Halo takes the virtual approach to developing a DMD treatment

Posted under Blog, Clinical Trials, Companies, Diagnostics, Duchenne muscular dystrophy, Funding, Halo Therapeutics, HT-100, Medical Devices, Medical Supply, Pharmaceuticals, Phase II, Pipeline, Startups, Universities, Videos by john

Halo Therapeutics may be the most virtual biotech company on the threshold of a mid-stage study. CEO Marc Blaustein counts himself as the only full-timer, supported by a cast of about 17 people who help support the development of a single program: HT-100 for Duchenne muscular dystrophy.

So far, Halo has been financed by two DMD patient organizations: Charley's Fund and the Nash Avery Foundation. They've reviewed the animal studies, the clinical results when Israel's Collgard had it in clinical studies for bladder cancer, the scientific literature on the treatment's anti-fibrotic properties and recently gained an endorsement of the science from an influential group of independent neuromuscular experts. Halo now wants to test its treatment in a mid-stage study of about 80 DMD patients. And Blaustein is scouting for around $8 million in industry money to fund the Phase II.

"Our preference is a strategic partner who can share our vision and move at the pace we can move," says Blaustein. "As a virtual company we have a core team of outstanding, experienced developers with DMD expertise." And Halo has done it with "no physical infrastructure" in place.

Duchenne muscular dystrophy is one of those orphan diseases that fits the classic portrait of an unmet medical need. There are no treatments, aside from corticosteroids. It appears early in boys, cripples them as youths and then kills them as young men.

DMD also has a clear trigger: A mutation in the dystrophin gene that blocks production of the protein needed to support muscle tissue development. And several companies--ranging from the Dutch biotech Prosensa, to AVI in Seattle and PTC Therapeutics, have developed experimental therapies intended to at least partially mend the genetic condition to trigger a surge in dystrophin production.   

"When boys miss this protein their muscles become much more susceptible to damage,' says Blaustein. Physically, they endure a vicious cycle, with an inflammatory response that damages muscles further as well as a fibrotic effect that disables the regeneration of healthy muscles. "HT-100 tips that balance back," he says, with an anti-inflammatory, anti-fibrosis approach that spurs muscle regeneration.

Importantly, this is not a genetic fix. Halo sees HT-100 as a potential monotherapy as well as a possible addition to a combination therapy with one of the other treatments now in development. 

It's certainly not an easy disease to tackle, though. Last fall Genzyme returned most of its rights to PTC's ataluren. Just days ago AVI announced that its mid-stage data demonstrated an increase in the production of dystrophin, but its stock took a beating as investors reacted to the absence of efficacy seen among patients in the study. 

Blaustein wants to get the mid-stage trial designed and underway in the second half of this year.

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

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NEA turns to Europe in $30M round for RNA-drug developer Prosensa

Posted under Blog, Companies, Diagnostics, Duchenne muscular dystrophy, Funding, GlaxoSmithKline, Medical Devices, Medical Supply, New Enterprise Associates, Pharmaceuticals, Prosensa, RNA, Startups, Universities, Videos by John Carroll

The Dutch biotech Prosensa has rounded up 23 million euros--about $30 million--in a new round earmarked to back its work on RNA drug development for Duchenne muscular dystrophy and other rare diseases. New Enterprise Associates led the fundraising round, spearheading its first investment in Europe and signaling its interest in more global deals. NEA joined Abingworth, Life Sciences Partners, Gimv, Idinvest Partners and MedSciences Capital.

"Over the past few years, we have made substantial progress in our research and development pipeline," says Prosensa CEO Hans Schikan. "Our lead drug candidate for Duchenne muscular dystrophy is in Phase III clinical trials in partnership with GlaxoSmithKline. We have advanced the development of five additional compounds in DMD and have announced preclinical testing for a compound for DM1. This financing will help us to further strengthen our position in rare diseases and will allow us to deliver on our promise of accelerated development of treatments for patients in need."

The round marks an encouraging advance for RNA, a development field which has been plagued by some major league exits in the past two years. In Prosensa's case investigators have been focused on a novel approach to correcting the mutation in the dystrophin gene that triggers DMD. They're using antisense oligonucleotides to induce exon skipping, essentially jumping past the genetic alterations associated with the disease. GlaxoSmithKline ($GSK) partnered with Prosensa back in 2009, pledging up to $680 million in their pact.

"While NEA is one of the largest venture firms in the world, with more than $11 billion in capital under management, Prosensa is our first biopharma investment in Europe and signals our openness to selectively consider investing in truly exceptional innovation in Europe as well as in our traditional geographies in the U.S., China and India," says David Mott, the general partner for NEA, which is managing $11 billion.

- here's the press release

Related Article:
Glaxo forges $680M RNA pact with Prosensa

Two families facing DMD launch their own biotech

Posted under Blog, Companies, Diagnostics, Duchenne muscular dystrophy, Funding, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, Videos by John Carroll

A severe illness would test any family. For the Secklers and Wicka families in the U.S., their sons' development of Duchenne muscular dystrophy has inspired a heroic attempt to develop a new treatment. The Wall Street Journal has the story, which tracks their decision to buy an experimental DMD drug from an Israeli biotech and set up a virtual company under the guidance of an industry veteran.

Dyax vet Marc Blaustein now runs Halo Therapeutics out of his living room. "We want to quickly determine if this is a good drug for Duchenne muscular dystrophy," Blaustein tells the Journal. And they're out raising $8 million to get the treatment through a mid-stage study.

They may face the same tough odds that anyone in biotechnology has to reckon with. But with patient advocacy groups receptive to the idea of helping fund the next stage of development, the two families have a shot at reaching the proof-of-concept stage.

- here's the story from the WSJ