Exosomes Head to Wall Street As Codiak Biosciences Lines Up IPO

Do Wall Street investors believe that exosomes, the tiny bubbles once thought to be just cellular garbage bins, may be the key to treating a slew of potential diseases? The coming IPO for Codiak Biosciences will present a test case. Codiak, a Cambridge, MA, company run by former Biogen (NASDAQ: [[ticker:BIIB]]) research chief Doug Williams, … Continue reading “Exosomes Head to Wall Street As Codiak Biosciences Lines Up IPO”

Biogen, Taking Its Crisis “Seriously,” Keeps Focus on Neuroscience

[Updated, 1:00 pm ET, see below.] A month after the failure of its once-promising Alzheimer’s disease drug aducanumab shattered its share price, Biogen is trying to pick up the pieces. Biogen (NASDAQ: [[ticker:BIIB]]) hosted a conference call Wednesday morning detailing its quarterly results. But financials aside, the call was about the Cambridge, MA, company’s future … Continue reading “Biogen, Taking Its Crisis “Seriously,” Keeps Focus on Neuroscience”

Bio Roundup: Nationwide’s Rise, a Surgeon’s Quest, CRISPR USA & More

Persistence and perseverance were the themes of two of our top stories this week. First there’s the story of Nationwide Children’s Hospital. The Columbus, Ohio, hospital chose to invest in gene therapy when the field was in its darkest days. After some ups and downs, the team it put in place helped advance some of … Continue reading “Bio Roundup: Nationwide’s Rise, a Surgeon’s Quest, CRISPR USA & More”

$100M for Talaris Gives Surgeon a Shot to Reinvent Organ Transplants

When Novartis dissolved its gene and cell therapy unit a few years ago, a bunch of projects the Swiss pharma giant had incubated were tossed aside. One of them was the work of surgeon Suzanne Ildstad, who has spent decades trying to develop a new way to improve the health outcomes of patients who need … Continue reading “$100M for Talaris Gives Surgeon a Shot to Reinvent Organ Transplants”

Alzheimer’s, Gene Therapy & More: Agenda For “What’s Hot” on May 21

The gene therapy boom. The frustration of failure after failure in Alzheimer’s disease. The critical interplay between venture firms and “crossover” investors in a volatile, unpredictable macro-environment. The growing need for (and role of) diversity initiatives within the life sciences community—and the personal stories behind them. These are just some of the topics Xconomy will … Continue reading “Alzheimer’s, Gene Therapy & More: Agenda For “What’s Hot” on May 21”

Another Week, Another Gene Therapy Buyout: Catalent Pays $1.2B for Paragon

The gene therapy boom continued this morning with yet another acquisition. Contract drug manufacturer Catalent (NYSE: [[ticker:CTLT]]), of Somerset, NJ, has agreed to buy Paragon Bioservices for $1.2 billion, grabbing hold of a privately held developer of the viral “vectors” that gene therapy companies use to deliver their treatments. In a gene therapy procedure, these … Continue reading “Another Week, Another Gene Therapy Buyout: Catalent Pays $1.2B for Paragon”

How an Ohio Kids’ Hospital Quietly Became Ground Zero for Gene Therapy

[Updated, 4/22/19, see below] If a once-modest regional hospital and its new biotech allies have their way, the capital of Ohio could one day rival America’s other biomedical hubs. “Our goal is to make Columbus the center of the universe for gene therapy,” says Doug Ingram, CEO of Sarepta Therapeutics. Sarepta (NASDAQ: [[ticker:SRPT]]) is based … Continue reading “How an Ohio Kids’ Hospital Quietly Became Ground Zero for Gene Therapy”

Alnylam Makes Case For 2nd RNAi Drug at Big Liver Disease Meeting

The “attacks,” as they’re known, are debilitating and even possibly fatal. A patient with the ultra-rare genetic disease acute hepatic porphyria (AHP) is struck with excruciating abdominal pain and rushes to a doctor’s office or hospital for some type of pain relief—even a surgery—to feel better. And then another attack could come, and another, driving … Continue reading “Alnylam Makes Case For 2nd RNAi Drug at Big Liver Disease Meeting”

