INVESTORS

Diversified Development Platform With High Value Programs Across Stages.

  • Targeting Series A of $35M to enable multiple clinical milestones

  • Lead program targets significant commercial opportunity for untreated rare disease with rapid path toapproval

  • Single registration study agreed with FDA/EMA – Approval in 2023

  • Potential expansion into additional mechanistically aligned rareindications

  • Potential approval in US and EU in ~ 3 years for untreated patient population with severe disease(Orphan & Fast-Track designations granted)

  • Initial disease market anticipated to be $200M-$300M; no known competition

  • Related indications expand peak sales opportunity substantially

  • Additional 1-2 IND filings within 24 months from current pipeline

  • Clinical or near-clinical acquisitions under evaluation have game-changing potential for patients; attainable under attractive terms

  • Management team, Board and Advisors with broad industry expertise in product development, technical operations, product supply, business and corporate development, and capital sourcing

Press Releases

15

Mar ‘21

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15

Mar ‘21

News 2

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15

Mar ‘21

News 1

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Events

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Mar 24 - Mar 28, 2021

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Mar 26 - Mar 30, 2021

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Apr 3 - Apr 9, 2021

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May 12 - May 18, 2021

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SEC Filings

Sec 112G

Mar 26, 2021

Sec 112G

Mar 28, 2021

Sec 112G

Apr 8, 2021

Sec 112G

Apr 22, 2021

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