FDA Landmark Nod: Cancer Drug To Match Tumor Genes, Not Tissue Type

For many years, doctors and researchers have known that cancers in different parts of the body can share genetic abnormalities. For the first time, the FDA has approved a drug based on those abnormalities and not on the organ in which the cancer originated. It’s a moment that a lot of people in the cancer … Continue reading “FDA Landmark Nod: Cancer Drug To Match Tumor Genes, Not Tissue Type”

Not Resting on Hemophilia Biz, Bioverativ Pays $400M For True North

Less than four months into its existence, Biogen spinoff Bioverativ has made its first big splash. The company has acquired True North Therapeutics, a privately held South San Francisco, CA, biotech, as part of a plan to forge an identity around rare blood diseases, not the core hemophilia business it was built on. Waltham, MA-based … Continue reading “Not Resting on Hemophilia Biz, Bioverativ Pays $400M For True North”

Radius Shares Jump as Amgen’s Bone Drug Suffers a Setback in Phase 3

Amgen revealed late Sunday that safety problems have emerged in clinical testing of its closely watched osteoporosis drug, romosozumab (Evenity)—a development that will at minimum delay the drug’s path to approval, if not keep it off the market altogether. The Thousand Oaks, CA, company said while romosozumab met its main goal and some key secondary … Continue reading “Radius Shares Jump as Amgen’s Bone Drug Suffers a Setback in Phase 3”

Bio Roundup: Termeer Tribute, ASCO Peeks, Califf’s New Gigs & More

Exhausted by the nonstop drama in Washington, DC? Immerse yourself instead in this week’s biotech headlines. Let’s start with a tribute to a life sciences icon, then we’ll head around the Xconomy network with early news from the nation’s big yearly cancer meeting. There was also momentum onthe IPO front and new gigs for high-profile … Continue reading “Bio Roundup: Termeer Tribute, ASCO Peeks, Califf’s New Gigs & More”

Stifling Immigration Is “Retrograde”: Highlights From “What’s Hot”

Drug pricing. The opioid epidemic. Boardroom diversity. Immigration crackdowns. The state of cancer care now, and in the future. These things aren’t just topics of conversation in Boston biotech circles, but nationwide. Connecting those two with some lively, candid discussions was the idea behind Xconomy’s “What’s Hot in Boston Biotech” event last week. Today we’re … Continue reading “Stifling Immigration Is “Retrograde”: Highlights From “What’s Hot””

Riding Recent Biotech IPO Wave, G1 Snags $105M to Fund Cancer Work

Cancer drugmaker G1 Therapeutics raised $105 million in an IPO last night, the latest development in a recent uptick of biotech offerings. G1, of Research Triangle Park, NC, sold 7 million shares at $15 apiece. G1 had to sell more shares than it had intended (6.25 million) to hit its goal, but still priced within … Continue reading “Riding Recent Biotech IPO Wave, G1 Snags $105M to Fund Cancer Work”

With Latest Deal, Biogen Bets $120M on Remedy Pharma Stroke Drug

Biogen has acquired an experimental drug for a type of deadly stroke, adding to the belief that the company, under new CEO Michel Vounatsos, will be more aggressive in pursuing deals to stock its pipeline. Biogen (NASDAQ: [[ticker:BIIB]]) is buying glyburide (CIRARA) from New York-based Remedy Pharmaceuticals for $120 million in cash plus potential future … Continue reading “With Latest Deal, Biogen Bets $120M on Remedy Pharma Stroke Drug”

Ionis Touts Phase 3 Win, But Safety Worries Cloud Nerve Drug Study

Two RNA-based drugs for the same disease, a rare, crippling nerve condition called familial amyloid polyneuropathy (FAP) with no available treatments in the U.S., could soon be under an FDA review at the same time. The first of those treatments, from Carlsbad, CA, Ionis Pharmaceuticals, is on its way there—but are the data are good … Continue reading “Ionis Touts Phase 3 Win, But Safety Worries Cloud Nerve Drug Study”

