With $20B to Spend, Sanofi, Celgene Bet Big on Hemophilia, CAR-T

Two top drugmakers, Sanofi and Celgene, agreed on Monday morning to shell out more than $20 billion combined, cinching deals for hemophilia drug maker Bioverativ and cell therapy developer Juno Therapeutics. Each agreement represents an expensive, risky bet on a crowded, rapidly changing field. In one acquisition, Sanofi is buying Waltham, MA-based Bioverativ (NASDAQ: [[ticker:BIVV]]), … Continue reading “With $20B to Spend, Sanofi, Celgene Bet Big on Hemophilia, CAR-T”

Why Spark and Watchdog ICER Don’t See Eye-to-Eye on $850K Gene Therapy

The first gene therapy approved in the U.S. costs $850,000. Announcing the price on Jan. 3, its owner Spark Therapeutics held out the possibility of some relief, such as installment payments, or slim rebates if the drug, a one-time shot into each eye to reduce or reverse inherited vision loss, doesn’t work or wears off. … Continue reading “Why Spark and Watchdog ICER Don’t See Eye-to-Eye on $850K Gene Therapy”

Bio Roundup: Spark’s Road Ahead, Juno Rumors, Merck’s Big Day & More

The most overblown health story of the week was President Trump’s health exam. America learned Trump is in perfect health and has “great genes.” He takes Propecia. He doesn’t have heart problems, had a perfect cognitive test score, and is one pound shy of obese. This scintillating information led to a new social media phenomenon, the … Continue reading “Bio Roundup: Spark’s Road Ahead, Juno Rumors, Merck’s Big Day & More”

Merck Is Latest to Tout Success as Lung Cancer Drug Combos Plow Forward

This morning has brought another positive development in the rapidly changing treatment landscape for lung cancer. Merck reported that a combination regimen utilizing its immunotherapy drug, pembrolizumab (Keytruda), has succeeded in a confirmational Phase 3 study. Merck (NYSE: [[ticker:MRK]]) said that a trial in which 614 patients just diagnosed with non-small cell lung cancer got … Continue reading “Merck Is Latest to Tout Success as Lung Cancer Drug Combos Plow Forward”

Spark CEO Marrazzo on Gene Therapy Pricing & Paving the Way for the Field

[Updated, 1/13/17, 11:15 a.m. ET]  After decades of research and scientific ups and downs, the first U.S. gene therapy, a single, long-lasting treatment called voretigene neparvovec (Luxturna) for rare, inherited blindness, is now on the market. But the commercial challenge facing its developer, Spark Therapeutics, is daunting. Can Spark lead the way in showing that … Continue reading “Spark CEO Marrazzo on Gene Therapy Pricing & Paving the Way for the Field”

Notes From the JPM ’18 Vortex: Gene Therapy’s Reckoning and Lonely Cabs

The annual J.P. Morgan Healthcare conference in San Francisco winds down today, meaning the remaining stragglers are either presenting to half-empty rooms or headed back to colder climes. Compared to a 2017 full of scientific milestones—FDA approvals of gene and cell therapies, a clinical victory for RNA interference, and more—the meeting itself was rather dull. … Continue reading “Notes From the JPM ’18 Vortex: Gene Therapy’s Reckoning and Lonely Cabs”

Gene Therapy 2.0: New Startups Aim to Fine-Tune Gene Delivery and Control

This past month brought a major milestone for gene therapy, a cutting edge type of medicine meant to permanently alter a patient’s genes to treat disease. For the first time, a gene therapy is available in the U.S., adding to other treatments previously approved in Europe. More are likely on the way, ushering in a … Continue reading “Gene Therapy 2.0: New Startups Aim to Fine-Tune Gene Delivery and Control”

Roche Bets $1.7B More on Precision Oncology With Ignyta Deal

In a buyout that marks the latest endorsement for targeted cancer drugs—and, potentially, the increasing utility of broad cancer DNA tests—Roche this morning agreed to acquire San Diego biotech Ignyta in an all-cash deal valued at $1.7 billion. Roche will pay $27 per share for Ignyta (NASDAQ: [[ticker:RXDX]]), a whopping 74 percent premium to the … Continue reading “Roche Bets $1.7B More on Precision Oncology With Ignyta Deal”

