A week after President Donald Trump signed the right-to-try bill into law, biotechnology companies are staking out their positions amid looming pressure.
The Trickett Wendler, Frank Mongiello, Jordan McLinn and Matthew Bellina Right To Try Act, or S.204, signed May 30 by the president, opens the door for terminally ill patients to request access to experimental, early-stage therapies outside the realm of clinical trials.
For supporters, it gives dying patients hope and the possibility of life-changing treatment; for detractors, it puts pressure on drugmakers to supply untested treatments in uncertain circumstances.
On the sidelines of the annual BIO International Convention in Boston, company executives discussed the path forward as the law becomes a reality.
"We would never use this new pathway," Alnylam Pharmaceuticals Inc. CEO John Maraganore said in an interview.
Alnylam, which is developing experimental therapies targeting RNA interference, suffered a setback last year after a patient died in a phase 2 trial for its hemophilia therapy, fitusiran. The Right To Try Act allows patients to request for therapies that have cleared the phase 1 clinical trial stage.
"We strongly believe in bringing our medicines to patients when we have confidence around their efficacy and safety," Maraganore, who is also this year's BIO board chair, said.
The biotech's partner Sanofi is now leading fitusiran's development program, while Alnylam expects to launch patisiran, a medicine treating hereditary liver disorder, in the U.S. and Europe later this year.
There have never been any regulatory impediments for Alnylam to get experimental therapies to patients, Maraganore said — a sentiment echoed by several executives who spoke with S&P Global Market Intelligence at the annual event.
The U.S. Food and Drug Administration's compassionate-use program, also known as expanded access, was the traditional approach to get experimental medicines to desperate patients. Under the program, doctors made a case for their patients and demonstrated that they had exhausted all available options. The ultimate decision was taken by the FDA. In 2016, 99% of those requests were approved, according to an agency report.
"If you have a good doctor, a willing patient and a willing company, you can get a drug in the U.S. right now," said Brian Schwartz, chief medical officer and head of research and development for ArQule Inc., a biomarker-targeting biopharmaceutical company focused on oncology and certain rare diseases.
ArQule's miransertib AKT, an experimental medicine in early clinical trial for treating the extremely rare disease Proteus — also known as Elephant Man disease — easily falls into the new law's focus area.
A quarter of people with the disease — marked by unhindered cell growth resulting in overgrown bone and tissue, as well as cancers and thrombosis — will die before they are 18 years old, according to Schwartz.
Overall, the Massachusetts-based company receives about two compassionate-use requests a month for its pipeline therapies, Schwartz said — and the FDA has yet to deny one.
The 'heart and head' debate
Yet many Americans feel they are not getting the options.
As he signed the bill into law, Trump mentioned a man suffering from late-stage amyotrophic lateral sclerosis, or ALS — a neurodegenerative disorder also known as Lou Gehrig's disease.
The patient planned to travel to Israel for an experimental treatment called NurOwn — a stem cell therapy in late stage trials — being developed by Brainstorm Cell Therapeutics Inc.
Brainstorm President and CEO Chaim Lebovits attended the bill signing.
Now, the pressure is on, Lebovits said in an interview.
Brainstorm scheduled a call with ALS patients, physicians and advocates on June 7 to weigh options and develop a path forward.
"There are two sides of the debate about this with no simple answer," Lebovits said a day before the call, adding that broadly, companies were worried about fueling false hope.
"It's the heart against the head. I'm afraid I'm going to go with the heart, but I don't know," he said.
Breaking down business
Drugmakers are protected from liability for right-to-try patients, but not necessarily shielded from other fallouts if a drug does not work, or harms a patient.
Bioasis Technologies Inc. is a small, Connecticut-based company led by executives with big pharma background and looking to transport drugs through the blood-brain barrier with a preclinical technology.
The therapy's success would open a door for treating terminal tumors such as glioblastoma — the illness that Sen. John McCain, R-Ariz., has been diagnosed with — and late-stage cancers that had spread to other parts of the body and had few to no therapy options left.
But the company is not rushing it, executives said.
"Right To Try makes me nervous, because if the drugs are given improperly, not controlled, or given to the wrong patients … that can shut down an entire program," Caroline Hill, senior vice president of R&D operations, said. "So, the millions of people that it could have helped will never have it now."
Hill came to the company from oncology giant Bristol-Myers Squibb Co., along with Bioasis' president and CEO Mark Day.
"Big pharma does get a bad rap for not approving every compassionate-use request, but that's why [they don't approve them]," Hill said.
One potential advantage for Bioasis is that while its technology is new, its cargo across the blood-brain barrier is familiar: the company is running trials with versions of Roche Holding AG's Herceptin and Avastin — two blockbuster cancer therapies with extensive clinical data.
"With these being well-established products, it could be a good thing for us and patients," Day said.
In a fireside chat on the last day of BIO, FDA Commissioner Scot Gottlieb said that the agency was formulating a process for handling requests under the Right To Try Act.