In its fourth substantial public offering in two years, Synageva Biopharma Corp. padded its balance sheet with a $155.7 million to accelerate its aspirations of becoming a global rare disease company replete with burgeoning pipeline programs, manufacturing and commercial infrastructure.

That’s a plan investors appear to be endorsing wholeheartedly, no doubt fueled by growing enthusiasm for the rare disease space, as evidenced by rampant buyout speculation involving Alexion Pharmaceuticals Inc. and Biomarin Pharmaceutical Inc. (See BioWorld Today, Sept. 20, 2013.)

Lexington, Mass.-based Synageva was able to price its offering of 2.57 million shares at nary a discount to Tuesday’s $56.63 closing price – Synageva director Felix Baker indicated an interest to purchase about 34 percent of the offering – and the stock (NASDAQ:GEVA) gained $5.05 Wednesday to close at $61 .68. It’s up roughly 30 percent on the year.

As of June 30, the firm had about $284.8 million in its coffers and no outstanding debt. The latest public offering could yield total gross proceeds of $179.1 million if underwriters exercise their full overallotment option.

Since the start of 2012, Synageva has raised about $500 million in public offerings, most recently raising $118.8 million in an upsized offering in January. (See BioWorld Today, Jan. 6, 2012, July 11, 2012, and Jan. 7, 2013.)

Proceeds will be used to support lead program, sebelipase alfa, which is in Phase III testing, while Synageva also plans to accelerate its early stage pipeline and build out manufacturing and global commercial capabilities.

“We have multiple value-driving events both this year and beyond,” CEO Sanj K. Patel told attendees at the Morgan Stanley conference earlier this month, adding that goals include “continuing to build this company for the long term.”

Synageva’s lead programs consist of enzyme replacement therapies (ERTs) for what Patel called “classic lysosomal storage disorders.” The firm anticipates completing enrollment in the first half of next year for its Phase III study, dubbed ARISE (Acid Lipase Replacement Investigating Safety and Efficacy), to test sebelipase alfa (SBC-102) in late-onset lysosomal acid lipase deficiency (LAL), also known as cholesteryl ester storage disease. A disease estimated to affect about 25 people per million births, late-onset LAL deficiency can lead to liver fibrosis, cirrhosis, live failure and death.

Sebelipase alfa is designed to treat LAL deficiency using glycan structures specifically recognized and internalized by specific receptors into target cells. It’s partnered with Mitsubishi Tanabe Pharma Corp., of Osaka, Japan, and has orphan status in both the U.S. and Europe.

Synageva has continued to report promising long-term data from the ongoing Phase I/II study, with one-year findings from seven patients reported earlier this month at the International Congress of Inborn Errors of Metabolism conference in Barcelona, Spain, showing reductions in liver damage and improvement in dyslipidemia associated with LAL deficiency. Patients also showed sustained reductions in both ALT and AST, and treatment was generally well tolerated.

The ARISE study is enrolling a total of 50 patients (adults as well as children older than 4) and will measure the proportion of subjects who achieve ALT normalization relative to placebo as the primary endpoint. Secondary endpoints include relative change in LDL, non-HDL and HDL cholesterol; relative change in triglycerides; and relative change in liver fat content and liver volume.

Synageva also is investigating sebelipase alfa in early onset LAL deficiency, also known as Wolman disease, for which it received breakthrough therapy status in May.

During the first half of next year, the company said it will move into the clinic with its second ERT, SBC-103, a recombinant form of the human NAGLU enzyme, aimed at treating mucopolysaccharidosis Type IIIB, or Sanfilippo B. Characterized by a marked decrease in alpha-N-acetyl-glucosaminidase enzyme activity, the disease leads to an accumulation of heparin sulfate disaccharides in the brain and other organs, causing severe cognitive decline, behavioral problems, speech loss, increasing loss of mobility and death.

Beyond those candidates, Synageva said in its prospectus that its platform technology has identified several new rare disease target programs, aiming to initiate between five and 10 additional programs.

The company also has expanded its manufacturing operations. In addition to 64,000 square feet of facilities in Georgia, it recently established an additional facility in Massachusetts of about 39,000 square feet to further supply protein therapeutics for sebelipase alfa, SBC-103 and additional pipeline products.

Goldman, Sachs & Co. is acting as the sole book-running manager for the public offering, with Morgan Stanley & Co., Cowen and Co. and Canaccord Genuity Inc. serving as co-managers.

Following the offering, Synageva will have about 30.3 million shares outstanding – 30.7 million if overallotment options are fully exercised.

In other follow-on offering news:

• Idera Pharmaceuticals Inc., of Cambridge, Mass., priced a public offering of about 13.7 million shares of common stock for $1.55 per share for gross proceeds of about $27.7 million. The company also issued pre-funded warrants to purchase up to an aggregate of 4.2 million shares of common stock at the per-share public offering price less the 1 cent-per-share exercise price for each such pre-funded warrant. Idera anticipates using the net proceeds to fund its planned Phase I/II trials designed to evaluate the use of IMO-8400, a Toll-like receptor (TLR) 7, 8 and 9 antagonist, in certain genetically defined forms of B-cell lymphomas. Proceeds also will support a Phase I trial of an additional TLR antagonist, IMO-9200, and for working capital and general corporate purposes. Piper Jaffray & Co. is acting as sole manager for the offering, set to close Sept. 30. Shares of Idera (NASDAQ:IDRA) closed Wednesday at $1.79, up 15 cents.