Drug Repurposing Market: Realities and Roadblocks

We receive a steady stream of enquiries about opportunities in drug repurposing, often from people who see a short, low risk pathway to large upsides. But, as is often the case, the closer you get, the less attractive a proposition it seems. In general, there’s much less repurposing being undertaken than some may think.

For a patented drug, academic researchers may try new indications and if successful they may try to license to the originator company (providing they submitted a timely patent application for the new use and have not undermined their proposition through public disclosures at conferences or the like before filing), but most are just done for medical research reasons. Very few companies will try to repurpose a patented drug – one example is Invex which is repurposing exenatide, a GLP-1 agonist currently indicated for Type 2 diabetes, (although patents expire by 2024 (EU) and 2026 (USA) providing the opportunity to license to companies other than AstraZeneca, the originator, or to commercialize directly).

For drugs that have been registered for at least one indication and are now off patent, repurposing opportunities divide into two unequal halves, namely those compounds that are still on the market (almost always as a low price generic) and those that were on the market but which were withdrawn. For the former, the challenge is to find a unique dose or delivery system for the new indication otherwise the cheap generic, albeit not registered for the new use, will cannibalize sales. For molecules that have been withdrawn, there has to be a compelling case to re-initiate manufacturing and purchasing access to regulatory data (which may need to be repeated if it is not up to current standards). This is also done for molecules that failed during the development of the initial indication(s), and we have supported clients doing just this; in practice, these are rare.

Looking at public announcements in the repurposing space, it is noteworthy that there aren’t many. A selection of recent press releases include:

