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-- FDA Clears Initiation of Efficacy Trial in aHUS (Atypical Hemolytic Uremic Syndrome) Patients An Efficacy Trial in Adult aHUS Patients Who Are Naïve to Complement Inhibitor Therapy (NCT05684159) Phase I trial in healthy volunteers shows that NM8074 completely blocks the alternative pathway (AP) without blocking the classical pathway (CP), an important advantage over other complement blockers already approved by the FDA NM8074 is expected to provide the selectivity and the specificity required for complete remission of thrombotic microangiopathy which remains as an unmet need in patients with aHUS Given the role of the AP in multiple complement-mediated rare diseases, NM8074 has the potential to treat not only aHUS but several other complement-mediated diseases including ocular, inflammatory, renal, neurological and hematological CLEVELAND, Jan. 30, 2023 (GLOBE NEWSWIRE) -- NovelMed announced today that the U.S. Food and Drug Administration (FDA) cleared the Company’s investigational drug NM8074 to initiate an efficacy trial in patients with aHUS in coming months. NovelMed is advancing its anti-Bb antibody (NM8074) to treat patients with evidence of treatment-related resistance or complement-mediated relapses in aHUS, a rare disease with unmet need. NovelMed is a clinical-stage biopharma company focused on the development of humanized monoclonal antibody treatments for several complement-mediated disorders. NM8074 has recently completed a Phase I trial in healthy volunteers with no safety concerns. Our results show that NM8074 completely blocks the alternative pathway (AP) without blocking the classical pathway (CP) consistent with the laboratory data generated by NovelMed scientists. This is an important advantage over other complement blockers already approved by the FDA or in development. NM8074 blocks the AP-mediated formation of C3b, an important molecule responsible for incurable anemia seen in both Soliris and Ultomiris treated patients. Opsonization controlled by the CP is not affected by NM8074, which is a key factor in targeted AP inhibition of the complement system. “We speculate that patients would not have to take the meningococcal vaccine in the future should the drug prove effective in the upcoming clinical trial of this rare disease” stated Dr. Bansal. "The FDA's clearance of the Phase II clinical trial for aHUS, a rare disease, is an important milestone in the active life of NM8074," said Rekha Bansal, Ph.D., Chief Executive Officer of NovelMed. “Our drug functions by binding Bb at its catalytic site and has no affinity for Factor B. As a result, the drug selectively blocks the alternative pathway (AP) without affecting the CP. Our proposed trial is entitled ‘An Open Label Trial to Evaluate Safety and Efficacy of NM8074 in Adult aHUS Patients Who Are Naïve to Complement Inhibitor Therapy’ (NCT05684159).” The AP plays a key role in controlling anemia, inflammation, and tissue damage in several complement-mediated disorders. “Based on our initial clinical data, it is clear that NM8074 could provide the selectivity and the specificity required for developing a novel biologic for the complete remission of thrombotic microangiopathy in patients with aHUS. Given the role of the AP in multiple complement-mediated rare diseases, NM8074 has the potential to treat not only aHUS but several other complement-mediated diseases as well, where AP is the sole contributor of the pathological outcome,” stated Dr. Bansal. Seeking Funds to Advance the Development of NM8074 for Rare Disease Platform aHUS is a rare disease characterized by exaggerated AP activation and hemolysis. “Because the disease is rare (orphan), its market size is significant,” states the Business Development Team at NovelMed. Hence, NM8074 has the potential to capture a significant portion of this market. NovelMed is currently seeking licensing, partnership, and acquisition opportunities to drive NM8074 through further development and approval in multiple rare disease indications. For more information, please visit . About NM8074 NM8074 is a humanized monoclonal antibody that binds Bb, the catalytic domain of Factor B, and selectively blocks the initiation and propagation of the alternative pathway (AP). Treatment with NM8074 would selectively target the AP while leaving the CP pathway intact. In addition to treating aHUS, NM8074 is expected to benefit patients suffering from a variety of kidney disorders where activation of the AP is the key driver of pathogenesis. Furthermore, NM8074 is expected to be therapeutically effective across a broad range of rare diseases and disorders, including hemolytic PNH (NCT 05646524, NCT05646563), hemolytic