Stop Being an “Initiative”: Highlights From NY Biotech—The Future Is Now

Vicki Sato, a longtime Boston biotech entrepreneur, laid out a challenge for New York life sciences in 2018. “Stop being an initiative,” she said. “Start being a player.” Sato, addressing the throng of attendees at Xconomy’s “New York Biotech: The Future Is Now” conference last week, has skin in the game. She’s now co-chairing an … Continue reading “Stop Being an “Initiative”: Highlights From NY Biotech—The Future Is Now”

Two Months Ahead of Schedule, FDA Clears Aerie Glaucoma Drug

The FDA has just approved a new medicine for glaucoma, a leading cause of blindness that affects close to 3 million Americans. Now the question is how eye doctors will use the drug, a treatment from Aerie Pharmaceuticals known as netarsudil (Rhopressa), and whether payers will cover the treatment. In a statement late Monday, Aerie … Continue reading “Two Months Ahead of Schedule, FDA Clears Aerie Glaucoma Drug”

Intellia Switches CEOs as EVP Leonard Steps in for Bermingham

Intellia Therapeutics (NASDAQ: [[ticker:NTLA]]) said that John Leonard, currently the company’s executive vice president of research and development, will become its president and CEO, effective Jan. 1. Leonard will step in for founding CEO Nessan Bermingham, who is returning to the venture capital industry, according to a prepared statement. Prior to running Intellia, Bermingham had … Continue reading “Intellia Switches CEOs as EVP Leonard Steps in for Bermingham”

In Week of Hemophilia News, Alnylam Drug Cleared to Move Forward

The FDA has cleared Alnylam Pharmaceuticals to resume testing of an experimental hemophilia drug after instituting a variety of measures meant to manage the possible risks of taking the therapy, known as fitusiran. Cambridge, MA-based Alnylam (NASDAQ: [[ticker:ALNY]]) can restart the Phase 3 study and Phase 2 open-label extension trials of fitusiran, a drug being … Continue reading “In Week of Hemophilia News, Alnylam Drug Cleared to Move Forward”

Gene Therapy Advances, But Hemophilia Is No Easy Target

Mark Skinner, the former longtime president of the World Federation of Hemophilia, has had the blood disease on his mind his entire life. He doesn’t have a choice. Skinner, 57, was born with a severe form of hemophilia A, meaning he has a tiny fraction of the necessary cellular machinery to clot blood. His disease … Continue reading “Gene Therapy Advances, But Hemophilia Is No Easy Target”

Bio Roundup: Trials to Watch, Mega Deals, Video Game Therapy & More

With 2018 around the corner, this was a week to look ahead. At Xconomy, we zeroed in on several clinical trials that could become major stories in the life sciences. Other healthcare milestones are on the way, too. The FDA will consider for the first time whether a video game should be approved as medicine. … Continue reading “Bio Roundup: Trials to Watch, Mega Deals, Video Game Therapy & More”

Sage Shares Soar Again As Depression Drug Hits Mark in Phase 2 Test

Sage Therapeutics disclosed another clinical trial victory this morning, sending shares of the Cambridge, MA, company to new highs. An experimental Sage drug for major depressive disorder (MDD), a psychiatric disease that affects millions of Americans, has succeeded in a Phase 2 study and will now head into late-stage testing. Sage (NASDAQ: [[ticker:SAGE]]) said that … Continue reading “Sage Shares Soar Again As Depression Drug Hits Mark in Phase 2 Test”

Last Chance to Have Your Voice Heard on NY Biotech’s Course in 2018

Want to take part in an interactive, constructive talk about what can be done, right now, to alter the course of the life sciences scene in New York? You’ve still got a shot. Next Monday evening, Dec. 11, at the Alexandria Center for Life Science, Xconomy is gathering a small group of New York biotech … Continue reading “Last Chance to Have Your Voice Heard on NY Biotech’s Course in 2018”

Gilman’s Next CEO Trick: Taming CAR-T Cells With Obsidian

Genetically modified medicine is here. Two CAR-T therapies, made from a patient’s living T cells, are on the market in the U.S., and many more are in various stages of clinical testing. As remarkable as they might seem, however, these medicines are still fairly crude, with no way to control them once inside a patient’s … Continue reading “Gilman’s Next CEO Trick: Taming CAR-T Cells With Obsidian”

