A new startup sets course against a constellation of rare diseases with a first shot repurposing the SIRP pathway
Despite some big advances in recent years, rare disease remains a mostly uncharted wilderness with many patients left underserved. A startup with galactic ambitions now hopes to bust open a wide range of such diseases, and its first effort has eyes on a familiar cancer target.
Star Therapeutics emerged from stealth Wednesday focused on cracking the code in a constellation of rare diseases with a first spinout company targeting the SIRP pathway for immunology and cancer.
Star, the brainchild of the R&D team behind Sanofi’s recently approved rare disease drug Enjaymo, came together after the acquisition of True North Therapeutics by Bioverativ in 2017, which later fell into Sanofi’s hands.
After handing off the reins to Enjaymo, a complement system therapy for the rare blood condition cold agglutinin disease (CAD), Star CEO and founder Adam Rosenthal said his experience with that drug helped set him off on a mission to explore the untapped potential in rare disease, where a lack of information often leaves patients underserved.
“To me, it was just eye-opening that this information about CAD was unknown, and there were patients living in the shadows with virtually nothing being done by the industry,” he told Endpoints News. “So when I was founding Star, I looked back on that experience and said, ‘How can we replicate this to find these (high unmet) diseases that no one’s focused on?'”
Looking to make the most impact with the fewest possible drugs, Rosenthal’s team in secret began looking for shared pathways among several different diseases, settling first on SIRP, a family of surface receptors on immune cells. The pathway has been a popular one in cancer research, notably the CD47-SIRP alpha pathway, which you might remember as the focal point of Pfizer’s $2.3 billion takeover of Trillium back in August.
But Star reckoned that SIRP could also be a useful target in rare immunology and set to develop a lead drug that could serve as a “pipeline-in-a-product” for a range of conditions.
That work culminated in the launch of Electra Therapeutics, Star’s first spinout that also hit the airwaves Wednesday with $84 million in financing from some big-name biotech blue-chippers led by Westlake Village BioPartners and OrbiMed.
The startup’s lead drug is ELA026, which targets the SIRP pathway to treat a rare inflammatory disease known as secondary hemophagocytic lymphohistiocytosis (sHLH), which is believed to be fatal in roughly 60% of adult patients. The drug is already in Phase I tests there, and Electra is looking for additional indications.
Meanwhile, Electra also has two other preclinical programs in the works, one targeting another rare immunology disorder and the other a potential immuno-oncology platform. That’s an understandable effort given the amount of focus on the SIRP pathway in cancer.
According to Rosenthal, Star’s goal of spinning out companies around its research rather than keeping everything under one roof stems from the desire to dedicate resources and expertise to narrower areas of focus. Keep in mind, there are some 7,000 known rare diseases and only 650 or so approved drugs so the opportunities are vast.
“I think it’s that focus that really allows us to explore the therapeutic potential,” Rosenthal said. Meanwhile, a siloed-off company also makes it easier to court financing and pursue business development opportunities, he said.
Meanwhile, Electra will serve as proof of concept for Star’s grand ambitions, which could get even grander. Rosenthal said Star already has a second spinout queued up with a lead drug in tow. Details on that initiative are slim for now, but will likely look much like Electra in terms of multiple potential indications.