RNAi Hits Europe as EMA Follows FDA, Approves Alnylam Drug

The first medicine that uses RNA interference, a method cells employ to mute a gene before it can make a harmful protein, has made its way to Europe. Three weeks after the FDA approved patisiran (Onpattro), from Alnylam Pharmaceuticeuticals (NASDAQ: [[ticker:ALNY]]), for a rare and deadly disease called hereditary transthyretin amyloidosis (hATTR), the European Medicines … Continue reading “RNAi Hits Europe as EMA Follows FDA, Approves Alnylam Drug”

Bristol Nabs Immunotherapy’s First FDA Nod for Small Cell Lung Cancer

Over the past few years, immunotherapy has rapidly begun to change how lung cancer is treated. But it hasn’t been an option for patients with a particularly aggressive form of the disease, small cell lung cancer (SCLC), until today. The FDA on Friday approved Bristol-Myers Squibb’s (NYSE: [[ticker:BMY]]) immunotherapy, nivolumab (Opdivo), for SCLC patients who … Continue reading “Bristol Nabs Immunotherapy’s First FDA Nod for Small Cell Lung Cancer”

With BioNTech Deal, Pfizer Becomes Latest to Bet on mRNA Vaccines

Pfizer has become the latest drug maker to bet that synthetic messenger RNA molecules (mRNA) can be a new source for vaccines. This morning, the New York pharma company cut a deal with BioNTech, of Germany, to co-develop mRNA vaccines for flu prevention. BioNTech will run the initial human tests on these treatments, then hand … Continue reading “With BioNTech Deal, Pfizer Becomes Latest to Bet on mRNA Vaccines”

Another Gilead Exec Exits as CMO Cheng Steps Down

Another top-level Gilead Sciences (NASDAQ: [[ticker:GILD]]) executive is stepping down. Andrew Cheng, a 19-year Gilead vet who was named chief medical officer of the Foster City, CA, company just four months ago, is leaving to “pursue another opportunity.” Former research chief Norbert Bischofberger left Gilead in March to run a biotech startup, Kronos Bio. And … Continue reading “Another Gilead Exec Exits as CMO Cheng Steps Down”

Acorda CTO Batycky Leaves to Run Biotech Startup

[Corrected 8/14/18, 9:45 am. See below.] Acorda Therapeutics (NASDAQ: [[ticker:ACOR]]) chief technology officer Rick Batycky is leaving the company to become the CEO of a private, venture-backed biotech. Batycky’s duties are being taken up by Acorda chief of business operations David Lawrence and chief medical officer Burkhard Blank, the company said. [Corrected title in headline. … Continue reading “Acorda CTO Batycky Leaves to Run Biotech Startup”

Fresh Off GSK Deal, Gene Therapy Startup Orchard Bags Another $150M

Another gene therapy startup looks primed for an IPO. Orchard Therapeutics has just landed another $150 million to help drive forward a group of gene therapies it bought from British pharma company GlaxoSmithKline earlier this year. The Series C funding round from Deerfield Management, RA Capital Management, Venrock, and others, includes a number of “crossover” … Continue reading “Fresh Off GSK Deal, Gene Therapy Startup Orchard Bags Another $150M”

Amicus Finally Wins FDA Nod, Will Price Fabry Drug Below Rivals

[Updated, 8/13/18, see below] The FDA has approved a new treatment for Fabry disease, a decision that represents the end of a winding, more than decade-long saga for the drug’s developer, Amicus Therapeutics. Its long journey also highlights how drug approval in the U.S. has changed just in the last two years, thanks to new … Continue reading “Amicus Finally Wins FDA Nod, Will Price Fabry Drug Below Rivals”

Alnylam Gets Landmark FDA OK For First-Ever RNAi Drug

[Updated at 4:08 pm ET, see below] Alnylam Pharmaceuticals made history this afternoon. The FDA has just approved Alnylam’s patisiran (Onpattro), making it the first medicine specifically cleared to treat a rare and deadly disease called hereditary transthyretin amyloidosis (hATTR). The decision is a scientific milestone too: it marks the first-ever approval for a medicine … Continue reading “Alnylam Gets Landmark FDA OK For First-Ever RNAi Drug”

