Synageva BioPharma Corp. priced its underwritten public offering of common stock, seeking to raise $78.3 million by selling 3,108,057 shares at $25.18 per share.

The Lexington, Mass., company, which focuses on orphan drug technologies, could add $11.7 million to the haul, granting underwriters a 30-day option to purchase 466,209 additional shares to cover overallotments.

The company reported 17.6 million outstanding shares and a market cap of $508.12 million prior to the offering.

Morgan Stanley and J.P. Morgan acted as joint book-running managers, with Cowen and Co., Leerink Swann and Wedbush PacGrow Life Sciences acting as co-managers.

Investors liked the move, with shares (NASDAQ:GEVA) climbing $3.72 on Thursday to close at $28.90 – a 52-week high and a gain of nearly 15 percent.

CEO Sanj K. Patel said strong demand for the company's stock drove the timing of the offering.

The proceeds will allow Synageva to complete a busy to-do list in 2012, including the transition of its fully enrolled Phase I/II study of lead compound SBC-102, a recombinant human lysosomal acid lipase (LAL), into an extension study. The Phase I/II study was designed to evaluate the safety and tolerability of SBC-102 administered weekly in patients with liver dysfunction due to late onset LAL deficiency.

The next step for the compound is a double-blind, placebo-controlled trial, which the company also plans to initiate at the end of this year or in early 2013.

The cash, which caps a year of successful fundraising, also pads Synageva's cushion to build its global development and commercialization infrastructure after completing a reverse merger with Trimeris Inc. in November that gave the company access to the public markets. (See BioWorld Today, June 14, 2011.)

That transaction also gave Synageva a modest source of additional cash by way of a 16 percent royalty stream from Roche AG, of Basel, Switzerland, for worldwide net sales of HIV fusion inhibitor Fuzeon (enfuvirtide). In 2010, the drug's revenue totaled about $88.4 million.

On top of that, Synageva closed a previous $25 million equity round earlier in 2011. (See BioWorld Today, March 22, 2011.)

All told, during the two years prior to the Trimeris acquisition, Synageva raised about $70 million. Patel declined to define the company's runway following the current offering and said he would offer guidance later in the year – perhaps as early as the J.P. Morgan Healthcare Conference next week.

Synageva's work in rare – even ultra-orphan – lysosomal storage disorders remains the company's primary focus, Patel confirmed. Although the offering will allow the company to invest in its early stage candidates, "clearly, our main focus is on our lead program, SBC-102," he told BioWorld Today.

LAL deficiency is a lysosomal storage disorder characterized by the buildup of fatty material in the liver, spleen and blood vessels. The disease is classified into two types: early onset – a rapidly fatal type also known as Wolman disease – and late-onset LAL deficiency.

When both parents are carriers, there is a one in four chance that their child will be born without the ability to produce the LAL enzyme, which breaks down fatty material. Lack of the LAL enzyme results in a build-up of these materials in the liver, the gut and other important organs, including blood vessel walls.

Late-onset LAL deficiency, sometimes called cholesteryl ester storage disease, affects 25 individuals per million births and may lead to liver fibrosis, cirrhosis, liver failure and death. Late-onset LAL deficiency also may increase an individual's stroke risk.

Synageva's SBC-102 is designed to treat both forms of the disease using glycan structures that are specifically recognized and internalized by specific receptors into key target cells. The company's technology is a "robust" system that allows Synageva to produce these proteins sustainably and consistently, Patel said.

SBC-102 has been granted orphan designation in the U.S. and fast-track designation in the European Union.

The company has a second compound, SBC-103, in preclinical development for lysosomal storage disorders. Three undisclosed preclinical candidates also target severe genetic or metabolic disorders.

"They're all focused on devastating medical conditions with unmet medical need," Patel said. "We want to stay very true to our mission, which is to make a transformational impact on the lives of patients."

In other financing news:

• Clearside Biomedical Inc., an ophthalmic startup based in Atlanta, launched with a $4 million Series A from Hatteras Venture Partners of Durham, N.C. Initially, the company is developing an ocular microinjection platform and testing its lead product for macular edema and retinal vein occlusion. Clearside's platform has been designed to deliver drugs nonsurgically to individual compartments of the eye. Its initial product candidate, CLS1001, is under development for retinal applications in the suprachoroidal space.

• Cowen Healthcare Royalty Partners, of Stamford, Conn., raised approximately $1 billion and closed Cowen Healthcare Royalty Partners II, LP (CHRP II), exceeding the fund's initial target by more than 75 percent. The company plans to maintain its broad strategy of investing in traditional passive royalties, synthetic royalties and structured financings. Similar to its first fund, CHRP II will target investments between $20 million and $100 million in the U.S., Europe and Asia, primarily for commercial-stage biopharmaceutical and medical device companies seeking growth capital and for universities and inventors seeking to monetize technology. The fund's limited partners include existing Cowen Royalty limited partners as well as public and corporate pension funds, financial institutions, insurance companies, funds-of-funds and university endowments. Credit Suisse Securities (USA) LLC acted as exclusive financial advisor/placement agent.

• Midatech Ltd., of Oxford, UK, raised £6.3 million (US$9.76 million) through a private investment round to strengthen and diversify its oncology product development portfolio; support clinical development of chemotherapeutic gold nanoparticles targeting ovarian, lung and breast carcinoma; and support development of therapeutic cancer vaccines through SynTara, its joint venture with Immunotope Inc., of Doylestown, Pa. Separately, Midatech and MonoSol Rx LLC, of Warren, N.J., launched a joint venture, MidaSol Therapeutics LP, to focus on the commercialization, via partnering or licensing, of products using the combined intellectual property of the companies' respective technologies in diabetes. The companies are conducting a Phase I study of their lead compound, which uses insulin-passivated gold glyconanoparticles formulated for buccal delivery. Results of the Phase I study are expected in the first half of 2012. (See BioWorld Today, July 13, 2010.)

• Syndexa Pharmaceuticals Corp., of Watertown, Mass., secured additional Series B1 financing from MP Healthcare Venture Management (MPH), a jointly owned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC) and Mitsubishi Chemical Holdings Corporation (MCHC). Total capital raised in the Series B1 round, originally led by existing investor Yalcin Ayasli in April 2011, now stands at $8.5 million. The proceeds will be used to advance the preclinical development of the company's endoplasmic reticulum (ER) modulating compounds toward development candidate selection and help to validate the therapeutic potential of compounds in various indications of ER dysfunction. The company closed a $15 million Series B in 2008. (See BioWorld Today, June 2, 2008.)

• 3-V Biosciences Inc., of Menlo Park, Calif., closed a preferred stock financing providing a minimum of $20 million with existing investors Kleiner Perkins Caufield & Byers and New Enterprise Associates. The company plans to use the proceeds to advance its hepatitis C virus program, earlier-stage pipeline opportunities and its screening platform.