-- 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 www.NovelMed.com.

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 www.clinicaltrials.gov. 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 www.novelmed.com.

Business Development Team
bd@novelmed.com
Tel: (216) 440 2696

NovelMed is on LinkedIn. Sign up to follow @NovelMed at https://www.linkedin.com/company/www.novelmedlinkedin.com

References

1.   NovelMed's Complement Alternative Pathway Specific Anti-Bb Antibody (NM8074) for Rare Diseases Achieves a Major Milestone. NovelMed. January 31, 2022.
https://www.prnewswire.com/news-releases/novelmeds-complement-alternative-pathway-specific-anti-bb-antibody-nm8074-for-rare-diseases-achieves-a-major-milestone-301466964.html

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.
https://www.prnewswire.com/news-releases/novelmed-announces-interim-positive-results-from-its-phase-i-clinical-trial-of-nm8074-a-bb-complement-alternative-pathway-blocker-antibody-301485271.html?tc=eml_cleartime

3.   Returning to complement – can NovelMed’s next-gen antibody outperform Ultomiris? NovelMed. April 22, 2022.
https://app.box.com/s/ljp0s20sqrjxp8oq6hh9j5pltfe8qx8x

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.
https://www.prnewswire.com/news-releases/alternative-pathway-blocker-anti-bb-antibody-nm8074-receives-us-fda-clearance-to-start-efficacy-trial-in-c3-glomerulopathy-c3g-patients-301605303.html?tc=eml_cleartime

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.
https://www.prnewswire.com/news-releases/anti-properdin-antibody-nm3086-reduced-hemolysis-ldh-and-free-hemoglobin-in-an-animal-model-of-paroxysmal-nocturnal-hemoglobinuria-pnh----a-rare-disease-301700471.html?tc=eml_cleartime

6.   NovelMed reports topline data with anti-properdin antibody NM-3086 in rabbit model of PNH. NovelMed. December 13, 2022
https://www.bioworld.com/articles/692378-novelmed-reports-topline-data-with-anti-properdin-antibody-nm-3086-in-rabbit-model-of-pnh?v=preview

7.   Study of NM8074 in Patients With aHUS With Evidence of Ongoing Thrombotic Microangiopathy.
clinicaltrials.gov; 2022. Accessed January 25, 2023. 
https://clinicaltrials.gov/ct2/show/NCT05684159?cond=NCT05684159&draw=2&rank=1

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. 
https://clinicaltrials.gov/ct2/show/NCT05646524?cond=NCT+05646524&draw=2&rank=1

9.   Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris
clinicaltrials.gov; 2022. Accessed January 25, 2023. 
https://clinicaltrials.gov/ct2/show/NCT05646563?cond=NCT05646563&draw=2&rank=1

10.   Study of NM8074 in Patients With aHUS With Evidence of Ongoing Thrombotic Microangiopathy
clinicaltrials.gov; 2022. Accessed January 25, 2023. 
https://clinicaltrials.gov/ct2/show/NCT05684159?cond=NCT05684159&draw=2&rank=1

11.   Study of NM8074 in Adult C3 Glomerulopathy Patients
clinicaltrials.gov; 2022. Accessed January 25, 2023. 
https://clinicaltrials.gov/ct2/show/NCT05647811?cond=NCT05647811&draw=2&rank=1