RARE Daily

Three Rare Disease Focused Biotechs Raise a Combined $630 Million in Fresh Capital

February 14, 2024

Rare Daily Staff

Taking advantage of renewed investor interest in the therapeutics sector, three rare disease focused biotech companies raised new funds in private and public financings.

Gene editing biotech CRISPR Therapeutics entered into an investment agreement to sell approximately $280 million of its common shares to a select group of institutional investors in a registered direct offering, at a price per share of $71.50, representing a premium of greater than 10 percent to CRISPR Therapeutics’ 30-day volume-weighted average price.

EcoR1 Capital and SR One led the financing with participation from existing investors and a leading healthcare specialist investor.

“We are well positioned to execute on our on-going clinical trials in oncology, cardiovascular and diabetes, and further accelerate our auto-immune and in vivo gene writing programs, setting up a catalyst-rich 12-18 months for the company,” said Samarth Kulkarni, CEO and chairman of CRISPR Therapeutics. “This financing bolsters our already strong balance sheet, provides the opportunity for additional value creation, and gives us the flexibility to reach sustainability without requiring additional capital.”

EnGene Holdings, a clinical-stage genetic medicines company whose non-viral lead program EG-70 is in a pivotal study for BCG-unresponsive non-muscle invasive bladder cancer (NMIBC), is selling 20 million shares of its common stock at $10.00 per share, representing a 31 percent premium over the closing price on February 13, 2024. EnGene anticipates the gross proceeds from the private placement to be $200 million, before deducting any offering-related expenses.

The financing included participation from new and existing investors, including Adage Capital Partners, LP, Blue Owl Healthcare Opportunities, Boxer Capital, Commodore Capital, Cormorant Asset Management, Deep Track Capital, an affiliate of Deerfield Management Company, Foresite Capital, Janus Henderson Investors, Logos Capital, Lumira Ventures, Marshall Wace, Perceptive Advisors, Soleus Capital, Surveyor Capital (a Citadel company), Venrock Healthcare Capital Partners, and a large investment management firm.

EnGene intends to use the proceeds from this financing to fund the continued development of EG-70, the company’s genetic medicine therapeutic candidate being evaluated in an ongoing pivotal study for BCG-unresponsive NMIBC, evaluation of expanded EG-70 development opportunities, potential new R&D programs and for working capital and general corporate purposes. The proceeds from this financing, combined with current cash and cash equivalents are expected to be sufficient to fund the current operating plan into 2027.

Larimar Therapeutics, a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, priced an underwritten public offering of 17.2 million shares of its common stock at a price to the public of $8.74 per share, the closing price of its common stock on February 13, 2024, for gross proceeds of approximately $150.0 million, before deducting underwriting discounts and commissions and other offering expenses. In addition, Larimar has granted the underwriters a 30-day option to purchase up to an additional 2,574,370 shares of its common stock at the public offering price, less underwriting discounts and commissions.

Larimar intends to use the net proceeds from the proposed offering to support the development of nomlabofusp (CTI-1601) as a potential treatment for ‘Friedreich’s ataxia and other pipeline candidates, and for working capital and general corporate purposes, including research and development expenses.


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