Merger aims to speed late-stage development of novel PNH treatment
New $132M private investment also backs Jasper's acquitions of Kira
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Jasper Therapeutics has announced its acquisition of Kira Pharmaceuticals in a deal that will provide new resources and expertise to help advance the late-stage clinical development of KP104, a treatment candidate for paroxysmal nocturnal hemoglobinuria (PNH), as well as other experimental therapies involving the immune system.
For KP104, which the company describes as a “potentially best-in-disease” PNH treatment, Jasper plans to use the merger to support regulatory discussions with the U.S. Food and Drug Administration (FDA). The company is working to define the next steps for KP104’s development, which are expected to be announced in the first half of 2027, according to a press release detailing the terms of the merger.
Those next steps may include launching a Phase 3 clinical trial in PNH, following promising safety and effectiveness data from a Phase 2 study (NCT05476887). That small study, conducted in China, tested the safety, tolerability, and pharmacological properties of KP104 in previously untreated people with PNH.
“As we advance as part of Jasper, our mission is to develop biologic agents designed to improve outcomes in patients suffering from numerous immunologically-driven disorders,” said Patrick Crutcher, Kira’s former chairman of the board. “We will leverage the Combined Company’s management team with deep expertise in antibody drug development and support from leading life science investors.”
The merger was accompanied by a $132 million private investment, with participation from leading life sciences investors and Mirador Therapeutics, which is expected to fund multiple clinical milestones from both companies until the second half of 2028.
A genetic condition, PNH is characterized by red blood cell destruction, or hemolysis, caused by abnormal activation of the complement cascade, a group of immune proteins. In PNH, hemolysis may occur both inside (intravascular) and outside (extravascular) blood vessels.
Standard therapies that block complement activation by targeting the C5 complement protein can help prevent hemolysis, particularly when it occurs inside blood vessels. However, about 20% of patients using these therapies continue to experience extravascular hemolysis.
Treatment seen to prevent red blood cell destruction in PNH
Developed initially by Kira, KP104 (vensobafusp alfa) targets both the C5 protein and complement factor H to help prevent both types of hemolysis. It is a lab-made protein that combines a C5-targeting antibody with part of complement factor H.
“Kira has built a truly differentiated complement portfolio that includes dual [mode of action] beyond single-pathway agents and long-acting complement inhibitors, reflecting the quality of their science and the deep expertise of their team,” said Jeet Mahal, Jasper’s president and CEO. “We are excited by the robust pipeline that this transaction creates, and are looking forward to advancing these important medicines for patients.”
The Phase 2 study enrolled 18 PNH patients not previously treated with a complement inhibitor. Each received KP104 every two weeks via subcutaneous, or under-the-skin, injections.
After six months, KP104 demonstrated a favorable safety profile and was shown to control both intravascular and extravascular hemolysis. Patients also stopped needing red blood cell transfusions and experienced a clinically meaningful reduction in fatigue.
Final results from that study, presented last year at the American Society of Hematology Annual Meeting, showed that all patients experienced a sustained rise in hemoglobin by at least 2 g/dL. Hemoglobin is the protein that carries oxygen in red blood cells. Mean hemoglobin levels rose by 6.7 g/dL, to a mean of 13.7 g/dL, with more than 80% of the patients achieving normal hemoglobin levels (12 g/dL or higher).
Additionally, almost all participants maintained normal levels of lactate dehydrogenase (LDH), a marker of hemolysis, and none required red blood cell transfusions throughout the study.
KP104 shown to be safe over 2 years of treatment
KP104 continued to show a favorable safety profile after two years of treatment. No serious adverse events or treatment discontinuations due to side effects were reported. The most frequent side effects included COVID-19, injection site reaction, and the common cold. Two cases of breakthrough hemolysis, or BTH, in which PNH symptoms suddenly returned despite ongoing treatment, were reported.
After stopping KP104, most participants maintained treatment benefits for several weeks. Normal hemoglobin levels were preserved for up to eight weeks in approximately 80% of participants, the data showed.
Similarly, two-thirds of patients maintained normal LDH levels four weeks after treatment discontinuation, although this proportion declined to 20% by eight weeks, the researchers noted.
KP104 has been granted orphan drug status by the FDA for the treatment of PNH. That designation aims to accelerate the development of medications to treat rare diseases, providing advantages such as exemption from regulatory fees, eligibility for tax credits, and seven years of market exclusivity if the treatment is approved.
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