With $59M, Casma and Ex-Constellation CEO Take Aim at Cellular Trash

Scientists have known for decades that cells can recycle their own trash, but they’ve only recently started to figure out how. Casma Therapeutics is trying to prove that those insights could be useful in treating a wide range of diseases. Formed by Third Rock Ventures, Casma has secured a $58.5 million Series A round and … Continue reading “With $59M, Casma and Ex-Constellation CEO Take Aim at Cellular Trash”

Express Scripts Picks Regeneron Over Amgen, Cuts Heart Drug Price

Regeneron Pharmaceuticals and partner Sanofi have cut an unusual deal with Express Scripts, the nation’s largest drug-buying middleman. The two drug makers will cut the price of their $14,000-a-year cholesterol-lowering therapy alirocumab (Praluent). In return, Express Scripts will quickly approve treatment requests. The deal also sidelines Amgen, whose rival heart drug evolocumab (Repatha) will no … Continue reading “Express Scripts Picks Regeneron Over Amgen, Cuts Heart Drug Price”

With New Data, Karyopharm Says It Will Ask for Myeloma Approval

Karyopharm Therapeutics (NASDAQ: [[ticker:KPTI]]) reported this afternoon that its cancer drug selinexor has passed a big test in treating some of the most desperate multiple myeloma patients—those who have failed at least five other therapies. The Newton, MA-based company will ask U.S. and European drug regulators to consider selinexor for approval. Karyopharm (NASDAQ: [[ticker:KPTI]]) reports … Continue reading “With New Data, Karyopharm Says It Will Ask for Myeloma Approval”

SMA Day: AveXis Gene Therapy Creeps Forward as Biogen Drug’s Sales Slow

Just a few years ago, there were no drugs available for patients with the rare genetic disease spinal muscular atrophy. Now two cutting-edge therapies look headed for a commercial battle, with each data and revenue update a key moment in the race. Biogen (NASDAQ: [[ticker:BIIB]]) was the first to bring an SMA drug to market, … Continue reading “SMA Day: AveXis Gene Therapy Creeps Forward as Biogen Drug’s Sales Slow”

With Acorda Data, Experts Weigh the Benefits of New Parkinson’s Drugs

Levodopa, the gold-standard medicine for Parkinson’s disease, has helped millions of patients since the 1970s manage the neurodegenerative disease. But there’s a caveat. Nothing is available that can slow or reverse the loss of brain cells, and what’s more, levodopa’s effects wane over time. So-called “off” episodes, when the drug isn’t working, can become more … Continue reading “With Acorda Data, Experts Weigh the Benefits of New Parkinson’s Drugs”

Sage Postpartum Depression Drug Heads to FDA Review

A new treatment for postpartum depression has inched closer to market. Sage Therapeutics this morning filed for FDA approval of brexanolone, which could become the company’s first marketed product as well as the first-ever drug approved specifically for PPD, which affects some 10 to 20 percent of women giving birth every year in the U.S. … Continue reading “Sage Postpartum Depression Drug Heads to FDA Review”

Biogen Pays $1B to Broaden Ionis Pact, Betting More on RNA Drugs

Biogen will pay Ionis Pharmaceuticals $1 billion to expand their current partnership, doubling down on the RNA drugmaking technology that brought the spinal muscular atrophy drug nusinersen (Spinraza) to market. Ionis (NASDAQ: [[ticker:IONS]]) gets $325 million in cash up front in the deal, while Biogen (NASDAQ: [[ticker:BIIB]]) will also buy 11,501,153 Ionis shares at $54.34 … Continue reading “Biogen Pays $1B to Broaden Ionis Pact, Betting More on RNA Drugs”

Bio Roundup: Lung Cancer Showdown, Gene Therapy Deals, IPO Busts

This weekend, cancer immunotherapy will once again take biopharma’s center stage. At one of the year’s big cancer meetings, investors, researchers, and many others will have a close eye on a group of presentations touting new lung cancer regimens that combine immunotherapies with other treatments. The presentations could shift the dynamics of the fast-moving landscape, … Continue reading “Bio Roundup: Lung Cancer Showdown, Gene Therapy Deals, IPO Busts”