Thwarting Opposition, Bristol Gets Shareholder OK for $74B Celgene Buyout

The early opposition has turned out to be just noise. Shareholders of Bristol-Myers Squibb today approved the pharma giant’s planned buyout of Celgene, paving the way for a deal that will create one of the largest biopharma organizations in the world, and send ripples throughout the sector. Bristol (NYSE: [[ticker:BMY]]) said Friday that more than … Continue reading “Thwarting Opposition, Bristol Gets Shareholder OK for $74B Celgene Buyout”

Alnylam Gets $800M From Regeneron to Bring RNAi to Eye, Brain Diseases

Alnylam and Regeneron Pharmaceuticals are announcing a wide-ranging alliance this morning to co-develop treatments for eye and neurological diseases using RNA interference, a method of drugmaking that has recently come of age after years of ups and downs. Regeneron (NASDAQ: [[ticker:REGN]]), of Tarrytown, NY, will pay Alnylam (NASDAQ: [[ticker:ALNY]]) $400 million in cash up front … Continue reading “Alnylam Gets $800M From Regeneron to Bring RNAi to Eye, Brain Diseases”

AACR 2019 Roundup: Notes from a Weekend of Early Stage Cancer Results

[Editor’s note: Alex Lash co-authored this report] The American Association for Cancer Research’s annual meeting is wrapping up in Atlanta today. The conference typically focuses on early research and clinical work, not the big trials that can change the way doctors practice medicine and that compete for headline space at conferences like the American Society … Continue reading “AACR 2019 Roundup: Notes from a Weekend of Early Stage Cancer Results”

SpringWorks, Armed with Ex-Pfizer Cancer Drugs, Reloads with $125M

SpringWorks Therapeutics, a startup spun out of Pfizer to develop drugs gathering dust on the New York pharma giant’s shelves, has closed its second nine-figure financing round in less than two years. The $125 million Series B funding gives the New York City startup the backing to bring two cancer drugs into what could be … Continue reading “SpringWorks, Armed with Ex-Pfizer Cancer Drugs, Reloads with $125M”

Panel Nod Moves Bluebird’s Gene Therapy Closer to Europe’s Market

A one-time, long-lasting treatment for the genetic blood disease beta thalassemia looks to be the next gene therapy on its way to market—at least, for now, in Europe. The Committee for Medicinal Products for Human Use, which guides drug approval decisions in Europe, has recommended a “conditional” approval—on a thinner body of evidence than normal—of … Continue reading “Panel Nod Moves Bluebird’s Gene Therapy Closer to Europe’s Market”

Bio Roundup: Amyloid Angst, NASH News, Brammer Bagged & More

A week after the failure of Biogen’s Alzheimer’s drug aducanumab, the ripple effects are still being felt—and not just by Biogen, which lost billions of dollars in market value in a flash. We’ll kick off this week’s roundup with the aducanumab reaction, which includes plenty of thoughts about the future of the so-called “amyloid hypothesis” … Continue reading “Bio Roundup: Amyloid Angst, NASH News, Brammer Bagged & More”

New Data Puts Aldeyra One Step Closer to FDA Filing for Eye Drug

Aldeyra Therapeutics has disclosed positive results from the first of multiple Phase 3 studies of a new type of inflammation-fighting medicine the Lexington, MA, company has been testing in a variety of conditions. The study Aldeyra (NASDAQ: [[ticker:ALDX]]) reports on this morning is a Phase 3 trial called “Alleviate,” which tested its drug reproxalap against … Continue reading “New Data Puts Aldeyra One Step Closer to FDA Filing for Eye Drug”

Thermo Bags Brammer For $1.7B as Gene Therapy Deal Spree Continues

Thermo Fisher Scientific late Sunday evening agreed to buy Brammer Bio for $1.7 billion, yet another bet by a large biopharmaceutical organization on the future of gene therapy. Thermo (NYSE: [[ticker:TMO]]), a Waltham, MA, developer of lab equipment, made an all-cash offer for Cambridge, MA-based Brammer, a contract manufacturer of gene therapy delivery tools known … Continue reading “Thermo Bags Brammer For $1.7B as Gene Therapy Deal Spree Continues”

