Taking advantage of a flurry of promising data for its lead immune disease candidate, RPC1063, including a late-October stock surge on positive ulcerative colitis data, Receptos Inc. priced a whopping $360 million public offering.

It's by far the biggest haul from a follow-on offering of 2014, according to BioWorld Snapshots – the next largest is Avanir Pharmaceuticals Inc.'s $230 million public offering in September – and Receptos could add another $54 million if underwriters exercise their overallotment option of 540,000 shares in full, bringing total gross proceeds to $414 million. That more than doubles the $302.9 million in cash and equivalents Receptos reported at the end of the third quarter and Wedbush Securities analysts are projecting a cash runway "well into 2018."

The company, which went public in May 2013 with shares opening at $15, priced its latest offering of 3.6 million shares at $100 apiece, a slight discount to Tuesday's close. The stock (NASDAQ:RCPT) gained another $4.97 Wednesday to close at $107.95. (See BioWorld Today, May 10, 2013.)

Overall, it's been a very good fall for the San Diego-based firm. Late last month, investors pushed shares up 28 percent on the back of top-line data showing that oral sphingosine 1-phosphate 1 receptor (S1P1R) small-molecule immunomodulator RPC1063 met its primary and secondary endpoints in ulcerative colitis in the phase II TOUCHSTONE trial. A month earlier, shares spiked after an abstract of the complete dataset from the phase II portion of the phase II/III RADIANCE trial in relapsing multiple sclerosis (MS) patients prompted analyst murmurings of a potential best-in-class S1P1R scenario for RPC1063. (See BioWorld Today, Sept. 15, 2014, and Oct. 29, 2014.)

Next up, Receptos watchers will be looking for detailed induction and maintenance data from the TOUCHSTONE study, expected in the first half and second half of 2015, respectively. Meanwhile, the RADIANCE study is continuing, with the phase III portion launched a year ago and expected to enroll 1,200 relapsing MS patients to test RPC1063 vs. Avonex (interferon beta-1a, Biogen Idec Inc.) in reducing annualized relapse rate (ARR) at 24 months. And a second phase III trial in relapsing MS is set to start in the first half of 2015, under a special protocol assessment agreement with the FDA, also testing RPC1063 vs. Avonex in reducing ARR at the end of 12 months of treatment.

Receptos holds full rights to RPC1063 and, while the firm noted in its SEC prospectus that it could seek a partner to preserve capital, proceeds from the latest offering could get the compound all the way to FDA review in relapsing MS, Wedbush analysts noted in a research report.

The company has further plans for RPC1063. A phase II study is set to start next year in Crohn's disease and other autoimmune disease applications are being explored.

Beyond RPC1063, Receptos has RPC4046, a monoclonal antibody directed against interleukin-13, currently in phase II testing in eosinophilic esophagitis, an orphan gastrointestinal disease, with top-line data expected in the first half of 2016. Its preclinical pipeline includes efforts to develop oral, small molecule, positive allosteric modulators, or PAMs, of the glucagon-like peptide-1 receptor for treating type 2 diabetes. Investigational new drug application-enabling studies for that program are slated to start in 2015.

Credit Suisse Securities (USA) LLC and Leerink Partners LLC are acting as joint lead book-running managers, while Evercore Group LLC. and BMO Capital Markets Corp. are acting as book-running managers and Wedbush Pacgrow Life Sciences and Nomura Securities International Inc. are acting as co-managers for the offering, set to close on or about Nov. 24.

AMICUS PAD COFFERS WITH $90M OFFERING

Like Receptos, Amicus Therapeutics Inc. priced a follow-on offering shortly after positive clinical data sent it stock rising.

The Cranbury, N.J.-based company, which saw shares jump 17.8 percent Monday after 18-month data disclosed at the American Society of Nephrology meeting in Philadelphia showed that oral, small-molecule chaperone migalastat met both co-primary endpoints in comparability to enzyme replacement therapy (ERT) in Fabry disease patients, priced 13.85 million shares at $6.50 per share.

Gross proceeds of $90 million – $103.5 million if underwriters exercise their full overallotment option – will support the building of commercialization infrastructure for migalastat monotherapy in Fabry disease, as well as continued investment in other product candidates.

