Managing Editor

Montreal-based Enobia Pharma Inc. padded its coffers via a $40 million private placement to support ongoing work on ENB-0040, an enzyme replacement therapy for rare genetic bone disorder hypophosphatasia (HPP).

Proceeds should carry the privately held firm "well into next year," said Robert Heft, president and CEO.

It's the first private placement for Enobia, which previously raised more than $100 million in venture capital, with the most recent round being a $50 million Series C closed in 2009. For the latest fundraising effort, Enobia decided to tap financial institutions and pharmaceutical firms. (See BioWorld Today, Aug. 11, 2009.)

To date, most of Enobia's focus has been on ENB-0040 (asfotase alfa), a recombinant fusion protein version of alkaline phosphatase, an enzyme involved in bone mineralization. HPP, which has been estimated to affect potentially 1 in 100,000 newborns, results from mutations in the gene for alkaline phosphatase and, depending on those mutations, the disease runs from mild conditions that affect the teeth to more severe forms that result in rickets and osteomalacia and, in the most severely affected can lead to death.

ENB-0040 is designed to replace the deficient enzyme, with modifications aimed at improving the pharmacokinetics compared to the natural enzyme while targeting the drug specifically to bone, Heft said.

Following promising preclinical studies, which showed that ENB-0040 significantly increased survival, prevented hypomineralization associated with HPP and appeared to heal bones that had been severely weakened by the disease, Enobia launched clinical programs. So far, there has been "a lot of excitement" around early data from two six-month Phase I/II studies, one in infants and one in pediatric patients ages 5 to 12, Heft told BioWorld Today, adding that updated results are expected this fall at the Society for the Study of Inborn Errors of Metabolism meeting in Geneva and at the American Society for Bone and Mineral Research meeting in San Diego.

Patients in both studies were rolled over into an extension trial. Those in the infant study include patients who have been on ENB-0040 for more than 2.5 years, Heft said.

Data from an ongoing Phase II trial in 19 adult and adolescent patients are expected in the second quarter of next year. That study is measuring the effect of ENB-0040 on HPP-related osteomalacia using trans-iliac crest bone biopsy as the primary endpoint.

Pending positive data from that trial, there's a chance Enobia could seek approval. Heft said the firm is in "multiple discussions" with regulatory agencies in the U.S. and Europe. "Our hope is that we will have a data package" that will be sufficient. For now, the firm holds all rights to the drug and has not yet finalized its strategy. But Enobia could easily take ENB-0040 into the market on its own in the main U.S. and European markets, as other small biotechs have done with ultra-orphan products.

ENB-0040 has orphan designation in both areas. And it recently added $1.2 million through an FDA Orphan grant for long-term treatment of infants and young children with severely debilitating or life-threatening HPP. That study currently has about 10 patients enrolled. Besides HPP, Enobia is hoping to build a pipeline of drugs for other rare bone disorders, and funds from the latest financing also will go toward an undisclosed preclinical program.

BofA Merrill Lynch acted as exclusive placement agent for the financing.