As Novartis Buys In to Gene Therapy, GSK Bows Out with Orchard Deal

Novartis just bet close to $9 billion on the future of gene therapy, a boost for a field that hasn’t yet seen commercial success. In the same week, GlaxoSmithKline, which has one of the few gene therapies on the market but has struggled to sell it, is asking out. The British pharma giant will send … Continue reading “As Novartis Buys In to Gene Therapy, GSK Bows Out with Orchard Deal”

With New Data, Drug Combos, Lung Cancer Experts Scramble to Keep Pace

When oncologist Renato Martins finished his medical training, advanced lung cancer was almost certainly a quick death sentence. “I knew, by name, every patient who had survived two years,” he says. Thanks in large part to the arrival of cancer immunotherapy, things are much different today. While lung cancer remains by far the leading cause … Continue reading “With New Data, Drug Combos, Lung Cancer Experts Scramble to Keep Pace”

Novartis Bets $8.7B on AveXis’s Gene Therapy for Spine Disease

A gene therapy for the rare disease spinal muscular atrophy could hit the U.S. market next year. If it does, the treatment will likely be sold by Novartis, not its developer, AveXis, thanks to a deal announced early this morning. Novartis has agreed to buy AveXis (NASDAQ: [[ticker:AVXS]]) and its experimental SMA gene therapy AVXS-101 … Continue reading “Novartis Bets $8.7B on AveXis’s Gene Therapy for Spine Disease”

Edge to Cut Jobs as Head Injury Drug Flops in Phase 3, Shares Tumble

For years, Edge Therapeutics has been building towards a crucial test of a drug meant to combat the potentially delayed effects of aneurysms and severe head injuries. The plan fell apart this morning: the company will shutter the study and cut its workforce because its experimental treatment, EG-1962, is likely to fail. An independent committee … Continue reading “Edge to Cut Jobs as Head Injury Drug Flops in Phase 3, Shares Tumble”

Racing Allergan, Biohaven Touts Migraine Drug Data, But Shares Sink

Biohaven Pharmaceuticals has become the latest to head to the FDA seeking approval of a new type of migraine drug. But it’s unclear whether the treatment, rimegepant, will stand out amidst a crowd of new medicines aiming to help people fend off the debilitating headaches after they start. Biohaven (NYSE: [[ticker:BHVN]]), of New Haven, CT, … Continue reading “Racing Allergan, Biohaven Touts Migraine Drug Data, But Shares Sink”

Bio Roundup: CMS Backs Cancer Tests, Right to Try Passes, AbbVie Sinks

We’ll start this week’s roundup with several stories from Washington, DC, that could have a lasting effect on public health. The FDA continued its campaign against cigarettes, while the Centers for Medicare & Medicaid Services (CMS) gave a much-needed boost to the developers of cancer diagnostics. In its omnibus spending bill, Congress set aside more … Continue reading “Bio Roundup: CMS Backs Cancer Tests, Right to Try Passes, AbbVie Sinks”

AbbVie Shares Plummet as $5.8B Bet on Stemcentrx Takes a Hit

There is still a chance that AbbVie’s $5.8 billion gamble on acquiring Stemcentrx, a privately held developer of cancer stem cell drugs, could pay off. But the deal’s prospects dimmed this morning with news that Stemcentrx’s lead drug disappointed in clinical testing, sending shares of Chicago-based AbbVie down. AbbVie (NYSE: [[ticker:ABBV]]) said that it won’t … Continue reading “AbbVie Shares Plummet as $5.8B Bet on Stemcentrx Takes a Hit”

Join Xconomy as We Bring Back the NY Biotech Expats on June 21

Here’s a question posed time and again by the those toiling away to build up New York’s biotech scene: What if we could just bring back the biopharma veterans who grew up here? You can call them the New York expats. They started out in Brooklyn, Queens, the Bronx, but like many others before them, … Continue reading “Join Xconomy as We Bring Back the NY Biotech Expats on June 21”

Arena Pharma’s Revamp Gains Traction With New Data For GI Drug

Arena Pharamceuticals’ plan to reshape itself is advancing with new data for an ulcerative colitis drug that could pose a threat to biotech giant Celgene. San Diego, CA-based Arena (NASDAQ: [[ticker:ARNA]]) said late Monday that its experimental compound etrasimod has succeeded in a Phase 2 trial in patients with moderate-to-severe ulcerative colitis, a form of … Continue reading “Arena Pharma’s Revamp Gains Traction With New Data For GI Drug”

