• The Phase I healthy trial revealed that NM8074 effectively blocked the alternative pathway (AP) and AP-mediated C3b deposition without affecting the classical pathway (CP). Importantly, NM8074 did not interfere with the deposition of C3b via the CP, a crucial marker of opsonization, across all cohorts in the Phase I trial.
• Regulatory approval for the Phase II trial in treatment-naïve PNH patients was granted based on the safety and tolerance demonstrated in the previous Phase I clinical trial involving healthy subjects. NM8074 proved to be safe and well-tolerated across various doses, ranging from 0.3 to 20 mg/kg.
• Subsequent dosing of NM8074 in treatment-naïve PNH patients yielded promising results. Notably, NM8074 led to significant reductions in Lactate Dehydrogenase (LDH), Reticulocytes, Free Hemoglobin, and D-dimer levels, while simultaneously increasing Hemoglobin and Haptoglobin levels.
• These results collectively suggest that NM8074 has the potential to selectively inhibit both intravascular hemolysis (IVH) and extravascular hemolysis (EVH) through the AP, while preserving the CP necessary for bacterial clearance and opsonization in treatment-naïve PNH patients.

CLEVELAND, Oct. 30, 2023 (GLOBE NEWSWIRE) -- NovelMed Therapeutics is excited to announce the commencement of its Phase II clinical trial targeting treatment-naïve Paroxysmal Nocturnal Hemoglobinuria (PNH) patients with its groundbreaking anti-Bb antibody, NM8074. This open-label, multi-dose, and multi-center study aims to assess the safety and efficacy of NM8074 in PNH patient population. To date, eight PNH patients have been enrolled in the trial, with two patients already receiving NM8074 treatment. Key efficacy parameters include increases in Hemoglobin, increase in RBC clone size, decreased Lactate Dehydrogenase (LDH) levels, reduced pRBC transfusions, and reduced D-Dimer levels. Positive outcomes on these measures will pave the way for NM8074 as a highly differentiated and innovative biologic for treatment-naïve PNH patients.

The Phase II study is divided into two cohorts, featuring a biweekly dosing schedule spanning a 3-month assessment period. Notably, the preliminary data indicates that NM8074 demonstrates a favorable safety profile, with no safety concerns. NM8074 was administered to treatment-naïve PNH patients with a clone size exceeding 10%. The results demonstrate significant reductions in LDH, Reticulocytes, Free Hemoglobin, and D-dimer, along with increases in Hemoglobin and Haptoglobin levels as expected. Furthermore, the clone size increased in a time-dependent manner. In summary, these observed changes in efficacy parameters validate the drug's mechanism of action and substantiate NM8074 as an effective PNH treatment option.

Dr. Rekha Bansal, CEO of NovelMed Therapeutics, commented, "Preliminary data from the Phase II clinical trial in treatment-naïve PNH patients confirms NM8074's potential to comprehensively address intravascular and extravascular hemolysis-mediated anemia and bolster patient host defense systems, thus reducing the risk of infections."

Previously, a Phase I trial involving forty healthy volunteers was successfully completed without any safety concerns. NM8074 was well tolerated across all doses, ranging from 0.3 to 20 mg/kg. These results corroborate NM8074's complete inhibition of the alternative pathway (AP) while preserving the classical pathway (CP), aligning with data generated by NovelMed's expert scientists. This selective mechanism distinguishes NM8074 from other complement blockers currently approved or in development by the FDA.

About Paroxysmal Nocturnal Hemoglobinuria (PNH)

PNH is a rare blood disorder characterized by the immune system's (Complement) attack on and destruction of PNH Red Blood Cells (RBC hemolysis). This leads to a decrease in hematocrit and hemoglobin levels, elevated LDH and D-Dimer, and an increased need for blood transfusions. PNH is a rare, chronic, and serious complement-mediated blood disorder with significant unmet needs. Despite the use of anti-C3 and anti-C5 therapies, many PNH patients continue to experience anemia, fatigue, and dependence on blood transfusions.

About NM8074

NM8074 is a monoclonal antibody with dual specificity, differentiating it from other therapies such as Iptacopan, which binds to Factor B. NM8074 targets Bb and selectively blocks the alternative pathway (AP). This high-affinity monoclonal antibody inhibits C3b formation and deposition and membrane-attack complex (MAC) formation, offering a significant advantage in preventing the total lysis of red blood cells in PNH. Furthermore, NM8074 is being evaluated for other clinical indications, including atypical Hemolytic Uremic Syndrome (aHUS), for which it has received regulatory approval. NM8074's potential extends to complement-mediated pathologies in renal disorders, ocular disorders, neurological disorders, dermatological disorders, and inflammation, illustrating its broad application across multiple diseases.

Dr. Rekha Bansal, Chief Executive Officer, emphasized, "This wide application to multiple diseases underscores our commitment to serving diverse patient populations."

In contrast to other complement blockers from third parties, which indiscriminately block both the alternative and classical complement pathways, NM8074 offers a unique mechanism of action. It is expected to function as a superior therapeutic option, potentially without the Black-Box warning associated with currently FDA-approved treatments.

About NovelMed Therapeutics

NovelMed is a clinical-stage biopharmaceutical company dedicated to developing innovative biologics for the treatment of a wide range of complement-mediated diseases. NovelMed proudly leads in the development of anti-Bb antibodies to address chronic complement-mediated and complement-associated disorders. The company is actively conducting clinical trials in treatment-naïve PNH patients to expedite the availability of this promising treatment. Moreover, NovelMed is committed to delivering life-changing therapies to patients in the fields of hematology, ophthalmology, nephrology, dermatology, and neurology. NovelMed is Seeking Opportunities to Advance NM8074 for Rare Diseases.

For more information, please visit https://www.novelmed.com/news

Contact:

Rekha Bansal
NovelMed Business Development Team
bd@novelmed.com
(216) 440 2696