Bio Roundup: Medicare Drug Prices, FDA Moves, Gene Therapy News & More

Two imminent FDA rulings on two separate drugs aren’t just noteworthy for the patients they’ll help, but the long and winding road their developers have taken to get to this point. First is Alnylam Pharmaceuticals, which has spent 16 years and over $2 billion to try to bring an unproven form of medicine, RNA interference … Continue reading “Bio Roundup: Medicare Drug Prices, FDA Moves, Gene Therapy News & More”

With Big Takeda Deal at Launch, Can Ambys Keep Control of Its Future?

A new biotech has debuted today aiming to make a variety of treatments for deadly liver diseases. The startup, Ambys Medicines, has formed an unusual, broad alliance with Takeda. Some early, wide-ranging partnerships between new biotechs and Big Pharma have fallen apart in the past and have been too limiting for the biotech, but Ambys’s … Continue reading “With Big Takeda Deal at Launch, Can Ambys Keep Control of Its Future?”

New Faces, Big Impact: Meet the Finalists For Xconomy’s Newcomer Award

Being a newcomer can mean a few different things: being new to the area (or newly back after being away) or new to an industry. The finalists for Xconomy’s Newcomer Award this year cover these definitions. One is a serial biotech entrepreneur who has now made an impact in multiple cities, most recently Boston. Another … Continue reading “New Faces, Big Impact: Meet the Finalists For Xconomy’s Newcomer Award”

Bluebird, Regeneron Team Up To Make Cell Therapies For Cancer

Regeneron Pharmaceuticals and Bluebird Bio have struck a wide-ranging partnership to broaden the reach of their respective cancer immunotherapy work, inking a deal to develop multiple cell therapies together. Regeneron (NASDAQ: [[ticker:REGN]]) develops antibody drugs, while Bluebird (NASDAQ: [[ticker:BLUE]]) is a gene and cell therapy specialist. In the deal, the two companies will work together … Continue reading “Bluebird, Regeneron Team Up To Make Cell Therapies For Cancer”

Patients Have “Fingers Crossed” As Alnylam Awaits Historic FDA Decision

[Updated, 8/6/18, see below] By next weekend, the FDA could for the first time approve a medicine that uses a biological trick that was only discovered two decades ago: RNA interference, which silences a gene before it can make a harmful protein. That medicine is patisiran (Onpattro), from Alnylam Pharmaceuticals (NASDAQ: [[ticker:ALNY]]). An approval wouldn’t … Continue reading “Patients Have “Fingers Crossed” As Alnylam Awaits Historic FDA Decision”

Going Against the Grain: Meet the Xconomy Contrarian Award Finalists

Our profiles of the finalists for this year’s Xconomy Awards roll on today with a look at the nominees for the Contrarian award. These finalists have been nominated for different reasons. One, for instance, may have paved the way for gene therapies to be commercially adopted in the U.S., by being the first insurer to … Continue reading “Going Against the Grain: Meet the Xconomy Contrarian Award Finalists”

Bio Roundup: An Alzheimer’s Head-Scratcher, OUTBio, GSK & Gilead Shakeups  

The devil is in the details, and key clinical trial results made that abundantly clear this week. Eisai and partner Biogen released the highly anticipated details of an Alzheimer’s disease study they had already deemed positive, after an initial failure. In one sense, the study was the success they have claimed. A high dose of … Continue reading “Bio Roundup: An Alzheimer’s Head-Scratcher, OUTBio, GSK & Gilead Shakeups  “

OUTBio, a Biopharma LGBTQ Group, Grows Fast & Ponders Its Future

In June, Jennifer Petter, the founder and chief scientific officer of biotech startup Arrakis Therapeutics, got an e-mail from a stranger. His name was Ramsey Johnson, a biotech veteran who works in clinical operations at Boston startup Phoenix Tissue Repair. Johnson was writing about OUTBio, a networking organization for LGBTQ members of the biopharma community. … Continue reading “OUTBio, a Biopharma LGBTQ Group, Grows Fast & Ponders Its Future”

