1cBio and Alesta Therapeutics Announce Exclusive Global Licensing Agreement for a Novel Potentially First-in-Class Small Molecule for Hypophosphatasia

SAN FRANCISCO and LEIDEN, Netherlands, Jan. 8, 2025 /PRNewswire/ -- 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).

HPP is a rare genetic disorder caused by mutations in the ALPL gene, leading to defective bone and tooth mineralization. This results in skeletal abnormalities, fragile bones, early tooth loss, and, in older patients, significant muscle weakness, fatigue, and pain. Affecting tens of thousands of patients worldwide, HPP is believed to be underdiagnosed, particularly in older populations. OC-1 is a novel oral therapeutic that inhibits a novel target to reduce levels of inorganic pyrophosphate (PPi), a key metabolite driving disease pathology. Currently progressing through GLP toxicology studies, OC-1 is expected to enter clinical trials in the second half of 2025.

OC-1 was developed by combining 1cBio's expertise in medicinal chemistry and translational sciences with thought partners in clinical and basic sciences to guide its innovation.

"We are excited to partner with Alesta Therapeutics, who is pioneering the development of transformative medicines for rare disease and can help us to reach HPP patients more rapidly," said Andrew Wong, Chief Executive Officer at 1cBio.  "OC-1 is another example of the 1cBio team's capabilities to identify white spaces in disease treatments and develop highly differentiated molecules for precision medicine. We look forward to collaborating with Alesta's innovative development team to efficiently progress a potentially first-in-class, oral therapy for HPP patients."

"With the support of 1cBio, the expertise and commitment of our team, and collaborations with leading academics and disease advocacy groups, we are poised to make significant strides in addressing the unmet needs of HPP patients," said Ilan Ganot, Chief Executive Officer of Alesta Therapeutics.  "We look forward to advancing OC-1 toward clinical proof of concept and bringing us closer to delivering this transformative therapy to address the entire spectrum of HPP in this underserved disease."

Under the terms of the agreement, Alesta will assume responsibility 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.

About 1cBio
1cBio is an emerging biotechnology company which strives to identify white spaces in cancer therapies and develop molecules that can serve as precision medicines. The company's programs are geared to treat the disease and preemptively counter mechanisms for acquired or intrinsic resistance. Core expertise in medicinal chemistry and translational sciences along with thought partners in clinical and basic sciences guides 1cBio's innovation. 1cBio's second lead program is OC-3, a selective-PARP1 inhibitor with the potential to be best-in-class for HR-deficient and HR-proficient tumors when combined with approved targeted therapies. For more information, visit https://1cbio.com.

About Alesta Therapeutics
Alesta Therapeutics is a Netherlands-based biotechnology company committed to developing novel oral small molecule therapies for underserved rare diseases. The company's lead asset, ALE1, is being developed for hypophosphatasia (HPP), a rare genetic disorder with significant unmet needs. Alesta is also advancing ALE2, a promising candidate to treat Charcot-Marie-Tooth (CMT) disease. In January 2025 Alesta announced a €65 million Series A financing supported by leading biotech and life science investors. For more information, visit https://alestatherapeutics.com/.

SOURCE 1cBio