Mergers & Acquisitions (M&A) Support for Advanced Therapies

---

Summary:Specialized guidance for mergers, acquisitions, and divestitures involving rare disease and advanced therapy assets or organizations. Support covers regulatory due diligence focused on rare disease and advanced therapy programs, QMS/SOP integration planning sensitive to specific needs, ensuring post-merger compliance maintains patient focus, risk assessment related to rare disease and advanced therapy assets, and aligning global regulatory strategies to preserve or enhance patient access.

Mergers and acquisitions are increasingly shaping the landscape for rare disease and advanced therapies, as organizations combine expertise, pipelines, or resources. These transactions, whether involving small biotechs, larger companies, or non-profit entities, carry unique regulatory and quality considerations crucial for success and, most importantly, for the continuity of patient care and access. Regal Intel, as a dedicated partner, provides specialized consulting services to guide organizations through the complexities of regulatory due diligence, integration planning, and post-transaction compliance, ensuring transitions are smooth, compliant, and ultimately serve the rare disease community.

We conduct thorough, objective regulatory due diligence specifically focused on the target's rare disease and advanced therapy assets and supporting systems:

• Assessing the status and robustness of orphan designations, pediatric plans, and compliance with any associated commitments.
• Evaluating the target's regulatory history, compliance status (GMP, GCP, GLP), product registrations, and agency interactions related to the rare disease programs.
• Analyzing the integrity and completeness of regulatory submissions (e.g., IND, BLA, MAA, PMA, HDE in eCTD format) and supporting data, especially from potentially small or complex studies.
• Reviewing the target company's QMS for suitability and sustainability in supporting the specific needs of the rare disease product (e.g., ATMP controls, specialized manufacturing).

We assist in developing and executing comprehensive integration plans designed to harmonize systems while preserving the integrity and specialized knowledge crucial for rare disease programs:

• Planning the seamless integration of quality management systems (QMS), regulatory processes, SOPs, and compliance programs, considering the unique aspects of the rare disease therapies involved.
• Managing the efficient transfer of product registrations, licenses, orphan designations, and regulatory approvals.
• Ensuring smooth integration of critical regulatory data, documentation, and knowledge base related to the rare disease assets.
• Prioritizing strategies that minimize disruption to ongoing clinical trials, patient supply, and specialized manufacturing processes.

We provide ongoing support following a merger, acquisition, or divestiture to ensure continued compliance and focus on the acquired rare disease commitments:

• Assisting in updating regulatory submissions, registrations, and documentation to reflect the new entity structure and responsibilities.
• Conducting post-integration audits to verify compliance effectiveness and identify areas for optimization within the combined system supporting the rare disease assets.
• Supporting interactions and meetings with regulatory agencies regarding the transition and ongoing commitments for the rare disease programs.

We identify, evaluate, and develop mitigation plans for risks specifically associated with M&A activities involving rare disease assets:

• Analyzing risks related to maintaining orphan designation status, fulfilling post-marketing requirements, or integrating complex quality systems (e.g., for ATMPs).
• Assessing risks associated with potential loss of specialized expertise, disruption to niche supply chains, or challenges in transferring unique manufacturing processes.
• Developing mitigation strategies centered on patient safety, program continuity, and sustained compliance.

We facilitate the alignment of global regulatory strategies for the combined entity, focusing on creating a cohesive plan that ensures continued or expanded global access for the rare disease therapy, leveraging the strengths of the new organization to better serve the patient community.

• Rare Disease & M&A Expertise: Deep knowledge of global regulatory requirements specifically impacting rare disease assets and programs, combined with a thorough understanding of the M&A process.
• Thorough Due Diligence: Comprehensive assessments focused on identifying potential risks and opportunities within the context of rare disease programs and their unique regulatory status.
• Mission-Focused Integration: Developing and executing integration plans that prioritize compliance, rare disease program continuity, and sustained patient access.
• Ongoing Support: Providing post-transaction support to ensure sustained compliance and continued focus on rare disease commitments.
• Non-Profit Perspective: Offering objective guidance that prioritizes the long-term benefit to the rare disease community and mission continuity over purely commercial outcomes.
• Cost-Effective Guidance: Delivering expert support tailored to the needs and resources within the rare disease ecosystem.