Orphan Therapeutics Accelerator Announces Foundational Partnerships

Founding Members Chiesi, BIAL Lead Initial Funding;

Landmark Bio, Uncommon Cures Join Clinical Development Network;

DVLP Medicines, Vibe Bio to Provide Integrated AI-based Solutions

The Orphan Therapeutics Accelerator (OTXL), a non-profit biotech organization focused on completing development and commercializing therapies for ultra-rare conditions, today announced collaborative partnerships with rare disease-focused global biotech and pharmaceutical companies, a contract development and manufacturing organization (CDMO), contract research organization (CRO), and AI-based platform providers, establishing the foundation for a new development and commercialization pathway for ultra-rare disease treatments.

Chiesi and BIAL have committed funding and will become the first OTXL Founding Members, helping to guide and support selection and late-stage development of a set of initial programs expected to be announced in the coming months.

Landmark Bio will help to lower CMC development and manufacturing costs and Uncommon Cures will work together with OTXL to streamline clinical trial processes. DVLP Medicines is providing an end-to-end drug development platform through an early access program, utilizing autonomous AI agents to perform complex analysis and generate plans, reports and other tools for expert use, at a fraction of the time and cost ordinarily required. Vibe Bio will provide AI-based solutions for due diligence and analysis of potential therapeutic assets, enabling highly efficient clinical development, asset management, and other vital functions. These organizations will be the first to participate in Orphan ClinDevNet, a connected ecosystem of partners OTXL will rely on for clinical- and commercial-stage services.

The Orphan Therapeutics Accelerator was established in June 2024 to overcome significant challenges facing ultra-rare disease drug development and commercialization in recent years, driven by shifting financial, market and policy dynamics, which have caused many promising investigational treatments to be abandoned or de-prioritized.

OTXL is focused on obtaining rights to these shelved therapeutic programs, advancing them efficiently and at low cost through clinical trials utilizing Orphan ClinDevNet partners, and providing commercial access through several potential pathways, including directly from academic or non-profit institutions, via spinouts under an affiliated LLC, or out-licensed to mission-aligned biotech partners.

“We’re excited to help launch this much-needed collaborative venture,” said Giacomo Chiesi, Head of Global Rare Diseases at Chiesi. “The recent market dislocation and capital rotation out of biotech have prevented many rare disease research programs from moving forward. We believe rare diseases are a long-term societal responsibility and, with this collaboration, we commit to build partnerships across stakeholders to meet these newly emerging challenges of ultra-rare diseases. Together, we can drive the innovation patients deserve.”

“There is a lot of promising science sitting on the sidelines that could be benefitting children and others facing rare diseases,” said Smitha Jagadish, Head of Rare Diseases of BIAL. “Often these treatments have delivered meaningful results for patients in clinical trials but research in the field of rare diseases cannot always be approached from a traditional perspective. Our aim in supporting this bold effort is to get more of these treatments back into clinical trials so they may deliver meaningful impact for patients as well as substantial value to those who invest in their development.”

“Many of us involved in this venture have been working in rare diseases for decades,” stated Craig Martin, Founder and CEO of the OTXL. “We’ve seen the ebbs and flows and know that this recent stretch isn’t merely a ‘market correction,’ it’s a sea change that we need to radically adjust our thinking to confront. We are laser-focused on aggressively addressing the need, at scale, and taking advantage of opportunities as a non-profit to work with regulators, payors, investors and other stakeholders to completely shift the model to work better for patients and families.”

“The landscape for rare diseases has evolved considerably over the past 40 years, but there’s still a great need for new ways to advance promising ultra-orphan therapies,” said Jim Geraghty, OTXL Board Chair. “I hope OTXL and its partners can make an important contribution to this need.”

Funding from Chiesi, BIAL and others will be used to complete evaluations, secure and initiate work on a set of 2-3 lead programs. Additional fundraising is underway for these and other portfolio programs to follow. A portion of the revenue generated from out-licensing or commercializing programs will be returned to the Accelerator to fund more programs as the model is established and expanded.

The Accelerator expects to be self-sustaining within 4-6 years. In so doing, the non-profit can help buffer future portfolios of abandoned ultra-rare disease therapies from market fluctuations, grow to scale and get dozens of these needed therapies to patients rapidly and efficiently.

About the Orphan Therapeutics Accelerator

The Orphan Therapeutics Accelerator (OTXL) is a patient-centered non-profit biotech focused on obtaining and reinitiating development of promising “shelved” clinical-stage treatments for ultra-rare conditions and providing a scalable and sustainable commercialization path to get these treatments to patients. OTXL leverages its non-profit status, success-based agreements and incentives to obtain rights to shelved programs and complete development at deferred or lower cost via a network of affiliated CDMOs, CROs and other partners. When a program is approved and generates revenue, net proceeds are used to repay and reward contributing partners, companies, and institutions, with a portion returned to the Orphan Therapeutics Accelerator to fund additional programs. Our team is deeply familiar with challenges faced in developing and commercializing treatments for rare conditions and committed to overcoming them. For more information, please visit orphantxl.com.

About Our Founding Members

Chiesi Group is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment.

By changing its legal status to a Benefit Corporation in Italy, the US, France and Colombia, Chiesi’s commitment to creating shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, Chiesi is part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035.

With 90 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,500 employees. The Group’s research and development centre in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden.

BIAL is a hundred-year-old innovation-driven biopharmaceutical company aiming to improve people’s lives worldwide. Key focus areas for the company are neurosciences and rare diseases. In Europe, BIAL has a production site and R&D unit in Portugal (headquarters) and affiliates in Spain, Germany, the United Kingdom, Italy, and Switzerland as well as in the US and some emerging markets. BIAL products are present in fifty countries, fulfilling its purpose of making a real difference in the lives of people living with severe diseases across the world. For more information about BIAL, please visit: www.bial.com.

About Our Partners

Landmark Bio was established to remove barriers in drug development, create accessible capability, expertise, and solutions, and offer a collaboration platform to advance manufacturing technologies for the new generation of medicines. (landmarkbio.com)

Uncommon Cures is clinical trial program that is dedicated to expediting the development of orphan drugs through innovative clinical trial design, streamlined clinical trial processes, and strategic relationships with relevant stakeholders and like-minded organizations. (uncommoncures.com)

DVLP Medicines uses autonomous AI agents to develop drugs by digitizing and de-enterprising all aspects of development, from company formation to funding to regulatory review, making it possible to advance molecules that were sidelined—not because they were ineffective therapies but because drug development is so costly and difficult that only a few high-priced medicines ever reach the clinic. (dvlpmedicines.com)

Vibe Bio uses AI to analyze and invest in promising drug programs. Vibe Bio’s pipeline analysis platform, VibeOne, uses artificial intelligence to help pharmaceutical companies use due diligence in drug research and development to understand strengths, risks, and probability of success. (vibebio.com)

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