Exclusive: BrainStorm will not provide ALS therapy under U.S. Right to Try act

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HEALTH NEWS
JUNE 26, 2018
Tova Cohen


TEL AVIV (Reuters) - BrainStorm Cell Therapeutics Inc (BCLI.O) will not make its experimental stem cell treatment for neurodegenerative diseases available under the new U.S. “Right to Try” (RTT) act, citing a failure to find a way to enable all patients to afford the treatment.

The U.S.-Israeli company said on Tuesday it will focus on completing its advanced study for NurOwn in patients with amyotrophic lateral sclerosis (ALS) as quickly as possible.

U.S. Congress last month approved the RTT bill that would allow terminally ill patients to try experimental treatments not yet approved by the Food and Drug Administration. The act, which was approved by the Senate in August, was signed in to law by President Donald Trump on May 30.

The bill does not require insurance companies to pay for the experimental treatments.

BrainStorm said four elements must be satisfied in order to be able to provide NurOwn to ALS patients under the RTT pathway.

It found solutions for three of them: treating a limited number of patients at clinical trial-experienced sites for safety reasons, ensuring patients are educated on the benefits and risks, and limiting inclusion to patients who do not meet the clinical trial criteria to avoid impacting the trial.

The fourth: finding alternative funding for patients who are unable to afford the high cost of cell therapies, was unmet.

“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,” BrainStorm CEO Chaim Lebovits said.

Lebovits has said that the Right to Try act cannot be only for the rich.

BrainStorm could not estimate what the cost of NurOwn will be after commercialisation. Personal cell and gene therapies can cost hundreds of thousands of dollars a year.

Lebovits told Reuters that BrainStorm would provide NurOwn under RTT to one patient, Matt Bellina, a naval aviator diagnosed with ALS who helped lead the fight to get the legislation passed. Lebovits said he would personally sponsor the treatment.

BrainStorm expects to have Phase III trial results at the end of 2019 or early 2020.

According to the ALS Association, 5,600 people in the United States are diagnosed each year with the illness, also known as Lou Gehrig’s Disease.

Reporting by Tova Cohen, editing by Stephen Farrell and Louise Heavens

 

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JUN 7, 2018
BRAINSTORM CONSIDERING RIGHT TO TRY FOR ALS THERAPY
BY STEVE USDIN

BrainStorm Cell Therapeutics Inc. (NASDAQ:BCLI) is considering offering NurOwn to treat amyotrophic lateral sclerosis under the recently enacted federal right-to-try law, BrainStorm President and CEO Chaim Lebovits told BioCentury.

He also discussed the company’s plans on a conference call Thursday with over 200 patients, caregivers and investors. While BrainStorm hasn’t made a final decision about providing NurOwn under right to try, Lebovits made it clear that he hopes to do so if legal, ethical and business challenges can be resolved. “I think you can hear between the lines where our heart is,” he said.

Responding to the wife of an ALS patient on the conference call, Lebovits said: “I understand you have no time. Patients currently have no time to wait for the end of Phase III. We understand that.”

He told BioCentury that BrainStorm “will make a decision very soon, the pressure is too high to wait long.”

NurOwn consists of autologous mesenchymal stromal cells induced to differentiate into neurotrophic factor-secreting cells.

In a double-blind, placebo-controlled, U.S. Phase II trial in 48 ALS patients, a single treatment of NurOwn was well tolerated with no treatment-related serious adverse events reported. At week 12 posttreatment, 40% of patients receiving NurOwn experienced an improvement in ALS Functional Rating Scale (ALS-FRS) slope of ≥50% from baseline vs. 17% of patients receiving placebo. In a subgroup of 23 rapidly progressing ALS patients, 94% of patients receiving NurOwn achieved a 100% improvement in ALS-FRS slope at week two vs. 20% of patients receiving placebo (p=0.0027). At week 24, 22% of patients in the subgroup receiving NurOwn achieved a 100% improvement in ALS-FRS slope vs. 0% of patients receiving placebo.

BrainStorm is conducting a U.S. Phase III trial of NurOwn in 200 ALS patients ages 18 to 60. The company received a $15.9 million grant from the California Institute for Regenerative Medicine (CIRM) to conduct the trial. BrainStorm’s trial is the only Phase III trial that is actively enrolling ALS patients in the U.S., according to ClinicalTrials.gov.

BrainStorm has received a “flood” of requests for access to NurOwn under right to try, Lebovits told BioCentury. Several patients and caregivers who said on the conference call that they were anxious to obtain NurOwn reported that they were ineligible for the Phase III trial because they were older than 60, the cut-off date for inclusion.

Lebovits told BioCentury and patients on the conference call that BrainStorm will not provide access under right to try if doing so compromises its ability to complete the Phase III trial or submit a BLA.

He also said that if it provides NurOwn under right to try, BrainStorm will charge for the therapy. The law does not restrict companies’ abilities to charge for unapproved therapies.

Lebovits declined to estimate the cost for NurOwn beyond saying that cost of manufacturing and delivering the therapy is similar to CAR T cancer therapies. Yescarta, a CAR T therapy from the Kite Pharma Inc. unit of Gilead Sciences Inc., has a wholesale acquisition cost (WAC) of $373,000.

Insurers are unlikely to cover the cost of an unapproved therapy, and BrainStorm is grappling with the ethics of pricing and access, Lebovits told BioCentury. “Will it be right to try for the rich only? Or if the poor can’t afford it, should the rich die? This is a very, very hard decision,” he said.

On the conference call, he said BrainStorm “won’t go forward [with providing access under right to try] unless we have at least a partial answer to those who cannot afford it.”

BrainStorm has received over 1,500 requests to provide NurOwn to ALS patients under right to try or under a pending application to offer it in Israel under the country’s hospital exemption regulation, Lebovits said on the call. He told BioCentury the company could potentially offer NurOwn under right to try to 40 or 50 patients per year.

BrainStorm is consulting ethicists, patients and clinicians to determine which patients would be given access if the company decides to make it available under right to try, Lebovits told BioCentury.

NurOwn used in clinical trials is being manufactured by the City of Hope, Lebovits said. City of Hope would not manufacture NurOwn for distribution under right to try, and BrainStorm is exploring the potential for manufacturing the product at other sites, he said on the call.

BrainStorm “has not heard from FDA” about providing NurOwn under right to try, Lebovits told BioCentury. He said the company hopes to organize a round table discussion with FDA, manufacturers of drugs for other conditions who are considering right to try, and legislators.