Bio Leaders Mourn Passing of Genzyme Exec, Orphan Drug Innovator Termeer

Henri Termeer, a biotechnology pioneer and the longtime CEO of Genzyme, has passed away at the age of 71. According to the Boston Globe, Termeer collapsed in his home in Marblehead, MA, on Friday night. Termeer (pictured) was an important figure in the development of biotech not just in the Boston community, but worldwide as … Continue reading “Bio Leaders Mourn Passing of Genzyme Exec, Orphan Drug Innovator Termeer”

Stalled or Rising Momentum? New York Wrestles With Its Biotech Future

[Updated, 6:25 a.m. ET, see below] New York City and state governments have finally put big dollars behind local development of the life sciences industry. But lots of cash is no guarantee of success, as panelists kicking off the annual NewYorkBio conference made clear Tuesday. “The question is, where are we as an industry, and … Continue reading “Stalled or Rising Momentum? New York Wrestles With Its Biotech Future”

With $145M Buyout, River Vision’s Plan For Stalled Roche Drug Pays Off

A bid by a group of investors to transform a stalled cancer drug into an eye disease treatment has paid off. Horizon Pharma this morning has acquired River Vision Development Corp., a New York entity formed to develop a therapy for an eye condition called Graves’ ophthalmopathy. Horizon (NASDAQ: [[ticker:HZNP]]), an Ireland based firm, is  … Continue reading “With $145M Buyout, River Vision’s Plan For Stalled Roche Drug Pays Off”

Bio Roundup: NIH Precision Boost, AHCA to Senate, New Drugs & More

Two dramatic healthcare stories played out this week. First, to the jubilation of the nation’s science community, Congress proposed to boost National Institutes of Health funding, not slash it as the Trump administration had outlined. Then the patchwork revival of the GOP’s healthcare plan, despite being panned by nearly every major medical group in the … Continue reading “Bio Roundup: NIH Precision Boost, AHCA to Senate, New Drugs & More”

With $75M Haul, Ovid Continues Flurry of Biotech IPOs

The recent biotech IPO surge continues. Ovid Therapeutics raised $75 million in an IPO late Thursday, becoming the fifth life sciences company to hit the Nasdaq in the past two weeks. Ovid (NASDAQ: [[ticker:OVID]]), a New York company developing drugs for rare brain diseases, sold 5 million shares at $15 apiece. Those numbers come in … Continue reading “With $75M Haul, Ovid Continues Flurry of Biotech IPOs”

Future At Stake, Zafgen Gives First Glimpse of New Obesity Drug Data

Zafgen took a big hit last year when two patient deaths forced it to abandon its most advanced drug, belaronib. The company is now looking to a new experimental weight loss drug for diabetics to turn its fortunes around, with the first glimpse at early data released this afternoon. Many caveats apply. The data are … Continue reading “Future At Stake, Zafgen Gives First Glimpse of New Obesity Drug Data”

Pieris Adds Another Partner, Gets $45M in AstraZeneca Lung Drug Deal

A protein drug developer with roots in Germany continues to grow in Boston, MA, with the help of another partnership. Pieris Pharmaceuticals today cut a deal with AstraZeneca to develop a group of drugs for respiratory diseases, adding a new name to an already wide list of pharmaceutical partners. Pieris (NASDAQ: [[ticker:PIRS]]) gets $45 million … Continue reading “Pieris Adds Another Partner, Gets $45M in AstraZeneca Lung Drug Deal”

FDA Wants More Safety Info on Psych Drug, Intra-Cellular Shares Drop

Despite mixed data from clinical trials, Intra-Cellular Therapies plans to seek FDA approval for an experimental schizophrenia drug, its only therapy even close to market. But that plan got more difficult this morning as the company revealed the FDA has some questions about the drug’s safety, causing shares to slide to their lowest levels since … Continue reading “FDA Wants More Safety Info on Psych Drug, Intra-Cellular Shares Drop”

Entrepreneurs, VCs, Bio Builders Talk Seizing Momentum in NY on May 31

It’s going to take more than just a government initiative for New York to forge an identity as a top commercial hub for life sciences. It’s going to take great ideas, entrepreneurs taking risks, developers making space their companies can grow in, and investors gambling on their success. Some of these things are already happening, … Continue reading “Entrepreneurs, VCs, Bio Builders Talk Seizing Momentum in NY on May 31”