  • 2023 - Medable, Inc., a technology platform company for patient-centered clinical trials, entered a partnership with non-profit Every Cure, which officially launched in September 2022 in partnership with the Clinton Global Initiative to scale up an innovative drug repurposing research approach to identify treatments for rare diseases.
  • 2022 - Healx, an AI-powered, patient-inspired drug repurposing company, announced an exclusive option from Ovid Therapeutics, a biopharmaceutical company developing medicines for neurological diseases, to license rights to develop and commercialize gaboxadol, a direct GABAA receptor agonist, originally investigated as an analgesic and anxiolytic. Under the agreement, Healx plans to investigate the compound as part of a potential combination therapy for Fragile X syndrome, as well as a treatment for other indications. Gaboxadol has previously been tested as a monotherapy for Fragile X syndrome in a Phase 2a clinical study by Ovid. Healx plans to find an optimal treatment for the condition by combining gaboxadol with other compounds, including HLX-0201, that have been identified by the company’s proprietary AI platform, Healnet.
  • 2021 - Standigm Inc., an artificial intelligence (AI)-based drug discovery company and SK Chemicals Co., Ltd., a life science and green chemicals company, announced that they found a new rheumatoid arthritis indication for an FDA-approved drug and have filed a patent. This is the first successful result of their open innovation partnership, formed in July 2019. The research collaboration is aimed at identifying novel lead compounds and repurposing existing drugs for rheumatoid arthritis and nonalcoholic steatohepatitis, leveraging Standigm’s AI-powered drug discovery platforms: Standigm BEST™, Standigm Insight™, and Standigm ASK™.
  • 2020 - Acacia Pharma announced that the FDA has approved BARHEMSYS® for the prevention and treatment of PONV in adult patients. The molecule had originally been approved as an anti-psychotic in the 1990s. This is an example of the advantage of repurposing in developing rapid clinical proof-of-concept, although, as is the case for all repurposed drugs, a Phase 3 trial was needed to reach NDA submission.
  • 2020 - In January, BenevolentAI set out to find an existing drug that could be repurposed as a COVID-19 treatment. Although their technology was designed to develop new drugs for disease — not identify new uses for existing medications — it could make this pivot based on its comprehensive data foundations and flexible, disease-agnostic approach. Apparently, within 48 hours, it identified baricitinib — a drug owned by Eli Lilly and approved for rheumatoid arthritis — as the strongest candidate. In May 2022, the FDA approved baricitinib for the treatment of COVID-19 in hospitalized adults requiring supplemental oxygen, non-invasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation.
  • 2020 - Repurpose.AI (now Model Medicines), an AI drug discovery company, launched an Open Innovation Partnership with LEO Pharma A/S, a global leader in medical dermatology, to discover and validate drugs to treat inflammatory and dermatology indications. The research collaboration leverages Repurpose.AI's ActivPred AI Drug Discovery Platform, an unbiased drug, target and disease agnostic digital chemistry engine, to discover drug candidates for inflammatory skin diseases. Previously, the company has utilized the platform to discover REP-001, REP-002 and REP-003 – three Phase II/III ready small molecule assets for the treatment of Gastric, Neurological and Weight disorders, respectively. LEO Pharma A/S will share inflammatory and dermatology targets they consider to be of interest and will evaluate compounds discovered by Repurpose.AI in disease assays for potential therapeutic use in inflammatory skin diseases.
  • 2020 - Addex Therapeutics, a clinical-stage pharmaceutical company pioneering allosteric modulation-based drug discovery and development, and the SIB Swiss Institute of Bioinformatics (SIB) received a CHF600K Innosuisse grant to apply computational approaches developed by SIB to identify new therapeutic indications for ADX10061, a potent and selective dopamine D1 receptor antagonist.
  • 2018 - The newly launched CMT Research Foundation, a 501(3) nonprofit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease, entered into a collaborative research partnership with AcuraStem to test thousands of compounds aimed at producing effective treatments for CMT2A
  • 2018 - Two experienced healthcare executives, David Martin Geliebter and Sven Martin Jacobson, founders of Remedy Pharmaceuticals, launched Martin Pharmaceuticals with the mission of repurposing already-approved drugs in order to offer therapeutic advances to patients afflicted with rare diseases or challenging medical conditions. Martin is the sixth healthcare company the pair has started since 2001. In May 2017, Remedy Pharmaceuticals sold its repurposed, orphan-designated CNS drug program, CIRARA to Biogen for a $120 million upfront payment plus future potential milestones and royalties. Martin also completed a seed round of financing from a group of outside investors to advance a clinical-stage, repurposed drug program.
  • 2016 - The University of Cambridge and King's College London jointly developed intellectual property regarding the underlying cellular mechanisms that cause age-related calcification of arteries and veins including a patent application and know-how relating to the use of PARP inhibitor drugs for the treatment of vascular disease. This IP has been licensed by Cambridge Enterprise, the commercialization arm of the University of Cambridge, to Cycle Pharmaceuticals.
  • 2015 - Cycle Pharmaceuticals entered into a partnership agreement with UCL Business PLC to develop existing drugs for new uses in treating liver diseases including a licence to IP developed at University College London.
  • 2015 - BioXcel Corporation, a provider of cloud-based pharma big data solutions for discovering novel products, entered into a partnership with Takeda for the repurposing of assets across the gamut of rare diseases. Takeda secured access to BioXcel's Big Data Innovation Lab – an integrated product discovery engine – and PharmGPS Orphan Disease Suite for drug discovery and repurposing.
  • 2013 - Cancer Research UK/Cancer Research Technology – the charity's development and commercialization arm –reached an agreement with AstraZeneca to take AZD2098, an experimental drug originally designed for asthma, into a clinical trial to treat kidney cancer.

To Conclude

As covered in our previous post on drug repurposing, although the development risks can be lower, the commercial risks are high and inevitably it is a balancing act between the two. Very few companies have successfully found this balance, but we nevertheless believe that the power of Big Data will deliver, albeit sparingly, some impactful repurposing opportunities. The unanswered question, though, is whether this will present a positive return on investment. AI-based platforms can be used to discover new molecules, and repurposing may turn out to be a secondary application, at least in terms of value creation. Perhaps the real value of “repurposing” will be the use of existing drugs as leads for the discovery of novel patentable APIs which will circumvent the commercialization issues that plague the repurposing segment.

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