CMS Starts to Cover Broad Cancer DNA Tests, Boosting Foundation, Thermo

The Centers for Medicare & Medicaid Services, which administers the federal Medicare insurance program, will begin covering FDA-approved diagnostic tests that scan tumors for a range of genetic mutations. The news is a boost for companies like Foundation Medicine and Thermo Fisher Scientific, who are among the few firms with such tests on the market. … Continue reading “CMS Starts to Cover Broad Cancer DNA Tests, Boosting Foundation, Thermo”

Ionis Sends Nerve Drug to Spinout Akcea as Battle with Alnylam Looms

Ionis Pharmaceuticals hasn’t gone far to find a new home for a therapy that could soon be one of two approved treatments for a rare neurodegenerative disease called transthyretin amyloidosis. It’s handing the drug, known as inotersen, to its own spinout, Akcea Therapeutics. In a deal being announced this morning, Cambridge, MA-based Akcea (NASDAQ: [[ticker:AKCA]]) … Continue reading “Ionis Sends Nerve Drug to Spinout Akcea as Battle with Alnylam Looms”

Solid Discloses More Problems As FDA Halts Duchenne Gene Therapy Study

Solid Biosciences recently completed a big IPO despite disclosing, last-minute, that the FDA had partially suspended a clinical test of its gene therapy for Duchenne muscular dystrophy. Less than two months later, the company has reported a new, and more ominous finding: the FDA has halted testing of the treatment, known as SGT-001, altogether. Cambridge, … Continue reading “Solid Discloses More Problems As FDA Halts Duchenne Gene Therapy Study”

Orexigen’s Weight-Loss Roller Coaster to End With a Bankruptcy Sale

Orexigen Therapeutics has filed for bankruptcy to sell its weight loss pill to the highest bidder. The news follows years of ups and downs for Orexigen, which piled up debt as it struggled to successfully market its obesity drug, known as Contrave. Orexigen (NASDAQ: [[ticker:OREX]]) has filed for Chapter 11 protection, which shields companies from … Continue reading “Orexigen’s Weight-Loss Roller Coaster to End With a Bankruptcy Sale”

Regeneron to Slash Price of Cholesterol Drug After Showing It Saved Lives

Regeneron Pharmaceuticals and partner Sanofi report new data this morning from a large, highly-anticipated trial suggesting that their drug alirocumab (Praluent) may reduce the risk of death for people with high cholesterol who have recently suffered heart attacks or strokes and are at risk of having another one or dying. And in an unusual move, Regeneron—based … Continue reading “Regeneron to Slash Price of Cholesterol Drug After Showing It Saved Lives”

Prevail Gets $75M For Parkinson’s Gene Therapy, Bolstering NY Biotech

A potential gene therapy for Parkinson’s disease has just gotten a big boost thanks to one of the larger Series A investment rounds in years for a New York biotech. The funding, for Prevail Therapeutics, provides another jolt for the city’s life science startup scene. The startup, seeded in New York last year by OrbiMed … Continue reading “Prevail Gets $75M For Parkinson’s Gene Therapy, Bolstering NY Biotech”

Nonprofit Odylia Wants to Resurrect Stranded Gene Therapies

Luk Vandenberghe calls it a “sad irony.” After decades of ups and downs, gene therapy, which offers a long-lasting genetic fix for a disease, has arrived. A product from Spark Therapeutics called Luxturna, for a rare, inherited type of vision loss, recently became the first-ever approved gene therapy in the U.S., adding to two others … Continue reading “Nonprofit Odylia Wants to Resurrect Stranded Gene Therapies”

Paying for Gene Therapy, CRISPR in People, RNAi at “What’s Hot” on May 16

A new age of medicine is underway, and we’re only just beginning to grapple with the implications. The first U.S. gene therapy, for a rare form of vision loss, is now on the market. In 2017, the FDA also approved the first two CAR-T cell therapies for cancer, the first cancer drug based on a … Continue reading “Paying for Gene Therapy, CRISPR in People, RNAi at “What’s Hot” on May 16”