Radius Nabs FDA OK For New Bone Drug With Rival Amgen Close Behind

At least one new treatment is on the way for osteoperosis patients, and possibly two before the end of the summer. Radius Health on Friday won FDA approval of an osteoperosis drug that, like a rival treatment from Eli Lilly, can help build up the strength of bones rather than just prevent them from becoming … Continue reading “Radius Nabs FDA OK For New Bone Drug With Rival Amgen Close Behind”

After Sarepta’s Surprising FDA Nod, CEO Kaye Plans to Resign

Ed Kaye was the chief medical officer of Sarepta Therapeutics for nearly six years before being thrust into the spotlight. In April 2015, CEO Chris Garabedian resigned, and Kaye took up the job of trying to notch the first-ever approval for a Duchenne muscular dystrophy drug, eteplirsen (Exondys 51)—with the slimmest of data, no less. … Continue reading “After Sarepta’s Surprising FDA Nod, CEO Kaye Plans to Resign”

Sarepta CEO Kaye Announces Plans to Resign

Sarepta Therapeutics (NASDAQ: [[ticker:SRPT]]) president and CEO Ed Kaye will resign on Sept. 20 “or some other future date” and will stay on as a board member and advisor afterwards, according to a regulatory filing. Kaye, Sarepta’s former chief medical officer, stepped in when ex-CEO Chris Garabedian resigned in 2015 and steered the company towards … Continue reading “Sarepta CEO Kaye Announces Plans to Resign”

Duchenne, SMA, and the Rise of Patient Power at “What’s Hot” on May 11

Two of the most noteworthy drugs to win FDA approval over the past year, eteplirsen (Exondys 51) and nusinersen (Spinraza), have something important in common: Patient groups have played a critical role in both of their stories. Eteplirsen, Sarepta Therapeutics’ (NASDAQ: [[ticker:SRPT]]) Duchenne muscular dystrophy drug, was a test case for the increasing power of … Continue reading “Duchenne, SMA, and the Rise of Patient Power at “What’s Hot” on May 11”

Will New Data Open “Bottlenecks” For Biogen’s Pricey Spine Drug?

Four months after its drug nusinersen (Spinraza) became the first ever approved to treat the rare genetic disease spinal muscular atrophy, Biogen (NASDAQ: [[ticker:BIIB]]) released study results Tuesday that could help more patients gain access to the expensive drug. The data, from a 126-patient study called CHERISH, provide the most detailed evidence to date that … Continue reading “Will New Data Open “Bottlenecks” For Biogen’s Pricey Spine Drug?”

The Medicines Co., Alnylam Take Plunge, Outline Big Test For Heart Drug

The Medicines Co. and Alnylam Pharmaceuticals have come to a deal with the FDA on the big Phase 3 test for their RNA-based cholesterol-lowering drug, inclisiran, a potentially longer-lasting alternative to a new group of drugs called PCSK9 inhibitors. Medicines Co. (NASDAQ: [[ticker:MDCO]]) and partner Alnylam (NASDAQ: [[ticker:ALNY]]) will collectively enroll 3,000 patients with atherosclerotic … Continue reading “The Medicines Co., Alnylam Take Plunge, Outline Big Test For Heart Drug”

Bio Roundup: Spinraza Woes, Science March, Baseline Launch & More

Rumblings of a new Obamacare replacement surfaced this week, while scientists and their supporters prepared for Saturday’s—a.k.a. Earth Day’s—nationwide Marches for Science. Our own reporting focused this week on the fallout from high drug prices, with a look at the frustration of families whose children have had trouble gaining access to the first drug ever … Continue reading “Bio Roundup: Spinraza Woes, Science March, Baseline Launch & More”

UniQure to Yank Pioneering Gene Therapy From Market in Europe

[Updated, 9:33 a.m. ET, see below] It took decades to get the first gene therapy in the Western world to market. And it will apparently take only five years since for that product, a treatment from UniQure for a rare metabolic disorder, to fizzle out after failing commercially. UniQure (NASDAQ: [[ticker:QURE]]), with operations in Amsterdam … Continue reading “UniQure to Yank Pioneering Gene Therapy From Market in Europe”

