By Michael Fitzhugh, Staff Writer Aadi LLC.

A small new biotech company led by Abraxane (nab-paclitaxel) co-inventor Neil Desai, is in-licensing a once-shelved nanoparticle therapy from Celgene Corp. to develop it for oncology and cardiovascular indications. Celgene acquired the drug, ABI-009, in its 2010 acquisition of Abraxis Bioscience Inc. and retains an option to reacquire it in the new deal. Desai kept terms of the deal under wraps, but allowed that Celgene has invested a lot in the nanoparticle albuminbound (NAB) platform on which both Abraxane and ABI-009 are based, so the big biotech will be keeping close tabs on the project. Originated by Abraxis, ABI-009 is a NAB version of the mTOR inhibitor sirolimus, also known as rapamycin.

While Celgene tested the drug as a potential treatment for solid tumors in a phase I trial that began in December 2007, Thomson Reuters Cortellis records indicate that two years later, the drug was still listed as sitting in phase I development, and no further updates followed. “Celgene’s busy with a lot of things,” Desai told BioWorld Today. So is Desai. In addition to supervising ongoing enrollment for a combined phase I/II trial of the ABI-009 for bladder cancer, his new company has two more indications in mind: peripheral artery disease and pulmonary arterial hypertension. To advance all three indications through proof of concept in phase II, he’s now seeking additional financing. Desai shepherded Abraxane from the lab straight through clinical development as senior vice president of R&D at Abraxane.

He was also involved in early development of ABI- 009 during those days. Still convinced that the experimental drug offers a great opportunity to develop an improved mTOR inhibitor, he approached Celgene with a plan to rapidly develop the drug at the Pacific Palisades, Calif.-based company. “If you look at the mTOR inhibitor space, there are several drugs approved with varying profiles of toxicity and efficacy,” he said. “With the NAB platform, we can get great efficacy while at the same time having minimal toxicity.” ABI-009’s safety profile means it can be used in ways that other mTORs cannot, such as administering it locally in the bladder, he said. Furthermore, because bladder cancers are typically treated by urologists instead of oncologists, it’s “a good place to get into the hyper-competitive oncology market,” possibly offering an easier regulatory pathway, Desai said. “With just three new drugs approved for bladder cancer in the last 30 years, there’s a massive unmet need to develop new agents,” he said.

The drug has already completed one earlier phase I trial in patients with advanced nonhematologic malignancies, which showed the drug to be well tolerated with evidence of activity in heavily pretreated patients. The company is now recruiting for the combined phase I/II study in recurrent nonmuscle-invasive bladder cancer. As many as 80 percent of the almost 70,000 new bladder cancer cases in the U.S. each year are nonmuscle-invasive bladder cancers. More than half will recur, even after treatment with bacillus Calmette-Guerin delivered by catheter straight to the bladder, the standard first-line treatment.

With an annual death toll of 14,990, the cost per bladder cancer patient is among the highest of all cancers, noted Desai when applying for a grant to fund the study. Aadi recently began recruiting for the study, and has now enrolled three patients in the phase I study, and expects to complete enrollment by year’s end. The study is backed by a fast-track Small Business Technology Transfer grant and a supplemental grant from the National Cancer Institute, totaling about $641,000 in its first phase.

New York based Columbia University will be the primary clinical trial site. Vanderbilt University, of Nashville, Tenn., will provide another site. “It is important that we continue to search for new treatments for bladder cancer patients who are all too often faced with surgical removal of their bladder as their only option,” said James McKiernan, the study’s principal investigator, and interim chairman of the department of urology at Columbia University Medical Center and New York Presbyterian Hospital and professor of urologic oncology.

Next, AADi Bioscience plans to start a phase I trial of ABI-009 in the treatment of peripheral artery disease in the second half of this year. Desai said the company plans another phase I study in pulmonary arterial hypertension in early 2015, in collaboration with the University of Pittsburgh. Celgene did not return a request for comment. Its shares (NASDAQ:CELG) closed Tuesday at $147.65, up 27 cents.