ruxoprubart (NM8074) / NovelMed - LARVOL DELTA

NovelMed’s Phase II Data in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients: Ruxoprubart Shows Best-in-Class Efficacy as Monotherapy (GlobeNewswire) - P2 | N=12 | NCT05646563 | Sponsor: NovelMed Therapeutics | "Interim results from this 12-subject, three-month, multi-dose Phase II efficacy trial are compelling. The drug was safe and well-tolerated in all treated PNH subjects with expected safety & efficacy, meeting all clinical endpoints with no reported side effects. In this efficacy trial, Ruxoprubart: Protected PNH Red Blood cells against lysis and enabled complete transfusion avoidance, meeting the primary endpoint; Increased hemoglobin levels above baseline by 1.4 to 2.0 g/dL in most subjects, with some showing intra-subject hemoglobin increase of 1.5 to 4.3 g/dL, exceeding the threshold set for the primary endpoint. An average hemoglobin increase of >1.6 g/dL was observed across the cohort from baseline to the end of the treatment period; Reduced LDH levels to baseline, meeting the primary efficacy endpoint."

P2 data • Paroxysmal Nocturnal Hemoglobinuria

Study of NM8074 in Soliris-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) (clinicaltrials.gov) - P2 | N=6 | Not yet recruiting | Sponsor: NovelMed Therapeutics | Trial completion date: Oct 2026 ➔ Mar 2028 | Trial primary completion date: Aug 2026 ➔ Sep 2027

Trial completion date • Trial primary completion date • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Study of NM8074 in Patients with Dermatomyositis (DM) (clinicaltrials.gov) - P2 | N=8 | Not yet recruiting | Sponsor: NovelMed Therapeutics

New P2 trial • Dermatomyositis • Immunology • Myositis

Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy (clinicaltrials.gov) - P2 | N=12 | Not yet recruiting | Sponsor: NovelMed Therapeutics | Trial completion date: Sep 2026 ➔ Sep 2028 | Trial primary completion date: Apr 2026 ➔ Aug 2027

Trial completion date • Trial primary completion date • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • HP

Study of NM8074 in Adult PNH Patients with Inadequate Response to Soliris (clinicaltrials.gov) - P2 | N=12 | Not yet recruiting | Sponsor: NovelMed Therapeutics | Trial completion date: Jan 2027 ➔ Jan 2028 | Trial primary completion date: Aug 2026 ➔ May 2027

Trial completion date • Trial primary completion date • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • HP

Study of NM8074 in Patients with Anti-Neutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis (AAV) (clinicaltrials.gov) - P2 | N=12 | Not yet recruiting | Sponsor: NovelMed Therapeutics | Trial completion date: Sep 2027 ➔ Sep 2028 | Trial primary completion date: Oct 2026 ➔ Oct 2027

Trial completion date • Trial primary completion date • ANCA Vasculitis • Vasculitis

United States FDA Approves NM8074 (Ruxoprubart) IND for Treating Dermatomyositis (DM): A Chronic Inflammatory Disorder of the Skin (GlobeNewswire) - "NovelMed Therapeutics...is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted clearance for Ruxoprubart (NM8074), an investigational drug, to begin an efficacy Phase II trial to treat Dermatomyositis (DM), a rare autoimmune disorder....This announcement follows the successful completion of a Phase I clinical trial involving 40 healthy volunteers, where Ruxoprubart demonstrated promising results and a clean safety profile, with no serious adverse events reported."

IND • New P2 trial • Dermatomyositis

FDA Clears Initiation of Phase 2 Efficacy Trial for Ruxoprubart in IgAN (HCPLive) - "The US Food and Drug Administration (FDA) has cleared the initiation of a Phase 2 efficacy clinical trial to evaluate the investigational new drug (IND) ruxoprubart for the treatment of Immunoglobulin A nephropathy (IgAN)...NovelMed added its search to seek strategic partners, investors, and acquisition opportunities that will allow the company to advance ruxoprubart to Phase 2/3 trials and ultimately achieve FDA approval."

IND • IgA Nephropathy

Study of NM8074 in Patients with AHUS with Evidence of Ongoing Thrombotic Microangiopathy (clinicaltrials.gov) - P2 | N=12 | Not yet recruiting | Sponsor: NovelMed Therapeutics | Trial completion date: Feb 2026 ➔ Feb 2027

Trial completion date • Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Nephrology • HP

Study of NM8074 in Adult C3 Glomerulopathy Patients (clinicaltrials.gov) - P1/2 | N=18 | Not yet recruiting | Sponsor: NovelMed Therapeutics | Trial completion date: Jul 2026 ➔ Jul 2027 | Trial primary completion date: Feb 2026 ➔ Feb 2027

Trial completion date • Trial primary completion date • Complement-mediated Rare Disorders • Glomerulonephritis

Study of NM8074 in Patients With Immunoglobulin A Nephropathy (IgAN) (clinicaltrials.gov) - P2 | N=10 | Not yet recruiting | Sponsor: NovelMed Therapeutics

