Aurinia Announces First Participant Dosed in AUR200 Single Ascending Dose Trial

Rhea-AI Summary

Aurinia Pharmaceuticals Inc. (NASDAQ: AUPH) has announced the dosing of the first participant in a Phase 1a single ascending dose (SAD) study of AUR200, a potential best-in-class therapy for autoimmune diseases. AUR200 targets both BAFF and APRIL, and is believed to be more potent with higher binding affinity compared to similar molecules. The study aims to assess safety, tolerability, pharmacokinetics, and biomarker changes in healthy volunteers, with data expected in the first half of 2025.

Aurinia plans to develop AUR200 for diseases with few market entrants, including one larger indication and one smaller indication meeting FDA criteria for orphan and rare diseases. The company will fund this development using available cash flow, without impacting post-restructuring operating expense targets.

Positive

• First participant dosed in Phase 1a single ascending dose study for AUR200
• AUR200 potentially a best-in-class therapy for autoimmune diseases
• AUR200 believed to be more potent with higher binding affinity than similar molecules
• Development to focus on diseases with few market entrants
• Funding from available cash flow, not impacting post-restructuring expense targets

Negative

• Data from the Phase 1a study not expected until first half of 2025
• Specific indications and development plans not yet communicated

Medical Research Analyst

The initiation of the Phase 1a single ascending dose (SAD) study for AUR200 marks a significant milestone in Aurinia's pipeline development. This novel dual-targeting therapy aims to inhibit both BAFF and APRIL, key factors in B-cell-mediated autoimmune diseases. The potential for AUR200 to be a best-in-class treatment is noteworthy, especially given the claimed higher potency and binding affinity compared to similar molecules.

However, investors should note that data is not expected until the first half of 2025, indicating a long timeline before potential commercialization. The company's strategy to target both a larger indication and a smaller, orphan disease is prudent, potentially balancing market size with faster regulatory pathways. The use of existing cash flow for development, without impacting post-restructuring expense targets, suggests efficient financial management.

Financial Analyst

From a financial perspective, Aurinia's approach with AUR200 appears strategically sound. By leveraging existing cash flow for development without altering expense targets, the company demonstrates fiscal discipline. The dual-pronged indication strategy could potentially maximize return on investment by balancing market size with development speed.

However, investors should be cautious about the extended timeline to data readout in 2025, which may impact short-term stock performance. The lack of specific indication targets at this stage also introduces uncertainty. While the potential for a best-in-class therapy is promising, it's important to monitor the competitive landscape and any interim updates that might affect the company's market position and valuation.

ROCKVILLE, Md. & EDMONTON, Alberta--(BUSINESS WIRE)-- Aurinia Pharmaceuticals Inc. (NASDAQ: AUPH) (Aurinia or the Company) announced today that the first participant has been dosed in a Phase 1a single ascending dose (SAD) study of AUR200, a differentiated, potential best-in-class therapy for autoimmune diseases that targets both BAFF (B-cell Activating Factor) and APRIL (A Proliferation-Inducing Ligand).

The SAD study will assess the safety, tolerability, pharmacokinetics, and changes in biomarkers for AUR200 in healthy volunteers, with data expected in the first half of 2025.

“The start of the single ascending dose study is an important milestone for developing AUR200, which has the potential to serve as a best-in-class treatment in autoimmune diseases with high unmet need. We believe AUR200 is a more potent compound with a higher binding affinity compared to the two other TACI-Fc molecules designed to bind both BAFF and APRIL that were tested in our preclinical research. We expect the data from this early-stage study to provide key points of differentiation that will inform further clinical development efforts,” said Dr. Greg Keenan, Chief Medical Officer of Aurinia.

The Company intends to develop AUR200 in disease states where there are currently few market entrants, including one larger indication and one fast-to-market smaller indication that meets the FDA criteria for orphan and rare diseases. The Company will communicate specific indications and development plans pending progress and outcomes of early-stage clinical trials and based on ongoing assessment of the competitive landscape in relevant indications. The Company anticipates funding this development program with available cash flow, which is not anticipated to impact previously announced post-restructuring operating expense targets.

About AUR200

AUR200, a highly potent and specific immune modulator, is an IgG4 Fc-fusion protein with no appreciable effector function. AUR200 contains a unique, structurally engineered B-cell maturation antigen (BCMA) domain for enhanced binding to both BAFF and APRIL, two cytokines that play important roles in regulating B-cell survival and differentiation. Dual inhibition of BAFF and APRIL is a clinically validated mechanism that has demonstrated great therapeutic potential for a wide range of autoimmune diseases.

In animal data presented at the annual American College of Rheumatology Convergence 2022, AUR200 dosed therapeutically reduced several markers of disease activity and improved overall survival in a mouse model of lupus. AUR200 was also well-tolerated in both mice and cynomolgus monkeys, with no adverse effects. These findings highlight the potential value of AUR200 in the treatment of autoimmune diseases.ⁱ

About Aurinia

Aurinia Pharmaceuticals is a fully integrated biopharmaceutical company focused on delivering therapies to people living with autoimmune diseases with high unmet medical needs. In January 2021, the Company introduced LUPKYNIS^® (voclosporin), the first FDA-approved oral therapy dedicated to the treatment of adult patients with active lupus nephritis. The Company’s head office is in Edmonton, Alberta, with its U.S. commercial office in Rockville, Maryland. The Company focuses its development efforts globally.

