— Precision Bb Targeting, Robust Efficacy, Transfusion Independence, Excellent Safety in Phase II PNH Study, and Subcutaneous Administration
Key Highlights: Monotherapy and Clinical Outcomes
The 12-16-week open-label study evaluated Ruxoprubart as monotherapy in treatment-naïve PNH patients. Key findings include:
- Hemoglobin (Hb) Improvement: Hemoglobin levels increased by 1.5–2.7 g/dL, with an average rise of ~2.13 g/dL during weekly dosing, reflecting meaningful correction from the severely depressed baseline common in untreated PNH.
- Complete Transfusion Independence: Throughout the weekly dosing period, 100% of patients required no blood transfusions, demonstrating effective control of hemolysis and clinically meaningful correction of anemia.
- PNH Red Blood Cells (RBCs) Survival & Expansion: PNH RBC populations expanded to up to 94% of their maximum level. This is consistent with near-complete suppression of ongoing complement-mediated hemolysis, a core driver of anemia and fatigue.
- Biochemical Control (LDH): Lactate Dehydrogenase (LDH) levels were significantly reduced, reflecting biochemical control of intravascular hemolysis (IVH).
- Quality-of-Life (QoL) Gains: Patient‑reported outcomes, including the Functional Assessment of Chronic Illness Therapy (FACIT) and the European Organization for Research and Treatment of Cancer (EORTC) questionnaires, showed meaningful improvements across fatigue, pain, and overall functional capacity.
- Safety and Tolerability: The therapy was well-tolerated with no drug-related Adverse Events (AEs) or Serious Adverse Events (SAEs) observed.
- Orphan Drug Designation: Ruxoprubart holds U.S. FDA Orphan Drug Designation (ODD) for PNH, highlighting its potential to address this rare and life-threatening condition.
CLEVELAND, March 09, 2026 (GLOBE NEWSWIRE) — NovelMed Therapeutics Inc., a clinical-stage biopharmaceutical company specializing in alternative pathway (AP) complement-mediated therapies, today announced two major milestones in the clinical development of Ruxoprubart (NM8074) for Paroxysmal Nocturnal Hemoglobinuria (PNH).
The company has received regulatory clearance to initiate a Phase II study for the subcutaneous (SC) route of administration, designed as a convenient, weekly, self-administered injection. Concurrently, NovelMed reported the full results of its Phase II intravenous (IV) monotherapy study in treatment-naïve adult patients with PNH. The study met all efficacy endpoints, including significant hemoglobin improvement and complete transfusion avoidance, with an exceptional safety profile.
“These milestones validate both our clinical progress and our development strategy,” said Dr. Rekha Bansal, Chief Executive Officer of NovelMed. “Regulatory clearance of the SC route represents the next logical step toward a patient-friendly treatment option, while our monotherapy data demonstrate the strong, consistent efficacy and tolerability of targeting Bb. We are now progressing into the next phase of clinical development.”
Advancing Two Complementary Tracks in PNH
NovelMed is progressing Ruxoprubart through two distinct delivery methods to maximize patient access and convenience:
- Subcutaneous (SC) Administration: Cleared for Phase II Development
The regulatory agency has cleared the SC route of administration, enabling a planned, weekly self-injection regimen at home. SC trials are planned but have not yet been initiated. This approach is expected to reduce patient and caregiver burden by eliminating the need for IV infusions in a clinical setting.
- Intravenous (IV) Monotherapy: Treatment-Naïve PNH Patients
The successful completion of the intravenous Phase II study establishes Ruxoprubart as a potent monotherapy capable of addressing the core drivers of PNH without adjunctive treatment.
“Ruxoprubart has proven its potential as a definitive monotherapy, delivering exceptional efficacy without compromise,” said Mr. Alex Kumar, Chief Strategy Advisor. “Coupled with FDA Orphan Drug Designation status and regulatory clearance for SC dosing, we have a clear, accelerated path forward. NovelMed is now uniquely positioned to lead the next generation of precision complement therapeutics on a global scale.”
Precision Targeting: The Bb Mechanism and Differentiation
PNH is an AP-driven hemolytic disorder. Ruxoprubart (NM8074) is a first-in-class monoclonal antibody engineered for precision targeting of Bb, the proteolytically activated catalytic fragment generated upon AP activation. By binding Bb rather than native Factor B, Ruxoprubart delivers precise AP-selective inhibition while preserving the Classical Pathway (CP), which remains essential for protecting patients from infections.
“The Phase II intravenous results demonstrate a clinical efficacy and safety profile that aligns well with FDA expectations for monotherapy development in PNH,” said Mr. Robert Bard. “With the SC route now cleared for evaluation under our Phase II program, we are advancing along a well-defined regulatory path. This progress brings us closer to enabling patient-friendly, self-administered treatment options supported by a mechanism designed for pathway-selective precision.”
