Glafabra Q3 Update - Pharma Partnering
Macro Headwinds and Funding Strategy
Despite significant macroeconomic headwinds and two large pharma companies publicly abandoning CGT, Glafabra remains optimistic, but acknowledges that its funding timeline has been delayed. Adjusted, the company is now executing a three-phase funding strategy:
Phase 1 (Current - Year End 2025): Actively raising $500,000 via SAFE agreements ($5M cap, 20% discount) through Mercury bank and a Wefunder campaign..Status:17% subscribed.
Phase 2 (Jan 2026): Targeting an $8M priced Seed round at an estimated $50M pre-money valuation.
Phase 3 (Jan 2027): Targeting a $45M priced Series A round at an estimated $250M pre-money valuation.
Clinical and Regulatory Focus
Glafabra is highly focused on entering the clinic in the USA by repeating the Canadian FACTS trial at a clinical site in the US. The immediate goals are to secure Orphan Drug Designation (ODD) and to request an INTERACT meeting with the FDA with the help of Uncommon Cures. The primary plan is a switching trial with 1-year eGFR and enzyme activity as endpoints.
The company is exploring a potential protocol amendment (or Expanded Access Protocol) to use reduced myeloablative conditioning (melphalan) to lower patient risk (e.g., sterilization) and to allow for repeat dosing of the therapy. Repeat dosing would enable tuning the therapy and align the treatment duration (a few years) with the average patient reimbursement window.
Corporate Development
New Partnership: A relationship with a large pharma partner is a high priority. We currently have a standing offer from a large pharma to potentially fund trial completion in exchange for their market exclusivity to a geographical region. As a result, Glafabra is now pursuing a dual clinical strategy to start trials in both the US and Japan concurrently.
New Leadership: Joining the management team is Kevin Marhenke, former Director of Global External Medical Engagement at Amgen. Kevin brings a wealth of experience in bringing therapies to market and will be a key resource for shaping our go to market approach.
