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Clinical studies for DMD drug, stopped for inefficiency reasons

  • by Daniel Arthur
  • 29 Days ago
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Pfizer Inc. announced it is terminating clinical studies for domagrozumab (PF-06252616), which was evaluated for efficacy and safety in the possible treatment of DMD. Termination was not decided for safety reasons, the company stated, but because the drug proved to be inefficient.

The clinical trial was stopped after one year of the two it was supposed to last, after data analysis revealed unsatisfactory results.

All subjects received either domagrozumab or a placebo, but all were given corticosteroids as background therapy.

Company officials thanked participants and their families, while assuring everyone they would keep on researching DMD. “We are disappointed by these results and while we are not progressing with the studies, the data will contribute to a greater understanding of this disease and we will evaluate the total data set to see if there is a place for this medicine in muscular diseases,” said Seng Cheng, PhD, senior vice president and chief scientific officer, Pfizer Rare Disease Research Unit. “We are extremely grateful to all those involved with this trial, especially the boys who participated, and their families.”

Duchenne muscular dystrophy (DMD) is a rare genetic disease, which affects 1 in every 3.500 to 5.000 male newborns each year. It can also affect girls, but that is a rarer occurrence.

Progressive muscle degeneration is a very serious disease, leading to weakness and a shortened life span–the main characteristics of the disease.  It also causes disabling myocardiopathy and respiratory failure. This debilitating illness has no known cure, so big pharma companies, advocacy associations and patients’ groups are trying to find a cure together.

Despite Pfizer’s decision, many biotech companies are interested in DMD research.  Some have turned to gene therapies, which could prove beneficial to the patients, in the long run. Finding treatments for patients who currently don’t have any remains the company’s purpose; Pfizer won’t give up researching DMD and other rare neuromuscular diseases.

They have been collaborating closely with advocacy associations and patients groups in order to help each other, in searching for ways to advance the research needed to find treatments for these debilitating diseases.

Since Pfizer will try to see whether the drug can be used in treating other muscular dystrophies, repurposing the drug, something Healx, for instance, also does, there is still hope for other rare disease patients. Biotech, AI and healthcare go hand-in-hand to find solutions for the future or, in this case for patients to even have a future.

Even if Pfizer failed in this case, pharma companies and drug discovery  represent an emerging business. For example, in a recent interview, Simon Sterzer, chairman for Biocardia Inc discussed how the company he co-founded uses stem cells to treat heart failure resulting from a heart attack. However, as Simon Sterzer also mentioned in a recent podcast, pharma companies may still have a long way to go as investments in this field are not without risk or limitations.

Overall, the health tech industry is booming and healthcare startups are turning a new leaf in attracting new funds in 2018. According to Healthcare Weekly, the top ten pharma healthcare startups have received a staggering $1.7 billion dollars in funding over the last year.

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