XFOR - X4 Pharmaceuticals: FDA Approval For WHIM Warrants Further Upside

2024-04-29 17:02:33 ET

Summary

• X4 Pharmaceuticals, Inc. received FDA approval for mavorixafor in WHIM syndrome, along with a priority review voucher (typically worth $100M).
• X4 Pharmaceuticals has announced very premium pricing considering the benefits of mavorixafor and ultra-orphan target indication. Based on this pricing, considerable (relevant to current EV) peak sales are expected.
• X4 Pharmaceuticals is well-funded to support mavorixafor commercialization, and I expect a further run-up towards upcoming data in chronic neutropenia indication.
• The major risk for X4 Pharmaceuticals now is setbacks with insurance coverage policies.

Thesis update

X4 Pharmaceuticals, Inc. ( XFOR ) has been performing well since my last coverage , but at the time of writing is still trading below the price of my original "Buy" recommendation. For a background on XFOR I refer readers to my prior coverage. Very briefly, XFOR's lead asset is mavorixafor, an oral CXR4 inhibitor being developed for the treatment of neutropenia. XFOR just announced FDA approval of mavorixafor for WHIM syndrome (a rare genetic syndrome causing neutropenia and recurrent infections among other manifestations), as well as receipt of a priority review voucher ((PRV))....

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