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Achillion Reports Positive Interim Data on Factor D Inhibitors

Zacks Equity Research

Achillion Pharmaceuticals, Inc. ACHN announced positive interim data from clinical studies evaluating its factor D inhibitors – ACH-4471, ACH-5228 and ACH-5548. Two phase II studies are evaluating the first-generation oral factor D inhibitor, ACH-4471, in patients with paroxysmal nocturnal hemoglobinuria (“PNH”) and C3 glomerulopathy (C3G). The company is developing its next-generation factor D inhibitors, ACH-5228 and ACH-5548, in two separate phase I studies.

Data from the mid-stage studies on ACH-4471 demonstrated proof of concept as monotherapy or in combination with a C5 Inhibitor in PNH patients. Moreover, ACH-4471 achieved proof of mechanism on C3G patients.  Achillion is currently enrolling patients in six and 12-month studies to evaluate ACH-4471 in C3G patients. The company is planning an end of phase II meeting in the fourth quarter of 2019.

Data from the early-stage studies evaluating ACH-5228 and ACH-5548 showed that these have the potential of higher alternative pathway inhibition along with a reduced dosing frequency. The company is planning to initiate a multiple ascending dose phase I study to evaluate ACH-5228 in January 2019.

Shares of Achillion rose 7.5% in after-market trading on Dec 17 following the news. However, the shares of the company have lost 16.7% year to date compared with the industry’s decline of 20.7%.


Achillion is focused on its factor D portfolio. The company is working on to develop oral inhibitors of complement Factor D. Targeted therapeutic areas include PNH, C3G, immune complex membranoproliferative glomerulonephritis, atypical hemolytic uremic syndrome and dry age-related macular degeneration.

Factor D, an essential protein of the complement pathway is integral to the human innate immune system. Though this area has commercial opportunity, the complement-mediated space is extremely crowded with many biotech companies working to introduce these treatments to the market.

We remind investors that another phase II study is evaluating ACH-4471 in combination with Alexion’s ALXN Soliris (eculizumab) for potentially long-term treatment of PNH patients, who are already being treated with Soliris.

Achillion Pharmaceuticals, Inc. Price


Achillion Pharmaceuticals, Inc. Price | Achillion Pharmaceuticals, Inc. Quote

Zacks Rank & Other Key Picks

Achillion currently carries a Zacks Rank #2 (Buy). Some other top-ranked stocks from the same sector include Genomic Health, Inc. GHDX and Asterias Biotherapeutics, Inc. AST. While Genomic Health sports a Zacks Rank #1 (Strong Buy), Asterias carries a Zacks Rank #2. You can see the complete list of today’s Zacks #1 Rank stocks here.

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