Sangamo Bags TxCell, and a Next-Gen Cell Therapy Tech, for $84M

Sangamo Biosciences has acquired French biotech TxCell, adding an experimental technology with the potential to treat autoimmune diseases with cell therapy. Sangamo (NASDAQ: [[ticker:SGMO]]), of Richmond, CA, will pay €72 million ($84.31 million), or $2.58 per share, for TxCell. It expects to complete the deal later this year. The deal gives Sangamo a technology that … Continue reading “Sangamo Bags TxCell, and a Next-Gen Cell Therapy Tech, for $84M”

Bio Roundup: Drug Prices, CRISPR Caveats, Rubius IPO Pop & More

After nearly two years of all smoke, no fire around President Trump’s promise to curtail drug prices, there’s finally some glimmer of action. Pfizer said last week it would halt its price hikes, and Novartis and Merck took similar actions this week. But we still don’t know the details of the administration’s drug-pricing policy, if … Continue reading “Bio Roundup: Drug Prices, CRISPR Caveats, Rubius IPO Pop & More”

Zogenix Epilepsy Data Sets Up Market Battle With GW’s Cannabis Drug

Just weeks ago, the FDA approved the first ever medicine made from a derivative of marijuana, a drug from GW Pharmaceuticals for a rare form of epilepsy. Yet competition already looms from Zogenix, thanks to new data the Emeryville, CA, company reports this morning. Zogenix (NASDAQ: [[ticker:ZGNX]]) said that its experimental drug ZX008 succeeded in … Continue reading “Zogenix Epilepsy Data Sets Up Market Battle With GW’s Cannabis Drug”

The Art of Perseverance: Meet Xconomy’s Six CEO Award Finalists

The Xconomy Awards are back, and today we’re kicking things off by taking a look at our first set of finalists—the top Boston biotech CEOs nominated by you, the readers. We’ve gone through all the nominations and come up with six finalists. Some of these CEOs have withstood years of ups and downs to take … Continue reading “The Art of Perseverance: Meet Xconomy’s Six CEO Award Finalists”

Bio Roundup: Big Bucks For Calico, Data Dumps & The IPO Wave Rolls On

If you’re running a privately held biotech, the time to go public is now. Clinical data already in hand or not, biotechs have charged to Wall Street at a record pace over the past two weeks, raising more than $1 billion in offerings that all met or exceeded their projected valuations. And there are no … Continue reading “Bio Roundup: Big Bucks For Calico, Data Dumps & The IPO Wave Rolls On”

Akebia Merges With Keryx as Anemia Drug Race Nears Finish Line

The race to treat anemia with a pill, not an injectable biologic, just took a new turn. Akebia Therapeutics (NASDAQ: [[ticker:AKBA]]), which is battling with FibroGen (NASDAQ: [[ticker:FGEN]]) to bring an anemia pill to market, has just announced plans to merge with kidney drug disease developer Keryx Pharmaceuticals (NASDAQ: [[ticker:KERX]]) in an all-stock deal that … Continue reading “Akebia Merges With Keryx as Anemia Drug Race Nears Finish Line”

AstraZeneca’s PARP Drug Is First to Help New Ovarian Cancer Patients

Over the past few years, a new class of drugs known as “PARP” inhibitors has begun to change how ovarian cancer is treated. Data released by AstraZeneca and partner Merck this morning, from a Phase 3 study of the drug olaparib (Lynparza), could continue the trend. AstraZeneca (NYSE: [[ticker:AZN]]) and Merck (NYSE: [[ticker:MRK]]) said that … Continue reading “AstraZeneca’s PARP Drug Is First to Help New Ovarian Cancer Patients”

Don’t Be Boston 2.0, And Photos From “Bringing Back the Expats”

Be your own biotech cluster, not a Boston redux. Accelerate the momentum that’s already begun, don’t let it fizzle. And above all, be fearless entrepreneurs. You’ll need to be if New York biotech will ever become what its champions hope. Those were just a few lessons ex-New Yorker biopharma veterans had for the nascent life … Continue reading “Don’t Be Boston 2.0, And Photos From “Bringing Back the Expats””

