Apic Bio, a privately held developer of gene therapies for rare disorders, has agreed to sell the rights to one of its experimental ALS treatments to the biotechnology company UniQure in a deal announced Tuesday.
Per deal terms, UniQure will pay $10 million up front for worldwide rights to the treatment, which was called APB-102 but will now be known as AMT-162. Apic could later be eligible for up to $45 million in additional payments should its therapy secure approval in the U.S. and Europe and hit certain sales goals.
The Food and Drug Administration previously cleared AMT-162 for human testing, and UniQure plans to start a Phase 1/2 trial in the back half of this year.
ALS, or amyotrophic lateral sclerosis, is a fatal neurodegenerative disorder that affects roughly 30,000 people in the U.S. While a majority of cases are sporadic, meaning their cause isn’t fully understood, it’s estimated that about 5%-10% are genetic, with one of the most common drivers being mutations in a gene called SOD1.
SOD1 has been at the center of ALS research efforts for decades. Normally, this gene provides helpful functions to cells. But it can mutate, and when it does, it’s been linked to ALS. Apic specifically designed AMT-162 to be a one-time treatment that blocks the expression of SOD1, with the goal of slowing down or reversing the progression of ALS in patients with genetic mutations.
Most gene therapies currently in development use a group of viruses known as adeno-associated viruses, or AAVs, to shuttle their therapeutic cargo into cells. With its therapy, Apic used a recombinant AAV that expresses an “miRNA” — a micro ribonucleic acid — directed at SOD1.
For UniQure, the new licensing deal offers diversification. The company has already been working on another therapy, AMT-161, which targets a different gene tied to ALS.
AMT-162 “provides uniQure with another clinical stage program that is strategically aligned with our current pipeline and highly complementary with our AMT-161 program,” said Ricardo Dolmetsch, president of research and development at uniQure.
“Together, these ALS gene therapy candidates have the potential to address most familial forms of ALS and transform the lives of thousands of patients around the world suffering from this devastating disease,” Dolmetsch added.
Joseph Schwartz, an analyst at SVB Securities, echoed some of those sentiments in a Jan. 31 note to clients, writing that his team views AMT-162 as a "good strategic fit" for UniQure's pipeline, especially given the company's "expertise with miRNA gene therapy development through [its] ... Huntington's disease program."
And while ALS is "largely viewed as a high risk/reward space for drug development, we appreciate that [UniQure] is pursuing genetic-targeted approaches," Schwartz wrote.
Currently, the FDA has approved just three medicines for the treatment of ALS, and healthcare providers note that the effects of each of them are modest.
The most recent addition, Relyvrio, was developed by Amylyx Pharmaceuticals and cleared for market in September. The clinical trial supporting its approval showed that patients treated with Relyvrio appeared to have slower functional decline than those given a placebo. They also lived a median of five months longer.
Typically, ALS patients live just two to five years after being diagnosed.