Drug Development

Brainstorm backs off a controversial plan to sell its experimental ALS med, but this issue is far from over

Brainstorm Cell Therapeutics $BCLI won’t be angling to make any profits off the new ‘Right to Try’ bill after all.

After stirring or hornet’s nest of angry Twitter memes after Bloomberg quoted CEO Chaim Lebovits saying that they were preparing to sell their drug to desperate ALS patients, including one who lent his name to the legislation, the micro-cap biotech this morning is backing off the idea.

Lebovits was thinking in terms of charging around $300,000 for the experimental med, which insurers were unlikely to back.

“As we are unable to identify a practical funding solution, we, unfortunately, are not in a position to initiate access to NurOwn under RTT at this time,” said Lebovits in a statement.

The hubbub over the suggestion, though, brightened the already intense spotlight on Right to Try, where lawmakers backed a proposal — pushed hard by President Donald Trump — that gives patients the right to ask for meds still in the clinic. There’s been some debate on this point, but Brainstorm believes that the bill’s provisions allows for a “modest” profit.

Brainstorm has made progress with its stem cell therapy for ALS, but it has a long way to go in Phase III — and not much in the way of hard data to prove it works.

If you look at the 24-week chart the company offered on the 48 patients in the small, exploratory study, the drug outperformed the placebo arm but any relative treatment effect they were tracking quickly evaporated for most of the patients suffering from this slow, lethal ailment. The p-values were a mess. The investigators involved said that if you excluded the slow progressors, the numbers looked better — but the same transient effect is evident, with negative p-values.

Brainstorm’s overnight fame may fade fast now, but don’t expect the issue to go away. That’s just begun.

Image: Chaim Lebovits (YouTube)

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Sr. Manager, Regulatory Affairs, CMC
CytomX Therapeutics San Francisco, CA
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