Adieu to Adu: Biogen’s Big Alzheimer’s Bet Flops, Shares Routed

Another once-promising drug for Alzheimer’s disease has failed. And the news is devastating for the drug’s developer, Biogen (NASDAQ: [[ticker:BIIB]]), which went all in on a high-stakes gamble that its treatment, aducanumab, might succeed where so many others haven’t. This morning, Biogen reported that it is stopping two parallel, highly anticipated Phase 3 studies in … Continue reading “Adieu to Adu: Biogen’s Big Alzheimer’s Bet Flops, Shares Routed”

CRISPR Diagnostic Race Heats Up as Sherlock Bio Launches With $35M

The high-stakes race to use CRISPR gene editing technology to create medicines has been underway for years now, and human trials are just starting. But the idea of using CRISPR as a tool to detect and diagnose a disease has been gaining traction, too, and the launch of a new startup today called Sherlock Biosciences … Continue reading “CRISPR Diagnostic Race Heats Up as Sherlock Bio Launches With $35M”

Pfizer Bolsters Gene Therapy Position with $51M Vivet Deal

Pfizer this morning added to its methodical push into the gene therapy field, paying $51 million for a 15 percent stake in Vivet Therapeutics and the option to buy the Parisian biotech in the future. Vivet is developing an experimental gene therapy, VTX-801, for the rare genetic disorder Wilson’s disease, which causes a potentially deadly … Continue reading “Pfizer Bolsters Gene Therapy Position with $51M Vivet Deal”

Flagship Reloads With $824M For Latest Growth Capital Fund

Flagship Pioneering, one of the Boston area’s top biotech company creators, has closed a new $824 million fund to help fuel the growth of its startups. The new fund—Special Opportunities Fund II—will complement the cash the firm uses to start its companies, much like the $285 million “Special Opportunities” fund the firm announced in 2016. In … Continue reading “Flagship Reloads With $824M For Latest Growth Capital Fund”

FDA OKs Depression Drug For New Moms: Fast Relief But Tough to Take

The FDA has just made brexanolone (Zulresso) the first medicine approved specifically for post-partum depression, a potentially devastating disorder that afflicts roughly one in every seven new mothers in the U.S., according to the American Psychological Association. Now the drug’s owner, Sage Therapeutics (NASDAQ: [[ticker:SAGE]]), of Cambridge, MA, must convince doctors to prescribe and new … Continue reading “FDA OKs Depression Drug For New Moms: Fast Relief But Tough to Take”

FDA Clears Aerie’s Combo Glaucoma Drug, But Will it Sell?

The FDA late Tuesday approved a new type of glaucoma drug from Aerie Pharmaceuticals that, in clinical testing, beat the generic eye drops known as prostaglandin analogues (PGAs) that are typically prescribed to slow the progression of the disease. Now Aerie (NASDAQ: [[ticker:AERI]]) will try to prove that the drug can overcome some of its … Continue reading “FDA Clears Aerie’s Combo Glaucoma Drug, But Will it Sell?”

The Life, Troubles, and Celgene Legacy of Deal Guru George Golumbeski

George Golumbeski is one of the most prolific dealmakers in the biopharmaceutical world. But the deal that changed his life is a pact he made with himself when he was ten years old. Playing with friends in front of his house in Hampton, VA, he watched his mother drag his dead-drunk father, a U.S. Air … Continue reading “The Life, Troubles, and Celgene Legacy of Deal Guru George Golumbeski”

Data in Hand, Alnylam to Seek OK for Second RNAi Drug

Alnylam Pharmaceuticals this morning released new data that it believes to be good enough to bring the second-ever RNA interference medicine to market, a treatment for a potentially deadly, rare genetic disease called acute hepatic porphyria. Alnylam’s (NASDAQ: [[ticker:ALNY]]) experimental drug givosiran met its main goal in a 94-patient Phase 3 study called Envision. The … Continue reading “Data in Hand, Alnylam to Seek OK for Second RNAi Drug”

After AGTC Deal Flops, Biogen Bets $800M on Gene Therapy Rival Nightstar

Biogen’s biggest recent foray into gene therapy, an ill-fated pact with AGTC, didn’t pay dividends. So the company this morning agreed to buy a rival, Nightstar Therapeutics, and its portfolio of gene therapies for rare eye diseases, for $800 million. Biogen (NASDAQ: [[ticker:BIIB]]) this morning agreed to pay $25.50 per share in cash for Nightstar … Continue reading “After AGTC Deal Flops, Biogen Bets $800M on Gene Therapy Rival Nightstar”