First Video Game Therapy Heads to FDA as Akili Touts ADHD Study Win

[Updated 12/4/17, 4:48 pm, with CEO comments.] For the first time ever, a mobile video game used to assess and treat a disease is headed for an FDA review. Akili Interactive Labs, a Boston and San Francisco Bay Area company created by PureTech Health, said Monday that AKL-T01, an experimental video game it has been … Continue reading “First Video Game Therapy Heads to FDA as Akili Touts ADHD Study Win”

15 For ’18: Key Clinical Data to Watch For Next Year (Part 1)

Many of biotech’s biggest stories in 2017 followed the highly anticipated data from clinical studies. There were monumental successes, like the first-ever approval of a genetically modified living cell therapy, the first FDA application for a gene therapy or a medicine using RNA interference. There were also stinging failures, such as the latest in a long … Continue reading “15 For ’18: Key Clinical Data to Watch For Next Year (Part 1)”

Will Foundation’s FDA, CMS Nod Help Cancer DNA Tests Break Through?

In what could be an important step forward for the mainstream acceptance of broad cancer genetic tests, a product from Foundation Medicine has simultaneously won FDA approval and gotten a coverage determination from the Centers for Medicare & Medicaid Services. Now the question is whether the ruling—the first decision of its kind—will help lead to … Continue reading “Will Foundation’s FDA, CMS Nod Help Cancer DNA Tests Break Through?”

With Good Data From Rival Migraine Drugs, Doctors Foresee Price Headaches

Amgen (NASDAQ: [[ticker:AMGN]]) and Teva Pharmaceutical (NYSE: [[ticker:TEVA]]) this afternoon are providing the most detailed look yet at two drugs in an emerging class of medicines meant to prevent migraine headaches, rather than just quell their symptoms. While the data for the two drugs, called erenumab (Aimovig, from Amgen and partner Novartis) and fremanezumab (from … Continue reading “With Good Data From Rival Migraine Drugs, Doctors Foresee Price Headaches”

VCs Put More Dollars Into Exosomes as Codiak Bio Lands $76M

Codiak BioSciences, the startup run by former Biogen (NASDAQ: [[ticker:BIIB]]) research chief Doug Williams, has rung up its third big round of cash in two years. The Cambridge, MA, company has closed a $76.5 million Series C round from a large group of investors, among them founding backers Flagship Pioneering and Arch Venture Partners, and … Continue reading “VCs Put More Dollars Into Exosomes as Codiak Bio Lands $76M”

As AMD Race Tightens, Regeneron’s Second Eye Drug Combo Falls Short

Another attempt to use a combination of drugs to improve upon the current treatments for age-related macular degeneration, a leading cause of vision loss in the elderly, has failed. The news is a setback for Tarrytown, NY-based Regeneron Pharmaceuticals (NASDAQ: [[ticker:REGN]]), which is trying to fend off the competition for its most successful drug, a … Continue reading “As AMD Race Tightens, Regeneron’s Second Eye Drug Combo Falls Short”

Biogen Finds Clancy’s Replacement, Names Capello New CFO

Biogen (NASDAQ: [[ticker:BIIB]]) has named Jeffrey Capello its new executive vice president and chief financial officer. Capello, a former Boston Scientific (NYSE: [[ticker:BSX]]), Ortho Clinical Diagnostics, and PerkinElmer (NYSE: [[ticker:PKI]]) executive, will start on December 11. Biogen has been searching for a CFO ever since Paul Clancy, who spent more than 16 years at the company, … Continue reading “Biogen Finds Clancy’s Replacement, Names Capello New CFO”

What Steps Should NY Biotech Take in 2018? Share Your View on Dec. 11

Change takes time. Particularly in the life sciences, where a single drug’s journey from idea to market can consist of more than a decade of small, yet critical steps. For New York, then, aspirations to become a region known for its biotech industry are—and always have been—a long game in which every year and bit … Continue reading “What Steps Should NY Biotech Take in 2018? Share Your View on Dec. 11”

