reading “CRISPR Recovery? “Furor” Briefly Snips $500M From Companies’ Values”
After 2nd Bet on Familiar Drug, Impact Bio Lands $1B Celgene Buyout
Celgene (NASDAQ: [[ticker:CELG]]) has kicked off J.P. Morgan week—the annual healthcare investment gathering in San Francisco—with a blockbuster deal, acquiring privately held Impact Biomedicines for $1.1 billion upfront in cash. More is in the offing if Impact’s drug fedratinib passes a series of regulatory and sales hurdles. The deal rewards the determination of San Diego … Continue reading “After 2nd Bet on Familiar Drug, Impact Bio Lands $1B Celgene Buyout”
Bio Roundup: Trump, Gottlieb, Gene Therapy Prices, IPO Hopes & More
Welcome to the new year. What will it bring? Hopefully a lot less hatred, greed, stupidity, and angst than 2017. But we’re not holding our breath. We emerged from the winter break pondering several of the year’s burning biopharma questions, including the volatility of the U.S. president and what it means for the biopharmaceutical business, … Continue reading “Bio Roundup: Trump, Gottlieb, Gene Therapy Prices, IPO Hopes & More”
First U.S. Gene Therapy, Approved for Vision Loss, to Cost $850,000
The first gene therapy approved in the United States will cost $850,000, its developer Spark Therapeutics (NASDAQ: [[ticker:ONCE]]) announced today. Spark hopes to reduce the sticker shock by offering refunds and other creative pricing ideas, but those carve-outs, at best, are limited, and some are unlikely to come to fruition, according to healthcare economists. The … Continue reading “First U.S. Gene Therapy, Approved for Vision Loss, to Cost $850,000”
Trump v. Drug Makers? And 6 More Burning Biopharma Questions for 2018
Next week, a big slice of the biomedical industry is gathering in San Francisco for the annual J.P. Morgan Healthcare Conference and other life sciences events that have emerged like sprouts around the base of an old redwood tree. One year ago, the pharmaceutical folks—by far the largest delegation to this annual mosh pit of … Continue reading “Trump v. Drug Makers? And 6 More Burning Biopharma Questions for 2018”
Bio Roundup: Tax Cuts, a Funding Rush, Hemophilia Questions & More
In the penultimate week before the end-of-year recess, Congress raced to provide the drug industry—and the rest of corporate America—a massive tax cut, with breaks for investors, too. Racing to finish their own end-of-year budgets, perhaps, investors poured hundreds of millions into private biotechs, too. If that doesn’t get your blood racing, then how about … Continue reading “Bio Roundup: Tax Cuts, a Funding Rush, Hemophilia Questions & More”
ASH 2017 Highlights: CAR-T Competition, Myeloma, Hemophilia & More
[Updated, 12/13/17, 1:50 p.m. ET. See below.] The annual American Society of Hematology meeting is nearly over. For now, we’ve chosen updates from three disease areas to highlight, plus the more notable market movements. No surprise, there’s a lot about the genetically engineered cell therapy known as CAR-T. Gene therapy and more standard forms of … Continue reading “ASH 2017 Highlights: CAR-T Competition, Myeloma, Hemophilia & More”
15 For ’18: Key Clinical Data to Watch For Next Year (Part 2)
[Corrected, 12/11/17, 1 p.m. ET. See below.] On Monday we posted the first part of our look at what should be some of 2018’s most important clinical data, including trials for lung cancer, heart disease, melanoma, Alzheimer’s disease, rare blood disorders, and more. With part two, we’re previewing studies for migraine, brain cancer, lymphoma, peanut … Continue reading “15 For ’18: Key Clinical Data to Watch For Next Year (Part 2)”
Bio Roundup: ICER Eyes Cost, Azar at HHS, Gates’s ALZ Cash & More
In our roundup this week, Alzheimer’s disease is in the news, thanks to billionaire Bill Gates and his personal pledge of $100 million toward R&D. That could equal the amount one startup hopes to raise in an IPO to develop drugs for Alzheimer’s and other brain diseases. If a treatment became available, could our health … Continue reading “Bio Roundup: ICER Eyes Cost, Azar at HHS, Gates’s ALZ Cash & More”