aHUS (NCT05684159), renal C3G (NCT05647811), ocular, neurological, and inflammatory disorders. Multiple clinical trials have been registered on NM8074 has the potential to treat many complement-mediated and complement-associated illnesses, especially those for which there are inadequate treatment regimens or complete unavailability of treatment. About NovelMed As a clinical-stage biopharmaceutical company, we are committed to innovating and developing novel biologics for the treatment of rare (orphan) diseases. Our goal is to develop an alternative pathway-specific drug that can treat a multitude of rare diseases and provide patients with low-cost treatment. NovelMed has created a strong portfolio of intellectual property with broad applications to rare and common diseases. This includes the use of NM8074 as a treatment for a range of complement-mediated disorders related to uncontrolled activation of the AP. As part of its antibody platform, NovelMed's portfolio also includes humanized antibodies to C3b and Factor P (Properdin) that block the formation of C3/C5 convertases. NovelMed is currently creating licensing, partnership, and acquisition opportunities to drive its antibody development through Phase III and approval in multiple rare disease indications. For more information, please visit . Business Development Team bd@novelmed.com Tel: (216) 440 2696 NovelMed is on LinkedIn. Sign up to follow @NovelMed at References 1. NovelMed's Complement Alternative Pathway Specific Anti-Bb Antibody (NM8074) for Rare Diseases Achieves a Major Milestone. NovelMed. January 31, 2022. 2. NovelMed Announces Interim Positive Results from Its Phase I Clinical Trial of NM8074, a Bb Complement Alternative Pathway Blocker Antibody. NovelMed. February 21, 2022. 3. Returning to complement – can NovelMed’s next-gen antibody outperform Ultomiris? NovelMed. April 22, 2022. 4. Alternative Pathway Blocker Anti-Bb Antibody (NM8074) Receives US FDA Clearance to Start Efficacy Trial in C3 Glomerulopathy (C3G) Patients. NovelMed. August 15, 2022. 5. Anti-Properdin Antibody (NM3086) Reduced Hemolysis, LDH, and Free Hemoglobin in an Animal Model of Paroxysmal Nocturnal Hemoglobinuria (PNH) -- a Rare Disease. NovelMed. December 12, 2022. 6. NovelMed reports topline data with anti-properdin antibody NM-3086 in rabbit model of PNH. NovelMed. December 13, 2022 7. Study of NM8074 in Patients With aHUS With Evidence of Ongoing Thrombotic Microangiopathy. clinicaltrials.gov; 2022. Accessed January 25, 2023. 8. Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy clinicaltrials.gov; 2022. Accessed January 25, 2023. 9. Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris clinicaltrials.gov; 2022. Accessed January 25, 2023. 10. Study of NM8074 in Patients With aHUS With Evidence of Ongoing Thrombotic Microangiopathy clinicaltrials.gov; 2022. Accessed January 25, 2023. 11. Study of NM8074 in Adult C3 Glomerulopathy Patients clinicaltrials.gov; 2022. Accessed January 25, 2023.

CLEVELAND, Aug. 15, 2022 /PRNewswire/ -- NovelMed Therapeutics is a clinical-stage biopharmaceutical company focused on the development of targeted therapies for complement-mediated rare diseases. The Company announced today that the U.S. Food and Drug Administration (FDA) cleared the Company's Investigational drug NM8074 to initiate an Efficacy trial in patients with C3G in the First Quarter of 2023. NM8074 has recently completed a Phase I trial in forty (40) healthy volunteers with no safety concerns. Figure: Flow Cytometry data showing total inhibition of C3b deposition with NM8074. The Untreated control demonstrates C3b deposition (C3b (+)ve), Soliris shows enhanced C3b deposition, and NM8074 exhibits a complete lack of C3b deposition on cells. "The FDA's decision conveys the unmet clinical need for and importance of NM8074 as a treatment for C3G," stated Rekha Bansal, Ph.D., Chief Executive Officer of NovelMed. "We believe that our drug's novel and well-differentiated mechanism of action is expected to result in a superior safety and efficacy profile compared to those in development for C3G indication" NM8074 functions by binding Bb and selectively blocking the alternative pathway by neutralizing the activity of C3 convertase and C5 convertase, the two powerful proteases of the alternative pathway (AP). Further, NM8074 also blocks the formation of these proteases by impairing the amplification loop of the AP. Interestingly, NM8074 does not block the classical pathway (CP) which is critical for host defense against infections. The AP plays a key role in the initiation and propagation of C3b deposition, inflammation, and tissue damage, as evident in complement-mediated diseases including C3G. AP-specific C3 convertase is responsible