CAMBRIDGE, Mass.--(BUSINESS WIRE)--Mitobridge, Inc., a pioneer in the discovery and development of products that improve mitochondrial function, today announces a key milestone with the initiation of the first-in-human Phase I trial of its PPAR-delta (PPAR) modulator, MA-0211 (also known as MTB-1). The study will assess the safety, tolerability, pharmacokinetics and pharmacodynamics of MA-0211 in healthy volunteers, which will provide the basis for a trial program in Duchenne Muscular Dystrophy (DMD) patients. MA-0211 is the first clinical compound to emerge from Mitobridges mitochondrial enhancement platform. The PPAR modulator aims to reverse the mitochondrial deficits in DMD, which play a key role in disease progression. DMD is a rare genetic disease caused by loss-of-function mutations in the dystrophin gene. This debilitating fatal disorder affects males and leads to progressive cardiac, skeletal and smooth muscle weakness and eventual loss of muscle mass. Mitobridges research has confirmed and expanded upon previous studies showing that mitochondrial defects contribute to abnormalities in the dystrophic muscle and play a central role in the etiology of DMD. PPAR modulation represents a promising therapeutic approach to improving mitochondrial function and muscle health in DMD patients, stated Mike Patane, CSO of Mitobridge. This milestone with our lead program further validates … Continue reading →

Shares of FibroGen are soaring more than 45 percent in midday trading Tuesday after the biotech reported positive study results for a potential blockbuster drug. The biotech company's drug pamrevlumab, a treatment for fatal lung disease idiopathic pulmonary fibrosis, successfully completed a mid-stage FDA test. "Significant value creation should occur now," Jefferies analyst Michael Yee wrote in a note after the results. The analyst believes the drug should add $3 to $4 billion to FibroGen's market cap eventually or $35 to $50 to its share price. The stock surged as much as 52 percent, or $17.45 a share, to $50.80 in early trading Tuesday. It is on pace for the best daily performance ever since it began trading publicly in November 2014. The stock traded more than twelve times its 30-day average volume, with nearly 5 million shares changing hands on Tuesday. While phase 2 testing for idiopathic pulmonary fibrosis is complete, FibroGen announced it will continue phase 2 testing of pamrevlumab as a treatment for both pancreatic cancer and Duchenne muscular dystrophy. Idiopathic pulmonary fibrosis is a disease which scars lung tissue, leading to organ dysfunction and eventual failure within a few years of diagnosis. FibroGen said in its … Continue reading →

People are always surprised at how faithful my family is. They think that because of my illness we would automatically throwour faith out the window, or that we would blame God for our circumstances. Its quite the opposite actually. Growing up, I wouldnt exactly say we were a family of devout Christians. I knew why we celebrated Christmas, we went to church as often as possible, and I even went to a Christian pre-school. But that was about it. I dont remember learning anything in Sunday school. As I became ill my parents would have to piggyback me upstairs, which was where the Sunday school classroom was. There were no elevators. It was a very impractical situation, so we stopped going. Fast forward a few years and we found ourselves at a new church that some friends attended. Its now our home church. You could say it was a match made in heaven. Every time we went to church I would follow the other kids to Sunday school. Later, I realized I wasnt learning anything worthwhile. I didnt want to learn the specifics of every story in the Bible, or to go over everything, chapter by chapter, verse by verse. … Continue reading →

In Duchenne muscular dystrophy one name has received nearly all the attention: Sarepta Therapeutics (OTCPK:SPHDF), which persuaded the US FDA to approve its Exondys 51 over objections that its supporting data were inadequate. Switzerland's Santhera Pharmaceuticals believes that its entry, Raxone, ought to be receiving at least as much attention. Raxone's Delos clinical trial is "still the only successful phase III trial in Duchene muscular dystrophy", its chief executive, Thomas Meier, notes. So far, it has not resulted in a broad launch, but this could change very soon with an EU advisory committee decision due later this year. This is a bold statement, to be sure, but Mr. Meier follows it up with ambitious commercial plans. "We have taken a position that we are not interested in licensing. We would like to keep the rights," he says, pointing to the example of its fellow Swiss group Actelion as a "prominent success case next door" for going it alone in speciality medicine. Beyond that they are both hoping to enter Duchenne muscular dystrophy (DMD) in quick succession, there are few comparisons between Raxone and Exondys, and they will not compete. The latter seeks to delay the disability requiring the use of … Continue reading →

Elena Silva gripped her cellphone, struggling to convey a sense of urgency to her husband, Brian Woodward, whose response was drowned out by the background din of a suburban Maryland swimming pool on a steamy July afternoon. You have to bring the kids here right now, Silva remembers insisting. She believed that the couples long-running quest for a diagnosis of their daughter Gabriela, known as Gg (pronounced Gigi), then 7, hinged on the little girls presence. Woodward had taken Gg and her older brother Elian to the pool while Silva, hoping to meet researchers and network with knowledgeable parents, was attending a 2014 meeting at the Clinical Center, the research hospital on the grounds of the National Institutes of Health, a few miles from the familys home. Once a year, specialized neurology researchers and families affected by a constellation of rare neuromuscular disorders get together, a confab that is both supportive and aimed at furthering research. Silva, who had learned of the event only that morning, was mingling with participants when a parent-advocate asked, Wheres your daughter? Why isnt she here? When Silva replied that Gg was at the pool, the woman advised Silva to get the little girl to … Continue reading →