Bio Roundup: Pharma in DC, Bristol-Cel in Trouble, Roche Gets A Spark

Could a shareholder revolt doom the planned marriage of Bristol-Myers Squibb and Celgene? Analysts have hinted at the possibility since the two companies shook hands in January, but the threat became real this week. Bristol (NYSE: [[ticker:BMY]]) and Celgene (NASDAQ: [[ticker:CELG]]) have campaignned heavily, starting with a joint presentation by their CEOs at the J.P. … Continue reading “Bio Roundup: Pharma in DC, Bristol-Cel in Trouble, Roche Gets A Spark”

Sarepta Touts More Gene Therapy Data, Snaps Up Myonexus For $165M

Sarepta Therapeutics (NASDAQ: [[ticker:SRPT]]) has been steadily building up its gene therapy capabilities for a few years now, and it took two more steps forward on that front this morning. First, it exercised an option to buy gene therapy startup Myonexus Therapeutics for $165 million. And then it disclosed early human results from the first … Continue reading “Sarepta Touts More Gene Therapy Data, Snaps Up Myonexus For $165M”

Blackstone, Novartis Start Up Anthos With $250M and a Heart Drug

Blackstone Life Sciences, the biopharma investing arm of private equity firm Blackstone Group, has made its first strike. It has joined with Novartis (NYSE: [[ticker:NVS]]) to start a new biotech, Anthos Therapeutics, backed with $250 million in cash and rights to an experimental drug originally discovered by the Swiss firm. The Cambridge, MA, startup, Anthos … Continue reading “Blackstone, Novartis Start Up Anthos With $250M and a Heart Drug”

No Details, But AstraZeneca Touts PARP Drug Win in Pancreatic Cancer

It’s no secret that so-called PARP inhibitors, a new class of cancer drugs, have struggled to gain traction commercially, even as they’ve shown promise treating multiple tumor types. But today brings news that could bolster the future prospect of PARP blockers: one of them, a drug known as olaparib (Lynparza) from AstraZeneca and partner Merck, … Continue reading “No Details, But AstraZeneca Touts PARP Drug Win in Pancreatic Cancer”

Oyster Point Gets $93M To Clear Up Dry Eye With a Nasal Spray

Dry eye disease affects millions of people in the U.S., and while several treatments, mostly eye drops, are available, they all have flaws. An emerging startup called Oyster Point Pharmaceuticals is trying a completely different approach—a nasal spray that coaxes the body into making tears. And the company just got enough cash to get that … Continue reading “Oyster Point Gets $93M To Clear Up Dry Eye With a Nasal Spray”

Roche Bets $4.8B on Spark Therapeutics and the Future of Gene Therapy

Roche has agreed to buy Spark Therapeutics for $4.8 billion, the latest strong signal that major pharmaceutical companies are ready to gamble on the promise of gene therapy despite its uncertain commercial prospects. The Swiss pharma giant will pay $114.50 per share, in cash, for Spark (NASDAQ: [[ticker:ONCE]]), a 122 percent premium to the gene … Continue reading “Roche Bets $4.8B on Spark Therapeutics and the Future of Gene Therapy”

With New Data, Intercept On Track for First-Ever Approved NASH Drug

The first-ever marketed medicine for the fatty liver disease known as nonalcoholic steatohepatitis, or NASH, could be on the horizon. The data, from a closely watched drug known as obeticholic acid, aren’t perfect. But they are nonetheless expected by the drug’s developer, Intercept Pharmaceuticals, to be good enough to support approval. Intercept (NASDAQ: [[ticker:ICPT]]) this … Continue reading “With New Data, Intercept On Track for First-Ever Approved NASH Drug”

Passage Bio Gets $116M As Wilson, Yamada Team Up On Gene Therapy Again

Longtime biopharmaceutical executive Tachi Yamada helped keep the work of gene therapy pioneer James Wilson alive 20 years ago when Wilson was mired in controversy and the emerging field was in its darkest days. Now that gene therapy has come of age, in part because of Wilson’s work, the two are teaming up again. They’ve … Continue reading “Passage Bio Gets $116M As Wilson, Yamada Team Up On Gene Therapy Again”

