Pfizer, GSK to Merge Consumer Health Units, Focus on Prescription Drugs

Two of the world’s top drugmakers want to focus on making prescription drugs. Pfizer and GlaxoSmithKline have agreed to merge their consumer health businesses and spin them off into a joint venture, a move that will both create a new consumer healthcare giant and increase the pressure on each company to churn out innovative medicines. … Continue reading “Pfizer, GSK to Merge Consumer Health Units, Focus on Prescription Drugs”

15 For ’19: Key Clinical Data to Watch For Next Year (Part 2)

On Monday, Xconomy began our annual look ahead at clinical trials that could define biotech and have profound healthcare effects in the coming year. Today we’re circling back with the rest of the list, which includes studies of drugs for nonalcoholic steatohepatitis, cystic fibrosis, spinal muscular atrophy and more. Read on for the details. [Editor’s … Continue reading “15 For ’19: Key Clinical Data to Watch For Next Year (Part 2)”

15 for ’19: Key Clinical Data to Watch for Next Year (Part 1)

It can take decades and billions of dollars to develop a drug, and its fate—and often that of its developer—rests in the outcome of clinical trials. Just look at some of 2018’s biggest biotech stories. The failure of a closely watched cancer immunotherapy combination sent ripples through the sector and led several companies to change … Continue reading “15 for ’19: Key Clinical Data to Watch for Next Year (Part 1)”

To Cover Debts, Synergy Inks $200M Bankruptcy Sale to Bausch Health

Synergy Pharmaceuticals has spent years looking for a buyer. The developer of a struggling drug for two types of chronic constipation finally has one—now that it’s gone bankrupt. New York-based Synergy (NASDAQ [[ticker:SGYP]]) cut a deal on Wednesday to sell itself to Bausch Health, a Canadian specialty pharma company, for roughly $200 million in cash. … Continue reading “To Cover Debts, Synergy Inks $200M Bankruptcy Sale to Bausch Health”

Roche Vet O’Day Tapped to Shape Gilead’s Future as Hep C Sales Fall

Daniel O’Day, a veteran executive from Swiss pharma giant Roche, has been tapped for one of the top jobs in biotech. He’s been named the CEO of Gilead Sciences (NASDAQ: [[ticker:GILD]]), which is searching for a new direction after the quick rise and fall of a once-lucrative business for hepatitis C medicines. The Foster City, … Continue reading “Roche Vet O’Day Tapped to Shape Gilead’s Future as Hep C Sales Fall”

Bio Roundup: ASH Wrap, CRISPR Baby Fallout, Moderna’s Record IPO

The biggest yearly meeting for blood disease research has come and gone, and yet the stories to come out of the American Society of Hematology’s gathering in San Diego represent just a fraction of what happened in biopharma this week. Even if ASH wasn’t on your radar, the CRISPR baby saga—the apparent birth of gene … Continue reading “Bio Roundup: ASH Wrap, CRISPR Baby Fallout, Moderna’s Record IPO”

Moderna Raises $604M and Nails Down Biotech’s Biggest IPO Ever

One of the biggest gambles in the history of the life sciences industry made it to Wall Street this evening when Moderna, a developer of messenger RNA drugs—an unproven technology with enormous potential—priced the largest biotech IPO ever. Now, finally under the scrutiny of investors and analysts, Moderna must prove that its technology has enough … Continue reading “Moderna Raises $604M and Nails Down Biotech’s Biggest IPO Ever”

BCMA Day: At ASH ‘18, Cell Therapies For Myeloma Take Center Stage

The treatment landscape for multiple myeloma, a deadly cancer of the bone marrow that affects about 30,000 Americans every year, has changed significantly over the past decade. And more changes look to be on the way. Take the results of a Phase 3 study just reported today at the American Society of Hematology’s (ASH) yearly … Continue reading “BCMA Day: At ASH ‘18, Cell Therapies For Myeloma Take Center Stage”

ASH 2018: Three Up, Two Down as Big Blood Disease Meeting Rolls On

[Updated 4:37 p.m. See below.] Last week, Xconomy previewed the American Society of Hematology’s big meeting in San Diego, the largest medical gathering in the country on blood diseases. We took a deep dive into five specific disease areas under scrutiny, from lymphoma to hemophilia to sickle cell disease and more. News related to those … Continue reading “ASH 2018: Three Up, Two Down as Big Blood Disease Meeting Rolls On”

