Uncovering New Value in High-Quality Drugs to Cure Rare Diseases and Underserved Medical Conditions
Capital and Business Alliance with GEXVal Inc.

The Alfresa Group has formulated its Medium-to Long-Term Vision outlining its strategic direction through to fiscal 2032. The Group’s four business segments are working together to enhance and expand total supply chain services, which provide seamless, end-to-end support from the development and manufacturing of pharmaceuticals to their distribution, dispensing at pharmacies, and ultimately reaching patients.

In April 2023, Alfresa Holdings Corporation established a new department, the Total Supply Chain Services Business Planning & Development Department, to advance this initiative. Mahito Hirai, who was previously with Alfresa Pharma Corporation, Alfresa Group’s pharmaceutical manufacturing business, was seconded to head this new department. He has since been leading the team alongside newly appointed members.
Hirai began his career in research and development (R&D) in the healthcare division of a major Japanese electronics manufacturer. He later transitioned to biotech ventures and other industries, gaining experience across a variety of roles. Most recently, he has been involved in project management, licensing negotiations, and alliance management for our R&D division, building extensive external networks in business development.
The Total Supply Chain Services Business Planning & Development Department is focused on building a system to facilitate smooth collaboration across the Alfresa Group companies and addressing challenges in specific projects with R&D-driven companies at home and abroad. The team works collectively to accomplish these objectives. As part of its efforts to explore new projects, the department participates in biotech partnering events in Japan and overseas, engaging in strategic networking to facilitate partnerships with companies looking to enter the Japanese pharmaceutical market. Hirai’s diverse experience and extensive network have been instrumental in these activities, contributing to the establishment and operation of the total supply chain services the Alfresa Group aims to offer.

His encounter with GEXVal Inc. dates back to the end of 2021. Hirai was deeply moved by GEXVal’s corporate philosophy of creating a society where patients and families living with rare diseases and underserved medical conditions can access innovative drugs more quickly. Hirai engaged in multiple discussions with GEXVal’s President & CEO, Juran Kato, Ph.D., and her team members, ultimately leading to the capital and business alliance between Alfresa Holdings and GEXVal. We spoke to Hirai in detail regarding this course of events.

2021: The First Encounter with GEXVal Inc.

Hirai’s introduction to GEXVal came via a meeting with Nobuaki Hirashima, GEXVal’s Director and Chief Business Officer (CBO), with whom he had a long-standing relationship in the pharmaceutical industry. He was particularly intrigued by GEXVal’s origins as a spin-out from Takeda Pharmaceutical Company Limited and found its business model compelling. Upon learning that GEXVal—driven by its patient-first approach and guided by its corporate philosophy—was actively engaged in developing new drugs, Hirai began exploring potential opportunities for collaboration. This led to extensive exchanges of information on the progress of GEXVal’s drug development efforts.

The pharmaceutical industry is home to many dormant yet promising drug candidates—hidden gemstones with untapped potential. GEXVal’s strength lies in its unique ability to uncover and refine such hidden gemstones using its proprietary technologies, delivering much-needed medicines to patients more quickly. In other words, GEXVal makes itself different from others by uniquely enabling a “circular economy” within the pharmaceutical industry—an economy in which overlooked but potentially valuable seeds are put back into the drug discovery process. The company’s new lead drug candidate, GXV-001, is an orphan drug for rare diseases, currently in the early stages of development. Its target indications are Fragile X syndrome, Rett syndrome, and postoperative delirium.

Hirai’s seconding company, Alfresa Pharma Corporation, upholds the management philosophy of “actively taking on the challenge of the new ‘Unmet Medical Needs’^*1 of the times.” The definition of rare diseases is not standardized worldwide, but it is estimated that there are about 300 million patients worldwide, with more than 6,000 identified rare diseases. The reality is that 95% of these diseases still lack established treatments^*2. Hirai is deeply committed to supporting patients and their families who are struggling with such conditions.

Reference
Notice Regarding Capital and Business Alliance with GEXVal Inc. (January 16, 2025.)

Alfresa Pharma Corporation’s website – Basic Philosophy page

*1. Refers to medical needs for conditions for which there is demand but no effective treatment available.

*2. Source: Survey on the Challenges Faced by Patients with Rare Diseases, Japan Pharmaceutical Manufacturers Association (JPMA)(available in Japanese only)

Indications for the New Lead Drug Candidate, GXV-001

Fragile X syndrome, a designated intractable disease, is caused by genetic abnormalities. Its primary symptoms include developmental disorders such as intellectual disability, autistic tendencies, attention deficit, hyperactivity, and aggression. Additionally, patients may experience sleep disturbances, anxiety, depression, and epilepsy. Some individuals also exhibit certain physical characteristics, such as a long face and large ears, though symptoms vary widely from person to person. A genetic test is required for a definitive diagnosis. In Japan, it is estimated that around 5,000 patients have Fragile X syndrome. However, the diagnosis rate remains extremely low compared to Western countries, as many cases go undiagnosed. Currently, no approved treatments exist, but various research and development efforts are underway. The development of effective therapeutic drugs is expected to promote early diagnosis and treatment, underscoring their importance.

Rett syndrome, another designated intractable disease that primarily affects girls, is a progressive neurological disorder caused by genetic mutations. While early development appears normal for the first 6 months to 2 years after birth, previously acquired language and motor skills gradually deteriorate. A hallmark symptom is loss of hand function, often causing the hands to remain in a clenched position or display repetitive, washing-like hand movements, making it difficult for individuals to use their hands in daily activities. Other autonomic nervous system-related symptoms associated with the condition include walking difficulties, sleep disturbances (often accompanied by nighttime agitation and loud vocalizations), epilepsy, breathing irregularities (such as apnea or hyperventilation), scoliosis (spinal curvature), and severe constipation. While symptomatic treatments are currently available, no therapies exist to halt the progression of the disease.

Postoperative delirium is a temporary state of confusion triggered by surgical procedures. After surgery, patients initially seem calm, but sudden changes emerge within a few days. Symptoms may be hyperactive in nature (characterized by agitation, pacing, or yelling), hypoactive (where the patient becomes lethargic, foggy, or unresponsive), or a mixture of both, sometimes accompanied by hallucinations or delusions. Postoperative delirium is especially common in elderly patients and can hinder recovery, treatment, and nursing care. Additionally, it has been linked to prolonged hospital stays, increased risk of dementia progression, and higher mortality rates. For these reasons, early detection and appropriate intervention are essential.

Supporting Improved Access to Pharmaceuticals Through Total Supply Chain Services

Hirai speaks passionately about the strengthening and expansion of total supply chain services, stating “This is not only my core mission but also a vision for shaping the future of the Alfresa Group.”

Hirai continued: “There are many hurdles in drug development, but through this capital and business alliance, Alfresa Pharma can leverage its expertise in drug development to support everything from co-development in neurological diseases to the manufacturing and sale of GXV-001. Additionally, the Ethical Pharmaceuticals Wholesaling Business segment of the Alfresa Group can provide support for domestic transportation and distribution. I believe that one of our Group’s key roles is to integrate and offer these capabilities as a comprehensive service.” Looking ahead, he emphasizes: “By maintaining close collaboration, we will continue working to improve access to pharmaceuticals so that patients and families living with rare diseases and underserved medical conditions can receive innovative drug treatments as quickly as possible.”

Notice: This document is a translation of the original Japanese document and is for reference purposes only. In the event of any discrepancy between this translated document and the original Japanese document, the latter shall prevail.