Amicus reported data from the second phase III study in Fabry disease in August and outlined plans for regulatory submissions. In its prospectus, the company said it plans to conduct a pre-submission meeting with the EMA this quarter. In the U.S., it hopes to meet with the FDA early next year to discuss the regulatory approval path. (See BioWorld Today, Aug. 21, 2014.)

Amicus owns worldwide rights to migalastat, and the firm said it expects to build its own commercial capabilities to sell the drug in both the U.S. and Europe.

Down the road, the company aims to see migalastat approved in combination with an ERT in Fabry disease. The firm also has in development Pompe disease drug AT2220, which so far has demonstrated promising results in a phase II safety and pharmacokinetics study in combination with ERTs.

J.P. Morgan Securities LLC is acting as sole book-running manager for the offering, set to close on or about Nov. 24, with Cowen and Co. LLC and Leerink Partners acting as lead managers and Janney Montgomery Scott acting as co-manager.

Shares of Amicus (NASDAQ:FOLD) closed Wednesday at $6.53, down 22 cents.

In other financings news:

Bellicum Pharmaceuticals Inc., of Houston, filed an S-1 for an initial public offering, though the number of shares or share price have not yet been determined. The immunotherapy company will use proceeds to fund ongoing and upcoming phase I/II trials of lead drug BPX-501, an adjunct T-cell therapy designed to administer after allogeneic hematopoietic stem cell transplantation, as well as the planned phase I/II trials of BPX-201, its dendritic cell cancer vaccine, in combination with checkpoint inhibitors. Funds also will support clinical and preclinical studies for other compounds, including CAR T-cell therapies and T-cell receptor therapies. Bellicum previously raised $55 million in an oversubscribed series C round in August and ended the third quarter with about $61.9 million on its balance sheet. The firm, while filed as an emerging growth company, is seeking a Nasdaq listing under the ticker BLCM. Jefferies LLC, Citigroup Global Markets Inc. and Piper Jaffray & Co. are acting as joint book running managers. (See BioWorld Today, Aug. 28, 2014.)

Benevir Biopharm Inc., of Rockville, Md., said it closed a series A round with Pansend LLC, an indirect wholly owned subsidiary of HC2 Holdings Inc. Founded in 2011, Benevir is developing core technology licensed by New York University to develop a pipeline of cancer immunotherapy candidates that affect antigen presentation in both tumor cells and antigen-presenting cells. Proceeds of the financing will be used to conduct initial proof-of-concept clinical studies of its lead product, further develop the pipeline and establish partnerships to test combination therapies in metastatic solid tumors.

N30 Pharmaceuticals Inc., of Boulder, Colo., said it raised $30 million in a mezzanine round of financing. New investors included Wellington Management, RA Capital Management, LLC, Jennison Associates LLC, Rock Springs Capital Management LP and Sabby Management LLC, who joined existing investor Deerfield Management Co. LP. Proceeds will primarily be used to advance the company's clinical program for N91115, an inhibitor of S-nitrosoglutathione reductase. The company recently completed a phase I dose-escalation trial of orally administered N91115 in healthy volunteers. The compound was found to be well tolerated over the 14 days of dosing, and trials in cystic fibrosis (CF) patients with the F508del-CFTR mutation are now under way. In preclinical studies, N91115 has been shown to increase the function of F508del-CFTR, the mutant protein that is estimated to be present in almost 90 percent of CF patients.

Prometic Life Sciences Inc., of Laval, Quebec, said it entered an agreement with a syndicate of underwriters led by Canaccord Genuity Corp., under which the underwriters agreed to buy, on a bought-deal basis, 13.2 million common shares at a price of $1.90 per share for gross proceeds of $25.1 million. Underwriters also have the option to purchase an additional 2 million shares, which, if exercised in full, will bring the total gross proceeds to $28.8 million. Funds will be used to advance fibrosis drug PBI-4050 and the firm's new plasma-derived orphan drugs, as well as for manufacturing scale up of orphan candidates and follow-on compounds to PBI-4050. Shares of Prometic (OTCQX:PFSCF) closed Wednesday at $1.80, down 5 cents.