With Roche Data, Cancer Combos Post First Win Vs. Small Cell Lung Cancer

Cancer immunotherapy combinations continue to chalk up wins against lung cancer. For the first time, an immunotherapy-chemotherapy combination has succeeded in a late-stage study of patients with small cell lung cancer (SCLC), a particularly aggressive form of the disease often linked to smoking. Roche’s South San Francisco, CA-based Genentech division said today that in a … Continue reading “With Roche Data, Cancer Combos Post First Win Vs. Small Cell Lung Cancer”

In a Genentech Redux, Roche Pays $2.4B for Rest of Foundation Medicine

Roche this morning agreed to acquire the part of Foundation Medicine it didn’t already own, another big vote of confidence in tests that analyze a patient’s cancer genes and the drugs that can target them. Roche will pay $137 per share, or $2.4 billion, for the remaining stock of Foundation (NASDAQ: [[ticker:FMI]]), of Cambridge, MA. … Continue reading “In a Genentech Redux, Roche Pays $2.4B for Rest of Foundation Medicine”

Sarepta’s Data Are First in Rush for Duchenne Gene Therapy

It’s far too early to say whether gene therapy can treat Duchenne muscular dystrophy, a deadly genetic disease. But the first clinical data have arrived, with Sarepta Therapeutics today reporting encouraging preliminary results from three patients. Sarepta (NASDAQ: [[ticker:SRPT]]), of Cambridge, MA, is conducting an early-stage study of its experimental treatment known as AAVrh74.MHCK7.micro-Dystrophin. The … Continue reading “Sarepta’s Data Are First in Rush for Duchenne Gene Therapy”

As Sarepta Preps Data, FDA Lifts Hold on Solid’s Duchenne Gene Therapy

The race to use gene therapy to treat Duchenne muscular dystrophy, a progressive, deadly genetic disease that affects boys, is heating up this week. The FDA just cleared Solid Biosciences to resume clinical testing of a Duchenne gene therapy, a day before Sarepta Therapeutics will report human clinical data from a rival treatment that also … Continue reading “As Sarepta Preps Data, FDA Lifts Hold on Solid’s Duchenne Gene Therapy”

Bio Roundup: Not-BIO Party Foul, CRISPR Drama, Sickle Cell Updates

This week brought a sobering reminder of how far away we are from true gender equality in the life sciences. An industry party held during BIO’s annual meeting in Boston last week featured scantily clad women with company logos painted on their bodies—two years after the infamous party at the J.P. Morgan party in San … Continue reading “Bio Roundup: Not-BIO Party Foul, CRISPR Drama, Sickle Cell Updates”

Bluebird’s Upgraded Gene Therapy Shows Promise For Sickle Cell Patients

Bluebird Bio has new clinical data this morning that, while early and only from a handful of patients, are the most significant evidence to date that gene therapy might help people with sickle cell disease, a genetic disorder without an effective treatment that afflicts millions around the world. The data, presented at the European Hematology … Continue reading “Bluebird’s Upgraded Gene Therapy Shows Promise For Sickle Cell Patients”

Westphal’s Flex Pharma To Cut Jobs and Seek Sale After Clinical Flop

Flex Pharma, a company formed four years ago by serial biotech executive Christoph Westphal to develop treatments for muscle cramps, will layoff more than half of its staff and look to sell itself after suffering another setback in clinical testing. Boston-based Flex (NASDAQ: [[ticker:FLKS]]) said it will halt two Phase 2 trials of experimental FLX-787 … Continue reading “Westphal’s Flex Pharma To Cut Jobs and Seek Sale After Clinical Flop”

Sage Speeds Ahead With Plans For a Short-Course Depression Drug

Sage Therapeutics has announced a potential fast path to approval for an experimental depression drug—as well as plans to treat patients with it for just two weeks until they feel relief, not chronically as with other treatments for the disease. After meeting with the FDA, Cambridge, MA-based Sage (NASDAQ: [[ticker:SAGE]]) said it will only have … Continue reading “Sage Speeds Ahead With Plans For a Short-Course Depression Drug”

