cBio and Alesta Sign Global Licensing Deal for Novel Hypophosphatasia Treatment

10 January 2025
SAN FRANCISCO and LEIDEN, Netherlands, Jan. 8, 2025 – 1cBio, Inc. and Alesta Pharmaceuticals have entered into a global licensing agreement granting Alesta the rights to develop, commercialize, and manufacture OC-1, a potential therapy for hypophosphatasia (HPP). This collaboration is aimed at addressing the significant unmet needs associated with this rare genetic disorder.

Hypophosphatasia is caused by mutations in the ALPL gene, leading to impaired bone and tooth mineralization. Patients with HPP experience a range of symptoms, including skeletal deformities, fragile bones, premature tooth loss, and in older individuals, muscle weakness, fatigue, and pain. The disease is often underdiagnosed, particularly among the elderly, but is believed to affect tens of thousands globally.

OC-1, also referred to as ALE1, is an innovative oral treatment targeting the reduction of inorganic pyrophosphate (PPi) levels, a metabolite linked to disease progression. The compound is currently undergoing GLP toxicology studies, with plans to initiate clinical trials in the latter half of 2025. The development of OC-1 was driven by 1cBio's adeptness in medicinal chemistry and translational sciences, coupled with collaboration with experts in clinical and basic sciences.

Andrew Wong, CEO of 1cBio, expressed enthusiasm about the partnership with Alesta, highlighting the potential of OC-1 to fill a critical gap in HPP treatment. He emphasized the partnership's capability to expedite the delivery of this promising therapy to patients. Wong described OC-1 as a testament to 1cBio's ability to innovate in precision medicine by identifying underserved areas in disease treatment.

Ilan Ganot, CEO of Alesta Therapeutics, shared the company's commitment to advancing OC-1 towards clinical trials, with the aim of providing a comprehensive solution for HPP. Ganot underscored the collaboration's potential impact on the lives of HPP patients, emphasizing the blend of expertise from both companies and partnerships with academia and disease advocacy groups.

According to the terms of the agreement, Alesta will manage Phase I preparation activities, including the submission of the necessary regulatory documentation, such as the Clinical Trial Application (CTA) and Investigational New Drug (IND) application. Alesta will also oversee all phases of clinical development and global commercialization. In return, 1cBio stands to receive milestone payments linked to development and regulatory achievements and tiered royalties from global sales, contingent on successful outcomes.

1cBio is a burgeoning biotech firm focused on discovering and developing precision medicines, particularly in cancer therapies. The company utilizes its strengths in medicinal chemistry and translational sciences to create molecules that counteract disease progression and resistance mechanisms. Alongside OC-1, 1cBio is advancing OC-3, a selective-PARP1 inhibitor designed to be potentially best-in-class for treating HR-deficient and HR-proficient tumors in combination with approved therapies.

Alesta Therapeutics, headquartered in the Netherlands, is dedicated to creating oral small molecule therapies for rare diseases. Alongside their work on ALE1 for HPP, the company is developing ALE2 for Charcot-Marie-Tooth disease, another condition with unmet medical needs. In January 2025, Alesta secured €65 million through a Series A financing round, backed by prominent biotech and life science investors.

This strategic collaboration between 1cBio and Alesta represents a significant step towards providing a viable treatment option for patients suffering from hypophosphatasia and exemplifies the potential of innovative partnerships in the biotech industry.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!