About three years ago fledgling San Diego-based startup PvP Biologics announced a $35 million deal with Takeda Pharmaceutical that gave the Japan pharma giant rights to acquire the company down the line.
That day is here. Takeda (NYSE: TAK) acquired the company for an undisclosed amount up front, plus up to $330 million in payments based on development and regulatory milestones for PvP’s experimental celiac disease drug KUMA062 (now TAK-062). The deal follows completion of a Phase 1 study evaluating the PvP drug.
Data from the trial weren’t made public, but they were compelling enough to get Takeda to follow through on its plan to snap up the company once it demonstrated the investigational therapy’s mechanism of action. (Takeda plans to submit data from the study for presentation at a future medical meeting.)
“Everything worked exactly as it was supposed to,” PvP Bio CEO Adam Simpson told Xconomy on Wednesday. “The idea was that Takeda could do a ‘build to buy’ deal where a little company could run fast and Takeda would feel comfortable with the quality of the work product and what they would be inheriting if they chose to move forward.”
Takeda inherits an experimental drug intended to treat uncontrolled celiac disease, an autoimmune disease in which ingestion of gluten—a group of proteins found in three omnipresent grains, wheat, barley, and rye—causes inflammation and damage in the small intestine.
Takeda’s Asit Parikh, who heads its gastroenterology therapeutics unit, said some people with celiac who strictly avoid gluten—the current standard of care—still experience severe symptoms. (In addition to bread and pasta, gluten is also present in less obvious foods, such as soy sauce, salad dressings that contain malt vinegar, and creamy soups thickened with flour.)
The PvP drug is a form of what the company calls “super glutenase,” a protein that breaks down gluten that has been ingested. Simpson said PvP engineered the enzyme to avoid issues encountered by other developmental glutenase treatments, which, among other challenges, struggled to have the intended impact in the acidic stomach environment.
The idea behind TAK-062 is to degrade the parts of gluten that prompt an immune reaction in people with celiac disease before they leave the stomach and cause symptoms, such as stomach pain, diarrhea, nausea, and vomiting, and intestinal damage. Long-term, the harm caused by celiac disease can lead to malnutrition, accelerated osteoporosis, nervous system issues, and reproductive problems. Takeda plans to evaluate the drug in a Phase 2b trial in patients on a gluten-free diet whose disease remains uncontrolled.
PvP spun out from the University of Washington’s Institute for Protein Design in late 2016 with plans to advance an engineered enzyme that showed promise as an oral celiac disease treatment. At its peak, PvP, had the equivalent of about 15 full-time employees, Simpson said. That included a team that formerly worked together at Meritage Pharma, which Shire (now part of Takeda) acquired in 2015. Tadataka “Tachi” Yamada, a former Takeda executive and venture partner at Frazier Healthcare Partners, served as chairman.
Takeda isn’t betting it all on the PvP drug when it comes to celiac, however. The pharma company has another potential celiac disease treatment in its pipeline: TAK-101, which the company acquired from Cour Pharmaceuticals in October. That investigational medicine works in a different way. TAK-101, which is in mid-stage testing, deploys a nanoparticle loaded with a component of gluten called gliadins to modify the immune system of patients with celiac disease by inducing tolerance to gluten.
Other companies are also working on treatments for celiac disease, which affects about 3 million people in the US and for which no FDA-approved treatments exist. GlaxoSmithKline (NYSE: GSK) in September agreed to buy Sitari Pharmaceuticals, a startup it helped launch as part of an alliance with San Diego life sciences venture capital firm Avalon Ventures, for its preclinical celiac drug.
Provention Bio (NASDAQ: PRVB) is advancing a potential treatment it licensed from Amgen (NASDAQ: AMGN) in late 2018. That drug, a monoclonal antibody, is designed to bind to an immune system protein; Provention plans to test it in a Phase 2b trial in patients who have what’s known as non-responsive celiac disease, characterized by continuing symptoms after months of gluten-free eating.