With $45M Translate Bio Deal, Sanofi Bets Again on mRNA Vaccines

It hasn’t been proven yet that synthetic messenger RNA molecules (mRNA) can be effective medicines. But their promise as a source for new vaccines, primarily for cancer, has enticed multiple pharmaceutical companies. The latest evidence comes from Sanofi, which just cut its second mRNA vaccine deal to date, this time with Translate Bio. Sanofi Pasteur, … Continue reading “With $45M Translate Bio Deal, Sanofi Bets Again on mRNA Vaccines”

Vertex CF Competitor Proteostasis Falters, Galapagos Up Next

Data released Wednesday afternoon looks to have eased a potential threat to Vertex Pharmaceuticals’ burgeoning cystic fibrosis franchise, though news from another challenger looms later this month. Proteostasis Therapeutics (NASDAQ: [[ticker:PTI]]) released data from a 49-patient Phase 1 study testing its experimental cystic fibrosis drug, PTI-801, in combination with Vertex’s (NASDAQ: [[ticker:VRTX]]) FDA-approved CF drug … Continue reading “Vertex CF Competitor Proteostasis Falters, Galapagos Up Next”

ASCO Wrap: Cancer Combos, Precision Meds, Stock Movers & More

The American Society of Clinical Oncology annual meeting, the world’s largest cancer conference, wraps up today in Chicago. Investors have dissected the data, picked winners and losers, and sent some biotech stocks soaring and others tumbling. In the days before ASCO, Xconomy previewed two major themes: the feverish and flawed race to develop cancer immunotherapy … Continue reading “ASCO Wrap: Cancer Combos, Precision Meds, Stock Movers & More”

Loxo Unveils More Data, Paves a Path Forward In Precision Oncology

An experimental cancer drug from Loxo Oncology has shown early signs that it might impact multiple tumor types, regardless of where in the body they originated. The clinical results come with caveats, but they add to a slowly growing body of evidence supporting so-called tumor “agnostic” drugs, which are markers for the progress of precision … Continue reading “Loxo Unveils More Data, Paves a Path Forward In Precision Oncology”

IDO Don’ts: After Immunotherapy Failure, Experts Say Slow Down

One year ago, on the eve of the yearly American Society of Clinical Oncology conference, a new class of drugs called IDO inhibitors seemed poised to become the next big thing in cancer care. They were a top choice to combine with the powerful but limited immunotherapies that have emerged this decade to fight many … Continue reading “IDO Don’ts: After Immunotherapy Failure, Experts Say Slow Down”

Roche Up, J&J Down as Cancer Drug Combos Yield More Mixed Results

Clinical testing of drug combinations that utilize cancer immunotherapies rolled on this weekend with mixed results. One Phase 3 study from Roche/Genentech in lung cancer succeeded, while another from Genmab and partner Johnson & Johnson spelled trouble for another possible drug combination. Roche’s South San Francisco, CA, Genentech unit said its IMpower130 study, which combines … Continue reading “Roche Up, J&J Down as Cancer Drug Combos Yield More Mixed Results”

Bio Roundup: Brii Does China, Hemophilia Updates, Right to Try & More

Sometimes disruption comes in waves. Since the 1990s, hemophilia patients have had to inject themselves with drugs a few times a week to prevent dangerous internal bleeding. That could soon change. One drug already approved, from Roche, may only need to be taken once a month by hemophilia A patients. Another experimental therapy from Alnylam … Continue reading “Bio Roundup: Brii Does China, Hemophilia Updates, Right to Try & More”

Biotech IPOs Roll On as Kiniksa, Scholar Rock Get $227M Combined

The IPO window remains wide open for biotechs. Two more, Kiniksa Pharmaceuticals and Scholar Rock, make their Wall Street debuts this morning having raised $227 million combined in their IPOs last night. Kiniksa exceeded its funding goals. The Lexington, MA, company sold 1.44 million more shares than it projected, or 8.44 million total, at $18 … Continue reading “Biotech IPOs Roll On as Kiniksa, Scholar Rock Get $227M Combined”