“Our Son’s Fate”: Parents Fighting for Kids’ Spine Drug Eye New Data

Melissa Marotta pulled no punches to get her three-year-old son C.J., who has the genetic disease spinal muscular atrophy, access to nusinersen (Spinraza), the first approved drug for his condition. For months, she couldn’t schedule a treatment date at Columbia University Medical Center/New York-Presbyterian Morgan Stanley Children’s Hospital, and for much of that time, she … Continue reading ““Our Son’s Fate”: Parents Fighting for Kids’ Spine Drug Eye New Data”

Ex-Dendreon CEO, Gold, Returns to Wall Street as Alpine, Nivalis Merge

Mitch Gold, the controversial executive who oversaw the rise of now-defunct cancer immunotherapy pioneer Dendreon, is about to head back to Wall Street. Gold’s latest startup, Seattle cancer and autoimmune drug developer Alpine Immune Sciences, has struck a deal to go public by reverse merging with struggling Nivalis Therapeutics (NASDAQ: [[ticker:NVLS]]). Nivalis, of Boulder, CO, … Continue reading “Ex-Dendreon CEO, Gold, Returns to Wall Street as Alpine, Nivalis Merge”

Bio Roundup: CRISPR Appeal, Bristol Deals, Stock Pumping & More

It may have been a short holiday week, but there was plenty of news on the CRISPR front. In the ongoing battle for rights to the landmark gene editing technology, the faction led by the University of California appealed the U.S. decision in favor of the Broad Institute. Elsewhere, Bristol-Myers Squibb flipped assets, a top … Continue reading “Bio Roundup: CRISPR Appeal, Bristol Deals, Stock Pumping & More”

On Tap For “What’s Hot” on May 11: Opioid Crisis, Immigration & More

We’re just a month away from Xconomy’s latest biotech bash in Boston, so it’s time to dish some details on the program. For “What’s Hot in Boston Biotech” on May 11 at Biogen’s headquarters in Cambridge, MA, we’re focusing on the big issues in life sciences—both in Boston and across the country. The opioid epidemic, … Continue reading “On Tap For “What’s Hot” on May 11: Opioid Crisis, Immigration & More”

Bristol Flips Remnants of Adnexus, iPierian to Roche, Biogen For $470M

Priorities change over time for drugmakers, as evidenced today by Bristol-Myers Squibb. The company announced two separate deals for experimental drugs that no longer fit its strategy—both of which were remnants of old acquisitions and a past plan to zero in on genetic diseases. In one deal, Bristol (NYSE: [[ticker:BMY]]) has sent Biogen (NASDAQ:[[ticker:BIIB]]), of … Continue reading “Bristol Flips Remnants of Adnexus, iPierian to Roche, Biogen For $470M”

With Ovid IPO, Ex-Teva CEO Levin Pitches Neuro Drug Plan to Wall Street

Ex-Teva Pharmaceutical and Bristol-Myers Squibb executive Jeremy Levin is once again on the verge of running a publicly traded company—this time, a small startup biotech. Startup Ovid Therapeutics, which Levin formed in New York a few years ago to develop treatments for rare brain diseases, has just filed paperwork outlining an IPO. The move isn’t … Continue reading “With Ovid IPO, Ex-Teva CEO Levin Pitches Neuro Drug Plan to Wall Street”

Frequency Nabs $32M, Will Push Hearing-Loss Drugs To Human Studies

Hearing loss, which affects millions of Americans with no effective drugs to fight it, has become a ripe field for biotech investment. The latest example is Frequency Therapeutics, which sprung from MIT earlier this year and has just corralled a big round of funding. The Woburn, MA-based startup has raised a $32 million Series A … Continue reading “Frequency Nabs $32M, Will Push Hearing-Loss Drugs To Human Studies”

