As a funding drought for biopharma is on the horizon, non-profit biotech Orphan Therapeutics Accelerator is championing an innovative funding and partnership strategy to save shelved therapies for ultra-rare diseases.
The often higher costs and lower profits for rare disease candidates place a particular burden on drug development in this area. More recently, government funding in the US has been restricted with the Trump administration introducing cuts at the National Institutes of Health, which are expected to negatively impact developers who struggle to keep rare disease programmes running. Several charitable nonprofits such as the Telethon Foundation have stepped in to drive such programmes instead of commercial interest.
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At the upcoming Interphex 2025 conference, Orphan Therapeutics Accelerator partnerships and innovation vice-president Deanna Portero will discuss the role that such nonprofits could play in spurring drug development for rare disorders. In a conversation with Pharmaceutical Technology, Portero talks about the alternate partnership models and funding strategies for drug developers in the rare disease space.
This interview has been edited for length and clarity.
Frankie Fattorini (FF): What is the aim of the Orphan Therapeutics Accelerator?
Deanna Portero (DP): Our goal at Orphan Therapeutics Accelerator is to provide a sustainable path for the development and access of patients to ultra-rare therapies. We develop an ecosystem of strategic partnerships with organisations like CDMOs (contract development and manufacturing organisations) and CROs (contract research organisations) under either lower cost or cost-deferred risk-sharing innovative agreements, which allow us to push the cost curve deeper into the revenue-generating phase for these products. Those two strategies combined lay a lot of the foundation for how we aim to rescue stalled clinical stage assets in the ultra-rare space.
FF: How can risk-sharing and innovative incentives bring down development costs?
DP: When you look at the projected revenue from ultra-rare disease therapies and the projected expense of keeping them on the market, they are profitable assets. The distinction is that if you are utilising traditional investor capital, they may not be able to provide the return on investment that that capital requires.
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By GlobalDataOften therapies with excellent Phase I/II data are inaccessible to patients because they’re not profitable enough. From a humanitarian lens, we don’t find that to be acceptable.
By incorporating ourselves as a 501(c)(3) nonprofit, we’re able to access sources of capital that are non-dilutive [and] charitable from a variety of partners. We are more successful in working with venture philanthropy, and social impact capital.
FF: How has the current drug development environment hampered candidates for ultra-rare diseases?
DP: Cell and gene therapy modalities are quite suited to targeting monogenic diseases, the majority of which are ultra-rare diseases; but the economics of targeting those diseases isn’t. We saw this as interest rates went up, and the increasing policy uncertainty about things like pricing, and [these have] resulted in a drawback in terms of investor interest.
When you have a corporate infrastructure, team and an operation that is capable of bringing a platform-based therapy to market, there’s the opportunity costs issue. Those teams are going to often have a structural pull to focus on larger market therapies. A group like ours thinks about sustainability, we centre the patient, and we’re most concerned with making sure patients with unmet clinical needs are ultimately accessing therapies.
We’re able to make that an equal consideration. Whereas the private sector [needs to] not only maximise profitability downstream but also make sure that they’re pursuing the opportunities that have the greatest potential for return on investment. It is challenging for organisations in those corporate environments to make decisions that are not the most financially rewarding.
FF: Can the Orphan Therapeutics Accelerator’s methods be implemented in the wider industry?
DP: We see evidence of this strategy being employed in the private sector. BridgeBio, for example, has announced that they have an innovative risk-sharing agreement with a CDMO.
We recognise that there’s a lot of untapped capacity right now in the CDMO space. There’s a lot of [manufacturing] build-up, not only in private CDMOs, but a lot of companies [have] built up their own manufacturing capacity, and that goes as leverage. Given our focus, I think we often require less substance and less work than groups that would be pursuing larger indications. So, we think there are a lot of opportunities for this model in the ultra-rare space, but there are more challenges as you look at indications with higher prevalences.
FF: What sort of impact could legislation like the ORPHAN Cures Act have on rare disease development?
DP: We’re in dialogue with and have a lot of admiration for groups such as Every Cure that are leading the way in rare and common disease drug repurposing. We see a lot of merit in extending the orphan drug exclusion provisions in the Inflation Reduction Act (IRA) for drugs that are repurposed specifically for other orphan indications.
Among legislative priorities, our greatest concern is the paediatric rare disease priority review voucher programme. That’s a programme that we feel is truly make-or-break about the economic viability of many ultra-rare diseases. However, we are also very interested in opportunities to innovate in clinical trial design and to identify greater efficiencies, and that includes looking at therapies that may have clinical benefits for multiple rare diseases.
FF: How fundamental are the FDA’s priority review vouchers for rare disease therapeutics?
DP: If we look at how this programme has been utilised, it has allowed small therapeutic developers to target diseases with smaller populations, less commercially attractive diseases and diseases that are more difficult to design clinical trials for, and sell these vouchers to larger pharmaceutical companies for whom the voucher has great economic benefit in certain scenarios.
This is a program that is run at almost no cost to the American taxpayer. This is just the administrative work of a fairly small number of employees at the FDA, but it enables larger pharma essentially to subsidise smaller biotech organisations to focus on these extremely underserved diseases. So we view it as a no-brainer.
This [priority review voucher value] is capital that can be distributed to partners, for example, from risk-sharing agreements. That can also bring groups to the table about the types of in-licensing deals that they’re willing to agree to.