Lacking Details, Roche Touts Drug Combo’s Success in Lung Cancer

[Corrected 10:15 a.m., 11/20/17. See below.] Roche’s Genentech division reported this morning that its cancer immunotherapy drug, atezolizumab (Tecentriq), has succeeded in a clinical study that could help change the standard of care for people with a certain type of lung cancer. The South San Francisco, CA-based Genentech said late Sunday night that in a … Continue reading “Lacking Details, Roche Touts Drug Combo’s Success in Lung Cancer”

Ed Kaye on Sarepta’s Duchenne Quest, Rising to CEO & His New Startup

[Updated, 2:45 pm ET, see below] Ed Kaye never thought he’d run a biotech —until waking up one day in 2015 realizing not only that he could, but that Sarepta Therapeutics, where he was chief medical officer at the time, may fall apart if he didn’t. In March 2015, Kaye (pictured) was one of many … Continue reading “Ed Kaye on Sarepta’s Duchenne Quest, Rising to CEO & His New Startup”

Adams Pivots From Advisory Role, Takes on Head Seat At Gamida Cell

Julian Adams, the former Infinity Pharmaceuticals R&D chief who famously helped shepherd along the multiple myeloma drug bortezomb (Velcade), has re-emerged as a full-time senior executive at a biotech company. Adams has been named the chairman and CEO of Jerusalem-based immunotherapy developer Gamida Cell, one of the portfolio companies of Israeli venture firm Clal Biotechnology … Continue reading “Adams Pivots From Advisory Role, Takes on Head Seat At Gamida Cell”

Acorda Takes Another Hit, Reports Patient Deaths in Parkinson’s Study

Acorda Therapeutics’ rebuilding plan just suffered another setback, leaving the fate of one of the experimental Parkinson’s disease drugs that is closest to market unclear. Ardsley, NY-based Acorda (NASDAQ: [[ticker:ACOR]]) said Wednesday morning that it has seen troubling safety problems in a Phase 3 trial of its experimental Parkinson’s disease drug tozadenant. Acorda reported cases … Continue reading “Acorda Takes Another Hit, Reports Patient Deaths in Parkinson’s Study”

Inozyme Gets $49M From Sanofi, Novo to Tackle Rare Infant Disease

Sanofi and Novo Nordisk this morning have put their venture arms to work. The two joined up with two venture firms to back a $49 million round for a fledgling startup, Inozyme Pharmaceuticals. The company aims to develop a therapy for an ultra-rare infant disorder—and possibly some more prevalent diseases too. The startup, based in … Continue reading “Inozyme Gets $49M From Sanofi, Novo to Tackle Rare Infant Disease”

ImmusanT Gets $40M From Arch, Vatera to Continue Celiac Vaccine Quest

ImmusanT has spent years developing an immunotherapy meant to help people with celiac disease tolerate gluten. Today, on the verge of its most substantial clinical test to date, the company has also come up with its biggest round of funding from some well-known backers. Arch Venture Partners has joined previous ImmusanT backer Vatera Healthcare Partners … Continue reading “ImmusanT Gets $40M From Arch, Vatera to Continue Celiac Vaccine Quest”

In a Precision Step, Bayer Writes Loxo $400M Check for Two Cancer Drugs

The latest evidence of the progress of targeted cancer drugs came this morning in the form of a $400 million check. That’s what Bayer will pay, up front, to Loxo Oncology, a company that has the chance to be the second, following Merck, to win approval of a drug for tumors with a specific genetic … Continue reading “In a Precision Step, Bayer Writes Loxo $400M Check for Two Cancer Drugs”

Bio Roundup: Barron Joins GSK, Sage Bounces Back, Valeant Dumps Addyi

Call it a week of second chances. Multiple biotechs breathed new life into drugs sorely in need of it. Sage Therapeutics proved a drug that had failed a trial a rare form of epilepsy could be the first ever approved therapy for postpartum depression. Otonomy, reviving a hearing loss drug thought to be on the … Continue reading “Bio Roundup: Barron Joins GSK, Sage Bounces Back, Valeant Dumps Addyi”

