AVI tanks after positive PhIIb fails to improve outcomes for DMD

Posted under AVI Biopharma, Blog, Clinical Trials, Companies, Diagnostics, DMD, Duchenne muscular dystrophy, dystrophin, eteplirsen, Funding, Medical Devices, Medical Supply, Pharmaceuticals, Pipeline, Prosensa, PTC Therapeutics, RNA, Startups, Universities, Videos by john

Looking to make a comeback, Bothell, WA-based AVI Biopharma--an RNA company--heralded the news that its lead therapy hit the primary endpoint in a Phase IIb study, increasing levels of dystrophin in a tiny trial for Duchenne muscular dystrophy. But when investors took a closer look and saw that the boys did not experience a significant improvement in walking ability, AVI's shares ($AVII) plunged about 25%.

Investigators noted that the small group of boys treated with eteplirsen benefited from a spike in levels of the protein, which is associated with muscle function. Raising levels of dystrophin is a key target for a number of developers trying to treat DMD, based on the logical theory that if you improve dystrophin levels a developer can have a positive impact on symptoms of the rare disease. AVI's drug uses an exon-skipping approach to skip exon 51 in the dystrophin gene, restoring its function. And the investigators noted the improvement of dystrophin after 24 weeks of treatment.

"This study represents a major advance in the field of DMD research as the results indicate that eteplirsen is producing consistent levels of dystrophin, which is the essential protein that these patients need," said principal investigator Jerry Mendell. "We anticipate that these levels of dystrophin could lead to significant clinical benefit if maintained over a longer course of treatment."

But clinicians didn't see a comparable improvement in the 6-minute walk test used to gauge the expected improvement. The company added that "Performance on the 6-minute walk test and other outcome measures were generally stable across most of the patients, including the placebo patients, suggesting that a longer period of observation will be required to demonstrate clinical effects of eteplirsen versus a placebo control." 

Prosensa is also developing an exon 51 skipping approach to DMD. And PTC Therapeutics has experienced its own problems in the DMD field.

Late last fall AVI announced a restructuring that cost the jobs of 28% of its workforce. The restructuring was brought about as CEO Chris Garabedian responded to a string of setbacks. It also hasn't helped that the RNA field in general has been operating under a cloud in recent years, as Big Pharma dropped some high-profile partnerships. Garabedian's strategy is closely focused on the successful development of eteplirsen, with other programs in place for Ebola and the Marburg virus.

- read the press release
- here's the story from Reuters

Related Articles:
Investigators herald antisense breakthrough on muscular dystrophy
NEA turns to Europe in $30M round for RNA-drug developer Prosensa
Two families facing DMD launch their own biotech

Biogen Idec strikes $299M deal for Isis antisense drug

Posted under Biogen Idec, Blog, Companies, Diagnostics, Funding, Isis Pharmaceuticals, Medical Devices, Medical Supply, Novartis, Pharmaceuticals, PTC Therapeutics, rare diseases, Roche, spinal muscular atrophy, Startups, Universities, Videos by John Carroll

Joining a race of pharma giants to treat rare cases of spinal muscular atrophy, Biogen Idec ($BIIB) has committed up to $299 million to grab rights to an antisense drug program in the pipeline at Isis ($ISIS). In the pact, Biogen will pay Isis $29 million upfront and up to $45 million in milestones for an option to a $225 million licensing pact on the treatment. And Isis says the partners are on track to a potential regulatory approval once proof-of-concept data arrive.

"This alliance is consistent with our business strategy to develop antisense drugs to proof-of-concept with a knowledgeable partner that is committed to supporting the rapid development of the drug," said Isis CEO Stanley T. Crooke. "Given the severity of the unmet need in SMA, our proof-of-concept studies should also serve as the registrational trials for ISIS-SMNRx. We believe that, together with Biogen Idec, we will be able to expeditiously develop this investigational drug in hopes of bringing to market an effective and desperately needed treatment to improve the lives of children with SMA."

Bloomberg's Robert Langreth believes that the deal gives Biogen Idec the inside track on a new treatment for the lethal condition, which afflicts some 9,000 people inside the U.S. Just weeks ago Roche ($RHHBY) committed $490 million to partner with PTC Therapeutics on its program for spinal muscular atrophy. And Novartis ($NVS) is also busy with its own program, another sign of the growing popularity of new treatments for rare conditions.

"This has the potential of being truly transformative, if the preclinical data are in any way reflected in the outcome of clinical studies," Columbia's Darryl De Vivo, an Isis consultant, tells Bloomberg.

- here's the press release
- read the Bloomberg story

Related Articles:
Isis bags $1.53B rare disease development deal with GSK
Roche, PTC strike potential $490M deal for genetic disease program
Biomarker study to aid clinical trial of Spinal Muscular Atrophy drug
Hedge fund operator proffers $100M to find a drug for his daughter

Roche, PTC strike potential $490M deal for genetic disease program

Posted under Blog, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, Pharmaceuticals, PTC Therapeutics, rare diseases, Roche, Spinal Muscular Atrophy Foundation, Startups, Universities, Videos by Ryan McBride

Swiss drug giant Roche has bought exclusive rights to PTC Therapeutics' program for spinal muscular atrophy (SMA) for $30 million upfront, providing PTC with another major pharma collaboration to fuel development of its pipeline. The deal, which offers up to $460 million in future payments to PTC, gives the South Plainfield, NJ-based developer a boost after suffering the setback of Genzyme walking away from PTC's troubled rare disease program in September.

Roche, which previously partnered with PTC in 2009, now has exclusive global rights to three preclinical compounds in development and backups against SMA. The companies say there are no good drugs for the disease, which is caused by a missing or mutated SMN1 gene and leads to lower levels of the survival motor neuron protein. It weakens muscles, interferes with key survival functions, and often claims the lives of its victims during childhood. PTC initially developed the program with the SMA Foundation, which will stay on as part of the collaboration with Roche.

"We found the science behind this program very compelling, with the potential to help treat a currently incurable condition," Luca Santarelli, global head of Roche Neuroscience, said in a statement. "This is the essence of Roche's entire strategy, focused on solid science and high unmet clinical need, and these compounds bolster our rich pipeline in central nervous system diseases."

PTC has been able to find larger drugmakers such as Roche and AstraZeneca ($AZN) to help take its programs forward, a sound strategy as funding remains tight for private biotech outfits. The new Roche deal, of course, is heavily back-ended, and PTC will have to achieve development and commercial goals to collect the bulk of the money. If the program succeeds, PTC is also in line for double-digit royalties on product sales.

- here's the release
- read this Bloomberg article

Related Articles:
Genzyme gives back drug rights to PTC Therapeutics
AZ financial backing to fuel PTC oncology cures