GSK Pays $5.1B For Tesaro, Setting Up “PARP” Battle With Rival AstraZeneca

GlaxoSmithKline on Monday agreed to pay $5.1 billion for Tesaro, becoming the latest firm to bet on a new type of cancer drug that has shown promise treating multiple tumor types but has yet to become a blockbuster seller. GSK (NYSE: [[ticker:GSK]]) said it will pay $75 per share in cash for Waltham, MA-based Tesaro … Continue reading “GSK Pays $5.1B For Tesaro, Setting Up “PARP” Battle With Rival AstraZeneca”

Alkermes Schizophrenia Drug Heads To FDA, But Questions Linger

Alkermes got some much-needed good news on Thursday, announcing that an experimental schizophrenia drug fared better than a staple drug for the debilitating disease in a closely watched study. But is that benefit big enough for doctors to prescribe the drug, if it’s approved, over cheap generics? Alkermes (NYSE: [[ticker:ALKS]]), of Dublin, Ireland, and Waltham, … Continue reading “Alkermes Schizophrenia Drug Heads To FDA, But Questions Linger”

X4 Pharma Heads to Nasdaq Via Reverse Merger with Arsanis

A tough setback for Arsanis has given privately held X4 Pharmaceuticals the chance to go public. Arsanis (NASDAQ: [[ticker:ASNS]]) and X4, two Boston-area biotech firms, agreed Tuesday morning to merge in a deal that will enable X4 to become a publicly traded company. The combined company will keep the X4 name and be led by … Continue reading “X4 Pharma Heads to Nasdaq Via Reverse Merger with Arsanis”

Genetic Medicine: FDA OKs 2nd Cancer Drug That Targets DNA, Not Tissues

[Updated 11/26/18. See below.] Going deeper into the new world of genetic medicine, the FDA has for the second time approved a drug that targets a tumor’s DNA fingerprint, no matter where in the body that cancer is found. And more could be on the way. The drug is larotrectinib (Vitrakvi), from Loxo Oncology (NASDAQ: … Continue reading “Genetic Medicine: FDA OKs 2nd Cancer Drug That Targets DNA, Not Tissues”

After Head Injury Drug Fails, Edge Merges with Immuno-Oncology Firm PDS

An experimental head injury drug took Edge Therapeutics all the way to the public markets. But one failed clinical trial later and the company is now heading for the exits. Edge (NASDAQ: [[ticker:EDGE]]) this morning announced plans to merge with a privately held company, PDS Biotechnology of North Brunswick, NJ, that is developing a group … Continue reading “After Head Injury Drug Fails, Edge Merges with Immuno-Oncology Firm PDS”

Data Starts Stretch Run for UniQure’s Upgraded Hemophilia Gene Therapy

The race to treat hemophilia with gene therapy, a one-time, long-lasting infusion, is entering its final stages. And with new data released this morning, Dutch and Lexington, MA, firm UniQure has finalized its plans to be among the top finishers. UniQure (NASDAQ: [[ticker:QURE]]) today disclosed preliminary results from a tiny mid-stage study of a gene … Continue reading “Data Starts Stretch Run for UniQure’s Upgraded Hemophilia Gene Therapy”

Eyeing $500M, Moderna Outlines Plans for Biotech’s Biggest IPO Ever

One of the biggest gambles in biotech history is about to be tested on Wall Street. Moderna, the high-flying yet secretive developer of messenger RNA therapeutics, filed papers on Friday afternoon outlining its long-awaited IPO. In its prospectus, Moderna set an initial target of $500 million for the offering, which would make it the largest … Continue reading “Eyeing $500M, Moderna Outlines Plans for Biotech’s Biggest IPO Ever”

Bio Roundup: Midterm Fallout, Alzheimer’s Search, Postpartum Panel

There was plenty at stake in this week’s midterm elections, and not just in terms of political wins and losses. With congress now divided—Democrats took back control of the House and Republicans extended their majority in the Senate—the implications are significant for U.S. healthcare. Just a day after the results, for instance, Senate Majority Leader … Continue reading “Bio Roundup: Midterm Fallout, Alzheimer’s Search, Postpartum Panel”

