19:58 , May 23, 2019 |  BC Extra  |  Company News

ICER questions price of DMD therapies, reviews MS and depression drugs

Even under its more lax value assessment framework for rare diseases, the Institute for Clinical and Economic Review found that Duchenne muscular dystrophy therapies Emflaza deflazacort, Exondys 51 eteplirsen and golodirsen were not cost-effective. An...
21:58 , Apr 26, 2019 |  BioCentury  |  Product Development

Rise of the independents, biotechs go to market

While product launch and commercialization has largely been the province of big biotech and big pharma, with smaller companies partnering to get their products over the finish line, BioCentury’s analysis of recent FDA approvals finds...
21:34 , Apr 16, 2019 |  BC Extra  |  Clinical News

Wave down on Phase I safety data for DMD therapy

Following a setback last week for its Huntington disease program, Wave lost $9.55 (28%) to $24.47 on Tuesday after the company announced plans to test two lower doses of suvodirsen in a Phase II/III trial...
20:46 , Apr 15, 2019 |  BC Extra  |  Company News

$1.2B Catalent-Paragon deal latest in race to lock up gene therapy manufacturing

The 6x multiple Catalent is paying for gene therapy manufacturer Paragon Bioservices suggests buyers believe gene therapy companies will remain heavily dependent on outsourcing as new therapies are approved in the next few years. Catalent...
00:16 , Apr 6, 2019 |  BioCentury  |  Finance

Room to rally in 2Q

Strong small and mid-cap performance coupled with robust M&A has brought the sector back from 4Q18 correction territory, and chances are good for the momentum to continue given the ample capital ready to be recycled...
00:06 , Apr 6, 2019 |  BioCentury  |  Finance

Big caps under pressure

Biogen’s fall in the wake of its decision to discontinue late-stage trials of Alzheimer’s therapy aducanumab is yet another reminder that large caps are in dire need of new pipeline products, and could serve as...
18:47 , Apr 5, 2019 |  BC Week In Review  |  Clinical News

Sarepta planning NDA for DMD candidate casimersen

Sarepta said it plans to submit an NDA to FDA in mid-2019 for casimersen (SRP-4045) to treat Duchenne muscular dystrophy amenable to exon 45 skipping. The company will seek approval for casimersen based on a...
10:30 , Apr 3, 2019 |  BioCentury  |  Finance

The dynamics of Dyne

Incubated by Atlas Venture and launched Wednesday with a $50 million series A round, Dyne Therapeutics Inc. is using its muscle-targeting platform to develop therapies for rare diseases, beginning with myotonic dystrophy type 1 (DM1)....
23:07 , Mar 25, 2019 |  BC Extra  |  Financial News

Biogen announces $5B stock buyback on heels of Alzheimer’s hit

For the second time in a year, Biogen has announced a share repurchase program on the heels of bad news from an Alzheimer's candidate. This time, the big biotech has authorized the repurchase of $5...
14:01 , Mar 25, 2019 |  BC Extra  |  Company News

Thermo Fisher buying gene therapy manufacturer Brammer Bio

Thermo Fisher will pay $1.7 billion to acquire gene and cell therapy contract development and manufacturing company Brammer Bio, giving it vector manufacturing sites in Massachusetts and Florida. Thermo Fisher Scientific Inc. (NYSE:TMO), which chiefly...