One of the emphases in today’s webinar (see Slide 10) was on how upcoming program milestones in each of PYC’s programs will not only add value to each of the assets, but also validate the potential of PYC’s delivery platform in a broader range of applications.
By Q4 this year, there should be initial safety and efficacy data for PYC’s second program, PYC-001 in ADOA. Positive results in the eye indications of both RP11 and ADOA would provide a strong indication that PYC’s PPMO approach can potentially be used to therapeutic effect across a broader range of eye diseases.
Rohan mentioned that work is currently being conducted at Melbourne University on models of both glaucoma and geographic atrophy. Like ADOA, these are eye diseases of bioenergetic deficiency. PYC-001 increases cellular bioenergetics. If it is successful in treating ADOA, PYC-001 may also hold therapeutic potential in glaucoma and geographic atrophy, both diseases with patient populations in the millions. Further, as Rohan has previously stated, any further indications for PYC-001 would be able to move straight to Phase 2.
Success in ADPKD could also have broader implications. Rohan described PYC-003 as having a “unique profile” with incredible bio distribution throughout the kidney. PYC’s approach here could potentially benefit other kidney conditions.
While the current PMS program uses a different backbone chemistry to the other programs which doesn’t involve a cell-penetrating peptide (the one used initially was found to be too powerful for administration via lumbar puncture), a new peptide is being worked on that holds potential across multiple CNS conditions. (At least, I think that’s what Rohan said, but he talks so much faster than I can write, that I could have that wrong! If anyone heard differently, please correct me.)
Once the company is seen to have a validated platform technology, the attraction and value of PYC should grow well beyond the current programs in development.
At the beginning of the webinar today, Rohan referred to two recently announced collaborations in which large pharma will use the RNA platforms of much smaller biotechs.
The first was a Collaboration and License Option Agreement between AbbVie and ADARx Pharmaceuticals which was announced two weeks ago. It concerns development of siRNA therapies across multiple therapeutic areas, including neuroscience, immunology and oncology. The collaboration will combine ADARx's RNA discovery expertise and proprietary siRNA technology with AbbVie’s antibody drug conjugates (ADCs) and tissue delivery approaches. Under the terms of the agreement, ADARx will receive a $335 million upfront payment and will be eligible to receive several billion dollars in additional contingent payments including option-related fees and milestone payments, as well as tiered royalties. ADARx is an unlisted, late-clinical stage biotech.
https://news.abbvie.com/2025-05-14-...A-Therapies-Across-Multiple-Therapeutic-AreasThe second research collaboration, announced this week, will see Biogen and City Therapeutics work together to develop select novel RNAi therapies. The initial focus is on a single target that mediates key CNS diseases. City Therapeutics will receive a US$16m upfront payment and an investment of $30m. It will also be eligible to receive up to $1bn in potential milestone payments plus tiered royalties in the high single-digit to low double-digit range based on net sales. Biogen has also taken the option to select one additional target in the collaboration, subject to an additional payment. City Therapeutics is an unlisted biotech unveiled just last year. Its co-founder and Executive Chair founded the US$38bn RNAi pioneer company, Alnylam Pharmaceuticals.
https://investors.biogen.com/news-r...city-therapeutics-announce-strategic-research
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