A few weeks ago, Johnson & Johnson nabbed FDA fast-track status for nipocalimab in fetal and neonatal alloimmune thrombocytopenia (FNAIT). Now, the Big Pharma is making a combined cash and equity investment of over $7 million in Rallybio in order to get the biggest benefits from both companies' programs centered on this rare blood disorder.
Under the deal, rare disease biotech Rallybio will receive an upfront $500,000 payment from J&J to raise awareness about the pharma's FNAIT therapy program as the biotech conducts a natural history study for RLYB212, a human monoclonal anti-HPA-1a antibody. RLYB212 is designed to prevent pregnant individuals from alloimmunizing—meaning it stops a pregnant person's immune system from attacking fetal platelets and therefore reduces the risk of severe bleeding complications for the baby.
Rallybio will also be eligible to receive additional milestone payments of up to $3.7 million. Furthermore, J&J’s investment arm JJDC is putting down $6.6 million in equity for RallyBio.
In return, Rallybio will share certain aggregated, anonymized natural history data from its FNAIT studies with J&J. The biotech will also "disseminate information to its FNAIT study sites related to J&J’s research and development of its complementary therapeutic approaches aimed at reducing the risk of FNAIT," the company told Fierce.
Rallybio is running the FNAIT natural history study with the mission of providing a modern dataset for HPA-1a alloimmunization frequency that is intended to support a future registrational study of RLYB212. The biotech is slated to launch a phase 2 dose confirmation trial for RLYB212 among pregnant individuals with a higher risk of alloimmunization and FNAIT in the second half of this year.
RLYB212 is the only known clinical-stage therapy designed for pregnant individuals at risk of FNAIT who haven’t alloimmunized, according to RallyBio.
Meanwhile, J&J is currently running a phase 3 trial of nipocalimab—a monoclonal antibody designed to target FcRn—among pregnant individuals who are already alloimmunized. While RLYB212 is designed to be preventative, nipocalimab is aimed at individuals who already have the alloantibodies that cause FNAIT.
There currently aren’t any approved therapies specifically targeting the prevention or prenatal treatment of FNAIT, Rallybio noted in an April 10 release.
“Our complementary approaches, if successful, would ensure that pregnant individuals at risk of developing FNAIT have a potential treatment option—regardless of their alloimmunization status,” Rallybio CEO Stephen Uden, M.D., said in the release. “Together, we can more effectively and expeditiously drive awareness of FNAIT, emphasize the importance of screening pregnant individuals for their risk of developing FNAIT and advance our complementary therapeutic approaches.”
The Big Pharma partnership comes a little more than two months after Rallybio sent 45% of its staff packing and zoomed in on two phase 2-ready programs—one of which is RLYB212—to extend its cash runway into the middle of 2026. The other program, RLYB116, is a C5 inhibitor that the biotech believes could potentially treat several diseases involving complement dysregulation.
Rallybio entered 2024 with cash, cash equivalents and marketable securities of $109.9 million.