OncoMed Shares Sink as One Drug Fails, Bayer Kicks Back Two Others

OncoMed Pharmaceuticals got a double dose of bad news on Monday. For the second time, one of its stem cell drugs has failed to help pancreatic cancer patients. And what’s more, partner Bayer has passed on options to two of its other cancer drugs. Redwood City, CA-based OncoMed (NASDAQ: [[ticker:OMED]]) said this morning that demcizumab, … Continue reading “OncoMed Shares Sink as One Drug Fails, Bayer Kicks Back Two Others”

With Fresh Pneumonia Data, Paratek To Seek 2018 Antibiotic Approval

The need for new antibiotics to battle drug-resistant bacteria is rising, and Paratek Pharmaceuticals has news that could give doctors a new weapon. Boston-based Paratek (NASDAQ: [[ticker:PRTK]]) reports that its antibiotic, omadacycline, has hit all its main and secondary goals in a study of 774 patients with hospital-acquired pneumonia. It is the second of two … Continue reading “With Fresh Pneumonia Data, Paratek To Seek 2018 Antibiotic Approval”

Cydan, NEA’s Orphan Drug Experiment, Pays Off in $200M Vtesse Deal

Four years into its existence, Cydan Development, New Enterprise Associates’ orphan drug startup accelerator, has notched its first big win. Vtesse, the first company to emerge from Cydan, has been sold in a deal valued at $200 million. Sucampo Pharmaceuticals (NASDAQ: [[ticker:SCMP]]), a Rockville, MD-based maker of a drug to treat various bowel conditions, will … Continue reading “Cydan, NEA’s Orphan Drug Experiment, Pays Off in $200M Vtesse Deal”

To Infinity and Beyond: Julian Adams on Missteps and His New Job

Julian Adams left Boston pharma Millennium Pharmaceuticals in 2003 having accomplished a rare feat. A medicinal chemist by training, Adams helped shepherd along a drug known as bortezomib (Velcade) that was the first-ever approved inhibitor of the proteasome, the cell’s garbage disposal unit. It was an approach most people thought would never work. “Everybody was … Continue reading “To Infinity and Beyond: Julian Adams on Missteps and His New Job”

Bio Roundup: New Drug Bonanza, Vertex’s Triple Play, CAR-T Race & More

As far as singular weeks go for biotech, this was a big one. Three highly anticipated drugs were approved. One is the first injectable medication for eczema. Another might help fend off the recurrence of ovarian cancer. And a third offers hope for people with severe forms of multiple sclerosis. Another drug, a new combination … Continue reading “Bio Roundup: New Drug Bonanza, Vertex’s Triple Play, CAR-T Race & More”

Next Stop on Father’s Duchenne Quest: Wall Street? Solid Raises $50M

It wasn’t too long ago that Ilan Ganot was a J.P. Morgan banker. Then his son, Eytani, was diagnosed with Duchenne muscular dystrophy, and he ended up the head of a startup trying to find an array of treatments for the progressive, deadly genetic disease. Now it appears that journey might return Ganot to a … Continue reading “Next Stop on Father’s Duchenne Quest: Wall Street? Solid Raises $50M”

With New Data, Vertex Touts Backbone Of Future Cystic Fibrosis Drugs

With new data released this evening, Vertex Pharmaceuticals is closer to bringing its third cystic fibrosis treatment to market, which would mean the company’s product line could potentially treat upwards of 40 percent of the roughly 70,000 people with the disease worldwide. The late-stage data for the combination of ivacaftor, already approved as Kalydeco, and … Continue reading “With New Data, Vertex Touts Backbone Of Future Cystic Fibrosis Drugs”

Bio Roundup: Trumpcare Wavers, Amgen Courts Payers, Critics Circle PTC

Who knew healthcare could be so complicated? This week, President Trump and the GOP saw first hand, as a feverish rally to get their healthcare bill through the House of Representatives on Thursday stalled before a vote. Elsewhere in Washington DC, drug pricing continued to be a political football. Two well known congressional drug pricing … Continue reading “Bio Roundup: Trumpcare Wavers, Amgen Courts Payers, Critics Circle PTC”