Bio Roundup: Funding Frenzy, Opioid Summit, Celgene Woes & More

Biotech startups rolled in cash this week. By our count, investors put more than $800 million into life-sciences startups in four days. The money came from a wide range of backers, not just traditional biotech venture firms. It’s early yet in 2018, but the current count could put life-sciences companies on pace to bust past … Continue reading “Bio Roundup: Funding Frenzy, Opioid Summit, Celgene Woes & More”

Rubius Rolls Up Another $100M As Bio Funding Frenzy Continues

Like water spewing from a firehose, venture dollars are dousing life-science startups this week. The latest biotech on the receiving end is Rubius Therapeutics, a cell therapy developer that announced a $100 million round this morning. With the new cash, a “crossover” round including investors that back both private and publicly traded companies, Rubius has … Continue reading “Rubius Rolls Up Another $100M As Bio Funding Frenzy Continues”

Activist Investor Scopia Gets Two Board Seats in Deal with Acorda

Acorda Therapeutics has cut a deal with Scopia Capital Management, enabling the activist investor that has previously pushed to sell the company, to name two directors to its board. Scopia will support the board members Acorda (NASDAQ: [[ticker:ACOR]]) nominates at its annual shareholder meeting this year as a part of the agreement. Scopia, which this … Continue reading “Activist Investor Scopia Gets Two Board Seats in Deal with Acorda”

Quentis Nets $48M For Cancer Drugs, Building Buzz For NY Bio Startups

After a string of announcements a few years back, there’s been few new, high-profile biotech startups taking shape in New York. But that quiet period might be coming to an end this morning with the arrival of Quentis Therapeutics, a cancer immunotherapy startup born from the lab of former Weill Cornell Medicine dean and immunologist … Continue reading “Quentis Nets $48M For Cancer Drugs, Building Buzz For NY Bio Startups”

Fresh Off $25M Raise, Generation Adds $100M For Next-Gen Gene Therapy

Today marks another noteworthy moment in the continued advancement of gene therapy, which now has multiple products on the market after decades of research. Generation Bio, one of the startups trying to leapfrog existing versions of the technology, has now raised $125 million in two separate rounds in less than two months. Cambridge, MA-based Generation … Continue reading “Fresh Off $25M Raise, Generation Adds $100M For Next-Gen Gene Therapy”

Continuing Cell Therapy Push, Gilead Aligns with Sangamo for CAR-T Boost

Gilead Sciences has formed a broad alliance with Sangamo Therapeutics this morning, continuing an aggressive push into cellular immunotherapy that began late last year. Gilead (NASDAQ: [[ticker:GILD]]) will pay Sangamo (NASDAQ: [[ticker:SGMO]]) $150 million up front in cash in the deal, through which Gilead will tap Sangamo’s gene editing techniques to try to develop a … Continue reading “Continuing Cell Therapy Push, Gilead Aligns with Sangamo for CAR-T Boost”

Will Fast-Growing Kallyope Stay in New York After New $66M Round?

Kallyope is unusual among biotech startups. Exclusively borne from the work of three Columbia University professors, the company started up in Manhattan, now has more than 40 employees, has gotten $110 million in venture dollars from high-profile investors, and still remains in New York City. Now the big question is: Will Kallyope stay in Manhattan … Continue reading “Will Fast-Growing Kallyope Stay in New York After New $66M Round?”

For Latest Combo, Merck Bets $394M on Viralytics, Cancer-Fighting Viruses

The immuno-oncology combination frenzy continues. Merck this morning agreed to pay $394 million for Viralytics, an Australian developer of oncolytic viruses, which could help boost the power of the pharma giant’s cancer immunotherapy pembrolizumab (Keytruda). The deal gives Kenilworth, NJ-based Merck (NYSE: [[ticker:MRK]]) rights to CVA21 (Cavatak), an experimental oncolytic virus therapy in multiple early- … Continue reading “For Latest Combo, Merck Bets $394M on Viralytics, Cancer-Fighting Viruses”

