Four Companies Raise $380.5 Million in New Capital to Advance Their Rare Disease Programs

Four public companies tapped the public and private markets for $380.5 million of new capital to advance their rare disease pipelines.

Fresh on the heels of a major deal with Bayer for a pair of autologous CAR T molecules that netted the company $60 million upfront, Atara Biotherapeutics went to the public markets to raise $175 million in a public offering of 5.1 million shares of its common stock at $24.50 a share.

The offering includes pre-funded warrants to certain investors to purchase 2 million shares of its common stock at $24.4999 per pre-funded warrant share, which represents the per share public offering price for the common stock, minus the $0.0001 per share exercise price of each such pre-funded warrant share. Atara also granted the underwriters a 30-day option to purchase up to an additional 1 million shares of its common stock at the public offering price, less the underwriting discounts and commissions.

Atara Biotherapeutics’s off-the shelf allogeneic cell therapies leverage its novel allogeneic EBV T-cell platform to develop transformative therapies for patients with serious diseases including solid tumors, rare hematologic cancers, and autoimmune diseases.

Marinus Pharmaceuticals, which develops innovative therapeutics to treat rare seizure disorders, raised $70 million through the sale of 5 million shares of its common stock. at $14 a share. In addition, Marinus granted the underwriters a 30-day option to purchase up to an additional 750,000 shares of common stock.

Marinus intends to use the net proceeds of the offering to fund the development of its product candidates and for general corporate purposes, which may include working capital, capital expenditures, research and development expenditures, clinical trial expenditures, acquisitions of new technologies, products or businesses, and investments.

Marinus lead candidate ganaxolone is a positive allosteric modulator of GABAA receptors that acts on a well-characterized target in the brain known to have anti-seizure, anti-depressant and anti-anxiety effects. Ganaxolone is being developed in IV and oral dose formulations intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Marinus recently completed the first ever phase 3 pivotal trial in children with CDKL5 deficiency disorder and is conducting a phase 2 trial in tuberous sclerosis complex, as well as a phase 2 biomarker-driven proof-of-concept trial in PCDH19-related epilepsy. The company is also initiating a phase 3 trial in status epilepticus.

Syros Pharmaceuticals, which develops small molecule medicines that control the expression of genes, raised $90.5 million in a private placement with a group of institutional accredited investors led by Bain Capital Life Sciences, with participation from new and existing investors, including Ally Bridge Group, Omega Funds, OrbiMed Advisors, EcoR1 Capital, and Samsara BioCapital.

Syros will use net proceeds from the financing to advance its clinical development pipeline, business development activities, working capital and other general corporate purposes.

Syros focuses on therapies for diseases that have eluded other genomics-based approaches. Syros’ clinical-stage pipeline, includes SY-1425, a first-in-class oral selective RARα agonist in RARA-positive patients with higher-risk myelodysplastic syndrome and acute myeloid leukemia, SY-2101, a novel oral form of arsenic trioxide in patients with acute promyelocytic leukemia, and SY-5609, a highly selective and potent oral CDK7 inhibitor in patients with select solid tumors.

Finally, rare disease focused biotech Amryt entered into securities purchase agreements with several institutional accredited investors for the private placement of 3.2 million American Depositary Shares (ADS), each representing five ordinary shares, at a purchase price of $12.50 per ADS, yielding expected gross proceeds of $40 million. The private placement includes a mix of new and existing investors including Stonepine Capital, Aquilo Capital Management, Amati Global Investors, Athyrium Capital Management, and Highbridge Capital Management, among others.

Proceeds from the private placement will be used for working capital and general corporate purposes, as well as to potentially acquire, in-license or invest in rare disease technologies, products, businesses or assets.

Amryt’s portfolio includes commercial and development assets. Lead development candidate, Filsuvz is a potential treatment for the cutaneous manifestations of EB, or epidermolysis bullosa, a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment.  In September and October 2020, Amryt reported positive results from its pivotal global phase 3 trial of Filsuvez in EB.  Filsuvez has been granted Rare Pediatric Disease and Fast Track designations from the U.S. Food and Drug Administration.