Start-up Akebia Therapeutics Inc. is moving through preclinical studies with two programs licensed from Procter & Gamble Pharmaceuticals Inc.
Joseph Gardner, CEO of Cincinnati-based Akebia, said the company's founding was spurred by P&G Pharmaceuticals' decision last year to discontinue internal drug discovery efforts and instead focus on licensing later-stage product candidates.
The preclinical products are an oral hypoxia inducible factor prolyl hydroxylase (HIF-PH) inhibitor designed to increase natural production of erythropoietin in anemic patients, and a first-in-class program targeting HPTP-beta phosphatase to stimulate angiogenesis in the treatment of peripheral artery disease.
Gardner formerly managed medicinal chemistry, core technologies and intellectual property at P&G Pharmaceuticals, a division of Cincinnati-based Procter & Gamble Co. He was tasked with developing a strategy for out-licensing the company's early stage assets, but he decided several of those assets were worth holding onto. He teamed up with Triathlon Medical Ventures, and by early 2007, he and Robert Shalwitz, Akebia's co-founder and chief medical officer, had incorporated their new company and secured its pipeline.
The first program Akebia licensed from P&G targets the anemia market, where sales of injectable erythropoietin stimulating agents reached $10 billion last year. And Gardner believes there is significant room for a product with a good safety profile to expand the anemia market.
Amgen Inc., which markets Aranesp (darbepoetin alfa) and Epogen (epoetin alfa), and Johnson & Johnson subsidiary Ortho Biotech, which markets Procrit (epoetin alfa), recently found themselves in the center of an ESA firestorm. Clinical trial data pointing to an increased risk of adverse cardiovascular events, as well as an increased risk of tumor growth in some patients, has led to a black-box warning for the products, reimbursement restrictions and discussion of labeling changes. (See BioWorld Today, Sept. 10, 2007, and Sept. 13, 2007.)
Gardner said Akebia's approach may prove to be safer than existing ESAs. While ESAs involve the injection of recombinant erythropoietin, Akebia inhibits HIF-PH to increase the natural production of endogenous erythropoietin. Akebia has identified a lead compound and a series of backup molecules that have "good oral pharmacokinetics" and have demonstrated the ability to raise erythropoietin levels in animal models, Gardner said. He added that pharmacology work is almost complete and GLP/Tox studies are slated to begin early next year.
FibroGen Inc. also is developing small-molecule inhibitors of HIF-PH, with a compound in Phase II and several others in earlier development. Last year, Astellas Pharma Inc. signed a deal worth up to $815 million for exclusive development and marketing rights to the compounds in Europe, the Commonwealth of Independent States, the Middle East and South Africa. (See BioWorld Today, May 1, 2006.)
The second program Akebia licensed from P&G grew out of research on HPTP-beta phosphatase, which P&G scientists identified as a negative regulator of all angiogenic growth factors. The enzyme is expressed only in vascular endothelial cells, making it a "really exciting new target" for stimulating blood vessel growth in ischemia, peripheral artery disease, coronary artery disease, wound healing and other applications, Gardner said.
Patients with PAD often are treated with antiplatelet drugs like Plavix (clopidogrel bisulfate, Bristol-Myers Squibb Co. and Sanofi Aventis Group), which may lessen the risk of blood clots, or Pletal (cilostazol, Otsuka America Pharmaceutical Inc.), a phosphodiesterase III inhibitor that reduces intermittent claudication, a transient pain due to occluded peripheral arteries.
Akebia's approach differs from them in that it focuses on growing new blood vessels to replace those that have become damaged, rather than preventing clots. Companies such as GenVec Inc. have tried and failed to stimulate blood vessel growth with vascular endothelial growth factor (VEGF), but Gardner said the advantage of HPTP-beta phosphatase is that its inhibition up-regulates not just VEGF but also other angiogenic growth factors like angiopoietin.
Akebia has identified a lead compound and a backup in that program, and has seen "really beautiful animal data in hind limb ischemia," Gardner said. Pre-investigational new drug application studies are slated to begin later this year or early next year.
Akebia holds worldwide rights to both its programs, although Gardner said the company has "started conversations with potential partners for certain applications." Akebia also is busy raising a Series A financing round.
Seed funding of $1.1 million came from Triathlon Medical Ventures, Sigvion Capital, Blue Chip Venture Co., Fort Washington Capital Partners Group, CincyTechUSA and the Cincinnati Angels.