Barely a month after Celladon Corp. priced its modest initial public offering (IPO), the San Diego-based firm saw its shares jump 25.5 percent Monday on news of an option deal with French pharma firm Servier based on SERCA2b modulators for metabolic disease.

Specific terms weren’t disclosed, but Servier picked up an exclusive option to license ex-U.S. rights to the early stage small-molecule program for use in diabetes and other metabolic disorders in exchange for up-front, research and milestone payments. Celladon also would receive royalties on product sales and would retain U.S. rights to any compounds developed under the research and license agreement.

“It essentially allows us to partner with a premier pharmaceutical company” that has 30 years of experience in the diabetes field, Krisztina M. Zsebo, Celladon’s president and CEO, said of the deal. If Servier exercises its option for further development – a decision is expected following the completion of in vitro and in vivo testing anticipated to wrap up in the first quarter of 2015 – the Suresnes, France-based pharma would shoulder all costs associated for development of the candidates outside the U.S.

Celladon, meanwhile, which is retaining U.S. rights to the program, would be able to use data generated by Servier. “We would leverage their R&D operation,” Zsebo told BioWorld Today.

The Servier pact marks Celladon’s first big pharma partnership, though the firm previously attracted corporate venture investments from the likes of Pfizer Venture Investments, Novartis Venture Funds and Johnson & Johnson Development Corp., driven largely by interest in its lead program, Mydicar, a gene therapy product designed to target SERCA2a – specifically, it aims to restore levels of SERCA2a, which are abnormally low in patients with advanced heart failure, to promote normal contractility, relaxation and calcium cycling.

Mydicar is being developed initially for patients with end-stage heart failure, who have limited treatment available. In fact, heart transplants and left ventricular assist device (LVAD) transplantation are the only available options, but the “astronomical” costs – roughly $200,000 per procedure – and the limited number of procedures that can be done each year mean that many patients are left without alternatives, Zsebo explained.

“There’s a huge treatment gap,” she said. About 350,000 patients in the U.S. alone would be eligible for Mydicar treatment. Right now, those patients are costing the health care system significantly, with costs running about $21 billion in 2012, she added.

Celladon presented data from the phase IIa CUPID 1 study in November at the American Heart Association Scientific Sessions in Dallas, showing that Mydicar reduced heart failure risk by 88 percent (p = 0.003) vs. placebo and showed a “nice trend in overall survival,” Zsebo noted.

Last year, the firm moved Mydicar into a phase IIb study in patients with ischemic or dilated cardiomyopathy and NYHA class III/IV symptoms of heart failure. That trial, dubbed CUPID (Calcium Up-Regulation by Percutaneous Administration of Gene Therapy in Cardiac Disease) 2, will test the gene therapy’s ability to reduce the frequency and/or delay heart failure-related hospitalizations vs. placebo. Full results expected in 2015. (See BioWorld Today, Aug. 30, 2013.)

Zsebo said it is considered a pivotal trial in Europe; however, in the U.S., the question of whether CUPID 2 can serve as a pivotal, registrational study is “still an open issue,” she said, which will be reviewed as data from the study become available.

Celladon retains worldwide rights to Mydicar and plans to commercialize the product on its own in the U.S., where a “very directed sales force” would be capable of reaching specialty cardiology practices in the country.

Proceeds from the company’s recent IPO are “enormously helping” the firm to conduct parallel development work for registration of Mydicar in its lead indication, as well as to expand beyond systolic heart failure patients and into patients with advanced heart failure with LVAD and diastolic heart failure.

Priced last month, the IPO was somewhat overshadowed by Dicerna Pharmaceuticals Inc., which went public the same day and saw its shares skyrocket 100 percent at opening. Celladon, meanwhile, cut the proposed offering price and increased the number of shares offered to 5.5 million for gross proceeds of $44 million, about half of what it had hoped to raise when filing the first S-1 paperwork last year. (See BioWorld Today, Oct. 14, 2013, and Jan. 31, 2014.)

Still, those funds, combined with the $23.2 million in the bank as of Sept. 30, should carry the firm until the end of 2015, well after Mydicar phase IIb data will be in hand.

If all goes well, the Servier deal could be advancing by that time, too. Like Mydicar, that program is targeting SERCA, a family of enzymes linked to calcium dysregulation and are believed to be implicated in multiple diseases, from heart failure to metabolic disorders to neurodegenerative indications. SERCA, or Sarco/endoplasmic reticulum Ca2+-ATPase, enzymes comprise a family of enzymes that help regulate intracellular calcium in cells.

Unlike Mydicar, however, the Servier collaboration will focus on a small-molecule rather than a gene therapy approach. And, instead of SERCA2a, the compounds will be designed to target the SERCA2b isoform, the idea to restore SERCA2b levels that fall due to endoplasmic reticulum (ER) stress. SERCA2b is responsible for directing calcium into the ER, so it’s possible an allosteric modulator could be used to replenish that calcium in the ER.

Zsebo called the discoveries in ER stress a “really exploding field,” one that might prove a promising treatment option in diabetes. “The enigma of diabetes is that it affects so many different tissues,” including eyes, neurons, liver, pancreas, etc. “But one of the uniform features is the ER stress.”

Beyond diabetes, modulating SERCA2b could work in other metabolic indications. The Servier deal isn’t limited to a specific number of compounds, so there’s a possibility the collaboration could yield more than one candidate.

Shares of Celladon (NASDAQ:CLDN), which had hovered just below the $8 IPO price since listing, jumped $1.93 to close Monday at $9.50, after trading as high as $14.68.

For Servier, the Celladon deal is the latest in a string of R&D collaborations. Earlier this month, it inked a deal on allogeneic CAR T-cell therapies with Paris-based Cellectis SA. And late last year, Servier signed agreements with Xention Ltd. for atrial fibrillation candidate XEN-DO103 and with China’s Shanghai Institute of Materia Medical for co-development of a breast cancer candidate. (See BioWorld Today, Oct. 4, 2013, and Oct. 8, 2013.)