Losing “Scalps”? Despite Pharma Fear, A Split on Trump Rx-Price Plans

Despite angry presidential tweets and other anti-pharma rhetoric, the drug industry has benefited under the Trump administration, thanks in part to a massive corporate tax cut. Indeed, for nearly two decades, the industry has been able to fend off its critics and gain win after win from politicians and regulators, including tax holidays, faster drug … Continue reading “Losing “Scalps”? Despite Pharma Fear, A Split on Trump Rx-Price Plans”

Matching Amgen, Regeneron to Cut List Price of Heart Drug by 60%

Regeneron Pharmaceuticals and partner Sanofi are cutting the list price of their heart drug alirocumab (Praluent) by 60 percent, the latest acknowledgement that the treatment—which has shown a striking ability to lower cholesterol, but has struggled to sell because of its high price tag—isn’t getting to the patients who need it. Starting in early March, … Continue reading “Matching Amgen, Regeneron to Cut List Price of Heart Drug by 60%”

Bio Roundup: New CRISPR Feud, A NY Splash, Pharma vs. Congress & More

If you’re into congressional spectacles, stay tuned later this month. A group of top biopharma executives—the full roster isn’t known yet—will testify at a Feb. 26 hearing before the Senate Committee on Finance to defend their drug pricing practices. No executives showed up for the previous hearing, but reports indicate industry has changed its stance … Continue reading “Bio Roundup: New CRISPR Feud, A NY Splash, Pharma vs. Congress & More”

With Its First Human Data, Solid Slips in Duchenne Gene Therapy Race

The race to treat Duchenne muscular dystrophy with gene therapy, a one-time, potentially long-lasting treatment, is being closely watched. But new data from Solid Biosciences cast doubt on whether the Cambridge, MA, company can keep pace with the current leader, Sarepta Therapeutics. Solid (NASDAQ: [[ticker:SLDB]]) this morning is disclosing the first results from the first … Continue reading “With Its First Human Data, Solid Slips in Duchenne Gene Therapy Race”

NYCEDC Finally Puts Its NY Bio Fund to Work With $60M For HiberCell

It’s been a question posed for years by many around the New York biotech scene: What is going on with the New York City Economic Development Corp.’s $150 million biotech fund, which was formed in 2013 but hadn’t made an investment? Finally, today, there’s an answer. A small piece of the money from the NYCEDC’s … Continue reading “NYCEDC Finally Puts Its NY Bio Fund to Work With $60M For HiberCell”

With $100M MeiraGTx Deal, J&J Signals Bigger Move Into Gene Therapy

Gene therapy has come of age over the past few years. And Johnson & Johnson this morning gave its biggest signal yet that it wants in on the emerging field. J&J (NYSE: [[ticker:JNJ]]) has inked a deal with MeiraGTX (NASDAQ: [[ticker:MGTX]]) to grab rights to a group of experimental gene therapies for inherited forms of … Continue reading “With $100M MeiraGTx Deal, J&J Signals Bigger Move Into Gene Therapy”

All Eyes on Biogen as Roche Alzheimer’s Drug Flunks Final Test

Roche this morning added yet another failure to the ever-growing number of experimental Alzheimer’s disease drugs to crumble in late-stage human testing. The Swiss pharma and partner AC Immune (NASDAQ: [[ticker:ACIU]]) will stop two Phase 3 studies of their drug crenezumab in Alzheimer’s early after an interim analysis by a committee of investigators showed those … Continue reading “All Eyes on Biogen as Roche Alzheimer’s Drug Flunks Final Test”

Neurocrine Pays $165M to Bankroll Voyager’s Gene Therapy for Parkinson’s

[Updated, 11:40 a.m. ET, see below] The road to potential approval for Voyager Therapeutics’ experimental gene therapy for Parkinson’s disease got a bit longer, and more expensive, last year. So this morning the company cut a deal with Neurocrine Biosciences to bankroll the journey—and forfeit some of the financial upside for the product, known as … Continue reading “Neurocrine Pays $165M to Bankroll Voyager’s Gene Therapy for Parkinson’s”

In Another Lung Cancer Setback, Bristol Yanks FDA App For Drug Combo

Bristol-Myers Squibb has lost yet more ground in its ongoing cancer immunotherapy battle with rival Merck. Along with its earnings, the pharma firm reported Thursday that it has pulled a key approval application to use a combo regimen of its already approved immunotherapies, nivolumab (Opdivo) and ipilimumab (Yervoy), in a portion of patients with newly … Continue reading “In Another Lung Cancer Setback, Bristol Yanks FDA App For Drug Combo”

