Brainstorm Cell Therapeutics Inc. will not make its experimental stem cell therapy broadly available to patients with amyotrophic lateral sclerosis under the Right to Try Act, saying it did not want the pathway open only to the rich.

The company, however, will permit one patient with ALS, Matthew Bellina — one of the people for whom the Right To Try Act was named — to use the product under the pathway.

The new law, signed into law May 30, permits Americans with life-threatening diseases or conditions to bypass the Food and Drug Administration's expanded-access program, also called compassionate use, which allows regulators to help terminally ill patients obtain and use experimental therapies outside of the clinical trial process.

Some groups have called the law "dangerous" and said it would open up greater potential for "snake oil" salesmen to take advantage of desperate patients.

A firestorm erupted last week after Bloomberg News reported that BrainStorm may potentially charge patients more than $300,000 for NurOwn, which is made by extracting a person's own stem cells from their bone marrow.

But on a June 26 conference call with reporters and analysts, BrainStorm President and CEO Chaim Lebovits denied ever citing that amount.

"We decided at the time not to rebuff it and I don't really want to comment on this," he said. "It's premature to give a price to our treatment at this time."

Last week, Lebovits told S&P Global Market Intelligence he was not ready to talk about the cost of its product, but said if NurOwn is approved it would likely be priced on par with other therapies in its class.

Preventing drugmakers from getting rich

The Right To Try Act contains a provision aimed at ensuring that drugmakers are not able to get rich off of their treatments.

Like the FDA's expanded-access process, the manufacturer of an investigational drug provided under Right to Try may recover only the direct costs of making the product available — those incurred by a drugmaker that can be "specifically and exclusively attributed to providing" the medicine for investigational use, such as manufacturing and shipping costs.

The manufacturing costs could include raw materials, labor, and nonreusable supplies and equipment used to make the quantity needed.

The direct costs could also include acquiring the experimental medicine from another manufacturing source.

On the call, Lebovits said it was "heartbreaking not to currently be able to provide access to more patients" beyond Bellina, who he said the company "couldn't see ourselves denying access."

But he said that after the company had carefully evaluated the ethical, legal and practical aspects of the Right to Try law and received detailed feedback from patients, clinicians, regulatory and legal experts, it decided it would not make the product available to others at this time.

"Should we move forward, it would only be on the condition that this would not be a right to try for the rich only," Lebovits said.

He said it must be a "ight to try for the rich and the poor."

Ethical framework

Lebovits said the company had established an ethical and practical framework with four key elements that must be satisfied in order to be able to provide NurOwn to ALS patients under the Right to Try pathway: Providing the treatment to a limited number of patients at clinical trial-experienced sites to ensure the necessary expertise and systems of care are in place to safely and effectively deliver the therapy; ensuring that patients are fully educated on the benefits and risks of the investigational therapy; limiting the inclusion to patients who do not meet the phase 3 clinical trial inclusion criteria to ensure that the Right to Try pathway does not adversely impact the clinical trial; and finding alternative funding solutions for patients who are unable to afford the high cost of regenerative medicine cell therapies.

BrainStorm said it has found practical solutions for all except the funding issue.

Focusing on late-stage study

Lebovits said his company would continue to focus its efforts on completing the ongoing NurOwn phase 3 study.

Ralph Kern, chief operating officer and chief medical officer, said all investigating cites were actively enrolling, and many study participants had received their second or third doses of NurOwn.

"All study procedures are being performed without any issues, and we are on track to meet our study milestones," Kern said on the call.

BrainStorm is expecting top-line data by the end of 2019 or early 2020, he said.

The company expects to convene the first data safety monitoring board in August to review the data from the phase 3 study, Kern added.