With FDA Panel’s Nod, First Postpartum Depression Drug Nears Approval

A panel of experts on Friday voted in favor of bringing what could be the first-ever drug approved specifically for postpartum depression to market. But should it get the expected green light from the FDA next month, how widely will the treatment, which requires a continuous, 60-hour intravenous infusion that a professional will have to … Continue reading “With FDA Panel’s Nod, First Postpartum Depression Drug Nears Approval”

Alkermes Depression Drug Faces Long Odds After FDA Advisors Say No

An FDA advisory panel Thursday panned a new experimental drug for depression from Alkermes, meaning the agency is likely to reject the treatment by early next year. By a 21 to 2 vote, the 23 experts decided that the data accrued by Alkermes (NASDAQ: [[ticker:ALKS]]) don’t support approval of the drug, currently known as ALKS-5461. … Continue reading “Alkermes Depression Drug Faces Long Odds After FDA Advisors Say No”

Orchard, Fueled by GSK’s Ex-Gene Therapy Biz, Nets $200M in IPO

GlaxoSmithKline’s former gene therapy portfolio is at the center of a $200 million IPO, among the larger biopharma offerings on Wall Street over the last several years. Orchard Therapeutics, a London, Boston, and San Francisco-based biotech, sold 14,285,715 shares in its IPO late Tuesday at $14 apiece, raising just over $200 million before discounts due … Continue reading “Orchard, Fueled by GSK’s Ex-Gene Therapy Biz, Nets $200M in IPO”

FDA Blasts Data, Trial Design For Alkermes Depression Drug

The FDA this morning has issued a scathing review of an experimental drug that could be, if the agency ends up approving it next year, a new option for the millions of Americans who struggle with depression and don’t respond to typical treatment. The drug is known as ALKS-5461, from Dublin and Waltham, MA-based Alkermes … Continue reading “FDA Blasts Data, Trial Design For Alkermes Depression Drug”

Free of Baggage, Dicerna Cuts Another RNAi Deal and Gets $200M

It wasn’t too long ago that pharma companies were abandoning the development of RNA interference medicines. But there are some small signs that interest has been rekindled. And Dicerna Pharmaceuticals, which recently ended a long-running legal spat with the field’s leader, Alnylam Pharmaceuticals, is reaping the rewards. This morning Eli Lilly (NYSE: [[ticker:LLY]]) teamed with … Continue reading “Free of Baggage, Dicerna Cuts Another RNAi Deal and Gets $200M”

Pursuing a “Crazy, Stupid” Idea & Photos From Disruptors in Boston

Michael Gilman was so close to retiring two years ago. His company, Padlock Therapeutics, was just acquired by Bristol-Myers Squibb (NYSE: [[ticker:BMY]]), and he was on his last week on the job. But at a board meeting, he saw an old friend, Jennifer Petter—then Russ Petter—and asked, what are you working on these days? Petter, … Continue reading “Pursuing a “Crazy, Stupid” Idea & Photos From Disruptors in Boston”

Vertex Hums Along as AbbVie Snatches Up a Rival’s CF Drugs for $45M

AbbVie has just reworked a long-running deal with partner Galapagos NV, paying $45 million up front—a small sum for the pharmaceutical giant—to gain rights to all of the Belgian company’s experimental cystic fibrosis drugs. The deal changes the competitive landscape for cystic fibrosis, a field currently dominated by Boston-based Vertex Pharmaceuticals (NASDAQ: [[ticker:VRTX]]). AbbVie (NYSE: … Continue reading “Vertex Hums Along as AbbVie Snatches Up a Rival’s CF Drugs for $45M”

With $90M Round, Stoke Eyes a “Spinraza For Epilepsy” And a 2019 IPO

Ed Kaye stepped aside from Sarepta Therapeutics (NASDAQ: [[ticker:SRPT]]) last year after leading the company through one of the most noteworthy and controversial drug approvals in recent memory, for the Duchenne muscular dystrophy drug eteplirsen (Exondys 51). But his hiatus from a publicly traded biotech could be short-lived. Kaye’s new startup, Bedford, MA-based Stoke Therapeutics, … Continue reading “With $90M Round, Stoke Eyes a “Spinraza For Epilepsy” And a 2019 IPO”

