UPDATED: Akebia craters as adverse events cloud positive PhIIb anemia data

Procter & Gamble ($PG) spinoff Akebia Therapeutics rolled out a batch of promising Phase IIb efficacy data for its experimental anemia drug today, but investors who came in early this year to back the biotech's IPO in the belief that it holds the key to a potential blockbuster were dinged with signs of safety issues.

Investigators reported that after 20 weeks of treatment, 54.9% of the patients in the drug arm met the primary endpoint--maintaining the right level of hemoglobin, which disperses oxygen in the body--compared to only about 1 in 5 of the patients receiving a placebo. But the drug arm was also hit by a higher rate of serious adverse events--23.9% vs. 15.3%, with the most common SAEs being renal-related. One of the 49 SAEs in the treatment arm was drug-related and two more, which included the death of a patient, were possibly drug-related, according to Akebia. A total of 209 patients were recruited for the study, with full data to be discussed at an upcoming scientific meeting.

That's not exactly the efficacy/safety profile that investors were looking for. Akebia's shares ($AKBA) dropped 32% as investors got a chance to size up the results. The biotech moved from Cincinnati to the biotech hot spot in Cambridge, MA, earlier this year.

Despite the adverse events, Akebia execs say they are on track to launch a Phase III study next year that will determine the ultimate fate of this drug, which isn't the only such therapy in the industry pipeline. The compound belongs to a class of inhibitors of hypoxia-inducible factor prolyl hydroxylase drugs, which includes roxadustat (FG-4592) from FibroGen and partnered with AstraZeneca ($AZN) and Astellas ($ALPMY) and GlaxoSmithKline's ($GSK) 1278863. Just weeks ago FibroGen, a 2013 Fierce 15 company, filed for a $150 million IPO. Its two deals are worth up to $2.5 billion in milestones.

Akebia raised $100 million in its IPO, and company execs say they are well positioned to take this into a pivotal study.

Akebia CEO John Butler

"We are extremely pleased with the results of our Phase 2b study. This marks a very important milestone for Akebia and sets a clear path forward for a global Phase 3 program in renal anemia," said John Butler, CEO of Akebia. "The strength of these data reinforces the best-in-class potential of AKB-6548 in anemia related to CKD and brings us a significant step closer to achieving our goal of bringing innovative therapies to renal patients who are in need of new treatment options. We look forward to discussing these data with U.S. and European regulatory agencies in preparation for launching Phase 3 registration studies next year."

- here's the release

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