Akashi Therapeutics has been given the FDA go-ahead to restart tests for its Duchenne Muscular Dystrophy (DMD) candidate HT-100 (delayed-release halofuginone), just over a year after the test was suspended after a death.
The Cambridge, MA-based biotech at the start of last year suspended its study after a patient was sent to the hospital, and then a week later the biotech reported that the trial subject has died.
Few extra details emerged, but at an interim point of the study last summer, Akashi (formerly Halo) said that the 10 patients in the early study demonstrated a significant increase in muscle strength after at least 6 months of treatment at a low dose, with no serious adverse events to report.
The drug is being researched for its ability to reduce fibrosis (scarring) and inflammation, while also boosting healthy muscle fiber regeneration in DMD patients. The muscle-wasting condition typically affects young boys and will prove fatal in most cases before they reach their 30's.
The FDA looked into what happened, but today the biotech said the agency old it: “The [FDA] has completed its review and concluded that Akashi may resume clinical development of HT-100.”
“The company plans to initiate the new clinical trial as quickly as possible, and is in discussions with potential investors and development partners regarding clinical development and commercialization of HT-100, as well as DT-200 (selective androgen receptor modulator) and AT-300 (cation channel modulator), all of which are novel, complementary compounds with potential to treat all DMD patients independent of their specific genetic mutation,” the biotech said in a statement.
The patient who died was said by the company to be on a high dose, but its new study will use lower doses across the board, something it believes will help with safety, but also remain effective. The new test will also not use anti-sickness drugs, something that may have been a factor in the trial death last year.
A lot has happened since last year: Two drugs have since been approved for the condition in the U.S., although both came under a controversial cloud.
In September, Sarepta, against the odds (a negative FDA adcomm and with weak data), managed to get its DMD med Exondys 51 (eteplirsen) approved for young boys with a certain genetic mutation, which can treat around 13% of all patients.
Perhaps more controversially, and this has also caused a political blow-back, Marathon got an old steroid approved as a new drug (known as Emflaza) via the FDA for Duchenne, and then promptly sold it to PCT Therapeutics for $140 million-plus.
Akashi’s oral drug is aiming to treat boys regardless of their genetic mutations, and is a small molecule designed to tamp down on inflammation and spur muscle growth.
This could work as part of a future cocktail of remedies for DMD patients, the biotech has previously said.
“We are pleased to learn that the FDA is allowing Akashi to resume the clinical development of HT-100,” said Pat Furlong, founding president and CEO of Parent Project Muscular Dystrophy (PPMD).
“Preventing fibrosis is an important target and an essential piece of a combination of therapies that will be required to end Duchenne. We are pleased to be partnering with Akashi to develop important therapies for Duchenne.” The biotech may also need additional funding for the new tests, and said it was in talks with potential investors.