Ahead of ASCO, Merck Touts Another Win for Lung Cancer Drug Combo

The rapidly changing treatment landscape for lung cancer could soon change again. This morning, Merck (NYSE: [[ticker:MRK]]) announced that a Phase 3 study called Keynote-407 has succeeded. The trial tested a combination of its immunotherapy pembrolizumab (Keytruda) and chemotherapy in 560 patients just diagnosed with advanced, squamous non-small cell lung cancer. Merck didn’t disclose specifics, … Continue reading “Ahead of ASCO, Merck Touts Another Win for Lung Cancer Drug Combo”

CAR-T’s Launch, Payers vs. Gene Therapy & Photos From “What’s Hot”

CAR-T. Gene therapy. RNA interference. These aren’t just science experiments. They’re real, cutting-edge medicines either just on the market or soon to be. And the implications of their arrival was the focus of a few spirited discussions at Xconomy’s “What’s Hot in Boston Biotech” event last week at the Broad Institute in Cambridge, MA. Today, … Continue reading “CAR-T’s Launch, Payers vs. Gene Therapy & Photos From “What’s Hot””

Data Could Make Roche Drug a Hemophilia Standard, But for How Long?

It’s been a bumpy ride for emicizumab (Hemlibra) in 2018. The Roche/Genentech drug, approved by the FDA in late 2017, has a chance to become the choice treatment for people with hemophilia A, and new data today help that case. But will safety concerns and emerging competition blunt its rise? At the World Federation of … Continue reading “Data Could Make Roche Drug a Hemophilia Standard, But for How Long?”

Verdine Picks Backers For FogPharma’s $66M Round, Keeping Firm Control

At first blush, the $66 million Series B round that startup FogPharma is announcing this morning seems standard fare, particularly this year, when private biotechs have raised gobs of cash hand over fist. But a closer look shows careful steps by an academic-turned-executive, Greg Verdine, to develop the type of company he wants. He’s amassed … Continue reading “Verdine Picks Backers For FogPharma’s $66M Round, Keeping Firm Control”

Trump Pledges Lower Drug Prices, But Blueprint Is Short on Details

[Editor’s note: Corie Lok co-authored this report.] In a much anticipated speech at the White House this afternoon, President Trump unveiled a long-awaited plan meant to cut the cost of prescription drugs in the U.S. The administration’s “American Patients First” plan, released via a speech and an accompanying 38-page “blueprint” document, laid out some relatively … Continue reading “Trump Pledges Lower Drug Prices, But Blueprint Is Short on Details”

Roche-Exelixis Study Fails as Mixed Year Continues For Cancer Drug Combos

This year is a big one for cancer drug combinations, and the results, so far, have been mixed. Some combinations have shown they may change how certain cancers are treated. Others are falling flat in clinical testing. The latest to come up short is a regimen of Roche/Genentech and Exelixis cancer drugs that failed a … Continue reading “Roche-Exelixis Study Fails as Mixed Year Continues For Cancer Drug Combos”

An Inside Look at CAR-T, Gene Therapy Launches at ‘What’s Hot’ May 16

Getting a new drugmaking technology through clinical development and to market is a major milestone, the culmination of years of scientific work. But the true test of how important that technology is comes afterwards, when the real world gets its hands on it. This type of story is playing out either right now, or soon … Continue reading “An Inside Look at CAR-T, Gene Therapy Launches at ‘What’s Hot’ May 16”

Moderna’s Billions: Will Building Big Before an IPO Pay Off?

When Merck invested $125 million and deepened its ties with Moderna Therapeutics last week, it was more than just an evolution of the relationship between the two companies. It continued a high-stakes strategy by Cambridge, MA-based Moderna and its backers, and made them a case study for a rarely used method of company-building in biotech: … Continue reading “Moderna’s Billions: Will Building Big Before an IPO Pay Off?”

Bio Roundup: IDO Fallout, Express Scripts Dumps Amgen, Isaly Out & More

Here’s a case study of how fast things can change in biopharma. For a few years, a type of cancer drug called an IDO inhibitor was all the rage, a promising path to expanding the reach of immunotherapy. Major drug makers were dealing, too. Through acquisitions and alliances, they rolled out a spate of large-scale … Continue reading “Bio Roundup: IDO Fallout, Express Scripts Dumps Amgen, Isaly Out & More”