Two Rare Disease Biotechs Rake in $170 Million in Public Equity Offerings

Rare Daily Staff

Solid Biosciences and X4 Pharmaceuticals together raised $170 million through public equity offerings to advance their rare disease therapeutics pipelines.

After reporting encouraging data earlier in the week from their ongoing clinical trial of its lead gene therapy candidate SGT-001 for the treatment of Duchenne muscular dystrophy, a rare muscle wasting disease, Solid Biosciences tapped the public markets to raise capital. The biotech priced a public offering of 21.7 million shares at $5.75 per share to raise $125 million before deducting underwriting discounts, commissions, and offering expenses. It also granted underwriters a 30-day option to purchase up to an addition 3.3 million shares at the public offering price.

Solid expects to use the proceeds from this offering to fund research and development expenses, including the advancement of SGT-001, and for working capital and other general corporate purposes.

X4 Pharmaceuticals opted for a different route. The company, which is focused on

novel therapies targeting rare diseases resulting from dysfunction of the CXCR4 pathway, agreed to sell an aggregate of 6.3 million shares of its common stock, including pre-funded warrants to purchase common stock, to certain institutional accredited investors in a private investment in public equity, or PIPE, financing to raise approximately $55 million, before deducting fees and expenses.

New investors including Abingworth, Altium Capital, Driehaus Capital Management, Lincoln Park Capital, Monashee Investment Management and Sio Capital participated in the financing, as well as existing investors Bain Capital Life Sciences, Ikarian Capital and OrbiMed.

X4 expects to use the proceeds to advance the clinical development of its pipeline, business development activities, working capital and general corporate purposes.

The company’s lead candidate, mavorixafor, is a first-in-class, small molecule antagonist of chemokine receptor CXCR4 being developed as a once-daily oral therapy. The efficacy and safety of mavorixafor, dosed once daily, is currently being evaluated in a global phase 3 clinical trial in patients with WHIM syndrome, and in two phase 1b clinical trials – in combination with ibrutinib in patients with Waldenstrom macroglobulinemia, and as monotherapy in patients with severe congenital neutropenia.