At Seattle Children’s, Another Piece of Ambitious CAR-T Plan in Place
The researchers and doctors at Seattle Children’s are embarking upon a complicated quest, involving multiple clinical studies of cutting-edge CAR-T cell therapies, to help kids and young adults with an aggressive form of leukemia. As of today, one key part of their plan can begin, thanks to a green light from the FDA. New clinical … Continue reading “At Seattle Children’s, Another Piece of Ambitious CAR-T Plan in Place”
IPO to Fuel Denali’s Drugs for Alzheimer’s, Other Brain Diseases
One of biotech’s richest private companies, Denali Therapeutics, has set its sights on an IPO to push ahead with treatments for Alzheimer’s and other confounding brain diseases. The filing marks $100 million as its IPO target, but market conditions often shift a company’s sights in the run-up to a debut. Denali’s disclosure comes on the … Continue reading “IPO to Fuel Denali’s Drugs for Alzheimer’s, Other Brain Diseases”
Juno Dissects ROCKET Deaths, Will Aim Better CAR-T at Leukemia
When five patients with a difficult type of leukemia died last year in a clinical trial run by Juno Therapeutics, it was a big setback for the Seattle biotech and its plans for cutting-edge CAR-T cancer therapy. A year later, Juno is finally explaining what happened and promising to return with a new product. The … Continue reading “Juno Dissects ROCKET Deaths, Will Aim Better CAR-T at Leukemia”
ASH Sneak Peeks: Novartis, Juno Update CAR-T Lymphoma Competition
[Corrected, 11/1/17, 5:20 p.m. ET. See below.] The annual American Society of Hematology meeting is next month. But previews of the big data presentations are out today, yielding more insight into what promises to be the first major competition in CAR-T cell therapy: Treatments for people with non-Hodgkin lymphoma (NHL) who have run out of … Continue reading “ASH Sneak Peeks: Novartis, Juno Update CAR-T Lymphoma Competition”
New Survey Highlights “Lethargic Pace” of Healthcare Gender Equity
To close the healthcare and life sciences gender gap, there have been programs to move women into the boardroom. People are building mentoring networks. And there have been public pledges to do better. But women in the industry aren’t optimistic about big changes coming soon, according to a new report and survey from digital healthcare … Continue reading “New Survey Highlights “Lethargic Pace” of Healthcare Gender Equity”
Bio Roundup: CBO’s Praise, Celgene’s Shock, Trump’s Opioid Plan & More
The White House and the FDA turned the spotlight on the nation’s opioid crisis. Blockbuster drugs from Alexion and Tesaro gained expanded approvals, but bad earnings news from Celgene and Biogen brought stocks down. Academics in the U.S. and China made progress with the gene editing technology CRISPR. And as the week’s biggest biotech deal … Continue reading “Bio Roundup: CBO’s Praise, Celgene’s Shock, Trump’s Opioid Plan & More”
As Drugs Fail, Some Researchers Urge a Return to Alzheimer’s Roots
As the quest to find treatments for Alzheimer’s disease keeps crashing against rocks, a group of researchers in the field want their peers to pay more attention to the part of the brain where Alzheimer’s disease first wreaks havoc. A new paper from the researchers, published this month in the journal Alzheimer’s & Dementia, is … Continue reading “As Drugs Fail, Some Researchers Urge a Return to Alzheimer’s Roots”
Kite Nabs First Adult CAR-T Approval With Notable Uptick Of Cures
For the second time in two months, remarkable recoveries from desperate cases of cancer have led to early approval of a cutting-edge therapy called CAR-T, which is made from a patient’s own genetically engineered cells. More than a month before the decision was due, the FDA has given the nod to axicabtagene ciloleucel, now branded … Continue reading “Kite Nabs First Adult CAR-T Approval With Notable Uptick Of Cures”
After Trump Sounds Off, Senate Has Adult Conversation on Drug Prices