Wave, Takeda Target RNA Technology at Neuro Diseases With $230M Pact

Wave Life Sciences this morning inked a wide-ranging alliance with Japanese pharma Takeda to develop RNA-based drugs for a variety of neurological diseases. Wave (NASDAQ: [[ticker:WVE]]), of Cambridge, MA, gets $110 million in up front cash from Takeda. The Japanese giant will also buy $60 million in Wave stock at $54.70 a share and fund … Continue reading “Wave, Takeda Target RNA Technology at Neuro Diseases With $230M Pact”

With $69M Deal, AbbVie Turns to Voyager for an Alzheimer’s Gene Therapy

At a time when uncertainty again swirls around the development of Alzheimer’s disease drugs, research continues in new ways. Through an alliance this morning, Voyager Therapeutics will team with AbbVie to try to use gene therapy tools to help attack the disease with a potential one-time, long-lasting treatment. Voyager (NASDAQ: [[ticker:VYGR]]) gets $69 million in … Continue reading “With $69M Deal, AbbVie Turns to Voyager for an Alzheimer’s Gene Therapy”

Mid-FDA Review, Tetraphase Stumbles Again on Latest Phase 3 Setback

Tetraphase Pharmaceuticals’ ups and downs have continued, as new data have clouded the future of the Watertown, MA, company’s experimental antibiotic, eravacycline, once again. Tetraphase (NASDAQ: [[ticker:TTPH]]) reported that eravacycline failed a 1,200-patient Phase 3 study called Ignite3, one of four late-stage trials the company ran to test the antibiotic against either complicated urinary tract … Continue reading “Mid-FDA Review, Tetraphase Stumbles Again on Latest Phase 3 Setback”

FDA’s Rachel Sherman on Right to Try, Succession Trouble & More

The opioid crisis. Right to Try legislation. The first human trials of CRISPR-Cas9 gene editing technology. These are just a few of the many topics at the feet of the FDA this year. Speaking at the BIO CEO & Investor Conference in New York on Tuesday, principal deputy commissioner Rachel Sherman, the FDA’s second in … Continue reading “FDA’s Rachel Sherman on Right to Try, Succession Trouble & More”

Execs Talk Hurdles As Immunotherapy Combos, CAR-T Launches Progress

With multiple products on the market and many more coming, it’s easy to think that cancer immunotherapy has arrived. In reality, however, we’ve only just begun to figure out how to coax the immune system into killing cancer. That’s why a mad dash is on to expand its reach, and a panel of experts invested … Continue reading “Execs Talk Hurdles As Immunotherapy Combos, CAR-T Launches Progress”

Bio Roundup: Lung Cancer Questions, Migraine Races, HIV Battle & More

The top biopharma story of the week involves the fast-moving field of lung cancer, which, as we’ve written previously, has several key trials reading out in 2018. A big domino fell this week: data from a two-drug immunotherapy combination developed by Bristol-Myers Squibb. But the results, touted as positive, left more questions than answers. First, … Continue reading “Bio Roundup: Lung Cancer Questions, Migraine Races, HIV Battle & More”

At Verastem, Infinity’s Former Blood Cancer Drug Heads to FDA

Verastem has filed for FDA approval of duvelisib, a blood cancer drug the Needham, MA, company took a shot on in a 2016 bargain deal with Infinity Pharmaceuticals. Verastem will seek approval of duvelisib as a treatment for patients who have one of three different blood cancers—chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma, or follicular … Continue reading “At Verastem, Infinity’s Former Blood Cancer Drug Heads to FDA”

With New Test, Schadt’s Sema4 Aims to Broaden Scope of Newborn Screening

Sema4, the company that computational geneticist Eric Schadt spun out of the Icahn School of Medicine at Mount Sinai last year, is making a new bet this morning. The New York and Connecticut startup is launching a diagnostic, Sema4 Natalis, meant to go well beyond the typical tests hospitals run on newborn babies to check … Continue reading “With New Test, Schadt’s Sema4 Aims to Broaden Scope of Newborn Screening”

As Migraine Races Heat Up, Allergan Touts New Data for Headache Pill

The push to treat migraines with a new class of medicines took another turn on Tuesday when Allergan presented clinical data for an experimental pill meant to help fend off the debilitating headaches after they start. Allergan’s (NASDAQ: [[ticker:AGN]]) ubrogepant, met its main goals in the first of two Phase 3 studies. In a trial … Continue reading “As Migraine Races Heat Up, Allergan Touts New Data for Headache Pill”

