Achillion Pharmaceuticals, Inc. (NASDAQ:ACHN) presented interim results from a phase 1 study with its novel small molecule factor D inhibitor, ACH-4471, at the 21st Congress of the European Hematology Association in Copenhagen, Denmark.

The e-poster entitled, “An Orally Administered Small Molecule Factor D Inhibitor (ACH-4471) For Treatment of PNH, C3G and Complement — Mediated Diseases: Interim Phase 1 Results In Healthy Volunteers” describes results from an ongoing phase 1 study led by Roderick B. Ellis-Pegler and Christian Schwabe of Auckland Clinical Studies Ltd, Auckland, New Zealand and a group of researchers from Achillion.

Across all four groups, ACH-4471 achieved peak plasma concentrations between 1 and 2.5 hours after oral dosing. Up to 100% inhibition of complement activity was achieved in all dose groups, and duration of inhibition was dose dependent. In addition, Group 4, which evaluated 1,200 mg of ACH-4471 given every twelve hours for 2 doses, achieved a median 99.5% inhibition (range 96 — 100%) of hemolysis at 24 hours.

“We are very encouraged by these data, which support our focus on developing potent, specific factor D inhibitor compounds, including ACH-4471, as a potentially novel approach to treating alternative pathway complement-mediated diseases such as PNH and C3G,” commented Milind Deshpande, Ph.D., President and Chief Executive Officer of Achillion. “Achieving sustained suppression of AP activity, as measured by the AP hemolysis and Wieslab assays, combined with modeling from these interim study results, suggest the potential for twice daily dosing to achieve sustained inhibition of AP activity.”

Study Design and Key Findings

The phase 1 study, initiated in February 2016, aims to assess the safety and tolerability of single ascending oral doses of ACH-4471 in healthy volunteers and to evaluate its pharmacokinetic (PK) and pharmacodynamic (PD) profile and PK/PD relationship as measured by the serum alternative pathway (AP) activity ex vivo.

In the study, 36 subjects have been dosed and evaluated. Trial participants were assigned to one of four groups:

  • Group 1: 200 mg, single dose (6 active + 6 placebo subjects)
  • Group 2: 600 mg, single dose (6 active + 2 placebo subjects)
  • Group 3: 1200 mg, single dose (6 active + 2 placebo subjects)
  • Group 4: 1200 mg x 2 doses (Q12H) (6 active + 2 placebo subjects)

For all groups, inhibition of serum AP activity was evaluated using the AP Wieslab and hemolysis assays. Blood samples were collected from days 1 through 7 to determine plasma concentrations. Trial participants were monitored through the last scheduled visit at day 28 for any adverse events.

The interim study data indicate ACH-4471 was well-tolerated at all dose levels examined with no safety trends noted. Evaluation of serum AP activity by ex vivo assessment suggests rapid inhibition of AP activity after oral dosing. Furthermore, the concentrations of Bb, the cleavage product of factor B by factor D, were determined for the assessment of the inhibitory effect of ACH-4471 on in vivo factor D activity. Bb levels were shown to decline after dosing with ACH-4471 with the lowest Bb levels observed at 16 hours post-dosing in Groups 3 and 4 and a gradual return to baseline by 48 hours after dosing.

Achillion plans to begin dosing in a phase 1 multiple-ascending dose study evaluating 14-days of dosing in healthy volunteers in June. The Company is also planning to initiate phase 2 studies of ACH-4471 in two complement-mediated diseases, PNH and C3G, by the end of 2016. (Original Source)

Shares of Achillion closed yesterday at $9.73, up $0.12 or 1.25%. ACHN has a 1-year high of $10.95 and a 1-year low of $5.57. The stock’s 50-day moving average is $8.76 and its 200-day moving average is $8.29.

On the ratings front, Achillion has been the subject of a number of recent research reports. In a report issued on May 31, Robert W. Baird analyst Brian Skorney reiterated a Buy rating on ACHN, with a price target of $10, which represents a slight upside potential from current levels. Separately, on May 24, Leerink Swann’s Joseph Schwartz reiterated a Hold rating on the stock and has a price target of $9.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Brian Skorney and Joseph Schwartz have a total average return of 2.9% and 16.6% respectively. Skorney has a success rate of 53.4% and is ranked #942 out of 3903 analysts, while Schwartz has a success rate of 55.0% and is ranked #102.

Achillion Pharmaceuticals, Inc. is a biopharmaceutical company that discovers, develops and commercializes innovative treatments for infectious diseases. The company is currently engaged in the development of antivirals for the treatment of chronic hepatitis C infection and resistant bacterial infections. Its products include ACH-3102, ACH-2684, Sovaprevir and ACH-3422. The company was founded on August 17, 1998 and is headquartered in New Haven, CT.