In advance of an anticipated Senate hearing on drug prices, President Donald Trump once again denounced drug companies as “getting away with murder.” The first time he did so, using the same phrase just before his inauguration, the markets shuddered. He also said at the time that “we’re going to start bidding,” a reference to … Continue reading “After Trump Sounds Off, Senate Has Adult Conversation on Drug Prices”
From UCSF, New Sequencer Reveals Cancer Blueprints, Cell By Cell
Cells within a single tumor can have a variety of genetic fingerprints. Understanding that variety is likely a key to fighting cancer in the near future. A new biotech firm has developed a device that analyzes tumors cell by cell. Single-cell analysis is nothing new. But Mission Bio, of South San Francisco, says it taken … Continue reading “From UCSF, New Sequencer Reveals Cancer Blueprints, Cell By Cell”
Bio Roundup: Light For Spark, California Drug Law, IPO Queue & More
In a year of tragedies across the U.S., natural or otherwise, it seemed this week was California’s turn. Fueled by high winds, low humidity, and ample dry vegetation, fires swept through several towns across the state and, as of this writing, have barely been contained. With our minds on friends and loved ones affected by … Continue reading “Bio Roundup: Light For Spark, California Drug Law, IPO Queue & More”
FDA To Advisors: What If One-Time Gene Therapy Needs Extra Doses?
In the run-up to a key vote on an experimental gene therapy, the FDA raised no major flags about the drug’s track record but said its long-term effect remains an open question. That question should receive a thorough airing Thursday, when a meeting of FDA outside advisors will weigh a recommendation of the one-shot gene … Continue reading “FDA To Advisors: What If One-Time Gene Therapy Needs Extra Doses?”
Bio Roundup: Nobel Pursuit, Fundraising Fruit, A Rhythmic IPO & More
The 2017 Nobel Prizes with a biomedical bent were announced this week. Three researchers shared the physiology and medicine prize for their work shining light on the circadian rhythms that govern the wake-and-sleep cycles of all organisms. That basic research is working its way into applications such as drugs that help regulate our body clocks. … Continue reading “Bio Roundup: Nobel Pursuit, Fundraising Fruit, A Rhythmic IPO & More”
CRISPR Therapeutics Switches CEOs As It Approaches First Clinical Study
With its first clinical trial possibly just months away, one of the leading biotech firms using CRISPR-Cas9 gene editing technology to make drugs is turning to a new CEO. CRISPR Therapeutics (NASDAQ: [[ticker:CRSP]]), a company split between Cambridge, MA, and Switzerland, said this morning that founding CEO Rodger Novak will hand the reins to the … Continue reading “CRISPR Therapeutics Switches CEOs As It Approaches First Clinical Study”
No Effect: Axovant Drug Is Latest Casualty in Alzheimer’s Battle
Another high-profile attempt to produce an Alzheimer’s treatment has failed. An unusual biotech company called Axovant Sciences (NASDAQ: [[ticker:AXON]]) reported today that its drug intepirdine did not show a meaningful difference between the patients taking a combination of intepirdine and donepezil (Aricept) and the patients taking only donepezil, which is one of just a handful … Continue reading “No Effect: Axovant Drug Is Latest Casualty in Alzheimer’s Battle”
On CAR-T’s Edge, Seattle Researchers Plot to Bypass Novartis Therapy
[Corrected, 10/10/17, 2 p.m. ET. See below.] It’s not a stretch to say that Greta Oberhofer is alive thanks to the genetically modified cell therapy called CAR-T. At three and a half months old, she was diagnosed with acute lymphoblastic leukemia, or ALL, an aggressive blood cancer. At 10 months old, she had a bone … Continue reading “On CAR-T’s Edge, Seattle Researchers Plot to Bypass Novartis Therapy”
Bio Roundup: Phase 3 Fails, Shkreli Jailed, Moderna Flip-Flops & More
It was a week of realignments. President Trump, who used to be known as “The Donald” but now goes by “Amnesty Don,” kept up the parlay-voo with his new friends Chuck and Nancy. Martin Shkreli, who was once known—and still is!—as the “pharma bro,” will have a new perspective for a while, through the bars … Continue reading “Bio Roundup: Phase 3 Fails, Shkreli Jailed, Moderna Flip-Flops & More”
