— FDA Clears Initiation of Clinical Trials for NM8074 Intended for Paroxysmal Nocturnal Hemoglobinuria —

CLEVELAND, OH, USA I January 31, 2022 I NovelMed Therapeutics announced today that its Investigational New Drug Application (IND) to initiate clinical trials of its NM8074 biologic has been cleared by the U.S. Food and Drug Administration (FDA). NM8074 is the first of the company’s three advanced, humanized monoclonal antibodies that control overactive inflammation and cellular damage contributing to pathology associated with multiple complement-mediated rare diseases. Through its targeted specificity to the complement alternative pathway (AP), NM8074 is expected to improve upon current treatments such as Soliris™, Ultomiris™, and Empaveli™, which are approved for treatment of a limited number of rare diseases.

There’s a well-established need for targeted treatments. Net sales of Soliris and Ultomiris totaled more than $5 billion in 2020. Empaveli was only recently approved by the FDA in May 2021 but the long-term effects of this non-specific C3 blocker remain unknown. Despite the limited scope of Empaveli, analysts believe its sales may reach approximately $130 million in 2022.

NovelMed’s alternative pathway blocker Anti-Bb (NM8074) is expected to treat multiple rare diseases

A Phase I placebo-controlled, double-blind, single-ascending-dose study to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of NM8074 administered intravenously to healthy subjects is nearing completion (Q1 2022). Early results from Phase I trials indicate that the biologic is safe at all doses tested, which has enabled planning of the first Phase II clinical trials in patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare disease characterized by red blood cell destruction, blood clots and impaired bone marrow function. Additional Phase II trials for treating complement-mediated rare diseases are being planned in parallel.

“The FDA’s decision conveys the clinical need and value of NM8074 towards PNH,” stated Rekha Bansal, Ph.D., Chief Executive Officer of NovelMed. “We believe that NM8074’s novel mechanism of action will result in a superior safety and efficacy profile compared with other drugs on the market for the treatment of this disorder. NM8074 selectively binds to complement alternative pathway specific protein Bb and inhibits the formation of C3a/C3b, C5a/C5b, and MAC, products that mediate inflammation and tissue damage in numerous diseases. NM8074 carries a mechanistic advantage over current platforms because it targets the disease-specific complement alternative pathway (AP) without blocking the classical pathway (CP) required for host defense. NM8074 is expected to function against multiple complement-mediated rare diseases; and we predict that the strong therapeutic rationale used in the development of NM8074 will position this biologic extremely well against currently FDA-approved drugs, as well as those in development.”

About NM8074

NM8074 selectively binds Bb of the alternative pathway with high affinity. It is an inhibitor of two powerful proteases produced by the disease-specific complement alternative pathway (AP). The AP plays a key role in the initiation and propagation of inflammation and tissue destruction, as evident in complement etiology of several chronic diseases. AP-specific C3 convertase is responsible for the conversion of C3 to C3a and C3b, while AP C5 convertase is responsible for the conversion of C5 to C5a and C5b. Unlike commercially available non-specific C3 and C5 blockers (Empaveli and Soliris/Ultomiris), treatment with NM8074 would provide selectivity and specificity, while sustaining the activity of the classical complement pathway (CP). This is extremely critical as resistance to infectious microorganisms in patients is mediated via the CP.  

Collectively, selective blockade of the AP, without impacting the CP, will allow patients to maintain host defense mechanisms that are responsible for the prevention of infections. As an investigational humanized monoclonal antibody, NM8074 has been extensively studied in a PNH model system to demonstrate that it:

a) blocks AP-driven erythrocyte hemolysis and C3b deposition in serum from PNH patients with and without Soliris treatment, confirming that this drug would prevent both intra- and extra-vascular hemolysis, and

b) sustains CP function, which is critical for host defense and protection against infectious agents.

Collectively, the drug is expected to block intravascular hemolysis, extravascular hemolysis, lactate dehydrogenase (LDH), and infections in PNH patients. NM8074 is expected to be therapeutically effective across a broad range of rare and common complement-mediated diseases, including hemolytic disorders, renal disorders, ocular disorders, neurological disorders, and inflammatory disorders.

About NovelMed Therapeutics

NovelMed is a clinical-stage biopharmaceutical company committed to innovating and developing biologics to treat rare diseases caused by the overactivation of the complement alternative pathway. NovelMed’s goal is to develop a target specific drug that can treat many rare diseases.

NovelMed has created a strong portfolio of intellectual property with broad applications, including the use of NM8074 for the treatment of a range of complement-mediated disorders related to excessive activation of the alternative pathway. As part of its antibody platform, NovelMed’s portfolio also includes humanized antibodies to C3b and properdin that block and prevent the formation of C3/C5 convertases, the two powerful proteases of the alternative pathway. NovelMed’s anti-properdin monoclonal antibody program is currently progressing towards multiple Phase II trials in other complement-mediated diseases. For more information, please visit www.novelmed.com.

SOURCE: Novelmed Therapeutics