Sage Bounces Back, Eyes FDA Filing for Postpartum Drug

There may be a new treatment on the horizon for postpartum depression, an often severe form of depression that affects women after childbirth. Sage Therapeutics reported this morning that an experimental neurological drug known as brexanolone succeeded in two Phase 3 trials, setting the stage for an FDA approval filing next year. Cambridge, MA-based Sage … Continue reading “Sage Bounces Back, Eyes FDA Filing for Postpartum Drug”

With $40M for Exonics, Duchenne’s Gene Therapy, CRISPR Race Heats Up

Exonics Therapeutics, one of the players in an emerging race to use gene therapy or gene editing techniques to treat Duchenne muscular dystrophy, has just landed a big round of funding to take its first treatment into clinical testing. The Cambridge, MA, company, seeded last year by a subsidiary of the nonprofit group CureDuchenne, will … Continue reading “With $40M for Exonics, Duchenne’s Gene Therapy, CRISPR Race Heats Up”

FDA Lifts Hold on Cellectis’s “Off the Shelf” CAR-T, With Caveats

The FDA has cleared Cellectis to resume testing an experimental “off the shelf” type of cell therapy, so long as it follows a new set of measures meant to quell the safety concerns that caused the company to halt a pair of clinical trials. Cellectis (NASDAQ: [[ticker:CLLS]]) said late Monday night that the agency will … Continue reading “FDA Lifts Hold on Cellectis’s “Off the Shelf” CAR-T, With Caveats”

Spero, Allena Nab $152M As Bio IPOs, With Insider Help, Continue Roll

Two biotechs, Spero Therapeutics and Allena Therapeutics, are headed for the Nasdaq this morning at valuations they’d been aiming for. Their stock offerings are the latest sign of the health of the biotech IPO market, and the willingness of insiders to make sure these deals succeed. Spero, of Cambridge, MA, raised $77 million late Wednesday … Continue reading “Spero, Allena Nab $152M As Bio IPOs, With Insider Help, Continue Roll”

KSQ Bets on CRISPR-Juiced Drug Discovery With $76M & Ex-Genzyme CEO

CRISPR-Cas9 makes headlines as a gene editing tool for potential therapies, but pharma and biotech are also using it to as a tool to discover new drugs. A high-powered startup, KSQ Therapeutics, is the latest firm to join their ranks. Flagship Pioneering and Polaris Partners have been incubating the Cambridge, MA, company since 2015. KSQ … Continue reading “KSQ Bets on CRISPR-Juiced Drug Discovery With $76M & Ex-Genzyme CEO”

FDA Panel Pans PTC’s Audacious Plan to Force Vote on Duchenne Drug

A panel of experts has voted against a Duchenne muscular dystrophy drug from PTC Therapeutics (NASDAQ: [[ticker:PTCT]]). The vote is a critical blow to the company’s unusual attempt to force the review of a drug the FDA has rejected twice already. The 11 member committee, one of many that help the FDA evaluate drugs for … Continue reading “FDA Panel Pans PTC’s Audacious Plan to Force Vote on Duchenne Drug”

Amid Cash Crunch, Genocea to Dump Herpes Vaccine, Focus on Cancer Work

Genocea Biosciences was able to go from startup to publicly traded company, in part, because of the early progress it made developing an experimental vaccine for genital herpes. Yet the Cambridge, MA, company couldn’t get the financial help to take that vaccine, GEN-003, through its final test. Instead it has shifted gears and will try … Continue reading “Amid Cash Crunch, Genocea to Dump Herpes Vaccine, Focus on Cancer Work”

SpringWorks Spins Out of Pfizer With $103M to Give Shelved Drugs New Life

Pfizer is testing out a new biotech experiment this morning. It has taken four drug compounds gathering dust on its shelves, and funneled them into a new startup, SpringWorks Therapeutics, that will try to develop them for diseases that don’t fit the hulking New York pharma giant’s core strategy. This isn’t a small experiment. SpringWorks, … Continue reading “SpringWorks Spins Out of Pfizer With $103M to Give Shelved Drugs New Life”

Bio Roundup: RNAi’s Big Day, CAR-T For Kids, Drugs From Fungi & More

It’s been a year of biomedical milestones in the U.S., including the first approval of a CAR-T cellular immunotherapy, the first smartphone app to treat substance abuse, and the first approval of a cancer drug based on genetic signature instead of the tumor’s organ of origin. Add to the list the first successful Phase 3 … Continue reading “Bio Roundup: RNAi’s Big Day, CAR-T For Kids, Drugs From Fungi & More”