Xconomy Award Finalists in the Eye of the National Drug Price Debate
[Corrected 12:20pm ET; see below.] The national debate over the high cost of healthcare, with prescription drugs at the center, resonates from the chambers of Congress to every American’s pocketbook. And it’s growing fiercer by the day. Last week, the first genetically modified cell therapy came to market with a price tag of $475,000. This … Continue reading “Xconomy Award Finalists in the Eye of the National Drug Price Debate”
Immunotherapy Safety Worries Halt Celgene Myeloma Study, Slow 5 Others
Amid growing concerns about the safety of a new class of cancer immunotherapy drugs when combined with other therapies to treat blood cancers, the FDA has put the brakes on another batch of clinical studies, this time run by the publicly traded biotech Celgene (NASDAQ: [[ticker:CELG]]). Celgene said this morning that the nation’s drug regulator … Continue reading “Immunotherapy Safety Worries Halt Celgene Myeloma Study, Slow 5 Others”
Bio Roundup: CAR-T’s Huge Week, Merck’s ‘Pib Choice & More
[Corrected 9/5/17, 12:22 p.m. See below.] The far-out idea of reprogramming a patient’s immune cells to fight cancer, known as CAR-T, entered the mainstream this week, and two numbers highlight the tale. The first is $11.9 billion, the price Gilead Sciences is paying for Kite Pharma and its cancer-fighting cell therapy pipeline. It’s one of … Continue reading “Bio Roundup: CAR-T’s Huge Week, Merck’s ‘Pib Choice & More”
Novartis: CAR-T Results In One Month, Or No Charge. Why One Month?
Novartis said Wednesday that it would not charge for its newly approved cellular immunotherapy, tisagenlecleucel (Kymriah)—the first so-called CAR-T product to ever come to market—if it fails to help patients within a month. If the cell therapy, approved for kids with leukemia who have run out of other options, starts to work within a month, … Continue reading “Novartis: CAR-T Results In One Month, Or No Charge. Why One Month?”
Novartis Wins Historic FDA Nod, Prices CAR-T For Leukemia at $475,000
Ahead of schedule, Novartis has received the first-ever approval of a genetically modified living cell therapy called CAR-T, which uses a cancer patient’s own immune cells as the medicine. Within days, children and young adults with a severe form of acute lymphoblastic leukemia could receive the treatment at a handful of centers across the U.S. … Continue reading “Novartis Wins Historic FDA Nod, Prices CAR-T For Leukemia at $475,000”
To Be More Than Quick Splash, Gilead’s CAR-T Purchase Faces Hurdles
[Updated 8/28/17, 6:45p.m. ET. See below.] Gilead Sciences CEO John Milligan promised today that the company’s $11.9 billion acquisition of Kite Pharma and its cutting-edge CAR-T cell therapy would not be a “one and done” proposition. “This is a long-term play with multiple product opportunities,” Milligan said, calling it the foundation of Gilead’s “fairly nascent” … Continue reading “To Be More Than Quick Splash, Gilead’s CAR-T Purchase Faces Hurdles”
70% Repatha Cut? Amgen Counters With Own Study To Justify Price
Writing in the Journal of the American Medical Association, researchers at the University of California, San Francisco argued this week that the price of the cholesterol-fighting drug evolocumab (Repatha) should come down 70 percent, to $4,215 a year, to be cost-effective. But a separate study, published today in JAMA Cardiology and funded by the drug’s … Continue reading “70% Repatha Cut? Amgen Counters With Own Study To Justify Price”
Bio Roundup: $1B Deals, Data Bumps, Acorda’s Lumps, Generic OKs & More
With President Trump holed up in New Jersey, rattling his saber at Kim Jong-un and Mitch McConnell and ignoring his own commission’s advice on the opioid crisis, there was still life sciences news from the nation’s capital. The FDA reported that generic drug approvals are set to hit a record; the agency under Commissioner Scott … Continue reading “Bio Roundup: $1B Deals, Data Bumps, Acorda’s Lumps, Generic OKs & More”
