23:21 , Aug 16, 2019 |  BC Extra  |  Company News

ICER’s DMD recommendations include changes to outcome measures, pricing

After finding that all three DMD therapies it reviewed lack clinical data to support a high price, ICER recommended that manufacturers and payers implement pricing policies better aligned with their value and suggested trial endpoints...
00:25 , Jul 26, 2019 |  BC Extra  |  Company News

July 25 Company Quick Takes: RegenxBio, Neurimmune in neurodegenerative gene therapy deal; plus Lilly, Epizyme, ElevateBio and more

RegenxBio partners with Neurimmune, expands gene therapy pipeline  RegenxBio Inc. (NASDAQ:RGNX) and Neurimmune AG (Zurich, Switzerland) partnered to develop and commercialize gene therapies that use the former's NAV vectors to deliver antibodies to treat neurodegenerative...
20:38 , Jul 3, 2019 |  BC Extra  |  Company News

After negative panel vote, FDA approves Karyopharm's multiple myeloma drug

FDA evidently saw enough data from an ongoing confirmatory trial to grant accelerated approval to Karyopharm's Xpovio selinexor to treat relapsed or refractory multiple myeloma. During the drug's extended review, the new data added to...
00:47 , Jun 29, 2019 |  BioCentury  |  Finance

All eyes on new modality launches, cancer and rare disease data in 3Q19

While the typical summer lull means fewer catalysts in the third quarter, investors will be keeping a close eye on launches of new modality drugs. Of special interest to multiple buysiders is the launch of...
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...
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)....
20:08 , Mar 1, 2019 |  BC Week In Review  |  Company News

Sarepta acquires Myonexus, reports LGMD trial results

Sarepta Therapeutics Inc. (NASDAQ:SRPT) exercised its option to acquire Myonexus Therapeutics Inc. (New Albany, Ohio), giving it a pipeline of five gene therapy candidates to treat forms of limb-girdle muscular dystrophy (LGMD). Sarepta also revealed...