Bio Roundup: Prices on TV, Novartis Hearts Radio, Warp Drive’s End

Two new battlegrounds emerged this week in the nation’s ongoing drug pricing debate. The first involved television: The federal government hopes that blaring prices in TV ads will essentially pressure drug makers into lowering them, and the pharma lobby fired back. The second centered around a group of pricey new migraine drugs: The nation’s largest … Continue reading “Bio Roundup: Prices on TV, Novartis Hearts Radio, Warp Drive’s End”

Alnylam Won’t Seek Quick FDA Nod For Second RNAi Drug

A few weeks ago, Alnylam Pharmaceuticals indicated it might seek a speedy FDA approval for what could be the second-ever RNA interference medicine to market. After meeting with regulators, however, the Cambridge, MA, company has come up with a different plan. Alnylam (NASDAQ: [[ticker:ALNY]]) said this morning that it won’t seek accelerated approval of an … Continue reading “Alnylam Won’t Seek Quick FDA Nod For Second RNAi Drug”

Bristol Stumbles in Lung Cancer Again as Rivals Play Catch-Up

Nivolumab (Opdivo) recently became the first immunotherapy ever approved for people with small cell lung cancer (SCLC), a particularly aggressive form of the deadly disease. But the lead given to its developer, Bristol-Myers Squibb, may be short-lived. Bristol (NYSE: [[ticker:BMY]]) said today that its immunotherapy nivolumab (Opdivo) failed a Phase 3 study called Checkmate 331. … Continue reading “Bristol Stumbles in Lung Cancer Again as Rivals Play Catch-Up”

FDA OKs Akcea Rare Disease Drug, Setting Up Market Clash with Alnylam

For the second time since August, the FDA has approved a new treatment for a rare and deadly disease called hereditary transthyretin amyloidosis (hATTR). The agency’s nod for inotersen (Tegsedi) sets the stage for a market battle between the drug’s developer, Akcea Therapeutics, and rival Alnylam Pharmaceuticals. The FDA approved inotersen for treating polyneuropathy, the … Continue reading “FDA OKs Akcea Rare Disease Drug, Setting Up Market Clash with Alnylam”

With Orchard’s IPO, GSK’s Old Gene Therapy Biz Gets a Wall Street Test

GlaxoSmithKline’s former gene therapy portfolio is headed to Wall Street—in the hands of Orchard Therapeutics. The London, Boston, and San Francisco, CA, company has filed for an IPO, aiming to back both a group of in-house gene therapies and others it acquired from GSK in April—among them Strimvelis, which is one of the few approved … Continue reading “With Orchard’s IPO, GSK’s Old Gene Therapy Biz Gets a Wall Street Test”

FDA Approves Roche Drug For All Hemophilia A Patients, But Rivals Loom

The treatment landscape for hemophilia is changing fast, and today marks a turning point. The FDA has just cleared Roche to sell its drug emicizumab (Hemlibra) to patients with any form of hemophilia A—making it, potentially, the choice treatment for the disease. At least for now. The FDA first approved emicizumab in late 2017 for … Continue reading “FDA Approves Roche Drug For All Hemophilia A Patients, But Rivals Loom”

Sarepta Touts New Data and a “Signal” With Duchenne Gene Therapy

For the first time, a gene therapy—a one-time, long-lasting treatment—has shown it may improve the lives of kids with Duchenne muscular dystrophy, a deadly genetic disease with no cure. Patients in a small trial showed improved motor function, such as walking and climbing stairs, and near-normal levels of a key muscle protein. But the data … Continue reading “Sarepta Touts New Data and a “Signal” With Duchenne Gene Therapy”

Bio Roundup: Amarin’s Stunner, Lung Cancer News, Data Dumps & More

Six years ago, a biotech called Amarin won FDA approval of a prescription fish-oil pill, Vascepa, because it could lower triglycerides, a type of fat in the blood. But Amarin didn’t have the evidence that lowering triglycerides with fish oil would really help people. Sales lagged. Amarin’s shares sank. While other groups tested other fish … Continue reading “Bio Roundup: Amarin’s Stunner, Lung Cancer News, Data Dumps & More”