Celgene Narrows Its Relationship With Sutro, No Acquisition Coming
Celgene (NASDAQ: [[ticker:CELG]]) is revamping its relationship with Sutro Biopharma, walking away from an option to acquire the privately held antibody drug developer but keeping potential rights to four of Sutro’s programs—none of which have yet reached clinical studies. Their first deal was in 2012, and they expanded it two years later with Celgene taking … Continue reading “Celgene Narrows Its Relationship With Sutro, No Acquisition Coming”
Report: Martin Shkreli Faces Prison Term After Fraud Conviction
After days of deliberation, a New York jury Friday afternoon found Martin Shkreli guilty of securities fraud. According to a report from CNBC, the jury in the civil case said that Shkreli is guilty on three of eight counts, two of which are securities fraud and the other conspiracy to commit securities fraud. Shkreli, according … Continue reading “Report: Martin Shkreli Faces Prison Term After Fraud Conviction”
CRISPR Pioneer Zhang Preaches Extra Caution In Human Gene Editing
A leading genome-editing researcher is urging extra caution as drug companies race to turn the landmark technology he helped create into human medicine. In a paper published today in Nature Medicine, Feng Zhang of the Broad Institute of MIT and Harvard and colleague David Scott argue that researchers should analyze the DNA of patients before … Continue reading “CRISPR Pioneer Zhang Preaches Extra Caution In Human Gene Editing”
Merck Drug Shows No Life-Extending Benefit in Head-and-Neck Cancer
The first immunotherapy approved in the U.S. to treat head and neck cancer has failed a big test, but it’s unclear if the FDA will exercise its right to pull it from the market. Merck (NYSE: [[ticker:MRK]]) announced late Monday that its blockbuster cancer drug pembrolizumab (Keytruda) did not meet its main goal of helping … Continue reading “Merck Drug Shows No Life-Extending Benefit in Head-and-Neck Cancer”
Bio Roundup: Obamacare Persists, FDA Approves, Vertex Wows & More
The attempt to repeal and replace the Affordable Care Act took a major hit this week from Senate GOP holdouts who said the Better Care Reconciliation Act either tore down Medicaid too much or left too much of the ACA in place. A straight-up repeal, floated by Senate majority leader Mitch McConnell, was also shot … Continue reading “Bio Roundup: Obamacare Persists, FDA Approves, Vertex Wows & More”
A.I. Prizes, Coming to Healthcare, Hit $1M Mark in Cancer Contests
This story is part of an Xconomy series on artificial intelligence in healthcare. Some of the other stories cover a genomics hackathon, A.I. and radiology, and the impact on doctors and patients. In the classic 1967 film “The Graduate,” Dustin Hoffman’s just-out-of-college character gets one word of career advice from a family friend: plastics. In … Continue reading “A.I. Prizes, Coming to Healthcare, Hit $1M Mark in Cancer Contests”
Vertex Touts Positive Triple-Drug Data For Cystic Fibrosis Patients
Vertex Pharmaceuticals (NASDAQ: [[ticker:VRTX]]) continued its march away from being the biggest loser in the hepatitis C wars and toward increasing dominance in cystic fibrosis. The Boston firm announced Tuesday that studies of three experimental drug combinations all reported notable improvement in patients who have a different underlying genetic pattern than patients already eligible for … Continue reading “Vertex Touts Positive Triple-Drug Data For Cystic Fibrosis Patients”
First Slice of Long-Term “100K” Study: Useful Data, No Health Outcomes
It’s easy to find hope that new biomedical technologies, from genetic sequencing to wearable fitness trackers, will lead to a healthier populace. It’s harder to find evidence. There has even been caution about the idea of analyzing the DNA of seemingly healthy people. But a small study in Seattle called the Pioneer 100 Wellness Project, … Continue reading “First Slice of Long-Term “100K” Study: Useful Data, No Health Outcomes”