1cBio, Inc. and Alesta Therapeutics
Type: Licensing agreement
In-Licensee: Alesta Pharmaceuticals
Out-Licensor: 1cBio, Inc.
Focus: OC-1
Financial impact: The rapidly growing rare disease market, projected to reach USD 448.48 billion by 2032, sets the stage for 1cBio’s partnership with Alesta to accelerate OC-1 delivery for HPP patients facing limited treatment options.
1cBio, Inc. ("1cBio"), an emerging biotech company developing precision medicines for significant unmet needs, and Alesta Pharmaceuticals ("Alesta"), a leader in developing first-in-class small molecules for rare diseases, announced an exclusive worldwide licensing agreement. Under this agreement, Alesta will develop, commercialize, and manufacture OC-1 (also known as ALE1), an orally active therapeutic candidate for hypophosphatasia (HPP). Alesta will be responsible for Phase I preparation activities, including submission of the Clinical Trial Application (CTA) and Investigational New Drug (IND) application, and all subsequent clinical-development activities and global commercialization activities. 1cBio is eligible to receive potential development, regulatory, and commercial milestone payments, including an equity investment, and tiered royalties on global sales, contingent upon successful development and commercialization.
OC 1 Treatment for Hypophosphatasia
OC-1 (also known as ALE1) is an oral small-molecule therapy developed by 1cBio for the treatment of hypophosphatasia (HPP). It is designed to modulate a key pathway involved in the regulation of inorganic pyrophosphate (PPi), which is abnormally elevated in individuals with HPP. Elevated PPi interferes with the formation of hydroxyapatite crystals, a critical process for normal bone and dental mineralization. By targeting this novel mechanism, OC-1 helps lower PPi levels, addressing the biochemical root of the disease and promoting proper mineralization.
Rare diseases market revenue (2023-2030)
The National Institutes of Health (NIH) recognizes over 7,000 rare diseases, with about 95 percent lacking effective treatments. A significant number of these conditions receive limited attention from medical researchers, resulting in many patients resorting to "off-label" treatments, which are not FDA-approved for their specific condition and can create challenges with insurance coverage. The rare disease market is both appealing and anticipated to expand rapidly. Although approximately 7,000 rare diseases have been identified, only around 130 have approved treatments available. There is a substantial unmet medical need, and existing treatments offer considerable benefits. Governance and regulatory incentives, such as expedited approval processes and enhanced protection under orphan designation, further enhance the market's attractiveness. Swedish Orphan Biovitrum AB, forecasts a 9% growth in the overall market revenue by 2030.
Forecasted market revenue of Rare disease (2023-2030)
Fig 13: Market revenue of Rare disease
Market Impact of This Partnership
The rare disease market is experiencing rapid growth, reflecting both increasing scientific innovation and heightened global focus. The market for rare disease treatments is projected to reach USD 448.48 billion by 2032, with a CAGR of 9.7% between 2025 and 2032. The rare disease diagnostics segment is also expanding significantly, expected to grow at a CAGR of 17.36%, reaching USD 8.23 billion by 2030.
This partnership with Alesta Pharmaceuticals, a company at the forefront of rare disease innovation, positions 1cBio to bring OC-1 to HPP patients more rapidly and effectively. Together, the companies aim to advance a much-needed therapeutic option for a patient population that currently faces limited treatment choices.