for the conversion of C3 to C3a/C3b, while AP C5 convertase is responsible for the conversion of C5 to C5a/C5b and Membrane Attack Complex (MAC). As a result, NM8074 would provide the selectivity and the specificity required for developing a novel treatment for C3G, a rare disease. "Given the role of the AP in multiple complement-mediated rare diseases, NM8074 has the potential to treat not only C3G but several other complement-mediated and complement-associated diseases as well," said Dr. Bansal. C3G is a rare disease collectively characterized by the deposition of C3/C3b on kidney cells, proteinuria, and reduced eGFR. In addition to C3G, other complement-mediated renal diseases include C3-glomerulonephritis (C3GN), dense deposit disease (DDD), membranoproliferative glomerulonephritis (MPGN), and immune complex membranoproliferative glomerulonephritis (IC-MPGN). "Whereas each of these diseases is rare, market size is significant," stated Dr. Bansal. With each potential indication's patient population combined and the price of a complement blocker (Soliris) applied, the annual market size could reach in billions of USD. In a Phase I placebo-controlled, a double-blind, single-ascending-dose study in healthy patients, NM8074 was shown to be safe while effectively inhibiting the AP in human serum over an extended period of time, indicating that it would be successful in treating C3G as well as other chronic diseases. Given NM8074's effectiveness in selectively blocking the AP, and its ability to maintain the CP and host defense, NM8074 has the potential to capture a significant portion of this market. About NM8074 NM8074 is a humanized monoclonal antibody that binds Bb with picomolar affinity and exhibits dual specificity. First, NM8074 selectively blocks the AP. Second, it is specific to Bb without binding Factor B, which is present in large amounts. In essence, this protease inhibitor monoclonal antibody blocks the formation of two powerful proteases (C3 and C5 convertases) produced by the activation of the AP. The dual specificity of NM8074 is important in the initiation and propagation of C3b deposition, inflammation, and tissue damage, as evident in complement-mediated and complement-associated chronic rare diseases. Mechanistically, AP-specific C3 convertase is responsible for the conversion of C3 to C3a/C3b, while AP C5 convertase is responsible for the conversion of C5 to C5a/C5b and MAC. Treatment with NM8074 would provide selectivity and specificity to the AP while sustaining the activity of the CP. Therefore, patients will be able to maintain their host defense mechanisms that are critical for the prevention of infections. The investigational drug product, NM8074, has been extensively studied in several AP model systems to demonstrate that it: a) Blocks AP activation upstream, confirming that NM8074 would prevent C3b deposition in C3G b) Blocks the formation of two potent anaphylatoxins responsible for acute and chronic inflammatory cascades c) Blocks the formation of MAC that causes tissue damage d) Is well differentiated from Soliris®/Ultomiris® and Empaveli® having no effect on CP-mediated host defense against infections Collectively, the drug is expected to benefit patients suffering from a variety of kidney disorders including C3G, where activation of the AP is the key driver of pathogenesis. Further, NM8074 is expected to be therapeutically effective across a broad range of rare and common AP-mediated diseases, including hemolytic, ocular, neurological, and inflammatory disorders. NM8074 has the potential to treat many complement-mediated and complement-associated illnesses, especially those for which there are inadequate treatment regimens or complete unavailability of treatment. About NovelMed As a clinical-stage biopharmaceutical company, we are committed to innovating and developing novel biologics for the treatment of rare (orphan) diseases. Our goal is to develop an alternative pathway specific drug that can treat a multitude of rare diseases and provide patients with low-cost treatment. NovelMed has created a strong portfolio of intellectual property with broad applications to rare and common diseases. This includes the use of NM8074 as a treatment for a range of complement-mediated disorders related to uncontrolled activation of the AP. As part of its antibody platform, NovelMed's portfolio also includes humanized antibodies to C3b and Factor P (Properdin) that also block the formation of C3/C5 convertases. NovelMed is currently seeking licensing, partnership, and acquisition opportunities to drive its antibody development through Phase III and approval in multiple rare disease indications. For more information, please visit www.NovelMed.com.