With CRISPR’s Help, KSQ Touts 13 Cancer Drugs and Bags Another $80M

Can CRISPR-Cas9, the landmark genome editing tool, help speed up drug discovery? That was the pitch behind KSQ Therapeutics when it started up in 2015. Three years later, the Cambridge, MA, startup says the CRISPR tool has allowed it to make the kind of progress it wouldn’t have otherwise, with 13 experimental cancer drugs in … Continue reading “With CRISPR’s Help, KSQ Touts 13 Cancer Drugs and Bags Another $80M”

With New Data, Alnylam Could Seek Speedy OK For Second RNAi Drug

Alnylam Pharmaceuticals was just cleared to market the first-ever RNA interference medicine. The second one may not be that far behind. Alnylam (NASDAQ: [[ticker:ALNY]]), of Cambridge, MA, said this morning that it could soon file for accelerated approval of an RNAi drug, givosiran, that the company is developing for acute hepatic porphyria, a rare genetic … Continue reading “With New Data, Alnylam Could Seek Speedy OK For Second RNAi Drug”

Syntimmune Turns Drug For Rare Anemia Into $400M Alexion Buyout

Syntimmune, a biotech startup formed by the veteran founders of the hemophilia drug developer Syntonix, has been acquired by Alexion Pharmaceuticals for $400 million in cash up front. Syntimmune is right in Alexion’s (NASDAQ: [[ticker:ALXN]]) wheelhouse. The Boston-based firm is best known for eculizumab (Soliris), a drug for ultra-rare blood diseases. In buying Syntimmune, Alexion … Continue reading “Syntimmune Turns Drug For Rare Anemia Into $400M Alexion Buyout”

Catch CRISPR 2.0, RNA-Targeting Drugs & More At “Disruptors” on Oct. 17

These days biotechs are raising cash and going public at a record pace. But before the Wall Street buzz and the big raises, they all started somewhere—with an idea, a pitch, and perhaps a little luck. Xconomy dives into these company creation stories at “Boston’s Life Science Disruptors,” our yearly look under the hood at … Continue reading “Catch CRISPR 2.0, RNA-Targeting Drugs & More At “Disruptors” on Oct. 17”

Morphic Nets Another $80M For Integrin Drugs & First Human Tests

Morphic Therapeutic, a startup from Harvard scientist-entrepreneur Tim Springer, has reloaded with an $80 million Series B round that should get the company to its first human tests. The round includes a number of crossover investors (including Invus and EcoR1; Novo Holdings and Omega Funds co-led the financing), which back both private and publicly traded … Continue reading “Morphic Nets Another $80M For Integrin Drugs & First Human Tests”

Another Precision Step for Roche as Ignyta Drug Heads to Regulators

There was more news this morning in the advancement of precision cancer drugs, which target a tumor’s genetic signature. Roche plans to file for approval of entrectinib, a drug the Swiss firm acquired when it bought San Diego, CA-based Ignyta for $1.7 billion last year. Roche’s Genentech unit provided updated data pooled from three early … Continue reading “Another Precision Step for Roche as Ignyta Drug Heads to Regulators”

Amarin Soars as Fish Oil Pill Cuts Risk of Strokes in Long-Awaited Study

Can fish oil help prevent, or reduce the risk of heart disease? Several studies have failed to show that it can. But Amarin this morning is releasing data from a massive study showing that its prescription-grade fish oil pill, Vascepa, has done that—at least for some patients. Amarin (NASDAQ: [[ticker:AMRN]]), of Bedminster, NJ, and Dublin, … Continue reading “Amarin Soars as Fish Oil Pill Cuts Risk of Strokes in Long-Awaited Study”

Europe Advisory Panel Rejects Sarepta’s Duchenne Drug Once Again

Sarepta Therapeutics won one of the most controversial drug approvals in recent memory, when the FDA in September 2016 approved the Duchenne muscular dystrophy drug eteplirsen (Exondys 51) off a slim set of data. The Cambridge, MA, company isn’t having the same luck in Europe. Sarepta (NASDAQ: [[ticker:SRPT]]) said today that the Committee for Medicinal … Continue reading “Europe Advisory Panel Rejects Sarepta’s Duchenne Drug Once Again”

