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Achillion's Danicopan Gets Breakthrough Therapy Tag for PHN

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Achillion Pharmaceuticals, Inc. announced that the FDA has granted Breakthrough Therapy designation to its oral factor D inhibitor, danicopan (previously ACH-4471), as a treatment for paroxysmal nocturnal hemoglobinuria (“PNH”), a rare, acquired blood disease.

The company is evaluating danicopan in combination with Alexion’s (ALXN - Free Report) C5 inhibitor, Soliris (eculizumab) in a phase II study in PNH patients who have sub-optimal response to Soliris alone.

FDA’s Breakthrough Therapy designation is granted to speed up the development and review of drugs that target serious or life-threatening conditions. Following the designation, danicopan will be eligible for accelerated approval and priority review depending upon certain criteria. The candidate also enjoys orphan drug status for this indication.

Shares of Achillion were up 2.8% on Sep 25 following the announcement. In fact, the company’s shares have soared 133% so far this year against the industry’s decrease of 3.2%.



Interim data from the phase II PNH study presented in May showed that addition of danicopan to Soliris nearly eliminated patients’ needs for blood transfusions. The Breakthrough Therapy designation was granted by the FDA based on this positive interim data. Top-line data is expected in the fourth quarter of 2019. The company has initiated an extension of the study to evaluate Alexion’s latest PNH drug, Ultomiris, in combination with danicopan.

The company anticipates to advance development of danicopan for PNH into phase III study in early 2020. Meanwhile, the company has completed enrollment in two phase II studies evaluating danicopan in C3 glomerulopathy, a rare kidney disorder.

The company is also developing its next-generation oral factor D inhibitors, ACH-5228 and ACH-5548, in two separate phase I studies targeting alternative pathway diseases. Data from the phase I study on ACH-5228 showed that the candidate achieved near complete and sustained alternative pathway inhibition in a 120 mg dose twice daily. The company expects to submit an investigational new drug application to the FDA to support phase II development of ACH-5228 in the fourth quarter of 2019.

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 treatments to the market.

Please note that there is growing interest in developing treatments for PNH and C3G. Apart from Alexion’s Soliris and the recently approved Ultomiris, complement inhibitor therapies for these indications are being developed by companies such as Ionis Pharmaceuticals (IONS - Free Report) , Novartis (NVS - Free Report) and Regeneron Pharmaceuticals among others.

Zacks Rank

Achillion currently carries a Zacks Rank #3 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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