Alnylam Data Puts a Nobel Discovery One Step Closer to Approved Drug

[Updated with comments at 9:49 am ET, see below] For the first time ever, a medicine using RNA interference, a method of muting a gene before it can make a harmful protein, is headed for an FDA review. If the agency grants a commercial license, it would be a major victory not just for the … Continue reading “Alnylam Data Puts a Nobel Discovery One Step Closer to Approved Drug”

Race to Mine Fungi for Drugs Revs up as Verdine’s LifeMine Gets $55M

Can the life sciences industry finally begin to efficiently and systematically exploit the potential treasure trove of drugs hidden in fungi? It seems promising, thanks to cheaper DNA sequencing technologies, artificial intelligence, new academic research, and now the largest funding yet for a startup trying to take on the task. This morning, LifeMine Therapeutics, a … Continue reading “Race to Mine Fungi for Drugs Revs up as Verdine’s LifeMine Gets $55M”

Ex-Cubist CEO Bonney Emerges at Kaleido, Flagship’s Latest Microbiome Startup

Flagship Pioneering has been as active as any venture firm at funneling its investment dollars into microbiome drug developers. It’s putting some more of that cash to work as of this morning, unveiling a startup, Kaleido Biosciences, that has been incubated within its walls for two years and is run by a prominent former Boston-area … Continue reading “Ex-Cubist CEO Bonney Emerges at Kaleido, Flagship’s Latest Microbiome Startup”

After Biogen Deal Fizzles, Rodin Bags $27M And Heads To First Trial

An option-to-buy deal, when one company agrees to buy another on prearranged terms after a series of milestones are met, doesn’t always lead to an acquisition. Sometimes, as with a 2016 deal between Biogen and Rodin Therapeutics, the arrangement fizzles and the smaller company charts a new course. Rodin, a Cambridge, MA, startup trying to … Continue reading “After Biogen Deal Fizzles, Rodin Bags $27M And Heads To First Trial”

Moderna Ends “Venture” Experiment, Dissolves Four Startups

Moderna Therapeutics, the high-flying private company with a $1 billion war chest, is reversing course. Three years ago, it decided to hatch startups as wholly-owned subsidiaries, all working on different aspects of its unproven biomedical technology called messenger RNA. Moderna announced today that it is ending the experiment. It is dissolving the four startups, or … Continue reading “Moderna Ends “Venture” Experiment, Dissolves Four Startups”

Amicus’s Scioderm Deal Flops as Skin Drug Fails in Phase 3

Epidermolysis bullosa is a rare, debilitating skin disease with no approved treatments, and unfortunately, that isn’t going to change anytime soon. Amicus Therapeutics reports this morning that an experimental drug it has been developing for the condition failed a Phase 3 trial. The Cranbury, NJ, company won’t invest in future studies. Amicus (NASDAQ: [[ticker:FOLD]]) said … Continue reading “Amicus’s Scioderm Deal Flops as Skin Drug Fails in Phase 3”

Stealthy NY Startup Rocket Pharma Merges With Inotek, Heads to Nasdaq

Rocket Pharmaceuticals, a stealthy gene therapy startup in New York City, has made its first splash. Through a merger with struggling Inotek Pharmaceuticals (NASDAQ: [[ticker:ITEK]]), Rocket, a company developing treatments for a variety of rare blood diseases, has taken itself public. Rocket shareholders are expected to own 81 percent of the combined company, with Inotek … Continue reading “Stealthy NY Startup Rocket Pharma Merges With Inotek, Heads to Nasdaq”

All Eyes on Depression Trials as Sage Neuro Drug Flunks Epilepsy Test

Sage Therapeutics this morning reported that its most advanced clinical prospect, a neurological drug known as brexanolone, failed its first big test. Sage (NASDAQ: [[ticker:SAGE]]), of Cambridge MA, said the drug didn’t do much better than a placebo in a Phase 3 trial of patients with a rare, life-threatening form of epilepsy called super refractory … Continue reading “All Eyes on Depression Trials as Sage Neuro Drug Flunks Epilepsy Test”