Competitive Landscape at a Glance
- Ruxoprubart (NovelMed): Targets activated Bb (FDA Orphan Drug Designation Holder). A precision monotherapy that provides selective AP inhibition while preserving the CP for host defense.
- Soliris® / Ultomiris® (AstraZeneca): Targets C5. A distal blocker of both AP and CP that fails to prevent C3b deposition, which leads to persistent anemia.
- Empaveli® / Syfovre® (Apellis): Targets C3. A proximal blocker that provides broad inhibition of both AP and CP, addressing C3b-mediated EVH but impacting overall complement surveillance.
- Fabhalta® (Novartis): Targets native Factor B. A proximal blocker that lacks precision by targeting the native protein rather than the proteolytically activated Bb fragment.
- Voydeya® (AstraZeneca): Targets Factor D. A proximal blocker, strictly approved as an add-on therapy to C5 inhibitors rather than a standalone monotherapy.
Key Pillars of the Regulatory Strategy
- Monotherapy Treatment: Ruxoprubart has demonstrated the ability to meet all primary endpoints as a standalone treatment. This allows for a more direct regulatory path, as the drug does not need to be tested or approved alongside existing C5 inhibitors to prove its value.
- Subcutaneous (SC) Administration: The regulatory clearance for the SC route is a major milestone. Having passed rigorous safety and manufacturing reviews, Ruxoprubart is now cleared for human trials using at-home injections. This shifts the treatment model from clinical IV infusions to a more convenient, patient-controlled approach.
- Precision Targeting of the Bb: Ruxoprubart selectively targets the AP while keeping the CP intact. This “selective precision” is designed to maintain the body’s natural ability to fight infections, a differentiated safety profile that supports the case for expedited regulatory designations like Fast Track or Breakthrough Therapy.
Figure. Complement-mediated destruction of red blood cells in paroxysmal nocturnal hemoglobinuria (PNH).
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare, acquired, and life-threatening hematologic disorder caused by a somatic mutation in the PIGA gene. This mutation results in the deficiency of GPI-anchored complement regulatory proteins, specifically CD55 and CD59, which act as protective shields for RBCs. Without these proteins, RBCs are highly vulnerable to persistent attack by the AP of the complement system, leading to chronic intravascular and extravascular hemolysis. This ongoing destruction manifests clinically as severe anemia, dangerously elevated Lactate Dehydrogenase (LDH) levels, and a significantly increased risk of life-threatening thrombosis and renal impairment. Effective treatment is measured by stabilization of hemoglobin and the expansion of the PNH RBC clone, indicating that these cells are successfully protected from complement-mediated attack.
About NovelMed Therapeutics
NovelMed Therapeutics Inc. is a clinical-stage biopharmaceutical company dedicated to developing next-generation immunotherapies that selectively target the complement AP. The company has pioneered a site-specific approach by engineering monoclonal antibodies, such as its lead candidates Ruxoprubart (NM8074) and NM5072, to target proteolytically activated fragments, such as Bb, rather than native proteins. This precision enables potent blockade of the disease-driving AP while intentionally preserving the CP’s critical role in host defense and immune surveillance. Supported by a robust portfolio of intellectual property and promising Phase II data, NovelMed is advancing a differentiated pipeline of first-in-class biologics designed to achieve durable clinical outcomes without compromising the patient’s ability to fight infections.
Strategic Partnership Opportunity
Following the achievement of 100% transfusion avoidance in its Phase II monotherapy study and the recent regulatory clearance for SC administration, NovelMed is actively pursuing a strategic partnership to propel Ruxoprubart through late-stage global development. This program is exceptionally well-positioned for expansion, supported by U.S. FDA Orphan Drug Designation (ODD) and a validated, precision mechanism that addresses significant unmet needs in the PNH market. NovelMed seeks a partner with extensive clinical and commercial expertise to accelerate the regulatory path towards approval and support the global launch of this patient-friendly, self-administered therapy. By combining NovelMed’s innovative science with a partner’s scale, the company aims to redefine the treatment landscape for rare complement-mediated diseases.
Media Contact:
Ya Gao, MS
NovelMed Therapeutics, Inc.
[email protected]
(216) 440 2696
Forward-Looking Statements
This press release contains forward-looking statements that reflect NovelMed’s current expectations and projections about future events. Ruxoprubart (NM8074) is an investigational therapy and has not been approved by any regulatory authority for commercial use. The subcutaneous administration route has received regulatory clearance for further study; no subcutaneous clinical trials have been initiated. Forward-looking statements include expectations about clinical development, regulatory interactions, future study plans, and potential outcomes. NovelMed is a privately held, clinical stage biotechnology company which undertakes no obligation to update these statements except as required by law.
A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/3011d906-8908-4ee6-aad1-bee88a2baaf2
