Tag Archives: epidermolysis bullosa

Bio Roundup: Clinical Trials Paused, New MS Drug, Kallyope’s Cash & More

Finding and enrolling patients for clinical trials is always challenging. The coronavirus pandemic has made it nearly impossible. Fallout from the spread of COVID-19 infections led several companies to declare this week that they would stop enrolling patients in clinical trials or postpone studies that have not yet started. Eli Lilly (NYSE: LLY) said that […]

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Bio Roundup: Sarepta’s Approval, Merck M&A, Drug Price Bill & More

If you’re keeping track, FDA drug approvals have been on a tear in the past month with several decisions coming well ahead of their targeted dates. And then there’s Sarepta Therapeutics. Late Thursday, the FDA announced approval of the Cambridge, MA, biotech’s drug for Duchenne muscular dystrophy in patients who have a specific mutation. The […]

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Biotech Roundup: Vaping Toll Rises, NASH News, Akcea Shakeup & More

Lung injuries linked to electronic cigarettes have topped 800 cases, including 12 deaths, according to the Centers for Disease Control and Prevention. Public health officials aren’t sure whether the injuries are from legally sold products made by companies such as Juul, or from black-market items such as those that allow users to “vape” the active […]

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Bio Roundup: Merger Drama, FDA Trauma, Big IPOs, CRISPR Fights & More

It was a busy week. If we had to choose a theme, it was all about getting together. Two huge drug companies, AbbVie and Allergan, said they’d be better as one. Two more huge drug companies, Celgene and Bristol-Myers Squibb, learned they would have to leave a big product behind if they want to merge. […]

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Amicus Therapeutics’ Pompe Drug Data Show Improved Muscle Movement

[Updated 10/5/17, 3:35 pm. See below.] Amicus Therapeutics has its sights set on a second approved drug after reporting that its Pompe disease treatment helped patients reduce the muscle-wasting effects of the genetic disease. The results were from a small, 20-patient study, however, and the Amicus drug will have to pass muster in a larger […]

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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: FOLD) said […]

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East Coast Biotech Roundup: X4, Rgenix, Alnylam, Intellia & More

What summer doldrums? It’s still not even Labor Day, yet the deals are already coming fast and furious. So fire up the grill and read on for some Series A rounds, crossover investments, data readouts, option-to-buy deals and more. —As Xconomy reported this week, a stealthy Cambridge, MA-based startup called X4 Pharmaceuticals raised a $37.5 […]

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With $229M Deal, Amicus Gets a New Drug And Perhaps a Voucher

Just how far has Amicus Therapeutics come in two years? In 2013, the Cranbury, NJ-based company was reeling from a trial failure and restructuring to conserve cash. Now, it’s eyeing its first drug approval and buying a startup in a nine-figure deal. Amicus (NASDAQ: FOLD) this morning is announcing the acquisition of Durham, NC-based Scioderm. […]

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