Maze Therapeutics has found a new buyer for its Pompe disease candidate, five months after scrutiny from the Federal Trade Commission led Sanofi to back out of a deal for the clinical-stage drug.
The new licensee is Shionogi, the companies said Friday morning. The Osaka, Japan-based pharma will dish out $150 million upfront and pay an undisclosed amount in biobucks. Almost a year ago to the day, Maze disclosed that Sanofi would dole out the same amount upfront for the oral candidate and could pay another $600 million in milestones.
But in December, the FTC said it would look to block the deal since Sanofi markets the Pompe drug Nexviazyme, a successor to its own earlier treatments Myozyme/Lumizyme. The regulator said the deal would “would eliminate a nascent competitor poised to challenge Sanofi’s monopoly in the Pompe disease therapy market.”
Sanofi decided to axe its deal with Maze instead of facing the US government in court. Maze never received money from the Sanofi deal, a spokesperson for the startup confirmed to Endpoints News.
This time, the Maze-Shionogi deal has already been completed before it’s announced. The 30-day waiting period under the Hart-Scott-Rodino Act has expired, the companies said.
If successful, MZE001 could become the first oral drug for patients with Pompe, a rare genetic disorder that leads to weakness and wasting in heart and skeletal muscles.
The small molecule went through a Phase 1 healthy volunteer trial in 2022. Unlike Sanofi’s treatments, this drug is not an enzyme replacement therapy. Instead, it works to tamp down on GYS1, a gene tied to glycogen production, and block the buildup of glycogen from happening in the first place.
Aro Biotherapeutics is also in the clinic with a candidate that goes after GYS1. The Philadelphia startup’s ABX1100, an IV infused treatment, is a CD71 Centyrin – GYS1 siRNA conjugate. It entered Phase 1 last October.
“The science behind MZE001 is differentiated and promising, and we look forward to developing the compound as both mono and add-on therapy to enzyme replacement therapies,” Shionogi CEO Isao Teshirogi said in a statement.
Maze’s future
With a new deal now in place, Maze’s focus and next steps become clearer.
Speaking to Endpoints News on the sidelines of January’s annual JP Morgan Healthcare Conference, CEO Jason Coloma said the company received a “lot of inbound interest” after the Sanofi pact was terminated.
Once Maze provided an update on the Pompe program, he said at the time, it would “have a better sort of guidance” on its next financing steps, which could be an initial public offering, he added.
Maze last disclosed a $190 million round in January 2022. Third Rock Ventures launched the startup in 2019, and the biotech has raised more than $400 million as a private company, according to venture data firm PitchBook. Matrix Capital Management Company is the lead investor and additional backers include General Catalyst, a16z, Woodline Partners, Casdin Capital, Foresite Capital and others.
Beyond Pompe, the biotech is also working on small molecules in chronic kidney disease with a Phase 1 underway in healthy volunteers. The oral small molecule, dubbed MZE829, is being explored as a potential treatment for APOL1 kidney disease.
Meanwhile, Maze is looking to partner its non-small molecule neuroscience programs by the end of 2024, Coloma said in the January interview. That includes a gene therapy for amyotrophic lateral sclerosis and an antisense oligonucleotide for the progressive neurodegenerative disease.
Maze will provide an update on the rest of its portfolio later this year, a spokesperson said.
The company has also lined up joint ventures in the past, including ophthalmology-focused Broadwing Bio and cardiovascular-centered Contour Therapeutics. The latter was a JV with BridgeBio that has since been unwound, Coloma previously told Endpoints.
Editor’s note: This story was updated to include information from a Maze spokesperson.