Amicus Shells Out $100M For Celenex And Dives Into Gene Therapy

Fresh off finally winning an FDA nod for its first product, Amicus Therapeutics is getting in on the gene therapy renaissance. This morning, Cranbury, NJ-based Amicus (NASDAQ: [[ticker:FOLD]]) agreed to pay $100 million up front to buy a privately held company, Celenex, spun out of Nationwide Children’s Hospital in Ohio. The acquisition gives Amicus a … Continue reading “Amicus Shells Out $100M For Celenex And Dives Into Gene Therapy”

Viking Liver Drug Shows Promise for NASH, Shares Soar on Study Data

[Updated, 2:45 pm ET, see below] There is another update today in the competitive race to treat nonalcoholic steatohepatitis (NASH), an increasingly common liver disease with no FDA-approved therapies. Viking Therapeutics released preliminary results from a Phase 2 study suggesting its experimental drug might impact some of the disease’s many dangerous effects on the liver. … Continue reading “Viking Liver Drug Shows Promise for NASH, Shares Soar on Study Data”

FDA Clears Mersana to Restart Cancer Drug Trial With New Safeguards

The FDA has cleared Mersana Therapeutics to resume enrolling patients in a study of an experimental cancer drug now that the Cambridge, MA, company is taking new precautions to protect patients from potential harm. The company says the FDA lifted a “partial clinical hold” on the Mersana (NASDAQ: [[ticker:MRSN]]) drug XMT-1522, a move that had … Continue reading “FDA Clears Mersana to Restart Cancer Drug Trial With New Safeguards”

Acorda’s Tough Week Continues as FDA Delays Parkinson’s Drug Review

Acorda Therapeutics has taken its second hit in less than a week. The FDA this morning extended its review of Acorda’s (NASDAQ: [[ticker:ACOR]]) experimental Parkinson’s disease drug Inbrija, an inhalable form of levodopa, by three months. The agency will now decide whether to approve the drug—whose success is critical to the future of the Ardsley, … Continue reading “Acorda’s Tough Week Continues as FDA Delays Parkinson’s Drug Review”

With OneOncology, Flatiron and Cancer Docs Aim to Boost Community Care

A number of new technologies, from immunotherapy to targeted medicines and diagnostics, have all helped change cancer care the past few years. But not all cutting-edge technologies are available to patients who seek treatment at smaller clinics. OneOncology, an unusual new startup being launched this morning, aims to help cancer patients in community settings gain … Continue reading “With OneOncology, Flatiron and Cancer Docs Aim to Boost Community Care”

FDA, Following EMA, Nixes Amicus’ Plans for Speedy OK of Pompe Drug

The FDA has rejected plans from Amicus Therapeutics to seek speedy approval of an experimental drug for Pompe disease, calling instead for more data the Cranbury, NJ, company hopes to accrue over the next year. The FDA told Amicus (NASDAQ: [[ticker:FOLD]]) that its “current clinical package is not sufficient” to support what’s known as accelerated … Continue reading “FDA, Following EMA, Nixes Amicus’ Plans for Speedy OK of Pompe Drug”

Bio Roundup: A Gene Editing 1st, China Rises, Schenkein Steps Away

If you’re still recovering from a Labor Day hangover or busy searching for the identity of the anonymous writer of the anti-Trump op-ed for the New York Times, you may have missed a few biomedical firsts. This week brought the first sliver of human data from an in-body gene editing procedure, and the results were … Continue reading “Bio Roundup: A Gene Editing 1st, China Rises, Schenkein Steps Away”

Working with Patients and a Fresh $80M, Fulcrum Eyes a 2019 IPO

Fulcrum Therapeutics, a Cambridge, MA, startup run by the former CEO of Epizyme, has closed an $80 million Series B round that lays the groundwork for a likely IPO next year, its CEO says. The new funding, led by Foresite Capital, comes from a wide array of crossover investors, such as Fidelity Management and Research, … Continue reading “Working with Patients and a Fresh $80M, Fulcrum Eyes a 2019 IPO”

After a High-Speed Decade, Agios CEO David Schenkein to Step Aside

David Schenkein, the longtime CEO of Agios Pharmaceuticals, will step aside next year after transforming what was once a high-risk Cambridge, MA-based startup into a public biotech with over 500 employees and two drugs on the market. Schenkein’s replacement will be Jacqualyn Fouse, former president and chief operating officer of Celgene (NASDAQ: [[ticker:CELG]]) and a … Continue reading “After a High-Speed Decade, Agios CEO David Schenkein to Step Aside”