Bio Roundup: Aimmune Drama, Shutdown Blues, Perlmutter Talks Cancer

The partial government shutdown, now the longest in U.S. history, began to take a bite out of biotech this week, served with a hot side dish of confusion. On Tuesday, Aimmune Therapeutics (NASDAQ: [[ticker:AIMT]]) said the shutdown would delay the review—and potential approval—of its experimental treatment for peanut allergy. Aimmune said it received a letter … Continue reading “Bio Roundup: Aimmune Drama, Shutdown Blues, Perlmutter Talks Cancer”

Merck And The Future of Immuno-Oncology: A Chat With Roger Perlmutter

Cancer immunotherapy is, in a word, tantalizing. It might save the life of someone at death’s door and keep the cancer at bay for years. Or it might not work at all. There’s no telling what a patient will experience. “There’s a real poverty of understanding of how the machine that is us works,” says … Continue reading “Merck And The Future of Immuno-Oncology: A Chat With Roger Perlmutter”

Government Shutdown Hits Biotech as FDA Punts Review of Aimmune Drug

[Updated, 11:25 am ET, see below] The partial U.S. government shutdown has just had its first tangible effects on the potential approval of a new drug: The FDA review of a peanut allergy treatment from Aimmune Therapeutics (NASDAQ: [[ticker:AIMT]]) has been delayed. In a regulatory filing posted late Wednesday, Aimmune, of Brisbane, CA, said that … Continue reading “Government Shutdown Hits Biotech as FDA Punts Review of Aimmune Drug”

In a Huge Biopharma Shakeup, Bristol-Myers to Buy Celgene For $74B

Bristol-Myers Squibb has agreed to acquire Celgene in a deal that would create one of the largest biopharma organizations in the world and send ripples throughout the life sciences sector. Bristol (NYSE: [[ticker:BMY]]) on Thursday morning inked a deal to buy Celgene (NASDAQ: [[ticker:CELG]]) in a deal that values the Summit, NJ, drugmaker at $74 … Continue reading “In a Huge Biopharma Shakeup, Bristol-Myers to Buy Celgene For $74B”

MyoKardia Goes Solo as Sanofi Ends Precision Heart Drug Deal

MyoKardia rode a partnership with Sanofi to the public markets a few years ago. But the two companies are cutting ties today, with MyoKardia regaining full rights to its drug programs and assuming all the upside—and risk—of their success or failure. MyoKardia (NASDAQ: [[ticker:MYOK]]) said the deal, which started in September 2014, will wrap up … Continue reading “MyoKardia Goes Solo as Sanofi Ends Precision Heart Drug Deal”

Atlas Venture Bags $250M For New Growth Capital Fund

Atlas Venture says it has raised a $250 million fund meant to continue backing its startups as they grow. The Cambridge, MA, life sciences venture firm, known for seeding and incubating biotech startups, will use the new “Atlas Venture Opportunity Fund I” to invest in portfolio companies that have progressed to a Series B round … Continue reading “Atlas Venture Bags $250M For New Growth Capital Fund”

Acorda Gets FDA Nod for New Parkinson’s Drug, But Rival Looms

The FDA late Friday approved a new treatment meant to help Parkinson’s disease patients cope with “off” episodes, when their medications stop working. The drug, Inbrija, from Acorda Therapeutics (NASDAQ: [[ticker:ACOR]]), is one of two similar treatments—along with APL-130277, from Sunovion Pharmaceuticals—expected to come to market in 2019. And its commercial success is critical for … Continue reading “Acorda Gets FDA Nod for New Parkinson’s Drug, But Rival Looms”

With New Results, FibroGen’s Anemia Pill Inches Closer to FDA Review

There was a new development today in the high-stakes race to use pills, not injectable biologic drugs, to treat anemia. San Francisco, CA-based FibroGen and partners AstraZeneca and Astellas Pharma have reported that their experimental drug, roxadustat, has succeeded in five more Phase 3 trials, which bolster its chances of approvals in the U.S. and … Continue reading “With New Results, FibroGen’s Anemia Pill Inches Closer to FDA Review”