Forward-Looking Statements

Certain statements made in this press release may constitute forward-looking information within the meaning of applicable Canadian securities law and forward-looking statements within the meaning of applicable United States securities law. These forward-looking statements or information include but are not limited to statements or information with respect to: Aurinia’s belief that AUR200 is a potential best-in-class therapy for autoimmune diseases that targets both BAFF and APRIL; Aurinia’s expectation that data from the SAD study will be available in the first half of 2025; Aurinia’s belief that AUR200 has the potential to serve as a best-in-class treatment in autoimmune diseases with high unmet need; Aurinia’s belief that AUR200 is a more potent compound with a higher binding affinity compared to other TACI-Fc molecules designed to bind both BAFF and APRIL; Aurinia’s expectations that the SAD study will provide key points of differentiation that will inform further clinical development efforts; Aurinia’s intention to develop AUR200 in diseases states where there are currently few market entrants; and Aurinia’s anticipation to fund the AUR200 development program with available cash flow. It is possible that such results or conclusions may change. Words such as “anticipate”, “will”, “believe”, “estimate”, “expect”, “intend”, “target”, “plan”, “goals”, “objectives”, “may” and other similar words and expressions, identify forward-looking statements. We have made numerous assumptions about the forward-looking statements and information contained herein, including among other things, assumptions about: the burn rate of Aurinia’s cash for operations; that pre-clinical data for AUR200 will be reproduceable in clinical studies; and that Aurinia’s third party service providers will comply with their contractual obligations. Even though the management of Aurinia believes that the assumptions made, and the expectations represented by such statements or information are reasonable, there can be no assurance that the forward-looking information will prove to be accurate.

Forward-looking information by their nature are based on assumptions and involve known and unknown risks, uncertainties and other factors which may cause the actual results, performance, or achievements of Aurinia to be materially different from any future results, performance or achievements expressed or implied by such forward-looking information. Should one or more of these risks and uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those described in forward-looking statements or information. Such risks, uncertainties and other factors include, among others, the following: Aurinia’s actual future financial and operational results may differ from its expectations; Aurinia may have to pay unanticipated expenses; the future prospects for AUR200 may not be as Aurinia has anticipated, or Aurinia may not be able to fully capitalize on the opportunities presented by AUR200; and Aurinia’s third party service providers may not, or may not be able to, comply with their obligations under their agreements with Aurinia. Although Aurinia has attempted to identify factors that would cause actual actions, events, or results to differ materially from those described in forward-looking statements and information, there may be other factors that cause actual results, performances, achievements, or events to not be as anticipated, estimated or intended. Also, many of the factors are beyond Aurinia’s control. There can be no assurance that forward-looking statements or information will prove to be accurate, as actual results and future events could differ materially from those anticipated in such statements. Accordingly, you should not place undue reliance on forward-looking statements or information.

All forward-looking information contained in this press release is qualified by this cautionary statement. Additional information related to Aurinia, including a detailed list of the risks and uncertainties affecting Aurinia and its business, can be found in Aurinia’s most recent Annual Report on Form 10-K available by accessing the Canadian Securities Administrators’ System for Electronic Document Analysis and Retrieval (SEDAR) website at www.sedarplus.ca or the U.S. Securities and Exchange Commission’s Electronic Document Gathering and Retrieval System (EDGAR) website at www.sec.gov/edgar, and on Aurinia’s website at www.auriniapharma.com.

^{___________________________________
i} Morales S, Cross J, Huizinga R. AUR200: An Improved BAFF/APRIL Inhibitor with Increased Potency and Safety for the Treatment of B Cell-Mediated Diseases [abstract]. Arthritis Rheumatol. 2022; 74 (suppl 9). https://acrabstracts.org/abstract/aur200-an-improved-baff-april-inhibitor-with-increased-potency-and-safety-for-the-treatment-of-b-cell-mediated-diseases/. Accessed December 6, 2023.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240905970274/en/

Media & Investor Inquiries:

Andrea Christopher,

Corporate Communications & Investor Relations, Aurinia

achristopher@auriniapharma.com

General Investor Inquiries:

ir@auriniapharma.com

Source: Aurinia Pharmaceuticals Inc.

FAQ

What is the purpose of Aurinia's Phase 1a study for AUR200?

The Phase 1a single ascending dose (SAD) study aims to assess the safety, tolerability, pharmacokinetics, and changes in biomarkers for AUR200 in healthy volunteers.

When are the results of Aurinia's AUR200 Phase 1a study expected?

The data from the Phase 1a study of AUR200 is expected in the first half of 2025.

What makes AUR200 potentially best-in-class for autoimmune diseases?

AUR200 is believed to be more potent with a higher binding affinity compared to other TACI-Fc molecules designed to bind both BAFF and APRIL, potentially making it a best-in-class treatment for autoimmune diseases.

How will Aurinia fund the development of AUR200?

Aurinia plans to fund the development of AUR200 using available cash flow, without impacting previously announced post-restructuring operating expense targets.

What types of indications is Aurinia targeting for AUR200 development?

Aurinia intends to develop AUR200 for diseases with few market entrants, including one larger indication and one smaller indication that meets FDA criteria for orphan and rare diseases.