Sanders, Cummings to PTC: Price Duchenne Steroid at Import Cost

[Updated, 5:30 pm E.T., see below] PTC Therapeutics inherited a drug pricing controversy last week when it paid $140 million for deflazacort (Emflaza), a steroid used to treat Duchenne muscular dystrophy. So it’s no surprise that just six days after the deal, congressional drug price hawks Bernie Sanders and Elijah Cummings have come calling. Sen. … Continue reading “Sanders, Cummings to PTC: Price Duchenne Steroid at Import Cost”

After a Clinical Trial Death, FDA OKs New Tests of Akashi’s Duchenne Drug

Akashi Therapeutics has been cleared by the FDA to resume testing an experimental Duchenne muscular dystrophy drug roughly a year after a patient taking the treatment died in a clinical trial. Now the question is whether the Cambridge, MA, company can get the funding to support a new study. Akashi plans to start a new … Continue reading “After a Clinical Trial Death, FDA OKs New Tests of Akashi’s Duchenne Drug”

Join Varmus, Foley & More at NY Biotech Seizes the Momentum on May 31

It’s not an understatement to say 2017 is a crucial year for the growth of the life sciences industry in New York. This past year, city and state governments agreed to put $1.15 billion into the sector in the Empire State. It was part of a sprawling plan to bring lab space online, nurture local … Continue reading “Join Varmus, Foley & More at NY Biotech Seizes the Momentum on May 31”

Waving White Flag, Cerulean Merges With Dare Bio, Sells Cancer Drugs

Cerulean Pharma may have been able to survive one brush with death, but the second has proven fatal. Today, the Waltham, MA, company announced plans to merge with a privately held women’s health company, Dare Bioscience, and sell off, for a pittance, the cancer drugs that were once its prized assets. Under the terms of … Continue reading “Waving White Flag, Cerulean Merges With Dare Bio, Sells Cancer Drugs”

First Trial Underway, CytomX Gets $200M More In Expanded Bristol Pact

It’s tough to stand out in the cancer immunotherapy crowd, where a slew of companies are testing a variety of drug combinations and new methods to help boost responses to existing treatments. But CytomX Therapeutics has clearly gotten the attention of one of the field’s major players, Bristol-Myers Squibb, which has just re-upped and expanded … Continue reading “First Trial Underway, CytomX Gets $200M More In Expanded Bristol Pact”

With New Data, Amgen Promises Refund If Cholesterol Drug Doesn’t Work

Stung by slow sales of its next-generation anti-cholesterol drug, Amgen hopes new clinical data, released this morning, will spur doctors to boost prescriptions and—perhaps more important—drive insurers to loosen their restrictions. The data, from a massive study of more than 27,000 patients, come at a time when drug and healthcare prices are a top U.S. … Continue reading “With New Data, Amgen Promises Refund If Cholesterol Drug Doesn’t Work”

First Trial in Sight, Editas Cuts $90M Eye Drug Deal With Allergan

Slowly but surely, CRISPR-Cas9 gene editing treatments are working their way towards human clinical testing, spurring more large pharmaceutical companies to get in on their future. The latest today is Allergan, which has just aligned itself with Editas Medicine on treatments for a group of genetic eye diseases. Allergan (NYSE: [[ticker:AGN]]) will pay Cambridge, MA-based … Continue reading “First Trial in Sight, Editas Cuts $90M Eye Drug Deal With Allergan”

Scott Gottlieb On Tap to Lead FDA, and Biopharma Exhales

[Updated 3/12/17 with more comments, see below] The Trump administration has nominated Scott Gottlieb to be the new commissioner of the FDA, a selection that had the life sciences industry breathing a sigh of relief Friday afternoon. The 44-year-old Gottlieb is a physician, a venture partner at New Enterprise Associates, and was also the deputy … Continue reading “Scott Gottlieb On Tap to Lead FDA, and Biopharma Exhales”

Biotech Roundup: Obamacare Lite, DePinho Out, Dunsire In & More

We’ll start the roundup in Washington DC. The repeal of the Affordable Care Act, also known as Obamacare, began on the first day of the Trump administration. This week, the “replace” phase began with a Republican plan championed by House Speaker Paul Ryan (R-WI). President Trump backed it, too, but a lot of Republicans have … Continue reading “Biotech Roundup: Obamacare Lite, DePinho Out, Dunsire In & More”