Moderna, Still Private, Scoops Up Yet Another $500M

Moderna Therapeutics, the secretive messenger RNA drugmaker known in part for its preponderance of massive funding raises, has struck once again. The Cambridge, MA, company has closed yet another huge round, a $500 million haul from a wide group of domestic and international investors. New backers include the Abu Dhabi Investment Authority, BB Biotech, and … Continue reading “Moderna, Still Private, Scoops Up Yet Another $500M”

Flagship Brings in New Partners Rosiello, Pontin

Cambridge, MA, venture firm Flagship Pioneering has hired Rob Rosiello and Jason Pontin as new partners. Rosiello, a former McKinsey & Co. executive, was named an executive partner and Flagship’s chief operating officer. Pontin, the longtime editor-in-chief of MIT Technology Review, is now Flagship’s chief editor and a senior partner.

Ex-Merrimack, Seragon Execs’ New Startup Gets $60M, Buys Sanofi Drug

Partner Therapeutics, a Boston startup launched last week by a pair of former Merrimack Pharmaceuticals and Seragon Pharmaceuticals executives, has raised $60 million and acquired an immune system-boosting drug from Sanofi for an undisclosed sum. Partner bought Sanofi’s (NYSE: [[ticker:SNY]]) sargramostim (Leukine), a drug used to help acute myeloid leukemia patients fight infections after undergoing bone … Continue reading “Ex-Merrimack, Seragon Execs’ New Startup Gets $60M, Buys Sanofi Drug”

Bio Buyout Spree Continues as SeaGen Bets $614M on Cascadian Cancer Drug

In the latest in a recent spate of biopharma buyouts, Seattle Genetics has struck a deal to acquire Cascadian Therapeutics, gambling on an experimental breast cancer drug in late-stage testing. SeaGen (NASDAQ: [[ticker:SGEN]]), of Bothell, WA, will pay $10 per share in cash, or $614 million, for Seattle’s Cascadian (NASDAQ: [[ticker:CASC]]) and its lead drug … Continue reading “Bio Buyout Spree Continues as SeaGen Bets $614M on Cascadian Cancer Drug”

After Paper Shakes Sector, Gene Therapy Leader Jim Wilson Talks Safety

[Updated, 1/31/18, see below] Gene therapy pioneer James Wilson and University of Pennsylvania colleagues sounded an alarm Tuesday morning about the use of gene therapy to treat severe diseases like spinal muscular atrophy and Duchenne muscular dystrophy, sending a chill across the sector. Shares of several companies inched downward as word spread about the UPenn … Continue reading “After Paper Shakes Sector, Gene Therapy Leader Jim Wilson Talks Safety”

Sunovion Parkinson’s Drug Heads to FDA, Setting Up Battle With Acorda

Another drug that may help alleviate some of the debilitating symptoms of Parkinson’s disease is on its way to an FDA review, setting up a possible commercial battle between two drug developers, Sunovion Pharmaceuticals and Acorda Therapeutics. Sunovion, of Marlborough, MA, said late Monday that its experimental Parkinson’s drug, APL-130277, succeeded in a 109-patient Phase … Continue reading “Sunovion Parkinson’s Drug Heads to FDA, Setting Up Battle With Acorda”

Solid Discloses Duchenne Problems in IPO & Raises Rival’s Hackles

[Updated, 1/26/18, see below] Solid Biosciences was expected to ride gene therapy’s wave of recent momentum to an IPO this week. But new revelations have clouded the offering, as the company revealed just before going public that its most advanced drug candidate has been under FDA scrutiny since mid-November. The gene therapy, SGT-001, is being … Continue reading “Solid Discloses Duchenne Problems in IPO & Raises Rival’s Hackles”

Have $100M, Looking For Ideas: NYCEDC Begins Search For a NY Bio Campus

If you had $100 million to create an epicenter for New York biotech, what would you do with it? The New York City Economic Development Corp., a quasi-governmental agency supporting job growth in New York, has put that question to the private sector. The NYCEDC has called for proposals asking a “mission-driven organization or joint … Continue reading “Have $100M, Looking For Ideas: NYCEDC Begins Search For a NY Bio Campus”