New P2 trial • Glomerulonephritis • IgA Nephropathy • Renal Disease

Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris (clinicaltrials.gov) - P2 | N=12 | Not yet recruiting | Sponsor: NovelMed Therapeutics | Trial completion date: Dec 2025 ➔ Jan 2027 | Trial primary completion date: Aug 2025 ➔ Aug 2026

Trial completion date • Trial primary completion date • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • HP

Study of NM8074 in Adult C3 Glomerulopathy Patients (clinicaltrials.gov) - P1/2 | N=18 | Not yet recruiting | Sponsor: NovelMed Therapeutics | Trial completion date: Jul 2025 ➔ Jul 2026 | Trial primary completion date: Mar 2025 ➔ Feb 2026

Trial completion date • Trial primary completion date • Complement-mediated Rare Disorders • Glomerulonephritis

Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy (clinicaltrials.gov) - P2 | N=12 | Not yet recruiting | Sponsor: NovelMed Therapeutics | Trial completion date: Nov 2025 ➔ Sep 2026 | Trial primary completion date: Jul 2025 ➔ Apr 2026

Trial completion date • Trial primary completion date • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • HP

Ruxoprubart (NM8074) Scores FDA Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment (GlobeNewswire) - "NovelMed today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Ruxoprubart, an alternative pathway (AP) blocker anti-Bb antibody, for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). FDA's recognition of Ruxoprubart as an orphan drug for PNH underscores its potential to fulfill a crucial need for individuals grappling with this disease condition."

Orphan drug • Anemia • Immunology • Paroxysmal Nocturnal Hemoglobinuria

Study of NM8074 in Patients With Anti-Neutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis (AAV) (clinicaltrials.gov) - P2 | N=12 | Not yet recruiting | Sponsor: NovelMed Therapeutics

New P2 trial • ANCA Vasculitis • Vasculitis

Study of NM8074 in Patients With aHUS With Evidence of Ongoing Thrombotic Microangiopathy (clinicaltrials.gov) - P2 | N=12 | Not yet recruiting | Sponsor: NovelMed Therapeutics | Trial completion date: Oct 2025 ➔ Feb 2026 | Initiation date: Jun 2024 ➔ Oct 2024 | Trial primary completion date: Aug 2025 ➔ Dec 2025

Trial completion date • Trial initiation date • Trial primary completion date • Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Nephrology • HP

Study of NM8074 in Adult C3 Glomerulopathy Patients (clinicaltrials.gov) - P1/2 | N=18 | Not yet recruiting | Sponsor: NovelMed Therapeutics | Trial completion date: Jan 2025 ➔ Jul 2025 | Initiation date: Sep 2023 ➔ Jan 2024 | Trial primary completion date: Nov 2024 ➔ Mar 2025

Trial completion date • Trial initiation date • Trial primary completion date • Complement-mediated Rare Disorders • Glomerulonephritis

Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris (clinicaltrials.gov) - P2 | N=12 | Not yet recruiting | Sponsor: NovelMed Therapeutics | Trial completion date: Nov 2024 ➔ Dec 2025 | Initiation date: Sep 2023 ➔ Mar 2024 | Trial primary completion date: Oct 2024 ➔ Aug 2025

Trial completion date • Trial initiation date • Trial primary completion date • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy (clinicaltrials.gov) - P2 | N=12 | Not yet recruiting | Sponsor: NovelMed Therapeutics | Trial completion date: Nov 2024 ➔ Nov 2025 | Initiation date: Nov 2023 ➔ May 2024 | Trial primary completion date: Oct 2024 ➔ Jul 2025

Trial completion date • Trial initiation date • Trial primary completion date • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Study of NM8074 in Adult C3 Glomerulopathy Patients (clinicaltrials.gov) - P1/2 | N=18 | Not yet recruiting | Sponsor: NovelMed Therapeutics | Trial completion date: Oct 2024 ➔ Jan 2025 | Initiation date: Jun 2023 ➔ Sep 2023 | Trial primary completion date: Aug 2024 ➔ Nov 2024

Trial completion date • Trial initiation date • Trial primary completion date • Complement-mediated Rare Disorders • Glomerulonephritis

Study of NM8074 in Soliris-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) (clinicaltrials.gov) - P2 | N=6 | Not yet recruiting | Sponsor: NovelMed Therapeutics

New P2 trial • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Anti-Bb Antibody (NM8074) Receives US FDA Clearance to Start Clinical Trial in Naïve aHUS Patients (Rare Disease) (GlobeNewswire) - "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....NM8074 has recently completed a Phase I trial in healthy volunteers with no safety concerns...."

New trial • Rare Diseases

Study of NM8074 in Adult PNH Patients (clinicaltrials.gov) - P2 | N=12 | Not yet recruiting | Sponsor: NovelMed Therapeutics | Trial completion date: Jun 2024 ➔ Nov 2024 | Initiation date: Feb 2023 ➔ Sep 2023 | Trial primary completion date: Feb 2024 ➔ Oct 2024

Trial completion date • Trial initiation date • Trial primary completion date • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Study of NM8074 in Patients With aHUS (clinicaltrials.gov) - P2 | N=12 | Not yet recruiting | Sponsor: NovelMed Therapeutics

New P2 trial • Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Nephrology • HP